Prilenia

Company Description

Prilenia is a clinical late-stage company led by Michael Hayden, MD, PhD focused on developing new treatments for neurodegenerative disorders. Prilenia’s lead asset, pridopidine, is under development for the treatment  of Huntington’s Disease (HD) and Amyotrophic Lateral Sclerosis (ALS).

Pridopidine is an oral drug candidate that selectively binds and activates a protein called the Sigma-1 receptor (S1R). The S1R is highly expressed in the brain where it regulates several cellular mechanisms which are important for neuronal health and are impaired in HD. Activation of the S1R by pridopidine enhances these mechanisms and improves neuronal functions and survival.

Pridopidine is currently being assessed for the treatment of early HD in the actively recruiting PROOF-HD phase 3 global trial. The primary endpoint is the effect of pridopidine on total functional Capacity (TFC) at 65 weeks. The Total Functional Capacity (TFC) scale is a widely used and acceptable tool to capture changes in HD patients’ capacity to continue working, perform household activities, eat, dress, walk, and complete simple tasks.

Pridopidine is the first drug to show in prior trials maintenance of functional capacity (measured as TFC) in HD patients. Extensive safety data from ~1000 HD patients demonstrates that 45 mg BID, the dose used in PROOF-HD, is safe and well-tolerated with a placebo-like adverse events profile.  

Prilenia is dedicated to using science to bring hope to patients and their families by developing much needed treatment options that may maintain function and quality of life.

Links

1. About pridopidine: https://www.prilenia.com/about-pridopidine
2. Prilenia patients and families page: https://www.prilenia.com/patients-and-families
3. HSG PROOF-HD website: https://huntingtonstudygroup.org/proof-hd/
4. PIs Andy Feigin and Ralf Reilmann discuss PROOF-HD: https://touchneurology.com/huntingtons-disease/conference-hub/andrew-feigin-and-ralf-reilmann-ad-pd-2021-the-proof-hd-study/

Documents

1. Q&A deck
2. List of sites
3. Clinical papers:
   1. McGarry et al., 2020 “Effects of Pridopidine on Functional Capacity in Early-Stage Participants from the PRIDE-HD Study”
   2. McGarry et al., 2020 “Additional Safety and Exploratory Efficacy Data at 48 and 60 Months From Open-HART, an Open-Label Extension Study of Pridopidine in Huntington Disease”
4. Preclinical papers
   1. Naia et al., 2021 “The Sigma-1 Receptor Mediates Pridopidine Rescue of Mitochondrial Function in Huntington Disease Models”.
   2. Eddings et al., 2019 “Pridopidine Protects Neurons from Mutant-Huntingtin Toxicity via the Sigma 1-Receptor”
   3. Ryskamp et al., 2017 “The sigma-1 receptor mediates the beneficial effects of pridopidine in a mouse model of Huntington disease”

Contact information

info@prilenia.com

+972-77-5558482