InnoCare Announces Approval of Clinical Trial of BCL2 Inhibitor ICP-248 in Combination with Orelabrutinib as First-Line Therapy for Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma in China

BEIJING–(BUSINESS WIRE)–InnoCare Pharma (HKEX: 09969; SSE: 688428), a leading biopharmaceutical company focusing on the treatment of cancer and autoimmune diseases, announced today the approval of the Investigational New Drug (IND) to conduct the clinical trial of B-cell lymphoma-2 (BCL2) inhibitor ICP-248 in combination with Bruton’s tyrosine kinase (BTK) inhibitor orelabrutinib as first-line therapy for chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) in China.

This multicenter, randomized-controlled, open-label clinical study is designed to evaluate the efficacy and safety of ICP-248 combined with orelabrutinib versus immunochemotherapy in treatment-naive patients with CLL/SLL.

ICP-248 is a novel, orally bioavailable BCL2-selective inhibitor, which aims to treat hematologic malignancies as a monotherapy or in combination with other therapies. BCL2 is an important regulatory protein of apoptosis pathway, and its abnormal expression is related to the development of various hematologic malignancies. ICP-248 has an anti-tumor effect by selectively inhibiting BCL2 and restoring the mechanism of programmed cell death. The preliminary result has demonstrated a good efficacy and safety profile.

Orelabrutinib has been approved for marketing in China and Singapore, and all three approved indications related to lymphoma have been included in the National Reimbursement Drug List (NRDL) in China. In the treatment of relapsed/refractor CLL/SLL patients with orelabrutinib, the overall response rate and complete response rate reached 93.8% and 30% respectively, demonstrating an outstanding efficacy and safety profile.

Dr. Jasmine Cui, the co-founder, chairwoman and CEO of InnoCare, said, “InnoCare has developed strong pipeline in hemato-oncology that covers a variety of important hemato-oncology targets, including BTK, BLC2, CD19, CD20xCD3, E-3 ligase, etc. ICP-248 and orelabrutinib are important global assets of our company in the field of hematology, with multiple clinical studies conducted in China and the United States. We will accelerate clinical development and look forward to providing CLL/SLL patients with more efficient and safe treatment options.”

CLL/SLL, one of the most common types of leukemia, is an indolent malignancy of B lymphocytes. There are 191,000 newly diagnosed CLL cases and 61,000 deaths every year globally1. The incidence rate of CLL/SLL is on the rise in China2.

About InnoCare

InnoCare is a commercial stage biopharmaceutical company committed to discovering, developing, and commercializing first-in-class and/or best-in-class drugs for the treatment of cancers and autoimmune diseases with unmet medical needs in China and worldwide. InnoCare has branches in Beijing, Nanjing, Shanghai, Guangzhou, Hong Kong, and the United States.

InnoCare Forward-looking Statements

This report contains the disclosure of some forward-looking statements. Except for statements of facts, all other statements can be regarded as forward-looking statements, that is, about our or our management’s intentions, plans, beliefs, or expectations that will or may occur in the future. Such statements are assumptions and estimates made by our management based on its experience and knowledge of historical trends, current conditions, expected future development and other related factors. This forward-looking statement does not guarantee future performance, and actual results, development and business decisions may not match the expectations of the forward-looking statement. Our forward-looking statements are also subject to a large number of risks and uncertainties, which may affect our short-term and long-term performance.

_________________________

1 American Journal of Hematology
2 Ybanlv

Contacts

Media
Chunhua Lu

86-10-66609879

chunhua.lu@innocarepharma.com

Investors
86-10-66609999

ir@innocarepharma.com

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 – kim@kmacconnect.com
info@astrocytepharma.com 

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 www.seismictx.com and follow us on LinkedIn and on X @Seismic_Tx.

Contacts

Media Contact
Kathryn Morris, The Yates Network

914-204-6412

kathryn@theyatesnetwork.com

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

Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-DM1 for Treatment of Type 1 Myotonic Dystrophy

– Preclinical data show ARO-DM1 reduces muscular DMPK expression and corrects spliceopathies, which could lead to improved muscle strength and function

PASADENA, Calif.–(BUSINESS WIRE)–$arwr–Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) announced today that it has dosed the first subjects in a Phase 1/2a double-blinded, placebo-controlled, dose-escalating study (NCT06138743) to evaluate single and multiple ascending doses of ARO-DM1, the company’s investigational RNA interference (RNAi) therapeutic, in up to 48 subjects with type 1 myotonic dystrophy (DM1). DM1 is the most common adult-onset muscular dystrophy.

Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities. Additionally, patients may become physically disabled and have a shortened life span. There is currently no approved disease-modifying therapy for DM1. Treatments have focused on symptomatic management, including physical therapy, exercise, ankle-foot orthoses, and assistive devices, such as wheelchairs.

ARO-DM1 is designed to reduce expression of the dystrophia myotonica protein kinase (DMPK) gene in the muscle. Pathogenesis of DM1 is driven by an expanded CUG trinucleotide repeat in the 3’-untranslated region of DMPK transcripts. These abnormal transcripts cause mis-regulated splicing, known as spliceopathy, for certain messenger RNAs which are directly linked to the clinical manifestations of DM1.

Preclinical data recently presented at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference showed that in nonhuman primates, ARO-DM1 achieved greater than 80% silencing of DMPK in skeletal muscle, which was maintained for longer than 85 days. In a mouse model of DM1 harboring a pathogenic DMPK transgene, a modified species-specific version of ARO-DM1 (S-ARO-DM1) decreased the DMPK-CUG expression and corrected the spliceopathies.

Presentation materials may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.

About Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

For more information, please visit www.arrowheadpharma.com, or follow us on X (formerly Twitter) at @ArrowheadPharma or on LinkedIn. To be added to the Company’s email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts.

Safe Harbor Statement under the Private Securities Litigation Reform Act:

This news release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline or product candidates, including anticipated regulatory submissions and clinical program results, prospects or benefits of our collaborations with other companies, or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties, including the impact of the ongoing COVID-19 pandemic on our business, the safety and efficacy of our product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.

Source: Arrowhead Pharmaceuticals, Inc.

Contacts

Arrowhead Pharmaceuticals, Inc.

Vince Anzalone, CFA

626-304-3400

ir@arrowheadpharma.com

Investors:
LifeSci Advisors, LLC

Brian Ritchie

212-915-2578

britchie@lifesciadvisors.com
www.lifesciadvisors.com

Media:
LifeSci Communications, LLC

Jason Braco, Ph.D.

646-751-4361

jbraco@lifescicomms.com
www.lifescicomms.com

NurExone Presenting Novel Regulatory Pathways for Exosomes Therapies at Global Summit

TORONTO and HAIFA, Israel, March 01, 2024 (GLOBE NEWSWIRE) — NurExone Biologic Inc. (TSXV: NRX) (Germany: J90) (the “Company” or “NurExone”), a pioneering biopharmaceutical company, is pleased to announce that Dr. Ina Sarel, the Head of CMC, Quality and Regulatory Affairs at NurExone, will be leading a workshop at the upcoming Exosome Characterization & Analytical Development Summit. Dr. Sarel will be sharing with her expert colleagues insight and information on the topic of “Regulatory Challenges in the Development of an Extracellular Vesicles (EVs) – Based Clinical Product.”

Dr. Sarel joins a prestigious lineup of speakers including representatives from leading exosome companies, e.g. AbbVie, RION Therapeutics, Aegle Therapeutics and universities, e.g. Harvard Medical school. With her experience in regulatory affairs and her deep understanding of the exosome field, Dr. Sarel is well-positioned to provide perspective on how to navigate the complex regulatory landscape and ensure the successful development of exosome-based therapeutics.

Dr. Sarel who led the activities that resulted in the grant of Orphan Drug Designation for ExoPTEN, an exosome-therapy being developed by the Company, stated “NurExone is developing a platform for using exosomes to create wide range of nanodrugs including our first drug – ExoPTEN, for treating patients with acute spinal cord injury. We are proud to share our development outcomes together with our regulatory expertise and are committed to advancing the field of exosome therapeutics for clinical use.”

The Exosome Characterization & Analytical Development Summit is a premier event that brings together experts from academia and industry to discuss the latest advancements in exosome characterization and analytical development. The summit will take place on April 23-25, 2024 in Boston, MA. To learn more about the summit and to register, please visit the Conference Website.

About NurExone Biologic Inc.

NurExone Biologic Inc. is a TSXV listed pharmaceutical company that is developing a platform for biologically-guided exosome-based therapies to be delivered, non-invasively, to patients who have suffered Central Nervous System injuries. The company’s first product, ExoPTEN for acute spinal cord injury, was proven to recover motor function in 75% of laboratory rats when administered intranasally. ExoPTEN has been granted Orphan Drug Designation bythe FDA. The NurExone platform technology is expected to offer novel solutions to drug companies interested in noninvasive targeted drug delivery for other indications.

For additional information, please visit www.nurexone.com or follow NurExone on LinkedIn, Twitter, Facebook, or YouTube.

For more information, please contact:

Dr. Lior Shaltiel
Chief Executive Officer and Director
Phone: +972-52-4803034
Email: info@nurexone.com

Thesis Capital Inc.
Investment Relation – Canada
Phone: +1 905-347-5569
Email: IR@nurexone.com

Dr. Eva Reuter
Investment Relation – Germany
Phone: +49-69-1532-5857
Email: e.reuter@dr-reuter.eu

FORWARD-LOOKING STATEMENTS

This press release contains certain forward-looking statements, including statements about the Company’s future plans and intellectual property, the scientific and development and commercial activities to be carried out by the company, the efficient loading of exosomes, future potential manufacturing, clinical, licensing and marketing activities and the treatment of certain conditions. Wherever possible, words such as “may”, “will”, “should”, “could”, “expect”, “plan”, “intend”, “anticipate”, “believe”, “estimate”, “predict” or “potential” or the negative or other variations of these words, or similar words or phrases, have been used to identify these forward-looking statements. These statements reflect management’s current beliefs and are based on information currently available to management as at the date hereof. Forward-looking statements involve significant risk, uncertainties and assumptions. Many factors could cause actual results, performance or achievements to differ materially from the results discussed or implied in the forward-looking statements. Certain assumptions include the ability of the Company to commercialize its intellectual property internally and through licensing and that the Company has the appropriate team in order to realize commercialization. Risks and uncertainties include, but are not limited to, risks related to the Company’s early stage of development, lack of revenues to date, government regulation, market acceptance for its products, rapid technological change, dependence on key personnel, protection of the Company’s intellectual property and dependence on the Company’s strategic partners. These factors should be considered carefully and readers should not place undue reliance on the forward-looking statements. Although the forward-looking statements contained in this press release are based upon what management believes to be reasonable assumptions, the Company cannot assure readers that actual results will be consistent with these forward-looking statements. These forward-looking statements are made as of the date of this press release, and the Company assumes no obligation to update or revise them to reflect new events or circumstances, except as required by law.

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

FogPharma Announces $145 Million Financing to Support Ongoing Clinical Development of FOG-001 and Accelerate Helicon Peptide Portfolio

Financing led by Nextech Invest and joined by new investors RA Capital Management, Rock Springs Capital, General Catalyst, Marshall Wace, Samsara Biocapital, Foresite Capital, Symbiosis, Catalio Capital Management, Sixty Degree Capital and Alex Gorsky

CAMBRIDGE, Mass.–(BUSINESS WIRE)–FogPharma®, a clinical-stage biopharmaceutical company dedicated to delivering a new class of therapies that go beyond the limits of currently available medicines using its Helicon™ peptide platform, today announced the successful closing of a $145 million Series E financing round. The financing was led by Nextech Invest with participation from other new investors including RA Capital Management, Rock Springs Capital, General Catalyst, Marshall Wace, Samsara Biocapital, Foresite Capital, Symbiosis, Catalio Capital Management, Sixty Degree Capital and former chairman and CEO of Johnson & Johnson, Alex Gorsky.

There was strong support by existing investors, including ARCH Venture Partners, Fidelity Management & Research Company, GV, Cormorant Asset Management, funds and accounts advised by T. Rowe Price Associates, Inc., Farallon Capital Management, venBio Partners, Invus and Milky Way Investments. Alexis Borisy, an accomplished investor and entrepreneur, also joined the company’s board of directors in the position designated to Nextech.

The financing will fund the ongoing clinical development of FOG-001, the company’s first-in-class intracellular TCF-blocking β-catenin inhibitor currently being evaluated in a Phase 1/2 study in solid tumors. The round will also accelerate the development of its robust portfolio of unique discovery programs, deepen its data science capabilities and strengthen its core Helicon therapeutics platform.

FOG-001 is a Helicon designed to block the key oncogenic step in the Wnt/β-catenin signaling pathway, one of the most frequently activated pathways in a variety of cancers. Mutations in the pathway are particularly prevalent in colorectal cancer, the second leading cause of all global cancer deaths, with an estimated two million new cases per year, according to the World Health Organization. Despite significant biological validation of the β-catenin:TCF interaction as a cancer driver, no existing treatment has been able to disrupt it due to its inaccessibility to antibody and traditional small molecule medicines.

“Millions of colorectal cancer patients have been told by their oncologists that no more can be done for them. We believe FOG-001 may represent the long-awaited major technological breakthrough needed to address one of the most common yet unaddressed oncogenic signaling pathways,” said Mathai Mammen, M.D., Ph.D., FogPharma’s chairman, president and chief executive officer. “This financing will allow us to execute on our expanded clinical development and commercialization strategy to deliver FOG-001 to patients, while simultaneously strengthening our discovery efforts against other compelling intracellular targets that drive a range of diseases.”

FOG-001 is the first of several wholly-owned programs generated by FogPharma’s Helicon platform being advanced to address biologically validated disease-driving intracellular targets by modulating protein-protein and protein-DNA interactions inside the cell. FogPharma’s Helicon platform combines ultra-diverse and tunable stabilized helical peptides with custom-built computational physics and artificial intelligence (AI) techniques to enable the discovery and development of novel programs across a vast array of intracellular targets that have never before been drugged.

“We are excited by the tremendous potential of FOG-001 to meaningfully change the therapeutic landscape through a novel approach, and are equally impressed by FogPharma’s robust pipeline of programs for significant drivers of cancer,” said Nextech Managing Partner Thilo Schroeder, Ph.D. “The company’s rapid progress across its growing pipeline demonstrates FogPharma’s team is enabled to deliver on the vast potential of its Helicon therapeutics platform.”

The financing follows a period of rapid growth for the company as its leadership team has expanded with industry veterans with track records in clinical, commercial and data science execution. This includes the company’s Chairman, President and Chief Executive Officer Mathai Mammen, M.D., Ph.D.; healthcare data science pioneer and Chief Data Officer Brandon Allgood, Ph.D.; Chief Technical Operations Officer Rohin Mhatre, Ph.D.; and Chief Business Officer Gregory Miller, MPH, MBA.

About FogPharma®

FogPharma is a biopharmaceutical company pioneering the discovery and development of Helicon™ therapeutics, which are peptides capable of efficient cell entry and modulation of both protein-protein and protein-DNA interactions. Through Helicon therapeutics, FogPharma is poised to revolutionize the medical possibilities for patients by precisely drugging intracellular targets long understood to be significant drivers of disease but never before drugged due to the limitations of existing drug modalities to act within the cell. FOG-001, the company’s first-in-class TCF-blocking β-catenin inhibitor, is being evaluated in a Phase 1/2 study for patients with advanced solid tumors, including colorectal cancer. FogPharma is fully leveraging the unprecedented potential Helicons present by deploying proprietary, custom-built machine learning and computational methods as part of its discovery and development process. FogPharma has raised more than $500 million to date from leading life sciences investors. FogPharma is headquartered in Cambridge, Mass. For more information, please visit: www.fogpharma.com.

Contacts

Media
Ten Bridge Communications

Rebecca Spalding

rebecca@tenbridgecommunications.com

MaaT Pharma Announces Positive Review from the DSMB on the Ongoing Phase 1 Clinical Trial Evaluating MaaT033 in Amyotrophic Lateral Sclerosis (ALS)

  • Independent Data Safety and Monitoring Board (DSMB) recommended that the trial proceeds as planned without modifications.
  • MaaT033 has shown a good safety profile and was generally well tolerated in the first 8 patients with ALS treated with MaaT033 used in a chronic setting.
  • MaaT Pharma (EURONEXT: MAAT – the “Company”), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival of patients with cancer, today announced that the DSMB reviewed safety data in the first 8 patients with Amyotrophic Lateral Sclerosis (ALS) treated with MaaT033 in the IASO clinical trial. The DSMB recommended that the trial continue without modifications.

    The DSMB, composed of 4 independent experts, including an ALS patient association representative, concluded that safety was good. More precisely, it should be noted that no serious or severe adverse events were observed, and no infectious events could be related to MaaT033. The preliminary results reinforce confidence in the safety of MaaT033, a drug candidate produced by combining the microbiota from multiple donors using a “pooling” process.

    MaaT033 is currently evaluated, in a chronic setting, in a Phase 1b pilot study (NCT05889572) in ALS (also known as Lou Gehrig’s disease in the US and Charcot’s disease in French-speaking countries). The Company has developed the clinical trial with the French academic experts FILSLAN/ ACT4ALS-MND and in collaboration with the French patients’ association Tous en Selles contre la SLA. Data readout is now expected in early H2 2024. MaaT033 is also being evaluated in the Phase 2b trial PHOEBUS (NCT05762211), the largest one to date in Europe for a microbiome therapy in oncology, dedicated to improving survival of patients with blood cancers receiving allogeneic hematopoietic stem cell transplantation (HSCT).

    About MaaT033

    MaaT033, a donor-derived, high-richness, high-diversity oral Microbiome Ecosystem TherapyTM containing anti-inflammatory ButycoreTM species, is currently being developed as an adjunctive therapy to improve overall survival in patients receiving HSCT and other cellular therapies. It aims to ensure optimal microbiota function and to address a larger patient population in a chronic setting. MaaT033 has been granted Orphan Drug Designation by the European Medicines Agency (EMA).

    About MaaT Pharma

    MaaT Pharma, a clinical stage biotechnology company, has established a complete approach to restoring patient-microbiome symbiosis in oncology. Committed to treating cancer and graft-versus-host disease (GvHD), a serious complication of allogeneic stem cell transplantation, MaaT Pharma launched, in March 2022, an open-label, single arm, phase 3 clinical trial in patients with acute GvHD (aGvHD), following the achievement of its proof of concept in a phase 2 trial. Its powerful discovery and analysis platform, gutPrint®, enables the identification of novel disease targets, evaluation of drug candidates, and identification of biomarkers for microbiome-related conditions. The company’s Microbiome Ecosystem Therapies are produced through a standardized cGMP manufacturing and quality control process to safely deliver the full diversity of the microbiome, in liquid and oral formulations. MaaT Pharma benefits from the commitment of world-leading scientists and established relationships with regulators to support the integration of the use of microbiome therapies in clinical practice. MaaT Pharma is listed on Euronext Paris (ticker: MAAT).

    Forward-looking Statements

    All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim”, “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements.

    Contacts

    MaaT Pharma – Investor Relations
    Guilhaume DEBROAS, Ph.D.

    Head of Investor Relations

    +33 6 16 48 92 50

    invest@maat-pharma.com

    MaaT Pharma – Media Relations
    Pauline RICHAUD

    Senior PR & Corporate Communications Manager

    +33 6 14 06 45 92

    media@maat-pharma.com

    Trophic Communications
    Stephanie MAY or

    Priscillia PERRIN

    +49 171 185 56 82

    maat@trophic.eu

    Animal Biosciences Announces New Canine Clinical Research Evaluating Reversal of Age-Related Signs in Dogs

    After five years of development, positive results are seen in old dogs in a rigorous placebo-controlled trial performed at a leading vet school

    BOSTON–(BUSINESS WIRE)–Animal Biosciences, Inc., a company aiming to extend the lifespan of pets, today announced new clinical research published to the preprint server BioRxiv that tested the effects of their “LeapYears” supplement designed to extend healthspan in dogs.

    After five years of development and testing in dogs, leveraging discoveries made at Harvard Medical School, the study shows the first clinical evidence that it is possible to reverse age-related decline in dogs.

    The supplement, formulated as a soft chew, is a combination of an “NAD booster” to mimic fasting, and a molecule that kills “zombie” senescent cells that cause aging. The combination was developed because the effects in vivo were better than the single molecules alone.

    The double-blind clinical study conducted at North Carolina State University Veterinary College, in collaboration with Dr. Natasha Olby, a Professor of Neurology and Neurosurgery, has confirmed the effects of the supplement to significantly improve cognitive function. The formulation may have broader effects on frailty, activity, and happiness, as assessed by owners.

    “The outcomes of the clinical trial, especially the enhancements in cognition, are encouraging and represent a unique achievement,” Dr. Olby noted. “This rigorous study, which acknowledges the difficulties aging pets and their owners encounter, shows dedication to scientific methods aimed at improving the quality of life for our canine companions.”

    Aging has been defined as the chronic dysregulation of gene expression networks over time leading to subsequent deteriorated tissue and organ function causing age-related functional decline. In senior dogs, as in humans, this age-related decline can manifest as cognitive decline, increased frailty and lower engagement. The results of the trial showed positive impacts in all these areas of decline.

    Dr. David A. Sinclair, AO, PhD, Professor of Genetics at Harvard Medical School and co-founder of Animal Biosciences stated, “I am very proud of the teams at NCSU and Animal Biosciences, who, after years of collaborative research and a clinical trial, have developed the first supplement proven to reverse aging in dogs.”

    Animal Biosciences Inc. is a Boston-based animal health company focused on delaying aging and extending the healthy lifespan of companion animals. Co-founded in 2017 by well-known longevity pioneer, Professor David A. Sinclair of Harvard Medical School, and his brother, Nick Sinclair, CEO, a dedicated dog-lover with experience in the pharmaceutical and food industries, Animal Biosciences is adapting small molecule-based therapies from human health specifically for companion animals and testing them in rigorous clinical trials. The company has a pipeline of additional small molecules and therapies targeting aging in companion animals.

    Contacts

    arianna@gcw.agency
    Phone: 646-964-4446

    Atara Biotherapeutics Receives FDA Clearance of IND Application in Lupus Nephritis for ATA3219, an Allogeneic CAR T Therapy

    Second IND Clearance for ATA3219 Following Non-Hodgkin’s Lymphoma (NHL) and First in an Autoimmune Disease Indication

    Initial Clinical Data in NHL Anticipated in H2 2024 and for Lupus Nephritis in H1 2025

    THOUSAND OAKS, Calif.–(BUSINESS WIRE)–$ATRA #CARTAtara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, today announced the U.S. Food and Drug Administration (FDA) has cleared an Investigational New Drug (IND) application for ATA3219, an allogeneic, anti-CD19 chimeric antigen receptor (CAR) T-cell monotherapy for the treatment of systemic lupus erythematosus (SLE) with kidney involvement (lupus nephritis [LN]).

    Expanding upon an extensive clinical experience encompassing the treatment of over 600 patients using our allogeneic T-cell platform in both oncology and autoimmune diseases, we are excited to clinically evaluate the potential of our differentiated allogeneic CAR T-cell approach. We are eager to address the significant unmet need in lupus nephritis as we initiate our Phase 1 trial,” said Pascal Touchon, President and Chief Executive Officer of Atara. “We look forward to bringing the promise and accessibility of a potentially curative off-the-shelf cell therapy option to patients with severe autoimmune diseases, potentially eliminating the burdens of autologous CAR T therapies like costly infrastructure and treatment delays.”

    The multi-center, Phase 1, open-label, single-arm, dose-escalation study will evaluate the safety and preliminary efficacy of ATA3219 in subjects with LN. Subjects will receive lymphodepletion treatment followed by ATA3219 at a dose of 40, 80, or 160 x 106 CAR+ T cells. ATA3219 is designed to be given as a one-time infusion and followed for safety and efficacy. Each dose level is designed to enroll 3-6 patients, with the first subject expected to be enrolled in the second half of 2024.

    Existing therapeutic agents for lupus nephritis yield suboptimal responses and have limitations due to their requirement for ongoing administration, susceptibility to treatment failures, and limited accessibility to inflamed tissues resulting in incomplete depletion of B cells,” said Rajani Dinavahi, Chief Medical Officer at Atara. “CAR T cells can naturally infiltrate deep into target tissues to mediate B-cell depletion and produce durable responses. Building on the encouraging academic data in lupus nephritis with autologous CD19 CAR T, ATA3219 is an off-the-shelf therapy that could significantly reduce constraints for patients and physicians like leukapheresis and long waiting times, therefore potentially improving access to a large population of patients.”

    Proof of concept for a CD19 CAR T approach in autoimmune disease was first demonstrated in early academic results from an investigator-sponsored study showing 100% (8/8) of LN patients rapidly attaining drug-free, durable remission with an autologous CD19-targeted CAR T therapy. The therapy eliminated the pathogenic, autoreactive B cells and allowed healthy B cells to return after treatment, enabling the patients’ immune system to function normally again with associated improvement of clinical symptoms.1 These early proof of concept clinical data with CD19 targeted CAR T support further development of CAR T for LN with differentiated and off-the-shelf allogeneic approaches.

    The ATA3219 IND submission included in vitro data reflecting the CD19 antigen-specific functional activity of ATA3219 and CAR-mediated activity against B cells from SLE patients. ATA3219 led to robust CD19-specific B-cell depletion compared to controls.

    LN is a serious and most common complication of SLE, a chronic multisystem autoimmune disease. The prevalence of SLE in the U.S. is 73 per 100,000 people, afflicting more than 200,000 U.S. patients alone, and occurs in women much more commonly than men. Up to 60% of adult patients with SLE develop renal disease during the course of their illness, and up to 70% of patients with LN are refractory to standard immunosuppressive therapies. Despite recent advances in treatment strategies, the response rate using existing therapies remains low, with significant risk of long-term morbidity and mortality associated with refractory LN.

    About ATA3219

    ATA3219 combines the natural biology of unedited T cells with the benefits of an allogeneic therapy. It consists of allogeneic Epstein-Barr virus (EBV)-sensitized T cells that express a CD19 CAR construct for the treatment of CD19+ relapsed or refractory B-cell malignancies, including B-cell non-Hodgkin’s lymphoma and B-cell mediated autoimmune diseases including systemic lupus erythematosus (SLE) with kidney involvement (lupus nephritis [LN]). ATA3219 has been optimized to offer a potential best-in-class profile, featuring off-the-shelf availability. It incorporates multiple clinically validated technologies including a modified CD3ζ signaling domain (1XX) that optimizes expansion and mitigates exhaustion, enrichment during manufacturing for a less differentiated phenotype for robust expansion and persistence and retains the endogenous T-cell receptor without gene editing as a key survival signal for T cells contributing to persistence.

    Next-Generation Allogeneic CAR T Approach

    Atara is focused on applying Epstein-Barr virus (EBV) T-cell biology, featuring experience in over 600 patients treated with allogeneic EBV T cells, and novel chimeric antigen receptor (CAR) technologies to meet the current limitations of autologous and allogeneic CAR therapies head-on by advancing a potential best-in-class CAR T pipeline in oncology and autoimmune disease. Unlike gene-edited approaches aimed at inactivating T-cell receptor (TCR) function to reduce the risk for graft-vs-host disease, Atara’s allogeneic platform maintains expression of the native EBV TCR that promote in vivo functional persistence while also demonstrating inherently low alloreactivity due to their recognition of defined viral antigens and partial human leukocyte antigen (HLA) matching. A molecular toolkit of clinically-validated technologies—including the 1XX costimulatory domain designed for better cell fitness and less exhaustion while maintaining stemness—offers a differentiated approach to addressing significant unmet need with the next generation CAR T.

    About Atara Biotherapeutics, Inc.

    Atara is harnessing the natural power of the immune system to develop off-the-shelf cell therapies for difficult-to-treat cancers and autoimmune conditions that can be rapidly delivered to patients from inventory. With cutting-edge science and differentiated approach, Atara is the first company in the world to receive regulatory approval of an allogeneic T-cell immunotherapy. Our advanced and versatile T-cell platform does not require T-cell receptor or HLA gene editing and forms the basis of a diverse portfolio of investigational therapies that target EBV, the root cause of certain diseases, in addition to next-generation AlloCAR-Ts designed for best-in-class opportunities across a broad range of hematological malignancies and B-cell driven autoimmune diseases. Atara is headquartered in Southern California. For more information, visit atarabio.com and follow @Atarabio on X and LinkedIn.

    Forward-Looking Statements

    This press release contains or may imply “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. For example, forward-looking statements include statements regarding the development, data, timing and progress, as applicable, of: (1) the ATA3219 program; (2) the potential characteristics and benefits of ATA3219; (3) the Company’s planned clinical study of ATA3219 to treat lupus nephritis, including the timing and enrollment thereof. Because such statements deal with future events and are based on Atara’s current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Atara could differ materially from those described in or implied by the statements in this press release. These forward-looking statements are subject to risks and uncertainties, including, without limitation, risks and uncertainties associated with the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success; the ongoing COVID-19 pandemic and the wars in Ukraine and the Middle East, which may significantly impact (i) our business, research, clinical development plans and operations, including our operations in Southern California and Denver and at our clinical trial sites, as well as the business or operations of our third-party manufacturer, contract research organizations or other third parties with whom we conduct business, (ii) our ability to access capital, and (iii) the value of our common stock; the sufficiency of Atara’s cash resources and need for additional capital; and other risks and uncertainties affecting Atara’s and its development programs, including those discussed in Atara’s filings with the Securities and Exchange Commission , including in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of the Company’s most recently filed periodic reports on Form 10-K and Form 10-Q and subsequent filings and in the documents incorporated by reference therein. Except as otherwise required by law, Atara disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

    1Mueller, F., et al. CD19-Targeted CAR-T Cells in Refractory Systemic Autoimmune Diseases: A Monocentric Experience from the First Fifteen Patients. Blood 2023; 142 (Supplement 1): 220.

    Contacts

    Investor and Media Relations:
    Jason Awe, Ph.D.

    Senior Director, Corporate Communications & Investor Relations

    (805) 217-2287

    jawe@atarabio.com

    Orbis Medicines Launches with €26 Million Seed Financing to Transform Macrocycle Drug Development through Next-Generation Orally Dosable ‘nCycles’

    Financing led by Novo Holdings and Forbion

    Orbis’ platform is first to systematically explore macrocycle chemical space using unique combination of high-throughput chemical synthesis and large-scale assaying, supported by machine learning

    Pipeline to initially focus on high-value oral alternatives to blockbuster biologic drugs and opening new targets to therapeutic intervention

    COPENHAGEN, Denmark–(BUSINESS WIRE)–Orbis Medicines, a leader in oral macrocycle drug discovery, today announces its launch with a 26 million financing led by global life sciences investor Novo Holdings and European life sciences venture firm Forbion. The funding will support Orbis’ expansion and advancement of its portfolio of next-generation macrocycle drugs it calls ‘nCycles’. Macrocycles are a large and diverse family of compounds with highly desirable therapeutic properties, defined by the presence of a cyclic structure. nCycles are systematically designed by Orbis’ nGen1 platform to be orally bioavailable and membrane permeable, solving decades-long challenges in macrocycle drug design.

    The Orbis pipeline includes programs against targets validated by blockbuster biologic drugs, with the goal of providing oral alternatives that will enable the treatment of many more patients. In addition, Orbis is developing a pipeline of nCycles for other attractive target classes, both intra- and extra-cellular. Oral macrocycle drugs can potentially address a very wide range of diseases based on their ability to target a majority of the potential molecular targets in the body.

    “After decades of challenging de novo synthesis, the universe of potential macrocycle targets is now available for exploration. Not only can Orbis reach a wealth of new targets, both inside and outside cells, but we can do this while preserving an oral format to pioneer a new therapeutic class of oral macrocycles. It’s a milestone for drug development,” said Morten Døssing, Chair of the Orbis Board and Partner at Novo Holdings. “We believe Orbis has the premier chemistry-led approach in the field, which gives us a distinct data advantage. We can generate hundreds of thousands of diverse compounds in record time and immediately apply a range of functional assays in a high-throughput fashion to each individual compound to home in on winners. Establishing this robust, reliable engine for candidate design was crucial for us as we look to bring macrocycle drugs to major patient populations.”

    Orbis was founded in 2021 by the Seeds Investment team of Novo Holdings, with Mr. Døssing as Executive Chair and João Ribas as interim CBO establishing the company’s team and building its corporate strategy. The scientific foundation of Orbis was developed by Prof. Christian Heinis and Sevan Habeshian at the Swiss Federal Institute of Technology in Lausanne (EPFL).

    “The broad potential of oral macrocycle drugs to address challenging targets untouchable by small molecules, coupled with recent momentum in the field, makes this an incredibly exciting development,” said Marco Boorsma, Ph.D., General Partner at Forbion. “With a compelling AI-enabled platform technology, scientific expertise and a clear strategy for growth, Orbis is attracting strong interest for its approach to finally delivering on the promise of this class for medicine. We believe Orbis’ combinatorial approach and ability to continuously generate big data from real compounds gives them an undeniable opportunity to unlock the potential of orally available macrocycle drugs for significantly larger patient populations.”

    The chemical methods by which Orbis rapidly generates vast libraries of diverse macrocycle compounds ready for immediate assay were previously published in Nature Communications. Authors of the paper, which included Orbis’ founders Professor Heinis and Dr. Habeshian, detail how the methods draw on a large range of components to build compounds, including both natural and synthetic amino acids, creating an extremely large chemical space for exploration. Additionally, research recently published in Nature Chemical Biology demonstrates nGen’s ability to deliver nCycles that are orally bioavailable, marking a new era for macrocycle drug discovery.

    About Orbis Medicines

    Orbis Medicines is a biotechnology company focused on pioneering a new era for oral macrocycle drug discovery. Macrocycles are a large and diverse family of compounds with highly desirable therapeutic properties, defined by the presence of a cyclic structure. Orbis’ nGen platform is designed to systematically explore the macrocycle chemical space using a highly automated chemistry platform and deliver oral macrocycle drug candidates – nCycles – suitable for both intra- and extracellular targets. Orbis’ programs are focused on high-value oral alternatives to blockbuster biologic drugs and targets challenging for established modalities. Orbis was founded by Novo Holdings. For more information, please visit: www.orbismedicines.com

    1About nGen

    nGen is Orbis’ technology platform for generating oral macrocycle drug candidates, which it calls nCycles. It consists of multiple proprietary integrated elements starting with hit finding libraries of 100 billion compounds. Hits identified are progressed using Orbis’ highly automated chemistry-based platform that rapidly creates and tests hundreds of thousands of individual analogues with a full suite of assays to identify the most desirable ones. The scale and quality of the data produced from these real compounds, paired with machine learning, creates an industry-leading platform that de-risks and accelerates development. Recently published research in Nature Chemical Biology demonstrates nGen’s ability to deliver nCycles that are orally bioavailable, marking a new era for macrocycle drug discovery.

    About Novo Holdings A/S

    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 Novozymes A/S and manages an investment portfolio with a long-term return perspective. In addition to managing a broad portfolio of equities, bonds, real estate, infrastructure and private equity assets, Novo Holdings is a world-leading life sciences investor. Through its Seeds, Venture, Growth, and Principal Investments teams, Novo Holdings invests in life science companies at all stages of development. As of year-end 2022, Novo Holdings had total assets of EUR 108 billion. www.novoholdings.dk

    About Forbion

    Forbion is a dedicated life sciences venture capital firm with offices in The Netherlands and Germany. Forbion invests in life sciences companies that are active in the biopharmaceutical space, managing €3 billion across multiple fund strategies that cover all stages of biopharmaceutical drug development. The firm’s current team consists of more than 30 life sciences investment professionals that have built an impressive performance track record since the late nineties with investments in over 100 companies across 8 funds. Forbion’s record of sourcing, building and guiding life sciences companies has resulted in many breakthrough therapies and valuable exits. In addition to its financial return objectives, Forbion selects investments that will positively affect the health and well-being of patients. The firm is a signatory to the United Nations Principles for Responsible Investment. More information can be found at www.forbion.com

    Contacts

    Media:
    Kit Rodophele

    Ten Bridge Communications

    617-999-9620

    krodophele@tenbridgecommunications.com