Anelixis Therapeutics Secures New Funding to Advance Phase II Development of AT-1501 for Amyotrophic Lateral Sclerosis

VCBeat (WeChat ID: vcbeat) learned from Business Wire that on November 18, 2019, Anelixis Therapeutics announced the completion of a new round of financing. The round was led by BVF Partners (BVF), with no other financial terms disclosed. Additionally, as part of this financing, Arman Gupta, a partner at BVF, was appointed to the Board of Directors of Anelixis.

Anelixis, founded in 2013, is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, USA. As a subsidiary of the ALS Therapy Development Institute, the company focuses on the development of immunomodulatory drugs for neurodegenerative diseases, specifically for the treatment of amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease (AD).

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that is typically fatal, with patients usually surviving only 3–5 years after diagnosis. The disease commonly affects middle-aged and older adults in their 40s and 50s, imposing a significant burden on patients and their families. ALS is an orphan disease, with approximately 5,000 new cases diagnosed annually in the United States. Fewer than 10% of ALS cases are hereditary, commonly referred to as familial ALS (FALS). The remaining 90% of cases are sporadic (SALS), and their etiology remains unclear.

Anelixis possesses a portfolio of antibodies that block CD40L activity, with its lead compound, AT-1501, currently under development. This antibody lacks Fc effector function and inhibits co-stimulatory signaling between lymphocytes and antigen-presenting cells, thereby slowing the progression of ALS. AT-1501 holds potential for the treatment of amyotrophic lateral sclerosis (ALS), organ and cell transplantation, and autoimmune conditions. It is predicted to be effective across all types of ALS.

Anelixis also announced the completion of the Phase 1 safety and pharmacokinetic study of AT-1501. AT-1501 demonstrated good tolerability across all dose cohorts, with its pharmacokinetic profile consistent with predictions for IgG1 antibodies. Anelixis expects these results to facilitate further clinical studies in ALS and multiple other indications.

Currently, AT-1501 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) as a potential therapy to slow the progression of ALS.

Dr. Steven Perrin, Founder and Chief Executive Officer of Anelixis, stated, “With strong support from BVF, Anelixis is poised to launch an aggressive clinical development program to evaluate the safety and efficacy of AT-1501 in ALS, islet cell transplantation, and other autoimmune diseases. The support and commitment from BVF and our founding investors fully validate the merit of our approach. ALS, allograft rejection, and autoimmune diseases represent areas of significant unmet medical need, and we aim to rapidly explore the potential value of AT-1501 by advancing it into a robust clinical research program.”

Arman Gupta stated, “We are thrilled about our investment in Anelixis. AT-1501 has the potential to deliver meaningful benefits to patients suffering from various autoimmune and neurodegenerative diseases. We are honored to advance this compelling and uniquely valuable program.”

BVF Partners, founded in 1933, is a private investment firm headquartered in San Francisco, USA, specializing in investments in biotechnology companies. BVF primarily makes concentrated, long-term investments in small biotechnology firms, subjecting them to rigorous due diligence and ongoing monitoring.

(Compiled by Jiang Ying)