About Us | ScientistACER Acer Therapeutics Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
|---|---|---|
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ACER-001
Urea cycle disorder (UCD)
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NDA Filing
NDA Filing
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Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
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EDSIVO (celiprolol)
Vascular Ehlers-Danlos Syndrome
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Phase 3
Phase 3
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CRL issued June 25, 2019. Phase 3 trial planned.
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Osanetant (ACER-801)
induced Vasomotor Symptoms (iVMS)
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Phase 1/2
Phase 1/2
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Phase 1/2 trial to be initiated 4Q 2021.
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Emetine
COVID-19
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Phase 2/3
Phase 2/3
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Phase 2/3 trial to be initiated 1H 2021.
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Latest News
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RALEIGH, NC / ACCESSWIRE / June 23, 2021 / Access to Giving - an investor conference themed around investor education and advocacy is set for July 13-15, 2021. There will be 50+ companies conducting virtual presentations as well as private meetings with qualified investors throughout the three-day event.
Company Presentations will begin July 13 at 10 am eastern time and go through July 15th. The event will also include a keynote speaker (to be announced) and three distinctive panels on:
- Investor Education, Advocacy and ESG
- Follow the Money - Investing 101
- Human Capital
Join us and gain an understanding and potential trends and key value drivers across a wide array of industries. If you are a qualified investor, register here and pledge to take meetings. If you're new to the investment world, come listen to some presentations, panels, and learn more.
The full agenda can be found here, and the full event website can be found here.
To receive additional information, request an invitation or to schedule a one-on-one meeting, please email Angie Goertz or call 919-228-6240.
Companies registered to date are below, and we are adding more every day.
"This conference, for us, is different than your typical investor conference," said Brian Balbirnie, CEO of Issuer Direct. "As a brand, it's important to find ways to educate and advocate and be a part of something that is bigger than all of us."
"Our technology has been behind a number of microcap and investment banking conferences and allows them to host in-person and virtual investor conferences," said Angie Goertz, Vice President of Events at Issuer Direct. "We believe this is a great opportunity to give back to our industry while helping fund investor education and financial literacy programs."
About Access to Giving
Access to Giving is the first-of-its-kind virtual investor conference and will be held July 13th - 15th, 2021. Companies will have the opportunity to tell their story and conduct 1x1's with qualified investors for charity. 100% of monies raised through donations for 1x1 meetings will be given to causes that are focused on financial literacy and financial education.About Issuer Direct Corporation
Issuer Direct® is an industry-leading communications and compliance company focusing on the needs of corporate issuers. Issuer Direct's principal platform, Platform id.™, empowers users by thoughtfully integrating the most relevant tools, technologies, and services, thus eliminating the complexity associated with producing and distributing financial and business communications. Headquartered in Raleigh, NC, Issuer Direct serves thousands of public and private companies globally. For more information, please visit www.issuerdirect.com.Contact Information:
Angie Goertz, Vice President of Events
Issuer Direct Corporation
Office: (919) 228-6240
Email: [email protected]SOURCE: Access to Giving - Issuer Direct
View source version on accesswire.com:
https://www.accesswire.com/652814/50-Companies-to-Present-at-the-Access-to-Giving-Virtual-Investor-Conference-on-July-13th--15th-2021
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Phase 3, randomized, double-blind, placebo-controlled, decentralized clinical trial estimated to take approximately 3.5 years to complete once fully enrolled
FDA indicates agreement with submission of proposed protocol under a Special Protocol Assessment (SPA) with possible breakthrough designation
NEWTON, Mass., June 10, 2021 (GLOBE NEWSWIRE) -- Acer Therapeutics Inc. (NASDAQ:ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced that following a recent Type B meeting with the U.S. Food and Drug Administration (FDA) regarding Acer's proposed EDSIVO™ (celiprolol) development plan, the company is now planning a pivotal clinical trial in patients with COL3A1+ vascular Ehlers-Danlos Syndrome (vEDS).
In the Type B meeting, Acer discussed conducting a U.S.-based prospective, randomized, double-blind, placebo-controlled, decentralized clinical trial in patients with COL3A1+ vEDS, and sought the Agency's opinion on various proposed design features of the study. The official meeting minutes Acer recently received from the FDA confirm details of that discussion, including: the acceptability of a decentralized (virtual) clinical trial design and use of an independent centralized adjudication committee; acceptability of a primary endpoint based on clinical events associated with disease outcome; agreement with modest safety data collection (based on the known safety profile of the drug1,2,3,4) and no pharmacokinetic (PK) data needing to be collected; and a statistical plan that considers the rare disease classification of vEDS.
In the second half of 2021, Acer intends to submit a protocol for the prospective study, along with an investigational new drug application (IND), request breakthrough therapy designation, and coordinate with FDA to reach agreement on a Special Protocol Assessment (SPA). Based on initial power calculations, Acer anticipates that the trial should enroll approximately 200 COL3A1+ vEDS patients in the U.S., and the duration of the clinical trial is estimated to be approximately 3.5 years to complete once fully enrolled.
"We appreciate the guidance and flexibility provided by FDA on the design of this proposed clinical trial," said Chris Schelling, CEO and Founder of Acer. "This trial, along with the results from the prior BBEST trial1, should answer whether EDSIVO™ demonstrates substantial evidence of effectiveness by reducing vEDS-related events. Over the next six months, we intend to work closely with FDA to finalize the protocol for the pivotal trial, collaborate with vEDS advocacy groups to identify COL3A1+ patients who may be interested in participating in the trial in order to expedite enrollment, and achieve first patient dosed as quickly as possible. Our progress to date on the EDSIVO™ program is the direct result of our unwavering and ongoing commitment to patients with rare diseases. We look forward to continuing its development for treatment of vEDS patients who desperately need a therapy."
For more information on the trial as it becomes available, please visit: www.discoverceliprolol.com.
The conduct and completion of the EDSIVO™ pivotal clinical trial is subject to Acer's ability to obtain up to approximately $10 million of funding. Such funding may come from one or more of a number of current and future sources, including but not limited to current cash, a strategic partnership, other non-dilutive sources and potential ACER-001 revenue. Acer believes its cash and cash equivalents available as of March 31, 2021, plus up to $20.0 million of Development Payments per the ACER-001 Collaboration Agreement with Relief Therapeutics, are sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for planned EDSIVO™ and osanetant clinical trials.
EDSIVO™ is an investigational drug and is not currently FDA approved for any indication. There can be no assurance that the funding to conduct and complete the clinical trial will be obtained, that the clinical trial will be successful, or that the resubmission of the original New Drug Application will be approved.
About Breakthrough Therapy Designation
The FDA's breakthrough therapy designation is intended to expedite the development and review of new treatments for serious or life-threatening conditions. The designation entitles the company developing a therapy to more intensive FDA guidance on an efficient and accelerated development program, as well as eligibility for other actions to expedite FDA review, such as rolling submission and priority review. To earn breakthrough designation, a treatment must show encouraging early clinical results demonstrating substantial improvement over available therapies with regard to a clinically significant endpoint, or it must fulfill an unmet need.About vEDS
Ehlers-Danlos syndrome (EDS) is an inherited disorder caused by mutations in the genes responsible for the structure, production, or processing of collagen, an important component of the connective tissues in the human body, or proteins that interact with collagen. EDS is a spectrum disorder where patients present with various forms, the most serious of which is vEDS, also known as EDS type IV, which is generally caused by a mutation in the COL3A1 gene. vEDS causes abnormal fragility in blood vessels, which can give rise to aneurysms, abnormal connections between blood vessels known as arteriovenous fistulas, arterial dissections, and spontaneous vascular ruptures, all of which can be potentially life-threatening. Gastrointestinal and uterine fragility or rupture also commonly occur in vEDS patients. Spontaneous arterial rupture has a peak incidence in the third or fourth decade of life in vEDS patients but may occur earlier and is the most common cause of sudden death in vEDS patients. Arterial rupture or dissection events occur in about 25% of patients before the age of 20 but increase to roughly 90% of patients by the age of 40. The median survival age of vEDS patients in the U.S. is 51 years, with arterial rupture being the most common cause of sudden death.5About EDSIVO™ (celiprolol)
Acer is developing EDSIVO™, a new chemical entity (NCE), for the treatment of COL3A1+ vEDS patients. Acer's EDSIVO™ NDA was originally submitted based on data obtained from the BBEST trial1 and accepted for filing in October 2018 with priority review. Following FDA review, Acer received a Complete Response Letter (CRL) stating that it will be necessary to conduct an adequate and well-controlled trial to determine whether celiprolol reduces the risk of clinical events in patients with vEDS. Acer subsequently appealed the FDA decision. While FDA denied the appeal, it described possible paths forward for Acer to explore. Following its Type B meeting with FDA in the second quarter of 2021, Acer has decided to conduct a clinical trial for EDSIVO™. There can be no assurance that the resulting data from the trial would be adequate to support approval of an NDA, although having a SPA agreement in place would indicate concurrence by FDA with the adequacy and acceptability of specific critical elements of overall protocol design (e.g., conduct, entry criteria, dose selection, endpoints, and planned analyses) for a study intended to support a future marketing application.About Acer Therapeutics Inc.
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer's pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer's product candidates is 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/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for development and commercialization of ACER-001. For more information, visit www.acertx.com.References
- Ong KT, 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(9751):1476-1484.
- Nawarskas J, et al. Cardiology in Review 2017;25: 247–253.
- Frank M, et al. Vascular Ehlers-Danlos Syndrome: Long-Term Observational Study. J Am Coll Cardiol. 2019 Apr, 73 (15) 1948–1957.
- Baderkhan, H, et al. Celiprolol Treatment in Patients with Vascular Ehlers-Danlos Syndrome. European Journal of Vascular and Endovascular Surgery. November 20, 2020.
- 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.
Forward-Looking Statements
This press release contains "forward-looking statements" that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, timelines for clinical study enrollment or regulatory actions, or otherwise, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions for EDSIVO™ or our other products; 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 fund our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, the substantial costs and diversion of management's attention and resources which could result from pending securities litigation, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our 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: 617-430-7578
[email protected]Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected]
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NEWTON, Mass. and GENEVA, Switzerland, May 25, 2021 (GLOBE NEWSWIRE) -- Acer Therapeutics Inc. (NASDAQ:ACER)("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, and RELIEF THERAPEUTICS Holding AG ((SIX: RLF, OTCQB:RLFTF)("Relief"), a biopharmaceutical company with its lead compound RLF-100TM (aviptadil) in advanced clinical development to treat severe COVID-19 patients, today announced the outcome of Acer's pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) for ACER-001 for the treatment of Urea Cycle Disorders (UCDs). ACER-001 is a proprietary immediate release multi-particulate powder formulation of sodium phenylbutyrate (NaPB) with a taste-masked coating. ACER-001 is being developed in collaboration with Relief.
The purpose of the pre-NDA meeting was to discuss the content of Acer's planned NDA submission. Based on FDA feedback, the Companies believe the proposed data package will be sufficient to support an NDA submission under the Section 505(b)(2) regulatory pathway of ACER-001 for the treatment of patients with UCDs. Following NDA submission and FDA determination of acceptance for filing, the FDA will conduct a substantive review before deciding upon the action on the application.
"We are pleased with the outcome of our recent pre-NDA meeting with FDA, supporting our belief that the studies and data we intend to include in our planned NDA for ACER-001 should be sufficient for NDA submission," said Chris Schelling, CEO and Founder of Acer. "We remain on track to complete the NDA submission in Q3 2021, provided that we obtain agreement with the FDA on our initial pediatric study plan (iPSP)."
Jack Weinstein, CFO and Treasurer of Relief Therapeutics, added: "The outcome of the pre-NDA meeting with the FDA is highly encouraging and marks an important milestone in support of the development and potential commercialization of ACER-001 worldwide. We are pleased with the progress of the ACER-001 program and look forward to working with the Acer team to potentially bring to market an alternative to existing NaPB-based treatments."
ACER-001 is an investigational product candidate which has not been approved by FDA. There can be no assurance that if submitted, an NDA will be accepted for filing and substantive review or, if filed, that an NDA would be approved.
About UCDs
UCDs are a group of disorders caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia. Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits. Common symptoms of both acute and chronic hyperammonemia also include seizures and psychiatric symptoms.1,2 The current treatment of UCDs consists of dietary management to limit ammonia production in conjunction with medications that provide alternative pathways for the removal of ammonia from the bloodstream. Some patients may also require individual branched-chain amino acid supplementation.Current medical treatments for UCDs include nitrogen scavengers, RAVICTI® and BUPHENYL®, in which the active pharmaceutical ingredients are glycerol phenylbutyrate (GPB) and sodium phenylbutyrate (NaPB), respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications have been shown to be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons referenced for non-compliance associated with some available medications include unpleasant taste, the frequency with which medication must be taken, the number of pills, and the high cost of the medication.3
About ACER-001
ACER-001 is being developed for the treatment of various inborn errors of metabolism, including UCDs and MSUD. ACER-001 is a proprietary immediate release powder formulation of sodium phenylbutyrate (NaPB). The formulation is a multi-particulate dosage formulation for oral administration consisting of a core center, a layer of active drug, and a taste-masked coating designed to avoid the bitter taste in the mouth while quickly dissolving in the low pH of the stomach. ACER-001's taste-masked formulation is aimed to improve the palatability of NaPB. Acer has been granted orphan drug designation by the FDA for the MSUD indication. ACER-001 is under clinical investigation and its safety and efficacy have not been established. There is no guarantee that this product candidate will receive U.S. FDA approval or become commercially available for the indications under investigation.About Acer Therapeutics Inc.
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer's pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer's product candidates is 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/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for the worldwide development and commercialization of ACER-001. For more information, visit www.acertx.com.About RELIEF THERAPEUTICS Holding AG
Relief focuses primarily on clinical-stage programs based on molecules with a history of clinical testing and use in human patients or a strong scientific rationale. Relief's lead drug candidate RLF-100™ (aviptadil), a synthetic form of Vasoactive Intestinal Peptide (VIP), is in late-stage clinical testing in the U.S. for the treatment of respiratory deficiency due to COVID-19. As part of its pipeline diversification strategy, in March 2021, Relief entered into a Collaboration and License Agreement with Acer Therapeutics for the worldwide development and commercialization of ACER-001. ACER-001 is a taste-masked and immediate release proprietary powder formulation of sodium phenylbutyrate (NaPB) for the treatment of Urea Cycle Disorders and Maple Syrup Urine Disease.RELIEF THERAPEUTICS Holding AG is listed on the SIX Swiss Exchange under the symbol RLF and quoted in the U.S. on OTCQB under the symbol RLFTF. For more information, visit
www.relieftherapeutics.com. Follow Relief on LinkedIn.References
- Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University of Washington, Seattle; 1993.
- Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
- Shchelochkov OA, et al. Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives. Mol Genet Metab. 2016;8:43-47.
Acer Forward-Looking Statements
This press release contains "forward-looking statements" that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; 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 fund our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, the substantial costs and diversion of management's attention and resources which could result from pending securities litigation, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our 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.Relief Forward-Looking Statements
This communication expressly or implicitly contains certain forward-looking statements concerning RELIEF THERAPEUTICS Holding AG and its businesses. The results reported herein may or may not be indicative of the results of future and larger clinical trials for ACER-001 for the treatment of UCDs and MSUD, nor whether the ongoing clinical trials of Relief's lead compound, RLF-100™ (aviptadil) in advanced clinical development to treat respiratory deficiency due to COVID-19, will be successful. Such statements involve certain known and unknown risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of RELIEF THERAPEUTICS Holding AG to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. RELIEF THERAPEUTICS Holding AG is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise.CORPORATE CONTACTS
Acer Therapeutics:
Jim DeNike
Acer Therapeutics Inc.
+1 844-902-6100
[email protected]RELIEF THERAPEUTICS Holding AG:
Jack Weinstein
Chief Financial Officer and Treasurer
[email protected]MEDIA CONTACTS
Relief (Europe):
Anne Hennecke
MC Services AG
[email protected]
+49 (0) 211-529-252-22INVESTOR RELATIONS CONTACTS
Acer Therapeutics:
Hans Vitzthum
LifeSci Advisors
+1 617-430-7578
[email protected]Relief (Europe):
Anne Hennecke
MC Services AG
[email protected]
+49 (0) 211-529-252-22 -
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NEWTON, Mass., May 17, 2021 (GLOBE NEWSWIRE) -- Acer Therapeutics Inc. (NASDAQ:ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today reported financial results for the first quarter ended March 31, 2021 and provided an update on the Company's recent corporate developments.
"We made significant progress in advancing our pipeline programs and strengthening our balance sheet in Q1 2021," said Chris Schelling, CEO and Founder of Acer. "Regarding ACER-001, we executed the Collaboration and License Agreement with Relief Therapeutics; we conducted a pre-NDA meeting with the FDA and are awaiting FDA meeting minutes; and we remain on track to submit an NDA in mid-2021 for ACER-001 as a treatment for UCDs, provided that no additional data are requested by the FDA and we reach agreement on the Pediatric Study Plan (PSP). In addition, we conducted a Type B meeting with the FDA to discuss our proposed path forward for EDSIVO™, and our dialogue is ongoing. We continue to prepare and target an IND submission for ACER-801 (osanetant) in late Q3 2021. Finally, we continue to seek non-dilutive capital to advance development of ACER-2820 (emetine)."
Q1 2021 and Recent Highlights
- ACER-001
- Announced in February 2021 topline results from Acer's bioequivalence trial in which ACER-001 showed similar relative bioavailability compared to BUPHENYL® (sodium phenylbutyrate) under fed conditions
- Announced in March 2021 that Acer and Relief Therapeutics Holding AG (Relief) entered into a Collaboration and License Agreement (Collaboration Agreement) for worldwide development and commercialization of ACER-001. Under the terms of the agreement, Relief has paid Acer $20 million, and will pay Acer up to an additional $21 million in development expense reimbursement and milestones plus royalties in exchange for net profit share and territory rights
- Conducted a Type B pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) – awaiting FDA meeting minutes
- EDSIVO™ (celiprolol)
- Conducted a Type B meeting with the FDA to discuss Acer's proposed plan to collect additional data with the goal of providing further evidence in support of celiprolol's potential benefit in treating COL3A1-positive vEDS patients – awaiting FDA meeting minutes
- ACER-801 (osanetant)
- Continued to advance development activities in support of an Investigational New Drug Application (IND) submission
- Corporate
- Hired Bill DeVincenzi in March 2021 as Vice President, Quality. Mr. DeVincenzi brings to Acer 29 years of experience in quality assurance, supporting the development and commercialization of several small-molecule pharmaceuticals across a wide spectrum of dosage forms
- Ended Q1 2021 with $15.9 million in cash and cash equivalents. Acer believes its cash and cash equivalents available as of March 31, 2021, plus up to $20.0 million of Development Payments per the Collaboration Agreement with Relief, are sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for a planned osanetant clinical trial
Anticipated Milestones
- ACER-001
- Q2 2021: Awaiting minutes from the Type B pre-NDA meeting with the FDA
- Mid-2021: ACER-001 NDA submission for treatment of patients with Urea Cycle Disorders (UCDs) is anticipated in mid-2021, provided that no additional data are requested by the FDA and ongoing development activities are successfully completed (including evaluation of product stability data and reaching agreement on the Pediatric Study Plan (PSP))
- EDSIVO™
- Q2 2021: Awaiting minutes from the Type B meeting with the FDA
- ACER-801 (osanetant)
- Q3 2021: IND submission for osanetant is anticipated in late Q3 2021
- Q4 2021: Clinical trial initiation is expected in late Q4 2021 designed to evaluate osanetant's PK/PD and safety and to identify the optimal dosing strategy for future efficacy studies in patients with induced Vasomotor Symptoms (iVMS), dependent upon successful IND submission and clearance, and subject to additional capital
- ACER-2820 (emetine)
- Ongoing: The Company believes that most of the emetine IND-enabling work is complete. Further advancement of the emetine program for treatment of certain infectious diseases, including COVID-19, is dependent on Acer's ability to raise non-dilutive capital
Q1 2021 Financial Results
Cash position. Cash and cash equivalents were $15.9 million as of March 31, 2021, compared to $5.8 million as of December 31, 2020. Acer believes its cash and cash equivalents available as of March 31, 2021, plus up to $20.0 million of Development Payments per the Collaboration Agreement with Relief, are sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for a planned osanetant clinical trial.
Revenue. Acer recognized revenue of $4.0 million during the three months ended March 31, 2021, related to the license of intellectual property as part of the Collaboration Agreement signed during the quarter.
Research and Development Expenses. Research and development expenses were $2.0 million for the three months ended March 31, 2021, as compared to $2.3 million for the three months ended March 31, 2020. An increase in employee-related expenses, which was driven by bonuses expensed during the quarter, was offset by a decrease in expenses for contract research and contract manufacturing, as well as an offset of $0.3 million associated with the recognition of collaboration funding from the Collaboration Agreement with Relief. Research and development expenses for the three months ended March 31, 2021 were comprised of $1.2 million related to ACER-001 offset by $0.3 million of collaboration funding, $0.5 million related to osanetant, $0.4 million related to emetine, and $0.2 million related to other development activities.
General and Administrative Expenses. General and administrative expenses were $3.5 million for the three months ended March 31, 2021, as compared to $2.6 million for the three months ended March 31, 2020. This increase of $0.9 million was primarily due to an increase in employee-related expenses, driven by bonuses expensed during the quarter, partially offset by a decrease in expenses related to precommercial activities.
Net Loss. Net loss for the three months ended March 31, 2021 was $1.5 million, or $0.11 net loss per share (basic and diluted), compared to a net loss of $4.9 million, or $0.49 net loss per share (basic and diluted), for the three months ended March 31, 2020.
For additional information, please see Acer's Quarterly Report on Form 10-Q filed today with the SEC.
About Acer Therapeutics Inc.
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer's pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer's product candidates is 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/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for worldwide development and commercialization of ACER-001. 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, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; 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 fund our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, the substantial costs and diversion of management's attention and resources which could result from pending securities litigation, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our 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: 617-430-7578
[email protected]Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected]ACER THERAPEUTICS INC.
CONSOLIDATED STATEMENTS OF OPERATIONS
(Unaudited)
Three Months Ended March 31, 2021 2020 Revenue $ 3,998,133 $ — Operating expenses: Research and development 2,005,905 2,322,905 General and administrative 3,514,141 2,648,551 Loss from operations (1,521,913 ) (4,971,456 ) Other income (expense), net: Interest and other income (expense), net (40,163 ) 25,742 Foreign currency transaction gain (loss) 48,904 (2,993 ) Total other income (expense), net 8,741 22,749 Net loss $ (1,513,172 ) $ (4,948,707 ) Net loss per share - basic and diluted $ (0.11 ) $ (0.49 ) Weighted average common shares outstanding - basic and diluted 14,139,916 10,097,107 SELECTED BALANCE SHEET DATA (Unaudited):
March 31, December 31, 2021 2020 Cash and cash equivalents $ 15,900,297 $ 5,761,568 Accounts receivable $ 10,000,000 $ — Prepaid expenses and other current assets $ 10,507,161 $ 679,461 Property and equipment, net $ 120,769 $ 130,081 Total assets $ 44,510,898 $ 14,613,688 Deferred collaboration funding $ 20,715,764 $ — Total liabilities $ 34,101,253 $ 6,389,354 Total stockholders' equity $ 10,409,645 $ 8,224,334 
- ACER-001
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NEWTON, Mass., March 29, 2021 (GLOBE NEWSWIRE) -- Acer Therapeutics Inc. (NASDAQ:ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced that Acer's management team will present and host one-on-one investor meetings at the upcoming 20th Annual Needham Virtual Healthcare Conference April 12-15, 2021.
Conference: 20th Annual Needham Virtual Healthcare Conference Format: Virtual presentation and one-on-one meetings Presentation: April 15, 2021 Time: 11 a.m. Eastern Time Webcast: https://acertx.com/investor-relations/events-presentations/ About Acer Therapeutics Inc.
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer's pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer's product candidates is 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/or accelerated paths for development through specific programs and procedures established by the U.S. FDA. On March 19, 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for worldwide development and commercialization of ACER-001. For more information, visit www.acertx.com.Investor Contact:
Hans Vitzthum
LifeSci Advisors
Ph: 617-430-7578
[email protected]Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected]