Edgewise Therapeutics Doses First Patient in Phase 2 CIRRUS-HCM Trial of EDG-7500 in Obstructive Hypertrophic Cardiomyopathy (HCM)

Rhea-AI Summary

Edgewise Therapeutics, Inc. (Nasdaq: EWTX) has dosed the first patient in the Phase 2 CIRRUS-HCM trial of EDG-7500, a novel oral cardiac sarcomere modulator designed to address issues associated with obstructive Hypertrophic Cardiomyopathy (HCM). The trial will assess safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with obstructive HCM. Positive preclinical data has led to the advancement of EDG-7500 into this trial, showing encouraging effects on left ventricular outflow tract gradient relief and ventricular function. The company expects to report data from the trial in the third quarter of 2024 and plans to initiate further trials in the second half of 2024 and fourth quarter of 2024.

Positive

• Encouraging preclinical data supporting EDG-7500's effects on left ventricular outflow tract gradient relief and ventricular function
• Advancement of EDG-7500 into Phase 2 CIRRUS-HCM trial signifying robust discovery and development capabilities of Edgewise Therapeutics
• Upcoming data reporting in the third quarter of 2024 and initiation of additional trials in the second half of 2024 and fourth quarter of 2024

Negative

• None.

Insights

Medical Research Analyst

The commencement of Edgewise Therapeutics' Phase 2 CIRRUS-HCM trial for EDG-7500 marks a significant step in the clinical development of a potential treatment for hypertrophic cardiomyopathy (HCM), a condition known for abnormal thickening of the heart muscle. The compound's unique mechanism targeting the cardiac sarcomere to manipulate contraction velocity and improve diastolic function is innovative in the field of cardiology. This research could address a substantial unmet medical need, as HCM can lead to serious complications like heart failure and sudden death.

The outcomes of the pharmacokinetics and pharmacodynamics studies will be critical in understanding the drug's behavior within the body and its biological effects. Given the chronic nature of HCM, long-term safety and efficacy data will be important for eventual market acceptance. The drug's ability to alleviate left ventricular outflow tract gradient without compromising ventricular contractility presents a noteworthy advancement over existing treatments.

Investors should monitor upcoming reports from the single-dose arm and Phase 1 trial in healthy volunteers expected in Q3 2024, which will provide early indications of the drug’s safety profile and dosing parameters. Subsequent phases and the open-label extension trial will further define its potential commercial viability. However, risks remain as the drug is still in the early stages and successful market entry will depend on consistent positive trial results and eventual regulatory approval.

Financial Analyst

For investors, Edgewise Therapeutics' advancement into Phase 2 trials for their HCM drug candidate, EDG-7500, can have meaningful financial implications. The company's narrow focus on muscle disease therapeutics indicates a high-risk, high-reward proposition. The successful development of EDG-7500 could enable the company to capture a significant share of the HCM treatment market.

Financially, the capital requirements to fund ongoing trials need to be evaluated. While no immediate revenue from the product is expected until after regulatory approval and commercialization, the progression to later-stage trials often serves as a positive catalyst for biotech stocks. The current and future funding status, including any potential partnership deals or financing rounds, will be pivotal in sustaining the development without diluting current shareholders' value.

The projected timelines for trial data releases and the start of a 28-day trial are concrete milestones that investors can use to track the company's progress. Should the trial outcomes be favorable, they can lead to an increase in stock valuation due to anticipated future earnings. However, in case of trial setbacks or delays, investor sentiment can be negatively affected.

BOULDER, Colo.--(BUSINESS WIRE)-- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced the dosing of the first patient in the Phase 2 CIRRUS-HCM trial of EDG-7500. EDG-7500 is a novel oral, selective, cardiac sarcomere modulator, specifically designed to slow early contraction velocity and address impaired cardiac relaxation associated with HCM and other diseases of diastolic dysfunction. The Phase 2 trial will assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of EDG-7500 in patients with obstructive HCM. Part A of the trial will evaluate single doses and Part B will evaluate multiple oral doses of EDG-7500 over 28 days.

“Based on the strength of clinical and preclinical data to-date, we are advancing EDG-7500 into the Phase 2 CIRRUS-HCM trial in patients with obstructive HCM,” said Marc Semigran, M.D., Chief Development Officer, Edgewise Therapeutics. “We are encouraged by the effects of EDG-7500 observed in a translatable preclinical disease model of obstructive HCM. EDG-7500 demonstrated potent left ventricular (LV) outflow tract gradient relief while maintaining overall normal LV contractility. Importantly, in preclinical models of non-obstructive HCM, both acute and chronic administration of EDG-7500 has also been observed to be associated with significant improvements in ventricular filling and diastolic function.”

Kevin Koch, Ph.D., President and Chief Executive Officer, Edgewise Therapeutics added, “Advancing EDG-7500 into patients with obstructive HCM is an important milestone for Edgewise. EDG-7500 is representative of the robust discovery and development capabilities at Edgewise and our commitment to patients with serious muscle diseases.”

The Company expects to report data from the single dose arm of this trial and a Phase 1 trial of EDG-7500 in healthy volunteers in the third quarter of 2024. Furthermore, the Company expects to initiate a 28-day trial in patients with obstructive and non-obstructive HCM in the second half of 2024. The Company also expects to begin an open-label extension trial of EDG-7500 in the fourth quarter of 2024.

About the Phase 2 CIRRUS-HCM Trial

CIRRUS-HCM is a multi-center, two-part, open-label trial of EDG-7500 in patients with obstructive HCM at up to 20 clinical sites in the U.S. The trial will evaluate the safety, tolerability, PK and pharmacodynamics of EDG-7500 in up to 30 adults with obstructive HCM. Participants enrolled in this trial will receive EDG-7500 as a single oral dose and may then receive multiple oral doses over 28 days. To learn more about this trial (NCT06347159), go to clinicaltrials.gov.

About Hypertrophic Cardiomyopathy

HCM is the most common form of genetic heart disease, affecting approximately one in 200-500 individuals. HCM is caused by abnormal proteins in the heart, including cardiac myosin, that lead to excessive cardiac contraction, referred to as hypercontractility. This disruption in cardiac muscle contractility leads to increased stress and thickening of the walls of the major pumping chamber of the heart, the LV. The LV becomes less compliant and therefore less able to fill with and pump blood resulting in a decrease in the LV chamber volume. Individuals with HCM can become extremely limited in their functional capacity and ability to perform the activities of daily living. In addition, these individuals are at increased risk of heart failure, stroke, atrial fibrillation, and sudden cardiac arrest. Despite advancements in the development of therapies to treat HCM, there is currently a significant unmet need for additional therapeutic approaches and options for patients.

About EDG-7500 for Hypertrophic Cardiomyopathy

EDG-7500 is a novel oral, selective, cardiac sarcomere modulator, specifically designed to slow early contraction velocity and address impaired cardiac relaxation associated with HCM and other diseases of diastolic dysfunction. Preclinical data in models of both obstructive and non-obstructive HCM suggest the ability to drive a broadly effective clinical response at a low risk of decreasing left ventricular ejection fraction below normal at all doses tested. Based on EDG-7500’s self-limiting effect on systolic contraction observed in preclinical models, the Company plans to investigate fixed-dose regimens of EDG-7500, thus potentially avoiding intensive safety monitoring of patients on current therapy.

The Company is advancing a randomized, placebo-controlled, single and multiple ascending dose Phase 1 trial (NCT06011317) evaluating safety, tolerability, PK and pharmacodynamics in healthy adults. The Company recently initiated the Phase 2 CIRRUS-HCM trial to evaluate the safety, tolerability, PK and pharmacodynamics of EDG-7500 in patients with obstructive HCM. To learn more about this trial (NCT06347159), go to clinicaltrials.gov.

About Edgewise Therapeutics

Edgewise Therapeutics is a leading muscle disease biopharmaceutical company developing novel therapeutics for muscular dystrophies and serious cardiac conditions. The Company’s deep expertise in muscle physiology is driving a new generation of novel therapeutics. Sevasemten is an orally administered skeletal myosin inhibitor in late-stage clinical trials in Becker and Duchenne muscular dystrophies. EDG-7500 is a novel cardiac sarcomere modulator for the treatment of hypertrophic cardiomyopathy and other diseases of diastolic dysfunction, currently in Phase 2 clinical development. The entire team at Edgewise is dedicated to our mission: changing the lives of patients and families affected by serious muscle diseases. To learn more, go to: www.edgewisetx.com or follow us on LinkedIn, X (formerly Twitter), Facebook, Instagram and Threads.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Such forward-looking statements include, among other things, statements regarding the potential of, and expectations regarding EDG-7500; statements regarding the timing of reporting data (including the single dose arm of the Phase 2 CIRRUS-HCM Trial of EDG-7500 and Phase 1 trial of EDG-7500 in healthy volunteers); statements regarding Edgewise’s expectations relating to its clinical trials, including the commencement of trials (including the Phase 2 trial of EDG-7500 in individuals with obstructive and non-obstructive HCM and open-label extension trial of EDG-7500); and statements by Edgewise’s president and chief executive officer and chief development officer. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. The forward-looking statements contained herein are based upon Edgewise’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those projected in any forward-looking statements due to numerous risks and uncertainties, including but not limited to: risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early clinical stage company including the potential for Edgewise’s product candidates to cause serious adverse events; Edgewise’s ability to develop, initiate or complete preclinical studies and clinical trials for, obtain approvals for and commercialize any of its product candidates; the timing, progress and results of preclinical studies and clinical trials for EDG-7500; Edgewise’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; the timing, scope and likelihood of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; the potential that the outcome of preclinical testing and early clinical trials may not be predictive of the success of later clinical trials; Edgewise’s ability to develop a proprietary drug discovery platform to build a pipeline of product candidates; Edgewise’s manufacturing, commercialization and marketing capabilities and strategy; the size of the market opportunity for Edgewise’s product candidates; the loss of key scientific or management personnel; competition in the industry in which Edgewise operates; Edgewise’s reliance on third parties; Edgewise’s ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled “Risk Factors” in documents that Edgewise files from time to time with the U.S. Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Edgewise assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law.

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Edgewise Contacts

Investors:

Michael Carruthers, Chief Financial Officer

ir@edgewisetx.com

Media:

Maureen Franco, VP Corporate Communications

media@edgewisetx.com

Source: Edgewise Therapeutics

FAQ

What is the Phase 2 trial conducted by Edgewise Therapeutics related to EDG-7500?

The Phase 2 trial is named CIRRUS-HCM and focuses on assessing the safety and effectiveness of EDG-7500 in patients with obstructive Hypertrophic Cardiomyopathy (HCM).

What are the expected outcomes of the Phase 2 trial for EDG-7500?

The trial aims to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of EDG-7500 in patients with obstructive HCM.

When will Edgewise Therapeutics report data from the Phase 2 trial and the Phase 1 trial in healthy volunteers?

The company plans to report data from the single dose arm of the Phase 2 trial and the Phase 1 trial in healthy volunteers in the third quarter of 2024.

What trials does Edgewise Therapeutics plan to initiate in the future related to EDG-7500?

The company expects to initiate a 28-day trial in patients with obstructive and non-obstructive HCM in the second half of 2024 and begin an open-label extension trial in the fourth quarter of 2024.