Boulder — Finding a way to stop diseases in their tracks drives medical researchers to keep long hours in their labs.
For Bill Marshall, the current goal is narrowed down to addressing three disease categories: blood cancers, which include leukemias and lymphomas; pathologic fibrosis; and cardiovascular disease.
His company focuses on developing molecules that influence microRNAs that are doing too much or too little to keep a body in balance. The result is intended to change the course of life-threatening and life-diminishing diseases.
As president, CEO, co-founder and director of miRagen Therapeutics Inc. in Boulder, Marshall and his team of 70 employees are seeing promising results of their work. The biopharmaceutical company currently has three drug candidate products in clinical trials.
“We see that we’re making significant differences in human lives that don’t really have an alternative,” Marshall said, describing the diseases miRagen is targeting as ones with high unmet medical needs.
“I’ve always focused on making a difference in medicine — I love the interaction between chemistry and biology,” he said. “When I saw the nature of this work I got excited about the potential of this new type of drug that should be able to treat diseases that have been hard to treat in the past.”
In 2007 Marshall co-founded miRagen when the work he and some colleagues had done started to show promise in altering the course of disease development by manipulating microRNAs. Today that work is turning heads in medical research fields.
The first of MiRagen’s drug candidates — MRG-106 — went into Phase 1 clinical trial in late 2015. The molecule is targeting a rare T-cell lymphoma, and over 90 percent of the patients who’ve received it in this trial are showing reduction in the way the disease progresses.
Phase 1 clinical trials look at the safety and tolerability of a drug in humans. Phase 2 evaluates the benefit a drug provides for a larger group. Phase 3 works with an even larger group of people and compares the treatment’s safety and effectiveness against current standard treatments.
“We used safety and clinical efficacy data to talk to the FDA about moving forward with a Phase 2 trial of Cobomarsen (MRG-106) and treating patients with established end points.”
Those end point markers include achieving 50 percent reduction with no progression of the disease in the blood and for the state to be stable for four months.
If the trial meets primary end points like these, miRagen plans to discuss getting accelerated approval with the FDA and taking the drug candidate to market rather than to Phase 3, according to Marshall.
MiRagen is currently designing the trial parameters with a plan to have the MRG-106 Phase 2 clinical trial up and running by the fourth quarter of 2018.
In addition to MRG-106 clinical trials, miRagen just entered Phase 2 trials for its second drug candidate — MRG-201. It is intended to address the formation of keloids, which are sometimes itchy and painful scars that can develop after an injury, as well as pulmonary and ocular fibrosis.
Another miRagen molecule — MRG-110 — currently in Phase 1 clinical trials, is intended to lead to a potential treatment for heart failure, another difficult-to-treat disease.
In 2011 MiRagen entered an agreement with Servier Laboratories, a French pharmaceutical company that specializes in medication for cardiological and rheumatological conditions. As a result, MiRagen maintains commercialization rights for MRG-110 in the U.S. and Japan while Servier has commercialization rights in the rest of the world.
Marshall describes his company as one that focuses on mircoRNAs that affect many different diseases rather than on starting with specific diseases and then looking for ways to treat them. “We’re a little agnostic in the diseases we look at,” he said.
“There’s a lot of demonizing examples of pharmaceutical companies but those stories are not about innovation,” he said. “What we’re doing is as innovative as it gets, should solve issues in diseases and potentially will change the way we treat diseases.