PhaseBio Pharmaceuticals, Inc. (Nasdaq: PHAS), a clinical-stage
biopharmaceutical company focused on the development and
commercialization of novel therapies for orphan diseases, today
announced the receipt of written minutes from the End-of-Phase 1
meeting with the U.S. Food and Drug Administration (“FDA”) that was
held in July 2019. The End-of-Phase 1 meeting was focused on
gaining alignment with the FDA regarding the clinical and
regulatory pathway for a potential U.S. approval of PB2452, a
novel, recombinant, human monoclonal antibody antigen-binding
fragment, or Fab, designed to reverse the antiplatelet activity of
ticagrelor in major bleeding and urgent surgery situations. Based
on the written minutes from the End-of-Phase 1 meeting, PhaseBio
believes that it has reached general agreement with the FDA on the
overall design of a single, non-randomized, open label Phase 3
trial of major bleeding and urgent surgical populations to support
the submission of a Biologics License Application (“BLA”) for
potential accelerated approval of PB2452.
End-of-Phase 1 Meeting
Details
During the End-of-Phase 1 meeting, the FDA
generally agreed with the proposed clinical development plan and
agreed that Accelerated Approval was the appropriate approval
pathway for PB2452.
To further support safety assessments, the FDA
recommended that PhaseBio include 200 active treatment subjects
across its Phase 1 and Phase 2 trials, which includes the planned
Phase 2b trial that will begin in the fourth quarter of 2019 and
run in parallel to the ongoing Phase 2a and planned Phase 3 trial.
To date, approximately 50 subjects have received PB2452 in the
completed Phase 1 trial and ongoing Phase 2a trial. The Phase
2b trial is expected to enroll a total of 200 subjects, including
150 subjects who will be randomized to receive PB2452.
The FDA also recommended an assessment of PB2452
reversal in patients who may have supratherapeutic blood levels of
ticagrelor as a result of ticagrelor overdosage or drug-drug
interactions. Based on the pharmacokinetic and pharmacodynamic
modeling conducted in earlier preclinical studies and clinical
trials of PB2452, PhaseBio believes it has a clear understanding of
the appropriate dosing regimen to reverse the antiplatelet effects
of supratherapeutic blood levels of ticagrelor and plans to address
this request in its ongoing Phase 2a trial, with dosing of these
subjects to begin this quarter. PhaseBio expects to complete the
Phase 2a trial in the fourth quarter of 2019.
With respect to the pivotal Phase 3 trial
design, the FDA agreed with PhaseBio’s proposed 200 patient,
non-randomized, open-label trial design and the proposed
pharmacodynamic, clinical and safety endpoints. The FDA also agreed
with the 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 to other biomarkers used
to measure platelet function. To support the BLA submission for
Accelerated Approval, the FDA recommended that an interim analysis
of the Phase 3 trial include data from the first 100 subjects
treated with PB2452, with approximately 50 subjects from each of
the major bleeding and surgical populations. To support full
approval for patients with major bleeding or requiring urgent
surgery, the FDA recommended enrollment of 200 total patients in
the Phase 3 trial. PhaseBio expects to initiate the Phase 3 trial
in the first quarter of 2020; based on an estimated 18-month
enrollment timeline, a BLA could potentially be submitted in the
second half of 2022. For post-approval commitments, the FDA
recommended the completion of the remaining portions of the Phase 3
trial and the establishment of a post-approval registry.
“We are pleased with the constructive and
collaborative discussion with FDA officials during the End-of-Phase
1 meeting,” said John Lee, M.D., Ph.D., Chief Medical Officer of
PhaseBio. “We believe the specific next steps discussed at the
meeting, and reflected in the minutes, represent a clear path
toward BLA submission. The level of clarity and collaboration we
received from the FDA is greatly appreciated. With PB2452 now
having a defined development path, coupled with Breakthrough
Therapy designation, we believe that we are well positioned to
execute our strategy to deliver this potentially life-saving
therapy to patients in need as soon as possible.”
Today’s Conference Call Information
PhaseBio will host a conference call and webcast
today at 8:30 a.m. EDT to discuss the End-of-Phase 1 meeting.
Analysts and investors can participate in the conference call by
dialing (866) 221-1776 for domestic callers and (270) 215-9926 for
international callers, using the conference ID 5869719. The webcast
can be accessed live on the Events and Presentations page in the
Investors section of the PhaseBio website, www.phasebio.com. The
webcast will be archived on the company’s website for 90 days and
will be available for telephonic replay for 14 days following the
call by dialing (855) 859-2056 (Domestic) or (404) 537-3406
(International), conference ID 5869719.
About PB2452
PB2452 is a novel, recombinant, human monoclonal
antibody Fab 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 anti-platelet 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 FDA. Breakthrough Designation may be granted by
FDA when preliminary clinical evidence indicates that the drug may
demonstrate substantial improvement over existing therapy. 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 to treat orphan diseases,
with an initial focus on cardiopulmonary disorders. The company’s
lead development candidate is PB2452, a novel reversal agent for
the antiplatelet therapy ticagrelor. PhaseBio is also leveraging
its proprietary elastin-like polypeptide (“ELP”) technology
platform to develop therapies with the potential for less-frequent
dosing and improved pharmacokinetics. PhaseBio’s second product
candidate PB1046, which is based on ELP, is a once-weekly
vasoactive intestinal peptide receptor agonist being developed for
the treatment of pulmonary arterial hypertension.
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, trial design or timing of our
clinical trials, timelines for regulatory submissions, and our
research, development and regulatory plans for PB2452, PB1046 and
our ELP research programs. 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 March
31, 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:Gina Cestari6 Degrees(917)
797-7904gcestari@6degreespr.com
- Bhatt DL, Pollack CV, Weitz JI, et al. Antibody-Based
Ticagrelor Reversal Agent in Healthy Volunteers. N Engl J
Med 2019;Mar 17.
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