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 Perrigo Company plc (NYSE; TASE: PRGO) reported that its Board of Directors declared a quarterly dividend of $0.19 per share, payable on December 18, 2018 to shareholders of record on November 30, 2018 (Press release, Perrigo Company, OCT 31, 2018, View Source [SID1234530459]).

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On October 31, 2018 Ophthotech Corporation (Nasdaq:OPHT) reported financial and operating results for the third quarter ended September 30, 2018 and provided a business update (Press release, Ophthotech, OCT 31, 2018, View Source [SID1234530416]).

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"During 2018 Ophthotech made significant progress in bringing together a diversified pipeline of therapeutic and gene therapy programs for the treatment of retinal diseases," stated Glenn P. Sblendorio, Chief Executive Officer and President of Ophthotech. "Today, we announced two transactions that add compelling opportunities to our existing pipeline of retinal programs, and through the acquisition of Inception 4, Inc. we are excited to welcome Versant Ventures as a major shareholder of Ophthotech. We are on track to provide topline data from our Zimura program in wet age-related macular degeneration by the end of this year, followed by topline data in geographic atrophy secondary to dry AMD in the second half of 2019 and in Stargardt disease in 2020. We look forward to advancing and expanding our pipeline of age-related and orphan retinal diseases and creating value for our shareholders."

Corporate Highlights

The following announcements will be discussed during today’s conference call/webcast (see full detailed press releases issued earlier today and call in information below).

As announced today, Ophthotech has acquired Inception 4, Inc., a privately held company backed by Versant Ventures, expanding Ophthotech’s therapeutic pipeline in age-related retinal indications. Through this acquisition, Ophthotech gains worldwide development and commercialization rights to Inception 4’s small molecule inhibitors of HtrA1 (high temperature requirement A serine peptidase 1 protein). As a major new investor with substantial geographic reach, Versant Ventures has agreed to help Ophthotech identify exceptional opportunities to expand the pipeline. As a result of the closing of the acquisition, Ophthotech obtained approximately $6.1 million in cash through Inception 4. As upfront consideration in the transaction, Ophthotech agreed to issue approximately 5.2 million shares to the shareholders of Inception 4. After giving effect to the transaction, Versant Ventures, through its affiliated investment funds, owns approximately 12.5% of the outstanding shares of Ophthotech’s common stock. In addition, Inception 4 equity holders will be entitled to receive post-closing payments upon the achievement of certain clinical and marketing approval milestones in certain AMD indications.
Ophthotech also announced today that it expanded its gene therapy pipeline with a novel product candidate to treat Best vitelliform macular dystrophy, also known as Best disease. The Company entered into its second series of gene therapy agreements with the University of Pennsylvania and the University of Florida, including an exclusive option agreement for rights to negotiate to acquire an exclusive global license to develop and commercialize novel adeno-associated virus (AAV) gene therapy product candidates for the treatment of Best disease.
Therapeutic Program Highlights

Complement Factor C5 Inhibitor Program: Zimura

Wet Age-related Macular Degeneration: The Company expects initial top-line data by the end of this year from its randomized, dose-ranging, open-label, uncontrolled, multi-center Phase 2a clinical trial of Zimura (avacincaptad pegol) in combination with the anti-vascular endothelial growth factor (anti-VEGF) agent Lucentis (ranibizumab) in patients with wet AMD who have not been previously treated with anti-VEGF therapies. This trial is designed to assess the safety of Zimura combination therapy at different dosages and to detect a potential efficacy signal at month six. The Company completed patient recruitment for this trial in April 2018.
Geographic Atrophy, an Advanced Form of Dry Age-related Macular Degeneration: In October 2018, the Company completed patient enrollment for its ongoing randomized, double-masked, sham controlled, multi-center Phase 2b clinical trial of Zimura for the treatment of geographic atrophy secondary to dry AMD. The Company expects initial top-line data for this trial to be available in the fourth quarter of 2019.
Autosomal Recessive Stargardt Disease: Patient enrollment in the Phase 2b randomized, double-masked, sham-controlled, multi-center clinical trial assessing the efficacy and safety of Zimura in patients with autosomal recessive Stargardt disease (STGD1) is currently on-going. Initial top-line data is expected to be available in 2020.
Gene Therapy Program Highlights

Rhodopsin-mediated Autosomal Dominant Retinitis Pigmentosa: In August 2018, Ophthotech announced that proof-of-concept study results of its adeno-associated virus (AAV) gene therapy product candidate for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP) in a naturally occurring canine model were published in the journal Proceedings of the National Academy of Sciences of the USA (PNAS). Ophthotech obtained a license for rights to develop and commercialize this AAV gene therapy product candidate in June 2018. This publication entitled: "Mutation-independent Rhodopsin Gene Therapy by Knockdown and Replacement with a Single AAV vector" was published by scientists at the University of Pennsylvania and University of Florida. Based on current timelines and subject to regulatory review, Ophthotech expects to initiate a Phase 1/2 clinical trial in RHO-adRP in 2020.
2018 Operational Update

As of September 30, 2018, the Company had $135.2 million in cash and cash equivalents. The Company increased its year end 2018 cash and cash equivalents estimate to range between $125 million and $130 million, an increase from the Company’s prior estimate of between $112 million and $117 million, reflecting the impact of the acquisition of Inception 4, expansion of the Company’s gene therapy research and development programs and the continuation of the Company’s development programs for Zimuraas currently planned. This estimate does not reflect any additional expenditures resulting from the potential in-licensing or acquisition of additional product candidates or technologies or associated development that the Company may pursue.

2018 Financial Highlights

Revenues: The Company did not have any collaboration revenue for the quarter and nine months ended September 30, 2018, compared to $206.7 million and $210.0 million for the same periods in 2017. Collaboration revenue decreased due to the completion of the Company’s licensing and commercialization agreement with Novartis Pharma AG and the recognition of all associated deferred revenue during the third quarter of 2017.
R&D Expenses: Research and development expenses were $9.4 million for the quarter ended September 30, 2018, compared to $10.7 million for the same period in 2017. For the nine months ended September 30, 2018, research and development expenses were $25.6 million compared to $58.3 million for 2017. The Company continues to pursue its ongoing Zimura development programs and gene therapy research and development programs. Research and development expenses decreased primarily due to decreases in expenses related to the discontinuation of the Company’s FovistaPhase 3 clinical program and decreases in costs associated with the Company’s 2017 reduction in personnel.
G&A Expenses: General and administrative expenses were $6 million for the quarter ended September 30, 2018, compared to $7.1 million for the same period in 2017. For the nine months ended September 30, 2018, general and administrative expenses were $17.9 million compared to $28.8 million for 2017. General and administrative expenses decreased primarily due to decreases in costs to support the Company’s reduced operations and infrastructure and decreases in costs associated with its 2017 reduction in personnel, which included facilities lease termination expenses incurred during the first quarter of 2017.
Net Income: The Company reported a net loss for the quarter ended September 30, 2018 of $14.7 million, or ($.41) per diluted share, compared to net income of $189.1 million, or $5.25 per diluted share, for the same period in 2017. For the nine months ended September 30, 2018, the Company reported a net loss of $41 million, or ($1.13) per diluted share, compared to net income of $123.7 million, or $3.44 per diluted share, for the same period in 2017.
Conference Call/Web Cast Information

Ophthotech will host a conference call/webcast to discuss the Company’s financial and operating results for the third quarter of 2018 and to provide a business update. The call is scheduled for October 31, 2018 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 888-204-4368 (USA) or 323-994-2082 (International), passcode 3714524. A live, listen-only audio webcast of the conference call can be accessed on the Investor Relations section of the Ophthotech website at: www.ophthotech.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA Toll Free), passcode 3714524.

On October 31, 2018 Thermo Fisher Scientific Inc. (NYSE: TMO), the world leader in serving science, reported that Marc N. Casper, president and chief executive officer, will present at the Jefferies 2018 London Healthcare Conference on Wednesday, November 14, 2018, at 10:40 a.m. (GMT) at the Waldorf Hilton, London, UK (Press release, Thermo Fisher Scientific, OCT 31, 2018, View Source [SID1234530460]).

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You can access the live webcast of the presentation in the Investors section of our website, www.thermofisher.com.

On October 31, 2018 Affimed N.V. (Nasdaq: AFMD), a clinical stage biopharmaceutical company focused on discovering and developing highly targeted cancer immunotherapies, reported that it will host a conference call on Wednesday, November 7, 2018 at 8:30 a.m. ET to discuss its third quarter financial results and recent corporate developments (Press release, Affimed, OCT 31, 2018, View Source [SID1234530417]).

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The conference call will be available via phone and webcast. To access the call, please dial (323) 794-2588 for U.S. callers, or +44 (0)330 336 9125 for international callers, and reference conference ID 6650897 approximately 15 minutes prior to the call.

An audio webcast of the conference call can be accessed in the "Events" section on the "Investors & Media" page of the Affimed website at View Source A replay of the webcast will be available on Affimed’s website shortly after the conclusion of the call and will be archived on the Affimed website for 30 days following the call