On November 5, 2018 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the third quarter ending September 30, 2018 (Press release, PTC Therapeutics, NOV 5, 2018, View Source [SID1234530863]).

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"We have been aggressively pursuing our vision to build a leading, fully integrated, multiplatform biotech company," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "The addition of gene therapy aligns with our goal of developing treatments for more patients with rare disorders and the in-licensing of Tegsedi and Waylivra leverages our commercial expertise. Over the last 20 years, our desire to bring new therapeutics to patients has been based on scientific innovation and we are continuing that mission."

Third Quarter Financial Highlights:
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Total revenues for the third quarter of 2018 were $53.6 million, compared to $41.9 million in the same period in 2017. The change in total revenue was primarily a result of revenue from Emflaza, which launched in May 2017.
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Translarna net product revenues were $30.4 million for the third quarter of 2018, compared to $32.0 million reported in the third quarter of 2017.
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Emflaza net product revenues were $22.6 million for the third quarter of 2018, compared to $9.8 million reported in the third quarter of 2017.
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GAAP R&D expenses were $54.4 million for the third quarter of 2018, compared to $30.0 million for the same period in 2017. Non-GAAP R&D expenses were $49.9 million for the third quarter of 2018, excluding $4.4 million in non-cash, stock-based compensation expense, compared to $26.4 million for the same period in 2017, excluding $3.6 million in non-cash, stock-based compensation expense. The increase in GAAP and non-GAAP R&D expense was primarily due to increased investment in research programs and advancement of the clinical pipeline, as well as the Akcea Therapeutics, Inc. ("Akcea") upfront licensing fee of $12 million paid during the third quarter of 2018.
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GAAP SG&A expenses were $38.4 million for the third quarter of 2018, compared to $31.4 million for the same period in 2017. Non-GAAP SG&A expenses were $33.9 million for the third quarter of 2018, excluding $4.5 million in non-cash, stock-based

compensation expense, compared to $27.9 million for the same period in 2017, excluding $3.5 million in non-cash, stock-based compensation expense. The increase in GAAP and non-GAAP SG&A expense was primarily due to continued investment in commercial activities for Emflaza and Translarna, as well as $1.5 million in expenses related to PTC’s acquisition of Agilis Biotherapeutics, Inc.
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Net loss for the third quarter of 2018 was $51.0 million, compared to a net loss of $33.7 million for the same period in 2017.
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Cash, cash equivalents, and marketable securities totaled approximately $249.4 million at September 30, 2018, compared to approximately $191.2 million at December 31, 2017.
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Shares issued and outstanding as of September 30, 2018 were 50.4 million.

2018 Guidance:
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PTC now anticipates full year 2018 net product revenues to be between $260 and $280 million, a decrease in the high-end range of its prior guidance of between $260 and $295 million. PTC reiterates Translarna net product revenue for the full year 2018 to be between $170 and $185 million. PTC projects a 5-year (December 31, 2022) compound annual growth rate of 15% for net product revenues, representing continued strong growth year-over-year by increasing penetration in current countries and pursuing opportunities for label expansion. PTC now anticipates full year 2018 Emflaza net product revenue to be between $90 and $95 million, a decrease in the high-end range of its prior guidance of between $90 and $110 million.
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GAAP R&D and SG&A expense for the full year 2018 are now anticipated to be between $315 and $325 million, an increase from PTC’s prior guidance of between $280 and $290 million. The increase in anticipated full year 2018 GAAP R&D and SG&A expense is primarily due to increased spend related to the Agilis acquisition and the Akcea upfront licensing fee of $12 million paid during the third quarter.
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Non-GAAP R&D and SG&A expense for the full year 2018 is now anticipated to be between $280 and $290 million, excluding estimated non-cash, stock-based compensation expense of approximately $35 million, an increase from PTC’s prior guidance of between $250 and $260 million, excluding estimated non-cash, stock-based compensation expense of approximately $30 million.

Key Third Quarter and Other Corporate Highlights:
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Completed acquisition of Agilis Biotherapeutics adding a Central Nervous System (CNS) gene therapy platform. Acquisition included three programs in rare CNS disorders including Aromatic L-Amino Acid Decarboxylase (AADC), Friedreich Ataxia and Angelman Syndrome. PTC plans to file a biologics license application (BLA) in AADC in 2019. Pre-commercial efforts, such as patient identification efforts are ongoing. PTC estimates that there are 5,000 AADC deficiency patients worldwide with 1,200 patients in the United States. In addition, PTC plans to file an investigational new drug application (IND) in Friedreich Ataxia in 2019.

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PTC in-licensed Latin America commercial rights to Tegsedi and Waylivra leverages strong commercial expertise. Tegsedi has been approved in the United States, European Union, and Canada for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). The polyneuropathic form of hATTR, occurs more frequently in individuals of Portuguese ancestry, where PTC estimates approximately 6,000 patients in Latin America are affected. PTC has started patient identification efforts and plan to submit an application for Tegsedi with ANVISA, the Brazilian regulatory authority in the first half of 2019.
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Initial STRIDE registry data demonstrates that Translarna delays loss of ambulation. Preliminary data from the first international drug registry for Duchenne patients receiving Translarna demonstrated participants continuing to walk years longer and are remaining more physically able than untreated children. The data confirms Translarna’s long-term clinical benefit in delaying irreversible muscle loss in patients and was presented to experts at the 23rd International Annual Congress of the World Muscle Society.
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Pursing label expansion with European Medicines Agency (EMA) for Translarna for non-ambulatory patients. In the third quarter, PTC filed for an extension of its existing label for Translarna to include non-ambulatory patients. The EMA has validated the application and the regulatory process is ongoing.
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Continued advancement of the spinal muscular atrophy (SMA) program. Data demonstrating the clinical benefits of risdiplam in all types of SMA were presented at the 23rd International Annual Congress of the World Muscle Society. Babies from FIREFISH Part 1 study showed increased functional developmental milestones including sitting. The pivotal portion of FIREFISH is enrolling. Clinical data was presented for the first time for the Type 2 & 3 patients from the open label portion of SUNFISH demonstrating a median 3-point increase in motor function score which was supported by the increase of SMN protein measured in the blood. The pivotal portion of SUNFISH Part 2 study in Type 2 & 3 patients has completed enrollment.
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Development in oncology program with two clinical advancements. PTC initiated a Phase 1 study evaluating the safety of PTC596 in patients with diffuse intrinsic pontine glioma (DIPG). Additionally, the PTC299 study is now actively enrolling patients in acute myeloid leukemia (AML).

Non-GAAP Financial Measures:
In this press release, the financial results and financial guidance of PTC are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude stock-based compensation expense. This non-GAAP financial measure is provided as a complement to financial measures reported in GAAP because management uses this non-GAAP financial measure when assessing and identifying operational trends. In management’s opinion, this non-GAAP financial measure is useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company’s future outlook. Quantitative reconciliations of non-GAAP

financial measures to their closest equivalent GAAP financial measures are included in the tables below.

Today’s Conference Call and Webcast Reminder:
Today’s conference call will take place at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 2477754. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. The accompanying slide presentation will be posted on the investor relations section of the PTC website. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for two weeks.

On November 5, 2018 Sunesis Pharmaceuticals, Inc. (Nasdaq: SNSS) reported financial results for the quarter ended September 30, 2018 (Press release, Sunesis, NOV 5, 2018, View Source [SID1234530858]). Loss from operations for the three and nine months ended September 30, 2018 was $6.3 million and $20.0 million. As of September 30, 2018, cash and cash equivalents totaled $20.2 million. This capital is expected to fund the company into the second quarter of 2019.

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"We remain focused on advancing our non-covalent BTK inhibitor vecabrutinib to help patients who have developed resistance to covalent BTK inhibitors such as ibrutinib," said Dayton Misfeldt, Interim Chief Executive Officer of Sunesis. "We continue to learn more about this unique asset and its market opportunity and will share an update at the ASH (Free ASH Whitepaper) Annual Meeting in December. As announced last week, one of our accepted abstracts for presentation at ASH (Free ASH Whitepaper) includes an update on the Phase 1b/2 trial of vecabrutinib, and as presented in the published abstract, the pharmacokinetic profile is consistent with the results from our Phase 1a study. We continue to believe that 100mg to 300mg will be the potentially active dose levels. Thus far, vecabrutinib appears well tolerated in the context of advanced disease. We continue with the dose escalation part of the trial and look forward to sharing a complete clinical update at the meeting next month and at a company-sponsored webcast event concurrent with the meeting."

Recent Highlights

Announced Presentations at ASH (Free ASH Whitepaper) Annual Meeting. In November 2018, the Company announced that three presentations will be made at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting to be held December 1-4, 2018 in San Diego, California. Among the abstracts is an update on the Phase 1b/2 trial of vecabrutinib, titled "Preliminary Safety, Pharmacokinetic, and Pharmacodynamic Results from a Phase 1b/2 Dose-Escalation and Cohort-Expansion Study of the Noncovalent, Reversible Bruton’s Tyrosine Kinase Inhibitor (BTKi), Vecabrutinib, in B-Lymphoid Malignancies," (Publication 3141) which will be presented on Sunday, December 2, in a session titled "CLL: Therapy, excluding Transplantation: Poster II," (Session 642) from 6:00-8:00pm at the San Diego Convention Center, Hall GH. The other poster, titled "Vecabrutinib Is Efficacious In Vivo in a Preclinical CLL Adoptive Transfer Model" will be presented on Saturday, December 1, and an oral presentation "High Prevalence of BTK Mutations on Ibrutinib Therapy after 3 Years of Treatment in a Real-Life Cohort of CLL Patients: A Study from the French Innovative Leukemia Organization (FILO) Group" will be presented in sessions on Monday, December 3. The posters will be available on the Sunesis website following the presentations.

Expanded Clinical Trial Sites. In the third quarter, we added three additional clinical sites to our Phase 1b/2 trial: Memorial Sloan Kettering Cancer Center, Moffitt Cancer Center and University California San Diego. We continue to identify and prepare for adding additional sites as we continue dose escalation and prepare for the Phase 2 expansion portion of the study.

Financial Highlights

Cash and cash equivalents totaled $20.2 million as of September 30, 2018, as compared to $31.8 million in cash, cash equivalents, and marketable securities as of December 31, 2017. This capital is expected to fund the company into the second quarter of 2019. The nine-month decrease of $11.6 million was primarily due to $17.9 million of net cash used in operating activities, partially offset by $6.3 million in net cash flows from financing activities.

Research and development expense was $3.6 million and $11.3 million for the three and nine months ended September 30, 2018, as compared to $6.8 million and $17.9 million for the same periods in 2017, primarily relating to the vecabrutinib and the vosaroxin development program in each period. The decreases of $3.2 million and $6.6 million between the comparable periods from last year was primarily due to a $2.5 million milestone payment made during the third quarter of 2017 to Biogen under the license agreement, a decrease in salary and personnel expenses, a decrease in professional services, and clinical trial expenses related to higher expenses incurred in 2017 due to the MAA with the EMA.

General and administrative expense was $2.7 million and $8.9 million for the three and nine months ended September 30, 2018, as compared to $3.2 million and $10.8 million for the same periods in 2017. The decreases of $0.5 million and $1.9 million between the comparable periods in 2017 were primarily due to reduced professional services, personnel, and commercial expenses.

Interest expense was $0.3 million and $0.9 million for the three and nine months ended September 30, 2018, as compared to $0.3 million and $1.1 million for the same periods in 2017. The decrease during the nine months period was primarily due to the decrease in the outstanding notes payable.

Cash used in operating activities was $17.9 million for the nine months ended September 30, 2018, as compared to $30.8 million for the same period in 2017. Net cash used in the 2018 periods resulted primarily from the net loss of $20.6 million, partly offset by net adjustments for non-cash items of $2.3 million and changes in operating assets and liabilities of $0.4 million. Net cash used in the 2017 period resulted primarily from the net loss of $28.8 million and changes in operating assets and liabilities of $4.6 million, partly offset by net adjustments for non-cash items of $2.6 million.

Loss from operations was $6.3 million and $20.0 million for the three and nine months ended September 30, 2018, as compared to $9.9 million and $28.0 million for the

On November 5, 2018 INSYS Therapeutics, Inc. (NASDAQ: INSY), a leader in the development, manufacture and commercialization of pharmaceutical cannabinoids and spray technology, reported financial results for its third quarter ended Sept. 30, 2018 (Press release, Insys Therapeutics, NOV 5, 2018, View Source [SID1234530857]).

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OVERALL HIGHLIGHTS

Commenced a strategic alternatives review process for the company’s opioid-related assets

Expanded collaborative partnership with University of California San Diego’s Center for Medicinal Cannabis Research (CMCR) to study the company’s cannabidiol (CBD) oral solution in two additional disease states (early psychosis and anxiety in anorexia nervosa)

Achieved net revenue of $18.3 million in the third quarter of 2018

Advanced R&D programs with a $14.5 million investment in the third quarter of 2018:

Received "Fast Track" designation from the FDA for epinephrine nasal spray as an investigational treatment for anaphylaxis

Continued enrollment of three company-sponsored CBD clinical studies:

childhood absence epilepsy (Phase 2)

Prader-Willi syndrome (Phase 2)

infantile spasms (Phase 3)

Completed pharmacokinetic study of dronabinol inhalation

Signed definitive licensing agreement with Lunatus to commercialize SUBSYS in the Middle East

Received FDA approval of supplemental NDA for SYNDROS to expand label, enabling use of the product with feeding tubes for patients with cancer and AIDS

Appointed Elizabeth Bohlen to the Board of Directors and added Mark Nance to the management team as chief legal officer and general counsel

Announced settlement agreement in principle with Department of Justice consistent with previous public statement and disclosures

Optimized commercial organization and related support functions to control operating costs in line with lower revenue

Received confirmation from the SEC that it concluded its investigation of the company, and does not intend to recommend an enforcement action

"Our commitment to further establish our position as a leader in pharmaceutical cannabinoids and spray technology was exemplified by the achievement of several milestones in the third quarter," said Saeed Motahari, president and chief executive officer of INSYS Therapeutics. "We continued to make progress on our pipeline and expanded our collaborative partnerships with leading research institutions, increasing the number of CBD clinical studies in which we’re the sole sponsor or a key collaborator to seven. Furthermore, our 99.5 percent pure pharmaceutical-grade CBD oral solution allows INSYS to meet the needs of clinical study patients and become a strategic partner across our industry and in the medical community."

Motahari concluded, "The decline in the overall TIRF market continues to impact sales of our primary commercial product, SUBSYS. Our proactive efforts to expand managed care access and educate appropriate HCPs have enabled us to maintain our leading share of the branded TIRF market. However, this decline in revenue has required us to contain costs and as a result, we have taken actions to adjust our commercial organization to align with the realities of the market. We will continue to be disciplined with our cost structure, while appropriately investing in our pipeline, which is the key to transforming INSYS into a leader in pharmaceutical cannabinoids and spray technology."

Financial & Operating Highlights

During the preparation of the consolidated financial statements as of and for the quarter ended Sept. 30, 2018, the company identified errors that impacted the 2017 financial information, which have been revised for the correction of this error.

The company had $113.0 million in cash, cash equivalents and short-term and long-term investments with no debt outstanding as of Sept. 30, 2018

Net revenue for the third quarter of 2018 was $18.3 million, compared to $30.7 million for the third quarter of 2017, driven primarily by declines in the TIRF market

Gross margin was 87.0 percent for the third quarter of 2018, compared to 75.6 percent in the same period of 2017

Sales and marketing investment was $7.4 million for the third quarter of 2018, compared to $12.8 million for the third quarter of 2017, as the company took action to better align its commercial organization to the lower revenue base

Research and development investment decreased to $14.5 million for the third quarter of 2018, compared to $19.6 million for the third quarter of 2017, primarily as a result of the timing of clinical trials

General and administrative expense of $8.9 million for the third quarter of 2018 declined compared to $11.3 million in the third quarter of 2017, as the company continued to look for opportunities to level-set its cost base

Legal expense increased to $16.0 million for the third quarter of 2018, compared to $4.4 million in the third quarter of 2017, as a result of the company’s legal proceedings, including

expenses associated with indemnification of former executives in connection with their pending trial, which constitutes approximately 60 percent of the total Q3 2018 expense. Management is disputing the reasonableness of certain of these indemnification-related expenses

Income tax expense was $240 thousand for the third quarter of 2018 compared to a benefit of ($9.0 million) during the revised third quarter of 2017

Net loss for the third quarter of 2018 was ($30.6 million), or ($0.41) per basic and diluted share, compared to a net loss of ($166.3 million), or ($2.28) per basic and diluted share, for the revised third quarter of 2017. Adjusted net loss for the third quarter of 2018 was ($0.37) per basic and diluted share

Adjusted EBITDA loss for the third quarter of 2018 was ($26.0 million), compared to Adjusted EBITDA loss of ($18.4 million) in the prior-year quarter. The reconciliation of net income to Adjusted EBITDA is included at the end of this news release

During the preparation of the consolidated financial statements as of and for the quarter ended Sept. 30, 2018, the company identified errors within the company’s consolidated financial statements for the year ended Dec. 31, 2017, resulting from the company’s adoption of ASU 2016-09 "Compensation – Stock Compensation (Topic 718): Improvements to Employee Share-Based Payment Accounting" on Jan. 1, 2017, which impacted the previously filed financial statements for the quarters ended March 31, June 30, and Sept. 30 of 2017 as well as the audited financial statements for the year ended Dec. 31, 2017. In addition, we identified an error in the calculation of our weighted average shares outstanding, which impacted the previously filed financial statements for the three and nine months ended Sept. 30, 2017, and the year ended Dec. 31, 2017. The company evaluated the errors and concluded that they were not material to the financial statements previously issued. Accordingly, the Dec. 31, 2017 financial information has been revised to include a cumulative adjustment of $2.9 million to correctly present uncertain tax position liabilities and accumulated deficit, resulting in a reduction to total liabilities and an increase in stockholders’ equity. These revisions will be reflected and disclosed in the company’s Form 10-Q for the period ended Sept. 30, 2018 to be filed with the SEC on or before Nov. 9, 2018. Additionally, in order to correctly present income tax benefit and net loss in the appropriate periods for comparative purposes, the financial results for the three and nine months ended Sept. 30, 2017, have been revised to include adjustments of $40,000 and $120,000, respectively. These revisions resulted in net loss per share for the three and nine months ended Sept. 30, 2017, and the year ended Dec. 31, 2017, changing from $2.30 to $2.28, $2.51 to $2.50 and $3.16 to $3.12, respectively.

Webcast Information

A conference call is scheduled for 5:00 p.m. Eastern Standard Time on Nov. 5, 2018, to discuss the financial and operational results for the third quarter 2018. Interested parties can listen to the call live as it occurs via the company’s website, View Source, on the Investors section’s Presentations & Events page; or by dialing 844-263-8304 (from inside the U.S.) or 213-358-0958 (from outside the U.S.), and using the Conference ID 6149699. A webcasted replay of the call will be available on the site a few hours after the event.

On November 5, 2018 Neurocrine Biosciences, Inc. (NASDAQ: NBIX) reported its financial results for the quarter ended September 30, 2018, and provided an update on the launch of INGREZZA (valbenazine) and its clinical development programs (Press release, Neurocrine Biosciences, NOV 5, 2018, View Source [SID1234530824]).

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"We remain committed to delivering hope for patients with serious unmet medical needs as evidenced by the growing adoption of INGREZZA and the recent commercial launch of ORILISSA – both treatments were discovered at Neurocrine Biosciences," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "With the recently completed salesforce expansion, we continue to focus on educating healthcare providers on the benefits of INGREZZA to treat patients with tardive dyskinesia. We have also made great progress in our research and development programs with the advancement of two new internally discovered compounds into the clinic and our research collaboration with Jnana Therapeutics to develop new medicines to treat neurological diseases."

Financial Results

Total revenue for the three and nine months ended September 30, 2018 was $151.8 million and $319.7 million, respectively, compared to $60.8 million and $67.1 million for the same periods in 2017. Total revenues were comprised of the following (in thousands):

INGREZZA was made available for commercial distribution on May 1, 2017 and ORILISSA (elagolix) was approved by the U.S. Food and Drug Administration (FDA) during the third quarter of 2018 with AbbVie sales beginning in August 2018. With the FDA approval of ORILISSA, the Company recognized a $40 million event-based milestone as revenue under the Company’s collaboration agreement with AbbVie during the third quarter of 2018. During the third quarter of 2017, Mitsubishi Tanabe initiated a pivotal trial of INGREZZA in Asia for the treatment of tardive dyskinesia which generated a $15 million milestone.

For the third quarter of 2018, the Company reported net income of $50.8 million, or $0.52 diluted net income per share, compared to a net loss of $11.1 million, or $0.13 net loss per share, for the same period in 2017. For the nine months ended September 30, 2018, the Company reported net income of $3.0 million, or $0.03 diluted net income per share, compared to a net loss of $149.4 million, or $1.70 net loss per share, for the same period in 2017. The increase in income across both periods is due to increased INGREZZA net product sales and the $40 million event-based milestone from AbbVie.

Research and development (R&D) expenses were $35.5 million during the third quarter of 2018, compared to $22.5 million for the third quarter of 2017. For the nine months ended September 30, 2018, R&D expenses were $121.4 million, compared to $96.2 million for the same period last year. The increase in R&D expenses across both periods is principally due to expanded research efforts, the advancement of the Company’s clinical programs including Tourette syndrome, congenital adrenal hyperplasia (CAH), and the two new programs announced today, as well as activities to prepare for the Company’s intended opicapone New Drug Application (NDA) submission in the second quarter of 2019.

Sales, general and administrative (SG&A) expenses increased to $60.4 million for the third quarter of 2018 from $43.9 million for the third quarter of 2017. For the nine months ended September 30, 2018, SG&A expenses were $180.0 million, compared to $113.6 million for the same period last year. The increase in SG&A expense for both periods is primarily due to the hiring of the Company’s sales force, including the third quarter 2018 sales force expansion, and commercialization activities for INGREZZA, which launched in the third quarter of 2017.

The Company’s balance sheet at September 30, 2018 reflected total assets of $933.7 million, including cash and investments of $820.6 million, compared with total assets at December 31, 2017 of $817.6 million.

Updated 2018 Expense Guidance

Ongoing operating expenses for 2018 are now expected to approximate $410 million to $420 million, which compares to the prior operating expense guidance of $395 million to $420 million. The operating expense guidance range includes the clinical pipeline expansion and the recently announced research collaboration with Jnana Therapeutics.

Pipeline Highlights

INGREZZA (valbenazine) 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.

Valbenazine 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 the fourth quarter of 2017, the Company initiated T-Force GOLD, a Phase IIb study of valbenazine in pediatric patients with Tourette syndrome. This study is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, which will evaluate the safety, tolerability, efficacy and optimized dosing 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 comparison of the change from baseline of the Yale Global Tic Severity Scale between placebo and active treatment groups at the end of week 12. Top-line data are expected in December 2018.

In the second quarter of 2018, the Company commenced enrollment into the open label extension study, T-Force GOLD Plus, for pediatric patients with Tourette syndrome. Patients who complete participation in the T-Force GOLD study are eligible to roll over into participation in this open label extension study for an additional six months of treatment with optimized doses of valbenazine. The study will collect longer-term safety and tolerability data in children and adolescents as well as providing useful information about the maintenance of efficacy in these patients over the six months period of dosing.

In the second quarter of 2018, the Company started T-Force PLATINUM, a double-blind, placebo-controlled, randomized withdrawal study of valbenazine in pediatric patients with Tourette syndrome. This study is designed to evaluate longer term efficacy and safety in patients who initially responded to open-label therapy with optimized doses of valbenazine. Approximately 180 patients will participate in the study with top-line data expected in late 2019.

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 Pharma Corporation (MTPC). In 2017, MTPC initiated a pivotal trial of INGREZZA in Japan for the treatment of tardive dyskinesia.

ORILISSA (elagolix) Update

On July 24, 2018, AbbVie, in collaboration with Neurocrine, announced FDA approval and in October 2018 Health Canada approval for ORILISSA 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 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. These replicate Phase III studies were randomized, parallel, double-blind, placebo-controlled clinical trials evaluating elagolix alone or in combination with low-dose hormone (add-back) therapy in women with heavy uterine bleeding associated with uterine fibroids. The studies enrolled approximately 400 patients each for an initial six-month placebo-controlled dosing period. At the end of the six months of placebo-controlled evaluation, patients were eligible to enter an additional six-month safety extension study. The primary efficacy endpoint of the study was an assessment of the change in menstrual blood loss utilizing the alkaline hematin method comparing baseline to month six. Additional secondary efficacy endpoints were evaluated including the change in fibroid volume and hemoglobin. Bone mineral density was assessed via dual-energy x-ray absorptiometry (DEXA) scan at baseline, at the conclusion of dosing, and at six months post-dosing. Results from these studies will form the basis for an anticipated 2019 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 – Portela & CA, S.A. (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 and based upon the BIPARK-I and BIPARK-II pivotal Phase III studies conducted by BIAL, the FDA did not require additional Phase III trials to form an NDA submission. The Company is in the process of preparing for an NDA submission which it anticipates will occur during the second quarter of 2019.

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

In the second quarter of 2017, the Company successfully completed the Phase I, Investigational New Drug (IND)-opening study of NBI-74788 in healthy volunteer participants. The study was a randomized, open-label, two-period crossover study to evaluate the pharmacokinetics, the effect of food on pharmacokinetics, 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 pharmacokinetics, 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 patients. The Company recently expanded this study to include additional patients to further optimize dosing flexibility and convenience. Data are expected during the first quarter of 2019.

New VMAT2 Inhibitor

The Company has filed an IND and initiated a Phase I study for a novel, internally discovered vesicular monoamine transporter 2 (VMAT2) inhibitor with potential use in the treatment of several neurology and/or psychiatry disorders. The initial randomized, double-blind, single ascending dose study to evaluate the safety, tolerability, and pharmacokinetic profile of the compound in healthy participants is anticipated to be completed during the fourth quarter of 2018.

New CNS Compound

The Company has filed an IND and completed dosing in a Phase I single ascending dose study for an internally discovered first-in-class central nervous system (CNS) compound with potential use in the treatment of several neurology and/or psychiatry disorders. This study is a randomized, double-blind, single ascending dose study to evaluate the safety, tolerability, and pharmacokinetic profile of the compound in healthy participants. The Company is currently analyzing the data from this study to inform the design of future clinical studies for the program.

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

Neurocrine 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 877-876-9176 (US) or 785-424-1667 (International) using the conference ID: NBIX. The webcast and supplemental information in the form of a slide presentation can also be accessed on Neurocrine’s website under Investors at www.neurocrine.com. The webcast and slide presentation will be archived for approximately one month.

About INGREZZA (valbenazine) Capsules

INGREZZA, a selective vesicular monoamine transporter 2 (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 or functional inhibition 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.

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 November 5, 2018 Cambrex Corporation (NYSE: CBM), the leading manufacturer of small molecule innovator and generic Active Pharmaceutical Ingredients (APIs), and finished dosage forms, reported that third quarter 2018 financial results will be released on Thursday, November 8, 2018 before the market opens (Press release, Cambrex, NOV 5, 2018, View Source [SID1234530775]).

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The Company will host a conference call to discuss the financial results.

Third Quarter 2018 Earnings Conference Call
When: Thursday, November 8, 2018 at 8:30 a.m. Eastern Time
Dial-in: 1-877-830-2649 for U.S.
+1-785-424-1824 for International
Passcode: 3109072
Dial-in Replay: 1-888-203-1112 for U.S.
+1-719-457-0820 for International
Passcode: 3109072
Available through Thursday, November 15, 2018
Webcast: www.cambrex.com