NBIX Neurocrine Biosciences Inc.

96.16
-1.54  -2%
Previous Close 97.7
Open 97.52
52 Week Low 72.14
52 Week High 136.265
Market Cap $8,967,388,492
Shares 93,254,872
Float 92,057,975
Enterprise Value $8,666,500,994
Volume 823,126
Av. Daily Volume 773,051
Stock charts supplied by TradingView

Upcoming Catalysts

Drug Stage Catalyst Date
Valbenazine
Huntington's disease
Phase 3
Phase 3
Premium membership is required to view catalyst dates, analyst ratings, earnings dates and cash burn data. Click here to unlock and sign up to a 14-day FREE TRIAL.

Drug Pipeline

Drug Stage Notes
VY-AADC02 (RESTORE-1)
Parkinson's Disease
Phase 2
Phase 2
Phase 2 trial to re-enroll 2H 2020. Phase 3 trial to commence 1H 2021.
NBI-921352
SCN8A developmental and epileptic encephalopathy (SCN8A-DEE)
Phase 2
Phase 2
Phase 2 trial to commence 2H 2020.
ACT-709478 (NBI-827104)
Pediatric epilepsy
Phase 2
Phase 2
Phase 2 trial to commence 2H 2020.
Crinecerfont
Congenital Adrenal Hyperplasia (CAH) - children
Phase 2
Phase 2
Phase 2a trial ongoing.
TAK-831
Negative symptoms of schizophrenia
Phase 2
Phase 2
Phase 2 trial is ongoing.
TAK-041
Anhedonia in depression
Phase 2
Phase 2
Phase 2 trial planned.
TAK-653
Treatment-resistant depression
Phase 2
Phase 2
Phase 2 trial planned.
Crinecerfont (NBI-74788)
Congenital Adrenal Hyperplasia (CAH) - adults
Phase 2
Phase 2
Phase 3 trial planned for 2H 2020.
Elagolix
Uterine Fibroids
Approved
Approved
FDA approval announced May 29, 2020.
Opicapone
Parkinson's disease
Approved
Approved
FDA Approval announced April 27, 2020.
Elagolix
Polycystic ovary syndrome (PCOS)
Phase 2
Phase 2
Phase 2 trial initiated 3Q 2019.
INGREZZA - T-Force GOLD
Tourette syndrome - juvenile
Phase 2b
Phase 2b
Phase 2 top-line data released December 12, 2018 did not meet primary endpoint.
Elagolix
Endometriosis
Approved
Approved
FDA approval announced July 24, 2018.
INGREZZA
Tardive dyskinesia
Approved
Approved
Approved April 11, 2017.
INGREZZA
Tourette syndrome - adults
Phase 2
Phase 2
Phase 2 data released January 18, 2017 did not meet primary endpoint.
INGREZZA
Tardive dyskinesia
Approved
Approved
Approval for sNDA 80 mg capsules announced October 5, 2017.

Latest News

  1. 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.

    View Full Article Hide Full Article
  2. 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.

    Neurocrine_Biosciences_Logo

     

    Cision View original content:http://www.prnewswire.com/news-releases/neurocrine-biosciences-announces-once-daily-ongentys-opicapone-now-available-in-the-us-as-an-add-on-treatment-for-patients-with-parkinsons-disease-experiencing-off-episodes-301130083.html

    SOURCE Neurocrine Biosciences, Inc.

    View Full Article Hide Full Article
  3. -- 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

     

    Primary Logo

    View Full Article Hide Full Article
  4. 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.

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/neurocrine-biosciences-presents-new-data-analyses-demonstrating-efficacy-of-fda-approved-once-daily-ongentys-opicapone-in-patients-with-parkinsons-disease-at-the-mds-virtual-congress-2020-301128393.html

    SOURCE Neurocrine Biosciences, Inc.

    View Full Article Hide Full Article
  5. 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.

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/neurocrine-biosciences-and-voyager-therapeutics-present-new-long-term-three-year-data-demonstrating-that-one-time-treatment-with-an-investigational-gene-therapy-showed-sustained-improvement-in-motor-function-in-patients-with-parki-301128391.html

    SOURCE Neurocrine Biosciences, Inc.

    View Full Article Hide Full Article
View All Neurocrine Biosciences Inc. News