About Checkmate Pharmaceuticals

Checkmate Pharmaceuticals, Inc., a clinical-stage biotechnology company, focuses on developing and commercializing its proprietary technology to harness the power of the immune system to combat cancer. The company’s product candidate, vidutolimod, is an advanced generation Toll-like receptor 9 (TLR9) agonist delivered as a non-infectious biologic virus-like particle (VLP) utilizing a cytosine-phosphate-guanine (CpG)-A oligonucleotide as a key component. When injected into a tumor, vidutolimod is designed to trigger the body’s innate immune system, thereby altering the tumor microenvironment and directing activated anti-tumor T cells to attack both the injected tumor and also tumors throughout the body. The company’s focus is to establish vidutolimod as a foundational immuno-oncology therapy that engages the innate immune system to fight cancer and improve outcomes for patients with a broad range of solid tumors. The company’s major is to advance the clinical development of vidutolimod toward marketing authorizations for the treatment of melanoma; and develop vidutolimod for use across other relevant tumor types and indications. It is conducting a Phase 2 trial of vidutolimod in combination with nivolumab, or OPDIVO, in anti- programmed death receptor 1 (PD-1) refractory melanoma. The company is also conducting a randomized Phase 2/3 trial of vidutolimod in combination with nivolumab in first-line melanoma. In addition to these studies in melanoma, it is evaluating new indications. including conducting a Phase 2 proof of concept trial of vidutolimod in combination with pembrolizumab, or KEYTRUDA, in recurrent/metastatic head and neck squamous cell carcinoma (HNSCC), and a separate, multi-indication study evaluating vidutolimod in combination with cemiplimab in non-melanoma skin cancers cutaneous squamous cell carcinoma (CSCC) and Merkel cell carcinoma (MCC). The company’s completed Phase 1b trial evaluated vidutolimod in subjects with advanced anti-PD-1 refractory melanoma in combination with pembrolizumab and as a monotherapy. The company also supported an investigator-sponsored Phase 2 trial with vidutolimod in combination with nivolumab in the pre-surgery, or neoadjuvant, setting in patients with resectable Stage III melanoma not previously treated with PD-1 blockade. The U.S. Food and Drug Administration (FDA) has granted vidutolimod a Toll-like receptor 9 (TLR9) agonist Orphan Drug Designation for the treatment of Stages IIb-IV melanoma and Fast Track designation for unresectable Stage III or Stage IV melanoma and unresectable or metastatic melanoma refractory to prior anti-PD-1 blockade. As of December 31, 2021, vidutolimod had been studied in more than 250 melanoma patients in clinical trials. On the strength of the clinical results observed in multiple settings of melanoma, the company is conducting additional trials with vidutolimod in combination with anti-PD-1 antibodies. Sales and Marketing The company has retained worldwide commercial rights for its product candidates. If its product candidates receive marketing approval, the company plans to commercialize them in the United States with its own focused, specialty sales force. The company also plans to evaluate options for delivering vidutolimod, if approved, to patients in other key markets, such as Europe, Japan, and China, which may include strategic collaborations. Collaboration with BMS In December 2020, the company entered into a clinical collaboration with Bristol-Myers Squibb Company (BMS), pursuant to which BMS agreed to provide nivolumab for its Phase 2 refractory melanoma study and Phase 2 first-line melanoma study, at its own expense. Intellectual Property For the core technology related to vidutolimodand its component parts, the company is prosecuting four families of patent applications, which it owns, including the composition of matter, the methods of use, combination therapies, drug delivery, dose volume, aggregation, packaging and biomarkers. Further, the company has an exclusive license for seven families of patents and patent applications, including the composition of matter, manufacturing methods, aggregation, packaging and synthesis claims. Some of its patents have issued in the United States and internationally and others are pending in the United States and internationally (either in foreign jurisdictions or under the Patent Cooperation Treaty (PCT)). As of March 6, 2022, the company owned or exclusively licensed the rights to 13 issued U.S. granted patents, 131 granted patents outside of the United States, and 29 pending patent applications worldwide. The company or its licensors have filed patent applications in 33 foreign jurisdictions, including the EU, Canada, Japan, China, Australia, Italy, the Netherlands, India, France and Germany. The patents include claims directed to combination therapy of TLR9 agonists and checkpoint inhibitors, volumes and methods for administration, biomarkers, aggregation and packaging and the methods of treatment claims. The company estimates, without taking potential patent term extensions into account, that these issued patents will expire between 2036 and 2038. License Agreement with Kuros In 2015, the company entered into an exclusive license agreement with Cytos Biotechnology LTD (now Kuros Biosciences AG (Kuros)) as amended in August 2017 and as further amended in January 2018 (the ‘Kuros License Agreement’). Pursuant to the Kuros License Agreement, the company was granted an exclusive, royalty-bearing, sublicensable, worldwide license, under all of Kuros’ intellectual property rights, including any intellectual property rights arising during the term of the agreement, to commercially develop, manufacture, use, distribute, and sell certain therapeutic products, including vidutolimod, or the Licensed Products, for the diagnosis, treatment and prevention of all indications in humans and animals. Under the terms of the Kuros License Agreement, the company is required to use commercially reasonable efforts to develop approximately one Licensed Product. The Kuros License Agreement will expire upon the expiration of the last-to-expire royalty term for the Licensed Products in the territory. Either party has the right to terminate the agreement in full for an uncured material breach of the agreement upon written 60 days’ notice to the other party. The company has the right to terminate the agreement for any reason upon 90 days’ written notice to Kuros. Clinical Trial Collaboration and Supply Agreements In May 2021, the company entered into a Supply and Non-Exclusive License Agreement (SNLA) with Regeneron Pharmaceuticals, Inc. (Regeneron). The SNLA dictates the general terms that govern specific collaborative studies between the company and Regeneron, including a Phase 2 proof of concept trial, with patient cohorts in anti-PD-1 naïve and anti-PD-1 refractory CSCC and anti-PD-1 refractory MCC. Pursuant to the SNLA, Regeneron agreed to provide cemiplimab, a drug to be used concurrently or in combination with vidutolimod in the aforementioned studies, at its own expense. As part of the SNLA, the parties granted each other non-exclusive licenses to use background intellectual property and regulatory documentation to seek regulatory approval of the other party’s compound solely for use as a combination therapy. In December 2020, the company entered into the Master Clinical Trial Collaboration Agreement (MCTCA) with BMS. The MCTCA dictates the general terms that govern specific collaborative studies between the companies, including its Phase 2 refractory melanoma study and Phase 2 first-line melanoma study (collectively, the ‘collaborative studies’). Pursuant to the MCTCA, BMS agreed to provide nivolumab, a drug to be used in combination with vidutolimod, in the collaborative studies, at its own expense. As part of the MCTCA, the parties granted each other non-exclusive licenses to use background intellectual property and regulatory documentation to seek regulatory approval of the other party’s compound solely for use as a combination therapy. Research and Development The company’s research and development expenses were $45.8 million in 2021. Government Regulation The FDA and other regulatory authorities at federal, state and local levels, as well as in foreign countries, extensively regulate, among other things, the research, development, testing, manufacture, quality control, import, export, safety, effectiveness, labeling, packaging, storage, distribution, record keeping, approval, advertising, promotion, marketing, post-approval monitoring and post-approval reporting of biologics, such as those the company is developing. In the United States, where the company is initially focusing its product development, the FDA regulates biologics under the Federal Food, Drug and Cosmetic Act and the Public Health Service Act and their implementing regulations. The Affordable Care Act includes provisions of importance to the company’s potential product candidate that created an annual, nondeductible fee on any entity that manufactures or imports specified branded prescription drugs and biologic products, apportioned among these entities according to their market share in certain government healthcare programs; expanded eligibility criteria for Medicaid programs by, among other things, allowing states to offer Medicaid coverage to certain individuals with income at or below 133% of the federal poverty level, thereby potentially increasing a manufacturer’s Medicaid rebate liability; expanded manufacturers’ rebate liability under the Medicaid Drug Rebate Program by increasing the minimum rebate for both branded and generic drugs and revising the definition of ‘average manufacturer price’, for calculating and reporting Medicaid drug rebates on outpatient prescription drug prices; addressed a new methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected; expanded the types of entities eligible for the 340B drug discount program; established the Medicare Part D coverage gap discount program by requiring manufacturers to provide point-of-sale-discounts off the negotiated price of applicable brand drugs to eligible beneficiaries during their coverage gap period as a condition for the manufacturers’ outpatient drugs to be covered under Medicare Part D; and created a Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research. Competition The company’s competitors include biopharmaceutical, biotechnology and therapeutics companies, specifically companies focused on cancer immunotherapies, such as Amgen, Inc.; AstraZeneca plc; BMS; Genentech, Inc.; GlaxoSmithKline PLC; Merck & Co., Inc.; Novartis AG; Pfizer Inc.; Regeneron; Roche Holding Ltd; and Sanofi S.A. Additionally, the company views companies that are developing innate immune activators as its direct potential competitors. These include approaches to activate Toll-like receptors, such as TriSalus Life Sciences, Idera Pharmaceuticals, Bolt Therapeutics, and Silverback; cytokine therapies, such as a plasmid IL-12 from Oncosec; and companies developing oncolytic viruses, such as Replimune. History Checkmate Pharmaceuticals, Inc. was founded in 2015. The company was incorporated in 2015 under the laws of the state of Delaware.