The U.S. FDA has granted Orphan Drug Designation for asedebart (Lu AG13909), Lundbeck's novel investigational anti-ACTH monoclonal antibody, for the treatment of endogenous Cushing's syndrome Endogenous Cushing's Syndrome is a rare endocrine disorder driven in most cases by excess adrenocorticotropic hormone, or ACTH, leading to chronic cortisol excess and substantial disease burden, with current treatment options often limited by suboptimal disease control and treatment-related complications 1–

First two patients dosed in the EXACT study Topline data from the initial 16-week placebo-controlled portion of the study expected mid-2027; final readout expected by the end of 2027 Sept. 11, 2026 -- Eloxx Pharmaceuticals, Inc. (Nasdaq: ELOX), a clinical-stage biopharmaceutical company developing exaluren for the treatment of rare kidney diseases, today announced that the first two patients have been dosed in the EXACT Study, a Phase 2b clinical trial evaluating exaluren in patients with nonse

Sept. 9, 2026 -- Insilico Medicine ("Insilico," HKEX: 3696), a clinical-stage biotechnology company powered by generative AI, today announced that it has dosed the first patient with Rentosertib (known as ISM001-055 / INS018_055) in GENESIS-IPF-3, a Phase III clinical trial (NCT07687459, CTR20262475) at Peking Union Medical College Hospital, while Shanghai Pulmonary Hospital has also enrolled its first patient on the same day. The Phase III clinical trial is a prospective, multi-center, randomi

If approved, Enspryng would be the first and only disease-modifying therapy for MOGAD (myelin oligodendrocyte glycoprotein antibody-associated disease) Filing acceptance is based on the Phase III METEOROID study showing that Enspryng reduced the risk of relapses by 68% MOGAD is a rare disease of the central nervous system characterized by unpredictable attacks of the optic nerves, spinal cord or brain Genentech, a member of the Roche Group (SIX: RO, ROP; OTCQX: RHHBY), announced today that the

HCB101 represents the first known U.S. Expanded Access case for a SIRPα Fc fusion protein enabling continued therapy beyond the parent study Sept. 9, 2026 -- HanchorBio, Inc. (TWSE: 7827), a global clinical-stage biotechnology company developing next-generation immunotherapies for oncology and immune-mediated diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its HCB101 Single-Patient Expanded Access Protocol (HCB101- EA-001) as "safe to proceed". The FDA det

First presentation of pivotal Phase 3 CAPLYTA ® data in adults with bipolar mania underscores the asset's potential across mood disorders New SPRAVATO ® analyses and schizophrenia research emphasize a focus on complex, high-burden neuropsychiatric conditions Sept. 8, 2026 -- Johnson & Johnson (NYSE: JNJ) today announced that 24 abstracts featuring clinical data and real-world evidence across the neuropsychiatry portfolio will be presented at the 2026 Psych Congress Annual Meeting (September 15-

Phase 1 study evaluates distinct 225Ac and 177Lu compounds in breast cancer and other solid tumors; data expected in late 2027 225Ac-A9-0642 is the first actinium-based GRPR-targeting compound to enter clinical evaluation with the potential to deliver enhanced efficacy compared to first-generation 177Lu approaches 177Lu-A9-0631 is a lutetium-based GRPR-targeting compound with best-in-class potential Alpha-9 takes a modular approach to molecular engineering, holistically optimizing each component

Sept. 8, 2026 -- PeproMene Bio, Inc. today announced the publication in The Lancet, one of the world's leading medical journals, of results from its Phase 1 clinical trial evaluating PMB-CT01, the company's first-in-class BAFF-R-targeted CAR T-cell therapy, in patients with relapsed or refractory B-cell non-Hodgkin lymphoma. The publication reports durable complete clinical responses, together with a more favorable safety profile compared with approved CAR T-cell therapies. Seven of nine treate

The POLARIS-1 study enrolled 147 patients with uncontrolled, moderate-to-severe asthma, and the interim analysis was conducted on the first 98 patients. The analysis demonstrated a half-life of up to 75 days, with clinical effects consistent with twice-yearly dosing. WIN378 produced significant and sustained effects on FEV1 (up to 174 mL mean increase, placebo-adjusted mean increases up to +256 mL, p=0.033), FeNO and blood eosinophils, key biomarkers that correlate with asthma exacerbations. Th

With $116 million in new funding on tap, BrainChild Bio said Tuesday that it has initiated a pivotal study of the B7-H3-targeted autologous CAR-T cell therapy BCB-276, in the hopes of delivering a much-needed new treatment option for patients with diffuse intrinsic pontine glioma (DIPG). With the current standard-of-care treatment for DIPG limited to palliative focal radiation therapy, median overall survival for this rare and aggressive paediatric brainstem tumour is about 11 months from diagn