VX-264 / Vertex - LARVOL DELTA

VX-264 Update (Businesswire) - "Vertex has completed enrollment and dosing in Parts A and B of the Phase 1/2 VX-264 (cells + device) study and the planned analysis at Day 90 for Part B. In Part B of the study, participants received the full dose of the investigational fully differentiated pancreatic islet cell therapy encapsulated in a proprietary immunoprotective device. There were two primary endpoints in Part B, safety and change in peak C-peptide during a mixed-meal tolerance test (MMTT) from baseline at Day 90. VX-264 was generally safe and well tolerated; however, the study did not meet the efficacy endpoint. Increases in C-peptide, a marker of insulin production, were not observed at levels necessary to deliver benefit. Therefore, VX-264 will not be advancing further in clinical trials."

Discontinued • Trial status • Type 1 Diabetes Mellitus

A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes (clinicaltrials.gov) - P1/2 | N=17 | Recruiting | Sponsor: Vertex Pharmaceuticals Incorporated

Trial completion date • Trial primary completion date • Diabetes • Metabolic Disorders • Type 1 Diabetes Mellitus

A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes (clinicaltrials.gov) - P1/2 | N=17 | Recruiting | Sponsor: Vertex Pharmaceuticals Incorporated | Not yet recruiting ➔ Recruiting

Enrollment open • Diabetes • Metabolic Disorders • Type 1 Diabetes Mellitus

A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes (clinicaltrials.gov) - P1/2 | N=17 | Not yet recruiting | Sponsor: Vertex Pharmaceuticals Incorporated

New P1/2 trial • Diabetes • Metabolic Disorders • Type 1 Diabetes Mellitus

"في خبر مفرح وأمل جديد لمرضى السكري أعلنت شركة @VertexPharma الدوائية عن موافقة هيئة الغذاء والدواء الأمريكية (FDA) و وكالة الصحة الكندية على البدء في التجارب السريرية لعلاجها التجريبي المحتمل لمرض السكر من النوع الأول VX-264 وهو خلايا جذعية مغلفة تفرز https://t.co/X27nFzwHAf" (@maralazemi)

"وافقت هيئة الغذاء والدواء الامريكية على استخدام VX-264 في الدراسات السريرية وهو عبارة عن خلايا جذعية بداخل كبسولة تحفز على افراز #الانسولين لمرضى #السكري من النوع الاول . #vertex خبر رائع جداً 👌🏼 @US_FDA @VertexPharma https://t.co/vjKsGnrCA7" (@NohaAshy)

Vertex Announces FDA Clearance of Investigational New Drug Application for VX-264, a Novel Encapsulated Cell Therapy for the Treatment of Type 1 Diabetes (Businesswire) - "Vertex Pharmaceuticals...today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-264, a stem cell-derived, fully differentiated pancreatic islet cell therapy encapsulated into a Vertex-developed, immunoprotective device with the potential to treat type 1 diabetes (T1D). The VX-264 program does not require the use of immunosuppression, which may broaden the population of people with T1D that this investigational therapy could reach. Vertex plans to initiate a Phase 1/2 clinical trial in the first half of 2023 to study the safety, tolerability and efficacy of VX-264 in patients with T1D. The company previously received approval from Health Canada on the Clinical Trial Application (CTA) for VX-264, and the Phase 1/2 trial is ongoing in Canada."

IND • New P1/2 trial • Endocrine Disorders • Type 1 Diabetes Mellitus