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May 02
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Eloxx Pharmaceuticals Announces FDA Clearance to Begin Single Ascending Dose Study of ZKN-013

By Eloxx Pharmaceuticals, Press Release, Private Companies
Press Release.

 

May 2, 2023
ZKN-013 is in development for potential treatment of recessive dystrophic and junctional epidermolysis bullosa (RDEB and JEB) and Familial
Adenomatous Polyposis (FAP)
ZKN-013 is Eloxx’s first drug candidate from its TURBO-ZM™ platform to gain clearance for clinical testing
WATERTOWN, Mass., May 02, 2023 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) (“Eloxx” or the “Company”), a leader in
ribosomal RNA-targeted genetic therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the
Company’s Investigational New Drug (IND) application to initiate a single ascending dose (SAD) clinical trial in healthy volunteers for ZKN-013 for the
potential treatment of recessive Dystrophic Epidermolysis Bullosa (RDEB) with nonsense mutations. RDEB is a rare skin disease characterized by
mutations in Collagen7 gene.
“FDA clearance to begin our planned single ascending dose trial is an important milestone towards providing a potential treatment option for patients
with RDEB and JEB,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “This update is also a momentous event for Eloxx, as
ZKN-013, our lead TURBO-ZM™ based molecule, is the first program developed from hit to lead and is now poised to enter clinical development.
Beyond ZKN-013, Eloxx has promising TURBO-ZM based discovery programs in cystic fibrosis (fully funded by the Cystic Fibrosis Foundation) and in
certain cMYC-overexpressing cancers.”
Further SAD and multiple ascending dose (MAD) testing is expected to be conducted following the completion of the planned dose cohorts in the SAD
study and discussion with the FDA. The MAD testing could potentially include RDEB patients given the strong benefit/risk in patients cited by the FDA.
ZKN-013 Preclinical Activity in RDEB, JEB and FAP
ZKN-013 has demonstrated robust functional preclinical activity in RDEB and JEB patient cells and in APCmin (multiple intestinal neoplasia) mice.
Eloxx also plans to develop ZKN-013 for the treatment of FAP patients with nonsense mutations characterized by proliferation of colon polyps and
progression to colon cancer. In January 2023, Eloxx published its preclinical results that showed treatment with ZKN-013 demonstrated a decrease in
intestinal polyps and adenomas, resulting in increased survival.
About Eloxx Pharmaceuticals
Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform
in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead
investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02
is in Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations. Eloxx also has preclinical programs
focused on select rare diseases, including inherited diseases, cancer caused by nonsense mutations, kidney diseases, including autosomal dominant
polycystic kidney disease, as well as rare ocular genetic disorders.
For more information, please visit www.eloxxpharma.com.
Forward-looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other
than statements of present and historical facts contained in this press release, including without limitation, statements regarding clinical testing of
ZKN-013 for the treatment of RDEB and JEB and the expected timing of trials for our other product candidates and the potential of our product
candidates to treat nonsense mutations are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,”
“would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,”
“seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words. Forward-
looking statements are based on management’s current plans, estimates, assumptions and projections based on information currently available to us.
Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ
materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to
progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical
studies are not always indicative of positive clinical results; the scope, rate and progress of our preclinical studies and clinical trials and other research
and development activities; the competition for patient enrollment from drug candidates in development; the impact of the global COVID-19 pandemic
on our clinical trials, operations, vendors, suppliers, and employees; our ability to obtain the capital necessary to fund our operations; the cost of filing,
prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financial in the future through product
licensing, public or private equity or debt financing or otherwise; our ability to meet the continued listing requirements of the Nasdaq Capital Market;
general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and
the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the
fiscal year ended December 31, 2022, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s
website at www.sec.gov and the “Financials & Filings” page of our website at https://investors.eloxxpharma.com/financials-filings.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to
update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances
or otherwise.
Contact
Investors
John Woolford
john.woolford@westwicke.com
443.213.0506
Media
Laureen Cassidy
laureen@outcomescg.com
SOURCE: Eloxx Pharmaceuticals, Inc.

Apr 21
Love0

Orphalan announces US commercial launch of Cuvrior™ for the treatment of Wilson disease

By Orphalan, Press Release, Private Companies
Press Release.

 

– Cuvrior™ now available in the US for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to penicillamine

– Oral administration offers dosing flexibility and portability

Paris, France, April 20, 2023 – Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialization company, today announces the commercial launch in the US of Cuvrior™, a new trientine tetrahydrochloride (TETA-4HCl). Cuvrior™ is now available for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to D-penicillamine.

Cuvrior™ was approved by the United States Food and Drug Administration (FDA) and was granted Orphan Drug Exclusivity (ODE) in April 2022 for this patient group. Physicians can now discuss the availability of Cuvrior™ as a treatment option with their patients and can access important information here about prescribing Cuvrior™.

Compared with the standard of care, Cuvrior™ facilitates precise dosing by providing a small, scored tablet in a blister pack. Additionally, its room temperature stability eliminates the need for refrigeration, making it a more practical solution for patients with busy lifestyles.

Wilson disease is a rare inherited disorder of copper transport primarily affecting the liver and brain. Since 2019, Orphalan has commercialized its trientine tetrahydrochloride product in Europe under the name of Cuprior® and approximatively 1,000 Wilson disease patients have received treatment, with a positive track record on safety and efficacy.

Cuvrior™ was approved by the FDA on the basis of data from the Company’s phase III CHELATE trial, the first prospective randomized trial comparing penicillamine with TETA-4HCl. During the trial, an innovative assay was developed to measure non-caeruloplasmin bound copper (NCC), the free and potentially toxic pool of copper in the blood. Using this NCC measurement in patients previously receiving maintenance penicillamine therapy, TETA-4HCl was determined to be non-inferior to penicillamine at the primary endpoint of the study (24 weeks), with the same observation at the end of the extension phase of the study (one year from randomization).

Dmitry Paramonov, President Orphalan US, commented:

“We are thrilled to introduce Cuvrior™ in the US, a safe and innovative treatment option for patients and caregivers managing Wilson disease. Our distribution partners have Cuvrior™ stocked and ready for immediate use. At Orphalan, our focus is on providing targeted solutions for rare diseases, as demonstrated by the successful launch of Cuprior® in Europe. We are eager to expand availability of Cuvrior™ to as many patients as possible, underscoring our dedication to making a meaningful impact on the lives of rare disease patients worldwide.”

 

                                                                         ENDS                        

About Orphalan

Orphalan is a pioneering, international orphan drug development and commercialization company. Founded in 2011, the company develops and delivers innovative therapies for people living with orphan diseases, and is initially focused on Wilson disease, a rare genetic disorder that can be life-threatening if untreated. Orphalan commercializes Cuprior®, its trientine tetrahydrochloride product for the treatment of Wilson disease in Europe, and has launched Cuvrior™ in the US. For more information visit www.orphalan.com and follow us on LinkedIn.

About Cuvrior™

Cuvrior™ is an innovative new oral formulation of trientine and the first new advance in Wilson disease in over 30 years. Cuvrior™’s FDA approval was supported by a global phase III trial, CHELATE, which met its primary efficacy endpoint by demonstrating that Cuvrior™ was non-inferior to penicillamine as measured by serum non-caeruloplasmin copper assay (NCC) [1,2].

About Wilson Disease

Wilson disease is a rare genetic disease that is characterized by gradual accumulation of dietary copper over time, possibly to life-threatening levels. Affected individuals are unable to effectively excrete copper naturally through the digestive tract. Common symptoms associated with Wilson disease include progressive liver disfunction, neurological disorders such as severe tremors, and mental health deterioration. About 1 in 15 patients eventually need a liver transplant. Wilson disease affects nearly 1 in every 30,000 people worldwide.

For more information, please contact: 

Orphalan:
Géraldine van den Broek, Head of Corporate & BD
Tel: +33 (0)1 42 49 82 64
info@orphalan.com

Consilium Strategic Communications: 
Mary-Jane Elliott, Tracy Cheung, Davide Salvi
Tel: +44 (0) 203 709 5700
orphalan@consilium-comms.com

References

[1] Trientine tetrahydrochloride versus penicillamine for maintenance therapy in Wilson disease (CHELATE): a randomized, open-label, non-inferiority, phase III trial, Schilsky et al, Lancet Gastroenterol Hepatol 2022, Published Online September 29, 2022

[2] CuvriorTM USPI, 215760s000lbl.pdf (fda.gov)

Apr 10
Love0

FDA Authorizes Marketing of MISHA™ Knee System for People Suffering from Knee Osteoarthritis

By Moximed, Press Release, Private Companies
Press Release.

 

FREMONT, Calif.–(BUSINESS WIRE)–Moximed, a medical device company on a mission to improve the standard of care for people with knee osteoarthritis (OA), today announced that the U.S. Food and Drug Administration (FDA) granted marketing authorization of the MISHA™ Knee System, an implantable shock absorber (ISA) for the knee. The MISHA Knee System is indicated to treat people with medial knee OA who failed to find relief from non-surgical or surgical treatment, continue to experience pain that interferes with daily activities, and are ineligible for, or unwilling to undergo, joint replacement due to age or absence of advanced OA.

“I’ve been lucky enough to have been exposed to many major orthopedic innovations, most notably total knee arthroplasty (TKA). TKA is a phenomenal operation, but it has its limitations. I still see a clinical void between arthroscopic knee preservation and arthroplasty,” said Anil Ranawat, M.D., Chief, Hip and Knee Division of Sports Medicine Institute, Hospital for Special Surgery in New York. “With the FDA’s clearance of the MISHA Knee System, millions of people with symptomatic knee OA will now have an opportunity to achieve high levels of pain relief, enjoy the lifestyle and activities that are important to them, and preserve the option for a primary knee arthroplasty in the future. This system fills that void for both surgeons and patients.”

“Knee replacement is undoubtedly the gold standard for well-selected patients with severe OA, but for every patient I see that is ready for joint replacement, I see as many, if not more, who are not yet good arthroplasty candidates,” said William A. Jiranek, M.D., former President of the American Association of Hip and Knee Surgeons (AAHKS). “In my current role as Vice Chair for Practice Innovation at Duke University, I consider treatments for OA across the entire disease continuum, rather than focusing only on the end stage. As such, I was invited to provide an independent, non-investigator assessment of Moximed’s clinical study protocol, and I provided an independent, periodic review of the data during the study. I’m excited that the study met its superiority composite endpoint, which included effectiveness and safety components. This new joint-preserving treatment provides arthritis surgeons the ability to intervene earlier in the disease process to help patients not ready for a joint replacement.”

Moximed utilized the clinically established benefits of load reduction on diseased joints to design the MISHA Knee System. Implanted during an outpatient-compatible procedure, the MISHA Knee System demonstrated superiority over high tibial osteotomy (HTO), a well-established surgery with decades of demonstrated clinical results, in its recent pivotal clinical study.i

“This is a milestone event for knee OA sufferers, and it’s the result of unwavering clinical research and development that spans more than 10 years. We offer special thanks to our study patients and surgeon investigators who helped advance the understanding of this new treatment for OA. Also, we recognize the dedicated reviewers at FDA for completing their thorough benefit-risk assessment of our breakthrough technology. We’re thrilled to now be in a position to make the surgery available to patients,” said Anton Clifford, PhD, founder and CEO of Moximed. “We are committed to providing excellent medical education and customer service, supporting selection and treatment of indicated patients, and demonstrating scalability of our business as we introduce the MISHA Knee System to the U.S.”

Osteoarthritis (OA) is a common, debilitating condition, affecting the lives of over 32 million adults in the U.S, and projected to impact 70 million Americans by 2040. Knee OA develops when the joint’s natural shock absorbers, cartilage and meniscus, no longer cushion the joint from daily activities, leading to chronic pain and activity limitation. Many people with mild to moderate OA are otherwise healthy, in their prime working years, and have busy lives to live. For these patients, total knee replacement is a reluctant option, as it is an end-stage treatment for end-stage disease. OA patients without end-stage disease seek options that preserve their knee, activity level, and quality of life.

About Moximed

Moximed was founded in 2008 and is dedicated to helping people with mild to moderate osteoarthritis preserve their knee joints while living healthy, active lives. The company’s technology, the MISHA™ Knee System, is the result of over a decade of clinical research and development and is the first implantable shock absorber being developed for the treatment of medial compartment knee osteoarthritis (OA). With experienced medtech leadership and strong investor support, Moximed is poised to elevate the standard of care and quality of life for millions of pre-arthroplasty knee OA sufferers hindered by arthritic knee pain and function loss. Moximed is based in Fremont, California.

To learn more, visit www.moximed.com.

About The MISHA™ Knee System

Reducing weight on painful osteoarthritic joints is known to reduce pain and improve function. The MISHA™ Knee System being developed is the first implantable shock absorber that reduces weight on the knee joint with every walking step, easing pain, preserving function, and possibly delaying joint replacement surgery. The implant is placed on the medial knee and moves with the natural joint, reducing about 30% of the peak force on the knee with every walking step.ii

i Diduch DR, Crawford DC, Ranawat AS, Victor J, Flanigan DC. Implantable Shock Absorber Provides Superior Pain Relief and Functional Improvement Compared With High Tibial Osteotomy in Patients with Mild-to-Moderate Medial Knee Osteoarthritis: A 2-Year Report. CARTILAGE. 2023;0(0). doi:10.1177/19476035231157335.
ii Morgan OJ, Hillstrom HJ, Ranawat A, Fragomen AT, Rozbruch SR, Hillstrom R. Effects of a Medial Knee Unloading Implant on Tibiofemoral Joint Mechanics During Walking. J Orthop Res. 2019;37(10):2149-2156. doi:10.1002/jor.24379.

MEDIA CONTACTS

Annika Parrish

Health+Commerce

Mar 29
Love0

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

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

 

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

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

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

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

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

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

About Eloxx Pharmaceuticals

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

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

Forward-looking Statements

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

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

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

Contact

Investors
John Woolford john.woolford@westwicke.com 443.213.0506

Media
Laureen Cassidy laureen@outcomescg.com

SOURCE: Eloxx Pharmaceuticals, Inc.

Feb 16
Love0

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

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

 

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

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

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

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

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

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

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

About Aura Biosciences

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

Forward-Looking Statements

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

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

 

Contacts:

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

Argot Partners
Matthew DeYoung
aura@argotpartners.com

Source: Aura Biosciences, Inc.

Jan 13
Love0

Pheno Therapeutics Announces Worldwide License From UCB to a Novel Program for Neurodegenerative Disease

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

 

Preclinical program with potential for disease modifying therapeutic

Edinburgh, UK January 11 2023 – Pheno Therapeutics Limited., a biotechnology company focused on the discovery and development of small molecule therapeutics that promote remyelination for the treatment of neurological diseases such as multiple sclerosis, announced today it has entered into an exclusive worldwide license agreement with UCB. The agreement grants Pheno Therapeutics access and rights to develop, manufacture and commercialize a preclinical-stage program of novel small molecules designed to promote remyelination.

“Pheno Therapeutics utilized its proprietary human phenotypic screening platform to discover novel and tractable therapeutic targets to modulate oligodendrocyte biology and promote remyelination. This license allows us to accelerate a promising drug target towards clinical development,” said Fraser Murray, PhD., Chief Executive Officer of Pheno Therapeutics. “Together with our human neurological drug development expertise, there is significant opportunity for Pheno Therapeutics to fast-track this program towards clinical proof-of-concept studies and potentially deliver transformational drugs for the treatment of  demyelination diseases.”

“License agreements like this are a demonstration of the value UCB scientists and our partners are creating through strong research productivity, and we are confident that Pheno Therapeutics, with its expertise in phenotypic screening, stem cell technology and myelin biology, will develop this preclinical program  to its full potential,” said Dhaval Patel, Executive Vice President and UCB’s Chief Scientific Officer.

Pheno Therapeutics will make a one-time upfront payment and will be responsible for development, manufacturing and global commercialization. UCB will receive milestones and tiered royalties on net sales. Further financial details of the agreement were not disclosed.

About the Program:

The preclinical program is a novel small molecule program designed to selectively modulate the activity of an undisclosed target that is preferentially expressed on human oligodendrocytes and is known to play a critical role in the biological pathway and process of myelination.  The therapeutic program is being developed by Pheno Therapeutics under an exclusive worldwide license from UCB.

About Pheno Therapeutics:

Pheno Therapeutics is a discovery and development focused biotechnology company developing small molecule therapeutics that boost the function of human oligodendrocytes and their natural ability to promote remyelination for the treatment of neurological diseases with high unmet medical need such as multiple sclerosis (MS).  MS is a devastating chronic disease with significant individual and societal impact that often manifests in young adults and is associated with a wide range of neurological symptoms which can progress to total physical and cognitive disability.  Pheno Therapeutics is seeking to promote remyelination and reverse the critical demyelination aspect of MS. MS demyelination occurs when the immune system attacks and damages the myelin sheaths that insulate and nourish axons and nerve fibres in the central nervous system, leading to multifocal demyelination, axonal injury and neurodegeneration. For more information on Pheno Therapeutics, please visit https://www.phenotherapeutics.com.

 

Contacts:

Fraser Murray, PhD.
Chief Executive Officer
Pheno Therapeutics
Fraser.Murray@PhenoTherapeutics.com

Katie Odgaard
Account Director
Zyme Communications
Katie.odgaard@zymecommunications.com

+44 (0)7787 502 947

Dec 19
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F2G Announces FDA Filing Acceptance of New Drug Application for Olorofim for the Treatment of Invasive Fungal Infections

By F2G, Press Release, Private Companies
Press Release.

 

Application submitted under priority review with PDUFA target action date set for June 17, 2023

NDA submission is based on positive data from ongoing Phase 2b open-label study of oral olorofim in 100 patients with invasive fungal infections with limited or no treatment options

F2G is continuing preparations for a possible U.S. commercial launch of oral olorofim in 2nd half of 2023

PRINCETON, New Jersey, December 19, 2022 – F2G Inc. today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) for olorofim for the treatment of invasive fungal infections in patients who have limited or no treatment options.

F2G has requested approval of the NDA under the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD pathway) for a limited, well-defined population with invasive fungal infections and limited or no treatment options. Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of June 17, 2023

The NDA is supported by strong efficacy data and a good tolerability profile seen during treatment of the first 100 patients in the Phase 2b open-label study, all of whom had limited or no treatment options for either proven invasive fungal infection (including aspergilllosis, lomentosporiosis, scedosporiosis, Scopulariopsis infections, and refractory extrapulmonary coccidioidomycosis) or probable pulmonary invasive aspergillosis (Study 32, NCT03583164).

“Invasive fungal infections cause substantial morbidity and mortality, particularly among immunosuppressed patients, and can prove to be lethal in also healthy individuals when they get into deeper tissues. Effective therapies do not currently exist for some of these fungi. And even when therapies exist, some patients with invasive infections may be refractory or unable to tolerate existing antifungal treatments, thus underscoring the urgent need for new and effective treatments,” said John H. Rex, MD, chief medical officer of F2G. “Olorofim is a novel mechanism antifungal therapy from the newly discovered orotomide class. It provides a new option for patients who have exhausted treatment alternatives.”

Francesco Maria Lavino, chief executive officer of F2G, added,

“We are committed to addressing rare fungal infections, and the acceptance of filing of olorofim NDA for use in this well-defined and high-need population marks a major milestone toward our goal of bringing new options to these patients. We are building an experienced commercial team in preparation for U.S. launch, pending FDA approval. If approved, olorofim will be the first of a new class of antifungal drugs.”

Olorofim is the only antifungal medication to be awarded Breakthrough Therapy Designation by the FDA. Olorofim works through a novel mechanism of action, different from existing classes of antifungals, exerting fungal cell death through inhibition of the enzyme dihydroorotate dehydrogenase (DHODH) in the pyrimidine synthesis pathway. It is active in vitro against Aspergillus spp. (including azole-resistant and cryptic species), rare molds (e.g., Lomentospora prolificans, Scedosporium spp., Scopulariopsis spp.), and dimorphic fungi (e.g., Coccidioides spp.).

About Olorofim

Olorofim (formerly, F901318) is F2G’s leading candidate from the orotomide class and is currently being investigated in a Phase 2b open-label study 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 AmBisome® followed by standard of care (SOC) in patients with proven or probable invasive fungal infection due to Aspergillus species (NCT05101187). Olorofim has received orphan drug designation from the FDA for the treatment of coccidioidomycosis, scedosporiosis (including lomentosporiosis), invasive Scopulariopsis, and invasive aspergillosis. Olorofim has also received orphan drug designation from the European Medicines Agency (EMA) for the treatment of invasive aspergillosis, invasive scedosporiosis (including lomentosporiosis), and invasive Scopulariopsis. 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. Olorofim has received Breakthrough Therapy Designation for both “treatment of invasive mold infections in patients with limited or no treatment options, including aspergillosis refractory or intolerant to currently available therapy, and infections due to Lomentospora prolificans, Scedosporium, and Scopulariopsis species” and “treatment of Central Nervous System (CNS) coccidioidomycosis refractory or otherwise unable to be treated with standard of care therapy.” Olorofim is not approved by the FDA or any other regulatory agency.

About F2G

F2G is a biotech 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

Forward-Looking Statements

This announcement contains forward-looking statements. These statements are based on expectations in light of the information currently available, assumptions that are subject to risks and uncertainties which could cause actual results to differ materially from these statements. Risks and uncertainties include general domestic and international economic conditions such as general industry and market conditions, and changes of interest rate and currency exchange rate. These risks and uncertainties particularly apply with respect to product-related forward-looking statements. Product risks and uncertainties include, but are not limited to, completion and discontinuation of clinical trials; obtaining regulatory approvals; claims and concerns about product safety and efficacy; technological advances; adverse outcome of important litigation; domestic and foreign healthcare reforms and changes of laws and regulations. Also, for existing products, there are manufacturing and marketing risks, which include, but are not limited to, inability to build production capacity to meet demand, lack of availability of raw materials and entry of competitive products. The company disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events or otherwise.

Media Contact:

Gloria Gasaatura
LifeSci Communications
+1 646 970 4688
ggasaatura@lifescicomms.com

Dec 01
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Aura Biosciences Announces Pricing of Public Offering of Common Stock

By Aura Biosciences, Press Release, Publicly Listed

Press Release.

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

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

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

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

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

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

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

Contacts
Investors and Media:

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

Argot Partners
Matthew DeYoung
aura@argotpartners.com

Nov 15
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Artax Biopharma Expands Company’s Scientific Advisory Board

By Artax Biopharma, Press Release, Private Companies
Press Release.

 

Company Welcomes James G. Krueger, M.D., Ph.D., and Ajay Nirula, M.D., Ph.D., Both Experts in T-Cell-Mediated Diseases.

Cambridge, MA, November 15, 2022 – Artax Biopharma, Inc., a clinical stage biotechnology company focused on transforming the treatment of T-cell-mediated diseases, today announces the Company is strengthening its Scientific Advisory Board (SAB) with the appointments of James G. Krueger, M.D., Ph.D., and Ajay Nirula, M.D., Ph.D.

Artax Biopharma has an ongoing Phase 1 clinical trial evaluating AX-158, the Company’s first-in-class, oral small molecule immunomodulating agent that selectively acts at the T-cell receptor to lower self-antigen triggered cytokines, a known cause of autoimmune disease. Importantly, AX-158 has the potential to treat T-cell-mediated diseases without immunosuppression.

“We are honored to welcome global experts Drs. Krueger and Nirula to our Scientific Advisory Board, as they each bring unique research and extensive clinical experience across T-cell-mediated diseases,”

said D. Scott Batty, Jr., M.D., Artax Biopharma’s Chief Medical Officer.

“Drs. Krueger and Nirula bring a profound understanding of T-cell biology and pathophysiology and have success in bringing these effective treatments to patients who need them most. We are very grateful for their expert guidance and developmental insights as we progress AX-158 through clinical trials,”

Dr. Batty, Jr. added.

Dr. Krueger serves as Head of the Laboratory for Investigative Dermatology and as the D. Martin Carter Professor in Clinical Investigation and Dermatology at The Rockefeller University. He is co-director for the Center for Clinical and Translational Science at The Rockefeller University Hospital and has been serving as the Hospital’s Chief Executive Officer since July of 2008. Dr. Krueger uses psoriasis as a model to study inflammatory diseases that involve Th17 cells, a subset of T cells. His work has formed the scientific basis for the highly successful treatment of psoriasis with a range of biologic immune drugs that target the “Type 17” inflammatory immune axis.

Dr. Krueger earned his bachelor’s degree from Princeton University and his Ph.D. degree in Virology and Cell Biology from The Rockefeller University. He received his medical degree from Cornell University Medical College, where he also completed an internship in internal medicine and a residency in dermatology. Dr. Krueger is board certified in dermatology by the American Board of Dermatology and has been the recipient of numerous notable academic and industry honors throughout his career.

Dr. Nirula is Senior Vice President, Immunology, for Eli Lilly and Company based at the Lilly Biotechnology Center in San Diego. Dr. Nirula joined Eli Lilly in 2015 and is responsible for the Company’s discovery, research, and early phase clinical development in immunology. He has also served as the medical leader for Eli Lilly’s work during the COVID pandemic that led to emergency authorization for the Company’s multiple therapeutic neutralizing antibodies. Prior to joining Eli Lilly, Dr. Nirula held leadership positions at Amgen, and at Biogen Idec, and was involved in several research programs and regulatory filings spanning diseases such as rheumatoid arthritis, systemic lupus erythematosus, multiple sclerosis, psoriasis, and vasculitis.

Dr. Nirula earned his undergraduate degree in molecular biology from UC Berkeley, his medical degree from UCLA School of Medicine, and his Ph.D. degree from the University of Texas Southwestern Medical School. He subsequently joined the faculty in the Division of Rheumatology at UCSF Medical Center. Dr. Nirula has published extensively in premier peer-reviewed medical journals such as The New England Journal of Medicine, JAMA, and Nature Immunology.

 

Drs. Krueger and Nirula are welcomed by Artax’s current SAB members:

  • Balbino Alarcón, Ph.D., Artax Founder and Program Director at the National Research Council of Spain (CSIC) and Head of TCR-mediated Signal Transduction Laboratory;
  • Raif Geha, M.D., James L. Gamble Professor of Pediatrics at Harvard Medical School and Chief of the Allergy/Immunology/Rheumatology and Dermatology Division at Children’s Hospital in Boston;
  • Menno de Rie, M.D., Vice-Chair of the Department of Dermatology of the Amsterdam University Medical Centres /University of Amsterdam since 2012;
  • Lawrence Steinman, M.D., Professor of Neurology and Neurological Sciences, Pediatrics, and Genetics at Stanford University; and
  • Cox Terhorst, Ph.D., Professor of Immunology at Harvard Medical School and Chief, Immunology at the Beth Israel Deaconess Medical Center.

 

About Artax Science and Immunomodulation

Artax believes immunomodulation – selective targeting of specific functions and operations of the immune system – holds great potential to address tremendous unmet need in immunology. Our investigational immunomodulating agents act directly at the T-cell receptor (TCR) to selectively target inappropriately activated areas of the immune system, thus eliminating a cause of T-cell-driven diseases while not impacting a patient’s ability to effectively fight foreign pathogens and infections.

T-cells are the central, critical cells orchestrating the immune response, protecting the body against foreign pathogens, infections and malignancy while importantly not reacting to an individual’s tissues and organs. The TCR activates and controls the many functions and responses of the T-cell, serving a critical role for both healthy immune system and T-cell response.

Dysregulated TCR signaling results in increased cytokine signaling, a root cause of T-cell-mediated diseases, including autoimmune diseases. In addition to autoimmune diseases, T-cell-mediated diseases include T-cell malignancies, and induced T-cell pathologies where therapeutic treatments result in unwanted immune reaction side effects. Such induced T-cell immune reactions include stem cell transplants resulting in acute graft-versus-host-disease, and immuno-oncology treatments resulting in immune-related-adverse events. This central role of dysregulated TCR signaling across several disease states therefore makes the TCR a highly attractive target for therapeutic intervention.

 

About AX-158

AX-158 is a first-in-class, oral, small molecule immunomodulating agent in clinical development for the treatment of T-cell-mediated diseases. The immunomodulatory effect of AX-158 is designed to safely return the immune system to a state of rebalance without immunosuppression.

AX-158 is a highly specific (SH3.1 domain) inhibitor of Nck, a protein that naturally amplifies T-cell signaling caused by self-antigens at the TCR. AX-158 inhibits Nck at the TCR, resulting in less TCR activation and lowered self-antigen triggered cytokines, including Th-1, Th-17 and Th-2 type cytokines. High levels of such various cytokines are associated with a broad set of T-cell mediated autoimmune diseases, such as rheumatoid arthritis, psoriasis, atopic dermatitis, inflammatory bowel disease, and many others. Importantly, preclinical data suggests AX-158 is not immunosuppressive, and so does not impact the immune system’s ability to mount a strong response to foreign pathogens and infections.

 

About Artax Biopharma

Artax Biopharma is a clinical-stage biotechnology company transforming the treatment of T-cell-driven diseases through innovative small molecules that modulate the immune system. Artax’s disruptive science holds broad potential to treat T-cell-mediated diseases such as autoimmune diseases, induced T-cell pathologies (such as acute graft-versus-host disease, and immune-oncology treatment-related adverse events), and T-cell malignancies, while simultaneously allowing the body to fight foreign pathogens and infections. For more information, please visit www.artaxbiopharma.com and connect with us on LinkedIn.

 

Contacts:
Media:
Linda Phelan Dyson, MPH
Corporate Communications
lpdyson@verizon.net
M: 973-986-5973

Karen LaRochelle, MBA
Chief Business Officer
Artax Biopharma
klarochelle@artaxbiopharma.com

Nov 10
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Aura Biosciences Reports Third Quarter 2022 Financial Results and Provides Clinical Development and Operational Highlights

By Aura Biosciences, Press Release, Publicly Listed

Press Release.

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

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

 

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

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

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

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

 

Recent Pipeline Developments

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

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

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

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

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

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

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

 

Recent Event

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

 

Third Quarter 2022 Financial Results

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

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

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

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

 

About Aura Biosciences

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

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

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

 

Forward Looking Statement

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

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

 

Investor and Media Contact:

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

Argot Partners

Matthew DeYoung
aura@argotpartners.com