Weekly Update for the Week Ending September 7, 2019

Regulatory Update
 
The FDA accepted the BLA for inebilizumab in September 2019 for the treatment of patients with neuromyelitis optica spectrum disorder.
 
The FDA accepted the NDA for voxelotor for the treatment of sickle cell disease in September 2019 and set a PDUFA date of 2/26/2020.
 
The FDA designated capmatinib a Breakthrough Therapy for the treatment of metastatic MET exon14 skipping-mutated non-smallcell lung cancer.
 
Announced Research Updates
 
Ardelyx announced that in the 4-week, 236 patient Phase III, AMPLIFY trial, treatment with tenapanor plus a phosphate binder decreased serum phosphorus 0.84 mg/dL compared to a 0.19 mg/dL decrease with only the phosphate binder in dialysis patients with hyperphosphatemia not controlled by a phosphate binder regimen.
 
Concertannouncedthat in a 24-week,149 patient, Phase II trial, treatment with CTP-543 resulted in achievement of a 50% reduction in the SALT score in 58% of patients with 12 mg, 47% with 8 mg and 21% with 4 mg compared to 9% with placebo in patients with moderate to severe alopecia areata. The 12 mg and 8 mg doses were significantly different from placebo, while the 4 mg dose did not achieve a significant difference.
 
The Medicines Company announced that in the 18-month, 1,617 patient, Phase III, ORION-11 trial, the placebo adjusted reduction in LDL with inclisiran was 54% in patients with atherosclerotic cardiovascular disease or risk equivalents and elevated LDL despite maximum tolerated dose of statin with or without ezetimibe.
 
AstraZeneca announced that in the 955 patient, Phase III, open-label, NEPTUNE trial, durvalumab plus tremelimumab did not improve overall survival compared to standard-of-care chemotherapy in patients with metastatic non-small cell lung cancer.
 
Published Research Updates
 
In the 12-month, 1,346 patient, Phase III SUNBEAM trial, treatment with oral ozanimod resulted in an annualised relapse rate of 0.18 with 1 mg and 0.24 with 0.5 mg compared to 0.35 with weekly intramuscular interferon beta-1a 30 mcg in patients with relapsing multiple sclerosis. 
 
In a 15-day, 89 patient, Phase II, trial, a daily oral dose of zuranolone decreased the Hamilton Rating Scale for Depression (HAMD-17) score by 17.4 points compared to a 10.7 point decrease with placebo in patients with major depressive disorder.