Mirum Pharmaceuticals, Inc. MIRM announced disappointing top-line results from the mid-stage EMBARK study.
The phase IIb EMBARK study is a randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of lead drug Livmarli (maralixibat) oral solution in patients with biliary atresia who have undergone a Kasai surgery.
Patients enrolled in the study were 21 days old or more but less than three months old at the time of the Kasai surgery and had a clinical diagnosis of biliary atresia. These patients underwent a Kasai surgery within three weeks prior to randomization in EMBARK. The primary endpoint was the mean change in total bilirubin through week 26, followed by secondary endpoints, including the mean change in total serum bile acids and the proportion of patients who required liver transplants or had another liver-related event over the 26 weeks.
The study did not meet its primary endpoint of mean change in bilirubin from baseline to week 26 or the key secondary endpoints.
The failure of the study disappointed investors and consequently, shares tanked 15.7%.
On a year-to-date basis, shares have gained 53% compared with the industry’s decline of 18%.
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Livmarli, an IBAT inhibitor, is already approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States. (three months and older), in Europe (two months and older) and in Canada.
Mirum is also seeking label expansion of the drug in the United States for the indication of cholestatic pruritus in progressive familial intrahepatic cholestasis (“PFIC”) patients three months of age and older and Europe in PFIC for patients two months of age and older.
A supplemental new drug application for Livmarli in PFIC is under review in the United States, with a target action date of Mar 13, 2024.
Apart from Livmarli, the company also has two other approved drugs in its portfolio, Cholbam (cholic acid) capsules and Chenodal (chenodiol) tablets, which were added after Mirum completed the acquisition of all the assets of Travere Therapeutics’ TVTX bile acid products in August 2023.
Mirum paid an upfront purchase price of $210.4 million in cash to Travere and may pay additional amounts of up to $235.0 million upon the achievement of certain milestones based on specified amounts of net annual sales of the bile acid products.
Cholbam is the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisome biogenesis disorder-Zellweger spectrum disorder. Chenodal is approved for the treatment of radiolucent stones in the gallbladder and has received medical necessity recognition by the FDA for the treatment of cerebrotendinous xanthomatosis (“CTX”).
In October 2023, Mirum reported positive top-line data from the RESTORE clinical trial evaluating Chenodal in patients with CTX and plans to submit a new drug application for Chenodal to the FDA in the first half of 2024.
Mirum has another candidate in its pipeline, volixibat, an oral IBAT inhibitor, for the treatment of primary sclerosing cholangitis (“PSC”) and primary biliary cholangitis (“PBC”). Two separate phase IIb studies, VISTAS and VANTAGE, are currently being conducted on volixibat in the PSC and PBC indications, respectively. Interim analysis results from both studies are expected in the first half of 2024.
Zacks Rank and Stocks to Consider
Mirum Pharma currently has a Zacks Rank #3 (Hold).
A couple of better-ranked stocks in the overall healthcare sector are Entrada Therapeutics TRDA and Dynavax Technologies DVAX. TRDA sports a Zacks Rank #1 (Strong Buy) and DVAX carries a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Entrada’s loss per share estimate for 2023 has narrowed from $2.07 to 9 cents in the past 60 days. The same for 2024 has narrowed from $2.35 to $2.04 during the same time frame.
Dynavax’s loss per share estimate for 2023 has narrowed from 23 cents to 12 cents in the past 30 days. Earnings estimate for 2024 rose from 3 cents to 18 cents during the same period. Shares of DVAX have risen 28.2% year to date.
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