Clinical-stage biotechnology company Abivax, has reported promising results from its phase 2a maintenance trial into rheumatoid arthritis (RA) after one year of continued daily treatment with 50mg ABX464.

After the 12-week, randomised, placebo-controlled induction study in 60 RA patients, 67% of the patients enrolled in the open-label extension maintenance study to receive the treatment orally once a day for an additional 52 weeks. 58% of the patients suffering from moderate to severe active RA completed 52 weeks of chronic treatment with ABX464.

In June 2021, Abivax communicated the results of the induction phase of its clinical study, administered in combination with methotrexate (MTX), for the treatment of active moderate to severe RA. The treatment demonstrated a good safety and tolerability profile in the overall patient population during the 12-week induction phase.

Professor Paul Emery, Arthritis Research Professor of Rheumatology, said: “The high levels of maintained response rates within this phase 2a maintenance trial with ABX464 in rheumatoid arthritis patients, especially when it comes to ACR50 and ACR70 responses, look very promising.

“The molecule also demonstrated a good safety profile, and no serious infections were observed. Along with its very different mode of action and clinical profile, ABX464 has the potential to play an important role in the future management of rheumatoid arthritis patients.”

Professor William Robinson, Chief of Division of Immunology and Rheumatology, Stanford University, US, added: “Patients suffering from chronic inflammatory diseases, such as RA, often struggle to find a suitable treatment that remains efficacious over time. The maintenance data is very encouraging and demonstrates a potential long-term efficacy and tolerability of ABX464 for the treatment of RA, even in patients who previously did not respond or stopped responding to available therapies.”

Sanofi and Swedish Orphan Biovitrum AB have announced positive results from the pivotal XTEND-1 phase III study into efanesoctocog alfa (BIVV001). The research involved previously treated patients with severe haemophilia A.

The primary endpoint was met, and the results showed a clinically meaningful prevention of bleeds in people with severe haemophilia A, who were receiving weekly prophylaxis with efanesoctocog alfa over a period of 52 weeks.

The study also met the key secondary endpoint, demonstrating that once-weekly efanesoctocog alfa was more effective than prior prophylactic replacement therapy. The treatment was well tolerated.

Haemophilia A is a rare genetic disorder in clotting factor VIII, which causes increased bleeding. It occurs in about one in 5,000 male births annually, and more rarely in females. Patients suffering from haemophilia can experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages.

BIVV001 is a novel and investigational recombinant factor VIII therapy, designed to extend protection from bleeds with once-weekly prophylactic dosing for people with haemophilia A. It is still under clinical investigation, and has not yet been evaluated by regulatory authorities.

Dietmar Berger, Global Head of Development and CMO, Sanofi, commented on the results: “While advances have been made in the treatment of haemophilia, unmet medical needs still exist. These positive top-line data, showing a very low annualised bleeding rate, enhance efanesoctocog alfa’s potential to transform hemophilia A therapy.

“We believe efanesoctocog alfa provides higher protection for longer duration with reduced treatment burden of once-weekly dosing, and we look forward to working with regulators to bring this therapy to patients as soon as possible.”

Anders Ullman, Head of R&D and CMO, Sobi, added: “We believe once-weekly efanesoctocog alfa has the potential to represent a new class of factor VIII therapy designed to provide high sustained factor VIII levels near normal for the majority of the week. We look forward to sharing these phase 3 results, including data on physical health, pain and joint health, at future medical meetings.”