26 Apr Can Edgewise Therapeutics Novel Approach help it gain the Winning Edge?
Edgewise Therapeutics, Inc. (NASDAQ: EWTX) is a clinical-stage biopharmaceutical company that is developing innovative precision medicine for debilitating musculoskeletal diseases. The Company is leveraging its knowledge and expertise in muscle biology, biophysics and small molecules to create a pipeline of candidates that address large unmet needs in rare muscle diseases.
The Company’s proprietary drug discovery platform uses custom-built high throughput and translatable systems to identify and develop small molecule precision medicines that can regulate key proteins in muscle tissues, primarily in rare neuromuscular and cardiac diseases.
Edgewise Therapeutics (NASDAQ: EWTX)
Market Cap: $1.33B; Current Share Price: 27.54 USD
Data by YCharts
An Extensive Pipeline of Precision Medicine Product Candidates
Edgewise Therapeutics lead product candidate is EDG-5506, a muscle stabilizer intended for the treatment of Duchenne, Becker and Limb Girdle Muscular Dystrophies. The Company’s approach focuses on understanding how the dystrophin protein complex plays a critical role in protecting muscles from injury and degradation and how the lack of this essential protein can be managed to preserve muscle function. The candidate has demonstrated the ability to protect susceptible muscle fibers and prevent long-term development of damage in diseased animal models.
Furthermore, the candidate is a small daily tablet / liquid suspension that is potentially appropriate for all ages and is not affected by mutations.
EDG-5506 is currently being evaluated in a phase 1 clinical trial in healthy volunteers, which will be followed by trials in patients suffering from DMD and BMD, based on the results from the phase 1 study and FDA/EMA approvals.
In addition, Edgewise’s pipeline also consists of EDG-6289, intended for the treatment of Spasticity Disorders Duchenne; EDG-002 Program, a muscle desensitizer for cardiac conditions and the EDG-003 Program, a muscle desensitizer that is targeting Neuromuscular Metabolic disorders.
The Muscular Dystrophy Association describes Duchenne Muscular Dystrophy (DMD) as a fatal, X-linked genetic neuromuscular disorder that leads to progressive, irreversible loss of muscle function, including the heart and lungs. The disease has an X-linked recessive inheritance pattern and is acquired by a “Carrier” mother. Globally, 1 in 3500 male births is affected by this rare neuromuscular disease. There are over 250,000 people in the United States alone who are afflicted by this disorder.
Becker Muscular Dystrophy (BMD), also known as Benign pseudohypertrophic muscular dystrophy, is an inherited condition caused by a mutation in the DMD gene. Both the conditions are almost similar except for the fact that the symptoms of BMD manifest much later and progress at a lower rate.
The growing awareness about this disease and increasing research efforts to find a cure, along with improved insurance coverage and favorable government regulations will drive the growth in the market. According to a report by Grand View Research, Inc the market of DMD therapeutics will grow at a CAGR of 41.3% and reach USD 4.11 billion by 2023.
Researchers have identified the lack of dystrophin, a protein that helps regulate the functioning of membrane (sarcolemma) of muscle cells as the cause of this disorder. Exon skipping is gaining credence as a potential treatment approach to correct for specific genetic mutations and restore production of dystrophin protein, with an increasing number of companies coming up with first-in-class therapeutics based on this approach. Moreover, the emergence of new mechanisms of action such as NF-κB inhibition, Gene therapy, and Exosome technology have the potential to redefine the therapeutic landscape.
A Stellar Initial Public Offering Debut
In March 2021, the Company made an initial public offering of 11,000,000 shares of its common stock at a public offering price of $16.00 per share for proceeds amounting to $202.4 million. The shares were up by almost 67 percent on their debut on NASDAQ and continue to do well.
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