ACER Acer Therapeutics Inc.

2.59
+0.05  (+2%)
Previous Close 2.54
Open 2.57
52 Week Low 1.08
52 Week High 7.25
Market Cap $30,853,973
Shares 11,912,731
Float 7,340,565
Enterprise Value $24,973,112
Volume 264,975
Av. Daily Volume 516,312
Stock charts supplied by TradingView

Upcoming Catalysts

Drug Stage Catalyst Date
ACER-001
Urea cycle disorder (UCD)
NDA Filing
NDA Filing
Premium membership is required to view catalyst dates, analyst ratings, earnings dates and cash burn data. Click here to unlock and sign up to a 14-day FREE TRIAL.

Drug Pipeline

Drug Stage Notes
Osanetant
induced Vasomotor Symptoms (iVMS)
Phase 1/2
Phase 1/2
Phase 1/2 trial to be initiated 1H 2021.
Emetine
COVID-19
Phase 2/3
Phase 2/3
Phase 2/3 trial to be initiated 1H 2021.
EDSIVO
Vascular Ehlers-Danlos Syndrome
CRL
CRL
CRL issued June 25, 2019.
Tovaxin (Tcelna)
Secondary Progressive MS (SPMS)
Phase 2b
Phase 2b
Phase 2b trial did not meet primary endpoint - October 28, 2016.

Latest News

  1. Advanced expanded pipeline of clinical stage product candidates, including emetine for COVID-19 and ACER-001 for Urea Cycle Disorders

    NEWTON, Mass., Aug. 13, 2020 (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 second quarter ended June 30, 2020, and provided an update on the Company's recent corporate developments.

    "The second quarter of 2020 was marked by significant progress in two of our lead programs – emetine and ACER-001," said Chris Schelling, CEO and Founder of Acer. "The need…

    Advanced expanded pipeline of clinical stage product candidates, including emetine for COVID-19 and ACER-001 for Urea Cycle Disorders

    NEWTON, Mass., Aug. 13, 2020 (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 second quarter ended June 30, 2020, and provided an update on the Company's recent corporate developments.

    "The second quarter of 2020 was marked by significant progress in two of our lead programs – emetine and ACER-001," said Chris Schelling, CEO and Founder of Acer. "The need for a therapeutic to treat confirmed COVID-19 patients before they progress and become hospitalized remains a global priority. The team has been working diligently for the past several months to advance emetine from a concept to a pre-IND opportunity and we look forward to the clinical evaluation of emetine, subject to the availability of the required capital. Regarding ACER-001, we reported encouraging new data showing that when ACER-001 is administered in a fasted state it may provide better disease management over currently approved therapies for UCDs that require administration with food. To that end, we submitted a Type C briefing package to the FDA to discuss these results and are expecting feedback later in the third quarter. We continue to target an ACER-001 NDA filing in the first half of 2021, subject to additional capital."

    Second Quarter 2020 and Recent Highlights

    • Emetine
      • Announced a research collaboration agreement with the National Center for Advancing Translational Sciences (NCATS), one of the National Institutes of Health (NIH), to develop emetine hydrochloride as a potential treatment for COVID-19, the disease caused by infection with the SARS-CoV-2 coronavirus
      • Working with federal agencies and private research organizations in an effort to secure non-dilutive funding in support of emetine development
      • Obtained multiple rounds of pre-Investigational New Drug (IND) feedback from the Division of Antivirals (DAV) at the U.S. Food and Drug Administration (FDA)
      • Secured active pharmaceutical ingredient (API) from extract for initiation of the planned Phase 2/3 trial evaluating emetine in high-risk COVID-19 outpatients, assuming the availability of capital. In parallel, we successfully established a fully synthetic development pathway for API exclusively in the U.S.
    • ACER-001
      • Announced results from a food effect study in healthy volunteers showing that administration of ACER-001 in fasted state increased systemic exposure of phenylbutyrate (PBA) levels compared to fed state, which according to in silico modeling may provide improved disease management in patients with urea cycle disorders (UCDs) when compared to currently approved treatments requiring administration with food
      • Submitted a Type C briefing package to the FDA
    • EDSIVO™
      • Continued to assess possible paths forward that could provide the confirmatory evidence needed to demonstrate the substantial evidence of effectiveness needed to support a potential resubmission of the EDSIVO™ NDA
    • Osanetant
      • Advanced API manufacturing and other steps to support IND submission
    • Corporate
      • Hired Stacey Bain, Ph.D. as Vice President, Clinical Operations, who brings 22 years of international clinical operational and drug development experience in pharmaceutical, biotechnology, and clinical research organization settings
      • Ended the second quarter with $5.9 million in cash and cash equivalents. Acer believes its cash position at the end of the second quarter, combined with an additional $4.7 million of net proceeds subsequently received from sales of common stock from the ATM facility and the insider private placement of common stock, will be sufficient to fund its current operations into the first quarter of 2021, excluding support for the planned emetine Phase 2/3 clinical trial

    Upcoming Milestones

    • Emetine
      • Seek to obtain potential non-dilutive funding
      • Targeting in the first half of 2021 an IND submission and clearance, followed by potential initiation of the planned Phase 2/3 trial evaluating emetine in high-risk COVID-19 outpatients, subject to ongoing discussions with the FDA and additional capital
    • ACER-001
      • Anticipate submitting NDA for urea cycle disorders (UCDs) in the first half of 2021, subject to additional capital, and assuming successful completion of nonclinical work and evaluation of long-term product stability data
    • EDSIVO™
      • Expect to request a meeting with FDA by the end of fourth quarter of 2020 to discuss Acer's proposed plan to provide sufficient confirmatory evidence and, if successful, potentially satisfy the substantial evidence of effectiveness needed to support a possible resubmission of the EDSIVO™ NDA. Neither EDSIVO™ NDA resubmission nor approval is assured
    • Osanetant
      • Anticipate submitting osanetant IND in the first quarter of 2021
      • Targeting initiation of Phase 1/2 pharmacokinetic/pharmocodynamic and safety trial in the first half of 2021, subject to additional capital, that will evaluate osanetant in patients with medically and/or surgically induced vasomotor symptoms (iVMS)

    Financial Results for the Second Quarter 2020

    Cash position. Cash and cash equivalents were $5.9 million as of June 30, 2020, compared to $12.1 million as of December 31, 2019. Acer believes its cash position at the end of the second quarter, combined with an additional $4.7 million of net proceeds subsequently received from sales of common stock under the ATM facility and the insider private placement of common stock, will be sufficient to fund its current operations into the first quarter of 2021, excluding support for the planned emetine Phase 2/3 clinical trial.

    Research and Development Expenses. Research and development expenses were $2.8 million for the three months ended June 30, 2020, compared to $4.2 million for the three months ended June 30, 2019. This decrease of $1.4 million was primarily due to decreases in employee-related expenses and clinical and regulatory consulting as a direct result of the Complete Response Letter for EDSIVO™ received from the FDA in June 2019, partially offset by increases in contract research and contract manufacturing expenses. Research and development expenses for the three months ended June 30, 2020 were primarily comprised of $1.0 million related to emetine, $0.9 million related to ACER-001, and $0.8 million related to osanetant.

    General and Administrative Expenses. General and administrative expenses were $3.0 million for the three months ended June 30, 2020, compared to $6.9 million for the three months ended June 30, 2019. This decrease of $3.9 million was primarily due to decreases in precommercial and employee-related expenses as a direct result of the Complete Response Letter for EDSIVO™ received from the FDA in June 2019, partially offset by costs related to implementing Acer's equity line purchase agreement with Lincoln Park Capital Fund, LLC.

    Net Loss. Net loss for the three months ended June 30, 2020 was $5.8 million, or $0.56 net loss per share (basic and diluted), compared to a net loss of $11.0 million, or $1.09 net loss per share (basic and diluted), for the three months ended June 30, 2019.

    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 clinical-stage candidates: emetine hydrochloride for the treatment of patients with COVID-19; ACER-001 (a taste-masked, immediate release formulation of 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; and osanetant for the treatment of induced Vasomotor Symptoms (iVMS). 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 the emetine program; the adequacy of our capital to support our future operations and our ability to successfully 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 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

     

    Jim DeNike

    Acer Therapeutics Inc.

    Ph: 844-902-6100

     

     
     
    ACER THERAPEUTICS INC.

    CONSOLIDATED STATEMENTS OF OPERATIONS

    (Unaudited)
      
     Three Months Ended
     June 30,
     2020  2019 
            
            
    Operating expenses:       
    Research and development$2,816,749  $4,246,244 
    General and administrative 2,953,137   6,919,476 
    Loss from operations (5,769,886)  (11,165,720)
            
    Other (expense) income, net:       
    Interest and other (expense) income, net (2,317   143,884 
    Foreign currency transaction gain (loss) 1,402   (19,671)
            
    Total other (expense) income, net (915   124,213 
            
    Net loss$(5,770,801) $(11,041,507)
            
    Net loss per share - basic and diluted$(0.56) $(1.09)
            
    Weighted average common shares outstanding - basic and diluted 10,365,767   10,090,883 
            



    SELECTED BALANCE SHEET DATA (Unaudited): 
          
      June 30,  December 31,
      2020  2019
            
    Cash and cash equivalents $5,880,861  $12,077,640
            
    Prepaid expenses and other current assets $475,005  $807,356
            
    Property and equipment, net $158,555  $193,974
            
    Total assets $14,791,229  $21,465,511
            
    Total liabilities $3,572,858  $3,095,195
            
    Total stockholders' equity $11,218,371  $18,370,316

    Primary Logo

    View Full Article Hide Full Article
  2. Model suggests ACER-001, Acer's taste-masked, immediate release formulation of sodium phenylbutyrate, may offer improved disease management in patients with Urea Cycle Disorders compared to current treatments

    Anticipate submitting ACER-001 NDA in H1 2021 assuming successful completion of additional nonclinical work and long-term stability data, and subject to additional capital

    NEWTON, Mass., July 08, 2020 (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 data from a food effect study in healthy volunteers showing that administration…

    Model suggests ACER-001, Acer's taste-masked, immediate release formulation of sodium phenylbutyrate, may offer improved disease management in patients with Urea Cycle Disorders compared to current treatments

    Anticipate submitting ACER-001 NDA in H1 2021 assuming successful completion of additional nonclinical work and long-term stability data, and subject to additional capital

    NEWTON, Mass., July 08, 2020 (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 data from a food effect study in healthy volunteers showing that administration of ACER-001 in a fasted state increased systemic exposure of phenylbutyrate (PBA), phenylacetate (PAA) and phenylacetylglutamine (PAGN) levels compared to fed state, and therefore based on modeling data may improve disease management in patients with urea cycle disorders (UCDs) when compared to currently approved treatments requiring administration with food.

    Results from Part B of the ACER-001 bioequivalence (BE) trial in healthy volunteers (n=36) announced in February 2020 showed that ACER-001 was bioequivalent to BUPHENYL® (sodium phenylbutyrate) and were within the parameters recommended by the FDA's Guidance for Industry, "Statistical Approaches to Establishing Bioequivalence." The BE trial included a food effect study, which evaluated the pharmacokinetics (PK) of sodium phenylbutyrate (NaPBA) showing that administration of ACER-001 in a fasted state achieved more than two times the maximum concentration (Cmax) of PBA compared to administration of the same dose of ACER-001 in a fed state. These results are consistent with previously published data by Nakano, et al1 that evaluated PK of NaPBA in patients with progressive familial intrahepatic cholestasis, also demonstrating that administration of NaPBA in a fasted state significantly increased PBA peak plasma concentration compared to administration of NaPBA in a fed state.

    Currently approved therapies for UCDs, including BUPHENYL®2 and RAVICTI®3 (glycerol phenylbutyrate), are required to be administered with food. BUPHENYL® is required to be administered in a fed state due to its aversive odor and taste, with side effects including nausea, vomiting and headaches, which often lead to discontinuation of treatment.4 Additionally, prescribing information states that BUPHENYL® food effect is unknown. RAVICTI® PK and pharmacodynamic (PD) properties were determined to be indistinguishable in fed or fasted states.5 ACER-001 is uniquely formulated with its multi-particulate, taste-masked coating to allow for administration in a fasted state, while still allowing for rapid systemic release.

    Based on the results from the food effect study within the ACER-001 BE trial, Acer commissioned Rosa & Co. LLC to create a PhysioPD® PK model to evaluate the potential food effect on exposure, tolerability and efficacy of ACER-001 in UCDs patients. Results from this in silico model suggest that administration of ACER-001 in a fasted state required approximately 30% less PBA to achieve comparable therapeutic benefit in a fed state. In addition, the model predicted that administration of ACER-001 in a fasted state compared to administration of BUPHENYL® or RAVICTI® (same amounts of PBA) in their required fed states is expected to result in higher peak blood PBA, PAA and PAGN concentrations, predicting a 43% increase in urinary PAGN levels (a negative correlation between blood ammonia area under the curve and 24-hour urinary PAGN amount has been demonstrated6).

    "For nearly a quarter century, phenylbutyrate has been prescribed to UCD patients with food while its effect on phenylbutyrate absorption was never determined. The results of the ACER-001 food effect study, published literature and in silico modeling suggest that ACER-001 administered in a fasted state, and likely just 10 minutes prior to meals, could offer UCD patients a safe and better disease management option compared to currently approved products that are required to be taken with food," said Chris Schelling, CEO and Founder of Acer. "We formulated ACER-001 to specifically improve palatability and tolerability, and we expect that this formulation should allow ACER-001 to be successfully administered without food. We look forward to discussing these findings with the FDA later in the third quarter." Schelling continued "Interestingly, the increased exposure seen under fasted conditions may have benefit in other patient populations we intend to study, such as Maple Syrup Urine Disease (MSUD), where the Cmax of phenylbutyrate is the active moiety."

    About 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.7,8

    The 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 (GPBA) and sodium phenylbutyrate (NaPBA), respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications can be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons given for non-compliance include the unpleasant taste associated with available medications, the frequency with which medication must be taken, the number of pills, and the high cost of the medication.9

    About ACER-001

    ACER-001 is a taste-masked, immediate-release proprietary formulation of sodium phenylbutyrate developed by Acer using a microencapsulation process. ACER-001 is being developed for the treatment of various inborn errors of metabolism, including UCDs and Maple Syrup Urine Disease (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, allowing taste to be neutralized while still allowing for rapid systemic release. This taste-masked formulation may result in better patient tolerability allowing for administration in a fasted state, and likely prior to a meal. 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 will receive FDA approval or become commercially available for the uses being investigated.

    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 clinical-stage candidates: emetine hydrochloride for the treatment of patients with COVID-19; EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a taste-masked, immediate release formulation of sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated. 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.

    References

    1. Nakano S, et al. Effect of food on the pharmacokinetics and therapeutic efficacy of 4-phenylbutyrate in progressive familial intrahepatic cholestasis. Sci Rep 9, 17075 (2019).
    2. https://www.hzndocs.com/BUPHENYL-Prescribing-Information.pdf
    3. https://www.hzndocs.com/RAVICTI-Prescribing-Information.PDF
    4. Pena-Qintana L, et al. Profile of sodium phenylbutyrate granules for the treatment of urea-cycle disorders: patient perspectives. Patient Preference and Adherence Volume 11:1489-1496, September 2017.
    5. United States Patent number US8642012B2.
    6. Lee et al. Phase 2 Comparison of A Novel Ammonia Scavenging Agent With Sodium Phenylbutyrate In Patients With Urea Cycle Disorders: Safety, Pharmacokinetics And Ammonia Control. Mol Genet Metab. 2010 July; 100(3): 221–228.
    7. Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University of Washington, Seattle; 1993.
    8. Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
    9. 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.

    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 the ACER-001 program; the adequacy of our capital to support our future operations and our ability to successfully 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 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

    Jim DeNike

    Acer Therapeutics Inc.

    Ph: 844-902-6100

    Primary Logo

    View Full Article Hide Full Article
  3. The Law Offices of Frank R. Cruz is investigating potential claims against the board of directors of Acer Therapeutics Inc. ("Acer" or the "Company") (NASDAQ:ACER) concerning whether the board breached its fiduciary duties to shareholders.

    If you are a shareholder, click here to participate.

    On June 25, 2019, the Company disclosed receipt of a Complete Response Letter ("CRL") from the U.S. Food and Drug Administration rejecting its new drug application for EDSIVO, a medication for the treatment of Ehlers-Danlos syndrome. According to the CRL, "an adequate and well-controlled trial" was required to determine whether EDSIVO "reduces the risk of clinical events" in patients with vascular Ehlers-Danlos syndrome.

    On this news, the Company's…

    The Law Offices of Frank R. Cruz is investigating potential claims against the board of directors of Acer Therapeutics Inc. ("Acer" or the "Company") (NASDAQ:ACER) concerning whether the board breached its fiduciary duties to shareholders.

    If you are a shareholder, click here to participate.

    On June 25, 2019, the Company disclosed receipt of a Complete Response Letter ("CRL") from the U.S. Food and Drug Administration rejecting its new drug application for EDSIVO, a medication for the treatment of Ehlers-Danlos syndrome. According to the CRL, "an adequate and well-controlled trial" was required to determine whether EDSIVO "reduces the risk of clinical events" in patients with vascular Ehlers-Danlos syndrome.

    On this news, the Company's share price fell $15.16, or nearly 79%, to close at $4.12 on June 25, 2019, thereby injuring investors.

    Our investigation concerns whether the Company's board of directors breached its fiduciary duties to shareholders and/or grossly mismanaged the Company in connection with the above alleged misconduct.

    Follow us for updates on Twitter: twitter.com/FRC_LAW.

    If you purchased Acer shares and wish to discuss this matter with us, or have any questions concerning your rights and interests with regards to this matter, please contact Frank R. Cruz, of The Law Offices of Frank R. Cruz, 1999 Avenue of the Stars, Suite 1100, Los Angeles, California 90067 at 310-914-5007, by email to , or visit our website at www.frankcruzlaw.com. If you inquire by email please include your mailing address, telephone number and number of shares purchased.

    This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and ethical rules.

    View Full Article Hide Full Article
  4. NEWTON, Mass., May 14, 2020 (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, 2020 and provided an update on the Company's recent corporate developments.

    "Over the last several months, we made significant progress in the expansion and further diversification of our product pipeline with the recent addition of emetine, a broad-acting and potent antiviral for the treatment of COVID-19 patients," said Chris Schelling, CEO and Founder of Acer. "We are very pleased to be collaborating…

    NEWTON, Mass., May 14, 2020 (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, 2020 and provided an update on the Company's recent corporate developments.

    "Over the last several months, we made significant progress in the expansion and further diversification of our product pipeline with the recent addition of emetine, a broad-acting and potent antiviral for the treatment of COVID-19 patients," said Chris Schelling, CEO and Founder of Acer. "We are very pleased to be collaborating with NCATS on the development of emetine and look forward to advancing this important program. We continued to advance our ACER-001 program for UCDs following the successful completion of our pivotal bioequivalence trial and are targeting NDA submission in early 2021, subject to additional capital. In addition, we are preparing to submit an IND for osanetant by the end of the year, and we intend to initiate a Phase 1/2 trial in patients with induced vasomotor symptoms in the first quarter of next year, subject to additional capital. To that end, we entered into a stock purchase agreement with Lincoln Park Capital."

    First Quarter 2020 and Recent Highlights

    • Emetine
      °  Announced a research collaboration agreement with the National Center for Advancing Translational Sciences (NCATS), one of the National Institutes of Health (NIH), to develop emetine hydrochloride as a potential treatment for COVID-19, the disease caused by infection with the SARS-CoV-2 coronavirus
      °  In discussion with the Division of Antivirals (DAV) at FDA after receiving its written responses to the Company's pre-Investigational New Drug (pre-IND) package
      °  Presented at the Biomedical Advanced Research and Development Authority (BARDA) CoronaWatch meeting on May 7, 2020. BARDA CoronaWatch is a funding program providing government support for selected coronavirus projects
    • ACER-001
      °  Initiated nonclinical work and evaluation of long-term product stability following the successful completion of the pivotal trial showing that ACER-001 is bioequivalent to BUPHENYL® (sodium phenylbutyrate)
    • EDSIVO™
      °  Received appeal response from the FDA's Office of New Drugs (OND) stating it denied Acer's appeal of the Complete Response Letter (CRL) in relation to the New Drug Application (NDA) for EDSIVO™. Acer is assessing possible paths forward, as described in the OND's response, that could provide the substantial evidence of effectiveness needed to support a potential resubmission of the EDSIVO™ NDA
    • Ended the first quarter with $7.0 million in cash and cash equivalents. Acer believes its cash position will be sufficient to fund its current operations into the fourth quarter of 2020, excluding support for a planned emetine clinical trial and for EDSIVO™ development and precommercial activities
    • Announced a common stock purchase agreement to potentially sell up to $15 million worth of shares to Lincoln Park Capital Fund, LLC over a 36-month period, following the filing and effectiveness of a registration statement and subject to various limitations including those under the Nasdaq listing rules

    Upcoming Milestones

    • Emetine
      °  Pursuing several potential financing options, including federally-funded research and grants, to support emetine development
      °  Working toward an IND submission in mid-2020
      °  Targeting initiation in the third quarter of 2020, subject to additional capital, an adaptive design Phase 2/3 randomized, blinded, placebo-controlled multi-center trial in high-risk, symptomatic adult COVID-19 patients not requiring hospitalization
    • ACER-001
      °  Anticipate submitting NDA for urea cycle disorders (UCDs) in the first quarter of 2021, subject to additional capital, and assuming successful completion of nonclinical work and evaluation of long-term product stability data
    • Osanetant
      °  Anticipate submitting osanetant IND in the fourth quarter of 2020
      °  Targeting initiation of Phase 1/2 pharmacokinetic/pharmocodynamic and safety trial in the first quarter of 2021, subject to additional capital, evaluating osanetant in patients with medically and/or surgically induced vasomotor symptoms (iVMS) in which Hormone Replacement Therapy (HRT) is contraindicated
    • EDSIVO™
      °  Continuing to evaluate possible next steps with the goal of resubmission of the EDSIVO™ NDA. Neither resubmission nor EDSIVO™ approval is assured

    Financial Results for the First Quarter 2020

    Cash position. Cash and cash equivalents were $7.0 million as of March 31, 2020, compared to $12.1 million as of December 31, 2019. Acer believes its cash position will be sufficient to fund its current operations into the fourth quarter of 2020, excluding support for a planned emetine clinical trial and for EDSIVO™ development and precommercial activities.

    Research and Development Expenses. Research and development expenses were $2.3 million for the three months ended March 31, 2020, compared to $3.9 million for the three months ended March 31, 2019. This decrease of approximately $1.6 million was primarily due to decreases in clinical and regulatory consulting, contract manufacturing, and employee-related expenses as a direct result of the Complete Response Letter for EDSIVO™ received from the FDA in June 2019, partially offset by an increase in contract research expenses. Research and development expenses for the three months ended March 31, 2020 were primarily comprised of approximately $0.5 million related to EDSIVO™ and approximately $1.4 million related to ACER-001.

    General and Administrative Expenses. General and administrative expenses were $2.6 million for the three months ended March 31, 2020, compared to $4.2 million for the three months ended March 31, 2019. This decrease of $1.6 million was primarily due to decreases in precommercial and employee-related expenses as a direct result of the Complete Response Letter for EDSIVO™ received from the FDA in June 2019.

    Net Loss. Net loss for the three months ended March 31, 2020 was $4.9 million, or $0.49 net loss per share (basic and diluted), compared to a net loss of $8.0 million, or $0.79 net loss per share (basic and diluted), for the three months ended March 31, 2019.

    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 clinical-stage candidates: emetine hydrochloride for the treatment of patients with COVID-19; EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a taste-masked, immediate release formulation of sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated. 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 the emetine program; the adequacy of our capital to support our future operations and our ability to successfully 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 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

    Jim DeNike
    Acer Therapeutics Inc.
    Ph: 844-902-6100


    ACER THERAPEUTICS INC.

    CONSOLIDATED STATEMENTS OF OPERATIONS

    (Unaudited)

      Three Months Ended
      March 31,
      2020   2019 
       
       
                 
    Operating expenses:            
    Research and development $ 2,322,905     $ 3,946,220  
    General and administrative   2,648,551       4,230,698  
    Loss from operations   (4,971,456 )     (8,176,918 )
                 
    Other income, net:            
    Interest income   22,742       185,143  
    Foreign currency transaction (loss) gain   (2,993 )     23,070  
                 
    Total other income, net   22,749       208,213  
                 
    Net loss $ (4,948,707 )   $ (7,968,705 )
                 
    Net loss per share - basic and diluted $ (0.49 )   $ (0.79 )
                 
    Weighted average common shares outstanding - basic and diluted   10,097,107       10,087,363  
                   

    SELECTED BALANCE SHEET DATA (Unaudited): 

      March 31,   December 31,
      2020   2019
               
    Cash and cash equivalents $ 7,007,260   $ 12,077,640
               
    Prepaid expenses and other current assets $ 624,520   $ 807,356
               
    Property and equipment, net $ 176,265   $ 193,974
               
    Total assets $ 16,140,378   $ 21,465,511
               
    Total liabilities $ 2,051,430   $ 3,095,195
               
    Total stockholders' equity $ 14,088,948   $ 18,370,316

    Primary Logo

    View Full Article Hide Full Article
  5. Targeting Q3 2020 initiation of an adaptive Phase 2/3 trial evaluating emetine in high-risk COVID-19 outpatients, following IND submission and clearance

    Acer to host conference call and webcast on Monday, May 11 at 5:30 pm Eastern Time

    NEWTON, Mass., May 11, 2020 (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 it has entered into a research collaboration agreement with the National Center for Advancing Translational Sciences (NCATS), one of the National Institutes of Health (NIH), to develop emetine hydrochloride as a potential treatment…

    Targeting Q3 2020 initiation of an adaptive Phase 2/3 trial evaluating emetine in high-risk COVID-19 outpatients, following IND submission and clearance

    Acer to host conference call and webcast on Monday, May 11 at 5:30 pm Eastern Time

    NEWTON, Mass., May 11, 2020 (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 it has entered into a research collaboration agreement with the National Center for Advancing Translational Sciences (NCATS), one of the National Institutes of Health (NIH), to develop emetine hydrochloride as a potential treatment for patients with COVID-19, the disease caused by infection with the SARS-CoV-2 virus. Under the terms of the agreement, Acer and NCATS will collaborate to accelerate the clinical development of emetine, a broad-acting and potent antiviral according to various preclinical and clinical studies.

    Acer is in ongoing discussion with the Division of Antivirals (DAV) at the FDA after receiving its initial written responses to the Company's pre-Investigational New Drug (pre-IND) package. Acer is working toward an IND submission in mid-2020 and targeting clinical trial initiation in the third quarter of 2020, subject to additional capital. The Company has proposed an adaptive design Phase 2/3 randomized, blinded, placebo-controlled multi-center trial to evaluate the safety and antiviral activity of emetine in high-risk, symptomatic adult patients with confirmed COVID-19 infection not requiring hospitalization. The trial objectives as planned are to determine the safety and efficacy of emetine via clinical status at a specific timepoint in addition to disease resolution.

    Acer is concurrently pursuing several financing options, including federally-funded research and grants, to support emetine development. For example, the Biomedical Advanced Research and Development Authority (BARDA) invited the Company to present the emetine development program at the BARDA CoronaWatch meeting on May 7, 2020. BARDA CoronaWatch is a funding program providing government support for selected coronavirus projects. While Acer plans to advance emetine through IND submission, initiation of the clinical trial of emetine is contingent on the timely availability of additional capital to fund this program.

    Emetine will be delivered as a sterile subcutaneous injection. Acer will oversee the contract synthesis and manufacturing of emetine for clinical development and potential commercialization.

    "We are very pleased to be selected by NCATS and look forward to collaborating on the development of emetine, a broad-acting and potent antiviral identified by NCATS as their best preclinical opportunity for further clinical development for the treatment of COVID-19," said Chris Schelling, CEO and Founder of Acer. "With a collaboration agreement in place, ongoing discussions with the FDA toward IND submission, and several potentially non-dilutive funding sources being pursued, we believe we are well-positioned to advance the clinical development of emetine."

    Conference Call and Webcast Details
    Interested parties can access the live call and webcast on Monday, May 11, 2020, at 5:30 pm Eastern Time (2:30 pm Pacific Time) from the Investors section of Acer's website or directly at
    http://public.viavid.com/index.php?id=139741. Participants can also access the call by dialing 800-458-4148 (US Toll Free) or 1-720-543-0206 (International Toll Number) and providing the Conference ID 8855853. A replay of the call will also be available under the Investors section of Acer's website.

    About Emetine Hydrochloride
    Acer and NCATS are working together to develop emetine for the treatment of patients with COVID-19, the disease  caused by the SARS-CoV-2 virus. Emetine is an active pharmaceutical ingredient of syrup of ipecac, given orally to induce emesis, and has also been formulated as an injectable to treat thousands of individuals with amebiasis. Several independent in vitro studies have demonstrated nanomolar potency against both DNA and RNA-replicating viruses, including Zika virus, Ebola virus1, Rabies Lyssavirus, human cytomegalovirus, human immunodeficiency virus 1, influenza A virus, Rift Valley fever virus, echovirus 1, human metapneumovirus, and herpes simplex virus type 22. Clinically, emetine has been used to treat approximately 700 patients (including pediatrics) with viral hepatitis3 and varicella-zoster virus4. Additionally, emetine is a potent inhibitor of multiple genetically-distinct coronaviruses and demonstrated in vitro the strongest anti-coronavirus activity in one study that screened and identified approved compounds with broad-spectrum efficacy against the replication of four coronaviruses5 and specifically against SARS-CoV-2.6

    Acer intends to initially seek FDA approval to market emetine in the U.S. using a regulatory pathway established under section 505(b)(2) of the Federal Food, Drug and Cosmetic Act that allows applicants to rely at least in part on third party data for approval. The Company intends to rely in part on the existing preclinical and clinical safety data for emetine, while supplementing with the COVID-19 safety and efficacy data to be generated in the Phase 2/3 trial as well as chemistry, manufacturing and controls information. If the Phase 2/3 trial is completed successfully, following IND submission and clearance, Acer anticipates submitting to the FDA the 505(b)(2) NDA for emetine for the treatment of COVID-19. The potential initiation of the Phase 2/3 trial, its conduct and completion and NDA submission are subject to the Company's ability to generate sufficient capital resources to fund this program. Emetine is an investigational drug for COVID-19 and is not currently FDA approved for any indication.

    About the National Center for Advancing Translational Sciences
    The National Center for Advancing Translational Sciences (NCATS) — one of 27 Institutes and Centers at the National Institutes of Health (NIH) — was established to transform the translational process so that new treatments and cures for disease can be delivered to patients faster. Its focus is to advance the science of translation, which is the process of turning observations into interventions to improve health. NCATS collaborates with researchers, the public and other stakeholder groups to design new approaches and technologies that ultimately will deliver more treatments to more people more quickly.

    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 clinical-stage candidates: emetine hydrochloride for the treatment of patients with COVID-19; EDSIVO™ (celiprolol), for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a taste-masked, immediate release formulation of sodium phenylbutyrate), for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant, for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated. 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.

    References

    1. Yang S, et al. Emetine inhibits Zika and Ebola virus infections through two molecular mechanisms: inhibiting viral replication and decreasing viral entry. Cell Discov (2018) 4:31. doi:10.1038/s41421-018-0034-1
    2. Andersen, P.I., et al. Novel Antiviral Activities of Obatoclax, Emetine, Niclosamide, Brequinar, and Homoharringtonine. Viruses 2019, 11, 964
    3. Del Puerto  et al. Pren. méd. argent., 55: 818, 1968
    4. Annamalai et al. Emetine Hydrochloride in the Treatment of Herpes Zoster. 1968
    5. Shen L, et al. High-Throughput Screening and Identification of Potent Broad-Spectrum Inhibitors of Coronaviruses. J Virol. 2019 May 29;93(12)
    6. Choy et al. Remdesivir, lopinavir, emetine, and homoharringtonine inhibit SARS-CoV-2 replication in vitro. Antiviral Res. 2020 Jun; 178: 104786

    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 the emetine program; the adequacy of our capital to support our future operations and our ability to successfully 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 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

    Jim DeNike
    Acer Therapeutics Inc.
    Ph: 844-902-6100

    Primary Logo

    View Full Article Hide Full Article
View All Acer Therapeutics Inc. News