Thu, Jan. 15, 7:30 AM
Mon, Jan. 12, 2:31 PM
Thu, Jan. 8, 11:31 AM
- Data from the Phase 3 trial (Study 011) and extension (Study 041) evaluating Amicus Therapeutics' (FOLD +5.6%) migalstat HCl for Fabry disease showed a positive effect on a key cardiac disease risk marker. Patients treated with migalstat HCl experienced a statistically significant mean decrease of 8% versus baseline in left ventricular mass index (LVMi) for up to 36 months.
- LVMi is a measure of cardiac hypertrophy, an increase in the size of the heart that is associated with a heightened risk of cardiac events in Fabry patients.
- Migalstat HCl demonstrated a similar effect on LVMi in an earlier trial (Study 012).
- Previously: Amicus Therapeutics' Fabry disease drug candidate improves cardiac health marker (Nov. 17, 2014)
Thu, Jan. 8, 9:15 AM
Dec. 23, 2014, 5:35 PM
Dec. 15, 2014, 12:36 PM
- Added to the Nasdaq Biotechnology Index, effective Dec. 22: AAVL, AERI, AKBA, ALDR, ALIM, AMPH, ANIP, ASPX, CARA, CGEN, CLDN, CNCE, ENTA, FLML, FMI, FOLD, FPRX, GWPH, HPTX, ICPT, INO, INSM, KITE, KPTI, MGNX, OPHT, PCYC, PETX, POZN, PTX, QLTI, RARE, RCPT, RDUS, RLYP, RTRX, RVNC, SAGE, TBPH, TTPH, VSAR, XLRN, ZFGN, ZSPH.
- Deleted: AMRN, AUXL, CRIS, EXPR, ICEL, ONTX, RIGL, ZGNX.
- Source: Press Release
Nov. 19, 2014, 7:13 AM
- Amicus Therapeutics (NASDAQ:FOLD) prices its public offering of 13.85M shares of common stock at $6.50 per share. Underwriters over-allotment is 2,077,500 shares. Closing date is November 24, 2014.
- Proceeds will be used to build the commercialization infrastructure for migalstat monotherapy for Fabry disease, the continued clinical development of its pipeline and for general corporate purposes.
Nov. 17, 2014, 5:40 PM
Nov. 17, 2014, 12:47 PM
Nov. 17, 2014, 9:15 AM
Nov. 17, 2014, 7:31 AM
- In a poster session at the American Society of Nephrology Kidney Week 2014, Amicus Therapeutics (NASDAQ:FOLD) presented additional data from its 60-patient Phase 3 clinical trial (Study 012) evaluating the safety and efficacy of its small molecule chaperone migalastat HCl as monotherapy for patients with Fabry disease.
- Patients who switched from enzyme replacement therapy (ERT), Fabrazyme (NYSE:SNY) and Replagal (NASDAQ:SHPG), to migalstat showed a statistically significant improvement from baseline in left ventricular mass index (LVMI), a measure of cardiac hypertrophy (increase in the size of the heart) that is associated with an increased risk of cardiac events in Fabry patients. Cardiac disease is a major cause of morbidity and mortality in this population.
- Migalstat baseline mean LVMI (g/m2) was 95.3 with a change of -6.6 to month 18. ERT baseline mean was 92.9 with a change of -2.0 at month 18.
- Migalstat also demonstrated a favorable outcome in a composite endpoint of Fabry-associated clinical events which are defined as a set of morbidities (worsening) in a Fabry patient's renal, cardiac and cerebrovascular status.
- The company expects to have a pre-submission meeting with European regulators this quarter and discussions with the FDA in early 2015.
- Shares are up 14% premarket on increased volume.
Nov. 6, 2014, 5:13 PM| Comment!
Nov. 3, 2014, 11:10 AM
Oct. 28, 2014, 9:13 AM
Oct. 20, 2014, 8:27 AM
- In two Phase 2a clinical trials, privately-held Genervon Biopharmaceuticals' drug candidate for Amyotrophic Lateral Sclerosis (ALS), GM6 (known as GM604 in the ALS trial and GM608 in a Parkinson's disease study), showed encouraging disease-modifying results albeit in a small number of patients.
- In the ALS study, GM604 significantly reduced the decline in ALSFRS-R versus the historical control (p=0.0047). Seven of eight patients has their ALS disease progression slowed or stopped at week 12 after six doses of GM604. Five of seven treated patients had their forced air capacity (FVC) disease progression slowed or reversed at week 12 compared to historical placebo (-11.5% compared to -4.7% after treatment).
- In the Parkinson's study, the difference in the treated patients' UPDRS scores versus historical placebo at week 12 were statistically significant (p=0.0085). Changes in secondary clinical outcomes measures at week 2 were statistically significant at the one-tailed 10% level for four of eight patients.
- Genervon has submitted these results to the FDA for guidance on how to make GM6 available for ALS and PD patients. GM604 for ALS has been designated Fast Track and an Orphan Drug by the FDA.
- ALS-related tickers:(NYSEMKT:CUR) (NASDAQ:BCLI) (NASDAQ:MNOV) (NASDAQ:BIIB) (NASDAQ:CYTK)
- PD-related tickers: (NASDAQ:ACOR) (Pending:CVTS) (NASDAQ:ACAD) (OTCQX:CYNAF) (NASDAQ:IPXL) (OTCQB:TTNP) (OTCQB:SGTH) (NYSE:TEVA) (NASDAQ:ADMS) (NASDAQ:PRAN) (NASDAQ:CHTP) (NASDAQ:PRTA) (NASDAQ:FOLD) (NASDAQ:AVNR) (NYSE:BSX) (NYSE:MRK)
Oct. 6, 2014, 5:36 PM
FOLD vs. ETF Alternatives
Other News & PR