Plans to Introduce 3000 IU IXINITY Assay for Enhanced Patient Convenience
Will Commence Pediatric Study Seeking U.S. Label Expansion
Explores Ex-US Licensing and Partnership Collaborations to Expand International IXINITY Footprint
SEATTLE, Oct. 24, 2018 (GLOBE NEWSWIRE) -- Aptevo Therapeutics Inc. (Nasdaq: APVO), a biotechnology company focused on developing novel oncology and hematology therapeutics, today provided an update on its growth strategy for IXINITY® [Coagulation Factor IX (Recombinant)] in the United States and ex-U.S. markets.
“We reached an important milestone in our IXINITY program this year as IXINITY became cash flow positive for our organization,” said Mike Adelman, Senior Vice President, Commercial Operations for Aptevo. “With our manufacturing process stabilized and our commercial efforts in the U.S. now solidly on track, Aptevo is intensifying its focus on additional market expansion opportunities for IXINITY both within and outside the U.S market.”
“Our team has been very successful firming up and expanding our IXINITY business in the U.S, and we look forward to continued success onboarding new patients onto IXINITY therapy. However, we recognize there remains considerable untapped opportunity for IXINITY in the marketplace, which we are now positioned to pursue,” continued Mr. Adelman.
Feedback from the Hemophilia B community suggests that a larger assay size for IXINITY would be very desirable from a patient dosing and convenience perspective and could promote broader adoption of IXINITY. As a result, Aptevo has initiated process development and manufacturing enabling activities to support the launch of a new 3000 IU vial assay for IXINITY, which it anticipates will be available in mid-2019.
Aptevo has also initiated plans to commence a Phase 4 study of IXINITY in approximately 20 patients under 12 years of age for potential label expansion of IXINITY in a pediatric setting. 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.
According to the World Federation of Hemophilia ‘Report on the Annual Global Survey 2016’ 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, if IXINITY receives a pediatric label expansion.
“Initiation of a pediatric study offers a valuable opportunity to make a limited strategic investment in IXINITY which could expand the U.S. market opportunity considerably,” said Mr. Adelman. “In addition to these efforts, Aptevo is also closely evaluating opportunities to license or partner distribution and marketing rights for IXINITY in ex-U.S. markets. Given Aptevo’s pre-existing relationships with various international distributors, formed through our hyperimmune business which was divested in late 2017, we believe there is an attractive opportunity to leverage these connections to expand our footprint internationally.”
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 37% of the global market is concentrated outside the United States.
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 novel oncology 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 or autoimmune diseases. Aptevo has two ADAPTIR antibody candidates currently in clinical development and 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; challenges in sales and marketing efforts; 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 13, 2018 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)
Senior Director, Investor Relations and Corporate Communications