Date: 2016-10-12
Type of
information: DSMB assessment
phase: 2
Announcement: DSMB assessment
Company: ProMetic Life Sciences (Canada)
Product: PBI-4050 - 3-pentylbenzenacetic acid sodium salt
Action
mechanism:
- antifibrotic agent. PBI-4050 is an orally active lead drug candidate with excellent safety and efficacy profiles confirmed in several in vivo experiments targeting fibrosis.
Disease: Alström syndrome
Therapeutic
area: Rare diseases - Genetic diseases
Country: UK
Trial
details:
- The ongoing Alström syndrome phase 2 clinical trial is an open label, single arm and single center study investigating the safety, tolerability and efficacy of ProMetic’s small molecule lead compound PBI-4050 in a total of 20 patients. The trial is being performed at the specialty center for the care of UK patients with Alström syndrome at the Queen Elizabeth Hospital, Birmingham, UK. This center has recently published data showing that many of these patients show evidence of non-alcoholic fatty liver disease and advanced liver fibrosis at an early age, confirming previous publications showing a very high incidence of progression of NAFLD into liver cirrhosis with associated mortality in Alström patients.
Latest
news:
- • On October 12, 2016, ProMetic Life Sciences announced today that the Drug Safety Monitoring Board (“DSMB”) recommended that patient enrolment should continue in its ongoing Alström syndrome phase 2 clinical trial. This recommendation follows the DSMB’s review of the safety data accumulated in the first eight (8) Alström syndrome patients that had received treatment with PBI-4050. The DSMB determined that no safety or tolerability issues had been observed in these patients.
The early efficacy results in this phase 2, open-label study demonstrate that the first five (5) patients (100%) who completed 12 weeks of treatment with PBI-4050 had a significant reduction of liver fibrosis, as measured by transient elastography (FibroScan®). This reduction was also sustained for the first patient, who continued to show reduced fibrosis after having completed 24 weeks of treatment. The initial efficacy results also demonstrate that those patients with the most elevated liver enzymes at baseline (aspartate aminotransferase [AST], alanine aminotransferase [ALT], and alkaline phosphatase) [ALP], had a significant reduction in all of these enzymes, to within normal ranges, after completing 4, 8 and 12 weeks of treatment with PBI-4050.
Is
general: Yes
