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)
|
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
|
Phase 2b trial did not meet primary endpoint - October 28, 2016.
|
Latest News
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NEWTON, Mass., Feb. 11, 2021 (GLOBE NEWSWIRE) -- Acer Therapeutics Inc. (NASDAQ:ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced topline results from its bioequivalence trial in which ACER-001 showed similar relative bioavailability compared to BUPHENYL® (sodium phenylbutyrate) under fed conditions. ACER-001 powder is a proprietary, taste-masked, immediate release formulation of sodium phenylbutyrate (NaPB) in development for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD).
The single-center, single-blind, randomized, single-dose crossover trial evaluated bioequivalence (BE) of ACER-001 compared to BUPHENYL® when administered under fed conditions in 36 healthy adults. The topline data from this trial showed ACER-001 to have similar pharmacokinetic (PK) profiles for both phenylbutyrate (PBA) and phenylacetate (PAA) compared to BUPHENYL® under fed conditions. Acer is initially developing ACER-001 for the treatment of patients with UCDs under Section 505(b)(2) of the Federal Food, Drug and Cosmetic Act, which provides a potentially streamlined path for sponsors that have developed drug products that rely upon data from drug products previously approved by the FDA.
"With topline data now in hand, we are moving forward with our plans to conduct a pre-NDA meeting with the FDA in the second quarter of 2021, assuming successful and timely completion of the ongoing development activities, including evaluation of long-term product stability data," said Chris Schelling, CEO and Founder of Acer. "Assuming no additional data is requested by the Agency during our pre-NDA meeting, we will plan to submit an NDA for ACER-001 for the treatment of UCDs in mid-2021. If ACER-001 is approved by the FDA, we believe its unique formulation will provide clinicians with an alternative to existing sodium phenylbutyrate-based treatments."
ACER-001 is an investigational product being studied for the treatment of patients with UCDs and MSUD and has not been approved by FDA for any indication. There can be no assurance that if submitted, a New Drug Application will be accepted by the FDA for filing and review or, if filed, that it will be approved.
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.1,2The current treatment of UCDs consists of dietary management to limit ammonia production in conjunction with medications that provide alternative pathways for the removal of ammonia from the bloodstream. Some patients may also require individual branched-chain amino acid supplementation.
Current medications for UCDs include nitrogen scavengers RAVICTI® and BUPHENYL® in which the active pharmaceutical ingredients are glycerol phenylbutyrate (GPB) and sodium phenylbutyrate (NaPB), respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications can be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons referenced for non-compliance associated with some available medications include unpleasant taste, the frequency with which medication must be taken, the number of pills, and the high cost of the medication.3
About ACER-001
ACER-001 is a powder formulation of sodium phenylbutyrate (NaPB). The proprietary formulation is designed to be both taste-masked and immediate release. ACER-001 is being developed using a microencapsulation process for the treatment of various inborn errors of metabolism, including UCDs and MSUD. ACER-001 microparticles consist of a core center, a layer of active drug, and a taste-masking coating that quickly dissolves in the stomach, allowing taste to be neutralized while still allowing for rapid systemic release. We believe that if ACER-001 is approved, its taste-masked properties will make it a viable alternative to existing NaPB-based treatments, as the unpleasant taste associated with NaPB is cited as a major impediment to patient compliance with those treatments.3 Acer has been granted orphan drug designation by the FDA for the MSUD indication. ACER-001 is under clinical investigation and its safety and efficacy have not been established. There is no guarantee that this product candidate will receive 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 programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer's product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit www.acertx.com.References
- Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University of Washington, Seattle; 1993.
- Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
- Shchelochkov OA, et al. Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives. Mol Genet Metab. 2016;8:43-47.
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]
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Acer to receive $1 million payment to obtain exclusivity and a $4 million loan from Relief
Companies working toward negotiation and execution of a definitive collaboration and license agreement by June 30, 2021
GENEVA, SWITZERLAND, and NEWTON, MA / ACCESSWIRE / January 25, 2021 / RELIEF THERAPEUTICS Holding AG ((SIX:RLF, OTCQB:RLFTF)("Relief"), a biopharmaceutical company with its lead compound RLF-100TM (aviptadil) in advanced clinical development to treat severe COVID-19 patients, and Acer Therapeutics Inc. (NASDAQ:ACER)("Acer"), a pharmaceutical company focused on the acquisition, development, and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced that the companies have signed an Option Agreement providing exclusivity for the right to negotiate a potential collaboration and license agreement for worldwide development and commercialization for ACER-001. ACER-001 (sodium phenylbutyrate) powder is a taste-masked, immediate release proprietary formulation in development for the treatment of urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD).
Under the terms of the Option Agreement, Acer will receive from Relief a $1 million non-refundable payment in return for exclusivity until June 30, 2021 to negotiate and enter into a definitive collaboration and license agreement between Acer and Relief for the development of ACER-001. Further, in connection with entering into the Option Agreement, Relief will make a $4.0 million loan to Acer. The loan, which will be secured by a lien on all of Acer's assets, will bear interest at the rate of 6% per annum and will be due in one year.
Under the terms of the proposed collaboration and license agreement, the key terms of which are set forth in the Option Agreement, if a definitive agreement is executed pursuant to these terms and closed by June 30, 2021, Acer will receive $15 million in cash (net $10 million, inclusive of the $1 million payment and offset by a repayment of the $4 million loan from Relief). In addition, Relief will agree to pay up to $20 million in U.S. development and commercial launch costs for the UCDs and MSUD indications. Further, Acer will retain development and commercialization rights in the U.S., Canada, Brazil, Turkey and Japan. The companies will split net profits from Acer's territories 60:40 in favor of Relief. Relief will also license the rights for the rest of the world, where Acer will receive from Relief a 15% net sales royalty on all revenues received in Relief's territories. Acer could also receive a total of $6 million in milestones based on the first European (EU) marketing approvals for UCDs and MSUD. There can be no assurance, however, that a definitive agreement will be successfully negotiated and executed between the parties on these terms, on other mutually acceptable terms, or at all. Except for the $1.0 million upfront payment to Acer and the $4.0 million one-year secured loan from Relief to Acer, the remaining proposed terms of the collaboration are not binding and are subject to change as a result of further diligence by Relief and negotiation of a definitive collaboration and license agreement between the parties.
Jack Weinstein, Relief's CFO and Treasurer, said, "We are excited about the opportunity to work with the Acer team to potentially develop and commercialize ACER-001 worldwide. This partnership is Relief's first initiative to build a pipeline of drugs beyond RLF-100(TM). While our core focus remains squarely on the rapid advancement of RLF-100(TM) for treatment of respiratory conditions, primarily acute respiratory distress syndrome (ARDS) due to COVID-19 infection, we are committed to establishing a diversified marketed product portfolio. ACER-001's stage of maturity fits perfectly within our strategic plan."
Chris Schelling, Acer's CEO and Founder, said, "I believe Relief shares the same values and vision that Acer has in supporting the rare disease community. This potential collaboration could provide important resources and additional expertise to help bring ACER-001 to patients worldwide suffering from debilitating diseases like UCDs and MSUD. We very much look forward to the possibility of working with the Relief team."
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ABOUT UREA CYCLE DISORDERS (UCDS)
Urea Cycle Disorders (UCDs) are a group of disorders caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia. Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits. Common symptoms of both acute and chronic hyperammonemia also include seizures and psychiatric symptoms.1,2The current treatment of UCDs consists of dietary management to limit ammonia production in conjunction with medications that provide alternative pathways for the removal of ammonia from the bloodstream. Some patients may also require individual branched-chain amino acid supplementation.
Current medical treatments for UCDs include nitrogen scavengers RAVICTI(R) and BUPHENYL(R) in which the active pharmaceutical ingredients are glycerol phenylbutyrate (GPB) and sodium phenylbutyrate (NaPB), respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications can be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons referenced for non-compliance associated with some available medications include unpleasant taste, the frequency with which medication must be taken, the number of pills, and the high cost of the medication.2
ABOUT MAPLE SYRUP URINE DISEASE
Maple Syrup Urine Disease (MUSD) is a rare but serious inherited condition whereby the human body cannot process certain amino acids, causing a harmful build-up of substances in the blood and urine. The human body breaks down protein foods such as meat and fish into amino acids. Other than a highly restricted diet of branched-chain amino acid (BCCA) free synthetic foods and formula, there are no currently approved treatments for MSUD.ABOUT ACER-001
ACER-001 is a powder formulation of sodium phenylbutyrate (NaPB). The formulation is designed to be both taste-masked and immediate release. ACER-001 is being developed using a microencapsulation process for the treatment of various inborn errors of metabolism, including UCDs and MSUD. ACER-001 microparticles consist of a core center, a layer of active drug, and a taste-masking coating that quickly dissolves in the stomach, allowing taste to be neutralized while still allowing for rapid systemic release. If ACER-001 is approved, its taste-masked properties could make it a compelling alternative to existing NaPB-based treatments, as the unpleasant taste associated with NaPB is cited as a major impediment to patient compliance with those treatments.3 Acer has been granted orphan drug designation by the FDA for the MSUD indication. ACER-001 is under clinical investigation and its safety and efficacy have not been established. There is no guarantee that this product candidate will receive FDA approval or become commercially available for the uses being investigated.ABOUT RELIEF THERAPEUTICS HOLDING AG
Relief focuses primarily on clinical-stage programs based on molecules of natural origin (peptides and proteins) with a history of clinical testing and use in human patients or a strong scientific rationale. Currently, Relief is concentrating its efforts on developing new treatments for respiratory disease indications. Its lead drug candidate RLF-100TM (aviptadil) is being investigated in two placebo-controlled U.S. phase 2b/3 clinical trials in respiratory deficiency due to COVID-19. Relief holds a patent issued in the United States and various other countries covering potential formulations of RLF-100TM.RELIEF THERAPEUTICS Holding AG is listed on the SIX Swiss Exchange under the symbol RLF and quoted in the U.S. on OTCQB under the symbol RLFTF. For more information, visit: www.relieftherapeutics.com.
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ABOUT ACER THERAPEUTICS INC.
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer's pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO(TM) (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer's product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit www.acertx.com.REFERENCES
- Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University of Washington, Seattle; 1993.
- Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
- Shchelochkov OA, et al. Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives. Mol Genet Metab. 2016;8:43-47.
RELIEF FORWARD-LOOKING STATEMENTS
This communication expressly or implicitly contains certain forward-looking statements concerning RELIEF THERAPEUTICS Holding AG, Inc. and its businesses. The results reported herein may or may not be indicative of the results of future and larger clinical trials for ACER-001 for the treatment of UCDs and MSUD, nor whether the ongoing clinical trials of Relief's lead compound, RLF-100(TM) (aviptadil) in advanced clinical development to treat severe COVID-19 patients, will be successful. Such statements involve certain known and unknown risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of RELIEF THERAPEUTICS Holding AG to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. RELIEF THERAPEUTICS Holding AG is providing this communication as of this date and do not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise.ACER FORWARD-LOOKING STATEMENTS
This press release contains "forward-looking statements" that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the structure, terms, timing and entry into a definitive agreement for the proposed collaboration between Acer and Relief with respect to ACER-001; the shared values, vision and results of the potential collaboration of Acer and Relief; the potential for ACER-001 to target diseases; the adequacy of Acer's capital to support its future operations and its ability to successfully continue its development programs; Acer's ability to secure the additional capital necessary to fund its various product candidate development programs; and the development and commercial potential of any of Acer's product candidates including ACER-001. Acer may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management's current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with Acer's ability to successfully negotiate and execute a definitive collaboration agreement with Relief on the proposed terms, on other mutually acceptable terms, or at all, Acer's ability to repay the $4 million secured loan from Relief, the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to fund Acer's various product candidate development programs and to meet its business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by Acer's intellectual property, risks related to the drug discovery and the regulatory approval process and the impact of competitive products and technological changes. Acer disclaims any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures Acer makes in its filings with the Securities and Exchange Commission, including its Quarterly Reports on Form 10-Q and its Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.CORPORATE CONTACTS
RELIEF THERAPEUTICS Holding AG:
Jack Weinstein
Chief Financial Officer and Treasurer
[email protected]
ACER Therapeutics:
Jim DeNike
Acer Therapeutics Inc.
+1 844-902-6100
[email protected]MEDIA CONTACTS
Relief (Europe):
Anne Hennecke / Brittney Sojeva
MC Services AG
[email protected]
+49 (0) 211-529-252-14INVESTOR RELATIONS CONTACTS
Relief (Europe):
Anne Hennecke / Brittney Sojeva
MC Services AG
[email protected]
+49 (0) 211-529-252-14
Acer Therapeutics:
Hans Vitzthum
LifeSci Advisors
+1 617-430-7578
[email protected]SOURCE: Relief Therapeutics Holdings AG
View source version on accesswire.com:
https://www.accesswire.com/625666/Relief-Therapeutics-and-Acer-Therapeutics-Sign-Option-Agreement-for-Exclusivity-to-Negotiate-a-Collaboration-and-License-Agreement-for-the-Worldwide-Development-and-Commercialization-of-ACER-001-for-the-Treatment-of-Urea-Cycle-Disorders-and-Maple-Syrup
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NEWTON, Mass., Dec. 22, 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 full enrollment of its pivotal trial evaluating the bioequivalence (BE) of ACER-001 compared to BUPHENYL® (sodium phenylbutyrate). Acer is developing ACER-001 for the treatment of urea cycle disorders (UCDs).
The single-center, single-blind, randomized, single-dose crossover trial is designed to demonstrate the BE of ACER-001 compared to BUPHENYL® under fed conditions, in approximately 36 healthy adults. Results of the trial are expected in the first quarter of 2021.
"We completed the first bridging study in the first quarter of 2020, the results of which demonstrated bioequivalence to BUPHENYL® under fasted conditions, but we also confirmed a significant food effect with sodium phenylbutyrate,"1 said Chris Schelling, CEO and Founder of Acer. "After meeting with the FDA in the third quarter of this year to discuss the development plan, it was determined that in light of the food effect a second BE study conducted under fed conditions would be needed to bridge to BUPHENYL® under a 505(b)(2) application. This was a challenging request in the face of COVID-19, and I am pleased to say that our partners at PPD, the global contract research organization, did an incredible job getting this trial fully enrolled before the end of the year."
Upon successful completion of ongoing development activities, Acer intends to submit a request to the U.S. Food and Drug Administration (FDA) in the first quarter of 2021 for a pre-NDA meeting on ACER-001.
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.2,3The current treatment of UCDs consists of dietary management to limit ammonia production in conjunction with medications that provide alternative pathways for the removal of ammonia from the bloodstream. Some patients may also require individual branched-chain amino acid supplementation.
Current medical treatments for UCDs include nitrogen scavengers RAVICTI® and BUPHENYL® in which the active pharmaceutical ingredients are glycerol phenylbutyrate (GPB) and sodium phenylbutyrate (NaPB), respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications can be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons referenced for non-compliance associated with some available medications include unpleasant taste, the frequency with which medication must be taken, the number of pills, and the high cost of the medication.3
About ACER-001
ACER-001 is a taste-masked, immediate-release proprietary powder formulation for oral administration of sodium phenylbutyrate (NaPB) via suspension. ACER-001 is being developed using a microencapsulation process 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. We believe that if ACER-001 is approved, its taste-masked properties will make it a compelling alternative to existing NaPB-based treatments, as the unpleasant taste associated with NaPB is cited as a major impediment to patient compliance with those treatments.4 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 programs: 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); emetine hydrochloride, a host-directed therapy against a variety of infectious diseases, initially 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; 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.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).
- 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 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] -
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NEWTON, Mass., Dec. 17, 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 the publication of additional long-term data from COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS) patients in the European Journal of Vascular and Endovascular Surgery (EJVES). The publication, entitled "Celiprolol Treatment in Patients with Vascular Ehlers-Danlos Syndrome,"1 can be found at https://www.ejves.com/action/showPdf?pii=S1078-5884%2820%2930930-8.
This published study describes outcomes in 40 patients with COL3A1-positive vEDS that were clinically monitored and treated with celiprolol in a single center retrospective study at Uppsala University Hospital, a national referral center for vEDS patients in Sweden, between the years 2011 and 2019. Patients were followed for a median of 22 months (range 1-98 months) with a total follow up of 106 patient years. Assessments were conducted by a multidisciplinary team, including vascular surgeons, angiologists and clinical geneticists. Celiprolol was administered twice daily and titrated up by 100 mg steps to a maximum of 400 mg per day. Some patients were treated concomitantly or separately with other medications. Sixty-five percent of the patients reached the target dose of 400 mg and the medication was generally well tolerated.
The annual risk of a major vascular event was 4.7% in this study, noted as being similar to that observed in the celiprolol treatment-arm of the BBEST2 trial (5%) and lower than in the BBEST trial control arm (12%). Five patients suffered major vascular events, four of which were fatal. No significant predictor of vascular events was identified by the authors.
"The results from this long-term registry study and the increasing length of follow up provide further evidence of celiprolol's potential protective effect in COL3A1-positive vEDS patients," said Martin Björck, MD, PhD, Professor Emeritus of Vascular Surgery, Department of Surgical Sciences at Uppsala University.
"Dr. Björck is an incredibly well-respected vascular specialist in Europe. We are encouraged by his team's results that add to the growing body of evidence in support of celiprolol's potential benefit in treating COL3A1-positive vEDS patients," said Chris Schelling, CEO and Founder of Acer Therapeutics. "Since our meeting with the FDA's Office of New Drugs (OND) last February, we have been working diligently to evaluate various forms of confirmatory evidence needed to meet the substantial evidence standard. With our partners, we believe we have identified a plan to collect additional data that supports the results from the COL3A1-positive analysis from the BBEST trial and could help meet the standard. As such, we intend to submit a request to the FDA in the next several weeks for a meeting to discuss the appropriateness of our approach."
An infographic accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/f7935f3b-34a5-4373-881a-dee9b971a174
About EDSIVO™
Ehlers-Danlos Syndrome (EDS) is a group of hereditary disorders of connective tissue. vEDS is the most severe subtype where patients suffer from life threatening arterial dissections and ruptures, as well as intestinal and uterine ruptures. The median age of death in the U.S. is 51 years.3 An Acer-commissioned patient-finder study phenotypically identified 4,169 vEDS patients in the U.S. from an analysis of a commercially available patient claims database with data of approximately 190 million unique patient lives. Based on that information, Acer estimates the prevalence of phenotypically-defined vEDS in the U.S. could be greater than 1 in 45,000. Currently, there are no FDA-approved therapies for vEDS.Acer is advancing EDSIVO™, a new chemical entity (NCE), for the treatment of COL3A1-positive vEDS based on published results from a randomized controlled clinical trial of celiprolol (BBEST).2 In addition, long-term data has now been published from separate French4 and Swedish1 vEDS patient registries.
The FDA granted a priority review of the EDSIVO™ NDA and subsequently issued a Complete Response Letter (CRL) in June 2019. The CRL stated that it will be necessary to conduct an adequate and well-controlled trial to determine whether celiprolol reduces the risk of clinical events in patients with vEDS. In March 2020, Acer received a response to its Formal Dispute Resolution Request (FDRR) from the OND of the FDA stating that it had denied its appeal of the CRL in relation to the NDA for EDSIVO™. In its Appeal Denied letter, the OND described 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 vEDS with a confirmed COL3A1 mutation. The OND referred to the FDA Guidance document issued in December 20195, where substantial evidence of effectiveness can be provided by two or more adequate and well-controlled studies demonstrating efficacy, or a single positive adequate and well-controlled study plus confirmatory evidence. Neither resubmission nor the prospect of approval of the EDSIVO™ NDA is assured. EDSIVO™ received FDA Orphan Drug Designation for the treatment of vEDS in 2015. EDSIVO™ is an investigational drug candidate that has not been approved by the FDA for any indication.
References
- Baderkhan, H, et al. Celiprolol Treatment in Patients with Vascular Ehlers-Danlos Syndrome. European Journal of Vascular and Endovascular Surgery. November 20, 2020.
- Ong KT, et al. Effect of celiprolol on prevention of cardiovascular events in vascular Ehlers-Danlos syndrome: a prospective randomised, open, blinded-endpoints trial. Lancet. 2010;376(9751):1476-1484
- Pepin M, et al. Clinical and genetic features of Ehlers-Danlos syndrome type IV, the vascular type. N Engl J Med. 2000; 342:673-80
- Frank M, et al. Vascular Ehlers-Danlos Syndrome: Long-Term Observational Study. J Am Coll Cardiol. 2019 Apr, 73 (15) 1948–1957
- FDA guidance "Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products", December 2019
About Acer Therapeutics Inc.
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer's pipeline includes four programs: ACER-001 (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); emetine hydrochloride, a host-directed therapy against a variety of infectious diseases, initially 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; 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] -
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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