Mirum Pharmaceuticals Announces Positive Phase 3 RESTORE Study Results Evaluating Chenodal in Patients with Cerebrotendinous Xanthomatosis

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced positive data from the Phase 3 RESTORE study evaluating Chenodal (chenodiol) tablets in 13 adult patients with cerebrotendinous xanthomatosis, or CTX. The study objective was to evaluate the safety and efficacy of Chenodal by measurement of urine bile alcohols and other secondary measures. The primary endpoint of reduction in bile alcohols (urine 23S-pentol) was highly statistically significant (p<0.0001). The difference between placebo and active Chenodal at the end of the randomized double-blind withdrawal period was 20-fold.

In CTX, a deficiency of the bile acid CDCA leads to a buildup of bile alcohols which precedes a toxic accumulation of cholestanol. Cholestanol is the key driver of symptomatic burden and disease progression, including irreversible neurologic dysfunction. Results from the RESTORE study demonstrated that treatment with Chenodal not only improved urine bile alcohols but also serum cholestanol. Additionally, a greater proportion of patients receiving placebo required blinded rescue therapy, demonstrating the robustness of the effect.

“The statistically significant reductions in bile alcohols and cholestanol underscore the potential for Chenodal to have a dramatic and meaningful impact on patients with CTX,” said Chris Peetz, president and chief executive officer at Mirum. “This is an extraordinary outcome, and we look forward to moving quickly to submit these data to the FDA with the goal of broadening the impact of Chenodal for patients with CTX. We are grateful to the patients and healthcare providers who made these results possible, and to the Travere team for their dedicated work leading this landmark study.”

	Difference at end of double-blind period (placebo relative to Chenodal)	P-value
Primary Endpoint
Urine 23S-Pentol (bile alcohol) ng/mL	20-fold increase (CI: 10.3, 43.5)	<0.0001
Key Secondary Endpoints
Rescue medication*	61.5% of placebo patients (CI: 31.6%, 86.1%)