Acer Therapeutics Announces Poster Presentation at Society for Vascular Medicine Annual Meeting

NEWTON, MA May 31, 2019 – 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 presentation and publication of results from its Pilot Study to Evaluate Effect of Antihypertensive Therapy on the Rates of Clinical Events in Patients with Vascular Ehlers-Danlos Syndrome (vEDS). These results were presented in a poster session at the Society for Vascular Medicine (SVM) 2019 Annual Scientific Sessions on May 30, 2019 and are available at https://acertx.com/edsivo-publications-and-presentations/. The results will also be published in the online and print versions of SVM’s journal, Vascular Medicine.

The goal of this pilot study was to better understand the extent of use of antihypertensive medications in vEDS patients and their potential benefit in reducing the rate of vEDS-related clinical events. There are currently no approved medications to treat vEDS in the U.S.; however, antihypertensive medications are used by some physicians in vEDS patients with hopes of lowering the occurrence of clinical events.1

Researchers conducted a retrospective analysis of U.S. insurance claims (Truven MarketScan®) identifying vEDS patients over a four-year period from January 1, 2014 to December 31, 2017. The insurance claims-based information was then stratified based on insurance claims for antihypertensive medications and no antihypertensive medication. Researchers then calculated and compared the clinical event rate, including arterial rupture and aneurysm, and other hollow organ rupture, for each group.

Of the 3,614 vEDS patients identified, 2,371 (65.6%) were determined not to be taking any antihypertensive medication and 1,243 (34.4%) were determined to be taking antihypertensive medications. There was no statistically significant difference between rate of clinical events in patients taking any of the antihypertensive medications compared to patients not taking an antihypertensive medication.

“While some physicians prescribe antihypertensive medications as possible preventative therapy in vEDS patients, the pilot study indicates that there is no evidence of their benefit in vEDS, other than the potential benefit displayed by celiprolol treatment in the only controlled clinical study of medical treatment in vEDS,”2 said William Andrews, MD, FACP, Chief Medical Officer of Acer. “While we recognize the limitations of drawing clinical conclusions from a pilot study based on retrospective analysis of insurance claims databases, we believe these results,  along with the high incidence of major complications in vEDS patients, underscore the serious need for an approved, safe and effective therapy for this disease.”

About vEDS and EDSIVO™ (celiprolol)

Ehlers-Danlos Syndrome (EDS) is a group of hereditary disorders of connective tissue. vEDS is the most severe subtype where patients suffer from life threatening arterial dissections and ruptures, as well as intestinal and uterine ruptures. The median mortality is 51 years of age.3 An Acer-commissioned patient-finder study phenotypically identified 4,169 vEDS patients in the U.S. from an analysis of a commercially available patient claims database with data of approximately 190 million unique patient lives. Based on that information, Acer estimates the prevalence of phenotypically-defined vEDS in the U.S. could be greater than 1 in 45,000. Currently, there are no FDA-approved therapies for vEDS. Acer is advancing EDSIVO™ (celiprolol), a new chemical entity (NCE), for the treatment of vEDS based on a randomized controlled clinical study of celiprolol2. FDA granted a priority review of the EDSIVO™ NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 25, 2019. EDSIVO™ received FDA Orphan Drug Designation for the potential treatment of vEDS in 2015.

About Acer Therapeutics

Acer, headquartered in Newton, MA, 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 three clinical-stage candidates: EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a fully taste-masked, immediate release formulation of sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant for the treatment of various neuroendocrine disorders. Acer’s product candidates are believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and an accelerated path for development, which may include utilizing expedited programs (e.g. Priority Review) established by the FDA and/or using the regulatory pathway established under section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act (FFDCA) that allows an applicant to rely at least in part on third-party data for approval, which may expedite the preparation, submission and approval of a marketing application.

Disclosure notice: EDSIVO™ is an investigational drug candidate that has not been approved by the FDA for any indication.

For more information, visit www.acertx.com.

References

  1. Byers PH et al. Am J Med Genet Part C Semin Med Genet. 2017;175C:40-47.
  2. Ong KT, et al: . Effect of celiprolol on prevention of cardiovascular events in vascular Ehlers-Danlos syndrome: a prospective randomised, open, blinded-endpoints trial. 2010;376(9751):1476-1484.
  3. Pepin M, et al. Clinical and genetic features of Ehlers-Danlos syndrome type IV, the vascular type. N Engl J Med. 2000; 342:673-80.

 

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, future financial position, future revenues, projected expenses, regulatory 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 expectations regarding our capital resources; the potential for EDSIVO™ (celiprolol), ACER-001 and osanetant to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity in any target indication; the adequacy of our capital to support our future operations and our ability to successfully initiate and complete clinical trials and regulatory submissions; our progress toward our pre-commercial launch plans for EDSIVO™; the ability to protect our intellectual property rights; our  strategy and business focus;  and the development, expected timeline and commercial potential of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to 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 of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.

Investor Contact:

Hans Vitzthum

LifeSci Advisors

Ph: 617-535-7743

hans@lifesciadvisors.com

 

Jim DeNike

Acer Therapeutics Inc.

Ph: 844-902-6100

jdenike@acertx.com

 

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Acer Therapeutics to Present at Upcoming Investor Conferences

NEWTON, MA May 23, 2019 – 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 provide a corporate overview at upcoming investor conferences in June, including: the William Blair 2019 Growth Stock Conference, the Jefferies 2019 Healthcare Conference, and the Raymond James Life Sciences & MedTech Conference.

 

Acer Conference Presentation Details:

 

Conference:    William Blair 2019 Growth Stock Conference

Date:               Wednesday, June 5, 2019

Time:               1:20 pm Central Time

Location:         Loews Chicago Hotel, Chicago

 

Conference:    Jefferies 2019 Healthcare Conference

Date:               Thursday, June 6, 2019

Time:               10:00 am Eastern Time

Location:         Grand Hyatt New York, New York

 

Conference:    Raymond James Life Sciences & MedTech Conference

Date:               Wednesday, June 19, 2019

Time:               8:35 am Eastern Time

Location:         Lotte New York Palace, New York

 

A webcast and replay of each presentation can be found in the “Events and Presentations” section of the company’s website or can by accessed via this link.

 

About Acer Therapeutics

Acer, headquartered in Newton, MA, 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 three clinical-stage candidates: EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a fully taste-masked, immediate release formulation of sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant for the treatment of various neuroendocrine disorders. Acer’s product candidates are believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and an accelerated path for development, which may include utilizing expedited programs (e.g. Priority Review) established by the FDA and/or using the regulatory pathway established under section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act (FFDCA) that allows an applicant to rely at least in part on third-party data for approval, which may expedite the preparation, submission and approval of a marketing application.

 

Disclosure notice: EDSIVO™ is an investigational drug candidate that has not been approved by the FDA for any indication.

 

For more information, visit www.acertx.com.

 

Investor Contacts:

Hans Vitzthum

LifeSci Advisors

Ph: 617-535-7743

hans@lifesciadvisors.com

 

Jim DeNike

Acer Therapeutics Inc.

Ph: 844-902-6100

jdenike@acertx.com

Acer Therapeutics Reports First Quarter 2019 Financial Results and Provides Corporate Update

NEWTON, MA May 14, 2019 – 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 first quarter ended March 31, 2019 and provided an update on the Company’s recent corporate developments.

“Building on our success in 2018, we continued to execute in the first quarter of 2019 on our pre-commercial launch strategy for EDSIVO™ (celiprolol) while growing and advancing our product pipeline,” said Chris Schelling, CEO and Founder of Acer. “With a targeted FDA PDUFA action date of June 25, 2019, we have made significant progress in preparing for a potential launch of EDSIVO™, including the addition of seasoned commercial and medical affairs leaders with extensive sales, marketing, market access and product launch experience in orphan and ultra-orphan markets. Also, in April 2019, we announced the publication of the Paris registry data in JACC that supplements the previously-reported safety and efficacy of celiprolol in vEDS patients with a confirmed type III collagen (COL3A1) mutation.”1

Mr. Schelling continued, “The first quarter was also marked by the growth and further advancement of our pipeline products, including the in-license of osanetant and preparation of our bridging study for ACER-001 with initial indication in Urea Cycle Disorders (UCDs). We look forward to a number of significant milestones throughout the second half of 2019, including the potential launch of our first commercially-available therapeutic product, provided it receives FDA approval, and the advancement of two additional therapeutic candidates with an expedited path to registration.”

First Quarter 2019 and Recent Highlights

  • Announced the publication of long-term data from a cohort of COL3A1-positive vascular Ehlers-Danlos syndrome (vEDS) patients in the Journal of the American College of Cardiology (JACC). The published data includes up to 17 years of safety data in this population, and the survival curve analysis shows that those patients not treated with celiprolol had a significantly worse outcome than celiprolol-treated patients. The authors also observed a relative decrease in hospitalization rates for acute arterial events during the time period in which the majority of patients were on celiprolol, suggesting a positive effect of celiprolol on the incidence and/or severity of new arterial events1
  • Filed an Investigational New Drug application (IND) for ACER-001 for initiation of a pivotal bridging study intended to evaluate bioequivalence and taste assessment of ACER-001 compared to BUPHENYL® in healthy subjects
  • Continued to build out the Acer management team with additional senior-level commercial, medial affairs, marketing, compliance and other core personnel, many with more than 20 years of commercial orphan and ultra-orphan leadership experience
  • Entered into an exclusive license agreement with Sanofi to acquire worldwide rights to osanetant, a clinical-stage, selective, non-peptide tachykinin NK3 receptor antagonist. Acer plans to initially pursue development of osanetant as a potential treatment for certain neuroendocrine-related disorders
  • Ended the first quarter with $31.8 million in cash and cash equivalents, which the Company believes is sufficient to fund its current operating and capital requirements into the first half of 2020

 

Upcoming Milestones

  • Prepare for Prescription Drug User Fee Act (PDUFA) action date for EDSIVO™ of June 25, 2019
  • Continue to execute on pre-commercial plan and launch preparation for EDSIVO™
  • Advance potential neuroendocrine indications for osanetant in the second half of 2019
  • Initiate a pivotal bridging study of ACER-001 in Q4 2019
  • Continue efforts toward advancing and expanding the company’s pipeline

 

Financial Results for the First Quarter 2019

Cash position. Cash and cash equivalents were $31.8 million as of March 31, 2019, compared to $41.7 million as of December 31, 2018. The Company believes its cash position will be sufficient to fund its current operating and capital requirements into the first half of 2020.

Research and Development Expenses. Research and development expenses were $3.9 million during the three months ended March 31, 2019, compared to $2.1 million during the three months ended March 31, 2018. This increase of $1.8 million was principally due to increases in regulatory and filing fees, expenses related to manufacturing services, and employee-related expenses, partially offset by a decrease in spending for contract research and regulatory consulting services. Research and development expenses for the three months ended March 31, 2019 were primarily comprised of approximately $3.3 million related to EDSIVO™ and approximately $0.6 million related to ACER-001.

General and Administrative Expenses. General and administrative expenses were $4.2 million for the three months ended March 31, 2019 compared to $1.9 million for the three months ended March 31, 2018. The increase of $2.3 million was primarily due to increases in employee-related expenses, including increased headcount, and expenses related to pre-commercial activities.

Net Loss. Net loss for the three months ended March 31, 2019 was $8.0 million, or $0.79 loss per share (basic and diluted), compared to a net loss of $4.0 million, or $0.53 loss per share (basic and diluted), for the three months ended March 31, 2018. The increase in loss per share (basic and diluted) was driven largely by increases in employee-related expenses, including increased headcount; regulatory and filing fees; expenses related to manufacturing services; and expenses related to pre-commercial activities, partially offset by a decrease in spending for contract research and regulatory consulting services, as well as by an increase in the number of shares outstanding at March 31, 2019 as compared to March 31, 2018.

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

About Acer Therapeutics

Acer, headquartered in Newton, MA, 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 three clinical-stage candidates: EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a fully taste-masked, immediate release formulation of sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant for the treatment of various neuroendocrine disorders. Acer’s product candidates are believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and an accelerated path for development, which may include utilizing expedited programs (e.g. Priority Review) established by the FDA and/or using the regulatory pathway established under section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act (FFDCA) that allows an applicant to rely at least in part on third-party data for approval, which may expedite the preparation, submission and approval of a marketing application.

Disclosure notice: EDSIVO™ is an investigational drug candidate that has not been approved by the FDA for any indication.

For more information, visit www.acertx.com.

References

  • Frank, M, et al. Vascular Ehlers-Danlos Syndrome: Long-Term Observational Study. JAm Coll Cardiol 2019; 73(15):1948–57

 

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, future financial position, future revenues, projected expenses, regulatory 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 expectations regarding our capital resources; the potential for EDSIVO™ (celiprolol), ACER-001 and osanetant to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity in any target indication; the adequacy of our capital to support our future operations and our ability to successfully initiate and complete clinical trials and regulatory submissions; our progress toward our pre-commercial launch plans for EDSIVO™; the ability to protect our intellectual property rights; our  strategy and business focus;  and the development, expected timeline and commercial potential of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to 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 of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.

ACER THERAPEUTICS INC.

CONSOLIDATED STATEMENTS OF OPERATIONS

(Unaudited)

Three Months Ended  
March 31,  
2019     2018    
     
               
 Operating expenses:
 Research and development $ 3,946,220 $ 2,073,971
 General and administrative 4,230,698 1,917,030
 Loss from operations (8,176,918 ) (3,991,001 )
 Other income:
 Interest income 185,143 30,690
 Foreign currency transaction gain (loss) 23,070 (23,293 )
 Total other income, net 208,213 7,397
 Net loss $ (7,968,705 ) $ (3,983,604 )
 Net loss per share – basic and diluted $ (0.79 ) $ (0.53 )
 Weighted average common shares outstanding – basic and diluted 10,087,363 7,497,433

 

SELECTED BALANCE SHEET DATA (Unaudited): 

  March 31,     December 31,  
    2019     2018  
                 
Cash and cash equivalents $ 31,831,427 $ 41,671,284
Prepaid expenses and other current assets $ 778,738 $ 1,075,021
Property and equipment, net $ 210,257 $ 130,867
Total assets $ 41,016,540 $ 50,663,419
Total liabilities $ 3,230,160 $ 5,580,261
Total stockholders’ equity $ 37,786,380 $ 45,083,158

Investor Contact:

Hans Vitzthum

LifeSci Advisors

Ph: 617-535-7743

hans@lifesciadvisors.com

 

Jim DeNike

Acer Therapeutics Inc.

Ph: 844-902-6100

jdenike@acertx.com

 

#  #  #

Acer Therapeutics to Present at UBS Global Healthcare Conference

NEWTON, MA May 7, 2019 – 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 provide a corporate overview at the UBS Global Healthcare Conference, May 20-22, 2019 at the Grand Hyatt New York.

 

Acer Conference Presentation Details:

 

Conference:    UBS Global Healthcare Conference

Date:               Tuesday, May 21, 2019

Time:               10 am Eastern Time (presentation followed by breakout session)

Location:         Grand Hyatt New York, New York

Webcast:         https://acertx.com/investor-relations/events-presentations/

 

About Acer Therapeutics

Acer, headquartered in Newton, MA, 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 three clinical-stage candidates: EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a fully taste-masked, immediate release formulation of sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant for the treatment of various neuroendocrine disorders. Acer’s product candidates are believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and an accelerated path for development, which may include utilizing expedited programs (e.g. Priority Review) established by the FDA and/or using the regulatory pathway established under section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act (FFDCA) that allows an applicant to rely at least in part on third-party data for approval, which may expedite the preparation, submission and approval of a marketing application.

 

Disclosure notice: EDSIVO™ is an investigational drug candidate that has not been approved by the FDA for any indication.

 

For more information, visit www.acertx.com.

 

Investor Contacts:

Hans Vitzthum

LifeSci Advisors

Ph: 617-535-7743

hans@lifesciadvisors.com

 

Jim DeNike

Acer Therapeutics Inc.

Ph: 844-902-6100

jdenike@acertx.com

 

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