Verve Therapeutics Announces Clearance of Investigational New Drug Application by the U.S. FDA for VERVE-102, an Investigational Gene Editing Medicine Designed to Durably Lower Cholesterol After a Single Dose
Verve Therapeutics has received FDA clearance for its Investigational New Drug (IND) application for VERVE-102, a novel gene editing medicine targeting cardiovascular disease. The treatment is designed as a single-course therapy to inactivate the PCSK9 gene in the liver, aiming to permanently lower blood LDL cholesterol levels.
The ongoing Heart-2 Phase 1b clinical trial has shown promising interim results across three dose cohorts (0.3 mg/kg, 0.45 mg/kg, and 0.6 mg/kg), with no treatment-related serious adverse events reported as of January 10, 2025. The company plans to announce initial safety and efficacy data in Q2 2025, with final dose escalation data expected in H2 2025.
The treatment targets patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD). Verve will begin activating U.S. trial sites and plans to deliver an opt-in data package to Eli Lilly while initiating Phase 2 clinical trials in the second half of 2025.
Verve Therapeutics ha ricevuto l'approvazione della FDA per la sua domanda di Nuovo Farmaco Investigativo (IND) per VERVE-102, un nuovo farmaco di editing genetico mirato alle malattie cardiovascolari. Il trattamento è progettato come una terapia a corso singolo per inattivare il gene PCSK9 nel fegato, con l'obiettivo di ridurre permanentemente i livelli di colesterolo LDL nel sangue.
Lo studio clinico in corso Heart-2 Phase 1b ha mostrato risultati promettenti in fase intermedia su tre coorti di dosaggio (0.3 mg/kg, 0.45 mg/kg e 0.6 mg/kg), senza eventi avversi gravi correlati al trattamento riportati fino al 10 gennaio 2025. L'azienda prevede di annunciare i dati iniziali di sicurezza ed efficacia nel secondo trimestre del 2025, con i dati finali di escalation della dose attesi nel secondo semestre del 2025.
Il trattamento è destinato a pazienti con ipercolesterolemia familiare eterozigote (HeFH) e/o malattia coronarica prematura (CAD). Verve inizierà ad attivare i siti di sperimentazione negli Stati Uniti e prevede di fornire un pacchetto dati opt-in a Eli Lilly mentre avvia gli studi clinici di fase 2 nella seconda metà del 2025.
Verve Therapeutics ha recibido la aprobación de la FDA para su solicitud de Nuevo Medicamento en Investigación (IND) para VERVE-102, un nuevo medicamento de edición genética dirigido a enfermedades cardiovasculares. El tratamiento está diseñado como una terapia de curso único para inactivar el gen PCSK9 en el hígado, con el objetivo de reducir permanentemente los niveles de colesterol LDL en la sangre.
El ensayo clínico en curso Heart-2 Phase 1b ha mostrado resultados intermedios prometedores en tres cohortes de dosis (0.3 mg/kg, 0.45 mg/kg y 0.6 mg/kg), sin eventos adversos graves relacionados con el tratamiento reportados hasta el 10 de enero de 2025. La compañía planea anunciar datos iniciales de seguridad y eficacia en el segundo trimestre de 2025, con datos finales de escalada de dosis esperados en la segunda mitad de 2025.
El tratamiento está dirigido a pacientes con hipercolesterolemia familiar heterocigota (HeFH) y/o enfermedad coronaria prematura (CAD). Verve comenzará a activar los sitios de ensayo en EE. UU. y planea entregar un paquete de datos de opción a Eli Lilly mientras inicia ensayos clínicos de fase 2 en la segunda mitad de 2025.
Verve Therapeutics는 심혈관 질환을 목표로 하는 새로운 유전자 편집 의약품 VERVE-102에 대한 임상시험 신약(IND) 신청이 FDA의 승인을 받았습니다. 이 치료법은 간에서 PCSK9 유전자를 비활성화하여 혈중 LDL 콜레스테롤 수치를 영구적으로 낮추기 위해 설계된 단일 치료 코스입니다.
진행 중인 Heart-2 1b 단계 임상 시험은 3개의 용량 코호트(0.3 mg/kg, 0.45 mg/kg 및 0.6 mg/kg)에서 유망한 중간 결과를 보여주었으며, 2025년 1월 10일 기준으로 치료와 관련된 심각한 부작용은 보고되지 않았습니다. 이 회사는 2025년 2분기에 초기 안전성 및 효능 데이터를 발표할 계획이며, 최종 용량 증가 데이터는 2025년 하반기에 예상됩니다.
이 치료는 이형접합 가족성 고콜레스테롤혈증(HeFH) 및/또는 조기 관상동맥 질환(CAD) 환자를 대상으로 합니다. Verve는 미국 시험 사이트를 활성화하기 시작하고, 2025년 하반기에 2상 임상 시험을 시작하면서 Eli Lilly에 선택적 데이터 패키지를 제공할 계획입니다.
Verve Therapeutics a reçu l'approbation de la FDA pour sa demande de Médicament Nouveau Investigational (IND) pour VERVE-102, un nouveau médicament d'édition génique ciblant les maladies cardiovasculaires. Le traitement est conçu comme une thérapie à dose unique pour inactiver le gène PCSK9 dans le foie, visant à réduire de manière permanente les niveaux de cholestérol LDL dans le sang.
L'essai clinique en cours Heart-2 Phase 1b a montré des résultats intermédiaires prometteurs dans trois cohortes de dose (0,3 mg/kg, 0,45 mg/kg et 0,6 mg/kg), sans événements indésirables graves liés au traitement signalés jusqu'au 10 janvier 2025. La société prévoit d'annoncer des données initiales de sécurité et d'efficacité au deuxième trimestre de 2025, avec des données finales d'escalade de dose attendues au second semestre de 2025.
Le traitement cible les patients atteints d'hypercholestérolémie familiale hétérozygote (HeFH) et/ou de maladie coronarienne précoce (CAD). Verve commencera à activer des sites d'essai aux États-Unis et prévoit de fournir un paquet de données opt-in à Eli Lilly tout en lançant des essais cliniques de phase 2 dans la seconde moitié de 2025.
Verve Therapeutics hat von der FDA die Genehmigung für seinen Antrag auf ein Neues Investigational Drug (IND) für VERVE-102 erhalten, ein neuartiges Genbearbeitungsmedikament, das auf Herz-Kreislauf-Erkrankungen abzielt. Die Behandlung ist als Einzeltherapie konzipiert, um das PCSK9-Gen in der Leber zu inaktivieren, mit dem Ziel, die LDL-Cholesterinwerte im Blut dauerhaft zu senken.
Die laufende Heart-2 Phase 1b klinische Studie hat vielversprechende Zwischenresultate in drei Dosisgruppen (0,3 mg/kg, 0,45 mg/kg und 0,6 mg/kg) gezeigt, ohne dass bis zum 10. Januar 2025 behandlungsbedingte schwerwiegende unerwünschte Ereignisse gemeldet wurden. Das Unternehmen plant, im 2. Quartal 2025 erste Sicherheits- und Wirksamkeitsdaten bekannt zu geben, während die endgültigen Dosissteigerungsdaten für das 2. Halbjahr 2025 erwartet werden.
Die Behandlung richtet sich an Patienten mit heterozygotem familiärem Hypercholesterinämie (HeFH) und/oder vorzeitiger koronarer Herzkrankheit (CAD). Verve wird beginnen, US-Studienstandorte zu aktivieren und plant, ein Opt-in-Datenpaket an Eli Lilly zu liefern, während es in der zweiten Hälfte des Jahres 2025 mit Phase-2-Studien beginnt.
- FDA clearance of IND application enables U.S. clinical trials expansion
- Positive initial safety data with no serious adverse events reported
- Treatment designed as single-dose therapy for lifelong cholesterol reduction
- Strategic partnership with Eli Lilly for PCSK9 program
- Efficacy data not yet available
- Early-stage clinical development with uncertain outcome
- Complex regulatory pathway ahead for novel gene editing therapy
Insights
Verve's IND clearance for VERVE-102 represents a significant regulatory milestone that validates their gene editing approach to cardiovascular disease. This in vivo base editing therapy targeting PCSK9 could potentially address a critical unmet need in cardiovascular medicine - the challenge of medication adherence.
The preliminary safety data from the first three dose cohorts (up to 0.6 mg/kg) appears encouraging with no treatment-related serious adverse events reported. While efficacy data remains pending until Q2 2025, the clean safety profile bodes well for future development.
VERVE-102's differentiated mechanism aims to provide durable LDL-C reduction after a single treatment - a potentially revolutionary approach compared to current therapies requiring ongoing administration. This positions Verve's platform as disruptive in a market dominated by statins, PCSK9 inhibitors, and other chronic therapies.
The FDA clearance accelerates Verve's development timeline by enabling parallel trial operations across international sites and the US. Looking ahead, the company has established clear clinical milestones with initial efficacy data expected in Q2 and the critical Eli Lilly opt-in package planned for H2 2025.
This FDA clearance substantially derisks Verve's lead program and enhances the company's strategic position with multiple catalysts ahead in 2025. For a clinical-stage biotech with
The IND clearance specifically enables US trial expansion, critical for both future commercialization pathways and potential partnership leverage. The existing Eli Lilly collaboration adds particular significance, as positive data could trigger the opt-in milestone later this year.
Verve's approach targets an enormous market opportunity. Cardiovascular disease remains the leading cause of death globally, and current therapies suffer from adherence issues that VERVE-102 directly addresses through its single-dose paradigm. This positions the company's technology to potentially capture significant value from improved treatment outcomes.
The clinical timeline clarity provides investors with defined value-inflection points: data readouts in Q2, full dose-escalation results in H2, the Lilly opt-in decision, and Phase 2 initiation - all within 2025. For a company at this stage, this regulatory milestone effectively enables this accelerated development pathway while validating their novel therapeutic approach.
BOSTON, March 24, 2025 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage company developing a new class of genetic medicines for cardiovascular disease, today announced the clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for VERVE-102 for the treatment of patients living with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD). VERVE-102 is a novel, investigational in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower blood low-density lipoprotein cholesterol (LDL-C).
“The IND clearance from the U.S. FDA represents an important step in our journey to advance a new class of in vivo gene editing medicines for people worldwide living with cardiovascular disease,” said Sekar Kathiresan, M.D., co-founder and chief executive officer of Verve Therapeutics. “There are multiple cholesterol lowering medicines currently available that can lower LDL-C at a single time point; however, time on treatment for these medicines remains low. This is a significant problem as heart attack risk reduction tracks most closely with cumulative cholesterol reduction, a function of both the magnitude of cholesterol reduction and time on treatment. To address this unmet need, Verve’s medicines are designed to deliver lifelong cholesterol lowering after a single course of treatment and, consequently, drive more meaningful efficacy. With our ongoing Heart-2 clinical trial for VERVE-102 progressing internationally, we are excited to begin activating trial sites in the U.S. as we expect it to play a key role in our continued clinical development.”
As part of the IND submission, Verve provided the FDA with interim clinical data from the dose-escalation portion of the ongoing Heart-2 Phase 1b clinical trial for VERVE-102. The Heart-2 clinical trial is evaluating the safety and tolerability of VERVE-102 in patients living with HeFH and/or premature CAD, with additional analyses for pharmacokinetics and changes in blood PCSK9 protein and LDL-C levels and is currently being conducted at sites outside of the U.S. Data submitted to the FDA had a cut-off date of January 10, 2025, and included participants across the first three dose cohorts (0.3 mg/kg, 0.45 mg/kg, and 0.6 mg/kg). As of the data cut-off date, VERVE-102 has been well-tolerated, with no treatment-related serious adverse events and no clinically significant laboratory abnormalities observed.
In the second quarter of 2025, Verve expects to announce demographic and initial safety and efficacy data from the Heart-2 clinical trial. Enrollment in the Heart-2 clinical trial is progressing well, and the initial data set is expected to include participants across the first three dose cohorts (0.3 mg/kg, 0.45 mg/kg, and 0.6 mg/kg) with at least 28 days of follow-up for each participant.
In addition, in the second half of 2025, Verve remains on track to report the final data for the dose escalation portion of the Heart-2 clinical trial, deliver the opt-in data package for the PCSK9 program to Eli Lilly and Company (Lilly), and initiate the Phase 2 clinical trial for the PCSK9 program.
About Verve Therapeutics
Verve Therapeutics, Inc. (Nasdaq: VERV) is a clinical-stage company developing a new class of genetic medicines for cardiovascular disease with the potential to transform treatment from chronic therapies to single-course gene editing medicines. The company’s lead programs –VERVE-102, VERVE-201, and VERVE-301 – target the three cholesterol drivers of atherosclerosis: LDL-C, remnant cholesterol, and Lp(a). VERVE-102 is designed to permanently turn off the PCSK9 gene in the liver and is being developed initially for heterozygous familial hypercholesterolemia (HeFH) and ultimately to treat patients with established atherosclerotic cardiovascular disease (ASCVD) who continue to be impacted by high LDL-C levels. VERVE-201 is designed to permanently turn off the ANGPTL3 gene in the liver and is initially being developed for refractory hypercholesterolemia, where patients still have high LDL-C despite treatment with maximally tolerated standard of care therapies, and homozygous familial hypercholesterolemia (HoFH). VERVE-301 is designed to permanently turn off the LPA gene to reduce Lp(a) levels. Lp(a) is a genetically validated, independent risk factor for ASCVD, ischemic stroke, thrombosis, and aortic stenosis. For more information, please visit www.VerveTx.com.
Cautionary Note Regarding Forward Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the company’s ongoing Heart-2 clinical trial; the timing and availability of data for the Heart-2 trial and timing for initiation of the Phase 2 clinical trial for the PCSK9 program; the timing of updates for the PCSK9 program; the timing of delivery of the opt-in data package to Lilly; and the potential advantages and therapeutic potential of the company’s programs. All statements, other than statements of historical facts, contained in this press release, including statements regarding the company’s strategy, future operations, future financial position, prospects, plans and objectives of management, are forward-looking statements. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the company’s limited operating history; the company’s ability to timely submit and receive approvals of regulatory applications for its product candidates; advance its product candidates in preclinical studies and clinical trials; initiate, enroll and complete its ongoing and future clinical trials on the timeline expected or at all; correctly estimate the potential patient population and/or market for the company’s product candidates; replicate in clinical trials positive results found in preclinical studies and/or earlier-stage clinical trials of VERVE-101, VERVE-102, and VERVE-201; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; obtain, maintain or protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the company’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in the company’s most recent filings with the Securities and Exchange Commission and in other filings that the company makes with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the company’s views as of the date hereof and should not be relied upon as representing the company’s views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company’s views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so.
Investor Contact
Jen Robinson
Verve Therapeutics, Inc.
jrobinson@vervetx.com
Media Contact
Ashlea Kosikowski
1AB
ashlea@1abmedia.com
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