Merck Reports P-II (WILLOW) Trial Data of Enpatoran for Cutaneous Lupus Erythematosus (CLE) and Systemic Lupus Erythematosus (SLE)

Shots: The P-II (WILLOW) basket trial assessed enpatoran (25, 50 &100mg, BID, PO) vs PBO + SoC in 2 Arms, where Arm 1 has SLE & CLE pts with active lupus rash & Arm 2 has active SLE pts. Discussions with health authorities on a global P-III program are underway Arm 1 met its 1EP,…

ByRidhi RastogiMay 22, 2025

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CRISPR Therapeutics Enters a Multi-Target Collaboration with Sirius Therapeutics to Develop Novel siRNA Therapies

Shots: CRISPR & Sirius have partnered to co-develop & co-market SRSD107, with CRISPR having the option to nominate ~2 siRNA targets, fund related research, & retain opt-in rights for clinical development & marketing As per the deal, Sirius will receive $25M cash & $70M in equity upfront, with both companies jointly developing SRSD107 under a…

ByRidhi RastogiMay 20, 2025

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Travere Therapeutics Reports the US FDA’s sNDA Acceptance of Filspari for Focal Segmental Glomerulosclerosis (FSGS)

Shots: The US FDA has accepted sNDA of Filspari for the treatment of FSGS, plus FDA has planned to hold advisory committee meeting to discuss the application (PDUFA: Jan 13, 2026) sNDA was supported by P-III (DUPLEX) trial & P-II (DUET) trial assessing Filspari vs irbesartan in FSGS pts; Pts completing the DUPLEX & DUET double-blind…

ByRidhi RastogiMay 16, 2025

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The US FDA Approves Incyte’s Zynyz to Treat Adults with Squamous Cell Anal Carcinoma (SCAC)

Shots: FDA has approved Zynyz (retifanlimab-dlwr) + Pt-based CT as 1L treatment of inoperable locally recurrent or metastatic SCAC, & as monotx. for locally recurrent or metastatic SCAC progressing on or intolerant to Pt-based CT; EMA & PMDA’s application are under review Approval was based on P-III (POD1UM-303/InterAACT2) trial assessing Zynyz + Pt-based CT vs…

ByRidhi RastogiMay 16, 2025

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AbbVie’s Emrelis Receives the US FDA’s Accelerated Approval for NSCLC With High c-Met Protein Overexpression

Shots: The US FDA has granted accelerated approval to Emrelis (telisotuzumab vedotin-tllv) for treatment-experienced pts with locally advanced or metastatic, c-Met overexpressing, non-squamous NSCLC Approval was backed by an ongoing P-II (LUMINOSITY) study assessing Emrelis as 2L/3L treatment of c-Met overexpressing NSCLC; showing an ORR of 35% & mDoR of 7.2mos. in pts (n=84) with…

ByRidhi RastogiMay 16, 2025

NEWS

Merck’s Welireg Receives the US FDA’s Approval for Pheochromocytoma and Paraganglioma

Shots: The US FDA has approved Welireg for the treatment of pts (≥12yrs.) with advanced, unresectable, or metastatic pheochromocytoma & paraganglioma (PPGL) Approval was based on Cohort A1 data from P-II (LITESPARK-015) trial assessing Welireg monotx. (120mg; QD) in 72 pts with unresectable or non-curable PPGL & stable blood pressure (BP <150/90 mm Hg, <135/85 mm…

ByRidhi RastogiMay 15, 2025

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MavriX Bio Reports the US FDA’s IND Clearance of MVX-220 for Angelman Syndrome

Shots: The US FDA has approved IND application of MVX-220 for the treatment of Angelman Syndrome (AS) to initiate P-I/II (ASCEND-AS) trial, with first pts dosing expected in H2’25 The P-I/II trial will evaluate the safety, tolerability, & effectiveness of MVX-220 in both adult & pediatric pts with multiple AS genotypes, incl. rare types like uniparental…

ByRidhi RastogiMay 13, 2025

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Cognition Therapeutics Reports Topline P-II (MAGNIFY) Trial Data of Zervimesine for Geographic Atrophy (GA)

Shots: Cognition has reported topline P-II (MAGNIFY/COG2201) trial data assessing zervimesine (200mg, QD, PO) vs PBO in adults with GA secondary to dry AMD; trial concluded after enrolling ~100 of 246 planned subjects In the trial, nearly 2/3 pts completed 12mos. & 1/3 completed 18mos. of dosing, showing a 28.6% slower GA progression & ~12%,…

ByRidhi RastogiMay 9, 2025

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FDA Grants Verastem Oncology’s Avmapki + Fakzynja Accelerated Approval for KRAS-Mutated Low-Grade Serous Ovarian Cancer (LGSOC)

Shots: This accelerated approval for the combination of Avmapki (avutometinib) + Fakzynja (defactinib) was granted in advance to the planned PDUFA of Jun 30, 2025, which will be available in the US within a week as a co-pack Approval was based on P-II (RAMP 201) trial assessing Avmapki (3.2mg, twice weekly) + Fakzynja (200 mg,…

ByDipanshu DixitMay 9, 2025

NEWS

Amgen Reports the MHRA’s Approval of Tepezza to Treat Thyroid Eye Disease

Shots: UK’s MHRA has approved Tepezza (teprotumumab) for the treatment of adult pts with mod. to sev. thyroid eye disease (TED) Approval was based on extensive clinical data incl. P-III (OPTIC) trial (n=83), P-II (TED01RV) trial (n=88), P-IV (HZNP-TEP-403) trial (n=62) & P-III (OPTIC-J) trial in Japan (n=54) OPTIC trial showed that 83% (34/41) of…

ByRidhi RastogiMay 8, 2025

Merck Reports P-II (WILLOW) Trial Data of Enpatoran for Cutaneous Lupus Erythematosus (CLE) and Systemic Lupus Erythematosus (SLE)

CRISPR Therapeutics Enters a Multi-Target Collaboration with Sirius Therapeutics to Develop Novel siRNA Therapies

Travere Therapeutics Reports the US FDA’s sNDA Acceptance of Filspari for Focal Segmental Glomerulosclerosis (FSGS)

The US FDA Approves Incyte’s Zynyz to Treat Adults with Squamous Cell Anal Carcinoma (SCAC)

AbbVie’s Emrelis Receives the US FDA’s Accelerated Approval for NSCLC With High c-Met Protein Overexpression

Merck’s Welireg Receives the US FDA’s Approval for Pheochromocytoma and Paraganglioma

MavriX Bio Reports the US FDA’s IND Clearance of MVX-220 for Angelman Syndrome

Cognition Therapeutics Reports Topline P-II (MAGNIFY) Trial Data of Zervimesine for Geographic Atrophy (GA)

FDA Grants Verastem Oncology’s Avmapki + Fakzynja Accelerated Approval for KRAS-Mutated Low-Grade Serous Ovarian Cancer (LGSOC)

Amgen Reports the MHRA’s Approval of Tepezza to Treat Thyroid Eye Disease

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