Update for January 27, 2021

Regulatory Update
 
The FDA approved vericiguat (Verquvo, Merck, Bayer), on 1/20/2021, to reduce the risk of cardiovascular death and heart failure hospitalization following a hospitalization for heart failure or need for outpatient intravenous (IV) diuretics in adults with symptomatic chronic heart failure with reduced ejection fraction.
 
The FDA approved voclosporin (Lupkynis, Aurinia), on 1/23/2021, for the treatment of lupus nephritis. 
 
The FDA approved cabotegravir/rilpivirine injection (Cabenuva, ViiV Healthcare), on 1/21/2021, for the treatment of HIV-1 infection as a replacement for a current antiretroviral regimen in those who are virologically suppressed on a stable antiretroviral regimen with no history of treatment failure and with no known or suspected resistance to either cabotegravir or rilpivirine. Cabotegravir/rilpivirine injection is given once a month. The FDA also approved cabotegravir tablets (Vocabria, ViiV Healthcare). The tablets are taken once a day in combination with oral rilpivirine for one month prior to initiating cabotegravir/rilpivirine long-acting injection to ensure the combination is well-tolerated.
 
The FDA accepted the NDA for odevixibat for the treatment of pruritus in patients with progressive familial intrahepatic cholestasis and set a PDUFA date for 7/20/2021.
 
A site inspection by the FDA has delayed approval of GSK’s dostarlimab. GSK had expected the drug to be approved by the end of 2020, but travel restrictions due to COVID-19 has delayed an inspection of the manufacturing facility. A date has not been set for the inspection or a new PDUFA target.
 
The FDA granted toripalimab Fast Track status as a first-line treatment of mucosal melanoma.
 
Announced Research Updates
 
LSKB announced that in a 30 patient, open-label, Phase II trial, treatment with camrelizumab plus apatinib resulted in a 43.3% overall response rate as second line therapy for Chinese patients with advanced esophageal squamous cell carcinoma.
 
GSK announced data from 106 patients enrolled in cohort F of the open-label, Phase I, GARNET trial, where treatment with dostarlimab resulted in a 38.7% objective response rate in patients with deficient mismatch mutation repair (dMMR) or POLEmut non-endometrial solid tumors.
 
Roche announced that in two 48-week, Phase III trials, which included the 671 patient TENAYA Trial and the 651 patient LUCERNE trial, the change in BCVA from treatment with faricimab, at dosing intervals from eight to 16-weeks, was non-inferior to aflibercept given every eight weeks in patients with neovascular age-related macular degeneration. Almost half (45%) of people in both trials were treated every 16-weeks with faricimab.
 
Published Research Updates
 
In a 3-month, 500 patient, Phase III trial, VM202 did not improve the 24-hour NRS pain score compared to placebo in patients with painful diabetic peripheral neuropathy. In an extension of the 500 patient, Phase III trial, 101 patients were followed for 12-months and VM202 decreased the 24-hour NRS pain score compared to placebo in patients with painful diabetic peripheral neuropathy. Greater pain reduction was found in patients not receiving gabapentin or pregabalin.
 
In the 56 patient, open label Phase I, NAVIGATOR trial, treatment with avapritinib resulted in an 91% overall response rate (ORR) in patients with gastrointestinal stromal tumors and a PDGFRA D842V-mutations.
 
In a 34-week, 112 patient open label extension of the OASIS trial, 84 patients received 2 mg of etrasimod during the extension study with 64% achieving a clinical response, 33% achieving clinical remission and 43% demonstrated an endoscopic improvement. Patients that achieved a clinical response, clinical remission, or endoscopic improvement at week 12 maintained the outcome to the end of treatment in 85%, 60%, or 69% of patients, respectively. 
 
In a 24-week, 33 patient, Japanese trial, patients treated with omarigliptin achieved a 0.2% decrease in HbA1c compared to a 0.4% increase with linagliptin in patients with type 2 diabetes mellitus.
 
In a 138 patient, Phase IV trial, treatment with carbetocin decreased blood loss during surgery by 184 ml compared to placebo in women undergoing abdominal myomectomy.
 
In a 138 patient, Phase III trial, treatment with ceftobiprole resulted in 95.7% of patients achieving early clinical response at day four and 90.4% achieving clinical cure compared to 93.2% and 97.7% with placebo in pediatric patients with pneumonia.
 
In a 12-week, 45 patient, Phase II trial, treatment with elafibranor resulted in a 48.3% decrease in serum alkaline phosphatase with 80 mg and a 40.6% decrease with 120 mg compared to a 3% increase with placebo in patients with primary biliary cholangitis that had an inadequate response to ursodeoxycholic acid. 
 
In a 124 patient, Phase Il trial, treatment with adavosertib plus gemcitabine resulted in progression free survival of 4.6 months compared to 3 months with gemcitabine alone in patients with p53 tumor suppressor gene (TP53)-mutated ovarian cancer. 
 
In the 190 patient, Phase II, open-label, POLARIS-02 trial, treatment with toripalimab resulted in an objective response rate of 20.5% in Chinese patients with recurrent or metastatic nasopharyngeal carcinoma.