Roche's Hemlibra follow-up set for phase 3 in haemophilia A

 Roche's Hemlibra treatment for haemophilia A is one of its top-selling drugs, making almost $5 billion last year, and the company has now lined up what looks like an impressive successor.

The pharma group has presented phase 1/2 results with new candidate NXT007 at the International Society on Thrombosis and Haemostasis (ISTH) congress in Washington DC that suggest it is able to normalise blood clotting in people with haemophilia A.

Hemlibra (emicizumab) is an antibody that mimics the action of factor VIII, while NXT007 is a bispecific antibody – derived from emicizumab – that brings together factor IXa and factor X and is designed to offer greater potency and efficacy while requiring less frequent dosing.

In the NXTAGE trial, 30 patients with haemophilia A and no inhibitors were administered NXT007 as a subcutaneous injection every two to four weeks during an induction period, followed by injections every two to six weeks in a maintenance phase. In contrast, Hemlibra is initially dosed once a week by injection and once every two or four weeks thereafter.

Data reported at ISTH showed that patients receiving the two highest doses of NXT007 (0.7mg/kg and 1.08mg/kg) had no reported bleeds, as well as no clotting side effects, over 16 weeks of follow-up.

There were two treatment discontinuations, both in the low-dose group (0.072mg/kg), one of which was a result of the patient developing antibodies (inhibitors) against NXT007. One other patient also developed inhibitors, but remained on treatment. In both cases, the inhibitors did not show any cross-reactivity with Hemlibra.

NXT007 is an important project for Roche, given Novo Nordisk recently reported phase 3 data with a similarly acting bispecific, codenamed Mim8, last year that suggested it could match Hemlibra with less frequent dosing. The company has previously said it is anticipating the first regulatory approval for Mim8 later this year.

Roche's chief medical officer, Levi Garraway, said the results were "promising" and suggest that NXT007 could build on the success of Hemlibra, which established "a new standard of care" for haemophilia A.

The next steps for the programme include additional phase 2 data, due later this year, and the start of a phase 3 programme based on three studies that will get underway in 2026. The latter will include a head-to-head comparison with Hemlibra.

https://pharmaphorum.com/news/roches-hemlibra-follow-set-phase-3-haemophilia