Category Archives: Biotech Companies CNS Neuroscience

Coave Therapeutics Launches coAAV-CSF-01, a Novel CNS-Targeted Gene Therapy Vector for Neurodegenerative and other CNS Diseases

Posted on by

New breakthrough data from studies with Coave’s ALIGATER™-engineered AAV2-based capsid coAAV-CSF-01 (S01coAAV2) demonstrates improved brain tissue transduction and safety following intra-cerebrospinal fluid administration in non-human primates

Data selected for Late-Breaking Abstract at ASGCT 2025

Paris, France – May 9, 2025 – Coave Therapeutics (“Coave”), a company pioneering the future of genetic medicines, today announced the launch of coAAV-CSF-01, a novel CNS-targeted gene therapy capsid developed using its proprietary ALIGATER™ platform. New breakthrough data from studies evaluating the vector (research code S01coAAV2) have been selected for presentation as a Late-Breaking Abstract at the 2025 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, to be held May 13-17, in New Orleans, LA, USA.

The data highlight the therapeutic potential of coAAV-CSF-01 (S01coAAV2) to transform central nervous system (CNS) gene therapy delivery. In non-human primate (NHP) studies, coAAV-CSF-01 was administered via intra-cerebrospinal fluid (intra-CSF) routes, including intracisternal magna (ICM) and intracerebroventricular (ICV). The results demonstrated higher transgene expression in targeted brain regions and a better safety profile using coAAV-CSF-01 compared to AAV9.

Key findings with coAAV-CSF-01 include:

  • 100-fold higher transgene expression in the cortex and 10,000-fold increase in the hippocampus compared to AAV9 at the same dose
  • Comparable CNS biodistribution and expression at one-fifth the dose of AAV9
  • Significantly reduced peripheral transduction (including strong liver de-targeting) and peripheral nerve safety, addressing key safety concerns in CNS gene therapy

“These exciting new data from coAAV-CSF-01 represent a major step forward in CNS gene therapy,” said Lolita Petit, CSO of Coave Therapeutics. “In non-human primates, the vector achieved robust brain transduction via intra-CSF delivery – a route long viewed as promising but historically constrained by limited efficacy and off-target effects. The enhanced biodistribution and improved safety demonstrated in these studies support its potential in developing new genetic medicines for neurodegenerative and neurodevelopmental CNS disorders.

“Furthermore, the vector was developed using our proprietary ALIGATER™ platform, enabling modular modification of AAV2 and other capsids to optimize tissue targeting and therapeutic performance – potentially opening the door to broader therapeutic applications. Together, the data position coAAV-CSF-01 as a potentially transformative advance in gene therapy delivery.”

coAAV-CSF-01 is part of Coave’s growing portfolio of proprietary AAV capsids engineered to optimize gene delivery in challenging therapeutic areas. The ALIGATER™ platform enables chemical conjugation-based modification of AAV vectors (coAAVs), offering a modular and scalable approach to enhance tissue targeting and transduction efficiency.

Late-Breaking Poster Presentation Details:

  • Title: Enhanced CNS transduction and safety of S01coAAV2 (coAAV-CSF-01) following intra-cerebrospinal fluid (CSF) administration in cynomolgus macaques
  • Session: Late-Breaking Abstracts
  • Date/Time: Thursday May 15, 2025, 4:30 – 6pm CDT
  • Location: New Orleans
  • Presenter: Julien Spatazza, Senior Director, Discovery & Preclinical Research at Coave Therapeutics

Coave’s abstract (#LBA78) can be viewed on ASGCT’s website (https://annualmeeting.asgct.org/).

***

About ALIGATER™
Coave’s proprietary ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform is a breakthrough technology addressing key limitations in the delivery of genetic payloads to extra-hepatic tissues, including limited tissue specificity, delivery efficiency and safety. ALIGATER™ enables conjugation of targeting ligands, such as small molecules, peptides, or antibody fragments, on AAV or non-viral vectors, offering superior delivery efficiency, tissue specificity and safety profile for a broad range of diseases. Importantly, the platform streamlines the manufacturing process by avoiding prior AAV capsid modifications. These capabilities will enable Coave to develop best-in-class gene therapies designed for specific indications.

About Coave Therapeutics
Coave Therapeutics is a genetic medicine company pioneering the development of innovative solutions to enhance the precision, safety, efficacy and manufacturability of genetic medicines. With its proprietary ALIGATER™ platform, Coave is at the forefront of addressing challenges in gene therapy delivery to extra-hepatic tissues, creating a robust pipeline targeting CNS, neuromuscular and eye diseases.

Headquartered in Paris, France, Coave Therapeutics is backed by leading international life sciences investors. For more information about the science, pipeline, and people, please visit coavetx.com and follow us on LinkedIn.

CONTACTS

Coave Therapeutics
Rodolphe Clerval, CEO
contact@coavetx.com

MEDiSTRAVA
Sylvie Berrebi, Mark Swallow
coavetx@medistrava.com

Serina Therapeutics Secures $10 Million Financing to Continue Advancing Lead IND Candidate into Phase 1 Clinical Trial in Advanced Parkinson’s Disease Patients

Posted on by

HUNTSVILLE, Dec. 02, 2024 (GLOBE NEWSWIRE) — Serina Therapeutics, Inc. (“Serina”) (NYSE American: SER), a clinical-stage biotechnology company, today announced a $10 million equity financing with strategic shareholder JuvVentures (UK) Limited. The transaction provides Serina with funding to continue advancing SER-252 (POZ-apomorphine), enabled by its proprietary POZ Platform™ drug optimization technology, into a Phase 1 clinical trial in advanced Parkinson’s disease patients in the second half of 2025.

Under the terms of the funding agreement, Serina will issue one million shares of common stock at $10 per share, a 120% premium to the closing price on November 26, 2024. The financing will be delivered in two tranches: the first $5.0 million tranche was received November 27, 2024, and the second $5.0 million tranche by January 31, 2025. Serina filed a Form 8-K with the SEC on December 2, 2024 that provides additional information regarding this transaction.

About SER-252 (POZ-apomorphine)

SER 252 is an investigational apomorphine therapy developed with Serina’s POZ platform and designed to provide continuous dopaminergic stimulation (CDS). CDS has been shown to reduce the severity of levodopa-related motor complications (dyskinesia) and enable greater on time, with reduced off time, in advanced Parkinson’s patients. SER-252 leverages strategic partner Enable Injections’ enFuse™ wearable drug delivery platform to enhance patient comfort and convenience, providing CDS to patients via an easy-to-administer, long-acting subcutaneous injection without skin reactions.

About the POZ Platform™

Serina’s proprietary POZ technology is based on a synthetic, water soluble, low viscosity polymer called poly(2-oxazoline). Serina’s POZ technology is engineered to provide greater control in drug loading and more precision in the rate of release of attached drugs delivered via subcutaneous injection. The therapeutic agents in Serina’s product candidates are typically well-understood and marketed drugs that are effective but are limited by pharmacokinetic profiles that can include toxicity, side effects and short half-life. Serina believes that by using POZ technology, drugs with narrow therapeutic windows can be designed to maintain more desirable and stable levels in the blood.

Serina’s POZ platform delivery technology has potential for use across a broad range of payloads and indications. Serina intends to advance additional applications of the POZ platform via out-licensing, co-development, or other partnership arrangements, including the non-exclusive license agreement with Pfizer, Inc. to use Serina’s POZ polymer technology for use in lipid nanoparticle drug (LNP) delivery formulations.

About Serina Therapeutics

Serina is a clinical-stage biotechnology company developing a pipeline of wholly owned drug product candidates to treat neurological diseases and other indications. Serina’s POZ PlatformTM provides the potential to improve the integrated efficacy and safety profile of multiple modalities including small molecules, RNA-based therapeutics and antibody-based drug conjugates (ADCs). Serina is headquartered in Huntsville, Alabama on the campus of the HudsonAlpha Institute of Biotechnology.

For more information, please visit https://serinatherapeutics.com.

Cautionary Statement Regarding Forward-Looking Statement

This release contains forward-looking statements within the meaning of federal securities laws. These statements are based on management’s current expectations, plans, beliefs or forecasts for the future, and are subject to uncertainty and changes in circumstances. Any express or implied statements in this press release that are not statements of historical fact, including statements about the potential of Serina’s POZ polymer technology, are forward-looking statements that involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any applications may be filed for any drug or vaccine candidates in any jurisdictions; whether and when regulatory authorities may approve any potential applications that may be filed for any drug or vaccine candidates in any jurisdictions, which will depend on a myriad of factors, including making a determination as to whether the product’s benefits outweigh its known risks and determination of the product’s efficacy and, if approved, whether any such drug or vaccine candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of any drug or vaccine candidates; and competitive developments. These risks as well as other risks are more fully discussed in the company’s Annual Report on Form 10-K for the year ended December 31, 2023, the company’s Current Report on Form 8-K that was filed with the SEC on April 1, 2024, and the company’s other periodic reports and documents filed from time to time with the SEC.

The information contained in this release is as of the date hereof, and Serina assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

For inquiries, please contact:

Investor.relations@serinatherapeutics.com
(256) 327-9630

**************************************************************************************************

More info for healthcare startups : Healthcare Investors Database and Healthcare Investors Matchmaking .

 

 

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

Posted on by

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

Seaport Therapeutics Closes $225 Million Oversubscribed Series B Financing Round

Posted on by

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

Posted on by
  • 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

Booster Therapeutics Launches to Pioneer New Class of Proteasome Activator Medicines for the Treatment of a Range of Complex Diseases

Posted on by
  • $15 million seed financing led by Apollo Health Ventures and Novo Holdings
  • New approach aims to expand the ability of therapeutics to degrade harmful proteins in the body
  • Booster small molecules re-activate cellular ‘quality control’ machinery of proteasomes, which is impaired in complex diseases
  • Dr. Patrick Trojer appointed as Chair of the Board of Directors

BERLIN–(BUSINESS WIRE)–Booster Therapeutics, a biotechnology company pioneering a new class of proteasome activator medicines to treat neurodegenerative and other diseases, launched today with the support of a $15 million financing led by noted life sciences investors Apollo Health Ventures and Novo Holdings. Booster was founded by Dr. Diogo Feleciano, Prof. Dr. Darci Trader, and Apollo Health Ventures, emerging from Apollo’s company creation investment strategy. The company is developing small molecule therapeutics that boost the innate activity of proteasomes to restore the body’s ability to remove a wide range of disease-causing proteins.


“Proteasome activation offers important advantages over more limited conventional protein degradation approaches and provides a powerful lever to combat the effects of the many types of deviant proteins that can accumulate in cells when proteasome function declines through age or disease. The implications for solving major degenerative conditions, such as Parkinson’s and Alzheimer’s, are enormous,” said Dr. Diogo Feleciano, Co-Founder and Chief Scientific Officer. “We’re grateful for the support of our investors, who have enabled us to assemble a world-class team to realize the untapped potential of proteasome activation against a range of difficult-to-treat diseases.”

Proteasomes, the cell’s natural quality control machinery, play a critical role in removing damaged or misfolded proteins. When their function is impaired, misfolded proteins accumulate and increase the risk of serious disease. Booster’s approach is a departure from current targeted protein degradation methods, which tag single disease proteins with the marker protein ubiquitin, leading to their degradation via 26S proteasomes. This can be effective, particularly in diseases driven by a single errant protein. But complex diseases are often driven by multiple protein dysfunctions. To achieve more widespread degradation of unwanted proteins, Booster’s compounds directly activate 20S proteasomes, which naturally recognize disordered proteins without the need for ubiquitin tagging.

“Booster exploits the cell’s universal protein surveillance machinery to drive restoration of protein homeostasis in various disease contexts,” said Dr. Patrick Trojer, Chair of Booster’s Board of Directors and Chief Executive Officer of TRIANA Biomedicines. “The application potential of this therapeutic frontier is highly attractive, and given the broad-spectrum activity of proteasome activators, we see opportunities for both monotherapy and combination approaches where increased proteasome activity may enhance the efficacy of other therapeutics.”

Booster is discovering small molecules through its DGRADX™ platform, which combines proprietary methods for automated high-throughput screening with advanced structural and computational tools. The company has built an extensive library of activator compounds with therapeutic potential and aims to develop a multi-disease pipeline to address proteinopathies.

“Emerging from Apollo’s company creation engine, Booster has made remarkable strides in pioneering this exciting new field of medicine. Booster’s focus on activating the 20S proteasome directly addresses a critical mechanism involved in the cellular stress of aging, offering us a new therapeutic perspective on major disease areas,” said Dr. Marianne Mertens, Partner at Apollo, Managing Director, and Board Member of Booster Therapeutics. “We look forward to working with the team and the strong syndicate to advance toward proof-of-concept and develop a pipeline of potential first-in-class medicines.”

“The pioneering work of Prof. Trader and Dr. Feleciano completely transformed our approach to protein degradation in proteinopathies. Booster’s data support the ability of the 20S proteasome to safely, effectively and specifically degrade a wider set of pathological proteins.” said Dr. João Ribas, Principal at Novo Holdings, Seed Investments and Interim Chief Business Officer and Board Member, Booster Therapeutics. “Proteinopathy is core to many of the most challenging complex diseases, so the potential to address them with a single agent is incredibly promising and could reshape the treatment landscape. We’re excited to join Apollo and help advance this novel therapeutic concept to the clinic.”

About Booster Therapeutics

Booster Therapeutics is pioneering a new class of medicines that activate the cell’s natural quality control machinery to treat a range of complex indications. Booster’s small molecule therapeutics, discovered through the DGRADX™ platform, are designed to directly boost the activity of 20S proteasomes to restore the body’s ability to clear disease-causing proteins. The company is developing a multi-disease pipeline, with an initial focus on neurodegenerative diseases associated with impaired proteasome function such as Parkinson’s disease and Alzheimer’s disease. Booster is based in Berlin, Germany. For more information, visit www.boostertx.com.

About Apollo Health Ventures

Apollo Health Ventures is a transatlantic venture capital firm specialized in developing and investing in data-driven biotechnology and health tech ventures. Apollo Health Ventures invests in game-changing companies at the seed or early stage and builds companies within the aging sector. Apollo’s team consists of entrepreneurs, seasoned biotech investors and scientists with remarkable track records in life science investments and venture creation. www.apollo.vc

About Novo Holdings

Novo Holdings is a holding and investment company that is responsible for managing the assets and the wealth of the Novo Nordisk Foundation. The purpose of Novo Holdings is to improve people’s health and the sustainability of society and the planet by generating attractive long-term returns on the assets of the Novo Nordisk Foundation.

Wholly owned by the Novo Nordisk Foundation, Novo Holdings is the controlling shareholder of Novo Nordisk A/S and Novonesis A/S and manages an investment portfolio with a long-term return perspective. Novo Holdings is a world-leading life sciences investor. Through its Seed, Venture, Growth, Principal Investments, Planetary Health Investments and Asia teams, Novo Holdings invests directly in life science companies at all stages of development. In addition, it manages a broad portfolio of Capital Investments, including equities, bonds, fixed income, real estate, and infrastructure assets. As of year-end 2023, Novo Holdings had total assets of EUR 149 billion. www.novoholdings.dk

Contacts

Kit Rodophele

Ten Bridge Communications

617-999-9620

krodophele@tenbridgecommunications.com

Tiziana Life Sciences Announces Six-Month Qualitative Improvement in Neuroimaging in 80% of Multiple Sclerosis Patients Receiving Intranasal Foralumab

Posted on by
  • Qualitative improvements in PET imaging seen in 80% of non-active Secondary Progressive Multiple Sclerosis (na-SPMS) Expanded Access patients receiving intranasal foralumab for at least 6-months.
  • FDA Allowance for an additional 20 Patients to be enrolled in the intranasal foralumab Multiple Sclerosis Expanded Access Program will allow further data collection and analysis.
  • Applied for FDA Orphan Drug Designation of foralumab for na-SPMS

NEW YORK, June 06, 2024 (GLOBE NEWSWIRE) — Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough immunomodulation therapies via novel routes of drug delivery, today announced the qualitative results for all 10 non-active Secondary Progressive Multiple Sclerosis (na-SPMS) patients enrolled in the intermediate-size patient population Expanded Access (EA) Program receiving foralumab for at least six months.

Tarun Singhal, M.B.B.S., M.D., Director of the PET Imaging Program in Neurologic Diseases, associate neurologist and nuclear medicine physician at Brigham and Women’s Hospital, a founding member of Mass General Brigham Healthcare System, and Associate Professor of Neurology at Harvard Medical School, commented, “Based on currently available data from the latest cohort of four Expanded Access patients, three out of the four subjects had findings that suggest a qualitative reduction in the microglial PET signal over a period of six months of treatment with nasal foralumab. When combined with my assessment of the first six Expanded Access patients at six months, eight of the ten suggest a qualitative reduction in microglial PET signal. Further studies are needed to confirm these findings using additional cases and quantitative approaches.”

Gabriele Cerrone, Chairman, acting CEO, and founder of Tiziana Life Sciences, added, “I am thrilled that 80% of the na-SPMS patients who received intranasal foralumab treatment for at least 6-months have a qualitative reduction of microglial activity as confirmed in these latest PET images. I am also greatly appreciative of Dr. Singhal’s research and look forward to the additional quantitative analysis of the data. With the allowance of an additional 20 patients in the EA program, the application for Orphan Drug Designation for na-SPMS, and the ongoing Phase 2a trial, Tiziana is rapidly progressing its intranasal foralumab program in multiple sclerosis.”

About Foralumab

Activated T cells play an important role in the inflammatory process. Foralumab, the only fully human anti-CD3 monoclonal antibody (mAb), binds to the T cell receptor and dampens inflammation by modulating T cell function, thereby suppressing effector features in multiple immune cell subsets. This effect has been demonstrated in patients with COVID and with multiple sclerosis, as well as in healthy normal subjects. The non-active SPMS intranasal foralumab Phase 2 trial (NCT06292923) began screening patients in November of 2023. Immunomodulation by nasal anti-CD3 mAb represents a novel avenue for treatment of neuroinflammatory and neurodegenerative human diseases.[1],[2]

About Tiziana Life Sciences

Tiziana Life Sciences is a clinical-stage biopharmaceutical company developing breakthrough therapies using transformational drug delivery technologies to enable alternative routes of immunotherapy. Tiziana’s innovative nasal approach has the potential to provide an improvement in efficacy as well as safety and tolerability compared to intravenous (IV) delivery. Tiziana’s lead candidate, intranasal foralumab, which is the only fully human anti-CD3 mAb, has demonstrated a favorable safety profile and clinical response in patients in studies to date. Tiziana’s technology for alternative routes of immunotherapy has been patented with several applications pending and is expected to allow for broad pipeline applications.

For further inquiries:

Tiziana Life Sciences Ltd
Paul Spencer, Business Development and Investor Relations
+44 (0) 207 495 2379
email: info@tizianalifesciences.com

Investors:
Irina Koffler
LifeSci Advisors, LLC
646.970.4681
ikoffler@lifesciadvisors.com

[1] https://www.pnas.org/doi/10.1073/pnas.2220272120
[2] https://www.pnas.org/doi/10.1073/pnas.2309221120

Sinaptica Therapeutics Selected for StartUp Health’s Alzheimer’s Moonshot

Posted on by

Moonshot supported by Gates Ventures and Alzheimer’s Drug Discovery Foundation (ADDF) to accelerate innovation around Alzheimer’s

Sinaptica is one of 14 companies initially selected that are advancing innovative solutions for Alzheimer’s to commercialization and scalability

Collaboration will support the company’s Phase 3 clinical development plans based on unprecedented Phase 2 data in Alzheimer’s showing >80% disease slowing on cognitive & functional endpoints

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sinaptica Therapeutics, Inc., a clinical-stage company leading the development of a new class of personalized neuromodulation therapeutics to treat Alzheimer’s (AD) and other neurodegenerative diseases, today announced that it has been selected to join StartUp Health’s Alzheimer’s Moonshot. This bold new initiative and global community of founders and funders collaborating to prevent, diagnose, manage, and ultimately cure Alzheimer’s disease is supported by the Alzheimer’s Drug Discovery Foundation (ADDF) and Gates Ventures.

The collaboration will help support the global development of Sinaptica’s patented neuromodulation therapy, which takes a precision medicine approach targeting the Default Mode Network in the brain. The company has prioritized mild/moderate Alzheimer’s Disease as its first indication and is preparing for a pivotal Phase 3 trial in 2025.

“Sinaptica is honored to be included in this hand-selected group of innovators working to end Alzheimer’s – a goal which we believe will require combinatorial approaches,” said Sinaptica CEO Ken Mariash. “Sinaptica brings a bold new personalized neuromodulation approach that can be combined with virtually any drug, given its near-total lack of side effects. Also, we know diagnostics are a key piece of the puzzle and will look to combine our novel electrophysiological biomarker with those of others in the Moonshot, to better characterize the disease and bring a more personalized, precision medicine approach to Alzheimer’s treatments.”

“We’re excited to welcome Sinaptica Therapeutics to the Alzheimer’s Moonshot and the StartUp Health family,” said Steven Krein, CEO and co-founder of StartUp Health. “We created the Alzheimer’s Moonshot to bring together trailblazers, support new approaches and rapidly accelerate progress in this challenging disease. Sinaptica is a perfect fit and we look forward to partnering with them.”

Sinaptica is building on unprecedented positive Phase 2 data in Alzheimer’s published by the company’s scientific co-founders, showing greater than 80% disease slowing on all four gold-standard AD cognitive and functional clinical endpoints, in the peer-reviewed Oxford University Press journal, Brain. Additional exciting 52-week data from the Phase 2 study will also be published in an upcoming issue of a peer-reviewed journal. Sinaptica’s technology has been granted FDA Breakthrough Device Designation, and the company is preparing for a larger Phase 3 clinical trial in Alzheimer s patients at multiple sites.

About StartUp Health

Since 2011, StartUp Health has been on a mission to solve the biggest health challenges of our time by creating and sustaining a global ecosystem of health moonshot communities. StartUp Health has provided support to more than 1,000 founders and contributed to the development of more than 500 health innovation companies. Our valued partners include The Helmsley Charitable Trust, Alzheimer’s Drug Discovery Foundation (ADDF) and Gates Ventures in addition to nearly 100 families and mission-aligned organizations. Visit startuphealth.com.

About Sinaptica Therapeutics

Sinaptica Therapeutics is a clinical-stage neuromodulation therapeutics company leading the development of a new class of personalized therapeutics to revolutionize the treatment of Alzheimer’s and neurodegenerative diseases. The company utilizes a patented novel, non-invasive approach to treating Alzheimer’s via precision neurostimulation of a key brain network involved in memory, the Default Mode Network. This novel approach slowed disease progression by >80% on all four gold-standard cognitive and functional clinical endpoints in a placebo-controlled Phase 2 clinical study, with results published in the peer-reviewed Oxford University Press journal, Brain. The technology was granted Breakthrough Device Designation by the FDA in 2022 and the company is preparing for a pivotal randomized controlled clinical trial in 2025. Sinaptica’s mission is to bring a safe, effective, and non-invasive neuromodulation therapy to Alzheimer’s patients that can help to significantly slow the progression of both cognitive and functional decline. Learn more at sinapticatx.com and follow us on LinkedIn and X @SinapticaTX.

The SinaptiStim™ System is for investigational use only. It has not been approved by the U.S. Food and Drug Administration and is not available for commercial sale in any geography.

Contacts

Kira Gordon, BrightPoint

kira@brightpointny.com
646-243-4920

Progentos Therapeutics Closes $65 Million Series A Financing to Advance Treatments for Multiple Sclerosis and Other Degenerative Diseases

Posted on by

Founded by Chief Executive Officer Chris Loose, Ph.D. and Chief Scientific Officer, Sanjay Magavi, Ph.D., Progentos to Drive Clinical Development of Novel Therapeutics for Remyelination

Financing Led by Forbion with other Leading Life Science Investors including Alta Partners, Mission BioCapital, Longwood Fund, and Dolby Family Ventures

WATERTOWN, Mass.–(BUSINESS WIRE)–Progentos Therapeutics, a biotech company addressing the critical unmet need to regenerate myelin and restore function for patients with Multiple Sclerosis (MS) and other demyelinating diseases, today announced its launch and the closing of a $65 million series A round. This funding will enable Progentos to advance its MS program through human proof of concept studies and expand its pipeline in additional degenerative diseases.

Progentos is developing first-in-class small molecules designed to induce remyelination of axons affected by MS. In MS, disability is caused by demyelination, damage to the myelin sheaths that support the function and survival of axons. The Company’s proprietary molecules outperform previous approaches in differentiating oligodendrocyte progenitor cells (OPCs) to generate new oligodendrocytes and regenerate myelin in in vivo models.

“While there are many treatments that are highly effective at slowing the progression of disease, there is a significant unmet need for new approaches that can regenerate myelin and restore function for patients with MS,” said Dr. Chris Loose, CEO of Progentos. “I am thrilled with the support received from this group of investors and appreciate their recognition of the need to advance the current standard of care for the millions of individuals impacted by MS and other demyelinating diseases.”

The series A financing was led by Netherlands-based Forbion which was joined by Alta Partners, Mission BioCapital, Longwood Fund, and Dolby Family Ventures.

“We are truly excited to be part of Progentos’ journey towards delivering potentially disease-modifying drugs for diseases like multiple sclerosis, diseases with high unmet clinical needs. Chris and Sanjay are bringing a high level of technical expertise, entrepreneurship, and leadership and we are honored to support them in this endeavor,” said Forbion General Partner Dmitrij Hristodorov, Ph.D.

About Progentos Therapeutics

Progentos is developing first-in-class compounds to induce endogenous oligodendrocyte progenitor cells to remyelinate axons in patients suffering from MS and other demyelinating diseases. Combining expertise in chemistry, biology and in vivo models, Progentos discovers and develops novel small-molecule drugs to regenerate tissues in patients with degenerative diseases. The Company will have operations in Watertown, MA, USA and Naarden, The Netherlands. More information can be found at www.progentos.com

Contacts

Jason Glashow

Glashow Strategic Communications for Progentos Therapeutics

jason@glashowstrategic.com

4M Therapeutics : Novel Preclinical Data Demonstrating Potent Effects of Inhibitors of GSK3β for the Treatment of Bipolar Disorder

Posted on by

– GSK3 inhibitors showed successful CNS target engagement and efficacy comparable to lithium at significantly lower doses in models of bipolar disorder

SKILLMAN, N.J.–(BUSINESS WIRE)–4M Therapeutics Inc., an early-stage biotechnology company developing treatments for neuropsychiatric and neurodegenerative conditions, today announced the presentation of a poster at the Society of Biological Psychiatry (SOBP) Annual Meeting, that occurred from May 9-11, 2024, in Austin, Texas. The poster, titled “The Effects of Novel Small Molecules in a Preclinical Model of Positive Affective State,” was presented on May 11, 2024.

“Our presentation underscored the capability of our novel small molecules to selectively target GSK3β and demonstrate efficacy for the treatment of bipolar disorder and acute mania,” said Dr. Pablo Lapuerta, Chief Executive Officer and Co-Founder of 4M Therapeutics. “We continue to accelerate the development of much-needed therapeutic options that have the potential to significantly improve upon the safety and efficacy of the standard of care for patients with neuropsychiatric conditions. We remain on track to initiate a Phase 1 clinical trial for our lead investigational compound in 2025.”

The poster examined the effects of 4M Therapeutics’ glycogen synthase kinase 3 beta (GSK3β) inhibitors, 4MT-A and 4MT-B, on amphetamine (AMP)-induced positive appetitive ultrasonic vocalizations (USVs), a validated model of positive affective state. Marked increases in USVs when treated with AMP were attenuated by lithium, and equal or greater reduction in USVs was observed when treated with 4MT-A and 4MT-B at 1/500th to 1/1000th the concentration of lithium. Similar patterns were observed across USV categories and treatment groups in both the first and second hour after AMP dosing. In addition, supporting data showed target engagement for 4MT-A through the reduction of pCRMP2 in vitro in excitatory cortical neurons and in vivo in rat brain. These statistically significant reductions in pCRMP2 demonstrated GSK3β inhibition affecting a key biomarker.

About 4M Therapeutics Inc.

4M Therapeutics Inc. (4MTx) is advancing treatments for neuropsychiatric and neurodegenerative conditions. The Company focuses on targets for a wide array of disorders and indications. 4MTx applies unique insights from its living human brain cell platform, which was developed through a collaboration between Harvard, MIT, and the University of Washington to identify and design more effective and safer therapeutics. The Company’s pipeline includes potential breakthrough treatments for bipolar mania, agitation in Alzheimer’s disease, neurodegeneration, and other CNS disorders. For more information, visit www.4mtx.net.

Contacts

For investor and media inquiries, please contact:
Kimberly Lee, DO

Chief Business Officer

ir@4mtx.net