Amylyx Pharmaceuticals receives FDA approval to resume AMX0114 Phase 1 trial

Amylyx Pharmaceuticals has recently shared exciting news concerning the FDA’s decision to lift the clinical hold on their Phase 1 clinical trial for AMX0114, an investigational antisense oligonucleotide designed to target calpain-2 for individuals living with amyotrophic lateral sclerosis (ALS). This development allows the company to move forward with establishing trial sites across North America, signifying a crucial step in the pursuit of novel treatments for this devastating disease.

The Phase 1 clinical trial, known as LUMINA, is set to begin in Canada in early 2025. This study is designed as a multicenter, randomized, placebo-controlled, multiple ascending dose trial that aims to evaluate both the safety and biological activity of AMX0114. Notably, the trial will also assess specific ALS biomarkers, including changes in neurofilament light (NfL) levels. Approximately 48 ALS patients will be enrolled in the study, with participants randomized to receive either AMX0114 or a placebo via intrathecal administration once every four weeks for up to four doses.

Dr. Camille L. Bedrosian, Chief Medical Officer of Amylyx, expressed optimism about the potential of AMX0114 based on preclinical efficacy studies and the known role of calpain-2 in axonal degeneration. Calpain-2 is a crucial target in various neurological conditions and is a protease that cleaves several essential proteins, including neurofilament, tau, and TDP43. Given its involvement in axonal degeneration and its association with neurofilament biology in prior studies, calpain-2 is considered a promising therapeutic target for ALS and other related diseases.

Amylyx Pharmaceuticals is eagerly anticipating early data from the LUMINA trial in 2025. The company remains dedicated to addressing unmet medical needs in communities affected by severe and fatal illnesses, with ongoing development programs focused on neurodegenerative, neuroendocrine, and endocrine disorders. With a commitment to using scientific advancements to drive decision-making, Amylyx is determined to make meaningful contributions to the field of medicine.

It is worth noting that ALS, also known as motor neuron disease, is a progressive and fatal neurodegenerative disorder characterized by the death of motor neurons in the brain and spinal cord. The loss of these motor neurons leads to muscle function deterioration, impaired movement and speech, respiratory paralysis, and ultimately, death. The vast majority of ALS cases are sporadic, with no clear familial link.

In conclusion, Amylyx Pharmaceuticals’ progress with the AMX0114 Phase 1 clinical trial signifies a significant advancement in the pursuit of innovative treatments for ALS. With the clinical hold lifted, the company can now focus on enrolling participants at trial sites across North America, with the ultimate goal of providing new hope for individuals grappling with this challenging condition.