BOSTON - Verve Therapeutics, a biotech firm working on cardiovascular gene editing treatments, has paused its Heart-1 clinical trial for VERVE-101 due to observed side effects. The decision comes after a participant developed asymptomatic but significant lab abnormalities. The company now plans to concentrate on advancing VERVE-102, which utilizes a different lipid nanoparticle delivery system.
The Heart-1 Phase 1b trial was assessing VERVE-101's safety and its impact on LDL cholesterol (LDL-C) levels in patients with specific cholesterol disorders. Among the six participants given a 0.45 mg/kg dose, one experienced transient liver enzyme elevation and thrombocytopenia, which resolved quickly without symptoms. Following this event, Verve, in consultation with the independent data and safety monitoring board, opted to halt further enrollment and investigate the abnormalities.
Despite the setback, earlier trial results showed promising LDL-C reductions ranging from 21% to 73% at the 0.45 mg/kg dose level. The treatment's LDL-C lowering effect appeared durable over 270 days in two patients.
Verve's next steps involve a focus on VERVE-102, which shares the same base editor and guide RNA targeting the PCSK9 gene as VERVE-101 but differs in its delivery system. The ionizable lipid used in VERVE-102 has been well-tolerated in other clinical trials. Regulatory clearances from the U.K. MHRA and Health Canada have been obtained, with the Heart-2 clinical trial set to begin in the second quarter of 2024.
VERVE-101 and VERVE-102 are designed as single-course treatments to permanently turn off the PCSK9 gene in the liver, thereby reducing LDL-C. VERVE-101 is being developed for heterozygous familial hypercholesterolemia (HeFH), while VERVE-102 will also target premature coronary artery disease (CAD).
Verve reassures that patient safety remains paramount as they work with regulatory authorities to determine the future of VERVE-101. Meanwhile, the company's financial position suggests a cash runway extending into late 2026.
This article is based on a press release statement from Verve Therapeutics.
InvestingPro Insights
In light of Verve Therapeutics' recent clinical trial pause, investors are closely monitoring the company's financial health and market performance. According to InvestingPro data, Verve Therapeutics currently holds a market capitalization of $1.07 billion. Despite the clinical challenges, one of the InvestingPro Tips highlights that Verve maintains a stronger liquidity position, holding more cash than debt on its balance sheet, which could provide resilience in navigating the clinical development landscape.
Another critical aspect for investors is the company's stock performance. Verve's stock has experienced notable volatility, with a 1-month price total return showing a decline of 21.49%. This volatility is further underscored by an InvestingPro Tip pointing out the stock's poor performance over the last month. Additionally, the company's revenue growth is impressive, with a surge of 505.77% over the last twelve months as of Q4 2023, indicating significant market interest in its gene editing technologies despite current setbacks.
For investors seeking a more in-depth analysis, InvestingPro offers additional insights, with a total of 9 InvestingPro Tips available for Verve Therapeutics. These tips provide a comprehensive view of the company's financial metrics and market potential. Interested readers can explore these tips and enhance their investment strategy by using the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription.
As Verve Therapeutics continues to navigate the challenges of drug development, these InvestingPro Insights offer valuable context for understanding the company's position in the market and its potential for long-term growth.
For more detailed analysis and tips on Verve Therapeutics, visit https://www.investing.com/pro/VERV.
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