Baricitinib

Immunology R&D News - Updated indication allows the use of RINVOQ® (upadacitinib) prior to the use of tumor necrosis factor (TNF) blocking agents in patients for whom use of these treatments is clinically inadvisable and who have received at least one approved systemic therapy NORTH CHICAGO, Ill., Oct. 13, 2025 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced the U.S. Food and Drug Administration (FDA) approval of a supplemental new drug application (sNDA) that updates the indication statement for RINVOQ® (upadacitinib) for the treatment of adults with moderately to severely active ulcerative colitis (UC) and moderately to severely active Crohn's disease (CD). Previously, RINVOQ was indicated for adults with moderately to severely active UC or CD who had an inadequate response or intolerance to one or more tumor necrosis factor (TNF) blockers. The updated indication statement also allows the use of RINVOQ for patients after they have received at least one approved systemic therapy in the event TNF blockers are clinically inadvisable. "At AbbVie, we are committed to addressing the ongoing needs of patients living with inflammatory bowel disease," said Kori Wallace, M.D., Ph.D., vice president, global head of immunology clinical development, AbbVie. "Ulcerative colitis and Crohn's disease can impact every aspect of a patient's life. This label update gives healthcare providers the option to prescribe RINVOQ for patients with moderately to severely active inflammatory bowel disease after the use of one approved systemic therapy if TNF blockers are deemed clinically inadvisable by the prescribing physician." RINVOQ SAFETY CONSIDERATIONS RINVOQ may cause serious side effects, including:Serious infections. RINVOQ can lower ability to fight infections. Serious infections, some fatal, occurred, including tuberculosis (TB) and infections caused by bacteria, fungi, or viruses. Increased risk of death in people age 50+ with at least 1 heart disease risk factor. Cancer and immune system problems. Increased risk of some cancers, including lymphoma and skin. Current or past smokers have higher risk for lymphoma and lung cancer. Increased risk of major cardiovascular events such as heart attack, stroke, or death in people 50+ with at least 1 heart disease risk factor, especially in current or past smokers. Blood clots, some fatal, in veins of the legs or lungs and arteries. This occurred more often in people 50+ with at least 1 heart disease risk factor. Serious allergic reactions. Do not take if allergic to RINVOQ or its ingredients. Tears in the stomach or intestines; changes in certain laboratory test results. Serious infections. RINVOQ can lower ability to fight infections. Serious infections, some fatal, occurred, including tuberculosis (TB) and infections caused by bacteria, fungi, or viruses. Increased risk of death in people age 50+ with at least 1 heart disease risk factor. Cancer and immune system problems. Increased risk of some cancers, including lymphoma and skin. Current or past smokers have higher risk for lymphoma and lung cancer. Increased risk of major cardiovascular events such as heart attack, stroke, or death in people 50+ with at least 1 heart disease risk factor, especially in current or past smokers. Blood clots, some fatal, in veins of the legs or lungs and arteries. This occurred more often in people 50+ with at least 1 heart disease risk factor. Serious allergic reactions. Do not take if allergic to RINVOQ or its ingredients. Tears in the stomach or intestines; changes in certain laboratory test results. For more information about RINVOQ, visit RINVOQ.com. Patient Access and Support AbbVie is committed to helping people access RINVOQ and other medicines, including offering a patient support program and a co-pay card that may reduce out-of-pocket costs to $0 per month for eligible, commercially insured patients. Patients who are uninsured, or who are otherwise unable to pay for their medication, may be eligible for AbbVie's Patient Assistance Program, myAbbVieAssist, at https://www.abbvie.com/PatientAccessSupport. About IBD Inflammatory bowel disease (IBD) is a group of diseases characterized by chronic inflammation of the gastrointestinal (GI) tract. Crohn's disease (CD) and ulcerative colitis (UC) are the most common forms of IBD. In both CD and UC, the immune system causes inflammation and damage to the mucosa – or lining – of the gut.1,2,3 Specifically, CD manifests as inflammation within the GI tract, most commonly in the area between the small intestine (ileum) and the colon, causing persistent diarrhea and abdominal pain.1,4 UC is a chronic, idiopathic, immune-mediated IBD of the large intestine that causes continuous mucosal inflammation extending, to a variable extent, from the rectum to the more proximal colon.3,5 The hallmark signs and symptoms of UC include rectal bleeding, abdominal pain, bloody diarrhea, tenesmus (a sense of pressure), urgency and fecal incontinence.3,5 CD and UC are progressive diseases, meaning they get worse over time and may lead to life-threatening complications or surgery.6,7 Because the signs and symptoms of CD and UC are unpredictable, they cause a significant burden on people living with the disease—not only physically, but also emotionally and economically.8 About RINVOQ® (upadacitinib)9 Discovered and developed by AbbVie scientists, RINVOQ is a JAK inhibitor that is being studied in several immune-mediated inflammatory diseases. In human leukocyte cellular assays, RINVOQ inhibited cytokine-induced STAT phosphorylation mediated by JAK1 and JAK1/JAK3 more potently than JAK2/JAK2 mediated STAT phosphorylation. The relevance of inhibition of specific JAK enzymes to therapeutic effectiveness and safety is not currently known. Upadacitinib (RINVOQ) is being studied in Phase 3 clinical trials for alopecia areata, hidradenitis suppurativa, Takayasu arteritis, systemic lupus erythematosus, and vitiligo. RINVOQ (upadacitinib) U.S. Uses and Important Safety Information RINVOQ is a prescription medicine used to treat: Adults with moderate to severe rheumatoid arthritis (RA) when 1 or more medicines called tumor necrosis factor (TNF) blockers have been used, and did not work well or could not be tolerated. Adults with active psoriatic arthritis (PsA) when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated. Adults with active ankylosing spondylitis (AS) when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated. Adults with active non-radiographic axial spondyloarthritis (nr-axSpA) with objective signs of inflammation when a TNF blocker medicine has been used, and did not work well or could not be tolerated. Adults with giant cell arteritis (GCA). Adults with moderate to severe ulcerative colitis (UC) when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated, or after taking a different injection or pill (systemic therapy) when your healthcare provider does not recommend TNF blockers. Adults with moderate to severe Crohn's disease (CD) when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated, or after taking a different injection or pill (systemic therapy) when your healthcare provider does not recommend TNF blockers. It is not known if RINVOQ is safe and effective in children with ankylosing spondylitis, non-radiographic axial spondyloarthritis, ulcerative colitis, or Crohn's disease. Adults and children 12 years of age and older with moderate to severe eczema (atopic dermatitis [AD]) that did not respond to previous treatment and their eczema is not well controlled with other pills or injections, including biologic medicines, or the use of other pills or injections is not recommended. It is not known if RINVOQ is safe and effective in children under 12 years of age with atopic dermatitis. It is not known if RINVOQ LQ is safe and effective in children with atopic dermatitis. RINVOQ/RINVOQ LQ is a prescription medicine used to treat: Children 2 years of age and older with active polyarticular juvenile idiopathic arthritis (pJIA) when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated. Children 2 to less than 18 years of age with active psoriatic arthritis (PsA) when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated. It is not known if RINVOQ/RINVOQ LQ is safe and effective in children under 2 years of age with polyarticular juvenile idiopathic arthritis or psoriatic arthritis. IMPORTANT SAFETY INFORMATION FOR RINVOQ/RINVOQ LQ (upadacitinib) What is the most important information I should know about RINVOQ*? RINVOQ may cause serious side effects, including: Serious infections. RINVOQ can lower your ability to fight infections. Serious infections have happened while taking RINVOQ, including tuberculosis (TB) and infections caused by bacteria, fungi, or viruses that can spread throughout the body. Some people have died from these infections. Your healthcare provider (HCP) should test you for TB before starting RINVOQ and check you closely for signs and symptoms of TB during treatment with RINVOQ. You should not start taking RINVOQ if you have any kind of infection unless your HCP tells you it is okay. If you get a serious infection, your HCP may stop your treatment until your infection is controlled. You may be at higher risk of developing shingles (herpes zoster). Increased risk of death in people 50 years and older who have at least 1 heart disease (cardiovascular) risk factor. Cancer and immune system problems. RINVOQ may increase your risk of certain cancers. Lymphoma and other cancers, including skin cancers, can happen. Current or past smokers are at higher risk of certain cancers, including lymphoma and lung cancer. Follow your HCP's advice about having your skin checked for skin cancer during treatment with RINVOQ. Limit the amount of time you spend in sunlight. Wear protective clothing when you are in the sun and use sunscreen. Increased risk of major cardiovascular (CV) events, such as heart attack, stroke, or death, in people 50 years and older who have at least 1 heart disease (CV) risk factor, especially if you are a current or past smoker. Blood clots. Blood clots in the veins of the legs or lungs and arteries can happen with RINVOQ. This may be life-threatening and cause death. Blood clots in the veins of the legs and lungs have happened more often in people who are 50 years and older and with at least 1 heart disease (CV) risk factor. Allergic reactions. Symptoms such as rash (hives), trouble breathing, feeling faint or dizzy, or swelling of your lips, tongue, or throat, that may mean you are having an allergic reaction have been seen in people taking RINVOQ. Some of these reactions were serious. If any of these symptoms occur during treatment with RINVOQ, stop taking RINVOQ and get emergency medical help right away. Tears in the stomach or intestines. This happens most often in people who take nonsteroidal anti-inflammatory drugs (NSAIDs) or corticosteroids. Get medical help right away if you get stomach-area pain, fever, chills, nausea, or vomiting. Changes in certain laboratory tests. Your HCP should do blood tests before you start taking RINVOQ and while you take it. Your HCP may stop your RINVOQ treatment for a period of time if needed because of changes in these blood test results. Do not take RINVOQ if you are allergic to upadacitinib or any of the ingredients in RINVOQ. See the Medication Guide or Consumer Brief Summary for a complete list of ingredients. What should I tell my HCP BEFORE starting RINVOQ? Tell your HCP if you: Are being treated for an infection, have an infection that won't go away or keeps coming back, or have symptoms of an infection, such as: Fever, sweating, or chills Shortness of breath Warm, red, or painful skin or sores on your body Muscle aches Feeling tired Blood in phlegm Diarrhea or stomach pain Cough Weight loss Burning when urinating or urinating more often than normal Have TB or have been in close contact with someone with TB. Are a current or past smoker. Have had a heart attack, other heart problems, or stroke. Have or have had any type of cancer, hepatitis B or C, shingles (herpes zoster), blood clots in the veins of your legs or lungs, diverticulitis (inflammation in parts of the large intestine), or ulcers in your stomach or intestines. Have other medical conditions, including liver problems, low blood cell counts, diabetes, chronic lung disease, HIV, or a weak immune system. Live, have lived, or have traveled to parts of the country, such as the Ohio and Mississippi River valleys and the Southwest, that increase your risk of getting certain kinds of fungal infections. If you are unsure if you've been to these types of areas, ask your HCP. Have recently received or are scheduled to receive a vaccine. People who take RINVOQ should not receive live vaccines. Are pregnant or plan to become pregnant. Based on animal studies, RINVOQ may harm your unborn baby. Your HCP will check whether or not you are pregnant before you start RINVOQ. You should use effective birth control (contraception) to avoid becoming pregnant during treatment with RINVOQ and for 4 weeks after your last dose. There is a pregnancy surveillance program for RINVOQ. The purpose of the program is to collect information about the health of you and your baby. If you become pregnant while taking RINVOQ, you are encouraged to report the pregnancy by calling 1-800-633-9110. Are breastfeeding or plan to breastfeed. RINVOQ may pass into your breast milk. Do not breastfeed during treatment with RINVOQ and for 6 days after your last dose. Fever, sweating, or chills Shortness of breath Warm, red, or painful skin or sores on your body Muscle aches Feeling tired Blood in phlegm Diarrhea or stomach pain Cough Weight loss Burning when urinating or urinating more often than normal Tell your HCP about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. RINVOQ and other medicines may affect each other, causing side effects. Especially tell your HCP if you take: Medicines for fungal or bacterial infections Rifampicin or phenytoin Medicines that affect your immune system If you are not sure if you are taking any of these medicines, ask your HCP or pharmacist. What should I avoid while taking RINVOQ? Avoid food or drink containing grapefruit during treatment with RINVOQ as it may increase the risk of side effects. What should I do or tell my HCP AFTER starting RINVOQ? Tell your HCP right away if you have any symptoms of an infection. RINVOQ can make you more likely to get infections or make any infections you have worse. Get emergency help right away if you have any symptoms of a heart attack or stroke while taking RINVOQ, including: Discomfort in the center of your chest that lasts for more than a few minutes or that goes away and comes back Severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw Pain or discomfort in your arms, back, neck, jaw, or stomach Shortness of breath with or without chest discomfort Breaking out in a cold sweat Nausea or vomiting Feeling lightheaded Weakness in one part or on one side of your body Slurred speech Tell your HCP right away if you have any signs or symptoms of blood clots during treatment with RINVOQ, including: Swelling Pain or tenderness in one or both legs Sudden unexplained chest or upper back pain Shortness of breath or difficulty breathing Tell your HCP right away if you have a fever or stomach-area pain that does not go away, and a change in your bowel habits. Discomfort in the center of your chest that lasts for more than a few minutes or that goes away and comes back Severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw Pain or discomfort in your arms, back, neck, jaw, or stomach Shortness of breath with or without chest discomfort Breaking out in a cold sweat Nausea or vomiting Feeling lightheaded Weakness in one part or on one side of your body Slurred speech Swelling Pain or tenderness in one or both legs Sudden unexplained chest or upper back pain Shortness of breath or difficulty breathing What are other possible side effects of RINVOQ? Common side effects include upper respiratory tract infections (common cold, sinus infections), shingles (herpes zoster), herpes simplex virus infections (including cold sores), bronchitis, nausea, cough, fever, acne, headache, swelling of the feet and hands (peripheral edema), increased blood levels of creatine phosphokinase, allergic reactions, inflammation of hair follicles, stomach-area (abdominal) pain, increased weight, flu, tiredness, lower number of certain types of white blood cells (neutropenia, lymphopenia, leukopenia), muscle pain, flu-like illness, rash, increased blood cholesterol levels, increased liver enzyme levels, pneumonia, low number of red blood cells (anemia), and infection of the stomach and intestine (gastroenteritis). A separation or tear to the lining of the back part of the eye (retinal detachment) has happened in people with atopic dermatitis treated with RINVOQ. Call your HCP right away if you have any sudden changes in your vision during treatment with RINVOQ. Some people taking RINVOQ may see medicine residue (a whole tablet or tablet pieces) in their stool. If this happens, call your HCP. These are not all the possible side effects of RINVOQ. How should I take RINVOQ/RINVOQ LQ? RINVOQ is taken once a day with or without food. Do not split, crush, or chew the tablet. Take RINVOQ exactly as your HCP tells you to use it. RINVOQ is available in 15 mg, 30 mg, and 45 mg extended-release tablets. RINVOQ LQ is taken twice a day with or without food. RINVOQ LQ is available in a 1 mg/mL oral solution. RINVOQ LQ is not the same as RINVOQ tablets. Do not switch between RINVOQ LQ and RINVOQ tablets unless the change has been made by your HCP. *Unless otherwise stated, "RINVOQ" in the IMPORTANT SAFETY INFORMATION refers to RINVOQ and RINVOQ LQ. This is the most important information to know about RINVOQ. For more information, talk to your HCP. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. If you are having difficulty paying for your medicine, AbbVie may be able to help. Visit AbbVie.com/PatientAccessSupport to learn more. Please click here for the Full Prescribing Information and Medication Guide. Globally, prescribing information varies; refer to the individual country product label for complete information. About AbbVie AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, neuroscience, oncology, and eye care - and products and services across our Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on X (formerly Twitter), Facebook, Instagram, YouTube or LinkedIn. About AbbVie in Gastroenterology With a robust clinical trial program, AbbVie is committed to cutting-edge research to drive exciting developments in IBD, like ulcerative colitis and Crohn's disease. By innovating, learning, and adapting, AbbVie aspires to eliminate the burden of IBD and make a positive long-term impact on the lives of people with IBD. For more information on AbbVie in gastroenterology, visit https://www.abbvie.com/our-science/therapeutic-focus-areas/immunology/immunology-focus-areas/gastroenterology.html. Forward-Looking Statements Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions and uses of future or conditional verbs, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, changes to laws and regulations applicable to our industry, the impact of global macroeconomic factors, such as economic downturns or uncertainty, international conflict, trade disputes and tariffs, and other uncertainties and risks associated with global business operations. Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," of AbbVie's 2024 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its Quarterly Reports on Form 10-Q and in other documents that AbbVie subsequently files with the Securities and Exchange Commission that update, supplement or supersede such information. AbbVie undertakes no obligation, and specifically declines, to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law. US-RNQ-250311 Global Media: Ana Paula Barboza +1 (847) 938-2144 ana-paula.barboza@abbvie.com U.S. Media: Jasmine Farley +1 (847) 309-2779 jasmine.farley@abbvie.com Investors: Todd Bosse +1 (847) 936-1182 todd.bosse@abbvie.com SOURCE AbbVie

– Transactions Provide Karyopharm with $100 Million of Financial Flexibility and Additional Capital – – Preliminary Total Revenue and U.S. XPOVIO® (selinexor) Net Product Revenue for the Third Quarter of 2025 Expected to be in the Range of $42 to $44 Million and Approximately $32 Million, Respectively – – Top-Line Data from the Phase 3 SENTRY Trial in Myelofibrosis on Track for March 2026 – NEWTON, Mass., Oct. 8, 2025 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced that it has entered into comprehensive financing and capital structure transactions expected to provide the Company with $100 million of financial flexibility and additional capital, extending the Company's cash runway into the second quarter of 2026 based on the Company's current operating plans. "Following the recent completion of enrollment of our Phase 3 SENTRY trial in myelofibrosis, we are excited to announce this strategic financing which is expected to provide us with the resources needed to deliver top-line data from this pivotal trial," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "I would like to thank our lenders, noteholders and equity investors for their ongoing support to strengthen our balance sheet, equitize near-term debt maturities and support our potentially transformational Phase 3 myelofibrosis program. We believe that selinexor plus ruxolitinib has the potential to be the first combination therapy approved for the treatment of myelofibrosis. By combining selinexor with the current standard of care, we have the potential to redefine the way people living with myelofibrosis are treated." Each of the financing and capital structure transactions is expected to close on or around October 10, 2025 (Closing Date), subject to the satisfaction of customary closing conditions. The Company's existing senior lenders have agreed to multi-faceted financing transactions with the following key components: $67.5 million in financial flexibility and new capital consisting of $27.5 million in new term loan borrowings and new convertible notes, $25 million of near-term deferrals of interest and royalty payments, and a $15 million temporary reduction in the Company's minimum liquidity covenant. In addition, holders of the Company's convertible notes due 2029 have agreed to exchange $15 million of their notes for newly issued shares of common stock or pre-funded warrants in lieu thereof. Holders of approximately $24.25 million aggregate principal amount of the Company's senior unsecured convertible notes due October 15, 2025 have agreed to exchange their notes and accrued interest due thereon at a discount to par value for newly issued shares of common stock, or pre-funded warrants in lieu thereof, plus warrants to purchase shares of common stock. In addition, the Company has entered into a securities purchase agreement for a private placement in which the Company agreed to sell 1,487,917 shares of common stock and accompanying warrants to purchase 1,317,771 shares of common stock with an exercise price of $6.64 per share. The warrants issued in the private placement will be exercisable on or before the 30th day following the public announcement of top-line results from the Company's XPORT-EC-042 trial in endometrial cancer, which is anticipated in mid-2026. The private placement is expected to result in gross proceeds of approximately $8.75 million before deducting any offering expenses. The Company intends to use the proceeds of the financing transactions to pay transaction expenses and for general corporate purposes, including to support the Company's ongoing and planned clinical trial activities. At closing of the financing transactions, the Company will issue an aggregate of 7,223,982 newly issued shares of common stock, newly issued pre-funded warrants to purchase an aggregate of 2,913,136 shares of common stock, and newly issued warrants to purchase an aggregate of 5,918,358 shares of common stock with an exercise price of $6.64 per share, a 15% premium to the Nasdaq minimum price. In addition, the Company will reduce the exercise price of outstanding warrants to purchase 3,068,417 shares from $16.50 per share to $6.64 per share. The financing transactions are intended to comply with Nasdaq rules, including being priced at the "minimum price" (as defined in the Nasdaq rules). Following consummation of the transactions, the Company expects to have an aggregate of 15,926,939 shares of common stock outstanding (assuming no exercise of any pre-funded warrants or warrants or conversions of any outstanding convertible notes). Additional details on the financing transactions will be available in a Form 8-K that the Company will file with the United States Securities and Exchange Commission. Preliminary Third Quarter 2025 Financial Results Based on preliminary financial information, the Company expects total revenue, which includes license and royalty revenue from partners, to be in the range of $42 to $44 million and U.S. XPOVIO net product revenue to be approximately $32 million for the three months ended September 30, 2025. Prior to the receipt of approximately $36 million of gross proceeds from the financing transactions, the Company expects to report that it had cash, cash equivalents, restricted cash and investments as of September 30, 2025 of approximately $46 million. The financial information presented in this press release reflects the Company's estimates with respect to total revenue, U.S. XPOVIO net product revenue and its cash balance and is based on currently available information, which is preliminary. The Company's final results may vary from these preliminary estimates as a result of the completion of customary quarterly review procedures, including those conducted by the Company's external auditors. Endometrial Cancer Program Enrollment continues in the Phase 3 XPORT-EC-042 (NCT05611931) trial evaluating selinexor as a maintenance-only therapy following systemic therapy versus placebo in patients with TP53 wild-type advanced or recurrent endometrial cancer. The Company continues to expect to report top-line data from this event-driven trial in mid-2026. Ongoing Efforts to Further Enhance Liquidity and Maximize Value The Company expects to continue exploring potential financing and strategic alternatives to enhance liquidity and maximize value with the assistance of its advisors, including its financial advisor Centerview Partners LLC. Centerview Partners LLC and J. Wood Capital Advisors LLC acted as financial advisors to Karyopharm and Sidley Austin LLP acted as legal counsel to Karyopharm in connection with these transactions. The offer and sale of the shares of common stock, pre-funded warrants, warrants, the New 2028 Notes, the New 2029 Notes or any other securities (including the shares of common stock issuable upon conversion of the New 2028 Notes, the New 2029 Notes and the shares of common stock issuable upon exercise of the warrants and pre-funded warrants issued in the financing transactions) are not being registered under the Securities Act, or any state securities laws. The shares of common stock, pre-funded warrants, warrants, the New 2028 Notes, the New 2029 Notes or any other securities (including the shares of common stock issuable upon conversion of the New 2028 Notes, the New 2029 Notes and the shares of common stock issuable upon exercise of the warrants and pre-funded warrants issued in the financing transactions) may not be offered or sold in the United States except pursuant to an exemption from the registration requirements of the Securities Act and any applicable state securities laws. This press release does not constitute an offer to sell or the solicitation of an offer to buy shares of common stock, pre-funded warrants, warrants, the New 2028 Notes, the New 2029 Notes or any other securities, nor shall there be any offer, solicitation or sale of shares of common stock, pre-funded warrants, warrants, the New 2028 Notes, the New 2029 Notes or any other securities (including the shares of common stock issuable upon conversion of the New 2028 Notes, the New 2029 Notes and the shares of common stock issuable upon exercise of the warrants and pre-funded warrants issued in the financing transactions) in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful. About the Phase 3 SENTRY Trial SENTRY (XPORT-MF-034; NCT04562389) is a Phase 3 clinical trial evaluating a once-weekly dose of 60 mg of selinexor in combination with ruxolitinib compared to placebo plus ruxolitinib in JAKi-naïve myelofibrosis patients with platelet counts >100 x 10 9/L. Patients are randomized 2-to-1 to the selinexor arm. The co-primary endpoints for this trial are spleen volume reduction ≥ 35% (SVR35) at week 24 and the average change in absolute total symptom score (Abs-TSS) over 24 weeks relative to baseline. About Myelofibrosis Myelofibrosis is a rare blood cancer that affects approximately 20,000 patients in the United States and 17,000 patients in the European Union1. The disease causes bone marrow fibrosis (scarring in the bone marrow), which makes it difficult for the bone marrow to make healthy blood cells, splenomegaly (enlarged spleen), progressive anemia which often leads to symptoms like fatigue and weakness, and other disease associated symptoms including abdominal discomfort, pain under the left ribs, early satiety, night sweats and bone pain. The only approved class of therapies to treat myelofibrosis are JAK inhibitors, including ruxolitinib. Patients treated with the most commonly prescribed JAK inhibitor often require blood transfusions, and more than 30% will discontinue treatment due to anemia.2 Anemia and transfusion dependence are strongly correlated with poor prognosis and shortened survival.3 About the Phase 3 XPORT-EC-042 Trial EC-042 (XPORT-EC-042; NCT05611931) is a global, Phase 3, randomized, double-blind clinical trial evaluating selinexor as a maintenance therapy following systemic therapy in patients with TP53 wild-type advanced or recurrent endometrial cancer. The EC-042 trial is expected to enroll approximately 276 patients who will be randomized 1:1 to receive either a 60 mg, once-weekly, administration of oral selinexor or placebo until disease progression. The trial includes two patient populations, for which, the primary endpoint of progression free survival will be tested sequentially and the key secondary endpoint of overall survival will be evaluated: 1) a modified intent to treat population (mITT) that will include patients with either, a) TP53 wild-type tumors with proficient mismatch repair status (pMMR); or, b) TP53 wild-type tumors with deficient mismatch repair status (dMMR), who are medically ineligible to receive checkpoint inhibitors; and, 2) the trial's original intent to treat (ITT) population, which will include all patients enrolled in the trial whose tumors are TP53 wild-type, regardless of MMR status. The mITT population is expected to include approximately 220 patients. In connection with the EC-042 trial, Karyopharm entered into a global collaboration with Foundation Medicine, Inc. to develop FoundationOne®CDx, a tissue-based comprehensive genomic profiling test to identify and enroll patients whose tumors are TP53 wild-type. About Karyopharm Therapeutics Karyopharm Therapeutics Inc. (Nasdaq: KPTI) is a commercial-stage pharmaceutical company whose dedication to pioneering novel cancer therapies is fueled by a belief in the extraordinary strength and courage of patients with cancer. Since its founding, Karyopharm has been an industry leader in oral compounds that address nuclear export dysregulation, a fundamental mechanism of oncogenesis. Karyopharm's lead compound and first-in-class, oral exportin 1 (XPO1) inhibitor, XPOVIO® (selinexor), is approved in the U.S. and marketed by the Company in three oncology indications. It has also received regulatory approvals in various indications in 50 ex-U.S. territories and countries, including Europe and the United Kingdom (as NEXPOVIO®) and China. Karyopharm has a focused pipeline targeting indications in multiple high unmet need cancers, including in multiple myeloma, endometrial cancer, myelofibrosis, and diffuse large B-cell lymphoma (DLBCL). For more information about our people, science and pipeline, please visit , and follow us on LinkedIn and on X at @Karyopharm. Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the anticipated benefits of the private placement and financing transactions; the Company's expectations for the use of proceeds from the financing transactions; the Company's expected cash runway; the expected closing date for the private placement and financing transactions; the Company's ability to complete the financing transactions; the timing of the top-line results from the Company's Phase 3 SENTRY trial; the Company's preliminary financial information for the third quarter of 2025; the ability of selinexor to treat patients with multiple myeloma, endometrial cancer, myelofibrosis, diffuse large B-cell lymphoma and other diseases; and expectations with respect to the clinical development plans and potential regulatory submissions of selinexor. Such statements are subject to numerous important factors, risks and uncertainties, many of which are beyond Karyopharm's control, that may cause actual events or results to differ materially from Karyopharm's current expectations. For example, there can be no guarantee that Karyopharm will successfully commercialize XPOVIO or that any of Karyopharm's drug candidates, including selinexor, will successfully complete necessary clinical development phases or that development of any of Karyopharm's drug candidates will continue. Further, there can be no guarantee that any positive developments in the development or commercialization of Karyopharm's drug candidate portfolio will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: the adoption of XPOVIO in the commercial marketplace, the timing and costs involved in commercializing XPOVIO or any of Karyopharm's drug candidates that receive regulatory approval; the ability to obtain and retain regulatory approval of XPOVIO or any of Karyopharm's drug candidates that receive regulatory approval; Karyopharm's results of clinical trials and preclinical trials, including subsequent analysis of existing data and new data received from ongoing and future trials; the content and timing of decisions made by the U.S. Food and Drug Administration and other regulatory authorities, institutional review boards at clinical trial sites and publication review bodies, including with respect to the need for additional clinical trials; the ability of Karyopharm or its third party collaborators or successors in interest to fully perform their respective obligations under the applicable agreement and the potential future financial implications of such agreement; Karyopharm's ability to enroll patients in its clinical trials; unplanned cash requirements and expenditures; substantial doubt exists regarding Karyopharm's ability to continue as a going concern; development or regulatory approval of drug candidates by Karyopharm's competitors for products or product candidates in which Karyopharm is currently commercializing or developing; the direct or indirect impact of the COVID-19 pandemic or any future pandemic on Karyopharm's business, results of operations and financial condition; and Karyopharm's ability to obtain, maintain and enforce patent and other intellectual property protection for any of its products or product candidates. These and other risks are described under the caption "Risk Factors" in Karyopharm's Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, which was filed with the Securities and Exchange Commission (SEC) on August 11, 2025, and in other filings that Karyopharm may make with the SEC in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and, except as required by law, Karyopharm expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. XPOVIO® and NEXPOVIO® are registered trademarks of Karyopharm Therapeutics Inc. SOURCE Karyopharm Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED