- Galapagos NV on Wednesday hailed positive clinical data for its lung disease drug, but some analysts are suggesting the celebration may be premature.
- After 12 weeks, patients with idiopathic pulmonary fibrosis (IPF) who took Galapagos' GLPG1690 demonstrated an 8 mL increase in their forced vital capacity (FVC), according to topline results from the Phase 2a FLORA trial. Conversely, the study's placebo arm showed an 87 mL reduction in FVC, which measures lung health by how much air a person can push out of the organ.
- While Galapagos leadership trumpeted their drug's ability to "halt disease progression," others weren't as convinced. Jefferies analyst Michael Yee highlighted in a Aug. 9 investor note about fellow IPF drugmaker FibroGen Inc. that, though promising, the FLORA investigation was limited by its small enrollment and short duration.
Investigators enrolled 23 patients in FLORA, with 17 in the treatment group and 6 in the control group, and ran the trial over roughly three months. Yee points out, however, that Phase 3 studies for rival therapies, such as FibroGen's FG-3019, employed much more robust enrollment and testing periods. That's important, because FVC tends to get worse over time, even when patients are receiving treatment.
It's "still early to make too many claims on halting disease," Yee said in his Aug. 9 note. He added that the early results don't look like they met statistical significance for FVC, though they did evince "attractive proof of concept" and some statistical significance with regard to functional respiratory imaging (FRI).
Galapagos, however, sees the trial design as a plus — reflective of its drug's fast-acting benefits for IPF patients.
"The stabilization of FVC over 12 weeks upon GLPG1690 treatment is a major milestone in IPF, where, by way of reference, the currently approved treatments show a decrease of approximately 30 mL over the same treatment period," Galapagos Chief Scientific Officer Piet Wigerinck said in an Aug. 9 statement.
What's more, the company believes the results reaffirm the promise of its drug discovery platform outside of its highly anticipated Janus kinase inhibitor filogotinib.
"Not only does GLPG1690 show early promise as a potential therapy for IPF, but it also marks an important milestone for Galapagos as a company: proof of concept in patients of a second mechanism of action coming from our target discovery platform. Galapagos has shown that this platform continues to deliver novel mechanisms of action beyond JAK-1 in inflammation," Wigerinck said.
In addition to FVC improvement, patients taking Galapagos' drug demonstrated disease stabilization, with FRI showing the treatment hit statistical significance for two specific parameters. The company didn't elaborate on what those parameters were in the Aug. 9 statement.
The company also said it has met with regulators and plans to quickly push GLPG1690 into late-stage testing.
A Food and Drug Administration approval of the drug would surely evolve Galapagos' revenues and market capitalization. The IPF patient population is large, yet there are few treatments green lit in the U.S. to treat the disease.
The medicines that have gained market access have so far been profitable. Roche AG, for instance, put down more than $8 billion to acquire InterMune, Inc. back in 2014. The deal gave the Swiss drugmaker control over the IPF drug pirfenidone, now branded as Esbriet. Revenue from the treatment totaled CHF 418 million ($435 million) in the first half of 2017.
GLPG1690 works by inhibiting autotaxin, an enzyme that creates signaling molecules and plays a role in a variety of bodily activities such as lymphocyte homing and tumor progression. Patients who took the drug in FLORA had lower levels of a biomarker for autotaxin inhibition, meaning the "level of target engagement observed in Phase 1 with healthy volunteers was confirmed in IPF patients in FLORA," according to Galapagos.
Galapagos shares climbed nearly 14% to $83.24 apiece between Wednesday afternoon and Thursday morning.