CGTLive's 2024 Pillars of Progress: Top News in Endocrinology/Dermatology

For all of 2024, our team was following the clinical development of targeted and novel engineered approaches to the treatment of patients with various medical disorders. These efforts included holding in-depth conversations with experts in the clinical care of these individuals, as well as in cell and gene therapy development, culminating in our coverage of each step of progress that the most exciting cellular and genetic treatments have made along the pipeline.

From major data publications and presentations to FDA decisions and medical meetings, the team spent all year bringing the latest information to the website's front page.

Among our areas of focus in 2024 were endocrinology and dermatology. The major news items appeared among the top pieces our team produced—but sometimes smaller stories reach those heights as well because of their clinical impact, their inventive mechanisms, or otherwise. Whatever the reason for the attention these stories got, their place here helps provide an understanding of the themes in endocrinology and dermatology over the course of 2024.

Here, we'll highlight some of the most-read content on CGTLive's endocrinology and dermatology pages this year. Click the buttons to read further into these stories.

Vertex’s Diabetes Cell Therapy on Track to Meet Endpoint of Eliminating Severe Hypoglycemic Events

“This positive data adds to the growing body of evidence for VX-880’s potential to revolutionize the treatment of type 1 diabetes that would give patients an alternative solution other than exogenously administered insulin. These findings will also support further evaluation of VX-880, and we hope to see this treatment become a pivotal development in type 1 diabetes care.”
—Piotr Witkowski, MD, PhD

Data continued to support VX-880 (Vertex Pharmaceuticals)’s potential to eliminate or reduce the need for insulin use in patients with type 1 diabetes (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.

Vertex Pauses Diabetes Cell Therapy Trial After Unrelated Patient Deaths

In January, Vertex Pharmaceuticals placed its diabetes type 1 cell therapy program on a protocol-specified pause following 2 patient deaths that occurred in the phase 1/2 clinical trial (NCT04786262). Vertex has stated that the deaths were unrelated to VX-880 therapy.

ProKidney Puts Focus on US Trial for T2D and CKD Cell Therapy Rilparencel

“We decided to prioritize PROACT 1 to accelerate potential US registration and commercial launch. We are confident that this strategic shift in our phase 3 program is the most expeditious and resource efficient approach to bring rilparencel to market in the US, our highest priority market. The recent positive REGEN-007 interim data update in June further supports the urgency to bring our innovation to patients with advanced CKD. We look forward to continuing our engagement with the FDA, under the RMAT designation, to bring rilparencel to market.”
—Bruce Culleton, MD

In September, ProKidney 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 would be discontinuing the phase 3 REGEN-016 (PROACT 2) clinical trial (NCT05286853), which was evaluating rilparencel in patients with T2D and CKD in Spain.

FDA Issues CRL for Abeona's Epidermolysis Bullosa Gene Therapy Pz-Cel

“While we are surprised and disappointed by this CRL, we are committed to providing the CMC information necessary to respond to the agency’s asks, with the goal of bringing pz-cel to patients with RDEB as quickly as possible. We are already hard at work generating the additional CMC information, and we expect that all of FDA’s requests will be addressable in a reasonable timeframe. We anticipate completing the BLA resubmission in the third quarter of 2024 with necessary updates to fully satisfy all the deficiencies outlined in the CRL.”
—Vish Seshadri

In April, the FDA issued a complete response letter (CRL) for the biologics license application (BLA) for prademagene zamikeracel (pz-cel; EB-101), an investigational autologous gene-corrected epidermal sheet therapy for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB), according to an announcement from Abeona Therapeutics, the therapy’s developer. Via the CRL, the FDA requested more chemistry, manufacturing, and controls (CMC) information, specifically with regard to validation requirements for specific manufacturing and release testing methods.

Kadimastem and iTolerance Work to Bring Immunosuppression-Free Diabetes Cell Therapy to Trials

“The INTERACT meeting is an important step forward for this development program. We are dedicated to advancing this program and providing diabetic patients with a potential solution to live without the need for insulin injections."
—Asaf Shiloni

In late 2023, Kadimastem and iTolerance requested an INTERACT meeting with the FDA to discuss their preclinical joint cell therapy in development for the potential treatment of diabetes, iTOL-102. Kadimastem and iTolerance previously signed a collaboration agreement in May 2023 for the development of a diabetes cell therapy that would not require chronic immunosuppression and received a grant of about 1 million dollars from the Binational Israel-U.S. Industrial R&D (BIRD) Foundation to pursue the research.