News Article | May 24, 2017
The company also announced that Dr. Nick Glover, President and Chief Executive Officer of Sierra Oncology, will present an overview of the company entitled "Beyond PARP – Next Generation DDR Therapeutics" at the Jefferies Healthcare Conference in New York. The presentation is scheduled for 2:00pm ET on June 8th. A live audio webcast and archive of the presentation will be accessible through www.sierraoncology.com. Title: A phase I study of SRA737 (formerly known as CCT245737) administered orally in patients with advanced cancer. Trials in Progress Abstract: #TPS2607 Poster: #93b Poster Session: Developmental Therapeutics — Clinical Pharmacology and Experimental Therapeutics Date and Time: Monday, June 5, 2017, 8:00 – 11:30am CT Location: McCormick Place, Event room: Hall A, 2301 S King Dr., Chicago, Illinois Title: A phase I study of oral SRA737 (formerly CCT245737) given in combination with gemcitabine plus cisplatin or gemcitabine alone in patients with advanced cancer. Trials in Progress Abstract: #TPS2613 Poster: #96b Poster Session: Developmental Therapeutics — Clinical Pharmacology and Experimental Therapeutics Date and Time: Monday, June 5, 2017, 8:00 – 11:30am CT Location: McCormick Place, Event room: Hall A, 2301 S King Dr., Chicago, Illinois The posters will be available on June 5, 2017 on the company's website at www.sierraoncology.com Title: Beyond PARP – Next Generation DDR Therapeutics Date and Time: June 8, 2017, 2:00 pm ET Live audio webcast and archive of the presentation will be accessible through www.sierraoncology.com. About Sierra Oncology Sierra Oncology is a clinical stage drug development company advancing next generation DNA Damage Response (DDR) therapeutics for the treatment of patients with cancer. Our lead drug candidate, SRA737, is a potent, highly selective, orally bioavailable small molecule inhibitor of Checkpoint kinase 1 (Chk1), a key regulator of important cell cycle checkpoints and central mediator of the DDR network. In cancer cells, replication stress induced by oncogenes (e.g., MYC or RAS) or genetic mutations in DNA repair machinery (e.g., BRCA1 or FA) combined with loss of function in tumor suppressors (e.g., TP53 or ATM) results in persistent DNA damage and genomic instability leading to an increased dependency on Chk1 for survival. Targeted inhibition by SRA737 may therefore be synthetically lethal to these cancer cells and have utility as a monotherapy in a range of tumor indications. Chk1 is also believed to facilitate tumor cell resistance to chemotherapy or radiation-induced DNA damage and the combination of SRA737 with these standards-of-care may provide synergistic anti-tumor activity. SRA737 is currently being investigated in two Phase 1 clinical trials in patients with advanced cancer. Sierra Oncology is also advancing SRA141, a potent, selective, orally bioavailable small molecule inhibitor of Cell division cycle 7 kinase (Cdc7) undergoing preclinical development. Cdc7 is a key regulator of DNA replication and is involved in the DDR network, making it a compelling emerging target for the potential treatment of a broad range of tumor types. For more information, please visit www.sierraoncology.com. Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Sierra Oncology's anticipated clinical development, protocol amendments, target indications, trial designs and potential benefits of Sierra Oncology's product candidates. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. These statements are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and assumptions that could cause actual results to differ materially from those described in the forward-looking statements. Such forward-looking statements are subject to risks and uncertainties, including, among others, the risk that Sierra Oncology may be unable to successfully develop and commercialize product candidates, SRA737 and SRA141 are at early stages of development and may not demonstrate safety and efficacy or otherwise produce positive results, Sierra Oncology may experience delays in the preclinical and anticipated clinical development of SRA737 or SRA141, Sierra Oncology may be unable to acquire additional assets to build a pipeline of additional product candidates, Sierra Oncology's third-party manufacturers may cause its supply of materials to become limited or interrupted or fail to be of satisfactory quantity or quality, Sierra Oncology's cash resources may be insufficient to fund its current operating plans and it may be unable to raise additional capital when needed, Sierra Oncology may be unable to obtain and enforce intellectual property protection for its technologies and product candidates and the other factors described under the heading "Risk Factors" set forth in Sierra Oncology's filings with the Securities and Exchange Commission from time to time. Sierra Oncology undertakes no obligation to update the forward-looking statements contained herein or to reflect events or circumstances occurring after the date hereof, other than as may be required by applicable law.
News Article | May 9, 2017
"I am very pleased with our continued success in advancing the Nektar pipeline, driven by our expanding research in immuno-oncology and immunology that continues to generate highly valuable new clinical candidates," said Howard W. Robin, President and Chief Executive Officer of Nektar. "In March, we announced overwhelmingly positive efficacy and safety results from our Phase 3 study of NKTR-181 in patients with chronic low back pain. Our Phase 1/2 study evaluating NKTR-214 as a combination regimen with Opdivo® in collaboration with Bristol-Myers Squibb is advancing and we look forward to reporting initial data from the first patients in this trial at ASCO. In Q1, we also initiated a first-in-human trial for NKTR-358, our proprietary Treg stimulator, which has the potential to become a first-in-class resolution therapeutic for a wide range of immune-mediated disorders. We plan to report the results from this trial at a medical meeting in the second half of 2017." Revenue for the first quarter of 2017 was $24.7 million as compared to $58.9 million in the first quarter of 2016. Revenue in the first quarter of 2016 was higher primarily because of the recognition of $28.0 million received from AstraZeneca for the sublicense of MOVENTIG® to Kirin in Europe. In addition, product sales were $4.8 million in the first quarter of 2017 as compared to $14.1 million in the first quarter of 2016. Total operating costs and expenses for the first quarter of 2017 were $79.2 million as compared to $68.4 million in the first quarter of 2016. Total operating costs and expenses increased primarily as a result of higher research and development (R&D) expense in the first quarter of 2017. R&D expense in the first quarter of 2017 was $61.1 million as compared to $49.3 million for the first quarter of 2016 and was higher in the first quarter of 2017 primarily due to expenses for our NKTR-214 and NKTR-358 programs. General and administrative expense was $12.0 million in the first quarter of 2017 as compared to $10.2 million in the first quarter of 2016. In the first quarter of 2017, net loss was $63.9 million, or $0.42 loss per share as compared to net loss of $19.5 million, or $0.14 loss per share in the first quarter of 2016. The loss was higher year over year primarily because of the recognition of $28.0 million received from AstraZeneca for the sublicense of MOVENTIG® to Kirin in Europe in the first quarter of 2016. The company also announced upcoming presentations at the following scientific congresses during the second quarter of 2017: Oral Presentation: "NKTR-255: Accessing The Immunotherapeutic Potential of IL-15" Presenter: Jonathan Zalevsky, Ph.D. Session: Pre-clinical Immuno-Oncology Date and Time: May 15, 2017 – 2:20 p.m. - 2:50 p.m. BST Key Note Address: "NKTR-181: Separating Analgesia from Euphoria in a Novel Opioid Agonist for Chronic Pain" Presenter: Stephen Doberstein, Ph.D. Session: Opioid Addiction Date and Time: May 23, 2017 – 9:50 a.m. - 10:30 a.m. BST Abstract 2545/Poster 37: "Effect of a novel IL-2 cytokine immune agonist (NKTR-214) on proliferating CD8+T cells and PD-1 expression on immune cells in the tumor microenvironment in patients with prior checkpoint therapy." Bernatchez, C., et al. Poster Session: Developmental Therapeutics—Clinical Pharmacology and Experimental Therapeutics Date and Time: June 5, 2017 – 8:00 a.m. - 11:30 a.m. CDT Location: Hall A Abstract TPS1120/Poster 105a: "ATTAIN: Phase 3 study of etirinotecan pegol (EP) vs treatment of physician's choice (TPC) in patients (pts) with metastatic breast cancer (MBC) who have stable brain metastases (BM) previously treated with an anthracycline, a taxane, and capecitabine (ATC)." Tripathy, D., et al. Poster session: Breast Cancer – Metastatic Date and Time: June 4, 2017 – 8:00 a.m. - 11:30 a.m. CDT Location: Hall A Abstract e14040: "A phase 1/2 study of a novel IL-2 cytokine, NKTR-214, and nivolumab in patients with select locally advanced or metastatic solid tumors." Diab, A., et al. Publication abstract to be included online in the 2017 ASCO Annual Meeting Proceedings, a Journal of Clinical Oncology supplement. 2017 International Conference on Opioids (ICOO 2017), Boston, MA Poster 31: "NKTR-181 Produces Full CNS µ-Opioid Agonism With Significantly Lower Abuse Potential": Odinecs, A., et al. Poster session: Session 2 Date and Time: Monday, June 12, 2017 – 8:00 a.m. - 6:00 p.m. EDT Oral Presentation: "NKTR-214 Plus NKTR-262, a Scientifically-Guided Rational Combination Approach for Immune Oncology" Presenter: Jonathan Zalevsky, Ph.D. Session: Rational Combination Immunotherapy Date and Time: June 15, 2017 – 12:00 p.m. EDT Nektar will host an analyst and investor event with clinical investigators during the 2017 American Society of Clinical Oncology (ASCO) Meeting in Chicago. The program will include a presentation and discussion of updated clinical data for the company's CD122-biased agonist, NKTR-214. Data from two studies of NKTR-214 will be reviewed at the event, including the Phase 1 dose-escalation study of NKTR-214 in combination with nivolumab in patients with melanoma, renal cell carcinoma and non-small cell lung cancer (PIVOT-02); and the Phase 1 study of monotherapy NKTR-214 in patients with advanced solid tumors (EXCEL). Presenters will include Dr. Adi Diab, Assistant Professor, Melanoma Medical Oncology at the University of Texas MD Anderson Cancer Center, Dr. Nizar Tannir, Professor, Genitourinary Medical Oncology at the University of Texas MD Anderson Cancer Center and Dr. Michael Hurwitz, Assistant Professor of Medicine (Medical Oncology) at Yale Cancer Center. Conference Call to Discuss First Quarter 2017 Financial Results Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time today, Tuesday, May 9, 2017. This press release and a live audio-only webcast of the conference call can be accessed through a link that is posted on the home page and Investors section of the Nektar website: http://www.nektar.com. The web broadcast of the conference call will be available for replay through June 12, 2017. To access the conference call, follow these instructions: In the event that any non-GAAP financial measure is discussed on the conference call that is not described in the press release, or explained on the conference call, related information will be made available on the Investors page at the Nektar website as soon as practical after the conclusion of the conference call. Nektar Therapeutics is a research-based biopharmaceutical company whose mission is to discover and develop innovative medicines to address the unmet medical needs of patients. Our R&D pipeline of new investigational medicines includes treatments for cancer, auto-immune disease and chronic pain. We leverage Nektar's proprietary and proven chemistry platform in the discovery and design of our new therapeutic candidates. Nektar is headquartered in San Francisco, California, with additional operations in Huntsville, Alabama and Hyderabad, India. Further information about the company and its drug development programs and capabilities may be found online at http://www.nektar.com. This press release contains uncertain or forward-looking statements which can be identified by words such as: "anticipate," "intend," "plan," "expect," "believe," "should," "may," "will" and similar references to future periods. Examples of forward-looking statements include, among others, statements we make regarding the potential therapeutic benefits of NKTR-181, the risks of opioid abuse resulting from pain medicines, future development plans for NKTR-181, the availability of data for NKTR-214 in combination with Opdivo, clinical development plans for our products (including NKTR-358), availability of future clinical results, the timing of planned regulatory filings, the potential of NKTR-214 in combination with other immunotherapy agents, and the potential of our research and development pipeline. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on our current beliefs, expectations and assumptions and are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of our control. Our actual results may differ materially from those indicated in the forward-looking statements and you should not rely on such statements. Important factors that could cause our actual results to differ materially from those indicated in the forward-looking statements include: (i) clinical study outcomes remain very unpredictable and it is possible that a clinical study could fail; (ii) the regulatory pathway to review and approve NKTR-181 for use in patients is subject to substantial uncertainty; (iii) regulations concerning access to opioid-based pharmaceuticals are strict and there is no guarantee which scheduling category will apply to NKTR-181 if regulatory approval is achieved; (iv) the CHMP and FDA have substantial discretion as to whether to grant marketing approval for pharmaceutical products; (v) our drug candidates and those of our collaboration partners are in various stages of clinical development and the risk of failure is high and can unexpectedly occur at any stage prior to regulatory approval for numerous reasons including negative safety and efficacy findings even after positive findings in previous preclinical and clinical studies; (vi) the commencement or end of clinical trials and the availability of clinical data may be delayed or unsuccessful; (vii) patents may not issue from our patent applications for our drug candidates, patents that have issued may not be enforceable, or additional intellectual property licenses from third parties may be required; and (viii) certain other important risks and uncertainties set forth in our Annual Report on Form 10-K for the year ended December 31, 2016 filed with the Securities and Exchange Commission on March 1, 2017. Any forward-looking statement made by us in this press release is based only on information currently available to us and speaks only as of the date on which it is made. We undertake no obligation to update any forward-looking statement. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/nektar-therapeutics-reports-financial-results-for-the-first-quarter-of-2017-300454595.html
News Article | May 5, 2017
Although pain, memory and nicotine addiction may not seem to be related, they actually share a common player: the nicotinic acetylcholine receptors. That’s why Texas A&M researchers are working to develop drugs to enhance the function of these receptors in the brain, which could have three very different applications: easing pain, slowing the cognitive decline associated with Alzheimer’s and other neuropsychiatric diseases and making it easier for people to stop smoking. All of these functions—and many more, from muscle contraction to modulation of sleep, attention, learning and memory—involve nicotinic acetylcholine receptors, which come in different varieties that determine their role. “Based on their location and subunit composition, nicotinic acetylcholine receptors have different functions, biophysical properties and pharmacological characteristics,” said Ayman K. Hamouda, BPharm, PhD, assistant professor in the Department of Pharmaceutical Sciences at the Texas A&M College of Pharmacy and the Department of Neuroscience & Experimental Therapeutics at the Texas A&M College of Medicine. “We’re trying to develop drugs to modulate those functions, but in a far more selective way than anything currently on the market.” It became relatively easy to develop what is called a nicotinic acetylcholine agonist, which is a compound that binds to and activates a receptor. Years of research have yielded a pretty good idea of the structure of the binding sites of acetylcholine and its agonists and what an agonist should look like. The problem with this approach is that the agonist binding sites in all nicotinic acetylcholine receptors are evolutionary conserved to bind acetylcholine. So, using an agonist that is similar to acetylcholine will activate multiple nicotinic acetylcholine receptor types and multiple side effects are very likely to occur. As everyone knows, nicotine is a highly addictive substance, which is why it is so difficult to quit smoking. Other than nicotine replacement therapy like nicotine patches or gum, the drug varenicline is the only nicotinic agonist that is approved by the Food and Drug Administration and clinically available in United States. However, it is hindered by low success rates (22 percent of people, at best, aren’t smoking a year later) and serious side effects like depression and suicidal thoughts occur fairly often. This is because available agonists bind at different nicotine receptors. Some of these interactions are good, because they help someone to quit smoking, but others cause these side effects. “If we can replace nicotine with a more selective and safer drug that’s not addictive, that would be great,” Hamouda said. Hamouda’s approach is to develop positive allosteric modulators, which enhance acetylcholine-mediated effects by binding at sites distinct from the acetylcholine binding sites. In this way, it will enhance, but not replace, acetylcholine in the brain. In other words, it will have a booster effect on the receptor, like a tool that could fit in one specific place in a car and encourage the driver—acetylcholine in this case—to drive faster. However, unlike other types of drugs that actually replace the driver, this metaphorical car will never move unless acetylcholine is also present to act as the driver. “We think our approach will be safer because we’re not changing the pattern of neural activity,” Hamouda said. “Instead, we’re changing the extent of neural activity so that the same signal that the brain designed is now at a higher level.” However, it is much more difficult to develop a clinically relevant positive allosteric modulator. “We don’t have a perfect 3-D structure of the human nicotinic receptors, nor do we know the number and location of allosteric binding sites,” he added. “Thus, it is unfeasible to design positive allosteric modulators a priori.” To accomplish this, Hamouda and his team use site-directed mutagenesis to substitute specific amino acid residues within a nicotinic acetylcholine receptor structure. They then evaluate the effect of these changes on the interaction of nicotinic receptor and positive allosteric modulators. In research published this week in the Journal of Biological Chemistry, using this approach along with computational analyses, Hamouda and his team identified a novel binding site for positive allosteric modulators that is unique to a small subpopulation of nicotinic acetylcholine receptors. “This study provides structural information that will make our mission to find a drug that bind to a less-conserved part of nicotinic receptors easier,” said Farah Deba, PhD, a postdoctoral research associate and a co-author in this study. Now that the team has a good starting point and a site to target, they have started using structure-based drug design to build novel compounds and teaming up with Hamed Aly-Ismail, BPharm, PhD, a synthetic chemist and assistant professor of pharmaceutical sciences at the College of Pharmacy, to synthesize these compounds. One often-overlooked property of nicotine is its ability to enhance memory. In fact, loss of cholinergic neurons and decrease in the number of brain nicotinic acetylcholine receptors are among the first deficits characteristic of Alzheimer’s disease. “The drugs that we are trying to develop will make the remaining receptors work harder, which should help enhance memory in people with less-than-optimal levels of acetylcholine receptors,” Hamouda said. A third possible application for a drug that acts on nicotinic acetylcholine receptors is alleviating pain while avoiding the abuse potential associated with opioid analgesics. “We are examining the anti-nociceptive effects of a naturally occurring positive allosteric modulator of a particular nicotinic acetylcholine receptor in an animal model of acute pain,” Deba said. “The results are still preliminary but very promising.” “We think this drug might also help with opioid abuse, if it can indeed treat pain without any addictive qualities,” Hamouda said. “I’m a pharmacist, so I want to develop drugs to bind to these receptors,” Hamouda added. “We’re not there yet; there is a lot to be done to understand the in vitro and in vivo pharmacology of these compounds, but we’re closer than ever to this goal.”
News Article | February 15, 2017
Physicians’ Education Resource® (PER®), will host the 13th Annual International Symposium on Melanoma and Other Cutaneous Malignancies® on Feb. 11 at Trump International Miami. The program will be led by Co-Chairs Dr. Jeffrey S. Weber, deputy director of the Laura and Issac Perlmutter Cancer Center and professor of medicine at NYU’s Langone Medical Center, and Dr. Omid Hamid, chief of translational and immuno-oncology and director of cutaneous malignancies at the Angeles Clinic and Research Institute and Director of Experimental Therapeutics at Cedars Sinai Medical Care Foundation in Los Angeles. In making the announcement, Dr. Jeffery S. Weber said: “We have just begun to scratch the surface in the process of developing the innovative techniques and therapies that we can use to fight cutaneous malignancies. Interactive forums like the current one allow those on the front lines to collaborate in order to propel the right initiatives forward to improve patient care.” The one-day educational meeting’s focal point will be on contemporary approaches and the future of direction of therapy in the management of melanoma and other cutaneous malignancies, and contains a fast-paced forum on key core areas of personalizing care for melanoma tumors including immunotherapies, targeted therapies, regional therapy, predictive/prognostic modeling, and integrated medicine/patient care. The symposium will be held at the Trump International Miami on Feb. 11 in Sunny Isles Beach, Florida, located 3.5 miles from the Miami International Airport and 13 miles from the Fort Lauderdale International Airport. For more information and to register please visit: http://www.gotoper.com/conferences/ime/meetings/13th-Annual-International-Symposium-on-Melanoma-and-Other-Cutaneous-Malignancies/registration About PER® Since 1995, PER has been the educational resource of choice for live and online activities focusing on oncology and hematology. PER provides high-quality, evidence-based activities featuring leading national and international faculty with a focus on practice-changing advances and standards of care in treatment and disease management. Activities also include topics on emerging strategies currently under investigation, supportive care, diagnosis and staging, prevention, screening and early detection, and practice management. With the rapid advances occurring in the field of oncology, understanding how to use molecular data to diagnose and stage patients, selecting the most appropriate candidates for novel therapeutic agents, individualizing treatment based on tumor type, and referring patients to clinical trials will continue to ensure the highest level of patient care is provided. PER serves the oncology healthcare community, including physicians, fellows, advanced practice nurses, nurses, physician assistants, pharmacists, and researchers. PER is part of the Cranbury, N.J.-based Michael J. Hennessy Associates, Inc. family of businesses. Learn more at http://www.gotoper.com and http://www.mjhassoc.com
News Article | February 16, 2017
Join us in Chicago or register for virtual newsroom access BETHESDA, Md., Feb. 16, 2017 - World-renowned scientists will present pioneering research and discuss key issues affecting the life sciences at the 2017 Experimental Biology meeting (EB 2017), the premier annual meeting of six scientific societies in Chicago to be held April 22-26. Register for a free onsite press pass to see these speakers in person. Or, stay up to date on all the exciting research news at EB 2017 through the new EB Virtual Newsroom, your one-stop shop for press releases, meeting information and blog posts. EB 2017 will feature the latest advances in anatomy, biochemistry and molecular biology, investigative pathology, nutrition, pharmacology and physiology. This year's speaker line-up includes presentations from the following leading scientists: Want to hear these speakers and follow hundreds of other research announcements at EB 2017? Here are your next steps: Learn More about the Virtual Newsroom: Get press releases, multimedia & news tips online View the Preliminary Program: Get the latest information on planned scientific sessions & events EB sponsoring societies include the American Association of Anatomists, American Physiological Society, American Society for Biochemistry and Molecular Biology, American Society for Investigative Pathology, American Society for Nutrition and American Society for Pharmacology & Experimental Therapeutics.
News Article | February 15, 2017
SOUTH SAN FRANCISCO, CA--(Marketwired - February 13, 2017) - VistaGen Therapeutics Inc. ( : VTGN), a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders, today reported financial results for the third quarter of fiscal 2017 ended December 31, 2016. The Company also provided a corporate update, including anticipated milestones for AV-101, its new generation, orally available CNS prodrug candidate in Phase 2 development, initially for the adjunctive treatment of major depressive disorder (MDD) in patients with an inadequate response to standard antidepressant therapies approved by the U.S. Food and Drug Administration (FDA). "We are excited about our progress during the last quarter, with several key advances related to our MDD-focused programs for AV-101, as well as potential regenerative medicine and drug rescue applications of our cardiac stem cell technology. Following productive discussions with the FDA last quarter, our team and key advisors have been working diligently to complete the diverse regulatory and technical activities necessary to support the planned launch of our Phase 2b study of AV-101 next quarter, a study we believe has game-changing potential for the millions of patients who battle MDD every day with inadequate therapies," commented Shawn Singh, Chief Executive Officer of VistaGen. "Also, our recent sublicense agreement with BlueRock Therapeutics was an important advance in our cardiac stem cell program while we remain primarily focused on our Phase 2 programs for AV-101. With potentially catalytic milestones in the coming quarters, we believe we are poised to unlock significant value for our shareholders throughout 2017," added Mr. Singh. The U.S. National Institute of Mental Health (NIMH) is currently conducting and fully funding a 20 to 25-patient Phase 2a study of AV-101 as a monotherapy for treatment-resistant MDD under VistaGen's Cooperative Research and Development Agreement (CRADA) with the NIMH (Phase 2a Study). Dr. Carlos Zarate Jr., Chief, Section on the Neurobiology and Treatment of Mood Disorders and Chief of Experimental Therapeutics and Pathophysiology Branch at the NIMH and a leading clinical expert on the use of ketamine for treatment-resistant MDD, is the Principal Investigator of the Phase 2a Study. Following recent guidance from the NIMH, the Company currently anticipates that the NIMH will complete the Phase 2a Study by the end of 2017. VistaGen is preparing to launch a 280-patient, multi-center, double-blind, placebo controlled Phase 2b efficacy and safety study evaluating AV-101 as a new generation adjunctive treatment for MDD patients with an inadequate response to standard, FDA-approved antidepressant therapies. The Company currently anticipates commencing patient enrollment in the Phase 2b Study in the second quarter of 2017. Dr. Maurizio Fava of Harvard University Medical School will serve as the Principal Investigator of VistaGen's AV-101 Phase 2b Study. Topline clinical results from the Phase 2b Study are currently anticipated by the end of 2018. Dr. Mark Smith, Chief Medical Officer of VistaGen, commented, "We look forward to starting patient enrollment in our Phase 2b study of AV-101 as an adjunctive therapy in the treatment of MDD. We believe we have significantly de-risked this Phase 2b study with a clinical trial methodology that is designed to overcome the challenge of placebo effects in psychiatric clinical trials. Based on the study protocol we have designed in collaboration with key opinion leaders in depression and neuroscience, including our Principal Investigator, Dr. Fava, we expect that achieving a successful outcome of our Phase 2b study will be integral in realizing AV-101's potential to displace atypical antipsychotics and non-drug interventions in the current depression treatment paradigm, representing a much needed treatment solution for physicians and patients, as well as an enormous opportunity for VistaGen." "The NIMH recently updated us on their timelines for the completion of the Phase 2a study of AV-101 as a monotherapy for MDD. The Phase 2a study protocol requires considerable time and dedication from both the study participants and the multi-disciplinary NIMH teams involved. Patient enrollment for the Phase 2a study remains ongoing and we currently anticipate the NIMH's completion of the study by the end of 2017. Our top priority is to execute our plans for our Phase 2b study of AV-101 as a new generation adjunctive treatment of MDD, and we remain on track to launch that important study in the second quarter. As part of our Phase 2 program, this Phase 2b study has been specifically designed to achieve important outcomes that will be key to advancing AV-101 into a pivotal program in MDD and more broadly beyond MDD, as we continue to advance our global commercialization strategy. We are confident that our Phase 2 program is a major step forward in positioning AV-101 as a potentially transformative adjunctive treatment of MDD and other CNS disorders," concluded Mr. Singh. Summary of Financial Results for the Third Quarter of Fiscal 2017 Ended December 31, 2016 The Company recognized $1.25 million in sublicense revenue pursuant to its cardiac stem cell technology sublicense agreement with BlueRock Therapeutics, a next generation regenerative medicine company established by Bayer AG and Versant Ventures, in the third fiscal quarter ended December 31, 2016. Research and development expense totaled $1.61 million for the third fiscal quarter ended December 31, 2016, compared to $806,300 for the quarter ended December 31, 2015, reflecting increasing focus on nonclinical and clinical development of AV-101 and preparations for launch of the AV-101 Phase 2b Study in the second quarter of 2017. General and administrative expense increased to $2.3 million in the third fiscal quarter ended December 31, 2016, from $1.3 million for the same period in the prior year. The increase in G&A expense is the result of increased noncash stock compensation expense attributable to option and warrant grants in the period to employees, independent members of the Company's Board of Directors and consultants and other noncash expense related to grants of equity securities in payment of certain professional services, and a combination of corporate expenses, including investor relations and corporate development initiatives. For the third fiscal quarter ended December 31, 2016, the Company reported a net loss of approximately $2.6 million, or a net loss attributable to common stockholders of $0.34 per common share, compared to a net loss of approximately $2.1 million, or a net loss attributable to common stockholders of $1.95 per common share for the same period in the prior year. As of December 31, 2016, the Company had approximately $5.6 million of cash, cash equivalents and short term receivables, including a $1.25 million short term sublicense fee receivable from BlueRock Therapeutics pursuant to the Company's December 2016 technology sublicense agreement with BlueRock Therapeutics. In January 2017, the Company received the $1.25 million sublicense fee payment from BlueRock Therapeutics and currently believes it has sufficient financial resources to fund its expected operations at least through the first half of 2017, including preparation for and launch of its planned AV-101 Phase 2b Study in MDD. VistaGen Therapeutics, Inc. ( : VTGN), is a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders. VistaGen's lead CNS product candidate, AV-101, is a new generation oral antidepressant drug candidate in Phase 2 development. AV-101's mechanism of action is fundamentally differentiated from all FDA-approved antidepressants and atypical antipsychotics used adjunctively to treat MDD, with potential to drive a paradigm shift towards a new generation of safer and faster-acting antidepressants. AV-101 is currently being evaluated by the U.S. National Institute of Mental Health (NIMH) in a Phase 2a monotherapy study in MDD being fully funded by the NIMH and conducted by Dr. Carlos Zarate Jr., Chief, Section on the Neurobiology and Treatment of Mood Disorders and Chief of Experimental Therapeutics and Pathophysiology Branch at the NIMH. VistaGen is preparing to launch a 280-patient Phase 2b study of AV-101 as an adjunctive treatment for MDD patients with inadequate response to standard, FDA-approved antidepressant therapies. Dr. Maurizio Fava of Harvard University will be the Principal Investigator of the Phase 2b study. AV-101 may also have the potential to treat multiple CNS disorders and neurodegenerative diseases in addition to MDD, including chronic neuropathic pain, epilepsy, Parkinson's disease and Huntington's disease, where modulation of the NMDAR, AMPA pathway and/or key active metabolites of AV-101 may achieve therapeutic benefit. VistaStem Therapeutics is VistaGen's wholly owned subsidiary focused on applying human pluripotent stem cell (hPSC) technology, internally and with third-party collaborators, to discover, rescue, develop and commercialize proprietary new chemical entities (NCEs), including small molecule NCEs with regenerative potential, for CNS and other diseases, and cellular therapies involving stem cell-derived blood, cartilage, heart and liver cells. In December 2016, VistaGen exclusively sublicensed to BlueRock Therapeutics LP, a next generation regenerative medicine company established by Bayer AG and Versant Ventures, rights to certain proprietary technologies relating to the production of cardiac stem cells for the treatment of heart disease. For more information, please visit www.vistagen.com and connect with VistaGen on Twitter, LinkedIn and Facebook. The statements in this press release that are not historical facts may constitute forward-looking statements that are based on current expectations and are subject to risks and uncertainties that could cause actual future results to differ materially from those expressed or implied by such statements. Those risks and uncertainties include, but are not limited to, risks related to the successful launch, continuation and results of the NIMH's Phase 2a (monotherapy) and/or the Company's planned Phase 2b (adjunctive therapy) clinical studies of AV-101 in MDD, and other CNS diseases and disorders, protection of its intellectual property, and the availability of substantial additional capital to support its operations, including the development activities described above. These and other risks and uncertainties are identified and described in more detail in VistaGen's filings with the Securities and Exchange Commission (SEC). These filings are available on the SEC's website at www.sec.gov. VistaGen undertakes no obligation to publicly update or revise any forward-looking statements.
News Article | February 28, 2017
CINCINNATI--Researchers at the University of Cincinnati (UC) College of Medicine have discovered a new potential strategy to personalize therapy for brain and blood cancers. These findings are reported in the Feb. 28 edition of Cell Reports. "We found a new combination of therapeutics that could treat cancers that lack a protein called PTEN. PTEN is an important tumor suppressor, which means that it stops cell growth and division according to the needs of the body," says David Plas, PhD, Anna and Harold W. Huffman Endowed Chair for Glioblastoma Experimental Therapeutics. Plas is an associate professor in the Department of Cancer Biology, a member of the University of Cincinnati Cancer Institute and a researcher in the Brain Tumor Center of the UC Gardner Neuroscience Institute. Atsuo Sasaki, PhD, and Hala Elnakat Thomas, PhD, both in the Division of Hematology Oncology at the UC College of Medicine, were collaborators on the study. In early work using experimental therapeutics in human cancer cells and in tumor models, the Plas laboratory showed that stopping the production and function of the protein S6K1 could eliminate PTEN-deficient glioblastoma cells. Glioblastoma, the most aggressive form of brain cancer, is difficult to treat with targeted therapeutics. "We used support from the Huffman Foundation to conduct a sophisticated biochemical analysis of how cells respond to S6K1 targeting," Plas says. "Combining the biochemical results with computational analysis gave us the insight that we needed--there are targets in addition to S6K1 that can be hit to trigger the elimination of PTEN-deficient cancer cells." With the new information, the research team tested pharmaceutical-grade drug combinations for the ability to eliminate PTEN-deficient cancer cells. Results showed that the drugs LY-2779964 and BMS-777607 work together to specifically eliminate PTEN-deficient cells. "This is a completely new combination of targets in oncology," Plas says. "We have great hope that our new data will lead academic and industry researchers to investigate S6K1 as the center of new combination strategies for cancers of the brain, blood and other tissues." Future work in the project will test the safety and efficacy of the new combination using tumor models, with the goal of preparing the combination strategy for clinical trial. Ronald Warnick, MD, medical director of the UC Brain Tumor Center and a professor in the Department of Neurosurgery within the UC College of Medicine, adds that this kind of project is necessary in finding new and beneficial therapies for brain tumors. "There is a desperate need for novel therapeutic agents for patients with glioblastoma," he says. "This combination of drugs has the potential to become a game-changer." This study was funded by the American Cancer Society, the National Institutes of Health (R01 CA133164, R01 CA168815, R21NS100077, R01NS089815), the UC Brain Tumor Center, the Anna and Harold W. Huffman Endowed Chair for Glioblastoma Experimental Therapeutics and the UC Medical Scientist Training Program. Plas cites no conflict of interest.
News Article | February 17, 2017
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Exelixis, Inc. (NASDAQ:EXEL) today announced results from a phase 1 trial of cabozantinib in combination with either nivolumab or nivolumab plus ipilimumab in patients with refractory genitourinary tumors. The primary endpoint of the trial is to determine the dose limiting toxicity (DLT) and recommended phase 2 doses of the doublet and triplet combinations. The findings were presented during a poster session (Abstract #293) on February 17 at the 2017 Genitourinary Cancers Symposium, which is being held in Orlando, Florida, February 16 – 18, 2017. Between July 22, 2015 and December 31, 2016, 48 patients were accrued with previously treated metastatic urothelial carcinoma (mUC, n=19), urachal adenocarcinoma (n=4), squamous cell carcinoma of the bladder or urethra (n=2), germ cell tumor (n=4), castration-resistant prostate cancer (n=9), renal cell carcinoma (n=4), trophoblastic tumor (n=1), sertoli cell tumor (n=1) or penile squamous cell carcinoma (n=4) and treated in two parts. In Part I, 30 patients were treated with the doublet combination of cabozantinib and nivolumab at four dose levels. In Part II, 18 patients were treated with the triplet combination of cabozantinib, nivolumab and ipilimumab at three dose levels. Among the 43 patients who were evaluable for response, the objective response rate (ORR) for all tumor types was 30 percent (38 percent for the doublet dosing schedule and 18 percent for the triplet dosing schedule), with a 7 percent complete response (CR) rate and a 23 percent partial response (PR) rate. Stable disease (SD) was reported in 56 percent of patients. The ORR for patients with mUC was 38 percent, and 2 of 16 patients achieved a CR, while 2 patients with squamous cell carcinoma of the bladder had objective responses (1 CR and 1 PR). In the mUC cohort, 15 of 16 patients had a CR, PR or SD as their best response. Grade 3 adverse events (>5 percent of patients) observed in the doublet combination included neutropenia (17 percent), hypophosphatemia (13 percent), hypertension (10 percent), lipase increase (7 percent), fatigue (7 percent), diarrhea (7 percent) and dehydration (7 percent). Grade 3 adverse events (>5 percent of patients) observed in the triplet combination included hypertension (17 percent), hypophosphatemia (17 percent), fatigue (13 percent), hyponatremia (13 percent), lipase increase (13 percent), nausea (13 percent) and rash (6 percent). There were limited numbers of grade 4 adverse events (10 percent including thrombocytopenia and lipase increase in the doublet combination, and 6 percent (lipase increase) in the triplet combination), and no grade 5 adverse events observed in either part of the trial. “ There is a significant unmet need for treatment regimens that can slow tumor progression in advanced, intractable cancers such as metastatic urothelial carcinoma. The use of combination therapies may be a strategy that could increase anti-tumor activity in these patients,” said Andrea Apolo, M.D., Genitourinary Malignancies Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, the principal investigator of the trial. “ Previously reported data from Part I of the trial showed that cabozantinib in combination with nivolumab provided an encouraging objective response rate and tolerability profile across a diverse range of genitourinary tumors. Data from Part II also demonstrate that using cabozantinib with two immunotherapy agents is well-tolerated with promising early activity. These results support the further evaluation of both regimens in these tumor types.” The recommended doses for the ongoing expansion cohorts were determined to be cabozantinib 40 mg daily plus nivolumab 3 mg/kg once every 2 weeks for the doublet and cabozantinib 40 mg daily, nivolumab 3 mg/kg plus ipilimumab 1 mg/kg every 3 weeks for 4 doses, then nivolumab 3 mg/kg every 2 weeks for the triplet. “ These early clinical results generated by our collaborators at the NCI-CTEP suggest that the combination of cabozantinib with either nivolumab or nivolumab and ipilimumab in patients with genitourinary malignancies is associated with an encouraging tolerability, safety and activity profile,” said Michael M. Morrissey, Ph.D., president and Chief Executive Officer of Exelixis. “ With these results in hand, we are committed to further examining the potential of cabozantinib in combination with a variety of immunotherapies to treat a broad range of genitourinary and other cancers.” The trial is sponsored by the U.S. National Cancer Institute (NCI) through Cooperative Research and Development Agreements between the NCI’s Cancer Therapy Evaluation Program (CTEP), Division of Cancer Treatment and Diagnosis, and both Bristol-Myers Squibb and Exelixis. Andrea Apolo, M.D., of the NCI’s Genitourinary Malignancies Branch, is the principal investigator. The trial is being conducted by the NCI and includes centers from its Experimental Therapeutics Clinical Trials Network. The primary endpoint of the phase 1 trial is to determine the dose limiting toxicity (DLT) and recommended phase 2 doses of the doublet and triplet combinations. The secondary endpoint is clinical response rate as assessed by RECIST 1.1. Part I of the study included four dosing levels: cabozantinib 40 mg daily plus nivolumab 1 mg/kg once every 2 weeks; cabozantinib 40 mg daily plus nivolumab 3 mg/kg once every 2 weeks; cabozantinib 60 mg daily plus nivolumab 1 mg/kg once every 2 weeks; and cabozantinib 60 mg daily plus nivolumab 3 mg/kg once every 2 weeks. Part II of the study included three dosing levels: cabozantinib 40 mg daily, nivolumab 1 mg/kg plus ipilimumab 1 mg/kg every 3 weeks for 4 doses, then nivolumab 1 mg/kg every 2 weeks; cabozantinib 40 mg daily, nivolumab 3 mg/kg plus ipilimumab 1 mg/kg every 3 weeks for 4 doses, then nivolumab 3 mg/kg every 2 weeks; and cabozantinib 60 mg daily, nivolumab 3 mg/kg plus ipilimumab 1 mg/kg every 3 weeks for 4 doses, then nivolumab 3 mg/kg every 2 weeks. Data from Part I of the study evaluating the combination of cabozantinib with nivolumab in patients with previously treated genitourinary tumors were presented by Dr. Apolo at the European Society for Medical Oncology 2016 Congress. Expansion cohorts assessing cabozantinib and nivolumab are currently being accrued with bladder, renal and rare genitourinary cancer patients. Data from these patients will be reported at a later date. Genitourinary cancers are those that affect the urinary tract, bladder, kidneys, ureter, prostate, testicles, penis or adrenal glands — parts of the body involved in reproduction and excretion — and include renal cell carcinoma and urothelial carcinoma.1 Kidney cancer is among the top ten most commonly diagnosed forms of cancer among both men and women in the U.S., according to the American Cancer Society’s 2016 statistics.2 Clear cell renal cell carcinoma is the most common type of kidney cancer in adults.3 If detected in its early stages, the five-year survival rate for RCC is high; for patients with advanced or late-stage metastatic RCC, however, the five-year survival rate is only 12 percent, with no identified cure for the disease.2 Approximately 30,000 patients in the U.S. and 68,000 globally require treatment.4 Prostate cancer is the second most common cause of cancer death in men, behind only skin cancer.5 There is a high survival rate for patients when prostate cancer is detected early, but once the disease has spread to other parts of the body the five-year survival rate is just 28 percent.6 Approximately 2,850,000 men were living with prostate cancer in the U.S. in 2013,7 and 180,000 new cases are diagnosed each year.5 Urothelial cancers encompass carcinomas of the bladder, ureter and renal pelvis at a ratio of 50:3:1, respectively.8 Urothelial carcinoma occurs mainly in older people, with 90 percent of patients aged 55 or older.9 Bladder cancer is the fourth most common cancer in men and accounts for about five percent of all new cases of cancer in the U.S. each year.9 In 2013, an estimated 587,426 people were living with bladder cancer in the U.S.10 CABOMETYX is the tablet formulation of cabozantinib. Its targets include MET, AXL and VEGFR-1, -2 and -3. In preclinical models, cabozantinib has been shown to inhibit the activity of these receptors, which are involved in normal cellular function and pathologic processes such as tumor angiogenesis, invasiveness, metastasis and drug resistance. CABOMETYX is available in 20 mg, 40 mg or 60 mg doses. The recommended dose is 60 mg orally, once daily. On April 25, 2016, the FDA approved CABOMETYX tablets for the treatment of patients with advanced renal cell carcinoma who have received prior anti-angiogenic therapy. On September 9, 2016, the European Commission approved CABOMETYX tablets for the treatment of advanced renal cell carcinoma in adults who have received prior vascular endothelial growth factor (VEGF)-targeted therapy in the European Union, Norway and Iceland. On February 29, 2016, Exelixis and Ipsen jointly announced an exclusive licensing agreement for the commercialization and further development of cabozantinib indications outside of the United States, Canada and Japan. On December 21, 2016, this agreement was amended to include commercialization rights for Ipsen in Canada. On January 30, 2017, Exelixis and Takeda Pharmaceutical Company Limited announced an exclusive licensing agreement for the commercialization and further clinical development of cabozantinib for all future indications in Japan, including RCC. Cabozantinib is not indicated for the treatment of refractory mUC and other genitourinary tumors. Hemorrhage: Severe hemorrhage occurred with CABOMETYX. The incidence of Grade ≥3 hemorrhagic events was 2.1% in CABOMETYX-treated patients and 1.6% in everolimus-treated patients. Fatal hemorrhages also occurred in the cabozantinib clinical program. Do not administer CABOMETYX to patients that have or are at risk for severe hemorrhage. Gastrointestinal (GI) Perforations and Fistulas: Fistulas were reported in 1.2% (including 0.6% anal fistula) of CABOMETYX-treated patients and 0% of everolimus-treated patients. GI perforations were reported in 0.9% of CABOMETYX-treated patients and 0.6% of everolimus-treated patients. Fatal perforations occurred in the cabozantinib clinical program. Monitor patients for symptoms of fistulas and perforations. Discontinue CABOMETYX in patients who experience a fistula that cannot be appropriately managed or a GI perforation. Thrombotic Events: CABOMETYX treatment results in an increased incidence of thrombotic events. Venous thromboembolism was reported in 7.3% of CABOMETYX-treated patients and 2.5% of everolimus-treated patients. Pulmonary embolism occurred in 3.9% of CABOMETYX-treated patients and 0.3% of everolimus-treated patients. Events of arterial thromboembolism were reported in 0.9% of CABOMETYX-treated patients and 0.3% of everolimus-treated patients. Fatal thrombotic events occurred in the cabozantinib clinical program. Discontinue CABOMETYX in patients who develop an acute myocardial infarction or any other arterial thromboembolic complication. Hypertension and Hypertensive Crisis: CABOMETYX treatment results in an increased incidence of treatment-emergent hypertension. Hypertension was reported in 37% (15% Grade ≥3) of CABOMETYX-treated patients and 7.1% (3.1% Grade ≥3) of everolimus-treated patients. Monitor blood pressure prior to initiation and regularly during CABOMETYX treatment. Withhold CABOMETYX for hypertension that is not adequately controlled with medical management; when controlled, resume CABOMETYX at a reduced dose. Discontinue CABOMETYX for severe hypertension that cannot be controlled with anti-hypertensive therapy. Discontinue CABOMETYX if there is evidence of hypertensive crisis or severe hypertension despite optimal medical management. Diarrhea: Diarrhea occurred in 74% of patients treated with CABOMETYX and in 28% of patients treated with everolimus. Grade 3 diarrhea occurred in 11% of CABOMETYX-treated patients and in 2% of everolimus-treated patients. Withhold CABOMETYX in patients who develop intolerable Grade 2 diarrhea or Grade 3-4 diarrhea that cannot be managed with standard antidiarrheal treatments until improvement to Grade 1; resume CABOMETYX at a reduced dose. Dose modification due to diarrhea occurred in 26% of patients. Palmar-Plantar Erythrodysesthesia Syndrome (PPES): Palmar-plantar erythrodysesthesia syndrome (PPES) occurred in 42% of patients treated with CABOMETYX and in 6% of patients treated with everolimus. Grade 3 PPES occurred in 8.2% of CABOMETYX-treated patients and in <1% of everolimus-treated patients. Withhold CABOMETYX in patients who develop intolerable Grade 2 PPES or Grade 3 PPES until improvement to Grade 1; resume CABOMETYX at a reduced dose. Dose modification due to PPES occurred in 16% of patients. Reversible Posterior Leukoencephalopathy Syndrome (RPLS): RPLS, a syndrome of subcortical vasogenic edema diagnosed by characteristic finding on MRI, occurred in the cabozantinib clinical program. Perform an evaluation for RPLS in any patient presenting with seizures, headache, visual disturbances, confusion, or altered mental function. Discontinue CABOMETYX in patients who develop RPLS. Embryo-fetal Toxicity: CABOMETYX can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with CABOMETYX and for 4 months after the last dose. Adverse Reactions: The most commonly reported (≥25%) adverse reactions are: diarrhea, fatigue, nausea, decreased appetite, PPES, hypertension, vomiting, weight decreased, and constipation. Drug Interactions: Strong CYP3A4 inhibitors and inducers: Reduce the dosage of CABOMETYX if concomitant use with strong CYP3A4 inhibitors cannot be avoided. Increase the dosage of CABOMETYX if concomitant use with strong CYP3A4 inducers cannot be avoided. Lactation: Advise a lactating woman not to breastfeed during treatment with CABOMETYX and for 4 months after the final dose. Reproductive Potential: Contraception―Advise females of reproductive potential to use effective contraception during treatment with CABOMETYX and for 4 months after the final dose. Infertility ―CABOMETYX may impair fertility in females and males of reproductive potential. Hepatic Impairment: Reduce the CABOMETYX dose in patients with mild (Child-Pugh score [C-P] A) or moderate (C-P B) hepatic impairment. CABOMETYX is not recommended for use in patients with severe hepatic impairment. Please see full Prescribing Information at https://cabometyx.com/downloads/cabometyxuspi.pdf. Exelixis, Inc. (Nasdaq: EXEL) is a biopharmaceutical company committed to the discovery, development and promotion of new medicines with the potential to improve care and outcomes for people with cancer. Since its founding in 1994, three products discovered at Exelixis have progressed through clinical development to receive regulatory approval. Currently, Exelixis is focused on advancing cabozantinib, an inhibitor of multiple tyrosine kinases including MET, AXL and VEGF receptors, which has shown clinical anti-tumor activity in more than 20 forms of cancer and is the subject of a broad clinical development program. Two separate formulations of cabozantinib have received regulatory approval to treat certain forms of kidney and thyroid cancer and are marketed for those purposes as CABOMETYX™ tablets (U.S. and EU) and COMETRIQ® capsules (U.S. and EU), respectively. Another Exelixis-discovered compound, COTELLIC® (cobimetinib), a selective inhibitor of MEK, has been approved in major territories including the United States and European Union, and is being evaluated for further potential indications by Roche and Genentech (a member of the Roche Group) under a collaboration with Exelixis. For more information on Exelixis, please visit www.exelixis.com or follow @ExelixisInc on Twitter. This press release contains forward-looking statements, including, without limitation, statements related to: the further evaluation of cabozantinib in combination with immunotherapies to treat a variety of genitourinary tumors; future data results from expansion cohorts assessing cabozantinib and nivolumab in bladder, renal and rare genitourinary cancer patients; Exelixis' commitment to the discovery, development and commercialization of new medicines with the potential to improve care and outcomes for people with cancer; Exelixis’ focus on advancing cabozantinib; and the continued development of cobimetinib. Words such as “may,” “further,” “committed,” “focused,” or other similar expressions identify forward-looking statements, but the absence of these words does not necessarily mean that a statement is not forward-looking. In addition, any statements that refer to expectations, projections or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements are based upon Exelixis’ current plans, assumptions, beliefs, expectations, estimates and projections. Forward-looking statements involve risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in the forward-looking statements as a result of these risks and uncertainties, which include, without limitation: Exelixis’ ability and the ability of its collaborators to conduct clinical trials of cabozantinib sufficient to achieve a positive completion; risks related to the potential failure of cabozantinib to demonstrate safety and efficacy in clinical testing; the availability of data at the referenced times; risks and uncertainties related to regulatory review and approval processes and Exelixis’ compliance with applicable legal and regulatory requirements; the degree of market acceptance of CABOMETYX and the availability of coverage and reimbursement for CABOMETYX; the risk that unanticipated developments could adversely affect the commercialization of CABOMETYX; Exelixis’ dependence on its relationships with Ipsen and Takeda, including, the level of their investment in the resources necessary to successfully commercialize cabozantinib in the territories where it is approved; Exelixis’ dependence on its relationship with Genentech/Roche with respect to cobimetinib and Exelixis’ ability to maintain its rights under the collaboration; Exelixis’ dependence on third-party vendors; Exelixis’ ability to protect the company’s intellectual property rights; market competition; changes in economic and business conditions, and other factors discussed under the caption “Risk Factors” in Exelixis’ quarterly report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 3, 2016, and in Exelixis’ future filings with the SEC. The forward-looking statements made in this press release speak only as of the date of this press release. Exelixis expressly disclaims any duty, obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in Exelixis’ expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based.
News Article | February 15, 2017
MOUNTAIN VIEW, CA--(Marketwired - February 13, 2017) - Amunix Operating Inc., a privately held biotechnology company and developer of a broad portfolio of biopharmaceuticals based on its novel XTEN® polymer half-life extension technology, XTEN drug-conjugate delivery technology and bispecific T-cell engager (ProTIA) pro-drug cancer therapeutics platforms, announced that it has appointed Frederick Hausheer, MD, FACP, as its Chief Medical Officer. Dr. Hausheer brings more than 25 years' experience in global oncology drug development, translational medicine, and drug discovery in commercial and academic settings to Amunix. Volker Schellenberger, Ph.D., President and Chief Executive Officer, said, "We are extremely pleased to have Fred join the Amunix management team. He brings exceptionally broad product development leadership and clinical expertise to the organization. His deep clinical experience in the oncology field will be especially valuable in the support of our ProTIA oncology platform. I look forward to working with Fred to accelerate the advancement of our clinical pipeline." Dr. Hausheer said, "It is a great privilege to be joining Amunix. I am exceedingly impressed with the technology, the Amunix team and the progress that has been made with the company's ProTIA T-cell engager cancer targeting platform which has broad potential to serve as a novel important therapeutic option over current cancer treatment regimens. I am excited about the prospects for advancing the pipeline of ProTIA platform products into patients and beyond. I believe that Amunix' innovative technologies will be an important advance for immuno-oncology drugs to improve patient outcomes." Over the course of his career, Dr. Hausheer has helped secure over $750 million in financial transactions involving capital raising, joint ventures, strategic alliances and licensing of compounds for partnership opportunities. He is experienced in trial design and execution, and involved in numerous regulatory interactions for drugs spanning from pre-IND and Phase I-III development in the United States, Europe, Asia/PAC and other territories. As founder, Chairman, CEO and President of BioNumerik from 1992-2015, he led the discovery and development of two novel oncologic therapeutics from laboratory through global Phase III development. In addition, he co-founded and served as Chairman of KI Pharma from 2005-2013, a joint venture between BioNumerik Pharmaceuticals, Inc. and ASKA Pharmaceutical Co., with a focus on development and commercialization of oncology products in Japan. Prior to joining industry, Dr. Hausheer was an Associate Professor of Medical Oncology and Associate Director Drug Development and Chief, Molecular Design at the University Texas Health Science Center and Cancer Therapy and Research Center and performed oncology drug research as a Senior Scientist at the National Cancer Institute. Dr. Hausheer completed his graduate training in Physiology and Biophysics at the University of Illinois-Champagne. He obtained his MD and completed his Internal Medicine residency at the University of Missouri School of Medicine, followed by completion of Medical Oncology fellowship training and joining the faculty at Johns Hopkins, where he focused on development of novel experimental therapeutics and pharmacology. He currently holds adjunct professorships at the University of Missouri in Internal Medicine and Oncology and The Johns Hopkins Oncology Center in Medical Oncology. He is board certified in Internal Medicine and Medical Oncology. Dr. Hausheer has published over 200 articles, abstracts and book chapters, and is an inventor or co-inventor on more than 400 issued/allowed United States and international patents. He has served as a board member to JP Morgan Chase, the Whittaker Institute for BioMedical Engineering at Johns Hopkins, the National Cancer Institute's (NCI) Experimental Therapeutics Study Section I, and the NCI Institutional Review Board. Amunix, based in Mountain View, CA, is a privately held biotechnology company focused on the discovery and development of biologics with improved in vivo half-lives. Amunix' half-life extension technology is based on XTEN -- hydrophilic, unstructured, biodegradable proteins that impart a number of favorable properties upon the molecules to which they are attached. XTEN can be recombinantly fused or chemically conjugated to peptides, proteins, and other pharmaceuticals. In addition to the advantages of reduced dosing frequency, XTENylation also stabilizes plasma drug concentrations, which often results in increased efficacy as well as reduced side effects. Two genetically fused XTENylated products have been tested clinically. VRS-859 (exenatide-XTEN) has been tested through Phase I in the treatment of diabetes and VRS-317 (human growth hormone-XTEN) is currently in Phase III testing. Amunix is also working with additional partners, including Eli Lilly, Bioverativ, Roche, Janssen, Naia and other undisclosed companies in a wide range of therapeutic areas. Amunix is developing an internal pipeline of ProTIA (Protease Triggered Immune Activator) immuno-oncology therapeutics. ProTIAs are bispecific molecules that bind tumor antigens and T cells. ProTIAs are administered as long-acting prodrugs that can be activated in the tumor environment by tumor-associated proteases. Amunix is actively seeking partnerships for applications of its XTEN technology and its ProTIA platform. For additional information about the company, please visit www.amunix.com.
News Article | February 21, 2017
NEW YORK, Feb. 21, 2017 (GLOBE NEWSWIRE) -- TG Therapeutics, Inc. (NASDAQ:TGTX) announced the publication of clinical data from a Phase 1/2 trial of TG-1101 (ublituximab), the Company's novel glycoengineered anti-CD20 monoclonal antibody, in patients with B-cell non-Hodgkin Lymphoma (NHL) or Chronic Lymphocytic Leukemia (CLL) previously exposed to rituximab. The data demonstrates single agent TG-1101 to be well tolerated with the most common adverse event observed being grade 1/2 infusion related reactions (IRR), with no grade 3/4 IRRs. TG-1101 monotherapy was active, with a 45% overall response rate (ORR) observed among heavily pretreated patients with NHL and CLL, including those who were refractory to prior anti-CD20 based therapy. These data are described further in the manuscript titled, “A phase 1/2 trial of ublituximab, a novel, glycoengineered anti-CD20 monoclonal antibody, in patients with B-cell non-Hodgkin lymphoma or chronic lymphocytic leukaemia previously exposed to rituximab,” which was published online today in the British Journal of Haematology. The online version of the article can be accessed at http://onlinelibrary.wiley.com/doi/10.1111/bjh.14534/full. “We want to thank Dr. Owen O’Connor, and the team from Columbia Presbyterian Medical Center and the Center for Lymphoid Malignancies for their work on this Phase 1/2 trial of single agent TG-1101 and congratulate them on the publication of these data. Since the inception of our Company, we have been focused on developing best-in-class agents with the goal of building novel combination therapies. This single agent data illustrates that TG-1101 is a safe and highly-active anti-CD20 monoclonal antibody on top of which additional treatments can be layered. The safety profile, speed of infusion, and response rates observed, with single agent TG-1101, especially in rituximab-refractory patients, serve as a foundation for our belief that TG-1101 is a best-in-class anti-CD20 monoclonal antibody,” stated Michael S. Weiss, Executive Chairman and Chief Executive Officer of TG Therapeutics. Mr. Weiss continued, “These Phase 1/2 data, as well as the combination data of TG-1101 plus ibrutinib published in the British Journal of Haematology late last year, further support our Phase 3 GENUINE trial of TG-1101 in combination with ibrutinib and we look forward to presenting top-line data from this study in the first half of this year.” “The addition of an anti-CD20 monoclonal antibody to other treatments, whether chemo-based or novel targeted therapies, has demonstrated to be an impactful way to enhance responses for patients with NHL and CLL. Acquired resistance to rituximab is a significant clinical issue for which many patients need an alternative effective agent to overcome the resistance. We are highly encouraged by the results we have seen in the clinic with ublituximab and believe the drug’s safety profile, as well as shortened infusion times as compared to other anti-CD20s, can provide meaningful benefit to patients,” stated Dr. Owen A. O’Connor, Professor of Medicine and Experimental Therapeutics, Director Lymphoid Malignancies at Columbia Presbyterian Medical Center. TG Therapeutics is a biopharmaceutical company focused on the acquisition, development and commercialization of novel treatments for B-cell malignancies and autoimmune diseases. Currently, the company is developing two therapies targeting hematological malignancies and autoimmune diseases. TG-1101 (ublituximab) is a novel, glycoengineered monoclonal antibody that targets a specific and unique epitope on the CD20 antigen found on mature B-lymphocytes. TG Therapeutics is also developing TGR-1202, an orally available PI3K delta inhibitor. The delta isoform of PI3K is strongly expressed in cells of hematopoietic origin and is believed to be important in the proliferation and survival of B‐lymphocytes. Both TG-1101 and TGR-1202 are in clinical development for patients with hematologic malignancies, with TG-1101 recently entering clinical development for autoimmune disorders. The Company also has pre-clinical programs to develop IRAK4 inhibitors, BET inhibitors, and anti-PD-L1 and anti-GITR antibodies. TG Therapeutics is headquartered in New York City. Some of the statements included in this press release, particularly those with respect to anticipating the timing of the completion of the GENUINE study, timing of top-line data for the GENUINE study, the usability of the results from GENUINE for accelerated approval, timing of initial data from the UNITY-DLBCL study, timing of the release of data and commencement of our MS pivotal program may be forward-looking statements that involve a number of risks and uncertainties. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. Among the factors that could cause our actual results to differ materially are the following: our ability to successfully and cost-effectively complete the GENUINE, the UNITY-CLL or the UNITY-DLBCL trials; the risk that the clinical results from the GENUINE, UNITY-CLL and/or UNITY-DLBCL studies will be not positive and/or will not support regulatory approval of TG-1101 or TGR-1202; the risk that the FDA will not grant us a pre-BLA meeting to discuss the results of the GENUINE study; the risk that we will not file a BLA for TG-1101 or an NDA for TGR-1202 based on either the GENUINE or the UNITY-CLL; the risk that despite early positive trends in enrollment in the UNITY-CLL study that enrollment will be delayed beyond our projections; the risk that the planned interim analysis will not allow early closure of the single agent arms in the UNITY-CLL study, necessitating enrollment beyond the projected 450 patients, which would extend enrollment beyond our projections; the risk that safety issues or trends will be observed in the GENUINE study, the UNITY-CLL and/or the UNITY-DLBCL study that prevent approval of either TG-1101 and/or TGR-1202 or require us to terminate either the GENUINE study or the UNITY-CLL or the UNITY-DLBCL study prior to completion; the risk that the data (both safety and efficacy) from future clinical trials will not coincide with the data produced from prior pre-clinical and clinical trials; the risk that the GENUINE study, as amended or the UNITY-CLL or the UNITY-DLBCL studies, or any of our other registration-directed clinical trials as designed or amended may not be sufficient or acceptable to support regulatory approval; the risk that trials will take longer to enroll than expected; the risk that the projected cost savings to be realized by amending the GENUINE trial will not be realized; our ability to achieve the milestones we project over the next year; our ability to manage our cash in line with our projections, and other risk factors identified from time to time in our reports filed with the Securities and Exchange Commission. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. This press release and prior releases are available at www.tgtherapeutics.com. The information found on our website is not incorporated by reference into this press release and is included for reference purposes only.