Biopharmaceutical firm Retrophin Inc. (RTRX:NASDAQ) shares are trading much lower today as the company announced that the Phase 3 FORT Study evaluating the safety and efficacy of fosmetpantotenate compared to placebo in patients with pantothenate kinase-associated neurodegeneration (PKAN) did not meet its primary endpoint and did not demonstrate a difference between treatment groups. Though fosmetpantotenate was observed to be generally safe and well tolerated in the study, the study also did not meet its secondary endpoint.
The company's CEO, Eric Dube, Ph.D, commented, "We are very disappointed in the topline results from the FORT Study, particularly because we have seen the devastating impact of PKAN on patients and their families, and a significant unmet need remains with no approved treatment option. We would like to thank the patients, their caregivers, study investigators and our employees, whose dedication made this study possible...We will work closely with the investigators to further analyze the results of the study and share them with the PKAN community to contribute to the growing knowledge of this rare disorder."
The firm indicated that the data from the FORT Study are expected to be presented at an upcoming scientific congress, and that Retrophin will be working with study investigators to determine the appropriate next steps for the FORT Study, including the ongoing open-label extension of the study.
The company advised that it still remains focused on progressing its two other pivotal Phase 3 programs evaluating sparsentan for the treatment of focal segmental glomerulosclerosis (FSGS) and IgA nephropathy, and continuing the advancement of its commercial portfolio.
The FORT Study was an international, randomized, double-blind, placebo-controlled, Phase 3 clinical trial assessing the safety and efficacy of fosmetpantotenate in 84 patients with PKAN, a rare, genetic and life-threatening neurological disorder estimated to affect up to 5,000 people worldwide. PKAN is characterized by a host of progressively debilitating symptoms that typically begin in early childhood. People suffering from PKAN may experience movement disorders such as dystonia (sustained muscle contraction leading to abnormal posture), rigidity, dysphagia (problems swallowing), and twisting and writhing, as well as visual impairment.
The firm notes that PKAN is caused by a mutation in the PANK2 gene, which encodes a critical protein that phosphorylates vitamin B5 (pantothenate), generating phosphopantothenate. The disruption of this metabolic pathway ultimately leads to decreased levels of coenzyme A (CoA), which is essential in biochemical reactions impacting energy metabolism, membrane integrity, signaling and other critical processes.
Retrophin describes its business as a biopharmaceutical company specializing in identifying, developing and delivering life-changing therapies to people living with rare disease. The Company's approach centers on its pipeline featuring late-stage assets targeting rare diseases with significant unmet medical needs, including fosmetpantotenate for pantothenate kinase-associated neurodegeneration (PKAN), and sparsentan for focal segmental glomerulosclerosis (FSGS) and IgA nephropathy (IgAN), disorders characterized by progressive scarring of the kidney often leading to end-stage renal disease. Retrophin's R&D efforts are supported by revenues from the company's commercial products Chenodal, Cholbam and Thiola.
RTRX shares opened lower today at $12.51 (-$4.89, -28.10%) from yesterday's closing price of $17.40. Today, the stock has traded between $11.84 and $12.98/share setting a 52-week intraday low price on greater than 15 times average volume. At present, shares are trading at $12.84 (-4.56%, -26.21%).
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