Brain Stem Infarctions

BUCHAREST, Romania, Jan. 09, 2024 (GLOBE NEWSWIRE) -- Er-Kim, an international pharmaceutical company specializing in the commercialization of novel therapies, today announced it has entered into an exclusive distribution agreement with Danish Company Ascendis Pharma A/S (Nasdaq: ASND) for its endocrinology portfolio. Under the agreement, Er-Kim is appointed as the exclusive Ascendis A/S endocrinology distributor in Albania, Bosnia and Herzegovina, Bulgaria, Croatia, Cyprus, Czechia, Estonia, Greece, Hungary, Kosovo, Latvia, Lithuania, Malta, Montenegro, North Macedonia, Poland, Romania, Serbia, Slovakia, Slovenia, and Türkiye. Once-weekly SKYTROFA (lonapegsomatropin) is a human growth hormone (hGH) approved in the European Union for growth failure in children and adolescents aged from 3 to 18 years due to insufficient endogenous growth hormone secretion (growth hormone deficiency [GHD]).2 YORVIPATH (palopegteriparatide) is a first-in-class parathyroid hormone (PTH) replacement therapy to treat chronic hypoparathyroidism, a rare and potentially serious condition where the body produces no or abnormally low levels of PTH. It is approved in the European Union for the treatment of adults with chronic hypoparathyroidism.3 A third product – TransCon CNP (navepegritide) – is in development by Ascendis Pharma for the treatment of achondroplasia (ACH), the most common genetic form of skeletal dysplasia and resulting disproportionate short stature, following successful Phase 2 trial results.4 “We are immensely honored to be selected as Ascendis Pharma’s partner in this pivotal agreement for our company,” stated Mert Zorlular, CFO of Er-Kim. He added, “Leveraging our exceptional expertise in rare diseases and our preeminent status in endocrinology throughout the region, we eagerly anticipate collaborating with Ascendis and the healthcare community. Our goal is to swiftly establish and broaden patient access to these innovative treatments in our markets.” “Making a meaningful difference in the lives of patients with rare diseases is a key focus for Ascendis Pharma,” said Camilla Harder Hartvig, Ascendis Pharma’s Executive Vice President and Chief Commercial Officer. “We are therefore very pleased to partner with Er-Kim, a company that shares this commitment, to broaden and accelerate access to our innovative Endocrinology Rare Disease treatment options across Central and Eastern Europe and Turkey.” About Er-Kim Established in 1981, Er-Kim Pharmaceuticals stands at the forefront of biopharmaceutical innovation, partnering with over 40 global leaders to revolutionize patient care in key international markets. Our pioneering business models, tailored for sustainability and flexibility, have positioned us as a full-service solution, extending our reach to over 500 million patients through our fully-owned affiliates. With a dedicated team of over 250 professionals worldwide and revenues exceeding EUR 100M, Er-Kim is not just a partner but a trailblazer in healthcare, continually setting new standards in commercialization and patient access. For more information, please visit . About Ascendis Pharma Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Please visit to learn more. About SKYTROFA (lonapegsomatropin) SKYTROFA™ (lonapegsomatropin, developed as TransCon™ hGH) is a prodrug of somatropin, designed to provide sustained release of unmodified somatropin. The unmodified, unbound somatropin released from lonapegsomatropin has the same 191 amino acid sequence and size as endogenous GH.5 TransCon hGH is approved and marketed as SKYTROFA (lonapegsomatropin-tcgd) in the United States1 and as SKYTROFA (lonapegsomatropin) in the European Union2 as a once-weekly treatment for children and adolescents with GHD. SKYTROFA single-use, prefilled cartridges are manufactured in nine dosage strengths, allowing for convenient dosing flexibility. They are designed for use only with the SKYTROFA Auto-Injector and may be stored at room temperature for up to six months. The recommended dose of SKYTROFA for treatment-naïve patients and patients switching from daily somatropin is 0.24 mg/kg body weight, administered once weekly.1,2 The dose may be adjusted based on the child’s weight and insulin-like growth factor-1 standard deviation score (IGF-1 SDS).2 TransCon hGH was studied in over 300 children with GHD across the Phase 3 program, which consisted of the Height Trial5 (for treatment-naïve patients), the Flight Trial6 (for treatment-experienced patients), and the Enlighten Trial7 (a long-term extension trial). Patients who completed the Height or Flight Trials were able to continue in Enlighten, with some on lonapegsomatropin treatment for over four years. Ascendis Pharma is also conducting the ongoing global Phase 3 Foresight Trial of TransCon hGH in adults with GHD.8 About Paediatric Growth Hormone Deficiency Paediatric GHD is a serious orphan disease caused when the pituitary gland does not produce enough GH. Children with GHD are not only characterised by short stature; they also may experience metabolic abnormalities, psychosocial challenges, and an overall poor quality of life. For decades, the standard of care for GHD has been a daily subcutaneous injection of hGH to improve growth and overall endocrine health. About YORVIPATH (palopegteriparatide) YORVIPATH™ (palopegteriparatide, developed as TransCon™ PTH) is a once-daily prodrug with sustained release of active PTH approved by the European Union as a PTH replacement therapy for the treatment of adults with chronic hypoparathyroidism. Treatment should be initiated and monitored by physicians or qualified healthcare professionals experienced in the diagnosis and management of patients with hypoparathyroidism.3 TransCon PTH met all primary and key secondary endpoints in the Phase 3 Pathway Trial, demonstrating a response rate of 78.7% compared to 4.8% for control (p-value <0.0001) for the primary composite endpoint, and statistically significant improvements compared to control on all key secondary endpoints, which included measures evaluating patient-reported disease symptoms and impacts.9 About Hypoparathyroidism Hypoparathyroidism is an endocrine disease caused by insufficient levels of PTH, the primary regulator of calcium/phosphate balance in the body, acting directly on bone and kidneys and indirectly on intestines. Hypoparathyroidism is considered chronic if it persists >6 months following surgery. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment.9 About TransCon CNP (navepegritide) TransCon™ CNP (navepegritide) is an investigational long-acting prodrug of C-type natriuretic peptide (CNP), designed to provide continuous exposure of CNP at safe, therapeutic levels, via a single, weekly subcutaneous dose, for the treatment of children with ACH.4 The Phase 2 Accomplish Trial, a randomised, double-blind, placebo-controlled, dose-escalation trial evaluating the safety and efficacy of once-weekly TransCon CNP compared to placebo in prepubertal children with ACH aged 2 to 10 years old, met its primary objectives, and demonstrated that TransCon CNP at 100 µg/kg/week was superior to placebo for the primary efficacy endpoint of annualised growth velocity (AGV) at 52 weeks4. All 57 randomised children completed the blinded portion of Accomplish and are currently continuing in the open label extension at the 100 μg/kg/week dose4. Ascendis Pharma recently confirmed that these 57 clinical trial patients have all completed one year of treatment with TransCon CNP at 100 µg/kg/week, and announced that TransCon CNP is the first investigational product to demonstrate improvements in health-related quality of life and disease impacts in children with ACH.10 About Achondroplasia Achondroplasia is the most common genetic form of skeletal dysplasia and resulting disproportionate short stature, caused by a genetic mutation in the fibroblast growth factor receptor 3 (FGFR3). This leads to an imbalance between the stimulatory and inhibitory signaling pathways involved in regulating bone growth. People living with ACH may experience serious complications and comorbidities due to inhibited skeletal development. Complications may include sleep apnoea and respiratory problems, chronic back and leg pain from lower spine impingement, and sudden infant death from compression of the brain stem. Chronic ear infections due to eustachian tube problems can lead to hearing loss and speech delay. Children with ACH may also experience social and emotional challenges. References SKYTROFA (lonapegsomatropin-tcgd) US Prescribing Information. SKYTROFA (lonapegsomatropin) EU Summary of Product Characteristics. YORVIPATH (palopegteriparatide) EU Summary of Product Characteristics. Savarirayan, R, Hoemschemeyer DG, Ljungberg M, et al., Lancet. 2023;65:1–10. Thornton PS, Maniatis AK, Aghajanova E, et al., J Clin Endocrinol Metab. 2021;106(11):3184–3195. Maniatis AK, Nadgir U, Saenger P, et al., Horm Res Paediatr. 2022;95(3):233–243. Maniatis AK, Casella SJ, Nadgir UM, et al. J Clin Endocrinol Metab. 2022;107(7): e2680–e2689. Foresight Trial (NCT05171855), . Khan AA, Rubin MR, Schwarz P, et al. J Bone Miner Res. 2023;38(1):14–25. Ascendis Pharma A/S, Significant Health and Quality of Life Improvements Achieved in Children with Achondroplasia Treated for One Year with TransCon™ CNP (Navepegritide) at 100 µg/kg/week, Press Release, 20 December 2023, . Media contact: Rosalia Scampoli Marketcom PR Tel: (914) 815-1465 rscampoli@marketcompr.com

Agreement between Ascendis Pharma A/S and ST is for the exclusive distribution and commercialisation of three Ascendis Pharma endocrinology therapies Two of the therapies are already internationally approved, the third is in development following successful Phase 2 data ST's exclusive distribution agreement covers Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand and Vietnam SINGAPORE, Jan. 7, 2024 /PRNewswire/ -- Independent biopharmaceutical company Specialised Therapeutics Asia Pte Ltd (ST) has added three new endocrinology therapies to its specialist portfolio, following an exclusive distribution agreement with Danish company Ascendis Pharma A/S (NASDAQ: ASND). Under the terms of the agreement, ST will commercialise Ascendis Pharma's weekly injectable paediatric human growth hormone treatment SKYTROFATM (lonapegsomatropin), hypoparathyroidism treatment YORVIPATHTM (palopegteriparatide) and investigational achondroplasia therapy TransCon™ CNP (navepegritide). The agreement spans ST's key regions of Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam. Two of the products included in this agreement are already internationally approved: Once-weekly SKYTROFA is a human growth hormone (hGH) approved in the United States for the treatment of paediatric patients aged >1 years weighing >11.5 kg with growth failure due to inadequate secretion of endogenous growth hormone (GH)1 and in the European Union for growth failure in children and adolescents aged from 3 to 18 years due to insufficient endogenous growth hormone secretion (growth hormone deficiency [GHD]).2 YORVIPATH is a first-in-class parathyroid hormone (PTH) replacement therapy to treat chronic hypoparathyroidism, a rare and potentially serious condition where the body produces no or abnormally low levels of PTH. It is approved in the European Union for the treatment of adults with chronic hypoparathyroidism.3 The third product – TransCon CNP – is in development by Ascendis Pharma for the treatment of achondroplasia (ACH), the most common genetic form of skeletal dysplasia and resulting disproportionate short stature, following successful Phase 2 trial results.4 Australian endocrinologist Dr Veronica Preda noted that YORVIPATH would be the first specialist therapeutic option for Australian patients living with hypoparathyroidism. "Hypoparathyroidism can seriously impact quality of life and has potentially life-threatening consequences," Dr Preda said. "To have an option that is able to treat the underlying cause of the disease, moving beyond standard oral calcium and active Vitamin D, is a great step forward." Announcing the partnership, ST Chief Executive Officer Carlo Montagner said this agreement was an important company milestone, signalling ST's expansion into both endocrinology and paediatric medicine. Mr Montagner commented: "We are delighted to have been selected as Ascendis Pharma's exclusive partner for commercialising their portfolio in Oceania and these South-East Asia countries and look forward to launching these critical endocrinology products in our regions as soon as possible. "All three products are valuable inclusions to our broad therapeutic pipeline and our international business, as we continue to leverage our substantial experience commercialising specialist medicines across multiple regions. "We look forward to working with endocrinologists across our territories to make these endocrine therapies available to all eligible patients who may benefit." Ascendis Pharma Executive Vice President and Chief Commercial Officer Camilla Harder Hartvig said ST had been selected to launch the endocrinology portfolio in these countries based on its strong track record commercialising specialist products in multiple regions. "We are delighted to partner with Specialised Therapeutics to broaden the reach of our endocrinology rare disease portfolio, contributing to our shared goal of making a meaningful difference for patients facing unmet medical needs," she said. About Specialised Therapeutics Founded in 2007, Specialised Therapeutics is the region's largest independent specialty pharmaceutical company, providing new therapies and technologies to patients in Australia, New Zealand and across Southeast Asia. Headquartered in Singapore, ST partners with global pharmaceutical, biotech and diagnostic companies to bring novel healthcare opportunities to patients who are impacted by a range of diseases. ST has built a strong track record of success, navigating complex regulatory, reimbursement and commercialisation environments in its diverse regions. The ST mission is to provide specialty therapies where there is an unmet need. The company's broad therapeutic portfolio currently includes novel agents in oncology, haematology, CNS, neurology, endocrinology, ophthalmology and supportive care, although it is not confined to these areas. ST is a member of the World Orphan Drug Alliance (WODA). Additional information: About Ascendis Pharma Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients' lives. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new and potentially best-in-class therapies. Please visit to learn more. About SKYTROFA (lonapegsomatropin) SKYTROFA™ (lonapegsomatropin, developed as TransCon™ hGH) is a prodrug of somatropin, designed to provide sustained release of unmodified somatropin. The unmodified, unbound somatropin released from lonapegsomatropin has the same 191 amino acid sequence and size as endogenous GH.5 TransCon hGH is approved and marketed as SKYTROFA (lonapegsomatropin-tcgd) in the United States1 and as SKYTROFA (lonapegsomatropin) in the European Union2 as a once-weekly treatment for children and adolescents with GHD. SKYTROFA single-use, prefilled cartridges are manufactured in nine dosage strengths, allowing for convenient dosing flexibility. They are designed for use only with the SKYTROFA Auto-Injector and may be stored at room temperature for up to six months. The recommended dose of SKYTROFA for treatment-naïve patients and patients switching from daily somatropin is 0.24 mg/kg body weight, administered once weekly.1,2 The dose may be adjusted based on the child's weight and insulin-like growth factor-1 standard deviation score (IGF-1 SDS).2 TransCon hGH was studied in over 300 children with GHD across the Phase 3 program, which consisted of the Height Trial5 (for treatment-naïve patients), the Flight Trial6 (for treatment-experienced patients), and the Enlighten Trial7 (a long-term extension trial). Patients who completed the Height or Flight Trials were able to continue in Enlighten, with some on lonapegsomatropin treatment for over four years. Ascendis Pharma is also conducting the ongoing global Phase 3 Foresight Trial of TransCon hGH in adults with GHD.8 About Paediatric Growth Hormone Deficiency Paediatric GHD is a serious orphan disease caused when the pituitary gland does not produce enough GH. Children with GHD are not only characterised by short stature; they also may experience metabolic abnormalities, psychosocial challenges, and an overall poor quality of life. For decades, the standard of care for GHD has been a daily subcutaneous injection of hGH to improve growth and overall endocrine health. About YORVIPATH (palopegteriparatide) YORVIPATH™ (palopegteriparatide, developed as TransCon™ PTH) is a once-daily prodrug with sustained release of active PTH approved by the European Union as a PTH replacement therapy for the treatment of adults with chronic hypoparathyroidism. Treatment should be initiated and monitored by physicians or qualified healthcare professionals experienced in the diagnosis and management of patients with hypoparathyroidism.3 TransCon PTH met all primary and key secondary endpoints in the Phase 3 Pathway Trial, demonstrating a response rate of 78.7% compared to 4.8% for control (p-value <0.0001) for the primary composite endpoint, and statistically significant improvements compared to control on all key secondary endpoints, which included measures evaluating patient-reported disease symptoms and impacts.9 About Hypoparathyroidism Hypoparathyroidism is an endocrine disease caused by insufficient levels of PTH, the primary regulator of calcium/phosphate balance in the body, acting directly on bone and kidneys and indirectly on intestines. Hypoparathyroidism is considered chronic if it persists >6 months following surgery. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment.9 About TransCon CNP (navepegritide) TransCon™ CNP (navepegritide) is an investigational long-acting prodrug of C-type natriuretic peptide (CNP), designed to provide continuous exposure of CNP at safe, therapeutic levels, via a single, weekly subcutaneous dose, for the treatment of children with ACH.4 The Phase 2 Accomplish Trial, a randomised, double-blind, placebo-controlled, dose-escalation trial evaluating the safety and efficacy of once-weekly TransCon CNP compared to placebo in prepubertal children with ACH aged 2 to 10 years old, met its primary objectives, and demonstrated that TransCon CNP at 100 µg/kg/week was superior to placebo for the primary efficacy endpoint of annualised growth velocity (AGV) at 52 weeks4. All 57 randomised children completed the blinded portion of Accomplish and are currently continuing in the open label extension at the 100 μg/kg/week dose4. Ascendis Pharma recently confirmed that these 57 clinical trial patients have all completed one year of treatment with TransCon CNP at 100 µg/kg/week, and announced that TransCon CNP is the first investigational product to demonstrate improvements in health-related quality of life and disease impacts in children with ACH.10 About Achondroplasia Achondroplasia is the most common genetic form of skeletal dysplasia and resulting disproportionate short stature, caused by a genetic mutation in the fibroblast growth factor receptor 3 (FGFR3). This leads to an imbalance between the stimulatory and inhibitory signaling pathways involved in regulating bone growth. People living with ACH may experience serious complications and comorbidities due to inhibited skeletal development. Complications may include sleep apnoea and respiratory problems, chronic back and leg pain from lower spine impingement, and sudden infant death from compression of the brain stem. Chronic ear infections due to eustachian tube problems can lead to hearing loss and speech delay. Children with ACH may also experience social and emotional challenges. References SKYTROFA (lonapegsomatropin-tcgd) US Prescribing Information. SKYTROFA (lonapegsomatropin) EU Summary of Product Characteristics. YORVIPATH (palopegteriparatide) EU Summary of Product Characteristics. Savarirayan, R, Hoemschemeyer DG, Ljungberg M, et al., Lancet. 2023;65:1–10. Thornton PS, Maniatis AK, Aghajanova E, et al., J Clin Endocrinol Metab. 2021;106(11):3184–3195. Maniatis AK, Nadgir U, Saenger P, et al., Horm Res Paediatr. 2022;95(3):233–243. Maniatis AK, Casella SJ, Nadgir UM, et al. J Clin Endocrinol Metab. 2022;107(7): e2680–e2689. Foresight Trial (NCT05171855), . Khan AA, Rubin MR, Schwarz P, et al. J Bone Miner Res. 2023;38(1):14–25. Ascendis Pharma A/S, Significant Health and Quality of Life Improvements Achieved in Children with Achondroplasia Treated for One Year with TransCon™ CNP (Navepegritide) at 100 µg/kg/week, Press Release, 20 December 2023, . SOURCE Specialised Therapeutics

Researchers have developed a brain-computer interface (BCI) that has enabled a woman with severe paralysis from a brainstem stroke to speak through a digital avatar. Researchers at UC San Francisco and UC Berkeley have developed a brain-computer interface (BCI) that has enabled a woman with severe paralysis from a brainstem stroke to speak through a digital avatar. It is the first time that either speech or facial expressions have been synthesized from brain signals. The system can also decode these signals into text at nearly 80 words per minute, a vast improvement over commercially available technology. Edward Chang, MD, chair of neurological surgery at UCSF, who has worked on the technology, known as a brain computer interface, or BCI, for more than a decade, hopes this latest research breakthrough, appearing Aug. 23, 2023, in Nature, will lead to an FDA-approved system that enables speech from brain signals in the near future. "Our goal is to restore a full, embodied way of communicating, which is really the most natural way for us to talk with others," said Chang, who is a member of the UCSF Weill Institute for Neuroscience and the Jeanne Robertson Distinguished Professor in Psychiatry. "These advancements bring us much closer to making this a real solution for patients." Chang's team previously demonstrated it was possible to decode brain signals into text in a man who had also experienced a brainstem stroke many years earlier. The current study demonstrates something more ambitious: decoding brain signals into the richness of speech, along with the movements that animate a person's face during conversation. Chang implanted a paper-thin rectangle of 253 electrodes onto the surface of the woman's brain over areas his team has discovered are critical for speech. The electrodes intercepted the brain signals that, if not for the stroke, would have gone to muscles in her, tongue, jaw and larynx, as well as her face. A cable, plugged into a port fixed to her head, connected the electrodes to a bank of computers. For weeks, the participant worked with the team to train the system's artificial intelligence algorithms to recognize her unique brain signals for speech. This involved repeating different phrases from a 1,024-word conversational vocabulary over and over again, until the computer recognized the brain activity patterns associated with the sounds. Rather than train the AI to recognize whole words, the researchers created a system that decodes words from phonemes. These are the sub-units of speech that form spoken words in the same way that letters form written words. "Hello," for example, contains four phonemes: "HH," "AH," "L" and "OW." Using this approach, the computer only needed to learn 39 phonemes to decipher any word in English. This both enhanced the system's accuracy and made it three times faster. "The accuracy, speed and vocabulary are crucial," said Sean Metzger, who developed the text decoder with Alex Silva, both graduate students in the joint Bioengineering Program at UC Berkeley and UCSF. "It's what gives a user the potential, in time, to communicate almost as fast as we do, and to have much more naturalistic and normal conversations." To create the voice, the team devised an algorithm for synthesizing speech, which they personalized to sound like her voice before the injury, using a recording of her speaking at her wedding. The team animated the avatar with the help of software that simulates and animates muscle movements of the face, developed by Speech Graphics, a company that makes AI-driven facial animation. The researchers created customized machine-learning processes that allowed the company's software to mesh with signals being sent from the woman's brain as she was trying to speak and convert them into the movements on the avatar's face, making the jaw open and close, the lips protrude and purse and the tongue go up and down, as well as the facial movements for happiness, sadness and surprise. "We're making up for the connections between the brain and vocal tract that have been severed by the stroke," said Kaylo Littlejohn, a graduate student working with Chang and Gopala Anumanchipalli, PhD, a professor of electrical engineering and computer sciences at UC Berkeley. "When the subject first used this system to speak and move the avatar's face in tandem, I knew that this was going to be something that would have a real impact." An important next step for the team is to create a wireless version that would not require the user to be physically connected to the BCI. "Giving people the ability to freely control their own computers and phones with this technology would have profound effects on their independence and social interactions," said co-first author David Moses, PhD, an adjunct professor in neurological surgery.