On October 31, 2018 ArQule, Inc. (Nasdaq: ARQL) reported its financial results for the third quarter of 2018 (Press release, ArQule, OCT 31, 2018, View Source [SID1234530415]).

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For the quarter ended September 30, 2018, the Company reported a net loss of $5,619,000 or $0.05 per share, compared with a net loss of $6,666,000 or $0.09 per share, for the third quarter of 2017. For the nine-month period ended September 30, 2018, the Company reported a net loss of $6,995,000 or $0.07 per share, compared with a net loss of $21,443,000 or $0.30 per share, for the nine-month period ended September 30, 2017.

At September 30, 2018, the Company had a total of approximately $105,088,000 in cash, equivalents and marketable securities.

Key Highlights

In July 2018, the Company raised approximately $70 million of gross proceeds in a public offering of common stock
In August 2018, extensive preclinical data on ARQ 531, our reversible BTK inhibitor, was published in the scientific journal, Cancer Discovery, highlighting the profile of this potential first and best-in-class molecule
In September 2018, miransertib (ARQ 092) was granted Fast Track Designation for the treatment of PROS (PIK3CA-Related Overgrowth Spectrum), opening the way for enhanced interactions with regulators
In October 2018, three clinical presentations for miransertib were given at the American Society of Human Genetics, confirming its potential for treating patients with Proteus syndrome and PROS
"We continue to execute on our strategy to develop rapidly ARQ 531 in hematological malignancies, miransertib in PROS and Proteus syndrome, as well as miransertib and ARQ 751 in hormone sensitive solid tumors," said Paolo Pucci, Chief Execute Officer of ArQule. "Each of our drug candidates holds tremendous promise and each candidate is being increasingly validated by the data that we are placing in the public domain."

Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer of ArQule said, "We are pleased with the Cancer Discovery publication for ARQ 531 and how the pre-clinical data highlighted in that paper is beginning to be validated in the on-going Phase 1 trial." "I am also proud of the work that we continue to perform with miransertib in PROS and Proteus syndrome and am encouraged by the accumulating clinical data that supports the potential utility of miransertib to fulfill the serious unmet medical need in these diseases, particularly in children."

Revenues and Expenses

Revenues for the quarter ended September 30, 2018, were $4,979,000 compared with revenues of zero for the quarter ended September 30, 2017. Research and development revenue in the quarter ended September 30, 2018 consisted of $2,852,000 from our February 2018 Sinovant licensing agreement, $1,996,000 from our April 2018 Basilea licensing agreement and $131,000 from a non-exclusive license agreement for certain of our library compounds.

Revenues for the nine months ended September 30, 2018, were $22,823,000 compared with revenues of zero for the nine months ended September 30, 2017. Research and development revenue in the nine months ended September 30, 2018 consisted of $5,852,000 from our February 2018 Sinovant licensing agreement $15,702,000 million from our April 2018 Basilea licensing agreement and $1,269,000 from a non-exclusive license agreement for certain of our library compounds.

Research and development expense in the third quarter of 2018 was $7,261,000 compared with $4,570,000 for the third quarter of 2017. The $2.7 million increase in research and development expense in the third quarter of 2018 was primarily due to higher outsourced preclinical, clinical and product development costs.

Research and development expense in the nine months ended September 30, 2018 was $19,860,000 compared with $14,747,000 in the nine months ended September 30, 2017. The $5.1 million increase in research and development expense in the nine months ended September 30, 2018 was primarily due to higher outsourced preclinical, clinical and product development costs.

General and administrative expense was $3,429,000 in the third quarter of 2018 compared with $1,762,000 in the third quarter 2017. The $1.7 million increase in general and administrative expense in the third quarter of 2018 was primarily due to higher consulting and professional fees of $1.4 million and labor and related costs of $0.2 million.

General and administrative expense was $8,014,000 in the nine months ended September 30, 2018 compared with $5,702,000 in the nine months ended September 30, 2017. The $2.3 million increase in general and administrative expense in the nine months ended September 30, 2018 was principally due to higher consulting and professional fees of $1.8 million and labor and related costs of $0.5 million.

2018 Updated Financial Guidance

As a result of our advancing development programs and collaborations and the likely timing of cash receipts and disbursements, we have updated our 2018 guidance. Net use of cash is expected to be approximately $34 million for the year, which is slightly higher than our previous guidance due primarily to an acceleration of the clinical development costs associated with proprietary and partnered clinical programs. Net loss is expected to range between $14 and $17 million for the year, and net loss per share to range between $(0.14) and $(0.17). We are also adjusting our revenue guidance upward to between $24 and $25 million primarily in connection with services provided to our derazantinib partners.

ArQule expects to end 2018 with approximately $100 million in cash and marketable securities, which we believe will be sufficient to finance our operations into 2021.

Conference Call and Webcast

The live webcast can be accessed in the "Investors and Media" section of our website, www.arqule.com, under "Events & Presentations." You may also listen to the call by dialing (877) 868-1831 within the U.S. or (914) 495-8595 outside the U.S. A replay will be available two hours after the completion of the call and can be accessed in the "Investors & Media" section of our website, www.arqule.com, under "Events and Presentations."

On October 31, 2018 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address cancer, liver, and inflammatory diseases, reported an update on its Phase II clinical trial of drug candidate Namodenoson (CF102) for the treatment of advanced hepatocellular carcinoma (HCC) in patients with Child Pugh B whose disease has progressed on sorafenib therapy (Press release, Can-Fite BioPharma, OCT 31, 2018, View Source [SID1234530414]). Due to patient survival, top line efficacy results are expected during the first quarter of 2019.

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Enrollment of 78 patients was completed in August 2017 and the trial continues treating subjects in a blinded fashion (either Namodenoson 25 mg BID or matching placebo).

The statistical plan for this trial requires that the primary efficacy analysis occurs when no more than 3 of the original 78 patients survive. At the outset of the trial, it was assumed that patients with advanced HCC with Child-Pugh B had a relatively poor prognosis, and that within approximately a year of enrollment of the last subject, primary efficacy analysis could be conducted. In order to maintain the statistical integrity of the trial as well as adhere to the principles of Good Clinical Practice, the Company estimates that it will un-blind the data during Q1/19.

Can-Fite’s CEO, Dr. Pnina Fishman, commented, "This unexpectedly prolonged longevity is unquestionably beneficial for the individual patients, and gives us hope that Namodenoson may eventually prove its value in this patient population."

Can-Fite received Orphan Drug Designation for Namodenoson in Europe and the U.S., as well as Fast Track Status in the U.S. as a second line treatment for HCC.

About Namodenoson

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson is being evaluated in Phase II trials for two indications, as a second line treatment for hepatocellular carcinoma, and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.

On October 31, 2018 Acorda Therapeutics, Inc. (Nasdaq:ACOR) reported a financial and pipeline update for the third quarter ended September 30, 2018 (Press release, Acorda Therapeutics, OCT 31, 2018, View Source [SID1234530411]).

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"Acorda’s highest priority is preparing for the expected launch of Inbrija. Our market research indicates that healthcare professionals, patients and care partners consider OFF periods, or the re-emergence of Parkinson’s symptoms, to be one of the most significant unmet needs in Parkinson’s, and that they are enthusiastic about the prospect of an inhaled formulation of levodopa as a treatment option," said Ron Cohen, M.D., Acorda’s President and CEO.

"We were disappointed and disagree with the decision of the Federal appeals court regarding Ampyra, and we have filed an en banc petition requesting review by the entire court. At the same time, we were prepared for that potential outcome, and our original projections had us well capitalized to fully fund the launch of Inbrija and to develop the ARCUS pipeline. We have taken several steps over the past year both to conserve and to increase cash. Based on these, as well as greater than forecasted Ampyra sales, we are in now in an even stronger financial position, and are increasing our guidance for both cash and Ampyra sales in 2018."

Third Quarter 2018 Financial Results

AMPYRA (dalfampridine) Extended Release Tablets, 10 mg – For the quarter ended September 30, 2018, the Company reported AMPYRA net revenue of $137.8 million compared to $132.6 million for the same quarter in 2017.

Research and development (R&D) expenses for the quarter ended September 30, 2018 were $22.9 million, including $1.1 million of share-based compensation compared to $33.3 million, including $2.0 million of share-based compensation, for the same quarter in 2017.

Sales, general and administrative (SG&A) expenses for the quarter ended September 30, 2018 were $43.6 million, including $4.0 million of share-based compensation compared to $40.7 million, including $4.6 million of share-based compensation for the same quarter in 2017.

Provision for income taxes for the quarter ended September 30, 2018 was $38.0 million, including $3.1 million of cash taxes, compared to a provision for income taxes of $18.9 million, including $3.7 million of cash taxes, for the same quarter in 2017.

The Company reported a GAAP net loss of $(13.9) million for the quarter ended September 30, 2018, or $(0.29) per diluted share. GAAP net loss in the same quarter of 2017 was $(25.2) million, or $(0.55) per diluted share.

Non-GAAP net income for the quarter ended September 30, 2018 was $8.1 million, or $0.17 per diluted share. Non-GAAP net income in the same quarter of 2017 was $20.1 million, or $0.43 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, intangible asset impairment charges, and restructuring costs. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At September 30, 2018, the Company had cash, cash equivalents and short-term investments of $460.9 million.

Guidance for 2018

AMPYRA 2018 net revenue guidance increased from $330-$350 million to more than $400 million.
R&D expenses for the full year 2018 reiterated and expected to be $100-$110 million including pre-launch 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 reiterated and 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 has increased projected 2018 year-end cash balance from more than $300 million to more than $400 million.
Third Quarter 2018 Highlights

INBRIJA (levodopa inhalation powder) in Parkinson’s disease
In September, the FDA extended the PDUFA goal date for its review of the New Drug Application (NDA) of INBRIJA from October 5, 2018 to January 5, 2019 based on submissions the Company made in response to requests from FDA for additional information on chemistry, manufacturing and controls (CMC). FDA determined that these submissions constituted a major amendment and will take additional time to review.
The Company reported that the inspection of its Chelsea, Massachusetts manufacturing facility and the Inbrija inhaler device manufacturer’s facility were successfully completed and closed without need for any further action by the FDA.
INBRIJA is an investigational treatment for symptoms of OFF periods in people with Parkinson’s disease taking a carbidopa/levodopa regimen.
AMPYRA (dalfampridine)
In September, the United States Court of Appeals for the Federal Circuit, by a 2-1 vote, upheld the United States District Court for the District of Delaware’s decision to invalidate four Ampyra patents.
In October, the Company filed a petition for en banc hearing with the United States Court of Appeals for the Federal Circuit.
The Company announced that it had settled with Mylan AG to market an authorized generic version of Ampyra. In mid-September, Mylan announced the U.S. launch of the authorized generic.
Webcast and Conference Call

The Company will host a conference call today at 8:30 a.m. ET. To participate in the conference call, please dial (833) 236-2756 (domestic) or (647) 689-4181 (international) and reference the access code 4468928. 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 October 31, 2018 until 11:59 p.m. ET on November 30, 2018. To access the replay, please dial (800) 585-8367 (domestic) or (416) 621-4642 (international); reference code 4468928. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

On October 31, 2018 Epigenomics AG (FSE: ECX, OTCQX: EPGNY) reported the Veterans Administration New York Harbor Healthcare System (VA-Manhattan) is commencing a study to assess the adherence impact of offering a blood-based colorectal cancer screening test and colonoscopy completion in patients who have refused colonoscopy and fecal immunochemical test (FIT) (Press release, Epigenomics, OCT 31, 2018, View Source [SID1234530405]).

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The VA-Manhattan has received grants from the American Society for Gastrointestinal Endoscopy (ASGE) and New York Society for Gastrointestinal Endoscopy (NYSGE) to assist in the performance of this study. The study will be managed through The Narrows Institute. Epigenomics will provide testing and budgetary support for testing.

Colorectal cancer (CRC) is the fourth most common cancer and the second deadliest cancer in the US. However, CRC is a preventable condition with screening being one of the most impactful public health contributions to prevention. Screening rates are suboptimal due to patient barriers to colonoscopy or stool-based testing. Unscreened patients contribute to approximately 43% of new CRC cases, 70% of CRC deaths, and 76% of CRC treatment expenditures.

The goal of the VA-Manhattan study is to assess the potential of a blood-based test as an acceptable alternative for these screen-resistant individuals. If the proposed study shows high acceptance of the blood test as well as reliable colonoscopy follow up for positive tests, then the blood test could play an important adjunctive role in improving overall screening rates.

"Some people simply don’t want colonoscopy or stool-based colorectal cancer screening, so we need to understand how effective alternative methods such as a blood-based test are at engaging these individuals," said Dr. Peter Liang, the study principal investigator and a gastroenterologist at the VA-Manhattan.

"Unscreened patients contribute significantly to new CRC cases, deaths, and treatment costs," said Greg Hamilton, CEO of Epigenomics AG. "Addressing the unscreened challenge with a blood test could unquestionably have an impact to colorectal cancer management. We look forward to the study outcomes as they have the potential to support the use of a blood test on screening-resistant patients."

On October 31, 2018 Gilead Sciences, Inc. (Nasdaq: GILD) and Tango Therapeutics, Inc., a company focused on the discovery and development of novel cancer therapies, reported a global strategic collaboration to discover, develop and commercialize a pipeline of innovative targeted immuno-oncology treatments for patients with cancer (Press release, Gilead Sciences, OCT 31, 2018, View Source;p=irol-newsArticle&ID=2374439 [SID1234530402]).

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Under the multi-year collaboration, Tango will perform target discovery and validation and Gilead will have options to worldwide rights on up to five targets emerging from Tango’s proprietary functional genomics-based discovery platform. For two programs directed to these targets, Tango will retain the option to co-develop and co-detail in the U.S. The collaboration does not include Tango’s lead programs, for which Tango will retain all rights.

"Tango has built a unique discovery platform that we hope will help create the next generation of cancer therapies," said John McHutchison, AO, MD, Gilead’s Chief Scientific Officer and Head of R&D. "Our collaboration will combine Tango’s innovative discovery technology alongside Gilead’s drug discovery and development capabilities to build a pipeline of novel immuno-oncology therapies."

"Gilead is the ideal partner to help us bring potentially transformative treatments to patients with cancer," said Barbara Weber, MD, Tango’s President and Chief Executive Officer. "This partnership has significant strategic value for us. With Gilead as our partner, we can maximize the applications of our platform in immuno-oncology, while continuing to independently advance our lead programs into the clinic and beyond."

Under the terms of the agreement, Tango will receive an upfront payment of $50 million. Tango will also be eligible to receive approximately $1.7 billion in total additional payments across all programs in the form of pre-clinical fees and development, regulatory and commercial milestone payments; and up to low double-digit tiered royalties on net sales. For those programs that Tango opts in to co-develop and co-detail, the parties will split profits and losses 50/50 for the U.S., development costs will be shared in a manner that is commensurate with product rights, and Tango will be eligible to receive milestone payments and royalties on ex-U.S. sales.