Baxalta Inc Takes a Step Ahead in Hemophilia A Treatment


Baxalta Inc Takes a Step Ahead in Hemophilia A Treatment

The company is working to cover the hemophilia therapeutic market in the US and the EU

Baxalta Incorporated (NYSE:BXLT) has announced it has initiated the first Phase 1 in-human clinical trial of BAX 826, a recombinant Factor VIII (rFVIII) for hemophilia A treatment. The company will use polysialic acid (PSA) technology for the enhancement of the half-life of its investigational drug.

BAX 826 belongs to the next-generation rFVIII treatment category, based on the full-length ADVATE. The molecule is modified with the help of PSA technology licensed from Xenetic Biosciences. Baxalta, in collaboration with Xenetic, is working on the development of novel forms of polysialylated blood coagulation factors. These factors play a vital role in increasing the circulating half-life, along with improving pharmacokinetic profile of therapeutic proteins, peptides, and small molecules.

The scientific study is looking to enroll approximately 30 patients for the evaluation of the efficacy, pharmacokinetics, and safety profile of BAX 826. The drug maker expects completing enrollment by the end of 2016.

Hemophilia A is a chronic disease and its treatment is complex and lengthy. The treatment regimen is based on regular infusions to reduce the risk of bleeding. Hemophilic patients have to work closely with physician, to manage their medication/infusion needs.

Worldwide, 400,000 people contract hemophilia every year, out of which 75% are unable to get proper treatment. At present, the therapeutic market for hemophilia is divided into plasma-derived therapies, with 30% sales pertaining to hemophilia A, 45% to hemophilia B, and 25% for inhibitors. The therapeutic market of hemophilia A is estimated to become worth $13.43 billion in 2019.

The number of drugs for the treatment of hemophilia is rising, but the patient ratio is low. As a result, drug makers and specialty pharmacies have started to hire patients and their families, to convince other patients to switch to the treatment they desire.

The BAX 826 trial is part of the drug maker’s plans to make progress in the field of hemophilia, with the aid of direct factor replacement treatments, including ADVATE and ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated]. These are approved in the US and are under regulatory review in Japan, Canada, and Switzerland. The company is also working on the treatment of hemophilia A, using gene therapy modulation.

Baxalta head of Research & Development and chief scientific officer John Orloff, M.D., said: “This is an important step in evolving extended half-life treatments as we aim to improve bleed protection for hemophilia A patients while potentially offering once-weekly dosing.”

He added: “Baxalta is dedicated to advancing innovative research on the principle of direct factor replacement, a proven treatment model, to support as many patients as possible.”


ADVATE established its efficacy and safety profile in multiple clinical studies. It is a full-length (derived from the complete FVIII gene) recombinant FVIII product. ADVATE is processed without any blood-based additives, so there are minimal-to-no chances of the transmission of the pathogens. The clinical studies proved no transmission of HIV, HBV, or HCV, with rFVIII treatments. ADVATE is already approved in 67 countries worldwide, including the United States, Canada, and the European Union

Baxalta is working to become the market leader in blood related disorders. The company has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA), seeking approval for Adynovi as a treatment for hemophilia A. This approval will enhance the sales. It has also already submitted the drug for approval in Canada, Japan, and Switzerland.

High profile companies battling for this segment include Biogen, Baxalta, Bayer, and CSL. These companies are offering long-lasting therapies that require very few infusions per week. Roche is also working to develop the first-in-class antibody. Alnylam, a Massachusetts-based company, is exploring options for treatment with gene therapies, to cure the disease altogether.

Barclays upgraded the target price on Baxalta from $38.00, to $40.00, and rated it Equal Weight. Zacks Investment Research moved Baxalta from a Buy, to a Hold rating.


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