VMAT2

NAARDEN, Netherlands & WALTHAM, Mass.--( BUSINESS WIRE )-- Prilenia Therapeutics B.V. , a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, announced its plan to submit a Marketing Authorization Application (MAA) for pridopidine for the treatment of Huntington’s disease (HD) to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP). This decision follows positive pre-submission meetings with regulatory authorities in the European Union. Submission is planned for mid-2024. “Pridopidine demonstrates consistent treatment benefits across independent measures that are important to patients and families. These measures include day-to-day function, cognition, motor and clinical progression in people living with HD. The benefits are clearly evident in those with HD who are not taking anti-dopaminergic medications (ADM),” said Dr. Michael R. Hayden, CEO of Prilenia. “We appreciate the constructive discussions with European regulators regarding our data for pridopidine. This provides a pathway to potential approval of a therapy for HD, a rare neurodegenerative disease with a predictable decline, and no currently approved treatments that address progression. We are now finalizing our MAA submission for mid-2024 and preparing to make pridopidine commercially available for patients in Europe, if approved.” ADMs include neuroleptics (also known as antipsychotics) and VMAT2 inhibitor (anti-chorea) drugs. Prilenia intends to discuss with the U.S. Food and Drug Administration (FDA) a potential path forward for pridopidine as a possible treatment for those living with HD in the United States. The Company will also consider global regulatory submissions for additional countries and regions following the regulatory review process in Europe. In addition to the progress in HD, Prilenia announced in January 2024 that it had completed discussions with global regulatory agencies regarding the next stage of development of pridopidine for amyotrophic lateral sclerosis (ALS) and is planning for a single pivotal Phase 3 study. About Pridopidine Pridopidine (45 mg twice daily) is an oral, highly selective and potent investigational S1R agonist that has exhibited a safety and tolerability profile similar to placebo in clinical studies to date. The safety dataset includes approximately 1,700 patients who have been on pridopidine for up to seven years. The S1R protein is highly expressed in the brain and spinal cord, where it regulates several key processes that are commonly impaired in various neurodegenerative diseases. Activation of the S1R by pridopidine stimulates multiple cellular protective pathways including enhancing autophagy, axonal transport, mitochondrial energy production and respiration, and restores calcium homeostasis. These effects are essential to neuronal function and survival, and lead to neuroprotective effects. Prilenia holds Orphan Drug designation for pridopidine in HD and ALS in the U.S. and EU. In addition, pridopidine has received Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of HD. About Huntington’s disease Huntington’s disease (HD) is a rare inherited (genetic) neurodegenerative disorder caused by a mutation in the huntingtin gene. Each child of a parent with HD has a 50 percent chance of developing the disease. HD causes neurons in the brain to degenerate and lose their ability to communicate with each other, resulting in functional, motor, cognitive and behavioral symptoms. HD affects about 100,000 people around the world with an additional 300,000 people at risk of developing HD. It is usually diagnosed between the ages of 30 and 50, although HD can also occur at any age including children and young adults (known as juvenile onset HD or JHD). The disease progresses slowly over 15 to 20 years, with patients slowly losing their ability to work, communicate, manage day to day life and take care of themselves. Currently, there is no treatment to address the progression of HD. About PROOF-HD PROOF-HD, a Phase 3, randomized, double-blind, placebo-controlled study, evaluated the safety and efficacy of pridopidine (45 mg twice daily for 65-78 weeks), in individuals with HD ( NCT04556656 ). The study enrolled 499 individuals and was conducted in the U.S., Canada, Austria, Czech Republic, France, Germany, Italy, the Netherlands, Poland, Spain and the United Kingdom. Pridopidine (45 mg bid) has shown consistent and sustained benefit on multiple, independent, clinically meaningful measures in HD participants not taking ADMs. These benefits were seen across multiple clinical endpoints that matter to people with HD and their families (function, cognition, motor, global disease progression and quality of life). In pre-specified analyses, there were improvements compared to placebo across progression, motor, cognition and HD QoL (as measured by TFC, cUHDRS, SWR, Q-Motor, HDQoL). These treatment benefits were sustained for up to 78 weeks at all time points. There was also unprecedented improvement from baseline for a minimum of 1 year in cUHDRS, cognition and motor function (Q-Motor). These PROOF-HD results are further supported by an independent integrated efficacy analysis from a 4 placebo-controlled, double-blind studies of pridopidine in HD which showed significance for cUHDRS, TFC and Q-Motor in patients off ADMs. In clinical studies to date, pridopidine’s safety and tolerability profile was comparable to placebo. The PROOF-HD study was conducted in collaboration with the Huntington Study Group (HSG) , a world leader in clinical research for HD and valued collaborator for Prilenia. About Prilenia Prilenia is a clinical stage biotechnology company founded in 2018 focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders. The initial focus of the company has been on HD and ALS. Prilenia is backed by a group of well-respected investors including: Forbion, Morningside, Sands Capital, SV Health Investors, Sectoral Asset Management, Talisman, Amplitude Ventures and the ALS Investment Fund. The Company is based in the Netherlands, Israel and Massachusetts in the U.S. For more information, visit www.prilenia.com and follow us on LinkedIn and Twitter . © 2024 Prilenia Therapeutics B.V. For a copy of this release, visit Prilenia’s website at www.prilenia.com .

SHANGHAI & TEL AVIV, Israel--(BUSINESS WIRE)-- Teva Pharmaceutical Investments Singapore Pte Ltd (TPIS), a subsidiary of Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) and Jiangsu Nhwa Pharmaceutical Co., Ltd (“Nhwa”) today announced it formed a partnership for the marketing and distribution of Teva’s AUSTEDO (deutetrabenazine) for the treatment of neurodegenerative and movement disorders - chorea associated with Huntington’s disease (HD) and tardive dyskinesia (TD) in adults. The partnership intends to increase patients’ access to Teva’s AUSTEDO, leveraging Nhwa’s leadership in China’s neuro-psychiatric health sector. “Nhwa has deep neuro-psychiatry expertise and capabilities, from research and development to commercialization,” commented Theodor Wee, General Manager of Teva Greater China. “Together, we can promote cost-effective delivery of safe, quality medicines and contribute to the sustainability of China’s healthcare system.” Huntington’s disease is a rare and fatal neurodegenerative disorder. Chorea, one of the most striking physical manifestations of Huntington’s disease, is a neurological disorder that causes involuntary, random muscle movements. It is one of the main symptoms of the disease, occurring in up to 90% of adults living with Huntington’s disease 1 . Tardive dyskinesia is another disorder that results in repetitive and uncontrollable movements of the tongue, lips, face, trunk and extremities, affecting 20-50% of patients on long-term antipsychotic medication. For patients with schizophrenia, who are more likely to be treated with antipsychotic medication, the prevalence of TD in China is higher (36.0%~46.5% [ 234 ]), than the global average (25.3% 5 ) . . “As the first deuterated drug approved in China, AUSTEDO offers an innovative treatment for chorea associated with Huntington’s disease and tardive dyskinesia in adults—debilitating disorders that directly impact daily function and quality of life,” added Sun Jiaquan, President and CEO of Nhwa. In a deuterated drug, hydrogen atoms are replaced with deuterium, which may significantly lower metabolism rates. The deuterium technology used in AUSTEDO allows less frequent dosing for patients. “We are committed to addressing the pressing needs of patients in China with more innovative medicines, in line with our commitment throughout our International Markets region and across the globe,” said Mark Sabag, EVP and Head of International Markets at Teva. “This partnership assists us with further growing AUSTEDO as a global leading brand, with over $1.2 billion in revenues in 2023, and to deliver on Teva’s Pivot to Growth strategy and providing better health for our patients.” About AUSTEDO AUSTEDO is the first and only vesicular monoamine transporter 2 (VMAT2) inhibitor approved by the U.S. Food and Drug Administration in adults for the treatment of tardive dyskinesia (TD) and for the treatment of chorea associated with Huntington’s disease (HD). About Teva Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) is a global pharmaceutical leader with a category-defying portfolio, harnessing our generics expertise and stepping up innovation to continue the momentum behind the discovery, delivery, and expanded development of modern medicine. For over 120 years, Teva's commitment to bettering health has never wavered. Today, the company’s global network of capabilities enables its ~37,000 employees across 58 markets to push the boundaries of scientific innovation and deliver quality medicines to help improve health outcomes of millions of patients every day. To learn more about how Teva is all in for better health, visit www.tevapharm.com . About Jiangsu Nhwa Pharmaceutical Jiangsu Nhwa Pharmaceutical Co., Ltd., founded in 1978, is a leading CNS company in China. Over the past 40 years, Nhwa is exclusively dedicated to developing innovative and differentiated pipeline in the areas of anesthesia, analgesia, psychiatry and neurology via in-house R&D and global partnership. As a fully integrated pharmaceutical company with more than 4000 employees, Nhwa has comprehensive capabilities in research, clinical development, manufacturing and commercialization of CNS drugs. In recent years, Nhwa has further strengthened its leadership in CNS field in China by providing the services of precision diagnosis of CNS disorders (Shanghai N-yuen Biotechnology Company), and investing the largest Chinese CNS internet health platform (Happy Mood). Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are based on management’s current beliefs and expectations and are subject to substantial risks and uncertainties, both known and unknown, that could cause our future results, performance or achievements to differ significantly from that expressed or implied by such forward-looking statements. You can identify these forward-looking statements by the use of words such as “should,” “expect,” “anticipate,” “estimate,” “target,” “may,” “project,” “guidance,” “intend,” “plan,” “believe” and other words and terms of similar meaning and expression in connection with any discussion of future operating or financial performance. Important factors that could cause or contribute to such differences include risks relating to: our ability to successfully commercialize AUSTEDO in China and globally; our strategic partnership with Jiangsu Nhwa; our ability to successfully compete in the marketplace including our ability to develop and commercialize additional pharmaceutical products; our ability to successfully execute our Pivot to Growth strategy, including to expand our innovative and biosimilar medicines pipeline and profitably commercialize the innovative medicines and biosimilar portfolio, whether organically or through business development, and to sustain and focus our portfolio of generics medicines; and other factors discussed in this press release, and in our Annual Report on Form 10-K for the year ended December 31, 2023, including in the sections captioned "Risk Factors” and “Forward Looking Statements.” Forward-looking statements speak only as of the date on which they are made, and we assume no obligation to update or revise any forward-looking statements or other information contained herein, whether as a result of new information, future events or otherwise. You are cautioned not to put undue reliance on these forward-looking statements. 1 Haddad MS, Cummings JL. Psychiatr Clin North Am 1997;20:791–807 2 Hui L, et al. Schizophr Res. 2017 Apr;182:104-109. 3 Uludag K, et al. Asian J Psychiatr. 2021 Dec;66:102877. 4 Liang Q, et al. J Psychiatr Res. 2022 Jul;151:181-187. 5 Carbon M, et al. J Clin Psychiatry. 2017 Mar;78(3):e264-e278 View source version on businesswire.com : https://www.businesswire.com/news/home/20240226932316/en/ IR Ran Meir, +1 (267) 468-4475 Yael Ashman, +972 (3) 914 8262 Sanjeev Sharma, +1 (973) 658 2700 PR Kelley Dougherty, +1 (973) 832-2810 Eden Klein, +972 (3) 906 2645 Source: Teva Pharmaceutical Industries Limited

FRIDAY, Jan. 19, 2024 -- The Tardive Dyskinesia Impact Scale (TDIS) patient-reported outcome measure captures information about the impact of TD, according to a study published online Jan. 4 in the Journal of Patient-Reported Outcomes . Robert H. Farber, Ph.D., from Neurocrine Biosciences Inc. in San Diego, and colleagues used data from qualitative studies and phase 3 trials of a VMAT2 inhibitor for treatment of TD to determine the psychometric properties of the TDIS. To assess how well the TDIS captured key domains of TD impact, qualitative research included concept elicitation and cognitive debriefing interviews with TD patients and their caregivers. The psychometric properties of the TDIS were examined with quantitative analysis including assessing construct validity and responsiveness to change. The researchers found that the key TD impacts reported by patients and caregivers were captured by the TDIS; the TDIS was interpreted as intended and relevant to patients' experiences based on qualitative results. Evidence of two underlying domains of the TDIS was found in quantitative results: physical and socioemotional. Compared with the clinician-rated outcome (Abnormal Involuntary Movement Scale), the TDIS captured unique content based on known groups and predictive validity. Responsiveness to change in treatment was seen with the TDIS, with improvement noted in TDIS scores over 48 weeks in two phase 3 trials. "The TDIS can be easily administered in a clinician's office or at a patient's home to provide insight about TD impact and whether the impact is lessening or increasing," the authors write. Several authors disclosed ties to biopharmaceutical companies, including Neurocrine Biosciences, which funded the study. Abstract/Full Text Disclaimer: Statistical data in medical articles provide general trends and do not pertain to individuals. Individual factors can vary greatly. Always seek personalized medical advice for individual healthcare decisions. Posted January 2024 More news resources FDA Medwatch Drug Alerts Daily MedNews News for Health Professionals New Drug Approvals New Drug Applications Drug Shortages Clinical Trial Results Generic Drug Approvals Subscribe to our newsletter Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.