β-galactosidase

HYOGO, Japan--( BUSINESS WIRE )-- JCR Pharmaceuticals Co., Ltd. (TSE: 4552) made significant contributions at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024, held in Porto, Portugal from September 3-6, 2024. We highlighted our proprietary J-Brain Cargo ® technology through two key presentations focused on advanced therapies for lysosomal storage disorders, demonstrating our pioneering role in medical innovation. "In treating lysosomal storage disorders, it's critical to address the neurological symptoms that often accompany these conditions,” said Shin Ashida, Chairman and President of JCR. “Our focus is on developing therapies that meet these unmet medical needs. Clinical data for JR-171 show its potential to be an effective treatment for both the somatic and neurological symptoms associated with mucopolysaccharidosis I (MPS I). Additionally, the preclinical trials in our GM1 gangliosidosis AAV gene therapy development program have demonstrated the potential of combining our J-Brain Cargo ® technology with gene therapy. We will continue to update on the progress of these programs." JCR showcased the following two presentations: JR-171: Mucopolysaccharidosis Type I (MPS I) Poster The following poster presentation provides a case report for four individuals with MPS I who were treated with JR-171: Neurobehavioural and somatic improvements observed in MPS I patients treated with lepunafusp alfa (JR-171): report of four cases (PO-238) Presenter: Ana Maria Martins, M.D., Ph.D. (Federal University of São Paulo, São Paulo, Brazil) This study reports clinical data from four individuals with MPS I in Brazil who were treated intravenously with JR-171 (2 mg/kg weekly, n=2; 4 mg/kg weekly, n=2), focusing on neurobehavioral changes. These individuals with MPS I experienced marked neurobehavioral and somatic improvements, which resulted in tangible, positive changes in their quality of life. These cases illustrate the positive neuropsychiatric and behavioral changes observed during the Phase 1/2 study of JR-171, as well as the drug’s somatic efficacy, and they demonstrate its potential as an effective treatment for the somatic symptoms and neuronopathy found in individuals with MPS I. Further neurocognitive and developmental evaluation is required to consolidate and establish the efficacy of JR-171 for neuronopathic symptoms. GM1 Gangliosidosis Poster This poster presentation was selected as one of the highest-ranked posters. It highlights preclinical data from an adeno-associated virus (AAV) gene therapy in a mice model of GM1 gangliosidosis: Gene therapy for GM1 gangliosidosis mediated by AAV vector carrying BBB-penetrable enzyme (PO-211) Presenter: Saki Matsushima, Ph.D. (Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan) This study reports preclinical data that demonstrated efficacy of AAV that expresses anti-transferrin receptor-antibody (anti-TfR antibody) fused β-galactosidase (TβGal). AAV was administered intravenously to the GM1 gangliosidosis mouse models. TβGal expressed in the liver was secreted into the blood and efficiently penetrated the central nervous system (CNS) as well as peripheral organs. Substrates in the CNS decreased in a dose-dependent manner. The data also demonstrated efficacy of TβGal in survival, histology, and behavioral evaluations. This therapeutic approach, which is the combination of AAV and anti-TfR antibody fused biotherapeutics, is expected to be applied for the treatment of several hereditary neurological diseases with CNS involvement. Further evaluation is required. About the Society for the Study of Inborn Errors of Metabolism (SSIEM) Established in 1963, the aim of the society is to foster the study of inherited metabolic disorders and related topics. An international annual symposium is held to promote exchange of ideas between professional leaders in different disciplines who are interested in inborn errors of metabolism. Learn more about SSIEM: https://www.ssiem.org/ . About the J-Brain Cargo ® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology J-Brain Cargo ® , to bring biotherapeutics into the central nervous system. The first drug developed based on this technology is IZCARGO ® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder. About Mucopolysaccharidosis I (Hurler, Hurler-Scheie, Scheie Syndrome) Mucopolysaccharidosis I (MPS I) is an autosomal recessive lysosomal storage disorders (LSDs) caused by a deficiency of α-L-iduronidase, an enzyme that breaks down glycosaminoglycans (mucopolysaccharides) in the body. The current worldwide prevalence of MPS I is estimated to be approximately 3,000-4,000 (according to JCR internal research). MPS I gives rise to a wide range of somatic and neurological symptoms. A major limitation of current ERT is that it does not address central nervous system (CNS) symptoms because of the enzyme’s inability cross the blood-brain barrier (BBB). MPS I is the only LSD in which hematopoietic stem cell transplantation (HSCT) is part of the standard of case for the severe form of the disease. Significant unmet medical need persists in all forms of MPS I. About GM1 Gangliosidosis GM1 gangliosidosis is an autosomal recessive disease caused by pathogenic mutations in the GLB1 gene which results in dysfunction or lack of production of lysosomal enzyme, β-galactosidase, responsible for the breakdown of GM1 gangliosides and other glycolipids. Accumulation of GM1 gangliosides in multiple tissues including the brain leads to severe neurodegeneration and systemic disease manifestations resulting in premature mortality. GM1 gangliosidosis is classified into three forms (Type I to III) based on the age of onset of clinical symptoms. About JR-171 JR-171 is a recombinant fusion protein of an antibody against the human transferrin receptor and α-L-iduronidase, the enzyme that is missing or malfunctioning in patients with MPS I. By crossing the blood brain-barrier (BBB) through transferrin receptor mediated transcytosis it is expected to be effective against central nervous system (CNS) signs and symptoms of the disease thereby addressing a significant unmet need for the treatment of MPS I. JR-171 previously was granted Fast Track designation by the US Food and Drug Administration (FDA). About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 49-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical advancement at a global level. Our core values – reliability, confidence, and persistence – benefit all our stakeholders, including employees, partners, and patients. For more information, please visit https://www.jcrpharm.co.jp/en/site/en/ . Cautionary Statement Regarding Forward-Looking Statements This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as “believe,” “estimate,” “anticipate,” “intend,” “plan,” “will,” “would,” “target” and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors’ pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future.

Valneva plans to use the funds from the priority review voucher sale to advance its research and development (R&D) efforts. Image Credit: MacroEcon / Shutterstock. Valneva has sold its US Food and Drug Administration (FDA) priority review voucher for €95m ($103m) to an undisclosed party. The French company received the priority review voucher when its chikungunya vaccine, Ixchiq, got FDA approval in November 2023. Valneva plans to use the funds from the voucher sale to advance its research and development (R&D) efforts, as per a 5 February press release. The voucher sale price is in line with the company’s previous projections of gaining €90m-€110m with such a sale. One of the projects that the company plans to use its proceeds for is the Lyme disease vaccine, VLA15 . Valneva is co-developing the vaccine in partnership with Pfizer . VLA15 is an experimental multivalent protein subunit vaccine that targets the outer surface protein A (OspA) of Borrelia. The vaccine is being evaluated in a placebo-controlled Phase III trial (NCT05477524). The trial suffered a delay when both companies paused patient recruitment and testing due to “Good Clinical Practices (GCP) violations” by a third-party contractor Care Access. The contract research organisation (CRO) later challenged these allegations and an FDA investigation found no misconduct by Care Access. However, the programme received a setback with the companies delaying the planned submission of a biologics licence application to the FDA and a marketing authorisation application to the European Medicines Agency by one year to 2026. See Also: Keytruda remains Merck & Co’s biggest 2023 success story as patent expiry looms Regeneron forms Regeneron Cell Medicines R&D unit Valneva also plans to use the funds to conduct additional clinical trials for Ixchiq and to expand its clinical pipeline. In December 2023, the 24-month follow-up results from the Phase IIIb trial (NCT04838444), following inoculation Ixchiq show that 97% of the 316 healthy adult subjects in the study had neutralising antibody titers above the seroresponse threshold. The FDA grants new priority review vouchers to a company when a drug for neglected tropical disease for rare paediatric disease or material threat medical countermeasure is approved. But there is no guarantee that a voucher will be awarded. bluebird bio missed out on gaining an FDA priority voucher through this mechanism for its sickle cell gene therapy, Lyfgenia (lovotibeglogene autotemcel), after the agency stated that the company was ineligible due to the therapy’s active ingredient already being used in the company’s FDA approved beta-thalassemia gene therapy Zynteglo (betibeglogene autotemcel). The FDA priority review vouchers reduce the FDA review time of a drug application from the usual ten months to six months. Vouchers do not need to be used by the company that received them and can be sold to raise funds .

BETHESDA, Md., Jan. 31, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain,” or the “Company”), a biotechnology company leading the discovery and development of allosteric small molecule therapies, today releases the following letter to stockholders from its President and Chief Executive Officer Matthias Alder. Dear Fellow Stockholders, With the start of a new year, I want to take a moment to reflect on Gain’s progress over the last 12 months and to share our strategy and plans for 2024. Research and development of pharmaceutical products for the treatment of neurodegenerative diseases has seen tremendous progress in recent years, with the approval of new drugs for the treatment of Alzheimer’s disease, multiple sclerosis and ALS. The pharmaceutical industry is taking note as evidenced by the recent acquisition of Cerevel Therapeutics for $8.7 billion by AbbVie, and the acquisition of preclinical-stage Caraway Therapeutics by Merck with an announced deal value of $610 million. In Parkinson’s disease, the second most common neurodegenerative disease after Alzheimer’s disease, new insights into the root cause of the disease have led to a number of drug candidates advancing in clinical development, but there are no approved therapies available to patients that can slow or stop the progression of the disease. At Gain, we seek to address this high unmet medical need with our clinical-stage lead drug candidate GT-02287, which we believe has disease-modifying potential for the treatment of Parkinson’s disease and other neurodegenerative diseases. Successful Transition to a Clinical-Stage Biotech Company In 2023, we advanced GT-02287 through preclinical development and initiated the company’s first clinical trial in September 2023 on time and on plan. The Phase 1 clinical trial is a single center, randomized, double-blind, placebo-controlled, single-ascending dose (SAD) and multiple-ascending dose (MAD) study to evaluate the safety and tolerability of GT-02287 administered orally in healthy adults. The dose escalation of the SAD phase is underway, and the MAD phase of the study is expected to begin in Q1 2024. The SAD/MAD part of the Phase 1 clinical trial is expected to be completed by mid-2024 with results expected to be reported in the second half of the year. In Q3 2024, we plan to start treatment of a cohort of 12-15 Parkinson’s patients as an extension of the ongoing Phase 1 clinical trial to establish a biomarker-based clinical proof of concept, replicating the effects of GT-02287 on biomarkers we have observed in our preclinical in vivo studies. We expect data from this study to be achieved in late 2024 or early 2025 ahead of starting a Phase 2 clinical trial in the first half of 2025. Cutting-Edge Science and Best-in-Class Preclinical Data Package In 2023, we made several data presentations of results of our GBA1 program in preclinical models of Parkinson’s disease and Alzheimer’s disease. For example, we presented new data demonstrating a reduction of the plasma neurodegeneration biomarker NfL after administration of GT-02287 in a GBA1 Parkinson’s disease model at the International Congress of Parkinson's Disease and Movement Disorders® last August. NfL is an emerging biomarker of neurodegeneration that was recently accepted by the FDA as a surrogate endpoint in the agency’s accelerated approval of a drug for the treatment of certain ALS patients. In that same preclinical model, GT-02287 also restored β-glucocerebrosidase (GCase) enzymatic function, reduced aggregated α-synuclein, neuroinflammation and neuronal death, increased dopamine levels and improved motor function. We presented additional data on our allosteric GCase modulators at the March 2023 International Conference on Alzheimer’s and Parkinson’s Diseases. In that poster presentation, we showed results that we believe support the disease-modifying potential of allosteric GCase regulators for the treatment of Alzheimer’s disease. Finally, we presented results of a preclinical model that we believe support the disease-modifying potential of a GCase-targeting small molecule for neuronopathic Gaucher disease at last year’s 19th Annual WORLDSymposium.™ The data generated in an animal model of neuronopathic Gaucher disease show that GT-02329 restores GCase activity, depletes accumulation of toxic lipid substrates, reduces neuroinflammation, and improves neuromuscular function. Additionally, we published data in PLOS ONE on our allosteric small molecule modulators of the GLB1 enzyme showing significantly restored β-Gal function and reduced intracellular toxic substrates as part of our program for GM1 gangliosidosis. These results were generated in collaboration with the Institute for Research in Biomedicine in Bellinzona, Switzerland. In 2024, we expect to present further data on GT-02287 from preclinical models at scientific conferences, starting with the 20th Annual WORLDSymposium™ being held February 4-9, 2024, in San Diego, CA. At that conference, we will present new in vitro and in vivo data that were accepted as a late-breaker abstract titled, “GT-02287, a clinical stage GCase enhancer, displays neuroprotection and restores motor function in preclinical models of Parkinson’s disease following delayed administration.” In addition, the abstract was selected for an oral presentation at the conference on February 9, 2024. Based on the preclinical data we have generated with GT-02287 across numerous in vitro and in vivo models of Parkinson’s disease, we believe that GT-02287 has a best-in-class preclinical profile with evidence of improving the entire disease cascade caused by GCase dysfunction and offering the potential to slow or even stop the progression of this devastating neurodegenerative disease. Magellan™ Platform During the course of 2023, we significantly upgraded our computational drug discovery platform. The platform now provides for an integrated, efficient workflow of existing and newly added tools and programming technologies that enable the identification of new binding sites on proteins and screening of the 50+ billion chemical spaces that have become available through providers like Enamine. Over the last 18 months, we also expanded the application of the platform beyond its original focus of enhancing and restoring enzyme function to new modalities that disrupt the function of target proteins. Based on the added capabilities and expanded application, we feel that the original platform name SEE-Tx (Site-specific Enzyme Enhancement Therapy) no longer adequately represents our platform technology. Magellan, the name of our next-generation platform, reflects its enhanced capabilities that enable the exploration of new protein biology and a vast chemical space to discover new allosteric small molecule therapies. Based on the unique combination of physics-based models and integrated AI/ML-enhanced virtual screening capabilities, we believe Magellan is highly differentiated in the field of computational drug discovery platforms and positions us to continue building our own pipeline of potential first-in-class drug candidates. In addition, we are excited about the prospect of deploying Magellan in drug discovery collaborations with academic institutions, biotechnology companies, CROs, and pharma companies, and will provide updates on our progress in these efforts throughout 2024. Cash Runway into 2025 In late November 2023, we completed a CMPO/PIPE financing that raised gross proceeds of $10.1 million. Based on our operational plans, we are now positioned to fund our operations into 2025, which we expect will enable the achievement of a biomarker-based clinical proof of concept of GT-02287 in the previously mentioned Phase 1 patient cohort. In addition, we received grants totaling approximately $3.4 million during the year to fund R&D activities for our pipeline programs. In 2024, we plan to continue tapping into grant opportunities to provide non-dilutive funding for our pipeline programs. Strategic Partnering and Business Development In 2023, we continued our active engagement with potential industry partners for our pipeline programs and platform technology. Following the J.P. Morgan Healthcare conference in early January, I am optimistic about our strategic partnering efforts, and we will continue to pursue opportunities for one or more of our R&D assets, whether for our lead drug candidate GT-02287, our earlier pipeline programs, or the Magellan platform or a combination thereof. We believe that potential transactions for any of our assets could provide significant non-dilutive funding and/or increase the inherent value of Gain by advancing partnered programs through value inflection points. Organizational Development With the transition from a discovery and research-stage company to a clinical-stage biotech company, we are continuously evaluating the organization and adding new skills required to achieve our goals in the next 2-3 years while maintaining our focus on prudent cash management. Our new status as a clinical company has necessitated the search for a Chief Medical Officer who will be charged with advancing the development of the clinical strategy for GT-02287 and leading the clinical development team and related functions at Gain. We look forward to updating you once we have secured the appropriate candidate. Also, individual career objectives can lead to changes in organizations. Unfortunately, we are saying goodbye to Dr. Xavi Barril, our current Chief Technology Officer, who has been instrumental in guiding the evolution of our computational drug discovery platform. Xavi is leaving the world of academia as a professor at the University of Barcelona and his part-time engagement with Gain to join a major pharmaceutical company. As we wish him luck, the Magellan platform remains in good hands with an experienced team led by Dr. Elena Cubero, who has been working with Xavi and the platform over the last 10 years. Outlook for 2024 In 2024, we expect to achieve a series of important value inflection points with Gain, which are summarized below: Q1 2024: Poster and Platform Presentation of new data in preclinical model of Parkinson’s disease at WORLDSymposiumStart of MAD cohort in Phase 1 clinical trial with GT-02287 Q2 2024 Completion of MAD cohort of Phase 1 clinical trial; potential for showing effect on GCase levels and activation Q3 2024 Start of Parkinson’s patient cohort in Phase 1 clinical trial Q4 2024 / Q1 2025 Completion of patient cohort; potential to show biomarker-based clinical proof of concept I look forward to updating you as we progress Gain’s business and programs throughout this year and thank you for your continued support along the way to providing new treatments for debilitating neurodegenerative diseases. Sincerely, Matthias AlderPresident and CEOGain Therapeutics, Inc. About Gain Therapeutics, Inc. Gain Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate GT-02287 for the treatment of GBA1 Parkinson’s disease, is currently being evaluated in a Phase 1 clinical trial. Leveraging AI-supported structural biology, proprietary algorithms and supercomputer-powered physics-based models, the company’s Magellan™ discovery platform can identify novel allosteric binding sites on disease-implicated proteins, pinpointing pockets that cannot be found or drugged with current technologies. Magellan is the next generation of Gain’s original SEE-Tx® (Site-Directed Enzyme Enhancement Therapy) platform, which was enhanced and expanded with new AI and machine-learning tools and virtual screening capabilities to access the emerging on-demand compound libraries covering vast chemical spaces of over 50 billion compounds. Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders and oncology. For more information, please visit GainTherapeutics.com and follow us on LinkedIn. Cautionary Note Regarding Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. All statements in this press release other than statements of historical facts are “forward-looking statements”. In some cases, you can identify these statements by forward-looking words such as "may," "might," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "predict," "goal, " "intend," "seek, " "potential" or "continue," the negative of these terms and variations of these words or similar expressions that are intended to identify forward-looking statements, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements regarding: the development of the Company’s current or future product candidates including GT-02287; expectations regarding the timing of results from a Phase 1 clinical trial for GT-02287 and the treatment of Parkinson’s patients in that clinical trial; the potential therapeutic and clinical benefits of the Company’s product candidates including GT-02287; and the execution of potential business development transactions and the effects thereof on the financial position of the Company. These forward-looking statements are based on the Company’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause the Company’s preclinical and future clinical development programs, future results or performance to differ materially from those expressed or implied by the forward-looking statements. These statements are not historical facts but instead represent the Company's belief regarding future results, many of which, by their nature, are inherently uncertain and outside the Company's control. Many factors may cause differences between current expectations and actual results, including the impacts of the post-COVID-19 environment and other global and macroeconomic conditions on the Company’s business; clinical trials and financial position; unexpected safety or efficacy data observed during preclinical studies or clinical trials, clinical trial site activation or enrollment rates that are lower than expected; changes in expected or existing competition; changes in the regulatory environment; the uncertainties and timing of the regulatory approval process; and unexpected litigation or other disputes. Other factors that may cause the Company’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are identified in the section titled “Risk Factors,” in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 23, 2023 and its other documents subsequently filed with or furnished to the Securities and Exchange Commission from time to time. All forward-looking statements contained in this press release speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Contacts: CORE IR(516) 222-2560 ir@gaintherapeutics.com