Phase III Study for PKAN Treatment Underway

A biopharmaceutical company based in California called Retrophin recently announced that the first patient has been administered treatment in their phase III study in to test RE-024 (fosmetpantotenate) as a treatment for patients with pantothenate kinase-associated neurodegeneration (PKAN).
The study, called FORT, will be placebo-controlled study and will an enroll an international range of patients. Retrophin plans to enroll 82 PKAN patients among the ages of 6 to 65 years in FORT.
Panthothenate Kinase-Associated Neurodegeneration, or PKAN, is a rare, inherited neurological movement disorder.

The rare disease is characterized by progressive degeneration of certain regions in the central nervous system. This disorder is the most common out of all neurodegeneration with brain iron accumulation (NBIA). NBIA is characterized as a group of clinical disorders that are associated with progressive abnormal involuntary movements.

Even though most common in this group, for a while, treatment for PKAN has historically been hard to pinpoint. There’s no specific treatment for patients with PKAN, but rather treatments aim to aid specific symptoms in each unique individual.

Needless to say, the PKAN community has long been in need of an actual, effective, overarching treatment because research has just been working on developing something more general.

In light of this, the news that a phase III trial for a specific PKAN treatment is underway is very exciting. Since PKAN causes lower levels of Coenzyme A, the new treatment RE-024 works as a replacement therapy for small molecules that initiates the creation of more Coenzyme A, treating the disease from its root biological cause.
Head of R and D for Retrophin, Bill Rote, PhD, stated his optimism in a press release, where he claimed that the first dosage was an

“important milestone for the RE-024 program.”

Further, Dr. Rote believes that RE-024 has the ability to generate an extremely meaningful difference for those affected by PKAN.

The endpoint of this study is an improvement in overall disease condition, and it will be measured on the Pantothenate Kinase-Associated Neurodegeneration Activities of Daily Living (PKAN-ADL) scale. It will follow the patients from day 0 to the end of the 24-week trial of treatment, and hopefully by day 24, significant improvement will have taken place.

If this phase III trial meets this primary endpoint, it will help prove this treatment’s efficacy to the U.S. FDA during the approval process. Our fingers are crossed!

To read more about this new clinical trial, click this link.

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