[Updated, 4:54 p.m.] Amylyx Pharmaceuticals’ two-pronged approach to neurodegenerative diseases has shown promise in amyotrophic lateral sclerosis (ALS) testing and the company now has $30 million to complete a separate clinical trial in Alzheimer’s disease.
The Series B round of financing announced Wednesday was led by Morningside Ventures, a firm that had previously invested in the Cambridge, MA-based biotech.
Amylyx’s experimental therapy, AMX0035, is a proprietary combination of two compounds, sodium phenylbutyrate and tauroursodeoxycholic acid. The company says the latter of the two is not the same as a dietary supplement by the same name that is available for purchase online. The Amylyx drug is intended to block cell death pathways that start in two components of a cell, the mitochondria and endoplasmic reticulum. [Paragraph updated to clarify description of the AMX0035 formulation.]
In an email, Amylyx Chief Financial Officer Jeffrey Trigilio said AMX0035’s approach offers neuroprotective properties that target pathways downstream from the genetic, cellular, or environmental causes of neurodegeneration. That means the drug has potential applications across a wide range of central nervous system disorders.
Last December, Amylyx reported that its drug achieved the main goal of a mid-stage clinical trial in ALS, beating a placebo at slowing the disease’s progression measured according to a rating scale used to assess patients. Trigilio says the company expects to publish full data from the ALS study “in the near term.” In the meantime, a separate Phase 2 study in Alzheimer’s disease completed patient enrollment in early June. Trigilio expects preliminary data from that clinical trial will become available in the first quarter of next year.
The new capital will be used to finish the Alzheimer’s study, Triglio said. The company will also use the cash to scale up the manufacturing process for AMX0035. At the same time, the company plans to speak with the FDA and drug agencies in other countries regarding the regulatory pathway for the drug.
Other investors joining Morningside in the latest Amylyx financing were not disclosed. Earlier investors in the biotech startup include the late Henri Termeer, who was the former CEO of Genzyme, and the ALS Investment Fund.