FDA Rejects Regulatory Filing For Zogenix Epilepsy Drug; GW Pharma Moves In Sympathy

Zogenix, Inc | April 09, 2019

Zogenix, Inc.,which develops therapies for rare diseases, said Monday afternoon the FDA issued a refusal to file letter regarding its NDA for Fintepla, chemically fenfluramine hydrochloride, which is being evaluated for the treatment of seizures associated with Dravet syndrome. The FDA determined the NDA wasn't sufficiently complete to permit a substantive review, primarily due to two reasons: non-submission of certain non-clinical studies for assessing chronic administration of the pipeline asset and an incorrect version of a clinical dataset, which prevented the completion of the review process required to support the filing. Fintepla, previously codenamed ZX008, is Zogenix's lead asset and was touted to be a multi-billion-dollar opportunity for the company. The adverse development suggests a serious setback to the small-cap biotech. Bank of America analyst Tazeen Ahmad said last September ZX008's worldwide, risk-adjusted peak sales is estimated to be $1.5 billion in 2029, comprising a $604-million opportunity in Dravet syndrome, $503 million in Lennox-Gastaut syndrome and $386 million in other pediatric epilepsy conditions.

Spotlight

It is well established that the use of nanotechnology in medicine and more specifically drug delivery is spreading rapidly. Driven by the diminishing rate of discovery of new biologically active compounds that can be exploited therapeutically to treat disease and with fewer new drugs entering the market every year, interest in the use of nanoparticle’s versatile and multifunctional structures for the delivery of existing drugs has grown rapidly. Nanoparticles offer better pharmacokinetic properties, controlled and sustained release, and targeting of specific cells, tissues or organs such (e.g. in new ways in which to cross the blood-brain barrier). All these features can improve the efficacy of existing drugs (Malam et al., 2011). Nanoparticles in this context have been defined as colloidal systems of submicron size that can be constructed from a large variety of materials in a large variety of compositions. Commonly defined nanoparticle vectors include: liposomes, micelles, dendrimers, solid lipid nanoparticles, metallic nanoparticles, semiconductor nanoparticles and polymeric nanoparticles. Therefore, nanoparticles have been extensively employed to deliver drugs, genes, vaccines and diagnostics into specific cells/tissues (Ram et al., 2011).


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PHARMACY MARKET

Harbour BioMed Announces IND Approval for Next-Gen Anti-TSLP Fully Human Monoclonal Antibody

Harbour BioMed | February 22, 2022

Harbour BioMed announced that China National Medical Products Administration had approved its investigational new drug (IND) application for HBM9378 a next-gen fully human antibody targeting thymic stromal lymphopoietin for the treatment of moderate-to-severe asthma. HBM9378/SKB378 is a co-development project conducted by HBM and Kelun-Biotech, who together equally share the global rights in respect of HBM9378. The IND approval of HBM9378 is an achievement of the strategic collaboration between the parties. According to the strategic collaboration and license agreement between HBM and Kelun-Biotech, the parties will jointly explore innovative therapies of monoclonal antibodies and antibody-drug conjugate. Building on HBM's innovative business model leveraging its productive R&D platform to lead the next generation of valuable and innovative therapies and its mission to address the unmet medical needs, HBM will continue to expand its collaboration with industry leading partners and further leverage on the combined capabilities of HBM and its collaborators. "HBM9378/SKB378, a next-gen fully human monoclonal antibody, is developed from HBM's H2L2 platform. Its long half-life optimization and outstanding biophysical properties support the favorable dosing advantage. The approval of this IND application once again proves HBM's powerful innovation capability. Unleashing the power of our unique R&D engine, we are highly confident and determined to defeat intractable diseases across the world." Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed About HBM9378/SKB378 HBM9378/SKB378 is a fully human monoclonal antibody against TSLP generated from two heavy chains and two light chains (H2L2) platform. It inhibits the TSLP mediated signaling pathway by blocking the interaction between TSLP and TSLP receptor. TSLP plays important roles in DC cell maturation, T helper 2 (Th2) cell polarization and inflammation, particularly in both eosinophilic and non-eosinophilic inflammation asthma. About Asthma Asthma is a heterogeneous disease defined by the history of respiratory symptoms that vary over time and in intensity, together with variable expiratory airflow limitation. The disease is estimated to affect more than 300 million people worldwide and affect more than 45.7 million adult people (≥20 years) in China. Approximately 5-10% of those afflicted with asthma have severe disease that is poorly controlled. Despite the use of medium to high dose inhaled corticosteroids (ICS) in combination with a long-acting β2-agonist (LABA), currently available biologic therapies and oral corticosteroids (OCS), many severe asthma remain uncontrolled. Current biologics are mainly targeting Type 2 severe asthma which manifests clinically with a combination of peripheral eosinophilia, sputum eosinophilia and/or elevated fractional exhaled nitric oxide (FENO). Severe asthma patients experience frequent exacerbations, significant limitations on lung function and a reduced quality of life. The healthcare burden associated with care for these patients is also high. Therefore, there are still significant unmet needs to develop safer and broader therapies for severe asthma. About Harbour BioMed Harbour BioMed is a global biopharmaceutical company committed to the discovery, development and commercialization of novel antibody therapeutics focusing on immunology and oncology. The Company is building its robust portfolio and differentiated pipeline through internal R&D capability, collaborations with co-discovery and co-development partners and select acquisitions.

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RESEARCH

DEBIOPHARM INVESTS IN VERISIM LIFE'S $15M SERIES A ROUND TO ADVANCE AI-ENABLED DRUG RESEARCH

Debiopharm International SA | January 11, 2022

Debiopharm a Swiss biopharmaceutical company, announced their co-investment in California-based start-up VeriSIM Life's $15 Million Series A Round to advance their mission to accelerate drug development via technology powered by artificial intelligence. VeriSIM Life's computational platform reduces time and cost of drug development as well as reduces the need for animal testing that, in the vast majority of cases, does not translate well to humans. Debiopharm's co-investment in VeriSIM contributes to the growth plan of the start-up and will help to expand and establish transformational partnerships with industry and academia using their first-in-class 'virtual drug development engine' BIOiSIM™. The investment aligns with Debiopharm's focus to invest in digital health solutions that improve the cancer patient journey, transform pharmaceutical R&D, and shift healthcare towards a more patient-centric approach. With the financing round led by Morpheus Ventures, Debiopharm Innovation Fund joins new investors including Colorcon Ventures along with existing investors OCA Ventures, Intel Capital, Serra Ventures and Susa Ventures. Founded in 2017 by Jo Varshney, DVM and PhD alongside a cross-functional team of pharmaceutical scientists, software engineers and AI/ML and simulation experts, the start-up offers a range of translational-based solutions, customized for pre-clinical and clinical programs. "We're moving into a time where AI-based technology will play a critical role in drug development. We absolutely need to reduce the time, costs & risks of drug development in order to be more efficient – that's exactly what the BIOiSIM™ is proven to do. The value of using this AI-based platform is the potential decrease in the need for animal testing and the acceleration of the pre-clinical, translational stage, helping drug research companies to more quickly select which early-stage medicines will most likely benefit patients and cure their disease." Tanja Dowe, CEO of the Debiopharm Innovation Fund More than 90% of drugs tested in animals fail to pass human clinical trials, resulting in delayed development and high costs. VeriSIM's BIOiSIM platform, driven by AI and machine learning (ML), de-risks R&D decisions by providing meaningful insights much earlier in the drug development process with unprecedented accuracy and scalability. VeriSIM's platform solves this decade-old translatability problem within the drug development phase and ensures clinical success of drugs intended for highly unmet needs. With its first-in-class platform, VeriSIM is transforming the way pharmaceutical and biotech companies address the most challenging diseases impacting humankind. About VeriSIM Life VeriSIM Life has developed a sophisticated computational platform that leverages advanced AI and ML techniques to significantly improve drug discovery and development by greatly reducing the time and money it takes to bring a drug to market. BIOiSIM is a first-in-class 'virtual drug development engine' that offers unprecedented value for the drug development industry by narrowing down the number of drug compounds that offer anticipated value for the treatment or cure of specific illnesses or diseases. The program not only reduces the time and cost of drug discovery and development, it also greatly reduces the need for animal testing that, in the vast majority of cases, does not translate to humans. Debiopharm's commitment to patients Debiopharm develops, manufactures and invests in innovative therapies and technologies that respond to high unmet medical needs in oncology and bacterial infections. We aim to provide strategic funding and guidance for companies with Smart Data & Digital Health solutions with the ambition to change the way drugs are developed and the way patients are treated. Our growing portfolio company achievements includes 18 FDA clearances or CE marks and 2 IPOs. Since 2018 Debiopharm has invested over USD 120 million, typically leading the investment rounds of its 16 portfolio companies.

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BUSINESS INSIGHTS

Cedars-Sinai Medical Center and AIkido Pharma Inc. to Codevelop Novel Immuno-Oncology Cancer Treatment

AIkido Pharma Inc. | March 15, 2022

AIkido Pharma Inc. announced that the Company and Cedars-Sinai Medical Center have agreed under their Master Collaboration Agreement to co-fund and codevelop a novel immuno-oncology treatment designed to promote the destruction of cancer cells by a patient's own "killer" T-cells. The Company and Cedars-Sinai will share costs, expenses and management of the drug co-development and will leverage the talent of Cedars-Sinai Technology Ventures and the lead scientists who originated the novel treatment. The parties will also share in the proceeds of any commercialization of the treatment. The compounds to be codeveloped target the cellular pathway that causes CD8+ T-cells to enter an "exhausted state," rendering them ineffective against cancer cells. The new treatment targets and blocks recently discovered transcription factors essential for T-cells to enter the "exhausted state," the goal of which is to strengthen the ability of a patient's T-cells to retain their capacity to destroy cancer cells. "Our agreement with Cedars-Sinai to collaborate in the funding and monetization of novel disease treatments is a major step for the Company. I am thrilled that Cedars-Sinai will be co-funding this project and providing its exceptional staff of scientists to advance this new cancer treatment, which I believe speaks volumes about its potential." Anthony Hayes, CEO of AIkido About AIkido Pharma Inc. AIkido Pharma Inc. was initially formed in 1967 and is a biotechnology Company with a diverse portfolio of small-molecule anticancer and antiviral therapeutics. The Company's platform consists of patented technology from leading universities and researchers, and we are currently in the process of developing an innovative therapeutic drug platform through strong partnerships with world renowned educational institutions, including The University of Texas at Austin and University of Maryland at Baltimore. Our diverse pipeline of therapeutics includes therapies for pancreatic cancer, prostate cancer. We are constantly seeking to grow our pipeline to treat unmet medical needs in oncology. The Company is also developing a broad-spectrum antiviral platform that may potentially inhibit replication of multiple viruses including Influenza virus, SARS-CoV MERS-CoV, Ebolavirus and Marburg virus.

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BUSINESS INSIGHTS

TIKOMED and IQVIA announce a strategic collaboration for the development of TIKOMED's lead drug platform candidate ILB®

Tikomed | May 09, 2022

Swedish biopharmaceutical company TikoMed AB and IQVIA announce a key strategic collaboration with the aim to accelerate the development of TikoMed's lead drug platform candidate ILB®, a broad-spectrum small molecule drug candidate with curative potential in a wide range of diseases driven by acute and chronic inflammation. The collaboration will support TikoMed's clinical development, regulatory and commercial strategies. IQVIA brings together deep therapeutic expertise, unparalleled data, and technology alongside flexible operating models to enable biopharma organisations such as TikoMed to accelerate the clinical development and commercialisation of innovative products that improve patient lives. "We are thrilled to have IQVIA involved supporting TikoMed with the next steps in our journey to improve human life by enhancing the body's natural ability to self-repair and regenerate. With our highly scalable proprietary drug platform we aim to provide safe, effective and affordable therapies to as many patients as possible across the globe", CEO Anders Kristensson Fiona Stewart, Head of Strategic Alliance Management Europe from IQVIA said "We are delighted TikoMed has selected IQVIA to strategically collaborate in the development of ILB®. We look forward to leveraging our depth of experience to help accelerate the availability of these medicines to the patients who need them the most." TikoMed was advised by The Connecting Architects on this strategic collaboration. TikoMed AB TikoMed is committed to improve human life by exploring and harnessing the medical potential of the body's ability to self-repair and regenerate. With an adaptive, multi modal mechanism of action, TikoMed's drug platform rebalances the body's inflammatory, immune and fibrotic responses to acute and chronic inflammation in order to enhance self-repair and regeneration. Currently applied as a therapy in neurodegeneration and an enabling technology for advanced therapies, the initial development programs include ALS, TBI and islet cell transplantation. Broader use of the drug platform will be considered for a wider range of diseases using a creative business and financing model. TikoMed's highly scalable proprietary technology aims to provide safe, qualitative and affordable medicine to as many patients as possible across the globe. TikoMed is privately-owned and based in Viken, Sweden. IQVIA IQVIA is a leading global provider of advanced analytics, technology solutions, and clinical research services to the life sciences industry. IQVIA creates intelligent connections across all aspects of healthcare through its analytics, transformative technology, big data resources and extensive domain expertise. IQVIA Connected Intelligence™ delivers powerful insights with speed and agility - enabling customers to accelerate the clinical development and commercialization of innovative medical treatments that improve healthcare outcomes for patients. With approximately 79,000 employees, IQVIA conducts operations in more than 100 countries.

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Spotlight

It is well established that the use of nanotechnology in medicine and more specifically drug delivery is spreading rapidly. Driven by the diminishing rate of discovery of new biologically active compounds that can be exploited therapeutically to treat disease and with fewer new drugs entering the market every year, interest in the use of nanoparticle’s versatile and multifunctional structures for the delivery of existing drugs has grown rapidly. Nanoparticles offer better pharmacokinetic properties, controlled and sustained release, and targeting of specific cells, tissues or organs such (e.g. in new ways in which to cross the blood-brain barrier). All these features can improve the efficacy of existing drugs (Malam et al., 2011). Nanoparticles in this context have been defined as colloidal systems of submicron size that can be constructed from a large variety of materials in a large variety of compositions. Commonly defined nanoparticle vectors include: liposomes, micelles, dendrimers, solid lipid nanoparticles, metallic nanoparticles, semiconductor nanoparticles and polymeric nanoparticles. Therefore, nanoparticles have been extensively employed to deliver drugs, genes, vaccines and diagnostics into specific cells/tissues (Ram et al., 2011).

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