Expands Options for Patients with Potential for
Reduced Reconstitution Time and Infusion Volume
SEATTLE, June 25, 2019 (GLOBE NEWSWIRE) -- Aptevo Therapeutics Inc. (APVO), a biotechnology company focused on developing novel oncology, autoimmune and hematology therapeutics, today announced that it has launched a new, larger 3000 IU (international unit) vial size for IXINITY® [Coagulation Factor IX (Recombinant)] into the U.S. market. The new 3000 IU assay offers important advantages for both patients and channel partners, which Aptevo anticipates will help drive future conversions to IXINITY therapy.
“Aptevo has a strong reputation in the Hemophilia B community for listening to and responding to the needs of the community and we’re pleased to once again deliver on our commitment to patients,” said Mike Adelman, Senior Vice President, Commercial Operations for Aptevo. “We’ve heard feedback from the community that more diversity in our assay offerings – particularly higher IU options is very desirable for a number of reasons. First, depending on the patient’s dose, larger assay sizes require less time to reconstitute product for infusion and also decreases overall infusion volume, which is a particularly important benefit for patients, especially for those with severe hemophilia who typically require higher doses. Moreover, larger assays also require fewer vials for dosing, equating to improved convenience for storage and travel. Higher IU assays also provide improved value for our channel partners due to the requirement for less storage space and the potential for reduced shipping expenses.”
“We are excited to continue to deliver on our promise to patients with this latest initiative,” said Jeff Lamothe, Chief Financial Officer for Aptevo. “Last year, Aptevo released a smaller package size for IXINITY in response to feedback received from the community and our channel partners. This year, we have introduced the larger 3000 IU IXINITY assay in response to overwhelming patient demand, both from current IXINITY patients and potential future IXINITY patients. We’re pleased with the market’s acceptance of IXINITY and our growth trajectory since launch and believe that the enhanced experience and convenience afforded by the larger presentation will help to drive ongoing conversions to IXINITY therapy in the months and years ahead.”
With its commercial efforts in the U.S. solidly on track, Aptevo is intensifying its focus on additional market expansion opportunities for IXINITY both within and outside the U.S market. Later this year Aptevo will begin a confirmatory Phase 4 study of IXINITY for potential label expansion in a pediatric setting. The study is expected to enroll approximately 20 Hemophilia B patients under the age of 12 years and will be conducted at several clinical study centers worldwide. Previously reported data showed that IXINITY appeared to be safe and well tolerated in this subject population and were comparable to the results from the overall patient population studied in the pivotal clinical trial of IXINITY.
Mr. Lamothe continued, “Approximately 33% of people treated for Hemophilia B in the U.S. are under the age of 13 years1, representing a sizeable new addressable market, should IXINITY receive a pediatric label expansion. We also continue to look for opportunities to make IXINITY available to patients around the world and are seeking opportunities to align with international distributors to expand our IXINITY footprint more broadly. I’m very excited about the momentum we are building in our IXINITY franchise and look forward to continued future growth of IXINITY in the marketplace.”
IXINITY is currently marketed exclusively in the United States by Aptevo. The global Hemophilia B market in 2016 is estimated to be approximately $1.02 billion and projected to grow to $1.3 billion by 20262. Approximately 59% of the estimated Factor IX global market is concentrated outside the United States as measured by volume (international units).3
IXINITY is an intravenous recombinant factor IX therapeutic for use in people 12 years of age or older with Hemophilia B – a hereditary bleeding disorder characterized by a deficiency of clotting factor IX in the blood, which is necessary to control bleeding. IXINITY is currently available in four assay sizes -500 IU, 1000 IU, 1500 IU and 3000 IU vials.
About Hemophilia B
Hemophilia B is a congenital bleeding disorder caused by a deficiency of coagulation factor IX. It affects approximately 1:25,000 male births, with approximately 4,000 persons affected in the U.S. The clinical spectrum may include spontaneous or trauma-induced bleeding into joints, muscles, and soft tissues, resulting in joint damage, reduction in mobility, and severe arthritis, all of which negatively impact health-related quality of life. The primary aim of care is to prevent and treat bleeding by replacing the deficient clotting factor.
IXINITY is indicated for the control and prevention of bleeding episodes and for perioperative management for adults and children ≥12 years of age with Hemophilia B. IXINITY is not indicated for induction of immune tolerance in patients with Hemophilia B. IXINITY contains recombinant coagulation factor IX (trenonacog alfa). Trenonacog alfa is a purified single chain glycoprotein derived from Chinese hamster ovary (CHO) cells and has an amino acid sequence that is comparable to the Thr148 allelic form of plasma-derived factor IX. No human or animal proteins are added during any stage of manufacturing or formulation of IXINITY. The recombinant factor IX is purified by a chromatography purification process. The process includes three validated steps for virus inactivation and removal. The process also includes a validated manufacturing step to reduce the presence of CHO proteins in the final drug product.
Indications and Important Risk Information
IXINITY [Coagulation Factor IX (Recombinant)] Lyophilized Powder for Solution for Intravenous Injection is a coagulation factor IX (recombinant) indicated in adults and children ≥ 12 years of age with Hemophilia B for control and prevention of bleeding episodes, and for perioperative management. IXINITY is not indicated for induction of immune tolerance in patients with Hemophilia B. IXINITY is contraindicated in patients who have known hypersensitivity to IXINITY or its excipients, including hamster protein.
Hypersensitivity reactions, including anaphylaxis, may occur following IXINITY administration. Discontinue use of IXINITY if hypersensitivity symptoms occur, and initiate appropriate treatment. Regularly evaluate patients for the development of factor IX inhibitors by appropriate clinical observations and laboratory tests. If expected factor IX activity plasma levels are not attained, or, if bleeding is not controlled as expected with a certain dose, perform an assay that measures factor IX inhibitor concentration. An association between the occurrence of a factor IX inhibitor and allergic reactions has been reported. Individuals with factor IX inhibitors may be at increased risk of severe hypersensitivity reactions or anaphylaxis if re-challenged.
Nephrotic syndrome may occur with IXINITY. Nephrotic syndrome has been reported following attempted immune tolerance induction in Hemophilia B patients with factor IX inhibitors and a history of allergic reactions. Thromboembolism may occur when using IXINITY (e.g., pulmonary embolism, venous thrombosis, and arterial thrombosis). Patients may develop hypersensitivity to hamster (CHO) protein as IXINITY contains trace amounts. The most common adverse drug reaction observed in >2% of patients in clinical trials was headache.
Please see full Prescribing Information at www.IXINITY.com.
About Aptevo Therapeutics Inc.
Aptevo Therapeutics Inc. is a clinical-stage biotechnology company focused on developing novel oncology, autoimmune and hematology therapeutics to meaningfully improve patients’ lives. Aptevo has a commercial product, IXINITY® coagulation factor IX (recombinant), approved and marketed in the United States for the treatment of Hemophilia B, and a versatile core technology – the ADAPTIR™ modular protein technology platform capable of generating highly-differentiated bispecific antibodies with unique mechanisms of action to treat cancer and autoimmune diseases. Aptevo has a broad pipeline of novel investigational-stage bispecific antibody candidates focused in immuno-oncology and autoimmune disease and inflammation. For more information, please visit www.aptevotherapeutics.com
Safe Harbor Statement
This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact, including, without limitation, statements regarding potential milestone payments, Aptevo’s outlook, financial performance or financial condition, Aptevo’s technology and related pipeline, collaboration and partnership opportunities, commercial portfolio, milestones, and any other statements containing the words “believes,” “expects,” “anticipates,” “intends,” “plans,” “forecasts,” “estimates,” “will” and similar expressions are forward-looking statements. These forward-looking statements are based on Aptevo’s current intentions, beliefs and expectations regarding future events. Aptevo cannot guarantee that any forward-looking statement will be accurate. Investors should realize that if underlying assumptions prove inaccurate or unknown risks or uncertainties materialize, actual results could differ materially from Aptevo’s expectations. Investors are, therefore, cautioned not to place undue reliance on any forward-looking statement. Any forward-looking statement speaks only as of the date of this press release, and, except as required by law, Aptevo does not undertake to update any forward-looking statement to reflect new information, events or circumstances.
There are a number of important factors that could cause Aptevo’s actual results to differ materially from those indicated by such forward-looking statements, including a deterioration in Aptevo’s business or prospects; adverse developments in research and development; adverse developments in the U.S. or global capital markets, credit markets or economies generally; and changes in regulatory, social and political conditions. Additional risks and factors that may affect results are set forth in Aptevo’s filings with the Securities and Exchange Commission, including its most recent Annual Report on Form 10-K, as filed on March 18, 2019 and its subsequent reports on Form 10-Q and current reports on Form 8-K. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from Aptevo’s expectations in any forward-looking statement.
1Source: World Federation of Hemophilia Report on the Annual Global Survey, 2016 (Publication Date: October 2017)
2Source: Global Data PharmaPoint: Hemophilia A and B Recombinant Factor Replacement Therapy – Global Drug Forecast and Market Analysis to 2026 (Publication Date: June 2017; Base year 2016)
3Sources: 2017 Annual Global Survey. World Federation of Hemophilia; Market Research Bureau (MRB) Global Forecast of FIX and Hemophilia B Market by Region 2016 to 2022 (published September 2017)
Senior Director, Investor Relations and Corporate Communications