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On Monday, Verve Therapeutics (NASDAQ: VERV), a $366 million market cap biotech company whose shares have surged nearly 39% in the past week, showcased promising data from its Phase Ib Heart-2 trial, which focuses on the company’s leading drug candidate, VERVE-102, for treating patients with heterozygous familial hypercholesterolemia (HeFH) or coronary artery disease (CAD). William Blair analyst Raju Prasad maintained an Outperform rating on the stock, reflecting optimism about the trial’s outcome. According to InvestingPro data, the stock currently trades near its Fair Value, with analysts setting price targets ranging from $15 to $39.
The trial analyzed VERVE-102’s efficacy in 14 participants across three dosing cohorts, with results surpassing the LDL-C reduction goals previously set by competing treatments. According to the data available as of March 13, 2025, VERVE-102 not only achieved but exceeded the targeted 40%-50% reduction in LDL-C levels, positioning it favorably against Leqvio (inclisiran) outcomes from the ORION studies. InvestingPro analysis shows the company maintains a strong liquidity position with a current ratio of 12.65, though it’s currently burning through cash with negative EBITDA of $222 million in the last twelve months.
The safety profile of VERVE-102 was also highlighted, with no serious adverse events (SAEs), dose-limiting toxicities, or cardiovascular events reported. The treatment’s tolerability was further evidenced by the absence of significant changes in liver enzymes, bilirubin, or platelets at any dose level. Only one grade 2 infusion-related reaction occurred, which was quickly resolved.
These findings represent a significant improvement over the company’s first-generation PCSK9 base editor, VERVE-101, which had previously raised safety concerns due to severe liver enzyme elevations and thrombocytopenia. The modifications made to VERVE-102’s lipid nanoparticle (LNP) components have effectively addressed these issues, suggesting a more favorable safety profile for the drug.
Verve Therapeutics is actively engaging with the investment community, offering a conference call to discuss the detailed findings of the Heart-2 trial. Interested parties can access call details through the company’s investor relations website. Further analysis and insights from the trial data will be provided following the conference call.
In other recent news, Verve Therapeutics has reported promising results from its Heart-2 Phase 1b clinical trial for VERVE-102, a gene-editing therapy aimed at reducing LDL cholesterol levels. The trial demonstrated substantial LDL-C reductions, with an average decrease of 53% and a peak reduction of 69% in the highest dose cohort, while maintaining a favorable safety profile. These findings have led Canaccord Genuity to increase their probability of success for the VERVE-102 program from 50% to 70% and raise the stock target to $39. Cantor Fitzgerald also upgraded Verve Therapeutics from Neutral to Overweight, citing the absence of serious adverse events and potential market advantage as a one-time treatment. Jefferies maintained a Buy rating with a $28 price target, highlighting the positive safety and pharmacodynamic data supporting VERVE-102’s potential efficacy.
Goldman Sachs noted the efficacy of VERVE-102 in reducing LDL-C levels and its promising safety profile, suggesting a positive outlook for the upcoming Phase 2 trials. Verve Therapeutics has a strong financial position, with $524.3 million in cash and equivalents, expected to fund operations into mid-2027. The company is preparing to present an option package to Eli Lilly (NYSE:LLY), with a decision anticipated in the second half of 2025. These developments underscore Verve Therapeutics’ continued progress in the biotechnology sector and its potential impact on cardiovascular disease treatment.
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