FOSTER CITY, Calif. - In a significant development for patients with primary biliary cholangitis (PBC), the U.S. Food and Drug Administration (FDA) has awarded Breakthrough Therapy Designation to Mirum Pharmaceuticals (NASDAQ:MIRM)' volixibat. This announcement follows a positive interim analysis from the Phase 2b VANTAGE study, which demonstrated a statistically significant improvement in cholestatic pruritus, a symptom of PBC, compared to placebo.
The designation is intended to expedite the development and review of drugs for serious or life-threatening conditions when preliminary clinical evidence suggests a substantial improvement over existing therapies. Volixibat, an oral medication that selectively inhibits the ileal bile acid transporter (IBAT), is designed to reduce systemic bile acids by preventing their recirculation. This could represent a novel treatment approach for adult cholestatic diseases.
The interim results from the VANTAGE study, released in June, showed not only an improvement in pruritus but also meaningful reductions in serum bile acids and improvements in fatigue among patients treated with the drug. The study reported no new safety concerns, with diarrhea being the most common adverse event, described as mild to moderate in severity.
Mirum Pharmaceuticals, a biopharmaceutical company focused on rare diseases, has three approved medications to its credit, including LIVMARLI for the treatment of cholestatic pruritus in Alagille syndrome and PFIC patients, CHOLBAM for certain bile acid synthesis disorders and peroxisomal disorders, and CHENODAL for cerebrotendinous xanthomatosis.
The company's pipeline features volixibat, which is also under evaluation for primary sclerosing cholangitis in the Phase 2 VISTAS study. Additionally, Mirum is engaged in a Phase 3 study, EXPAND, to explore further the potential of LIVMARLI in additional cholestatic pruritus settings.
The full results of the VANTAGE study are expected to be available following the completion of enrollment in 2026. This announcement is based on a press release statement from Mirum Pharmaceuticals.
In other recent news, Mirum Pharmaceuticals has made significant strides in its product offerings. The U.S. Food and Drug Administration (FDA) has approved an expanded indication for the company's drug, LIVMARLI® (maralixibat) oral solution. This label expansion permits the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) aged 12 months and older, following the promising results of the MARCH Phase 3 study.
Furthermore, Mirum Pharmaceuticals reported a strong financial performance with total net product revenue reaching $69 million in the first quarter of 2024, with LIVMARLI contributing $43 million. The company's revenue forecast for 2024 is projected to be between $310 million and $320 million. Analyst firms JMP Securities, Stifel, and H.C. Wainwright have shown confidence in the company's performance, with JMP Securities estimating a revenue of $316 million.
In more recent developments, Mirum Pharmaceuticals received European Union approval for LIVMARLI for PFIC patients aged three months and older, expected to broaden the drug's U.S. label. Additionally, the company has submitted a supplemental New Drug Application for a higher concentration formulation of LIVMARLI, targeting its use for younger patients. These developments underscore Mirum Pharmaceuticals' commitment to advancing treatments for rare diseases.
InvestingPro Insights
Mirum Pharmaceuticals' recent FDA Breakthrough Therapy Designation for volixibat aligns with the company's strong revenue growth trajectory. According to InvestingPro data, Mirum's revenue grew by an impressive 128.34% over the last twelve months as of Q2 2023, with quarterly revenue growth of 107.68% in Q2 2023. This robust growth reflects the company's success in bringing innovative treatments to market for rare diseases.
Despite the positive news and strong revenue growth, InvestingPro Tips highlight that Mirum is not currently profitable, with a negative operating income of $108.43 million over the last twelve months. This is not uncommon for biopharmaceutical companies investing heavily in research and development. However, the company's liquid assets exceed short-term obligations, suggesting a stable financial position to support ongoing clinical trials and potential commercialization of volixibat.
The market appears to be recognizing Mirum's potential, as evidenced by the 47.68% price increase over the past six months. This aligns with the company's pipeline progress and recent regulatory milestones. For investors seeking more comprehensive analysis, InvestingPro offers 8 additional tips for Mirum Pharmaceuticals, providing deeper insights into the company's financial health and market position.
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