CD49d

GIVI-MPC targets site of muscle injury via direct injection and systemic delivery and creates new functional skeletal muscle for treatment of both Sarcopenia & Duchenne Muscular Dystrophy in aged mice HOUSTON--(BUSINESS WIRE)-- IPS HEART, a privately held cell therapy company advancing its stem cell platform to develop new skeletal muscle and cardiac muscle generation treatments for Duchenne Muscular Dystrophy (a rare disease) and heart failure, today announced the peer-reviewed publication of in vivo and in vitro study with its GIVI-MPC stem cell therapy candidate in this month's issue of Cells (volume 12, issue 14). The study, “Chemokine/ITGA4 Interaction Directs iPSC-Derived Myogenic Progenitor Migration to Injury Sites in Aging Muscle for Regeneration,” demonstrated GIVI-MPC stem cell therapy’s ability to successfully differentiate and create new human skeletal muscle in extremely aged 18-month aged mice for sarcopenia repair. Sarcopenia is characterized by muscle mass loss and function decline with age and poses a threat to the quality of lifelong health as well as the general well-being of the elderly population. Weaker muscles with low mass and function are likely more susceptible to muscle damage and injury. Studies suggest that muscle mass decreases by about 3 to 8 percent by decade after age 30. Thus far, there is no effective therapeutic approved for muscle loss during aging. “During aging, increased inflammation and oxidative damages in skeletal muscle tissues occur,” said Rauf Ashraf, Chief Executive Officer of IPS HEART. “Given that GIVI-MPCs possess anti-oxidative and anti-inflammatory properties, and exhibit superior migration characteristics, GIVI-MPCs administered in 18-month aged NSG mice resulted in significant improved muscle regeneration, with hindlimb grip strength doubling and an over 30% reduction in fibrosis. We are thrilled with these significant results and are even more pleased that GIVI-MPC has been featured in a second peer reviewed publication.” IPS HEART previously reported that GIVI-MPC developed into new human skeletal muscle, and reduced fibrosis & necrosis upon transplantation in dystrophic mice and pigs. In the current publication, successful delivery via IV/systemic delivery and direct injection were both achieved as well as new skeletal muscle generation with full length human dystrophin developed in 16-month aged dystrophic mice. This development further validates the idea that the Company’s therapeutic candidate may not only slow the rate of decline as gene therapy approaches aim for, but more importantly can potentially help reverse Duchenne Muscular Dystrophy in both young and older DMD patients. Heart disease continues to kill more people annually than all forms of cancer combined. IPS HEART’s first stem cell therapeutic (ISX9-CPC) helps longevity by creating new heart functional heart muscle and when paired with GIVI-MPC, new skeletal muscle can be created for sarcopenia thus providing tangible aging therapeutics for improved longevity. About IPS HEART IPS HEART is advancing its stem cell platform to develop new skeletal & cardiac muscle generation therapies.

7 Hills Pharma LLC (“7 Hills” or “7HP”) announced today that it has been awarded $13.5MM in nondilutive grant funds from the Cancer Prevention & Research Institute of Texas (CPRIT) to advance 7HP349, its first-in-class oral selective integrin activator into a Phase 1b/2a clinical trial in patients who are resistant to PD-1-based therapies. The new CPRIT funds follow over $13MM in federal grant awards, in collaboration with world-class institutions such as The Texas Heart Institute, the University of Texas MD Anderson Cancer Center, the University of Texas Health Science Center, and the Icahn School of Medicine, that supported the development of the platform technology from 7 Hills and fully funded a Phase 1 clinical trial of 7HP349. Immune checkpoint inhibitors, such as KEYTRUDA® (pembrolizumab) and OPDIVO® (nivolumab), command global sales of over $30 billion per annum due to their curative potential. However, up to 90% of patients with solid tumors were found not to respond, leaving these patients with limited treatment options. Alintegimod, also known as 7HP349, has the potential to offer a novel mechanism to safely overcome resistance to a broad range of immunotherapies, including anti-tumor immune checkpoint inhibitors and infectious disease vaccines. The CPRIT-funded clinical trial will test the ability of 7HP349 to augment T-cell activation in patients with checkpoint blockade-resistant tumors, a novel approach that may offer patients a compelling treatment option with no additional toxicities. “Developing first-in-class therapeutics, especially in Houston, is a challenging task,” said Dr. Michael S. Perry, chairman of the 7 Hills Board of Managers. “CPRIT’s support of early-stage Texas talent and technology is critical to enabling next-generation innovations like alintegimod to thrive in our underserved regional venture capital ecosystem and to attract follow-on investment and strategic partnering.” 7 Hills Chief Medical Officer Lionel Lewis, MB BCh., MD, added, “We are grateful for CPRIT’s support as we bring our revolutionary technology and novel mechanism of action to patients with advanced solid tumors for the first time. 7 Hills is inspired to finally deliver the full promise of immunotherapy and we are excited to demonstrate alintegimod’s distinctive benefits when combined with checkpoint blockade.” About 7 Hills Pharma LLC 7 Hills Pharma is a clinical-stage immunotherapy company developing a platform of novel, oral small molecules for the treatment of cancer and prevention of infectious diseases. Our compounds are first-in-concept allosteric integrin activators that leverage a unique mechanism of action to stabilize the cell-cell interactions required to mount an effective immune response. Our lead clinical candidate, 7HP349 (also known as alintegimod), is the only reported systemically safe immune stimulant that can activate both cellular and humoral immunity. For more information, visit 7hillspharma.com About Alintegimod Alintegimod, also designated 7HP349, is the lead, clinical-stage integrin agonist from 7 Hills Pharma. It is a first-in-class, orally delivered small molecule that safely and selectively activates the integrins LFA-1 and VLA-4 to augment antigen-specific immune responses. In preclinical models, alintegimod has been shown to improve the effectiveness of a broad range of immune checkpoint inhibitors and a wide variety of infectious disease vaccines. In a Phase 1 clinical trial, alintegimod demonstrated oral bioavailability and a clean safety profile at exposures well exceeding therapeutic levels. The content above comes from the network. if any infringement, please contact us to modify.

World first study utilizing Somalogic SomaScan® assay# to assess up to 7,000 plasma proteins in Long COVID-19 patients has elucidated novel blood markers as potential diagnostic and therapeutic targetsProvisional patent applications have been filed in the United States (US) to seek protection for these new inventionsA potential therapeutic marker known to be modulated by ATL1102 in DMD patients has been identified as suggestive of its therapeutic potential as a treatment for Long COVID-19Collaboration with global leader in the clinical research of neurological aspects of Long COVID-19 Dr Koralnik to continue with application for grant fundingCompany plans to review its new patent applications with targeted pharmaceutical and diagnostic companies for potential commercial discussions New York, NY, Aug. 22, 2022 (GLOBE NEWSWIRE) -- Antisense Therapeutics Limited [ASX: ANP | US OTC: ATHJY | FSE: AWY] (the Company) is pleased to advise of outcomes from its collaboration to study the neurological aspects of Long COVID-19 (Long Neuro COVID-19) with US based researchers led by global leader in the field, Dr Igor Koralnik, at the Northwestern Medicine Neuro-COVID clinic in Chicago, USA. The study has elucidated novel blood markers as potential diagnostic and therapeutic targets in the treatment of Long COVID-19 patients. Three (3) provisional patent applications have been filed in the US to seek protection for these new inventions.* Under the collaboration, blood samples that had been collected from Long COVID-19 patients who had not been hospitalized (focused on those with neurological symptoms including brain fog, where blood immune cell changes were observed1), were used to generate data on up to 7,000 proteins in the blood utilising a large-scale protein analysis known as proteomics. Industry leading proteomics group Somalogic in Boulder Colorado USA undertook the analysis, successfully testing the samples using their SomaScan® assay and then the data was statistically analyzed using their Dataviz program2. The analyzed data has identified a number of proteins that are significantly modulated in the blood of Long Neuro COVID-19 patients when compared to convalescent subjects who had recovered from Long COVID-19 infection with no persistent symptoms and to healthy subjects. This data has been included in recently filed patent applications as potential diagnostic and therapeutic targets for the treatment of Long COVID-19. Certain targets when combined (as few as 5) identified all 48 Neuro Covid-19 patients and the 42 of 44 subjects who were convalescent or healthy controls suggestive of these targets’ diagnostic potential. A number of targets (<15) have been identified as potentially amenable to treatment by currently available drugs or other therapeutic approaches on the market. The mechanisms of action of those drugs are known to modulate the discovered target proteins, therefore the marketers/developers of those drugs have been identified as initial prospects for partnering interest. A smaller number of diagnostic markers have been detected that could assist in the identification of Neuro Long Covid patients for better designed clinical trials and potentially for earlier treatment intervention. Accordingly, the Company also plans to review its newly generated intellectual property (IP) with targeted pharmaceutical and diagnostic companies for potential commercial discussions, noting that for these discussions to progress, the Company and potential partner companies would need to agree on licensing and/or joint development of this newly generated IP to advance as either diagnostic or therapeutic programs. Of the 94.7 million people in the US diagnosed as infected and surviving COVID-193, approximately 82 million (87%) people are non-hospitalized4, and 45% of non-hospitalized patients5 have developed some manifestation of Long COVID-19 syndrome which suggests more than 24 million people are afflicted by the condition to some extent. The main neurological symptom is brain fog (defined with the established memory tests conducted) and reported in 81% suggesting an impact on nearly 20 million people in the US. Identification of appropriate biomarkers of Long COVID-19 have proved elusive.6 The National Institute of Health (NIH) in the US is funding a national research effort focused on understanding and treating Long COVID-19 beyond US$1billion it has already committed.7 One of the aims of the proteomics analysis was to assess if Neuro Long COVID-19 patients may have been amendable to treatment with ANP’s immunomodulatory drug ATL1102 which has previously demonstrated biologic activity in MS patients11 and the ability to reduce T cells and modulate proteins involved in the blood of DMD patients (data presented at the 2021 World Muscle Society conference WMS-ATL1102-DMD-PROTEOMICS-Poster). Encouragingly, one of the potential therapeutic markers in Long COVID-19 patients identified from this proteomics analysis is also known as having the potential to be significantly modulated by ATL1102 in DMD patients and therefore is suggestive of its therapeutic potential in Long COVID-19. The Company is looking to further explore the clinical potential of ATL1102 in this setting via applying for grant funding opportunities (such as that as offered by NIH) in collaboration with Professor Koralnik. Dr Koralnik said, “The collaboration with Antisense Therapeutics has generated promising novel data in Long COVID-19 patients in identifying potential disease biomarkers and represents an important advance towards the goal of establishing effective disease diagnostics and interventional treatments. We look forward to continuing our scientific collaboration with Antisense Therapeutics and to advancing such endeavors through our active involvement and support in seeking grant applications including with bodies such as the NIH.” Dr George Tachas Director of Drug Discovery at Antisense Therapeutics said, “We are delighted to report on the initial outcomes from this novel and leading scientific collaboration with Professor Koralnik and his team. Our data has identified potential new avenues towards diagnoses and treatment of a disease that has negatively impacted the lives of over a hundred million people around the world. We look forward to continuing scientific advancements in the space in collaboration with Professor Koralnik and the further important IP that we anticipate emerging from this important scientific collaboration.” For further details please refer to the presentation following this announcement. This announcement has been authorised for release by the Board. For more information please contact: Antisense TherapeuticsInvestment EnquiriesUS/European IR &PRMark DiamondGennadi KoutchinLaine Yonker/Joe GreenManaging DirectorXEC PartnersEdison Investor Relations+61 (0)3 9827 8999gkoutchin@xecpartners.com.aulyonker@edisongroup.comwww.antisense.com.au1300 932 037+1 646-653-7035 # ANP is the first Company to utilize Somalogic’s proprietary SomaScan® assay for the analysis of plasma proteins in Long COVID-19 patients. The SomaScan® assay is ‘the first and only platform that can simultaneously measure 7,000 proteins across a wide range of concentrations’. https://somalogic.com/ * Patent application names: “Biomarkers and Uses thereof” “Methods for treating neurological post-acute sequelae of COVID-19 (NPASC)” “Methods for diagnosing and treating neurological post-acute sequelae of COVID-19 (NPASC)” About Antisense Therapeutics Limited [ASX:ANP | US OTC:ATHJY | FSE:AWY], is an Australian publicly listed biotechnology company, developing and commercializing antisense pharmaceuticals for large unmet markets in rare diseases. The products are in-licensed from Ionis Pharmaceuticals Inc. (NASDAQ: IONS), an established leader in antisense drug development. The Company is developing ATL1102, an antisense inhibitor of the CD49d receptor, for Duchenne muscular dystrophy (DMD) patients and reported highly promising Phase II trial results. ATL1102 has also successfully completed a Phase II efficacy and safety trial, significantly reducing the number of brain lesions in patients with relapsing-remitting multiple sclerosis (RRMS). About ATL1102 ATL1102 is an antisense inhibitor of CD49d, a subunit of VLA-4 (Very Late Antigen-4). Antisense inhibition of VLA-4 expression has demonstrated activity in a number of animal models of inflammatory disease. ATL1102 has also shown to be very effective in reducing inflammatory brain lesions in patients with MS (Limmroth, V. et al Neurology, 2014; 83(20): 1780-1788) and recently delivered highly promising clinical results in patients with Duchenne muscular dystrophy (DMD) a rare and fatal muscle wasting disease where inflammation in the muscle leads to fibrosis and death of muscle tissue. About Long COVID-19 In the US of the first 80 million people diagnosed as infected and surviving COVID-193, approximately 30% of hospitalized patients9 and 45% of non-hospitalized patients5 have developed some manifestation of Long COVID-19 syndrome (i.e more than 24 million people). According to the US Centre for Disease Control and Prevention “Post-COVID conditions are associated with a spectrum of physical, social, and psychological consequences, as well as functional limitations that can present substantial challenges to patient wellness and quality of life”.10 Confirmed infections numbers which increases daily can be followed on these links below3. Long COVID-19 cases in Australia were recently estimated at 475,00011. A body of published evidence now demonstrates that the SARS-CoV-2 virus causes long COVID-19 with impacts that last by definition for 28 days post COVID-19 symptom onset1 and that can last for 12 weeks and well beyond9. Long COVID-19 can occur post severe COVID-19, and post mild to moderate COVID-19. In the US Long COVID-19 patients comprise 13% of subjects post severe COVID-19 who have previously being hospitalized and 87% of subjects post mild-moderate COVID-19 who have not been hospitalized. In the latter there is ~2:1 ratio of females to males, like in autoimmune disease multiple sclerosis and the main neurological symptoms reported were 81% brain fog (defined with the established memory tests conducted), 68% headache, 60% numbness/tingling 59% dysgeusia, 55% anosmia, and 55% myalgias, and additionally 85% experienced fatigue, which are not well understood.4 While COVID-19 incidence and severity is being reduced with vaccines numerous studies connect the SARS-CoV-2 virus infection to long term neurological conditions in both hospitalized and non-hospitalized patients after vaccination.12 References Visvabharathy L, and Koralnik, I.J et al “Neuro-COVID long-haulers exhibit broad dysfunction in T cell memory generation and responses to vaccination.” https://www.medrxiv.org/content/10.1101/2021.08.08.21261763v3https://somalogic.com/stats-data-viz/ and https://somalogic.com/life-sciences/ 7,000 analytes, 450,000+ samples run, >400 publications, >500 patents.https://coronavirus.jhu.edu/map.html and https://www.worldometers.info/coronavirus/country/us/ as of 16 August 2022, 94.7 million people in the USA were diagnosed with COVID-19, 1.063 million deaths have been recorded, and 89.9 million people have recovered.Graham E.L and Koralnik I.J et al. “Persistent neurological symptoms and cognitive dysfunction in non-hospitalized COVID-19 “Long Haulers”. Ann. Clinical and Translational Neurol 2021, 8(5):1073-1085 https://onlinelibrary.wiley.com/doi/epdf/10.1002/can3.51350 Estiri H et al “Evolving phenotypes of non-hospitalized patients that indicated long COVID”. BMC Medicine (2021) 19: 249 https://doi.org/10.1186/s12916-021-02115-0https://www.msn.com/en-us/health/medical/brain-fog-and-other-long-covid-symptoms-can-last-more-than-a-year-according-to-study/vi-AA10gBishttps://www.cbsnews.com/news/long-covid-research-funding/. Limmroth V, Barkhof F, Desem N, Diamond M.P and Tachas G. “CD49d antisense drug ATL1102 reduces disease activity in patients with relapsing-remitting MS”. Neurology, 2014; 83(20): 1780-1788) https://pubmed.ncbi.nlm.nih.gov/25239835/Chen Chen et al “Global prevalence of Post-Acute Sequelae of COVID-19 (PASC) or Long COVID: A meta-Analysis and Systematic Review. https://www.medrxiv.org/content/10.1101/2021.11.15.21266377v1https://www.cdc.gov/coronavirus/2019-ncov/hcp/clinical-care/post-covid-clinical-eval.htmlhttps://www.abc.net.au/news/2022-08-11/long-covid-and-chronic-fatigue-syndrome-pathology-overlap/101318522Ledford H “Do vaccines protect against Long COVID? What the data say”, Vaccines reduce the risk of developing COVID-19- but studies disagree on their protective effect against long COVID. 23November 2021 https://www.nature.com/articles/d41586-021-03495-2“Mass disability event” warning as huge numbers diagnosed with Long Covid” Matt Young https://www.news.com.au/technology/science/human-body/mass-disability-event-warning-as-huge-numbers-diagnosed-with-long-covid/news-story/d541286fc71ac1b2a262ac798238d4ec