Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company
focused on the acquisition, development and commercialization of
therapies for serious, rare and life-threatening diseases with
significant unmet medical needs, today reported financial results
for the third quarter ended September 30, 2022 and provided an
update on Acer’s recent corporate developments.
“I am pleased with our team’s progress in the third quarter,
highlighted by the timely resubmission of our New Drug Application
(NDA) following the Complete Response Letter (CRL) issued by the US
Food and Drug Administration (FDA) in response to ACER-001’s NDA
submission,” stated Chris Schelling, CEO and Founder of Acer. “As
the FDA continues its review of our resubmitted NDA for UCDs, we
remain focused on advancing ACER-001 for the treatment of Maple
Syrup Urine Disease (MSUD), having submitted our (Investigational
New Drug) IND application to the FDA for a Phase 2a trial and
receiving Orphan Drug Designation (ODD) from the European
Commission.”
“In addition to the advancement of ACER-001, we made significant
progress with the continued development of our other pipeline
programs, including the initiation of our pivotal, Phase 3 DiSCOVER
trial of EDSIVO™ (celiprolol) for the treatment of COL3A1-positive
vascular Ehlers-Danlos Syndrome (vEDS) patients -- the only ongoing
clinical trial in this patient population, to our knowledge -- and
expansion of our ACER-801 program into treatment and prevention of
Post-traumatic Stress Disorder (PTSD) through an
investigator-sponsored trial to be conducted by the University of
North Carolina (UNC),” Schelling continued. “And early next year,
we look forward to reporting topline results from our ongoing Phase
2a clinical trial of ACER-801 for the treatment of moderate to
severe Vasomotor Symptoms (VMS) in post-menopausal women.”
Q3 and Recent Highlights
- ACER-001 (sodium phenylbutyrate)
- In July 2022, Acer resubmitted its NDA for ACER-001 to the FDA
following receipt of a CRL in June 2022, in which the FDA stated
that satisfactory inspection of Acer’s third-party contract
packaging manufacturer is required before the ACER-001 NDA may be
approved
- In July 2022, the FDA accepted Acer’s NDA resubmission (Class
2) and assigned a Prescription Drug User Fee Act (PDUFA) target
action date of January 15, 2023
- In July 2022, the China National Intellectual Property
Administration (CNIPA) issued a utility model patent for ACER-001
covering certain dosage form claims related to ACER-001’s
polymer-coated formulation, with an expiration date of August 24,
2031
- In July 2022, Acer submitted its IND application to the FDA,
which is now in effect, for the investigation of ACER-001 in a
Phase 2a, open-label dose-ranging efficacy clinical trial that
would evaluate the treatment effect of different doses of ACER-001
on blood leucine and other branched-chain amino acid (BCAA) levels
in MSUD patients (initiation of this trial is subject to additional
capital)
- In August 2022, the European Commission granted orphan
medicinal product designation in the EU to ACER-001 for the
potential treatment of MSUD
- In October 2022, Acer received a Notice of Allowance from the
US Patent and Trademark Office (USPTO) for US patent application
No. 16/624,834 for claims related to a kit comprising sodium
phenylbutyrate or glycerol phenylbutyrate and sodium
benzoate
- ACER-801 (osanetant)
- In October 2022, Acer announced expansion of its ACER-801
(osanetant) program into PTSD, an indication that affects over 12
million adults in the US alone. The worldwide exclusive rights,
related patents and patent applications, were licensed from Emory
University based on preclinical data in which osanetant was able to
block fear memory consolidation in mice
- In October 2022, the Department of Defense (DoD) awarded a $3
million grant to the UNC Institute for Trauma Recovery to support a
proposed 180-patient, randomized, placebo-controlled trial
evaluating osanetant in trauma patients
- EDSIVO™ (celiprolol)
- In October 2022, Acer announced the USPTO issued a Notice of
Allowance for Acer’s patent application No. 16/930,208 for claims
related to certain methods of treating vEDS with celiprolol
- Corporate
- Ended Q3 2022 with $6.4 million in cash and cash equivalents,
which Acer believes will be sufficient to fund its currently
anticipated operating and capital requirements into, but not
through, the fourth quarter of 2022
Anticipated Milestones (Subject to Available
Capital)
- ACER-001 (sodium phenylbutyrate)
- January 15, 2023: The FDA has assigned a new PDUFA target
action date of January 15, 2023, on the pending NDA, following the
FDA’s acceptance for review of Acer’s resubmitted NDA for ACER-001
for the treatment of patients with UCDs
- 2023: Acer plans to initiate in 2023 its Phase 2a trial to
evaluate the efficacy and safety of ACER-001 for the potential
treatment of patients with MSUD
- ACER-801 (osanetant)
- Q1 2023: Acer expects to announce topline results in Q1 2023
from its ongoing Phase 2a randomized, double-blind,
placebo-controlled, dose-ranging trial of ACER-801 for the
treatment of moderate to severe VMS in post-menopausal women
- H1 2023: UNC expects to initiate in H1 2023 the proposed
investigator-sponsored trial (OASIS) to evaluate the potential for
ACER-801 to reduce the frequency and severity of PTSD
- EDSIVO™ (celiprolol)
- Q4 2023: Acer anticipates the DiSCOVER trial for celiprolol in
patients with COL3A1-positive vEDS will be fully enrolled in Q4
2023. Once fully enrolled, the duration of the DiSCOVER trial is
currently estimated to be up to approximately 3.5 years to
completion (based on statistical power calculations and number of
primary events). One interim analysis (based on the number of
primary events occurring) is also planned at approximately 24
months after full enrollment
Q3 2022 Financial Results
Cash Position. Cash and cash equivalents were
$6.4 million as of September 30, 2022, compared to $12.7 million as
of December 31, 2021. Acer believes its cash and cash equivalents
available as of September 30, 2022 will be sufficient to fund its
currently anticipated operating and capital requirements into, but
not through, the fourth quarter of 2022.
Research and Development Expenses. Research and
development expenses were $2.7 million, net of collaboration
funding of $2.2 million, for the three months ended
September 30, 2022, as compared to $1.4 million, net of
collaboration funding of $3.5 million, for the three months ended
September 30, 2021. This increase of $1.3 million was
primarily due to increases in expenses for clinical studies related
to ACER-801 and EDSIVO™, contract research, and employee-related
expenses, partially offset by decreases in expenses related to
contract manufacturing. Research and development expenses related
to ACER-001 decreased in the three months ended September 30, 2022,
resulting in a decrease in the recognition of the collaboration
funding from the Collaboration Agreement with Relief. Research and
development expenses for the three months ended September 30,
2022 were comprised of $2.3 million related to ACER-001, offset by
$2.2 million of collaboration funding; $1.2 million related to
ACER-801; $1.0 million related to EDSIVO™; and $0.4 million related
to other development activities.
General and Administrative Expenses. General
and administrative expenses were $2.6 million, net of collaboration
funding of $1.4 million, for the three months ended
September 30, 2022, as compared to $1.8 million, net of
collaboration funding of $1.0 million, for the three months ended
September 30, 2021. This increase of $0.8 million was
primarily due to increases in employee-related expenses,
precommercial expenses, and audit and consulting fees, partially
offset by an increase in the recognition of the collaboration
funding from the Collaboration Agreement with Relief.
Net Loss. Net loss for the three months ended
September 30, 2022 was $5.0 million, or $0.31 net loss per share
(basic and diluted), compared to a net loss of $3.3 million, or
$0.23 net loss per share (basic and diluted), for the three months
ended September 30, 2021.
For additional information, please see Acer’s Quarterly Report
on Form 10-Q filed today with the Securities and Exchange
Commission (SEC).
Completion of Financial RestatementAcer has
completed the restatement of its previously issued financial
statements for the fiscal quarter ended June 30, 2022 (Restated
Period), and has filed an amended Quarterly Report on Form 10-Q/A
for the Restated Period with the SEC. A non-cash error was made in
applying certain interpretive accounting guidance related to ASC
260, Earnings Per Share, which affected the Company’s presentation
of its diluted earnings per share calculation. The correction of
this non-cash error resulted in a restatement of Acer’s unaudited
condensed interim financial statements and financial data for the
Restated Period. The restatement had no impact on Acer’s cash
position or operating expenses or its ongoing operations or future
plans.
About Acer TherapeuticsAcer is a pharmaceutical
company focused on the acquisition, development and
commercialization of therapies for serious rare and
life-threatening diseases with significant unmet medical needs.
Acer’s pipeline includes four investigational programs: ACER-001
(sodium phenylbutyrate) for treatment of various inborn errors of
metabolism, including urea cycle disorders (UCDs) and Maple Syrup
Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced
Vasomotor Symptoms (iVMS) and post-traumatic stress disorder
(PTSD); EDSIVO™ (celiprolol) for treatment of vascular
Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III
collagen (COL3A1) mutation; and ACER-2820 (emetine), a
host-directed therapy against a variety of viruses, including
cytomegalovirus, Zika, dengue, Ebola and COVID-19. For more
information, visit www.acertx.com.
Acer Forward-Looking StatementsThis press
release contains “forward-looking statements” that involve
substantial risks and uncertainties for purposes of the safe harbor
provided by the Private Securities Litigation Reform Act of 1995.
All statements, other than statements of historical facts, included
in this press release are forward-looking statements. Examples of
such statements include, but are not limited to, statements
regarding the sufficiency and duration of our cash and cash
equivalents, our anticipated milestones, and our plans with respect
to future clinical trials, results and timing thereof for ACER 001,
ACER 801 and EDSIVO™. Our pipeline products (including ACER-801)
are under investigation and their safety and efficacy have not been
established and there is no guarantee that any of our
investigational products in development will receive health
authority approval or become commercially available for the uses
being investigated. We may not actually achieve the plans, carry
out the intentions or meet the expectations or projections
disclosed in the forward-looking statements and you should not
place undue reliance on these forward-looking statements. Such
statements are based on management’s current expectations and
involve risks and uncertainties. Actual results and performance
could differ materially from those projected in the forward-looking
statements as a result of many factors, including, without
limitation, the availability of financing to fund our pipeline
product development programs and general corporate operations as
well as risks related to drug development and the regulatory
approval process, including the timing and requirements of
regulatory actions. We disclaim any intent or obligation to update
these forward-looking statements to reflect events or circumstances
that exist after the date on which they were made. You should
review additional disclosures we make in our filings with the
Securities and Exchange Commission, including our Annual Report on
Form 10-K and Quarterly Reports on Form 10-Q. You may access these
documents for no charge at http://www.sec.gov.
ACER THERAPEUTICS INC.
CONSOLIDATED STATEMENTS OF
OPERATIONS
(Unaudited)
|
Three Months Ended |
|
|
September 30, |
|
|
2022 |
|
|
2021 |
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
Research and development (in
the three months ended September 30, 2022 and 2021, net of
collaboration funding of $2,178,078 and $3,454,881,
respectively) |
$ |
2,681,680 |
|
|
$ |
1,435,461 |
|
General and administrative (in
the three months ended September 30, 2022 and 2021, net of
collaboration funding of $1,432,889 and $958,590,
respectively) |
|
2,557,370 |
|
|
|
1,843,360 |
|
Loss from operations |
|
(5,239,050 |
) |
|
|
(3,278,821 |
) |
|
|
|
|
|
|
|
|
Other income (expense),
net: |
|
|
|
|
|
|
|
Costs of debt issuance |
|
(122,400 |
) |
|
|
— |
|
Changes in fair value of debt
instruments |
|
324,550 |
|
|
|
— |
|
Interest and other income
(expense), net |
|
19,431 |
|
|
|
(2,471 |
) |
Foreign currency transaction
gain |
|
10,524 |
|
|
|
8,284 |
|
Total other income (expense),
net |
|
232,105 |
|
|
|
5,813 |
|
|
|
|
|
|
|
|
|
Net loss |
$ |
(5,006,945 |
) |
|
$ |
(3,273,008 |
) |
|
|
|
|
|
|
|
|
Net loss per share - basic and
diluted |
$ |
(0.31 |
) |
|
$ |
(0.23 |
) |
|
|
|
|
|
|
|
|
Weighted average common shares
outstanding - basic and diluted |
|
16,089,019 |
|
|
|
14,310,244 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
SELECTED BALANCE SHEET DATA
(Unaudited):
|
September 30, |
|
|
December 31, |
|
|
2022 |
|
|
2021 |
|
|
|
|
|
|
|
|
|
Cash and cash equivalents |
$ |
6,377,923 |
|
|
$ |
12,710,762 |
|
|
|
|
|
|
|
|
|
Collaboration receivable |
$ |
— |
|
|
$ |
5,000,000 |
|
|
|
|
|
|
|
|
|
Prepaid expenses |
$ |
920,710 |
|
|
$ |
1,094,229 |
|
|
|
|
|
|
|
|
|
Other current assets |
$ |
5,436 |
|
|
$ |
9,283,625 |
|
|
|
|
|
|
|
|
|
Property and equipment, net |
$ |
92,502 |
|
|
$ |
114,112 |
|
|
|
|
|
|
|
|
|
Total assets |
$ |
15,324,533 |
|
|
$ |
36,256,951 |
|
|
|
|
|
|
|
|
|
Deferred collaboration
funding |
$ |
10,598,201 |
|
|
$ |
24,487,047 |
|
|
|
|
|
|
|
|
|
Bridge loan payable, at fair
value |
$ |
4,478,359 |
|
|
$ |
— |
|
|
|
|
|
|
|
|
|
Convertible note payable, at fair
value |
$ |
3,603,000 |
|
|
$ |
— |
|
|
|
|
|
|
|
|
|
Total liabilities |
$ |
27,531,350 |
|
|
$ |
37,980,556 |
|
|
|
|
|
|
|
|
|
Total stockholders’ deficit |
$ |
(12,207,317 |
) |
|
$ |
(1,723,605 |
) |
|
|
|
|
|
|
|
|
Acer ContactsCorporate contact:Jim DeNikeAcer
Therapeutics Inc.jdenike@acertx.com+1-844-902-6100
Investor contact:Nick ColangeloGilmartin
Groupnick@gilmartinIR.com+1-332-895-3226
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