Huntington’s disease (HD) is an inherited neurodegenerative disease that affects about 100,000 people around the globe with an additional ~ 300,000 individuals at risk (mutations carriers).
The underlying cause of HD is a mutation in the Huntingtin gene, which causes neurons in the brain to degenerate. Neurons suffer increasing stress and lose their ability to communicate with each other, which is manifested in functional, motor, cognitive, emotional, and behavioral symptoms.
HD is usually diagnosed between the ages of 30 and 50, and progresses slowly over 15-20 years.
Patients will progressively lose their ability to hold a job, manage their finances, perform household tasks and/or important activities of daily living including taking care of themselves.
HD is one of the most devastating neurodegenerative disorders, impacting not only patients but their families as well.
At the present time, there is no treatment to slow progression of disease. Sadly, every child of an affected parent has a 50% chance of having inherited the DNA change causing HD.
Pridopidine for HD
Pridopidine is currently being assessed in the ongoing phase 3 global clinical trial PROOF-HD (PRidopidine Outcome On Function in Huntington’s Disease). The successful outcome of PROOF-HD may lead to its approval as a treatment for Huntington’s Disease.
In prior clinical trials in HD patients, pridopidine demonstrated a beneficial effect in maintaining functional capacity after 1 year.
HD patients and families highlight decreased functional capacity as a major burden on daily life. The Total Functional Capacity (TFC) scale is widely used by clinicians to assess disease stage and progression. TFC captures changes in HD patients' capacity to continue working, perform household activities, eat, dress, walk, and complete simple tasks.
Pridopidine Maintains Functional Capacity (TFC)
Pridopidine is the first drug to show maintenance of TFC at one year. This effect may also be seen and maintained for five years, as demonstrated in an open-label trial
Pridopidine beneficial effects on TFC may be maintained over 5 years
Prilenia holds Orphan Drug Designation for pridopidine in Huntington’s Disease (HD) in both the U.S. and Europe. In addition, pridopidine recently received Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of HD. Drugs that receive Fast Track designation may be eligible for more frequent communications with the FDA and may also qualify for accelerated approval and priority review of new drug applications.
The PROOF-HD phase 3 study is being conducted in~ 60 sites in collaboration with the Huntington Study Group (HSG) in North America and Europe.
It has recently completed patient enrollment ahead of schedule and with numbers over the enrollment target.
The purpose of the study is to evaluate the effect of pridopidine on functional capacity, as well as on motor and behavioral features over 65 weeks.
The study is designed to confirm previous findings that demonstrated pridopidine’s effect in maintaining functional capacity in patients with early HD.
Patients who complete the double-blind placebo-controlled study will have the opportunity to enroll into the open-label study and receive pridopidine (no patients will receive placebo in the open-label study).
Learn more about pridopidine for HD: