Piper Jaffray analyst Edward Tenthoff started Proteostasis Therapeutics (PTI) with an Overweight rating and $14 price target. The company recently reported positive Phase I data on proprietary CFTR potentiator PTI-808 and corrector PTI-801 in homozygous F508del cystic fibrosis patients, Tenthoff tells investors in a research note. He believes Proteostasis "can win" either by competing with TripleRx or as an add-on to existing Symdeko patients. The analyst sees a "huge valuation disconnect" between cystic fibrosis market leader Vertex (VRTX) at $44B versus Proteostasis at $286M.

Vertex Pharmaceuticals announced that the European Commission has granted approval for the label expansion of KALYDECO for the treatment of infants down to 1 month of age with cystic fibrosis who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator gene: R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. As a result of existing access agreements in Austria, Czech Republic, Denmark, Ireland, Norway, Sweden, and The Netherlands, eligible patients will have access to the expanded indication of KALYDECO shortly following regulatory approval by the European Commission. Vertex will continue to work with reimbursement authorities across the European Union to ensure access for all other eligible patients. In the U.K., following MHRA approval at the end of 2023, and as a result of the existing reimbursement agreement between Vertex and the National Health Service, eligible infants ages 1 month and older in the U.K. have access to this expanded indication for KALYDECO.