Rickets

精彩内容 米内网最新数据显示，2024上半年中国城市实体药店终端西药（化学药+生物药）的销售规模继续保持正增长，达898亿元，8个大类呈现正增长。177个西药品牌卖过亿，青岛双鲸药业的维生素D滴剂重回TOP1宝座，“流感神药”宜昌东阳光长江药业的磷酸奥司他韦颗粒则大跌52%；10个过亿品牌增长率超过100%，正大天晴南京顺欣制药的利妥昔单抗注射液暴涨6428%。从集团来看，跨国药企实力依然强劲，国内药企正大制药、华润三九、恒瑞医药、石药控股成功挤进TOP15榜单。 图1：近几年中国城市实体药店终端西药（化学药+生物药）的销售情况（万元） 在中国城市实体药店终端，西药的销售规模一直保持正增长态势，2023年达到峰值1800亿元，2024上半年上涨1.81%，达898亿元。 2024上半年有8个大类销售额呈现正增长，其中抗肿瘤和免疫调节剂占比在22.04%，销售额增长了16.11%；呼吸系统用药占比在6.44%，销售额增长了10.21%；全身用激素类制剂(不含性激素和胰岛素)占比在0.37%，销售额增长了19.55%。6个销售额下滑的大类中，消化系统及代谢药的占比在22.94%，销售额下滑超过8%。 177个西药合揽超420亿！国产品牌夺冠，“流感神药”折戟 据统计，2024上半年销售额超过1亿元的西药品牌有177个（同一产品名涉及同一集团多家生产企业的，暂不合并统计，下同），涉及12个大类（38个亚类）。从亚类来看，抗肿瘤药以42个过亿品牌遥遥领先，糖尿病用药、矿物质补充剂、高血压用药各占12个。 表1：2024上半年销售额在5亿元以上的西药品牌 2024上半年在中国城市实体药店终端，177个销售额超过1亿元的西药品牌合揽超过420亿市场，超10亿品牌有2个，均为非独家。 青岛双鲸药业的维生素D滴剂用于预防维生素D缺乏性佝偻病，预防骨质疏松症等。在中国城市实体药店终端，该品牌在2019年及2021年占领化学药（含生物药）市场“销冠”宝座，2022-2023年的销售额保持在21亿元以上，2024上半年再次“封王”。 在化学药（含生物药）维生素类市场，青岛双鲸药业的维生素D滴剂市场份额在2015年仅占6.03%，2018年起上涨至20%以上，2024上半年为22.83%，是该类药物市场中唯一一个市场份额超过20%的品牌。 图2：康方药业的卡度尼利单抗注射液销售情况（单位：万元） 卡度尼利单抗注射液是康方药业在2022年获批的国产1类新药，是全球首创PD-1/CTLA-4双特异性抗体。在中国城市实体药店终端，该品牌在2023年突破10亿元大关，2024上半年增长了35.29%，销售额超过7亿元。在化学药（含生物药）抗肿瘤药市场，康方药业的卡度尼利单抗注射液已成为了最畅销的国产品牌。 图3：翰森制药的甲磺酸阿美替尼片销售情况（单位：万元） 甲磺酸阿美替尼片是翰森制药在2020年获批的国产1类新药，是第三代EGFR-TKI。在中国城市实体药店终端，该品牌在2023年突破11亿元，2024上半年增长了47.58%，销售额为6.7亿元。在化学药（含生物药）抗肿瘤药市场，翰森制药的甲磺酸阿美替尼片已成为了最畅销的内服品牌。 图4：宜昌东阳光长江药业的磷酸奥司他韦颗粒销售情况（单位：万元） 近几年在新冠及流感频繁刺激下，奥司他韦的销售可谓跌宕起伏，宜昌东阳光长江药业的磷酸奥司他韦颗粒作为该品种的龙头品牌，受影响程度较为严重。 在中国城市实体药店终端，该品牌在2016-2019年经历了疯狂暴涨时期，2019年销售额达到了8亿元以上，2020-2021年进入急速下滑期，连续暴跌了16.89%、58.47%，2022-2023年恢复强势增长，分别增长了240.45%、159.48%，2023年销售额达到峰值24.7亿元，2024上半年猛降52.24%。 2024上半年在化学药（含生物药）市场，宜昌东阳光长江药业的磷酸奥司他韦颗粒的品牌排名跌至TOP9，但依然是唯一一个销售额在5亿元以上的全身用抗病毒药，该品牌能否在今年下半年力挽狂澜，我们拭目以待。 98个品牌飞速增长！正大天晴暴涨6428%，国产新药发威 2024上半年销售额超过1亿元的西药品牌中98个品牌有正增长，最高涨幅达6428.27%，9个品牌增长率达三位数。值得注意的是，这98个销售额飞速增长的西药品牌中，有18个为国产新药。 表2：2024上半年销售额过亿且增长率在60%以上的西药品牌图5：正大天晴南京顺欣制药两款注射剂的销售情况（单位：万元） 正大天晴南京顺欣制药的利妥昔单抗注射液和贝伐珠单抗注射液均是2023年获批的生物药3.3类新药，两大品牌获批当年在中国城市实体药店终端的销售额分别为5000万元、1.1亿元水平。2024上半年，正大天晴南京顺欣制药的利妥昔单抗注射液暴涨6428.27%，销售额突破1亿元，公司的贝伐珠单抗注射液也飙涨了731.69%，销售额已超过去年全年。 上述两大产品均为中国城市实体药店终端（化+生）抗肿瘤药重磅注射剂，目前正大天晴南京顺欣制药已成为了利妥昔单抗注射液的领军企业、成为了和贝伐珠单抗注射液的TOP2企业。 图6：石药欧意药业的甲钴胺片销售情况（单位：万元） 在中国城市实体药店终端，石药欧意药业的甲钴胺片一直保持高速增长态势，从2015年的600万元一路飞涨至2023年的1.6亿元，2024上半年继续保持60%以上的增长，销售额超过了1亿元。 甲钴胺片是中国城市实体药店终端抗贫血制剂TOP2产品，石药欧意药业已成为该产品的龙头企业。 表3：2024上半年销售额过亿且正增长的国产新药18个国产新药2024上半年在中国城市实体药店终端销售额超过1亿元且有正增长：创新药龙头恒瑞医药3款1类新药上榜，其中阿得贝利单抗注射液大涨214.64%；翰森制药两款1类新药均有双位数增长，潜力不容小觑；上海艾力斯医药的甲磺酸伏美替尼片、沈阳三生制药的重组人血小板生成素注射液增速均超过80%，贝达药业的盐酸恩沙替尼胶囊增长了73.38%。 最新TOP15集团出炉！正大制药、华润三九、石药畅销产品霸屏 2024上半年在中国城市实体药店终端，177个销售额超过1亿元的西药品牌归属80多个集团，跨国巨头阿斯利康、拜耳、强生、辉瑞、诺华牢牢占据前五位，过亿品牌在8个以上，国内药企方面，正大制药以6个过亿品牌突围，华润三九、恒瑞医药、石药控股各有4个过亿品牌。 图7：2024上半年过亿品牌≥4个的集团 正大制药6个过亿品牌合揽超过10亿元，最畅销的是正大天晴药业集团的盐酸安罗替尼胶囊，2024上半年销售额接近3.3亿元，增长率为9.35%，此外还有4个品牌呈现正增长，江苏正大清江制药的盐酸氨基葡萄糖片增长了99.72%、北京泰德制药的氟比洛芬凝胶贴膏增长了5.68%、正大天晴南京顺欣制药的贝伐珠单抗注射液和利妥昔单抗注射液分别增长了731.69%、6428.27%。 华润三九4个过亿品牌合揽超过13亿元，最畅销的是澳诺(中国)制药的葡萄糖酸钙锌口服溶液，2024上半年销售额超过10亿元，此外还有2个品牌呈现正增长，华润三九(南昌)药业的糠酸莫米松凝胶增长了27.04%、华润三九(黄石)药业的小儿氨酚黄那敏颗粒增长了12.32%。 恒瑞医药4个过亿品牌均为1类新药，合揽超过6亿元，最畅销的是苏州盛迪亚生物医药的注射用卡瑞利珠单抗，2024上半年销售额超过2亿元，此外还有3个品牌呈现正增长，江苏恒瑞医药的海曲泊帕乙醇胺片和马来酸吡咯替尼片分别增长了38.90%、11.58%，上海盛迪医药的阿得贝利单抗注射液增长了214.64%。 石药控股4个过亿品牌合揽超过5亿元，最畅销的是石药中诺药业(石家庄)的阿莫西林胶囊，2024上半年销售额接近2亿元，增长率为1.02%，此外石药恩必普药业的丁苯酞软胶囊增长了0.03%，石药欧意药业的头孢克肟胶囊和甲钴胺片分别增长了32.17%、64.97%。 资料来源：米内网数据库注：米内网《中国城市实体药店终端竞争格局》，统计范围是：全国地级及以上城市实体药店，不含县乡村药店；上述销售额以产品在终端的平均零售价计算。 本文为原创稿件，转载请注明来源和作者，否则将追究侵权责任。投稿及报料请发邮件到872470254@qq.com稿件要求详询米内微信首页菜单栏商务及内容合作可联系QQ：412539092 【分享、点赞、在看】点一点不失联哦

The XLH Network and Kyowa Kirin publish findings from community survey, accompanying white paper New evidence shows wide-ranging impact of XLH, including finances, daily activities, and mental health Survey insights will be shared at the NORD Breakthrough Summit in Washington, D.C., October 20-22 PRINCETON, N.J., Oct. 15, 2024 /PRNewswire/ -- Kyowa Kirin, Inc., a wholly owned subsidiary of Kyowa Kirin Co. Ltd., along with The XLH Network, a non-profit patient advocacy organization, today announced the publication of Insights for Change: Findings from the XLH Community Impact Survey, an inside look at the experiences of people living with X-linked hypophosphatemia (XLH), a rare, progressive, genetic disease that impacts the bones and muscles in both children and adults.1 Continue Reading XLH joint logo View PDF The white paper reveals the broader impact of XLH on U.S. patients and their families, including personal finances and out of pocket expenses; physical, mental, and social health; and the challenges faced when accessing expert care. The full survey data report and white paper are the result of a collaboration between Kyowa Kirin and The XLH Network and are now available along with other resources at XLHimpact.com. "For generations, families living with XLH have struggled to explain the disease to many of the professionals involved in their care. Without the data to back them up, patients have often reported feeling dismissed, or that their concerns were not validated," said Susan Faitos, Executive Director of The XLH Network. "Research projects like this, which focus on the actual lived experience of someone with XLH, will lead to a greater understanding of XLH and facilitate meaningful discussions between XLHers and all those involved in their care. The XLH Network is proud to be a part of this project." More than 85 adults with XLH and caregivers of children with XLH responded to the online survey, and 56 of these respondents completed additional follow-up phone interviews. The following key findings illustrate the ongoing challenges of living with XLH. Access to Care: XLHers face unique challenges accessing care. Survey respondents rated their XLH doctors as fairly knowledgeable; however, they spent considerable time and money to access their doctors. Survey respondents reported traveling on average 180 miles roundtrip, taking over three hours just to get to and from their XLH doctor's office. These travel requirements proved to be particularly costly, amounting to on average $1,000 and as much as $15,000 in annual travel costs for survey respondents. Financial Burden: The broad health implications of living with XLH are reflected in the wide array of expenses reported for direct XLH care and other expenses such as home modifications, dental procedures, and travel to doctor offices. Survey respondents indicated that they spent an average of 6% – and in one case as much as 40% – of their annual household income on out of pocket (OOP) expenses related to managing or living with XLH in the last year. In addition, over the past year, 61% of respondents paid OOP for dental care, with annual costs averaging $2,775. Impact on Daily Activities: The survey and white paper also document the considerable toll XLH has on an individuals' day-to-day and social life. When asked to identify the most burdensome impacts on their daily lives, XLHers commonly cited absenteeism, reduced productivity at work or school, and challenges with personal relationships. Some adult survey respondents noted that XLH restricts daily activities such as getting dressed, housework, driving, shopping, and cooking. Mental Health Impact: XLH can strain patients' and families' mental and emotional health resources. A majority of respondents – 83% of adults and 90% of children – reported at least one mental health issue related to their XLH. While most expressed concerns about their overall mental health, very few said they were seeing a mental health professional to address these concerns (13% of adults, 3% of children). Transition of Care: Because of the lifelong, progressive nature of XLH, most XLHers are diagnosed during childhood but continue to experience symptoms into adulthood.1 Nevertheless, an overwhelming majority (94%) of adult XLHers who completed the survey reported they did not formally transition their care from a pediatrician to an adult healthcare professional (HCP). When asked for a reason, 14 of the 49 adults who were diagnosed before age 18, stated they were advised the disease "would go away over time." "Kyowa Kirin is deeply committed to listening to and working with the XLH community to advance the understanding of XLH and the challenges this disease places on patients and their families," said Lauren Walrath, Vice President of Public Affairs at Kyowa Kirin North America. "As this survey makes clear, the needs of every XLHer are unique and while progress has been made, more work is needed to make care accessible and to ease the burden on families. We look forward to working with The XLH Network, healthcare providers, and other stakeholders on solutions that provide meaningful change at the community level." About the XLH Community Impact Survey The XLH Community Impact Survey is a collaboration between Kyowa Kirin and The XLH Network. The purpose of the survey was to quantify and deepen understanding of the lived experiences of adults, caregivers, and children with XLH in the United States. The survey evaluated the impact of XLH on personal finances and physical, mental, and social health, as well as access to expert quality care. Between November 2023 and February 2024, a total of 87 adults living with XLH and caregivers of children with XLH completed an online survey, with follow-up phone interviews conducted with 56 of the respondents. Survey responses reflect respondent variation across key demographics such as age, geography, and insurance status, and may not reflect the experiences of all individuals with XLH. Adult males, young adults aged 18-44, and individuals from rural settings were under-represented among respondents. Engage Health designed, fielded, and conducted analysis of the survey with analytic and editorial support from SmithSolve and input from The XLH Network and Kyowa Kirin, Inc. To learn more about XLH and the survey results, and to access additional resources, visit XLHimpact.com. X-linked hypophosphatemia X-linked hypophosphatemia is a rare, lifelong, genetic disease that can impact the bones and muscles in both children and adults.1 In individuals with XLH, the body doesn't hold on to enough phosphorus, which is an essential mineral for bone health.1 This is due to the production of excess fibroblast growth factor 23 (FGF23), causing the body to release too much phosphorus through the urine.1 When phosphorus levels are too low (hypophosphatemia), it can cause the softening and weakening of growing bones in children (rickets) and of mature bones in children and adults (osteomalacia).1 In children, XLH typically appears as bowed legs or knock knees.1 Over time, bone weakening can also lead to bone abnormalities in the legs, delayed growth, and short stature.1 In adults, XLH may cause osteomalacia, fractures and pseudo-fractures, and hypophosphatemia.1 About Kyowa Kirin Kyowa Kirin aims to discover novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients affected by severe and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites us across Japan, North America, and EMEA/International. You can learn more about Kyowa Kirin North America at: kkna.kyowakirin.com or LinkedIn: Kyowa Kirin Inc. U.S. About The XLH Network The XLH Network supports families and individuals living with X-Linked Hypophosphatemia (XLH). Our mission includes promoting awareness and education, providing opportunities for connection and community, and to foster the search for a cure. Our vision is early, accurate diagnosis; readily available, well-managed treatment; and healthy quality of life for those who are affected with XLH and related disorders. COR-US-CRY-0096 October 2024 1Dahir K, et al. X-linked hypophosphatemia: a new era in management. J Endocr Soc. 2020;4(12):bvaa151. SOURCE Kyowa Kirin WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

- Data underscore the urgent need for therapies that address the cardiovascular and musculoskeletal complications and long-term systemic effects of ENPP1 and ABCC6 Deficiencies in children - - Inozyme and GACI Global launched PROPEL Registry to further understanding of these rare, life-threatening diseases (NCT06302439) - BOSTON, Sept. 26, 2024 (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, is presenting four posters at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting, including new data that demonstrate the progression and impact of rare bone and blood vessel diseases in children. These presentations include findings highlighting the significant medical burden associated with ENPP1 Deficiency and early-onset ABCC6 Deficiency, new data from a natural history study that provides critical insights into the progression of ENPP1 Deficiency, and the launch of a global registry to enhance our understanding of these diseases. “Given their high mortality rate, there is an urgent unmet need for patients affected by these rare bone and blood vessel diseases. For instance, more than half of all infants with ENPP1 Deficiency die within six months, and those that survive often face major and potentially fatal complications throughout their lives,” said Kurt Gunter, M.D., Chief Medical Officer of Inozyme Pharma. “These new data underscore the significant and multifaceted impact of ENPP1 and ABCC6 Deficiencies, further highlighting the significant need for therapies that address the immediate and long-term systemic complications associated with these conditions. At Inozyme, we are dedicated to unlocking the full potential of our lead candidate, INZ-701, a potentially transformative treatment for patients and their families facing these diseases.” “As the data from these retrospective analyses demonstrates, the impact of these life-threatening conditions is more significant in both the short and long-term than we had understood, particularly in terms of cardiovascular and musculoskeletal complications,” said Christine O’Brien, co-president of GACI Global. “We’re excited to work with Inozyme to launch the PROPEL Registry to prospectively collect clinical data and patient-reported outcomes that will provide a comprehensive understanding of the burden of illness and progressive nature of ENPP1 Deficiency and early-onset ABCC6 Deficiency.” Natural History Study of 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. Key findings of the retrospective analysis included: In the 84 patients with ENPP1 Deficiency, cardiovascular complications and arterial calcifications developed early in life, predominately within the first few months.A striking 88% of patients experienced ectopic calcifications, with 81% showing arterial calcification and 66.7% showing aortic calcification. Cardiovascular complications, including hypertension, heart valve defects and heart failure, were noted in 76% of patients.Among the 84-patient cohort, 51 were diagnosed with Generalized Arterial Calcification of Infancy (GACI) alone and only 19 survived beyond infancy; 22 were diagnosed with GACI, survived beyond infancy and progressed to Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2); and 11 patients presented initially with ARHR2.Musculoskeletal complications, including phosphate-wasting rickets, bowed extremities, and pain, were documented in all patients diagnosed with ARHR2, with 45% of ENPP1 Deficiency survivors showing symptoms by age 3 and 75% by age 11.The data highlight that 95% of surviving ENPP1 Deficiency patients are expected to experience cardiovascular, musculoskeletal, and other organ complications, underscoring the lifelong burden of the disease. Title: Lifetime Accumulation of Calcification and Clinical Complications in Patients with ENPP1 Deficiency (GACI and ARHR2)Format: Poster PresentationAuthor/Presenter: Carlos Ferreira, M.D., of the National Institutes of Health (NIH)Session Dates and Times: Rare Bone Disease Symposium: Thursday, September 26, 2024, 8:00 a.m. – 6:00 p.m. ET Early Career Spotlight: Friday, September 27, 2024, 4:54 p.m. ET Plenary during Welcome Reception: Friday, September 27, 2024, 5:30 – 7:30 p.m. ET Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET Retrospective Observational Study of ENPP1 Deficiency and Early-onset ABCC6 Deficiency Like ENPP1 Deficiency, ABCC6 Deficiency is a progressive and debilitating rare disease that affects blood vessels and soft tissues. Infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI Type 2), which is similar to GACI Type 1, the infant form of ENPP1 Deficiency. This study described the clinical events and interventions in patients with ENPP1 Deficiency (n=14) and early-onset ABCC6 Deficiency (n=9).ENPP1 Deficiency was associated with a high burden of cardiovascular complications in infancy, with additional frequent medical interventions including orthopedic surgery (64%) and hearing aids (43%).Early-onset ABCC6 Deficiency patients showed significant cardiovascular complications, and 44% experienced stroke. Title: The Medical Burden of ENPP1 Deficiency and Early-Onset ABCC6 Deficiency from a Retrospective Observational StudyFormat: Poster PresentationAuthor/Presenter: David R. Weber, M.D., MSCE, Attending Physician and Medical Director of the Center for Bone Health at Children’s Hospital of PhiladelphiaSession Dates and Times: Rare Bone Disease Symposium: Thursday, September 26, 2024, 8:00 a.m. – 6:00 p.m. ET Plenary during Welcome Reception: Friday, September 27, 2024, 5:30 – 7:30 p.m. ET Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET Additional poster presentations include: Title: PROPEL: Prospective Observational Registry for ENPP1 Deficiency and Infantile-Onset ABCC6 DeficiencyFormat: Poster PresentationAuthor/Presenter: Lothar Seefried, M.D., of the Orthopedic Department of the University Hospital at the Julius-Maximilians University Würzburg, GermanySession Dates and Times: Energy Metabolism in Skeletal Development and Disease: Thursday, September 26, 2024, 8:30 a.m. – 5:30 p.m. ET Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET Title: Therapeutic Application of ENPP1-Fc Prevent Muscle Calcification Following Cardiotoxin-induced-muscle Damage in Abcc6 -/- miceFormat: Poster PresentationAuthor/Presenter: Kevin O’Brien, Principal Scientist, Inozyme PharmaSession Date and Time: Late Breaking Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET 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 rickets, specifically 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. Biallelic ENPP1 Deficiency affects approximately 1 in 64,000 pregnancies worldwide. Initially, it was believed to only impact individuals with two copies of the mutated gene. However, many individuals with just one copy of the mutated gene (monoallelic ENPP1 Deficiency) also exhibit severe symptoms. This suggests that the worldwide prevalence of ENPP1 Deficiency may be much higher than current estimates, which are based solely on biallelic cases. Currently, there are no approved therapies for ENPP1 Deficiency. About ABCC6 Deficiency ABCC6 Deficiency is a progressive and debilitating rare disease that affects blood vessels and soft tissues. Infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI Type 2), which is similar to GACI Type 1, the infant form of ENPP1 Deficiency. Pediatric patients who survive beyond the first year of life may develop neurological disease, including strokes, and cardiovascular diseases due to ongoing vascular calcification and stenosis. In older individuals, ABCC6 Deficiency manifests as pseudoxanthoma elasticum (PXE), characterized by abnormal mineralization in blood vessels and soft tissues, affecting the skin, visual function, and vascular system. Biallelic ABCC6 Deficiency is estimated to affect 1 in 25,000 to 1 in 50,000 individuals worldwide. Initially, it was believed to only impact individuals with two copies of the mutated gene. However, many people with just one copy of the mutated gene (monoallelic ABCC6 Deficiency) also exhibit severe symptoms. This suggests that the worldwide prevalence of ABCC6 Deficiency may be much higher than current estimates, which are based solely on biallelic cases. Currently, there are no approved therapies for ABCC6 Deficiency. About Inozyme PharmaInozyme Pharma is a pioneering clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics for rare diseases that affect bone health and blood vessel function. We are experts in the PPi-Adenosine Pathway, where the ENPP1 enzyme generates inorganic pyrophosphate (PPi), which regulates mineralization, and adenosine, which controls intimal proliferation (the overgrowth of smooth muscle cells inside blood vessels). Disruptions in this pathway impact the levels of these molecules, leading to severe musculoskeletal, cardiovascular, and neurological conditions, including ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis, and ossification of the posterior longitudinal ligament (OPLL). Our lead candidate, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) designed to increase PPi and adenosine, enabling the potential treatment of multiple diseases caused by deficiencies in these molecules. It is currently in clinical development for the treatment of ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis. By targeting the PPi-Adenosine Pathway, INZ-701 aims to correct pathological mineralization and intimal proliferation, addressing the significant morbidity and mortality in these devastating diseases. For more information, please visit https://www.inozyme.com/ or follow Inozyme on LinkedIn, X, and Facebook. Cautionary Note Regarding Forward-Looking StatementsStatements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the potential benefits of INZ-701. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to conduct its ongoing clinical trials of INZ-701 for ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis; enroll patients in ongoing and planned trials; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain, and protect intellectual property rights related to its product candidates; manage expenses; comply with covenants under its outstanding loan agreement; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. Contacts Investors:Inozyme PharmaStefan Riley, Senior Director of IR and Corporate Communications(857) 330-8871Stefan.riley@inozyme.com Media:Biongage CommunicationsTodd Cooper(617) 840-1637Todd@biongage.com