Vertex Announces Program Updates for Type 1 Diabetes Portfolio
Vertex Pharmaceuticals (VRTX) has announced significant updates to its Type 1 diabetes (T1D) portfolio. The company reported that while VX-264, their encapsulated pancreatic islet cell therapy, was safe and well-tolerated, it failed to meet efficacy endpoints and will not advance further in clinical trials.
On a positive note, zimislecel (formerly VX-880), their fully differentiated islet cell therapy, is progressing well in Phase 3 trials. The pivotal trial is expected to complete enrollment and dosing in H1 2025, with global regulatory submissions planned for 2026. If approved, zimislecel could benefit approximately 60,000 people with severe T1D across the U.S. and Europe.
The company continues to pursue research-stage T1D programs, including alternative immunosuppressive regimens, gene-edited hypoimmune stem-cell derived islet cell therapies, and novel encapsulation devices.
Vertex Pharmaceuticals (VRTX) ha annunciato aggiornamenti significativi al suo portafoglio per il diabete di tipo 1 (T1D). L'azienda ha riferito che, sebbene VX-264, la loro terapia con cellule isletiche pancreatiche incapsulate, fosse sicura e ben tollerata, non ha raggiunto gli obiettivi di efficacia e non proseguirà ulteriormente negli studi clinici.
In una nota positiva, zimislecel (precedentemente VX-880), la loro terapia con cellule isletiche completamente differenziate, sta progredendo bene negli studi di fase 3. Si prevede che lo studio cruciale completi l'arruolamento e la somministrazione nella prima metà del 2025, con le presentazioni normative globali pianificate per il 2026. Se approvato, zimislecel potrebbe beneficiare circa 60.000 persone con T1D grave negli Stati Uniti e in Europa.
L'azienda continua a perseguire programmi di ricerca per il T1D, inclusi regimi immunosoppressivi alternativi, terapie con cellule isletiche derivate da cellule staminali ipoimmuni modificate geneticamente e nuovi dispositivi di incapsulamento.
Vertex Pharmaceuticals (VRTX) ha anunciado actualizaciones significativas en su cartera de diabetes tipo 1 (T1D). La compañía informó que, aunque VX-264, su terapia con células de islote pancreático encapsuladas, fue segura y bien tolerada, no logró cumplir con los objetivos de eficacia y no avanzará más en los ensayos clínicos.
En una nota positiva, zimislecel (anteriormente VX-880), su terapia con células de islote completamente diferenciadas, está avanzando bien en ensayos de fase 3. Se espera que el ensayo pivotal complete la inscripción y la dosificación en la primera mitad de 2025, con presentaciones regulatorias globales planeadas para 2026. Si se aprueba, zimislecel podría beneficiar a aproximadamente 60,000 personas con T1D severo en EE. UU. y Europa.
La compañía continúa persiguiendo programas de investigación sobre T1D, incluidos regímenes inmunosupresores alternativos, terapias con células de islote derivadas de células madre hipoinmunes editadas genéticamente y nuevos dispositivos de encapsulación.
Vertex Pharmaceuticals (VRTX)는 제1형 당뇨병(T1D) 포트폴리오에 대한 중요한 업데이트를 발표했습니다. 회사는 VX-264라는 캡슐화된 췌장 섬세포 치료제가 안전하고 잘 견뎌졌지만 효능 목표를 충족하지 못해 임상 시험에서 더 이상 진행되지 않을 것이라고 보고했습니다.
긍정적인 소식으로, zimislecel (이전의 VX-880)이라는 완전히 분화된 섬세포 치료제가 3상 시험에서 잘 진행되고 있습니다. 주요 시험은 2025년 상반기 내에 등록과 투여를 완료할 것으로 예상되며, 2026년에는 글로벌 규제 제출이 계획되어 있습니다. 승인이 된다면, zimislecel은 미국과 유럽에서 약 60,000명의 중증 T1D 환자에게 혜택을 줄 수 있습니다.
회사는 대체 면역억제 요법, 유전자 편집된 저면역 줄기세포 유래 섬세포 치료제 및 새로운 캡슐화 장치를 포함한 T1D 연구 단계 프로그램을 계속 추진하고 있습니다.
Vertex Pharmaceuticals (VRTX) a annoncé des mises à jour significatives concernant son portefeuille de diabète de type 1 (T1D). L'entreprise a rapporté que, bien que VX-264, leur thérapie par cellules îlot pancréatiques encapsulées, ait été sûre et bien tolérée, elle n'a pas atteint les objectifs d'efficacité et ne progressera pas davantage dans les essais cliniques.
Sur une note positive, zimislecel (anciennement VX-880), leur thérapie par cellules îlot entièrement différenciées, progresse bien dans les essais de phase 3. L'essai pivot devrait terminer le recrutement et la posologie au premier semestre 2025, avec des soumissions réglementaires mondiales prévues pour 2026. Si approuvé, zimislecel pourrait bénéficier à environ 60 000 personnes atteintes de T1D sévère aux États-Unis et en Europe.
L'entreprise continue de poursuivre des programmes de recherche sur le T1D, y compris des régimes immunosuppresseurs alternatifs, des thérapies par cellules îlot dérivées de cellules souches hypo-immunes modifiées génétiquement et de nouveaux dispositifs d'encapsulation.
Vertex Pharmaceuticals (VRTX) hat bedeutende Aktualisierungen seines Portfolios für Typ-1-Diabetes (T1D) angekündigt. Das Unternehmen berichtete, dass VX-264, ihre verkapselte Therapie mit Inselzellen der Bauchspeicheldrüse, zwar sicher und gut verträglich war, jedoch nicht die Wirksamkeitsziele erreicht hat und nicht weiter in klinischen Studien voranschreiten wird.
Positiv ist zu vermerken, dass zimislecel (ehemals VX-880), ihre vollständig differenzierte Therapie mit Inselzellen, gut in Phase-3-Studien vorankommt. Die entscheidende Studie soll die Rekrutierung und Dosierung in der ersten Hälfte von 2025 abschließen, mit globalen regulatorischen Einreichungen, die für 2026 geplant sind. Wenn genehmigt, könnte zimislecel etwa 60.000 Menschen mit schwerem T1D in den USA und Europa zugutekommen.
Das Unternehmen verfolgt weiterhin Forschungsprogramme zu T1D, einschließlich alternativer immunsuppressiver Regime, genetisch bearbeiteter hypoimmuner Stammzell-abgeleiteter Inselzelltherapien und neuartiger Verkapselungsgeräte.
- Zimislecel Phase 3 trial on track for completion of enrollment in H1 2025
- Potential market of 60,000 patients for zimislecel in US and Europe
- Multiple regulatory fast-track designations received for zimislecel
- VX-264 failed to meet efficacy endpoints and discontinued from further development
- No immediate revenue potential from VX-264 program
Insights
Vertex's T1D portfolio update presents a mixed clinical picture with significant implications for their diabetes pipeline strategy. The discontinuation of VX-264 (cells + device approach) represents a setback in their quest to develop an immunoprotection solution that would eliminate the need for immunosuppression. Despite being safe, VX-264 simply didn't produce sufficient C-peptide levels to demonstrate meaningful efficacy - a critical threshold for any diabetes cell therapy.
More encouragingly, zimislecel (formerly VX-880) continues advancing through pivotal trials with global regulatory submissions targeted for 2026. The therapy's multiple regulatory accelerations (RMAT, Fast Track, PRIME designations) reflect its potential significance. However, it's worth understanding that zimislecel requires standard immunosuppression, which meaningfully restricts its use to patients with severe disease where benefit/risk is most favorable.
Vertex's continued investment in alternative approaches - particularly gene-edited hypoimmune cells and novel encapsulation devices - demonstrates their commitment to addressing T1D's underlying biology through multiple mechanisms. The company is strategically focusing on severe hypoglycemic events (SHEs) with impaired awareness of hypoglycemia as their initial target population - an appropriate entry point where unmet need justifies immunosuppression.
Vertex's update contains both a pipeline setback and confirmation of continued advancement for their lead diabetes asset. The VX-264 discontinuation removes one potential value driver from Vertex's diabetes portfolio, but the company's decisive action to halt development preserves R&D capital for more promising opportunities.
The estimated 60,000-patient addressable market for zimislecel provides meaningful commercial potential. Cell therapies typically command premium pricing, suggesting zimislecel could become a significant revenue contributor if approved. Vertex's proactive investment in manufacturing and commercial infrastructure signals confidence in zimislecel's regulatory prospects.
Vertex's diabetes portfolio strategy follows a logical progression - first establishing clinical proof-of-concept with zimislecel (requiring immunosuppression), while pursuing research on next-generation approaches that might eliminate immunosuppression requirements. While the device approach setback extends the timeline for achieving an immunosuppression-free solution, Vertex maintains multiple alternative approaches.
The confirmation that zimislecel remains on track for H1 2025 enrollment completion and 2026 regulatory submissions provides timeline clarity for investors. Diabetes represents a diversification opportunity beyond Vertex's cystic fibrosis franchise, and despite this setback, their lead candidate continues advancing on schedule.
- VX-264 Phase 1/2 enrollment and dosing complete in Parts A and B: VX-264 was generally safe and well tolerated; efficacy data are not supportive of further clinical advancement -
- Zimislecel (VX-880) pivotal trial on track to complete enrollment and dosing in H1 2025; Vertex expects to submit marketing applications to global regulators in 2026 -
- Continue to progress multiple novel, research-stage immunoprotective approaches -
VX-264 Update
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. Vertex plans to conduct further analyses, including of explanted devices, to better understand these findings.
Zimislecel Update
Zimislecel (formerly VX-880), Vertex’s investigational fully differentiated islet cell therapy with standard immunosuppression, is in the Phase 3 portion of the Phase 1/2/3 study in patients with T1D with severe hypoglycemic events (SHEs) and impaired awareness of hypoglycemia. This pivotal trial is well underway and on track to complete enrollment and dosing in the first half of 2025, setting up global regulatory submissions in 2026. Zimislecel has previously been granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the
T1D Research Update
Vertex is also pursuing research-stage T1D programs to evaluate additional approaches that could provide transformative benefit to people with T1D and reduce or eliminate the need for standard immunosuppressive regimens. These approaches include alternative immunosuppressive regimens, gene-edited hypoimmune stem-cell derived islet cell therapies, and novel devices to encapsulate islet cells.
“We’d like to thank the patients, physicians and T1D community who participated in the VX-264 study. Today’s data show that more work needs to be done to advance the ‘cells plus device’ program, and we are committed to doing so,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex Pharmaceuticals. “Equally, we are very pleased with the rapid progress of our zimislecel program, which is on track to complete enrollment and dosing in the Phase 3 study this summer, positioning us for global regulatory submissions in 2026. We’re excited for the opportunity to bring the promise of zimislecel to patients as quickly as possible.”
About Type 1 Diabetes
T1D results from the autoimmune destruction of insulin-producing beta cells in pancreatic islets. Insulin deficiency results in hyperglycemia and can lead to acute life-threatening complications such as diabetic ketoacidosis.
People with T1D are reliant on lifelong treatment with exogenous insulin that requires careful monitoring of blood glucose levels. Even with the availability of advanced exogenous insulin delivery and glucose monitoring systems, people with T1D can have periods of very low and very high blood sugar levels. Exogenous insulin has a narrow therapeutic range and carries an inherent risk of causing low blood sugar levels or hypoglycemic events, which can potentially result in arrhythmias, seizures, coma and even death. Due to the limitations and complexities of exogenous insulin treatment, it can be difficult for people with T1D to achieve and maintain good glucose control. Exposure to prolonged periods of high blood glucose levels, or hyperglycemia, can lead to long-term complications such as nerve damage, kidney disease/failure, eye disease (including vision loss), cardiovascular disease, stroke and even death.
HbA1c is a measure of average blood glucose over the most recent ~2-3 months, and the consensus guidance is to maintain an HbA1c of <
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1.
Vertex was founded in 1989 and has its global headquarters in
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Carmen Bozic, M.D., and statements regarding Vertex’s plans and expectations for the clinical trial evaluating VX-264, including plans to conduct further analyses, expectations for the clinical trial evaluating zimislecel, including expectations for the trial to complete enrollment and dosing in the first half of 2025 and expectations for global regulatory submissions in 2026, plans to invest in manufacturing and commercial capabilities to ensure zimislecel launch readiness, expectations for the patient population that could benefit from zimislecel, and plans to progress multiple novel, research-stage immunoprotective approaches. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's research and development programs may not support development or registration of its compounds due to safety, efficacy or other reasons, that clinical trial data might not be available on the expected timeline, and other risks listed under Risk Factors in Vertex's most recent annual report filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
(VRTX-GEN)
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