Novartis gets positive results back from oral therapy for PNH

By Ben Hargreaves

- Last updated on GMT

© ExperienceInteriors / Getty Images
© ExperienceInteriors / Getty Images

Related tags Novartis

The company announced Phase III results showing that hemoglobin levels were increased when patients were treated with investigational oral monotherapy, iptacopan.

Novartis is looking to advance iptacopan to treat paroxysmal nocturnal hemoglobinuria (PNH), and a positive topline readout​ from a Phase III will likely help the oral therapy’s cause. The results mark the second positive Phase III trial for the treatment, with the company looking to begin global regulatory submissions in 2023.

During the most recent trials, iptacopan was shown to achieve clinically meaningful hemoglobin-level increases of 2 g/dL or more from baseline, without the need for blood transfusions at 24 weeks.

PNH is a rare blood disorder that is present when part of the immune system attacks and damages red blood cells and platelets. If left untreated, the condition can cause hemolytic anemia, chronic kidney disease or thrombosis.

In October 2022​, Novartis released results where iptacopan was tested against anti-C5 therapies, eculizumab or ravulizumab, which are currently used to treat PNH.

The company was able to reveal that topline results showed a statistically significant increase in the proportion of patients treated with its drug candidate achieving hemoglobin levels of 2 g/dL, compared with anti-C5 treatments.

Novartis stated on the existing anti-C5s treatments and those that receive them: “A large proportion of people with PNH remain anemic, fatigued and dependent on blood transfusions.”

Eculizumab is known by the brand name Soliris, and ravulizumab by Ultomiris, both treatments were developed by Alexion Pharmaceuticals, but are now sold by AstraZeneca, after it acquired the company in 2021​.

Both Soliris and Ultomiris are monoclonal antibodies (mAbs) and are delivered by injection. If approved, iptacopan would become the first oral monotherapy for PNH.

The treatment is delivered twice daily orally, while Soliris is delivered every week for five weeks, followed by every two weeks. Due to the mAb treatments being delivered by intravenous infusion, a healthcare professional is required, and an oral treatment could represent an advantage due to the simpler means of delivery.

Wider than PNH, Novartis also has expectations for the treatment to expand into other indications. The small molecule is currently being explored for the complement-mediated kidney diseases (CMKDs) C3 glomerulopathy, IgA nephropathy, and atypical hemolytic uremic syndrome.

Novartis previously stated​ that it expects the drug candidate to reach ‘multi-billion’ potential in sales, if it can prove its efficacy across multiple indications.

The positive read out for the trial is important news for the company, as it looks to reorganize its business​ to ensure greater growth and to focus on its R&D pipeline.

Related topics Clinical Development Phase III-IV

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