Amicus Therapeutics (FOLD) Updated Phase 2 on HCI for Fabry Disease
Tweet
Share
E-mailGet Alerts FOLD Hot Sheet
Join SI Premium – FREE
Amicus Therapeutics (Nasdaq: FOLD) today announced updated data from an open-label, Phase 2 extension study (Study 205/FAB-CL-205) to investigate the long-term safety, tolerability and pharmacodynamics of migalastat HCl in 23 patients with Fabry disease who completed an initial Phase 2 study. Amicus in collaboration with GlaxoSmithKline (GSK) is developing the investigational pharmacological chaperone Migalastat HCl for the treatment of Fabry disease. Results1 from Study 205 were presented at the American Society of Nephrology (ASN) Kidney Week 2012.
Pol F. Boudes, MD, Chief Medical Officer of Amicus Therapeutics stated, "Fabry is a broad spectrum disease that affects males and females resulting in various symptoms. In our Phase 2 studies, kidney biopsies from both males and females with amenable mutations have shown decreases in interstitial capillary GL-3, a key biomarker of disease. Collectively these results support the ongoing investigation of migalastat HCl monotherapy in Phase 3 studies. We believe that migalastat HCl has the potential to become an important new treatment option for Fabry patients with amenable mutations."
Key Study 205 Results Presented at ASN:
Enrolled 23 total subjects who completed primary treatment period (12 or 24 weeks) and treatment extension (24 to 84 weeks) in 4 Phase 2 studies (Studies FAB-CL-201-204).
No drug-related serious adverse events. The most common adverse events in 6 out of 23 subjects were arthralgia, fatigue, back pain, and pain in extremity.
8 patients had evaluable paired kidney biopsies from baseline in primary study and follow-up in Study 205 (60-week median treatment duration in Study 205 prior to biopsy). Median decrease in interstitial capillary globotriaosylceramide (GL-3) was 78% in the 5 patients (2 males and 3 females) with genetic mutations amenable to migalastat HCl monotherapy in a cell-based assay. Median increase in interstitial capillary GL-3 was 114% in 3 females with non-amenable mutations.
Renal function remained stable in patients with amenable mutations as demonstrated by estimated glomerular filtration rate (eGFR) and proteinuria.
Median treatment duration on migalastat HCl was 5.2 years (4.7 to 6.4 years) in 17 subjects who completed Study 205 – final data analysis ongoing
16 out of 17 subjects who completed Study 205 are now enrolled in a separate open-label extension study (MGM116041)
GL-3 in Renal Peritubular Capillary Cells (PTCs, or Interstitial Capillaries)
GL-3 is the lipid substrate that accumulates in tissues affected by Fabry disease, including the kidney. GL-3 inclusions in PTCs - also referred to as interstitial capillaries - are measured by histology from kidney biopsies. Reduction of GL-3 in renal PTCs previously supported conditional approval of enzyme replacement therapy (ERT) for Fabry disease by the U.S. Food and Drug Administration (FDA).
Previous scientific presentations2 highlighted changes in interstitial capillary GL-3 from baseline to various time points in initial Phase 2 studies (Studies 202-204). Changes in interstitial capillary GL-3 from baseline (in Studies 202-204) to follow-up (Study 205) were presented for the first time at ASN 2012. Pathologists blinded to biopsy sequence assessed a total of 8 evaluable paired kidney biopsies by histology using the published, quantitative Barisoni Lipid Inclusion Scoring System (BLISS)3 by virtual microscopy.
Renal Function
Estimated glomerular filtration rate (eGFR) remained stable. In 9 male evaluable patients, decreases in proteinuria from baseline were observed in 8 patients with amenable mutations, with an increase reported in 1 non-amenable patient.
Pol F. Boudes, MD, Chief Medical Officer of Amicus Therapeutics stated, "Fabry is a broad spectrum disease that affects males and females resulting in various symptoms. In our Phase 2 studies, kidney biopsies from both males and females with amenable mutations have shown decreases in interstitial capillary GL-3, a key biomarker of disease. Collectively these results support the ongoing investigation of migalastat HCl monotherapy in Phase 3 studies. We believe that migalastat HCl has the potential to become an important new treatment option for Fabry patients with amenable mutations."
Key Study 205 Results Presented at ASN:
Enrolled 23 total subjects who completed primary treatment period (12 or 24 weeks) and treatment extension (24 to 84 weeks) in 4 Phase 2 studies (Studies FAB-CL-201-204).
No drug-related serious adverse events. The most common adverse events in 6 out of 23 subjects were arthralgia, fatigue, back pain, and pain in extremity.
8 patients had evaluable paired kidney biopsies from baseline in primary study and follow-up in Study 205 (60-week median treatment duration in Study 205 prior to biopsy). Median decrease in interstitial capillary globotriaosylceramide (GL-3) was 78% in the 5 patients (2 males and 3 females) with genetic mutations amenable to migalastat HCl monotherapy in a cell-based assay. Median increase in interstitial capillary GL-3 was 114% in 3 females with non-amenable mutations.
Renal function remained stable in patients with amenable mutations as demonstrated by estimated glomerular filtration rate (eGFR) and proteinuria.
Median treatment duration on migalastat HCl was 5.2 years (4.7 to 6.4 years) in 17 subjects who completed Study 205 – final data analysis ongoing
16 out of 17 subjects who completed Study 205 are now enrolled in a separate open-label extension study (MGM116041)
GL-3 in Renal Peritubular Capillary Cells (PTCs, or Interstitial Capillaries)
GL-3 is the lipid substrate that accumulates in tissues affected by Fabry disease, including the kidney. GL-3 inclusions in PTCs - also referred to as interstitial capillaries - are measured by histology from kidney biopsies. Reduction of GL-3 in renal PTCs previously supported conditional approval of enzyme replacement therapy (ERT) for Fabry disease by the U.S. Food and Drug Administration (FDA).
Previous scientific presentations2 highlighted changes in interstitial capillary GL-3 from baseline to various time points in initial Phase 2 studies (Studies 202-204). Changes in interstitial capillary GL-3 from baseline (in Studies 202-204) to follow-up (Study 205) were presented for the first time at ASN 2012. Pathologists blinded to biopsy sequence assessed a total of 8 evaluable paired kidney biopsies by histology using the published, quantitative Barisoni Lipid Inclusion Scoring System (BLISS)3 by virtual microscopy.
Renal Function
Estimated glomerular filtration rate (eGFR) remained stable. In 9 male evaluable patients, decreases in proteinuria from baseline were observed in 8 patients with amenable mutations, with an increase reported in 1 non-amenable patient.
Serious News for Serious Traders! Try StreetInsider.com Premium Free!
You May Also Be Interested In
- FDA authorizes 11 ZYN Ultra nicotine pouches for Philip Morris
- Gossamer Bio raises up to $250M in private placement for PAH drug
- Blue Star Gold reports phase 2 drilling results at Nutaaq target
Create E-mail Alert Related Categories
FDASign up for StreetInsider Free!
Receive full access to all new and archived articles, unlimited portfolio tracking, e-mail alerts, custom newswires and RSS feeds - and more!
