Almac Discovery Ltd.

DUBLIN, March 27, 2023 /PRNewswire/ -- The "Lipid Nanoparticles in Drug Delivery: Intellectual Property Landscape" report has been added to ResearchAndMarkets.com's offering. This report features an extensive study of some of the key historical and contemporary intellectual property (IP) documents (featuring granted patents, patent applications and other documents), describing the various applications of lipid nanoparticles in drug delivery. The insights generated in this report have been presented across two deliverables, namely a MS Excel workbook and a MS PowerPoint deck, summarizing the ongoing activity in this domain. Key inclusions are briefly described below: Low drug solubility and its corresponding impact on bioavailability have remained a primary cause of concern with several marketed as well as clinical stage drugs. According to the Journal of Analytical and Pharmaceutical Research, 70% of the 50 drugs approved by the USFDA in 2021 were poorly soluble. Moreover, approximately 40% of marketed pharmacological products and close to 90% of drug candidates under development, have been associated with solubility and / or permeability issues, resulting in poor bioavailability. It is worth noting that, every year, a large number of drugs fail to reach the market due to poor bioavailability and issues associated with aqueous solubility. As a result, the industry is actively seeking various tools / methods to mitigate this challenge. Amongst the various approaches for bioavailability enhancement of therapeutic interventions, lipid nanoparticles (LNPs) and other lipidic excipients have gained sufficient attention from drug developers owing to their ability to act as drug carriers for complex, albeit highly promising therapeutics. Many studies have indicated that lipid-based formulations are capable of solving the complex drug delivery challenges currently faced by this industry. This can be attributed to the versatility of such molecules in terms of structure and functionality, as well as their ability to enhance the solubility and bioavailability of poorly water-soluble (hydrophobic) drugs. It is worth highlighting that, currently, lipid nanoparticles / lipids are the most commonly used drug delivery systems, specifically for mRNA therapeutics and vaccines. In fact, LNPs have been used in several mRNA-based COVID-19 vaccines to encapsulate and deliver genetic material to the target cells. As a result, the demand for lipid nanoparticles as drug delivery systems has grown considerably over the years. Presently, several studies are underway to evaluate the potential of next-generation lipid nanoparticles as effective drug delivery systems, aiming to enable the development of therapies with high efficacy and bioavailability. Given the extensive research activity, the intellectual capital related to the use of LNPs in drug delivery has also grown over time. Therefore, it is important to keep track of both pockets of innovation and key areas of improvement for stakeholders to remain competitive in this upcoming field of the healthcare domain. This report captures some of the key R&D trends and provides competitive intelligence on intellectual property related to lipid nanoparticles and their applications in drug delivery. Overall Intellectual Property Landscape An analytical perspective of the various patents and affiliated IP documents that have been published related to lipid nanoparticles in drug delivery, since 1977. An in-depth analysis of published IP documents, representing unique patent families across various global jurisdictions, featuring insightful inferences related to both historical and recent R&D trends within this niche, yet rapidly evolving segment of the biotechnology and pharmaceutical industry. Popular / Relevant Prior Art Search Expressions An examination of IP literature, shortlisting key words and phrases used to describe lipid nanoparticles in drug delivery. The analysis also includes details on the historical use of the aforementioned terms across different IP filings, key affiliated terms (which can be used to identify other relevant IP search terms and establish relationships between prior art search expressions), and other related trends. Patent Valuation Analysis A competitive benchmarking and valuation analysis of the key members of unique patent families captured in the report, taking into consideration important parameters, such as type of IP document, year of application, time to expiry, number of citations and jurisdiction (factoring in value associated with the gross domestic product (GDP) of a particular region). Patentability and Freedom to Operate A systematic approach to identify relevant areas of innovation by analyzing published IP documents (representative of unique patent families), by defining the uniqueness of patented / patent pending innovations, in order to assess the scope of patentability in this domain, and pinpoint jurisdictions wherein new and / or modified claims may be filed without infringing on existing IP. Analysis of Patent Applications A detailed summary of the various patent applications (representative of unique patent families) that were filed across different jurisdictions and their relative value in the IP ecosystem. The analysis classified the intellectual capital in terms of type of innovation and the innovation (such as a product class, enabling technologies or method of use), thereby, offering the means to identify active arenas of research and assess innovation-specific IP filing trends. Analysis of Granted Patents An analysis of the granted patents (representative of unique patent families) across different global jurisdictions and their relative value in the IP ecosystem. The analysis also features a meaningful classification system, segregating granted IP into relevant categories (namely type of innovation and innovation) to help develop a detailed perspective on the diversity of intellectual capital (having marketing exclusivity) related to lipid nanoparticles in drug delivery, and the assessing likelihood for innovators to enter into promising research areas. Pockets of Innovation and White Spaces An insightful analysis of the various CPC codes used in published IP literature (representative of unique patent families) and their affiliated families, offering the means to identify historical and existing pockets of innovation (based on the functional area / industry described by the elaborate and systematic IP classification approach, mentioned earlier); the analysis also features a discussion on prevalent white spaces (based on type of innovation and innovation) in this field of research. Claim Analysis One of the objectives of the report was to analyze and summarize key inferences from the independent claims mentioned in granted, active patents (representative of unique patent families) in the dataset. Using a systematic segregation approach, we have analyzed trends associated with the preamble, type of patent (product patent or method patent), type of claim (open ended claim or closed ended claim) and key elements of a claim (individual aspects of an innovation that are covered in a singular claim). Key Topics Covered: Excel Deliverable 1. Research Notes 2. Summary Dashboard A. Overall Intellectual Property Landscape B. Intellectual Property Landscape (Grouped by Simple Families) C. Key Prior Art Search Expressions D. Key Trends related to Patent Applications (featuring Patentability and Freedom to-Operate) E. Key Trends related to Granted Patents (featuring Patentability and Freedom-to-Operate) F. Claims Analysis G. Key CPC Symbols H. Key Applicants 3. Overall Intellectual Property Landscape Dataset 4. Prior Art Search Expressions (Keyword Analysis) 5. Patent Applications Dataset 6. Granted Patents Dataset 7. Claims Analysis 8. Key Applicants Analysis 9. CPC Analysis 10. Appendix I: Pivot Tables 11. Appendix II: Country / Geography Codes 12. Appendix III: Innovation Categories PowerPoint Deliverable 1. Context 2. Project Approach 3. Project Objectives 4. Executive Summary 5. Lipid Nanoparticles in Drug Delivery 5.1. Overview 5.2. Introduction to Lipid Nanoparticles 5.3. Advantages and Applications of Lipid Nanoparticles in Pharmaceutical Industry 5.4. Future Perspectives 6. Overall Intellectual Property Landscape 6.1. Overview 6.2. Analysis of Simple Patent Families 6.3. Key Innovation Categories 6.4. Insights from Patent Applications 6.5. Insights from Granted Patents 7. Key Prior Art Search Expressions 7.1. Overview 7.2. Analysis of Prior Art Search Expressions 8. Intellectual Property Valuation Analysis 8.1. Valuation Overview 8.2. Analysis of Individual Value Ranks 8.2.1. Rank 1 IP Documents 8.2.2. Rank 2 IP Documents 8.2.3. Rank 3 IP Documents 8.2.4. Rank 4 IP Documents 8.2.5. Rank 5 IP Documents 8.3. Concluding Remarks 8.4. List of Rank 1 IP Documents 9. Analysis of Patent Applications 9.1. Overview 9.2. Relative Valuation of Patent Applications 9.3. Patentability and Freedom-to-Operate 10. Analysis of Granted Patents 10.1. Overview 10.2. Relative Valuation of Grated Patents 10.3. Patentability and Freedom-to-Operate 10.4. Analysis of Patent Claims (Granted Active Patents) 11. Key Applicants 11.1. Overview 11.2. Analysis of Key Applicants 12. Pockets of Innovation and White Spaces 12.1. Overview 12.2. Pockets of Innovation 12.3. White Spaces 12.4. Concluding Remarks 13. Future Outlook 13.1. Lipid Nanoparticles in Drug Delivery: IP Filing Growth Trends 13.2. Relevance of IP for Business Development and Growth 13.3. Contemporary Sentiments and Predictions 13.4 Anticipated Future Developments and Trends 14. Appendices Companies Mentioned Aarhus University 3M Innovative Properties Abbott Abcombi Biosciences ABIONYX Pharma AC BioScience AC Immune Academia Sinica Access Pharma ACM Biolabs ACT Therapeutics Acuitas Therapeutics Adjuvantix Aduro BioTech Advanced BioScience Laboratories (ABL) Advanced Cardiovascular Systems Advanced Inhalation Research Advaxis Aegis Therapeutics AfGin Pharma Agency for Science, Technology and Research (A*STAR) AGENE Research Institute Agenus Agilent Technologies AgoneX Biopharmaceuticals AgResearch AgroSource Akagera Medicines Albany Medical College Albert Einstein College of Medicine Albumedix Alcon Alcyone Therapeutics Aleph Therapeutics Alexion Pharmaceuticals Alloplex Biotherapeutics Almac Discovery Alnylam Pharmaceuticals Alpharma ALSATECH Altea Therapeutics Altus Pharmaceuticals ALZA Corporation Alzeca Biosciences Amorepacific Corporation Amur Pharmaceuticals Ancora Pharmaceuticals Angarus Therapeutics Angioslide And Many More Companies! For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets

Focused on developing treatments for rare diseases associated with vascular malformations Recruited first patients in Proof-of-Concept study for lead drug candidate VAD044 Financed by Medicxi in 2020 BASEL, Switzerland, Aug. 22, 2022 /PRNewswire/ -- Vaderis Therapeutics AG (Vaderis), a clinical stage biotechnology company focused on developing treatments for rare diseases associated with vascular malformations, today announced its emergence from stealth and initiation of its INSIGHT proof-of-concept clinical trial in patients suffering from Hereditary Haemorrhagic Telangiectasia (HHT), also known as Osler-Weber-Rendu Syndrome. Vaderis was established in 2019 and in 2020 raised over CHF18 million from Medicxi, enabling progression of its lead asset into the clinic and successful completion of Phase 1a. Emergence from Stealth Vaderis was founded with the unique aim to develop therapeutics for rare and orphan diseases associated with vascular malformations. In April 2020 Vaderis acquired a portfolio of allosteric AKT inhibitors from Almac Discovery Ltd of Belfast, UK. AKT is a serine kinase which plays a crucial role in vascular growth and in vascular overgrowth. There is a significant number of rare diseases such as HHT in which upstream genetic mutations trigger over-activation of the serine kinase AKT, resulting in vascular overgrowth. Such diseases frequently manifest in ways that are debilitating, disfiguring, painful and sometimes life-threatening. The vast majority of these diseases are currently left untreated except for symptomatic interventions which tend to offer patients limited temporary relief of individual symptoms. VAD044 is a once daily, orally administered, allosteric AKT inhibitor which has the potential to treat the underlying cause of these diseases. If successful, Vaderis would be the first company in the world to develop a medicine for the treatment of HHT and other diseases associated with vascular malformations. Initiation of Clinical PoC in HHT In pursuit of the company's core objective, Vaderis announces the initiation of the first study of VAD044 in HHT patients. Known as the INSIGHT proof-of-concept trial (INternational Study InvestiGating HHT), the study is unique in its robust design aimed at achieving a thorough understanding of the safety of VAD044 in HHT patients. A total of 80 HHT patients across the USA, Canada and Europe will be randomised in a double-blind, controlled trial comparing two doses of VAD044 to placebo. Initiation of the INSIGHT trial follows Health Authority approvals including FDA, Health Canada and key European agencies. J.J. Mager, MD, PhD, pulmonologist at St. Antonius Hospital Utrecht, Chairman of the Global Research and Medical Advisory Board of Cure HHT and lead investigator of the INSIGHT trial commented, "The first patients recruited into the INSIGHT trial mark an exciting milestone in HHT clinical research. If successful, this trial may demonstrate the potential of this new drug to be effective beyond the symptomatic treatments we currently offer HHT patients, by addressing the course of this rare, progressive, and debilitating disease." Nicholas Benedict, CEO of Vaderis added, "Working together with the HHT community, both caregivers and patient associations, Vaderis hopes to be the catalyst which transforms patient care in HHT. Success in the INSIGHT trial would be a major step towards developing the first ever registered treatment for HHT anywhere in the world." Giovanni Mariggi, Partner at Medicxi and Chairman of Vaderis said, "Our vision is to create a unique company dedicated to delivering breakthrough therapies for rare diseases caused by vascular malformations. Initiation of the INSIGHT trial is an important milestone on the way to realising this vision. It is testament to the significant progress made by the company over the last two years". About Vaderis Vaderis is a clinical stage biotech company with the aim to develop medicinal treatments for rare and orphan diseases associated with vascular malformations. Since its founding in 2019, Vaderis raised a Series A financing from Medicxi and acquired a portfolio of allosteric AKT inhibitors from Almac Discovery Ltd of Belfast, UK. There is a significant number of debilitating and largely untreated rare diseases, such as HHT (Hereditary Haemorrhagic Telangiectasia), in which patients suffer from over-activation of AKT triggered by upstream genetic mutations resulting in vascular overgrowth. There are no drugs approved anywhere in the world which specifically treat HHT. Vaderis aims to be the first company to develop a medicine for the treatment of HHT and aims to treat other diseases associated with vascular malformations. About Medicxi Medicxi is a healthcare-focused investment firm with the mission to create and invest in companies across the full drug development continuum. Leveraging deep expertise in drug development and company creation spanning over two decades, Medicxi invests in early and late-stage therapeutics with a product vision that can fulfill a clear unmet medical need. For more information, please visit: Logo - SOURCE Vaderis Therapeutics AG

In 2017, two Novartis clinicians — Pierre Saint-Mezard and Damien Picard — approached Nicholas Benedict, who’d left Novartis in 2007 and went on to head a series of biotechs, with an idea: use Akt inhibitors, typically used to treat cancers, for rare diseases that cause blood vessel malformations. The three of them founded Vaderis Therapeutics in 2019 out of Basel, Switzerland, with CHF 18 million (just under $19 million USD) in backing from Medicxi Ventures, and are launching from stealth today as they take their idea, now in drug form, into the clinic for an 80-patient proof-of-concept study. Having started their company right at the cusp of the pandemic, Benedict recalls his last business trip before lockdown was to London, to secure funding for Vaderis from Medicxi. They finalized the deal in April, making Vaderis Medicxi’s “first pandemic investment,” Medicxi partner Giovanni Mariggi said. Akt sits at the center of a number of important cell functions, including survival, growth and death. In cancer, Akt inhibitors put a block in that pathway, stopping tumor cells from multiplying and causing them instead to die. The Vaderis trio wants to apply that concept to rare blood vessel diseases, starting out with hereditary hemorrhagic telangiectasia, or HHT for short. Patients with HHT have blood vessels that grow abnormally, sometimes without the necessary capillaries, leading to excessive bleeding episodes and anemia, and current treatments focus mainly on treating the symptoms associated with the disease. With an Akt inhibitor, Vaderis hopes it can address abnormal blood vessel overgrowth associated with the disease. However, when looking for an Akt inhibitor for HHT, Benedict said that they didn’t want to repurpose a cancer drug. “What we found fairly early in was that many, if not most, of the Akt inhibitors that are available or are being studied right now are not suitable for repurposing, and they’re not suitable for non-oncological indications, largely because of the toxicities that they hold,” Benedict said. That led them to Almac Discovery, from which they acquired a portfolio of new Akt inhibitors. Mariggi echoed the safety point. He noted other points on the pathway that Akt is part of that could be blocked, but added that those treatments come with serious side effects. “When you’re an HHT patient that hopefully has a relatively long life ahead of you, they’re just unacceptable,” Mariggi said, referencing the side effects. But with an Akt inhibitor, “we felt that it was going to have a much better tolerability and therefore be more suited to chronic use than using a PI3 kinase inhibitor or an mTOR inhibitor,” Mariggi said. Alongside its launch from stealth, Vaderis will begin a clinical trial to test the safety of its drug, dubbed VAD044, in 80 patients across the US, Canada, and Europe. Benedict says the biotech has enough funding to see it through the clinical trial, which is set to read out in 2024, according to clinicaltrials.gov.