By GREGORY ZELLER //
There’s a long tunnel ahead, but for millions around the world suffering from lupus, the light at the end shines brightly.
That’s the fervent belief of Meggan Mackay, a board-certified rheumatologist and assistant professor at the Hofstra Northwell School of Medicine. Mackay is initiating a Phase 2 clinical trial of a drug she says might rival today’s best treatments for the autoimmune disease but without the devastating side effects.
There’s no cure for lupus, and while current treatments like Prednisone – a steroid used to treat many inflammatory diseases – can mitigate symptoms, the fallout can be nasty. Most common: weakened and dead bone mass, diabetes, weight gain and increased susceptibility to other infections.
“Many lupus patients must be on long-term steroid use,” Mackay noted. “That’s where we hope this drug might play a role.”
This drug is JBT-101, a cannabinoid derivative also known as ajulemic acid. While it lacks psychoactive properties, doctors are high on the synthetic pharmaceutical’s analgesic and anti-inflammatory capabilities.
Patent holder Corbus Pharmaceuticals is putting the drug, branded Resunab, through its clinical paces. The Massachusetts firm is already engaged in late-Phase 2 trials pitting Resunab against cystic fibrosis and other maladies, and now Mackay – Northwell Health’s associate chairwoman of medicinal development – is launching a Phase 2 lupus investigation at the Feinstein Institute for Medical Research.
Actually, the clinical trial will be conducted at 10 sites around the country, including three University of California campuses, the University of South Carolina and NYU. But Feinstein Institute researchers, led by Mackay, are running the show, through a $1.5 million National Institutes of Health grant designating Feinstein as an Autoimmunity Center of Excellence.
The lupus trial is a long time coming; in fact, this is the second time Mackay has secured a grant to test ajulemic acid against the inflammatory disease.
The graduate of New York Medical College first learned of the drug 11 years ago, when she heard rheumatologist Robert Zurier discuss it in a talk at the University of Massachusetts. Realizing the acid’s “very powerful anti-inflammatory properties” might be just the ticket for lupus patients, the Feinstein Institute researcher became fast friends with the now-retired Zurier.
She’s also done her homework, boning up on JBT-101’s ability to both cause an inflammatory reaction in response to an infection and stop the inflammation when necessary – just like a healthy immune system.
The NIH liked the idea in 2012, when Mackay first applied for funding to put ajulemic acid in the ring with lupus, and granted her $250,000 – enough to begin protocol development for a lupus study and even recruit test sites. But with the IP bouncing between early-stage companies, including a startup launched by Zurier and JBT-101 co-creator Sumner Burstein, the NIH got cold feet, and “we were left to look for more grants,” Mackay said.
While she looked, she also paid close attention to the drug’s Phase 1 safety trials, in which JBT-101 was administered to healthy people and “no serious adverse events” were recorded, Mackay said.
Now, with Corbus Pharmaceuticals pushing forward and the new NIH funding in hand, Mackay is “very happy the trial is finally going.”
It may be going and going, for quite a while. The Phase 2 lupus trial will include 100 patients who must first be selected – “you choose 150 to test 100,” Mackay noted – and thoroughly screened to ensure they meet the right entry criteria. Each patient will then undergo a 12-week “active treatment” followed by a month of observation.
Only then will investigators start breaking down the data, according to Mackay, who estimates the Phase 2 trial will take up to three years to complete.
And a successful Phase 2 will only lead to a Phase 3 trial, which does it all over again on a much larger scale.
Bottom line: Assuming Resunab performs exactly as hoped, assuming the Phase 2 patients continue to report side effects as benign as mild headaches and nausea, assuming thousands of patients are quickly identified, screened and accepted into a Phase 3 that goes just as smoothly, it will still be “many years” until Resunab is commercially available as a lupus treatment, Mackay noted.
But when that day comes, it may prove worth the wait for the 1.5 million Americans and millions of others around the globe suffering from lupus. The drug won’t cure the disease, but may ultimately provide symptom control similar to the hard-hitting Prednisone, without the debilitating effects. It may even have muscle enough to keep lupus in remission.
“Virtually all lupus patients would benefit, if it has an equivalent efficacy to the corticosteroids,” Mackay said. “That’s the long-term goal, to replace the steroids.”
In addition to sparing lupus patients the egregious side effects, a successful Resunab could also alter the lupus-treatment market. Mackay compared monthly intravenous steroid treatments that can exceed $35,000 a year to an ajuemic acid pill that would be “a fraction of the cost.”
Between there and here, however, lie many miles of research. Mackay is confident, even though she hasn’t gleaned much from the other double-blind Phase 2 Resunab studies, other than the fact that patients are still reporting no serious adverse effects.
“Which is great,” she said.
Also great, the Feinstein Institute researcher noted, is her study’s specific focus on Resunab’s effects on the immune system itself, “something the big pharmaceuticals don’t do when they run their big clinical trials.”
“We have an incredible opportunity here, with scientists looking at the basic action of a drug and a drug company that’s bending over backwards, giving us free drugs and placebos and doing everything they can to control the costs of this study,” she said. “Everybody wants this to work, so everybody is working together.
“You have NIH backing, and Corbus and Feinstein partnering up, and researchers across the country,” Mackay added. “It’s incredibly rare.”