From discovery to commercial launch, drug development often takes years. New tools available to the industry, like the use of biomarkers and patient-reported outcomes, promise to help inform and speed up the process.
“Being able to pick out the appropriate patients using the appropriate biomarkers and establishing a relationship between on-target pharmacology and any safety issues that might exist in novel products is both the most exciting as well as the most challenging part of our business,” said Kenneth Koblan, a 25-year veteran of the pharmaceutical industry.
As head of global translational medicine and early development & discovery sciences at Sunovion Pharmaceuticals, Koblan is tasked with leading the group that identifies and develops new therapeutic candidates for the company’s pipeline. The Marlborough, Mass.-based group has centered its efforts around drugs that target the brain and respiratory system.
Developing therapies for neurological conditions is especially challenging – it’s not easy getting at the molecular information needed to guide drug development for the brain.
“In the CNS space, we have the challenge of the brain being as inaccessible as it is. A patient who is suffering from schizophrenia or bipolar depression, for example, you can’t just go and measure their blood pressure to tell you whether or not they’re depressed that day,” he said.
But Sunovion’s team of scientists know that they need strategies to better understand how drugs can address the array of complexities that exist among groups of patients dealing with the same condition.
“As we are learning more and more about the brain, we’re appreciating that diseases don’t always have the same underlying pathology. That’s why we need to be able to ensure target engagement and ensure that we have the appropriate dose for all of our patient segments, whether they’re children and adolescents, normal healthy adults or elderly patients,” he said.
As an example, Koblan pointed to a drug that the Sunovion began working on seven years ago when he first joined the company. Dasotraline, a dual-acting dopamine and norepinephrine reuptake inhibitor discovered by Sunovion, is under review at the FDA for the treatment of attention deficit hyperactivity disorder.
To better understand the appropriate dosing for dasotraline, Koblan’s team using PET imaging to study occupancy at the product’s target receptor, he said.
“It was a disease that had previously never been studied with this novel molecular entity,” he said. “So we had to pick the dose and get it right.”
Molecular clues are essential in the drug development process. But Sunovion, like others in the industry, is also using patient preferences to help inform its drug development, according to Koblan. The company’s sublingual film for Parkinson’s disease, which is under review by the FDA, is one example of a formulation that was born from understanding patients’ needs.
“The product is a drug that’s known in the field – apomorphine. It’s actually sold as an injection. But when a Parkinson’s patient has an ‘off’ period, where they’re immobile, by definition they’re not able to take the needle cap off and inject themselves. And loved ones and caregivers find injecting other people rather bothersome. What we’ve been able to do is develop a thin film strip,” he said.
The product, designed to help patients who are experiencing a resurgence of symptoms usually controlled by their L-dopa regimen, allows for oral absorption through a patient’s buccal cells.
In a Phase III, 109-patient study, Sunovion reported that people who used the sublingual film had statistically significant improved motor function after 12 weeks compared to those in the placebo group. The trial also found that a significantly greater proportion of people treated with the drug had an “on” response within 30 minutes after dosing at 12 weeks compared to the placebo group.
“That makes a big difference to a Parkinson’s patient who ran out of their traditional L-dopa therapy and is now sitting in a chair, not able to talk or swallow or get up and move around,” Koblan said.
“Sometimes, that unmet need is about how the patient actually receives the therapy,” he added.
Sunovion’s sublingual film for Parkinson’s disease isn’t the first product that the company has tried to differentiate using a unique administration strategy. In April, Sunovion launched its Lonhala Magnair product in the U.S. for the long-term maintenance treatment of airflow obstruction in people with chronic obstructive pulmonary disease.
The company’s product is delivered using its Magnair nebulizer system, which is designed to administer medication in two to three minutes.
“[Patients] traditionally take that drug over 20 minutes and they usually have to take it in two consecutive 20-minute periods,” Koblan said. “If you spend 40 minutes sitting there, taking your inhaled therapy and you have something that’s as big as a shoebox plugged into the wall and you’re stuck in your room doing that – that’s pretty limiting. Our new product is a two-minute inhalation and it’s the size of your cell phone. It can fit in your pocket.”
Looking ahead, Koblan and his team at Sunovion see a future where the industry’s drug development efforts are informed by new tools like digital biomarkers and patient-reported outcomes.
“The registration endpoints of the past drug labels will not meet our patients’ needs going forward,” he said. “We are very much focused on how we take the field of big data and come up with new questions around how we can turn those data for patient outcomes into novel registration endpoints and deliver on patients’ needs.”
Steve MacMillan took over as CEO of Hologic in 2013, drawing on his experience at medtech titans like Stryker and Johnson & Johnson. Since then, Hologic has grown into a $3 billion business.
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