Sentynl Therapeutics Licenses Progerinin for Hutchinson-Gilford Progeria Syndrome Treatment
核心洞察
Sentynl Therapeutics (搜索), a subsidiary of Zydus Lifesciences, has entered into a licensing agreement with PRG S&T (搜索) to acquire rights to Progerinin (SLC-D011), an investigational oral small-molecule drug for Hutchinson-Gilford Progeria Syndrome (搜索).
Progerinin demonstrated significant efficacy in mouse models, extending average lifespan from 16.8 weeks in untreated controls to 25.2 weeks in treated animals (p < 0.001).
The drug has received FDA orphan drug designation and is currently finalizing a Phase 2A clinical trial with data expected by the end of the first half of 2026.
Sentynl Therapeutics (搜索) Inc., a U.S.-based biopharmaceutical company and wholly-owned subsidiary of Zydus Lifesciences Limited, has announced a licensing agreement with PRG S&T (搜索) to acquire rights to Progerinin (SLC-D011), an investigational treatment for Hutchinson-Gilford Progeria Syndrome (搜索) (HGPS (搜索)). The agreement enables Sentynl to begin immediate collaboration with the Korean rare disease specialist to advance clinical development of the orphan drug-designated therapy.
Promising Preclinical Results Drive Acquisition
Progerinin demonstrated encouraging outcomes in mouse models of HGPS (搜索), significantly extending survival compared to untreated controls. In mice with a severe form of the disease, treatment increased lifespan by 8-10 weeks and improved body weight. The untreated control group had a shorter lifespan with an average of 16.8 weeks and maximum of 18 weeks, whereas the treated group exhibited a significantly extended lifespan with an average of 25.2 weeks and maximum of 26 weeks (p < 0.001).
"This acquisition marks an important step in growing our portfolio of therapies for Hutchinson-Gilford Progeria Syndrome (搜索), which can have severe impacts on patient health if left untreated," said Dr. Sharvil P. Patel, Managing Director of Zydus Lifesciences Limited. "Supporting patients in living healthy, fulfilled lives is core to what we do, and the agreement with PRG S&T (搜索) directly furthers this mission by advancing orphan therapies for patients and families impacted by rare diseases."
Targeting Progerin Protein Dysfunction
Progerinin is an investigational, orally active small-molecule drug designed to treat HGPS (搜索), a rare genetic disorder characterized by accelerated aging in children. The disease is caused by the accumulation of progerin (搜索), an abnormal form of the lamin A (搜索) protein produced by mutations in the LMNA (搜索) gene, which disrupts nuclear structure and leads to premature cellular aging.
The drug candidate works by inhibiting the interaction and harmful effects of progerin (搜索) within cells, thereby improving nuclear integrity and reducing cellular damage. In HGPS (搜索), the LMNA (搜索) mutation causes cells to produce progerin instead of normal lamin A (搜索), leading to defects in the cell nucleus and accelerating aging symptoms.
Addressing Critical Unmet Medical Need
HGPS (搜索) is an ultra-rare, fatal genetic disorder affecting children, with patients commonly dying of atherosclerosis (搜索) at an average age of 14.5 years. Disease manifestations include severe failure to thrive, scleroderma-like skin, global lipodystrophy, alopecia, joint contractures, skeletal dysplasia, global accelerated atherosclerosis with cardiovascular decline, and debilitating strokes.
Currently, Zokinvy (lonafarnib) is the only approved treatment for HGPS (搜索) and certain processing-deficient Progeroid Laminopathies in the U.S., European Union, Great Britain, Israel, and Japan, leaving significant unmet need for additional therapeutic options.
"Children with Hutchinson-Gilford Progeria Syndrome (搜索) face an unforgiving disease. However, we are seeing real progress in progeria (搜索) research, with new science changing what's possible," said Matt Heck, CEO of Sentynl. "This agreement, which will add Progerinin to our progeria portfolio, represents our commitment to translating that progress into another real therapy for children and families who need them."
Clinical Development Timeline
The program is currently finalizing a Phase 2A clinical trial with data expected before the end of the first half of 2026. Under the agreement conditions, Sentynl will acquire full rights to the molecule for HGPS (搜索) upon meeting certain milestones, making Progerinin the company's second therapy intended for the treatment of HGPS.
The Progeria Research Foundation (搜索), which funded the foundational research that led to Progerinin's development, welcomed the announcement. "PRF's mission is to find treatments and the cure for Progeria (搜索), and we are grateful for the efforts of PRG S&T (搜索) and Sentynl to improve the lives of the children and young adults in our Progeria patient community," said Leslie Gordon, MD, PhD, Medical Director at Progeria Research Foundation.
Progerinin is not currently approved by FDA or any other health authority but has received orphan drug designation from the United States Food and Drug Administration.