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Kura Oncology and Kyowa Kirin
NEWS

Kura Oncology and Kyowa Kirin Report the US FDA’s Approval Komzifti (Ziftomenib) for NPM1-Mutated Acute Myeloid Leukemia (AML)

Shots:The US FDA has granted full approval to Komzifti (QD, PO) to treat adults with r/r AML with NPM1 mutation who have no satisfactory alternative treatment options before the PDUFA date of Nov 30, 2025Approval was backed by P-I/II (KOMET-001) trial in 112 r/r NPM1-mutant AML pts, showing 21.4% CR + CRh with a median…
November 14, 2025

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NEWS

Neurogene Reports Interim P-I/II Trial Results on NGN-401 to Treat Pediatric Patients with Rett Syndrome

Shots:Neurogene has reported interim data from the pediatric cohort (4-10yrs.) of the ongoing P-I/II trial assessing NGN-401 for the treatment of females with Rett syndromeAs of Oct 30, 2025, all pediatric pts (n=8) had functional gains across Rett syndrome domains, achieving 35 developmental milestones in fine motor, communication & ambulation. These gains were…
November 13, 2025

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NEWS

March Biosciences’ MB-105 Receives the Regenerative Medicine Advanced Therapy Designation for R/R CD5+ T-Cell Lymphoma

Shots:The US FDA has granted RMAT designation to MB-105 for the treatment of r/r CD5-positive T-cell lymphomaDesignation was based on the ongoing P-II trial, which showed clinical activity with a favorable safety profile; initial data to be presented at ASH’25, with additional updates expected in 2026March Bio will pursue clinical & commercial…
November 12, 2025

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NEWS

AmacaThera Inks a ~$230M Exclusive Global Licensing Deal with Pacira BioSciences to Advance AMT-143

Shots:AmacaThera has entered into an exclusive global license agreement with Pacira to develop & commercialize AMT-143, a long-acting non-opioid anesthetic, leveraging AmacaThera’s tunable hydrogel platform to treat post-operative painAs per the deal, AmacaThera & Pacira will jointly advance AMT-143, with AmacaThera leading select clinical studies & Pacira funding development through commercialization. In exchange, AmacaThera…
November 6, 2025

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NEWS

Neurona Therapeutics’ NRTX-1001 Secures the EMA’s Priority Medicines (PRIME) Designation for Drug-Resistant Focal Epilepsy

Shots:The EMA has granted PRIME designation to NRTX-1001 for adults with drug-resistant focal epilepsy, based on preclinical & emerging clinical dataNRTX-1001 is also being evaluated in 2 ongoing P-I/II trials for drug-resistant unilateral & bilateral mesial temporal lobe epilepsy (MTLE), with P-III (EPIC) trial planned to begin in H2’25 for drug-resistant MTLEThe…
November 6, 2025

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NEWS

UCB Reports the US FDA’s Approval of Kygevvi (Doxecitine + Doxribtimine) for Thymidine Kinase 2 Deficiency (TK2d)

Shots:FDA has approved Kygevvi (2g/2g; PO) for adults & pediatric pts with TK2d whose symptom onset occurred ≤12yrs. of age. Kygevvi is expected to launch in the US by Q1’26, & is under EMA's review, with additional regulatory filings plannedApproval was backed by a P-II trial, 2 retrospective chart review studies, & an…
November 4, 2025

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Syndax
NEWS

Syndax Pharmaceuticals’ Revuforj (Revumenib) Receives the US FDA’s Approval for NPM1-Mutated R/R Acute Myeloid Leukemia

Shots:The US FDA has approved label expansion of Revuforj to treat pts (≥1yrs.) with nucleophosmin 1 (NPM1)-mutated r/r acute myeloid leukemia (AML) who have no satisfactory alternative treatment optionsApproval was supported by P-II results from the P-I/II (AUGMENT-101) trial, which showed a 23% CR + CRh rate (15/65), median response time of 2.8mos.,…
October 28, 2025

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