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CORAL GABLES, Fla., Feb. 21, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (“Catalyst” or “Company”) (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 that the peer-reviewed journal Neurology has published Santhera Pharmaceutical's ("Santhera") study titled “Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy” presenting the findings from Santhera's VISION-DMD study conducted by Santhera [1]. Catalyst holds the exclusive rights to commercialize AGAMREE® (vamorolone) in North America. Santhera reports that the VISION-DMD study was a randomized, double-blind, placebo-controlled, and prednisone-controlled clinical trial of 2 doses of vamorolone in participants with Duchenne muscular dystrophy (“DMD”) in the ages four years to younger than seven years at baseline that was conducted at 33 sites in 11 countries from 2018 to 2021. Based on the published study, Santhera has reported that the results of the VISION-DMD study support the long-term efficacy and safety pro vamorolone and conclude that vamorolone was generally well tolerated, consistent with the 24-week study findings, as published previously in JAMA Neurology [2]. As cited in Santhera's recent press release, the Neurology publication of their study states: “Vamorolone is a dissociative corticosteroid that selectively binds to the glucocorticoid receptor and has shown similar efficacy and reduced safety concerns in comparison with prednisone in Duchenne muscular dystrophy (DMD)[2]. This study [VISION-DMD] was conducted to determine the efficacy and safety of vamorolone over 48 weeks and to study crossover participants (prednisone to vamorolone; placebo to vamorolone). A total of 121 participants with DMD were randomized. Vamorolone at a dose of 6 mg/kg/d showed maintenance of improvement for all motor outcomes to week 48 (e.g., for primary outcome, time to stand from supine [TTSTAND] velocity, week 24 least squares mean [LSM] [SE] 0.052 [0.0130] rises/s vs week 48 LSM [SE] 0.0446 [0.0138]). After 48 weeks, vamorolone at a dose of 2 mg/kg/d showed similar improvements as 6 mg/kg/d for North Star Ambulatory Assessment (NSAA) (vamorolone 6 mg/kg/d– vamorolone 2 mg/kg/d LSM [SE] 0.49 [1.14]; 95% CI −1.80 to 2.78, p = 0.67), but less improvement for other motor outcomes. The placebo to vamorolone 6 mg/kg/d group showed rapid improvements after 20 weeks of treatment approaching benefit seen with 48-week 6 mg/kg/d of vamorolone treatment for TTSTAND, time to run/walk 10 m, and NSAA. There was significant improvement in linear growth after crossover in the prednisone to vamorolone 6 mg/kg/d group, and rapid reversal of prednisone-induced decline in bone turnover biomarkers in both crossover groups. There was an increase in BMI after 24 weeks of treatment that then stabilized for both vamorolone groups. Improvements of motor outcomes seen with 6 mg/kg/d of vamorolone at 24 weeks of treatment were maintained to 48 weeks of treatment. Vamorolone at a dose of 6 mg/kg/d showed better maintenance of effect compared with vamorolone at a dose of 2 mg/kg/d for most (3/5) motor outcomes. Bone morbidities of prednisone (stunting of growth and declines in serum bone biomarkers) were reversed when treatment transitioned to vamorolone.” AGAMREE® (vamorolone) oral suspension 40 mg/ml has been approved for commercialization in the United States for the treatment of DMD. About AGAMREE® (vamorolone) AGAMREE's unique mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity. As such, it is considered a novel corticosteroid with dissociative properties in maintaining efficacy that we hope has the potential to demonstrate comparable efficacy to steroid, with the potential for a better-tolerated side effect profile. This mechanism of action may allow vamorolone to emerge as an effective alternative to the current standard of care corticosteroids in children, adolescents, and adult patients with DMD. In the pivotal VISION-DMD study, vamorolone met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting, and vitamin D deficiency. Adverse events were generally of mild to moderate severity. AGAMREE was granted U.S. FDA approval on October 26, 2023, and was granted Orphan Drug and Rare Pediatric Disease designation status for DMD in the U.S., making it eligible for seven years of orphan drug exclusivity upon approval. AGAMREE also has issued and pending patents that could provide protection until 2040. In Europe, it has received Promising Innovative Medicine (PIM) status from the UK MHRA for DMD. References: [1] Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link. [2] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link. [3] Liu X et al. (2020). Proc Natl Acad Sci USA 117:24285-24293 [4] Heier CR et al (2019). Life Science Alliance DOI: 10.26508 [5] Ward et al., WMS 2022, FP.27 - Poster 71. Link. [6] Hasham et al., MDA 2022 Poster presentation. Link. [7] Applicable drug labeling: Summary of Product Characteristics (SmPC). English. German. About Duchenne muscular dystrophy (DMD) DMD, the most common form of muscular dystrophy, is a rare and life-threatening neuromuscular disorder characterized by progressive muscle dysfunction, ultimately leading to loss of ambulation, respiratory failure, and fatality. Current standard treatment for DMD involves corticosteroids, which often come with significant side effects. It is estimated that between 11,000 and 13,000 patients in the U.S. are affected by DMD, with approximately 70% of patients currently receiving concomitant corticosteroid treatment. About Catalyst Pharmaceuticals 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. 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 for commercialization in the U.S. on October 26, 2023. For more information about Catalyst Pharmaceuticals, Inc., please visit the Company's website at  . For Full Prescribing and Safety Information for FIRDAPSE®, please visit . For Full Prescribing Information, including Boxed WARNING for FYCOMPA®, please visit . For Full Prescribing Information for AGAMREE®, please visit . Forward-Looking Statements This press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. These include statements regarding Catalyst’s expectations, beliefs, plans, or objectives regarding the intended use of net proceeds therefrom. 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 AGAMREE will be found to have a better safety pro other corticosteroids, (ii) whether Catalyst's commercial launch of AGAMREE in the United States will be successful, and (iii) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2022 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. Investor Contact Mary Coleman, Catalyst Pharmaceuticals, Inc. (305) 420-3200 mcoleman@catalystpharma.com Media Contact David Schull, Russo Partners (858) 717-2310 david.schull@russopartnersllc.com

Results from 112 patients with DMD who completed the study confirm maintenance of efficacy and benefits in safety and tolerability of treatment with vamorolone over 48 weeks AGAMREE® has shown safety benefits in patients switching from standard of care corticosteroids in terms of recovery of bone health and growth More than 200 patients have now been treated with vamorolone for up to 84 months across clinical studies and access programs AGAMREE® is the only approved medication in the European Union (EU) for treating all patients from age 4 years with DMD [1], and the first DMD treatment approved across the U.S., EU and UK February 14, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces the publication of the paper “Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy” in the peer-reviewed journal Neurology [2]. The publication reports the results of the 48-week treatment with vamorolone in patients with DMD in the VISION-DMD study, supporting the long-term efficacy and safety profile of vamorolone and concluding that vamorolone was generally well tolerated, consistent with the 24-week study findings, as published previously in JAMA Neurology [3]. The Neurology publication states: “Vamorolone is a dissociative corticosteroid that selectively binds to the glucocorticoid receptor and has shown similar efficacy and reduced safety concerns in comparison with prednisone in Duchenne muscular dystrophy (DMD) [3]. This study [VISION-DMD] was conducted to determine the efficacy and safety of vamorolone over 48 weeks and to study crossover participants (prednisone to vamorolone; placebo to vamorolone). A total of 121 participants with DMD were randomized. Vamorolone at a dose of 6 mg/kg/d showed maintenance of improvement for all motor outcomes to week 48 (e.g., for primary outcome, time to stand from supine [TTSTAND] velocity, week 24 least squares mean [LSM] [SE] 0.052 [0.0130] rises/s vs week 48 LSM [SE] 0.0446 [0.0138]). After 48 weeks, vamorolone at a dose of 2 mg/kg/d showed similar improvements as 6 mg/kg/d for North Star Ambulatory Assessment (NSAA) (vamorolone 6 mg/kg/d–vamorolone 2 mg/kg/d LSM [SE] 0.49 [1.14]; 95% CI −1.80 to 2.78, p = 0.67), but less improvement for other motor outcomes. The placebo to vamorolone 6 mg/kg/d group showed rapid improvements after 20 weeks of treatment approaching benefit seen with 48-week 6 mg/kg/d of vamorolone treatment for TTSTAND, time to run/walk 10 m, and NSAA. There was significant improvement in linear growth after crossover in the prednisone to vamorolone 6 mg/kg/d group, and rapid reversal of prednisone-induced decline in bone turnover biomarkers in both crossover groups. There was an increase in BMI after 24 weeks of treatment that then stabilized for both vamorolone groups. Improvements of motor outcomes seen with 6 mg/kg/d of vamorolone at 24 weeks of treatment were maintained to 48 weeks of treatment. Vamorolone at a dose of 6 mg/kg/d showed better maintenance of effect compared with vamorolone at a dose of 2 mg/kg/d for most (3/5) motor outcomes. Bone morbidities of prednisone (stunting of growth and declines in serum bone biomarkers) were reversed when treatment transitioned to vamorolone.” AGAMREE® is the first and only medicinal product for DMD to have received full approval in the EU and, following approval in the U.S. last October and in the UK in January, it is the first authorized treatment for patients with the disease in all three territories. Vamorolone is a novel drug with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity and is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes that may be responsible for local tissue amplification and corticosteroid-associated toxicity in local tissues [3-5]. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore vamorolone is positioned as an alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD [3-5]. In the pivotal VISION-DMD study, vamorolone met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile [4]. The most commonly reported side effects were cushingoid features, vomiting, weight increase and irritability. Side effects were generally of mild to moderate severity. Currently available data show that vamorolone, unlike corticosteroids, has no restriction of growth [6] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [7]. AGAMREE (vamorolone), an orphan medicinal product, is approved for use in the United States (Prescribing Information), the European Union (Summary of Product Characteristics) and the United Kingdom. References: [1] Applicable drug labeling: Summary of Product Characteristics (SmPC). English. German. [2] Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link. [3] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link. [4] Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293 [5] Heier CR et al (2019). Life Science Alliance DOI: 10.26508 [6] Ward et al., WMS 2022, FP.27 - Poster 71. Link. [7] Hasham et al., MDA 2022 Poster presentation. Link. Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD. Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with high unmet medical need. The Company has an exclusive license from ReveraGen for all indications worldwide to AGAMREE® (vamorolone), a dissociative steroid with novel mode of action, which was investigated in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. AGAMREE for the treatment of DMD is approved in the U.S. by the Food and Drug Administration (FDA), in the EU by the European Medicines Agency (EMA), and in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA). Santhera has out-licensed rights to vamorolone for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics. The clinical stage pipeline also includes lonodelestat to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases. For further information, please visit www.santhera.com. The content above comes from the network. if any infringement, please contact us to modify.

A Takeda Pharmaceutical drug for an allergic condition affecting the esophagus is now approved, a regulatory decision that comes a little more than two years after the FDA initially turned down the pharma giant’s application. The regulatory decision gives Takeda the opportunity to offer patients a drug with different dosing than a blockbuster product that Sanofi markets for the condition. The FDA approval announced Monday covers the treatment of pediatric and adult patients who have eosinophilic esophagitis, a condition in which eosinophils, a type of white blood cell, build up in the esophagus, causing inflammation and swallowing difficulty. Consequently, food often gets stuck in the esophagus, leading to emergency room visits. Eosinophilic esophagitis has been treated with corticosteroids used off label. Takeda’s new drug, brand name Eohilia, is also a corticosteroid, a twice-daily oral suspension formulation of an old drug called budesonide. This anti-inflammatory drug reduces swelling in the airways, which led to initial approval of inhaled formulations of the molecule for the prevention of asthma attacks. The drug, which works by binding to glucocorticoid receptors, later found additional uses in other diseases. The exact way this mechanism treats eosinophilic esophagitis is not known, but inflammation is key part of the chronic condition’s progression. Eohilia is one of the drugs that came to Takeda via the $62 billion acquisition of Shire in 2019. The Japanese pharma giant continued late-stage clinical development of the drug, then submitted a new drug application in eosinophilic esophagitis in 2020. The following year, the FDA turned down Takeda’s application. According to the company, the regulator recommended another clinical study. Rather than do that, Takeda opted to stop further development. This past September, Takeda revealed that the FDA accepted the company’s resubmission for oral budesonide. Takeda did not conduct another clinical trial. Instead, the pharma giant said it reanalyzed the clinical trial data. Discussions with the FDA led to the resubmission of the drug as a treatment for short-term treatment of eosinophilic esophagitis. “For most of us, eating is a simple experience. But for people living with eosinophilic esophagitis, sitting down for a meal can include painful and difficult swallowing, chest pain and a choking sensation,” Brandon Monk, senior vice president and head, U.S. Gastroenterology Business Unit, Takeda, said in a prepared statement. “With Eohilia, patients and their physicians now have the first and only FDA-approved oral treatment option for [eosinophilic esophagitis] that was shown during two 12-week clinical studies to reduce esophageal inflammation and improve the ability to swallow.” The prescribing information recommends treatment with the drug for no longer than 12 weeks. The label includes warnings of a higher risk of developing infections, which is consistent with the risks for other corticosteroids. The prescribing information also cautions that treatment can lead to systemic effects such as too much or too little production of the adrenal hormone cortisol, another known complication of steroid drugs. The first FDA-approved drug for eosinophilic esophagitis was Dupixent, an antibody drug from partners Sanofi and Regeneron Pharmaceuticals that has regulatory approvals for treating multiple autoimmune conditions. The 2022 approval of Dupixent in eosinophilic esophagitis covered the treatment of adults as well as children age 12 or older. Approval in this indication has since expanded to children as young as age 1. Dupixent is administered as a once-weekly injection. Unlike Eohilia, Dupixent’s prescribing information does not recommend limiting the duration of treatment, which could be a competitive advantage given that eosinophilic esophagitis is a chronic condition. Takeda had previously said it would record an impairment charge due to the discontinuation of its eosinophilic esophagitis drug, formerly known ats TAK-721. The company now says it is assessing the financial impacts of Eohilia’s FDA approval, including a reversal of the impairment loss for the fiscal year ending March 31, 2024. The company added that it does not anticipate this impact will be material.