Demonstrating better tolerability and safety in comparison with standard of care, Edgewise Therapeutics Inc.’s EDG-7500 is moving toward a phase III trial for both obstructive and nonobstructive hypertrophic cardiomyopathy (HCM), the most common form of genetic heart disease. Edgewise reported top-line phase II results from part D of the Cirrus-HCM trial, showing meaningful responses in terms of hemodynamics, biomarkers and patient-reported measures. The oral, selective cardiac sarcomere modulator was generally well-tolerated, and there were no meaningful changes in left ventricular ejection fraction (LVEF) or reductions in LVEF to below 50%, as is commonly seen with cardiac myosin inhibitors like Camzyos (mavacamten, Bristol Myers Squibb Co.) and Myqorzo (aficamten, Cytokinetics Inc.), both of which have boxed warnings. “At some point, you can completely eliminate cardiac contraction,” Kevin Koch, Edgewise’s CEO, said during an investor call June 16. EDG-7500 modulates cardiac contraction as a partial inhibitor. “That means we can never drive the ejection fraction down into dangerous levels.”

Briefing documents made public ahead of Moderna flu vaccine adcom hearing

Four months after Moderna Inc. took receipt of a refusal-to-file (RTF) letter from the U.S. FDA regarding the seasonal influenza vaccine mRNA-1010, the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) is taking up the matter. Moderna said the RTF concerns were “inconsistent with feedback” the company got from regulators during pre-phase III as well as pre-BLA-submission talks. Briefing documents released ahead of the VRBPAC sit-down June 17 sound mostly even-handed, though they highlight gaps in evidence as a “key uncertainty. Clinical efficacy data are available for one influenza season only,” reviewers point out. How well the shot works in immunocompromised people “and very frail older adults has not been established,” the documents say – potentially a problem, since such people “face the highest absolute risk of severe influenza-related complications and may respond differently to mRNA-based vaccine platforms.” Shares of Moderna (NASDAQ:MRNA), which have been climbing the past five days, were trading at $53.62, up $1.49.

2025 annual report reflects the changing role of EMA

The EMA’s 2025 annual report highlights the pressure it is under to streamline and simplify assessment processes, and the expanded – and explicit – role the agency now has in boosting the competitive position of the EU in the development and manufacturing of drugs. In addition to placing greater emphasis on how the EMA supports the competitiveness of Europe’s pharmaceutical industry by reducing red tape and speeding up procedures, the EMA has a brief to promote the use of artificial intelligence tools in drug development. “We are operating in a period of rapid transformation, defined by new ways of working and major legislative changes,” said Rui Santos Ivo, chair of the EMA’s management board, in his foreword to the 2025 report, published June 11.

AI liability in health care: Who is responsible?

Liability remains one of the biggest barriers to the adoption of AI in health care. As more tools get developed for use in clinical settings, a key question persists: Who is ultimately at fault when something goes wrong – the hospital, the clinician or the developer? That uncertainty is making clinicians hesitant to adopt new technologies, delegates heard at the HLTH Europe conference in Amsterdam on June 16.

Dimerix out-licenses DMX-200 to Everest in AU$481M deal

Dimerix Ltd. has signed an exclusive licensing deal with Everest Medicines Ltd. to commercialize its phase III kidney disease candidate DMX-200 in a transaction worth up to AU$481 million (US$340 million), plus sales royalties. Under the deal terms, Dimerix will receive a AU$14.1 million up-front payment, and up to $AU467 million in potential milestone payments as well as sales-based royalties. In exchange, Everest gains exclusive rights to commercialize DMX-200 for treating focal segmental glomerulosclerosis in greater China, South Korea and certain Southeast Asian countries (Singapore, Malaysia, Thailand, Indonesia, Vietnam and Philippines).

For clonal hematopoiesis, epigenetics can be in driver’s seat

Clonal hematopoiesis (CH), where few blood stem cells produce a significant fraction of mature blood cells that are genetically identical, is partly an inevitable feature of aging. Certainly, it is near universal in those older than 60. Some CH is driven by the death of blood-forming stem cells, which last longer than most other cells but are not immortal. Younger people can have tens or hundreds of thousands of stem cells. In older people, that number tends to be between 10 and 20. Another, more dangerous form of CH results from competition rather than attrition, in the form of mutations that blood stem cells acquire as they age. Some of those mutations will give cells a growth advantage, and over time, they will produce more daughter cells – in some cases, vastly more daughter cells.

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