Vectura was studying VR475, a drug-device combination consisting of budesonide delivered by Vectura’s proprietary nebulizer inhalation system as a treatment for adolescent and adult patients with severe uncontrolled asthma.
Shares of Vectura Group plc have fallen more than 9 percent on the London Stock Exchange after the Chippenham, U.K.-based company announced its late-stage asthma study failed to meet its primary endpoints.
Vectura was studying VR475, a drug-device combination consisting of budesonide delivered by Vectura’s proprietary nebulizer inhalation system as a treatment for adolescent and adult patients with severe uncontrolled asthma.
In its announcement this morning, Vectura said that top-line results from the Phase III trial indicated a trend in the reduction of the annualized rate of clinically significant exacerbations among patients who received one of two dose levels of VR475. The trial patients received the assigned doses of the medication twice per day over the course of a year, or a placebo. However, while the data was clinically significant, Vectura said the results did not reach statistical significance. Additionally, Vectura said the open-label arm with conventional nebulizer also failed to reach statistical significance compared to placebo.
The Phase III study included an assessment of the safety and efficacy of two doses of nebulized budesonide delivered by the VR475 inhalation system vs. placebo. The study included 713 patients who has severe, uncontrolled asthma in spite of stable high doses of inhaled corticosteroids plus a second controller medication. The study was powered at 80 percent to show superiority vs. placebo for the VR475 1mg treatment arm on the primary endpoint, Vectura said.
As a result of the trial data, Vectura said it has decided to no longer pursue the further development and partnering of VR475. Vectura will complete the full analysis of the primary and secondary data and intends to present the complete study results at an upcoming medical conference. Also, the company said it will seek to have the data published in a peer-reviewed journal. Vectura noted that an initial review of the available secondary endpoints and the safety data show “certain endpoints achieving statistically significant and clinically meaningful differences between VR475 and placebo, and versus conventionally nebulized budesonide.” Those results reinforce the “differential characteristics of our guided inhalation system versus conventional nebulization, and confidence in the ongoing VR647 program and three additional early-stage nebulization programs, using non-budesonide molecules, announced earlier this year,” Vectura said this morning. Safety data for VR475 remained consistent with the known profile of inhaled budesonide.
Gonzalo de Miquel, Vectura’s chief medical officer, expressed disappointment with the Phase III results. Despite the failure to reach statistical significance, de Miquel said that he has confidence in Vectura’s proprietary technology and development capabilities.
“Vectura remains fully committed to enhancing respiratory medications by developing better formulations and superior inhalation systems for patients. I would like to thank patients, caregivers, investigators and our staff for their participation in what has been a well-conducted and executed study,” de Miquel said in a statement.
Tim Harrison, principal investigator of the Phase III trial, said the primary endpoint in the difficult-to-treat patient population “presented a high hurdle.” Despite the failure to achieve statistical significance, Harrison said the trial results suggest that nebulized budesonide is not an appropriate treatment alternative to biologic therapy in patients with severe asthma. Harrison went on to say that it remains to be seen if there is a specific severe asthma phenotype who could gain greater benefit from nebulized
“I believe the technology behind VR475 has the potential to be beneficial in the treatment of a wide range of respiratory diseases including asthma,” Harrison said.
