NICE recommends Revlimid as multiple myeloma alternative

pharmatimes | May 17, 2019

The National Institute of Health and Care Excellence (NICE) has recommended Revlimid (lenalidomide), a drug that helps to support the immune system to fight blood cancer. The treatment will now be available on the NHS for patients with a form of blood cancer as a first- and second-line drug, after a new guideline recommended it in combination with dexamethasone as an option for previously untreated multiple myeloma in adults who are not eligible for a stem cell transplant and cannot take thalidomide. The new indication will benefit approximately 2,100 patients, as the company has a commercial arrangement (via the patient access scheme) which makes lenalidomide available to the NHS with a discount. The new drug will “not only improve the length of time people live, but it will also have fewer side effects compared with current treatments”, said Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE.

Spotlight

New cancer treatment drugs have been added to the United Nations list of essential medicines, announced the World Health Organization on July 10. These cancer drugs don’t have to be injected. They can be swallowed.

Spotlight

New cancer treatment drugs have been added to the United Nations list of essential medicines, announced the World Health Organization on July 10. These cancer drugs don’t have to be injected. They can be swallowed.

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BUSINESS INSIGHTS

Sanofi Global Health launches nonprofit Impact® brand for 30 medicines in low-income countries

Sanofi | July 05, 2022

Sanofi Global Health announces the launch of Impact®, a new brand of standard of care medicines produced by Sanofi dedicated for nonprofit distribution to at-risk populations in the world’s most impoverished countries. The Impact® brand, which includes insulin, glibenclamide and oxaliplatin amonst others, will enable the secure distribution of 30 Sanofi medicines in 40 lower-income countries. Considered essential by the World Health Organization, the medicines cover a wide range of therapeutic areas, including diabetes, cardiovascular disease, tuberculosis, malaria and cancer. The launch of the Impact® brand is among the steps taken since the formation last year of Sanofi Global Health, a nonprofit unit within the company aiming to increase access to healthcare through the distribution of medicines, and the building and bolstering of local healthcare systems in countries with among the lowest per capita GDP. Sanofi Global Health is the first and only global initiative to provide access to such a broad portfolio of medicines in so many countries and across multiple therapeutic areas while funding local support programs and strengthening local inclusive businesses. “At Sanofi, we believe we have a responsibility to make a difference for the health of those most in need, and we know we have the ability and the ambition to bring about lasting change. With critical medicines, relentless drive and impactful partnerships, we can take our innovation beyond the lab and use it to strengthen health systems and access to medicines for those most vulnerable communities of patients. Sanofi Global Health aims to improve the lives of millions of people who now cannot get the help they need. Sanofi’s renewed purpose is to chase the miracles of science to improve people’s lives. And our quest to make life better for all people must include helping to provide better access to care and quality medicines for underserved populations.” Paul Hudson Chief Executive Officer, Sanofi The company also announces the establishment of an Impact fund that will support startup companies and other innovators that can deliver scalable solutions for sustainable healthcare in underserved regions. By providing inclusive businesses financing and technical assistance, the fund will complement the GHU mission of leveraging global, regional and local investment to support the training of healthcare professionals and aiding communities in running sustainable care systems. The announcements come as Sanofi gathers key global health stakeholders to discuss how to build effective end-to-end health programs that are embedded in the communities in which they serve, to best reach, treat and manage patients’ health effectively and sustainably. About Sanofi We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.

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PHARMA TECH

Antengene Announces Clinical Trial Collaboration with BeiGene to Evaluate Selinexor in Combination with Tislelizumab in T and NK-Cell Lymphoma

Antengene Corporation Limited | June 27, 2022

Antengene Corporation Limited a leading innovative, commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class therapeutics in hematology and oncology, today announced that it has entered into a clinical trial collaboration with BeiGene to evaluate the safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of selinexor in combination with BeiGene's anti-PD-1 checkpoint inhibitor, tislelizumab. This multi-center, open-label Phase I/II trial will evaluate the investigational combination as a potential treatment option for patients with T and NK-cell lymphoma. "We are delighted to partner with BeiGene, a company that strives for innovation and excellence, and is committed to developing best-in-class or first-in-class anti-cancer therapies for patients across the globe. These qualities are very similar to those of our vision at Antengene. We look forward to advancing the combination of selinexor and tislelizumab to clinical development. With good data we will be able to bring this treatment regimen to patients with T and NK-cell lymphoma, diseases that are endemic in Asia but underserved by current therapies." Dr. Jay Mei, Antengene's Founder, Chairman and CEO "At Antengene, we believe that the combinational use of immuno-oncology drugs and Selective Inhibitor of Nuclear Export compounds possesses huge potential as novel treatment regimens for cancer patients," said Dr. Kevin Lynch, Antengene's Chief Medical Officer. "The mechanism of action of selinexor in inhibiting the nuclear export protein XPO1 facilitates the intranuclear accumulation of tumor suppressors, making it a good partner in multiple combination treatment regimens. Preclinical research we conducted demonstrated that selinexor combined with a checkpoint inhibitor increased anti-tumor activity in multiple tumor models. In addition, deep and durable responses were also seen in multiple case reports of patients with T and NK-cell lymphoma treated with selinexor in combination with an anti-PD-1 checkpoint inhibitor. We hope to confirm that selinexor can synergize with tislelizumab to deliver an effective treatment regimen and help address the huge unmet medical needs in T and NK-cell lymphoma in the Asia Pacific regions and around the world." continued Dr. Lynch. Tislelizumab is a PD-1 inhibitor designed to help aid the body's immune cells to detect and fight tumors. Tislelizumab, a humanized monoclonal antibody, is specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. About T and NK-Cell Lymphoma T and NK-cell lymphoma is a set of heterogeneous diseases, accounting for 25-30% of Non-Hodgkin Lymphoma cases in China and only about 10% in USA and Europe. There has been little improvement in the past decade when compared to B-cell Non-Hodgkin Lymphoma (B-NHL) as 5-year overall survival rate was only 30% in most common subtypes[1]. The unmet medical needs remain as agents with new mechanism of action to be explored and possibility to improve the treatment paradigm for the disease. About the SINE Compounds Selective Inhibitor of Nuclear Export compounds are inhibitors of the major nuclear export protein Exportin 1 Currently, there are three oral SINE compounds, ATG-010 ATG-016 and ATG-527, under clinical development. Antengene has obtained exclusive development and commercialisation rights from Karyopharm Therapeutics Inc. to these three compounds in certain APAC markets. About XPOVIO® XPOVIO® is the world's first approved orally-available, selective inhibitor of the nuclear export protein XPO1. It offers a novel mechanism of action, synergistic effects in combination regimens, fast onset of action, and durable responses. XPOVIO® is approved in Australia for two indications In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma (MM) who have received at least one prior therapy. In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least three prior therapies and whose disease is refractory to at least one proteasome inhibitor at least one immunomodulatory agent, and an anti-CD38 monoclonal antibody. XPOVIO® is approved in Singapore for three indications In combination with bortezomib and dexamethasone for treatment of adult patients with multiple myeloma (MM) who have received at least one prior therapy. In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. As a monotherapy for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy who are not eligible for haematopoietic cell transplant. About Antengene Antengene Corporation Limited is a leading commercial-stage R&D-driven global biopharmaceutical company focused on the discovery, development, manufacturing and commercialization of innovative first-in-class/best-in-class therapeutics for the treatment of hematologic malignancies and solid tumors, in realizing its vision of "Treating Patients Beyond Borders". About BeiGene BeiGene is a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide. With a broad portfolio of more than 40 clinical candidates, we are expediting development of our diverse pipeline of novel therapeutics through our own capabilities and collaborations. We are committed to radically improving access to medicines for two billion more people by 2030. BeiGene has a growing global team of over 8,000 colleagues across five continents.

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BUSINESS INSIGHTS

C-Path and Replica Analytics Collaboration Aims to Accelerate Rare Disease Research

Replica Analytics | June 30, 2022

Critical Path Institute and Replica Analytics, an Aetion company, announced a new partnership that involves leveraging synthetic data to further catalyze the generation of actionable solutions to accelerate drug development for rare diseases. Replica Analytics will help generate synthetic datasets across rare and orphan indications in which patient-level datasets are often quite small, which heightens considerations regarding data privacy and accessibility. Together with real data, these synthetic data will help maximize the utility of C-Path's Rare Disease Cures Accelerator-Data and Analytics Platform which provides a centralized and standardized infrastructure to support and accelerate rare disease characterization targeted to accelerate drug development. Additionally, the platform includes a framework that supports the rigorous conduct of natural history studies, with attention to established data quality standards, to be most useful to clinical trial design and regulatory review. It includes a robust, integrated database and analytics hub that allows for the aggregation of rare disease data from various sources and the efficient and effective interrogation of that data. Once added to RDCA-DAP, the synthetic data will also represent valuable real-world, electronic health record (EHR) based data available on the platform, opening doors to modeling and research based on longitudinal focused analyses. "Our collaboration with Replica Analytics is important because synthetic data can add value to real-world data while observing data privacy considerations, which will help accelerate overall rare disease drug development. The goal of RDCA-DAP is to provide a centralized and standardized infrastructure to support and accelerate rare disease characterization and therapy development, this collaboration is in line with our efforts." RDCA-DAP Scientific Director Alexandre Betourne, Pharm.D., Ph.D Synthetic data generation is a privacy enhancing technology that has been gaining rapid adoption, particularly in the life sciences sector. SDG uses AI to create machine learning models that learn the statistical patterns and properties of real datasets to generate data that retain the same characteristics as the original dataset, but with no one-to-one mapping back to an identifiable person. SDG can help amplify small datasets, simulate virtual patients to augment patients in existing datasets, and optimize the design of small sample clinical trials. "We are certainly seeing a growing opportunity to partner with organizations like C-Path for generating datasets that are fit-for-purpose," says Khaled El Emam, Ph.D., SVP and GM of Replica Analytics. "Synthetic data, which preserves the integrity and utility of source data, as well as being privacy-protective, can be a very valuable tool to enable this collaboration." In May 2022, C-Path hosted a webinar in which the Replica Analytics team introduced SDG and discussed the role the technology can play in researching and treating rare diseases. The event was recorded and can be viewed on C-Path's YouTube channel. About C-Path Critical Path Institute is an independent, non-profit organization established in 2005 as a public and private partnership. C-Path's mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. Critical Path Institute is supported by the Food and Drug Administration of the U.S. Department of Health and Human Services and is 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government sources, totaling $11,196,634. The contents are those by the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government. About Replica Analytics, an Aetion company Replica Analytics is the premier science-based SDG technology provider to the healthcare industry. The company a pioneer in the development of unique technologies for generating privacy-protective synthetic data that maintain the statistical properties of real-world data. The company was acquired in late 2021 by Aetion, the leading regulatory-grade real-world evidence technology provider. Replica Synthesis software provides a full suite of synthetic data generation and evaluation capabilities that can solve multiple grand challenges facing the life sciences industry, and health research in general. About Aetion Aetion is a healthcare analytics company that delivers real-world evidence for the manufacturers, purchasers, and regulators of medical treatments and technologies. The Aetion Evidence Platform® analyzes data from the real world to produce transparent, rapid, and scientifically validated answers on safety, effectiveness, and value. Founded by Harvard Medical School faculty members with decades of experience in epidemiology and health outcomes research, Aetion informs healthcare's most critical decisions—what works best, for whom, and when—to guide product development, commercialization, and payment innovation.

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