• Acorda Therapeutics Inc., of Ardsley, N.Y., reported that in a Phase II proof-of-concept study in people with post-stroke deficits, dalfampridine extended release was well tolerated and improved walking as measured by the 25-foot walk test. The study enrolled 83 patients with a stroke at least six months prior to treatment with chronic motor deficits. Participants received dalfampridine-ER or placebo twice daily for 14 days, followed by a one-week wash-out period, then a second 14-day period on the opposite treatment. The efficacy results showed a significant increase in walking speed for patients in the active drug group (p = 0.027). Results were presented at the American Neurological Association 2013 Annual Meeting in New Orleans.

• NPS Pharmaceuticals Inc., of Bedminster, N.J., reported additional findings from its STEPS 2 study supporting the long-term use of Gattex (teduglutide [rDNA origin]) for injection in adult patients with short bowel syndrome (SBS). In the two-year open-label extension study, long-term use of Gattex in 88 patients with SBS resulted in additional reductions in the volume and days per week of parenteral support. In addition, 10 of the 13 patients who achieved complete independence from parenteral support were those who received 30 months of Gattex in the six-month STEPS pivotal study and the 24-month STEPS 2 study. Two patients who received placebo in STEPS and 24 months of Gattex in STEPS 2 and one patient who bypassed STEPS and was enrolled directly into STEPS 2 also achieved independence from parenteral support. No new unexpected safety concerns were observed with long-term Gattex treatment. NPS submitted a supplemental new drug application to the FDA to revise the Gattex product label to include long-term data from STEPS 2. The data were presented at the American College of Gastroenterology Annual Scientific Meeting and Postgraduate Course in San Diego. (See BioWorld Today, Dec. 26, 2012.)

• Protalix Biotherapeutics Inc., of Carmiel, Israel, reported its Phase I trial of oral glucocerebrosidase, or oral GCD (PRX-112), was well tolerated in patients with Gaucher disease and active enzyme was detected in their blood. The orally available form of the plant cell-expressed enzyme, GCD, is the active substance in the company’s approved enzyme replacement therapy, Elelyso (taliglucerase alfa). In the open-label safety and pharmacokinetic study, patients received one of three doses of re-suspended carrot cells in a single oral administration during the first segment of the trial and three consecutive daily administrations during the second segment. Overall, oral GCD was found to be safe and well tolerated in all 12 patients across the three doses. Some patients who suffered from thrombocytopenia and had low platelet counts demonstrated a meaningful improvement in platelet count, so the trial has been extended to enroll and evaluate additional Gaucher patients with low platelet counts. The amended study is expected to conclude during the fourth quarter. The company said it will present additional data from the study at the 10th Annual WORLD Symposium in February 2014 in San Diego. (See BioWorld Today, May 3, 2012.)