Update for February 2, 2021

Regulatory Update
 
The FDA accepted the NDA for Iterum Therapeutics’ oral sulopenem etzadroxil/probenecid for the treatment of uncomplicated urinary tract infections in patients with a quinolone non-susceptible pathogen and set a PDUFA date for 7/25/2021.
 
The FDA extended the review date for aducanumab by three months to allow additional time to review new data Biogen has submitted to satisfy an FDA information request. The PDUFA date is 6/7/2021.
 
The FDA granted Junshi Biosciences’ toripalimab Fast Track status for the treatment of mucosal melanoma.
 
ICER estimated the annual cost for inclisiran to be $3,600 to $6,000 a year to be cost-effective. The range for inclisiran, a PCSK9 RNA silencing drug, is similar to the price for two PCSK9 monoclonal antibodies, evolocumab at $5,400 and alirocumab at $5,850
 
Announced Research Updates
 
Agios announced that in the 24-week, 27 patient, Phase III, open-label, ACTIVATE trial, 37% of patients treated with mitapivat achieved at least a 33% reduction in the number of units of red blood cells (RBC) transfused compared to the patient’s historical RBC transfusion total in patients with Pyruvate Kinase Deficiency.
 
BeiGene announced results from the 512 patient, open-label, Phase III, Chinese RATIONALE 302 trial, where treatment with tislelizumab improved overall survival compared to investigator’s choice of paclitaxel, docetaxel or irinotecan in patients with advanced unresectable or metastatic esophageal squamous cell carcinoma.
 
Chi-Med announced final results from the Phase Ib, open-label TATTON trial, where treatment with osimertinib plus savolitinib resulted in an overall response rate (ORR) of 33% in patients who received a prior third-generation EGFR TKI, a 65% ORR in patients who did not have a prior third-generation EGFR TKI and were T790M-negative and a 67% ORR in patients who had no prior third-generation EGFR TKI and were T790M-positive.
 
Incyte announced that in the 94-patient, Phase II, open-label, POD1UM-202 trial, treatment with retifanlimab resulted in an objective response rate of 13.8% in patients with locally advanced or metastatic squamous cell carcinoma of the anal canal, who progressed on, or are intolerant of, platinum-based chemotherapy.
 
LSKB and Hengrui announced that in a 31 patient, open-label, Phase II trial, adding apatinib to irinotecan or docetaxel resulted in progression-free survival of 7.43 months in patients with small cell lung cancer.
 
Published Research Updates
 
In a 16-week, 123 patient, Japanese trial, patients treated with omarigliptin achieved a 0.61% decrease in HbA1c compared to a 0.29% increase with linagliptin in patients with type 2 diabetes mellitus. After 16 weeks, all patients received omarigliptin and HbA1c was decreased by 0.57% no matter what patients had received in the first phase of the trial.
 
In the 52-week, 1,755 patient, Phase III, FINCH 1 trial, the percentage of patients that achieved ACR20 at week 12 was 76.6% with filgotinib 200 mg and 69% with filgotinib 100 mg compared to 49.9% with placebo in patients with moderate-to-severe rheumatoid arthritis with an inadequate response to MTX.
 
In a 24 patient, open-label, Phase II trial, treatment with durvalumab, tremelimumab and radiation therapy resulted in an objective response rate of 8.3% in patients with metastatic colorectal cancer.