JCR Pharmaceuticals to Present at the American Society of Gene and Cell Therapy 28
The oral presentation details are listed below, and the full program can be found on the ASGCT congress website at https://annualmeeting.asgct.org/.
Title: Incorporation of transferrin receptor binder and surface mutations into AAV enables efficient brain delivery and reduced liver tropism
Session: AAV Gene Transfer (A): Crossing the Blood-Brain Barrier
Abstract Number: 92
Date/Time: Wednesday, May 14, 2:30-2:45pm CT
Presenter: Yuhei Ashida, Associate Senior Scientist, at JCR Pharmaceuticals
About the American Society of Gene and Cell Therapy (ASGCT)
The American Society of Gene and Cell Therapy (ASGCT) is the primary professional membership organization for gene and cell therapy. The Society's members are scientists, physicians, patient advocates, and other professionals. The mission of the ASGCT is to advance knowledge, awareness, and education, leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease. For more information, please visit www.asgct.org.
About the J-Brain Cargo ® Platform Technology
JCR Pharmaceuticals has developed a proprietary blood-brain barrier (BBB)-penetrating technology, J-Brain Cargo ®, to bring biotherapeutics into the central nervous system (CNS). The first drug developed based on this technology is IZCARGO ® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder.
About JCR Pharmaceuticals Co., Ltd.
JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 50-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients' lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler- Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical advancement at a global level. Our core values – reliability, confidence, and persistence – benefit all our stakeholders, including employees, partners, and patients. For more information, please visit https://www.jcrpharm.co.jp/en/site/en/.
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The Phase 3 AQUILA study (NCT03301220) is the largest randomised study of a well-defined high-risk SMM population, evaluating the efficacy and safety of fixed-duration, monotherapy daratumumab SC (n=194) compared with active monitoring (n=196).1 At a median follow-up of 65.2 months (range, 0-76.6), patients who received daratumumab SC showed statistically significant improved progression-free survival (PFS; defined as progression to active multiple myeloma, as assessed according to the International Myeloma Working Group diagnostic criteria for multiple myeloma [SLiM-CRAB], or death) compared to patients who underwent active monitoring; 63.1 percent in the daratumumab arm versus 40.8 percent in the active monitoring arm remained alive and progression-free at 60 months (hazard ratio [HR], 0.49; 95 percent confidence interval [CI], 0.36-0.67; p<0.001).1 Among patients who were retrospectively categorised as having high-risk SMM, per the current Mayo 2018 criteria (20/2/20), median PFS was not reached in the daratumumab arm and was 22.1 months in the active monitoring arm (HR, 0.36; 95 percent CI, 0.23-0.58).1 Overall survival was also extended with daratumumab SC, with 5-year survival rates of 93.0 percent vs 86.9 percent for active monitoring (HR, 0.52; 95 percent CI, 0.27-0.98).1 Additionally, patients who received daratumumab SC saw a higher overall response rate of 63.4 percent compared to 2.0 percent with active monitoring (p<0.001).1 Median time to first-line multiple myeloma treatment was not reached for patients receiving daratumumab SC compared to 50.2 months with active monitoring (HR, 0.46; 95 percent CI, 0.33-0.62; nominal p<0.0001).1,6 Daratumumab demonstrated a safety profile consistent with previous studies of daratumumab in other indications, with a low rate of treatment discontinuation due to treatment-emergent adverse events (TEAEs).1 Grade 3/4 TEAEs occurred in 40.4 percent of patients treated with daratumumab SC and 30.1 percent of patients actively monitored.1 The most common (≥5 percent in either group) Grade 3/4 TEAE was hypertension (5.7 percent vs 4.6 percent, respectively).1 The frequency of TEAEs leading to discontinuation of daratumumab SC was low (5.7 percent), as was the incidence of fatal TEAEs in both groups (1.0 percent vs 2.0 percent, respectively).1 'Until now, there have been no approved treatment options for patients diagnosed with high-risk smouldering multiple myeloma,' said Jordan Schecter, M.D., Vice President, Disease Area Leader, Multiple Myeloma, Johnson & Johnson Innovative Medicine. 'With today's approval, Johnson & Johnson has an innovative therapy for every stage of the disease. We can now offer physicians and patients the option to treat with daratumumab earlier, significantly delaying progression and the need for more intensive, continuous therapy, as well as extending overall survival. We remain steadfast in our mission to get in front of cancer.' About the AQUILA Study AQUILA (NCT03301220) is a randomised, multicentre Phase 3 study investigating daratumumab SC versus active monitoring in patients (n=390) with high-risk smouldering multiple myeloma (SMM).7 The primary endpoint is progression-free survival and secondary endpoints include time to progression, overall response rate and overall survival.7 Patients in the study were diagnosed with SMM in the last five years and were excluded if they had prior exposure to approved or investigational treatments for SMM or multiple myeloma.7 About Smouldering Multiple Myeloma SMM is an asymptomatic intermediate disease state of multiple myeloma where abnormal cells can be detected in the bone marrow.2,8 Patients living with SMM tend not to show signs or symptoms typically associated with active myeloma, such as bone pain, bone fractures, kidney problems, or anaemia, however as abnormal plasma cells are present, organ damage may begin and progress asymptomatically.1,9 Approximately 15 percent of all cases of newly diagnosed multiple myeloma are classified as SMM, and half of those diagnosed with high-risk SMM are estimated to progress to active multiple myeloma within two years.10 About Multiple Myeloma Multiple myeloma is currently an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.11,12 In multiple myeloma, these malignant plasma cells continue to proliferate, accumulating in the body and crowding out normal blood cells, as well as often causing bone destruction and other serious complications.11,12 In the European Union, it is estimated that more than 35,000 people were diagnosed with multiple myeloma in 2022, and more than 22,700 patients died.13 Patients living with multiple myeloma experience relapses which become more frequent with each line of therapy while remissions become progressively shorter.14,15,16 Whilst some patients with multiple myeloma initially have no symptoms, others can have common signs and symptoms of the disease, which can include bone fracture or pain, low red blood cell counts, fatigue, high calcium levels, infections, or kidney damage.17 About Daratumumab and Daratumumab SC Johnson & Johnson is committed to exploring the potential of daratumumab for patients with multiple myeloma across the spectrum of the disease. In August 2012, Janssen Biotech, Inc., a Johnson & Johnson company, and Genmab A/S entered a worldwide agreement, which granted Johnson & Johnson an exclusive licence to develop, manufacture and commercialise daratumumab. Since launch, daratumumab has become a foundational therapy in the treatment of multiple myeloma, having been used in the treatment of more than 618,000 patients worldwide.18 Daratumumab is the only CD38-directed antibody approved to be given subcutaneously to treat patients with multiple myeloma.19 Daratumumab SC is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE® drug delivery technology.19 CD38 is a surface protein that is present in high numbers on multiple myeloma cells, regardless of the stage of disease.19 Daratumumab binds to CD38 and inhibits tumour cell growth causing myeloma cell death.19 Daratumumab may also have an effect on normal cells.19 Data across ten Phase 3 clinical trials, in both the frontline and relapsed settings, have shown that daratumumab-based regimens resulted in significant improvement in progression-free survival and/or overall survival.20,21,22,23,24,25,26,27,28 For further information on daratumumab, please see the Summary of Product Characteristics at: About Johnson & Johnson At Johnson & Johnson, we believe health is everything. 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These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialise, actual results could vary materially from the expectations and projections of Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; competition, including technological advances, new products and patents attained by competitors; challenges to patents; changes in behaviour and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's most recent Annual Report on Form 10-K, including in the sections captioned 'Cautionary Note Regarding Forward-Looking Statements' and 'Item 1A. Risk Factors,' and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at or on request from Johnson & Johnson. Johnson & Johnson does not undertake to update any forward-looking statement as a result of new information or future events or developments. *Professor Meletios A. Dimopoulos, M.D., National and Kapodistrian University of Athens School of Medicine, has provided consulting, advisory, and speaking services to Johnson & Johnson; he has not been paid for any media work. 1 Dimopoulos MA, et al. Phase 3 Randomized Study of Daratumumab Monotherapy versus Active Monitoring in Patients with High-risk Smoldering Multiple Myeloma: Primary Results of the AQUILA study. Oral presentation. American Society of Hematology (ASH) Annual Meeting; December 7-10, 2024.2 Myeloma UK. Smouldering Myeloma. Available at: Last accessed: July 2025. 3 European Medicines Agency. DARZALEX (daratumumab) Summary of Product Characteristics. July 2025.4 Oben B, et al. Whole-Genome Sequencing Reveals Progressive Versus Stable Myeloma Precursor Conditions as Two Distinct Entities. Nature Communications. 2021; 12(1861).5 Maura F, et al. Targeting the Tumor and The Immune System in Smoldering Multiple Myeloma. The New England Journal of Medicine. 2025;392:1858-1860.6 Dimopoulos MA, et al. Phase 3 Randomized Study of Daratumumab Monotherapy Versus Active Monitoring in Patients With High-risk Smoldering Multiple Myeloma: Primary Results of the AQUILA Study. Abstract #773. American Society of Hematology (ASH) Annual Meeting; December 7-10, 2024. 7 A Study of Subcutaneous Daratumumab Versus Active Monitoring in Participants With High-Risk Smoldering Multiple Myeloma. Available at: . Last accessed: July 2025.8 WebMD. Smoldering Multiple Myeloma. Available at: Last accessed: July 20259 American Cancer Society. About Multiple Myeloma. Available at: Last accessed: July 2025.10 Rajkumar SV, et al. Smoldering Multiple Myeloma Current Treatment Algorithms. Blood Cancer J. 2022;12(9):129.11 Abdi J, et al. Drug Resistance in Multiple Myeloma: Latest Findings on Molecular Mechanisms. Oncotarget. 2013;4(12):2186-2207.12 American Society of Clinical Oncology. Multiple Myeloma: Introduction. Available at: Last accessed: July 2025.13 ECIS - European Cancer Information System. Estimates of Cancer Incidence and Mortality in 2022, by Country. Multiple Myeloma. Available at: Last accessed: July 2025.14 Bhatt P, et al. Relapsed/Refractory Multiple Myeloma: A Review of Available Therapies and Clinical Scenarios Encountered in Myeloma Relapse. Curr Oncol. 2023;30(2):2322-2347.15 Hernández-Rivas JÁ, et al. The Changing Landscape of Relapsed and/or Refractory Multiple Myeloma (MM): Fundamentals and Controversies. Biomark Res. 2022;10(1):1-23.16 Gavriatopoulou M, et al. Metabolic Disorders in Multiple Myeloma. Int J Mol Sci. 2021;22(21):11430.17 American Cancer Society. Multiple Myeloma: Early Detection, Diagnosis and Staging. Available at: Last accessed: July 2025.18 J&J Data on File (RF-452129). Number of Patients Treated with DARZALEX Worldwide as of December 2024.19 Janssen EMEA. European Commission Grants Marketing Authorisation for DARZALEX® (Daratumumab) Subcutaneous Formulation for All Currently Approved Daratumumab Intravenous Formulation Indications. Available at: Last accessed: July 2025.20 Moreau P, et al. Bortezomib, Thalidomide, and Dexamethasone With or Without Daratumumab Before and After Autologous Stem-Cell Transplantation for Newly Diagnosed Multiple Myeloma (CASSIOPEIA): A Randomised, Open-label, Phase 3 Study. Lancet. 2019;394(10192):29-38.21 Facon T, et al. MAIA Trial Investigators. Daratumumab Plus Lenalidomide and Dexamethasone for Untreated Myeloma. New England Journal of Medicine. 2019;380(22):2104-2115.22 Mateos MV, et al. Overall Survival with Daratumumab, Bortezomib, Melphalan, and Prednisone in Newly Diagnosed Multiple Myeloma (ALCYONE): A Randomised, Open-label, Phase 3 Trial. The Lancet. 2020;395:132-141.23 Dimopoulos MA, et al. APOLLO Trial Investigators. Daratumumab Plus Pomalidomide and Dexamethasone Versus Pomalidomide and Dexamethasone Alone in Previously Treated Multiple Myeloma (APOLLO): An Open-label, Randomised, Phase 3 Trial. Lancet Oncol. 2021;22(6):801-812.24 Palladini G, et al. Daratumumab Plus CyBorD for Patients with Newly Diagnosed AL Amyloidosis: Safety Run-in Results of ANDROMEDA. Blood 2020;2;136(1):71-80.25 Chari A, et al. Daratumumab Plus Pomalidomide and Dexamethasone in Relapsed and/or Refractory Multiple Myeloma. Blood. 2017;130(8):974-981.26 Bahlis NJ, et al. Daratumumab Plus Lenalidomide and Dexamethasone in Relapsed/Refractory Multiple Myeloma: Extended Follow-up of POLLUX, A Randomized, Open-label, Phase 3 study. Leukemia. 2020;34(7):1875-1884.27 Mateos MV, et al. Daratumumab, Bortezomib, and Dexamethasone Versus Bortezomib and Dexamethasone in Patients with Previously Treated Multiple Myeloma: Three-Year Follow-up of CASTOR. Clin Lymphoma Myeloma Leuk. 2020;20(8):509-518.28 Usmani S Z, et al. Daratumumab + Bortezomib/Lenalidomide/Dexamethasone in Patients with Transplant-Ineligible or Transplant-Deferred Newly Diagnosed Multiple Myeloma: Results of the Phase 3 CEPHEUS Study. Oral Presentation. 21st International Myeloma Society (IMS) Annual Meeting. September 25 – 28, 2024. CP-529642 July 2025 CONTACT: Media contact: Jenni Mildon jmildon@ +44 7920 418 552 Investor contact: Lauren Johnson investor-relations@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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