VERV - Verve Therapeutics Announces Pipeline Progress and Reports First Quarter 2024 Financial Results | Benzinga
First patient dosed in Heart-2 Phase 1b clinical trial of VERVE-102
VERVE-201 clinical trial initiation on track for the second half of 2024
Received first milestone payment from Eli Lilly for collaboration on an in vivo gene editing program targeting lipoprotein(a) (Lp(a))
Cash, cash equivalents and marketable securities of $606.4 million; cash runway into late 2026
BOSTON, May 08, 2024 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today reported pipeline updates and financial results for the quarter ended March 31, 2024.
"Our accomplishments in the first quarter bring us closer to realizing our mission of protecting patients from cardiovascular disease through single-course gene editing medicines. We are excited to investigate VERVE-102 in the Heart-2 clinical trial, having recently announced the first patient dosing in the trial," said Sekar Kathiresan, M.D., co-founder and chief executive officer of Verve Therapeutics. "We also remain on track to initiate the Phase 1b clinical trial for VERVE-201 targeting ANGPTL3 in the second half of the year and are pleased with the progress we're making on our Lp(a) collaboration with Eli Lilly. Following these recent achievements and program updates, we are focused on continued execution across our pipeline as we develop our clinical and preclinical programs addressing a robust set of validated targets in areas of high unmet need."
First Patient Dosed with VERVE-102 in Heart-2 Clinical Trial
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 to reduce disease-driving low-density lipoprotein cholesterol (LDL-C). VERVE-102 is being developed initially as a treatment for patients with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD). VERVE-102 consists of messenger RNA expressing an adenine base editor and an optimized guide RNA targeting the PCSK9 gene, identical to VERVE-101. However, VERVE-102 uses a different delivery system than VERVE-101, which includes a different ionizable lipid and Verve's proprietary GalNAc liver-targeting ligand, which allows the lipid nanoparticle (LNP) to access liver cells using either the asialoglycoprotein receptor (ASGPR) or the low-density lipoprotein receptor (LDLR).
Verve recently dosed its first patient in the Heart-2 Phase 1b clinical trial. Heart-2 is an open-label Phase 1b clinical trial designed to evaluate the safety and tolerability of VERVE-102 in adult patients with HeFH or premature CAD who require additional lowering of LDL-C, with additional analyses for pharmacokinetics and changes in blood PCSK9 protein and LDL-C levels. The company received clearances of its Clinical Trial Applications (CTAs) in Canada and the U.K. Verve expects to provide a data update on the PCSK9 program in 2025.
Ongoing Analysis of Heart-1 Clinical Trial of VERVE-101
Enrollment remains paused in the Heart-1 trial as Verve continues to investigate the observed laboratory abnormalities which Verve believes are attributable to the LNP delivery system. As the company continues to work with regulatory authorities to define a potential path forward, the VERVE-101 Investigational New Drug Application (IND) in the U.S. and CTAs in the U.K. and New Zealand remain active.
VERVE-201 on Track for Clinical Trial Initiation in Second Half of 2024
VERVE-201, an in vivo base editing medicine delivered as a one-time intravenous infusion, is designed to inactivate the ANGPTL3 gene in liver cells, turning off liver production of blood ANGPTL3 protein and thereby durably reducing blood LDL-C and triglyceride-rich lipoproteins. For VERVE-201, Verve is utilizing its proprietary GalNAc-LNP delivery technology. VERVE-201 is being developed for the treatment of patients living with homozygous familial hypercholesterolemia (HoFH), a rare and often fatal inherited subtype of premature accelerated atherosclerotic cardiovascular disease (ASCVD) characterized by extremely high blood LDL-C. VERVE-201 aims to reduce the heavy treatment burden associated with available therapies for HoFH, including the requirement for multiple oral, injectable, and intravenous infusions, often administered over decades. VERVE-201 is also being developed for ASCVD patients with refractory hypercholesterolemia, who have high LDL-C despite treatment with maximally-tolerated standard of care therapies.
Verve has completed preclinical studies to support regulatory submissions for clinical development. Verve expects to initiate the VERVE-201 Phase 1b clinical trial in the second half of 2024, subject to regulatory clearances.
First Milestone Achieved in Global Collaboration with Eli Lilly
Verve achieved its first research and development milestone related to its exclusive research collaboration with Eli Lilly focused on advancing Verve's research stage in vivo gene editing program targeting LPA. Elevated Lp(a) is an established and genetically validated independent risk factor for ASCVD, ischemic stroke, and aortic stenosis.
Under the terms of the collaboration established in June 2023, Verve will advance the research and development of the Lp(a) program through the completion of Phase 1 clinical development. Lilly will be responsible for subsequent development, manufacturing, and commercialization of the Lp(a) program. Verve is eligible to receive up to $465 million in research, development, and commercial milestones, as well as tiered royalties on global net sales. Following the completion of Phase 1 clinical trials, Verve has the right to opt-in to co-fund and share margins globally on the Lp(a) program (in lieu of receipt of milestones and royalties).
Upcoming Medical Meeting Presentations
American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting: Verve will present an overview of off-target analyses for VERVE-101.
Presentation title: Characterization of Guide RNA Site Consistency Across Ancestries and the Potential for Off-Target Editing with the Clinical-Stage Base Editing Medicine, VERVE-101
Track: Base Editing and Prime Editing II
Date/time: Saturday, May 11, 2024, 10:30 – 10:45 a.m. ET
Location: Ballroom 3, Baltimore Convention Center, Baltimore, MD
TIDES 2024: Verve will present previously disclosed nonclinical and clinical data from the company's PCSK9 program.
Presentation title: Proof-of-concept for in vivo Base Editing to Inactivate the PCSK9 Gene and Lower LDL-Cholesterol in Humans
Track: Genome Editing Technology and Applications
Date/time: Friday, May 17, 2024, 11:15 – 11:45 a.m. ET
Location: Hynes Convention Center, Boston, MA
Upcoming Investor Event
Verve plans to participate in a fireside chat at the RBC Global Healthcare Conference on Wednesday, May 15, 2024, at 8:00 a.m. ET in New York.
A live webcast will be available in the investor section of the company's website at www.vervetx.com. The webcast will be archived for 30 days following the presentation.
First Quarter 2024 Financial Results
Cash Position: Verve ended the first quarter of 2024 with $606.4 million in cash, cash equivalents, and marketable securities. Verve continues to expect its capital position to be sufficient to fund its operations into late 2026.
Collaboration Revenue: Collaboration revenue was $5.7 million for the first quarter of 2024, compared to $1.4 million for the first quarter of 2023. The increase was primarily due to an increase in research services performed under the company's collaboration agreements and cost reimbursements.
Research & Development (R&D) Expenses: R&D expenses were $48.4 million for the first quarter of 2024, compared to $47.1 million for the first quarter of 2023. Stock-based compensation expense included in R&D expenses was $5.6 million and $4.5 million for the first quarter of 2024 and 2023, respectively.
General & Administrative (G&A) Expenses: G&A expenses were $14.2 million for the first quarter of 2024, compared to $12.6 million for the first quarter of 2023. Stock-based compensation expense included in G&A expenses was $4.7 million and $3.5 million for the first quarter of 2024 and 2023, respectively.
Net Loss: Net loss ...