About Cidara Therapeutics

Cidara Therapeutics, Inc. operates as a biotechnology company. The company focuses on the discovery, development and commercialization of long-acting therapeutics designed to transform the standard of care for patients facing serious diseases. The company focuses on infectious diseases and oncology. The company’s lead product candidate is rezafungin (trade name REZZAYO), an intravenous formulation of a novel echinocandin antifungal. Rezafungin is being developed as a once-weekly, high-exposure therapy for the treatment and prevention of serious, invasive fungal infections. The company’s primary focus is using its Cloudbreak platform to develop a potential new class of drugs called drug-Fc conjugates, or DFCs, for the prevention and treatment of serious diseases. This technology couples potent inhibitors to a human antibody fragment to create long-acting DFCs designed to inhibit multiple disease targets. The company’s most advanced DFC program is CD388, a highly potent, long-acting antiviral designed to deliver universal prevention and treatment of seasonal and pandemic influenza, which is in Phase 1 and Phase 2a clinical trials. Additional programs are targeting the SARS-CoV-2 strains causing COVID-19 and multiple solid tumor oncology indications. In January 2023, the company nominated CBO-212 as its first oncology DFC candidate. With its Cloudbreak oncology program the company seeks to develop a new generation of immunotherapies, and its lead oncology DFC candidate, CD421 (which is an enhanced version of CBO-212), is a first-in-class CD73 inhibitor that combines the strengths of small molecules and monoclonal antibodies targeting CD73. The company is advancing CD421 that confers reduced immunogenic properties through investigational new drug application, or IND, -enabling studies. In February 2023, the company expanded its existing collaboration with WuXi XDC, a leading global contract manufacturing organization, or CMO, dedicated to end-to-end bioconjugates services, under which WuXi XDC will provide investigational new drug application -enabling chemistry and manufacturing and controls, or CMC, development services for its Cloudbreak oncology program. On January 24, 2023, the U.S. Food and Drug Administration, or FDA, Antimicrobial Drugs Advisory Committee voted favorably 14 to 1 that the company, as part of its New Drug Application, or NDA, provided sufficient evidence supporting a favorable benefit-risk assessment for a limited use indication for rezafungin for the treatment of candidemia and invasive candidiasis in adult patients with limited or no alternative treatment options. On March 22, 2023, the U.S. Food and Drug Administration, approved REZZAYO (rezafungin for injection) for the treatment of candidemia and invasive candidiasis in adults with limited or no alternative treatment options. REZZAYO is the first new treatment option approved for patients with candidemia and invasive candidiasis in over a decade, and is the only available once-weekly echinocandin. On February 9, 2023, the company received formal notice from The Nasdaq Stock Market, LLC, or Nasdaq, Hearings Panel, or the Panel, stating that it has regained compliance with the minimum bid price requirement set forth in Nasdaq Listing Rule 5550(a)(2), subject to a discretionary Panel Monitor until November 9, 2023. On March 1, 2023, the company announced efficacy and safety data from a planned interim analysis of its ongoing Phase 2a trial evaluating the pre-exposure prophylactic activity of CD388 against the H3N2 influenza A virus strain, as of a February 13, 2023 data cut-off. The Phase 2a trial is a single-center, randomized, double-blind influenza challenge study in healthy volunteers designed to assess the prophylactic antiviral activity, safety, tolerability and pharmacokinetics of CD388 in healthy volunteers. The interim analysis is based on 56 subjects enrolled in the trial, with 28 subjects receiving a single dose of CD388 (150 mg) and 28 subjects receiving a placebo. The company’s Cloudbreak platform has the potential to offer a fundamentally new approach to prevent and treat serious diseases, by developing product candidates designed to provide potent disease targeting activity and immune system engagement in a single long-acting molecule. Because serious disease often results when a pathogen or cancer cell evades or overcomes the host immune system, the company’s Cloudbreak DFC candidates are designed to counter diseases in two ways: prevention of disease proliferation or immune evasion by directly targeting and, where applicable, by focusing the immune system on a pathogen or infected cell. The company’s lead Cloudbreak candidates are CD388, a DFC for the prevention and treatment of influenza, or influenza DFC, and CD421, a CD73-targeting DFC for the treatment of solid tumors. In September 2020, the company nominated CD388, its influenza DFC, as a development candidate. The company submitted an investigational new drug application for CD388 in December 2021 and initiated a Phase 1 trial (NCT05285137) in March 2022. The Phase 1 trial is a randomized, double-blind, dose-escalation study to determine the safety, tolerability and pharmacokinetics of intramuscular and subcutaneous administration of CD388 in healthy subjects. In September 2022, the company initiated a Phase 2a trial (NCT05523089) to evaluate the pre-exposure prophylactic activity of CD388 against influenza virus. The Phase 2a trial, which dosed its first healthy volunteer in September 2022, is a single-center, randomized, double-blind, placebo-controlled, proof-of-concept study to assess the prophylactic antiviral activity, safety, tolerability and pharmacokinetics of CD388 against influenza via a human viral challenge (influenza) model. Multiple dose levels of CD388 will be evaluated in volunteers who will receive a single administration of CD388 or placebo prior to influenza viral challenge. In December 2022, the company received the first U.S. patent for CD388. The patent includes claims directed to the composition of matter of CD388. The patent is projected to expire in 2039 plus any available patent term extension. The Cloudbreak platform has also enabled the company to expand the development of DFCs to target other life-threatening viral diseases, including the SARS-CoV-2 strains causing COVID-19. In addition, the company has expanded the Cloudbreak platform beyond infectious diseases, to discover and develop highly potent DFCs that can target multiple immune checkpoint pathways with a single DFC for oncologic diseases. The company’s lead oncology development candidate, CD421, targets CD73 in the adenosine pathway, which contributes to immune evasion in solid cancers by flooding the tumor microenvironment with adenosine, a potent immune cell suppressor. CD73 is highly expressed on a variety of tumor and stromal cells, as well as immunosuppressive cell populations, such as regulatory T cells and myeloid-derived suppressor cells. CD421 is designed to address the potency, efficacy, pharmacokinetic and safety limitations of small molecule and monoclonal antibody candidates targeting CD73. On March 31, 2021, the company entered into the exclusive, worldwide license and collaboration agreement, or the Janssen Collaboration Agreement, with Janssen Pharmaceuticals, Inc., or Janssen, one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to develop and commercialize one or more DFCs based on its Cloudbreak platform for the prevention and treatment of influenza. Under the terms of the Janssen Collaboration Agreement, the company is collaborating in the research, preclinical and early clinical development of CD388, under a mutually-agreed research plan with the objective of advancing development through Phase 1 clinical trials and the first Phase 2a clinical trial. The company is responsible for performing all investigational new drug application -enabling nonclinical studies and early-stage clinical trials under the research plan. Both parties are responsible for conducting certain specified chemistry, manufacturing and controls development activities under the research plan. Rezafungin is a novel molecule in the echinocandin class of antifungals. The company is developing rezafungin for the treatment and prevention of serious, invasive fungal infections which are associated with high mortality rates. In December 2021, the company reported positive topline results from ReSTORE, its Phase 3 pivotal clinical trial in patients with candidemia and/or invasive candidiasis (NCT03667690). ReSTORE was a global, randomized, double-blind, controlled trial evaluating the efficacy and safety of rezafungin as a potential first-line treatment for candidemia and invasive candidiasis. ReSTORE enrolled 187 patients and evaluated one 400 milligram, or mg, loading dose of rezafungin for the first week followed by 200 mg of rezafungin dosed once-weekly for up to four weeks in total. The treatment arm was compared to approved daily dosing of caspofungin in a 1:1 randomization. The company is conducting the ReSPECT, single, global, randomized, double-blind, controlled Phase 3 pivotal clinical trial (NCT04368559) in patients undergoing allogeneic blood and marrow transplant to assess rezafungin in a 90-day prophylaxis regimen to prevent infections due to Candida, Aspergillus and Pneumocystis. Rezafungin, dosed at 400 mg for the first week followed by 200 mg once weekly out to 90 days, is being compared to a regimen containing two drugs (an azole and Bactrim) dosed once daily for 90 days. The primary efficacy outcome for this trial for the U.S. Food and Drug Administration, and EMA is fungal-free survival at Day 90. While the ReSPECT trial remains open for enrollment, the company continues to monitor the near- and long-term impact of COVID-19 on the ability of its clinical investigators to recruit patients at each of its global clinical trial sites. The study is enrolling in the European Union, or EU, Canada and the U.S. On July 26, 2022, the company entered into a License Agreement, or the Melinta License Agreement, with Melinta Therapeutics, LLC, or Melinta, under which it granted Melinta an exclusive license to develop and commercialize products that contain or incorporate rezafungin in the U.S. Melinta will be solely responsible for the commercialization of rezafungin in the U.S., at its sole expense. The company is responsible for conducting an agreed upon development plan that includes, among other activities, completion of the ongoing ReSPECT Phase 3 pivotal clinical trial for the prevention of invasive fungal infections in adult allogeneic blood and marrow transplant recipients. On September 3, 2019, the company announced a strategic partnership with Mundipharma Medical Company (Mundipharma) to develop and commercialize rezafungin in an intravenous formulation for the treatment and prevention of invasive fungal infections. Under the terms of the Collaboration and License Agreement, or the Mundipharma Collaboration Agreement, with Mundipharma Medical Company, or Mundipharma, the company granted Mundipharma an exclusive, royalty-bearing license to develop, register and commercialize rezafungin outside the U.S. and Japan. The company acquired rezafungin, a novel echinocandin antifungal agent, in 2014. Rezafungin is being developed for both the treatment and prevention of serious, invasive fungal infections. The company has sought multiple designations from regulators, including the U.S. Food and Drug Administration, and the EMA, to support the rapid evaluation of rezafungin in the development phase and to enhance the commercial attributes of rezafungin in the commercial phase. The company, together with its partners Mundipharma and Melinta, plan to seek orphan drug designation for rezafungin for prophylactic use in the U.S. and Europe. The Cloudbreak DFC platform has the potential to offer a fundamentally new approach to prevent and treat serious diseases. The company’s product candidates called DFCs are designed to provide both potent disease targeting activity and immune system engagement in a single molecule. Because serious disease often results when a pathogen or cancer cell evades or overcomes the host immune system, the company’s Cloudbreak DFC candidates are designed to counter diseases in two ways: prevention of disease proliferation or immune evasion by directly targeting and, where applicable, by focusing the immune system on a pathogen or infected cell. The Cloudbreak platform has enabled the company to expand the development of DFCs to target other life-threatening viral diseases, including the SARS-CoV-2 strains causing COVID-19. In addition, the company has expanded the Cloudbreak platform beyond infectious diseases to discover and develop highly potent DFCs that can target multiple immune checkpoint pathways with a single DFC for oncologic diseases. The company is leveraging the Cloudbreak platform to address multiple diseases. Each DFC targets a life-threatening disease, focused on infectious diseases and oncology. The company’s DFC research and development programs include Influenza; Cancer (solid tumors); and COVID-19. The company has developed a novel DFC that provides a direct and sustained antiviral effect and retains potent activity in immune compromised hosts. The results of multiple preclinical studies with CD388 indicate that it is effective in both the treatment and prevention of influenza infections. CD388 has been engineered to extend half-life and has the potential to extend the duration of protection in prophylactic applications. The company evaluated CD388 in vitro for its ability to inhibit viral replication in cell-based assays versus a large panel of seasonal and pandemic Influenza A strains, including 2009 H1N1 pandemic strain, H3N2 and H5N1, H7N9, oseltamivir (Tamiflu)-resistant H1N1, and influenza B strains, including zanamivir (Relenza)-resistant influenza B. CD388 showed potent activity against all the strains tested, including influenza B, which is less sensitive to oseltamivir phosphate. The company evaluated CD388 in vivo in lethal mouse models against multiple H1N1, H3N2 and influenza B strains. CD388 provided full protection against all strains tested H1N1 with single, low doses. The company tested mean plasma concentrations of CD388 in mice and based on the long half-life it observed, it evaluated CD388 in vivo in a lethal mouse model of H1N1 by administering CD388 to the mice 7 days before lethal influenza challenge. With its Cloudbreak oncology program the company seeks to develop a new generation of immunotherapies, and its CD421 DFC is a first-in-class CD73 inhibitor that combines the strengths of small molecules and monoclonal antibodies targeting CD73. The company is advancing CD421 through investigational new drug application -enabling studies. CD421 targets CD73 in the adenosine pathway, which contributes to immune evasion in solid cancers by flooding the microenvironment surrounding tumors with adenosine, a potent immune cell suppressor. CD73 is highly expressed on a variety of tumor and stromal cells, as well as immunosuppressive cell populations, such as regulatory T cells and myeloid-derived suppressor cells. CD421 is designed to address the potency, efficacy, pharmacokinetic and safety limitations of small molecule and monoclonal antibody candidates targeting CD73 in clinical development. CD421 has several attributes that potentially differentiate it from the most advanced small molecule and mAb inhibitors in clinical development: License and Collaboration Agreements Mundipharma Collaboration Agreement In September 2019, the company entered into the Mundipharma Collaboration Agreement with Mundipharma for a strategic collaboration to develop and commercialize rezafungin in an intravenous formulation, or the Mundipharma Licensed Product, for the treatment and prevention of invasive fungal infections. Collaboration: Under the Mundipharma Collaboration Agreement, the company is responsible for leading the conduct of an agreed global development plan, or the Global Development Plan, that includes its ongoing ReSTORE Trial of the Mundipharma Licensed Product, and its ongoing ReSPECT Trial of the Mundipharma Licensed Product, as well as specified GLP-compliant non-clinical studies and CMC development activities for the Mundipharma Licensed Product. Mundipharma is responsible for performing all development activities, other than Global Development Plan activities, that may be necessary to obtain and maintain regulatory approvals for the Mundipharma Licensed Product outside of the U.S. and Japan, or the Mundipharma Territory, at Mundipharma’s sole cost. Licenses: Pursuant to the Mundipharma Collaboration Agreement, the company granted Mundipharma an exclusive, royalty-bearing license to develop, register and commercialize the Mundipharma Licensed Product in the Mundipharma Territory. The company also granted Mundipharma an option to obtain exclusive licenses to develop, register and commercialize rezafungin in a formulation for subcutaneous administration, or Subcutaneous Product, and in formulations for other modes of administration, or Other Products, in the Mundipharma Territory, subject to similar rights retained by the company to conduct mutually agreed global development activities for such products. In addition, the company granted Mundipharma a co-exclusive, worldwide license to manufacture the Mundipharma Licensed Product and rezafungin. Until the seventh anniversary of the first commercial sale of the Mundipharma Licensed Product in the Mundipharma Territory, each party has granted the other party an exclusive, time-limited right of first negotiation to obtain a license to any anti-fungal product (other than Mundipharma Licensed Product, Subcutaneous Product and Other Products) that such party proposes to out-license in the other party’s territory. Retained Rights: The company retains the exclusive right to develop, register and commercialize the Mundipharma Licensed Product, Subcutaneous Product and Other Products in Japan, or the company Territory, and Mundipharma has granted it certain licenses under Mundipharma-controlled technology and jointly-developed technology to develop, register and commercialize Mundipharma Licensed Product, Subcutaneous Product and Other Products in the company Territory and to manufacture such products and rezafungin worldwide. Janssen Collaboration Agreement On March 31, 2021, the company entered into the Janssen Collaboration Agreement with Janssen to develop and commercialize one or more DFCs based on its Cloudbreak platform, for the prevention and treatment of influenza, including CD388 and CD377, or the Products. The effectiveness of the Janssen Collaboration Agreement, including the effectiveness of the terms and conditions described below, was subject to the expiration or earlier termination of all applicable waiting periods under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, or HSR. HSR clearance was obtained on May 12, 2021 and the Janssen Collaboration Agreement became effective on the same date. Licenses: Upon the effectiveness of the Janssen Collaboration Agreement, the company granted Janssen an exclusive, worldwide, royalty-bearing license to develop, register and commercialize Products, subject to its retained right to conduct Research Plan activities. In addition, the company granted Janssen an exclusive right of first negotiation until December 31, 2021, to negotiate and enter into a separate definitive agreement pursuant to which the parties would collaborate in the research and development of DFCs for the treatment or prevention of respiratory syncytial virus. This right of first negotiation expired on December 31, 2021. Melinta License Agreement On July 26, 2022, the company entered into the Melinta License Agreement with Melinta Therapeutics, LLC (Melinta) under which it granted Melinta an exclusive license to develop and commercialize products that contain or incorporate rezafungin, or the Melinta Licensed Product, in the U.S., or the Melinta Territory. Licenses: Pursuant to the Melinta License Agreement, the company granted Melinta an exclusive, royalty-bearing license (including the right to sublicense through multiple tiers), to develop, register and commercialize the Melinta Licensed Product for all uses in humans and non-human animals in the Melinta Territory, subject to its retained right, as described below. Commercialization: Melinta will be solely responsible for the commercialization of rezafungin in the Melinta Territory, at its sole expense. Retained Rights: The company retains the non-exclusive right to practice the intellectual property rights licensed to Melinta in the Melinta Territory solely for the purpose of performing its obligations under the Melinta License Agreement and Mundipharma Collaboration Agreement. The company also retains the right to grant licenses under the intellectual property rights licensed to Melinta to third parties to which it has granted licenses or rights to market, promote and sell Melinta Licensed Product outside the Melinta Territory, to make and have made Melinta Licensed Product anywhere in the world solely to develop, register, use, sell, have sold, offer for sale, commercialize and import Melinta Licensed Product outside the Melinta Territory, subject to the terms of the Melinta License Agreement. Intellectual Property The company have established, and will continue to build, proprietary positions for rezafungin, CD388, CD421, and other product candidates and technology in the U.S. and abroad. As of March 23, 2023, the company’s portfolio included 10 families of patents and patent applications related to various aspects of rezafungin, 7 families of patents and patent applications related to various aspects of CD388, and one patent family of patent applications related to various aspects of CD421. For its issued patents related to rezafungin, the company expects the last to expire in 2038, excluding any additional term for patent term adjustments or applicable patent term extensions. With respect to CD388, the latest of any patents that result from the company’s pending applications would be expected to expire in 2042, should they be issued, excluding any additional term for patent term adjustments or applicable patent term extensions. With respect to CD421, the latest of any patents that result from the company’s pending applications would be expected to expire in 2043, should they be issued, excluding any additional term for patent term adjustments or applicable patent term extensions. The U.S. Food and Drug Administration, has granted rezafungin designations as an Orphan Drug, QIDP and Fast Track for the treatment of candidemia and invasive candidiasis which together provide 12 years of marketing exclusivity in the U.S. to be granted at the time of the U.S. Food and Drug Administration, approval. The U.S. Food and Drug Administration, has also granted rezafungin designations for QIDP and Fast Track for prophylactic use in patients undergoing allogeneic blood and marrow transplant which provides a five-year extension to any marketing exclusivity period for which the drug qualifies on approval. The European Commission has granted rezafungin Orphan Drug Designation for the treatment of invasive candidiasis which provides 10 years of market exclusivity protection from similar medicines with similar indications upon approval of the marketing authorization, and the potential for two additional years of market exclusivity when the results from pediatric studies compliant with an approved PIP are included in the SmPC. Further, the company seeks trademark protection in the U.S. and internationally where available and when appropriate. The company has filed for trademark protection in several countries for the Cidara trademark, which it uses in connection with its pharmaceutical research and development services and its pharmaceutical compounds. The company has registered trademarks for the Cidara mark in the U.S., the EU, Australia and Canada. Strategy The key elements of the company’s strategy are to develop product candidates from its Cloudbreak platform; and advance rezafungin to approva. Research and development expenses The company’s research and development expenses were $75.5 million for the year ended December 31, 2022. Government Regulations Any drug manufactured or distributed by the company pursuant to the U.S. Food and Drug Administration, approvals is subject to pervasive and continuing regulation by the U.S. Food and Drug Administration, including among other things, requirements relating to recordkeeping, periodic reporting, product sampling and distribution, advertising and promotion and reporting of adverse experiences with the product. History The company was founded in 2012. It was incorporated in Delaware in 2012 as K2 Therapeutics, Inc. and changed its name to Cidara Therapeutics, Inc. in 2014.