2. Edgewise Therapeutics Inc. (NASDAQ:EWTX)
Market Capitalization as of April 23: $1.52 billion
Number of Hedge Fund Holders: 49
Average Upside Potential as of April 23: 245.54%
Edgewise Therapeutics Inc. (NASDAQ:EWTX) is a biopharmaceutical company that discovers, develops, and commercializes therapies to treat muscle disorders. Its lead product candidate, EDG-5506, is an orally administered small molecule that is in Phase II clinical trials. It is designed to address the root cause of dystrophinopathies, such as Duchenne muscular dystrophy and Becker muscular dystrophy.
A Scotiabank analyst recently initiated coverage on Edgewise with a price target of $50 and an Outperform rating. The analyst noted promising early clinical data from sevasemten, which is being studied for both Becker and Duchenne muscular dystrophy, and EDG-7500 for Hypertrophic Cardiomyopathy (HCM). Edgewise’s EDG-7500 program, in particular, is showing significant promise.
Top-line data from the Phase 2 CIRRUS-HCM 4-week trial, announced in April, demonstrated clinically meaningful reductions in Left Ventricular Outflow Tract gradients (LVOT-G) in participants with obstructive HCM. Building on these positive results, Edgewise is optimizing its dosing strategy in Part D of the CIRRUS-HCM trial, with initial data expected in H2 2025, and plans to initiate a Phase 3 trial in H1 2026. Given the significant unmet need for additional therapies in HCM, a disease affecting ~1 in 500 people, the development of EDG-7500 represents a growth opportunity for Edgewise.
Baron Health Care Fund is is highly positive on the company and stated the following regarding Edgewise Therapeutics, Inc. (NASDAQ:EWTX) in its Q4 2024 investor letter:
“We initiated a position in Edgewise Therapeutics, Inc. (NASDAQ:EWTX), which develops drugs that target muscle physiology, including sevasemten to treat Becker’s muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD), and EDG-7500 to treat hypertrophic cardiomyopathy (HCM). Sevasemten is a myosin inhibitor that is designed to protect fast-twitch muscle against contraction-induced injury in muscular dystrophy patients. Sevasemten recently showed impressive Phase 2 data in Becker’s patients that suggests a meaningful benefit in muscle function that will likely translate into Phase 3 success. There are no medicines approved specifically for Becker’s patients today, and we think this could be a $1 billion drug. We think the drug should also work in Duchenne’s muscular dystrophy, since Duchenne’s is driven by the same underlying dysfunction in dystrophin protein as Becker’s. Edgewise is also developing EDG-7500, which targets cardiac sarcomere to treat hypertrophic cardiomyopathy. Although it’s early in development, EDG-7500 has shown early data that suggested impressive efficacy on LVOT gradient reduction without meaningful safety risks in the form of changes in ejection fraction. If this is borne out in more clinical trials, this would be an important point of differentiation versus existing treatments for HCM, which require patient monitoring and dose titration to avoid safety risks.”
