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Dec 06
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Beacon Therapeutics Announces Positive 3-Month Data from Phase 2 DAWN Trial of laru-zova (AGTC-501) in Patients with X-Linked Retinitis Pigmentosa (XLRP)

By Beacon Therapeutics, Press Release, Private Companies
Press Release.

 

Beacon Therapeutics Announces Positive 3-Month Data from Phase 2 DAWN Trial of laru-zova (AGTC-501) in Patients with X-Linked Retinitis Pigmentosa (XLRP)

  • To date, laru-zova has been well-tolerated by all participants in the Phase 2 DAWN study.
  • Data show promising early improvements in low luminance visual acuity (LLVA), a critical measure of visual function.
  • Data build confidence in laru-zova as a potential treatment for patients with XLRP.
  • Pivotal Phase 2/3 VISTA trial for laru-zova in XLRP is currently enrolling.

London, UK, Cambridge, MA, 6 December 2024 – Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), a leading ophthalmic gene therapy company with a purpose to save and restore the vision of patients with blinding retinal diseases, today announced the presentation of 3-month interim safety and efficacy results of the Phase 2 DAWN trial in patients with XLRP at the FLORetina-ICOOR Meeting 2024 in Florence, Italy.

Key presentation highlights:

  • The three-month data show that laru-zova has been well-tolerated by all participants.
  • No study agent-related treatment emergent adverse events (TEAEs) were reported, including no ocular inflammatory adverse events.
  • Data also show promising early improvements in LLVA, a critical measure of visual function.
  • The benefit-risk profile supports on-going clinical development for the treatment of patients with XLRP caused by RPGR mutations.

DAWN is a non-randomized, open-label study of laru-zova in participants with XLRP who have previously been treated with a full-length AAV-vector based gene therapy targeting the RPGR protein. The purpose of DAWN is to assess two different doses of laru-zova for efficacy, safety and tolerability in the untreated eye of participants who previously received gene therapy for XLRP.

XLRP is a severe, aggressive, inherited retinal disease that often leads to blindness by middle age, with no treatment options available. XLRP primarily affects young males with an estimated prevalence of 1 in 25,000 males in US, Europe and Australia having XLRP with RPGR mutations. Laru-zova expresses the full length RPGR protein and is therefore expected to address the entirety of photoreceptor damage caused by XLRP, including both rod and cone loss, representing a potential best-in-class treatment for progressive vision loss in patients with XLRP.

Lance Baldo, MD, chief executive officer of Beacon Therapeutics, stated, “We are encouraged by the early results from the Phase 2 DAWN study. The strong safety profile observed to date is complemented by promising early improvements in low luminance visual acuity – a meaningful and functional measure of vision in patients with XLRP. These data not only support the ongoing pivotal VISTA study, but also strengthen our commitment to this opportunity to bring hope to patients and families affected by this devastating disease.”

Beacon Therapeutics is also enrolling patients for its pivotal Phase 2/3 VISTA trial of laru-zova as it develops this potential treatment for patients with XLRP.

Presentations –
• Subretinal Gene Therapy laru-zova (laru-zova (AGTC-501)) for X-Linked Retinitis Pigmentosa (XLRP) Phase 2 Multicenter Study (DAWN): Preliminary Results

• RPGR gene therapy and the Beacon clinical trials: Beacon Therapeutics Subretinal Gene Therapy laru-zova (laru-zova (AGTC-501)) for X-Linked Retinitis Pigmentosa (XLRP)

Presenting Author – Professor Paulo Eduardo Stanga, Founder and Chief Medical Officer, The Retina Clinic London

Contact:
info@beacontx.com

Media:
beacontherapeutics@edelman.com

About Beacon Therapeutics
Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness.

The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP) and two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting an inherited cone-rod dystrophy (CRD). Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space.

Lead development candidate laru-zova (laru-zova (AGTC-501)), is a gene therapy program currently being investigated for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss, primarily in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Laru-zova expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss.

Beacon is supported by funds from Syncona Limited, Forbion, Oxford Science Enterprises, TCGX, Advent Life Sciences and additional investors.

Find out more about Beacon Therapeutics at beacontx.com.

Dec 05
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Arrakis Therapeutics Unveils Data for its Lead, Rationally Designed, RNA-Targeted Small Molecule (rSM) Drug Program Demonstrating Disease-Modifying Binding to RNA to Treat Myotonic Dystrophy

By Arrakis Therapeutics, Press Release, Private Companies
Press Release.

 

Arrakis Therapeutics Unveils Data for its Lead, Rationally Designed, RNA-Targeted Small Molecule (rSM) Drug Program Demonstrating Disease-Modifying Binding to RNA to Treat Myotonic Dystrophy

Preclinical data presented at Cell Symposia conference show a small molecule directed at a pathogenic RNA achieved reversal of myotonia in an animal model of myotonic dystrophy Type 1 (DM1)

Supports additional product development with Company’s multi-modality rSM drug platform, broadening the reach of genetic medicine and offering the potential to treat diseases at their root cause with oral medicines

Waltham, Mass., December 4, 2024 – Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced the first presentation of data for its RNA-targeted small molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1), a form of muscular dystrophy. These preclinical data demonstrate Arrakis’s leading-edge capability to rationally design a disease-modifying RNA-targeted small molecule, using its pioneering and comprehensive drug discovery platform. The data are being presented today at the Cell Symposia conference Chemical Biology in Drugging the Undrugged, taking place in San Francisco, CA on December 2-4, 2024.

The preclinical data from in vivo animal studies demonstrated the reversal of myotonia with Arrakis rSMs binding the target pathogenic CUG repeat RNA element responsible for DM1. Arrakis molecules selectively bind the CUG repeat, disrupt the formation of nuclear aggregates, release sequestered splicing factors, specifically Muscleblind-Like Splicing Regulator 1 (MBNL1), and correct splicing defects responsible for myotonia. Arrakis’s proprietary RNA‐specific chemical, biological, and structural methods and RNA-directed medicinal chemistry enabled structure-based small molecule drug design targeting the trinucleotide (CUG) repeat expansion in the mRNA of DMPK (myotonic dystrophy protein kinase) that drives DM1 pathology. The methodologies applied to the DM1 drug program are being used by Arrakis to advance its pipeline of proprietary and partnered rSM drug candidates for a range of other diseases.

“While there have been serendipitous discoveries of small molecule drugs that bind to RNA, we believe this is one of the clearest examples of a rationally designed small molecule that has been purpose-built to address an RNA disease target,” said Michael Gilman, PhD, Chief Executive Officer of Arrakis Therapeutics. “With the many tools we’ve built and lessons we’ve learned along the way, we believe that our DM1 program is the first of many potential RNA-targeted small molecule drug candidates that will emerge from our platform in coming years.”

Dr. Gilman added, “From the beginning, we have believed in the power of targeting RNA and committed to deeply understand RNA sequence, structure and function. rSMs combine the specificity, validation, and impact of genetic medicines with the well understood benefits of small molecule drugs, including systemic biodistribution, oral delivery, and more streamlined and cost-efficient supply chains. With our platform now built and scaled, we look forward to advancing our pipeline and vastly expanding the reach of RNA-targeted medicines by deploying our industry’s most established drug modality, oral small molecules.”

A presentation at Cell Symposia describes results from several preclinical studies. Highlights include:

  • High-resolution X-ray structures revealing the interaction of multiple Arrakis rSMs bound to the CUG RNA repeat.
  • Arrakis rSMs selectively bind the target RNA and displace MBNL1, a splicing factor sequestered in nuclear aggregates caused by CUG RNA repeats associated with DM1.
  • Arrakis rSMs neutralize repeat aggregates and correct splicing defects in DM1 patient-derived myocytes.
  • Arrakis rSMs modulate splicing and reverse myotonia in the HSALR mouse, the gold standard murine model of muscle dysfunction in DM1.

The presentation from Cell Symposia is available here on Arrakis’s website.

“We are pleased to share data on our DM1 program. For the first time, we are showing how our in silico, wet lab, and structural biology capabilities enable Arrakis to identify druggable RNA target sites and to rationally design and optimize small molecules that selectively bind to an RNA structure to modify its function,” said Jennifer Petter, PhD, Founder and Chief Scientific Officer at Arrakis Therapeutics and presenter of the data at Cell Symposia. “While there are promising muscle-directed oligonucleotide therapies in clinical development for the treatment of DM1, our rSMs have the potential to address the full systemic manifestations of the disease and represent a new class of genetic medicine that use small molecules to directly address the genetic basis of disease by targeting pathogenic RNA.”

 

About the rSM Program to Treat Myotonic Dystrophy Type 1 (DM1)

Myotonic dystrophy type 1 (DM1) is a form of muscular dystrophy and a genetic neuromuscular disease affecting at least 1 in 8,000 people worldwide or approximately 45,000 people in the United States. It is a multi-system disease, affecting the skeletal muscle, heart, diaphragm, central nervous system, and gastro-intestinal tract. DM1 is caused by a trinucleotide (CUG) repeat expansion of the RNA encoding DMPK (myotonic dystrophy protein kinase), resulting in the formation of nuclear aggregates that bind and sequester splicing factors important for cellular function. Arrakis’s rSM selectively binds to CUG repeats, disrupting aggregate formation in cells, liberating splicing factors, and correcting splicing defects in genes that drive DM1 pathology, including myotonia.

 

About Arrakis Therapeutics

Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. Arrakis is building a proprietary pipeline of RNA-targeted small molecule (rSM) medicines focused on genetically validated targets and numerous disease areas. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life sciences investors. The company is located in Waltham, Massachusetts. For more information, please visit www.arrakistx.com and engage with us on LinkedIn.

 

Media Contact:

Kathryn Morris
The Yates Network LLC
914-204-6412
kathryn@theyatesnetwork.com

Nov 13
Love0

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

Oct 25
Love0

Iterum Therapeutics Receives U.S. FDA Approval of ORLYNVAH™ (Oral Sulopenem) for the Treatment of Uncomplicated Urinary Tract Infections

By Iterum, Press Release, Publicly Listed
Press Release.

 

ORLYNVAH™ is the first oral penem approved for use in the U.S. and the second FDA-approved treatment for uUTIs in the past two decades

–Company to Host Conference Call on Monday, October 28th at 8:30 a.m. EDT–

DUBLIN and CHICAGO, Oct. 25, 2024 (GLOBE NEWSWIRE) — Iterum Therapeutics plc (Nasdaq: ITRM) (Iterum), today announced that the U.S. Food and Drug Administration (FDA) has approved Iterum’s new drug application for ORLYNVAH™ (sulopenem etzadroxil and probenecid) for the treatment of uncomplicated urinary tract infections (uUTIs) caused by the designated microorganisms Escherichia coli, Klebsiella pneumoniae, or Proteus mirabilis in adult women who have limited or no alternative oral antibacterial treatment options. This is the first approved indication for ORLYNVAH™ and the first FDA-approved product for Iterum.

“We are so pleased to have achieved this historic milestone and would like to thank all the patients, investigators, Iterum colleagues and Iterum consultants and vendors who participated in the development of ORLYNVAH™. ORLYNVAH™ offers new hope for patients suffering from difficult-to-treat uUTIs. The introduction of novel products, like ORLYNVAH™, is an important way to combat antimicrobial resistance to other approved oral agents and offers a potential solution to patients and physicians,” said Corey Fishman, Iterum’s Chief Executive Officer. “As the first oral penem approved in the U.S., ORLYNVAH™ offers an excellent alternative treatment option for appropriate patients in the underserved uUTI market. With FDA approval and a clear label, we will renew our efforts to achieve a strategic transaction involving ORLYNVAH™ with the goal of maximizing value for our stakeholders.”

The FDA approval of ORLYNVAH™ was based on a clinical development program supported by a robust data package, including two pivotal, Phase 3 clinical trials (known as SURE 1 and REASSURE) that evaluated the safety and efficacy of ORLYNVAH™ compared to ciprofloxacin (SURE 1) and Augmentin™ (REASSURE) in the treatment of adult women with uUTI. SURE 1 showed superiority to ciprofloxacin in fluoroquinolone resistant infections, while REASSURE showed non-inferiority and statistical superiority to Augmentin™ in the Augmentin™ susceptible population. ORLYNVAH™ was generally well tolerated in both SURE 1 and REASSURE clinical trials.

“The FDA approval of ORLYNVAH™ is tremendous news for those of us who have been hoping for a new option to treat appropriate at-risk patients suffering from UTIs,” said Marjorie Golden, MD, FIDSA, Site Chief, Infectious Disease, St. Raphael Campus Yale New Haven Hospital. “Based on the totality of clinical data generated, ORLYNVAH™ has the potential to be an important treatment alternative for use in the community.”

Conference Call

Iterum will host a conference call on Monday, October 28, 2024, at 8:30 a.m. Eastern Time. The dial-in information for the call is as follows:

United States: 1 833 470 1428 / International: 1 404 975 4839

Access code: 936149

The conference call replay will be available in the Events & Presentations section of Iterum’s website following the call.

About uUTIs

UTIs are among the most common bacterial infections encountered in the community. uUTIs are infections of the bladder occurring mainly in women. Up to 60% of women will have a uUTI in their lifetime. Up to 40% of women with a history of uUTI will have a recurrence of their infection. There are approximately 40 million uUTI prescriptions generated annually in the United States, and we estimate approximately 1% of those infections are caused by pathogens that are resistant to all commonly available classes of oral antibiotics. Rising antibiotic resistance, an aging population with comorbidities and sub-optimal safety profiles of existing oral treatment options are making antibiotic selection more challenging for treating physicians.

About ORLYNVAH™

ORLYNVAH™ is a novel oral penem antibiotic for the treatment of uUTI. ORLYNVAH™ possesses potent activity against species of Enterobacterales including those that encode extended spectrum beta-lactamase (ESBL) or AmpC-type beta-lactamases that confer resistance to third generation cephalosporins.

INDICATION AND IMPORTANT SAFETY INFORMATION

INDICATIONS & USAGE

ORLYNVAH™ a combination of sulopenem etzadroxil, a penem antibacterial, and probenecid, a renal tubular transport inhibitor, is indicated for the treatment of uUTI caused by the designated microorganisms Escherichia coli, Klebsiella pneumoniae, or Proteus mirabilis in adult women who have limited or no alternative oral antibacterial treatment options.

Limitations of Use

ORLYNVAH™ is not indicated for the treatment of:

  • Complicated urinary tract infections (cUTI) or as step-down treatment after intravenous antibacterial treatment of cUTI.
  • Complicated intra-abdominal infections (cIAI) or as step-down treatment after intravenous antibacterial treatment of cIAI.

Usage to Reduce Development of Drug-Resistant Bacteria

To reduce the development of drug-resistant bacteria and maintain the effectiveness of ORLYNVAH™ and other antibacterial drugs, ORLYNVAH™ should be used only to treat uUTI that are proven or strongly suspected to be caused by susceptible bacteria. Culture and susceptibility information should be utilized in selecting or modifying antibacterial therapy.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

  • Patients with a history of hypersensitivity to the components of ORLYNVAH™ (sulopenem etzadroxil and probenecid) or other beta-lactam antibacterial drugs.
  • Patients with known blood dyscrasias.
  • Patients with known uric acid kidney stones.
  • Concomitant use of ORLYNVAH™ and ketorolac tromethamine is contraindicated.

WARNINGS AND PRECAUTIONS

  • Hypersensitivity Reactions: Hypersensitivity reactions have been reported in patients treated with ORLYNVAH™. Serious and occasionally fatal hypersensitivity reactions, including anaphylaxis, have been reported with beta-lactam antibacterial drugs. Severe allergic reactions and anaphylaxis have been reported with the use of probenecid (a component of ORLYNVAH™). If an allergic reaction to ORLYNVAH™ occurs, discontinue the drug and institute appropriate therapy.
  • Clostridioides difficile-Associated Diarrhea (CDAD): This has been reported with nearly all systemic antibacterial agents. Evaluate if diarrhea occurs.
  • Exacerbation of Gout: When prescribing ORLYNVAH™ to patients with a known history of gout, ensure appropriate therapy of gout is instituted.

ADVERSE REACTIONS

The most common adverse reactions (≥2%) in patients treated with ORLYNVAH™ were diarrhea, nausea, vulvovaginal mycotic infection, headache, and vomiting.

DRUG INTERACTIONS

  • Ketoprofen: Concomitant use is not recommended.
  • See full prescribing information for additional clinically significant drug interactions with ORLYNVAH™.

USE IN SPECIFIC POPULATIONS

  • There are no available data on ORLYNVAH™ use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage or adverse maternal or fetal outcomes.
  • There are no data on the presence of ORLYNVAH™ or its metabolite in human milk, the effects on the breastfed infant, or the effects on milk production.
  • The safety and effectiveness of ORLYNVAH™ in pediatric patients have not been established.
  • No dosage adjustment based on age is required. ORLYNVAH™ is known to be substantially excreted by the kidney, and geriatric patients are anticipated to have reduced renal function. Recommendations for use in elderly patients should be based on renal function.
  • Increases in sulopenem plasma concentrations were observed with mild, moderate and severe renal impairment; however, the available safety information does not suggest a need for dosage adjustments in these patients. Administration of ORLYNVAH™ is not recommended in patients with creatinine clearance (CrCL) less than 15 mL/min and patients on hemodialysis because the pharmacokinetics of sulopenem have not been studied in this population.

To report SUSPECTED ADVERSE REACTIONS, contact Iterum Therapeutics plc at 1-866-414-SULO or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

About Iterum Therapeutics plc

Iterum Therapeutics plc is focused on delivering differentiated anti-infectives aimed at combatting the global crisis of multi-drug resistant pathogens to significantly improve the lives of people affected by serious and life-threatening diseases around the world. Iterum is advancing the development of its first compound, sulopenem, a novel penem anti-infective compound, with an oral formulation and IV formulation. Sulopenem has demonstrated potent in vitro activity against a wide variety of gram-negative, gram-positive and anaerobic bacteria resistant to other antibiotics. Iterum has received approval of its NDA for ORLYNVAH™ (oral sulopenem) for the treatment of uncomplicated urinary tract infections caused by the designated microorganisms Escherichia coli, Klebsiella pneumoniae, or Proteus mirabilis in adult women with limited or no alternative oral antibacterial treatment options by the U.S. Food and Drug Administration and has received Qualified Infectious Disease Product (QIDP) and Fast Track designations for its oral and IV formulations of sulopenem in seven indications. For more information, please visit www.iterumtx.com.

Cautionary Note Regarding Forward-looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These forward-looking statements include, without limitation, statements regarding Iterum’s plans, strategies and prospects for its business, including the development, therapeutic and market potential of ORLYNVAH™ and Iterum’s strategic process to sell, license, or otherwise dispose of its rights to ORLYNVAH™. In some cases, forward-looking statements can be identified by words such as “may,” “believes,” “intends,” “seeks,” “anticipates,” “plans,” “estimates,” “expects,” “should,” “assumes,” “continues,” “could,” “would,” “will,” “future,” “potential” or the negative of these or similar terms and phrases. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Iterum’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Forward-looking statements include all matters that are not historical facts. Actual future results may be materially different from what is expected due to factors largely outside Iterum’s control, including risks and uncertainties concerning the outcome, impact, effects and results of Iterum’s evaluation of strategic alternatives, including the terms, timing, structure, value, benefits and costs of any strategic alternatives, Iterum’s ability to complete a strategic alternative transaction, the market opportunity for and the potential market acceptance of ORLYNVAH™ for uUTIs caused by certain designated microorganisms in adult women who have limited or no alternative oral antibacterial treatment options, uncertainties inherent in the conduct of clinical and non-clinical development, changes in regulatory requirements or decisions of regulatory authorities, the timing or likelihood of regulatory filings and approvals, changes in public policy or legislation, commercialization plans and timelines, the actions of third-party clinical research organizations, suppliers and manufacturers, the accuracy of Iterum’s expectations regarding how far into the future Iterum’s cash on hand will fund Iterum’s ongoing operations, Iterum’s ability to maintain its listing on the Nasdaq Capital Market and other factors discussed under the caption “Risk Factors” in its Quarterly Report on Form 10-Q filed with the SEC on August 14, 2024, and other documents filed with the SEC from time to time. Forward-looking statements represent Iterum’s beliefs and assumptions only as of the date of this press release. Except as required by law, Iterum assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

Investor Contact:
Judy Matthews
Chief Financial Officer
312-778-6073
IR@iterumtx.com

Oct 24
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Epitopea Closes USD $31 million Pre-Series A Financing

By Epitopea, Press Release, Private Companies
Press Release.

 

Epitopea Closes USD $31 million Pre-Series A Financing

Proceeds support strategic development and clinical entry of lead Cryptigen immunotherapeutics

CAMBRIDGE, UK and MONTREAL, CANADA, 24 October 2024 – Epitopea, a transatlantic cancer immunotherapeutics company developing accessible off-the-shelf RNA-based immunotherapies, announces the closing of a USD $31 million pre-Series A financing.

The pre-Series A financing brings the total capital raised by Epitopea to over USD $45 million. New investors Investissement Québec, adMare BioInnovations and Jonathan Milner join existing investors Advent Life Sciences, CTI Life Sciences, Cambridge Innovation Capital, Fonds de Solidarité FTQ, the Harrington Discovery Institute, IRICoR and Novateur Ventures, all of whom also participated in this financing round.

Dr. Alan C. Rigby, Epitopea’s CEO, said, “We believe that more durable cancer therapies are needed to transform the lives of cancer patients diagnosed with hard-to-treat solid tumors where overall survival is still poor for most patients. We continue to make significant strides toward the clinic, which has strengthened investor enthusiasm, and resulted in an over-subscribed pre-Series A in support of our forward-looking mission and vision. This additional financing will support strategic pre-clinical discovery in solid tumors of interest while accelerating our near-term clinical development plans for Epitopea’s next generation, tumor selective, off-the-shelf, RNA-based immunotherapies that we believe have the potential to extend the durability of clinical response in patients.”

“In this round we would like to warmly welcome new investors Investissement Québec, adMare BioInnovations and Jonathan Milner, whose participation complements the continued passionate support of our existing transatlantic investor syndicate. This pre-Series A raise provides the necessary finances to accelerate our development campaigns and transition to a clinical-stage company with a focused commitment of delivering our transformational RNA cancer immunotherapy into the clinic in 2026,” he added.

Satish Jindal, Ph.D., General Partner with Advent Life Sciences added, “As a founding investor of Epitopea, we are pleased to continue our robust support of the company as it makes major strides in exploiting its proprietary CryptoMapTM platform to identify novel targets that can serve as the basis for the development of a range of ground-breaking cancer immunotherapies. Through our investment in this exciting and growing company, we look forward to Epitopea advancing a first off-the-shelf RNA cancer immunotherapy into the clinic, which could bring significant improvements to cancer patients’ quality and duration of life.”

“adMare is thrilled to invest in Epitopea and support the development of their highly differentiated RNA therapeutic platform. Epitopea’s innovative approach to cancer treatment aligns with our mission to drive scientific progress and bring transformative therapies to patients. We look forward supporting the growth of this promising company,” said Frédéric Lemaître Auger, Vice President Investments at adMare BioInnovations.

“Alongside other key players in the financial ecosystem, Investissement Québec is proud to participate in this round of funding designed to accelerate preclinical stages and research into cancer immunotherapies. Our support will not only promote the development of homegrown expertise in Québec, but also help drive the growth of a promising young Montréal business at the heart of the strategic life-sciences sector”, said Bicha Ngo, President and CEO, Investissement Québec.

  • ENDS –

Notes To Editors

Contact information

Epitopea

Dr. Alan C. Rigby – CEO

Alan.Rigby@epitopea.com

 

Scius Communications

Katja Stout

+44 7789 435990

katja@sciuscommunications.com

 

Daniel Gooch

+44 7747 875479

Daniel@sciuscommunications.com

 

About Epitopea

Epitopea is a transatlantic cancer immunotherapeutics company developing accessible off-the-shelf RNA-based immunotherapies for use in hard-to-treat cancers by targeting a new class of untapped tumor-specific antigens, which are known as CryptigenTM TSAs, that are broadly shared across multiple patients with the same tumor type.

The company has created an extensive library of novel CryptigenTM TSAs, discovered by its proprietary CryptoMapTM platform that leverages immunopeptidomics, genomics, and a bioinformatics pipeline, allowing the identification of aberrantly-expressed, tumour-specific antigens (aeTSA’s) that are hidden within cancer’s ’junk’ DNA.  These hidden CryptigenTM TSAs were first discovered through research led by Drs. Claude Perreault and Pierre Thibault at the Institute for Research in Immunology and Cancer at the Université de Montréal.

Epitopea is backed by leading life science investors including Advent Life Sciences, CTI Life Sciences, Cambridge Innovation Capital, Le Fonds de Solidarité FTQ, Investissement Québec, adMare BioInnovations, Jonathan Milner, the Harrington Discovery Institute, IRICoR and Novateur Ventures. To date the company has raised financing of more than USD $45 million. Epitopea was founded in 2021 and consists of sister companies based in Cambridge, UK and in Montreal, Canada. For additional information, please visit www.epitopea.com and follow us on LinkedIn.

About Investissement Québec

Investissement Québec’s mission is to play an active role in Quebec’s economic development by stimulating business innovation, entrepreneurship, and business acquisitions, as well as growth in investment and exports. Operating in all the province’s administrative regions, the Corporation supports the creation and growth of businesses of all sizes with investments and customized financial solutions. It also assists businesses by providing consulting services and other support measures, including technological assistance available from Investissement Québec Innovation. In addition, through Investissement Québec International, the Corporation prospects for talent and foreign investment, and assists Quebec businesses with export activities.

About adMare BioInnovations

With a strong track record of globally competitive scientific discovery, Canadian life sciences are primed to lead the world. To make this a reality, adMare BioInnovations uses its scientific and commercial expertise, specialized R&D infrastructure, and seed capital to build strong life sciences companies, develop robust ecosystems, and foster industry-ready talent. It re-invests its returns into the Canadian industry to ensure its long-term sustainability. adMare has helped build 36 companies, of which 25 are still active. These companies have attracted $2.4 billion of risk capital, have a combined value of $5.7 billion, and have created around 1,000 jobs in Canada. Our dynamic Montreal and Vancouver Innovation Centers have been home to 48 life science companies to date, with the 27 current resident companies employing over 350 life sciences professionals. The adMare Academy has trained more than 750 alumni — 92% of whom are employed in the life sciences industry. For more information, please visit www.admarebio.com.

Oct 18
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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

Oct 09
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U.S. Food and Drug Administration (FDA) accepts New Drug Application for elinzanetant

By Advent Life Sciences, KaNDy Therapeutics, Press Release
Press Release.

 

U.S. Food and Drug Administration (FDA) accepts New Drug Application for elinzanetant

  • The New Drug Application (NDA) for elinzanetant, an investigational compound for the treatment of moderate to severe vasomotor symptoms associated with menopause, is based on the positive data from three Phase III studies OASIS 1, 2 and 3
  • Elinzanetant is the first dual neurokinin-1 and 3 (NK-1 and 3) receptor antagonist, in late-stage clinical development for the non-hormonal treatment of moderate to severe VMS associated with menopause, administered orally once daily
BERLIN–(BUSINESS WIRE)–Bayer today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for the investigational compound elinzanetant seeking approval for the treatment of moderate-to-severe vasomotor symptoms (VMS, also known as hot flashes) associated with menopause.

“If approved, elinzanetant will offer a new non-hormonal option to women seeking treatment for moderate to severe vasomotor symptoms and we look forward to the review by the agency.”

Post this

“The NDA acceptance of elinzanetant by the FDA marks a significant milestone in our efforts to advance menopause care for women in the U.S.,” said Christine Roth, Executive Vice President, Global Product Strategy and Commercialization, Member of the Pharmaceutical Leadership Team at Bayer.“If approved, elinzanetant will offer a new non-hormonal option to women seeking treatment for moderate to severe vasomotor symptoms and we look forward to the review by the agency.”

The NDA application is based on the positive results from the OASIS 1, 2 and 3 Phase III studies evaluating the efficacy and safety of the investigational compound elinzanetant versus placebo. Findings from OASIS 1 and 2 were published in August 2024 in JAMA.1 Detailed results of the Phase III study OASIS 3 providing supporting efficacy and additional long-term safety data were presented at The Menopause Society (TMS) annual meeting in September 2024.

Bayer is continuing to submit applications for marketing authorizations of elinzanetant to further health authorities as well.

About the Elinzanetant clinical development program
The Phase III clinical development program of elinzanetant, OASIS, currently comprises four Phase III studies: OASIS 1, 2, 3 and 4. OASIS 1 and 2 investigated the efficacy and safety of elinzanetant administered orally once daily in women with moderate to severe VMS associated with menopause over 26 weeks and randomized 396 and 400 postmenopausal women between 40 and 65 years across 184 sites in 15 countries. Patients in the elinzanetant arm received a 120 mg dose of elinzanetant once daily for 26 weeks and patients in the control arm received a matching placebo once daily for 12 weeks, followed by elinzanetant 120 mg dose for 14 weeks. OASIS 3 investigated the efficacy and safety of elinzanetant for the treatment of vasomotor symptoms over 52 weeks in postmenopausal women and randomized 628 postmenopausal women between 40 and 65 years across 83 sites in 9 countries. The OASIS 4 study is an expansion of the clinical Phase III program and investigates the efficacy and safety of elinzanetant in women with moderate to severe VMS caused by endocrine therapy for treatment or prevention of breast cancer.

In addition to the OASIS program, Bayer is conducting NIRVANA (NCT06112756), an exploratory Phase II randomized, parallel-group treatment, double-blind study. The primary objective is to explore the efficacy of elinzanetant on sleep disturbances associated with menopause (SDM) as determined by polysomnography (PSG). PSG is a validated method to study sleep and underlying causes of sleep disturbances. Additional objectives include exploring the efficacy of elinzanetant on SDM as determined by patient-reported outcomes and further evaluating the safety of elinzanetant.

About Elinzanetant
Elinzanetant is the first dual neurokinin-1 and 3 (NK-1 and 3) receptor antagonist, in late-stage clinical development for the non-hormonal treatment of moderate to severe VMS associated with menopause, administered orally once daily. Elinzanetant may address moderate to severe VMS by modulating a group of estrogen sensitive neurons in the hypothalamus region of the brain (the KNDy neurons) which, with the decrease of estrogen, become hypertrophic and lead to a hyperactivation of the thermoregulatory pathway, consequently disrupting body heat control mechanisms resulting in VMS. Based on key secondary endpoints of OASIS 1 and 2, Elinzanetant may also decrease sleep disturbances associated with menopause.

About Vasomotor Symptoms
Vasomotor symptoms (VMS; also referred to as hot flashes) result from hyperactivation of the thermoregulatory pathway mediated by hypertrophy of the KNDy neurons. This is due to a decrease of estrogen, which can result from the progressive reduction of ovarian function due to natural menopause or medical intervention by bilateral oophorectomy or endocrine therapy.

VMS are reported by up to 80% of women at some point during the menopausal transition and are one of the leading causes for seeking medical attention during this phase of a woman’s life. Over one-third of menopausal women report severe symptoms, which can last 10 years or more after the last menstrual period, with relevant impact on quality of life.

VMS may also be caused by endocrine therapy, for the treatment or prevention of breast cancer, impacting quality of life and treatment adherence. For these women, there are currently no approved treatment options.

About Menopause
By 2030, the global population of women experiencing menopause is projected to increase to 1.2 billion, with 47 million women entering this phase each year. Menopause is a transitional phase in women’s lives, related to the progressive decline of ovarian function. It usually occurs in women during their 40s or early 50s. It can also be the result of surgical or medical treatment such as breast cancer treatment. The hormonal decline can lead to various symptoms which can substantially affect a woman’s health, quality of life, healthcare utilization and work productivity. The most frequently reported and disruptive symptoms during the menopausal transition are VMS, sleep disturbances and mood changes. Addressing these symptoms is key to maintaining functional ability and quality of life in menopause which is highly relevant from both a healthcare and socio-economic perspective.

About Women’s Healthcare at Bayer
Women’s Health is in Bayer’s DNA. As a global leader in women’s healthcare Bayer has a long-standing commitment to delivering science for a better life by advancing a portfolio of innovative treatments. Bayer offers a wide range of effective short- and long-acting birth control methods as well as therapies for menopause management and gynecological diseases. Bayer is also focusing on innovative options to address the unmet medical needs of women worldwide and to broadening treatment choices such as in menopause. Additionally, Bayer intends to provide 100 million women per year in low-and-middle income countries by 2030 with access to family planning by funding multi-stakeholder aid programs for capacity building and by ensuring the supply of affordable modern contraceptives. This is part of the comprehensive sustainability measures and commitments from 2020 onwards and in line with the Sustainable Development Goals of the United Nations.

About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to www.bayer.com.

Find more information at https://pharma.bayer.com/
Follow us on Facebook: http://www.facebook.com/bayer
Follow us on LinkedIn: Bayer | Pharmaceuticals

kw (2024-0157E)

Forward-Looking Statements
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conformthem to future events or developments.

_________________________
1 https://jamanetwork.com/journals/jama/fullarticle/2822766

Contacts

Contact for US media inquiries:
Courtney Ambrosi, phone 1 (908) 798-1107
Email: courtney.ambrosi@bayer.com

Contact for global media inquiries:
Katja Wiggers, phone +49 30 221541614
Email: katja.wiggers@bayer.com

Oct 08
Love0

Astellas and AviadoBio Announce Exclusive Option and License Agreement for Gene Therapy AVB-101 Targeting Frontotemporal Dementia and Other Indications

By AviadoBio, Press Release, Private Companies
Press Release.

 

Astellas and AviadoBio Announce Exclusive Option and License Agreement for Gene Therapy AVB-101 Targeting Frontotemporal Dementia and Other Indications

  • Astellas receives an exclusive option to license AVB-101, an investigational, AAV-based gene therapy in Phase 1/2 development
  • AviadoBio receives $20 million equity investment, up to $30 million in upfront payments and is eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties if Astellas exercises its option

LONDON, and TOKYO, – October 8, 2024 – AviadoBio Ltd. (“AviadoBio”) and Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, “Astellas”) today announced an exclusive option and license agreement for AVB-101, an investigational, AAV-based gene therapy in Phase 1/2 development for patients with frontotemporal dementia with progranulin mutations (FTD-GRN).

FTD is a devastating form of early-onset dementia that typically leads to death within three to 13 years from diagnosis. People with FTD commonly experience a rapid decline in executive function (attention control, working memory, problem-solving etc.), uncharacteristic behaviors, loss of language, apathy, and reduced mobility. It is an important cause of dementia in those under the age of 65 and is often underrecognized, and misdiagnosed. ¹,²,³,⁴

Under the terms of the agreement, Astellas will have the option to receive a worldwide exclusive license for the development and commercialization rights to AVB-101 in FTD-GRN and other potential indications. Astellas will make a $20 million equity investment and up to $30 million in upfront payments for the option to license AVB-101. AviadoBio is also eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties if Astellas exercises its option.

Lisa Deschamps, CEO, AviadoBio
“As we complete dosing of the first cohort of patients in our Phase 1/2 ASPIRE-FTD trial of AVB-101, we are excited about the potential of this collaboration to help address the unmet need that exists today in frontotemporal dementia. This strategic collaboration will combine our promising gene therapy candidate for FTD-GRN and delivery expertise with Astellas’ global capabilities in development and commercialization of gene therapies. Together, we can further accelerate delivering this investigational medicine to families around the world who so desperately need treatment options for FTD-GRN and other neurological diseases.”

Adam Pearson, Chief Strategy Officer, Astellas
“We look forward to collaborating with the team at AviadoBio as we expand our gene therapy pipeline to help a broader range of people living with debilitating, neurodegenerative diseases. AVB-101 represents a truly innovative approach to the treatment of FTD-GRN and has the potential to be part of the next generation of gene therapy products through the creation of this agreement. Genetic regulation remains a cornerstone of our primary focus strategy at Astellas and this agreement helps us to continue to provide potential solutions for patients in need.”

 

  1. Onyike CU and Diehl-Schmid J. Int Rev Psychiatry. 2013;25(2):130–137;
  2. Riedl L et al. Neuropsychiatr Dis Treat. 2014;10:297–310; 
  3. Onyike CU. Neuroepidemiology. 2011;37:166–167;
  4. Kansal K et al. Dement Geriatr Cogn Disord. 2016;41:109–122;

About AviadoBio

At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with neurological conditions. With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the company for success in bringing transformative medicines to patients.

AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), SV Health Investor’s Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Life Sciences (Dementia Fund), and LifeArc Ventures.

For more information, please visit www.aviadobio.com and follow us on X @AviadoBio and LinkedIn at AviadoBio.

About Astellas

Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into VALUE for patients. For more information, please visit our website at https://www.astellas.com/en.

Astellas Cautionary Notes

In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management’s current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas’ intellectual property rights by third parties. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice.

 

About Astellas Gene Therapies

Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. Our gene therapy drug discovery engine is built around innovative science, a validated AAV platform, and industry leading internal manufacturing capability with a particular focus on rare diseases of the eye, CNS and neuromuscular system. Astellas Gene Therapies will also be advancing additional Astellas gene therapy programs toward clinical investigation. Astellas Gene Therapies is based in South San Francisco, with manufacturing and laboratory facilities in South San Francisco, Calif., Sanford, N.C. and Tsukuba, Japan.

 

Company and Media Contacts

AviadoBio

Farah Speer
SVP, Head of Communications and External Relations
fspeer@aviadobio.com
+1-312-543-2881

Astellas Pharma Inc.

R&D Communications
Sara Zelkovic
+1-347-226-1459
Sara.zelkovic@astellas.com

Corporate Communications
+81-3-3244-3201 

Sep 12
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F2G Announces $100 Million Financing to Advance Late-Stage Development and Commercialization of Novel Antifungal Drug Candidate Olorofim in the US

By F2G, Press Release, Private Companies
Press Release.

 

F2G Announces $100 Million Financing to Advance Late-Stage Development and Commercialization of Novel Antifungal Drug Candidate Olorofim in the US

Financing is being led by new investor AMR Action Fund with strong participation from new investor ICG Life Sciences and existing investors

MANCHESTER, United Kingdom, September 12, 2024 – F2G Ltd, a clinical-stage biopharmaceutical company focused on the discovery and development of novel therapies to treat life-threatening rare fungal infections with a high unmet medical need, today announced a $100 million financing round. The financing is being led by new investor AMR Action Fund and co-led by ICG, with strong participation from existing investors Novo Holdings, Advent Life Sciences, Sofinnova Partners, Forbion, Blue Owl Healthcare Opportunities, Morningside Ventures, Brace Pharmaceuticals, and Merifin Capital. The proceeds from the financing will enable F2G to complete late-stage development, seek regulatory approval, and prepare for commercialization in the US of olorofim, a novel oral antifungal therapy to treat invasive aspergillosis (IA) and other invasive fungal infections. Henry Skinner, Ph.D., Chief Executive Officer of AMR Action Fund and Toby Sykes Ph.D., Managing Director of ICG Life Sciences, will join the F2G Board of Directors. The financing is subject to customary Foreign Direct Investment (FDI) clearance by local authorities in Austria, where F2G has a 100%-owned subsidiary, and expected to close in the fourth quarter of 2024.

Olorofim is the first orotomide antifungal, an entirely novel class of antifungal agents and is the only antifungal medication to be awarded a Breakthrough Therapy Designation for multiple indications by the US Food and Drug Administration (FDA). Olorofim works through a novel mechanism of action, different from existing classes of antifungals, exerting fungicidal activity through inhibition of the pyrimidine synthesis pathway. It is anticipated to be used to treat patients with a serious invasive, rare fungal disease where existing treatments are inappropriate or no longer effective.

Francesco Maria Lavino, Chief Executive Officer of F2G, said: “We are delighted to attract this additional capital investment into F2G, and we are thrilled to be working with the AMR Action Fund, an important player in the antimicrobial space, and ICG, a leading alternative asset manager. The addition of Dr. Skinner and Dr. Sykes to our Board will be invaluable as we move to our next stage of growth. This is a pivotal period for the Company as we conclude the final stages of development and preparation for commercialization of olorofim in the US and elsewhere. If approved, olorofim is expected to be the first of a new class of antifungal agents with a novel, differentiated mechanism of action and will address genuine unmet needs in conditions with high morbidity and mortality.”

Dr. Skinner commented: “Fungal infections are a growing threat to patients around the world and have a disproportionate impact on vulnerable populations, yet there has been a paucity of innovation in the field of antifungals. For decades, clinicians have relied on a handful of antifungal classes, with few mechanisms of action and significant limitations due to spectrum of activity, drug toxicities, or drug-drug interactions. These therapies are increasingly failing in patients. We are thrilled to support F2G’s team as they develop this urgently needed therapy and seek regulatory approval.”

Dr. Sykes commented: “Fungal pathogens continue to emerge and spread. This evolving threat underscores an urgent unmet medical need for advanced antifungal treatments. By investing in the development of novel antifungal drug candidates like olorofim, we hope to address this critical gap, ultimately making a meaningful and sustainable social impact on patients, their caregivers, and healthcare systems.”

About F2G
F2G is a clinical-stage biopharmaceutical company with operations in the UK, US, and Austria focused on the discovery and development of novel therapies to treat potentially life-threatening invasive fungal infections. F2G has discovered and developed a completely new class of antifungal agents called the orotomides which selectively target a key enzyme in the de novo pyrimidine biosynthesis pathway. This is a completely different mechanism from that of the currently marketed antifungal agents and gives the orotomides fungicidal activity against a broad range of rare and resistant fungal mold infections. For more information, please visit: www.f2g.com

About olorofim
Olorofim (formerly, F901318) is F2G’s leading candidate from the orotomide class and is currently in a Phase 2b open-label study (ClinicalTrials.gov Identifier: NCT03583164) in patients who have limited treatment options for difficult-to-treat invasive, rare fungal mold infections such as azole-resistant aspergillosis, scedosporiosis, lomentosporiosis, and other rare mold infections. F2G has initiated a global Phase 3 trial (“OASIS”) to compare treatment with olorofim versus liposomal amphotericin B followed by standard of care (SOC) in patients with invasive fungal disease caused by proven Invasive Aspergillus or probable lower respiratory tract disease by Aspergillus species. Olorofim has received orphan drug status from the European Medicines Agency for the treatment of invasive aspergillosis and invasive scedosporiosis. Olorofim has also received orphan drug status from the FDA for the treatment of coccidioidomycosis, scedosporiosis, and invasive aspergillosis. Olorofim has been granted Qualified Infectious Disease Product (QIDP) designation for invasive aspergillosis, invasive scedosporiosis, invasive lomentosporiosis, coccidioidomycosis, invasive disease due to Scopulariopsis species, and invasive fusariosis.

About invasive aspergillosis
Aspergillosis is a fungal infection caused by Aspergillus species of mold which are commonly found all over the world. Most of these molds, in most people, do not cause disease. However, Aspergillus is transmitted to humans through inhalation and may cause a broad spectrum of disease ranging from hypersensitivity reactions to direct invasion and destruction of tissue, especially in immunocompromised hosts. Invasive aspergillosis is a rare disease that can occur in over 10% of some high-risk immunosuppressed populations with mortality exceeding 80%.

Media Contact
Kristin Politi, Ph.D.
LifeSci Communications
kpoliti@lifescicomms.com
646-876-4783

Sep 12
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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