CyNRGY™ Platform

First-in-class innovation

The CyNRGY platform is led by RRx-001, a first-in-class investigational treatment sourced from an exclusively licensed portfolio of aerospace-derived small molecules. RRx-001 is a multifaceted treatment, which was selected given its dual-functioning mechanism that starts under “normal” conditions, in other words healthy tissues that are reasonably well-oxygenated, and the activity changes in poorly oxygenated (hypoxic) tissues, which is a distinguishing characteristic of cancerous tumors.

The hypoxic triggered mechanism in cancer is similar in function to hypoxia activated prodrugs (HAPs), meaning drugs that are activated in low-oxygen conditions. In contrast, RRx-001’s 2-stage mechanism, with utility under normal conditions coupled with the hypoxia triggered activity, is what places it apart in a class of its own and supports potential benefit across many therapeutic indications.

RRx-001 initial mechanism

The “Cy” in the platform name refers to the preferential and rapid binding to cysteines that occurs with the CyNRGY portfolio of small molecules. The program’s lead compound, RRx-001, binds to cysteines found on red blood cells and macrophages, promoting antioxidant and anti-inflammatory properties through induction of the Nrf2 protein and inhibition of the inflammasome. The antioxidation activity, combined with the anti-inflammatory inflammasome inhibition, supports a protection mechanism in healthy tissues

Protection indications are currently being evaluated in clinical trials, demonstrating potential utility of RRx-001 in reducing the harmful and debilitating side effects of effective yet toxic treatments like radiotherapy and chemotherapy.

The Inflammasome

Regulating chronic inflammation

The Inflammasome is responsible for activation of inflammatory responses. With chronic inflammation, tissue destruction that occurs outpaces the regeneration of damaged tissues. Eventually, over time, the normal function of these tissues is reduced or lost, and chronic inflammation that is improperly regulated by dysfunctional inflammasomes can lead to numerous chronic inflammatory disorders including autoimmune and neurodegenerative diseases.

RRx-001 inhibition of the inflammasome represents an exciting potential in the treatment and prevention of chronic inflammation driven diseases

Preclinical studies are underway in hopes of unlocking this potential and addressing a long list of inflammation (inflammasome) regulated disorders with a persisting unmet medical need.

TONY REID, M.D. Ph.D.

Chief Executive Officer & Chief Scientific Office

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FRANCK BRINKHAUS, Ph.D.

Chief Financial Officer

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BRYAN ORONSKY, M.D. Ph.D.

Chief Medical Officer

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CHRIS LARSON,
M.D. Ph.D.
Vice President of Viral Therapy

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MEAGHAN STIRN,
M.B.A
Controller & VP of Special Projects

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SCOTT CAROEN
Senior Director of Operations & Corporate Development

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Rajan Kumar
ESQ
Chief Executive Officer & Chief Scientific Office

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Control inflammation & control the disease

RRx-001 inhibits the inflammasome

The Inflammasome is responsible for activation of inflammatory responses. With chronic inflammation tissue destruction that occurs outpaces the regeneration of damaged tissues. Eventually, over time, the normal function of these tissues is reduced or lost. In chronically inflamed tissues, which may result from unresolved sources of foreign bodies, irritants or infections, the inflammasome fuels an inflammatory response for weeks to months or even years, resulting in a range of metabolic, neurological, autoimmune disorders as well as in the initiation of cancer.

The Answer: AdAPT-001 programs infected cancer cells to produce a TGFβ “trap” molecule that is designed to neutralize TGFβ within the infected tumor. Eliminating the t-cell silencing TGFβ protein allows for the immune system to remain activated against cancer, making AdAPT-001 unlike any other immunotherapy.

AdAPT-001 doesn’t just create a cancer targeted infection, it produces a proprietary TGFβ “trap” protein to eliminate this tumor defense mechanism and allow for sustained immune response against cancer – alone or in combination with immune checkpoint inhibitors.

RRx-001 in Oncology

In “abnormal” conditions, such as with cancer, RRx-001 generates an alternate mechanism that has shown in preclinical and clinical studies to normalize blood flow in tumors, allowing oxygenation of tumor tissue and modifying the tumor immune environment with reduction in CD47 expression and reprogramming of tumor associated macrophages.

In effect, RRx-001 is normalizing the environment of the abnormal environment of tumors, allowing functional blood flow and oxygenation.

Cancer prefers to control its environment, creating an impenetrable defense supported by growing irregular and poorly functioning vessels that inhibit blood flow. This restricts delivery of therapies and surveillance from the body’s immune system. Therefore, normalizing the environment of tumors makes them susceptible to treatment that may otherwise fail because it was previously unable to penetrate cancerous tissues.