BioMatters Spring/Summer 2020

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CAN CER ’ S AC H IL LE’ S H E E L

One Michigan Company’s Unique Approach to “The Cancer Problem” BY JASRAI S. GILL, M.D., EXECUTIVE CLINICAL DIRECTOR, PENROSE THERAPEUTX

Cancer is a scary word. In 2020, there will be an estimated 1.9 million new cancer diagnoses in the United States, with approximately 630,000 of those patients predicted to die from the condition.

Penrose TherapeuTx, a privately-held biopharmaceutical research and development company, has developed orally-formulated small molecules with dramatic, standalone anti-cancer activity across multiple cancer subtypes, including lung, colon, breast, and leukemia. The therapy was inspired by existing small-molecule agents, never-before FDA approved in cancer, that have functioned as tools to elucidate a completely novel anti-cancer mechanism of action within the field of cancer metabolism.

That is the equivalent of losing 1,720 people to cancer every day and makes cancer the second most common cause of death in the U.S. behind only heart disease. The cost is not just in lives, in fact, the U.S. National Cancer Institute estimates that the total cost of cancer care in 2020 will be more than $173 billion. The familial, social, and economic impact of the disease are massive, and for decades people have been trying to solve “the cancer problem” with limited success. Penrose TherapeuTx is a Michigan-based company with a unique solution.

The Company’s Mitochondrial Modifying Agent (MMA) development platform targets the uniquely reprogrammed metabolism of cancer mitochondria. Malignant cancer cell survival hinges on its ability to alter its use of energy, and to protect the most critical part of its energy-producing machinery, the mitochondria. A major player in this process is Hypoxia-Inducible Factor 1-alpha (HIF-1-alpha). In reprogramming the healthy cell via HIF-1-alpha, the cancer cell creates a selective vulnerability, or an Achilles Heel, whereby treatment with Penrose’s MMA drugs leads to cancer cell death while leaving healthy cells untouched. Using data

While the oncology drug development ecosystem has realized significant progress in the fields of immuno-oncology and genome-driven targeted therapies, there remains too little hope for stage 4/metastatic cancer patients. Five-year survival rates for that class of patient are staggeringly low, in large part because cancers either don’t respond to initial therapies or because they develop resistance to the therapy that is being used.

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MICHBIO | michbio.org


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