1. SAN DIEGO, Feb. 23, 2021 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) will present at the Cowen 41st Annual Health Care Conference at 1:30 p.m. Eastern Time on Tuesday Mar. 2, 2021. Kevin Gorman, Chief Executive Officer, will present at the conference.

    The live presentation will be webcast and may be accessed on the Company's website under Investors at www.neurocrine.com. A replay of the presentation will be available on the website approximately one hour after the conclusion of the events and will be archived for approximately one month.

    About Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company dedicated to discovering, developing and delivering life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis*, uterine fibroids* and clinical programs in multiple therapeutic areas. For nearly three decades, Neurocrine Biosciences has specialized in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn(*in collaboration with AbbVie)

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    SOURCE Neurocrine Biosciences, Inc.

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  2. SAN DIEGO, Feb. 4, 2021 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced its financial results for the fourth quarter and full-year ended December 31, 2020 and provided full-year 2021 financial expense guidance.

    "In 2020, we served more patients with tardive dyskinesia than ever before despite the pandemic weighing on the development of the overall market. We are pleased with the recently updated guidelines from the American Psychiatric Association that now recommend first-line treatment for tardive dyskinesia with a VMAT2 inhibitor, which we hope will benefit even more patients as the vast majority of patients living with tardive dyskinesia remain undiagnosed," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "In 2021, we plan to initiate eight mid-to-late stage clinical studies and look forward to important data read-outs for NBI-1065844 in the negative symptoms of schizophrenia and valbenazine for the treatment of chorea associated with Huntington's Disease."

    Financial Highlights



    Three Months Ended

    December 31,



    Twelve Months Ended

    December 31,

    (unaudited, in millions, except per share data)

    2020



    2019



    2020



    2019

    Revenues:















    Product sales, net

    $

    241.3





    $

    237.9





    $

    994.1





    $

    752.9



    Collaboration revenue

    6.6





    6.2





    51.8





    35.2



    Total revenues

    $

    247.9





    $

    244.1





    $

    1,045.9





    $

    788.1



















    GAAP Research and Development (R&D)

    $

    66.7





    $

    55.3





    $

    275.0





    $

    200.0



    Non-GAAP R&D

    $

    59.4





    $

    47.9





    $

    221.3





    $

    164.2



















    GAAP Selling, General and Administrative (SG&A)

    $

    106.5





    $

    101.3





    $

    433.3





    $

    354.1



    Non-GAAP SG&A

    $

    92.8





    $

    87.4





    $

    367.0





    $

    304.6



















    GAAP net income

    $

    347.9





    $

    34.0





    $

    407.3





    $

    37.0



    GAAP net income per share – diluted

    $

    3.58





    $

    0.35





    $

    4.16





    $

    0.39



















    Non-GAAP net income

    $

    88.1





    $

    102.2





    $

    402.3





    $

    283.8



    Non-GAAP net income per share – diluted

    $

    0.91





    $

    1.05





    $

    4.11





    $

    2.96



















    (unaudited, in millions)



    December 31,

    2020



    December 31,

    2019

    Total cash, cash equivalents and debt securities available-for-sale

    $

    1,028.1





    $

    970.2



    Fourth Quarter and Full-Year Net Product Sales and Revenues Highlights:

    • INGREZZA net product sales for the fourth quarter and full-year 2020 were $240 million and $993 million, respectively, representing an increase of 1% and 32% versus respective 2019 comparable periods
    • INGREZZA inventory adjusted net product sales for the fourth quarter of 2020 were $258 million representing 4% sequential growth vs. the third quarter of 2020
    • INGREZZA end of fourth quarter 2020 days-on-hand channel inventory decreased by $18 million relative to the third quarter
    • INGREZZA new prescriptions increased in the fourth quarter of 2020 vs. the third quarter of 2020
    • Refill and persistency rates continued to be strong for existing INGREZZA patients
    • ONGENTYS launched in the United States in late September 2020 and net product sales for the fourth quarter of 2020 were approximately $1 million reflecting growing uptake throughout the quarter
    • Elagolix royalties received from AbbVie on combined fourth quarter 2020 net sales of ORILISSA® (elagolix tablets) and ORIAHNNTM (elagolix, estradiol and norethindrone acetate capsules and elagolix capsules) totaled $6 million

    Financial Highlights:

    • Fourth quarter 2020 GAAP net income and diluted earnings per share were approximately $348 million and $3.58, respectively, compared with approximately $34 million and $0.35, respectively, in the fourth quarter of 2019, primarily driven by a non-cash tax benefit of $296 million related to the release of substantially all of the Company's valuation allowance against its deferred tax assets on December 31, 2020
    • Fourth quarter 2020 non-GAAP net income and diluted earnings per share were approximately $88 million and $0.91, respectively, compared with approximately $102 million and $1.05, respectively, in the fourth quarter of 2019 driven by:
      • Increased Research and Development (R&D) expense primarily due to increased investment across our expanded pipeline programs and higher headcount costs
      • Increased Selling, General and Administrative (SG&A) expense primarily due to increased investment in marketing initiatives and higher headcount costs
    • 2020 full-year GAAP and non-GAAP net income of $407 million and $402 million, respectively, represents year-over-year growth of approximately 10 times and 41%, respectively
    • Total debt outstanding decreased by $136 million to $381 million after repurchase of approximately 26% of debt outstanding during the fourth quarter of 2020. The total aggregate repurchase price of $187 million was paid in cash and the transaction resulted in an $18 million loss recognized during the fourth quarter of 2020.
    • At December 31, 2020, the Company had cash, cash equivalents and debt securities available-for-sale of $1.0 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.

    Income Tax Benefit:

    The Company's fourth quarter financial results include the reversal of substantially all of the valuation allowance recorded against the deferred tax assets of the Company. This reversal resulted in the recognition of a non-cash income tax benefit in the fourth quarter 2020 of $296 million, or $3.05 earnings per diluted share. The Company has performed a continuing evaluation of its deferred tax asset valuation allowance on a quarterly basis. The Company has now concluded that, as of December 31, 2020, it is more likely than not that the Company will generate sufficient taxable income within the applicable net operating loss and R&D carryforward periods to realize substantially all of its deferred tax assets. This conclusion, and the resulting reversal of the deferred tax asset valuation allowance, is based upon consideration of a number of factors, including the Company's strong financial performance over the past few years and its forecast of future profitability.

    After recognizing the valuation allowance reversal, the Company's net deferred tax assets totaled $319 million at December 31, 2020, net of a valuation allowance of $50 million. The ability to recognize the remaining deferred tax assets that continue to be subject to a valuation allowance will be evaluated on a quarterly basis to determine if there are significant events that would affect the Company's ability to utilize these deferred tax assets. As a result of this reversal, the Company will begin recording federal and state tax expense on its earnings beginning in the first quarter of 2021. No federal cash tax is expected in 2021 based upon a net operating loss position of approximately $500 million entering 2021.

    Recent Events

    • In October 2020, the U.S. Food and Drug Administration (FDA) requested additional non-clinical data to support the Investigational New Drug Application (IND) we submitted in August 2020 in support of a Phase II clinical study for NBI-921352 in patients with SCN8A Developmental Epileptic Encephalopathy (SCN8A-DEE). Based on feedback received in January 2021, we plan to initiate a Phase II clinical study in adolescent patients (aged 12 years and older) with SCN8A-DEE in the third quarter of 2021, and the study protocol will be amended to include younger pediatric patients (aged 2-11 years) with SCN8A-DEE as soon as the FDA has reviewed and approved additional non-clinical information. We are also advancing clinical plans to initiate a Phase II clinical study of NBI-921352 for the treatment of adult focal epilepsy in 2021. In addition, in October 2020, we announced the FDA granted us Rare Pediatric Disease Designation for NBI-921352 for the treatment of SCN8A-DEE.
    • In November 2020, the Company announced the initiation of a Phase II study of NBI-827104 (formerly ACT-709478) in Epileptic Encephalopathy with Continuous Spike and Wave during Sleep (CSWS), a rare pediatric epilepsy. NBI-827104 was licensed from Idorsia and is a potent, selective, orally active and brain penetrating T-type calcium channel blocker.
    • In February 2021, the Mitsubishi Tanabe Pharma Corporation (MTPC) reported positive top-line results from the J-KINECT Phase III Study, designed to evaluate the efficacy and safety of valbenazine in tardive dyskinesia. Detailed results from this trial will be presented at a future medical conference. With positive data in hand, a marketing authorization with the Ministry of Health and Welfare is planned for 2021 in Japan. In addition, MTPC submitted filings for marketing authorization in South Korea, Thailand, Singapore, Indonesia, and Malaysia in 2020.
    • In February 2021, the Company notified Voyager Therapeutics, Inc. (Voyager) of the Company's termination of the NBIb-1817 (VY-AADC) development program in Parkinson's disease (the Program). The Company will work to transfer the rights to the Program to Voyager by August 2, 2021.

    Full-Year 2021 Expense Guidance



    Range

    (in millions)

    Low



    High

    Combined GAAP R&D and SG&A expenses

    $

    800





    $

    850



    Combined Non-GAAP R&D and SG&A expenses

    $

    675





    $

    725



     

    • GAAP and Non-GAAP expense guidance range reflects increased investment in:
      • R&D expense including meaningful investments in collaboration programs (specifically with Idorsia, Xenon and Takeda) and the planned initiation of eight mid-to-late-stage clinical studies
      • INGREZZA and ONGENTYS marketing costs
    • GAAP-only guidance includes approximately $125 million of share-based compensation. GAAP-only guidance does not include any potential milestones or in-process research and development costs associated with current collaborations or future business development activities.

    2021 Expected Milestones and Key Activities

    Program

    Indication

    2021 Milestones / Key Activities

    Valbenazine

    Chorea in Huntington's Disease

    Phase III Top-Line Data Expected in Q4 2021

    Tardive Dyskinesia

    Marketing Authorization with Ministry of Health and Welfare in Japan

    Neurological Indication

    Initiate Phase III Registrational Study

    Psychiatric Indication

    Initiate Registrational Study

    Crinecerfont

    Congenital Adrenal Hyperplasia (Adult)

    Continue Phase III Registrational Study Enrollment

    Congenital Adrenal Hyperplasia (Pediatric)

    Initiate Phase III Registrational Study

    NBI-1065844

    Negative Symptoms of Schizophrenia

    Phase II Top-Line Data Expected in Q1 2021

    NBI-1065845

    Treatment Resistant Depression

    Initiate Phase II

    NBI-1065846

    Anhedonia in Depression

    Initiate Phase II

    NBI-827104

    Rare Pediatric Epilepsy: CSWS

    Continue Phase II Enrollment

    Neurological Indication

    Initiate Phase II

    NBI-921352

    Focal Onset Seizure in Adults

    Initiate Phase II

    Rare Pediatric Epilepsy: SCN8A-DEE

    Initiate Phase II

    Conference Call and Webcast Today at 4:30 PM Eastern Time

    Neurocrine Biosciences will hold a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.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 www.neurocrine.com. 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 dedicated to discovering, developing and delivering life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis*, uterine fibroids* and clinical programs in multiple therapeutic areas. For nearly three decades, Neurocrine Biosciences has specialized in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*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: milestone payments received from licenses and collaborations, milestones paid related to licenses and collaborations, non-cash collaboration revenue, acquired in-process research and development, share-based compensation expense, non-cash interest expense related to convertible debt, changes in fair value of equity security investments 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 research and development and sales, general, and administrative expenses on both a GAAP and a non-GAAP basis. The guidance regarding GAAP research and development expenses and sales, general and administrative expenses does not include estimates for expenses associated with any potential future business development activities. A reconciliation of these GAAP financial results to non-GAAP financial results is included in the attached financial information. In addition, INGREZZA net sales are presented in accordance with GAAP and as inventory-adjusted net sales, which is a non-GAAP financial measure. The difference between INGREZZA net sales and inventory-adjusted net sales reflects changes in channel inventory that are not representative of the underlying prescription demand. Management uses inventory-adjusted net sales to manage the Company's business and evaluate its performance.

    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 launch of ONGENTYS; our financial and operating performance, including our future expenses; our collaborative partnerships; expectations regarding the impact of COVID-19 on our business; expectations regarding our ability to adapt our business to the evolving COVID-19 pandemic, mitigate its impact on our business and maintain business continuity, including our ability to protect the safety and well-being of our employees, to continue to support uninterrupted supply of INGREZZA, patient in-person access to their healthcare provider, to continue our ongoing clinical trials and other development activities, and to otherwise advance our business objectives; 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 associated with the commercialization of INGREZZA and ONGENTYS; the impact of the COVID-19 pandemic on our business and the business operations of our customers; risks and uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risk and uncertainties related to any COVID-19 quarantines, shelter-in-place, social distancing and other government orders that are currently in place or that may be put in place in the future, including the impact of such orders on our and our customers' business operations and the business operations of the third parties on which we rely; risks related to the development of our product candidates; risks associated with our dependence on third parties for development and manufacturing 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 associated with our dependence on AbbVie for the commercialization of ORILISSA and ORIAHNN, as well as the continued development of elagolix; risks that clinical development activities may not be initiated or completed on time or at all, or may be delayed for regulatory, manufacturing, COVID-19 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; and other risks described in our periodic reports filed with the SEC, including without limitation our quarterly report on Form 10-Q for the quarter ended September 30, 2020. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof.

    TABLE 1



    NEUROCRINE BIOSCIENCES, INC.

    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

    (unaudited)





    Three Months Ended

    December 31,



    Twelve Months Ended

    December 31,

    (in millions, except per share data)

    2020



    2019



    2020



    2019

    Revenues:















    Product sales, net

    $

    241.3





    $

    237.9





    $

    994.1





    $

    752.9



    Collaboration revenue

    6.6





    6.2





    51.8





    35.2



    Total revenues

    247.9





    244.1





    1,045.9





    788.1



    Operating expenses:















    Cost of sales

    2.9





    2.5





    10.1





    7.4



    Research and development

    66.7





    55.3





    275.0





    200.0



    Acquired in-process research and development





    36.2





    164.5





    154.3



    Selling, general and administrative

    106.5





    101.3





    433.3





    354.1



    Total operating expenses

    176.1





    195.3





    882.9





    715.8



    Operating income

    71.8





    48.8





    163.0





    72.3



    Other (expense) income:















    Interest expense

    (7.8)





    (8.2)





    (32.8)





    (32.0)



    Unrealized loss on equity securities

    (5.5)





    (7.2)





    (17.7)





    (13.0)



    Loss on extinguishment of convertible senior notes

    (18.4)









    (18.4)







    Investment income and other, net

    1.6





    5.2





    12.6





    19.2



    Total other expense, net

    (30.1)





    (10.2)





    (56.3)





    (25.8)



    Income before (benefit from) provision for income taxes

    41.7





    38.6





    106.7





    46.5



    (Benefit from) provision for income taxes

    (306.2)





    4.6





    (300.6)





    9.5



    Net income

    $

    347.9





    $

    34.0





    $

    407.3





    $

    37.0



















    Net income per share, basic

    $

    3.72





    $

    0.37





    $

    4.38





    $

    0.40



    Net income per share, diluted

    $

    3.58





    $

    0.35





    $

    4.16





    $

    0.39



















    Weighted average common shares outstanding, basic

    93.5





    92.2





    93.1





    91.6



    Weighted average common shares outstanding, diluted

    97.2





    97.2





    97.8





    95.7



     

    TABLE 2



    NEUROCRINE BIOSCIENCES, INC.

    CONDENSED CONSOLIDATED BALANCE SHEETS

    (unaudited)



    (in millions)

    December 31,

    2020



    December 31,

    2019

    Cash, cash equivalents and debt securities available-for-sale

    $

    801.0





    $

    670.5



    Other current assets

    215.2





    160.5



    Total current assets

    1,016.2





    831.0



    Debt securities available-for-sale

    227.1





    299.7



    Right-of-use assets

    82.8





    74.3



    Equity securities

    38.2





    55.9



    Property and equipment, net

    44.6





    41.9



    Deferred tax assets

    319.4







    Restricted cash and other long-term assets

    6.4





    3.2



    Total assets

    $

    1,734.7





    $

    1,306.0











    Convertible senior notes

    $





    $

    408.8



    Other current liabilities

    186.5





    156.5



    Total current liabilities

    186.5





    565.3



    Convertible senior notes

    317.9







    Operating lease liabilities

    94.4





    86.7



    Other long-term liabilities

    9.7





    17.1



    Stockholders' equity

    1,126.2





    636.9



    Total liabilities and stockholders' equity

    $

    1,734.7





    $

    1,306.0



     

    TABLE 3



    NEUROCRINE BIOSCIENCES, INC.

    RECONCILIATION OF GAAP TO NON-GAAP FINANCIAL RESULTS

    (unaudited)





    Three Months Ended

    December 31,



    Twelve Months Ended

    December 31,

    (in millions, except per share data)

    2020



    2019



    2020



    2019

    GAAP net income

    $

    347.9





    $

    34.0





    $

    407.3





    $

    37.0



    Adjustments:















    Milestones received from licenses and collaborations A









    (30.0)





    (20.0)



    Non-cash collaboration revenue B

    (0.9)





    (0.9)





    (2.7)





    (0.9)



    Acquired in-process research and development (IPR&D) C





    36.2





    164.5





    154.3



    Milestones paid related to licenses and collaborations - R&D









    20.0





    10.0



    Share-based compensation expense - R&D

    7.3





    7.4





    33.7





    25.8



    Share-based compensation expense - SG&A

    13.7





    13.9





    66.3





    49.5



    Loss on extinguishment of convertible senior notes D

    18.4









    18.4







    Non-cash interest related to convertible senior notes

    5.2





    5.2





    21.4





    20.3



    Changes in fair value of equity security investments E

    5.5





    7.2





    17.7





    13.0



    Income tax effect related to reconciling items F

    (309.0)





    (0.8)





    (314.3)





    (5.2)



    Non-GAAP net income

    $

    88.1





    $

    102.2





    $

    402.3





    $

    283.8



















    Net income per diluted common share:















    GAAP

    $

    3.58





    $

    0.35





    $

    4.16





    $

    0.39



    Non-GAAP

    $

    0.91





    $

    1.05





    $

    4.11





    $

    2.96





    A During 2020, the Company recognized a $30.0 million event-based milestone as revenue upon FDA approval for ORIAHNN for uterine fibroids. During 2019, the Company recognized a $20.0 million event-based milestone as revenue upon FDA acceptance of the New Drug Application for elagolix for uterine fibroids.



    B The Company recognized non-cash collaboration revenue under the collaboration and license agreement entered into with Mitsubishi Tanabe Pharma Corporation in 2015.



    C The Company incurred IPR&D expenses of $164.5 million during 2020 in association with the exclusive license agreement entered into with Takeda Pharmaceutical and the collaboration and license agreement entered into with Idorsia Pharmaceuticals. During 2019, the Company incurred IPR&D expenses of $154.3 million in association with collaboration and license agreements entered into with Voyager Therapeutics and Xenon Pharmaceuticals.



    D The Company recognized a loss on extinguishment of $18.4 million related to the partial repurchase of its convertible debt in the fourth quarter of 2020.



    E The Company recognized an unrealized loss to adjust its equity security investments to fair value.



    F Estimated income tax effect of non-GAAP reconciling items are calculated using applicable statutory tax rates, taking into consideration any valuation allowance. In the fourth quarter of 2020, the Company recognized a non-cash tax benefit of approximately $296 million related to the release of substantially all of its valuation allowance against its deferred tax assets on December 31, 2020. The fourth quarter 2020 benefit associated with the valuation allowance release has been excluded from non-GAAP net income.

     

    TABLE 4



    NEUROCRINE BIOSCIENCES, INC.

    RECONCILIATION OF GAAP TO NON-GAAP EXPENSES

    (unaudited)





    Three Months Ended

    December 31,



    Twelve Months Ended

    December 31,

    (in millions)

    2020



    2019



    2020



    2019

    GAAP R&D

    $

    66.7





    $

    55.3





    $

    275.0





    $

    200.0



    Adjustments:















    Milestones paid related to licenses and collaborations









    20.0





    10.0



    Share-based compensation expense

    7.3





    7.4





    33.7





    25.8



    Non-GAAP R&D

    $

    59.4





    $

    47.9





    $

    221.3





    $

    164.2



















    GAAP SG&A

    $

    106.5





    $

    101.3





    $

    433.3





    $

    354.1



    Adjustments:















    Share-based compensation expense

    13.7





    13.9





    66.3





    49.5



    Non-GAAP SG&A

    $

    92.8





    $

    87.4





    $

    367.0





    $

    304.6



     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/neurocrine-biosciences-reports-fourth-quarter-and-full-year-2020-financial-results-301222645.html

    SOURCE Neurocrine Biosciences, Inc.

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  3. CAMBRIDGE, Mass., Feb. 02, 2021 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ:VYGR) today announced that Neurocrine Biosciences, Inc. (NASDAQ:NBIX) provided notice of termination of the Parkinson's disease portion of the collaboration agreement, effective August 2, 2021. The Friedreich's ataxia program and two discovery programs that are also part of the agreement are not impacted and remain under active collaboration.

    Voyager's understanding is that Neurocrine's decision to terminate the NBIb-1817 (VY-AADC) program was based on a portfolio review and prioritization of its current pipeline assets.   Voyager plans to support Neurocrine, the IND holder and sponsor of the RESTORE-1 Phase 2 clinical trial, on any ongoing matters related…

    CAMBRIDGE, Mass., Feb. 02, 2021 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ:VYGR) today announced that Neurocrine Biosciences, Inc. (NASDAQ:NBIX) provided notice of termination of the Parkinson's disease portion of the collaboration agreement, effective August 2, 2021. The Friedreich's ataxia program and two discovery programs that are also part of the agreement are not impacted and remain under active collaboration.

    Voyager's understanding is that Neurocrine's decision to terminate the NBIb-1817 (VY-AADC) program was based on a portfolio review and prioritization of its current pipeline assets.   Voyager plans to support Neurocrine, the IND holder and sponsor of the RESTORE-1 Phase 2 clinical trial, on any ongoing matters related to additional imaging and clinical assessments requested by the Data Safety & Monitoring Board (DSMB) and other information that may be requested by the U.S. Food and Drug Administration (FDA).  

    In December 2020, Voyager announced that the FDA had notified Neurocrine that it had placed a clinical hold on the RESTORE-1 clinical trial of NBIb-1817 (VY-AADC). The FDA notification followed a request by the study's independent DSMB for a pause in dosing pending the receipt of information about magnetic resonance imaging (MRI) abnormalities observed in trial participants. In January 2021, the FDA informed Neurocrine of the information required to provide a complete response to the FDA in connection with the clinical hold. Information required by the FDA includes an assessment of how the investigational product may have given rise to the adverse findings, a mitigation plan to manage the adverse findings, and supportive data to justify that a favorable benefit/risk profile remains for the product.

    Voyager is evaluating the complete financial impact of the termination and the future of the Parkinson's program and expects to provide a subsequent update.

    About Parkinson's Disease and NBIb-1817 (VY-AADC)

    Parkinson's disease is a chronic, progressive, and debilitating neurodegenerative disease that affects approximately one million people in the U.S. and ten million people worldwide. It is characterized by a loss of dopamine and neuronal degeneration with a concomitant loss of the aromatic L-amino acid decarboxylase (AADC) enzyme required to synthesize dopamine in the brain, leading to associated impairment in motor, neuropsychiatric, and autonomic functions. Dopamine is a chemical "messenger" that is produced in the brain and is involved in the control of movement. It is made when AADC converts the chemical levodopa to dopamine. As Parkinson's disease progresses, there is less AADC enzyme in parts of the brain where levodopa is converted to dopamine.

    NBIb-1817 (VY-AADC) is an investigational recombinant adeno-associated viral (AAV) serotype 2 vector encoding the gene for human AADC that is designed to help produce the AADC enzyme in brain cells where it can convert levodopa to dopamine. NBIb-1817 (VY-AADC) is administered into the brain using intraoperative monitoring with magnetic resonance imaging (MRI)-facilitated targeted delivery.

    About Voyager Therapeutics

    Voyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyager's wholly-owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinson's disease, Huntington's disease, Friedreich's ataxia, and other severe neurological diseases. For more information on Voyager Therapeutics, please visit the company's website at www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and LinkedIn.

    Voyager Therapeutics® is a registered trademark of Voyager Therapeutics, Inc.

    Voyager Therapeutics Forward-Looking Statements

    This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "might," "will," "would," "should," "expect," "plan," "anticipate," "believe," "estimate," "undoubtedly," "project," "intend," "future," "potential," or "continue," and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding the progress, activities, goals and reporting of results of its clinical trials, the potential benefits and future operation of its collaboration with Neurocrine and the activities thereunder, Voyager's ability to perform its obligations under the collaboration agreement with Neurocrine, the ability of Neurocrine and Voyager to gather additional information to further characterize the safety profile of NBIb-1817 (VY-AADC) and to work with the FDA to determine the next steps for the RESTORE-1 clinical trial, the regulatory pathway of, and the timing or likelihood of its regulatory filings and approvals for, any of Voyager's product candidates, and its anticipated financial results, including the financial impact of the termination of the NBIb-1817 (VY-AADC) program, are forward looking.  

    All forward-looking statements are based on estimates and assumptions by Voyager's management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, those related to the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the expectations for regulatory communications, submissions and approvals; the continued development of the gene therapy platform and its TRACER system; Voyager's scientific approach and general development progress; the sufficiency of its cash resources; the availability or commercial potential of Voyager's product candidates; and the ability of Neurocrine and Voyager to complete their evaluation and to meet the information requests of, and to resolve questions raised by, the FDA required to bring an end to the clinical hold on the RESTORE-1 clinical trial. These statements are also subject to a number of material risks and uncertainties that are described in Voyager's Annual Report on Form 10-K filed with the Securities and Exchange Commission, as updated by its subsequent filings with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

    Contact Information

    Voyager Therapeutics

    Investors:        

    Media:        

    Sheryl Seapy

    W2Opure

    949-903-4750



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  4. SAN DIEGO, Jan. 20, 2021 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) announced today that it will report fourth quarter and year-end 2020 financial results after the Nasdaq market closes on Thursday, Feb. 4, 2021. Neurocrine will then host a conference call and webcast to discuss its financial results and provide a company update that day at 1:30 p.m. Pacific Time (4:30 p.m. Eastern 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's website under Investors at www.neurocrine.com. 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 dedicated to discovering, developing and delivering life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis*, uterine fibroids* and clinical programs in multiple therapeutic areas. For nearly three decades, Neurocrine Biosciences has specialized in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn(*in collaboration with AbbVie)

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    SOURCE Neurocrine Biosciences, Inc.

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  5. SAN DIEGO, Jan. 8, 2021 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today provided an update on its business performance, including preliminary net product and inventory adjusted sales results of INGREZZA® (valbenazine) for 2020, and key commercial and clinical development milestones for 2021. Kevin Gorman, Chief Executive Officer of Neurocrine Biosciences, will discuss these updates as part of a webcast presentation at the 39th Annual J.P. Morgan Healthcare Conference to be held virtually on Monday, January 11 at 2:00 p.m. Eastern Time, followed by a Question and Answer session at approximately 2:20 p.m. Eastern Time.

    Preliminary Fourth Quarter and Full-Year 2020 INGREZZA® (valbenazine) Net Product Sales and Inventory Adjusted Net Product Sales (Unaudited)

    Based on preliminary unaudited financial information, the Company expects INGREZZA net product sales for the three months and full-year ended December 31, 2020 to be approximately $240 million and $993 million respectively. Preliminary analysis of INGREZZA net product sales suggests:

    - INGREZZA inventory adjusted net product sales for the fourth quarter were approximately $258 million reflecting an $18 million channel inventory decrease in Q4

    - New prescriptions and refills increased in the fourth quarter of 2020 vs. the third quarter of 2020

    - Full-year 2020 total INGREZZA prescriptions grew 32% to approximately 175,700 versus 2019 total prescriptions of approximately 132,700 

    "I am very proud of our team's resilience and adaptability to bring INGREZZA to patients living with tardive dyskinesia this past year and we remain encouraged by the continued strength in persistence and refill rates, which is a testament to the many benefits of INGREZZA. We also continue to focus on healthcare provider educational initiatives, patient outreach programs and investing in telemedicine capabilities to improve diagnosis and treatment rates for the estimated 80% of patients with tardive dyskinesia who have not yet been diagnosed," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "Adding to our movement disorder portfolio, we launched our second commercial treatment, ONGENTYS, and continue to make great progress in our development programs with plans to initiate seven mid-to-late stage clinical studies in 2021 focused on neurological, endocrine and psychiatric disorders."

    2021 Expected Milestones and Key Activities

    Program

    Indication

    2021 Milestones / Key Activities

    Valbenazine

    Chorea in Huntington Disease

    Phase III Top-Line Data Expected in Q4 2021

    Neurological Indication

    Initiate Phase III

    Psychiatric Indication

    Initiate Phase II

    Crinecerfont

    Congenital Adrenal Hyperplasia (Adult)

    Continue Phase III Enrollment

    Congenital Adrenal Hyperplasia (Pediatric)

    Initiate Phase III

    NBI-1065844

    Negative Symptoms of Schizophrenia

    Phase II Top-Line Data Expected in

    1st Half of 2021

    NBI-1065845

    Treatment Resistant Depression

    Initiate Phase II

    NBI-1065846

    Anhedonia in Depression

    Initiate Phase II

    NBI-827104

    Rare Pediatric Epilepsy:

    Epileptic Encephalopathy with Continuous Spike and Wave During Sleep

    Continue Phase II Enrollment

    Neurological Indication

    Initiate Phase II

    NBI-921352

    Focal Onset Seizure in Adults

    Initiate Phase II

    Rare Pediatric Epilepsy: SCN8A-DEE

    Ongoing Dialogue with FDA

    NBIb-1817

    Gene Therapy for Parkinson's Disease

    Determine Regulatory Path with FDA

    About Tardive Dyskinesia (TD)

    Tardive dyskinesia (TD) is a movement disorder that is characterized by uncontrollable, abnormal and repetitive movements of the face, torso and/or other body parts, which may be disruptive and negatively impact patients. The condition is caused by prolonged use of treatments that block dopamine receptors in the brain, such as antipsychotics commonly prescribed to treat mental illnesses such as schizophrenia, bipolar disorder and depression, and certain anti-nausea medications. In patients with TD, these treatments are thought to result in irregular dopamine signaling in a region of the brain that controls movement. The symptoms of TD can be severe and are often persistent and irreversible. TD is estimated to affect at least 500,000 people in the U.S.

    About INGREZZA® (valbenazine) Capsules

    INGREZZA, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is the first FDA-approved product indicated for the treatment of adults with tardive dyskinesia, a condition associated with uncontrollable, abnormal and repetitive movements of the face, torso and/or other body parts.

    INGREZZA is thought to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function, helping to regulate nerve signaling in adults with tardive dyskinesia. VMAT2 is a protein in the brain that packages neurotransmitters, such as dopamine, for transport and release in presynaptic neurons. INGREZZA, developed in Neurocrine Biosciences's laboratories, is novel in that it selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic, or muscarinic receptors. Additionally, INGREZZA can be taken for the treatment of tardive dyskinesia as one capsule, once-daily, together with psychiatric medications such as antipsychotics or antidepressants.

    Important Information

    Approved Use

    INGREZZA® (valbenazine) capsules is a prescription medicine used to treat adults with movements in the face, tongue, or other body parts that cannot be controlled (tardive dyskinesia).

    It is not known if INGREZZA is safe and effective in children.

    Important Safety Information

    Do not take INGREZZA if you are allergic to valbenazine, or any of the ingredients in INGREZZA.

    INGREZZA may cause serious side effects, including:

    • Sleepiness (somnolence). Do not drive, operate heavy machinery, or do other dangerous activities until you know how INGREZZA affects you.
    • Heart rhythm problems (QT prolongation). INGREZZA may cause a heart problem known as QT prolongation.
    • Symptoms of QT prolongation may include: fast, slow, or irregular heartbeat, shortness of breath, dizziness or fainting.
    • Parkinson-like symptoms. Symptoms include: shaking, body stiffness, trouble moving or walking, or keeping your balance.

    Tell your healthcare provider right away if you have a change in your heartbeat (a fast or irregular heartbeat), or if you faint.

    Before taking INGREZZA, tell your healthcare provider about all of your medical conditions including if you: have liver or heart 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.

    The most common side effect of INGREZZA is sleepiness (somnolence). Other side effects include changes in balance (balance problems, dizziness) or an increased risk of falls, headache, feelings of restlessness, dry mouth, constipation, and blurred vision.

    These are not all of the possible side effects of INGREZZA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

    Please see INGREZZA full Product Information.

    About Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company dedicated to discovering, developing and delivering life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis*, uterine fibroids* and clinical programs in multiple therapeutic areas. For nearly three decades, Neurocrine Biosciences has specialized in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*in collaboration with AbbVie)

    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: our preliminary unaudited financial information; 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, our collaborative partnerships; expectations regarding the impact of COVID-19 on our business, including patient and healthcare provider access to INGREZZA, our ability to continue our ongoing clinical trials and other development activities, and to otherwise advance our business objectives; and the timing of 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: risks and uncertainties associated with items that may be identified during the financial statement closing process that cause adjustments to the estimates included in this press release; our future financial and operating performance; risks associated with the commercialization of INGREZZA and ONGENTYS; the impact of the COVID-19 pandemic on our business and the business operations of our customers; risks and uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risk and uncertainties related to any COVID-19 quarantines, shelter-in-place, social distancing and other government orders that are currently in place or that may be put in place in the future, including the impact of such orders on our business operations and the business operations of the third parties on which we rely; risks related to the development of our product candidates; risks associated with our dependence on third parties for development and manufacturing activities related to INGREZZA and our 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 associated with our dependence on AbbVie for the commercialization of ORILISSA and ORIAHNN, as well as the continued development of elagolix; risks associated with our dependence on BIAL for manufacturing activities for ONGENTYS, and our ability to manage BIAL; risks that clinical development activities may not be completed on time or at all, or may be delayed for regulatory, manufacturing, COVID-19 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; and other risks described in our periodic reports filed with the SEC, including without limitation our quarterly report on Form 10-Q for the quarter ended September 30, 2020. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof.

    This press release refers to preliminary unaudited net sales in certain non-GAAP financial measures. These non-GAAP financial measures should not be considered replacements for, and should be read together with, the comparable GAAP financial measures, which are included in this press release.

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    SOURCE Neurocrine Biosciences, Inc.

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  6. SAN DIEGO, Jan. 4, 2021 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) will present at the 39th Annual J.P. Morgan Healthcare Conference at 2:00 p.m. Eastern Time on Monday Jan. 11, 2021. Kevin Gorman, Chief Executive Officer, will present at the conference.

    The live presentation will be webcast and may be accessed on the Company's website under Investors at www.neurocrine.com. A replay of the presentation will be available on the website approximately one hour after the conclusion of the events and will be archived for approximately one month.

    About Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company dedicated to discovering, developing and delivering life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis*, uterine fibroids* and clinical programs in multiple therapeutic areas. For nearly three decades, Neurocrine Biosciences has specialized in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn(*in collaboration with AbbVie)

    ###

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    SOURCE Neurocrine Biosciences, Inc.

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  7. CAMBRIDGE, Mass., Dec. 22, 2020 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ:VYGR) today announced that the U.S. Food and Drug Administration (FDA) has notified Neurocrine Biosciences (NASDAQ:NBIX) that it has placed a clinical hold on the RESTORE-1 clinical trial of NBIb-1817 (VY-AADC). As previously announced, trial sites participating in RESTORE-1 had not been screening, enrolling or dosing patients as a result of the COVID-19 pandemic and more recently, as a result of the independent Data Safety Monitoring Board (DSMB)'s request to pause dosing pending its review of additional data. The DSMB has requested additional patient level data from the trial and now plans to review these data in early 2021. The clinical hold follows the…

    CAMBRIDGE, Mass., Dec. 22, 2020 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ:VYGR) today announced that the U.S. Food and Drug Administration (FDA) has notified Neurocrine Biosciences (NASDAQ:NBIX) that it has placed a clinical hold on the RESTORE-1 clinical trial of NBIb-1817 (VY-AADC). As previously announced, trial sites participating in RESTORE-1 had not been screening, enrolling or dosing patients as a result of the COVID-19 pandemic and more recently, as a result of the independent Data Safety Monitoring Board (DSMB)'s request to pause dosing pending its review of additional data. The DSMB has requested additional patient level data from the trial and now plans to review these data in early 2021. The clinical hold follows the submission by Neurocrine Biosciences of an IND Safety Report related to the observation of MRI abnormalities in some RESTORE-1 study participants. The clinical implications of this observation are currently unknown and are being evaluated.

    RESTORE-1 is a Phase 2 clinical trial of NBIb-1817 (VY-AADC), an intracerebral AAV-based investigational gene therapy, in development for the treatment of Parkinson's disease. The RESTORE-1 DSMB has been informed of the clinical hold, as have the study investigators and central and local ethics committees. Neurocrine Biosciences and Voyager will work closely with the FDA and the DSMB to determine the next steps for the RESTORE-1 clinical trial.

    About Parkinson's Disease and NBIb-1817 (VY-AADC)

    Parkinson's disease is a chronic, progressive, and debilitating neurodegenerative disease that affects approximately one million people in the U.S. and ten million people worldwide. It is characterized by a loss of dopamine and neuronal degeneration with a concomitant loss of the aromatic L-amino acid decarboxylase (AADC) enzyme required to synthesize dopamine in the brain, leading to associated impairment in motor, neuropsychiatric, and autonomic functions. Dopamine is a chemical "messenger" that is produced in the brain and is involved in the control of movement. It is made when AADC converts the chemical levodopa to dopamine. As Parkinson's disease progresses, there is less AADC enzyme in parts of the brain where levodopa is converted to dopamine.

    NBIb-1817 (VY-AADC) is an investigational recombinant adeno-associated viral (AAV) serotype 2 vector encoding the gene for human AADC that is designed to help produce the AADC enzyme in brain cells where it can convert levodopa to dopamine. NBIb-1817 (VY-AADC) is administered into the brain using intraoperative monitoring with magnetic resonance imaging (MRI)-facilitated targeted delivery.

    About Voyager Therapeutics

    Voyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyager's wholly-owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinson's disease, Huntington's disease, Friedreich's ataxia, and other severe neurological diseases. For more information on Voyager Therapeutics, please visit the company's website at www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and LinkedIn.

    Voyager Therapeutics® is a registered trademark of Voyager Therapeutics, Inc.

    Voyager Therapeutics Forward-Looking Statements

    This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "might," "will," "would," "should," "expect," "plan," "anticipate," "believe," "estimate," "undoubtedly," "project," "intend," "future," "potential," or "continue," and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding the ability of Neurocrine and Voyager to gather additional information to further characterize the safety profile of NBIb-1817 (VY-AADC) and to work with the FDA to determine the next steps for the RESTORE-1 clinical trial.

    All forward-looking statements are based on estimates and assumptions by Voyager's management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the ability of Neurocrine and Voyager to complete their evaluation and to meet the information requests of, and to resolve questions raised by, the FDA required to bring an end to the clinical hold on the RESTORE-1 clinical trial. These statements are also subject to a number of material risks and uncertainties that are described in Voyager's Annual Report on Form 10-K filed with the Securities and Exchange Commission, as updated by its subsequent filings with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

    Contact Information

    Voyager Therapeutics

    Investors:        

    Media:        

    Sheryl Seapy

    W2Opure

    949-903-4750

     



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  8. SAN DIEGO, Nov. 19, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced that Neurocrine Biosciences management will present at the following investor conferences:

    • Matt Abernethy, Chief Financial Officer, and Eiry Roberts, Chief Medical Officer, will present at the Piper Sandler 32nd Annual Virtual Healthcare Conference at 10:00 a.m. Eastern Time on Monday, November 23, 2020.



    • Kevin Gorman, Chief Executive Officer, and Matt Abernethy, Chief Financial Officer, will present at the Evercore ISI 3rd Annual HealthCONx Conference at 1:50 p.m. Eastern Time on Tuesday, December 1, 2020.

    The live presentations will be webcast and may be accessed on the Company's website under Investors at www.neurocrine.com. A replay of the presentations will be available on the website approximately one hour after the conclusion of the events and will be archived for approximately one month.

    About Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company dedicated to discovering, developing and delivering life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis*, uterine fibroids* and clinical programs in multiple therapeutic areas. For nearly three decades, Neurocrine Biosciences has specialized in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn(*in collaboration with AbbVie)

     

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  9. SAN DIEGO, Nov. 19, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced that the Company has entered into separate, privately negotiated transactions (the "Agreements") with certain holders of its existing 2.25% Convertible Senior Notes due 2024 (the "2024 Notes") to repurchase approximately $83 million aggregate principal amount of the 2024 Notes for an aggregate repurchase price of an amount of cash estimated to be the sum of (i) approximately $110 million based on the Company's November 18, 2020 closing stock price of $86.91 per share, (ii) an amount based in part on the daily volume-weighted average prices per share of the Company's common stock during a five-trading day pricing period following execution of the…

    SAN DIEGO, Nov. 19, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced that the Company has entered into separate, privately negotiated transactions (the "Agreements") with certain holders of its existing 2.25% Convertible Senior Notes due 2024 (the "2024 Notes") to repurchase approximately $83 million aggregate principal amount of the 2024 Notes for an aggregate repurchase price of an amount of cash estimated to be the sum of (i) approximately $110 million based on the Company's November 18, 2020 closing stock price of $86.91 per share, (ii) an amount based in part on the daily volume-weighted average prices per share of the Company's common stock during a five-trading day pricing period following execution of the Agreements and (iii) accrued and unpaid interest. The 2024 Notes repurchases are expected to close on December 2, 2020, subject to customary closing conditions. Such repurchases of the 2024 Notes could affect the market price of the Company's common stock. 

    This press release does not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any offer or sale of these securities in any state or jurisdiction in which the offer, solicitation, or sale would be unlawful prior to the registration or qualification thereof under the securities laws of any such state or jurisdiction.

    About Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company dedicated to discovering, developing and delivering life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The Company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis*, uterine fibroids* and clinical programs in multiple therapeutic areas. For nearly three decades, Neurocrine Biosciences has specialized in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*in collaboration with AbbVie)

    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 amount of 2024 Notes to be repurchased, the timing of completion of the repurchases, and the impact of the repurchases on the market price of the Company's common stock.  Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are: changes in the price of the Company's common stock; changes in the convertible note and other capital markets; and other risks described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's quarterly report on Form 10-Q for the quarter ended September 30, 2020. The Company disclaims any obligation to update the statements contained in this press release after the date hereof.

     

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  10. SAN DIEGO, Nov. 9, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced its financial results for the third quarter ended September 30, 2020 and provided revised full-year 2020 financial expense guidance.

    "Our focus remains on helping as many patients with tardive dyskinesia as possible and we are adapting to the challenges posed by the pandemic, including the slow recovery of in-person visits with psychiatrists. We are encouraged by recent trends and remain confident in the intermediate and long-term opportunity of INGREZZA to help many more patients with tardive dyskinesia," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "We are pleased to expand our reach with neurologists with the addition of ONGENTYS to our movement disorder portfolio and continue to advance our development programs focused on neurological, endocrine and psychiatric disorders."

    Financial Highlights



    Three Months Ended

    September 30,



    Nine Months Ended

    September 30,

    (unaudited, in millions, except per share data)

    2020



    2019



    2020



    2019

    Revenues:















    Product sales, net

    $

    254.1





    $

    198.1





    $

    752.8





    $

    515.0



    Collaboration revenue

    4.4





    24.0





    45.2





    29.0



    Total revenues

    $

    258.5





    $

    222.1





    $

    798.0





    $

    544.0



















    GAAP Research and Development (R&D)

    $

    69.1





    $

    45.3





    $

    208.3





    $

    144.7



    Non-GAAP R&D

    $

    60.3





    $

    38.3





    $

    161.9





    $

    116.3



















    GAAP Selling, General and Administrative (SG&A)

    $

    112.5





    $

    84.5





    $

    326.8





    $

    252.8



    Non-GAAP SG&A

    $

    94.6





    $

    71.2





    $

    274.2





    $

    217.2



















    GAAP net (loss) income

    $

    (57.6)





    $

    53.8





    $

    59.4





    $

    3.0



    GAAP net (loss) income per share – diluted

    $

    (0.62)





    $

    0.56





    $

    0.61





    $

    0.03



















    Non-GAAP net income

    $

    96.0





    $

    86.7





    $

    314.2





    $

    181.6



    Non-GAAP net income per share – diluted

    $

    0.97





    $

    0.90





    $

    3.21





    $

    1.91



















    (unaudited, in millions)









    September 30,

    2020



    December 31,

    2019

    Total cash and cash equivalents and debt securities available-for-sale

    $

    1,126.1





    $

    970.2



    Net Product Sales Highlights:

    • INGREZZA net product sales for the third quarter of 2020 were $254 million, representing a year-over-year increase of 28%. Inventory adjusted net sales for the third quarter of 2020 were approximately $248 million.
    • New prescriptions increased slightly in the third quarter of 2020 vs. the second quarter of 2020.
    • Refill and persistency rates for existing INGREZZA patients in the third quarter 2020 moderated slightly vs. the second quarter of 2020 and remained at the higher end of historical averages.
    • ONGENTYS launched in the United States in late September 2020 and net product sales for the third quarter of 2020 were approximately $0.1 million.

    Financial Highlights:

    • Third quarter 2020 GAAP net loss and loss per share were approximately $58 million and $0.62, respectively, compared with net income and diluted earnings per share of approximately $54 million and $0.56, respectively, in the third quarter of 2019, primarily driven by higher In-Process Research and Development (IPR&D) costs and operating expenses, offset by higher INGREZZA sales.
    • Third quarter 2020 non-GAAP net income and diluted earnings per share were approximately $96 million and $0.97, respectively, compared with approximately $87 million and $0.90, respectively, in the third quarter of 2019 driven by higher INGREZZA sales.
    • Research and Development (R&D) expense increased in the third quarter of 2020 versus the third quarter of 2019, primarily due to increased investment across our expanded pipeline programs and higher headcount costs.
    • Selling, General and Administrative (SG&A) expense increased in the third quarter of 2020 versus the third quarter of 2019, primarily due to increased investment in marketing initiatives and higher headcount costs.
    • At September 30, 2020, the Company had cash, cash equivalents and debt securities available-for-sale of $1.1 billion.

    A reconciliation of GAAP to non-GAAP quarterly financial results can be found in Table 3 at the end of this earnings release.

    Recent Events

    • In September 2020, the Company launched ONGENTYS® (opicapone), the first and only once-daily COMT inhibitor, as an adjunctive treatment to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes - periods of time when motor symptoms such as tremor, slowed movement and difficulty walking occur. ONGENTYS also increases "on" time without troublesome dyskinesia, the time when the motor symptoms of a patient with Parkinson's disease are better controlled.
    • In October 2020, the U.S. Food and Drug Administration (FDA) provided feedback on an Investigational New Drug (IND) application submitted by Neurocrine Biosciences in support of a Phase II clinical trial of NBI-921352 in pediatric SCN8A developmental and epileptic encephalopathy syndrome patients. As part of its review of the IND, the FDA is requesting additional non-clinical data to support dose justification in this pediatric study. Together with Xenon, the Company will engage with the FDA to address the feedback received with the goal of initiating a Phase II clinical trial in 2021. In parallel, the Company is advancing clinical plans to develop NBI-921352 for the treatment of adult focal epilepsy.
    • In November 2020, the Company announced the initiation of a Phase II study of NBI-827104 (formerly ACT-709478) in CSWS, a rare pediatric epilepsy. NBI-827104 was licensed from Idorsia and is a potent, selective, orally-active and brain penetrating T-type calcium channel blocker.
    • In November 2020, the Data Safety Monitoring Board (DSMB) for the RESTORE-1 Phase II clinical trial reviewed patient imaging data from the ongoing trial. The DSMB requested additional data and recommended the Company pause dosing of subjects in RESTORE-1 until the DSMB can review these additional data, which is expected by year-end. The DSMB informed the Company and Voyager Therapeutics that they could continue screening patients for potential enrollment into the trial. However, as previously announced, trial sites participating in RESTORE-1 are not currently screening and enrolling patients as a result of the COVID-19 pandemic. In response to the DSMB's recommendation, the Company and Voyager Therapeutics have decided to delay the planned resumption this quarter of patient screening in the RESTORE-1 trial until the DSMB is able to complete its evaluation. The Company is preparing an expedited safety report that will be submitted to the FDA within the 15-day reporting window.

    Full-Year 2020  Expense Guidance



    Range

    (in millions)

    Low



    High

    Combined GAAP R&D and SG&A expenses

    $

    880





    $

    900



    Combined Non-GAAP R&D and SG&A expenses

    $

    590





    $

    610



     

    • Previously, the Company expected combined GAAP R&D and SG&A expenses in the range of $850 million to $900 million and combined non-GAAP R&D and SG&A expenses in the range of $570 million to $610 million.

    Conference Call and Webcast Today at 4:30 PM Eastern Time

    Neurocrine Biosciences will hold a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). Participants can access the live conference call by dialing 877-830-2596 (US) or 785-424-1744 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine Biosciences' website under Investors at www.neurocrine.com. 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 dedicated to discovering, developing and delivering life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis*, uterine fibroids* and clinical programs in multiple therapeutic areas. For nearly three decades, Neurocrine Biosciences has specialized in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*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 certain non-GAAP financial measures. When preparing these supplemental 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, the non-GAAP measures exclude: milestones received from licenses and collaborations, milestones paid related to licenses and collaborations, non-cash collaboration revenue, acquired in-process research and development, share-based compensation expense, non-cash interest expense related to convertible debt, changes in fair value of equity security investments and certain adjustments to income tax expense. These non-GAAP 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 research and development and sales, general, and administrative expenses on both a GAAP and a non-GAAP basis. The guidance regarding GAAP research and development expenses and sales, general and administrative expenses does not include estimates for expenses associated with any potential future business development activities. A reconciliation of the 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 launch of ONGENTYS; our financial and operating performance, including our future expenses; our collaborative partnerships; expectations regarding the impact of COVID-19 on our business; expectations regarding our ability to adapt our business to the evolving COVID-19 pandemic, mitigate its impact on our business and maintain business continuity, including our ability to protect the safety and well-being of our employees, to continue to support uninterrupted supply of INGREZZA, including patient and healthcare provider access to INGREZZA, to continue our ongoing clinical trials and other development activities, and to otherwise advance our business objectives; and the timing of 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 associated with the commercialization of INGREZZA and ONGENTYS; the impact of the COVID-19 pandemic on our business and the business operations of our customers; risks and uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risk and uncertainties related to any COVID-19 quarantines, shelter-in-place, social distancing and other government orders that are currently in place or that may be put in place in the future, including the impact of such orders on our business operations and the business operations of the third parties on which we rely; risks related to the development of our product candidates; risks associated with our dependence on third parties for development and manufacturing activities related to INGREZZA and our 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 associated with our dependence on AbbVie for the commercialization of ORILISSA and ORIAHNN, as well as the continued development of elagolix; risks associated with our dependence on BIAL for manufacturing activities for ONGENTYS, and our ability to manage BIAL; risks that clinical development activities may not be completed on time or at all, or may be delayed for regulatory, manufacturing, COVID-19 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; and other risks described in our periodic reports filed with the SEC, including without limitation our quarterly report on Form 10-Q for the quarter ended September 30, 2020. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof.

    This press release refers to certain non-GAAP financial measures. These non-GAAP financial measures should not be considered replacements for, and should be read together with, the most comparable GAAP financial measures. Reconciliations of non-GAAP financial results to the most directly comparable GAAP financial results are included at the end of this press release, which has been filed with the SEC in a Current Report on Form-8-K dated as of event date herewith. In addition, Neurocrine provides guidance regarding combined research and development and sales, general and administrative expenses on both a GAAP and non-GAAP basis.

     

    TABLE 1



     

    NEUROCRINE BIOSCIENCES, INC.

    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

    (unaudited)





    Three Months Ended

    September 30,



    Nine Months Ended

    September 30,

    (in millions, except per share data)

    2020



    2019



    2020



    2019

    Revenues:















    Product sales, net

    $

    254.1





    $

    198.1





    $

    752.8





    $

    515.0



    Collaboration revenue

    4.4





    24.0





    45.2





    29.0



    Total revenues

    258.5





    222.1





    798.0





    544.0



    Operating expenses:















    Cost of sales

    2.7





    2.2





    7.2





    4.9



    Research and development

    69.1





    45.3





    208.3





    144.7



    Acquired in-process research and development

    118.5









    164.5





    118.1



    Selling, general and administrative

    112.5





    84.5





    326.8





    252.8



    Total operating expenses

    302.8





    132.0





    706.8





    520.5



    Operating (loss) income

    (44.3)





    90.1





    91.2





    23.5



    Other (expense) income:















    Interest expense

    (8.5)





    (8.0)





    (25.0)





    (23.8)



    Unrealized loss on equity securities

    (7.0)





    (28.5)





    (12.2)





    (5.8)



    Investment income and other, net

    2.7





    4.8





    11.0





    14.0



    Total other expense, net

    (12.8)





    (31.7)





    (26.2)





    (15.6)



    (Loss) income before provision for income taxes

    (57.1)





    58.4





    65.0





    7.9



    Provision for income taxes

    0.5





    4.6





    5.6





    4.9



    Net (loss) income

    $

    (57.6)





    $

    53.8





    $

    59.4





    $

    3.0



















    Net (loss) income per share, basic

    $

    (0.62)





    $

    0.59





    $

    0.64





    $

    0.03



    Net (loss) income per share, diluted

    $

    (0.62)





    $

    0.56





    $

    0.61





    $

    0.03



















    Weighted average common shares outstanding, basic

    93.3





    91.9





    93.0





    91.4



    Weighted average common shares outstanding, diluted

    93.3





    96.1





    98.0





    95.2



     

    TABLE 2



     

    NEUROCRINE BIOSCIENCES, INC.

    CONDENSED CONSOLIDATED BALANCE SHEETS

    (unaudited)



    (in millions)

    September 30,

    2020



    December 31,

    2019

    Cash and cash equivalents and debt securities available-for-sale

    $

    944.7





    $

    670.5



    Other current assets

    212.2





    160.5



    Total current assets

    1,156.9





    831.0



    Debt securities available-for-sale

    181.4





    299.7



    Right-of-use assets

    71.0





    74.3



    Equity securities

    43.7





    55.9



    Property and equipment, net

    43.0





    41.9



    Restricted cash and other long-term assets

    6.6





    3.2



    Total assets

    $

    1,502.6





    $

    1,306.0











    Convertible senior notes

    $

    425.0





    $

    408.8



    Other current liabilities

    186.0





    156.5



    Total current liabilities

    611.0





    565.3



    Operating lease liabilities

    83.0





    86.7



    Other long-term liabilities

    4.3





    17.1



    Stockholders' equity

    804.3





    636.9



    Total liabilities and stockholders' equity

    $

    1,502.6





    $

    1,306.0



     

    TABLE 3



     

    NEUROCRINE BIOSCIENCES, INC.

    RECONCILIATION OF GAAP TO NON-GAAP FINANCIAL RESULTS

    (unaudited)





    Three Months Ended

    September 30,



    Nine Months Ended

    September 30,

    (in millions, except per share data)

    2020



    2019



    2020



    2019

    GAAP net (loss) income

    $

    (57.6)





    $

    53.8





    $

    59.4





    $

    3.0



    Adjustments:















    Milestones received from licenses and collaborations A





    (20.0)





    (30.0)





    (20.0)



    Non-cash collaboration revenue B

    (0.5)









    (1.8)







    Acquired in-process research and development (IPR&D) C

    118.5









    164.5





    118.1



    Milestones paid related to licenses and collaborations - R&D









    20.0





    10.0



    Share-based compensation expense - R&D

    8.8





    7.0





    26.4





    18.4



    Share-based compensation expense - SG&A

    17.9





    13.3





    52.6





    35.6



    Non-cash interest related to convertible debt

    5.5





    5.1





    16.2





    15.1



    Changes in fair value of equity security investments D

    7.0





    28.5





    12.2





    5.8



    Income tax effect related to reconciling items E

    (3.6)





    (1.0)





    (5.3)





    (4.4)



    Non-GAAP net income

    $

    96.0





    $

    86.7





    $

    314.2





    $

    181.6



















    Net (loss) income per diluted common share:















    GAAP

    $

    (0.62)





    $

    0.56





    $

    0.61





    $

    0.03



    Non-GAAP F

    $

    0.97





    $

    0.90





    $

    3.21





    $

    1.91





    A During the nine months ended September 30, 2020, the Company recognized a $30.0 million event-based milestone as revenue upon FDA approval for ORIAHNN for uterine fibroids. During the nine months ended September 30, 2019, the Company recognized a $20.0 million event-based milestone as revenue upon FDA acceptance of the New Drug Application for elagolix for uterine fibroids.

    B During the nine months ended September 30, 2020, the Company recognized non-cash collaboration revenue from Mitsubishi Tanabe Pharma Corporation under the collaboration and license agreement entered into in 2015.

    C The Company incurred IPR&D expenses of $118.5 million and $164.5 million during the three and nine months ended September 30, 2020, respectively, in association with the exclusive license agreement entered into with Takeda and the collaboration and license agreement entered into with Idorsia, and $118.1 million during the nine months ended September 30, 2019, in association with the collaboration and license agreement entered into with Voyager Therapeutics in 2019.

    D The Company recognized an unrealized loss of $7.0 million and $28.5 million for the three months ended September 30, 2020 and 2019, respectively, and $12.2 million and $5.8 million for the nine months ended September 30, 2020 and 2019, respectively, to adjust its equity security investments to fair value.

    E Estimated income tax effect of non-GAAP reconciling items are calculated using applicable statutory tax rates, taking into consideration any valuation allowance.

    F Non-GAAP net income per diluted common share for the three months ended September 30, 2020, reflects diluted shares of 98.9 million, which were calculated in accordance with the guidance on earnings per share in ASC 260.

     

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  11. SAN DIEGO, Nov. 9, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced that Neurocrine Biosciences management will present at the following investor conferences:

    • Kevin Gorman, Chief Executive Officer, and Kyle Gano, Chief Business Development and Strategy Officer, will present at the Stifel 2020 Virtual Healthcare Conference at 11:20 a.m. Eastern Time on Monday, November 16, 2020.
    • Kevin Gorman, Chief Executive Officer, and Matt Abernethy, Chief Financial Officer, will present at the Jefferies Virtual London Healthcare Conference at 2:40 p.m. Greenwich Mean Time (9:40 a.m. Eastern Time) on Tuesday, November 17, 2020.

    The live presentations will be webcast and may be accessed on the Company's website under Investors at www.neurocrine.com. A replay of the presentations will be available on the website approximately one hour after the conclusion of the events and will be archived for approximately one month.

    About Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company dedicated to discovering, developing and delivering life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis*, uterine fibroids* and clinical programs in multiple therapeutic areas. For nearly three decades, Neurocrine Biosciences has specialized in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn(*in collaboration with AbbVie)

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  12. U.S. Food and Drug Administration (FDA) Requests Additional Non-Clinical Data to Support Dose Justification Before Initiation of a Phase II Clinical Trial with NBI-921352 in Pediatric SCN8A-DEE Patients

    FDA Grants Rare Pediatric Disease Designation for NBI-921352 for the Treatment of SCN8A-DEE

    SAN DIEGO and BURNABY, British Columbia, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) and Xenon Pharmaceuticals Inc. (NASDAQ:XENE) today provided an update on the ongoing collaboration for the clinical development of NBI-921352, previously known as XEN901. Neurocrine Biosciences has an exclusive license to NBI-921352, a clinical stage selective Nav1.6 sodium channel inhibitor with potential in SCN8A developmental and…

    U.S. Food and Drug Administration (FDA) Requests Additional Non-Clinical Data to Support Dose Justification Before Initiation of a Phase II Clinical Trial with NBI-921352 in Pediatric SCN8A-DEE Patients

    FDA Grants Rare Pediatric Disease Designation for NBI-921352 for the Treatment of SCN8A-DEE

    SAN DIEGO and BURNABY, British Columbia, Oct. 08, 2020 (GLOBE NEWSWIRE) -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) and Xenon Pharmaceuticals Inc. (NASDAQ:XENE) today provided an update on the ongoing collaboration for the clinical development of NBI-921352, previously known as XEN901. Neurocrine Biosciences has an exclusive license to NBI-921352, a clinical stage selective Nav1.6 sodium channel inhibitor with potential in SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE), a rare pediatric epilepsy, and other forms of epilepsy, including focal epilepsy. The U.S. Food and Drug Administration (FDA) provided feedback on an Investigational New Drug (IND) application submitted by Neurocrine Biosciences in support of a Phase II clinical trial in pediatric SCN8A-DEE patients. As part of its review of the IND, the FDA is requesting additional non-clinical data to support dose justification in this pediatric study. Neurocrine Biosciences and Xenon will engage with the FDA to address the feedback received with the goal of initiating a Phase II clinical trial in 2021. In parallel with this interaction, Neurocrine Biosciences is advancing clinical plans to develop NBI-921352 for the treatment of adult focal epilepsy. In addition, the FDA recently granted Rare Pediatric Disease Designation for NBI-921352 for the treatment of SCN8A-DEE.

    "We expect to engage with the FDA in the near term to discuss their request for additional non-clinical data to enable a pediatric trial in SCN8A-DEE patients. In parallel, we are continuing to develop plans to study NBI-921352 in patients with adult focal epilepsy. We are committed to working with the FDA to address their feedback in a timely manner, with the goal of initiating a Phase II pediatric clinical trial in 2021," said Eiry W. Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences.

    "The recent Rare Pediatric Disease Designation from the FDA underscores that SCN8A-DEE is a devastating pediatric epilepsy, with a lack of approved treatments, that results in serious, life-threatening seizures and neurodevelopmental impairment, further validating our ‘precision medicine' approach to develop treatments for pediatric epilepsies. We are now working with the team at Neurocrine Biosciences to respond to the FDA's request for information and also to support the clinical development plans for NBI-921352 in adult focal epilepsy," said Dr. Simon Pimstone, Xenon's Chief Executive Officer.

    Pursuant to the Collaboration Agreement, upon FDA acceptance of an IND for NBI-921352 in either SCN8A-DEE or another major indication, Xenon is eligible to receive a milestone payment of either $25 million or $10 million, respectively, with 55% of the amount in the form of an equity investment in Xenon at a 15% premium to Xenon's 30-day trailing volume weighted average price at that time.

    About Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis* and uterine fibroids*, with three pivotal and five mid-stage clinical programs in multiple therapeutic areas. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*in collaboration with AbbVie)

    About Xenon Pharmaceuticals Inc.

    Xenon Pharmaceuticals is a clinical stage biopharmaceutical company committed to developing innovative therapeutics to improve the lives of patients with neurological disorders. Xenon is advancing a novel product pipeline of neurology therapies to address areas of high unmet medical need, with a focus on epilepsy. For more information, please visit www.xenon-pharma.com.

    Neurocrine Biosciences 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 collaboration with Xenon Pharmaceuticals Inc., Neurocrine Biosciences' expectations with regard to its interactions and communications with the FDA, the timing of completion of our clinical, regulatory, and other development activities, and the potential to receive a priority review voucher. 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; the impact of the COVID-19 pandemic and efforts to mitigate its spread on our business; risks and uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risk and uncertainties related to any COVID-19 quarantines, shelter-in-place and similar government orders that are currently in place or that may be put in place in the future, including the impact of such orders on our business operations and the business operations of the third parties on which we rely; risks related to the development of our product candidates; risks associated with our dependence on third parties for development and manufacturing activities related to our product candidates, and our ability to manage these third parties; risks that the FDA or other regulatory authorities may make adverse decisions regarding our product candidates; risks that clinical development activities may not be completed on time or at all, or may be delayed for regulatory, manufacturing, COVID-19 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 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; and other risks described in our periodic reports filed with the SEC, including without limitation our quarterly report on Form 10-Q for the quarter ended June 30, 2020. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof.

    Xenon Pharmaceuticals' Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995 and Canadian securities laws. These forward-looking statements are not based on historical fact, and include statements regarding the timing of and results from clinical trials and other development activities, including those related to NBI-921352 and the other pre-clinical compounds covered by our collaboration with Neurocrine Biosciences; the potential efficacy, safety profile, future development plans, addressable market, regulatory success and commercial potential of NBI-921352 and the other pre-clinical compounds covered by our collaboration with Neurocrine Biosciences; the anticipated initiation of future clinical trials for NBI-921352 and the other pre-clinical compounds covered by our collaboration with Neurocrine Biosciences; our ability to achieve milestones in our collaboration with Neurocrine Biosciences and our other development programs; and the timing and results of our and our collaborators' interactions with regulators. These forward-looking statements are based on current assumptions that involve risks, uncertainties and other factors that may cause the actual results, events or developments to be materially different from those expressed or implied by such forward-looking statements. These risks and uncertainties, many of which are beyond our control, include, but are not limited to: the impact of the COVID-19 pandemic on our and our collaborators' business, research and clinical development plans and timelines and results of operations, including impact on our clinical trial sites, collaborators, and contractors who act for or on our behalf, may be more severe and more prolonged than currently anticipated; clinical trials may not demonstrate safety and efficacy of any of our or our collaborators' product candidates; any of our or our collaborators' product candidates may fail in development, may not receive required regulatory approvals, or may be delayed to a point where they are not commercially viable; we may not achieve additional milestones in our proprietary or partnered programs; regulatory agencies may not permit certain of our product candidates to advance directly into a Phase 2 or later clinical trials, may impose additional requirements or delay the initiation of clinical trials; regulatory agencies may be delayed in reviewing, commenting on or approving any of our or our collaborators' clinical development plans as a result of the COVID-19 pandemic, which could further delay development timelines; the impact of competition; the impact of new or changing laws and regulations; adverse conditions in the general domestic and global economic markets; as well as the other risks identified in our filings with the Securities and Exchange Commission and the securities commissions in British Columbia, Alberta and Ontario. These forward-looking statements speak only as of the date hereof and we assume no obligation to update these forward-looking statements, and readers are cautioned not to place undue reliance on such forward-looking statements.

    "Xenon" and the Xenon logo are registered trademarks or trademarks of Xenon Pharmaceuticals Inc. in various jurisdictions. All other trademarks belong to their respective owner.

    Neurocrine Biosciences, Inc. Contacts:

    Navjot Rai (Media), 858-617-7623,

    Todd Tushla (Investors), 858-617-7143,

    Xenon Pharmaceuticals Inc. Contact:

    Jodi Regts (Media and Investors), 604-484-3353,

    Primary Logo

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  13. SAN DIEGO and BOSTON, Oct. 7, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) and Boston-based Me2/Orchestra, the world's only known classical music organization created for people with mental illness and the people that support them, today announced that they have teamed up with the mental health advocacy community to launch Monumental Moments. This new community platform and charitable initiative aims to foster a spirit of togetherness and provide support for people in the mental health community – along with those in the general public – who have experienced a heightened sense of anxiety and stress during this challenging year of social distancing and unrest.

    SAN DIEGO and BOSTON, Oct. 7, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) and Boston-based Me2/Orchestra, the world's only known classical music organization created for people with mental illness and the people that support them, today announced that they have teamed up with the mental health advocacy community to launch Monumental Moments. This new community platform and charitable initiative aims to foster a spirit of togetherness and provide support for people in the mental health community – along with those in the general public – who have experienced a heightened sense of anxiety and stress during this challenging year of social distancing and unrest.

    To coincide with Mental Illness Awareness Week (Oct. 410) and before World Mental Health Day (Oct. 10), Monumental Moments creates an opportunity for people to share their personal moments of success, big or small, in caring for their mental health during this difficult time by posting on social media using the hashtag #MonumentalMoments. The program's microsite, MonumentalMoments.com, will serve as the platform where people can learn more about the initiative, how they can participate, and be connected to a variety of mental health resources.

    "During this time of isolation and tragedy, it's vital that no one feels alone in their mental health struggle. NAMI is proud to be part of the Monumental Moments initiative aimed at providing support through a community platform for people dealing with mental health concerns of varying degrees," said Daniel H. Gillison, Jr., Chief Executive Officer of the National Alliance on Mental Illness (NAMI), the nation's largest grassroots mental health organization. "The platform aims to amplify the positive success stories of lived experience with the hope that others will find solace and encouragement and stay socially connected while remaining physically distant due to the pandemic. We can work together to help create a community where no one feels alone in their struggle."

    As part of Monumental Moments, Me2/Orchestra is unveiling an original musical score inspired by the real-life experiences of its members. The score – created in collaboration with award-winning composer Paul Swartzel – brings to life the emotional impact this year has had on many and symbolizes the Monumental Moments movement. The score will debut as a virtual video performance October 28th on MonumentalMoments.com and will feature up to 100 of its members aged 20 to 80 from around the world, 50% of whom are living with a mental illness.

    "Music can lift our spirits, bring us together and empower us during the hardest of times," said Ronald Braunstein, Me2/Orchestra founder. "The creation of this score for Monumental Moments enables us to channel our emotions and connect with the broad community of people who are feeling the impact of this extraordinary year. The score emphasizes moments of discovery, determination and joy. We hope that it will inspire people to share how they are dealing with the daily stress and challenges of the past year to achieve their own 'monumental moment' and help people realize that we are all going through this unusual time together."

    Recent research from the July KFF Health Tracking Poll, a Kaiser Family Foundation survey tool, has shown that stress and worry related to the pandemic has had a negative impact on mental health, taking an emotional toll on 53% of adults in the U.S. About one third of people surveyed expressed that the stress from the pandemic has negatively impacted their sleep (36%) or appetite (32%).1 For individuals with mental illness, whose conditions often tend to be isolating, the anxiety and increased isolation resulting from these serious times can intensify.2

    People living with mental illness may also be impacted by tardive dyskinesia (TD), an involuntary movement disorder associated with prolonged use of antipsychotics, commonly prescribed to treat schizophrenia, bipolar disorder and depression.3 The uncontrollable movements of TD may be disruptive to people's lives due to the symptoms themselves and the impact they have on emotional and social well-being, causing people to feel embarrassed or withdrawn from society.4,5,6

    Public posts on social media using the hashtag #MonumentalMoments will help support people living with mental health conditions and the challenges that they may experience. On behalf of posts shared, Neurocrine Biosciences will be making donations to several mental health organizations that are working hard to meet the needs of the communities they serve during these difficult times, including the Depression and Bipolar Support Alliance (DBSA), International Bipolar Foundation, Mental Health America (MHA), National Alliance on Mental Illness (NAMI), National Council for Behavioral Health, National Organization for Tardive Dyskinesia and the Schizophrenia and Related Disorders Alliance of America (SARDAA).

    "We are proud to partner with the Me2/Orchestra and mental health advocates in creating Monumental Moments, a new online platform to build a community that aims to uplift and share how we are coping during these trying times while also supporting people struggling with mental health challenges," said Kevin Gorman, Ph.D., Chief Executive Officer at Neurocrine Biosciences. "This initiative builds on our commitment to support people living with mental health conditions and the challenges that they experience, including tardive dyskinesia. Our hope is to empower people to join the Monumental Moments community and reduce the stigma associated with mental illness."

    For more information, visit MonumentalMoments.com and join the conversation online by sharing #MonumentalMoments.

    About Tardive Dyskinesia (TD)

    Tardive dyskinesia (TD) is a movement disorder that is characterized by uncontrollable, abnormal, and repetitive movements of the face, torso and/or other body parts, which may be disruptive and negatively impact patients. The condition is caused by prolonged use of treatments that block dopamine receptors in the brain, such as antipsychotics commonly prescribed to treat mental illnesses such as schizophrenia, bipolar disorder and depression, and certain anti-nausea medications. In patients with TD, these treatments are thought to result in irregular dopamine signaling in a region of the brain that controls movement. TD is estimated to affect at least 500,000 people in the U.S. The symptoms of TD can be severe and are often persistent and irreversible. For more information, visit talkabouttd.com.

    About Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis* and uterine fibroids*, with three pivotal and five mid-stage clinical programs in multiple therapeutic areas. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*in collaboration with AbbVie)

    About Me2/Orchestra

    Me2/Orchestra is the world's only known classical music organization created specifically for individuals living with mental illness and the people who support them. Founded in 2011 by Ronald Braunstein and Caroline Whiddon, both of whom struggle with mental health conditions, it serves as a model organization where people with and without mental illnesses work together in an environment where acceptance is an expectation, patience is encouraged, and supporting each other is a priority. Together, they have found music to be a medium that helps inspire people and bring them together. Me2/Orchestra's mission is to erase the stigma surrounding mental illness through inspiring performances and supportive classical music ensembles.

    1 L. Hamel, A. Kearney, A. Kirzinger, L. Lopes, C. Munana and M. Brodie. KFF Health Tracking Poll – July 2020. Kaiser Family Foundation, July 27, 2020. https://www.kff.org/coronavirus-covid-19/report/kff-health-tracking-poll-july-2020/. Accessed on September 21, 2020.

    2 Centers for Disease Control and Prevention. Coronavirus Disease 2019 (COVID-19) Coping with Stress. https://www.cdc.gov/coronavirus/2019-ncov/daily-life-coping/managing-stress-anxiety.html. Accessed on September 21, 2020.

    3 American Psychiatric Association. Diagnostic and Statistical Manual of Mental Disorders. 5th ed. Arlington, VA: American Psychiatric Association; 2013

    4 Task Force on Tardive Dyskinesia. Tardive Dyskinesia: A Task Force Report of the American Psychiatric Association. American Psychiatric Association; Washington, DC; 1992.

    5 Boumans C, de Mooij K, Koch P, Al. E. Is the Social Acceptability of Psychiatric Patients Decreased by Orofacial Dyskinesia? Schizophr Bull. 1994;20(2):339-344.

    6 Citrome L. Clinical management of tardive dyskinesia: Five steps to success. J. Neurol Sci. 2017;383:199-204.

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    SOURCE Neurocrine Biosciences, Inc.

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  14. SAN DIEGO, Oct. 6, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) announced today that it will report third quarter financial results after the Nasdaq market closes on Monday, November 9, 2020. Neurocrine will then host a conference call and webcast to discuss its financial results and provide a company update that day at 1:30 p.m. Pacific Time (4:30 p.m. Eastern Time).

    Participants can access the live conference call by dialing 877-830-2596 (US) or 785-424-1744 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine's website under Investors at www.neurocrine.com. 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, Inc.

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis* and uterine fibroids*, with three pivotal and five mid-stage clinical programs in multiple therapeutic areas. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn(*in collaboration with AbbVie)

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/neurocrine-biosciences-announces-conference-call-and-webcast-of-third-quarter-2020-financial-results-301147076.html

    SOURCE Neurocrine Biosciences, Inc.

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  15. SAN DIEGO, Sept. 28, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced it will present data analyses evaluating the efficacy and safety of ONGENTYS® (opicapone) capsules, recently approved by the U.S. Food and Drug Administration (FDA) as the first and only once-daily catechol-O-methyltransferase (COMT) inhibitor as an add-on to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes. New data from a post-hoc, sub-group analysis of Phase III data showed that ONGENTYS led to greater reductions in overnight "off" time and time to morning "on" time compared to entacapone in patients with Parkinson's disease with motor fluctuations. In another Phase III post-hoc sub-group analysis, long-term…

    SAN DIEGO, Sept. 28, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced it will present data analyses evaluating the efficacy and safety of ONGENTYS® (opicapone) capsules, recently approved by the U.S. Food and Drug Administration (FDA) as the first and only once-daily catechol-O-methyltransferase (COMT) inhibitor as an add-on to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes. New data from a post-hoc, sub-group analysis of Phase III data showed that ONGENTYS led to greater reductions in overnight "off" time and time to morning "on" time compared to entacapone in patients with Parkinson's disease with motor fluctuations. In another Phase III post-hoc sub-group analysis, long-term use of ONGENTYS in patients with Parkinson's disease with motor fluctuations reduced "on" time with troublesome dyskinesia and increased good "on" time without troublesome dyskinesia. In this analysis, long-term use of ONGENTYS was associated with a reduction in the patient's average daily levodopa dosage requirement. These data will be presented in collaboration with BIAL at the American Neurological Association (ANA) 2020 Virtual Meeting on October 4–9, 2020.

    "As Parkinson's disease progresses and the benefit of treatment with levodopa/carbidopa begins to wear off between doses, many patients experience increased fluctuation and unpredictability in their motor function, which can include more 'off' time overnight and during the day, and a reduction in good 'on' time where movement is close to normal," said Eiry W. Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences. "These post-hoc analyses from the Phase III clinical trials of ONGENTYS provide further insight on how adding once-daily ONGENTYS to levodopa/carbidopa can help patients with Parkinson's disease better manage disruptive motor fluctuations over the course of the day." 

    The six ONGENTYS abstracts that will be presented at the ANA 2020 Virtual Meeting are:

    • Long-Term Efficacy of Opicapone in the Reduction of ON-Time with Troublesome Dyskinesia in Parkinson's Disease Patients with Motor Fluctuations and Reporting Troublesome Dyskinesia (Poster #489)
    • Effects of Once-Daily Opicapone on Duration of Overnight OFF and Time to Morning ON in Patients with Parkinson's Disease and Motor Fluctuations (Poster #494)
    • Efficacy of Opicapone at Different Levodopa Regimens up to a Threshold of 600 mg/day Levodopa in Parkinson's Disease Patients with Motor Fluctuations (Poster #490)
    • Effect of Opicapone and Entacapone on Early Morning-OFF Pattern in Parkinson's Disease Patients with Motor Fluctuations (Poster #477)
    • Characterization of the Pattern of Daily Motor Fluctuations in Parkinson's Disease Patients Based on Home Diaries (Poster #493)
    • Onset of Drug-Related Adverse Events in Parkinson's Disease Patients with Motor Fluctuations Treated with Opicapone in Clinical Practice: OPTIPARK Post-Hoc Analysis (Poster #485)

    About Parkinson's Disease

    Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disorder that affects approximately one million people in the United States and six million people worldwide. Parkinson's disease is caused by low dopamine levels produced in the brain. Dopamine helps transmit signals between the areas of the brain that control all purposeful movements, including talking, walking and writing. As Parkinson's disease progresses, dopamine production steadily decreases, resulting in increased problems with motor symptoms including slowed movement (bradykinesia), tremor, rigidity, impaired posture and balance, and difficulty with speech and writing.

    There is presently no cure for Parkinson's disease and management of the disease consists of the use of treatments that attempt to control motor symptoms primarily through dopaminergic mechanisms. The current gold standard for treatment of motor symptoms is levodopa/carbidopa. While levodopa/carbidopa improves patients' motor symptoms, as the disease progresses, the beneficial effects of levodopa begin to wear off more quickly. Patients then experience motor fluctuations throughout the day between "on" time, periods when the medication is working and Parkinson's disease symptoms are controlled, and "off" time, when the medication is not working and motor symptoms return.

    About ONGENTYS® (opicapone) Capsules 

    ONGENTYS is a unique, once-daily, oral, peripheral, selective and reversible catechol-O-methyltransferase (COMT) inhibitor approved by the U.S. Food and Drug Administration (FDA) as an add-on treatment to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes. ONGENTYS inhibits the COMT enzyme, which breaks down levodopa, making more levodopa available to reach the brain.

    In June 2016, BIAL – Portela & CA, S.A. (BIAL) received approval from the European Commission for ONGENTYS as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors in adult patients with Parkinson's disease and end-of-dose motor fluctuations who cannot be stabilized on those combinations. BIAL currently markets ONGENTYS in Germany, United Kingdom, Spain, Portugal and Italy. Neurocrine Biosciences in-licensed opicapone from BIAL in 2017 and has exclusive development and commercialization rights in the U.S. and Canada.

    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 of 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 Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis* and uterine fibroids*, with three pivotal and five mid-stage clinical programs in multiple therapeutic areas. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*in collaboration with AbbVie)

    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 ONGENTYS; and the continued success of the launch of ONGENTYS; our expectations regarding business and financial impacts of the COVID-19 pandemic, including with respect to ONGENTYS availability and the ONGENTYS commercial supply chain and other business operations; and whether results from ONGENTYS's clinical trial results are indicative of real-world results. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are: risks and uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risk and uncertainties related to any COVID-19 quarantines, shelter-in-place and similar government orders that are currently in place or that may be put in place in the future, including the impact of such orders on our business operations and the business operations of the third parties on which we rely; risks and uncertainties associated with Neurocrine Biosciences' business and finances in general, as well as risks and uncertainties associated with the commercialization of ONGENTYS; whether ONGENTYS receives adequate reimbursement from third-party payors; the degree and pace of market uptake of ONGENTYS; risks and uncertainties relating to competitive products and technological changes that may limit demand for ONGENTYS; risks that additional regulatory submissions, for ONGENTYS or other product candidates, may not occur or be submitted in a timely manner; risks that the FDA or other regulatory authorities may make adverse decisions regarding ONGENTYS; risks that post-approval ONGENTYS commitments or requirements may be delayed; risks that ONGENTYS may be precluded from commercialization or continued commercialization by the proprietary rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; risks and uncertainties relating to competitive products and technological changes that may limit demand for ONGENTYS; risks associated with the Company's dependence on BIAL for the commercial supply of, and manufacturing activities related to, ONGENTYS, and the ability of the Company to manage BIAL; and other risks described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's quarterly report on Form 10-Q for the quarter ended June 30, 2020. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof.

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/neurocrine-biosciences-to-present-new-data-analyses-of-once-daily-ongentys-opicapone-in-patients-with-parkinsons-disease-at-the-american-neurological-association-2020-virtual-meeting-301139245.html

    SOURCE Neurocrine Biosciences, Inc.

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  16. SAN DIEGO, Sept. 14, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced that 50 mg capsules of ONGENTYS® (opicapone), the first and only FDA-approved once-daily catechol-O-methyltransferase (COMT) inhibitor, are now available by prescription in the United States. ONGENTYS was approved by the U.S. Food and Drug Administration (FDA) on April 24, 2020, as an add-on treatment to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes.

    Experience the interactive Multichannel News Release here: https://www.multivu.com/players/English/8773451-neurocrine-biosciences-ongentys-now-available/

    Parkinson's disease is the second most common neurodegenerative disorder in the United States after Alzheimer's…

    SAN DIEGO, Sept. 14, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced that 50 mg capsules of ONGENTYS® (opicapone), the first and only FDA-approved once-daily catechol-O-methyltransferase (COMT) inhibitor, are now available by prescription in the United States. ONGENTYS was approved by the U.S. Food and Drug Administration (FDA) on April 24, 2020, as an add-on treatment to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes.

    Experience the interactive Multichannel News Release here: https://www.multivu.com/players/English/8773451-neurocrine-biosciences-ongentys-now-available/

    Parkinson's disease is the second most common neurodegenerative disorder in the United States after Alzheimer's disease. About one million Americans have Parkinson's disease and each year, an estimated 50,000 people in the United States are newly diagnosed with this chronic, progressive and debilitating neurodegenerative disorder.

    "ONGENTYS is a new treatment option that decreases 'off' time, the period of time during the day when Parkinson's disease symptoms are bothersome, and increases 'on' time without troublesome dyskinesia, the period of time during the day when Parkinson's disease symptoms are better controlled," said Rebecca Gilbert, M.D., Ph.D., Vice President and Chief Scientific Officer of the American Parkinson Disease Association. "The approval of ONGENTYS is welcome news to people with Parkinson's disease who are looking for additional medication possibilities to help control the often difficult symptoms of the disease that negatively impact their lives."

    ONGENTYS is an oral, selective COMT inhibitor that helps block the COMT enzyme that breaks down levodopa, the gold standard therapy for controlling motor symptoms in patients with Parkinson's disease. ONGENTYS helps protect levodopa by reducing its breakdown in the bloodstream, making more levodopa available to reach the brain.

    "Parkinson's disease is a progressive, debilitating condition where people often struggle to control motor fluctuations, impacting many aspects of their daily life," said Eiry W. Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences. "The availability of ONGENTYS offers hope to patients by significantly reducing daily 'off' time, when symptoms return between regular doses of levodopa/carbidopa. The availability of ONGENTYS underscores our commitment to delivering innovative therapies that address unmet medical needs in patients living with movement disorders."

    Neurocrine Biosciences INBRACE® Support Program supports patients who are prescribed ONGENTYS along their treatment journey including information on prescription fulfillment, navigating health insurance coverage requirements and financial assistance. For more information, patients may visit www.INBRACEsupportprogram.com

    The FDA approval of ONGENTYS is supported by data from 38 clinical studies, including two multinational Phase III clinical studies (BIPARK-1 and BIPARK-2), with more than 1,000 Parkinson's disease patients treated with ONGENTYS. In the BIPARK-1 trial, approximately 600 patients with Parkinson's disease and motor fluctuations received one of three doses of ONGENTYS (5 mg, 25 mg or 50 mg), placebo, or 200 mg doses of the COMT inhibitor entacapone for 14 or 15 weeks. In the BIPARK-2 trial, approximately 400 patients received one of two doses of ONGENTYS (25 mg or 50 mg) or placebo for 14 or 15 weeks. Both studies included a one-year open-label extension. Data from both trials showed that ONGENTYS 50 mg significantly reduced "off" time from baseline to week 14 or 15 compared to placebo. "On" time without troublesome dyskinesia also increased from baseline to week 14 or 15 compared to placebo.

    Pooled safety data from the BIPARK-1 and BIPARK-2 studies indicated that the most common adverse reactions across all patients treated with ONGENTYS (incidence at least 4% and greater than placebo) were dyskinesia, constipation, blood creatine kinase increase, hypotension/syncope, and weight decrease.

    In June 2016, BIAL – Portela & CA, S.A. (BIAL) received approval from the European Commission for ONGENTYS as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors in adult patients with Parkinson's disease and end-of-dose motor fluctuations who cannot be stabilized on those combinations. BIAL currently markets ONGENTYS in Germany, United Kingdom, Spain, Portugal and Italy. Neurocrine Biosciences in-licensed opicapone from BIAL in 2017 and has exclusive development and commercialization rights in the United States and Canada.

    About Parkinson's Disease

    Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disorder that affects approximately one million people in the United States and six million people worldwide. Parkinson's disease is caused by low dopamine levels produced in the brain. Dopamine helps transmit signals between the areas of the brain that control all purposeful movements, including talking, walking and writing. As Parkinson's disease progresses, dopamine production steadily decreases, resulting in increased problems with motor symptoms including slowed movement (bradykinesia), tremor, rigidity, impaired posture and balance, and difficulty with speech and writing.

    There is presently no cure for Parkinson's disease and management of the disease consists of the use of treatments that attempt to control motor symptoms primarily through dopaminergic mechanisms. The current gold standard for treatment of motor symptoms is levodopa/carbidopa. While levodopa/carbidopa improves patients' motor symptoms, as the disease progresses, the beneficial effects of levodopa begin to wear off more quickly. Patients then experience motor fluctuations throughout the day between "on" time, periods when the medication is working and Parkinson's disease symptoms are controlled, and "off" time, when the medication is not working and motor symptoms return.

    About ONGENTYS® (opicapone) Capsules

    ONGENTYS is a unique 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. ONGENTYS inhibits the COMT enzyme, which breaks down levodopa, making more levodopa available to reach the brain.

    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 Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis* and uterine fibroids*, with three pivotal and five mid-stage clinical programs in multiple therapeutic areas. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*in collaboration with AbbVie).

    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 ONGENTYS; the size of the potential market for ONGENTYS; the value ONGENTYS brings to patients; the timing of ONGENTYS's availability; the ability of Neurocrine Biosciences to ensure patients have access to ONGENTYS; our expectations regarding business and financial impacts of the COVID-19 pandemic, including with respect to ONGENTYS availability and the ONGENTYS commercial supply chain and other business operations; and whether results from ONGENTYS's clinical trial results are indicative of real-world results. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are: risks and uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risk and uncertainties related to any COVID-19 quarantines, shelter-in-place and similar government orders that are currently in place or that may be put in place in the future, including the impact of such orders on our business operations and the business operations of the third parties on which we rely; risks and uncertainties associated with Neurocrine Biosciences' business and finances in general, as well as risks and uncertainties associated with the commercialization of ONGENTYS; whether ONGENTYS receives adequate reimbursement from third-party payors; the degree and pace of market uptake of ONGENTYS; risks and uncertainties relating to competitive products and technological changes that may limit demand for ONGENTYS; risks that additional regulatory submissions, for ONGENTYS or other product candidates, may not occur or be submitted in a timely manner; risks that the FDA or other regulatory authorities may make adverse decisions regarding ONGENTYS; risks that post-approval ONGENTYS commitments or requirements may be delayed; risks that ONGENTYS may be precluded from commercialization by the proprietary rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; risks and uncertainties relating to competitive products and technological changes that may limit demand for ONGENTYS; risks associated with the Company's dependence on BIAL for the commercial supply of, and manufacturing activities related to, ONGENTYS, and the ability of the Company to manage BIAL; and other risks described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's quarterly report on Form 10-Q for the quarter ended June 30, 2020. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof.

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  17. -- Data for Investigational Gene Therapy Treatment NBIb-1817 (VY-AADC) Presented at the MDS Virtual Congress 2020 --

    • NBIb-1817 Treatment Showed Sustained Improvement in Motor Function, Including Greater "On" Time without Troublesome Dyskinesia and Reduction in Unified Parkinson's Disease Rating Scale (UPDRS) Part III Scores, and Reduction in the Amount of Medications Up to Three Years in Patients with Parkinson's Disease

    • 14 of 15 Patients Treated with NBIb-1817 Continued to Experience an Improvement in Disease Staging after Three Years, as Assessed by the Modified Hoehn & Yahr Scale
       
    • Re-Initiation of Enrollment in Registrational RESTORE-1 Clinical Trial of NBIb-1817 Planned for Later this Year

    SAN DIEGO and CAMBRIDGE, Mass., Sept. 11, 2020 (GLOBE…

    -- Data for Investigational Gene Therapy Treatment NBIb-1817 (VY-AADC) Presented at the MDS Virtual Congress 2020 --

    • NBIb-1817 Treatment Showed Sustained Improvement in Motor Function, Including Greater "On" Time without Troublesome Dyskinesia and Reduction in Unified Parkinson's Disease Rating Scale (UPDRS) Part III Scores, and Reduction in the Amount of Medications Up to Three Years in Patients with Parkinson's Disease



    • 14 of 15 Patients Treated with NBIb-1817 Continued to Experience an Improvement in Disease Staging after Three Years, as Assessed by the Modified Hoehn & Yahr Scale

       
    • Re-Initiation of Enrollment in Registrational RESTORE-1 Clinical Trial of NBIb-1817 Planned for Later this Year

    SAN DIEGO and CAMBRIDGE, Mass., Sept. 11, 2020 (GLOBE NEWSWIRE) -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) and Voyager Therapeutics, Inc. (NASDAQ:VYGR) today announced data from  PD-1101, a Phase Ib open-label, three-year efficacy and safety study, demonstrating that a one-time treatment with investigational gene therapy, NBIb-1817 (VY-AADC), showed sustained improvement in motor function including greater "On" time without troublesome dyskinesia, reduction in Unified Parkinson's Disease Rating Scale (UPDRS) Part III scores, and reduction in the amount of medications in patients with Parkinson's disease. In the PD-1101 study, NBIb-1817 reduced average "Off" time by up to -1.91 hours and improved average "On" time without troublesome dyskinesia by up to +2.23 hours in patients with advanced Parkinson's disease after three years across three cohorts. In addition, 14 out of 15 patients treated with NBIb-1817 continued to show an improvement in disease staging after three years, as assessed by the modified Hoehn & Yahr scale. These new data, along with two-year data from another open-label Phase Ib trial, PD-1102, were presented today at the MDS Virtual Congress 2020, September 12–16, 2020 (www.mdscongress.org/Congress/Registration.htm).

    In data from the three-year PD-1101 trial, the one-time treatment with NBIb-1817 showed sustained reduction in diary "Off" time by an average of -0.15 to -1.91 hours (baseline 4.28 to 4.93 hours) and improved diary "On" time without troublesome dyskinesia by an average of +0.26 to +2.23 hours (baseline 10.32 to 10.46 hours) across the cohorts as reported by 15 patients with advanced Parkinson's disease. NBIb-1817 also showed sustained improvement in motor function after three years, as measured by UPDRS Part III off medication scores, by -10.2 to -19.0 points (baseline 35.8 to 38.2 points) across the cohorts, per clinician assessment. Requirements for Parkinson's disease medications were also reduced in cohorts 2 and 3 (daily levodopa-equivalent dose reductions, average of -322.0 and -441.2 mg/day, respectively; baseline 1507.0 and 1477.0 mg/day, respectively). Two-year data from the PD-1102 trial for 7 patients showed that NBIb-1817 reduced diary "Off" time by an average of -3.2 hours and increased diary good "On" time by +2.1 hours (baselines 9.3 hours and 6.6 hours, respectively). In this study, NBIb-1817 showed sustained improvement in motor function after two years, with improved UPDRS Part III off medication scores of -12.0 points (baseline 34.4). Requirements for Parkinson's disease medications were also reduced (daily levodopa-equivalent dose reduction, average pf -439.5 mg/day; baseline 1500.9 mg/day). Preliminary safety data from both studies suggest that NBIb-1817 was well-tolerated, with no study drug-related serious adverse events (SAEs) reported. The most common adverse events reported were headache, hypoesthesia, and musculoskeletal pain (PD-1101), and upper respiratory tract infection, headache, nausea, and depression (PD-1102). 

    "It is promising to see that after three years, a single administration of one-time investigational gene therapy treatment NBIb-1817 showed sustained reduction in "Off" time, as well as improvement in "On" time without troublesome dyskinesia and other measures of motor function in patients with Parkinson's disease," said Chad Christine, M.D., primary author, a lead investigator of the study and Professor of Neurology at the University of California, San Francisco (UCSF) Weill Institute for Neurosciences. "Parkinson's disease patients' motor function would be expected to worsen over three years, making these results very encouraging. The standard of care for advanced Parkinson's disease has not significantly changed in decades and it is our hope that NBIb-1817 has the potential to become the first gene therapy for Parkinson's disease."

    Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disorder that affects approximately one million people in the U.S. and six million people worldwide. It is characterized by a loss of dopamine from neuronal degeneration, with a concomitant loss of the aromatic L-amino acid decarboxylase (AADC) enzyme required to synthesize dopamine in the brain, leading to associated impairment in motor, neuropsychiatric, and autonomic functions.

    "We are pleased that the results from these studies show that one-time treatment with investigational NBIb-1817 may help restore the brain's ability to convert levodopa into dopamine," said Eiry Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences. "Our hope is that NBIb-1817 will help patients experience less "Off" time and more "On" time and improve motor symptom control. We plan to re-initiate enrollment in our registrational RESTORE-1 clinical trial with NBIb-1817 this year and look forward to further evaluating NBIb-1817 in patients with Parkinson's disease."

    NBIb-1817 is an investigational recombinant adeno-associated viral serotype 2 vector encoding the gene for human AADC that is designed to help produce the AADC enzyme in brain cells where it can convert levodopa to dopamine.

    "We are encouraged by the congruence of long-term data, including clinician- and patient-reported clinical outcomes in our clinical studies," said Omar Khwaja, M.D., Ph.D., Chief Medical Officer and Head of Research and Development at Voyager Therapeutics. "These results are promising and show that the approach has the potential to transform the treatment of Parkinson's disease, and help improve the lives of patients and their families."

    Additional information about PD-1101 and PD-1102 will be available on demand for registered participants through October 1, 2020 on the MDS meeting website (www.mdscongress.org/Congress/Registration.htm).

    • Christine CW, Richardson RM, Van Laar AD, et al. Three-Year Safety and Clinical Outcomes from the PD-1101 Trial of AADC Gene Therapy for Advanced Parkinson's Disease

      Poster # 879: Update on Genetics of Movement Disorders, September 13, 2020, 10:30–12:30pm EST (10-minute prerecorded presentation)
    • Factor SA, Van Laar AD, Richardson RM, et al. AADC Gene Therapy Administered via a Posterior Approach: 24-Month Results from the PD-1102 Trial in Advanced Parkinson's Disease

      Poster # 889: Poster Tour, launches on-demand on September 11, 2020 8:00am EST (5-minute prerecorded presentation)

    About Parkinson's Disease and NBIb-1817 (VY-AADC)

    Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disease that affects approximately one million people in the U.S. and six million people worldwide. It is characterized by a loss of dopamine and neuronal degeneration with a concomitant loss of the aromatic L-amino acid decarboxylase (AADC) enzyme required to synthesize dopamine in the brain, leading to associated impairment in motor, neuropsychiatric, and autonomic functions. Dopamine is a chemical "messenger" that is produced in the brain and is involved in the control of movement. It is made when AADC converts the chemical levodopa to dopamine. As Parkinson's disease progresses, there is less AADC enzyme in parts of the brain where levodopa is converted to dopamine.

    NBIb-1817 is an investigational recombinant adeno-associated viral (AAV) serotype 2 vector encoding the gene for human AADC that is designed to help produce the AADC enzyme in brain cells where it can convert levodopa to dopamine. NBIb-1817 is administered into the brain using intraoperative monitoring with magnetic resonance imaging (MRI)-facilitated targeted delivery.

    About the RESTORE-1 Clinical Trial

    Paused temporarily in April 2020 due to the COVID-19 pandemic, Neurocrine Biosciences and Voyager Therapeutics plan to re-initiate RESTORE-1, a Phase 2, randomized, placebo-surgery controlled, double-blinded, multi-center clinical trial, to evaluate the safety and efficacy of NBIb-1817 in patients who have been diagnosed with Parkinson's disease for at least four years and have at least three hours of "Off" time during the day as measured by a validated self-reported patient diary.

    For more information about the RESTORE-1 clinical trial, including eligibility criteria, please visit clinicaltrials.gov and restore1study.com.

    About the RESTORE-2 Clinical Trial

    Preparations are ongoing for the RESTORE-2 global registrational trial that will include clinical sites within and outside the U.S.

    About Neurocrine Biosciences and Voyager Therapeutics Strategic Collaboration

    In 2019, Neurocrine Biosciences and Voyager Therapeutics entered into a strategic collaboration focused on the development and commercialization of gene therapy programs, VY-AADC for Parkinson's disease and VY-FXN01 for Friedreich's ataxia, as well as rights to two programs to be determined. This collaboration combines Neurocrine Biosciences' expertise in neuroscience, drug development and commercialization with Voyager's innovative gene therapy programs targeting severe neurological diseases.

    About Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis* and uterine fibroids*, with three pivotal and five mid-stage clinical programs in multiple therapeutic areas. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*in collaboration with AbbVie)

    About Voyager Therapeutics

    Voyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyager's wholly owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinson's disease, Huntington's disease, Friedreich's ataxia, and other severe neurological diseases. For more information on Voyager, please visit the company's website at www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and LinkedIn.

    Voyager Therapeutics® is a registered trademark of Voyager Therapeutics. 

    Neurocrine Biosciences Forward-Looking Statements

    In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. Among the factors and risks that could cause actual results to differ materially from those indicated in the forward-looking statements are risks that the product candidates licensed from Voyager may not obtain regulatory approval from the FDA or other regulatory agencies, or such approval may be delayed or conditioned; risks that development activities related to the product candidates licensed from Voyager may not be completed on time or at all; risks associated with the Company's dependence on Voyager for research, development and manufacturing activities; risks that ongoing or future clinical trials may not be successful or replicate previous clinical trial results, or may not be predictive of real-world results or of results in subsequent clinical trials; risks and uncertainties relating to competitive products and technological changes that may limit demand for product candidates licensed from Voyager; risks that the product candidates licensed from Voyager may be precluded from commercialization by the proprietary rights of third parties; the impact of the COVID-19 pandemic and efforts to mitigate its spread on our business; risks and uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risk and uncertainties related to any COVID-19 quarantines, shelter-in-place and similar government orders that are currently in place or that may be put in place in the future, including the impact of such orders on our business operations and the business operations of the third parties on which we rely; risks related to the development of our product candidates; and other risks that are described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's quarterly report on Form 10-Q for the quarter ended June 30, 2020. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof.

    Voyager Therapeutics Forward-Looking Statements

    This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "might," "will," "would," "should," "expect," "plan," "anticipate," "believe," "estimate," "undoubtedly," "project," "intend," "future," "potential," or "continue," and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager Therapeutics makes regarding the potential impact or significance of the long-term medical data for patients treated in the PD-1101 and PD-1102 clinical trials; the re-initiation of RESTORE-1 Phase 2 clinical trial prior to year-end, the initiation of the RESTORE-2 Phase 3 clinical trial during the first half of 2021; the initiation, timing, progress, activities, goals and reporting of results of other activities associated with the PD program, and the potential benefits, timing and future operation of the collaboration with Neurocrine Biosciences are forward looking. All forward-looking statements are based on estimates and assumptions by Voyager's management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, risks that ongoing or future clinical trials may not be successful or replicate previous clinical trial results, or may not be predictive of real-world results or of results in subsequent clinical trials; risks and uncertainties relating to competitive products and technological changes that may limit demand for product candidates now being evaluated in clinical trials; the impact of the COVID-19 pandemic and efforts to mitigate its spread on our clinical trials and our business generally; risks related to the initiation and conduct of preclinical studies and clinical trials; the sufficiency of preclinical and clinical data to support applications for additional studies and marketing approval of our PD drug development candidates; changes in expectations from the FDA and other regulatory authorities as to the requirements for obtaining product approvals; the decisions of the FDA and other regulatory authorities in response to applications we file in connection with our product candidates under our PD program and otherwise in our conduct of PD drug development activities; the priorities, capabilities, diligence and efforts of Neurocrine Biosciences, our collaboration partner for the PD program, and other collaborators and vendors supporting our PD program; and the  commercial potential of PD product candidates that may be developed as part of our PD program. These statements are also subject to a number of material risks and uncertainties that are described in Voyager Therapeutics' Annual Report on Form 10K, Voyager Therapeutics' Quarterly Reports on Form 10-Q and other reports filed by Voyager Therapeutics with the Securities and Exchange Commission, as may be updated by its subsequent filings with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager Therapeutics undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

    Contact: Neurocrine Biosciences

    Navjot Rai (Media)

    858-617-7623

    Todd Tushla (Investors)

    858-617-7143

    Contact: Voyager Therapeutics

    Paul Cox (Investors)

    857-201-3463

    Sheryl Seapy

    W2Opure

    949-903-4750

     

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  18. SAN DIEGO, Sept. 11, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced new data from two post-hoc analyses of Phase III data, demonstrating that once-daily ONGENTYS® (opicapone) capsules decreased "off" time and increased "on" time without troublesome dyskinesia as an add-on therapy to levodopa/carbidopa in patients with Parkinson's disease who experience motor fluctuations. Neurocrine Biosciences also presented real-world data showing the increased burden motor fluctuations have over time on Parkinson's disease patients and the healthcare system through significantly more hospitalizations and emergency room visits. These data are among several studies and analyses of ONGENTYS being presented in collaboration…

    SAN DIEGO, Sept. 11, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced new data from two post-hoc analyses of Phase III data, demonstrating that once-daily ONGENTYS® (opicapone) capsules decreased "off" time and increased "on" time without troublesome dyskinesia as an add-on therapy to levodopa/carbidopa in patients with Parkinson's disease who experience motor fluctuations. Neurocrine Biosciences also presented real-world data showing the increased burden motor fluctuations have over time on Parkinson's disease patients and the healthcare system through significantly more hospitalizations and emergency room visits. These data are among several studies and analyses of ONGENTYS being presented in collaboration with BIAL at the MDS Virtual Congress 2020 on September 12–16 (www.mdscongress.org/Congress/Registration.htm).

    ONGENTYS, approved by the U.S. Food and Drug Administration in April 2020, is the first and only once-daily catechol-O-methyltransferase (COMT) inhibitor approved as an add-on to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes and will be available to wholesalers in September.

    "As Parkinson's disease progresses and treatment with levodopa/carbidopa begins to wear off between treatment doses, many patients begin to experience increased motor fluctuations," said Robert A. Hauser, M.D., Professor of Neurology and Director, University of South Florida Parkinson's Disease and Movement Disorders Center. "The Phase III post-hoc data analyses demonstrated the benefit of adding once-daily ONGENTYS to levodopa/carbidopa in patients with Parkinson's disease who have motor fluctuations. Patients with Parkinson's disease now have a new treatment option to help provide more control of motor symptoms."

    Data from a pooled post-hoc, sub-group analysis of Phase III studies demonstrated that ONGENTYS 50 mg significantly reduced "off" time by more than an hour compared to placebo when used as an add-on treatment in patients with Parkinson's disease treated with levodopa/carbidopa plus placebo at baseline (109.2 minutes for ONGENTYS 50 mg [n=67] vs. 40.3 minutes for placebo [n=59]; p=0.0161).

    In a separate post-hoc analysis, ONGENTYS significantly increased absolute "on" time by approximately one additional hour compared with entacapone (124 minutes [n=50] vs. 60 minutes [n=47]; p=0.0344) when used as the first COMT inhibitor add-on therapy to levodopa/carbidopa in patients with Parkinson's disease recently diagnosed with motor fluctuations.

    "These data analyses demonstrated the benefit of adding once-daily ONGENTYS to levodopa/carbidopa earlier in the treatment regimen of patients with Parkinson's disease. In addition to decreasing 'off' time the data also show that ONGENTYS significantly increased 'on' time compared to an older COMT inhibitor to help control motor fluctuations in patients with Parkinson's disease," said Eiry W. Roberts, MD, Chief Medical Officer, Neurocrine Biosciences. "We are looking forward to bringing ONGENTYS to patients as a new add-on treatment option as data from our real-world study show that the debilitating symptoms of Parkinson's disease result in more hospitalizations and emergency room visits, impacting the healthcare system in the U.S."

    Neurocrine Biosciences also presented real-world data from a retrospective medical chart review of adult patients with Parkinson's disease who began experiencing motor fluctuations while taking levodopa. Of the 310 patients included in the review, 117 (38%) had a history of motor fluctuations of ≥ 2 years. Data show that emergency department visits were significantly more frequent in patients with Parkinson's disease with a longer history of motor fluctuations (≥2 years) compared to patients with a shorter history (13% [n=15] vs. 3% [n=5]; P<0.001). Similarly, hospitalizations were significantly more frequent in patients with a longer history of motor fluctuations (15% [n=18] vs 6% [n=12], P<0.01). Among patients who were hospitalized, the mean length of stay was shorter in patients with motor fluctuations ≥2 years versus patients with motor fluctuations <2 years, but the difference was not statistically significant (0.5 vs 1.1 days; P>0.05).

    About the BIPARK-1 Study

    BIPARK-1 was a Phase III, randomized, double-blind placebo- and active-controlled study of ONGENTYS as an adjunct to levodopa therapy in which approximately 600 patients with Parkinson's disease and motor fluctuations received once-daily doses of opicapone (5 mg, 25 mg, or 50 mg), placebo, or 200 mg doses of the COMT inhibitor entacapone for 14 to 15 weeks. The primary endpoint was the change from baseline in absolute time in the "off" state, as assessed by patient diaries. The initial study period was followed by a one-year open-label phase during which all patients received treatment with opicapone.

    About the BIPARK-2 Study

    BIPARK-2 was a Phase III, randomized, double-blind placebo-controlled study of opicapone as an adjunct to levodopa therapy in which approximately 400 patients with Parkinson's disease and motor fluctuations received once-daily doses of opicapone (25 mg or 50 mg) or placebo for 14 to 15 weeks. The primary endpoint was the change from baseline in absolute time in the "off" state, as assessed by patient diaries. The initial study period was followed by a one-year open-label phase during which all patients received treatment with opicapone.

    BIPARK-1 and BIPARK-2 were conducted by BIAL – Portela & CA, S.A. (BIAL). Neurocrine Biosciences in-licensed opicapone from BIAL in 2017 and has exclusive development and commercialization rights in the U.S. and Canada. BIAL received approval from the European Commission for ONGENTYS as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors in adult patients with Parkinson's disease and end-of-dose motor fluctuations who cannot be stabilized on those combinations. BIAL currently markets ONGENTYS in Germany, United Kingdom, Spain, Portugal and Italy. 

    About Parkinson's Disease

    Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disorder that affects approximately one million people in the United States and six million people worldwide. Parkinson's disease is associated with low dopamine levels produced in the brain. Dopamine helps transmit signals between the areas of the brain that control all purposeful movements, including talking, walking and writing. As Parkinson's disease progresses, dopamine production steadily decreases, resulting in increased problems with motor symptoms including slowed movement (bradykinesia), tremor, rigidity, impaired posture and balance, and difficulty with speech and writing.

    There is presently no cure for Parkinson's disease and management of the disease consists of the use of treatments that attempt to control motor symptoms primarily through dopaminergic mechanisms. The current gold standard for treatment of motor symptoms is levodopa/carbidopa. While levodopa/carbidopa improves patients' motor symptoms, as the disease progresses, the beneficial effects of levodopa begin to wear off more quickly. Patients then experience motor fluctuations throughout the day between "on" time, periods when the medication is working and Parkinson's disease symptoms are controlled, and "off" time, when the medication is not working, and motor symptoms return.

    About ONGENTYS® (opicapone) Capsules 

    ONGENTYS is a unique, once-daily, oral, peripheral, selective and reversible catechol-O-methyltransferase (COMT) inhibitor approved by the U.S. Food and Drug Administration (FDA) as an add-on treatment to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes. ONGENTYS inhibits the COMT enzyme, which breaks down levodopa, making more levodopa available to reach the brain.

    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 of 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 Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis* and uterine fibroids*, with three pivotal and five mid-stage clinical programs in multiple therapeutic areas. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn(*in collaboration with AbbVie)

    Forward Looking Statement

    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 ONGENTYS; the size of the potential market for ONGENTYS; the value ONGENTYS brings to patients; the timing of ONGENTYS's availability; the ability of Neurocrine Biosciences to ensure patients have access to ONGENTYS; our expectations regarding business and financial impacts of the COVID-19 pandemic, including with respect to ONGENTYS availability and the ONGENTYS commercial supply chain and other business operations; and whether results from ONGENTYS's clinical trial results are indicative of real-world results. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are: risks and uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risk and uncertainties related to any COVID-19 quarantines, shelter-in-place and similar government orders that are currently in place or that may be put in place in the future, including the impact of such orders on our business operations and the business operations of the third parties on which we rely; risks and uncertainties associated with Neurocrine Biosciences' business and finances in general, as well as risks and uncertainties associated with the commercialization of ONGENTYS; whether ONGENTYS receives adequate reimbursement from third-party payors; the degree and pace of market uptake of ONGENTYS; risks and uncertainties relating to competitive products and technological changes that may limit demand for ONGENTYS; risks that additional regulatory submissions, for ONGENTYS or other product candidates, may not occur or be submitted in a timely manner; risks that the FDA or other regulatory authorities may make adverse decisions regarding ONGENTYS; risks that post-approval ONGENTYS commitments or requirements may be delayed; risks that ONGENTYS may be precluded from commercialization by the proprietary rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; risks and uncertainties relating to competitive products and technological changes that may limit demand for ONGENTYS; risks associated with the Company's dependence on BIAL for the commercial supply of, and manufacturing activities related to, ONGENTYS, and the ability of the Company to manage BIAL; and other risks described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's quarterly report on Form 10-Q for the quarter ended June 30, 2020. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof.

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    SOURCE Neurocrine Biosciences, Inc.

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  19. SAN DIEGO and CAMBRIDGE, Mass., Sept. 11, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) and Voyager Therapeutics, Inc. (NASDAQ:VYGR) today announced data from  PD-1101, a Phase Ib open-label, three-year efficacy and safety study, demonstrating that a one-time treatment with investigational gene therapy, NBIb-1817 (VY-AADC), showed sustained improvement in motor function including greater "On" time without troublesome dyskinesia, reduction in Unified Parkinson's Disease Rating Scale (UPDRS) Part III scores, and reduction in the amount of medications in patients with Parkinson's disease. In the PD-1101 study, NBIb-1817 reduced average "Off" time by up to -1.91 hours and improved average "On" time without troublesome dyskinesia…

    SAN DIEGO and CAMBRIDGE, Mass., Sept. 11, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) and Voyager Therapeutics, Inc. (NASDAQ:VYGR) today announced data from  PD-1101, a Phase Ib open-label, three-year efficacy and safety study, demonstrating that a one-time treatment with investigational gene therapy, NBIb-1817 (VY-AADC), showed sustained improvement in motor function including greater "On" time without troublesome dyskinesia, reduction in Unified Parkinson's Disease Rating Scale (UPDRS) Part III scores, and reduction in the amount of medications in patients with Parkinson's disease. In the PD-1101 study, NBIb-1817 reduced average "Off" time by up to -1.91 hours and improved average "On" time without troublesome dyskinesia by up to +2.23 hours in patients with advanced Parkinson's disease after three years across three cohorts. In addition, 14 out of 15 patients treated with NBIb-1817 continued to show an improvement in disease staging after three years, as assessed by the modified Hoehn & Yahr scale. These new data, along with two-year data from another open-label Phase Ib trial, PD-1102, were presented today at the MDS Virtual Congress 2020, September 12–16, 2020 (www.mdscongress.org/Congress/Registration.htm).

    In data from the three-year PD-1101 trial, the one-time treatment with NBIb-1817 showed sustained reduction in diary "Off" time by an average of -0.15 to -1.91 hours (baseline 4.28 to 4.93 hours) and improved diary "On" time without troublesome dyskinesia by an average of +0.26 to +2.23 hours (baseline 10.32 to 10.46 hours) across the cohorts as reported by 15 patients with advanced Parkinson's disease. NBIb-1817 also showed sustained improvement in motor function after three years, as measured by UPDRS Part III off medication scores, by -10.2 to -19.0 points (baseline 35.8 to 38.2 points) across the cohorts, per clinician assessment. Requirements for Parkinson's disease medications were also reduced in cohorts 2 and 3 (daily levodopa-equivalent dose reductions, average of -322.0 and -441.2 mg/day, respectively; baseline 1507.0 and 1477.0 mg/day, respectively). Two-year data from the PD-1102 trial for 7 patients showed that NBIb-1817 reduced diary "Off" time by an average of -3.2 hours and increased diary good "On" time by +2.1 hours (baselines 9.3 hours and 6.6 hours, respectively). In this study, NBIb-1817 showed sustained improvement in motor function after two years, with improved UPDRS Part III off medication scores of -12.0 points (baseline 34.4). Requirements for Parkinson's disease medications were also reduced (daily levodopa-equivalent dose reduction, average of -439.5 mg/day; baseline 1500.9 mg/day). Preliminary safety data from both studies suggest that NBIb-1817 was well-tolerated, with no study drug-related serious adverse events (SAEs) reported. The most common adverse events reported were headache, hypoesthesia, and musculoskeletal pain (PD-1101), and upper respiratory tract infection, headache, nausea, and depression (PD-1102). 

    "It is promising to see that after three years, a single administration of one-time investigational gene therapy treatment NBIb-1817 showed sustained reduction in 'Off' time, as well as improvement in 'On' time without troublesome dyskinesia and other measures of motor function in patients with Parkinson's disease," said Chad Christine, M.D., primary author, a lead investigator of the study and Professor of Neurology at the University of California, San Francisco (UCSF) Weill Institute for Neurosciences. "Parkinson's disease patients' motor function would be expected to worsen over three years, making these results very encouraging. The standard of care for advanced Parkinson's disease has not significantly changed in decades and it is our hope that NBIb-1817 has the potential to become the first gene therapy for Parkinson's disease."

    Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disorder that affects approximately one million people in the U.S. and six million people worldwide. It is characterized by a loss of dopamine from neuronal degeneration, with a concomitant loss of the aromatic L-amino acid decarboxylase (AADC) enzyme required to synthesize dopamine in the brain, leading to associated impairment in motor, neuropsychiatric, and autonomic functions.

    "We are pleased that the results from these studies show that one-time treatment with investigational NBIb-1817 may help restore the brain's ability to convert levodopa into dopamine," said Eiry Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences. "Our hope is that NBIb-1817 will help patients experience less 'Off' time and more 'On' time and improve motor symptom control. We plan to re-initiate enrollment in our registrational RESTORE-1 clinical trial with NBIb-1817 this year and look forward to further evaluating NBIb-1817 in patients with Parkinson's disease."

    NBIb-1817 is an investigational recombinant adeno-associated viral serotype 2 vector encoding the gene for human AADC that is designed to help produce the AADC enzyme in brain cells where it can convert levodopa to dopamine.

    "We are encouraged by the congruence of long-term data, including clinician- and patient-reported clinical outcomes in our clinical studies," said Omar Khwaja, M.D., Ph.D., Chief Medical Officer and Head of Research and Development at Voyager Therapeutics. "These results are promising and show that the approach has the potential to transform the treatment of Parkinson's disease, and help improve the lives of patients and their families."

    Additional information about PD-1101 and PD-1102 will be available on demand for registered participants through October 1, 2020 on the MDS meeting website (www.mdscongress.org/Congress/Registration.htm).

    • Christine CW, Richardson RM, Van Laar AD, et al. Three-Year Safety and Clinical Outcomes from the PD-1101 Trial of AADC Gene Therapy for Advanced Parkinson's Disease

      Poster # 879: Update on Genetics of Movement Disorders, September 13, 2020, 10:30–12:30pm EST (10-minute prerecorded presentation)



    • Factor SA, Van Laar AD, Richardson RM, et al. AADC Gene Therapy Administered via a Posterior Approach: 24-Month Results from the PD-1102 Trial in Advanced Parkinson's Disease

      Poster # 889: Poster Tour, launches on-demand on September 11, 2020 8:00am EST (5-minute prerecorded presentation)

    About Parkinson's Disease and NBIb-1817 (VY-AADC)

    Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disease that affects approximately one million people in the U.S. and six million people worldwide. It is characterized by a loss of dopamine and neuronal degeneration with a concomitant loss of the aromatic L-amino acid decarboxylase (AADC) enzyme required to synthesize dopamine in the brain, leading to associated impairment in motor, neuropsychiatric, and autonomic functions. Dopamine is a chemical "messenger" that is produced in the brain and is involved in the control of movement. It is made when AADC converts the chemical levodopa to dopamine. As Parkinson's disease progresses, there is less AADC enzyme in parts of the brain where levodopa is converted to dopamine.

    NBIb-1817 is an investigational recombinant adeno-associated viral (AAV) serotype 2 vector encoding the gene for human AADC that is designed to help produce the AADC enzyme in brain cells where it can convert levodopa to dopamine. NBIb-1817 is administered into the brain using intraoperative monitoring with magnetic resonance imaging (MRI)-facilitated targeted delivery.

    About the RESTORE-1 Clinical Trial

    Paused temporarily in April 2020 due to the COVID-19 pandemic, Neurocrine Biosciences and Voyager Therapeutics plan to re-initiate RESTORE-1, a Phase 2, randomized, placebo-surgery controlled, double-blinded, multi-center clinical trial, to evaluate the safety and efficacy of NBIb-1817 in patients who have been diagnosed with Parkinson's disease for at least four years and have at least three hours of "Off" time during the day as measured by a validated self-reported patient diary.

    For more information about the RESTORE-1 clinical trial, including eligibility criteria, please visit clinicaltrials.gov and restore1study.com.

    About the RESTORE-2 Clinical Trial

    Preparations are ongoing for the RESTORE-2 global registrational trial that will include clinical sites within and outside the U.S.

    About Neurocrine Biosciences and Voyager Therapeutics Strategic Collaboration

    In 2019, Neurocrine Biosciences and Voyager Therapeutics entered into a strategic collaboration focused on the development and commercialization of gene therapy programs, VY-AADC for Parkinson's disease and VY-FXN01 for Friedreich's ataxia, as well as rights to two programs to be determined. This collaboration combines Neurocrine Biosciences' expertise in neuroscience, drug development and commercialization with Voyager's innovative gene therapy programs targeting severe neurological diseases.

    About Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis* and uterine fibroids*, with three pivotal and five mid-stage clinical programs in multiple therapeutic areas. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*in collaboration with AbbVie)

    About Voyager Therapeutics

    Voyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyager's wholly owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinson's disease, Huntington's disease, Friedreich's ataxia, and other severe neurological diseases. For more information on Voyager, please visit the company's website at www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and LinkedIn.

    Voyager Therapeutics® is a registered trademark of Voyager Therapeutics.

    Neurocrine Biosciences Forward-Looking Statements

    In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. Among the factors and risks that could cause actual results to differ materially from those indicated in the forward-looking statements are risks that the product candidates licensed from Voyager may not obtain regulatory approval from the FDA or other regulatory agencies, or such approval may be delayed or conditioned; risks that development activities related to the product candidates licensed from Voyager may not be completed on time or at all; risks associated with the Company's dependence on Voyager for research, development and manufacturing activities; risks that ongoing or future clinical trials may not be successful or replicate previous clinical trial results, or may not be predictive of real-world results or of results in subsequent clinical trials; risks and uncertainties relating to competitive products and technological changes that may limit demand for product candidates licensed from Voyager; risks that the product candidates licensed from Voyager may be precluded from commercialization by the proprietary rights of third parties; the impact of the COVID-19 pandemic and efforts to mitigate its spread on our business; risks and uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risk and uncertainties related to any COVID-19 quarantines, shelter-in-place and similar government orders that are currently in place or that may be put in place in the future, including the impact of such orders on our business operations and the business operations of the third parties on which we rely; risks related to the development of our product candidates; and other risks that are described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's quarterly report on Form 10-Q for the quarter ended June 30, 2020. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof.

    Voyager Therapeutics Forward-Looking Statements

    This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "may," "might," "will," "would," "should," "expect," "plan," "anticipate," "believe," "estimate," "undoubtedly," "project," "intend," "future," "potential," or "continue," and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager Therapeutics makes regarding the potential impact or significance of the long-term medical data for patients treated in the PD-1101 and PD-1102 clinical trials; the re-initiation of RESTORE-1 Phase 2 clinical trial prior to year-end, the initiation of the RESTORE-2 Phase 3 clinical trial during the first half of 2021; the initiation, timing, progress, activities, goals and reporting of results of other activities associated with the PD program, and the potential benefits, timing and future operation of the collaboration with Neurocrine Biosciences are forward looking. All forward-looking statements are based on estimates and assumptions by Voyager's management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, risks that ongoing or future clinical trials may not be successful or replicate previous clinical trial results, or may not be predictive of real-world results or of results in subsequent clinical trials; risks and uncertainties relating to competitive products and technological changes that may limit demand for product candidates now being evaluated in clinical trials; the impact of the COVID-19 pandemic and efforts to mitigate its spread on our clinical trials and our business generally; risks related to the initiation and conduct of preclinical studies and clinical trials; the sufficiency of preclinical and clinical data to support applications for additional studies and marketing approval of our PD drug development candidates; changes in expectations from the FDA and other regulatory authorities as to the requirements for obtaining product approvals; the decisions of the FDA and other regulatory authorities in response to applications we file in connection with our product candidates under our PD program and otherwise in our conduct of PD drug development activities; the priorities, capabilities, diligence and efforts of Neurocrine Biosciences, our collaboration partner for the PD program, and other collaborators and vendors supporting our PD program; and the commercial potential of PD product candidates that may be developed as part of our PD program. These statements are also subject to a number of material risks and uncertainties that are described in Voyager Therapeutics' Annual Report on Form 10K, Voyager Therapeutics' Quarterly Reports on Form 10-Q and other reports filed by Voyager Therapeutics with the Securities and Exchange Commission, as may be updated by its subsequent filings with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager Therapeutics undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

     

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    SOURCE Neurocrine Biosciences, Inc.

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  20. SAN DIEGO, Sept. 8, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) will present at the Morgan Stanley 18th Annual Global Healthcare Conference at 1:30 p.m. ET on Monday, September 14, 2020. Kevin Gorman, Chief Executive Officer, and Matt Abernethy, Chief Financial Officer, will present at the conference.

    The live presentation will be webcast and may be accessed on the Company's website under Investors at www.neurocrine.com. A replay of the presentation 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 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis* and uterine fibroids*, with three pivotal and five mid-stage clinical programs in multiple therapeutic areas. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn(*in collaboration with AbbVie)

     

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    SOURCE Neurocrine Biosciences, Inc.

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  21. SAN DIEGO, Sept. 3, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced it will present new data as part of 30 abstracts accepted from its movement disorder programs for tardive dyskinesia and Parkinson's disease at the upcoming virtual scientific meetings, 2020 Psych Congress on September 10–13 and the MDS Virtual Congress 2020 on September 12–16.

    Key highlights include data analyses from long-term Phase III studies of INGREZZA® (valbenazine) capsules in adult patients with tardive dyskinesia demonstrating sustained clinical benefit, safety and tolerability; new Phase III data analyses of ONGENTYS® (opicapone) capsules as an add-on therapy to levodopa/carbidopa in patients with Parkinson's disease experiencing motor "off" episodes, and new Phase Ib data of an investigational gene therapy, NBIb-1817 (VY-AADC), evaluating the three-year safety and clinical outcomes of patients with advanced Parkinson's disease.

    "We are excited to present new data at this year's Psych Congress and MDS that illustrate the depth and breadth of our movement disorder programs for tardive dyskinesia and Parkinson's disease in clinical research," said Eiry W. Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences. "These data for INGREZZA, ONGENTYS and our investigational gene therapy NBIb-1817 underscore our ongoing commitment to improve the lives of patients living with these movement disorders."

    2020 Psych Congress

    INGREZZA® (valbenazine) Capsules

    • Onset and Resolution of Key Adverse Events in Valbenazine-Treated Patients with Tardive Dyskinesia: Pooled Post-Hoc Analyses from Two Long-Term Clinical Trials (Poster # 179)
    • Item 8 of the Abnormal Involuntary Movement Scale (AIMS) as an Overall Index of Improvement in Patients with Tardive Dyskinesia (Poster # 166)
    • Clinician-Reported Patient Awareness of Symptoms and Severity of Tardive Dyskinesia in Patients Prescribed VMAT2 Inhibitors (Poster # 122)
    • Concerns from Community Mental Health Center (CMHC) Patients Regarding Drug-Induced Movement Disorders: Impact on Functioning and Treatment Beliefs (Poster # 126)
    • Understanding the Evolving Continuing Medical Education Needs of Physicians Managing Patients with TD (Poster # 212)
    • Knowledge of the Recognition and Management of Tardive Dyskinesia Markedly Improved Among Psychiatrists: Assessing the Impact of Online Medical Education (Poster # 167)
    • TD360: Outcomes of a Tardive Dyskinesia Educational Curriculum – A Focus on Quality of Life, Increased Detection and Novel Treatments (Poster # 159)

    MDS Virtual Congress 2020

    INGREZZA® (valbenazine) Capsules – Tardive Dyskinesia

    • Real-World Evaluation of Patient Characteristics and Disease Management in Long-Term Valbenazine Treatment in Adults with Tardive Dyskinesia (Poster # 99)
    • Real-World Use and Impact of VMAT2 Inhibitors in Patients with Tardive Dyskinesia (Poster # 102)
    • Patient Perspective of Tardive Dyskinesia: Results from a Social Media Listening Study (Poster # 101)

    ONGENTYS® (opicapone) Capsules – Parkinson's Disease (in collaboration with BIAL)

    • Opicapone as First-Line Adjunctive Levodopa Treatment in Parkinson's Disease Patients with Motor Fluctuations: Findings from BIPARK-I and BIPARK-II Combined Post-Hoc Analysis (Poster # 999)
    • Opicapone's Added Benefit as a First-Line Adjunctive Therapy to Levodopa and When Used Promptly in the Motor Fluctuations Spectrum of Parkinson's Disease: a Post-Hoc Analysis of BIPARK-I and BIPARK-II (Poster # 994)
    • Efficacy and Safety of Opicapone in Parkinson's Disease Patients According to Duration of Motor Fluctuations: Post-Hoc Analysis of BIPARK-I and BIPARK-II (Poster # 993)
    • Super-Responders to Opicapone Adjunct Treatment to Levodopa in Parkinson's Disease Patients with Motor Fluctuations: Combined Post-Hoc Analysis of BIPARK-I and BIPARK-II (Poster # 974)
    • Efficacy of Opicapone in Different Levodopa-Containing Treatment Regimens in Parkinson's Disease Patients with Motor Fluctuations (Poster # 973)
    • Opicapone in Clinical Practice in Parkinson's Disease Patients with Motor Fluctuations: Findings from the OPTIPARK Study (Poster # 1049)
    • Onset of Drug-Related Adverse Events in Parkinson's Disease Patients with Motor Fluctuations Treated with Opicapone in Clinical Practice: OPTIPARK Post-Hoc Analysis (Poster # 1029)
    • Effect of Opicapone and Entacapone on Daily Pattern of Motor Fluctuations in Parkinson's Disease Patients (Poster # 1027)
    • Effect of Opicapone and Entacapone on Levodopa Short Duration Response (Poster # 1030)
    • Effect of Opicapone and Entacapone on Early Morning-OFF Pattern in Parkinson's Disease Patients with Motor Fluctuations (Poster # 1071)
    • Efficacy and Safety/Tolerability of Opicapone in Catechol-O-Methyltransferase Inhibitor-Naïve Parkinson's Disease Patients Recently Diagnosed with Motor Fluctuations (Poster # 1028)
    • Efficacy of Opicapone Compared to Entacapone in Catechol-O-Methyltransferase Inhibitor-Naïve Parkinson's Disease Patients Recently Diagnosed with Motor Fluctuations: a Post-Hoc Conservative Analysis (Poster # 998)
    • Motor Fluctuations in a Real-World Sample of Patients with Parkinson's Disease (Poster # 343)
    • Real-World Healthcare Resource Utilization in Patients with Parkinson's Disease and Motor Fluctuations (Poster # 344)
    • Patient Characteristics, Treatment Patterns and Disease Burden in People with Parkinson's Disease: Insights from the Parkinson's Disease Real-World Impact Assessment (PRISM) Study (Poster # 1128)
    • Impulse Control Behaviours in People with Parkinson's Disease: Findings from the Parkinson's Disease Real-World Impact Assessment (PRISM) Study (Poster # 695)
    • Burden of Care-Partners of People with Parkinson's Disease: Findings from Parkinson's Disease Real-World Impact Assessment (PRISM) Study (Poster # 1143)
    • Characterization of the Pattern of Daily Motor Fluctuations in Parkinson's Disease Patients Based on Home Diaries (Poster # 1011)

    Investigational NBIb-1817- Parkinson's Disease (in collaboration with Voyager)

    • Three-Year Safety and Clinical Outcomes from the PD-1101 Trial of AADC Gene Therapy for Advanced Parkinson's Disease – Poster # 879: Update on Genetics of Movement Disorders, September 13, 2020, 10:30–12:30pm EST (10-minute prerecorded presentation)
    • AADC Gene Therapy Administered via a Posterior Approach: 24-month Results from the PD-1102 Trial in Advanced Parkinson's Disease – Poster # 889: Poster Tour, launches on-demand on September 11, 2020 8:00am EST (5-minute prerecorded presentation)

    About Tardive Dyskinesia (TD)

    Tardive dyskinesia (TD) is a movement disorder that is characterized by uncontrollable, abnormal and repetitive movements of the face, torso and/or other body parts, which may be disruptive and negatively impact patients. The condition is caused by prolonged use of treatments that block dopamine receptors in the brain, such as antipsychotics commonly prescribed to treat mental illnesses such as schizophrenia, bipolar disorder and depression, and certain anti-nausea medications. In patients with TD, these treatments are thought to result in irregular dopamine signaling in a region of the brain that controls movement. The symptoms of TD can be severe and are often persistent and irreversible. TD is estimated to affect at least 500,000 people in the U.S.

    About INGREZZA® (valbenazine) Capsules

    INGREZZA, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is the first FDA-approved product indicated for the treatment of adults with tardive dyskinesia, a condition associated with uncontrollable, abnormal and repetitive movements of the face, torso and/or other body parts.

    INGREZZA is thought to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function, helping to regulate nerve signaling in adults with tardive dyskinesia. VMAT2 is a protein in the brain that packages neurotransmitters, such as dopamine, for transport and release in presynaptic neurons. INGREZZA, developed in Neurocrine Biosciences's laboratories, is novel in that it selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic, or muscarinic receptors. Additionally, INGREZZA can be taken for the treatment of tardive dyskinesia as one capsule, once-daily, together with psychiatric medications such as antipsychotics or antidepressants.

    Important Information

    Approved Use

    INGREZZA® (valbenazine) capsules is a prescription medicine used to treat adults with movements in the face, tongue, or other body parts that cannot be controlled (tardive dyskinesia).

    It is not known if INGREZZA is safe and effective in children.

    Important Safety Information

    Do not take INGREZZA if you are allergic to valbenazine, or any of the ingredients in INGREZZA.

    INGREZZA may cause serious side effects, including:

    • Sleepiness (somnolence). Do not drive, operate heavy machinery, or do other dangerous activities until you know how INGREZZA affects you.
    • Heart rhythm problems (QT prolongation). INGREZZA may cause a heart problem known as QT prolongation.
    • Symptoms of QT prolongation may include: fast, slow, or irregular heartbeat, shortness of breath, dizziness or fainting.
    • Parkinson-like symptoms. Symptoms include: shaking, body stiffness, trouble moving or walking, or keeping your balance.

    Tell your healthcare provider right away if you have a change in your heartbeat (a fast or irregular heartbeat), or if you faint.

    Before taking INGREZZA, tell your healthcare provider about all of your medical conditions including if you: have liver or heart 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.

    The most common side effect of INGREZZA is sleepiness (somnolence). Other side effects include changes in balance (balance problems, dizziness) or an increased risk of falls, headache, feelings of restlessness, dry mouth, constipation, and blurred vision.

    These are not all of the possible side effects of INGREZZA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

    Please see INGREZZA full Product Information.

    About Parkinson's Disease

    Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disorder that affects approximately one million people in the United States and six million people worldwide. Parkinson's disease is caused by low dopamine levels produced in the brain. Dopamine helps transmit signals between the areas of the brain that control all purposeful movements, including talking, walking and writing. As Parkinson's disease progresses, dopamine production steadily decreases, resulting in increased problems with motor symptoms including slowed movement (bradykinesia), tremor, rigidity, impaired posture and balance, and difficulty with speech and writing.

    There is presently no cure for Parkinson's disease and management of the disease consists of the use of treatments that attempt to control motor symptoms primarily through dopaminergic mechanisms. The current gold standard for treatment of motor symptoms is levodopa/carbidopa. While levodopa/carbidopa improves patients' motor symptoms, as the disease progresses, the beneficial effects of levodopa begin to wear off more quickly. Patients then experience motor fluctuations throughout the day between "on" time, periods when the medication is working and Parkinson's disease symptoms are controlled, and "off" time, when the medication is not working and motor symptoms return.

    About ONGENTYS® (opicapone) Capsules 

    ONGENTYS is a novel, once-daily, oral, peripheral, selective and reversible catechol-O-methyltransferase (COMT) inhibitor approved by the U.S. Food and Drug Administration (FDA) as an add-on treatment to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes. ONGENTYS inhibits the COMT enzyme, which breaks down levodopa, making more levodopa available to reach the brain.

    In June 2016, BIAL – Portela & CA, S.A. (BIAL) received approval from the European Commission for ONGENTYS as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors in adult patients with Parkinson's disease and end-of-dose motor fluctuations who cannot be stabilized on those combinations. BIAL currently markets ONGENTYS in Germany, United Kingdom, Spain, Portugal and Italy. Neurocrine Biosciences in-licensed opicapone from BIAL in 2017 and has exclusive development and commercialization rights in the U.S. and Canada.

    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 of 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 NBIb-1817 (VY-AADC)

    Dopamine is a chemical "messenger" that is produced in the brain and is involved in the control of movement. It is made in the brain when the enzyme AADC (Aromatic l-amino acid decarboxylase) converts the chemical levodopa to dopamine. As Parkinson's disease progresses, there is less AADC enzyme in parts of the brain where levodopa is converted to dopamine. This may be associated with patients' motor function worsening with less predictable response to medications.

    NBIb-1817 is an investigational recombinant adeno-associated viral (AAV) vector serotype 2 encoding the gene for human AADC that is designed to produce the AADC enzyme in brain cells where it can convert levodopa to dopamine. NBIb-1817 is infused into the putamen using intraoperative monitoring with magnetic resonance imaging (MRI)-facilitated targeted delivery.

    In January 2019, Neurocrine Biosciences and Voyager Therapeutics announced a strategic collaboration focused on the development and commercialization of this and other gene therapy programs.

    About Neurocrine Biosciences

    Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis* and uterine fibroids*, with three pivotal and five mid-stage clinical programs in multiple therapeutic areas. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*in collaboration with AbbVie)

    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 Neurocrine's products and product candidates. 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 associated with the commercialization of INGREZZA and our other products; risks that the launch of ONGENTYS may be delayed; the impact of the COVID-19 pandemic and efforts to mitigate its spread on our business; risks and uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risk and uncertainties related to any COVID-19 quarantines, shelter-in-place and similar government orders that are currently in place or that may be put in place in the future, including the impact of such orders on our business operations and the business operations of the third parties on which we rely; risks related to the development of our product candidates; risks associated with our dependence on third parties for development and manufacturing activities related to INGREZZA and our 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 associated with our dependence on BIAL for manufacturing activities for ONGENTYS, and our ability to manage BIAL; risks that clinical development activities may not be completed on time or at all, or may be delayed for regulatory, manufacturing, COVID-19 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; and other risks described in our periodic reports filed with the SEC, including without limitation our quarterly report on Form 10-Q for the quarter ended June 30, 2020. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof.

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/neurocrine-biosciences-to-present-new-data-from-its-movement-disorder-programs-for-tardive-dyskinesia-and-parkinsons-disease-at-upcoming-scientific-meetings-301124175.html

    SOURCE Neurocrine Biosciences, Inc.

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  22. SAN DIEGO, Sept. 2, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) will present at the Citi 15th Annual BioPharma Virtual Conference at 12:35 p.m. ET on Wednesday, September 9, 2020. Kevin Gorman, Chief Executive Officer, will present at the conference.

    The live presentation will be webcast and may be accessed on the Company's website under Investors at www.neurocrine.com. A replay of the presentation 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 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis* and uterine fibroids*, with three pivotal and five mid-stage clinical programs in multiple therapeutic areas. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn(*in collaboration with AbbVie)

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/neurocrine-biosciences-to-present-at-the-citi-15th-annual-biopharma-virtual-conference-301123291.html

    SOURCE Neurocrine Biosciences, Inc.

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  23. SAN DIEGO, Aug. 3, 2020 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced its financial results for the second quarter ended June 30, 2020 and provided revised full-year 2020 financial expense guidance.

    "I want to thank healthcare providers and our employees for the perseverance they showed during the second quarter to ensure patients had uninterrupted access to INGREZZA under the challenging circumstances caused by the COVID-19 pandemic. As we move into the second half of 2020, we remain focused on improving the diagnosis and treatment rates for people with tardive dyskinesia, preparing to make ONGENTYS available to people living with Parkinson's disease, and advancing our programs in clinical development," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "With four U.S. FDA approved treatments that address four unique patient populations and a diverse and expanding pipeline, Neurocrine Biosciences is well positioned to be a leading neuroscience-focused biopharmaceutical company."

    Financial Highlights



    Three Months Ended

    June 30,



    Six Months Ended

    June 30,

    (unaudited, in millions, except per share data)

    2020



    2019



    2020



    2019

    Revenues:















    INGREZZA product sales, net

    $

    267.6





    $

    180.5





    $

    498.7





    $

    316.9



    Collaboration revenue

    34.8





    3.0





    40.8





    5.0



    Total revenues

    $

    302.4





    $

    183.5





    $

    539.5





    $

    321.9



















    GAAP Research and Development (R&D)

    $

    80.9





    $

    61.7





    $

    139.2





    $

    99.4



    Non-GAAP R&D

    $

    51.0





    $

    45.7





    $

    101.6





    $

    78.0



















    GAAP Selling, General and Administrative (SG&A)

    $

    96.5





    $

    80.8





    $

    214.3





    $

    168.3



    Non-GAAP SG&A

    $

    76.9





    $

    68.9





    $

    179.6





    $

    146.0



















    GAAP net income (loss)

    $

    79.6





    $

    51.3





    $

    117.0





    $

    (50.8)



    GAAP net income (loss) per share – diluted

    $

    0.81





    $

    0.54





    $

    1.20





    $

    (0.56)



















    Non-GAAP net income

    $

    139.1





    $

    67.2





    $

    218.2





    $

    94.9



    Non-GAAP net income per share – diluted

    $

    1.42





    $

    0.71





    $

    2.24





    $

    1.00



















    (unaudited, in millions)









    June 30,

    2020



    December 31,

    2019

    Total cash and cash equivalents and debt securities available-for-sale

    $

    1,143.5





    $

    970.2



     

    Second Quarter Net Product Sales Highlights:

    • INGREZZA net product sales for the second quarter of 2020 were $268 million, representing a year-over-year increase of 48%.
    • Continued strength in refill and persistency rates for existing INGREZZA patients.
    • End of second quarter 2020 days-on-hand channel inventory increased relative to end of first quarter 2020, resulting in an approximate $12 million benefit to net product sales.

    Financial Highlights:

    • Second quarter 2020 GAAP net income and diluted earnings per share were approximately $80 million and $0.81, respectively, compared with approximately $51 million and $0.54, respectively, in the second quarter of 2019, primarily driven by higher INGREZZA sales offset by higher in-process Research and Development (IPR&D) costs and operating expenses.
    • Second quarter 2020 non-GAAP net income and diluted earnings per share were approximately $139 million and $1.42, respectively, compared with approximately $67 million and $0.71, respectively, in the second quarter of 2019 driven by higher INGREZZA sales.
    • Research and Development (R&D) expense increased in the second quarter of 2020 versus the second quarter of 2019, primarily due to milestone payments to BIAL associated with the approval of ONGENTYS and increased headcount costs.
    • Selling, General and Administrative (SG&A) expense increased in the second quarter of 2020 versus the second quarter of 2019, primarily due to increased headcount costs.
    • At June 30, 2020, the Company had cash, cash equivalents and debt securities available-for-sale of $1.1 billion.

    A reconciliation of GAAP to non-GAAP quarterly financial results can be found in Table 3 at the end of this earnings release.

    Recent Events

    • In April 2020, the FDA approved ONGENTYS® (opicapone), the first and only once-daily COMT inhibitor, as an adjunctive treatment to levodopa/carbidopa in patients with Parkinson's disease experiencing "off" episodes – periods of time when motor symptoms such as tremor, slowed movement and difficulty walking occur. ONGENTYS also increases "on" time without troublesome dyskinesia, the time when the motor symptoms of a patient with Parkinson's disease are better controlled. The FDA approval of ONGENTYS for Parkinson's disease triggered a $20 million milestone payment to BIAL. The commercial launch of ONGENTYS is expected to occur later in 2020.
    • In May 2020, AbbVie received approval from the FDA for ORIAHNNTM (elagolix, estradiol, and norethindrone acetate capsules; elagolix capsules) for the management of heavy menstrual bleeding associated with uterine fibroids in pre-menopausal women. FDA approval for ORIAHNN for uterine fibroids resulted in the achievement of a $30 million milestone. The Company will receive royalties at tiered percentage rates on net sales of ORIAHNN.
    • In May 2020, the Company exercised its option with Idorsia Pharmaceuticals Ltd. paying $45 million to license the global rights to NBI-827104 (ACT-709478), a potent, selective, orally active and brain penetrating T-type calcium channel blocker, in clinical development for the treatment of a rare pediatric epilepsy. The option also included a research collaboration to discover novel T-type calcium channel blockers.
    • In June 2020, the Company reported positive Phase II data for crinecerfont in adults with congenital adrenal hyperplasia (CAH) and highlighted the resumption of enrollment in the Phase IIa pediatric study in adolescents with classic CAH. In July 2020, the Company initiated the CAHtalyst Study (www.cahtalyststudy.com), a single, global registrational study of crinecerfont in adult patients with classic CAH.
    • In June 2020, the Company entered an exclusive license with Takeda Pharmaceutical Company Limited, or Takeda, for the right to develop and commercialize certain compounds in Takeda's early-to-mid-stage psychiatry pipeline. Specifically, Takeda granted the Company an exclusive license to seven pipeline programs, including three clinical-stage assets for negative effects of schizophrenia, treatment-resistant depression, and anhedonia. The agreement became effective in July 2020, upon expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, at which time the Company paid $120 million upfront (minus an earnest money deposit already paid by the Company to Takeda) to gain the exclusive license.

    Full-Year 2020 Revised Expense Guidance



    Range

    (in millions)

    Low



    High

    Combined GAAP R&D and SG&A expenses

    $

    850





    $

    900



    Combined Non-GAAP R&D and SG&A expenses

    $

    570





    $

    610



     

    • Previously, the Company expected combined GAAP R&D and SG&A expenses in the range of $675 million to $725 million and combined non-GAAP R&D and SG&A expenses in the range of $550 million to $600 million.
    • The $175 million increase in GAAP expense guidance range primarily reflects $45 million paid to Idorsia upon exercising the option to license the global rights to NBI-827104 (ACT-709478) and $120 million paid to Takeda for the exclusive license for the right to develop and commercialize certain compounds in Takeda's early-to-mid-stage psychiatry pipeline.
    • GAAP-only guidance includes approximately $105 million of share-based compensation. GAAP-only guidance does not include any other potential milestones or in-process research and development costs associated with current collaborations or potential future business development activities.

    Conference Call and Webcast Today at 4:30 PM Eastern Time

    Neurocrine Biosciences will hold a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). Participants can access the live conference call by dialing 877-876-9173 (US) or 785-424-1667 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine Biosciences' website under Investors at www.neurocrine.com. 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 28 years of experience discovering and developing life-changing treatments for people with serious, challenging and under-addressed neurological, endocrine and psychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, Parkinson's disease, endometriosis* and uterine fibroids*, with three pivotal and five mid-stage clinical programs in multiple therapeutic areas. Headquartered in San Diego, Neurocrine Biosciences specializes in targeting and interrupting disease-causing mechanisms involving the interconnected pathways of the nervous and endocrine systems. For more information, visit neurocrine.com, and follow the company on LinkedIn. (*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 certain non-GAAP financial measures. When preparing these supplemental 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, the non-GAAP measures exclude: milestones received from licenses and collaborations, milestones paid related to licenses and collaborations, non-cash collaboration revenue, acquired in-process research and development, share-based compensation expense, non-cash interest expense related to convertible debt, changes in fair value of equity security investments and certain adjustments to income tax expense. These non-GAAP 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 research and development and sales, general, and administrative expenses on both a GAAP and a non-GAAP basis. The guidance regarding GAAP research and development expenses and sales, general and administrative expenses does not include estimates for expenses associated with any potential future business development activities. A reconciliation of the 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; the timing of our launch of ONGENTYS; our financial and operating performance, including our future expenses; our collaborative partnerships; expectations regarding the impact of COVID-19 on our business; expectations regarding our ability to adapt our business to the evolving COVID-19 pandemic, mitigate its impact on our business and maintain business continuity, including our ability to protect the safety and well-being of our employees, to continue to support uninterrupted supply of INGREZZA, including patient and healthcare provider access to INGREZZA, to continue our ongoing clinical trials and other development activities, and to otherwise advance our business objectives; and the timing of 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 associated with the commercialization of INGREZZA and our other products; risks that the launch of ONGENTYS may be delayed; the impact of the COVID-19 pandemic and efforts to mitigate its spread on our business; risks and uncertainties associated with the scale and duration of the COVID-19 pandemic and resulting global, national, and local economic and financial disruptions; risk and uncertainties related to any COVID-19 quarantines, shelter-in-place and similar government orders that are currently in place or that may be put in place in the future, including the impact of such orders on our business operations and the business operations of the third parties on which we rely; risks related to the development of our product candidates; risks associated with our dependence on third parties for development and manufacturing activities related to INGREZZA and our 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 associated with our dependence on AbbVie for the commercialization of ORILISSA and ORIAHNN, as well as the continued development of elagolix; risks associated with our dependence on BIAL for manufacturing activities for ONGENTYS, and our ability to manage BIAL; risks that clinical development activities may not be completed on time or at all, or may be delayed for regulatory, manufacturing, COVID-19 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; and other risks described in our periodic reports filed with the SEC, including without limitation our quarterly report on Form 10-Q for the quarter ended June 30, 2020. Neurocrine disclaims any obligation to update the statements contained in this press release after the date hereof.

    This press release refers to certain non-GAAP financial measures. These non-GAAP financial measures should not be considered replacements for, and should be read together with, the most comparable GAAP financial measures. Reconciliations of non-GAAP financial results to the most directly comparable GAAP financial results are included at the end of this press release, which has been filed with the SEC in a Current Report on Form-8-K dated as of event date herewith. In addition, Neurocrine provides guidance regarding combined research and development and sales, general and administrative expenses on both a GAAP and non-GAAP basis.

     

    TABLE 1



    NEUROCRINE BIOSCIENCES, INC.

    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

    (unaudited)





    Three Months Ended

    June 30,



    Six Months Ended

    June 30,

    (in millions, except per share data)

    2020



    2019



    2020



    2019

    Revenues:















    Product sales, net

    $

    267.6





    $

    180.5





    $

    498.7





    $

    316.9



    Collaboration revenue

    34.8





    3.0





    40.8





    5.0



    Total revenues

    302.4





    183.5





    539.5





    321.9



    Operating expenses:















    Cost of sales

    2.4





    1.6





    4.5





    2.7



    Research and development

    80.9





    61.7





    139.2





    99.4



    Acquired in-process research and development

    46.0





    5.0





    46.0





    118.1



    Selling, general and administrative

    96.5





    80.8





    214.3





    168.3



    Total operating expenses

    225.8





    149.1





    404.0





    388.5



    Operating income (loss)

    76.6





    34.4





    135.5





    (66.6)



    Other income (expense):















    Interest expense

    (8.3)





    (7.9)





    (16.5)





    (15.8)



    Unrealized gain (loss) on equity securities

    11.3





    21.0





    (5.2)





    22.7



    Investment income and other, net

    3.6





    4.6





    8.3





    9.2



    Total other income (expense), net

    6.6





    17.7





    (13.4)





    16.1



    Income (loss) before provision for income taxes

    83.2





    52.1





    122.1





    (50.5)



    Provision for income taxes

    3.6





    0.8





    5.1





    0.3



    Net income (loss)

    $

    79.6





    $

    51.3





    $

    117.0





    $

    (50.8)



















    Net income (loss) per share, basic

    $

    0.86





    $

    0.56





    $

    1.26





    $

    (0.56)



    Net income (loss) per share, diluted

    $

    0.81





    $

    0.54





    $

    1.20





    $

    (0.56)



















    Weighted average common shares outstanding, basic

    93.0





    91.4





    92.8





    91.2



    Weighted average common shares outstanding, diluted

    98.2





    94.8





    97.6





    91.2



     

     

    TABLE 2



    NEUROCRINE BIOSCIENCES, INC.

    CONDENSED CONSOLIDATED BALANCE SHEETS

    (unaudited)



    (in millions)

    June 30,

    2020



    December 31,

    2019

    Cash and cash equivalents and debt securities available-for-sale

    $

    948.3





    $

    670.5



    Other current assets

    197.9





    160.5



    Total current assets

    1,146.2





    831.0



    Debt securities available-for-sale

    195.2





    299.7



    Right-of-use assets

    72.1





    74.3



    Equity securities

    50.7





    55.9



    Property and equipment, net

    44.6





    41.9



    Restricted cash and other long-term assets

    6.8





    3.2



    Total assets

    $

    1,515.6





    $

    1,306.0











    Convertible senior notes

    $

    419.5





    $

    408.8



    Other current liabilities

    153.5





    156.5



    Total current liabilities

    573.0





    565.3



    Operating lease liabilities

    84.3





    86.7



    Other long-term liabilities

    27.1





    17.1



    Stockholders' equity

    831.2





    636.9



    Total liabilities and stockholders' equity

    $

    1,515.6





    $

    1,306.0



     

     

    TABLE 3



    NEUROCRINE BIOSCIENCES, INC.

    RECONCILIATION OF GAAP TO NON-GAAP FINANCIAL RESULTS

    (unaudited)





    Three Months Ended

    June 30,



    Six Months Ended

    June 30,

    (in millions, except per share data)

    2020



    2019



    2020



    2019

    GAAP net income (loss)

    $

    79.6





    $

    51.3





    $

    117.0





    $

    (50.8)



    Adjustments:















    Milestones received from licenses and collaborations A

    (30.0)









    (30.0)







    Non-cash collaboration revenue B









    (1.3)







    Acquired in-process research and development (IPR&D) C

    46.0





    5.0





    46.0





    118.1



    Milestones paid related to licenses and collaborations - R&D

    20.0





    10.0





    20.0





    10.0



    Share-based compensation expense - R&D

    9.9





    6.0





    17.6





    11.4



    Share-based compensation expense - SG&A

    19.6





    11.9





    34.7





    22.3



    Non-cash interest related to convertible debt

    5.4





    5.1





    10.7





    10.0



    Changes in fair value of equity security investments D

    (11.3)





    (21.0)





    5.2





    (22.7)



    Income tax effect related to reconciling items E

    (0.1)





    (1.1)





    (1.7)





    (3.4)



    Non-GAAP net income

    $

    139.1





    $

    67.2





    $

    218.2





    $

    94.9



















    Net income (loss) per diluted common share: