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TLC Reports Fourth Quarter and Full Year 2018 Financial Results and Business Update

On April 1, 2019 TLC (Nasdaq: TLC, TWO: 4152), a clinical-stage specialty pharmaceutical company dedicated to the development and commercialization of novel nanomedicines designed to target areas of unmet medical need in osteoarthritis, pain management, ophthalmology and oncology, reported financial results for the fourth quarter and full year ended December 31, 2018, and provided a business update (Press release, Taiwan Liposome Company, APR 1, 2019, View Source;p=irol-newsArticle&ID=2392882 [SID1234534828]).

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"We had a transformative year in 2018, highlighted by outstanding results from the Phase II clinical trial of our lead program TLC599 for knee osteoarthritis pain, commencement of the first-in-human clinical trial of TLC590 for postsurgical pain management, and completion of a U.S. public offering on Nasdaq," said TLC President George Yeh. "As we move into 2019, we expect to continue to deliver strong progress in our clinical pipeline and company operations by further advancing our lead product candidates closer to pivotal clinical stages while capitalizing on strategic opportunities with our scalable manufacturing capabilities."

Clinical Pipeline Update and Upcoming Milestones

Phase II trial top-line data readout of TLC599 for knee osteoarthritis pain showed significant reductions in pain that sustained through 24 weeks. TLC599 demonstrated statistically significant improvements over placebo, both through 12, 16, 20, and 24 weeks and at every scheduled visit on week 1, 4, 8, 12, 16, 20, and 24. Over half of the patients treated with TLC599 maintained at least a 30% reduction in pain – over twice as many as placebo – throughout the study.

Phase I/II trial top-line data readout of TLC590 for post-surgical pain showed statistically significant and clinically meaningful improvement over the standard of care through 96 hours. In this first-in-human study of TLC590 in patients following inguinal hernia surgery, greater levels of pain reduction were maintained through the entire 168 hours of the study. In the TLC590 group, 58.3% were opioid-free throughout the study. Among those who used opioids, time to first post-surgical use was four times that of the ropivacaine (standard of care) group, and mean total opioid consumption was 54% less.

First patients dosed in Phase II trial of TLC590. The randomized, double-blind, comparator- and placebo-controlled two-part clinical trial will evaluate the safety, pharmacokinetics, and efficacy of TLC590 compared with ropivacaine and placebo in ~200 patients following bunionectomy. Data from Part 1 is expected mid-2019.

TLC178 received clearance from the FDA to initiate Phase I/II clinical trial in pediatric rhabdomyosarcoma. Recruitment of pediatric patients is scheduled to commence once the optimal dose in adults is identified in an analysis scheduled for the second half of 2019.

TLC178 received EMA orphan drug designation for the treatment of soft tissue sarcoma. The designation provides incentives and scientific advice from the EMA during product development phase and 10 years of marketing exclusivity in the EU following product approval. An orphan designation was previously granted by the US FDA, which provides 7 years of marketing exclusivity in the US following product approval.
Corporate Highlights

Completed U.S. IPO. TLC’s American Depositary Shares began trading on The Nasdaq Global Market under the ticker "TLC" on November 21, 2018, becoming the first Taiwan-based biotech company to be listed on Nasdaq. TLC has been listed on the Taipei Exchange since 2012, where it is consistently ranked in the top 5% among all listed companies in corporate governance evaluations.

Appointment of new Chief Medical Officer. Dr. George Spencer-Green, MD, MS, joined TLC in February 2019, bringing experience from serving as VP and clinical head of Pfizer’s biosimilars development program, and as medical director at the likes of Vertex Pharmaceuticals, Abbot Laboratories, and Enbrel, where he led the development of clinical programs, including Humira for rheumatoid arthritis.

Received an upfront payment in March 2019 as part of the commercialization agreement with 3SBio Inc. TLC is eligible for further regulatory and sales milestone payments, as well as a double-digit share of the potential profits from product sales.

Growing global intellectual property protection with 54 granted patents and 68 applications worldwide as of December 31, 2018.
Fourth Quarter and Full Year 2018 Financial Results

Operating revenue for the fourth quarter of fiscal 2018 was NT$17.4 million (US$0.6 million), a 40% increase compared to NT$12.4 million in the fourth quarter of fiscal 2017. Operating expenses for the fourth quarter of fiscal 2018 was NT$326.8 million (US$10.7 million), a 14.2% increase compared to NT$286.1 million in the fourth quarter of fiscal 2017. Net loss for the fourth quarter of fiscal 2018 was NT$306.7 million (US$10.0 million), compared to a loss of NT$262.6 million in the fourth quarter of 2017, or a net loss of NT$5.23 (US$0.17) per share for the fourth quarter of fiscal 2018, compared to a net loss of NT$4.73 per share for the fourth quarter of fiscal 2017.

Operating revenue for the fiscal year 2018 was NT$62.3 million (US$2.0 million), a 25.6% increase compared to NT$49.6 million in the fiscal year 2017. Operating expenses for the fiscal year 2018 was NT$980.3 million (US$32.0 million), a 3.4% increase compared to NT$948.1 million in the fiscal year 2017. Net loss for the fiscal year 2018 was NT$901.6 million (US$29.5 million), compared to a loss of NT$874.0 million in the fiscal year 2017, or a net loss of NT$14.37 (US$0.47) per share for the fiscal year 2018, compared to a net loss of NT$15.75 per share for the fiscal year 2017.

The Company’s cash and cash equivalents and time deposits with maturity over three months (which are classified as "current financial assets at amortized cost" in the Company’s consolidated financial statements) were NT$1,114.6 million (US$36.4 million) as of December 31, 2018, compared to NT$951.7 million at the end of the fourth quarter of fiscal 2017.

ASCO selects PharmaMar’s lurbinectedin
monotherapy trial for an oral presentation

On April 1, 2019 PharmaMar (PHM:MSE) reported that it has been notified by the lead author, Dr. Luis Paz-Ares, MD, PhD, Professor of Medicine at the Hospital Universitario 12 de Octubre in Madrid, Spain, that the abstract titled "Efficacy and safety profile of Lurbinectedin in second-line SCLC patients: results from a phase II single-agent trial" has been accepted by ASCO (Free ASCO Whitepaper) (American Society of Clinical Oncology) and has been selected for an oral presentation at the 19th Annual Meeting in Chicago, Illinois (Press release, PharmaMar, APR 1, 2019, View Source [SID1234534827]). Dr. Paz-Ares will present the data on June 1st.

In this oral presentation, the results referring to the phase II study of lurbinectedin, in monotherapy, with the 105 patients who have participated will be made public.

PharmaMar has already announced that the trial met its primary endpoint, as determined by both the investigator and the IRC (Independent Review Committee) assessments.

Dr. Paz-Ares and PharmaMar management will host a conference call after the data presentation. More details will follow in due course.

Legal warning
This press release does not constitute an offer to sell or the solicitation of an offer to buy securities, and shall not constitute an offer, solicitation or sale in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that jurisdiction

BerGenBio: Bemcentinib in combination with low-dose chemotherapy achieves efficacy endpoint in AML patients

On April 1, 2019 BerGenBio ASA (OSE:BGBIO) reported that on a top-line, preliminary basis, the first efficacy endpoint has been met in a Phase II clinical trial (BGBC003, NCT02488408) evaluating bemcentinib, a first-in-class selective oral AXL inhibitor, in combination with low-dose cytarabine (LDAC) as a potential new treatment regimen for acute myeloid leukaemia (AML) patients unable to tolerate intensive therapy (Press release, BerGenBio, APR 1, 2019, View Source [SID1234534826]). A second cohort evaluating bemcentinib in combination with decitabine is also ongoing and data is further maturing.

The pre-specified efficacy endpoint in this trial requires at least three patients out of the first 14 patients (21%) to achieve clinical responses* when treated with the novel drug combination.

The cohort evaluating bemcentinib in combination with LDAC, summarised below, has met its primary endpoint; data is further maturing:

In total, 15 patients were enrolled of which 10 are evaluable for response to date. Among these, 4 were newly diagnosed and 6 relapsed or refractory (R/R).
In newly-diagnosed patients, one complete remission (CR) and one CR with incomplete haematologic recovery(CRi) were observed (2 out of 4) and one CR among R/R patients (1 out of 6), yielding a CR/CRi rate of 30% (3 out of 10).
All responders were above the age of 75, at least one had secondary disease, and two had unfavourable cytogenetic profiles. Responses had an early onset (after 1 to 2 cycles), were durable and deepened over time and the combination of bemcentinib with LDAC was well tolerated causing no overlapping or new toxicities compared to each agent given alone.
An extensive translational programme continues to explore potential biomarkers for bemcentinib combination therapy. Further details will be presented at upcoming medical conferences.

Furthermore, the Company is proceeding with preparations for a late-stage bemcentinib monotherapy trial in AML. The study, expected to start in the second half of 2019, will aim to confirm previously reported monotherapy efficacy of bemcentinib in AML patients whose disease has progressed on standard of care therapy. Correlation with relevant biomarkers will be explored with the ambition to pursue an accelerated development path in a selected patient population.

Professor Dr Dr Sonja Loges, attending physician at the University Hospital Hamburg-Eppendorf and lead investigator of the BGBC003 trial, commented: "Data gathered by our group and others have established AXL as a negative prognostic factor in patients with AML and other malignancies. AXL acts both as a driver of leukaemic cell proliferation and as negative regulator of anti-tumour immunity and therefore represents a novel and promising target in AML. Bemcentinib, an oral tablet, targets AXL selectively and I am pleased to see activity in my patients receiving bemcentinib alone or in combination with standard low-intensity therapies while side effects thus far have proven to be limited and manageable. These early results warrant further investigation of bemcentinib in a larger trial addressing AML patients unfit for intensive chemotherapy."

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "Clearing the efficacy threshold for bemcentinib in combination with LDAC is very encouraging, particularly as we have seen responses in a less fit AML patient population who are considered to have unfavourable prognosis after the failure of first-line therapies, or those with high risk cytogenetics. AML patients represent a challenging patient population for clinical trials, but we are keen to pursue AML monotherapy as our first indication based on the high unmet need, strength of clinical proof-of-concept with biomarker correlation, excellent safety and potential for a fast and first route to registration. We look forward to providing more details on the trial design upon initiation and reporting further data with bemcentinib combinations in this indication as it matures." *defined as objective response (OR), as measured by the revised recommendations of the International Working Group (IWG) in AML

END

About AML and the BGBC003 trial
AML is the most common form of acute leukaemia in adults where malignant AML blasts interfere with the normal functioning of the bone marrow leading to a multitude of complications like anaemia, infections and bleeding. AML is diagnosed in over 20,000 patients in the US annually and is rapidly lethal if left untreated. Successful treatment typically requires intensive therapy or bone marrow transplantation, and relapse and resistance are common. Consequently, there is an urgent need for effective novel therapies in relapsed/refractory patients, particularly those that are ineligible for intensive therapy or bone marrow transplant.

The BGBC003 trial is a phase Ib/II multi-centre open label study of bemcentinib in combination with cytarabine (part B2) and decitabine (part B3) in patients with AML who are unsuitable for intensive chemotherapy as a result of advanced age or existing-co-morbidities.

For more information please access trial NCT02488408 at www.clinicaltrials.gov.

About AXL
AXL kinase is a cell membrane receptor and an essential mediator of the biological mechanisms underlying life-threatening diseases. In cancer, AXL suppresses the body’s immune response to tumours and drives cancer treatment failure across many indications. AXL inhibitors, therefore, have potential high value at the centre of cancer combination therapy, addressing significant unmet medical needs and multiple high-value market opportunities. Research has also shown that AXL mediates other aggressive diseases.

Thermo Fisher Scientific to Hold Earnings Conference Call on Wednesday, April 24, 2019

On April 1, 2019 Thermo Fisher Scientific Inc. (NYSE: TMO), the world leader in serving science, reported that it will release its financial results for the first quarter of 2019 before the market opens on Wednesday, April 24, 2019, and will hold a conference call on the same day at 8:30 a.m. EDT (Press release, Thermo Fisher Scientific, APR 1, 2019, View Source [SID1234534825]).

During the call, the company will discuss its financial performance, as well as future expectations. To listen, call (877) 273-7122 within the U.S. or (647) 689-5496 outside the U.S. You may also listen to the call live on the "Investors" section of our website, www.thermofisher.com. The earnings press release and related information can be found in that section of our website under "Financial Results." A replay of the call will be available under "Webcasts and Presentations" through Friday, May 10, 2019.

Fate Therapeutics Presents Late-Breaking Preclinical Data Highlighting Unique Advantages of Clonal Master Engineered iPSC Lines for Off-the-Shelf CAR T-cell Therapy at 2019 AACR Annual Meeting

On April 1, 2019 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported late-breaking preclinical data highlighting the Company’s unmatched ability to mass produce uniformly engineered chimeric antigen receptor (CAR) T cells for off-the-shelf cancer immunotherapy at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 in Atlanta, Georgia (LB-073/18: Generation of Novel Single Cell-derived Engineered Master Pluripotent Cell Line as a Renewable Source for Off-the-shelf TCR-less CAR T Cells in support of First-of-kind Clinical Trial) (Press release, Fate Therapeutics, APR 1, 2019, View Source [SID1234534823]).

The Company’s proprietary approach to CAR T-cell therapy utilizes a one-time genetic modification event followed by high-throughput selection of a single gene-edited induced pluripotent stem cell (iPSC). In contrast to repeatedly engineering large populations of patient- or donor-derived T cells which results in batch-to-batch and cell-to-cell variability, the Company has shown that a single gene-edited iPSC can be maintained as a clonal master engineered iPSC line which can be repeatedly used for production of uniformly engineered CAR T cells.

"The efficiency, accuracy, and uniformity of genetic modifications have the potential to impact the safety and efficacy of CAR T-cell therapy. We know that, when engineering a population of T cells, current gene-editing technologies create genomic heterogeneity and result in undesired byproducts, such as DNA mutagenesis and translocations. The selection of a single gene-edited cell for the derivation of a clonal master engineered iPSC line ensures that the effects of gene editing can be fully characterized and that only engineered T cells meeting rigorous quality standards are administered to patients," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics.

New preclinical data presented today at AACR (Free AACR Whitepaper) for FT819, the Company’s universal, off-the-shelf CAR19 T-cell product candidate being developed under a collaboration with Memorial Sloan Kettering Cancer Center (MSK) led by Michel Sadelain, M.D., Ph.D., demonstrate the production of clonal master engineered iPSC lines having complete elimination of T-cell receptor (TCR) expression and insertion of a novel 1XX CAR signaling domain into the T-cell receptor alpha (TRAC) locus. These synthetic features are intended to mitigate the risk of graft-versus-host disease, a severe life-threatening condition that occurs when donor T cells attack a patient’s healthy tissue, and to regulate CAR expression to enhance the therapeutic profile of CAR T cells.

Scientists from the Company and MSK are using clonal master engineered iPSC lines under the collaboration to overcome the complexity, heterogeneity, and substantial costs associated with engineering T cells from a patient or a donor. As proof-of-principle for the therapeutic advantages arising from selecting a single gene-edited cell for the production of CAR T-cell therapy, the scientists engineered a population of 545 cells using CRISPR-directed gene editing and found that only about 5% of cells contained both bi-allelic disruption of the TCR and insertion of the CAR into the TRAC locus. Upon further characterization for off-target genomic modifications and functional performance, only about 2% of the cell population were determined to meet the Company’s standards for genomic integrity and overall quality.

Previously published work by Dr. Sadelain in the journal Nature has shown that directing a CD19-specific CAR to the TRAC locus enhances T-cell potency, and that TRAC-targeted CAR T cells vastly outperform conventionally-generated CAR T cells in a mouse model of acute lymphoblastic leukemia (View Source). Additionally, Dr. Sadelain has shown in published work described in the journal Nature Medicine that the third-generation 1XX CAR signaling domain balances T-cell effector and memory programs, resulting in CAR T cells with potent activity and longer durability (View Source).

Fate Therapeutics has exclusively licensed from MSK foundational intellectual property covering the production and composition of iPSC-derived T cells for human therapeutic use. Additionally, in May 2018, the Company expanded its existing license agreement with MSK to include certain patents and patent applications relating to a novel 1XX CAR construct and off-the-shelf CAR T cells, including the use of CRISPR and other innovative technologies for their production. In addition, Fate Therapeutics owns an extensive intellectual property portfolio that broadly covers compositions and methods for the genome editing of iPSCs using CRISPR and other nucleases, including the use of CRISPR to insert a CAR in the TRAC locus for endogenous transcriptional control.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered in repeat doses to mediate more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf to treat many patients. As a result, the Company’s platform is uniquely capable of addressing the limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is fraught with batch-to-batch and cell-to-cell variability that can affect safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 100 issued patents and 100 pending patent applications.