Amplyx Inks Novartis Deal for Another Compromised-Immune System Drug

Xconomy San Diego — 

Amplyx Pharmaceuticals, which is testing a new type of drug against life-threatening infections acquired by patients with compromised immune systems, has acquired the rights to another drug that could also be of use in treating those with limited ability to fight off sickness.

On Monday, the clinical-stage San Diego biotech company announced it had added an experimental drug developed by Swiss pharma giant Novartis (NYSE: NVS) to its pipeline through a licensing deal. That compound, MAU868, is designed to target a protein associated with BK viral disease, an infection that occurs most often in people who have received a kidney or stem cell transplant.

Although most of us have the BK virus, it’s generally latent, or inactive—until a person’s immune system weakens, which can prompt the virus to reactivate.

Kidney transplant patients in whom the virus becomes reactivated can end up with nephropathy, which can cause their immune system to reject the new organ. And, in patients who have received a stem cell transplant, BK virus (BKV) can cause hemorrhagic cystitis, a painful bladder infection.

There are no FDA-approved treatments for either renal nephropathy or hemorrhagic cystitis caused by BKV, according to Amplyx.

Recently, Amplyx has been studying a compound called fosmanogepix (APX001) as a treatment for candidemia—a serious infection of the blood stream caused by Candida, a strain of fungus. It’s also testing the experimental drug in patients with other difficult-to-treat fungal infections, all caused by organisms that have exhibited resistance to available treatments.

Financial terms of the licensing deal with Novartis, which gives Amplyx worldwide rights to MAU868, weren’t disclosed. The company plans to start Phase 2 testing of the potential antiviral drug in early 2020.

The new compound is complementary to the company’s existing program, said CEO Ciara Kennedy (pictured), since a subset of the patients who would be likely to need the experimental BKV drug, such as people undergoing cancer treatment, could also potentially need a drug like Amplyx’s fosmanogepix.

“It’s got a great overlap in terms of both how you conduct clinical trials in this space, and, ultimately, the types of physicians that will be prescribing both of these drugs,” she said in a phone interview.

The company also announced Monday that a review of data from the first 10 patients in the Phase 2 trial of its lead compound—in which it anticipates enrolling 20 patients in total—showed “a high level of treatment success,” according to an independent review panel. A second independent panel recommended its continuation, according to a news release.

The company said successful treatment meant the patient was alive, with the fungus cleared from their blood, and received no other systemic antifungal therapies during the study.

It was the first indication the company has shared publicly about how the trial is proceeding.

Kennedy says the potential for a drug that works in a different way from existing treatments to attack fungal infections has drawn attention. Amplyx’s experimental drug attacks a fungal enzyme, Gwt1, and in doing so, interrupts the work of a protein that’s essential to the cell wall in fungi, causing damage that leads to cell death. The FDA hasn’t approved a new class of anti-fungal agents since 2001.

After demonstrating that fosmanogepix could be safely administered in Phase 1 testing, it is now under scrutiny as to its efficacy, she said.

“The question that’s left is, does it work, will it deal with the infection?” she said. “This data certainly supports that, and also supports that the preclinical data translates well to clinical efficacy, which is really important because we have an enormous amount of preclinical data across many different types of life-threatening fungal infections … So now we are feeling really good about the translatability of that preclinical data into a drug that could help a lot of different types of patients.”

Amplyx is also testing the drug as a treatment for infections caused by invasive aspergillosis, a mold; Candida auris, a pathogen the Centers for Disease Control and Prevention calls a “serious global health threat”; and cryptococcus, which can cause meningitis, a brain infection that occurs most commonly in people with weakened immune systems as a result of HIV/AIDS.