Cambridge startup Amylyx Pharmaceuticals is developing a new combination drug product that could help treat neurodegenerative diseases like ALS, Alzheimer’s disease and mitochondrial disorders.
It just raised $1.3 million in a new equity round, according to an SEC filing. The startup also brought in $600,000 from the ALS Finding a Cure Foundation and the Cure Alzheimer’s Fund to support IND-enabling studies for its lead candidate, AMX0035.
AMX0035 is comprised of two existing small molecule drugs that are meant to work in tandem to address nerve cell death and inflammation. It plans to launch clinical trials for ALS next year.
“The ALS Finding a Cure Foundation is committed to pursuing multiple avenues toward a cure for ALS,” Merit Cudkowicz, the foundation’s chief medical officer as well as chief of neurology at Massachusetts General Hospital, said in a statement. “AMX0035 has shown promise in preclinical models. This grant will underwrite studies needed to begin clinical trials.”
The degradation and stress on mitochondria and the endoplasmic reticulum is now considered a key mediator of both nerve cell death and the neuroinflammatory process. These two organelles rely upon one another and are in constant communication – so if one of these cellular processes goes awry, the company says, the stress can either kill the cell or cause inflammation.
Amylyx posits that AMX0035, a combination of two disparate compounds, can block the stressors on both of these cellular organelles.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
The company’s only done preclinical testing thus far, but says it has shown efficacy in blocking nerve cell death and neuroinflammation in disease-specific models of ALS, Alzheimer’s and mitochondrial disease.
Notably, the startup’s scientific advisory board is chaired by Dr. Rudolph Tanzi, director of the genetics and aging research unit at Massachusetts General Hospital.
The startup was launched in 2013, but this is its first round of equity financing.
