Hutchinson-Gilford progeria syndrome

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.

SEATTLE, Aug. 6, 2024 /PRNewswire/ -- YouthBio Therapeutics (YouthBio), a pioneering longevity biotechnology company developing partial reprogramming-based gene therapies, today announced significant research results from its collaboration with Dr. Alejandro Ocampo, Professor at the University of Lausanne and a leading figure in aging research whose pioneering work has given rise to the partial reprogramming field. The study, titled "Neuron-specific partial reprogramming in the dentate gyrus impacts mouse behavior and ameliorates age-related decline in memory and learning," demonstrates that brain-specific induction of partial reprogramming can ameliorate cognitive decline associated with aging. These findings add to the growing body of evidence for the therapeutic potential of in vivo partial reprogramming for the prevention and treatment of neurodegenerative disorders. Key Findings This study used two different approaches: a neuron-specific transgenic reprogrammable mouse model and a neuron-specific targeted lentiviral delivery of OSKM reprogramming factors. Both methods showed that partial reprogramming of adult neurons in the dentate gyrus of the hippocampus impacts animal behavior and cognitive function, and mitigates age-related decline in memory and learning. "These exciting findings from our collaboration with Dr. Ocampo validate our approach and highlight the transformative potential of partial reprogramming in combating age-related cognitive decline," said Yuri Deigin, CEO and co-founder of YouthBio. "We are thrilled to see such promising results and are committed to advancing this research further." Dr. Ocampo added, "I am delighted to collaborate with YouthBio to accelerate clinical translation of partial cellular reprogramming for treating age-related diseases, particularly neurodegenerative conditions. It is also very exciting to see other research groups demonstrating lifespan extension in wildtype mice through partial reprogramming. I believe that once we fully understand how to use this approach in a tissue-specific manner – something that YouthBio has recognized since the company's inception – we will see even greater increases in lifespan and more beneficial therapeutic effects in various disease models." "The potential of partial reprogramming for brain rejuvenation and the treatment of neurodegenerative disorders is very promising," said João Pedro de Magalhães, Ph.D., Chief Scientific Officer of YouthBio. "Our recent findings suggest that partial reprogramming could play a significant role in addressing age-related cognitive decline. While there is still much to learn and refine, these early results are encouraging and provide a solid foundation for further research. We are cautiously optimistic that with continued investigation, we can develop effective therapies that may one day benefit patients suffering from conditions like Alzheimer's disease." Future Plans and Alzheimer's Research YouthBio is committed to investigating the use of partial reprogramming in the CNS with a particular focus on Alzheimer's disease. In another study using transgenic ARTE10 mice treated with brain-specific inducible OSKM vectors, YouthBio observed reduced amyloid beta levels and a lower brain plaque burden in pivotal areas such as the hippocampus, and improved cognitive performance in behavioral tests compared to untreated controls. Based on these promising results, YouthBio has filed a provisional patent application with the USPTO. YouthBio's results are independently validated by a team of researchers from Altos Labs, the University of Barcelona, and the Max Planck Institute, who demonstrated favorable outcomes in preventing Alzheimer's symptoms through brain-specific partial reprogramming in a 5xFAD mouse model. Their study, "Expansion of the Neocortex and Protection from Neurodegeneration by In Vivo Transient Reprogramming," corroborates YouthBio's positive findings. "Our positive Alzheimer's data, along with the independent positive results by Altos Labs and its collaborators, reinforces our confidence in partial reprogramming's ability to provide a novel treatment paradigm for neurodegenerative conditions," said Deigin. "Our next key milestone is to bring our Alzheimer's therapy to clinical trials, and we are fully committed to achieving this goal." Clinical Trials and Beyond YouthBio aims to advance its Alzheimer's disease therapy to clinical trials within three years. During this period, the company will conduct the necessary preclinical studies, GMP manufacturing, and regulatory preparations essential for filing an Investigational New Drug (IND) application with the FDA. Concurrently, YouthBio will continue its discovery program, led by CSO Dr. João Pedro de Magalhães, to identify superior rejuvenating factors beyond the Yamanaka factors for use in subsequent generations of its tissue-specific gene therapies. The company will also conduct several proof-of-concept animal studies in other CNS indications and continue its progeria program. In their progeria program, YouthBio was granted a Rare Pediatric Disease Designation by the FDA for YB-001 for the treatment of Hutchinson-Gilford progeria syndrome (HGPS). This designation makes YouthBio eligible to receive a Priority Review Voucher (PRV) if YB-001 is approved for treating progeria. The PRV can be redeemed to receive an expedited priority review for any subsequent marketing application or sold. Historical PRV transaction values have exceeded $100mm, reaching as high as $350mm. *** About YouthBio Therapeutics, Inc. YouthBio Therapeutics () is a longevity company developing gene therapies that leverage partial reprogramming to restore a more youthful gene expression profile in targeted cells, leading to positive therapeutic effects in corresponding organs. It is currently focused on brain-specific gene therapies targeting CNS diseases, with Alzheimer's disease being the primary program. Contact: Yuri Deigin [email protected] Photo(s): Press release distributed by PRLog SOURCE YouthBio Therapeutics Inc.

The therapy is indicated for treating Hutchinson-Gilford Progeria Syndrome and processing-deficient progeroid laminopathies. Credit: LeviaUA / Shutterstock.com. Zydus Lifesciences subsidiary Sentynl Therapeutics has announced the worldwide acquisition of the Zokinvy (lonafarnib) programme from Eiger BioPharmaceuticals for the treatment of progeria. Zokinvy is the first and only treatment to obtain US FDA approval that targets the cause and symptoms of Hutchinson-Gilford Progeria Syndrome (HGPS) and processing-deficient progeroid laminopathies (PDPL), collectively called progeria. The treatment is indicated for usage in individuals aged 12 months and above. An ultra-rare, fatal genetic condition, progeria causes premature aging in young patients, leading to accelerated mortality. Since its approval in the US in 2020, Zokinvy has also received authorisation in the European Union and Great Britain in 2022, and most recently in Japan in January this year. See Also: Novartis acquires Mariana in $1.75bn deal to strengthen radioligand portfolio Vertex Pharmaceuticals gets grant for treatment of alpha-1 antitrypsin deficiency with compound of formula (i) The latest sale comes after Eiger and its subsidiaries filed for Chapter 11 bankruptcy in the US Bankruptcy Court in April this year. Subsequently, an auction was held as part of Eiger’s court-supervised sale process of Zokinvy. Sentynl emerged as the winning bidder for net base price of $45.2m. The Bankruptcy Court subsequently approved the sale to Sentynl with the transaction finalised on 3 May. Following the acquisition, Sentynl holds the worldwide rights to Zokinvy and will oversee its manufacturing and commercialisation efforts. The new deal will bolster Sentynl’s portfolio of biopharmaceutical products for rare and ultra-rare diseases. Sentynl president and CEO Matt Heck said: “It is an honour to add Zokinvy to our portfolio of products that have a tangible impact on the lives of rare disease patients, whose needs are too often unmet or overlooked. “We are firmly committed to provide best-in-class global access to Zokinvy and are eager to serve the patients and their families affected by progeria. We are grateful to Eiger and The Progeria Research Foundation for their dedicated effort to develop and secure availability of this life-changing product.”