Ziftomenib

Issued on behalf of GT Biopharma, Inc. USA News Group News Commentary VANCOUVER, BC, Dec. 4, 2025 /PRNewswire/ -- Triple-therapy immunotherapy approaches are demonstrating complete leukemia elimination in preclinical models by reprogramming how cancer cells die to trigger powerful immune responses[1], while breakthrough natural killer cell research is revealing new pathways to supercharge the body's innate tumor-fighting capabilities[2]. These developments position GT Biopharma, Inc. (NASDAQ: GTBP), Geron Corporation (NASDAQ: GERN), Kura Oncology, Inc. (NASDAQ: KURA), SELLAS Life Sciences Group, Inc. (NASDAQ: SLS), and TScan Therapeutics, Inc. (NASDAQ: TCRX). The FDA's November approval of the first once-daily menin inhibitor for relapsed NPM1-mutated AML[3] validates targeted blood cancer therapies as investors and institutions recognize timing advantages before late-stage clinical catalysts materialize across the competitive landscape. GT Biopharma, Inc. (NASDAQ: GTBP) recently announced successful completion of the formal safety review for its ongoing Phase 1 clinical trial of GTB-3650's third dosing group (Cohort 3), with no safety or tolerability issues observed. With all the significant recent progress of GTB-3650, GTBP is currently advancing innovative immunotherapy treatments designed to combat some of the world's most challenging cancer types. This latest milestone has allowed GT Biopharma to advance into Cohort 4, where patients will receive 10μg/kg/day. Now the company is actively screening patients for Cohort 4 and anticipates initiating dosing in the coming weeks, with the next comprehensive update expected in the first quarter of 2026. The Phase 1 study is testing GTB-3650 in patients battling relapsed or refractory blood cancers that express the CD33 protein, specifically acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). These represent some of the most difficult cancer cases to treat, involving patients whose disease either came back after initial therapy or never responded to conventional treatment options. GTB-3650 works by stimulating the patient's natural killer cells, a type of immune cell that naturally hunts down and destroys abnormal cells, to specifically target cancer cells. Patients receive the therapy through continuous infusions following a structured schedule: two weeks of treatment followed by two weeks of rest, repeating this cycle for up to four months based on how they respond. The six patients enrolled across Cohorts 1 through 3 have all been successfully treated with GTB-3650, demonstrating the therapy's tolerability at progressively higher dose levels. According to the company, the Cohort 4 dose level of 10μg/kg/day is more reflective of the potential clinical efficacy threshold. This assessment is based on positive trends observed across multiple immunological biomarkers from the previous six patients, the complete absence of dose-limiting toxicities throughout all three completed cohorts, and recognition that the earlier cohorts utilized lower dose levels that may have been below the therapeutic range where meaningful clinical benefit occurs. The Phase 1 design calls for testing GTB-3650 in approximately 14 patients across seven cohorts, with two patients per cohort receiving progressively higher doses from 1.25μg/kg/day in Cohort 1 up to 100μg/kg/day in Cohort 7 if necessary. Beyond the current Cohort 4, three additional higher-dose cohorts remain available: Cohort 5 at 25μg/kg/day, Cohort 6 at 50μg/kg/day, and Cohort 7 at the maximum planned dose of 100μg/kg/day. This wide dosing range reflects the trial's goal of identifying where GTB-3650 delivers optimal therapeutic benefit while maintaining an acceptable safety profile. The trial is measuring safety, pharmacokinetics, pharmacodynamics, in vivo expansion of endogenous patient natural killer cells, and clinical activity. Beyond blood cancers, the company is developing GTB-5550, which targets B7H3, a protein commonly found across various solid tumor types including breast, lung, ovarian, pancreatic, bladder, and prostate cancers. GT Biopharma plans to file its regulatory application to begin human trials of GTB-5550 either in the fourth quarter of 2025 or in January 2026. GTB-5550 is being designed as a subcutaneous injection that patients might eventually self-administer at home. Both candidates utilize GT Biopharma's proprietary TriKE platform technology, which employs specialized antibody fragments originally found in camels and llamas. These molecules offer advantages over conventional antibodies due to their smaller size and greater stability. The company holds an exclusive worldwide license from the University of Minnesota for this technology. CONTINUED… Read this and more news for GT Biopharma, Inc. at: Geron Corporation (NASDAQ: GERN) achieved $47.2 million in RYTELO net product revenue in the third quarter of 2025 and completed enrollment of 320 patients in its Phase 3 IMpactMF clinical trial evaluating imetelstat in relapsed/refractory myelofibrosis. The commercial-stage biopharmaceutical company strengthened its leadership team with the appointment of Ahmed ElNawawi as Chief Commercial Officer and now has approximately 1,150 ordering accounts for RYTELO, an increase of approximately 150 quarter-over-quarter. "The high unmet need in lower-risk MDS is well known, and RYTELO is a therapy with a novel mechanism of action and the potential to significantly impact the treatment paradigm," said Harout Semerjian, President and CEO of Geron. " Geron is positioned, with our realigned leadership team, to strengthen commercial execution, increase both physician and patient awareness, and drive RYTELO sales in the U.S." Geron expects the IMpactMF interim analysis readout for overall survival in the second half of 2026 and final analysis in the second half of 2028. The company reduced its 2025 total operating expenses guidance to between $250 million and $260 million from the previously announced $270 million to $285 million, with cash, cash equivalents, restricted cash and marketable securities of approximately $421.5 million as of September 30, 2025. Kura Oncology, Inc. (NASDAQ: KURA) announced that KOMZIFTI (ziftomenib), the first and only once-daily FDA-approved menin inhibitor for adults with relapsed or refractory acute myeloid leukemia with a susceptible NPM1 mutation, has been included in the NCCN Guidelines as a Category 2A recommended treatment option. The drug received full FDA approval on November 13, 2025, supported by data from the KOMET-001 clinical trial demonstrating a 21.4% complete response rate and median duration of response of 5 months, and is now commercially available to prescribers in the U.S. through a limited network of specialty pharmacies and distributors. "The addition of KOMZIFTI to the NCCN Guidelines in Oncology underscores the potential impact of KOMZIFTI for patients with R/R NPM1-mutated AML and supports our commitment to ensuring that patients have access to this important treatment option," said Mollie Leoni, M.D., Chief Medical Officer of Kura Oncology. "NPM1-mutated disease carries a substantial risk of relapse and historically poor outcomes in the relapsed or refractory setting. We are pleased to have received inclusion in the NCCN guidelines so rapidly after FDA approval and are committed to making KOMZIFTI available to patients in the United States." Kura Oncology is advancing zanzalintinib's development program in front-line AML treatment with the potential for combination with approved therapies to benefit a broader spectrum of patients earlier in their disease course. The company maintains a focus on pioneering advancements in menin inhibition and farnesyl transferase inhibition for high-need hematologic malignancies and solid tumors. SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) continues to advance its Phase 3 REGAL trial of GPS in acute myeloid leukemia patients who achieved complete remission following second-line salvage therapy, with final analysis anticipated by year-end 2025 once 80 deaths are reached. The company's Phase 2 data for SLS009, a selective CDK9 inhibitor, in combination with azacitidine and venetoclax for relapsed or refractory AML have been accepted for presentation at the American Society of Hematology (ASH) Annual Meeting in December 2025. "We remain highly encouraged by the continued, strong execution across our programs and the expanding body of clinical and preclinical data reinforcing the strength of our AML-focused pipeline," said Angelos Stergiou, MD, ScD h.c., President and CEO of SELLAS. "The Phase 3 REGAL trial of GPS is advancing as planned as highlighted by the key opinion leaders at our recent R&D event. Momentum around SLS009 also continues to build—our positive Phase 2 data were accepted for presentation at the upcoming ASH Annual Meeting, and we recently presented preclinical results at ESMO demonstrating clear survival benefits in T-PLL." SELLAS received approximately $54.6 million in gross proceeds from warrant exercises in September and October 2025, providing the company with $44.3 million in cash as of September 30, 2025, plus an additional $29.1 million received in October. The company plans to initiate an 80-patient trial in newly diagnosed first-line AML patients in the first quarter of 2026. TScan Therapeutics (NASDAQ: TCRX) reached agreement with the FDA on its pivotal trial design for TSC-101 following a productive end of Phase 1 meeting, with the agency approving a study design that mirrors the current ALLOHA Phase 1 trial using a biologically-assigned internal control arm expected to enable efficient enrollment and streamlined assessment of study endpoints. The company also implemented a commercial-ready manufacturing process that shortens manufacturing time by five days while lowering cost of goods and reducing ex vivo T cell expansion, with initial technology transfer to an external contract development and manufacturing organization completed. "Following a productive meeting with the FDA, we now have a clearly defined pivotal trial design for TSC-101, and we also have an improved commercial-ready manufacturing process in place," said Gavin MacBeath, Ph.D., CEO of TScan Therapeutics. "We are focused on advancing this promising program for patients with AML and MDS and look forward to sharing updated results from the ALLOHA Phase 1 trial at ASH next month." The company made the strategic decision to prioritize clinical development of its heme program and pause further enrollment in its PLEXI-T solid tumor Phase 1 trial while focusing preclinical efforts on in vivo engineering for solid tumors and target discovery in autoimmunity. With cash, cash equivalents, and marketable securities of $184.5 million as of September 30, 2025, TScan expects to fund operations into the second half of 2027, with plans to present updated clinical data including two-year relapse data at the upcoming 67th American Society of Hematology Annual Meeting and launch its pivotal trial for TSC-101 in the second quarter of 2026. Article Sources: CONTACT: USA NEWS GROUP [email protected] (604) 265-2873 DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. 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This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ/BAY does not own any shares of GT Biopharma, Inc. but reserve the right to buy and sell, and will buy and sell shares of GT Biopharma, Inc. at any time without any further notice commencing immediately and ongoing. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material, including this article, which is disseminated by MIQ has been approved on behalf of GT Biopharma, Inc. by CDMG; this is a paid advertisement, we currently own shares of GT Biopharma, Inc. and will buy and sell shares of the company in the open market, or through private placements, and/or other investment vehicles. While all information is believed to be reliable, it is not guaranteed by us to be accurate. Individuals should assume that all information contained in our newsletter is not trustworthy unless verified by their own independent research. Also, because events and circumstances frequently do not occur as expected, there will likely be differences between the any predictions and actual results. 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药械追踪 No.1 / 诺和诺德将启动一项CagriSema新研究，用于超重儿童及青少年 据路透社2025年12月2日报道，诺和诺德正计划在超重儿童和青少年中评估其在研减重新药CagriSema。该公司已登记了一项后期临床研究，预计于1月启动，将在全球97个中心招募460名低至8岁的儿童，研究中心分布在美国、中国和欧洲等地。 CagriSema由胰淀素类似物cagrilintide和Wegovy活性成分司美格鲁肽组合而成。这项临床研究的主要终点为受试者在68周后相对于基线的体重变化情况，入组受试者将随机接受CagriSema、cagrilintide、司美格鲁肽或安慰剂。 ->点击文末阅读原文，解锁完整双语新闻 No.2 / 默沙东两款阿尔茨海默病新药报告初步数据，其一获FDA快速通道资格 2025年12月1日，默沙东（纽约证券交易所代码：MRK）公布了MK-2214和MK-1167用于治疗阿尔茨海默病（AD）的首次人体试验数据。该公司还宣布，MK-2214已获得美国食品药品监督管理局（FDA）授予的用于治疗阿尔茨海默病的快速通道资格认定（FTD）。 MK-2214是一种在研新型抗体，靶向磷酸化丝氨酸413（pS413）tau蛋白。该分子由默沙东与日本帝人制药合作开发。三项I期临床试验的数据表明，MK-2214在健康志愿者以及患有轻度认知障碍和轻中度阿尔茨海默病的患者中，具有良好的安全性、耐受性和药代动力学特性。 MK-1167是一种在研、口服的α7烟碱型乙酰胆碱受体正变构调节剂。首次人体I期研究评估了单剂量MK-1167对健康成年男性志愿者前额叶皮质谷氨酸代谢的影响，该研究结果为正在进行的II期试验的剂量选择提供了依据。 ->点击文末阅读原文，解锁完整双语新闻 No.3 / 复宏汉霖地舒单抗生物类似药HLX14在华提交上市申请 2025年12月2日，复宏汉霖（2696.HK）宣布，公司自主研发的地舒单抗HLX14（重组抗RANKL全人单克隆抗体注射液）60 mg/mL规格的上市注册申请（NDA），已获国家药监局药审中心受理，拟用于治疗骨折高风险的绝经后妇女、男性及糖皮质激素诱导的骨质疏松症，覆盖原研普罗力于中国大陆境内已获批上市的所有适应证。 HLX14为复宏汉霖按照中国、欧盟和美国等生物类似药相关法规自主开发的地舒单抗生物类似药。本次HLX14的NDA主要基于HLX14对比原研普罗力产生的数据，包括分析相似性研究及非临床和临床比对研究。这些数据都充分证明了HLX14与原研药在质量、安全性和有效性方面的高度相似。 复宏汉霖已于2022年与Organon达成授权许可和供应合作，授予其对包括HLX14在内的两款候选生物类似药在除中国以外的全球区域进行独家商业化的权益，协议覆盖美国、欧盟、加拿大等市场。2025年下半年，HLX14两个规格 BILDYOS（60 mg/mL）和BILPREVDA（120mg/1.7mL）相继获得美国FDA、欧洲药品管理局（EMA）和英国药品和健康产品管理局（MHRA）批准上市。 ->点击文末阅读原文，解锁完整双语新闻 No.4 / Kura抗癌新药KOMZIFTI在美上市，触发协和麒麟1.35亿美元里程碑付款 2025年12月3日，Kura Oncology公司（纳斯达克股票代码：KURA）宣布，KOMZIFTI（ziftomenib）在美国的首笔商业销售已顺利完成。根据Kura与协和麒麟（Kyowa Kirin）签署的合作与许可协议，这一里程碑事件将触发协和麒麟向Kura支付1.35亿美元款项，Kura预计将在年底前收到该笔款项。 Ziftomenib是一种每日一次口服menin抑制剂，用于治疗成人复发或难治性NPM1突变型急性髓系白血病。该产品于2025年11月13日获美国食品药品监督管理局（FDA）批准上市。Kura Oncology和协和麒麟于2024年11月达成许可协议；根据协议，Kura Oncology授予协和麒麟Ziftomenib在美国以外地区的权益，协议总价值最高可达15亿美元。 ->点击文末阅读原文，解锁完整双语新闻 全球医疗情报领导者 解锁隐藏在数据中的商业潜力  关于 G B I ” 自从2002年成立以来，GBI始终以技术为驱动，为药企、器械及行业相关服务商提供贯穿生命周期的全球药品市场竞争数据、全球行业资讯、HCPs洞察、全国医疗器械数据等商业信息与洞察，助力企业在进行战略布局和决策时，脱颖而出。历经20余年的深耕细作GBI已成为95%以上跨国药企、国内头部药企、咨询与投资机构等医疗圈灯塔用户值得信赖的长期合作伙伴。 联系我们 投稿 | 发稿 | 媒体合作 ▶ olivia.xu@generalbiologic.com 数据库 | 咨询服务 | 资讯追踪 ▶ 点击左下“阅读原文”完成表单填写 点击阅读原文，解锁完整双语新闻