Armed with positive ALS data, Amylyx scores $30M in fresh funding to complete Alzheimer's PhII
Four years after announcing themselves to the biotech world with a new idea for drugging neurodegeneration, backing by the late Henri Termeer and $5 million from Morningside Venture, the young entrepreneurs at Amylyx are back for round 2.
Morningside continued to lead the $30 million Series B, with participation from Termeer’s widow, Belinda, and other unnamed investors. Having celebrated a topline Phase II win for its lead program in amyotrophic lateral sclerosis, Amylyx expects the cash to fund talks with regulators as well as a separate trial for the same drug in Alzheimer’s — for which they had just finished enrolling.
Aside from the CENTAUR trial, the theme at the biotech for the past year has been hiring. Including the co-founders — CEO Josh Cohen and president Justin Klee — and longtime CSO Kent Leslie, the team now consists of 15 full-time employees.
“Up until about a year ago, it was three people for about six years,” Jeff Trigilio, who joined as CFO in February, told Endpoints News.
Also new to the C-suite are biopharma vets like CMO Patrick Yeramian and chief commercial officer Margaret Olinger, who helped launch Soliris for Alexion. Tom Holmes was recruited from Biogen to head up the global supply chain.
Under Yeramian, the clinical team has generated data showing that AMX0035 slowed ALS disease progression by a statistically significant margin from placebo.
“With these results, Amylyx now has a responsibility to move ahead as efficiently as possible, as people living with ALS don’t have time to wait,” Klee said when the company released the results in December.
The drug has since been granted orphan status by the EMA. Stay tuned for the full data and next steps, Trigilio said.
As for the Alzheimer’s study, PEGASUS, 96 of the previously planned 100 patients have been dosed and topline results are expected in the first quarter of next year.
Here’s CSO Leslie for a refresher on how AMX-0035, which started out as a research project at Brown, targets neurodegeneration by going after neuronal apoptosis:
Many previous approaches have focused on targeting the pathognomonic features of Alzheimer’s Disease, amyloid and/or tau. We designed a neuro-protective investigational medication, AMX0035, to target key pathways in the mitochondria and endoplasmic reticulum which are thought to drive cellular death. Amylyx’s approach is irrespective of amyloid or tau, targeting neuronal damage pathways that may be downstream of these species but also which may arise due to other cellular or environmental factors.