On February 11, 2020 Biodesix, Inc. reported initiation of the next phase of their biomarker development program with Merck KGaA, Darmstadt, Germany and Pfizer Inc., New York, NY (Press release, Biodesix, FEB 11, 2020, View Source [SID1234554175]). The companies have completed initial discovery and development of a new proteomic test that identifies likely responders to the anti-PD-L1 checkpoint inhibitor (BAVENCIO) (avelumab). Development efforts will now focus on transferring the test into Biodesix’s CLIA lab in Boulder, CO for clinical phase test validation. The new test was developed through retrospective analysis of the circulating proteome of cancer patients treated with the investigational drug candidate, utilizing the proprietary Biodesix Diagnostic Cortex artificial intelligence (AI) platform. Data from the initial test discovery and development program will be presented at an upcoming scientific meeting in 2020.

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The Diagnostic Cortex platform is based on modern AI techniques to design clinically useful tests that are reproducible and robust, and typically use hundreds of different markers. Based on data found in the circulating proteome and genome, Biodesix designs tests that support treatment decisions including patient selection for immunotherapies, novel therapy combinations, and alternative treatment pathways.

"We are pleased to announce this next phase in our collaboration with Merck KGaA, Darmstadt, Germany and Pfizer with the potential to extend treatment options to more patients. This relationship combines Biodesix’s strengths in test discovery and development, artificial intelligence, and their applications to immunotherapy, with the pharmaceutical leadership of Merck KGaA, Darmstadt, Germany and Pfizer," said Scott Hutton, Biodesix CEO. "The successful progression of this project is another example of how our proprietary Diagnostic Cortex AI-based biomarker platform is uniquely suited to advancing clinical research and investigating new indications for existing therapies."

Avelumab Approved Indications

Avelumab (BAVENCIO) in combination with axitinib is indicated in the US, EU, Japan and other countries for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

The US Food and Drug Administration (FDA) also granted accelerated approval for avelumab (BAVENCIO) for the treatment of (i) adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (mMCC) and (ii) patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy, or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. These indications are approved under accelerated approval based on tumor response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.

Avelumab is currently approved for patients with mMCC in 50 countries globally, with the majority of these approvals in a broad indication that is not limited to a specific line of treatment.

Avelumab Important Safety Information from the US FDA-Approved Label

The warnings and precautions for avelumab (BAVENCIO) include immune-mediated adverse reactions (such as pneumonitis and hepatitis [including fatal cases], colitis, endocrinopathies, nephritis and renal dysfunction and other adverse reactions [which can be severe and have included fatal cases]), infusion-related reactions, hepatotoxicity, major adverse cardiovascular events (MACE) [which can be severe and have included fatal cases], and embryo-fetal toxicity.

Common adverse reactions (reported in at least 20% of patients) in patients treated with BAVENCIO monotherapy include fatigue, musculoskeletal pain, diarrhea, nausea, infusion-related reaction, peripheral edema, decreased appetite/hypophagia, urinary tract infection and rash. Common adverse reactions (reported in at least 20% of patients) in patients receiving BAVENCIO in combination with axitinib include diarrhea, fatigue, hypertension, musculoskeletal pain, nausea, mucositis, palmar-plantar erythrodysesthesia, dysphonia, decreased appetite, hypothyroidism, rash, hepatotoxicity, cough, dyspnea, abdominal pain and headache. Grade 3-4 clinical chemistry and hematology laboratory value abnormalities reported in at least 10% of patients treated with BAVENCIO monotherapy include hyponatremia, lymphopenia, GGT increased; in patients receiving BAVENCIO in combination with axitinib, grade 3-4 clinical chemistry and hematology laboratory value abnormalities included blood triglyceride increased and lipase increased.

On February 11, 2020 Medivir AB (Nasdaq Stockholm: MVIR) reported investors, analysts and the media to an R&D Day in Stockholm, Monday March 2, 2020, at 14:00 to 16:30 CET (Press release, Medivir, FEB 11, 2020, View Source [SID1234554191]).

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The presentations will, among other things, address new clinical results from the phase Ia study with MIV-818 in liver cancer patients. Members of Medivir’s management team and Professor Jeff Evans, Director of the Institute of Cancer Sciences at the University of Glasgow, will give presentations.

The meeting will be held at Helio GT30, Grev Turegatan 30 in Stockholm, on March 2 at 14:00 to 16:30 CET.

To register now, send an e-mail to: [email protected]

A formal invitation with a full program will be sent out and be available on www.medivir.se.

Warm welcome!

About MIV-818
MIV-818 is a pro-drug designed to selectively treat liver cancers and to minimize side effects. It has the potential to become the first liver-targeted, orally administered drug to benefit patients with HCC and other forms of liver cancer.

On February 11, 2020 Marker Therapeutics, Inc. (NASDAQ:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on Marker’s planned trial investigating safety and efficacy of its novel MultiTAA T cell therapy in patients with post-transplant acute myeloid leukemia (AML) (Press release, TapImmune, FEB 11, 2020, View Source [SID1234554140]).

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Marker previously announced on November 12, 2019, that the FDA placed the trial on clinical hold. The FDA requested additional information and technical specifications for two legacy reagents supplied by third parties used in the MultiTAA-specific T cell manufacturing process. The technical specifications and data requested by the FDA could not be produced by the original suppliers. The Company identified alternative suppliers, satisfying the Agency’s request.

Based on data Marker provided, the FDA permitted the Company to initiate its AML trial, beginning with a safety lead-in portion. The FDA placed a partial clinical hold on the trial for the use of the MultiTAA-specific T cell product manufactured using one of the reagents supplied by the alternative supplier, until the final data and certificate of analysis for the reagent are reviewed and accepted by the Agency. The safety lead-in portion of the trial is expected to enroll approximately six patients as part of the amended trial design. Three patients will be dosed with MultiTAA-specific T cells manufactured using the legacy reagent, and three patients will be dosed with T cells manufactured using the reagent from the alternative supplier.

Marker currently estimates that the alternative supplier will deliver the final reagent, along with the final data and certificate of analysis required by the FDA, by the end of the second quarter of 2020. Marker anticipates to complete enrollment of the first three patients and submission of the final technical specifications and comparability data of the new reagents to the FDA during the second half of 2020, thereby satisfying the requirements for lifting the partial hold on the clinical trial. Given this expected timing, Marker does not currently expect the partial clinical hold to significantly impact site and patient enrollment of the AML trial.

The safety lead-in will be followed by the 160-patient randomized portion of the study at approximately 20 transplant centers. Group 1 will comprise 120 adjuvant (disease-free) patients, with the primary endpoint of relapse-free survival of patients receiving MultiTAA-specific T cell therapy versus a control group. Group 2 will comprise 40 active disease patients in a single arm, with primary endpoints of complete remission and duration of complete remission.

"With a clear path identified for getting our study of MultiTAA-specific T cell therapy underway in patients with AML, we’re focused on addressing the remaining requirements from the FDA and enrolling up to 20 clinical centers to conduct our Phase 2 trial," stated Peter L. Hoang, President and CEO of Marker Therapeutics. "We appreciate the productive dialogue with the FDA throughout the process and look forward to advancing MultiTAA-specific T cell therapy for patients with post-transplant AML in a randomized and multicenter clinical trial."

On February 11, 2020 SIRION Biotech GmbH ("SIRION"), a world leader in viral vector-based gene delivery technologies for gene and cell therapy, reported preliminary financial results for 2019 (Press release, Sirion Therapeutics, FEB 11, 2020, View Source [SID1234554176]). SIRION’s annual service and licensing revenues in 2019 exceeded €10M (US$11.2M). With a growing staff of 35 employees, the company reports a record profit and plans to invest a portion of these proceeds into an additional Munich site with expanded lab and process development capacities. In addition, SIRION’s offices in Paris and Boston will continue to expand.

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In addition, the company’s intellectual property is included in more than 10 clinical programs by leading drug developers, one of which was granted market approval in 2019. SIRION’s core fee-for-service business, providing vector materials for discovery and pre-clinical applications, experienced historic growth of more than 50% over the previous year. The company expanded into a new avenue of growth by assigning its share in joint intellectual property rights to both a client in USA and the Danish start-up, InProTher Aps, in return for shares and milestone payments as well as royalties on product net sales.

SIRION Biotech’s business model comes from service revenue and licensing fees. The company provides high-grade viral vector materials for preclinical use, offering up to 1015 GMP-ready AAV vector genomes and 1010 infectious units of Lentivirus, currently paving all the way to toxicology studies. In addition, the company is forecasting significant increased demand for its clinical and commercial grade vectors.

As SIRION continues its growth trajectory, it will focus on attracting funding to expand preclinical development with collaborators from its large client network. With more than 2,000 single projects for over 200 recurring clients worldwide, SIRION is committed to building significant intelligence and insights into latest gene therapy drug developments.

"After only 12 years in business, SIRION has been instrumental in bringing new gene therapy treatments to patients. Assigning intellectual properties to collaborators validates our comprehensive viral vector technology platforms and the highly skilled and creative lab staff that stands behind them. We continue our dedication and commitment to developing gene therapy on a global scale with the opening of our second Munich site this year that will expand our capacity for research and process development," said Dieter Lingelbach, Chief Operating Officer of SIRION.

Mr. Lingelbach continued, "In 2020, we look forward to continued expansion of our US presence through our wholly-owned subsidiary, SIRION Biotech International Inc., as well as continued growth in Paris."

Gene and cell therapies are among the hottest topics in modern drug development. Viral vector technologies are the most promising gene editing tools available today, with significant potential both as therapeutics for a growing number of indications, for tumor vaccines and for further technical improvements. Key challenges include quality, yields, and improved transduction in order to make novel gene therapies reliable and affordable. Costs for gene therapies per patient remain high, and can be prohibitive for larger patient populations, even in well-developed markets.

On February 11, 2020 BerGenBio ASA (OSE: BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for multiple cancer indications, reported its results for the fourth quarter and full year 2019 (Press release, BerGenBio, FEB 11, 2020, View Source [SID1234554192]).

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A presentation and live webcast by the Company’s management will take place today at 10.00 am CET in Oslo, please see below for details.

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "I am pleased to report on another eventful quarter for BerGenBio. During the period bemcentinib was approved for Fast Track Designation by the USA FDA for AML, and we continued to see encouraging data emerge from our ongoing phase II clinical trials with bemcentinib. The primary endpoint was met in our lung cancer trail in combination with Merck’s checkpoint inhibitor Keytruda. We were particularly pleased to see responses among patients for whom checkpoint inhibitors would not usually be expected to be effective. In January we reported achieving a similar endpoint in NSCLC patients who had previously relapsed on checkpoint inhibitors. Looking forward, we anticipate reporting clinical and translational data updates at medical conferences, later in the year. In January we closed a PIPE funding providing an additional NOK 220m, subject to EGM approval, such that the Company is well-financed well beyond current milestones."

Operational Highlights – fourth quarter and full year 2019

Bemcentinib met primary endpoint in first cohort of Phase II NSCLC study in combination with KEYTRUDA
Primary endpoint, Overall Response Rate, was been met in predominantly PD-L1 negative/low patients
Secondary endpoint, median Progression Free Survival, exceeds expectations in AXL positive patients
Data was presented at Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 34th Annual Meeting on 8 November 2019
Preliminary clinical data from Phase II combination trial of bemcentinib and LDAC in elderly AML patients at ASH (Free ASH Whitepaper) 2019
Phase II trial data showed bemcentinib in combination with low-dose cytarabine (LDAC) in elderly AML patients unfit for intensive therapy is well tolerated and shows promising efficacy
Long duration of response (>9.9 mo., still maturing) with 50% complete response (CR/Cri) in 6 evaluable newly diagnosed patients receiving the bemcentinib-LDAC combination
Clinical benefit demonstrated in >2L relapsed and refractory AML patients with 1 CR/CRi and 3 SD out of 6 evaluable patients
Data presented in a poster presentation at the 61st Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting, on 7-10 December in Orlando, Florida.
U.S. Food and Drug Administration (FDA) approved Fast Track Designation for bemcentinib for the treatment of elderly patients with acute myeloid leukaemia (AML) whose disease has relapsed.
With this Fast Track designation, BGB is eligible for:

More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval
Eligibility for Accelerated Approval and Priority Review
Clinical trial updates were presented at several major cancer scientific congresses and consistently confirm that NSCLC and AML are the optimal target indications
Phase II development programme read-outs according to plan
AML bemcentinib + LDAC: readouts at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and European Haematology Association (EHA) (Free EHA Whitepaper)
NSCLC bemcentinib + KEYTRUDA: readouts at ASCO (Free ASCO Whitepaper), World Conference on Lung Cancer (WCLC), European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper), The Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)
Proprietary composite AXL tumour-immune (cAXL) score developed to identify & diagnose patients that show durable benefit
Completed Phase Ia trial with tilvestamab (BGB149) in healthy volunteers
Continued to build out the organisation with strategic medical, clinical, operational and regulatory hires
Private placement completed in June, raising NOK 74.2 million
Q4 2019 / FY 2019 Financial Highlights

(Figures in brackets = same period 2018 unless otherwise stated)

Revenue amounted to NOK 0.2 million (NOK 2.3 million) for the fourth quarter and NOK 8.9 million (NOK 2.3 million) for the full year 2019
Total operating expenses for the fourth quarter were NOK 59.3 million (NOK 53.2 million) and total operating expenses for the full year 2019 amounted to 213.3 million (NOK 196.9 million)
Research and development expenses accounted for 77% (74%) of total operating expenses in the full year 2019
The operating loss for the quarter came to NOK 59.1 million (NOK 50.9 million) and NOK 204.4 million (NOK 194.5 million) for the full year 2019, reflecting the level of activity related to the clinical trials BerGenBio is conducting
Cash and cash equivalents amounted to NOK 253.6 million at the end of December 2019 (NOK 360.4 million)
Post-period events

Private placement of NOK 219.9 million successful placed through new share allocation

Net proceeds to be used to advance bemcentinib trial programs in AML and NSCLC, manufacturing scale-up of bemcentinib, biomarker and companion diagnostic development, and phase 1b development of tilvestamab.
Presentation and Webcast Details

A presentation by BerGenBio’s senior management team will take place today at 10:00 am CEST at:

Hotel Continental
Stortingsgata 24/26, Oslo

The presentation will be webcast live and the link will be available at www.bergenbio.com in the section Investors/Financial Reports. A recording will be available shortly after the webcast has finished.

The results report and presentation will be available at www.bergenbio.com in the section: Investors/Financial Reports from 7:00 am CET the same day.