First Patient Dosed in Phase IIa Trial of Regenerative Cell Therapy for End-Stage Liver Disease
Open label, dose-escalation trial is testing the efficacy of LyGenesis’ first-in-class allogenic regenerative cell therapy.
The first patient has been dosed in an open-label, dose-escalation Phase IIa clinical trial (NCT04496479) evaluating a first-in-class allogenic regenerative cell therapy in patients with end-stage liver disease (ESLD), according to a press release.1
LyGenesis' LYG-LIV-001 is an allogeneic cell therapy that is transplanted into patients’ lymph nodes as a potential treatment for ESLD. It is a biologic therapy that is created starting from donated otherwise unmatched livers.
"In a medical first, we have now dosed our first patient in a clinical trial using their own lymph nodes as living bioreactors to regenerate an ectopic organ," Michael Hufford, PhD, co-founder and CEO of LyGenesis said in the press release. "This therapy will potentially be a remarkable regenerative medicine milestone by helping patients with ESLD grow new functional ectopic livers in their own body. If our study is successful and we obtain FDA approval, our allogenic cell therapy could enable one donated liver to treat many dozens of ESLD patients, which could help to tilt the current organ supply-demand imbalance in favor of patients."
According to the American Liver Foundation, more than 100 million people in the United States have some form of liver disease with 4.5 million adults having been diagnosed. It is estimated that 80-100 million adults in the US have fatty liver disease and many do not know they have it. Chronic liver disease/cirrhosis was the 12th leading cause of death in the US in 2020.2
The Phase IIa trial is recruiting 12 patients with ESLD. They will be monitored for over a year through follow-up visits. A data safety monitoring board monitors each cohort of patients to evaluate near-term safety prior to dose escalation for the next cohort of patients.
"This clinical trial heralds more than an important milestone in the study of bench-to-bedside translational medicine, it is a prime example of LyGenesis' ongoing investigational efforts to translate cutting-edge innovative scientific discoveries, such as using the lymph nodes as a bioreactor to grow functioning ectopic organs, into clinical reality for patients suffering from life-threatening illnesses. LyGenesis' platform of regenerative cell therapies extends beyond ESLD to Type 1 diabetes, renal disease, and aging," Richard Marshall CBE, MD, PhD, CEO of Juvenescence, an investor in LyGenesis said in the press release.
"LyGenesis' cell therapy platform represents a truly remarkable potential commercial opportunity and may be transformative for chronic liver failure patients who do not have access to a donor liver,” Justin Briggs, operating partner at Prime Movers Lab, another investor in LyGenesis said in the press release. “Their use of an endoscopic ultrasound as a low risk and low cost route of cell therapy administration is another way this pioneering technology could provide patients with access to life-saving therapies and address complex medical challenges by upending transplant medicine.”
In 2022, LyGenesis entered into a joint research collaboration with iTolerance to evaluate the potential of iTOL-201, a product candidate which utilizes iTolerance's SA FasL microgel immune tolerance platform (iTOL-100) combined with LYG-LIV-100, to enable ectopic livers to be grown without the need or immune suppression.3
References
1. LyGenesis Announces First Patient Dosed in its Phase 2a Clinical Trial of a First-in-Class Regenerative Cell Therapy for Patients with End-Stage Liver Disease. News release. April 2, 2024. Accessed April 2, 2024. https://www.prnewswire.com/news-releases/lygenesis-announces-first-patient-dosed-in-its-phase-2a-clinical-trial-of-a-first-in-class-regenerative-cell-therapy-for-patients-with-end-stage-liver-disease-302105734.html
2. How Many People Have Liver Disease? American Liver Foundation. Updated September 11, 2023. Accessed April 2, 2024. https://liverfoundation.org/about-your-liver/facts-about-liver-disease/how-many-people-have-liver-disease/#:~:text=Left%20untreated%2C%20liver%20disease%20can,in%20the%20U.S%20in%202020.
3. iTolerance and LyGenesis Announce a Joint Research Collaboration to Regrow Functioning, Ectopic Livers Without the Need for Immune Suppression. News release. June 30, 2022. Accessed April 2, 2024. https://www.prnewswire.com/news-releases/itolerance-and-lygenesis-announce-a-joint-research-collaboration-to-regrow-functioning-ectopic-livers-without-the-need-for-immune-suppression-301578136.html
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