CRISPR Therapeutics’ ( CRSP Quick Quote CRSP - Free Report) lead pipeline candidate is exagamglogene autotemcel (exa-cel, formerly CTX001), an investigational ex-vivo CRISPR gene-edited therapy. The candidate is being developed for treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) under a co-development and co-commercialization agreement with Vertex Pharmaceuticals ( VRTX Quick Quote VRTX - Free Report) .
Exa-cel is currently being evaluated in two separate phase III studies for treating TDT and severe SCD. Both the late-stage studies are now fully enrolled. The regulatory submission for the therapy in both indications is anticipated in Europe by 2022-end. Discussion regarding the U.S. submission package for exa-cel is ongoing with the FDA.
SCD and TDT have a significant unmet medical need, and if successfully developed and commercialized, exa-cel can provide a huge boost to CRISPR Therapeutics’ prospects. CRSP, along with Vertex Pharmaceuticals, initiated two new phase III studies of exa-cel in pediatric patients with SCD and TDT.
Both CRSIPR Therapeutics and Vertex Pharmaceuticals are also developing therapies to address cystic fibrosis and Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1). CRSP’s top line primarily comprises collaboration revenues and milestone payments received from VRTX.
Shares of CRSP have declined 15.9% so far this year compared with the
industry’s 24.0% decline. Image Source: Zacks Investment Research
Apart from exa-cel, CRISPR Therapeutics is developing other candidates in its pipeline. CRSP is currently evaluating three chimeric antigen receptor T cell (CAR-T) therapy candidates, namely CTX110, CTX120 and CTX130, which target hematological and solid-tumor cancer indications.
CRISPR Therapeuticsis currently evaluating CTX110 in a pivotal phase I CARBON study to treat relapsed/refractory B-cell malignancies. An ongoing phase I study is evaluating the safety and efficacy of several dose levels of CTX120 for treating relapsed or refractory multiple myeloma. Two independent phase I studies are also evaluating the safety and efficacy of several dose levels of CTX130 for treating solid tumors such as renal cell carcinoma (COBALT-RCC study) and certain T-cell and B-cell hematologic malignancies (COBALT-LYM study).
In June 2022, management announced preliminary results from the COBALT-LYM study which showed that treatment with CTX130 can produce broad activity and deep responses in patients with relapsed/refractory T-cell lymphomas, especially at higher dose levels. Preliminary data from the COBALT-RCC showed signs of meaningful activity in solid tumors.
CRISPR Therapeutics has also advanced its first program incorporating stem-cell therapy for the treatment of diabetes in collaboration with ViaCyte (now acquired by Vertex Pharmaceuticals). The first patient is already dosed in a phase I study to evaluate the safety, tolerability and immune evasion of VCTX210 for type I diabetes (T1D).
With no approved products in its commercial portfolio, CRISPR Therapeutics lacks a source of a stable stream of income. Failure in the ongoing studies will hurt the stock significantly. While the company’s lead candidate is still a couple of years away from commercialization, other pipeline candidates are several years away from the commercialization stage. Moreover, CRSP’s dependence on Vertex Pharmaceuticals for collaboration revenues remains a concern, as termination of their mutual contract in the near future would adversely impact the stock’s prospects.
Zacks Rank & Key Picks
CRISPR Therapeutics currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the same sector are
Morphic ( MORF Quick Quote MORF - Free Report) and Sesen Bio ( SESN Quick Quote SESN - Free Report) , each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here .
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