Jasper Therapeutics, Inc., a new biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies, today announced the launch of the company with a $35 million total Series A financing. Abingworth LLP and Qiming Venture Partners USA served as lead investors, with further investment from Surveyor Capital (a Citadel company) and participation from Alexandria Venture Investments, LLC. The proceeds will be used to advance the clinical development of the company's lead product candidate, JSP191, which is designed to replace or reduce the toxicity of chemotherapy and radiation therapy as a conditioning regimen to prepare patients for hematopoietic cell transplant.

Jasper's development of JSP191 is also supported by a collaboration with the California Institute for Regenerative Medicine (CIRM), which has been funding the program and is committed to providing a total of $23 million in grant support. As part of the Series A financing, Amgen, which discovered JSP191 (formerly AMG191), has licensed worldwide rights to Jasper that also include translational science and materials from Stanford University.

Jasper was co-founded by Judith Shizuru, M.D., Ph.D., a hematopoietic stem cell transplant expert at Stanford University, and Susan Prohaska, Ph.D., a Stanford University-trained immunologist, stem cell biologist and early-stage drug development professional. Dr Shizuru's CIRM-funded lab advanced the understanding of the ability of anti-CD117 to impact hematopoietic stem cells and, together with the Lucile Packard Children's Hospital Stanford and University of California, San Francisco (UCSF) pediatric transplant teams, was the first to study an anti-CD117 antibody in the clinic as a conditioning agent. That humanized antibody, now called JSP191, was first studied for conditioning for transplant in immune-deficient patients in collaboration with Amgen, UCSF and CIRM.

"Stem cell transplantation is a potential curative therapy for people with hematologic cancers, autoimmune diseases, and debilitating genetic diseases. However, the pre-transplant conditioning required to prepare patients for transplant involves highly toxic chemotherapy, which can be life-threatening and limits the number of people who are able to benefit," said Dr. Shizuru, co-founder and member of the Board of Directors of Jasper Therapeutics. "JSP191 is the only anti-CD117 antibody to demonstrate safety and efficacy in severely ill patients receiving stem cell transplant in the clinic. We plan to expand clinical development to patients receiving transplants for acute myeloid leukemia/ myelodysplastic syndrome or autoimmune diseases and to patients receiving stem cell-directed gene therapies."

Dr. Shizuru added, "With an experienced executive team of biotech veterans and a strong syndicate of healthcare-focused investors, Jasper Therapeutics is well positioned to achieve our vision of building a leading biotech company starting with JSP191 and expanding to other novel therapies for immune modulation, graft engineering and cell and gene therapies."

JSP191 is currently being evaluated in an ongoing Phase 1 clinical trial as a conditioning agent to enable stem cell transplantation in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that failed. This severe genetic immune disorder leaves patients without a functioning immune system. Interim results of the study will be presented in an oral presentation (abstract #800) on Monday, December 9, at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition in Orlando, Fla. Clinical studies to evaluate the safety and efficacy of JSP191 as a conditioning agent in patients undergoing hematopoietic cell therapy for hematologic cancers are planned for 2020.

Founding Management Team

William Lis, Executive Chairman and Interim Chief Executive Officer (CEO), responsible for leading Jasper's Series A financing. Previously, Mr. Lis served as Chief Executive Officer and a Director of Portola Pharmaceuticals, Inc. Under his leadership, Portola successfully grew from a discovery-stage company to a fully integrated R&D and commercial organization, and independently discovered and developed Andexxa® and Bevyxxa® through commercial launch, and advanced cerdulatinib into clinical development. He led corporate partnerships and private and public financings including an initial public offering in 2013. The company's valuation grew from $250 million to $2.8 billion during his tenure. Mr. Lis held executive positions at Scios, Inc. (a Johnson & Johnson company) where he last served as Sr. Vice President of Business Development and New Product Development, having led efforts for the in-licensing, strategic development and pre-commercial launch for Xarelto®; He also held positions of increasing responsibility at Millennium Pharmaceuticals, Inc. (previously COR Therapeutics, Inc.) and Rhone Poulenc Rorer in sales, marketing, medical affairs and business development. Mr. Lis served as a member of the Bio Board of Directors for Emerging Companies and is currently an independent Director of Eidos Therapeutics, Inc. and Zai Laboratories, Inc.
Jeet Mahal, M.B.A., M.E., M.B., Chief Financial Officer and Chief Business Officer. Mr. Mahal joined Jasper Therapeutics from Portola Pharmaceuticals, where he worked for 11 years and held a number of positions of increasing leadership, including Vice President, Business Development, and Vice President, Strategic Marketing. He led the successful execution of multiple business development partnerships for Andexxa, Bevyxxa and cerdulatinib and played a key role in the company's capital financings including the initial public offering and multiple royalty transactions. Earlier in his career, he was Director, Business and New Product Development at Johnson & Johnson on the Xarelto development and strategic marketing team. He started his career in the drug development laboratories at COR Therapeutics.
Susan Prohaska, Ph.D., Co-Founder and Vice President of Operations & Program Management. Dr. Prohaska is a Stanford University-trained scientist in immunology and stem cell biology with over 20 years of experience in preclinical and early clinical research in immunology, hematopoiesis, oncology and leading monoclonal antibody development from discovery through early clinical stage. As Program Officer and Associate Director for Translational Programs at the Stanford Institute of Stem Cell Biology and Regenerative Medicine, and Program Director for JSP191 development for the Division of Blood and Marrow transplant, she managed CMC, toxicology, regulatory, preclinical and clinical operations for anti-CD47 and JSP191 drug development programs at Stanford University. Prior to founding Jasper, she served as Associate Director for CMC overseeing clinical manufacturing of FSI-5F9 (now magrolimab) at Forty Seven, Inc. Prior to the recruitment of Mr. Lis, Dr. Prohaska served as founding CEO of Jasper.
Wendy Pang, M.D., Ph.D., Executive Director, Discovery Research, and Early Clinical Development. Dr. Pang will be joining Jasper Therapeutics from Stanford University, where she is an Instructor in the Division of Blood and Marrow Transplantation. A physician-scientist with more than 18 years of experience conducting translational research in hematology/oncology and hematopoietic stem cell biology and transplantation, she was the lead scientist in the preclinical drug development of an anti-CD117 antibody at Stanford. She was the lead author on the proof-of-concept studies showing that an anti-CD117 antibody therapy targets disease-initiating human hematopoietic (blood cell-forming) stem cells in myelodysplastic syndrome (MDS). She has authored numerous publications on the characterization of hematopoietic stem and progenitor cell behavior in hematopoietic malignancies, including MDS and acute myeloid leukemia (AML), and in hematopoietic stem cell transplantation.
Robert Sikorski, M.D., Ph.D., Executive Consultant and Acting Chief Medical Officer. Dr. Sikorski currently serves as the Managing Director of Woodside Way Ventures, a consulting and investment firm that helps biotechnology companies and investors advance lifesaving technologies through clinical development. Prior to that, he was Chief Medical Officer of Five Prime Therapeutics. Earlier in his career, he played a leading role in building MedImmune's oncology portfolio through partnering and acquisition efforts. Before joining Medimmune, he led late-stage clinical development and post-marketing efforts for several commercial drugs and drug candidates at Amgen. Dr. Sikorski began his career as a Howard Hughes Research Fellow and Visiting Scientist at the National Cancer Institute and the National Human Genome Research Institute in the laboratory of Nobel Laureate Harold Varmus.
Dr. Shizuru and Mr. Lis are joined on the Jasper Therapeutics' Board of Directors by Kurt von Emster, Managing Partner of Abingworth LLP, and Anna French, Ph.D., Principal at Qiming Venture Partners USA. Dr. Prohaska is a Board observer.

"With our investment in this program, we're able to realize our mission of fast-tracking stem cell treatments by helping academic researchers rapidly advance the most promising discoveries in the lab into the clinics and to drug development with commercialization partners," said Maria T. Millan, M.D., President and CEO of CIRM. "Jasper's two co-founders took a novel antibody with unique properties and moved it from the bench to the bedside relatively quickly, and we're thrilled to partner with this talented team to potentially impact a broad group of people who could benefit from stem cell therapy."

About Stem Cell Transplantation

Blood-forming, or hematopoietic, stem cells are cells that reside in the bone marrow and are responsible for the generation and maintenance of all blood and immune cells. These stem cells can harbor inherited or acquired abnormalities that lead to a variety of disease states, including immune deficiencies, blood disorders or hematologic cancers. Successful transplantation of hematopoietic stem cells is the only cure for most of these life-threatening conditions. Replacement of the defective or malignant hematopoietic stem cells in the patient's bone marrow is currently achieved by subjecting patients to toxic doses of radiation and/or chemotherapy that cause DNA damage and lead to short- and long-term toxicities, including immune suppression and prolonged hospitalization. As a result, many patients who could benefit from a stem cell transplant are not eligible. New approaches that are effective but have minimal to no toxicity are urgently needed so more patients who could benefit from a curative stem cell transplant could receive the procedure.

Safer and more effective hematopoietic cell transplantation regimens could overcome these limitations and enable the broader application of hematopoietic cell transplants in the cure of many disorders. These disorders include hematologic cancers (e.g., myelodysplastic syndrome [MDS] and acute myeloid leukemia [AML]), autoimmune diseases (e.g., lupus, rheumatoid arthritis, multiple sclerosis and Type 1 diabetes), and genetic diseases that could be cured with genetically-corrected autologous stem cells (e.g., severe combined immunodeficiency syndrome [SCID], sickle cell disease, beta thalassemia, Fanconi anemia and other monogenic diseases).

About JSP191

JSP191 (formerly AMG191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in an animal model of MDS. This creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 80 healthy volunteers and patients. It is currently being evaluated as a sole conditioning agent in a Phase 1 dose-escalation trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID, which is curable only by this type of treatment. For more information about the design of the clinical trial, visit www.clinicaltrials.gov (NCT02963064). Clinical development of JSP191 will be expanded to also study patients with AML or MDS who are receiving hematopoietic cell transplant.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies. Jasper Therapeutics' lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a stem cell transplant. For more information, please visit us at www.jaspertherapeutics.com.