Allyx Therapeutics Gets $3.3M NIH Grant for ALX-001 Clinical Studies

Allyx Therapeutics, based in New Haven, Connecticut, has secured a $3.3 million grant from the National Institutes of Health (NIH) under its Small Business Innovation Research Commercial Readiness Pilot program. This financial support will enable further research into ALX-001, a groundbreaking oral therapy currently in clinical development for Alzheimer’s and Parkinson’s diseases.

The company plans to use the grant to conduct a Phase I clinical pharmacokinetic drug-drug interaction study, identified as NCT06632990. This study aims to assess how multiple doses of ALX-001 interact with various cytochrome P450 enzymes. Recently, initial doses of ALX-001 have been administered to patients with Alzheimer’s and Parkinson’s in two Phase Ib clinical trials (NCT05804383 and NCT06309147). The drug is positioned to advance to Phase 2 clinical development and is not dependent on the completion of ongoing or upcoming studies.

Tim Siegert, Co-Founder and Chief Operating Officer of Allyx Therapeutics, expressed gratitude for the NIH grant, emphasizing the importance of the metabolic study funded by this grant. Understanding the metabolic profile of ALX-001 is crucial for treating patient populations prone to complex polypharmacy and various comorbid conditions beyond neurodegenerative diseases.

Including the latest grant, the ALX-001 program has amassed over $23 million in funding. Previous financial support came from the NIH, the U.S. Government’s Small Business Innovation Research (SBIR) program, the Alzheimer’s Association, and The Michael J. Fox Foundation for Parkinson’s Research, among others.

ALX-001, formerly known as BMS-984923, stands out as a first-in-class compound due to its unique mechanism as a silent allosteric modulator of the mGluR5 receptor. This compound selectively inhibits the pathological activation of the receptor while preserving its normal physiological glutamate signaling, crucial for cognitive functions. This selective action provides ALX-001 with a broad therapeutic window, enabling receptor saturation without inducing the on-target toxicity associated with negative allosteric modulators. The mGluR5 receptor is vital for mediating synaptic dysfunction and loss caused by multiple misfolded extracellular protein species, presenting a novel treatment approach for Alzheimer’s and Parkinson’s diseases.

Importantly, ALX-001 is an orally bioavailable, brain-penetrant small molecule with demonstrated selective engagement of mGluR5. Initially identified by Bristol Myers Squibb, the compound’s mechanism of action for neurodegenerative diseases and its identification as a disease-modifying treatment for Alzheimer’s was discovered by Stephen Strittmatter, a scientific founder at Allyx, at Yale School of Medicine. Allyx Therapeutics holds an exclusive worldwide license for ALX-001 from Bristol Myers Squibb and Yale School of Medicine.

Founded in 2019, Allyx Therapeutics was established by a group of experienced biopharma executives, venture capitalists, and scientific experts. The company is dedicated to developing novel treatments to preserve and protect synapses in individuals living with neurodegenerative diseases. ALX-001, their lead compound, exemplifies this mission with its innovative mechanism of action targeting mGluR5, showing promise as a first-in-class oral therapy for Alzheimer’s and Parkinson’s diseases.