Ionis and Akcea Present New Data from ATTR Amyloidosis Program

Ionis Pharmaceuticals and its affiliate, Akcea Therapeutics, announced Phase III data showing that inotersen-treated patients with hereditary ATTR (hATTR) amyloidosis who were treated for up to 27 months in the NEURO-TTR and open-label extension (OLE) studies continued to demonstrate sustained benefit in measures of quality of life and neuropathy. The NEURO-TTR study was a Phase III randomized (2:1), double-blind, placebo-controlled, international study in 172 patients with polyneuropathy due to hATTR. The 15-month study measured the effects of inotersen on neurological dysfunction and on quality-of-life by measuring the change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total score. The NEURO-TTR OLE is an ongoing study for patients who completed the NEURO-TTR study and is intended to evaluate the long-term efficacy and safety profile of inotersen. Significant benefit was observed in inotersen-treated patients with cardiac disease at baseline in both primary endpoints (Norfolk QoL-DN, p=0.036 and mNIS+7, p<0.001) and in the way they felt and functioned in the SF-36 Health Survey endpoint (p=0.025) at 15 months, compared to placebo.

Ablynx Announces Results From Systemic Lupus Erythematosus Study

Ablynx announced that the Phase II dose-ranging study of vobarilizumab, the Company’s anti-IL-6R Nanobody, did not meet the primary endpoint of dose response based on the modified BILAG-based combined lupus assessment (mBICLA) at Week 24. This multi-center, randomized, double-blind, placebo-controlled, dose-range finding Phase II study enrolled 312 patients with moderate to severe, active seropositive SLE across the U.S., Europe, South America and Asia. The study enrolled patients across five treatment arms (four dose regimens of vobarilizumab and placebo). Safety findings through Week 58 were favourable for vobarilizumab. Treatment-related serious adverse events were reported in 2.0 percent of all vobarilizumab-treated patients compared to 6.5 percent in the placebo group. The percentage of patients experiencing a serious infection was also lower in the vobarilizumab arms compared to the placebo arm (2.8 percent versus 6.5 percent).

Allecra Therapeutics Announces Positive Results from Phase II Study

Allecra Therapeutics announced positive top-line results from the Phase II study of its lead antibiotic candidate, AAI101. The Phase II CACTUS study (Randomized, Double-Blind, Multi-Center Study of Cefepime/AAI101 in hospitalized adults with Complicated UTIs) met all study objectives. AAI101 was given intravenously to patients in combination with cefepime for the treatment of cUTI including acute pyelonephritis (AP). The study was designed to determine the optimal dose of cefepime/AAI101 to be taken forward into future Phase III studies. 45 patients were randomized 2:1 into two cohorts, each with a separate control. Patients randomized into Cohort 1 received either 500mg of AAI101 combined with 1g of cefepime (n=15), or 1g of cefepime monotherapy (n=7). Patients randomized into Cohort 2 received 750mg of AAI101 combined with 2g cefepime (n=15), or 2g of cefepime monotherapy (n=8). Dosing was conducted intravenously three times daily for seven to ten days.

Novartis Reports Data on Secondary Progressive MS Study

Novartis announced the full results from the Phase III EXPAND study of oral, once-daily siponimod (BAF312) in secondary progressive multiple sclerosis (SPMS). The EXPAND study is a randomized, double-blind, placebo-controlled Phase III study, comparing the efficacy and safety of siponimod versus placebo in people with SPMS. It is the largest randomized, controlled study in SPMS to date. The results show significant reductions in the risk of three- (primary endpoint) and six-month confirmed disability progression with siponimod versus placebo and favorable outcomes in other relevant measures of MS disease activity. Full data from EXPAND show that siponimod reduced the risk of three-month confirmed disability progression by a statistically significant 21 percent versus placebo (p=0.013; primary endpoint); efficacy was consistent across many pre-defined sub groups. Other clinically relevant endpoint data show that siponimod reduced the risk of six-month confirmed disability progression by 26 percent (p=0.0058) and slowed the rate of brain volume loss by 23 percent (relative difference; mean across 12 and 24 months, p=0.0002), when compared to placebo.