Fragmin approved by FDA as first anticoagulant for children

pharmatimes | May 17, 2019

The US Food and Drug Administration has approved Pfizer’s Fragmin (dalteparin sodium) injection to reduce the recurrence of symptomatic venous thromboembolism (VTE) in paediatric patients one month of age and older. The drug was initially approved by the FDA in 1994 for adults and is a type of heparin, which works as an anticoagulant. The FDA also granted this application Priority Review designation. The efficacy of Fragmin in children was based on a single trial with 38 paediatric patients with symptomatic deep vein thrombosis and pulmonary embolism who were were treated with Fragmin for up to three months, with starting doses by age and weight. At study completion, 21 patients achieved resolution of the qualifying VTE, seven patients showed regression, two patients showed no change, no patients experienced progression of the VTE and one patient experienced recurrence of VTE.

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BUSINESS INSIGHTS

BioMarin Announces Advancements in FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) for Adults with Severe Hemophilia A

BioMarin Pharmaceutical Inc. | November 24, 2022

BioMarin Pharmaceutical Inc. announced advancements in the U.S. Food and Drug Administration review of the Biologics License Application of ROCTAVIAN™ for adults with severe hemophilia A. The Company was recently notified by the FDA that after further consideration, at this time, the Agency no longer plans to hold an advisory committee meeting to discuss the BLA for ROCTAVIAN that is currently under review. Previously, the FDA communicated to the Company that it did intend to hold an advisory committee meeting but did not specify a date. The Company also remains on track to host the scheduled FDA Pre-Licensure Inspection (PLI) of BioMarin's gene therapy manufacturing facility located in Novato, CA. "The review of a BLA is a dynamic process, and we appreciate FDA's ongoing engagement as we work toward delivering a potentially transformative treatment choice to those patients with severe hemophilia A. We look forward to further dialogue with the Agency as it reviews our application." Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin About valoctocogene roxaparvovec The FDA granted Regenerative Medicine Advanced Therapy designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received for valoctocogene roxaparvovec in 2017. In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also received orphan drug designation from the EMA and FDA for the treatment of severe hemophilia A. Orphan drug designation is reserved for medicines treating rare, life-threatening, or chronically debilitating diseases. The European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name ROCTAVIAN™ on August 24, 2022. Robust Clinical Program BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of severe hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is also conducting a Phase 3, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with severe hemophilia A. Also ongoing is a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Factor VIII inhibitors. About Hemophilia A People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate or mild disease show a much-reduced propensity to bleed. Individuals with severe hemophilia A are treated with a prophylactic regimen of intravenous Factor VIII infusions administered 2-3 times per week or a bispecific monoclonal antibody that mimics the activity of Factor VIII administered 1-4 times per month. Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life. Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A. About BioMarin BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening genetic diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases.

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BUSINESS INSIGHTS, PRACTICE MANAGEMENT

Telix Announces Executive Leadership Appointments

Telix Pharmaceuticals Limited | December 05, 2022

Telix Pharmaceuticals Limited announces several key executive leadership appointments and promotions, reflecting the increased commercial focus of the Company and ongoing succession planning. Richard Valeix promoted to Group Chief Commercial Officer Richard Valeix, who has been in the role of Chief Executive Officer of Telix's Europe Middle East and Africa operations since joining Telix in May 2021, has been appointed to the newly created role of Group Chief Commercial Officer. This key global leadership role will oversee commercialisation strategy and activities for Telix's late-stage pipeline assets including the global rollout of Illuccix and launch of TLX250-CDx, Telix's kidney cancer imaging agent for which the Company recently reported positive Phase III data. Richard is a highly experienced business leader, with marketing and sales and general management experience in large pharmaceutical companies, having previously held senior roles at Advanced Accelerator Applications a Novartis company, Ipsen and Roche. Raphael Ortiz has been promoted to Chief Executive Officer - EMEA. He has held the position of Chief Operating Officer - EMEA since joining in January 2022. Raphael has more than 20 years of pharmaceutical industry experience across finance, business development, marketing, sales and general management and has previously held senior roles at AAA, where he established radioligand therapy operations for the Asia Pacific region. Genevieve holds Honours degrees in Science and Law from Monash University and a Graduate Diploma of Applied Corporate Governance from the Governance Institute of Australia. Genevieve is a Solicitor of the Supreme Court of Victoria and a Fellow of the Governance Institute of Australia. She replaces Melanie Farris who has retired from the role of Group Company Secretary to take on a broader portfolio as Senior Vice President Global Governance, Risk and Compliance. Melanie has served as Group Company Secretary since March 2017, prior to the Company's listing on the ASX, and has over 18 years' experience in governance and corporate operations. "These leadership appointments are a result of ongoing succession planning to ensure Telix has an optimal mix of skills and experience as we prepare to enter a new phase with a portfolio of multiple commercial products. I congratulate Richard, Raphael and Melanie on their new roles and am pleased that we are able to identify and develop talent to support the Company as it continues to grow. I'm also pleased to welcome Genevieve Ryan as Group Company Secretary, who brings a new depth of experience working within ASX-200 companies." Dr. Christian Behrenbruch, Telix Group CEO and Managing Director About Telix Pharmaceuticals Limited Telix is a biopharmaceutical company focused on the development and commercialisation of diagnostic and therapeutic radiopharmaceuticals. Telix is headquartered in Melbourne, Australia with international operations in the United States, Europe and Japan. Telix is developing a portfolio of clinical-stage products that aims to address significant unmet medical need in oncology and rare diseases. Telix is listed on the Australian Securities Exchange.

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VIEWS AND ANALYSIS, PHARMA TECH

Anaqua and PatSnap Team Up to Deliver an End-to-End Pharma IP Management Solution

Anaqua and PatSnap | December 08, 2022

Anaqua, the leading provider of innovation and intellectual property management technology announced it is partnering with innovation intelligence platform PatSnap. The collaboration will deliver an enhanced IP management solution to provide Anaqua’s pharmaceutical industry clients with a more detailed and informed view of the market and competitive landscape for key use cases. The combined Anaqua AQX® Pharma and PatSnap Synapse solutions will help AQX Pharma clients further enhance competitiveness, keep up with the fast-paced requirements of the current market, eliminate duplicated work across functions, as well as identify and realize new areas of investment. PatSnap is a platform for innovation intelligence, utilizing AI technology – including machine learning, computer vision and Optical Character Recognition that facilitates access to an unrivaled breadth of data that is connected in a meaningful way. PatSnap’s Synapse solution will give AQX Pharma users access to millions of data points, sourced utilizing best-in-class algorithms and expert manual curation, providing a comprehensive 360-degree market view and adding strategic value to drug development processes and strategy. The new integration is in response to client requests to access more pharmaceutical market data within the AQX platform to improve internal team efficiency and streamline processes. The integration will support sharing deeper intelligence between clients and partners and simplify the route of administration through features such as docket personalization that will allow teams to focus on their most strategic initiatives. Specifically, the combined system will address the use case of conducting stage gate reviews and automatically kick off relevant workflows. “AQX Pharma helps clients manage the business aspects of pharmaceutical IP management, while Synapse powers the external aspects of drug discovery through external and competitor drug discovery processes. Together, we’re using our unique capabilities to enable best practices in Life Science lifecycle stage gate reviews. We’re pleased to offer a complete pharma innovation solution, which can be further complemented by leveraging Anaqua’s strategic integrations, such as its innovation management solution, ideaPoint.” Vincent Brault, SVP of Product & Innovation at Anaqua Ray Chohan, Co-founder and VP of Corporate Development at PatSnap, said, “PatSnap is thrilled to team up with Anaqua, who are true innovators in life sciences IP management. Synapse will empower users of AQX Pharma to comb through millions of data points to discover and commercialize new therapies.” About Anaqua Anaqua, Inc. is a premier provider of integrated intellectual property management technology solutions and services for corporations and law firms. Its IP management software solutions, AQX® and PATTSY WAVE®, both offer best practice workflows with big data analytics and tech-enabled services to create an intelligent environment designed to inform IP strategy, enable IP decision-making, and streamline IP operations, tailored to each segment’s need. Today, nearly half of the top 100 U.S. patent filers and global brands, as well as a growing number of law firms worldwide, use Anaqua’s solutions. Over one million IP executives, attorneys, paralegals, administrators, and innovators use the platform for their IP management needs. The company’s global operations are headquartered in Boston, with offices across the U.S., Europe, Australia, and Asia. About PatSnap PatSnap’s AI-powered innovation intelligence platform transforms billions of innovation data points into actionable intelligence for over 10,000 global customers in more than 50 countries. With an unrivaled breadth of data, including patents, drug information, technology news, market reports, scientific literature, and more, PatSnap breaks barriers to connect innovators with insights.

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PHARMACY MARKET

A novel azapeptide editing method provides potential for new drug development, Feinstein Institutes research shows

Feinstein Institutes | November 29, 2022

Peptides, short sections of proteins made up of chains of amino acids, are an important area of new drug development due to their potential for increased specificity and fewer side effects. Their main drawback is the rapid degradation by the body’s own enzymes, which has led to the need to develop longer-lasting peptide alternatives. Azapeptides are one type of peptide alternative that has shown great potential as therapeutics, exemplified by the HIV drug, Atazanavir. A new paper published in the journal Nature Communications by researchers at The Feinstein Institutes for Medical Research details proprietary reagents and methods that can selectively replace only those amino acids known to be targeted by enzymes with more stable versions to create azapeptides. The paper, authored by Yousef Al-Abed, PhD, co-director of the Institute of Bioelectronic Medicine at the Feinstein Institutes, describes the proprietary building blocks and method used to conduct systematic and robust peptide editing, replacing targeted amino acids with aza-amino acids to form more stable and efficient azapeptides. The azapeptides created could expedite the novel therapies to treat many diseases and conditions, including metabolic disease, influenza, pulmonary arterial hypertension, Crohn’s disease, arthritis, and irritable bowel diseases, among others. The paper, titled “Thiocarbazate building blocks enable the construction of azapeptides for rapid development of therapeutic candidates,” provides multiple examples of azapeptide construction, screening, and selection using a known peptide inhibitor of inflammation and bradykinin. “Peptides as drug candidates are easy to discover yet difficult to develop as final drugs because of their fast degradation in our body. A technology that circumvents these problems inherent to native peptides will change the future landscape of drug discovery. Our technology provides a platform of tools that enable minimal modification of any peptide at highly prone degradation sites, thereby increasing its life span in our body, allowing it more time to find its target and neutralize it.” Dr. Al-Abed Through peptide editing, going from peptide to azapeptide, using methods that Dr. Al-Abed and his team detail in the new paper, scientists can create potential therapies that have enhanced characteristics compared to natural peptides, like longer stabilities in a more efficient manner. Dr. Al-Abed and his team have precisely engineered thiocarbazates (a novel functional group) in the proprietary process to achieve an almost universal and more efficient means to selectively replace amino acids in a peptide chain. “Peptide drugs are a crucial resource for patients and the global pharmaceutical industry,” said Kevin J. Tracey, MD, president and CEO of the Feinstein Institutes and Karches Family Distinguished Chair in Medical Research. “Dr. Al-Abed and his team invented a fully automated chemical strategy using novel chemistry to make new peptide drugs which is an important new avenue for peptide drug discovery.” About the Feinstein Institutes The Feinstein Institutes for Medical Research is the research arm of Northwell Health, the largest health care provider and private employer in New York State. Home to 50 research labs, 3,000 clinical research studies and 5,000 researchers and staff, the Feinstein Institutes raises the standard of medical innovation through its five institutes of behavioral science, bioelectronic medicine, cancer, health innovations and outcomes, and molecular medicine. We make breakthroughs in genetics, oncology, brain research, mental health, autoimmunity, and are the global scientific leader in bioelectronic medicine – a new field of science that has the potential to revolutionize medicine.

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