Exicure, Inc. (NASDAQ: XCUR), a pioneer of gene regulatory and immunotherapeutic drugs using spherical nucleic acid (SNA ™) technology, today announced that the U.S. Food and Drug Administration (FDA) United has granted Fast Track designations for its clinical product candidate, cavrotolimod (AST-008), for two development programs:
cavrotolimod in combination with anti-programmed death-1 (PD-1) therapy for the treatment of patients with locally advanced or metastatic Merkel cell carcinoma (MCC) refractory to previous anti-PD-1 blockade; and
cavrotolimod in combination with anti-PD-1 / anti-PD-ligand 1 (anti-PD- (L) 1) therapy for the treatment of patients with locally advanced or metastatic cutaneous squamous cell carcinoma (CSCC) refractory to anti -DD anterior – (L) 1 blockade
Fast Track is a designation granted by the FDA intended to facilitate the development and expedite review of drugs to address an unmet medical need in the treatment of serious life-threatening illness, and for which data Non-clinical or clinical have demonstrated the potential of the drug candidate to meet this medical need.
“There is an urgent need to study new immunotherapeutic agents such as cavrotolimod that can be administered to improve the clinical efficacy of immunotherapy, particularly in patients with refractory solid tumors,” said Dr Adil Daud, MD , Clinical Professor at UCSF Helen Diller Family Comprehensive Cancer Center and Principal Investigator in the Phase 1b / 2 clinical trial of cavrotolimod.
Cavrotolimod is a spherical nucleic acid receptor 9 (TLR9) agonist designed to robustly activate the innate and adaptive immune systems of the patient to potentially induce potent anti-cancer immune responses. The phase 1b dose escalation step of the multicenter, open-label trial was designed to assess the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of intratumoral injections of cavrotolimod alone and in combination with intravenous pembrolizumab in patients with advanced solid tumors. Patients in phase 1b included those with advanced or metastatic MCC, squamous cell carcinoma of the head and neck, CSCC, melanoma, and leiomyosarcoma. A summary of the main highlights of Phase 1b of the trial includes:
– No serious adverse event (“SAE”) related to the treatment or toxic limiting the dose (DLT) was observed;
– Overall response rate (ORR) confirmed in 21% of evaluable patients (4/19 patients) at the Phase 1b dose escalation stage at all doses, reflecting 1 complete response and 3 partial responses;
– TRG confirmed in 33% of patients evaluated (2/6 patients) in the highest dose cohort (32 mg), which was selected as the recommended dose for phase 2;
– Global responses occurred in two patients with advanced MCC and two patients with melanoma;
– Three of the four responders were progressing on anti-PD-1 treatment at the time of inclusion in the trial;
– Long-lasting and continuous responses, with progression-free survival greater than six months in the four responders and 16 months in two responders;
– In addition to the four overall responses, a decrease in target tumor occurred in one CSCC patient and two melanoma patients;
– Increase in leukocytes in tumors injected after cavrotolimod (AST-008) alone and in combination with pembrolizumab compared to inclusion. Uninjected tumors also showed increased levels of immune cells after patients received cavrotolimod (AST-008) plus pembrolizumab;
– Dose-dependent activation of key immune cells, including cytotoxic T cells and natural killer cells, as well as increases in cytokine / chemokine levels in the blood of patients after treatment with cavrotolimod (AST-008) alone and cavrotolimod (AST-008) plus pembrolizumab treatment;
– Systemic (abscopal) effects were observed, with regression in non-injected tumors far from the injected lesions; and
– The pharmacodynamic profile of cavrotolimod corroborated the efficacy data, as an increase in serum cytokines / chemokines, activated immune cells and tumor infiltration by immune cells were observed.
“I am encouraged by the results of the Phase 1b dose escalation and excited about the potential of cavrotolimod to address the significant unmet needs facing these patients,” said Dr. Michael Wong, MD, Ph.D. ., professor at the MD Anderson Cancer Center and principal investigator in the phase 1b / 2 clinical trial of cavrotolimod.
“These Fast Track designations underscore the pressing need to develop new therapies to treat refractory non-melanoma skin cancers as well as the promising preclinical and initial clinical results of cavrotolimod in patients with locally advanced Merkel cell carcinoma. or metastatic and cutaneous squamous cell carcinoma. said Dr. Shailender Bhatia, MD, associate professor at the University of Washington / Fred Hutchinson Cancer Research Center and principal investigator in the phase 1b / 2 clinical trial of cavrotolimod.
About Exicure, Inc.
Exicure, Inc. is a clinical-stage biotechnology company that develops therapies for neurology, immuno-oncology, inflammatory diseases, and other genetic disorders based on our proprietary spherical nucleic acid or ANS technology. Exicure believes its proprietary SNA architecture has distinct chemical and biological properties that may offer advantages over other nucleic acid-based therapies and may have therapeutic potential to target diseases that are typically not treated with d other nucleic acid therapies. Exicure is in preclinical development of XCUR-FXN, an ANS-based therapeutic candidate, for the treatment of Friedreich’s ataxia (FA). Exicure’s therapeutic candidate, cavrotolimod, is in a Phase 1b / 2 clinical trial in patients with advanced solid tumors. Exicure is based in Chicago, IL and Cambridge, MA.
For more information, visit the Exicure website at www.exicuretx.com.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release other than statements of historical fact can be considered as forward-looking, including, but not limited to Limited therein, statements regarding the company’s ability to advance cavrotolimod (AST-008) into a phase 2 clinical trial and its potential benefits as a treatment for Merkel cell carcinoma (MCC) and cutaneous squamous cell carcinoma. The forward-looking statements contained in this press release speak only as of the date of this press release, and the company does not undertake to update these forward-looking statements. Forward-looking statements are based on management’s current beliefs and assumptions which are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement due to various factors, including, but not limited to: the risks that the ongoing COVID-19 pandemic could disrupt the business of the company and / or the global healthcare system more severely than it is to date or more severe than expected, which may impact or delay the Company’s ongoing Phase 1b / 2 clinical trial; unforeseen costs, charges or expenses that reduce the capital resources of the business; the Company’s preclinical or clinical programs do not advance or result in approved products in a timely or cost-effective manner, or at all; the results of the first clinical trials are not always predictive of future results; the cost, timing and results of clinical trials; that many drug candidates do not become approved drugs in a timely or cost-effective manner, if at all; the ability to enroll patients in clinical trials; possible safety and efficacy concerns; regulatory changes; and the company’s ability to protect its intellectual property rights. For a discussion of other risks and uncertainties, and other important factors, each of which could cause the Company’s actual results to differ from those contained in forward-looking statements, see the section entitled “Risk Factors” in the company’s quarterly report on Form 10-Q for the quarter ended September 30, 2020, as updated by the company’s subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of publication, and the company does not undertake to update this information except as required by law.
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