ACER Acer Therapeutics Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
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ACER-001
Urea cycle disorder (UCD)
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NDA Filing
NDA Filing
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Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
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Osanetant (ACER-801)
induced Vasomotor Symptoms (iVMS)
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Phase 1/2
Phase 1/2
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Phase 1/2 trial to be initiated 4Q 2021.
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Emetine
COVID-19
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Phase 2/3
Phase 2/3
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Phase 2/3 trial to be initiated 1H 2021.
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EDSIVO
Vascular Ehlers-Danlos Syndrome
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CRL
CRL
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CRL issued June 25, 2019.
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Tovaxin (Tcelna)
Secondary Progressive MS (SPMS)
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Phase 2b
Phase 2b
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Phase 2b trial did not meet primary endpoint - October 28, 2016.
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Latest News
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NEWTON, Mass., May 17, 2021 (GLOBE NEWSWIRE) -- 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, 2021 and provided an update on the Company's recent corporate developments.
"We made significant progress in advancing our pipeline programs and strengthening our balance sheet in Q1 2021," said Chris Schelling, CEO and Founder of Acer. "Regarding ACER-001, we executed the Collaboration and License Agreement with Relief Therapeutics; we conducted a pre-NDA meeting with the FDA and are awaiting FDA meeting minutes; and we remain on track to submit an NDA in mid-2021 for ACER-001 as a treatment for UCDs, provided that no additional data are requested by the FDA and we reach agreement on the Pediatric Study Plan (PSP). In addition, we conducted a Type B meeting with the FDA to discuss our proposed path forward for EDSIVO™, and our dialogue is ongoing. We continue to prepare and target an IND submission for ACER-801 (osanetant) in late Q3 2021. Finally, we continue to seek non-dilutive capital to advance development of ACER-2820 (emetine)."
Q1 2021 and Recent Highlights
- ACER-001
- Announced in February 2021 topline results from Acer's bioequivalence trial in which ACER-001 showed similar relative bioavailability compared to BUPHENYL® (sodium phenylbutyrate) under fed conditions
- Announced in March 2021 that Acer and Relief Therapeutics Holding AG (Relief) entered into a Collaboration and License Agreement (Collaboration Agreement) for worldwide development and commercialization of ACER-001. Under the terms of the agreement, Relief has paid Acer $20 million, and will pay Acer up to an additional $21 million in development expense reimbursement and milestones plus royalties in exchange for net profit share and territory rights
- Conducted a Type B pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) – awaiting FDA meeting minutes
- EDSIVO™ (celiprolol)
- Conducted a Type B meeting with the FDA to discuss Acer's proposed plan to collect additional data with the goal of providing further evidence in support of celiprolol's potential benefit in treating COL3A1-positive vEDS patients – awaiting FDA meeting minutes
- ACER-801 (osanetant)
- Continued to advance development activities in support of an Investigational New Drug Application (IND) submission
- Corporate
- Hired Bill DeVincenzi in March 2021 as Vice President, Quality. Mr. DeVincenzi brings to Acer 29 years of experience in quality assurance, supporting the development and commercialization of several small-molecule pharmaceuticals across a wide spectrum of dosage forms
- Ended Q1 2021 with $15.9 million in cash and cash equivalents. Acer believes its cash and cash equivalents available as of March 31, 2021, plus up to $20.0 million of Development Payments per the Collaboration Agreement with Relief, are sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for a planned osanetant clinical trial
Anticipated Milestones
- ACER-001
- Q2 2021: Awaiting minutes from the Type B pre-NDA meeting with the FDA
- Mid-2021: ACER-001 NDA submission for treatment of patients with Urea Cycle Disorders (UCDs) is anticipated in mid-2021, provided that no additional data are requested by the FDA and ongoing development activities are successfully completed (including evaluation of product stability data and reaching agreement on the Pediatric Study Plan (PSP))
- EDSIVO™
- Q2 2021: Awaiting minutes from the Type B meeting with the FDA
- ACER-801 (osanetant)
- Q3 2021: IND submission for osanetant is anticipated in late Q3 2021
- Q4 2021: Clinical trial initiation is expected in late Q4 2021 designed to evaluate osanetant's PK/PD and safety and to identify the optimal dosing strategy for future efficacy studies in patients with induced Vasomotor Symptoms (iVMS), dependent upon successful IND submission and clearance, and subject to additional capital
- ACER-2820 (emetine)
- Ongoing: The Company believes that most of the emetine IND-enabling work is complete. Further advancement of the emetine program for treatment of certain infectious diseases, including COVID-19, is dependent on Acer's ability to raise non-dilutive capital
Q1 2021 Financial Results
Cash position. Cash and cash equivalents were $15.9 million as of March 31, 2021, compared to $5.8 million as of December 31, 2020. Acer believes its cash and cash equivalents available as of March 31, 2021, plus up to $20.0 million of Development Payments per the Collaboration Agreement with Relief, are sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for a planned osanetant clinical trial.
Revenue. Acer recognized revenue of $4.0 million during the three months ended March 31, 2021, related to the license of intellectual property as part of the Collaboration Agreement signed during the quarter.
Research and Development Expenses. Research and development expenses were $2.0 million for the three months ended March 31, 2021, as compared to $2.3 million for the three months ended March 31, 2020. An increase in employee-related expenses, which was driven by bonuses expensed during the quarter, was offset by a decrease in expenses for contract research and contract manufacturing, as well as an offset of $0.3 million associated with the recognition of collaboration funding from the Collaboration Agreement with Relief. Research and development expenses for the three months ended March 31, 2021 were comprised of $1.2 million related to ACER-001 offset by $0.3 million of collaboration funding, $0.5 million related to osanetant, $0.4 million related to emetine, and $0.2 million related to other development activities.
General and Administrative Expenses. General and administrative expenses were $3.5 million for the three months ended March 31, 2021, as compared to $2.6 million for the three months ended March 31, 2020. This increase of $0.9 million was primarily due to an increase in employee-related expenses, driven by bonuses expensed during the quarter, partially offset by a decrease in expenses related to precommercial activities.
Net Loss. Net loss for the three months ended March 31, 2021 was $1.5 million, or $0.11 net loss per share (basic and diluted), compared to a net loss of $4.9 million, or $0.49 net loss per share (basic and diluted), for the three months ended March 31, 2020.
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 programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer's product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for worldwide development and commercialization of ACER-001. For more information, visit www.acertx.com.Forward-Looking Statements
This 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, 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 product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability 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; 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. 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, the substantial costs and diversion of management's attention and resources which could result from pending securities litigation, 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 Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.Investor Contact:
Hans Vitzthum
LifeSci Advisors
Ph: 617-430-7578
[email protected]Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected]ACER THERAPEUTICS INC.
CONSOLIDATED STATEMENTS OF OPERATIONS
(Unaudited)
Three Months Ended March 31, 2021 2020 Revenue $ 3,998,133 $ — Operating expenses: Research and development 2,005,905 2,322,905 General and administrative 3,514,141 2,648,551 Loss from operations (1,521,913 ) (4,971,456 ) Other income (expense), net: Interest and other income (expense), net (40,163 ) 25,742 Foreign currency transaction gain (loss) 48,904 (2,993 ) Total other income (expense), net 8,741 22,749 Net loss $ (1,513,172 ) $ (4,948,707 ) Net loss per share - basic and diluted $ (0.11 ) $ (0.49 ) Weighted average common shares outstanding - basic and diluted 14,139,916 10,097,107 SELECTED BALANCE SHEET DATA (Unaudited):
March 31, December 31, 2021 2020 Cash and cash equivalents $ 15,900,297 $ 5,761,568 Accounts receivable $ 10,000,000 $ — Prepaid expenses and other current assets $ 10,507,161 $ 679,461 Property and equipment, net $ 120,769 $ 130,081 Total assets $ 44,510,898 $ 14,613,688 Deferred collaboration funding $ 20,715,764 $ — Total liabilities $ 34,101,253 $ 6,389,354 Total stockholders' equity $ 10,409,645 $ 8,224,334 
- ACER-001
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NEWTON, Mass., March 29, 2021 (GLOBE NEWSWIRE) -- 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 announced that Acer's management team will present and host one-on-one investor meetings at the upcoming 20th Annual Needham Virtual Healthcare Conference April 12-15, 2021.
Conference: 20th Annual Needham Virtual Healthcare Conference Format: Virtual presentation and one-on-one meetings Presentation: April 15, 2021 Time: 11 a.m. Eastern Time Webcast: https://acertx.com/investor-relations/events-presentations/ 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 programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer's product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the U.S. FDA. On March 19, 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for worldwide development and commercialization of ACER-001. For more information, visit www.acertx.com.Investor Contact:
Hans Vitzthum
LifeSci Advisors
Ph: 617-430-7578
[email protected]Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected] -
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Relief to potentially pay Acer up to $36 million and royalties in exchange for net profit share and territory rights
ACER-001 pre-NDA meeting with U.S. FDA scheduled in Q2 2021
GENEVA and NEWTON, Mass., March 22, 2021 (GLOBE NEWSWIRE) -- RELIEF THERAPEUTICS Holding AG ((SIX: RLF, OTCQB:RLFTF)("Relief"), a biopharmaceutical company with its lead compound RLF-100™ (aviptadil) in advanced clinical development to treat severe COVID-19 patients, and Acer Therapeutics Inc. (NASDAQ:ACER)("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 announced that the companies entered into a Collaboration and License Agreement ("CLA") for worldwide development and commercialization of ACER-001. ACER-001 is a proprietary powder formulation of sodium phenylbutyrate (NaPB) designed to be both taste-masked and immediate release.
Under the terms of the CLA, Acer will receive an approximately $10 million cash payment within 15 business days of CLA execution (originally $14 million, to be offset by repayment of the $4.0 million outstanding balance of the prior loan, plus interest, from Relief to Acer). Relief will also pay Acer up to $20 million in U.S. development and commercial launch costs for the UCDs and MSUD indications. Acer will retain development and commercialization rights in the U.S., Canada, Brazil, Turkey, and Japan. The companies will split net profits from Acer's territories 60%:40% in favor of Relief. In addition, Relief has licensed the rights for the rest of the world, where Acer will receive from Relief a 15% royalty on all revenues received in Relief's territories. Acer may also receive a total of $6 million in development milestone payments following the first European (EU) marketing approvals for UCDs and MSUD.
Jack Weinstein, Chief Financial Officer and Treasurer of Relief, said, "We are excited to continue moving forward with the Acer team to develop and commercialize ACER-001 around the globe to address important unmet needs for patients suffering from these rare diseases. This collaboration is an important step in Relief's plan to build a diversified late-stage pipeline beyond our lead candidate, RLF-100™, which is currently in development for the treatment of respiratory illnesses due to COVID-19 infection. We are pleased to have been able to conclude this agreement, as the advanced stage of development and market opportunity with ACER-001 make this compound a perfect fit for Relief's strategy."
Chris Schelling, Acer's CEO and Founder, said, "Our collaboration with Relief will provide important resources and additional expertise to advance the development of ACER-001 toward our goal of bringing this product candidate to patients suffering from UCDs and MSUD. We look forward to partnering with the team at Relief to advance this program and to potentially provide a much-needed treatment option for patients with these rare and debilitating diseases."
An ACER-001 pre-NDA meeting with the U.S. FDA is scheduled to occur in the second quarter of 2021. Acer expects to receive official meeting minutes approximately 30 days after the meeting.
ACER-001 is an investigational product being studied for the treatment of patients with UCDs and MSUD and has not been approved by the U.S. FDA or any regulatory agency outside the U.S. for any indication. There can be no assurance that if submitted, a New Drug Application or equivalent will be accepted by the U.S. FDA or any other regulatory agency for filing and review or, if filed, that it will be approved.
About Urea Cycle Disorders (UCDs)
UCDs are a group of disorders caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia. Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits. Common symptoms of both acute and chronic hyperammonemia also include seizures and psychiatric symptoms.1,2The current treatment of UCDs consists of dietary management to limit ammonia production in conjunction with medications that provide alternative pathways for the removal of ammonia from the bloodstream. Some patients may also require individual branched-chain amino acid supplementation.
Current medical treatments for UCDs include nitrogen scavengers, RAVICTI® and BUPHENYL®, in which the active pharmaceutical ingredients are glycerol phenylbutyrate (GPB) and sodium phenylbutyrate (NaPB), respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications have been shown to be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons referenced for non-compliance associated with some available medications include unpleasant taste, the frequency with which medication must be taken, the number of pills, and the high cost of the medication.3
About Maple Syrup Urine Disease (MSUD)
MSUD is a rare inherited disorder caused by defects in the mitochondrial branched-chain ketoacid dehydrogenase complex, which results in elevated blood levels of the branched-chain amino acids (BCAA), leucine, valine, and isoleucine, as well as the associated branched-chain ketoacids (BCKA) in a patient's blood. Left untreated, this can result in neurological damage, mental disability, coma or death. There are currently no approved pharmacologic therapies in the U.S. or the European Union for MSUD. Treatment of MSUD consists primarily of a severely restricted diet to limit the intake of BCAA, with aggressive medical interventions when blood-levels of BCAA or BCKA become elevated.About ACER-001
ACER-001 is a proprietary powder formulation of sodium phenylbutyrate (NaPB). The formulation is designed to be both taste-masked and immediate release. ACER-001 is being developed using a microencapsulation process for the treatment of various inborn errors of metabolism, including UCDs and MSUD. ACER-001 microparticles consist of a core center, a layer of active drug, and a taste-masking coating that quickly dissolves in the stomach, to avoid a bitter taste while still allowing for rapid systemic release. If ACER-001 is approved, its taste-masked properties could make it a compelling alternative to existing NaPB-based treatments, as the unpleasant taste associated with NaPB is cited as a major impediment to patient compliance with those treatments.3 Acer has been granted orphan drug designation by the FDA for the MSUD indication. ACER-001 is under clinical investigation and its safety and efficacy have not been established. There is no guarantee that this product candidate will receive U.S. FDA approval or become commercially available for the uses being investigated.About RELIEF THERAPEUTICS Holding AG
Relief focuses primarily on clinical-stage programs based on molecules with a history of clinical testing and use in human patients or a strong scientific rationale. Relief's lead drug candidate RLF-100™ (aviptadil), a synthetic form of Vasoactive Intestinal Peptide (VIP), is in late-stage clinical testing in the U.S. for the treatment of respiratory deficiency due to COVID-19. As part of its pipeline diversification strategy, In March 2021, Relief entered into a Collaboration and License Agreement with Acer Therapeutics for the worldwide development and commercialization of ACER-001. ACER-001 is a taste-masked and immediate release proprietary powder formulation of sodium phenylbutyrate (NaPB) for the treatment of Urea Cycle Disorders and Maple Syrup Urine Disease.RELIEF THERAPEUTICS Holding AG is listed on the SIX Swiss Exchange under the symbol RLF and quoted in the U.S. on OTCQB under the symbol RLFTF. For more information, visit www.relieftherapeutics.com. Follow Relief on LinkedIn.
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 programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer's product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the U.S. FDA. On March 19, 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for worldwide development and commercialization of ACER-001. For more information, visit www.acertx.com.References
- Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University of Washington, Seattle; 1993.
- Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
- Shchelochkov OA, et al. Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives. Mol Genet Metab. 2016;8:43-47.
Relief Forward-Looking Statements
This communication expressly or implicitly contains certain forward-looking statements concerning RELIEF THERAPEUTICS Holding AG and its businesses. The results reported herein may or may not be indicative of the results of future and larger clinical trials for ACER-001 for the treatment of UCDs and MSUD, nor whether the ongoing clinical trials of Relief's lead compound, RLF-100™ (aviptadil) in advanced clinical development to treat respiratory deficiency due to COVID-19, will be successful. Such statements involve certain known and unknown risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of RELIEF THERAPEUTICS Holding AG to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. RELIEF THERAPEUTICS Holding AG is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise.Acer Forward-Looking Statements
This 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, 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 benefits and results of the Collaboration and License Agreement between Acer and Relief with respect to ACER-001; the potential for ACER-001 to target diseases; the adequacy of Acer's capital to support its future operations and its ability to successfully continue its development programs; Acer's ability to secure the additional capital necessary to fund its various product candidate development programs; and the development and commercial potential of any of Acer's product candidates including ACER-001. Acer 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 Acer's ability to benefit from and achieve the results contemplated by the Collaboration and License Agreement with Relief, the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to fund Acer's various product candidate development programs and to meet its 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 Acer's intellectual property, risks related to the drug discovery and the regulatory approval process and the impact of competitive products and technological changes. Acer disclaims 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 Acer makes in its filings with the Securities and Exchange Commission, including its Quarterly Reports on Form 10-Q and its Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.CORPORATE CONTACTS
RELIEF THERAPEUTICS Holding AG:
Jack Weinstein
Chief Financial Officer and Treasurer
[email protected]ACER Therapeutics:
Jim DeNike
Acer Therapeutics Inc.
+1 844-902-6100
[email protected]MEDIA CONTACTS
Relief (Europe):
Anne Hennecke / Brittney Sojeva
MC Services AG
[email protected]
+49 (0) 211-529-252-14INVESTOR RELATIONS CONTACTS
Relief (Europe):
Anne Hennecke / Brittney Sojeva
MC Services AG
[email protected]
+49 (0) 211-529-252-14Acer Therapeutics:
Hans Vitzthum
LifeSci Advisors
+1 617-430-7578
[email protected] -
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NEWTON, Mass., March 01, 2021 (GLOBE NEWSWIRE) -- 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 fourth quarter and full year ended December 31, 2020 and provided an update on the Company's recent corporate developments.
"In spite of the challenges presented by a global pandemic in 2020, we continued to advance our existing programs while expanding our pipeline," said Chris Schelling, CEO and Founder of Acer. "As a result, we anticipate a number of key milestones next quarter, including a proposed ACER-001 pre-NDA meeting with FDA, continuing to work toward a potential ACER-001 collaboration and license agreement with Relief Therapeutics pursuant to the existing option agreement between the companies, and a Type B meeting with the FDA to discuss a possible path forward for EDSIVO™. We are also working toward initiation of a Phase 2 clinical trial of osanetant in BRCA-positive patients with induced vasomotor symptoms in the fourth quarter of 2021, subject to successful IND filing and additional capital, and continuing to seek non-dilutive capital to advance emetine development."
Fourth Quarter 2020 and Recent Highlights
- ACER-001
- Signed an Option Agreement with Relief Therapeutics Holding AG on January 25, 2021 providing Relief with exclusivity until June 30, 2021 for the right to pursue a potential collaboration and license agreement for worldwide development and commercialization for ACER-001. In return, Acer received a $1.0 million upfront nonrefundable payment and a $4.0 million secured loan from Relief
- Announced in February 2021 topline results from Acer's bioequivalence trial in which ACER-001 showed similar relative bioavailability compared to BUPHENYL® (sodium phenylbutyrate) under fed conditions
- EDSIVO™ (celiprolol)
- Announced in December 2020 publication by Swedish investigators of additional long-term data from COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS) patients in the European Journal of Vascular and Endovascular Surgery entitled "Celiprolol Treatment in Patients with Vascular Ehlers-Danlos Syndrome" (https://www.ejves.com/action/showPdf?pii=S1078-5884%2820%2930930-8)
- Submitted a Type B meeting request to the FDA in February 2021 to discuss Acer's proposed plan to collect additional data and provide confirmatory evidence in support of celiprolol's potential benefit in treating COL3A1-positive vEDS patients
- Announced in December 2020 publication by Swedish investigators of additional long-term data from COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS) patients in the European Journal of Vascular and Endovascular Surgery entitled "Celiprolol Treatment in Patients with Vascular Ehlers-Danlos Syndrome" (https://www.ejves.com/action/showPdf?pii=S1078-5884%2820%2930930-8)
- ACER-801 (osanetant)
- Completed active pharmaceutical ingredient (API) manufacturing and advanced other development activities to support an Investigational New Drug Application (IND) submission
- Completed active pharmaceutical ingredient (API) manufacturing and advanced other development activities to support an Investigational New Drug Application (IND) submission
- Corporate
- Promoted Jefferson Davis to Chief Business Officer in February 2021. A veteran life science executive and transaction specialist with over 25 years of experience in business and corporate development, Mr. Davis has led or supported over 40 transactions with an aggregate value of more than $2 billion across all major therapeutic platforms, including antibodies, small molecules, aptamers, gene therapy, vaccines, and proteins
- Ended the fourth quarter with $5.8 million in cash and cash equivalents. Acer believes its cash position as of December 31, 2020, combined with an additional $3.2 million of net proceeds subsequently received from the sales of common stock under its ATM facility and through its equity line purchase agreement with Lincoln Park Capital, along with the $1.0 million nonrefundable payment and $4.0 million secured loan received from Relief Therapeutics following the signing of the ACER-001 Option Agreement, will be sufficient to fund its operations into the third quarter of 2021
- Promoted Jefferson Davis to Chief Business Officer in February 2021. A veteran life science executive and transaction specialist with over 25 years of experience in business and corporate development, Mr. Davis has led or supported over 40 transactions with an aggregate value of more than $2 billion across all major therapeutic platforms, including antibodies, small molecules, aptamers, gene therapy, vaccines, and proteins
Upcoming Milestones
- ACER-001
- Q2 2021: Targeting a pre-New Drug Application (NDA) meeting with the FDA in the second quarter of 2021, assuming successful completion of the ongoing development activities
- Mid-2021: ACER-001 NDA submission for treatment of patients with Urea Cycle Disorders (UCDs) is anticipated in mid-2021, provided that no additional data is requested by the FDA during Acer's pre-NDA meeting and ongoing development activities are successfully completed (including evaluation of long-term product stability data)
- Q2 2021: Targeting a pre-New Drug Application (NDA) meeting with the FDA in the second quarter of 2021, assuming successful completion of the ongoing development activities
- EDSIVO™
- Q2 2021: Obtain FDA feedback from a Type B meeting in the second quarter of 2021 regarding Acer's proposed plan to collect additional data which, if discussions are successful and the collected data is sufficient, could potentially satisfy the substantial evidence of effectiveness needed to support a resubmission of the EDSIVO™ NDA (although neither EDSIVO™ NDA resubmission nor approval is assured)
- ACER-801 (osanetant)
- Q3 2021: IND submission for osanetant is anticipated in the third quarter of 2021
- Q4 2021: Initiation of a Phase 2 clinical trial of osanetant in BRCA-positive patients who have undergone a prophylactic bilateral salpingo-oophorectomy (PBSO) is expected in the fourth quarter of 2021, dependent upon successful IND filing and subject to additional capital
- Q3 2021: IND submission for osanetant is anticipated in the third quarter of 2021
- ACER-2820 (emetine)
- Ongoing: Further advancement of the emetine program for infectious diseases, including COVID-19, is dependent on Acer's ability to raise non-dilutive capital
- Ongoing: The Company believes that most of the emetine IND-enabling work is in progress or complete, and intends to minimize future emetine spending as it continues to work with federal agencies and private research organizations toward the goal of securing non-dilutive funding
- Ongoing: Further advancement of the emetine program for infectious diseases, including COVID-19, is dependent on Acer's ability to raise non-dilutive capital
Financial Results for the Fourth Quarter and Full Year 2020
Cash position. Cash and cash equivalents were $5.8 million as of December 31, 2020, compared to $12.1 million as of December 31, 2019. Acer believes its cash and cash equivalents available as of December 31, 2020, combined with an additional $3.2 million of net proceeds subsequently received from the sales of common stock under its ATM facility and through its equity line purchase agreement with Lincoln Park Capital, along with the $1.0 million nonrefundable payment and $4.0 million secured loan received from Relief Therapeutics following the signing of the ACER-001 Option Agreement, will be sufficient to fund its operations into the third quarter of 2021.
Research and Development Expenses. Research and development expenses were $3.5 million for the three months ended December 31, 2020, compared to $2.8 million for the three months ended December 31, 2019. Research and development expenses for the three months ended December 31, 2020 were comprised of $1.3 million related to ACER-001, $1.5 million related to emetine, $0.6 million related to osanetant, and $0.1 million related to EDSIVO™. Research and development expenses were $11.8 million for the year ended December 31, 2020, compared to $13.9 million for the year ended December 31, 2019. This decrease of approximately $2.1 million was primarily due to decreases in employee-related expense and in spending related to clinical and other consulting services, partially offset by an increase in contract research expenses.
General and Administrative Expenses. General and administrative expenses were $2.7 million for the three months ended December 31, 2020, compared to $2.4 million for the three months ended December 31, 2019. General and administrative expenses were $11.0 million for the year ended December 31, 2020, compared to $16.0 million for the year ended December 31, 2019. This decrease of $5.0 million was primarily due to decreases in employee-related expenses and precommercial activities expenses, partially offset by an increase in legal expenses. This decrease in employee-related expenses resulted from a decrease in headcount as a consequence of the restructuring initiative undertaken after receipt of the Complete Response Letter from the FDA for EDSIVO™ in June 2019.
Net Loss. Net loss for the three months ended December 31, 2020 was $6.2 million, or $0.50 net loss per share (basic and diluted), compared to a net loss of $5.2 million, or $0.51 net loss per share (basic and diluted), for the three months ended December 31, 2019. Net loss for the year ended December 31, 2020 was $22.9 million, or $2.06 loss per share (basic and diluted), compared to a net loss of $29.4 million, or $2.91 loss per share (basic and diluted), for the year ended December 31, 2019.
For additional information, please see Acer's Annual Report on Form 10-K 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 programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer's product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit www.acertx.com.Forward-Looking Statements
This 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, 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 product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability 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; 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. 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, the substantial costs and diversion of management's attention and resources which could result from pending securities litigation, 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 Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.Investor Contact:
Hans Vitzthum
LifeSci Advisors
Ph: 617-430-7578
[email protected]Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected]ACER THERAPEUTICS INC.
CONSOLIDATED STATEMENTS OF OPERATIONS
Three Months Ended Years Ended December 31, December 31, 2020 2019 2020 2019 (unaudited) Operating expenses: Research and development $ 3,481,200 $ 2,829,767 $ 11,847,902 $ 13,851,018 General and administrative 2,691,246 2,362,572 10,954,923 16,046,423 Loss from operations (6,172,446 ) (5,192,339 ) (22,802,825 ) (29,897,441 ) Other (expense) income, net: Interest and other (expense) income, net (4,095 ) 50,919 13,578 471,267 Foreign currency transaction (loss)/gain (61,143 ) (14,864 ) (96,202 ) 8,205 Total other (expense) income, net (65,238 ) 36,055 (82,624 ) 479,472 Net loss $ (6,237,684 ) $ (5,156,284 ) $ (22,885,449 ) $ (29,417,969 ) Net loss per share - basic and diluted $ (0.50 ) $ (0.51 ) $ (2.06 ) $ (2.91 ) Weighted average common shares outstanding - basic and diluted 12,487,688 10,095,176 11,121,039 10,092,179 SELECTED BALANCE SHEET DATA:
December 31, December 31, 2020 2019 Cash and cash equivalents $ 5,761,568 $ 12,077,640 Prepaid expenses and other current assets $ 679,461 $ 807,356 Property and equipment, net $ 130,081 $ 193,974 Total assets $ 14,613,688 $ 21,465,511 Total liabilities $ 6,389,354 $ 3,095,195 Total stockholders' equity $ 8,224,334 $ 18,370,316 - ACER-001
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NEWTON, Mass., March 01, 2021 (GLOBE NEWSWIRE) -- 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 announced that Acer's management team will present and host one-on-one investor meetings at the upcoming H.C. Wainwright Global Life Sciences Virtual Conference and the 33rd Annual Roth Virtual Conference.
Conference: H.C. Wainwright Global Life Sciences Virtual Conference Dates: March 9-10, 2021 Format: One-on-one virtual meetings Conference: 33rd Annual Roth Virtual Conference Format: Panel Presentation and one-on-one virtual meetings Panel: Therapeutics Against SARS-CoV-2 Date: Monday, March 15, 2021 Time: 12 p.m. Eastern Time Webcast: https://acertx.com/investor-relations/events-presentations/ 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 programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer's product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit www.acertx.com.Investor Contact:
Hans Vitzthum
LifeSci Advisors
Ph: 617-430-7578
[email protected]Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected]