CRISPR Therapeutics (NASDAQ: CRSP) isn’t a stock that’s wanting for reasons to invest. Between the solid launch of its first commercialized therapy and a promising pipeline that’s continuing to deliver, this biotech is on track for growth. Plus, investors just got another big green flag to invest in its stock.
Here’s what you need to know.
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Per the company’s presentation at the American Society of Hematology’s (ASH) annual meeting on Dec. 9, the Food and Drug Administration (FDA) recently granted its cell therapy program called CTX112 a Regenerative Medicine Advanced Therapy (RMAT) designation.
In case you’re not aware, CTX112 is intended to treat certain relapsed or refractory B-cell malignancies like follicular lymphoma, and it’s currently being tested in phase 1/2 clinical trials. And when it comes to cell therapies as promising as CTX112, getting an RMAT designation from regulators is a big deal.
Here’s why. As with all of the FDA’s special designations, RMAT designations confer certain privileges and advantages for businesses that receive them.
For one, it grants the bearer all of the upsides associated with the fast-track and breakthrough-therapy designations. That means it can dramatically shorten the research and development (R&D) cycle by granting companies more seamless access to discussions with regulators as well as the right to a prompt review of any applications for commercialization or to advance into the next clinical-trial stage.
Furthermore, rather than needing 100% of the clinical data necessary for a full approval up front, as is the case for nearly all medicines, programs with an RMAT designation can potentially be preliminarily approved on the basis of partial data, with post-approval trials producing the rest of the information needed to get the final green light.
So it also offers holders significantly more financial flexibility as there’s a chance of registering sales revenue in advance of getting a full approval, which traditionally requires costly late-stage clinical trials to attain.
For CRISPR Therapeutics specifically, that financial flexibility is not necessarily needed even if it’s a plus. As of the most recent quarter, Q3, it reported having around $1.9 billion in cash, equivalents, and short-term investments, and just $402 million in trailing-12-month operating expenses.
Its first drug, a cell therapy called Casgevy, which it developed with the help of Vertex Pharmaceuticals, is in the process of rolling out. Still, there’s no world in which it’s bad news for a biotech to get a shorter pathway to the approval for its next most advanced candidate, to which it fully owns the rights.
As positive as this new development is, unfortunately CRISPR is not guaranteed to succeed with CTX112 just because it picked up the RMAT designation. The FDA won’t approve any medicine where there is not convincing data regarding safety and efficacy even if it is willing to extend more lenient terms under some conditions. Therefore, there is still a substantial risk of a late-stage clinical-trial failure if CTX112 doesn’t perform as well as it has so far.
Nonetheless, there’s an additional wrinkle that makes CTX112 particularly important for this biotech’s long-term outlook. Whereas many modern cell therapies require patients to donate a portion of their own cells to use as the raw material for manufacturing the medicine, typically involving some genetic engineering along the way, CRISPR’s process with CTX112 means that it is intended to be produced as an “off-the-shelf” intervention.
Therefore, it can be centrally manufactured and distributed to patients without needing to do the expensive and inherently small-scale manufacturing of each patient’s individualized dose. Put differently, the company’s logistics for the candidate’s cell-therapy manufacturing process are apt to be far simpler, which may equate to the drug product being cheaper.
Another advantage is that an off-the-shelf approach could make for a more reliably performing medicine. Patients who are eligible for CTX112 are already experiencing relapsed or refractory cancers, meaning that their health is in a poor state. That would leave the cells they’d donate to a cell-therapy manufacturing process in poor condition as well, which would ultimately make for a less effective therapy.
With the off-the-shelf method, there’s no variability in the health of the cells that are raw materials, so patients likely respond more consistently relative to each other, which is a big plus when it comes to getting regulators on board with granting an approval for commercialization.
Overall, the new designation is another sign that CRISPR’s stock is worth buying. If the risks associated with biotech stocks don’t bother you, it’s ready for purchase today, so long as you’re willing to hold it for a handful of years.
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Alex Carchidi has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends CRISPR Therapeutics and Vertex Pharmaceuticals. The Motley Fool has a disclosure policy.
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