VBI Vaccines Announces Dosing of First Recurrent GBM Patient in Phase 2a
Study of Cancer Vaccine Candidate, VBI-1901, in Combination with GSK’s AS01B
Adjuvant System

On March 2, 2020 VBI Vaccines Inc. (NASDAQ: VBIV) ("VBI"), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported that the first patient has been dosed in the second study arm in the ongoing Phase 2a clinical study of VBI-1901, VBI’s cancer vaccine immunotherapeutic candidate (Press release, VBI Vaccines, MAR 2, 2020, View Source [SID1234555047]). The Phase 2a study is a two-arm, open-label study, enrolling 20 first-recurrent GBM patients to receive VBI-1901 in combination with either GM-CSF or AS01B, GlaxoSmithKline’s (GSK) proprietary adjuvant system, as immunomodulatory adjuvants.

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"We are excited to initiate enrollment in this study arm to evaluate VBI-1901 in combination with AS01B, GSK’s effective proprietary adjuvant system," said David E. Anderson, Ph.D., VBI’s Chief Scientific Officer. "AS01B has been shown to boost T-cell mediated immunity and, building on the encouraging early immunologic and tumor response data we’ve seen to-date from the VBI-1901 with GM-CSF study arm, we look forward to evaluating the benefit this combination could have for patients with GBM, a devastating disease with few treatment options."

"We are pleased that this study is now underway as this is the first time AS01B will be assessed in oncology for GBM patients in a clinical setting. We are looking forward to seeing initial results later this year and hope that this technology will be able to make a real difference for GBM patients. Applying our expertise in adjuvant technologies in new fields of vaccines research is a core part of our innovation strategy," said Emmanuel Hanon, Senior Vice President, Head of R&D at GSK Vaccines.

VBI’s ongoing two-part study is being conducted at The Neurological Institute of New York Columbia University Medical Center, Dana-Farber Cancer Institute, Massachusetts General Hospital, and the Ronald Reagan UCLA Medical Center.

About the Phase 1/2a Study Design

VBI’s two-part Phase 1/2a study is a multi-center, open-label, dose-escalation study of VBI-1901 in up to 38 patients with recurrent GBM:

●Part A

●Dose-escalation phase that defined the safety, tolerability, and optimal dose level of VBI-1901 adjuvanted with granulocyte-macrophage colony-stimulating factor (GM-CSF) in recurrent GBM patients, with any number of prior recurrences.
●This phase enrolled 18 recurrent GBM patients across three dose cohorts of VBI-1901: 0.4 µg, 2.0 µg, and 10.0 µg.
enrollment completed in December 2018

●Part B

●Subsequent extension of the optimal dose level, 10.0 µg, as defined in the Part A dose escalation phase.
●This phase is a two-arm study, enrolling 10 first-recurrent GBM patients in each arm, assessing 10.0 µg of VBI-1901 in combination with either GM-CSF or GSK’s proprietary AS01B adjuvant system as immunomodulatory adjuvants.
●Enrollment in each study arm is ongoing

VBI-1901 is administered intradermally when adjuvanted with GM-CSF and will be administered intramuscularly when adjuvanted with AS01B adjuvant system. Patients in the study will receive the vaccine immunotherapeutic every four weeks until clinical disease progression.

Additional information, including a detailed description of the study design, eligibility criteria, and investigator sites, is available at ClinicalTrials.gov using identifier NCT03382977.

Veracyte Announces Publication of Nearly 700-Patient Study Using Prosigna Breast Cancer Test to Identify Patients Likely to Benefit from Aggressive Chemotherapy

On March 2, 2020 Veracyte, Inc., (Nasdaq: VCYT) reported the publication of a large, retrospective study in which the Danish Breast Cancer Group (DBCG) used the Prosigna breast cancer prognostic gene signature assay to identify which women were more likely to benefit from aggressive chemotherapy based on their intrinsic breast cancer subtype (Press release, Veracyte, MAR 2, 2020, View Source [SID1234555046]). The findings appear online in npj Breast Cancer, a peer-reviewed open-source journal, and suggest that women with HER2-enriched subtypes may benefit from commonly used anthracycline-containing chemotherapy regimens, while those with luminal breast cancer subtypes – which accounted for 30 percent of the women in the study – could potentially be spared such treatment regimens, which have significant cardiotoxicity in some patients.

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The findings are from a retrospective analysis of the DBCG89D trial – a prospectively designed, randomized trial of 980 pre- and post-menopausal Danish women with early-stage breast cancer who were administered adjuvant chemotherapy regimens that contained anthracycline (epirubicin) or were CMF- (cyclophosphamide, methotrexate and fluorouracil) based. Among the 686 women with Prosigna results, overall survival was significantly different in anthracycline- versus non-anthracycline treated patients with HER2-enriched (Hazard Ratio of 0.72, 95% Confidence Interval 0.52; 0.99) tumors, but not in patients with luminal A (Hazard Ratio of 1.62, 95% Confidence Interval 0.97; 2.71) or luminal B (Hazard Ratio of 1.41, 95% Confidence Interval 0.80; 2.47) cancers. Of the 686 women with Prosigna results, the test identified 132 women with luminal A, 78 with luminal B, 259 with basal-like and 217 with HER2-enriched breast cancer subtypes.

"Our study was a carefully planned, formal re-analysis of an important Danish clinical trial that helped establish the use of anthracycline chemotherapies in breast cancer — an aggressive treatment that saves lives but can have serious side effects, including heart damage," said Torsten Nielsen, M.D., Ph.D., professor of pathology and laboratory medicine at BC Cancer, the University of British Columbia, a co-author of the study and a developer of the gene signature on which the Prosigna test is based.

"By applying Prosigna technology, which had not yet been invented when the tumor tissue from this trial was originally collected and stored, we found that patients with luminal subtypes did not benefit from putting anthracycline drugs into the chemotherapy regimen. These drugs only provided added benefit to those with non-luminal molecular subtypes. The study contributes to the accumulating body of evidence that the breast cancer molecular subtypes and risk scores identified with Prosigna technology can help identify which women can safely back off of aggressive chemotherapy."

The Prosigna test uses advanced genomic technology to inform next steps for patients with early-stage breast cancer, based on the genomic make-up of their disease. The test analyzes the activity of 50 genes known as the PAM50 gene signature, along with clinical-pathological features, and can provide a hormone-receptor positive early breast cancer patient and her physician with a prognostic score indicating the probability of cancer recurrence during the next 10 years. Outside of the United States, it is also utilized to provide PAM50 molecular subtype information.

"These new data suggest that the Prosigna test may be able to offer new levels of individualized treatment for women with early-stage breast cancer, potentially enabling many to avoid more aggressive chemotherapy regimens," said Bonnie Anderson, Veracyte’s chairman and chief executive officer. "These findings also suggest exciting future expansion opportunities for the Prosigna test’s positioning in global markets where intrinsic breast cancer subtypes are reported in the test results."

Veracyte acquired the Prosigna test from NanoString Technologies, Inc. in December 2019 as part of its acquisition of the exclusive global diagnostic rights to the nCounter FLEX Analysis System. The DBCG retrospective study was supported by funds from NanoString Technologies.

About the Prosigna Breast Cancer Prognostic Gene Signature Assay

Physicians use Prosigna to help guide therapeutic decisions so that patients receive therapeutic interventions, such as chemotherapy, only if clinically warranted. The in vitro diagnostic test is indicated in female breast cancer patients who have undergone surgery in conjunction with locoregional treatment consistent with standard of care, either as:

A prognostic indicator for distant recurrence‐free survival at ten years in post‐menopausal women with Hormone Receptor‐Positive (HR+), lymph node‐negative, Stage I or II breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors; or
A prognostic indicator for distant recurrence‐free survival at ten years in post‐menopausal women with Hormone Receptor‐Positive (HR+), lymph node‐positive (1‐3 positive nodes), Stage II breast cancer to be treated with adjuvant endocrine therapy alone, when used in conjunction with other clinicopathological factors. The device is not intended for patients with 4 or more positive nodes.
The Prosigna test is FDA 510(k) cleared in the United States for use on the nCounter Dx Analysis System and is available for use when ordered by a physician. The test is performed on formalin-fixed and paraffin-embedded tissue. The Prosigna test has been CE-marked, showing that it conforms with European Union regulations, and is available for use by healthcare professionals in the European Union and other countries that recognize the CE mark, as well as in Canada, Israel, Australia, New Zealand and Hong Kong. The test is covered by Medicare and leading private payers in the United States and is widely covered by government and private payers in the countries where it is available.

Immunocore Secures $130 Million Series B Financing

On March 2, 2020 Immunocore, a pioneering, clinical-stage T cell receptor biotechnology company working to develop and commercialize a new generation of transformative medicines to address unmet needs in cancer, infection and autoimmune disease, reported the completion of its Series B private financing round, generating more than $130 million (Press release, Immunocore, MAR 2, 2020, View Source [SID1234555045]).

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Immunocore’s Series B round is led by General Atlantic, a leading global growth equity firm and new investor in Immunocore. Other new investors participating in this round include CCB International, JDRF T1D Fund, Rock Springs Capital, Terra Magnum Capital Partners and WuXi AppTec’s Corporate Venture Fund. Five existing shareholders in the Company, including Eli Lilly and Company and RTW Investments, are also participating, as well as the Bill & Melinda Gates Foundation through conversion of its outstanding loan note.

The proceeds will enable Immunocore to further expand and accelerate its rapidly growing clinical stage pipeline of ImmTAX (Immune mobilising monoclonal TCRs Against Cancer, Infectious Diseases and Autoimmune) molecules that includes three oncology programs in MAGE-A4 (in collaboration with Genentech), NYESO-1 (in collaboration with GSK), and the lead program tebentafusp (IMCgp100), which is in pivotal clinical studies as a potential treatment for patients with metastatic uveal melanoma.

The proceeds will allow the company to advance two wholly owned clinical-stage internal programs for chronic Hepatitis B and for PRAME, a target expressed in a wide range of tumors. This investment is also expected to accelerate Immunocore’s novel platform to treat autoimmunity, including type one diabetes (in collaboration with the JDRF T1D Fund), advance the TCR platform and expand its database of rich and novel targets.

Bahija Jallal, Ph.D., Chief Executive Officer and Director of Immunocore, commented: "We are extremely pleased that General Atlantic is leading our Series B round and welcome them, along with our new investors, to Immunocore. This new funding – from an international cadre of health care investors joined by some of our existing shareholders – represents a further endorsement of our unique and powerful platform technology, our novel class of TCR-based biologic therapies, the accomplished scientists at Immunocore and our mission to transform the lives of people with serious diseases."

Rob Perez, Operating Partner, General Atlantic, said: "We are pleased to partner with Immunocore at this key time in its development, with lead candidate tebentafusp on the cusp of commercialization and two further assets in the clinic. We are excited by the broad applicability of the science within the Company’s ImmTAX platform and the Company’s promising pipeline across oncology, infectious and autoimmune diseases. We look forward to providing impactful support to Immunocore as it continues on its strong growth trajectory."

Marker Therapeutics and Aspire Capital Enter into a Common Stock Purchase Agreement for up to $30 million

On March 2, 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 Company has entered into a Common Stock Purchase Agreement (the "Agreement") of up to $30 million with Aspire Capital Fund, LLC ("Aspire"), a Chicago-based institutional investor and long-term Marker shareholder (Press release, TapImmune, MAR 2, 2020, View Source [SID1234555044]).

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Under the terms of the Agreement, Aspire has committed to purchase up to $30 million of the Company’s common stock at Marker’s discretion from time to time during a 30-month period at prices based on the market price at the time of each sale. Marker will retain full control as to the timing and amount of any sale of shares of common stock to Aspire, subject to certain limitations specified in the Agreement.

There are no warrants, options, financing swaps, derivatives or other securities associated with this Agreement. Additionally, there are no financial covenants or restrictions on future financings and there are no rights of first refusal, participation rights, penalties or liquidated damages. Lastly, Marker maintains the right to terminate the Agreement at any time, at its discretion, without any additional cost or penalty. Proceeds from the Agreement will be used to further advance the Company’s pipeline including its post-transplant acute myeloid leukemia (AML) Phase 2 trial, which is expected to begin in 2020, as well as for general corporate purposes.

"This Agreement with Aspire provides Marker with the opportunity to access capital in an efficient manner," stated Peter L. Hoang, President and CEO of Marker. "The financial flexibility provided by this transaction will further support the advancement of our clinical programs including the first Marker-sponsored clinical trial this year investigating our novel MultiTAA cell therapy."

As consideration for Aspire’s entering into the Agreement, Marker issued 345,357 shares to Aspire as a commitment fee. Additional detail regarding the Agreement is set forth in Marker’s Current Report on Form 8-K, filed today with the SEC.

Primmune Therapeutics Raises $7 Million Seed Financing to Develop Novel Oral TLR7 Immunotherapies for Cancer and Acute Viral Diseases

On March 2, 2020 Primmune Therapeutics reported that it has raised $7 million in seed financing to support the development of novel orally-administered, small molecule toll-like receptor 7 (TLR7) agonists for long-term systemic activation of innate immunity (Press release, Primmune Therapeutics, MAR 2, 2020, View Source [SID1234555043]). Investors in the seed financing were CAM Capital, Charlie McDermott, BioBrit and BioRock Ventures.

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In conjunction with the financing, Scott Morenstein, Managing Director at CAM Capital will join the board of directors. Ezra Cohen, M.D., Chief, Division of Hematology-Oncology and Associate Director, Translational Science, Moores Cancer Center at the University of California, San Diego and Isan Chen, M.D., Chief Medical and Development Officer at Mirati Therapeutics will join the company’s clinical advisory board.

"The recent advances in cancer immunotherapy have resulted in medicines that have had profound benefit for cancer patients; yet, a majority of those patients do not respond or relapse. There is a strong rationale for combining agents that activate systemic innate immunity, like a TLR7 agonist, with drugs that engage systemic adaptive immunity, like checkpoint inhibitors, to potentially address patients who have an inadequate response to approved treatments," said Charlie McDermott, Chief Executive Officer of Primmune Therapeutics. "We are pleased to have secured financial support from our investors and local biotech executives who are committed to supporting the advancement of our small molecule TLR7 agonists into human studies in oncology and virology."

Primmune is being led by an experienced and dedicated team, which includes:

• Charlie McDermott, Chairman, President & Chief Executive Officer
• James Appleman, Ph.D., Co-founder, Board Member, SVP, R&D and Chief Scientific Officer
• Paulo Rangel, President, Co-founder, Board Member, Chief Business Officer
• Stephen Webber, Ph.D., Co-founder and SVP Medicinal Chemistry
• Todd Harris, Ph.D., Board Member, CEO of Tyra Biosciences

Mr. McDermott is Chairman, President & CEO of Primmune. He brings more than 25 years of experience in the life sciences industry and has held roles of increasing responsibility in drug discovery, regulatory affairs, corporate development, commercial planning, and executive management. Most recently, Mr. McDermott was President, Chief Business Officer, and a member of the board of directors of Impact Biomedicines, Inc. While at Impact, Mr. McDermott raised more than $110 million in venture and non-dilutive royalty financing to fully fund the development and commercialization of fedratinib (INREBIC) for the treatment of adult patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis (MF). Impact was acquired by Celgene in 2018 for $1.1 billion upfront and up to $7 billion overall, if all contingent payments are realized. Prior to joining Impact, Mr. McDermott was President and Chief Business Officer of Kala Pharmaceuticals, Inc., where he helped lead the transformation of Kala from a private pre-clinical stage company to a pre-commercial stage public company. To this end, Mr. McDermott helped raise approximately $190 million via private equity, debt, and an initial public offering. In August of 2018, Kala received NDA approval for Inveltys, an internally developed drug for ocular indications. Before joining Kala, Mr. McDermott worked at Allergan as Vice President, Global Business Development for the Eye Care and Drug Delivery business units. At Allergan, Mr. McDermott led efforts to license and acquire a variety of technologies and therapeutics including Lastacaft, Acuvail, Restasis MultiDose, Latisse, and Abicipar. Before joining Allergan, Mr. McDermott held positions as Associate Director of Business Development at deCODE genetics and before that, in drug discovery and regulatory affairs at Pfizer/Agouron. Mr. McDermott has an M.A. in molecular, cellular, and developmental biology from University of California, Santa Barbara, an MBA from the University of San Diego and a B.A. in biochemistry and molecular biology from the University of California, Santa Cruz.

Dr. Appleman is SVP, R&D and CSO, Co-founder, Director of Primmune. Dr. Appleman brings nearly 30 years of experience in building successful drug discovery organizations while having served as an advisor to biotech, hi-tech, and diagnostic firms. Dr. Appleman is a Co-founder of eFFECTOR Therapeutics where he established both the technology base and bioinformatics infrastructure to support eFFECTOR’s unique programs targeting dysregulated mRNA translation. He previously served as Senior Vice President, Research and Chief Scientific Officer at Anadys Pharmaceuticals, Inc. where he played a pivotal role in the business process culminating in the acquisition of Anadys for $230M (256% premium to market cap) by Roche. Additionally, he led the invention, characterization and clinical development of the Anadys’ internally discovered drug candidates setrobuvir and ANA773, a novel oral TLR7 agonist for the treatment of cancer and hepatic viral diseases. Prior to joining Anadys, Dr. Appleman held positions at Gensia, Inc. and its subsidiary Metabasis Therapeutics and was a faculty member at St. Jude Children’s Research Hospital. He received a Ph.D. in biochemistry from Oklahoma State University and completed his postdoctoral training at Dartmouth Medical School.

Mr. Rangel is Chief Business Officer, Co-founder, Director of Primmune. He brings 25 years of experience in the pharmaceutical and related industries in both small and large companies. Before Primmune, Mr. Rangel was President & CEO and Co-founder of Evince Biosciences, a precursor to Primmune Therapeutics. Before Evince, Mr. Rangel was a Partner at ProPharma Partners International, an international consulting group where he focused on working with biopharmaceutical and medical device companies on worldwide in- and out- licensing, valuations, business plans and market research. Before ProPharma, Mr. Rangel was Head of Global Business Development at Besins Healthcare, a global pharmaceutical company with products targeted at women’s and men’s health. Mr. Rangel was a founder of, and key executive in, two medical device start-ups, Lasercure Sciences and 5i Sciences (now Sommetrics). In those positions, he managed the financing, clinical studies and collaborations, and was responsible for all intellectual property. Mr. Rangel has filled key roles in a number of other biotechnology companies and worked at Amgen and Hybritech/Lilly early on in his career. Mr. Rangel received a B.A. in chemistry and biochemistry from the University of California, San Diego and an MBA from The Fuqua School of Business at Duke University.

Dr. Webber is Co-founder and SVP of Medicinal Chemistry at Primmune. He brings 33 years of experience in drug discovery in the biotech and pharmaceutical industry. Prior to Primmune, Dr. Webber was Executive Director of Medicinal Chemistry at Polaris Pharmaceuticals where he was responsible for the discovery and synthesis of small molecules against various oncology targets using structure-based drug design. Before joining Polaris, Dr. Webber was a Co-founder and Director of Medicinal Chemistry at eFFECTOR Therapeutics, a company devoted to the discovery and development of translation regulators for cancer. He is a Co-inventor of tomivosertib, an orally active MNK1/2 inhibitor. Previously, Dr. Webber led discovery chemistry efforts at Anadys Pharmaceuticals Inc. where he served at various senior levels until the company was acquired by Roche. His contributions include the discovery of anti-HCV clinical candidates setrobuvir, a non-nucleoside NS5B inhibitor and ANA-773, a second generation orally active TLR-7 agonist prodrug demonstrating antiviral and anticancer activity. Dr. Webber’s career extends back to the inception of Agouron Pharmaceuticals Inc. where he helped pioneer protein crystal structure- and computer-based drug design. Dr. Webber discovered and invented several PARP inhibitors, including Rucaparib (marketed as Rubraca), a FDA approved medicine to treat ovarian cancer in patients with the BRCA, or BRCA-like mutations. Dr. Webber also discovered and co-invented several clinical candidates including nolatrexed dihydrochloride (Thymitaq). He also made significant contributions to the rhinovirus protease project leading to the advancement of ruprintrivir as a clinical candidate. Dr. Webber received his Ph.D. from the University of Pennsylvania and his B.S. from Philadelphia University.