Type 1 diabetes cures and prevention competitive landscape  – March 19, 2026

Program discontinued in March 2025, following Part B Day-90 results, which showed that while the device was safe and well tolerated, but failed to deliver meaningful increases in C-peptide production.

Part A of the trial completed and Part B initiated in 4Q23; FDA cleared IND in March 2023; trial in Canada ongoing and US trial expected to initiate in 1H23.

All 12 participants in the phase 1/2 FORWARD trialdemonstrated engraftment, with 10 achieving insulin independence and >90% time in range (TIR) shown ADA 2025; Regulatory submission for approval is expected in 2026 as of 1Q25; VX-880 named zimislecel at JPM 2025; Zimislecel advanced into a pivotal phase 1/2/3 trial(n=52) in 3Q24; positive phase 1/2 results also highlighted at DUK 2025, ATTD 2025, and EASD 2024.

Resumed following a protocol-specified pause announced in January 2024 after two unrelated patient deaths; Vertex has completed enrollment and dosing in Part B of the Phase 1/2 study of VX-880 and expects to present data at ADA 2023 in June; Vertex intends to begin Part C of the study with concurrent dosing; granted EMA designation in March 2023; Vertex acquires ViaCyte for $320 million in July 2022; FDA lifts clinical hold on phase 1/2 trial in July 2022; New CGM data for first two patients shared at ADA 2022, reinforcing extremely positive data for first patient; Positive data for first two patients released May 2022alongside announcement of FDA placing clinical hold on phase 1/2 trial; Preliminary data from phase 2 study released in October 2021 shows extremely promising results; Naked cell phase 1/2 launch and FDA Fast Track Designation received in March 2021; Naked cell program IND approved January 2021; Vertex on track to submit IND filing for islet cell transplant program in “late 2020,” phase 2 to commence in 2021 as of 3Q20 update; Cell therapy to move into clinic by late 2020/early 2021, as of JPM 2020; Semma acquired by Vertex Pharmaceuticals for $950 million in September 2019; Published in Nature in 2019 detailing improved beta cell generation/purification process; Semma Therapeutics founded in April 2015 to translate Dr. Doug Melton’s beta cell generation procedure into therapy.

Announced positive one-year results of CELZ-201 in people with late-stage T2D in February 2025; Announced that partner, Greenstone Biosciences, has successfully developed a human induced pluripotent stem cell (iPSC) pipeline for the ImmCelz platform in May 2023; Announced positive topline results for cell therapy program in T2D in April 2023;

Announced IRB approval of phase 1/2 trial of CELZ-201 as a treatment for recent onset T1D in February 2023; expected to initiate in 1Q23.

Confirmed to still be in clinical testing as of Jan 2025.

VCTX-211 continues to run in collaboration with CRISPR in a phase 1/2 study, expected to complete in August 2025.

In 2006, ViaCyte published the first study demonstrating successful in vitro differentiation of human embroyonic stem cells into pancreatic tissue; In 2014 BetaLogics (now part of ViaCyte) published the first paper on using in vitro stem-cell derived beta cells to reverse diabetes in mice; ViaCyte is currently in phase 1/2 clinical studies with its stem cell-derived insulin producing cells.

In July 2025, Sernova announced an agreement with Eledon Pharmaceuticals to evaluate Eledon’s immunosuppressive agent tegoprubart (AT-1501), an investigational anti-CD40L antibody, in Sernova’s ongoing phase 1/2 clinical trial. Tegoprubart would be used in place of tacrolimus, a standard immunosuppressivedrug used in organ transplantation to prevent rejection that has notable side effects and potential toxicity toward insulin-producing beta cells.

As of September 2024, a phase 1/2 trial of the Cell Pouch in combination with allogeneic islets in T1D showed that all six participants in Cohort A achieved sustained insulin independence.

IND filing for SC451 is planned for 2026.

An investigator-sponsored trial using HIP-modified cells (a precursor to SC451) is expected to begin in late 2025.

Additional mouse model data released March 2024 showed durable glycemic control for up to 15 months with no safety concerns.

Preclinical data published in Nature Biotechnology in May 2023 showing survival of hypoimmune pluripotent stem cell-derived islets in non-human primate models for 40 weeks.

Preclinical data published in April 2023, showing that hypoimmune human islet cells, engineered with CRISPR, successfully evaded immune rejection and normalized glucose levels in immunocompetent type 1 diabetes mouse models; an investigator-sponsored trial testing these cells in patients with T1D is expected to initiate later this year; aims to file IND in 2024 for SC451, hypoimmune stem cell-derived islet cell therapy.

Approved

Phase 4 for children <8 years

Phase 3 extension trial

Phase 4 PETITE-T1D (n=20) for children under eight years and phase 3 PROTECT Extension trial (n=188) in people with recently diagnosed T1D ongoing, with expected completion in 2026.

EU and China regulatory decisions expected 2H25, as of 1Q25.

Teplizumab approved as Tzield in November 2022with dedicated conference calladdressing patient assistance; Provention and Sanofi enter co-promotion agreement in October 2022; Provention hosts investor event in May 2022; phase 3 PROTECT trialin newly diagnosed T1D to complete in May 2023; Teplizumab’s original PDUFA date of August 17. 2022 was extended to November 17, 2022; Provention received a CRL from the FDA in July 2021; In January 2021ProventionBio filed the BLA with the FDA for type 1 diabetes delay, under the company’s requested Priority Review, MAA filing in Europe slated for 2021, BLA submission completed to FDA with AdComm expected as of 3Q20 update; Follow-up data showing sustained delay of three years presented at ADA 2020; FDA Breakthrough Therapy Designation in August 2019; First therapy shown to delay type 1 diagnosis (ADA 2019); Phase 3 PROTECT study underway as of April 2019, following acquisition of candidate from MacroGeneics.

Dr. Danijela Tatovic (Cardiff University, UK) presented an overview of the SMART STUDY of combining IL-2 and abatacept at DUK 2025.

Phase 2 trial was completed in 2022; preliminary results released in 2020 showed preservation of C-peptide levels and improvements in insulin sensitivity.

Verapamil has shown beta cell preservation in adults and children with new-onset T1D. VER-A-T1D completed in April 2025; additional trials like MELD-ATG and WAVE T1D testing it alone and in combination (e.g., with ATG, teplizumab). At ADA 2025, poster 1849-P reported verapamil inhibits vascular adhesion molecules in islets. Preclinical comparisons show newer TXNIP inhibitors like TIX100 may be more potent.

CLVer trial results, presented at ATTD 2023 and published in JAMA, show that stimulated C-peptide levels in the verapamil group (n=47) were 30% higher compared to placebo (n=41) at 52 weeks (p=0.04), showing that verapamil had partially preserved beta cell function.

A phase 2 study published in Nature Medicine in 2018 reported that once daily oral verapamil promoted endogenous beta cell function while reducing insulin requirements and hypoglycemia in adults with recent-onset T1D; C-peptide levels in the verapamil group (n=11) were 35% higher compared to placebo (n=13) after 12 months.

Verapamil was first approved in 1981, and is indicated for hypertension, chronic stable angina, unstable angina, supraventricular tachycardia, and paroxysmal supraventricular tachycardia.

Full 52-week results of COVALENT-111 trial expected in 2H25; topline results announced in December 2024 following data released at ATTD 2024 and WCIRDC 2023.

Phase 2 COVALENT-112 trial in T1D open-label results expected 2H25; Preliminary data from first two patients announced in April 2024shows improvement in C-peptide levels after four weeks of dosing; FDA clearance of IND application for BMF-219 in T1D announced in October 2023.

In July 2025, announced positive feedback from the FDA following a pre-IND meeting, with plans to apply for Fast Track Breakthrough or RMAT designation within the IND filing . The study may also be considered registrational, potentially expediting U.S. approval. 

In September 2024, announced positive results showing clinical remission and insulin independence in some patients for up to 12 years; Phase 2 trials to launch in 2H24.