09:00 uur 29-04-2020

FDA approves ONGENTYS® ▼ (opicapone)

PRESS RELEASE FOR EUROPEAN MEDICAL AND TRADE MEDIA ONLY

PORTO, Portugal–(BUSINESS WIRE)– BIAL announced today that the U.S Food and Drug Administration (FDA) has approved ONGENTYS^® (opicapone) as an add-on treatment to levodopa/carbidopa in patients with Parkinson’s disease experiencing “off” episodes.

“We are very pleased to achieve this major regulatory milestone for opicapone, which offers patients living with Parkinson´s disease an effective, once-daily adjunctive treatment option to the gold standard levodopa/dopa-decarboxylase inhibitors preparations. This approval is a landmark in BIAL’s ongoing commitment to the quality of life of Parkinson’s patients and their caregivers. We look forward to working with our partner in the U.S., Neurocrine Biosciences, to make this therapy available to patients.” said António Portela, CEO of BIAL.

In February 2017, BIAL and Neurocrine Biosciences entered into an exclusive licensing agreement for the development and commercialization of opicapone in the U.S. and Canada. The commercial launch of opicapone in the U.S. is expected to occur later in 2020.

The FDA approval of opicapone is supported by data from 38 clinical studies, including two multinational Phase III clinical studies (BIPARK-1 and BIPARK-2). In BIPARK-1, a randomized, double-blind placebo- and active-controlled study, approximately 600 patients with Parkinson’s disease and end-of-dose motor fluctuations received once-daily doses of opicapone (5 mg, 25 mg, or 50 mg), placebo or 200 mg of the COMT (catechol-O-methyltransferase) inhibitor entacapone for 14 to 15 weeks as adjunct to levodopa therapy. In BIPARK-2, which followed a similar study design, approximately 400 patients received once-daily doses of opicapone (25 mg or 50 mg) or placebo.

The primary endpoint for both studies was the change from baseline in absolute time in the OFF state, as assessed by patient diaries. The initial study period in each BIPARK was followed by a one year open-label phase during which all eligible patients received treatment with opicapone. Treatment with opicapone 50 mg was found to be superior to placebo in both studies. The beneficial effects of opicapone 50 mg at reducing the time in the OFF state were accompanied by a corresponding increase in time in the ON state without troublesome dyskinesia. Results from both open-label phases indicated a maintenance of effect for patients previously treated with opicapone 50 mg. Overall, opicapone was found to be generally well tolerated. Primary outcomes from the BIPARK-1 study are published in Lancet Neurology¹, with the outcomes from the open-label extension phase published in Neurology². Primary outcomes from the BIPARK-2 study and the open-label extension are published in JAMA Neurology³.

Parkinson’s disease is a neurodegenerative, chronic and progressive disease. The clinical manifestations usually start after the age of 50 years (average age for diagnosis is approximately 60 years) and the prevalence is estimated at 300 per 100,000 inhabitants, increasing to 1/100 over the age of 55–60 years.

In the U.S. each year, an estimated 50,000 people are diagnosed with this neurodegenerative disorder and about one million Americans have the condition⁴. The European Parkinson’s Disease Association (EPDA) estimates that 1.2 million people have Parkinson’s disease in the European Union⁵.

About Opicapone ⁶

Opicapone is a once-daily, peripherally-acting, third-generation, highly-selective COMT inhibitor.

Opicapone works by decreasing peripheral levodopa’s conversion rate into 3-O-methyldopa, thereby prolonging the duration of levodopa’s effect in reducing the OFF-time period of Parkinson’s and extending the ON-time period.

In June 2016, the European Commission authorized Ongentys^® (opicapone) as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors (DDCIs) in adult patients with Parkinson’s disease and end-of-dose motor fluctuations who cannot be stabilized on those combinations. In Europe opicapone is currently marketed in Germany, United Kingdom, Spain, Portugal and Italy.

About BIAL

Founded in 1924, BIAL’s mission is to research, develop and provide therapeutic solutions within the area of health. In the last decades, BIAL has focused strategically on quality, innovation and internationalisation. BIAL is strongly committed to therapeutic innovation, investing more than 20% of its annual turnover into research and development within neurosciences and the cardiovascular system. The company expects to introduce new drugs on the market in the coming years, strengthening its international presence based on proprietary drugs and achieving its goal of supplying innovative products to patients worldwide.

For more information on BIAL: www.bial.com

References

1. Ferreira J., et al. Lancet Neurol. 2016 Feb;15(2):154-165

2. Ferreira J., et al. Neurology. 2018 May 22;90(21):e1849-e185

3. Lees A., et al. JAMA Neurol. 2017 Feb 1;74(2):197-206

4. Parkinson’s News Today. Available at https://parkinsonsnewstoday.com/parkinsons-disease-statistics/. Accessed April 2020

5. ICM Institute. Neurogenerative Diseases. Available at https://icm-institute.org/en/key-figures/. Accessed March 2020

6. Ongentys^® EU SMPC. Last updated 22/04/2020.

ON/APR20/G/301

Date of preparation: April 2020