Bemdaneprocel

WHIPPANY, NJ, USA I January 14, 2025 I On the occasion of the 43rd J.P. Morgan Healthcare Conference in San Francisco, Bayer AG announces further progress in its pharmaceutical growth strategy with multiple regulatory filing submissions underway for key growth drivers darolutamide, finerenone, and elinzanetant. “We are successfully delivering on our ambitious business goals despite significant headwinds. At the same time, the value of our pipeline is growing by accelerating breakthrough innovations,” said Stefan Oelrich, Member of the Board of Management, Bayer AG, and President of Bayer’s Pharmaceuticals Division. “Our new operating model is visibly becoming a key enabler to drive growth and efficiency gains.” Topline improved and poised for future growth Bayer continues to strengthen its leadership position in prostate cancer with the anticipated launch of a third indication in 2025 for the oral androgen receptor inhibitor (Ari)darolutamide, marketed under the brand name NUBEQA ® ,supported by data from the ARANOTE trial. Darolutamide plus androgen deprivation therapy (ADT) now has positive data both with and without chemotherapy (docetaxel) based on two pivotal Phase III studies (ARASENS and ARANOTE) in metastatic hormone-sensitive prostate cancer (mHSPC). Bayer recently submitted an application to the Center of Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) for this third indication for darolutamide in addition to filings in the U.S. and the EU earlier in 2024. NUBEQA achieved global blockbuster status in September 2024 after crossing the threshold of one billion euros in annual sales. Additionally, NUBEQA is now the fastest growing androgen receptor inhibitor in the U.S., with nearly 45,000 patients in the U.S. and 100,000 patients globally treated as of the end of 2024. For important risk and use information about NUBEQA, please see the full Prescribing Information . In Cardiovascular, Bayer submitted a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) and CDE of China’s NMPAfor finerenone, marketed as KERENDIA ® , for the treatment of patients with heart failure (HF) with a left ventricular ejection fraction (LVEF) of ≥40%, initiating a major step forward in expanding its reach. Further regulatory filings are planned with a potential market launch by the end of 2025. Filing submissions are based on positive results from the Phase III FINEARTS-HF trial that showed finerenone achieved a statistically significant reduction of the composite of cardiovascular death and total (first and recurrent) HF events, defined as either an unplanned hospitalization for HF or an urgent HF visit, by 16% in patients with HF and a LVEF of ≥40% compared to placebo in addition to a patients’ prescribed treatment regimen. The robust data from Kerendia’s clinical development program underscore its potential to become a pillar of therapy for patients with chronic kidney disease associated with Type 2 Diabetes and patients with heart failure with an LVEF of ≥40%. For important risk and use information about KERENDIA, please see the full Prescribing Information . In Women´s Healthcare, another significant clinical milestone for investigational compound elinzanetant was secured in early 2025 following three positive Phase III readouts in 2024 (OASIS 1,2,3). OASIS 4, which was conducted outside of the U.S., is a Phase III study evaluating elinzanetant as a non-hormonal treatment for moderate to severe vasomotor symptoms caused by adjuvant endocrine therapy in women with breast cancer or at high risk of developing breast cancer. Elinzanetant successfully met the primary endpoints of the study demonstrating statistically significant reductions in the frequency of moderate to severe vasomotor symptoms (VMS, also known as hot flashes) from baseline to week 4 and 12 compared to placebo. The study also achieved all secondary endpoints demonstrating reductions in severity of VMS at week 4 and 12, VMS frequency reduction at week 1 as well as maintaining the effects over the study period. The detailed results from OASIS 4 are planned to be presented at an upcoming scientific congress. Elinzanetant is the first dual neurokinin-1 and 3 (NK-1 and 3) receptor antagonist in late-stage clinical development for the non-hormonal treatment of moderate to severe VMS associated with menopause. In Radiology, Bayer is advancing its pipeline. A significant milestone was achieved with its Phase III QUANTI clinical development program, which evaluated the investigational compound gadoquatrane. The first data from the program showed that gadoquatrane successfully achieved its primary endpoint, highlighting its potential to offer a low dose contrast agent option to patients. The QUANTI program showcases Bayer’s leadership in radiology and its commitment to advancing innovation in medical imaging. Pipeline value improved and early lead positions strengthened Bayer is making significant progress with its cell and gene therapy platform, achieving important clinical trial milestones, especially in the field of Parkinson´s disease. Bemdaneprocel will directly advance to Phase III clinical development in Parkinson’s disease, based on positive data from the Phase I exPDite trial. Bemdaneprocel is an investigational stem cell-based therapy that surgically implants dopamine-generating nerve cell precursors into the brain. The FDA granted bemdaneprocel Regenerative Medicine Advanced Therapy (RMAT) designation for its innovative potential in treating Parkinson’s disease. AB-1005 is advancing in Phase II with the randomization of participants in the REGENERATE-PD clinical trial focusing on patients with moderate-stage Parkinson’s disease. AB-1005 is an investigational AAV-based gene therapy that delivers the human glial cell line-derived neurotrophic factor (GDNF) transgene to the brain to potentially protect and restore dopamine-generating neurons. AB-1005 has received U.S. FDA Fast Track and UK Medicines and Healthcare products Regulatory Agency (MHRA) Innovation Passport designations, highlighting its potential to address significant unmet medical needs. As part of its transformation, Bayer has sharpened its focus in R&D to build a highly differentiated pipeline for long-term growth in oncology, cardiology and renal diseases, neurology and rare diseases, and immunology. Through rigorous assessment and prioritization, Bayer Pharmaceuticals is now fully focused on the areas of greatest unmet need and highest value potential. Targeted investments in R&D and platform companies in recent years are already strengthening Bayer’s early and late pipelines. With Vividion´s acquisition of the precision oncology platform company Tavros Therapeutics, Bayer continues to leverage its chemoproteomics platform technology to unlock traditionally undruggable targets with precision small-molecule therapeutics. It has initiated Phase I trials with oral KEAP1 activator in solid tumors and oral STAT3 inhibitor in solid and hematologic malignancies and has IND-enabling programs including a RAS-P13KCA program for RAS-driven cancers. Additionally in the field of precision oncology, investigational BAY 2927088, an oral, small molecule, tyrosine kinase inhibitor as a potential new targeted therapy for patients with non-small cell lung cancer (NSCLC) harboring HER2 activating mutations, showed promising results as a second line therapy in the ongoing Phase I/II SOHO-01 study evaluating safety and efficacy. Its potential is underscored by Breakthrough Therapy Designations from both the FDA and Chinese CDE. Beyond the SOHO-01 trial, BAY 2927088 is also being assessed for its potential as a first-line therapy in patients with advanced non-small cell lung cancer (NSCLC), whose tumors have activating HER2 mutations in the SOHO-02 trial. Further, a Phase I clinical trial with BAY3498264, an investigational oral selective Son of Sevenless Homologue 1 (SOS1) inhibitor, has recently been initiated (outside of the US). The open-label, first-in-human, dose escalation study will evaluate patients with KRAS G12C-mutated metastatic cancer. This innovative approach has the potential to enhance the treatment options available for patients, offering the possibility to reduce or stop tumor progression. Targeted Radionuclide Therapy (TRT) is a strategic area of focus in Oncology precision drug development at Bayer, building on more than 10 years of real-world experience with radium Ra 223 dichloride injection therapy, marketed as XOFIGO ® , for patients with metastatic castration-resistant prostate cancer. Bayer´s evolving TRT portfolio includes novel targeting approaches which combine alpha radionuclides such as actinium-225 with different targeting moieties, including antibodies, small molecules or peptide-based molecules. 225 Ac-pelgifatamab (BAY 3546828) and 225 Ac-PSMA-Trillium (BAY 3563254), two candidates targeting PSMA (prostate specific membrane antigen), are currently in Phase I clinical trials in patients with advanced metastatic castration resistant prostate cancer (mCRPC). For important risk and use information about XOFIGO, please see the full Prescribing Information . In the field of cardiovascular diseases, Bayer is making progress with its Phase II assets. With BAY3283142, an investigational soluble guanylate cyclase (sGC) activator in patients with chronic kidney disease (CKD), Bayer has entered into a Phase II clinical study. By modulating sGC via a heme-independent pathway, the investigational sGC activator now represents a new class of potential future drugs that could offer an advantage in conditions of high oxidative stress such as in diabetic nephropathy. With the anti-alpha2 antiplasmin antibody program, Bayer is developing a new modality and mechanism of action-based thrombolytic agent. Based on investigational research, the antibody works by specifically blocking the endogenous plasmin inhibitor, α2AP, potentially leading to the lysis of acute embolic or thrombotic clots with a tolerable safety profile. The investigational antibody is currently being evaluated in Phase II in patients with deep vein thrombosis and could be used for indications of high medical relevance. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to www.bayer.com . © 2025 Bayer BAYER, the Bayer Cross and NUBEQA are registered trademarks of Bayer. Find more information at www.pharma.bayer.com Our online press service is just a click away: www.bayer.us/en/newsroom Follow us on Facebook: http://www.facebook.com/pharma.bayer Follow us on X: https://X.com/BayerUS SOURCE: Bayer

1 月 14 日，拜耳宣布，旗下 BlueRock Therapeutics 公司预计于 2025 年上半年启动针对帕金森病研究性细胞疗法 Bemdaneprocel（BRT-DA01）的 III 期 exPDite-2 临床试验，同时拜耳计划基于目前的试验结果与其他数据提交该产品的上市申请。新闻稿指出，这将是神经退行性疾病同种异体细胞疗法开发的一个重要里程碑。 截图来源：企业官微 exPDite-2 是一项双盲试验，将评估 Bemdaneprocel 与对照组相比的有效性、安全性和总体影响。该试验旨在招募约 102 名患有中度帕金森病的受试者。该研究的主要终点取决于「PD 日记」（该日记将患者症状得到良好控制时分类为「ON」状态，当症状恶化时分类为「OFF」状态）ON 状态持续时间从基线到第 78 周的变化，前提是患者每天保持 16 小时清醒状态，且没有发生严重运动障碍。 Bemdaneprocel 是一种研究性疗法，由多能干细胞产生的多巴胺能神经元组成，可以通过手术植入帕金森病患者大脑。这些细胞在被移植后将有可能重塑被帕金森病破坏的神经网络，有望恢复患者的运动和非运动功能。 2024 年 9 月，拜耳公布了 Bemdaneprocel 的 I 期临床试验 exPDite 24 个月的积极数据。exPDite 是一项多中心、开放标签、非随机、非对照的 I 期研究。12 名被诊断患有帕金森病的受试者接受了手术移植，将 2 种不同剂量水平的 Bemdaneprocel 注射到双侧连合后壳核，并接受为期 1 年的免疫抑制治疗。队列 A（5 名受试者）接受的剂量为每壳核 0.9 百万个细胞。队列 B（7 名受试者）接受的剂量为每壳核 2.7 百万个细胞。主要终点是在 12 个月时评估安全性和耐受性。 数据显示： 使用 Hauser PD 日记，与基线相比，高剂量组中的 7 名受试者在 24 个月后，处于「Good ON」状态的时间增加了 1.8 小时，处于「OFF」状态的时间平均减少了 1.9 小时。低剂量组的 5 名受试者在「Good ON」状态的时间平均减少了 0.8 小时，「OFF」状态的时间增加了 0.4 小时； 使用 MDS-UPDRS 第 III 部分在「OFF」状态下对运动症状进行测量，结果显示，与基线相比，高剂量组运动症状评分减少 21.9 分，低剂量组平均减少 8.3 分； 使用 MDS-UPDRS 第 II 部分对日常生活活动能力的影响进行测量，结果显示，与基线相比，高剂量组平均减少 3.4 分，低剂量组平均增加 2.0 分。 在安全性方面，24 个月的安全性与早期发现一致，表明患者对 Bemdaneprocel 的耐受性仍然良好，未报告与 Bemdaneprocel 相关的不良事件。 值得一提的是，2024 年 5 月，该疗法获得了美国 FDA 再生医学先进疗法认定。 封面来源：企业 Logo 免责声明：本文仅作信息分享，不代表 Insight 立场和观点，也不作治疗方案推荐和介绍。如有需求，请咨询和联系正规医疗机构。 编辑：ccai PR 稿对接：微信 insightxb 投稿：微信 insightxb；邮箱 insight@dxy.cn 多样化功能、可溯源数据…… Insight 数据库网页版等你体验 点击阅读原文，立刻解锁！