Repurposing a cholesterol drug candidate for cholestatic liver disease
June 19, 2013
Lumena Pharmaceuticals Inc. is converting a compound previously tested as a cholesterol-lowering agent into an experimental treatment for cholestatic liver diseases. The molecule inhibits a so-called transporter that normally helps the body recycle bile acid.
RESEARCH TRIANGLE PARK, NC– May 21, 2013 – Liquidia Technologies today announced that its collaborative agreement with PATH, a global health nonprofit organization, has been extended. The extension of this agreement allows for the continuation of preclinical proof-of-concept studies on a next generation pneumococcal vaccine. If successful, the vaccine could potentially allow for broadened efficacy and manufacturing efficiencies that translate to greater access to the vaccine by the global population. Pneumonia kills approximately 1.3 million children under the age of five years every year—more than any other illness. Nearlyhalf of these deaths are caused by Streptococcus pneumoniae, a bacterium that has many variations globally and is becoming increasingly resistant to antibiotics.
“We are exceptionally proud of the work we have done with PATH and excited about the opportunity to continue this work with them,” said Neal Fowler, Chief Executive Officer at Liquidia Technologies. “Our shared goal to improve vaccine performance and reduce manufacturing costs using our transformative technology remains unchanged as does our confidence that this collaboration has the potential to significantly impact the spread of pneumonia in the developing world.”
NOVATO, CA – May 15, 2013 - Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced a Phase 1/2 study of UX003 for mucopolysaccharidosis type 7 (MPS 7, or Sly Syndrome). UX003 is a recombinant human β-glucuronidase intended as an enzyme replacement therapy (ERT) for the treatment of MPS 7, an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme β-glucuronidase and a severe multi-system disease. MPS 7 has no approved therapies.
New Data in High Risk Patients Presented at Heart Rhythm Society
Shows a 70% Reduction in CIED Infections
Denver, CO (May 10, 2013): Use of TYRX, Inc.’s AIGISRx® Antibacterial Envelope significantly reduced device infection rates in patients at high risk for device infection who received cardiovascular implantable electronic device (CIED) implants, according to a study presented today at Heart Rhythm 2013, the Heart Rhythm Society’s 34th Annual Scientific Sessions.
MORRISVILLE, N.C. — Nanotechnology company Liquidia Technologies plans to form a new ophthalmology company with an experimental glaucoma treatment expected to start clinical trials in 2014.
Morrisville-based Liquidia said today that the new company would be able to advance Liquidia’s efforts to create products in ophthalmology and other therapeutic areas while also maximizing the return for the company’s investors. WRAL Tech Wire reported in February that Liquidia was considering spinning out one or more companies this year.
Liquidia offered few other details in today’s announcement, other than to say it would have more information later in the second quarter. In a statement, CEO Neal Fowler said that the structure for creating and financing the new company will be considered for other assets and therapeutic areas.
Liquidia’s proprietary “PRINT” technology enables a nanoparticle manufacturer to control the size and shape of the nanoparticles. Changing the size and shape of a nanoparticle can improve drug delivery and reduce side effects. Vaccines were Liquidia’s first target, a pursuit that GlaxoSmithKline liked enough to strike a licensing deal that gave GSK exclusive rights to develop new vaccines with the technology.
Liquidia said in today’s announcement that it independent from the new ophthalmology business, Liquidia will continue to focus on vaccine and pulmonary product development, including the work that has already begun under the GSK partnership.
Liquidia’s technology is a platform technology that has medical as well as consumer product applications. Fowler has acknowledged a partnership with Proctor & Gamble (NYSE: PG). In February, Fowler said that Liquidia’s PRINT technology can be used to make nanoparticles that improve how a product is delivered to the skin. At the time, he said an ophthalmology company could be spun out first, followed by a consumer product with P&G.
NOVATO, CA –May 1, 2013 - Ultragenyx Pharmaceutical Inc., a biotechnology company focused on the development of treatments for rare and ultra-rare genetic disorders, today announced it has appointed Eric Yuen, MD as its Chief Medical Officer and Senior Vice President. In this new role, Dr. Yuen will provide leadership and direction to Clinical Operations, Clinical Affairs, Clinical Sciences, Data Management/Biometrics, and Drug Safety. He will report to Dr. Emil Kakkis, CEO, and will serve on the Senior Management Team.
Dr. Yuen brings over 19 years of clinical development experience in academia and industry. In 2004, Dr. Yuen joined Johnson & Johnson as Senior Director and Neurology Franchise Medical Leader. During his tenure at J&J, he also held several executive positions including Vice President, Therapeutic Area Head, CNS in 2006 and Vice President, Scientific Licensing from 2008 to 2009. Dr. Yuen joins Ultragenyx from Janssen Alzheimer Immunotherapy R&D, LLC, a J&J affiliate, where he was the Head of Clinical Development since 2009. Prior to J&J, Dr. Yuen also served as Director of Clinical Research, Department of Clinical Neuroscience at the Merck Research Laboratories.
“As a board-certified neurologist, Dr. Yuen’s addition to the management team at Ultragenyx will provide critical support in advancing our clinical programs,” said Dr. Kakkis. “His clinical vision and proven leadership, as well as his extensive medical and research experience, will strengthen our clinical development efforts and support the progress of our product pipeline.”
Dr. Yuen commented, “I am excited about being a part of a dedicated team focused on developing novel treatments for rare disease patients who have no approved treatments and applying my experiences in clinical development and neuromuscular diseases to help solve the devastating medical problems of these metabolic genetic diseases.”
Dr. Yuen received his undergraduate training at Stanford University and his M.D. from the University of Chicago, Pritzker School of Medicine. He completed his residency in Neurology and post-doctoral fellowships at the University of California, San Francisco. Before joining industry in 2000, Dr. Yuen was a faculty member at the University of Washington, serving as a clinical investigator, studying treatments for ALS, neuropathy and multiple sclerosis.
About Ultragenyx
Ultragenyx is a privately held, development-stage biotechnology company committed to bringing to market life-transforming therapeutics for patients with rare and ultra-rare metabolic genetic diseases. The company focuses on diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no effective treatments.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’ strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company’s website at www.ultragenyx.com.
Monmouth Junction, NJ. (April 15, 2013): TYRX, Inc., the leader in the commercialization of implantable medical devices intended to help reduce surgical site infections, announced today that the first implantation of its new AIGISRx R Fully Resorbable Antibacterial Envelope has taken place at the Quebec Heart and Lung Institute in Quebec City, Canada by Dr. François Philippon. TYRX announced on January 31, 2013 that it had received a license from Health Canada to market its AIGISRx R Fully Resorbable technology.
DURHAM, N.C., April 12, 2013 — Chimerix's debut as a public company produced a whopping climb in stock price.
The Durham company went public at $14 per share, raising $102 million for the antivirals company.
But Chimerix’s public debut proved to be even stronger and the stock marched upward throughout the day topping the $18 per share mark in afternoon trading. Chimerix (Nasdaq: CMRX) closed its first day of trading at $18.79 per share, up 34 percent from its offering price.
The closing price was the high for the day. Some 4.5 million shares were traded.
Chimerix sold 7.3 million shares, which is some 20 percent more than the 6.1 million shares that the company had originally planned to sell. Morgan Stanley and Cowen and Company are joint book-running managers for the offering. William Blair and Lazard Capital Markets are co-managers.
Chimerix can raise even more money. Underwriters have 30 days to purchase up to an additional 1 million shares of common stock to cover over-allotments, if any. The offering is expected to close on April 16.
RESEARCH TRIANGLE PARK, Apr 11, 2013 (GLOBE NEWSWIRE via COMTEX) -- Chimerix, Inc. CMRX 0.00% , a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, announced today the pricing of its initial public offering of 7,320,000 shares of its common stock at a price to the public of $14.00 per share. The shares of Chimerix's common stock are scheduled to begin trading on the NASDAQ Global Market under the symbol "CMRX" on April 11, 2013. All of the shares of common stock are being offered by Chimerix. In addition, Chimerix has granted the underwriters a 30-day option to purchase up to an additional 1,098,000 shares of common stock to cover over-allotments, if any. The offering is expected to close on April 16, 2013, subject to customary closing conditions.
Morgan Stanley and Cowen and Company are acting as joint book-running managers for the offering. William Blair and Lazard Capital Markets are acting as co-managers.
A registration statement relating to these securities was declared effective by the Securities and Exchange Commission on April 10, 2013. The offering will be made only by means of a prospectus. Copies of the prospectus relating to the offering may be obtained from: Morgan Stanley & Co. LLC, Attn: Prospectus Department, 180 Varick Street, 2 Floor, New York, NY 10014, or by calling toll free 1-866-718-1649 or emailing at prospectus@morganstanley.com; or Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, by calling (631) 274-2806, or faxing (631) 254-7140.
This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or other jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction.
First patient enrolled in integrated program designed to improve knowledge of rare disease
NOVATO, CA –April 8, 2013 -Ultragenyx Pharmaceutical Inc. today announced the launch of a unique Disease Monitoring Program (DMP) for Hereditary Inclusion Body Myopathy (HIBM), also known as GNE Myopathy. The goal of the HIBM-DMP is to improve the body of knowledge about this rare disease and its typical course. This novel program is being conducted in partnership with the University of Newcastle's TREATNMD organization, a global neuromuscular physician network in Newcastle, England.The main objectives of the HIBM-DMP are to expand knowledge of the clinical presentation, progression and variation of HIBM patients; identify and qualify biomarkers and other efficacy measures; inform the design and interpretation of clinical studies of investigational products for HIBM and eventually to optimize patient management.
