Progenics Pharmaceuticals to Initiate a Basket Trial

Progenics Pharmaceuticals (NASDAQ:PGNX) has announced it has reached a decision with the US Food and Drug Administration (FDA) on plans to pursue a tissue agnostic indication for an expanded label for AZEDRA to treat patients with unresectable or metastatic neuroendocrine tumors (NETs) which are MIBG avid.

As quoted in the press release:

Following a Type B meeting with the FDA, the Company plans to conduct a basket study that will evaluate AZEDRA in patients with NETs that are MIBG avid, including gastroenteropancreatic neuroendocrine tumors (GEP-NETs) and other NETs, with a dosing regimen that potentially enables outpatient administration. AZEDRA is the first and only approved therapy in the U.S. for the treatment of adult and pediatric patients 12 years and older with iobenguane (MIBG) scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy.

NETs are a group of rare tumors of neural crest origin, most commonly found in the gastrointestinal or respiratory tracts, although they may also be found in the central nervous system, thyroid gland, skin, breast, colon, and urogenital system, including prostate cancer, among other locations. NETs overexpress the norepinephrine reuptake transporter on their cell surface. MIBG is a known substrate for the transporter and, therefore, MIBG avidity can be used to enrich for patients who are most likely to respond to AZEDRA therapy. NETs are considered rare tumors, although the incidence has continued to increase in the last 10-15 years, with approximately 12,000 patients being diagnosed in the U.S. each year.

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