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The FDA granted fast track designation to AVZO-103 for urothelial cancer previously treated with enfortumab vedotin.
The FDA had granted fast track designation to the novel Nectin4- and TROP2-directed bispecific antibody-drug conjugate (ADC) AVZO-103 for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received enfortumab vedotin-ejfv (Padcev).1
The ongoing, first-in-human, open-label phase 1/2 AVZO-103-1001 trial (NCT07193511) is evaluating AVZO-103 as monotherapy and as part of combination therapy in patients with locally advanced or metastatic urothelial cancer or other solid tumors.1,2
“Receiving fast track designation for AVZO-103 highlights the significant need for treatment options for patients with urothelial cancer who have progressed on enfortumab vedotin,” Mohammad Hirmand, MD, co-founder and chief medical officer of Avenzo Therapeutics, stated in a news release.1 “We believe AVZO-103 has the potential to become a promising treatment option for patients and we are committed to rapidly advancing its clinical development.”
Enfortumab vedotin is currently approved as a single agent for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received a PD-1/PD-L1 inhibitor and platinum-containing chemotherapy, or are ineligible for cisplatin-containing chemotherapy and have previously received one or more prior lines of therapy; in combination with pembrolizumab (Keytruda) or pembrolizumab and berahyaluronidase alfa-pmph (Keytruda Qlex) for the treatment of adult patients with locally advanced or metastatic urothelial cancer; and in combination with pembrolizumab or pembrolizumab and berahyaluronidase alfa as neoadjuvant treatment and then continued after cystectomy as adjuvant treatment for the treatment of adult patients with muscle-invasive bladder cancer who are ineligible for cisplatin-containing chemotherapy.3
AVZO-103-1001 is a dose-escalation and -expansion study enrolling patients at least 18 years of age with histologically or cytologically confirmed locally advanced/metastatic urothelial cancer or other solid tumors.2 Patients need to have an ECOG performance status of 0 or 1, a life expectancy of more than 3 months, and measurable disease per RECIST 1.1 criteria.
Investigators are excluding patients with active central nervous system metastases, although those with asymptomatic and treated brain metastases are eligible if they have radiologically stable metastases for at least 4 weeks prior to the first dose of study treatment and do not require steroids. Those with suspected or confirmed leptomeningeal disease are not eligible, regardless of treatment status. Other key exclusion criteria comprise prior Stevens-Johnson syndrome/toxic epidermal necrolysis; a history of drug-induced interstitial lung disease; a history of any serious cardiovascular condition; infections requiring IV antibiotics, antivirals, or antifungals within 2 weeks prior to first dose; and a history of allogeneic stem cell or solid organ transplant.
During phase 1, investigators are assessing AVZO-103 as a single agent to determine the ADC’s safety and tolerability, determine the maximum tolerated dose, and establish the recommended phase 2 dose. Phase 2 will serve as the trial’s expansion portion, where AVZO-103 will be evaluated for antitumor activity both as a single agent and as part of combination regimens.
The primary end points of phase 1 include safety/tolerability, the incidence of dose-limiting toxicities, and to determine the MTD/RP2D. In phase 2, objective response rate (ORR) will serve as the primary end point. Secondary end points include ORR (phase 1), duration of response (phase 1 and 2), disease control rate (phase 1 and 2), progression-free survival (phase 1 and 2), overall survival (phase 1 and 2), and pharmacokinetics (phase 1).
The study will have an estimated enrollment of 355 patients. After initiating in October 2025, the study has an estimated primary completion date of March 2030.
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