SAN MATEO, Calif. — CARGO Therapeutics, Inc. (CARGO), a biotechnology company advancing a next generation of CAR T-cell therapies for cancer, today announced the close of a $200 million oversubscribed and upsized Series A financing. The financing was co-led by Third Rock Ventures, RTW Investments, LP and Perceptive Xontogeny Venture Fund, and includes additional new investors Nextech, Janus Henderson Investors, Ally Bridge Group, Wellington Management, funds and accounts advised by T. Rowe Price Associates, Inc., Cormorant Asset Management and Piper Heartland. Founding investor Samsara BioCapital and existing seed investors Red Tree Venture Capital and Emerson Collective also participated in the financing. CARGO is advancing what will potentially be a first and best-in-class autologous CD22 chimeric antigen receptor T-cell (CAR T) therapy as well as a pipeline of next generation CAR T-cell therapies for cancer using its proprietary cell engineering platform technologies.
The proceeds from the financing will be used to advance CARGO’s autologous CD22 CAR T-cell therapy candidate, CRG-022, through a pivotal multicenter Phase 2 trial in patients with LBCL whose disease has relapsed or is refractory to CD19 CAR T-cell therapy. CARGO also intends to advance its proprietary platform technologies and discovery-stage programs to deliver better outcomes for patients with cancer who have high unmet need.
“Approximately sixty percent of patients with LBCL who are treated with CD19 CARs do not experience durable responses due to disease resistance. Additionally, many patients lack access to these potentially curative therapies due to challenges related to manufacturing and reimbursement. CARGO Therapeutics is building upon deep expertise in understanding resistance to CAR T-cell therapies to deliver next generation cell products for patients,” said Gina Chapman, CEO of CARGO Therapeutics. “Our recent accomplishments and this new funding enable us to build a truly differentiated platform that is poised to meaningfully advance the cell therapy field. We are extremely pleased to have the support of seasoned new and current investors who share our vision of developing the next generation of best-in-class CARs that have the potential to extend life for many more patients.”
The ongoing Phase 1 clinical trial (NCT04088890) is evaluating CD22 CAR T-cell therapy in patients with LBCL that is R/R to CD19 CAR cell therapy. Initial results from this trial were reported in Blood in April 2021. Updated results were presented at the Transplantation & Cellular Therapies meetings of ASTCT and CIBMTR on February 17, 2023 at the “Best Abstracts” session. Forty-one patients were enrolled, 40 underwent leukapheresis and 38 had successful manufacturing (95%). Thirty-eight participants were dosed at two different levels and had a median follow-up of 18.4 months. Infused patients were a median age of 65 years old, received a median of 4 prior lines of therapy (range 3-8), and 37 (97%) progressed after prior CAR19 therapy. The majority of patients had diffuse LBCL.
The overall response (ORR) and complete response (CR) rates are 68% and 53% in the 38 patients. Only 1 of the 20 patients who achieved a CR has relapsed to date. At this interim analysis, the median overall survival was 22.5 months. Overall the CD22 CAR T toxicity profile was manageable.
On the basis of the Phase 1 data, CD22 CAR has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA), which is intended to expedite the development and review of drugs for patients with significant unmet need. In 2023, CARGO plans to commence a pivotal multicenter Phase 2 clinical trial to study the safety and efficacy of CRG-022 in patients with LBCL that is R/R to CD19 CAR cell therapy. CARGO also plans to study CRG-022 in pediatric B-cell Acute Lymphoblastic Leukemia (B-ALL).
“CARGO’s unique approach to engineering CAR T-cell therapy has the potential to deliver best-in-class treatments for LBCL, for which there is no approved CD22-directed therapy,” said Reid Huber, PhD, partner at Third Rock Ventures. “Given CARGO’s progress to date and its experienced leadership team, CARGO is well-positioned to be first-to-market with an autologous CD22 CAR T-cell therapy that may extend life and give hope for a cure for patients with CD19 relapsed/refractory B-cell malignancies.”
In 2021, internationally recognized experts focused on immuno-oncology and CAR T-cell engineering, collaborated with Samsara BioCapital to operationalize CARGO Therapeutics (formerly Syncopation Life Sciences). Crystal Mackall, MD, and Robbie Majzner, MD, founded the Company with Nancy Goodman, JD. The founders’ overarching mission was to overcome CAR resistance to extend the clinical benefit of CARs for patients in need. Co-founder Crystal Mackall added, “CARGO was founded to make a meaningful difference to patients’ lives. The differentiated CD22 clinical profile studied in more than 100 patients has shown impressive activity with a very favorable safety profile, and in combination with CARGO’s proprietary platforms, positions us to build and deliver the next generation of CAR T therapies.”
CRG-022 is CARGO’s investigational cell therapy that is composed of autologous T-cells transduced with a lentiviral vector (m971-BBZ) expressing a CD22-targeting CAR. CD22 is a transmembrane protein expressed on normal B-cells and B-cell malignancies. CRG-022 is currently being evaluated in a Phase 1/1b clinical trial of autologous CD22 CAR T-cells in children and young adults with recurrent or refractory (R/R) B-cell malignancies. Initial results in adults demonstrate the safety and antitumor activity of CAR T-cell therapy targeting CD22 in patients with LBCL that is R/R to CD19 CAR cell therapy. CARGO’s CD22 CAR has a unique design which is associated with efficacy in the clinic that has not been seen with other CD22 CARs. CARGO believes that CRG-022 has the potential to treat LBCL in patients for whom prior CD19 CAR T-cell therapies have failed.
About Large B-Cell Lymphoma (LBCL)
Lymphoma that affects B lymphocytes are called B-cell lymphomas. B-cells make antibodies to fight infections and are an important part of the human immune system. B-cell lymphomas account for approximately 85% of non-Hodgkin lymphomas (NHL) in the United States. LBCL is an aggressive (fast-growing) lymphoma that occurs most commonly in people over the age of 60, though it can occur in childhood.
About CARGO Therapeutics (formerly Syncopation Life Sciences)
Our mission is to outsmart cancer and develop the next generation of best-in-class chimeric antigen receptor T-cell (CAR T) therapies. Our proprietary platform technologies enable us to engineer and develop next-generation best-in-class CARs that incorporate complex therapeutic cargo with the potential to achieve durable responses that are curative for more cancer patients. Our founders are pioneers and world experts in CAR T-cell therapy, and our team has experience and success developing, manufacturing, launching and commercializing oncology and cell therapy products. For more information, please visit the CARGO Therapeutics website at https://cargo-tx.com/
About Third Rock Ventures
Third Rock Ventures is a leading healthcare venture firm focused on advancing disruptive areas of science and medicine to deliver breakthroughs to patients. By starting with big ideas and fostering collaboration among people with expertise in science, medicine, business and strategy, we aim to discover, launch and build innovative companies that can transform the lives of patients. Our mission is to be the preferred partner for entrepreneurs, investors, employees and industry to build great companies that discover and develop products that make a difference for the patients we serve. For more information, please visit www.thirdrockventures.com.
About RTW Investments, LP
RTW Investments, LP is a New York-based, global, full life-cycle investment firm that focuses on identifying transformational and disruptive innovations across the biopharmaceutical and medical technologies sectors. As a leading partner of industry and academia, RTW combines deep scientific expertise with a solution-oriented investment approach to advance emerging medical therapies by building and supporting the companies and/or academics developing them. For further information about RTW, please visit www.RTWfunds.com.
About Perceptive Xontogeny Venture Funds
The Perceptive Xontogeny Venture Funds (“PXV Funds”) are Perceptive Advisors investment vehicles focused purely on early-stage, private venture investments in life sciences companies. Primary investments for the Funds include companies that are seeking a lead investor for Series A and B financings, which include both companies that are seeded and incubated at Xontogeny and companies that are seeded and incubated by other organizations, accelerators and seed investors. The PXV Funds are also open to participating in syndicated Series A and B financings as a co-lead or passive investor with other venture capital firms. For more information, visit www.perceptivelife.com.
About Samsara BioCapital
Samsara BioCapital is a new breed of biotech investment fund focused on translating cutting-edge biology into new therapies to treat patients with unmet medical needs. Founded in 2016 by Srinivas Akkaraju, M.D., Ph.D., our team of scientists, investors and entrepreneurs takes a long-term view on value creation across all stages of public and private life science companies. We believe in a collaborative, hands-on approach, working closely with entrepreneurs to harness exciting scientific advances and build leading companies. Samsara actively manages assets on behalf of endowments, foundations and family offices. For more information, please visit www.samsaracap.com.
About Crystal Mackall, MD
Dr. Mackall is the Ernest and Amelia Gallo Family Professor of Pediatrics and Internal Medicine at Stanford University. She serves as Founding Director of the Stanford Center for Cancer Cell Therapy, Associate Director of Stanford Cancer Institute, Leader of the Cancer Immunology and Immunotherapy Program, and Director of the Parker Institute for Cancer Immunotherapy at Stanford. During a 27-year tenure at NCI, culminating as Chief of the Pediatric Oncology Branch, and now at Stanford, she has led an internationally recognized translational research program focused on immuno-oncology. Dr. Mackall’s lab invented the CD22 CAR that is the focus of this license application (Haso, Blood 2014), and she played a leading role in the NCI CD22 CAR clinical trial (Fry, Nat Med 2018; Shah, JCO, 2020).
About Robbie Majzner, MD
Dr. Majzner is the Taube Distinguished Scholar for Pediatric Immunotherapy and an Assistant Professor of Pediatrics at Stanford University School of Medicine. His work has identified target antigen density as a major contributor to CAR T-cell efficacy, and he has generated novel CAR architectures capable of overcoming low target antigen density (Majzner et al., Cancer Discovery, 2020). He has also generated and optimized novel receptors to recognize antigens over-expressed on pediatric solid tumors such as GD2 (Mount/Majzner et al., Nature Medicine, 2018) B7-H3 (Majzner et al., Clinical Cancer Research, 2019), and ALK (Walker/Majzner et al., Molecular Therapy, 2017), several of which are now in first-in-child clinical trials.
About Nancy Goodman, JD
Ms. Goodman is the CEO of Kids v Cancer, author and lead advocate of the RACE for Children Act and the Creating Hope Act Rare Pediatric Priority Review Voucher Program. Among the honors she has received are: American Association of Cancer Research Distinguished Advocacy Award, Fast Company Magazine as top ten most innovative nonprofits (award to Kids v Cancer), US Food and Drug Administration Group Recognition Award, Member of U.S. Vice President Biden’s Blue Ribbon Pediatric Oncology Panel for the Cancer Moonshot, Member of National Cancer Institute Board of Scientific Counselors.