Amylyx Pharmaceuticals Inc. (NASDAQ: AMLX) 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).
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.
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 prospective identification of a novel lead series of Bax inhibitors that may be ready to enter Investigational New Drug (IND)-enabling studies.
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