Vertex has boosted an agreement with CRISPR Therapeutics to $900 million upfront as the companies race to beat bluebird bio to the market with a new gene editing therapy for sickle cell disease and beta thalassemia called CTX001.
Boston-based Vertex will pay CRISPR $900 million upfront with a potential for $200 million in milestone payments if the therapy is approved. Vertex will be responsible for 60% of the program costs and receive the same in profits from future worldwide sales of CTX001. The new agreement is a 10% increase from the former deal.
CTX001 is under development as a potential cure for the blood diseases sickle cell disease and transfusion-dependent beta thalassemia.
Sickle cell disease is an inherited disorder that causes red blood cells to turn into a sickle shape, which die and cause a shortage. They can also clog blood flow in the body, causing infection, stroke or other problems. The disease is most often found in Black or African Americans. The only curative treatment is a blood or bone marrow transplant, but medicines or transfusions can manage complications when caught early.
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Beta thalassemia is another inherited blood disorder that causes the body to make less hemoglobin than normal, which can lead to mild or severe anemia. Patients are typically treated with frequent blood transfusions.
Vertex and CRISPR are currently in a race against bluebird bio to bring to market a new treatment option for patients with the two conditions. While bluebird bio is ahead in its regulatory plans for Zynteglo—with a European approval already in the bag—the therapy has stumbled in sickle cell disease after safety concerns were raised in a clinical trial. Today, bluebird also announced that German cost regulators couldn't find a price for its gene therapy that bluebird would agree to, so it has been pulled in that market.
It also announced it was slashing staffers on the continent as it struggles to find a commerical path forward. Vertex is clearly bullish that it can make it work, and with a gene editing approach, rather than a gene therapy method, but the coincidental timing of these unrelated releases shows the risks inherent in the space.
“As we take the lead on CTX001, we want to acknowledge the foundational contributions by the team at CRISPR Therapeutics,” said Vertex Executive Chairman Jeffrey Leiden, M.D., Ph.D., in a statement. “Our increased investment in our partnership with CRISPR is based on the compelling clinical profile of CTX001, which shows its potential to be a durable cure for patients with SCD and TDT, and the rapid progress that we and our partners at CRISPR have made toward registration and commercialization.”
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The two companies originally signed a research agreement in 2015 to develop therapies using CRISPR/Cas9 to address genetic diseases. CTX001 is the first therapy that has emerged from the partnership, which was originally worth $75 million in cash paid to CRISPR and $30 million in the company’s equity for Vertex. The therapy has been granted a number of designations from the FDA and European regulators for sickle cell disease and beta thalassemia, including Regenerative Medicine Advanced Therapy, fast-track and orphan-drug tags.
Vertex is perhaps best known for its collection of approved cystic fibrosis medicines. While the newest of those therapies, Trikafta, was just approved in 2019, Vertex may be eyeing the patent cliff on the other three as it goes all in on the CRISPR partnership.