SAN DIEGO, CA, USA I February 20, 2018 I Vical Incorporated (Nasdaq:VICL) announced today the initiation of a Phase 2 trial of the Company’s VL-2397 novel antifungal compound. The multicenter, open label randomized clinical study, will compare the efficacy and safety of VL-2397 to standard treatment for invasive aspergillosis in acute leukemia patients and recipients of allogeneic hematopoietic cell transplant (HCT).
The U.S. Food and Drug Administration (FDA) has advised that VL‑2397 would be eligible for a Limited Use Indication (LUI) assuming a successful outcome of a single Phase 2 trial carried out in accordance with a protocol and statistical analysis plan consistent with the Agency’s advice. The final determination whether the drug is approvable will be made by FDA after review of all relevant data. The LUI is a provision of the Limited Population Pathway established under the 21st Century Cures Act of 2016.
“The Limited Population Pathway enables Vical to develop and potentially commercialize VL-2397 on an accelerated basis for a Limited Use Indication, and we are pleased that the Phase 2 trial is now underway,” said Vijay Samant, Vical’s President and CEO. “New antifungals, especially with novel mechanisms of action, are desperately needed to enhance treatments options when currently marketed therapies are inadequate due to their associated toxicities, drug-drug interactions, and the emergence of azole-resistant strains.”
The global Phase 2 trial is expected to enroll approximately 200 patients who will be randomized on a 2:1 basis with approximately 134 patients treated with VL-2397 and 66 patients treated with a standard 6-week course of their physician’s choice of voriconazole, isavuconazole, or liposomal amphotericin B. The patients in the VL-2397 arm will receive daily treatment with VL-2397 for 4 weeks, followed by a 2-week course of physician’s choice of the comparator. The primary endpoint of the trial is all-cause mortality at 4 weeks and the key secondary endpoint is all-cause mortality at 6 weeks. The trial will be conducted at selected sites in North America, Europe and Asia.
“The preclinical and clinical data to date for VL-2397 support the potential profile of an ideal antifungal agent which must be fast-acting, with low risk for toxicity and drug-drug interactions,” commented Peter Pappas, MD, Principal Investigator of the Mycoses Study Group (MSG) and Professor of Medicine in the Division of Infectious Diseases, Department of Medicine at the University of Alabama in Birmingham. “The MSG has been pleased to work with Vical in the development of this protocol, and we believe that this compound represents a potential alternative to existing antifungal agents to which resistance has developed, or where significant drug-associated toxicities are encountered.”
About the Limited Population Pathway
The LPP is designed to streamline development programs for certain antimicrobial agents intended to treat specific groups of patients who are not well addressed by available therapies for their serious or life-threatening infections. Under this pathway, the drug can be used to treat only the limited population for which it is approved while additional trials are conducted to establish safety and effectiveness for broader indications. Standards for a new drug application must be met for LUI approval. In the case of VL‑2397, the limited population approval would be for patients for whom alternative regimens are not available to treat their invasive aspergillosis. A Phase 3 trial would be required to support full approval of VL-2397 for the treatment of invasive aspergillosis in a broader population.
VL-2397 is Vical’s novel antifungal compound that was licensed from Astellas Pharma in 2015. VL-2397 was isolated from a leaf litter fungus collected in a Malaysian national park and represents the first agent in a potentially new class of antifungal drugs. The FDA has granted Vical Qualified Infectious Disease Product (QIDP), Orphan Drug and Fast Track designations for VL-2397 in the treatment of invasive aspergillosis.
About Invasive Aspergillosis
Invasive aspergillosis is a life-threatening infection that typically affects immunocompromised patients, including those with acute leukemia and recipients of allogeneic HCT or lung transplants. Infection typically starts in the lungs and rapidly disseminates to other tissues. More than 200,000 cases of invasive aspergillosis are diagnosed annually worldwide.
Vical develops biopharmaceutical products for the prevention and treatment of chronic or life-threatening infectious diseases, including antiviral and antifungal candidates in clinical development. Additional information on Vical is available at www.vical.com.