Neurosyphilis

Duchenne muscular dystrophy is a rare, debilitating and life-shortening neuromuscular disease1 Upon approval, vamorolone would be the first and only treatment option indicated for patients diagnosed with Duchenne muscular dystrophy in Canada The Canadian regulatory submission of vamorolone follows approvals in the US in 2023, and the European Union in early 2024 This New Drug Submission reflects Kye Pharmaceuticals' commitment to advancing treatments for rare diseases and serving the unmet needs of Canadian patients MISSISSAUGA, ON, April 8, 2025 /PRNewswire/ - Kye Pharmaceuticals, Inc. ("Kye") announced today it has submitted a New Drug Submission (NDS) to Health Canada for the regulatory review and approval of AGAMREE® (vamorolone). If approved, AGAMREE® would be the first and only therapy approved by Health Canada with an indication for the treatment of Duchenne muscular dystrophy (DMD). Health Canada has granted AGAMREE® a priority review and marketing authorization could be granted before the end of 2025. News of the AGAMREE® priority review was welcomed by national patient organizations. "Defeat Duchenne Canada is thrilled that Health Canada has accepted AGAMREE® for priority review. There are currently no approved treatments available in Canada for young people living with Duchenne muscular dystrophy. We urge Health Canada to approve AGAMREE® to ensure all Canadians for whom this treatment would be beneficial, can access it as soon as possible." Lisa McCoy, Chief Executive Officer, Defeat Duchenne. "Muscular Dystrophy Canada (MDC) is pleased that Health Canada has granted Priority Review for AGAMREE® (vamorolone). While corticosteroids have been a key treatment for decades, they are not currently approved for DMD, and their significant side effects have been a challenge for the Duchenne muscular dystrophy community. If approved, this promising treatment will provide options for the Duchenne muscular dystrophy community." Stacey Lintern, Chief Executive Officer, MDC. The Canadian Neuromuscular Disease registry estimates that there are more than 800 boys and young men living with DMD in Canada and coping with its debilitating symptoms.2 Duchenne muscular dystrophy (DMD) is a type of muscular dystrophy that causes weakness and a wasting of the muscle due to the absence of a protein called dystrophin. If the body is lacking dystrophin, muscle cells become damaged and movements that are considered natural such as getting up from the floor and walking become challenging. Boys with DMD lose the ability to walk in early teens and have cardiac and respiratory difficulties. Bone thinning and scoliosis are also common for those with Duchenne.1 "Health Canada granting vamorolone a priority review is a promising first step for patients with Duchenne having access to approved medicines in Canada," said Dr. Jean Mah, Director of the Pediatric Neuromuscular Program at the Alberta Children's Hospital in Calgary. "If approved, vamorolone will provide Duchenne families access to an approved therapy that will positively impact their disease. We would like to thank the patients and families from DMD communities across Canada who have contributed to advancing vamorolone research." This initial step in the review process is a culmination of years of research, including clinical trials of vamorolone conducted in Canada.3 Kye commends the courage of patients and families who participated in these studies, including five clinical trial sites in Canada, generating the data to support the approval of this novel treatment in countries around the world. "This submission is an important milestone for DMD treatments in Canada and we are grateful for our close partnership with Catalyst Pharmaceuticals," stated John McKendry, President and CEO at Kye Pharmaceuticals. "We look forward to working collaboratively with the DMD community and Health Canada throughout the priority review process." 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 has the potential to demonstrate comparable efficacy to steroids, with the potential for a better-tolerated side effect profile. This mechanism of action may allow AGAMREE® 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, AGAMREE® 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, psychiatric disorders, vomiting, weight increases, and vitamin D deficiency. Adverse events were generally of mild to moderate severity.4 About Kye Pharmaceuticals Kye Pharmaceuticals is a growth-stage Canadian specialty pharmaceutical company committed to bringing value to Canadians by identifying, licensing, and commercializing novel prescription medicines that may not otherwise be available to patients across Canada. With a growing pipeline of novel medicines, Kye's portfolio spans a range of therapeutic areas, including cardiology, psychiatry, pediatrics, rare diseases, hematology, and neurology. Kye Pharmaceuticals is a private company headquartered in Toronto focused on bringing medications to the Canadian market which fulfill clinically significant unmet needs. Kye is committed to licensing and launching medicines that matter by delivering better outcomes to our partners, Canadian healthcare professionals, and, most importantly, patients across Canada. For more information about the company, its management, portfolio and pipeline, please visit About Catalyst Pharmaceuticals Catalyst Pharmaceuticals, Inc. (Nasdaq: CPRX) is a biopharmaceutical company committed to improving the lives of patients with rare diseases. With a proven track record of bringing life-changing treatments to the market, Catalyst is focused on in-licensing, commercializing, and developing innovative therapies. Guided by its deep commitment to patient care, Catalyst prioritizes accessibility, ensuring patients receive the care they need through a comprehensive suite of support services designed to provide seamless access and ongoing assistance. Catalyst maintains a well-established U.S. presence while actively seeking to expand its global commercial footprint through strategic partnerships. Catalyst, headquartered in Coral Gables, Fla., was recognized on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies and on the 2024 Deloitte Technology Fast 500™ list as one of North America's fastest-growing companies. For more information, please visit Catalyst's website at . References: National Institute of Neurological Disorders and Stroke. Muscular dystrophy: hope through research. Available at [last accessed March 2021] Hodgkinson et al. The Canadian Neuromuscular Disease Registry 2010–2019, Journal of Neuromuscular Diseases 8 (2021) 53–6 Worsfold, N. (2020, June 10). Defeatduchenne.ca. Available at Defeat Duchenne Canada: $1M Grant Towards Clinical Trial – Defeat Duchenne Canada Dang UJ et al. (2024) Neurology 024;102:e208112. doi.org/10.1212/WNL.0000000000208112 SOURCE Kye Pharmaceuticals Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

CORAL GABLES, Fla., April 08, 2025 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (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 reported that its sub-licensee in Canada, Kye Pharmaceuticals, Inc. (Kye), has announced that Health Canada has accepted the New Drug Submission (NDS) for AGAMREE®, a novel corticosteroid treatment for Duchenne muscular dystrophy (DMD), for review. The submission has been granted Priority Review, expediting the regulatory process with the potential for marketing authorization before the end of 2025. If approved, AGAMREE would be the first and only treatment option indicated for patients diagnosed with DMD in Canada. “We are pleased that Health Canada has accepted the New Drug Submission for AGAMREE with a Priority Review, recognizing its potential to address a significant unmet need for patients with Duchenne muscular dystrophy in Canada,” stated Richard J. Daly, President and CEO of Catalyst. “In collaboration with our Canadian sub-licensee, Kye, we remain committed to bringing this innovative therapy to DMD patients and ensuring access, particularly for those in underserved communities.” AGAMREE (40 mg/mL oral suspension) received U.S. Food and Drug Administration (FDA) approval on October 26, 2023, for the treatment of DMD in patients aged two years and older and became commercially available in the U.S. on March 13, 2024. Kye currently markets FIRDAPSE®, Catalyst’s flagship product for the treatment of Lambert-Eaton myasthenic syndrome (LEMS), in Canada. LEMS is a rare neuromuscular disorder characterized by debilitating and progressive muscle weakness and fatigue. About Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects males, with symptoms typically appearing in early childhood, around ages three to five. DMD is caused by mutations in the gene that encodes dystrophin, a protein that plays a crucial role in maintaining the structure and function of muscle fibers. Without dystrophin, muscle cells become fragile and easily damaged, leading to progressive muscle degeneration. Symptoms of DMD usually begin with difficulty in walking, frequent falls, and muscle weakness, particularly in the legs and pelvis. As the disease progresses, individuals may experience difficulty standing, climbing stairs, and eventually, complete loss of mobility. Other complications can include respiratory and cardiac issues due to muscle weakness. 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 designed to achieve dissociative properties while maintaining efficacy that has the potential to demonstrate comparable efficacy to steroids, with the potential for a better-tolerated side effect profile. This mechanism of action may allow AGAMREE 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, AGAMREE 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, psychiatric disorders, vomiting, weight increases, and vitamin D deficiency. Adverse events were generally of mild to moderate severity. About Kye Pharmaceuticals Kye Pharmaceuticals is a growth-stage Canadian specialty pharmaceutical company committed to bringing value to Canadians by identifying, licensing, and commercializing novel prescription medicines that may not otherwise be available to patients across Canada. With a growing pipeline of novel medicines, Kye's portfolio spans a range of therapeutic areas, including cardiology, psychiatry, pediatrics, rare diseases, hematology, and neurology. Kye Pharmaceuticals is a private company headquartered in Toronto focused on bringing medications to the Canadian market which fulfill clinically significant unmet needs. Kye is committed to licensing and launching medicines that matter by delivering better outcomes to our partners, Canadian healthcare professionals, and, most importantly, patients across Canada. For more information about the company, its management, portfolio and pipeline, please visit www.kyepharma.com About Catalyst Pharmaceuticals Catalyst Pharmaceuticals, Inc. (Nasdaq: CPRX) is a biopharmaceutical company committed to improving the lives of patients with rare diseases. With a proven track record of bringing life-changing treatments to the market, we focus on in-licensing, commercializing, and developing innovative therapies. Guided by our deep commitment to patient care, we prioritize accessibility, ensuring patients receive the care they need through a comprehensive suite of support services designed to provide seamless access and ongoing assistance. Catalyst maintains a well-established U.S. presence while actively seeking to expand its global commercial footprint through strategic partnerships. Catalyst, headquartered in Coral Gables, Fla., was recognized on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies and on the 2024 Deloitte Technology Fast 500™ list as one of North America’s Fastest-Growing Companies. For more information, please visit Catalyst's website at www.catalystpharma.com. Forward-Looking Statements This 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 (i) whether Health Canada will approve AGAMREE either in the anticipated timeline or at all, (ii) if approved, whether Kye can successfully commercialize AGAMREE in Canada, and (iii) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2024 and its subsequent 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. Source: Catalyst Pharmaceuticals, Inc.

– New trontinemab data continue to support rapid and deep, dose-dependent reduction of amyloid plaques in Phase Ib/IIa Brainshuttle™ AD study – – Data on the Elecsys ® pTau181 plasma test demonstrate potential to accurately rule out amyloid pathology, one of the hallmarks of Alzheimer’s disease – – A Phase III program for trontinemab later this year will be initiated based on totality of data – SOUTH SAN FRANCISCO, CA, USA I April 03, 2025 I Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that new data were presented at the AD/PD 2025 International Conference on Alzheimer’s and Parkinson’s Diseases in Vienna, Austria. Highlights included presentations from the ongoing trontinemab Phase Ib/IIa Brainshuttle™ AD study demonstrating dose-dependent rapid amyloid depletion from the brain and the potential of the Elecsys ® pTau181 plasma test to rule out amyloid pathology. A Phase III program for trontinemab will be initiated later this year. “We are pleased with the progress across our Alzheimer’s portfolio as we move ahead with a Phase III trontinemab program and continue to expand our diagnostic solutions,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “Well over 55 million people worldwide are living with dementia, of which around 70% have Alzheimer’s. Moreover, up to three-quarters of people experiencing symptoms of Alzheimer’s remain undiagnosed. This growing population needs more accurate, less invasive diagnostic approaches paired with effective disease-modifying treatments to slow neurodegeneration as early as possible.” Trontinemab Preliminary results for trontinemab from 114 participants in the 1.8 or 3.6 mg/kg double-blind period suggest rapid and deep, dose-dependent reduction of amyloid plaques in the brain as measured by amyloid positron emission tomography (PET). Trontinemab reduced amyloid levels below the 24 centiloid threshold in 81% of participants (n=21/26) in the 3.6 mg/kg dose group after 28 weeks. Based on data in the field, both the speed of amyloid lowering, and the ability to lower below the amyloid positivity threshold early on, are important to achieve clinically meaningful benefit in early Alzheimer’s disease. These data were reinforced by early and significant reductions in fluid biomarkers of Alzheimer’s disease including total tau, phosphorylated Tau (pTau)181, pTau217 and neurogranin measured in cerebrospinal fluid (CSF) and plasma. Trontinemab continues to show a favorable safety and tolerability profile. Amyloid-related imaging abnormalities-edema/effusion (ARIA-E) were observed in <5% (n=3/114) of participants (blinded data), which were radiographically mild, and there was one case associated with mild symptoms. Trontinemab is currently being studied in the Phase Ib/IIa Brainshuttle AD study assessing the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of trontinemab in participants with Alzheimer’s disease. Diagnostics Results from 604 participants in a multicenter study of Roche’s Elecsys pTau181 plasma test were presented, demonstrating its potential to accurately rule out amyloid pathology, a hallmark of Alzheimer’s disease, in people with cognitive impairment. The Elecsys pTau181 test is a minimally invasive blood test that measures pTau181 protein in plasma. By ruling out those without signs of amyloid pathology, the test can help to avoid unnecessary testing using CSF or PET, which are more invasive and often come with long waiting times and high costs. This can result in further delays to diagnosis and cost to healthcare systems. Roche anticipates tests being available in Europe by late 2025, with the U.S. following after. Other activities at the AD/PD 2025 International Conference Parkinson’s disease Results from the Phase IIb PADOVA study were presented for the first time, investigating prasinezumab in 586 people with early-stage Parkinson’s disease while on stable symptomatic treatment. Although the study missed its primary endpoint, the totality of data suggest possible benefit in early-stage Parkinson’s disease. Prasinezumab showed potential clinical efficacy in the primary endpoint of time to confirmed motor progression with a hazard ratio (HR)=0.84 [0.69-1.01] and p=0.0657, missing statistical significance. In a pre-specified analysis, the effect of prasinezumab was more pronounced in the population treated with levodopa (75% of participants), HR=0.79 [0.63-0.99]. Consistent positive trends across multiple secondary and exploratory clinical endpoints were also observed, in addition to a potential biological effect of prasinezumab on MRI biomarkers. Prasinezumab continues to be well tolerated and no new safety signals were observed in the study. Given the high level of unmet need for these patients, Roche is further evaluating study data to determine next steps. About trontinemab Trontinemab is an investigational Brainshuttle™ bispecific #2+1 amyloid-beta antibody specifically engineered for enhanced access to the brain to enable rapid reduction of amyloid in people with Alzheimer’s disease. Trontinemab is designed for the efficient transport across the blood-brain barrier to target aggregated forms of amyloid beta and remove amyloid plaques in the brain. The uniqueness of trontinemab is based on Roche’s proprietary Brainshuttle technology combining an amyloid beta-binding monoclonal antibody with a transferring receptor (TfR1) shuttle module. As a result, high central nervous system (CNS) exposure of trontinemab may be achieved at low doses, leading to a rapid and deep amyloid clearance. Due to its unique properties, trontinemab might unlock the full potential of disease-modifying monoclonal antibodies by effectively penetrating the brain and potentially leading to slowing of disease progression. About prasinezumab Prasinezumab is an investigational monoclonal antibody designed to selectively bind aggregated alpha-synuclein (α-syn) and reduce neuronal toxicity. By targeting the build-up of α-syn protein in the brain, prasinezumab can potentially prevent further accumulation and spreading between cells, thereby slowing down the progression of the disease. The evidence supporting targeting α-syn aggregates as a mechanism of action in Parkinson’s disease is based on a wide range of scientific evidence in the field. Prasinezumab is currently being assessed in ongoing open-label extensions of the Phase II PASADENA and Phase IIb PADOVA studies. The safety database for prasinezumab consists of data from more than 900 Parkinson’s disease study participants that have been treated with the investigational medicine, including more than 500 who were treated over 1.5-5 years. Roche entered into a Licensing, Development, and Commercialization agreement with Prothena in December 2013 to develop and commercialize monoclonal antibodies targeting α-syn, such as prasinezumab, for the treatment of Parkinson’s disease. About Genentech in Neuroscience Neuroscience is a major focus of research and development at Genentech. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases. Genentech and Roche are investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today. About Genentech Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com . SOURCE: Genentech