World Diabetes Day 2024: Looking Back at Progress for Cell and Gene Therapy
In observance of World Diabetes Day, held annually on November 14, we took a look back at the past few months' news in cell and gene therapy for diabetes indications.
According to the International Diabetes Federation, 537 million people across the globe are currently living with diabetes. Notably, nearly 50% of people with diabetes are undiagnosed, and 3 in 4 people with diabetes live in low-income or middle-income nations.
An important new frontier for the management of diabetes is cell therapy and gene therapy. A number of companies and academic institutions are now pursuing the development of a range of advanced therapeutics for diabetes that fall into these categories. In honor of World Diabetes Day, observed annually on November 14 to commemorate the birthday of insulin codiscoverer Sir Frederick Banting, MD, CGTLive® is taking a look back at some of the progress that has been made for cell and gene therapy candidates in diabetes over the past year. Click the "READ MORE" buttons for more details and information about each item.
ProKidney Puts Focus on US Trial for T2D and CKD Cell Therapy Rilparencel
September 12, 2024 — ProKidney has announced a new strategic focus on its phase 3 REGEN-006 (PROACT 1) clinical trial (NCT05099770), which is evaluating rilparencel (also known as REACT), a renal autologous cell therapy, in patients in the United States who have type 2 diabetes (T2D) and chronic kidney disease (CKD).
On the other hand, the company reported that it is discontinuing the phase 3 REGEN-016 (PROACT 2) clinical trial (NCT05286853), which was evaluating rilparencel in patients with T2D and CKD in Spain. ProKidney stated that based on a comprehensive analysis it does not believe PROACT 2 is necessary to achieve regulatory approval of rilparencel in the US and as such will redirect the resources saved by discontinuing the study. Furthermore, the company determined that rilparencel may be able to receive an expedited approval in the US, pending completed results of PROACT 1, via its regenerative medicine advanced therapy (RMAT) designation, which it attained from the FDA in October 2021. In light of the reorganization of priorities, ProKidney now expects that topline findings from PROACT 1 will be available in the third quarter of 2027.
PROACT 1, which is a randomized, blinded, sham controlled study, is expected to enroll around 685 patients in total. Earlier this year, the study’s protocol was modified to focus on patients with stage 4 CKD and late stage 3b CKD with accompanying albuminuria.
Amarna Therapeutics Secures Regulatory Guidance From FDA for Diabetes Gene Therapy AM510
August 29, 2024 — Amarna Therapeutics has received regulatory guidance from the FDA regarding AM510, its investigational gene therapy product intended to treat type 1 diabetes (T1D), in the context of an Initial Targeted Engagement for Regulatory Advice (INTERACT) meeting.
Amarna noted that the meeting, which took place on July 31, 2024, provided the company with feedback on the overall development plan for AM510, which currently remains a preclinical-stage program. Furthermore, Amarna specifically noted that the discussion in particular focused on chemistry, manufacturing, and controls; the nonclinical program; and the planned clinical trial design from a safety perspective. Amarna plans to incorporate the feedback as it progresses towards submission of an investigational new drug (IND) application for the therapy; notably, it stated that it anticipates a pre-IND meeting in less than 2 years. The company additionally noted that the meeting, which was held virtually, included participation from Amarna’s teams in both Leiden and Seville, some members of the company’s T1D scientific advisory board, and regulatory consultants from Veristat.
"Our AM510 tolerance induction program for T1D just hit a major milestone with the FDA INTERACT meeting,” Henk Streefkerk, MD, PhD, the chief executive officer and medical director of Amarna Therapeutics, said in a statement. “We received extremely valuable feedback, confirming our integrated development strategy and outlining a clear regulatory path. We're eager to push forward with our preclinical work towards a successful pre-IND meeting, because we want to start treating and hopefully curing T1D patients as soon as possible."
Vertex’s Diabetes Cell Therapy on Track to Meet Endpoint of Eliminating Severe Hypoglycemic Events
June 26, 2024 — Data continue to support VX-880 (Vertex Pharmaceuticals)’s potential to eliminate or reduce the need for insulin use in patients with T1D.
Updated data from the phase 1/2 FORWARD trial (NCT04786262) were presented at the American Diabetes Association 84th Scientific Sessions, held June 21-24 in Orlando, Florida, and virtually, by investigator Piotr Witkowski, MD, PhD, Professor of Surgery, Director, Pancreatic and Islet Transplant Program, University of Chicago, Illinois.
“This positive data adds to the growing body of evidence for VX-880’s potential to revolutionize the treatment of T1D that would give patients an alternative solution other than exogenously administered insulin,” Witkowski said in a statement. “These findings will also support further evaluation of VX-880, and we hope to see this treatment become a pivotal development in T1D care.”
First Patient With Diabetic Foot Ulcers Screened for SkinTE Pivotal Trial
April 28, 2024 — The first patient with Wagner grade 1 diabetic foot ulcers (DFUs) has been screened for a phase 3 pivotal trial (NCT06140303) of SkinTE, an autologous heterogenous skin construct (AHSC) that is based on PolarityTE’s proprietary minimally polarized functional unit (MPFU) technology.
The trial is entitled "Closure Obtained with Vascularized Epithelial Regeneration for DFUs with SkinTE II," or "COVER DFUS II", and will be conducted under PolarityTE’s open IND for SkinTE.
“Every second, someone around the world develops a diabetic foot ulcer. These patients are at immediately high risk not only for amputation but also for hospitalization and even death. This is why I’m so thrilled that we are evaluating the ability of a promising therapy that might help us stem the tide of needless suffering for our patients and their families,” PolarityTE board director David Armstrong, DPM, PhD, Professor of Surgery and director, Southwestern Academic Limb Salvage Alliance (SALSA), Keck School of Medicine, said in a statement.
