Deals, Gene Editing

Vertex sharpens focus on gene-editing in licensing deal with Merck KGaA

Two years ago Merck KGaA splurged $230 million to license all the rights to four oncology programs, including a DNA-dependent protein kinase (DNA-PK) inhibitor, from cystic fibrosis drugmaker Vertex. On Thursday, the German drugmaker allowed Vertex access to the compound via a licensing deal for use in the field of gene editing.

No financial details were provided for the new transaction, which also enables Vertex to access to an undisclosed preclinical compound. The DNA-PK inhibitor — M9831 (formerly known as VX-984) — and the preclinical drug were licensed to Merck from Vertex in January 2017 and are now part of the Darmstadt-based company’s arsenal of DNA Damage Response (DDR) inhibitors.

Damage to the DNA in cells occurs every day and consequently the DDR monitors impact, initiates repair and halts cell growth during the rebuilding process. But in patients with cancer there is a high frequency of DDR defects which culminate in the accumulation of DNA damage, giving rise to mutations that promote unbridled cancer cell growth and/or enable normal cells to evade apoptosis. Although cancer cells may benefit from DDR defects, other functioning DNA repair systems are required for survival. DDR inhibitors thus offer an opportunity to fight cancer by selectively killing cancer cells via the suppression of standby DDR mechanisms and sparing normal healthy cells that are not dependent on these pathways.

Merck’s portfolio of DDR inhibitors includes two ATR inhibitors, an ATM inhibitor and an investigational small-molecule of DNA-PK, which is a key enzyme that could potentially enhance the efficacy of DNA-damaging agents such as radiotherapy and chemotherapy. Preclinical data has shown DNA-PK inhibition can also augment CRISPR/Cas9-mediated gene editing, the company said.

With this deal, Vertex is sharpening its focus into gene editing. The Boston biotech, which ousted its COO and interim CFO Ian Smith for improper personal conduct on Wednesday, is already collaborating with CRISPR Therapeutics to evaluate the latter’s CRISPR gene editing technology to potentially fix the mutations in the CFTR gene understood to result in the defective protein that causes cystic fibrosis, as well as other genetic disorders. Their experimental treatment, CTX001, was granted fast track status by the FDA earlier this month for sickle cell disease.

Under the new Merck/Vertex deal, Merck will receive an upfront payment and is eligible to receive milestone payments and  royalties from potential drug sales, and Vertex has the option to add indications to the license grant. Overall, Merck retains the rights to both assets in all other disease areas, including oncology, and can develop both these compounds in-house, or license them to other companies for use in the gene editing field.


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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
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Catalytic Data Science Charleston, SC
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Flatiron Health New York City or San Francisco

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