First Human Trial for ALS Treatment Candidate AT-1501 Begins Dosing

Anelixis Therapeutics has begun dosing patients in the first human trial of its amyotrophic lateral sclerosis (ALS) treatment candidate, AT-1501, the company announced.

The trial is currently enrolling both healthy volunteers and eight ALS patients to test AT-1501’s safety and tolerability. Researchers will also examine how the agent behaves inside the body, including its metabolism and excretion.

“[The] dosing of the first person with ALS is a significant milestone in determining the potential utility of this approach in battling back this horrific disease,” Steve Perrin, PhD, president and CEO of Anelixis Therapeutics, said in a press release.

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.

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.

Last week, the ALS Association announced it had awarded Anelixis an additional $1 million to help fund the trial.

“We are proud to partner with Anelixis and other ALS organizations to help advance this promising compound into clinical trials,” said Calaneet Balas, president and CEO of the ALS Association. “Through this new funding, we are continuing our support of novel, early-stage potential treatments into human clinical trials.”

“We are excited to work closely with Anelixis to accelerate bringing AT-1501 to people with ALS,” said Merit Cudkowicz, director of the Healey Center for ALS at Massachusetts General Hospital, and chief medical officer for ALS Finding a Cure.

“The support from other ALS organizations for this trial has been crucial to its successful launch,” said Steve Perrin, who also serves as the president & CEO of the ALS Therapy Development Institute.

The results of the Phase 1 trial, if positive, will support a subsequent Phase 2a study to further evaluate AT-1501’s safety and tolerability, and other clinical parameters, such as potential biomarkers.

“The ALS ONE-supported trial design team members at the Healey Center are already working together with Anelixis to design the next trial to assess longer-term safety and efficacy. It is critical to move faster from exciting lab-based scientific discovery to clinical trials in our patients,” Cudkowicz said.