Pridopidine Arm of HEALEY ALS Platform Trial Fully Enrolled

Marta Figueiredo, PhD avatar

by Marta Figueiredo, PhD |

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Prilenia Therapeutics announced the completion of patient enrollment in the pridopidine arm of the HEALEY ALS platform trial, the first study to simultaneously test multiple experimental therapies for amyotrophic lateral sclerosis (ALS).

Prilenia’s oral therapy pridopidine was selected as the trial’s fourth regimen (NCT04615923), with 163 ALS patients recruited ahead of schedule at the trial’s 52 U.S. sites since January 2021.

“We selected pridopidine to participate in the HEALEY Platform Trial out of approximately 30 investigational treatments due to its demonstrated potential to become a new therapy option for patients with ALS,” Merit Cudkowicz, MD, HEALEY’s principal investigator and sponsor, and director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, said in a press release.

“Completing enrollment for the fourth regimen in the Platform Trial brings us closer to discovering new treatments for people living with ALS,” Cudkowicz said, adding that this type of trial “allows faster recruitment of patients because of shared placebo data across treatment arms.”

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By testing several treatment candidates at the same time, platform trials are also expected to speed the identification and development of those showing the most promise, and at lower costs than single therapy trials.

“We are honored to participate in the trial led by the Healey Center and look forward to our continued collaboration as we work towards our goal and hope to have a positive impact on the ALS community,” said Michael Hayden, MD, PhD, Prilenia’s CEO and founder.

Patient enrollment is also complete for HEALEY’s first three regimens: Clene Nanomedicine’s CNM-Au8 (NCT04414345), Biohaven Pharmaceuticals’ verdiperstat (NCT04436510), and UCB’s zilucoplan (NCT04436497), trial organizers announced in a November release. Top-line study data are expected later this year.

A fifth treatment arm — Seelos Therapeutics’ SLS-005 (trehalose) — was cleared to launch in September (NCT05136885) and enrollment may have started. Contact information is available here.

HEALEY “is disrupting the pace of drug development by testing several investigational products in close collaboration with the patient community and with several clinical trial sites, industry and academic partners,” Sabrina Paganoni, MD, PhD, a physician-scientist at the Healey & AMG Center and co-lead investigator of the trial, said in a separate press release.

“The exceptional enrollment rates we are seeing are a testament to the power of the platform approach,” Paganoni said. “We will continue to add investigational treatments until we find cures for all people living with ALS.”

In each arm of the HEALEY platform trial (NCT04297683), about 160 ALS patients are randomly assigned to receive either an investigational therapy (120 patients) or a placebo (40 patients) for 24 weeks (about six months). To strengthen trial data, the placebo group will be shared between regimens.

With pridopidine, participants are given an oral capsule of either 45 mg of the therapy or a placebo twice daily.

HEALEY’s main goal is to assess the safety of each treatment and to evaluate their effectiveness at slowing ALS progression, as measured by the ALS Functional Rating Scale-Revised. Secondary goals include changes in muscle strength, lung function, and survival.

Pridopidine is an orally available small molecule designed to protect nerve cells against injury and death by activating the sigma-1 receptor (S1R). Once activated, S1R switches on pathways that support the growth, function, and survival of nerve cells.

In preclinical models of ALS, pridopidine was shown to prevent nerve cell death, boost connections between neurons, and preserve motor function.

Mutations in the gene that codes for S1R have been detected in ALS patients, and previous research suggested that S1R activation may improve swallowing, chewing, and speech in this patient population. Thus, secondary goals of pridopidine’s arm also include changes in these functions.

The therapy was designated an orphan drug in the U.S. and in Europe as a potential ALS treatment. This status is meant to accelerate pridopidine’s clinical development and review by providing financial benefits and regulatory support, as well as a period of marketing exclusivity (seven years in the U.S. and 10 in Europe) upon regulatory approval.

In November, Prilenia announced having raised $43 million to support potential regulatory submissions and marketing of pridopidine for ALS. Pridopidine is also being evaluated in late clinical trials as a potential therapy for Huntington’s disease.