Tiny Catalyst soars after rare disease drug scores 'breakthrough' at FDA

Catalyst CEO Patrick McEnany

Tiny Catalyst Pharmaceutical has won a rare victory for a small-cap biotech, snagging the FDA's breakthrough therapy designation (BTD) for a drug designed to treat a lethal autoimmune disease. The treatment, Firdapse, is marketed in Europe by BioMarin ($BMRN) and is now in a Phase III study designed to win an FDA approval. And its shares ($CPRX), battered last fall after its lead drug flopped in a Phase IIb trial for cocaine addiction, instantly zoomed up 48% on the news.

The FDA gave its blessing to Firdapse (amifampridine phosphate), a therapy designed to treat symptoms of Lambert-Eaton myasthenic syndrome (LEMS), which is characterized by muscle weakness.

So far this year the FDA has handed out more than two dozen BTDs, but the field has clearly been dominated by giant companies which either own the drugs outright or partnered on them in the expectation of winning blockbuster approvals. The BTD category was established by law and the FDA has vowed to tailor their regulatory reviews to help speed along the development process and accelerate potential approvals.

Coral Gables, FL-based Catalyst--which had a market cap of only $58 million at the close yesterday--in-licensed the therapy from BioMarin last October, just weeks before a devastating clinical setback. Last February, Catalyst told its investors that amifampridine was slated to read out top-line results in the second quarter of 2014 and be ready for an NDA in 2015 with marketing to begin in 2016--though that timeline is likely to change now.

BioMarin won European approval for the drug in 2009 and has a big stake in Catalyst's fortunes. The $9 billion BioMarin acquired a $5 million stake in the biotech when it executed the licensing pact, handing the money over along with responsibility for an ongoing late-stage trial. And BioMarin gained shares based on the lower value assessed after the Phase IIb failure. Since the deal, some investors have speculated that BioMarin might acquire Catalyst.

Analysts have mixed feelings about the BTD. But Pharmacyclics ($PCYC), allied with J&J ($JNJ), and other biopharma companies on the preferred developer list say that the designation has made a real difference in pushing ahead earlier on new drug applications. So far one solo biotech, Scioderm, has made the list. Novartis ($NVS) leads the industry with a record three BTDs to its credit,

"We are very pleased to have received Breakthrough Therapy Designation for Firdapse and we are excited by the FDA's decision to place our product in a category that may enable expedited development and review for patients with LEMS," said Catalyst CEO Patrick McEnany in a statement. "With no approved or effective symptomatic treatment currently available for LEMS, Firdapse has the potential to be the first-line treatment option for patients with this rare condition."

- here's the press release

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