Update for December 7, 2021

Regulatory Update
 
The FDA rejected plinabulin for the prevention of chemotherapy-induced neutropenia and requested an additional clinical trial.
 
The FDA delayed the PDUFA date for pacritinib to treat myelofibrosis with severe thrombocytopenia, by three months, to allow additional time to review newly submitted clinical data. The new PDUFA date is 2/28/2022.
 
Announced Research Updates
 
Vertex announced that in a 13-week, 13 patient, open-label, Phase II trial, treatment with VX-147 resulted in a 47.6% reduction in the urine protein to creatinine ratio (UPCR) in patients with APOL1-mediated focal segmental glomerulosclerosis (FSGS).
 
ImmunoGen announced that in the 106 patient, open-label, Phase III, SORAYA trial, treatment with mirvetuximab soravtansine resulted in a 32.4% ORR in patients with platinum-resistant ovarian cancer whose tumors express high levels of folate receptor alpha. 
 
SAB Biotherapeutics announced that in a 28-day, 60 patient, Phase IIa trial, treatment with SAB-176 reduced the nasopharyngeal viral load and influenza signs and symptoms compared to placebo in patients challenged with a pandemic influenza virus strain (pH1N1).
 
Kazia announced that in a 30 patient, Phase II trial, where treatment with paxalisib resulted in overall survival (OS) of 15.7 months and progression-free survival (PFS) of 8.4 month compared to 12.7 months OS and 5.3 months PFS with a historic group treated with temozolomide in patients with glioblastoma with an unmethylated O6-methylguaninemethyltransferase (MGMT) promotor following surgery and radiotherapy.
 
Biohaven announced that in a 1,405 patient, Phase II/III trial, 24% of migraine patients treated with intranasal zavegepant were pain free at two hours compared to 15% with placebo. Patients were free of their most bothersome symptoms at two hours in 40% of zavegepant patients compared to 31% of placebo patients.
 
Published Research Updates
 
An FDA review of bardoxolone questioned whether the Phase III CARDINAL trial had study design flaws and therefore if it truly demonstrated an improvement in renal function. The FDA also expressed concerns regarding long-term safety of bardoxolone due to an increase in albuminuria and blood pressure.