FDA Approves Matinas BioPharma's Single Phase 3 Trial for MAT2203 Against Invasive Aspergillosis

Matinas BioPharma, a clinical-stage biopharmaceutical company, is set to conduct a pivotal Phase 3 study known as ORALTO. The trial will investigate MAT2203, an oral step-down treatment for adults suffering from invasive aspergillosis who have few or no other treatment options. The study will involve around 216 participants and will focus on all-cause mortality at day 42 as its primary endpoint. A key secondary endpoint will assess the superiority of MAT2203 over AmBisome® in terms of treatment-related toxicities that result in treatment adjustments or discontinuations.

The ORALTO trial is designed following the successful Phase 2 EnACT trial, which established MAT2203’s efficacy and safety as a step-down and all-oral therapy for HIV patients with cryptococcal meningitis. The study will also draw on Matinas’ ongoing Compassionate/Expanded Use Access Program that provides treatment to individuals with severe invasive fungal infections who have exhausted other options.

Dr. Theresa Matkovits, Chief Development Officer at Matinas, expressed satisfaction with the FDA's alignment on the Phase 3 trial design, highlighting the importance of collaborative work with the FDA in reaching a consensus on the registration path for MAT2203. Jerome D. Jabbour, CEO of Matinas, emphasized the significance of FDA support and the company's drive to secure a partnership to expedite MAT2203’s advancement into the Phase 3 trial.

The ORALTO trial is structured as a randomized, multicenter, open-label study with an adjudicator-blinded approach. It aims to evaluate MAT2203’s efficacy and safety compared to the standard of care in patients with limited treatment options. The trial’s primary efficacy endpoint is all-cause mortality at day 42, with secondary objectives including the superiority of MAT2203 in reducing treatment-related toxicities, long-term survival benefits, and the impact on healthcare resource utilization and quality of life.

Enrollment will target approximately 216 adults diagnosed with probable or proven invasive aspergillosis, who are unable to receive IV mold-active azoles and have limited alternatives. After initial treatment with AmBisome, eligible participants will be randomized in a 2:1 ratio to receive MAT2203 or continued AmBisome treatment.

Participants will receive up to 12 weeks of treatment, starting from the first day of AmBisome treatment. An independent Data Review Committee will adjudicate primary and secondary endpoints, and a Data Safety Monitoring Board will review the overall mortality rate once approximately 75% of participants are enrolled.

The global ORALTO trial is expected to commence in the second half of 2024 across 65 sites and will likely take around 24 months to complete.

Invasive aspergillosis is a serious fungal infection predominantly affecting immunocompromised patients, such as those with hematological malignancies or transplant recipients. The condition has been escalating globally and was recognized by the World Health Organization as a critical public health concern. The one-year mortality rate for IA patients is high, particularly in transplant recipients, and treatment options are often limited by toxicities and drug resistance.

MAT2203 is being developed as a potential oral broad-spectrum treatment for invasive fungal infections. It aims to overcome the limitations of current amphotericin B products by offering targeted oral delivery, which could reduce toxicity risks and create an ideal antifungal agent. MAT2203 has shown promise in the Phase 2 EnACT study for cryptococcal meningitis.

Matinas BioPharma is leveraging its lipid nanocrystal (LNC) platform delivery technology to develop groundbreaking therapies. The company's LNC technology has the potential to address challenges in the intracellular delivery of various molecular cargos, positioning it as a next-generation orally available intracellular drug delivery platform.