Severe Combined Immunodeficiency

REDWOOD CITY, Calif., Oct. 24, 2024 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a clinical stage biotechnology company focused on development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) to address mast cell driven diseases such as chronic spontaneous urticaria (CSU), chronic inducible urticaria (CIndU) and asthma, is presenting data from preclinical studies evaluating briquilimab at the American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, taking place October 24-28, 2024, in Boston, Massachusetts. “We are pleased to present promising preclinical results from multiple studies at ACAAI this year,” said Wendy Pang, M.D., Ph.D., Senior Vice President, Research and Translational Medicine. “Both the in-vitro data and the non-human primate data generated in these studies provide further support for the pharmacodynamics of briquilimab, particularly its ability to bind c-Kit and drive mast cell depletion with relatively high potency.” Details of the presentations are as follows: Abstract Title: Briquilimab is Well-Tolerated and Effectively Depletes Tissue Resident Mast Cells in Non-Human PrimatesAbstract ID: 8271ePoster ID: R056 Presentation Date / Time: Friday, October 25, 2024; 3:45 p.m. - 4:00 p.m. EDTLocation: Exhibit Hall A Abstract Title: Briquilimab Potently Inhibits Stem Cell Factor (SCF)/c-Kit Signaling, and Mast Cell (MC) Degranulation and SurvivalAbstract ID: 8282ePoster ID: R058 Presentation Date / Time: Friday, October 25, 2024; 4:15 p.m. - 4:30 p.m. EDTLocation: Exhibit Hall A Abstract Title: Briquilimab Potently Blocks Stem Cell Factor (SCF)/c-Kit Signaling in Primary Human Mast CellsAbstract ID: 8298ePoster ID: R059 Presentation Date / Time: Friday, October 25, 2024; 4:30 p.m. - 4:45 p.m. EDTLocation: Exhibit Hall A Copies of the presentations will be available on the ACAAI website as well as the Events & News – Events page on the Jasper Therapeutics IR website. About BriquilimabBriquilimab is a targeted aglycosylated monoclonal antibody that blocks stem cell factor from binding to the cell-surface receptor c-Kit, also known as CD117, thereby inhibiting signaling through the receptor. This inhibition disrupts the critical survival signal, leading to the depletion of the mast cells via apoptosis which removes the underlying source of the inflammatory response in mast cell driven diseases such as chronic urticaria. Jasper is currently conducting clinical studies of briquilimab as a treatment in patients with CSU or with CIndU and is initiating a clinical study in patients with asthma. Briquilimab is also currently in clinical studies as a treatment for patients with LR-MDS and as a conditioning agent for cell therapies for rare diseases. To date, briquilimab has a demonstrated efficacy and safety profile in more than 160 dosed participants and healthy volunteers, with clinical outcomes in CIndU, and as a conditioning agent in severe combined immunodeficiency (SCID), acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), Fanconi anemia (FA), and sickle cell disease (SCD). About JasperJasper is a clinical-stage biotechnology company developing briquilimab, a monoclonal antibody targeting c-Kit (CD117) as a therapeutic for chronic mast and stem cell diseases such as chronic urticaria, asthma and lower to intermediate risk MDS and as a conditioning agent for stem cell transplants for rare diseases such as SCD, FA and SCID. To date, briquilimab has a demonstrated efficacy and safety profile in more than 160 dosed participants and healthy volunteers, with clinical outcomes in CIndU and as a conditioning agent in SCID, AML, MDS, FA, and SCD. For more information, please visit us at www.jaspertx.com. Forward-Looking StatementsCertain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “should,” “would,” “plan,” “predict,” “potential,” “seem,” “seek,” “future,” “outlook” and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding briquilimab’s potential, including with respect to its potential in mast cell driven diseases such as CSU, CIndU, and asthma and its ability to bind c-Kit and drive mast cell depletion with relatively high potency; and Jasper’s presentation at ACAAI. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of Jasper and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Many actual events and circumstances are beyond the control of Jasper. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that the potential product candidates that Jasper develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that prior test, study and trial results may not be replicated in continuing or future studies and trials; the risk that Jasper will be unable to successfully market or gain market acceptance of its product candidates; the risk that prior study results may not be replicated; the risk that Jasper’s product candidates may not be beneficial to patients or successfully commercialized; patients’ willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jasper’s business; the risk that third parties on which Jasper depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jasper’s business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics; the risk that Jasper will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; and other risks and uncertainties indicated from time to time in Jasper’s filings with the SEC, including its Annual Report on Form 10-K for the year ended December 31, 2023 and subsequent Quarterly Reports on Form 10-Q. If any of these risks materialize or Jasper’s assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. While Jasper may elect to update these forward-looking statements at some point in the future, Jasper specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Jasper’s assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements. Contacts: Joyce Allaire (investors)LifeSci Advisors617-435-6602jallaire@lifesciadvisors.com Alex Gray (investors)Jasper Therapeutics650-549-1454 agray@jaspertx.com Lauren Walker (media)Real Chemistry646-564-2156lbarbiero@realchemistry.com

REDWOOD CITY, Calif., Oct. 23, 2024 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a clinical stage biotechnology company focused on development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) to address mast cell driven diseases such as chronic spontaneous urticaria (CSU), chronic inducible urticaria (CIndU) and asthma, today announced that it is commencing an open-label extension study in chronic urticarias that will roll over patients from the BEACON and SPOTLIGHT studies upon completion of their initial follow up period. Additionally, Jasper also announced it has obtained regulatory clearance in the US and in the EU to further expand the BEACON study in CSU by adding a 360mg single-dose cohort (n=4), and that enrollment in this cohort has commenced. Jasper continues to plan to report initial data from all doses of the BEACON study up through 240mg in CSU during the week of January 6th, 2025. Data from the newly added 360mg single-dose cohort is expected to be reported in the first half of 2025. “I am very pleased with the ability of our clinical and regulatory teams to efficiently advance our clinical development plan in support of generating a robust data set in chronic urticaria,” said Ronald Martell, President and Chief Executive Officer of Jasper. “The addition of the 360mg single-dose cohort in the BEACON study in CSU will allow us to evaluate another potential loading dose leveraging the ongoing clinical trial, and the rollover extension study will allow us to gather longer-term safety and efficacy data at therapeutic doses for patients in both BEACON and SPOTLIGHT. The expanded breadth of clinical data provided in CSU and CindU should further support optimal biologic dosing in our registrational studies, planned to commence in the second half of 2025.” About Briquilimab Briquilimab is a targeted aglycosylated monoclonal antibody that blocks stem cell factor from binding to the cell-surface receptor c-Kit, also known as CD117, thereby inhibiting signaling through the receptor. This inhibition disrupts the critical survival signal, leading to the depletion of the mast cells via apoptosis which removes the underlying source of the inflammatory response in mast cell driven diseases such as chronic urticaria. Jasper is currently conducting clinical studies of briquilimab as a treatment in patients with CSU or with CIndU and is initiating a clinical study in patients with asthma. Briquilimab is also currently in clinical studies as a treatment for patients with LR-MDS and as a conditioning agent for cell therapies for rare diseases. To date, briquilimab has a demonstrated efficacy and safety profile in more than 160 dosed participants and healthy volunteers, with clinical outcomes in CIndU, and as a conditioning agent in severe combined immunodeficiency (SCID), acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), Fanconi anemia (FA), and sickle cell disease (SCD). About Jasper Jasper is a clinical-stage biotechnology company developing briquilimab, a monoclonal antibody targeting c-Kit (CD117) as a therapeutic for chronic mast and stem cell diseases such as chronic urticaria, asthma and lower to intermediate risk MDS and as a conditioning agent for stem cell transplants for rare diseases such as SCD, FA and SCID. To date, briquilimab has a demonstrated efficacy and safety profile in more than 160 dosed participants and healthy volunteers, with clinical outcomes in CIndU and as a conditioning agent in SCID, AML, MDS, FA, and SCD. For more information, please visit us at www.jaspertx.com. Forward-Looking Statements Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “intend,” “expect,” “should,” “would,” “plan,” “predict,” “potential,” “seem,” “seek,” “future,” “outlook” and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding briquilimab’s potential, including with respect to its potential in mast cell driven diseases such as CSU, CIndU, and asthma; the expected number of participants in the 360mg single-dose cohort; Jasper’s expected timing for presenting initial data from all doses of the BEACON study up through the 240mg dose cohort in CSU and from the 360mg single-dose cohort; Jasper’s expectations regarding the commencement and timing of registrational studies; and Jasper’s expectation that the expanded clinical data should further support optimal biologic dosing in its registrational studies. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of Jasper and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Many actual events and circumstances are beyond the control of Jasper. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that the potential product candidates that Jasper develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that prior test, study and trial results may not be replicated in continuing or future studies and trials; the risk that Jasper will be unable to successfully market or gain market acceptance of its product candidates; the risk that prior study results may not be replicated; the risk that Jasper’s product candidates may not be beneficial to patients or successfully commercialized; patients’ willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jasper’s business; the risk that third parties on which Jasper depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jasper’s business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics; the risk that Jasper will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; and other risks and uncertainties indicated from time to time in Jasper’s filings with the SEC, including its Annual Report on Form 10-K for the year ended December 31, 2023 and subsequent Quarterly Reports on Form 10-Q. If any of these risks materialize or Jasper’s assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. While Jasper may elect to update these forward-looking statements at some point in the future, Jasper specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Jasper’s assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements. Contacts:Alex Gray (investors)Jasper Therapeutics650-549-1454 agray@jaspertx.com Joyce Allaire (investors)LifeSci Advisors617-435-6602jallaire@lifesciadvisors.com Lauren Walker (media)Real Chemistry646-564-2156lbarbiero@realchemistry.com

18 October 2024 – Novo Nordisk today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending approval of Alhemo® (concizumab) as the first once-daily subcutaneous prophylactic treatment for people aged 12 years or older living with haemophilia A or B with inhibitors. Haemophilia is a rare bleeding disorder that impairs the body’s ability to make blood clots, a process needed to stop bleeding. Haemophilia is often treated by replacing the missing clotting factor via intravenous infusions, also known as replacement therapy. However, sometimes the body can produce inhibitors as an immune response to the clotting factors in the therapy, which means replacement therapy does not work and limits treatment options overall. Currently, it is estimated that up to 30% of people living with severe haemophilia A develop inhibitors,1 and 5-10% of those with severe haemophilia B develop inhibitors.2,3 Alhemo® is an anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody that, if approved by the European Commission, will offer the first once-daily subcutaneous prophylactic treatment to people with haemophilia A or B with inhibitors.4 Alhemo® is designed to block a protein called TFPI in the body that stops blood from clotting. By blocking TFPI, Alhemo® ensures the production of thrombin, which helps to clot the blood and prevent bleeding, even when the other clotting factors are missing or deficient. Importantly, this means Alhemo® allows blood clots to happen, even in the presence of inhibitors.5 “The positive opinion from the CHMP for Alhemo® is a major milestone for patients,” said Stephanie Seremetis, chief medical officer for haemophilia at Novo Nordisk. “If approved, Alhemo® would be an important addition to our growing haemophilia portfolio as it offers the potential of everyday prophylaxis to prevent bleeds for people living with haemophilia who have developed the complication of inhibitors. It could alleviate the physical, emotional, and overall treatment burden for people living with haemophilia, as it is delivered in a pre-filled, multi-use, portable pen that can be stored at room temperature for up to four weeks. This could enable patients to have greater confidence in the pursuit of daily activities, which is especially important for people living with haemophilia B with inhibitors, who currently have very limited treatment options.” The CHMP positive opinion is based on data from the phase 3 explorer7 study, which was designed to evaluate the efficacy and safety profile of Alhemo® for people living with haemophilia A or B with inhibitors.6 If approved, Alhemo® will be provided in a portable, pre-mixed and prefilled pen, enabling quick and easy subcutaneous administration with the aim of easing the treatment burden of regular intravenous infusions.7 Novo Nordisk expects a final approval by the European Commission within approximately two months. Alhemo® (concizumab) is an anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody designed to block a protein in the body that stops blood from clotting. By blocking TFPI, Alhemo® ensures the production of thrombin, which helps to clot the blood and prevent bleeding.5 Alhemo® is currently approved in Australia8 and Switzerland9 for the treatment of adolescents and adults (12 years or older) with haemophilia A or B with inhibitors. In Japan,10 Alhemo® is currently approved for the treatment of adolescents and adults (12 years or older) with haemophilia A or B with and without inhibitors and is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes. In explorer7, 133 males (aged 12 years or older) were randomised 1:2 to either no prophylaxis (arm one; ≥24 weeks) or concizumab prophylaxis (arm two; ≥32 weeks) or assigned to concizumab prophylaxis (arm three and four).11 The primary analysis compared the number of treated spontaneous and traumatic bleeding episodes, measured as annualised bleeding rate (ABR), between arms one and two. The results showed an 86% reduction in treated spontaneous and traumatic bleeds when on concizumab prophylaxis, with an estimated mean ABR of 1.7 compared to 11.8 with no prophylaxis.12 The overall median ABR of concizumab was zero, compared to 9.8 for no prophylaxis. The safety and tolerability profile of concizumab in this study was within the expected range.12 Haemophilia is a rare inherited bleeding disorder that impairs the body’s ability to make blood clots, a process needed to stop bleeding. It is estimated to affect approximately 1,125,000 people worldwide.12 Due to the nature of haemophilia being an x-linked recessive disorder, it often presents differently in males compared to females, with roughly 88% of people diagnosed with haemophilia worldwide being male.13,14 There are different types of haemophilia, which are characterised by the type of clotting factor protein that is defective or missing. Haemophilia A is caused by a missing or defective clotting Factor VIII (FVIII), and haemophilia B is caused by a missing or defective clotting Factor IX (FIX). Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat serious chronic diseases, built upon our heritage in diabetes. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 69,000 people in 80 countries and markets its products in around 170 countries. For more information, visit novonordisk.com, Facebook, Instagram, X, LinkedIn and YouTube. References 1 Kim JY, You CW. The prevalence and risk factors of inhibitor development of FVIII in previously treated patients with hemophilia A. Blood Res. 2019 Sep;54(3):204-209. doi: 10.5045/br.2019.54.3.204 2 Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020;26 Suppl 6:1-158. doi:10.1111/hae.14046 3 Male C et al. Inhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B: a PedNet study. Haematologica. 2021 Jan 1;106(1):123-129. doi: 10.3324/haematol.2019.239160. PMID: 31919092; PMCID: PMC7776246 4 Shapiro AD. Concizumab: a novel anti-TFPI therapeutic for hemophilia. Blood Adv. 2021;5(1):279 5 Matsushita T, Shapiro A, Abraham A, et al. Phase 3 Trial of Concizumab in Hemophilia with Inhibitors. N Engl J Med 2023;389:783-794 Available at: https://www.nejm.org/doi/full/10.1056/NEJMoa2216455 Accessed: October 2024 6 Frei-Jones M, Cepo K, d’Oiron R, et al. Subcutaneous concizumab prophylaxis in patients with hemophilia A or B with inhibitors: efficacy and safety results by hemophilia subtype from the phase 3 explorer7 trial. Abstract presented at the 64th American Society of Hematology (ASH) annual meeting and exposition 2022. 7 Kahr Rasmussen et al. The Concizumab Pen-Injector is Easy to Use and Preferred by Hemophilia Patients and Caregivers: A Usability Study Assessing Pen-Injector Handling and Preference. Patient Preference and Adherence 2024:18 1713–1727 8 Therapeutic Goods Administration (TGA), Product Information. Available at: https://www.tga.gov.au/resources/auspar/auspar-alhemo 9 SwissMedic. Switzerland Product Information. Available at: https://www.swissmedic.ch/swissmedic/en/home/about-us/publications/public-summary-swiss-par/public-summary-swiss-par-alhemo.html 10 Japanese Product Information. Latest revision date: June 2024 11 Jiménez-Yuste V, Angchaisuksiri P, Castaman G, et al. Concizumab prophylaxis in patients with haemophilia A or B with inhibitors: Efficacy and safety results from the 32-week primary analysis of the phase 3 explorer7 trial. Presented at ISTH 2022 12 Iorio A, Stonebraker JS, Chambost H, Makris M, Coffin D, Herr C, Germini F; Data and Demographics Committee of the World Federation of Hemophilia. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries. Ann Intern Med. 2019 Oct 15;171(8):540-546. doi: 10.7326/M19-1208. 13 Centers for Disease Control and Prevention. What is Hemophilia? Available at: https://www.cdc.gov/hemophilia/about/?CDC_AAref_Val=https://www.cdc.gov/ncbddd/hemophilia/facts.html Accessed: October 2024 14 Statista. Distribution of people with bleeding disorders worldwide in 2020, by gender. Available at: https://www.statista.com/statistics/495675/percentager-of-people-with-bleeding-disorders-in-worldwide-bygender/#:~:text=This%20statistic%20displays%20the%20percentage,with%20hemophilia%20A%20were%20males Accessed: October 2024 The content above comes from the network. if any infringement, please contact us to modify.