Tags: Catalyst | Pharma | Drug | Study

Catalyst Pharma's Lead Drug Succeeds in Late-stage Study

Monday, 29 September 2014 07:12 PM

Catalyst Pharmaceutical Partners Inc. said its lead drug was found superior in treating symptoms associated with a rare autoimmune disorder, compared with a placebo.

The company's shares rose about 22 percent in extended trading on Monday.

The late-stage trial tested the drug, Firdapse, in 38 patients with Lambert-Eaton Myasthenic Syndrome, the company said.

Named after two neurologists at a Minnesota-based Mayo Clinic — Edward Lambert and Lee Eaton — the neuromuscular disease causes progressive muscle weakness, and is often associated with cancer.

Firdapse is already sold in the European Union by BioMarin Pharmaceutical Inc, which out-licensed the North American rights of the drug to Catalyst in 2012.

The drug will soon become accessible under the U.S. Food and Drug Administration's Expanded Access Program, which allows registered physicians to prescribe unapproved drugs to seriously ill patients when no other options exist.

Catalyst Chief Executive Patrick McEnany told Reuters he expects the first patient to be dosed around the end of October under the program.

Analysts said they expected the company to launch Firdapse by early 2016, if approved, and target the estimated 3,500 LEMS patients in the United States.

The other available treatment in the United States is a compounded drug, 3,4-DAP, which is the same as Firdapse, from privately held Jacobus Pharmaceutical, under compassionate use programs.

While Jacobus is also testing 3,4-DAP in a mid-stage study, its infrastructure is still not FDA-compliant, analysts said.

"Catalyst will win FDA approval for Firdapse first," Aegis Capital analyst Raghuram Selvaraju said.

"Once they have that approval — since they have orphan-drug status — they will effectively be able to lock Jacobus out of the market for seven years."

An orphan-drug status, granted to drugs developed to treat rare diseases, provide the developer several incentives including a period of marketing exclusivity.

Data gathered for Catalyst by independent researchers show that reimbursement agencies would support a price point for Firdapse of around $175,000 per patient per year, Selvaraju said.

CEO McEnany said Firdapse has the potential to rake in sales exceeding $100 million annually.

Firdapse could generate peak sales of $300 million by 2022, H.C. Wainwright analyst Andrew Fein estimated.

The drug generated net revenue of $9.3 million in the first six months of 2014 in the European Union, where it is also designated as an orphan drug.

Firdapse or 3,4-DAP is a potassium channel inhibitor that enhances the release of the neurotransmitter acetylcholine to relieve muscle weakness.

The company announced in January the results of a study that showed Firdapse did not have an adverse effect on the heart rate of patients.

Coral Gables, Florida-based Catalyst's stock closed at $2.99 on the Nasdaq.

© 2020 Thomson/Reuters. All rights reserved.

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Catalyst Pharmaceutical Partners said its lead drug was found superior in treating symptoms associated with a rare autoimmune disorder, compared with a placebo.
Catalyst, Pharma, Drug, Study
Monday, 29 September 2014 07:12 PM
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