Bezuclastinib NDA submitted under the FDA’s RTOR program based on positive results from Phase 3 PEAK trial; bezuclastinib previously granted Breakthrough Therapy Designation in GIST Bezuclastinib combination demonstrated 16.5 month mPFS and 46% ORR in imatinib-resistant GIST patients, dramatically improving upon the current standard of care April 01, 2026 -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseas

April 01, 2026 -- Actinogen Medical Limited (ASX: ACW) announces that the first participant has been treated in the open-label extension (OLE) phase of the XanaMIA trial following completion of 36 weeks of treatment in the randomized phase. Open-label phase of up to 25 months duration, during which all participants will receive active Xanamem® 10 mg once daily, with no placebo control group Open to all former and current participants who have completed the randomized phase of the XanaMIA pivot

After Spinraza's FDA approval nearly a decade ago, Biogen's spinal muscular atrophy (SMA) drug captured rapid momentum and delivered blockbuster-level sales in just its second year on the market. While it's been a reliable revenue contributor ever since, rival meds from Roche and Novartis have taken away some of Spinraza's steam over the years. Now, Biogen is ready with its answer, as the FDA has approved the company's high-dose version of its SMA blockbuster. The new version features an "accel

Results of dose-ranging study suggest potential for best-in-class clinical profile across efficacy, safety and ease of use, including simplified dosing regimen with minimal to no titration necessary HRS-1893 treatment resulted in rapid and substantial reductions in left ventricular outflow tract gradient (Valsalva LVOT-G complete gradient response (<30 mmHg) of up to 86%) with minimal change in left ventricular ejection fraction (LVEF; range of decrease between 1.8% and 2.7%) Data highlight

March 30, 2026 -- Cure SMA, the leading nonprofit organization supporting those impacted by spinal muscular atrophy (SMA), celebrates that Biogen today announced FDA approval of the High Dose regimen of SPINRAZA™ (nusinersen) to treat people living with SMA. With broad label approval for all those living with SMA, this is an excellent result for the SMA community. SPINRAZA is an antisense oligonucleotide therapy that increases production of survival motor neuron (SMN) protein, essential for moto

March 30, 2026 -- Nanoscope Therapeutics, Inc., a biotechnology company committed to developing and commercializing novel, disease-agnostic therapies for patients with photoreceptor loss and vision impairment due to retinal degeneration, today announced that it has received Orphan Drug Designation in the Kingdom of Saudi Arabia for its lead therapy, MCO-010, for the treatment of patients with inherited retinal dystrophies (IRDs) having severe vision loss. The broad designation from Saudi regula

KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced new interim results from its KONFIDENT-KID clinical trial evaluating EKTERLY® (sebetralstat) for the on-demand treatment of hereditary angioedema (HAE) attacks in children ages 2-11 presented at the 2026 Global Angioedema Leadership Conference. KONFIDENT-KID is the largest pediatric trial ever conducted in HAE. It features a proprietary oral disintegrating tablet (ODT) formulation of sebetralstat in a population that currently relies

March 30, 2026 -- Sobi® (STO: SOBI) today announced that the European Medicines Agency (EMA) has validated an indication extension application for Tryngolza® (olezarsen) for the treatment of adult patients with severe hypertriglyceridemia (sHTG) ≥880 mg/dL (≥10 mmol/L). Patients with elevated triglyceride levels have substantially higher risks of all-cause mortality, atherosclerotic cardiovascular events, and acute pancreatitis[1]. The submission is supported by results from the pivotal Phase 3

Poster #76954 highlights QTORIN™ rapamycin’s single phase anhydrous gel formulation designed to optimize dermal bioavailability of rapamycin for mTOR-driven skin diseases while overcoming the crystallization, stability, and dermal penetration challenges posed by rapamycin Poster #76929 highlights a qualitative patient and caregiver interview study evaluating the burden of living with porokeratosis, a serious, rare genetic skin disease characterized by numerous pre-cancerous, pruritic lesions and

In the ADlong Phase 3b study, nearly all EBGLYSS-treated patients achieved meaningful skin improvement (EASI-75) for up to four years 75% of patients achieved a high bar of near-complete skin clearance (EASI-90) and 78% experienced significant itch relief (Pruritus NRS ≤4), one of the most bothersome symptoms for patients 80% of patients achieved durable results without the need for topical corticosteroids March 27, 2026 -- New long-term data show Eli Lilly and Company's (NYSE: LLY) EBGLYSS (le