Novartis NVS announced new encouraging data from a mid-stage study on pipeline candidate, LNP023, an investigational oral treatment for paroxysmal nocturnal hemoglobinuria (PNH) at the virtually held 2020 European Society for Blood and Marrow Transplantation (EBMT) Annual Meeting.

LNP023 is a first-in-class, orally-administered, potent and highly selective factor B inhibitor of the alternative complement pathway.

Data showed that LNP023 demonstrated improvements in hematological response and biomarkers of disease activity in patients with active hemolysis despite treatment with Alexion’s ALXN Soliris (eculizumab). Additionally, patients taking LNP023 as an add-on to Soliris saw significant reductions in their lactate dehydrogenase levels, a biomarker of intravascular hemolysis, and marked improvement in their hemoglobin (Hb) levels.

On a comparative basis, LNP023 increased Hb by a clinically relevant 2.87 g/dL (p<0.001), and all but two patients (80%) achieved Hb levels >12 g/dL in the absence of red blood cell (RBC) transfusions. All patients required RBC transfusions prior to starting the LNP023 treatment.

Importantly, data showed that after at least six months of stable LNP023 add-on therapy, and at the investigators’ discretion, seven patients (70%) have discontinued Soliris and remained on LNP023 as monotherapy.  The candidate also demonstrated a favorable safety and tolerability profile with no serious treatment-related infections or thromboembolic events.

We note that the FDA and the EMA have granted orphan drug designation to LNP023 for the treatment of PNH.

Separately, Novartis also announced results from a post-hoc analysis of pooled data from the phase III ORION-10 and -11 studies evaluating the individual responses of patients on low-density lipoprotein cholesterol (LDL-C) reduction with inclisiran, an experimental treatment for hyperlipidemia in adults.

Per the new data, inclisiran showed a highly consistent effect, with a safety and tolerability profile similar to placebo, on a twice-yearly dosing schedule after an initial dose and one 3 months later, across individual patients with atherosclerotic cardiovascular disease (ASCVD) or risk equivalents over 17 months of treatment.

Per the data, 99% of patients treated with inclisiran showed placebo-adjusted reduction in low-density lipoprotein cholesterol (LDL-C) of ≥30% with a mean reduction of 54.1% from baseline. Moreover, 88% of inclisiran-treated patients achieved an LDL-C placebo-adjusted reduction of at least 50% at any time point during the study. The candidate is currently under review with the FDA and the EMA for the treatment of primary hyperlipidemia (including heterozygous familial hypercholesterolemia) in adults who have elevated LDL-C while being on a maximally tolerated dose of statin therapy.

Approval of new drugs holds the key for Novartis as it looks to strengthen its core pharma business after a series of restructuring measures.

The stock has lost 8.8% in the year so far against the industry’s growth of 1.4%.

While the coronavirus pandemic has impacted sales in the year so far, Novartis’ Cosentyx maintains momentum for the company on recent label expansions, despite stiff competition from AbbVie’s ABBV Humira, and Taltz and Johnson & Johnson’s JNJ Tremfya. However, the FDA recently approved a label update for ophthalmology drug, Beovu, to include additional safety information regarding retinal vasculitis and retinal vascular occlusion. The growth trajectory that Beovu takes up following this label update is yet to be seen.

Novartis currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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