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OrsoBio has reported topline P-IIa PoC trial data assessing TLC-2716 (6 or 12mg, QD, PO) vs PBO in 30 overweight pts with severe hypertriglyceridemia & metabolic dysfunction–associated steatotic liver disease (MASLD)
The study met its 1EP, showing statistically significant reductions in fasting triglycerides & remnant cholesterol, plus improved liver fat by MRI-PDFF, with stronger lipid…
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THX has granted Biocodex an exclusive global license to develop & commercialize Batten-1 for juvenile Batten disease (CLN3), along with exclusive US & Canada rights to develop & commercialize TX01 for Gaucher & Niemann-Pick type C disease
As per the deal, THX will lead clinical development with financial & scientific support from Biocodex, which will…
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Insilico Medicine has entered into multiple AI-powered drug discovery collaborations with CMS, focusing on advancing therapeutic programs across CNS & autoimmune disease indications
As per the deal, Insilico will leverage its Pharma.AI platform for target discovery & small-molecule design & optimization, while CMS will lead clinical development, regulatory strategy, trials, & commercialization
Under the…
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The US FDA has granted RMAT Designation to KB707 for the treatment of advanced or metastatic NSCLC
Designation was supported by the ongoing P-I/II (KYANITE-1) trial assessing KB707 (administered via nebulization) across 7 cohorts: Arms 1-4 as monotx., Arm 5 with Keytruda, Arm 6 with Keytruda & CT & Arm 7 with CT alone…
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The P-II trial of obexelimab (250mg, SC, QW) vs PBO in RMS, met its 1EP with a 95% relative reduction in cumulative new Gd-enhancing T1 lesions over wks. 8 & 12, with near-complete suppression by wk. 8 sustained through wk. 12.
Adjusted mean new lesions was 0.01 vs 0.23, only 2 vs 19 total…
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The US FDA has accepted NDA & granted priority review to oveporexton (TAK-861) for the treatment of NT1, with PDUFA goal date in Q3’26
NDA was supported by robust clinical data, incl. global P-III (FirstLight & RadiantLight) trials assessing oveporexton in NT1 pts
Oveporexton showed objective and patient-reported improvements in wakefulness, EDS, cataplexy, attention,…
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The Chinese NMPA has accepted the application of Arexvy RSV vaccine for the prevention of LRTD caused by respiratory syncytial virus (RSV) in adults aged ≥60yrs.; regulatory decision is expected by 2027
Submission was based on extensive clinical data, incl. results from the P-III trial assessing safety & immunogenicity of Arexvy in adults (≥60yrs.)…
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Iambic has partnered with Takeda in a multi-year tech & discovery collaboration to utilize Iambic’s AI drug discovery models toward advancing select high-priority small molecule programs in Takeda’s Oncology, & GI & Inflammation therapeutic areas
The collaboration will leverage Iambic’s AI drug discovery suite & wet lab capabilities, with Takeda also gaining access to…
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Eli Lilly has entered into a definitive agreement to acquire Orna Therapeutics, gaining a broad platform to drive long-term innovation in genetic medicine & in vivo cell engineering
As per the deal, Lilly will acquire Orna, with Orna’s shareholders receiving up to $2.4B in cash, incl. an upfront & subsequent clinical development milestones payment
Acquisition…
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MannKind has enrolled the first patient in the P-II (INHALE-1st) trial assessing initiation of Afrezza(insulin human) Inhalation Powder in pediatric pts shortly after their type 1 diabetes diagnosis
Trial will evaluate Afrezza over 13wks., followed by an optional extension phase for pts continuing to use Afrezza with basal insulin up to 26wks., assessing the proportion…

