FDA Clears IND for Novel AML Degrader, Approves Prostate Cancer Imaging Agent, Accepts Skin Cancer Therapy Filing

The U.S. Food and Drug Administration has recently taken a series of significant regulatory actions in oncology, clearing an investigational new drug application for a novel leukemia treatment, approving a new formulation of a prostate cancer imaging agent, and accepting a supplemental application for a skin cancer therapy.

Amphista Therapeutics announced that the FDA has cleared its IND application for AMX-883, an orally available, non-cereblon-degrader of BRD9, for the treatment of acute myeloid leukaemia (AML). The company states that AMX-883 achieves nearly complete BRD9 degradation within two hours and is expected to enter a Phase I monotherapy dose-escalation clinical trial for patients with relapsed or refractory AML in the second half of 2026. The trial will also include patients with high-risk myelodysplastic syndrome (MDS). The company noted that after establishing the monotherapy profile, it intends to explore AMX-883 in combination with venetoclax and azacitidine in earlier lines of therapy. Amphista's chief medical officer highlighted that AML remains one of the most devastating blood cancers, with a five-year survival rate of just 33% and resistance to standard-of-care treatments such as venetoclax a critical challenge.

Separately, the FDA approved a new formulation of Lantheus Holdings' prostate cancer imaging agent, Pylarify, called Pylarify TruVu. The new version maintains the same diagnostic performance but is engineered to handle higher radioactive concentrations, allowing for larger production batches and more efficient distribution. The company expects the new formulation to increase batch size by about 50% and help suppliers reach geographic areas where clinics do not yet have access to PSMA PET scans. Lantheus plans to launch Pylarify TruVu in the fourth quarter of 2026 with a phased rollout, timed to coincide with new reimbursement codes taking effect on October 1. Studies have shown that Pylarify achieves an 86% median true-positive rate in identifying recurrent prostate cancer.

In a third development, Biofrontera announced the FDA has accepted its supplemental New Drug Application (sNDA) for Ameluz (aminolevulinic acid hydrochloride) topical gel, combined with the RhodoLED red-light lamp, to treat superficial basal cell carcinoma (sBCC). If approved, Ameluz would become the first and only photodynamic therapy (PDT) photosensitizer for sBCC in the United States, expanding its existing indication for actinic keratosis. Basal cell carcinoma is the most common skin cancer in the country, with 3.6 million cases diagnosed annually, of which an estimated 10–25% are superficial. The company stated the FDA acknowledged no filing deficiencies in its sNDA.