RARE Daily

Prilenia Raises $43 Million to Advance Huntington’s Disease and ALS Programs to Regulatory Submission

November 3, 2021

Prilenia Therapeutics said it raised $43 million in an oversubscribed series B financing round that it will use to prepare for the potential registration and commercialization of its lead drug candidate, pridopidine, for patients with the neurodegenerative disorders Huntington’s disease and amyotrophic lateral sclerosis, as well as to accelerate timelines and expand the company’s executive team and operations.

Photo: Michael Hayden, CEO and founder of Prilenia

New investor Sands Capital led the funding round, alongside Forbion and Morningside. Amplitude Ventures also joined the round as a new investor, in addition to existing investors Sectoral Asset Management, Talisman, and the ALS Investment Fund. The series B financing brings the total capital invested in Prilenia Therapeutics since its founding in September 2018 to $133.5 million.

“With the support of this group of leading investors, we are well-capitalized and resourced to continue advancing our programs towards potential registration and commercialization,” said Michael Hayden, CEO and founder of Prilenia. “The recent completion of patient enrollment in our PROOF-HD phase 3 clinical trial ahead of schedule and above target enrollment numbers is a significant milestone. We look forward to advancing our programs in both HD and ALS.”

Pridopidine acts as a highly selective and potent sigma-1 receptor (S1R) agonist. Extensive safety data demonstrate pridopidine has a favorable safety and tolerability profile. Currently, pridopidine is the only phase 3 clinical stage drug candidate assessing Huntington’s disease (HD) progression as measured by TFC and is also being assessed for treatment of amyotrophic lateral sclerosis (ALS) in the HEALEY ALS phase 2/3 platform trial.

Huntington’s disease (HD) is a fatal, inherited, neurodegenerative disorder. Every offspring of an HD patient has a 50 percent chance of inheriting the gene. Usually starting at around 40 years of age, HD patients suffer from a movement disorder, progressive functional and cognitive decline, psychiatric disturbances, and behavioral symptoms. Following diagnosis, functional, motor, and cognitive functions decline, ultimately leading to immobility, dementia, and premature death.

Pridopidine has demonstrated maintenance of functional capacity in HD patients, as measured by Total Functional Capacity (TFC), in a post hoc analysis of a phase 2 clinical trial. This effect was most prominent in early-stage HD patients, who showed functional benefit from pridopidine 45 mg, taken twice a day.

Amyotrophic lateral sclerosis (ALS) is the most prevalent adult-onset progressive motor neuron disease, affecting approximately 20,000 people in the United States. ALS causes the degeneration of motor neurons, resulting in progressive muscle weakness and atrophy and eventually death. There are currently two FDA therapies approved specifically for treating ALS—riluzole and edaravone.

Compelling preclinical data supports the potential use of pridopidine as a therapeutic for ALS. Pridopidine exerts its neuroprotective effects via activation of the S1R. In in vivo ALS models pridopidine reduces toxic protein aggregates and ameliorates muscle fiber wasting. Previous clinical data also suggests that S1R is a promising target for ALS therapy, indicating that S1R activation may enhance bulbar and speech function in ALS patients. The sigma 1 receptor has been genetically validated for ALS, as patients with mutations in this gene develop ALS.

Author: Rare Daily Staff

Stay Connected

Sign up for updates straight to your inbox.