Update for January 19, 2021

Regulatory Update 
 
The FDA accepted the NDA for finerenone for the treatment of chronic kidney disease in patients with type 2 diabetes, suggesting a PDUFA date of 7/12/2021.
 
The FDA designated ligelizumab a Breakthrough Therapy for the treatment of chronic spontaneous urticaria in patients who have an inadequate response to H1-antihistamine treatment.
 
The FDA granted padeliporfin a Fast Track designation for the treatment of low-grade and unifocal high-grade Upper Tract Urothelial Cancer. 
 
Announced Research Updates
 
Mesoblast announced that in the 30-month, 537 patient, Phase III, DREAM-HF trial, treatment with a single dose of rexlemestrocel-L resulted in a 20.6% incidence of cardiac death, heart attack or stroke compared to 30% incidence with placebo in patients with chronic heart failure with reduced left ventricular ejection fraction, that were receiving optimal oral therapy.
 
Published Research Updates
 
In a 162 patient, Phase II, open-label trial, treatment with olmutinib resulted in an objective response rate of 46.3% in patients with T790M-positive non-small cell lung cancer (NSCLC) after treatment with an epidermal growth factor receptor-tyrosine kinase inhibitor (EGFR-TKI).
 
In the 96-week, 38 patient, Phase III, RESCUE trial, the eyes treated with lenadogen nolparvovec had an improvement in best corrected visual acuity of 10 letters on the ETDRS scale compared to a 9 letter gain in the eyes that received sham injection in patients with Leber Hereditary Optic Neuropathy.
 
In the 12-week, 88 patient, dose-ranging, Phase II STARS trial, 66.7% of patients treated with gaboxadol reduced their Clinical Global Impressions-Improvement scale (CGI-I) score compared to 39.3% with placebo in patients with Angelman syndrome. A post-hoc analysis examined the CGI-I score to determine the number of patients that were very much improved or much improved and found that 32.1% of patients that received gaboxadol once daily reached this threshold compared to 7.1% of placebo patients. There was not a significant improvement compared to placebo with gaboxadol twice daily.
 
In a 48-week, 75 patient, Phase II trial, patients treated with bimagrumab had a 20.5% decrease in total body fat mass compared to a 0.5% decrease with placebo in patients with obesity and type 2 diabetes.
 
In the 52-week, 1,252 patient, Phase III, FINCH 3 trial, the percentage of patients that achieved ACR20 at 24 weeks was 81% with filgotinib 200 mg plus methotrexate and 80% with filgotinib 100 mg plus methotrexate compared to 71% with methotrexate alone in patients with moderate-to-severe rheumatoid arthritis naive to methotrexate. There was no difference between the number of patients that achieved AR20 with filgotinib 200 mg alone compared to methotrexate alone.