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BioLineRx Reports Second Quarter 2016 Financial Results

On August 11, 2016 BioLineRx Ltd. (NASDAQ/TASE: BLRX), a clinical-stage biopharmaceutical company dedicated to identifying, in-licensing and developing promising therapeutic candidates, reported its financial results for the second quarter ended June 30, 2016 (Filing, Q2, BioLineRx, 2016, AUG 11, 2016, View Source [SID:1234514504]).

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Highlights and achievements during second quarter of 2016 and to date:

· Submission of regulatory filings to initiate Phase 2a study in pancreatic cancer for BL-8040 in combination with Merck’s KEYTRUDA, under immuno-oncology collaboration with Merck announced earlier this year

· Signing of additional immuno-oncology collaboration, this time with MD Anderson Cancer Center, for second Phase 2a study in pancreatic cancer for BL-8040 in combination with Merck’s KEYTRUDA

· Continued enrollment in large (n=194), randomized Phase 2b study for BL-8040 as consolidation treatment for AML patients following standard induction treatment

· Commercial launch of BL-5010 as OTC treatment for non-surgical removal of skin lesions by Omega Pharma (a division of Perrigo), following CE Mark approval in March

· In-licensing of liver fibrosis project under Novartis collaboration

· Philip A. Serlin appointed Chief Executive Officer, effective October 2016

Expected upcoming significant milestones for remainder of 2016:

· Initiation of Phase 2a study in pancreatic cancer, under immuno-oncology collaboration with Merck, following expected regulatory approval in Q3 2016

· Second Phase 2a immuno-oncology study in pancreatic cancer, under collaboration with MD Anderson Cancer Center, expected to commence by end of 2016

· Full set of data from Phase 2a study for BL-8040 in r/r AML to be presented at the Society of Hematologic Oncology (SOHO) annual meeting, September 7-10, 2016, in Houston, Texas

· Partial results from Phase 2 study for BL-8040 in stem-cell mobilization for allogeneic transplantation expected by end of 2016

· Regulatory submission for BL-7010 clinical efficacy study, for marketing purposes as food supplement

· Expansion of commercial rollout of BL-5010 by Omega to additional countries and development of 2nd OTC indication for the product

Philip A. Serlin, Chief Financial and Operating Officer of BioLineRx, remarked, "The second quarter of 2016 highlighted the continued execution of our plans as we advance and expand our lead oncology platform, BL-8040; see the initial market penetration of BL-5010; continue the development of BL-7010 as a food supplement; and maintain active asset screening and in-licensing activities with Novartis."

"We are pleased to have entered into an immuno-oncology collaboration with MD Anderson for a second Phase 2a study of BL-8040 with Merck’s KEYTRUDA in pancreatic cancer, which provides additional validation of the potential of our lead oncology drug platform in the cancer immunotherapy space. We continue to examine other potential collaborations in this space. In addition, we are looking forward to announcing full results from our successful Phase 2a study for relapsed and refractory AML at the upcoming Society of Hematology Oncology Meeting in September and we continue to push forward in our Phase 2b trial in an earlier treatment line for AML as a consolidation treatment following standard induction treatment. We also look forward to initiating our Phase 2a study in pancreatic cancer under our collaboration with Merck, expected by the end of this quarter," added Mr. Serlin.

"BL-5010, our first product in the market, is already being sold in a number of countries in Europe. Omega Pharma plans to continue to gradually launch the product in additional European countries over the next 6-9 months, and beyond that time frame, to additional territories. To date, we have not recorded material revenues from this collaboration, but we expect revenues to gradually increase as the first product launch expands and the second product launch commences. In addition, we have in-licensed a drug candidate for the treatment of liver fibrosis, specifically nonalcoholic steatohepatitis (NASH), under our strategic collaboration with Novartis for the co-development of selected Israeli-sourced novel drug candidates, and we expect to in-license additional promising projects to the collaboration in the next few months," continued Mr. Serlin.

"In closing, we ended the second quarter with $41.8 million of cash on our balance sheet. With our focus on achieving our expected milestones, we remain well positioned to carry out our strategic and operational plans," Mr. Serlin concluded.

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Financial Results for Second Quarter Ended June 30, 2016

Research and development expenses for the three months ended June 30, 2016 were $2.7 million, a decrease of $0.2 million, or 5.2%, compared to $2.9 million for the comparable period in 2015. The small decrease resulted primarily from lower spending on BL-7010 in the 2016 period, partially offset by increased spending related to clinical trial preparations for BL-8040. Research and development expenses for the six months ended June 30, 2016 were $5.3 million, a decrease of $0.8 million, or 13.5%, compared to $6.1 million for the comparable period in 2015. The decrease resulted primarily from lower expenditures for BL-7010 during the 2016 period, as well as the conclusion of one of the clinical trials for BL-8040 in 2015.

Sales and marketing expenses for the three months ended June 30, 2016 were $0.3 million, similar to the comparable period in 2015. Sales and marketing expenses for the six months ended June 30, 2016 were $0.5 million, similar to the comparable period in 2015.

General and administrative expenses for the three months ended June 30, 2016 were $0.9 million, a decrease of $0.1 million, or 12.5%, compared to $1.0 million for the comparable period in 2015. The small decrease resulted primarily from a decrease in salary-related payments and depreciation. General and administrative expenses for the six months ended June 30, 2016 were $1.8 million, similar to the comparable period in 2015.

The Company’s operating loss for the three months ended June 30, 2016 amounted to $3.8 million, compared with an operating loss of $4.2 million for the corresponding 2015 period. The Company’s operating loss for the six months ended June 30, 2016 amounted to $7.6 million, compared with an operating loss of $8.5 million for the corresponding 2015 period.

Non-operating income (expenses) for the three and six months ended June 30, 2016 and 2015 primarily relate to fair-value adjustments of warrant liabilities on the Company’s balance sheet. These fair-value adjustments, which were not material in the 2016 periods, are highly influenced by the Company’s share price at each period end (revaluation date).

Financial income (expenses), net for the three and six months ended June 30, 2016 and 2015 primarily relate to investment income earned on bank deposits, as well as banking fees. The decrease from 2015 to 2016 reflects a lower cash balance and a continued reduction in global investment yields.

The Company’s net loss for the three months ended June 30, 2016 amounted to $3.7 million, compared with a net loss of $4.8 million for the corresponding 2015 period. The Company’s net loss for the six months ended June 30, 2016 amounted to $7.2 million, compared with a net loss of $9.1 million for the corresponding 2015 period.

The Company held $41.8 million in cash, cash equivalents and short-term bank deposits as of June 30, 2016.

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Net cash used in operating activities was $7.4 million for the six months ended June 30, 2016, compared with net cash used in operating activities of $7.1 million for the comparable period in 2015. The $0.3 million increase in net cash used was primarily the result of a decrease in trade payables and accruals.

Net cash provided by investing activities for the six months ended June 30, 2016 was $4.2 million, compared to net cash used in investing activities of $17.9 million for the comparable period in 2015. The changes in cash flows from investing activities relate primarily to investments in, and maturities of, short-term bank deposits and other investments during the respective periods.

Net cash provided by financing activities for the six months ended June 30, 2016 was $1.5 million, compared to net cash provided by financing activities of $28.6 million for the comparable period in 2015. The decrease in cash flows from financing activities reflects the underwritten public offering which was completed in March 2015.

CANbridge Signs Agreement with Boehringer Ingelheim to Manufacture Inhibitory Antibody, CAN-017, for Esophageal Squamous Cell Cancer

On August 11, 2016 Boehringer Ingelheim and CANbridge Life Sciences reported that both parties have signed an agreement for the manufacture of CAN-017, an ErbB3 (HER3) inhibitory antibody to treat esophageal squamous cell cancer (ESCC), CANbridge plans to initiate a Phase IIa clinical trial for the treatment of ESCC in Greater China (Press release, CANbridge Life Sciences, AUG 11, 2016, View Source [SID:1234514523]).

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ESCC is the most prevalent form of esophageal cancer worldwide, with a high incidence in China and other Asian and developing countries. Every year, there are over 450,000 new cases diagnosed and over 50% of these case are occurring in China.

CANbridge acquired worldwide rights (exclusive of North America) to CAN-017 from the US company AVEO Oncology, where it was being developed as AV-203, and had completed a successful Phase I study in patients with solid tumor cancers. With the acquistion of CAN-017, CANbridge plans to expand outside of Asia for the first time, bringing the therapy to regions where ESCC patients have few options, after demonstrating proof-of-concept in China.

"Boehringer Ingelheim is a truly global manufacturer of biologics, capable of executing the intricacies of CAN-017 manufacture and meeting the requirements of the regulatory authorities," said James Xue, CANbridge Chairman and CEO. "It is a powerful ally for CANbridge as we look to advance the promising Western drug candidates we develop in Asia to other parts of the world, where patients are underserved. With the help of the manufacturing agreement with Boehringer Ingelheim, we look forward to bringing the new drug to China as early as possible."

"We are excited to enter into this important agreement with CANbridge Life Sciences. Boehringer Ingelheim is a leading player in developing and manufacturing biopharmaceuticals," said David Preston, Chairman and CEO of Boehringer Ingelheim mainland China, Hong Kong and Tai Wan, commented. "With our world-class bioprocess capabilities, we will help CANbridge and more biopharma enterprises step up efforts to bring innovative bio-medicines to the market and benefit patients quickly.

China initiated a pilot Marketing Authorization Holder (MAH) program in 10 provinces and municipalities recently, providing breakthrough policies breakthrough and a legal basis for biopharma CMO. Boehringer Ingelheim China was selected as one of the first biopharma CMO pilot projects in China as part of the MAH trial. Boehringer Ingelheim Biopharmaceuticals China will leverage its advanced manufacturing platform and management expertise to help Chinese enterprises bring their products into the global markets.

Checkpoint Therapeutics Announces Acceptance of IND Application for CK-101 – A Novel Third Generation EGFR Inhibitor

On August 11, 2016 Checkpoint Therapeutics, Inc. ("Checkpoint"), a Fortress Biotech (NASDAQ:FBIO) company, reported that its Investigational New Drug ("IND") application for CK-101, its novel, oral, third generation EGFR inhibitor product candidate, has been accepted by the U.S. Food and Drug Administration ("FDA") and is now active (Press release, Fortress Biotech, AUG 11, 2016, View Source [SID:1234514509]). The IND acceptance enables Checkpoint to begin a Phase 1/2 clinical trial of CK-101. The trial, expected to begin in September, is designed to assess the safety, tolerability and efficacy of CK-101 in EGFR T790M mutation-positive non-small cell lung cancer patients.

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"The acceptance of this IND marks an important milestone for Checkpoint and the CK-101 program," said James F. Oliviero, III, President and CEO of Checkpoint Therapeutics. "We look forward to commencing clinical trials for CK-101, which we plan to develop as both a monotherapy and in combination with our portfolio of immuno-oncology agents."

Checkpoint holds an exclusive worldwide license (except with respect to certain Asian countries) to CK-101 (also known as RX518), which it acquired from NeuPharma, Inc. in 2015.

About Checkpoint Therapeutics
Checkpoint Therapeutics, Inc. ("Checkpoint"), a Fortress Biotech company, is an innovative, immuno-oncology biopharmaceutical company focused on the acquisition, development and commercialization of novel, non-chemotherapy, immune-enhanced combination treatments for patients with solid tumor cancers. Checkpoint aims to acquire rights to these technologies by licensing the rights or otherwise acquiring an ownership interest in the technologies, funding their research and development and eventually either out-licensing or bringing the technologies to market. Currently, Checkpoint is developing a portfolio of fully human immuno-oncology targeted antibodies generated in the laboratory of Dr. Wayne Marasco, MD, PhD, a professor in the Department of Cancer Immunology and AIDS at the Dana-Farber Cancer Institute. The portfolio of antibodies Checkpoint licensed from Dana-Farber includes antibodies targeting Programmed death-ligand 1 ("PD-L1"), Glucocorticoid-induced TNFR related protein ("GITR") and carbonic anhydrase IX ("CAIX"). Checkpoint plans to develop these novel immuno-oncology and checkpoint inhibitor antibodies on their own and in combination with each other, as data suggests that combinations of these targets may work synergistically together. Checkpoint has also licensed and is developing three oral, small molecule, targeted anti-cancer agents, consisting of an inhibitor of epidermal growth factor receptor ("EGFR") mutations, an inhibitor of the bromodomain and extra-terminal ("BET") protein, BRD4, and an inhibitor of poly (ADP-ribose) polymerase ("PARP"). Checkpoint will also seek to add additional immuno-oncology drugs and other targeted therapies in order to create wholly-owned proprietary combinations that leverage the immune system and complimentary mechanisms. Checkpoint is headquartered in New York City. For more information, visit www.checkpointtx.com.

Advaxis Announces that Phase 2 Head and Neck Cancer Study with AXAL Advances to Second Stage

On August 11, 2016 Advaxis, Inc. (NASDAQ:ADXS), a clinical-stage biotechnology company developing cancer immunotherapies, reported that its Phase 2 "window of opportunity" clinical study of its lead immunotherapy candidate, axalimogene filolisbac (AXAL), in patients with late-stage HPV-associated oropharyngeal cancer (HPVOPC) met its stage 1 primary objective and is advancing into the second stage of the clinical study (Press release, Advaxis, AUG 11, 2016, View Source [SID:1234514508]).

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This is an investigator-initiated prospective clinical study of patients with stage I-IV HPVOPC who are to undergo ablative trans-oral robotic surgery (TORS) as a preoperative treatment. The clinical study leverages the five- to six-week period between diagnosis and TORS, making it possible to analyze and compare the tumor microenvironment as well as peripheral blood samples collected before and after AXAL treatments.

The clinical study is designed to show that AXAL is highly immunogenic and worth further investigation if the overall rate of vaccine-induced T-cell responses is 75 percent or more. By looking at both IFN-γ and TNFα expressing T cells in the peripheral blood, it was found that systemic HPV-reactive T-cell responses were increased in enough patients treated with AXAL to meet the stage 1 immune response target. This evidence of systemic response is consistent with a recent abstract presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) describing increased cytotoxic T-cell infiltration into the tumor microenvironment in HPVOPC patients treated with AXAL.

"The assessment of the TNFα and IFN-γ response based on data from eight of the anticipated nine patients to be enrolled in stage 1 confirmed that the clinical study has already met the target for the overall rate of vaccine-induced T-cell response, paving the way for us to progress to stage 2," said the study’s lead investigator Andrew G. Sikora, MD, PhD, of the Bobby R. Alford Department of Otolaryngology-Head and Neck Surgery at Baylor College of Medicine. "Together with our prior published data showing increased intratumoral T-cell infiltration in a significant number of AXAL treated patients, these data provide the confidence needed to move forward with the definitive evaluation of its immunogenicity."

Stage 2 of the clinical study will enroll up to 13 patients with late-stage HPVOPC. This stage of the clinical study will be conducted at the Icahn School of Medicine at Mount Sinai and a second investigative site anticipated to be the Baylor College of Medicine. The study received a three-year $1.1 million grant from the U.S. Food and Drug Administration’s Office of Orphan Products Development, which funds research for the development of products for rare diseases.

HPV-Associated Head and Neck Cancers

More than 90 percent of head and neck squamous cell oropharyngeal cancers originate from the mucosal linings of the oral cavity, pharynx or larynx. Currently, 60 to 80 percent of these cancers are caused by HPV. Head and neck cancers are treated by surgical removal of the cancer and lymph nodes, often followed by radiation and chemotherapy based on the extent of the disease. While patients may achieve good long-term survival, standard treatments can change their physical appearance and are associated with significant short and long-term toxicities which may interfere with salivary gland function, taste, smell, and the ability to swallow.

The incidence of HPV-associated head and neck cancers has been increasing at an epidemic rate, while head and neck cancers from other causes have been decreasing. According to the World Health Organization, approximately 15 to 20 percent of the 400,000 new cases of head and neck cancer are HPV-related. In the US, there are about 12,000 new cases of HPV-associated head and neck cancer per year and it affects men about 3 times more frequently than women. HPV-associated head and neck cancer is growing fastest in developed countries like the U.S.

About Axalimogene Filolisbac

AXAL is Advaxis’ lead Lm Technology immunotherapy candidate for the treatment of HPV-associated cancers and is in clinical trials for three potential indications: invasive cervical cancer, head and neck cancer, and anal cancer. AXAL has Orphan Drug Designation in the U.S. for the treatment of these three indications. In a completed randomized Phase 2 study in recurrent/refractory cervical cancer, AXAL showed apparent prolonged survival, objective tumor responses, and a manageable safety profile alone or in combination with chemotherapy, supporting further development of the company’s Lm Technology.

Epizyme Doses First Patient in Global Phase 2 Study Evaluating Tazemetostat in Mesothelioma

On August 11, 2016 Epizyme, Inc. (NASDAQ:EPZM), a clinical-stage biopharmaceutical company creating novel epigenetic therapies, reported that the first patient has been dosed in the Company’s global phase 2 study evaluating tazemetostat, a first-in-class EZH2 inhibitor, for the treatment of adults with mesothelioma characterized by BAP1 loss-of-function (Press release, Epizyme, AUG 11, 2016, View Source [SID:1234514505]).

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"Mesothelioma remains a cancer that is very difficult to treat and has an extremely poor prognosis," said Marjorie Zauderer, M.D., medical oncologist at Memorial Sloan Kettering Cancer Center. "The overwhelming majority of patients are refractory to or relapse following first-line treatment with chemotherapy leaving them with no approved treatment options. The preclinical data demonstrating that EZH2 inhibition may play a role in the treatment of mesothelioma is promising, and I look forward to exploring the potential for tazemetostat as a targeted agent for patients with this devastating disease."

Mesothelioma affects nearly 12,000 people each year in major markets, of which 40 to 60% have tumors characterized by BAP1 loss-of-function. The median overall survival for all patients with mesothelioma generally ranges from eight to 14 months from the time of diagnosis. The most commonly used first-line treatment consists of cisplatin or carboplatin combined with pemetrexed, which is the only drug approved (2004) for the treatment of mesothelioma1.

"Patient dosing in this phase 2 study marks another milestone for Epizyme and the expansion of our clinical development program into a new cancer indication for which there is a significant medical need," said Peter Ho, M.D., Ph.D., Executive Vice President and Chief Medical Officer, Epizyme. "With this study, Epizyme now has four registration-supporting monotherapy trials underway with tazemetostat, and plans for two combination trials to be initiated later this year."

Phase 2 Study Design
The international, open-label, phase 2 study will evaluate tazemetostat as a monotherapy in a total of 67 patients at multiple sites in the United States, United Kingdom and France. Tazemetostat is orally administered at a dose of 800 mg twice-daily. The safety and pharmacokinetic profile of tazemetostat will first be evaluated in 12 patients with relapsed or refractory mesothelioma regardless of BAP1 status. Subsequently, a total of 55 patients with relapsed or refractory mesothelioma characterized by BAP1 loss-of-function will be treated using a two-stage study design. The primary endpoint of the study is disease control rate (complete response, partial response or stable disease) at 12 weeks. Secondary endpoints include overall response rate, progression-free survival, duration of response and overall survival.

EZH2 Inhibition in Mesothelioma
Tazemetostat is a first-in-class small molecule inhibitor of EZH2 created by Epizyme using its proprietary drug development platform. Aberrant EZH2 activity results in misregulation of genes that control cell proliferation and has been associated with a diverse set of human cancers. Preclinical findings from published reports suggest that mesothelioma, and particularly mesothelioma characterized by BAP1 loss of function, may be sensitive to EZH2 inhibition2. Mesothelioma characterized by BAP1 loss of function accounts for 40 to 60 percent of the approximately 12,000 new mesothelioma cases each year in major markets3-6.

About the Tazemetostat Clinical Trial Program
Tazemetostat, a first-in-class EZH2 inhibitor, is currently being studied in ongoing phase 2 programs in non-Hodgkin lymphoma (NHL), certain genetically defined solid tumors, including INI1-negative and SMARCA4-negative tumors and synovial sarcoma, and mesothelioma.

The company plans to initiate additional clinical trials of tazemetostat in 2016, including a combination with R-CHOP in collaboration with the Lymphoma Study Association and a combination with Tecentriq (atezolizumab) in collaboration with Genentech, a member of the Roche Group, both in patients with diffuse large B-cell lymphoma.