Aiming at a single target turned out to be a smart strategy for lifitegrast’s third pivotal trial. After Shire’s dry eye project returned mixed data on multiple primary endpoints in earlier studies, the Irish-domiciled group kept Opus-3 to a single main measure and got the results it was looking for.
Shire says it will use these data in its resubmission for lifitegrast, which was rejected by US regulators just 11 days ago. The FDA has asked for an additional clinical study, but Shire has not said explicitly what the agency sought; if the group is to launch lifitegrast in 2016 the design of Opus-3, under way for a year, had better be acceptable.
Lifitegrast, a twice-daily anti-inflammatory eye drop, was significantly better than placebo on patient-reported symptoms of eye dryness at 12 weeks of treatment by an average of 7.16 points on the 100-point eye dryness score, along with key secondary endpoints.
The earlier Opus-2 trial had used co-primary endpoints of eye dryness and inferior corneal staining score, the latter of which it missed. The Opus-1 trial missed on eye dryness symptoms, and the inconsistent findings likely contributed to the FDA’s complete response letter (Shire feels the heat, October 19, 2015).
That corneal staining endpoint, a biological rather than symptomatic marker, is not listed on Opus-3’s record at clinicaltrials.gov, so if Shire is not measuring it Opus-3 surely could not fail. Whether FDA wants that data and whether this will have an impact on the agency’s decision is anybody’s guess; confirmation that lifitegrast can target symptoms ought to improve its odds.
Strengthening its hand?
For now, Shire can revel in the best news it has seen in several weeks – a repeat of positive data for its second-biggest growth driver.
Shares rose 6% to £49.01 in mid-afternoon trading on its home market in London today after the announcement, taking them back to values not seen since early September. Along with the rest of biopharma Shire suffered a late-summer slump from which it has been difficult to bounce back, particularly as pricing has risen as a political issue again.
This slump has not made it easier to execute on what might be Shire's most important strategic imperative: getting bigger to keep from being swallowed (Shire needs to acquire, and Actelion fits the bill, June 8, 2015). Its move on Baxalta has sputtered, although chief executive Flemming Ornskov reiterated Shire’s commitment to the deal in a third-quarter earnings call last week.
As this was an all-shares proposal, some $5bn has been lopped off Shire’s original $30bn offer, although Baxalta has likewise slipped. Meanwhile, the acquisitive giants of speciality pharma are always rumoured to be hunting for more prey. One of those, Valeant, is no longer in a position to strike, but Allergan, owner of the dry-eye leader Restasis, still has an open chequebook.
And today Pfizer reiterated that it was looking at opportunities, noting that the stock of numerous speciality groups had come off in the recent downturn – even if the expectations of those firms' management teams had not.
If Shire's M&A strategy has not been quite as fruitful as some had hoped, a lifitegrast win at least shows that it can succeed in the clinic. Following up next year with approval would be even more welcome.