- Shares of Aldeyra Therapeutics spiked nearly 25% Wednesday morning as the Massachusetts biotech reported positive data from a Phase 2b study of its lead candidate, reproxalap, in patients with dry eye disease. It's unclear, however, whether that readout will remain as encouraging over time.
- Investigators found patients using topical ocular reproxalap, in comparison to vehicle, experienced statistically significant reductions in disease severity across two different scoring systems. Aldeyra said its drug was well-tolerated and side effects, when they arose, were mostly mild.
- Yet the company noted in a presentation that p-values, which gauge whether the treatment effects seen in clinical trials are true, could change upon further quality control analysis — raising questions if the current results may not look as solid down the line.
Dry eye drugs are in high demand, both among patients and pharmaceutical companies. Ireland's Shire, for instance, said product sales in its ophthalmics business climbed 75% year over year to $100 million during the second quarter because of strong growth in the Xiidra (lifitegrast) brand.
Clinical-stage Aldeyra is looking to break into that lucrative market, and argues its drug has advantages over currently available treatments.
"One major challenge with Restasis and Xiidra, which are the only two products that are approved for dry eye disease today, is that both of those take a while to work, particularly Restasis, which takes months in some cases to demonstrate any efficacy, by which time the patients have forgotten what they felt like before," company CEO Todd Brady said on a second quarter earnings call in August.
The results announced Wednesday look as though they'll support reproxalap advancing in dry eye. David McMullin, senior vice president of corporate development and strategy, explained in an interview with BioPharma Dive that the next steps will be to meet with regulators and get the go-ahead on a Phase 3 program, with a first late-stage study pegged for 2019.
Not everyone was as gung-ho, however. An Aldeyra presentation said the Phase 2b results were based on the study's intent-to-treat population with observed data, which had some commentators wondering whether the company wasn't offering a full view of how all patients' responded to reproxalap.
also note 'values subject to change based on quality control analysis' on slides— zach (@zbiotech) September 26, 2018
rush to get data, not true ITT, and the above...just give the legit ITT/QC'd data$ALDX
Aldeyra clarified to BioPharma Dive that the "intent-to-treat population with observed data" covered all patients enrolled in the trial. Sixteen of the 300 enrolled patients discontinued, but all data leading up to those drop outs were included in the results, according to company executives. They also said patients who stayed in the trial met all their follow-up visits.
Executives have underscored that they weren't expecting their drug to hit statistical significance in the Phase 2b trial because it wasn't statistically powered, due to the fact that this was the first time vehicle was used in reproxalap dry eye testing. Rather, the point was to learn how to power future Phase 3 studies.
As such, "We were pleasantly surprised to see the statistical significance that we did," McMullin said.