Vitamin D Deficiency

- Data from the largest retrospective analysis of ENPP1 Deficiency provides insights into the evolution of the disease’s serious cardiovascular and musculoskeletal complications - - Findings highlight the urgent need for early and improved diagnosis, care and treatments that address the long-term systemic effects of ENPP1 Deficiency - BOSTON, April 10, 2025 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage biopharmaceutical company developing innovative therapeutics for rare diseases that affect bone health and blood vessel function, today announced the publication of a paper titled, “Phenotypic characterization of ENPP1 deficiency: generalized arterial calcification of infancy and autosomal recessive hypophosphatemic rickets type 2” in JBMR Plus that characterizes the severity and progression of ENPP1 Deficiency. Inozyme collaborated with leading disease experts Carlos Ferreira, M.D., of the National Institutes of Health (NIH), Frank Rutsch, M.D., of Münster University Children’s Hospital and other global contributors to collect natural history data in the largest retrospective analysis of ENPP1 Deficiency published to date. “This comprehensive study illustrates the devastating, systemic nature of ENPP1 Deficiency, highlighting its severe cardiovascular implications starting as early as infancy and evolving to significant musculoskeletal complications over a lifetime as individuals go through childhood, adolescence, and then adulthood,” said Matt Winton, Ph.D., Senior Vice President and Chief Operating Officer of Inozyme Pharma. “The findings underscore a critical need for earlier diagnosis and effective treatments. Our lead investigational therapy, INZ-701, is uniquely positioned as a potential transformative therapy to address the underlying causes and systemic impacts of this severe condition.” Detailed Analysis Reveals Severity and Disease Progression ENPP1 Deficiency frequently manifests as Generalized Arterial Calcification of Infancy (GACI) or Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2), representing age-dependent phenotypes that evolve on a continuum over a lifetime. Of the 84 individuals with ENPP1 Deficiency in the analysis, 51 had a recorded diagnosis of GACI, only 19 of whom survived beyond infancy; 22 were diagnosed with GACI and progressed to ARHR2; and 11 presented initially with ARHR2. Importantly, a majority of patients (60%) with a history of GACI had prenatal findings, and a GACI diagnosis (median age of 0.8 months at diagnosis) was usually associated with early-onset arterial calcification, respiratory distress, heart failure, and hypertension, necessitating acute inpatient care. Additional findings from the publication included: By age 55, over 95% of patients with ENPP1 Deficiency will have had cardiovascular, musculoskeletal, and other organ complications.Vascular calcification and cardiovascular complications onset predominately in infancy – 60% of patients had arterial or aortic calcification within the first 3 months of life. Notably, cardiovascular complications were evident in patients without a diagnosis of GACI – 64% of the group diagnosed with ARHR2-only had cardiovascular manifestations.By age 10, approximately 70% of patients developed serious musculoskeletal complications, primarily rickets significantly impairing quality of life. Additional complications associated with ARHR2 include hearing impairment and ongoing risk of cardiovascular problems. These findings highlight that ENPP1 Deficiency is a progressive, lifelong condition requiring coordinated, multidisciplinary care. Even for those who survive infancy, the majority will face ongoing cardiovascular, skeletal, and systemic complications, underscoring the need for early diagnosis and long-term management to improve outcomes. About ENPP1 Deficiency ENPP1 Deficiency is a serious and progressive rare disease that affects blood vessels, soft tissues, and bones. Individuals who present in utero or in infancy are typically diagnosed with generalized arterial calcification of infancy (GACI Type 1), with about 50% of these infants not surviving beyond six months. Children with this condition typically develop autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), while adolescents and adults may develop osteomalacia, or softened bones. ARHR2 and osteomalacia cause pain and difficulty with movement. Additionally, patients may experience hearing loss, calcification in arteries and joints, and heart problems. ENPP1 Deficiency is an autosomal recessive disease and biallelic mutations are estimated to occur in approximately 1 in 64,000 pregnancies worldwide. Many individuals with just one copy of the mutated gene (monoallelic ENPP1 Deficiency) exhibit severe symptoms, suggesting that the worldwide prevalence of ENPP1 Deficiency may be much higher than current estimates. Currently, there are no approved therapies for ENPP1 Deficiency. About Inozyme Pharma Inozyme Pharma is a clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics that target the PPi-Adenosine Pathway, a key regulator of bone health and blood vessel function. Disruptions in this pathway underlie a range of severe diseases, including ENPP1 Deficiency. Our lead investigational therapy, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) designed to restore PPi and adenosine levels. INZ-701 is currently in late-stage clinical development in ENPP1 Deficiency, with the potential to expand into additional indications where deficiencies in the PPi-Adenosine Pathway contribute to disease pathology. Through our pioneering work, we aim to transform treatment options for patients affected by these devastating conditions. For more information, please visit https://www.inozyme.com/ or follow Inozyme on LinkedIn, X, and Facebook. Contacts Investors:Inozyme PharmaStefan Riley, Senior Director of IR and Corporate Communications(617) 461-2442stefan.riley@inozyme.com Media:Biongage CommunicationsTodd Cooper(617) 840-1637todd@biongage.com A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/eb5daae6-15b5-4d55-9f50-d2e8d88a974d

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.