AcerUser, Author at Acer Therapeutics

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

Posted on October 6, 2022October 6, 2022 by AcerUser

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.acertx.com.

References

Sidran Institute. Traumatic Stress Education & Advocacy Fact Sheet.
National Center for PTSD. How Common is PTSD in Adults?
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
Thompson 2015. Unlocking the secrets of PTSD. Time 2015;185:40–43
Zhang et al. ACS Chemical Neuroscience 2020 11 (19), 2935-2943
Al Abed et. Al, Biological Psychiatry 2021
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
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
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 Contacts

Corporate Contact:
Jim DeNike
Acer Therapeutics Inc.
jdenike@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

Posted on October 5, 2022October 5, 2022 by AcerUser

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.

References

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
Sidran Institute. Traumatic Stress Education & Advocacy Fact Sheet.
National Center for PTSD. How Common is PTSD in Adults?
Sidran Institute. Traumatic Stress Education & Advocacy Fact Sheet.
Zhang et al. Tacr3/NK3R: Beyond Their Roles in Reproduction. ACS Chemical Neuroscience 2020 11 (19), 2935-2943
Al Abed et. Al, Biological Psychiatry 2021
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
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 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

Posted on October 3, 2022October 3, 2022 by AcerUser

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.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

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.
https://www.accessdata.fda.gov/drugsatfda_docs/label/2005/020645lbl.pdf

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@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

# # #

Acer Therapeutics to Participate in August and September 2022 Investor Conferences

Posted on August 23, 2022August 23, 2022 by AcerUser

NEWTON, MA – August 23, 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 that Acer’s management team will virtually present at, and participate in, the upcoming Gilmartin Group Emerging Growth Company Showcase and H.C. Wainwright 24^th Annual Global Investment Conference.

Conference: Gilmartin Group Emerging Growth Company Showcase
Format: Recorded corporate presentation
Date and Time: Recorded presentation available at 10:30 am ET, August 31, 2022
Register: https://gilmartin-emerging-growth-conference.open-exchange.net/registration
Webcast: https://kvgo.com/corporate-services/acer-therapeutics-aug-2022

Conference: H.C. Wainwright 24^th Annual Global Investment Conference
Format: On-demand recorded corporate presentation and one-on-one virtual meetings
Date and Time: On-demand corporate presentation available beginning at 7 am ET, September 12, 2022
Webcast: https://journey.ct.events/view/09133963-6072-4f07-864c-3ed9b50db443

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.acertx.com.

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

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

Acer Therapeutics Reports Q2 2022 Financial Results and Provides Corporate Update

Posted on August 15, 2022August 15, 2022 by AcerUser

NEWTON, MA – August 15, 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 reported financial results for the second quarter ended June 30, 2022, and provided a corporate update.

“I am very pleased with our team’s quick turnaround of our New Drug Application (NDA) resubmission following receipt of the Complete Response Letter (CRL) in response to our ACER-001 NDA for urea cycle disorders (UCDs),” stated Chris Schelling, CEO and Founder of Acer. “We have already announced the U.S. Food & Drug Administration (FDA) has accepted our NDA resubmission for ACER-001 in UCDs, assigned a new PDUFA date of January 15, 2023, and we are awaiting the agency’s inspection of our third-party contract packaging manufacturer. Meanwhile, we have made additional progress on our ACER-001 program, including the submission of an Investigational New Drug (IND) application for clinical investigation in Maple Syrup Urine Disease (MSUD) patients, and the issuance of a key utility patent in China.”

Schelling continued, “In parallel to our efforts in support of ACER-001 development, we also initiated in the second quarter, our Phase 3 pivotal DiSCOVER trial of EDSIVO™ for the treatment of vascular Ehlers-Danlos Syndrome (vEDS) under our Special Protocol Assessment (SPA) agreement with the FDA. Looking ahead, we anticipate topline results in our Phase 2a clinical trial of ACER-801 for the treatment of moderate to severe Vasomotor Symptoms (VMS) in post-menopausal women in Q4 of 2022.”

Q2 2022 and Recent Highlights

ACER-001 (sodium phenylbutyrate) for oral suspension
- In April and May 2022, data was presented evaluating the bioavailability, bioequivalence and taste attributes of ACER-001 compared to sodium phenylbutyrate (BUPHENYL®) powder during poster sessions at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting and the Genetic Metabolic Dieticians International (GMDI) Conference, respectively
- In May 2022, Acer launched SeeUCDifferently, a U.S. disease awareness campaign intended to provide education and information about UCDs, including www.SeeUCDifferently.com, a new online resource that provides general UCDs disease education information
- In July 2022, Acer resubmitted its NDA for ACER-001 to the FDA following receipt of a CRL in June 2022, in which the FDA stated that satisfactory inspection of Acer’s third-party contract packaging manufacturer is required before the ACER-001 NDA may be approved. Acer notified the FDA in the NDA resubmission that its third-party contract packaging manufacturer is ready for inspection and is currently awaiting completion of this requirement
- In July 2022, the FDA accepted Acer’s NDA resubmission (Class 2) and assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 15, 2023
- In July 2022, the China National Intellectual Property Administration (CNIPA) issued a utility model patent for ACER-001 covering dosage form claims related to ACER-001’s polymer coated formulation, with an expiration date of August 24, 2031
- In July 2022, Acer submitted its IND application to the FDA for ACER-001 for the potential treatment of MSUD. The proposed Phase 2a, open-label dose-ranging clinical trial will evaluate the treatment effect of different doses of ACER-001 on blood leucine and other branched-chain amino acid (BCAA) levels in MSUD patients
- In August 2022, the European Commission granted orphan medicinal product designation in the EU to ACER-001 for the potential treatment of MSUD
EDSIVO™ (celiprolol)
- In April 2022, the FDA granted celiprolol Breakthrough Therapy designation (BTD) in the U.S. for the treatment of patients with COL3A1-positive vEDS
- In May 2022, confirmed agreement with the FDA under a SPA for a pivotal Phase 3 clinical trial of celiprolol for the treatment of patients with COL3A1-positive vEDS
- In June 2022, initiated a pivotal Phase 3, randomized, double-blind, placebo-controlled, decentralized clinical (DiSCOVER) trial for celiprolol in patients with COL3A1-positive vEDS under Acer’s SPA agreement with the FDA
Corporate
- In January 2022, promoted Bernie Paul to Chief People Officer. Mr. Paul has over 25 years of experience in human resources, having served most recently as Vice President of Human Resources at Acer. Over his career he has played a major role in developing organizational cultures within various companies assisting them in scaling through different stages of growth while achieving significant milestones including initial public offerings, acquisition integrations and product commercialization
- In June 2022, promoted Tanya Hayden as Chief Operating Officer. Ms. Hayden has extensive experience in operational excellence and is tasked with commercial and operational readiness for a potential commercial approval and launch of ACER-001 in UCDs. Prior to joining Acer, Ms. Hayden spent 20 years at Lonza, where she was responsible for business unit planning, clinical and commercial contract manufacturing, as well as portfolio program management
- Ended Q2 2022 with $14.5 million in cash and cash equivalents, which Acer believes will be sufficient to fund its currently anticipated operating and capital requirements through Q3 2022

Anticipated Milestones

ACER-001 (sodium phenylbutyrate)
- January 15, 2023: The FDA has assigned a new PDUFA target action date of January 15, 2023, following its acceptance for review of Acer’s resubmitted NDA for ACER-001 for the treatment of patients with UCDs. Acer notified the FDA in the NDA resubmission that its third-party contract packaging manufacturer is ready for inspection and is awaiting completion of this requirement
- Q1 2023: Acer plans to initiate in Q1 2023 its Phase 2a trial to evaluate the efficacy and safety of ACER-001 for the potential treatment of patients with MSUD, subject to IND clearance and available capital
ACER-801 (osanetant)
- Q4 2022: Acer expects to announce topline results in Q4 2022 from its ongoing Phase 2a randomized, double-blind, placebo-controlled, dose-ranging trial of ACER-801 for the treatment of moderate to severe VMS in post-menopausal women, subject to additional capital
EDSIVO™ (celiprolol)
- Mid-2023: Based on current enrollment rates, Acer anticipates the DiSCOVER trial for celiprolol in patients with COL3A1-positive vEDS will be fully enrolled in mid-2023. Once 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 beyond Q3 2022. One interim analysis (based on the number of primary events occurring) is also planned at approximately 24 months after full enrollment

Q2 2022 Financial Results

Cash position. Cash and cash equivalents were $14.5 million as of June 30, 2022, compared to $12.7 million as of December 31, 2021. Acer believes its cash and cash equivalents available as of June 30, 2022 will be sufficient to fund its currently anticipated operating and capital requirements through Q3 2022.

Research and Development Expenses. Research and development expenses were $3.4 million, net of collaboration funding of $1.6 million, for the three months ended June 30, 2022, as compared to $1.4 million, net of collaboration funding of $1.0 million, for the three months ended June 30, 2021. This increase of $2.0 million was primarily due to increases in expenses for clinical studies. Research and development expenses for the three months ended June 30, 2022 were comprised of $2.1 million related to ACER-801; $1.8 million related to ACER-001, offset by $1.6 million of collaboration funding; $0.8 million related to EDSIVOä; and $0.3 million related to other development activities.

General and Administrative Expenses. General and administrative expenses were $3.6 million, net of collaboration funding of $3.3 million, for the three months ended June 30, 2022, as compared to $2.3 million, net of collaboration funding of $0.6 million, for the three months ended June 30, 2021. This increase of $1.3 million was primarily due to increases in precommercial expenses, audit and consulting fees, and employee-related expenses, partially offset by a decrease in the recognition of the collaboration funding from the Collaboration Agreement with Relief.

Net Loss. Net loss for the three months ended June 30, 2022 was $2.7 million, or $0.17 net loss per share (basic and diluted), compared to a net loss of $3.1 million, or $0.21 net loss per share (basic and diluted), for the three months ended June 30, 2021.

For additional information, please see Acer’s Quarterly Report on Form 10-Q filed today with the SEC.

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

INVESTOR RELATIONS CONTACT
Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-332-895-3226

# # #

Relief Therapeutics and Acer Therapeutics Announce that the European Commission Has Granted Orphan Drug Designation for ACER-001 in Maple Syrup Urine Disease

Posted on August 12, 2022August 12, 2022 by AcerUser

GENEVA, SWITZERLAND and NEWTON, MA – August 12, 2022 – RELIEF THERAPEUTICS Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (Relief), and its collaboration partner, Acer Therapeutics Inc. (Nasdaq: ACER) (Acer), today announced that the European Commission has granted orphan medicinal product designation in the EU to ACER-001 (sodium phenylbutyrate) for the potential treatment of patients with Maple Syrup Urine Disease (MSUD). ACER-001 was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) in the U.S. for MSUD in 2014.

“Orphan designation by the European Commission is another important milestone for the ACER-001 program that provides further validation of the important role we believe ACER-001 will play in the potential treatment of multiple rare diseases,” stated Raghuram (Ram) Selvaraju, Ph.D., Chairman of the Board of Directors of Relief. “We look forward to the continued advancement of ACER-001 in MSUD and Urea Cycle Disorders.”

“We are very pleased to receive orphan medicinal product designation in both the U.S. and now the EU, underscoring the urgent need for an approved treatment for MSUD,” said Adrian Quartel, MD, FFPM, Chief Medical Officer of Acer. “Currently, the only treatment option for patients with MSUD is a lifelong, protein-restricted diet, however, they still remain at serious risk for a wide range of life-threatening complications.”

Orphan drug designation is granted to medicines that treat or prevent a life-threatening or chronically debilitating rare disease, with a prevalence in the EU of not more than 5 in 10,000, and with either no currently approved method of prevention or treatment, or with significant benefit to those affected by the disease. The designation potentially provides certain benefits to ACER-001, including the potential for up to 10-year EU market exclusivity upon regulatory approval, if received, reductions in EMA application fees, and access to study protocol assistance.

Rationale for ACER-001 Treatment in MSUD
Multiple investigational trials evaluating sodium phenylbutyrate in urea cycle disorder (UCD) patients suggest treatment with sodium phenylbutyrate is associated with selective reduction in branched chain amino acids (BCAA) despite adequate restricted dietary protein intake.^1,2,3,4 Analysis of data from a longitudinal multicenter study of 553 UCD patients treated with sodium phenylbutyrate demonstrated that sodium phenylbutyrate decreased plasma BCAA in patients with UCDs and could serve as a therapy in maple syrup urine disease and other common complex disorders with dysregulation of BCAA metabolism.²

Based on this clinical observation, investigators at Baylor College of Medicine explored the potential of sodium phenylbutyrate treatment to lower BCAA and corresponding branched-chain α-ketoacid (BCKA) levels in both healthy subjects and patients with MSUD. The investigators found that sodium phenylbutyrate, when dosed over three days, showed a statistically significant reduction of leucine in all three healthy subjects and in three out of the five MSUD patients who participated in the trial.⁵

In November 2020, study results evaluating the effect of sodium phenylbutyrate in the management of acute metabolic decompensation in pediatric MSUD patients (n=10) were published by investigators from Istanbul University-Cerrahpasa Medical Faculty in the peer-reviewed Journal of Pediatric Endocrinology and Metabolism showing a significant reduction in leucine levels in MSUD patients experiencing an acute attack.⁶ The results suggested that sodium phenylbutyrate could be safely administered in combination with emergency protocol using other active pharmaceuticals and supports additional investigation of potential clinical benefit beyond emergency protocol alone.

About MSUD
MSUD is a rare inherited disorder caused by a deficiency of branched-chain alpha-keto acid dehydrogenase complex, resulting in elevated blood levels of the (BCAA) leucine, valine, and isoleucine, as well as the associated (BCKA) in a patient’s blood. Left untreated, this can result in neurological damage, mental disability, coma, or death. The most severe presentation of MSUD, known as “classic” MSUD, accounts for 80% of cases and can result in neonatal onset with encephalopathy and coma. Although metabolic management of the disease is possible via a highly restrictive diet, the outcome is unpredictable, and a significant portion of affected individuals are mentally impaired or experience neurological complications.

MSUD is typically diagnosed at birth via newborn screening and incidence is estimated at 1 in 185,000 people worldwide and 1 in 220,000 people in the United States.⁷ The disorder occurs more frequently in the Old Order Mennonite population, with an estimated incidence of about 1 in 380 newborns, and the Ashkenazi Jewish population, with an estimated incidence of 1 in 26,000.⁸

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 (sodium phenylbutyrate) is an immediate-release, polymer coated, multi-particulate formulation of sodium phenylbutyrate for oral administration via suspension, that is designed to improve palatability. ACER‑001 (sodium phenylbutyrate) has been granted orphan drug designation by the FDA for MSUD. In July 2022, the FDA accepted Acer’s NDA resubmission (Class 2) and assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 15, 2023. This investigational product candidate has not been approved by FDA, the European Medicines Agency (EMA), or any other regulatory authority. There can be no assurance that the resubmitted ACER-001 NDA for UCDs will be approved by the FDA, or that ACER-001 (sodium phenylbutyrate) will otherwise be approved for any indication.

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). 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.

References

Muelly 2011 Neuropsychiatric and Neurochemical Sequelae of MSUD.
L.C. Burrage, et al., Sodium phenylbutyrate decreases plasma branched-chain amino acids in patients with urea cycle disorders, Mol. Genet. Metab. (2014)
Scaglia F. New insights in nutritional management and amino acid supplementation in urea cycle disorders. Mol Genet Metab. 2010;100 Suppl 1(Suppl 1):S72-6.
Häberle, J., Boddaert, N., Burlina, A. et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet J Rare Dis 7, 32 (2012)
Brunetti-Pierri et al. Phenylbutyrate therapy for maple syrup urine disease. Hum Mol Genet. 2011 February 15; 20(4): 631–640.
Zubarioglu T, et al. Impact of sodium phenylbutyrate treatment in acute management of maple syrup urine disease attacks: a single-center experience. J Pediatr Endocrinol Metab. 2020 Nov 11;34(1):121-126.
Chapman, K, et al. (2018). Incidence of maple syrup urine disease, propionic acidemia, and methylmalonic aciduria from newborn screening data. Molecular Genetics and Metabolism Reports. 15. 106-109.
Strauss KA, et al. Maple Syrup Urine Disease. In: Pagon RA, Adam MP, Ardinger HH, al. e, eds. GeneReviews® [Internet]. https://www.ncbi.nlm.nih.gov/books/NBK1319/: University of Washington, Seattle; 2006. Accessed March 22, 2017

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) whether the FDA will approve Acer’s NDA for ACER-001 for the treatment of UCDs, (ii) 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, (iii) 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, (iv) whether the FDA will approve Acer’s IND to evaluate ACER-001 for the treatment of MSUDs, (v) the timing of Acer’s Phase 2b trial evaluating ACER-001 for the treatment of MSUDs, (vi) whether ACER-001’s currently proposed trial and any future required trials of ACER-001 for MSUDs will be undertaken and successful, (vii) whether ACER-001 will ever be approved for the treatment of MSUDs in the United States, (viii) 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 (ix) 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

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

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

INVESTOR RELATIONS CONTACTS

Relief Therapeutics Holding SA:
Michael Miller
Rx Communications Group
mmiller@rxir.com
+1-917-633-6086

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

# # #

Acer Therapeutics and Relief Therapeutics Announce ACER-001 IND Submission for the Treatment of Maple Syrup Urine Disease

Posted on July 28, 2022July 28, 2022 by AcerUser

NEWTON, MA and GENEVA, SWITZERLAND – July 28, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER) (Acer) and its collaboration partner, RELIEF THERAPEUTICS Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (Relief), today announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to evaluate the efficacy and safety of ACER-001 (sodium phenylbutyrate) for the potential treatment of patients with Maple Syrup Urine Disease (MSUD).

MSUD is a rare, life-threatening metabolic disorder caused by a deficiency in an enzyme complex that metabolizes branched chain ketoacids, the breakdown products of the three branched-chain amino acids (BCAAs), leucine, valine, and isoleucine. Left untreated, MSUD leads to elevated plasma concentrations of these amino acids, which can lead to chronic and acute neurological damage, ranging from developmental delays in children, seizures, cognitive challenges and in some cases death. Currently, the only treatment option for patients with MSUD is a life-long, protein-restricted diet.

“We are very pleased to expand ACER-001’s clinical development into a second rare disease, and one for which there are currently no approved pharmacologic therapies,” said Adrian Quartel, MD, FFPM, Chief Medical Officer of Acer. “Even with strict dietary management, people with MSUD still remain at serious risk for a wide range of life-threatening complications. We look forward to the initiation of this investigational trial and learning more about ACER-001’s potential to reduce branched-chain amino acids, and specifically leucine levels, in MSUD patients.”

The proposed initial Phase 2a, open-label dose-ranging trial is designed to evaluate the effect of different doses of ACER-001 (sodium phenylbutyrate) on blood leucine and other branched-chain amino acid (BCAA) levels in MSUD patients.

Rationale for ACER-001 Treatment in MSUD
Multiple investigational trials evaluating sodium phenylbutyrate in urea cycle disorder (UCD) patients suggest treatment with sodium phenylbutyrate is associated with selective reduction in BCAA despite adequate dietary protein intake.^1,2,3,4 Analysis of data from a longitudinal multicenter study of 553 UCD patients treated with sodium phenylbutyrate demonstrated that sodium phenylbutyrate decreased plasma BCAA in patients with UCDs and could serve as a therapy in maple syrup urine disease and other common complex disorders with dysregulation of BCAA metabolism.²

In November 2020, study results evaluating the effect of sodium phenylbutyrate in the management of acute metabolic decompensation in pediatric MSUD patients (n=10) were published by investigators from Istanbul University-Cerrahpasa Medical Faculty in the peer-reviewed Journal of Pediatric Endocrinology and Metabolism showing a significant reduction in leucine levels in MSUD patients experiencing an acute attack.⁶ The results suggested that sodium phenylbutyrate could be safely administered in combination with emergency protocol using other active pharmaceuticals and may provide additional clinical benefit beyond emergency protocol alone.

About MSUD
MSUD is a rare inherited disorder caused by a deficiency of branched-chain alpha-keto acid dehydrogenase complex, resulting in elevated blood levels of the branched-chain amino acids (BCAA) leucine, valine, and isoleucine, as well as the associated branched-chain ketoacids (BCKA) in a patient’s blood. Left untreated, this can result in neurological damage, mental disability, coma, or death. The most severe presentation of MSUD, known as “classic” MSUD, accounts for 80% of cases and can result in neonatal onset with encephalopathy and coma. Although metabolic management of the disease is possible via a highly restrictive diet, the outcome is unpredictable, and a significant portion of affected individuals are mentally impaired or experience neurological complications.

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 (sodium phenylbutyrate) is an immediate-release, polymer coated, multi-particulate formulation of sodium phenylbutyrate for oral administration via suspension, that is designed to improve palatability. ACER‑001 (sodium phenylbutyrate) has been granted orphan drug designation by the FDA for MSUD. In July 2022, Acer resubmitted its New Drug Application (NDA) to the FDA for ACER-001 (sodium phenylbutyrate) for oral suspension for the treatment of patients with UCDs in response to the FDA’s Complete Response Letter. This investigational product candidate has not been approved by FDA, the European Medicines Agency (EMA), or any other regulatory authority. There can be no assurance that the resubmitted ACER-001 NDA for UCDs will be approved by the FDA, or that ACER-001 (sodium phenylbutyrate) will otherwise be approved for any indication.

About RELIEF THERAPEUTICS Holding SA
Relief focuses primarily on clinical-stage programs based on molecules with a history of clinical testing and use in human patients or a strong scientific rationale. 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). Relief also continues to study aviptadil for several possible lung related conditions. Finally, 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.

References

Muelly 2011 Neuropsychiatric and Neurochemical Sequelae of MSUD.
L.C. Burrage, et al., Sodium phenylbutyrate decreases plasma branched-chain amino acids in patients with urea cycle disorders, Mol. Genet. Metab. (2014)
Scaglia F. New insights in nutritional management and amino acid supplementation in urea cycle disorders. Mol Genet Metab. 2010;100 Suppl 1(Suppl 1):S72-6.
Häberle, J., Boddaert, N., Burlina, A. et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet J Rare Dis 7, 32 (2012)
Brunetti-Pierri et al. Phenylbutyrate therapy for maple syrup urine disease. Hum Mol Genet. 2011 February 15; 20(4): 631–640.
Zubarioglu T, et al. Impact of sodium phenylbutyrate treatment in acute management of maple syrup urine disease attacks: a single-center experience. J Pediatr Endocrinol Metab. 2020 Nov 11;34(1):121-126.
Chapman, K, et al. (2018). Incidence of maple syrup urine disease, propionic acidemia, and methylmalonic aciduria from newborn screening data. Molecular Genetics and Metabolism Reports. 15. 106-109.
Strauss KA, et al. Maple Syrup Urine Disease. In: Pagon RA, Adam MP, Ardinger HH, al. e, eds. GeneReviews® [Internet]. https://www.ncbi.nlm.nih.gov/books/NBK1319/: University of Washington, Seattle; 2006. Accessed March 22, 2017

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) whether the FDA will approve Acer’s NDA for ACER-001 for the treatment of UCDs, (ii) 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, (iii) 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, (iv) whether the FDA will approve Acer’s IND to evaluate ACER-001 for the treatment of MSUDs, (v) the timing of Acer’s Phase 2b trial evaluating ACER-001 for the treatment of MSUDs, (vi) whether ACER-001’s currently proposed trial and any future required trials of ACER-001 for MSUDs will be undertaken and successful, (vii) whether ACER-001 will ever be approved for the treatment of MSUDs in the United States, (viii) 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 (ix) 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@ktharaldsenacertx-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
+1-332-895-3226

Relief Therapeutics Holding SA:
Michael Miller
Rx Communications Group
mmiller@rxir.com
+1-917-633-6086

# # #

Acer Therapeutics and Relief Therapeutics Announce FDA Acceptance for Review of NDA Resubmission for ACER-001 for Treatment of UCDs

Posted on July 28, 2022July 28, 2022 by AcerUser

NEWTON, MA and GENEVA, SWITZERLAND – July 28, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER) (Acer) and its collaboration partner, RELIEF THERAPEUTICS Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (Relief), today announced the U.S. Food and Drug Administration (FDA) has accepted for review Acer’s resubmitted New Drug Application (NDA) for ACER-001 (sodium phenylbutyrate) for oral suspension for the treatment of patients with urea cycle disorders (UCDs). The FDA designated the NDA as a Class 2 resubmission and set a Prescription Drug User Fee Act (PDUFA) target action date of January 15, 2023.

“We are very pleased to receive confirmation that the FDA is commencing its review of our NDA resubmission,” said Chris Schelling, Founder & Chief Executive Officer of Acer. “We have notified the FDA that our third-party contract packaging manufacturer is ready for inspection and are hopeful that the Agency’s review can be completed in a timely manner. While NDA approval is not assured, if approval is received, we are prepared to execute on our comprehensive launch plan and provide a new treatment option to underserved UCDs patients. These efforts reinforce our ongoing commitment to developing new and differentiated treatment options for those affected by rare diseases.”

In June 2022, as previously announced, the FDA issued Acer a Complete Response Letter (CRL) stating that satisfactory inspection of Acer’s third-party contract packaging manufacturer is required before the ACER-001 NDA may be approved. Acer notified the FDA in the NDA resubmission that the third-party contract packaging manufacturer is ready for inspection. The FDA did not cite any other approvability issues in the CRL pertaining to the NDA, nor request any additional clinical or pharmacokinetic studies be conducted prior to FDA action. Additional existing nonclinical information as requested by the FDA in the CRL but identified as “not an approvability issue”, as well as labeling and other required updates to the original NDA, were provided in the resubmission of the NDA.

There can be no assurance that ACER-001 will be approved for any indication.

CORPORATE CONTACTS

Acer Therapeutics:
Jim DeNike
Acer Therapeutics Inc.
jdenike@ktharaldsenacertx-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
+1-332-895-3226

Relief Therapeutics Holding SA:
Michael Miller
Rx Communications Group
mmiller@rxir.com
+1-917-633-6086

# # #

Acer Therapeutics Announces Resubmission of New Drug Application for ACER-001 for Treatment of UCDs

Posted on July 18, 2022July 18, 2022 by AcerUser

NEWTON, MA – July 18, 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 resubmission of Acer’s New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for ACER-001 (sodium phenylbutyrate) for oral suspension for the treatment of patients with urea cycle disorders (UCDs). Acer believes the resubmission addresses in full the items raised by the FDA in the Complete Response Letter (CRL).

In June 2022, as previously announced, the FDA issued Acer a CRL stating that satisfactory inspection of its third-party contract packaging manufacturer is required before the ACER-001 NDA may be approved. Acer notified the FDA in the resubmission that the third-party contract packaging manufacturer is ready for inspection. FDA did not cite any other approvability issues in the CRL pertaining to the NDA, nor request any additional clinical or pharmacokinetic studies be conducted prior to FDA action. Additional existing nonclinical information as requested by the FDA in the CRL but identified as “not an approvability issue”, as well as labeling and other routine updates to the original NDA, were provided in the resubmission of the NDA.

“Our team did an outstanding job resubmitting the NDA one month after we received the CRL,” said Chris Schelling, Founder & Chief Executive Officer of Acer. “Our third-party contract manufacturing partner has been incredibly responsive and has confirmed that it is ready for inspection. Our manufacturing partner is regarded as a global leader in clinical supply chain and commercial packaging services with more than 70 years of experience. Along with the other requested updates we provided to the FDA in our resubmitted NDA, assuming it is accepted for review, we now look forward to assisting the FDA in the completion of its review of our NDA as soon as possible.”

Acer expects to be notified by the Agency of its decision to accept or reject the resubmission for review within 14 calendar days of receipt of the NDA resubmission.¹ If the resubmission is deemed complete by the FDA, a resubmission classification (Class 1 or 2) will be assigned and a new Prescription Drug User Fee Act (PDUFA) target action date will be established of either two or six months from the resubmission date depending on the classification and an inspection of the facility will be requested. However, there can be no assurance the resubmission will be accepted for review, classified for action, or the target action deadline will be established or met.

References

FDA SOPP 8405.1: Procedures for Resubmissions to an Application or Supplement Version: 7 Effective Date: November 1, 2021 https://www.fda.gov/media/84417/download

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

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

# # #

Acer Therapeutics and Relief Therapeutics Announce That the China National Intellectual Property Administration (CNIPA) Issued Utility Model Patent 11,202,767 Covering ACER-001 Dosage Form

Posted on July 8, 2022July 8, 2022 by AcerUser

NEWTON, MA and GENEVA, SWITZERLAND – July 8, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER) (Acer) and its collaboration partner, RELIEF THERAPEUTICS Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (Relief), today announced that the China National Intellectual Property Administration (CNIPA) issued Electronic Patent Certificate ZL202122004991.9 on May 24, 2022, for Utility Model directed to ACER-001 (sodium phenylbutyrate). Specifically, the newly issued patent covers dosage form claims related to ACER-001’s polymer coated formulation for oral administration as a potential treatment for urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD). The newly issued patent has an expiration date of August 24, 2031.

“The receipt of this patent from the CNIPA for ACER-001, is an important milestone for Acer and Relief, adding key protection to the growing intellectual portfolio of this coated formulation of sodium phenylbutyrate, which could be an important treatment for patients with UCDs and we hope to extend its use as a potential treatment for MSUD and other indications,” stated Raghuram (Ram) Selvaraju, Chairman of Relief. “The issuance of this patent also marks an important step in our pursuit of possible ACER-001 commercialization in China.”

About RELIEF
Relief focuses primarily on clinical-stage programs based on molecules with a history of clinical testing and use in human patients or a strong scientific rationale. 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). Relief also continues to study aviptadil for several possible lung related conditions. Finally, 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.

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 for clinical study enrollment or regulatory actions, or otherwise, 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 investigational product candidates to safely and effectively treat diseases and to be approved for marketing; our ability to submit regulatory filings (including for ACER-001 for oral suspension for the treatment of patients with UCDs) within intended timeframes, to address satisfactorily the requirements for regulatory approval (including through FDA inspection of our third-party contract packaging manufacturer for ACER-001 for oral suspension for the treatment of patients with UCDs), or to otherwise address satisfactorily the requirements for and to obtain regulatory approval (including approval by the FDA of a resubmitted NDA for ACER-001 for oral suspension for the treatment of patients with UCDs); our ability to close upon and obtain the proceeds of any financing arrangements as well as to satisfy the ongoing conditions and requirements for maintaining the financing facilities and avoiding default or an accelerated payment requirement; 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 for ACER-001, ACER-801, EDSIVO™ or our other product candidates; 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. 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.

CORPORATE CONTACTS

Acer Therapeutics:
Jim DeNike
Acer Therapeutics Inc.
jdenike@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:
Michael Miller
Rx Communications Group
mmiller@rxir.com
+1-917-633-6086

# # #