Cidara Therapeutics, Inc. operates as a biotechnology company. The company focuses on developing targeted therapies designed to save lives and improve the standard of care for patients facing serious diseases.
The company’s first commercially approved product in the United States, or U.S., is REZZAYO (rezafungin for injection) which is indicated for the treatment of candidemia and invasive candidiasis in adults with limited or no alternative treatment options.
On July 31, 2023, Melinta Therape...
Cidara Therapeutics, Inc. operates as a biotechnology company. The company focuses on developing targeted therapies designed to save lives and improve the standard of care for patients facing serious diseases.
The company’s first commercially approved product in the United States, or U.S., is REZZAYO (rezafungin for injection) which is indicated for the treatment of candidemia and invasive candidiasis in adults with limited or no alternative treatment options.
On July 31, 2023, Melinta Therapeutics, LLC, or Melinta, the company’s commercial partner in the U.S., initiated the commercial launch of REZZAYO in the U.S. On October 2, 2023, Melinta announced receipt from the Centers for Medicare & Medicaid Services, or CMS, of both a product-specific J-Code and a new technology add-on payment, or NTAP, for REZZAYO. Outside the U.S. and Japan, the company’s development and commercial partner for REZZAYO is Mundipharma Medical Company, or Mundipharma. In December 2023, the European Medicines Agency, or EMA, granted approval for REZZAYO in the European Union, or EU, for the treatment of invasive candidiasis in adults. In January 2024, the United Kingdom, or the U.K., Medicines and Healthcare products Regulatory Agency, or MHRA, granted approval for REZZAYO for the treatment of invasive candidiasis in adults.
Although the company has shifted its primary research focus to its proprietary Cloudbreak platform, it continues to execute on the ongoing ReSPECT Phase 3 pivotal clinical trial for the prevention of invasive fungal infections in adult allogeneic blood and marrow transplant recipients. A significant portion of its future royalties and milestones to be received under both Melinta and Mundipharma licensing agreements are tied to the successful completion of the ReSPECT Phase 3 trial.
The company’s proprietary Cloudbreak platform enables development of novel drug-Fc conjugates, or DFCs, that inhibit specific disease targets while simultaneously engaging the immune system. The company’s most advanced DFC program is CD388, a highly potent 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 multiple oncology and autoimmune indications.
Cloudbreak Platform
The company’s Cloudbreak platform has the potential to offer a fundamentally new approach to treat and prevent serious diseases, such as solid tumor cancers and viral infections, 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, its Cloudbreak DFC candidates are designed to counter diseases in two ways: prevention of disease proliferation and immune evasion by directly targeting and, where applicable, by focusing the immune system on a pathogen or infected cell. This is a potentially transformative approach, distinct from current therapies, including antibody drug conjugates, or ADCs, monoclonal or multispecific antibodies and vaccines.
The company’s lead Cloudbreak candidate for the prevention of influenza is CD388, a DFC in Phase 1 and Phase 2a clinical trials. The company’s lead oncology DFC is CBO421, a development candidate targeting CD73 for the treatment of solid tumors, which is in investigational new drug application, or IND, -enabling studies.
Cloudbreak Influenza Program
In June 2023, the U.S. Food and Drug Administration, or FDA, granted Fast Track designation to CD388 for the prevention of influenza A and B infection in adults who are at high risk of influenza complications due to underlying immunodeficiency and may not mount an adequate response to influenza vaccine or are at high risk of severe influenza despite influenza vaccination, including those for whom vaccines are contraindicated. Fast Track designation aims to facilitate the development and expedite the review of drugs to treat serious conditions with unmet medical needs. The purpose is to get important new drugs to patients earlier.
Final CD388 Phase 2a Results
In its recent R&D Day, on September 21, 2023, the company announced efficacy and safety data from its Phase 1 and Phase 2a trials evaluating the pre-exposure prophylactic activity of CD388 against an H3N2 influenza A virus strain.
Janssen Collaboration Agreement
On March 31, 2021, the company entered into an 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 IND-enabling nonclinical studies and early-stage clinical trials under the research plan. Both parties are responsible for conducting certain specified chemistry, manufacturing and controls, or CMC, development activities under the research plan. Janssen is solely responsible, and reimburses it for internal personnel and out-of-pocket costs incurred in performing the research plan activities in accordance with an agreed budget. As part of a recent prioritization of its R&D business, in July 2023 Janssen disclosed its intention to discontinue internal development of multiple product candidates in its infectious disease pipeline, including CD388. However, in September 2023 Janssen delivered its Election to Proceed Notice for CD388 whereby Janssen will assume the future development, manufacturing and commercialization activities of CD388 but intends to transfer its rights and obligations under the Janssen Collaboration Agreement to another transferee. The company continues to work in collaboration with Janssen to complete the Phase 1 and Phase 2a clinical trials and will be reimbursed for all ongoing development activities by Janssen as per the Janssen Collaboration Agreement.
Cloudbreak Oncology Programs
The company has expanded the Cloudbreak platform beyond infectious diseases, to discover and develop highly potent DFCs that can target multiple immune checkpoint pathways within a single DFC for oncologic diseases.
The company’s lead oncology DFC candidate, CBO421, is a potential best-in-class CD73 inhibitor that combines the strengths of small molecules and monoclonal antibodies targeting CD73. CBO421 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. The CD73 pathway is clinically validated in early/mid-stage clinical studies to reduce tumor growth in combination with PD-1/ PD-L1 inhibitors in disease areas that do not historically respond to checkpoint inhibition alone, such as advanced colorectal cancer, or CRC, and non-small cell lung cancer, or NSCLC. As a monotherapy and in combination with PD-1 inhibitors, CBO421 has demonstrated formation of immunologic memory in multiple murine tumor models, along with potential best-in-class activity in T-cell reactivation assays and tumor penetration compared with the most advanced CD73 antibody therapeutics in clinical development. The company is advancing CBO421 through IND-enabling studies and expect to file an IND in mid-2024.
In February 2023, the company expanded its existing collaboration with WuXi XDC, a leading global contract manufacturing organization dedicated to end-to-end bioconjugates services, under which WuXi XDC will provide IND-enabling CMC development services for its Cloudbreak Oncology program.
Rezafungin
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.
FDA Approval of Rezafungin for the Treatment of Candidemia and Invasive Candidiasis
In March 2023, the FDA approved REZZAYO 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 15 years, and is the only available once-weekly echinocandin.
EMA and UK MHRA Approval of Rezafungin for the Treatment of Invasive Candidiasis in Adults
In December 2023, the EMA granted approval for REZZAYO in the EU for the treatment of invasive candidiasis in adults. In January 2024, the UK MHRA granted approval for REZZAYO for the treatment of invasive candidiasis in adults.
REZZAYO Commercialization in the U.S. by Melinta
On July 31, 2023, Melinta initiated the commercial launch of REZZAYO in the U.S.
ReSTORE Phase 3 clinical trial in China.
In December 2023, enrollment in the ReSTORE Phase 3 trial in China, evaluating the efficacy and safety of rezafungin as a treatment for candidemia and invasive candidiasis, was completed. The portion of the trial conducted in China included 52 patients diagnosed with candidemia and/or invasive candidiasis. ReSTORE (NCT03667690) is a global, randomized, double-blind, controlled Phase 3 pivotal clinical trial evaluating the efficacy and safety of once-weekly intravenous dosing of rezafungin compared to once-daily dosing of caspofungin, the current standard of care, to treat patients with candidemia and/or invasive candidiasis. Data from this study are expected in the second quarter of 2024.
ReSPECT Phase 3 Clinical Trial
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 FDA and EMA is fungal-free survival at Day 90. The company expects this trial to enroll approximately 462 patients. The study is currently enrolling in the EU, Canada and the U.S.
Melinta License Agreement
On July 26, 2022, the company entered into a License Agreement with Melinta, or the Melinta License Agreement, under which it granted Melinta an exclusive license to develop and commercialize products that contain or incorporate rezafungin in the U.S.
Melinta is 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.
Mundipharma Collaboration Agreement
On September 3, 2019, the company announced a strategic partnership with 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 with Mundipharma, or the Mundipharma Collaboration Agreement, the company granted Mundipharma an exclusive, royalty-bearing license to develop, register and commercialize rezafungin outside the U.S. and Japan.
License and Collaboration Agreements
Mundipharma Collaboration Agreement
On September 3, 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.
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, subject to its retained right.
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.
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 the company 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.
The company continues to work in collaboration with Janssen to complete the Phase 1 and Phase 2a clinical trials and will be reimbursed for all ongoing development activities by Janssen as per the Janssen Collaboration Agreement.
Melinta License Agreement
On July 26, 2022, the company entered into the Melinta License Agreement with 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 is 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 has established, and will continue to build, proprietary positions for rezafungin, CD388, CBO421, and other product candidates and technology in the U.S. and abroad. As of April 22, 2024, the company’s patent 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 CBO421.
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 CBO421, 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 FDA 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 FDA approval. In March 2023, the FDA approved REZZAYO for the treatment of candidemia and invasive candidiasis in adults with limited or no alternative treatment options. The FDA 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. In December 2023, the EMA approved REZZAYO in the EU for the treatment of invasive candidiasis in adults.
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 $68.5 million for the year ended December 31, 2023.
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.
