Spinal Cord Compression

TUESDAY, Sept. 24, 2024 -- In a clinical review conducted by an international committee of multiple sclerosis (MS) experts in pediatric and adult MS and published online Sept. 16 in JAMA Neurology, consensus guidance is provided for diagnosing pediatric and late-onset MS. Le H. Hua, M.D., from the Lou Ruvo Center for Brain Health at the Cleveland Clinic in Las Vegas, and colleagues provide guidance on approaches to differential diagnosis in suspected MS specific to those aged younger than 18 years and older than 50 years. The authors note that compared with typical adult-onset MS, unique biological factors are seen in children younger than 18 years and adults older than 50 years. These biological differences may influence clinical presentation, resilience to neuronal injury, and differential diagnosis. Children have high immunological reactivity, increasing the likelihood of other mimicking disorders; consequently, MS needs to be differentiated from other neuroimmunological conditions. The clinical disease course of MS is distinct in children; progressive onset at presentation should warrant investigation for alternative diagnoses. Children are likely to reach disability milestones at earlier ages than adults, underscoring the need for early diagnosis to allow for timely intervention. In older adults, MS should be differentiated from more common conditions such as vascular disease, spinal stenosis/compressive myelopathy, and arthritis. Since inflammatory activity is reduced during older age, relapses triggered by inflammation occur less often, but disability progression tends to accelerate. Nonspecific white matter changes in the brain in older populations are more likely due to vascular disease or migraines, potentially leading to misdiagnosis of MS. "There are important implications for when MS treatment begins, and improving diagnostic approach is an important step forward in providing tailored care as our research into these patient subsets continues," Hua said in a statement. Several authors disclosed ties to the biopharmaceutical industry.

One oral presentation and nine posters will shed light on real-world experiences of people living with XLH and the impact of burosumab treatment TOKYO, Sept. 19, 2024 /PRNewswire/ -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE:4151) will share new research that enhances understanding about the use of burosumab in real-world clinical practice as well as the burden of those living with X-linked hypophosphatemia (XLH), a rare genetic metabolic bone disease. The findings will be presented at the American Society for Bone and Mineral Research (ASBMR) 2024 annual meeting, which takes place September 27-30 in Toronto. The oral presentation of "Patient reported outcomes (PRO) from a real-world study of burosumab treatment in adults with X-linked hypophosphatemia in the UK" will be made by Judith Bubbear, MD, Royal National Orthopaedic Hospital (UK) on Saturday, September 28, 11:30 am EDT (Presentation #1022). "As we learn more about XLH and how it impacts people's lives, real-world evidence can provide much-needed insight into potential strategies for managing this debilitating disease," said Angela Williams, PhD, Vice President and Global Head of Health Economics and Outcomes Research at Kyowa Kirin. "The breadth of our ASBMR data reflects our continued focus on advancing the science and understanding of XLH and improving the lives of people living with XLH." ASBMR XLH poster presentations: Real-world impact of burosumab treatment in people living with XLH: "Effectiveness of burosumab versus conventional therapy in adults with X-linked hypophosphatemia in a real-world setting from the XLH Disease Monitoring Program" (Poster#SAT-430) Lead author: Pablo Florenzano, MD, Pontificia Universidad Católica de Chile Poster Session I: Saturday, September 28, 2:15 – 3:45 pm EDT "Biochemical measurements according to age group in burosumab-treated patients with X-linked hypophosphatemia (XLH) in a real-world setting: an analysis of the XLH Disease Monitoring Program" (Poster #Fri-424, #Sun-424) Lead author: Leanne M. Ward, MD, Children's Hospital of Eastern Ontario Welcome Reception/Plenary Poster Session: Friday, Sept. 27, 5:30 – 7:30 pm EDT Poster Session II; Sunday, Sept. 29, 2:15 – 3:45 pm EDT "Symptoms experienced by adolescents living with X-linked hypophosphatemia at the end of skeletal growth (EOSG) treated with burosumab—a mixed methods analysis" (Poster #SAT-445) Lead author: Vrinda Saraff, MD, Birmingham Women's and Children's Hospital (UK) Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT "Efficacy and safety of burosumab in adult Chinese patients with X-linked hypophosphatemic rickets/osteomalacia (XLH): an open-label, multi-center, single-cohort, post-marketing phase IV study" (Poster#SUN-119) Lead author: Wei Liu, MD, Peking Union Medical College Hospital Poster Session II: Sunday, Sept. 29, 2:15 – 3:45 pm EDT "Efficacy and safety of burosumab in pediatric Chinese patients with X-linked hypophosphatemic rickets/osteomalacia (XLH): an open-label, multi-center, single-cohort, post-marketing phase IV study" (Poster#SAT-121) Lead author: Xiaoping Luo, MD, Tongji Medical College, Huazhong University of Science and Technology Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT XLH burden on multiple aspects of patients' lives: "Association between pain medication use and patient-reported outcomes in adults with X-linked hypophosphatemia: an exploratory analysis of a phase 3 study" (Poster #Sat-025) Lead author: Angela Williams, PhD, Kyowa Kirin International Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT "Individuals with hereditary hypophosphatemia are not prone to early death despite significant earlier development of co-morbidities: a retrospective Danish register study" (Poster #Sun-024) Lead author: Signe Sparre Beck-Nielsen, MD, PhD, Aarhus University Hospital (Denmark) Poster Session II: Sunday, Sept. 29, 2:15 – 3:45 pm EDT "X-linked Hypophosphatemia Community Impact Survey: psychosocial health, symptoms, and self-care" (Poster #SAT-LB 543) Lead author: Jill Simmons, MD, Vanderbilt University Medical Center Late Breaking Poster Session I: Saturday, Sept. 28, 2:15 – 3:45 pm EDT Bridging evidence gaps to improve clinical decision-making in XLH: "Advancing Patient Evidence in XLH (APEX): rational and design of real-world XLH global data unification program" (Poster #SAT-427) Lead author: Maria Luisa Brandi, MD, PhD, FIRMO Foundation, Florence, Italy and the University Vita-Salute San Raffaele, Milan, Italy Poster Session I: Saturday, Sep 28, 2:15-3:45 pm EDT About X-linked hypophosphatemia X-linked hypophosphatemia is a rare, lifelong, genetic disease that can impact the bones and muscles in both children and adults. In individuals with XLH, the body doesn't hold on to enough phosphorus, which is an essential mineral for bone health. This is due to the production of excess fibroblast growth factor 23 (FGF23), causing the body to release too much phosphorus through the urine. When phosphorus levels are too low (hypophosphatemia), it can cause the softening and weakening of growing bones in children (rickets) and of mature bones in adults (osteomalacia). In children, XLH typically appears as bowed legs or knock knees. Over time, bone weakening can also lead to bone abnormalities in the legs, delayed growth, and short stature. In adults, XLH may cause osteomalacia, fractures and pseudo-fractures, and hypophosphatemia. About CRYSVITA® (burosumab-twza) Injection CRYSVITA is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, which binds to and inhibits the biological activity of FGF23, the underlying cause of hypophosphatemia in XLH. By blocking FGF23, CRYSVITA helps to restore phosphorus reabsorption in the kidneys and increase the production of active vitamin D, which enhances intestinal absorption of phosphate and calcium. U.S. CRYSVITA Indication CRYSVITA is a fibroblast growth factor (FGF23) blocking antibody indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older. Important Safety Information CONTRAINDICATIONS CRYSVITA is contraindicated: In concomitant use with oral phosphate and/or active vitamin D analogs (e.g., calcitriol, paricalcitol, doxercalciferol, calcifediol) due to the risk of hyperphosphatemia. When serum phosphorus is within or above the normal range for age. In patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism. WARNINGS AND PRECAUTIONS Hypersensitivity Hypersensitivity reactions (e.g., rash, urticaria) have been reported in patients with CRYSVITA. Discontinue CRYSVITA if serious hypersensitivity reactions occur and initiate appropriate medical treatment. Hyperphosphatemia and Risk of Nephrocalcinosis Increases in serum phosphorus to above the upper limit of normal may be associated with an increased risk of nephrocalcinosis. For patients already taking CRYSVITA, dose interruption and/or dose reduction may be required based on a patient's serum phosphorus levels. Injection Site Reactions Administration of CRYSVITA may result in local injection site reactions. Discontinue CRYSVITA if severe injection site reactions occur and administer appropriate medical treatment. ADVERSE REACTIONS Pediatric Patients Adverse reactions reported in 10% or more of CRYSVITA-treated pediatric XLH patients across three studies are: pyrexia (55%, 44%, and 62%), injection site reaction (52%, 67%, and 23%), cough (52%), vomiting (41%, 48%, and 46%), pain in extremity (38%, 46%, and 23%), headache (34% and 73%), tooth abscess (34%, 15%, and 23%), dental caries (31%), diarrhea (24%), vitamin D decreased (24%, 37%, and 15%), toothache (23% and 15%), constipation (17%), myalgia (17%), rash (14% and 27%), dizziness (15%), and nausea (10%). Postmarketing experience reported in CRYSVITA-treated pediatric XLH patients: blood phosphorus increased. Adult Patients Adverse reactions reported in more than 5% of CRYSVITA-treated adult XLH patients and in at least 2 patients more than placebo in one study are: back pain (15%), headache (13%), tooth infection (13%), restless legs syndrome (12%), vitamin D decreased (12%), dizziness (10%), constipation (9%), muscle spasms (7%), and blood phosphorus increased (6%). Spinal stenosis is prevalent in adults with XLH, and spinal cord compression has been reported. It is unknown if CRYSVITA therapy exacerbates spinal stenosis or spinal cord compression. USE IN SPECIFIC POPULATIONS There are no available data on CRYSVITA use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-844-768-3544. There is no information regarding the presence of CRYSVITA in human milk or the effects of CRYSVITA on milk production or the breastfed infant. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for CRYSVITA and any potential adverse effects on the breastfed infant from CRYSVITA or from the underlying maternal condition. PATIENT COUNSELING INFORMATION Advise patients not to use any oral phosphate and/or active vitamin D analog products. Instruct patients to contact their physician if hypersensitivity reactions, injection site reactions, and restless legs syndrome induction or worsening of symptoms occur. You may report side effects to the FDA at (800) FDA-1088 or . You may also report side effects to Kyowa Kirin, Inc. at 1-844-768-3544. For important risk and use information, please see the full Prescribing Information for CRYSVITA. About Kyowa Kirin Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato oncology, and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites us across the globe. Learn more about Kyowa Kirin at: kyowakirin.com. COR-US-CRY-0049 September 2024 SOURCE Kyowa Kirin WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

TORONTO and HAIFA, Israel, Sept. 06, 2024 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX), (OTCQB: NRXBF), (Germany: J90) (the “Company” or “NurExone”) is pleased to announce compelling new findings that highlight the therapeutic potential of ExoPTEN for patients with spinal cord injuries. In a recent preclinical study using a spinal cord compression model, our team demonstrated that ExoPTEN has a strong ability to target and accumulate at the injury site, even when administered up to one week after the injury occurred. This finding is crucial because it suggests a long window of time in which treatment can be effectively administered. Dr. Lior Shaltiel, NurExone Chief Executive Officer, emphasized the real-world significance of this capability by stating that “the ability to treat patients up to 7 days post-injury could broaden the range of patients eligible for treatment and extend the window of effectiveness, leading to enhanced recovery. Moreover, the findings can enhance significantly the ability to recruit more patients to clinical trials and to expand the numbers of treatable patients, without being limited by a short therapeutic window and hospital administration challenges." He continued, "With the global incidence of spinal cord injury estimated between 250,000 and 500,000i cases annually and given that some patients do not receive immediate treatment, the potential market for a therapy effective up to 1-week post-injury could be substantial." As shown in Figure 1, the ExoPTEN was labelled with a fluorescent mark and administered to rats with induced spinal cord compression injuries. The administration was conducted at four different time points: on the day of injury (day 0), 3 days later, 5 days later, and 7 days later, and compared to each other and to an untreated control group. The goal was to evaluate how well ExoPTEN targets and accumulates at an injury site over time. Using an advanced In Vivo Imaging System (“IVIS”), it was observed that ExoPTEN consistently accumulated at the injury site. A notable gradient of homing capacity was observed, with later administration times resulting in progressively higher levels of accumulation. The highest accumulation was seen in those treated 7 days post-injury with a statistically significant dose-dependent accumulation of ExoPTEN at the injury site. These results underscore the exceptional homing capacity of ExoPTEN, even 7 days post-injury, suggesting a broad therapeutic window for intervention. This creates new possibilities for the timing and flexibility of treatment, enhancing the potential for recovery in patients with spinal cord injuries. Dr. Noa Avni, Director of research and development stated that “we are excited about the implications of these findings for our phase I/II clinical trial design and patient care. The extended therapeutic window we have demonstrated not only highlights the potency of our exosome-based therapy but also offers hope for adaptable treatment regimens in clinical settings." Figure 1: Quantification and Distribution of ExoPTEN in Rat Spinal Cords Following Minimal-Invasive Administration Post-Spinal Cord Injury About NurExone NurExone Biologic Inc. is a TSX Venture Exchange (“TSXV”) and OTCQB 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 by the 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 ShaltielChief Executive Officer and DirectorPhone: +972-52-4803034Email: info@nurexone.com Thesis Capital Inc.Investor Relations - CanadaPhone: +1 905-347-5569Email: IR@nurexone.com Dr. Eva ReuterInvestor Relations - GermanyPhone: +49-69-1532-5857Email: e.reuter@dr-reuter.eu Allele Capital PartnersInvestor Relations - USPhone: +1 978-857-5075Email: aeriksen@allelecapital.com FORWARD-LOOKING STATEMENTS This press release contains certain “forward-looking statements” that reflect the Company’s current expectations and projections about its future results. 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. Forward-looking statements in this press release include, but are not limited to, statements relating to the results and implications of the study; the timeframe for the administration of treatment and the potential eligibility of patients; the treatment leading to enhanced recovery; there being a broad therapeutic window for intervention; there being adaptable treatment regimens in clinical settings; the Company’s ExoPTEN nanodrug being a potential treatment for acute spinal cord injuries and other central nerve system indications; the Company engaging with collaboration partners, industry leaders, researchers and innovators; and the NurExone platform technology offering novel solutions to drug companies interested in noninvasive targeted drug delivery for other indications. These statements reflect management’s current beliefs and are based on information currently available to management as at the date hereof. In developing the forward-looking statements in this press release, we have applied several material assumptions, including the general business and economic conditions of the industries and countries in which we operate; the general market conditions; the ability to secure additional funding; partnerships having their intended impact on the Company and its business; patents safeguarding NurExone’s technology; the Company’s drug products having its intended benefits and effects; the Company making progress through new partnerships and technologies to move towards commercialization of their products; the Company’s intellectual property and technology being novel and inventive; the intellectual property having the intended impact on the Company and its business; exosomes becoming an ideal and natural choice for drug delivery; the Company making advancements in the manufacturing process of exosomes; exosomes holding immense promise for regenerative medicine; the Company’s production methods continuing to be reliable; the Company will have flexibility in optimizing its exosome production method; exosomes will serve as an excellent, targeted system for drug delivery; the Company will pave the way to regenerative medicine treatments for a variety of clinical indications by the Company and with future collaboration partners; the Company’s ExoPTEN nanodrug being a potential treatment for acute spinal cord injuries and other central nerve system indications; the Company will engage with collaboration partners, industry leaders, researchers and innovators; the study will yield its intended results and have its intended implications on the Company and its business; ExoPTEN will have a longer timeframe for the administration of treatment and therefore there will be additional eligible patients; the treatment will lead to enhanced recovery; there will be a broad therapeutic window for intervention; there will be an adaptable treatment regimens in clinical settings; and the NurExone platform technology offering novel solutions to drug companies. 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. These 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; dependence on the Company’s strategic partners; the fact that preclinical drug development is uncertain, and the drug product candidates of the Company may never advance to clinical trials; the fact that results of preclinical studies and early-stage clinical trials may not be predictive of the results of later stage clinical trials; the uncertain outcome, cost, and timing of product development activities, preclinical studies and clinical trials of the Company; the uncertain clinical development process, including the risk that clinical trials may not have an effective design or generate positive results; the potential inability to obtain or maintain regulatory approval of the drug product candidates of the Company; the introduction of competing drugs that are safer, more effective or less expensive than, or otherwise superior to, the drug product candidates of the Company; the initiation, conduct, and completion of preclinical studies and clinical trials may be delayed, adversely affected or impacted by unforeseen issues; the potential inability to obtain adequate financing; the potential inability to obtain or maintain intellectual property protection for the drug product candidates of the Company; the NurExone platform technology being unable to offer novel solutions to drug companies; risks that the Company’s intellectual property and technology won’t have the intended impact on the Company and/or its business; the Company’s inability to realize upon partnerships; risk that the exosomes will not become an ideal and/or natural choice for drug delivery; risk that the company will be unable to make advancements in the manufacturing process of exosomes; risk that exosomes will not be a viable option in regenerative medicine; risk that the Company’s production methods will become unreliable; risk that the Company will not have flexibility in optimizing its exosome production method; risk that exosomes will not serve as a targeted system for drug delivery; risk that the Company will be unable to pave the way to regenerative medicine treatments for a variety of clinical indications by the Company and/or with future collaboration partners; risk that the Company’s ExoPTEN nanodrug will not work as a potential treatment for acute spinal cord injuries and/or other central nerve system indications; risk that the Company will be unable to engage with collaboration partners, industry leaders, researchers and/or innovators at the conferences or at all; risk that the NurExone platform technology will be unable to offer novel solutions to drug companies interested in noninvasive targeted drug delivery for other indications; risk that the study will not yield its intended results and/or have its intended implications on the Company and its business; risk that the timeframe for the administration of treatment will not be longer and/or will not lead to more eligible patients; risk that the treatment will not lead to enhanced recovery; risk that there will not be a broader therapeutic window for intervention; risk that there will not be adaptable treatment regimens in clinical settings; and the risks discussed under the heading “Risk Factors” on pages 29 to 36 of the Company’s Annual Information Form dated March 30, 2023, a copy of which is available under the Company’s SEDAR+ profile at www.sedarplus.ca. 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 TSXV nor its Regulation Services Provider (as that term is defined in the policies of the TSXV) accepts responsibility for the adequacy or accuracy of this release. _____________________ihttps://iris.who.int/bitstream/handle/10665/94190/9789241564663_eng.pdf?sequence=1 A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/6349d08a-be42-434d-9a78-e2158d074d9b