UroGen Gets FDA Nod for Low-Grade Upper Tract Urothelial Cancer Drug

Xconomy New York — 

The FDA on Wednesday approved a drug developed by UroGen Pharma to treat low-grade upper tract urothelial cancer, which rarely spreads but often recurs and can cause kidney damage in some patients.

Urothelial cancer is the most common type of bladder cancer. Upper tract urothelial cancer (UCTC), however, affects the lining of the urinary system, arising in the lining of the kidney or in the ureter, the skinny tube that links that kidney to the bladder. In some patients the tumors block the ureter or kidney, which can cause swelling and infections that may impair kidney function.

About 6,000 to 8,000 people are diagnosed with low-grade UTUC yearly. Care consists of treating visible tumors and trying to preserve the urinary tract. The UroGen (NASDAQ: URGN) drug, mitomycin gel (Jelmyto), is the first therapy approved to treat the condition.

Investors didn’t seem impressed: UroGen’s share price closed at $21.04 Wednesday, down nearly 15 percent compared to $24.67 per share the day prior.

The treatment, a gel formulation of a chemotherapy, was designed by the Princeton, NJ-based biotech as an alternative to surgical removal of the kidney and ureter, a procedure called a nephroureterectomy.

The FDA approved the drug based on the results of a clinical trial involving 71 patients with low-grade UTUC, who had never undergone treatment or whose disease recurred, and had at least one measurable tumor attached to the inner lining of an organ. Patients received the drug once a week for six weeks. At that point, the results showed that the tumor disappeared entirely in 58 percent of those who were treated, or 41 patients. Patients for whom the tumor disappeared received the drug once per month for up to 11 more months. That response persisted in 19, or 46 percent of those patients, at the one-year mark.

Common side effects included a blockage in the ureter, pain on the side of the body, urinary tract infection, the blood in the urine, renal dysfunction, fatigue, nausea, abdominal pain, painful or difficult urination, and vomiting. More than half of the patients who received the drug experienced a ureteral obstruction, and 88 percent of those underwent a procedure to fix the blockage.

In the company’s fourth quarter and full year 2019 financial results, it said it planned to launch the drug in the second quarter.

UroGen is also advancing the drug, the first it will bring to market, as a treatment for low-grade intermediate risk non-muscle invasive bladder cancer (NMIBC). Another of its investigational drugs is being evaluated by Allergan (NASDAQ: AGN) in a Phase 2 trial, in combination with Botox as a treatment for overactive bladder. The UroGen pipeline also includes an investigational local immunotherapy for high-grade NMIBC.

Founded in Israel, UroGen is headed by president and CEO Liz Barrett, who joined the company in January 2019 from Novartis (NYSE: NVS). Barrett was previously CEO of Novartis Oncology. UroGen recently relocated its headquarters to Princeton; it maintains operations in New York and Israel.

Just ahead of its first commercial launch, however, the company is losing its chief operating officer. COO Stephen Mullennix’s last day is April 30, according to a regulatory filing. The company said Mullennix, who joined the company in February 2018 in COO and also served for a time as interim chief financial officer, is leaving to “pursue other opportunities.” UroGen didn’t provide details.

The company recently promoted its senior vice president of commercial, Jeff Bova, to chief commercial officer, and its senior vice president of clinical development, Elyse Seltzer, to chief development officer.