Safety and efficacy results of LIS1, a new induction treatment in organ transplantation by the French biotech XENOTHERA

9 June 2022

Results from the clinical trial of LIS1, XENOTHERA’s polyclonal glyco-humanised antibody and induction immunosuppressant in solid organ transplantation, were presented at the American Transplant Congress (ATC) (ATC) held in Boston (USA) from 4 to 8 June 2022.

At the American Transplant Congress, Professor Ondrej Viklicky from the IKEM Institute – one of Europe’s leading kidney transplant centers for several decades (Prague, Czech Republic) – principal investigator of the NCT04431219 trial, presented the first results of the LIS1 Phase I/II First In Human (FIH) clinical trial, which ran from September 5, 2019 to March 28, 2022 (last patient visit).

The clinical trial of the transplant dedicated immunosuppressant LIS1 consisted of two cohorts of first-time kidney transplant patients with limited immunological risk who received 5 consecutive administrations of LIS1 starting the day after transplantation. The AD1 cohort of 5 patients received escalating doses of LIS1 ranging from 0.6 to 8 mg/kg. The TD2 cohort of 5 patients received therapeutic doses of LIS1.

The primary endpoint was product safety, LIS1 was well tolerated. The majority of adverse events were mild or moderate, and all recovered. All ten patients, 4 females and 6 males, recovered their renal function within the usual time frame after transplantation. No side effects such as leucopenia or thrombocytopenia were observed. In total, the study results of this trial provide a reassuring global safety profile for LIS1.

In parallel, the first pharmacokinetic and pharmacodynamic results are promising. Pharmacokinetic analyses showed a long half-life and no anti-drug antibodies (ADA3) were detected. The pharmacodynamics show a unique new mechanism of action, combining lymphocyte depletion and inhibition of alloreactivity, which gives LIS1 a promising status as a new induction treatment in solid organ transplantation.

XENOTHERA is the sponsor of the first human clinical study of LIS1, a product issued from its glyco-humanized antibody platform, indicated for the prevention of acute rejection in transplantation.