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Retrophin Down on Neurological Disorder Drug Study Failure

Zacks Equity Research

Shares of Retrophin, Inc. RTRX lost 22% after it announced the failure of its late-stage study evaluating fosmetpantotenate in patients with pantothenate kinase-associated neurodegeneration (PKAN), a rare genetic neurological disorder.

Notably, shares of the company have plunged 40.1% in the year so far compared with the industry’s decline of 1.2%.


The phase III study, FORT, was evaluating the safety and efficacy of fosmetpantotenate compared to placebo in 84 PKAN patients. Patients received either three times daily dosing of fosmetpantotenate or placebo using a 1:1 randomization over 24 weeks. The primary endpoint of the study was the change from baseline in the PKAN-ADL scale through 24 weeks of treatment. After completing the 24-week treatment period, all patients were eligible to receive fosmetpantotenate as part of an open-label extension.

However, neither did the study meet its primary endpoint nor did it demonstrate a difference between treatment groups. The study also did not meet its secondary endpoint.

The company will discuss the study results with its investigators and determine the appropriate next steps for the FORT Study, including the ongoing open-label extension of the study.

Investors were disappointed as fosmetpantotenate was a late-stage candidate and a potential approval would have boosted the top line significantly. Moreover, the failure of the study will most likely lead to the termination of the entire clinical program on this candidate.

As a result of this failure, Retrophin will now focus on the other pipeline candidate — sparsentan. The candidate is being evaluated in two phase III programs for the treatment of focal segmental glomerulosclerosis (FSGS) and IgA nephropathy. The company secured a license to sparsentan from Ligand Pharmaceuticals, Inc. LGND and Bristol-Myers Squibb Company BMY.

Retrophin also has three commercial products — Chenodal, Cholbam and Thiola — in its portfolio. In June 2019, the FDA approved 100 mg and 300 mg tablets of Thiola EC (tiopronin), a new enteric-coated formulation of Thiola (tiopronin).

Zacks Rank & Stock to Consider

Retrophin currently carries a Zacks Rank #4 (Sell). A better-ranked stock in the biotech sector is Acorda Therapeutics ACOR, which carries a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Acorda’s loss per share estimates have narrowed from $3.59 to $2.74 for 2019 in the past 60 days.

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Bristol-Myers Squibb Company (BMY) : Free Stock Analysis Report
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