Deciphera Announces First Patient Treated in Phase 1 Study of DCC-3116 in Patients with Advanced or Metastatic Tumors with a Mutant RAS or RAF Gene

WALTHAM, Mass.--(BUSINESS WIRE)-- Deciphera Pharmaceuticals LLC, (NASDAQ: DCPH), a commercial-stage biopharmaceutical company developing innovative medicines to improve the lives of people with cancer, today announced dose administration for the first patient in the Phase 1, multicenter, open-label, first-in-human study of DCC-3116. DCC-3116 is an investigational ULK kinase inhibitor designed to inhibit autophagy and is being studied as a single agent and in combination with trametinib, a U.S. Food and Drug Administration (FDA) approved MEK inhibitor, in patients with advanced or metastatic tumors with a mutant RAS or RAF gene.

“Approximately one third of all cancers, including a high percentage of pancreatic, lung, colorectal, and melanoma cancers, are driven by mutations in RAS or RAF genes, representing what we believe to be one of the largest unmet medical needs in oncology,” said Matthew L. Sherman, MD, Executive Vice President and Chief Medical Officer of Deciphera Pharmaceuticals. “DCC-3116, a first-in-class, highly selective switch-control ULK kinase inhibitor, is designed to suppress autophagy and may offer a novel approach to targeting a broad array of cancers. We look forward to advancing our fourth active clinical development program generated from our switch-control kinase inhibitor platform and further evaluating the role of ULK kinase inhibition and its potential to represent a new treatment paradigm for cancers caused by RAS or RAF mutations.”

Autophagy, a catabolic process in which cells recycle components to generate energy, is often upregulated in cancer cells when cells are stressed or damaged due to anti-cancer treatments. The ULK kinase initiates the autophagy pathway and provides a potential targeted approach to selectively inhibiting autophagy in cancers caused by RAS or RAF mutations. In preclinical studies, DCC-3116 was observed to potently and durably inhibit autophagy in RAS and RAF mutant cancer cell lines through the inhibition of ULK kinase. In addition, in preclinical studies, DCC-3116 also blocked the increase in autophagy induced by inhibitors of the MAPK pathway as a resistance mechanism. The Company’s in vitro and in vivo studies have demonstrated that DCC-3116 in combination with inhibitors of the MAPK pathway may block the growth of cancers caused by RAS or RAF mutations.

The clinical development plan for DCC-3116 will focus on documented RAS and RAF cancer mutations, which utilize autophagy for tumor growth and survival. The Phase 1, multicenter, open-label, first-in-human study will evaluate DCC-3116 as a single agent and in combination with trametinib, an FDA-approved MEK inhibitor, in patients with advanced or metastatic tumors with a mutant RAS or RAF gene. Assuming positive results in the dose escalation phase, combination expansion cohorts are currently planned in patients with advanced or metastatic pancreatic ductal adenocarcinoma (PDAC) with KRAS or BRAF mutations, non-small cell lung cancer (NSCLC) with KRAS, NRAS, or BRAF mutations, colorectal cancer (CRC) with KRAS, NRAS, or BRAF mutations, and melanoma with NRAS or BRAF mutations. Combination expansion cohorts are planned to evaluate DCC-3116 in combination with trametinib.

About Deciphera Pharmaceuticals

Deciphera is a biopharmaceutical company focused on discovering, developing and commercializing important new medicines to improve the lives of people with cancer. We are leveraging our proprietary switch-control kinase inhibitor platform and deep expertise in kinase biology to develop a broad portfolio of innovative medicines. In addition to advancing multiple product candidates from our platform in clinical studies, QINLOCK^® is Deciphera’s FDA-approved switch-control kinase inhibitor for the treatment of fourth-line gastrointestinal stromal tumor (GIST). QINLOCK is also approved for fourth-line GIST in Australia, Canada, China, and Hong Kong. For more information, visit www.deciphera.com and follow us on LinkedIn and Twitter (@Deciphera).

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, our expectations regarding our belief that DCC-3116 may offer a novel approach to targeting a broad array of cancers, ULK kinase inhibition’s potential to represent a new treatment paradigm for cancers caused by RAS or RAF cancers and a potential targeted approach to selectively inhibiting autophagy in cancers caused by RAS or RAF mutations, the DCC-3116/MAPK pathway inhibitor combination’s potential to block the growth of cancers caused by RAS or RAF mutations and our planned expansion cohorts. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “seek,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks and uncertainties related to the severity and duration of the impact of COVID-19 on our business and operations, our ability to successfully demonstrate the efficacy and safety of our drug candidates and in additional indications for our existing drug, the preclinical or clinical results for our product candidates, which may not support further development of such product candidates, our ability to manage our reliance on sole-source third parties such as our third party drug substance and drug product contract manufacturers, comments, feedback and actions of regulatory agencies, our ability to commercialize QINLOCK and execute on our marketing plans for any drugs or indications that may be approved in the future, our ability to build and scale our operations to support growth in additional geographies, the inherent uncertainty in estimates of patient populations, competition from other products, our ability to obtain and maintain reimbursement for any approved product and the extent to which patient assistance programs are utilized, our ability to comply with healthcare regulations and laws, our ability to obtain, maintain and enforce our intellectual property rights, any or all of which may affect the initiation, timing and progress of clinical studies and the timing of and our ability to obtain additional regulatory approvals, and other risks identified in our Securities and Exchange Commission (SEC) filings, including our Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, and subsequent filings with the SEC. We caution you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. We disclaim any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent our views only as of the date hereof and should not be relied upon as representing our views as of any subsequent date. We explicitly disclaim any obligation to update any forward-looking statements.

QINLOCK and the QINLOCK logo are registered trademarks, and Deciphera and the Deciphera logo are trademarks, of Deciphera Pharmaceuticals, LLC.

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