Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer's disease and other neurodegenerative diseases, today announced it has closed an oversubscribed $30 million Series B financing, led by Morningside Ventures, with participation from other investors.

"The results AMX0035 achieved for ALS patients in the CENTAUR trial paired with Amylyx' strong partnerships in the ALS and Alzheimer's 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."

"We share Morningside's confidence in AMX0035 and we remain steadfast in our commitment to patients with ALS, Alzheimer's and other neurodegenerative diseases," said Jeffrey Trigilio, Chief Financial Officer at Amylyx. "Amylyx has made remarkable progress, completing one phase II trial in ALS and completing enrollment in a second Phase II trial in Alzheimer's. This funding will help us continue to press ahead as rapidly as possible because every day counts for patients with ALS, Alzheimer's and other neurological conditions."

Amylyx anticipates publication of full data from the CENTAUR ALS trial in the near term. The PEGASUS trial in Alzheimer's disease is in Phase II with topline data expected in the first quarter of 2021.

About CENTAUR
CENTAUR was a 24-week, randomized, double-blind, placebo-controlled Phase II clinical trial that evaluated the safety and tolerability of AMX0035 and assessed the drug's impact on disease progression as measured by the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) when compared to placebo. The trial also assessed the effects of AMX0035 on other measures that are critical to people with ALS, including muscle strength, lung vital capacity, and biomarkers of neuronal degeneration.

CENTAUR enrolled patients 18-80 years old with definite ALS and within 18 months of symptom onset. The trial did not restrict patients from receiving edaravone or riluzole. The CENTAUR study was conducted by investigators at 25 top ALS medical centers through the Northeast ALS (NEALS) consortium. NEALS is an international, independent, non-profit group of researchers who collaboratively conduct clinical research in Amyotrophic Lateral Sclerosis (ALS) and other motor neuron diseases. NEALS researchers are dedicated to finding treatments for people with ALS and motor neuron disease as rapidly as possible.

More information on the CENTAUR trial can be found at https://amylyx.com/trials/ or www.clinicaltrials.gov, NCT03127514.

About PEGASUS
PEGASUS (NCT03533257) is a 3:2 randomized, double-blind, multi-center, placebo-controlled study evaluating the safety, tolerability and activity of AMX0035 in patients with late mild cognitive impairment or early dementia due to Alzheimer's disease over 24 weeks. The biomarker-focused trial design will assist in understanding the effects of AMX0035 and its potential in treating Alzheimer's disease.

About AMX0035
AMX0035 is a first-in-class investigational therapy designed to reduce neuronal death and dysfunction. AMX0035 targets mitochondrial and endoplasmic reticulum-dependent neuronal degeneration pathways.

About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working on developing a novel therapeutic for amyotrophic lateral sclerosis (ALS), Alzheimer's disease and other neurodegenerative diseases. For more information, visit www.amylyx.com and follow us on LinkedIn and Twitter.

About Morningside Ventures

Founded in 1986 by the Chan family, Morningside is a technology and life science venture investment firm. With offices in Boston, Shanghai and London, the firm invests globally. Its portfolio emphasizes spin-outs from universities. In 2014, the founders of Morningside made a gift to Harvard University which renamed its public health school the Harvard T.H. Chan School of Public Health. To learn more, visit www.morningside.com.