FOSTER CITY, Calif. - Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) recently announced the publication of the Phase 3 MARCH-PFIC study results in The Lancet Gastroenterology and Hepatology, highlighting the efficacy and safety of LIVMARLI® (maralixibat) oral solution for patients with progressive familial intrahepatic cholestasis (PFIC). The study is the largest of its kind, being randomized, double-blind, and placebo-controlled.
According to the study, LIVMARLI showed significant benefits for patients with a broad range of genetic PFIC types. The main efficacy analyses included two groups: the BSEP deficiency cohort and the All-PFIC cohort, which encompassed various genetic deficiencies associated with the disease.
The primary efficacy endpoint of mean change in pruritus severity score and the key secondary endpoint of mean change in total serum bile acid concentration were both met with significant improvements.
These improvements began as early as Week 2 and were sustained over the 26-week study period. Additional observed benefits included better bilirubin concentrations, growth, and sleep disturbances when compared to the placebo.
The safety profile of LIVMARLI was consistent throughout the study, with diarrhea being the most common adverse event, generally described as mild and transient.
Dr. Alexander Miethke from Cincinnati Children's Hospital and lead author on the publication emphasized the potential of LIVMARLI to significantly improve pruritus and other parameters impacting PFIC patients. He also noted the potential for improvements in native liver survival, given the reductions in serum bile acid and bilirubin levels.
Pam Vig, Ph.D., chief scientific officer and head of research at Mirum, expressed satisfaction with the publication of the data, underscoring the significant impact LIVMARLI could have on clinical signs and symptoms seen in PFIC patients.
LIVMARLI is an ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and PFIC. It has also been approved by the European Commission and Health Canada for similar indications and is currently under review for approval in Europe for PFIC patients two months of age and older.
The medication has received Breakthrough Therapy designation for ALGS and PFIC type 2, as well as orphan designation for these conditions. Mirum has a late-stage pipeline that includes other investigational treatments for liver diseases.
The publication of the MARCH-PFIC study results provides evidence for the therapeutic benefit of LIVMARLI across a range of PFIC types, offering a non-surgical option for improving outcomes in patients with this severe cholestatic condition. This information is based on a press release statement from Mirum Pharmaceuticals.
InvestingPro Insights
As Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) continues to make strides in the treatment of PFIC with their drug LIVMARLI, the financial perspective of the company is equally important for investors considering the stock. According to InvestingPro, analysts have tempered their expectations for the company's earnings in the upcoming period, signaling caution as the company strives for profitability. Furthermore, while Mirum is not expected to be profitable this year, it's noteworthy that the company's liquid assets surpass its short-term obligations, providing a degree of financial stability.
InvestingPro Data indicates that Mirum has a market cap of approximately $1.24 billion USD, reflecting investor confidence in its potential despite the lack of current profitability, as indicated by a negative P/E ratio of -6.47. The company's revenue has shown impressive growth over the last twelve months as of Q4 2023, with a 141.85% increase, which may be indicative of the market's positive reception to its products like LIVMARLI. However, it's essential to note that Mirum operates with a moderate level of debt and does not pay a dividend to shareholders, which could be relevant factors for certain investment strategies.
For investors seeking a deeper analysis, there are additional InvestingPro Tips available for Mirum Pharmaceuticals, which can be accessed at InvestingPro. These tips provide insights that could help in making a more informed investment decision. To enhance your InvestingPro experience, use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription. With this offer, investors can gain access to valuable metrics and insights on companies like Mirum Pharmaceuticals.
It's worth mentioning that the fair value of Mirum's stock, as estimated by analysts, stands at $48 USD, while InvestingPro's fair value assessment is $30.86 USD, suggesting there may be room for growth or a need for reassessment depending on the company's future performance and market conditions. The next earnings date slated for May 8, 2024, will be a critical time for investors to watch for potential shifts in the company's financial trajectory.
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