Genetic Engineering & Biotechnology News

MAY1 2015

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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Genetic Engineering & Biotechnology News | | MAY 1, 2015 | 35 TRANSLATIONAL MEDICINE Catecholamine-Resistant Hypotension > La Jolla Pharmaceutical initiated its ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 trial, a Phase III clini- cal trial of LJPC-501 for the treatment of cat- echolamine-resistant hypotension (CRH). The trial is a multicenter, randomized, dou- ble-blind, placebo-controlled study of LJPC- 501 in patients with CRH. The trial is designed to enroll approximately 315 patients. Patients will be randomized 1:1 to receive either LJPC- 501 plus standard-of-care vasopressors, or placebo plus standard-of-care vasopressors. Randomized patients will receive their as- signed treatment via continuous intravenous infusion for up to seven days. The primar y efficacy endpoint is to compare the change in mean arterial pres- sure between the two groups in the trial. Secondary endpoints include comparison of changes in Sequential Organ Failure As- sessment scores and the safety and toler- ability of LJPC-501 in patients with CRH. Cystic Fibrosis > Vertex Pharmaceuticals presented data from a 12-week Phase II study evalu- ating VX-661 in combination with ivacaftor in 39 people diagnosed with cystic fbrosis and known to be at least 18 years of age and possessed of two copies of the F508del mutation. The study evaluated two doses of VX-661 (100 mg once daily or 50 mg ev- ery 12 hours) in combination with ivacaftor (150 mg every 12 hours). The primary endpoint of the study was safety. The study showed that the combination regimen was generally well tolerated, and all patients completed 12 weeks of treatment. The most common adverse events were pulmonary exacer- bation and cough. Secondary endpoints evaluated the ef- fect of the combination on lung function (percent predicted forced expiratory volume in one second; ppFEV1). The mean within- group absolute improvement from baseline in ppFEV1 for those who received 100 mg of VX-661 in combination with ivacaftor (n = 15) was 4.4 (p = .009) and 3.0 (p = .026) percent- age points at week 4 and through 12 weeks of treatment, respectively. The company said the study showed a rapid improvement in lung function within four weeks of treat- ment, and after patients completed treat- ment, lung function returned to baseline. Psoriasis > Celgene presented results from its ongoing Phase III LIBERATE trial evaluat- ing Otezla® (apremilast), its oral, selec- tive inhibitor of phosphodiesterase 4 , in patients with moderate to severe plaque psoriasis. The study evaluated the clinical efcacy and safety of either oral Otezla 30 mg twice daily or weekly subcutaneous etanercept 50 mg compared with placebo at week 16 in 250 patients. It also exam- ined the relative safety of a switch from etanercept to Otezla after week 16. At week 16, patients receiving Otezla 30 mg twice daily demonstrated statistically sig- nifcant and clinically meaningful improve- ment when compared with placebo, as mea- sured by the Psoriasis Area and Severity Index (PASI). A 75% reduction in PASI (PASI 75) was observed in 40% (n = 33/83) of the patients given Otezla and 12% (n = 10/84) of the pa- tients given placebo (p < .0001). At week 16, statistical signifcance was also achieved for patients receiving weekly injections of etan- ercept 50 mg when compared with placebo [48% (n = 40/83) with etanercept, 12% (n = 10/84) with placebo (p < .0001]. Among patients randomized to Otezla at baseline, more patients achieved a PASI 75 response at week 32 than at week 16 [53% (n = 44/83) vs. 40% (n = 33/83), respectively]. Among patients who switched from etan- ercept to Otezla at week 16, more patients achieved a PASI 75 response at week 32 than at week 16 [61% (n = 51/83) vs. 48% (n = 40/83), respectively]. Adverse events reported in at least 5% of patients taking Otezla in the LIBER- ATE study were diarrhea, nausea, vomiting, and headache (including tension headache). n Clinical Trials

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