- TZIELD is the first disease-modifying therapy in T1D, a
life-threatening autoimmune disease
- In a clinical trial, in Stage 2 T1D patients, TZIELD delayed
the median onset of Stage 3 T1D by 25 months, or approximately 2
years, compared to placebo
- Stage 3 T1D is associated with significant health risks,
including diabetic ketoacidosis, which can be life threatening
- Patients who progress to Stage 3 T1D eventually require insulin
injections for life
- Investor Conference call at 8:00am ET
tomorrow
RED
BANK, N.J., Nov. 17,
2022 /PRNewswire/ -- Provention Bio, Inc. (Nasdaq:
PRVB) (the "Company"), a biopharmaceutical company dedicated to
intercepting and preventing immune-mediated diseases, today
announced that the United States Food and Drug Administration (FDA)
approved the Biologics License Application (BLA) for TZIELD
(teplizumab-mzwv), an anti-CD3-directed antibody, for intravenous
use, as the first and only immunomodulatory treatment to delay the
onset of Stage 3 T1D in adult and pediatric patients aged 8 years
and older with stage 2 T1D. The most common adverse reactions
(>10%) that occurred during treatment and through 28 days after
the last study drug administration from the TN-10 study were
lymphopenia (73% TZIELD, 6% Placebo), rash (TZIELD 36%, Placebo
0%), leukopenia (TZIELD 21%, Placebo 0%) and headache (TZIELD 11%,
Placebo 6%).
![(PRNewsfoto/Provention Bio, Inc.) (PRNewsfoto/Provention Bio, Inc.)](https://mma.prnewswire.com/media/720849/Provention_Bio_Logo.jpg)
"This is a historic occasion for the T1D community and a
paradigm shifting breakthrough for individuals aged 8 years and
older with Stage 2 T1D who now have a therapy approved by the FDA
to delay the onset of Stage 3 disease. It cannot be emphasized
enough how precious a delay in the onset of Stage 3 T1D can be from
a patient and family perspective; more time to live without
and, when necessary, prepare for the burdens, complications
and risks associated with Stage 3 disease" noted Ashleigh Palmer, Co-Founder and CEO of
Provention Bio. "We would especially like to acknowledge and thank
all the researchers, scientists, developers, and investigators,
especially TrialNet and NIDDK; all the clinical trial participants
and their families; the FDA and its reviewers; and JDRF and other
patient advocacy groups and champions who have, over more than two
decades, tirelessly and selflessly contributed to the development
and approval of this first and only therapy to address the
underlying autoimmunity of T1D, thereby fundamentally changing the
course of the disease rather than just treating its symptoms."
"The progression of T1D can be particularly onerous; patients
who progress from Stage 2 to Stage 3 T1D can develop diabetic
ketoacidosis, which can be life threatening and is experienced by
up to 50% of Stage 3 patients at the time of presentation. The
onset of Stage 3 T1D is a life-changing moment - once
insulin-producing cells are no longer capable of maintaining normal
glycemic control, this irreversible condition can lead to the need,
in just one year, for a patient, 1,460 finger sticks to check blood
glucose levels, around 1,100 insulin injections, and experiencing
an average of 127 episodes of hypoglycemia. These complications can
cause stress, fear, and anxiety in patients as they work to manage
their T1D diagnosis and provide perspective on the meaning of a
delay in the onset of Stage 3 T1D." notes Dr. Eleanor Ramos, Chief Medical Officer at
Provention Bio. "As described in the approved label, TZIELD binds
to CD3 (a cell surface antigen present on T lymphocytes) and its
mechanism is believed to involve partial agonistic signaling and
deactivation of pancreatic beta cell autoreactive T lymphocytes.
TZIELD leads to an increase in the proportion of regulatory T cells
and exhausted CD8+ T cells in peripheral blood."
In October 2022 the company
announced a co-promotion agreement for the U.S. launch of TZIELD
for delay in onset of clinical T1D in at-risk individuals with
Sanofi. Olivier Bogillot, Head of U.S. General Medicines,
Sanofi, stated, "This approval is a profound and long-awaited
victory for the diabetes community. We applaud Provention Bio for
its unwavering determination to bring the first ever
disease-modifying therapy for T1D to patients. We look forward to
leveraging Sanofi's established infrastructure and expertise in
endocrinology to deliver for individuals in need across the
U.S."
Provention Bio has launched COMPASS, a patient support program
with a staff of dedicated personnel available to answer questions
and help navigate coverage, reimbursement and access for patients
that are prescribed TZIELD. Provention Bio offers financial
assistance options (e.g. copay assistance) to eligible patients for
out-of-pocket costs. Patients are encouraged to speak to their
healthcare providers to find out whether TZIELD is appropriate for
them, and patients who have been prescribed TZIELD and their
healthcare providers can call 1-844-778-2246, Monday through Friday
from 8AM-8PM EST or email a COMPASS
Navigator at COMPASS@proventionbio.com.
Conference Call and Webcast Information
Provention Bio
will discuss this business update via a conference call tomorrow
at 8:00 am ET. To access the call, please dial
1-888-347-7861 (domestic) or 1-412-902-4247 (international) ten
minutes prior to the start time and ask to be connected to the
"Provention Bio Call." An audio webcast will also be available on
the "Events and Webcasts" page of the Investors section of the
Company's website, www.proventionbio.com. An archived webcast
will be available on the Company's website approximately two hours
after the conference call.
The Unmet Need in T1D
Over 1.8 million Americans have
T1D, an autoimmune disease caused by the destruction of beta cells.
Diagnosis of T1D usually occurs in children and young adults, but
it can happen at any age after symptoms appear when a person cannot
make enough insulin. However, T1D starts in the body long before
any symptoms appear and can be detected at this pre-symptomatic
stage through a blood test. The psychological impact of T1D is hard
to quantify, but a diagnosis is life-altering, and regular
monitoring and maintenance can be extremely stressful. T1D
typically takes more than a decade off a person's life, and life
expectancy is reduced by 16 years on average for people diagnosed
before the age of 10. Insulin therapy and glucose monitoring are
currently the standard of care for treating clinical-stage, or
Stage 3 T1D, and are necessary to keep T1D patients alive. The
constant monitoring and administration of insulin represents a
significant life-long burden for patients.
About TZIELD®
TZIELD (teplizumab-mzwv) is a
CD3-directed antibody indicated to delay the onset of Stage 3 T1D
in adults and pediatric patients aged 8 years and older with Stage
2 T1D. TZIELD injection is supplied as a sterile,
preservative-free, clear and colorless solution in a 2 mg/2 mL
(1 mg/mL) single-dose vial for intravenous use. TZIELD should
be administered by intravenous infusion (over a minimum of 30
minutes) once daily for 14 days. Please see full prescribing
information for the dosing schedule.
If a patient needs help paying for TZIELD, Provention Bio's
Patient Assistance Program may be able to help. While co-pay
amounts vary based on individual coverage, with the Provention Bio
Copay Program, commercially or privately insured individuals
enrolled in COMPASS may pay as little as $0 for TZIELD. If a patient qualifies, their
COMPASS Navigator can help enroll them into the program so they may
be able to lower their out-of-pocket costs. Interested
patients and healthcare providers can contact COMPASS for questions
about our available assistance programs at
COMPASS@proventionbio.com and 1-844-778-2246, Monday through Friday
from 8AM-8PM EST.
Important Safety Information about
TZIELD®
WARNINGS AND PRECAUTIONS
- Cytokine Release Syndrome (CRS): CRS occurred in
TZIELD-treated patients during the treatment period and through 28
days after the last drug administration. Prior to TZIELD treatment,
premedicate with antipyretics, antihistamines and/or antiemetics,
and treat similarly if symptoms occur during treatment. If severe
CRS develops, consider pausing dosing for 1 day to 2 days and
administering the remaining doses to complete the full 14-day
course on consecutive days; or discontinue treatment. Monitor liver
enzymes during treatment. Discontinue TZIELD treatment in patients
who develop elevated alanine aminotransferase or aspartate
aminotransferase more than 5 times the upper limit of normal (ULN)
or bilirubin more than 3 times ULN.
- Serious Infections: Use of TZIELD is not recommended in
patients with active serious infection or chronic infection other
than localized skin infections. Monitor patients for signs and
symptoms of infection during and after TZIELD administration. If
serious infection develops, treat appropriately, and discontinue
TZIELD.
- Lymphopenia: In clinical trials, lymphopenia occurred in
78% of TZIELD-treated patients. For most patients, lymphocyte
levels began to recover after the fifth day of treatment and
returned to pretreatment values within two weeks after treatment
completion and without dose interruption. Monitor white blood cell
counts during the treatment period. If prolonged severe lymphopenia
develops (<500 cells per mcL lasting 1 week or longer),
discontinue TZIELD.
- Hypersensitivity Reactions: Acute hypersensitivity
reactions including serum sickness, angioedema, urticaria, rash,
vomiting and bronchospasm occurred in TZIELD-treated patients. If
severe hypersensitivity reactions occur, discontinue TZIELD and
treat promptly.
- Vaccinations: The safety of immunization with
live-attenuated (live) vaccines in TZIELD-treated patients has not
been studied. TZIELD may interfere with immune response to
vaccination and decrease vaccine efficacy. Administer all
age-appropriate vaccinations prior to starting TZIELD.
-
- Administer live vaccines at least 8 weeks prior to treatment.
Live vaccines are not recommended during treatment, or up to 52
weeks after treatment.
- Administer inactivated (killed) vaccines or mRNA vaccines at
least 2 weeks prior to treatment. Inactivated vaccines are not
recommended during treatment, or 6 weeks after completion of
treatment.
ADVERSE REACTIONS: Most common adverse reactions
(>10%) were lymphopenia, rash, leukopenia, and headache.
Call your doctor for medical advice about side effects. You
are encouraged to report negative side effects of prescription
drugs to the FDA. Visit www.fda.gov/medwatch, or call
1-800-FDA-1088 or contact Provention Bio at 1-844-778-2246.
USE IN SPECIFIC
POPULATIONS
- Pregnancy: May cause fetal harm.
- Lactation: A lactating woman may consider pumping and
discarding breast milk during and for 20 days after TZIELD
administration.
Please see accompanying Prescribing Information.
TN-10 Study
TZIELD was investigated in the TN-10
Study, a pivotal randomized, double-blind, event driven, placebo
controlled clinical trial which evaluated TZIELD for the delay of
T1D (Stage 3, or clinical T1D) in Stage 2 T1D patients, defined by
the presence of two or more T1D-related autoantibodies and
dysglycemia. Seventy-six patients (TZIELD N=44, placebo N=32) were
enrolled ages 8 to 49, with 72% under the age of 18, and randomized
to receive a single 14-day course of either teplizumab or placebo
by IV infusion. The primary efficacy endpoint in this study was the
time from randomization to development of Stage 3 T1D
diagnosis.
In Study TN-10, Stage 3 T1D was diagnosed in 20 (45%) of the
TZIELD-treated patients and in 23 (72%) of the placebo-treated
patients. A Cox proportional hazards model was used to analyze the
time to Stage 3 T1D diagnosis, stratified by age and oral glucose
tolerance test status at randomization. The median time from
randomization to Stage 3 T1D diagnosis was 50 months in the TZIELD
group and 25 months in the placebo group, for a difference of 25
months. With a median follow-up time of 51 months, therapy with
TZIELD resulted in a statistically significant delay in the
development of Stage 3 T1D, hazard ratio 0.41 (95% CI: 0.22 to
0.78; p=0.0066).
The most common adverse reactions (>10%) that occurred during
treatment and through 28 days after the last study drug
administration from the TN-10 study were lymphopenia (73% TZIELD,
6% Placebo), rash (TZIELD 36%, Placebo 0%), leukopenia (TZIELD 21%,
Placebo 0%) and headache (TZIELD 11%, Placebo
6%).
About Sanofi US Co-Promotion
In October 2022, Provention entered into a
co-promotion agreement with Sanofi U.S. for the launch of TZIELD.
Under the terms of the agreement, Sanofi will commit commercial
resources in the United States,
including diabetes field specialists, account directors,
field-based reimbursement, and medical science liaisons to expand
the number of key healthcare professionals reached in the United States. In exchange, Provention
will reimburse field force-related expenses that Sanofi will incur
in connection with commercializing teplizumab under the
agreement.
Provention retains all rights to TZIELD and maintains
responsibility for the commercialization strategy.
About Provention Bio, Inc.
Provention Bio, Inc.
(Nasdaq: PRVB) is a biopharmaceutical company focused on
advancing the development of investigational therapies that may
intercept and prevent debilitating and life-threatening
immune-mediated diseases. The Company's pipeline includes
clinical-stage product candidates that have demonstrated in
pre-clinical or clinical studies proof-of-mechanism and/or
proof-of-concept in autoimmune diseases, including T1D, celiac
disease and lupus. Visit www.ProventionBio.com for more
information and follow us on Twitter: @ProventionBio.
Internet Posting of Information
Provention Bio, Inc.
uses its website, www.proventionbio.com, as a means of
disclosing material nonpublic information and for complying with
its disclosure obligations under Regulation F.D. Such disclosures
will be included on the Company's website in the "News" section.
Accordingly, investors should monitor this portion of the Company's
website, in addition to following its press releases, SEC filings
and public conference calls and webcasts.
Forward-Looking Statements
Certain statements in this
press release are forward-looking, including but not limited to,
statements relating to the unmet need in T1D, the safety and
efficacy of and the ability for TZIELD to address the unmet need
for its approved indication, plans to bring TZIELD to patients and
related commercialization plans with Sanofi. These statements may
be identified by the use of forward-looking words such as "may" and
"potential," among others. These forward-looking statements are
based on the Company's current expectations and actual results
could differ materially. There are a number of factors that could
cause actual events to differ materially from those indicated by
such forward-looking statements. These factors include, but are not
limited to, failure to maintain FDA approval for TZIELD; the
planned commercial launch in the US for TZIELD may not be
successful in part or at all for various reasons including the
actual market size and drug supply needed may not be consistent
with the company's expectations and its executed commercial
readiness plans; the degree to which TZIELD is accepted by patients
and prescribed by physicians; the efficiency of our manufacturing,
sales, distribution and specialty pharmacy network in getting
TZIELD to the market and future economic, competitive,
reimbursement and regulatory conditions that could negatively
impact the commercial launch of TZIELD; the post-marketing
commitment studies for TZIELD may not yield data consistent with
prior results; we may not be able to execute on our business plans
including meeting our expected or planned regulatory milestones and
timelines, clinical development plans and successfully bringing our
product candidates to market, for various reasons, including
factors outside of the Company's control, such as possible
limitations of Company financial and other resources, competition,
manufacturing limitations that may not be anticipated or resolved
for in a timely manner or at all, and regulatory, court or agency
decisions, such as decisions by the United States Patent and
Trademark Office with respect to patents that cover our product
candidates, the potential for noncompliance with FDA regulations;
the potential impacts of COVID-19 on our business and financial
results; changes in law, regulations, or interpretations and
enforcement of regulatory guidance; uncertainties of patent
protection and litigation; competition and the risks listed under
"Risk Factors" in the Company's quarterly report on Form 10-Q for
the quarter ended September 30, 2022 and any subsequent
filings with the Securities and Exchange Commission. As with any
pharmaceutical under development, there are significant risks in
the development, regulatory approval, and commercialization of new
products. Provention does not undertake an obligation to update or
revise any forward-looking statement, whether as a result of new
information, future developments or otherwise, except as may be
required by applicable law. The information set forth herein speaks
only as of the date hereof.
Investor Contacts:
Thierry Chauche, Chief Financial
Officer
tchauche@proventionbio.com
Brendan Strong
Argot Partners
Proventionbio@argotpartners.com
212-600-1902
Media Contact:
Kaelan
Hollon, VP of Communications
khollon@proventionbio.com
202-421-4921
Provention Bio and the Provention Bio logo are registered
Trademarks of Provention Bio, Inc. TZIELD and Provention Bio
COMPASS are trademarks of Provention Bio, Inc.
View original content to download
multimedia:https://www.prnewswire.com/news-releases/tzield-teplizumab-mzwv-approved-by-fda-as-the-first-and-only-treatment-indicated-to-delay-the-onset-of-stage-3-type-1-diabetes-t1d-in-adult-and-pediatric-patients-aged-8-years-and-older-with-stage-2-t1d-301682218.html
SOURCE Provention Bio, Inc.