Update for July 14, 2020

Regulatory Update
 
The FDA approved the oral combination of decitabine and cedazuridine (Inqovi, Astex Pharmaceuticals), on 7/7/2020, for treatment of myelodysplastic syndrome or chronic myelomonocytic leukemia.
 
The EU approved J&J’s Ad26.ZEBOV (Zabdeno) and MVA-BN-Filo (Mvabea), a two dose vaccine regimen for the prevention of Ebola virus infection.
 
The FDA designated Concert Pharmaceuticals’ CTP-543 a Breakthrough Therapy for the treatment of moderate-to-severe alopecia areata.
 
The FDA accepted the BLA for AstraZeneca/Leo Pharma’s tralokinumab for treatment of moderate-to-severe atopic dermatitis.
 
Biogen filed a BLA for aducanumab for the treatment of Alzheimer’s Disease.
 
ChemoCentryx submitted an NDA for avacopan for the treatment of patients with ANCA-associated vasculitis.
 
Announced Research Updates
 
Idorsia announced that in a 3-month, 924 patient, Phase III trial, treatment with daridorexant 25 mg improved sleep maintenance and total sleep time compared to placebo in patients with insomnia. The 25 mg dose did not improve sleep onset and daytime functioning compared to placebo and the 10 mg dose did not improve any of the four measures.
 
Bayer announced that in the 48-month, 5,734 patient, Phase III, FIDELIO-DKD trial, patients treated with finerenone had a lower incidence of kidney failure and renal death compare to placebo in patients with chronic kidney disease and type 2 diabetes. Kidney failure was defined as a sustained decrease of estimated glomerular filtration rate (eGFR) greater than or equal to 40 percent from baseline over a period of at least four weeks.
 
Atox Bio announced that in the 28-day, Phase III, ACCUTE trial, involving 290 patients with severe necrotizing soft tissue infection, treatment with reltecimod did not improve a composite of morbidity, no more than three debridements, no further amputations, and resolution of organ dysfunction compared to placebo (48.6% vs 39.9%) in a modified Intent-to-Treat responder analysis (primary endpoint), but demonstrated a benefit in a per protocol analysis (54.3% vs 40.3%) after 17 patients were excluded.
 
Published Research Updates
 
In a 15-month, 891 patient, Phase III trial, treatment with hydromethylthionine did not improve the Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) or the Alzheimer's Disease Co-operative Study-Activities of Daily Living Inventory (ADCS-ADL) scores compared to placebo in patients with mild to moderate Alzheimer's disease. Most patients were receiving acetylcholinesterase inhibitors, memantine or both. A separate sub-group analysis of hydromethylthionine monotherapy suggested a benefit on cognitive scores.
 
In a 16-week, 143 patient, Phase III trial, treatment with topical agents plus nemolizumab decreased the pruritus VAS score by 42.8% compared to a 21.4% decrease with only topical agents in Japanese patients with atopic dermatitis and moderate-to-severe pruritus and an inadequate response to topical agents. 
 
In a 12-month, 50 patient, open-label, Phase II trial, treatment with axalimogene resulted in overall survival of 38% in patients with persistent or recurrent metastatic carcinoma of the cervix.
 
In a 95 patient, Phase II trial, treatment with margetuximab plus pembrolizumab resulted in an overall response rate of 18.48% in patients with metastatic HER2-positive gastroesophageal adenocarcinoma previously treated with trastuzumab and chemotherapy.
 
In the 48-week, 374 patient, Phase II ARPEGGIO trial, treatment with laquinimod did not differ from placebo in the percentage brain volume change compared to placebo in primary progressive MS patients.