[Translation] A multicenter, randomized, double-blind, placebo-controlled clinical study to evaluate the efficacy and safety of Opicapone in patients with primary Parkinson's disease who experience end-of-dose motor fluctuations after treatment with levodopa/dopamine decarboxylase inhibitors

[Translation]

A multicenter, randomized, double-blind, placebo-controlled clinical study to evaluate the efficacy and safety of Opicapone in patients with primary Parkinson's disease who experienced end-of-dose motor fluctuations after treatment with levodopa/dopamine decarboxylase inhibitors

Start Date17 Apr 2023

Sponsor / Collaborator

BIAL-Portela & Cia SA

[+1]

NCT04990284 / Active, not recruitingPhase 4

A Randomized, Parallel Group, Multicentre, Multinational, Prospective, Open-label Exploratory Study to Evaluate the add-on Effect of Opicapone 50 mg or Levodopa 100 mg as First Strategy for the Treatment of Wearing-off in Patients With Parkinson's Disease.

This is a randomized, parallel group, multicentre, multinational, prospective, open-label exploratory study in Parkinson's disease (PD) patients to evaluate the add-on efficacy of opicapone 50 mg or an extra dose of levodopa (L-DOPA) 100 mg as first strategy for the treatment of wearing-off.

Start Date29 Nov 2021

Sponsor / Collaborator

BIAL-Portela & Cia SA

NCT04821687 / CompletedPhase 4

A Randomized, Parallel Group, Multicenter, Prospective, Open-label, Exploratory, Phase 4 Study to Evaluate the Add-on Effect of Opicapone 50 mg or Levodopa 100 mg as First Strategy for the Treatment of Wearing-off in Patients With Parkinson's Disease

A Study to evaluate the add-on efficacy and safety of opicapone 50 mg or an extra dose of L-DOPA 100 mg for the treatment of wearing-off in patients with PD.

Start Date17 Jun 2021

Sponsor / Collaborator

SK Chemicals Co. Ltd.

100 Clinical Results associated with Opicapone

100 Translational Medicine associated with Opicapone

100 Patents (Medical) associated with Opicapone

123

Literatures (Medical) associated with Opicapone

01 Dec 2024·eNeurologicalSci

Levodopa / opicapone as a complement to STN-DBS in clinical practice. A retrospective single-centre analysis.

Article

Author: Loeffler, Moritz A ; Klocke, Philipp ; Gharabaghi, Alireza ; Weiss, Daniel ; Cebi, Idil ; Loeffler, Moritz A.

Objective:

Deep brain stimulation of the subthalamic nucleus (STN-DBS) is a well-established treatment option in Parkinson's disease with motor and non-motor fluctuations allowing for postoperative reduction of dopaminergic medication. However, evidence is scarce on optimal medication adjustments following STN-DBS implantation. Opicapone allows for long-lasting inhibition of the catechol-O-methyltransferase (COMT) thereby enabling more constant dopaminergic stimulation compared to levodopa alone. However, especially COMT inhibitors are regularly discontinued after STN-DBS surgery. In this single-centre retrospective analysis, we aimed to analyse the clinical phenotype of patients selected for opicapone treatment following STN-DBS implantation and to define clinical determinants of patients requiring more intense dopamine-stabilising strategies after STN-DBS implantation.

Methods:

A patient cohort treated with STN-DBS + levodopa + opicapone (n = 16) was compared to an age-matched control cohort without opicapone treatment at baseline before and ≥ 5 months post-surgery. As main outcomes we assessed the MDS-UPDRS III and IV scores and reduction of the cumulative dopaminergic medication quantified by the levodopa equivalent dosages (LED).

Results:

Whilst the MDS-UPDRS III (median [min - max]) in patients with STN-DBS as well as anatomical electrode positions did not differ significantly between the opicapone 20 [4-40] and control cohort 14 [1-44], the patients selected for opicapone treatment showed a significantly higher degree of dyskinesias already preoperatively as reflected by a UPDRS-IV A subscore of 2 [0-4] compared to controls 0 [0-4]. Postoperatively, the opicapone cohort showed stronger motor fluctuations MDS-UPDRS IV 6 [0-14] compared to the controls 0 [0-10], albeit without statistical significance. Moreover, the opicapone cohort showed significantly less reduction of dopaminergic medication (-36.4 % vs. -46.2 % in the control cohort) following STN-DBS implantation independent from the intake of dopamine agonists.

Conclusion:

These results indicate a clinical phenotype characterised by more motor fluctuations requiring a more stable dopamine replacement therapy to address the patients' disease biology. In these cases, levodopa + COMT inhibition by opicapone represents a therapeutic approach but determination of the potential clinical benefit requires further prospective studies.

01 Oct 2024·JOURNAL OF NEUROLOGY

Opicapone for the treatment of early wearing-off in levodopa-treated Parkinson’s disease: pooled analysis of patient level data from two randomized open-label studies

Article

Author: Ferreira, Joaquim J ; Castilla-Fernández, Guillermo ; Rodrigues, Daniela M. ; Lee, Jee-Young ; Fonseca, Miguel M ; Poewe, Werner ; Ma, Hyeo-il ; Rocha, José-Francisco ; Rodrigues, Daniela M ; Holenz, Joerg ; Ma, Hyeo-Il ; Jeon, Beomseok ; Antonini, Angelo ; Stocchi, Fabrizio ; Fonseca, Miguel M. ; Ferreira, Joaquim J. ; Rascol, Olivier

Abstract:

Background:

The wearing-off phenomenon is a key driver of medication change for patients with Parkinson’s disease (PD) treated with levodopa. Common first-line options include increasing the levodopa dose or adding a catechol-O-methyltransferase (COMT) inhibitor, but there are no trials comparing the efficacy of these approaches. We evaluated the effectiveness of adjunct opicapone versus an additional 100 mg levodopa dose in PD patients with early wearing-off using pooled data from 2 randomized studies.

Methods:

The ADOPTION study program included two similarly designed 4-week, open-label studies conducted in South Korea (NCT04821687) and Europe (NCT04990284). Patients with PD, treated with 3–4 daily doses of levodopa therapy and with signs of early wearing-off were randomized (1:1) to adjunct opicapone 50 mg or an additional dose of levodopa 100 mg. Patient-level data from the two studies were pooled.

Results:

The adjusted mean [SE] change from baseline to week 4 in absolute OFF time (key endpoint) was − 62.8 min [8.8] in the opicapone group and − 33.8 min [9.0] in the levodopa 100 mg group, the difference significantly favoring opicapone (− 29.0 [− 53.8, − 4.2] min, p = 0.02). Significant differences in the Movement Disorder Society—Unified Parkinson’s Disease Rating Scale Part III subscore (− 4.1 with opicapone vs − 2.5 with levodopa 100 mg), also favored opicapone (− 1.7 [− 3.3, − 0.04], p < 0.05). Dyskinesia was the most frequently reported adverse event (opicapone 7.2% vs. levodopa 100 mg 4.2%).

Conclusions:

In these short-term trials, introducing adjunct opicapone was more effective at reducing OFF time than adding another 100 mg levodopa dose in PD patients with early signs of wearing-off.

01 Sep 2024·Drugs-Real World Outcomes

Disease Stage and Motor Fluctuation Duration Predict Drug Tolerability: A Real-Life, Prospective Italian Multicenter Study on the Use of Opicapone in Parkinson’s Disease

Article

Author: Bacchin, Ruggero ; Liccari, Marco ; Giometto, Bruno ; Antonutti, Lucia ; Malaguti, Maria Chiara ; Catalan, Mauro ; Manganotti, Paolo

BACKGROUND:

Opicapone is a third-generation catechol-O-methyl-transferase inhibitor currently used for the treatment of motor fluctuations in Parkinson's disease. Its benefit and safety have been established by clinical trials; however, data about its use in a real-life context, and particularly in an Italian population of patients with Parkinson's disease, are missing.

OBJECTIVES:

We aimed to gather data about the real-life tolerability/safety of opicapone when used for the treatment of Parkinson's disease-related motor fluctuations.

METHODS:

We enrolled 152 consecutive patients with Parkinson's disease and followed them for 2 years after opicapone introduction. We obtained baseline clinical and demographical information, including disease duration, stage, phenotype, as well as axial and non-motor symptoms. We collected the reasons for any treatment interruption and adverse events emerging after opicapone introduction.

RESULTS:

Eighty-nine (58%) patients reported adverse events and 46 (30%) patients discontinued the treatment. Adverse events occurred less frequently in "earlier" patients accordingly to the disease course and L-Dopa treatment pathway; a motor fluctuation duration ≥12 months and Hoehn and Yahr scale score ≥2.5 were the main predictors of therapy withdrawal.

CONCLUSIONS:

This study confirms the good tolerability/safety profile of opicapone in a real-life setting and provides country-specific data for Italian patients with Parkinson's disease.

News (Medical) associated with Opicapone

07 Dec 2023

·www.businesswire.com

Amneal and BIAL Announce U.S. Licensing Agreement for ONGENTYS® (opicapone)

BRIDGEWATER, N.J. & PORTO, Portugal--( BUSINESS WIRE )--Amneal Pharmaceuticals, Inc. (NYSE: AMRX) (“Amneal”) and BIAL - Portela & Ca., S.A. (“BIAL”), today announced a licensing agreement where Amneal will have exclusive rights to market and distribute ONGENTYS ® (opicapone) in the U.S. starting on December 18, 2023. Amneal expects to begin distribution of ONGENTYS ® in early 2024. ONGENTYS ® is BIAL’s proprietary once-daily, peripherally-acting, highly-selective catechol-O-methyltransferase inhibitor (COMT inhibitor), approved by the U.S. Food and Drug Administration (“FDA”) in 2020 as an add-on treatment to carbidopa/levodopa (CD/LD) in patients with Parkinson’s disease (PD) experiencing “Off” episodes. Carbidopa/levodopa (CD/LD), which works to control the symptoms of PD, has been the gold-standard treatment for PD since the 1970s. As the disease progresses, patients on LD start experiencing motor complications such as the “wearing-off phenomenon,” which are periods where LD is no longer providing enough relief from PD symptoms and people experience what is referred to as “Off” time. Wearing-off is common, with around 50% of patients reporting it in the first five years after PD diagnosis. 1 “Off” time can greatly disrupt a patient’s daily routine by inhibiting their ability to perform tasks or care for themselves. ONGENTYS ® works by inhibiting the COMT enzyme – which breaks down LD – making more LD available to reach the brain, thereby reducing “Off” time. ONGENTYS offers patients living with Parkinson´s disease an effective, once-daily adjunctive treatment option for “Off” episodes. It is the first and only LD optimizer approved for once-daily use. Amneal views ONGENTYS ® as highly complementary to Rytary ® and IPX203. “As a company committed to the Parkinson’s community, Amneal continues to look for more ways to serve and support people with PD, their loved ones, and the physicians who treat them,” said Joe Renda, Senior Vice President, Chief Commercial Officer Specialty. “We understand the importance of minimizing ‘Off’ time and increasing “On” time without troublesome dyskinesia when treating PD. We look forward to working with BIAL to ensure ONGENTYS ® remains available for patients in the U.S. and look to further grow this key adjunctive therapy. This is an exciting complement to our specialty branded portfolio and pipeline of best-in-class treatment options for PD.” “We are fully committed to creating value for people living with severe neurological conditions worldwide, being the U.S. is a key geography for BIAL. Amneal shares our long-term vision for ONGENTYS ® , and our partnership will allow us to expand its accessibility, continuing to make a difference for patients suffering from Parkinson’s disease in the U.S.,” said Max Bricchi, Executive Vice President, Chief Commercial Officer of BIAL. The financial terms of the agreement were not disclosed, and any incremental expenses associated with this product are contemplated within Amneal’s guidance. Important Information Approved Use ONGENTYS ® (opicapone) capsules is a prescription medicine used with levodopa and carbidopa in people with Parkinson's disease (PD) who are having "OFF" episodes. It is not known if ONGENTYS ® is safe and effective in children. Important Safety Information Do not take ONGENTYS ® if you: take a type of medicine called a non-selective monoamine-oxidase (MAO) inhibitor. have a tumor that secretes hormones known as catecholamines. Before taking ONGENTYS ® , tell your healthcare provider about all of your medical conditions, including if you: have daytime sleepiness from a sleep disorder, have unexpected periods of sleep or sleepiness, or take a medicine to help you sleep or that makes you feel sleepy. have had intense urges or unusual behaviors, including gambling, increased sex drive, binge eating, or compulsive shopping. have a history of uncontrolled sudden movements (dyskinesia). have had hallucinations or psychosis. have liver or kidney problems. are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Especially tell your healthcare provider if you take nonselective MAO inhibitors (such as phenelzine, tranylcypromine, and isocarboxazid) or catecholamine medicines (such as isoproterenol, epinephrine, norepinephrine, dopamine, and dobutamine), regardless of how you take the medicine (by mouth, inhaled, or by injection). ONGENTYS ® and other medicines may affect each other causing side effects. ONGENTYS ® may affect the way other medicines work, and other medicines may affect how ONGENTYS ® works. What should I avoid while taking ONGENTYS ® ? Do not drive, operate machinery, or do other dangerous activities until you know how ONGENTYS ® affects you. What are the possible side effects of ONGENTYS ® ? ONGENTYS ® may cause serious side effects, including: Falling asleep during normal activities such as driving a car, talking or eating while taking ONGENTYS ® or other medicines used to treat Parkinson's disease, without being drowsy or without warning. This may result in having accidents. Your chances of falling asleep while taking ONGENTYS ® are higher if you take other medicines that cause drowsiness. Low blood pressure or dizziness , light headedness, or fainting. Uncontrolled sudden movements (dyskinesia). ONGENTYS ® may cause uncontrolled sudden movements or make such movements worse or happen more often. Seeing, hearing, or feeling things that are not real (hallucinations), believing things that are not real (delusions), or aggressive behavior. Unusual urges (impulse control and compulsive disorders) such as urges to gamble, increased sexual urges, strong urges to spend money, binge eating, and the inability to control these urges. Tell your healthcare provider if you experience any of these side effects or notice changes in your behavior. The most common side effects of ONGENTYS ® include uncontrolled sudden movements (dyskinesia), constipation, increase in an enzyme called blood creatine kinase, low blood pressure, and weight loss. These are not all the possible side effects of ONGENTYS ® . Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. Please see ONGENTYS ® full Product Information . About Parkinson’s Disease Parkinson’s disease (PD) has become the fastest-growing neurological disorder worldwide, with approximately 1 million people diagnosed in the U.S. 2,3 It is a progressive disorder of the central nervous system (CNS) that affects dopamine-producing neurons in the brain that affect movement. PD is characterized by slowness of movement, stiffness, resting tremor, and impaired balance. 4 While PD is not considered a fatal disease, it is associated with significant morbidity and disability. 5 The average age at diagnosis for people with PD is 60; as people live longer, the number of people living with PD is predicted to grow significantly over the coming decades. 2,6 About ONGENTYS ® (opicapone): ONGENTYS ® is a once-daily, oral, peripheral, selective and reversible catechol-O-methyltransferase (COMT) inhibitor approved by the FDA as an add-on treatment to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes. The FDA approval of ONGENTYS ® is supported by data from two pivotal trials in patients with Parkinson's disease and end-of-dose motor fluctuations (BIPARK-I and II), and the results demonstrated an acceptable tolerability profile, combined with efficacy in reducing OFF-time in adult patients with Parkinson's disease and end-of-dose motor fluctuations. 7 These studies also led to the approval of opicapone in the European Union, UK, Japan, Korea, Australia, and other countries. 8 About Amneal Amneal Pharmaceuticals, Inc. (NYSE: AMRX), headquartered in Bridgewater, NJ, is a fully integrated global pharmaceuticals company. We make healthy possible through the development, manufacturing, and distribution of a diverse portfolio of approximately 270 pharmaceutical products, primarily within the United States. In its Generics segment, the Company is expanding across a broad range of complex product categories and therapeutic areas, including injectables and biosimilars. In its Specialty segment, Amneal has a growing portfolio of branded pharmaceuticals focused primarily on central nervous system and endocrine disorders, with a pipeline focused on unmet needs. Through its AvKARE segment, the Company is a distributor of pharmaceuticals and other products for the U.S. federal government, retail, and institutional markets. For more information, please visit www.amneal.com . About BIAL BIAL is an innovation-driven pharmaceutical company aiming to improve people’s lives worldwide. With 99 years of experience, BIAL is a fully integrated company strongly committed to therapeutic innovation, being neurosciences as its major area of research. Based on its innovative medicines and with a consistent partnering program, BIAL has extended its presence worldwide. The company has affiliates in three different continents – Europe, America, and Africa – and its products are present in fifty countries, including the US, Japan, Germany, Canada, Korea, and Australia, fulfilling its purpose of making a real difference in the lives of people living with severe diseases across the world. For more information about BIAL, please visit: www.bial.com . Cautionary Statement on Forward-Looking Statements Certain statements contained herein, regarding matters that are not historical facts, may be forward-looking statements (as defined in the U.S. Private Securities Litigation Reform Act of 1995). Such forward-looking statements include statements regarding management’s intentions, plans, beliefs, expectations, financial results, or forecasts for the future, including among other things: discussions of future operations, including international expansion; expected or estimated operating results and financial performance; the Company’s growth prospects and opportunities as well as its strategy for growth; product development and launches; the successful commercialization and market acceptance of new products, and other non-historical statements. Words such as “plans,” “expects,” “will,” “anticipates,” “estimates,” and similar words, or the negatives thereof, are intended to identify estimates and forward-looking statements. The reader is cautioned not to rely on these forward-looking statements. These forward-looking statements are based on current expectations of future events, including with respect to future market conditions, company performance and financial results, operational investments, business prospects, new strategies and growth initiatives, the competitive environment, and other events. If the underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of the Company. Such risks and uncertainties include, but are not limited to: our ability to successfully develop, license, acquire and commercialize new products on a timely basis; the competition we face in the pharmaceutical industry from brand and generic drug product companies, and the impact of that competition on our ability to set prices; our ability to obtain exclusive marketing rights for our products; our ability to manage our growth through acquisitions and otherwise; our revenues are derived from the sales of a limited number of products, a substantial portion of which are through a limited number of customers; the continuing trend of consolidation of certain customer groups; our dependence on third-party suppliers and distributors for raw materials for our products and certain finished goods; our ability to complete the proposed holding company reorganization on the anticipated timeline or at all and to realize the expected benefits of such reorganization; our substantial amount of indebtedness and our ability to generate sufficient cash to service our indebtedness in the future, and the impact of interest rate fluctuations on such indebtedness; our ability to secure satisfactory terms when negotiating a refinancing or other new indebtedness; our dependence on third-party agreements for a portion of our product offerings; legal, regulatory and legislative efforts by our brand competitors to deter competition from our generic alternatives; risks related to federal regulation of arrangements between manufacturers of branded and generic products; our reliance on certain licenses to proprietary technologies from time to time; the significant amount of resources we expend on research and development; the risk of product liability and other claims against us by consumers and other third parties; risks related to changes in the regulatory environment, including U.S. federal and state laws related to healthcare fraud abuse and health information privacy and security and changes in such laws; changes to Food and Drug Administration product approval requirements; the impact of healthcare reform and changes in coverage and reimbursement levels by governmental authorities and other third-party payers; our potential expansion into additional international markets subjecting us to increased regulatory, economic, social and political uncertainties, including recent events affecting the financial services industry; our ability to identify, make and integrate acquisitions or investments in complementary businesses and products on advantageous terms; the impact of global economic, political or other catastrophic events; our ability to attract, hire and retain highly skilled personnel; our obligations under a tax receivable agreement may be significant; and the high concentration of ownership of our Class A Common Stock and the fact that we are controlled by the Amneal Group. The forward-looking statements contained herein are also subject generally to other risks and uncertainties that are described from time to time in the Company’s filings with the Securities and Exchange Commission, including under Item 1A, “Risk Factors” in the Company’s most recent Annual Report on Form 10-K and in its subsequent reports on Forms 10-Q and 8-K. Investors are cautioned not to place undue reliance on any such forward-looking statements, which speak only as of the date they are made. Forward-looking statements included herein speak only as of the date hereof and we undertake no obligation to revise or update such statements to reflect the occurrence of events or circumstances after the date hereof. References: Stocchi F, et al. Parkinsonism Relat Disord. 2014;20(2):204-11 Dorsey ER et al. JAMA Neurol. 2018;75(1):9-10. Marras et al. NPJ Parkinsons Dis. 2018;4:21. NINDS. Parkinson’s disease: challenges, progress, and promise. Reviewed August 2019. Accessed April 16, 2021. Data Monitor: Gibrat et al., 2009; Goldenberg, 2008; Muangpaisan et al., 2009; Pringsheim et al., 2014. John Hopkins Medicine. Young-Onset Parkinson’s disease. Accessed August 17, 2021. Ferreira, J. et al., Eur J Neurol. 2019 Jul;26(7) 953-960 Ferreira, J. et al., Neurol Ther. 2022 Sep;11(3):1409-1425

Drug ApprovalClinical ResultLicense out/in

03 May 2023

·www.biospace.com

Neurocrine Biosciences Reports First Quarter 2023 Financial Results

INGREZZA® (valbenazine) First Quarter Net Product Sales of $410 Million INGREZZA® (valbenazine) 2023 Net Product Sales Guidance Reiterated at $1.67 - $1.77 Billion SAN DIEGO, May 3, 2023 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced its financial results for the first quarter ended March 31, 2023 and provided an update on its 2023 financial guidance. "INGREZZA's first quarter results highlight the steady progress we are making to help improve the lives of patients living with tardive dyskinesia, yet we still have a tremendous opportunity to help even more patients for many years to come," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "We are executing well across our clinical portfolio and anticipate several important milestones in the months ahead including the August 20th PDUFA date for valbenazine for the treatment of chorea associated with Huntington disease. In addition, we remain on-track for top-line data in the second half of this year for several investigational programs, including crinecerfont for the treatment of pediatric and adult congenital adrenal hyperplasia." First Quarter INGREZZA Net Product Sales Highlights: INGREZZA first quarter 2023 net product sales were $410 million and grew 36% compared vs. the first quarter 2022 Record number of new patients received therapy during the first quarter of 2023 First Quarter Financial Highlights: First quarter 2023 GAAP net loss and loss per share of $77 million and $0.79, respectively, compared with first quarter 2022 GAAP net income and earnings per share of $14 million and $0.14, respectively First quarter 2023 non-GAAP net loss and loss per share of $50 million and $0.51, respectively, compared with first quarter 2022 non-GAAP net income and earnings per share of $30 million and $0.30, respectively Differences in first quarter 2023 GAAP and non-GAAP operating expenses compared with first quarter 2022 driven by: Acquired In-Process R&D (IPR&D) expense of $144 million in first quarter 2023 associated with expansion of strategic partnership with Voyager Therapeutics, Inc. (Voyager) Increased R&D expense in support of an expanded and advancing clinical portfolio Increased SG&A expense primarily due to ongoing commercial initiatives, including the deployment of the expanded salesforce in April 2022 At March 31, 2023, the Company had cash, cash equivalents and marketable securities of approximately $1.1 billion A reconciliation of GAAP to non-GAAP financial results can be found in Table 3 and Table 4 at the end of this earnings release. Recent Events: On January 8, 2023, we entered into a new strategic collaboration with Voyager, which became effective on February 21, 2023, to acquire the worldwide rights to Voyager's GBA1 gene therapy program for Parkinson's disease and other GBA1-mediated diseases and three gene therapy programs directed to rare central nervous system targets for up-front consideration of $175 million. In the first quarter of 2023, we provided BIAL with written notice of termination of the license agreement to commercialize and market ONGENTYS® (opicapone), an approved adjunctive therapy for patients with Parkinson's disease, in the United States and Canada. We determined that continued commercialization of ONGENTYS is unsustainable. The termination is anticipated to be effective in December 2023. ONGENTYS is an important, safe and effective adjunctive treatment option for Parkinson's disease patients. We intend to work with BIAL to ensure an orderly transition of the commercialization of ONGENTYS and to ensure patients have continued access to ONGENTYS. Reiterated 2023 INGREZZA Sales and Updated Operating Expense Guidance: 2023 Expected Pipeline Milestones and Key Activities Key: VMAT2 = Vesicular Monoamine Transporter 2; CFR1 = Corticotropin-Releasing Factor Type 1; NaV1.6 = Sodium Channel, Voltage-Gated; M1 / M4 = M1 / M4 Muscarinic Receptor; GPR = Orphan G Protein Coupled Receptor Neurocrine Biosciences Partners: * Mitsubishi Tanabe Pharma Corporation has commercialization rights in East Asia; ** In-Licensed from Xenon Pharmaceuticals; † Partnered with Takeda Pharmaceutical Company Limited; ‡ In-Licensed from Sosei Group Corporation Conference Call and Webcast Today at 8:00 AM Eastern Time Neurocrine Biosciences will hold a live conference call and webcast today at 8:00 a.m. Eastern Time (5:00 a.m. Pacific Time). Participants can access the live conference call by dialing 800-895-3361 (US) or 785-424-1062 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine Biosciences' website under Investors at . A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month. About Neurocrine Biosciences Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs, but few options. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neurological, neuroendocrine, and neuropsychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis* and uterine fibroids*, as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas. For three decades, we have applied our unique insight into neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly pursue medicines to ease the burden of debilitating diseases and disorders, because you deserve brave science. For more information, visit neurocrine.com, and follow the company on LinkedIn, Twitter and Facebook. (*in collaboration with AbbVie) Non-GAAP Financial Measures In addition to the financial results and financial guidance that are provided in accordance with accounting principles generally accepted in the United States (GAAP), this press release also contains the following non-GAAP financial measures: non-GAAP R&D expense, non-GAAP SG&A expense, and non-GAAP net income and net income per share. When preparing the non-GAAP financial results and guidance, the Company excludes certain GAAP items that management does not consider to be normal, including recurring cash operating expenses that might not meet the definition of unusual or non-recurring items. In particular, these non-GAAP financial measures exclude: non-cash stock-based compensation expense, loss on extinguishment of convertible senior notes, non-cash interest expense related to convertible debt, non-cash amortization expense related to acquired intangible assets, acquisition-related transaction costs, changes in fair value of equity security investments, changes in foreign currency exchange rates and certain adjustments to income tax expense. These non-GAAP financial measures are provided as a complement to results provided in accordance with GAAP as management believes these non-GAAP financial measures help indicate underlying trends in the Company's business, are important in comparing current results with prior period results and provide additional information regarding the Company's financial position. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally and to manage the Company's business and evaluate its performance. The Company provides guidance regarding combined R&D and SG&A expenses on both a GAAP and a non-GAAP basis. A reconciliation of these GAAP financial results to non-GAAP financial results is included in the attached financial information. Forward-Looking Statements In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements related to: the benefits to be derived from our products and product candidates; the value our products and/or our product candidates may bring to patients; the continued success of INGREZZA; our financial and operating performance, including our future revenues, expenses, or profits; our collaborative partnerships; expected future clinical and regulatory milestones; and the timing of the initiation and/or completion of our clinical, regulatory, and other development activities and those of our collaboration partners. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are: our future financial and operating performance; risks and uncertainties associated with the commercialization of INGREZZA; risks related to the development of our product candidates; risks associated with our dependence on third parties for development, manufacturing, and commercialization activities for our products and product candidates, and our ability to manage these third parties; risks that the FDA or other regulatory authorities may make adverse decisions regarding our products or product candidates; risks that clinical development activities may not be initiated or completed on time or at all, or may be delayed for regulatory, manufacturing, or other reasons, may not be successful or replicate previous clinical trial results, may fail to demonstrate that our product candidates are safe and effective, or may not be predictive of real-world results or of results in subsequent clinical trials; risks that the potential benefits of the agreements with our collaboration partners may never be realized; risks that our products, and/or our product candidates may be precluded from commercialization by the proprietary or regulatory rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; risks associated with U.S. federal or state legislative or regulatory and/or policy efforts which may result in, among other things, an adverse impact on our revenues or potential revenue; risks associated with potential generic entrants for our products; and other risks described in our periodic reports filed with the SEC, including our Quarterly Report on Form 10-Q for the quarter ended March 31, 2023. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof. Company Codes: NASDAQ-NMS:NBIX

License out/inFinancial StatementGene Therapy

04 Nov 2022

·www.prnewswire.com

Fosun Participates in the CIIE for the Fifth Consecutive Year with the Debut of Innovative Products in China

HONG KONG, Nov. 4, 2022 /PRNewswire/ -- The 5th China International Import Expo (hereinafter referred to as the "CIIE") will be held in Shanghai from 5 November to 10 November. Fosun will participate in the CIIE for the fifth consecutive year with its overseas member companies and partners from the global pharmaceutical and health industry, presenting a variety of China's first and industry-leading medical devices and innovative drugs to showcase Fosun's latest achievements in technology and innovation and global industrial operations and promote more "exhibits" to launch as "products" in China. The CIIE is the world's first import-themed national-level expo, showcasing the world's most cutting-edge and representative high-quality products and services. A total of 145 countries, regions and international organizations will take part in this year's CIIE, and 284 Fortune Global 500 companies and industry-leading companies will participate in the Enterprise & Business Exhibition, exceeding last year's. At this year's CIIE, the da Vinci Xi surgical system and the Ion robotic-assisted bronchoscopy system to be presented at Fosun Health's booth have drawn a lot attention. The da Vinci surgical system introduced by Fosun has been a popular exhibit for five consecutive years since its debut at the first CIIE in 2018. In the past five years, Fosun has continued to promote the business expansion of the da Vinci surgical systems and technologies in the Chinese market. As of now, more than 300 da Vinci surgical systems have been installed in China, benefiting more than 366,000 patients in China. In August 2022, Intuitive Fosun's Medical Robot Manufacturing and R&D Center started construction in Pudong, Shanghai. It is expected to be completed in 2025 and reach production the following year to benefit more patients in China. In the field of innovative drugs, Yi Kai Da (Yescarta), the CAR-T cell therapy presented by Fosun at the first CIIE, was officially approved by the National Medical Products Administration (NMPA) in June 2021 and became the first CAR-T cell therapy approved for marketing in China. As of the end of September 2022, Yi Kai Da has been included in urban customized commercial health insurance of 54 provinces and municipalities and over 60 commercial insurances, while the number of treatment centers on file has reached nearly 120. At this year's CIIE, Fosun Kite will further showcase the latest achievements of Yi Kai Da in the fields of drug research and development, production and operation, and innovative commercialization. The MRIdian Linac magnetic resonance guided radiation therapy system, which has been featured at the CIIE for three consecutive years, will be presented at the Fosun Health booth as a "product" this year. As of now, 55 systems of MRIdian Linac have been set up worldwide, treating more than 25,000 patients. MRIdian Linac has been approved by the NMPA in August 2022, providing more Chinese cancer patients with a new option for radiation therapy. Fosun Health will also present the world's first oral targeted drug for psoriasis, Otelza (apremilast tablets), the third-generation COMT inhibitor, Ongentys (opicapone), a medical-grade home beauty device powered by Israel's energy-based technology, LMNT one, and many other world-leading innovative drugs and medical devices. In addition, the 5th Hongqiao International Health Technology Innovation Forum, a supporting event of the CIIE hosted by Fosun, will be held on the morning of 6 November. Themed around "Gathering Innovation and Breakthroughs, Building a Healthy Future", the forum will bring together experts and professionals to discuss the global medical technology innovation trends and the innovative development of China's health industry from a global perspective. After years of accumulation, globalization and innovation have become the core competencies of Fosun's sustainable and steady development. As of now, Fosun has established profound industrial presence in over 30 countries and regions. Fosun's investment in technology and innovation in 2021 reached RMB8.9 billion, while its investment in technology and innovation for the first half of 2022 reached RMB4.6 billion, representing a year-on-year increase of 21%. As of the end of 2021, Fosun owned over 1,500 patents for inventions with more than 500 new products under development. "The CIIE is an excellent display window for Fosun to connect with global technologies and continuously create happier lives for families." Guo Guangchang, Chairman of Fosun International, said that as a global innovation-driven consumer group, Fosun will, as always, adhere to the globalization strategy, continue to increase investment in research and development, and continuously meet the needs of global families with high-quality products and services to create happier lives. SOURCE Fosun

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100 Deals associated with Opicapone

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KEGG	Wiki	ATC	Drug Bank
D10825	Opicapone	N04BX04	DB11632

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Approved

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Indication	Country/Location	Organization	Date
Parkinson Disease	EU	BIAL-Portela & Cia SA	24 Jun 2016
Parkinson Disease	IS	BIAL-Portela & Cia SA	24 Jun 2016
Parkinson Disease	LI	BIAL-Portela & Cia SA	24 Jun 2016
Parkinson Disease	NO	BIAL-Portela & Cia SA	24 Jun 2016

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Indication	Highest Phase	Country/Location	Organization	Date
Primary Parkinson's disease	Phase 3	CN	BIAL-Portela & Cia SA	17 Apr 2023
Primary Parkinson's disease	Phase 3	CN	Shanghai Fosun Pharmaceutical Industrial Development Co., Ltd.	17 Apr 2023
Young onset Parkinson disease	Phase 3	BG	BIAL-Portela & Cia SA	31 May 2021
Epilepsy	Phase 1	-	BIAL-Portela & Cia SA	01 Feb 2014
Parkinsonian Disorders	Phase 1	CA	BIAL-Portela & Cia SA	01 May 2008

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


MDS2023	Not Applicable	-	-	Opicapone	svsydoddxi(bnkyxaempy) = oysgqzgnyu deayfiaeze (tonwrkcrxq )	Positive	27 Aug 2023
MDS2023	Not Applicable	-	-	Entacapone	svsydoddxi(bnkyxaempy) = iklvmmzsef deayfiaeze (tonwrkcrxq )	Positive	27 Aug 2023
MDS2023	Not Applicable	Psychotic Disorders	-	Opicapone	phjjclielr(ktibictssv) = mqpkkqrnzz ejdrbawjpq (nngengafyb )	-	27 Aug 2023
P14-11.014 (AAN2023)	Phase 1	-	-	Opicapone 50 mg	qqdcoojvfl(gwszmsjwoa) = ykrizlkpjq qlgcbswctq (xpotqreedy, 80.0 - 82.9)	Positive	25 Apr 2023
OPEN-PD (MDS2022)	Not Applicable	Parkinson Disease	33	Opicapone	gbphqyygql(vbopuqtzmq) = A total of 13 adverse events in 11 patients (33.3%) were reported, 1 of which was severe (not related to opicapone). Dyskinesias and nausea were the most frequent (6.1%) wzjinnfpvo (cxiscnlpiu )	Positive	15 Sep 2022
MDS2022	Not Applicable	Parkinson Disease	187	Opicapone	pfmjfjniqr(bolnoulgqq) = cbvinrwhzk vsguzzxtrd (oxucokrvhe ) View more	Positive	15 Sep 2022
MDS2022	Phase 1	-	-	Once-Daily Opicapone 50 mg	yntrmevcah(vagbhrrrtp) = nxkvsdweof taihzqpbwp (dtiggfzagn )	-	15 Sep 2022
MDS2022	Not Applicable	-	8	Levodopa/DCI	xneimbffra(hakcabonoj) = wftbqquyac xyntaggpca (enrtiokoeh, (0.15))	Positive	15 Sep 2022
MDS2022	Not Applicable	-	8	Opicapone 25 mg	xneimbffra(mtnusebmaq) = sjkdvgecbr vudfbeyosp (vfbsxtoylr, (0.13))	Positive	15 Sep 2022
OPTI-ON (ANA2022)	Not Applicable	-	243	Sinemet	huiisggvlx(lehldxwcex) = rwavpllonb otsykogijm (dtuniqtine )	-	14 Sep 2022
OPTI-ON (ANA2022)	Not Applicable	-	243	Rytary	huiisggvlx(lehldxwcex) = lcwbsxxolr otsykogijm (dtuniqtine )	-	14 Sep 2022
MDS2021	Not Applicable	-	-	Opicapone	rccgrdvoce(omkzuxunxt) = shdjijhugt waayffuurd (kxakyoinuk ) View more	-	17 Sep 2021
OPTIPARK (MDS2021)	Not Applicable	-	363	Opicapone 50 mg	axfggavepr(jqmoskqaaz) = 4.4% knjvsigjer (xmehekmpar ) View more	Positive	17 Sep 2021

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