dm+d

Unassigned

New Medicines

Familial primary hypoalphalipoproteinaemia

Information

New molecular entity
Cerenis Therapeutics
Cerenis Therapeutics

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes

Category

A recombinant high-density lipoprotein (HDL) mimetic: a complex of a human apolipoprotein A-I (ApoA-I) and phospholipids
FPHA includes patients with a heritable genetic defect in one of the key genes involved in HDL particle production or maturation (apoA-I, ABCA-1, LCAT). It is defined clinically as a HDL-cholesterol <1.0mmol/L in men and <1.3mmol/L in women. Patients with FPHA have a very low plasma concentration of apolipoprotein A-I (apoA-I)[3]. Prevalence of Apo A-I deficiency is unknown (so less than 1 per 1million people), and has been described in about 30 families [1].
Familial primary hypoalphalipoproteinaemia
Intravenous infusion

Trial or other data

Dec 18: In the Phase III TANGO study, the primary endpoint (change from baseline after 24 wks vs. placebo on carotid Mean Vessel Wall Area (MVWA)) was not met [5].


Oct 17;According to a Cerenis Therapeutics results from PIII TANGO trial are expected at the end of Q1 2018 [4]


Sep 15: Cerenis Therapeutics initiates the PII/III TANGO trial to evaluate the efficacy, pharmacokinetics and safety of CER 001 8 mg/kg IV infusion to prevent or reduce atherosclerosis in patients with familial primary hypoalphalipoproteinemia (CER001CLIN009; EudraCT2015-003713-23). The primary endpoint is to determine the change from baseline after 24 weeks treatment with CER 001 on carotid Mean Vessel Wall Area (MVWA) using 3T-MRI. The 48-week, double-blind, parallel, placebo-controlled, randomised trial is recruiting 30 patients in the Netherlands and will extend to Brazil, Canada, Israel, Turkey and the US [2].

Evidence based evaluations