• Amylyx Pharmaceuticals

  Led · Equity · Nov 2015

  Amylyx Pharmaceuticals is a Cambridge, Mass. pharmaceutical company developing novel therapeutics for ALS, Alzheimer’s disease and other neurodegenerative diseases. Its lead product candidate, AMX0035, is a co‑formulation of sodium phenylbutyrate (PB) and taurursodiol (TURSO/ursodoxicoltaurine) designed to reduce neuronal death by targeting endoplasmic reticulum and mitochondrial‑dependent degeneration pathways. Amylyx has published data on AMX0035 in the New England Journal of Medicine and Muscle and Nerve. The company filed a New Drug Submission (NDS) for AMX0035 with Health Canada in June and intends to submit a Marketing Authorization Application (MAA) to the EMA’s CHMP by the end of 2021. Amylyx plans to initiate a Phase 3 clinical trial and will continue to update the global ALS community, including plans involving the US FDA. The company closed an oversubscribed Series C to support late‑stage clinical development, potential launch planning for AMX0035, and further R&D in other neurodegenerative diseases. Amylyx Pharmaceuticals, based in Cambridge, Mass., is developing AMX0035, a first-in-class investigational therapy designed to reduce neuronal death by targeting mitochondrial and endoplasmic reticulum–dependent degeneration pathways. The company completed a Phase II ALS trial (CENTAUR) and has completed enrollment in a second Phase II Alzheimer’s trial (PEGASUS). CENTAUR was a 24-week, randomized, double-blind, placebo-controlled study assessing safety, tolerability and disease progression using ALSFRS-R and other measures; full CENTAUR data are anticipated for publication in the near term. PEGASUS is a biomarker-focused 24-week study in late mild cognitive impairment or early dementia due to Alzheimer’s, with topline data expected in the first quarter of 2021. Financially, Amylyx has closed an oversubscribed $30 million Series B financing led by Morningside Ventures to support ongoing clinical development. Company leadership states the proceeds will help the firm continue to advance trials and press ahead as rapidly as possible for patients with ALS, Alzheimer’s and other neurological conditions. Amylyx Pharmaceuticals is developing AMX0035, a proprietary fixed‑dose combination of sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA) for treatment of ALS. AMX0035 is designed to target neuroinflammation and nerve‑cell death; preclinical models showed synergistic activity and the individual agents have prior clinical testing showing safety, tolerability and preliminary signs of efficacy. The company plans to begin a Phase II clinical trial to evaluate safety, tolerability, functional outcomes, biomarkers of cell function/neuronal damage and inflammation, and a new objective measure of muscle strength. The IND for the Phase II trial is on schedule for the fourth quarter of 2016 and the trial is expected to start shortly thereafter, with over 20 U.S. clinical sites already expressing interest. Financially, Amylyx completed a $5.0 million Series A and received a $2.96 million grant for the trial; the company reports $10 million in combined grant funding and private financing to advance AMX0035. Investor board members have joined the company to support clinical development as Amylyx advances its candidate in a disease area with significant unmet need. Amylyx Pharmaceuticals is developing AMX0035, a combination of two existing small-molecule drugs intended to work in tandem to address nerve cell death and inflammation in neurodegenerative diseases. AMX0035 is designed to block stress on mitochondria and the endoplasmic reticulum, processes the company says mediate nerve cell death and neuroinflammation. The company has completed only preclinical testing to date and reports efficacy in disease-specific models of ALS, Alzheimer's and mitochondrial disease. Amylyx plans to launch clinical trials for ALS next year and will use the new funding to support IND-enabling studies for its lead candidate. According to an SEC filing it raised $1.3 million in a new equity round and additionally received $600,000 in grants from the ALS Finding a Cure Foundation and the Cure Alzheimer's Fund, totaling $1.9 million. Founded in 2013 and based in Cambridge, this is Amylyx's first round of equity financing; its scientific advisory board is chaired by Dr. Rudolph Tanzi.

No current team members are available.