Data Presented at the Hemostasis and
Thrombosis Research Society 2017 Scientific
Symposium
Aptevo Therapeutics Inc. (Nasdaq:APVO), a biotechnology company
focused on developing novel oncology and hematology therapeutics,
today announced the presentation of new clinical data evaluating
the safety and efficacy of IXINITY® [Coagulation Factor IX
(Recombinant)] in previously treated patients under 12 years of age
with Hemophilia B. The data suggest that IXINITY appears to
be safe and well tolerated in this subject population, and is
comparable to the results from the overall patient population
studied in the pivotal clinical trial of IXINITY.
“We’re encouraged to see data in a pediatric
setting demonstrating the safety and efficacy of IXINITY in this
important population,” said Scott Stromatt, Senior Vice President
and Chief Medical Officer. “The key efficacy outcomes from
this study are comparable to what has previously been demonstrated
in our pivotal study of IXINITY, which formed the basis for our
licensure in the United States in patients 12 years of age or
older. In this pooled analysis, IXINITY was well tolerated
and effective in preventing and controlling bleeding episodes in
previously treated patients under the age of 12 with Hemophilia
B. According to the Centers for Disease Control and
Prevention Community Counts public health monitoring program,
approximately 22% of people treated for Hemophilia B are under the
age of 10 years, representing a sizeable new addressable market for
IXINITY, should we be successful in expanding the labeled
indication for IXINITY to include this patient population.”
Launched in the United States in 2015 and
wholly-owned by Aptevo, 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.
Study Design
The data presented at the Hemostasis and
Thrombosis Research Society 2017 Scientific Symposium represent a
pooled analysis of 2 prospective, multi-center, non-randomized,
open-label studies of 12 children with Hemophilia B under the age
of 12 years. Patients were treated with either a prophylactic
or on demand regimen based on investigator discretion. Bleed
control efficacy was evaluated using: annualized bleeding rate;
subject’s rating of efficacy for the degree of bleed control; and
the number of infusions required to treat a bleeding episode.
Subjects were monitored for adverse events and regularly assessed
for the development of factor IX inhibitors.
Results
Among the 11 subjects receiving a prophylactic
regimen of IXINITY, the median number of bleeding episodes was 1.0
(range 0-11) and the median annualized bleeding rate was 0.3 (range
0-4.0). Of the 61 bleeding episodes experienced by all
subjects in this study, 6 (10%) resolved with no infusions of
IXINITY, 44 (72%) resolved after one infusion of IXINITY, 5 (8%)
required two infusions, and 6 (10%) required three, four, or five
infusions of IXINITY. Of the 34 bleeding episodes rated,
subjects rated bleed control as “excellent” for 22 (65%) of the
bleeding episodes; “good” for 11 (32%) of the bleeding episodes;
“fair” for 1 (3%) of the bleeding episodes; and “poor” for no
episodes.
Adverse events reported in this study which were
believed to be related to IXINITY were hyperhidrosis (excessive
sweating) and fever in one patient, and hyperhidrosis in another
patient. None of the subjects developed factor IX inhibitors during
the study.
“As patients are known to respond to different
factor IX options differently, IXINITY offers people with
Hemophilia B another option to dose strategically to prevent and
control bleeding episodes. We see value in having a variety
of options available for patients and look forward to building on
the data set in a pediatric setting to support a label expansion
for IXINITY in patients under 12 years of age,” said Dr.
Stromatt.
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.
About IXINITY
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 biotechnology
company focused on novel oncology and hematology therapeutics to
meaningfully improve patients’ lives. Our core technology is the
ADAPTIR™ (modular protein technology) platform. Aptevo has four
commercial products in the areas of hematology and infectious
diseases, as well as various investigational stage product
candidates in immuno-oncology.
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 IXINITY, our technology and related pipeline, the
potential for regulatory approvals, collaboration and partnership
opportunities, commercial portfolio, Aptevo’s future growth rates,
Aptevo’s ability to timely manufacture its products, 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 our actual results to differ materially from those
indicated by such forward-looking statements, including possible
negative effects on our business operations, assets or financial
results as a result of the separation; a deterioration in our
business or prospects; the ability to obtain regulatory approvals;
the ability of our contractors and suppliers to supply product and
materials; our ability and the ability of our contractors and
suppliers to maintain compliance with cGCP and other regulatory
obligations; the results of regulatory inspections; adverse
developments in our customer-base or markets and our ability to
retain patients; 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 our filings with
the Securities and Exchange Commission, including Aptevo’s most
recent Annual Report on Form 10-K, as filed on March 31, 2017. The
foregoing sets forth many, but not all, of the factors that could
cause actual results to differ from our expectations in any
forward-looking statement.
Source:
Aptevo Therapeutics
Stacey Jurchison
Senior Director, Investor Relations and Corporate Communications
206-859-6628
JurchisonS@apvo.com
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