Otsuka Holdings Co., Ltd.

Otsuka Pharmaceutical and U.S.-based Ionis Pharmaceuticals announced on the 22nd that their jointly developed treatment for hereditary amyotrophic lateral sclerosis, ulefnersen, met its primary goal in a late-stage clinical trial. In patients with FUS mutation ALS, a rare inherited form of ALS that damages the nerve cells controlling movement, ulefnersen improved function and extended survival compared with the placebo group. The drug reduced markers of nerve cell damage and slowed disease progression, and most side effects were mild or moderate, indicating a favorable safety profile. No approved treatment currently targets the genetic cause of FUS-ALS, and the two companies plan to discuss the results with the U.S. Food and Drug Administration and global health authorities as they explore a path toward accelerated approval. Separately, Otsuka Pharmaceutical has launched a global early access program for FUS-ALS patients unable to participate in the trial, allowing physicians to request access to ulefnersen before approval.

Ionis Pharmaceuticals announced positive late-stage results from two partnered phase III programs. The FUSION study of ulefnersen in amyotrophic lateral sclerosis caused by mutations in the fused in sarcoma gene met its primary endpoint assessing functional impairment and survival at 72 weeks, with Ionis reporting the results as statistically significant without disclosing supporting numbers; Otsuka Pharmaceutical, which holds worldwide commercialization rights licensed in 2024, plans to discuss the data with the FDA and other global health authorities regarding potential expedited regulatory submission pathways. Separately, the IMAgINATION study of sefaxersen in adults with primary immunoglobulin A nephropathy, conducted by Ionis' partner Roche, met its primary endpoint in a prespecified interim analysis, showing statistically significant and clinically meaningful reductions in proteinuria after 37 weeks, and will continue in a blinded manner to evaluate kidney function over two years with estimated glomerular filtration rate at week 105 as the longer-term measure. Roche licensed sefaxersen from Ionis in 2022 and is responsible for the phase III study and future global development, regulatory and commercialization activities, while Ionis is eligible for milestone payments and tiered royalties on net sales of both drugs. The two wins follow back-to-back cardiovascular setbacks for Ionis, including the phase III CARDIO-TTRansform failure of Wainua with AstraZeneca in July and the phase III Lp(a)HORIZON miss for pelacarsen with Novartis, and come after the FDA approval of Zanvastro for Alexander disease earlier this month. Year to date, Ionis shares have lost 42% compared with the industry's 2% decline.

Otsuka Holdings has revised upward its full-year earnings forecast for the fiscal year ending December 2026. The company raised its operating profit forecast by 30.3 percent from the previous projection to 469 billion yen, and its profit attributable to owners of the parent by 34.0 percent to 355 billion yen. It also plans to increase the annual dividend to 200 yen this fiscal year, up from 140 yen in the previous year. In the first half, revenue rose 12.8 percent year on year to 1.3323 trillion yen, operating profit increased 15.0 percent to 278.5 billion yen, and profit attributable to owners of the parent grew 24.3 percent to 215.6 billion yen, marking higher revenue and profit. The upward revision was supported by strong sales of its main antipsychotic drug Rexulti and an improved selling, general and administrative expense ratio due to cost optimization. The share price climbed further after the earnings release and has risen about 10 percent over the past month.

A new report from DelveInsight reveals that more than 28 companies are actively developing over 30 pipeline drugs for tuberculosis. Key players include Biofabri, Otsuka Pharmaceutical, GlaxoSmithKline, Archivel Farma, BioNTech, CanSino Biologics Inc., Medincell, and others, with promising therapies such as MTBVAC, Quabodepistat, GSK3036656, RUTI, BNT164, and Ad5-105K in various clinical stages. Approximately four-plus drugs are in late-stage development, and notable mechanisms of action include leucyl-tRNA synthetase inhibitors, immunostimulants, and DprE1 protein inhibitors. The report also highlights recent milestones, including the first patient dosed in a Phase IIb trial of alpibectir-ethionamide in March 2026 and the initiation of a new cohort in the IMAGINE trial of MTBVAC in February 2026.