Acorda Touts Success in Parkinson’s Drug Study, Awaits Safety Data

Xconomy New York — 

Acorda Therapeutics took a calculated risk in 2014 when it bought Civitas Therapeutics in a bid to start accumulating drugs for Parkinson’s disease. But that deal has a chance to soon pay off for the Ardsley, NY, company.

Acorda (NASDAQ: ACOR) said that CVT-301, its experimental Parkinson’s drug, succeeded in a 339-patient Phase 3 trial. The drug—an inhalable form of the decades-old Parkinson’s treatment levodopa—bested a placebo at improving the motor function of Parkinson’s patients suffering “off” episodes, when their medications aren’t working.

About a million people in the U.S. suffer from Parkinson’s, according to the National Parkinson Foundation, and levodopa pills have remained the standard of care for the disease since the 1960s. But over time patients’ responses start to levodopa start fluctuating. There are fewer hours per day of “on” time, when the drug is working, and more “off” time, when symptoms re-emerge. To compensate, patients increase their levodopa doses, but that can cause dyskinesia, or uncontrollable, spastic movements. Acorda’s drug—as well as a rival from Sunovion Pharmaceuticals also in Phase 3 testing—are meant to combat the fluctuating response problem.

Patients in Acorda’s trial used either a low (60 mg) or high (84 mg) dose of CVT-301 or a placebo during “off” episodes. The study’s main goal was for the high dose to improve patients’ motor function as measured by changes of the Unified Parkinson’s Disease Rating Scale (UPDRS)—a well-established measure of patients’ motor symptoms—compared to placebo. The measure was taken 30 minutes after treatment during week 12 of the study. Patients who used a high dose of CVT-301 during their “off” episodes saw their UPDRS scores drop by 9.83 points, compared to a 5.91 point drop for placebo patients—a roughly 4-point difference.

Still, Acorda didn’t disclose the UPDRS score for the low dose of CVT-301. It also didn’t say how CVT-301 performed on a variety of secondary measures, such as helping patients achieve “on” time within 60 minutes of treatment and sustain it for another 60 minutes. More details will come at a future medical meeting, and the additional data will be telling to really measure the benefit of the drug.

In a research note, for instance, Leerink Partners analyst Paul Matteis noted that the roughly 4-point UPDRS score benefit for CVT-301 was much smaller than the 9-point difference between CVT-301 and placebo seen in Phase 2 testing. But the placebo effect in Acorda’s Phase 3 also seems higher, he wrote.

“We’d imagine that the benefit is still clinically meaningful (given that patients are not supposed to change their oral [levodopa] regimen to get out of an off state), though secondary endpoint data (including earlier efficacy cuts) will be important,” he wrote.

Acorda did say the drug’s safety results were consistent with what it’s seen in earlier studies. The most common side effects were coughs and upper respiratory tract infections. While a patient on a low dose of CVT-301 committed suicide, it wasn’t deemed by trial investigators to be related to Acorda’s drug. But the data expected shortly will paint a more detailed picture of CVT-301’s safety. Two long-term safety studies—in which patients used CVT-301 for up to 12 months—should produce data by the end of the first quarter. If all goes well, Acorda will file approval applications in the U.S. in the second quarter and in Europe by the end of the year.

Shares of Acorda were up about 8 percent in pre-market trading Wednesday.

CVT-301 is a critical drug for Acorda, which has dealt with some setbacks in a long-running plan to diversify beyond its multiple sclerosis drug, dalfampridine (Ampyra). In 2012, for instance, the company acquired Neuronex for its nasal spray form of the old seizure control drug diazepam, but shelved it four years later after disappointing data. The company had also hoped dalfampridine might help post-stroke patients walk better, but that effort failed last year.

Acorda has used a few deals to place bets on experimental Parkinson’s drugs. It bought Civitas—a former spinoff of Alkermes—for $525 million in September 2014, just as Civitas was preparing to go public. And in January 2016, Acorda bought Biotie Therapies for $363 million to get SYN115, which, like CVT-301, is meant to help Parkinson’s patients during their “off” episodes. SYN115 is also in Phase 3 testing.