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Philochem and RayzeBio (BMS) entered into a development and commercialization agreement under which Philochem granted RayzeBio exclusive worldwide rights to develop, manufacture, and commercialize its radiopharmaceutical agent OncoACP3 for prostate cancer
Under the agreement, Philochem will receive $350M up front and is eligible for up to $1B in development, regulatory, and commercial milestones, plus…
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Genrix Bio has granted Cullinan exclusive global license to velinotamig, excl. Greater China for all indications, with Cullinan planning to develop it in autoimmune diseases
As per the deal, Genrix will receive $20M upfront, ~$292M in development & regulatory milestones & ~$400M in sales-based milestones, with net sales-based tiered royalties from mid-single digits up to…
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The EC has approved label extension of Evrysdi (5mg oral tablet) for pts with spinal muscular atrophy (SMA)
Approval was based on P-I study assessing Evrysdi 5mg tablet vs oral solution, showing bioequivalence with comparable efficacy & safety; data was presented at SMA Europe 2024
Evrysdi, an SMN2 splicing modifier, treats SMA caused by…
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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…
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The US FDA has received the IND application for UTRxM1-18, a novel therapy targeting c-MYC-driven cancers, to initiate P-I trial in 2026 pending approval, focusing on c-MYC driven tumors incl. triple-negative breast, pancreatic, colorectal, & ovarian cancer
UTRxM1-18 has demonstrated strong, dose-dependent efficacy against human-derived tumors from multiple cancer subtypes, with no dose-limiting toxicities…
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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,…
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Eli Lilly has entered into a licensing agreement with Alchemab for ATLX-1282 to treat ALS & other neurodegenerative conditions
As per the deal, Alchemab will receive ~$415M, incl. an upfront payment, potential discovery, development, & commercial payments, as well as royalties, plus will handle early P-I trial activities, with Lilly leading further development &…
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Chengdu Origen & Vanotech has reported the first pts dosing in Vanotech-led P-I (VAN-2401) trial assessing KH658 for wet age-related macular degeneration (wet AMD) in the US
The P-I trial will assess safety, tolerability, & efficacy of single suprachoroidal KH658 gene therapy in ~9 previously treated wet AMD pts that are responsive to anti-VEGF therapy…
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China’s NMPA has received 2 IND applications for HF158K1 (K1) & HFK2 (K2) to initiate P-I dose escalation trial in China to evaluate safety & PK of K1 alone & its preliminary efficacy with K2 to treat pts with solid tumors refractory to prior therapies
Preclinical studies of K1, K2, & their combination showed…
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The IIT P-II (IPAX-Linz) trial assessed TLX101 (IV) in 8 glioblastoma pts with recurrent tumors & PET-confirmed amino acid uptake, who received adaptive dosing with ~4GBq before & ~2GBq after, 2L EBRT; 5/8 had MGMT-unmethylated tumors
Trial showed mOS of 12.4mos. or 32.2mos. from initial diagnosis, complementing P-I (IPAX-1) trial results of 13mos. & 23mos.,…

