IPO details
Company intro
According to its regulatory filings, Amylyx Pharmaceuticals is focused on developing therapies that change the treatment paradigm for amyotrophic lateral sclerosis (ALS) and a broad range of neurodegenerative diseases by keeping neurons alive. Most cells in the body tend to die regularly and be replaced as part of healthy function but mature neurons are normally resistant to cell death and generally cannot regenerate.
The company’s product candidate, AMX0035, is a dual UPR-Bax apoptosis inhibitor composed of sodium phenylbutyrate (PB), and TURSO, also known as tauroursodeoxycholic acid, or TUDCA. Through the resolution of the unfolded protein response (UPR) and by inhibiting translocation of the Bcl-2 Associated X-protein (Bax) to the outer mitochondrial membrane, Amylyx has shown in multiple models that AMX0035 can keep neurons alive under a variety of different conditions and stresses.
Amylyx submitted a New Drug Submission (NDS) in Canada in the second quarter of 2021 for AMX0035 for the treatment of ALS, which was accepted for review in the third quarter of 2021. It also submitted a New Drug Application (NDA) to the US FDA in the fourth quarter of 2021, which was accepted for priority review in the same quarter. The company intends to submit a Marketing Authorization Application (MAA) in Europe early in the first quarter of 2022. Amylyx is pursuing ALS as its first indication as it is a disease of rapid and profound neurodegeneration.
Financials
For the nine months ended September 30, 2021, Amylyx reported grant revenue of $285,000. Net loss amounted to $59.5 million compared to $33.6 million in the same period a year ago. Pro forma net loss per share amounted to $1.30. Total operating expenses were $54.6 million compared to $30.4 million in the year-ago quarter.
Market opportunity
Neurodegeneration is one of the most significant unmet medical needs today. The development of therapies that preserve neuron health has been challenging, mainly due to a rudimentary understanding of the underlying biology as well as results seen in preclinical studies not translating to clinical trials. The currently approved therapies generally modify only symptoms and have shown limited efficacy. There remains an urgent need for novel approaches to address most neurodegenerative diseases, especially severe conditions such as ALS.