Prilenia to Present Latest Research from its Pridopidine Programs for Huntington Disease and ALS at AAN 2024

Prilenia Therapeutics B.V., a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, today announced the presentation of the latest research from the pridopidine Huntington disease (HD) and amyotrophic lateral sclerosis (ALS) programs at the American Academy of Neurology (AAN) Annual Congress, in Denver, Colorado, April 13-18.

"Pridopidine is now in pre-registration phase in HD, with a first submission planned Mid 2024ⁱ, and is also set to commence a global Phase 3 study in ALS later this year. We are making significant strides forward and this is reflected by presentation of the data at one of the most important medical congresses in the field of Neurology," said Jina Swarz, MD. Ph.D., the recently appointed Chief Medical Officer of Prilenia. "Pridopidine is one of the most advanced investigational new drugs in HD and ALS and has shown consistent treatment benefits across independent measures that are important to patients and families. The presented data advances knowledge in the field, providing important learnings for both clinical practice and for the design of our programs going forward. This advance is evident with novel findings from studies showing benefit with pridopidine in those with HD who are not taking anti-dopaminergic medication (ADMs) and a positive impact on speech and prolonged survival with pridopidine treatment in ALS."

Two oral and one poster presentations will be made at AAN 2024:

Oral Presentations:
Analyses of the Phase 3 Trial of Pridopidine's Outcome On Function in Huntington Disease (PROOF-HD) Demonstrates Efficacy in Participants Without Antidopaminergic Medication
Session name - S30: Movement Disorders: Clinical Trials in Movement Disorders; Program Number: S30.002. Presented by: Michael R Hayden, Founder and CEO, Prilenia Therapeutics; Wed, April 17, at 1:12 PM (MT)

Pridopidine for the Treatment of ALS ? Results from the Phase 2 HEALEY ALS Platform Trial
Session name - S5: ALS and CMT: New Therapeutic Approaches; Program Number: S5.003. Presented by: Jeremy M Shefner, Professor, Department of Neurology, Barrow Neurological Institute; Sun, April 14, at 3:54 PM (MT)

Poster Presentation:
Analyses of the Phase 3 Trial of Pridopidine's Outcome On Function in Huntington Disease (PROOF-HD) Demonstrate Efficacy in Participants Without Antidopaminergic Medication
Session name - P6: Movement Disorders: Huntington's and Other Choreas 1; Program Number: P6.003. Presented by: Randal Hand, Director of Neuroscience, Prilenia Therapeutics; Tue, April 16, from 8:00-9:00a (MT)

About Pridopidine

Pridopidine (45 mg twice daily) is an oral, highly selective and potent investigational S1R agonist that has exhibited a safety and tolerability profile similar to placebo in clinical studies to date. The safety dataset includes over 1,700 patients who have been on pridopidine for up to seven years. The S1R protein is highly expressed in the brain and spinal cord, where it regulates several key processes that are commonly impaired in various neurodegenerative diseases. Activation of the S1R by pridopidine stimulates multiple cellular protective pathways including enhancing autophagy, axonal transport, mitochondrial energy production and respiration, and restores calcium homeostasis. These effects are essential to neuronal function and survival, and lead to neuroprotective effects.

Prilenia holds Orphan Drug designation for pridopidine in HD and ALS in the U.S. and EU. In addition, pridopidine has received Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of HD.

About Prilenia

Prilenia is a clinical stage biotechnology company founded in 2018 focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders. The initial focus of the company has been on HD and ALS.

Prilenia is backed by a group of respected investors including: Forbion, Morningside, Sands Capital, SV Health Investors, Sectoral Asset Management, Talisman, Amplitude Ventures and the ALS Investment Fund. The Company is based in the Netherlands, Israel and Massachusetts in the U.S.

For more information, visit www.prilenia.com and follow us on LinkedIn and X (formerly known as Twitter).

^©2024 Prilenia Therapeutics B.V.

For a copy of this release, visit Prilenia's website at www.prilenia.com.

ⁱ First submission planned for the EU, with other regulatory submissions to follow