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HONG KONG, Dec. 23, 2024 /PRNewswire/ -- A research led by Hong Kong Baptist University (HKBU) and the Shanghai Sixth People's Hospital Affiliated to School of Medicine at Shanghai Jiao Tong University (Shanghai Sixth People's Hospital) has discovered that an aptamer developed by HKBU can be used to treat X-linked hypophosphatemia (XLH), a rare bone disease. The aptamer, originally developed to treat osteogenesis imperfecta, has been granted Orphan Drug Designation and Pediatric Rare Disease Designation by the U.S. Food and Drug Administration (FDA). Continue Reading Professor Zhang Ge, Associate Dean (Research) of Chinese Medicine and Director of the Law Sau Fai Institute for Advancing Translational Medicine in Bone and Joint Diseases at HKBU, in collaboration with other institutions, has developed an aptamer for the treatment of X-linked hypophosphatemia, which has been granted Orphan Drug Designation and Pediatric Rare Disease Designation by the U.S. FDA. XLH ("X" denotes X-chromosome, a sex-determining chromosome) is a rare bone disease characterised by hypophosphatemia (i.e., low phosphate in blood). It is caused by a mutation in the phosphate regulating endopeptidase homolog X-linked (PHEX) gene, and its inheritance pattern follows an X-linked dominant mode. The bone tissue of XLH patients cannot mineralise properly, thereby affecting the hardness of the bones. The cartilage between the ribs of children with XLH grows and connects like beads strung together. They also result in limb deformities and growth retardation. Adult patients experience symptoms such as osteomalacia, bone pain, changes in body shape, shorter stature and pseudo-fractures, leading to reduced mobility or even disability. Professor Zhang Ge, Associate Dean (Research) of Chinese Medicine and Director of the Law Sau Fai Institute for Advancing Translational Medicine in Bone and Joint Diseases at HKBU, and Professor Zhang Zhenlin, Director of Osteoporosis and Bone Diseases of the Shanghai Sixth People's Hospital, along with Aptacure Therapeutics Ltd., have developed a long-acting sclerostin-loop3 oligonucleotide aptamer for the treatment of XLH. This aptamer has been granted Orphan Drug Designation and Pediatric Rare Disease Designation by the U.S. FDA for treatment of XLH earlier this year. Researchers conducted serum tests on 51 patients with XLH from the Shanghai Sixth People's Hospital. The results showed that their serum sclerostin levels were approximately 4.5 times higher than those of healthy individuals of the same age and gender. Sclerostin is a protein that inhibits bone growth while playing a protective role in the cardiovascular system. Genetic evidence shows that sclerostin deficiency significantly elevates serum phosphorus levels and increases bone mass in mice. However, the monoclonal sclerostin antibody currently used increases the risk of heart attacks, stroke and cardiovascular diseases. The research team discovered that sclerostin inhibits bone formation and protects the cardiovascular system through different domains of the protein. When sclerostin loses the "loop3 domain", bone mass and strength increase, but its cardiovascular protective effect remains unaffected. The "loop3 domain" of sclerostin can therefore serve as a therapeutic molecular target to promote bone formation, with no safety concern in the cardiovascular system. The researchers then screen out oligonucleotide aptamers Apc001 that can inhibit the sclerostin loop3. Animal experiments showed that Apc001 can promote bone formation without increasing cardiovascular risk. It can also significantly increase the blood phosphorus levels of mice with XLH. This is expected to provide a drug candidate for the precise treatment of XLH patients. According to the regulations of the U.S. FDA, once a drug under development obtains Orphan Drug Designation, its subsequent research and development may allow for a reduction in the number of clinical trial samples, be exempt from new drug marketing authorisation fees, and enjoy seven years of market exclusivity. Pediatric Rare Disease Designation allows drugs to be reviewed with priority. Having been designated as both an orphan drug and a pediatric rare disease by the U.S. FDA, Apc001 can accelerate its clinical translation and is expected to benefit patients with XLH sooner. Currently, the pilot scale production of Apc001 has been completed and is undergoing the preclinical toxicological assessment by a third party. Apc001 is scheduled to enter clinical trials in both Mainland China and the U.S. SOURCE Hong Kong Baptist University WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

- Financing led by Bain Capital Life Sciences with significant participation from existing and new investors - Proceeds will support the development of AGA2118, AGA2115, AGA111 and other pipeline assets for the treatment of serious musculoskeletal diseases - Norbert Riedel, Ph.D., will join the Board of Directors WOODLAND HILLS, Calif., Dec. 11, 2024 (GLOBE NEWSWIRE) -- Angitia Biopharmaceuticals (“Angitia” or “the Company”), a clinical-stage biotechnology company focused on the discovery and development of innovative therapeutics for serious musculoskeletal diseases, today announced the closing of a $120 million Series C financing round. Bain Capital Life Sciences led the financing, with participation from new investor Janus Henderson and existing investors OrbiMed, 3H Health Investment, Yonghua Capital, Legend Capital, and Elikon Venture. Proceeds from the Series C will support Angitia’s robust pipeline of novel, differentiated treatments for serious musculoskeletal diseases. “The broad support for Angitia in this financing validates the hard work of our team, the clinical progress of our programs, and the quality of our emerging data,” said Dr. David Ke, M.D., Chief Executive Officer of Angitia. “We express gratitude to our investors, new and returning, for their support in our journey to provide novel and effective treatments for patients with musculoskeletal disease, and we look forward to continuing to execute on developing these valuable medicines.” Angitia is advancing AGA2118 and AGA2115, bispecific antibodies targeting sclerostin and DKK1, through clinical development for osteoporosis and osteogenesis imperfecta (OI), respectively. The two molecules represent the next generation of dual-acting treatments for skeletal disease, increasing bone formation and decreasing bone resorption. With these two bispecifics, Angitia seeks to promote stronger, more organized skeletal development in patients. The Company is also developing AGA111, a biologic to promote spinal fusion in patients with degenerative disc disease. In conjunction with the financing, Dr. Norbert Riedel, Ph.D., will join the Company’s Board of Directors. A seasoned scientist and biopharmaceutical executive, he brings decades of leadership experience to Angitia. Dr. Riedel serves on the board of directors of Jazz Pharmaceuticals and Eton Pharmaceuticals and is Chairman of the Board of Alcyone Therapeutics. He recently completed his tenure on the board of Cerevel Therapeutics. Dr. Riedel previously founded Aptinyx, Inc. and served as CEO of Naurex, which was acquired by Allergan in 2015. He also held senior roles at Baxter International and Hoechst-Marion Roussel (now Sanofi). Dr. Riedel holds a diploma in biochemistry and a Ph.D. in biochemistry from the University of Frankfurt. Angitia is enrolling patients in a Phase 2 study in postmenopausal women with AGA2118 (NCT06577935). AGA2115 is being developed for the treatment of OI and is currently in a first-in-human study (NCT06086613). AGA111 is being explored for use in patients undergoing lumbar interbody fusion in a Phase 3 study (NCT06115512). About Angitia Biopharmaceuticals Angitia Biopharmaceuticals is a clinical-stage biotechnology company focused on the discovery and development of innovative therapeutics for serious musculoskeletal diseases. Angitia is currently studying 3 biologic product candidates in the clinic for the treatment of osteoporosis, osteogenesis imperfecta (OI), and spinal fusion. Leveraging the team's extensive experience and scientific acumen in novel drug development, Angitia is committed to providing groundbreaking therapies to satisfy key unmet medical needs. Learn more at www.angitiabio.com. Investor & Media Contact: William WindhamSolebury Strategic Communicationswwindham@soleburystrat.com646-378-2946 Forward-Looking Statements This press release is prepared by Angitia (the “Company”, “We”) for informational purposes only. Forward-looking statements include all statements that are not historical facts, and in some cases, can be identified by terms such as “anticipate”, “expect”, “intend”, “plan”, “believe”, “continue”, “could”, “potential”, “may”, “will”, “goal” or similar expressions and the negatives of those terms. However, not all forward-looking statements contain these identifying words. These forward-looking statements involve substantial known and unknown risks, uncertainties, and other factors that are beyond the Company’s control and are difficult to predict and may cause our actual results, timing of results, or achievements to be materially different from the information expressed or implied by these forward-looking statements. We anticipate that subsequent events and developments may cause our expectations and assumptions to change, and we undertake no obligation to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise, except as may be required by law. Except as expressly required by law, the Company and/or its officers, directors, employees, and agents shall not assume responsibility for the accuracy and completeness of the forward-looking statements in the information provided.

Angitia Biopharmaceuticals, a California and China biotech developing musculoskeletal biologics, has closed a $120 million Series C led by Bain Capital Life Sciences, the drug developer exclusively told Endpoints News . The 83-employee biotech will put the capital to work on an ongoing Phase 3 trial in China and two global Phase 2 studies. The financing comes about 10 months after Angitia — named after the Goddess of healing — had done another extension to its Series B. The company has now raised a total of $330 million. CEO David Ke founded Angitia in June 2018 after helping lead UCB and Amgen’s romosozumab toward approval. The FDA greenlit the osteoporosis drug in 2019 as Evenity . Prior to his three-year stint at UCB, Ke spent about a decade at Amgen. It was there that he collaborated with Willard Dere, who also worked on four anti-osteoporosis drugs and led Amgen’s global clinical development. Ke is the “best bone biologist in industry,” Dere said in an interview. That’s why he wanted Ke to join him at Amgen. Both would go on to leave Amgen in 2014. Dere returned to academia at the University of Utah and took up board seats at BioMarin, Mersana, Seres and Metagenomi. Ke would go on to a VP role at UCB. Then, after years of tinkering with the idea of starting a company, he launched Angitia. A small, focused biotech would be more efficient and more his speed than big pharma. Ke set up a discovery shop in Guangzhou, China, and a clinical development team in Woodland Hills, CA. Then, two years ago, Dere reunited with Ke as chief medical officer of Angitia. Now, they’re full speed ahead with a fully enrolled Phase 3 in China for AGA111, which is being tested for patients with degenerative disc disease who are undergoing a lumbar interbody fusion. The company has started talks with the FDA about potential studies in the US, but the discussions aren’t finalized and Angitia hasn’t made any concrete decisions yet on the future of the biologic, Ke said. The biotech also has two bispecific antibodies targeting sclerostin and DKK1. The lead one, AGA2118, just entered Phase 2 for post-menopausal women with low bone mass as a potential treatment for osteoporosis, an area with which Ke and Dere are quite familiar. The third clinical-stage asset is dubbed AGA2115. It recently completed a Phase 1 healthy volunteer trial that will have data in the first quarter of 2025 and will move into Phase 2 by the end of next year for osteogenesis imperfecta, Ke said. The Series C financing will get Angitia all the way through the Phase 2 studies and give it flexibility on needing to raise additional capital, Ke said. The biotech will likely seek a pharma partner for a Phase 3 and beyond in osteoporosis , the CEO said. In its discovery labs, Angitia’s scientists are also exploring additional muscle programs and cartilage regeneration targets, Ke said. In the muscle arena, that could include muscle diseases, aging-related muscle loss or therapeutic-induced muscle loss, he added. The latter has been seen with the blockbuster GLP-1 class, and obesity drugmakers are racing to make medicines that preserve muscle, or lean body mass, during the weight-loss process. The financing was led by Bain’s life sciences team, which disclosed a $3 billion fourth fund in September. Angitia is one of multiple biotechs with operations in China that Bain has supported over the years. It’s invested in Beijing oncology biotech Avistone and CNS-focused Tenacia Biotechnology , and it’s helped set up new companies like Kailera and Aiolos to in-license assets from Hengrui and develop the experimental medicines on a broader scale. Joining Bain was new investor Janus Henderson and existing backers like OrbiMed, 3H Health Investment, Legend Capital, Elikon Venture and Yonghua Capital.