2021-04-21| R&DTechnology

Vertex to Reap Majority Profits from Gene Editing Deal with CRISPR Therapeutics

by Ruchi Jhonsa
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Vertex and CRISPR Therapeutics have been jointly working on a CRISPR-based therapy, CTX001, for treating beta-thalassemia and sickle cell disease for several years now.

On April 20th, the companies said that they amended their agreement according to which Vertex’s monetary contribution to the drug development will increase, but so will its profits once the drug gets regulatory approval.


New Agreement Puts Vertex at the Forefront

According to the new agreement, Vertex will pay 60% of all developmental costs associated with the drug but will also reap 60% profits from future sales of the drug. This figure is 10% higher than the originally decided one. CRISPR, on the other hand, will pay only 40% of the costs and would receive 40% of the profits. The amended agreement also hands CRISPR a $900 million upfront payment, with the potential for $200 million more upon regulatory approval.

Under the original terms of the deal, CRISPR would have led to the commercialization of any treatment that would have emerged from the partnership. Now, Vertex will perform that role by leveraging its “established global capabilities and proven experience in manufacturing, development, regulatory, and commercialization.”

“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,” said Jeffrey Leiden, M.D., Ph.D., Executive Chairman of Vertex.

“We see a significant commercial opportunity for CTX001, and we believe we will be able to further enhance that opportunity by fully leveraging the breadth of Vertex’s capabilities – including our established and proven R&D and commercialization expertise in serious diseases – to bring CTX001 to more patients around the world, more quickly,” he added.


CTX001 Shows Promise

This amendment comes after the stellar data from the early-stage trial of the CRISPR drug CTX001 rose Vertex’s interest. The breakthrough featured in GeneOnline’s list of top ten headlines of 2020.

The data presented at ASH2020 last December from the two ongoing studies showed that seven patients with beta-thalassemia no longer needed blood transfusions for months after receiving the drug.

Three sickle cell patients treated with the drug also went months without experiencing a vaso-occlusive crisis, a common disease symptom. This is attributed to the drug’s effect on hemoglobin levels, which restored to normal to near-normal levels in these patients. Based on the progress, the drug has been granted Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the USFDA.

“Working with Vertex, we have made tremendous progress with CTX001, the first CRISPR/Cas9-based therapy to demonstrate proof of concept in the clinic, and together we have broken new ground in the treatment of genetic diseases. We have now dosed more than 30 patients with CTX001, with longest follow-up beyond two years, and we are on track to complete enrollment in both clinical trials this year,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics.

The duo will seek approval from the FDA once they have treated enough patients in the two ongoing studies. Vertex believes that with all the support they are getting from the FDA in the form of fast track and orphan drug designation, they can receive early approval. This may sound surprising as typically, new drugs require testing in hundreds or thousands of patients across several phases of the study. However, with gene editing or gene therapy treatments, the companies can make a convincing case to regulators with far fewer patients if the drug effect is striking.

CRISPR Therapeutics is one of the many firms that featured in GeneOnline’s list of top ten gene therapy companies of this year.

Related Article: The Transformative Potential of Gene Therapies for Hemophilia

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