Update for April 13, 2021

Regulatory Update 
 
The FDA extended the review period for Pfizer’s abrocitinib by three months and moved the PDUFA date to the end of July.
 
The FDA granted eftilagimod alpha a Fast Track designation for the treatment of recurrent or metastatic head and neck squamous cell carcinoma.
 
Announced Research Updates
 
Fibrogen announced that safety data for roxadustat had inadvertently been miscalculated using post-hoc stratification factors. When the calculations were done using the pre-specified stratification factors approved by the FDA, the hazard ratios for MACE (all-cause mortality, stroke, and myocardial infarction), MACE+ (hospitalization due to heart failure and unstable angina added to MACE) and all-cause mortality were slightly higher for dialysis and non-dialysis patients.
 
The FDA requested additional information from Provention Bio regarding how the pharmacokinetic and pharmacodynamic (PK/PD) properties compare between templizumab manufactured by Lilly and AGC Biologics. Prevention Bio has used teplizumab manufactured by Lilly for its clinical trials. The company plans to have AGC Biologics manufacturer the drug for commercial use. The FDA did not find a PK/PD bridging study to support AGC Biologics manufactured templizumab to be comparable to the Lilly manufactured drug, so additional data would be required. While Prevention Bio believes this requirement will lead to a delay in approval of templizumab, there has not been a change in the PDUFA date or advisory committee meeting.
 
Published Research Updates 
 
ICER estimated the cost of treatment with ciltacabtagene autoleucel as $317,000 to be cost effective at $100,000 per quality adjusted life year (QALY) and $427,000 to be cost effective at $150,000 per QALY. ICER found the clinical evidence to support at least a small or substantial health benefit with a high likelihood of at least a net health benefit. 
 
The 748 patient, Phase III, MERU trial was terminated after an interim analysis did not find a survival benefit with rovalpituzumab as first-line maintenance therapy after platinum-based chemotherapy compared to placebo in patients with extensive-stage small cell lung cancer (SCLC) compared to placebo.
 
In the 26-week, 24 patient, Phase III, open-label CARDINAL trial, 13/24 (54.2%) patients treated with sutimlimab achieved an increase in hemoglobin of at least 2 g/dL or an average hemoglobin level of at least 12 g/dL and did not require a transfusion or pharmacotherapy between weeks 5 to 26 in patients with cold agglutinin disease.
 
In a 91 patient, Chinese, Phase II study anlotinib demonstrated an average progression free survival of 20.7 months compared to 11.1 months in patients with relapsed medullary thyroid carcinoma.
 
In the 6-week, 37 patient, Phase II, HELP trial, levosimendan did not reduce pulmonary capillary wedge pressure (PCWP) during exercise compared to placebo in patients with pulmonary hypertension and heart failure with preserved ejection fraction.