NPS Pharmaceuticals Inc. (NPSP) is a clinical-stage biopharmaceutical company focused on developing therapeutics for rare gastrointestinal and endocrine disorders. With two key catalysts coming up in the near-term, it is worth keeping an eye on this stock.
For readers who are new to NPS Pharma, here's what to expect in the coming months...
The company's most advanced product candidates are Teduglutide for adult short bowel syndrome and NPSP558 for hypoparathyroidism.
NPS Pharma remains on track to submit its application seeking FDA approval for Teduglutide in adult short bowel syndrome before the end of this year. Teduglutide has received orphan drug designation for the treatment of short bowel syndrome from the FDA and the European Medicines Agency.
Short bowel syndrome, or SBS for short, is a condition in which nutrients are not properly absorbed due to severe intestinal disease or surgical removal of a large portion of the small intestine, and such patients receive their nutrients and fluids intravenously, which is known as parenteral nutrition. Teduglutide is meant to grow the intestinal lining and improve dietary absorption of nutrients and fluids.
Teduglutide will be known as Gattex in the U.S. and as Revestive in Europe.
A potential first-in-class drug, Gattex is a proprietary analog of naturally-occurring human glucagon-like peptide 2, a peptide secreted primarily in the distal intestine and involved in the regeneration and repair of the intestinal epithelium.
Earlier in the year, the company reported positive results from a 24-week, pivotal phase III study, dubbed STEPS, which compared the efficacy, safety and tolerability of Gattex to placebo. The study met the primary efficacy endpoint of reducing parenteral nutrition dependence in patients with adult short bowel syndrome.
NPS licensed the ex-US Gattex rights to Nycomed, a Swiss drugmaker, in a deal worth up to $185 million in 2007. Nycomed, which is set to be acquired by Japanese pharma company Takeda, submitted a Marketing Authorization Application to the European Medicines Agency for clearance to market Teduglutide under the brand name Revestive as a once-daily subcutaneous treatment for short bowel syndrome in March of this year.
Current therapies for SBS include parenteral nutrition, and somatropin (rDNA origin) for injection, a human growth hormone marketed by Serono and glutamine in combination with somatropin (rDNA origin) for injection. According to NPS Pharma there are 10,000 to 15,000 short bowel syndrome patients in the U.S. who are parenteral nutrition-dependent, the direct cost of which can exceed $100,000 annually per patient. If approved, it is estimated that the U.S. sales of Gattex could range between $150 million and $250 million in the indication of SBS.
Next up is NPSP558 in the treatment of hypoparathyroidism. This product candidate is being evaluated in a phase III registration study, known as REPLACE.
Hypoparathyroidism is a rare disorder in which the body produces insufficient levels of parathyroid hormone, the principal regulator of calcium and phosphorus, leading to muscular and neurological symptoms, as well as bone impairments. There is no approved treatment for hypoparathyroidism and is currently managed with large doses of oral calcium and vitamin D supplementation. It is estimated that roughly 60,000 to 65,000 patients suffer from hypoparathyroidism in the U.S.
NPSP558 mimics the action of natural parathyroid hormone and is believed to have the potential to treat hypoparathyroidism and offer a more physiological treatment outcome than what is available with existing treatments.
The company initiated the REPLACE study in December 2008 with a primary objective of demonstrating that once-daily subcutaneous dosing with NPSP558 over a period of 24 weeks is a safe and effective treatment for patients with hypoparathyroidism. The company completed randomization of 135 patients in the study in March of this year. Top line results from the REPLACE study are expected next quarter (4Q).
NPSP558 has been granted orphan drug status for the treatment of hypoparathyroidism and has the potential to be the first hormone replacement therapy for this rare disorder, if approved.
The company has also evaluated NPSP558 for osteoporosis under the brand name PREOS, and this product candidate had been at the FDA altar in 2005. But PREOS couldn't get past the finish line as the FDA expressed concern regarding hypercalcemia associated with the proposed daily dose of PREOS and sought additional clinical information. NPS intends to continue development of PREOS for osteoporosis in the U.S. market only if it secures a partner.
In addition to products in development, NPS Pharma has a royalty-based portfolio of marketed products that includes agreements with Amgen Inc. (AMGN), Kyowa Kirin and Nycomed.
The marketed products include Sensipar for the treatment of secondary hyperparathyroidism in chronic kidney disease patients on dialysis and for the treatment of elevated calcium levels (hypercalcemia) in patients with parathyroid carcinoma, whose worldwide rights are licensed to Amgen , Regpara - marketed in Asia for treating secondary hyperparathyroidism, licensed to Kyowa Kirin and Preotact, marketed in Europe for the treatment of osteoporosis in post-menopausal women at high risk of fractures, whose ex-US rights are licensed to Nycomed.
Now, a quick look at the balance sheet...
From inception in 1986 through June 30, 2011, the company has incurred cumulative losses of about $969.5 million. Revenue is derived from royalties, license fees, milestone payments and product sales from its licensees and collaborators.
In the recent second quarter, the company's net loss narrowed to $6.13 million or $0.07 per share from $6.30 million or $0.11 per share in the year-ago quarter. Total revenues for the quarter were up 13 percent to $27 million. NPS Pharma has $202 million in cash and expects cash burn for this year to range between $85 million and $100 million.
Shares of NPS Pharma have a 52-week price range of $5.80 to $10.75. The stock closed Wednesday's trading at $7.80 on a volume of 2 million shares.
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