After raising questions surrounding dosing levels, the FDA has freed Amylyx’s early-stage amyotrophic lateral sclerosis (ALS) candidate from a clinical hold.

When Amylyx had asked the FDA to approve the launch of in-human studies for AMX0114—an investigational antisense oligonucleotide—the agency restricted dosing to an amount that was lower than the biopharma’s proposed starting dose of 12.5 mg, according to a November 2024 company release. The FDA then requested more information, resulting in the clinical hold, according to Amylyx, which prompted the biopharma to prioritize clinical efforts for the ALS candidate in Canada.

Now, the hold has been lifted on the phase 1 trial, dubbed LUMINA, and the Massachusetts-based company intends to open U.S. sites for patients, according to a Jan. 21 release.

LUMINA is still set to begin in Canada in the beginning of 2025, according to the company. The multiple ascending dose trial is expected to recruit 48 patients with ALS and administer up to four doses of placebo or AMX0114 per participant.

Amylyx expects early cohort data to be available sometime this year, according to the release.

AMX0114 is designed to target calpain-2, a calcium-dependent protease known to play a role in several neurodegenerative diseases.

“Based on several preclinical efficacy studies and what is known about the central role of calpain-2 in the process of axonal degeneration, we believe AMX0114 has the potential to be a treatment for ALS and other diseases,” Amylyx Chief Medical Officer Camille Bedrosian, M.D., said in the release.

The investigational antisense oligonucleotide was well tolerated in in vivo preclinical safety studies, according to Amylyx.

The trial marks a continuation of Amylyx’s efforts in the disease following last year’s market withdrawal of Relyvrio (sodium phenylbutyrate and taurursodiol). The drug failed to beat placebo at improving patients’ physical function in a phase 3 study, resulting in its eventual withdrawal.

The phase 3 miss also prompted Amylyx to send 70% of its workforce packing and later purchase a GLP-1 receptor antagonist from Eiger Biopharma for $35.1 million. Now, the company is revving up for a commercial launch of the GLP-1, known as avexitide, tapping Dan Monahan earlier this month to oversee the move as chief commercial officer.

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