On August 13, 2018 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology and immunology, reported its financial results for the second quarter ended June 30, 2018 (Press release, BioLineRx, AUG 13, 2018, View Source [SID1234528824]).

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Highlights and achievements during the second quarter 2018 and to date:

Continued progress made on multiple clinical trials for the Company’s lead oncology program, BL-8040:

Positive data from successful completion of first lead-in patient cohort of the GENESIS trial, a double-blind, placebo-controlled, Phase 3 trial comparing BL-8040 in combination with granulocyte colony-stimulating factor (G-CSF), to G-CSF alone, for the mobilization of hematopoietic stem cells (HSCs) used for autologous transplantation in multiple myeloma patients. Results from first 11 patients prompted Data Monitoring Committee (DMC) to recommend early continuation to randomized placebo-controlled part 2 of trial; data show that 9/11 patients (82%) reached primary endpoint threshold of ≥ 6×106 CD34 cells/kg with only one dose of BL-8040 and in up to 2 apheresis sessions;
Expansion of immuno-oncology collaboration with Merck & Co., Inc., Kenilworth, N.J. (Merck), supporting a Phase 2a program investigating BL-8040 in combination with KEYTRUDA in pancreatic cancer patients. Under the expansion, a triple combination arm investigating the safety, tolerability and efficacy of BL-8040, KEYTRUDA and chemotherapy will be added to the ongoing COMBAT/KEYNOTE-202 study, with a specific focus on second line patients;
Presentation at the 2018 European Hematology Association (EHA) (Free EHA Whitepaper) Conference of very encouraging long-term overall survival results in Phase 2a trial in relapsed/refractory AML, demonstrating that the combination of BL-8040 with high-dose Cytarabine (HiDAC) significantly improved overall survival, compared with historical data for HiDAC monotherapy;
Grant of European patent covering use of BL-8040 with Cytarabine for treating AML; valid through March 2034 with up to five years’ patent term extension.
The Company also announced advancements made in its second immuno-oncology compound, AGI-134:

Initiation of multicenter, open-label Phase 1/2a study in the UK and Israel, with possible expansion to the US and additional countries in Europe in 2019; study will evaluate the safety and tolerability of AGI-134, as a monotherapy and in combination with an immune checkpoint inhibitor, in unresectable metastatic solid tumors.
Expected significant milestones in next 18 months:

Top-line results in immuno-oncology Phase 2a COMBAT study in pancreatic cancer for BL-8040 in combination with KEYTRUDA, under collaboration with Merck, to be presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in October 2018;
Partial results in Phase 1b/2 study in pancreatic cancer under Genentech immuno-oncology collaboration, investigating BL-8040 in combination with Genentech’s atezolizumab, expected in H2 2018;
Potential interim analysis of Phase 2b BLAST study in AML consolidation in mid-2019:
Results from additional cohort in Phase 2a COMBAT study under expansion of Merck collaboration, investigating triple combination of BL-8040, KEYTRUDA and chemotherapy in pancreatic cancer, by end of 2019;
"We are very encouraged by the clinical results achieved to-date in the major indications being developed under our BL-8040 platform, as we continue to advance the asset towards registration," stated Philip A. Serlin, Chief Executive Officer of BioLineRx. "Specifically, positive results from the initial lead-in cohort in the Phase 3 GENESIS trial, and the resulting DMC recommendation for an early continuation to the randomized, double-blind, placebo-controlled part of the trial, is an important clinical achievement for BL-8040. The data continue to demonstrate BL-8040’s robust efficacy in stem-cell mobilization for autologous transplantation, including the potential to reduce the number of required apheresis sessions to one session in the majority of patients, versus multiple sessions under current practice. Building on this success, data from our ongoing COMBAT Phase 2a immuno-oncology trial support continued development in pancreatic cancer, as we expand our collaboration with Merck, with the addition to the existing study of a new cohort with 30-50 patients investigating the triple combination of BL-8040, KEYTRUDA and chemotherapy. The new cohort will focus on second-line pancreatic cancer patients and we are hopeful for significant synergies from the triple drug combination in this very difficult-to-treat population."

Mr. Serlin added, "In addition, we are excited about the data we continue to accumulate from our Phase 2a study in relapsed/refractory AML, with further significant improvement in overall survival data recently presented at EHA (Free EHA Whitepaper). We are focused on determining the appropriate next clinical development steps for this indication, in light of this very encouraging data. With regarding to our second main asset, AGI-134, we were pleased to initiate a Phase 1/2a study in multiple solid tumors. AGI-134 represents a new mechanistic class of cancer immunotherapies with a unique and highly differentiated mode of action, and we are pleased to begin our clinical evaluation of its potential."

"Over the next few quarters, we look forward to reporting on key milestones. These include top line results in our Phase 2a COMBAT study, partial results in the Phase 1b/2 pancreatic cancer trial under our collaboration with Genentech, and a potential interim analysis on the Phase 2b BLAST study in AML consolidation therapy," concluded Mr. Serlin.

Financial Results for the Second Quarter Ended June 30, 2018

Research and development expenses for the three months ended June 30, 2018 were $4.5 million, an increase of $0.4 million, or 10.4%, compared to $4.1 million for the three months ended June 30, 2017. The increase resulted primarily from higher expenses associated with AGI-134, including final preparations for initiation of the Phase 1/2a study, and expenses associated with BL-1230. Research and development expenses for the six months ended June 30, 2018 were $9.6 million, an increase of $1.9 million, or 24.9%, compared to $7.7 million for the six months ended June 30, 2017. The increase resulted primarily from higher expenses associated with new BL-8040 clinical studies commenced during 2017, as well as higher expenses associated with AGI-134, including final preparations for initiation of the Phase 1/2a study, and expenses associated with BL-1230.

Sales and marketing expenses for the three months ended June 30, 2018 were $0.4 million, an increase of $0.1 million, or 25%, compared to $0.3 million for the three months ended June 30, 2017. The increase resulted primarily from one-time consulting fees related to market research in the 2018 period. Sales and marketing expenses for the six months ended June 30, 2018 were $0.9 million, a decrease of $0.1 million, or 12.9%, compared to $1.0 million for the six months ended June 30, 2017. The decrease resulted primarily from one-time legal fees related to AGI-134 paid in the 2017 period.

General and administrative expenses for the three months ended June 30, 2018 were $0.9 million, similar to the comparable period in 2017. General and administrative expenses for the six months ended June 30, 2018 were $1.9 million, similar to the comparable period in 2017.

The Company’s operating loss for the three months ended June 30, 2018 amounted to $5.7 million, compared with an operating loss of $5.2 million for the corresponding 2017 period. The Company’s operating loss for the six months ended June 30, 2018 amounted to $12.4 million, compared with an operating loss of $10.5 million for the corresponding 2017 period.

Non-operating income (expenses) for the three and six months ended June 30, 2018 primarily relate to fair-value adjustments of warrant liabilities on the Company’s balance sheet and the capital gain from realization of the investment in iPharma. Non-operating income (expenses) for the three and six months ended June 30, 2017 primarily relate to fair-value adjustments of warrant liabilities on the Company’s balance sheet.

Net financial income amounted to $0.3 million for the three months ended June 30, 2018, similar to the comparable period in 2017. Net financial income for both periods relates primarily to gains recorded on foreign currency hedging transactions and investment income earned on bank deposits. Net financial income amounted to $0.3 million for the six months ended June 30, 2018, compared to net financial income of $0.8 million for the six months ended June 30, 2017. Net financial income for the 2018 period primarily relates to investment income earned on bank deposits, offset by losses recorded on foreign currency hedging transactions. Net financial income for the 2017 period relates primarily to gains recorded on foreign currency hedging transactions and investment income earned on bank deposits.

The Company’s net loss for the three months ended June 30, 2018 amounted to $4.8 million, compared with a net loss of $4.9 million for the corresponding period. The Company’s net loss for the six months ended June 30, 2018 amounted to $11.0 million, compared with a net loss of $9.8 million for the corresponding 2017 period.

The Company held $41.1 million in cash, cash equivalents and short-term bank deposits as of June 30, 2018.

Net cash used in operating activities was $13.0 million for the six months ended June 30, 2018, compared with net cash used in operating activities of $8.0 million for the six months ended June 30, 2017. The $5.0 million increase in net cash used in operating activities during the six-month period in 2018, compared to the six-month period in 2017, was the result of increased research and development expenses in the 2018 period, as well as a decrease in accounts payable and accruals.

Net cash provided by investing activities was $10.8 million for the six months ended June 30, 2018, compared to net cash used in investing activities of $16.0 million for the six months ended June 30, 2017. 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 in 2017 and realization of the investment in iPharma in 2018.

Net cash provided by financing activities was $2.8 million for the six months ended June 30, 2018, compared to net cash provided by financing activities of $28.3 million for the six months ended June 30, 2017. The decrease in cash flows from financing activities reflects the public offering completed in April 2017.

Conference Call and Webcast Information

BioLineRx will hold a conference call today, August 13, 2018 at 10:00 a.m. EDT. To access the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0664 internationally. The call will also be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until August 16, 2018; please dial +1-877-456-0009 from the U.S. or +972-3-925-5937 internationally.

On August 13, 2018 Cellectar Biosciences (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to CLR 131, the company’s lead Phospholipid Drug Conjugate (PDC) product candidate, for the treatment of Ewing’s sarcoma, a rare pediatric cancer (Press release, Cellectar Biosciences, AUG 13, 2018, View Source [SID1234528817]).

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"We are delighted to announce receipt of our third RPDD from the FDA, which underscores Cellectar’s commitment to rare pediatric cancers. There is a critical need to develop new therapies to fight deadly childhood cancers such as Ewing’s sarcoma, and CLR 131 has shown early promise in this arena," said John Friend, M.D., chief medical officer of Cellectar Biosciences. "This designation, combined with our receipt of FDA Orphan Drug Designation for Ewing’s sarcoma last month, will help support our efforts to optimize the drug development path in this indication and, if successful, enable this new therapeutic candidate is made available to patients as rapidly as possible."

Since March 2018 the FDA has granted RPDDs to CLR 131 for the treatment of three separate rare disease indications including neuroblastoma, rhabdomyosarcoma and now Ewing’s sarcoma. Should CLR 131 be approved by the FDA in any of these indications, the RPDD may enable Cellectar to receive a priority review voucher. Priority review vouchers can be used by the sponsor to receive priority review designation for a future NDA or BLA submission, which could reduce the FDA review time from twelve months to eight months. Currently, these vouchers can also be transferred or sold to another entity. Since the beginning of 2017, six priority review vouchers were sold for between $80 million and $150 million each.

The FDA grants RPDD for diseases that primarily affect children from birth to age 18, and affect fewer than 200,000 persons in the U.S. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

Cellectar plans to evaluate CLR 131 in a Phase 1 clinical study for the treatment of pediatric patients with Ewing’s sarcoma, rhabdomyosarcoma, osteosarcoma, neuroblastoma, high-grade glioma and lymphomas. Cellectar has received clearance from the FDA for an accelerated Phase 1 trial designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of CLR 131 in pediatric patients with these cancer types. Further details about the trial can be found at clinicaltrials.gov using the identifier number NCT03478462.

About Ewing’s Sarcoma

Ewing’s sarcoma is the second most common bone malignancy among children and adolescents. According to a study published in the Journal of Hematology/Oncology, the incidence is about 3 cases per 1 million per year in children younger than age 20. Despite the favorable prognosis, an American Cancer Society study showed that approximately 30-40% of patients develop metastases or local recurrence, and the long-term survival rate for refractory or recurrent disease is only 22-24%. The relapsed and refractory statistics underscore the need for new treatment options.

About CLR 131

CLR 131 is Cellectar’s investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. CLR 131, is in a Phase 2 clinical study in relapsed or refractory (R/R) MM and a range of B-cell malignancies and a Phase 1 clinical study in patients with (R/R) MM exploring fractionated dosing. The company is currently initiating a Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma and is planning a second Phase 1 study in combination with external beam radiation for head and neck cancer.

On August 10, 2018 Artios Pharma Limited (Artios), a leading DNA Damage Response (DDR) company developing innovative treatments for cancer, reported the completion of a $84 million (£65 million) Series B financing following high interest from investors (Press release, Artios Pharma, AUG 10, 2018, View Source [SID1234529507]).

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The financing, which was significantly oversubscribed, was led by Andera Partners (formerly EdRIP) and LSP (Life Sciences Partners), with participation by additional new investors Pfizer Ventures and Novartis Venture Fund (NVF). Artios’ existing shareholders Arix Bioscience, SV Health Investors, M Ventures, IP Group plc and AbbVie Ventures also participated in the fundraise.

Commenting on today’s announcement, Raphael Wisniewski, Partner of Andera Partners, said: "We believe Artios’ DDR programmes have the potential to bring real impact to cancer patients. DDR is an exciting field of biology, which has been clinically validated by the first generation PARP inhibitors currently on the market. The new funds will allow Artios to advance its portfolio of first-in-class, small molecule DDR programmes including its lead programme targeting DNA polymerase theta (Polθ), through clinical proof of concept trials. We are delighted to work with the management team in building a world class DDR targeted oncology company."

Dr Rene Kuijten, Managing Partner of LSP, commented: "Artios represents a unique opportunity to deliver a truly world class biotech company. LSP has worked with Artios’ team before at KuDOS (successfully sold to AstraZeneca) which developed olaparib, the first approved PARP inhibitor and used in ovarian and breast cancer, creating a billion dollar market. We are very excited to support this proven management team once again as it seeks to build on its leading position in the DDR field by advancing its programmes into clinical development."

Niall Martin, Chief Executive Officer of Artios, added: "We are delighted to welcome Andera Partners, LSP, Pfizer Ventures and Novartis Venture Fund to Artios and I would like to thank our existing investors for their continued support, which will help us develop and deliver our exciting DDR targeted therapies to cancer patients. This investor syndicate creates a very strong and committed shareholder base with a track record of supporting successful next generation companies. The oversubscribed Series B fundraise is a strong endorsement of our world-leading development pipeline and reflects the opportunity for DDR to yield new breakthrough oncology products."

Artios is actively developing a pipeline of highly promising first-in-class DDR therapies identified from a global network of leading researchers in the DDR field, including through Cancer Research UK. The inhibition of novel DNA repair targets like Polθ, in tumours where DNA damage response factors have been lost or down regulated, will lead to cancer cells being selectively killed without harming normal cells. This creates an opportunity for such products to be used both in monotherapy and in combination with existing and future cancer therapies.

In conjunction with the Series B financing, Raphael Wisniewski from Andera Partners, Rene Kuijten from LSP and Barbara Dalton from Pfizer Ventures, will join the Board of Artios as directors and Florian Muellershausen from NVF will join as an observer. This financing follows a $36 million Series A fundraise that was completed in September 2016.

On August 10, 2018 PharmaMar (MSE:PHM) has reported that it has been notified by ESMO (Free ESMO Whitepaper) (European Society for Medical Oncology) that its abstract titled "Phase III trial of Lurbinectedin versus PLD or Topotecan in platinum-resistant ovarian cancer patients: Results of CORAIL trial" has been accepted as an oral presentation which will take place October 19th at 1415 CET at the 2018 Congress in Munich, Germany

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On August 10, 2018 Sangamo Therapeutics, Inc. (NASDAQ: SGMO) reported that management will present a corporate overview at the 2018 Wedbush PacGrow Healthcare Conference in New York City (Press release, Sangamo Therapeutics, AUG 10, 2018, View Source [SID1234528840]). The presentation is scheduled for Tuesday, August 14th at 4:15 p.m. ET.

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The presentation will be webcast live and may be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. The presentation will be archived on the Sangamo website following the event.