
Developing Medicines
to defeat diseases
when your immune system can't

Our Focus
Amplyx is focused on developing innovative therapies for debilitating and life-threatening diseases in patients with compromised immune systems. Our dedicated and experienced team is advancing development programs with the potential to protect these vulnerable patients, including those with cancer and transplants, and the critically ill.
At Amplyx, we are dedicated to developing innovative, safe and effective therapies to address these debilitating and life-threatening diseases in patients with compromised immune systems.
WHEN A FUNGAL INFECTION IS SCARIER THAN CANCER
Blake Gauss was diagnosed with leukemia at 2 years old. But it was a deadly fungal infection that nearly took his life. Read his story here.
About Us
Amplyx was founded with the mission to develop innovative therapies for debilitating and life-threatening diseases in patients with compromised immune systems. Our dedicated and experienced team is advancing development programs with the potential to protect these vulnerable patients, including those with cancer and transplants, and the critically ill.
The company’s two lead products are fosmanogepix (APX001) for the treatment of life-threatening fungal infections caused by pathogens such as Candida, Aspergillus and rare molds; and MAU868, a monoclonal antibody designed to neutralize the BK virus which can cause significant morbidity and mortality in transplant patients.
“Too often the recovery of patients struggling to overcome cancer, or a lifesaving transplant, is put at risk by the very treatments necessary to save their lives. Our mission is to bring innovative therapies to these vulnerable, immunocompromised patients and keep them on the path to renewed health.”
— Ciara Kennedy, Ph.D.,
President and CEO, Amplyx Pharmaceuticals
Management
Ciara Kennedy, Ph.D.
Mike Hodges, M.D.
Karen Joy Shaw, Ph.D.
Alan Fuhrman
Paul Bien
Susan Dubé
Elizabeth Gordon, Ph.D.
Pamela Wedel
News
Amplyx Pharmaceuticals Licenses Phase 2 Drug Candidate for BK Viral Disease in Transplant Patients
Amplyx Pharmaceuticals Announces Positive Preliminary Data from Ongoing Fosmanogepix Phase 2 Study in Candidemia
FDA Grants Fast Track Status to Amplyx Pharmaceuticals for IV and Oral Formulations of Fosmanogepix (APX001) for Seven Different Indications
FDA Grants Orphan Drug Designation to Amplyx Pharmaceuticals for APX001 for Treatment of Cryptococcosis
Press Release Archive | Poster Archive | Manuscript Archive
If you’re a member of the media and would like to reach Amplyx, please contact:
Heidi Chokeir, Ph.D.
Senior Vice President, Canale Communications
(619) 849-5377
heidi@canalecomm.com
Our Products

Fosmanogepix (APX001)
Fosmanogepix is a novel antifungal therapy with broad spectrum activity. Fosmanogepix is currently in Phase 2 clinical trials evaluating the efficacy and safety of both intravenous and oral formulations for the treatment of patients with fungal infections. Manogepix (APX001A), the active moiety of fosmanogepix (APX001), inhibits the highly conserved fungal enzyme Gwt1, compromising growth of fungal pathogens. The novel mechanism of action of fosmanogepix translates into a highly versatile drug that demonstrates activity against drug-resistant strains and can be delivered in both oral and intravenous formulations. In multiple nonclinical studies, manogepix has shown broad-spectrum activity against common species of Candida spp. and Aspergillus spp., including multi-drug resistant strains, such as C. auris and C. glabrata, as well as rare, hard-to-treat molds including Fusarium spp., Scedosporium spp., and fungi from the Mucorales order.
Invasive infections due to Aspergillus, Fusarium, Scedosporium and fungi from the Mucorales order are especially difficult to treat resulting in high mortality rates (50-80%), even when patients receive standard of care treatment. The frequency of fungi resistant to both the azole and echinocandin classes of drugs is increasing. Thus, there remains a significant unmet medical need for a new broad-spectrum antifungal to treat serious, invasive fungal infections and reduce the existing high morbidity and mortality.

MAU868
MAU868 is a novel human monoclonal antibody (immunoglobulin G, IgG1) that potently neutralizes all four BKV serotypes. MAU868 also neutralizes the closely related JC virus. MAU868 binds the viral capsid protein, VP1, which is responsible for binding to the surface of host cells. It recognizes a conformational epitope on VP1 that is highly conserved, resulting in a high barrier to resistance in vitro. MAU868 is being developed for the prevention and treatment of clinically significant BKV infection in kidney transplant and hematopoietic stem cell transplant patients.
Antibodies to BKV are found in approximately 80 to 90 percent of adults worldwide, indicating previous infection or exposure to the virus. Initial infection with BKV is usually asymptomatic or associated with a mild flu-like illness. After primary infection, BKV remains inactive, or latent, in kidney and bladder cells. A weakened immune system may give the virus a chance to reactivate and cause serious disease. In patients who have had kidney transplant, BK viral disease results in nephropathy and potential loss of graft function. In the absence of effective treatment, reduction of immunosuppression is recommended, but this ironically can lead to acute organ rejection. BKV re-activation can also occur after hematopoietic stem cell transplants, resulting in hemorrhagic cystitis, the symptoms of which include painful urination, blood in the urine and hemorrhage. Severe cases require bladder irrigation, clot evacuation, blood transfusion, stenting and nephrostomy. There are currently no approved treatments for BKV- associated disease.
Pipeline
Fosmanogepix
Preclinical
Phase 1
Phase 1b
Phase 2
Phase 3
APX001
Invasive Candidiasis/Candidemia
APX001
Invasive Aspergillosis
APX001
Candida auris
APX001
Cryptococcus
MAU868
Preclinical
Phase 1
Phase 1b
Phase 2
Phase 3
MAU868
BKV-associated nephropathy
MAU868
BKV-associated hemorrhagic cystitis
For additional information, please see Amplyx Clinical Trials.
Clinical Trials
An Open-Label Study to Evaluate the Efficacy and Safety of APX001 in Non-Neutropenic Patients with Candidemia, with or without Invasive Candidiasis, Inclusive of Patients with Suspected Resistance to Standard of Care Antifungal Treatment
An Open-Label, Multicenter Study to Determine the Safety and Pharmacokinetics of Intravenous and Oral APX001 in Patients Undergoing Chemotherapy for Acute Myeloid Leukemia with Neutropenia
A Phase 1, Randomized, Placebo-Controlled Study to Investigate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Oral Doses of APX001, to Investigate the Effect of Food on APX001 and to Investigate the Drug-Drug Interaction Potential of APX001
A First-in-Human, Randomized, Dose-Escalation, Double-Blind, Placebo Controlled Study to Assess Safety, Tolerability and Pharmacokinetics of APX001 Administered by Intravenous Infusion to Healthy Subjects
For additional information, please see Amplyx Clinical Trials.
Expanded Access
Amplyx is committed to developing safe and effective first-in-class products for the treatment of life-threatening infections, with a near-term focus on deadly fungal pathogens in vulnerable, immunocompromised patients. Our goal is to provide access to our investigational therapies at the appropriate time and in the correct manner for patients.
Amplyx believes that 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. At this time, Amplyx’s Expanded Access program is currently under development. We will update this page with additional information on this program once it is available. If you have any questions, please contact medinfo@amplyx.com.
Expanded Access
Amplyx is committed to developing safe and effective first-in-class products for the treatment of life-threatening diseases in immunocompromised patients. Our goal is to provide access to our investigational therapies at the appropriate time and in the correct manner for patients.
Amplyx believes that 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. At this time, Amplyx’s Expanded Access program is currently under development. We will update this page with additional information on this program once it is available. If you have any questions, please contact medinfo@amplyx.com.
Careers
Amplyx is driven by a mission to develop the best treatments for immunocompromised patients with life-threatening diseases. Our growing team brings a long history of drug development experience, effective company leadership and spirited collaboration. As we expand, we continue to welcome passionate and talented individuals who share our desire to create new possibilities in medicine.
Amplyx offers a competitive compensation package and a rewarding work environment that fosters innovation. We invite you to explore our career opportunities. If you don’t find a perfect job match on our website, but think Amplyx may be the right place for you, please submit your resume to careers@amplyx.com.
