Weekly Update for the Week ending August 24, 2019

Regulatory Update
 
The FDA approved both oral and intravenous formulations of lefamulin (Xenleta, Nabriva Therapeutics) on 8/19/2019 for the treatment of community-acquired pneumonia. The WAC price per day for the IV dose is $205 and the oral dose is $275 per day.
 
The FDA rejected Sarepta Therapeutics' golodirsen (Vyondys 53) for the treatment of Duchenne muscular dystrophy amenable to exon 53 skipping due to the risk of infection with intravenous infusion ports and renal toxicity seen in pre-clinical studies. Sarepta has shown that golodirsen will increase dystrophin levels, but has not completed studies to demonstrate animprovement in muscle function.
 
The FDA accepted the NDA for VX-445 (elexacaftor, tezacaftor and ivacaftor) for the treatment of cystic fibrosis in patients with one F508del mutation and one minimal function mutation or two F508del mutations. A PDUFA date has been set for 3/19/2020.
 
The FDA granted Fast Track status to Moderna’s investigational Zika vaccine, mRNA-1893.
 
Celgene plans to submit a BLA for lisocabtagene maraleucelfor the treatment of multiple myeloma in the first half of 2020.
 
DBV submitted a BLA for Viaskin Peanut for the treatment of peanut-allergic children.
 
The FDA accepted the BLA for zanubrutinib for the treatment of mantle cell lymphoma and assigned a PDUFA date of 2/27/2020.
 
Celgene submitted a BLA for Zbb2121 for the treatment of multiple myeloma that has reappeared after remission or is resistant to treatment.
 
Announced Research Updates
 
ViiV announced that in the 48-week, 1,045 patient, Phase III ATLAS-2M trial, cabotegravir and rilpivirine given eight weeks was non-inferior to monthly injections in maintaining HIV-1 RNA < 50 copies/mL.
 
Published Research Updates
 
In the 465 patient, Phase II/III ZEUS trial, 64.7% of patients treated with injectable fosfomycin achieved clinical cure and microbiologic eradication that was non-inferior to the 54.5% overall success achieved with piperacillin-tazobactam in patients with urinary tract infection (cUTI) or acute pyelonephritis (AP). The original ZEUS data was calculated based on the 2015 FDA Guidance for the development of antibiotics to treat cUTI/AP. Nabriva announced that in a reassessment of the ZEUS data based on the 2018 FDA Guidance, which reduced the microbial eradication threshold, 78.6% for patients in the fosfomycin group achieved overall success compared to 33.3% of patients in the piperacillin-tazobactam group, in patients with piperacillin-tazobactam-resistant infections. In patients with piperacillin-tazobactam-susceptible pathogens, the overall success rate was 64.7% for patients in the fosfomycin group and 56.7% for patients in the piperacillin-tazobactam group.
 
In the 6,545 patient, Phase III trial, Norvartis’ serelaxinwas no different than placebo in preventing further decompensation over the first 5 days (6.9% vs 7.7%) or in reducing CV death at 6-months (8.7% vs 8.9%).
 
In a 38 patient, Phase II trial, treatment with Peregrine Pharmaceuticals’bavituximab plus sorafenib resulted in a median time to progression of 6.7 months in patients with advanced hepatocellular carcinoma.
 
In a 30-patient, Phase Ib, open-label, trial, treatment with Roche’s taselisib plus tamoxifen resulted in progression free survival of 3.7 months in patients with ER-positive, metastatic breast cancer who had failed prior endocrine therapy.