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Popular Science Names BioMarin’s Brineura® (cerliponase alfa) One of the Top Health Innovations of 2017 with “Best of What’s New” Award
SAN RAFAEL, Calif., Oct. 18, 2017 /PRNewswire/ — BioMarin announced today that Brineura® (cerliponase alfa) has been awarded the 2017 Popular Science “Best of What’s New” award in the health category. Each year, Popular Science reviews thousands of new products and innovations across 11 categories for its annual “Best of What’s New” issue, selecting those that represent a significant step forward in their category.
Brineura is the first therapy approved by the U.S. Food and Drug Administration to treat children with a form of Batten disease, CLN2 disease. CLN2 disease is an ultra-rare and fatal pediatric brain disorder that affects less than one in a million U.S. residents. Brineura is a prescription medication used to slow loss of ability to walk or crawl (ambulation) in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. It was approved in the U.S. in April 2017 and the European Union in June 2017.
“We are honored that Brineura has been recognized as one of the top scientific accomplishments in 2017 by Popular Science,” said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. “We share this recognition with the Batten disease patient and medical research communities who played an integral role in making Brineura available to children affected by CLN2 disease. As the first enzyme replacement therapy to be directly administered to the brain, our hope is that this scientific breakthrough blazes a trail for more therapies to treat other neurodegenerative diseases in a meaningful way.”
“Until this year, there was no approved treatment for children with any form of Batten disease. Our desire is that this therapy for the CLN2 form of the disease and this recognition of scientific achievement will encourage research into other forms of this devastating disease,” said Margie Frazier, PhD, LISW-S, Executive Director of Batten Disease Support and Research Association. “Brineura represents a collaborative scientific effort among patients, industry and academia to bring forth a treatment that seemed impossible just five years ago.”
Every year approximately 20 children are born in the U.S. with CLN2 disease. These affected children suffer from drug resistant seizures, movement disorder and completely lose the ability to walk and talk by the time they reach six years of age. During the later stages of the disease, feeding and tending to everyday needs become very difficult with death often occurring between eight and 12 years of age.
Brineura is the first enzyme replacement therapy to be directly administered to the brain, treating the underlying cause of the condition by helping to replace the deficient TPP1 enzyme. Using an established technique most often used in oncology – intraventricular administration – the therapy is delivered directly into fluid surrounding the brain, known as the cerebrospinal fluid.
“The ‘Best of What’s New’ awards honor the innovations that shape the future,” said Joe Brown, Editor-in-Chief, Popular Science. “From life-saving technology to incredible space engineering to gadgets that are just breathtakingly cool, this is the best of what’s new.”
Brineura was approved in less than four years from the start of the first clinical trial to approval, a significant achievement for a condition that progresses so rapidly.
About Best of What’s New
Each year, the editors of Popular Science review thousands of products in search of the top 100 tech innovations of the year—breakthrough products and technologies that represent a significant leap in their categories. The winners, the Best of What’s New, are awarded inclusion in the much-anticipated December issue of Popular Science, the most widely read issue of the year since the debut of Best of What’s New in 1988. Best of What’s New awards are presented to 100 new products and technologies in 11 categories: Automotive, Aerospace, Engineering, Entertainment, Gadgets, General Innovation, Security, Software, Home, Health and Recreation.
About Popular Science
Founded in 1872, Popular Science is the world’s largest science and technology magazine with a circulation of 1.3 million and 6.8 million monthly readers. Each month, Popular Science reports on the intersection of science and everyday life, with an eye toward what’s new and why it matters. Popular Science is published by Bonnier Active Media, a subsidiary of Bonnier Corporation.
About CLN2 Disease
Children with CLN2 disease typically begin experiencing seizures between the ages of 2 and 4 years old, preceded in the majority of cases by language development delay. The disease progresses rapidly with most affected children losing the ability to walk and talk by approximately 6 years of age. Initial symptoms are followed by movement disorders, motor deterioration, dementia, blindness, and death usually occurring between the ages of 8 and 12 years of age. During the later stages of the disease, feeding and tending to everyday needs become very difficult. BioMarin estimates the incidence of CLN2 disease is approximately one in 200,000 with up to 1,200 to 1,600 children in the regions of the world where BioMarin operates, many of whom are undiagnosed.
The neuronal ceroid lipofuscinoses (NCLs) are a heterogeneous group of lysosomal storage disorders that includes the autosomal recessive neurodegenerative disorder CLN2 disease. CLN2 disease is caused by mutations in the TPP1 gene resulting in deficient activity of the enzyme tripeptidyl peptidase 1 (TPP1). In the absence of TPP1, lysosomal storage materials normally metabolized by this enzyme accumulate in many organs, particularly in the brain and retina. Buildup of these storage materials in the cells of the nervous system contribute to the progressive and relentless neurodegeneration which manifests as loss of cognitive, motor, and visual functions.
Brineura is a recombinant form of human tripeptidyl peptidase 1 (TPP1), the enzyme deficient in patients with CLN2 disease. It is an enzyme replacement therapy designed to restore TPP1 enzyme activity and break down the storage materials that cause CLN2 disease. In order to reach the cells of the brain and central nervous system, the treatment is delivered directly into the fluid surrounding the brain (cerebrospinal fluid) using BioMarin’s patented technology.
For additional information regarding this product, please contact BioMarin Medical Information at email@example.com.
Brineura® (cerliponase alfa) is a prescription medication used to slow loss of ability to walk or crawl (ambulation) in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.
Important Safety Information
Brineura is a prescription medicine. Before treatment with Brineura, it is important to discuss your child’s medical history with their doctor. Tell the doctor if they are sick or taking any medication and if they are allergic to any medicines. Your child’s doctor will decide if Brineura is right for them. If you have questions or would like more information about Brineura, contact your child’s doctor.
Brineura is only given by infusion into the fluid of the brain (known as an intraventricular injection) and using sterile technique to reduce the risk of infection. An intraventricular access device or port must be in place at least 5 to 7 days prior to the first infusion. Intraventricular access device-related infections were observed with Brineura treatment. If any signs of infection occur, contact your child’s doctor immediately. Your child’s intraventricular access device may need to be replaced over time.
Brineura should not be used in patients with active intraventricular access device-related complications (e.g., leakage, device failure, or device-related infection) and with shunts used to drain extra fluid around the brain.
Low blood pressure and/or slow heart rate may occur during and following the Brineura infusion. Contact your child’s doctor immediately if these reactions occur.
Undesirable or hypersensitivity reactions related to Brineura treatment, including fever, vomiting, and irritability, may occur during treatment and as late as 24 hours after infusion. Your child may receive medication such as antihistamines before Brineura infusions to reduce the risk of reactions. Serious and severe allergic reactions (anaphylaxis) may occur. If a reaction occurs, the infusion will be stopped and your child may be given additional medication. If a severe reaction occurs, the infusion will be stopped and your child will receive appropriate medical treatment. If any signs of anaphylaxis occur, immediately seek medical care.
Safety and effectiveness in pediatric patients below 3 years of age have not been established.
The most common side effects reported during Brineura infusions included fever, problems with the electrical activity of the heart, decreased or increased protein in the fluid of the brain, vomiting, seizures, hypersensitivity, collection of blood outside of blood vessels (hematoma), headache, irritability, and increased white blood cell count in the fluid of the brain, device-related infection, slow heart rate, feeling jittery, and low blood pressure. Intraventricular device-related side effects included infection, delivery system-related complications, and increased white blood cell count in fluid of the brain.
These are not all of the possible side effects with Brineura. Talk to your child’s doctor if they have any symptoms that bother them or that do not go away.
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Please see accompanying full Prescribing Information, or visit www.Brineura.com.
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. The company’s portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.
For additional information, please visit www.BMRN.com. Information on BioMarin’s website is not incorporated by reference into this press release.
BioMarin® and Brineura® are registered trademarks of BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc.
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SOURCE BioMarin Pharmaceutical Inc.
NORTH CHICAGO, Ill. and SOUTH SAN FRANCISCO, Calif., Oct. 18, 2017 /PRNewswire/ — AbbVie (NYSE: ABBV), a global biopharmaceutical company, and Harpoon Therapeutics, a biotechnology company developing novel T-cell recruiting biologic therapies, announced today that they have entered an immuno-oncology research collaboration. The goal of the collaboration is to incorporate Harpoon’s tri-specific T-cell activating construct (TriTAC™) platform with AbbVie’s research-stage immuno-oncology targets to develop novel cancer therapeutics.
Under the terms of the agreement, Harpoon will engineer TriTAC molecules directed against selected cancer targets using its proprietary platform, evaluate the molecules for pharmacologic properties, and provide AbbVie the right to pursue further development and commercialization of these molecules. Financial terms were not disclosed.
“This collaboration is the first for Harpoon and highlights the high level of industry interest in best-in-class platform technologies. We are excited about partnering with AbbVie to help generate novel T-cell engagers for the treatment of cancer based on the combination of T-cell receptors with TriTACs,” said Jerry McMahon, Ph.D., chief executive officer, Harpoon Therapeutics.
“T-cell therapies represent the next wave of innovation in cancer treatment,” said Tom Hudson, M.D., vice president, oncology early discovery and development, AbbVie. “Harpoon’s technology offers the potential for a unique approach to engage the immune system through AbbVie’s investigational therapies in development.”
About Harpoon Therapeutics
Harpoon Therapeutics is an immuno-oncology company founded by Patrick Baeuerle, Ph.D., a pioneer in the development of T-cell engaging therapies, and MPM Capital. The company is focused on the discovery and development of novel T-cell engaging biologics for the treatment of cancer and other diseases. Harpoon Therapeutics created a novel T-cell recruiting drug discovery platform called TriTAC™ (tri-specific T-cell activating construct) to unleash the targeted cell-killing properties of a patient’s own immune system through T-cell activation. This approach is being optimized to penetrate tissues and extend serum exposure, and has the potential to address a broad range of cancers, including solid tumors, and immunologic diseases. HPN424, a prostate-specific membrane antigen (PSMA)-targeting TriTAC™ biologic, is in development for the treatment of prostate cancer and is expected to enter a clinical trial in 2018. For more information, please visit www.harpoontx.com.
AbbVie is a global, research-driven biopharmaceutical company committed to developing innovative advanced therapies for some of the world’s most complex and critical conditions. The company’s mission is to use its expertise, dedicated people and unique approach to innovation to markedly improve treatments across four primary therapeutic areas: immunology, oncology, virology and neuroscience. In more than 75 countries, AbbVie employees are working every day to advance health solutions for people around the world. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook or LinkedIn.
AbbVie Forward-Looking Statements
Some statements in this news release may be forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words “believe,” “expect,” “anticipate,” “project” and similar expressions, among others, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those indicated in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, and changes to laws and regulations applicable to our industry.
Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie’s operations is set forth in Item 1A, “Risk Factors,” in AbbVie’s 2016 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission. AbbVie undertakes no obligation to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law.
Biocept Launches Pathology Partnership Initiative Expanding Access of Proprietary Liquid Biopsy Testing to Community Pathologists and Hospitals
First and only platform that enables local pathologists to obtain molecular biomarker information from a simple blood sample using cutting-edge circulating tumor cell (CTC) technology
SAN DIEGO, Oct. 18, 2017 /PRNewswire/ — Biocept, Inc. (NASDAQ: BIOC), a leading commercial provider of liquid testing designed to provide physicians with clinically actionable information to improve the outcomes of cancer patients, announces the launch of its molecular pathology partnership initiative, the aim of which is to incorporate community pathologists into the review of biomarkers found in liquid biopsy for patients diagnosed with cancer. Patient specimens will continue to be sent to Biocept for processing in its CLIA-certified, CAP-accredited laboratory.
“Having pathologists involved in the interpretation of liquid biopsy is important, as community physicians and hospitals want to take advantage of leading-edge medical solutions by providing the best patient care in their local health system,” said Michael Terry, Senior Vice President, Commercial Operations at Biocept. “Combining liquid biopsy and the expertise of the local pathologist can provide better overall continuity of care when it comes to cancer diagnosis and treatment as they are the one of the first to know when a patient could benefit from a liquid biopsy. Our initial roll-out of this program showed strong acceptance by pathologists and clinicians, and we are now beginning to offer this program to additional cancer treatment centers around the country.”
“We believe that local pathologists will value Biocept’s ability to provide them with access to proprietary liquid biopsy technology, and we are looking forward to expanding and building new relationships in hospitals around the country with this program,” said Biocept’s President and Chief Executive Officer Michael Nall. “We continue to execute on expanding our physician services and our menu of non-invasive biomarker tests, in addition to our objective to increase the number of cancer indications addressed by our platform, making Biocept the go-to choice in liquid biopsy.”
Biocept’s Target Selector™ liquid biopsy tests are performed in its CLIA-certified, CAP-accredited laboratory located in San Diego, California. To order a liquid biopsy test or participate in the Pathology Partnership program, please contact Customer Service at 888-332-7729 or firstname.lastname@example.org.
Biocept, Inc. is a molecular diagnostics company with commercialized assays for lung, breast, gastric, colorectal and prostate cancers, and melanoma. The Company leverages its proprietary liquid biopsy technology to provide physicians with clinically actionable information for treating and monitoring patients diagnosed with cancer. Biocept’s patented Target Selector™ liquid biopsy technology platform captures and analyzes tumor-associated molecular markers in both circulating tumor cells (CTCs) and in circulating tumor DNA (ctDNA). With thousands of tests performed, the platform has demonstrated the ability to identify cancer mutations and alterations to inform physicians about a patient’s disease and therapeutic options. For additional information, please visit www.biocept.com.
Forward-Looking Statements Disclaimer Statement
This news release contains forward-looking statements that are based upon current expectations or beliefs, as well as a number of assumptions about future events. Although we believe that the expectations reflected in the forward-looking statements and the assumptions upon which they are based are reasonable, we can give no assurance that such expectations and assumptions will prove to be correct. Forward-looking statements are generally identifiable by the use of words like “may,” “will,” “should,” “could,” “expect,” “anticipate,” “estimate,” “believe,” “intend” or “project,” or the negative of these words or other variations on these words or comparable terminology. To the extent that statements in this news release are not strictly historical, including, without limitation, statements as to our ability to identify specific clinical conditions or improve the outcomes of cancer patients, the utility and effectiveness of our intellectual property protections, the financial impact of new contracts, and our ability to increase the number of products or services provided or the value of the Company, such statements are forward-looking, and are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The reader is cautioned not to put undue reliance on these forward-looking statements, as these statements are subject to numerous risk factors as set forth in our Securities and Exchange Commission (SEC) filings. The effects of such risks and uncertainties could cause actual results to differ materially from the forward-looking statements contained in this news release. We do not plan to update any such forward-looking statements and expressly disclaim any duty to update the information contained in this press release except as required by law. Readers are advised to review our filings with the SEC at www.sec.gov
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SOURCE Biocept, Inc.
Trovagene’s PLK1 Inhibitor (PCM-075) Demonstrates Synergy with Zytiga® (abiraterone acetate) in Castration Resistant Prostate Cancer Tumor Cells
PCM-075 enhances activity of abiraterone in mCRPC tumors cells and may represent a novel treatment option to extend the benefit of anti-androgen therapy
SAN DIEGO, Oct. 18, 2017 /PRNewswire/ — Trovagene, Inc. (NASDAQ: TROV), a precision medicine biotechnology company, today announced positive data from preclinical research demonstrating synergy of PCM-075, its oral, highly-selective Polo-like Kinase 1 (PLK1) Inhibitor, in combination with abiraterone (Zytiga® – Johnson & Johnson). Abiraterone is a potent and irreversible inhibitor of CYP17, a critical enzyme in androgen biosynthesis and is indicated for the treatment of mCRPC in combination with prednisone. This research was performed in collaboration with a major cancer research institution.
“The synergy observed when we combined PCM-075 with abiraterone appears to work through a novel mechanism that may modulate a signaling pathway previously unknown to be associated with this combination,” said Dr. Mark Erlander, Chief Scientific Officer of Trovagene. “This unique combination appears to enhance the PCM-075 mechanism of action of arresting cells during mitosis with subsequent tumor cell death.”
Metastatic Prostate Cancer is the third leading cause of cancer death in men. Approximately 25,000 men progress to metastatic Castration-Resistant Prostate Cancer (mCRPC) annually and receive anti-androgen therapy with a mean progression-free survival of less than two years. Abiraterone is the leading global anti-androgen therapy, marketed by Centocor Ortho Biotech, Inc., a member of the Johnson & Johnson family of companies, with 2016 sales in excess of $2.0 billion. Even with the broad adoption of abiraterone there continues to be a large medical need to extend the benefit of response to abiraterone in mCRPC.
“We are encouraged by the preclinical data of PCM-075 in mCRPC,” said Bill Welch, Chief Executive Officer of Trovagene. “We previously completed a Phase 1 trial in metastatic solid tumor cancers, which provided a recommended Phase 2 dose and dosing schedule for PCM-075 in a combination regimen. We are working closely with key investigators to develop a Phase 2 clinical trial protocol with oral dosing of PCM-075 and abiraterone utilizing our existing solid tumor IND.”
PCM-075 is a highly-selective adenosine triphosphate (ATP) competitive inhibitor of the serine/threonine polo-like-kinase 1 (PLK 1) enzyme, which is over-expressed in multiple hematologic and solid tumor cancers. Studies have shown that inhibition of polo-like-kinases can lead to tumor cell death, including a Phase 2 study in Acute Myeloid Leukemia (AML) where response rates up to 31% were observed when used in conjunction with a standard therapy for AML (low-dose cytarabine-LDAC) versus treatment with LDAC alone with a 13.3% response rate. A Phase 1 open-label, dose escalation safety study of PCM-075 has been completed in patients with advanced metastatic solid tumor cancers, and published in Investigational New Drugs. Trovagene is initiating a Phase 1b/2 clinical trial with PCM-075 in AML that was accepted by the National Library of Medicine (NLM) and is now publicly viewable on www.clinicaltrials.gov. The NCT number assigned by clinicaltrials.gov for this study is NCT03303339. PCM-075 has been granted Orphan Drug Designation by the FDA for the treatment of patients with AML.
PCM-075 only targets PLK1 isoform (not PLK2 or PLK3), is oral, has a 24-hour drug half-life with reversible on-target hematologic toxicities. Trovagene believes that targeting only PLK1 with reversible on-target activity and an improved dose/scheduling protocol can significantly improve on the long-term outcome observed in previous studies with a PLK inhibitor in AML.
PCM-075 has demonstrated synergy in preclinical studies with over 10 chemotherapeutic and target agents used in hematologic and solid tumor cancers, including FLT3 and HDAC inhibitors, taxanes, and cytotoxins. Trovagene believes the combination of its targeted PLK-1 inhibitor, PCM-075, with other compounds has the potential for improved clinical efficacy in Acute Myeloid Leukemia (AML), Castration-Resistant Prostate Cancer (CRPC), Non-Hodgkin Lymphoma (NHL), Triple Negative Breast Cancer (TNBC) and Adrenocortical Carcinoma (ACC).
About Castration-Resistant Prostate Cancer (CRPC)
Castration-Resistant Prostate Cancer (CRPC) is defined by disease progression despite androgen-deprivation therapy (ADT) and may present as one or any combination of a continuous rise in serum levels of prostate-specific antigen (PSA), progression of pre-existing disease, or appearance of new metastases. Prognosis is associated with several factors, including performance status, presence of bone pain, extent of disease on bone scan, and serum levels of alkaline phosphatase. Bone metastases will occur in 90% of men with CPRC and can produce significant morbidity, including pain, pathologic fractures, spinal cord compression, and bone marrow failure. Paraneoplastic effects are also common, including anemia, weight loss, fatigue, hypercoagulability, and increased susceptibility to infection. Institution of treatment and the choice of systemic or local therapy depend on a number of factors.
About Trovagene, Inc.
Trovagene is a precision medicine biotechnology company developing oncology therapeutics for improved cancer care by leveraging its proprietary Precision Cancer Monitoring® (PCM) technology in tumor genomics. Trovagene has broad intellectual property and proprietary technology to measure circulating tumor DNA (ctDNA) in urine and blood to identify and quantify clinically actionable markers for predicting response to cancer therapies. Trovagene offers its PCM technology at its CLIA/CAP – accredited laboratory and plans to continue to vertically integrate its PCM technology with precision cancer therapeutics. For more information, please visit https://www.trovagene.com.
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as “anticipate,” “believe,” “forecast,” “estimated” and “intend” or other similar terms or expressions that concern Trovagene’s expectations, strategy, plans or intentions. These forward-looking statements are based on Trovagene’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, our need for additional financing; our ability to continue as a going concern; clinical trials involve a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive of future trial results; our clinical trials may be suspended or discontinued due to unexpected side effects or other safety risks that could preclude approval of our product candidates; uncertainties of government or third party payer reimbursement; dependence on key personnel; limited experience in marketing and sales; substantial competition; uncertainties of patent protection and litigation; dependence upon third parties; our ability to develop tests, kits and systems and the success of those products; regulatory, financial and business risks related to our international expansion and risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations. There are no guarantees that any of our technology or products will be utilized or prove to be commercially successful, or that Trovagene’s strategy to design its liquid biopsy tests to report on clinically actionable cancer genes will ultimately be successful or result in better reimbursement outcomes. Additionally, there are no guarantees that future clinical trials will be completed or successful or that any precision medicine therapeutics will receive regulatory approval for any indication or prove to be commercially successful. Investors should read the risk factors set forth in Trovagene’s Form 10-K for the year ended December 31, 2016, and other periodic reports filed with the Securities and Exchange Commission. While the list of factors presented here is considered representative, no such list should be considered to be a complete statement of all potential risks and uncertainties. Unlisted factors may present significant additional obstacles to the realization of forward-looking statements. Forward-looking statements included herein are made as of the date hereof, and Trovagene does not undertake any obligation to update publicly such statements to reflect subsequent events or circumstances.
VP, Corporate Communications
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SOURCE Trovagene, Inc.
SAN FRANCISCO, Oct. 18, 2017 /PRNewswire/ — Trianni, Inc. (“TRIANNI”) today announced that Juno Therapeutics (NASDAQ: JUNO) has entered into a license agreement for The Trianni Mouse™, a best-in-class monoclonal antibody discovery platform. Juno intends to use The Trianni Mouse with its proprietary high throughput, single cell sequencing technology and direct-to-CAR screening to rapidly identify fully-human binders for conversion to chimeric antigen receptors in its engineered T cell programs.
“TRIANNI is pleased to bolster Juno’s lead generation capabilities through a license to The Trianni Mouse,” stated Dr. David Meininger, TRIANNI’s Chief Business Officer. “We are confident that Juno’s deep expertise in developing engineered T cells will potently synergize with the industry-leading human antibody repertoires provided by The Trianni Mouse.”
“The Trianni Mouse offers an in vivo platform for generating a diverse human antibody response to tumor targets of interest,” said Hy Levitsky, M.D., Juno’s Chief Scientific Officer.
“We look forward to using the technology to advance our discovery efforts and build more effective, next-generation CAR T cells.”
No financial details were disclosed.
About Trianni, Inc.
Trianni, Inc. is a privately held biotech company specializing in antibody discovery technology. TRIANNI’s lead technology, The Trianni Mouse™, is a powerful, next-generation platform enabling efficient generation of fully- human monoclonal antibodies. TRIANNI’s transgenic platform leverages a novel approach to design made possible by advances in DNA synthesis and genomic modification technology making it a best-in-class therapeutic antibody discovery platform. The company is headquartered in San Francisco, CA. Additional information about TRIANNI is available through its corporate website, www.trianni.com.
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, Section 27A of the Securities Act of 1933, and Section 21E of the Securities Exchange Act of 1934, including statements regarding Juno’s mission, progress, and business plans, and the potential of The Trianni Mouse to advance Juno’s discovery efforts and build more effective, next-generation CAR T cells. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from such forward-looking statements, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to, risks associated with: the success, cost, and timing of Juno’s product development activities and clinical trials; Juno’s ability to obtain regulatory approval for and to commercialize its product candidates; Juno’s ability to establish a commercially-viable manufacturing process and manufacturing infrastructure; regulatory requirements and regulatory developments; success of Juno’s competitors with respect to competing treatments and technologies; Juno’s dependence on third-party collaborators and other contractors in Juno’s research and development activities, including for the conduct of clinical trials and the manufacture of Juno’s product candidates; Juno’s ability to attract and retain key scientific, quality control/assurance, manufacturing or management personnel; Juno’s dependence on Celgene for the development and commercialization outside of North America and China of Juno’s CD19 product candidates and any other product candidates for which Celgene exercises an option; Juno’s dependence on JW Therapeutics (Shanghai) Co., Ltd, over which Juno does not exercise complete control, for the development and commercialization of product candidates in China; Juno’s ability to obtain, maintain, or protect intellectual property rights related to its product candidates; amongst others. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Juno’s business in general, see the information Juno has included it its periodic reports and other documents filed with the Securities and Exchange Commission. These forward-looking statements speak only as of the date hereof. Juno disclaims any obligation to update these forward-looking statements.
Contact– Trianni, Inc.
Chief Business Officer
Director of Marketing
SOURCE Trianni, Inc.
In advance of an anticipated Senate hearing on drug prices, President Donald Trump once again denounced drug companies as “getting away with murder.” The first time he did so, using the…
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Key business optimization features and operational enhancements reduce complexities and speed study startup
SAN FRANCISCO, Oct. 17, 2017 /PRNewswire/ — goBalto, Inc., the leading provider of cloud-based clinical study startup (SSU) solutions, announced today its latest release of goBalto Activate. The third major release of 2017 offers new features that further accelerate clinical study startup.
SSU is an array of activities performed at the outset of studies, including: investigative site selection and initiation, regulatory document submission, contract and budget negotiations, and enrolling the first subject. The process of initiating clinical trials remains unwieldy, challenging, and often behind schedule, making SSU one of the poorest performing aspects of clinical trials. At a time when SSU remains a perpetual bottleneck, leading companies are positioning themselves ahead of the curve by embracing solutions that confront these real challenges, thereby emphasizing differentiation and operational efficiencies.
New APIs and functionality in the release allow for the exchange of alert or task information between Activate and the Shared Investigator Platform (SIP), and other industry site portals.
“The enterprise APIs enable other systems or transformation layers to interact with the application data in much the same way that a user would,” said Sujay Jadhav, goBalto‘s CEO. goBalto‘s proven integration points encompass the eClinical suite and business applications (e.g., CTMs, CRM, eTMF, data warehouses) with key integration components (i.e., data staging, business rule compliance, target data staging and API processing) provided to ensure a seamless and successful integration.
“The integration of Activate with SIP is key to ensuring improved efficiencies across the clinical trial process and compliance with evolving industry standards,” said Jadhav. “Activate workflows aid global regulatory and SOP compliance, accelerating site activation, leading to greater cost savings and faster market entry.”
The Configuration Designer functionality in Activate provides a self-service configuration wizard for workflows, providing the flexibility needed to rapidly respond to dynamic regulatory and SOP changes for studies, currently in-progress or planned.
“The enhancements to the application interface, contract management functionality, and others of this most recent release improve overall usability and ease of adoption,” stated Lorena R. Gomez, Director, Global Study Start Up & Essential Documents, Allergan. “It’s clear that goBalto listens to their customers’ feedback and incorporates it into subsequent enhancements.”
With over 300 pharma and CRO companies benefiting through a networked model using Activate to manage, track, and complete study startup tasks in over 2,000 studies, utilizing over 70,000 sites, in 70+ countries, goBalto has the largest industry-proven set of country workflows and associated performance metrics. Standard ‘out-of-the-box’ country specific regulatory business process workflows allow for quick site activation, leading to greater cost savings and faster market entry, making valuable therapies available to patients sooner.
“Today, our SSU platform is embedded into the business processes of leading life science organizations, that take advantage of new releases to enhance overall value. This release illustrates our commitment to our customers as we enhance interoperability with leading portals, and provide new functionality to tackle entrenched pain points in SSU,” said Jadhav. “This release reinforces goBalto‘s leadership position as we continue to provide next-generation, industry proven study startup solutions, which are key to stemming inefficiencies endemic in industry.”
goBalto is the industry leader in cloud-based study startup software for the global life sciences industry, offering the only complete end-to-end platform for starting clinical trials, from site identification, feasibility assessment and selection through to activation, with comprehensive metrics to track adherence to timelines and budget. Committed to accelerating clinical trials through innovation, product excellence, and customer success, goBalto works with four of the top five CROs and more than two-thirds of the top 25 pharmas. Our customers include: Allergan, Covance, CMIC HOLDINGS, Genentech Roche, ICON, INC Research, Novartis, and PSI CRO. goBalto is headquartered in San Francisco, with offices in Philadelphia and Singapore. For more information, visit www.gobalto.com.
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SOURCE goBalto, Inc.
Tocagen to Present Updated Durable Response Data from Phase 1 Trial of Toca 511 & Toca FC in Recurrent Brain Cancer at the 2017 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics
Data selected for inclusion in official press program of AACR-NCI-EORTC on October 27, 2017
SAN DIEGO, Oct. 17, 2017 /PRNewswire/ — Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, today announced that updated data from the company’s Phase 1 study of Toca 511 & Toca FC in recurrent high grade glioma (brain cancer), have been selected for an oral presentation at the annual International Conference on Molecular Targets and Cancer Therapeutics of the American Association for Cancer Research (AACR), the National Cancer Institute, and the European Organisation for Research and Treatment of Cancer (EORTC), or AACR-NCI-EORTC, held Oct. 26-30 in Philadelphia. Clinical data demonstrating durable responses have been selected for inclusion in the conference’s official press program. Additional updated Phase 1 data and preclinical results, as well as previously disclosed data, will also be presented.
Details of the AACR-NCI-EORTC presentations are as follows:
Presentation Type: Oral (Abstract: A085; selected for inclusion in the official press program of AACR-NCI-EORTC.)
Title: Durable responses observed in recurrent high-grade glioma (rHGG) with Toca 511 and Toca FC treatment
Presenter: Clark Chen, M.D., Ph.D., Lyle French Chair in Neurosurgery and head of the University of Minnesota Medical School Department of Neurosurgery
Date and Time: Friday, Oct. 27, 12:10 p.m.-12:20 p.m. ET (Note: Webcast available Nov. 6.)
Presentation Type: Poster (Abstract: A085; selected for inclusion in the official press program of AACR-NCI-EORTC.)
Title: Durable responses observed in recurrent high-grade glioma (rHGG) with Toca 511 and Toca FC treatment
Presenter: Asha Das, M.D., senior vice president and chief medical officer at Tocagen
Date and Time: Saturday, Oct. 28, 12:30 p.m.-4:00 p.m. ET
Presentation Type: Poster (Abstract: B010)
Title: Anti-tumor cellular immune response elicited by Toca 511 and Toca FC therapy in preclinical and clinical studies
Presenter: Tiffany Montellano, Ph.D., medical science liaison at Tocagen
Date and Time: Sunday, Oct. 29, 12:30 p.m.-4:00 p.m. ET
About Toca 511 & Toca FC
Tocagen’s lead product candidate is a cancer-selective immunotherapy comprised of an investigational biologic, Toca 511, and an investigational small molecule, Toca FC, that are designed to be used together. Toca 511 is an injectable retroviral replicating vector (RRV) that encodes a prodrug activator enzyme, cytosine deaminase (CD). CD is derived from yeast, and humans do not naturally have this gene. Its selective delivery to cancer cells means that the infected cancer cells selectively carry the CD gene and produce CD. Toca FC is an investigational orally administered prodrug, 5-fluorocytosine (5-FC) that is inactive as an anti-cancer drug. In animal models, Tocagen has shown that 5-FC is converted into the anticancer drug, 5-FU, at high concentrations in Toca 511-infected cancer cells that are producing CD. Together, the Toca 511 & Toca FC combination directly kills cancer cells and immune-suppressive myeloid cells resulting in activation of the immune system against the cancer.
Tocagen is a clinical-stage, cancer-selective gene therapy company developing first-in-class, broadly applicable product candidates designed to activate a patient’s immune system against their own cancer. Tocagen is developing its lead investigational product candidate, Toca 511 & Toca FC, initially for the treatment of recurrent high grade glioma (HGG), a disease with significant unmet medical need. The U.S. Food and Drug Administration (FDA) granted Toca 511 & Toca FC Breakthrough Therapy Designation for the treatment of recurrent HGG and the European Medicines Agency (EMA) granted Toca 511 PRIME (PRIority MEdicines) designation for the treatment of HGG.
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding our business plans and objectives, expectations regarding our cash position, timing and success of our clinical trials and planned clinical trials, timing of results from our clinical trials, timing of updates from communications with the FDA and our plans regarding selection of additional product candidates. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost and timing of our product candidate development activities and planned clinical trials; our ability to execute on our strategy; regulatory developments in the United States and foreign countries; and our estimates regarding expenses, future revenue and capital requirements. These and other risks and uncertainties are described more fully under the caption “Risk Factors” and elsewhere in Tocagen’s filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Tocagen undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
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SOURCE Tocagen Inc.
Forge & Evotec Expand Strategic Alliance With Launch Of BLACKSMITH Platform To Discover Novel Metalloenzyme Inhibitors Across Broad Therapeutic Areas
– BLACKSMITH Platform Initially Focused on the Development of Novel Classes of Antibiotics — Expansion Highlights Significance of US-UK Biotech Partnerships –
SAN DIEGO, Oct. 17, 2017 /PRNewswire/ — Forge Therapeutics, Inc. (Forge) announced today that the companies have expanded their existing strategic alliance and launched the BLACKSMITH platform to discover a broader range of therapeutics targeting metalloenzymes.
“Over the last two years, we have experienced the power of Forge’s discovery capabilities with the discovery of the first novel antibiotic class targeting LpxC and we are pleased to have expanded our strategic alliance with the launch of the BLACKSMITH platform,” said Mario Polywka, Chief Operating Officer of Evotec. “Evotec is focused on investing in companies and technologies that can truly advance the field of drug development – our continued relationship with Forge opens many opportunities to rapidly develop new therapies in almost any therapeutic indication where metalloenzymes are critical to disease advancement.”
“All six major enzyme classes contain a significant amount of metalloenzymes and, in fact, over 30 percent of all known enzymes across all species are metalloenzymes,” Prof. Seth Cohen, UCSD and co-founder of Forge. “Despite the rich target space for drug development there are, however, only a few examples of clinically approved metalloenzyme inhibitors as medicines which we believe is due to a shortcoming in the chemical space employed to bind the active site metal ion.”
David Puerta, COO of Forge, added, “Distinct from traditional approaches for ‘hit’ discovery using high throughput screens, the Forge approach starts with metal-ligand interactions to identify selective metal-binding fragment pharmacophores, or MBPs, from a proprietary library of greater than 500 MBPs. Intelligently selected fragments result in greater and more effective chemical diversity to rapidly identify key interactions between a fragment and the metalloenzyme active site. Using bioinorganic and medicinal chemistry principles, Forge transforms these MBP fragments into therapeutic leads using a novel fragment growth strategy incorporating our computational chemistry and structural biology.”
Zachary A. Zimmerman, Ph.D., CEO of Forge, commented, “The time for collaborative work in our industry is now and we are pleased to expand our relationship with Evotec to discover additional, novel metalloenzyme inhibitors against a wide range of diseases with our proprietary technology. Through this expanded relationship, we gain valuable resources that will accelerate our discovery engine to advance multiple therapeutic candidates toward the clinic.”
BLACKSMITH Platform & Strategy
Forge’s platform called BLACKSMITH comprises a deep knowledge of metalloenzymes, bioinorganic and medicinal chemistry know-how, and a focused library of proprietary metal-binding fragment pharmacophores (MBPs) that provide selective & diverse starting points for novel inhibitors. Our strategy is to use the BLACKSMITH platform to discover new chemistry for the treatment of a broad range of diseases in areas of unmet needs with initial efforts in the area of infectious disease. To date, Forge has performed over 50 metalloenzyme screens with library hit rates of >15%, providing multiple starting points to build potent selective inhibitors of metalloenzymes across a variety of therapeutic areas.
About Forge Therapeutics
At Forge Therapeutics, we are developing medicines targeting metal-dependent enzymes found in nature. Over 30% of known enzymes are metalloenzymes covering all major enzymes classes: oxidoreductases, transferases, hydrolases, lyases, isomerases, and ligases. Metal ions, including magnesium, zinc, iron, manganese, calcium, cobalt, and copper are the essential ingredient in these metalloenzymes. At Forge, we are the blacksmiths of modern medicine, providing the tools to address any metalloenzyme challenge.
Forge’s lead effort is focused on LpxC, a zinc metalloenzyme found only in Gram-negative bacteria and which is essential for bacteria to grow. Forge has a strategic alliance with leading drug discovery alliance and development partnership company Evotec AG and has been awarded multiple government awards including CARB-X. In addition, Forge has amassed a rich intellectual property estate on metalloprotein inhibitors to protect its BLACKSMITH platform and pipeline including technology licensed from UCSD. For further information, please visit the company’s website www.ForgeTherapeutics.com and follow us on Twitter @ForgeThera.
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SOURCE Forge Therapeutics, Inc.
PASADENA, Calif., Oct. 16, 2017 /PRNewswire/ — Episona Inc., an epigenetics information company focused on reproductive health, announced today that CEO Alan Horsager, PhD, will provide a corporate presentation at the 2017 BIO Investor Forum taking place in San Francisco Oct. 17-18 at the Westin St. Francis Hotel. The presentation will take place on Wednesday, Oct. 18 at 9:30 a.m. in presentation room Elizabethan D.
About Episona Inc.
Episona is an epigenetics information company focused on improving reproductive health outcomes. The company’s first commercial product, Seed, evaluates epigenetic changes on DNA to predict the risk of male factor fertility and embryo quality. Epigenetics is the study of the environmental and external modifications to DNA that alter gene expression without changing the DNA sequence. The company was founded in 2013 and is based in Pasadena, CA.
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SOURCE Episona Inc.