On February 13, 2018 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported its financial results for the fourth quarter and year-end of 2017, and provided an update on the launch of INGREZZA (valbenazine) and clinical development programs for 2018 (Press release, Neurocrine Biosciences, FEB 13, 2018, View Source;p=RssLanding&cat=news&id=2332208 [SID1234523953]).

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"This past year was a year of tremendous progress for Neurocrine with the approval and successful launch of INGREZZA. I am proud of our team’s dedication and commitment to successfully launching INGREZZA while also advancing key development programs in our pipeline," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "For 2018, we remain focused on maximizing our opportunity with INGREZZA to aid patients impacted by tardive dyskinesia through further disease state awareness and positive patient outcomes. In addition, we expect to make great strides in our many research and development programs, including a potential FDA approval from our collaboration with AbbVie for elagolix in endometriosis, results from a Phase IIb study of INGREZZA for Tourette syndrome, and progressing opicapone for the treatment of Parkinson’s disease."

Fourth Quarter and Year-End 2017 Sales Results

Neurocrine reported net product sales for INGREZZA of $64.5 million for the fourth quarter ended December 31, 2017. Total Company revenues for the fourth quarter were $94.5 million inclusive of a $30 million milestone payment received from AbbVie for the U.S. Food and Drug Administration (FDA) acceptance of the elagolix endometriosis NDA filing in the fourth quarter.

For the year ended December 31, 2017, net product sales of INGREZZA were $116.6 million and total Company revenues of $161.6 million inclusive of $45 million revenue recognized from our collaboration agreements with AbbVie and Mitsubishi Tanabe Pharma Corporation (Mitsubishi Tanabe).

No similar net product sales were reported for the comparable periods of 2016. INGREZZA was made available for commercial distribution on May 1, 2017, and the Company recognizes revenue using a sell-in methodology when products are delivered to select pharmacies or distributors.

For the fourth quarter of 2017, the Company reported net income of $6.9 million, or $0.07 diluted earnings per share, compared to a net loss of $44.7 million, or $0.51 loss per share for the same period in 2016. For the year ended December 31, 2017, the Company reported a net loss of $142.5 million, or $1.62 loss per share, as compared to a net loss of $141.1 million, or $1.63 loss per share for 2016.

Research and development (R&D) expenses were $25.6 million during the fourth quarter of 2017 compared to $22.6 million for the fourth quarter of 2016. The increase in R&D expense is principally due to increased program activity in R&D. For the year ended December 31, 2017, R&D expenses were $121.8 million, compared to $94.3 million for the same period in 2016. This increase is primarily due to a $30 million payment in the first quarter of 2017 from the Company’s entering into an exclusive licensing agreement with BIAL – Portela & CA, S.A. (BIAL) for the development and commercialization of opicapone in the United States and Canada, which was expensed as in-process R&D.

Sales, general and administrative (SG&A) expenses increased to $56.3 million for the fourth quarter of 2017 from $23.7 million for the fourth quarter of 2016. For the year ended December 31, 2017, SG&A expenses were $169.9 million, compared to $68.1 million for the same period in 2016. The increase in SG&A expense, across both periods, is primarily due to commercialization activities for INGREZZA.

The Company’s balance sheet at December 31, 2017, reflected total assets of $817.6 million, including cash, investments and receivables of $797.6 million, compared with total asset balances at December 31, 2016 of $365.1 million.

2018 Financial Guidance

Revenue milestones under the AbbVie agreement for 2018 are expected to be $40 million contingent on an FDA approval of elagolix for endometriosis. Ongoing operating expenses for 2018 should approximate $365 to $395 million. The 2018 anticipated expenses include an estimated $50 million of share-based compensation expense. The increase in operating expenses is largely attributable to increased investment in INGREZZA sales and marketing activities coupled with increased R&D efforts for Tourette syndrome, opicapone, new early stage programs, and post-marketing clinical activities.

Pipeline Highlights and Upcoming Events in 2018

INGREZZA Update

INGREZZA received FDA approval on April 11, 2017, becoming the first medicine approved in the United States for the treatment of adults with tardive dyskinesia. Full commercial efforts for the 40 mg capsule of INGREZZA began on May 1, 2017. On October 4, 2017, the FDA approved the supplemental New Drug Application (NDA) for the 80 mg capsule strength of INGREZZA.

In March 2015, the Company announced that it had entered into an exclusive collaboration and licensing agreement for the development and commercialization of INGREZZA in Japan and other select Asian markets with Mitsubishi Tanabe. In 2017, Mitsubishi Tanabe initiated a pivotal trial of INGREZZA in Asia for the treatment of tardive dyskinesia which generated a $15 million milestone during the third quarter of 2017.

INGREZZA is being investigated in Tourette syndrome and has been granted Orphan Drug Designation by the FDA for the treatment of pediatric patients with Tourette syndrome. Orphan Drug Designation is granted by the FDA to drugs that are intended to treat rare diseases or conditions in the United States.

In addition, the Company has advanced the valbenazine Tourette syndrome program into Phase IIb by initiating the T-Force GOLD study in pediatric patients with Tourette syndrome. This study is a multicenter, randomized, double-blind, placebo-controlled, parallel group, Phase IIb study to evaluate the safety, tolerability, efficacy and optimal dose of once-daily valbenazine in up to 120 pediatric patients with moderate to severe Tourette syndrome over 12 weeks of treatment. The primary endpoint of this study is the change from baseline of the Yale Global Tic Severity Scale between placebo and active treatment groups at the end of week 12 with top-line data expected in late 2018.

Elagolix Update

On October 27, 2017, AbbVie, in collaboration with Neurocrine, announced that the FDA had granted Priority Review to elagolix for the management of endometriosis with associated pain. The FDA grants Priority Review to medicines that it determines have potential to provide significant improvements in the safety and effectiveness of the treatment of a serious disease. Priority Review shortens the FDA review timeframe from ten months from acceptance of the NDA filing to six months. The Prescription Drug User Fee Act (PDUFA) date for the FDA to complete its review is in the second quarter of 2018.

AbbVie is currently completing two replicate Phase III randomized, parallel, double-blind, placebo-controlled clinical trials evaluating elagolix alone or in combination with add-back therapy in women with heavy uterine bleeding associated with uterine fibroids. The studies enrolled approximately 400 subjects each for an initial six-month placebo-controlled dosing period. At the end of the six months of placebo-controlled evaluation, subjects are eligible to enter an additional six-month safety extension study. The primary efficacy endpoint of the study is an assessment of the change in menstrual blood loss utilizing the alkaline hematin method comparing baseline to month six. Additional secondary efficacy endpoints will be evaluated including assessing the change in fibroid volume and hemoglobin. Bone mineral density will be assessed via dual-energy x-ray absorptiometry (DEXA) scan at baseline, at the conclusion of dosing, and six months post-dosing. AbbVie expects initial top-line efficacy data from the uterine fibroid Phase III program during the first quarter of 2018. These two studies will form the basis for an anticipated 2019 supplemental NDA submission to the FDA for the approval of elagolix in the treatment of uterine fibroids.

Opicapone Update

In February 2017, the Company entered into an exclusive licensing agreement with BIAL for the development and commercialization of opicapone in the United States and Canada. Opicapone is a once-daily, peripherally-acting, highly-selective catechol-o-methyltransferase inhibitor being developed as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors for adult patients with Parkinson’s disease and motor fluctuations. The Company met with the FDA in January to discuss the necessary activities to support an NDA submission and will provide an update on the regulatory path and program timing after official meeting minutes are received later this month.

Congenital Adrenal Hyperplasia (CAH) Program (NBI-74788) Update

In the second quarter of 2017, the Company successfully completed the Phase I, IND-opening study of NBI-74788 in healthy volunteer subjects. The study was a randomized, open-label, two-period crossover study to evaluate the pharmacokinetics (PK), the effect of food on PK, and the safety of NBI-74788 in a total of 16 healthy adults.

The Company began recruitment for a Phase II, proof-of-concept study examining the PK, pharmacodynamics, and safety of NBI-74788 in adult males and females with classic, 21-hydroxylase deficiency CAH in November of 2017. The study will evaluate the relationship between NBI-74788 exposures and specific steroid hormone levels in these subjects and expect data to be available during the first half of 2018.

Conference Call and Webcast Today at 5:00 PM Eastern Time

Neurocrine will hold a live conference call and webcast today at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). Participants can access the live conference call by dialing 888-632-3389 (US) or 785-424-1673 (International) using the conference ID: NBIX. The call can also be accessed via the webcast through the Company’s website at View Source

About INGREZZA (valbenazine) Capsules
INGREZZA, a selective VMAT2 inhibitor, is the first FDA approved product indicated for the treatment of adults with tardive dyskinesia, a condition associated with uncontrollable, abnormal and repetitive movements of the trunk, extremities and/or face.

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

Important Safety Information
Warnings & Precautions
Somnolence
INGREZZA can cause somnolence. Patients should not perform activities requiring mental alertness such as operating a motor vehicle or operating hazardous machinery until they know how they will be affected by INGREZZA.

QT Prolongation
INGREZZA may prolong the QT interval, although the degree of QT prolongation is not clinically significant at concentrations expected with recommended dosing. INGREZZA should be avoided in patients with congenital long QT syndrome or with arrhythmias associated with a prolonged QT interval. For patients at increased risk of a prolonged QT interval, assess the QT interval before increasing the dosage.

Adverse Reactions
The most common adverse reaction (≥5% and twice the rate of placebo) is somnolence. Other adverse reactions (≥2% and >placebo) include: anticholinergic effects, balance disorders/falls, headache, akathisia, vomiting, nausea, and arthralgia.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch at www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see INGREZZA full Prescribing Information at www.INGREZZA.com/HCP