MUSC Foundation for Research Development welcomes Art Pappas to board
Pappas to share venture capital expertise in pharmaceutical and life sciences industries
CHARLESTON, SC - April 23, 2014 - The Medical University of South Carolina Foundation for Research Development (FRD) has announced that venture capitalist Art Pappas has joined the organization’s Board of Directors, effective immediately.
Data Presented at American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting
NOVATO, Calif., March 27, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of preliminary data from the Phase 1/2 study of recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of mucopolysaccharidosis 7 (MPS 7, Sly syndrome).
Funding to support clinical development of treatments for rare cholestatic liver diseases and nonalcoholic steatohepatitis
SAN DIEGO – March 11, 2014 – Lumena Pharmaceuticals (Lumena), a biopharmaceutical company focused on the development and commercialization of novel products for rare cholestatic liver diseases and serious metabolic disorders, today announced that it has secured $45 million in Series B financing. New Enterprise Associates (NEA) led the financing, with Adage Capital Management and RA Capital Management participating and joining existing investors Pappas Ventures, RiverVest Venture Partners and Alta Partners. Founded in 2011 by Pappas Ventures, Lumena will use the funding to advance the clinical development of LUM001, its lead product candidate, for the treatment of rare cholestatic liver disease in pediatric and adult patients, as well as LUM002 for the treatment of nonalcoholic steatohepatitis (NASH).
Novato, CA, March 11, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, announced the first patient enrolled in the Phase 2 study of triheptanoin (UX007) for the treatment of glucose transporter type-1 deficiency syndrome (Glut1 DS), at Columbia University. Glut1 DS, also known as De Vivo disease, is a rare, severely debilitating disease characterized by seizures, developmental delay, and movement disorder.
- IMPACT-PCP Study Results Reinforce the Clinical Utility of Corus CAD in the Primary Care Setting, Where Almost 8,000 Patients Present With Non-Acute Chest Pain and Related Symptoms Every Day -
- Clinicians Modified Their Diagnostic Strategy in 58% of Patients Following a Corus CAD Test Score -
PALO ALTO, Calif. – March 10, 2014 – CardioDx, Inc., a molecular diagnostics company specializing in cardiovascular genomics, today announced the publication of the IMPACT-PCP (Investigation of a Molecular Personalized Coronary Gene Expression Test on Primary Care Practice Pattern) trial reinforcing the value of Corus® CAD as an initial diagnostic test for the evaluation of patients presenting with non-acute symptoms suggestive of obstructive coronary artery disease (CAD) in the primary care setting. The study appears in the March 2014 issue of the Journal of the American Board of Family Medicine.
Company to Advance Its Preclinical and Clinical Rare Disease Programs
BOSTON, MA--(Marketwired - Feb 24, 2014) - Marina Biotech, Inc. (PINKSHEETS: MRNA), a leading oligonucleotide-based drug discovery and development company focused on rare diseases, announced today that it has entered into a binding term sheet with certain qualified investors, led by Steven T. Newby, a long-time biotechnology investor, for the issuance of convertible preferred stock at a conversion price equivalent to $0.75 per share of common stock resulting in gross proceeds of $6 million. In addition, the Company will issue to the investors warrants to purchase 6 million shares of common stock. The warrants will have an exercise price of $0.75 per share and are exercisable for a period of five years after the Company regains compliance with its reporting obligations under the Securities Exchange Act. The offering is expected to close on or about March 7, 2014, subject to the execution of a customary Securities Purchase Agreement regarding the transaction and the satisfaction of customary closing conditions. The Company also announced that the holders of the Company's Promissory Note have agreed to convert the remaining principal and interest on the Note to common stock at a conversion price of $0.75 and release their lien on the Company's intellectual property. Proceeds from the financing will be used to restart certain day-to-day operations, repay the Company's outstanding obligations, regain compliance with the Company's Exchange Act reporting obligations and advance the Company's preclinical and clinical rare disease programs.
NOVATO, Calif., Feb. 12, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced data presentations from a single patient treated with recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of mucopolysaccharidosis 7 (MPS 7, Sly syndrome).
Open-label study to assess safety and clinical effects of triheptanoin
NOVATO, CA – February 11, 2014 – Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the first patient has been enrolled in an open-label Phase 2 study to assess safety and clinical effects of triheptanoin, also known as UX007, in patients severely affected by long-chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD are a group of autosomal recessive genetic disorders characterized by metabolic deficiencies in which the body is unable to break down and convert long chain fatty acids into energy.
NOVATO, Calif., Feb. 6, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the appointment of Clay B. Siegall, Ph.D. and Matthew Fust to the company's Board of Directors effective January 30, 2014. Dr. Siegall, currently President, Chief Executive Officer and Chairman of the Board of Seattle Genetics, Inc., and Mr. Fust, formerly Executive Vice President and Chief Financial Officer of Onyx Pharmaceuticals, Inc. will serve as independent directors for Ultragenyx. With these additions, Ultragenyx extends gratitude and appreciation for the counsel provided by Mr. Ben Auspitz, who has resigned from his position on the Board effective January 30, 2014.
NOVATO, Calif., Feb. 5, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the closing of its initial public offering of 6,624,423 shares of common stock at an initial public offering price of $21.00 per share, which includes the exercise in full by the underwriters of their option to purchase up to 864,054 additional shares of common stock. All of the shares of common stock were offered by Ultragenyx. The company's common stock is listed on The NASDAQ Global Select Market under the ticker symbol "RARE." Ultragenyx estimates net proceeds from the offering to be approximately $126.4 million, after deducting underwriting discounts and commissions and estimated offering expenses.
