Pacritinib

WALTHAM, Mass., May 24, 2024 (GLOBE NEWSWIRE) -- Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®), today announced the presentation of three abstracts that highlights data from its myelofibrosis treatment option at the American Society of Clinical Oncology (ASCO) Annual Meeting taking place in Chicago from May 31 – June 4, 2024. Sobi’s commitment to delivering innovative treatments for people living with hematological diseases is seen in global studies spanning multiple rare disorders, including myelofibrosis. A retrospective analysis will be presented that demonstrates the efficacy of pacritinib in spleen volume reduction, symptom benefit and red blood cell transfusion response, compared with best available therapy, in patients with myelofibrosis who have both thrombocytopenia and anemia. An additional retrospective analysis will be presented that shows the substantial symptom benefit pacritinib provides compared with best available therapy or low-dose ruxolitinib, specifically in patients who required red blood cell transfusion at the time of pacritinib initiation. The number of patients experiencing treatment emergent Grade 3 anaemia was similar between pacritinib and BAT groups. New real-world data will be presented that demonstrates treatment with pacritinib provides stability or improvement in thrombocytopenia and/or anemia in patients with myelofibrosis, regardless of baseline counts, and has favorable overall survival similar to other JAK inhibitor historical controls. Key Sobi data to be presented at ASCO 2024 MyelofibrosisVonjo(pacritinib)Impact of pacritinib on symptoms in patients with thrombocytopenia and myelofibrosis who require red blood cell transfusionPoster presentation #6577Monday, 3 June, 2024; 9:00 - 12:00 CDTEfficacy of pacritinib in patients with myelofibrosis who have both thrombocytopenia and anemiaPoster presentation #6578Monday, 3 June, 2024; 9:00 - 12:00 CDTReal-world treatment patterns and outcomes in patients with myelofibrosis treated with pacritinib in the United StatesPoster presentation #6579Monday, 3 June, 2024; 9:00 - 12:00 CDT All abstracts can be accessed via the official ASCO website. ContactsFor details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media, click here. About Vonjo®VONJO is approved for the treatment of adults with intermediate- or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. This indication is approved under FDA accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Sobi is conducting the Phase 3 PACIFICA study of VONJO in patients with myelofibrosis and severe thrombocytopenia as a post-marketing requirement. Vonjo Indication and Important Safety InformationVONJO is a prescription medicine used to treat adults with certain types of myelofibrosis (MF) who have a platelet count below 50,000 per microliter. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may depend on proof and description of clinical benefit in a confirmatory trial(s). It is not known if VONJO is safe and works in children. Important Safety Information What are the serious side effects of VONJO? Bleeding. VONJO may cause severe bleeding, which can be serious and, in some cases, may lead to death. Do not take VONJO if you are bleeding. If you start bleeding, stop VONJO and call your healthcare provider. Your healthcare provider will do a blood test to check your blood cell counts before you start VONJO and regularly during your treatment with VONJO. Tell your healthcare provider right away if you develop any of these symptoms: unusual bleeding, bruising, and fever. You will need to stop taking VONJO 7 days before any planned surgery or invasive procedures (such as a heart catheterization, stent placement in a coronary artery in your heart, or a procedure for varicose veins). Your healthcare provider should tell you when you can start taking VONJO again. Diarrhea. Diarrhea is common with VONJO, but can be severe, and cause loss of too much body fluid (dehydration). Tell your healthcare provider if you have diarrhea and follow instructions for what to do to help treat diarrhea. If you notice any change in how often you have bowel movements, if they become softer or you have diarrhea, your health care provider may tell you to start taking an antidiarrheal medicine as soon as you notice changes. Worsening low platelet counts. Your healthcare provider will do blood tests to check your blood counts before you start taking VONJO and during treatment with VONJO. Changes in the electrical activity of your heart called QTc prolongation. QTc prolongation can cause irregular heartbeats that can be life-threatening. Your healthcare provider will check the electrical activity of your heart before you start and during treatment with VONJO, as needed. If you have a history of low blood potassium, it is important that you get your blood tests done as ordered by your healthcare provider to monitor electrolytes in your blood. Tell your healthcare provider right away if you feel dizzy, lightheaded, faint or feel palpitations. Increased risk of major cardiovascular events such as heart attack, stroke, or death in people who have cardiovascular risk factors and who are current or past smokers have happened in some people taking another Janus associated kinase (JAK) inhibitor to treat rheumatoid arthritis. Get emergency help right away if you have any symptoms of a heart attack or stroke while taking VONJO, 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; or slurred speech. Increased risk of blood clots. Blood clots in the veins of your legs (deep vein thrombosis) or lungs (pulmonary embolism) have happened in some people taking another JAK inhibitor and may be life-threatening. Tell your healthcare provider if you have had blood clots in the veins of your legs or lungs in the past. Tell your healthcare provider right away if you have any signs and symptoms of blood clots during treatment with VONJO, including: swelling, pain, or tenderness in one or both legs; sudden, unexplained chest pain; or shortness of breath/difficulty breathing. Possible increased risk of new (secondary) cancers. People who take another JAK inhibitor for rheumatoid arthritis have an increased risk of new (secondary) cancers, including lymphoma and other cancers. The risk of new cancers is greater if you smoke or have smoked in the past. Risk of infection. People who have certain blood cancers and take another JAK inhibitor have an increased risk of serious infections. People who take VONJO may develop serious infections, including bacterial, mycobacterial, fungal, and viral infections. If you have a serious infection, your healthcare provider may not start you on VONJO until your infection is treated. Tell your healthcare provider right away if you develop any of the following symptoms of infection: chills, aches, fever, nausea, vomiting, weakness, painful skin rash, or blisters. What should I tell my healthcare provider before starting VONJO? Current or past smoking historyPrevious medical conditions such as any other cancers, blood clot, heart attack, other heart problems, stroke, infection, diarrhea, commonly loose stools, nausea, vomiting, liver problems, or kidney problemsHave active bleeding, have had severe bleeding or plan to have surgery. You should stop taking VONJO 7 days before any planned surgery or invasive procedures because you may be at higher risk for bleedingAre pregnant, plan to become pregnant, are breastfeeding, or plan to breastfeed. It is not known if VONJO will harm your unborn baby or if it passes into breast milk. You should not breastfeed during treatment and for 2 weeks after your last dose of VONJOPlan to father a child. VONJO may affect fertility in males. You may have problems fathering a child Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements and remedies. What are the most common side effects with VONJO?Nausea, vomiting, low red blood cell count (anemia), and swelling of your ankles, legs, and feet. These are not all the possible side effects of VONJO. If you suspect that you have experienced a side effect, call your healthcare provider. You may also report side effects to the US Food and Drug Administration (FDA) at 1-800-FDA-1088. Please see the full Prescribing Information for VONJO. About Sobi North AmericaAs the North American affiliate of international biopharmaceutical company Sobi, the Sobi North America team is committed to Sobi’s vision of being a leader in providing innovative treatments that transform lives for individuals with rare diseases. Our product portfolio includes multiple approved treatments focused on immunology, hematology and specialty care. With U.S. headquarters in the Boston area, Canadian headquarters in the Toronto area, and field sales, medical and market access representatives spanning North America, our growing team has a proven track record of commercial excellence. More information is available at https://www.sobi.com/usa or at www.sobi.com. About Sobi®Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of hematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube.

More than a decade after the FDA approved the first JAK inhibitor to treat myelofibrosis, Ajax Therapeutics thinks it has improved on the formula by targeting a less-well-known, inactive conformation of the kinase to boost efficacy and tolerability. The biotech raised $95 million in an oversubscribed series C, led by Goldman Sachs Alternatives, to bring its potentially first-in-class inhibitor, AJ1-11095, to the clinic next half. Monday’s round – which also saw participation from new investors Eli Lilly, Vivo Capital, RA Capital Management, Point72, as well as existing investors EcoR1 Capital, Boxer Capital, Schrödinger, and Inning One Ventures – adds to $40 million raised in 2021.Scientific originsAround 2017, five experts on the biology of the JAK pathway got together with one goal in mind – creating an improved kinase inhibitor that elicits a deeper and more durable response in patients with myelofibrosis. One of the scientists was Ross Levine, an oncologist at Memorial Sloan Kettering Cancer Center who helped discover the mutations in JAK2 associated with myelofibrosis.“We all were excited when the discoveries were made, and then hopeful that the drugs that were developed – ruxolitinib, pacritinib, momelotinib, fedratinib – would really be game changers for patients. And they brought real, meaningful benefits to patients, but they don't lead to molecular or clinical regressions,” Levine told FirstWord.Additionally, patient response isn’t durable, with most patients discontinuing treatment within a few years due to a loss of efficacy. And once a myelofibrosis patient fails on a JAK inhibitor, their life expectancy is around a year or two.“There's quite a lot of room between where these molecules are… and what would be optimal engagement,” Levine said.Out of the scientific consortium, an idea arose to design pleiotropic molecules that engage the inactive, Type II conformation of JAK2, rather than the active, Type 1 conformation, which the four myelofibrosis-approved JAK inhibitors target. Thus, Ajax – an abbreviation of “attack JAK” – was born in 2019, with Levine as a co-founder and serving as chair of the scientific advisory board.Preclinical potency The researchers had developed early chemical matter and structures that showed better efficacy than approved molecules, which gave the newco a foothold to begin designing a completely different kind of JAK inhibitor.But for the Type II conformation, “it’s not as easy as just screening the kinase domain and finding an inhibitor,” Ajax co-founder and CEO Martin Vogelbaum told FirstWord.So the team turned to digital chemistry company Schrödinger, which, along with serving as a co-founding investor, signed an exclusive collaboration with Ajax to apply its computational molecular discovery methods and expertise in structure based drug design to transform the biotech’s early work into a potent, specific and clinically tractable Type II JAK2 inhibitor.  Now, after five years of work optimising a highly selective compound, Ajax thinks AJ1-11095 is a safer molecule that produces a deeper, more durable response – and is ready to enter the clinic. A Phase I study in myelofibrosis is expected to begin in the second half.Vogelbaum said that repeated mice experiments suggest Ajax’s lead candidate has similar, if not better, efficacy than ruxolitinib alone or in combination with another approved agent.“Our preclinical data suggest that we can achieve greater target engagement at doses with a wide range that are efficacious – well beyond the current drugs – and are very well tolerated,” Ross said, adding that the team feels “very confident” that AJ1-11095 won’t have the off-target toxicities of others in the JAK class.