Top-line data from the Reveal-2 phase III trial of elegrobart to treat chronic thyroid eye disease (TED) drove Viridian Therapeutics Inc.’s stock (NASDAQ:VRDN) up by 31.4% in early trading May 5, positioning the company for a BLA filing in the first quarter of 2027. The trial met both primary endpoints: proptosis responder rate, as measured by exophthalmometry, required for a filing with the U.S. FDA; and the overall responder rate, required for an EMA filing. Subcutaneously delivered through an autoinjector for at-home use, elegrobart (VRDN-003) is a half-life-extended monoclonal antibody targeting the insulin-like growth factor-1 receptor. TED, also known as Graves’ eye disease, is a progressive autoimmune disease that occurs when muscles and fatty tissues behind the eye become inflamed, causing proptosis, or the outward bulging of eyes. If approved, elegrobart would go up against Amgen Inc.’s Tepezza (teprotumumab), approved for TED in 2020, and potentially Viridian’s own breakthrough therapy drug veligrotug, which is under FDA review with a PDUFA date of June 30.

HCM phase III data with aficamten boosts Cytokinetics

Cytokinetics Inc. shares (NASDAQ:CYTK) were trading at $77.85, up 17%, or $11.80 on positive top-line results in symptomatic, non-obstructive hypertrophic cardiomyopathy (HCM) from the pivotal phase III Acacia-HCM trial with aficamten, an allosteric and reversible inhibitor of cardiac myosin motor activity. The study met its dual primary endpoints, turning up statistically significant improvements from baseline to week 36 compared to placebo in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score and in maximal exercise performance. Aficamten was cleared by the U.S. FDA for obstructive HCM in December under the brand name Myqorzo for Cytokinetics.

MMI’s Symani system treats Alzheimer’s patient in IDE study

Medical Microinstruments Inc.’s (MMI) Symani surgical system was used to treat a patient with Alzheimer’s disease as part of a study evaluating robotic-enabled microsurgery for the condition. The procedure investigated whether restoring lymphatic drainage pathways in the deep cervical lymph nodes could improve the clearance of harmful neurotoxins believed to contribute to the progression of Alzheimer’s.

White House projects nearly $530B in 10-year MFN savings

Amid calls for transparency on the most-favored nation (MFN) pricing deals U.S. President Donald Trump has reached with 17 biopharma companies, the White House Council of Economic Advisers released a report today detailing how the volunteer MFN pricing policy works and touting the nearly $530 billion in savings it’s projected to deliver over the next 10 years. To achieve those savings, the administration said it expects to reach similar agreements with most manufacturers of sole-source drugs and biologics. It also is working with Congress to codify the voluntary agreements into law so patients can continue to benefit from the price discounts.

Cytospire raises £61M series A to target EGFR in solid tumors

Next-generation T-cell engager (TCE) specialist Cytospire Therapeutics Ltd. has raised £61 million (US$82.7 million) in a series A round, equipping it to advance the lead program CYT-X300 to the clinic in the treatment of EGFR-positive solid tumors. The company’s pan gamma delta (γδ) TCEs are designed to overcome problems with cytokine release syndrome, on-target effects on healthy cells, and the excessive activation of CD3 that have occurred with earlier bispecific antibodies that bind to the CD3 receptor on T cells.

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