NBIA NEWS & INFORMATION

Clinical trial of Retrophin drug fails; Shows no benefit for PKAN patients

August 2019

A much-anticipated drug therapy has failed to show any benefit for individuals affected with PKAN, or Pantothenate Kinase-Associated Neurodegeneration, one of the most common forms of NBIA.

The drug’s maker, Retrophin Inc., announced the disappointing results Aug. 22 for its Phase 3 Fosmetpantotenate Replacement Therapy, or FORT, study.

Seventy-eight PKAN individuals had completed the 24-week randomized, double-blind study, meaning that neither the patients nor the doctors knew who was randomly selected to get the drug or the placebo. At the end of the study, 76 patients decided to participate in the open-label program in which all received the drug.

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Retrophin CEO, Eric Dube, wrote a letter to the NBIA Disorders Association and NBIA community regarding the disappointing results of this clinical trial.

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Deferiprone trial results produce positive findings for some with PKAN

August 2019

The long-awaited results from the first international clinical trial for NBIA — testing deferiprone in individuals with PKAN — are in.

They show that the iron-chelating drug slowed the progression of the disorder in older patients with a later-onset, or atypical, form of PKAN, but did not have a similar benefit for younger patients with classic PKAN, which starts in early childhood.

In addition to those findings, the study showed that the drug successfully reduced the amount of accumulated iron in the brain for PKAN individuals, regardless of onset age.

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Spoonbill Foundation Contract

May 2019

NBIA Disorders Association recently signed a contract for $50,000 with the Spoonbill Foundation, a nonprofit founded by Drs. Susan Hayflick and Penny Hogarth from OHSU, to help support the design, build, and testing phases of the database for the upcoming CoA-Z clinical trial in PKAN. This database, built in an application called REDCap Cloud, will be structured to meet the highest FDA standards. The database will be customized to meet the exact needs of the study, including the necessary fields to collect data and track specimens and study product, and built-in checks to monitor the data quality and accuracy. Developing this type of sophisticated database will directly promote the success of the trial and pave the way for future steps beyond the trial.