In the wake of the news that the US Food and Drug Administration is placing a clinical hold on CRISPR Therapeutics's new drug application, CTX001 which treats sickle cell disease, shares in the gene-editing company fell sharply.
Investors have been thrown off by the FDA's news, sending the group's shares down 8.7% in afternoon trade to US$67.17.
But Leah Rush Cann, an analyst with Oppenheimer, is still keeping her Perform rating on the stock. “We are surprised by this clinical hold by the FDA,” she wrote in a note. “This clinical hold does not impact our outlook for timing or for collaboration revenue.”
The FDA’s clinical hold on CTX001 will remain in place until certain questions about this drug therapy are cleared up.
A new drug application was first submitted by CRISPR and its partner Vertex Pharmaceuticals to the FDA last April to support the start of a Phase1/2 trial in the US in adults with sickle cell disease.
CTX001 is a stem-cell therapy for patients suffering from B-thalassemia and sickle cell disease. The start of a Phase1/2 trial of CTX001 is still expected to go ahead in Europe by the second half of this year.
CRISPR and Vertex hope to glean additional information about the FDA’s questions in the near future and work rapidly to resolve them in a bid to advance towards the drug’s approval.
Rush Cann expects that CRISPR will make most of its money from its current collaboration agreements and projects that the company’s total revenue will reach US$357.3mln by 2022.
“We do not anticipate CRISPR Therapeutics will have a commercial product prior to 2022 and therefore estimate that collaborative agreement payments will continue to be the primary contributor to revenue through 2022,” she concluded.