Alexions Soliris gets new, and lucrative, rare disease indication.

pharmaphorum | June 28, 2019

Alexion’s rare disease drug Soliris has a new indication after the FDA approved it for a disease that attacks the optic nerves.Approval of Soliris (eculizumab) for  neuromyelitis optica spectrum disorder (NMOSD) in adults who have the anti-aquaporin-4 antibody opens up further revenues for Alexion, which has already built a blockbuster franchise around the drug. Although only approved in a handful of rare diseases, the complement-inhibiting drug generated $962 million for Alexion in Q1 alone, up from $800 million in the previous year’s Q1. Although the disease only affects between 4,000-8,000 patients in the US, at the prices Alexion charges this could generate significant revenues. Dosage varies according to a patient’s weight but it’s estimated to cost around $400,000 per patient annually. First approved in 2007 for paroxysmal nocturnal haemoglobinuria, Soliris is threatened by the possibility of biosimilar competition as its US patent expires in March 2021 and it goes off patent in Europe in May next year. Amgen is developing a biosimilar that could undercut Soliris on price if approved and marketed, and Alexion is hoping to maximise Soliris’ revenues in its last years of its patent life, as well as developing a longer-lasting alternative, Ultomiris. NMOSD is an autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal cord.

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Albert Liou, Vice Chairman of Asia Pacific PAREXEL International, goes over the latest trends in drug development in the Asia Pacific region. Learn the trends to watch, including regulatory harmonization.


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BUSINESS INSIGHTS

Iktos and The University of Dundee, Drug Discovery Unit (DDU) Announce a Collaboration to Use AI for Drug Design and Retrosyntheis

Iktos | December 20, 2021

Iktos, a company specialized in Artificial Intelligence for new drug design, and The Drug Discovery Unit (DDU), School of Life Sciences, The University of Dundee, focused on the discovery of therapeutics for neglected diseases and the translation of novel biology through small molecule drug discovery, announced entering a collaboration to apply Iktos’s generative modelling artificial intelligence (AI) technology in one of DDU’s drug discovery programs. Under the agreement, Iktos will apply its de novo ligand and structure-based generative modelling technologies, its AI-based retrosynthesis analysis and planning tool Spaya™, and know-how complementing DDU’s drug discovery capabilities to expedite the identification of potential pre-clinical candidates and to identify additional novel chemical matter with suitable properties. The DDU was established in 2006 to respond to a lack of drug targets identified for neglected tropical diseases being translated into therapeutics for diseases impacting the poorest people in the world. The DDU translates basic science into lead compounds to validate putative drug targets, to use as tools to investigate disease pathways and, when appropriate, advance to pre-clinical drug candidates for multiple diseases, e.g. TB and cancer. Iktos’s AI technology, based on deep generative models, helps to bring new insights and directions into the drug discovery process based on a comprehensive data-driven chemical structure generation technology. This technology automatically designs virtual novel molecules with all of the characteristics of a successful drug molecule. This approach, validated through Iktos’s other collaborations, is a novel solution to one of the key challenges in drug design: rapid identification of molecules that simultaneously satisfy multiple parameters, such as potency, selectivity, safety, and project-specific properties. This approach uniquely enables the exploration of chemical space and produces innovative molecule designs with greater freedom to operate. Iktos has recently diversified its R&D efforts into the development of an AI technology for retrosynthesis. Identifying and selecting synthetic pathways is one of the most challenging and time-consuming tasks in synthetic and medicinal chemistry. Iktos has developed AI based retrosynthesis analysis and planning tool Spaya™ by harnessing the power of data-driven retrosynthesis algorithm for systematic exploration and prioritisation of synthetic routes for a desired compound in minutes. “We are thrilled to be working with Iktos to drive our projects towards drug candidates. Iktos AI platform combined with our own in-house computational and medicinal chemistry design teams provide a powerful combination for innovative new drug design.” Professor Paul Wyatt, Head of the DDU “We are thrilled and proud to join forces with DDU with the aim to help in the discovery of therapeutics for neglected diseases and the translation of novel biology through small molecule drug discovery,” commented Yann Gaston-Mathé, President and CEO of Iktos. “Pleased to have earned DDU’s trust, we are confident that together with DDU and their established R&D partners, we will be able to identify promising novel chemical matter and solve complex multiparametric optimisation problems. The feedback from DDU’s research team will be highly valuable as we improve our product offerings. Our strategy has always been to tackle challenging problems alongside our collaborators where we can demonstrate value generation for new and on-going drug discovery projects.” About DDU The Drug Discovery Unit (DDU) (est. 2006) within the School of Life Sciences at The University of Dundee is a fully integrated, Biotech-style, drug discovery centre working across multiple disease areas. The DDU collaborates with partners to translate world-class biology research into novel drug targets and candidate drugs to address unmet medical need. About Iktos Incorporated in October 2016, Iktos is a French start-up company specialized in the development of artificial intelligence solutions applied to chemical research, more specifically medicinal chemistry and new drug design. Iktos is developing a proprietary and innovative solution based on deep learning generative models, which enables, using existing data, to design molecules that are optimized in silico to meet all the success criteria of a small molecule discovery project. The use of Iktos technology enables major productivity gains in upstream pharmaceutical R&D. Iktos offers its technology both as professional services and as a SaaS software platform, Makya™. Iktos is also developing Spaya™, a synthesis planning software based upon Iktos’s proprietary AI technology for retrosynthesis.

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VIEWS AND ANALYSIS

CHMP Recommends Approval of Dupixent® (dupilumab) for Children Aged 6 to 11 Years with Severe Asthma with Type 2 Inflammation

Regeneron Pharmaceuticals, Inc | January 31, 2022

Regeneron Pharmaceuticals, Inc. and Sanofi today announced that the European Medicines Agency's Committee for Medicinal Products for Human Use has adopted a positive opinion, recommending to extend the approval of Dupixent® (dupilumab) in the European Union (EU) to include add-on maintenance treatment for children aged 6 to 11 years with severe asthma with type 2 inflammation characterized by raised blood eosinophils and/or raised fractional exhaled nitric oxide (FeNO) who are inadequately controlled on two maintenance therapies. The European Commission is expected to announce a final decision on the Dupixent application in the coming months. The CHMP positive opinion is supported by Phase 3 data recently published in the New England Journal of Medicine showing that Dupixent added to standard of care significantly reduced the rate of severe asthma attacks and rapidly improved lung function within two weeks, with sustained improvement up to 52 weeks, in children with uncontrolled moderate-to-severe asthma. The safety results from the trial were generally consistent with the known safety profile of Dupixent in patients aged 12 years and older with uncontrolled moderate-to-severe asthma. Adverse events more commonly observed with Dupixent compared to placebo included injection site reactions, viral upper respiratory tract infections and eosinophilia. Helminth infections were also more commonly observed with Dupixent compared to placebo in patients aged 6 to 11 years. Asthma is one of the most common chronic diseases in children. Up to 85% of children with asthma may have type 2 inflammation and are more likely to have higher disease burden. Despite treatment with current standard-of-care inhaled corticosteroids (ICS) and bronchodilators, these children may continue to experience serious symptoms such as coughing, wheezing and difficulty breathing. Severe asthma may impact children's developing airways and cause potentially life-threatening exacerbations. Children with severe asthma also may require the use of multiple courses of systemic corticosteroids that carry significant risks. Uncontrolled severe asthma can interfere with day-to-day activities like sleeping, attending school and playing sports. On October 20, 2021, Dupixent was approved by the FDA as an add-on maintenance treatment for patients aged 6 to 11 years with moderate-to-severe asthma characterized by an eosinophilic phenotype or with oral corticosteroid-dependent asthma. The use of Dupixent in children aged 6 to 11 years with uncontrolled severe asthma is investigational in the EU and is not yet approved. About Dupixent Dupixent, which was invented using Regeneron's proprietary VelocImmune® technology, is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant. IL-4 and IL-13 are key and central drivers of the type 2 inflammation that plays a major role in atopic dermatitis, asthma and chronic rhinosinusitis with nasal polyposis (CRSwNP). About Regeneron Regeneron is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to nine FDA-approved treatments and numerous product candidates in development, almost all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, pain, hematologic conditions, infectious diseases and rare diseases. Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite technologies, such as VelocImmune, which uses unique genetically humanized mice to produce optimized fully human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world. About Sanofi Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions. With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.

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PHARMACY MARKET

Ultivue Announces Co-Marketing Agreement with Aignostics for Unique Spatial AI-Powered Imaging Solutions for Translational Medicine

Ultivue, Inc., | March 10, 2022

Ultivue, Inc., an industry leader in multiplexing tools and novel image analysis solutions for tissue biomarker studies, and Aignostics GmbH, a global leader in Artificial Intelligence (AI) powered precision diagnostics focused on pathology to assist with research, clinical trials and CDx development, announced a partnership to collaborate on the co-development of AI-powered spatial multiplexed immunophenotype solutions for translational research groups and Biopharma. The companies have already been working together since early 2021 to adapt Aignostics’ client platform to support visualization and analysis for some of Ultivue’s products and services, as well as provisioning of results and corresponding reports. The resulting co-developed platform will now be made available to joint clients. Additionally, proof of concept work conducted on the Ultivue 12-plex Immunophenotyping biomarker panel in over 1,000 clinical NSCLC cases has demonstrated encouraging results for the capacity of AI-based analysis of multiplexed images for more consistent and reproducible readouts at scale. These results will be published later this year and respective AI models made available via the platform. The companies are now expanding their relationship under a co-marketing agreement to offer the multiplex immunofluorescence (mIF) platform to their respective biotech and pharma clients, initially for research use, with a GCP-compliant version to follow later this year. Moreover, Ultivue and Aignostics will offer AI-powered image analysis services within the platform via Aignostics “Explainable AI” approach for the consistent and precise analysis of mIF cohorts at scale, for both translational research and clinical trial research services in Ultivue’s CLIA accredited clinical research laboratory. “We are excited to extend our workflow offering to include mIF AI for highly automated image analysis by partnering with Aignostics,” says Florian Leiss, VP Digital Health at Ultivue. “Improved viewing capabilities enable us to interactively explore the interplay of mIF-labeled cell populations in the histological context of same-slide H&E.” “We believe that development of Ultivue’s assays with our AI-powered image analysis will create insights from data at scale that are unprecedented in spatial biology today. We are excited to start offering this approach to our clients for translational science and clinical trials,” Viktor Matyas, CEO of Aignostics Besides offering research and development services to its clients, Ultivue and Aignostics will also develop products for standardized analysis of certain Ultivue products, paving the way for more widespread use of these technologies in both clinical trials and clinical routine. About Ultivue Ultivue provides researchers and scientists in translational medicine with multiplex biomarker assays for tissue phenotyping and digital pathology. Its proprietary InSituPlex® technology enables advanced exploration and interrogation of tissue samples for precision medicine research. These highly customizable solutions coupled with our scientific consultative approach strengthen and accelerate biomarker discovery and drug development programs. About Aignostics Aignostics, a Berlin based company with offices in the US, is a digital pathology company dedicated to novel precision diagnostics through the use of a proprietary “Explainable AI” platform in pre-clinical and translational research and clinical trials. The use of such technology is expected to be crucial to implementation of AI-powered algorithms in routine diagnostics in order to verify (explain) the model’s decision outputs. Additionally, through its close alignment to Charité and TU Berlin amongst other collaborators, only Aignostics offers the combination of data access, sample testing and pathology expertise to develop digital pathology algorithms spanning multi-modal biomarker assay detection systems such as H&E, IHC and mIF, amongst others.

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BUSINESS INSIGHTS

Virpax Pharmaceuticals Enters Into CRADA With The U.S. Army Institute of Surgical Research

Virpax® Pharmaceuticals, Inc. | May 06, 2022

Virpax® Pharmaceuticals, Inc. a company specializing in developing non-addictive products for pain management, post-traumatic stress disorder, central nervous system (CNS) disorders and anti-viral indications, today announced that it has entered into a cooperative research and development agreement with the U.S. Army Institute of Surgical Research to evaluate Virpax’s Probudur™, an injectable long-acting liposomal bupivacaine in a hydrogel formulation that is injected at the wound site. Probudur is being developed to significantly reduce or eliminate the need for opioids after surgery in approved indications. Probudur is a local anesthetic that binds to the sodium channel, preventing pain signals from reaching the brain. In pre-clinical trials, Probudur has shown long duration pain control for at least 96 hours. The USAISR is the U.S. Department of Defense’s (DOD) primary laboratory for developing solutions for trauma and critical care challenges in combat casualties. “We are excited to announce this CRADA with the DOD as these military and government research collaborations are an integral part of our non-dilutive funding strategy,” Anthony P. Mack, chairman and CEO of Virpax In August of 2020, Virpax entered into a CRADA with the National Center for Advancing Translational Sciences, an institute of the National Institutes of Health (NIH), for the development of Envelta™, it’s intranasal spray product candidate for severe post-cancer pain and non-cancer pain. Under that agreement, multiple contracts to support the research, development, and manufacturing of Envelta have been awarded by the NIH to continue the product’s progress. About Virpax Pharmaceuticals Virpax is developing branded product candidates for non-addictive pain management and neurological disorders using its proprietary technologies to optimize and target drug delivery. Virpax is initially seeking FDA approval of its three patented drug delivery platforms. Epoladerm™ is a topical diclofenac spray film formulation being developed to manage osteoarthritis pain. Probudur™ is a single injection long-acting liposomal bupivacaine formulation being developed to manage post-operative pain. Envelta™ is an intranasal Molecular-Envelope Technology (MET) enkephalin formulation being developed for the management of post-cancer pain and non-cancer pain, as well as post-traumatic stress disorder (PTSD) under the name PES200. MET technology is also used in AnQlar™, Virpax’s product candidate to inhibit viral replication caused by influenza or SARS-CoV-2. Virpax recently acquired global rights to VRP324, a product candidate for the nasal delivery of a pharmaceutical-grade cannabidiol (CBD) for the management of rare pediatric epilepsy.

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Spotlight

Albert Liou, Vice Chairman of Asia Pacific PAREXEL International, goes over the latest trends in drug development in the Asia Pacific region. Learn the trends to watch, including regulatory harmonization.

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