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      4. DARZALEX® (daratumumab) SC-based regimens improve MRD negativity and progression-free survival in newly diagnosed multiple myeloma, and overall survival in AL amyloidosis
      Oncology

      DARZALEX® (daratumumab) SC-based regimens improve MRD negativity and progression-free survival in newly diagnosed multiple myeloma, and overall survival in AL amyloidosis

      New analysis from Phase 3 CEPHEUS study demonstrates 85 percent of patients who achieved MRD negativity (10-6) with daratumumab SC were progression free at 4.5 years1

      AURIGA study shows higher rates of MRD negative conversion in subgroup patient populations disproportionately impacted by multiple myeloma2

      Final analysis of Phase 3 ANDROMEDA study shows the addition of daratumumab to VCd significantly improves overall survival for patients with newly diagnosed AL amyloidosis3

      BEERSE, BELGIUM (9 December 2024) – Janssen-Cilag International NV, a Johnson & Johnson company, announced data highlighting that DARZALEX® (daratumumab) subcutaneous (SC)-based regimens improve overall and sustained minimal residual disease (MRD) negativity rates and progression-free survival (PFS) in patients with newly diagnosed multiple myeloma (NDMM), regardless of transplant status and in patients with poor prognostic disease features.1,2,3 These findings were demonstrated in an expanded MRD analysis of the Phase 3 CEPHEUS study (Abstract #362) and a post hoc analysis of clinically relevant subgroups in the Phase 3 AURIGA study (Abstract #675), which were both featured as oral presentations at the 2024 American Society of Hematology (ASH) Annual Meeting, taking place in San Diego, California, United States from 7-10 December.

      Data from the expanded MRD analysis of the Phase 3 CEPHEUS study show the addition of daratumumab SC to bortezomib, lenalidomide and dexamethasone (D-VRd) leads to improved and deepened rates of overall and sustained MRD negativity (both 10-5 and 10-6 sensitivity thresholds in patients who achieved a complete response or better) vs VRd alone, and shows significantly improved PFS.1 CEPHEUS is the fifth Phase 3 study showing that the addition of daratumumab improves depth and duration of response, leading to improved PFS in NDMM.2,4,5,6

      At a median follow-up of 58.7 months, overall MRD negativity rates were significantly higher with D- VRd vs VRd at both 10-5 (60.9 percent vs 39.4 percent; odds ratio [OR], 2.37; 95 percent confidence interval [CI], 1.58-3.55; p<0.0001) and 10-6 (46.2 percent vs 27.3 percent; OR, 2.24; 95 percent CI, 1.48-3.40; p=0.0001) sensitivity thresholds.1 Treatment with D-VRd showed continued benefit of sustained MRD negativity for two years (10-5: 42.1 percent vs 22.7 percent; 10-6: 27.9 percent vs 13.6 percent).1 Additionally, the deep and sustained MRD negativity rates with D-VRd led to improved PFS – the estimated 54-month PFS rates were 86.2 percent for D-VRd vs 79.0 percent for VRd in MRD negative (10-6) patients, and 51.0 percent vs 36.5 percent for MRD positive patients.1

      “This analysis from the CEPHEUS study comparing daratumumab-VRd versus VRd, showed higher rates of both overall and sustained MRD negativity alongside promising trends in progression-free survival,” said Sonja Zweegman, M.D., PhD, head of the Department of Hematology, Amsterdam University Medical Centre.* “This regimen has the potential to improve outcomes for patients with newly diagnosed multiple myeloma who are ineligible for transplant or for whom transplant is not planned as initial therapy.”

      As presented previously, the overall safety profile of D-VRd was consistent with the known safety profiles for daratumumab and VRd.7 The most common (>10 percent) Grade 3/4 haematologic and non-haematologic adverse events with D-VRd vs VRd were neutropenia (44.2 percent vs 29.7 percent), thrombocytopenia (28.4 percent vs 20.0 percent), anaemia (13.2 percent vs 11.8 percent), peripheral neuropathies (8.1 percent vs 8.2 percent), diarrhoea (12.2 percent vs 9.2 percent), and COVID-19 (11.2 percent vs 4.6 percent).7

      Addition of daratumumab SC to maintenance regimen resulted in higher MRD negativity rates across clinically relevant subgroups by age, race, disease stage and cytogenetic risk

      In a post hoc analysis of the Phase 3 AURIGA study, a maintenance regimen of daratumumab SC combined with lenalidomide (R) resulted in consistently improved MRD negative conversion rates after 12 months. These results were consistent across anti-CD38 naïve patient subgroups who were MRD positive post-autologous stem cell transplant.2 In patients older than 65 years, MRD negative rates were higher when treated with D-R maintenance therapy compared to R alone (52.6 percent vs 17.5 percent; OR, 5.24; 95 percent CI, 1.86-14.74).2 Maintenance therapy with D-R showed a consistently higher conversion to MRD negativity in Black patients (n=20) compared to R alone (60.0 percent vs 16.7 percent; OR, 7.50; 95 percent CI, 1.85-30.34) and White patients (n=67) (46.3 percent vs 20.6 percent; OR, 3.32; 95 percent CI, 1.55-7.10).2

      “Patients over 65, Black individuals, and those with advanced or high-risk disease are disproportionately impacted by multiple myeloma and historically have had fewer treatment options that yield deep and durable results,” said Imran Khan, M.D., PhD, Vice President, Medical Affairs, Hematology, Johnson & Johnson Innovative Medicine. “Evaluating MRD negativity in these patients underlies its importance as a recognised predictor of long-term progression-free survival. The data being presented at ASH this year emphasise the potential of daratumumab SC in helping newly diagnosed patients achieve MRD negativity.”

      Data also show that the maintenance regimen of D-R resulted in higher MRD negative conversion rates for patients with advanced-stage disease (Stage III) as defined by the International Staging System (65.2 percent vs 13.0 percent; OR, 12.50; 95 percent CI, 2.83-55.25) and patients with high cytogenetic risk per the standard definition (31.8 percent vs 6.7 percent; OR, 6.53; 95 percent CI, 0.71-60.05) or the revised definition (43.8 percent vs 13.3 percent; OR, 5.06; 95 percent CI, 1.43- 17.88).2

      Safety results were consistent with the known safety profile of daratumumab SC. The most common Grade 3/4 treatment-emergent adverse events (TEAEs) in patients 65 years or older included neutropenia, leukopenia and hypokalaemia.2 There were no unexpected safety concerns in this age group or in Black patients.2

      “Data from CEPHEUS and AURIGA reinforce the transformational role of daratumumab-based regimens as foundational first-line therapies across the patient spectrum, including in maintenance therapy for transplant-eligible newly diagnosed multiple myeloma,” said Edmond Chan, MBChB, M.D. (Res), EMEA Therapeutic Area Lead Haematology, Johnson & Johnson Innovative Medicine. “With ANDROMEDA, we add to the growing body of evidence from Phase 3 studies demonstrating the overall survival benefit of daratumumab-based regimens for people with difficult-to-treat haematologic disorders.”

      Final analysis of Phase 3 ANDROMEDA study reinforces daratumumab SC-based regimen showing significant overall survival in patients with newly diagnosed light chain (AL) amyloidosis

      The final analysis of the Phase 3 ANDROMEDA study was also presented (Abstract #891), showing that the addition of daratumumab SC to bortezomib, cyclophosphamide, and dexamethasone (D-VCd) demonstrated deeper and more rapid haematologic responses, resulting in a statistically significant improvement in both OS and major organ deterioration PFS (MOD-PFS) (i.e. end-stage renal or cardiac disease, haematologic progression, or death) for patients with newly diagnosed AL amyloidosis, a rare plasma cell disorder associated with the deterioration of vital organs.3 After a median follow-up of 61.4 months, patients treated with D-VCd showed a 56 percent reduction in the risk of end-stage renal or cardiac disease, haematologic progression or death (hazard ratio [HR]=0.44, p<0.0001).3 The median MOD-PFS was not reached for D-VCd, while it was 30.2 months for VCd.3 Additionally, D-VCd also provided significant survival benefits with an HR of 0.62 (p=0.0121), indicating a 38.0 percent reduction in the risk of death compared to VCd.3 The five-year survival rate was 76.1 percent for D-VCd vs 64.7 percent for VCd.3 Safety results were consistent with the known safety profiles for daratumumab and VCd, with the most common Grade 3/4 TEAEs (≥5 percent) being lymphopenia (13.0 percent vs 10.0 percent), pneumonia (8.0 percent vs 4.0 percent), diarrhoea (6.0 percent vs 4.0 percent), cardiac failure (congestive; 6.0 percent vs 3.0 percent), neutropenia (5.0 percent vs 3.0 percent) and syncope (6.0 percent vs 6.0 percent), fatigue (5.0 percent vs 3.0 percent), hypokalaemia (2.0 percent vs 5.0 percent), and peripheral oedema (3.0 percent vs 6.0 percent), respectively.3

      About the CEPHEUS Study
      CEPHEUS (NCT03652064) is an international, randomised, open-label, Phase 3 study comparing subcutaneous daratumumab, bortezomib, lenalidomide, and dexamethasone (D-VRd) with standard bortezomib, lenalidomide, and dexamethasone (VRd).7,8 The trial enrolled 395 patients with newly diagnosed multiple myeloma who were either ineligible for stem cell transplantation (SCT) or for whom SCT is not planned.7 The primary endpoint was overall MRD negativity rate.7 The minimum age for participation was 18 years for patients in both the D-VRd and VRd arms, with a median patient age of 70 (range 31-80).7 The study was conducted in 13 countries across North America, South America, and Europe.

      About the AURIGA Study
      AURIGA (NCT03901963) is a randomised study which includes 200 patients aged 18-79 years with newly diagnosed multiple myeloma who are MRD positive after frontline autologous stem cell transplant.9 Patients received 1800mg daratumumab by subcutaneous injection in combination with lenalidomide (orally) as maintenance therapy for a maximum of 36 cycles.2,6 Each cycle was 28 days.2 Patients in the comparative arm received lenalidomide (orally) alone as maintenance therapy for a maximum of 36 cycles.2

      About the ANDROMEDA Study
      ANDROMEDA (NCT03201965) is an ongoing Phase 3, randomised, open-label study investigating the safety and efficacy of daratumumab SC in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd), compared to VCd alone, for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis.10 The study includes 388 patients with newly diagnosed AL amyloidosis with measurable haematologic disease and one or more organs affected.3 The primary endpoint is overall complete haematologic response rate by intent-to-treat.3 Patients received daratumumab SC, 800 mg/30,000 units administered subcutaneously once weekly from weeks one to eight, once every two weeks from weeks nine to 24 and once every four weeks starting with week 25 until disease progression or unacceptable toxicity or a maximum of two years.3 Among patients who received D-VCd, 74 percent were exposed for 6 months or longer and 32 percent were exposed for greater than one year.11

      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 585,000 patients worldwide.12 Daratumumab is the only CD38-directed antibody approved to be given subcutaneously to treat patients with multiple myeloma.13 Daratumumab SC is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology.14

      CD38 is a surface protein that is present in high numbers on multiple myeloma cells, regardless of the stage of disease.14 Daratumumab binds to CD38 and inhibits tumour cell growth causing myeloma cell death.14 Daratumumab may also have an effect on normal cells.14 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.1,13,14,15,16,17,18,19,20,21

      For further information on daratumumab, please see the Summary of Product Characteristics at: https://www.ema.europa.eu/en/documents/product-information/darzalex-epar-product-information_en.pdf. 22

      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.23,24 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.25 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.26 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.22

      About AL Amyloidosis
      Light chain (AL) amyloidosis is a rare and potentially fatal haematologic disorder that can affect the function of multiple organs.27 The disease occurs when the bone marrow produces abnormal proteins called light chains, which clump together to form a substance called amyloid.27 These clumps of amyloid are deposited in tissues and vital organs and interfere with normal organ function, eventually causing organ deterioration.27,28 AL amyloidosis is the most common type of systemic amyloidosis.29 It frequently affects the heart, kidneys, digestive tract, liver and nervous system.30 Diagnosis is often delayed and prognosis is poor due to advanced, multi-organ, particularly cardiac, involvement.31 Approximately 30,000 to 45,000 patients in the European Union and the United States have AL amyloidosis.30

      About Johnson & Johnson
      At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity.

      Learn more at www.innovativemedicine.jnj.com/emea. Follow us at www.linkedin.com/company/jnj-innovative-medicine-emea. Janssen Pharmaceutica NV, Janssen-Cilag Limited, Janssen Biotech, Inc., and Janssen Research & Development, LLC are Johnson & Johnson companies.

      Cautions Concerning Forward-Looking Statements
      This press release contains “forward-looking statements” as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of daratumumab. The reader is cautioned not to rely on these forward-looking statements. 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 Janssen Pharmaceutica NV, Janssen-Cilag Limited, Janssen Biotech, Inc., Janssen Research & Development, LLC and/or 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 Annual Report on Form 10-K for the fiscal year ended December 31, 2023, 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 http://www.sec.gov/, http://www.jnj.com/ or on request from Johnson & Johnson. None of Janssen Pharmaceutica NV, Janssen-Cilag Limited, Janssen Biotech, Inc., Janssen Research & Development, LLC nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.

      *Sonja Zweegman, MD, PhD, head of the Department of Hematology, Amsterdam University Medical Centre, has provided consulting, advisory, and speaking services to Janssen; she has not been paid for any media work.

      Media contact:
      Jenni Mildon
      [email protected]
      +44 7920 418 552

      Investor contact:
      Lauren Johnson
      [email protected]

      CP-492203
      December 2024

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      2 Foster L et al., Daratumumab Plus Lenalidomide (D-R) Versus Lenalidomide (R) Alone as Maintenance Therapy in Newly Diagnosed Multiple Myeloma (NDMM) After Transplant: Analysis of the Phase 3 AURIGA Study Among Clinically Relevant Subgroups. Oral presentation. American Society of Hematology (ASH) Annual Meeting; December 7-10, 2024
      3 Kastritis E et al., Subcutaneous Daratumumab (DARA) + Bortezomib, Cyclophosphamide, and Dexamethasone (VCd) in Patients With Newly Diagnosed Light Chain (AL) Amyloidosis: Final Analysis of the Phase 3 ANDROMEDA Study. Abstract #891. American Society of Hematology (ASH) Annual Meeting; December 7-10, 2024.
      4 Rodríguez-Otero P, et al. Daratumumab (DARA) + bortezomib/lenalidomide/dexamethasone (VRd) in transplant-eligible (TE) patients (pts) with newly diagnosed multiple myeloma (NDMM): Analysis of minimal residual disease (MRD) in the PERSEUS trial. 2024 American Society for Clinical Oncology Annual Meeting. June 3, 2024.
      5 Corre, J., et al. Daratumumab (DARA) + Bortezomib/Thalidomide/ Dexamethasone (D-VTd) and DARA Maintenance in Transplanteligible Newly Diagnosed Multiple Myeloma (NDMM): CASSIOPEIA Minimal Residual Disease (MRD) Update. IMS 2024. September 27, 2024.
      6 San-Miguel J., et al., Sustained minimal residual disease negativity in newly diagnosed multiple myeloma and the impact of daratumumab in MAIA and ALCYONE, Blood 2022
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      8 Clinicaltrials.gov. A Study Comparing Daratumumab, VELCADE (Bortezomib), Lenalidomide, and Dexamethasone (D-VRd) With VELCADE, Lenalidomide, and Dexamethasone (VRd) in Participants With Untreated Multiple Myeloma and for Whom Hematopoietic Stem Cell Transplant is Not Planned as Initial Therapy. Available at: https://clinicaltrials.gov/study/NCT03652064. Last accessed: December 2024.
      9 Clinicaltrials.gov. A Study of Daratumumab Plus Lenalidomide Versus Lenalidomide Alone as Maintenance Treatment in Participants With Newly Diagnosed Multiple Myeloma Who Are Minimal Residual Disease Positive After Frontline Autologous Stem Cell Transplant (AURIGA). Available at: https://clinicaltrials.gov/study/NCT03901963. Last accessed: December 2024.
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      12 Johnson & Johnson [data on file]. RF-430506. Number of patients treated with DARZALEX® worldwide as of 30 June 2024.
      13 Janssen EMEA. European Commission Grants Marketing Authorisation for DARZALEX® (Daratumumab) Subcutaneous Formulation for All Currently Approved Daratumumab Intravenous Formulation Indications. Available at: www.businesswire.com/news/home/20200604005487/en/European-Commission-GrantsMarketingAuthorisation-forDARZALEX%C2%AE%E2%96%BC-daratumumab-SubcutaneousFormulation-for-all-CurrentlyApproved-DaratumumabIntravenous-Formulation-Indications. Last accessed: December 2024.
      14 Facon T, et al. MAIA Trial Investigators. Daratumumab plus Lenalidomide and Dexamethasone for Untreated Myeloma. N Engl J Med 2019;380(22):2104-2115
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      16 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:P132-141.
      17 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.
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      19 Chari A, et al. Daratumumab plus pomalidomide and dexamethasone in relapsed and/or refractory multiple myeloma. Blood 2017;130(8):974-981.
      20 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.
      21 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.
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