Both trials, EXCEED-ET and ECLIPSE-PV,
completed enrollment at 142% and 110% of target, respectively,
demonstrating high levels of clinical site interest
PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia
Corporation (TWSE: 6446), a global biopharmaceutical innovator
based in Taiwan leveraging deep expertise and proven scientific
principles to deliver new biologics in hematology, oncology and
immunology, today announced completion of enrollment for two
clinical trials evaluating its ropeginterferon alfa-2b-njft
(BESREMi®).
The Phase 2b EXCEED-ET trial (NCT05482971), which is evaluating
the effectiveness and safety of ropeginterferon alfa-2b-njft in
adult patients with essential thrombocythemia (ET), has exceeded
the enrollment goal of 64 patients to include 91 patients.
EXCEED-ET is evaluating people diagnosed with ET who are either
treatment naïve or have received previous ET treatment with
hydroxyurea or anagrelide but require a treatment change due to
intolerance or because the previous treatment is no longer
effective. This trial is being conducted in the United States and
Canada and will use the accelerated dosing schedule (250, 350, 500
mcg). This accelerated dosing schedule has been previously assessed
in Asian clinical trials.
The Phase 3b ECLIPSE-PV trial (NCT05481151), assessing the
effectiveness and safety of two dosing regimens of ropeginterferon
alfa-2b-njft in adult patients with polycythemia vera (PV), has
also exceeded the enrollment goal of 100 patients to include 111
patients. ECLIPSE-PV is evaluating two ropeginterferon alfa-2b-njft
doses, including the accelerated dosing schedule (as described
above) in comparison to the current recommended dosing regimen. The
ECLIPSE-PV study is being performed in the United States and
Canada.
"Myeloproliferative neoplasms, including ET and PV, are chronic
blood diseases that can impact patients' quality of life and lead
to life-threatening complications, including the development of
specific types of blood cancers, and PharmaEssentia is committed to
developing new therapeutic solutions for these patients,” said
Robert B. Geller, M.D., Head of Medical at PharmaEssentia USA. “We
are thrilled with the pace at which we have enrolled patients,
which we believe reflects a high level of interest in both the
EXCEED-ET and ECLIPSE-PV trials. Our goal with each of these
programs is to redefine the early treatment paradigm of
myeloproliferative neoplasms.”
Follow PharmaEssentia USA on Twitter and LinkedIn for news and
updates.
About Polycythemia Vera (PV)
Polycythemia vera (PV) is a cancer originating from a
disease-initiating stem cell in the bone marrow resulting in a
chronic increase of red blood cells, white blood cells, and
platelets. PV may result in cardiovascular complications such as
thrombosis and embolism, and often transforms to secondary
myelofibrosis or leukemia. While the molecular mechanism underlying
PV is still subject of intense research, current results point to a
set of acquired mutations, the most important being a mutant form
of JAK2.1
About Essential Thrombocythemia (ET)
Essential thrombocythemia (ET) is a myeloproliferative neoplasm
(MPN) characterized by an overproduction of platelets in the blood
that results from a genetic mutation; data indicates a JAK2 gene
mutation is present in approximately half of diagnosed patients. ET
is estimated to affect up to 57 per 100,000 people in the U.S. The
disease is most commonly diagnosed through routine blood work and
is most common in people over the age of 50, with women 1.5 more
times more likely to be diagnosed than men. As a chronic,
progressive disease, ET requires regular monitoring and appropriate
treatment. Over time, the disease may progress into more deadly
conditions such as myelofibrosis or acute leukemia.2,3
About BESREMi® (ropeginterferon alfa-2b-njft)
BESREMi is an innovative monopegylated, long-acting interferon.
With its unique pegylation technology, BESREMi has a long duration
of activity in the body and is aimed to be administered once every
two weeks (or every four weeks with hematological stability for at
least one year), allowing flexible dosing that helps meet the
individual needs of patients.
BESREMi has orphan drug designation for the treatment of
polycythemia vera (PV) in adults in the United States. The product
was approved by the European Medicines Agency (EMA) in 2019, by the
US Food and Drug Administration (FDA) in 2021, and has recently
received approval in Taiwan and South Korea. The drug candidate was
invented by PharmaEssentia and is manufactured in the company’s
Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA
in January 2018. PharmaEssentia retains full global intellectual
property rights for the product in all indications.
BESREMi was approved with a boxed warning for risk of serious
disorders including aggravation of neuropsychiatric, autoimmune,
ischemic and infectious disorders.
Please see full Prescribing Information, including Boxed
Warning.
Indication
BESREMi is indicated for the treatment of adults with
polycythemia vera.
Important Safety Information
WARNING: RISK OF SERIOUS DISORDERS
Interferon alfa products may cause or
aggravate fatal or life-threatening neuropsychiatric, autoimmune,
ischemic, and infectious disorders. Patients should be monitored
closely with periodic clinical and laboratory evaluations. Therapy
should be withdrawn in patients with persistently severe or
worsening signs or symptoms of these conditions. In many, but not
all cases, these disorders resolve after stopping therapy.
CONTRAINDICATIONS
- Existence of, or history of severe psychiatric disorders,
particularly severe depression, suicidal ideation, or suicide
attempt
- Hypersensitivity to interferons including interferon alfa-2b or
any of the inactive ingredients of BESREMi.
- Moderate (Child-Pugh B) or severe (Child-Pugh C) hepatic
impairment
- History or presence of active serious or untreated autoimmune
disease
- Immunosuppressed transplant recipients
WARNINGS AND PRECAUTIONS
- Depression and Suicide: Life-threatening or fatal
neuropsychiatric reactions have occurred in patients receiving
interferon alfa-2b products, including BESREMi. These reactions may
occur in patients with and without previous psychiatric illness.
Other central nervous system effects, including suicidal ideation,
attempted suicide, aggression, bipolar disorder, mania and
confusion have been observed with other interferon alfa products.
Closely monitor patients for any symptoms of psychiatric disorders
and consider psychiatric consultation and treatment if such
symptoms emerge. If psychiatric symptoms worsen, it is recommended
to discontinue BESREMi therapy.
- Endocrine Toxicity: These toxicities may include worsening
hypothyroidism and hyperthyroidism. Do not use BESREMi in patients
with active serious or untreated endocrine disorders associated
with autoimmune disease. Evaluate thyroid function in patients who
develop symptoms suggestive of thyroid disease during BESREMi
therapy. Discontinue BESREMi in patients who develop endocrine
disorders that cannot be adequately managed during treatment with
BESREMi.
- Cardiovascular Toxicity: Toxicities may include cardiomyopathy,
myocardial infarction, atrial fibrillation and coronary artery
ischemia. Patients with a history of cardiovascular disorders
should be closely monitored for cardiovascular toxicity during
BESREMi therapy. Avoid use of BESREMi in patients with severe or
unstable cardiovascular disease, (e.g., uncontrolled hypertension,
congestive heart failure (≥ NYHA class 2), serious cardiac
arrhythmia, significant coronary artery stenosis, unstable angina)
or recent stroke or myocardial infarction.
- Decreased Peripheral Blood Counts: These toxicities may include
thrombocytopenia (increasing the risk of bleeding), anemia, and
leukopenia (increasing the risk of infection). Monitor complete
blood counts at baseline, during titration and every 3-6 months
during the maintenance phase. Monitor patients for signs and
symptoms of infection or bleeding.
- Hypersensitivity Reactions: Toxicities may include serious,
acute hypersensitivity reactions (e.g., urticaria, angioedema,
bronchoconstriction, anaphylaxis). If such reactions occur,
discontinue BESREMi and institute appropriate medical therapy
immediately. Transient rashes may not necessitate interruption of
treatment.
- Pancreatitis: Pancreatitis has occurred in 2.2% of patients
receiving BESREMi. Symptoms may include nausea, vomiting, upper
abdominal pain, bloating, and fever. Patients may experience
elevated lipase, amylase, white blood cell count, or altered
renal/hepatic function. Interrupt BESREMi treatment in patients
with possible pancreatitis and evaluate promptly. Consider
discontinuation of BESREMi in patients with confirmed
pancreatitis.
- Colitis: Fatal and serious ulcerative or hemorrhagic/ischemic
colitis have occurred in patients receiving interferon alfa
products, some cases starting as early as 12 weeks after start of
treatment. Symptoms may include abdominal pain, bloody diarrhea,
and fever. Discontinue BESREMi in patients who develop these signs
or symptoms. Colitis may resolve within 1 to 3 weeks of stopping
treatment.
- Pulmonary Toxicity: Pulmonary toxicity may manifest as dyspnea,
pulmonary infiltrates, pneumonia, bronchiolitis obliterans,
interstitial pneumonitis, pulmonary hypertension, and sarcoidosis.
Some events have resulted in respiratory failure or death.
Discontinue BESREMi in patients who develop pulmonary infiltrates
or pulmonary function impairment.
- Ophthalmologic Toxicity: These toxicities may include severe
eye disorders such as retinopathy, retinal hemorrhage, retinal
exudates, retinal detachment and retinal artery or vein occlusion
which may result in blindness. During BESREMi therapy, 23% of
patients were identified with an eye disorder. Eyes disorders ≥5%
included cataract (6%) and dry eye (5%). Advise patients to have
eye examinations before and during BESREMi therapy, specifically in
those patients with a retinopathy-associated disease such as
diabetes mellitus or hypertension. Evaluate eye symptoms promptly.
Discontinue BESREMi in patients who develop new or worsening eye
disorders.
- Hyperlipidemia: Elevated triglycerides may result in
pancreatitis. Monitor serum triglycerides before BESREMi treatment
and intermittently during therapy and manage when elevated.
Consider discontinuation of BESREMi in patients with persistently,
markedly elevated triglycerides.
- Hepatotoxicity: These toxicities may include increases in serum
alanine aminotransferase (ALT), aspartate aminotransferase (AST),
gamma-glutamyl transferase (GGT) and bilirubin. Liver enzyme
elevations have also been reported in patients after long-term
BESREMi therapy. Monitor liver enzymes and hepatic function at
baseline and during BESREMi treatment. Discontinue BESREMi in
patients who develop evidence of hepatic decompensation
(characterized by jaundice, ascites, hepatic encephalopathy,
hepatorenal syndrome or variceal hemorrhage) during treatment.
- Renal Toxicity: Monitor serum creatinine at baseline and during
therapy. Avoid use of BESREMi in patients with eGFR <30 mL/min.
Discontinue BESREMi if severe renal impairment develops during
treatment.
- Dental and Periodontal Toxicity: These toxicities may include
dental and periodontal disorders, which may lead to loss of teeth.
In addition, dry mouth could have a damaging effect on teeth and
mucous membranes of the mouth during long-term treatment with
BESREMi. Patients should have good oral hygiene and regular dental
examinations.
- Dermatologic Toxicity: These toxicities have included skin
rash, pruritus, alopecia, erythema, psoriasis, xeroderma,
dermatitis acneiform, hyperkeratosis, and hyperhidrosis. Consider
discontinuation of BESREMi if clinically significant dermatologic
toxicity occurs.
- Driving and Operating Machinery: BESREMi may impact the ability
to drive and use machinery. Patients should not drive or use heavy
machinery until they know how BESREMi affects their abilities.
Patients who experience dizziness, somnolence or hallucination
during BESREMi therapy should avoid driving or using
machinery.
- Embryo-Fetal Toxicity: Based on the mechanism of action,
BESREMi can cause fetal harm when administered to a pregnant woman.
Pregnancy testing is recommended in females of reproductive
potential prior to treatment with BESREMi. Advise females of
reproductive potential to use an effective method of contraception
during treatment with BESREMi and for at least 8 weeks after the
final dose.
ADVERSE REACTIONS
The most common adverse reactions reported in > 40% of
patients in the PEGINVERA study (n=51) were influenza-like illness,
arthralgia, fatigue, pruritis, nasopharyngitis, and musculoskeletal
pain. In the pooled safety population (n=178), the most common
adverse reactions greater than 10%, were liver enzyme elevations
(20%), leukopenia (20%), thrombocytopenia (19%), arthralgia (13%),
fatigue (12%), myalgia (11%), and influenza-like illness (11%).
DRUG INTERACTIONS
Patients on BESREMi who are receiving concomitant drugs which
are CYP450 substrates with a narrow therapeutic index should be
monitored to inform the need for dosage modification for these
concomitant drugs. Avoid use with myelosuppressive agents and
monitor patients receiving the combination for effects of excessive
myelosuppression. Avoid use with narcotics, hypnotics or sedatives
and monitor patients receiving the combination for effects of
excessive CNS toxicity.
USE IN SPECIFIC POPULATIONS
- Pregnancy: Based on mechanism of action and the role of
interferon alfa in pregnancy and fetal development, BESREMi may
cause fetal harm and should be assumed to have abortifacient
potential when administered to a pregnant woman. There are adverse
effects on maternal and fetal outcomes associated with polycythemia
vera in pregnancy. Advise pregnant women of the potential risk to a
fetus.
- Lactation: There are no data on the presence of BESREMi in
human or animal milk, the effects on the breastfed child, or the
effects on milk production. Because of the potential for serious
adverse reactions in breastfed children from BESREMi, advise women
not to breastfeed during treatment and for 8 weeks after the final
dose.
- Females of Reproductive Potential: BESREMi may cause
embryo-fetal harm when administered to a pregnant woman. Pregnancy
testing prior to BESREMi treatment is recommended for females of
reproductive potential. Advise female patients of reproductive
potential to use effective contraception during treatment with
BESREMi and for at least 8 weeks after the final dose.
- Pediatric Use: Safety and effectiveness in pediatric patients
have not been established.
- Geriatric Use: In general, dose selection for an elderly
patient should be cautious, usually starting at the low end of the
dosing range, reflecting the greater frequency of decreased
hepatic, renal, or cardiac function and of concomitant disease or
other therapy.
About PharmaEssentia
PharmaEssentia (TPEx: 6446), headquartered in Taipei, Taiwan, is
a global and rapidly growing biopharmaceutical innovator.
Leveraging deep expertise and proven scientific principles,
PharmaEssentia aims to deliver effective new biologics for
challenging diseases in the areas of hematology, oncology, and
immunology with one approved product and a diversifying pipeline.
Founded in 2003 by a team of Taiwanese-American executives and
renowned scientists from U.S. biotechnology and pharmaceutical
companies, today PharmaEssentia is expanding its global presence
with operations in the U.S., Japan, China, and Korea, along with a
world-class biologics production facility in Taichung, Taiwan.
For more information about PharmaEssentia USA, visit the
website, LinkedIn or Twitter.
Forward Looking Statement
This press release may contain forward looking statements,
including statements regarding the clinical benefits to be derived
from ropeginterferon alfa-2b, the commercial opportunity and
competitive positioning, new indications or labeling for
ropeginterferon alfa-2b, and business prospects for ropeginterferon
alfa-2b. For those statements, we claim the protection of the safe
harbor for forward-looking statements contained in the Private
Securities Litigation Reform Act of 1995 and similar legislation
and regulations under Taiwanese law. These forward-looking
statements are based on management expectations and assumptions as
of the date of this press release, and actual results may differ
materially from those in these forward looking statements as a
result of various factors. These factors include whether BESREMi is
successfully commercialized and adopted by physicians and patients,
the extent to which reimbursement is available for BESREMi, and the
ability to receive FDA and other regulatory approvals for
additional indications for BESREMi. We do not undertake to update
any of these forward-looking statements to reflect events or
circumstances that occur after the date hereof.
© 2024 PharmaEssentia Corporation. All rights reserved.
PharmaEssentia, the PharmaEssentia logo, and BESREMi are
trademarks or registered trademarks of PharmaEssentia
Corporation.
1 Cerquozzi S, Tefferi A. Blast transformation and fibrotic
progression in polycythemia vera and essential thrombocythemia: a
literature review of incidence and risk factors. Blood Cancer J.
2015;5, e366; DOI:10.1038/bcj.2015.95
2 Mehta J, Wang H, Iqbal SU, Mesa R. Epidemiology of
myeloproliferative neoplasms in the United States. Leuk Lymphoma.
2014 Mar;55(3):595-600
3 “What is Essential Thrombocythemia?” MPN Research Foundation.
2020. Available at:
http://www.mpnresearchfoundation.org/Essential-Thrombocythemia
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version on businesswire.com: https://www.businesswire.com/news/home/20240625915519/en/
Media Contacts: Muriel Huang,
muriel_huang@pharmaessentia.com