Lonafarnib

New Trial to Study Progerinin Treatment for Ultra-Rare Condition BUSAN, KOREA; PEABODY, MA; and GERMANTOWN, MD / ACCESSWIRE / October 2, 2024 / The United States Food and Drug Administration (FDA) has authorized the start of patient enrollment for a Phase 2a clinical trial of Progerinin, an investigational drug being developed for the treatment of Progeria. Advancing the development of Progerinin could potentially provide an additional treatment for this fatal disease. PRG Science & Technology logo Hutchinson-Gilford Progeria Syndrome (“Progeria”) is an ultra-rare, genetic, “rapid-aging” condition affecting approximately 1 in 18 million people. Without treatment, children with Progeria die of heart disease at an average age of 14.5 years old. Progeria is caused by a random genetic mutation that produces an overabundance of the toxic protein, progerin. The accumulation of progerin occurs with aging in the general population, in the cardiovascular system and elsewhere in the body, such as the skin. In children and young adults with Progeria, the rate of progerin accumulation is highly accelerated, resulting in progressive cellular damage and atherosclerotic heart disease. Progerinin was developed by Korean-based biotech company PRG Science & Technology Co., Ltd. (PRG S&T) with research funding from The Progeria Research Foundation (PRF). The trial is a collaborative effort between PRG S&T, PRF, and trial site Boston Children’s Hospital, and will be managed by contract research organization Amarex. In the Phase 2a randomized open label trial, Progerinin will be administered in combination with the current standard-of-care, Zokinvy, to 10 patients with Progeria and Progeroid Laminopathies (PL) who are one year of age and older. Zokinvy has been shown to increase lifespan in a Progeria mouse model by 25%, and data published in the journal Cells in April 2023 indicated that Progerinin showed a potential increase in Progeria mouse lifespan by 50%. Zokinvy works by blocking the toxic, disease-causing protein, progerin, from developing and attacking normal cells, while Progerinin appears to reduce the level of progerin that accumulates in the body. Currently, Zokinvy is the only treatment for Progeria and PL approved by the FDA (in 2020), the European Medicines Agency (EMA, in 2022) and the Japanese Ministry of Health, Labor and Welfare (MHLW, in 2024). “Since its establishment, PRG S&T has focused on developing rare genetic disease treatments. This clinical trial demonstrates PRG S&T’s R&D capabilities to improve access to new drugs and expand treatment opportunities for children with progeria worldwide,” said Bumjoon Park, president of PRG S&T. “PRG S&T will focus on R&D and technological innovation to accelerate the development of treatments, including preparation for follow-up clinical trials, and lead to the development of new drugs.” “This new trial represents another step forward in our mission to treat and cure these amazing children and young adults with Progeria,” said PRF Executive Director Audrey Gordon, Esq. “Each Progeria clinical trial builds on learnings from our previous trials, so that we are continually making progress toward even better treatments and the cure. Most importantly, we are grateful for the courage and resilience of these children and young adults with Progeria and their families for participating in research programs and contributing to this important mission.” “Patients with Progeria and their families deserve more options,” said Dr. Kush Dhody, President, Amarex Clinical Research. “This is an especially devastating diagnosis given the shortened lifespan it causes. We’re honored to support a study that brings more hope to these patients.” About Progeria Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS), and PL are ultra-rare, multisystemic, premature aging diseases that accelerate mortality in young patients. Progeria is caused by a genetic mutation in the LMNA (“lamin A”) gene, and results in a disease-causing abnormal protein called progerin. There are approximately 400-450 children and young adults worldwide with Progeria. Thanks to PRF-funded research, we now know that progerin is produced in all of us as we age, but at a much lower rate than in children with Progeria. Due to this discovery of the biological connection between Progeria, heart disease and aging, finding the cure for one of the rarest diseases on earth could provide keys for treating millions of adults with heart disease and stroke associated with the natural aging process, as well as help the entire aging population. About PRG Science & Technology Co., Ltd. PRG S&T is a research & development company specializing in the treatment of rare genetic diseases. PRG S&T has been developing therapeutics for rare genetic diseases (laminopathies, neurodegenerative diseases, neuro-oncology etc.) with small molecules, which are derived from target sites on aberrant Protein-Protein Interactions (PPI). All four pipelines currently in PRG S&T’s possession have been designated as orphan drugs by the US FDA. Progerinin, the investigational treatment for Progeria has been approved by the FDA to enter phase 2 clinical trials, and is about to enter Phase2 at Boston Children’s Hospital. The non-clinical study of PRG-N-01, the treatment for neurofibromatosis type2, has been completed. IND for Phase I/II of PRG-N-01 has been approved by the Korea FDA and has been administered to patients at Asan Medical Center (South Korea) in June 2024. If these two drugs, Progerinin and PRG-N-01, are developed successfully, they are expected to become first-in-class innovative new drugs. For more information, please visit . About The Progeria Research Foundation The Progeria Research Foundation (PRF) was established in 1999 by the family of Sam Berns, a child with Progeria. Within four years of its founding, the PRF Genetics Consortium discovered the Progeria gene, a collaboration led by Dr. Francis Collins, former Director of the National Institutes of Health (NIH). PRF has funded and co-coordinated all lonafarnib-associated clinical trials for Progeria and Progeroid Laminopathies, conducted at Boston Children’s Hospital, and supports scientists who conduct Progeria research worldwide. PRF’s International Patient Registry includes over 370 children with Progeria in 70 countries. PRF is the only non-profit organization solely dedicated to finding treatments and the cure for Progeria and its aging-related conditions, including heart disease. The organization fills a void, putting these children and Progeria at the forefront of scientific efforts. For more information and to support PRF’s mission, please visit . About Amarex Clinical Research, LLC, an NSF company Amarex Clinical Research, LLC, is a global, full-service Contract Research Organization (CRO) with significant expertise conducting biomedical research. The leadership team’s combined experience includes the design and conduct of several hundred clinical research projects in many therapeutic indications. Amarex provides services in Project Management: Phase I-IV, BE/BA, PK/PD; Regulatory Affairs: FDA Applications and meetings, applications to International Health Authorities, GxP Compliance Audits; Clinical Operations; Adaptive Study Designs; Statistical Analysis; Data Management; Medical Monitoring; Safety and Pharmacovigilance; and General Consulting. Amarex can take your product through the entire approval process, from creating the regulatory approval strategy, to conducting trials, to writing the marketing approval application. Join our growing list of clients with approved products assisted by quality, cost-efficient services. For more information visit . Contact Information Eleanor Maillie PRF emaillie@progeriaresearch.org 978.879.9244 Sunghee Kim PRG S&T shkim@prgst.com Kalli DeWeese Amarex kdeweese@nsf.org Related Images PRG Science & Technology logo Amarex Clinical Research logo PRF logo SOURCE: The Progeria Research Foundation View the original press release on newswire.com.

近期，GoodRx再次列出了市场上最昂贵的10种药物，包括药房出售的药物以及仅在医院中使用的药物。列表中的药物是基于价格标价，与消费者在药房或通过药品支持计划支付的价格或许存在差异。 NO.1 Zolgensma 诺华用于治疗脊髓性肌萎缩症的基因疗法Zolgensma以212.5万美元的价格位居榜首。  NO.2 Zokinvy Zokinvy在2020年11月刚刚获批上市，是Eiger生物制药公司针对Hutchinson-Gilford早老综合征（HGPS或早衰症）和早衰样核纤层蛋白病开发的首个治疗方法。以50mg胶囊717美元的价格计算，月费用将达到86040美元。因此，也就使得Zokinvy像大多数孤儿药物一样，年费用高达1032480美元。  NO.3 Danyelza Danyelza是昂贵药物俱乐部的新晋成员。纽约Y-mAbs制药公司开发的这款药物同样于2020年11月刚刚获得批准，用于治疗骨或骨髓复发或难治性高危神经母细胞瘤的小儿患者，每年的费用为977664美元。  NO.4 Myalept Myalept用于治疗全身性脂肪分布异常的全身性脂肪营养不良和瘦素缺乏症，是同靶点唯一一款上市的药物，甚至该疾病也没有其他处于临床后期药物，患者治疗选择极少。 Myalept的价格在今年2月从每月71306美元增加到74159美元，每年费用近89万美元。 NO.5 Luxturna Luxturna的价格为每年85万美元，位列第五位。Luxturna是一种基因疗法，可治疗遗传性视网膜营养不良，这种疾病会导致视力丧失，甚至完全失明。Luxturna只能在院内使用，需要医生向每只眼睛中注射药物，但标价昂贵，每瓶价格为42.5万美元。 NO.6 Folotyn Folotyn属于二氢叶酸还原酶（dihydrofolate reductase，DHFR）抑制剂，被批准用于治疗外周T细胞淋巴瘤，年费用近80万美元。Folotyn最早于2009年率先在美国上市，2020年也在中国获批。 NO.7 Brineura Brineura是First-in-class的酶替代疗法药物，2017年首次上市，用于治疗晚期婴儿神经元类神经脂类融合蛋白2型（CLN2）Batten病（CLN2-Batten）。 由于技术问题，无论是靶点还是疾病考虑，Brineura的竞争严重不足。2021年1月，300mg规格Brineura再次调高2.14%至28090美元，推荐剂量为每2周300mg，这意味着在一年的时间里费用总计为73万美元。  NO.8 Blincyto Blincyto是安进开发的一款CD3/CD19双特异性抗体，2014年在美国上市，用于治疗复发或难治的CD19阳性B细胞前体急性淋巴细胞白血病。通常每年需要使用约168瓶，按目前的定价，达到每瓶4242美元，年度费用为712672美元。  NO.9 Ravicti Ravicti（苯丁酸甘油）用于治疗尿素循环疾病，是Horizon公司针对其相同适应症药物苯丁酸钠开发的一种升级疗法，患者每年需要服用132瓶。2021年1月，Ravicti的标价上涨了4.8％，至每瓶5273美元，按标价计算的年费用为69万美元。  NO.10 Soliris Soliris的费用为每年678392美元，用于治疗阵发性夜间血红蛋白尿、非典型溶血性尿毒症综合征、重症肌无力和视神经脊髓炎。 参考：制药工艺与装备 投稿/内容沟通：华籍美人（Ww_150525） 近期精华文章