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
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 1Q 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 3Q 2020.
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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
|
Phase 2b trial did not meet primary endpoint - October 28, 2016.
|
Latest News
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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 [email protected], 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 source version on businesswire.com: https://www.businesswire.com/news/home/20200624005755/en/
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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
[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, 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 
- Emetine
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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-2.6Acer 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
- 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
- Andersen, P.I., et al. Novel Antiviral Activities of Obatoclax, Emetine, Niclosamide, Brequinar, and Homoharringtonine. Viruses 2019, 11, 964
- Del Puerto et al. Pren. méd. argent., 55: 818, 1968
- Annamalai et al. Emetine Hydrochloride in the Treatment of Herpes Zoster. 1968
- Shen L, et al. High-Throughput Screening and Identification of Potent Broad-Spectrum Inhibitors of Coronaviruses. J Virol. 2019 May 29;93(12)
- 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
[email protected]Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected] -
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NEWTON, Mass., April 30, 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 common stock purchase agreement for up to $15 million with Lincoln Park Capital Fund, LLC (LPC), a Chicago-based institutional investor.
Under the terms of the purchase agreement and following the filing and effectiveness of a registration statement, Acer will have the right at its sole discretion, but not the obligation, to sell to LPC up to $15 million worth of shares over the 36-month term of the agreement, subject to various limitations including those under the Nasdaq listing rules. There are no upper limits to the price per share LPC may pay to purchase the shares, and the purchase price of the shares will be based on the then prevailing market prices at the time of each sale to LPC. Acer controls the timing and amount of any future sales of its stock to LPC. There are no warrants, derivatives, financial or business covenants associated with the agreements, and LPC has agreed not to cause or engage in any direct or indirect short selling or hedging of Acer's common stock. Acer may terminate the purchase agreement at any time, at its discretion, without any cost or penalty.
Acer intends to use any net proceeds from the sale of its common stock to LPC for working capital and general corporate purposes. In consideration for LPC entering into the purchase agreement, Acer issued shares of its common stock to LPC as a commitment fee.
Additional information regarding the purchase agreement with LPC is available in the Current Report on Form 8-K that Acer filed today with the Securities and Exchange Commission.
The offer and sale of the securities in the above transaction have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state securities laws and may not be offered or sold in the United States absent registration under the Securities Act and any applicable state securities laws or an applicable exemption from such registration.
This press release shall not constitute an offer to sell or a solicitation of an offer to buy any shares of common stock, nor shall there be any sale of shares of common stock in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction.
About Lincoln Park Capital Fund, LLC
LPC is a long-only institutional investor headquartered in Chicago, Illinois. LPC's experienced professionals manage a portfolio of investments in public and private entities. These investments are in a wide range of companies and industries emphasizing life sciences and technology. LPC's investments range from multi-year financial commitments to fund growth to special situation financings to long-term strategic capital offering companies' flexibility and consistency. For more information, please visit www.lpcfunds.com.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 three clinical-stage candidates: 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 possible sales of common stock pursuant to the purchase agreement with LPC and our financing needs are forward-looking statements. 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, including, without limitation, uncertainties related to our working capital, our ability to carry on our existing operations and to obtain needed financing. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors. 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 Current Reports on Form 8-K, 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] -
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OND denies Acer's appeal but describes possible paths forward for EDSIVO™
ACER-001 bioequivalence trial complete; New Drug Application submission anticipated early 2021
Targeting osanetant Phase 1/2 trial initiation by end 2020
NEWTON, Mass., March 18, 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 fourth quarter and full year ended December 31, 2019 and provided an update on the Company's recent corporate developments.
"Over the last 12 months, we have made considerable progress in advancing our pipeline of clinical-stage product candidates," said Chris Schelling, CEO and Founder of Acer. "While the Office of New Drugs recently denied our appeal of the EDSIVO™ Complete Response Letter, we are encouraged by its description of possible paths forward. While neither resubmission nor the prospect of approval is assured, we are evaluating our possible next steps with the goal of resubmission of the EDSIVO™ NDA. Concurrently, we continue to advance our ACER-001 program for Urea Cycle Disorders following the successful completion of our pivotal bioequivalence trial and are working toward a New Drug Application submission in early 2021. In addition, we are preparing to initiate an osanetant Phase 1/2 trial in patients with induced vasomotor symptoms by the end of the year, subject to additional capital."
Fourth Quarter 2019 and Recent Highlights
- EDSIVO™
- Today announced the Office of New Drugs (OND) of the U.S. Food and Drug Administration (FDA) denied Acer's appeal of the Complete Response Letter (CRL) in relation to the New Drug Application (NDA) for EDSIVO™. In its Appeal Denied letter, the OND describes possible paths forward for Acer to explore that could provide the substantial evidence of effectiveness needed to support a potential resubmission of the EDSIVO™ NDA for the treatment of patients with vascular Ehlers-Danlos syndrome (vEDS) with a confirmed COL3A1 mutation
- Today announced the Office of New Drugs (OND) of the U.S. Food and Drug Administration (FDA) denied Acer's appeal of the Complete Response Letter (CRL) in relation to the New Drug Application (NDA) for EDSIVO™. In its Appeal Denied letter, the OND describes possible paths forward for Acer to explore that could provide the substantial evidence of effectiveness needed to support a potential resubmission of the EDSIVO™ NDA for the treatment of patients with vascular Ehlers-Danlos syndrome (vEDS) with a confirmed COL3A1 mutation
- ACER-001
- Announced successful completion and final data from Part B of the pivotal trial showing that ACER-001 was bioequivalent to BUPHENYL® (sodium phenylbutyrate)
- Initiated remaining nonclinical work and evaluation of long-term product stability
- Ended the fourth quarter with $12.1 million in cash and cash equivalents. Acer believes its cash position will be sufficient to fund its current operations through the end of 2020, excluding support for EDSIVO™ development and precommercial activities and the planned osanetant clinical trial
Upcoming Milestones
- EDSIVO™
- While neither resubmission nor the prospect of approval is assured, evaluate possible next steps with the goal of resubmission of the EDSIVO™ NDA
- While neither resubmission nor the prospect of approval is assured, evaluate possible next steps with the goal of resubmission of the EDSIVO™ NDA
- ACER-001
- Submit NDA for UCDs in early 2021, subject to additional capital, and assuming successful completion of nonclinical work and evaluation of long-term product stability data
- Submit NDA for UCDs in early 2021, subject to additional capital, and assuming successful completion of nonclinical work and evaluation of long-term product stability data
- Osanetant
- Submit osanetant IND in 2H 2020
- Target initiation of Phase 1/2 pharmacokinetic/pharmocodynamic and safety trial by end of 2020, 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
Financial Results for the Fourth Quarter and Full Year 2019
Cash position. Cash and cash equivalents were $12.1 million as of December 31, 2019, compared to $41.7 million as of December 31, 2018. Acer believes its cash position will be sufficient to fund its current operations through the end of 2020, excluding support for EDSIVO™ development and precommercial activities, and the planned osanetant clinical trial.
Research and Development Expenses. Research and development expenses were $2.8 million for the three months ended December 31, 2019, compared to $5.3 million for the three months ended December 31, 2018. Research and development expenses for the three months ended December 31, 2019 were primarily comprised of approximately $0.3 million related to EDSIVO™ and approximately $2.4 million related to ACER-001. Research and development expenses were $13.9 million for the year ended December 31, 2019, compared to $12.5 million for the year ended December 31, 2018. This increase of approximately $1.4 million was primarily due to increases in spending related to contract manufacturing services, regulatory consulting, and medical affairs services during the first half of 2019 in preparation for the potential launch of EDSIVO™, as well as to an increase in employee-related expenses. The increase in employee-related expenses was driven by increased headcount during the first half of 2019, as well as $0.5 million restructuring expense and increased stock-based compensation expense. These increases were partially offset by a decrease in spending related to license fees.
General and Administrative Expenses. General and administrative expenses were $2.4 million for the three months ended December 31, 2019, compared to $3.4 million for the three months ended December 31, 2018. General and administrative expenses were $16.0 million for the year ended December 31, 2019, compared to $9.3 million for the year ended December 31, 2018. This increase of $6.7 million was primarily due to a $4.6 million increase in employee-related expenses, which included $1.0 million restructuring expense, increased headcount and travel during the first half of 2019, and increased stock-based compensation expense. The remaining increase in general and administrative expenses was primarily due to an increase in expenses related to precommercial activities.
Net Loss. Net loss for the three months ended December 31, 2019 was $5.2 million, or $0.51 net loss per share (basic and diluted), compared to a net loss of $8.5 million, or $0.85 net loss per share (basic and diluted), for the three months ended December 31, 2018. Net loss for the year ended December 31, 2019 was $29.4 million, or $2.91 loss per share (basic and diluted), compared to a net loss of $21.3 million, or $2.49 loss per share (basic and diluted), for the year ended December 31, 2018.
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 three clinical-stage candidates: 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; 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
[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, 2019 2018 2019 2018 (unaudited) Operating expenses: Research and development $ 2,829,767 $ 5,312,378 $ 13,851,018 $ 12,452,424 General and administrative 2,362,572 3,387,147 16,046,423 9,261,570 Loss from operations (5,192,339 ) (8,699,525 ) (29,897,441 ) (21,713,994 ) Other income, net: Interest income 50,919 206,637 471,267 412,553 Foreign currency transaction (loss)/gain (14,864 ) (3,398 ) 8,205 20,550 Total other income, net 36,055 203,239 479,472 433,103 Net loss $ (5,156,284 ) $ (8,496,286 ) $ (29,417,969 ) $ (21,280,891 ) Net loss per share - basic and diluted $ (0.51 ) $ (0.85 ) $ (2.91 ) $ (2.49 ) Weighted average common shares outstanding - basic and diluted 10,095,176 10,054,482 10,092,179 8,555,039 SELECTED BALANCE SHEET DATA:
December 31, December 31, 2019 2018 Cash and cash equivalents $ 12,077,640 $ 41,671,284 Prepaid expenses and other current assets $ 807,356 $ 1,075,021 Property and equipment, net $ 193,974 $ 130,867 Total assets $ 21,465,511 $ 50,663,419 Total liabilities $ 3,095,195 $ 5,580,261 Total stockholders' equity $ 18,370,316 $ 45,083,158 - EDSIVO™