Held successful Type B pre-biologics license
application (BLA) meeting with U.S. FDA for future submission of
BLA for bentracimab
BLA submission for bentracimab is planned for
early in the fourth quarter of 2022 and PhaseBio is preparing for
commercialization in U.S., if approved
Presented positive results from Phase 2b trial
for bentracimab at ACC.22
Completed the Process Performance Qualification
(PPQ) campaign demonstrating commercial scale manufacturing ability
for bentracimab
PhaseBio Pharmaceuticals, Inc. (Nasdaq: PHAS), a clinical-stage
biopharmaceutical company focused on the development and
commercialization of novel therapies for cardiovascular diseases,
today reported financial results for the first quarter ended March
31, 2022, and provided an update on corporate activities.
“The first quarter of 2022 was a period of significant momentum
for PhaseBio, as we continued to advance our pipeline and lay the
groundwork for the commercialization of bentracimab in the United
States. Our progress was driven primarily by the successful
completion of a Type B pre-BLA meeting with the FDA, which
positions us to submit our BLA early in the fourth quarter of this
year,” said Jonathan Mow, Chief Executive Officer of PhaseBio
Pharmaceuticals. “In addition to receiving encouraging formal
written feedback from federal regulators, we were pleased to
announce positive results from our completed Phase 2b trial for
bentracimab at ACC.22, data that will be key in bolstering our BLA
submission package. We remain focused on advancing our broader
pipeline for cardiovascular conditions, swiftly moving our planned
BLA submission forward, and progressing our pre-commercial and
manufacturing capabilities as we work to deliver the first
ticagrelor reversal agent to patients with a significant unmet
medical need, if approved.”
Bentracimab Program
Highlights
- Announced Successful Pre-BLA Meeting with U.S. FDA for
Bentracimab: In May 2022, PhaseBio announced the completion of
a successful Type B pre-biologics license application (pre-BLA)
meeting with the U.S. Food and Drug Administration (FDA) for
bentracimab. Based on this pre-BLA meeting and the formal written
minutes received from the FDA, PhaseBio intends to submit the BLA
for bentracimab early in the fourth quarter of 2022. During the
pre-BLA meeting, the FDA agreed that the company’s plans to submit
a BLA with data from 25-30 patients with uncontrolled bleeding,
together with data from the fully completed surgical cohort,
appeared reasonable to support a label with both bleeding and
surgical indications, but would be a review issue based on the data
submitted. To date, and subject to final adjudication, the
REVERSE-IT trial has enrolled 35 patients taking ticagrelor who
experienced uncontrolled bleeding events. PhaseBio previously
intended to base its BLA submission on data from the interim
analysis of the Phase 3 REVERSE-IT trial published in December
2021, and the recently presented Phase 2b trial data. Based on the
feedback received from the FDA during the pre-BLA meeting, PhaseBio
will include in the BLA submission the additional bleeding patients
who have enrolled in the REVERSE-IT trial since the pre-specified
interim analysis was completed to support a potential bleeding
indication.
- Presented Positive Results from Phase 2b Trial for
Bentracimab: In April 2022, PhaseBio announced the complete
results from its Phase 2b clinical trial of bentracimab and
presented these results during a late breaking featured clinical
research presentation at the American College of Cardiology’s 71st
annual scientific session (ACC.22). The Phase 2b trial was a
multi-center, randomized, double-blind, placebo-controlled study,
which enrolled a total of 205 older volunteers (50-80 years old),
with 154 subjects receiving bentracimab and 51 subjects receiving
placebo, after all were pretreated with dual antiplatelet therapy
composed of ticagrelor and low-dose aspirin. The primary efficacy
endpoint for the Phase 2b trial was reversal of ticagrelor’s
inhibition of platelet function in actively treated subjects versus
placebo as measured using the point-of-care VerifyNow® PRUTest®
platelet function assay (VerifyNow). In the Phase 2b trial,
bentracimab significantly restored platelet function within five
minutes of administration (p<0.001), as measured by multiple
assays including VerifyNow. The reversal results were tightly
correlated across all assays used in the study to measure platelet
function. VerifyNow is also the primary measurement used to
evaluate efficacy in the ongoing REVERSE-IT Phase 3 trial. These
complete safety and efficacy data from the Phase 2b trial are
consistent with results from PhaseBio’s previously completed Phase
1 trial, conducted in healthy younger volunteers treated with
ticagrelor alone and not aspirin, and its Phase 2a trial, conducted
in healthy, older (ages 50-80) subjects on dual antiplatelet
therapy of ticagrelor and low-dose aspirin and with topline results
previously announced for the Phase 2b trial on November 3,
2021.
- Announced Completion of Bentracimab BLA Enabling
Manufacturing: In March 2022, PhaseBio announced the completion
of the drug substance and drug product Process Performance
Qualification (PPQ) campaign for bentracimab. The PPQ campaign
consisted of multiple commercial scale runs required for the
validation of the bentracimab manufacturing process and the
demonstration of batch-to-batch manufacturing consistency, at
commercial scale. The inclusion of commercial-scale material in the
company’s completed Phase 2b trial and ongoing Phase 3 trial for
bentracimab, coupled with the completion of the PPQ campaign,
positions PhaseBio to be ready to supply global demand for
bentracimab at launch, once approved. PhaseBio remains on track to
submit the bentracimab BLA to the FDA early in the fourth quarter
of 2022.
- SFJ Financing and Co-Development Agreement Update: In
January 2020, PhaseBio entered into the SFJ Agreement, pursuant to
which SFJ provides the company funding to support the global
development of bentracimab. Under the agreement, SFJ agreed to pay
the company up to $120.0 million to support the clinical
development of bentracimab. In addition to $90.0 million of initial
funding, the company has elected to receive an additional $30.0
million of funding having met specific, pre-defined clinical
development milestones for bentracimab. From the inception of the
SFJ Agreement through March 31, 2022, SFJ has provided funding and
paid for amounts on the company’s behalf in the aggregate amount of
$94.6 million. PhaseBio expects that SFJ will fund or reimburse an
additional $25.4 million of clinical trial costs and other
expenses.
Quarter Ending Mar. 31,
2022
- Cash and cash equivalents on March 31, 2022, were $18.7
million, compared to $41.8 million at December 31, 2021. The
decrease primarily reflects cash used in operating activities.
- Net loss for the quarter was $11.1 million, compared to a net
loss of $27.4 million for the prior-year period.
- Research and development expense decreased to $14.3 million, as
compared to $22.3 million for the same period in 2021. The decrease
was primarily attributable to greater drug manufacturing activity
in 2021, higher study site startup costs in 2021 related to the
Phase 2b trial bentracimab, and higher costs in 2021 related to the
Phase 2b trial of pemziviptadil, which was voluntarily ended in the
fourth quarter of 2021. The decreases in research and development
spending were partially offset by an increase in personnel costs
and other costs associated with our general research and
development efforts.
- General and administrative expense increased to $4.0 million,
compared to $3.3 million for prior-year period.
About Bentracimab (PB2452)
Bentracimab is a novel, recombinant, human monoclonal antibody
antigen-binding fragment designed to reverse the antiplatelet
activity of ticagrelor in patients who present with uncontrolled
bleeding or require surgery. In a Phase 1 clinical trial,
bentracimab 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 this antiplatelet drug.
Data from the Phase 1 clinical trial of bentracimab in healthy
volunteers was published in the New England Journal of Medicine in
March 2019. In April 2019, bentracimab received Breakthrough
Therapy Designation from the FDA. In September 2019, PhaseBio
completed a Phase 2a trial in which bentracimab was investigated in
healthy, older subjects on dual antiplatelet therapy of ticagrelor
and low-dose aspirin. Additionally, the Phase 2a trial investigated
a bentracimab regimen for the reversal of supratherapeutic doses of
ticagrelor in healthy younger subjects. In November 2021, PhaseBio
completed a Phase 2b trial in which bentracimab was investigated in
older subjects on dual antiplatelet therapy of ticagrelor and
low-dose aspirin, with complete results announced and presented in
April 2022. In all active treatment arms in both the Phase 2a and
Phase 2b trials, bentracimab achieved immediate and sustained
reversal of the antiplatelet effects of ticagrelor and was
generally well-tolerated, with only minor adverse events reported.
These results are consistent with the results observed in healthy
younger subjects treated with ticagrelor in the previously
published Phase 1 trial. PhaseBio initiated REVERSE-IT, a pivotal
Phase 3 clinical trial of bentracimab, in March 2020 to support a
potential Biologics License Application for bentracimab to treat
patients with uncontrolled bleeding or requiring surgery. Interim
results from the Phase 3 REVERSE-IT trial were presented in
November 2021 and subsequently published in NEJM Evidence in
December 2021.
About PhaseBio
PhaseBio Pharmaceuticals, Inc. is a clinical-stage
biopharmaceutical company focused on the development and
commercialization of novel therapies for cardiovascular diseases.
The Company’s pipeline includes: bentracimab (PB2452), a novel
reversal agent for the antiplatelet therapy ticagrelor; 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, 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, and follow us on
Twitter @PhaseBio and LinkedIn.
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,” “potential,” “projects,” “target,” “will,” “would” 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, including
enrollment, and our research, development and regulatory plans for
our product candidates, the timing of availability or disclosure of
data from those clinical trials and the timing of planned
regulatory submissions, the potential for these product candidates
to receive regulatory approval from the FDA, EMA or equivalent
foreign regulatory agencies, and whether, if approved, these
product candidates will be successfully distributed, marketed and
commercialized, including having sufficient product supply at
launch, and our ability to complete post-approval requirements.
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 Annual
Report on Form 10-K for the year ended December 31, 2021. 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.
PhaseBio Pharmaceuticals,
Inc.
Condensed Balance
Sheets
(in thousands)
(unaudited)
March 31,
2022
December 31,
2021
Assets:
Cash and cash equivalents
$
18,688
$
41,800
Other receivables, prepaid expenses and
other current assets
8,881
6,984
Property and equipment, net
9,697
10,230
Operating lease right-of-use assets
1,341
1,469
Other non-current assets
57
57
Total assets
$
38,664
$
60,540
Liabilities and stockholders' equity
(deficit):
Current portion of long-term debt
$
5,425
$
5,413
Current portion of deferred sublicense
revenue
$
1,594
1,547
Accounts payable, accrued expenses and
other current liabilities
14,669
20,923
Long-term debt, net
—
1,359
Operating lease liabilities, net
969
1,073
Deferred sublicense revenue, net
7,458
7,622
Development derivative liability
110,944
114,843
Other long-term liabilities
—
794
Stockholders’ equity (deficit)
(102,395
)
(93,034
)
Total liabilities and stockholders' equity
(deficit)
$
38,664
$
60,540
PhaseBio Pharmaceuticals,
Inc.
Condensed Statements of
Operations
(in thousands, except share
and per share amounts)
(unaudited)
Quarter Ended March
31,
2022
2021
Sublicense Revenue
$
117
$
—
Operating expenses:
Research and development
14,336
22,320
General and administrative
4,009
3,327
Total operating expenses
18,345
25,647
Loss from operations
(18,228
)
(25,647
)
Other expense
7,087
(1,711
)
Net loss
$
(11,141
)
$
(27,358
)
Net loss per common share, basic and
diluted
$
(0.23
)
$
(0.87
)
Weighted average common shares
outstanding, basic and diluted
48,635,034
31,282,662
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220516005344/en/
Investor Contact: John Sharp PhaseBio Pharmaceuticals,
Inc. Chief Financial Officer (610) 981-6506
john.sharp@phasebio.com
Media Contact: Will Zasadny Canale Communications, Inc.
(619) 961-8848 will.zasadny@canalecomm.com
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