Mirum Pharmaceuticals Q4 Earnings Call Highlights

25 Feb 2026, 17:24 by Amy Steele · The Cerbat Gem

Mirum Pharmaceuticals (NASDAQ:MIRM) used its fourth-quarter and full-year 2025 earnings call to outline continued commercial momentum, provide 2026 sales guidance, and preview a busy slate of upcoming clinical catalysts across its cholestatic liver disease franchise and newly added hepatitis delta virus (HDV) program.

2025 sales topped guidance; 2026 outlook calls for continued growth

Chief Executive Officer Chris Peetz said 2025 was “a year of disciplined execution and growth,” with net product sales of $521 million, above the upper end of the company’s guidance range. Peetz broke out 2025 performance as $245 million in U.S. LIVMARLI sales, $115 million internationally, and $161 million from the company’s bile acid medicines.

Management attributed the “strong finish” to continued leadership in Alagille syndrome, accelerating PFIC uptake, and growing demand internationally. For 2026, Mirum guided to $630 million to $650 million in net product sales.

During the Q&A, management said the company’s growth outlook is “definitely more LIVMARLI driven.” Chief Financial Officer Eric Bjerkholt also noted that Japan revenue in 2025 included $22 million related to inventory buildup, which is expected to pressure reported Japan revenue in 2026 even as the launch “is going as expected.” The bile acid portfolio is expected to continue growing, but at a steadier pace than LIVMARLI.

Pipeline updates: multiple potentially registrational readouts ahead

Mirum highlighted several clinical and regulatory milestones achieved in 2025, including approval of Ctexli for CTX, approval of a tablet formulation of LIVMARLI, completion of enrollment in the VISTA study of volixibat in primary sclerosing cholangitis (PSC), and the addition of the phase 3 brelovitug program for chronic HDV via the Bluejay acquisition.

Peetz said the company now expects four potentially registrational clinical readouts over the next 18 months. He added that integration of the Bluejay transaction has progressed smoothly and that the combination is expected to create operating leverage alongside a potential volixibat launch.

Volixibat in PSC: pruritus endpoint and FDA alignment

Chief Medical Officer Joanne Quan said Mirum expects to report top-line VISTA data in PSC in the second quarter of 2026. The primary endpoint, aligned with the FDA, is pruritus. The study will also evaluate safety, changes in certain bile acids, other symptoms, and quality-of-life measures. Quan reminded listeners that the trial exceeded a pre-specified efficacy threshold at a blinded interim analysis in 2024 and continued with a selected 20 mg twice-daily dose.

In response to questions about variability of itch in PSC, management said the company is enrolling patients with persistent pruritus and tracking pruritus response throughout the study. Quan referenced prior work indicating a high proportion of PSC patients report pruritus and fatigue and that about half reported disruption of daily life activities.

Mirum also addressed regulatory planning for volixibat. Peetz said the company expects the current VISTA PSC study to provide a sufficient safety database for the setting, based on prior FDA interactions. After top-line data, Mirum expects to meet with FDA on the submission plan and said it believes it can submit in the second half of the year.

On commercialization, Peetz said pricing considerations for volixibat are still being evaluated. While PPARs in PBC were described as a useful benchmark for planning, he emphasized the company will make final decisions closer to launch and with data in hand, noting that PSC has no approved medicines, which could shape positioning.

Brelovitug in HDV: interim and top-line data expected in 2026

Quan said all four studies in the AZURE program are progressing well. The company expects interim results from the phase 2b portion of AZURE-1 in the second quarter, based on the first 50 patients evaluated at the week 24 time point. AZURE-1 is studying treatment-naive HDV patients randomized to brelovitug or delayed treatment and uses a 24-week composite endpoint of virologic response and ALT normalization, which management said is aligned with FDA.

AZURE-1 and AZURE-4, described as the two phase 3 studies intended to form the basis of an FDA registration package, are expected to complete enrollment soon, with 24-week top-line data anticipated in the second half of 2026. Quan added that AZURE-2 and AZURE-3 are active-controlled studies designed to support European registration and provide additional long-term safety and efficacy data.

In discussing the competitive landscape, Peetz said there is currently nothing specifically labeled for HDV in the U.S., while Hepcludex is approved in Europe and “has been performing well.” He said Mirum expects the landscape to evolve, citing the potential for U.S. review of Hepcludex and other approaches in development. Peetz said the company is encouraged by brelovitug’s profile as a potential best-in-class single agent regimen, citing “impressive response rates” and an “attractive safety profile,” including a “100% viral response at week 48” and “65%–82% composite endpoint.”

Expenses, cash position, and R&D investment plans

Bjerkholt reported fourth-quarter 2025 net product sales of $149 million, up from $99 million in the prior-year quarter. For the full year, net product sales rose to $521 million from $336 million, representing 55% year-over-year growth.

Total operating expense was $153 million for the quarter and $543 million for the year, including:

$186 million in R&D
$257 million in SG&A
$100 million in cost of sales

The company reported $95 million of non-cash expenses for the year, including stock-based compensation and intangible amortization; $24 million of intangible amortization and other non-cash items were reflected in cost of sales.

Mirum ended 2025 with $391 million in cash, cash equivalents, and investments, up from $293 million at the end of 2024. Bjerkholt also said Mirum completed two private placements concurrent with the Bluejay acquisition that generated aggregate gross proceeds of $268.5 million, which he said effectively covered the cash outlay for the transaction. The company achieved positive cash flow from operations in 2025.

Looking ahead, Mirum expects R&D expense to increase in 2026, driven primarily by brelovitug clinical investments and CMC work to support an anticipated BLA submission next year. Bjerkholt said the increase is fully funded and that the company expects a return to positive cash flow in 2027. On the magnitude of investment, he estimated roughly a $150 million increase in R&D tied to brelovitug, with about half related to CMC, adding that strong enrollment is compressing expenses into 2026.

In closing remarks, Peetz reiterated that the company enters 2026 with commercial momentum, a strong financial position, and multiple near-term data readouts that could support new indications and products in rare disease settings.

About Mirum Pharmaceuticals (NASDAQ:MIRM)

Mirum Pharmaceuticals, Inc is a late-stage biopharmaceutical company dedicated to the development and commercialization of innovative therapies for rare cholestatic liver diseases. The company’s primary focus lies in addressing the unmet medical needs of patients suffering from genetic and progressive forms of pediatric liver disorders, where limited treatment options currently exist.

Mirum’s lead product candidate, maralixibat (Livmarli), is an ileal bile acid transporter inhibitor designed to reduce systemic bile acid accumulation and alleviate associated pruritus and liver damage.