Neurocrine Biosciences, Inc. has announced its financial results for the quarter ended March 31, 2015, in which the company reported a $1.2 million net loss — or $0.01 loss per share — compared to a net loss of $11.8 million, or $0.17 loss per share, for the same period in 2014.
During the quarter, the Company completed a public offering of eight million shares of common stock that resulted in net proceeds of approximately $270 million, and additionally entered into a collaboration and license agreement with Japan’s Mitsubishi Tanabe Pharma Corporation for development and commercialization of its VMAT2 inhibitor, NBI-98854, in that country and other select Asian markets, resulting in a $30 million up-front payment.
“The first quarter of 2015 was very successful for Neurocrine; starting with positive top-line elagolix Phase III data in endometriosis, a successful capital raise and signing an Asian partnership with Mitsubishi Tanabe for NBI-98854. This capital raise provides the funding necessary to continue to advance our pipeline, including moving NBI-98854 into commercialization upon successful completion of the Phase III program and FDA approval,” said Neurocrine Biosciences President and Chief Executive Officer Kevin Gorman, Ph.D.. “For the balance of 2015, we turn our focus to executing across our pipeline and advancing a novel compound from research into the clinic. Over the coming eight months, we look forward to seven clinical trials reading out across four distinct programs.”
The $19.8 million in revenue for the first quarter of 2015 represents recognized revenue in the form of license fees from the NBI-98854 collaboration and license agreement with Mitsubishi Tanabe that was executed on March 31, 2015. Research and development expenses increased to $16.6 million during the first quarter of 2015 from $8.6 million during the same period in 2014 — sn increase primarily attributable to higher external clinical development expenses and associated internal costs related to the Company’s VMAT2 inhibitor, NBI-98854, which initiated Phase III development in the second half of 2014.
Additionally, expenses related to the Company’s congenital adrenal hyperplasia program increased from the first quarter of 2014. General and administrative expenses increased from $4.2 million in the first quarter of 2014 to $5.5 million for the first quarter of 2015, primarily due to higher personnel related costs, including a $0.5 million increase in share-based compensation expense.
In 2014, the Company initiated a Phase III study of NBI-98854, the Kinect 3 study, which, along with previous efficacy studies of NBI-98854, is designed to complete the placebo-controlled clinical efficacy evaluation of NBI-98854 in tardive dyskinesia. The primary endpoint in the Kinect 3 study is the mean change from baseline in the Abnormal Involuntary Movement Scale (AIMS) as assessed by blinded central raters.
According to the National Institutes of Health’s National Institute of Neurological Disorders and Stroke Tardive dyskinesia is a neurological syndrome caused by the long-term use of neuroleptic drugs. Neuroleptic drugs are generally prescribed for psychiatric disorders, as well as for some gastrointestinal and neurological disorders. Tardive dyskinesia is characterized by repetitive, involuntary, purposeless movements. Features of the disorder may include grimacing, tongue protrusion, lip smacking, puckering and pursing, and rapid eye blinking. Rapid movements of the arms, legs, and trunk may also occur. Involuntary movements of the fingers may be present.
NBI-98854 is a potent, highly selective, VMAT2 inhibitor that is effective in regulating the levels of dopamine release during nerve communication, while at the same time having minimal impact on the other monoamines. This selectivity may reduce the likelihood of “off target” side effects. Additionally, we have designed this novel compound to provide low, sustained, plasma and brain concentrations of the active drug to minimize the potential side effects associated with excessive dopamine depletion. With these features, NBI-98854 is anticipated to be well tolerated in patients.
The Kinect 3 study includes approximately 240 subjects randomized to either placebo, once daily 40mg of NBI-98854, or once daily 80mg of NBI-98854 for six weeks of placebo-controlled dosing followed by an extension of active dosing through Week 48. Top-line efficacy data from the initial six weeks of placebo-controlled dosing is expected in the second half of 2015.
A separate one-year open-label safety study of NBI-98854 has also been initiated to support the anticipated 2016 filing of a New Drug Application in tardive dyskinesia, and as announced previously, Neurocrine has also received Breakthrough Therapy Designation from the FDA for NBI-98854 in the treatment of tardive dyskinesia.
The Company is also exploring NBI-98854 in an initial Tourette syndrome clinical trial, the T-Force study — an open-label, multi-dose, two-week evaluation of 36 subjects with Tourette syndrome. Children and adolescents enrolled in the trial receive a once-daily dose of NBI-98854 during a two-week treatment period to assess both the safety and tolerability of NBI-98854. Additionally, the Yale Global Tic Severity Scale and the Premonitory Urge for Tics Scale are being utilized during the study to assess the impact of NBI-98854 on the patients’ Tourette symptoms. Data read out from the T-Force study is expected in the second half of 2015.
As noted above, on March 31, 2015 the Company entered into an exclusive collaboration and licensing agreement for the development and commercialization of its VMAT2 inhibitor, NBI-98854, in Japan and other select Asian markets with Mitsubishi Tanabe Pharma Corporation. Upon commercialization of the drug, Neurocrine will receive royalties on product sales from select territories in Asia, and will also support Mitsubishi Tanabe’s clinical efforts in developing NBI-98854 for patients suffering from the chorea associated with Huntington’s disease and tardive dyskinesia.
During the first quarter of 2015, Neurocrine’s partner firm AbbVie Inc. \ announced positive top-line results from the first of two ongoing Phase III clinical trials, dubbed “the Violet Petal Study,” designed to evaluate the efficacy and safety of elagolix — a novel, orally administered formulation of gonadotropin-releasing hormone antagonist, or GnRH Antagonist currently being investigated in patients with pain from endometriosis. Elagolix inhibits gonadatropin releasing hormone (GnRH) receptors in the pituitary gland and ultimately reduces circulating sex hormone levels. To date, elagolix has been studied in over 40 clinical trials totalling more than 3,000 patients. Elagolix is believed to have its effect by altering the level of pituitary GnRH suppression and, as a result, titrating circulating estrogen levels. By this method, it is believed that elagolix will provide relief from the pain associated with conditions such endometriosis and uterine fibroids, without a need to actively manage bone loss. in premenopausal women with endometriosis.
Results from the trial show that after six months of treatment, both doses of elagolix (150 mg once daily and 200 mg twice daily) met the study’s co-primary endpoints (p<0.001) of reducing scores of non-menstrual pelvic pain (NMPP) and menstrual pain (or dysmenorrhea) associated with endometriosis at month three, as well as month six, as measured by the Daily Assessment of Endometriosis Pain scale. Elagolix’s observed safety profile in the Violet Petal Study was consistent with observations from prior studies. Among the most common adverse events (AEs) were hot flush, headache, nausea and fatigue. While most AEs were similar across treatment groups some, such as hot flush and bone mineral density (BMD) loss, were dose-dependent.
AbbVie is also conducting the second Phase III study of elagolix for endometriosis — the Solstice Study — which is similar in design to the Violet Petal Study and will assess 788 women, age 18 to 49, with moderate to severe endometriosis-associated pain at more than 200 sites globally. Top-line efficacy data from this study is expected in late 2015.
Elagolix is also being evaluated in women with uterine fibroids. AbbVie is conducting a Phase IIb clinical trial evaluating the change in menstrual blood loss of 520 women, age 18-51, with heavy menstrual bleeding associated with uterine fibroids. Patient recruitment has recently been completed and top-line data from this study is expected in 2015.
Corticotropin Releasing Factor (Congenital Adrenal Hyperplasia) Update
Neurocrine also recently announced the completion of a pilot clinical trial of NBI-77860 (verucerfont a potent, selective, non-peptide CRF1 receptor antagonist as demonstrated in a range of in vitro and in vivo assays as well as human clinical studies. NBI-77860 is being studied for the treatment of classical congenital adrenal hyperplasia against placebo in adult females with refractory classic congenital adrenal hyperplasia (CAH). This eight person single dose exploratory study showed that NBI-77860 was effective in reducing the key biomarkers of adrenocorticotropic hormone (ACTH) and 17-hydroxyprogesterone androgen (17-OHP). A full description of the study results and related data was presented at the Endocrine Society’s 97th Annual Meeting in San Diego on March 5, 2015.
Neurocrine has initiated a second clinical trial assessing three doses of NBI-77860 in an open-label, sequential cohort, single ascending dose pharmacokinetic/pharmacodynamic study. Fifteen adolescent females with classic CAH will be split into three cohorts and each will receive one dose of NBI-77860 once a day. Biomarker measurements include ACTH, 17-OHP, androgen and cortisol levels collected the morning after dosing. Data from this study is expected later in 2015.
A replay of the Neurocrine 1Q15 financial results live conference call and webcast can be accessed via the Company’s website at:
http://www.neurocrine.com, or by telephone by dealing 800-839-5127(US) or 402-220-2692(International) using the conference ID: NBIX. The call will be archived for one month.
For more information, visit:
Neurocrine Biosciences, Inc.
National Institute of Neurological Disorders and Stroke
National Institutes of Health