Ionis Pharmaceuticals provided an update on two trials in HAE during reporting of financial results for the second quarter of 2023
Ionis Pharmaceuticals, who are developing Donidalorsen (formerly IONIS-PKK-LRx) for the prevention of HAE attacks, provided an update on two trials of the medicine while reporting on financial results for the second quarter of 2023.
The company confirmed that they had:
• Completed enrollment of the Phase 3 OASIS-HAE study of donidalorsen in patients with hereditary angioedema; on track for data readout in H1:2024
• Reported positive topline Phase 2 open label extension data of donidalorsen in patients with hereditary angioedema treated for two years
Brett P. Monia, Ph.D., Chief Executive Officer of Ionis Pharmaceuticals, said: “Ionis is creating significant value in 2023 as we successfully execute on our strategy to bring a steady cadence of transformational medicines to the market. Our growing late-stage pipeline now includes eight medicines for 10 indications.”
Donidalorsen, is an investigational ligand-conjugated antisense (LICA) medicine designed to target the prekallikrein (PKK) pathway. PKK plays an important role in the activation of inflammatory mediators associated with acute attacks of HAE. By inhibiting the production of PKK, donidalorsen could be an effective prophylactic approach to preventing HAE attacks.
(Source: Ionis Pharmaceuticals Inc)
