4Moving Biotech Secures €7.6M France 2030 Grant for Pioneering First-In-Class Disease-Modifying Osteoarthritis Drug

  • France 2030 validates 4Moving Biotech’s scientific pioneering approach
  • This strategic support positions the 4P004 drug candidate at the forefront of knee osteoarthritis innovation
  • This additional funding accelerates 4P004’s development toward market launch by 2030

LILLE, France & PARIS–(BUSINESS WIRE)–4Moving Biotech (4MB), a spin-off of 4P-Pharma developing the first potential disease-modifying drug (DMOAD) for knee osteoarthritis, announces that it has been awarded €7.6 million in funding under the France 2030 plan to accelerate the clinical development of its lead candidate, 4P004, currently in a Phase 2a trial.

France 2030 is a highly selective and strategic initiative by the French government designed to fast-track the next generation of breakthrough innovations. 4MB’s selection is a significant endorsement, granted through a rigorous, multi-stage evaluation process by leading scientific, clinical, and technological experts.

This recognition highlights the robust scientific foundations of the 4P004 program and its potential to deliver a truly transformative therapy for OA’s unmet medical need and significant socio-economic impact with over 600 million people worldwide, including 10 million in France.

“We are honored by the renewed confidence through the France 2030 program,” said Revital Rattenbach, Co-founder and President of 4MB. “This highly competitive grant is a powerful validation of the scientific strengths of our 4P004 program and its potential to address one of the most pressing unmet medical needs.”

This additional funding will accelerate the 4P004 INFLAM MOTION Phase 2a clinical study, a GLP-1 analog designed to harness anti-inflammatory, analgesic, anti-catabolic, and anabolic effects across all joint tissues. The trial’s primary objective is to evaluate the efficacy of a single intra-articular injection of 4P004 in reducing knee pain while also assessing structural improvements via contrast-enhanced MRI and exploring novel biomarkers for disease progression and delayed need for total knee replacement.

“4MB gains extended momentum through The France 2030 grant marks an important milestone in the 4MB roadmap,” said Luc Boblet, CEO of 4MB. “With our phase 2a already initiated, this funding strengthens the value of our work, from early-stage discovery to clinical stage, and highlights the need to bring transformative therapies for patients suffering from OA”.

With the support of the France 2030 initiative, 4MB is fully equipped to deliver clinical proof-of-concept on time, as a critical inflection point that will unlock the next stage of development and reveal the full potential of 4P004 as a first-in-class DMOAD, creating significant attractiveness for future partnerships.

About 4MB

Incorporated in mid-2020 as a spin-off of 4P-Pharma, 4MB is a clinical stage biotechnology company dedicated to the development of the Disease-Modifying Osteoarthritis Drug (DMOAD). Its mission is to provide a sustainable therapeutic solution to the significant unmet medical need of osteoarthritis. The company is headquartered at the Pasteur Institute in Lille, France.

Website: https://www.4movingbiotech.com/
LinkedIn: https://fr.linkedin.com/company/4moving-biotech

The France 2030 investment plan

France 2030 is unprecedented in its scale: €54 billion is being invested so that our businesses, our schools, our universities and our research organizations fully succeed with their transitions in these strategic sectors. The challenge is to enable them to respond in a competitive way to the world’s future ecological and attractiveness challenges, and to showcase the future champions of our sectors of excellence. France 2030 is defined by two overarching objectives, which will see 50% of expenses given over to the decarbonization of the economy, and 50% to emerging key players, those at the forefront of innovation, without neglecting the environment (in the sense of the “Do No Significant Harm” principle).

France 2030 is being implemented collectively: designed and deployed in consultation with economic, academic, local and European key players to establish strategic guidelines and decisive action. Project holders are invited to submit their entries via the current application process, which is a demanding and selective one, to benefit from state support.

France 2030 is being led by the French General Secretariat for Investment, in charge of France 2030, on behalf of the Prime Minister, and in partnership with the relevant government ministries.

France 2030 is being implemented by the French National Research Agency (ANR), the French Agency for Ecological Transition (ADEME), Bpifrance and the Caisse des Dépôts et consignation (CDC).

More information : france2030.gouv.fr | @SGPI_avenir

About Bpifrance

Bpifrance is the French national investment bank: it finances businesses – at every stage of their development – through loans, guarantees, equity investments and export insurances. Bpifrance also provides extra financial services (training, consultancy) to help entrepreneurs meet their challenges (innovation, export…).

For more information, please visit: https://www.bpifrance.com/ Follow us on Twitter: @Bpifrance – @BpifrancePresse and LinkedIn

The only version of the 4Moving Biotech press release that is legally binding is the one in its original language. Translations must always be compared to the source text, which will establish precedence. The press release text resulting from a translation should not be considered official in any way.

Contacts

Press
Emmanuel Dadjé

Communication Manager

+33 6 30 06 12 13

Tune Therapeutics Completes Over $175M in Series B Financing to Advance Field-Leading Epigenome Editing Programs

Strong investor syndicate backs clinical-stage Hepatitis B program while advancing pioneering gene-tuning platform

DURHAM, N.C. & SEATTLE–(BUSINESS WIRE)–Leading epigenome editing company Tune Therapeutics (Tune) today announced the completion of over $175M in financing led by New Enterprise Associates, Yosemite, Regeneron Ventures and Hevolution Foundation.

“It is deeply gratifying to have seen this platform and company evolve so far,” said Tune Co-Founder Dr. Charles Gersbach, whose research at Duke University formed the basis for Tune’s TEMPO epi-editing platform. “Tune has already achieved a global landmark in the field, in the clinical application of epi-editing to a common and chronic disease. Thanks to the support of our investors, we anticipate the development of many more new epi-editing therapies in the years to come.”

The funding will accelerate the development of the company’s existing pipeline, currently anchored by Tune-401 – its clinical-stage epigenetic silencing drug for chronic Hepatitis B (HBV). It will also support the development of additional gene, cell, and regenerative therapy programs already underway at Tune, and to progress its broader mission of bringing the power and versatility of epigenetic therapies to bear on common and chronic diseases.

“We are incredibly proud to see Tune progress successfully into the clinic,” said Reed Jobs, Founder and Investor at Yosemite. “The Yosemite team has been an enthusiastic backer of Tune from the beginning, as we feel that few technologies have the biological power of epigenetic medicine to transform disease outcomes for the better. The range of potential applications and indications is vast and will only continue to expand.”

“To date, modern medicine and pharmacology has done much to extend our lifespans, but far less for our active healthspans,” explains William Greene, Chief Investment Officer at Hevolution Foundation. “Chronic diseases of ageing are accelerating in incidence, prevalence, and severity, and current approaches are simply inadequate. It is our belief that epigenetic editing may prove to be the transformative modality we need to enable a new era of regenerative medicine.”

Since its founding in 2021, Tune has made impressive strides in the development and application of its novel and potentially transformative epigenome editing platform. At the 2023 ASGCT conference, Tune announced a global first in the field: the durable repression of a therapeutically relevant gene (PCSK9) in non-human primates using genetic tuning. This gene repression was accompanied by an enduring reduction of LDL cholesterol levels that is still ongoing almost 2 years after a single, transient delivery of the epi-silencing construct. Later that year, the company unveiled Tune-401, a first-in-class epigenetic silencer for chronic Hepatitis B, a condition that impacts over 250 million people, and is the leading cause of liver cancer worldwide.

In November of 2024, Tune announced it was moving to the clinical stage, having received approval to begin clinical trials in New Zealand, and subsequently in Hong Kong – supported by world-renowned hepatologists and Principal Investigators Dr. Ed Gane and Dr. Man-Fung Yuen, respectively. With its lead program now in the clinic, Tune is leveraging this momentum to enhance its platform capabilities and develop its other gene and cell therapy programs.

“With this renewed support, we are well-positioned to advance our HBV clinical program, to invest in our platform, and to expand our pipeline,” said Akira Matsuno, Co-Founder, President and CFO of Tune Therapeutics. “We are grateful to all our investors for their deep confidence in our team and approach, backed by compelling data that continues to underscore the transformational potential of epi-editing as a therapeutic modality.”

About Tune Therapeutics

Armed with its powerful and innovative genetic tuning platform (TEMPO), Tune Therapeutics aims to bring gene, cell, and regenerative therapies into a new era of human medicine – expanding their range of application to common and chronic diseases.

About Tune-401

Tune-401 is a first-in-class investigational product candidate for treating Hepatitis B (HBV) infection. Tune-401 utilizes the company’s versatile, modular TEMPO platform to epigenetically silence viral HBV intDNA and cccDNA necessary for sustained HBV infection. Lipid nanoparticle technology for Tune-401 has been provided by Acuitas Therapeutics Inc.

Contacts

Glenn Murphy, Director of Communications: glenn.murphy@tunetx.com

 

 

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Immunis Closes $25 Million Series A-1 Financing Round

IRVINE, Calif.–(BUSINESS WIRE)–#ClinicalTrialsImmunis, Inc., a clinical-stage biotech developing multi-active biologics for age and disease-related immune dysregulation, has closed a $25 million Series A-1 financing round to fund two Phase 2 clinical trials. Follow-on investments from all major existing investors, notably, Remiges Ventures, Continuum Health Ventures and BOLD Capital Partners were joined by new investments from LifeSpan Vision Ventures and JLS Fund among others, including a leading global insurance company.

Mitigating muscle loss and improving metabolic function as we age are unmet medical needs. Following a successful Phase 1/2a clinical trial demonstrating the safety, tolerability and suggestive efficacy of IMM01-STEM in people with age-related muscle atrophy, the FDA granted permission to proceed with a Phase 2 clinical trial testing the efficacy of IMM01-STEM in ameliorating muscle loss and reversing metabolic dysfunction in sarcopenic, overweight and obese elderly people.

“Our Phase 1/2a data in nine elderly patients showed our therapy to be safe with no serious adverse events. Additionally, patients experienced striking improvements in quality-of-life measures related to physical function and pain as well as a clinically relevant increase in gait speed,” says Mark Cabato, Chief Business Officer of Immunis. “By running randomized, controlled studies in elderly populations with degenerative disease and metabolic dysfunction, we hope to demonstrate IMM01-STEM’s broad applicability in multiple medical conditions.”

Preclinical studies of Immunis’ novel therapy in aged mouse models of muscle disuse atrophy and metabolism were published in two notable journals, GeroScience and Aging Cell. The multi-active therapy was shown to reverse muscle atrophy, improve muscle function, enhance metabolism and decrease body and liver fat. Immunis is currently the leading biotech with the most advanced stem cell-derived multi-active biologic in human clinical trials.

“We are grateful for the support of our Board and investors for our mission to maximize health and minimize disease through the development of novel, stem cell-derived therapies,” adds Cabato.

About Immunis Inc.

Immunis is a private biotechnology company developing a novel immunomodulatory multi-active product for the various manifestations of age and disease-related immune decline. The investigational product line leverages Immunis’ leading-edge capabilities in multi-active secretome technology to deliver a product of all natural, all human immune modulators in their natural relative physiological concentrations. For additional information about Immunis’ Phase 1/2a clinical trial please visit: https://immunisbiomedical.com/clinical-trials/

Cautionary Note Regarding Forward-Looking Statements

This communication contains statements that constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as applicable. Forward-looking statements include, but are not limited to, statements regarding our plans, beliefs, expectations and assumptions, as well as other statements that are not necessarily historical facts. You are cautioned that these forward-looking statements are only predictions and involve risks and uncertainties. Further, any forward-looking statement speaks only of the date as of which it is made, and we do not intend to update or revise any forward-looking statements. This communication also contains market data related to our business and industry which includes projections that are based on several assumptions we believe are reasonable and most significant to the projections as of the date of this communication. If any of our assumptions prove to be incorrect, our actual results may significantly differ from our projections based on these assumptions. This communication is neither an offer to sell nor a solicitation of an offer to buy any of the securities described herein.

Contacts

contact@immunisbiomedical.com

 

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Verdiva Bio, a New Clinical-Stage Cardiometabolic Company, Launches with Over $410M in Series A Financing to Advance Next-Generation Therapies

 

  • Developing a broad portfolio of oral and injectable treatments with best-in-class potential to treat obesity, cardiometabolic disorders, and related complications
  • Groundbreaking potential of once-weekly oral GLP-1 and oral amylin therapies for weight loss and maintenance
  • Differentiated and clinically validated proprietary oral delivery technology designed to enable patient-friendly dosing, greater scalability, and broader patient access
  • Co-founded by Khurem Farooq and former members of the Aiolos Bio team with Mr. Farooq named Chief Executive Officer (CEO) and Mark Pruzanski named Chairman of the Board
  • Financing co-led by Forbion and General Atlantic, with participation from RA Capital Management, OrbiMed, Logos Capital, Lilly Asia Ventures, and LYFE Capital

LONDON & SAN FRANCISCO–(BUSINESS WIRE)–Verdiva Bio Limited (“Verdiva” or “the Company”) today announced its launch as a clinical-stage biopharmaceutical company focused on developing innovative therapies for obesity and other cardiometabolic disorders. The Company is advancing a portfolio of next-generation oral and injectable treatments with first-in-class or best-in-class potential. Verdiva launches with an oversubscribed Series A financing of $411M, co-led by Forbion and General Atlantic, with additional participation from RA Capital Management, OrbiMed, Logos Capital, Lilly Asia Ventures, and LYFE Capital (collectively, “the Investor Group”).

Khurem Farooq, formerly the CEO of Aiolos Bio and Gyroscope Therapeutics, will serve as Chief Executive Officer leading an experienced team of drug developers and biotech company builders. People living with obesity and its complications deserve better options at each stage of their treatment journey, including oral therapies with less frequent dosing regimens, the potential for improved efficacy and tolerability, and innovative combination therapies in pursuit of healthier weight loss and, equally importantly, maintenance of metabolically healthy weight. We created Verdiva Bio to accelerate the development of differentiated medicines that address these significant unmet medical needs,” said Farooq.

Addressing Verdiva’s lead asset, Farooq noted, Our most mature program has the potential to be a first-in-class, once-weekly oral treatment for obesity and weight loss maintenance that could dramatically improve patient access and affordability.”

Developing a broad portfolio of oral and injectable treatments

Verdiva acquired global development and commercialization rights outside of greater China and South Korea to their industry-leading portfolio from Sciwind Biosciences in 2024. Verdiva plans to advance the development of these next-generation therapies via a mix of monotherapy and combination programs, including:

  • A phase 2-ready, potential first-in-class, once-weekly oral GLP-1 receptor agonist
  • A potential first-in-class, once-weekly oral amylin agonist for use as monotherapy or in combination with an oral GLP-1 agonist
  • A long-acting, subcutaneous amylin agonist for use as monotherapy or in combination with a proprietary GLP-1 peptide

Senior leadership team

Dr. Mohamed Eid, MD, MSc, MHA joins Verdiva Bio as Chief Medical Officer from Boehringer Ingelheim, where he was Head of Clinical Development and Medical Affairs for cardiovascular, kidney, and metabolic medicines in the US. Previously, Dr. Eid held senior clinical, medical, and regulatory roles at Novo Nordisk.

Dr. Eid notes, We are excited by the potential of our innovative, investigational medicines. These programs represent next-generation potential against multiple targets and are anticipated to improve treatment adherence and offer a more sustainable solution for maintaining weight loss. Furthermore, by applying our clinically validated, oral delivery technology, we are confident that we can deliver highly efficacious and well tolerated therapies at markedly lower doses.”

In addition, the Company announced the appointment of several other leadership positions, including:

  • Dr. Jane Hughes, Chief Scientific Officer, formerly CSO of Aiolos Bio and Gyroscope Therapeutics
  • Dr. Tapan Maniar, Chief Business Officer, formerly CBO of Aiolos Bio and Principal at Bain Capital Life Sciences
  • Ashley Taylor, Chief Technology Officer, formerly CTO of Aiolos Bio and Head of Network Strategy at Roche/Genentech

In conjunction with the Company’s launch, Mark Pruzanski was named Chairman of the Board. A physician and entrepreneur with over 30 years of life sciences experience, Dr. Pruzanski previously served as Chairman and CEO of Versanis Bio (acquired by Eli Lilly) and was the founder and CEO of Intercept Pharmaceuticals.

This significant Series A financing will be used to progress the clinical development of our existing assets as well as to expand our industry-leading cardiometabolic portfolio, and we are grateful to this top-tier investor syndicate for their support,” said Dr. Pruzanski.

Substantial Series A financing

The Series A financing was co-led by Forbion and General Atlantic. We are thrilled to co-lead Verdiva’s Series A financing and support its mission of transforming lives by accelerating innovation in cardiometabolic health,” said Wouter Joustra, General Partner at Forbion, and Brett Zbar, Managing Director and Global Head of Life Sciences at General Atlantic. With a proven leadership team, a pipeline of potential next-generation oral therapies, and a clear vision for tackling some of the most pressing global health challenges, we both believe Verdiva Bio is well-positioned to deliver groundbreaking innovations and advance these promising therapies through clinical development and beyond.”

About Verdiva Bio

Verdiva Bio is committed to developing next-generation therapies to help people living with obesity, cardiometabolic disorders, and related complications achieve better outcomes via more patient-friendly therapeutic options. Verdiva’s most advanced therapy is VRB-101, an oral GLP-1 peptide in clinical development that has demonstrated best-in-class efficacy potential in a phase 1 study in Australia, which also confirmed the viability of once-weekly dosing. The Company is also developing a portfolio of amylin molecules, including oral and subcutaneous agonists, and other undisclosed programs that offer the potential for enhanced efficacy, improved tolerability, and healthier weight loss. The Verdiva team will harness the emerging science in gut-brain biology and leverage their history of successful drug development to advance novel therapeutic options aiming to transform the lives of millions living with obesity worldwide.

For more information, please visit www.verdivabio.com.

Contacts

Verdiva Bio
Tapan Maniar, CBO

Peter MacBride, VP, Strategy & Communications

info@verdivabio.com

Vigo Consulting (Media)
Melanie Toyne-Sewell

+44 7890 022 814

Rozi Morris

+44 7740 859 962

VerdivaBio@VigoConsulting.com

 

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

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

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

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

Seaport Therapeutics Closes $225 Million Oversubscribed Series B Financing Round

Financing led by General Atlantic with participation from T. Rowe Price Associates, Foresite Capital, Invus, Goldman Sachs Alternatives, Canada Pension Plan Investment Board (CPP Investments) as well as other new investors

Founding investors ARCH Venture Partners, Sofinnova Investments, Third Rock Ventures, and PureTech Health also participated

Proceeds will support key clinical milestones in Seaport’s pipeline of first and best-in-class neuropsychiatric medicines

BOSTON–(BUSINESS WIRE)–Seaport Therapeutics (“Seaport” or the “Company”), a clinical-stage biopharmaceutical company that is advancing novel neuropsychiatric medicines with a proven strategy and team, today announced the closing of an oversubscribed $225 million Series B financing round. The syndicate was led by General Atlantic, a leading global growth investor, with participation from funds and accounts advised by T. Rowe Price Associates, Inc., Foresite Capital, Invus, Goldman Sachs Alternatives, CPP Investments, and other new investors. Founding investors ARCH Venture Partners, Sofinnova Investments, Third Rock Ventures, and co-founder PureTech Health also participated.
The financing brings the total capital raised by Seaport to $325 million since the Company’s launch in April 2024. Seaport will use the proceeds to advance its clinical-stage pipeline of first and best-in-class medicines through important clinical milestones as well as further advance the capabilities of the Glyph™ technology platform, which has demonstrated clinical proof-of-concept.

“We are grateful to have the partnership of this incredible group of new and existing investors who share our commitment of delivering better medicines for those suffering from depression, anxiety and other neuropsychiatric disorders,” said Daphne Zohar, Founder and CEO of Seaport Therapeutics. “Seaport is advancing novel therapeutics that have proven clinical efficacy but had previously been held back by an issue we can now address with our Glyph platform. This financing enables the important clinical work that brings us another step closer to delivering new medicines to make a difference in the lives of patients and their families.”

“We are excited to partner with Daphne Zohar, Steve Paul and the team at Seaport,” said Brett Zbar, M.D., Managing Director and Global Head of Life Sciences at General Atlantic. “We are impressed with the team’s outstanding CNS clinical track record, as well as Seaport’s Glyph platform and innovative pipeline. The approach to clinical development and trial design demonstrates the deep neuropsychiatric expertise around the table, which we believe offers unique advantages that will contribute to Seaport’s success. We look forward to supporting the Company’s next phase of development.”

The programs in Seaport’s pipeline use the Glyph platform, which is designed to enable and enhance oral bioavailability, avoid first-pass metabolism and reduce liver enzyme elevations or hepatotoxicity and other side effects to advance clinically active drugs that were previously hindered by those limitations. The most advanced therapeutic candidate in the pipeline is SPT-300, an oral prodrug of allopregnanolone that is being advanced into a Phase 2b study for major depressive disorder with or without anxious distress that has the potential to be registration-enabling. Allopregnanolone is an endogenous neurosteroid with clinically validated rapid anti-depressant and anxiolytic activity, and SPT-300 retains this activity in an oral form.

“The development of important new neuropsychiatric medicines is often halted due to poor drug-like properties or unacceptable tolerability, challenges that our Glyph platform can now uniquely address,” said Steve Paul, M.D., Founder and Board Chair at Seaport Therapeutics. “For instance, xanomeline was an effective drug that faced tolerability challenges, but once resolved, led to the FDA approval of Cobenfy™ (formerly KarXT) for schizophrenia. With Glyph, we believe each of Seaport’s programs could create similar life-changing value for patients.”

SPT-320, a novel prodrug of agomelatine being advanced into Phase 1 studies for the treatment of generalized anxiety disorder (GAD), has the potential to be the first new mechanism for GAD in decades. SPT-320 uses Glyph to bypass liver first-pass metabolism and thus has the potential to lower the dose and reduce liver exposure while retaining efficacious systemic exposure of agomelatine that has been validated in multiple clinical studies in GAD. The reduction in dose has the potential to eliminate the need for liver function monitoring that has previously held back agomelatine’s development in GAD. SPT-348, a prodrug of a non-hallucinogenic neuroplastogen in development for the treatment of mood and other neuropsychiatric disorders, uses Glyph to create a potential first-in-class treatment. Beyond these programs, Seaport has multiple discovery and preclinical programs underway.

About the Glyph™ Platform

Glyph is Seaport’s proprietary technology platform which uses the lymphatic system to enable and enhance the oral administration of drugs. With the Glyph platform, drugs are absorbed like dietary fats through the intestinal lymphatic system and transported into circulation. The Glyph platform has the potential to be widely applied to many therapeutic molecules that have high first-pass metabolism leading to low bioavailability and/or side effects, including liver enzyme elevations or hepatotoxicity. Seaport exclusively licensed this technology from Monash University based on the pioneering research of the Porter Research Group. Advanced initially at PureTech Health and now at Seaport, Glyph has been applied to create therapeutic candidates for the Company’s pipeline resulting in new intellectual property, including composition of matter. The group and its collaborators have published research in Nature Metabolism, Frontiers in Pharmacology and the Journal of Controlled Release supporting the Glyph platform’s capabilities. See Glyph in action here.

About Seaport Therapeutics

Seaport Therapeutics is a clinical-stage biopharmaceutical company advancing the development of novel neuropsychiatric medicines in areas of high unmet patient needs. The Company has a proven strategy of advancing clinically validated mechanisms previously held back by limitations that are overcome with its proprietary Glyph technology platform. All the therapeutic candidates in its pipeline of first and best-in-class medicines are based on the Glyph platform, which is uniquely designed to enable oral bioavailability, bypass first-pass metabolism and reduce liver enzyme elevations or hepatotoxicity and other side effects. Seaport is led by an experienced team that invented and advanced important neuropsychiatric medicines and are guided by an extensive network of renowned scientists, clinicians and key opinion leaders. For more information, please visit www.seaporttx.com.

Contacts

Seaport Therapeutics
Shannon Costello

publicrelations@seaporttx.com

Firefly Neuroscience Announces Use of Its Brain Network Analytics Platform in Phase 1 Study of SP-624, the First Potential Treatment Designed for Women with Major Depressive Disorder

  • Study to Leverage Firefly’s BNA™ Technology for Biomarker Identification, with Potential Applications for Broader Neurological Research

TORONTO, Oct. 15, 2024 (GLOBE NEWSWIRE) — Firefly Neuroscience, Inc. (“Firefly,” or the “Company”) (NASDAQ: AIFF), an Artificial Intelligence (“AI”) company developing innovative solutions that improve brain health outcomes for patients with neurological and mental disorders, today announced that its FDA-510(k) cleared Brain Network Analytics (BNA™) biomarker discovery AI platform will be used to support Arrivo Bioventures’ (“Arrivo”) Phase 1 exploratory study of its first-in-class SIRT6 activator, SP-624, in healthy volunteers and patients with major depressive disorder (MDD).

The study, which has enrolled its first subject, is designed to evaluate the impact of the epigenetic mechanism of action of SP-624 on neurological pathways and assess changes in various cognitive domains. SP-624 is also currently being studied in a large Phase 2b study in patients diagnosed with MDD, with efficacy in females as the primary endpoint.

“We believe that utilizing Firefly’s BNA™ technology in the SP-624 study will help Arrivo better understand SP-624’s activity and uncover critical biomarkers that may inform future research across neurological conditions,” said Jon Olsen, CEO of Firefly. “Together with Arrivo, this study underscores our shared commitment to advancing targeted treatments for women with major depressive disorder, which continues to be an unmet medical need in mental health.”

In an earlier Phase 2 study of SP-624, Arrivo explored efficacy over placebo during a four week treatment period. In a post-hoc analysis, the company found statistically significant improvement versus placebo consistent across both investigator and patient measures of MDD in female subjects, which was achieved as early as week 2. In male subjects, there were no statistically significant changes detected. While differences in MDD between males and females have long been discussed, these results support recent published literature citing differences in gene expression between males and females with MDD. Using Firefly’s BNA technology, Arrivo will also identify target engagement and biomarkers in the Phase 1 study that can be used in future studies across a variety of neurological conditions, including MDD.

“There is a growing body of literature suggesting that targeting SIRT6 can play an important role in multiple neuropsychiatric, neurodegenerative, inflammatory, and metabolic diseases,” said Steve Butts, CEO of Arrivo. “This study should help us in our ongoing efforts to characterize the activity of SP-624 and look for potential biomarkers.”

The Phase 1, single-center, double-blind, randomized, placebo-controlled study is being conducted at Alivation Research with Walt Duffy, M.D., founder, CEO and Chief Medical Officer, as the Principal Investigator.

About Firefly
Firefly (NASDAQ: AIFF) is an Artificial Intelligence (“AI”) company developing innovative solutions that improve brain health outcomes for patients with neurological and mental disorders. Firefly’s FDA-510(k) cleared Brain Network Analytics (BNA™) technology revolutionizes diagnostic and treatment monitoring methods for conditions such as depression, dementia, anxiety disorders, concussions, and ADHD. Over the past 15 years, Firefly has built a comprehensive database of brain wave tests, securing patent protection, and achieving FDA clearance. The Company is now launching BNA™ commercially, targeting pharmaceutical companies engaged in drug research and clinical trials, as well as medical practitioners for clinical use.

Brain Network Analytics was developed using artificial intelligence and machine learning on Firefly’s extensive proprietary database of standardized, high-definition longitudinal electroencephalograms (EEGs) of over 17,000 patients representing twelve disorders, as well as clinically normal patients. BNA™, in conjunction with an FDA-cleared EEG system, can provide clinicians with comprehensive insights into brain function. These insights can enhance a clinician’s ability to accurately diagnose mental and cognitive disorders and to evaluate what therapy and/or drug is best suited to optimize a patient’s outcome.

Please visit https://fireflyneuro.com/ for more information.

About Arrivo BioVentures
Arrivo BioVentures is propelled forward by its insatiable curiosity and drive to solve complex problems and help millions of patients globally. Working in partnership with investors, innovators, and pharmaceutical companies, Arrivo is always seeking solutions for unmet medical needs. Arrivo has a portfolio of diverse drug candidates with the potential to be first-in-class or best-in-class. Arrivo is based in Morrisville, N.C., on the edge of Research Triangle Park. For more information, visit www.arrivobio.com.

Forward-Looking Statements
Certain statements in this press release and the information incorporated herein by reference may constitute “forward-looking statements” for purposes of the federal securities laws concerning Firefly. These forward-looking statements include express or implied statements relating to Firefly’s management teams’ expectations, hopes, beliefs, intentions, or strategies regarding the future. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “intends,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. These forward-looking statements are based on current expectations and beliefs concerning future developments and their potential effects. There can be no assurance that future developments affecting Firefly will be those that have been anticipated. These forward-looking statements involve a number of risks, uncertainties (some of which are beyond Firefly’s control) or other assumptions that may cause actual results or performance to be materially different from those expressed or implied by these forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to development and commercialization of BNA™ technology; risks related to Firefly’s ability to recognize the anticipated benefits of the merger (the “Merger”) with WaveDancer, Inc. (“WaveDancer”); risks related to Firefly’s ability to correctly estimate its operating expenses and expenses associated with the Merger and other events and unanticipated spending and costs that could reduce Firefly’s cash resources; the ability of Firefly to protect its intellectual property rights; competitive responses to the business combination; unexpected costs, charges or expenses resulting from the Merger; potential adverse reactions or changes to business relationships resulting from the completion of the Merger; legislative, regulatory, political and economic developments; and those factors described under the heading “Risk Factors” in the in the registration statement on Form S-4 filed by WaveDancer with the Securities and Exchange Commission on January 22, 2024, as amended, and declared effective on February 6, 2024. Should one or more of these risks or uncertainties materialize, or should any of Firefly’s assumptions prove incorrect, actual results may vary in material respects from those projected in these forward-looking statements. It is not possible to predict or identify all such risks. Forward-looking statements included in this press release only speak as of the date they are made, and Firefly does not undertake any obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws.

Arrivo Bio Media Contact
ICR Healthcare
Alexis Feinberg, Vice President
Alexis.feinberg@westwicke.com

Firefly Neuroscience Investor Contacts
KCSA Strategic Communications
Valter Pinto / Jack Perkins
(212) 896-1254
Firefly@KCSA.com

Firefly Neuroscience Media Contact
KCSA Strategic Communications
Raquel Cona, Vice President
(516) 779-2630
Rcona@KCSA.com

Bayer announces positive topline results for NUBEQA® (darolutamide) from Phase III trial in men with metastatic hormone-sensitive prostate cancer (mHSPC)

  • Phase III ARANOTE trial met primary endpoint, significantly increasing radiological progression-free survival (rPFS) with NUBEQA plus androgen deprivation therapy (ADT) compared to placebo plus ADT
  • Results were consistent with NUBEQA’s established safety profile with no new signals observed
  • NUBEQA now has positive mHSPC data both with and without docetaxel based on two pivotal Phase III studies
  • Bayer plans to present the pivotal data at a forthcoming scientific congress and discuss these data with the U.S. Food and Drug Administration (FDA) for regulatory approval

WHIPPANY, N.J.–(BUSINESS WIRE)–The Phase III ARANOTE trial, investigating NUBEQA® (darolutamide) plus androgen deprivation therapy (ADT) in patients with metastatic hormone-sensitive prostate cancer (mHSPC), has met its primary endpoint of radiological progression-free survival (rPFS). NUBEQA plus ADT demonstrated a statistically significant and clinically meaningful increase in rPFS compared to placebo plus ADT.

Results were consistent with NUBEQA’s established safety profile with no new signals observed. Detailed results from this randomized, double-blind, placebo-controlled trial are planned to be presented at a forthcoming scientific congress.

NUBEQA is currently indicated in the U.S. for the treatment of adult patients with mHSPC in combination with docetaxel and for the treatment of adult patients with non-metastatic castration-resistant prostate cancer (nmCRPC).1

We are excited to share the positive results from this Phase III trial. Following potential regulatory approval, physicians will be able to tailor NUBEQA treatment plans with or without docetaxel based on individual patient’s needs,” said Christian Rommel, Ph.D., Head of Research and Development at Bayer’s Pharmaceuticals Division. “Today’s results build on the established efficacy and tolerability profile of NUBEQA. We are looking forward to future outcomes of our clinical development program investigating the compound across multiple prostate cancer stages and indications.”

Bayer plans to present the pivotal data at a forthcoming scientific conference and discuss these data with the U.S. FDA regarding submission for regulatory approval.

About the ARANOTE Trial

The ARANOTE trial (NCT04736199) is a randomized, double-blind, placebo-controlled Phase III study designed to assess the efficacy and safety of NUBEQA plus androgen deprivation therapy (ADT) in patients with metastatic hormone-sensitive prostate cancer (mHSPC). 669 patients were randomized to receive 600mg of NUBEQA twice daily or matching placebo in addition to ADT.

The primary endpoint of this study is radiological progression-free survival (rPFS), as measured as the time from the date of randomization to the date of first documentation of radiological progressive disease or death due to any cause, whichever occurs first. Secondary endpoints include overall survival, time from randomization to the date of death from any cause, time from randomization to the date of first castration-resistant event, time to initiation of subsequent anti-cancer therapy, time to prostate-specific antigen (PSA) progression, PSA undetectable rates, time to pain progression, and safety assessments.

About NUBEQA® (darolutamide)1

NUBEQA® (darolutamide) is an androgen receptor inhibitor (ARi) with a distinct chemical structure that competitively inhibits androgen binding, AR nuclear translocation, and AR-mediated transcription.

NUBEQA is being evaluated in a robust clinical development program, which includes studies across various stages of prostate cancer, including in the Phase III ARANOTE trial evaluating NUBEQA plus androgen deprivation therapy (ADT) versus ADT alone for mHSPC, the ARASTEP Phase III trial evaluating NUBEQA plus ADT versus ADT alone in HSPC patients with high-risk biochemical recurrence (BCR) and no evidence of metastatic disease by conventional imaging and a positive PSMA PET/CT at baseline, as well as in the Australian and New Zealand Urogenital and Prostate Cancer Trials Group (ANZUP) led international Phase III co-operative group DASL-HiCaP (ANZUP1801) trial evaluating NUBEQA as an adjuvant treatment for localized prostate cancer with very high risk of recurrence. Information about these trials can be found at www.clinicaltrials.gov.

NUBEQA is currently indicated in the U.S. in combination with docetaxel and ADT for the treatment of adult patients with mHSPC and for the treatment of adult patients with non-metastatic castration-resistant prostate cancer (nmCRPC) with ADT.1

NUBEQA is developed jointly by Bayer and Orion Corporation, a globally operating Finnish pharmaceutical company.

INDICATIONS

NUBEQA® (darolutamide) is an androgen receptor inhibitor indicated for the treatment of adult patients with:

  • Non-metastatic castration-resistant prostate cancer (nmCRPC)
  • Metastatic hormone-sensitive prostate cancer (mHSPC) in combination with docetaxel

IMPORTANT SAFETY INFORMATION

NUBEQA® (darolutamide) is an androgen receptor inhibitor indicated for the treatment of adult patients with:

  • Non-metastatic castration-resistant prostate cancer (nmCRPC)
  • Metastatic hormone-sensitive prostate cancer (mHSPC) in combination with docetaxel

IMPORTANT SAFETY INFORMATION

Warnings & Precautions

Ischemic Heart Disease – In a study of patients with nmCRPC (ARAMIS), ischemic heart disease occurred in 3.2% of patients receiving NUBEQA versus 2.5% receiving placebo, including Grade 3-4 events in 1.7% vs. 0.4%, respectively. Ischemic events led to death in 0.3% of patients receiving NUBEQA vs. 0.2% receiving placebo. In a study of patients with mHSPC (ARASENS), ischemic heart disease occurred in 3.2% of patients receiving NUBEQA with docetaxel vs. 2% receiving placebo with docetaxel, including Grade 3-4 events in 1.3% vs. 1.1%, respectively. Ischemic events led to death in 0.3% of patients receiving NUBEQA with docetaxel vs. 0% receiving placebo with docetaxel. Monitor for signs and symptoms of ischemic heart disease. Optimize management of cardiovascular risk factors, such as hypertension, diabetes, or dyslipidemia. Discontinue NUBEQA for Grade 3-4 ischemic heart disease.

Seizure – In ARAMIS, Grade 1-2 seizure occurred in 0.2% of patients receiving NUBEQA vs. 0.2% receiving placebo. Seizure occurred 261 and 456 days after initiation of NUBEQA. In ARASENS, seizure occurred in 0.6% of patients receiving NUBEQA with docetaxel, including one Grade 3 event, vs. 0.2% receiving placebo with docetaxel. Seizure occurred 38 to 340 days after initiation of NUBEQA. It is unknown whether antiepileptic medications will prevent seizures with NUBEQA. Advise patients of the risk of developing a seizure while receiving NUBEQA and of engaging in any activity where sudden loss of consciousness could cause harm to themselves or others. Consider discontinuation of NUBEQA in patients who develop a seizure during treatment.

Embryo-Fetal Toxicity – Safety and efficacy of NUBEQA have not been established in females. NUBEQA can cause fetal harm and loss of pregnancy. Advise males with female partners of reproductive potential to use effective contraception during treatment with NUBEQA and for 1 week after the last dose.

Adverse Reactions

In ARAMIS, serious adverse reactions occurred in 25% of patients receiving NUBEQA vs. 20% of patients receiving placebo. Serious adverse reactions in ≥1% of patients who received NUBEQA included urinary retention, pneumonia, and hematuria. Fatal adverse reactions occurred in 3.9% of patients receiving NUBEQA vs. 3.2% of patients receiving placebo. Fatal adverse reactions in patients who received NUBEQA included death (0.4%), cardiac failure (0.3%), cardiac arrest (0.2%), general physical health deterioration (0.2%), and pulmonary embolism (0.2%). The most common adverse reactions (>2% with a ≥2% increase over placebo), including laboratory test abnormalities, were increased AST, decreased neutrophil count, fatigue, increased bilirubin, pain in extremity and rash. Clinically relevant adverse reactions occurring in ≥2% of patients treated with NUBEQA included ischemic heart disease and heart failure.

In ARASENS, serious adverse reactions occurred in 45% of patients receiving NUBEQA with docetaxel vs. 42% of patients receiving placebo with docetaxel. Serious adverse reactions in ≥2% of patients who received NUBEQA with docetaxel included febrile neutropenia (6%), decreased neutrophil count (2.8%), musculoskeletal pain (2.6%), and pneumonia (2.6%). Fatal adverse reactions occurred in 4% of patients receiving NUBEQA with docetaxel vs. 4% of patients receiving placebo with docetaxel. Fatal adverse reactions in patients who received NUBEQA included COVID-19/COVID-19 pneumonia (0.8%), myocardial infarction (0.3%), and sudden death (0.3%). The most common adverse reactions (≥10% with a ≥2% increase over placebo with docetaxel) were constipation, rash, decreased appetite, hemorrhage, increased weight, and hypertension. The most common laboratory test abnormalities (≥30%) were anemia, hyperglycemia, decreased lymphocyte count, decreased neutrophil count, increased AST, increased ALT, and hypocalcemia. Clinically relevant adverse reactions in <10% of patients who received NUBEQA with docetaxel included fractures, ischemic heart disease, seizures, and drug-induced liver injury.

Drug Interactions

Effect of Other Drugs on NUBEQA – Combined P-gp and strong or moderate CYP3A4 inducers decrease NUBEQA exposure, which may decrease NUBEQA activity. Avoid concomitant use.

Combined P-gp and strong CYP3A4 inhibitors increase NUBEQA exposure, which may increase the risk of NUBEQA adverse reactions. Monitor more frequently and modify NUBEQA dose as needed.

Effects of NUBEQA on Other Drugs – NUBEQA inhibits breast cancer resistance protein (BCRP) transporter. Concomitant use increases exposure (AUC) and maximal concentration of BCRP substrates, which may increase the risk of BCRP substrate-related toxicities. Avoid concomitant use where possible. If used together, monitor more frequently for adverse reactions, and consider dose reduction of the BCRP substrate.

NUBEQA inhibits OATP1B1 and OATP1B3 transporters. Concomitant use may increase plasma concentrations of OATP1B1 or OATP1B3 substrates. Monitor more frequently for adverse reactions and consider dose reduction of these substrates.

Review the Prescribing Information of drugs that are BCRP, OATP1B1, and OATP1B3 substrates when used concomitantly with NUBEQA.

For important risk and use information about NUBEQA, please see the accompanying full Prescribing Information.

About Metastatic Hormone-Sensitive Prostate Cancer

Prostate cancer is the second most common cancer in men and the fifth most common cause of cancer death in men worldwide.2 In 2020, an estimated 1.4 million men were diagnosed with prostate cancer, including almost 300,000 cases in the U.S., and about 375,000 died from the disease worldwide.3,4

At the time of diagnosis, most men have localized prostate cancer, meaning their cancer is confined to the prostate gland and can be treated with curative surgery or radiotherapy. Upon relapse when the disease will metastasize or spread, androgen deprivation therapy (ADT) is the cornerstone of treatment for this hormone-sensitive disease. Approximately 10% of men will already present with mHSPC when first diagnosed.5,6,7 Men with metastatic hormone-sensitive prostate cancer (mHSPC) will start their treatment with hormone therapy, such as ADT, androgen receptor inhibitor (ARi) plus ADT or a combination of the chemotherapy docetaxel and ADT. Despite this treatment, most men with mHSPC will eventually progress to castration-resistant prostate cancer (CRPC), a condition with limited survival.

About Oncology at Bayer

Bayer is committed to delivering science for a better life by advancing a portfolio of innovative treatments. The oncology franchise at Bayer includes six marketed products and several other assets in various stages of clinical development. Together, these products reflect the company’s approach to research, which prioritizes targets and pathways with the potential to impact the way that cancer is treated.

About Bayer

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to www.bayer.com.

© 2024 Bayer

BAYER, the Bayer Cross and NUBEQA are registered trademarks of Bayer.

Forward-Looking Statements

This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

References

  1. NUBEQA (darolutamide) [Prescribing Information]. Whippany, NJ: Bayer HealthCare Pharmaceuticals, Inc.; October 2023.
  2. Hyuna S et al. Ca Cancer J Clin 2021; 71:209–249.
  3. Prostate Cancer: Statistic. Cancer.Net. https://www.cancer.net/cancer-types/prostate-cancer/statistics. Accessed: January 2024.
  4. American Cancer Society. Cancer Facts & Figures 2024. Accessed: January 2024.
  5. Piombino C et al. Cancers (Basel). 2023 Oct 11;15(20):4945.
  6. Helgstrand JT et al. Cancer. 2018;124(14):2931-2938.
  7. Buzzoni C et al. Eur. Urol. 2015;68:885–890.

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Contacts

Sue Ann Pentecost, Tel + 910.221.6446

Email: sueann.pentecost@bayer.com

Artelo Biosciences Receives FDA Clearance of its IND Application for ART26.12, a Selective Fatty Acid Binding Protein 5 Inhibitor

ART26.12 seeks to address a critical need in painful neuropathies, including chemotherapy-induced peripheral neuropathy for which there is no FDA-approved treatment

Phase 1 trial results expected in the first half of 2025

SOLANA BEACH, Calif., July 15, 2024 (GLOBE NEWSWIRE) — Artelo Biosciences, Inc. (Nasdaq: ARTL), a clinical-stage pharmaceutical company focused on modulating lipid-signaling pathways to develop treatments for people living with cancer, pain, dermatologic and neurological conditions, today announced that the U.S. Food and Drug Administration (FDA) has issued a “Study May Proceed” letter for the Company’s Investigational New Drug (IND) application for ART26.12, for the treatment of chemotherapy-induced peripheral neuropathy (CIPN). FDA clearance of the ART26.12 IND application enables the Company to initiate its first-in-human Phase 1 single ascending dose study. Study startup activities have been initiated in collaboration with the internationally known contract research organization Worldwide Clinical Trials.

ART26.12 is the lead compound in the Company’s proprietary Fatty Acid Binding Protein (FABP) platform and the first selective FABP5 inhibitor to enter clinical trials. The FABP5 target is an intracellular protein involved in lipid signaling and represents a promising mechanism of action for drug candidates that can modify the cellular lipidome. ART26.12 is being developed as a non-opioid approach to the management of painful neuropathies. The Company’s FABP inhibitor platform, and ART26.12 in particular, has garnered interest from a range of potential partners due to its preclinical demonstation of efficacy, novel mechanism, and strong patent estate.

“Receiving IND clearance validates our development efforts and underscores the potential impact of ART26.12 to improve patients’ lives,” said Gregory D. Gorgas, President and Chief Executive Officer of Artelo Biosciences. “We look forward to sharing the initial clinical results with ART26.12 next year. As the leading company pursuing FABP inhibiton we are committed to building on the unique, lipid-modulating mechanism of our FABP inhibitor platform to address life-altering pathologies for which there are few, if any, safe and effective pharmaceutical treatments.”

About ART26.12

Fatty Acid Binding Proteins (FABPs) are a family of intracellular proteins that chaperone lipids involved in cellular signaling. FABPs are often overexpressed and associated with abnormal lipid signaling in a number of pathologies. ART26.12, Artelo’s lead FABP inhibitor program in clinical development, is a potent and selective small molecule inhibitor of FABP5 being developed as an orally delivered, peripherally acting, non-opioid, new chemical entity for cancer patients suffering from chemotherapy- induced peripheral neuropathy. Invented by Distinguished Professor Iwao Ojima working in collaboration with Professor Martin Kaczocha, both at Stony Brook University, the extensive library of FABP inhibitors was exclusively licensed to Artelo with global rights. Preclinical evidence to date suggest FABP inhibition has broad therapeutic promise for the treatment of multiple cancers, painful neuropathies, cancer bone pain, dermatologic conditions and anxiety disorders.

About CIPN

Chemotherapy-induced peripheral neuropathy (CIPN) is a type of neuropathic pain caused by chemotherapy. Some chemotherapies result in CIPN with 90% frequency. CIPN often results in dose reduction or cessation of the cancer treatment leading to negative impacts on efficacy and survival. Acute CIPN occurs during chemotherapy treatment while chronic CIPN can last months to years. No FDA- approved treatment currently exists for CIPN. A new treatment or preventative intervention for CIPN holds promise to not only address debilitating pain, but also serve as an enabler of essential anti-cancer therapy.

About Artelo Biosciences

Artelo Biosciences, Inc. is a clinical-stage pharmaceutical company dedicated to the development and commercialization of proprietary therapeutics that modulate lipid-signaling pathways. Artelo is advancing a portfolio of broadly applicable product candidates designed to address significant unmet needs in multiple diseases and conditions, including anorexia, cancer, anxiety, dermatologic conditions, pain, and inflammation. Led by proven biopharmaceutical executives collaborating with highly respected researchers and technology experts, the Company applies leading-edge scientific, regulatory, and commercial discipline to develop high-impact therapies. More information is available at www.artelobio.com and Twitter: @ArteloBio.

Forward Looking Statements

This press release contains certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934 and Private Securities Litigation Reform Act, as amended, including those relating to the Company’s product development, clinical and regulatory timelines, market opportunity, competitive position, possible or assumed future results of operations, business strategies, potential growth opportunities and other statement that are predictive in nature. These forward-looking statements are based on current expectations, estimates, forecasts and projections about the industry and markets in which we operate and management’s current beliefs and assumptions. These statements may be identified by the use of forward-looking expressions, including, but not limited to, “expect,” “anticipate,” “intend,” “plan,” “believe,” “estimate,” “potential,” “predict,” “project,” “should,” “would” and similar expressions and the negatives of those terms. These statements relate to future events or our financial performance and involve known and unknown risks, uncertainties, and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Such factors include those set forth in the Company’s filings with the Securities and Exchange Commission, including our ability to raise additional capital in the future. Prospective investors are cautioned not to place undue reliance on such forward-looking statements, which speak only as of the date of this press release. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise, except to the extent required by applicable securities laws.

Investor Relations Contact:
Crescendo Communications, LLC
Tel: 212-671-1020
Email: ARTL@crescendo-ir.com