Verve Therapeutics Receives U.S. FDA Fast Track Designation for VERVE-102, an In Vivo Base Editing Medicine Targeting PCSK9
Verve Therapeutics has received FDA Fast Track designation for VERVE-102, an investigational base editing medicine targeting PCSK9 for treating hyperlipidemia patients with high cardiovascular risk. VERVE-102 is designed as a single-course treatment to permanently deactivate the PCSK9 gene in the liver and reduce LDL-C levels.
The treatment is currently in Phase 1b Heart-2 clinical trial, evaluating safety and tolerability in patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD). The company plans to announce initial safety and efficacy data from the first three dose cohorts (0.3, 0.45, and 0.6 mg/kg) in Q2 2025.
The Fast Track status may enable more frequent FDA communications and potential Priority Review. Verve plans to report final dose escalation data, deliver the opt-in package to Eli Lilly, and begin Phase 2 trials in H2 2025.
Verve Therapeutics ha ricevuto la designazione di Fast Track dalla FDA per VERVE-102, un farmaco sperimentale di editing genetico mirato al PCSK9 per il trattamento di pazienti con iperlipidemia ad alto rischio cardiovascolare. VERVE-102 è concepito come un trattamento a singola somministrazione per disattivare permanentemente il gene PCSK9 nel fegato e ridurre i livelli di LDL-C.
Il trattamento è attualmente in fase 1b del trial clinico Heart-2, che valuta la sicurezza e la tollerabilità in pazienti con ipercolesterolemia familiare eterozigote (HeFH) e/o malattia coronarica prematura (CAD). L'azienda prevede di annunciare i dati iniziali di sicurezza ed efficacia dai primi tre gruppi di dosaggio (0,3, 0,45 e 0,6 mg/kg) nel secondo trimestre del 2025.
Lo status di Fast Track potrebbe consentire comunicazioni più frequenti con la FDA e una potenziale Revisione Prioritaria. Verve prevede di riportare i dati finali sull'aumento della dose, consegnare il pacchetto di opt-in a Eli Lilly e iniziare gli studi di fase 2 nel secondo semestre del 2025.
Verve Therapeutics ha recibido la designación de Fast Track por parte de la FDA para VERVE-102, un medicamento de edición genética en investigación que tiene como objetivo el PCSK9 para tratar a pacientes con hiperlipidemia y alto riesgo cardiovascular. VERVE-102 está diseñado como un tratamiento de una sola dosis para desactivar permanentemente el gen PCSK9 en el hígado y reducir los niveles de LDL-C.
El tratamiento se encuentra actualmente en la fase 1b del ensayo clínico Heart-2, evaluando la seguridad y tolerabilidad en pacientes con hipercolesterolemia familiar heterocigota (HeFH) y/o enfermedad coronaria prematura (CAD). La empresa planea anunciar los datos iniciales de seguridad y eficacia de los primeros tres grupos de dosis (0.3, 0.45 y 0.6 mg/kg) en el segundo trimestre de 2025.
El estatus de Fast Track puede permitir comunicaciones más frecuentes con la FDA y una posible Revisión Prioritaria. Verve planea informar sobre los datos finales de escalado de dosis, entregar el paquete de opt-in a Eli Lilly y comenzar los ensayos de fase 2 en la segunda mitad de 2025.
Verve Therapeutics는 고위험 심혈관 질환 환자의 고지혈증 치료를 위한 PCSK9을 표적으로 하는 실험적인 유전자 편집 약물 VERVE-102에 대해 FDA의 패스트 트랙 지정을 받았습니다. VERVE-102는 간에서 PCSK9 유전자를 영구적으로 비활성화하고 LDL-C 수치를 감소시키기 위해 단일 치료 과정으로 설계되었습니다.
현재 이 치료는 이형접합 가족성 고콜레스테롤혈증(HeFH) 및/또는 조기 관상동맥 질환(CAD) 환자에서 안전성과 내약성을 평가하는 Heart-2 임상 시험 1b 단계에 있습니다. 이 회사는 2025년 2분기에 첫 세 가지 용량 집단(0.3, 0.45 및 0.6 mg/kg)에서 초기 안전성 및 유효성 데이터를 발표할 계획입니다.
패스트 트랙 상태는 FDA와의 보다 빈번한 커뮤니케이션 및 잠재적인 우선 검토를 가능하게 할 수 있습니다. Verve는 최종 용량 증가 데이터를 보고하고 Eli Lilly에 옵트인 패키지를 전달하며 2025년 하반기에 2상 시험을 시작할 계획입니다.
Verve Therapeutics a reçu la désignation Fast Track de la FDA pour VERVE-102, un médicament expérimentale d'édition de base ciblant le PCSK9 pour traiter les patients atteints d'hyperlipidémie à haut risque cardiovasculaire. VERVE-102 est conçu comme un traitement à dose unique pour désactiver de manière permanente le gène PCSK9 dans le foie et réduire les niveaux de LDL-C.
Le traitement est actuellement en phase 1b de l'
Le statut Fast Track pourrait permettre des communications plus fréquentes avec la FDA et une éventuelle révision prioritaire. Verve prévoit de rapporter les données finales sur l'escalade des doses, de livrer le package d'option à Eli Lilly et de commencer les essais de phase 2 au second semestre 2025.
Verve Therapeutics hat von der FDA die Fast Track-Zulassung für VERVE-102 erhalten, ein experimentelles Medikament zur Basiseditierung, das auf PCSK9 abzielt, um Patienten mit Hyperlipidämie und hohem kardiovaskulären Risiko zu behandeln. VERVE-102 ist als eine einmalige Behandlung konzipiert, um das PCSK9-Gen in der Leber dauerhaft zu deaktivieren und die LDL-C-Spiegel zu senken.
Die Behandlung befindet sich derzeit in der Phase 1b der Heart-2-Studie, die Sicherheit und Verträglichkeit bei Patienten mit heterozygotem familiären Hypercholesterinämie (HeFH) und/oder vorzeitiger koronarer Herzkrankheit (CAD) bewertet. Das Unternehmen plant, im zweiten Quartal 2025 erste Sicherheits- und Wirksamkeitsdaten aus den ersten drei Dosierungsgruppen (0,3, 0,45 und 0,6 mg/kg) bekannt zu geben.
Der Status als Fast Track könnte häufigere Kommunikationen mit der FDA und eine mögliche Prioritätsprüfung ermöglichen. Verve plant, die endgültigen Daten zur Dosissteigerung zu berichten, das Opt-in-Paket an Eli Lilly zu übergeben und im zweiten Halbjahr 2025 mit Phase-2-Studien zu beginnen.
- FDA Fast Track designation received, potentially accelerating development and review process
- Phase 1b trial progressing with three dose cohorts
- Partnership with Eli Lilly for PCSK9 program
- Clear development timeline with multiple catalysts in 2025
- Product still in early clinical phase with unproven efficacy
- Final trial results and FDA approval not guaranteed
- Competitive market for LDL-C lowering treatments
Insights
The FDA Fast Track designation for VERVE-102 represents a significant regulatory milestone for Verve Therapeutics that could accelerate their development timeline. This designation acknowledges both the seriousness of hyperlipidemia and the potential of VERVE-102 to address an unmet medical need in cardiovascular disease management.
What makes this particularly valuable is that Fast Track provides several tangible benefits: more frequent FDA interactions, potential for rolling review, and possible eligibility for Priority Review. For a small-cap biotech (
VERVE-102's mechanism - a one-time base editing treatment targeting PCSK9 - addresses a critical limitation in current hyperlipidemia management where nearly
The upcoming data readouts represent critical inflection points: Q2 2025 will provide the first meaningful look at both safety and efficacy across multiple dosing cohorts, while H2 2025 brings the Eli Lilly opt-in decision point. This partnership structure with Lilly provides validation from a major pharmaceutical company while maintaining significant upside potential if the data proves compelling.
BOSTON, April 11, 2025 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage company developing a new class of genetic medicines for cardiovascular disease, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for VERVE-102 for the treatment of patient groups with hyperlipidemia and high lifetime cardiovascular risk to reduce low-density lipoprotein cholesterol (LDL-C). VERVE-102 is the company’s novel, in vivo, investigational base editing medicine designed to be a single-course treatment that permanently turns off the PCSK9 gene in the liver and durably reduces disease-driving LDL-C. VERVE-102 is currently being tested in the Phase 1b Heart-2 clinical trial, which is designed to evaluate the safety and tolerability of VERVE-102 administration in adult patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD) who require additional lowering of LDL-C.
“Despite available treatment options to lower LDL-C, there remains a pressing need to provide sustained LDL-C lowering and thereby, improve efficacy," said Sekar Kathiresan, M.D., co-founder and chief executive officer of Verve Therapeutics. “Nearly
The FDA’s Fast Track designation is designed to facilitate the development and expedite the review of drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address an unmet medical need. A drug that receives Fast Track designation may be eligible for more frequent meetings and communications with the FDA and rolling review of any application for marketing approval. A drug receiving Fast Track designation also may be eligible for Priority Review if relevant criteria are met.
In the second quarter of 2025, Verve expects to announce demographic and initial safety and efficacy data from the Heart-2 clinical trial. 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 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 delivery of the opt-in package to Lilly; the potential advantages and therapeutic potential of the company’s programs; and expectations regarding the potential benefits of Fast Track designation. 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 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
FAQ
What does FDA Fast Track designation mean for VERV's VERVE-102 development timeline?
When will Verve Therapeutics (VERV) release initial Heart-2 trial data for VERVE-102?
What patient populations are targeted in VERV's VERVE-102 Heart-2 clinical trial?
How does VERVE-102 differ from current LDL-C lowering treatments?
What are the next major milestones for VERV's PCSK9 program in 2025?
