Jasper Therapeutics Announces European Union Orphan Drug Designation for Briquilimab as a Conditioning Treatment for Patients Prior to Receiving a Stem Cell Transplant
“The EMA’s decision to grant orphan drug designation to briquilimab highlights the clear need for non-genotoxic, targeted conditioning for patients receiving hematopoietic stem cell transplant,” said
Jasper is currently conducting clinical studies of briquilimab as a conditioning agent prior to hematopoietic stem cell transplant in patients with SCID, a rare, life-threatening, pediatric disorder affecting an estimated 1/58,000 births in the general population, and separately in patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). In these diseases, prognosis is poor for patients and transplant rates are low due to the highly toxic conditioning required.
Jasper’s ongoing clinical trial in SCID is evaluating briquilimab as a conditioning agent to enable stem cell transplantation in patients who are either transplant-naive or who received a prior stem cell transplant with a poor outcome. Jasper presented data from this study at multiple scientific conferences, which demonstrated that briquilimab has been well tolerated with no treatment-related adverse events across multiple patients ranging from 3 months to 38 years old. Successful stem cell engraftment and immune reconstitution have also been observed.
Jasper has also presented data from a study that has demonstrated a tolerable safety profile and full donor chimerism in 24 of 24 patients with AML or MDS. To date, there have been no reports of briquilimab-related significant adverse events, classical acute grade II-IV graft versus host disease, or transplant related mortality within 100 days. MDS are a group of disorders in which immature blood-forming cells in the bone marrow become abnormal and do not make new blood cells or make defective blood cells, leading to low numbers of normal blood cells, especially red blood cells. In about one in three patients, MDS can progress to AML, a rapidly progressing cancer of the bone marrow cells. Both are diseases of the elderly with high mortality.
About Orphan Drug Designation in
Orphan drug designation in the
About Briquilimab (formerly known as JSP191)
Briquilimab is a targeted, monoclonal antibody that inhibits the cell-surface receptor c-KIT, also known as CD117. It is currently being evaluated as a conditioning agent for cell and gene therapies, as well as a standalone therapy. 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), myelodysplastic syndromes (MDS), Fanconi anemia (FA), and sickle cell disease (SCD). In addition, briquilimab is being advanced as a transformational non-genotoxic conditioning agent for gene therapy and as a primary therapeutic in low-risk MDS patients. Clinical studies also suggest briquilimab can be used as a primary therapeutic to treat mast cell diseases such as chronic spontaneous urticaria (CSU), chronic inducible urticaria (CIndU), and allergic asthma.
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 the potential long-term benefits of hematopoietic stem cells engraftment following targeted briquilimab conditioning. 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. In addition, a positive opinion from the EMA on Jasper’s application for orphan drug designation for briquilimab is not a guarantee of trial success or approval. 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 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, including the ongoing COVID-19 pandemic; 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
Source: Jasper Therapeutics