Prilenia: a potential new treatment for Huntington’s Disease & ALS

Written Nov 3, 2021. Wrote the blog post for Amplitude Ventures' investment in Prilenia. You can find the original here.

This week Prilenia announced the close of a $43M US oversubscribed and upsized Series B Financing. Amplitude decided to support the company to advance their lead program, Pridopidine, through Phase 3 clinical trials in Huntington’s disease and a phase 2/3 study in ALS.

The company was founded in September 2018 by Dr. Michael Hayden, an experienced biotech entrepreneur who is the world’s most-cited author for Huntington’s Disease. Following Michael’s successful career as a leading genetics researcher, multiple biotech co-founder and a pharma executive, he established Prilenia with the hope to provide Huntington’s patients with a treatment that helps improve their ability to function. Following the successful spin-out of Prilenia out of Teva, Michael recruited an exceptional management team including Henk Schuring and Limor Ben Har. Henk is Prilenia’s chief regulatory and commercialization officer who previously led rare neurological disease development at Sanofi Genzyme while Limor, the chief operating officer, brings her decades of experience, including most recently being the Chief of Staff of Global R&D at Teva.

Huntington’s disease (HD) is a debilitating neurodegenerative condition with no available treatments that can maintain function. There are only two drugs approved by the FDA for treating chorea in HD.

It is an inherited disease caused by a mutation in the huntingtin gene that typically presents between the ages of 30–50.

This mutation typically causes a progressive decline in neurological function and patients often develop changes in mood, cognition, and personality before losing coordination and control of their body. As the disease advances, uncoordinated, involuntary body movements known as chorea become more apparent and the patient’s physical abilities gradually worsen over time until movement becomes difficult, and the person loses the ability to speak or maintain usual functions such as employment or activities of daily living.

HD remains a significant problem for patients and their family members as it affects approximately 100,000 people around the world with death usually occurring between 15–20 years after initial onset.

How is Prilenia solving this problem:

Prilenia is advancing Pridopidine through phase 3 trials in HD. Pridopidine is a highly selective and potent sigma-1 receptor agonist. This phase 3 trial (PROOF-HD) is a randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of pridopidine in individuals with early-stage Huntington’s Disease. The aim of the study is to evaluate pridopidine’s impact on total functional capacity (TFC) score as the primary endpoint.

TFC is a validated, clinically meaningful, and regulatory accepted endpoint that measures the patient’s ability to maintain function in different domains. Currently, pridopidine is the only phase 3 clinical-stage drug candidate assessing the progression of HD as measured by TFC.

The company recently announced it has completed patient enrollment of 480 participants ahead of schedule in its global PROOF-HD trial.

Pridopidine could be the first therapy to be approved specifically for Huntington’s disease patients that maintain or improve their function.

Why we’re excited:

Prilenia is a great fit with Amplitude’s vision of creating, building, and growing world-class companies that aim to make a meaningful difference in patients’ lives. With an exceptional management team led by one of the world leaders in HD, a safe and potentially effective molecule in Pridopidine, and a great group of investors. We are excited to see Prilenia progress as they advance through phase 3 trials, thereby providing the Huntington’s disease community with a treatment that may improve patients’ quality of life.

-Amplitude team

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