6 Companies Report Positive Data in Phase III Clinical Trials
Quite a few companies released results from late-stage clinical trials. Here’s a look at some of the top stories.
AveXis Releases More Data for Zolgensma for SMA
AveXis, a Novartis company, announced interim data from its Phase III STR1VE clinical trial of Zolgensma (onasemnogene abeparvovec-xioi) in spinal muscular atrophy (SMA) Type 1 a couple of weeks ago. Yesterday, they released more data, in this case, interim data from the Phase I STRONG trial, additional data from the STR1VE trial, and the first data from the Phase III SPR1NT trial.
Zolgensma is a gene therapy in development as a one-time infusion for SMA Type 1. It uses a virus vector to deliver a copy of the human SMN gene. SMA is a severe neuromuscular disease noted by the loss of motor neurons, which leads to progressive muscle weakness and paralysis.
The STRONG Phase I trial looked at two groups based on age at time of dosing—6 to 24 months of age and 24 to 60 months. All showed improvements.
The STR1VE trial is to evaluate the efficacy and safety of Zolgensma in SMA patents less than six months of age at the time of the therapy. Of 15 patients who could have reached 13.6 months of age or discontinued the study previous to that age, 13 survived without need for permanent ventilation.
SPR1NT is a Phase III trial to evaluate the safety and efficacy of Zolgensma in pre-symptomatic patients with SMA and two or three copies of SMN2 who are younger than six weeks of age. As of March 8, 2019, all patients were alive and event-free.
Neurocrine’s Opicapone Plus Levodopa in Parkinson’s
Neurocrine Biosciences presented data from two Phase III clinical trials of opicapone, a once-daily, oral catechol-O-methyltransferase (COMT) inhibitor for Parkinson’s disease. They found that patients receiving opicapone 50 mg with levodopa, the standard of care, had a significant and sustained improvement in symptoms.
Neurocrine reported on two Phase III studies, BIPARK-1 and BIPARK-2. BIPARK-1 looked at about 600 patients with Parkinson’s and motor fluctuations. They received once-daily doses of three separate doses of opicapone, placebo or 200 mg of the COMT inhibitor entacapone for 14 to 15 weeks. The primary endpoint was the change from baseline in absolute time in the OFF state. Both trials had one-year extension phases.
BIPARK-2 looked at about 400 patients with Parkinson’s and motor fluctuations. They received once-daily doses of opicapone of either 25 mg or 50 mg or placebo for 14 to 15 weeks. The primary endpoint was change from baseline in absolute time in the OFF state.
The data from these two Phase III trials showed that adding once-daily opicapone to levodopa significantly increased ON time without troublesome dyskinesia.
Urovant’s Vibegron in Overactive Bladder
Urovant Sciences presented Phase III data at the 2019 American Urological Association Annual Meeting (AUA). The drug is being evaluated in adults with overactive bladder. The EMPOWUR trial showed statistical significance with the drug over placebo in reducing daily urge urinary incontinence (UUI) episodes and reduction in daily micturitions.
“There is a need for a new treatment option with rapid onset in a once-daily, convenient dose for patients suffering from OAB symptoms,” stated David Staskin, a urologist with St. Elizabeth’s Medical Center and Associate Professor of Urology at Tufts University School of Medicine. “The EMPOWUR data suggests that vibegron, if approved, could be suitable for a large base of patients.”
The company expects to file a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) by early 2020.
Eli Lilly’s Emgality for Migraine
Eli Lilly announced it is presenting results from the Phase III EVOLVE-1 and EVOLVE-2 studies in Emgality (galcanezumab-gnlm) in monthly migraine headaches this week at the Annual Meeting of the American Academy of Neurology (AAN).
“Migraine causes people to suffer from substantial pain and impairment, no matter how frequently they occur,” stated Gudarz Davar, vice president, Neurology Development, Lilly Bio-Medicines. “These collective analyses of more than 1,700 patients with episodic migraine from our Phase III Emgality program reflect our continued commitment to aid people living with this serious neurologic disorder.”
Emgality was approved by the FDA in September 2018 for prevention of migraine in adults. These two studies stratified patients by frequency of migraine and evaluated two different doses of the drug for its ability to reduce the different subgroups’ headache frequency.
UroGen’s UGN-101 for Upper Tract Urothelial Cancer
UroGen announced results from a secondary analysis of its Phase III OLYMPUS trial of UGN-101 (mitomycin gel) in patients with endoscopically unresectable low-grade upper tract urothelial cancer (UTUC). The results were presented at the American Urological Association Annual Meeting.
Patients in the study showed a 59% complete response rate, at least in a subset of patients. Of the evaluated complete responses, 27 patients have had a six-month evaluation, and 24 out of 27, or 89%, are disease-free at six months. Overall, 5 out of 41 patients who achieved a complete response have relapsed at any time during the study.
“The results from the OLYMPUS trial continue to be compelling for new and recurrent LG UTUC, as well as for those who have unresectable tumors and would be immediate candidates for kidney removal,” stated Mark P. Schoenberg, UroGen’s chief medical officer.
The company initiated a rolling submission of UGN-101’s NDA to the FDA in December 2018.
Biogen’s Spinraza in SMA Shows Durable Treatment Effect
Biogen presented data at the AAN meeting showing that its Spinraza (nusinersen) had a durable treatment effect in patients with SMA. Babies receiving the drug were followed up to four years. The infants showed additional or new motor function improvements during that period. These were the results of two trials, SHINE and NURTURE.
“Each milestone in these studies marks a new chapter in what it means to live with SMA, and the insight provided is invaluable in understanding the long-term experience of this rare disease,” stated Alfred Sandrock, executive vice president and chief medical officer at Biogen. “These results confirm previous data from our trials, which demonstrated that early treatment with Spinraza can make a critical difference in survival and achieving motor milestones.”