New User:

-or-
Username:
Password:
Forgot your password?

Stock Market & Financial Investment News

News Breaks
March 27, 2014
09:06 EDTRAREUltragenyx announces preliminary data from phase 1/2 study of rhGUS, UX003
Ultragenyx Pharmaceutical announced the presentation of preliminary data from the Phase 1/2 study of recombinant human beta-glucuronidase, or rhGUS, UX003, an investigational therapy for the treatment of mucopolysaccharidosis 7. The Phase 1/2 open-label clinical study is assessing the safety, efficacy, and dose of rhGUS administered every other week via intravenous infusion in a 12-week primary analysis phase, which will be followed by dose-exploration and long-term extension. Preliminary results from three patients who have been administered 2 mg/kg of rhGUS every other week for two, six, and 12 weeks show evidence of clearance of lysosomal storage as indicated by the decline in urinary glycosaminoglycan excretion beginning at two weeks of treatment of approximately 30-50%. At the 12 week assessment of the first patient, absolute liver size was reduced by approximately 11%. This represents a 46% decrease in the excess liver size above normal for age and gender. The remaining patients have not yet reached the 12 week time point for liver size assessment. No serious adverse events were observed during up to 12 weeks of treatment, and no infusion-associated reactions were observed after a total of 13 infusions to date in these three subjects. The study will continue and additional 12-week interim data are expected in 2014. If the efficacy and safety results for all treated subjects are supportive and the dose confirmed, the company intends to initiate a pivotal Phase 3 study enrolling at least 12 patients in 2014.
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.
September 1, 2015
10:05 EDTRAREOn The Fly: Analyst Initiation Summary
Today's noteworthy initiations include: 8point3 Energy (CAFD) initiated with an Equal Weight at Barclays... AMAG Pharmaceuticals (AMAG) initiated with an Outperform at Raymond James... Acadia (ACHC) initiated with an Outperform at Leerink... Acorda Therapeutics (ACOR) initiated with a Market Perform at Raymond James... Alexion (ALXN) initiated with a Strong Buy at Raymond James... Amedisys (AMED) initiated with an Outperform at Wells Fargo... Amgen (AMGN) initiated with a Market Perform at Raymond James... Amplify Snack Brands (BETR) initiated with a Neutral at Credit Suisse... BioMarin (BMRN) initiated with an Outperform at Raymond James... Biogen (BIIB) initiated with a Strong Buy at Raymond James... Celgene (CELG) initiated with a Strong Buy at Raymond James... CoreLogic (CLGX) initiated with a Neutral at SunTrust... Digi International (DGII) initiated with an Overweight at Stephens... First Solar (FSLR) initiated with an Overweight at Barclays... LHC Group (LHCG) initiated with an Outperform at Wells Fargo... Lancaster Colony (LANC) initiated with a Buy at BB&T... McCormick (MKC) initiated with a Buy at BB&T... NephroGenex (NRX) initiated with a Buy at H.C. Wainwright... Omega Protein (OME) initiated with a Hold at BB&T... Preferred Apartment (APTS) initiated with an Outperform at FBR Capital... Qorvo (QRVO) initiated with an Outperform at CLSA... Regeneron (REGN) initiated with an Outperform at Raymond James... Skyworks (SWKS) initiated with an Outperform at CLSA... SunPower (SPWR) initiated with an Equal Weight at Barclays... TESARO (TSRO) initiated with a Strong Buy at Raymond James... Ultragenyx (RARE) initiated with an Outperform at Raymond James... VEREIT (VER) initiated with a Buy at Goldman... Westmoreland (WLB) initiated with an Outperform at BMO Capital.
09:08 EDTRAREUltragenyx presents data from LC-FAOD patients treated with triheptanoin
Ultragenyx Pharmaceutical announced the presentation of open-label data from five infants with cardiomyopathy due to long-chain fatty acid oxidation disorders, or LC-FAOD, treated with triheptanoin. Severely affected LC-FAOD patients can present early in life with severe cardiomyopathy, arrhythmia, heart failure, hypoglycemia, hepatic dysfunction, and rhabdomyolysis that can lead to death. The data were presented at the Society for the Study of Inborn Errors of Metabolism, or SSIEM, Annual Symposium in Lyon, France. Case reports from five infant patients with moderate or severe cardiomyopathy due to LC-FAOD were presented. All patients were detected by newborn screening and managed with standard treatment, including medium-chain triglyceride oil. While on the standard of care, the patients were hospitalized with heart failure that required cardiac support and, in some cases, resuscitation. The patients discontinued medium-chain triglyceride oil and then began to receive triheptanoin on an expanded access basis.
August 31, 2015
19:26 EDTRAREUltragenyx initiated with an Outperform at Raymond James
Subscribe for More Information

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