As Athersys Inc. moves toward a Phase 3 clinical trial for its stroke treatment, the Cleveland-based biotech company has received a federal designation that CEO Gil Van Bokkelen says is a recognition of the therapy's importance.
The U.S. Food & Drug Administration awarded Athersys' clinical program for stroke fast track designation, an expedited drug development and review program for new drugs or biologics to treat serious or life-threatening conditions. In the government's fiscal year 2016, the FDA granted 132 of these requests.
"It's really a sign that the FDA is trying to support to the fullest extent possible what we are doing, because they recognize that we've targeted something that has shown promise and that is really focused on a serious unmet medical need," Van Bokkelen said.
Athersys' MultiStem is an adult stem cell therapy for ischemic stroke patients.
The designation means Athersys' clinical program for stroke is eligible for three main things, Van Bokkelen said: rolling submission, in which the company can submit data as it goes through the trial; accelerated approval, making the program eligible for expedited consideration in certain respects; and priority review, which shortens the timeframe of the review cycle.
In the near term, he said, it allows Athersys to engage more frequently with the FDA and "if our data warrants it, to then be in a position to really get through that regulatory process as quickly and efficiently as possible."
While shaving off about six months of the process may seem small, Van Bokkelen stresses that it is a "really big deal."
"If you put that in the context in terms of the number of patients that we might otherwise be able to help, six months is actually very important," he said. "It's very valuable."
In February 2016, Athersys said that MultiStem performed well in a smaller Phase 2 trial involving stroke patients. Two white papers released earlier this year laid out the results from the Phase 2 trial, showing that if administered within 36 hours after a stroke has occurred, MultiStem can help prevent damage from the stroke.
At the time, Van Bokkelen said that the company was steadily ticking off important milestones "and now we're in the home stretch," he said. "And that's pretty exciting, because in the not terribly far off future, we might be in the position to have developed a new therapy for stroke."
Dr. Ken Uchino, research director at the Cleveland Clinic's Cerebrovascular Center, participated in the Phase 2 trial. He noted that a large portion of patients don't receive needed therapy because they're too late for the three-hour window that the standard stroke treatment requires.
With MultiStem, that window extends out 36 hours after time of stroke. This was critical for Kristopher Little, who three years ago woke up around 4 a.m. unable to move. Upon seeking medical care, the Newcomerstown resident eventually was transported to the Clinic.
Little presented with a "moderately severe stroke," said Uchino, also an associate professor of medicine (neurology) at the Cleveland Clinic Lerner College of Medicine of Case Western Reserve University.
Unsure of when overnight he had the stroke, the time that had passed made Little a candidate for the Athersys Phase 2 trial. After receiving treatment, he did weeks of patient rehab, where he re-learned how to walk.
"It's not 100%," Little said, "but it's good enough I can use it."
Uchino notes that cannot draw a one-to-one correlation, but from early on, Little did very well. And while he of course doesn't know what to expect from Phase 3, Uchino remains "hopeful and optimistic."
The FDA designation comes as Healios K.K., Athersys' partner in Japan, gears up for patient enrollment of its 220-patient study.
By the end of the year, Athersys plans to launch a 300-patient Phase 3 trial in the United States and Canada, as well as in the European Union and in the United Kingdom. This summer, the company is focused on ensuring regulatory alignment with European regulators.
Last fall, Athersys' design of its planned Phase 3 clinical trial received FDA approval under what's called a "Special Protocol Assessment."
Although the company hasn't said anything about specific dollars needed for the Phase 3 trial, Athersys is exploring potential partnerships with pharmaceutical companies that could help fund the study, according to a spokesperson.
The fast track designation is a positive indicator for companies that Athersys is in partnership discussions with, Van Bokkelen said.
"They see that we have the (Special Protocol Assessment), they see the Fast Track Designation. Those are all things that are pointing in the right direction for potential partners," he said. "And it's I think, just one more tangible thing for them to be able to look at to say, 'Yeah, these guys are doing it right.' "