As head-to-head with Alexion heats up, Novartis flashes data for next wave of trials for star kidney molecule
Novartis has rarely invested much in kidney drugs, but over the last couple of years, they’ve tried to build a pipeline out of a single drug.
On Sunday, that drug, a small molecule called iptacopan, delivered another success. Novartis announced that it cleared a Phase II trial for the rare kidney disease IgA nephropathy, significantly reducing a risk factor for kidney health. The data burnish the Swiss pharma’s confidence in a pivotal Phase III study already underway in the condition, along with the mid- or late-stage studies they’ve launched in a handful of uncommon renal afflictions.
Collectively, they add a secondary set of markets for the drug, even if it proves unsuccessful in unseating Alexion in PNH, where the drug recently won an FDA breakthrough designation.
“Complement-driven renal diseases, such as IgAN, are devastating and mostly affect young adults, imposing a high disease burden,” Novartis CMO John Tsai said in a statement. “These new data in IgAN add to the growing body of evidence around the potential of iptacopan to target a key driver in these rare renal diseases.”
In the 112-person study, IgA nephropathy patients were randomized to receive placebo or various doses of iptacopan. Patients who received the highest dose — the one they brought forward into Phase III — saw a 23% reduction in proteinuria, or the amount of protein, patients released in urine. A correlate for how well the kidneys are filtering, the FDA has recently accepted it as a surrogate endpoint for accelerated approval.
Novartis said the drug also showed a “trend toward stabilization” in glomerular filtration rate, a more established measure for kidney function, but declined to disclose the actual data in their press release. The two measures are co-primary endpoints on the Phase III studies.
IgA nephropathy has no approved treatments, but as the FDA has updated its requirements for approving new kidney drugs and pharma and VC cash has poured into the space, the hunt for one has grown hotly competitive. Swedish biotech Calliditas snared a $90 million IPO last year mostly on the promise of its Phase III steroid for the disease. In January, GV-backed Trucode Gene Repair revealed they decided to abruptly scrap their entire gene editing platform and sickle cell disease focus on the hope that they could turn an old Merck KGaA drug into the first treatment for the disease. (They rebranded as Vera and went for a modest IPO.)
Like the other diseases Novartis is firing iptacopan at, IgA nephropathy is driven by a dysfunctional immune system, in particular problems with a cascade called the complement system. The most advanced program is for PNH, the most well-known complement disorder, but one where Novartis faces stiff competition in established giant Alexion and a newcomer in Apellis, whose drug Empaveli won approval last month.
The company will have a clearer path to commercial dominance in kidney diseases, where few, if any, other options are available. On Monday, the company announced updated data from their Phase II trial for CG nephropathy, also known as C3G, showing improved kidney function from patients on the drug. A Phase III trial will start later this year.