New User:

Forgot your password?

Stock Market & Financial Investment News

News Breaks
July 1, 2014
09:12 EDTRAREUltragenyx initiates Phase 2 study of KRN23
Ultragenyx Pharmaceutical announced the first patient screened and enrolled in the Phase 2 study of the human monoclonal anti-FGF23 antibody KRN23 in pediatric patients with X-linked hypophosphatemia, or XLH. XLH is an inherited metabolic bone disease characterized by short stature, skeletal deformities, bone pain, fractures, and muscle weakness. The multi-center, randomized, open-label, dose-finding Phase 2 clinical study will evaluate safety and efficacy in approximately 30 prepubertal pediatric XLH patients. The primary objectives of the study are to identify a dose and dosing regimen and to establish the safety profile of treatment with KRN23 in pediatric XLH patients. Preliminary clinical effects of KRN23 treatment on bone health and deformity as measured by radiographic assessments, growth, muscle strength, and motor function will also be assessed, as well as markers of bone health and patient-reported outcomes of pain, disability, and quality of life. The study has been evaluated and accepted for conduct by the FDA, the United Kingdom Medicinal and Health Regulatory Authority, or MHRA, and the Dutch Medicines Evaluation Board, or CBG-MEB.
News For RARE From The Last 14 Days
Sign up for a free trial to see the rest of the stories you've been missing.
October 2, 2015
09:05 EDTRAREUltragenyx says it filed MAA with EMA for GNE Myopathy tablets
Subscribe for More Information
September 22, 2015
06:19 EDTRAREClinton to roll out plan to rein in prescription drug costs, USA Today reports
Subscribe for More Information
September 21, 2015
14:04 EDTRAREClinton vow to end drug price gouging sinks biotechs
Biotech stocks have declined across the board in afternoon trading following a tweet by Presidential candidate Hillary Clinton criticizing the "outrageous" prices of specialty drugs. TURING STEPS UP PRICING: Clinton's tweet directly referenced Turing Pharmaceuticals' Daraprim treatment for toxoplasmosis, which recently had its price hiked from just $13.50 per pill to $750 after the privately-held company acquired the drug from Impax Laboratories (IPXL). Importantly, Daraprim was first approved by the FDA in 1953 and its patents have long since expired. No generic versions of the drug currently exist, perhaps in part due to Turing executive chairman Martin Shkreli's strategy of tightly controlling distribution, according to a New York Post article on the issue. In a Bloomberg TV interview this morning, Shkreli claimed that the drug remains underpriced compared to what he called its peers in the rare disease space. However, note that a World Health Organization analysis from 2013 reported that over 190,000 cases of toxoplasmosis occur globally every year and Daraprim continues to be listed on the organization's list of essential medicines for a basic health-care system. INDUSTRY CRITICISM: Turing's Daraprim pricing prompted a response from the Infectious Diseases Society of America and the HIV Medicine Association, which criticized the "unjustifiable" new cost of the drug and urged a "rational" pricing strategy. The news surrounding Daraprim also comes on the heels of Presidential candidate Bernie Sanders issuing a letter in August to Valeant (VRX) and Hospira (HSP), in which he requested information on the "enormous" price increases of two drugs Valeant acquired earlier in the year. REPUBLICAN CONGRESS: In a research note released this afternoon, RBC Capital analyst Michael Yee reminded investors that Republicans still control Congress, offering a sobering reminder that campaign trail promises often fall by the wayside once the candidate steps into office. Yee explained that, though drug pricing debates during next year's election will probably generate headline risk for the biotech space, the Republican-controlled Congress is unlikely to sign into law any material changes on the issue. BIOTECH STOCK ACTION: Hillary Clinton's promise to "lay out a plan" tomorrow to tackle "price gouging" in the specialty drug market sent numerous biotech stocks plunging. Aerie Pharmaceuticals (AERI) has crashed 12.6% at time of writing, while Retrophin (RTRX), Ultragenyx (RARE), and PTC Therapeutics (PTCT) are down 14.7%, 13.4%, and 11%, respectively. Biotech giants AbbVie (ABBV), Gilead (GILD), Bristol-Myers Squibb (BMY) and Biogen (BIIB) have also slipped in intraday trading, while the NASDAQ Biotechnology index is down roughly 4%.

Sign up for a free trial to see the rest of the stories you've been missing.
I agree to the disclaimer & terms of use