Voyager Therapeutics announced an update to its VY-AADC clinical program for Parkinson’s disease. In December 2018, the company held a Type B meeting with the U.S. FDA to discuss the overall development program for VY-AADC. Based on the meeting discussion and subsequent written feedback from the FDA, Voyager plans to submit a revised trial protocol that will include an increase in the target number of patients in the RESTORE-1 Phase 2 trial, resulting in 75 to 100 total patients in the trial, and to conduct a staggered-parallel Phase 3 trial of similar size and design to RESTORE-1. These updates incorporate guidance from the FDA from the Type B meeting to conduct two adequate and well-controlled clinical trials for a large patient population such as Parkinson’s disease. “Our recent meeting with the FDA was informative and helps to clarify the expected regulatory pathway for VY-AADC,” said Andre Turenne, president and CEO of Voyager Therapeutics. “We look forward to continuing to engage with the FDA and other regulators as we advance our clinical development program and our work to bring VY-AADC to patients in need.” The RESTORE-1 Phase 2 trial is currently enrolling patients who have been diagnosed with Parkinson’s disease for at least four years, are not responding adequately to oral medications, and have at least three hours of OFF time during the day as measured by a validated self-reported patient diary. The primary efficacy endpoint of RESTORE-1 is ON time without troublesome dyskinesia, or good ON time, as measured by a validated self-reported patient diary at 12 months. Voyager expects RESTORE-1 will take approximately 15 to 21 months to enroll. Voyager plans to begin enrolling patients in RESTORE-2 in both active Phase 2 sites and additional sites globally in the first half of 2020. Voyager anticipates that, if positive, results from RESTORE-1 and RESTORE-2 could potentially form the basis for submission of a biologics license application to the FDA for VY-AADC for the treatment of Parkinson’s disease.
Monday, January 7, 2019
Regeneron sees 2019 Sanofi collaboration revenue $510M-$560M
Sees 2019: Effective tax rate 14%-16%; CapEx $410M-$490M.
ProQR Therapeutics announces design on ILLUMINATE trial
ProQR Therapeutics announced that it has reached agreement with the FDA on the design of a Phase 2/3 pivotal trial for QR-110, now renamed to sepofarsen, in patients with Leber’s congenital amaurosis 10, or LCA10 due to the p.Cys998X mutation in the CEP290 gene. LCA is the leading genetic cause of childhood blindness. The trial is expected to start in the first half of 2019. We anticipate top line results for base-case of 30 trial participants around year-end 2020. ILLUMINATE will be a randomized, double-masked, sham-controlled trial initially enrolling 30 adults and children assigned equally to three parallel arms with 10 participants in each arm. The trial incorporates adaptive sample size re-estimation and is designed with potential to serve as the sole registration trial for the program. Participants will receive a dose of sepofarsen or a sham-injection at the start of the trial, at three months and then every six months. The primary efficacy endpoint for the trial will be change in visual acuity from baseline in the treated arm compared to sham-treated control arm at the 12 month time point. Sham-treated participants may be offered cross-over to active treatment after 12 months, and all participants will continue to receive treatment for a total of 24 months after which they may be offered participation in an open label extension trial. Treatment of the second eye will be adaptively incorporated into the trial.
Cara Therapeutics completes enrollment of KALM-1 Phase 3 trial of Korsuva
Cara Therapeutics announced completion of enrollment in the KALM-1 Phase 3 trial of KORSUVA Injection in hemodialysis patients with moderate-to-severe chronic kidney disease-associated pruritus. More than 350 hemodialysis patients with CKD-aP have now been randomized across approximately 60 clinical sites in the United States. KALM-1 is a multicenter, randomized, double-blind, placebo-controlled 12-week treatment trial in the U.S. with a 52-week open label extension phase that is designed to evaluate the safety and efficacy of 0.5 mcg/kg KORSUVA CR845/difelikefalin injection in 350 hemodialysis patients with moderate-to-severe pruritus. The primary efficacy endpoint is the proportion of patients achieving at least a 3-point improvement from baseline in the weekly mean of the daily 24-hour Worst Itch Numeric Rating Scale score at week 12. In a completed Phase 2 trial, the proportion of patients with an improvement from baseline in the weekly mean Worst Itch NRS score of greater than or equal to3 points at week 8 was statistically significantly higher in the CR845/difelikefalin 0.5 mcg/kg group compared to the placebo group . Secondary endpoints include assessment of itch-related quality of life changes measured using the validated self-assessment 5-D itch and Skindex-10 scales, as well as the proportion of patients achieving greater than 4-point improvement from baseline in weekly mean of the daily 24-hour Worst Itch NRS score at week 12.
Arrowhead files for regulatory clearance to begin ARO-APOC3 Phase 1 study
Arrowhead Pharmaceuticals announced that it has filed an application for approval to begin a Phase 1 clinical trial of ARO-APOC3, an RNAi-based investigational medicine targeting Apolipoprotein C-III being developed for the treatment of hypertriglyceridemia. Pending approval, Arrowhead intends to proceed with AROAPOC31001, a Phase 1 single and multiple dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of ARO-APOC3 in adult healthy volunteers, hypertriglyceridemic patients, and patients with familial chylomicronemia syndrome. The study is designed to enroll up to 63 subjects.
Invitae sees FY19 revenue of more than $220M, consensus $200M
Looking ahead to 2019, Invitae anticipates test volume of more than 500,000 samples generating more than $220M in revenue for the year.
Allergan to establish R&D presence in Cambridge, Massachusetts
Allergan announced it will establish an R&D presence in the Kendall Square section of Cambridge, Massachusetts, a hub of science, innovation and business opportunities. The site will be led by Don Frail, PhD, Senior Vice President of Research and External Scientific Innovation and Non-Clinical and Translational Sciences.
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