Amylyx to Continue Pioneering New Treatments for Neurodegenerative Diseases with $30M in Funding – BioSpace

Massachusetts-based Amylyx Pharmaceuticals, a company focused on developing new treatments for neurodegenerative diseases, announced today that it had concluded a $30 million Series B round of financing. The initiative was led by Morningside Ventures, along with other investors.

Amylyx is currently focused on advancing its first-in-class investigational therapy, AMX0035, which is designed to reduce neuronal death and dysfunction. AMX0035 works by targeting mitochondrial and endoplasmic reticulum-dependent neuronal degeneration pathways in the body.

The company is anticipating the publication of data from its CENTAUR trial in the near future. CENTAUR, which was a 24-week, randomized, double-blind, placebo-controlled Phase II clinical trial, examined the safety and tolerability of AMX0035 in patients between the ages of 18 and 80 with definite Amyotrophic Lateral Sclerosis (ALS). The investigational therapy was assessed for its impact on disease progression, as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), and compared to a placebo.

ALS, which is sometimes referred to as Lou Gehrigs disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. It eventually leads to the death of motor neurons and the loss of the ability of the brain to initiate and control muscle movement.

The results AMX0035 achieved for ALS patients in the CENTAUR trial paired with Amylyx strong partnerships in the ALS and Alzheimers communities give credence to continued success, said Isaac Cheng, M.D., of Morningside Ventures. By targeting mitochondrial and endoplasmic reticulum dependent neuronal degeneration pathways, Amylyx can have a very significant impact for patients suffering from neurodegenerative diseases, a critical threat to global public health.

Amylyx announced back in December 2019 that AMX0035 had demonstrated a significant treatment benefit for people with ALS in the CENTAUR study. Participants who received the product showed a statistically significant slowing of ALS disease progression, as measured on the ALSFRS-R, compared to those who received the placebo.

At the time, subjects were given the option to enroll in an open-label extension study to receive treatment with AMX0035, and nearly 90% of those who completed the CENTAUR trial elected to join.

We are proud to have supported AMX0035 and Amylyx from an early stage and are very excited about what AMX0035 may accomplish for people with ALS, said Dr. Neil Thakur, executive vice president for mission strategy at The ALS Association, at the time of the announcement. This company and study team have focused on the patient perspective during the design and conduct of this study and we are happy to work with and innovate together with them. We are excited to continue to collaborate on this therapy in the future.

Interim data from the ongoing extension study is expected to be presented later in 2020. In addition, Amylyx intends to provide an update on regulatory plans and further details on expanded access plans as the year goes on.

Back in April 2020, the company announced that the European Medicines Agency granted Orphan Drug Designation to AMX0035, specifically for the treatment of ALS. This status is typically given to products intended for the treatment, prevention or diagnosis of rare, life-threatening, or chronically debilitating conditions where it may provide a significant benefit over existing treatments.

ALS patients worldwide are in need of new therapies, said Joshua Cohen, Co-CEO, Chairman, and Co-Founder of Amylyx, at the time of the announcement. Were excited about todays news and look forward to working with the European Medicines Agency on the next steps for AMX0035.

Original post:
Amylyx to Continue Pioneering New Treatments for Neurodegenerative Diseases with $30M in Funding - BioSpace