Update for November 16, 2021

Regulatory Update
 
The FDA approved ropeginterferon alfa-2b (Besremi, PharmaEssentia), on 11/12/2021, for the treatment of polycythemia vera. Ropeginterferon alfa-2b was approved with a Black Box Warning regarding fatal or life-threatening neuropsychiatric, autoimmune, ischemic, and infectious disorders. 
 
The FDA placed a hold on the giroctocogene fitelparvovec Phase III, AFFINE trial, in hemophilia A patients, to allow time to review changes to the trial’s protocol after high factor VIII levels were found in some treated patients.
 
Announced Research Updates
 
Compass Pathways announced that in a 12-week, 233 patient, Phase IIb clinical trial, treatment with psilocybin 25 mg decreased the MADRS by 6.6 points more than a 1 mg dose, at week three, in patients with treatment-resistant depression. The 10 mg dose was not significantly different from 1 mg. There was no placebo arm in the study.
 
Madrigal announced results from 171 patients enrolled in the 52-week, open-label portion of the 1,200 patient, Phase III, MAESTRO-NAFLD-1 trial, where treatment with resmetirom reduced the MRI-proton density fat fraction (MRI-PDFF) by > 50%, liver volume by > 20% and fibrosis compared to placebo in patients with non-cirrhotic NASH.
 
BMS announced 48-week data from 43 patients enrolled in the Phase II MAVERICK-LTE trial, a long-term extension of the MAVERICK-HCM trial, where treatment with mavacamten resulted in a 67% reduction of NT-proBNP.
 
In a draft analysis of tirzepatide for the treatment of type 2 diabetes, ICER found that treatment with tirzepatide reduced HbA1c and weight more than background therapy. Analyzing data from a direct comparison with semaglutide and a network meta-analysis with empagliflozin, tirzepatide has a greater decrease in HbA1c and weight loss compared to empagliflozin. While semaglutide and empagliflozin have shown improvement in cardiovascular (CV) outcomes, the tirzepatide CV outcomes trial has not been completed. Due to the lack of the CV outcomes data, ICER found tirzepatide to provide comparable or better net health benefits to semaglutide and empagliflozin. Using the same price for tirzepatide as semaglutide, ICER found tirzepatide to have a QALY < $100,000 when compared to empagliflozin and less costly and less effective compared to semaglutide.
 
Published Research Updates
 
In the 52-week, 191 patient, Phase II, SERENITY trial, a 50% reduction in the severity in the Simple Endoscopic Score for Crohn's Disease (SES-CD) was achieved at week 12 by 25.8% of patients treated with mirikizumab 200 mg, 37.5% with 600 mg and 43.8% with 1,000 mg compared to 10.9% with placebo in patients with moderate to severe Crohn's disease. Mirikizumab responders were re-randomized to continue their IV induction dose or receive mirikizumab 300 mg subcutaneously every four weeks. After 52-weeks, 58.5% of IV patients maintained a 50% reduction in SES-CD compared to 58.7% who received the subcutaneous dose.
 
In a six-week, 260 patient, Phase II trial, treatment with ansofaxine decreased the HAMD17 score compared to placebo in major depressive disorder.
 
In the 144 patient, Phase III, E2902 trial, treatment with tipifarnib did not improve disease-free survival compared to observation in patients with acute myeloid leukemia in remission.