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 |
|---|---|---|
|
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 2H 2021.
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Emetine
COVID-19
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Phase 2/3
Phase 2/3
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Phase 2/3 trial to be initiated 1H 2021.
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EDSIVO
Vascular Ehlers-Danlos Syndrome
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CRL
CRL
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CRL issued June 25, 2019.
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Tovaxin (Tcelna)
Secondary Progressive MS (SPMS)
|
Phase 2b
Phase 2b
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Phase 2b trial did not meet primary endpoint - October 28, 2016.
|
Latest News
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NEWTON, Mass., Nov. 10, 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 third quarter ended September 30, 2020, and provided an update on the Company's recent corporate developments.
"This quarter, we continued to make important progress in the advancement of our four programs," said Chris Schelling, CEO and Founder of Acer. "We received valuable feedback from the FDA regarding our regulatory strategy for ACER-001 for urea cycle disorders patients, and assuming all our development activities stay on track, we anticipate having a pre-NDA meeting in the mid-first half of 2021. Regarding our emetine program, several in vivo studies are currently being conducted to evaluate the drug's safety and its specific activity against SARS-CoV-2 in the Syrian Golden hamster and ferret models. Coupled with the drug's in vitro data and emetine's long history of use in humans for other indications, we should have a good understanding of this candidate's potential as a therapeutic against COVID-19 relatively soon."
Third Quarter 2020 and Recent Highlights
- Emetine
- Animal studies ongoing to further evaluate emetine's safety and antiviral activity against COVID-19, the disease caused by infection with the SARS-CoV-2 virus
- Obtained additional pre-Investigational New Drug (IND) feedback from the Division of Antivirals (DAV) at the U.S. Food and Drug Administration (FDA)
- Progressing toward U.S.-based cGMP synthetic active pharmaceutical ingredient (API) manufacturing in 2021
- Continued to work with federal agencies and private research organizations toward the goal of securing non-dilutive funding in support of emetine development
- ACER-001
- Received feedback on the Type C meeting with the FDA regarding requirements for the planned ACER-001 505(b)(2) New Drug Application (NDA) submission for treatment of urea cycle disorders (UCDs)
- Planning to conduct a single-center, single-blind, randomized, single-dose crossover trial designed to demonstrate bioequivalence (BE) of ACER-001 compared to BUPHENYL® under fed conditions, in approximately 36 healthy adults
- Gained insight from market research with key stakeholders – geneticists, dietitians and UCDs patients – to better understand the reaction to ACER-001's value proposition and begin quantifying the potential market opportunity in UCDs
- EDSIVO™
- Developing a plan to generate confirmatory evidence needed to support the potential resubmission of the EDSIVO™ NDA (although neither EDSIVO™ NDA resubmission nor approval is assured)
- Osanetant
- Continued to advance active pharmaceutical ingredient (API) manufacturing and other necessary steps to support IND submission
- Corporate
- Ended the third quarter with $6.2 million in cash and cash equivalents. Acer believes its cash position at the end of the third quarter, combined with an additional $1.0 million of proceeds subsequently received through October 26, 2020 from the sales of common stock under its ATM facility and through its equity purchase agreement with Lincoln Park Capital, 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, which is also subject to ongoing discussions with the FDA
- Ended the third quarter with $6.2 million in cash and cash equivalents. Acer believes its cash position at the end of the third quarter, combined with an additional $1.0 million of proceeds subsequently received through October 26, 2020 from the sales of common stock under its ATM facility and through its equity purchase agreement with Lincoln Park Capital, 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, which is also subject to ongoing discussions with the FDA
Upcoming Milestones
- Emetine
- Anticipate in vivo efficacy results from ongoing animal studies evaluating emetine's anti-viral activity against COVID-19 by the end of the fourth quarter 2020
- Targeting IND submission and potential initiation of the planned Phase 2/3 trial evaluating emetine in high-risk COVID-19 outpatients in the first half of 2021, subject to ongoing discussions with the FDA and additional capital
- ACER-001
- Targeting completion of a BE trial of ACER-001 compared to BUPHENYL® under fed conditions, in approximately 36 healthy adults, in the first quarter of 2021
- Anticipate holding a pre-NDA meeting with FDA in mid-first half of 2021, assuming successful and timely completion of the ongoing development activities (including the planned BE fed trial, nonclinical work and evaluation of long-term product stability data) and subject to additional capital
- EDSIVO™
- Expect to submit a meeting request with FDA to discuss Acer's proposed plan to provide sufficient confirmatory evidence by the end of the fourth quarter of 2020
- If successful, potentially satisfy the substantial evidence of effectiveness needed to support a possible resubmission of the EDSIVO™ NDA (although neither EDSIVO™ NDA resubmission nor approval is assured)
- Osanetant
- Targeting IND submission in the second quarter of 2021 subject to discussions with FDA
- Plan to initiate a Phase 1/2 pharmacokinetic/pharmocodynamic and safety trial evaluating osanetant in patients with medically and/or surgically induced vasomotor symptoms (iVMS) in the second half of 2021, subject to additional capital
Financial Results for the Third Quarter 2020
Cash position. Cash and cash equivalents were $6.2 million as of September 30, 2020, compared to $12.1 million as of December 31, 2019. Acer believes its cash position at the end of the third quarter, combined with an additional $1.0 million of proceeds subsequently received through October 26, 2020 from the sales of common stock under its ATM facility and through its purchase agreement with Lincoln Park Capital, 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, which is also subject to ongoing discussions with the FDA.
Research and Development Expenses. Research and development expenses were $3.2 million for the three months ended September 30, 2020, compared to $2.8 million for the three months ended September 30, 2019. This increase of $0.4 million was primarily due to an increase in expenses for contract research related to preclinical studies, partially offset by decreases in regulatory consulting and contract manufacturing expenses. Research and development expenses for the three months ended September 30, 2020 were primarily comprised of $1.8 million related to emetine, $1.1 million related to ACER-001, and $0.3 million related to osanetant.
General and Administrative Expenses. General and administrative expenses were $2.7 million for the three months ended September 30, 2020, compared to $2.5 million for the three months ended September 30, 2019. This increase of $0.2 million was primarily due to an increase in expenses for consulting and professional services, partially offset by decreases in expenses related to travel and information technology.
Net Loss. Net loss for the three months ended September 30, 2020 was $5.9 million, or $0.51 net loss per share (basic and diluted), compared to a net loss of $5.3 million, or $0.52 net loss per share (basic and diluted), for the three months ended September 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 programs: 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 our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management's current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to fund our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, the substantial costs and diversion of management's attention and resources which could result from pending securities litigation, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.Investor Contact:
Hans Vitzthum
LifeSci Advisors
Ph: 617-430-7578
[email protected]Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected]ACER THERAPEUTICS INC.
CONSOLIDATED STATEMENTS OF OPERATIONS
(Unaudited)
Three Months Ended September 30, 2020 2019 Operating expenses: Research and development $ 3,227,048 $ 2,828,787 General and administrative 2,661,989 2,533,678 Loss from operations (5,889,037 ) (5,362,465 ) Other (expense) income, net: Interest and other (expense) income, net (4,129 ) 91,321 Foreign currency transaction (loss) gain (35,092 ) 19,671 Total other (expense) income, net (39,221 ) 110,992 Net loss $ (5,928,258 ) $ (5,251,473 ) Net loss per share - basic and diluted $ (0.51 ) $ (0.52 ) Weighted average common shares outstanding - basic and diluted 11,514,254 10,095,176 SELECTED BALANCE SHEET DATA (Unaudited):
September 30, December 31, 2020 2019 Cash and cash equivalents $ 6,157,448 $ 12,077,640 Prepaid expenses and other current assets $ 846,399 $ 807,356 Property and equipment, net $ 143,145 $ 193,974 Total assets $ 15,366,903 $ 21,465,511 Total liabilities $ 4,863,391 $ 3,095,195 Total stockholders' equity $ 10,503,512 $ 18,370,316 
- Emetine
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NEWTON, Mass., Oct. 01, 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 that Acer's management team will provide a virtual corporate overview at, and participate in, the BIO Investor Forum Digital taking place on October 13-15, 2020. The presentation will be available on-demand during the conference.
More information on Acer and a replay of the presentation can be found in the "Events and Presentations" section of Acer's website at https://www.acertx.com/investor-relations/events-presentations/.
About Acer Therapeutics Inc.
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer's pipeline includes four 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.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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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
[email protected]Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected]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 - Emetine
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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.7,8The 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
- 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).
- https://www.hzndocs.com/BUPHENYL-Prescribing-Information.pdf
- https://www.hzndocs.com/RAVICTI-Prescribing-Information.PDF
- 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.
- United States Patent number US8642012B2.
- 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.
- Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University of Washington, Seattle; 1993.
- Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
- Shchelochkov OA, et al. Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives. Mol Genet Metab. 2016;8:43-47.
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
[email protected]Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected] -
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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.
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