Sobi to unveil new myelofibrosis data at ASCO 2024

Sobi North America, an affiliate of Swedish Orphan Biovitrum AB (Sobi®), announced its participation in the upcoming American Society of Clinical Oncology (ASCO) Annual Meeting from May 31 to June 4, 2024, in Chicago. The company will present three abstracts showcasing data related to its myelofibrosis treatment, pacritinib. These studies underline Sobi’s dedication to developing innovative treatments for individuals with hematological conditions, particularly rare disorders like myelofibrosis.

One of the key presentations will be a retrospective analysis demonstrating the effectiveness of pacritinib in reducing spleen volume, improving symptoms, and enhancing red blood cell transfusion responses, compared to the best available therapy, in myelofibrosis patients with both thrombocytopenia and anemia.

Another retrospective analysis will highlight the significant symptom relief pacritinib offers compared to the best available therapy or low-dose ruxolitinib, specifically in patients who required red blood cell transfusions at the start of pacritinib treatment. The rates of Grade 3 anemia emerging during treatment were similar between the pacritinib and best available therapy (BAT) groups.

Additionally, newly gathered real-world data will be presented, showing that pacritinib treatment results in stable or improved thrombocytopenia and/or anemia in myelofibrosis patients, regardless of initial counts. This treatment also shows favorable overall survival rates comparable to historical JAK inhibitor controls.

The abstracts to be presented at ASCO 2024 are:
1. Impact of pacritinib on symptoms in patients with thrombocytopenia and myelofibrosis who require red blood cell transfusion (#6577).
2. Efficacy of pacritinib in patients with myelofibrosis who have both thrombocytopenia and anemia (#6578).
3. Real-world treatment patterns and outcomes in patients with myelofibrosis treated with pacritinib in the United States (#6579).

Vonjo® (pacritinib) is approved for treating adults with intermediate- or high-risk primary or secondary myelofibrosis who have a platelet count below 50 × 10^9/L. This approval is based on the drug’s ability to reduce spleen volume, with continued approval contingent upon verification and description of clinical benefits in confirmatory trials. Sobi is currently conducting the Phase 3 PACIFICA study for VONJO in myelofibrosis patients with severe thrombocytopenia as a post-marketing requirement.

The critical safety information for VONJO includes several serious side effects. Severe bleeding is a potential risk, and patients should stop taking VONJO if bleeding occurs. Another common but severe side effect is diarrhea, which can lead to dehydration. Patients might also experience worsening low platelet counts, changes in heart electrical activity (QTc prolongation), increased cardiovascular events, blood clots, secondary cancers, and serious infections. Patients with specific medical histories or those planning surgery should discuss these factors with their healthcare provider before starting VONJO. The most common side effects of VONJO include nausea, vomiting, low red blood cell count (anemia), and swelling of ankles, legs, and feet.

Sobi North America, based in the Boston area with Canadian headquarters in Toronto, is dedicated to providing innovative treatments for rare diseases. The company boasts a robust product portfolio focusing on immunology, hematology, and specialty care. Sobi North America's growing team has demonstrated commercial excellence across North America.

Sobi® is an international biopharmaceutical company with approximately 1,800 employees worldwide. The company focuses on hematology, immunology, and specialty care, aiming to transform the lives of people with rare and debilitating diseases. In 2023, Sobi reported revenues of SEK 22.1 billion, and its shares are listed on Nasdaq Stockholm.

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