CAMBRIDGE, Mass.--(BUSINESS WIRE)--Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced the appointment of Camille L. Bedrosian, MD, as Chief Medical Officer (“CMO”). In this role, Dr. Bedrosian will lead global functions in medical affairs, regulatory, safety and pharmacovigilance, biometrics, clinical development, and clinical operations. Dr. Bedrosian brings nearly 30 years of experience addressing unmet medical needs for people with rare and serious diseases through successful clinical and translational research programs, including as CMO at Ultragenyx Pharmaceutical Inc. (“Ultragenyx”), Alexion Pharmaceuticals, Inc. (“Alexion”), and ARIAD Pharmaceuticals Inc. (“ARIAD”). Amylyx’ current CMO, Patrick Yeramian, MD, MBA, will stay on as an advisor through the end of the year and will then transition into retirement, as announced in December 2022.
“We are thrilled to have Dr. Bedrosian join us on our mission to end the suffering caused by neurodegenerative diseases and to help usher in a new era of innovation for the communities we are serving,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. “Dr. Bedrosian brings extensive experience having developed multiple successful medicines and facilitated global access to them, and her experience will be invaluable at Amylyx as we advance on our mission.”
Prior to joining Amylyx, Dr. Bedrosian served most of her nearly six years at Ultragenyx as the Executive Vice President and CMO where she was responsible for leading drug development programs and supporting translational research programs. During her time, she helped Ultragenyx grow and greatly advance its many pipeline programs in rare genetic diseases to their current and exciting pivotal stages. While CMO for Alexion, she led the development of drugs and drug candidates, including those designed to address devastating rare diseases caused by defects in the complement system for people with paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. Prior to Alexion, Dr. Bedrosian was CMO for ARIAD, where she led drug development and built the clinical organization. She serves as a Member of the MIT Corporation Visiting Committee for the Department of Biology. She also currently serves on the board of directors of Crinetics Pharmaceuticals, Inc. as well as Rhythm Pharmaceuticals, Inc.
“The team at Amylyx has made great strides in redefining how ALS is treated with the launch of RELYVRIO and ALBRIOZA, the first and only drug to show a benefit on both function and survival in people living with ALS in a single clinical trial. I’m excited to join Amylyx and contribute to bringing innovative, new treatment options to individuals and communities suffering from neurodegenerative diseases. I look forward to working with this talented team to further Amylyx’ leadership in ALS as well as in broader neurodegenerative diseases on behalf of people living with these conditions,” said Dr. Bedrosian.
“We also want to thank our longtime Chief Medical Officer, Dr. Patrick Yeramian, for his many years of dedication and commitment to building Amylyx into the successful company it is today. Dr. Yeramian was critical in the development of RELYVRIO and ALBRIOZA and the Health Canada conditional approval and FDA approval as well as building a world-class medical organization, and Amylyx would not be what it is today without his leadership,” said Mr. Cohen and Mr. Klee, Co-CEOs of Amylyx.
About RELYVRIO®/ALBRIOZA™/AMX0035
RELYVRIO® (also known as AMX0035), an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol (known as ursodoxicoltaurine outside of the U.S.), is approved to treat amyotrophic lateral sclerosis (ALS) in adults in the U.S. and approved with conditions as ALBRIOZA™ for the treatment of ALS in Canada. AMX0035 is being studied as an investigational drug in several other regions, as well as for the potential treatment of ALS and other neurodegenerative diseases. The formulation of RELYVRIO, ALBRIOZA and AMX0035 are identical.
RELYVRIO® (sodium phenylbutyrate and taurursodiol) Safety Information for United States
WARNINGS AND PRECAUTIONS
Risk in Patients with Enterohepatic Circulation Disorders, Pancreatic Disorders, or Intestinal Disorders
RELYVRIO contains taurursodiol, which is a bile acid. In patients with disorders that interfere with bile acid circulation, there may be an increased risk for worsening diarrhea, and patients should be monitored appropriately for this adverse reaction. Pancreatic insufficiency, intestinal malabsorption, or intestinal diseases that may alter the concentration of bile acids may also lead to decreased absorption of either of the components of RELYVRIO. Because different enterohepatic circulation, pancreatic, and intestinal disorders have varying degrees of severity, consider consulting with a specialist. Patients with disorders of enterohepatic circulation (e.g., biliary infection, active cholecystitis), severe pancreatic disorders (e.g., pancreatitis), and intestinal disorders that may alter concentrations of bile acids (e.g., ileal resection, regional ileitis) were excluded from the study; therefore, there is no clinical experience in these conditions.
Use in Patients Sensitive to High Sodium Intake
RELYVRIO has a high salt content. Each initial daily dosage of 1 packet contains 464 mg of sodium; each maintenance dosage of 2 packets daily contains 928 mg of sodium. In patients sensitive to salt intake (e.g., those with heart failure, hypertension, or renal impairment), consider the amount of daily sodium intake in each dose of RELYVRIO and monitor appropriately.
ADVERSE REACTIONS
The most common adverse reactions (at least 15% and at least 5% greater than placebo) with RELYVRIO were diarrhea, abdominal pain, nausea, and upper respiratory tract infection. Gastrointestinal-related adverse reactions occurred throughout the study but were more frequent during the first 3 weeks of treatment.
Please click here for RELYVRIO Full U.S. Prescribing Information.
About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is committed to supporting and creating more moments for the neurodegenerative disease community through the discovery and development of innovative new treatments. Amylyx is headquartered in Cambridge, Massachusetts and has operations in Canada and EMEA. For more information, visit amylyx.com and follow us on LinkedIn and Twitter. For investors, please visit investors.amylyx.com.
Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the potential approval of AMX0035 for the treatment of ALS in countries other than the United States and Canada; the potential of AMX0035 as a treatment for ALS globally and the Company’s plans to explore the use of AMX0035 for other neurodegenerative diseases; and expectations regarding the Company’s longer-term strategy. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: Amylyx’ ability to fund operations, the success, cost, and timing of Amylyx’ program development activities, Amylyx’ ability to execute on its commercial and regulatory strategy, the possibility that Amylyx may not obtain additional or maintain regulatory approvals for AMX0035, expectations regarding the timing of EMA review of AMX0035 for the treatment of ALS, the possibility that Amylyx may fail to achieve degree of market acceptance needed to become profitable, healthcare insurance coverage and reimbursement may be limited or unavailable for RELYVRIO in the U.S. and ALBRIOZA in Canada, competitive products may reduce or eliminate the commercial opportunity for AMX0035, Amylyx’ reliance on third parties, including to conduct clinical trials and manufacture products, and ongoing impacts of the COVID-19 pandemic, global macroeconomic uncertainty and geopolitical instability will have on Amylyx’ operations, as well as the risks and uncertainties set forth in Amylyx’ United States Securities and Exchange Commission (“SEC”) filings, including Amylyx’ Annual Report on Form 10-K for the fiscal year ended December 31, 2022, and subsequent filings with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, subject to any obligations under applicable law.
