Our Pipeline
Our drug development pipeline is focused on developing therapies that overcome life-threatening diseases caused by life-saving therapies.
Fosmanogepix (APX001)
Highly differentiated antifungal therapy in new therapeutic class
Fosmanogepix is a unique antifungal therapy with a novel mechanism of action and broad spectrum activity, including to strains that are resistant to standard of care therapy. Fosmanogepix is currently in Phase 2 clinical trials evaluating the efficacy and safety of both intravenous (IV) and oral formulations for the treatment of patients with fungal infections caused by aspergillosis and candidemia, including candidemia caused by C. auris.
In Phase 1 trials, both IV and oral formulations of fosmanogepix were well-tolerated with a favorable safety profile at clinically effective doses. Both formulations have a low propensity for clinically important drug-drug interactions. In vitro studies have demonstrated that manogepix, the active moiety of fosmanogepix, has activity against echinocandin-resistant Candida, azole-resistant Aspergillus, and multidrug-resistant fungi, and can be widely distributed to various tissues including the brain, lung, kidney and eye. With both IV and oral formulations, Fosmanogepix allows for a convenient transition from IV to oral, and continuation of care outside of the hospital.
Fosmanogepix has a novel mechanism of action that inhibits the highly conserved fungal enzyme Gwt1, which is essential for trafficking and anchoring mannoprotein to the outer cell wall in fungi. Mannoproteins are required for fungal cell wall integrity, adhesion, pathogenicity, and for evading the host immune system. Fosmanogepix does not inhibit the closest human homolog to Gwt1, the PIGW protein, and as a result, fosmanogepix compromises the growth of fungal pathogens without harming healthy cells of patients.
Fosmanogepix Publications
Posters and Presentations
Manuscripts
MAU868
Highly potent anti-BKV neutralizing antibody with potential to be the first treatment for BKV disease
The BK virus, which remains latent in up to 75-90% of people worldwide, can cause significant complications in immunocompromised individuals, particularly kidney transplant and hematopoietic cell transplant (HCT) recipients. MAU868 is a potent, human monoclonal antibody (immunoglobulin G, IgG1/λ isotype subclass) that potently neutralizes all four BKV serotypes. It recognizes a conformational epitope on the viral capsid protein, VP1, which is responsible for binding to and the infection of new cells.
MAU868 is being developed as the first antiviral drug for the prevention and/or treatment of BKV disease in kidney transplant recipients and HCT recipients, potentially transforming post-transplantation care and management. MAU868 also has neutralizing activity against the closely related JC virus, the cause of progressive multifocal leukoencephalopathy.
MAU868 has the potential to off-set expensive complications in renal transplant patients by avoiding the most common cause of viral allograft loss and significantly simplify the immunosuppressive therapy regimen that clinicians currently face. Successful treatment or prevention of hemorrhagic cystitis in HCT recipients would eliminate or reduce a complication that is associated with significant morbidity and mortality that extends hospitalizations, blood transfusions and acute graft-versus-host disease.
MAU868 Publications
CLINICAL TRIALS
What Are Clinical Trials?
Clinical trials play a crucial role in the process of bringing new therapies to patients, and lead us towards new possibilities to diagnose, treat and prevent diseases. This requires volunteers who may potentially benefit from a new innovative therapy when there are no available treatments, or when current treatments are poorly tolerated or ineffective. Studies evaluating all investigational drugs are highly regulated by the U.S. Food and Drug Administration (FDA) and other global regulatory authorities, and are only initiated if there is a potential therapeutic benefit supported by data. Clinical trials may also lead to a better understanding of the disease, and the therapies under development to treat those diseases.
Our Clinical Trials
Amplyx is developing therapeutics with proven modalities to treat those who are susceptible to life-threatening conditions that arise when the immune system is compromised. Fosmanogepix is a novel small molecule being developed for the treatment of invasive fungal infections that are often resistant to current antifungals. MAU868 is a monoclonal antibody that potently neutralizes the BK virus and is under development for BKV-associated nephropathy and BVK-associated hemorrhage cystitis.
We are currently conducting several clinical trials of fosmanogepix and MAU868 for people with life-threatening conditions as a result of a compromised immune system.
Current Clinical Trials
Below is a list of our ongoing trials. For more information, please email us at clinical@amplyx.com or visit www.clinicaltrials.gov.
An Efficacy and Safety Study of APX001 in Non-Neutropenic Patients With Candidemia
(Enrollment Complete)
An Open-label Study of APX001 for Treatment of Patients With Candidemia/Invasive Candidiasis Caused by Candida Auris (APEX)
(Enrollment Complete)
Expanded Access
Amplyx is committed to developing safe and effective first-in-class therapies to address severe and potentially life-threatening diseases that affect vulnerable, immunocompromised patients. Our goal is to provide access to our investigational therapies at the appropriate time and in the correct manner for patients.
The most appropriate mechanism to access investigational therapies is in the context of a clinical trial. We recognize, however, that there are circumstances that may arise that prohibit a patient’s participation in a clinical trial. We are committed to making investigational products available to seriously ill patients who have exhausted other treatment options.
A treating physician practicing within the United States may request information about how to apply for access to Amplyx’s investigational products by contacting us at medinfo@amplyx.com. Requests from treating physicians outside of the United States are not supported at this time.