Category: News

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

 

#  #  #

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

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

 

#  #  #

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

 

#  #  #

NEWTON, MA – April 16, 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 critical unmet medical needs, today announced the publication of long-term data from a cohort of COL3A1-positive vEDS patients in the Journal of the American College of Cardiology (JACC). The publication, entitled “Vascular Ehlers-Danlos Syndrome: Long-Term Observational Study,” was authored by Michael Frank, MD, Xavier Jeunemaitre, MD, PhD, and Pierre Boutouyrie, MD, PhD, et al.

An audio summary by Editor-in-Chief of JACC, Dr. Valentin Fuster, may be accessed (for free) via the following link:

http://hwcdn.libsyn.com/p/7/a/d/7adfc876add223e8/JACC7315_fustersummary_06.mp3?c_id=39923912&cs_id=39923912&expiration=1555355369&hwt=978a334a3160927aae2b8c4dd9c25209

The publication may be accessed (for purchase) here: http://www.onlinejacc.org/content/73/15/1948

The editorial may be accessed (for purchase) here: http://www.onlinejacc.org/content/73/15/1958

This published study describes outcomes in 144 COL3A1-positive vEDS patients clinically monitored and treated at the French National Referral Center for Rare Vascular Diseases (Paris, France) between the years 2000 and 2017. Patients were followed for a median of 5.3 years, and up to 20 years. At the initial work up, 50% of patients were not treated regularly and only 33.3% were taking celiprolol; by the end of the study period, the majority (90.3%) were treated with celiprolol alone or in combination with other medications. Once the maximum tolerated dose of celiprolol was reached, 90 (62.5%) patients remained at this dose throughout their follow-up. Only 5 (3.5%) patients required dose reduction due to fatigue, and no serious drug-related adverse event was recorded.

Patients had a lower mortality rate than that expected from the natural history of the disease as described in previous U.S. reports (1). Survival curve analysis showed that those not treated with celiprolol had a significantly worse outcome than celiprolol-treated patients: survival was 80.7% (95% CI 67.8%–93.6%) in those treated with celiprolol versus 48.5% (95% CI 19.7%–77.4%) in those not treated (p<0.001) after 11.1 years of follow-up. Survival was significantly higher in patients treated with a median dose of celiprolol of 400mg/day (n=83) vs. patients treated with a lower median dose of 217mg/d [100-300mg/day] (n=27), suggesting a dose effect and that 400mg/day should be considered the optimal dose. 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 events. The authors concluded that in this large, long-term cohort study, vEDS patients had a higher survival rate than expected relative to the known natural history of the disease and a lower annual occurrence of arterial complications, and that celiprolol use was potentially associated with these significant improvements in clinical outcomes.

“The higher overall survival in patients treated with celiprolol in this long-term study in COL3A1-positive vEDS patients appears to correlate with the significant event-free survival advantage that was reported in the Ong, et al. study of celiprolol treatment in vEDS patients (2),” said Michael Frank, MD, clinical investigator from the Paris group and first author of the publication.

“We are pleased to see this publication from the vEDS clinical investigator group in Paris which provides patients and physicians with a greater understanding of this chronic disease, including data suggesting a positive impact of celiprolol, which has a unique pharmacological profile,” said William Andrews, MD, FACP, Chief Medical Officer of Acer.

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 critical 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 (UCD) 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) Pepin, et al. Survival is affected by mutation type and molecular mechanism in vascular Ehlers–Danlos syndrome (EDS type IV). Genet Med. 2014 Dec;16(12):881-8.

(2) Ong, et al. Effect of celiprolol on prevention of cardiovascular events in vascular Ehlers-Danlos syndrome: a prospective randomised, open, blinded-endpoints trial. Lancet. 2010; 376: 1476–84.

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 capitalization and 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; the ability to protect our intellectual property rights; the nature, strategy and our focus; future economic conditions or performance; 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 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 Contacts:

Hans Vitzthum

LifeSci Advisors

Ph: 617-535-7743

hans@lifesciadvisors.com

 

Jim DeNike

Acer Therapeutics

Ph: 844-902-6100

jdenike@acertx.com

NEWTON, MA – March 13, 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 critical unmet medical need, today announced that Acer’s management team will provide a corporate overview at upcoming investor conferences in March and April, including: the Roth Capital Partners Growth Stock Conference, William Blair Late Stage Therapeutics Conference, and Needham & Company Healthcare Conference.

Acer Conference Presentation Details:

Conference:    Roth Capital Partners 31^st Annual Growth Stock Conference

Date:               Monday, March 18 – Orphan Drug Panel

Time:               11 am Pacific Time

Date:               Tuesday, March 19 – Fireside Chat

Time:               9:30 am Pacific Time

Location:         Ritz Carlton, Dana Point, Calif.

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

Conference:    William Blair Late Stage Therapeutics Conference

Date:               Thursday, April 4

Location:         Omni Berkshire Hotel, New York

Conference:    Needham & Company 18^th Annual Healthcare Conference

Date:               Wednesday, April 10 – Company Presentation

Time:               3:30 pm Eastern Time

Location:         Westin NY Grand Central Hotel, 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 critical unmet medical need. 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 (UCD) 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.

For more information, visit www.acertx.com.

Investor Contacts:

Hans Vitzthum

LifeSci Advisors

Ph: 617-535-7743

hans@lifesciadvisors.com

 

Jim DeNike

Acer Therapeutics

Ph: 844-902-6100

jdenike@acertx.com

#  #  #

NEWTON, MA – March 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 critical unmet medical need, today reported financial results for the quarter and full year ended December 31, 2018 and provided an update on the Company’s recent corporate developments.

“We were very active in 2018 preparing for some significant potential milestones in 2019. Most importantly, in December, the FDA accepted our NDA for EDSIVO™ (celiprolol) under priority review, with a targeted PDUFA action date of June 25, 2019. In order to prepare for the potential U.S. launch of EDSIVO™, we strengthened our balance sheet with a successful financing in August, which allowed us to accelerate our pre-commercial activities,” said Chris Schelling, CEO and Founder of Acer. “Additionally, we made a strategic decision to expand our pipeline of clinical-stage product candidates by in-licensing the global rights to osanetant from Sanofi in January. If successful, Acer is positioned to become a commercial pharmaceutical company for serious rare and life-threatening diseases in the second half of this year with one therapeutic on the market (if the FDA approves our NDA for EDSIVO™) and other therapeutic candidates (ACER-001 and osanetant) in development.”

2018 and Recent Highlights

• S. Food and Drug Administration (FDA) accepted for review in December 2018 Acer’s New Drug Application (NDA) for EDSIVO™ for the treatment of vEDS in patients with a confirmed type III collagen (COL3A1) mutation. The FDA also granted a priority review of the NDA, which is a designation granted by the FDA to accelerate the review process for drugs that offer a significant improvement in treatment or provide treatment where no satisfactory alternative therapy exists
• Entered into an additional agreement with Assistance Publique—Hôpitaux de Paris (AP-HP) in September 2018, pursuant to which Acer obtained the exclusive worldwide intellectual property rights to three European patent applications relating to certain uses of celiprolol. The Company subsequently filed three U.S. patent applications on this subject matter
• Raised $46 million, including underwriters’ full exercise of their option to purchase additional shares to cover over-allotments, through an underwritten public offering in August 2018
• Entered into an exclusive license agreement with Sanofi in January 2019 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 rare or life-threatening neuroendocrine-related disorders
• Ended 2018 with $41.7 million in cash and cash equivalents and no debt, which the Company believes is sufficient to fund its current operating and capital requirements into the first half of 2020

 

Upcoming Milestones

• Potential publication of celiprolol vEDS Patient Registry data, which is currently under peer review
• Prescription Drug User Fee Act (PDUFA) action date for EDSIVO™ of June 25, 2019
• Continuing pre-commercial activities for EDSIVO™
• Continue efforts toward advancing and expanding its pipeline
• Continue efforts toward building out the management team with additional senior-level commercial hires, as well as adding to the commercial and medical affairs teams and other core personnel

Financial Results for the Fourth Quarter and Full-Year 2018

Cash position. Cash and cash equivalents were $41.7 million as of December 31, 2018, compared to $15.6 million as of December 31, 2017. 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 $5.3 million during the three months ended December 31, 2018, compared to $1.8 million during the three months ended December 31, 2017. Research and development expenses for the three months ended December 31, 2018 were primarily comprised of approximately $3.1 million related to EDSIVO™ and approximately $1.7 million related to ACER-001. Research and development expenses were $12.5 million during the year ended December 31, 2018, compared to $8.7 million during the year ended December 31, 2017. This increase of $3.8 million was principally due to increases in employee-related expenses, regulatory consulting expenses, and expenses related to manufacturing services, partially offset by a decrease in spending for contract research services.

General and Administrative Expenses. General and administrative expenses were $3.4 million for the three months ended December 31, 2018 compared to $2.4 million for the three months ended December 31, 2017. General and administrative expenses were $9.3 million for the year ended December 31, 2018 compared to $5.2 million for the year ended December 31, 2017. The increase of $4.1 million was primarily due to increases in employee-related expenses, expenses related to pre-commercial activities, and insurance expense.

Net Loss. Net loss for the three months ended December 31, 2018 was $8.5 million, or $0.85 loss per share (basic and diluted), compared to a net loss of $4.2 million, or $0.63 loss per share (basic and diluted), for the three months ended December 31, 2017. Net loss for the year ended December 31, 2018 was $21.3 million, or $2.49 loss per share (basic and diluted), compared to a net loss of $14.2 million, or $3.84 loss per share (basic and diluted), for the year ended December 31, 2017. The decrease in loss per share (basic and diluted) was driven largely by an increase in shares outstanding at December 31, 2018 compared to December 31, 2017.

For additional information, please see Acer’s Annual Report on Form 10-K 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 critical unmet medical need. 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 (UCD) 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.

For more information, visit www.acertx.com.

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 capitalization and 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; the ability to protect our intellectual property rights; the nature, strategy and our focus; future economic conditions or performance; 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 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

	Three Months Ended								Years Ended
	December 31,								December 31,
	2018				2017				2018				2017
Operating expenses:
Research and development	$	5,312,378			$	1,776,210			$	12,452,424			$	8,725,026
General and administrative		3,386,160				2,430,677				9,260,583				5,223,101
Loss from operations		(8,698,538	)			(4,206,887	)			(21,713,007	)			(13,948,127	)
Other income (expense):
Interest income		206,637				10,029				412,553				14,848
Interest and other expense		(987	)			(2,079	)			(987	)			(245,061	)
Foreign currency transaction (loss)/gain		(3,398	)			(15,124	)			20,550				(16,091	)
Total other income (expense), net		202,252				(7,174	)			432,116				(246,304	)
Net loss	$	(8,496,286	)		$	(4,214,061	)		$	(21,280,891	)		$	(14,194,431	)
Net loss per share – basic and diluted	$	(0.85	)		$	(0.63	)		$	(2.49	)		$	(3.84	)
Weighted average common shares outstanding – basic and diluted		10,054,482				6,637,179				8,555,039				3,694,388

 

SELECTED BALANCE SHEET DATA: 

		December 31,				December 31,
		2018				2017
Cash and cash equivalents		$	41,671,284			$	15,644,355
Other current assets		$	1,075,021			$	881,887
Property and equipment, net		$	130,867			$	62,984
Total assets		$	50,663,419			$	24,368,741
Total liabilities		$	5,580,261			$	2,033,204
Total stockholders’ equity		$	45,083,158			$	22,335,537

Investor Contact:

Hans Vitzthum

LifeSci Advisors

Ph: 617-535-7743

hans@lifesciadvisors.com

#  #  #

NEWTON, MA – January 2, 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 critical unmet medical need, today announced that it has 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. Financial terms of the license agreement were not disclosed.

“We are thrilled to expand our pipeline of product candidates by in-licensing the global rights to osanetant,” said Chris Schelling, CEO and Founder of Acer. “The drug perfectly fits Acer’s acquisition and development model of de-risked assets – it already has robust non-clinical, pharmacokinetic and human safety data, and based on recent studies involving antagonism of the NK3 receptor, we believe it can be successfully repurposed to treat a variety of neuroendocrine disorders.” Mr. Schelling concluded, “We very much appreciate the opportunity to expand our relationship with Sanofi.”

“We are pleased to sign this agreement with Acer, which illustrates our strategy of collaborating with partners who bring a credible repurposing strategy to non-core molecules in our broad portfolio,” said Alban De-La-Sabliere, Global Head of Business Development and Licensing at Sanofi.  “By providing access to these select R&D programs, we can continue to support efforts to address serious unmet medical need.”

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 critical unmet medical need. Acer’s pipeline now 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 (UCD) and Maple Syrup Urine Disease (MSUD), and osanetant for the treatment of various neuroendocrine disorders. Acer’s product candidates are de-risked, 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.

For more information, visit www.acertx.com.

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 capitalization and 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; the ability to protect our intellectual property rights; the nature, strategy and our focus; future economic conditions or performance; 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 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

 

#  #  #

Acer continues progress toward goal of commercializing EDSIVO™

 

NEWTON, MA – December 26, 2018 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and ultra-rare diseases with critical unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review Acer’s New Drug Application (NDA) for EDSIVO™ for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. The FDA also granted a priority review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 25, 2019. Priority review is a designation granted by the FDA to accelerate the review process for drugs that offer a significant improvement in treatment or provide treatment where no satisfactory alternative therapy exists.

 

“The acceptance of our NDA for EDSIVO™ is an important step in our efforts to help patients with vEDS, who suffer with a devastating disease that currently has no approved treatment,” said William Andrews, M.D., FACP, Chief Medical Officer of Acer. “We have had the honor of learning about the significant challenges of living with vEDS directly from patients and their families. This has in large part driven the hard work, passion and complete dedication that our small team has given to this effort, and we will continue to do so as the FDA reviews our NDA for EDSIVO™. We are excited about the possibility of making EDSIVO™ available in the U.S. for patients in the near future.”

 

“We continue to accelerate our pre-commercial activities supporting the potential U.S. launch of EDSIVO™ for the treatment of vEDS if it is approved by the FDA,” said Chris Schelling, CEO and Founder of Acer. “Additionally, we are working diligently on advancing and expanding our pipeline with the goal of bringing multiple products to patients with serious rare diseases over the next several years.”

 

About EDSIVO™ and vEDS

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 average mortality is 51 years of age. 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 celiprolol⁽¹⁾. 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 patients with serious rare and ultra-rare diseases with critical unmet medical need. Acer’s late-stage clinical pipeline includes two candidates for severe genetic disorders: EDSIVO™ (celiprolol) for vascular Ehlers-Danlos syndrome (vEDS), and ACER-001 (a fully taste-masked, immediate release formulation of sodium phenylbutyrate) for urea cycle disorders (UCD) and Maple Syrup Urine Disease (MSUD). There are no FDA-approved drugs for vEDS and MSUD and limited options for UCD, which collectively impact approximately 7,000 patients in the U.S. Acer’s product candidates have clinical proof-of-concept and mechanistic differentiation, and Acer intends to seek approval for them in the U.S. by 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.

 

For more information, visit www.acertx.com.

 

References

⁽¹⁾ Ong KT, et al. Effect of celiprolol on prevention of cardiovascular events in vascular Ehlers-Danlos syndrome: a prospective randomized, open, blinded-endpoints trial. Lancet. 2010; 376: 1476–84.

 

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 capitalization and resources; the potential for EDSIVO™ (celiprolol) and ACER-001 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; the ability to protect our intellectual property rights; the nature, strategy and our focus; future economic conditions or performance; 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 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

Acer continues to progress towards goal of commercializing EDSIVO™

 

Company appoints VPs of Corporate Development and IT & Database Administrator

 

NEWTON, MA – November 9, 2018 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and ultra-rare diseases with critical unmet medical need, today reported financial results for the third quarter ended September 30, 2018 and provided an update on the Company’s recent corporate developments. The Company also announced that it has appointed Ms. Jeannie Kim as Vice President, Corporate Development; and Mr. Jay Robinson as Vice President, IT & Database Administrator.

 

“We have been quite active in the third quarter preparing for the opportunities ahead.  We strengthened our balance sheet with a successful financing in August. This raise has allowed us to accelerate our pre-commercial activities supporting the potential U.S. launch of EDSIVO™ for the treatment of vascular Ehlers-Danlos syndrome (vEDS). Additionally, we are working diligently on advancing and expanding our pipeline with the goal of bringing multiple products to patients with serious orphan diseases over the next several years,” said Chris Schelling, CEO and Founder of Acer.

 

Mr. Schelling continued, “As part of our pipeline expansion efforts and pre-commercial preparation, we have made two important senior-level hires. Our new team members bring deep industry knowledge and experience across corporate development and IT / database administration, which will be invaluable as we move forward. We warmly welcome Jeannie, who will report to our Chief Legal Officer, and Jay, who will report to our Chief Operating and Financial Officer.”

 

Third Quarter 2018 and Recent Highlights

• Submitted NDA for EDSIVO™ (celiprolol) for the treatment of vEDS to the FDA in October 2018
• Entered into an additional agreement with Assistance Publique—Hôpitaux de Paris (“AP-HP”) in September 2018, pursuant to which we obtained the exclusive worldwide intellectual property rights to three European patent applications relating to certain uses of celiprolol including (i) the optimal dose of celiprolol in treating vEDS patients, (ii) the use of celiprolol during pregnancy, and (iii) the use of celiprolol to treat kyphoscoliotic Ehlers-Danlos syndrome (type VI). We subsequently filed three U.S. patent applications on this subject matter.
• Raised $46 million, including underwriters’ full exercise of their option to purchase additional shares to cover over-allotments, through an underwritten public offering in August 2018
• Ended third quarter with $46.2 million in cash and cash equivalents and no debt, which we believe will be sufficient to fund our current operating and capital requirements into the first half of 2020
• Expanded management team by hiring Jeannie Kim as Vice President, Corporate Development; and Jay Robinson as Vice President, IT & Database Administrator

 

Upcoming Milestones

• Potential acceptance of NDA for filing by the end of 2018 and grant of priority review for EDSIVO™ by the FDA, which if granted could result in a Prescription Drug User Fee Act (PDUFA) action date of late second quarter 2019
• Potential publication of celiprolol vEDS Patient Registry data, which is currently under peer review
• Continuing pre-commercial activities for EDSIVO™
• Continued efforts toward advancing and expanding our pipeline
• Continued efforts toward building out the management team with additional senior-level commercial hires, as well as adding to the commercial and medical affairs teams and other core personnel

 

Financial Results for the Third Quarter 2018

 

Cash position. Cash and cash equivalents were $46.2 million as of September 30, 2018, compared to $15.6 million as of December 31, 2017. We believe our cash position will be sufficient to fund our current operating and capital requirements into the first half of 2020.

 

Research and Development Expenses. Research and development expenses were $2.4 million during the three months ended September 30, 2018, compared to $2.1 million during the three months ended September 30, 2017. This increase of $0.3 million was principally due to increases in spending for employee-related costs and regulatory consulting, partially offset by a decrease in contract research and manufacturing services, all relating to EDSIVO™. Research and development expense for the three months ended September 30, 2018 was primarily comprised of approximately $2.2 million related to EDSIVO™ and $0.2 million related to ACER-001.

 

General and Administrative Expenses. General and administrative expenses were $1.7 million for the three months ended September 30, 2018 compared to $1.3 million for the three months ended September 30, 2017. This increase of $0.4 million was primarily due to an increase in employee-related costs and pre-commercial costs, partially offset by lower legal and consulting related expenses.

 

Net Loss. Net loss for the three months ended September 30, 2018 was $4.0 million, or $0.43 loss per share (basic and diluted), compared to a net loss of $3.5 million, or $1.09 loss per share (basic and diluted), for the three months ended September 30, 2017. The decrease in loss per share (basic and diluted) was driven largely by an increase in shares outstanding at September 30, 2018 compared to September 30, 2017.

 

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

 

 

 

About Jeannie Kim

Ms. Kim joined Acer as Vice President, Corporate Development in October 2018. She brings over 20 years of experience in the global health care industry across pharmaceuticals and its growing integration across multiple life science verticals, from ‘-omics’ diagnostics in precision/translational medicine and digital health to clinical advancements through drug delivery innovations. During her career, Ms. Kim has successfully driven over 50 agreements and alliance ecosystems across oncology, infectious disease, autoimmune and metabolic disease. Her background includes expertise in strategic planning, marketing, portfolio development and continuous improvement methods. Prior to joining Acer, Ms. Kim held multiple leadership roles in business/corporate development, strategic alliances and commercialization at several organizations including BioElectron (private start-up in ultra-rare/orphan disease), Thermo Fisher Scientific, PATH (Global Health NGO), Nektar /Novartis, Philips Medical, Alza/Johnson & Johnson and GD Searle/Pharmacia/Pfizer. She began her career as an engineer designing surgical instruments and peritoneal dialysis products for Baxter International. Ms. Kim received a B.S. degree in Biomedical Mechanical Engineering and a Masters in Engineering Management from Northwestern University, and an M.B.A. from Kellogg School of Management.

 

About Jay Robinson

Mr. Robinson joined Acer as Vice President, IT & Database Administrator in September 2018. He has over 20 years of information technology experience with expertise ranging from business transformation to cloud to strategic sourcing. Prior to joining Acer, Mr. Robinson was a Director at Apps Associates where during his tenure he operationalized two strategic offerings (Oracle EBS R12 migration and Application Managed Services) with responsibilities that spanned business development, financial management and operations. Prior to Apps Associates, he held roles spanning business analysis through project management in technology companies such as Oracle, CSC, Akamai and Siemens. Mr. Robinson received a B.S. degree in Accounting from Boston College and a M.S. degree in Information Technology from Bentley University.

 

About Acer Therapeutics

Acer, headquartered in Newton, MA, is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for patients with serious rare and ultra-rare diseases with critical unmet medical need. Acer’s late-stage clinical pipeline includes two candidates for severe genetic disorders: EDSIVO™ (celiprolol) for vascular Ehlers-Danlos syndrome (vEDS), and ACER-001 (a fully taste-masked, immediate release formulation of sodium phenylbutyrate) for urea cycle disorders (UCD) and Maple Syrup Urine Disease (MSUD). There are no FDA-approved drugs for vEDS and MSUD and limited options for UCD, which collectively impact approximately 7,000 patients in the U.S. Acer’s product candidates have clinical proof-of-concept and mechanistic differentiation, and Acer intends to seek approval for them in the U.S. by 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.

 

For more information, visit www.acertx.com.

 

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 capitalization and resources; the potential for EDSIVO™ (celiprolol) and ACER-001 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; the ability to protect our intellectual property rights; the nature, strategy and our focus; future economic conditions or performance; 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 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 September 30,
		2018				2017
Operating expenses:
Research and development		$	2,377,916			$	2,057,421
General and administrative			1,729,446				1,302,401
Total operating expenses			4,107,362				3,359,822
Loss from operations			(4,107,362	)			(3,359,822	)
Other income (expense):
Interest income			138,671				2,993
Interest expense			—				(120,229	)
Foreign currency transaction gain			3,180				—
Loss on disposal of asset			—				(967)
Total other income (expense), net			141,851				(118,203	)
Net loss		$	(3,965,511	)		$	(3,478,025	)
Net loss per share – basic and diluted		$	(0.43	)		$	(1.09	)
Weighted average common shares outstanding – basic and diluted			9,136,321				3,199,796

 

 

 

 

SELECTED BALANCE SHEET DATA: 

(unaudited)

		September 30,				December 31,
		2018				2017
Cash and cash equivalents		$	46,185,410			$	15,644,355
Other current assets		$	1,144,094			$	881,887
Property and equipment, net		$	131,288			$	62,984
Total assets		$	55,260,687			$	24,368,741
Total liabilities		$	2,154,957			$	2,033,204
Total stockholders’ equity		$	53,105,730			$	22,335,537

 

Investor Contact:

Hans Vitzthum

LifeSci Advisors

Ph: 617-535-7743

hans@lifesciadvisors.com