Acer Therapeutics Receives Notice of Allowance of Key US Patent Application Covering EDSIVO™ (celiprolol)

NEWTON, MA – Oct. 26, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER), a clinical stage 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 US Patent and Trademark Office (USPTO) has issued a Notice of Allowance for Acer’s patent application No. 16/930,208, exclusively licensed from Assistance Publique—Hôpitaux de Paris, for claims related to certain methods of treating vascular Ehlers-Danlos syndrome (vEDS) with celiprolol. The allowed patent claims in the application titled, “Method of Providing Celiprolol Therapy to a Patient,” include the dosing regimen in Acer’s ongoing Phase 3 DiSCOVER (Decentralized Study of Celiprolol on vEDS-related Event Reduction) clinical trial of EDSIVO™ (celiprolol) for the treatment of patients with COL3A1-positive vEDS.

“We are extremely pleased to receive this Notice of Allowance from the USPTO for our method of treatment of vEDS with celiprolol, as we continue to advance its development for the potential treatment of COL3A1-positive vEDS patients and other possible indications,” said Jeff Davis, Chief Business Officer at Acer. “These allowed method of use claims are based on the higher survival rate observed in vEDS patients when using a dose titration regimen to reach what we believe could be the preferred celiprolol dose as previously described in the ‘Long-Term Observational Study’ published in the Journal of American College of Cardiology (JACC)1. When issued, this patent will build on the potential regulatory exclusivities from celiprolol’s Orphan Drug Designation and New Chemical Entity (NCE) status. We look forward to the continued advancement of this program.”

Acer’s patent is expected to be issued in Q4 2022 and expire in 2038. If it receives marketing approval for EDSIVO™ after completion of the DiSCOVER trial and NDA resubmission, Acer intends to submit the patent for listing by the FDA in its Approved Drug Products with Therapeutic Equivalence Evaluations, or Orange Book.

About the DiSCOVER Trial
The DiSCOVER trial is a prospective, Phase 3, randomized, double-blind, placebo-controlled efficacy trial designed to evaluate EDSIVO™ in patients with genetically confirmed COL3A1-positive vEDS using a decentralized clinical trial design and an independent adjudication committee. The primary objective of the trial is to determine whether EDSIVO™ reduces the occurrence of vEDS-related clinical events requiring medical attention, including fatal and non-fatal cardiac or arterial events, uterine rupture, intestinal rupture, and/or unexplained sudden death, relative to placebo as measured by time to event. Acer plans to enroll approximately 150 COL3A1-positive vEDS patients, all in the U.S., randomized 2:1 to receive either EDSIVO™ or placebo, respectively. Individuals seeking more information on the EDSIVO™ pivotal clinical trial are invited to visit www.discoverceliprolol.com.

Once the trial is fully enrolled, the duration of the DiSCOVER trial is currently estimated to be up to approximately 3.5 years to completion (based on statistical power calculations and number of primary events), which will require additional capital in Q4 2022. One interim analysis (based on number of primary events) is also planned at approximately 24 months after full enrollment.

EDSIVO™ (celiprolol) is an investigational product candidate which has not been approved by the FDA. There is no guarantee that this product candidate will receive regulatory authority approval or become commercially available for any indications in the US.

About vEDS
Ehlers-Danlos syndrome (EDS) is an autosomal inherited disorder caused by mutations in the genes responsible for the structure, production, or processing of collagen, an important component of the connective tissues in the human body, or proteins that interact with collagen. EDS is a spectrum disorder where patients present with various forms, the most serious of which is vascular Ehlers-Danlos syndrome (vEDS), also known as vEDS type IV, which is generally caused by a mutation in the COL3A1 gene resulting in reduced collagen levels. vEDS causes abnormal fragility in blood vessels, which can give rise to aneurysms, abnormal connections between blood vessels known as arteriovenous fistulas, arterial dissections, and spontaneous vascular ruptures, all of which can be potentially life-threatening. Gastrointestinal and uterine fragility or rupture also commonly occur in vEDS patients.

Spontaneous arterial rupture has a peak incidence in the third or fourth decade of life in vEDS patients but may occur earlier and is the most common cause of sudden death in vEDS patients. Arterial rupture or dissection events occur in about 25% of patients before the age of 20 but increase to roughly 90% of patients by age 40. The median survival age of vEDS patients in the U.S. is 51 years, with arterial rupture being the most common cause of sudden death.2 Based on an analysis of diagnosed vEDS patients from the Truven MarketScan® database and U.S. population data, Acer projects the total COL3A1-positive vEDS patient prevalence in the U.S. could be as high as 7,000 patients. Currently, there are no approved pharmacologic therapies anywhere in the world for vEDS.

About EDSIVO™ (celiprolol)
EDSIVO™ (celiprolol) is a new chemical entity (NCE) currently in Phase 3 development for the treatment of COL3A1-positive vEDS patients to potentially reduce the risk of arterial and other hollow organ clinical events. In October 2010, data was published in the Lancet from the BBEST trial designed to assess the preventative effect of celiprolol for major cardiovascular events in patients with vEDS via a multicenter, prospective, randomized, open trial with blinded evaluation of clinical events.3 In addition, data from long-term observational studies of patients treated with celiprolol in France and Sweden were published in JACC in April 20191 and in the European Journal of Vascular and Endovascular Surgery (EJVES) in November 20204, respectively. Data from these and other publications can be found at www.www.acertx.com. Acer’s original NDA was submitted based on data obtained from the BBEST trial and accepted for filing in October 2018 with priority review. Following FDA review, Acer received a Complete Response Letter (CRL) in June 2019 stating that it will be necessary to conduct an adequate and well-controlled trial to determine whether EDSIVO™ reduces the risk of clinical events in patients with vEDS. In April 2022, the FDA granted celiprolol Breakthrough Therapy designation in the US for the treatment of patients with COL3A1-positive vEDS. In May 2022, Acer confirmed agreement with the FDA under an SPA for its Pivotal Phase 3 clinical trial of EDSIVO™ for the treatment of patients with COL3A1-positive vEDS, and patient screening was initiated in June 2022. Celiprolol received FDA Orphan Drug Designation for the treatment of vEDS in 2015.

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 investigational programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS) and post-traumatic stress disorder (PTSD); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of viruses, including cytomegalovirus, Zika, dengue, Ebola and COVID-19. For more information, visit www.www.acertx.com.

References

  1. Frank M, et al. Vascular Ehlers-Danlos Syndrome: Long-Term Observational Study. J Am Coll Cardiol. 2019 Apr, 73 (15) 1948–1957
  2. Pepin, et al. Survival is affected by mutation type and molecular mechanism in vascular Ehlers–Danlos syndrome (EDS type IV). Genet Med. 2014 Dec;16(12):881-8.
  3. 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
  4. Björck M, et al. Celiprolol Treatment in Patients with Vascular Ehlers-Danlos Syndrome. European Journal of Vascular and Endovascular Surgery. November 20, 2020.

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, including statements regarding the Company’s plans with respect to the continued advancement of the method of treatment of vEDS with celiprolol, the expected patent issuance date and duration, that the Company intends to submit the patent for listing by the FDA in its Approved Drug Products with Therapeutic Equivalence Evaluations program, the objectives, potential results, and duration of the DiSCOVER trial, including with respect to the Company’s plans for patient enrollment, the interim analysis and timing thereof, and the need for additional capital in Q4 2022, as well as statements regarding any future regulatory approval or commercial availability of our product candidate EDSIVO™ (celiprolol), are forward-looking statements. Our pipeline products are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. 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, 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 Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Acer Contacts

Corporate contact:
Jim DeNike
Acer Therapeutics Inc.
jdenike@www.acertx.com
+1-844-902-6100

Investor contact:
Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-332-895-3226

# # #

Acer Therapeutics Announces University of North Carolina Receives Department of Defense Grant to Support the Proposed Investigator Sponsored OASIS Trial of ACER-801 (Osanetant) to Reduce the Frequency and Severity of Post-Traumatic Stress Disorder

NEWTON, MA — October 6, 2022 –Acer Therapeutics Inc. (Nasdaq: ACER), a clinical stage 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 University of North Carolina (UNC) Institute for Trauma Recovery has been awarded a $3 million grant from the Department of Defense (DoD) to investigate the potential of ACER-801 (osanetant) to reduce the frequency and severity of acute stress disorder and post-traumatic stress disorder (PTSD). Acute stress disorder refers to the body’s immediate response to trauma, whereas PTSD is the long-term effects of trauma.

“Historically, we have been able to provide emergency care to address immediate and long-term problems after visible wounds using tools such as sutures and antibiotics. However, we still have nothing to offer trauma survivors, whether in the emergency department or on the battlefield immediately after trauma, to prevent the development of ‘invisible wounds’,” said Samuel McLean, MD, Professor at UNC School of Medicine, and lead principal investigator of the proposed study. Dr. McLean continued, “We need to investigate potential treatments like ACER-801 in an effort to better address these challenges.”

The proposed Osanetant After Stress to Increase recovery Success (OASIS) trial will examine the safety and efficacy of ACER-801 to reduce acute stress response symptoms, post-traumatic stress disorder symptoms and behavioral changes among patients presenting to the emergency department after a motor vehicle collision. It is intended to enroll a total of 180 subjects who will be randomized in the emergency department, to receive a low or high dose of ACER-801 or placebo in the emergency department and be discharged with a two-week supply of study drug. Participating sites would include Washington University in St. Louis, University of Massachusetts Chan Medical School, Rhode Island Hospital, University of Florida College of Medicine – Jacksonville, and Indiana University School of Medicine.

Initiation of patient enrollment in the proposed investigator-sponsored OASIS trial is anticipated in the first half of 2023, subject to Investigational New Drug (IND) application filing and US Food and Drug Administration (FDA) clearance.

The OASIS trial will build upon a foundation of knowledge and infrastructure developed through the UNC-led, $40 million AURORA initiative. The AURORA study is a major national research initiative to improve the understanding, prevention, and recovery of individuals who have experienced a traumatic event. AURORA is supported by funding from NIH, One Mind, private foundations, and partnerships with leading tech companies such as Mindstrong Health and Verily Life Sciences, the healthcare arm of Google’s parent company Alphabet.

“We are proud to be partnering with a leading academic institution in the field of trauma recovery as we seek to explore ACER-801 as a treatment option to reduce the frequency and severity of PTSD. Leveraging the support from the AURORA initiative allows Acer and UNC to streamline trial efficiency, reduce costs and increase trial power through enriching the target patient population, while utilizing the Department of Defense’s non-dilutive capital to primarily fund OASIS,” said Adrian Quartel, MD, FFPM, Chief Medical Officer of Acer. “The data from thousands of motor vehicle collisions collected through the AURORA initiative should allow us to better predict the correlation of the emergence of acute stress disorder or PTSD symptoms following a motor vehicle collision. We look forward to the planned OASIS trial following IND filing and FDA clearance.”

Added Brandon Staglin, President of One Mind “We are thrilled to see how our funding to the AURORA initiative over the last five years is accelerating further advancements such as the OASIS Trial. The targeted outcomes of the OASIS Trial are the types of results that One Mind supports and of incredible value to anyone who experiences trauma and traumatic stress.”

Acute and chronic stress disorders can affect both civilian and military populations. According to the National Center for PTSD, in the US about 60% of men and 50% of women experience at least one trauma in their lives.2 In the US alone, one-third of emergency department visits (40-50 million patients per year) are for evaluation after trauma exposures, and in a 2014 study involving 3,157 US veterans, 87% reported exposure to at least one potentially traumatic event during their service.3 Moreover, as many as 500,000 US troops who served in wars between 2001 and 2015 were diagnosed with PTSD.4

Rationale for ACER-801 (osanetant) Evaluation in Post-Traumatic Stress Disorder
The Tacr3 gene encodes tachykinin receptor 3 (NK3R), which belongs to the tachykinin receptor family. This family of proteins includes typical G protein-coupled receptors and belongs to the rhodopsin subfamily. NK3R functions by binding to its high-affinity ligand, Neurokinin B (NKB), which is encoded by the Tac3 (human) gene. The role of NKB-NK3R in growth and reproduction has been extensively studied, but NKB-NK3R is also widely expressed in the nervous system from the spinal cord to the brain and is involved in both physiological and pathological processes in the nervous system.5  In animal models, Tac2 (mice) mRNA levels are rapidly up-regulated during fear consolidation 30 minutes after fear conditioning, and subsequent NKB-NK3R activation can lead to over stress sensitization and the consolidation of fear,6 and treatment with osanetant has been shown to block a critical fear/stress sensitization step in the brain.7,8,9 An effective therapeutic to reduce acute and persistent/long-term psychological and somatic symptoms would fulfill a large unmet need.

About Acer Therapeutics
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 investigational programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS) and post-traumatic stress disorder (PTSD); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of viruses, including cytomegalovirus, Zika, dengue, Ebola and COVID-19. For more information, visit www.www.acertx.com.

References

  1. Sidran Institute.  Traumatic Stress Education & Advocacy Fact Sheet.
  2. National Center for PTSD.  How Common is PTSD in Adults?
  3. Wisco et al. 2014 J Clin Psychiatry.  Posttraumatic stress disorder in the US veteran population: results from the National Health and Resilience in Veterans Study. J Clin Psychiatry . 2014 Dec;75(12):1338-46
  4. Thompson 2015. Unlocking the secrets of PTSD. Time 2015;185:40–43
  5. Zhang et al. ACS Chemical Neuroscience 2020 11 (19), 2935-2943
  6. Al Abed et. Al, Biological Psychiatry 2021
  7. Andero R, Dias BG, Ressler KJ. A role for Tac2, NkB, and Nk3 receptor in normal and dysregulated fear memory consolidation. Neuron. 2014;83(2):444-454
  8. Andero R, Daniel S, Guo JD, et al. Amygdala-Dependent Molecular Mechanisms of the Tac2 Pathway in Fear Learning. Neuropsychopharmacology. 2016;41(11):2714-2722
  9. Zelikowsky M, Ding K, Anderson DJ. Neuropeptidergic Control of an Internal Brain State Produced by Prolonged Social Isolation Stress. Cold Spring Harb Symp Quant Biol. 2018;83:97-103

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 are forward-looking statements. Examples of such statements include, but are not limited to, statements about the role we believe ACER-801 could play in reducing the frequency and severity of PTSD, the planned clinical evaluation of ACER-801 for such indication, plans with respect to the OASIS trial, including enrollment, timing and participants, and the continued development of ACER-801 for treatment of iVMS. Our pipeline products (including ACER-801) are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. 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, the availability of financing to fund our pipeline product development programs and general corporate operations as well as risks related to drug development and the regulatory approval process, including the timing and requirements of regulatory actions. 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 Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Acer Contacts

Corporate Contact:
Jim DeNike
Acer Therapeutics Inc.
jdenike@www.acertx.com
+1-844-902-6100

Investor contact:
Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-332-895-3226

# # #

Acer Therapeutics Announces Expansion of ACER-801 (osanetant) Development Indications to Include Post-Traumatic Stress Disorder

NEWTON, MA October 5, 2022 – 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 expansion of ACER-801 (osanetant) into a new indication, for the reduction of the frequency and severity of acute stress disorder and post-traumatic stress disorder (PTSD). Acute stress disorder refers to the body’s immediate response to trauma, whereas PTSD is defined as the long-term effects of trauma.

Studies conducted at Emory University screened thousands of genes that were activated in the brains of mice following fear conditioning events. The top gene identified was Tac2, which is responsible for the production of the peptide, Neurokinin B (NKB), in mice. The researchers showed that the Tac2 gene, expressed by neurons specifically within the amygdala, is required for modulating fear memories, and that NKB, and its specific receptor, NK3R, are also involved in the consolidation of fear memories. By administering the potent and specific NK3R antagonist, osanetant, they were able to block fear memory consolidation shortly after exposure to a trauma, potentially providing a novel therapeutic approach for disorders with altered fear learning such as PTSD.1

“Immediately – hours to several days – after trauma exposure, memory remains in a labile state, called the memory consolidation period, and blocking fear memory consolidation after trauma exposure could lower the frequency and severity of PTSD in trauma patients,” said Kerry Ressler, MD, PhD, Chief Scientific Officer and James and Patricia Poitras Chair in Psychiatry at McLean Hospital.

“Activation of the NK3 receptor pathway in the central amygdala is necessary and sufficient for the modulation of fear memories, and we have learned that by blocking this pathway with osanetant, we can block the consolidation of fear memories in animal models,”1 added Dr. Ressler. “Osanetant is a promising agent that could reduce the frequency and or severity of PTSD for millions of people who experience a traumatic event.” 

Acer previously entered into an agreement with Emory for an exclusive worldwide license to US Patent No. 10,314,835, US Application 15/320,952, and European Patent No. EP3160469 covering certain methods of treating or preventing PTSD with osanetant. 

“We are pleased to further expand our ACER-801 development program into PTSD, an increasingly prevalent psychiatric disorder that affects millions every year. Today’s announcement further validates Acer’s strategy of identifying and developing treatments based on promising technology that can be applied in new ways for use in diseases with high unmet need,” commented Chris Schelling, CEO and Founder of Acer Therapeutics. “While the role of the NK3R pathway in the hypothalamus to manage thermoregulation is well-established in clinical trials, this opportunity explores an entirely different mechanism of action for the drug. We look forward to presenting our clinical development plan for ACER-801 for the reduction of frequency and severity of PTSD in the near future.”

According to the National Center for PTSD, in the US about 6 of every 10 men (or 60%) and 5 of every 10 women (or 50%) experience at least one trauma in their lives leading to about 12 million adults in the U.S. have PTSD during a given year.3  In the US alone, one-third of emergency department visits are for evaluation after trauma exposures and up to 20% of people who have experienced a traumatic event will develop PTSD.4

Rationale for ACER-801 (osanetant) Evaluation in Post-Traumatic Stress Disorder
The Tacr3 gene encodes tachykinin receptor 3 (NK3R), which belongs to the tachykinin receptor family. This family of proteins includes typical G protein-coupled receptors and belongs to the rhodopsin subfamily. NK3R functions by binding to its high-affinity ligand, Neurokinin B (NKB), which is encoded by the Tac3 (human) gene. The role of NKB-NK3R in growth and reproduction has been extensively studied, but NKB-NK3R is also widely expressed in the nervous system from the spinal cord to the brain and is involved in both physiological and pathological processes in the nervous system.5  In animal models, Tac2 (mice) mRNA levels are rapidly up-regulated during fear consolidation 30 minutes after fear conditioning, and subsequent NKB-NK3R activation can lead to over stress sensitization and the consolidation of fear,6 and treatment with osanetant has been shown to block a critical fear/stress sensitization step in the brain.1,7,8  An effective therapeutic to reduce acute and persistent/long-term psychological and somatic symptoms would fulfill a large unmet need.

About Acer Therapeutics
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 investigational programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS) and post-traumatic stress disorder (PTSD); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of viruses, including cytomegalovirus, Zika, dengue, Ebola and COVID-19. For more information, visit www.www.acertx.com.

References

  1. Andero R, Dias BG, Ressler KJ. A role for Tac2, NkB, and Nk3 receptor in normal and dysregulated fear memory consolidation. Neuron. 2014;83(2):444-454
  2. Sidran Institute.  Traumatic Stress Education & Advocacy Fact Sheet.
  3. National Center for PTSD.  How Common is PTSD in Adults?
  4. Sidran Institute.  Traumatic Stress Education & Advocacy Fact Sheet.
  5. Zhang et al. Tacr3/NK3R: Beyond Their Roles in Reproduction. ACS Chemical Neuroscience 2020 11 (19), 2935-2943
  6. Al Abed et. Al, Biological Psychiatry 2021
  7. Andero R, Daniel S, Guo JD, et al. Amygdala-Dependent Molecular Mechanisms of the Tac2 Pathway in Fear Learning. Neuropsychopharmacology. 2016;41(11):2714-2722
  8. Zelikowsky M, Ding K, Anderson DJ. Neuropeptidergic Control of an Internal Brain State Produced by Prolonged Social Isolation Stress. Cold Spring Harb Symp Quant Biol. 2018;83:97-103

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 are forward-looking statements. Examples of such statements include, but are not limited to, statements about the role we believe ACER-801 could play in reducing the frequency and severity of PTSD, the planned clinical evaluation of ACER-801 for such indication, and the continued development of ACER-801 for treatment of iVMS. Our pipeline products (including ACER-801) are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. 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, the availability of financing to fund our pipeline product development programs and general corporate operations as well as risks related to drug development and the regulatory approval process, including the timing and requirements of regulatory actions. 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 Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Acer Contacts
Corporate Contact:
Jim DeNike
Acer Therapeutics Inc.
jdenike@ktharaldsenacertx-com
+1-844-902-6100

Investor contact:
Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-332-895-3226

# # #

Acer Therapeutics and Relief Therapeutics Announce Receipt of Notice of Allowance of US Patent Application Covering a Kit Comprising Phenylbutyrate and Sodium Benzoate

NEWTON, MA and GENEVA, SWITZERLAND – October 3, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER) (Acer) and RELIEF THERAPEUTICS Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (Relief) today announced that the US Patent and Trademark Office (USPTO) has issued a Notice of Allowance to Acer for US patent application No. 16/624,834 for claims related to a kit comprising a combination therapeutic product composed of sodium phenylbutyrate or glycerol phenylbutyrate and sodium benzoate. The patent application is exclusively licensed to Acer from Baylor College of Medicine.

“This Notice of Allowance expands ACER-001’s patent protection and adds an additional component to our product expansion strategy as we evaluate how to maximize its potential,” said Jeff Davis, Chief Business Officer at Acer. “The combination of phenylbutyrate and sodium benzoate was synergistic at removing ammonia in healthy subjects based on data from a study published in Genetics in Medicine in 2018.1 As a result, this combination offers the potential to use lower drug doses of each agent while maintaining equivalent ammonia removal in urea cycle disorder patients and becomes part of our lifecycle planning for ACER-001, subject to FDA approval in this indication.”

About Phenylbutyrate and Sodium Benzoate
Phenylbutyrate and sodium benzoate are nitrogen-binding agents that are used in the prevention and treatment of hyperammonemia in patients with UCDs.  Sodium benzoate for oral administration is available from compounding pharmacies and is widely used as a food preservative but has not been approved as a single agent by the U.S. Food and Drug Administration (FDA) or any regulatory authority for the treatment of UCDs. Sodium benzoate in combination with sodium phenylacetate is approved in the US and marketed as AMMONUL® (sodium phenylacetate and sodium benzoate) Injection.2 

About ACER-001
ACER-001 (sodium phenylbutyrate) is being developed for the treatment of various inborn errors of metabolism, including UCDs and Maple Syrup Urine Disease (MSUD). ACER-001 is a nitrogen-binding agent in development for use as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). ACER-001 is a polymer coated formulation that, when taken within 5 minutes, helps prevent the coating from dissolving. ACER-001 has been granted orphan drug designation by the FDA for MSUD. ACER-001 is an investigational product candidate which has not been approved by FDA, the European Medicines Agency (EMA), or any other regulatory authority. There can be no assurance that ACER-001 will be approved for any indication.

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 investigational programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of viruses, including cytomegalovirus, Zika, dengue, Ebola and COVID-19. For more information, visit www.www.acertx.com.

About RELIEF THERAPEUTICS Holding SA
Relief is a Swiss, commercial-stage, biopharmaceutical company focused on identification development and commercialization of novel, patent protected products intended for the treatment of metabolic, dermatological and pulmonary rare diseases with a portfolio of clinical and marketed assets that serve unmet patient needs.  Relief has a Collaboration and License Agreement with Acer Therapeutics for the worldwide development and commercialization of ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including UCDs and Maple Syrup Urine Disease (MSUD). The FDA has accepted for review Acer’s New Drug Application (“NDA”) resubmission under the 505(b)(2) pathway for ACER-001, for oral suspension, for the treatment of patients with UCDs. The FDA designated the NDA as a Class 2 resubmission and set a PDUFA target action date of January 15, 2023. Relief also continues to develop aviptadil for several rare pulmonary indications; Relief’s 2021 acquisitions of APR Applied Pharma Research SA and AdVita Lifescience GmbH brought to Relief a diverse pipeline of marketed and development-stage programs.

RELIEF THERAPEUTICS Holding SA is listed on the SIX Swiss Exchange under the symbol RLF and quoted in the U.S. on OTCQB under the symbols RLFTF and RLFTY. For more information, visit www.relieftherapeutics.com Follow Relief on LinkedIn.

Reference

  1. Nagamani et al. A randomized trial to study the comparative efficacy of phenylbutyrate and benzoate on nitrogen excretion and ureagenesis in healthy volunteers. Genet Med. 2018 Jul; 20(7): 708–716.
  2. https://www.accessdata.fda.gov/drugsatfda_docs/label/2005/020645lbl.pdf

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, including statements regarding the expected patent issuance date and duration, our strategy, our positioning, our products and regulatory actions are forward-looking statements. Our pipeline products are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. 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, 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 Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Relief Forward-Looking Statements
This communication expressly or implicitly contains certain forward-looking statements concerning RELIEF THERAPEUTICS Holding SA and its businesses.  Such statements involve certain known and unknown risks, uncertainties and other factors, including (i) the intellectual property protection provided by the patent discussed above, (ii) whether the FDA will approve Acer’s NDA for ACER-001 for the treatment of UCDs, (iii) whether RELIEF THERAPEUTICS Holding SA will submit an application for approval of ACER-001 in Europe for the treatment of UCDs and the timing of filing such application, (iv) whether any application submitted to European authorities seeking marketing authorization for ACER-001 for the treatment of patients in Europe with UCDs will be approved, (v) whether the FDA will approve Acer’s IND to evaluate ACER-001 for the treatment of MSUDs, (vi) the timing of Acer’s Phase 2b trial evaluating ACER-001 for the treatment of MSUDs, (vii) whether ACER-001’s currently proposed trial and any future required trials of ACER-001 for MSUDs will be undertaken and successful, (viii) whether ACER-001 will ever be approved for the treatment of MSUDs in the United States, (ix) whether Relief will ever file the necessary applications in Europe to seek the right to commercialize ACER-001 in Europe for the treatment of MSUDs and whether any such applications filed will be granted, and (x) those other risks, uncertainties and factors described in RELIEF THERAPEUTICS Holding SA’s press releases and filings with the SIX Swiss Exchange and the U.S. Securities and Exchange Commission, all of which could cause the actual results, financial condition, performance or achievements of RELIEF THERAPEUTICS Holding SA to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. RELIEF THERAPEUTICS Holding SA is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise.

CORPORATE CONTACTS
Acer Therapeutics:
Jim DeNike
Acer Therapeutics Inc.
jdenike@www.acertx.com
+1-844-902-6100

RELIEF Therapeutics Holding SA:
Jack Weinstein
Chief Financial Officer and Treasurer
contact@relieftherapeutics.com

INVESTOR RELATIONS CONTACTS
Acer Therapeutics:
Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-339-225-1047

Relief Therapeutics Holding SA:
Irina Koffler
LifeSci Advisors
ikoffler@lifesciadvisors.com
+1-917-734-7387

#  #  #

QUICK
NAV