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

Zogenix, Inc | April 09, 2019

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

What do you see as Pharma's biggest opportunity for improving patient safety over the next 5-10 years. Biopharmaceuticals are among the most sophisticated and elegant achievements of modern science. The huge, complex structures of these drugs don’t just look extraordinary in the 3-D modeling systems used to design them; they also perform their jobs remarkably well, offering high efficacy and few side effects.


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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

Stallergenes Greer Strengthens Precision Medicine Approach in Allergen Immunotherapy With Imperial College London Research Collaboration

Stallergenes Greer | January 17, 2022

Stallergenes Greer, a global healthcare company specialising in allergen immunotherapy (AIT), today announced that the company has entered into a research collaboration on the discovery of biomarkers of AIT efficacy with Imperial College London, a global top ten university with a world-class reputation in science, engineering, business and medicine. This long-term research collaboration, which combines the long-standing expertise of Stallergenes Greer in AIT with the prestigious research capabilities of Imperial College London, aims to push the boundaries of allergic patient care by identifying biomarkers of AIT efficacy and expanding the knowledge of the pathophysiology of allergic diseases and their treatment with AIT. “We are thrilled about this collaboration with Imperial College London which will contribute to both deciphering the mode of action of AIT and identifying key markers that can be used in the routine practice of allergology for the benefit of patients and healthcare practitioners. Based on the understanding of specific phenotypes and the responses of patients with allergies, allergologists will be able to tailor treatment modalities with the right dosing at the right time for each individual. This underscores our commitment to advancing precision medicine for allergy sufferers”, Amer Jaber, Executive VP Operations Europe and International and President of Stallergenes SAS “This research collaboration is a remarkable opportunity for a fruitful partnership between Imperial College London, a pioneer and world-leading university in the biomarker field, and Stallergenes Greer. It will build on the recent advances of biomarker research to further develop precision medicine in AIT for the benefit of patients with allergic diseases,” said Dr Mohamed Shamji, Head of the Immunomodulation and Tolerance Group, Imperial College London. The results of this research collaboration will be shared in international congresses and peer review publications. About Allergen Immunotherapy Allergies are the most prevalent and fastest growing chronic diseases in the industrialised world, affecting over one billion people worldwide. Allergen immunotherapy uniquely alters the natural course of respiratory allergies. It is the only therapeutic class capable of modifying disease progression and potentially preventing the onset of the disease by inducing tolerance in the immune system. About Stallergenes Greer Headquartered in London (UK), Stallergenes Greer Ltd is a global healthcare company specialising in the diagnosis and treatment of allergies through the development and commercialisation of allergen immunotherapy products and services. Stallergenes Greer Ltd is the parent company of Greer Laboratories, Inc. About Imperial College London Imperial College London is one of the world's leading universities. The College's 20,000 students and 8,000 staff are working to solve the biggest challenges in science, medicine, engineering, and business. Imperial is University of the Year 2022 in the Times and Sunday Times Good University Guide. It is the world’s fifth most international university, according to Times Higher Education, with academic ties to more than 150 countries. Reuters named the College as the UK's most innovative university because of its exceptional entrepreneurial culture and ties to industry.

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PHARMACY MARKET

KromaTiD Announces Launch of KromaTiD Pinpoint FISH TP53/CEP17 Kit for Synthetic Oligonucleotide-Based FISH Assay

KromaTiD Inc | January 14, 2022

KromaTiD is excited to announce early access to our KromaTiD Pinpoint FISH TP53/CEP17 Kit, our new product designed to supply the highest resolution, lowest background, and lowest limit of detection available. KromaTiD's Pinpoint FISH™ Kit, allows for better hybridization conditions and eliminating strand degradation steps. Pinpoint FISH™ allows researchers to expand beyond the capabilities of conventional FISH probes to detect smaller targets and design ultra-high specificity tests. Our technology is fully compatible with your standard FISH equipment/conditions and BAC probes and works with your established samples, workflow, and imaging system. "Single-cell genomic measurements from KromaTiD have unique capabilities to analyze and understand the underlying genomic drivers of human disease. Pinpoint FISH has an unmatched resolution, extraordinary ease of use, consistency from analysis to analysis, and an unprecedented ability to distinguish fine genomic structural variation" Dr. Tompkins continues, "While this is the first catalog product from KromaTiD, we have a ten-year history of building a custom assay for Pharma and Biotech companies based on the PPF and dGH platforms. As we begin to build out a comprehensive line of blood cancer measurement kits, we are delighted to bring Pinpoint FISH to a worldwide market". Pinpoint FISH™ is available now from KromaTiD for researchers' use in oligonucleotide-based FISH assays. Christopher Tompkins, KromaTiD's Chief Technology Officer About KromaTiD, Inc. KromaTiD is transforming the fields of genome engineering through the discovery and characterization of genomic structural changes that help leading gene editing and pharmaceutical companies advance therapies to market. KromaTiD offers a powerful suite of products and services for studying genomic rearrangements, custom assay development services and preclinical research support. KromaTiD's proprietary Pinpoint FISH™ and directional Genomic Hybridization platforms (dGHTM) have applications throughout genomics, supplying direct, definitive data on structural variations that no other technologies can provide. We provide the essential genomic structural context to sequence.

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BUSINESS INSIGHTS

Pack4U acquires leading pharmacy technology company Catalyst Healthcare

Pack4U | January 07, 2022

Pack4U, a connected health services company, acquires long-time technology partner Catalyst Healthcare whose patented AdhereNet® platform links pharmacists, patients, and care teams to effectively manage drug complexity and improve medication adherence. "We are dedicated to improving patient outcomes at scale. Bringing this domain knowledge in-house is vital for Pack4U. Catalyst's CEO Shane Bishop is a pharmacy thought leader and we are excited about building the next phase of Pack4U with the Catalyst team on board. Catalyst's proprietary and patented platform strengthens our ability to meet the needs of patients, providers and health plans by offering a full, end-to-end, connected care solution seamlessly integrated with our vast clinical team across all states." Pack4U President, Rahul Chopra Pack4U provides in-home and virtual services that connect patients with pharmacists for oversight, medication delivery, remote monitoring, and care coordination. Our complete suite of connected health IP fortifies Pack4U's ability to personalize care, improve health outcomes, and lower total cost of care across North America. "I am extremely excited to see these organizations come together and to join the team that is transforming in-home healthcare," said Shane Bishop. "Pack4U's extensive and diverse team of pharmacists combined with Catalyst's adherence solutions, create opportunity for a new patient monitoring paradigm. With central fills for scale and community pharmacy adoption, we will reach more people to change more lives." "This collaboration follows our aggressive growth in 2021. It expands our vision of in-home care via a full suite of managed services featuring patient specific, in-home medicine dispensing, always-on patient engagement, electronic medicine administration record and supply chain software for centralized filling," said Rahul Chopra. About Pack4U Pack4U Inc is a tech-enabled health services company. We connect people with community pharmacists to manage drug complexity and maximize health benefits through personalized medication delivery, virtual monitoring, and proactive care. Our fully automated, high-volume central fill pharmacy hubs, proprietary health platform and medication adherence network power a robust, scalable solution to chronic condition management that leads to better patient outcomes and total cost of care reduction.

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Spotlight

What do you see as Pharma's biggest opportunity for improving patient safety over the next 5-10 years. Biopharmaceuticals are among the most sophisticated and elegant achievements of modern science. The huge, complex structures of these drugs don’t just look extraordinary in the 3-D modeling systems used to design them; they also perform their jobs remarkably well, offering high efficacy and few side effects.

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