Cathie Wood is one of the most influential investors in the area of high tech, and her Ark Innovation ETF (ARKK -2.77%) has been a top performer this year. One of the stocks that Wood has been buying aggressively recently is Verve Therapeutics (VERV -4.44%), a company developing base-edited therapies for cardiovascular diseases. The company's base-editing platform is based on tech licensed from Beam Therapeutics.
Verve Therapeutics is a pioneer in the field of in vivo base editing, a technique that allows genes to be modified inside the body. The company's lead product candidate, VERVE-101, is designed to permanently lower LDL cholesterol (the "bad" cholesterol) in patients with heterozygous familial hypercholesterolemia (HeFH), a genetic disorder that causes high cholesterol levels and increases the risk of heart attacks and strokes.
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Background
VERVE-101 has shown impressive results in preclinical studies, demonstrating a 69% average reduction in LDL cholesterol in non-human primates. The therapy is presently in a phase 1 trial called the "heart-1 study." The trial is designed to evaluate ascending doses of VERVE-101 in patients with HeFH in New Zealand and the United Kingdom. Top-line results are due out in late 2023.
If successful in its clinical program, VERVE-101 could be the first gene-editing therapy approved for cardiovascular diseases, and could potentially transform the treatment landscape for millions of patients who suffer from high cholesterol and its complications.
However, the biotech is also developing another therapy, VERVE-102, for the same condition. VERVE-102 uses a more advanced delivery tech known as GalNAc-LNP, which may lead to fewer side effects and a more robust clinical response. The biotech plans on initiating a phase 1b trial for VERVE-102 in the first half of 2024.
Beyond its HeFH assets, Verve is also developing a novel base-edited therapy known as VERVE-201 for patients with homozygous familial hypercholesterolemia (HoFH). HoFH is a rare and often fatal genetic form of premature atherosclerotic cardiovascular disease characterized by extremely high levels of bad cholesterol in the blood. The biotech expects to kick off a phase 1b trial for the therapy in the second half of 2024.
Verve's base-editing approach to cardiovascular disease has also attracted some notable partners. Chief among them is biopharma heavyweight Eli Lilly (LLY -1.38%). The duo reportedly plans on treating cardiovascular disease by targeting lipoprotein(a) via base editing. Last June, Verve received an upfront payment and equity investment totaling $60 million from Lilly as part of this collaboration.
Is Verve Therapeutics worth the risk?
Wood clearly sees the potential of Verve Therapeutics, as she has been adding more shares of the stock to her flagship Ark Innovation ETF this summer. As of Aug. 30, the exchange-traded fund held over 2.25 million shares of Verve Therapeutics, worth about $29.5 million. Wood has also bought shares of Verve Therapeutics for her Ark Genomic Revolution ETF in the past, which holds roughly 2.91 shares of the base editor as of this writing. That's a ringing endorsement from one of the world's best investors.
What's the near-term investing thesis for this speculative biotech stock? VERVE-101's upcoming data readout could be a boon for the stock. Wall Street, in fact, seems to think these data could cause the stock to more than quadruple in value.
The big deal is that these data should provide insight into both the therapy's safety profile, along with a preliminary look at its efficacy in this high-value setting. Keeping with this theme, cardiovascular disease represents one of the largest therapeutic markets in existence, and there is a clear need for new treatment options -- especially ones that can truly bend the curve on this devastating condition.
Verve Therapeutics is not without risks, however. Base-editing is still an emerging and unproven field, and the company faces competition from several players in the field of cardiovascular disease at large. A clinical setback or underwhelming trial results for VERVE-101 could cause the biotech's shares to crater. So, if you're considering this name ahead of this clinical catalyst, it might be a smart move to keep any starter position on the small side for the time being.
