Weekly Update for the Week ending August 31, 2019

Regulatory Update
 
The FDA approved istradefylline (Nourianz, Kyowa Kirin) on 8/27/2019 for the treatment of off episodes n adults with Parkinson's disease already receiving levodopa/carbidopa.
 
Announced Research Updates
 
AbbVie announced the early discontinuation of the small cell lung cancer MERU study after an interim analysis found that rovalpituzumab provided no survival benefit when used as a first-line maintenance therapy. AbbVie discontinued development of rovalpituzumab after discontinuation of the MERU trial.
 
Esperion announced that in a 12-week, 179 patient, Phase II trial, treatment with bempedoic acid and ezetimibe lowered LDL-C by 40% more than ezetimibe monotherapy in patients with hypercholesterolemia and type 2 diabetes being treated with stable diabetes medication and washed out of lipid modifying therapy.
 
MyoKardia announced 24-week interim data from 12 patient enrolled in the Phase II extension of the PIONEER-HCM trial (PIONEER-OLE), suggested that 10/13patients treated with mavacamten had improved their NYHA Classification of at least one class and 9/13 were asymptomatic and improvements in biomarkers were maintained at 36 weeks.
 
Idorsia announced that in an 8-week, 430 patient, Phase II, dose ranging trial, treatment with aprocitentan lowered blood pressure 10.3/6.3, 15.0/9.9, 18.5/12.0 and 15.1/10.0 mmHg with 5, 10, 25, and 50 mg, compared to a decrease of 7.7/4.9 mmHg with placebo and 12.8/8.4 mmHg with lisinopril. Idorsia is evaluating aprocitentan in two Phase III trials, the PRECISION study in patients whose hypertension is uncontrolled by triple therapy and the INSPIRE-CKD study in patients with uncontrolled blood pressure and chronic kidney disease.
 
Published Research Updates
 
In a 272 patient, Phase I/II, dose escalation, open-label trial, treatment with olmutinib resulted in an overall objective response rate of 55.1% in patients with epidermal growth factor receptor (EGFR)-mutated non-small cell lung cancer (NSCLC) who had failed ≥ 1 previous line of EGFR-tyrosine kinase inhibitor (TKI) therapy.
 
In a 24-week, 226 patient, dose ranging, Phase II trial, patients treated with nemolizumab 30 mg sub-q every four weeks reduced the Eczema Area and Severity Index score by 68.8% compared to a 52.1% decrease with placebo in in patients receiving topical corticosteroids with moderate-to-severe atopic dermatitis, severe pruritus and inadequate control with topical treatment. The 10 mg and 90 mg doses of nemolizumab did not perform as well against placebo at 24 weeks and may not have reached statistical significance. 
 
In a 19 patient, Phase II open-label trial, treatment with 6 months of talazoparib prior to surgery resulted in a residual cancer burden pathologic complete response (RCB-0) rate of 53% and RCB-0/I of 63% in patients with germline BRCA pathogenic variant (gBRCA-positive) and operable breast cancer.
 
In an extension of a 1,046 patient, 3-dose, Phase II, trial, 116 patients received a booster dose of Sanofi’s dengue vaccine 5-6 years after completing the initial dosing series and achieved serotype titers that were similar to titers reached after the third dose of the initial series.
 
In a 17 patient Phase I, open label trial, treatment with enfortumab vedotin resulted in a 35.3% objective responserate in Japanese patients with locally advanced or metastatic urothelial cancer treated with prior chemotherapy, or ineligible for cisplatin.
 
In a 45 patient, Phase II, open label trial, pracinostatadded to hypomethylating agents did not improve outcomes in patients with myelodysplastic syndrome that did not respond to hypomethylating agent therapy.
 
In a 6-week, 580 patient, Phase III trial, patients that received a daily blonanserin transdermal patch decreased their PANSS score 5.6 points with 40 mg and 10.4 points with 80 mg compared to placebo in Japanese patients with acutely exacerbated schizophrenia.
 
In a 32-patient, Phase II, open-label trial, treatment with quizartinib resulted in complete remission in 53.8% of patients with a median duration of 16.1 weeks and overall survival of 34.1 weeks in Japanese patients with FLT3-ITD positive relapsed/refractory AML.