Catalyst Pharmaceuticals Announces Its Support of Spinal Muscular Atrophy Awareness Month

Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq: CPRX), a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today announced its support of Spinal Muscular Atrophy (SMA) Awareness Month. Taking place in August, SMA Awareness Month involves activities worldwide to spotlight the work the SMA community does year-round to raise awareness of the debilitating and potentially deadly disease.

“We are proud to be part of the global movement to raise awareness of SMA and recognize the importance of developing new evidence-based medicines required to improve the lives of patients, families and caregivers afflicted with SMA,” said Patrick J. McEnany, Chairman and CEO of Catalyst.

“We are pleased to be leading the Catalyst sponsored pilot study of Firdapse® as a possible treatment option for SMA Type 3,” stated Dr. Lorenzo Maggi at the Carlo Besta Neurological Institute in Milan, Italy. “Defects in the neuromuscular junction appear to be concomitant to degeneration of motor neurons. The literature suggests that amifampridine may have clinical utility in these patients through its action on the impaired neuromuscular junction transmission.”

Out of every 10,000 births, one baby is born with SMA. SMA is a genetic disease that affects the part of the nervous system that controls voluntary muscle movement. It is caused by malfunctioning genes that affect how nerve cells and muscle cells communicate across the neuromuscular junction—the place where nerve cells meet muscle cells. Patients with SMA can have severe muscle weakness in the upper arms, legs, and shoulders. In some instances, SMA may lead to paralysis or death.

About Catalyst Pharmaceuticals

Catalyst Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton myasthenic syndrome (LEMS), anti-MuSK antibody positive myasthenia gravis (MuSK-MG), congenital myasthenic syndromes (CMS), and spinal muscular atrophy (SMA) Type 3. Catalyst's new drug application for Firdapse® (amifampridine) 10 mg tablets for the treatment of adults with LEMS was approved in November 2018 by the U.S. Food & Drug Administration ("FDA"), and Firdapse is now commercially available in the United States. Prior to its approval, Firdapse for LEMS had received breakthrough therapy designation and orphan drug designation from the FDA.

Firdapse is being evaluated in clinical trials for the treatment of MuSK-MG, CMS, and SMA Type 3 and has received Orphan Drug Designation from the FDA for myasthenia gravis and CMS. Firdapse (amifampridine) 10 mg tablets is the first and only approved drug in Europe for the symptomatic treatment in adults with LEMS.

Forward-Looking Statements

This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including (i) whether Catalyst’s proof-of-concept study evaluating Firdapse for the treatment of SMA Type 3 will be successful, and (ii) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2018 and its other filings with the U.S. Securities and Exchange Commission (SEC), could adversely affect Catalyst. Copies of Catalyst's filings with the SEC are available from the SEC, may be found on Catalyst's website, or may be obtained upon request from Catalyst. Catalyst does not undertake any obligation to update the information contained herein, which speaks only as of this date.

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Catalyst Pharmaceuticals
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