Mon, Nov. 16, 8:51 AM
- Raptor Pharmaceuticals (NASDAQ:RPTP) is down 15% premarket on light volume in response to its announcement of additional data from its CyNCh study assessing lead product candidate RP103 for the treatment of children with moderate-to-severe nonalcoholic fatty liver disease (NAFLD). The results will be presented today at The Liver Meeting in San Francisco.
- As previously reported on September 14, there was no statistically valid difference in response rate between the RP103 cohort and placebo (28% vs. 22%; p=0.34). No statistically significant difference was observed in the components of the NAS score or fibrosis, but an improvement in the number of subjects with lobular inflammation was seen (36% vs. 21%), although this also fell short of statistical significance when adjusted for multiple comparisons.
- A post-hoc analysis showed a statistically significant difference in the primary endpoint of improvement in liver histology versus placebo in patients weighing no more than 65 kg (p=0.01), about 28% of the study population. No improvement in liver histology was seen in subjects weighing more than 65 kg.
- Statistically significant improvements compared to placebo in the liver enzymes ALT, AST and GGT (p=0.02, p=0.008 and p=0.02, respectively) from baseline to week 52 were also seen.
- Raptor has yet to recover from its plunge in September in response to its initial report of results from CyNCh. Shares are still down over 60% from early summer.
Mon, Sep. 14, 12:45 PM
Mon, Sep. 14, 9:14 AM
Mon, Sep. 14, 7:43 AM
- Raptor Pharmaceutical (NASDAQ:RPTP) reports that its product candidate for the treatment of nonalcoholic steatohepatitis (NASH) in adolescents aged 8 - 17, RP103 (cysteamine bitartrate), failed to achieve its primary endpoint of a two point decrease in NAFLD Activity Score (NAS) and no worsening of fibrosis compared to placebo (p=0.34) in a Phase 2b study.
- The trial, called CyNCh, assessed RP103 in 169 children. Subjects were randomized to receive either 600 mg/day, 750 mg/day, 900 mg/day of RP103 or placebo for 52 weeks.
- The results will be submitted for publication and presented at the American Association for the Study of Liver Diseases meeting in November in San Francisco, CA.
- Based on the results, the company will cease development of RP103 for NASH. Its development for Huntington's disease and mitochondrial diseases in ongoing.
- Shares are off 36% premarket on robust volume.
Wed, Jun. 10, 5:38 PM
Tue, Mar. 24, 5:35 PM
Wed, Jan. 7, 8:50 AM
- Thinly-traded nano cap Galectin Therapeutics (NASDAQ:GALT) is up 49% premarket on robust volume in response to its report of results from a 30-patient Phase 1 study of GR-MD-02 in patients with nonalcoholic steatohepatitis (NASH).
- The principal objective of the trial was to assess the safety, tolerability and pharmacokinetics of GR-MD-02 but investigators also noted a potential therapeutic effect on fibrosis as measured by a serum biomarker (FibroTest) and liver stiffness (FibroScan). Patients in cohort 3 who received 8 mg/kg GR-MD-02 showed a statistically significant reduction in FibroTest score versus placebo in one of the cohorts.
- Fibrotest is a composite score of six blood tests that correlate to the extent of liver fibrosis.
- NASH represents the next big potential payoff for drug firms after HCV.
- Previously: Galectin NASH treatment meets primary endpoint in Phase 1 (Nov. 10, 2014)
- NASH-associated tickers: (NASDAQ:GILD) (NASDAQ:SHPG) (NASDAQ:LJPC) (NASDAQ:CNAT) (NASDAQ:ICPT) (NASDAQ:RPTP)
Aug. 8, 2014, 3:49 PM
- First the good news:
- Tekmira (TKMR +38.1%) on a massive 25M share turnover; Raptor Pharmaceuticals (RPTP +28.2%) on a 4x surge in volume; Clovis Oncology (CLVS +11.6%) on 50% higher volume; RadNet (RDNT +20.4%) on 2x higher volume and Relypsa (RLYP +15%) on 2x higher volume.
- Now the bad news:
- TearLab (TEAR -21.2%) on 4x higher volume; Masimo (MASI -10.6%) on 3x higher volume; Volcano (VOLC -19.9%) on a 12x surge in volume and Hanger (HGR -24.9%) on a 16x surge in volume.
Aug. 8, 2014, 12:47 PM
Jun. 20, 2014, 5:36 PM
Jun. 17, 2014, 5:35 PM
Mar. 14, 2014, 12:48 PM
Mar. 13, 2014, 5:42 PM
Feb. 20, 2014, 12:47 PM
Feb. 20, 2014, 11:28 AM
- "Procysbi should become an important alternative for treating patients with nephropathic cystinosis," writes analyst William Tanner reiterating his Outperform and $17 price target. "The pace of market uptake has somewhat debunked the notion that the cost of therapy would adversely position the drug vs. Cystagon."
- Today's "encouraging" data on RP103 for Huntington's Disease, may also include evidence the drug could be "effective for treating a broad range of conditions characterized by mitochondrial disorders and intracellular cystine accumulation."
- RPTP +13.4%
Feb. 20, 2014, 9:11 AM
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