FDA approvals and encouraging results for innovative therapies

Idorsia announced today that the US Food and Drug Administration (FDA) has approved TRYVIO (aprocitentan) for the treatment of hypertension in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not adequately controlled on other drugs.1 Lowering blood pressure reduces the risk of fatal and non-fatal cardiovascular events, primarily strokes and myocardial infarctions.

This is the first oral anti-hypertensive therapy which works via a new therapeutic pathway to be approved in almost 40 years. TRYVIO (aprocitentan) is an endothelin receptor antagonist that inhibits the binding of endothelin (ET)-1 to ETA and ETB receptors. Until the approval of TRYVIO, no systemic antihypertensive medications targeted the ET pathway.

TRYVIO was evaluated as a monotherapy in a Phase 2 study in patients with hypertension, and as an add-on therapy in a Phase 3 study called PRECISION in patients with confirmed resistant hypertension. In PRECISION, aprocitentan was well tolerated and superior to placebo in lowering blood pressure at week 4, with a sustained effect at week 40.

Orphan Drug Designation for iOnctura

After assessing its novel chemical and biological properties including an attractive non-clinical safety and efficacy profile, the US FDA has granted Orphan Drug Status to iOnctura’s autotaxin inhibitor cambritaxestat for the treatment of pancreatic cancer, conferring certain benefits during development and commercialization.

Cambritaxestat is being developed as a first-in-class therapy for highly fibrotic cancer indications. The drug's lead indication is metastatic pancreatic cancer where it is being combined with standard of care nab-paclitaxel and gemcitabine in the Phase I AION-02 study. 

Inhibition of autotaxin is a novel treatment strategy that offers a three-pronged attack on the tumor through direct cancer cell inhibition, immune effector stimulation and inhibition of fibrotic processes, giving drugs and immune cells better access to the tumor.

Translational research showing the potential of cambritaxestat in multiple cancer models, including pancreatic cancer, has recently been published in the ESMO journal Immuno-Oncology and Technology (IOTECH), Cancer Research, the Journal of Experimental & Clinical Cancer Research, and Cancers. Across these publications, cambritaxestat showed strong reduction of metastasis and tumor outgrowth in preclinical models, as well as safe and tolerable dosing in healthy volunteers.

Encouraging phase 1 results for TORQUR

TORQUR AG announces the publication of promising phase 1 clinical trial results for bimiralisib, its innovative anti-cancer drug candidate. The data, showcasing bimiralisib's potential as a targeted therapy for various solid tumors, were published in the prestigious journal *Cancers* on March 13, 2024.

The trial marks a significant milestone in cancer therapy, involving collaboration with top-tier cancer centers worldwide, including The University of Texas MD Anderson Cancer Center, Mayo Clinic Rochester, Roswell Park Comprehensive Cancer Center, Cleveland Clinic, Taussig Cancer Center, Vall d’Hebron Institute of Oncology, University Hospital Basel, UCL Cancer Institute, and Oxford University Hospitals.

Bimiralisib targets the PI3K, Akt, and mTOR signaling pathways, which are crucial for cell growth and survival. In this trial, bimiralisib has demonstrated an encouraging safety profile, with manageable adverse events and favorable pharmacokinetics (PK) profile, paving the way for future development in biomarker-selected patient populations and combinations with other therapies.

(Press release / SK)