- Galapagos N.V. has new positive data in hand for one of its leading cystic fibrosis candidates, but some analysts aren't sold that the drug — or the company, for that matter — poses a substantial threat to Vertex Pharmaceuticals Inc., the heavyweight in the therapeutic area.
- Topline results from the Phase 2 ALBATROSS study showed Galapagos' C1 corrector GLPG2222 was well-tolerated, with investigators reporting no serious adverse events or patient dropouts due to adverse events. All enrolled patients had a gating mutation on one allele and an F508del mutation on the other allele, and all were on long-term, stable Kalydeco treatment before and throughout the study.
- The study tested two doses of GLPG2222, 150 mg and 300 mg, versus placebo. Jefferies analyst Peter Welford explained in a Nov. 20 note that dose modeling will be vital for Galapagos' future triple combination therapies. To that end, ALBATROSS patients in both experimental arms demonstrated dose-dependent responses with regard to their sweat chloride concentrations and their mean percent predicted FEV1 (ppFEV1) levels.
Galapagos' new data was met with mixed feedback from analysts. On the one hand, GLPG2222 did show positive signs of efficacy and safety, reinforcing Welford's Buy rating for Galapagos.
The results "importantly again confirm the drug is safe and validate the cystic fibrosis (CF) platform, also providing Galapagos insights into dosing," Welford said. "Efficacy data are hard to interpret as demonstrating a benefit in this CF subset on top of standard-of-care Kalydeco is challenging, nevertheless dose-response and signs of activity are encouraging, in our view."
On the other hand, it's hard to envision a scenario wherein Galapagos gives Vertex a run for its money. Vertex already has two cystic fibrosis products on the market: its potentiator Kayldeco (ivacaftor) and its potentiator/corrector combo Orkambi (lumacaftor/ivacaftor), which together raked in $550 million during the third quarter alone. The Food and Drug Administration is also set to make an approval decision on another Vertex pairing, Kalydeco and the investigational tezacaftor, in early 2018.
That arsenal of drugs not only gives Vertex a large head start on the market, it also helps usher therapies through the clinic faster. The biotech is currently investigating a variety of triple combos with the Kalydeco and tezacaftor backbone, and should be well into Phase 3 testing for them by early 2018, according to Jefferies analyst Michael Yee.
Galapagos, however, doesn't have the same infrastructure, meaning it likely won't advance into late-stage trials until mid-2018.
"The FDA is likely to require [Galapagos] to run combinations against combinations to prove three drugs are better than two," Yee wrote in a Nov. 19 note to Vertex investors. "Vertex was also required to do this and can easily do this for their triple given it will already have [tezacaftor/ivacaftor] approved early next year as a control. It’s not clear how [Galapagos] will be able to do this without creating a problem."
Galapagos stock opened at $94.83 per share on Monday, up 4.4% from Friday's close-of-market. Vertex shares were down less than 1% to $146.92 apiece at market's open on Monday, but they continued to fall across morning trading.
"The results of this trial are encouraging as they show that the addition of the novel CFTR corrector molecule GLPG2222 on top of highly efficacious CFTR modulator treatment already given for years in patients with gating mutations was well tolerated and may bring additional benefit to patients," Scott Bell, the principal investigator for ALBATROSS, said in a Sunday statement from Galapagos.
Patients taking the 300 mg dose of GLPG2222 had a 6 millimoles per litre decrease in their sweat chloride concentration, while those taking the 150 mg dose had a 3.8 millimoles per litre decrease. Both decreases were statistical significant compared to the 5.6 millimoles per litre increase seen with patients on placebo.
Though not statistically significant, another outcome observed was that patients on the 300 mg and 150 mg regimens had better ppFEV1 levels than those in the placebo arm. There was a 2.2% increase in the 300 mg arm, 0.6% decrease in the 150 mg patients and 0.8% decrease for the placebo group over the course of the study.
"We are impressed with the magnitude of the effects we saw on sweat chloride and FEV1 in patients whose treatment with ivacaftor has been optimized following years of therapy," Galapagos' Chief Scientific Officer Piet Wigerinck said in the statement.