"We take pride in our expanded commitment to individuals affected by rare diseases, which we are pleased to highlight at this year's ASH meeting, with research that spans a range of rare and debilitating conditions. We are continuing our strong legacy of elevating standards of care for haemophilia A with two oral presentations. Additionally, we mark our entry into the myelofibrosis field with several poster presentations," said Lydia Abad-Franch, MD, MBA, Head of Research, Development, and Medical Affairs (RDMA), and Chief Medical Officer at Sobi. "We look forward to collaborating and connecting in person at this year's meeting."
Key data to be presented at ASH 2023
+-------------+------------------+---------------------------------------------+
|Haemophilia A                                                                 |
+-------------+------------------+---------------------------------------------+
|Efanesoctocog|Once-Weekly       |Oral presentation.                           |
|Alfa         |Efanesoctocog Alfa|                                             |
|             |Prophylaxis       |#506                                         |
|             |Provided High     |                                             |
|             |Sustained Factor  |Session: 322                                 |
|             |VIII Activity     |                                             |
|             |Levels,           |Sunday, December 10, 2023, 12:00 PM - 1:30 PM|
|             |Independent of    |                                             |
|             |Blood Group, in   |Presentation Time: 12:15 PM                  |
|             |Children <12 Years|                                             |
|             |of Age with Severe|                                             |
|             |Hemophilia A      |                                             |
+-------------+------------------+---------------------------------------------+
|Experiences  |Oral presentation.|
|with         |                  |
|Efanesoctocog|#507              |
|Alfa: Exit   |                  |
|Interviews   |Session: 322      |
|with         |                  |
|Caregivers of|Sunday, December  |
|Previously   |10, 2023, 12:00 PM|
|Treated      |- 1:30 PM         |
|Patients with|Presentation Time:|
|Hemophilia A |12:30 PM          |
|from the     |                  |
|XTEND-Kids   |                  |
|Phase 3      |                  |
|Clinical     |                  |
|Trial        |                  |
+-------------+------------------+---------------------------------------------+
|Experience   |Poster            |
|with         |presentation.     |
|Accelerometer|                  |
|Activity     |#2360             |
|Tracking in  |                  |
|Patients with|Session: 904      |
|Hemophilia A:|                  |
|Results from |Saturday, December|
|the XTEND-1  |9, 2023, 5:30 PM -|
|Efanesoctocog|7:30 PM           |
|Alfa Phase 3 |                  |
|Trial        |                  |
+-------------+------------------+---------------------------------------------+
|Treatment of |Poster            |
|Bleeding     |presentation.     |
|Episodes with|                  |
|Efanesoctocog|#3993             |
|Alfa in      |                  |
|Children with|Session: 322      |
|Severe       |                  |
|Hemophilia A |Monday, December  |
|in the XTEND |11, 2023, 6:00 PM |
|-Kids Phase 3|- 8:00 PM         |
|Study        |                  |
+-------------+------------------+---------------------------------------------+
+----------------+-----------------+---------------------+
|Haemophilia A                                           |
+----------------+-----------------+---------------------+
|                |Real-World Unmet |Poster presentation. |
|                |Needs in         |                     |
|                |Patients with    |#2380                |
|                |Hemophilia A     |                     |
|                |Without          |Session: 904         |
|                |Inhibitors in    |                     |
|                |the US: Results  |Saturday, December 9,|
|                |from the Picnic  |2023, 5:30 PM - 7:30 |
|                |Health Database  |PM                   |
+----------------+-----------------+---------------------+
|Investigating   |Poster           |
|the Relationship|presentation.    |
|between         |                 |
|Endogenous      |#5125            |
|Factor VIII     |                 |
|Levels and      |Session: 904     |
|Annual Bleed    |                 |
|Rates and Health|Monday, December |
|-Related Quality|11, 2023, 6:00   |
|of Life in      |PM - 8:00 PM     |
|Patients with   |                 |
|Hemophilia A Not|                 |
|Treated with    |                 |
|Factor VIII     |                 |
|Prophylaxis     |                 |
+----------------+-----------------+---------------------+
|Paroxysmal                                              |
|Nocturnal                                               |
|Hemoglobinuria                                          |
+----------------+-----------------+---------------------+
|Empaveli®       |Efficacy and     |Oral presentation.   |
|(pegcetacoplan) |Safety Is        |                     |
|                |Maintained in    |#574                 |
|                |Adult Patients   |                     |
|                |with Paroxysmal  |Session: 508         |
|                |Nocturnal        |Sunday, December 10, |
|                |Hemoglobinuria   |2023, 4:30 PM - 6:00 |
|                |Receiving        |PM                   |
|                |Pegcetacoplan    |Presentation Time:   |
|                |for up to 3      |5:15 PM              |
|                |Years            |                     |
+----------------+-----------------+---------------------+
|Immune                                                  |
|Thrombocytopenia                                        |
+----------------+-----------------+---------------------+
|Doptelet®       |Interim Baseline |Poster presentation. |
|(avatrombopag)  |Characteristics  |                     |
|                |of Adult         |#1226                |
|                |Patients with    |                     |
|                |Immune           |Session: 311         |
|                |Thrombocytopenia |                     |
|                |Enrolled in the  |Saturday, December 9,|
|                |Observational    |2023, 5:30 PM - 7:30 |
|                |Multicenter      |PM                   |
|                |Phase 4 ADOPT    |                     |
|                |Study to         |                     |
|                |Evaluate the Use |                     |
|                |and              |                     |
|                |Effectiveness of |                     |
|                |Avatrombopag     |                     |
+----------------+-----------------+---------------------+
|Interim Analysis|Poster           |
|of Platelet     |presentation.    |
|Response in a   |#2577            |
|Prospective     |                 |
|Phase 4 Study in|Session: 311,    |
|Adult Immune    |Sunday, 10       |
|Thrombocytopenia|December 2023,   |
|(ITP) Subjects  |6:00 PM - 8:00   |
|after Switching |PM               |
|from Eltrombopag|                 |
|(ELT) or        |                 |
|Romiplostim     |                 |
|(ROM) to        |                 |
|Avatrombopag    |                 |
|(AVA)           |                 |
+----------------+-----------------+---------------------+
|Interim Analysis|Poster           |
|of Treatment    |presentation.    |
|satisfaction    |                 |
|from a          |#3954            |
|Prospective     |                 |
|Phase 4 Study in|Session: 311     |
|Adult Immune    |                 |
|Thrombocytopenia|Monday, 11       |
|(ITP) Subjects  |December 2023,   |
|after Switching |6:00 PM - 8:00   |
|from Eltrombopag|PM               |
|or Romiplostim  |                 |
|to Avatrombopag |                 |
+----------------+-----------------+---------------------+
|Real-World      |Poster           |
|Treatment       |presentation.    |
|Patterns and    |                 |
|Outcomes in     |#5127            |
|Patients with   |                 |
|Immune          |Session: 904     |
|Thrombocytopenia|                 |
|Treated with    |Monday, December |
|Avatrombopag in |11, 2023, 6:00   |
|the United      |PM - 8:00 PM     |
|States: REAL-AVA|                 |
|2.0 Study Design|                 |
+----------------+-----------------+---------------------+
|Relapsed/Refracto                                       |
|ry Diffuse Large                                        |
|B-Cell Lymphoma                                         |
+----------------+-----------------+---------------------+
|Zynlonta®       |Early and        |Poster presentation. |
|(loncastuximab  |Sustained        |                     |
|tesirine)       |Circulating      |#3133                |
|                |Tumor DNA        |                     |
|                |Response         |Session: 627         |
|                |Dynamics after   |                     |
|                |Loncastuximab    |Sunday, December 10, |
|                |Tesirine for     |2023, 6:00 PM - 8:00 |
|                |Relapsed/Refracto|PM                   |
|                |ry Diffuse Large |                     |
|                |B-Cell Lymphoma  |                     |
+----------------+-----------------+---------------------+
+-------------------+--------------------+---------------------+
|Myelofibrosis                                                 |
+-------------------+--------------------+---------------------+
|Vonjo® (pacritinib)|Impact of Symptom   |Poster presentation. |
|                   |Benefit and         |                     |
|                   |Transfusion Response|#3207                |
|                   |on Survival in      |                     |
|                   |Myelofibrosis       |Session: 634         |
|                   |Patients Treated    |                     |
|                   |with Pacritinib:    |Sunday, December 10, |
|                   |PERSIST-2 Landmark  |2023, 6:00 PM - 8:00 |
|                   |Survival Analysis   |PM                   |
+-------------------+--------------------+---------------------+
|Platelet Response  |Poster presentation.|
|in Pacritinib      |                    |
|-Treated Patients  |#4554               |
|with Cytopenic     |                    |
|Myelofibrosis: a   |Session: 634        |
|Retrospective      |                    |
|Analysis of PERSIST|Monday, 11 December |
|-2 and PAC203      |2023, 6:00 PM -8:00 |
|Studies            |PM                  |
+-------------------+--------------------+---------------------+
|Retrospective      |Poster presentation.|
|Analysis of the    |                    |
|Relationship       |#4566               |
|between Transfusion|                    |
|Independence and   |Session: 634        |
|Bone Marrow        |                    |
|Fibrosis Reduction |Monday, 11 December |
|in Patients with   |2023, 6:00 PM -8:00 |
|Myelofibrosis      |PM                  |
|Treated with       |                    |
|Pacritinib Versus  |                    |
|Ruxolitinib        |                    |
+-------------------+--------------------+---------------------+
|An Analysis of     |Poster presentation.|
|Ruxolitinib Dosing |                    |
|for Myelofibrosis  |#5186               |
|in Real-World      |                    |
|Practice           |Session: 906        |
|                   |                    |
|                   |Monday, December 11,|
|                   |2023, 6:00 PM - 8:00|
|                   |PM                  |
+-------------------+--------------------+---------------------+
|Haemophagocytic                                               |
|Lymphohistiocytosis                                           |
+-------------------+--------------------+---------------------+
|Gamifant®          |Emapalumab, a Fully |Poster presentation. |
|(emapalumab)       |Human Anti          |                     |
|                   |-Interferon Gamma   |#1174                |
|                   |Monoclonal Antibody,|                     |
|                   |in Pediatric        |Session: 203         |
|                   |Patients With       |                     |
|                   |Primary             |Saturday, December 9,|
|                   |Hemophagocytic      |2023, 5:30 PM - 7:30 |
|                   |Lymphohistiocytosis:|PM                   |
|                   |Long-Term Follow-up |                     |
|                   |of a Phase 2/3 Study|                     |
+-------------------+--------------------+---------------------+
|Real-World         |Poster presentation.|
|Treatment Patterns |                    |
|and Outcomes Among |#2534               |
|Patients with      |                    |
|Primary            |Session: 201        |
|Hemophagocytic     |                    |
|Lymphohistiocytosis|Sunday, December 10,|
|with and without   |2023, 6:00 PM - 8:00|
|Infection at       |PM                  |
|Diagnosis and      |                    |
|Treated with       |                    |
|Emapalumab: The    |                    |
|REAL-HLH Study     |                    |
+-------------------+--------------------+---------------------+
|Real-World         |Poster presentation.|
|Treatment Patterns |                    |
|and Outcomes Among |#3909               |
|Patients with      |                    |
|Secondary          |Session: 201        |
|Hemophagocytic     |                    |
|Lymphohistiocytosis|Monday, December 11,|
|Treated with       |2023, 6:00 PM - 8:00|
|Emapalumab in the  |PM                  |
|United States: The |                    |
|REAL-HLH Study     |                    |
+-------------------+--------------------+---------------------+
About efanesoctocog alfa
Efanesoctocog alfa [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for a significant parts of the week, improving bleed protection in a once-weekly dose for people with haemophilia A. Efanesoctocog alfa builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® polypeptides toextend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. It was approved as ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the US in February 2023.
About the Sobi and Sanofi collaboration
Sobi and Sanofi collaborate on the development and commercialisation of Alprolix® and Elocta®/Eloctate®. The companies also collaborate on the development and commercialisation of efanesoctocog alfa, an investigational factor VIII therapy with the potential to provide high sustained factor activity levels with once-weekly dosing for people with haemophilia A. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory.
About Aspaveli®/ Empaveli®
Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as EMPAVELI®/ASPAVELI in the United States, European Union, and other countries globally. Aspaveli/Empaveli is also under investigation for several other rare diseases across haematology and nephrology.
About the Sobi and Apellis collaboration
Sobi and Apellis have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-US commercialisation rights for systemic pegcetacoplan, and Apellis has exclusive US commercialisation rights for systemic pegcetacoplan and retains worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy (GA).
About Doptelet®
Doptelet (avatrombopag) is an orally administered thrombopoietin receptor agonist (TPO-RA) that mimics the biologic effects of TPO in stimulating the development and maturation of megakaryocytes, resulting in increased platelet count. It is approved in over 30 countries worldwide, including the EU and the US, for the treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo an invasive procedure, and for the treatment of thrombocytopenia in adult patients with primary chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. Chronic ITP is a rare autoimmune bleeding disorder characterised by low number of platelets. The incidence of primary ITP in adults is 3.3/100,000 adults per year with a prevalence of 9.5 per 100,000 adults1.
1. Lambert et al. Blood 2017.
About Zynlonta®
Zynlonta (loncastuximab tesirine) is a CD19-directed antibody drug conjugate. Once bound to a CD19-expressing cell, Zynlonta is internalised by the cell, where enzymes release a pyrrolobenzodiazepine payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell
cycle arrest and tumour cell death. Zynlonta® is a registered trademark of ADC Therapeutics SA. Zynlonta is currently approved for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL), after two or more lines of systemic therapy, in the United States, European Union and Great Britain.
About ADC Therapeutics
ADC Therapeutics (NYSE: ADCT) is a commercial-stage biotechnology company improving the lives of those affected by cancer with its next-generation, targeted antibody drug conjugates (ADCs). The Company is advancing its proprietary PBD-based ADC technology to transform the treatment paradigm for patients with hematologic malignancies and solid tumours.
ADC Therapeutics is based in Lausanne (Biopôle), Switzerland and has operations in London, the San Francisco Bay Area and New Jersey. For more information, please visit adctherapeutics.com (https://www.adctherapeutics.com/) and follow the Company on Twitter (https://twitter.com/adctherapeutics)andLinkedIn (https://www.linkedin.com/company/adc-therapeutics/).
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). CTI is conducting the Phase 3 PACIFICA study of VONJO in patients with myelofibrosis and severe thrombocytopenia as a post-marketing requirement. For more information, please visit www.ctibiopharma.com.
About Gamifant®
Gamifant (emapalumab) is an anti-interferon gamma (IFNγ) monoclonal antibody that binds to and neutralises IFNγ. In the USA, Gamifant is indicated for the treatment of adult and paediatric (newborn and older) patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Gamifant is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT).
Sobi®
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Providing sustainable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East and Asia. In 2022, revenue amounted to SEK 18.8 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi atsobi.com (http://www.sobi.com/), LinkedIn and YouTube.
Contacts
For details on how to contact the Sobi Investor Relations Team, please click here (http://www.sobi.com/en/investors). For Sobi Media contacts, click here. (https://www.sobi.com/en/media)