Neurodegenerative eye 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.
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 completed patient enrollment ahead of schedule and with numbers over the enrollment target. 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 has recently completed patient enrollment for the pridopidine arm across 52 sites from the Northeast ALS Consortium (NEALS) in the United States.
We understand that, in some cases, patients with serious diseases or conditions may not be eligible for clinical trials and may not have other treatment options. Under limited circumstances, we will consider making investigational medicines available for individual patients to treat a serious disease or condition outside of a clinical trial under so called early access. Early access is generally referred to as “expanded access” or “compassionate use”.