Edgewise Therapeutics announces positive top-line results from Phase 2 CIRRUS-HCM trial of EDG-7500 in hypertrophic cardiomyopathy

Edgewise Therapeutics reported encouraging top-line results from its Phase 2 CIRRUS-HCM four-week study evaluating EDG-7500 in patients with both obstructive and nonobstructive hypertrophic cardiomyopathy (HCM). EDG-7500 is a novel, oral cardiac sarcomere modulator designed to slow early contraction velocity and improve impaired cardiac relaxation without compromising systolic function.

In participants with obstructive HCM, the 100 mg dose of EDG-7500 achieved a 71% mean reduction in resting left ventricular outflow tract gradient (LVOT-G) and a 58% reduction post-Valsalva, with no meaningful declines in left ventricular ejection fraction (LVEF). The treatment also led to a 62% mean reduction in NT-proBNP, a key heart failure biomarker, and a 23-point average improvement on the Kansas City Cardiomyopathy Questionnaire (KCCQ). Furthermore, 78% of participants improved by at least one NYHA class, and 67% reached Class I (asymptomatic).

Among nonobstructive HCM participants, EDG-7500 demonstrated a 42% reduction in NT-proBNP, early improvements in diastolic function, and strong gains on KCCQ scores.

EDG-7500 was generally well-tolerated. The most frequent adverse events were dizziness, upper respiratory tract infections, and atrial fibrillation (AF). Two participants required cardioversion for AF, and one discontinued due to moderate dizziness. No cases of LVEF below 50% were observed.

Part D of CIRRUS-HCM, a 12-week dosing study with dose optimization, is underway, with data expected in the second half of 2025. A Phase 3 trial is planned for the first half of 2026.

Edgewise Therapeutics prices $200 million stock offering to advance muscular and cardiac therapies

On April 2, 2025, Edgewise Therapeutics announced the pricing of an underwritten public offering of its common stock, aiming to raise approximately $200 million. Shares were priced at $20.13 each. The offering is expected to close on April 3, 2025, pending customary closing conditions.

The deal attracted participation from prominent institutional investors including Braidwell LP, Cormorant Asset Management, Driehaus Capital Management, Invus, Janus Henderson, MPM BioImpact, OrbiMed, Paradigm BioCapital, Perceptive Advisors, RA Capital, and Sofinnova Investments.

Edgewise plans to use the proceeds to support the potential U.S. commercial launch of its lead candidate sevasemten for Becker muscular dystrophy, if approved, and to advance Phase 3 trials of the same therapy for Duchenne muscular dystrophy. Additional funds will go toward Phase 3 trials of EDG-7500 for hypertrophic cardiomyopathy and other research and development efforts, as well as general corporate needs.

Leerink Partners, Piper Sandler, Guggenheim Securities, and Truist Securities served as joint book-running managers for the offering.

Edgewise specializes in developing treatments for muscular dystrophies and cardiac conditions, with two main clinical programs: sevasemten, a skeletal myosin inhibitor, and EDG-7500, a cardiac sarcomere modulator.

Edgewise Therapeutics reported its fourth-quarter and full-year 2024 financial results along with recent business updates. The company completed enrollment for the GRAND CANYON global pivotal study of sevasemten in Becker muscular dystrophy and announced positive top-line results from the Phase 2 CANYON trial in Becker. It expects to release data from the Phase 2 CIRRUS-HCM trial of EDG-7500 in hypertrophic cardiomyopathy in the first quarter of 2025 and results from the Phase 2 LYNX and FOX trials in Duchenne muscular dystrophy in the first half of 2025.

Financially, Edgewise ended 2024 with $470.2 million in cash, cash equivalents, and marketable securities. Research and development expenses for Q4 2024 were $36.4 million, while general and administrative expenses totaled $9.2 million. The company reported a net loss of $39.7 million for the quarter, compared to $34.1 million in the previous quarter.

Edgewise remains focused on advancing its muscular dystrophy and cardiovascular programs, engaging with regulatory authorities, and continuing its outreach to the scientific and patient communities.

Edgewise Therapeutics, Inc. announced the promotion of Behrad Derakhshan, Ph.D., to Chief Operating Officer, effective January 8, 2025. Dr. Derakhshan has served as the company’s Chief Business Officer since September 2020 and has an extensive background in biotechnology, including leadership roles at VectivBio, Therachon, and Alexion Pharmaceuticals. He also has experience in business development and strategic evaluation, with prior management consulting experience and a strong academic foundation, including a Ph.D. in Biochemistry.

In connection with his promotion, Dr. Derakhshan’s annual base salary increased from $478,400 to $520,000, and his target annual bonus rose from 40% to 45% of his salary for fiscal year 2025. There are no family relationships or arrangements tied to his appointment.

This promotion reflects Edgewise Therapeutics’ commitment to strengthening its leadership team as the company continues to advance its mission in the biotechnology sector.