Shots:

• The FDA’s BT designation is based on P-II study results assessing PTI-428 (50 mg- qd) vs PBO in 24 CF with F508del mutation on background treatment with Orkambi for 28 days
• The P-II study results: improvement in forced expiratory volume in 1 second (ppFEV1) 5.2%; increase in nasal mucosal CFTR protein
• Proteostasis’ PTI-428 is a part of triple combination therapy including PTI-808 (potentiator) & PTI-801 (corrector) involved in increasing the level of CFTR protein to treat CF patients who are homozygous for the F508del mutation in the CFTR gene

Ref: Globe NewsWire | Image: Contract Pharma