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.

On August 2, 2018 Zymeworks Inc. (NYSE/TSX: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, reported that management will present at the upcoming Canaccord Genuity 38th Annual Growth Conference taking place August 8-9, 2018 in Boston, MA, USA (Press release, Zymeworks, AUG 2, 2018, View Source [SID1234528351]).

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The Company will provide a corporate update and present upcoming catalysts on Wednesday, August 8, 2018 at 2:00 p.m. ET. Interested parties can access a live webcast of the presentation via a link from Zymeworks’ website at View Source, which will also host a recorded replay available afterwards.

On August 2, 2018 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its consolidated financial results for the second quarter 2018 and highlighted recent progress in advancing its pipeline (Press release, Alnylam, AUG 2, 2018, View Source [SID1234528348]).

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"The second quarter and recent period mark a milepost in the history of Alnylam that has been 16 years in the making – the recommendation from the EU Committee for Medicinal Products for Human Use (CHMP) that the European Medicines Agency approve the first-ever RNAi therapeutic, ONPATTRO. We believe the positive CHMP opinion in the EU signals the potential for a new therapeutic paradigm in medicine, raising hope for patients and caregivers impacted by hATTR amyloidosis. With this achievement, the expected FDA action by our August 11 PDUFA date, and plans to file our JNDA in Japan later this year, we believe we are poised to deliver on the promise of ONPATTRO on a truly global scale," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam.

"In parallel, we have advanced our three other wholly owned late-stage programs. We achieved robust enrollment in our ENVISION Phase 3 study of givosiran and are on track for an interim analysis by the end of September in support of a potential accelerated approval. In addition, with our recent alignment with the FDA on a Phase 3 trial design for lumasiran, we are gearing up to initiate a pivotal study for this program in the coming weeks. Finally, we’re pleased to announce today that we have reached alignment with the FDA on a Phase 3 trial design for ALN-TTRsc02 in hATTR amyloidosis patients, where we’re on track to start the study by year’s end. All together, we believe our efforts position us to achieve our Alnylam 2020 strategy of building a multi-product, global, commercial-stage company with a deep and sustainable clinical pipeline by the end of 2020."

Second Quarter 2018 and Recent Significant Corporate Highlights

Received a positive opinion from CHMP recommending marketing authorization of ONPATTRO (patisiran) – a first-of-its-kind RNAi therapeutic – for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
The European Commission (EC) decision on approval of ONPATTRO is now expected in September, and the recommended Summary of Product Characteristics (SmPC) includes data from secondary and exploratory study endpoints in the APOLLO Phase 3 trial, including cardiac results.
The Company is on track in the U.S. with an August 11 PDUFA date for ONPATTRO with the FDA.
Published APOLLO study results for patisiran in the July 5, 2018 issue of The New England Journal of Medicine.
Presented additional data from the APOLLO Phase 3 study at the 4th Congress of the European Academy of Neurology (EAN) and the Peripheral Nerve Society (PNS) 2018 Annual Meeting.
To date the Company has fulfilled over 200 requests by physicians for eligible patients to begin treatment in the early access or compassionate use programs for patisiran in the U.S. and EU.
Advanced givosiran, an investigational RNAi therapeutic in development for the treatment of acute hepatic porphyrias (AHPs).
Completed enrollment of the cohort of patients in the ENVISION Phase 3 study that will comprise the planned interim analysis in support of a potential accelerated approval.
The Company remains on track to report topline results of the interim analysis by the end of September and, pending Company and FDA review of the program at the time of interim analysis and assuming positive results and acceptable safety, the Company expects to submit an NDA at or around year-end 2018 seeking an accelerated approval.
The interim analysis is based on lowering of urinary aminolevulinic acid (ALA) levels at three months of treatment as a surrogate biomarker that is reasonably likely to predict clinical benefit.
Alnylam announced today that it has achieved robust enrollment in ENVISION and expects to complete full patient accrual by the end of September, ahead of schedule.
As a result, the Company now expects to report topline results on the primary endpoint of annualized attack rate in early 2019.
Advanced lumasiran, an investigational RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1), with new positive data from the Phase 1/2 study presented at the OxalEurope European Hyperoxaluria Consortium.
The Company is on track to initiate a Phase 3 pivotal trial in mid-2018, with results expected in late 2019 supporting a potential NDA filing in early 2020.
Advanced ALN-TTRsc02, a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis.
Alnylam announced today that it has reached alignment with the FDA on the design of a pivotal Phase 3 study for ALN-TTRsc02 in patients with hATTR amyloidosis.
The Phase 3 pivotal trial will be an open-label study with co-primary endpoints of mNIS+7 and Norfolk-QOL at nine months comparing the effects of ALN-TTRsc02 in approximately 120 patients with hATTR amyloidosis to results from the placebo arm from the APOLLO Phase 3 study of patisiran. In addition, certain cardiac parameters will be included as endpoints.
An additional reference arm of approximately 30 patients receiving patisiran will be included.
The Company is on track to start the Phase 3 study in late 2018 and plans to start additional Phase 3 studies of ALN-TTRsc02, including in wild-type ATTR amyloidosis, in 2019.
Alnylam announces today that, due to slower than anticipated enrollment, it expects that initial data from the Phase 2 trial of cemdisiran in atypical hemolytic-uremic syndrome (aHUS) will be reported in 2019. The Company had previously guided for initial data to be reported in late 2018.
Alnylam’s partner, The Medicines Company, announced in June that the Independent Data Monitoring Committee for the ongoing inclisiran Phase 3 clinical trials (ORION 9, 10, and 11) conducted its third, planned review of safety and efficacy data from the ORION trials and recommended that they continue without modification.
At the time of review, substantially all patients in the trials had received two doses of inclisiran or placebo.
The Company has accumulated more than 1,550 patient-years of safety data for inclisiran.
Enrollment in the fitusiran Phase 3 ATLAS program is ongoing.
Announced successful delivery of novel siRNA conjugates to the central nervous system (CNS) in rats and plans to advance a pipeline of CNS-targeted investigational RNAi therapeutics into clinical development.
Upcoming Events

In mid-2018, Alnylam intends to:

Achieve FDA approval and launch ONPATTRO in the U.S.
Gain regulatory approval for ONPATTRO from the EC; the Company expects to launch ONPATTRO in certain European markets shortly thereafter.
File a Japanese NDA for ONPATTRO with the Pharmaceuticals and Medical Device Agency.
Report topline interim analysis results from the ENVISION Phase 3 trial of givosiran in support of a potential accelerated approval.
Initiate the lumasiran Phase 3 study.
In late 2018, Alnylam intends to:

File for regulatory approval for ONPATTRO in additional global markets.
File an NDA for givosiran with the FDA for accelerated approval, assuming positive results and acceptable safety from the interim analysis of the ENVISION Phase 3 study and pending FDA review.
Present 12-month safety and efficacy results from the Global Open-Label Extension (OLE) study of ONPATTRO at the annual meeting of the American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) on October 10th in Washington, D.C.
Present updated data from the Phase 1/2 and OLE studies of lumasiran, at the European Society for Pediatric Nephrology (ESPN) Annual Meeting in Antalya, Turkey and at the American Society of Nephrology (ASN) Kidney Week Meeting in San Diego, CA., respectively, in October.
Initiate the Phase 3 study for ALN-TTRsc02 in hATTR amyloidosis.
File new Investigational New Drug (IND) or Clinical Trial Applications (CTA), including ALN-AAT02, in development for the treatment of alpha-1 antitrypsin deficiency-associated liver disease, and ALN-HBV02 (also known as VIR-2218), in development in partnership with Vir Biotechnology for the treatment of chronic hepatitis B virus infection.
Complete selection of its first CNS-targeted development candidate (DC).
Financial results for the quarter ended June 30, 2018

"Alnylam’s strong balance sheet with approximately $1.48 billion in cash and investments allows us to execute on preparations for our anticipated product launches for patisiran in 2018 and givosiran in 2019, assuming regulatory approvals," said Manmeet Soni, Chief Financial Officer of Alnylam.

Cash and Investments
At June 30, 2018, Alnylam had cash, cash equivalents and marketable debt securities, and restricted investments, excluding equity securities, of $1.48 billion, as compared to $1.73 billion at December 31, 2017.

GAAP and Non-GAAP Net Loss
The net loss according to accounting principles generally accepted in the U.S. (GAAP) for the second quarter of 2018 was $163.6 million, or $1.63 per share on both a basic and diluted basis, as compared to a net loss of $118.4 million, or $1.34 per share on both a basic and diluted basis, for the same period in the previous year.

The non-GAAP net loss for the second quarter of 2018 was $161.9 million, or $1.61 per share on both a basic and diluted basis, as compared to a non-GAAP net loss of $94.4 million, or $1.07 per share on both a basic and diluted basis for the same period in the previous year.

The non-GAAP net loss excludes stock-based compensation expense and gain on litigation settlement. See "Use of Non-GAAP Financial Measures" below for a description of non-GAAP financial measures and a reconciliation between GAAP and non-GAAP net loss appearing later in this press release.

Revenues
Revenues were $29.9 million in the second quarter of 2018, as compared to $15.9 million in the second quarter of 2017. Revenues for the second quarter of 2018 included $23.1 million from the Company’s alliance with Sanofi Genzyme and $6.8 million from other sources.

GAAP and Non-GAAP Research and Development Expenses
GAAP research and development (R&D) expenses were $137.6 million in the second quarter of 2018 as compared to $90.6 million in the second quarter of 2017.

Non-GAAP R&D expenses were $126.0 million in the second quarter of 2018 as compared to $77.4 million in the second quarter of 2017. Non-GAAP R&D expenses exclude stock-based compensation expense. A reconciliation between GAAP and non-GAAP R&D expenses appears later in this press release.

GAAP and Non-GAAP General and Administrative Expenses
GAAP general and administrative (G&A) expenses were $84.7 million in the second quarter of 2018 as compared to $45.8 million in the second quarter of 2017.

Non-GAAP G&A expenses were $74.1 million in the second quarter of 2018 as compared to $35.0 million in the second quarter of 2017. Non-GAAP G&A expenses exclude stock-based compensation expense. A reconciliation between GAAP and non-GAAP G&A expenses appears later in this press release.

Gain on Litigation Settlement
In April 2018, we and Dicerna Pharmaceuticals, Inc. entered into a settlement agreement and general release resolving all ongoing litigation between the companies. As a result, during the second quarter of 2018, we recorded $20.6 million as a gain on litigation settlement that includes the $10.0 million valuation of Dicerna common stock received at the settlement date, the $2.0 million upfront cash payment received in the second quarter of 2018, and $8.6 million, which represents the discounted present value as of the settlement date of the $13.0 million cash payment due from Dicerna by April 18, 2022 under the terms of the settlement agreement. The non-GAAP net loss for the second quarter of 2018 excludes the gain on litigation settlement.

2018 Financial Guidance
Alnylam reiterates its expectations to end 2018 with approximately $1.0 billion of cash, cash equivalents and marketable debt securities, restricted cash and restricted investments, excluding equity securities.

The Company reiterates its expectations for its 2018 annual non-GAAP R&D expenses to be in the range of $420 million to $460 million and non-GAAP selling, general and administrative (SG&A) expenses to be in the range of $280 million to $320 million. Both non-GAAP R&D and SG&A expenses exclude stock-based compensation expenses.

Use of Non-GAAP Financial Measures
This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses and non-recurring gains outside the ordinary course of the Company’s business. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies.

The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in the press release are stock-based compensation expense and the gain on litigation settlement. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impacts the fair value of these awards. The Company has excluded the impact of the gain on litigation settlement because the Company believes this item is a one-time event occurring outside the ordinary course of the Company’s business.

The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between GAAP and non-GAAP measures is provided later in this press release.

Conference Call Information
Management will provide an update on the Company and discuss second quarter 2018 results as well as expectations for the future via conference call on Thursday, August 2, 2018 at 8:30 am ET. To access the call, please dial 877-312-7507 (domestic) or 631-813-4828 (international) five minutes prior to the start time and refer to conference ID 1365915. A replay of the call will be available beginning at 11:30 am ET on the day of the call. To access the replay, please dial 855-859-2056 (domestic) or 404-537-3406 (international) and refer to conference ID 1365915.

On August 2, 2018 OncoMed Pharmaceuticals, Inc. (NASDAQ:OMED), a clinical-stage biopharmaceutical company focused on discovering and developing novel anti-cancer therapeutics, reported second quarter 2018 financial results and provided a corporate update (Press release, OncoMed, AUG 2, 2018, View Source [SID1234528346]). As of June 30, 2018, cash, cash equivalents, and short-term investments totaled $79.9 million.

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"The first half of the year has been focused on effective execution in the development of our pipeline of oncology candidates, including navicixizumab, etigilimab (anti-TIGIT) and GITRL-Fc which are being investigated in ongoing clinical trials," said John Lewicki, Ph.D., President and Chief Executive Officer of OncoMed. "These activities will culminate in important data announcements later this year. We are particularly encouraged by the potential of navicixizumab, our lead product candidate, in the treatment of late-stage ovarian cancer and look forward to sharing single-agent and chemotherapy combination data by the end of the year. We also plan to report first-in-human dose escalation Phase 1a data from our etigilimab program in the fourth quarter of 2018, while early clinical data from our GITRL-Fc program will likely be available in the first half of 2019."

Pipeline Highlights

Navicixizumab (anti-DLL4/VEGF bispecific; OMP-305B83)

An abstract summarizing interim Phase 1b clinical trial data of navicixizumab in combination with paclitaxel for the treatment of heavily pretreated, platinum-resistant ovarian cancer patients has been accepted as a poster presentation on October 20, 2018 at the European Society for Medical Oncology meeting in Munich, Germany. In addition, the company expects to publish the final results of the Phase 1a single-agent

navicixizumab study in all-comers solid tumor patients, which included several late-stage ovarian cancer patients, by the end of 2018.

The company has decided to prioritize navicixizumab development resources in platinum-resistant ovarian cancer, where it believes navicixizumab offers promise and potential as a meaningful therapeutic for patients who lack effective later-line therapeutic alternatives. Consequently, OncoMed has decided to discontinue enrollment of additional patients in its Phase 1b trial of navicixizumab in combination with FOLFIRI or FOLFOX in second-line colorectal cancer as it pursues expanded development in ovarian cancer. The Phase 1b ovarian cancer trial was recently expanded from 30 patients to enroll up to 60 patients.

Etigilimab (Anti-TIGIT; OMP-313M32)

In the second quarter, OncoMed initiated the Phase 1b portion of the Phase 1a/b study of etigilimab in combination with anti-PD1 (nivolumab) in patients with advanced or metastatic solid tumors. The company also completed dose-escalation in the Phase 1a portion of the trial and is now enrolling patients in the single-agent expansion phase of this study. The company expects to report data from the Phase 1a dose-escalation portion of the trial, designed to assess safety and tolerability of escalating doses of etigilimab monotherapy, in the fourth quarter of 2018.

GITRL-Fc (OMP-336B11)

Enrollment continues in the Phase 1a single-agent study of OncoMed’s wholly-owned GITRL-Fc in patients with advanced or metastatic solid tumors. GITRL-Fc is a fusion protein with an Fc-linked fully human trimer ligand and is designed to activate the co-stimulatory receptor GITR (glucocorticoid-induced tumor necrosis factor receptor-related protein) to enhance T-cell modulated immune responses. Data from the Phase 1a trial are expected to be presented in 2019.

Second Quarter 2018 Financial Results

Cash, cash equivalents and short-term investments totaled $79.9 million as of June 30, 2018, compared to $103.1 million as of December 31, 2017.

Revenues were $6.9 million for the second quarter of 2018, an increase of $0.7 million, compared to $6.2 million for the same period in 2017. The change in revenue was a result of the new revenue recognition standard adopted in the first quarter of 2018.

Research and development (R&D) expenses were $8.1 million for the second quarter of 2018, a decrease of $7.0 million, compared to $15.1 million for the same period in 2017. The decrease in R&D expenses was due to decreases in clinical development costs and reduced headcount as a result of the restructuring actions in April 2017.

General and administrative (G&A) expenses were $3.7 million for the second quarter of 2018, a decrease of $0.4 million, compared to $4.1 million for the same period in 2017. The decrease in G&A expenses was primarily due to a decrease in personnel cost, including stock-based compensation.

Net loss was $4.0 million ($0.10 per share) for the second quarter of 2018, compared to $15.2 million ($0.40 per share) for the same period of 2017. The change in year-over-year net loss was primarily due to lower operating expenses in the second quarter of 2018.

2018 Financial Guidance

With resource reprioritization and additional cash management measures, OncoMed’s current cash runway has been extended by one quarter and is now estimated to fund operations through at least the fourth quarter of 2019, without taking into account future potential milestone or opt-in payments from its partners. OncoMed estimates 2018 operating cash burn to be less than $55 million, before considering potential milestone or opt-in payments