Core Insights - Nuvation Bio has enrolled the first patient in a global, randomized study (G203) to evaluate safusidenib for maintenance treatment of high-grade IDH1-mutant glioma, a condition with no currently FDA-approved targeted therapies [1][2][3] Study Details - The G203 study will assess the efficacy and safety of safusidenib versus placebo in approximately 300 patients with newly diagnosed IDH1-mutant astrocytoma, either grade 3 with high-risk features or grade 4, following standard treatment [2] - Patients will be randomized 1:1 to receive either 250 mg of safusidenib or placebo twice daily, with the primary endpoint being progression-free survival (PFS) [2] - The study has received alignment from the FDA, indicating that PFS could support full approval for safusidenib in this setting [2] Clinical Background - IDH1-mutant gliomas are the most common type of brain cancer in adults, with nearly 2,400 new diagnoses annually in the U.S. [4] - Patients with IDH1 mutations generally have longer survival times compared to those with wild-type IDH1, but prognosis worsens for high-grade tumors [4] Drug Profile - Safusidenib is described as a novel, oral, potent, brain-penetrant targeted inhibitor of mutant IDH1, which has shown promising activity in a Phase 1 study [5] - The drug was well-tolerated and demonstrated anti-tumor activity along with high blood-brain barrier penetration [5] Company Overview - Nuvation Bio is focused on developing therapies that address significant unmet needs in cancer treatment, with a diverse pipeline that includes safusidenib and other innovative therapies [6][7] - The company was founded in 2018 by David Hung, M.D., and has offices in major cities including New York, San Francisco, Boston, and Shanghai [7]

Core Insights - TransCode Therapeutics, Inc. has completed its Phase 1a clinical trial for TTX-MC138, an investigational inhibitor of microRNA-10b, achieving its primary safety endpoint and establishing a recommended Phase 2 dose [1][5] Group 1: Clinical Trial Results - The primary objectives of the trial included safety, tolerability, pharmacokinetics, and establishing a Phase 2 dose, with 16 patients treated across four escalating dose levels [2] - No significant treatment-related safety events or dose-limiting toxicities were observed during the trial [2] - The median treatment duration was four months, with a range of two to twelve cycles, indicating good tolerability and disease control [3] - 44% of patients (7 out of 16) exhibited stable disease lasting four months or longer [3][8] Group 2: Efficacy and Future Plans - Preliminary data showed positive pharmacodynamic effects across all dose levels, consistent with preclinical models [3] - A patient with thyroid cancer showed a reversal in thyroglobulin levels during treatment, reaching undetectable levels [3] - The observed safety profile and durability of TTX-MC138's anti-tumor effects support advancing to a Phase 2a clinical trial [5][8] Group 3: About TTX-MC138 - TTX-MC138 is a first-in-class therapeutic candidate targeting microRNA-10b, which is critical in the progression of many metastatic cancers [6][7] - The drug has shown evidence of delivery to metastatic lesions and pharmacodynamic activity even at microdoses, suggesting a broad therapeutic window [6]

Core Viewpoint - Cellectar Biosciences and ITM Isotope Technologies Munich have entered a supply agreement for Actinium-225 to support the clinical development of Cellectar's CLR 121225, a novel radiopharmaceutical for treating solid tumors, including pancreatic cancer [1][2][3] Company Overview - Cellectar Biosciences is a late-stage clinical biopharmaceutical company focused on discovering and developing proprietary drugs for cancer treatment, utilizing its Phospholipid Drug Conjugate delivery platform [5] - ITM Isotope Technologies Munich SE specializes in radiopharmaceuticals and aims to provide innovative therapeutics and diagnostics for hard-to-treat tumors, leveraging over two decades of expertise in medical isotope manufacturing [9] Product Pipeline - Cellectar's product pipeline includes iopofosine I 131, which has received Breakthrough Therapy Designation from the FDA, and CLR 121225, targeting solid tumors with significant unmet needs, such as pancreatic cancer [6][7] - CLR 121225 is designed to selectively target and eradicate cancer cells using a proprietary PLE delivery platform [2][6] Strategic Partnership - The agreement between Cellectar and ITM ensures a continuous, high-quality supply of Actinium-225, which is crucial for advancing Cellectar's pipeline candidates and exploring the benefits of targeted alpha therapy for cancer patients [3][4] - ITM's joint venture, Actineer, is dedicated to advancing Ac-225 technologies and aims to meet the growing global demand for this critical isotope [10] Market Context - The scarcity of high-quality Ac-225 has hindered the development of Ac-225 based programs, prompting Cellectar to establish a network of suppliers to ensure sufficient access [3][4] - The partnership reflects a strategic commitment to enhancing global access to radiopharmaceuticals and improving patient outcomes through innovative therapies [4]