Progenics: The FDA Accepted the NDA for Its Cancer Product Azedra® and Granted It Priority Review

Progenics Pharmaceuticals (PGNX) has good news today. The U.S. Food and Drug Administration (FDA) has accepted for review the firm’s New Drug Application (NDA) for its product Azedra® for recurrent and/or unresectable pheochromocytoma and paraganglioma — rare neuroendocrine tumors. The FDA has also granted Progenics’ request for Priority Review and has set an action date of April 30, 2018, under the Prescription Drug User Fee Act (PDUFA).

The NDA is supported by data from a pivotal phase 2b open-label, multi-center trial that was conducted under a Special Protocol Assessment (SPA) with the FDA. The trial met the primary endpoint evaluating the proportion of pheochromocytoma and paraganglioma patients who achieved a 50% or greater reduction of all antihypertensive medication for at least six months, and showed favorable results from a key secondary endpoint evaluating the proportion of patients with overall tumor response as measured by Response Evaluation Criteria in Solid Tumors (RECIST).

Azedra was also shown to be safe and generally well tolerated.

The Drug

AZEDRA (iobenguane I 131) is a high-specific-activity radiotherapeutic product candidate in development as a treatment for malignant, recurrent, or unresectable pheochromocytoma and paraganglioma, which are rare neuroendocrine tumors of neural crest origin.

The product is a substrate for norepinephrine reuptake transporter which is highly expressed on the cell surface of neuroendocrine tumors.  Azedra has been granted Orphan Drug designation, Fast Track status, and Breakthrough Therapy designation in the U.S.  Under an SPA agreement with the FDA, a phase 2b pivotal study has been completed in patients with malignant, recurrent, or unresectable pheochromocytoma and paraganglioma.

There are currently no FDA-approved therapies for the treatment of this ultra-rare disease.

Pheochromocytoma and Paraganglioma 

Pheochromocytoma and paraganglioma are rare neuroendocrine tumors that arise from cells of the autonomic nervous system. Pheochromocytoma forms in the adrenal medulla, whereas paragangliomas form outside the adrenal gland.

Standard treatment options for these tumors include surgery, palliative therapy and symptom management.

Pheochromocytoma and paraganglioma tumors frequently secrete high levels of hormones that can lead to life-threatening hypertension, heart failure, and stroke in these patients.

Malignant and recurrent pheochromocytoma and paraganglioma may result in unresectable disease with a poor prognosis, representing a significant management challenge with very limited treatment options and no approved anti-tumor therapies.

Progenics

Progenics Pharmaceuticalsof Tarrytown, NY is a biopharmaceutical company focusing on the development and commercialization of therapeutic products to treat the unmet medical needs of patients with debilitating conditions and life-threatening diseases.

 Pipeline

– Therapeutic agents designed to precisely target cancer (Azedra® and 1095),

– PSMA-targeted imaging agents for prostate cancer (1404 and PyL™), and

– Imaging Analysis Tools.

Progenics’ marketed product, Relistor® (methylnaltrexone bromide) for opioid-induced constipation is partnered with Valeant Pharmaceuticals International, Inc.

Progenics’ Stock

Symbol:                        PGNX

Stock Exchange:        NASDAQ

Stock Price:                $6.55 UP $0.70 on Today’s News

Market Cap:                $461.67

Prohost Observations

Progenics is a biotech firm that managed to put a product on the market. Today’s news might be the beginning of the evolution of this firm towards bringing revenues that make it self-sufficient and earning growing firm. We will follow up on this firm.

We will not forget that April 30, 2018, is Azedras action date under the Prescription Drug User Fee Act (PDUFA).

Do Not miss Prohost Letter “The Year in Review”, coming soon.

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