CRISPR Therapeutics Has 2 Key Catalysts Coming by the End of 2026. Is the Market Underestimating Them?

Proving a technology works and proving it can win a share of a crowded market are two different things. CRISPR Therapeutics (NASDAQ: CRSP) will soon be put to the test on both.

The biotech expects to report data for two clinical trial readouts in the coming months. For the stock to do well, the market will need to be impressed by the data, not merely have its (high) expectations confirmed. Here's what to expect.

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This RNA drug is aiming at a crowded market

The first catalyst on deck for CRISPR Therapeutics stems from its CTX611 program.

CTX611 is a small interfering RNA (siRNA). It prompts a patient's body to make less of a protein called Factor XI (FXI), which is responsible for helping to form blood clots. Many medicines for that purpose exist, but the candidate's edge is that it might only need to be given two times per year, while most blood thinners on the market right now are formulated as daily pills.

An update from its Phase 2 trial is expected before the end of 2026. That study compares three dose levels of CTX611 against enoxaparin, a standard injectable blood thinner, in patients having knee replacement surgery. The precedent to know here is that in a 2021 trial of another FXI-blocking candidate called abelacimab, clots appeared in 4% of knee surgery patients on the top dose, compared to 22% of those treated with enoxaparin.

So CRISPR's trial is already calibrated to have good odds of one-upping the standard of care, and it won't be the only candidate in development to do so if it manages to clear that bar.

Therefore, to wow the market and create a strong boost in the company's share price from the readout catalyst, CTX611 will likely need to perform better than both the comparator from its trial (enoxaparin) and other programs in development by rival biopharmas with different modalities.

The second catalyst is subject to the same dynamic

The second catalyst for CRISPR is another data update due before the end of the year, this time for CTX310, an infusion that edits the genomes of liver cells to disrupt the gene for ANGPTL3.

That gene codes for a protein that reduces the rate at which fat-carrying particles are cleared from the patient's blood. Disabling it aims to decrease both the patient's level of triglycerides and their level of low-density lipoprotein (LDL) cholesterol -- both of which would be highly lucrative.

In a Phase 1a trial of CTX310, one year after treatment, patients who received the maximum tested dose saw their triglycerides fall by an average of 48%, and their LDL by 53%. The data update coming out is centered around people with severe hypertriglyceridemia (sHTG), whose very high triglycerides raise the risk of acute pancreatitis.

But CRISPR might have the same issue with CTX310 as it does with CTX611.

Arrowhead Pharmaceuticals reported that its drug plozasiran, which is given four times per year and is already approved for a rare, inherited form of the disease, reduced patients' median triglycerides by about 80% in two Phase 3 trials. It plans to apply for U.S. Food and Drug Administration (FDA) approval in sHTG before the end of 2026.

Different trials with different treatment modalities can't be compared rigorously. Still, a gene editing therapy like CTX310 can't be undone once a patient has been dosed, and the bar for it to clear is a drug that doctors can simply stop prescribing at any time if necessary. That's before even getting into the stark differences in efficacy or potential side effects.

So, is the market underestimating these catalysts? Probably not by much.

If CRISPR ends up publishing some stunning and positive data that there was no hint of in its prior releases, the stock could still go on a tear, but don't hold your breath. The competing candidates appear to be strong, and they might be viewed as less risky for patients too.

Alex Carchidi has no position in any of the stocks mentioned. The Motley Fool recommends CRISPR Therapeutics. The Motley Fool has a disclosure policy.