SAN DIEGO—Amplyx Pharmaceuticals, Inc. has advanced the development of the company’s lead program, fosmanogepix (APX001), by completing enrollment in a Phase 2 trial evaluating the safety and efficacy of fosmanogepix for the treatment of Candida infections. Top-line data from the trial is expected by July, and an initial analysis of the first 10 patients enrolled showed a high level of treatment success.
“There is an urgent need for effective new antifungal agents, especially those with activity against Candida auris and Aspergillus,” said Thomas F. Patterson, M.D., chief, division of infectious diseases, UT Health San Antonio. “These infections are increasingly difficult to treat, and the rise in the rate of infections is alarming. The need for safe and effective therapies with new and different mechanisms of actions is critical, especially with the growing number of immunocompromised patients who are at increased risk for developing these life-threatening infections.”
Fosmanogepix is a novel, broad-spectrum antifungal agent. APX001 has a novel mechanism of action, and its active moiety has shown broad-spectrum activity against common species of Candida and Aspergillus — including multi-drug-resistant strains, such as C. auris and C. glabrata — as well as rare, hard-to-treat molds like Fusarium, Scedosporium and some fungi from the Mucorales order.
The therapy is being evaluated in multiple clinical trials in patients with life-threatening fungal infections. The first patients in the Phase 2 Candida trial have also completed treatment in two additional Phase 2 clinical trials evaluating the safety and efficacy of APX001 for the treatment of Aspergillus and rare molds, as well as a study of invasive candidiasis caused by Candida auris.
Invasive fungal infections result in high mortality rates (30-80 percent), despite standard-of-care treatment. The frequency of fungi resistant to both the azole and echinocandin classes of drugs is increasing, and there is a significant unmet medical need for a new broad-spectrum antifungal agent. In 2019, the U.S. Centers for Disease Control recognized drug-resistant C. auris as an urgent threat to global public health. Other drug-resistant Candida strains are recognized as serious threats, and the emergence of drug-resistant Aspergillus has been added to the watch list for global health threats. This underscores the need for innovation in antifungal development.
“Given the increasing threat of drug-resistant fungal pathogens, including certain strains that are resistant to all currently approved classes of antifungal drugs, there is a clear need to develop compounds that have new mechanisms of action. With its unique mechanism of action and activity against drug-resistant strains, fosmanogepix has the potential to address this important unmet need,” added Ciara Kennedy, Ph.D., president and chief executive officer of Amplyx.
The ongoing Aspergillus Phase 2 study is evaluating the safety and efficacy of both intravenous and oral fosmanogepix for the treatment of patients with invasive mold infections caused by Aspergillus species and rare molds. The C. auris Phase 2 study is also ongoing, evaluating the safety and efficacy of both intravenous and oral fosmanogepix for the treatment of patients with candidemia, caused by C. auris —including patients with suspected or confirmed resistance to standard-of-care antifungal treatments.
Fosmanogepix has received Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration for seven separate indications, and is designated as a Qualified Infectious Disease Product for the treatment of four indications.
Amplyx also reported today the closing of a $53 million Series C extension, which brings the Series C round total to over $90 million. The financing was led by Sofinnova Investments, with participation from existing investors. New equity investment from Pfizer Inc. and Adage Capital Management completed the round. Amplyx plans to use the proceeds of the financing to advance the clinical development of the company’s product candidates, fosmanogepix and MAU868.
“It is important that the growing threat of current and emerging treatment-resistant fungal pathogens is met by research and development efforts that deliver new treatment options for patients with invasive fungal infections,” noted Annaliesa Anderson, Ph.D., FAAM, vice president and chief scientific officer, bacterial vaccines and hospital, Pfizer. “We look forward to supporting Amplyx as they work to develop therapies with a novel approach.”