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Catalysts That Could Move Catalyst Pharma

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Shares of Catalyst Pharmaceuticals Inc. (CPRX) have lost 26% of their value so far this year while the iShares Nasdaq Biotechnology ETF (IBB) has fallen about 2.6% during the same period.

Catalyst Pharma is a biopharmaceutical company focused on developing therapies for people with rare debilitating, chronic neuromuscular and neurological diseases.

The Company's resubmitted New Drug Application for Firdapse for the treatment of Lambert-Eaton myasthenic syndrome is under FDA review - with a decision expected today (November 28).

Lambert-Eaton myasthenic syndrome, or LEMS in short, is a rare autoimmune disorder characterized by the gradual onset of muscle weakness, especially of the pelvic and thigh muscles. Approximately 60 percent of LEMS cases are associated with a small cell lung cancer (SCLC), and the onset of LEMS symptoms often precedes the detection of the cancer. (Source: National Organization for rare Disorders).

Catalyst licensed the North American Rights to Firdapse from BioMarin Pharmaceutical Inc. (BMRN) in October 2012. The Company's attempt to get approval for Firdapse in the U.S. in 2015 went in vain as the FDA determined that the NDA for Firdapse was not sufficiently complete and even refused to accept the application then.

The Firdapse NDA, which was resubmitted in March of this year, also includes positive top-line results from a second phase III clinical trial of Firdapse for the treatment of LEMS, which was conducted under a protocol agreed to by the FDA through the Special Protocol Assessment process.

In the European Union, Firdapse received the regulatory nod in December 2009, and has been marketed there by BioMarin since April 2010.

Firdapse fetched annual revenue of $18.8 million in 2017 for BioMarin compared to $18 million in 2016.

A privately held company Jacobus Pharmaceutical is providing 3,4-Diaminopyridine base (3,4-DAP), the same ingredient as in Catalyst's Firdapse, for LEMS patients for free for a number of years through the FDA's Expanded Access (compassionate use) program. However, this medication is not approved in the U.S.

Firdapse is also being evaluated for the treatment of congenital myasthenic syndromes (CMS) and myasthenia gravis patients with autoantibodies to muscle-specific tyrosine kinase (MuSK), and phase III trials in these indications are underway. The top-line results from the phase III CMS and MuSK-MG trials are expected in the second half of 2019.

The potential of Firdapse is also being explored in Spinal Muscular Atrophy (SMA) Type 3, ambulatory, and a phase III proof-of-concept clinical study is ongoing. The study is designed to include approximately 12 patients, with top-line results expected in the first half of 2020.

CPRX has traded in a range of $2.18 to $4.51 in the last 1 year. The stock closed Tuesday's trading at $2.91, down 0.68%.

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