Alector Announces First Participant Dosed in Phase 2 Study Evaluating AL001 in Amyotrophic Lateral Sclerosis (ALS)
Randomized, placebo-controlled Phase 2 trial will enroll patients with C9orf72-associated ALS
AL001 is a potential first-in-class monoclonal antibody designed to elevate progranulin, a key regulator of immune activity in the brain. Alector is also actively enrolling the Phase 3 INFRONT-3 pivotal clinical study of AL001 in individuals at-risk for and symptomatic patients with frontotemporal dementia (FTD) who carry a progranulin mutation (FTD-GRN), as well as currently enrolling the Phase 2 INFRONT-2 study in symptomatic patients with FTD who carry a C9orf72 mutation (FTD-C9orf72).
“AL001 has shown encouraging results in patients with FTD-GRN, rapidly increasing progranulin levels and normalizing inflammatory biomarkers along the disease cascade that contribute to neurodegeneration,” said
Both decreased progranulin levels and mutations in the chromosome 9 open reading frame 72 (C9orf72) gene are associated with abnormal accumulation of the TAR DNA-binding protein 43 (TDP-43). Excess aggregation of TDP-43 in brain cells is thought to lead to neuronal cell death and is associated with multiple neurodegenerative diseases, including ALS, where 95% of patients with ALS have TDP-43 pathology. In preclinical studies using multiple models of acute and chronic neurodegeneration, increasing progranulin levels has been shown in the literature to reverse and be protective against TDP-43 pathology.
“ALS, also known as Lou Gehrig’s disease, is a progressive and ultimately fatal neurodegenerative condition, with few FDA-approved treatment options. As we learn more about the genetic and biologic underpinnings of ALS, there is an opportunity to develop targeted treatments that address the mechanism of disease,” said
The Phase 2 trial of AL001 is a randomized, double-blind, placebo-controlled, multicenter study that is expected to enroll approximately 45 adult participants with C9orf72-associated ALS. Participants will receive AL001 or placebo intravenously every four weeks for 24 weeks added to their current treatment regimen. The primary endpoint of the study is safety, tolerability, pharmacokinetics and pharmacodynamics of AL001, including plasma and cerebrospinal fluid (CSF) progranulin levels. The Phase 2 will also gather data on changes to multiple liquid biomarkers. Taken together, these data are intended to inform trial design and dosing for future efficacy studies of AL001 in ALS.
About Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS) is a devastating, fatal, progressive neurodegenerative disorder. In ALS, the motor neurons in the brain and spinal cord die, resulting in weakness, muscle atrophy, paralysis and frequently, cognitive impairment, before resulting in death from respiratory failure. Each year, more than 5,000 people in the
Decreased levels of PGRN, a key regulator of immune response, lysosomal function, and neuronal survival in the brain, are genetically linked to many neurodegenerative disorders. AL001 is a novel human monoclonal antibody that elevates levels of progranulin by blocking the sortilin receptor responsible for progranulin degradation. AL001 is currently in a pivotal Phase 3 clinical study in people at risk for or with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). AL001 is also being evaluated in a Phase 2 study in symptomatic patients with FTD who carry a C9orf72 mutation, and a Phase 2 study in patients with amyotrophic lateral sclerosis (ALS) who carry a C9orf72 mutation. In
Alector is a clinical-stage biotechnology company pioneering immuno-neurology, a novel therapeutic approach for the treatment of neurodegenerative diseases.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from current expectations and beliefs, including but not limited to risks and uncertainties related to market conditions, Alector and its business as set forth in our Quarterly Report on Form 10-Q, as filed on
VP, Communications and Investor Relations
Source: Alector, Inc.