MIRM 最新10-Q变化

相对上期新增的文字 · 来源:10-Q · 2026-11-03

• We are a biopharmaceutical company dedicated to transforming the treatment of rare diseases. Our strategy is to acquire, develop and commercialize medicines for rare diseases, with a focus on driving commercial growth and building a diversified portfolio with meaningful value creation potential. We …

• Our commercial organization is focused on two areas, rare liver diseases and rare genetic diseases, with dedicated field teams serving each business. Our rare liver disease business is currently our larger commercial business and is led by LIVMARLI® (maralixibat) (“Livmarli”). Our rare genetic disea…

• Livmarli (maralixibat) is a novel, orally administered, minimally-absorbed ileal bile acid transporter inhibitor (“IBATi”) that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (“ALGS”) in the United States (“U.S.”), the European Union (“EU”) and various other…

• In addition, we are developing maralixibat for certain rare cholestatic conditions through the Phase 3 EXPAND study. We announced topline results from EXPAND in October 2026 demonstrating a statistically significant improvement in pruritus compared with placebo in the primary analysis population of …

• We are advancing our product candidate, volixibat, a novel, oral, minimally-absorbed IBATi, for the treatment of cholestatic pruritus in patients with primary sclerosing cholangitis (“PSC”) and primary biliary cholangitis (“PBC”), for which the U.S. Food and Drug Administration (“FDA”) has granted B…

相对上期删除的文字 · 来源:10-Q · 2026-08-05

• We are a biopharmaceutical company dedicated to transforming the treatment of rare diseases. We have three approved medicines: LIVMARLI® (maralixibat) (“Livmarli”), Cholbam® (cholic acid) capsules (“Cholbam”), and CTEXLI® (chenodiol) tablets (“Ctexli”).

• Livmarli is a novel, orally administered, minimally-absorbed ileal bile acid transporter (“IBAT”) inhibitor (“IBATi”) that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (“ALGS”) in the United States (“U.S.”), the European Union (“EU”) and various other coun…

• We market and commercialize Ctexli and Cholbam (also known as Kolbam) (and together with chenodiol, the “Bile Acid Medicines”) in the U.S. directly and indirectly in certain foreign markets. The U.S. Food and Drug Administration (the “FDA”) approved Cholbam in March 2015, as the first FDA-approved t…

• We are advancing our product candidate, volixibat, a novel, oral, minimally-absorbed agent designed to inhibit IBAT, for the treatment of adult patients with cholestatic liver diseases. We are developing volixibat in the setting of primary sclerosing cholangitis (“PSC”) and primary biliary cholangit…

• In addition, we are developing Livmarli for certain rare cholestatic conditions through the Phase 3 EXPAND study, which we initiated in the fourth quarter of 2024. We completed enrollment of the EXPAND study in the first half of 2026 with topline data expected in the fourth quarter of 2026.

相对上期新增的文字 · 来源:10-Q · 2026-11-03

• Our cash, cash equivalents and investments as of September 30, 2026 consist of readily available checking and money market funds and investments. The primary objective of our investment activities is to preserve our capital to fund operations. We may invest in highly liquid and high-quality governme…

• We have outstanding $59.0 million and $690.0 million aggregate principal of the 2029 Notes and 2032 Notes, respectively, as of September 30, 2026. The interest rate on the 2029 Notes is fixed and therefore it does not expose us to risk related to changing interest rates. The 2032 Notes do not bear i…

• Our operations include activities in the U.S., the Netherlands, Switzerland and certain other countries. As a result, our financial results may be affected by factors such as changes in foreign currency exchange rates or weak economic conditions in the foreign markets in which we sell our products. …

• Based on our overall foreign currency denominated exposures as of September 30, 2026, we believe that a near-term 10% fluctuation of the USD exchange rate could result in a potential change in the fair value of our net assets and liabilities denominated in foreign currency by approximately $7.5 mill…

相对上期删除的文字 · 来源:10-Q · 2026-08-05

• Our cash, cash equivalents and investments as of June 30, 2026 consist of readily available checking and money market funds and investments. The primary objective of our investment activities is to preserve our capital to fund operations. We may invest in highly liquid and high-quality government an…

• exposure to market risk is interest income sensitivity, which is affected by changes in the general level of U.S. interest rates. Due to the strategies we employ (including the short-term nature of the instruments in our portfolio and the low risk profile of our investments), as of the date of this …

• We have outstanding $79.0 million and $690.0 million aggregate principal of the 2029 Notes and 2032 Notes, respectively, as of June 30, 2026. The interest rate on the 2029 Notes is fixed and therefore it does not expose us to risk related to changing interest rates. The 2032 Notes do not bear intere…

• Our operations include activities in the U.S., the Netherlands, Switzerland and certain other countries in Europe. As a result, our financial results may be affected by factors such as changes in foreign currency exchange rates or weak economic conditions in the foreign markets in which we sell our …

• Based on our overall foreign currency denominated exposures as of June 30, 2026, we believe that a near-term 10% fluctuation of the USD exchange rate could result in a potential change in the fair value of our net assets and liabilities denominated in foreign currency by approximately $5.4 million. …

相对上期新增的文字 · 来源:10-Q · 2026-11-03

• candidates, if approved, is unavailable or limited in scope or amount, our revenues from sales by us or our partners and the potential profitability of our approved medicines or any future product candidates, if approved, in those countries would be negatively affected.

• In clinical studies of brelovitug, the most common treatment-related AEs were injection-related events and cases of flu-like illness that were mild to moderate in severity. There were no treatment-related serious adverse events (“SAEs”), Grade ≥3 AEs, or AEs leading to study discontinuation.

• In clinical studies of Atebrioz, the most common adverse reactions were headache, arthralgia, upper respiratory tract infection, epistaxis and nausea. Most AEs were mild or moderate in severity, and no AEs led to treatment discontinuation or dose reduction.

• expensive and time consuming and could have a material adverse effect on our business, results of operations and financial condition. In addition, in the event that the CMS were to terminate our rebate agreement, no federal payments would be available under Medicaid or Medicare for our covered outpa…

• design to address these considerations, the FDA, European Commission or comparable foreign regulatory authorities may not approve our product candidates.

相对上期删除的文字 · 来源:10-Q · 2026-08-05

• marketing approval, any of which may adversely impact our business, financial condition, operating results and prospects.

• In clinical studies of brelovitug, the most common treatment-related AEs were injection-related events that resolved within 1-2 days, and cases of flu-like illness that were mild-moderate in severity.

• problems with a product, such as AEs of unanticipated severity or frequency, or problems with the facility where, or processes by which, the product is manufactured, such events may result in, among other things:

• Prior to obtaining approval to commercialize a product candidate in the U.S. or internationally, we must demonstrate with substantial evidence from adequate and well-controlled clinical trials, and to the satisfaction of the FDA, EMA or comparable foreign regulatory authorities, that such product ca…

• to market our product candidates, which would significantly harm our business, financial condition, results of operations and prospects.