MIRM — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-05-06

• 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.

• In October 2024, we completed a license agreement with Enthorin Therapeutics, LLC and Dart Neuroscience LLC granting us the worldwide right to develop and commercialize MRM-3379, an allosteric inhibitor of Phosphodiesterase 4D (“PDE4D”). We are currently enrolling patients in the BLOOM Phase 2 clini…

• On January 23, 2026, we completed the acquisition (the “Bluejay Acquisition”) of Bluejay Therapeutics, Inc. (“Bluejay”) and its lead product candidate brelovitug (BJT-778). We are advancing brelovitug for the treatment of chronic hepatitis D virus (“HDV”) infection. Brelovitug is a fully human IgG1 …

Text removed vs the prior filing · source: 10-Q · 2025-11-04

• On October 22, 2024, we completed a license agreement with Enthorin Therapeutics, LLC and Dart Neuroscience LLC granting us the worldwide right to develop and commercialize MRM-3379, an allosteric inhibitor of Phosphodiesterase 4D (“PDE4D”). We intend to develop MRM-3379 for the treatment of Fragile…

• On August 31, 2023, we completed the acquisition of assets of Travere Therapeutics, Inc. (“Travere”) that are primarily related to the development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of chenodiol and Cholbam (also known as Kolbam) (and togeth…

• The FDA approved Cholbam in March 2015, as 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 peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectr…

• agreements with several rare disease companies for the commercialization of Cholbam and chenodiol in additional countries.

• 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 expect to complete enrollment of the EXPAND study in 2026 with topline data expected in the first half of 2027. In addition, we are advancing our …

Text added vs the prior filing · source: 10-Q · 2026-05-06

• interest rates to have a material effect on our interest rate risk in future reporting periods. For example, a hypothetical change in interest rates of 10% would not have a material impact on the fair market value of our cash equivalents and investments as of March 31, 2026. In addition, we maintain…

• We have outstanding $316.2 million aggregate principal of the Notes as of March 31, 2026. The interest rates on these Notes are fixed and therefore they do not expose us to risk related to changing interest rates. As of March 31, 2026, the approximate fair value of our Notes was $946.3 million.

Text removed vs the prior filing · source: 10-Q · 2025-11-04

• We have outstanding $316.2 million aggregate principal of the Notes as of September 30, 2025. The interest rates on these Notes are fixed and therefore they do not expose us to risk related to rising interest rates. As of September 30, 2025, the approximate fair value of our Notes was $760.9 million…

Text added vs the prior filing · source: 10-Q · 2026-05-06

• On December 19, 2025, we, along with Satiogen Pharmaceuticals, Inc. and Shire Human Genetic Therapies, Inc. as co-plaintiffs, filed four complaints against Sandoz Inc. (“Sandoz”); Annora Pharma Private Limited, Hetero Labs Limited, and Hetero USA Inc. (together, “Hetero”); Zenara Pharma Private Limi…

Text added vs the prior filing · source: 10-Q · 2026-05-06

• To successfully commercialize our approved medicines, we must grow, maintain and appropriately scale our marketing, sales, distribution, managerial and other non-technical capabilities or make arrangements with third parties to perform these services. We have established our own commercial capabilit…

• A trend in the healthcare industry is cost containment. Third-party payors are developing increasingly sophisticated methods of controlling healthcare costs by, among other methods, limiting or preventing (for example via prior authorization, prior therapy or step edit requirements) coverage for par…

• We expect that legislators, policymakers and healthcare insurance funds in the EU Member States will continue to propose and implement cost-containing measures, such as lower maximum prices, lower or lack of reimbursement coverage and incentives to use cheaper, usually generic, products as an altern…

• medicinal products as of 2030. Select high-risk medical devices also came into scope in 2026. It is intended to boost cooperation among EU Member States in assessing health technologies, including new medicines. The Regulation establishes a framework for joint clinical assessments, increasing cooper…

• In clinical trials of volixibat, the most common AEs reported in the volixibat treatment group were mild to moderate gastrointestinal (“GI”) events (diarrhea, abdominal pain, nausea and vomiting) and vitamin D deficiency. In the interim analysis of the PBC VANTAGE study, the incidence of diarrhea in…

Text removed vs the prior filing · source: 10-Q · 2025-11-04

• To successfully commercialize our approved medicines, we must grow, maintain and appropriately scale our marketing, sales, distribution, managerial and other non-technical capabilities or make arrangements with third parties to perform these services. We have established our own commercial capabilit…

• markets and Canada and have entered into a limited number of partner and distributor agreements in other select geographies. We plan to continue to evaluate opportunities to partner with pharmaceutical companies that have established sales and marketing capabilities to commercialize our approved med…

• A trend in the healthcare industry is cost containment. Third-party payors are developing increasingly sophisticated methods of controlling healthcare costs by, among other methods, limiting or preventing (for example via prior authorization, prior therapy or step edit requirements) coverage for par…

• We expect that legislators, policymakers and healthcare insurance funds in the EU Member States will continue to propose and implement cost-containing measures, such as lower maximum prices, lower or lack of reimbursement coverage and incentives to use cheaper, usually generic, products as an altern…

• Additionally, in respect of our approved medicines or if one or more of our product candidates receives marketing approval, and we or others (including regulatory approval authorities) later identify undesirable side effects caused by our approved medicines or such product candidates or other produc…