Sabrina Serani

On July 30, the FDA will convene the Cellular, Tissue, and Gene Therapies Advisory Committee to determine whether data from the IGNYTE trial can support an accelerated approval of RP1 plus nivolumab.

A 75.5% complete response rate surpassed historical expectations for BCG-unresponsive CIS, including patients previously exposed to gemcitabine-docetaxel or pembrolizumab. Durability was notable, with 60.1% maintaining response at 24 months and median DOR not reached at ≥27.9 months; rare long-term disease-free survival exceeded 51 months.

ARTEMIS-011 randomized 117 patients 2:1 to risvutatug rezetecan 12.0 mg/m² IV q3w versus gemcitabine/docetaxel after failure of ≥2 prior systemic lines. Primary endpoint PFS was met with statistically significant, clinically meaningful improvement, and efficacy trends were consistent across key secondary endpoints including overall survival.

Dual fast track and RMAT designations enable more frequent FDA engagement and potentially accelerated development for MRD-positive LBCL consolidation after first-line response. ALPHA3 randomizes ctDNA MRD–positive patients in remission post-chemoimmunotherapy to single-dose cema-cel or standard observation, using Natera’s CLARITY phased-variant MRD assay.

Priority review positions subcutaneous amivantamab as a potential first-in-class EGFR/MET-targeted option after platinum and PD-1/PD-L1 failure in HPV-unrelated R/M HNSCC. Blinded central review showed 42% ORR with 15% CR; 56% of responses persisted ≥6 months and 63% were ongoing at analysis.

A 10-3 advisory vote endorsed using ORR and DOR to support accelerated approval in anti–PD-1-refractory advanced melanoma, emphasizing unmet need and perceived magnitude beyond historical outcomes. RP1 is an HSV-1 oncolytic virus encoding GM-CSF and GALV-GP-R−, intended to lyse injected tumors and prime systemic immunity when combined with nivolumab.

Orphan designation for ALL strengthens the development pathway for enzomenib and may facilitate investment, regulatory engagement, and future commercialization in molecularly defined acute leukemias. Enzomenib disrupts menin–KMT2A biology, with selective antiproliferative effects in KMT2A-rearranged or NPM1-mutant leukemia models and downstream suppression of HOXA9/MEIS1 transcriptional programs.

Regulatory clearance covers PSMA-positive mAPMN/S disease, emphasizing PSMA PET–based patient selection using gallium Ga 68 gozetotide or another approved PSMA PET product. PSMAddition randomized 1144 patients to [177Lu]Lu-PSMA-617 plus ARPI versus ARPI alone, with continuous ADT via GnRH agonist/antagonist or prior orchiectomy.

Erika Hamilton, MD, discusses HER2CLIMB-05 data showing tucatinib plus HP maintenance extends PFS in HER2+ breast cancer, including brain metastases.

Sara Hurvitz, MD, discusses VIKTORIA-1 data on gedatolisib in PIK3CA-mutant HR+/HER2- breast cancer, following the drug's recent FDA approval.