Latest news with #PFIC
Yahoo
30-04-2025
- Business
- Yahoo
Mirum Pharmaceuticals to Announce First Quarter 2025 Financial Results and Host Conference Call on May 7, 2025
FOSTER CITY, Calif., April 30, 2025--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that it will report first quarter 2025 financial results on Wednesday, May 7, 2025. Mirum will also host a conference call to discuss the first quarter 2025 financial results and recent corporate progress. Conference call details: Wednesday, May 7, 2025 4:30 p.m. ET / 1:30 p.m. PT Dial-in: U.S./Toll-Free: + 1 833 470 1428 International: + 1 404 975 4839 Passcode: 549600 You may also access the call via webcast by visiting the Events & Presentations section on Mirum's website. A replay of this webcast will be available for 30 days. About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). View source version on Contacts Investor Contacts:Andrew McKibbenir@ Media Contact:Erin Murphymedia@ Sign in to access your portfolio
Business Wire
30-04-2025
- Business
- Business Wire
Mirum Pharmaceuticals to Announce First Quarter 2025 Financial Results and Host Conference Call on May 7, 2025
FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that it will report first quarter 2025 financial results on Wednesday, May 7, 2025. Mirum will also host a conference call to discuss the first quarter 2025 financial results and recent corporate progress. Conference call details: 4:30 p.m. ET / 1:30 p.m. PT Dial-in: U.S./Toll-Free: + 1 833 470 1428 International: + 1 404 975 4839 Passcode: 549600 You may also access the call via webcast by visiting the Events & Presentations section on Mirum's website. A replay of this webcast will be available for 30 days. About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI ® (maralixibat) oral solution/LIVMARLI ® (maralixibat) tablets, CHOLBAM ® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X).
Forbes
30-04-2025
- Business
- Forbes
Diversification Strategies For American Expat Investors
Tom Zachystal is president of International Asset Management providing financial planning and investment advice for Americans living abroad. Making the right investments is the key to building long-term wealth. American expat investors, however, face added complexities of global investment opportunities, being subject to both local and U.S. tax laws and regulations, and international currency fluctuations that can affect relative asset values or mean losses when transferring between countries. Given these challenges, deciding where to allocate funds can be overwhelming for overseas-based American investors. While many investors may be aware that diversification is a smart way to manage investment risk and build wealth long term, in this article, I'll discuss strategies Americans living abroad might consider when seeking to diversify their portfolios. Diversification in investing refers to reducing the risk of losses by holding different asset classes such as stocks, bonds and real estate, or holding assets in different countries or regions. A well-balanced portfolio helps protect against downturns in any one sector or country. Expat investors can still invest in the U.S., but also often discover local investment opportunities abroad. They then have to weigh whether it makes more sense for them to invest locally or to invest in global shares or funds in the U.S. This may depend on where their funds are located, and where they plan to be living when they will sell or draw on the investments, to minimize losses from transferring funds between currencies. There are other rules that may be limiting, such as EU rules that may prevent residents in the EU from buying most U.S. ETFs and mutual funds. Nonetheless, the principle stands that investors should seek to diversify across different regions, countries and asset classes. Expats often face the challenge of fluctuating exchange rates. To manage this, you might consider diversifying your holdings across multiple currencies. While stable currencies such as the U.S. dollar, euro and British pound offer security, emerging market currencies can bring higher returns but can come with risks such as inflation and devaluation. Focus on stability by investing in assets that align with your financial goals and future plans. Foreign currency accounts and globally diversified ETFs can also help hedge against currency risk, keeping your investments balanced and diversified. As an American expat, you're subject to U.S. taxes no matter where you live, so your investments must consider both U.S. tax rules as well as your host country's laws. Investing in foreign mutual funds may trigger IRS Passive Foreign Investment Company (PFIC) rules, resulting in higher taxes and complex reporting. To avoid this, you can stick to U.S.-based ETFs or buy individual stocks. Tax treaties can help, too, in some cases offering preferential treatment for U.S. retirement accounts such as IRAs and 401(k)s and reducing foreign withholding taxes on dividends. Consult with both a financial advisor and tax professional with experience working with expats in your host country to optimize your taxes and investment returns. Real estate offers many expats a tangible, diversified investment with the potential for steady income and a hedge against inflation. If you plan to stay in your host country long-term, then investing in local property can potentially be a smart move. However, local property laws, taxes and financing can be complex, so seek advice. For a more passive approach, you can consider real estate investment trusts (REITs). These allow you to invest in global real estate without the burden of property management. Before committing, make sure to research the local market conditions and understand the tax implications, making sure to consider any local property, capital gains and wealth taxes. Diversification isn't just about the assets you own; it's also about where you hold them. As an expat, you can still hold U.S.-based brokerage and retirement accounts. However, many U.S. brokerage firms won't deal with non-U.S. residents, so you may need to change your broker when you move abroad or risk your account being restricted or closed. Having local retirement and investment accounts can avoid currency exchange costs and can provide local tax benefits. Combining both can be a useful way to diversify and maximize local and U.S. tax perks. Market shifts can cause a portfolio's asset allocation to drift over time. If one sector, such as international equities, outperforms, it could, over time, make up a larger portion of your overall investment portfolio, increasing exposure to a single asset class or region. Rebalancing is key to keeping your investments on track with your long-term financial objectives. A good rule of thumb is to review your portfolio annually. Regular adjustments help keep your portfolio aligned with your risk tolerance and goals. As an expat, you have both increased global opportunities, but also new challenges as an investor. By spreading investments across regions, currencies and asset classes, you can manage risk while capitalizing on growth opportunities both in the U.S. and overseas. The key to success is staying proactive and regularly reviewing and adjusting your portfolio, as needed. It's also important to work with a financial advisor who specializes in cross-border investing and will help you align your investments with your financial goals. With the right strategies, you'll position yourself for long-term success, no matter where in the world you live. The information provided here is not investment, tax or financial advice. You should consult with a licensed professional for advice concerning your specific situation.
Forbes
23-04-2025
- Business
- Forbes
Foreign Information Returns & The IRS: When Time Limits Never Expire
IRS audits Generally, the IRS has three years to select an income tax return for audit. But if the taxpayer never files an income tax return, the three-year statute of limitations period doesn't start, providing the agency with unlimited time to make additional tax adjustments. Although many taxpayers are familiar with this rule, few taxpayers recognize that a similar rule applies to unfiled foreign information tax returns. Because there are numerous foreign information return obligations under federal tax law, it is fairly easy and even common for taxpayers to miss a filing deadline. In these instances, the statute of limitations for the IRS to make adjustments remains open indefinitely until the taxpayer files the information return and for three years thereafter. This extended statute of limitations period applies even if the taxpayer properly and timely filed an income tax return, although the extent of items subject to adjustment depends on whether the taxpayer had reasonable cause for the delinquent foreign information return. Not all foreign information returns are subject to this extended statute of limitations rule. Specifically, section 6501(c)(8) only identifies eight information returns that extend the audit period, which include: (i) IRS Form 8621; (ii) IRS Form 5471; (iii) IRS Form 8865; (iv) IRS Form 926; (v) IRS Form 8938; (vi) IRS Form 3520 (but only for foreign trust reporting); (vii) IRS Form 3520-A; and (viii) IRS Form 5472. A summary of these foreign information returns follows. U.S. persons with interests in passive foreign investment companies (PFICs) must generally file an IRS Form 8621, Information Return by a Shareholder of a Passive Foreign Investment Company or Qualified Electing Fund. PFICs are foreign corporations that meet either an income test or an asset test. Under the income test, a foreign corporation qualifies as a PFIC if 75% or more of its gross income in a tax year originates from passive income (e.g., interest, dividends, etc.). A foreign corporation can also qualify as a PFIC under the asset test if at least 50% of its assets produce passive income or are held for the production of passive income. Under these tests, many foreign mutual funds qualify as PFICs. U.S. persons with ownership or other interests in a foreign corporation must often file an IRS Form 5471, Information Return of U.S. Persons With Respect to Certain Foreign Corporations. The reporting requirements for an IRS Form 5471 are some of the more complex foreign information reporting obligations under the Code as U.S. persons must determine whether they fall under one or more reporting categories (each of which requires the disclosure of certain information). A common IRS Form 5471 reporting obligation arises when a U.S. person has 'control' over a foreign corporation—i.e., more than 50% of the vote or value of the corporation's stock. A lesser-known reporting category relates to U.S. persons who are officers or directors of a foreign corporation when a U.S. person acquires 10% or more of the corporation's stock in a tax year. For more information on IRS Form 5471, see here. There is also a foreign information return requirement for U.S. persons who have ownership or other interests in foreign partnerships. Similar to the IRS Form 5471 reporting requirements, the IRS Form 8865, Return of U.S. Persons With Respect to Certain Foreign Partnerships, must be filed if a U.S. person falls within one or more reporting categories. For example, a U.S. person is a category one filer if the person controls the foreign partnership at any time during the partnership's tax year. For these purposes, control means more than 50% of the profits or capital interests of the partnership or 50% of the deductions or losses. In addition, U.S. persons must file an IRS Form 8865 if they make certain contributions to foreign partnerships in a tax year, provided the person has at least a 10% interest in the partnership and meets a specified dollar threshold. U.S. persons who make certain contributions to foreign corporations or who enter into reorganization transactions with a foreign corporation party must file IRS Form 926, Return by a U.S. Transferor of Property to a Foreign Corporation. For more information on IRS Form 926, see here. U.S. persons with interests in 'specified foreign financial assets' must usually file an IRS Form 8938, Statement of Specified Foreign Financial Assets. Generally, a 'specified foreign financial asset' includes: (i) foreign financial accounts (e.g., banking or investment accounts); (ii) ownership in foreign entities (e.g., partnerships and corporations); (iii) debt issued by foreign persons; and (iv) interests in foreign trusts and estates. For more information on IRS Form 8938, see here. U.S. persons who make contributions to or receive distributions from a foreign trust must report these transactions on IRS Form 3520, Annual Return to Report Transactions With Foreign Trusts and Receipt of Certain Foreign Gifts. In addition, U.S. persons who are treated as grantors or deemed owners of foreign trusts must file an IRS Form 3520-A, Annual Information Return of Foreign Trust with a U.S. Owner. For more information on IRS Form 3520, see here For more information on IRS Form 3520-A, see here. Certain corporations (domestic and foreign) and certain foreign-owned U.S. disregarded entities must file an IRS Form 5472, Information Return of a 25% Foreign-Owned U.S. Corporation or a Foreign Corporation Engaged in a U.S. Trade or Business. For example, a U.S. corporation with at least one foreign person who owns 25% or more of the corporation's vote or value must file an IRS Form 5472. For more information on IRS Form 5472, see here. Taxpayers who fail to timely file any one or more of these foreign information returns are subject to extended statute of limitations periods. Worse yet, the statute of limitations period for the IRS to conduct an audit never expires if the taxpayer fails to file the applicable foreign information return. Taxpayers interested in regaining compliance should consult with a tax professional as there are IRS programs available that may provide relief for delinquent foreign information returns, including the Streamlined Filing Compliance Procedures.
Yahoo
27-03-2025
- Health
- Yahoo
LIVMARLI Now Approved in Japan for ALGS and PFIC
- LIVMARLI is the first and only treatment available for cholestatic pruritus in patients with Alagille syndrome and Progressive Familial Intrahepatic Cholestasis in Japan FOSTER CITY, Calif., March 27, 2025--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that its partner, Takeda Pharmaceutical Company Limited, has received approval by the Japanese Ministry of Health, Labour, and Welfare for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) in Japan. "We are thrilled to see LIVMARLI approved as the first and only medication available in Japan for patients living with pruritus related to ALGS and PFIC," said Chris Peetz, chief executive officer at Mirum. "Takeda is a leading global pharmaceutical company with demonstrated success in the development and commercialization of medicines for rare diseases. Under Takeda's leadership, we are confident that LIVMARLI could have a meaningful impact on the lives of patients with ALGS and PFIC in Japan." Mirum and Takeda entered into an exclusive license agreement in September 2021 for the development and commercialization of LIVMARLI for rare cholestatic pediatric liver diseases in Japan. Under the terms of the agreement, Mirum is eligible to receive a percentage of Takeda's annual net sales. LIVMARLI is currently approved for the treatment of pruritus related to ALGS in more than 40 countries and for PFIC in more than 30 countries worldwide. About LIVMARLI® (maralixibat) oral solution LIVMARLI® (maralixibat) oral solution is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration and European Medicines Agency for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum's clinical trials section on the company's website. U.S. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including: Liver injury. Changes in certain liver tests are common in patients with Alagille syndrome and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you. A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. US Prescribing Information EU SmPC Canadian Product Monograph About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is also initiating the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the impact of LIVMARLI on patients in Japan, the ability of Takeda to successfully commercialize in the territory and the size of the Japanese market for LIVMARLI. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "will," "could," "can," "would," "potential," "hope," "opportunity," and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of macroeconomic and geopolitical developments, and the other risks described in Mirum's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. A further description of risks and uncertainties can be found in Mirum's Quarterly Report on Form 10-K for the quarter ended December 31, 2024 and subsequent filings with the U.S. Securities and Exchange Commission and available at View source version on Contacts Media Contact:Erin Murphymedia@ Investor Contact:Andrew McKibbenir@
