Full Press Release Details
Jasper Therapeutics to Present New Positive
Conditioning in Patients with Fanconi Anemia at the 2023 Fanconi
Anemia Research Fund Scientific Symposium
REDWOOD CITY, Calif., September 21, 2023 (GLOBE
NEWSWIRE) - Jasper Therapeutics, Inc. (Nasdaq: JSPR) ("Jasper"), a biotechnology company focused on developing novel
antibody therapies targeting c-Kit (CD117) to address diseases such as chronic spontaneous urticaria and lower to intermediate risk myelodysplastic
syndromes (MDS) as well as novel stem cell transplant conditioning regimes, today announced new positive Phase 1b data on briquilimab
as a conditioning agent in the treatment of Fanconi Anemia (FA).
The data will be featured in a presentation at
the 2023 Fanconi Anemia Research Fund (FARF) Scientific Symposium, taking place on September 28 - October 1, 2023, in Vancouver,
The ongoing investigator initiated Phase 1/2 clinical
trial is evaluating a conditioning regimen that includes briquilimab as a potential treatment for FA patients in bone marrow failure.
Utilizing briquilimab, the regimen eliminates the need for busulfan chemotherapy or total body irradiation.
"The updated results from the Phase 1b study,
which will be presented at FARF, are very encouraging," said Ronald Martell, President, and Chief Executive Officer of Jasper. "All
three FA patients who underwent conditioning with briquilimab achieved full blood count recovery. The treatment was tolerated without
any complications, leading to remarkable levels of donor chimerism and no briquilimab-related adverse events or toxicities. These outcomes
underscore the potential for briquilimab conditioning regimen to redefine the landscape of FA therapy. We look forward to the expansion
of the study to Phase 2a by Stanford."
The details of the presentation are as follows:
Abstract Title: Radiation and Busulfan-free
Transplant Using JSP191 Antibody-Conditioning and TCR + T-Cell/CD19+ B-Cell Depleted Grafts
Author: Rajni Agarwal, M.D., Professor
of Pediatrics and Stem Cell Transplantation, Stanford Medicine
Jasper thanks its Stanford collaborators, The
Center for Definitive and Curative Medicine and investigators Dr. Rajni Agarwal, Dr. Agnieszka Czechowicz and Dr. Alice Bertaina, for
testing briquilimab safety and efficacy in the allo-HSCT setting in the vulnerable FA patient population.
Briquilimab (formerly JSP191) is a targeted, 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. Jasper intends to start clinical studies of briquilimab as a primary treatment in Chronic Spontaneous Urticaria
and Lower to Intermediate Risk myelodysplastic syndromes (MDS). It is also being studied as a conditioning agent for cell and gene therapies
for rare diseases. To date, briquilimab has a demonstrated efficacy and safety profile in 130 dosed subjects and healthy volunteers, with
clinical outcomes as a conditioning agent in severe combined immunodeficiency (SCID), acute myeloid leukemia (AML), MDS, Fanconi anemia
(FA), and sickle cell disease (SCD).
About Fanconi Anemia
Fanconi Anemia (FA) is a rare but serious blood
disorder that prevents the bone marrow from making sufficient new red blood cells. The disorder can also cause the bone marrow to make
abnormal blood cells. FA typically presents at birth or early in childhood between five and ten years of age. Ultimately, it can lead
to serious complications, including bone marrow failure and severe aplastic anemia. Cancers such as acute myeloid leukemia (AML) and myelodysplastic
syndromes (MDS) are other possible complications. Treatment may include blood transfusions or medicine to create more red blood cells,
but a hematopoietic stem cell transplant (HSCT) is currently the only cure.
About Phase 1/2 clinical trial (NCT04784052)
The Stanford sponsored, investigator initiated
Phase 1/2 study is an open-label clinical trial evaluating briquilimab as a potential treatment for FA patients in bone marrow failure
(BMF) requiring allogeneic transplant. Utilizing briquilimab, the regimen eliminates the need for busulfan chemotherapy or total body
irradiation. Participants with FA with BMF receive allo-HCT with TCR + T-cell/CD19+ B-cell depleted hematopoietic grafts from
10/10 unrelated, 9/10 unrelated or haploidentical family donors. A 0.6 mg/kg dose of briquilimab is administered in combination with standard
FA dosing of anti-thymocyte globulin (ATG), cyclophosphamide, fludarabine, and rituximab as lymphodepletion. The primary outcomes include
safety, efficacy, and engraftment success.
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 and lower to intermediate risk myelodysplastic syndromes (MDS) and as a conditioning agent for stem cell transplants
for rare diseases such as sickle cell disease (SCD), Fanconi anemia (FA) and severe combined immunodeficiency (SCID). To date, briquilimab
has a demonstrated efficacy and safety profile in over 130 dosed subjects and healthy volunteers, with clinical outcomes as a conditioning
agent in SCID, acute myeloid leukemia (AML), MDS, FA, and SCD. In addition, briquilimab is being advanced as a transformational non-genotoxic
conditioning agent for gene therapy.
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 as a treatment for Fanconi Anemia (FA) and FA patients with bone
marrow failure, its potential to eliminate the need for busulfan chemotherapy or total body irradiation, its potential to redefine the
landscape of FA therapy and the expected expansion of the study to Phase 2a. 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. Actual events and circumstances
are difficult or impossible to predict and will differ from assumptions. 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; risks relating to uncertainty regarding the regulatory pathway
for Jasper's product candidates; the risk that prior study results may not be replicated; 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 Jasper will be
unable to successfully market or gain market acceptance of its product candidates; 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, 2022
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.
John Mullaly (investors)
Jeet Mahal (investors)
Lauren Barbiero (media)