Update for the Week Ending January 11, 2020

Regulatory Update
 
The FDA approved avapritinib (Ayvakit, Blueprint Medicines Corp) on 1/9/20 for the treatment of unresectable or metastatic gastrointestinal stromal tumors (GIST) that harbor a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation.
 
Incyte licensed tafasitamab from MorphoSys and will co-promote the drug in the U.S. and exclusively market the drug outside the U.S.
 
Announced Research Updates
 
Apellis announced that in the 16-week, 80 patient, Phase III, PEGASUS trial, patients treated with pegcetacoplan had a 2.4 g/dL increase in hemoglobin compared to a 1.5 g/dL decrease with eculizumab in patients with paroxysmal nocturnal hemoglobinuria.
 
Blueprint announced that in the 80 patient, Phase I/II, open label, ARROW trial treatment with pralsetinib resulted in a 61% ORR in patients with RET fusion-positive non-small cell lung cancer previously treated with platinum-based chemotherapy. Pralsetinib is available in an expanded access program through NCT04204928.
 
DBV Technologies announced that in the 3-year, 141 patient, open-label PEOPLES trial extension of the PEPITES trial, 75.9% of patients that received a Viaskin peanut patch increased the amount peanut protein they tolerated and 51.8% could tolerate at least 1,000 mg peanut protein.
 
Published Research Updates
 
In the 26-week, 59 patient, Phase II REAL 3 trial, height velocity achieved with weekly somapacitan (doses 0.04, 0.08 and 0.16 mg/kg/week) was 8 to 12.9 cm/year compared to 11.4 cm/year with daily growth hormone (0.034 mg/kg/day) in children with with growth hormone deficiency. In a 26-week extension (52 weeks total) of the REAL 3 trial, height velocity achieved with weekly somapacitan was 4.72 to 8.6 cm/year compared to 7.41 cm/year with daily growth hormone.