Crispr Therapeutics said Tuesday it will begin asking the Food and Drug Administration to approve what would be the first-ever gene-editing treatment to hit the market using the CRISPR technology from where it gets its company name. CRSP stock jumped.
For FDA approval, Crispr and partner Vertex Pharmaceuticals said they would begin submitting their application for the blood diseases treatment in November. They plan to finish that process in the first quarter. They are seeking approval in sickle cell disease and beta thalassemia.
The timeline is pushed back slightly, but that's unlikely to make much commercial difference, RBC Capital Markets analyst Brian Abrahams said in a report to clients. Still, he remains cautious on the ultimate uptake for the drug, currently dubbed exa-cel.
"We remain conservative on (exa-cel's) sales potential relative to (Vertex's) revenue base," he said. He sees $550 million in long-term sales potential.
But on today's stock market, the news prodded CRSP stock 4.1% higher, to 63.63. Vertex stock also rose 2.9% to 284.74.
CRSP Stock: Long-Term Data Needed
Abrahams has a sector perform rating on Vertex stock and doesn't offer a rating for CRSP stock. He notes this CRISPR gene-editing treatment requires a patient to first undergo pretreatment with a form of chemotherapy. He's waiting on gene-editing drugs that don't require this step.
Crispr and Vertex are using gene-editing to restore a patient's ability to produce healthy blood cells. In a recent data release, the companies had yearlong safety data for 23 patients and two-year data for four patients, he said. That's out of 75 patients total.
The FDA likely wants more long-term data, Abrahams said.
"We expect by the end of the first quarter, they should have roughly 48 patients at one-year and 23 patients at two-year follow-up," he said.
CRSP stock remained well below its 50-day moving average as of Monday's close, according to MarketSmith.com.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
