Merck's acquisition of Modifi Biosciences will give it access to preclinical cancer drug candidates that target DNA repair processes.

Modifi Biosciences, a spin out of Yale University founded in 2021, has been snapped up by Merck for $30 million paid upfront, and up to $1.3 billion in potential milestone payments. Modifi focuses on the development of small molecule drugs that target cancer cells with impaired DNA repair.

"In founding Modifi Biosciences, we sought to radically change the oncology treatment paradigm for cancer patients with glioblastoma and other tumors," said Ranjit Bindra, co-founder of Modifi Biosciences, professor of therapeutic radiology at Yale School of Medicine, and Scientific Director of the Yale Brain Tumor Center. "We are honored to have Merck recognize the potential of our science, and as an oncology company, they are perfectly positioned to advance our innovations through clinical trials and commercialization."

Modifi Biosciences has developed a new class of small molecule drugs that specifically target cancer cells that lack the expression of O6-methylguanine methyltransferase (MGMT), a protein that plays a key role in DNA repair. Mutations that affect DNA repair are common in cancer cells that can be exploited against them to treat drug-resistant forms of cancer.

"DNA repair defects are a frequent hallmark of tumor cells and a major cause of resistance to cancer therapy," said Dr. David Weinstock, vice president, discovery oncology, Merck Research Laboratories. "The talented Modifi Biosciences team has developed an innovative approach that we believe has potential for treating some of the most refractory cancer types."

Research published by Modifi's scientific founders in 2022 showed that this approach had promising results in mouse models of drug-resistant glioma, a type of cancer that affects the brain and spinal cord. Since then, the company has been conducting preclinical trials in models of glioma and glioblastoma; It is estimated that up to 80% of gliomas and approximately half of all glioblastomas lack the MGMT protein, making these types of cancer the ideal first target for Modifi's new drug class.

"We designed our small molecules to have the ability to uniquely overcome clinical resistance mechanisms that have been known for decades but until now have been non-actionable. Additionally, we created the molecules in a manner which allows them to be rapidly progressed from bench-to-bedside," said Seth Herzon, co-founder of Modifi Biosciences and Professor of Chemistry at Yale.