BUSINESS INSIGHTS

Can-Fite Granted Key NASH Patent in Israel

Can-Fite BioPharma Ltd. | May 17, 2022

Can-Fite
Can-Fite BioPharma Ltd. a biotechnology company advancing a pipeline of proprietary small molecule drugs that address inflammatory, cancer and liver diseases, announced that its patent titled "An A3 Adenosine Receptor Ligand For Use In Treating Ectopic Fat Accumulation" has been granted by the Israel Patent Office.

This patent has been issued in approximately 40 countries and territories including Japan, South Korea, Hong Kong, Mexico, and in the European Union. It addresses the use of the A3 Adenosine Receptor ligand, the target receptor for Can-Fite's drug platform technology, for the treatment of ectopic fat accumulation particularly in fatty liver as manifested in non-alcoholic fatty liver disease and non-alcoholic steatohepatitis. The treatment of NASH is a market estimated to reach $35 billion by 2025.

Can-Fite is currently enrolling and treating patients in a Phase IIb NASH study of its liver drug candidate Namodenoson. The multi-center, randomized, double blind, and placebo controlled study of biopsy-confirmed NASH patients will measure efficacy periodically through biomarkers, with a primary efficacy endpoint determined by liver biopsy at the end of the treatment period. In a prior Phase IIa study, Namodenoson met endpoints including reduced liver fat content, anti-inflammatory effects, and decreased body weight with excellent safety.

The treatment of NASH is an enormous unmet need that Can-Fite seeks to meet through our advanced stage clinical trial and our expanding patent estate for the use of our target A3AR in the treatment of fatty liver disease,” 

Can-Fite CEO Dr. Pnina Fishman

About Namodenoson
Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor. Namodenoson is being evaluated in a pivotal Phase III trial as a second line treatment for hepatocellular carcinoma, and in a Phase IIb trial as a treatment for non-alcoholic steatohepatitis. A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.

About Can-Fite BioPharma Ltd.
Can-Fite BioPharma Ltd. is an advanced clinical stage drug development Company with a platform technology that is designed to address multi-billion dollar markets in the treatment of cancer, liver, and inflammatory disease. The Company's lead drug candidate, Piclidenoson has completed enrollment in a Phase III trial for psoriasis. Can-Fite's liver drug, Namodenoson, is being evaluated in a Phase IIb trial for the treatment of non-alcoholic steatohepatitis (NASH), and enrollment is expected to commence in a Phase III trial for hepatocellular carcinoma (HCC), the most common form of liver cancer. Namodenoson has been granted Orphan Drug Designation in the U.S. and Europe and Fast Track Designation as a second line treatment for HCC by the U.S. Food and Drug Administration. Namodenoson has also shown proof of concept to potentially treat other cancers including colon, prostate, and melanoma. CF602, the Company's third drug candidate, has shown efficacy in the treatment of erectile dysfunction. These drugs have an excellent safety profile with experience in over 1,500 patients in clinical studies to date. 

Spotlight

The collection and analysis of data generated outside of restricted clinical trials are now critical to pharma companies. Data gathered in the routine delivery of healthcare can be mined to uncover insights into treatment efficacies and outcomes in the real world. Such insights, known as real-world evidence (RWE), benefits teams working across the drug lifecycle by showing how larger, heterogeneous patient populations interact with the healthcare system and respond to medicines.


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BUSINESS INSIGHTS

Clinical research and drug development accelerated via analytics

SAS | June 22, 2022

Bringing life-changing therapies to market requires pharmaceutical companies to efficiently design and run clinical trials, gather and analyze massive amounts of data, and manage a complicated regulatory review process. SAS, a leader in clinical research analytics, delivers powerful technologies to support the life sciences industry – including machine learning, AI and analytics for IoT – through the SAS® Life Science Analytics Framework on Azure. The advanced analytics platform – a single, open, cloud-native statistical computing environment for clinical trial analysis and submission – helps pharmaceutical companies navigate the heavily regulated world of clinical research analytics and bring new therapies to the world faster. Delivering the power of analytics in the cloud From drug discovery to regulatory approval, developing a new medicine can take over a decade with a price tag in the billions. The clinical research required to achieve approval from the US Food and Drug Administration (FDA), European Medicines Agency (EMA), and other regulatory authorities involves massive amounts of data that can be difficult to manage, share and analyze, creating delays that affect patients' well-being. The cloud-based SAS Life Science Analytics Framework on Azure features an integrated and collaborative environment to manage and analyze clinical trial information as well as deliver trial results for the FDA and other global regulatory agencies' review. With the analytic solution, life sciences organizations can extract valuable insights from clinical data, mitigate risk, increase efficiency, and speed time to market for lifesaving pharmaceuticals. "The primary goal is to get drugs approved so that they can reach patients. The faster you can do that, the greater benefit for the patient and to help save lives." Bhawna Goel, CEO of Gunvatta Gunvatta USA, Inc, a contract research organization (CRO) headquartered in Washington, DC, helps clients collect, manage, analyze and visualize the terabytes of clinical and healthcare data produced by pharmaceutical and biotechnology companies, hospitals, and provider groups. With the SAS Life Science Analytics Framework, Gunvatta has modernized the way it approaches clinical trials. The CRO helps life sciences organizations reduce risk with secure data access, ensure data and results are validated, maintain an audit trail, and report their data to health authorities like the FDA. "The biggest advantage of the SAS Life Science Analytics Framework is that the analytics platform is cloud-based," said Goel. "I feel like SAS has jumped 15 to 20 steps ahead of where the market is." For more about Gunvatta's experience, see the customer story Helping pharmaceutical companies bring new therapies to the world faster using analytics for life sciences. Developing cutting-edge clinical research tools SAS life science experts will attend the DIA Global Annual Meeting this week in Chicago to share next generation approaches for driving excellence and innovation in clinical trial operations. At DIA, SAS will preview SAS Clinical Enrollment Simulation Cloud – a cloud-native, solution based in SAS® Viya® 4 – that enables life sciences and contract research organizations to simulate the outcome of the complex clinical trial enrollment process in a virtual world, resulting in faster, more strategic clinical trial enrollment plans. The new SaaS offering – available later this year on the Microsoft Azure Marketplace – uses a powerful discrete event simulation analytical engine to model the clinical trial enrollment process as it evolves over time, facilitating improved insights to guide enrollment strategy and meet contracted patient targets. "SAS Clinical Enrollment Simulation Cloud is highly differentiated from traditional methodologies for predicting trial enrollment," said Jim Box, Life Sciences Principal Data Scientist at SAS. "The technology helps life sciences organizations reduce timelines, minimize costs, and gain greater insight to inform clinical enrollment planning and rescue." Modernizing clinical trials for the future While the current clinical development research model can be an obstacle to the creation of safe, cost-effective therapies, the COVID-19 pandemic and other events set the stage for disruption in clinical trials. "The silver lining of the pandemic for the life sciences industry is the acceleration of digital transformation and modernization of clinical research," said Simon Tilley, Health and Life Sciences Product Director at SAS. "We experienced widespread adoption of innovative approaches, such as decentralized clinical trials for the development of new vaccines." "While the business benefits of modernizing clinical research include significant cost savings, efficiency improvements, accelerated time to regulatory approval and faster time to market, it is patients who stand to gain the most from clinical trial innovation." About SAS SAS is the leader in analytics. Through innovative software and services, SAS empowers and inspires customers around the world to transform data into intelligence. SAS gives you THE POWER TO KNOW®. SAS and all other SAS Institute Inc. product or service names are registered trademarks or trademarks of SAS Institute Inc. in the USA and other countries. ® indicates USA registration. Other brand and product names are trademarks of their respective companies. Copyright © 2022 SAS Institute Inc. All rights reserved.

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Spotlight Therapeutics Appoints Visionary Global Pharma Drug Developer Antoine Yver, M.D., M.Sc. as First Independent Board Member

Spotlight Therapeutics | July 18, 2022

Spotlight Therapeutics, Inc. a biotechnology company applying new insights to develop cell-targeted in vivo CRISPR gene editing biologics, announced the appointment of Antoine Yver, M.D., M.Sc. to the Spotlight Board of Directors. Trained as an oncologist and immunologist, Dr. Yver’s extensive industry experience and perceptive acumen will be extremely valuable as Spotlight continues to strengthen its highly differentiated TAGE platform and advance lead therapeutic candidates towards the clinic. “We are thrilled to welcome Antoine to Spotlight’s Board of Directors. Few people in our industry enjoy such a remarkable track record of successful drug development, exercising extreme rigor in following the science to deliver practice-changing medicines that serve patients with significant unmet medical needs.” Mary Haak-Frendscho, Ph.D., President and Chief Executive Officer of Spotlight As Executive Vice President and Global Head of Oncology R&D at Daiichi Sankyo, Dr. Yver played a key role in the development of the new breakthrough cancer biologic, Enhertu®, that is redefining breast cancer treatment and promises to set a new standard of care, as well as other Daiichi Sankyo DXd ADCs. From 2009 to 2016, Dr. Yver held executive leadership positions at AstraZeneca, including SVP & Global Head of Oncology Development. Under his leadership, Tagrisso® and Lynparza® were successfully developed and commercialized for patients. Prior to joining AstraZeneca, Dr. Yver held roles at Merck, Johnson & Johnson, Aventis and Rhone-Poulenc Rorer. Dr. Yver is Executive Vice President and Chairman of Development at Centessa Pharmaceuticals plc and currently serves as an Independent Director of the Board of Directors at Sanofi. “I am delighted to join the Spotlight Board and support the mission to unlock the full potential of gene editing and enable effective single administration therapeutics for patients,” said Dr. Yver. “Spotlight’s biologics-based delivery approach has the potential to forge a new generation of cell-targeted in vivo CRISPR gene editing medicines across multiple therapeutic areas.” “Antoine’s deep insights and vast global pharma experience will help propel Spotlight to becoming a clinical stage company,” said Craig Gordon, M.D., Spotlight board member, Founder, CEO, and CIO of GordonMD™ Global Investments. “We look forward to working together to impact the strategic decisions of the company that will ultimately provide benefit to patients.” About Spotlight Therapeutics Established in mid-2018, Spotlight Therapeutics is a privately held biotechnology company advancing a pipeline of cell-targeted in vivo CRISPR gene editing therapies. Spotlight's proprietary technology platform TAGE is a new class of biologics, CRISPR effectors engineered for direct delivery in vivo, to achieve cell-selective therapeutic genome editing. Spotlight's pipeline is advancing its modular programmable CRISPR effectors towards clinical studies in immuno-oncology, ophthalmic diseases and hemoglobinopathies.

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ApiJect Announces Investment by Royalty Pharma and Jefferies

ApiJect Systems Corp. | May 19, 2022

ApiJect Holdings, Inc., the parent company of ApiJect Systems, Corp., a medical technology public-benefit corporation transforming the fill and finish and delivery of injectable vaccines and medicines, announced today that it had completed a $111 million private round of investment. This investment round values ApiJect at approximately $300 million. The new round was led by Royalty Pharma and Jefferies Financial Group (NYSE: JEF), which acquired a revenue interest in addition to equity. Jefferies and ApiJect's other lenders also converted all of their debt into ApiJect equity, such that following this transaction, ApiJect has no debt on its balance sheet. The funds will be used for continued development of ApiJect's proprietary Blow-Fill-Seal (BFS) plastic prefilled injection system, to deploy its equipment and proprietary technologies to licensees, and for general working capital purposes. Royalty Pharma and Jefferies are represented on ApiJect's Board of Directors, along with ApiJect co-founders Jay Walker and Marc Koska, and Hanjin In, the CEO of Tae-Chang Industrial Co., Ltd., ApiJect's South Korean cannula supplier. ApiJect is developing an innovative portfolio of drug delivery devices using high-speed BFS manufacturing, which is recognized by the FDA as an advanced aseptic process that forms, fills and seals a single unit dose in a continuous, automated manufacturing step. BFS technology greatly reduces the risk of contamination and error during one of the most critical steps of drug production. BFS is cost-efficient for short and long runs, and highly suitable to scale quickly in order to meet unexpected spikes in demand such as for a rapid response to population-scale health emergencies. Advancements developed by ApiJect for temperature management now enable BFS manufacturing of a vast array of sterile injectable drugs, including ultra-cold mRNA vaccines. "This is an important day for injection device innovation, and, with that, the future health of billions of people throughout the world. The investment provided by Royalty Pharma and Jefferies supports further development of an injection technology that can scale rapidly and efficiently to meet global demand for injectable medicines." ApiJect Chief Executive Officer Jay Walker Mr. Walker further said: "We welcome Royalty Pharma's commitment to the ApiJect mission. Royalty Pharma has a proven track record in selecting successful partners and in assisting in advancing its partners' goals. We could not ask for a better strategic partner from within the pharma industry. In Jefferies, we have a partner that has been with us from virtually the beginning of ApiJect." "We are delighted to form this win-win partnership with ApiJect," said Pablo Legorreta, Royalty Pharma's Founder and Chief Executive Officer. "The global pandemic highlighted the clear unmet need for a new kind of prefilled syringe that can be rapidly produced to respond to public health threats, and at massive scale that would be capable of delivering hundreds of millions, potentially billions, of doses to people in the U.S. as well as globally. ApiJect is developing a truly innovative technology, that could transform the manufacturing and delivery of life-saving injectable drugs in a way that would also make these medicines safer and more readily accessible for patients around the world." "We are proud of our association with ApiJect and believe strongly that their solutions will be a positive gamechanger in many of the most critical aspects of improving health globally," said Rich Handler, CEO of Jefferies. The first potential device made on the ApiJect Platform is the Prefilled ApiJect Injector, a single-dose prefilled injector designed to efficiently deliver a 0.5mL dose into a patient with a simple squeeze of the BFS container by the healthcare professional. This new type of prefilled injector will be ApiJect's first product submitted for regulatory review and approval. The Prefilled ApiJect Injector and its manufacturing process include An aseptically filled single-dose container, designed to enable efficient and intuitive injection administration. A pen-needle-style hub that turns the BFS container into an injection device. Innovations in temperature management to expand the range of medicines and vaccines suitable for BFS packaging. ApiJect recently launched its Technology Development Center in the greater Orlando area to bring to the U.S. critical capabilities for device design and engineering, and BFS mold development, as well as for small-scale manufacturing of single-dose, prefilled injectors and other injectable devices. Its purpose is to dramatically reduce the time and processes necessary for product development. ApiJect's Technology Development Center supports the company's existing fill-finish lines at its manufacturing partner site in South Carolina, which currently has the capacity to produce up to 540 million single-dose prefilled injectors annually. About ApiJect Systems ApiJect Systems, Corp., is a public-benefit medical technology company working to use our platform to bring more prefilled, single-dose injections to patients everywhere. The ApiJect Platform enables pharmaceutical and biotech companies to design scalable prefilled injectors and efficiently fill-finish them with their injectable drug products. This can be done either on one of their own Blow-Fill-Seal packaging lines with ApiJect-licensed technology, or at one of ApiJect's world-class manufacturing partners.

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BUSINESS INSIGHTS

Foundation Medicine Announces Strategic Collaboration with Arvinas

Foundation Medicine | June 06, 2022

Foundation Medicine, Inc., a pioneer in molecular profiling for cancer, announced a collaboration with Arvinas, Inc., to develop FoundationOne®Liquid CDx as a companion diagnostic for use with Arvinas’ bavdegalutamide an investigational novel PROTAC® protein degrader targeting the androgen receptor (AR). Arvinas’ bavdegalutamide is being developed for the potential treatment of men with metastatic castration resistant prostate cancer who have progressed on existing therapies. Arvinas is a clinical-stage biotechnology company and a pioneer in the rapidly growing field of targeted protein degradation. Arvinas’ proprietary PROTAC® targeted protein degraders, or proteolysis-targeting chimeras, work by harnessing the body’s natural protein disposal system to selectively and efficiently degrade and remove disease-causing proteins. AR activity is a key driver of prostate cancer, which makes the ability to regulate AR signaling an important factor in controlling disease progression. “We look forward to collaborating with Foundation Medicine to develop a companion diagnostic aimed at improving patient access. Foundation Medicine’s deep understanding of cancer genomics, scalable solutions, and regulatory expertise makes them an ideal partner for us as we develop bavdegalutamide as a potential new therapy for men with prostate cancer.” Ron Peck, M.D., chief medical officer at Arvinas Foundation Medicine’s portfolio of FDA-approved comprehensive genomic profiling tests offer physicians both blood- and tissue-based testing options for detecting genomic alterations that help guide personalized treatment decisions. As companion diagnostics, FoundationOne®CDx and FoundationOne®Liquid CDx allow oncologists to identify patients who may be appropriate for FDA-approved targeted therapies. “We are proud to serve as an end-to-end partner for Arvinas as they pioneer this new approach to treat cancer,” said Sanket Agrawal, chief biopharma business officer, Foundation Medicine. “Bringing our capabilities to this emerging area of biotechnology sets us on an exciting path to deepen our collective understanding of cancer biology and deliver more novel treatment options to patients now and in the future.” Foundation Medicine is an essential partner for biopharma organizations navigating the complexity of cancer care and research. This latest collaboration adds to its more than 65 global biopharma and biotechnology partnerships aimed at getting targeted cancer treatments to patients faster. About FoundationOne®CDx FoundationOne CDx is a next-generation sequencing based in vitro diagnostic device for detection of substitutions, insertion and deletion alterations (indels), and copy number alterations (CNAs) in 324 genes and select gene rearrangements, as well as genomic signatures including microsatellite instability (MSI) and tumor mutational burden (TMB) using DNA isolated from formalin-fixed, paraffin-embedded (FFPE) tumor tissue specimens. FoundationOne CDx is for prescription use only and is intended as a companion diagnostic to identify patients who may benefit from treatment with certain targeted therapies in accordance with their approved therapeutic product labeling. Additionally, FoundationOne CDx is intended to provide tumor mutation profiling to be used by qualified health care professionals in accordance with professional guidelines in oncology for patients with solid malignant neoplasms. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Some patients may require a biopsy. About FoundationOne®Liquid CDx FoundationOne Liquid CDx is a qualitative next generation sequencing based in vitro diagnostic test for prescription use only that uses targeted high throughput hybridization-based capture technology to analyze 324 genes utilizing circulating cell-free DNA (cfDNA) isolated from plasma derived from anti-coagulated peripheral whole blood of advanced cancer patients. The test is FDA-approved to report short variants in over 300 genes and is a companion diagnostic to identify patients who may benefit from treatment with specific therapies (listed in Table 1 of the Intended Use) in accordance with the approved therapeutic product labeling. Additional genomic findings may be reported and are not prescriptive or conclusive for labeled use of any specific therapeutic product. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Patients who are negative for companion diagnostic mutations should be reflexed to tumor tissue testing and genomic alteration status confirmed using an FDA-approved tumor tissue test, if feasible. About Foundation Medicine Foundation Medicine is a pioneer in molecular profiling for cancer, working to shape the future of clinical care and research. We collaborate with a broad range of partners across the cancer community and strive to set the standard for quality, scientific excellence, and regulatory leadership. Our deep understanding of cancer biology helps physicians make informed treatment decisions for their patients and empowers researchers to develop new medicines. Every day, we are driven to help our partners find answers and take action, enabling more people around the world to benefit from precision cancer care.

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Spotlight

The collection and analysis of data generated outside of restricted clinical trials are now critical to pharma companies. Data gathered in the routine delivery of healthcare can be mined to uncover insights into treatment efficacies and outcomes in the real world. Such insights, known as real-world evidence (RWE), benefits teams working across the drug lifecycle by showing how larger, heterogeneous patient populations interact with the healthcare system and respond to medicines.

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