Category Archives: Biotech Companies

Vaxxas Expands Global Intellectual Property Portfolio for Exclusive Manufacture and Sale of Needle-Free Vaccination Technology

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BRISBANE, Australia & CAMBRIDGE, Mass.–(BUSINESS WIRE)–Vaxxas, a clinical-stage biotechnology company, has expanded its global intellectual property portfolio to 42 patents, supporting its exclusive claim to manufacture and sell its proprietary vaccination technology, the high-density microarray patch (HD-MAP), in the United States, Europe, Asia and Australia.

Vaxxas’ patent portfolio covers all significant proprietary aspects of the company’s vaccine delivery platform and products, including the manufacture and use of the HD-MAP technology and novel applicator device; vaccine formulations; and methods for formulating, loading and coating vaccines on HD-MAP technology.

The latest patent issued to Vaxxas in November 2024 covers the specific design and use of the HD-MAP and applicator technology for vaccine delivery to the skin in the United States.

Following issuance of the company’s latest US patent, Vaxxas CEO and President David Hoey said, “Vaxxas is aiming to transform the vaccine market by improving the performance of vaccines and reducing the economic and logistical challenges typically associated with needle-and-syringe vaccination.

“The growing breadth of our patent portfolio protects our exclusive use of the technologies necessary to achieve this goal, as well as freely manufacture and sell our products at commercial scale and in a safe and regulatory compliant manner.”

Vaxxas Chief Technology Officer Dr Angus Forster said the company has been very intentional in building its global intellectual property portfolio.

“The coverage of Vaxxas’ issued patents, both geographically and technologically, sets us apart as we continue to mature our vaccination platform and expand our range of products,” said Dr Forster.

“In the past 24 months alone, we have filed seven new patent applications covering new aspects of our technology platform, including HD-MAP delivery of mRNA vaccines, the fastest growing segment of the vaccine market. These patent applications, if granted, would extend our cover through to 2045.”

Vaxxas is scaling up to manufacture and distribute the world’s first commercially available vaccine patches from its global headquarters and state of the art biomedical facility in Brisbane.

Vaxxas Chief Operating Officer Scott Fry added, “Because we’re developing a combination product that includes a vaccine or medicine component with a medical device, we have broad patenting opportunities.

“We not only file patents around the vaccines we formulate and deliver, but also the systems and processes to manufacture the HD-MAPs and applicators at scale. This latest US patent is further evidence of the value our team of engineers brings to technology that could transform the vaccine industry.”

Vaxxas’ HD-MAP technology has completed five successful Phase I clinical trials involving over 500 participants with vaccines that address some of the world’s biggest health challenges including COVID-19, flu, and measles and rubella.

Vaxxas is currently conducting its first US IND-enabled Phase I clinical study for a pre-pandemic influenza vaccine involving 258 participants, with funding from the United States Biomedical Advanced Research and Development Authority (BARDA).

About Vaxxas

Vaxxas is a privately held biotechnology company focused on enhancing the performance of existing and next-generation vaccines with its proprietary high-density microarray patch (HD-MAP). Vaxxas is targeting initial applications in infectious diseases and oncology.

With success in several completed human clinical trials, Vaxxas’ HD-MAP vaccine delivery platform is advancing toward commercialisation. The company has completed Phase I clinical trials for COVID-19, seasonal influenza, and measles and rubella vaccine patches involving more than 500 participants; and conducted other vaccine studies targeting pandemic influenza with funding from the Biomedical Advanced Research and Development Authority (BARDA) – part of the Administration for Strategic Preparedness and Response within the U.S. Department of Health and Human Services.

Vaxxas’ core technology was initially developed at The University of Queensland (UQ), and the company was established as a start-up in 2011 by UQ’s commercialisation group UniQuest. The company was founded with the completion of an initial equity financing led by OneVentures Innovation Fund I with co-investors Brandon Capital Partners and US-based HealthCare Ventures, followed by further financing led by OneVentures, joined by UQ.

OneVentures Innovation Fund I and Brandon BioCatalyst are supported by the Australian Government’s Innovation Investment Fund (IIF) program. The IIF is an Australian Government venture capital initiative that provides investment capital and managerial expertise through licensed venture capital fund managers to investee companies. Learn more at OneVentures and Brandon Capital.

About HD-MAP needle-free vaccines

The Vaxxas high-density microarray patch (HD-MAP) is comprised of thousands of microscopic projections moulded into a small patch. Each microprojection is ‘printed’ with a small dose of vaccine in a dried formulation. When applied to the skin, the patch delivers the vaccine to the abundant immune cells that naturally reside immediately below the skin surface.

HD-MAP vaccine delivery has the potential to overcome challenges faced by traditional needle and syringe delivery of vaccines. For example, the dried form of the vaccine has been shown in early clinical studies to be more stable at higher temperatures than vaccines in liquid formulations, potentially reducing the need for cold-chain storage and distribution.1

Previous studies have also shown the safety and tolerability of Vaxxas’ HD-MAP for use in vaccine delivery and inducing equal or greater immune responses to injected vaccines at lower doses.1 Compared with needle and syringe systems, HD-MAP vaccines are designed to be easier to administer and have potential use in future pandemic responses.1

Caution

The Vaxxas HD-MAP delivered vaccines are under investigation and available only for investigational uses. They are not available anywhere in the world for sale or purchase. As such, Vaxxas makes no claim that the vaccines are reliable, durable, dependable, safe, or effective, and makes no claim that it is superior to any other vaccine or vaccine delivery technology.

References

1 Baker, B., Hacker, E., Siller, G., Lee, M., Mursaliyev, N., & Forster, A. (2023). Evaluation of the self-administration potential of high-density microarray patches to human skin: A preliminary study. Human Vaccines & Immunotherapeutics, 19(1). https://doi.org/10.1080/21645515.2023.2189409

Contacts

Australia
Amy Miller

WE Communications

+61 431 072 422

amymi@we-worldwide.com

United States
Kathryn Morris

The Yates Network

+1 914 204 6412

kathryn@theyatesnetwork.com

Zarminali Health Launches with $40M in Seed Funding to Transform Pediatric Care through Nationwide, Multi-specialty Practice Group

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New model for outpatient pediatric care combines seamlessly coordinated primary and specialty care with cutting-edge technology to transform the patient and family experience, and address physician burnout

CHICAGO, Nov. 20, 2024 (GLOBE NEWSWIRE) — Zarminali Health, the first outpatient pediatric destination purpose-built to provide modern, seamlessly coordinated primary and specialty care nationwide, today announced $40M in seed financing led by General Catalyst. The company will leverage the latest healthcare technology and an integrated approach to both primary and specialty care that alleviates family burdens and revitalizes patient and clinician experiences. This substantial funding, including a venture debt facility, will fuel the company’s go-to-market vision and continue to support the opening of its initial locations in fall 2024 and beyond.

Pediatric care today is long overdue for change. The current system is highly fragmented, and the disconnect between primary care providers, specialists, and ancillary services places the responsibility of navigating the healthcare system and complex care coordination squarely on families. Meanwhile, pediatricians are overburdened with high caseloads and unsustainable administrative tasks, clinical teams are under-supported with manual processes and outdated technology, and pediatric specialists remain siloed from primary care teams. All of these factors and inefficiencies exacerbate clinician burnout and hinder access to timely care for one of the most vulnerable populations in the country: children.

Zarminali was formed to deliver a renewed promise for pediatric care by streamlining care journeys for families, improving health outcomes for children, and enabling pediatric clinicians to focus on delivering quality care to their patients. Founded by Danish Qureshi, co-founder and former President and COO of LifeStance Health (NASDAQ: LFST), the company’s mission is directly informed by Danish’s personal experiences coordinating care for a child with an autoimmune disorder, and the many instances of breakdown in care across pediatric specialists.

“Zarminali is being painstakingly designed by parents like myself who have struggled against the current flaws of our healthcare system and know that every child deserves amazing care, from birth to adulthood,” said Qureshi. “After navigating the broken system with my daughter, I wanted to apply my experience transforming outpatient mental healthcare at LifeStance to improving pediatric care on a national scale. This initial funding round is an immense validator and kickstarts our mission to create a world where every child’s health journey is seamlessly coordinated, where advanced technology enhances every interaction, and where our clinicians and team members support each family every step of the way.”

“Pediatrics has long been overlooked, and we deeply share in Zarminali Health’s belief that our children and families deserve better care,” said Holly Maloney, Managing Director of General Catalyst. “With a strong founding team, collaborative care model, and advanced technology, we believe the team is on the right path to transform pediatric healthcare nationwide.”

Zarminali Health’s early team is a highly motivated and experienced group determined to improve pediatric care for all stakeholders. With backgrounds from other scaled national healthcare startups such as VillageMD (Walgreens and the Cigna Group), the team brings years of direct operating experience to tackle the challenges of pediatric care delivery. Additionally, Zarminali Health added its first independent board member in Brandon Kerns, bringing direct, relevant experience from Landmark Health (acquired by United Healthcare), Carebridge (acquired by Elevance), and Main Street Health.

To learn more about Zarminali Health, and how the company is transforming pediatric care, visit: https://zarminali.com/

About Zarminali Health
Zarminali Health, Inc. is building the nation’s leading pediatric multispecialty group, focused on supporting families to shape healthy futures for their children – from birth through adulthood – by completely transforming the way pediatric care is delivered nationwide. Zarminali is tackling today’s challenges of increased administrative burden on clinicians and a siloed approach to pediatric care through intentional design of care delivery, enhanced by leading technology and collaborative, expert care teams. We are leading the way towards a healthier future for pediatric patients and a happier future for pediatric clinicians nationwide. Visit zarminali.com for more information.

About General Catalyst
General Catalyst is a global investment and transformation company that partners with the world’s most ambitious entrepreneurs to drive resilience and applied AI. We support founders with a long-term view who challenge the status quo, partnering with them from seed to growth stage and beyond. With offices in San Francisco, New York City, Boston, Berlin, Bangalore, and London, we have supported the growth of 700+ businesses, including Airbnb, Anduril, Applied Intuition, Commure, Glean, Guild, Gusto, Helsing, Hubspot, Kayak, Livongo, Mistral, Ramp, Samsara, Snap, Stripe, Sword, and Zepto.

Media Contact
LaunchSquad for Zarminali Health
zarminalihealth@launchsquad.com

Steritas Announces Collaboration with argenx to Advance Evidence for Novel Steroid-sparing Therapeutics

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First-in-class steroid-toxicity outcome assessments licensed by argenx to support development of novel steroid-sparing therapeutics for autoimmune diseases

BOSTON–(BUSINESS WIRE)–Steritas LLC., a company dedicated to enhancing care for steroid-treated patients, today announced a licensing agreement with argenx, a global immunology company, for use of its STOX® Suite of steroid-toxicity clinical outcome assessments (COAs) across argenx’s development programs for differentiated immunology therapeutics.

The STOX Suite comprises first-in-class digital instruments that provide a standardized and validated measure of the full scope of steroid-toxicity. The COAs can be applied in research and at the point-of-care to demonstrate the safety and efficacy of new steroid-sparing treatments, and to enable clinicians to optimize treatment, taper steroid usage, and improve patient outcomes.

Under the terms of the multi-year agreement, argenx will incorporate the full STOX Suite as an innovative approach across its Immunology Innovation Program, including in clinical trials, health economics and outcome research, and real-world evidence. The strategic collaboration aims to strengthen the body of research evaluating chronic steroid usage in clinical groups and large populations, and to support development of argenx’s differentiated portfolio of novel antibody-based, medicines for the treatment of autoimmune diseases.

Following the first clinical use of cortisone in 1948, steroids have been used in almost all areas of medicine and are still considered first-line treatment for a wide range of conditions caused by inflammation, despite modern improvements in therapy options. The detrimental impact of long-term steroid use has led to an urgent need to research, monitor and reduce the use of steroids in patients and populations. Recognition of this need led to the development of the Glucocorticoid Toxicity Index (GTI), the first validated COA for quantifying steroid-toxicity, and forming the foundations of Steritas’ STOX Suite.

“Unmasking the damage of long-term steroid use is essential to countering this hidden epidemic – not only when developing new therapies for autoimmune disease, but also for other chronic conditions in which steroids are the standard of care.” commented Martha Stone, CEO at Steritas. She added: “This multi-year agreement with argenx to use the STOX Suite across its entire research program is recognition that steroid-toxicity is a vital area of study, with significance in routine patient care. This bold move by argenx to hold steroids accountable after more than 75 years of overuse will help alleviate patient suffering and healthcare burden.”

“Our pipeline of precision therapeutics is designed to bring relief to patients, both from the burden of the disease they are living with and the burden of existing treatments,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer, argenx. “Collaborating with Steritas to better understand the often profoundly negative impact of long-term steroid use is yet another way that argenx is focusing on the real-world impact our medicines can have for the patients we serve. We are grateful for the partnership with the Steritas team and look forward to deepening the body of evidence in favor of innovative steroid-sparing treatments.”

“Evaluating the burden of steroids in the different conditions we treat is generating important data about the extent of their use and detrimental long-term effects,” said Glenn Phillips, Ph.D., Global Head, VP HEOR, argenx. “Steritas has been a great partner in helping us explore and better understand the measurable negative impact of long-term steroid use and the potential benefit to patients of reducing the burdens associated with steroid-toxicity.”

Contacts

Lily Jeffery

Zyme Communications

Tel: +44 (0) 7891 477378

Email: lily.jeffery@zymecommunications.com

Cancer Targeted Technology Receives Fast Track $2.4M Grant to Develop an Innovative PSMA-Targeted Small Molecule Drug Conjugate for Prostate Cancer

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SEATTLE–(BUSINESS WIRE)–Cancer Targeted Technology (CTT), a privately-held Seattle-based biotechnology firm focusing on cancer diagnostics and therapeutics, announced that the National Cancer Institute awarded the Phase I portion ($400K) of the fast track Phase I/II ($2.4M) Small Business Innovation Research (SBIR) grant to develop a new drug to treat metastatic prostate cancer. The grant focuses on a promising new prodrug, CTT2274, that targets Prostate-Specific Membrane Antigen (PSMA) on prostate cancer and is designed to release a toxic drug, MMAE, within the cell that takes up the prodrug. PSMA is over-expressed on prostate cancer and expression increases as the cancer metastasizes and becomes castrate resistant. CTT’s unique phosphoramidate-based agents, bind irreversibly to PSMA and unlike other agents targeting PSMA, this distinctive mode of binding enhances uptake and internalization by tumor cells, leading to increased accumulation of the therapeutic payload and improved efficacy.

Studies to date using CTT2274 treatment of mice bearing human prostate tumors have shown remission of tumor growth and an overall increase in survival. In addition, because of the prodrug release within the tumor cells, safety of the drug, at doses that are effective at inhibiting or reversing tumor growth, is excellent. In the Phase I portion of the grant, to be completed in Q2 2025, CTT will conduct additional non-clinical efficacy studies and manufacturing optimization. In Phase II of the grant, to be completed in Q2, 2026, CTT will conduct additional manufacturing and safety assessments necessary to advance CTT2274 to an Investigational New Drug (IND) application. These IND studies will support the initial clinical trial in metastatic prostate cancer planned for 2026.

“CTT2274 has a unique structure and linker that maximizes tumor uptake and allows for release of the chemotherapeutic drug only within the tumor cell thus minimizing potential side effects from the chemotherapy. No other prodrug like this is being developed for prostate cancer and CTT2274 holds great promise as a future treatment for men suffering from prostate cancer.” stated Dr. Beatrice Langton-Webster, CEO of CTT and Principal Investigator on the grant. Visit CTT’s website at http://www.cancertargetedtechnology.com to learn more about CTT2274.

Contacts

Dr. Langton-Webster at bealw@cancertargetedtechnology.com

Adicet Bio Presents Clinical Biomarker Data for Off-the-Shelf CAR T Cell Therapy in an Oral Session at the American College of Rheumatology (ACR) Convergence 2024

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-Data demonstrate characteristics essential for treatment of autoimmune diseases, including robust tissue homing and complete CD19+ B cell depletion in secondary lymphoid tissue

-Results highlight the potential benefits unique to gamma delta 1 CAR T cell therapy and ADI-001’s potential as a best-in-class off-the-shelf cell therapy for autoimmune diseases

-The company is pursuing ADI-001 in a basket study across six indications including lupus nephritis (LN), systemic lupus erythematosus (SLE), systemic sclerosis (SSc), idiopathic inflammatory myopathy (IIM), stiff person syndrome (SPS) and anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV)

REDWOOD CITY, Calif. & BOSTON–(BUSINESS WIRE)–Adicet Bio, Inc. (Nasdaq: ACET), a clinical stage biotechnology company discovering and developing allogeneic, gamma delta T cell therapies for autoimmune diseases and cancer, today announced that clinical biomarker data from the ADI-001 Phase 1 GLEAN trial which demonstrates robust tissue homing, significant CAR T cell activation, and complete CD19+ B cell depletion in secondary lymphoid tissue will be featured in an oral session at ACR Convergence 2024 in Washington, D.C., November 14-19.

“We believe the key to advancing care for autoimmune patients is to develop a therapeutic candidate that demonstrates robust tissue homing, complete CD19+ B cell depletion in tissue, and superior drug exposure in secondary lymphoid tissue with a positive safety profile. We are proud to share data and analyses, including clinical biomarker data, at ACR that support the potential of ADI-001 in autoimmune diseases,” said Francesco Galimi, M.D., Ph.D., Chief Medical Officer. “After activating clinical trial sites for LN and receiving investigational new drug application (IND) clearances to pursue additional autoimmune indications, we are committed to advancing ADI-001 in a basket study across six autoimmune indications. This strategy highlights our focus on addressing the significant unmet medical needs of patients who urgently require innovative and effective new treatment options.”

A summary of the results is reported below:

  • ADI-001 demonstrated significant levels of CAR T cell activation and tissue exposure in lymph node biopsies in the GLEAN trial, representing a range of 27-64% of total cellular material detected by ddPCR in evaluable biopsies at the 1E9 dose, and exceeding levels previously reported for patients who received autologous alpha-beta CAR T therapies. CAR T cells detected in tissues also demonstrated a robust activation profile, based on in situ levels of granzyme B.
    • Recently published studies have demonstrated depletion of CD19+ plasmablasts, memory B cells and naïve B cells in peripheral blood using anti-CD20 targeted antibodies, however, these CD20-targeted antibody modalities failed to fully deplete B cells within secondary lymphoid tissue.
  • Concurrent with ADI-001 tissue trafficking and activation, complete depletion of CD19+ B cells within analyzed lymph node tissue was also observed. These results support ADI-001’s potential for achieving complete B-cell depletion in peripheral blood and within tissues.

Details of the oral presentation

Title: ADI-001: An Allogeneic CD20-targeted γδ CAR T Cell Therapy with Potential for Improved Tissue Homing in Autoimmune Indications

Session Name: Abstracts: Miscellaneous Rheumatic & Inflammatory Diseases II

Abstract Number: 1866169

Presenting Author: Monica Moreno, Ph.D.

Date and Time: November 19, 2024; 12:00 p.m. – 12:15 p.m. ET

About ADI-001 in Autoimmune Diseases

ADI-001 is an investigational allogeneic gamma delta CAR T cell therapy targeting B-cells via an anti-CD20 CAR. ADI-001 was granted Fast Track Designation by the FDA for the potential treatment of relapsed/refractory class III or class IV lupus nephritis (LN). Adicet is exploring the potential of ADI-001 in a basket study across six indications including lupus nephritis (LN), systemic lupus erythematosus (SLE), systemic sclerosis (SSc), idiopathic inflammatory myopathy (IIM), stiff person syndrome (SPS) and anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV).

For more information about becoming a study site, please email clinicaltrials@adicetbio.com or visit https://www.adicetbio.com/hcp/autoimmune/.

About the Phase 1 Clinical Trial

The Phase 1 study has four separate arms, enrolling LN and SLE patients into one arm, SSc patients into a second arm, IIM and SPS patients in a third arm and AAV patients into a fourth arm. Enrolled patients will receive a single dose of ADI-001. The dose-limiting toxicity window is 28 days with response and safety assessments conducted on Day 28 and during the follow-up period on months 3, 6, 9, 12, 18 and 24. The primary objectives of the study are to evaluate the safety and tolerability of ADI-001. Secondary objectives include measuring cellular kinetics, pharmacodynamics, changes in autoantibody titers, and appropriate disease activity scores in each indication.

About Adicet Bio, Inc.

Adicet Bio, Inc. is a clinical stage biotechnology company discovering and developing allogeneic gamma delta T cell therapies for autoimmune diseases and cancer. Adicet is advancing a pipeline of “off-the-shelf” gamma delta T cells, engineered with chimeric antigen receptors (CARs), to facilitate durable activity in patients. For more information, please visit our website at https://www.adicetbio.com.

Forward-Looking Statements

This press release contains “forward-looking statements” of Adicet within the meaning of the Private Securities Litigation Reform Act of 1995 relating to the business and operations of Adicet. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements include, but are not limited to, express or implied statements regarding: the potential safety, tolerability and efficacy of ADI- 001 in multiple autoimmune indications; the potential for ADI-001 to be best-in-class allogenic cell therapy for autoimmune diseases; and the clinical development of ADI-001 in LN, SLE, SSc, IIM, SPS and AAV.

Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements, including without limitation, the effect of global economic conditions and public health emergencies on Adicet’s business and financial results, including with respect to disruptions to our preclinical and clinical studies, business operations, employee hiring and retention, and ability to raise additional capital; Adicet’s ability to execute on its strategy including obtaining the requisite regulatory approvals on the expected timeline, if at all; that positive results, including interim results, from a preclinical or clinical study may not necessarily be predictive of the results of future or ongoing studies; clinical studies may fail to demonstrate adequate safety and efficacy of Adicet’s product candidates, which would prevent, delay, or limit the scope of regulatory approval and commercialization; and regulatory approval processes of the U.S. Food and Drug Administration and comparable foreign regulatory authorities are lengthy, time-consuming, and inherently unpredictable; and Adicet’s ability to meet production and product release expectations. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Adicet’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Adicet’s most recent annual report on Form 10-Q and subsequent filings with the U.S. Securities and Exchange Commission (SEC), as well as discussions of potential risks, uncertainties, and other important factors in Adicet’s other filings with the SEC. All information in this press release is as of the date of the release, and Adicet undertakes no duty to update this information unless required by law.

Contacts

Adicet Bio, Inc.
Investor and Media Contacts

Investors:
Anne Bowdidge

abowdidge@adicetbio.com

Janhavi Mohite

Precision AQ

212-362-1200

janhavi.mohite@precisionaq.com

Media:
Kerry Beth Daly

kbdaly@adicetbio.com

Epirium Bio Presented Late-Breaking Preclinical Data of MF-300 (15-PGDH Enzyme Inhibitor) at the Gerontological Society of America Annual Scientific Meeting 2024

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MF-300 is a first-in-class, orally administered, 15-PGDH enzyme inhibitor shown to restore muscle quality and strength in aged mice

SAN DIEGO–(BUSINESS WIRE)–Epirium Bio, Inc. (Epirium), a biopharmaceutical company advancing medicines for neuromuscular and fibrotic diseases, presented preclinical data for its lead candidate, MF-300, at the Gerontological Society of America’s (GSA) annual meeting being held November 13-16, 2024, in Seattle, WA.

MF-300 is a first-in-class, orally administered, 15-hydroxyprostaglandin dehydrogenase (15-PGDH) enzyme inhibitor in development for the treatment of age-induced muscle weakness, or sarcopenia. According to the U.S. Food and Drug Administration’s (FDA) Patient-Focused Drug Development Initiative report on sarcopenia, up to a third of Americans over the age of 60 are affected by sarcopenia, which increases their risk for falls, fractures and mortality. There are no FDA-approved therapies for sarcopenia, a disease that disproportionately affects fast-twitch muscle fibers, and is characterized by decreased muscle quality and muscle mass.

Epirium’s study, presented at GSA, tested whether MF-300 could improve muscle force in aged mice. After 12 weeks of oral administration at its target dose, MF-300 significantly increased absolute and specific force (i.e., force per unit of muscle) of isometric plantar flexion in aged animals compared to vehicle. The study also demonstrated that MF-300 significantly increased specific force and contraction rate in clinically relevant fast-twitch muscle. Together, these data show that MF-300 has the potential to reverse the decline in muscle quality (i.e., the loss of specific muscle force) that occurs with aging.

“The capacity of muscle to generate force is a better indicator of muscle quality, health, overall functional capacity, and survival than the quantity of muscle mass. MF-300’s novel mechanism of action on intrinsic muscle properties has the potential to improve functional outcomes and allow people to live physically independent lives longer,” said Dr. Scott Delp, Professor of Bioengineering, Stanford University Schools of Medicine and Engineering.

Details of the poster presentation include:

Title: MF-300 (15-PGDH Enzyme Inhibitor) Reverses Age-Related Muscle Weakness in Mice by Restoring Muscle Quality

Presenter: Micah Webster, Ph.D.

Poster Number: 1914819

The presentation is available in the “Posters and Publications” section of Epirium’s website, www.epirium.com.

About MF-300

MF-300 is an orally bioavailable small molecule that reversibly binds to the Prostaglandin E2 (PGE2)-binding site of 15-hydroxyprostaglandin dehydrogenase (15-PGDH), an enzyme that converts PGE2 to an inactive metabolite. MF-300 target engagement inhibits 15-PGDH activity, stabilizing and increasing levels of PGE2 in a cell-based assay and in vivo in skeletal muscle in preclinical studies.

In humans and rodents, 15-PGDH gene expression is elevated in muscle coincident with the onset of age-induced muscle weakness. PGE2, a lipid signaling molecule with multiple beneficial effects on the motor unit, including enhanced muscle quality and improved function of the neuromuscular junction, is reduced in skeletal muscle of aged mice due to increased activity of 15-PGDH. Inhibiting 15-PGDH in aged muscle may be a strategy to increase physiologic levels of PGE2 to improve muscle quality and function.

About Epirium Bio

Epirium, a biopharmaceutical company based in San Diego, California, has identified and established an IP-protected platform of orally bioavailable small molecules that constitute a new class of therapeutics with the potential to improve function in neuromuscular diseases, including sarcopenia and spinal muscular atrophy. Epirium has generated preclinical data in a broader scope of indications with significant unmet medical need, including fibrosis, which Epirium’s development pipeline has the potential to address.

To learn more about Epirium, please visit www.epirium.com and follow us on LinkedIn.

Contacts

Email: info@epirium.com

Anavex’s Blarcamesine Achieves Pre-specified Efficacy in Phase IIb/III Alzheimer’s Trial: Data Presented at CTAD Conference 2024

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Data of Blarcamesine confirm upstream SIGMAR1 activation

Presented as Late Breaking Oral Communications at Clinical Trials on Alzheimer’s Disease (CTAD) Conference 2024

Oral, once daily blarcamesine meaningfully slowed clinical decline for early Alzheimer’s disease patients with good comparative safety profile and no associated neuroimaging adverse events

NEW YORK, Oct. 31, 2024 (GLOBE NEWSWIRE) — Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative, neurodevelopmental, and neuropsychiatric disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome, schizophrenia, and other central nervous system (CNS) diseases, today presented new data from the Phase IIb/III study showing that blarcamesine (ANAVEX®2-73), once daily orally, demonstrates pre-specified clinical efficacy through upstream SIGMAR1 activation.

Clinical data confirmed the mechanism of action (MoA) by pre-specified SIGMAR1 gene analysis in people with early Alzheimer’s disease (AD). The data were presented by Marwan Noel Sabbagh, MD, Professor of Neurology at Barrow Neurological Institute and Chairman of the Anavex Scientific Advisory Board at the Clinical Trials on Alzheimer’s Disease (CTAD) conference, which is taking place October 29 – November 1, 2024, in Madrid, Spain.

SIGMAR1 is an integral membrane protein which activates an upstream compensatory process: Blarcamesine induces autophagy through SIGMAR1 activation resulting in restoring cellular homeostasis. In Alzheimer’s disease patients, mutations (variants) of genes have generally been identified as disease risk factors. Likewise, impaired SIGMAR1 function (gene mutation, variants) leads to potential suboptimal function. Hence, patients who carry the non-mutated, common SIGMAR1 wild type (WT)1 gene, are expected to have stronger beneficial response to blarcamesine than patients with a SIGMAR1 mutation (variant), who nevertheless also benefited from treatment.2

This was confirmed in the Phase IIb/III study analysis: Over 48 weeks, blarcamesine significantly slowed clinical progression by 36.3% in the primary endpoint ADAS-Cog13 [LS mean ADAS-Cog13 difference of -2.027; P=0.008] in the ITT analysis. This signal was even stronger in the pre-specified common SIGMAR1 wild type (WT) group with slowed clinical progression by 49.8% at 48 weeks in the active group vs. placebo, respectively [LS mean ADAS-Cog13 difference of -2.317; P=0.015]. Equal analysis with CDR-SB led to comparable consistent results.

“These data are very exciting, particularly featuring blarcamesine’s novel upstream mechanism of action, enhancing autophagy through SIGMAR1 activation, a key clearance mechanism that removes protein aggregates and misfolded proteins across the Alzheimer’s disease continuum,” said Juan Carlos Lopez-Talavera, MD, PhD, Head of Research and Development of Anavex. “The advantage of blarcamesine is that it is a small oral molecule that exerts clinical benefits on cognition and neurodegeneration and could be appealing because of its route of administration and good comparative safety profile. We are on track for regulatory submission of blarcamesine in Europe (EMA) in the current quarter 2024.”

Overall, blarcamesine, a small molecule administered orally once daily, demonstrated clinically meaningful improvement over 48 weeks with primary endpoint ADAS-Cog13 score being larger than 2 points.3 This suggests superior numerical clinical efficacy compared to approved therapies while also slowing neurodegeneration in early AD patients. Blarcamesine’s safety profile indicates not requiring routine MRI monitoring, and given its differentiated mechanism of action, could represent a novel treatment that could be complementary or an alternative to anti-beta amyloid monoclonal antibody drugs.

“Alzheimer’s disease is such a devastating disease that affects tens of millions worldwide. We believe, the clinically meaningful study results provide the potential for patients and their families to have a better and longer quality of life,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “We believe the scalable and convenient features of blarcamesine could reduce crucial barriers within the currently complex healthcare ecosystem for Alzheimer’s disease and provide broader access to a diverse population with early Alzheimer’s disease.”

The presentation is available on the Investors section of the Company’s website at www.anavex.com.

This release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that any investigational uses of such product will successfully complete clinical development or gain health authority approval.

About Anavex Life Sciences Corp.

Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of novel therapeutics for the treatment of neurodegenerative, neurodevelopmental, and neuropsychiatric disorders, including Alzheimer’s disease, Parkinson’s disease, schizophrenia, Rett syndrome, and other central nervous system (CNS) diseases, pain, and various types of cancer. Anavex’s lead drug candidate, ANAVEX®2-73 (blarcamesine), has successfully completed a Phase 2a and a Phase 2b/3 clinical trial for Alzheimer’s disease, a Phase 2 proof-of-concept study in Parkinson’s disease dementia, and both a Phase 2 and a Phase 3 study in adult patients and one Phase 2/3 study in pediatric patients with Rett syndrome. ANAVEX®2-73 is an orally available drug candidate designed to restore cellular homeostasis by targeting SIGMAR1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. ANAVEX®2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson’s Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX®2-73 for the treatment of Parkinson’s disease. We believe that ANAVEX®3-71, which targets SIGMAR1 and M1 muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer’s disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid, and tau pathologies. In preclinical trials, ANAVEX®3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at www.anavex.com. You can also connect with the Company on Twitter, Facebook, Instagram, and LinkedIn.

Forward-Looking Statements

Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company’s most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.

For Further Information:
Anavex Life Sciences Corp.
Research & Business Development
Toll-free: 1-844-689-3939
Email: info@anavex.com

Investors:
Andrew J. Barwicki
Investor Relations
Tel: 516-662-9461
Email: andrew@barwicki.com

1 WT = homozygous dominant (TT)
2 Hampel H, Williams C, Etcheto A, et al. A precision medicine framework using artificial intelligence for the identification and confirmation of genomic biomarkers of response to an Alzheimer’s disease therapy: Analysis of the blarcamesine (ANAVEX2-73) Phase 2a clinical study. Alzheimers Dement (N Y). 2020; 6(1):e12013.
3 Muir RT, Hill MD, Black SE, Smith EE. Minimal clinically important difference in Alzheimer’s disease: Rapid review. Alzheimers Dement. 2024;20(5):3352-3363. doi:10.1002/alz.13770

Resalis Therapeutics Gains Strategic Equity Investment from Sanofi

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TORINO, Italy–(BUSINESS WIRE)–Resalis Therapeutics announced today a strategic equity investment from Sanofi. The company intends to use the proceeds to accelerate the development of its lead investigational candidate, RES-010, through a Phase 2 proof-of-concept clinical trial. RES-010 represents a first-in-class antisense oligonucleotide targeting miR-22, a non-coding RNA that plays a pivotal role in the molecular pathways underlying obesity. This innovative approach positions Resalis to potentially be at the forefront of next-generation therapies in metabolic diseases.


Alessandro Toniolo, CEO of Resalis Therapeutics, stated: “The therapeutic landscape for obesity is rapidly evolving, and Sanofi’s investment highlights the potential of our approach. As we prepare to initiate a Phase 1 clinical trial for RES-010, this investment not only validates the robust data we’ve generated so far but also positions us to quickly advance through Phase 2 trials, bringing us closer to offering a potentially transformative treatment for patients living with obesity. We believe this will be a catalyst for further interest in this growing market.”

About RES-010

RES-010 is designed to reprogram metabolic pathways, providing a potential disease-modifying therapeutic impact that could include high-quality, sustained weight loss. By specifically targeting miR-22, a master regulator of lipid biosynthesis, mitochondrial function, and adipose tissue transformation, RES-010 has the potential to go beyond conventional obesity treatments that often focus solely on appetite reduction. This targeted approach has the potential to offer a more durable solution, addressing the complex biological underpinnings of obesity. The regulation of these pathways may result in a reduction of fat mass across various body districts, including visceral fat and hepatic stores. With its robust preclinical results, RES-010 has the potential to complement and enhance the efficacy of existing anti-obesity drugs, such as GLP-1 receptor agonists, and may extend their impact by providing a more comprehensive therapeutic solution.

About Resalis Therapeutics

Resalis Therapeutics is dedicated to developing RNA-based therapies that tackle the root causes of complex metabolic disorders. With its deep expertise in non-coding RNA and lipid metabolism, Resalis Therapeutics is advancing RES-010. Strong preclinical data support the rapid advancement of RES-010 through clinical trials, positioning Resalis Therapeutics as a key company in the evolving landscape of obesity treatment. Resalis is currently supported by investors Claris Ventures and Sunstone Life Science Ventures and private investors from Italian Angels for Growth and Club degli Investitori.

For more information visit our website www.resalistherapeutics.com

Contacts

Trophic Communications
+49 171 351 2733

resalis@trophic.eu

Agomab Announces $89 Million Series D Financing to Support Broad Fibrosis-Focused Pipeline

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— Round includes participation from new investors Sanofi and Invus, as well as existing investors —

— Proceeds will fund clinical development of lead candidate AGMB-129 in fibrostenosing Crohn’s disease, AGMB-447 in idiopathic pulmonary fibrosis and AGMB-101 in liver cirrhosis —

ANTWERP, Belgium–(BUSINESS WIRE)–Agomab Therapeutics NV (‘Agomab’) today announced a $89 million (€82.1 million) Series D financing round, with participation from new investors Sanofi and Invus, as well as existing investors.


The proceeds from the Series D will be used to further advance the ongoing clinical development of Agomab’s lead candidate, AGMB-129, a gut-restricted oral small molecule inhibitor of ALK5 (TGFβ1R), in patients with fibrostenosing Crohn’s disease (FSCD). Interim data from the Phase 2a STENOVA trial are expected in the first quarter of 2025. In addition, proceeds will be used to advance the clinical development for AGMB-447, a lung-restricted inhaled small molecule inhibitor of ALK5, that is currently in a Phase 1 clinical trial in healthy subjects and patients with idiopathic pulmonary fibrosis (IPF), as well as initial clinical development of AGMB-101, a full MET agonistic antibody currently in the final stages of IND-enabling studies, which the company intends to develop for liver cirrhosis.

“We are thrilled to have Sanofi and Invus joining our investor syndicate. Their investment as well as the continued support from our existing shareholders is another validation of the trailblazing work our team is conducting in the fields of fibrostenosing Crohn’s disease and idiopathic pulmonary fibrosis,” said Tim Knotnerus, Chief Executive Officer at Agomab Therapeutics. “Through this financing round we will create further optionality and the capital will allow us to accelerate our efforts in developing our novel potential therapies.”

About Agomab

Agomab is focused on achieving disease modification by modulating fibrosis and regeneration in chronic indications such as fibrostenosing Crohn’s disease and idiopathic pulmonary fibrosis. We do this by targeting biologically validated pathways – including Transforming Growth Factor β and Hepatocyte Growth Factor – and by applying specialized capabilities in organ-restricted small molecules and high affinity antibodies. With a differentiated clinical pipeline across several fibrotic disorders, end-to-end research and development capabilities, a proven BD track-record and a strong investor base, Agomab is building a leading biopharma company.

Contacts

For Agomab Therapeutics

Tim Knotnerus, CEO

E-Mail: tim.knotnerus@agomab.com

Media Requests for Agomab
Dr. Stephanie May

Trophic Communications

Phone: +49 171 1855682

E-Mail: agomab@trophic.eu

Qurient Launches Clinical Trial for Acute Myeloid Leukemia Treatment with Adrixetinib (Q702)

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SEONGNAM, South Korea–(BUSINESS WIRE)–#Qurient–Qurient Co. Ltd. (KRX: 115180) has announced the commencement of a clinical trial for adrixetinib (formerly named Q702), following the clearance of its investigational new drug (IND) application by the U.S. FDA.


The trial is a Phase 1 dose escalation and expansion study designed to assess the safety and preliminary efficacy of Q702 as a single agent and in a triplet combination with venetoclax and azacitidine for patients with relapsed/refractory acute myeloid leukemia (AML) (ClinicalTrials.gov Identifier: NCT06445907). Adrixetinib, an orally administered, selective inhibitor of AXL/MER/CSF1R kinases, has demonstrated significant anti-tumor activities, enhancing chemo-sensitivity and immune response across various tumor models. The innovative combination therapy is expected to work synergistically, potentially improving patient outcomes in AML treatment.

The trial’s principal investigator is Abhishek Maiti, M.D., assistant professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center in Houston.

The expression of AXL and MER (TAM family kinases) and CSF1R has been linked to a poorer prognosis in AML, positioning them as critical therapeutic targets within the cell and tumor microenvironment. Preclinical studies have shown the efficacy of AXL/MER inhibition, both as a standalone treatment and in combination with venetoclax, in various AML models, including aggressive FLT3-ITD bearing AML and venetoclax-resistant primary AML samples. CSF1R inhibition also targets AML by disrupting supportive microenvironmental signals, while AXL inhibition in macrophages may enhance the myeloid-centered anti-leukemia immune response.

Dr. Kiyean Nam, CEO of Qurient, conveyed his enthusiasm for the company’s strategic trajectory, stating, “The establishment of adrixetinib’s Phase 2 recommended dosage has set the stage for further clinical collaboration with leading American medical institutions for patients diagnosed with AML, but who may not be eligible for intensive chemotherapy. I believe that adrixetinib’s unique underlying biology can help these patients in combination with venetoclax and azacitidine.”

About Adrixetinib (Q702)

Adrixetinib, an innovative immunotherapy conceived by Qurient, is a selective triple kinase inhibitor that targets AXL, MER, and CSF1R. It enhances the body’s innate immune defenses and increases the susceptibility of cancer cells to therapeutic interventions. Q702 is currently in clinical development for the treatment of select advanced solid tumors and hematologic malignancies.

About Qurient

Qurient is a pioneering clinical-stage biopharmaceutical company headquartered in South Korea, listed on the Korea Exchange (KRX 115180). The company is dedicated to the development of novel therapeutics, from discovery to human proof-of-concept stages, utilizing a virtual R&D project management platform. For more information, visit www.qurient.com.

Contacts

Peter Yu, +(82)-31-8060-1600, media@qurient.com