What’s Next for Acucela? Wait & See

Now that Acucela and Otsuka Pharmaceutical of Japan have torn up their collaborative deal to develop emixustat, which failed to meet its primary endpoint in a Phase IIb/III trial in people with geographic atrophy, the Seattle-based pharmaceutical company seems to be piecing together a plan for its next steps.

However, no one at the company will talk off script – that is, beyond what press releases say – about those next steps. The press release on Otsuka terminating the agreement said Acucela will continue to “explore the benefits of emixustat for the treatment of retinal diseases,” despite the failure of the SEATTLE study of the drug’s efficacy in the treatment of geographic atrophy secondary to age-related macular degeneration (AMD). A Phase II trial of emixustat in proliferative diabetic retinopathy is also in progress, and Acucela is sticking with its plan to start a clinical trial of the drug in Stargardt disease by the end of the year.

Acucela also announced it would postpone its annual shareholders’ meeting, previously scheduled for August 2, and it’s also scratching plans to change its domicile from Seattle to Japan. The company will update its fiscal year 2016 forecast in light of these recent events.

In the meantime, Acucela’s stock price has been lagging since peaking at ¥6,790 ($65.11) on May 24 (the stock trades on the Tokyo Exchange), a day before the company announced the SEATTLE trial results. Within a week, it had plunged to ¥1,190 ($11.41) since trending upward into the ¥1,400 range ($13.42) last week – which actually is still more than double its low in the past year, so maybe investors have found something to grab onto.

However, the stock price did climb 70% in the week before release of the SEATTLE trial results. Its sharp plunge the day the trial results came out – it fell to its 24% limit on the Tokyo exchange – may have caught the attention of the exchange’s operator for hints of insider trading, Bloomberg reported. Acucela later told Bloomberg that it found no evidence of anyone within the company leaking trial results before their release. (And if they had, one has to think it would have hurt, not helped the stock price before the release.)

The SEATTLE trial involved 508 patients with geographic atrophy secondary to AMD. Two-year results showed no difference in lesion growth between the emixustat and placebo groups.

“We are carefully reviewing the data in geographic atrophy before we decide on our next steps with emixustat in this indication,” Ryo Kubota, MD, PhD, and chairman, president, and CEO of Acucela, said at the time. “We will continue to advance our in-licensed projects as well as our in-house research.”

At the time, Acucela said it would further analyze the SEATTLE trial data “in collaboration with Otsuka Pharmaceutical in the ensuing months.” Now, Acucela is going it alone.