ALS Candidate AT-1501 Advances to Phase 2 Studies, Anelixis Therapeutics Announces

Anelixis Therapeutics has completed its Phase 1 clinical trial testing AT-1501, an investigational antibody, in healthy volunteers and eight amyotrophic lateral sclerosis (ALS) patients. The company also announced it has secured the funds to proceed to a Phase 2 clinical trial of AT-1501.

The results of the Phase 1 trial showed that AT-1501 was well-tolerated at all doses tested and its processing inside the body (pharmacokinetics) was as researchers predicted. Details of the study will be presented at future scientific meetings.

The company hopes that the data will support further clinical studies in ALS and other disorders to further evaluate AT-1501’s safety and tolerability, and other clinical parameters, such as potential biomarkers.

The funding for the Phase 2 clinical development of AT-1501 has been led by Biotechnology Value Fund, L.P., and other affiliates of BVF Partners L.P., a San Francisco-based investment firm that invests exclusively in biotechnology companies.

AT-1501 is an investigational antibody that targets the CD40 ligand (CD40L), a protein present at the surface of some white blood cells that is involved in inflammation. In ALS, the CD40L protein is produced in excess and is thought to be involved in neurodegeneration. This therapy has received orphan drug designation from the U.S. Food and Drug Administration (FDA).

AT-1501 was developed by the ALS Therapy Development Institute (ALS TDI), with support from several ALS organizations, including the ALS Association, ALS ONE, and ALS Finding a Cure. The ALS Association funded the Phase 1 trial by more than $1 million.

“With significant support from BVF, Anelixis is poised to launch an aggressive clinical development program for evaluating the safety and efficacy of AT-1501 for ALS, islet cell transplant, and other autoimmune diseases. BVF’s support and commitment, along with that of our founding investors, is a substantial endorsement of our approach,” Steven Perrin, PhD, founder and CEO of Anelixis, said in a press release.

“ALS, allograft [transplant] rejection and autoimmune diseases represent devastating unmet medical needs and we are eager to explore the potential value of AT-1501 as rapidly and diligently as possible. Advancement of AT-1501 into a robust clinical research program could offer tremendous potential opportunity for patients, including those in the ALS and diabetes communities,” he said.

“We are very excited by our investment in Anelixis. AT-1501 has the potential to provide meaningful benefits to patients across a range of autoimmune and neurodegenerative diseases. It is a privilege to help advance this compelling and differentiated program,” said Arman Gupta, principal at BVF, and now joining the Anelixis board of directors.