(RTTNews) – Vertex Pharmaceuticals Inc. (VRTX) announced positive data from a pre-specified Week 36 interim analysis of the ongoing Phase 3 RAINIER trial evaluating povetacicept, an engineered fusion protein and dual inhibitor of BAFF (B cell activating factor) and APRIL (a proliferation-inducing ligand) cytokines, in patients with immunoglobulin A nephropathy (IgAN).
The trial successfully met its primary objective. In the interim analysis population, patients treated with povetacicept achieved a 52.0% reduction from baseline in urine protein-to-creatinine ratio (UPCR) at Week 36. This represented a statistically significant and clinically meaningful 49.8% reduction compared to placebo. Importantly, the reduction in proteinuria was consistent across all pre-specified subgroups.
The study also met its secondary objectives. For the first secondary endpoint, patients receiving povetacicept demonstrated a 77.4% reduction from baseline in serum galactose-deficient IgA1 (Gd-IgA1), compared to an increase of +9.1% in the placebo group. This translated to a 79.3% reduction versus placebo (P0.0001). For the second secondary endpoint, among patients with baseline hematuria, 85.1% achieved hematuria resolution in the povetacicept group compared to 23.4% in the placebo group, yielding a 61.7% improvement over placebo.
According to the company, povetacicept was generally safe and well tolerated. The majority of adverse events (AEs) were mild to moderate. There were no serious adverse events (SAEs) related to povetacicept and no deaths in the trial. There were no opportunistic infections, and no discontinuations due to infections. As expected, anti-drug antibodies (ADAs) were observed; these ADAs had no impact on efficacy or the risk profile.
The U.S. Food and Drug Administration has granted rolling review of the Biologics License Application (BLA) for povetacicept in IgAN. Vertex has already submitted several modules and will complete the full BLA submission by the end of March for potential accelerated approval. As announced previously, Vertex is using a priority review voucher to expedite the review of the povetacicept BLA from ten months to six months.
If povetacicept is approved by the FDA, Vertex plans to launch povetacicept in a low volume (0.5 mL) subcutaneous auto-injector delivered once every four weeks via at-home administration.
The company noted that the RAINIER Phase 3 trial continues in a blinded manner, and final analysis will occur at two years of treatment, with a primary endpoint of total estimated glomerular filtration rate (eGFR) slope through Week 104.
VRTX closed Monday’s regular trading at $460.87 up $4.18 or 0.92%. In the after-hours trading, the stock further gained $21.59 or 4.68%.
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