• Project to support identification of Bax and Bak inhibitor therapeutic candidates with the potential to treat ALS and other neurodegenerative diseases

Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced a two-year sponsored research agreement with Sunnybrook Research Institute (SRI) to expedite the identification of novel drug candidates that inhibit Bax and Bak with high potency, specificity and appropriate pharmacokinetic properties for the development as therapeutics for neurodegenerative diseases, specifically in amyotrophic lateral sclerosis (ALS).

“We have been closely following research into Bax and Bak inhibition considering the criticality of these proteins in pathways of cell death and axonal degeneration and the work that has been done on these pathways in ALS. We are excited about the work conducted at SRI which we believe could be important for ALS and other neurodegenerative diseases,” said Joshua Cohen, Co-CEO and Co-Founder of Amylyx. Justin Klee, Co-CEO and Co-Founder of Amylyx added, “ALS is a relentless and complex disease, so we must also be relentless in our pursuit of new therapies to fulfill our mission to end the suffering caused by ALS and neurodegenerative diseases.”

ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually death. The pathophysiologic mechanisms leading to motor neuron death in ALS are multifactorial and include programmed cell death pathways involving Bax and Bak. Preclinical studies have shown promise for targeted inhibition of Bax and Bak in ALS, with results showing the preservation of motor neurons, delayed symptom onset, prolonged survival, and no toxicity concerns in an ALS mouse model.

“We have been investigating the inhibition of Bax and Bak as a potential mechanism of action for neurodegenerative diseases, especially ALS, for a number of years, and we are optimistic about where this research can go,” said David Andrews, Ph.D., Director and Senior Scientist in Biological Sciences at SRI, Tier 1 Canada Research Chair, and Principal Investigator. “We are thrilled to work with Amylyx to have a laser-focus on identifying potentially novel therapeutic candidates for ALS and other neurodegenerative diseases.”

At the end of this two-year agreement, Amylyx will have the right to license and develop any drug candidates that are identified by the research being conducted by SRI. SRI will be responsible for identification of multiple rounds of new Bax and Bak inhibitor compounds. The new analogs will be subjected to a robust screening cascade of biochemical, cell-based assays, and in vitro and in vivo neurodegenerative disease models, leading to the potential identification of a novel lead series of Bax inhibitors that may be ready to enter Investigational New Drug (IND)-enabling studies.

About Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is committed to supporting and creating more moments for the neurodegenerative 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.

About Sunnybrook Research Institute

Sunnybrook Research Institute (SRI) is the research arm of Sunnybrook Health Sciences Centre, an internationally recognized academic health sciences centre fully affiliated with the University of Toronto. SRI is one of the fastest growing hospital-based research enterprises in Canada with well-established programs in basic and applied sciences which span across three scientific platforms and ten clinical programs, developing innovations in care for the more than 1.3 million patients the hospital cares for annually.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning the of 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 ability to identify and develop compounds that inhibit Bax and Bak proteins and the potential of such compounds as a treatment for ALS and other neurodegenerative diseases, as well as the potential success of the [collaboration]/[partnership] between Amylyx and SRI. Any forward-looking statements in this communication are based on Amylyx 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: the success, cost, and timing of Amylyx’ program development activities, Amylyx’ ability to execute on its strategy, regulatory developments, expectations regarding the timing of regulatory review of Amylyx’ product candidates, the success of pre-clinical and clinical programs, Amylyx’ ability to fund operations, and the impact that the ongoing COVID-19 pandemic will have on Amylyx’ operations, as well as those 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 year ended December 31, 2021, 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.

Becky Gohsler
Finn Partners
(646) 307-6307

Lindsey Allen
Amylyx Pharmaceuticals, Inc.
(857) 320-6244

Source: Amylyx Pharmaceuticals, Inc.