Update for August 18, 2020

​Regulatory Update
 
The FDA approved viltolarsen (Viltepso, NS Pharma), on 8/12/2020, for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the DMD gene amenable to exon 53 skipping. Viltolarsen will compete with golodirsen (Vyondys 53, Sarepta), which was approved in 2019 for the treatment of DMD amenable to exon 53 skipping. Efficacy has not been established with either drug. Both will require a study to demonstrate they can improve the time to stand in DMD patients. If the trials fail to prove efficacy, the FDA may start proceedings to withdraw approval.
 
The FDA approved satralizumab (Enspryng, Genentech), on 8/17/2020, for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 or AQP4 antibody-positive. 
 
An FDA review of Mesoblast’s remestemcel-L data questioned the use of a single arm study design and felt the overall response rate used to determine efficacy was too low. However, the FDA’s Oncologic Drugs Advisory Committee voted 8-2 to recommend approval of remestemcel-L for the treatment of pediatric patients with steroid-refractory acute graft-versus-host disease (GVHD). 
 
The FDA accepted the NDA for G1 Therapeutics’ trilaciclib to preserve bone marrow and immune system function in patients being treated with chemotherapy for small cell lung cancer and set a PDUFA date of 2/15/2021.
 
The FDA accepted the BLA for Protalix BioTherapeutics’ pegunigalsidase alfa for the treatment of adult patients with Fabry disease and set a PDUFA date of 1/27/2021.
 
The FDA accepted the NDA for TG Therapeutics’ umbralisib for the treatment of follicular lymphoma and marginal zone lymphoma patients who have received at least one prior anti-CD20 based regimen. A PDUFA date of 2/15/21 has been set.
 
Announced Research Updates
 
Fulcrum reports interim data from 29 patients enrolled in the 48-week, 80 patient, Phase IIb ReDUX4 trial, where treatment with losmapimod did not reduce DUX4-driven gene expression in affected skeletal muscle biopsies at 16-weeks, compared to placebo in patients with facioscapulohumeral muscular dystrophy. Despite the lack of effect in an interim analysis, Fulcrum plans to continue the ReDUX4 trial, because in patients with the highest pre-treatment DUX4-driven gene expression, losmapimod reduced DUX4-driven gene expression more than placebo.
 
Verona announced that in a 4-week, 416 patient, Phase IIb, dose ranging trial, ensifentrine plus tiotropium increased FEV1 compared to tiotropium alone (ensifentrine dose-dependent increase of 78 mL to 124 mL) in patients with chronic obstructive pulmonary disease. 
 
Novo halted the Phase II explorer5 trial and the Phase III explorer7 and 8 trials, in March 2020, due to thrombotic events in three patients. All three trails were evaluating concizumab as prophylactic treatment in both hemophilia A and B regardless of inhibitor status. Novo restarted all three trials in August 2020 with new safety measures in place.
 
Published Research Updates
 
In the 24-week, 2,691 patient, Phase III, SAKURA 3 trial (a long -term follow-up with patients that participated in the SAKURA 1 & 2 trials), treatment with daxibotulinumtoxinA resulted in a reduction in the Investigator Global Assessment-Frown Wrinkle Severity (IGA-FWS) score, which peaked at two to four weeks where 96% of patients had an IGA-FWS score of none to mild and at 24-weeks, 32% of patients had a similar score. The most common ADR were headache (5.9%), nasopharyngitis (4.4%), injection site pain (3.9%), and injection site erythema (3.3%).