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Aura Biosciences

Nov 13
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Aura Biosciences Reports Third Quarter 2024 Financial Results and Business Highlights

By Aura Biosciences, Press Release, Private Companies
Press Release.

 

Positive Phase 2 End of Study Data with Bel-sar in Early-Stage Choroidal Melanoma; Ongoing Phase 3 CoMpass Trial Recently Received Authorization to Start Enrolling Patients in Europe

Multiple Clinical Complete Responses Observed with Single Low Dose of Bel-sar in Ongoing Phase 1 Trial in Non-Muscle Invasive Bladder Cancer (NMIBC); Phase 1 Expansion Preparation in Progress

Strong Cash Position Expected to Support Operations into 2H 2026

BOSTON, Nov. 12, 2024 (GLOBE NEWSWIRE) — Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing precision therapies for solid tumors designed to preserve organ function, today reported financial results for the third quarter ended September 30, 2024, and provided recent business highlights.

“This is a transformative time for our Company, as we presented the first positive data in NMIBC, which we believe provides clinical evidence of the potential of bel-sar in solid tumors beyond the eye,” said Elisabet de los Pinos, PhD, Chief Executive Officer of Aura Biosciences. “We believe that bel-sar’s innovative mechanism of action may provide the first immune-ablative treatment in bladder cancer, with the goal to potentially offer safe and durable responses with a focal approach that is easily delivered by urologists in the office.”

In addition to positive early data from an ongoing Phase 1 trial of bel-sar in patients with NMIBC, the Company also recently presented positive Phase 2 end of study data in early-stage choroidal melanoma and continues to progress the ongoing Phase 3 CoMpass trial.

“I am excited for bel-sar’s potential for patients who are diagnosed with indeterminate lesions or small choroidal melanoma where we currently have no good treatment options. We either wait for the disease to progress or treat with radiation, which leads to irreversible vision loss,” said Carol L. Shields, MD, Chief of the Ocular Oncology Service at Wills Eye Hospital and Professor of Ophthalmology at Thomas Jefferson University in Philadelphia. “If approved, bel-sar may represent the opportunity to treat choroidal melanoma at an earlier stage of medical intervention and set a new standard of care in a disease that has had no new therapies approved for decades.”

Recent Pipeline Developments 

Early-Stage Choroidal Melanoma
Early-stage choroidal melanoma represents an area of high unmet need with no drugs approved. The Company previously received Orphan Drug Designation from the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and Fast Track designation from the FDA for the treatment of early-stage choroidal melanoma.

Update on Ongoing Phase 3 CoMpass Trial: CoMpass is the first registration-enabling study in early-stage choroidal melanoma. The study is a global, Phase 3, randomized trial evaluating bel-sar treatment against a sham control arm and includes an enrichment strategy to enroll 100 patients with documented growth, an approach agreed upon under a Special Protocol Assessment (SPA) agreement with the FDA.

  • The Company recently received authorization from the EMA to commence the trial under the European Union (EU) Clinical Trial Regulation (CTR) process. This approval was later than anticipated due to a requirement for additional testing to support drug substance characterization that has been successfully met. This authorization permits the Company to start enrolling patients in the study in the EU. The study started enrolling in the United States in December 2023 and currently has sites activated in the United Kingdom, Australia and Canada.
  • To identify appropriate patients to meet the enrichment strategy of documented growth, the Company has enabled a pre-screening ‘run in’ period. Globally, since June 2024, investigators have registered over 100 patients in pre-screening as having met initial enrollment criteria for the study. The Company continues to monitor the overall timeline for the study, with European sites in the process of being activated.

The Company announced positive Phase 2 end of study results evaluating bel-sar as a first-line treatment for early-stage choroidal melanoma.

Clinical Data: The Phase 2 results demonstrated that bel-sar achieved an 80% tumor control rate (n=8/10) and durability of response at 12 months among Phase 3-eligible patients who received the therapeutic regimen. Visual acuity preservation was achieved in 90% of these patients. We believe the Phase 2 results are a significant achievement that support the design of the ongoing Phase 3 trial.

Safety Data: The safety profile of bel-sar was highly favorable in all participants. There were no treatment-related serious adverse events reported. Ocular treatment-related adverse events were mild (Grade 1).

Additional Ocular Oncology Indications:

In addition to early-stage choroidal melanoma, bel-sar is being explored for metastases to the choroid and cancers of the ocular surface. These three ocular oncology indications have a collective incidence of greater than 60,000 patients annually in the United States and Europe.

Metastases to the Choroid

The Company is initiating clinical development for bel-sar as a potential treatment for metastases to the choroid, an indication with high unmet medical need and no approved therapies. The Company aims to enroll the first patients in a Phase 2 trial in 2024. Metastases to the choroid represents the second potential ocular oncology indication for bel-sar, affecting approximately 20,000 patients annually in the United States and Europe. The Company previously received FDA Fast Track designation for bel-sar as a treatment in this indication.

Cancers of the Ocular Surface

The Company’s third potential ocular oncology indication is cancers of the ocular surface, which affect approximately 35,000 patients in the United States and Europe annually. The Company continues to advance its preclinical work designed to be IND-enabling in cancers of the ocular surface.

Bladder Cancer

The Company announced positive early data from an ongoing Phase 1 clinical trial of bel-sar in patients with NMIBC.

Clinical Data: In these early data from the first 8 patients treated with a single low dose of bel-sar with light activation, a clinical complete response was observed in 4 out of 5 patients with low grade disease; visual tumor shrinkage was observed on cystoscopy in 2 out of 3 patients with high grade disease where tumor cells were still present on histopathological evaluation. For this analysis, clinical complete response was defined as the absence of tumor cells on histopathologic evaluation. In addition, immune activation was noted in all patients with available immune staining in both treated target and untreated non-target bladder tumors with rapid infiltration of effector CD8+ and CD4+ T-cells within days after treatment. This data provides evidence of a bladder urothelial field effect, potentially indicating a broader immune response in the bladder beyond the target tumor in these patients.

Safety Data: In the safety analysis as of the September 9, 2024 data cut-off date (n=12), bel-sar was well-tolerated, with less than 10% Grade 1 and no Grade 2 or higher drug-related adverse events reported. No serious adverse events have been reported.

Future Development: The Company plans to continue development of bel-sar in bladder cancer with an initial focus on low-grade, intermediate risk NMIBC patients, through a planned trial expansion to test additional doses and treatment regimens with the opportunity to assess early durability of response at 3 months. In parallel, the Company is planning regulatory discussions on the design of the next trial with the goal of expediting clinical development in this patient population.

Recent Corporate Events

  • The Company announced the appointment of Sabine Doris Brookman-May, MD, FEBU as the Company’s Senior Vice President, Therapeutic Area Head Urologic Oncology.
  • The Company hosted a virtual ocular oncology investor event featuring Ivana Kim, MD (Mass Eye and Ear) and Prithvi Mruthyunjaya, MD, MHS (Stanford University Byers Eye Institute) to discuss the Phase 2 end of study data on Thursday, September 12, 2024. A replay of the webcast is available on the “Investors & Media” page under the “Events & Presentations” section of Aura’s website at https://ir.aurabiosciences.com/events-and-presentations.
  • The Company hosted a virtual urologic oncology investor event featuring Max Kates, MD (Johns Hopkins), Joe Jacob, MD (Syracuse University), Neal Shore, MD (Carolina Urologic Research Center) and Gary Steinberg, MD (RUSH University) to discuss the early Phase 1 data on Thursday, October 17, 2024. A replay of the webcast is available on the “Investors & Media” page under the “Events & Presentations” section of Aura’s website at https://ir.aurabiosciences.com/events-and-presentations.

Third Quarter 2024 Financial Results

  • As of September 30, 2024, the Company had cash and cash equivalents and marketable securities totaling $174.4 million. The Company believes its current cash and cash equivalents and marketable securities are sufficient to fund its operations into the second half of 2026.
  • Research and development expenses increased to $17.0 million for the three months ended September 30, 2024 from $15.4 million for the three months ended September 30, 2023 primarily due to manufacturing and development costs for bel-sar and higher personnel expenses related to growth of the Company.
  • General and administrative expenses increased to $6.2 million for the three months ended September 30, 2024 from $5.1 million for the three months ended September 30, 2023. General and administrative expenses include $1.6 million and $1.2 million of stock-based compensation for the three months ended September 30, 2024 and 2023, respectively. The increase was primarily driven by personnel expenses, as well as increases in general corporate expenses related to the growth of the Company.
  • Net loss for the three months ended September 30, 2024 was $21.0 million compared to $18.5 million for the three months ended September 30, 2023.

About Aura Biosciences

Aura Biosciences is a clinical-stage biotechnology company focused on developing precision therapies for solid tumors that aim to preserve organ function. Our lead candidate, bel-sar (AU-011), is currently in late-stage development for primary choroidal melanoma and in early-stage development in other ocular oncology indications and bladder cancer. Aura Biosciences is headquartered in Boston, MA. Our mission is to grow as an innovative global oncology company that positively transforms the lives of patients.

For more information, visit aurabiosciences.com. Follow us on X (formerly Twitter) @AuraBiosciences and visit us on LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “may,” “will,” “could,” “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions can be used to identify forward-looking statements. These forward-looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of various cancers; statements regarding Aura’s plans and expectations for its ongoing and future clinical trials of bel-sar in various oncology indications and the preclinical development of bel-sar in cancers of the ocular surface; statements regarding Aura’s beliefs and expectations for bel-sar’s ability to provide durable responses in bladder cancer patients and choroidal melanoma patients; statements regarding Aura’s expectations for an improved quality of life of patients after treatment with bel-sar and changes to the treatment paradigm for patients; statements regarding bel-sar’s potentially immune ablative effects; statements regarding bel-sar’s safety profile; statements regarding Aura’s beliefs and expectations for the high unmet medical need for an effective local treatment in ocular and urologic oncology; statements regarding Aura’s expectations for the estimated patient populations and related market opportunities for bel-sar; statements regarding the potential for regulatory approval of bel-sar; and statements regarding the Company’s expected cash runway.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s preclinical and clinical trials may not be predictive of future results in connection with future clinical trials; the risk that early or interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; the risk that governmental authorities may disagree with Aura’s clinical trial designs even where Aura has obtained agreement with governmental authorities on the design of such trials, such as the Phase 3 SPA agreement with the FDA; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; Aura’s ongoing and planned preclinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the United States Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

Investor Contact:
Alex Dasalla
Head of Investor Relations and Corporate Communications
IR@aurabiosciences.com

Media Contact:
Kimberly Ha
KKH Advisors
kimberly.ha@kkhadvisors.com
917-291-5744

Aura Biosciences, Inc.
Condensed Consolidated Statement of Operations and Comprehensive Loss
(Unaudited)
(in thousands, except share and per share amounts)
    Three Months Ended
September 30,		   Nine Months Ended
September 30,		  
    2024     2023   2024     2023  
Operating Expenses:  
Research and development $ 17,036 $ 15,428 $ 50,968 $ 44,952
General and administrative 6,196 5,060 17,341 15,256
Total operating expenses 23,232 20,488 68,309 60,208
Total operating loss (23,232 ) (20,488 ) (68,309 ) (60,208 )
Other income (expense):
Interest income, including amortization and accretion income 2,258 1,981 7,395 5,981
Other expense (25 ) (5 ) (83 ) (50 )
Total other income 2,233 1,976 7,312 5,931
Loss before income taxes (20,999 ) (18,512 ) (60,997 ) (54,277 )
Income tax provision, net (43 ) — (88 ) —
Net loss $ (21,042 ) $ (18,512 ) $ (61,085 ) $ (54,277 )
Net loss per common share—basic and diluted $ (0.42 ) $ (0.48 ) $ (1.23 ) $ (1.43 )
Weighted average common stock outstanding—basic and diluted 49,663,532 38,185,197 49,554,930 37,943,139
Comprehensive loss:
Net loss $ (21,042 ) $ (18,512 ) $ (61,085 ) $ (54,277 )
Other comprehensive items:
Unrealized gain (loss) on marketable securities 790 89 68 (62 )
Total other comprehensive income (loss) 790 89 68 (62 )
Total comprehensive loss $ (20,252 ) $ (18,423 ) $ (61,017 ) $ (54,339 )
Aura Biosciences, Inc.
Condensed Consolidated Balance Sheets
(Unaudited)
(in thousands, except share and per share amounts)
    September 30,
2024		     December 31,
2023		  
Assets  
Current assets:
Cash and cash equivalents $ 25,407 $ 41,063
Marketable securities 148,970 185,087
Restricted cash and deposits — 19
Prepaid expenses and other current assets 9,104 5,625
Total current assets 183,481 231,794
Restricted cash and deposits, net of current portion 768 768
Right of use assets – operating lease 17,744 18,854
Other long-term assets 22 509
Property and equipment, net 3,325 3,150
Total Assets   $ 205,340 $ 255,075
Liabilities and Stockholders’ Equity  
Current liabilities:
Accounts payable 1,991 1,787
Short-term operating lease liability 3,126 2,687
Accrued expenses and other current liabilities 9,597 7,883
Total current liabilities 14,714 12,357
Long-term operating lease liability 15,958 16,870
Total Liabilities   30,672 29,227
Commitments and Contingencies  
Stockholders’ Equity:  
Common stock, $0.00001 par value, 150,000,000 authorized at September 30, 2024 and December 31, 2023, and 49,778,861 and 49,350,788 shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively — —
Additional paid-in capital 522,454 512,617
Accumulated deficit (348,393 ) (287,308 )
Accumulated other comprehensive income 607 539
Total Stockholders’ Equity   174,668 225,848
Total Liabilities and Stockholders’ Equity   $ 205,340 $ 255,075
Oct 18
Love0

Multiple Clinical Complete Responses Demonstrated Following Single Low Dose Administration of Bel-sar in Patients with Non-Muscle-Invasive Bladder Cancer (NMIBC) in Ongoing Phase 1 Trial

By Aura Biosciences, Press Release, Private Companies
Press Release.

 

Multiple Clinical Complete Responses Demonstrated Following Single Low Dose Administration of Bel-sar in Patients with Non-Muscle-Invasive Bladder Cancer (NMIBC) in Ongoing Phase 1 Trial

October 17, 2024

Clinical Complete Responses Observed in 4 out of 5 Patients in Subset of Patients with Low Grade Disease; Evidence of Bladder Urothelial Field Effect in Non-Target Tumors

Favorable Safety Profile Observed; Only Grade 1 Drug-Related Adverse Events Reported in Less Than 10% of Patients

Aura Hosting Virtual Urologic Oncology Investor Event with Key Opinion Leaders at 4.30 pm ET Today

BOSTON, Oct. 17, 2024 (GLOBE NEWSWIRE) — Aura Biosciences, Inc. (NASDAQ: AURA), today announced positive early data from an ongoing Phase 1 clinical trial of bel-sar (AU-011) in patients with NMIBC. To date, the trial includes 13 patients, with the primary endpoints of evaluating the safety and feasibility of local administration of bel-sar alone (n=5) and bel-sar with light activation (n=8). The secondary endpoints are to evaluate biological activity and immune mediated changes in the tumor microenvironment (TME). 10 of 13 study participants had low grade disease, approximating the 70% incidence of this patient population among all NMIBC patients. The other 3 study participants had high grade disease. In patients receiving bel-sar with light activation (n=8), 4 out of 5 patients with low grade disease demonstrated a clinical complete response with no tumor cells remaining on histopathological evaluation. 2 out of 3 patients with high grade disease demonstrated visual tumor shrinkage observed on cystoscopy. Aura will host a Virtual Urologic Oncology Investor Event at 4:30 pm ET today.

“We are highly encouraged by this positive early data, which shows that bel-sar has the potential to be a transformative cancer treatment,” said Sabine Brookman-May, MD, FEBU, Senior Vice President, Therapeutic Area Head Urologic Oncology of Aura Biosciences. “A potentially differentiating aspect of this novel treatment is the rapid tumor response accompanied by an immune oncology (IO) effect such as a marked CD8+ T-cell infiltration observed in just a matter of days with a single low dose. We believe this could have the potential to translate into a durable response. In parallel with expanding the ongoing Phase 1 trial, we are preparing for a Phase 2 trial to further evaluate bel-sar’s clinical activity and durability of response.”

“Bel-sar has the potential to change the treatment paradigm for NMIBC,” said Neal Shore, MD, FACS, Medical Director, Carolina Urologic Research Center, AUC Urology Specialists. “Based on this early data, bel-sar’s positive clinical activity and evidence of a bladder urothelial field effect with a single dose, may position bel-sar to be the first immune ablative treatment option for early-stage bladder cancer patients delivered with an in-office procedure.”

Bel-sar is a virus-like drug conjugate, designed to have a dual mechanism of action, that induces direct tumor cell necrosis and elicits a robust and durable anti-tumor immune response.

Trial Design: The ongoing Phase 1 trial (NCT05483868) is a two-part, open-label clinical trial, designed to assess the safety and feasibility of bel-sar as a monotherapy. The study treatment is administered 7 to 12 days before the scheduled transurethral resection of bladder tumor (TURBT), the standard of care procedure. The participants are followed for safety monitoring over a 56-day period. The trial is also evaluating bel-sar’s biological activity with histopathological evaluation of tissue samples collected at the time of TURBT (regardless of tumor response) with evaluation of focal necrosis and immune changes in the tumor microenvironment. Part 1 (n=5) of the trial is complete, with patients receiving a single bel-sar dose without light activation. Part 2 (n=10) of the trial is ongoing. 8 patients with a confirmed tumor at time of treatment have received either 100ug or 200ug of bel-sar as a single dose. Of these 8 patients, 5 had low grade disease and 3 had high grade disease. 7 of these 8 patients had a history of recurrent bladder cancer and had undergone multiple TURBTs and adjuvant treatments such as Bacillus Calmette-Guerin (BCG), mitomycin, gemcitabine, cetrelimab and tamoxifen prior to trial enrollment. In the Phase 1 trial expansion, Aura plans to test additional doses and treatment regimens.

Safety Data: In the safety analysis as of the September 9, 2024 data cut-off date (n=12), bel-sar was well-tolerated, with less than 10% of patients reporting Grade 1 and no Grade 2 or higher drug-related adverse events reported. No serious adverse events have been reported. No significant differences between the light-activated and non-light activated cohorts have been observed.

Biological Activity: The data in these 8 patients receiving bel-sar with light activation showed clinical activity detectable as soon as 7 days after a single low dose of bel-sar with light activation. This was demonstrated by histopathological evidence of clinical complete response, necrosis, immune activation or visual tumor shrinkage observed on cystoscopy. For this analysis, “clinical complete response” was defined as the absence of tumor cells on histopathologic evaluation. Of the patients with low-grade disease, 4 out of 5 exhibited a clinical complete response (1 of 4 based on local pathology with central review ongoing), with no tumor cells detected in histopathological evaluation post-treatment in the target and in several non-target bladder tumors. 2 of 3 of the patients with high grade disease demonstrated visual tumor shrinkage observed on cystoscopy, while tumor cells were still present on histopathological evaluation. Immune activation was noted in all patients in both treated target and untreated non-target bladder tumors with infiltration of effector CD8+ and CD4+ T-cells (where immune staining was available). This data provides evidence of a bladder urothelial field effect with a single low dose of bel-sar with light activation, potentially indicating a broader immune response in the bladder beyond the target tumor in these patients.

Aura Virtual Urologic Oncology Investor Event

Aura will host a Virtual Urologic Oncology Investor Event featuring Max Kates, MD (Johns Hopkins), Joe Jacob, MD (Syracuse University), Neal Shore, MD (Carolina Urologic Research Center) and Gary Steinberg, MD (RUSH University) to discuss the early Phase 1 data on Thursday, October 17, 2024, at 4:30 pm Eastern Time. To register for the event, click here. A live question and answer session will follow the formal discussion.

The live webcast of Aura’s Virtual Urologic Oncology Investor Event will be available on the “Investors & Media” page under the “Events & Presentations” section of Aura’s website at https://ir.aurabiosciences.com/events-and-presentations, where a replay of the webcast will be archived for 90 days following the presentation date.

About Aura Biosciences

Aura Biosciences is a clinical-stage biotechnology company focused on developing precision therapies for solid tumors that aim to preserve organ function. Our lead candidate, bel-sar (AU-011), is currently in late-stage development for primary choroidal melanoma and in early-stage development in other ocular oncology indications and bladder cancer. Aura Biosciences is headquartered in Boston, MA. Our mission is to grow as an innovative global oncology company that positively transforms the lives of patients.

For more information, visit aurabiosciences.com. Follow us on X (formerly Twitter) @AuraBiosciences and visit us on LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “may,” “will,” “could,” “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions can be used to identify forward-looking statements. These forward-looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including bladder cancer; statements regarding Aura’s plans and expectations for the ongoing Phase 1 and future trials of bel-sar for bladder cancer; statements regarding Aura’s beliefs and expectations for bel-sar’s ability to provide durable responses in bladder cancer patients; statements regarding Aura’s expectations for an improved quality of life of patients after treatment with bel-sar and changes to the treatment paradigm for patients; statements regarding Aura’s beliefs and expectations for the high unmet medical need for an effective local treatment in urologic oncology; and statements regarding Aura’s expectations for the estimated patient populations and related market opportunities for bel-sar.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s preclinical and clinical trials may not be predictive of future results in connection with future clinical trials; the risk that early data from ongoing clinical trials may not be predictive of final data from completed clinical trials; the risk that governmental authorities may disagree with Aura’s clinical trial designs; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; Aura’s ongoing and planned preclinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the United States Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

Investor Contact:

Alex Dasalla

Head of Investor Relations and Corporate Communications

IR@aurabiosciences.com

Media Contact:

Kimberly Ha

KKH Advisors

kimberly.ha@kkhadvisors.com

917-291-5744

Sep 12
Love0

Aura Biosciences Reports Positive Phase 2 End of Study Results Evaluating Bel-sar as a First-Line Treatment for Early-Stage Choroidal Melanoma

By Aura Biosciences, Press Release, Private Companies
Press Release.

 

Aura Biosciences Reports Positive Phase 2 End of Study Results Evaluating Bel-sar as a First-Line Treatment for Early-Stage Choroidal Melanoma

Bel-sar Demonstrated 80% Tumor Control Rate, 90% Visual Acuity Preservation, and a Highly Favorable Safety Profile

Aura to Host a Virtual Ocular Oncology Investor Event Featuring Key Opinion Leaders Today at 8:00 am ET

BOSTON, Sept. 12, 2024 (GLOBE NEWSWIRE) — Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing precision therapies for solid tumors designed to preserve organ function, today announced positive Phase 2 end of study results evaluating bel-sar (AU-011) for the first-line treatment of early-stage choroidal melanoma (CM), a vision and life-threatening ocular cancer. The results were presented at The Retina Society Annual Meeting, on Thursday, September 12, 2024, in Lisbon, Portugal.

The Phase 2 study (NCT04417530) is an open-label, ascending single and repeat dose escalation trial in patients with early-stage CM (small CM and indeterminate lesions) designed to evaluate the safety, tolerability, and efficacy of up to three cycles of bel-sar treatment. The trial included both single and multiple ascending dose cohorts, with a total of 22 patients enrolled. Patients were closely monitored over a twelve-month follow-up period to assess tumor control, visual acuity preservation, and tumor growth rate.

Tumor Control and Visual Acuity Preservation

The Phase 2 results demonstrated that bel-sar achieved an 80% tumor control rate (n=8/10) among Phase 3-eligible patients who received the therapeutic regimen, with complete cessation of growth following treatment among responders (post-treatment average growth rate of 0.011 mm/yr among responders compared to 0.351 mm/yr prior to study entry; p<0.0001). Visual acuity preservation was achieved in 90% of these 10 patients. Importantly, 80% of these 10 patients were at high risk for vision loss with tumors close to the fovea or optic disc, highlighting the potential for vision preservation with this novel class of drugs. Of note, the current standard of care is radiotherapy, which leads to visual acuity of <20/200 (the cutoff for legal blindness) in the treated eye in up to 87% of patients.1 The Phase 2 results are a significant achievement considering the typically poor prognosis associated with choroidal melanoma, a rare and life-threatening ocular cancer, where there are no approved vision-preserving therapies to date.

Highly Favorable Safety Profile with No Dose-Limiting Toxicities

The safety profile of bel-sar was highly favorable in all participants regardless of dose. There were no treatment-related serious adverse events (SAEs) reported. Ocular treatment-related AEs (TRAEs) were mild (Grade 1), included anterior chamber inflammation (18%) or cell (9%) and resolved without sequelae. The vast majority (~70%) of the anterior chamber inflammation/cell events were self-limited, requiring no treatment, and resolved in a median of 6 days. For those events that did require treatment, topical steroid eye drops, administered for a median of 6 days, achieved complete resolution of the inflammation. Eye pain occurred in 9% of patients and was mild (Grade 1). Importantly, no treatment-related posterior inflammation events (no vitritis, choroiditis, retinitis, retinal pigment epithelium changes, or vasculitis) were reported.

“Many patients with early-stage choroidal melanoma currently face the difficult choice of whether to treat the cancer and risk losing their vision in the treated eye, or delay treatment and risk the tumor progressing,” said Dr. Ivana Kim, Director of the Ocular Melanoma Center, Mass Eye and Ear / Harvard Medical School. “The Phase 2 end of study data that I presented at The Retina Society Annual Meeting showed 80% tumor control rate, 90% vision preservation, and a highly favorable safety profile in early-stage CM. Bel-sar has the potential to become the first treatment that achieves the dual goals of treating the tumor while also preserving vision, which could change the treatment paradigm for patients with this disease.”

“We believe these Phase 2 results provide clinical evidence for bel-sar as a potential vision-sparing, first-line treatment option for patients with early-stage CM,” said Dr. Jill Hopkins, Chief Medical Officer and President of Research and Development at Aura Biosciences. “Bel-sar is potentially a first-in-class novel therapy and we are excited to continue to advance this program, which is currently enrolling patients in our ongoing global Phase 3 CoMpass trial.”

Aura received written agreement from the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the design and planned analysis of the global Phase 3 CoMpass trial indicating concurrence by the FDA with the adequacy of the study, if successful, to address the objectives necessary to support Aura’s planned biologics license application submission. Aura Biosciences is focused on enhancing treatment options and improving outcomes for patients with CM and other cancers.

Aura Virtual Ocular Oncology Investor Event

Aura will host a virtual ocular oncology investor event featuring Dr. Ivana Kim, MD (Mass Eye and Ear) and Dr. Prithvi Mruthyunjaya, MD, MHS (Stanford University Byers Eye Institute) to discuss the Phase 2 end of study data on Thursday, September 12, 2024, at 8:00 am Eastern Time. To register for the event, click here. A live question and answer session will follow the formal discussion.

The live webcast of Aura’s virtual ocular oncology investor event will be available on the “Investors & Media” page under the “Events & Presentations” section of Aura’s website at https://ir.aurabiosciences.com/events-and-presentations, where a replay of the webcast will be archived for 90 days following the presentation date.

About Aura Biosciences

Aura Biosciences is a clinical-stage biotechnology company focused on developing precision therapies for solid tumors that aim to preserve organ function. Our lead candidate, bel-sar (AU-011), is currently in late-stage development for primary choroidal melanoma, and in early-stage development in other ocular oncology indications and bladder cancer. Aura Biosciences is headquartered in Boston, MA. Our mission is to grow as an innovative global oncology company that positively transforms the lives of patients.

For more information, visit aurabiosciences.com. Follow us on X (formerly Twitter) @AuraBiosciences and visit us on LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward looking statements. Words such as “may,” “will,” “could,” “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. These forward-looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including early-stage CM and other oncology indications; statements regarding Aura’s expectations for the Phase 3 clinical trial of bel-sar for early-stage CM; statements regarding Aura’s expectations for an improved quality of life of patients after treatment with bel-sar; statements regarding Aura’s beliefs and expectations for the urgent need for an effective local treatment in ocular and other oncology indications to preserve organ function; statements regarding Aura’s expectations for the estimated patient populations and related market opportunities for bel-sar; and the potential for regulatory approval of bel-sar.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s preclinical and clinical trials may not be predictive of future results in connection with future clinical trials; the risk that interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; the risk that governmental authorities may disagree with Aura’s clinical trial designs, even where Aura has obtained agreement with governmental authorities on the design of such trials, such as the Phase 3 SPA agreement with the FDA; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; Aura’s ongoing and planned preclinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the United States Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

Investor Contact:
Alex Dasalla
Head of Investor Relations and Corporate Communications
IR@aurabiosciences.com

Media Contact:
Kimberly Ha
KKH Advisors
kimberly.ha@kkhadvisors.com
917-291-5744

Dec 07
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Aura Biosciences Announces First Patient Dosed in Global Phase 3 CoMpass Trial

By Aura Biosciences, Press Release, Private Companies
Press Release.

 

Aura Biosciences Announces First Patient Dosed in Global Phase 3 CoMpass Trial Evaluating the Safety and Efficacy of Belzupacap Sarotalocan (Bel-sar) for First-Line Treatment of Early-Stage Choroidal Melanoma

 

BOSTON–(BUSINESS WIRE)– Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the first patient has been dosed in the global Phase 3 CoMpass trial evaluating the safety and efficacy of bel-sar for the first-line treatment of adult patients with early-stage choroidal melanoma.

“Dosing of the first patient in this global Phase 3 trial is a major milestone for Aura, and for patients with early-stage choroidal melanoma, a life-threatening rare disease with no approved targeted therapies,” said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura. “With the SPA agreement from the FDA, and a strong endorsement from the ocular oncology community, we continue to move bel-sar closer to potentially being approved as a first-line vision preserving treatment for patients living with this disease.”

The CoMpass trial is designed as a superiority trial comparing bel-sar versus a sham control. The trial is a global Phase 3, randomized, multi-center, masked study, intended to enroll approximately 100 patients randomized 2:1:2 to receive high dose regimen of bel-sar, low dose regimen of bel-sar with suprachoroidal (SC) administration or a sham control. Aura received written agreement from the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the overall design of the CoMpass trial.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage biotechnology company developing VDCs, a novel class of therapies, for the treatment of multiple oncology indications. Aura’s lead VDC candidate, belzupacap sarotalocan (bel-sar; AU-011), consists of a virus-like particle conjugated with an anti-cancer agent. Bel-sar is designed to selectively target and destroy cancer cells and activate the immune system with the potential to create long-lasting, anti-tumor immunity. Bel-sar is currently in development for ocular cancers, and Aura is enrolling patients in the global Phase 3 trial evaluating first-line treatment of early-stage choroidal melanoma, a vision- and life-threatening form of eye cancer where the standard of care with radiotherapy leaves patients with severe comorbidities, including significant vision loss. Aura plans to pursue development of bel-sar across its ocular oncology franchise including for the treatment of patients with choroidal metastasis. In addition, leveraging Aura’s technology platform, Aura is developing bel-sar more broadly across multiple cancers, including in patients with non-muscle invasive and muscle invasive bladder cancer. Aura is headquartered in Boston, MA.

For more information, visit aurabiosciences.com, or follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward looking statements. Words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. These forward looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including choroidal melanoma, choroidal metastasis, muscle invasive bladder cancer and non-muscle invasive bladder cancer; any express or implied statements regarding Aura’s expectations for the Phase 3 clinical trial of bel-sar for early-stage choroidal melanoma; and the potential approvability of bel-sar.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s clinical trials may not be predictive of future results in connection with future clinical trials; the risk that interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; the risk that governmental authorities may disagree with Aura’s clinical trial designs, even where Aura has obtained agreement with governmental authorities on the design of such trials, such as the Phase 3 SPA agreement with FDA; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; Aura’s ongoing and planned preclinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties, and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

View source version on businesswire.com: https://www.businesswire.com/news/home/20231207348086/en/

Contacts

Investor and Media Contact:

Alex Dasalla
Head of Investor Relations and Corporate Communications
adasalla@aurabiosciences.com

Nov 06
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Aura Biosciences Receives FDA Agreement for CoMpass Phase 3 Clinical Trial

By Aura Biosciences, Press Release, Private Companies
Press Release.

 

Aura Biosciences Receives FDA Agreement Under Special Protocol Assessment (SPA) for CoMpass Phase 3 Clinical Trial of Belzupacap Sarotalocan (Bel-sar) in Early-stage Choroidal Melanoma
November 6, 2023

Positive Clinical Efficacy Updates of Bel-sar for Early-Stage Choroidal Melanoma from the Ongoing Phase 2 Clinical Trial with Suprachoroidal Administration Presented at AAO 2023

Preliminary Data from Phase 1 Trial in Bladder Cancer – First Patient Utilizing a Single Dose of Bel-sar with Light Activation Demonstrated a Clinical Complete Response

BOSTON–(BUSINESS WIRE)–Nov. 6, 2023– Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced it has received agreement from the U.S. Food and Drug Administration (FDA) under an SPA for the design and planned analysis of CoMpass, the Company’s global Phase 3 clinical trial of bel-sar for the first-line treatment of adult patients with early-stage choroidal melanoma (CM). The Company also announced the presentation of positive Phase 2 safety and efficacy data of bel-sar with 90% of patients at twelve months of follow-up evaluating two key clinical endpoints: tumor control and visual acuity preservation using suprachoroidal (SC) route of administration for the first-line treatment of adult patients with early-stage CM. The results were presented at the American Academy of Ophthalmology (AAO) 2023 Annual Meeting in San Francisco, California.

“This written agreement from the FDA is consistent with our regulatory strategy and reaffirms that the design and planned analysis of the CoMpass Phase 3 clinical trial, if successful, adequately address the objectives necessary to support a biologics license application submission for bel-sar for the treatment of early-stage CM,” said Dr. Jill Hopkins, Chief Medical Officer and President of R&D of Aura Biosciences. “We are excited with the momentum in the ocular oncology community to participate in what would be the first ever registration-enabling clinical trial for early-stage CM.”

“The Phase 2 data presented today, with 90% of patients at twelve months follow-up, show results that are highly consistent with and strongly support the assumptions for the design of the CoMpass trial. We have observed 80% tumor control and 90% visual acuity preservation for patients that have been treated with the therapeutic regimen of bel-sar and meet the Phase 3 enrollment criteria. The safety profile continues to be favorable with no treatment-related serious adverse events or significant adverse events. This is very encouraging as most of these patients had tumors close to the fovea or optic disk and would have likely experienced severe and irreversible vision loss with the current standard of care with radiotherapy,” said Dr. Carol Shields, Chief of the Ocular Oncology Service at Wills Eye Hospital and Professor of Ophthalmology at Thomas Jefferson University (USA). “We are excited for the possibility to have for the first time a vision preserving therapy, expanding the possibility to treat patients earlier than we do today.”

Agreement from the FDA Under an SPA for the Global Phase 3 CoMpass Trial:

The Company received written agreement from the FDA under an SPA for the design and planned analysis of the Global Phase 3 CoMpass trial indicating concurrence by the FDA with the adequacy of the study, if successful, to address the objectives necessary to support the Company’s planned biologics license application submission. The Phase 3 trial is designed as a superiority trial comparing bel-sar versus sham. The trial is a global, multi-center, masked study, and it is intended to enroll approximately 100 patients randomized 2:1:2 to receive high dose regimen of bel-sar with SC administration, low dose regimen of bel-sar with SC administration, or a sham control. The primary endpoint is time to tumor progression, and the first key secondary endpoint is a composite time to event analysis that will compare the tumor control and visual acuity of the bel-sar high dose regimen to sham when the last patient completes their 15 months of follow up. The trial is powered at greater than 90%. The Company is on track to dose the first patient in Q4 2023.

Updated Safety and Efficacy Data from the Phase 2 Trial with SC Administration

The Phase 2 trial (NCT04417530) is assessing the safety and preliminary efficacy of single- and multiple ascending-doses of bel-sar up to three cycles of treatment via SC administration for the first-line treatment of early-stage CM. A total of 22 adult patients have been enrolled in the trial including the single dose Cohorts 1-3 (n=6) and multiple dose escalation Cohorts 4-6 (n=16). Cohorts 5 and 6 (n=13) received up to three cycles of therapy, which was considered the therapeutic regimen for evaluation. One patient in Cohort 5 (n=3) received two cycles of therapy and two patients in Cohort 5 received three cycles of therapy (40 μg/dose). All patients from Cohort 6 (n=10) were assigned to receive three cycles of therapy at the highest dose (80 μg/dose). One patient from Cohort 6, who discontinued after one cycle due to unrelated serious adverse events (SAEs), is not included in the analysis. All patients in Cohorts 5 and 6 had active tumor growth at study entry, as an enrichment strategy to evaluate preliminary efficacy. This group of patients with active growth treated at the therapeutic regimen of three cycles was evaluated for tumor control, visual acuity preservation and tumor growth rate as the defined clinical endpoints to evaluate preliminary efficacy. The results, with 90% of patients at twelve months of follow-up who received three cycles of therapy in Cohorts 5 and 6, and who match the criteria for the global Phase 3 trial, showed a tumor control rate of 80% (8/10) and the visual acuity preservation rate was 90% (9/10). The majority of patients being at high-risk for vision loss with tumors close to the fovea or optic disk. For the 80% of patients that responded, data showed a statistically significant reduction in tumor growth rate (-0.382 mm/yr, p = <0.0001) compared to each patient’s documented growth rate at study entry. The overall tolerability profile of bel-sar was favorable, with no dose-limiting toxicities, treatment-related SAEs or significant AEs reported as of August 3, 2023. There was no posterior inflammation and only mild anterior inflammation (Grade 1) in ~18% of the patients which was self-limited or resolved with a short course of topical steroids. Treatment-related AEs were predominantly mild and resolved without sequalae. We believe these updated results indicate that bel-sar may offer a targeted, vision preserving therapy for the first-line treatment of primary CM, where 80% of patients are diagnosed at an early stage and have no approved therapies to date.

The presentation can be accessed on the Company’s website here.

Preliminary Data from Phase 1 Trial in Bladder Cancer

The trial has completed enrollment of the cohort that received bel-sar injection without light activation. Protocol mandated safety review found no safety issues and the study has proceeded to the bel-sar injection plus light activation cohorts. Preliminary data from the first patient in the light activated cohort of the trial, utilizing a single dose of bel-sar with light activation, demonstrated a clinical complete response demonstrated by absence of cancer cells on histopathology with evidence of extensive necrosis and immune activation.

Bel-sar is a novel investigational agent designed with a dual mechanism of action that includes targeted cytotoxicity and immune activation. The ongoing Phase 1 multi-center, open-label clinical trial is expected to enroll approximately 19 adult patients. The trial is designed to assess the safety and tolerability of bel-sar as a single agent. The trial will provide histopathological evaluation after the local treatment to assess bel-sar’s biological activity. In addition, the FDA has allowed an amendment to the protocol of the Company’s ongoing Phase 1 trial evaluating bel-sar, allowing the inclusion of adult patients with muscle invasive bladder cancer in addition to non-muscle invasive bladder cancer. The Company expects to provide more data mid-2024.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage biotechnology company developing VDCs, a novel class of therapies, for the treatment of multiple oncology indications. Aura’s lead VDC candidate, belzupacap sarotalocan (bel-sar; AU-011), consists of a virus-like particle conjugated with an anti-cancer agent. Bel-sar is designed to selectively target and destroy cancer cells and activate the immune system with the potential to create long-lasting, anti-tumor immunity. Bel-sar is currently in development for ocular cancers, and Aura has initiated activities for the global Phase 3 trial evaluating first-line treatment of early-stage choroidal melanoma, a vision- and life-threatening form of eye cancer where the standard of care with radiotherapy leaves patients with severe comorbidities, including significant vision loss. Aura plans to pursue development of bel-sar across its ocular oncology franchise including for the treatment of patients with choroidal metastasis. In addition, leveraging Aura’s technology platform, Aura is developing bel-sar more broadly across multiple cancers, including in patients with non-muscle invasive and muscle invasive bladder cancer. Aura is headquartered in Boston, MA.

For more information, visit aurabiosciences.com, or follow us on Twitter and LinkedIn

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward looking statements. Words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. These forward looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including choroidal melanoma, muscle invasive bladder cancer, non-muscle invasive bladder cancer and choroidal metastasis; any express or implied statements regarding the Company’s expectations for the Phase 2 and Phase 3 clinical trials of bel-sar for early-stage choroidal melanoma and the Phase 1 trial of bel-sar for muscle invasive bladder cancer and non-muscle invasive bladder cancer; and the potential approvability of bel-sar; the Phase 2 trial of bel-sar for choroidal metastasis.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s clinical trials may not be predictive of future results in connection with future clinical trials; the risk that interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; the risk that governmental authorities may disagree with Aura’s clinical trial designs, even where Aura has obtained agreement with governmental authorities on the design of such trials, such as the Phase 3 SPA agreement with FDA; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; Aura’s ongoing and planned preclinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties, and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

ACTIVE/125869873.9

Investor and Media Contact:
Alex Dasalla
Head of Investor Relations and Corporate Communications
adasalla@aurabiosciences.com

Mar 29
Love0

Eloxx Pharmaceuticals Announces Submission of Investigational New Drug (IND) Application for ZKN-013

By Aura Biosciences, Press Release, Private Companies, Publicly Listed
Press Release.

 

  • ZKN-013 in development for treatment of recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB)
  • Additional IND filing for ZKN-013 for treatment of familial adenomatous polyposis (FAP) expected in first half of 2023
  • Recent preclinical results have provided additional support for ZKN-013 in RDEB and FAP

WATERTOWN, Mass., March 28, 2023 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced the submission of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration for ZKN-013 for the treatment of recessive Dystrophic Epidermolysis Bullosa (RDEB) with nonsense mutations. RDEB is a rare skin disease characterized by mutations in Collagen7 gene.

“This IND application is an important milestone towards providing a treatment option for patients with RDEB and JEB, as there are currently no approved disease-modifying treatments,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “We look forward to advancing ZKN-013, our lead TURBO-ZMTM based molecule, into clinical development, as we believe it has the potential to have a positive impact on the treatment of these two painful and debilitating diseases.”

Recent preclinical results demonstrated read-through activity of ZKN-013 in multiple COL7 genotypes across multiple RDEB patient derived fibroblasts and keratinocytes. Read-through activity resulted in up to an 18-fold increase in full-length COL VII protein levels. Prolonged treatment with ZKN-013 further increased COL VII protein levels. Functionality of the restored full-length COL VII protein was confirmed. These results have been accepted for presentation at an upcoming medical conference.

An additional IND filing for ZKN-013 for the treatment of familial adenomatous polyposis (FAP) is planned in the first half of 2023. FAP, a rare inherited disease with no approved drug therapies, is characterized by proliferation of colon polyps. Eloxx is targeting a subset of patients that have nonsense mutations in the Adenomatous Polyposis Coli (APC) gene that is truncated in these patients.

In January 2023, Eloxx published positive results from a study in the APCMin (multiple intestinal neoplasia) model evaluating the potential of ZKN-013 to treat FAP. The APCMin mouse is a translationally validated model for drug development for FAP. In the APCMin model, treatment with ZKN-013 demonstrated a decrease in intestinal polyps and adenomas, resulting in increased survival. The publication also included in vitro and in vivo demonstrating that ZKN-013 inhibited the growth of human colon carcinoma cells with APC nonsense mutations, and promoted read through of premature stop codons in the APC gene, leading to functional restoration of full-length APC protein.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZMTM chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ELX-02 for the treatment of CF patients with nonsense mutations. In addition, ELX-02 has also been granted Orphan Drug Designation for the treatment of CF patients with nonsense mutations by the FDA and orphan medicinal product designation by the European Commission. ELX-02 is in clinical development, focusing on cystic fibrosis (US Trial NCT04135495, EU/IL Trial NCT04126473). Eloxx also has preclinical programs focused on select rare diseases, including inherited diseases, cancer caused by nonsense mutations, kidney diseases, including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.

For more information, please visit www.eloxxpharma.com.

Forward-looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of present and historical facts contained in this press release, including without limitation, the expected timing of trials of our product candidates and the potential of our product candidate to treat nonsense mutations are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,”

“believe,” “estimate,” “predict,” “potential,” “seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections based on information currently available to us. Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical studies are not always indicative of positive clinical results; the scope, rate and progress of our preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development; the impact of the global COVID-19 pandemic on our clinical trials, operations, vendors, suppliers, and employees; our ability to obtain the capital necessary to fund our operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financial in the future through product licensing, public or private equity or debt financing or otherwise; general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2022, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website atwww.sec.gov and the “Financials&Filings” page of our website at https://investors.eloxxpharma.com/financials-filings.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Contact

Investors
John Woolford john.woolford@westwicke.com 443.213.0506

Media
Laureen Cassidy laureen@outcomescg.com

SOURCE: Eloxx Pharmaceuticals, Inc.

Feb 16
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Aura Biosciences Announces Positive Interim Phase 2 Safety and Efficacy Data of Belzupacap Sarotalocan (Bel-sar) for the First-Line Treatment of Patients with Early-Stage Choroidal Melanoma with Suprachoroidal Administration

By Aura Biosciences, Press Release, Private Companies, Publicly Listed
Press Release.

 

Aura Biosciences Announces Positive Interim Phase 2 Safety and Efficacy Data of Belzupacap Sarotalocan (Bel-sar) for the First-Line Treatment of Patients with Early-Stage Choroidal Melanoma with Suprachoroidal Administration

Boston, USA, February 16 2023 – Aura Biosciences, Inc. (“Aura”) (Nasdaq: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the presentation of positive interim Phase 2 safety and efficacy data of bel-sar with 9-10 months of follow up evaluating two key clinical endpoints: tumor control and visual acuity preservation using the suprachoroidal (SC) route of administration for the first-line treatment of patients with early-stage choroidal melanoma (indeterminate lesions and small choroidal melanoma (IL/CM)). The results were presented at the Macula Society 46th Annual Meeting held February 15-18, 2023, in Miami, FL.

“The data presented today with an average of nine months of follow up for patients treated with three cycles of therapy, show an excellent response to the therapy with 89-100% tumor control. In addition, the safety profile to date has been favorable with only one patient losing visual acuity and no treatment-related SAEs or significant AEs, which is encouraging given that the majority of these patients had tumors close to the fovea or optic disk and would have likely experienced severe and irreversible vision loss with the current standard of care with radiotherapy,” said Dr. Ivana Kim, Director of the Ocular Melanoma Center, Massachusetts Eye and Ear. “These latest results strongly support the potential of bel-sar to be used as a first line treatment option for patients with early-stage choroidal melanoma.”

“We are excited with the interim efficacy data of the Phase 2 study which strongly supports the assumptions for the success of the global Phase 3 trial,” said Dr. Cadmus Rich, Chief Medical Officer of Aura Biosciences. “Collectively, we believe these interim data provide strong confidence to support the launch of a global Phase 3 trial which is on track to begin enrollment this year.”

The presentation can be accessed on the Company’s website: link

Updated Safety and Efficacy Data from the Ongoing Phase 2 Trial with SC Administration

This Phase 2 trial (NCT04417530) is assessing the safety and preliminary efficacy of single- and multiple ascending-doses of bel-sar up to three cycles of treatment via SC administration for the first-line treatment of early-stage choroidal melanoma. A total of 20 adult patients have been enrolled in the trial including the single dose Cohorts 1-3 (n=6) and multiple dose escalation Cohorts 4-6 (n=14). Cohorts 5 and 6 received up to three cycles of therapy, which was considered the therapeutic regimen for evaluation. One patient in Cohort 5 (n=3) received two cycles of therapy and two patients in Cohort 5 received three cycles of therapy (40 μg/dose). All patients from Cohort 6 (n=8) were assigned to receive three cycles of therapy at the highest dose (80 μg/dose). One patient from Cohort 6, who discontinued after one cycle due to unrelated serious adverse events (SAEs), is not included. All patients in Cohorts 5 and 6 had active growth at study entry, as an enrichment strategy to evaluate preliminary efficacy. This group of patients with active growth treated at the therapeutic regimen of three cycles was evaluated for tumor growth rate, tumor control, and visual acuity preservation as the defined clinical endpoints to evaluate preliminary efficacy. The results, with an average of nine months of follow up in patients who received three cycles of therapy in Cohorts 5 and 6, and who match the criteria for the planned global Phase 3 trial, showed a statistically significant reduction in the tumor growth rate (-0.289 mm/yr, p = <0.0001) compared to each patient’s documented growth rate at study entry, and a 100% (8/8) tumor control rate. In addition, the visual acuity preservation rate was 88% (7/8) in these cohorts, with the majority of patients being at high-risk for vision loss with tumors close to fovea or optic disk. The overall tolerability profile of bel-sar was generally favorable, with no dose-limiting toxicities, treatment-related SAEs or significant AEs reported as of January 10, 2023. There was no posterior inflammation and only mild anterior inflammation (Grade 1) in 20% of the patients. Treatment-related AEs were predominantly mild and resolved without sequalae. We believe these interim results indicate that bel-sar may offer a targeted, vision preserving therapy for the first-line treatment of primary CM, where 80% of patients are diagnosed early and have no approved therapies to date.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage biotechnology company developing virus-like drug conjugates (VDCs), a novel class of therapies, for the treatment of multiple oncology indications. Aura’s lead VDC candidate, belzupacap sarotalocan (bel-sar; AU-011), consists of a virus-like particle conjugated with an anti-cancer agent. Bel-sar is designed to selectively target and destroy cancer cells and activate the immune system with the potential to create long-lasting anti-tumor immunity. Bel-sar is currently in development for ocular cancers, and Aura has initiated activities for the global Phase 3 trial evaluating first-line treatment of early-stage choroidal melanoma, a vision- and life-threatening form of eye cancer where standard of care with radiotherapy leaves patients with severe comorbidities, including major vision loss. Aura plans to pursue development of bel-sar across its ocular oncology franchise including for the treatment of patients with choroidal metastasis. In addition, leveraging Aura’s technology platform, Aura is developing bel-sar more broadly across multiple cancers, including in patients with non-muscle invasive bladder cancer (NMIBC). Aura is headquartered in Boston, MA.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward looking statements. Words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. These forward looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including choroidal melanoma, non-muscle invasive bladder cancer and choroidal metastases; any express or implied statements regarding the Company’s expectations for the Phase 2 and Phase 3 clinical trials of bel-sar for early-stage choroidal melanoma; and Aura’s expectations regarding the estimated patient populations and related market opportunities for bel-sar.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, an improved quality of life of patients after treatment with bel-sar; a potential paradigm shift in the approach to the treatment of choroidal melanoma; the urgent need for a vision preserving targeted therapy; the potential of bel-sar compared to the existing standard of care for patients with choroidal melanoma; uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s clinical trials may not be predictive of future results in connection with future clinical trials; the risk that interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; risks, assumptions and uncertainties regarding the impact of the continuing COVID-19 pandemic on Aura’s business, operations, strategy, goals and anticipated timelines; Aura’s ongoing and planned pre-clinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties, and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

 

Contacts:

Alex Dasalla
Head of Investor Relations and Corporate Communications
adasalla@aurabiosciences.com

Argot Partners
Matthew DeYoung
aura@argotpartners.com

Source: Aura Biosciences, Inc.

Dec 01
Love0

Aura Biosciences Announces Pricing of Public Offering of Common Stock

By Aura Biosciences, Press Release, Publicly Listed

Press Release.

BOSTON–(BUSINESS WIRE)– Aura Biosciences, Inc. (“Aura”) (Nasdaq: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the pricing of an underwritten public offering of 6,700,000 shares of its common stock at a price to the public of $12.00 per share. The gross proceeds from the offering to Aura are expected to be $80.4 million, before deducting underwriting discounts and commissions and other offering expenses. The offering is expected to close on or about December 5, 2022, subject to customary closing conditions. In addition, Aura has granted the underwriters a 30-day option to purchase up to 1,005,000 additional shares of common stock at the public offering price, less the underwriting discount.

SVB Securities, Cowen and Evercore ISI are acting as joint bookrunning managers for the offering and JMP Securities, a Citizens Company, and BTIG are acting as co-managers for the offering.

A shelf registration statement relating to the shares of common stock offered in the public offering described above was filed with the Securities and Exchange Commission (the “SEC”) on November 1, 2022 and declared effective by the SEC on November 7, 2022. The offering is being made only by means of a written prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement and accompanying prospectus relating to the offering has been filed with the SEC and is available on the SEC’s website at www.sec.gov. A final prospectus supplement and accompanying prospectus will be filed with the SEC. When available, copies of the final prospectus supplement and the accompanying prospectus may also be obtained by contacting SVB Securities LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, by telephone at (800) 808-7525, ext. 6105, or by email at syndicate@svbsecurities.com; Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, by telephone at (833) 297-2926, or by email at PostSaleManualRequests@broadridge.com; or Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, New York 10055; by telephone at (888) 474-0200, or by email at ecm.prospectus@evercore.com.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy the securities being offered, nor shall there be any sale of the securities being offered in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

About Aura Biosciences
Aura Biosciences, Inc. is a clinical-stage biotechnology company developing virus-like drug conjugates (VDCs), a novel class of therapies, for the treatment of multiple oncology indications. Aura’s lead VDC candidate, belzupacap sarotalocan (bel-sar; AU-011), consists of a virus-like particle conjugated with an anti-cancer agent. Bel-sar is designed to selectively target and destroy cancer cells and activate the immune system with the potential to create long-lasting anti-tumor immunity. Bel-sar is currently in development for ocular cancers, and Aura has initiated a global Phase 3 trial evaluating first-line treatment of choroidal melanoma, a vision- and life-threatening form of eye cancer where standard of care with radiotherapy leaves patients with severe comorbidities, including major vision loss. Aura plans to pursue development of bel-sar across its ocular oncology franchise including for the treatment of patients with choroidal metastasis. In addition, leveraging Aura’s technology platform, Aura is developing bel-sar more broadly across multiple cancers, including in patients with non-muscle invasive bladder cancer (NMIBC). Aura is headquartered in Boston, MA.

Forward-Looking Statements
Various statements in this release concerning the timing and completion of the public offering on the anticipated terms or at all may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. All such forward-looking statements are based on management’s current expectations of future events and are subject to a number of substantial risks and uncertainties, many of which are outside Aura’s control, that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include fluctuations in Aura’s stock price, changes in market conditions and satisfaction of customary closing conditions related to the public offering, as well as those risks more fully discussed in the section entitled “Risk Factors” in the prospectus supplement and registration statement referenced above, Aura’s Annual Report on Form 10-K for the year ended December 31, 2021, filed on March 23, 2022 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. There can be no assurance that Aura will be able to complete the public offering on the anticipated terms. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Aura undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, unless required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20221130006201/en/

Contacts
Investors and Media:

Alex Dasalla
Head of Investor Relations and Corporate Communications
adasalla@aurabiosciences.com

Argot Partners
Matthew DeYoung
aura@argotpartners.com

Nov 10
Love0

Aura Biosciences Reports Third Quarter 2022 Financial Results and Provides Clinical Development and Operational Highlights

By Aura Biosciences, Press Release, Publicly Listed

Press Release.

Announced the Global Phase 3 Trial Design with Suprachoroidal Route of Administration of Belzupacap Sarotalocan in Early-Stage Choroidal Melanoma

First Patient Dosed in the Phase 1 Study Evaluating Belzupacap Sarotalocan for the Treatment of Non-Muscle Invasive Bladder Cancer

 

BOSTON, MA – November 10, 2022 – Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today reported financial results for the third quarter ended September 30, 2022 and provided clinical development and operational highlights.

“We are encouraged by the meaningful clinical advances that we have made in both our ocular and urologic oncology programs in the third quarter,”

said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura.

“Aligning with regulatory agencies on the global Phase 3 trial design with suprachoroidal administration following positive Phase 2 data are key milestones supporting our goal of having the first approved vision preserving therapy for patients with early-stage choroidal melanoma. In addition, successfully dosing a first patient in non-muscle invasive bladder cancer is a meaningful achievement as we expand our platform into broad oncology indications.”

 

Recent Pipeline Developments

Belzupacap Sarotalocan (bel-sar) is being developed for the first-line treatment of early-stage choroidal melanoma (CM), a life-threatening rare disease with no approved therapies.

Aura finalized the global Phase 3 design in alignment with regulatory agencies and selected suprachoroidal (SC) route of administration to evaluate the efficacy and safety of bel-sar in early-stage CM. The Phase 3 trial is randomized and masked and will include three arms, where the primary analysis will compare bel-sar to sham. Aura is planning to enroll approximately 75 adult patients with early-stage CM, including patients with indeterminate lesions and small choroidal melanoma. Patients will be enrolled with documented growth as an enrichment strategy intended to increase the efficiency of the trial and which will include an adaptive design to further increase the probability of success.

Positive interim Phase 2 data evaluating SC administration of bel-sar for the first-line treatment of patients with early-stage CM were presented at AAO 2022. The results, with an average of six-months follow up in patients that received three cycles of therapy in Cohorts 5 and 6, showed a statistically significant reduction in the tumor growth rate (-0.296 mm/yr, p = 0.0007) compared to each patient’s documented growth rate at study entry, and an 88.9% (8/9) tumor control rate. In addition, the visual acuity preservation rate was 88.9% (8/9) in these cohorts, with the majority of patients being at high risk for vision loss with tumors close to fovea or optic disk. The overall safety profile of bel-sar was favorable, with no dose-limiting toxicities or treatment-related serious adverse advents reported as of August 19, 2022. There was no posterior inflammation and only mild anterior inflammation (Grade 1) in 20% of the patients. Treatment-related adverse events (AEs) were predominantly mild and resolved quickly without sequalae.

Aura dosed the first patient in a Phase 1 clinical trial of bel-sar for the treatment of non-muscle invasive bladder cancer (NMIBC) an area of high unmet need with approximately 80,000 patients diagnosed in the U.S. every year. Aura received Fast Track Designation from the U.S. Food and Drug Administration in Q2.

The Phase 1 multi-center, open-label clinical trial is expected to enroll approximately 23 adult patients. The trial is designed to assess the safety and tolerability of bel-sar as a single agent. The primary endpoint of the Phase 1 clinical trial is the incidence and severity of treatment- related AEs and serious adverse events and the incidence of dose-limiting toxicities. Aura expects to report initial Phase 1 data in 2023.

Beyond early-stage CM, the Company continues to build its ocular oncology franchise, with the goal of having choroidal metastasis, an unmet medical need with no approved therapies, as the second ocular indication. Aura plans to file an IND for choroidal metastasis with the FDA in Q4 of 2022.

Preclinical data supporting bel-sar’s broad tumor targeting potential and immune mediated mechanism of action was presented at the 22nd EURETINA Congress. Preclinical results highlighted bel-sar’s targeted cytotoxicity towards tumor cells derived from the most common cancer types known to metastasize to the choroid, supporting its potential use for the treatment of choroidal metastases, a key second ocular oncology indication. The presentation also included preclinical data that supported the activity of bel-sar as a single agent as well as in combination with checkpoint inhibitors, highlighting the possibility to treat not only primary tumors in the eye but also potentially distant metastases by an abscopal effect.

 

Recent Event

Aura hosted a virtual Investor Day on October 3, 2022. The program included preclinical data on bel- sar as a single agent and in combination with checkpoint inhibitors, two-year visual acuity data from the retrospective matched case control study of bel-sar vs. plaque radiotherapy, and interim data from the ongoing Phase 2 trial evaluating SC administration in early-stage choroidal melanoma. Aura’s executive management team was joined by ocular oncology leaders Dr. Carol Shields, Chief of the Ocular Oncology Service at Wills Eye Hospital and Professor of Ophthalmology at Thomas Jefferson University; Dr. Martine Jager, Professor of Ophthalmology, Leiden University, and Past President of the International Society of Ocular Oncology and the Association for Research in Vision and Ophthalmology; and Dr. Ivana Kim, Director of the Ocular Melanoma Center, Massachusetts Eye and Ear and Associate Professor of Ophthalmology, Harvard Medical School. The webcast is available here.

 

Third Quarter 2022 Financial Results

As of September 30, 2022, Aura had cash and cash equivalents and marketable securities totaling $111.5 million. Aura believes its current cash and cash equivalents and marketable securities are sufficient to fund its operations into 2024.

Research and development expenses increased to $11.3 million for the three months ended September 30, 2022 from $6.4 million for the three months ended September 30, 2021, primarily due to ongoing preclinical costs, manufacturing and development costs for bel-sar, and higher personnel expenses from growing headcount.

General and administrative expenses increased to $4.8 million for the three months ended September 30, 2022 from $2.5 million for the three months ended September 30, 2021. General and administrative expenses include $1.1 million and $0.4 million of stock-based compensation for the three months ended September 30, 2022 and 2021, respectively. The increase was primarily driven by personnel expenses, as well as increases in general corporate expenses related to operating as a public company.

Net loss for the three months ended September 30, 2022 was $15.9 million compared to $8.8 million for the three months ended September 30, 2021.

 

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage biotechnology company developing virus-like drug conjugates (VDCs), a novel class of therapies, for the treatment of multiple oncology indications. Aura’s lead VDC candidate, belzupacap sarotalocan (bel-sar; AU-011), consists of a virus-like particle conjugated with an anti-cancer agent. Bel-sar is designed to selectively target and destroy cancer cells and activate the immune system with the potential to create long-lasting anti-tumor immunity. Bel-sar is currently in development for ocular cancers, and Aura has initiated a global Phase 3 trial evaluating first-line treatment of choroidal melanoma, a vision- and life-threatening form of eye cancer where standard of care with radiotherapy leaves patients with severe comorbidities, including major vision loss. Aura plans to pursue development of bel-sar across its ocular oncology franchise including for the treatment of patients with choroidal metastasis. In addition, leveraging Aura’s technology platform, Aura is developing bel-sar more

broadly across multiple cancers, including in patients with non-muscle invasive bladder cancer (NMIBC). Aura is headquartered in Boston, MA.

For more information visit aurabiosciences.com, or follow us on Twitter and LinkedIn.

 

Forward Looking Statement

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward looking statements. Words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. These forward looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including choroidal melanoma, non-muscle invasive bladder cancer and choroidal metastases; any express or implied statements regarding the Company’s expectations for the Phase 2 and Phase 3 clinical trials of bel-sar for early-stage choroidal melanoma; and Aura’s expectations regarding the estimated patient populations and related market opportunities for bel-sar.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, an improved quality of life of patients after treatment with bel-sar; a potential paradigm shift in the approach to the treatment of choroidal melanoma; the urgent need for a vision preserving targeted therapy; the potential of bel-sar compared to the existing standard of care for patients with choroidal melanoma; uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s clinical trials may not be predictive of future results in connection with future clinical trials; the risk that interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; risks, assumptions and uncertainties regarding the impact of the continuing COVID-19 pandemic on Aura’s business, operations, strategy, goals and anticipated timelines; Aura’s ongoing and planned pre-clinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties, and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

 

Investor and Media Contact:

Alex Dasalla – adasalla@aurabiosciences.com
Head of Investor Relations and Corporate Communications

Argot Partners

Matthew DeYoung
aura@argotpartners.com

Nov 10
Love0

Aura Announces Global Phase 3 Trial Design with Suprachoroidal Route of Administration Based on Positive Phase 2 Interim Data of Belzupacap Sarotalocan in Early-Stage Choroidal Melanoma

By Aura Biosciences, Press Release, Publicly Listed
Press Release.

 

BOSTON, MA – November 10, 2022 – Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced that it has initiated startup activities for the global Phase 3 trial. After presenting positive interim data at the American Academy of Ophthalmology from its ongoing Phase 2 trial, Aura has aligned with regulatory agencies and finalized the design of the global Phase 3 trial. The trial will evaluate the efficacy and safety of belzupacap sarotalocan (bel-sar) with suprachoroidal administration, for the first-line treatment of early-stage choroidal melanoma (CM).

“Finalizing the study design and selecting the optimal route of administration for the Phase 3 trial are key milestones in progressing towards a potential approval for bel-sar as a first-line vision preserving therapy in patients with early-stage choroidal melanoma,” said Dr. Cadmus Rich, Chief Medical Officer of Aura Biosciences. “We are pleased to have aligned with regulatory agencies on the overall Phase 3 trial design, including the primary and key secondary endpoints. We remain focused on improving the standard of care for patients with early-stage choroidal melanoma, a life-threatening disease that has no approved therapies.”

The Phase 3 trial has a three arm randomized and masked design, where the primary analysis will compare bel-sar to sham. Aura is planning to enroll approximately 75 adult patients with eary-stage CM, including patients with indeterminte lesions and small choroidal melanoma. Patients will be enrolled with documented growth as an enrichment strategy intended to increase the efficiency of the trial which will include an adaptive design to further increase the probability of success.

 

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage biotechnology company developing virus-like drug conjugates (VDCs), a novel class of therapies, for the treatment of multiple oncology indications. Aura’s lead VDC candidate, belzupacap sarotalocan (bel-sar; AU-011), consists of a virus-like particle conjugated with an anti-cancer agent. Bel-sar is designed to selectively target and destroy cancer cells and activate the immune system with the potential to create long-lasting anti-tumor immunity. Bel-sar is currently in development for ocular cancers, and Aura has initiated a global Phase 3 trial evaluating first-line treatment of choroidal melanoma, a vision- and life-threatening form of eye cancer where standard of care with radiotherapy leaves patients with severe comorbidities, including major vision loss. Aura plans to pursue development of bel-sar across its ocular oncology franchise including for the treatment of patients with choroidal metastasis. In addition, leveraging Aura’s technology platform, Aura is developing bel-sar more broadly across multiple cancers, including in patients with non-muscle invasive bladder cancer (NMIBC). Aura is headquartered in Boston, MA.

For more information, visit aurabiosciences.com, or follow us on Twitter and LinkedIn.

 

Forward Looking Statement

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward looking statements. Words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. These forward looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including choroidal melanoma; any express or implied statements regarding the Company’s expectations for the Phase 2 and Phase 3 clinical trials of bel-sar; and Aura’s expectations regarding the estimated patient populations and related market opportunities for bel-sar.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, an improved quality of life of patients after treatment with bel-sar; a potential paradigm shift in the approach to the treatment of choroidal melanoma; the urgent need for a vision preserving targeted therapy; the potential of bel-sar compared to the existing standard of care for patients with choroidal melanoma; uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s clinical trials may not be predictive of future results in connection with future clinical trials; the risk that interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; risks, assumptions and uncertainties regarding the impact of the continuing COVID-19 pandemic on Aura’s business, operations, strategy, goals and anticipated timelines; Aura’s ongoing and planned pre-clinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties, and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

Investor and Media Contact:
Alex Dasalla
Head of Investor Relations and Corporate Communications
adasalla@aurabiosciences.com

Argot Partners
Matthew DeYoung
aura@argotpartners.com