NPS Pharmaceuticals, a specialty pharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, reported its results for the fourth quarter and full year 2009. The company also reported that based on the current pre-randomization dropout rate, there are a sufficient number of patients enrolled, scheduled or identified to achieve the 86-patient randomization target in STEPS, a Phase 3 registration study of GATTEX (teduglutide) in parenteral nutrition (PN) dependent short bowel syndrome (SBS).
Financial Highlights
NPS reported a net loss of $2.0 million or $0.04 per diluted share for the fourth quarter of 2009, compared to a net loss of $8.5 million or $0.18 per diluted share for the fourth quarter of 2008. For the full year, NPS reported a net loss of $17.9 million or $0.37 per diluted share for 2009, versus a net loss of $31.7 million or $0.67 per diluted share for 2008.
The company’s cash, cash equivalents and short- and long-term investments totaled $74.9 million at December 31, 2009 versus $106.1 million at December 31, 2008. On March 1, 2010, the company received an additional $38.4 million of capital from the sale of certain of its REGPARA royalty rights. The company’s net cash burn was $42.1 million for 2009 versus revised guidance of $43 to $50 million. The company’s reported cash burn is defined as the net change in cash, cash equivalents, and marketable investment securities, excluding changes in the fair value of auction-rate security (ARS) investments (approximately $2 million) and proceeds from external financing activities (approximately $9 million). Cash burn is a non-GAAP financial measure that may be considered in addition to results prepared in accordance with U.S. generally accepted accounting principles (GAAP). This non-GAAP measure should not be considered a substitute for, or superior to, GAAP results. NPS believes that cash burn is relevant and useful information for the company and its investors as it provides a meaningful way of determining net cash used for operations of the company.
Product Pipeline Update
The company’s activities in 2009 were largely focused on advancing Phase 3 registration programs for GATTEX in SBS and NPSP558 in hypoparathyroidism, with key highlights summarized below:
GATTEX (teduglutide)
* The company believes that a sufficient number of patients are currently enrolled, scheduled or identified to achieve the 86-patient randomization target for STEPS. The study’s enrollment target is estimated based on the percentage of patients who have discontinued prior to randomization. While the company believes that a sufficient number of patients have been identified, it has elected to continue enrolling patients in STEPS until the study has been fully randomized to ensure the full and timely completion of the study. Once the randomization target is achieved, any remaining patients who complete the stabilization phase will be enrolled in STEPS 2, an open label study in which all participants will receive up to 24 months of GATTEX therapy. STEPS is an international, double-blind, placebo controlled safety and efficacy study to confirm previously reported data that showed GATTEX was well tolerated and had a beneficial impact on reducing PN dependence in SBS patients. The company believes positive results from STEPS will enable it to seek U.S. marketing approval for GATTEX for adult patients with PN-dependent SBS. NPS is advancing STEPS and STEPS 2 based on the collaboration agreement entered into with Nycomed, which provides for the sharing of external clinical costs.
* Clinical investigators presented new data from the first 24-week, placebo-controlled Phase 3 clinical trial of GATTEX at the June 2009 Digestive Disease Week or DDW conference and the October 2009 American College of Gastroenterology or ACG Annual Scientific Meeting and Postgraduate Course. Data continue to underscore the potential efficacy and safety of GATTEX in patients with SBS.
* NPS has executed long-term supply agreements for the commercial manufacture and supply of active pharmaceutical ingredient, finished injectable product, and other commercial supply-chain services.
* NPS held a pre-investigational new drug application (pre-IND) meeting with the U.S. Food and Drug Administration (FDA) to discuss teduglutide in chemotherapy-induced gastrointestinal mucositis, and the company is currently defining its clinical development strategy for this indication.
* Conduct and analysis of preclinical studies supporting the development of teduglutide in pediatric SBS are substantially complete.
NPSP558 (recombinant parathyroid hormone 1-84 [rDNA origin] injection)
* NPS has implemented a number of proactive steps to accelerate enrollment in REPLACE, including transitioning certain activities to a new service provider, adding new clinical sites, and expanding the base of eligible patients to better match the target patient population. The changes are beginning to take hold with recent enrollment rates accelerating. The company now expects to achieve its enrollment target for REPLACE in the second half, rather than the middle of, 2010. REPLACE is an international, double-blind, placebo-controlled Phase 3 registration study evaluating NPSP558 for the treatment of hypoparathyroidism in adults.
* Results from an investigator-initiated Phase 2 open-label proof-of-concept study demonstrated that NPSP558 can potentially be used effectively and safely as a therapeutic agent in hypoparathyroidism. Thirty hypoparathyroidism subjects participated in the study and were treated with NPSP558 at a dose of 100 mcg every-other-day by subcutaneous injection for 24 months. Investigators were able to maintain serum calcium within the normal range while significantly reducing the intake of supplemental calcium and active vitamin D.
* NPS has executed long-term supply agreements for the commercial manufacture and supply of active pharmaceutical ingredient, finished injectable product, and other commercial supply-chain services.
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