Acer Therapeutics and Reduction Therapeutics Announce FDA


NEWTON, Mass. and GENEVA, Oct. 06, 2021 (GLOBE NEWSWIRE) — Acer Therapeutics Inc. (Nasdaq: ACER) (“Acer”) and its collaboration companion, RELIEF THERAPEUTICS Holding SA (SIX: RLF, OTCQB: RLFTF) (“Reduction”), immediately introduced that the U.S. Meals and Drug Administration (FDA) has accepted for submitting the New Drug Utility (NDA) for ACER-001 (sodium phenylbutyrate) for the therapy of sufferers with Urea Cycle Issues (UCDs). The FDA has assigned a Prescription Drug Consumer Charge Act (PDUFA) goal motion date of June 5, 2022.

Acer’s 505(b)(2) NDA is supported by outcomes from two beforehand introduced bioequivalence (BE) trials during which ACER-001 confirmed related relative bioavailability for each phenylbutyrate (PBA) and phenylacetate (PAA), the lively metabolite of sodium phenylbutyrate, in comparison with the reference listed drug, BUPHENYL® (sodium phenylbutyrate).

“With FDA commencing a substantive evaluate of our NDA, ACER-001 is one step nearer to doubtlessly offering an alternate therapy choice for sufferers with UCDs,” stated Chris Schelling, Chief Government Officer and Founding father of Acer. “We look ahead to working with the FDA throughout their evaluate of our software. As well as, we proceed to collaborate with our companions to make sure we’re nicely positioned to assist a profitable business launch of ACER-001, topic to FDA approval.”

Jack Weinstein, Chief Monetary Officer and Treasurer of Reduction, added, “Our collaboration with Acer is prospering and we’re happy with the progress they’ve made in advancing ACER-001. In parallel with Acer’s actions, we proceed to execute on our world business technique for ACER-001 which incorporates our meant submission of a Advertising and marketing Authorization Utility (MAA) for the therapy of sufferers with UCDs in Europe in Q2/Q3 2022.”

Events within the ACER-001 program for UCDs might join updates at:
https://www.acertx.com/rare-disease-research/acer-001-for-urea-cycle-disorders-ucds/

ACER-001 is an investigational product candidate which has not been permitted by FDA, the European Medicines Company (EMA), or some other regulatory authority. There isn’t a assure that this product candidate will obtain regulatory authority approval in any territory or develop into commercially accessible for the indications underneath investigation.

About UCDs
UCDs are a bunch of problems attributable to genetic mutations that end in a deficiency in one of many six enzymes that catalyze the urea cycle, which might result in an extra accumulation of ammonia within the bloodstream, a situation referred to as hyperammonemia. Acute hyperammonemia could cause lethargy, somnolence, coma, and multi-organ failure, whereas continual hyperammonemia can result in complications, confusion, lethargy, failure to thrive, behavioral adjustments, and studying and cognitive deficits. Frequent signs of each acute and continual hyperammonemia additionally embody seizures and psychiatric signs.1,2 The present therapy of sufferers with UCDs consists of dietary administration to restrict ammonia manufacturing along with drugs that present various pathways for the elimination of ammonia from the bloodstream. Some sufferers may additionally require particular person branched-chain amino acid supplementation.

Present medical remedies for sufferers with UCDs embody nitrogen scavengers, RAVICTI® and BUPHENYL®, during which the lively pharmaceutical substances are glycerol phenylbutyrate and sodium phenylbutyrate, respectively. In keeping with a 2016 research by Shchelochkov et al., revealed in Molecular Genetics and Metabolism Studies, whereas nitrogen scavenging drugs have been proven to be efficient in serving to to handle ammonia ranges in some sufferers with UCDs, non-compliance with therapy is frequent. Causes referenced for non-compliance related to some accessible drugs embody disagreeable style, frequency with which medicine should be taken, required variety of drugs, and the excessive value of the medicine.3

About ACER-001
ACER-001 (sodium phenylbutyrate) is being developed for the therapy of assorted inborn errors of metabolism, together with UCDs and Maple Syrup Urine Illness (MSUD). ACER-001 is a nitrogen-binding agent in improvement to be used as adjunctive remedy within the continual administration of sufferers with UCDs involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). The formulation is a multi-particulate dosage formulation for oral administration consisting of a core middle, a layer of lively drug, and a taste-masked coating designed to keep away from the bitter style of sodium phenylbutyrate within the mouth whereas shortly dissolving within the low pH of the abdomen. Acer can be being developed for MSUD and has been granted orphan drug designation by the FDA for this indication. ACER-001 is an investigational product candidate which has not been permitted by FDA, the European Medicines Company (EMA), or some other regulatory authority.

About Acer Therapeutics Inc.
Acer is a pharmaceutical firm targeted on the acquisition, improvement and commercialization of therapies for severe uncommon and life-threatening illnesses with important unmet medical wants. Acer’s pipeline consists of 4 packages: ACER-001 (sodium phenylbutyrate) for therapy of assorted inborn errors of metabolism, together with urea cycle problems (UCDs) and Maple Syrup Urine Illness (MSUD); ACER-801 (osanetant) for therapy of induced Vasomotor Signs (iVMS); EDSIVO™ (celiprolol) for therapy of vascular Ehlers-Danlos syndrome (vEDS) in sufferers with a confirmed kind III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed remedy in opposition to quite a lot of infectious illnesses, together with COVID-19. Every of Acer’s product candidates is believed to current a relatively de-risked profile, having a number of of a good security profile, medical proof-of-concept information, mechanistic differentiation and/or accelerated paths for improvement by way of particular packages and procedures established by the FDA. In March 2021, Acer entered right into a Collaboration and License Settlement with Reduction for improvement and commercialization of ACER-001. For extra data, go to www.acertx.com.

About RELIEF THERAPEUTICS Holding SA
Reduction focuses totally on clinical-stage packages based mostly on molecules with a historical past of medical testing and use in human sufferers or a powerful scientific rationale. Reduction’s lead drug candidate RLF-100™ (aviptadil), an artificial type of Vasoactive Intestinal Peptide (VIP), is in late-stage medical testing within the U.S. for the therapy of respiratory deficiency as a result of COVID-19. As a part of its pipeline diversification technique, in March 2021, Reduction entered right into a Collaboration and License Settlement with Acer Therapeutics for improvement and commercialization of ACER-001. ACER-001 is a taste-masked and rapid launch proprietary powder formulation of sodium phenylbutyrate (NaPB) for the therapy of Urea Cycle Issues and Maple Syrup Urine Illness. As well as, Reduction’s not too long ago accomplished acquisitions of APR Utilized Pharma Analysis SA and AdVita Lifescience GmbH, deliver to Reduction a various pipeline of marketed and development-stage packages, a business infrastructure in chosen European nations and an inner, R&D functionality, which Reduction hopes to leverage for each inner pipeline merchandise in addition to for third get together product improvement on a price for service foundation.

RELIEF THERAPEUTICS Holding SA is listed on the SIX Swiss Change underneath the image RLF and quoted within the U.S. on OTCQB underneath the image RLFTF. For extra data, go to
www.relieftherapeutics.com. Comply with Reduction on LinkedIn.

References

  1. Ah Mew N, et al. Urea cycle problems overview. Gene Evaluations. Seattle, Washington: College of Washington, Seattle; 1993.
  2. Häberle J, et al. Recommended tips for the analysis and administration of urea cycle problems. Orphanet Journal of Uncommon Illnesses. 2012;7(32).
  3. Shchelochkov OA, et al. Limitations to drug adherence within the therapy of urea cycle problems: Evaluation of affected person, caregiver and supplier views. Mol Genet Metab. 2016;8:43-47.

Acer Ahead-Trying Statements
This press launch incorporates “forward-looking statements” that contain substantial dangers and uncertainties for functions of the protected harbor offered by the Personal Securities Litigation Reform Act of 1995. All statements, apart from statements of historic info, included on this press launch relating to technique, future operations, timelines, future monetary place, future revenues, projected bills, regulatory submissions, actions or approvals, money place, liquidity, prospects, plans and aims of administration are forward-looking statements. Examples of such statements embody, however aren’t restricted to, statements regarding the potential for our product candidates to soundly and successfully deal with illnesses and to be permitted for advertising; the business or market alternative of any of our product candidates in any goal indication and any territory; our capability to safe the extra capital essential to fund our numerous product candidate improvement packages; the adequacy of our capital to assist our future operations and our capability to efficiently fund, provoke and full medical trials and regulatory submissions; the power to guard our mental property rights; our technique and enterprise focus; and the event, anticipated timeline and business potential of any of our product candidates. We might not truly obtain the plans, perform the intentions or meet the expectations or projections disclosed within the forward-looking statements and you shouldn’t place undue reliance on these forward-looking statements. Such statements are based mostly on administration’s present expectations and contain dangers and uncertainties. Precise outcomes and efficiency might differ materially from these projected within the forward-looking statements because of many elements, together with, with out limitation, dangers and uncertainties related to the power to venture future money utilization and reserves wanted for contingent future liabilities and enterprise operations, the supply of ample sources to fund our numerous product candidate improvement packages and to fulfill our enterprise aims and operational necessities, the truth that the outcomes of earlier research and trials is probably not predictive of future medical trial outcomes, the safety and market exclusivity offered by our mental property, dangers associated to the drug improvement and the regulatory approval course of, together with the timing and necessities of regulatory actions, and the affect of aggressive merchandise and technological adjustments. We disclaim any intent or obligation to replace these forward-looking statements to mirror occasions or circumstances that exist after the date on which they have been made. You must evaluate further disclosures we make in our filings with the Securities and Change Fee, together with our Quarterly Studies on Type 10-Q and our Annual Report on Type 10-Okay. It’s possible you’ll entry these paperwork for no cost at http://www.sec.gov.

Reduction Ahead-Trying Statements
This communication expressly or implicitly incorporates sure forward-looking statements regarding RELIEF THERAPEUTICS Holding SA and its companies. Such statements contain sure identified and unknown dangers, uncertainties and different elements, together with (i) whether or not the FDA will approve Acer’s NDA for ACER-001, (ii) whether or not Reduction will be capable to submit an software for approval of ACER-001 in Europe in Q2/Q3 2022 (or in any respect), (iii) whether or not any such software submitted to European authorities looking for advertising authorization for ACER-001 for the therapy of affected person in Europe with UCDs will probably be permitted, (iv) whether or not RLF-100 (aviptadil) will obtain emergency use authorization in the US, (v) whether or not RLF-100 (aviptadil) will ever be submitted for authorization in Europe, (vi) whether or not Reduction’s ongoing disputes with its U.S. collaboration companion for RLF-100 (aviptadil) might be resolved amicably, and (vii) these different dangers, uncertainties and elements described in Reduction’s annual and periodic filings with the SIX Inventory Change, all of which might trigger the precise outcomes, monetary situation, efficiency or achievements of RELIEF THERAPEUTICS Holding SA to be materially completely different from any future outcomes, efficiency or achievements expressed or implied by such forward-looking statements. RELIEF THERAPEUTICS Holding SA is offering this communication as of this date and doesn’t undertake to replace any forward-looking statements contained herein because of new data, future occasions or in any other case.

INVESTOR RELATIONS CONTACTS  
Acer Therapeutics:
Hans Vitzthum
LifeSci Advisors
hans@lifesciadvisors.com
+1 617-430-7578
RELIEF THERAPEUTICS Holding SA:
Michael Miller
Rx Communications Group
mmiller@rxir.com
+1-917-633-6086



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