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 syndrome As 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…

ByRidhi RastogiNovember 13, 2025

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 lymphoma Designation 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 2026 March Bio will pursue clinical & commercial…

ByRidhi RastogiNovember 12, 2025

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 pain As per the deal, AmacaThera & Pacira will jointly advance AMT-143, with AmacaThera leading select clinical studies & Pacira funding development through commercialization. In exchange, AmacaThera…

ByRidhi RastogiNovember 6, 2025

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 data NRTX-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 MTLE The…

ByRidhi RastogiNovember 6, 2025

NEWS

Boehringer Ingelheim and CDR-Life Expand their Partnership with a Global Licensing Deal for CDR111

Shots: Boehringer Ingelheim and CDR-Life signed a global licensing deal to develop CDR111, a trispecific M-gager T-cell engager targeting and depleting B cells to help reset the immune system in autoimmune diseases As per the deal, CDR-Life could receive up to CHF 456M (~$570M), including CHF 38M (~$ 48M) upfront and near-term payments, plus tiered…

ByDipanshu DixitNovember 5, 2025

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 planned Approval was backed by a P-II trial, 2 retrospective chart review studies, & an…

ByRidhi RastogiNovember 4, 2025

NEWS

Calluna Pharma’s CAL101 Receives the US FDA’s Orphan Drug Designation to Treat Idiopathic Pulmonary Fibrosis (IPF)

Shots: The US FDA has granted ODD to CAL101 for the treatment of idiopathic pulmonary fibrosis (IPF) CAL101 (IV, QM for 7mos.) is being investigated in a P-II (AURORA) trial against PBO in 150 IPF pts, with enrolment open in the sites across US, UK, EU, Turkey, & South Korea, evaluating lung function per forced vital…

ByRidhi RastogiNovember 3, 2025

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 options Approval 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.,…

ByRidhi RastogiOctober 28, 2025

NEWS

Sanofi Reports P-II (ElevAATe) Trial Data on Efdoralprin Alfa for Alpha-1 Antitrypsin Deficiency (AATD) Emphysema

Shots: Sanofi has reported the P-II (ElevAATe) trial data assessing efdoralprin alfa (Q3W or Q4W) vs SoC plasma-derived augmentation therapy (QW) in 97 adults with AATD emphysema for ~32wks. Trial met its 1EP with a greater mean increase in functional AAT (fAAT) levels at Wk. 32 & achieved key 2EPs of higher fAAT levels &…

ByRidhi RastogiOctober 24, 2025

NEWS

Insmed’s Brinsupri (Brensocatib) Receives the CHMP’s Positive Opinion to Treat Non-Cystic Fibrosis Bronchiectasis (NCFB)

Shots: The CHMP has recommended Brinsupri (Brensocatib) for treating NCFB pts (≥12yrs.) with two or more exacerbations in the prior 12 mos. Opinion was based on P-III (ASPEN) trial (n=1721) & P-II (WILLOW) trial (n=256) assessing Brinsupri (10 or 25mg, PO) vs PBO in NCFB pts for 52 & 24wks., respectively, with WILLOW enrolling pts who had…

ByDipanshu DixitOctober 21, 2025

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

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

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

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

Boehringer Ingelheim and CDR-Life Expand their Partnership with a Global Licensing Deal for CDR111

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

Calluna Pharma’s CAL101 Receives the US FDA’s Orphan Drug Designation to Treat Idiopathic Pulmonary Fibrosis (IPF)

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

Sanofi Reports P-II (ElevAATe) Trial Data on Efdoralprin Alfa for Alpha-1 Antitrypsin Deficiency (AATD) Emphysema

Insmed’s Brinsupri (Brensocatib) Receives the CHMP’s Positive Opinion to Treat Non-Cystic Fibrosis Bronchiectasis (NCFB)

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