Swiss drugmaker Novartis has announced positive topline results for its twice-daily oral monotherapy Fabhalta (iptacopan) from Phase 3B APPULSE-PNH clinical study.
Fabhalta is a Factor B inhibitor of the alternative complement pathway, indicated for adults with paroxysmal nocturnal haemoglobinuria (PNH) who switched from anti-C5 therapies.
APPULSE-PNH is a multicentre, single-arm, open-label, Phase 3B study that evaluated the safety and efficacy of twice-daily oral Fabhalta in treating 52 adults with PNH.
In the study, treatment with Fabhalta improved the average haemoglobin (Hb) level compared to the baseline, after 24 weeks.
Fabhalta showed a safety profile that was consistent with previously reported data.
APPULSE-PNH trial lead investigator Antonio Risitano said: “Treatment goals for patients with PNH have greatly evolved, and we can now aim to resolve signs and symptoms of disease in most patients. It is promising to see this evolution, and we will continue to make progress to best support these patients.”
Fabhalta secured the US Food and Drug Administration (FDA) approval in December last year, and the European Medicines Agency (EMA) in May this year.
The drug received FDA accelerated approval for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression.
Novartis cardiovascular, renal and metabolism development unit global head David Soergel said: “Across multiple clinical trials, Fabhalta has consistently shown clinically meaningful benefits for patients with PNH, and the APPULSE-PNH trial is a compelling addition to this body of evidence.
“These data reinforce our confidence in Fabhalta, the first and only oral monotherapy currently available for the treatment of adults with PNH, to provide meaningful haemoglobin improvement, regardless of previous treatment experience.”
In a separate development, Novartis announced positive, longer-term results for its Scemblix (asciminib) from the Phase 3 ASC4FIRST study.
In the Phase 3 study, Scemblix was evaluated in adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukaemia in the chronic phase (Ph+ CML-CP).
The drug was compared with investigator-selected standard-of-care (SoC) tyrosine kinase inhibitors (TKIs) (imatinib, nilotinib, dasatinib and bosutinib) and to imatinib alone.
The trial met both primary endpoints with Scemblix showing superior major molecular response (MMR) rates compared to investigator-selected SoC TKIs and imatinib alone.
In addition, the treatment also met the secondary, non-powered endpoint, said Novartis.
Novartis executive vice president, oncology development global head Jeff Legos said: “Novartis’ decades-long work in CML and deep relationships within the community have informed our Scemblix clinical trial program of over 10 years, the centrepiece of our continuing drive to address ongoing unmet medical needs for people with CML.
“These latest findings reinforce the differentiated efficacy, safety and tolerability profile of Scemblix in newly diagnosed and previously treated adult CML patients.”
