Health and Healthcare

CRISPR Faces FDA Clinical Hold

Chris Lange

Published: May 31, 2018 8:35 am

Last Updated: January 11, 2020 10:58 pm

CRISPR Therapeutics A.G. (NASDAQ: CRSP) shares saw a significant drop on Thursday morning after an announcement from the U.S. Food and Drug Administration (FDA). Specifically, the FDA placed a clinical hold on the Investigational New Drug Application (IND) for CTX001 for the treatment of sickle cell disease, pending the resolution of certain questions that will be provided by the FDA as part of its review of the IND.

The IND was submitted to the FDA in April to support the planned initiation of a Phase 1/2 trial in the United States in adult patients with sickle cell disease. CRISPR and Vertex Pharmaceuticals Inc. (NYSE: VRTX) expect to obtain additional information on the FDA’s questions in the near future and plan to work rapidly with the FDA toward a resolution.

For some background: CTX001 is an investigational, gene-edited autologous hematopoietic stem cell therapy for patients suffering from beta-thalassemia and sickle cell disease.

The planned initiation of a Phase 1/2 trial of CTX001 in Europe in adult patients with transfusion-dependent beta-thalassemia is unchanged, and the companies expect to initiate the trial in the second half of 2018.

Excluding Thursday’s move, CRISPR has outperformed the broad markets, with its stock up about 416% in the past 52 weeks. In just 2018 alone, the stock is up 213%.

Shares of CRISPR closed Wednesday at $73.59, with a consensus analyst price target of $69.31 and a 52-week range of $13.54 to $73.90. Following the announcement, the stock was down about 16% at $62.00 in early trading indications Thursday.

Shares of Vertex closed Wednesday at $156.64, with a consensus analyst price target of $191.39 and a 52-week range of $120.77 to $178.15.