Even wat vers nieuws van de concurrent: Vertex Pharmaceuticals (VRTX)
announced its 2015 business outlook on Sunday night, ahead of Monday's presentation at the J.P. Morgan Healthcare Conference. There were a some interesting tidbits offered:
1. Vertex did not announce results from the 12-week, phase II study of next-generation cystic fibrosis drug VX-661 combined with Kalydeco (those data are coming later in the quarter.) However, Vertex did say a broad phase III program for VX-661/Kalydeco will start in February, which is a wee bit earlier than expected. Vertex had been guiding to start these registration studies in the first half of the year but is accelerating the program based on an internal, interim look at the VX-661 data and in consultation with FDA, a company spokesman told me.
The first of the VX-661/Kalydeco phase III studies, starting in February, will enroll patients with two copies (homozygous) of the 508del mutation. The remaining three studies are expected to start in the second quarter and will enroll cystic fibrosis patients with a single copy of the 508del mutation plus one other mutation. Together, these patients make up the large "heterozygous" 508del mutation cystic fibrosis group. These studies will treat patients for eight to 12 weeks, so Vertex is likely to have top-line results at the back end of this year, which is definitely earlier than investors were expecting.
VX-661 is a very important drug for Vertex because it's believed to be more potent and effective than VX-809 (lumacaftor) and thereby represents a way for the company to maintain its dominance in cystic fibrosis.
2. Check out this quote from Vertex CEO Jeff Leiden: "We enter 2015 with a strong cash position of approximately $1.4 billion, which, when combined with continued revenue growth, will allow us to invest in key internal and external opportunities in CF and other diseases to bolster our pipeline and position the company to advance other transformative medicines in the coming years." [Emphasis added.]
Invest in external cystic fibrosis opportunities. Sounds like Vertex wants to be a buyer of assets. ProQR Therapeutics (PRQR) , perhaps? Just a guess on my part.
3. The FDA approval decision date for the Kalydeco/lumacaftor combination therapy in homozygous 508del mutation patients is July 5. In Europe, the combination therapy has been granted accelerated review. Don't be surprised if FDA approves early.
4. Vertex gave Kalydeco 2015 net sales guidance of $560-$580 million, well below the $675 million consensus. The company says Kalydeco sales will be negatively affected in 2015 by approximately 200 cystic fibrosis patients expected to enroll in the VX-661 phase III studies instead of being treated with commercial Kalydeco. The more important investment story line for Vertex in 2015 is the approval and launch of Kalydeco/lumacaftor, and now, the outcome of the phase III VX-661 studies.
Vertex CEO Leiden presents at the J.P. Morgan conference at 12:30 PM ET today.