Epilepsies, Partial

CORAL GABLES, Fla., March 28, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare and difficult-to-treat diseases, today announced its endorsement of the inaugural Lambert-Eaton myasthenic syndrome ("LEMS") Awareness Day. This landmark event, now officially observed annually on March 30th, signifies a meaningful milestone for the LEMS community. The chosen date holds profound historical significance, coinciding with the publication of Dr. Edward Lambert and Dr. Lee Eaton's pioneering research on LEMS, which has greatly advanced the understanding and treatment of this rare neuromuscular disorder. "Creating an awareness day for LEMS is a huge win for our community," said Price Wooldridge, Founder and Board Member of the LEMS Family Association. "This brings us one step closer to our goal, improving the awareness of our rare condition in the larger population. We couldn't be more thrilled to celebrate the milestone of this first LEMS Awareness Day together." "In the realm of rare diseases, awareness is the key that unlocks paths to diagnosis and treatment. By shedding light on LEMS, we pave the way for better support, understanding, and care for those who need it most," stated Richard J. Daly, CEO of Catalyst. "At Catalyst, we are proud to stand alongside the LEMS Family Association in honoring this pivotal event. Together, we forge pathways of support and understanding for those affected by rare diseases, illuminating a brighter future for all." LEMS is a rare neuromuscular disease that affects at least 3,600 and potentially up to 5,400 LEMS patients in the United States. For more information, please visit www.lemsfamily.org and www.lemsaware.com. About Catalyst Pharmaceuticals, Inc.With exceptional patient focus, Catalyst is committed to developing and commercializing innovative first-in-class medicines that address rare and difficult-to-treat diseases. Catalyst's flagship U.S. commercial product is FIRDAPSE® (amifampridine) Tablets 10 mg, approved for the treatment of Lambert-Eaton myasthenic syndrome ("LEMS") for adults and for children ages six to seventeen. In January 2023, Catalyst acquired the U.S. commercial rights to FYCOMPA® (perampanel) CIII, a prescription medicine approved in people with epilepsy aged four and older alone or with other medicines to treat partial-onset seizures with or without secondarily generalized seizures and with other medicines to treat primary generalized tonic-clonic seizures for people with epilepsy aged 12 and older. Further, Canada's national healthcare regulatory agency, Health Canada, has approved the use of FIRDAPSE for the treatment of adult patients in Canada with LEMS. On July 18th, 2023, Catalyst acquired an exclusive license for North America for AGAMREE® (vamorolone) oral suspension 40 mg/mL, a novel corticosteroid treatment for Duchenne Muscular Dystrophy. AGAMREE previously received FDA Orphan Drug and Fast Track designations and was approved by the FDA for commercialization in the U.S. on October 26th, 2023. AGAMREE became available in the U.S. by prescription on March 13th, 2024. For more information about Catalyst Pharmaceuticals, Inc., visit the Company's website at www.catalystpharma.com. For Full Prescribing and Safety Information for FIRDAPSE®, visit www.firdapse.com. For Full Prescribing Information, including Boxed WARNING for FYCOMPA®, please visit www.fycompa.com. For Full Prescribing Information for AGAMREE®, please visit https://www.agamree.com. Forward-Looking StatementsThis press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. 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 those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2023 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. Source: Catalyst Pharmaceuticals, Inc.

CORAL GABLES, Fla., March 27, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare and difficult-to-treat diseases, today announced that Richard J. Daly, CEO of Catalyst, along with other members of Catalyst’s management team, will participate in the 2024 Cantor Virtual Muscular Dystrophy Symposium taking place virtually on April 2-3, 2024. Presentation Details:Event:Cantor Virtual Muscular Dystrophy SymposiumDate: April 2, 2024Time:3:10 PM ET The presentation webcast will be available on the Investors section of the Company's website, and a replay will be accessible for at least 14 days. About Catalyst PharmaceuticalsWith exceptional patient focus, Catalyst is committed to developing and commercializing innovative first-in-class medicines that address rare and difficult-to-treat diseases. Catalyst's flagship U.S. commercial product is FIRDAPSE® (amifampridine) Tablets 10 mg, approved for the treatment of Lambert-Eaton myasthenic syndrome ("LEMS") for adults and for children ages six to seventeen. In January 2023, Catalyst acquired the U.S. commercial rights to FYCOMPA® (perampanel) CIII, a prescription medicine approved in people with epilepsy aged four and older alone or with other medicines to treat partial-onset seizures with or without secondarily generalized seizures and with other medicines to treat primary generalized tonic-clonic seizures for people with epilepsy aged 12 and older. Further, Canada's national healthcare regulatory agency, Health Canada, has approved the use of FIRDAPSE for the treatment of adult patients in Canada with LEMS. Finally, on July 18, 2023, Catalyst acquired an exclusive license for North America for AGAMREE® (vamorolone) oral suspension 40 mg/mL, a novel corticosteroid treatment for Duchenne Muscular Dystrophy. AGAMREE previously received FDA Orphan Drug and Fast Track designations and was approved by the FDA for commercialization in the U.S. on October 26, 2023. AGAMREE became commercially available by prescription in the U.S. on March 13, 2024. For more information about Catalyst Pharmaceuticals, Inc., please visit the Company's website at www.catalystpharma.com. For Full Prescribing and Safety Information for FIRDAPSE®, please visit www.firdapse.com. For Full Prescribing Information, including Boxed WARNING for FYCOMPA®, please visit www.fycompa.com. For Full Prescribing Information for AGAMREE®, please visit www.agamree.com. Forward-Looking StatementsThis press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. 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 those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2023 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. Source: Catalyst Pharmaceuticals, Inc.

Praxis Precision Medicines revealed Phase IIa findings for its experimental PRAX-628 therapy, demonstrating a 100% complete response in the high-dose group of patients with photosensitive epilepsy. The positive readout Tuesday paves the way for Praxis to advance the programme later this year."The strength and consistency of response across both study arms, combined with a continued positive tolerability and safety profile, build on our earlier conviction that PRAX-628 has the potential to be the first precision sodium channel modulator for focal epilepsy patients," said CEO Marcio Souza.The study included 10 epilepsy patients who demonstrated photo paroxysmal response (PPR) during screening, with eight eligible for assessment. PPR studies can reveal abnormal patterns of brain activity in response to flashing or flickering lights. Analysing EEG signatures after intermittent photic stimulation can serve as a gauge for the effectiveness of potential anti-epileptic treatments, according to the company.The study is evaluating two dose levels. Earlier this year, Praxis said a preliminary analysis of the lower 15mg cohort had exceeded its expectations, driving up shares by 23% at the time. On Tuesday, the company said four of the five patients (80%) in that group achieved a complete response, which was defined as the complete elimination of generalised PPR events at any assessment period versus baseline.In the higher 45mg cohort, all three patients (100%) achieved a complete response. The combined response rate was 88% across the two cohorts. In a note prior to the readout, Jefferies analysts said they viewed ≥50% complete/partial suppression as "an important threshold to meet/exceed" in to order to demonstrate proof-of-concept.