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  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Botox (onabotulinumtoxin A) / GSK, AbbVie
    New P2 trial:  Firdapse for Post-BOTOX Vocal Weakness (clinicaltrials.gov) -  Nov 5, 2021   
    P2,  N=10, Recruiting, 
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    [VIRTUAL] A Family with an Autosomal Dominantly Inherited CHRNA1 Mutation and Congenital Myasthenia Syndrome (ANA 2021) -  Oct 21, 2021 - Abstract #239; Author: Jaudat Masood, MD1; Jun Li, MD., PhD2; Ryan Castoro, DO1;    
    This mutation was previously reported in compound heterozygosity in a CMS cases however with care ful phenotyping and whole genome sequencing we deter mined this mutant as likely causative in heterozygosity. Additionally we show clinical response to 3,4DAPP in this phenotype, suggestive of a slow-channel physiology.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Journal:  Lambert-Eaton myasthenic syndrome revealed after surgery of lung adenocarcinoma. (Pubmed Central) -  Oct 4, 2021   
    One month later patient present a muscle weakness in both lower limbs and fatigability followed by an inability to walk. The diagnosis of LEMS was made from the distinctive electromyogram (EMG) findings and a treatment with Amifampridine (3, 4-diaminopyridine phosphate [3, 4-DAP]) was prescribed with evident efficacy for symptoms.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    [VIRTUAL] A juvenile case of Lambert-Eaton myasthenic syndrome resembling a congenital Myasthenic Syndrome (EAN 2021) -  May 30, 2021 - Abstract #EPO-473; Author: B. Vicente; M. Oliveira Santos; C. Campos; I. Conceição; Presentation Time: June 20, 2021; 19:30-20:15   
    Our case illustrates a juvenile case of primary autoimmune LEMS. Unlike adults, non-paraneoplastic LEMS prevailed in children but close surveillance for development of cancer for at least two years should be performed.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Clinical; Trial completion date; Trial primary completion date:  Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 (clinicaltrials.gov) -  Feb 14, 2021   
    P2,  N=12, Enrolling by invitation, 
    Trial completion date: Apr 2021 -> Apr 2022 | Trial primary completion date: Dec 2020 -> Dec 2021
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Journal:  Amifampridine to treat Lambert-Eaton myasthenic syndrome. (Pubmed Central) -  Nov 13, 2020   
    The recommended dose is 80 mg a day, divided 3 or 4 times a day. Side effects are usually mild, and the most frequently reported are paresthesia.
  • ||||||||||  Quinamed (amonafide) / Teva, Firdapse (amifampridine) / BioMarin, Catalyst Pharma, metamizole / Generic mfg.
    Biomarker; Journal; Review:  Arylamine N-acetyltransferase acetylation polymorphisms: paradigm for pharmacogenomic-guided therapy- a focused review. (Pubmed Central) -  Oct 23, 2020   
    Studies in human subjects and cryopreserved human hepatocytes show evidence for rapid, intermediate and slow acetylator phenotypes, with further data suggesting genetic heterogeneity within the slow acetylator phenotype. Incorporation of more robust NAT2 genotype/phenotypes relationships, including genetic heterogeneity within the slow acetylator phenotype should lead to further advancements in both health outcomes and cost benefit for prevention and treatment of tuberculosis.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Journal:  Probing the Strength and Mechanism of Binding Between Amifampridine and Calf Thymus DNA. (Pubmed Central) -  Oct 22, 2020   
    Competitive fluorescence experiments using Acridine Orange (AO) and Hoechst 33258 (HO) probes also reveal that amifampridine binds to DNA via an intercalation mode of binding. Finally, the molecular docking studies also suggest that amifampridine tends to bind with the G-C rich region of DNA.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals Announces First Quarter 2020 Financial Results and Provides Corporate Update (GBN) -  May 11, 2020   
    CORAL GABLES, Fla., May 11, 2020 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq:CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today reported financial results for the first quarter ended March 31, 2020 and provided a corporate update.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals Provides Update on Impact of COVID-19 Pandemic on its Business Activities (GBN) -  Apr 8, 2020   
    CORAL GABLES, Fla., April 08, 2020 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Catalyst) (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today provided an update on certain impacts of COVID-19 on its business operations.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals Reports Fourth Quarter and Year-End 2019 Financial Results and Provides Corporate Update (GBN) -  Mar 16, 2020   
    (Catalyst) (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, today provided an update on certain impacts of COVID-19 on its business operations. -Firdapse® Q4 Net Revenues of $30 Million, FY 19 Net Revenues of $102 Million
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Clinical; Enrollment closed; Trial completion date; Trial primary completion date:  Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG (clinicaltrials.gov) -  Feb 1, 2020   
    P3,  N=93, Active, not recruiting, 
    Recruiting -> Active, not recruiting | Trial completion date: Dec 2019 -> Mar 2020 | Trial primary completion date: Oct 2019 -> Jan 2020
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Journal; Review:  Amifampridine tablets for the treatment of Lambert-Eaton myasthenic syndrome. (Pubmed Central) -  Oct 23, 2019   
    Hence, the drug represents a substantial step forward in the symptomatic treatment of the disease due to its efficacy, safety and reliable GMP formulation. As Amifampridine Phosphate works by enhancing the release of acetylcholine at the neuromuscular junction by blocking K+ efflux at the pre-synaptic membrane, it is also conceivable to use it for other diseases of the neuromuscular junction in which such an effect is searched for.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Journal; Review:  Recent Advances and Therapeutic Options in Lambert-Eaton Myasthenic Syndrome. (Pubmed Central) -  Oct 23, 2019   
    Multiple treatment options have been introduced in the past and, recently, a new drug, amifampridine, has been approved by the Food and Drug Administration (FDA) for the treatment of weakness associated with these patients. We summarize this newly introduced drug with a brief description of other treatment options available.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals to Participate in the 2019 Cantor Global Healthcare Conference (GBN) -  Sep 25, 2019   
    (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 that it will participate in the 2019 Cantor Global Healthcare Conference in New York, NY. Patrick J. McEnany, Catalyst’s Chief Executive Officer and Steven Miller, Ph.D., Chief Scientific Officer/Chief Operating Officer will represent the company in a fireside chat and will host one-on-one meetings on Wednesday, October 2nd, 2019.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Journal:  Amifampridine for the treatment of Lambert-Eaton myasthenic syndrome. (Pubmed Central) -  Sep 20, 2019   
    AFPs are also the drug of choice and life-saving for LEMS crisis. For the long-term usage, it is proven to be safe and AFPs can be supplemented with liberal amount of pyridostigmine to sustain a symptomatic improvement without any undue side-reaction.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    3,4-diaminopyridine enhances muscle contraction strength by increasing asynchronous synaptic vesicle fusion to generate multiple muscle action potentials (Neuroscience 2019) -  Sep 2, 2019 - Abstract #284.02/B74; Author: J. B. MACHAMER, K. T. PAGARIGAN, P. M. MCNUTT; Presentation Time: October 21, 2019; 09:00-10:00   
    By inhibiting muscle contraction while maintaining mAPs using the myosin ATPase inhibitor 3-(N-butylethanimidoyl)-4-hydroxy-2H-chromen-2-one (BHC), we find that DAP enhances muscle contraction by generating multiple mAPs. The extra action potentials are not triggered by prolonged EPPs resulting from the synchronous fusion of increased numbers of synaptic vesicles, but instead are a consequence of large numbers of asynchronous vesicle fusions that induce stochastic depolarization of the membrane above the threshold potential required for mAP generation.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals to Participate in the 14th Annual Citi Biotech Conference on September 4th, 2019 (GBN) -  Aug 28, 2019   
    28, 2019 (GLOBE NEWSWIRE) -- 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 that Patrick J. McEnany, Catalyst’s Chief Executive Officer and Steven Miller, Ph.D., Chief Scientific Officer/Chief Operating Officer will host one-on-one meetings with investors at the 14th Annual Citi Biotech Conference on September 4th, 2019 at the Four Seasons Boston, Massachusetts.
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio, Soliris (eculizumab) / AstraZeneca
    Journal:  Diagnosis and treatment of myasthenia gravis. (Pubmed Central) -  Aug 7, 2019   
    CBAs can lead to a significant reduction of seronegative patients, improving myasthenia gravis diagnostic process. New biological drugs offer innovative approaches to treat myasthenic patients with generalized disease, promising to change the paradigm of treatment and to significantly enhance therapeutic success within a precision medicine framework.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals Announces Second Quarter 2019 Financial Results and Provides Corporate Update (GBN) -  Aug 7, 2019   
    New biological drugs offer innovative approaches to treat myasthenic patients with generalized disease, promising to change the paradigm of treatment and to significantly enhance therapeutic success within a precision medicine framework. Firdapse® Launch Momentum Continues with Second Quarter Net Revenues of $28.8 Million
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals Announces Its Support of Spinal Muscular Atrophy Awareness Month (GBN) -  Aug 1, 2019   
    (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.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals to Hold Second Quarter 2019 Financial Results Conference Call and Webcast on Thursday, August 8th, 2019 (GBN) -  Jul 31, 2019   
    (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 that it will release second quarter 2019 financial result after market close on Wednesday, August 7, 2019. Further, Catalyst management will host an investment-community conference call at 8:30 a.m. ET on Thursday, August 8th, 2019 to discuss the financial results and to provide a corporate update.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Journal:  Amifampridine for the Management of Lambert-Eaton Myasthenic Syndrome: A New Take on an Old Drug. (Pubmed Central) -  Jul 20, 2019   
    Relevance to Patient Care and Clinical Practice: With an improved stability profile and decreased dose variability, amifampridine will likely assume the role of first-line management of LEMS. Amifampridine has been shown to improve symptoms of LEMS and is generally well tolerated.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals to Present at the Jefferies 2019 Global Healthcare Conference (GBN) -  Jun 4, 2019   
    Patrick J. McEnany, Catalyst’s Chief Executive Officer and Steven Miller, Ph.D., Chief Scientific Officer/COO, will provide an overview of the Company and its key programs on Thursday, June 6 at 10:30 am ET. The Company’s presentation materials will be available on the “Investors” section of the Company’s website, www.catalystpharma.com following the presentation.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals Announces Expansion of Firdapse License to Include Japan (GBN) -  May 30, 2019   
    (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 that it has amended its license agreement for Firdapse® to expand its commercial territory. The original license was for North America, and has been amended to include Japan.  Upon the achievement of a certain milestone in Japan, Catalyst will have the option to expand the territory further to include most of Asia and Central and South America.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals Announces First Quarter 2019 Financial Results and Provides Corporate Update (GBN) -  May 13, 2019   
    The original license was for North America, and has been amended to include Japan.  Upon the achievement of a certain milestone in Japan, Catalyst will have the option to expand the territory further to include most of Asia and Central and South America. -Firdapse® Launch Off to Strong Start with First Quarter Net Revenues of $12.4 Million
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals to Hold First Quarter Financial Results Conference Call and Webcast on Monday, May 13th, 2019 (GBN) -  May 6, 2019   
    CORAL GABLES, Fla., May 06, 2019 (GLOBE NEWSWIRE) -- 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 that the first quarter result’s press release will be released at 6:03 a.m. (ET) on Monday, May 13th, followed by an investment-community conference call hosted by Catalyst management at 8:30 a.m. (ET) on Monday, May 13th, 2019 to discuss the financial results and to provide a corporate update.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Clinical; Trial completion date; Trial primary completion date:  Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG (clinicaltrials.gov) -  Mar 28, 2019   
    P3,  N=93, Recruiting, 
    Trial completion date: May 2019 -> Sep 2019 | Trial primary completion date: Mar 2019 -> Aug 2019
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals Announces Fourth Quarter and Year-End 2018 Financial Results and Provides Corporate Update (GBN) -  Mar 18, 2019   
    CORAL GABLES, Fla., March 18, 2019 (GLOBE NEWSWIRE) -- 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 reported financial results for the fourth quarter and year-ended December 31, 2018 and provided a corporate update.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals to Hold Fourth Quarter Financial Results and Corporate Update Conference Call and Webcast on Tuesday, March 19th, 2019 (GBN) -  Mar 11, 2019   
    (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 that it will release fourth quarter and year end 2018 financial results after market close on Monday, March 18, 2019. Further, Catalyst management will host an investment-community conference call at 8:30 a.m. ET on Tuesday, March 19, 2019 to discuss the financial results and to provide a corporate update.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Catalyst Pharmaceuticals Announces Support of Rare Disease Day 2019 (GBN) -  Feb 28, 2019   
    (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 Rare Disease Day 2019. Taking place today, Rare Disease Day involves activities worldwide to raise awareness of rare diseases and their impact on patients’ lives among the general public and decision-makers in industry and government.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma
    Clinical; Journal; P3 data:  Amifampridine Phosphate (Firdapse) Is Effective in a Confirmatory Phase 3 Clinical Trial in LEMS. (Pubmed Central) -  Feb 26, 2019   
    Taking place today, Rare Disease Day involves activities worldwide to raise awareness of rare diseases and their impact on patients’ lives among the general public and decision-makers in industry and government. This phase 3 randomized, double-blind, placebo-controlled withdrawal trial in adults with LEMS provided class I evidence of efficacy of amifampridine phosphate as symptomatic treatment in LEMS.