Ultragenyx Initiates Novel Disease Monitoring Program for Hereditary Inclusion Body Myopathy

Ultragenyx Initiates Novel Disease Monitoring Program for Hereditary Inclusion Body Myopathy

 

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.

Planning IPO, Chimerix says its antiviral makes FDA fast track

RESEARCH TRIANGLE PARK, N.C., March 12, 2013 — Chimerix, the antiviral drug company that last week filed for an initial public offering of stock, is now on a faster regulatory path for its lead drug candidate.

The Food and Drug Administration’s “fast track” designation for CMX001 gives the Research Triangle Park company’s compound priority review. That faster review is reserved for new drugs addressing serious or life threatening conditions that have few if any alternative treatments. CMX001 is a compound that Chimerix is developing to prevent cytomegalovirus (CMV) infection, an infection that can severely sicken patients with already compromised immune systems. The antiviral works by blocking replication of double-stranded DNA (dsDNA) viruses.

Chimerix has completed phase II clinical trials studying CMX001 as a way of preventing CMV in patients undergoing hematopoietic stem cell transplant procedures. The company expects to start a phase III study later this year in patients undergoing these transplants.

Man with a plan: Emil Kakkis aims to lure big-market drugs toward rare diseases

 

February 26, 2013

Emil Kakkis is making another run at rare diseases.

The president and CEO of Ultragenyx Pharmaceutical Inc. and former chief medical officer at BioMarin Pharmaceutical Inc. is laying out a new strategy Tuesday to get more drugs into children with rare diseases. Under the banner of the EveryLife Foundation for Rare Diseases, which Kakkis founded and has largely bankrolled, he is proposing that companies with already-approved, big-market drugs be rewarded with a two-month extension of that drug’s patent life if it wins a new Food and Drug Administration-approved label in an orphan disease.

Ernest Mario named Chairman of Chimerix

 

February 20, 2013

Chimerix, a Durham, N.C.-based biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, has appointed Ernest Mario, Ph.D., as chairman of its board of directors.

During the course of his career, Mario has served as the CEO, chairman and a director of several pharmaceutical companies. From 1989 to 1993, he served as chief executive of Glaxo Holdings, then the second-largest drug company in the world. Mario led drug delivery technology company ALZA from 1993 until its acquisition by Johnson & Johnson in 2001. He served as chairman and CEO of Reliant Pharmaceuticals from 2003 until its acquisition by GlaxoSmithKline in 2007. He also previously served as the chairman of multinational CRO Pharmaceutical Product Development (PPD) from 1993 to 2011.

Mario currently serves as chairman and CEO of Capnia, a privately held pharmaceutical company, as well as chairman of the American Foundation for Pharmaceutical Education.

LipoScience Announces Closing of Initial Public Offering

 

RALEIGH, N.C.  – January  30, 2013  – LipoScience, Inc. (NASDAQ:  LPDX) today announced it has closed its previously announced initial public offering of 5,000,000 shares of common stock at a price to the public of $9.00 per share.  In connection with the initial public offering, the underwriters exercised in full their option to purchase an additional 750,000 shares from the company.  As a result, the total initial public offering size was  5,750,000 shares. LipoScience expects to receive, after deducting underwriting discounts and commissions and estimated offering expenses payable by  the company, net proceeds of approximately $44.9 million from the offering.

Ultragenyx Announces In-Licensing of Clinical-Stage Product Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorders

NOVATO, CA –January 10, 2013— Ultragenyx Pharmaceutical Inc., a biotechnology company focused on the development of treatments for rare and ultra-rare genetic disorders, announced that it has in-licensed rights for triheptanoin,  a promising treatment for long-chain fatty acid oxidation disorders (FAOD), from Baylor Research Institute in Dallas, TX. Triheptanoin is intended as a substrate replacement therapy to restore deficient intermediates in the mitochondria and to enable energy metabolism in patients with FAOD and potentially other genetic metabolic disorders.

LIPOSCIENCE COLLABORATES WITH A LEADING ACADEMIC MEDICAL CENTER TO IDENTIFY RISK FACTORS AIMED AT IMPROVING MANAGEMENT OF ACUTE CORONARY SYNDROME

RALEIGH, N.C. – January 09, 2013 – LipoScience, Inc., an in vitro diagnostic company committed to advancing patient care in cardiovascular, metabolic and other diseases, today announced that it has entered into a research agreement with Duke University School of Medicine. The research will be aimed at identifying cardiovascular risk factors that can be used alone or in combination with other clinical
measures to improve stratification and management of acute coronary syndrome. The agreement encompasses studies to be performed with Duke’s CATHGEN (CATHeterizationGENetics) and other patient registries.

Ultragenyx Raises $75 Million in Oversubscribed Crossover Financing

NOVATO, CA – December 20, 2012 - Ultragenyx Pharmaceutical Inc., a biotechnology company focused on the development of treatments for rare and ultra-rare genetic disorders, today announced the successful completion of a $75 million Series B round financing led by Adage Capital Partners, LP.  Joining Adage as new investors in this financing are mutual funds and separate account clients advised by T. Rowe Price Associates, Inc., Jennison Associates LLC (on behalf of clients), funds and accounts under management by subsidiaries of BlackRock, Inc., Sanofi-Genzyme BioVentures 1, Shire plc and additional blue chip public market funds.  Existing investors, TPG Biotech, Fidelity Biosciences, HealthCap and Pappas Ventures also participated in the transaction.

The Wistar Institute Welcomes Three New Board Members

PHILADELPHIA—(November 16, 2012) – The Wistar Institute recently welcomed Van Billet, Joseph Goldblum and Art Pappas to its board of trustees.

Art Pappas has over 30 years of operating experience as a pharmaceutical and biotechnology industry executive and venture capital investor in life science companies. Currently, he serves as managing partner at Pappas Ventures, a life sciences venture capital firm with more than $300 million in capital under management. The firm has invested in over 50 biotechnology, biopharmaceutical, specialty pharmaceutical, drug delivery and medical device companies throughout the U.S.

 

Chimerix Appoints Michelle Berrey Chief Medical Officer

RESEARCH TRIANGLE PARK, NC, November 12, 2012 — Chimerix, Inc., a biotechnology company developing orally-available antiviral therapeutics, announced today that Michelle Berrey, MD, MPH, has joined the company as Chief Medical Officer.

"We are delighted to have Michelle join our leadership team. Dr. Berrey is a seasoned drug developer who has established a strong track record in antivirals, in particular with nucleotide analogs. She joins Chimerix at an important time for the company as our lead compound, CMX001, advances into Phase 3 clinical testing,” said Kenneth I. Moch, Chimerix President and Chief Executive Officer. “Her extensive background in antiviral drug development will be invaluable to our already strong team.”