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BioLineRx Reports Second Quarter 2017 Financial Results

On August 8, 2017 BioLineRx Ltd. (NASDAQ/TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology and immunology, reported its financial results for the second quarter ended June 30, 2017 (Filing, Q2, BioLineRx, 2017, AUG 8, 2017, View Source [SID1234520089]).

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Highlights and achievements during the second quarter 2017 and to date:

Continued execution on multiple clinical development programs for the Company’s lead project, BL-8040:

Announced plans to initiate Phase 3 pivotal study with BL-8040 as novel stem cell mobilization treatment for autologous bone-marrow transplantation in H2 2017, following successful meeting with the FDA;
Reported regulatory submissions of three Phase 1b/2 trials for BL-8040 in combination with atezolizumab, Genentech’s anti-PDL1 cancer immunotherapy agent, in pancreatic, gastric and non-small cell lung cancers, under immunotherapy collaboration with Genentech, a member of the Roche Group. All three studies will be conducted as part of MORPHEUS, Roche’s Novel Cancer Immunotherapy Development Platform, and are expected to commence in H2 2017;
Reported filing of regulatory submissions to commence Phase 1b/2 trial for BL-8040 in combination with Genentech’s atezolizumab in acute myeloid leukemia (AML), to be led by BioLineRx. This study is expected to commence in H2 2017;
Announced initiation of first Phase 1b/2 trial under immunotherapy collaboration with Genentech – in pancreatic cancer;

The Company also announced an additional, direct investment by BVF Partners, L.P., its largest shareholder, increasing its economic interest in the Company to 24.9%. The $9.6 million investment was priced at $1.13 per unit, each unit consisting of 1 ordinary share, 0.35 of Series A warrant with an exercise price of $2.00 per share, and 0.35 of Series B warrant with an exercise price of $4.00 per share. The warrants are exercisable for a period of 4 years.

Expected significant upcoming milestones for 2017 and 2018:

Partial results from immuno-oncology Phase 2a study in pancreatic cancer for BL-8040 in combination with Merck’s KEYTRUDA expected in H2 2017; top line results expected in H2 2018;
Initiation of Phase 3 pivotal study for BL-8040 in stem-cell mobilization for autologous transplantation planned for H2 2017;
Initiation of Phase 1b/2 immuno-oncology studies for BL-8040 in combination with Genentech’s atezolizumab in gastric cancer and non-small cell lung cancer, as well as AML, all expected in H2 2017; partial results expected in H2 2018;
Initiation of Phase 1 immuno-oncology study for AGI-134 in several solid tumor indications expected in H1 2018;
Top-line results of Phase 2 study for BL-8040 in stem-cell mobilization for allogeneic transplantation expected by mid-2018.

Philip A. Serlin, Chief Executive Officer of BioLineRx, remarked, "We are pleased to report second quarter-to-date activities that reinforce our focus to deliver on our objectives. This included timely initiation of our cancer immunotherapy collaboration with Genentech for pancreatic cancer, as well as regulatory advancements for additional indications in other solid tumors, AML, and stem cell mobilization. Thus, by the end of the year, we remain poised to have one Phase 3 and seven Phase 2 or 1b/2 clinical trials up and running, in addition to announcing partial results in our Phase 2 study in pancreatic cancer under our immunotherapy collaboration with Merck."

"We are also pleased to have received a strong vote of confidence from BVF Partners last month. The $9.6 million direct investment we received will allow us to accelerate the development of our clinical programs. With over $60 million in cash on a pro forma basis, including BVF’s investment, as of June 30th, we have a strong balance sheet which will enable us to fully execute on our current operating plans," Mr. Serlin concluded.

Financial Results for the Second Quarter Ended June 30, 2017

Research and development expenses for the three months ended June 30, 2017 were $4.0 million, an increase of $1.3 million, or 48.2%, compared to $2.7 million for the three months ended June 30, 2016. The increase resulted primarily from spending on AGI-134 and BL-8040 in the 2017 period. Research and development expenses for the six months ended June 30, 2017 were $7.7 million, an increase of $2.4 million, or 45.0%, compared to $5.3 million for the six months ended June 30, 2016. The reason for the increase is the same as that presented in the three-month comparison above.

Sales and marketing expenses for the three months ended June 30, 2017 were $0.3 million, similar to the comparable period in 2016. Sales and marketing expenses for the six months ended June 30, 2017 were $1.0 million, an increase of $0.5 million, or 86.3%, compared to $0.5 million for the six months ended June 30, 2016. The increase resulted primarily from market research activities and one-time professional fees related to business development activities.

General and administrative expenses for the three months ended June 30, 2017 were $0.8 million, similar to the comparable period in 2016. General and administrative expenses for the six months ended June 30, 2017 were $1.8 million, similar to the comparable period in 2016.

The Company’s operating loss for the three months ended June 30, 2017 amounted to $5.2 million, compared with an operating loss of $3.9 million for the corresponding 2016 period. The Company’s operating loss for the six months ended June 30, 2017 amounted to $10.5 million, compared with an operating loss of $7.6 million for the corresponding 2016 period.

Non-operating income (expenses) for the three and six months ended June 30, 2017 and 2016 were not material, and primarily relate to fair-value adjustments of warrant liabilities on the balance sheet.

Net financial income amounted to $0.3 million for the three months ended June 30, 2017 compared to net financial income of $0.1 million for the three months ended June 30, 2016. Net financial income amounted to $0.8 million for the six months ended June 30, 2017 compared to net financial income of $0.2 million for the six months ended June 30, 2016. The increase in net financial income relates primarily to gains recorded on foreign currency hedging transactions and investment income earned on our bank deposits.

The Company’s net loss for the three months ended June 30, 2017 amounted to $4.9 million, compared with a net loss of $3.7 million for the corresponding 2016 period. The Company’s net loss for the six months ended June 30, 2017 amounted to $9.8 million, compared with a net loss of $7.2 million for the corresponding 2016 period.

The Company held $52.6 million in cash, cash equivalents and short-term bank deposits as of June 30, 2017. In July 2017, the Company completed a direct placement of its securities for net proceeds of $9.5 million.

Net cash used in operating activities was $8.0 million for the six months ended June 30, 2017, compared with net cash used in operating activities of $7.5 million for the six months ended June 30, 2016. The $0.5 million increase in net cash used in operating activities during the six-month period in 2017, compared to the six-month period in 2016, was primarily the result of an increase in the Company’s operating loss in the 2017 period.

Net cash used in investing activities for the six months ended June 30, 2017 was $16.0 million, compared to net cash provided by investing activities of $4.2 million for the six months ended June 30, 2016. The changes in cash flows from investing activities relate primarily to investments in, and maturities of, short-term bank deposits, as well as the investment in Agalimmune.

Net cash provided by financing activities for the six months ended June 30, 2017 was $28.3 million, compared to net cash provided by financing activities of $1.6 million for the six months ended June 30, 2016. The increase in cash flows from financing activities primarily reflects the public offering completed in April 2017.

Aptose Reports Results for the Second Quarter Ended June 30, 2017

On August 8, 2017 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ:APTO) (TSX:APS), a clinical-stage company developing highly differentiated therapeutics that target the underlying mechanisms of cancer, reported financial results for the three months ended June 30, 2017 and reported on corporate developments (Press release, Aptose Biosciences, AUG 8, 2017, View Source [SID1234520084]). Unless specified otherwise, all amounts are in Canadian dollars.

The net loss for the quarter ended June 30, 2017 was $3.2 million ($0.15 per share) compared with $5.6 million ($0.46 per share) in the quarter ended June 30, 2016. Total cash and cash equivalents and investments as of June 30, 2017 were $14.2 million (or $10.9 million US dollars) which, based on information currently available and current expected cash burn, provides the Company with sufficient resources to fund research and development and operations into Q3 2018.

"We have made tremendous progress with both of our hematology product candidates, CG’806 and APTO-253, during the second quarter of this year," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer.
"CG’806 is an oral first-in-class pan-FLT3/BTK multi-kinase inhibitor that we are developing for patients with acute myeloid leukemia (AML) and certain B-cell malignancies. Importantly, we’ve developed a scalable manufacturing process and have selected the formulation that we intend to advance into clinical trials in 2018. For APTO-253, a clinical-stage compound that inhibits expression of the c-Myc oncogene, we generated preliminary data that point to the root cause of the manufacturing and stability setbacks with the drug product. Formal root cause studies are underway and may allow us to bring APTO-253 back into the clinic as a potential treatment for acute myeloid leukemia."

Corporate Highlights
CG’806 preclinical data presented publicly – In May 2017, the company presented preclinical data for its pan-FLT3/BTK multi-kinase inhibitor CG’806 in two separate posters at the 2017 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Conference for Hematologic Malignancies in Boston, MA. Aptose scientists, along with researchers from the Knight Cancer Institute at Oregon Health & Science University (OHSU), presented data relating to the potency of CG’806 against specimens derived from patients with various hematologic malignancies. In a separate presentation, Aptose scientists, with researchers from the MD Anderson Cancer Center, presented data demonstrating potency of CG’806 against AML cells harboring wild type or specific mutant forms of FLT3.

CG’806 manufacturing and preclinical progress – Aptose has developed a scalable synthetic route for the manufacture of bulk quantities of CG’806 and has selected an oral formulation that we plan to take into first-in-human clinical trials. Preclinical studies with various types of AML and B cell malignancy cells demonstrated differential actions on distinct kinases and pathways in different genetic backgrounds, leading to unanticipated potency against a wide range of these cancer cells. In addition, we solved the x-ray crystal structures of CG’806 with the BTK enzyme, which further elucidated the molecule’s strong interaction with the BTK active site and explains why CG’806 continues to inhibit the C481S mutant form of BTK. Together, the data support development of CG’806 for various forms of AML and for patients who i) have relapsed, ii) are refractory or iii) are intolerant to ibrutinib or other BTK inhibitors with B cell malignancies that may continue to be sensitive to CG’806.

Intellectual Property Protection – On August 4th, 2017 we received a notice from the USPTO stating that our U.S. Patent Application for CG’806 has been allowed for issuance as a patent. The application claims numerous compounds, including the CG’806 compound, pharmaceutical compositions comprising the CG’806 compound, and methods of treating various diseases. It is important to note that the notice of allowance is not a grant of patent rights and although it is uncommon, the USPTO can withdraw the allowed application from issuance.

APTO-253 root cause analysis update – Preliminary root cause analyses studies have provided preliminary data that could point to the identification of the reason for the manufacturing and stability issues that resulted in a clinical hold of the Phase lb clinical trial of APTO-253. If the ongoing studies confirm these data, Aptose will bring these findings to the FDA with the hope of returning APTO-253 to a state that it can be reintroduced into the clinical trial. APTO-253, which effectively inhibits expression of the c-Myc oncogene, is a potential treatment for AML, and we now have identified a cellular target that can explain the means by which APTO-253 inhibits the c-Myc gene expression and induces other cellular sequelae.

ASH abstracts submitted – Aptose has submitted five abstracts for CG’806 and two abstracts for APTO-253 for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December. CG’806 has been shown to be a multi-targeted pan-FLT3/BTK inhibitor, but it also impacts other relevant oncogenic targets. APTO-253 has demonstrated activity as a c-Myc inhibitor though the interaction with a specific cellular target and can result in a synthetic lethal when cancer cells house specific mutations. This research and other mechanistic findings for both molecules are highlighted and expanded upon in the abstracts.
Financial Results
Net loss for the three months ended June 30, 2017 was $3.2 million ($0.15 per share) compared with $5.6 million ($0.46 per share) during the three months ended June 30, 2016. Our net loss for the six months ended June 30th, 2017 was $7.6 million ($0.39 per share) compared with a loss of $10.7 million ($0.88 per share) during the six months ended June 30, 2017.
The decrease in the net loss during the three and six months ended June 30, 2017 compared with the three and six months ended June 30, 2016 is primarily related to the $1.3 million option fee paid to CrystalGenomics for its CG’806 technology in June of 2016, the cancellation of the LALS/Moffitt collaboration, and lower costs associated with the APTO-253 program, offset by development activities related to the CG’806 development program which started in the second half of fiscal 2016.
Aptose utilized cash of $3.6 million in operating activities in the three months ended June 30, 2017 compared with $4.6 million in the three months ended June 30, 2016. The decrease in cash used in operating activities in the current period is due mostly to lower operating expenses in the current period, offset by decrease in accounts payable and accrual balances during the three months ended June 30, 2017. For the six months ended June 30, 2017, Aptose utilized cash of $7.1 million compared with $9.2 million in the six months ended June 30, 2016. The decrease in cash used in operations is due mostly to lower operating expenses in the current period.
Research and Development
Research and development expenses totaled $1.5 million in the three months ended June 30, 2017 compared with $3.3 million in the three months ended June 30, 2016. Research and development expenses totaled $3.8 million in the six months ended June 30, 2017 compared with $5.6 million in the six months ended June 30, 2016. Research and development costs consist of the following:
Components of research and development expenses:
Three months ended Six months ended
June 30, June 30, June 30, June 30,
(in thousands) 2017 2016 2017 2016

CrystalGenomics Option Fee - 1,294 - 1,294
Program costs – CG ‘806 479 19 1,019 19
Program costs – APTO-253 451 834 1,553 1,874
Program costs – LALS/Moffitt - 464 - 949
Salaries 422 562 988 1,284
Stock based compensation 98 109 166 165
Depreciation of equipment 12 11 31 23
$ 1,462 $ 3,293 $ 3,757 $ 5,608
Expenditures for the three and six months ended June 30, 2017 were lower than the expenses incurred in the three and six months ended June 30, 2016 mostly related to the US$1.0 million option fee paid to CrystalGenomics in June of 2016 and savings on the APTO-253 program as the Company’s decision to reprioritize its resources towards its CG’806 program. Higher program costs associated with the Company’s CG’806 program were offset by savings that resulted from the decision made in December 2016 to cancel the LALS/Moffitt collaboration.
The changes in research and development expenses resulted from the following:
In the comparative period, the Company paid US$1.0 million ($1.3 million) to CrystalGenomics for an option fee related to the CG’806 technology and in that period began research and development activities for this program.
Research and development activities, including formulation studies and PK studies, related to CG’806 development program;
Reduced expenditures on the APTO-253 program. In the current three and six month periods, the Company was conducting root cause analysis to determine the cause of the manufacturing issues. In the comparative periods the Company was actively manufacturing a new clinical batch.
Lower salaries expense mostly related to severance payments made in the three months ended March 31, 2016 when research headcount was reduced and savings resulting from the reduced headcount.
Savings from cancellation of the LALS/Moffitt collaboration which was active in the three and six months ended June 30, 2016. There are no costs related to this program in the current period.
General and Administrative
General and administrative expenses totaled $1.8 million in the three months ended June 30, 2017, compared to $2.3 million in the three months ended June 30, 2016. For the six month period ended June 30, 2017, general and administrative expenses totaled $3.9 million compared with $5.0 million in the same period in the prior year. General and administrative costs consist of the following:
Components of general and administrative expenses:
Three months ended Six months ended
June 30, June 30, June 30, June 30,
(in thousands) 2017 2016 2017 2016

General and administrative excluding salaries $ 755 $ 822 $ 1,697 $ 1,955
Salaries 596 823 1,731 1,798
Stock-based compensation 463 677 476 1,156
Depreciation of equipment 19 21 30 42
$ 1,833 $ 2,343 $ 3,934 $ 4,951
General and administrative expenses, excluding salaries, decreased in the three months ended June 30, 2017, compared with the three months ended June 30, 2016. The decrease is mostly the result of lower travel, consulting and rent costs in the current year related to cost containment initiatives taken in the prior fiscal year. Salary expenses in the three months ended June 30, 2017, decreased in comparison with the three months ended June 30, 2016, mostly due to reduced headcount.
General and administrative expenses excluding salaries, decreased in the six months ended June 30, 2017, compared with the six months ended June 30, 2016. The decrease is mostly the result of lower travel, consulting and rent costs in the current year related to cost containment initiatives taken in the prior fiscal year. Salaries expense for the six months ended June 30, 2017 is comparable to the salaries expense in the six months ended June 30, 2016. Severance and separation costs incurred in the three months ended March 31, 2017 are offset by savings in the three months ended June 30, 2017 as a result of the lower headcount.
Stock-based compensation decreased in the three and six months ended June 30, 2017, compared with the three and six months ended June 30, 2016, due to large forfeitures in the three months ended March 31, 2017 and also due to grants in prior periods having a greater fair value than the grants issued in the three months ended June 30, 2017, and therefore contributing to higher stock-based compensation in the three and six months period ended June 30, 2016.

Aptose Biosciences Inc.
Condensed Consolidated Interim Statements of Loss and Comprehensive Loss
(unaudited)

Three Three Six Six
months ended months ended months ended months ended
(amounts in 000’s of Canadian Dollars except for per common share data) June 30, 2017 June 30, 2016 June 30, 2017 June 30, 2016
REVENUE $ - $ - $ - $ -

EXPENSES
Research and development 1,462 3,293 3,757 5,608
General and administrative 1,833 2,343 3,934 4,951
Operating expenses 3,295 5,636 7,691 10,559
Finance expense – 9 – 205
Finance income (54 ) (33 ) (95 ) (80 )
Net financing income (54 ) (24 ) (95 ) 125
Net loss for the period 3,241 5,612 7,596 10,684

Other comprehensive loss
Items that may subsequently be reclassified to earnings:
Foreign currency translation loss 365 – 488 –
Comprehensive loss for the period 3,606 5,612 8,084 10,684

Basic and diluted loss per common share $ 0.15 $ 0.46 $ 0.39 $ 0.88

The press release, the financial statements and the management’s discussion and analysis for the quarter ended June 30, 2017 will be available on SEDAR at www.sedar.com and EDGAR at www.sec.gov/edgar.shtml

Agios Reports Second Quarter 2017 Financial Results

On August 8, 2017 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported business highlights and financial results for the second quarter ended June 30, 2017 (Filing, Q2, Agios Pharmaceuticals, 2017, AUG 8, 2017, View Source [SID1234520080]). In addition, Agios highlighted select corporate milestones and preclinical and clinical data from our clinical development programs .

"Our second quarter progress was followed by a remarkable milestone for any biotechnology company, the full approval of our first product, IDHIFA, a treatment for patients with IDH2m R/R AML developed in partnership with Celgene," said David Schenkein, M.D., chief executive officer at Agios. "We are pursuing a similar regulatory strategy for ivosidenib, our wholly owned IDH1m inhibitor, and we remain on track to submit our NDA by year-end. With the pivotal program designed for AG-348 in PK deficiency, we are working to initiate two trials in the first half of 2018 for this rare anemia, which currently has no approved therapies."

SECOND QUARTER 2017 HIGHLIGHTS & RECENT PROGRESS

IDH Mutant Inhibitors:

On August 1, 2017, the U.S. Food and Drug Administration (FDA) granted Celgene full approval of IDHIFA (enasidenib) for the treatment of patients with relapsed or refractory AML (R/R AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA approved test. IDHIFA, an oral targeted inhibitor of the IDH2 enzyme, is the first and only FDA-approved therapy for patients with R/R AML and an IDH2 mutation.
Presented updated data from the Phase 1 trial of IDHIFA in IDH2m R/R AML at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and updated data from the Phase 1/2 trial of IDHIFA in IDH2m R/R AML at the 22ndCongress of the European Hematology Association (EHA) (Free EHA Whitepaper). The data presentations demonstrated durable complete responses in patients with IDH2m R/R AML and a safety profile consistent with previously reported data. Read the ASCO (Free ASCO Whitepaper) data here and the EHA (Free EHA Whitepaper) data here.
Presented the first data from the cholangiocarcinoma expansion cohort of the ongoing Phase 1 trial of ivosidenib in advanced IDH1m positive solid tumors at ASCO (Free ASCO Whitepaper). Read the ASCO (Free ASCO Whitepaper) data here.
Initiated a global, registration-enabling Phase 3 study (AGILE) combining ivosidenib and VIDAZA in newly diagnosed AML patients with an IDH1 mutation ineligible for intensive chemotherapy.
Completed enrollment of the dose-escalation phase of the ongoing Phase 1 study of AG-881 in IDHm positive glioma.
Rare Genetic Diseases

Finalized two global, pivotal trial designs evaluating AG-348 in adults with pyruvate kinase (PK) deficiency:
– A randomized, placebo-controlled trial with a 1:1 randomization expected to enroll approximately 80-100 non-transfusion dependent patients. The primary endpoint of the study is the proportion of patients who achieve at least a 1.5 gram per deciliter (g/dL) increase in hemoglobin.
– A single arm trial of approximately 20 regularly transfused patients with a primary endpoint of reduction in transfusion burden over six months.
Presented updated data from the AG-348 Phase 2 DRIVE PK trial in PK deficiency at EHA (Free EHA Whitepaper) showing consistent safety and efficacy data. Read the EHA (Free EHA Whitepaper) data here.
Discovery Research

In April, we entered into a new global license agreement with Aurigene Discovery Technologies Limited to research, develop and commercialize small molecule inhibitors for an undisclosed cancer metabolism target.
Corporate:

In April, Agios completed an underwritten public offering of 5,808,080 shares of common stock, which included the full exercise of the underwriters’ option to purchase 757,575 shares, at the offering price of $49.50 per share, resulting in proceeds, net of underwriting discounts and commissions, of approximately $270.2 million.
EXPECTED 2H 2017 DATA PRESENTATIONS

First preclinical data of AG-881 in IDHm solid and hematologic malignancies have been submitted for presentation at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in October.
Updated data from the glioma expansion cohort of the ongoing Phase 1 trial of ivosidenib in advanced IDH1m positive solid tumors have been submitted for presentation at the 2017 Society for NeuroOncology Annual Meeting in November.
First data from the expansion phase of the ongoing Phase 1 trial of ivosidenib in IDH1m R/R AML and advanced hematologic malignancies have been submitted for presentation at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH) (Free ASH Whitepaper) in December.
First data from the ongoing Phase 1 combination trial of IDHIFA or ivosidenib with standard-of-care intensive chemotherapy ("7 +3" and consolidation) in patients with newly diagnosed AML with an IDH2 or IDH1 mutation have been submitted for presentation at ASH (Free ASH Whitepaper).
Updated data from the Phase 2 DRIVE PK trial with AG-348 in patients with PK deficiency, including longer follow-up and secondary analyses, have been submitted for presentation at ASH (Free ASH Whitepaper).
Updated data from the PK Deficiency Natural History Study being conducted with Boston Children’s Hospital have been submitted for presentation at ASH (Free ASH Whitepaper).
KEY UPCOMING MILESTONES

The company expects to achieve the following key milestones:

Submit an NDA (New Drug Application) to the U.S. FDA for ivosidenib for IDH1m positive R/R AML by the end of 2017.
Initiate two global, pivotal trials of AG-348 in PK deficiency in the first half of 2018.
Submit an Investigational New Drug (IND) application for AG-270, the development candidate targeting MTAP-deleted tumors, by the end of 2017.
SECOND QUARTER 2017 FINANCIAL RESULTS

Collaboration revenue was $11.3 million for the quarter ended June 30, 2017, compared to $7.0 million for the comparable period in 2016. Collaboration revenue increased compared to the prior year period partially due to reimbursement by Celgene of our share of the commercialization effort for IDHIFA.

Research and development (R&D) expense was $79.8 million, including $8.2 million of stock-based compensation expense, for the quarter ended June 30, 2017, compared to $50.8 million, including $6.6 million in stock-based compensation expense, for the quarter ended June 30, 2016. The increase in R&D expense was primarily attributable to activities related to the ivosidenib program, including manufacturing-related activities needed to prepare for a potential NDA submission in 2017, start-up costs for the Phase 3 AGILE clinical trial, and on-going site activation and patient enrollment of the Phase 3 ClarIDHy clinical trial. In addition, Celgene was responsible for approximately half of the development costs for ivosidenib during the quarter ended June 30, 2016. As of August 2016, Agios is responsible for all ivosidenib development costs. R&D expense also increased compared to the quarter ended June 30, 2016 due to preparations for initiation of the AG-348 pivotal program in the first half of 2018, including manufacturing-related activities. Lastly, the $3.0 million upfront payment as part of the Aurigene license agreement was included in R&D during the quarter ended June 30, 2017.

General and administrative (G&A) expense was $16.1 million, including $4.0 million stock-based compensation expense, for the quarter ended June 30, 2017, compared to $12.6 million, including $4.4 million of stock-based compensation expense, for the quarter ended June 30, 2016. The increase in G&A expense was attributed to an increase of $1.0 million in personnel costs related to an increase in our internal headcount and an increase of $1.9 million related to support our growing commercial organization.

Net loss for the quarter ended June 30, 2017 was $83.1 million, compared to a net loss of $56.0 million for the quarter ended June 30, 2016.

Cash, cash equivalents and marketable securities as of June 30, 2017 were $715.9 million, compared to $573.6 million as of December 31, 2016. The increase in cash was driven by net proceeds of $270.2 million from the April financing, $8.1 million of program funding received under our collaboration agreements with Celgene and $6.8 million received from employee stock award transactions. This was offset by expenditures to fund operations of $142.5 million during the six months ended June 30, 2017.

The company expects that its cash, cash equivalents and marketable securities as of June 30, 2017, together with anticipated interest income, and anticipated expense reimbursements under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2019.

Kite Submits Investigational New Drug (IND) Application for KITE-585, Anti-BCMA CAR-T Therapy Candidate for Multiple Myeloma

On August 8, 2017 Kite Pharma, Inc. (Nasdaq:KITE), a leading cell therapy company, reported the submission of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to initiate a Phase 1, first-in-human trial of KITE-585, a CAR-T cell therapy engineered to target B-cell maturation antigen (BCMA) in patients with relapsed/refractory multiple myeloma (Press release, Kite Pharma, AUG 8, 2017, View Source [SID1234520079]).

"KITE-585 has the potential to become Kite’s next significant advance in cell therapy for patients with cancer. It is the result of an extensive preclinical development effort that included candidate screening, engineering, and testing by Kite’s internal research team and it reflects the company’s deep experience in CAR design and cellular therapeutics," said David Chang, M.D., Ph.D., Executive Vice President of Research and Development and Chief Medical Officer of Kite. "As we look ahead, we are confident that the cutting-edge design and manufacturing process of KITE-585 together with our proven capability with engineered T cells will support rapid execution of the clinical program."

BCMA is expressed on the surface of malignant plasma cells in most patients with multiple myeloma. In addition, it is found on normal plasma cells and certain mature B-cell lineage cells but is absent from other tissues. Because BCMA has been shown to play a potential role in survival and growth of myeloma cells, it is viewed as an attractive CAR T-cell therapeutic target.

About KITE-585

KITE-585 is an anti-BCMA CAR construct designed for high binding affinity to BCMA expressed on the cell surface. KITE-585 contains a receptor derived from a fully human monoclonal antibody and a CD28 costimulatory domain intended for optimized T-cell expansion and function. In preclinical studies, KITE-585 demonstrated activity across a range of low and high BCMA expressing targets and its activity was not impaired by soluble BCMA. In the presence of cell-bound BCMA, KITE-585 induced polyfunctional T cell expansion, and no tonic signaling in its absence. Advanced processes and materials used in the manufacturing of KITE-585 are designed to achieve enhanced cell potency.

About Multiple Myeloma

Multiple myeloma is a cancer of plasma cells in the bone marrow, which make antibodies to fight infections. Abnormal plasma cells can grow out of control and suppress the growth of other cells in the bone marrow. This suppression may result in bone damage, anemia, excessive bleeding, and a decreased ability to fight infection. In 2017, there will be an estimated 30,280 new cases of multiple myeloma in the United States and 12,590 deaths due to the disease. Approximately half of patients survive five years after being diagnosed with multiple myeloma.1

BioLineRx Reports Second Quarter 2017 Financial Results

On August 8, 2017 BioLineRx Ltd. (NASDAQ/TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology and immunology, reported its financial results for the second quarter ended June 30, 2017 (Press release, BioLineRx, AUG 8, 2017, View Source [SID1234520076]).

Highlights and achievements during the second quarter 2017 and to date:

Continued execution on multiple clinical development programs for the Company’s lead project, BL-8040:

Announced plans to initiate Phase 3 pivotal study with BL-8040 as novel stem cell mobilization treatment for autologous bone-marrow transplantation in H2 2017, following successful meeting with the FDA;
Reported regulatory submissions of three Phase 1b/2 trials for BL-8040 in combination with atezolizumab, Genentech’s anti-PDL1 cancer immunotherapy agent, in pancreatic, gastric and non-small cell lung cancers, under immunotherapy collaboration with Genentech, a member of the Roche Group. All three studies will be conducted as part of MORPHEUS, Roche’s Novel Cancer Immunotherapy Development Platform, and are expected to commence in H2 2017;
Reported filing of regulatory submissions to commence Phase 1b/2 trial for BL-8040 in combination with Genentech’s atezolizumab in acute myeloid leukemia (AML), to be led by BioLineRx. This study is expected to commence in H2 2017;
Announced initiation of first Phase 1b/2 trial under immunotherapy collaboration with Genentech – in pancreatic cancer;

The Company also announced an additional, direct investment by BVF Partners, L.P., its largest shareholder, increasing its economic interest in the Company to 24.9%. The $9.6 million investment was priced at $1.13 per unit, each unit consisting of 1 ordinary share, 0.35 of Series A warrant with an exercise price of $2.00 per share, and 0.35 of Series B warrant with an exercise price of $4.00 per share. The warrants are exercisable for a period of 4 years.

Expected significant upcoming milestones for 2017 and 2018:

Partial results from immuno-oncology Phase 2a study in pancreatic cancer for BL-8040 in combination with Merck’s KEYTRUDA expected in H2 2017; top line results expected in H2 2018;
Initiation of Phase 3 pivotal study for BL-8040 in stem-cell mobilization for autologous transplantation planned for H2 2017;
Initiation of Phase 1b/2 immuno-oncology studies for BL-8040 in combination with Genentech’s atezolizumab in gastric cancer and non-small cell lung cancer, as well as AML, all expected in H2 2017; partial results expected in H2 2018;
Initiation of Phase 1 immuno-oncology study for AGI-134 in several solid tumor indications expected in H1 2018;
Top-line results of Phase 2 study for BL-8040 in stem-cell mobilization for allogeneic transplantation expected by mid-2018.

Philip A. Serlin, Chief Executive Officer of BioLineRx, remarked, “We are pleased to report second quarter-to-date activities that reinforce our focus to deliver on our objectives. This included timely initiation of our cancer immunotherapy collaboration with Genentech for pancreatic cancer, as well as regulatory advancements for additional indications in other solid tumors, AML, and stem cell mobilization. Thus, by the end of the year, we remain poised to have one Phase 3 and seven Phase 2 or 1b/2 clinical trials up and running, in addition to announcing partial results in our Phase 2 study in pancreatic cancer under our immunotherapy collaboration with Merck.”

“We are also pleased to have received a strong vote of confidence from BVF Partners last month. The $9.6 million direct investment we received will allow us to accelerate the development of our clinical programs. With over $60 million in cash on a pro forma basis, including BVF’s investment, as of June 30th, we have a strong balance sheet which will enable us to fully execute on our current operating plans,” Mr. Serlin concluded.

Financial Results for the Second Quarter Ended June 30, 2017

Research and development expenses for the three months ended June 30, 2017 were $4.0 million, an increase of $1.3 million, or 48.2%, compared to $2.7 million for the three months ended June 30, 2016. The increase resulted primarily from spending on AGI-134 and BL-8040 in the 2017 period. Research and development expenses for the six months ended June 30, 2017 were $7.7 million, an increase of $2.4 million, or 45.0%, compared to $5.3 million for the six months ended June 30, 2016. The reason for the increase is the same as that presented in the three-month comparison above.

Sales and marketing expenses for the three months ended June 30, 2017 were $0.3 million, similar to the comparable period in 2016. Sales and marketing expenses for the six months ended June 30, 2017 were $1.0 million, an increase of $0.5 million, or 86.3%, compared to $0.5 million for the six months ended June 30, 2016. The increase resulted primarily from market research activities and one-time professional fees related to business development activities.

General and administrative expenses for the three months ended June 30, 2017 were $0.8 million, similar to the comparable period in 2016. General and administrative expenses for the six months ended June 30, 2017 were $1.8 million, similar to the comparable period in 2016.

The Company’s operating loss for the three months ended June 30, 2017 amounted to $5.2 million, compared with an operating loss of $3.9 million for the corresponding 2016 period. The Company’s operating loss for the six months ended June 30, 2017 amounted to $10.5 million, compared with an operating loss of $7.6 million for the corresponding 2016 period.

Non-operating income (expenses) for the three and six months ended June 30, 2017 and 2016 were not material, and primarily relate to fair-value adjustments of warrant liabilities on the balance sheet.

Net financial income amounted to $0.3 million for the three months ended June 30, 2017 compared to net financial income of $0.1 million for the three months ended June 30, 2016. Net financial income amounted to $0.8 million for the six months ended June 30, 2017 compared to net financial income of $0.2 million for the six months ended June 30, 2016. The increase in net financial income relates primarily to gains recorded on foreign currency hedging transactions and investment income earned on our bank deposits.

The Company’s net loss for the three months ended June 30, 2017 amounted to $4.9 million, compared with a net loss of $3.7 million for the corresponding 2016 period. The Company’s net loss for the six months ended June 30, 2017 amounted to $9.8 million, compared with a net loss of $7.2 million for the corresponding 2016 period.

The Company held $52.6 million in cash, cash equivalents and short-term bank deposits as of June 30, 2017. In July 2017, the Company completed a direct placement of its securities for net proceeds of $9.5 million.

Net cash used in operating activities was $8.0 million for the six months ended June 30, 2017, compared with net cash used in operating activities of $7.5 million for the six months ended June 30, 2016. The $0.5 million increase in net cash used in operating activities during the six-month period in 2017, compared to the six-month period in 2016, was primarily the result of an increase in the Company’s operating loss in the 2017 period.

Net cash used in investing activities for the six months ended June 30, 2017 was $16.0 million, compared to net cash provided by investing activities of $4.2 million for the six months ended June 30, 2016. The changes in cash flows from investing activities relate primarily to investments in, and maturities of, short-term bank deposits, as well as the investment in Agalimmune.

Net cash provided by financing activities for the six months ended June 30, 2017 was $28.3 million, compared to net cash provided by financing activities of $1.6 million for the six months ended June 30, 2016. The increase in cash flows from financing activities primarily reflects the public offering completed in April 2017.