On November 9, 2017 ArQule, Inc. (Nasdaq: ARQL) reported its financial results for the third quarter of 2017 (Press release, ArQule, NOV 9, 2017, View Source [SID1234521832]).

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For the quarter ended September 30, 2017, the Company reported a net loss of $6,666,000 or $0.09 per share, compared with a net loss of $5,817,000 or $0.08 per share, for the third quarter of 2016. For the nine-month period ended September 30, 2017, the Company reported a net loss of $21,443,000 or $0.30 per share, compared with a net loss of $15,898,000 or $0.23 per share, for the nine-month period ended September 30, 2016.

At September 30, 2017, the Company had a total of approximately $27,603,000 in cash, equivalents and marketable securities. In October and November 2017, the Company raised approximately $25 million in net proceeds from two equity offerings.

Key Highlights

Miransertib (ARQ 092), lead AKT inhibitor, has met its primary endpoint of determining a biologically active dose in a phase 1 trial for Proteus syndrome lead by the National Human Genome Research Institute (NHGRI) of the National Institutes of Health (NIH). The NIH presented preliminary data from the ongoing phase 1 clinical trial for Proteus syndrome at the Proteus Syndrome Foundation Family Conference in September 2017. In five of the six patients, a reduction of a least 50% of phospho-AKT levels was reported. This met the primary objective of the study. Most significantly the NIH observed disease modification in the cerebriform connective tissue nevus (CCTN) lesions which are considered one of the hallmarks of the disease.
Miransertib was granted by the U.S. Food and Drug Administration (FDA) Rare Pediatric Disease Designation. Under the FDA’s rare pediatric disease priority review voucher program, the sponsor may be eligible for a voucher that can be used to obtain a priority review for a subsequent human drug application.
Miransertib continues to dose in the phase 1/2 trial for Overgrowth Diseases driven by PI3K and AKT mutations and opened its first U.S. clinical trial site. This trial is part of the company’s continued progress towards expanding its rare disease strategy in Overgrowth Diseases.
ARQ 531, an orally bioavailable, potent and reversible BTK inhibitor, continues dosing as planned in a phase 1a/b trial. The trial is enrolling patients with B-cell malignancies, including B-cell lymphomas, chronic lymphocytic leukemia, and Waldenstrom’s macroglobulinemia, who are refractory to other therapeutic options, including ibrutinib. Up to 120 patients can be enrolled in the trial.
Derazantinib (ARQ 087), a pan-FGFR inhibitor, in a registrational trial in FGFR2 fusion positive second-line intrahepatic cholangiocarcinoma (iCCA) is recruiting with six active sites in the U.S. The trial is planned to enroll up to 100 iCCA patients and provides an opportunity for a conditional approval as part of a fast-to-market strategy and includes an interim analysis that will be performed after the first 40 patients have been enrolled and evaluated for response.
Company raised approximately $29 million in capital through a private placement of $15.7 million of common stock, a private placement of $9.5 million of convertible preferred stock and an additional $4 million through unrelated business development activities and other sources. Net proceeds will be used to advance ArQule’s proprietary pipeline and for general business purposes, including working capital.
"The ArQule clinical pipeline is the strongest it has ever been, and the most recent positive developments are the compelling clinical data in Proteus syndrome and the granting of Rare Pediatric Disease Designation for miransertib in this indication," said Paolo Pucci, Chief Executive Officer of ArQule. "We are now well capitalized to see our pipeline assets through major inflection points."

"Based on the preliminary results from the phase 1 NIH-sponsored Proteus syndrome trial, miransertib is the first drug to demonstrate activity and achieve clinical proof of concept in this indication," said Dr. Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule. "We are thankful to the NIH for their work in identifying the mutation driving the disease, conducting the first clinical trial and ultimately identifying potential clinical endpoints. We plan to engage regulatory authorities to define a clinical path to registration in this indication. In parallel and consistent with the regulatory interactions we will continue to enroll in our ongoing phase 1/2 trial in Overgrowth Diseases driven by mutations in the PI3K or AKT pathway, including Proteus syndrome, and provide the drug under compassionate policy."

Revenues and Expenses

Revenues for the quarter ended September 30, 2017, were zero compared with revenues of $1,223,000 for the quarter ended September 30, 2016. Revenues in the nine-months ended September 30, 2017 were zero compared with revenues of $3,522,000 in the nine-months ended September 30, 2016. Revenue in the three and nine-month periods of 2016 is comprised of revenue from the Daiichi Sankyo tivantinib development agreement and the Kyowa Hakko Kirin exclusive license agreement. No further revenue is anticipated from these agreements.

Research and development expense in the third quarter of 2017 was $4,570,000, compared with $5,265,000 for the third quarter of 2016. Research and development expense decreased $0.7 million in the third quarter of 2017 primarily due to lower outsourced preclinical, clinical and product development costs.

Research and development expense in the nine-months ended September 30, 2017 was $14,747,000 compared with $13,800,000 in the nine-months ended September 30, 2016. The $0.9 million increase in research and development expense in the nine-months ended September 30, 2017 was primarily due to higher outsourced clinical and product development costs.

General and administrative expense was $1,762,000 in the third quarter of 2017 compared with $1,824,000 in the third quarter 2016.

General and administrative expense was $5,702,000 in the nine-months ended September 30, 2017 compared with $5,755,000 in the nine-months ended September 30, 2016.

2017 Financial Guidance

As a result of ArQule’s recent stock offerings and business development activities the company is updating 2017 guidance. For 2017, ArQule expects net use of cash to range between $25 and $27 million. Net loss is expected to range between $28 and $30 million, net loss per share is expected to range between $(0.38) and $(0.40) for the year. ArQule expects to end 2017 with between $47 and $49 million in cash and marketable securities.

Conference Call and Webcast

ArQule will hold its third quarter 2017 financial results call today, November 9, 2017 at 9:00 a.m. ET. The live webcast can be accessed in the "Investors & 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 "Investor and Media" section of our website, www.arqule.com, under "Events & Presentations."

On November 9, 2017 ArQule, Inc. (Nasdaq: ARQL) reported its financial results for the third quarter of 2017 (Press release, ArQule, NOV 9, 2017, View Source [SID1234521832]).

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For the quarter ended September 30, 2017, the Company reported a net loss of $6,666,000 or $0.09 per share, compared with a net loss of $5,817,000 or $0.08 per share, for the third quarter of 2016. For the nine-month period ended September 30, 2017, the Company reported a net loss of $21,443,000 or $0.30 per share, compared with a net loss of $15,898,000 or $0.23 per share, for the nine-month period ended September 30, 2016.

At September 30, 2017, the Company had a total of approximately $27,603,000 in cash, equivalents and marketable securities. In October and November 2017, the Company raised approximately $25 million in net proceeds from two equity offerings.

Key Highlights

Miransertib (ARQ 092), lead AKT inhibitor, has met its primary endpoint of determining a biologically active dose in a phase 1 trial for Proteus syndrome lead by the National Human Genome Research Institute (NHGRI) of the National Institutes of Health (NIH). The NIH presented preliminary data from the ongoing phase 1 clinical trial for Proteus syndrome at the Proteus Syndrome Foundation Family Conference in September 2017. In five of the six patients, a reduction of a least 50% of phospho-AKT levels was reported. This met the primary objective of the study. Most significantly the NIH observed disease modification in the cerebriform connective tissue nevus (CCTN) lesions which are considered one of the hallmarks of the disease.
Miransertib was granted by the U.S. Food and Drug Administration (FDA) Rare Pediatric Disease Designation. Under the FDA’s rare pediatric disease priority review voucher program, the sponsor may be eligible for a voucher that can be used to obtain a priority review for a subsequent human drug application.
Miransertib continues to dose in the phase 1/2 trial for Overgrowth Diseases driven by PI3K and AKT mutations and opened its first U.S. clinical trial site. This trial is part of the company’s continued progress towards expanding its rare disease strategy in Overgrowth Diseases.
ARQ 531, an orally bioavailable, potent and reversible BTK inhibitor, continues dosing as planned in a phase 1a/b trial. The trial is enrolling patients with B-cell malignancies, including B-cell lymphomas, chronic lymphocytic leukemia, and Waldenstrom’s macroglobulinemia, who are refractory to other therapeutic options, including ibrutinib. Up to 120 patients can be enrolled in the trial.
Derazantinib (ARQ 087), a pan-FGFR inhibitor, in a registrational trial in FGFR2 fusion positive second-line intrahepatic cholangiocarcinoma (iCCA) is recruiting with six active sites in the U.S. The trial is planned to enroll up to 100 iCCA patients and provides an opportunity for a conditional approval as part of a fast-to-market strategy and includes an interim analysis that will be performed after the first 40 patients have been enrolled and evaluated for response.
Company raised approximately $29 million in capital through a private placement of $15.7 million of common stock, a private placement of $9.5 million of convertible preferred stock and an additional $4 million through unrelated business development activities and other sources. Net proceeds will be used to advance ArQule’s proprietary pipeline and for general business purposes, including working capital.
"The ArQule clinical pipeline is the strongest it has ever been, and the most recent positive developments are the compelling clinical data in Proteus syndrome and the granting of Rare Pediatric Disease Designation for miransertib in this indication," said Paolo Pucci, Chief Executive Officer of ArQule. "We are now well capitalized to see our pipeline assets through major inflection points."

"Based on the preliminary results from the phase 1 NIH-sponsored Proteus syndrome trial, miransertib is the first drug to demonstrate activity and achieve clinical proof of concept in this indication," said Dr. Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule. "We are thankful to the NIH for their work in identifying the mutation driving the disease, conducting the first clinical trial and ultimately identifying potential clinical endpoints. We plan to engage regulatory authorities to define a clinical path to registration in this indication. In parallel and consistent with the regulatory interactions we will continue to enroll in our ongoing phase 1/2 trial in Overgrowth Diseases driven by mutations in the PI3K or AKT pathway, including Proteus syndrome, and provide the drug under compassionate policy."

Revenues and Expenses

Revenues for the quarter ended September 30, 2017, were zero compared with revenues of $1,223,000 for the quarter ended September 30, 2016. Revenues in the nine-months ended September 30, 2017 were zero compared with revenues of $3,522,000 in the nine-months ended September 30, 2016. Revenue in the three and nine-month periods of 2016 is comprised of revenue from the Daiichi Sankyo tivantinib development agreement and the Kyowa Hakko Kirin exclusive license agreement. No further revenue is anticipated from these agreements.

Research and development expense in the third quarter of 2017 was $4,570,000, compared with $5,265,000 for the third quarter of 2016. Research and development expense decreased $0.7 million in the third quarter of 2017 primarily due to lower outsourced preclinical, clinical and product development costs.

Research and development expense in the nine-months ended September 30, 2017 was $14,747,000 compared with $13,800,000 in the nine-months ended September 30, 2016. The $0.9 million increase in research and development expense in the nine-months ended September 30, 2017 was primarily due to higher outsourced clinical and product development costs.

General and administrative expense was $1,762,000 in the third quarter of 2017 compared with $1,824,000 in the third quarter 2016.

General and administrative expense was $5,702,000 in the nine-months ended September 30, 2017 compared with $5,755,000 in the nine-months ended September 30, 2016.

2017 Financial Guidance

As a result of ArQule’s recent stock offerings and business development activities the company is updating 2017 guidance. For 2017, ArQule expects net use of cash to range between $25 and $27 million. Net loss is expected to range between $28 and $30 million, net loss per share is expected to range between $(0.38) and $(0.40) for the year. ArQule expects to end 2017 with between $47 and $49 million in cash and marketable securities.

Conference Call and Webcast

ArQule will hold its third quarter 2017 financial results call today, November 9, 2017 at 9:00 a.m. ET. The live webcast can be accessed in the "Investors & 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 "Investor and Media" section of our website, www.arqule.com, under "Events & Presentations."

On November 9, 2017 Northern Biologics Inc., a developer of first-in-class immuno-oncology products, reported that Philip Vickers, Ph.D., is joining the company as CEO and a member of the board. Stefan Larson, Ph.D., who served as Northern Biologics’ founding CEO since 2014, will remain on the company’s board (Press release, Northern Biologics, NOV 9, 2017, View Source [SID1234521915]).

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Dr. Vickers is a preeminent R&D leader with more than two decades of experience in the biotech and pharmaceutical industry. Most recently, he served as global head of R&D and a member of the Executive Committee for Shire during a period of great growth and multiple regulatory approvals for the company. Under Dr. Vickers’ tenure the clinical development pipeline grew to approximately 40 programs.

Prior to joining Shire, Dr. Vickers held a range of R&D positions of increasing responsibility at Merck & Co. Inc., Pfizer Inc., Boehringer Ingelheim and Resolvyx Pharmaceuticals Inc. Dr. Vickers holds a Ph.D. in biochemistry from the University of Toronto.

"We feel very fortunate to have attracted someone of Phil’s reputation and caliber as the new leader of Northern Biologics. He brings critical experience and a track record in drug development to take Northern Biologics into its next phase of growth," said Brad Bolzon, Ph.D., chairman of Northern Biologics and Versant Ventures.

"I am very excited to lead Northern Biologics at a time that we are going through the critical stage of transitioning programs from research into clinical development," said Dr. Vickers. "Our lead antibody against LIF and our portfolio of novel therapeutic candidates have potential to impact areas of oncology where there is a significant unmet medical need."

Early next year, Northern Biologics will begin clinical testing of its lead immuno-oncology asset, MSC-1, in a range of cancers, working with world-leading cancer institutes in Canada, Europe and the U.S. MSC-1 is a humanized antibody against a soluble cytokine called LIF (see "About LIF" below) with the desired effects of blocking immunosuppression and inhibiting the self-renewal of cancer stem cells. In addition, the company’s pipeline includes several antibodies acting on other relevant oncology targets, including those within the TIGIT and the TIM-3 pathways.

"I would like to thank Stefan for guiding Northern through its launch phase and for setting the company on its current path to success," said Dr. Bolzon. "We are very pleased that Stefan is returning to Versant as a Venture Partner with responsibility for leading new investments within Ontario and the broader region of Eastern Canada."

About LIF

LIF, or leukemia inhibitory factor, is an exciting emerging target in the immuno-oncology space. Northern Co-Founder Joan Seoane first elucidated a role for the cytokine in cancer in a seminal 2009 publication in Cancer Cell. Since that time, several independent labs have verified and published the role of LIF in many cancers. For example, as part of the recent mounting evidence, researchers reported this fall that high levels of LIF occur in pancreatic tumors. Reports on several other LIF findings, including the elucidation of the MSC-1 binding site, are expected within the next few months.

On November 9, 2017 Medigene AG (FSE: MDG1, Frankfurt, Prime Standard, TecDAX), a clinical stage immuno-oncology company focusing on the development of T cell immunotherapies for the treatment of cancer, reported its financial results for the first nine months of 2017 (Press release, MediGene, NOV 9, 2017, View Source [SID1234521817]).

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Major events:
– Clinical trial application submitted for its first study with proprietary T cell receptor (TCR)-modified T cells, MDG1011 – Study start expected for end of 2017
– Automated high throughput screening platform for the identification and validation of T cell receptors (TCRs) and neoantigens presented at various international conferences
– Preclinical data on Medigene’s first immunotherapy MDG1011 presented at AACR (Free AACR Whitepaper)
– Academic partner Oslo University presented additional data on compassionate use for DC vaccines against acute myeloid leukemia (AML) at AACR (Free AACR Whitepaper)
– Medigene raised EUR 20.7 m gross proceeds through placement of new shares with institutional investors in May

Key figures:
– Growing revenue from core business immunotherapies (bluebird bio partnership) of EUR 3,435 k (9M 2016: EUR 0 k) – collaboration progressing according to plan
– Increased total revenue of EUR 7,176 k (9M 2016: EUR 7,052 k); non-recurring gain in previous year from the sale of EndoTAG rights was largely offset by revenue from the partnership with bluebird bio
– Research and development expenses increased as planned to EUR 11,073 k (9M 2016: EUR 7,985 k) due to progress in proprietary immunotherapy programs
– Thereby EBITDA loss increased as planned to EUR 10,143 k (9M 2016: EUR 9,095 k) and net loss to EUR 10,690 k (9M 2016: net loss of EUR 5,725 k positively influenced by special effect / sale of financial assets of EUR 4,242 k in 2016)
– Cash, cash equivalents and time deposits of EUR 55,374 k as at September 30, 2017 (12/31/2016: EUR 52,630 k)
– Confirmation of financial guidance 2017

Prof. Dolores Schendel, CEO/CSO at Medigene AG, commented: "Medigene has made important progress in the past three quarters. We look forward to start the clinical development of our core program MDG1011, a TCR-based immunotherapy, which is expected to commence by the end of the year. In parallel, we are continuing the phase II part of the clinical trial with our DC vaccines, in which all patients will soon be included. In addition, development cooperation with bluebird bio is progressing very well and we are confident that we will be able to report concrete results of this collaboration in the coming months."

Dr. Thomas Taapken, CFO of Medigene AG, adds: "We are fully on track with our financial result. This demonstrates Medigene’s consistent commitment to the speedy implementation and expansion of our immunotherapy programs. Progress in collaboration with bluebird bio is already evident in increasing revenue from our core business immunotherapies. This underlines the power of our TCR platform which also fuels our own development programs we are driving forward independently."

Financial guidance 2017
Medigene confirms its financial guidance for 2017 as published in the 2016 annual report. The forecast reflects Medigene’s continued focus on the core business of immunotherapies.

– Revenues: The Company is expecting to generate total revenue of EUR 8 – 10 m in 2017. This forecast does not include future milestone payments from the existing R&D partnership with bluebird or revenue from any potential new transactions.
– R&D expenses: Due to the progress of Medigene’s clinical development programs in the field of immunotherapies and a further increase in headcount, Medigene is forecasting rising research and development expenses in the amount of EUR 16 – 18 m.
– Planned EBITDA loss of EUR 16 – 18 m in fiscal year 2017.
– For 2017, Medigene anticipates cash utilization to be EUR 23 – 27 m, partly due to non-recurring effects such as investments in laboratory infrastructure. This forecast does not include any cash inflows from possible future milestone payments from the existing R&D partnership with bluebird bio or from potential new transactions.
– Based on its current planning, the Company has sufficient financial resources for well beyond the forecast horizon of two years and up to the time that data from the DC trial and TCR trials become available.

The full version of the quarterly statement 9M-2017 can be downloaded here: www.medigene.com/investors-media/reports-presentations/

Conference call and webcast: A telephone conference (webcast) in English will be held today at 3:00 pm CEST (Munich/Frankfurt) / 9:00 am EDT (New York) and transmitted live in the internet. Access and transmission of the synchronized presentation slides and a recording of the presentation is available on the homepage of Medigene at www.medigene.com/investors-media/reports-presentations/webcasts/

On November 8, 2017 Kymab reported that its team will present data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 32nd Annual Meeting on its novel anti-ICOS antibody KY1044, the lead candidate in its immuno-oncology pipeline, which shows strong potential for inhibiting tumour growth (Press release, Kymab, NOV 8, 2017, View Source [SID1234537008]). The poster is available online.

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Kymab’s data, which is based on non-human tumour models, suggests that KY1044 strongly inhibits tumour growth, including complete remissions, in a number of cancer models, both as a monotherapy and acting synergistically in combination with checkpoint inhibitors such as anti-PD-L1.

"We are excited to unveil KY1044, the lead development product in our growing immuno-oncology franchise, where we are focusing on enabling the patient’s own immune system to boost its intrinsic ability to eradicate tumours," says Dr David Chiswell, CEO of Kymab.

"We believe that KY1044 has considerable potential and will provide patient benefits in a significant number of cancer indications."

KY1044 is a fully human antibody that allows the immune system to better recognise and kill tumours.

KY1044 has a dual mode of action: stimulating immune cells which are fighting the tumour, while also killing a type of cell known as T regulatory cells (Tregs). Tregs are often found in tumours and inhibit the immune response and prevent or slow the activity of anti-tumour T-cells.

Intratumoural Tregs express a high level of ICOS on their surface. Removing these cells using KY1044 allows the immune system to work more effectively at killing cancer cells.

Kymab plans to file an investigational new drug application for KY1044 in late 2018 and commence clinical trials in 2019. KY1044 will be evaluated in cancer patients both as a monotherapy and in combination with other immunotherapies.

The SITC (Free SITC Whitepaper) meeting is being held on 8-12 November 2017 at the Gaylord National Hotel & Convention Center, in Maryland, USA. The Kymab poster will be presented on Saturday, 11 November, 2017 at 12.30 to 14.00 and 18.00 to 20.00. Please see below for more information.

Notes to Editors
Issued Cambridge, UK, 8 November 2017
Read the PDF version of the issued Press Release

About Kymab’s SITC (Free SITC Whitepaper) Poster
Title: KY1044, a novel anti-ICOS antibody, elicits long term in vivo anti-tumour efficacy as monotherapy or in combination with immune checkpoint inhibitors.
Poster Number: 288
Date: Saturday, November 11, 2017
Session Time: 12.30 to 14.00 and 18.00 to 20.00
Location: Prince George’s Exhibition Hall DE, Gaylord National Hotel & Convention Center, in Maryland, USA
The Kymab poster is available at: View Source