Analysts start Amylyx on strong ALS data and possible regulatory approval

Amylyx Pharmaceuticals

Analysts for SVB Leerink and H.C. Wainwright launched coverage of Amylyx Pharmaceuticals (NASDAQ:AMLX) with “outperform” and “buy” ratings, and price targets of $50 and $35, respectively. The stock closed at $23.18 on Jan. 31.

Amylyx is a clinical-stage biopharmaceutical company developing a novel disease-modifying approach to target a key underlying cause of motor neuron cell death in amyotrophic lateral sclerosis (ALS). 

SVB Leerink analyst Marc Goodman writes that AMX0035 has demonstrated a “clinically meaningful effect on functional decline and survival in ALS, the first agent to successfully have a positive impact on both in one trial, the Phase 2 CENTAUR.”

In addition, he said AMX0035 is a relatively de-risked asset, given that the FDA has asked the company to file with the strong Phase 2 data even before completion of a confirmatory study, the ongoing Phase 3 PHOENIX clinical trial. 

“Significant unmet need in ALS with only two existing standard-of-care agents that don’t work very well should drive expedited regulatory approvals of AMX0035 and strong uptake, in combination with standard-of-care agents,” he added. 

Mr. Goodman also said that AMX0035 may show benefit in diseases other than ALS as it targets two cellular apoptotic pathways implicated broadly in neurodegenerative disorders.

Wainwright analyst Andrew Fein agreed, writing that AMX0035 is the first drug to show benefit in both function and survival, “thereby suggesting potential as a foundational therapy for ALS.”

Based on the robust data, Mr. Fein said the company is pursuing the commercialization of AMX0035. The company has filed an NDA with the FDA, which was accepted, and the drug was given priority review with a PDUFA date of June 29, 2022. It also filed an NDS in Canada and submitted a marketing authorization application with EMA. 

Based on prior dialogue with FDA, including the pre-NDA meeting, “we believe the company might not need the Phase 3 data for the final approval,” Mr. Fein added.