Update for June 4, 2020

Regulatory Update
 
Chi-Med plans to initiate a rolling NDA at the end of 2020 for surufatinib as a treatment of advanced neuroendocrine tumors.  
 
CHMP recommended that the EMA approve Ad26.ZEBOV and MVA-BN-Filo to prevent Ebola virus infection. The J&J Ebola vaccine is a two injection series. Zabdeno (Ad26.ZEBOV) is given first and followed two months later by Mvabea (MVA-BN-Filo).
 
Announced Research Updates
 
BioMarin announced that in a 4-year, 15 patient, Phase I/II, open-label extension trial, treatment with valoctocogene roxaparvovec resulted in a cumulative annualized bleeding rate of < 1, but Factor VIII levels declined while remaining in a range to provide hemostatic efficacy. Valoctocogene roxaparvovec is undergoing a priority review of its BLA due to being designated a Breakthrough Therapy. The drug has a PDUFA date of 8/21/2020.
 
Immutep announced interim data from the 109-patient, Phase II, open-label TACTI-002 trial evaluating eftilagimod alpha plus pembrolizumab in the treatment of patients with non-small cell lung cancer (NSCLC) or head and neck squamous cell carcinoma (HNSCC). The data suggested an overall response rate (ORR) of 53% in 17 NSCLC patients and 39% in 18 patients with HNSCC.
 
Iterum Therapeutics announced that in the 1,395 patient, Phase III SURE 2 trial, IV sulopenem followed by oral sulopenem/probenecid did not meet non-inferiority compared to IV ertapenem followed by oral ciprofloxacin/metronidazole or amoxicillin-clavulanate in the treatment of complicated urinary tract infections.
 
Aveo  announced that in the 38-month, 350 patient, Phase III, TIVO-3 trial, of patients treated with tivozanib, progression-free survival (PFS) was not significantly different from sorafenib (16.4 vs 19.2 months) in patients with highly refractory advanced or metastatic renal cell carcinoma that had failed at least two prior therapies, including a vascular EGFR TKI. (Note: results after 19-months have been published and suggested a PFS of 5.6 months with tivozanib compared to 3.9 months with sorafenib.
 
Blueprint announced results from the 116 patient, Phase I/II, open label, ARROW trial. In the RET fusion–positive non-small cell lung cancer cohort, treatment with pralsetinib resulted in a 73% objective response rate (ORR) in treatment-naïve patients and 61% in patients that were previously treated with platinum-based chemotherapy. In the cohort of patients with RET-mutant medullary thyroid cancer, treatment-naïve patients had an ORR of 74% and previously treated patients had an ORR of 60%. The FDA accepted the NDA for pralsetinib for the treatment of RET fusion–positive non-small cell lung cancer and set a PDUFA date of 11/23/2020.
 
Published Research Updates
 
In the 1-year, 291 patient, Phase II, HER2CLIMB trial, treatment with tucatinib in combination with trastuzumab and capecitabine resulted in time to intracranial progression or death (CNS-PFS) of 9.9 months and overall survival of 18.1 months compared to 4.2 months CNS-PFS and 12 months OS with trastuzumab and capecitabine alone in patients with HER2-positive breast cancer with brain metastases.
 
In the 48-week, 217 patient, Phase IIb BE ABLE 2 trial (a long-term follow-up to the Phase IIb BE ABLE 1 trial), 80 to 100% of bimekizumab responders maintained PASI90, 69-83% maintained PASI100 and 78-100% had an Investigator’s Global Assessment of 0/1.
 
In the 406 patient, Phase III ADVANCE study, 16% of patients treated with eflapegrastim developed severe neutropenia compared to 24% with pegfilgrastim in patients with early-stage breast cancer. 
 
In the 55 patient, Phase II, open-label, INSIGHT trial, treatment with tepotinib did not improve progression free survival compared to standard platinum doublet chemotherapy (4.9 vs 4.4 months) in patients with epidermal growth factor receptor (EGFR)-mutant non-small-cell lung cancer (NSCLC) with MET overexpression (immunohistochemistry [IHC]2+ or IHC3+) or MET amplification having acquired resistance to EGFR inhibition. However the trial was terminated early due to poor recruitment, so all analyses are considered to be exploratory.