Verve Therapeutics Announces Pipeline Progress and Reports Third Quarter 2024 Financial Results
Verve Therapeutics reported progress in its clinical trials and Q3 2024 financial results. Seven participants were dosed in the Heart-2 Phase 1b trial of VERVE-102, with initial data expected in H1 2025. The first participant was dosed in the Pulse-1 Phase 1b trial of VERVE-201. The company reported $539.9M in cash and equivalents, with runway through 2026. Q3 financial results showed collaboration revenue of $6.9M, R&D expenses of $49.9M, and a net loss of $50.1M ($0.59 per share). VERVE-102 has been well-tolerated with no serious adverse events observed.
Verve Therapeutics ha riportato progressi nei suoi studi clinici e risultati finanziari per il terzo trimestre del 2024. Sette partecipanti hanno ricevuto il trattamento nello studio Heart-2 di Fase 1b di VERVE-102, con dati iniziali attesi nella prima metà del 2025. Il primo partecipante è stato trattato nello studio Pulse-1 di Fase 1b di VERVE-201. L'azienda ha segnalato 539,9 milioni di dollari in contante e equivalenti, con fondi disponibili fino al 2026. I risultati finanziari del terzo trimestre hanno mostrato ricavi da collaborazioni di 6,9 milioni di dollari, spese per ricerca e sviluppo di 49,9 milioni di dollari e una perdita netta di 50,1 milioni di dollari (0,59 dollari per azione). VERVE-102 è stato ben tollerato senza eventi avversi seri osservati.
Verve Therapeutics reportó avances en sus ensayos clínicos y resultados financieros del tercer trimestre de 2024. Siete participantes recibieron la dosis en el ensayo Heart-2 de Fase 1b de VERVE-102, con datos iniciales esperados para la primera mitad de 2025. El primer participante fue dosificado en el ensayo Pulse-1 de Fase 1b de VERVE-201. La empresa reportó $539.9 millones en efectivo y equivalentes, con fondos disponibles hasta 2026. Los resultados financieros del tercer trimestre mostraron ingresos por colaboración de $6.9 millones, gastos en I+D de $49.9 millones y una pérdida neta de $50.1 millones ($0.59 por acción). VERVE-102 ha sido bien tolerado, sin eventos adversos graves observados.
Verve Therapeutics는 임상 시험의 진전과 2024년 3분기 재무 결과를 보고했습니다. VERVE-102의 Heart-2 1b 단계 시험에서 7명의 참가자가 투여받았으며, 초기 데이터는 2025년 상반기에 예상됩니다. VERVE-201의 Pulse-1 1b 단계 시험에서 첫 번째 참가자가 투여되었습니다. 회사는 5억 3천 9백 90만 달러의 현금 및 현금성 자산을 보고했으며, 2026년까지 자금을 확보하고 있습니다. 3분기 재무 결과는 협력 수익이 690만 달러, 연구개발 비용이 4천 9백 90만 달러, 순손실이 5천 1백 만 달러(주당 0.59 달러)로 나타났습니다. VERVE-102는 잘 견뎌졌으며 심각한 부작용은 관찰되지 않았습니다.
Verve Therapeutics a rapporté des avancées dans ses essais cliniques et les résultats financiers du troisième trimestre 2024. Sept participants ont été dosés dans l'essai Heart-2 de phase 1b de VERVE-102, avec des données initiales prévues pour le premier semestre 2025. Le premier participant a été dosé dans l'essai Pulse-1 de phase 1b de VERVE-201. L'entreprise a signalé 539,9 millions de dollars en liquidités et équivalents, avec des ressources suffisantes jusqu'en 2026. Les résultats financiers du troisième trimestre ont montré des revenus de collaboration de 6,9 millions de dollars, des dépenses de R&D de 49,9 millions de dollars et une perte nette de 50,1 millions de dollars (0,59 dollar par action). VERVE-102 a été bien toléré, sans événements indésirables graves observés.
Verve Therapeutics berichtete über Fortschritte in seinen klinischen Studien und die finanziellen Ergebnisse für das dritte Quartal 2024. Sieben Teilnehmer wurden in der Heart-2 Phase 1b Studie mit VERVE-102 behandelt, wobei erste Daten für die erste Hälfte von 2025 erwartet werden. Der erste Teilnehmer wurde in der Pulse-1 Phase 1b Studie mit VERVE-201 dosiert. Das Unternehmen berichtete über 539,9 Millionen Dollar in Bar- und Barmitteläquivalenten, mit ausreichenden Mitteln bis 2026. Die finanziellen Ergebnisse für das dritte Quartal zeigten Kooperationsumsätze von 6,9 Millionen Dollar, F&E-Aufwendungen von 49,9 Millionen Dollar und einen Nettoverlust von 50,1 Millionen Dollar (0,59 Dollar pro Aktie). VERVE-102 wurde gut vertragen, ohne beobachtete schwerwiegende unerwünschte Ereignisse.
- Strong cash position of $539.9M with runway through 2026
- Collaboration revenue increased to $6.9M from $3.1M YoY
- VERVE-102 showing positive safety profile with no serious adverse events
- Successful progression in clinical trials with 7 participants dosed in Heart-2 trial
- Net loss increased to $50.1M from $45.8M YoY
- R&D expenses increased to $49.9M from $43.8M YoY
- G&A expenses rose to $13.8M from $11.7M YoY
Insights
The Q3 results and pipeline updates reveal significant progress in Verve's gene editing programs. Key highlights include:
The Heart-2 trial for VERVE-102 shows promising early safety data with 7 participants dosed and no serious adverse events. The VERVE-101 durability data demonstrates sustained LDL-C reductions of
While cash burn remains high, the strong cash position and expanding clinical pipeline position Verve well for multiple catalysts in 2025, including Heart-2 initial data and Phase 2 PCSK9 program initiation.
Seven participants across two cohorts dosed in the Heart-2 Phase 1b clinical trial of VERVE-102 targeting PCSK9; Initial data planned for the first half of 2025
First participant dosed in the Pulse-1 Phase 1b clinical trial of VERVE-201 targeting ANGPTL3; Regulatory clearances in Australia, Canada, and the U.K.
Cash, cash equivalents, and marketable securities of
BOSTON, Nov. 05, 2024 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage company developing a new class of genetic medicines for cardiovascular disease, today reported pipeline updates and financial results for the quarter ended September 30, 2024.
“In the third quarter, we made considerable progress towards our mission to develop a new class of genetic medicines where a one-time treatment leads to lifelong lowering of blood cholesterol,” said Sekar Kathiresan, M.D., co-founder and chief executive officer of Verve Therapeutics. “We continue to execute on the Heart-2 clinical trial and are pleased to share that as of October 29, 2024, seven participants have been dosed. VERVE-102 has been well-tolerated, with no serious adverse events and no clinically significant laboratory abnormalities observed. We look forward to providing initial data from the Heart-2 clinical trial and an update on the PCSK9 program in the first half of 2025.”
Dr. Kathiresan continued, “In addition, we are excited to announce that the first participant in the Pulse-1 Phase 1b clinical trial for our ANGPTL3 product candidate, VERVE-201, was recently dosed. With cash runway through 2026, we are well-positioned to execute additional important milestones across our pipeline and advance our early-stage programs, including our program targeting LPA. With two product candidates being tested in the clinic, we expect 2025 to be an eventful year for Verve as we develop a new approach for the treatment of cardiovascular disease.”
PCSK9 Program
Enrollment Ongoing in Heart-2 Phase 1b Clinical Trial Evaluating VERVE-102
- VERVE-102 is a novel, investigational gene editing medicine designed to be a single course treatment that permanently turns off the PCSK9 gene in the liver and durably reduces disease-driving low-density lipoprotein cholesterol (LDL-C). VERVE-102 consists of messenger RNA expressing an adenine base editor and an optimized guide RNA targeting the PCSK9 gene identical to VERVE-101, the company’s initial PCSK9 product candidate that showed proof-of concept for this mechanism. However, compared to VERVE-101, VERVE-102 uses a different lipid nanoparticle (LNP) delivery system, which includes a different ionizable lipid and Verve’s proprietary GalNAc liver-targeting ligand, designed to allow the LNP to access liver cells using either the low-density lipoprotein receptor (LDLR) or the asialoglycoprotein receptor (ASGPR).
- VERVE-102 is being evaluated in the Heart-2 open-label Phase 1b clinical trial in two patient populations who require deep and durable reductions of LDL-C levels in the blood: adults living with heterozygous familial hypercholesterolemia (HeFH) and adults living with premature coronary artery disease (CAD). The Heart-2 clinical trial is expected to include four dose cohorts, each comprised of three to nine patients with either HeFH or premature CAD.
- As of October 29, 2024, dosing has been completed in seven participants in the first two dose cohorts, 0.3 mg/kg and 0.45 mg/kg, in the Heart-2 clinical trial. VERVE-102 has been well-tolerated. No serious adverse events and no clinically significant laboratory abnormalities have been observed. Following the standard review from the independent data and safety monitoring board (DSMB), the company expects to continue the dose escalation portion of the clinical trial.
- Verve recently received clearance of its Clinical Trial Applications (CTAs) for the Heart-2 clinical trial in Israel and New Zealand. Enrollment remains ongoing in Australia, Canada, and the U.K.
- In November 2024, Verve plans to present a moderated digital poster at the American Heart Association (AHA) Scientific Sessions describing the design of the Heart-2 Phase 1b clinical trial.
- Verve expects to provide initial data from the Heart-2 clinical trial and an update on the PCSK9 program in the first half of 2025. The company also plans to initiate the Phase 2 clinical trial for the PCSK9 program in the second half of 2025.
Analyses of Heart-1 Phase 1b Clinical Trial of VERVE-101
- Verve recently reported updated durability data from the Heart-1 clinical trial of VERVE-101 at the European Society of Gene and Cell Therapy (ESGCT) 2024 Congress and the American Society of Nephrology Kidney Week 2024 Meeting. Mean, time-averaged PCSK9 protein reductions of greater than
60% were observed in each of the two higher dose cohorts (0.45 mg/kg and 0.6 mg/kg), and mean, time-averaged LDL-C reductions of42% at 0.45 mg/kg (n=6) and time-averaged LDL-C reduction of57% at 0.6 mg/kg (n=1) were observed. In the single participant in the highest dose cohort, LDL-C reduction has now been sustained out to 18 months after the single dose. No new treatment related adverse events have been reported since March 2024. Verve believes that these new durability data further support the potential of once-and-done gene editing medicines for the treatment of cardiovascular disease.
- Verve has completed a series of nonclinical studies as part of its investigation into the previously disclosed laboratory abnormalities observed with VERVE-101. In order to isolate the role of the LNP and determine whether the laboratory abnormalities observed in the Heart-1 clinical trial were due to the LNP delivery system, these studies used a version of VERVE-101 with a non-targeting guide RNA designed to preclude base editing. Data from these studies continue to support Verve’s understanding that the LNP in VERVE-101 is likely the primary driver of the laboratory abnormalities observed in the Heart-1 clinical trial. The Heart-1 clinical trial will remain paused during the dose escalation portion of the Heart-2 clinical trial evaluating VERVE-102.
ANGPTL3 Program
First Participant Dosed with VERVE-201 in the Pulse-1 Phase 1b Clinical Trial
- VERVE-201 is a novel, investigational gene editing medicine designed to be a single course treatment that permanently turns off the ANGPTL3 gene in the liver to reduce disease-driving LDL-C as well as remnant cholesterol and utilizes Verve’s proprietary GalNAc-LNP delivery technology. VERVE-201 is being developed in two patient populations: patients with refractory hypercholesterolemia (RH), defined as those who are unable to achieve adequate LDL-C reduction with maximally tolerated standard of care therapies, potentially including PCSK9 inhibitors, and patients living with homozygous familial hypercholesterolemia (HoFH), a rare and often fatal inherited cause of premature ASCVD characterized by extremely high blood LDL-C. The aim of this medicine is to reduce the heavy treatment burden associated with available therapies, including the requirement for multiple oral, injectable, and intravenous infusions, often administered over decades.
- Verve announced today that the first participant has been dosed with VERVE-201 in its Pulse-1 Phase 1b clinical trial.
The Pulse-1 clinical trial is designed to evaluate the safety and tolerability of VERVE-201 administration in adult patients with RH who require additional lowering of LDL-C despite treatment with maximally tolerated standard of care therapies, potentially including PCSK9 inhibitors. Endpoints also include pharmacokinetics and changes in blood ANGPTL3 protein and LDL-C levels. The Pulse-1 clinical trial is a single-ascending dose study that has an adaptive design. The Pulse-1 clinical trial is expected to include four dose cohorts each comprised of three to nine patients with RH.
- Verve recently received regulatory clearances to initiate the Pulse-1 clinical trial in Australia, Canada, and the U.K.
Upcoming Investor Events
Verve plans to participate in fireside chats during the following upcoming investor events:
- Guggenheim's Inaugural Healthcare Innovation Conference, November 11 at 4:00 PM ET, Boston, MA
- Stifel 2024 Healthcare Conference, November 18 at 3:00 PM ET, New York, NY
- Jefferies London Healthcare Conference, November 20 at 4:00 PM GMT, London, UK
Upcoming Medical Meeting Presentations
Verve plans to present a moderated digital poster at the American Heart Association (AHA) Scientific Sessions in Chicago, IL. Details of the poster session are as follows:
Title: Design of Heart-2: a phase 1b clinical trial of VERVE-102, an in vivo base editing medicine delivered by a GalNAc-LNP and targeting PCSK9 to durably lower LDL cholesterol
Session: Genomic Therapies for Cardiovascular Disease
Date and Time: November 16 at 10:35 AM CT
Third Quarter 2024 Financial Results
Cash Position: Verve ended the third quarter of 2024 with
Collaboration Revenue: Collaboration revenue was
Research & Development (R&D) Expenses: R&D expenses were
General & Administrative (G&A) Expenses: G&A expenses were
Net Loss: Net loss was
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 management to single-course gene editing medicines. The company’s lead programs – VERVE-101, VERVE-102, and VERVE-201 – target genes that have been extensively validated as targets for lowering low-density lipoprotein cholesterol (LDL-C), a root cause of atherosclerotic cardiovascular disease (ASCVD). VERVE-101 and VERVE-102 are designed to permanently turn off the PCSK9 gene in the liver and are being developed initially for heterozygous familial hypercholesterolemia (HeFH) and ultimately to treat patients with established 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). 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 and Pulse-1 clinical trial; the timing and availability of data for the Heart-2 trial, PCSK9 program and Pulse-1 trial; expectations for the company’s Heart-1 clinical trial; its research and development plans; the potential advantages and therapeutic potential of the company’s programs; and the period over which the company believes that its existing cash, cash equivalents and marketable securities will be sufficient to fund its operating expenses. 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
| Verve Therapeutics, Inc. | |||||||||||||
| Selected Condensed Consolidated Financial Information | |||||||||||||
| (in thousands, except share and per share amounts) | |||||||||||||
| (unaudited) | |||||||||||||
| Three months ended September 30, | Nine months ended September 30, | ||||||||||||
| Condensed consolidated statements of operations | 2024 | 2023 | 2024 | 2023 | |||||||||
| Collaboration revenue | $ | 6,865 | $ | 3,117 | $ | 19,252 | $ | 6,614 | |||||
| Operating expenses: | |||||||||||||
| Research and development | 49,938 | 43,765 | 149,299 | 138,135 | |||||||||
| General and administrative | 13,837 | 11,686 | 42,546 | 37,655 | |||||||||
| Total operating expenses | 63,775 | 55,451 | 191,845 | 175,790 | |||||||||
| Loss from operations | (56,910 | ) | (52,334 | ) | (172,593 | ) | (169,176 | ) | |||||
| Other income (expense): | |||||||||||||
| Change in fair value of success payment liability | (6 | ) | 802 | 1,743 | 878 | ||||||||
| Interest and other income, net | 6,887 | 5,841 | 22,452 | 16,825 | |||||||||
| Total other income, net | 6,881 | 6,643 | 24,195 | 17,703 | |||||||||
| Loss before provision for income taxes | (50,029 | ) | (45,691 | ) | (148,398 | ) | (151,473 | ) | |||||
| Provision for income taxes | (104 | ) | (67 | ) | (276 | ) | (243 | ) | |||||
| Net loss | $ | (50,133 | ) | $ | (45,758 | ) | $ | (148,674 | ) | $ | (151,716 | ) | |
| Net loss per common share, basic and diluted | $ | (0.59 | ) | $ | (0.72 | ) | $ | (1.77 | ) | $ | (2.43 | ) | |
| Weighted-average common shares used in net loss per share, basic and diluted | 84,632,952 | 63,211,849 | 83,999,797 | 62,322,965 | |||||||||
| Condensed consolidated balance sheet data | September 30, 2024 | December 31, 2023 | |||||
| Cash, cash equivalents and marketable securities | $ | 539,920 | $ | 623,950 | |||
| Total assets | $ | 663,906 | $ | 752,688 | |||
| Total liabilities | $ | 155,355 | $ | 153,186 | |||
| Total stockholders' equity | $ | 508,551 | $ | 599,502 | |||
FAQ
What are the latest clinical trial results for VERVE-102 as of Q3 2024?
What is Verve Therapeutics' (VERV) current cash position and runway?
What was VERV's net loss per share in Q3 2024?
