Progenics Pharmaceuticals said it aims to submit an NDA for its radiotherapeutic drug Azedra® (iobenguane I-131) during mid 2017 on the back of positive data from a registrational, open-label Phase IIb study in patients with malignant and/or recurrent pheochromocytoma or paraganglioma.  The study, conducted through the FDA’s Special Protocol Assessment program, met its primary endpoint, by demonstrating that 25% of 68 evaluable patients experienced a 50% or greater reduction in all antihypertensive medications for at least 6 months.

The Phase IIb trial also showed that 92.2% of heavily pretreated patients who received at least one Azedra dose achieved partial response or stable disease.

Azedra has been granted Breakthrough Therapy and Orphan Drug designations in the U.S., as well as Fast Track status. “The positive data from this trial are clinically meaningful and provide compelling evidence for the use of Azedra to treat malignant and/or recurrent pheochromocytoma and paraganglioma,” said the trial’s lead investigator, Daniel Pryma, M.D., associate professor of radiology & radiation oncology and chief, Division of Nuclear Medicine & Clinical Molecular Imaging at the Perelman School of Medicine at the University of Pennsylvania. “Without any approved therapeutics in the U.S. for these rare and devastating life-threatening tumors, patients face a poor prognosis and few options. The tumor response data, in particular for the patients that received two doses, along with the adverse event profile from this trial, suggest that Azedra has the potential to be a true breakthrough in addressing these difficult-to-treat patients.”

“We intend to move quickly to complete our New Drug Application submission by mid 2017, as these topline results underscore the potential of Azedra in this ultra-orphan indication,” added Mark Baker, CEO at Progenics.

Progenics is developing a pipeline of imaging, therapeutic, and supportive care drugs for cancer. The firm’s FDA-approved drug Relistor® (methylnaltrexone bromide) is partnered with Valeant (previously Salix Pharmaceuticals, which Valeant purchased for $14.5 billion in early 2015) and was first approved in 2008 for the treatment of opioid-induced constipation. In July last year, the FDA approved the first oral formulation of Relistor for treating opioid-induced constipation in adults with chronic noncancer pain. 

Last month Progenics confirmed the start of an investigator-led Phase I study with 131I-MIP-1095 (1095) for the treatment of prostate cancer. 1095 is one of the firm’s prostate-specific membrane antigen (PSMA)-targeting small-molecule radiopharmaceuticals in development for prostate cancer indications.