Vertex Pharmaceuticals (VRTX) Conference Summary Company Overview - **Company**: Vertex Pharmaceuticals - **Event**: Conference at the American Diabetes Association 85th Scientific Sessions - **Date**: June 20, 2025 Key Industry and Company Insights Type 1 Diabetes (T1D) Landscape - T1D is caused by the destruction of insulin-producing beta cells in the pancreas, leading to a lifelong dependency on insulin therapy, which has not significantly changed since 1921 [7][9] - Approximately 4 million people are diagnosed with T1D in North America and Europe, with an estimated 60,000 patients experiencing severe hypoglycemic events [11][42] - Severe hypoglycemic events can lead to serious complications, including seizures and increased mortality rates, with those affected having a fivefold increased risk of death [12] Zamyla Cell Overview - **Zamyla Cell**: An investigational islet cell therapy derived from stem cells, designed to replace destroyed islet cells in T1D patients [13] - Administered via infusion into the hepatic portal vein, protected from immune destruction through a steroid-free immunosuppressive regimen [14] - The pivotal Phase 1/2/3 study is progressing, with enrollment expected to complete in summer 2025 [15] Clinical Trial Data - The FORWARD study has transitioned to a Phase 3 pivotal study, focusing on patients with severe hypoglycemia and impaired awareness [20] - Primary efficacy endpoints include freedom from severe hypoglycemia and achieving a hemoglobin A1c (HbA1c) of less than 7% [20] - Data from 12 participants showed restored endogenous insulin production and significant reductions in HbA1c, with 10 out of 12 participants eliminating insulin requirements by 12 months [23][25] Regulatory and Market Potential - Vertex has received several regulatory designations, including RMAT and fast track designations in the US, and PRIME designation in Europe, highlighting the high unmet need for T1D therapies [15] - Anticipated regulatory submissions for Zamyla Cell are expected in 2026, with preparations for commercialization underway [42] Additional Insights Patient Population and Treatment Considerations - Ideal candidates for Zamyla Cell therapy are adults with long-standing T1D and impaired awareness of hypoglycemia [64] - The therapy is expected to significantly improve the quality of life for patients, addressing the burdens of daily diabetes management [39] Future Innovations - Vertex is exploring next-generation therapies, including gene editing and novel immunotherapies, to enhance treatment options for T1D [60] - Manufacturing capabilities are being expanded to meet anticipated demand, with partnerships established to support production [61] Challenges and Considerations - Continuous glucose monitoring has reduced severe hypoglycemic events, but a significant portion of patients still experience these events, indicating a continued need for therapies like Zamyla Cell [49] - The potential for redosing Zamyla Cell exists, as it is an off-the-shelf therapy that can be supplied on demand [54] Conclusion - Zamyla Cell represents a transformative potential for T1D treatment, addressing a significant unmet medical need for patients suffering from severe hypoglycemia [42] - Vertex Pharmaceuticals is positioned to lead advancements in T1D therapies, with ongoing research and development efforts aimed at improving patient outcomes and quality of life [60]

撰文丨王聪 编辑丨王多鱼 排版丨水成文 1 型糖尿病 （T1D） 是一种慢性自身免疫疾病，其特征是胰腺中产生胰岛素的 β 细胞 被破坏，这种破坏 是由免疫相关机制引发的。这种病症的一个显著特征是需要立即进行外源性胰岛素替代治疗，并且需要终 身治疗。 国际糖尿病联盟的流行病学数据显示，近年来，全球 1 型糖尿病的发病率和患病率呈上升趋势，这一情况 令人担忧。虽然胰岛素替代疗法仍是 1 型糖尿病管理的主要手段，但它并不能解决导致该病的潜在自身免 疫失调问题。因此，人们投入了大量研究精力来开发新型抗糖尿病药物和新兴疗法，例如免疫调节方法、 基于细胞的干预措施以及胰腺 β 细胞移植，旨在为治疗 1 型糖尿病提供新途径，尤其是实现治愈。 2025 年 5 月 9 日，南京医科大学附属逸夫医院 刘煜 、 李阳阳 ，复旦大学人类表型组研究院 吴浩 等人 在 Cell 子刊 Cell Reports Medicine 上发表了题为： Restoration of intestinal secondary bile acid synthesis: A potential approach to improve pancreat ...