In a recent interview with PharmaShots, Dr. Eiry Roberts, Chief Medical Officer of Neurocrine Biosciences shares her insights and highlights on data of Ongentys.

Shots:

• In a post-hoc, sub-group analysis of the P-III BIPARK-1 study demonstrated greater reductions in overnight "off" time and time to morning "on" time compared to entacapone in patients with PD with motor fluctuations
• The new P-III post-hoc sub-group analysis demonstrated that long-term treatment with Ongentys when used as an add-on therapy reduced "On" time with troublesome dyskinesia and increased good "On" time without troublesome dyskinesia in patients with PD with motor fluctuations
• Ongentys (qd, PO) is a selective and reversible COMT inhibitor and has received the US FDA's approval as an add-on treatment to levodopa/carbidopa in patients with PD experiencing "off" episodes

Tuba: We understand that six abstracts of ONGENTYS were presented during ANA 2020. Can we have a quick highlight for our readers of all the presentations/ abstracts?

Eiry:  The data that Neurocrine Biosciences presented in collaboration with BIAL at the ANA 2020 Virtual Meeting provide further insight on how adding once-daily ONGENTYS® (opicapone) capsules to levodopa/carbidopa therapy, the gold standard for treatment of motor symptoms, can help patients with Parkinson's disease better manage disruptive motor fluctuations over the course of the day. Highlights include:

• Data from two post-hoc, sub-group analyses of Phase III data showed that patients treated with ONGENTYS saw greater reductions in overnight "off" time and time to morning "on" time compared to entacapone in patients with Parkinson's disease with motor fluctuations. A third post-hoc analysis showed that ONGENTYS led to a greater increase in the portion of patients who woke up on in "on" status from first-morning levodopa intake.
• In a separate 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.

Tuba: Is Neurocrine planning to evaluate the potential of ONGENTYS in other Neurological Diseases?

Eiry:  Neurocrine does not plan to evaluate ONGENTYS in other neurological diseases at this time.

Neurocrine Biosciences is building a world-class, neuroscience-focused pipeline with candidates to address a variety of serious and underserved neurology, neuroendocrinology, and psychiatry conditions with a high unmet need, including Parkinson's disease

Tuba: Does the company have any other pipeline or marketed products targeting PD?

Eiry:  Neurocrine Biosciences is building a world-class, neuroscience-focused pipeline with candidates to address a variety of serious and underserved neurology, neuroendocrinology, and psychiatry conditions with a high unmet need, including Parkinson's disease.

Neurocrine Biosciences is developing NBIb-1817 (VY-AADC) for the treatment of Parkinson's disease with Voyager Therapeutics as part of a strategic collaboration announced in January 2019. NBIb-1817 is an investigational gene therapy designed to restore AADC enzyme activity in brain cells where it can convert levodopa to dopamine. Small open-label studies performed with NBIb-1817 have shown that a single administration of the NBIb-1817 gene therapy can provide clinically meaningful improvements in motor function in patients with Parkinson’s disease. These studies also demonstrated that one-time treatment with investigational NBIb-1817 could lead to an increase in activity of the AADC enzyme measured by positron emission tomography (PET).  Our hope is that NBIb-1817 will help patients experience less off time and more on time and improve motor symptom control.

NBIb-1817 has the potential to add to the armamentarium of treatment options for patients with Parkinson's disease and could be complementary to oral therapies, such as ONGENTYS. Clinical studies to support the registration of NBIb-1817 are ongoing with a new pivotal study, RESTORE 2, currently planned to start in 2021. 

Tuba: The Phase III Clinical Studies (BIPARK-1 and BIPARK-2) supports its efficacy in adults with Parkinson's disease (PD) who are having "OFF" episodes. Is there any study evaluating its efficacy in children and pregnant ladies?

Eiry:  We do not have plans to evaluate ONGENTYS for children and pregnant women at this time. Of note, most people with Parkinson’s disease are diagnosed in their 60s.  

Tuba: In collaboration with BIAL, you have targeted multiple countries, including the US, EU, and Canada. What are the other geographies you are focusing on?

Eiry:  Neurocrine Biosciences received FDA approval of ONGENTYS in April 2020. We are pleased to bring this important new treatment option to the Parkinson's community. We are committed to ensuring that all patients with Parkinson's disease who might benefit from ONGENTYS have access to it in the U.S.

Tuba: How Neurocrine's INBRACE Support Program aid patients living with movement disorders?

Eiry:  The INBRACE® Support Program is designed to help patients who are prescribed ONGENTYS, from prescription fulfillment to navigating coverage requirements and providing financial assistance information to support patients along their treatment journey.

Source: Parkinson's NSW

Tuba: What should be the next steps in terms of research or development in Parkinson's disease?

Eiry:  There remains a significant unmet need for new treatment options that optimize motor symptom control in patients with Parkinson's disease. 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 approved gene therapy for Parkinson's disease.

Tuba: What Neurocrine thinks about digital health technologies could be used to improve PD care?

Eiry:  Parkinson's care may be improved by new advances in digital technologies. Digital devices that provide personalized information on health measures collected during patients daily life activities could be useful to patients and their health care providers for monitoring of Parkinson's disease manifestations beyond standard in-office visits.

Tuba: Is there any digital tool the company is working on that helps in understanding the daily Parkinson's experience?

Eiry:  As part of our ongoing commitment to improve the lives of patients living with movement disorders, including Parkinson's disease, we are looking into ways to help improve care for patients with Parkinson's disease, including digital technologies. 

Tuba: Would you like to note any additional takeaways on the ONGENTYS?

Eiry:  ONGENTYS was 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. The availability of ONGENTYS offers hope to patients by significantly decreasing "off" time and increasing "on" time without troublesome dyskinesia, which helps lead to more consistent motor symptom control, presenting an exciting new option for patients. The data we shared at the 2020 ANA Virtual Conference reinforces why it may be valuable to add once a day ONGENTYS to a patient's treatment regimen as early as possible.

About Dr. Eiry Roberts:

Ms. Eiry Wyn Roberts was appointed as Chief Medical Officer in January 2018 and is responsible for all clinical development and medical affairs activities at Neurocrine Biosciences. Dr. Roberts has over 25 years of research and development experience in the pharmaceutical industry across all phases of drug development from research through commercialization in multiple therapeutic areas, including neuroscience, inflammation, oncology and metabolic diseases.

Image Source: Medical News Today

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