THPO

Avatrombopag PDUFA date set for July 24, 2025WALTHAM, Mass., Dec. 12, 2024 (GLOBE NEWSWIRE) -- Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®), today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for avatrombopag (DOPTELET®) for the treatment of thrombocytopenia in pediatric patients one year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a prior therapy. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of July 24, 2025. The FDA also accepted Sobi’s New Drug Application (NDA) for avatrombopag for oral suspension. Avatrombopag for oral suspension is a capsule where its granules are sprinkled onto a soft food or liquid to prepare for administration. “These application acceptances for avatrombopag brings us one step closer to much-needed, additional treatment options for adolescents and children with persistent and chronic ITP,” said Jamie Freedman, MD, PhD, Chief Medical Officer, Sobi North America. “Now, there’s an opportunity to address an unmet need in our pediatric population, with the added bonus of an additional formulation, an oral suspension option that may be more suitable for some patients.” The application is based on results from the AVA-PED-301 study, evaluating the efficacy and safety of avatrombopag for the treatment of pediatric patients with ITP of at least 6 months’ duration. The study met the primary endpoint of durable platelet response defined as the proportion of patients achieving at least six out of eight weekly platelet counts ≥50×109/L during the last eight weeks of the 12-week treatment period in the core phase in the absence of rescue medication. The alternative primary efficacy endpoint of two consecutive PC ≥50× 109 /L was also met. ContactsFor details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media, click here. About immune thrombocytopeniaImmune thrombocytopenia (ITP) is an autoimmune disorder characterized by low numbers of platelets that results in an increased risk of bleeding. Symptoms can be highly variable and include bruising, a rash of small dots that represent small hemorrhages, bleeding from the gums or nose, blood in the urine/stools and debilitating fatigue. It is estimated that up to 100 people per million live with ITP. The disorder is considered chronic when symptoms last more than 12 months.i Long-term, primary chronic ITP is associated with increased risk of infections, bleeding episodes requiring hospitalization, hematologic malignancies, and mortality. No cure is available, and patients with chronic ITP usually relapse after various treatments and still require treatment to reduce the risk of clinically significant bleeding. About DOPTELET (avatrombopag)DOPTELET is an orally bioavailable, small molecule thrombopoietin receptor agonist (TPO-RA) that stimulates the proliferation and differentiation of megakaryocytes from bone marrow progenitor cells, resulting in increased production of platelets. DOPTELET does not compete with TPO for binding to the TPO receptor. INDICATION DOPTELET® (avatrombopag) is is a thrombopoietin receptor agonist indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia who have had an insufficient response to a previous treatment. IMPORTANT SAFETY INFORMATION Thrombotic/Thromboembolic Complications. DOPTELET is a thrombopoietin (TPO) receptor agonist and TPO receptor agonists have been associated with thrombotic complications in patients with chronic liver disease (0.4%; (1/274) in DOPTELET-treated patients) and thromboembolic complications in patients with chronic immune thrombocytopenia (7%; (9/128) in DOPTELET-treated patients). Portal vein thrombosis has been reported in patients with chronic liver disease, and thromboembolic events (arterial and venous) have been reported in patients with chronic immune thrombocytopenia treated with TPO receptor agonists. Consider the potential increased thrombotic risk when administering DOPTELET to patients with known risk factors for thromboembolism, including genetic prothrombotic conditions and acquired risk factors. DOPTELET should not be administered to patients with chronic liver disease or chronic immune thrombocytopenia in an attempt to normalize platelet counts. Monitor platelet counts, and for signs and symptoms of thromboembolic events and institute treatment promptly. Serious Adverse Reactions Serious adverse reaction that occurred more frequently in patients treated with DOPTELET (9%; 12/128) compared to placebo (5%; 1/22) was headache, occurring in 1.6% (2/128). Adverse Reactions The most common adverse reactions (≥10%) in patients with chronic immune thrombocytopenia were headache, fatigue, contusion, epistaxis, upper respiratory tract infection, arthralgia, gingival bleeding, petechiae, and nasopharyngitis. About Sobi North America As 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.

The TPO receptor, also known as the thrombopoietin receptor, plays a crucial role in the human body's production of platelets. Thrombopoietin, a hormone produced in the liver and kidneys, binds to the TPO receptor, stimulating the production and maturation of megakaryocytes, which are the precursor cells of platelets. This interaction is essential for maintaining adequate platelet levels in the blood, which are vital for normal blood clotting and preventing excessive bleeding. Understanding the TPO receptor's function has led to the development of drugs that target this receptor, offering potential therapeutic options for conditions associated with low platelet counts, such as immune thrombocytopenia and chemotherapy-induced thrombocytopenia. The analysis of the target TPO receptor reveals a competitive landscape with multiple companies actively involved in research and development. The highest stage of development is the "Approved" phase, with several companies having drugs in this phase. Thrombocytopenia and Purpura, Thrombocytopenic, Idiopathic are the most common indications for drugs under this target. Small molecule drugs, Fusion proteins, and Biosimilars are progressing most rapidly, indicating intense competition. China, the United States, the European Union, and Japan are the leading countries in terms of development under this target. Further analysis is required to understand the specific R&D progress of each company, the research institutions involved in biosimilar development, and the factors contributing to the progress in China. How do they work?TPO receptor agonists are a type of medication that act on the thrombopoietin (TPO) receptor in the body. Thrombopoietin is a hormone that regulates the production of platelets, which are essential for blood clotting. TPO receptor agonists mimic the action of thrombopoietin and stimulate the production of platelets in the bone marrow. From a biomedical perspective, TPO receptor agonists are used in the treatment of certain medical conditions that result in low platelet counts, such as immune thrombocytopenia (ITP) and chemotherapy-induced thrombocytopenia. By increasing platelet production, these medications help prevent bleeding episodes and improve overall blood clotting function. TPO receptor agonists can be administered orally or through injection, depending on the specific drug. They work by binding to the TPO receptor on the surface of bone marrow cells, which triggers a signaling cascade leading to increased platelet production. These medications are typically used when other treatments, such as corticosteroids, have been ineffective or are not suitable for the patient. It is important to note that TPO receptor agonists may have potential side effects, such as headache, nausea, fatigue, and muscle pain. Close monitoring of platelet counts and regular follow-up with a healthcare professional are necessary during treatment with these medications to ensure optimal effectiveness and safety. List of TPO receptor AgonistsThe currently marketed TPO receptor agonists include: Herombopag OlamineLusutrombopagAvatrombopag MaleateEltrombopag DiolamineRomiplostimRecombinant human thrombopoietin(Shenyang Sunshine Pharmaceutical)Eltrombopag cholineNIP-022NIP-022SCB-219MFor more information, please click on the image below. What are TPO receptor agonists used for?TPO receptor agonists are used in the treatment of certain medical conditions that result in low platelet counts, such as immune thrombocytopenia (ITP) and chemotherapy-induced thrombocytopenia. For more information, please click on the image below to log in and search. How to obtain the latest development progress of TPO receptor agonists?In the Synapse database, you can keep abreast of the latest research and development advances of TPO receptor agonists anywhere and anytime, daily or weekly, through the "Set Alert" function. Click on the image below to embark on a brand new journey of drug discovery!

Medikine’s first therapeutic candidate, MDK-703, an Interleukin-7 (IL-7) mimetic with an extended half-life, will be studied in healthy volunteers, with top-line results expected in the fourth quarter of 2022 Company plans to investigate MDK-703 in solid tumors following completion of the Phase 1 clinical trial MDK-703 is an immunoglobulin Fc fragment-peptide fusion protein incorporating an IL-7 PEPTIKINE™ discovered using Medikine’s innovative platform technology to improve drug properties MENLO PARK, Calif.--(BUSINESS WIRE)-- Medikine, Inc., a biopharmaceutical company developing transformative therapeutics for cancer, autoimmune disorders, and infectious diseases using its novel PEPTIKINE™ technology, today announced it has initiated dosing of MDK-703, an extended half-life IL-7 mimetic. The Phase 1 clinical trial, in healthy volunteers, will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single, ascending intramuscular doses of MDK-703 compared to placebo. Top-line results from the clinical trial are expected in the fourth quarter of 2022. MDK-703 is the First IL-7 Mimetic to be Studied in Humans MDK-703 is an investigational biologic drug incorporating an IL-7 PEPTIKINE discovered using Medikine’s platform technology. Medikine’s PEPTIKINES are substantially smaller molecules than the cytokines they emulate, structurally unrelated to natural cytokines, engineered for low immunogenicity, and readily amenable to incorporating targeting and other pharmacological features. IL-7 is a cytokine critical for the development and maintenance of T cells, including enhancing generation, function, and survival of memory T cells. In oncology, treatments with these biological attributes, alone or in combination with other immune-based therapies, may enhance the rate, depth, and durability of clinical response. In addition to these anti-tumor properties, MDK-703 has the beneficial property that it will not generate anti-drug antibodies (ADAs) that neutralize native IL-7, an issue previously observed with IL-7 agents that have been studied in humans. The company plans to investigate MDK-703 in solid tumors following the completion of the Phase 1 clinical trial in healthy volunteers. Medikine has shown that MDK-703 behaves similarly to IL-7 in human immune cell in vitro studies and in humanized mice and non-human primates in vivo studies. In addition to increasing the total number of CD8 T cells and CD4 T cells, MDK-703 increased the number of memory T cells, particularly T memory stem (TSCM) cells. TSCM cells are a subset of memory T cells that have the potential to self-renew and differentiate, thereby reconstituting the entire spectrum of memory and effector cell types.1 In addition, MDK-703 had negligible-to-no impact on the expansion of CD4 T regulatory and natural killer (NK) cell populations. “Based on preclinical data, we believe that MDK-703 has best-in-class potential, with biology consistent with facilitating the differentiation, maintenance, and survival of the T cell subsets that are critical for durable anti-tumor activity in humans. The initial study of MDK-703 in healthy subjects is designed to provide important proof-of-pharmacology that is relevant to cancer treatment, including the impact on specific T-cell populations and the lack of neutralizing IL-7 ADAs,” commented Dr. Joseph Leveque, president and chief medical officer of Medikine. Medikine Applies Expertise in PEPTIKINE Technology to Next-Generation Cytokine Drug Development Medikine’s founding team of Ronald W. Barrett, Ph.D. (chief executive officer and chairman of the Board), William J. Dower, Ph.D. (scientific advisor), and Michael C. Needels, Ph.D. (chief technology officer), have worked together since the 1990s. They are experts in peptide library technology and its application to the discovery of cytokine receptor agonists and antagonists. Their groundbreaking work at the Affymax Research Institute resulted in the discovery of peptide mimetics of erythropoietin and thrombopoietin. At Medikine, they have been taking this concept to a new level of sophistication and utility by engineering novel PEPTIKINES for multi-subunit cytokine receptors. As Medikine moves from PEPTIKINE discovery research into clinical development, the company’s leadership team is well prepared with the recent addition of Joseph Leveque, M.D., as president and chief medical officer, and Marcos Milla, Ph.D., as interim chief scientific officer. Dr. Leveque previously has held senior executive and therapeutic area leadership roles at Mirati Therapeutics, Synthorx, ARMO BioSciences, Bristol Meyers Squibb, and Amgen. Dr. Milla is currently a venture partner at Samsara BioCapital and was previously chief scientific officer at Synthorx, head of Therapeutics Discovery at Adaptive Biotechnologies, and head of Emerging Science and Innovation of Janssen Discovery Sciences. Research by Drs. Leveque and Milla at Synthorx to discover and develop a “non-alpha” IL-2 derivative led to Synthorx’s acquisition by Sanofi for $2.5 billion in late 2019. Dr. Milla commented, “With its versatile drug discovery platform, Medikine is well-positioned to develop transformative therapies for cancer and other serious diseases. I am particularly intrigued by the pharmacology of MDK-703, with its unique action on key T cell memory populations of great importance to find and destroy cancer cells―one that I believe could offer advantages over other cytokine-based therapies in development. I am thrilled to help realize the possibilities of this lead program and the rest of Medikine’s pipeline.” Medikine’s Pipeline of PEPTIKINE Therapeutics In addition to its lead product candidate, MDK-703, Medikine also has identified novel PEPTIKINES that activate the IL-2/15βγ receptor complex. The company is exploring their use for engineering bispecific cytokine mimetics with differentiated pro including an IL-7 and IL-2/15βγ dual receptor agonist, and a cell-targeted IL-2/15βγ attenuated receptor agonist. Medikine also has identified multiple high-affinity peptide ligands for both subunits of the IL-18 receptor that do not bind to the IL-18 decoy soluble receptor. Medikine is exploring the use of these peptide ligands as part of IL-18 PEPTIKINES. ABOUT MEDIKINE Medikine is a biopharmaceutical company with a mission to transform the discovery of oncology, autoimmune disorder, and infectious disease therapeutics by employing a versatile drug discovery platform that generates modular “PEPTIKINES”- peptide mimetics of cytokines that are smaller in molecular size than, and structurally unrelated to, the natural cytokines they emulate. These PEPTIKINES are engineered for low immunogenicity and are readily amenable to the incorporation of targeting and other pharmacological features. Medikine’s lead candidate, MDK-703, currently in a Phase 1 clinical trial in healthy volunteers, is an IL-7 PEPTIKINE fused to an immunoglobulin Fc-domain. MDK-703 emulates the beneficial properties of IL-7, a cytokine critical for maintenance of T cell responses. For more information, please visit . REFERENCES Gattinoni L, Speiser D, Lichterfeld M, et al. T memory stem cells in health and disease. Nat Med. 2017;23:18–27. doi:10.1038/nm.4241