Patients undergoing immunosuppressive treatments, like chemotherapy, end up with compromised immune systems. As a consequence invasive fungal infections have emerged as a significant problem among cancer patients, causing both morbidity and mortality. In fact, at least 40-50% of fatal infections are caused by fungi. However, existing therapies for these infections are extremely limited. They are restricted by drug interactions, toxic side effects, ineffective against more potent fungi that have developed resistance, and not to mention expensive.
In order to overcome these limitations, the San-Diego-based startup Amplyx Pharmaceuticals is developing novel, broad-spectrum antifungal agents for the treatment of life-threatening invasive fungal infections. Their small-molecule therapy, APX001, inhibit the Gwt1 fungal enzyme, and therefore maintain its activity against fungal organisms that have developed resistance to other drug classes. The drug candidate has been extensively evaluated in preclinical studies and have demonstrated broad-spectrum activity against infections caused by Candida, Aspergillus, as well as rare and hard-to-treat molds. In addition, the startup is developing both an IV and oral formulation of the drug to address the need for hospital administration, as well as continued dosage after hospital discharge.
Increased awareness among physicians and biotechnological advances have had considerable impact on improving diagnosis of invasive fungal infections. However, it remain difficult to manage, and there is an urgent need for improved treatment. APX001 has the potential to be used as a front line agent, either alone or in combination with existing antifungal drugs. It could overcome limitations associated with existing therapies, and significantly improve treatment efficacy. Annually, there are over 600,000 cases of invasive fungal infections due to Candida, Aspergillus and rare molds, and the work by Amplyx could give new hope to every single one of these patients.
APX001 is expected to enter clinical development this year, and Amplyx has raised over $50 million in venture capital with investors including 3x5 Special Opportunity Fund, Biomed Ventures, New Enterprise Associates, RiverVest and Tech Coast Angels. The startup has also received more than $10 million in grants from the National Institutes of Health to support its drug discovery and development efforts. The startup’s research operations is located at Johnson & Johnson Innovation, JLABS.
The drug candidate has been granted Qualified Infectious Product (QIDP) by FDA, a designation established to encourage pharmaceutical companies to develop new antimicrobial drugs to treat serious and life-threatening infections. The designation provide significant incentives for the development of the drug, including priority FDA review, eligibility for Fast Track designation and a five-year extension of marketing exclusivity.