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BIOGEN TO PRESENT AT THE BERNSTEIN 34TH ANNUAL STRATEGIC DECISIONS CONFERENCE

On May 22, 2018 Biogen Inc. (NASDAQ: BIIB) reported that it will present at the Bernstein 34th Annual Strategic Decisions Conference (Press release, Biogen, MAY 22, 2018, View Source [SID1234526855]). The webcast will be live on Wednesday, May 30, 2018 at 9:00 a.m. ET. To access the live webcast, please visit Biogen’s Investors section at www.biogen.com/investors. An archived version of the webcast will be available following the presentation.

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Genoscience Pharma extends its first clinical trial of GN561 in patients with advanced liver cancers to France

On May 22, 2018 Genoscience Pharma, a clinical-stage biotechnology company dedicated to discovering and developing anticancer drugs, reported the extension to France of its first-in-human Phase 1b/2a clinical study using the oral small molecule GNS561 in advanced liver cancers (cholangiocarcinoma and hepatocellular carcinoma) (Press release, GenoScience, MAY 22, 2018, View Source [SID1234526852]). GNS561 received an IND for a two-year international clinical trial involving up to 50 patients with liver cancer. It is small molecule, administered orally, with a new mechanism of action which works primarily by dysregulating zinc homeostasis in cancer cells and by inducing cancer cell death.

This international Phase 1/2a study, which began in April 2018 at the Jules Bordet Institute in Brussels, Belgium, will be performed in several European countries and the United States. It will evaluate the safety, activity, pharmacokinetic and pharmacodynamic properties of escalating doses of GNS561. Professor Ghassan Abu Alfa of Memorial Sloan Kettering in New York is co-principal investigator in the United States. Professor Ahmad Awada, head of the Department of Medical Oncology at the Jules Bordet Institute is the lead investigator in Belgium. The French study is led by Professor Philippe Merle, head of the Medical Oncology Department and principal investigator at Croix Rousse Hospital, Lyon, France.

Up to 36 patients will be enrolled in six cohorts during the escalation phase. Additional patients will be included in the continuous treatment phase to obtain a total of 20 evaluable subjects at the recommended dose.

"This approval from the French regulatory authorities (ANSM) for our clinical program represents a paradigm shift for our company. It expands our international clinical capacity and offers the opportunity to investigate GNS561’s potential for patients in France," said Professor Philippe Halfon, president and CEO of Genoscience Pharma.

"We are pleased that our clinical program was quickly approved by the regulatory authorities in Belgium and France and by the institutional review boards and ethics committees in both countries. The first cohort of patients having been initiated in Belgium, we are now excited to continue our international evaluation of GNS561 by opening new centers in France. The enrollment and treatment of patients with advanced cholangiocarcinoma represents a new milestone for Genoscience Pharma," said Professor Eric Raymond, chief medical officer.

"We are excited to be enrolling our first patient with GNS561. The mechanism of action of this novel anticancer agent offers great promise. We are hopeful that GNS561 will prove to be a significant and effective weapon against liver cancer, specifically for patients with cholangiocarcinoma for whom there are limited options beyond gemcitabine-platinum based therapy," said Professor Philippe Merle, principal investigator.

About Cholangiocarcinoma
Cholangiocarcinoma (CCAs) is a heterogeneous group of bile duct cancers that arise from cholangiocytes that line the biliary tree. CCAs are classified based on their anatomic location, as follows: (1) intrahepatic CCA (iCCA), (2) perihilar CCA (pCCA) or (3) distal CCA (dCCA). iCCA represents 15% of CCA and is the second most common primary hepatic cancer, after hepatocellular carcinoma, with about 12,000 new cases every year in the US and in five combined European countries (France, UK, Italy, Germany, Spain, Belgium). In past decades, differing from overall cancer trends, iCCA incidence has more than doubled worldwide and its mortality rates have been escalating with a 39% increase. It is predicted to keep dramatically progressing due to a sedentary lifestyle, exposure to chemicals and an ageing population. Due to the late appearance of symptoms and late diagnosis, the tumour is unresectable in 60 to 70% of cases; patients are only eligible for chemotherapy.
Systemic chemotherapy options are critically limited: the standard practice is the first-line use of platinum and gemcitabine combinations, which leads to on average only a moderate overall survival benefit of 11.7 months. To date, despite numerous studies, no second-line treatment is recommended in clinical guidelines as no benefit from the further use of chemotherapy, targeted therapy and immunotherapy was observed.

About GNS561
GNS561 is a novel Solute Carrier Transporter (SLCT) inhibitor demonstrating potent antitumor activity against a range of human cancer cell lines, including HCC. It also shows activity in cell lines resistant to current standard-of-care treatment options for HCC. GNS561 is an orally bioavailable compound initially being developed for the treatment of
primary liver cancer, including advanced HCC. It is also being investigated preclinically in other solid tumors.

Approval of UCART123 Amendment in AML to Accelerate Clinical Development

On May 22, 2018 Cellectis (Euronext Growth: ALCLS – Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported the approval of an amendment to the protocol for the Phase 1 clinical trial of Cellectis’ UCART123 product candidate in patients with acute myeloid leukemia (AML) (Press release, Cellectis, MAY 22, 2018, file:///C:/Users/LENOVO/Downloads/20180522_PR_UCART123_Protocol_Amendment_addendum%20(1).pdf [SID1234526851]).

The main changes to the protocol include:

Dose level 1 to be administered increases from 6.25×104 to 2.5×105 UCART123 cells per kilogram. Dose levels 2 and 3 are now respectively at 6.25×105 and 5.05×106. Dose level -1 is now at 1.25×105. The product’s safety and tolerability profile allowed Cellectis to increase dose levels with a capping at 80kg equivalent.
The dose limiting toxicities (DLT) observation period decreases from 42 to 28 days post-UCART123 infusion, except for patients with aplastic bone marrow at Day 28 for whom the DLT observation period remains 42 days.
The time interval between the first and the second patient for UCART123 infusion at each new dose level tested shortens from 42 days to 28 days (42 days in case of aplastic anemia) then to 14 days for subsequent patients.
A potential second UCART123 infusion is implemented.
In addition, a new AML clinical center has been opened at MD Anderson Cancer Center in Houston, Texas, aiming at increasing the patient enrollment pace. The study is led by Prof. Hagop Kantarjian, MD, Department Chair, Department of Leukemia, Division of Cancer Medicine, and Dr. Naveen Pemmaraju, MD, Assistant Professor, being Principal Investigator.

"This amendment approval for Cellectis’ UCART123 protocol is an important step in the progression of our study, and opening another clinical site at MD Anderson – one of the world’s most premier cancer centers – puts the Company on solid ground to help as many AML patients as possible with this innovative new therapy," said Prof. Stéphane Depil, Senior Vice President, R&D, and Chief Medical Officer at Cellectis. "Off-the-shelf gene editing immunotherapy is continuing to revolutionize the landscape of modern medicine, and we hope that this approach leads to a lifesaving treatment for AML patients in the near future."

"As Cellectis has been working very closely with the concerned parties to review the details of UCART123 study to date, we are eager to hit the ground running with the new protocol in an effort to find a truly effective treatment for AML patients with high unmet medical needs," added Stéphan Reynier, Chief Regulatory and Compliance Officer at Cellectis. "We look forward to obtaining additional data so that we can address such a rare and devastating disease."

The FDA review period for this protocol amendment has passed and Cellectis obtained IRB’s approval.

More information about this trial is available at ClinicalTrials.gov.

About UCART123 clinical trial

Our first wholly controlled product candidate, UCART123, is a gene edited T-cell investigational drug that targets CD123, an antigen expressed at the surface of leukemic cells in AML. Cellectis received in February 2017 an Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA) to conduct Phase 1 clinical trial with UCART123 in patients with AML. This marks the first allogeneic, "off-the-shelf" gene-edited CAR T-cell product candidate that the FDA has approved for clinical trial.

UCART123 clinical trial in AML is a Phase 1, open label dose-escalation and dose-expansion study to evaluate the safety, expansion, persistence and clinical activity of UCART123 (allogeneic engineered T-cells expressing anti-CD123 chimeric antigen receptor), administered in patients with relapsed/refractory AML, and patients with newly diagnosed high-risk AML.

The clinical research is coordinated by principal investigator Prof. Gail J. Roboz, MD, at Weill Cornell, Professor of Medicine at Weill Cornell Medicine and Director of the Clinical and Translational Leukemia Programs at Weill Cornell Medicine and NewYork-Presbyterian.

AML is a devastating clonal hematopoietic stem cell neoplasm that is characterized by uncontrolled proliferation and accumulation of leukemic blasts in bone marrow, peripheral blood and, occasionally, in other tissues. These cells disrupt normal hematopoiesis and rapidly cause bone marrow failure and death. In the U.S. alone, there are in 2017 an estimated 21,000 new AML cases per year, with 10,000 estimated deaths per year

About UCART123 clinical trial
Our first wholly controlled product candidate, UCART123, is a gene edited T-cell investigational drug that targets CD123, an antigen expressed at the surface of leukemic cells in AML. Cellectis received in February 2017 an Investigational New Drug (IND) approval from the U.S. Food and Drug Administration (FDA) to conduct Phase 1 clinical trial with UCART123 in patients with AML. This marks the first allogeneic, "off-the-shelf"
gene-edited CAR T-cell product candidate that the FDA has approved for clinical trial.

UCART123 clinical trial in AML is a Phase 1, open label dose-escalation and doseexpansion study to evaluate the safety, expansion, persistence and clinical activity of UCART123 (allogeneic engineered T-cells expressing anti-CD123 chimeric antigen receptor), administered in patients with relapsed/refractory AML, and patients with newly
diagnosed high-risk AML.

AML is a devastating clonal hematopoietic stem cell neoplasm that is characterized by uncontrolled proliferation and accumulation of leukemic blasts in bone marrow, peripheral blood and, occasionally, in other tissues. These cells disrupt normal hematopoiesis and rapidly cause bone marrow failure and death. In the U.S. alone, there
are in 2017 an estimated 21,000 new AML cases per year, with 10,000 estimated deaths per year.

BioLineRx Reports First Quarter 2018 Financial Results

On May 22, 2018 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology and immunology, reported its financial results for the first quarter ended March 31, 2018 (Press release, BioLineRx, MAY 22, 2018, View Source;p=RssLanding&cat=news&id=2350176 [SID1234526848]).

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

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

Partial monotherapy results from Phase 2a COMBAT study, investigating the combination of BL-8040 and Merck’s PD-1 inhibitor, Keytruda (pembrolizumab), in pancreatic cancer, showed significantly increased infiltration of T cells into liver metastases in almost half of the pancreatic cancer patients who underwent a biopsy, as well as an increase in the number of total immune cells in the peripheral blood, alongside a decrease in the frequency of peripheral blood regulatory T cells (Tregs) – all of which support the mechanism of action proposed by pre-clinical studies. Study enrollment has been completed, with top-line results expected in H2 2018;
Results from Phase 2 study for BL-8040 as novel stem cell mobilization treatment for allogeneic bone-marrow transplantation support BL-8040 as a one-day dosing regimen for rapid mobilization of stem cells; primary endpoint of collection of ≥2 million CD34 cells/kg recipient weight after up to 2 leukapheresis (LP) sessions was reached in over 90% of patients (100% of patients at optimal BL-8040 dose of 1.25 mg/kg); all 19 transplanted recipients were successfully engrafted with BL-8040-mobilized grafts, and preliminary graft-versus-host disease (GVHD) data are in line with current standard-of-care incidence rates;
Overall long-term survival results in Phase 2a trial in relapsed/refractory AML demonstrated that the combination of BL-8040 with high-dose Ara-C (HiDAC) significantly improved overall survival, compared with historical data of HiDAC monotherapy. In the BL-8040 dose selected for expansion (1.5 mg/kg), the overall response rate was 39% (N=23) and median overall survival for this cohort was 9.2 months with 1-year and 2-year survival rates of 31.6% and 21.1%, respectively;
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, thus providing significant additional patent protection in AML, one of BL-8040’s key indications.
The Company also announced advancements made in its second immuno-oncology compound, AGI-134:

Pre-clinical data presented at ASCO (Free ASCO Whitepaper)-SITC showed direct regression of established primary tumors after injection with AGI-134 in the majority of mice treated, and that this regression is associated with activation of the innate immune system;
Notice of Allowance issued by the United States Patent and Trademark Office (USPTO) for a patent application claiming the use of AGI-134 for the treatment of solid cancer tumors; this patent, when issued, will be valid until May 2035 with a possibility of up to five years patent term extension. Additional corresponding patent applications for AGI-134 are pending in Europe, Japan, China, Canada, Australia and Israel.
Expected significant upcoming milestones for 2018:

Results from the lead-in part of the Phase 3 GENESIS study in stem-cell mobilization for autologous transplantation are due mid-year 2018;
Top-line results in immuno-oncology Phase 2a COMBAT study in pancreatic cancer for BL-8040 in combination with KEYTRUDA, under collaboration with Merck, expected in H2 2018;
Initiation of Phase 1/2a immuno-oncology study for AGI-134 in several solid tumor indications expected in mid-2018;
Additional overall long-term survival data from Phase 2a trial in relapsed/refractory AML to be presented at EHA (Free EHA Whitepaper) in June 2018;

Full top-line results of Phase 2 study for BL-8040 in stem-cell mobilization for allogeneic transplantation to be presented at the 23rd Congress of European Hematology Association (EHA) (Free EHA Whitepaper) in June 2018.

Philip A. Serlin, Chief Executive Officer of BioLineRx, stated, "We continue to strongly focus on clinical execution of our oncology programs. Since the beginning of 2018, we have made significant progress with BL-8040, our lead clinical asset, with clinical results from our Phase 2a COMBAT study in pancreatic cancer showing robust mobilization and increased infiltration of anti-tumor-specific T cells into the tumor microenvironment; positive results from our Phase 2 study in allogeneic bone marrow transplantation; very encouraging overall survival data from our proof-of-concept Phase 2a study in relapsed/refractory AML; as well as significant strengthening of our patent protection for BL-8040 in the AML space. In addition, we also reported very encouraging pre-clinical data on our near-clinical second oncology asset, AGI-134, demonstrating induced regression of primary tumors following intra-tumoral injection.

"Over the next three to nine months, we look forward to reporting on key milestones. This includes the results from the lead-in part of our Phase 3 GENESIS trial in autologous stem cell mobilization, data read-outs from our Phase 2a COMBAT study in pancreatic cancer, and initiation of a Phase 1/2a study in multiple solid tumor indications for AGI-134," concluded Mr. Serlin.

Financial Results for the First Quarter Ended March 31, 2018

Research and development expenses for the three months ended March 31, 2018 were $5.1 million, an increase of $1.5 million, or 41.2%, compared to $3.6 million for the three months ended March 31, 2017. The increase resulted primarily from higher expenses associated with new BL-8040 clinical studies commenced during 2017, spending on our new AGI-134 near-clinical project, and higher expenses related to our BL-1230 project.

Sales and marketing expenses for the three months ended March 31, 2018 were $0.5 million, a decrease of $0.2 million, or 28.9%, compared to $0.7 million for the three months ended March 31, 2017. The decrease resulted primarily from one-time legal fees related to AGI-134 incurred in the 2017 period.

General and administrative expenses for the three months ended March 31, 2018 were $1.1 million, similar to the comparable period in 2017.

The Company’s operating loss for the quarter ended March 31, 2018 amounted to $6.6 million, compared with an operating loss of $5.3 million for the quarter ended March 31, 2017.

Non-operating income (expenses) for both periods primarily relate to fair-value adjustments of warrant liabilities. These fair-value adjustments were highly influenced by the Company’s share price at each period end (revaluation date).

The Company recorded an immaterial amount of net ﬁnancial expenses for the three months ended March 31, 2018 compared to net financial income of $0.5 million for the three months ended March 31, 2017. Net financial expenses for the 2018 period primarily relate 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 March 31, 2018 amounted to $6.2 million, compared with a net loss of $4.9 million for the corresponding period.

The Company held $44.2 million in cash, cash equivalents and short-term bank deposits as of March 31, 2018.

Net cash used in operating activities was $6.8 million for the three months ended March 31, 2018, compared with net cash used in operating activities of $3.8 million for the three months ended March 31, 2017. The $3.0 million increase in net cash used in operating activities during the three-month period in 2018, compared to the three-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.

Net cash provided by investing activities was $8.1 million for the three months ended March 31, 2018, compared to net cash provided by investing activities of $1.4 million for the three months ended March 31, 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 period.

Net cash provided by financing activities was $1.4 million for the three months ended March 31, 2018, compared to net cash provided by financing activities of $2.1 million for the three months ended March 31, 2017. The cash flows from financing activities result primarily from funding under an ATM facility in the 2018 period and a share purchase agreement with Lincoln Park Capital in the 2017 period.

Conference Call and Webcast Information

BioLineRx will hold a conference call today, May 22, 2018 at 10:00 a.m. EDT. To access the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0685 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 May 25, 2018; please dial +1-877-456-0009 from the U.S. or +972-3-925-5942 internationally.

Alpine Immune Sciences to Present at Jefferies 2018 Global Healthcare Conference

On May 22, 2018 Alpine Immune Sciences, Inc. (NASDAQ:ALPN), a company focused on discovering and developing innovative, protein-based immunotherapies targeting the immune synapse to treat cancer, autoimmune/inflammatory, and other diseases, reported the company will present at the Jefferies 2018 Global Healthcare Conference on Tuesday, June 5 2018 at 4:30 pm Eastern Time in New York, NY (Press release, Alpine Immune Sciences, MAY 22, 2018, View Source [SID1234526847]).

A live webcast of the presentation will be available online in the investor relations section of the company’s website at View Source A replay of the presentation will be available on the company website for 90 days following the webcast.