Update for October 28, 2020

Regulatory Update
 
The FDA granted the combination of ublituximab and umbralisib fast track status for the treatment of adults with chronic lymphocytic leukemia.
 
Due to travel problems from the COVID-19 pandemic, the FDA had to delay an inspection of the Spectrum plant that will be used to manufacturer eflapegrastim, which caused a delay in approval of the BLA.
 
The EMA designated leniolisib an orphan drug for the treatment of activated phosphoinositide 3-kinase delta syndrome.
 
Announced Research Updates
 
In Biogen’s 3Q20 quarterly report it was announced that opicinumab had failed to meet primary or secondary endpoints in the AFFINITY trial. Biogen discontinued development of opicinumab after failure in the AFFINITY trial.
 
Catabasis announced that in the 12-month, 131 patient, Phase III, PolarisDMD trial, treatment with edasalonexent did not improve the North Star Ambulatory Assessment (NSAA) compared to placebo in patients with Duchenne muscular dystrophy (DMD). Catabasis discontinued development of edasalonexent after failure of the PolarisDMD trial.
 
Galera announced interim data from 19 patients enrolled in a 1-year, Phase I/II trial, where treatment with avasopasem in combination with stereotactic body radiation therapy demonstrated an improvement in overall survival (OS) in patients with locally advanced pancreatic cancer.
 
Published Research Updates
 
In a 6-month, 2,472 patient, Phase III trial, cytisinicline plus behavioral support did not improve smoking cessation compared to behavioral support alone in tuberculosis patients that smoke and live in Bangladesh and Pakistan.
 
In the 12-week, 155 patient, Phase II, CAPACITY trial, treatment with praliciguat did not improve peak VO2 compared to placebo in patients with heart failure with preserved ejection fraction.
 
In the 24-week, 789 patient, Phase II, dose ranging, VITALITY-HFpEF trial, vericiguat did not improve the Kansas City Cardiomyopathy Questionnaire (KCCQ) score compared to placebo in patients with chronic HF with preserved ejection fraction.
 
In the 12-week, 12 patient, Phase II/III, TAZPOWER crossover trial, treatment with elamipretide did not improve distance traveled in a 6-minute walk test (6MWT) or improve the BTHS Symptom Assessment (BTHS-SA) score compared to placebo in patients with Barth syndrome. In an open label, 36-week extension of the TAZPOWER trial, eight patients achieved a 95.9 meter increase in their 6MWT and a 2.1 point improvement in their BTHS score. 
 
In the 15-month, 642 patient, Phase III SP12 trial, treatment with MD1003 did not improve the expanded disability status scale (EDSS) score or the timed 25-foot walk (TW25) compared to placebo in patients with progressive multiple sclerosis who were receiving disease-modifying therapies.
 
In a 19 patient, Phase II, open-label trial, treatment with imlifidase resulted in 89.5% of the transplanted patients converting positive crossmatch to negative within 24 hours with 88.9% graft survival at 6 months in highly-HLA sensitized kidney transplant candidates. 
 
In a 24-week, 120 patient, Phase III trial, treatment with berotralstat reduced hereditary angioedema (HAE) attacks to 1.65 attacks/month with 110 mg and 1.31 attacks/month with 150 mg compared to 2.35 attacks/month with placebo in patients with Type I and II Hereditary Angioedema.
 
In the 48-month, 5,734 patient, Phase III, FIDELIO-DKD trial, patients treated with finerenone had a lower incidence of a combination of kidney failure, 40% decrease in eGFR or death from renal causes compared to placebo (17.8% vs 21.1%) in patients with chronic kidney disease and type 2 diabetes.