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IMMUTEP ENTERS INTO CLINICAL TRIAL COLLABORATION AND SUPPLY AGREEMENT WITH MSD

On March 12, 2018 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or the "Company") has reported that it has entered into a clinical trial collaboration and supply agreement with Merck & Co., Inc., Kenilworth, NJ, USA (known as MSD outside the United States and Canada), through a subsidiary, to evaluate the combination of Immutep’s lead immunotherapy product candidate eftilagimod alpha ("efti" or "IMP321") with MSD’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) in a new clinical trial that will evaluate the combination in several different solid tumours (Press release, Immutep, MAR 12, 2018, View Source [SID1234524702]).

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The planned Phase II clinical trial, referred to as TACTI-002 (Two ACTive Immunotherapies), will evaluate the safety and efficacy of this novel immunotherapy combination in patients with non-small cell lung cancer ("NSCLC"), head and neck cancer, or ovarian cancer. The TACTI-002 clinical trial will be a Phase II, Simon two-stage, non-comparative, open-label, single-arm, multicentre clinical study. Up to 120 patients across the three indications are planned to be treated in medical centres in Europe and the United States with the trial expected to commence in the second half of 2018.

"We are extremely pleased to be collaborating with MSD, one of the world’s leading immuno-oncology companies" said Marc Voigt, CEO of Immutep. "This clinical trial will evaluate a novel combination of two complementary immuno-oncology treatments in three cancer indications simultaneously, which could lead to more rapid drug development subject to successful outcomes. The data generated thus far from our ongoing TACTI-mel clinical trial has supported our hypothesis that there is a compelling therapeutic synergy in administering efti in combination with another immuno-oncology treatment. This new Phase II clinical trial significantly builds on the momentum we are delivering in the evaluation of efti in cancer, with two Phase I clinical trials and now two Phase II clinical trials in our program for 2018."

The trial combines two immuno-oncology treatments with complementary mechanisms of action, analogous to releasing the brakes and pushing the accelerator of the body’s immune system at two different positions in the cancer immunity cycle. Immutep’s efti is a first-in-class antigen presenting cell ("APC") activator which stimulates cancer-fighting T cells, while KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells.

Cellectis Reports 4th Quarter and Full Year 2017 Financial Results

On March 12, 2018 Cellectis (Paris:ALCLS) (NASDAQ:CLLS) (Euronext Growth: ALCLS – Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported its results for the three-month period ended December 31, 2017 and for the year ended December 31, 2017 (Press release, Cellectis, MAR 12, 2018, View Source [SID1234524700]).

"I would like to highlight what remarkable progress we made in 2017, by transforming the off-the-shelf CAR T-cell concept into reality. I believe I can say without a doubt that we have only just scratched the surface of what a powerful treatment CAR T-cell therapy represents. 2018 will be a turning point for Cellectis, extending our lead in the allogeneic CAR T-cell field," said André Choulika, Chairman and Chief Executive Officer, Cellectis.

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1 Cash position includes cash, cash equivalents and current financial assets.

Earnings Call Details

Cellectis to hold a conference call for investors on Tuesday, March 13, 2018 at 8 a.m. EDT – 1 p.m. Paris Time. The call will include the company’s fourth quarter 2017 and year-end financial results.

The live dial-in information for the conference call is:

US & Canada only: 877-407-3104

International: 201-493-6792

In addition, a replay of the call will be available for 6 months following the conference by calling 877-660-6853 (Toll Free US & Canada); 201-612-7415 (Toll Free International).

The archived webcast of this event will be available archived for 6 months:

https://78449.themediaframe.com/dataconf/productusers/clls/mediaframe/23530/indexl.html

Cellectis – Therapeutics

UCART19: TALEN gene-edited, allogeneic CAR T-Cell product candidate in ALL patients, exclusively licensed to Servier

Intermediary results from the two Phase I clinical trials of UCART19 were presented by Servier at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Atlanta. UCART19 is an investigational allogeneic anti-CD19 CAR T-cell product candidate, used in adult and pediatric patients with relapsed or refractory (R/R) CD19-positive B-cell acute lymphoblastic leukemia (B-ALL). These first-in-human data demonstrated the safety and tolerability of UCART19, resulting in an 83% complete remission rate across the adult and pediatric patient populations at day 28 post CAR T-cell infusion. Our commercial partner Servier is currently expanding the UCART19 clinical studies in multiple centers in the U.S. and Europe and we are expecting further clinical updates by year-end 2018.

Additional results from the two Phase I clinical trials with UCART19 will be presented on March 21, 2018 during the European society for Blood and Marrow Transplantation (EBMT) Annual Meeting to be held in Lisbon, Portugal.

Successful GMP manufacturing of UCART22, representing already the third allogeneic, off-the-shelf, TALEN gene-edited CAR T-cell campaign after UCART19 and UCART123

UCART22 is currently in GMP manufacturing, expected to yield clinical supplies for the planned Ph1 study in ALL patients. Pending the completion of the manufacturing campaign, Cellectis plans to file an Investigational New Drug (IND) application in the first half of 2018. The UCART22 manufacturing campaign represents already the third consecutive manufacturing campaign of a TALEN gene-edited CAR T-cell campaign after UCART19 and UCART123, positioning Cellectis as a leader in the allogeneic, off-the-shelf CAR T-cell space.

UCART123: Cellectis’ TALEN gene-edited, allogeneic CAR T product candidate in AML and BPDCN Patients

In December 2017, patient enrollment has resumed in both Phase I clinical trials of UCART123 in acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN). On November 6, 2017, Cellectis announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold, previously announced on September 4, 2017, on both Phase I trials of UCART123. In connection with the lifting of the clinical hold, Cellectis agreed with the FDA to certain revisions to be implemented in Phase I UCART123 protocols.

Corporate

Cellectis announced on December 4, 2017 the appointments of Ms. Elsy Boglioli to the role of Executive Vice President, Strategy and Corporate Development, and Prof. Stéphane Depil, MD, PhD, to the role of Senior Vice President Research & Development and Chief Medical Officer. Ms. Boglioli’s responsibilities include directing the long-term strategy and current business priorities of Cellectis to ensure that the overall mission of the Company is fulfilled. Ms. Boglioli joins Cellectis from Boston Consulting Group (BCG), where she served as Partner and Managing Director, and leader of BCG’s biotech-focused business in Europe. Prof. Depil’s responsibilities include bringing Cellectis’ product candidates to clinical-stage development, strategic and operational management of all therapeutic activities, and supervising research and development projects for the Company. Prof. Depil continues his academic and research activities as adjunct Professor at Léon Bérard Cancer Center & University Claude Bernard in Lyon, France.

Scientific Publications

A poster has been presented at the Keystone Conference in February 2018, showcasing the high gene-editing efficiency of TALEN. A T-cell was edited using TALEN, to knock out the TCR alpha and beta chain, knock out the B2M molecule, knock in the CAR construct and knock in an NK cell inhibitor. This simultaneous double knock out and double knock in resulted at a 68.1% efficiency.

A study has been published in November 2017 in Molecular Therapy — Nucleic Acids describing the educated engineering of highly specific and efficient TAL nucleases (TALEN) targeting PD1, a key T-cell immune checkpoint.

Upcoming Investor Conferences

Cellectis will participate in Oppenheimer’s 28th Annual Healthcare Conference & Sachs BioCapital USA Forum both on March 21 in New York, and Guggenheim Conference on Disruptive Technologies in Immuno-Oncology on March 27, 2018 in New York.

Calyxt

On December 12, 2017 Calyxt signed a partnership with Farmer’s Business Network, Inc (FBNSM), the independent farmer-to-farmer network, to expand the distribution and grower base of Calyxt’s identity-preserved high oleic soybeans in the upper Midwest region, including South Dakota and Minnesota. This new partnership enables FBN DirectTM to distribute Calyxt’s identity-preserved high oleic soybean seeds to growers in its network.

Calyxt announced on March 1, 2018 having contracted over 10,000 acres with 50 farmers in the Midwest. Overall, these growers collectively farm over 100,000 acres, half of which are expected to produce soybeans. Twenty percent of the soybeans that are anticipated to be planted consist of Calyxt’s high-oleic variety.

Corporate

Initial Public Offering: On July 25, 2017, Calyxt completed an initial public offering of its common stock, selling an aggregate of 8,050,000 shares of common stock at a price of $8.00 per share (including 1,050,000 shares of common stock pursuant to the exercise by the underwriters of their option to purchase additional shares). Calyxt received net proceeds of approximately $58.0 million, after deducting underwriting discounts and commissions and offering expenses. As part of the IPO, Cellectis purchased 2,500,000 shares of common stock for a value of $20.0 million, which is included in the net proceeds that Calyxt received. Calyxt used $5.7 million of the proceeds to cover a portion of the outstanding obligations owed to Cellectis. As of February 28, 2018, Cellectis owns 79.3% of the outstanding Calyxt’s common shares.

Financial Results

Cellectis’ consolidated financial statements have been prepared in accordance with International Financial Reporting Standards, or IFRS, as issued by the International Accounting Standards Board ("GAAP").

Effective in the third quarter of 2017, Cellectis changed the presentation currency of its consolidated financial statements from the euro to the U.S. dollar, in order to enhance comparability with peers, which present their financial statements primarily in U.S. dollar.

Fourth quarter 2017 Financial Results

Cash: As of December 31, 2017, Cellectis had $297.0 million in total cash, cash equivalents and current financial assets compared to $304.1 million as of September 30, 2017. This decrease of $7.1 million reflects (i), the net cash flows used by operating activities of $9.5 million and (ii) net cash provided by investing activities of $0.5 million, partially offset by (iii) the unrealized positive translation effect of exchange rate fluctuations on U.S. dollar cash, cash equivalents and current financial assets of $2.0 million and (iv) net cash flows provided by financing activities of $0.9 due to the exercise of Cellectis warrants and stock options during the period.

Selecta Biosciences Announces First Patient Dosed in Phase 1 Trial of SVP-Rapamycin and LMB-100 Combination Therapy in Mesothelioma

On March 12, 2018 Selecta Biosciences, Inc. (Nasdaq:SELB), a clinical-stage biopharmaceutical company focused on unlocking the full potential of biologic therapies by avoiding unwanted immune responses, reported that the first patient has been dosed in a Phase 1 clinical trial of SEL-403, Selecta’s product candidate consisting of SVP-Rapamycin in combination with LMB-100 (Press release, Selecta Biosciences, MAR 12, 2018, View Source [SID1234524682]). The trial (ClinicalTrials.gov Identifier# NCT03436732 ), is enrolling patients with malignant pleural or peritoneal mesothelioma who have undergone at least one regimen of chemotherapy, and it is being conducted under a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), part of the National Institutes of Health.

SVP-Rapamycin is Selecta’s proprietary, clinical-stage anti-drug antibody (ADA) prevention and immune tolerance technology. LMB-100 is a recombinant immunotoxin that targets mesothelin, a protein expressed in nearly all mesotheliomas and pancreatic adenocarcinomas, and a high percentage of other malignancies, including lung, breast and ovarian cancers. A paper co-authored by Selecta and researchers at the NCI discussing preclinical work completed with this combination therapy candidate was recently published in Proceedings of the National Academies of Sciences (PNAS, 2018 Jan 23;115(4): E733-E742).

"Mesothelioma remains one of the deadliest and most challenging-to-treat forms of cancer," stated Raffit Hassan, M.D., Senior Investigator, Thoracic and GI Oncology Branch in NCI’s Center for Cancer Research and Principal investigator of the trial. "Recombinant immunotoxins hold the potential to induce marked anti-tumor activity if anti-drug antibodies are prevented and sufficient cycles of therapy can be administered. We are pleased to get this clinical investigation underway to determine if patients may indeed benefit from a combination therapy consisting of LMB-100 and SVP-Rapamycin."

Patients in this open-label dose-escalation trial will receive up to four treatment cycles, each treatment cycle consisting of an initial dose of the combination of SVP-Rapamycin and LMB-100 on day 1 followed by two doses of LMB-100 alone on days 3 and 5. The study, which is expected to enroll up to 18 patients, is designed to evaluate the safety and tolerability of this treatment and provide data on pharmacokinetics, anti-drug antibody (ADA) levels, as well as an objective response rate assessment.

For Patients

Patients interested in enrolling please contact NCI’s toll-free number 1-800-4-Cancer (1-800-422-6237) (TTY: 1-800-332-8615) and/or the Web site: View Source

About Mesothelioma
Mesothelioma is a mesothelin-expressing cancer predominantly affecting the layer of tissue lining the lungs and chest wall. This type of cancer has been linked to asbestos exposure. According to the American Cancer Society, approximately 3,000 people are diagnosed with this disease each year in the United States. The prognosis for mesothelioma is very poor, with an average life expectancy of 12-18 months following diagnosis.

Portola Pharmaceuticals Announces New Interim Results from Ongoing ANNEXA-4 Study of Factor Xa Inhibitor Reversal Agent AndexXa® (Andexanet Alfa) in Patients with Life-Threatening Bleeding

On March 12, 2018 Portola Pharmaceuticals, Inc. (Nasdaq:PTLA) reported new interim results from ANNEXA-4, the Company’s ongoing Phase 3b/4 trial of its investigational universal Factor Xa inhibitor antidote AndexXa (andexanet alfa) among patients experiencing acute major bleeding while taking a Factor Xa inhibitor (Press release, Portola Pharmaceuticals, MAR 12, 2018, View Source;p=RssLanding&cat=news&id=2337591 [SID1234524680]). Interim data from 228 patients (of which 132 were adjudicated for efficacy) showed that AndexXa rapidly and significantly reversed anti-Factor Xa activity (the anticoagulant mechanism of these drugs) when administered as a bolus, and sustained this reversal when followed by a 120-minute infusion. In addition, 83 percent of these patients achieved excellent or good hemostasis (stoppage of bleeding) over a 12-hour period following treatment with AndexXa. Thrombotic events (11 percent) and death rates (12 percent) were consistent with previous ANNEXA-4 trial results and with the high background thrombotic risk of the enrolled patient population. Results were presented today in a Late-Breaking Clinical Trial Session at the American College of Cardiology’s 67th Annual Scientific Session & Expo (ACC.18).

"These data are particularly compelling when you consider the high-risk profile of the ANNEXA-4 population, which includes a substantial number of elderly patients presenting with intracranial hemorrhage and anticoagulated for venous thromboembolism, and the lack of any FDA- or EMA-approved reversal agent for these patients," said Stuart J. Connolly, M.D., ANNEXA-4 Executive Committee chairman and professor in the Department of Medicine of the Faculty of Health Sciences at McMaster University in Hamilton, Ontario. "The interim efficacy and safety data continue to support the promising role of AndexXa as an antidote to reverse anticoagulation in Factor Xa-associated bleeding."

The use of Factor Xa inhibitors is continuing to grow at a significantly steady pace because of their demonstrated efficacy in preventing embolic diseases such as stroke and pulmonary embolism. However, the incidence of hospital admissions and death related to Factor Xa-inhibitor bleeding is also increasing. In the U.S. alone, there were approximately 117,000 hospital admissions attributable to Factor Xa-related bleeding in 2016 and more than 2,000 bleeding-related deaths per month.

"The ANNEXA-4 trial continues to demonstrate efficacy and safety results that are consistent with that of other therapies approved for anticoagulant reversal based on a single-arm study," said John Curnutte, M.D., Ph.D., executive vice president, research and development at Portola. "We remain confident in the potential of AndexXa to address a clear and growing unmet need and we look forward to sharing these results with the U.S. and European regulatory authorities as they consider our marketing application for andexanet alfa."

The interim results included safety data from 227 of the 228 enrolled patients who experienced intracranial hemorrhage (ICH) (61 percent), gastrointestinal bleeding (27 percent) or bleeding from another site (11 percent) within 18 hours of administration of apixaban (117 patients), rivaroxaban (90 patients), enoxaparin (17 patients) or edoxaban (3 patients). Safety data for the one remaining patient, who was enrolled and active in the study, was not available at the time of this analysis.

During the 30-day follow-up period, the thrombotic event rate was 11 percent (n=24) for the entire population and 12 percent (n=17) among patients experiencing an ICH. The mortality rate for all patients was 12 percent (n=27). The rate of these events occurred within the range expected in this population given the severity of the bleeding, their advanced age and underlying thrombotic risk, and the percentage who restarted anticoagulant therapy (57 percent) following their bleeding episode.

Two of the 228 patients experienced an infusion reaction and none developed antibodies to Factor Xa or Factor X or neutralizing antibodies to AndexXa.

Data from the adjudicated efficacy population of 132 patients, who were confirmed to have major bleeding by the independent adjudication committee, and whose baseline anti-Factor Xa activity was substantially elevated (>75 ng/ml or 0.25 IU/mL if receiving enoxaparin), demonstrate that AndexXa rapidly and substantially reversed anti-Factor Xa activity, and these levels were sustained for the duration of administration.

Specifically, anti-Factor Xa activity, the co-primary efficacy endpoint, decreased by a median of greater than 90 percent for both apixaban and rivaroxaban following the bolus dose, which was sustained at similar levels for the duration of the two-hour infusion.

The independent adjudication committee determined that 109 of 132 patients (83 percent) achieved effective hemostasis, as defined by a hemostatic efficacy rating of "excellent" or "good" (the criteria used by the adjudication committee were based on similar criteria used in a pivotal study of Kcentra, approved for the reversal of Vitamin K antagonists). Among patients with gastrointestinal bleeding, 86 percent had effective hemostasis, as did 81 percent of patients with intracranial bleeding. Hemostatic efficacy was similar for patients on apixaban (82 percent) and rivaroxaban (83 percent).

Portola is developing andexanet alfa as a universal antidote for patients anticoagulated with an oral or injectable Factor Xa inhibitor, including apixaban and rivaroxaban, who experience a serious uncontrolled or life-threatening bleeding event or who require urgent or emergency surgery. Andexanet alfa is currently under review by the U.S. Food and Drug Administration (FDA), with an assigned action date of May 4, 2018, and by the European Medicines Agency (EMA), with an expected decision in 2019.

ANNEXA-4 Study Design
ANNEXA-4 is a global, single-arm, open-label clinical trial designed to evaluate andexanet alfa in patients who present with an acute major bleed while receiving apixaban, rivaroxaban, edoxaban or enoxaparin. This multi-center, prospective cohort study is not randomized and all participants receive andexanet alfa given as a bolus dose over 20-30 minutes followed by a two-hour (120 minute) infusion. Patients receive a low or high dose depending on which Factor Xa inhibitor they have received and the time they received the last dose. Patients are evaluated for 30 days following andexanet alfa administration. The co-primary efficacy endpoints are the maximum percent reduction in anti-Factor Xa activity and assessment of hemostasis over 12 hours following the infusion. Hemostatic efficacy is assessed by an independent endpoint adjudication committee as either excellent, good or poor/none.

Investor Event Webcast Information
Members of Portola’s senior management team, together with Dr. C. Michael Gibson, ANNEXA-4 Executive Committee member, Harvard Medical School professor and chairman of the PERFUSE Study Group, will review these new interim results during a conference call and live audio webcast today at 12:30 p.m. ET (9:30 a.m. PT) following the Late-Breaking Clinical Trial Session.

The conference call can be accessed by phone by calling (844) 452-6828 from the United States and Canada or 1 (765) 507-2588 internationally and using the passcode 8493604. To access the live and subsequently archived webcast, go to the Investor Relations section of the company’s website at View Source A replay will be available for 30 days following the live event.

Illumina Names Dr. Phil Febbo Chief Medical Officer

On March 12, 2018 -Illumina, Inc. (NASDAQ:ILMN) reported the appointment of Dr. Phil Febbo, a leading physician scientist, as Chief Medical Officer (CMO), starting March 26, 2018 (Press release, , DEC 12, 2018, View Source [SID1234524691]). In his new role, Dr. Febbo will be responsible for developing and executing on the Company’s medical strategy to drive genomic testing into healthcare practice.

"We are very excited to welcome Dr. Febbo, who comes to us at a transformative time in the evolution of genomic medicine"

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"We are very excited to welcome Dr. Febbo, who comes to us at a transformative time in the evolution of genomic medicine," said Garret Hampton, Ph.D., Executive Vice President of Clinical Genomics at Illumina. "Phil brings a wealth of clinical and scientific experience to Illumina, from understanding the genomic basis of common human cancers, to the development of clinical tests based on next generation sequencing. We believe that his experience will ensure that Illumina is uniquely positioned to change medical practice with the use of genomics."

For the past 25 years, Dr. Febbo has worked at leading institutions throughout the United States, most recently serving as CMO of Genomic Health (NASDAQ:GHDX). Prior to joining Genomic Health, Dr. Febbo served as Professor of Medicine and Urology at the University of California, San Francisco (UCSF), where his laboratory focused on using genomics to understand the biology and clinical behavior of prostate cancer, and his clinical practice focused on genitourinary oncology. While at UCSF, Dr. Febbo was the co-leader of the Prostate Cancer Program at the Helen Diller Family Comprehensive Cancer Center and the Program Principal Investigator of the Translational Research Program for the Alliance for Clinical Trials in Oncology.

Dr. Febbo holds a Bachelor of Arts degree in Biology from Dartmouth College, received his M.D. degree at UCSF, and completed his internal medicine residency at the Brigham and Women’s Hospital. After his fellowship in medical oncology at the Dana-Farber Cancer Institute, he was an Attending Physician in the Genitourinary Oncology Center at Dana-Farber, Instructor at Harvard Medical School, and a post-doctoral fellow in Dr. Todd Golub’s laboratory at Dana-Farber, as well as the Whitehead Institute Center for Genomic Research of MIT (now the Broad Institute). In 2004, Dr. Febbo moved to Duke University Medical Center’s Institute of Genome Sciences and Policy. He has been a member of the American Society for Clinical Investigation since 2009.