NPS Pharmaceuticals, Inc. (NASDAQ:NPSP), a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, reported its results for the third quarter.
“We are excited by the continued success of the Gattex launch in the U.S.,” said Francois Nader, MD, president and chief executive officer of NPS Pharmaceuticals. “We are seeing strong demand and sales have exceeded our expectations so far with 235 patients now on Gattex. We are also pleased to raise our full-year net sales guidance to $28 to $32 million given the favorable results and trends we have seen so far. In addition, recent progress includes data from STEPS 2, which showed that patients can continue to achieve clinically meaningful reductions in parenteral support requirements or even complete independence with Gattex treatment beyond one year. With respect to our international expansion, we are quite proud of the significant progress we have made. The transfer of the Revestive EU Marketing Authorization is complete and we are preparing to initiate pricing and reimbursement discussions early in 2014. We also expect to begin launching our first named-patient programs around the end of this year.”
Dr. Nader added, “We were very pleased with the recent submission of our Biologic License Application for Natpara and we look forward to continuing to work with the FDA to complete its review. Natpara could be the first and only FDA-approved parathyroid hormone replacement therapy for hypoparathyroidism and market research supports the unmet medical need and significant global market potential.”
(Ex-U.S.) (teduglutide [rDNA origin]) for injection
(recombinant human parathyroid hormone 1-84 (rhPTH 1-84))
- Gattex sales totaled $11 million for the third quarter of 2013. Gattex is the first prescription medicine for the long-term treatment of adults with Short Bowel Syndrome (SBS) who are dependent on parenteral support.
- As of November 1, 452 prescriptions have been received and 235 patients are on therapy. As previously guided, it is expected to take an average of 90 to 120 days to dispense Gattex to patients after it is prescribed. Prior to dispensing Gattex, NPS Advantage works with patients to secure prior authorizations and reimbursement, obtain co-pay assistance, confirm colonoscopies, and coordinate nursing visits.
- Investigators presented new data from the two-year open-label STEPS 2 extension study in an award-winning poster at the Annual American College of Gastroenterology Meeting in October 2013. Long-term treatment with 30 months of Gattex resulted in clinically meaningful reductions in parenteral support with a significant percentage of patients achieving complete independence from parenteral support. The data support the long-term use of Gattex and the product’s safety profile remains consistent with the product’s label. These data have been submitted to the U.S. Food and Drug Administration (FDA) as a Supplemental New Drug Application (sNDA) to revise the Gattex label.
- The transfer of the Marketing Authorization for Revestive in the European Union was completed in October.
- NPS is finalizing its core value dossier and will begin tailoring it to the relevant countries for pricing/reimbursement negotiations. The company continues to expect to begin pricing/reimbursement discussions with authorities in selected EU countries during the first quarter of 2014.
- NPS is planning to begin early access/named-patient programs for Revestive in certain countries in late 2013 or early 2014.
- The company expects that it will begin to recognize meaningful revenues from ex-U.S. territories in the second half of 2014.
New indications and product pipeline
- The company submitted its Biologic License Application to the U.S. Food and Drug Administration (FDA) in October 2013. Natpara (recombinant human parathyroid hormone 1-84, rhPTH(1-84)) is the first bioengineered replica of human parathyroid hormone developed for hypoparathyroidism, a rare, complex endocrine disorder in which the body produces insufficient parathyroid hormone.
- The company’s Phase 3 study of Natpara was published in The Lancet Diabetes & Endocrinology. The study, known as REPLACE, confirmed the potential of Natpara as a parathyroid hormone replacement therapy for adult hypoparathyroidism. REPLACE is the largest randomized, placebo-controlled study in hypoparathyroidism to date.
- Investigators presented findings from the company’s pivotal Phase 3 study of Natpara, known as REPLACE, suggesting that it has a potential beneficial effect on bone health as shown by improvements in bone turnover markers in patients with hypoparathyroidism. Researchers also reported findings from the PARADOX burden of illness survey suggesting that patients with hypoparathyroidism have a substantial burden of illness associated with the skeleton and joints. These data were presented at the Annual Meeting of the American Society for Bone and Mineral Research in October.
- The company continues to expect to begin enrollment in its global registration study of Gattex/Revestive in pediatric patients with SBS before the end of 2013.
- NPS remains on track to initiate a Phase 2a proof-of-concept study in mid-2014 for NPSP795, a calcilytic compound with potential application in autosomal dominant hypocalcemia or ADH, an ultra-rare disorder caused by a gain-of-function mutation in the calcium-sensing receptor gene.
Financial results for the third quarter of 2013