CELL AND GENE THERAPY
BioAge Labs, Inc. | October 14, 2022
BioAge Labs, Inc. a privately held clinical-stage biotechnology company developing therapeutics that target the molecular causes of aging to extend healthy human lifespan, announced that it will present updates on its NLRP3 inhibitor program at two upcoming conferences in the United States, Neurodegeneration Targets and the Inflammasome Therapeutics Summit, and at the BIO-Europe 2022 conference in Leipzig, Germany. BioAge is developing its novel, proprietary class of NLRP3 inhibitors, which have distinct structural and biological properties and include molecules that penetrate the blood–brain barrier, for neurodegenerative and neurosensory disorders associated with aging.
“Our family of potent, structurally differentiated NLRP3 inhibitors demonstrates BioAge’s capability to discover novel compounds for promising targets emerging from our discovery platform. I am excited about the potential of these drugs to treat diseases driven by brain aging, and our team is looking forward to the opportunity to share recent data from this program at multiple important scientific gatherings.”
BioAge CEO and co-founder Kristen Fortney, PhD
At the inaugural Neurodegeneration Targets meeting hosted by Cambridge Healthtech Institute [link], Rusty Montgomery, PhD, Vice President, Biology, will deliver a talk titled “Identification of a Novel Class of Highly Potent, CNS-Penetrant NLRP3-Specific Inhibitors with Excellent Drug-Like Physical Features” in Boston, MA on Oct. 20, 2022. Kevin Willhelmsen, PhD, Associate Director of Immunology, will present on the same topic at the Inflammasome Therapeutics Summit [link] in Boston on Nov. 29, 2022.
In addition, at BIO-Europe [link], Peng Leong, PhD, MBA, Chief Business Officer and Head of Brain Aging at BioAge, will speak on a panel titled “Immuno-inflammation: A Gateway to Therapeutic Areas and New Partners” on Oct. 24, 2022. He will be joined by panelists Vishal Sahni, Director, Corporate Business Development and Strategy at H. Lundbeck A/S; Duncan Emmerton, Executive Director, Pharma Intelligence; and Andrew Mackie, Chief Business Officer, Imcyse.
“The BioAge target discovery platform revealed that NLRP3 activity is correlated with mortality and cognitive decline,” Leong said, “Inhibition of NLRP3 and other mediators of chronic inflammation identified by our platform has the potential to prevent multiple age-related disorders driven by pathologic inflammation.”
Leong will be available at BIO-Europe for potential partnership discussions. Attendees can send meeting requests to BioAge via the contact information in this release.
About BioAge Labs, Inc.
BioAge is a clinical-stage biotechnology company developing a pipeline of treatments to extend healthy lifespan by targeting the molecular causes of aging. The company uses its discovery platform, which combines quantitative analysis of proprietary longitudinal human samples with detailed health records to map out the key molecular pathways that impact healthy human aging. By targeting the mechanisms of aging with a large and mechanistically diverse portfolio of drugs, BioAge is unlocking opportunities to treat or even prevent age-related disease in entirely new ways. BioAge currently has multiple clinical-stage programs in its growing portfolio targeting muscle, immune, and brain aging. To date, BioAge has raised $127M from Andreessen Horowitz, Kaiser Foundation Hospitals, and others.
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CELL AND GENE THERAPY
Tiziana Life Sciences Ltd. | September 16, 2022
Tiziana Life Sciences Ltd. a biotechnology company developing breakthrough immunomodulation therapies via novel routes of drug delivery, today announces that a Lawrence & Isabel Barnett Drug Development Program Grant will be awarded to the Ann Romney Center for Neurologic Diseases at the Brigham and Women’s Hospital by the ALS Association for the study of an intranasal anti-CD3 monoclonal antibody in an animal model of Amyotrophic Lateral Sclerosis (ALS).
Howard L. Weiner, M.D., Co-Director of the Ann Romney Center for Neurologic Diseases at BWH and Chairman of Tiziana's Scientific Advisory Board, stated, “This prestigious research grant will be used to further study the role of intranasal anti-CD3 mAb in dampening the microglial activation which amplifies ALS disease progression. This research follows our recently presented positive findings on intranasal anti-CD3 mAb in Alzheimer’s Disease preclinical models of neuroinflammation. Additionally, we are currently studying foralumab, the first entirely human anti-CD3 mAb, in patients with secondary progressive multiple sclerosis.”
Gabriele Cerrone, Executive Chairman and interim Chief Executive Officer of Tiziana, remarked, “Intranasal foralumab has demonstrated potential across multiple Central Nervous System (CNS) indications. We are encouraged by the preclinical research using an intranasal anti-CD3 mAb in the neuroinflammatory related diseases of ALS and Alzheimer’s, as well as the impressive clinical benefits we have already shown for foralumab in patients with multiple sclerosis. While our initial focus is on our ongoing MS program which will continue to generate clinical read-outs, we are excited by foralumab’s potential to help highly debilitated ALS patients with limited therapeutic options and high unmet need.”
“We have now seen the potential of intranasal foralumab to dampen microglial activation in three major neuroinflammatory-related diseases, which creates significant optionality for exploring its benefits in some of the most important and burdensome medical conditions of our time.”
Matthew W. Davis, M.D., RPh, Chief Medical Officer of Tiziana
About the Barnett Drug Development Grant
The ALS Association’s Barnett Drug Development grant program supports preclinical drug discovery and development of new or repurposed treatments for ALS. There is an urgent need for new and improved therapies for ALS, as there is still no cure. The Lawrence and Isabel Barnett Drug Development Program is open to industry and academic investigators proposing to develop novel or repositioning approaches for ALS. The Association seeks applications for the preclinical assessment of therapeutics for ALS that have a high probability of reaching the clinic within three years.
About Foralumab
Foralumab the only entirely human anti-CD3 mAb, has shown reduced release of cytokines after IV administration in healthy volunteers and in patients with Crohn's disease. In a humanized mouse model it was shown that while targeting the T-cell receptor, orally administered foralumab modulates immune responses of the T-cells and enhances regulatory T-cells thereby providing therapeutic benefit in treating inflammatory and autoimmune diseases without the occurrence of potential adverse events usually associated with parenteral mAb therapy. Once a day treatment for 10 consecutive days with intranasal foralumab was both well tolerated and produced clinical responses in COVID-19 patients. Based on these studies, the intranasal and oral administration of Foralumab offers the potential to become a well-tolerated immunotherapy for autoimmune and inflammatory diseases by the induction of Tregs.
About Tiziana Life Sciences
Tiziana Life Sciences is a clinical-stage biopharmaceutical company developing breakthrough therapies using transformational drug delivery technologies to enable alternative routes of immunotherapy. Tiziana’s innovative nasal, oral and inhalation approaches in development have the potential to provide an improvement in efficacy as well as safety and tolerability compared to intravenous (IV) delivery. Tiziana’s two lead candidates, intranasal foralumab, the only fully human anti-CD3 mAb, and milciclib, a pan-CDK inhibitor, have both demonstrated a favorable safety profile and clinical response in patients in studies to date. Tiziana’s technology for alternative routes of immunotherapy has been patented with several applications pending and is expected to allow for broad pipeline applications.
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INDUSTRIAL IMPACT
FogPharma | November 22, 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,” today 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 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 (NCI) 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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