Weekly Update for the Week Ending October 7, 2019

Regulatory Update
 
The FDA approved trifarotene cream (Aklief, Galderma) on October 4 for the treatment of acne vulgaris.
 
The FDA has granted Fast Track status to imetelstat for the treatment of adult patients with relapsed or refractory myelofibrosis, including whose disease has relapsed after or is refractory to janus kinase inhibitor treatment. 
 
Announced Research Updates
 
MacroGenics announced that in a 92 patient, Phase II trial, treatment with margetuximab plus pembrolizumab resulted in an overall response rate of 21.7% and progression-free survival of 2.7 months in patients with metastatic HER2-positive gastroesophageal adenocarcinoma previously treated with trastuzumab and chemotherapy.
 
BeiGene announced interim data from a 104 patient, Phase II, open label trial, where treatment with tislelizumab resulted in an overall response rate of 23.1% in Chinese and South Korean patients with with PD-L1+ locally advanced or metastatic urothelial carcinoma previously treated with a platinum-containing chemotherapy regimen.
 
Resverlogix announced that in the Phase III, BETonMACE trial, apabetalone added to standard of care did not reduce major adverse cardiovascular events (cardiovascular death, non-fatal myocardial infarction or stroke) compared to placebo in high-risk patients with type 2 diabetes, recent acute coronary syndrome, and low HDL cholesterol.
 
Amgen announced interim data from 12 of 76 patients enrolled in a Phase I, open-label, dose-ranging trial, received the highest dose of AMG 51 (960 mg) with 1 achieving a partial response and 10 achieving stable disease in previously treated patients with KRAS G12C-mutated colorectal cancer.
 
Elevar announced that in the 460 patient, Phase III ANGEL study, treatment with rivoceranib plus best supportive care did not improve overall survival compared to plus best supportive care only (5.8 vs 5.1 months) in the overall population of patients with gastric or gastroesophageal junction cancer who have failed at least 2 prior lines of therapy. Overall survival was improved in 185 patients that had received 3 or more courses of chemotherapy before treatment with rivoceranib compared to placebo (6.43 vs 4.73  months).
 
Shionogi  announced that in the 14-day, 452 patient, Phase III, APEKS-NP trial, the mortality rate with cefiderocol was 12.4% compared to 11.6% with meropenem, which met non-inferiority criteria, in patients with nosocomial pneumonia.
 
Rebiotix announced results from a 24-month, 95 patient, Phase II open-label, extension of the PUNCH CD trial, where 91% of patients that did not develop a CDI at 8 weeks remained CDI-free at 24 months post-RBX2660 treatment.
 
Published Research Updates
 
In a 160 patient, Phase I/Ib, open-label trial, umbralisib plus ublituximab resulted in an overall response rate of 46% with 17% achieving a complete response in patients with chronic lymphocytic lymphoma or non-Hodgkin lymphoma.
 
In the 48-week, 103 patient, Phase IIb PRAISE trial, patients treated with pamrevlumab had a reduction of 2.9% in the percentage of predicted FVC compared to 7.2% decrease with placebo in patients with mild-to-moderate idiopathic pulmonary fibrosis. Disease progression was seen in 10% of patients treated with pamrevlumab compared to 31.4% of patients in the placebo group.
 
In a 12-week, 267 patient, Phase IIb trial, 43.8% of patients treated with abrocitinib 200 mg and 29.6% that received 100 mg achieved an Investigator Global Assessment (IGA) score of clear or almost clear skin and had a 2 point or > improvement in their IGA score compared to 5.8% with placebo in patients with moderate to severe atopic dermatitis.
 
In a 12-week, 382 patient, Phase IIb trial, patients treated with ligelizumab achieved a weekly hives-severity score of 0 in 30% of patients treated with 24 mg, 51% treated with 72 mg and 42% treated with 240 mg compared to 26% treated with omalizumab and none with placebo in patients with chronic spontaneous urticaria.
 
In a 30-day, 828 patient, Phase III trial, treatment with selepressin did not improve ventilator- and vasopressor-free days compared to placebo in patients with septic shock receiving norepinephrine.
 
In a 6-month, 94 patient, Phase III, open-label, SONICS trial, 82% of patients treated with levoketoconazole had their mean 24-h urinary free cortisol (mUFC) normalized during a titration phase in patients with Cushing’s syndrome. During the 6-month maintenance phase of the trial 31% of patients achieved and maintained normal mUFC.