Avacincaptad pegol

Takeda's first-in-class orexin treatment won an FDA nod in narcolepsy. Daiichi Sankyo faced investor pushback after admitting to an accounting error. Astellas booked a new quarterly revenue record. And more. 1. Takeda gains FDA nod for first-in-class narcolepsy treatment Orzeyful Takeda’s Orzeyful (oveporexton) has become the first orexin treatment to reach the market. Approved by the FDA for narcolepsy type 1, the drug is also the first medicine to address the root cause of the disease, rather than its symptoms. Takeda has pegged Orzeyful’s peak sales at between $2 billion and $3 billion. 2. Daiichi Sankyo flags accounting error, as shares tank despite revenue beat Daiichi Sankyo’s stock price tanked 11% after the company reported $182 million in extra expenses that it had missed in its fiscal year 2025 report. The Japanese pharma attributed the incident to “booking errors in accounts payable for suppliers.” Meanwhile, despite major antibody-drug conjugate sales growth, questions remain around the firm’s profitability. 3. Astellas’ revenue hits new record as strategic brands drive major beat (release, PDF) Astellas’ quarterly revenue hit a new high. At 640.9 billion Japanese yen ($4.05 billion) for the three months ended in June, it marked 26.7% growth year over year, crushing analysts’ consensus by 15%. Sales from key brands, including Pfizer-partnered Padcev, eye drug Izervay, stomach cancer therapy Vyloy and menopause med Veozah, all topped expectations. 4. AstraZeneca, CSPC to form joint venture for biologics manufacturing AstraZeneca has teamed up with CSPC Pharmaceutical again, this time on a new manufacturing joint venture in China. The new 51-49 joint venture, located in Shijiazhuang, will initially focus on producing “mutually agreed biologics drug substances for the global markets,” CSPC said. The two firms have multiple ongoing R&D collaborations. 5. From Abu Dhabi to Brazil, every region is vying for the ‘new emerging economy’ of biotech Asian countries are vying to build out their own biopharma ecosystems. “South Korea stands out as a reliable and fully compliant alternative that offers operational continuity without regulatory friction,” James Choi, chief marketing officer at Samsung Biologics, told Fierce. 6. In a first, Otsuka’s Voyxact stabilizes IgAN patients’ kidney function decline to normal levels Otsuka’s Voyxact has become the first drug to stabilize kidney function decline to the normal rate in a phase 3 trial among patients with IgA nephropathy. Results from the phase 3 Visionary trial linked Voyxact to an annualized estimated glomerular filtration rate (eGFR) slope of a 0.3 mL/min/1.73m² gain over two years, versus a 4.2 mL/min/1.73m² decline for placebo. 7. Once again, child dies in gene-editing trial in China, rekindling debate on transparency and safety (Stat) 8. Braveheart charges past expectations with $382.5M IPO to fund cardio drug from Hengrui 9. Sanofi-backed Expedition uses $115M series B to advance COPD drug on phase 2 journey 10. Shionogi makes the call on COVID pill Xocova TV spot 11. Ono, Mediar enter antibody discovery pact for fibro-inflammatory diseases (release)

Inflammasome Therapeutics reported six-month Phase II results showing its investigational drug K8 slowed geographic atrophy lesion growth by more than half in the trial’s mid-dose cohort, according to a press release issued by the Massachusetts-based company. The K8 geographic atrophy data, presented at the American Society of Retina Specialists meeting in Montreal, also showed a statistically significant visual-acuity advantage over control eyes — an endpoint neither of the two currently approved GA therapies met in their pivotal trials. For a disease with no cure and only modest treatment options, a signal of preserved vision alongside slower lesion growth addresses a key limitation of existing therapies and may warrant advancement into late-stage development. Geographic atrophy is an advanced form of dry age-related macular degeneration marked by progressive death of retinal tissue, affecting an estimated eight million people worldwide. K8 (kamuvudine-8) is a dual inflammasome inhibitor delivered as a bioerodible, sustained-release intravitreal implant dosed once every three months; it is designed to block inflammasome activation triggered by multiple toxic drivers of GA — including complement, retrotransposons and oxidative stress — rather than a single upstream pathway. K8 remains fully investigational and has not been approved by the FDA or any other regulatory authority. The multicenter Phase II trial enrolled 30 participants with bilateral GA across nine US centers — 60 eyes total, with the worse-seeing eye receiving a K8 implant and the fellow eye serving as an untreated control. Three doses (0.3 mg, 0.7 mg, 1.05 mg) were tested, administered at baseline and again at month three. In the 0.7 mg cohort, the mean rate of GA lesion growth was 54% lower than in the pooled control group over six months (p\=0.016), using the FDA-preferred linear mixed-effects slope model. A prespecified analysis restricted to eyes with extrafoveal lesions — those with the greatest potential for vision preservation — showed a covariate-adjusted 4.0-letter mean BCVA advantage for K8-treated eyes versus controls (p\=0.004); BCVA changes were positive at every visit in the K8 group and negative at every visit in controls. No drug-related serious adverse events or dose-limiting toxicities were reported through month six, and there were no cases of endophthalmitis, intraocular inflammation, neovascular AMD, retinal vasculitis or optic neuropathy. The results extend an earlier readout from the same trial, which showed a 66% reduction in lesion growth at three months in an initial five-patient cohort — a result the company used to justify expanding enrollment to 60 eyes. Two therapies are approved in the US for GA: Apellis Pharmaceuticals’ Syfovre (pegcetacoplan), a complement C3 inhibitor cleared in February 2023, and Astellas’ Izervay (avacincaptad pegol), a complement C5 inhibitor approved in August 2023. Both require monthly or near-monthly intravitreal injections and reduced GA lesion growth by roughly 13-14% versus control over their first six months of dosing, without demonstrating a visual-acuity benefit in Phase III testing. Neither has secured EMA approval, reportedly reflecting European regulators’ emphasis on functional vision outcomes rather than anatomic lesion measures alone. Inflammasome Therapeutics frames the 54% growth reduction and paired BCVA signal as clearing “a high bar” relative to those benchmarks, though the comparison is cross-trial rather than head-to-head, and the underlying patient populations, endpoints and control designs differ. The dosing interval is a further point of contrast. K8’s implant is designed for administration every three months, versus monthly injections for the approved complement inhibitors — a difference that, if durability holds up in larger studies, could reduce the treatment burden that has limited real-world uptake of existing GA therapies. The fellow-eye control design used in this trial, where each patient serves as their own comparator, also strengthens the internal consistency of the result, since natural-history data cited by the company indicate GA progresses at nearly identical rates between a patient’s two eyes absent treatment. The visual-acuity finding is what distinguishes this readout from prior GA trials. Both approved drugs demonstrated anatomic effects — slower lesion expansion — without translating into measurable functional benefit, a disconnect that has tempered enthusiasm among retina specialists about their real-world impact. If replicated in larger, randomized studies, a therapy that demonstrates both slower structural loss and preserved visual function would address one of the principal limitations of existing GA treatments Caution is warranted given the sample size: the BCVA analysis was conducted in just 30 eyes (14 treated, 16 control), and the primary efficacy analysis, while statistically significant, comes from a single Phase II study rather than a confirmatory trial. Inflammasome Therapeutics has said it plans to move directly into a global Phase III pivotal program. Access the AllSci platform to explore the science behind the news. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website Privacy Terms About Us Copyright © 2026. AllSci Corp. All rights reserved.