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PIPELINE & CLINICAL TRIALS 

PIPELINE

INDICATION

PRE CLINICAL

PHASE 1

PHASE 2

PHASE 3

Prodromal HD

Rett Syndrome

Fragile X

Neurodegenerative eye disease

Parkinson's Disease

Pridopidine, Prilenia’s lead asset is a first in class selective and potent Sigma-1-receptor agonist with neuroprotective properties. It is administered in a small easy-to-swallow capsule twice a day. Pridopidine has an extensive safety and tolerability profile and was already evaluated in >1300 people including long term evaluation for >5 years.


Prilenia is currently developing pridopidine for the treatment of Huntington’s Disease in a global phase 3 clinical study, PROOF-HD. In addition to this, pridopidine is being evaluated in a clinical trial for the treatment of ALS in a phase 2/3 study in collaboration with The Healey Center.


Prilenia is continuing the clinical and preclinical evaluation of pridopidine in various neurodegenerative and neurodevelopmental indications and plans to initiate additional clinical studies in the future.

CLINICAL TRIALS

Pridopidine Outcome On Function in Huntington’s Disease (PROOF-HD) is an active global phase 3 trial conducted in collaboration with the Huntington Study Group (HSG). 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 and clinically meaningful endpoint that measures the patient’s ability to conduct certain activities. Pridopidine is the first treatment to ever achieve maintenance of function in early HD patients (as measured by TFC) in the previous PRIDE-HD study. PROOF-HD is the only late-stage trial in HD targeting clinical progression.

The PROOF-HD study enrolled the first participants in the US in October 2020, followed by the first European participant in January 2021. It is on track to reach its target readout in early 2023.

THE HEALEY ALS PLATFORM TRIAL

The platform trial is designed to accelerate development of promising new treatments by enabling investigators to simultaneously assess multiple therapeutic candidates, enabling pooling of placebo data across regimens (which minimizes the number of patients on placebo).


Pridopidine one of the first 4 drug candidates being studied in the Healey ALS platform trial. It was selected from an international competition of >30 potential therapeutics by an independent expert review committee based on compelling human genetic validation data, efficacy in preclinical models and favorable safety profile. 


The Phase 2/3 clinical study enrolled its first participant in January 2021 and is on track to generate results for the pridopidine regimen in Q3 2022.