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 first quarter ended March 31, 2022 and provided an update
on the Company’s recent corporate developments.
“Progress in Q1 2022 was marked by a transformative debt
financing in March, with continued advancement toward potential
commercial launch of ACER-001 for UCDs and further development of
our other pipeline programs,” said Chris Schelling, CEO and Founder
of Acer. “Our advances to date have positioned Acer for potential
achievement of a number of important planned milestones through the
rest of 2022, including an FDA decision on our ACER-001 New Drug
Application in June, the planned initiation of our EDSIVO™ pivotal
Phase 3 trial in vascular Ehlers-Danlos Syndrome under our SPA
agreement with FDA by end of Q2 2022, and ACER-801 Phase 2a trial
results in Vasomotor Symptoms in H2 2022.”
Q1 2022 and Recent Highlights
- ACER-001 (sodium phenylbutyrate)
- Announced in April 2022 the presentation of data evaluating the
bioavailability, bioequivalence and taste attributes of ACER-001
(sodium phenylbutyrate) compared to sodium phenylbutyrate
(BUPHENYL®) powder during poster sessions at the Society for
Inherited Metabolic Disorders (SIMD) Annual Meeting
- Announced in May 2022 the presentation of data evaluating the
bioavailability, bioequivalence and taste attributes of ACER-001
(sodium phenylbutyrate) compared to sodium phenylbutyrate
(BUPHENYL®) powder during poster sessions at the Genetic Metabolic
Dieticians International (GMDI) Conference. Publications from SIMD
and GMDI are available at:
https://www.acertx.com/publications-and-presentations/
- Launched SeeUCDifferently, a national U.S. disease awareness
campaign intended to provide education and information about urea
cycle disorders (UCDs), including www.SeeUCDifferently.com, a new
online resource that provides general UCDs diagnosis and disease
education information
- ACER-801 (osanetant)
- Announced in March 2022 enrollment of the first patient in a
Phase 2a (NCT05325775) randomized, double-blind,
placebo-controlled, dose-ranging trial evaluating the
pharmacokinetics (PK), safety, and efficacy of ACER-801 at
different doses, compared to placebo, for the treatment of moderate
to severe Vasomotor Symptoms (VMS) in post-menopausal women.
Results from this trial could provide proof of concept data in
post-menopausal women and could inform ACER-801 dosing and a
development path forward in patients with induced Vasomotor
Symptoms (iVMS)
- EDSIVO™ (celiprolol)
- In January 2022, the U.S. Food and Drug Administration (FDA)
cleared the celiprolol IND for the treatment of patients with
COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS)
- In April 2022, announced FDA granted celiprolol Breakthrough
Therapy designation (BTD) in the U.S. for the treatment of patients
with COL3A1-positive vEDS
- In May 2022, announced agreement with FDA under a Special
Protocol Assessment (SPA) for pivotal Phase 3 clinical trial of
celiprolol for the treatment of patients with COL3A1-positive vEDS.
More information on the DiSCOVER (Decentralized Study of Celiprolol
on vEDS-related Event Reduction) trial is available at
https://discoverceliprolol.com/
- Corporate
- In February 2022, announced the appointment of Adrian Quartel,
M.D., FFPM, as Chief Medical Officer. Dr. Quartel is an industry
veteran with over 20 years of drug development experience tasked
with overseeing Acer’s clinical development, medical affairs,
regulatory affairs and other scientific and medical functions. Dr.
Quartel joins Acer from Adamas Pharmaceuticals where he served as
Chief Medical Officer overseeing research and development, as well
as medical affairs and regulatory functions. Prior to Adamas, Dr.
Quartel held senior medical leadership positions at BioMarin
Pharmaceutical Inc., Astellas, Chiltern, and ICON Clinical
Research
- In March 2022, announced convertible note and loan financing
facilities for up to $48.5 million with affiliates of Marathon
Asset Management L.P. (Marathon) and SWK Holdings Corporation,
subject to certain conditions. Proceeds from these financings will
be used to advance pipeline and for general corporate purposes.
Further information with respect to the debt financing agreements
with Marathon and SWK is contained in a Current Report on Form 8-K
filed with the Securities and Exchange Commission on March 7,
2022
- Ended Q1 2022 with $20.8 million in cash and cash equivalents,
which Acer believes will be sufficient to fund its currently
anticipated operating and capital requirements into Q3 2022
Anticipated Milestones
- ACER-001 (sodium phenylbutyrate)
- June 5, 2022: FDA has assigned a PDUFA target action date of
June 5, 2022, following its acceptance for filing of the NDA for
ACER-001 for the treatment of patients with UCDs
- End of Q2 2022: Acer plans to submit an Investigational New
Drug (IND) application by the end of Q2 2022 for a clinical study
evaluating ACER-001 in Maple Syrup Urine Disease (MSUD)
- ACER-801 (osanetant)
- H2 2022: Results from the ongoing ACER-801 Phase 2a clinical
trial in women with moderate to severe VMS are anticipated in H2
2022
- EDSIVO™ (celiprolol)
- End of Q2 2022: Acer intends to initiate by end of Q2 2022 the
pivotal Phase 3, randomized, double-blind, placebo-controlled,
decentralized clinical trial for celiprolol for patients with
COL3A1-positive vEDS under its SPA agreement with FDA. The duration
of the DiSCOVER clinical trial is currently estimated to be
approximately 3.5 years to completion, once fully enrolled, and
will require additional capital beyond Q3 2022
Q1 2022 Financial Results
Cash position. Cash and cash equivalents were
$20.8 million as of March 31, 2022, compared to $12.7 million as of
December 31, 2021. Acer believes its cash and cash equivalents
available as of March 31, 2022 will be sufficient to fund its
currently anticipated operating and capital requirements into Q3
2022.
Research and Development Expenses. Research and
development expenses were $3.2 million, net of collaboration
funding of $3.0 million, for the three months ended March 31, 2022,
compared to $2.0 million, net of collaboration funding of $0.3
million, for the three months ended March 31, 2021. This increase
of $1.2 million was primarily due to increases in contract
manufacturing expenses, employee-related expenses including a
one-time bonus accrual, and clinical and medical affairs expenses,
partially offset by the recognition of $3.0 million of the
collaboration funding from the Collaboration Agreement with Relief.
Research and development expenses for the three months ended March
31, 2022 were comprised of $3.1 million related to ACER-001, offset
by $3.0 million of collaboration funding; $1.3 million related to
ACER-801; $1.2 million related to EDSIVO™; $0.5 million related to
other development activities.
General and Administrative Expenses. General
and administrative expenses were $3.9 million, net of collaboration
funding of $2.4 million for the three months ended March 31, 2022,
compared to $3.5 million for the three months ended March 31, 2021.
This increase of $0.4 million was primarily due to increases in
precommercial expenses, audit and consulting fees, and
employee-related expenses including a one-time bonus accrual,
partially offset by the recognition of $2.4 million of the
collaboration funding from the Collaboration Agreement with
Relief.
Net Loss. Net loss for the three months ended
March 31, 2022 was $9.2 million, or $0.64 net loss per share (basic
and diluted), compared to a net loss of $4.6 million, or $0.33 net
loss per share (basic and diluted), for the three months ended
March 31, 2021.
For additional information, please see Acer’s Quarterly Report
on Form 10-Q filed today with the SEC.
About Acer Therapeutics Inc.
Acer 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); 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 regarding strategy, future operations,
timelines for clinical study enrollment or regulatory actions, or
otherwise, future financial position, future revenues, projected
expenses, regulatory submissions, actions or approvals, cash
position, liquidity, prospects, plans and objectives of management
are forward-looking statements. Examples of such statements
include, but are not limited to, statements relating to the
potential for our investigational product candidates to safely and
effectively treat diseases and to be approved for marketing; our
ability to close upon and obtain the proceeds of any identified
financing arrangements as well as to satisfy the ongoing conditions
and requirements for maintaining the financing facilities and
avoiding default or an accelerated payment requirement; the
commercial or market opportunity of any of our product candidates
in any target indication and any territory; our ability, in
addition to the currently identified financings, to secure the
additional capital necessary to fund our various product candidate
development programs; the adequacy of our capital to support our
future operations and our ability to successfully fund, initiate
and complete clinical trials and regulatory submissions for
ACER-001, ACER-801, EDSIVO™ or our other product candidates; the
ability to protect our intellectual property rights; our strategy
and business focus; and the development, expected timeline and
commercial potential of any of our product candidates. Our pipeline
products 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, risks and uncertainties associated with the ability to
project future cash utilization and reserves needed for contingent
future liabilities and business operations, the availability of
sufficient resources to fund our various product candidate
development programs and to meet our business objectives and
operational requirements, the fact that the results of earlier
studies and trials may not be predictive of future clinical trial
results, the protection and market exclusivity provided by our
intellectual property, risks related to the drug development and
the regulatory approval process, including the timing and
requirements of regulatory actions, and the impact of competitive
products and technological changes. 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 Report 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 |
|
|
March 31, |
|
|
2022 |
|
2021 |
|
|
|
Revenue |
$ |
— |
|
|
$ |
900,000 |
|
Operating expenses: |
|
|
|
|
|
|
Research and development (in
the three months ended March 31, 2022 and 2021, net of
collaboration funding of $2,999,371 and $286,103,
respectively) |
|
3,171,639 |
|
|
|
2,005,905 |
|
General and administrative (in
the three months ended March 31, 2022 and 2021, net of
collaboration funding of $2,372,175 and $0, respectively) |
|
3,875,601 |
|
|
|
3,514,141 |
|
Loss from operations |
|
(7,047,240 |
) |
|
|
(4,620,046 |
) |
|
|
|
|
|
|
|
Other (expense) income,
net: |
|
|
|
|
|
|
Costs of debt issuance |
|
(1,168,065 |
) |
|
|
— |
|
Changes in fair value of debt
instruments |
|
(962,400 |
) |
|
|
— |
|
Interest and other (expense)
income, net |
|
(2,838 |
) |
|
|
(40,163 |
) |
Foreign currency transaction
gain |
|
1,539 |
|
|
|
48,904 |
|
Total other (expense) income,
net |
|
(2,131,764 |
) |
|
|
8,741 |
|
|
|
|
|
|
|
|
Net loss |
$ |
(9,179,004 |
) |
|
$ |
(4,611,305 |
) |
|
|
|
|
|
|
|
Net loss per share - basic and
diluted |
$ |
(0.64 |
) |
|
$ |
(0.33 |
) |
|
|
|
|
|
|
|
Weighted average common shares
outstanding - basic and diluted |
|
14,310,244 |
|
|
|
14,139,916 |
|
SELECTED BALANCE SHEET DATA
(Unaudited):
|
|
March 31, |
|
|
December 31, |
|
|
|
2022 |
|
|
2021 |
|
|
|
|
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
20,800,384 |
|
|
$ |
12,710,762 |
|
|
|
|
|
|
|
|
|
|
Collaboration receivable |
|
$ |
— |
|
|
$ |
5,000,000 |
|
|
|
|
|
|
|
|
|
|
Prepaid expenses |
|
$ |
1,525,016 |
|
|
$ |
1,094,229 |
|
|
|
|
|
|
|
|
|
|
Other current assets |
|
$ |
1,028,427 |
|
|
$ |
9,283,625 |
|
|
|
|
|
|
|
|
|
|
Property and equipment, net |
|
$ |
122,436 |
|
|
$ |
114,112 |
|
|
|
|
|
|
|
|
|
|
Total assets |
|
$ |
31,469,491 |
|
|
$ |
36,256,951 |
|
|
|
|
|
|
|
|
|
|
Deferred collaboration
funding |
|
$ |
19,115,501 |
|
|
$ |
24,487,047 |
|
|
|
|
|
|
|
|
|
|
Bridge loan payable, at fair
value |
|
$ |
6,172,969 |
|
|
$ |
— |
|
|
|
|
|
|
|
|
|
|
Convertible note payable, at fair
value |
|
$ |
6,962,400 |
|
|
$ |
— |
|
|
|
|
|
|
|
|
|
|
Total liabilities |
|
$ |
41,570,972 |
|
|
$ |
37,980,556 |
|
|
|
|
|
|
|
|
|
|
Total stockholders’ deficit |
|
$ |
(10,101,481 |
) |
|
$ |
(1,723,605 |
) |
Corporate Contact:Jim DeNikeAcer Therapeutics
Inc.+1-844-902-6100jdenike@acertx.com
Investor Relations Contact:Nick
ColangeloGilmartin Group+1-339-225-1047nick@gilmartinIR.com
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