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Cytokinetics
News

Cytokinetics Reports P-III (MAPLE-HCM) Trial Data of Aficamten for Obstructive Hypertrophic Cardiomyopathy

Shots:Cytokinetics has reported P-III (MAPLE-HCM) trial data assessing aficamten vs SoC metoprolol in pts (n=175) with symptomatic obstructive hypertrophic cardiomyopathy (HCM); regulatory review is ongoing in the US FDA (PDUFA: Dec 26, 2025) & EMA, with its NDA also under NMPA's priority review The trial met its 1EP, showing superior improvement in peak oxygen…
May 14, 2025

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Regenxbio
News

REGENXBIO Reports the US FDA’s BLA Acceptance and Priority Review of RGX-121 for Treating Mucopolysaccharidosis II

Shots:The US FDA has accepted BLA & granted priority review to RGX-121 (clemidsogene lanparvovec) for the treatment of Mucopolysaccharidosis II (MPS II or Hunter syndrome), with PDUFA action date of Nov 09, 2025As per Jan 2025 partnership, NS Pharma (Nippon Shinyaku’s subsidiary) will commercialize RGX-121 in the US post approval while REGENXBIO will handle…
May 14, 2025

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Lenz Therapeutics and Lotus
News

LENZ Therapeutics Collaborates with Lotus Pharmaceutical to Commercialize LNZ100 in the Republic of Korea and Southeast Asia

Shots:LENZ has granted Lotus exclusive development, manufacturing, registration & marketing rights of LNZ100 to treat presbyopia in the Republic of Korea & certain Southeast Asian countries incl. Thailand, Philippines, Vietnam, Malaysia, Brunei, Indonesia & SingaporeAs per the deal, LENZ will obtain ~$125M in upfront, regulatory & commercial milestones, along with double-digit net sales-based…
May 12, 2025

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Vanda Pharmaceuticals
News

Vanda Pharmaceuticals Reports the US FDA’s NDA Acceptance of Bysanti (Milsaperidone) for Acute Bipolar I Disorder & Schizophrenia

Shots:The US FDA has accepted NDA of Bysanti, an active metabolite of iloperidone to treat acute bipolar I disorder & schizophrenia (PDUFA: Feb 21, 2026). If approved, Bysanti may receive 5yrs. of data exclusivity, with pending patents extending into the 2040sNDA is backed by various iloperidone trials, incl. 2 in acute schizophrenia, 1…
May 6, 2025

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Merck
News

Merck Reports P-III (KEYNOTE-689) Trial Data of Perioperative Keytruda for Locally Advanced Head and Neck Squamous Cell Carcinoma

Shots:The P-III (KEYNOTE-689) trial assessed neoadj. Keytruda (200mg, IV, Q3W × 2 cycles), followed by adj. Keytruda (× 15 cycles) + SoC radiotherapy (RT) ± cisplatin vs adj. SoC RT without cisplatin in treatment-naïve pts (n=714) with stage III/IVA resected LA-HNSCC; sBLA under the FDA’s review (PDUFA: Jun 23, 2025)At 38.3 mos. mFU,…
April 28, 2025

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PTC Therapeutics
News

PTC Therapeutics’ Sephience Receives the CHMP’s Positive Opinion for the Treatment of Phenylketonuria (PKU) 

Shots: The CHMP has issued a positive opinion recommending marketing authorization for Sephience (sepiapterin) to treat PKU in patients of all ages and disease severities, expected the EC approval within two mos., with the decision applying across the EU including Iceland, Norway, and Liechtenstein Sepiapterin's NDA is under FDA review (PDUFA: July 29, 2025), and…
April 25, 2025

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