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

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

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 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.


Kira Gordon, BrightPoint

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

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


Jason Glashow

Glashow Strategic Communications for Progentos Therapeutics

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

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


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

Chief Business Officer

Newron Pharmaceuticals announces positive top-line results from potentially pivotal Phase II/III study 008A with evenamide in schizophrenia patients

Primary endpoint and key secondary endpoint of study met; drug was well tolerated, with no safety concerns identified

Glutamatergic inhibition mechanism of action offers an innovative therapeutic option to schizophrenia patients not benefitting from current antipsychotic treatments

Next step: Potentially pivotal, Phase III, one-year, randomized, double-blind, placebo-controlled trial in treatment resistant schizophrenia (TRS)

Milan, Italy, April 30, 2024, 07:00 am CEST – Newron Pharmaceuticals S.p.A. (“Newron”) (SIX: NWRN, XETRA: NP5), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system (CNS), today announced positive top-line results from its potentially pivotal study 008A, evaluating the safety, tolerability and efficacy of evenamide (30 mg bid) in patients with chronic schizophrenia currently being treated with a second-generation antipsychotic including clozapine, but demonstrating an inadequate response to that treatment. The study met its primary endpoint of improvement on the Positive and Negative Syndrome Scale (PANSS) Total Score as well as the key secondary endpoint of improvement of the Clinical Global Impression of Severity (CGI-S).

Study 008A was a four-week, international, randomized, double-blind and placebo-controlled add-on Phase II/III study performed in 45 centers in 11 countries in Europe, Asia and Latin America. 291 patients were randomized to treatment either with evenamide or placebo as add-on to their current antipsychotic therapy.

Evenamide confirmed its favourable safety and tolerability profile, with a high rate of completion. Two hundred and eighty of the 291 patients completed the study with only three patients discontinuing the study due to adverse events, two of them on evenamide and one patient on placebo who died during the study.  No new or specific concerns were raised in the study; only 25% of the patients in the study experienced at least one adverse event (evenamide 25% versus placebo 25.8%). There was no difference in the incidence of CNS, psychiatric, gastrointestinal or other adverse events between evenamide and placebo. The most common adverse events reported in patients treated with evenamide were headache, vomiting and nasopharyngitis (three patients, each); similar numbers of patients on placebo experienced these adverse events.

In the study, the addition of 30 mg (bid) of evenamide to the patients’ current antipsychotic medication was associated with a highly statistically significant (p-value 0.006) reduction in the PANSS Total Score of 10.2 points, compared to 7.6 points in patients treated with placebo at day 29; the least square mean difference (LS mean difference) was 2.5. For the key secondary measure, the Clinical Global Impression of Severity (CGI-S), the LS mean difference between patients treated with evenamide and placebo was 0.16; the corresponding p-value was 0.037.

Ravi Anand, MD, Chief Medical Officer of Newron, stated: “The results seen in study 008A with evenamide are ground-breaking and unique from many perspectives. This is the first major international study to demonstrate the significant benefit of adding a new chemical entity (NCE) to poorly responding, compliant schizophrenia patients being treated with a second-generation antipsychotic. It is also the first demonstration of efficacy in a placebo-controlled trial of a NCE acting exclusively through glutamatergic inhibition. These results, together with the recently reported one-year efficacy results in treatment-resistant patients, substantiate the pivotal role of glutamate in finding new therapeutic options for schizophrenia patients.”

Additional details from the study will be disclosed in the coming weeks.

Media/analyst/investor Conference Call today at 3 pm CET

Newron’s management team will be available today to discuss the top-line results from study 008A. Please dial in five to ten minutes prior to the beginning of the call using one of the following telephone numbers:

  • Switzerland/Europe: +41 (0)58 310 50 00
  • Germany: +49 (0)69 505 0 0082
  • United Kingdom: +44 (0)207 107 0613
  • United States: +1 (1)631 570 5613
  • Japan: +81 35 050 5361

About evenamide

Evenamide, an orally available new chemical entity, specifically blocks voltage-gated sodium channels (VGSCs) and is devoid of biological activity at >130 other CNS targets. It normalizes glutamate release induced by aberrant sodium channel activity (veratridine-stimulated), without affecting basal glutamate levels, due to inhibition of VGSCs. Combinations of ineffective doses of evenamide and other APs, including clozapine, were associated with benefit in animal models of psychosis, suggesting synergies in mechanisms that may provide benefit in patients who are poor responders to current APs, including clozapine.

About Newron Pharmaceuticals

Newron (SIX: NWRN, XETRA: NP5) is a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system. The Company is headquartered in Bresso near Milan, Italy. Xadago®/safinamide has received marketing authorization for the treatment of Parkinson’s disease in the European Union, Switzerland, the UK, the USA, Australia, Canada, Latin America, Israel, the United Arab Emirates, Japan and South Korea, and is commercialized by Newron’s Partner Zambon. Supernus Pharmaceuticals holds the commercialization rights in the USA. Meiji Seika has the rights to develop and commercialize the compound in Japan and other key Asian territories. Newron is also developing evenamide as the potential first add-on therapy for the treatment of patients with symptoms of schizophrenia. For more information, please visit:

For more information, please contact:

Stefan Weber – CEO
+39 02 6103 46 26

Simon Conway / Ciara Martin / Natalie Garland-Collins, FTI Consulting
+44 20 3727 1000

Valentin Handschin, IRF
+41 43 244 81 54

Anne Hennecke / Caroline Bergmann, MC Services
+49 211 52925222

Paul Sagan, LaVoieHealthScience
+1 617 374 8800, Ext. 112

Important Notices

This document contains forward-looking statements, including (without limitation) about (1) Newron’s ability to develop and expand its business, successfully complete development of its current product candidates, the timing of commencement of various clinical trials and receipt of data and current and future collaborations for the development and commercialization of its product candidates, (2) the market for drugs to treat CNS diseases and pain conditions, (3) Newron’s financial resources, and (4) assumptions underlying any such statements. In some cases, these statements and assumptions can be identified by the fact that they use words such as “will”, “anticipate”, “estimate”, “expect”, “project”, “intend”, “plan”, “believe”, “target”, and other words and terms of similar meaning. All statements, other than historical facts, contained herein regarding Newron’s strategy, goals, plans, future financial position, projected revenues and costs and prospects are forward-looking statements. By their very nature, such statements and assumptions involve inherent risks and uncertainties, both general and specific, and risks exist that predictions, forecasts, projections and other outcomes described, assumed or implied therein will not be achieved. Future events and actual results could differ materially from those set out in, contemplated by or underlying the forward-looking statements due to a number of important factors. These factors include (without limitation) (1) uncertainties in the discovery, development or marketing of products, including without limitation difficulties in enrolling clinical trials, negative results of clinical trials or research projects or unexpected side effects, (2) delay or inability in obtaining regulatory approvals or bringing products to market, (3) future market acceptance of products, (4) loss of or inability to obtain adequate protection for intellectual property rights, (5) inability to raise additional funds, (6) success of existing and entry into future collaborations and licensing agreements, (7) litigation, (8) loss of key executive or other employees, (9) adverse publicity and news coverage, and (10) competition, regulatory, legislative and judicial developments or changes in market and/or overall economic conditions. Newron may not actually achieve the plans, intentions or expectations disclosed in forward-looking statements and assumptions underlying any such statements may prove wrong. Investors should therefore not place undue reliance on them. There can be no assurance that actual results of Newron’s research programs, development activities, commercialization plans, collaborations and operations will not differ materially from the expectations set out in such forward-looking statements or underlying assumptions. Newron does not undertake any obligation to publicly update or revise forward-looking statements except as may be required by applicable regulations of the SIX Swiss Exchange or the Dusseldorf Stock Exchange where the shares of Newron are listed. This document does not contain or constitute an offer or invitation to purchase or subscribe for any securities of Newron and no part of it shall form the basis of or be relied upon in connection with any contract or commitment whatsoever.

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Ventus Therapeutics Announces Results from Phase 1 Clinical Trial of VENT-02, a Novel, Orally Administered, Brain-Penetrant NLRP3 Inhibitor

VENT-02 demonstrated robust pharmacodynamic modulation, a broad therapeutic index, and potential for once-daily dosing

VENT-02 provided full target coverage with a single dose

No dose-limiting toxicities or serious adverse events observed

Initiation of clinical trials in CNS diseases expected beginning in the second half of 2024

WALTHAM, Mass. & MONTREAL–(BUSINESS WIRE)–Ventus Therapeutics, a clinical-stage biopharmaceutical company utilizing its proprietary structural biology and computational chemistry platform, ReSOLVE, to develop differentiated small molecule therapeutics, today announced results from its Phase 1 clinical trial of VENT-02, a novel, oral, brain-penetrant NLRP3 inhibitor. The Phase 1 trial evaluated the pharmacodynamics, pharmacokinetics, safety, and tolerability of VENT-02 across a broad range of single and multiple ascending doses in adult healthy volunteers.

We are pleased with the results of this trial in which VENT-02 demonstrated a broad therapeutic index and complete target coverage, underscoring its potential to treat diseases of the CNS as well as peripheral diseases with high unmet needs,” said Xavier Valencia, M.D., Head of Clinical Development at Ventus. “Our next trial for VENT-02, a Phase 1b trial in patients with Parkinson’s disease, will evaluate multiple doses and dose regimens against a placebo control and assess a collection of markers that map multiple components of inflammation to disease activity. Based on the promising pharmacodynamic and safety data from this Phase 1 trial and our clinical development plans, we believe VENT-02 is poised to demonstrate best-in-class potential.”

In this Phase 1 clinical trial, VENT-02 was well-tolerated, with no dose-limiting toxicities or serious adverse events reported and only mild or moderate treatment-related adverse events observed. The moderate adverse events included headache and nausea and were only observed at a dose multiple times higher than the intended therapeutic doses.

VENT-02 demonstrated full target engagement through 100% inhibition of IL-1ß in the blood in an ex vivo whole blood challenge assay, significant drug levels in the CSF for 24 hours, and robust reduction of inflammatory biomarkers such as hsCRP. Based on the half-life and target coverage observed in the trial, VENT-02 has demonstrated the potential for once-daily dosing.

The excellent properties of VENT-02 are a reflection of Ventus’ industry-leading exploration of NLRP3, including demonstrating proof-of-concept in preclinical studies in multiple disease-relevant models, validating biomarkers, and collaborating with academic institutions and the Michael J. Fox Foundation,” said Ventus President and CEO, Marcelo Bigal, M.D., Ph.D. “As a result, we are ready to bring VENT-02 into Parkinson’s disease and other pre-defined diseases where the CNS plays an important role, capitalizing on innovative trials that we are crafting to be informative in reducing risk in the future steps of clinical development. In addition, we expect that our planned trials will allow us to explore the potential impact of NLRP3 inhibition across multiple patient populations suffering from serious diseases.”

Ventus expects to initiate a Phase 1b trial of VENT-02 in patients with Parkinson’s disease in the second half of 2024 and a Phase 2 trial in patients with treatment-refractory epilepsy in 2025.

About NLRP3

NLRP3 is the best understood member of a family of proteins known as inflammasomes. Inflammasomes are multiprotein complexes that regulate the innate immune system and are involved in intracellular surveillance of danger and pathogen signals that trigger an intense inflammatory response, including the release of IL-1β and IL-18 and the induction of pyroptosis, an inflammatory form of cell death. Therapeutic inhibition of NLRP3 can prevent the formation of the NLRP3 inflammasome, which in turn inhibits the production of IL-1β and IL-18 as well as pyroptosis. Aberrant activation of the NLRP3 inflammasome has been associated with systemic conditions, including fibrotic, dermatological, and rheumatological diseases, and is a key driver of disease pathology in several neurological disorders, including Parkinson’s disease, Alzheimer’s disease, and treatment-refractory epilepsy.

About Ventus Therapeutics

Ventus Therapeutics is a clinical-stage biopharmaceutical company deploying deep protein science expertise and a proprietary computational chemistry platform to develop novel small molecule therapeutics for immunology, inflammation, and neurology disorders. Using its proprietary drug discovery platform, ReSOLVE™, the company screened its first target in 2020, selected three development candidates in 2022, and advanced its two wholly-owned product candidates into the clinic in 2023: VENT-03, a potent, selective, oral cGAS inhibitor in Phase 1, and VENT-02, a potent, brain-penetrant, oral NLRP3 inhibitor in Phase 1. In addition, Ventus has out-licensed VENT-01, a peripherally-restricted NLRP3 inhibitor, to Novo Nordisk A/S. These programs exemplify Ventus’ robust discovery capabilities and focus on differentiated programs for multi-indication immunology and neurology targets. For more information, please visit and engage with Ventus on LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements. These statements involve known and unknown risks, uncertainties, and other important factors that may cause Ventus’ actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “would,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential,” or “continue” or the negative of these terms or other similar expressions. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the timing, progress, and results of Ventus’ clinical trials, the anticipated timing of initiation and completion of trials, the period during which the results of the trials will become available, and Ventus’ development plans. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond Ventus’ control, you should not rely on these forward-looking statements as predictions of future events. Such risks include, but are not limited to, the risk that Ventus is unable to further advance VENT-02 and VENT-03 in clinical development, obtain regulatory approval, and ultimately commercialize VENT-02 and VENT-03, or experience significant delays in doing so; that Ventus will require substantial additional funding to finance its operations; that the development and commercialization of pharmaceutical products is subject to extensive regulation, and the regulatory approval processes of the U.S. Food and Drug Administration and comparable foreign authorities are lengthy, time-consuming, and inherently unpredictable; and that Ventus’ success depends on its, its current and future licensors’, and its exclusive licensees’ ability to obtain, maintain, enforce, and protect its intellectual property and its proprietary technologies. Forward-looking statements included herein are based upon information available to Ventus as of the date of this press release, and while Ventus believes such information forms a reasonable basis for such statements, such information may be limited or incomplete, and Ventus’ statements should not be read to indicate that it has conducted an exhaustive inquiry into, or review of, all potentially available relevant information. The events and circumstances reflected in the forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable law, Ventus does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances, or otherwise.


Dan Budwick


Astrocyte Pharmaceuticals Announces FDA Clearance of its Investigational New Drug Application for AST-004

Green Light for Phase 2 Trial of AST-004 in Acute Ischemic Stroke Patients

GROTON, Conn., March 12, 2024 (GLOBE NEWSWIRE) — Astrocyte Pharmaceuticals Inc., a clinical-stage biopharmaceutical company committed to advancing cerebroprotective therapies for individuals suffering from stroke, traumatic brain injury (TBI), and concussion, has announced today the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for its lead program, AST-004, for acute ischemic stroke. The company is preparing to initiate a Phase 2 clinical trial. The IND application clearance also paves the way for additional studies further exploring AST-004 in stroke, as well as TBI, and concussion.

Currently, only ~5% of stroke patients receive pharmaceutical therapy, largely due to the short treatment windows of available thrombolytic medicines, as well as their need for imaging before those treatments can be initiated. AST-004 was developed with the potential to treat all stroke patients. Unlike existing therapies, treatment with AST-004 is designed for use without the need for imaging in the emergency room and for use at any point after diagnosis. And it has the potential to treat stroke occurring in any sized blood vessel in the brain.

The upcoming Phase 2 trial in acute ischemic stroke patients builds upon the recent successful stroke clinical trial designs used by medical device companies, heralding a new era in patient care. “The past decade has witnessed a transformative shift in stroke trials, thanks to sophisticated imaging techniques that enable high-quality real-time characterization of stroke and ensure selection of homogeneous study groups,” said Dr. Kevin Sheth, Co-Director of the Center for Brain & Mind Health at the Yale School of Medicine and Chief Medical Advisor to Astrocyte. “The new study of AST-004 will first focus on treating those stroke patients with large vessel occlusions, offering hope to significantly diminish brain damage.”

AST-004 is an innovative small molecule therapy that has demonstrated exceptional promise in preclinical studies. In a critical non-human primate stroke model, a significant reduction in brain lesion growth by 64% and an overall lesion size decrease by 45% were observed with a high dose (as reported in ‘The Journal Stroke’ in 2022). Astrocyte previously completed two Phase 1 safety trials in Europe, involving 80 participants in total, and saw no significant adverse effects or safety concerns.

Dr. William Korinek, CEO of Astrocyte Pharmaceuticals, commented, “Since our founding in 2014, we have been focused on demonstrating the potential of cerebroprotective benefits of AST-004. Our robust preclinical program and Phase 1 studies have laid the foundation for our ongoing development efforts, and we are eager to move forward with the planned Phase 2 study. Stroke is the second leading cause of death globally, and the need for advances in treatment is enormous. We believe AST-004 can be part of that solution.”

About Astrocyte Pharmaceuticals Inc.

Astrocyte Pharmaceuticals Inc. is a privately held, clinical-stage drug discovery and development company dedicated to accelerating the recovery and well-being of brain injury patients. The company is committed to proving the cerebroprotective benefits of enhancing astrocyte function and advancing breakthrough therapeutic agents for treating brain injury resulting from stroke, traumatic brain injury, concussion, and neurodegenerative disorders such as Alzheimer’s disease. For more information on Astrocyte Pharmaceuticals Inc. and the AST-004 program, please visit us at Astrocyte Pharmaceuticals Inc.  

Forward-Looking Statement

This press release contains certain forward-looking statements regarding, among other things, statements relating to goals, plans, and projections regarding the company’s financial position, results of operations, market position, product development, and business strategy. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert, or change any of them and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statements can be guaranteed, and actual results may differ materially from such statements. The information in this release is provided only as of the date of this release, and the company undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law.

Media contact:
Kimberly Macleod – 

Seismic Therapeutic – Preclinical Data for Novel Immunoglobulin Sculpting Enzyme

WATERTOWN, Mass.–(BUSINESS WIRE)–Seismic Therapeutic, Inc., the machine learning immunology company, today announced that the company will present preclinical data for its pan-immunoglobulin G (IgG) sculpting enzyme candidate, S-1117, at the American Academy of Neurology (AAN) annual meeting taking place on April 13-18 in Denver.

S-1117 is a novel engineered pan-IgG protease for the potential treatment of a range of chronic and acute autoantibody mediated diseases, including the chronic neuromuscular autoimmune disorder, myasthenia gravis. The presentation at the AAN annual meeting will outline key preclinical in vitro and in vivo results supporting the differentiated profile of S-1117 compared to therapeutic benchmarks in chronic autoantibody mediated diseases. S-1117 addresses multiple mechanisms of autoimmunity by lowering IgG and immune complex levels, reducing IgG effector functions and cleaving the antigen receptor (BCR) on memory B cells. Moreover, in a mouse pharmacokinetic model, S-1117 was able to achieve deep, sustained and rapid reduction in human IgG.

Presentation details:

Title: Preclinical Pharmacology of S-1117, a Novel Engineered Fc-fused IgG Cleaving Enzyme, for Chronic Treatment of Autoantibody-mediated Diseases

Session: P4 – Autoimmune Neurology: Peripheral Autoimmunity

Date: Monday, April 15, 2024

Time: 11:45 a.m. – 12:45 p.m. MT

Area: 14

Poster number: 018

Abstract number: 6869

About Seismic Therapeutic

Seismic Therapeutic™ is a biotechnology company making a major shift in how immunology therapies are discovered and developed, enabled by machine learning. The company has a growing preclinical stage best-in-class and first-in-class biologics pipeline, derived from its integrated IMPACT platform, to control dysregulated adaptive immunity and address multiple autoimmune diseases. The company is backed by a strong syndicate of life sciences investors and is located in the Boston biotechnology hub. For more information, please visit and follow us on LinkedIn and on X @Seismic_Tx.


Media Contact
Kathryn Morris, The Yates Network


Seismic Therapeutic to Present Preclinical Data for Novel Immunoglobulin Sculpting Enzyme at AAN Annual Meeting

ARMGO Pharma – developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

ARMGO Pharma is a privately held biopharmaceutical company dedicated to applying original, targeted science to the discovery and development of novel small-molecule therapeutics to treat debilitating cardiac, musculoskeletal, and neurological disorders. The company’s proprietary drugs, known as Rycals®, are a new class of oral agents that repair calcium leaks through the Ryanodine Receptor (RyR).

ARMGO Pharma - developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

ARM210 is a potential disease modifying therapy for genetic diseases, which targets the Ryanodine Receptor calcium channel (RyR), an intracellular calcium-release channel that becomes leaky in these and other diseases. Intracellular calcium leaks via mutant RyR1 channels impair muscle contraction leading to muscle weakness and loss of function, and activate toxic pathways that damage muscle, causing the symptoms in RYR1-RM.

ARM210 is a small molecule that binds to leaky RyR channels and repairs the leak, as demonstrated in vitro in muscle biopsies from RYR1-RM patients. The unique mechanism of action of ARM210 makes it an ideal potential disease modifying therapy for RYR1-RM. Muscle biopsies of the patients in this trial have been previously shown to respond biochemically to ARM210 in vitro. This trial will evaluate the safety and explore the biochemical effect of oral administration of ARM210 in these same patients.

ARMGO Pharma has been awarded an exclusive, worldwide license from Columbia University for its RyR technology based on the research of founding scientist Andrew R. Marks, M.D. In December 2021, ARMGO Pharma raises a $35 million Series B  funding round to progress clinical studies of lead molecule ARM210 in cardiac and skeletal muscle diseases.

ARMGO Pharma – developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

More info :

Keywords : ARMGO Pharma , small molecule , therapeutics , RyR technology , ARM210 , cardiovascular , musculoskeletal disorders , neurological disorders , CNS , neuroscience , Rycals , Ryanodine Receptor , RyR , Ryanodine Receptor modulators

Nanostics – micro-flow cytometry to quantify extracellular vesicle size, concentration and marker abundance, with advanced machine learning algorithms to determine disease states

Nanostics is focused on development and commercialization of novel, non-invasive diagnostic tests for cancer. Its core technology is an advanced liquid biopsy platform that can accurately diagnose cancer from a single drop of blood. Its lead product, ClarityDx Prostate, is the most accurate diagnostic test to diagnose aggressive prostate cancer, and is positioned to emerge as the world’s leading diagnostic tool for prostate cancer.

Nanostics - micro-flow cytometry to quantify extracellular vesicle size, concentration and marker abundance, with advanced machine learning algorithms to determine disease states

EVs carrying disease-specific biomarkers like nucleic acid and proteins are continuously released from cells and can be found in biological fluids including blood, urine, semen, and cerebrospinal fluid.  The levels of disease-specific EVs are closely related to disease progression making EVs promising targets for minimally invasive diagnostic assays.

Technology designed to accurately detect and measure EVs has the potential to greatly improve liquid biopsy diagnostics. At Nanostics, we use micro-flow cytometry (µFCM) to quantify EV size, concentration, and marker abundance for millions of EVs in minutes. We then use advanced machine learning to analyze the vast amount of data generated using µFCM to provide rapid and precise results that continuously improve with every test.

The EV and machine learning platform ClarityDX® is set to transform the diagnostic landscape and make easy-to-use, minimally invasive predictive tests a reality in the near future.

At Nanostics, we use micro-flow cytometry (µFCM) to quantify EV size, concentration, and marker abundance for millions of EVs in minutes. We then use advanced machine learning to analyze the vast amount of data generated using µFCM to provide rapid and precise results that continuously improve with every test.

The EV and machine learning platform ClarityDX® is set to transform the diagnostic landscape and make easy-to-use, minimally invasive predictive tests a reality in the near future.

The ability to detect and measure EVs is transforming the diagnostic landscape in immunology, neurology, cardiology, and oncology. The ClarityDX® platform technology is well-positioned for future pipeline products including tests for screening, early diagnosis, complementary diagnosis, monitoring, and management for many diseases and medically-related events. Nanostics’ R&D team continues to expand the product pipeline through in-house and partnered projects to include additional liquid biopsy diagnostic tests to improve patient care.

Nanostics – micro-flow cytometry to quantify extracellular vesicle size, concentration and marker abundance, with advanced machine learning algorithms to determine disease states

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Keywords : machine learning, exosomes, extracellular vesicles, diagnostics, oncology, cancer, prostate cancer, cardiovascular, neuroscience, biofluids , liquid biopsy , micro-flow cytometry

Alpha Cognition – developing novel treatments for neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis

Alpha Cognition is a clinical stage, biopharmaceutical company dedicated to developing novel treatments for under-served neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis (ALS).

Alpha Cognition - developing novel treatments for neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis

ALPHA-1062, a patented new chemical entity that has demonstrated safety and improved ALPHA-1062, is a patented new chemical entity being developed as a new generation acetylcholinesterase inhibitor for the treatment of Alzheimer’s disease, with expected minimal gastrointestinal side effects. ALPHA-1062’s active metabolite is differentiated from donepezil and rivastigmine in that it binds neuronal nicotinic receptors, most notably the alpha-7 subtype, which is known to have a positive effect on cognition. ALPHA-1062 is also being developed in combination with memantine to treat moderate to severe Alzheimer’s dementia and as an intranasal formulation for traumatic brain injury.

ALPHA-0602 (Progranulin) is expressed in several cell types in the central nervous system and in peripheral tissues, promotes cell survival, regulates certain inflammatory processes, and plays a significant role in regulating lysosomal function and microglial responses to disease. Its intended use for the treatment of neurodegenerative diseases has been patented by the Company and Alpha-0602 has been granted an Orphan Drug Designation for the treatment of Amyotrophic Lateral Sclerosis by the FDA.

ALPHA-0702 and ALPHA-0802 are Granulin Epithelin Motifs, or GEMs, derived from full length progranulin which have therapeutic potential across multiple neurodegenerative diseases. Progranulin is comprised of 7½ Granulin Epithelin Motifs (GEMs). GEMs are important in regulating cell growth, survival, repair, and inflammation. Individual granulin domain peptides are approximately 60 amino acids in length (~7kDa). GEMs are metabolically stable and resistant to proteolysis (long half-life). Evidence suggests that not all GEMs are equally potent at promoting cell survival signaling or reducing proteinopathy, and may compete (e.g., binding to effectors) with active GEMs and effectively decrease their potency. Specific GEM combinations have been shown to be more effective than full length progranulin in promoting Cathepsin D maturation. GEMs have been shown to be important in regulating cell growth, survival, repair, and inflammation. Alpha-0702 and ALPHA-0802 are designed to deliver this with potentially lower toxicity, and greater therapeutic effect.

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Keywords : Alpha Cognition , neurodegenerative diseases , dementia , Alzheimer’s Dementia , Alzheimer Disease , Amyotrophic Lateral Sclerosis , acetylcholinesterase inhibitor , orphan drug , CNS , neuroscience , traumatic brain injury , Granulin Epithelin Motifs , Gene Therapy