On August 2, 2018 Evotec AG (Frankfurt Stock Exchange: EVT, TecDAX, ISIN: DE0005664809) reported that it will report its financial results for the first half of 2018 on Thursday, 09 August 2018 (Press release, Evotec, AUG 2, 2018, View Source;announcements/press-releases/p/evotec-ag-to-report-first-half-year-2018-results-on-09-august-2018-5707 [SID1234528362]).

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The Company is going to hold a conference call to discuss the results as well as to provide an update on its performance. The conference call will be held in English.

Conference call details

Date: Thursday, 09 August 2018

Time: 02.00 pm CEST (01.00 pm BST/08.00 am EDT)

From Germany: +49 69 22 22 29 043

From France: +33 170 750 705

From Italy: +39 023 601 3806

From UK: +44 20 3009 2452

From USA: +1 855 402 7766

Access Code: 37969784#

A simultaneous slide presentation for participants dialling in via phone is available at View Source

Webcast details

To join the audio webcast and to access the presentation slides you will find a link on our homepage www.evotec.com shortly before the event.

A replay of the conference call will be available for 24 hours and can be accessed in Europe by dialling +49 69 22 22 33 985 (Germany) or +44 20 3426 2807 (UK) and in the USA by dialling +1 866 535 8030. The access code is 654573#. The on-demand version of the webcast will be available on our website: View Source

On August 2, 2018 Emergent BioSolutions Inc. (NYSE: EBS) reported financial results for the quarter and six months ended June 30, 2018 (Press release, Emergent BioSolutions, AUG 2, 2018, View Source;p=RssLanding&cat=news&id=2361890 [SID1234528361])

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Q2 2018 AND RECENT BUSINESS ACCOMPLISHMENTS

Completed Mutual Recognition Procedure for market authorization of BioThrax (Anthrax Vaccine Adsorbed) in five Concerned Member States within the European Union – Italy, the Netherlands, Poland, the U.K. and France; to date, BioThrax has received market authorization in four of the five countries.
Initiated an investment of up to $50 million over the next three years in the Camden fill/finish facility located in Baltimore, an expansion project that will significantly enhance the capabilities of this key site within the Company’s CDMO Business Unit.
Announced Framework Partnering Agreement under which the Company will provide technical and manufacturing support for the development and manufacture of a vaccine against Nipah virus in collaboration with Profectus BioSciences, Inc. and CEPI (Coalition for Epidemic Preparedness Innovations); under a separate agreement with Profectus, Emergent will retain the exclusive option to license and assume control of development activities for the Nipah virus vaccine from Profectus.
Initiated a Phase 1 clinical study of ZIKV-IG, the Company’s anti-Zika virus immune globulin being developed as a therapeutic intervention against Zika virus disease; the candidate was granted Fast Track designation by the U.S. Food and Drug Administration in December 2017.
2018 FINANCIAL PERFORMANCE

(I) Quarter Ended June 30, 2018 (Unaudited)

Revenues

Total Revenues

For Q2 2018, total revenues were $220.2 million, an increase of 118% over 2017. Total revenues reflect a significant increase in product sales.

Product Sales

For Q2 2018, product sales were $180.1 million, an increase of 183% as compared to 2017. The increase is principally attributable to sales of BioThrax and ACAM2000, (Smallpox (Vaccinia) Vaccine Live) previously expected in the first quarter as well as continued sales of both products in the second quarter.

Contract Manufacturing

For Q2 2018, revenue from the Company’s contract manufacturing operations was $23.6 million, an increase of 46% as compared to 2017. The increase primarily reflects manufacturing services at the Company’s Canton site.

Contracts and Grants

For Q2 2018, revenue from the Company’s development-based contracts and grants was $16.5 million, a decrease of 21% as compared to 2017. The decrease primarily reflects a reduction in R&D activities related to certain ongoing funded development programs.

Operating Expenses

Cost of Product Sales and Contract Manufacturing

For Q2 2018, cost of product sales and contract manufacturing was $89.2 million, an increase of 158% as compared to 2017. The increase was primarily attributable to the increase in product sales and contract manufacturing activities at the Company’s Bayview and Canton facilities.

Research and Development (Gross and Net)

For Q2 2018, gross R&D expenses were $24.7 million, a decrease of 4% as compared to 2017. The decrease primarily reflects lower costs associated with contract development services.

For Q2 2018, net R&D expense (calculated as gross research and development expenses minus contracts and grants revenue) was $8.2 million, an increase of $3.4 million as compared to 2017, reflecting increased investment in development-stage programs not currently funded in whole or in part by third-party partners. These include costs associated with the Raxibacumab (Anthrax Monoclonal Antibody) technology transfer and the SIAN device, an intranasal antidote spray device for the treatment of known or suspected acute cyanide poisoning.

Selling, General and Administrative

For Q2 2018, selling, general and administrative expenses were $39.5 million, an increase of 24% as compared to 2017, attributable primarily to increased professional services and compensation-related costs.

Income Taxes

For Q2 2018, the provision for income tax expense in the amount of $15.7 million includes a discrete benefit of $0.9 million primarily related to stock compensation activity resulting in an effective tax rate of 24%. Excluding the discrete benefit, the Q2 2018 effective tax rate was 25%.

Net Income & Adjusted Net Income

For Q2 2018, the Company recorded net income of $50.1 million, or $0.98 per diluted share, versus net income of $4.6 million, or $0.11 per diluted share, in 2017. (1).

For Q2 2018, the Company recorded adjusted net income of $54.7 million, or $1.07 per diluted share, versus adjusted net income of $6.6 million, or $0.13 per diluted share, in 2017. (1) (2)

(I) Six Months Ended June 30, 2018 (Unaudited)

Revenues

Total Revenues

For the six months of 2018, total revenues were $338.0 million, an increase of 55% over 2017. Total revenues reflect a significant increase in product sales.

Product Sales

For the six months of 2018, product sales were $255.8 million, an increase of 76% as compared to 2017. The increase is principally attributable to sales of ACAM2000 and Raxibacumab, both of which were acquired in Q4 2017.

Selling, General and Administrative

For the six months of 2018, selling, general and administrative expenses were $79.7 million, an increase of 19% as compared to 2017, attributable primarily to increased professional services and compensation-related costs.

Income Taxes

For the six months of 2018, the provision for income tax expense in the amount of $11.2 million includes a discrete benefit of $3.2 million primarily related to stock compensation activity resulting in an effective tax rate of 20%. Excluding the discrete benefit, the six months of 2018 effective tax rate was 25%.

Net Income & Adjusted Net Income

For the six months of 2018, the Company recorded net income of $45.2 million, or $0.89 per diluted share, versus net income of $15.1 million, or $0.35 per diluted share, in 2017. (1)

For the six months of 2018, the Company recorded adjusted net income of $53.1 million, or $1.04 per diluted share, versus adjusted net income of $20.8 million, or $0.42 per diluted share, in 2017. (1) (2)

2018 FINANCIAL FORECAST & OPERATIONAL GOALS

The Company is reaffirming its full year 2018 financial performance forecast:

Total Revenue
$715 million to $755 million
Pre-Tax Income
$120 million to $140 million
Net Income (3)
$95 million to $110 million
Adjusted Net Income (2) (3)
$110 million to $125 million
EBITDA (2) (3)
$175 million to $190 million
The Company is also reaffirming its full year 2018 operational goals:

Advance NuThrax development to enable Emergency Use Authorization filing with the FDA in 2018
Complete ACAM2000 deliveries; establish a multi-year follow-on contract with the U.S. government
Deliver Raxibacumab doses under current contract; advance technology transfer to the Company’s Bayview facility in Baltimore, Maryland
Progress pipeline to have at least four product candidates in advanced development
Complete an acquisition that generates revenue within 12 months of closing
Q3 2018 FINANCIAL FORECAST

The Company forecast for Q3 2018 total revenue is $165 million to $190 million.

FOOTNOTES

(1) See "Calculation of Diluted Earnings Per Share."
(2) See "Reconciliation of Net Income to Adjusted Net Income and EBITDA" for a definition of terms and a reconciliation table.
(3) Reflects an estimated tax rate that includes the expected effects of the United States Tax Cuts and Jobs Act of 2017 on the Company’s 2018 income tax provision.

CONFERENCE CALL AND WEBCAST INFORMATION

Company management will host a conference call at 5:00 pm (Eastern Time) today, August 2, 2018, to discuss these financial results. This conference call can be accessed live by telephone or through Emergent’s website:

Live Teleconference Information:
Dial in: [US] (855) 766-6521; [International] (262) 912-6157
Conference ID: 93342423
Live Webcast Information:
Visit View Source for the live webcast feed.
A replay of the call can be accessed at www.emergentbiosolutions.com under "Investors."

ABOUT EMERGENT BIOSOLUTIONS INC.

Emergent BioSolutions Inc. is a global life sciences company seeking to protect and enhance life by focusing on providing specialty products for civilian and military populations that address accidental, intentional, and naturally occurring public health threats. Through our work, we envision protecting and enhancing 50 million lives with our products by 2025. Additional information about the company may be found at www.emergentbiosolutions.com. Follow us on Twitter @emergentbiosolu and Instagram @life_at_emergent.

SAFE HARBOR STATEMENT

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact, including, without limitation, our financial guidance, and any other statements containing the words "will," "believes," "expects," "anticipates," "intends," "plans," "targets," "forecasts," "estimates" and similar expressions in conjunction with, among other things, discussions of the Company’s outlook, financial performance or financial condition, financial and operation goals, strategic goals, growth strategy, acquisition strategy, product sales, government development or procurement contracts or awards, government appropriations, manufacturing capabilities, product development and delivery timeline, and Emergency Use Authorization (EUA) and the timing of other regulatory approvals or expenditures are forward-looking statements. These forward-looking statements are based on our current intentions, beliefs and expectations regarding future events. We cannot guarantee that any forward-looking statement will be accurate. Investors should realize that if underlying assumptions prove inaccurate or unknown risks or uncertainties materialize, actual results could differ materially from our expectations. Investors are, therefore, cautioned not to place undue reliance on any forward-looking statement. Any forward-looking statement speaks only as of the date of this press release, and, except as required by law, we do not undertake to update any forward-looking statement to reflect new information, events or circumstances.

There are a number of important factors that could cause the Company’s actual results to differ materially from those indicated by such forward-looking statements, including the availability of funding and the exercise of options under our BioThrax and NuThrax contracts; appropriations for the procurement of our products; our ability to secure EUA pre-authorization approval and licensure of NuThrax from the FDA within the anticipated timeframe, if at all; availability of funding for our U.S. government grants and contracts; our ability to complete expected deliveries of BioThrax, ACAM2000 and Raxibacumab; our ability to establish a multi-year follow-on contract for ACAM2000; our ability to advance the technology transfer of Raxibacumab to the Company’s Bayview facility; our ability to identify and acquire or in-license products or product candidates that satisfy our selection criteria; our ability to successfully integrate and develop the products or product candidates, programs, operations and personnel of any entities, businesses or products that we may acquire; whether anticipated synergies and benefits from an acquisition or in-license will be realized within expected time periods, if at all; our ability to utilize our manufacturing facilities and expand our capabilities; our ability and the ability of our contractors and suppliers to maintain compliance with Current Good Manufacturing Practices and other regulatory obligations; the results of regulatory inspections; the outcome of the class action lawsuit filed against us and possible other future material legal proceedings; the success of our ongoing and planned development programs; the timing and results of clinical trials; the timing of and our ability to obtain and maintain regulatory approvals for our product candidates; and our commercialization, marketing and manufacturing capabilities and strategy. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our periodic reports filed with the Securities and Exchange Commission, when evaluating our forward-looking statements.

RECONCILIATION OF NET INCOME TO ADJUSTED NET INCOME AND EBITDA

This press release contains two financial measures (Adjusted Net Income and EBITDA (Earnings Before Interest, Taxes, Depreciation and Amortization)) that are considered "non-GAAP" financial measures under applicable Securities and Exchange Commission rules and regulations. These non-GAAP financial measures should be considered supplemental to and not a substitute for financial information prepared in accordance with generally accepted accounting principles. The Company’s definition of these non-GAAP measures may differ from similarly titled measures used by others. Adjusted Net Income adjusts for specified items that can be highly variable or difficult to predict, or reflect the non-cash impact of charges resulting from purchase accounting. EBITDA reflects net income excluding the impact of depreciation, amortization, interest expense and provision for income taxes. The Company views these non-GAAP financial measures as a means to facilitate management’s financial and operational decision-making, including evaluation of the Company’s historical operating results and comparison to competitors’ operating results. These non-GAAP financial measures reflect an additional way of viewing aspects of the Company’s operations that, when viewed with GAAP results and the reconciliations to the corresponding GAAP financial measure, may provide a more complete understanding of factors and trends affecting the Company’s business.

The determination of the amounts that are excluded from these non-GAAP financial measures are a matter of management judgment and depend upon, among other factors, the nature of the underlying expense or income amounts. Because non-GAAP financial measures exclude the effect of items that will increase or decrease the Company’s reported results of operations, management strongly encourages investors to review the Company’s consolidated financial statements and publicly filed reports in their entirety.

On August 2, 2018 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported business highlights and financial results for the second quarter ended June 30, 2018 (Press release, Agios Pharmaceuticals, AUG 2, 2018, View Source [SID1234528359]). In addition, Agios highlighted select corporate milestones and clinical data from its development programs.

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"The first half of 2018 has been productive across all aspects of our business, culminating in the recent approval and launch of our second internally discovered medicine," said David Schenkein, M.D., chief executive officer at Agios. "This achievement sets us well on the path to becoming a sustainable, multiproduct company with a thriving research engine on track to submit its 7th IND and a broad clinical development program with multiple trials planned or underway to expand our oncology and rare genetic disease portfolios."

SECOND QUARTER 2018 HIGHLIGHTS & RECENT PROGRESS

Received full approval from the U.S. Food and Drug Administration (FDA) on July 20, 2018 for TIBSOVO (ivosidenib) for the treatment of patients with relapsed or refractory AML (R/R AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA approved test.
Entered into an exclusive license agreement with CStone Pharmaceuticals to develop and commercialize ivosidenib in Greater China, resulting in a $12 million upfront payment and the potential for $412 million in development and commercial milestones.
Initiated ACTIVATE, a global, placebo-controlled, pivotal trial for mitapivat (AG-348) in approximately 80 adults with PK deficiency who do not receive regular blood transfusions. A second pivotal trial (ACTIVATE-T) in PK deficiency patients who receive regular blood transfusions is ongoing.
Presented new and updated data from the IDH programs at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago. Links to the data presentations, including updated data from the Phase 1 trial combining ivosidenib and azacitidine in the frontline AML setting can be found here.
Secured publication of the ivosidenib Phase 1 data in patients with IDH1m advanced hematological malignancies in theNew England Journal of Medicine.
Supported publication of the results from the Pyruvate Kinase Deficiency Natural History Study in the journal Blood.
Disclosed active research programs in three rare genetic diseases: phenylketonuria, erythroid porphyria and Friedreich’s ataxia, as part of a preclinical pipeline update at the company’s Investor Day in May.
KEY UPCOMING MILESTONES

The company expects to achieve the following remaining milestones in 2018:

Cancer:

Submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for TIBSOVO (ivosidenib) for the treatment of patients with R/R AML and an IDH1 mutation in the fourth quarter of 2018.
Support, in conjunction with Celgene, the initiation of HO150, an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib with standard induction and consolidation chemotherapy in frontline AML patients with an IDH1 or IDH2 mutation in the fourth quarter of 2018.
Rare Genetic Diseases:

Initiate a Phase 2 proof of concept trial of mitapivat (AG-348) in thalassemia in the fourth quarter of 2018.
Research:

Submit an investigational new drug (IND) application for AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies in the fourth quarter of 2018.
EXPECTED FOURTH QUARTER CLINICAL DATA PRESENTATIONS

Updated data from the ongoing Phase 1 combination trial of ivosidenib or enasidenib with standard-of-care intensive chemotherapy in patients with newly diagnosed AML with an IDH2 or IDH1 mutation has been submitted to the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition on December 1-4 in San Diego.
Updated data in untreated AML from the ongoing Phase 1 study of ivosidenib in IDH1m hematologic malignancies has been submitted to ASH (Free ASH Whitepaper).
Updated data in myelodysplastic syndrome (MDS) from the ongoing Phase 1 study of ivosidenib in IDH1m hematologic malignancies has been submitted to ASH (Free ASH Whitepaper).
SECOND QUARTER 2018 FINANCIAL RESULTS

Revenue for the quarter ended June 30, 2018 was $40.4 million, which includes $26.4 million of collaboration revenue and $1.6 million of royalty revenue from net U.S. sales of IDHIFA under our collaboration agreements with Celgene, and $12.4 million of collaboration revenue under our agreement with CStone. Revenue for the quarter ended June 30, 2017 was $11.3 million and consisted solely of collaboration revenue under our agreements with Celgene. The year over year increase in collaboration revenue for the second quarter was primarily driven by the $15.0 million milestone related to Celgene’s filing of an MAA to the EMA for IDHIFA and $12.4 million related to the delivery of the license under the CStone Agreement.

Research and development (R&D) expenses were $86.7 million, including $9.7 million of stock-based compensation expense, for the quarter ended June 30, 2018, compared to $79.8 million, including $8.2 million in stock-based compensation expense, for the comparable period in 2017. The increase in R&D expense was primarily attributable to start-up costs for the mitapivat (AG-348) pivotal program in PK deficiency, including the initiation of the ACTIVATE-T trial. R&D expense also increased as a result of IND enabling activities for AG-636, our DHODH inhibitor.

General and administrative (G&A) expenses were $26.6 million, including $6.8 million of stock-based compensation expense, for the quarter ended June 30, 2018, compared to $16.1 million, including $4.0 million of stock-based compensation expense, for the quarter ended June 30, 2017. The increase in G&A expense was primarily attributable to the growth in our U.S. commercial organization to support the launch of TIBSOVO.

Net loss for the quarter ended June 30, 2018 was $68.7 million, compared to a net loss of $83.1 million for the quarter ended June 30, 2017.

Cash, cash equivalents and marketable securities as of June 30, 2018 were $936.6 million, compared to $567.8 million as of December 31, 2017. The increase in cash was driven by the net proceeds of $516.2 million from the January follow on offering, $8.9 million of cost reimbursements under our collaboration agreements with Celgene and $22.0 million received from employee stock transactions. This was offset by expenditures to fund operations of $178.1 million during the six months ended June 30, 2018.

The company expects that its cash, cash equivalents and marketable securities as of June 30, 2018, together with anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements under our collaboration and license agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2020.

CONFERENCE CALL INFORMATION

Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss second quarter 2018 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 1497883. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On August 2, 2018 Acorda Therapeutics, Inc. (Nasdaq: ACOR) reported its financial and pipeline update for the quarter ended June 30, 2018 (Press release, Acorda Therapeutics, AUG 2, 2018, View Source [SID1234528357]).

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"Our outstanding quarter reflected the continued excellence of our specialty neurology sales force and commercial, patient advocacy and affiliated teams. Our primary focus now is on the approval and launch of INBRIJA, which will benefit from these same capabilities," said Ron Cohen, M.D., Acorda’s President and CEO. "We expect INBRIJA, if approved, to help address the large unmet medical need for the approximately 350,000 people in the U.S. who are challenged by OFF periods related to Parkinson’s disease. Based on our continued market research, we believe the market opportunity for INBRIJA in the U.S. is greater than $800 million."

"The company’s strong execution year to date is fueling our ability to launch INBRIJA, to invest in the ARCUS pipeline and remain well capitalized throughout the INBRIJA launch," Dr. Cohen continued.

Second Quarter 2018 Financial Results

AMPYRA (dalfampridine) Extended Release Tablets, 10 mg – For the quarter ended June 30, 2018, the Company reported AMPYRA net revenue of $150.3 million compared to $131.6 million for the same quarter in 2017.

Research and development (R&D) expenses for the quarter ended June 30, 2018 were $25.9 million, including $1.5 million of share-based compensation compared to $51.2 million, including $3.0 million of share-based compensation for the same quarter in 2017.

Sales, general and administrative (SG&A) expenses for the quarter ended June 30, 2018 were $44.3 million, including $3.7 million of share-based compensation compared to $49.3 million, including $7.8 million of share-based compensation for the same quarter in 2017.

Provision for income taxes for the quarter ended June 30, 2018 was $8.4 million compared to a provision for income taxes of $5.5 million for the same quarter in 2017.

The Company reported GAAP net income of $46.2 million for the quarter ended June 30, 2018, or $0.98 per diluted share. GAAP net loss in the same quarter of 2017 was $8.2 million, or $0.18 per diluted share.

Non-GAAP net income for the quarter ended June 30, 2018 was $65.9 million, or $1.40 per diluted share. Non-GAAP net income in the same quarter of 2017 was $13.3 million, or $0.29 per diluted share. This quarterly non-GAAP net income measure, more fully described below under "Non-GAAP Financial Measures," excludes share-based compensation charges, non-cash interest charges on our debt, changes in the fair value of acquired contingent consideration, and restructuring costs. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At June 30, 2018, the Company had cash, cash equivalents and short-term investments of $391.7 million.

Guidance for 2018

The Company reiterates AMPYRA 2018 net revenue guidance of $330-$350 million.
R&D expenses for the full year 2018 are expected to be $100-$110 million and include manufacturing expenses associated with INBRIJA. This guidance is a non-GAAP projection that excludes share-based compensation, as more fully described below under "Non-GAAP Financial Measures."
SG&A expenses for the full year 2018 are expected to be $170-$180 million. This guidance is a non-GAAP projection that excludes share-based compensation, as more fully described below under "Non-GAAP Financial Measures."
The Company expects to end 2018 with a year-end cash balance in excess of $300 million.
This guidance may be revised with a positive outcome of the pending appeal.
Second Quarter 2018 Updates

INBRIJA (levodopa inhalation powder)
The Company’s Marketing Authorization Application (MAA) for INBRIJA was validated by the European Medicines Agency (EMA) and the application currently is under review. After the adoption of an opinion on the application by the Agency’s Committee for Medicinal Products for Human Use (CHMP), a final decision regarding the MAA will be issued by the European Commission.
In June, the Company presented four INBRIJA abstracts at the 2nd Pan American Parkinson’s Disease and Movement Disorders Congress in Miami. These data were previously presented at the American Academy of Neurology Annual Meeting in April 2018.
AMPYRA Patent Appeal
In June, the oral argument in the AMPYRA patent litigation took place at the U.S. Court of Appeals for the Federal Circuit. The Company is awaiting the Court’s decision.
On July 24, the Federal Circuit denied the Company’s motion for a preliminary injunction to prevent generic at risk launch pending the Court’s decision.
Webcast and Conference Call

Acorda will host a conference call and webcast to review its 2Q18 update and financial results on Thursday, August 2 at 8:30 a.m. ET. To participate in the conference call, dial (866) 393-4306 (domestic) or (734) 385-2616 (international) and reference the access code 4898766. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 11:30 a.m. ET on August 2, 2018 until 11:59 p.m. ET on September 1, 2018. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international); reference code 4898766. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

Non-GAAP Financial Measures

This press release includes financial results prepared in accordance with accounting principles generally accepted in the United States (GAAP), and also certain historical and forward-looking non-GAAP financial measures. In particular, Acorda has provided non-GAAP net income, adjusted to exclude the items below, and has provided 2018 guidance for R&D and SG&A expenses on a non-GAAP basis. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. However, the Company believes the presentation of non-GAAP net income, when viewed in conjunction with our GAAP results, provides investors with a more meaningful understanding of our ongoing and projected operating performance because this measure excludes (i) non-cash compensation charges and benefits that are substantially dependent on changes in the market price of our common stock, (ii) non-cash interest charges related to the accounting for our outstanding convertible debt which are in excess of the actual interest expense owing on such convertible debt as well as non-cash interest charges related to the Fampyra royalty monetization, the asset based loan which was terminated in 2017 and acquired Biotie debt, (iii) changes in the fair value of acquired contingent consideration which do not correlate to our actual cash payment obligations in the relevant periods, (iv) acquisition related expenses and related foreign currency gains that pertain to a non-recurring event, and (v) expenses that pertain to non-routine restructuring events. The Company believes its non-GAAP net income measure helps indicate underlying trends in the Company’s business and is important in comparing current results with prior period results and understanding projected operating performance. Also, management uses this non-GAAP financial measure to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

In addition to non-GAAP net income, we have provided 2018 guidance for R&D and SG&A expenses on a non-GAAP basis. Due to the forward looking nature of this information, the amount of compensation charges and benefits needed to reconcile these measures to the most directly comparable GAAP financial measures is dependent on future changes in the market price of our common stock and is not available at this time. The Company believes that these non-GAAP measures, when viewed in conjunction with our GAAP results, provide investors with a more meaningful understanding of our ongoing and projected R&D and SG&A expenses. Also, management uses these non-GAAP financial measures to establish budgets and operational goals, and to manage the Company’s business and to evaluate its performance.

On August 2, 2018 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, reported its financial results and corporate update for the quarter ended June 30, 2018 (Press release, Ultragenyx Pharmaceutical, AUG 2, 2018, View Source [SID1234528353]).

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"Enthusiasm from the XLH community has resulted in promising commercial uptake of Crysvita in the United States, and momentum continues to grow among both pediatric and adult patients," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In the second half of the year we will continue to focus on the launches of both Crysvita and Mepsevii, and advance the rest of our pipeline including our clinical-stage gene therapy programs with key data readouts expected for both programs."

Financial Results

For the second quarter of 2018, Ultragenyx reported a net loss of $52.7 million, or $1.06 per share, basic and diluted, compared with a net loss for the second quarter of 2017 of $72.9 million, or $1.72 per share, basic and diluted. The loss for the second quarter of 2018 includes a $40.3 million gain from Ultragenyx’s portion of the sale of the priority review voucher (PRV) received with the Crysvita (burosumab) approval. For the six months ended June 30, 2018, net loss was $22.5 million, or $0.46 per share, basic and diluted, compared with a net loss for the same period in 2017 of $141.2 million, or $3.35 per share, basic and diluted. In addition to the Crysvita PRV, the loss from the first six months also includes the sale of the Mepsevii (vestronidase alfa) PRV in January 2018 for net proceeds of $130.0 million. The net loss for the first six months of 2018 reflected cash used in operations of $165.6 million compared to $110.0 million for the same period in 2017.

Net Revenues

For the second quarter of 2018, Ultragenyx reported $12.8 million in total revenue. Ultragenyx recognized $8.9 million in revenue from the research agreement with Bayer. For Crysvita, Ultragenyx recognized $1.6 million in profit sharing and royalty revenue from the collaboration and license agreement with Kyowa Hakko Kirin. This includes $1.1 million in collaboration revenue in the U.S profit share territory, where Crysvita became commercially available on April 27, 2018, as well as $0.5 million in royalty revenue in the European territory, where Crysvita received conditional marketing authorization on February 23, 2018. There were nominal net product sales for Crysvita in other regions. Mepsevii product revenue for the second quarter of 2018 was $2.0 million, and UX007 named patient revenue was $0.2 million.

Operating Expenses

Total operating expenses for the second quarter of 2018 were $107.7 million compared with $78.4 million for the same period in 2017, including non-cash stock-based compensation of $19.6 million and $16.8 million in the second quarter of 2018 and 2017, respectively. Total operating expenses for the six months ended June 30, 2018 were $214.9 million compared with $148.4 million for the same period in 2017, including non-cash stock-based compensation of $38.4 million and $31.3 million in the first six months of 2018 and 2017, respectively. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows and advances its pipeline.

Cash, cash equivalents, and investments

Cash, cash equivalents, and investments were $547.1 million as of June 30, 2018.

Recent Highlights

Crysvita in X-Linked Hypophosphatemia (XLH)

Positive data from the Phase 3 pediatric study demonstrated that Crysvita was superior to oral phosphate and active vitamin D (conventional therapy) in improving rickets in children with XLH after 40 weeks of treatment.
Mepsevii in mucopolysaccharidosis VII (MPS VII)

In Europe, Mepsevii received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP), recommending the marketing authorization under exceptional circumstances of Mepsevii for the treatment of non-neurological manifestations of MPS VII. A decision from the European Commission, which has the authority to approve medicines for the European Union, is expected in the third quarter of 2018.
UX007 in long-chain fatty acid oxidation disorders (LC-FAOD)

Discussions are ongoing with FDA and EMA to determine the acceptability of filing UX007 for the treatment of LC-FAOD based on the totality of currently available data. The data from the Phase 2 study for UX007 show a significant reduction in major clinical events; however, the FDA continues to believe that the data are confounded and are not sufficient to support a New Drug Application (NDA). We continue to pursue potential filings with FDA and EMA based on the current data and expect to conclude discussions in the second half of 2018. These discussions also should provide further clarity regarding whether an additional study would be required for approval.
DTX401 gene therapy in glycogen storage disease type Ia (GSDIa)

The first patient has been dosed in the Phase 1/2 study of DTX401, our adeno-associated virus 8 (AAV8) gene therapy program for the treatment of patients with GSDIa. Data from the three-patient first dose cohort are expected in the second half of 2018.
The U.S. FDA granted fast-track designation to DTX401 for the treatment of GSDIa. This designation is designed to facilitate the development and expedite the review of drugs that are intended to treat serious conditions and fill an unmet medical need, and it allows for more frequent interaction with the FDA review team. It also enables eligibility for priority review if relevant criteria are met and the potential for a rolling review of the Biologic License Application (BLA) as data become available.
Upcoming Key Milestones

Crysvita (burosumab) in tumor-induced osteomalacia (TIO)

Data from all patients in the Phase 2 study in TIO are expected in the second half of 2018. This is an open label Phase 2 study evaluating the safety and efficacy of burosumab in adult patients with TIO.
UX007 in LC-FAOD and glucose transporter type-1 deficiency syndrome (Glut1 DS)

In LC-FAOD, additional clarity from FDA and EMA on the regulatory pathway is expected in the second half of 2018.
The fully-enrolled Phase 3 movement disorder study in patients with Glut1 DS is on track and data are expected in second half of 2018.
DTX301 gene therapy in ornithine transcarbamylase (OTC) Deficiency

Results from the fully-enrolled cohort 2 of the Phase 1/2 study are expected in second half of 2018.
DTX401 Gene Therapy in GSDIa

Data from the first, lowest dose cohort are expected in the second half of 2018.
Conference Call & Webcast Information

Ultragenyx will host a conference call today, Thursday, August 2, 2018 at 5pm ET to discuss second quarter 2018 financial results and to provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial 855-797-6910 (USA) or 262-912-6260 (international) and enter the passcode 2895644. The replay of the call will be available for one year.