NPS Pharma soars as Gattex meets primary efficacy endpoint in Ph III study

Shares of US specialty drug developer NPS Pharmaceuticals (Nasdaq: NPSP) soared 22% to $9.30 in early trading in Monday on the news that its Phase III pivotal study of Gattex (teduglutide) met the primary efficacy endpoint of reducing parenteral nutrition (PN) dependence in patients with adult short bowel syndrome (SBS). The 24-week randomized, double-blind study, known as STEPS, was designed to compare the efficacy, safety and tolerability of Gattex to placebo.

The product is being co-developed with independent Swiss drugmaker Nycomed. NPS licensed ex-US Gattex rights to Nycomed in deal worth up to $185 million plus royalties (The Pharma Letter October 8, 2007).

The study reached statistical significance for the primary efficacy endpoint, defined as the percentage of patients who achieved a 20% or greater reduction in weekly PN volume at Weeks 20 and 24, compared to baseline. In an intent-to-treat analysis, 63% (27/43) of Gattex-treated patients responded versus 30% (13/43) of the placebo-treated arm (p=0.002). Patients treated with Gattex for 24 weeks also achieved significantly greater reductions in weekly PN volume versus placebo. On average, patients who received the drug experienced a 4.4 liter reduction in weekly PN volume from a pre-treatment baseline of 12.9 liters; those who received placebo experienced a 2.3 liter reduction from a pre-treatment baseline of 13.2 liters (p less than or equal to 0.001).