zimislecel (VX-880) / Vertex - LARVOL DELTA

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) - "Available on Friday, June 13, 2025 at 08:00am CDT."

Clinical • Diabetes • Metabolic Disorders • 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.'"

Audio

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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First patient gains insulin independence with novel stem cell-derived islet cell therapy (Healio) - "'This is the first-in-the-world administration of stem cell-derived islets in this manner, where they are infused into the liver,' James F. Markmann, MD, PhD...said...'The team with Vertex is working with the FDA to address the issues that were brought up regarding having enough justification to do dose escalation,' Markmann said...'That is in progress, and we hope it will be resolved quickly.'"

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T1D Patient Achieves Full Insulin Independence After Stem Cell Transplant - But clinical trial currently under FDA hold as dosing is discussed (MedPageToday) - "Following a 40-year history of T1D riddled with complications, a 64-year-old man received a single infusion with the investigational treatment VX-880 at a half target dose, said James F. Markmann, MD, PhD...during the American Diabetes Association (ADA) annual meeting."

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Stem Cell–Derived, Fully Differentiated Islet Cells for Type 1 Diabetes (ADA 2022) - "There was 1 serious AE of rash (mild, unrelated to VX-880), which resolved.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

"En el día 270, tras recibir la mitad de la dosis objetivo de VX-880, el paciente 1 es independiente de la insulina con una HbA1c del 5,2% y una TIR del 99,9% #ADA2022 #vertex" (@BeyondType2ESP)

Metabolic Disorders

"At Day 270, after receiving half the target dose of VX-880, Patient 1 is insulin independent with HbA1c of 5.2% and TIR 99.9% #ADA2022 #vertex" (@ADA_DiabetesPro)

Clinical • Diabetes • Metabolic Disorders

"@VertexPharma VX-880 Hello, Is there a roadmap? how long does it take for the second phase? Is it possible to speed up the process? can someone explain clearly on youtube or any other online channels? Thanks" (@ozgurablak)