“PKAN is a rare and life-threatening neurological disorder characterized by a host of progressively debilitating movement disorders, and it is encouraging to see persistent improvement of function over a 30-month period in this patient with PKAN who received fosmetpantotenate treatment,” said
A poster presentation titled, “Thirty Months of Treatment of a Patient with Pantothenate Kinase-Associated Neurodegeneration (PKAN) with Fosmetpantotenate (RE-024), A Phosphopantothenate Replacement Therapy,” reported results from an open label, uncontrolled, physician-initiated treatment showing that an adult patient with PKAN experienced persistent improvement of functioning over a 30-month treatment period upon receiving fosmetpantotenate, with the newly-reported results covering months 15 to 30. These improvements were measured by the treating physician using the Unified Parkinson’s Disease Rating Scale (UPDRS) Part II and the EuroQol 5-Dimensional 3-Level Scale (EQ-5D-3L). The treating physician reported that gait, and thus functional independence, also improved, and this improvement remained stable over the 30-month treatment period. Symptoms of dystonia also showed stabilization, and symptoms of parkinsonism were more variable over the 30-month time period, though overall did not show deterioration. Data describing the patient’s initial 12-month period of treatment were originally presented at the 2016
Also at the CNS 26th Annual Meeting, the Company presented a poster titled, “Fosmetpantotenate (RE-024) for Pantothenate Kinase-Associated Neurodegeneration (PKAN): Maximum Tolerated Dose (MTD) and Therapeutic-Dose Food Effect Studies in
About the Phase 3 FORT Study
The FOsmetpantotenate Replacement Therapy (FORT) Study is an international, randomized, double-blind, placebo-controlled study evaluating fosmetpantotenate for the treatment of PKAN. This Phase 3 clinical trial commenced patient dosing in
The PKAN-ADL is a novel, PKAN-specific, patient-reported outcome scale measuring motor abilities to function in daily living for patients with PKAN. The scale is an adaptation of Part II of the comprehensive and widely-referenced Unified Parkinson's Disease Rating Scale (UPDRS). For the purposes of this trial, the UPDRS was adapted to be optimally relevant to PKAN through a systematic revision involving experts, patient advocacy leaders and regulatory interaction.
The FORT Study is being conducted under a Special Protocol Assessment (SPA) agreement, which indicates concurrence by the U.S. Food and Drug Administration (
For additional information about the FORT Study and to learn more about eligibility, patients can visit pkanfortstudy.com.
About Pantothenate Kinase-Associated Neurodegeneration (PKAN)
PKAN is a rare, genetic and life-threatening neurological disorder 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. PKAN is estimated to affect up to 5,000 people worldwide.
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).
Fosmetpantotenate (previously known as RE-024) is a novel investigational small molecule replacement therapy designed to pass the blood-brain barrier and be converted to phosphopantothenic acid (PPA), with the potential to be the first approved treatment targeting the underlying cause of PKAN. PPA synthesis is a key step in the biosynthesis of CoA, which is essential in biochemical reactions impacting energy metabolism, membrane integrity, signaling and other critical processes.
Preclinical findings suggest fosmetpantotenate has the ability to pass the blood-brain barrier and restore CoA levels. In a Phase 1 study, fosmetpantotenate was found to be safe and well-tolerated in healthy volunteers. Fosmetpantotenate has been granted orphan drug designation for the treatment of PKAN by the FDA and European Commission, as well as Fast Track status in the U.S.
This press release contains "forward-looking statements" as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words "may", "might", "believes", "thinks", "anticipates", "plans", "expects", "intends" or similar expressions. In addition, expressions of our strategies, intentions or plans are also forward-looking statements. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with the Company’s business and finances in general, success of its commercial products, as well as risks and uncertainties associated with the Company's preclinical and clinical stage pipeline. Specifically, with respect to fosmetpantotentate, the Company faces risk that the Phase 3 clinical trial of fosmetpantotenate will not demonstrate that fosmetpantotenate is safe or effective or serve as the basis for an NDA filing as planned; risk that observations from the physician-initiated treatment that is the subject of this press release will not be replicated in the FORT study; risk that fosmetpantotenate will not be approved for efficacy, safety, regulatory or other reasons, risk associated with enrollment of clinical trials for rare diseases and risk the clinical trial may not succeed or may be delayed for safety, regulatory or other reasons. The Company faces risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates; risk relating to the Company's dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and intellectual property rights of third parties; and risks and uncertainties relating to competitive products and technological changes that may limit demand for the Company's products. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Investors are referred to the full discussion of risks and uncertainties as included in the Company's most recent Form 10-K, Form 10-Q and other filings with the
Vice President, Investor Relations & Corporate Communications
Source: Retrophin, Inc.