Building a rare disease company from the ground up

Chris Peetz, co-founder and CEO, shared his ups and downs of founding Mirum Pharmaceuticals

Chris Peetz’s journey into rare disease drug development began with an unexpected opportunity. After working across various functional areas in smaller biotech companies, he was approached with an unconventional offer.

“We have this new company creation effort. How would you like to take a huge cut in your pay and spend a couple years doing something that may amount to nothing, but we will give you office space,” Peetz told the Rare Disease Summit audience.

Despite the risks, Peetz embraced the challenge, joining Frazier Life Sciences to search for deprioritized pharmaceutical assets that could be developed by a smaller, more focused company. The turning point came when examining data for a drug that had supposedly failed in clinical trials.

“What was missed by the prior sponsor was that the endpoint that matters most to the patient was dramatically improving," Peetz explained. The drug, which would later become Livmarli, showed remarkable efficacy in reducing severe itching in children with Alagille Syndrome—a symptom so debilitating that patients would sometimes require liver transplants.Chris Peetz, CEO and co-founder Mirum Pharma

Frazier Life Sciences became a significant investor in Mirum, which was founded in May 2018 by Niall O'Donnell, Michael Grey, and Chris Peetz. It went public in July 2019.

Ups and downs

Like many companies in the rare disease space, patient voices are central to Mirum’s mission. Peetz recalls two pivotal meetings at a liver conference just weeks after founding the company.

“I met with the chairwoman of the Alagille Syndrome Alliance and the head of the Progressive Familial Intrahepatic Cholestasis Network. They’re both moms of patients with the respective genetic liver condition. It changed from a cool project in a job into a mission.”

This commitment to patient-centered development led Mirum to innovative approaches, including hiring “a mother of an Alagille Syndrome patient into our marketing group—who better to understand the situation and how to unlock great conversations with physicians?”

However, the entrepreneurial journey wasn’t without obstacles. Peetz describes the process as “a year and a half of looking around and a lot more failure than success” and acknowledges it “feels a little lonely" when starting out.

Even after founding Mirum, there were setbacks. When a study for biliary atresia didn’t work out, rather than abandoning the indication, the team pivoted. “Instead of saying ‘we have a failed study,’ it was more about maybe we’re not looking at the right spot to evaluate it.” This led to a basket study design capturing multiple rare types of cholestasis where Livmarli might benefit patients.

For Peetz, Livmarli’s first approval was transformative. “It was the first step on the mission that had been achieved. We actually got a new medicine approved, and that doesn’t get taken away.”

Looking forward

Today, Mirum has evolved beyond its initial product, acquiring additional assets and reaching cash-flow positive. Peetz sees opportunity in the gap that exists for companies willing to develop smaller products that don’t meet big pharma’s revenue thresholds.

“There’s a gap in the industry for companies that care about small products. Big pharma is looking for $2 billion plus products, and for Mirum, something that’s ultra-rare can make a big difference for us. To get through another Livmarli-type approval is why we’re here.”

Image: Mirum Pharmaceuticals.

Header image: Depositphotos@val.suprunovich