BOSTON (TheStreet) -- After a brief hiatus, the Biotech Stock Mailbag returns.
ProQR Therapeutics (PRQR) went public just last week at $13 per share. The company's lead product, QR-010, is an RNAi therapeutic for cystic fibrosis still in preclinical testing. ProQR expects to seek permission from U.S. and European regulators to start human studies in the fourth quarter. A phase 1b study in cystic fibrosis patients will start soon after but won't have data available until the fourth quarter of next year.
Cystic fibrosis is caused by mutations in the gene responsible for the production of a protein known as CFTR. The mutations create defective CFTR protein. The lack of functional CFTR protein causes mucus to build up in the airways of the lungs. As a result, cystic fibrosis patients suffer from severe breathing problems and bacterial infections. Fatal respiratory failure is a common outcome.
Vertex management has been on the road this fall speaking at investor conferences and meeting with analysts and institutional investors. The outreach is clearly working. I've read the recent analyst notes and spoken with investors who've met with Vertex management in recent weeks. What I hear is Vertex is exuding confidence in the future of its cystic fibrosis business. The Kalydeco/VX-809 data from the "Traffic" and "Transport" studies (treating homozygous F508del patients) will be presented next month at the North American Cystic Fibrosis Conference . When Vertex announced top-line results in June, some investor griped about the clinical relevance of the 3.3% improvement in lung function. Those worries have faded. The 30% reduction in pulmonary exacerbations reported in the studies is huge and will be a highlight of the upcoming cystic fibrosis conference. Based on Vertex comments, investors are getting more bullish on Kalydeco/VX-809 pricing, which could come in above consensus.