Two years after canceling three hemophilia trials, Novo Nordisk plans road to FDA with new PhIII data

Two years after canceling three hemophilia trials, Novo Nordisk plans road to FDA with new PhIII data

Novo Nordisk continues to advance its once-daily monoclonal antibody therapy for hemophilia A or B with the goal of stopping excessive bleeding events before they occur, promoting new Phase 3 data on Sunday.

The Danish pharmaceutical company has put word that a pivotal trial of conecizumab achieved its primary goal, which is to reduce spontaneous bleeding and shock by 86% in a protective setting compared to the control arm. Novo Nordisk is developing the filter so that patients with hemophilia A or B can take it regardless of whether they develop immune responses to other medications.

“One of the most important complications in the treatment of hemophilia is the development of inhibitors, as they render standard alternative therapy ineffective and severely limit treatment options for hemophilia B,” lead researcher Victor Jimenez Yusti said in a statement.

Sunday’s news points to a rebound for conecizumab, just over two years after Novo experienced major setbacks in drug development. In March 2020, Novo Nordisk halted two Phase 3 studies and one Phase 2 trial after three patients experienced non-fatal blood clots. The Food and Drug Administration has also placed the program on clinical hold.

But Novo resumed development after finding a “new path forward” in August, the company said at the time, which included changes to dosing regimens and frequency of administration. With Phase Three now read, Novo plans to file its FDA bid before the end of this year.

The strategy was to develop a treatment that could stop spontaneous bleeding in patients whether or not they were already taking an alternative treatment, and to develop an immune response known as inhibitors. Concizumab is a once-daily antibody to a tissue factor inhibitor (TFPI) pathway given in an EpiPen-style syringe.

The researchers tested the drug on 133 boys and men aged 12 or older (Novo says males account for about 88% of hemophilia cases). Patients were randomized 2 to 1 to either the prevention or non-prevention arm and measured for at least 32 or 24 weeks.

Novo compared the number of treated bleeding episodes, known as ABR, between these two groups for primary analysis. In the prevention arm, the average estimated ABR was 1.7, while in the non-preventive arm the average ABR was 11.8. In addition, the mean total ABR of conecizumab was zero, compared to 9.8 for no prophylactic treatment.

Safety proved much better this time around, with Novo saying that the drug did not produce any thromboembolic events after treatment was resumed.

Although Novo is going the antibody route, many big-name hemophilia players have tried their hand at gene therapies instead. These programs from Pfizer/Sangamo and BioMarin that pursue hemophilia A each have their own problems, however, with the former only being released from clinical comment in May and the latter receiving a CRL in 2020.

Despite this, Novo is ostensibly trying to compete with Roche’s flagship drug Hemlibra and Spark Therapeutics, which brought in about $870 million globally in the first quarter of this year. Hemlibra is only approved for hemophilia A patients taking factor VIII inhibitors, while Novo is designed for either hemophilia A or hemophilia B.

Not to be outdone, Roche released new data on Hemlibra early Monday morning as well, saying that the drug has shown effective control of bleeding in mild or moderate hemophilia A patients who do not have factor VIII inhibitors. Two-thirds of the 72 study patients experienced no bleeding events at a median follow-up of 55.6 weeks.

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