Alloy Therapeutics Signs Collaboration and License Agreement with Takeda to Develop Cell Therapy Platform

On November 20, 2024 Alloy Therapeutics Inc. ( "Alloy"), a biotechnology ecosystem company dedicated to democratizing access to cutting edge drug discovery technologies reported a strategic collaboration and license agreement with Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK "Takeda") to develop Takeda’s proprietary induced pluripotent stem cell (iPSC) derived CAR-T cell platform (iCAR-T) and iPSC-derived CAR-NK platform (iCAR-NK) (Press release, Alloy Therapeutics, NOV 20, 2024, View Source [SID1234648512]). Alloy will focus on accelerating the development of key therapies to overcome solid and hematological malignancies.

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iCAR-T technology was developed as part of the T-CiRA joint research program between Takeda and the Center for iPS Research and Application (CiRA) at Kyoto University. The core technology to differentiate iPSC into immune cells originates from CiRA’s Shin Kaneko’s laboratory. iCAR-T has potential to develop ‘off-the-shelf’ cell therapies, offering the potential for best-in-class performance with enhanced potency, and significantly lower manufacturing costs compared to autologous cell therapy.

"iCAR-T is one of our flagship projects graduating from T-CiRA, our decade-long joint research program between Takeda and CiRA. This agreement between Takeda and Alloy aims to advance iCAR-T from discovery to clinical development," said Yasushi Kajii, Head of R&D Japan Region at Takeda. "We are impressed with Alloy’s corporate culture of valuing platform technologies, flexibility in collaboration, patient-centric mindset, and company creation abilities, all of which were key elements to our decision to work with Alloy. We look forward to seeing iCAR-T and iCAR-NK blossom as the technology advances to its next phase."

Under the agreement, Alloy gains co-exclusive rights to commercialize iCAR-T and iCAR-NK products for oncology indications. Alloy will leverage synergies across its unique business model to further advance the iCAR-T/NK platform while enabling broader access to the technology for biotech and pharma partners to develop therapies for cancers including solid tumors. Future clinical validation and platform enhancements will further strengthen the platform.

"We are honored and excited to be chosen by Takeda to collaborate in fulfilling the promise of iPSC," said Errik Anderson, Founder, Chairman & CEO of Alloy. "This is another instance of how Alloy’s flexible approach allows us to enable the global scientific community with cutting edge technologies designed for rapid, successful drug development. We believe a robust engagement with the ecosystem will play a critical role in unlocking the vast potential of iPSC and enabling the next generation of cancer therapeutics."

To support these efforts, Alloy is establishing a Japanese subsidiary at Shonan Health Innovation Park in Kanagawa Prefecture, to be led by Victor Stone (Yoshihide Ishii) as the Head of Alloy Cell Therapies and Alloy Therapeutics Japan.

Recursion and Exscientia, two leaders in the AI drug discovery space, have officially combined to advance the industrialization of drug discovery

On November 20, 2024 The business combination of two AI-powered drug discovery and development companies, Recursion (Nasdaq: RXRX) and Exscientia has been completed, with Exscientia becoming a wholly owned subsidiary of Recursion creating a vertically-integrated and technology-enabled drug discovery platform. Exscientia ADSs (Nasdaq: EXAI) ceased trading and will be delisted from Nasdaq (Press release, Recursion Pharmaceuticals, NOV 20, 2024, View Source [SID1234648509]).

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"I believe the combination of the incredible teams and platforms at Exscientia and Recursion position us as the leader of the AI-enabled drug discovery and development space," said Chris Gibson, Ph.D., Co-Founder and CEO of Recursion. "With more than 10 clinical and preclinical programs in the internal pipeline, more than 10 partnered programs and over $450M in upfront and realized milestone payments received from partners to date out of more than $20B possible, we are advancing a flywheel of discovery and creating value in our pipeline through technology."

"The combination of our platforms and people make us the company to beat," said David Hallett, Ph.D., former CSO and Interim CEO of Exscientia and newly appointed Chief Scientific Officer at Recursion. "With our combined strength of real-world proprietary data and the models we’ve created – hypothesizing, testing and learning in a continuous loop – we’re redefining the space by shrinking timelines and costs, identifying and optimizing lead candidates faster than traditional methods."

The Company is pleased to share updates on the combined entity’s pipeline, partnerships, and platform below:

Pipeline

The combined pipeline represents more than 10 clinical and preclinical programs. In addition there are approximately 10 advanced discovery programs in the current pipeline.

Updated guidance is bulleted below as well as a snapshot of our pipeline:

REC-617 (CDK7 inhibitor; Advanced Solid Tumors): Initial Phase 1 monotherapy safety and PK/PD data expected at the AACR (Free AACR Whitepaper) Special Conference on December 9th 2024, and a webinar to follow on December 10th 2024.
REV102 (ENPP1 inhibitor; Hypophosphatasia): Development candidate nomination expected in Q4 2024
REC-4881 (MEK1/2 inhibitor, Familial Adenomatous Polyposis): Phase 1b/2 safety and early efficacy data expected in H1 2025
REC-2282 (pan-HDAC inhibitor; Neurofibromatosis Type 2): PFS6 futility analysis expected by H1 2025
REC-3565 (MALT1 inhibitor, B-Cell Malignancies): Phase 1 first patient dosed (FPD) expected in Q1 2025
REC-4539 (LSD1 inhibitor, Small-Cell Lung Cancer): Phase 1 first patient dosed (FPD) expected in H1 2025
REC-994 (Superoxide scavenger, Cerebral Cavernous Malformation): Further data to be shared at an upcoming medical conference / publication / webinar in H1 2025; regulatory update expected by H2 2025
REC-394 (C. difficile Toxin B selective inhibitor, C. difficile): Phase 2 update expected in Q1 2026
REC-1245 (RBM39 degrader; Solid Tumors and Lymphoma): Phase 1 dose-escalation data update expected in H1 2026
REC-4209 (undisclosed target; Idiopathic Pulmonary Fibrosis): IND-enabling studies are ongoing
REC-4881 in APC/AXIN1 indications have been deprioritized as part of a disciplined strategic prioritization of the portfolio. Study status will be updated on clinicaltrials.gov
Recursion’s Pipeline of Programs

Partnerships

The combined company’s therapeutic partnerships represent more than 10 partnered programs in areas such as oncology and immunology. The combined company has received approximately $450M in upfront and milestone payments from partnerships to date. Through these partnerships, we have the potential to receive more than approximately $20B in additional milestone payments before royalties.

Recursion’s Partnership

Platform

With chemical design and synthesis methods from Exscientia and over 60 petabytes of proprietary data generated in house or licensed from partners like Helix and Tempus, the combined entity will strengthen the Recursion OS to be a first-in-class and best-in-class drug discovery and development platform.

Recursion Model2

The platform will continue to drive iterative loops of hypotheses and active learning all the way from research to development, with the goal of eventually creating virtual cells that will allow the company to execute clinical trials at scale.

Company, Board, and Leadership Updates

The combined company will have approximately 800 employees with the headquarters remaining in Salt Lake City, and primary offices in Toronto, Montreal, Milpitas, New York, the Oxford area, and London.

Individual board and executive leadership changes of Recursion, effective as of November 20, 2024, are summarized below:

Franziska Michor, a former member of the Board of Directors of Exscientia, was appointed as a Class II Director of the Board of Directors of Recursion, with her initial term to extend until the 2026 Annual Meeting of Stockholders of Recursion.
Ben Taylor, former Chief Financial and Strategy Officer of Exscientia, was appointed as the Chief Financial Officer of the Company and President of Recursion UK.
Dave Hallett, former Interim Chief Executive Officer of Exscientia, was appointed as Chief Scientific Officer of the Company.
Kristen Rushton, Chief Business Operations Officer of the Company, was promoted to Chief Operating Officer of the Company.
Matthew Kinn, Senior Vice President, Business Development and Corporate Initiatives of the Company was promoted to serve as Chief Business Officer of the Company.
Lina Nilsson, Senior Vice President, Emerging Technologies of the Company, was promoted to serve on the executive team as Senior Vice President, Head of Platform of the Company.
Michael Secora, Tina Marriott, and Laura Schaevitz will transition from their executive roles into advisor roles for the combined company. All three have provided many years of dedicated service to the Company and we wish to express our heartfelt gratitude for each of them. Recursion would not be where it is today without their dedication and efforts.
Update Call Information

Recursion will host an update call today at 7:30 a.m. ET / 5:30 a.m. MT / 12:30 p.m. GMT. The Company will broadcast the live stream from Recursion’s X (formerly Twitter), LinkedIn and YouTube accounts, and on Exscientia’s LinkedIn account. Questions can be submitted via this link ahead of time or during the livestream.

Lantern Pharma Announces First Patient Dosed in Japan for The Expansion Cohort in The Phase 2 HARMONIC™ Clinical Trial of LP-300 in Never-Smoker NSCLC Patients

On November 19, 2024 Lantern Pharma Inc. (NASDAQ: LTRN), an artificial intelligence (AI) company developing targeted cancer therapies using its proprietary RADR AI platform, reported that the first patient has been dosed – as part of the expansion cohort – in Japan for its Phase 2 HARMONIC clinical trial evaluating LP-300 in never-smoker patients with non-small cell lung cancer (NSCLC) who have progressed after receiving treatment with tyrosine kinase inhibitors (TKIs) (Press release, Lantern Pharma, NOV 19, 2024, View Source [SID1234648508]).

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The dosing of the first patient in Japan marks a significant expansion of the HARMONIC trial into Asia, where there is a notably higher prevalence of never-smoker NSCLC patients compared to Western populations. The trial is being conducted at five sites across Japan, including the National Cancer Center Japan under the leadership of Dr. Yasushi Goto, a renowned physician-researcher focused on lung cancer.

"The initiation of patient dosing in Japan is a significant milestone for the HARMONIC trial and our mission to develop LP-300 as a potential new treatment option for never-smoker NSCLC patients," said Panna Sharma, President and CEO of Lantern Pharma. "Japan and other Asian countries have a significantly higher proportion of never-smokers among NSCLC patients, with estimates suggesting that 33-40% of new lung cancer cases in Japan occur in never-smokers. This expansion allows us to accelerate enrollment while addressing a critical unmet need in a region where this disease has a particularly high impact."

Dr. Reggie Ewesuedo, VP of Clinical Development at Lantern Pharma commented, "This marks the achievement of a significant milestone for the Harmonic Trial. It is the beginning of an anticipated acceleration in enrollment following activation of sites in Japan as well as active screening of patients across sites in Taiwan. We are encouraged by the high level of interest from our clinical partners in Asia in this unique trial aimed at improving survival among never-smokers with lung cancer after relapsing from tyrosine kinase inhibitor-based treatment regimens."

The expansion into Japan and Taiwan follows encouraging preliminary results from the U.S. trial’s initial safety lead-in cohort, which demonstrated an 86% clinical benefit rate and 43% objective response rate among the first seven patients. The HARMONIC trial is evaluating LP-300 in combination with carboplatin and pemetrexed in never-smoker NSCLC patients who have recurrent NSCLC and have stopped responding to tyrosine kinase inhibitor (TKI) based therapies.

ABOUT THE HARMONIC TRIAL

The HARMONIC trial is a multicenter, open-label, randomized Phase 2 trial designed to evaluate the efficacy and safety of LP-300 in combination with standard-of-care chemotherapy (pemetrexed/carboplatin) versus chemotherapy alone. The trial is expected to enroll approximately 90 patients across sites in the United States and Asia. The primary endpoints are progression-free survival (PFS) and overall survival (OS).

Illumina announces expansion of TruSight Oncology portfolio

On November 19, 2024 Illumina, Inc. (NASDAQ: ILMN), a global leader in DNA sequencing and array-based technologies, reported that it will release TruSight Oncology 500 v2 (TSO 500 v2), a new version of its flagship cancer research assay to enable comprehensive genomic profiling (CGP) (Press release, Illumina, NOV 19, 2024, View Source [SID1234648507]). The assay is currently under development, with global release planned for mid-2025. Detailed plans for the product will be shared November 21 in a spotlight presentation at the annual meeting of the Association of Molecular Pathology (AMP) in Vancouver, British Columbia.

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TSO 500 v2 assesses hundreds of genes across all variant classes, and immuno-oncology biomarkers, in a single assay from one sample, to facilitate therapy selection research.

Key features of TSO 500 v2 include:

Faster turnaround time and reduced hands-on time
Sensitive variant calling and improved coverage of difficult genomic regions
Gold-standard Myriad Genomic Instability Score (GIS) to determine homologous recombination deficiency (HRD) status, included for all samples
New kit configurations with 50% less packaging, 70% fewer tubes, and improved usability
Automation methods available with flexible batch sizes
Integrated and automated data analysis, from sequencer to insights supported with DRAGEN secondary analysis and Illumina Connected Insights, or Velsera’s Clinical Genomics Workspace (CGW)
Broad platform compatibility
At AMP, several abstracts accepted for poster presentations will demonstrate preliminary analytical performance data and automation compatibility of TSO 500 v2. The studies support the assay’s applications for clinical research in identifying rare genetic biomarkers and fusion biomarkers.

TSO 500 v2 early access customer response

Wei Song, MD, PhD, director of Clinical Genomics and Molecular Pathology at the University of California, San Diego, is an early access customer testing TSO 500 v2.

"We are excited to assess the new features of the TruSight Oncology 500 v2 tissue assay, such as the faster workflow, improved coverage, and lower DNA/RNA input levels," he said. "Such improvements are highly relevant to clinical research laboratories and can positively affect time to results, quality of results, and the processing of challenging tissue samples."

TruSight Oncology portfolio enables clinical research and in-vitro diagnostic CGP solutions

Illumina’s oncology portfolio has evolved over time to fit customers’ diverse needs for versatility and scalability. The TSO portfolio encompasses research-use-only (TSO 500 products) and in-vitro diagnostic (TSO Comprehensive) solutions across a range of low-, mid-, and high-throughput instrumentation. Illumina also announced today that TSO Comprehensive kits are now available to ship. In August, the company announced FDA approval of its TSO Comprehensive test and its first two companion diagnostic indications.

"In listening to our broad range of customers, we understand there are different needs for biomarker profiling, and Illumina has successfully introduced a continuum of solutions to address varying levels of need," said Traci Pawlowski, vice president of Clinical Solutions at Illumina.

CGP is a critical tool for identifying actionable alterations, including rare mutations, which enables precision medicine in accordance with professional guidelines. At AMP, several customer-led poster presentations will provide further evidence for the clinical utility of CGP and demonstrate its versatility in hospital and community oncology care settings.

Illumina will cohost a workshop with Bayer, featuring a panel discussion of key opinion leaders on the challenges and practice gaps in precision medicine implementation, and the coordination of a multidisciplinary team to ensure optimal biomarker detection and targeted therapy utilization

Johnson & Johnson to showcase strength of its broad hematology portfolio and pipeline at the 2024 American Society of Hematology Annual Meeting

On November 19, 2024 Johnson & Johnson (NYSE:JNJ) reported more than 90 abstracts featuring data from the Company’s differentiated blood cancer portfolio and pipeline will be presented at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego from December 7-10 (Press release, Johnson & Johnson, NOV 19, 2024, View Source;johnson-to-showcase-strength-of-its-broad-hematology-portfolio-and-pipeline-at-the-2024-american-society-of-hematology-annual-meeting-302310074.html [SID1234648506]). Clinical trial and real-world data will highlight the Company’s broad and expanding portfolio of hematologic therapies, deepening its leadership in novel approaches to treat multiple myeloma as well as myeloid and B-cell malignancies. Six additional abstracts focus on the Company’s commitment and patient insights in warm autoimmune hemolytic anemia (wAIHA), a rare autoantibody-driven disease, and fetal and neonatal alloimmune thrombocytopenia (FNAIT), an alloimmune disorder of pregnancy.

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"This year’s data line-up at ASH (Free ASH Whitepaper) highlights our unwavering commitment to transform outcomes for patients with hematologic malignancies," said Yusri Elsayed, M.D., M.H.Sc., Ph.D., Global Therapeutic Area Head, Oncology, Johnson & Johnson Innovative Medicine. "Our relentless pursuit to provide each person diagnosed with blood cancer with treatment options at every stage of their disease inspires us to continue driving innovation in this space."

"The breadth of scientific evidence being presented at ASH (Free ASH Whitepaper) speaks to our drive to deliver life-changing treatments for patients with blood cancer," said June Lanoue, U.S. President, Hematology, Johnson & Johnson Innovative Medicine. "We look forward to highlighting the latest clinical trial and real-world data that demonstrate how we are addressing unmet needs for these patients."

New data highlight progress across all treatment stages of multiple myeloma, including differentiated and promising combination regimens
Key clinical and real-world studies focus on providing healthcare professionals with important data that may help better inform their choice of treatment regimens for patients, including:

Phase 3 Randomized Study of DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) Monotherapy Versus Active Monitoring in Patients with High-Risk Smoldering Multiple Myeloma: Primary Results of the AQUILA Study (Oral #733)
Phase 3 Randomized Study of DARZALEX FASPRO + Bortezomib, Lenalidomide and Dexamethasone (VRd) Versus VRd Alone in Patients with Transplant-Ineligible Newly Diagnosed Multiple Myeloma or for Whom Transplant is Not Planned as Initial Therapy: Analysis of Minimal Residual Disease in the CEPHEUS Trial (Oral #362)
DARZALEX FASPRO Plus Lenalidomide Versus Lenalidomide Alone as Maintenance Therapy in Newly Diagnosed Multiple Myeloma After Transplant: Analysis of the Phase 3 AURIGA Study Among Clinically Relevant Subgroups (Oral #654)
Subcutaneous DARZALEX FASPRO + Bortezomib, Cyclophosphamide, and Dexamethasone (VCD) in Patients with Newly Diagnosed Light Chain Amyloidosis: Overall Survival and Final Major Organ Deterioration Progression-Free Survival Results from the Phase 3 ANDROMEDA Study (Oral #891)
CARVYKTI (ciltacabtagene autoleucel; cilta-cel) vs Standard of Care in Patients with Lenalidomide-Refractory Multiple Myeloma After 1–3 Lines of Therapy: Minimal Residual Disease Negativity in the Phase 3 CARTITUDE-4 Trial (Oral #1032)
Phase 3 Study of TECVAYLI (teclistamab-cqyv) in Combination with Lenalidomide and TECVAYLI Alone Versus Lenalidomide Alone in Newly Diagnosed Multiple Myeloma as Maintenance Therapy Following Autologous Stem Cell Transplantation: Safety Run-in Results from the MajesTEC-4/EMN30 Trial (Oral #494)
Phase 2 Study of TECVAYLI-Based Induction Regimens in Patients with Transplant-eligible Newly Diagnosed Multiple Myeloma: Results From the GMMG-HD10/DSMM-XX (MajesTEC-5) Trial (Oral #493)
Pharmacodynamic Signatures and Correlatives of Response in Patients with Relapsed/Refractory Multiple Myeloma Treated with TALVEY (talquetamab-tgvs) or TECVAYLI Plus DARZALEX (daratumumab) and Pomalidomide (Oral #594)
Continued clinical innovation in treatment of B-cell malignancies to be shown through new and updated data
Ongoing studies of IMBRUVICA (ibrutinib) fixed-duration combination provide an opportunity to demonstrate long-term benefits of IMBRUVICA in chronic lymphocytic leukemia. Key presentations:

First-Line IMBRUVICA Plus Venetoclax vs Chlorambucil Plus Obinutuzumab in Elderly or Comorbid Patients with Chronic Lymphocytic Leukemia: GLOW Study 64-Month Follow-Up and Adverse Event-Free Progression-Free Survival Analysis (Poster #1871)
Consistently High 5.5-Year Progression-Free Survival Rates in Patients with and without Bulky Baseline Lymphadenopathy ≥5 cm are Associated with High Undetectable Minimal Residual Disease (uMRD4) Rates After First-Line Treatment with Fixed-Duration Ibrutinib + Venetoclax for Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma in the Phase 2 CAPTIVATE Study (Poster #1869)
Initiating First-Line Fixed-Duration IMBRUVICA and Venetoclax in Patients with Chronic Lymphocytic Leukemia Improves Overall Survival Outcomes to Rates Approximating an Age-Matched General European Population (Poster #3254)
A suite of oral presentations from independent investigators will further inform the clinical understanding and application of IMBRUVICA in chronic lymphocytic leukemia, as well as its potential in the treatment of previously untreated mantle cell lymphoma.

Phase 1 program for the menin inhibitor bleximenib demonstrates commitment to addressing unmet needs in acute myeloid leukemia for patients with both KMT2Ar and NPM1m alterations
Johnson & Johnson is investigating new targets with a focus on unmet needs in myeloid malignancies. Data will be presented from the Company’s lead asset for the treatment of acute myeloid leukemia in both newly diagnosed and relapsed/refractory patients:

Phase 1b Study of Menin-KMT2A Inhibitor Bleximenib in Combination with Intensive Chemotherapy in Newly Diagnosed Acute Myeloid Leukemia with KMT2Ar or NPM1 Alterations (Oral #215)
Bleximenib Dose Optimization and Determination of RP2D From a Phase 1 Study in Relapsed/Refractory Acute Leukemia Patients with KMT2A and NPM1 Alterations (Oral #212)
Research showcases unmet need in hematologic allo- and autoantibody-driven diseases including wAIHA and FNAIT
Johnson & Johnson studies on the lived experience of patients and utilization of health resources in people living with wAIHA highlight the hardship faced by those impacted by the disease and need for research into investigational treatment options that may offer sustained disease control and minimize disease exacerbations. Additionally, an overview of an ongoing Phase 3 FNAIT clinical study design will be shared.

Health Resource Utilization Among Patients with Warm Autoimmune Hemolytic Anemia in Sweden: A Retrospective Registry-Based Study (Poster #2255)
A Retrospective Database Analysis of Healthcare Resource Utilization in Patients with Warm Autoimmune Hemolytic Anemia in the United States (Poster #2324)
Sentiment analysis applied to digital conversations among Warm Autoimmune Hemolytic Anemia patients receiving rituximab and/or blood transfusion (Poster #3705)
Design of a Phase 3, Multicenter, Randomized, Open-Label Study of Nipocalimab or IVIG in Pregnancies at Risk for Fetal and Neonatal Alloimmune Thrombocytopenia (FREESIA-3) (Poster #1193.1)
Insights on the Lived Experience of Warm Autoimmune Hemolytic Anemia from an Ongoing Patient Council (Online Only)
Qualitative Examination of Treatment Experiences Among Individuals Living with Warm Autoimmune Hemolytic Anemia (Online Only)
Information on Johnson & Johnson sponsored abstracts is available on JNJ.com.

About multiple myeloma

Multiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.1 In multiple myeloma, these plasma cells proliferate and spread rapidly and replace normal cells in the bone marrow with tumors.2 Multiple myeloma is the third most common blood cancer worldwide and remains an incurable disease.3 In 2024, it was estimated that more than 35,000 people will be diagnosed with multiple myeloma in the U.S. and more than 12,000 people would die from the disease.4 People living with multiple myeloma have a 5-year survival rate of 59.8 percent.5 While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels and kidney problems or infections.6,7

About smoldering multiple myeloma

Smoldering multiple myeloma is an asymptomatic precursor state to multiple myeloma (MM). Patients with SMM have higher levels of abnormal plasma cells in the bone marrow and an elevated monoclonal protein (M-protein) level in the blood, but they do not yet exhibit the symptoms commonly associated with active multiple myeloma, particularly end-organ damage. Fifteen percent of all cases of newly diagnosed multiple myeloma are classified as smoldering multiple myeloma, and half of those diagnosed with high-risk disease will progress to active multiple myeloma within two years.8

About warm autoimmune hemolytic anemia

Warm autoimmune hemolytic anemia (wAIHA) is a rare, life-threatening condition where autoantibodies lead to the premature destruction of red blood cells (RBCs), resulting in anemia, which can cause symptoms like debilitating fatigue, dizziness, shortness of breath, jaundice and in severe cases, chest pain or loss of consciousness.9 Approximately 1-3 new people per 100,000 are affected by wAIHA per year, and about 1 in 8,000 individuals are living with the condition.9,10 This condition affects both women and men and can affect people at any age with incidence increasing over the age of 50.10,11

There are no Food and Drug Administration (FDA)-approved drugs indicated for wAIHA, and treatment typically consists of corticosteroids, broad immunosuppressants and B-cell directed therapies.9 With an unmet need for treatment in wAIHA, continued research for evidence-based potential therapies is critical.12

About fetal and neonatal alloimmune thrombocytopenia

Fetal and neonatal alloimmune thrombocytopenia (FNAIT) is a rare and potentially life-threatening alloimmune condition in which a pregnant person’s immune system develops alloantibodies against fetal or newborn platelet antigens, leading to thrombocytopenia (low platelet counts) in the fetus or newborn.13

FNAIT can result in severe bleeding complications for a fetus or newborn and is characterized by organ bleeding in the gastrointestinal tract, lungs, or eyes.13 If a severe bleed occurs in the brain, termed intracranial hemorrhage (ICH), death or life-long neurologic effects may occur.13 ICH occurs in up to 26 percent of untreated pregnancies with FNAIT.14

It has an estimated incidence rate of 1 in 1000 pregnancies.13,15 There are no approved therapies for the treatment of FNAIT. Because FNAIT is not routinely screened for during pregnancy, the diagnosis of an affected FNAIT pregnancy often occurs postnatally.13

About DARZALEX and DARZALEX FASPRO

DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) received U.S. FDA approval in May 2020 and is approved for nine indications in multiple myeloma, four of which are for frontline treatment in newly diagnosed patients who are transplant eligible or ineligible.16 It is the only subcutaneous CD38-directed antibody approved to treat patients with MM. DARZALEX FASPRO is co-formulated with recombinant human hyaluronidase PH20, Halozyme’s ENHANZE drug delivery technology.

DARZALEX (daratumumab) received U.S. FDA approval in November 2015 and is approved in eight indications, three of which are in the frontline setting, including newly diagnosed patients who are transplant eligible and ineligible.17

DARZALEX is the first CD38-directed antibody approved to treat multiple myeloma.17 DARZALEX-based regimens have been used in the treatment of more than 585,000 patients worldwide and more than 239,000 patients in the U.S. alone.

In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive license to develop, manufacture and commercialize daratumumab.

Since 2020, the National Comprehensive Cancer Network (NCCN) has recommended daratumumab-based combination regimens for the treatment of newly diagnosed multiple myeloma and relapsed and refractory multiple myeloma.† For newly diagnosed multiple myeloma in non-transplant candidates, the NCCN guidelines recommend daratumumab in combination with lenalidomide and dexamethasone as a Category 1 preferred regimen; daratumumab in combination with bortezomib, melphalan, and prednisone as another recommended Category 1 regimen; and daratumumab in combination with bortezomib, cyclophosphamide, and prednisone as another recommended Category 2A regimen. For newly diagnosed multiple myeloma in transplant candidates, the NCCN guidelines recommend daratumumab in combination with bortezomib, lenalidomide and dexamethasone as another recommended Category 2A regimen; daratumumab in combination with bortezomib, thalidomide and dexamethasone as a Category 2A regimen useful in certain circumstances; daratumumab in combination with carfilzomib, lenalidomide and dexamethasone as a Category 2A regimen useful in certain circumstances; and daratumumab in combination with cyclophosphamide, bortezomib and dexamethasone as a Category 2A regimen useful in certain circumstances. For maintenance in transplant candidates, the NCCN guidelines recommend daratumumab in combination with lenalidomide as useful in certain circumstances. In relapsed/refractory myeloma, four daratumumab regimens are listed as Category 1 preferred regimens for early relapses (1-3 prior therapies): daratumumab in combination with lenalidomide and dexamethasone; daratumumab in combination with bortezomib and dexamethasone; daratumumab in combination with carfilzomib and dexamethasone; and daratumumab in combination with pomalidomide and dexamethasone [after one prior therapy including lenalidomide and a proteasome inhibitor]. The NCCN also recommends daratumumab in combination with cyclophosphamide, bortezomib and dexamethasone as another Category 2A regimen for early relapses (1-3 prior therapies) and as monotherapy as a Category 2A regimen useful in certain circumstances for early relapse patients after at least three prior therapies, including a proteasome inhibitor and an immunomodulatory agent, or for patients who are double refractory to a PI and an immunomodulatory agent.

For more information, visit www.DARZALEX.com.

About CARVYKTI

CARVYKTI is a BCMA-directed, genetically modified autologous T-cell immunotherapy that involves reprogramming a patient’s own T-cells with a transgene encoding chimeric antigen receptor (CAR) that directs the CAR-positive T cells to eliminate cells that express BCMA. BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B cells and plasma cells. The CARVYKTI CAR protein features two BCMA-targeting single domains designed to confer high avidity against human BCMA. Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.

CARVYKTI (cilta-cel) received U.S. Food and Drug Administration approval in February 2022 for the treatment of adults with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. In April 2024, CARVYKTI was approved in the U.S. for treatment of adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy including a proteasome inhibitor, an immunomodulatory agent, and who are refractory to lenalidomide, following a unanimous (11 to 0) FDA Oncologic Drugs Advisory Committee (ODAC) recommendation in support of this new indication. In April 2024, the European Medicines Agency (EMA) approved a Type II variation for CARVYKTI for the treatment of adults with relapsed and refractory multiple myeloma who have received at least one prior therapy, including an immunomodulatory agent and a proteasome inhibitor, have demonstrated disease progression on the last therapy, and are refractory to lenalidomide.

In December 2017, Janssen Biotech, Inc., a Johnson & Johnson company, entered into an exclusive worldwide license and collaboration agreement with Legend Biotech USA, Inc. to develop and commercialize CARVYKTI.

For more information, visit www.CARVYKTI.com.

About TECVAYLI

TECVAYLI (teclistamab-cqyv) received approval from the U.S. FDA in October 2022 as an off-the-shelf (or ready-to-use) antibody that is administered as a subcutaneous treatment for adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody.18 The EC granted TECVAYLI conditional marketing authorization (CMA) in August 2022 as monotherapy for the treatment of adult patients with RRMM who have received at least three prior therapies, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody, and have demonstrated disease progression since the last therapy. In August 2023, the EC granted the approval of a Type II variation application for TECVAYLI, providing the option for a reduced dosing frequency of 1.5 mg/kg every two weeks in patients who have achieved a complete response or better for a minimum of six months. TECVAYLI is a first-in-class, bispecific T-cell engager antibody therapy that uses innovative science to activate the immune system by binding to the CD3 receptor expressed on the surface of T-cells and to the B-cell maturation antigen (BCMA) expressed on the surface of multiple myeloma cells and some healthy B-lineage cells. In February 2024, the U.S. FDA approved the supplemental Biologics License Application for TECVAYLI for a reduced dosing frequency of 1.5 mg/kg every two weeks in patients with relapsed or refractory multiple myeloma who have achieved and maintained a CR or better for a minimum of six months.

For more information, visit www.TECVAYLI.com.

About TALVEY 

TALVEY (talquetamab-tgvs) received approval from the U.S. FDA in August 2023 as a first-in-class GPRC5D-targeting bispecific antibody for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.19 Since FDA approval, 1,800 patients were treated with TALVEY. The European Commission (EC) granted conditional marketing authorization (CMA) of TALVEY (talquetamab-tgvs) in August 2023 as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.20

TALVEY is a bispecific T-cell engaging antibody that binds to the CD3 receptor expressed on the surface of T-cells and G protein-coupled receptor class C group 5 member D (GPRC5D), a novel multiple myeloma target which is highly expressed on the surface of multiple myeloma cells and non-malignant plasma cells, as well as some healthy tissues such as epithelial cells of the skin and tongue.  

About IMBRUVICA

IMBRUVICA (ibrutinib) is a once-daily oral medication that is jointly developed and commercialized by Janssen Biotech, Inc., and Pharmacyclics LLC, an AbbVie company. IMBRUVICA blocks the BTK protein, which is needed by normal and abnormal B cells, including specific cancer cells, to multiply and spread. By blocking BTK, IMBRUVICA may help move abnormal B cells out of their nourishing environments and inhibit their proliferation.21,22, 23

IMBRUVICA is approved in more than 100 countries and has been used to treat more than 300,000 patients worldwide over the last decade. There are more than 50 company-sponsored clinical trials, including 18 Phase 3 studies, spanning more than 11 years evaluating the efficacy and safety of IMBRUVICA.

IMBRUVICA was first approved by the U.S. FDA in November 2013, and today is indicated for adult patients in four disease areas. These include indications to treat adults with chronic lymphocytic leukemia/small lymphocytic lymphoma with or without 17p deletion; adults with Waldenström’s macroglobulinemia; and adult and pediatric patients aged one year and older with previously treated chronic graft versus host disease after failure of one or more lines of systemic therapy.24

About Nipocalimab

Nipocalimab is an investigational monoclonal antibody, designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies potentially without impact on other immune functions. This includes autoantibodies and alloantibodies that underlie multiple conditions across three key segments in the autoantibody space including Rare Autoantibody diseases, Maternal Fetal diseases mediated by maternal alloantibodies and Prevalent Rheumatology.25,26,27,28,29,30,31,32,33 Blockade of IgG binding to FcRn in the placenta is also believed to limit transplacental transfer of maternal alloantibodies to the fetus.34,35

The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted several key designations to nipocalimab including:  

U.S. FDA Fast Track designation in hemolytic disease of the fetus and newborn (HDFN) and wAIHA in July 2019, gMG in December 2021 and FNAIT in March 2024
U.S. FDA Orphan drug status for wAIHA in December 2019, HDFN in June 2020, gMG in February 2021, chronic inflammatory demyelinating polyneuropathy (CIDP) in October 2021 and FNAIT in December 2023
U.S. FDA Breakthrough Therapy designation for HDFN in February 2024 and for Sjögren’s disease (SjD) in November 2024
EU EMA Orphan medicinal product designation for HDFN in October 2019