zimislecel (VX-880) / Vertex - LARVOL DELTA

Durable glycaemic control and elimination of exogenous insulin use with VX-880 in patients with type 1 diabetes: VX-880-101 (FORWARD) (EASD 2025) - P3 | "These durable clinical benefits of VX-880 with elimination of SHEs, improved glycemic control, and freedom from exogenous insulin support the curative potential of VX-880, the first and only allogeneic, stem-cell derived, islet cell therapy in pivotal development."

Clinical • Diabetes • Hypoglycemia • Metabolic Disorders • Type 1 Diabetes Mellitus

Achievement of target CGM metrics in people with type 1 diabetes treated with zimislecel (VX-880) in the FORWARD study (EASD 2025) - P3 | "A single infusion of zimislecel resulted in reductions or elimination of insulin use, reduced HbA1c, and improved CGM metrics to levels above ADA/EASD endorsed targets for time-in, -below and -above range. These data demonstrate the transformative potential of zimislecel for people with T1D experiencing SHEs."

Diabetes • Hypoglycemia • Metabolic Disorders • Type 1 Diabetes Mellitus

Stem Cell-Derived Islets FORWARD VX-880 Trial Update (ADA 2025) - No abstract available

Metabolic Disorders

Durable Glycemic Control and Elimination of Exogenous Insulin Use with VX-880 in Patients with Type 1 Diabetes (T1D)—VX-880-101 (FORWARD) (ADA 2025) - "These durable clinical benefits of VX-880 with elimination of SHEs, improved glycemic control, and freedom from exogenous insulin support the curative potential of VX-880, the first and only allogeneic, stem-cell derived, islet cell therapy in pivotal development."

Clinical • Diabetes • Hypoglycemia • Metabolic Disorders • Type 1 Diabetes Mellitus

Stem Cell-Derived, Fully Differentiated Islets for Type 1 Diabetes. (PubMed, N Engl J Med) - P3 | "The results of this small, short-term study involving persons with type 1 diabetes support the hypothesis that zimislecel can restore physiologic islet function, warranting further clinical investigation. (Funded by Vertex Pharmaceuticals; VX-880-101 FORWARD ClinicalTrials.gov number, NCT04786262.)."

Journal • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Dementia • Diabetes • Hematological Disorders • Hypoglycemia • Infectious Disease • Metabolic Disorders • Neutropenia • Type 1 Diabetes Mellitus

Vertex Presents Positive Data for Zimislecel in Type 1 Diabetes at the American Diabetes Association 85th Scientific Sessions (Vertex Press Release) - P1/2/3 | N=52 | FORWARD-101 (NCT04786262) | Sponsor: Vertex Pharmaceuticals Incorporated | " Vertex Pharmaceuticals Inc...announced simultaneous presentation and publication of updated data from the Phase 1/2 portion of the Phase 1/2/3 FORWARD-101 clinical trial...The data are from 12 patients who received the full dose of zimislecel as a single infusion and were followed for at least one year, as of October 2024. Results from the study to date continue to demonstrate the transformative potential of zimislecel with consistent and durable patient benefit with longer follow-up. All 12 participants: Demonstrated engraftment with glucose-responsive endogenous C-peptide production, which was durable through one year of follow-up. Achieved the ADA targets of HbA1c 70%. Were free of SHEs from day 90 onwards. Had a reduction in exogenous insulin use (mean reduction in daily insulin dose: 92%). 10/12 (83%) no longer required exogenous insulin at Month 12."

P1/2 data • Type 1 Diabetes Mellitus

SR-02, a Novel Islet Replacement Therapy for Insulin-Requiring Diabetes (ASGCT 2025) - P1/2 | "Allogeneic cadaveric islets infused to the portal hepatic circulation with concurrent immune suppression (Lantidra) and an experimental, embryonic stem cell-derived product (Zimislecel), also infused to the portal circulation with immune suppression, have both resulted in insulin independence in patients of type 1 diabetes...Omental administration and elimination of de novo systemic immune suppression therapy will make islet replacement therapy widely accessible to millions of patients in need. Disease Focus of Abstract:Diabetes"

Diabetes • Metabolic Disorders • Solid Organ Transplantation • Type 1 Diabetes Mellitus

An Efficacy, Safety, and Tolerability Study of VX-880 in Participants With Type 1 Diabetes With a Kidney Transplant (clinicaltrials.gov) - P3 | N=10 | Recruiting | Sponsor: Vertex Pharmaceuticals Incorporated | Phase classification: P2 ➔ P3 | Trial completion date: Sep 2030 ➔ Sep 2027

Phase classification • Trial completion date • Diabetes • Diabetic Nephropathy • Metabolic Disorders • Transplantation • Type 1 Diabetes Mellitus

IMPROVEMENT IN GLYCEMIC CONTROL AND ELIMINATION OF EXOGENOUS INSULIN USE IN PATIENTS WITH TYPE 1 DIABETES INFUSED WITH FULLY DIFFERENTIATED ISLET CELLS: TRIAL VX-880-101 (FORWARD) (ATTD 2025) - "VX-880 safety was consistent with immunosuppressive regimen, islet infusion procedure, and long-standing T1D. Conclusions VX-880 has curative potential in T1D with elimination of severe hypoglycemia, significantly improved glycemic control, and freedom from exogenous insulin use."

Clinical • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Diabetes • Infectious Disease • Metabolic Disorders • Otorhinolaryngology • Severe Hypoglycemia • Type 1 Diabetes Mellitus • Vascular Neurology

An Efficacy, Safety, and Tolerability Study of VX-880 in Participants With Type 1 Diabetes With a Kidney Transplant (clinicaltrials.gov) - P2 | N=10 | Recruiting | Sponsor: Vertex Pharmaceuticals Incorporated | Not yet recruiting ➔ Recruiting

Enrollment open • Diabetes • Diabetic Nephropathy • Metabolic Disorders • Transplantation • Type 1 Diabetes Mellitus

An Efficacy, Safety, and Tolerability Study of VX-880 in Participants With Type 1 Diabetes With a Kidney Transplant (clinicaltrials.gov) - P2 | N=10 | Not yet recruiting | Sponsor: Vertex Pharmaceuticals Incorporated

New P2 trial • Diabetes • Diabetic Nephropathy • Metabolic Disorders • Transplantation • Type 1 Diabetes Mellitus

A Safety, Tolerability, and Efficacy Study of VX-880 in Participants With Type 1 Diabetes (clinicaltrials.gov) - P3 | N=52 | Recruiting | Sponsor: Vertex Pharmaceuticals Incorporated | Phase classification: P1/2 ➔ P3 | N=37 ➔ 52 | Trial completion date: Feb 2030 ➔ Jun 2030 | Trial primary completion date: Feb 2026 ➔ Jun 2027

Enrollment change • Phase classification • Trial completion date • Trial primary completion date • Diabetes • Metabolic Disorders • Severe Hypoglycemia • Type 1 Diabetes Mellitus

Improvement in glycaemic control and elimination of exogenous insulin use in patients with type 1 diabetes infused with fully differentiated islet cells (VX-880) (EASD 2024) - P1/2 | "These results demonstrate that a single infusion of VX-880, an allogeneic stem cell-derived, fully differentiated islet cell therapy, has the potential to eliminate severe hypoglycemia, significantly improve glycemic control, and achieve freedom from use of exogenous insulin. Data support the curative potential of VX-880 in people with T1D."

Clinical • Late-breaking abstract • Diabetes • Metabolic Disorders • Severe Hypoglycemia • Type 1 Diabetes Mellitus

A Safety, Tolerability, and Efficacy Study of VX-880 in Participants With Type 1 Diabetes (clinicaltrials.gov) - P1/2 | N=37 | Recruiting | Sponsor: Vertex Pharmaceuticals Incorporated | N=17 ➔ 37 | Trial completion date: Jan 2028 ➔ Feb 2030 | Trial primary completion date: Jan 2024 ➔ Feb 2026

Enrollment change • Trial completion date • Trial primary completion date • Diabetes • Metabolic Disorders • Severe Hypoglycemia • Type 1 Diabetes Mellitus

Targeting mRNA Modifications to counter type 1 diabetes (ATTD 2024) - "5) Vertex Announces Positive Day 90 Data for the First Patient in the Phase 1/2 Clinical Trial Dosed With VX-880, a Novel Investigational Stem Cell-Derived Therapy for the Treatment of Type 1 Diabetes...7) Reardon, S. First pig-to-human heart transplant: what can scientists learn? Nature 601, 305–306 (2022)."

Diabetes • Metabolic Disorders • Transplantation • Type 1 Diabetes Mellitus

Glucose-Dependent Insulin Production and Insulin-Independence in Type 1 Diabetes from Stem Cell-Derived, Fully Differentiated Islet Cells: Updated Data from the VX-880 Clinical Study (IPITA-IXA-CTRMS 2023) - " These results demonstrate that a single infusion of stem cell-derived islets (VX-880) can restore insulin production and glucose regulation, leading to insulin independence in patients with T1D. Based on this, VX-880 has the potential to be a functional cure for patients with T1D. Part B is fully enrolled and multiple additional patients have been dosed with the target dose."

Clinical • Diabetes • Metabolic Disorders • Severe Hypoglycemia • Type 1 Diabetes Mellitus

Glucose-dependent insulin production and insulin-independence in patients with type 1 diabetes infused with stem cell-derived, fully differentiated islet cells (VX-880) (EASD 2023) - P1/2 | "These results demonstrate that a single infusion of stem cell-derived islets (VX-880) can restore insulin production and glucose regulation, leading to insulin independence in patients with T1D. Based on this, VX-880 has the potential to be a functional cure for patients with T1D. Part B is fully enrolled and multiple additional patients have been dosed with the target dose."

Clinical • Diabetes • Metabolic Disorders • Severe Hypoglycemia • Type 1 Diabetes Mellitus

Glucose-Dependent Insulin Production and Insulin-Independence in Type 1 Diabetes from Stem Cell–Derived, Fully Differentiated Islet Cells—Updated Data from the VX-880 Clinical Trial (ADA 2023) - "Longer-term data on both patients in Part A and new data on patients who received the full (target) dose in Part B will be provided in the presentation. These results are the first from a clinical trial of allogeneic, fully differentiated, insulin producing, stem cell-derived islets which has demonstrated the potential to restore insulin production and glycemic control and provide insulin independence in patients with T1D."

Clinical • Diabetes • Metabolic Disorders • Severe Hypoglycemia • Type 1 Diabetes Mellitus

Glucose-Dependent Insulin Production and Insulin-Independence in Type 1 Diabetes from Stem Cell-Derived, Fully Differentiated Islet Cells—Updated Data from the VX-880 Clinical Trial (ADA 2023) - No abstract available

Clinical • Diabetes • Metabolic Disorders • Type 1 Diabetes Mellitus

"Trevor Reichman @VertexPharma #ADA2023 VX-880 clinical trial update," (@ERobertson02)

Clinical • Metabolic Disorders

STEM CELL-DERIVED, FULLY DIFFERENTIATED ISLET CELLS FOR TYPE 1 DIABETES (ATTD 2023) - "These unprecedented results, at half target dose,are the first evidence stem cell-derived islets can restore insulinproduction and glycemic control in patients with T1D."

Diabetes • Metabolic Disorders • Severe Hypoglycemia • Type 1 Diabetes Mellitus

Type 1 Diabetes Patient Achieves Insulin Independence (Diabetes Self Manag) - "According to lead study author James F. Markmann, MD...'His life was being destroyed by diabetes. He couldn't work, he crashed his motorcycle from lows and really was tremendously appreciative that he could participate.'"

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Stem cell-derived, fully differentiated islet cells for type 1 diabetes (EASD 2022) - P1/2 | "We report the first patient administered VX-880, an investigational allogeneic stem cell-derived, fully differentiated, pancreatic islet cell replacement therapy... These unprecedented results are the first evidence that stem cell-derived islets can restore insulin production and glucose control in T1D. The study continues to enroll and dose patients."

Diabetes • Hypoglycemia • Metabolic Disorders • Type 1 Diabetes Mellitus

"💡فيرتيكس @VertexPharma تلقّت✅الموافقة من هيئة الغذاء والدواء @US_FDA لاستكمال التجارب الإكلينيكية المرحلة1️⃣لعلاج السكري النوع الأول باستخدام خلايا بيتا البنكرياس الجذعية💉VX-880 سيتم تجربة الجرعة الكاملة على5أشخاص ثم التوسع بالعدد لدخول المرحلة2️⃣ https://t.co/JX3kOhLnDC (٩)" (@YAYshamsaldeen)

Vertex Releases New Data on Potential Cure for Type 1 Diabetes (diaTribe) - "Data from the first two participants in the trial were presented at the conference by Dr. James Markmann...who has over 20 years of experience in kidney, pancreas, and pancreatic islet transplantation....'We use a standard regimen of immunosuppressants, also used in patients receiving kidney transplants, an established regimen we’ve found to be generally well-tolerated,' Markmann said."

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