On July 30, 2019 Eli Lilly and Company (NYSE: LLY) reported that Verzenio (abemaciclib) demonstrated a statistically significant improvement in overall survival in the Phase 3 MONARCH 2 clinical trial (Press release, Eli Lilly, JUL 30, 2019, View Source [SID1234537878]). These results were from a pre-planned interim analysis and are definitive. MONARCH 2 evaluated Verzenio in combination with fulvestrant for the treatment of women with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced or metastatic breast cancer previously treated with endocrine therapy. The study included both pre/peri- and postmenopausal women.

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The analysis showed that treatment with Verzenio in combination with fulvestrant met its secondary endpoint of overall survival. The MONARCH 2 study previously demonstrated a statistically significant improvement in progression-free survival, the trial’s primary endpoint which served as the basis for its approval of this regimen in more than 50 countries around the world. No new safety signals were observed in this analysis of MONARCH 2, consistent with the established safety profile of Verzenio.

"I believe we must continue to fight this devastating disease because the women who are living with metastatic breast cancer want to do everything they can to lead more fulfilling lives and be there for those who need them most," said Anne White, president, Lilly Oncology. "While Verzenio had already shown an impressive benefit for progression-free survival, we are delighted that Verzenio is the first and only CDK4 & 6 inhibitor in combination with fulvestrant that has significantly extended life for both pre/peri- and postmenopausal women."

Lilly plans to submit these data to regulatory authorities and present the detailed data at an upcoming medical meeting later this year.

"This definitive overall survival analysis from MONARCH 2 showed significant improvement in overall survival for women living with HR+, HER2- metastatic breast cancer, a complex disease that remains incurable," said Maura Dickler, M.D., vice president, late stage development, Lilly Oncology. "For many doctors and patients overall survival is the most important endpoint. It’s been difficult in the past to achieve meaningful improvements in this endpoint for women with advanced breast cancer, including those whose cancer progressed after prior endocrine therapy."

About MONARCH 2
MONARCH 2 is a Phase 3, randomized, double-blind, placebo-controlled trial that enrolled 669 patients with HR+, HER2- metastatic breast cancer who progressed on endocrine therapy. Patients were randomized 2:1 to Verzenio plus fulvestrant or placebo plus fulvestrant. Verzenio was dosed on a continuous dosing schedule until disease progression or unacceptable toxicity. The primary endpoint was progression-free survival (PFS). Key secondary endpoints were objective response rate (ORR), overall survival (OS), and duration of response (DoR). Patients enrolled in the study had experienced disease progression on or within 12 months of receiving endocrine treatment in the neoadjuvant or adjuvant setting or while receiving first-line endocrine therapy for metastatic disease. Patients could not have received chemotherapy or more than one line of endocrine therapy for metastatic breast cancer.

About Advanced Breast Cancer
Breast cancer is the most common cancer in women worldwide, with more than 2 million new cases diagnosed in 2018.1 An estimated 268,600 new cases of invasive breast cancer are expected to be diagnosed in women in the U.S. in 2019.2 Advanced breast cancer includes metastatic breast cancer, meaning cancer that has spread from the breast tissue to other parts of the body, and locally or regionally advanced breast cancer, meaning the cancer has grown outside the organ where it started but has not yet spread to other parts of the body.3 Of all early stage breast cancer cases diagnosed in the U.S., approximately 30 percent will become metastatic4 and an estimated 6 to 10 percent of all new breast cancer cases are initially diagnosed as being metastatic.5 Survival is lower among women with a more advanced stage at diagnosis: five-year relative survival is 99 percent for localized disease, 85 percent for regional disease, and 27 percent for metastatic disease.6 Other factors, such as tumor size, also impact five-year survival estimates.6

About Verzenio (abemaciclib)
Verzenio (abemaciclib) is an inhibitor of cyclin-dependent kinases (CDK)4 & 6, which are activated by binding to D-cyclins. In estrogen receptor-positive (ER+) breast cancer cell lines, cyclin D1 and CDK4 & 6 promote phosphorylation of the retinoblastoma protein (Rb), cell cycle progression, and cell proliferation.

In vitro, continuous exposure to Verzenio inhibited Rb phosphorylation and blocked progression from G1 to S phase of the cell cycle, resulting in senescence and apoptosis (cell death). Preclinically, Verzenio dosed daily without interruption resulted in reduction of tumor size. Inhibiting CDK4 & 6 in healthy cells can result in side effects, some of which may be serious. Clinical evidence also suggests that Verzenio crosses the blood-brain barrier. In patients with advanced cancer, including breast cancer, concentrations of Verzenio and its active metabolites (M2 and M20) in cerebrospinal fluid are comparable to unbound plasma concentrations.

Verzenio is Lilly’s first solid oral dosage form to be made using a faster, more efficient process known as continuous manufacturing. Continuous manufacturing is a new and advanced type of manufacturing within the pharmaceutical industry, and Lilly is one of the first companies to use this technology.

INDICATION
Verzenio is indicated for the treatment of HR+, HER2- advanced or metastatic breast cancer:

in combination with an aromatase inhibitor for postmenopausal women as initial endocrine-based therapy
in combination with fulvestrant for women with disease progression following endocrine therapy
as a single agent for adult patients with disease progression following endocrine therapy and prior chemotherapy in the metastatic setting
IMPORTANT SAFETY INFORMATION
Diarrhea occurred in 81% of patients receiving Verzenio plus an aromatase inhibitor in MONARCH 3, 86% of patients receiving Verzenio plus fulvestrant in MONARCH 2 and 90% of patients receiving Verzenio alone in MONARCH 1. Grade 3 diarrhea occurred in 9% of patients receiving Verzenio plus an aromatase inhibitor in MONARCH 3, 13% of patients receiving Verzenio plus fulvestrant in MONARCH 2 and in 20% of patients receiving Verzenio alone in MONARCH 1. Episodes of diarrhea have been associated with dehydration and infection.

Diarrhea incidence was greatest during the first month of Verzenio dosing. In MONARCH 3, the median time to onset of the first diarrhea event was 8 days, and the median duration of diarrhea for Grades 2 and 3 were 11 and 8 days, respectively. In MONARCH 2, the median time to onset of the first diarrhea event was 6 days, and the median duration of diarrhea for Grades 2 and 3 were 9 days and 6 days, respectively. In MONARCH 3, 19% of patients with diarrhea required a dose omission and 13% required a dose reduction. In MONARCH 2, 22% of patients with diarrhea required a dose omission and 22% required a dose reduction. The time to onset and resolution for diarrhea were similar across MONARCH 3, MONARCH 2, and MONARCH 1.

Instruct patients that at the first sign of loose stools, they should start antidiarrheal therapy such as loperamide, increase oral fluids, and notify their healthcare provider for further instructions and appropriate follow-up. For Grade 3 or 4 diarrhea, or diarrhea that requires hospitalization, discontinue Verzenio until toxicity resolves to ≤Grade 1, and then resume Verzenio at the next lower dose.

Neutropenia occurred in 41% of patients receiving Verzenio plus an aromatase inhibitor in MONARCH 3, 46% of patients receiving Verzenio plus fulvestrant in MONARCH 2 and 37% of patients receiving Verzenio alone in MONARCH 1. A Grade ≥3 decrease in neutrophil count (based on laboratory findings) occurred in 22% of patients receiving Verzenio plus an aromatase inhibitor in MONARCH 3, 32% of patients receiving Verzenio plus fulvestrant in MONARCH 2 and in 27% of patients receiving Verzenio alone in MONARCH 1. In MONARCH 3, the median time to first episode of Grade ≥3 neutropenia was 33 days, and in MONARCH 2 and MONARCH 1, was 29 days. In MONARCH 3, median duration of Grade ≥3 neutropenia was 11 days, and for MONARCH 2 and MONARCH 1 was 15 days.

Monitor complete blood counts prior to the start of Verzenio therapy, every 2 weeks for the first 2 months, monthly for the next 2 months, and as clinically indicated. Dose interruption, dose reduction, or delay in starting treatment cycles is recommended for patients who develop Grade 3 or 4 neutropenia.

Febrile neutropenia has been reported in <1% of patients exposed to Verzenio in the MONARCH studies. Two deaths due to neutropenic sepsis were observed in MONARCH 2. Inform patients to promptly report any episodes of fever to their healthcare provider.

Grade ≥3 increases in alanine aminotransferase (ALT) (6% versus 2%) and aspartate aminotransferase (AST) (3% versus 1%) were reported in the Verzenio and placebo arms, respectively, in MONARCH 3. Grade ≥3 increases in ALT (4% versus 2%) and AST (2% versus 3%) were reported in the Verzenio and placebo arms respectively, in MONARCH 2.

In MONARCH 3, for patients receiving Verzenio plus an aromatase inhibitor with Grade ≥3 increases in ALT or AST, median time to onset was 61 and 71 days, respectively, and median time to resolution to Grade <3 was 14 and 15 days, respectively. In MONARCH 2, for patients receiving Verzenio plus fulvestrant with Grade ≥3 increases in ALT or AST, median time to onset was 57 and 185 days, respectively, and median time to resolution to Grade <3 was 14 and 13 days, respectively.

For assessment of potential hepatotoxicity, monitor liver function tests (LFTs) prior to the start of Verzenio therapy, every 2 weeks for the first 2 months, monthly for the next 2 months, and as clinically indicated. Dose interruption, dose reduction, dose discontinuation, or delay in starting treatment cycles is recommended for patients who develop persistent or recurrent Grade 2, or Grade 3 or 4, hepatic transaminase elevation.

Venous thromboembolic events were reported in 5% of patients treated with Verzenio plus an aromatase inhibitor as compared to 0.6% of patients treated with an aromatase inhibitor plus placebo in MONARCH 3. Venous thromboembolic events were reported in 5% of patients treated with Verzenio plus fulvestrant in MONARCH 2 as compared to 0.9% of patients treated with fulvestrant plus placebo. Venous thromboembolic events included deep vein thrombosis, pulmonary embolism, pelvic venous thrombosis, cerebral venous sinus thrombosis, subclavian and axillary vein thrombosis, and inferior vena cava thrombosis. Across the clinical development program, deaths due to venous thromboembolism have been reported. Monitor patients for signs and symptoms of venous thrombosis and pulmonary embolism and treat as medically appropriate.

Verzenio can cause fetal harm when administered to a pregnant woman based on findings from animal studies and the mechanism of action. In animal reproduction studies, administration of abemaciclib to pregnant rats during the period of organogenesis caused teratogenicity and decreased fetal weight at maternal exposures that were similar to the human clinical exposure based on area under the curve (AUC) at the maximum recommended human dose. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with Verzenio and for at least 3 weeks after the last dose. There are no data on the presence of Verzenio in human milk or its effects on the breastfed child or on milk production. Advise lactating women not to breastfeed during Verzenio treatment and for at least 3 weeks after the last dose because of the potential for serious adverse reactions in breastfed infants. Based on findings in animals, Verzenio may impair fertility in males of reproductive potential.

The mostcommon adverse reactions (all grades, ≥10%) observed in MONARCH 3 for Verzenio plus anastrozole or letrozole and ≥2% higher than placebo plus anastrozole or letrozole vs placebo plus anastrozole or letrozole were diarrhea (81% vs 30%), neutropenia (41% vs 2%), fatigue (40% vs 32%), infections (39% vs 29%), nausea (39% vs 20%), abdominal pain (29% vs 12%), vomiting (28% vs 12%), anemia (28% vs 5%), alopecia (27% vs 11%), decreased appetite (24% vs 9%), leukopenia (21% vs 2%), creatinine increased (19% vs 4%), constipation (16% vs 12%), ALT increased (16% vs 7%), AST increased (15% vs 7%), rash (14% vs 5%), pruritus (13% vs 9%), cough (13% vs 9%), dyspnea (12% vs 6%), dizziness (11% vs 9%), weight decreased (10% vs 3%), influenza-like illness (10% vs 8%), and thrombocytopenia (10% vs 2%).

The most common adverse reactions (all grades, ≥10%) observed in MONARCH 2 for Verzenio plus fulvestrant and ≥2% higher than placebo plus fulvestrant vs placebo plus fulvestrant were diarrhea (86% vs 25%), neutropenia (46% vs 4%), fatigue (46% vs 32%), nausea (45% vs 23%), infections (43% vs 25%), abdominal pain (35% vs 16%), anemia (29% vs 4%), leukopenia (28% vs 2%), decreased appetite (27% vs 12%), vomiting (26% vs 10%), headache (20% vs 15%), dysgeusia (18% vs 3%), thrombocytopenia (16% vs 3%), alopecia (16% vs 2%), stomatitis (15% vs 10%), ALT increased (13% vs 5%), pruritus (13% vs 6%), cough (13% vs 11%), dizziness (12% vs 6%), AST increased (12% vs 7%), peripheral edema (12% vs 7%), creatinine increased (12% vs <1%), rash (11% vs 4%), pyrexia (11% vs 6%), and weight decreased (10% vs 2%).

The most common adverse reactions (all grades, ≥10%) observed in MONARCH 1 with Verzenio were diarrhea (90%), fatigue (65%), nausea (64%), decreased appetite (45%), abdominal pain (39%), neutropenia (37%), vomiting (35%), infections (31%), anemia (25%), thrombocytopenia (20%), headache (20%), cough (19%), leukopenia (17%), constipation (17%), arthralgia (15%), dry mouth (14%), weight decreased (14%), stomatitis (14%), creatinine increased (13%), alopecia (12%), dysgeusia (12%), pyrexia (11%), dizziness (11%), and dehydration (10%).

The most frequently reported ≥5% Grade 3 or 4 adverse reactions that occurred in the Verzenio arm vs the placebo arm of MONARCH 3 were neutropenia (22% vs 2%), diarrhea (9% vs 1%), leukopenia (8% vs <1%), ALT increased (7% vs 2%), and anemia (6% vs 1%).

The most frequently reported ≥5% Grade 3 or 4 adverse reactions that occurred in the Verzenio arm vs the placebo arm of MONARCH 2 were neutropenia (27% vs 2%), diarrhea (13% vs <1%), leukopenia (9% vs 0%), anemia (7% vs 1%), and infections (6% vs 3%).

The most frequently reported ≥5% Grade 3 or 4 adverse reactions from MONARCH 1 with Verzenio were neutropenia (24%), diarrhea (20%), fatigue (13%), infections (7%), leukopenia (6%), anemia (5%), and nausea (5%).

Lab abnormalities (all grades; Grade 3 or 4) for MONARCH 3 in ≥10% for Verzenio plus anastrozole or letrozole and ≥2% higher than placebo plus anastrozole or letrozole vs placebo plus anastrozole or letrozole were increased serum creatinine (98% vs 84%; 2% vs 0%), decreased white blood cells (82% vs 27%; 13% vs <1%), anemia (82% vs 28%; 2% vs 0%), decreased neutrophil count (80% vs 21%; 22% vs 3%), decreased lymphocyte count (53% vs 26%; 8% vs 2%), decreased platelet count (36% vs 12%; 2% vs <1%), increased ALT (48% vs 25%; 7% vs 2%), and increased AST (37% vs 23%; 4% vs <1%).

Lab abnormalities (all grades; Grade 3 or 4) for MONARCH 2 in ≥10% for Verzenio plus fulvestrant and ≥2% higher than placebo plus fulvestrant vs placebo plus fulvestrant were increased serum creatinine (98% vs 74%; 1% vs 0%), decreased white blood cells (90% vs 33%; 23% vs 1%), decreased neutrophil count (87% vs 30%; 33% vs 4%), anemia (84% vs 33%; 3% vs <1%), decreased lymphocyte count (63% vs 32%; 12% vs 2%), decreased platelet count (53% vs 15%; 2% vs 0%), increased ALT (41% vs 32%; 5% vs 1%), and increased AST (37% vs 25%; 4% vs 4%).

Lab abnormalities (all grades; Grade 3 or 4) for MONARCH 1 with Verzenio were increased serum creatinine (98%; <1%), decreased white blood cells (91%; 28%), decreased neutrophil count (88%; 27%), anemia (68%; 0%), decreased lymphocyte count (42%; 14%), decreased platelet count (41%; 2%), increased ALT (31%; 3%), and increased AST (30%; 4%).

Strong and moderate CYP3A inhibitors increased the exposure of abemaciclib plus its active metabolites to a clinically meaningful extent and may lead to increased toxicity. Avoid concomitant use of the strong CYP3A inhibitor ketoconazole. Ketoconazole is predicted to increase the AUC of abemaciclib by up to 16-fold. In patients with recommended starting doses of 200 mg twice daily or 150 mg twice daily, reduce the Verzenio dose to 100 mg twice daily with concomitant use of strong CYP3A inhibitors other than ketoconazole. In patients who have had a dose reduction to 100 mg twice daily due to adverse reactions, further reduce the Verzenio dose to 50 mg twice daily with concomitant use of strong CYP3A inhibitors. If a patient taking Verzenio discontinues a strong CYP3A inhibitor, increase the Verzenio dose (after 3 to 5 half-lives of the inhibitor) to the dose that was used before starting the inhibitor. With concomitant use of moderate CYP3A inhibitors, monitor for adverse reactions and consider reducing the Verzenio dose in 50 mg decrements. Patients should avoid grapefruit products.

Avoid concomitant use of strong or moderate CYP3A inducers and consider alternative agents. Coadministration of strong or moderate CYP3A inducers decreased the plasma concentrations of abemaciclib plus its active metabolites and may lead to reduced activity.

With severe hepatic impairment (Child-Pugh Class C), reduce the Verzenio dosing frequency to once daily. The pharmacokinetics of Verzenio in patients with severe renal impairment (CLcr <30 mL/min), end stage renal disease, or in patients on dialysis is unknown. No dosage adjustments are necessary in patients with mild or moderate hepatic (Child-Pugh A or B) and/or renal impairment (CLcr ≥30-89 mL/min).

Please see full Prescribing Information for Verzenio.

On July 30, 2019 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported its financial results for the quarter ended June 30, 2019 and provided an update on the launch of INGREZZA (valbenazine) and its clinical development programs (Press release, Neurocrine Biosciences, JUL 30, 2019, View Source [SID1234537867]).

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"Over the past two years, our team has done an excellent job building awareness of tardive dyskinesia within the healthcare community and providing patients access to INGREZZA. We continue to focus on educating about tardive dyskinesia and the benefits of INGREZZA in providing relief for patients suffering from this debilitating movement disorder," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "We have also made progress across our development pipeline with the FDA acceptance of our New Drug Application for opicapone and the anticipated regulatory submission of elagolix for uterine fibroids by AbbVie. In addition, we continued to advance our congenital adrenal hyperplasia program with the recent initiation of our pediatric trial and look forward to our planned discussion with the FDA on the adult program. We are pleased with this exciting progress and remain on track to have three FDA-approved treatments in four indications by 2020."

Financial Results

Total revenues for the three and six months ended June 30, 2019, was $183.6 million and $322.0 million, respectively, compared to $96.9 million and $168.0 million for the same periods in 2018.

Total revenues were comprised of the following (unaudited):

For the second quarter of 2019, the Company reported net income of $51.3 million, or $0.54 diluted earnings per share, compared to a net loss of $5.9 million, or $0.07 net loss per share, for the same period in 2018. The increase in net income is due to increased INGREZZA net product sales and a $21.0 million unrealized gain on the Company’s Voyager Therapeutics equity investment. For the six months ended June 30, 2019, the Company reported a net loss of $50.8 million, or $0.56 net loss per share, compared to a net loss of $47.7 million, or $0.53 net loss per share, for the same period in 2018. The increase in net loss for the first half of 2019 is primarily due to $118.1 million of in-process research and development (IPR&D) in connection with the strategic collaboration with Voyager. This was partially offset by increased INGREZZA net product sales and a $22.6 million unrealized gain on the Company’s equity investment in Voyager.

Research and development (R&D) expenses increased to $61.7 million for the second quarter of 2019 from $37.0 million for the same period in 2018. The increase in R&D expenses primarily reflects funding of development activities and a $10.0 million milestone expense related to the U.S. Food and Drug Administration (FDA) acceptance of the opicapone New Drug Application (NDA). For the six months ended June 30, 2019, R&D expenses increased to $99.3 million, compared to $85.9 million for the same period in 2018. The increase in R&D expenses for the first half of 2019 primarily reflects funding of development activities in connection with the Company’s collaboration with Voyager.

In connection with the Voyager collaboration, the Company recognized IPR&D of $118.1 million during the first six months of 2019, including a $5.0 million payment during the second quarter to obtain rights outside the United States to the Friedreich’s ataxia program. In addition, the Company made an equity investment in Voyager which is required to be marked to market each quarter resulting in an unrealized gain of $21.0 million and $22.6 million for the second quarter and first six months, respectively, and is reflected in Other Income.

Sales, general and administrative (SG&A) expenses increased to $80.8 million for the second quarter of 2019, from $60.9 million for the same period in 2018. For the six months ended June 30, 2019, SG&A expenses increased to $168.4 million, compared to $119.6 million for the same period in 2018. The increase in SG&A expenses for both periods is primarily due to the sales force expansion completed in the third quarter of 2018, the national launch of a patient-focused disease state awareness campaign, Talk About TD, and an increase in the Branded Pharmaceutical Drug fee expense.

As of June 30, 2019, the Company’s cash and available-for-sale investments was $766.5 million.

Updated 2019 SG&A and R&D Expense Guidance

SG&A, IPR&D, and R&D expenses for 2019 are expected to be $658 million to $688 million. Ongoing SG&A and R&D expenses for 2019, excluding IPR&D, are now expected to approximate $540 to $570 million, which compares to the prior SG&A and R&D expense guidance of $550 million to $600 million.

Pipeline Highlights

Opicapone Update

In February 2017, the Company entered 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. Opicapone is a once-daily, oral, selective catechol-O-methyltransferase inhibitor, being developed as an adjunctive therapy to levodopa/carbidopa in patients with Parkinson’s disease experiencing OFF episodes. The Company met with the FDA in January 2018 and based upon the BIPARK-1 and BIPARK-2 pivotal Phase III studies conducted by BIAL, the FDA did not require additional Phase III trials to form an NDA submission. The NDA for opicapone was submitted to the FDA during the second quarter of 2019. The NDA was accepted by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of April 26, 2020. As a result of the FDA’s acceptance of the NDA submission, the Company will pay a $10.0 million milestone payment to BIAL.

Elagolix Update

On July 24, 2018, AbbVie, in collaboration with Neurocrine Biosciences, announced FDA approval and in October 2018 Health Canada approval for ORILISSA (elagolix) for the management of endometriosis with associated moderate to severe pain. The FDA granted priority review to ORILISSA. The FDA grants priority review designation to medicines that, if approved, would provide a significant improvement in the safety or effectiveness of treatment of a serious condition. AbbVie began commercialization of ORILISSA in the United States in August 2018.

AbbVie provided positive top-line efficacy data from two Phase III studies in women with uterine fibroids in the first quarter of 2018 and from the associated six-month safety extension study during the third quarter of 2018. The ELARIS UF-I and UF-II studies of elagolix met all primary and ranked secondary endpoints at month six. Results from these studies will form the basis for an anticipated NDA submission to the FDA for the approval of elagolix in the treatment of uterine fibroids in 2019.

AbbVie intends to initiate a study of elagolix in women with polycystic ovary syndrome (PCOS).

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

The Company began an adaptive, Phase II proof-of-concept study examining the pharmacokinetics, pharmacodynamics, and safety of NBI-74788 in adults with classic 21-hydroxylase deficiency congenital adrenal hyperplasia (CAH) in November 2017. This study evaluates the safety and tolerability of NBI-74788 in patients with CAH together with the relationship between exposure and specific steroid hormone levels in these patients. In March 2019, positive interim results from this ongoing study demonstrated a clinically meaningful reduction in key biomarkers associated with the management of CAH. NBI-74788 was shown to be well tolerated with no serious adverse events reported to date. The Company plans to discuss the NBI-74788 program in adult CAH patients with the FDA during the third quarter of 2019.

In July 2019, the Company initiated an adaptive, Phase II proof-of-concept study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NBI-74788 in pediatric patients with classic CAH.

Voyager Collaboration and VY-AADC Program

During the first quarter of 2019, Neurocrine Biosciences formed a strategic collaboration with Voyager Therapeutics focused on the development and commercialization of Voyager’s 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.

Based on the results from the VY-AADC Phase I programs in Parkinson’s disease, RESTORE-1, a Phase II, randomized, placebo-surgery controlled, double-blinded, multi-center, clinical trial was initiated to evaluate the safety and efficacy of VY-AADC in patients who have been diagnosed with Parkinson’s disease for at least four years, are not responding adequately to oral medications, and have at least three hours of OFF time during the day as measured by a validated self-reported patient diary.

Conference Call and Webcast Today at 4:30 PM Eastern Time

Neurocrine Biosciences will hold a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). Participants can access the live conference call by dialing 866-342-8591 (US) or 203-518-9713 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

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 face, torso, and/or other body parts.

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 from 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

Contraindications
INGREZZA is contraindicated in patients with a history of hypersensitivity to valbenazine or any components of INGREZZA. Rash, urticaria, and reactions consistent with angioedema (e.g., swelling of the face, lips, and mouth) have been reported.

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.

Parkinsonism
INGREZZA may cause Parkinsonism in patients with tardive dyskinesia. Parkinsonism has also been observed with other VMAT2 inhibitors. Reduce the dose or discontinue INGREZZA treatment in patients who develop clinically significant parkinson-like signs or symptoms.

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.

On July 30, 2019 City of Hope reported that it has been named the 11th best cancer hospital in the nation by U.S. News & World Report’s 2019-20 Best Hospitals: Specialty Ranking (Press release, City of Hope, JUL 30, 2019, View Source [SID1234537861]). This makes City of Hope the highest ranked cancer hospital in the West and marks the 13th consecutive year it has been distinguished as one of the nation’s elite cancer hospitals.

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"City of Hope is extremely honored to be recognized by U.S. News & World Report for our long-standing commitment to providing the best treatment and care to our patients," said Robert Stone, City of Hope president and CEO. "It’s a reflection of our exceptional, compassionate care and our dedication to turning powerful science into lifesaving new treatments for our patients."

"A steadfast collaboration between City of Hope scientists and physicians, our strong translational research model and our top-notch biomedical research and manufacturing facilities enable us to deliver excellent cancer care," Stone added.

This year, U.S. News & World Report added data from Hospital Consumer Assessment of Healthcare Providers and Systems, which tracks exemplary patient experience. City of Hope was fourth in the country out of the 885 hospitals surveyed.

City of Hope was also ranked "high-performing" in lung cancer surgery and colon cancer surgery.

"Although City of Hope is not driven by the accolades we receive, we know that patients and their families often turn to rankings and ratings to decide where to get their care," said Michael Caligiuri, M.D., president of City of Hope National Medical Center and the Deana and Steve Campbell Physician-in-Chief Distinguished Chair. "We are gratified to be recognized as a leading-edge cancer center where a patient’s hope can turn into reality through our innovative treatments and exemplary care."

The cancer hospital ranking is compiled based on data from 885 eligible cancer hospitals nationwide; U.S. News & World Report’s rankings cover 16 specialty areas in total. Only 12.5% of the more than 4,500 hospitals analyzed in all the specialty areas received the "Best" status. A cancer hospital’s overall score reflects performance in such areas as advanced technologies, designation as a National Cancer Institute comprehensive cancer center and survival score data.

City of Hope is one of 50 comprehensive cancer centers in the nation, the highest designation possible from the National Cancer Institute and a metric used by U.S. News & World Report. To earn this designation, a center must undergo a rigorous peer-review process, maintain a high number of cancer research projects and be heavily involved in community outreach, as well as meet other academic and public service requirements. City of Hope earned its comprehensive cancer center designation in 1998 and has maintained it since.

City of Hope is also a founding member of the National Comprehensive Cancer Network, making it a leader in advancing research and treatment protocols throughout the nation. City of Hope is currently conducting more than 500 clinical trials, enrolling more than 6,200 patients.

The American College of Surgeons Commission on Cancer recently awarded City of Hope a "Three-Year with Commendation" designation, its highest level of accreditation recognizing exceptional cancer care.

City of Hope was a pioneer in bone marrow and stem cell transplants, and continues to have one of the largest, most successful such programs of its kind in the U.S. The Center for International Blood and Marrow Transplant Research Center has ranked City of Hope as an "overperforming" transplant center, making it the only U.S. transplant center with this recognition for 14 consecutive years. Interlink COE Networks and Programs also recently designated City of Hope’s bone marrow and stem cell transplant program as No. 1 in the nation.

A leader in the CAR T field for being the first to offer CAR T trials for specific targets on glioblastoma and acute myeloid leukemia cells, City of Hope has treated more than 350 patients in CAR T trials and currently has 20 ongoing CAR T and T cell trials.

Complete lists of U.S. News’ "Best Hospitals" by specialties and their ranking methodology are available online beginning July 30 at 12:01 a.m. EDT.

On July 29, 2019 Athenex, Inc. (Nasdaq: ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer, reported that it will release second quarter and six-months ended June 30, 2019 financial results on Wednesday, August 7, 2019, before the market opens, and host a conference call and live audio webcast 8:00am Eastern Time to discuss the financial results and provide a business update (Press release, Athenex, JUL 29, 2019, View Source [SID1234573889]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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To participate in the call, dial 877-407-0784 (domestic) or 201-689-8560 (international) fifteen minutes before the conference call begins and reference the conference passcode 13691069. The live conference call and replay can also be accessed via audio webcast at the Investor Relations section of the Company’s website, located at View Source

On July 29, 2019 OncoImmunity AS reported that it has been acquired by NEC Corporation, the renowned Japanese IT and network company, that recently launched its Artificial Intelligence (AI) driven drug discovery business (Press release, NEC OncoImmunity, JUL 29, 2019, View Source [SID1234555210]). OncoImmunity AS will now become a subsidiary of NEC and operate under the name of NEC OncoImmunity AS.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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OncoImmunity AS, founded in 2014, develops software that identifies neoantigen targets for truly personalized cancer vaccines, cell therapies and optimal patient selection for cancer immunotherapy clinical trials. Prior to the acquisition, OncoImmunity AS was funded by a consortium of Norwegian life science and tech investors led by Radforsk, who played a proactive role in developing the company under the guidance of the company’s Chairman Anders Tuv, who was instrumental for and also managed the sales process on behalf of all shareholders. Under the acquisition, NEC will maintain its focus on drug discovery, while NEC OncoImmunity AS continues its neoantigen prediction services.

Both NEC and OncoImmunity AS share a common vision that individualized cancer treatments can be significantly improved by harnessing the power of AI. AI holds tremendous promise in enabling each and every patient to receive the optimal personalized therapy. OncoImmunity AS will benefit from NEC’s proprietary neoantigen prediction modules and its wide array of IT expertise such as data management and security. The OncoImmunity AS platform has unique advantages in accurately predicting antigen presentation to the tumor cell surface, a key determinant for identifying clinically relevant immunogenic neoantigens. By integrating complementary knowledge and resources, this acquisition makes NEC OncoImmunity AS a forerunner in the field of neoantigen prediction. The acquisition signals positive momentum in the field of personalized cancer immunotherapy, where the prediction of immunogenic neoantigens remains a central challenge for clinical success, with two of the predominant players in the neoantigen prediction field now joining forces.

"This acquisition creates a scientific synergy that will enable OncoImmunity AS’s continued dedication to the development of bioinformatics solutions for truly personalized cancer immunotherapy. The combined technology will be of tremendous benefit to our current and future clinical partners," said Dr. Trevor Clancy, Chief Scientific Officer and Co-founder of OncoImmunity AS.

"The OncoImmunity AS team are delighted to be joining the NEC Group and firmly believe that the union will realize great synergies, marrying the mutual strengths of OncoImmunity AS’s and NEC’s neoantigen prediction pipelines with NEC’s expertise in AI, data management and security. These synergies will help strengthen NEC’s individualized immunotherapy programs, whilst simultaneously positioning NEC OncoImmunity AS as the leading neoantigen prediction service provider in the field," said Dr. Richard Stratford, Chief Executive Officer and Co-founder of OncoImmunity AS.

"NEC strongly believes that healthcare based on genomics paves the way for individualized medicine. OncoImmunity AS is a recognized player in the neoantigen prediction field, and their compelling systems are expected to form valuable synergies with NEC. We are confident that this acceleration of development will provide promising new avenues that lead to better cancer treatments," said Osamu Fujikawa, Senior Vice President, NEC Corporation.