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.

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.

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. 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.

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

from off-label and compassionate care use of particular MMA drugs in terminal cancer patients and through iterative testing in animal models of cancer, Penrose has arrived upon a true stand-alone, oral anti-cancer protocol with incredible societal potential to solve “the cancer problem.”

The Company is currently preparing itself for a clinical trial supervised by the Food and Drug Administration (FDA) expected to begin in the second half of 2021. Resource dependent, the Company is planning on lead indications in relapsed/refractory Stage 4 Acute Myeloid Leukemia (an orphan or rare disease) and relapsed and refractory Stage 4 Non-Small Cell Lung Cancer (the leading cause of cancer deaths worldwide). Next-generation drugs are designed and are currently being tested in cell and mouse models in Plymouth, MI, by the Penrose Science Team. This research leads to the potential for a truly universal anti-cancer platform. The Company has grown its roots in the Michigan Life Science and Innovation Center, going from a handful of people to 20+ driven team members working hard to advance their scientific efforts. In Michigan, they are surrounded by great talent due to the proximity of leading academic institutions and are proud to be a part of that community.

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