Globenewswire | June 01, 2023
The Janssen Pharmaceutical Companies of Johnson & Johnson today announced the submission of a Type II variation application to the European Medicines Agency (EMA) seeking approval of a new indication for CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) for the treatment of adult patients with relapsed and lenalidomide-refractory multiple myeloma.
“The previous European Commission approval recognised the potential for cilta-cel to positively impact outcomes for people living with relapsed and refractory multiple myeloma,” said Edmond Chan, MBChB M.D. (Res), Senior Director, EMEA Therapeutic Area Lead Haematology, Janssen-Cilag Limited. “Today’s submission to the EMA is an important step towards helping patients benefit from this CAR-T therapy earlier in their treatment journey. If approved, this will be the first and only CAR-T therapy available to treat relapsed and refractory multiple myeloma patients as early as second line.”
The application is supported by data from the CARTITUDE-4 study the first randomised Phase 3 study evaluating the efficacy and safety profile of cilta-cel versus pomalidomide, bortezomib and dexamethasone (PVd) or daratumumab, pomalidomide and dexamethasone (DPd) in the treatment of patients with relapsed and lenalidomide-refractory multiple myeloma who received one to three prior lines of therapy.1
The CARTITUDE-4 study results will be presented in a special session at the upcoming American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, IL on Monday 5 June 2023 at 16:45 CEST.
“This submission is a testament to our relentless commitment to advance science, transform outcomes, challenge what a multiple myeloma diagnosis means for patients and ultimately, work towards our goal of one day curing this complex disease,” said Sen Zhuang, M.D., Ph.D., Vice President, Clinical Research and Development, Janssen Research & Development, LLC. “We look forward to collaborating with the EMA to bring this potential new indication for cilta-cel to the multiple myeloma community as soon as possible.”
About Ciltacabtagene Autoleucel (cilta-cel)
Cilta-cel received conditional marketing authorisation from the European Commission in May 2022, for the treatment of adults 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.3,4 In February 2022, the FDA approved cilta-cel 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.5
Cilta-cel is a B-cell maturation antigen (BCMA)-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient’s own T-cells with a transgene encoding a chimeric antigen receptor (CAR) that identifies and eliminates cells that express BCMA.6 BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells.7,8 The cilta-cel CAR protein features two BCMA-targeting single domain antibodies designed to confer high avidity against human BCMA.6 The CAR-modified T-cells express fusion proteins of antigen receptors against tumour-associated surface antigens and T-cell activation domains, and upon binding to BCMA-expressing cells redirect the effector T-cells and enhance tumour-specific immunosurveillance.9
In December 2017, Janssen Biotech, Inc. (Janssen) entered into a worldwide license and collaboration agreement with Legend Biotech USA, Inc. to develop and commercialise cilta-cel.10
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.11 In multiple myeloma, these malignant plasma cells change and grow out of control.8 In Europe, more than 50,900 people were diagnosed with multiple myeloma in 2020, and more than 32,400 patients died.12 While 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, tiredness, high calcium levels, or kidney failure.13
About the Janssen Pharmaceutical
At Janssen, we're creating a future where disease is a thing of the past. We're the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.
Businesswire | May 22, 2023
Closed Loop Medicine Ltd, a leading TechBio company, developing combination prescription drug plus software therapy products that enable personalized dose optimization, and Pharmanovia, a global pharmaceutical company that commercializes novel medicines and revitalizes, extends and expands the lifecycle of established medicines, today announced that they have entered a global co-development partnership.
The partnership will initially focus on the development and launch of a drug + software combination version of a first-line anti-hypertensive in the UK, before phased global roll-out and additional therapies are added.
Hypertension is the leading preventable cause of morbidity and premature death worldwide, impacting over 1 in 4 men and 1 in 5 women, and costing the NHS alone over £2.1 billion/year (1, 2). Managing high-blood pressure through medication and lifestyle modifications significantly reduces associated health risks and improves quality of life; research in the US demonstrating improved standards of care could prevent 91,900 heart attacks, 139,000 strokes and 115,400 cardiovascular deaths within just 5 years (3). However, poor drug tolerability and treatment-related side effects significantly impact adherence, with less than 50% of patients maintaining their treatment regimen after a year, ultimately hindering treatment effectiveness, patient outcomes and overall disease management (4).
As part of this partnership, Closed Loop Medicine’s proprietary Software-as-a-Medical Device (SaMD) dosing optimization technology will be combined with Pharmanovia’s established anti-hypertensive medicines, under a single prescription. Recent preliminary clinical trial results have demonstrated that Closed Loop Medicine’s technology may provide the ability to dose optimize drug therapy, improve blood pressure control, and minimize side effects (5). By developing a new class of combination products for the treatment of hypertension, both companies aim to support patients and healthcare professionals to better manage high blood pressure.
Dr. Hakim Yadi OBE, CEO & Co-Founder of Closed Loop Medicine, commented: “This pioneering partnership is a great example of where the power of software integration can add value to iconic medicines to make them more effective for patients. Through this co-development agreement, we will bring our IP, insight and technical know-how of dose optimized drug plus software integration alongside Pharmanovia’s innovative business model, to enable well-known and trusted brands to be positioned as value-driven precision medicine product solutions globally.”
Dr. James Burt, CEO of Pharmanovia, said: “Combining our experience in lifecycle management with Closed Loop Medicine’s unique approach and technology is an exciting next step in our strategy. It enables us to use innovative technology to enhance established medicines and bring much needed innovation to an area of large-scale unmet need. Together we aim to deliver a first in class combination regulated software and medicine – with the aim of delivering better patient outcomes, while enabling healthcare systems to reduce costly hospital procedures through better adherence and patient engagement.”
Pharmanovia is a global lifecycle management healthcare company. Our purpose is to make medicines fit for tomorrow, to improve the lives of patients globally. We do this by enhancing established medicines either by rediscovering, repurposing or re-engineering iconic brands to improve patient outcomes and experiences both through in-house development and through strategic partnerships.
BUSINESS INSIGHTS, PHARMA TECH
businesswire | April 19, 2023
Simulations Plus, Inc. a leading provider of modeling and simulation software and services for pharmaceutical safety and efficacy, announced that the U.S. FDA has renewed its licenses to the DILIsym® software platform, the flagship quantitative systems toxicology (QST) software for predicting and investigating drug-induced liver injury (DILI). The one-year renewal provides continued access to DILIsym software for authorized FDA employees across all FDA divisions.
Dr. Paul B. Watkins, chair of the Scientific Advisory Board of the DILI-sim Initiative, commented, “The FDA’s renewal of these licenses endorses the value of DILIsym software in both predicting liver safety liabilities and defining the underlying mechanisms, which are critical to evaluating risk. The recent signing of the FDA Modernization Act 2.0 by President Biden, which encourages the FDA to limit animal use in drug development, should be a positive catalyst for greater utilization of DILIsym software in drug development. In my role as Chair of the SAB, I am very excited about the direction of the public-private partnership that is guiding further development of the DILIsym platform that will benefit many key stakeholders.”
Dr. Brett A. Howell, President of the DILIsym Services division at Simulations Plus, added, “Given the effect of rising costs on providing vital healthcare, the use of novel simulation technologies to improve the efficiency of drug development is more critical than ever. An important first step is ensuring that regulatory agencies have the most advanced technology tools to fulfill their essential mission. We are delighted the FDA has opted to renew once again and will continue using DILIsym and other Simulations Plus tools to enable reviews and enhance regulatory science.”
DILIsym software results support crucial drug development decisions by predicting potential DILI risk of new drug candidates. The modeling also identifies the biochemical events that lead to DILI caused by a drug and can thereby predict certain subgroups of patients at increased risk for DILI from that drug. The information from DILIsym modeling serves to help guide go/no-go decisions on major drug development projects, potentially avoiding the disastrous financial effects of failed clinical trials, or better, providing assurances that DILI will not be an insurmountable obstacle to regulatory approval. For the past 13 years, DILIsym Services, a division of Simulations Plus, has coordinated the DILI-sim Initiative, which is a public-private partnership that has guided development of the DILIsym software package. DILIsym is available to the pharmaceutical and chemical industries for direct use to predict and understand liabilities via membership in the DILI-sim Initiative consortium and/or commercial licenses. The DILIsym Services division also routinely uses DILIsym for comprehensive consulting services on safety-related issues.
Companies interested in a free trial version of the DILIsym software can request it here.
About Simulations Plus
Serving clients worldwide for more than 25 years, Simulations Plus is a leading provider in the biosimulation market providing software and consulting services supporting drug discovery, development, research, and regulatory submissions. We offer solutions that bridge artificial intelligence (AI)/machine learning, physiologically based pharmacokinetics, quantitative systems pharmacology/toxicology, and population PK/PD modeling approaches. Our technology is licensed and applied by major pharmaceutical, biotechnology, and regulatory agencies worldwide.