With the acquisition of two commercial medicines from Travere Therapeutics, liver disease specialist Mirum Pharmaceuticals has gone from a company with one product to having a “franchise,” according to CEO Chris Peetz.
Now—in addition to its potential blockbuster Livmarli—Mirum has gained bile acid treatments Cholbam and Chenodal for $210 million upfront and $235 million in potential milestones. Those payments will be tiered to net sales thresholds of the products ranging between $125 million and $500 million annually, the companies said.
For San Francisco-based Mirum, which entered its commercial phase when it gained FDA approval for Livmarli in 2021, the acquisition adds “operating scale to our business in terms of revenue and cash flow,” Peetz said in an interview with Fierce Pharma.
“This really is a unique fit,” Peetz added. “Cholbam and Chenodal are largely prescribed by the same specialists that prescribe Livmarli, so it’s an ability to expand what we have to offer and what we bring to pediatric hepatologists and how they care for their patients.”
As for Travere, the deal strengthens its financial foundation and allows the company to focus on the launch of Filspari, a first-in-class therapy which was approved five months ago for immunoglobulin A nephropathy (IgAN). The drug, which is a castoff from Bristol Myers Squibb, has blockbuster potential, according to William Blair analyst Tim Lugo.
“We believe this deal makes strategic sense for Travere as it allows the company to realize several years of value from the bile acid portfolio upfront, extending the company’s runway and allowing it to focus on its growth portfolio,” Lugo wrote in a note to investors. “We also view this as a bullish sign of management’s confidence in the ongoing launch of Filspari.”
Lugo also pointed to the enhanced ability of Travere to develop pipeline assets sparsentan in focal segmental glomerulosclerosis and pegtibatinase in homocystinuria—both of which are “future treatment standards in their respective indications,” the company’s CEO Eric Dube, Ph.D., said in a release.
The sale, which is expected to be complete in the third quarter, also helps the San Diego-based company distance itself from its past as it was founded in 2014 by disgraced “Pharma Bro” Martin Shkreli.
Travere told Fierce Pharma that the companies are “working together to transition select employees to Mirum to support this portfolio.” Peetz added that Mirum will “extend offers to some of the Travere team that was working on these programs.”
The drugs Mirum acquires accounted for $102.6 million in sales last year, up from $95.7 million in 2021.
Cholbam, which was approved in 2015, is for patients with bile acid synthesis disorders because of single enzyme defects and for adjunctive treatment of patients with peroxisome biogenesis disorder-Zellweger spectrum disorder.
Chenodal, which has been approved for radiolucent gallstones, also received orphan-drug designation for the treatment of cerebrotendinous xanthomatosis (CTX) in 2010. The drug never gained approval but has been classified as a “medical necessity” by the FDA and has been used off label as a standard of care for CTX for decades.
Mirum will also take over a phase 3 trial of Chenodal, which could lead to an approval which would allow the company to promote its use in the indication and be more proactive in screening those with the disease.
“We think that we’ll be able to diagnose more undiagnosed patients,” Peetz said. “And by treating this disease earlier, you can prevent some of the accumulation of irreversible defects.”
As for Livmarli, Mirum reported sales of $75 million last year and $29 million in the first quarter of this year. Livmarli has been approved to treat Alagille syndrome (AS) and is up for an FDA decision in progressive familial intrahepatic cholestasis (PFIC).
In the pediatric liver disease arena, Mirum is in a two-way race with AstraZeneca spinoff Albireo, which was bought out by Ipsen in January of this year.
With an approval in June for Bylvay to treat AS, on top of a previous nod for PFIC, Albireo has had a regulatory head start in the battle. But Mirum has had more success in generating sales for Livmarli, and its stellar clinical results in PFIC suggest the trend could continue.
Both companies—Albireo in phase 3 and Mirum in phase 2—have trials underway in biliary atresia, which is the largest of the three indications.