Mirum Pharmaceuticals Announces Positive Phase 3 RESTORE Study Results Evaluating Chenodal in Patients with Cerebrotendinous Xanthomatosis - Seite 2

0.0006

Plasma cholestanol µg/mL

2.8-fold increase (CI: 1.5, 5.2)

0.0083

Plasma 7αC4 ng/mL

50-fold increase (CI: 25.0, 66.7)

<0.0001

* Proportion of patients on placebo requiring rescue Chenodal during double-blind withdrawal periods

The most commonly reported adverse events while on Chenodal were diarrhea (n=5) and headache (n=3). The majority of adverse events reported were mild or moderate in severity and not considered to be treatment-related.

“Because the diagnostic odyssey for CTX is typically long and arduous and the complications serious, it is critical that patients are treated quickly following diagnosis to ameliorate or prevent symptoms that can severely impact their lives,” said Robert Steiner, MD, professor, geneticist, and CTX Alliance medical and scientific advisory board member. “These data demonstrate a strong potential to help patients avoid the devastating effects of CTX.”

“CTX is a rare multi-symptom disease that affects each patient very differently, and can significantly impact a person’s quality of life,” said Jean Pickford, executive director, CTX Alliance. “For many patients and their families, the diagnostic journey is challenging and navigating the symptoms is ongoing. Once diagnosed, patients and their families are thrust into the world of a rare disease that can feel isolating and overwhelming. However, we are thrilled to see these impressive data from the RESTORE study. A change in bile alcohols and cholestanol have the potential to reduce the progressive symptoms associated with this rare disease.”

Data from the RESTORE study will be presented at an upcoming scientific congress, including forthcoming results from the open-label pediatric group of patients. In addition, Mirum will be submitting a new drug application to the U.S. FDA in the first half of 2024.

About the RESTORE Phase 3 Study

The Phase 3 RESTORE study is a randomized withdrawal, placebo-controlled clinical trial which evaluated the safety and efficacy of Chenodal in patients with cerebrotendinous xanthomatosis (CTX). Chenodal is administered at 250 mg three times daily in tablet format. The objective of the RESTORE study is to understand how the body responds, as measured by change in blood and urine biomarkers associated with CTX, when treated with Chenodal. The study involved a screening period (4 weeks), four treatment periods (totaling 6 months), and a follow-up phone call (30 days after last dose was administered). The four treatment periods consisted of: an 8-week open-label Chenodal period, a 4-week randomized withdrawal period (placebo or Chenodal), a second 8-week open-label Chenodal period for all patients, and a second 4-week randomized withdrawal period (alternate treatment to first withdrawal period).