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All three sickle cell participants treated with briquilimab successfully received a neutrophil transplant within 12-16 days
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The first two participants with peripheral blood chimerism 60 days after allogeneic stem cell transplantation achieved 100% donor myeloid chimerism
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The first participant treated has a total hemoglobin level of 13.3 g/dL at five months of follow-up, increased from 8 to 9 g/dL at baseline
REDWOOD CITY, Calif., Jan. Feb. 3, 2023 (GLOBE NEWSWIRE) — Jasper Therapeutics, Inc. (NASDAQ: JSPR), a biotechnology company focused on transforming the field of hematopoietic stem cell therapies, announced today Today positive clinical data from the first three participants in an investigator-initiated Phase 1/2 clinical trial (NCT05357482) evaluating the addition of briquilimab (formerly known as JSP191), Jasper’s anti-c-KIT monoclonal antibody, to an existing bone marrow transplant regimen (NCT00061568) in people with sickle cell disease (SCD) and beta-thalassemia considered to be at high risk of complications or ineligible for standard myeloablative hematopoietic stem cell transplantation. The addition of briquilimab is being investigated as a potential way to achieve a higher percentage of stem cell transplantation from healthy donors (donor chimerism) without increased toxicity. The Phase 1/2 clinical study is being led by Dr. John F. Tisdale, Director of the Cellular and Molecular Therapeutics Laboratory, NHLBI.
“While stem cell infusion with healthy donor stem cells or genetically corrected cells are potentially curative options for sickle cell disease and beta-thalassemia, they are both limited by the toxicity of current conditioning regimens using busulfan. or melphalan, which are often cited as the biggest safety concerns.” risks to transplant patients and physicians,” said Ronald Martell, President and CEO of Jasper Therapeutics. “With briquilimab, we hope to provide a highly targeted conditioning regimen to directly address conditioning toxicity as a barrier limiting patients’ ability to access curative hematopoietic stem cell therapies.”
For sickle cell disease and beta-thalassemia, transplanting healthy stem cells from a donor is a multi-step process. After donor cells are collected, a human subject’s existing stem cells must be removed from the bone marrow to make room for the transplanted cells, which is known as bone marrow conditioning. Then the newly transplanted cells must survive and replicate in the bone marrow, which is known as bone marrow transplantation. The extent of the transplant is measured by the proportion of donor cells and the human subject’s own cells, which is known as donor chimerism. As demonstrated, improving chimerism is crucial to lead to a sufficient proportion of healthy donor stem cells that produce healthy red blood cells and reverse the sickle phenotype after stem cell transplantation.
The primary objective of the study is to determine whether the addition of briquilimab would increase the proportion of patients with donor myeloid chimerism ≥98% at 1 year post-transplant. Briquilimab has the potential to improve disease-free survival in combination with low-dose irradiation as part of a transplant conditioning regimen. The study is currently in registration with the NHLBI.
In this study, briquilimab was added to the NHLBI regimen of alemtuzumab, low-dose irradiation, and sirolimus prior to infusion of mobilized peripheral blood cells from related donors compatible with human leukocyte antigen. All three sickle cell study participants treated with briquilimab successfully burned without any serious briquilimab-related adverse events. Participant 1 obtained a neutrophil transplant 12 days post-transplant and a platelet transplant 17 days post-transplant. Participant 2 obtained a neutrophil transplant at 12 days and a platelet transplant at 10 days. Participant 3 obtained a neutrophil transplant at 16 days and a platelet transplant at 8 days. The first two participants with peripheral blood chimerism achieved 100% donor myeloid chimerism 60 days post-transplant. Five months after the transplant, the first participant treated with briquilimab had a total hemoglobin of 13.3 g/dL, increased from 8 to 9 g/dL at baseline.
About Briquilimab (formerly known as JSP191)
Briquilimab is a targeted monoclonal antibody that inhibits the c-KIT cell surface receptor, also known as CD117. It is currently being evaluated as a conditioning agent for cell and gene therapies, as well as a stand-alone therapy. To date, briquilimab has a demonstrated efficacy and safety profile in 130 treated subjects and healthy volunteers, with clinical results as a conditioning agent in severe combined immunodeficiency (SCID), acute myeloid leukemia (LAM), myelodysplastic syndromes (MDS), Fanconi anemia (FA) and sickle cell anemia (SCD). Additionally, briquilimab is advanced as a transformational non-genotoxic conditioning agent for gene therapy and as a primary therapeutic in patients with low-risk MDS. Clinical studies also suggest that briquilimab can be used as a primary therapy to treat mast cell diseases such as chronic spontaneous urticaria (CSU), chronic inducible urticaria (CIndU), and allergic asthma.
About Jasper Therapeutics
Jasper Therapeutics is a clinical-stage biotechnology company focused on unlocking access to healing therapies by targeting and eliminating diseased stem cells. Jasper’s lead program is briquilimab, a first-in-class monoclonal antibody targeting c-KIT (CD117), an important receptor found on stem cells and mast cells. In parallel, Jasper is advancing its mRNA cellular programming platform which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell transplants by transiently modifying stem cells with mRNA, augmenting them for treat several blood and bone marrow diseases. Both innovative programs have the potential to enable curative therapies for more patients with life-threatening cancers, genetic disorders and inflammatory diseases. For more information, visit us at jaspertherapeutics.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”, “can”, “will”, “estimate”, “continue”, “anticipate”, “intend”, “expect”, “should”, “would”, “plan”, “predicts”, “potential”, “appears”, “seeks”, “future”, “outlook” and similar expressions that predict or indicate future events or trends or are not statements of historical matters. Such forward-looking statements include , but not limited to, statements regarding the potential long-term benefits of hematopoietic stem cell transplantation after targeted conditioning with briquilimab in the treatment of sickle cell disease and beta-thalassemia and the ability of Jasper to po attempt to deliver an untargeted stem cell transplant. genotoxic conditioning agent to patients with these indications. These statements are based on various assumptions, whether or not identified in this press release, and on Jasper’s current expectations and are not predictions of its actual performance. These forward-looking statements are provided for informational purposes only and are not intended to serve as, and should not be relied upon by, any investor as a guarantee, assurance, prediction or definitive statement of fact or probability. Actual events and circumstances are difficult or impossible to predict and will differ from the assumptions. Many real events and circumstances are beyond Jasper’s control. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that potential product candidates developed by Jasper may not progress through clinical development or receive required regulatory approvals on schedule or at all; risks related to uncertainty regarding the regulatory path for Jasper’s product candidates; the risk that clinical trials may not confirm the safety, potency or other product characteristics described or implied in this press release; the risk that Jasper may not be able to successfully commercialize or achieve market acceptance of its product candidates; the risk that Jasper’s product candidates will not benefit patients or be successfully marketed; the willingness of patients to try new therapies and the willingness of physicians to prescribe those therapies; the effects of competition on Jasper’s business; the risk that third parties upon which Jasper depends for laboratory, clinical development, manufacturing and other critical services may not operate satisfactorily; the risk that Jasper’s business, operations, clinical development plans and timelines and supply chain will be affected by the effects of health outbreaks, including the ongoing COVID-19 pandemic; the risk that Jasper may not be able to obtain and maintain sufficient intellectual property protection for its survey products or infringe the intellectual property protection of others; and other risks and uncertainties stated from time to time in Jasper’s filings with the SEC. Should any of these risks materialize, or should Jasper’s assumptions prove incorrect, actual results could differ materially from the results implied by such forward-looking statements. Although Jasper may choose to update these forward-looking statements at some time in the future, Jasper expressly disclaims any obligation to do so. These forward-looking statements should not be taken to represent Jasper’s assessments as of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed on forward-looking statements.
contacts:
John Mullaly (investors)
LifeSci Advisors
617-429-3548
jmullaly@lifesciadvisors.com
Jeet Mahal (investors)
Therapeutic Jasper
650-549-1403
jmahal@jaspertherapeutics.com
Lauren Barbiero (media)
real chemistry
646-564-2156
lbarbiero@realchemistry.com
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