Apros Therapeutics Announces IND Clearance from U.S. FDA to Initiate the Trial of APR003, an Orally-Administered Gastrointestinal/Liver-Targeted TLR7 Agonist for Treatment of Advanced Colorectal Cancer (CRC) with Malignant Liver Lesions

Apros Therapeutics, Inc., a drug discovery and development company focused on tissue-targeted Toll-Like Receptor 7 (TLR7) agonists for the treatment of cancer and infectious diseases, announced that its Investigational New Drug (IND) application for a Phase 1 dose escalation trial of APR003, the company’s first-in-class orally-administered gastrointestinal- and liver-targeted TLR7 agonist development candidate, was cleared by the FDA. The Phase 1 trial aims to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and initial anti-tumor activity of APR003 in patients with advanced unresectable CRC with malignant liver lesions. The trial will be conducted at multiple sites in the United States, and the company intends to report top-line results upon completion.

“We are excited to enter the clinic with our lead drug APR003 that was designed utilizing our tissue-targeted chemistry to deliver a TLR7 agonist in abundance to liver and GI tissue with little-to-no systemic distribution,” stated Dr. Aaron Weitzman, M.D., FACP, Acting Chief Medical Officer at Apros Therapeutics. “We are encouraged by APR003’s unique profile and significant single agent efficacy at well-tolerated doses observed in our preclinical studies, and we are pleased to advance this program into the clinic as we believe it will offer an important step forward in creating a new immunotherapeutic treatment option for advanced colorectal cancer patients, a disease with a large unmet medical need.”

ABOUT APR003

APR003 is a first-in-class, orally-administered, TLR7 agonist designed specifically to localize to the GI and liver to promote local immune activation in the targeted tissues and drive tumor eradication. Given the immune-activating capacity of this class of drugs, the tissue-targeted approach taken with APR003 is predicted to exhibit an improved tolerability profile compared to other oral non-targeted approaches, which possess side effects associated with systemic immune activation and inflammation. TLR7 activation in the liver of patients with malignancies that are metastatic to the liver may lead to innate immune priming, release of cytokines and, ultimately, enhanced anti-tumor immune responses. Although APR003 may have applications in several GI and liver malignancies, Apros will focus the initial evaluation of APR003 in patients with unresectable CRC that is metastatic to the liver given the unique bio-distribution and mechanism of action of this first-in-human investigational agent. CRC is the 3^rd most common cancer in the world, and 50% of patients with advanced disease will develop liver metastasis. While most patients with CRC are non-responsive to immunotherapies, largely due to the immune-quiescent nature of the disease, APR003 aims to turn these “cold” tumors into “hot” immune-permissive tumors.

ABOUT APROS THERAPEUTICS

Founded in 2016, and based in San Diego, CA, Apros Therapeutics is focused on the discovery and development of tissue-targeted TLR7 agonists for the immunotherapy of cancer and infectious diseases. Apros Therapeutics has employed a chemistry-based platform to develop a portfolio of small molecule TLR7 agonists using a variety of proprietary approaches with the aim of restricting drug distribution, controlling half-life and dose in specific tissues. Each development candidate is engineered to exert precise spatiotemporal bio-distribution to target a tumor/tissue type, in order to uncouple efficacy from systemic toxicity. This platform ultimately aims to widen the therapeutic window and extend the application of innate immune agonists to treat a broad range of cancer and infectious disease indications. Apros Therapeutics is currently advancing multiple candidates into clinical development. For more information, please visit www.aprostx.com.

Forward-Looking Statements –

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding Apros’s plans for developing APR003 for the treatment of CRC and the potential benefits of APR003 for CRC. Risks that contribute to the uncertain nature of the forward-looking statements include uncertainties related to the impact of certain translational research, completion of clinical trials and whether the results from clinical trials will validate and support the safety and efficacy of APR003 for CRC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Apros undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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