PhaseBio Pharmaceuticals, Inc. (Nasdaq: PHAS), a clinical-stage
biopharmaceutical company focused on the development and
commercialization of novel therapies for cardiopulmonary diseases,
today announced that PB2452 has been granted PRIority MEdicines
(PRIME) designation by the European Medicines Agency (EMA) for
reversal of the antiplatelet effects of ticagrelor in patients with
uncontrolled major or life-threatening bleeding or requiring urgent
surgery or an invasive procedure. Additionally, PhaseBio announced
receipt of written guidance from the Committee for Medicinal
Products for Human Use (CHMP) of the EMA that generally agrees with
PhaseBio’s proposed development plan for PB2452. After reviewing
the Scientific Advice from CHMP and based on prior interactions
with the U.S. Food and Drug Administration (FDA), PhaseBio believes
that the development plan for PB2452 has been designed to support
regulatory filings in the United States and the European Union.
Specifically, based on the written guidance, CHMP appears aligned
with PhaseBio’s plan to conduct a non-randomized, open-label Phase
3 trial of major bleeding and urgent surgical populations to
support a Marketing Authorization Application (MAA) for PB2452.
John Lee, M.D., Ph.D., the Chief Medical Officer
of PhaseBio, said, "We are extremely pleased that the EMA
recognizes the potential of PB2452 to help address a significant
unmet medical need by granting PRIME designation to PB2452 and
generally agreeing to our overall development plan in support of an
EU MAA submission. Building on the Breakthrough Therapy Designation
for PB2452 granted by the FDA in April 2019, we believe that EMA
PRIME designation, coupled with the written Scientific Advice we
recently received from CHMP, further validates the potential of our
streamlined development program. We are committed to working
closely with regulatory agencies to make PB2452 available to
patients and care providers as quickly as possible."
PB2452 Granted PRIME Designation
PRIME designation is granted by the EMA to
enhance support for the development of medicines that demonstrate
the potential to address substantial unmet medical need based on
early clinical data. The EMA prioritizes PRIME designated drugs for
special support, including enhanced interactions and dialogue with
EMA during development, as well as a pathway for accelerated
evaluation and review for marketing authorization. The program is
intended to optimize development plans and potentially expedite the
review and approval process so that these medicines may reach
patients as early as possible.
In its review comments for PRIME designation,
EMA recognized the unmet need for an effective reversal agent for
ticagrelor-induced platelet inhibition. EMA acknowledged that,
while infrequent, ticagrelor-related major bleeding events are
associated with relevant morbidity and mortality. Urgent surgical
interventions (such as coronary artery bypass grafting) that cannot
be delayed to allow full washout of ticagrelor are associated with
an increased risk for intra- and post-procedural bleeding events.
EMA also acknowledged that there are no established effective
treatment options to reverse ticagrelor-induced platelet
inhibition.
EMA further agreed that the preliminary clinical
data provided in the application suggested that PB2452 is likely to
be able to address, to a significant extent, the targeted unmet
medical need. As the basis of the application, EMA reviewed
PB2452 clinical trial results from the recently completed Phase 2a
study, which demonstrated immediate and sustained reversal of the
antiplatelet effects of ticagrelor in older and elderly patients
treated with dual antiplatelet therapy (low-dose aspirin and
ticagrelor) and in healthy subjects treated with supratherapeutic
doses of ticagrelor. The clinical data corroborated non-clinical
findings that were also reviewed, which demonstrated selective,
high-affinity binding of PB2452 to ticagrelor and its active
metabolite and its potential to provide rapid reversal of
ticagrelor. Additionally, EMA cited literature evidence indicating
that the platelet-function assays utilized in the PB2452
development program are likely to be predictive of clinical
outcomes in cardiovascular patients.
Scientific Advice Regarding the PB2452
Development Program
Separately, PhaseBio met with the Scientific
Advice Working Party (SAWP) of CHMP to discuss the acceptability of
the overall development program to support an MAA for PB2452.
PhaseBio has concluded these interactions and has now received
written guidance from the CHMP.
With respect to the clinical development
program, CHMP generally agreed with PhaseBio’s plan to conduct a
single-arm, open-label, pivotal clinical study of PB2452 in the
target population of patients on ticagrelor who experience a major
bleeding event or are in need of an urgent surgical procedure.
Regarding the overall number of patients targeted to participate in
the Phase 3 trial, CHMP agreed with PhaseBio’s proposal to conduct
the trial in a total of 200 patients, with results from the first
100 patients expected to be included in the MAA package seeking
conditional marketing authorization (CMA). CHMP also agreed with
PhaseBio’s proposed use of the VerifyNow PRUTest® biomarker as the
primary endpoint for the Phase 3 trial. PhaseBio has used VerifyNow
PRUTest in its Phase 1 and Phase 2a clinical trials, where it
demonstrated a high degree of correlation with other biomarkers
used to measure platelet function.
About PB2452
PB2452 is a novel, recombinant, human monoclonal
antibody antigen-binding fragment designed to reverse the
antiplatelet activity of ticagrelor in major bleeding and urgent
surgery situations. In a Phase 1 clinical trial, PB2452
demonstrated the potential to bring life-saving therapeutic benefit
through immediate and sustained reversal of ticagrelor’s
antiplatelet activity, mitigating concerns regarding bleeding risks
associated with the use of antiplatelet drugs. The Phase 1 clinical
trial of PB2452 in healthy volunteers was published in the New
England Journal of Medicine in March 2019.1 In April 2019, PB2452
received Breakthrough Therapy designation from the FDA.
Breakthrough Therapy designation may be granted by FDA when
preliminary clinical evidence indicates that the drug may
demonstrate substantial improvement over existing therapy. PhaseBio
plans to initiate a single pivotal Phase 3 clinical trial of PB2452
in the first quarter of 2020 to support a Biologics License
Application for PB2452 in both major bleeding and urgent surgery
indications. There are currently no approved reversal agents for
ticagrelor or any other antiplatelet drugs.
About PhaseBio
PhaseBio Pharmaceuticals, Inc. is a
clinical-stage biopharmaceutical company focused on the development
and commercialization of novel therapies for cardiopulmonary
diseases. The company’s pipeline includes: PB2452, a novel reversal
agent for the antiplatelet therapy ticagrelor; PB1046, a
once-weekly vasoactive intestinal peptide receptor agonist for the
treatment of pulmonary arterial hypertension; and PB6440, an oral
agent for the treatment of resistant hypertension. PhaseBio’s
proprietary elastin-like-polypeptide technology platform enables
the development of therapies with potential for less-frequent
dosing and improved pharmacokinetics, including PB1046, and drives
both internal and partnership drug development opportunities.
PhaseBio is located in Malvern, PA and San Diego, CA. For more
information, please visit www.phasebio.com.
Forward-Looking Statements
This press release contains forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995. Words such as “anticipates,” “believes,”
“expects,” “intends,” “projects,” and “future” or similar
expressions are intended to identify forward-looking
statements.
Forward-looking statements include statements
concerning or implying the conduct or timing of our clinical trials
and our research, development and regulatory plans for PB2452,
the potential for PB2452 to receive regulatory approval from the
FDA or equivalent foreign regulatory agencies, and whether, if
approved, PB2452 will be successfully distributed and marketed.
Forward-looking statements are based on management's current
expectations and are subject to various risks and uncertainties
that could cause actual results to differ materially and adversely
from those expressed or implied by such forward-looking statements.
Accordingly, these forward-looking statements do not constitute
guarantees of future performance, and you are cautioned not to
place undue reliance on these forward-looking statements.
Risks regarding our business are described in
detail in our Securities and Exchange Commission filings,
including in our Quarterly Report on Form 10-Q for the quarter
ended September 30, 2019. These forward-looking statements
speak only as of the date hereof, and PhaseBio
Pharmaceuticals, Inc. disclaims any obligation to update these
statements except as may be required by law.
Investor Contact:
John SharpPhaseBio Pharmaceuticals, Inc. Chief Financial
Officer(610) 981-6506john.sharp@phasebio.com
Media Contact:
Will ZasadnyCanale Communications Account Director(619)
961-8848will@canalecomm.com
1 Bhatt DL, Pollack CV, Weitz JI, et al. Antibody-Based
Ticagrelor Reversal Agent in Healthy Volunteers. N Engl J Med 2019;
380:1825-1833
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