Last year’s bear market dragged down many stocks. And CRISPR Therapeutics (CRSP -1.24%) wasn’t an exception. The biotech company was down by 46%. But what makes this loss surprising is the fact that CRISPR is closer than ever to a major milestone — one that could highlight the strength of its science and result in significant revenue.
I’m talking about the potential approval of CRISPR’s very first product. CRISPR and partner Vertex Pharmaceuticals have applied for authorization of exa-cel, a candidate to treat blood disorders. If all goes well, Wall Street’s average forecast for a 70% jump in CRISPR shares within the coming 12 months could happen.
Let’s take a closer look at what we may expect from this exciting stock.
Fixing faulty genes
CRISPR specializes in gene editing. This involves correcting faulty genes that are involved in a disease process. CRISPR and Vertex use this technology in exa-cel to tackle the blood disorders beta thalassemia and sickle cell disease.
Today, treatment options for these illnesses are limited. And patients face hospitalizations and blood transfusions throughout their lifetime.
The companies’ exa-cel could change all of that because exa-cel is designed as a one-time curative treatment for the two illnesses in adults. This clearly could attract patients and doctors. And it may eventually result in lower costs for payers, as it should lead to fewer hospitalizations for blood disorder patients over the years.
CRISPR and Vertex have completed submissions of exa-cel to regulators in the U.K. and Europe. And the companies aim to complete their U.S. submission at the end of the first quarter of this year. In the U.S., decisions usually take between six and 10 months. So CRISPR is getting very close to transitioning into the world of commercial-stage companies.
Initially, the companies aim for approval in the adult population. But they’re also studying exa-cel in phase 3 trials for children. This could result in a wider audience in the not-too-distant future.
Reasons to be optimistic
If regulators offer CRISPR and Vertex a nod for exa-cel in adults, CRISPR shares could indeed take off as Wall Street predicts — and there’s plenty of room for additional gains over time. Here’s why I’m so optimistic about this decision pushing the stock higher.
As I’ve hinted, an approval is a vote of confidence for CRISPR’s technology. This doesn’t mean it will work for every candidate CRISPR has in the pipeline. But it would suggest this technology has the ability to treat — and even cure — some illnesses. That could easily draw more investors to CRISPR stock.
I also think the promise of product revenue — and the growth likely to follow — may push the stock higher. Yes, Vertex does hold rights to the biggest share of revenue at 60%. But CRISPR’s 40% still is significant, especially since we’re talking about the company’s first product — and a product with blockbuster potential.
Finally, today CRISPR shares are trading at a lower level than they were a while back, when we had less visibility on the safety and efficacy of exa-cel.
At the same time, CRISPR also has advanced other candidates in the pipeline. For example, oncology candidate CTX-110 has generated positive trial data — and CRISPR recently launched a phase 2 trial that may even support a regulatory submission. This could result in a second potential product in the not-too-distant future.
CRISPR shares look pretty reasonable right now, considering the strength of its technology as we’ve seen in clinical trials — and the fact that it’s getting very close to the finish line with two candidates. And that’s why I wouldn’t be surprised if this innovative biotech stock meets or beats Wall Street’s share price forecasts in the coming year.
Adria Cimino has positions in Vertex Pharmaceuticals. The Motley Fool has positions in and recommends CRISPR Therapeutics and Vertex Pharmaceuticals. The Motley Fool has a disclosure policy.