- Vertex Pharmaceuticals on Monday in-licensed an experimental variant of its cystic fibrosis drug Kalydeco (ivacaftor), paying Concert Pharmaceuticals $160 million upfront for worldwide rights in a deal which was well received by analysts.
- Concert's CTP-656 is a deuterated version of Kalydeco that could enable once-daily dosing regimens for Vertex's cystic fibrosis (CF) combo treatments. The compound entered Phase 2 testing in CF patients with gating mutations last December and top-line data from that study is expected by year end.
- News of the licensing deal pushed up Concert's stock by more than 35% in early trading Monday morning. The influx of upfront cash from Vertex will extend Concert's cash runway by about three years, the Lexington, MA-based biotech said.
While Kalydeco is the older of Vertex's two CF drugs, the newer Orkambi (lumacaftor/ivacaftor) also uses ivacaftor as part of its formulation. The company's lead pipeline candidate, tezacaftor or VX-661, is being tested in combination with ivacaftor.
Unlike ivacaftor, which is dosed twice daily, CTP-656 has the potential to be dosed once a day — an advantage that typically helps improve patient adherence.
Picking up rights to CTP-656 could help Vertex develop once-daily triple combos or a more convenient alternative for patients already treated with Kalydeco, analysts from Jefferies and Evercore ISI said.
Per the deal with Concert, Vertex would pay up to another $60 million if CTP-656 wins approval in the U.S. and secures reimbursement in the U.K., Germany or France.
For Concert, the agreement substantially boosts its financial resources, extending its funding runway into 2021. Current cash holdings, roughly $96 million as of December 31 last year, would have been sufficient to fund operations through the second quarter of 2018.
With additional cash on hand, Concert can better fund development of its wholly-owned hair loss candidate CTP-543. Concert also is partnered with Jazz Pharmaceuticals and Celgene on two early-stage candidates.
Vertex, almost entirely dependent on revenues from its CF drugs, has staked out cautious sales forecasts for 2017, predicting $1.1 billion to $1.3 billion in net sales of Orkambi. Sales of Kalydeco are expected to be roughly flat.