EMA announces medicine approval recommendations from November meeting

European Pharmaceutical Review | November 18, 2019

The European Medicines Agency’s (EMA) human medicines committee (CHMP) has announced the results of its November 2019 meeting, recommending seven medicines for approval. The committee suggested granting a marketing authorisation for Isturisa (osilodrostat), a treatment for Cushing’s syndrome, a disorder that occurs when the body produces too much corticosteroid hormone. Mayzent (siponimod), for the treatment of adult patients with secondary progressive multiple sclerosis and Sunosi (solriamfetol), for the treatment of excessive daytime sleepiness in patients with narcolepsy and obstructive sleep apnoea, both received a positive opinion from the committee. Tavlesse (fostamatinib) also received a positive opinion from the CHMP to treat primary immune thrombocytopenia, an acquired immune-mediated disorder characterised by the destruction of platelets and impaired platelet production. A conditional marketing authorisation was recommended for Polivy (polatuzumab vedotin) indicated for relapsed/refractory diffuse large B-cell lymphoma.

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Annogen Announces Research Project with Pfizer to Test Non-coding Genetic Sequences

Annogen | December 14, 2022

Annogen, the Amsterdam based biotech company behind the SuRE™ technology for the functional annotation of the non-coding part of the genome, today announced it has begun a research project with Pfizer to functionally test tens of thousands of disease-related non-coding sequence variants for their effect on gene regulation. The results may contribute to drug discovery by identifying functionally relevant non-coding variants that play a central role in diseases. More than 95 percent of disease- and trait-related variants are found in the non-coding genome. However, identifying the important causal variants amongst the thousands of non-functional ones is a major challenge, as non-coding variant functionality cannot be deduced from sequence alone. Using its SuRE™ methodology, Annogen can obtain a functional read-out for up to millions of non-coding variants in parallel. “We are very proud of this research project with Pfizer. We believe the non-coding part of the genome represents a huge opportunity for drug discovery that has been largely overlooked. We are now working with several top-tier biopharma companies on a broad array of projects, which demonstrates the interest in the SuRE™ platform and the AIM™ service for gene & cell therapy, drug discovery, and recombinant protein production”. Joris van Arensbergen, Annogen’s founder and CEO, is pleased to establish another project in this field About Annogen At Annogen we use our SuRE™ technology to identify regulatory DNA elements to be used for controlled gene expression valuable for gene & cell therapy, as well as for recombinant protein production. In addition, we offer the AIM™ service to identify favorable gene insertions and their expression levels for more than 100,000 integrations in parallel. These approaches enable researchers to qualitatively interpret the non-coding genome in humans, animals and plants.

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PHARMACY MARKET

FogPharma Announces $178 Million Series D Financing to Advance Pipeline of First-in-Class Helicon Polypeptide Therapeutics

FogPharma | November 28, 2022

FogPharma®, a biopharmaceutical company pioneering a new class of precision medicines that could ultimately prove applicable to the vast majority of therapeutic targets, including those previously considered “undruggable,” announced a $178 Million Series D financing. The financing round includes new investors ARCH Venture Partners, Milky Way Investments and Fidelity Management & Research Company and existing investors VenBio Partners, Deerfield Management, GV, Cormorant Asset Management, funds and accounts advised by T. Rowe Price Associates, Inc., Invus, Farallon Capital Management, HBM Healthcare Investments, Casdin Capital, and PagsGroup, also participated. Proceeds from the Series D financing will be used to advance and accelerate FogPharma’s growing pipeline of hyperstabilized α-helical (Helicon™) polypeptide therapeutics, a proprietary new class of drugs designed to overcome the limitations of today’s precision medicines with broad applicability to the vast majority of disease targets and therapeutic areas. FogPharma’s lead Helicon polypeptide development candidate, FOG-001, a first-and-only-in-class direct TCF-blocking β-catenin inhibitor with potential applicability to significant cancer patient populations, is expected to enter clinical development in mid-2023. In addition, FogPharma is advancing other first-in-class programs against important, biologically validated cancer targets that have remained elusive to other approaches including TEAD, NRAS, Pan-KRAS, ERG and Cyclin E1. “FogPharma continues to make rapid progress on our moonshot mission to achieve universal druggability – a world where no targets are off-limits to medicine. We believe that Helicon polypeptides, a compelling new therapeutic modality, represent the future of precision medicine. We are thrilled by the support of our investors and will continue to build our platform capabilities, product pipeline which aims to address a significant percentage of cancer patient populations, and our phenomenal team across all levels as we aim to create one of the most impactful new classes of drugs in history.” Gregory Verdine, Ph.D., founder, chairman and chief executive officer of FogPharma In connection with the Series D Financing, Rick Klausner, M.D., has been appointed to FogPharma’s board of directors. In addition, Dr. Verdine has been appointed as chairman of the board. “The team at FogPharma is building an unprecedented new therapeutic modality and robust pipeline with the potential to make a meaningful difference in the lives of cancer patients,” said Dr. Klausner. “I am excited to join the board of directors and be part of something special particularly at this important time as FogPharma continues to impressively scale its science, team, operations and infrastructure, with the goal of advancing its first Helicon polypeptide therapeutic into the clinic.” Dr. Klausner is currently the founder and chief scientist of Altos Labs and founder and chairman of Lyell Immunopharma. Dr. Klausner was founder and director of Juno Therapeutics and founder and director of GRAIL. He is also the chairman of Sonoma Biotherapeutics and co-founder and chairman of Lifemine Therapeutics. Previously, Dr. Klausner served as senior vice president, chief medical officer and chief opportunity officer of Illumina Corporation and as executive director for global health for the Bill and Melinda Gates Foundation. Dr. Klausner was appointed by Presidents Clinton and Bush as the eleventh director of the U.S. National Cancer Institute between 1995 and 2001. Dr. Klausner served as chief of the Cell Biology and Metabolism Branch of the National Institute of Child Health and Human Development as well as a past president of the American Society of Clinical Investigation. He has served in senior advisory roles to the U.S., Norwegian, Qatari and Indian governments. About FogPharma’s Universal Druggability Platform and Helicon™ Polypeptide Therapeutics Existing drug classes are limited in both reach and applicability, with more than 80% of known human protein disease targets considered “undruggable” because they are beyond the reach of both antibodies and small molecules. FogPharma’s Helicon peptide drug discovery engine integrates directed evolution, proprietary α-helix conformational hyperstabilization chemistry, highly multiplexed drug optimization technology, artificial intelligence including deep learning and machine learning, structure-based drug discovery, cancer genomics and biology, and multiscale manufacturing to rapidly discover Helicon polypeptide therapeutics. This novel therapeutic modality combines the targeting strength and specificity of antibodies with the broad tissue distribution, intracellular target engagement and oral dosing optionality of small molecules to address the limitations of today’s precision medicines and reach the most difficult targets – achieving universal druggability. About FOG-001 FogPharma’s lead Helicon polypeptide development candidate, FOG-001, a first-and-only-in-class direct TCF-blocking β-catenin inhibitor. Dysregulation of the Wnt/β-catenin signaling pathway has been shown to occur in at least 20% of all human cancers. In the U.S. alone, FOG-001 has the potential to become a new treatment option for >1 million patients suffering from a broad range of intractable cancers. In biochemical and cellular studies, FOG-001 has been shown to potently, precisely and selectively disrupt the interaction of β-catenin with its obligate downstream transcription factor, TCF. Preclinical studies have demonstrated the ability of FOG-001 to cause tumor growth inhibition and regression by disrupting β-catenin-dependent signaling. FOG-001 is the inaugural member of FogPharma’s TCF-Catenix family of direct-acting β-catenin antagonists and combines key features that distinguish it from previously reported Wnt/β-catenin pathway modulators: FOG-001 acts inside the cell, where it directly binds the key oncogenic driver β-catenin; and FOG-001 blocks TCF-β-catenin engagement at the most downstream node in the canonical Wnt pathway, thus abrogating the signal transmission mechanism by which most, if not all, known Wnt pathway mutations are believed to drive oncogenesis. About FogPharma FogPharma is a biopharmaceutical company pioneering the discovery and development of Helicon™ polypeptides. Through this novel therapeutic modality, FogPharma aims to address the limitations of existing drug classes and achieve universal druggability – a world where no targets are off-limits to new medicines. Spun out of Harvard University by pioneering academic scientist and successful biotech company builder Dr. Gregory Verdine, FogPharma is advancing a broad pipeline of Helicon polypeptide therapeutics against important and biologically validated cancer targets that have remained elusive to other approaches, with the goal of providing new targeted treatment options for significant cancer patient populations. FogPharma is headquartered in Cambridge, Mass., and has raised more than $360 million to date from leading life sciences investors.

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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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PHARMACY MARKET, PHARMA TECH

MTTI Obtains FDA Allowance of Investigational New Drug (IND) for Hürthle Cell Thyroid Cancer

Molecular Targeting Technologies, Inc | December 09, 2022

Molecular Targeting Technologies, Inc. a clinical stage radiopharmaceutical therapy company focused on therapies for rare diseases, announced today the allowance of an Investigational New Drug application by the U.S. Food and Drug Administration. It is now waiting for Institutional Review Board approval. Once approved by IRB, it will enable a Phase I/II clinical study of the Safety, Dosimetry and Efficacy of EBTATE in adult patients with metastatic, radioactive iodine non-responsive Hürthle cell thyroid cancer.” EBTATE is a new generation of peptide receptor radiotherapeutic drug that has demonstrated potential clinical superiority over standard of care. It selectively binds to somatostatin receptor 2 on neuroendocrine and other tumors, which are then killed by the radionuclide. EvaThera platform products were designed to bind to serum albumin, due to the Evans blue moiety, extending in vivo residence time, enabling lower, less frequent dosing of the radiopharmaceutical and reducing risk of renal injury vs. the current standard of care. Dr. Joanna Klubo-Gwiezdzinska, MD, Ph.D, MHSc, acting section chief of Thyroid Tumors and Functional Thyroid Disorders in the National Institute of Diabetes and Digestive and Kidney Diseases, part of the National Institutes of Health (NIH) and study author commented: “Hürthle cell thyroid cancer, which currently lacks effective treatment options, is characterized by a particularly high expression of SSTR2*. With this phase I/II clinical trial, we hope to implement individualized dosimetry-based dosing for each patient with Hürthle cell thyroid cancer that spread outside of the thyroid gland and analyze how much of an active agent is being accumulated in the tumor tissue to establish a threshold associated with the best efficacy and safety.” “The clearance of our IND is an important milestone for MTTI. Having solidified our clinical trial preparedness and manufacturing readiness, we are well-positioned to advance EBTATE to target Hürthle cell thyroid cancer.” Dr. Chris Pak, President & CEO of MTTI About Molecular Targeting Technologies, Inc. (MTTI) Molecular Targeting Technologies, Inc., is a privately held, rapidly growing, well financed, clinical-stage biotech company developing next-generation targeted radiotherapeutics and diagnostics for rare cancers. Evathera platform technology product has shown application for glioblastoma multiforme (GBM) cancer patients overexpressing integrin. MTTI is committed to building value by acquiring and translating innovative imaging, radiopharmaceutical and theranostics assets to improve human health, reduce healthcare costs and reward stakeholders. MTTI expects to be orchestrating multiple clinical trials in 2023. The content in this release is the sole responsibility of the authors and does not necessarily represent the official views or imply endorsement of the National Institutes of Health.

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Randomized, double-blind clinical trials are the gold standard for adequate and well-controlled studies in modern
times. However, prior to the late 1940s, randomization and blinding were not used in medicine and as result,
bias was common.

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