Prilenia Achieves 25% Enrollment in its Phase 3 HD Trial and Appoints Dr. Goldberg as CSO

May 13, 2021 12:00 UTC


  • Prilenia’s global phase 3 trial in Huntington’s Disease (PROOF-HD) has enrolled 120 patients on schedule reaching 25% of its target
  • PROOF-HD is the most advanced clinical trial in HD assessing impact on clinical progression
  • Dr. Y Paul Goldberg joins Prilenia to enhance the broader scientific strategy for pridopidine

NAARDEN, Netherlands--(BUSINESS WIRE)-- Prilenia Therapeutics B.V., a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, is pleased to announce that it has now enrolled more than 120 patients in its Phase 3 trial of pridopidine in HD (PROOF-HD), reaching 25% of the total target enrollment. The study remains on target to complete enrollment by Q4 2021 as originally planned.

The study is being conducted in collaboration with the Huntington Study Group at 60 sites across North America and Europe. To date, no safety signals of concern have emerged and none of the participants have dropped out from the study. This is consistent with the previously established favorable tolerability and safety profile of pridopidine.

The Company also announces the appointment of Dr. Y Paul Goldberg (MB ChB, PhD) as Chief Scientific Officer and Head of Early Clinical Development. Dr. Goldberg will lead the scientific strategy and build the early clinical development plan for pridopidine in new indications including evaluation of opportunities for expanding Prilenia’s pipeline.

Dr. Goldberg brings more than 20 years of industry experience, working across clinical development, drug discovery and medical genetics. Most recently, he served as VP of Clinical development at Ionis Pharmaceuticals where he led rare disease programs with a strong neurological focus. Prior to this, Dr. Goldberg served in various roles at Xenon Pharmaceuticals including Senior VP, Clinical Development. Dr. Goldberg obtained an MB ChB and PhD at the University of Cape Town. He obtained his fellowship in medical genetics (FRCPC) from the University of British Columbia and practiced as a medical geneticist for many years with a focus on adult neurogenetic disorders.

Dr. Michael R. Hayden, CEO and Founder of Prilenia, commented: “We are delighted that Dr. Y Paul Goldberg joins the Prilenia team. His proven track record in clinical development and his strong academic background will be of enormous value to our company.”

Dr. Y Paul Goldberg, Chief Scientific Officer and Head of Early Clinical Development said: “Joining Prilenia presents a fantastic opportunity to help advance a new and promising oral therapy offering relief for severely undertreated neurological diseases. I look forward to working with the team to accelerate the development of pridopidine, to potentially provide a much needed treatment option for patients and their families.”


Notes to Editors

About Prilenia

Prilenia is a clinical-stage biotech founded in 2018 with the purpose of improving lives of patients and families by developing treatments for neurodegenerative and neurodevelopmental disorders. Prilenia raised $ 88.5 million thus far and is backed by a group of well-respected investors: Talisman, Forbion, Morningside and Sectoral and ALS Investment Fund. It’s based in Naarden, Netherlands, Herzliya, Israel and Boston, U.S.

Its lead asset, pridopidine, is a first-in-class, highly selective S1R agonist with established safety profile and therapeutic potential in several neurodegenerative diseases.



PROOF-HD is a Phase 3, randomized, double-blind, placebo-controlled study evaluating efficacy, safety and effect of 45mg bid pridopidine on functional capacity, motor and behavioral features in early-stage HD patients.


Prilenia Therapeutics
Limor Ben Har, COO

Instinctif Partners
Melanie Toyne-Sewell / Agnes Stephens
T: +44 (0) 207 457 2020


Source: Prilenia Therapeutics

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