March 2023 - Acer Therapeutics

Acer Therapeutics Reports Q4 and Full Year 2022 Financial Results and Provides Corporate Update

Posted on March 27, 2023March 27, 2023 by AcerUser

NEWTON, MA – March 27, 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 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 benzoate
- In 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.com
- In 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 2023
Q2 2023: Acer expects OLPRUVA™ wholesale acquisition cost (WAC) pricing to be listed publicly in Q2 2023
Early July 2023: Acer anticipates OLPRUVA™ drug availability in early July 2023

Q4 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 events
Acer 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 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. 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.www.acertx.com.

Acer Forward-Looking Statements This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release are forward-looking statements. Examples of such statements include, but are not limited to, statements about 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.

Corporate and IR Contact

Jim DeNike
Acer Therapeutics Inc.
[email protected]
+1-844-902-6100

Nick Colangelo
Gilmartin Group
[email protected]
+1-332-895-3226

Acer Therapeutics Announces Data Presented at Society for Inherited Metabolic Disorders Annual Meeting Identifying Preferred Urea Cycle Disorder Treatment Attributes

Posted on March 22, 2023March 22, 2023 by AcerUser

NEWTON, MA – March 22, 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 data was presented from a survey designed to quantify preferences of healthcare providers for Urea Cycle Disorders (UCDs) at the 44^th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD), March 18^th-21^st in Salt Lake City, Utah. Results from the discrete choice experiment presented showed that taste and odor were the most important attributes, as identified by UCD healthcare providers, that influence overall prescription of, and patient adherence to, UCD treatments when evaluating nitrogen-binding medications (such as sodium phenylbutyrate and glycerol phenylbutyrate).

“Nitrogen-binding medications, such as sodium phenylbutyrate or glycerol phenylbutyrate, can be efficacious in the treatment of UCDs if patients are adherent to their prescribed treatment^1,2,” stated Robert Steiner, M.D., Professor at the University of Wisconsin School of Medicine and Public Health. “However, 25% of life-threatening hyperammonemic crises in patients with UCDs may be precipitated by a lack of adherence to medications and/or diet and certain attributes of existing nitrogen-binding medications may negatively impact adherence³. Given these results, alternative treatment options are urgently needed.”

Healthcare Provider UCD Treatment Preference Data Presented at SIMD 2023
A copy of the poster presentation from the 2023 SIMD Meeting is available on Acer’s website at: https://www.acertx.com/wp-content/uploads/2023/03/2023-SIMD-DCE-Poster.pdf

Poster #97: Quantifying Preferences for Urea Cycle Disorder Treatments Using a Discrete-Choice Experiment⁴

This poster presented at SIMD summarizes results from a web-based, quantitative survey study conducted on Acer’s behalf, using a discrete choice experiment (DCE) methodology. This study was designed to quantify the most-desired product attributes that may influence overall prescription of, and patient adherence to, nitrogen-binding medications (such as sodium phenylbutyrate and glycerol phenylbutyrate) for the treatment of UCDs as identified by the survey participants.

Of the 51 healthcare providers that completed the survey, most HCPs reported dissatisfaction with current treatment options [mean rating (SD)=5.4 (1.7); Likert scale with 1 = not at all satisfied through 9 = extremely satisfied]. The results of the survey showed that taste and odor were the most important attributes for both prescribing and for patient adherence and compliance . The authors concluded that optimizing nitrogen-binding medications for UCD treatment to facilitate and encourage increased patient adherence through masking taste and odor, and/or enhancing other aspects of the patient experience, may support improved outcomes in UCDs.

“OLPRUVA™⁵ leverages the well-established efficacy of sodium phenylbutyrate in an innovative dual-coating formulation designed for palatability,” said Adrian Quartel, MD, FFPM, CMO of Acer. “We are pleased to offer an innovative, responsibly priced treatment option to patients with UCDs and remain committed to working closely with the UCD community to ensure that those affected by this terrible disease live their lives with as few challenges and interruptions as possible.”

ACER-001 (sodium phenylbutyrate) Palatability Data Presented at SIMD and GMDI 2022
Taste-Masked Coating of Sodium Phenylbutyrate (ACER-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders^6,7

Results from two Phase 1, open-label, repeated measures, taste assessment studies of ACER-001 (sodium phenylbutyrate) suspension and sodium phenylbutyrate (BUPHENYL®) powder were presented at the SIMD Annual Meeting and Genetic Metabolic Dieticians International (GMDI) Conference in April 2022 and May 2022, respectively. ACER-001 for the treatment of certain UCDs is now marketed in the U.S. as OLPRUVA™ (sodium phenylbutyrate) following FDA approval in December 2022. Results from both studies concluded that ACER-001 (sodium phenylbutyrate) suspension was shown to have overall lower flavor intensity scores than sodium phenylbutyrate (BUPHENYL®) powder when administered within five minutes of preparation.

New FDA-Approved UCD Treatment Option: OLPRUVA™
ACER-001 (sodium phenylbutyrate) was approved for the treatment of certain UCDs in December 2022 and is marketed under the brand name, OLPRUVA™. OLPRUVA™ (sodium phenylbutyrate) for oral suspension 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 urea cycle disorders (UCDs), involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).⁴ Please see Important Safety Information and full Prescribing Information, including Patient Information.

References

Batshaw ML, Tuchman M, Summar M, Seminara J, Members of the Urea Cycle Disorders C. A longitudinal study of urea cycle disorders. Mol Genet Metab. 2014;113(1-2):127-130.
Pena-Quintana L, Llarena M, Reyes-Suarez D, Aldamiz-Echevarria L. Profile of sodium phenylbutyrate granules for the treatment of urea-cycle disorders: patient perspectives. Patient Prefer Adherence. 2017;11:1489-1496.
Pena-Quintana L, Llarena M, Reyes-Suarez D, Aldamiz-Echevarria L. Profile of sodium phenylbutyrate granules for the treatment of urea-cycle disorders: patient perspectives. Patient Prefer Adherence. 2017;11:1489-1496.
Edelblut J, et al. Quantifying Preferences for Urea Cycle Disorder Treatments Using a Discrete-Choice Experiment. SIMD March 2023
OLPRUVA^TM (sodium phenylbutyrate) for oral suspension. Prescribing information. Newton, MA: Acer Therapeutics Inc.
Steiner R, et al. The Pharmacokinetics of Taste-Masked Sodium Phenylbutyrate (ACER-001) for the Treatment of Urea Cycle Disorders Under Fasting and Fed Conditions in Healthy Volunteers. SIMD April 2022
Cederbaum S, et al. Taste-Masked Coating of Sodium Phenylbutyrate (ACER-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders. GMDI May 2022

Acer Forward-Looking Statements
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release are forward-looking statements. Examples of such statements include, but are not limited to, statements about survey data to be presented, including the timing, detail and content thereof, and OLPRUVA™ launch activities, including the addition of resources, the launch of patient support services, discussions with payers and organizations, and the Company’s pricing strategy and related plans and expectations. 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) 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 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 Contacts
Jim DeNike
Acer Therapeutics Inc.
jdenike@medinfo+1-844-902-6100

Nick Colangelo
Gilmartin Group
[email protected]
+1-332-895-3226

Acer Therapeutics Announces $2.675 Million Registered Direct Offering Priced At-the-Market Under Nasdaq Rules

Posted on March 22, 2023March 22, 2023 by AcerUser

NEWTON, MA – March 22, 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 that it has entered into a definitive agreement for the purchase and sale of 2,920,306 shares of the Company’s common stock (or common stock equivalents) at a purchase price of $0.916 per share of common stock (or common stock equivalent) in a registered direct offering priced at-the-market under Nasdaq rules. In addition, in a concurrent private placement, the Company will issue warrants to purchase up to 2,920,306 shares of common stock. The warrants will have an exercise price of $0.791 per share, will be exercisable immediately following issuance and have a term of five and one-half years from the issuance date.

H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The closing of the offering is expected to occur on or about March 24, 2023, subject to the satisfaction of customary closing conditions. The gross proceeds to the Company from the offering are expected to be approximately $2.675 million, before deducting the placement agent’s fees and other offering expenses payable by the Company. The Company intends to use the net proceeds from this offering for general corporate purposes and working capital.

The shares of common stock described above (but not the warrants issued in the concurrent private placement or the shares of common stock underlying such warrants) are being offered by the Company pursuant to a “shelf” registration statement on Form S-3 (File No. 333-261342) initially filed with the Securities and Exchange Commission (the “SEC”) on November 24, 2021 and declared effective by the SEC on December 7, 2021. The offering of the shares of common stock is made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and accompanying prospectus relating to the registered direct offering will be filed with the SEC. Electronic copies of the final prospectus supplement and accompanying prospectus may be obtained, when available, on the SEC’s website at http://www.sec.gov or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (212) 865-5711 or e-mail at [email protected].

The warrants described above were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the “Securities Act”), and, along with the shares of common stock underlying the warrants, have not been registered under the Securities Act, or applicable state securities laws. Accordingly, the warrants and underlying shares of common stock may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

Acer Forward-Looking Statements
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release are forward-looking statements. Examples of such statements include, but are not limited to, statements regarding the consummation of the offering, the satisfaction of closing conditions and the use of proceeds from the offering, as well as statements about the continued development of ACER-801 for treatment of VMS or other indications, the continued development of EDSIVO™, and launch preparations for OLPRUVA™. 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, market and other conditions, the availability of financing to fund our pipeline product development programs, commercialization efforts 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 Contacts
Jim DeNike
Acer Therapeutics Inc.
jdenike@medinfo+1-844-902-6100

Nick Colangelo
Gilmartin Group
[email protected]
+1-332-895-3226

Acer Therapeutics Announces Topline Results from ACER-801 (Osanetant) Phase 2a Trial

Posted on March 17, 2023March 17, 2023 by AcerUser

NEWTON, MA – March 17, 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 that topline results from its 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 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. Concurrently, Acer is continuing to focus on preparation for its commercial launch of OLPRUVA™ (sodium phenylbutyrate) for oral suspension for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS), and the ongoing pivotal Phase 3 clinical evaluation of EDSIVO™ (celiprolol) for vascular Ehlers-Danlos Syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation, subject in each instance to availability of additional capital beyond early in Q2 2023.

“We are surprised and disappointed the Phase 2a trial did not meet its efficacy objectives in treating VMS given the extensive body of non-clinical and clinical evidence previously generated,” stated Chris Schelling, CEO and Founder of Acer. “We intend to conduct a comprehensive analysis of the totality of the clinical trial data – including the pharmacokinetic data, which has not yet been analyzed – which will inform our path forward for the program, including our collaborations for prostate cancer and post-traumatic stress disorder (PTSD). We wish to thank the patients, investigators and partners who participated in this clinical trial. 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 our FDA-approved product, OLPRUVA™, for oral suspension for the treatment of UCDs involving deficiencies of CPS, OTC or AS, as well as our pivotal Phase 3 clinical trial of EDSIVO™ for vEDS.”

About ACER-801 (osanetant)
ACER-801 (osanetant) is an investigational non-hormonal, neurokinin 3 receptor (NK3R) antagonist being studied for the potential treatment of patients with Vasomotor Symptoms (VMS), prostate cancer and post-traumatic stress disorder (PTSD). In December 2018, Acer entered into an exclusive license agreement with Sanofi to acquire worldwide rights to ACER-801 (osanetant). In the Phase 2a study, forty-nine postmenopausal women aged 40-65 who experienced moderate to severe hot flashes were randomized 1:1:1:1 and received either twice daily 50 mg, 100 mg, 200 mg of ACER-801 or placebo over a 14-day treatment period, followed by a 14-day safety follow-up assessment. Primary endpoints were safety and pharmacokinetics, with efficacy compared to placebo as a secondary endpoint.

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. 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 Vasomotor Symptoms (VMS), post-traumatic stress disorder (PTSD) and prostate cancer; and EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. For more information, visit www.www.acertx.com.

Acer currently believes that its existing cash and cash equivalents will be sufficient to fund its anticipated operating and capital requirements into early in the second quarter of 2023. Acer will require additional financing 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, as well as to complete development and seek to obtain marketing approval of Acer’s other product candidates and, if approved, to commercialize such other product candidates. There can be no assurance that Acer will be able to obtain needed capital on acceptable timing and terms, or at all. A failure in this regard could force Acer to delay, limit, reduce or terminate its product development, other operations or commercialization efforts, or to suspend or restructure its business.

Corporate and IR Contacts

Jim DeNike
Acer Therapeutics Inc.
jdenike@medinfo+1-844-902-6100

Nick Colangelo
Gilmartin Group
[email protected]
+1-332-895-3226

Acer Therapeutics Announces Presentation of HCP Urea Cycle Disorder Treatment Preference Data at the Society for Inherited Metabolic Disorders Annual Meeting and Provides OLPRUVA™ Program Update

Posted on March 15, 2023March 15, 2023 by AcerUser

NEWTON, MA – March 15, 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 data will be presented from a survey designed to quantify preferences of healthcare providers for Urea Cycle Disorders (UCDs) at the upcoming 44^th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD), March 18^th-21^st in Salt Lake City, Utah. Results from the discrete choice experiment will be available on Acer’s website following the poster presentation at SIMD on March 19 by Dr. Robert Steiner (University of Wisconsin School of Medicine and Public Health) at https://www.acertx.com/publications-and-presentations/.

Poster #97: Quantifying Preferences for Urea Cycle Disorder Treatments Using a Discrete-Choice Experiment
This poster summarizes results from a web-based, quantitative survey study using a discrete choice experiment (DCE) methodology. This study sought to capture the perspectives of healthcare providers (HCPs) with experience treating UCDs to quantify the attributes of nitrogen-binding medications (such as sodium phenylbutyrate and glycerol phenylbutyrate) for UCDs that may influence overall prescription and patient adherence.

“OLPRUVA™¹ leverages the well-established efficacy of sodium phenylbutyrate in an innovative dual-coating formulation designed for palatability,” said Adrian Quartel, MD, FFPM, CMO of Acer. “We look forward to the presentation of the survey results at SIMD as we continue to prepare for the launch of OLPRUVA™, and to the delivery of an important alternative treatment option to those facing the unmet needs of existing therapies.”

Results from two Phase 1, open-label, repeated measures, taste assessment studies of ACER-001 (sodium phenylbutyrate) suspension and sodium phenylbutyrate (BUPHENYL®) powder were presented at the SIMD Annual Meeting and Genetic Metabolic Dieticians International (GMDI) Conference in April 2022 and May 2022, respectively. ACER-001 for the treatment of UCDs is now marketed in the U.S. as OLPRUVA™ (sodium phenylbutyrate) following FDA approval in December 2022. Results from both studies concluded that ACER-001 (sodium phenylbutyrate) suspension was shown to have overall lower flavor intensity scores than sodium phenylbutyrate (BUPHENYL®) powder when administered within five minutes of preparation.

OLPRUVA™ Program Update
Acer also provided an update on its commercial launch activities for OLPRUVA™ in advance of the SIMD Annual Meeting, where it will be exhibiting in booths 312 and 213. To support the launch of OLPRUVA™ in Q2 2023, the company is actively adding resources to establish its commercial and medical affairs presence in the U.S. As a part of its OLPRUVA™ commercialization strategy, Acer has recently introduced its patient support service, OLPRUVA™ Navigator by Acer Therapeutics, that is designed to assist UCD patients with support, access, education, and patient adherence to treatment. Representatives will begin accepting prescriptions in late Q2 2023. Acer is also actively engaged in negotiations regarding access for OLPRUVA™ with the major commercial payers and state Medicaid organizations.

In connection with the company’s ongoing support for the rare disease patient community, Acer has established a pricing strategy that reflects its commitment to deliver innovative treatments that are responsibly priced and accessible to those in need. As a result, Acer intends to price OLPRUVA™ competitively, at a significant discount to the currently available commercial product RAVICTI®, while implementing predictable pricing that will not increase beyond the rate of inflation. The company also plans to invest a portion of OLPRUVA™ revenue back into additional solutions aimed at improving outcomes for UCD patients. More information will be available at OLPRUVA.com.

“We are proud that we can provide OLPRUVA™ — an innovative, responsibly priced treatment option — to patients with UCDs. And we are committed to working closely with the UCD community to ensure that those affected by this terrible disease live their lives with as few challenges and interruptions as possible,” commented Chris Schelling, CEO and Founder of Acer. “While our future plans are subject to the availability of working capital moving forward, we feel strongly that Acer is well positioned to access additional sources of capital to fund day-to-day operations and OLPRUVA’s™ launch requirements.”

New FDA-Approved UCD Treatment Option: OLPRUVA™
ACER-001 (sodium phenylbutyrate) was approved for the treatment of certain UCDs in December 2022 and is marketed under the brand name, OLPRUVA™. OLPRUVA™ (sodium phenylbutyrate) for oral suspension 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 urea cycle disorders (UCDs), involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).¹ Please see Important Safety Information and full Prescribing Information, including Patient Information.

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. 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 Vasomotor Symptoms (VMS), 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.www.acertx.com. In March 2021, Acer entered into a Collaboration and License Agreement with RELIEF THERAPEUTICS Holding SA for development and commercialization of OLPRUVA™ (ACER-001) in which Acer retains development and commercialization rights in the U.S., Canada, Brazil, Turkey, and Japan. For more information, visit www.www.acertx.com.

References

Corporate and IR Contacts

Jim DeNike
Acer Therapeutics Inc.
jdenike@medinfo+1-844-902-6100

Nick Colangelo
Gilmartin Group
[email protected]
+1-332-895-3226