Restless Legs Syndrome

Telix Pharmaceuticals continued its acquistion spree with a deal worth up to $250 million for Florida-based RLS Radiopharmacies. Telix Pharmaceuticals tallied its third acquisition of the year with a deal worth up to $250 million for Florida-based RLS Radiopharmacies, expanding the Australia-based company’s footprint in the radiopharmaceuticals space.The deal, which remains contingent on regulatory, shareholder and certain third-party approvals, is expected to close in the first quarter of next year, RLS said in a Sept. 23 press release.With the acquisition, Telix gets access to RLS’s network of 31 radiopharmacies spread out over 18 U.S. states. Last fall, RLS expanded several of its U.S. sites to add contract development and manufacturing capabilities.Under terms of the sale, RLS will receive an upfront cash payment of $230 million and an additional $20 million upon reaching certain milestones during the four quarters following the closing.The acquisition of RLS marks the third purchase by Telix since the start of the year. In February, Telix announced it would shell out $13.6 million in a cash and stock deal to snap up Texas-based CDMO Iso Therapeutics. In the past 2 years, Telix said it has spent about $1 billion on acquisitions, with most of the money going for U.S. companies.That sale was followed up in March with an $82 million cash and stock offer for ARTMS, a British Columbia-based company with a cyclotron-based isotope production platform. Along with that deal, Telix picked up a manufacturing plant and a stockpile of ultrapure rare metals. ARTMS was formed in 2013 as a spinout from Canada’s national particle accelerator center.“Our vision is to build a radiometal production and distribution network fit for the future,”  Dr. Christian Behrenbruch, Telix managing director and group chief executive officer, said in Monday's release. “By combining the ARTMS platform and the RLS network, we can scale up the production of key isotopes and build a stable and consistent supply of PET and SPECT diagnostic tracers, along with therapeutic radiopharmaceuticals across the U.S. for the benefit of Telix, our partners and the patients we serve.” Telix had been considering a move to go public in June, however, the company dropped its plan for a $200 million IPO debut on the Nasdaq later that month. The company has been listed on the Australian Securities Exchange since 2017.The biopharma industry has been in a fever rush to get into the radiopharmaceuticals space. In October 2023, Eli Lilly shelled out $1.4 billion to snap up Point Biopharma. Later in the year, BMS picked up RayzeBio for more than $4 billion.But radiopharmaceuticals are tricky to produce. The radioactive components of the drugs have a very short shelf-life and must be produced in a customized fashion, thereby requiring regional manufacturing and same-day delivery services.

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

The deal is expected to close in the fourth quarter and will not weigh on UCB’s 2024 financial guidance, the company explained in a release. Restructuring seems to be the name of the game for many large drugmakers these days, and Belgium-based UCB is no exception.Early Monday, UCB revealed that it’s selling off its mature neurology and allergy business in mainland China to local healthcare asset manager CBC Group and Abu Dhabi-based investment firm Mubadala. UCB is in line to receive $680 million from the transaction, which covers the seizure drugs Keppra and Vimpat, Neupro for Parkinson’s disease and restless leg syndrome, and the allergy meds Zyrtec and Xyzal. UCB is also pawning off a manufacturing site in the city of Zhuhai in China’s Guangdong Province as part of the divestment deal, the company said in a release.  UCB says the deal will help more patients benefit from the company’s past innovations, noting that CBC and Mubadala are partnering to develop and operate a “new leading neurology” company at scale in China leveraging UCB’s drug portfolio and factory. "In the short term, UCB is exploring the launch of novel medicines in immunology, neurology, and rare diseases in China," the company’s CEO, Jean-Christophe Tellier, said in a statement. “Building on our 28-year presence in the country, we are committed to driving patient outcomes through continued collaboration with local partners and fostering innovation.” The deal is expected to close in the fourth quarter and will not weigh on UCB’s 2024 financial guidance, the company added. UCB’s divestment follows a similar trend in which large pharmas have recently opted to enlist local partners to help commercialize their medicines in China. Pfizer in November inked a deal with Keyuan Pharma, granting the Shanghai drugmaker exclusive Chinese rights to distribute and promote its pneumococcal vaccine Prevenar 13, which goes by the name Prevnar 13 in the United States. Prior to that, GSK tapped Chongqing Zhifei Biological Products—the China marketer of Merck’s HPV short Gardasil—to distribute its shingles vaccine Shingrix in the country. Biogen and Sanofi have also reworked parts of their China businesses in recent months. Leveraging local partners in China has been on the upswing lately, partly because “there is increasing pricing and competitive pressure in the market, especially for mature products, leaving reduced [return on investment] for in-house commercial resources,” Justin Wang, head of L.E.K Consulting’s China practice, told Fierce Pharma last year.  Meanwhile, certain drugmakers—especially in the U.S.—are considering shying away from Chinese partners in the wake of the BIOSECURE Act, which aims to block certain Chinese biotech equipment and service providers from the U.S. market over national security concerns. Among U.S.-based life sciences companies, confidence in working with Chinese companies has plunged, according to a L.E.K. survey released earlier this summer. The issue is less pronounced with companies outside the U.S., where L.E.K. reported a milder decrease in confidence levels.