Pyruvate Dehydrogenase Complex Deficiency Disease

NEWTON, Mass., March 27, 2023 (GLOBE NEWSWIRE) -- Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious, rare and life-threatening diseases with significant unmet medical needs, today reported financial results for Q4 and full year ended December 31, 2022, and provided an update on the Company’s recent corporate developments. “2022 marked Acer’s first U.S. Food and Drug Administration (FDA) product approval, for OLPRUVA™ (sodium phenylbutyrate), an innovative and responsibly priced treatment option for certain people with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS),” commented Chris Schelling, CEO and Founder of Acer. “We remain committed to our mission to provide transformative treatments to underserved and overlooked patients with rare and life-threatening diseases and will continue to focus our resources on launch preparation for OLPRUVA™ as well as continued pivotal Phase 3 clinical trial evaluation of EDSIVO™ for vEDS, both of which are subject to additional capital.” Q4 2022 and Recent Highlights OLPRUVA™ (sodium phenylbutyrate) for oral suspension In October 2022, Acer was issued a Notice of Allowance from the US Patent and Trademark Office (USPTO) for US patent application No. 16/624,834 (now issued as US Patent No. 11,517,547) for claims related to a kit comprising a combination therapeutic product composed of sodium phenylbutyrate or glycerol phenylbutyrate and sodium benzoateIn December 2022, Acer announced FDA approval of OLPRUVA™ (sodium phenylbutyrate) for oral suspension, a prescription medicine used along with certain therapy, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with UCDs, involving deficiencies of CPS, OTC or AS. OLPRUVA™ is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment. More information is available at www.OLPRUVA.comIn March 2023, Acer announced an update on its OLPRUVA™ U.S. commercial launch activities, including the ongoing addition of commercial and medical affairs resources, the introduction of its OLPRUVA™ Navigator by Acer Therapeutics patient support service, a predictable and responsible price commitment, and anticipated drug availability by early July 2023 (subject to additional capital)In March 2023, Acer announced results from a survey designed to quantify preferences of healthcare providers for Urea Cycle Disorders (UCDs) presented at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting. The authors concluded that optimizing nitrogen-binding medications for UCD treatment to facilitate and encourage increased patient adherence through masking taste/odor and/or enhancing other aspects of the patient experience may support improved outcomes in UCDs EDSIVO™ (celiprolol) In October 2022, Acer announced that the USPTO issued a Notice of Allowance for Acer’s patent application No. 16/930,208 and subsequently issued on December 13, 2022, as US Patent #11,523,997, for claims related to certain methods of treating vascular Ehlers-Danlos syndrome (vEDS) with celiprolol ACER-801 (osanetant) In March 2023, Acer announced that topline results from Acer’s Phase 2a proof of concept clinical trial to evaluate ACER-801 (osanetant) as a potential treatment for moderate to severe Vasomotor Symptoms (VMS) associated with menopause showed that ACER-801 was safe and well-tolerated but did not achieve statistical significance when evaluating ACER-801’s ability, when compared to placebo, to decrease the frequency or severity of hot flashes in postmenopausal women. As a result, Acer is pausing the ACER-801 program until Acer has conducted a thorough review of the full data set Corporate Ended Q4 2022 with $2.3 million in cash and cash equivalents. Acer believes its cash and cash equivalents available at December 31, 2022, together with the gross proceeds of $7.0 million from a second term loan with SWK Funding LLC which closed on January 31, 2023, $4.1 million from Acer’s ATM facility subsequent to December 31, 2022, and $2.7 million from a sale of securities (including pursuant to a registered direct offering of shares of common stock and prefunded warrants) which closed on March 24, 2023, will be sufficient to fund Acer’s anticipated operating and capital requirements into the middle of Q2 2023 Anticipated Milestones (Subject to Available Capital) Q2 2023: Acer intends to continue to add resources to establish its commercial and medical affairs presence in the U.S. in support of its OLPRUVA™ launch expected in Q2 2023 Q2 2023: Negotiations with the major commercial payers and state Medicaid organizations regarding access for OLPRUVA™ are ongoing, and representatives from Acer’s OLPRUVA™ Navigator by Acer Therapeutics patient support service are expected to begin accepting prescriptions in late Q2 2023Q2 2023: Acer expects OLPRUVA™ wholesale acquisition cost (WAC) pricing to be listed publicly in Q2 2023Early July 2023: Acer anticipates OLPRUVA™ drug availability in early July 2023Q4 2023: Acer anticipates completing enrollment in its ongoing, pivotal Phase 3 DiSCOVER trial of EDSIVO™ (celiprolol) in patients with COL3A1-positive vEDS. The double-blind portion of the DiSCOVER trial is designed to end if statistical significance is reached at an interim analysis which occurs at accrual of 28 vEDS-related events, estimated to occur as early as approximately 18 months after completion of full enrollment, or after accrual of 46 vEDS-related clinical eventsAcer intends to explore additional lifecycle opportunities for OLPRUVA™ (sodium phenylbutyrate) in various disorders where proof of concept data exists, including in Maple Syrup Urine Disease (MSUD), Pyruvate Dehydrogenase Complex Deficiency (PCDC), rare pediatric epilepsies and various liver disorders Q4 and Full Year 2022 Financial Results Cash Position. Cash and cash equivalents were $2.3 million as of December 31, 2022, compared to $12.7 million as of December 31, 2021. Acer believes its cash and cash equivalents as of December 31, 2022, together with the gross proceeds of $7.0 million from a second term loan with SWK Funding LLC which closed on January 31, 2023, $4.1 million from Acer’s ATM facility subsequent to December 31, 2022, and $2.7 million from a sale of securities (including pursuant to a registered direct offering of shares of common stock and prefunded warrants) which closed on March 24, 2023, will be sufficient to fund its currently anticipated operating and capital requirements into the middle of Q2 2023. Research and Development Expenses. Research and development expenses were $2.6 million, net of collaboration funding of $1.0 million, for the three months ended December 31, 2022, as compared to $1.7 million, net of collaboration funding of $1.3 million, for the three months ended December 31, 2021. Research and development expenses for the three months ended December 31, 2022 were comprised of $1.1 million related to ACER-001, offset by $1.0 million of collaboration funding; $1.2 million related to ACER-801; $1.0 million related to EDSIVO™; and $0.3 million related to other development activities. Research and development expenses were $11.9 million, net of collaboration funding of $7.8 million, for the year ended December 31, 2022, as compared to $6.5 million, net of collaboration funding of $6.1 million, for the year ended December 31, 2021. This increase of $5.4 million was primarily due to increases in employee-related expenses and expenses related to clinical studies, partially offset by the increase in recognition of contra-expense from the collaboration funding from Acer’s Collaboration and License Agreement with Relief Therapeutics Holding AG (Relief) for the development and commercialization of OLPRUVA™ (Collaboration Agreement). General and Administrative Expenses. General and administrative expenses were $2.6 million, net of collaboration funding of $1.2 million, for the three months ended December 31, 2022, as compared to $3.1 million, net of collaboration funding of $1.6 million, for the three months ended December 31, 2021. General and administrative expenses were $12.7 million, net of collaboration funding of $8.2 million, for the year ended December 31, 2022, as compared to $10.7 million, net of collaboration funding of $3.2 million, for the year ended December 31, 2021. This increase of $2.0 million was primarily due to increases in precommercial activities, employee-related expenses, professional services, as well as information technology, partially offset by the increase in the recognition of contra-expense from the collaboration funding from the Collaboration Agreement with Relief. Net Loss. Net loss for the three months ended December 31, 2022 was $9.4 million, or $0.54 net loss per share (basic and diluted), compared to a net loss of $4.4 million, or $0.31 net loss per share (basic and diluted), for the three months ended December 31, 2021. Net loss for the year ended December 31, 2022 was $26.2 million, or $1.66 net loss per share (basic and diluted), compared to a net loss of $15.4 million, or $1.08 net loss per share (basic and diluted), for the year ended December 31, 2021. For additional information, please see Acer’s Annual Report on Form 10-K filed today with the SEC. About Acer TherapeuticsAcer 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. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various disorders, including Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-801 (osanetant) for treatment of Vasomotor Symptoms (VMS), post-traumatic stress disorder (PTSD) and prostate cancer. For more information, visit www.acertx.com. Acer Forward-Looking StatementsThis 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 plans for the commercialization of OLPRUVA™ for oral suspension in the U.S. for the treatment of certain patients with UCDs involving deficiencies of CPS, OTC, or AS, including negotiations with commercial payers and Medicaid organizations regarding access as well as the timing of drug availability and the expected commercial launch, statements about plans and potential milestones for the continued clinical development of OLPRUVA™ in other indications, statements about plans and potential milestones for the continued clinical development of EDSIVO™ for treatment of vEDS in patients with a confirmed type III collagen (COL3A1) mutation, statements about plans for the development of ACER-801, statements about our anticipated 2023 milestones, and statements about our capital requirements and sufficiency and duration of our current cash and cash equivalents. Our efforts to commercialize OLPRUVA™ for oral suspension in the U.S. for the treatment of certain patients with UCDs involving deficiencies of CPS, OTC, or AS are at an early stage, we currently do not have fully developed marketing, sales or distribution capabilities, and there is no guarantee that we will be successful in our commercialization efforts. Our pipeline products (including OLPRUVA™ for indications other than UCDs as well as EDSIVO™ and 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 commercialization efforts, our pipeline product development programs and our 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 THERAPEUTICS INC.CONSOLIDATED STATEMENTS OF OPERATIONS Three Months Ended Years Ended December 31, December 31, 2022 2021 2022 2021 (unaudited) Revenue$— $360,000 $— $1,260,000 Operating expenses: Research and development (in the three months ended December 31, 2022 and 2021, net of collaboration funding of $999,183 and $1,288,376, respectively, and in the twelve months ended December 31, 2022 and 2021, net of collaboration funding of $7,825,263 and $6,055,295, respectively)$2,644,745 $1,705,402 $11,924,837 $6,508,055 General and administrative development (in the three months ended December 31, 2022 and 2021, net of collaboration funding of $1,186,048 and $1,636,535, respectively, and in the twelve months ended December 31, 2022 and 2021, net of collaboration funding of $8,248,813 and $3,197,659, respectively) 2,618,378 3,074,325 12,689,422 10,700,334 Total operating expenses 5,263,123 4,779,727 24,614,259 17,208,389 Loss from operations (5,263,123) (4,419,727) (24,614,259) (15,948,389) Other (expense) income, net: Costs of debt issuance (229,500) — (1,720,094) — Changes in fair value of debt instruments (loss) gain (3,846,472) — 245,138 — Interest and other income (expense), net 21,209 (2,641) (101,432) 519,639 Foreign currency transaction (loss) gain (66,444) 1,725 (46,668) 54,757 Total other (expense) income, net (4,121,207) (916) (1,623,056) 574,396 Net loss$(9,384,330) $(4,420,643) $(26,237,315) $(15,373,993) Net loss per share - basic$(0.54) $(0.31) $(1.66) $(1.08) Weighted average common shares outstanding - basic 17,359,964 14,310,244 15,767,152 14,268,245 Net loss per share - diluted$(0.54) $(0.31) $(1.66) $(1.08) Weighted average common shares outstanding - diluted 17,359,964 14,310,244 15,767,152 14,268,245 SELECTED BALANCE SHEET DATA: December 31, December 31, 2022 2021 Cash and cash equivalents $2,329,218 $12,710,762 Collaboration receivable $— $5,000,000 Prepaid expenses $759,292 $1,094,229 Deferred financing costs $408,000 $— Other current assets $20,188 $9,283,625 Property and equipment, net $214,578 $114,112 Total assets $11,624,226 $36,256,951 Deferred collaboration funding $8,412,971 $24,487,047 Original Term Loan payable, at fair value $5,567,231 $— Convertible note payable, at fair value $6,047,532 $— Total liabilities $28,385,498 $37,980,556 Total stockholders’ deficit $(16,761,272) $(1,723,605) Corporate and IR Contact Jim DeNikeAcer Therapeutics Inc.jdenike@acertx.com+1-844-902-6100 Nick ColangeloGilmartin Groupnick@gilmartinIR.com+1-332-895-3226

By signing an agreement with Medosome Biotec By providing support for clinical trial treatment of GBM at the University of Florida By starting a collaborative research project in rare pediatric cancers with a leading cancer center ROSWELL, Ga, DUBLIN and HAMILTON, Bermuda, March 16, 2023 /PRNewswire/ -- Saol Therapeutics ("Saol"), a privately held, clinical-stage pharmaceutical company, announced today the signing of an agreement with Medosome Biotec, LLC to provide Saol the rights to use their patented genotype test for all potential indications where SL-1009 might be used as a therapy. The test is performed to identify potential mutations at GSTZ1 (glutathione S-transferase zeta 1 human enzyme) that categorize individuals as fast or slow metabolizers of DCA (SL-1009). Utilization of the genotype testing enables individual dosing, potentially reducing the incidence of treatment related adverse events. Prior to this agreement Saol only had rights to the patented genotype test for the indication of Pyruvate Dehydrogenase Complex Deficiency (PDCD). "We are pleased to have executed these agreements to support our pursuit of expanded opportunities beyond PDCD" As part of the agreement Saol will aid patient recruitment efforts for the Phase IIA Trial of DCA in Glioblastoma Multiforme (GBM) by providing additional funding and resource support. Glioblastoma is aggressive and the most common type of primary brain cancer in adults. GBM is difficult to treat and there is no known cure. This study, sponsored by the University of Florida and the Food and Drug Administration (FDA) (NCT05120284), is further supported in partnership with Medosome Biotec and Saol. Dr. Peter Stacpoole is Principal Investigator for the PDCD trial and the GBM trial and is Professor of Medicine at University of Florida. Dr. Stacpoole stated, "We are pleased to expand our partnership with Saol Therapeutics. Our partnership has led to over-enrollment of our Phase III study in PDCD (NCT02616484) and we mutually see the broader potential application of DCA as the prototypic inhibitor of Pyruvate Dehydrogenase Kinases (PDKs) that, in turn, inhibit PDC enzymatic activity. We look forward to collaborating with Saol on potential additional indications for DCA. Expansion of our collaboration on the GBM trial will result in the addition of more study sites, allowing for quicker recruitment of patients. We are hopeful that DCA will be an important addition to the treatment of GBM." Saol also recently signed a sponsored research agreement with a leading cancer center to evaluate DCA in rare pediatric solid tumors. The preclinical research will investigate DCA alone and in combination with chemotherapy in several different pediatric tumor models, such as Wilms, Neuroblastoma, Rhabdoid Tumor, Osteosarcoma, Ewing Sarcoma, and Rhabdomyosarcoma. Literature suggests that DCA could be effective in several rare, pediatric, solid tumors. The rationale for the pediatric tumor screening includes the fact that DCA has been widely studied in animals and humans and has a mechanism of action that might override the Warburg effect seen in cancer cells1,2,3,4. Saol is currently studying this medicine in children using a proprietary formulation developed for pediatric use (NCT02616484). "If the pre-clinical assessment provides encouraging data, the work already conducted in pediatric patients may allow us to quickly transition toward clinical investigation", commented Dr. Virinder Nohria, Chairman and Chief Medical Officer at Saol. These agreements, and the additional investments behind them, represent an important next step in the evolution of Saol Therapeutics as an emerging pharmaceutical company. "Now that we have completed enrollment in the PDCD trial we are rapidly transitioning to other important therapeutic areas, and are evaluating the utility of DCA (SL-1009) in adult and pediatric oncology indications with limited treatment options", said Saol CEO David Penake. "We are pleased to have executed these agreements to support our pursuit of expanded opportunities beyond PDCD". Saol expects to have topline data in the PDCD trial in the 3rd quarter of 2023. Should the results of the trial support a subsequent FDA approval, it will be the first and only medicine approved for this rare, pediatric indication. References Aminzadeh S, Vidali S, Sperl W, Kofler B, Feichtinger RG. Energy metabolism in neuroblastoma and Wilms tumor. Transl Pediatr. 2015 Jan;4(1):20-32. doi: 10.3978/j.issn.2224-4336.2015.01.04. Guo JQ, Tang HY, Wang CD, Sang BT, Liu X, Yi FP, Liu GL, Wu XM. Influence of Dichloroacetate on Wilms' Tumor in vitro. Ann Clin Lab Sci. 2022 Jan;52(1):101-108. PMID: 35181623. Kankotia S, Stacpoole PW. Dichloroacetate and cancer: new home for an orphan drug? Biochim Biophys Acta. 2014 Dec;1846(2):617-29. doi: 10.1016/j.bbcan.2014.08.005. Tataranni T, Piccoli C. Dichloroacetate (DCA) and Cancer: An Overview towards Clinical Applications. Oxid Med Cell Longev. 2019 Nov 14;2019:8201079. doi: 10.1155/2019/8201079. PMID: 31827705. About Dichloroacetate (DCA; SL-1009) DCA, a pan- PDK inhibitor, has the potential to be the 1st approved medication for the mitochondrial disease PDCD and will be available as an oral solution. Gene mutations in the mitochondrial Pyruvate Dehydrogenase Complex (PDC) lead to congenital PDCD. However, PDC is also inhibited by PDKs, that may be over-expressed in PDCD. DCA inhibits PDKs to stimulate residual PDC activity and increase energy (ATP) production by mitochondria. DCA dosing is based on a proprietary genetic test that dichotomizes subjects into "fast" and "slow" drug metabolizers, providing individualized dosing. About Saol Therapeutics Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland and Hamilton, Bermuda. Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases. Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations. For more information, visit . About Medosome Biotec Medosome Biotec, LLC is a privately held, preclinical and early-stage clinical pharmaceutical company with operations in Alachua, FL and Bloomington, IN. The Company focuses on pediatric diseases with an emphasis on developing and offering genetic tests for diagnosing rare diseases and providing personalized dosing of pharmaceutical drugs. For more information, visit Media contact: Brian Nappi, Senior Vice President Strategy, bnappi@saolrx.com

NEWTON, Mass., Jan. 09, 2023 (GLOBE NEWSWIRE) -- NEWTON, MA – January 09, 2023 – 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 a corporate update and provided anticipated key development milestones for 2023. “The U.S. approval of OLPRUVA™ (sodium phenylbutyrate) for oral suspension by the U.S. Food and Drug Administration (FDA) is a culmination of Acer’s collective efforts and ongoing dedication to develop and provide new treatments to patients suffering from rare diseases. Moreover, OLPRUVA™’s approval marks a significant milestone for patients in need, offering a new, responsibly priced sodium phenylbutyrate treatment option, that will be supported by Navigator by Acer Therapeutics, our patient services program designed to support patients and caregivers,” stated Chris Schelling, CEO and Founder of Acer. “Our commitment to patients is the cornerstone of our mission, and we are proud to have secured our first FDA approval. We look forward to providing further updates in due course as the commercial launch of OLPRUVA™ progresses.” Mr. Schelling continued, “In addition to the approval of OLPRUVA™, our clinical team has been diligently executing on the clinical trials for ACER-801, being investigated in Vasomotor Symptoms (VMS), Post-traumatic Stress Disorder (PTSD) and now prostate cancer. We are eager to report the topline results from our ongoing Phase 2a trial of ACER-801 in moderate to severe VMS in post-menopausal women in Q1 of 2023. These trial results will provide important insight into ACER-801’s therapeutic potential in induced VMS (iVMS). Additionally, our pivotal Phase 3 trial of EDSIVO™ (celiprolol) in COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS) patients remains ongoing as planned, and we look forward to providing an update on full enrollment later this year.” Review of Acer’s Pipeline OLPRUVA™ (sodium phenylbutyrate) for oral suspension On December 27, 2022, Acer announced that the U.S. Food and Drug Administration (FDA) approved OLPRUVA™ (sodium phenylbutyrate) for the treatment of certain patients with UCDs involving deficiencies of CPS, OTC or ASOLPRUVA™ is a prescription medicine used along with certain therapy, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with UCDs, involving deficiencies of CPS, OTC or AS. OLPRUVA™ is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment. For additional Important Safety Information, see full Prescribing Information, Patient Information and discuss with your doctorFormation of Navigator by Acer Therapeutics: Navigator is Acer’s patient access program, a suite of integrated patient support services designed to help provide more convenient access to OLPRUVA™. The Navigator by Acer Therapeutics support services are intended to facilitate support, education, and treatment adherence to the UCD patient community ACER-801 (osanetant) Acer’s ongoing Phase 2a randomized, double-blind, placebo-controlled, dose-ranging trial of ACER-801 (osanetant) for the treatment of moderate to severe VMS in post-menopausal women is expected to readout in Q1 2023. The data from the Phase 2a trial will inform Acer’s development path for ACER-801 (osanetant) in iVMSIn October 2022, Acer announced the expansion of its ACER-801 (osanetant) program into PTSD, a disorder that affects over 12 million adults in the U.S. each year.1 Related to this program expansion, Acer licensed the worldwide exclusive rights and corresponding intellectual property portfolio from Emory University based on preclinical data that showed osanetant’s ability to block fear memory consolidation in miceConcurrent with this expansion, the US Department of Defense (DoD) awarded a $3 million grant to the University of North Carolina Institute for Trauma Recovery to support a proposed investigator-sponsored Phase 2 trial evaluating osanetant in 180 trauma patients. The randomized, placebo-controlled Phase 2 trial is currently expected to begin in Q2 2023In January 2023, Acer announced the initiation of two Phase 2, single-arm investigator-sponsored trials evaluating ACER-801 (osanetant) in men with adenocarcinoma of the prostate. The POSH-MAP (Pilot of Osanetant for Severity of Hot Flashes in Men with Adenocarcinoma of the Prostate) and PORT-MAP (Pilot of Osanetant to Reduce Testosterone in Men with Adenocarcinoma of the Prostate) trials are being sponsored and conducted by The University of Kansas Cancer Center in partnership with Acer EDSIVO™ (celiprolol) Acer’s ongoing Phase 3 DiSCOVER trial is a randomized, double-blind, placebo-controlled efficacy trial designed to evaluate EDSIVO™ (celiprolol) in patients with genetically confirmed COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS) Expected 2023 Development Milestones (Subject to Available Capital) Q1 2023: Acer expects to announce topline results from its ongoing Phase 2a randomized, double-blind, placebo-controlled, dose-ranging trial of ACER-801 (osanetant) for the treatment of moderate to severe VMS in post-menopausal womenQ2 2023: Acer expects initiation of the UNC investigator-sponsored trial to evaluate the potential for ACER-801 (osanetant) to reduce the frequency and severity of PTSD and other trauma related disordersQ4 2023: Acer anticipates completing enrollment in its ongoing, pivotal Phase 3 DiSCOVER trial of EDSIVO™ (celiprolol) in patients with COL3A1-positive vEDS. The double-blind portion of DiSCOVER trial is intended to end if statistical significance is reached at an interim analysis which occurs at accrual of 28 vEDS-related events, estimated to occur as early as approximately 18 months after completion of full enrollment, or after accrual of 46 vEDS-related clinical eventsAcer intends to explore additional lifecycle opportunities for OLPRUVA™ (sodium phenylbutyrate) in various disorders where proof of concept data exists, including in Maple Syrup Urine Disease (MSUD), Pyruvate Dehydrogenase Complex Deficiency (PCDC), rare pediatric epilepsies and various liver disorders About Acer TherapeuticsAcer 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. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various disorders, including Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS), Post-traumatic Stress Disorder (PTSD) and prostate cancer; 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 National Center for PTSD. How Common is PTSD in Adults? Acer Forward-Looking StatementsThis 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, the continued development of ACER-801 for treatment of iVMS and our expected 2023 milestones. 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. Corporate and IR ContactJim DeNikeAcer Therapeutics Inc.jdenike@acertx.com+1-844-902-6100 Nick ColangeloGilmartin Groupnick@gilmartinIR.com+1-332-895-3226