Our R & D projects are to deliver attractive new products in Biovitrum focus area: protein drugs for specialist indications for a global market.
The portfolio is currently composed of six Phase II projects and one Phase III project. Furthermore, we have several projects in the research phase.
We have projects in the following therapeutic areas: hemophilia / hematology, fat malabsorption and supportive cancer treatment.
Factor IX Fc (rFIXFc) for the treatment of hemophilia B
In January, the first patients were dosed in the registrational, open-label, multicenter study, to evaluate the safety, pharmacokinetics and efficacy of the long-acting, recombinant Factor IX Fc fusion protein (rFIXFc) in hemophilia B patients. The trial, called the B-LONG study, will include approximately 75 previously-treated patients with severe hemophilia B. The FIXFc program is partnered with Biogen Idec. The results from the first clinical phase I/II study will be presented at “Hemophilia 2010 World Congress”, to be held in Buenos Aires, Argentina, mid-July 2010.
Kiobrina® for the treatment of fat malabsorption in premature infants
On April 21, Swedish Orphan Biovitrum announced the results from the second Kiobrina® clinical phase II study. The study demonstrated an improvement in preterm infant growth velocity, when Kiobrina® was administered in pasteurized breast milk. As a consequence of this outcome, and the previously announced positive results from a phase II study in preterm infant formula, Swedish Orphan Biovitrum has taken the decision to move Kiobrina® into phase III development. Interim Report January 1st to March 31st, 2010.
The combined results of the two clinical studies showed a statistically significant increase in growth velocity (p<0.001), which is a medical relevant parameter. The safety profile was comparable to that of placebo, and no drug-related serious adverse events were reported. The results from these studies will be published during 2010, starting with a presentation of the results from the first clinical study with infant formula, at “The Power of Programming 2010. International conference on developmental origins of health and disease” in Munich, Germany, May 6-8, 2010.
Multiferon® for the second-line treatment of hepatitis C
Multiferon is currently approved in several EU countries, with a broad second-line indication, as well as an adjunctive treatment of high-risk patients with malignant melanoma. A phase III study, aiming to support further territorial expansion in EU, and to support price and reimbursement, is currently in the final planning stage. The application to start the study (CTA) in second line treatment of hepatitis C has been submitted to regulatory authorities in March.
Factor VIIIFc (FVIIIFc) for the treatment of hemophilia A
A phase I/IIa study of the long-acting fully-recombinant Factor VIII Fc fusion (rFVIIIFc) protein is ongoing. This open-label study will assess the safety, tolerability and pharmacokinetics of rFVIIIFc in severe, previously-treated, hemophilia A patients. The rFVIIIFc program is partnered with Biogen Idec.
Exinalda® for the treatment of fat malabsorption due to pancreatic insufficiency
An open label exploratory phase II study on Exinalda (rhBSSL) in patients with cystic fibrosis and pancreatic insufficiency has been completed. The aim was to study the effect of Exinalda on fat absorption as well as safety in this patient population. The results showed that Exinalda is safe and tolerable at a dose level of 170 mg three times a day. In terms of efficacy (coefficient of fat absorption) the primary end-point was not met. Swedish Orphan Biovitrum is currently evaluating next steps in the development of Exinalda.
Sym001 for the treatment of immune thrombocytopenic purpura (ITP) and Rhesus immunization prophylaxis
A phase I study has been successfully completed. A clinical study that shows that Sym001 can eliminate Rhesus (RhD) positive red blood cells from the circulation of RhD negative healthy volunteers has also been concluded. In addition, a clinical phase II study with the aim to study the safety and efficacy of Sym001 in ITP patients is ongoing at 23 clinics in Europe. Good progress has been achieved since the previous quarter. Three out of four planned dose cohorts have now been treated, and the independent safety committee has recommended a continuation to the next dose group. This study is expected to be completed in the second half of 2010.
Kepivance® for the treatment of oral mucositis associated with blood cancer treatment in children
A clinical study in children with acute leukemia who are undergoing stem cell transplants is currently ongoing. The purpose of the study is primarily to study safety and pharmacokinetics. The study, conducted in the US, will also document the therapeutic effect on inflammation in the mouth and throat. Approximately 27 children aged 1 to 16 years are expected to be included. Results of this study will be available during the second half of 2010.
