US FDA Approves Clinical Trial for Progeria Treatment Development

The United States Food and Drug Administration (FDA) has granted authorization to commence patient enrollment for a Phase 2a clinical trial of Progerinin, an experimental drug aimed at treating Hutchinson-Gilford Progeria Syndrome (Progeria). This ultra-rare genetic disorder, characterized by rapid aging, affects approximately 1 in 18 million people globally. Without any intervention, children diagnosed with Progeria typically succumb to heart disease by the age of 14.5 years. The disease is caused by a random genetic mutation that leads to an excessive production of the toxic protein progerin, which accumulates rapidly in the cardiovascular system and various other parts of the body, causing severe cellular damage and atherosclerosis.

Progerinin is being developed by PRG Science & Technology Co., Ltd. (PRG S&T), a biotech company based in Korea, with funding support from The Progeria Research Foundation (PRF). The Phase 2a trial is a collaborative effort involving PRG S&T, PRF, and Boston Children’s Hospital, with contract research organization Amarex overseeing the trial’s management.

The trial aims to administer Progerinin in combination with the existing standard-of-care treatment, Zokinvy, to ten patients aged one year and older who are suffering from Progeria and Progeroid Laminopathies (PL). Zokinvy has already been shown to extend the lifespan of a Progeria mouse model by 25%. Moreover, research published in the journal *Cells* in April 2023 suggested that Progerinin could potentially increase the lifespan of Progeria mice by 50%. Zokinvy functions by inhibiting the formation of progerin, thereby protecting normal cells, while Progerinin helps in reducing the accumulated levels of progerin in the body. Zokinvy is currently the only FDA-approved treatment for Progeria and PL, with approvals also from the European Medicines Agency in 2022 and the Japanese Ministry of Health, Labor and Welfare in 2024.

Bumjoon Park, the president of PRG S&T, highlighted the company's dedication to developing treatments for rare genetic diseases. He emphasized that this clinical trial showcases PRG S&T's research and development capabilities and underlines their commitment to enhancing access to new medications. Park noted that the company would focus on further R&D and technological innovation to expedite the development of treatments, including preparing for subsequent clinical trials and aiming to develop new drugs.

Audrey Gordon, Executive Director of PRF, expressed optimism about the new trial, considering it a significant step forward in the quest to find treatments and cures for Progeria. She acknowledged the contributions of previous clinical trials and the ongoing progress they represent. Gordon also extended gratitude to the children and young adults participating in these trials for their bravery and resilience.

Kush Dhody, President of Amarex Clinical Research, emphasized the importance of offering more treatment options to Progeria patients and their families, noting the devastating impact of the disease on life expectancy. He expressed honor in supporting a study that brings hope to those afflicted by this condition.

Progeria and Progeroid Laminopathies are extremely rare, multisystemic premature aging diseases caused by mutations in the LMNA gene, resulting in the production of an abnormal protein called progerin. Globally, there are approximately 400-450 children and young adults diagnosed with Progeria. Research funded by PRF has indicated that progerin is produced in all humans as they age, albeit at much lower levels compared to Progeria patients. The discovery underscores the biological link between Progeria, heart disease, and the natural aging process, suggesting that finding a cure for Progeria could provide insights into treating heart disease and stroke in the broader population.

PRG S&T specializes in developing treatments for rare genetic diseases, focusing on small molecule therapeutics targeting aberrant Protein-Protein Interactions. Their current medical pipeline includes four orphan drugs designated by the FDA, including Progerinin. If successful, these drugs could become pioneering treatments in their respective fields.