Edgewise Therapeutics Reports Third Quarter 2024 Financial Results and Recent Business Highlights
Edgewise Therapeutics (EWTX) reported Q3 2024 financial results and updates on its clinical programs. The company maintains a strong cash position of $492.5 million as of September 30, 2024. Q3 net loss was $34.1 million ($0.36 per share), compared to $31.5 million in Q2. R&D expenses increased to $32.2 million from $30.7 million in Q2.
The company is advancing multiple Phase 2 trials: CANYON for Becker muscular dystrophy (top-line results expected December 2024), LYNX and FOX for Duchenne muscular dystrophy, and CIRRUS-HCM for hypertrophic cardiomyopathy. Positive Phase 1 data was reported for EDG-7500 in healthy subjects and Phase 2 CIRRUS-HCM trial in obstructive HCM patients.
Edgewise Therapeutics (EWTX) ha riportato i risultati finanziari del terzo trimestre 2024 e aggiornamenti sui suoi programmi clinici. L'azienda mantiene una posizione di liquidità solida di $492.5 milioni al 30 settembre 2024. La perdita netta del terzo trimestre è stata di $34.1 milioni ($0.36 per azione), rispetto a $31.5 milioni nel secondo trimestre. Le spese di R&S sono aumentate a $32.2 milioni rispetto ai $30.7 milioni del secondo trimestre.
L'azienda sta portando avanti diversi studi di Fase 2: CANYON per la distrofia muscolare di Becker (risultati preliminari attesi per dicembre 2024), LYNX e FOX per la distrofia muscolare di Duchenne, e CIRRUS-HCM per la cardiomiopatia ipertrofica. Sono stati riportati dati positivi di Fase 1 per EDG-7500 in soggetti sani e nello studio di Fase 2 CIRRUS-HCM in pazienti con HCM ostruttiva.
Edgewise Therapeutics (EWTX) informó sobre los resultados financieros del tercer trimestre de 2024 y las actualizaciones de sus programas clínicos. La compañía mantiene una posición de efectivo sólida de $492.5 millones al 30 de septiembre de 2024. La pérdida neta del tercer trimestre fue de $34.1 millones ($0.36 por acción), en comparación con $31.5 millones en el segundo trimestre. Los gastos en I+D aumentaron a $32.2 millones desde $30.7 millones en el segundo trimestre.
La compañía está avanzando en múltiples ensayos de Fase 2: CANYON para la distrofia muscular de Becker (se esperan resultados preliminares en diciembre de 2024), LYNX y FOX para la distrofia muscular de Duchenne, y CIRRUS-HCM para la cardiomiopatía hipertrófica. Se informaron datos positivos de Fase 1 para EDG-7500 en sujetos sanos y en el ensayo de Fase 2 CIRRUS-HCM en pacientes con HCM obstructiva.
엣지와이즈 테라퓨틱스 (EWTX)는 2024년 3분기 재무 결과와 임상 프로그램에 대한 업데이트를 발표했습니다. 회사는 2024년 9월 30일 현재 $492.5 백만의 강력한 현금 보유고를 유지하고 있습니다. 3분기 순손실은 $34.1 백만 ($0.36 주당)으로, 2분기의 $31.5 백만과 비교됩니다. 연구개발 비용은 2분기의 $30.7 백만에서 $32.2 백만으로 증가했습니다.
회사는 여러 개의 2상 시험을 진행하고 있습니다: 베커 근육형성이상증을 위한 CANYON (2024년 12월에 주요 결과 예상), 뒤셴 근육형성이상증을 위한 LYNX 및 FOX, 비후성 심근병증을 위한 CIRRUS-HCM. 건강한 피험자에 대한 EDG-7500의 1상 데이터와 폐쇄성 HCM 환자에 대한 2상 CIRRUS-HCM 시험의 긍정적인 결과가 보고되었습니다.
Edgewise Therapeutics (EWTX) a publié les résultats financiers du troisième trimestre 2024 ainsi que des mises à jour sur ses programmes cliniques. La société maintient une solide position de trésorerie de $492.5 millions au 30 septembre 2024. La perte nette du troisième trimestre s'est élevée à $34.1 millions ($0.36 par action), contre $31.5 millions au deuxième trimestre. Les dépenses de R&D ont augmenté à $32.2 millions, contre $30.7 millions au deuxième trimestre.
La société fait avancer plusieurs essais de phase 2 : CANYON pour la dystrophie musculaire de Becker (les résultats préliminaires sont attendus en décembre 2024), LYNX et FOX pour la dystrophie musculaire de Duchenne, et CIRRUS-HCM pour la cardiomyopathie hypertrophique. Des données positives de phase 1 ont été rapportées pour EDG-7500 chez des sujets sains et pour l'essai de phase 2 CIRRUS-HCM chez des patients atteints d'HCM obstructive.
Edgewise Therapeutics (EWTX) hat die Finanzberichte für das dritte Quartal 2024 sowie Updates zu seinen klinischen Programmen veröffentlicht. Das Unternehmen hält zum 30. September 2024 eine starke Liquiditätsposition von $492.5 Millionen. Der Nettoverlust im dritten Quartal betrug $34.1 Millionen ($0.36 pro Aktie), verglichen mit $31.5 Millionen im zweiten Quartal. Die F&E-Ausgaben stiegen auf $32.2 Millionen von $30.7 Millionen im zweiten Quartal.
Das Unternehmen führt mehrere Phase-2-Studien durch: CANYON für die Becker-Muskeldystrophie (Top-Line-Ergebnisse werden für Dezember 2024 erwartet), LYNX und FOX für die Duchenne-Muskeldystrophie und CIRRUS-HCM für die hypertrophe Kardiomyopathie. Positive Phase-1-Daten wurden für EDG-7500 in gesunden Probanden und die Phase-2-CIRRUS-HCM-Studie bei Patienten mit obstruktiver HCM berichtet.
- Strong cash position of $492.5 million
- Positive Phase 1 trial results for EDG-7500 showing 67% mean reduction in resting LVOT pressure gradient
- 99% enrollment rate in MESA open-label extension trial
- FOX trial over-enrolled within two months showing strong patient interest
- Increased net loss to $34.1 million in Q3 from $31.5 million in Q2
- R&D expenses increased to $32.2 million from $30.7 million quarter-over-quarter
- G&A expenses increased to $8.2 million from $7.4 million quarter-over-quarter
Insights
Q3 2024 results show
Multiple catalysts approaching with CANYON trial results in December 2024 and CIRRUS-HCM data in Q1 2025. The company's robust pipeline development across muscular dystrophy and cardiovascular programs demonstrates efficient capital deployment.
The clinical pipeline shows strong momentum across multiple indications. Key developments include:
- CANYON trial in Becker muscular dystrophy (40 adults, 29 adolescents) reaching completion with data expected December 2024
- Impressive
99% retention rate in MESA extension trial indicates strong patient confidence - EDG-7500 showing promising early results in HCM with
67% mean reduction in resting LVOT gradient and55% reduction in provokable LVOT gradient
The rapid enrollment in the FOX trial for Duchenne patients previously treated with gene therapy suggests high unmet need and community support.
– On track to announce top-line results from Phase 2 CANYON trial of sevasemten in adults with Becker in December 2024 –
– Advanced Phase 2 LYNX and FOX trials of sevasemten in children and adolescents with Duchenne –
– Advanced Phase 2 CIRRUS-HCM trial of EDG-7500 in patients with obstructive and non-obstructive Hypertrophic Cardiomyopathy (HCM) –
– Announced positive top-line data from Phase 1 trial in healthy subjects and Phase 2 CIRRUS-HCM trial in patients with obstructive HCM –
“We continue to make strong progress on our cardiac and skeletal muscle programs,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. “Based on the strength of clinical and preclinical data to-date, we are treating patients with obstructive and non-obstructive HCM in the 28-day part of CIRRUS-HCM trial. We look forward to sharing important updates on both our CIRRUS-HCM and CANYON Phase 2 programs over the coming months.”
Recent Highlights
Muscular Dystrophy Program / Sevasemten
Becker Muscular Dystrophy (Becker)
Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to protect unstable muscle against contraction-induced muscle damage in muscular dystrophies including Becker and Duchenne. There are currently no approved therapies for individuals with Becker, a serious genetic, progressive neuromuscular disorder with significant unmet need.
CANYON Phase 2 placebo-controlled trial in adults with Becker: CANYON, the largest interventional Becker trial, includes 40 adults and 29 adolescents with a sevasemten treatment period of 12 months. The primary endpoint of CANYON is change from baseline in creatine kinase (CK) over the treatment period with additional measures collected, including North Star Ambulatory Assessment (NSAA), North Star Assessment for Muscular Dystrophies (NSAD), 100-meter timed test, biomarkers of muscle damage, MRI and patient-reported outcomes. The Company expects to report CANYON data in December 2024.
GRAND CANYON, a global pivotal cohort in Becker: GRAND CANYON, an expansion of the CANYON placebo-controlled trial, is a multi-center, randomized, double-blind, placebo-controlled cohort to evaluate the safety and efficacy of sevasemten over 18 months in 120 adults with Becker. The primary endpoint of GRAND CANYON is change from baseline in NSAA. In addition, other functional assessments, biomarkers of muscle damage, MRI, patient-reported outcomes and safety will be assessed. Data from GRAND CANYON, if positive, could support a marketing application. To learn more, go to clinicaltrials.gov (NCT05291091) or the GRAND CANYON microsite: https://www.beckergcstudy.com.
Duchenne Muscular Dystrophy (Duchenne)
LYNX Phase 2 trial in boys with Duchenne: LYNX is a 2-part multi-center, dose-finding Phase 2 trial to evaluate the effect of sevasemten on safety, PK, and biomarkers of muscle damage in children aged 4 to 9 years with Duchenne treated with oral, once-daily sevasemten. The trial will also explore changes from baseline in functional measures such as NSAA, stride velocity 95th centile (SV95C) and self-reported/caregiver-reported outcomes.
The Company plans to report LYNX data in the fourth quarter of 2024. The Company will rely on LYNX data, along with data from the FOX trial of Duchenne children previously treated with gene therapy, to guide the design and powering of a Phase 3 trial in Duchenne, planned to be initiated in 2025. For more information on LYNX go to clinicaltrials.gov to learn more about this trial (NCT05540860).
FOX Phase 2 trial in boys with Duchenne (previously treated with gene therapy): The Company is advancing FOX, a Phase 2 placebo-controlled trial to assess the effect of sevasemten over 12 weeks on safety, PK and biomarkers of muscle damage in children and adolescents aged 6 to 17 years with Duchenne who have been previously treated with gene therapy. The trial will also explore changes in baseline in functional measures such as NSAA, SV95C and self-reported/caregiver-reported outcomes. There has been exceptional enthusiasm from the Duchenne community for this trial, evident in the Company’s ability to over-enroll the trial within two months. Go to clinicaltrials.gov to learn more about this trial (NCT06100887).
Cardiovascular Program / 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. Preclinical data in models of both obstructive and non-obstructive HCM suggest the ability to drive beneficial responses with a low risk of decreasing left ventricular ejection fraction (LVEF) below normal. The Company is enrolling CIRRUS-HCM, a four-part, multi-center, open-label trial, in approximately 55 patients with HCM at up to 20 clinical sites in the
Phase 1 Trial of EDG-7500: During the quarter, the Company announced top-line data of EDG-7500 from the Phase 1 trial in healthy subjects and the single-dose arm of the Phase 2 CIRRUS-HCM trial in patients with obstructive HCM. In the placebo-controlled Phase 1 single ascending dose (SAD) trial (n=48), healthy subjects received single doses of EDG-7500, ranging from 5 to 300 mg. In the multiple ascending dose (MAD) portion of the trial (n=24), healthy subjects received 25 to 100 mg once daily for 14 days. EDG-7500 was well tolerated in both the SAD and MAD; there were no clinically meaningful changes or trends in vital signs, clinical chemistry, hematology, or electrocardiograms. There were no meaningful changes in LVEF for all SAD and MAD subjects across a broad range of EDG-7500 exposures. In the MAD, a half-life of approximately 30 hours was observed, and steady state was achieved in approximately 4 days after the start of once-daily dosing. Generally, dose proportional increases in exposure were observed in both SAD and MAD.
Phase 2 CIRRUS-HCM trial of EDG-7500: In CIRRUS-HCM Part A, patients with obstructive HCM received a single dose of 50, 100 or 200 mg of EDG-7500. A
Across the Phase 1 and CIRRUS-HCM trials, no subjects had a meaningful change to LVEF or a reduction to below
Strengthened Engagement with the Scientific and Patient Communities
The Company continued its education and outreach in the HCM and muscular dystrophy medical and patient communities. Presentations were made at the HCM Society Scientific Sessions and the 29th International Annual Congress of the World Muscle Society. The Company served in a leadership role at the Becker Education and Engagement Day (BEED) events in the US and in
Third Quarter Financial Results
Cash, cash equivalents and marketable securities were approximately
Research and development (R&D) expenses were
General and Administrative (G&A) expenses were
Net loss and net loss per share for the third quarter of 2024 was
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 first-in-class fast 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 , Facebook and Instagram.
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, Edgewise’s product candidates and programs, including sevasemten and EDG-7500; statements regarding Edgewise’s expectations relating to its clinical trials, including timing of reporting data (including the CANYON Phase 2 trial, LYNX Phase 2 trial, CIRRUS-HCM 28-day data); statements regarding the advancement of Edgewise’s research and development programs; the timing of the initiation of a Phase 3 trial of sevasemten in Duchenne; the possibility of data from GRAND CANYON to support a marketing application; statements regarding Edgewise’s pipeline of product candidates and programs; statements regarding Edgewise’s anticipated milestones; and statements by Edgewise’s President and Chief Executive 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 Edgewise’s limited operating history, its products being early in development and not having products approved for commercial sale; risks associated with Edgewise not having generated any revenue to date; Edgewise’s ability to achieve objectives relating to the discovery, development and commercialization of its product candidates, if approved; Edgewise’s need for substantial additional capital to finance its operations; Edgewise’s substantial dependence on the success of its sevasemten; Edgewise’s ability to develop and commercialize sevasemten and EDG-7500 and discover, develop and commercialize product candidates in future programs; risks related to Edgewise’s clinical trials of its product candidates not demonstrating safety and efficacy; risks related to Edgewise’s product candidates causing serious adverse events, toxicities or other undesirable side effects; the outcome of preclinical testing and early clinical trials not being predictive of the success of later clinical trials and the risks related to the results of Edgewise’s clinical trials not satisfying the requirements of regulatory authorities; delays or difficulties in the enrollment and/or maintenance of patients in clinical trials; risks related to failure to capitalize on other indications or product candidates; risks related to competition; risks relating to interim, topline and preliminary data from Edgewise’s clinical trials changing as more patient data becomes available; risks related to the regulatory approval processes being lengthy, time consuming and inherently unpredictable; risks related to regulatory authorities not accepting data from trials conducted in locations outside of their jurisdiction; risks relating to Edgewise’s ability to attract and retain highly skilled executive officers and employees; Edgewise’s ability to obtain and maintain intellectual property protection for its product candidates; Edgewise’s reliance on third parties; 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
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| Edgewise Therapeutics, Inc. | ||||||
| Condensed Statement of Operations | ||||||
| (in thousands except share and per share amounts, unaudited) | ||||||
|
|
|||||
|
Three months ended | |||||
|
September 30, 2024 |
|
June 30, 2024 | |||
| Operating expenses: |
|
|
||||
| Research and development | $ |
32,222 |
|
$ |
30,680 |
|
| General and administrative |
|
8,210 |
|
|
7,427 |
|
| Total operating expenses |
|
40,432 |
|
|
38,107 |
|
| Loss from operations |
|
(40,432 |
) |
|
(38,107 |
) |
| Interest income |
|
6,303 |
|
|
6,610 |
|
| Net loss | $ |
(34,129 |
) |
$ |
(31,497 |
) |
| Net loss per share - basic and diluted | $ |
(0.36 |
) |
$ |
(0.34 |
) |
| Weighted-average shares outstanding, basic and diluted |
|
93,813,346 |
|
|
93,515,356 |
|
| Edgewise Therapeutics, Inc. | ||||
| Condensed Balance Sheet Data | ||||
| (in thousands, unaudited) | ||||
| September 30, | December 31, | |||
2024 |
2023 |
|||
| Assets | ||||
| Cash, cash equivalents and marketable securities | $ | 492,536 |
$ | 318,393 |
| Other assets | 18,746 |
21,642 |
||
| Total assets | $ | 511,282 |
$ | 340,035 |
| Liabilities and stockholders' equity | ||||
| Liabilities | 22,405 |
21,205 |
||
| Stockholders' equity | 488,877 |
318,830 |
||
| Total liabilities and stockholders' equity | $ | 511,282 |
$ | 340,035 |
View source version on businesswire.com: https://www.businesswire.com/news/home/20241107168709/en/
Edgewise Contacts
Investors:
Michael Carruthers, Chief Financial Officer
ir@edgewisetx.com
Media:
Maureen Franco, VP Corporate Communications
media@edgewisetx.com
Source: Edgewise Therapeutics
FAQ
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