Acurx Announces Positive Opinion from EMA on Pediatric Investigation Plan for Ibezapolstat Use in Children with C. difficile Infection
Acurx Pharmaceuticals (NASDAQ:ACXP) has received a positive opinion from the European Medicines Agency's (EMA) Paediatric Committee (PDCO) on its Pediatric Investigation Plan (PIP) for ibezapolstat in treating children with C. difficile infection (CDI). This marks a significant regulatory milestone as the company prepares for Phase 3 clinical trials.
The company has already secured FDA QIDP and Fast-Track Designation, along with EMA's SME designation. Both the FDA and EMA are aligned with Acurx's clinical trial program for adults, with clear regulatory pathways established for EU MAA and U.S. NDA submissions. The development addresses a critical need, as CDI affects 20,000 children annually, with a 20-30% recurrence rate.
Upon successful completion, ibezapolstat could receive 11 years of marketing exclusivity in Europe, including an additional year on top of the standard 10-year period for new antibiotics.
[ "Positive EMA opinion received for pediatric investigation plan (PIP)", "FDA QIDP and Fast-Track Designation already secured", "Potential for 11 years of marketing exclusivity in Europe", "Clear regulatory pathway established with both FDA and EMA", "Addresses significant unmet need in pediatric CDI treatment" ]Acurx Pharmaceuticals (NASDAQ:ACXP) ha ottenuto un parere favorevole dall\'EMA Paediatric Committee (PDCO) sul Piano di Indagine Pediatrica (PIP) per ibezapolstat nel trattamento dei bambini con infezione da C. difficile (CDI). Si tratta di una tappa regolamentare significativa mentre l\'azienda si prepara per i trial di fase 3. L\'azienda ha già ottenuto FDA QIDP e Designazione Fast-Track, insieme alla designazione SME dell\'EMA. Sia FDA che EMA sono allineate con il programma di trial clinici per adulti, con percorsi regolatori chiari per le submission EU MAA e US NDA. Lo sviluppo risponde a una necessità critica, poiché CDI colpisce 20.000 bambini all\'anno, con un tasso di ricorrenza del 20-30%. Al completamento con successo, ibezapolstat potrebbe ottenere 11 anni di esclusività di marketing in Europa, inclusa una anno aggiuntivo oltre il periodo standard di 10 anni per i nuovi antibiotici.
Acurx Pharmaceuticals (NASDAQ:ACXP) ha recibido una opinión positiva del Comité de Pediatría de la Agencia Europea de Medicamentos (EMA) sobre su Plan de Investigación Pediátrica (PIP) para ibezapolstat en el tratamiento de niños con infección por C. difficile (CDI). Esto marca un hito regulatorio significativo mientras la empresa se prepara para ensayos clínicos de fase 3. La empresa ya ha obtenido QIDP de la FDA y Designación de Fast Track, junto con la designación SME de la EMA. Tanto la FDA como la EMA están alineadas con el programa de ensayos clínicos para adultos, con rutas regulatorias claras para las presentaciones EU MAA y US NDA. El desarrollo aborda una necesidad crítica, ya que la CDI afecta a 20,000 niños al año, con una tasa de recurrencia del 20-30%. Una vez completado con éxito, ibezapolstat podría recibir 11 años de exclusividad de comercialización en Europa, incluida una año adicional además del periodo estándar de 10 años para nuevos antibióticos.
Acurx Pharmaceuticals (NASDAQ:ACXP)가 C. difficile 감염(CDI)을 앓고 있는 어린이 치료를 위한 ibezapolstat의 소아 연구계획(PIP)에 대해 유럽 의약품청(EMA) 산하 페디아트릭 위원회(PDCO)로부터 긍정적 의견을 받았습니다. 이는 회사가 3상 임상을 준비하는 동안 중요한 규제 이정표가 됩니다. 회사는 이미 FDA의 QIDP 및 Fast-Track 지정을 확보했으며, EMA의 SME 지정도 받았습니다. FDA와 EMA는 성인용 임상시험 프로그램에 대해 일치하고 있으며 EU MAA 및 US NDA 제출에 대한 명확한 규제 경로가 확립되어 있습니다. 이 개발은 CDI가 매년 20,000명의 아동에게 영향을 미치고 재발률이 20-30%인 중요한 수요를 다룹니다. 성공적으로 완료되면 ibezapolstat은 유럽에서 11년의 마케팅 독점권을 얻을 수 있으며, 신형 항생제의 표준 10년 기간 외에 추가로 1년이 포함됩니다.
Acurx Pharmaceuticals (NASDAQ:ACXP) a reçu un avis favorable du Comité Pédiatrique (PDCO) de l\'Agence européenne des médicaments (EMA) sur son Plan d\'Investigation Pédiatrique (PIP) pour ibezapolstat dans le traitement des enfants atteints d\'infection à C. difficile (CDI). Cela marque une étape réglementaire clé alors que l\'entreprise se prépare à des essais de phase 3. L\'entreprise a déjà obtenu la QIDP de la FDA et la désignation Fast-Track, ainsi que la désignation SME de l\'EMA. FDA et EMA sont alignées sur le programme d\'essais cliniques pour les adultes, avec des parcours réglementaires clairs pour les soumissions EU MAA et US NDA. Le développement répond à un besoin critique, car la CDI affecte 20 000 enfants par an, avec un taux de récurrence de 20-30%. À l\'issue d\'un succès, ibezapolstat pourrait bénéficier de 11 ans d\'exclusivité marketing en Europe, incluant une année supplémentaire au-delà des 10 ans standard pour les nouveaux antibiotiques.
Acurx Pharmaceuticals (NASDAQ:ACXP) hat eine positive Stellungnahme des Pädiatrie-Ausschusses (PDCO) der Europäischen Arzneimittelagentur (EMA) zu ihrem Pediatric Investigation Plan (PIP) für ibezapolstat bei der Behandlung von Kindern mit C. difficile-Infektion (CDI) erhalten. Dies markiert einen bedeutenden regulatorischen Meilenstein, während das Unternehmen sich auf Phase-3-Studien vorbereitet. Das Unternehmen hat bereits FDA QIDP- und Fast-Track-Bewertung erhalten, zusammen mit der EMA-SME-Bezeichnung. Sowohl die FDA als auch die EMA stimmen dem klinischen Prüfprogramm für Erwachsene zu, mit klaren regulatorischen Wegen für EU-MAA und US-NDA-Einreichungen. Die Entwicklung adressiert einen kritischen Bedarf, da CDI jährlich 20.000 Kinder betrifft, mit einer Rezidivrate von 20-30%. Bei erfolgreichem Abschluss könnte ibezapolstat in Europa 11 Jahre Marketing-Exklusivität erhalten, einschließlich eines zusätzlichen Jahres über die standard 10 Jahre für neue Antibiotika hinaus.
Acurx Pharmaceuticals (Nasdaq: ACXP) حصلت على رأي إيجابي من لجنة طب الأطفال (PDCO) التابعة لوكالة الأدوية الأوروبية (EMA) فيما يخص خطة البحث pediatrics لدواء ibezapolstat لعلاج الأطفال المصابين بعدوى C. difficile (CDI). هذا يمثل علامة تنظيمية مهمة بينما تستعد الشركة لتجارب المرحلة الثالثة. الشركة قد حصلت بالفعل على تصميم QIDP وفئة Fast-Track من FDA، إضافة إلى تصميم SME من EMA. كل من FDA و EMA متوافقة مع برنامج التجارب السريرية لدى البالغين، مع مسارات تنظيمية واضحة لتقديم EU MAA و US NDA. التطوير يلبي حاجة حيوية، حيث تؤثر CDI على 20,000 طفل سنوياً، مع معدل تكرار 20-30%. عند الانتهاء بنجاح، قد يحصل ibezapolstat على 11 عاماً من حصرية التسويق في أوروبا، بما في ذلك عام إضافي بجانب الفترة القياسية البالغة 10 سنوات للنُهُب الجديدة من المضادات الحيوية.
Acurx Pharmaceuticals（NYSE: ACXP） 已就 ibezapolstat 的儿童调查计划（PIP）获得欧洲药品管理局（EMA）儿科委员会（PDCO）的积极意见，用于治疗患有艰难梭菌感染（CDI）的儿童。这标志着公司在进入第三阶段临床试验前的重要监管里程碑。公司已获得 FDA 的 QIDP 与快速通道（Fast-Track）指定，以及 EMA 的 SME 指定。FDA 与 EMA 均与成人临床试验计划保持一致，EU 的 MAA 提交与 US 的 NDA 提交也有明确的监管路径。该开发解决了关键需求，因为 CDI 每年影响 2万名儿童，复发率为 20-30%。如成功完成，ibezapolstat 可能在欧洲获得 11 年的上市独占权，并在标准的 10 年外再增加一年以应对新抗生素。
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Insights
Acurx's ibezapolstat advances toward Phase 3 trials with positive EMA pediatric plan opinion, strengthening its regulatory pathway for C. difficile treatment.
This positive opinion from the EMA's Paediatric Committee (PDCO) represents a significant regulatory milestone for Acurx's lead candidate ibezapolstat. The approval of the Pediatric Investigation Plan (PIP) satisfies a crucial prerequisite for initiating Phase 3 clinical trials in the European Union, marking the final major regulatory hurdle before late-stage development can begin internationally.
The regulatory positioning for ibezapolstat is particularly strong, with alignment between both EMA and FDA on the clinical trial program for adults, alongside clearly defined pathways to European Marketing Authorization Application (MAA) and US New Drug Application (NDA). The drug has already secured valuable FDA designations including QIDP (Qualified Infectious Disease Product) and Fast-Track status, plus SME designation from the EMA.
The pediatric focus is strategically important - C. difficile infection (CDI) affects approximately 20,000 children annually, with recurrence rates of 20-30%, similar to adults. While the company didn't disclose specific PIP details, the approval indicates regulatory confidence in their pediatric development approach. If successful, ibezapolstat would gain an additional year of marketing exclusivity in Europe beyond the standard 10-year period.
As a novel class of antibiotics targeting a serious infection with limited treatment options, particularly for children, ibezapolstat addresses a clear unmet medical need. The regulatory progress positions Acurx to advance into the final clinical development phase before potential commercialization, representing a derisking event in the company's development timeline.
- The Paediatric Committee (PDCO) of the European Medicines Agency issued a positive opinion on Acurx's Pediatric Investigation Plan (PIP) for ibezapolstat use in children with C. difficile infection
- Receipt of this positive opinion caps off the EMA's requirement to have the PIP agreed to by the initiation of ibezapolstat Phase 3 clinical trials in the European Union
- Acurx previously announced that both the EMA and the FDA are aligned with its clinical trial program in the adult population along with clearly defined requirements for the regulatory pathway to an EU MAA and a
U.S. NDA - In addition to receiving this positive EMA opinion, Acurx will proceed with its integrated PIP submission to the FDA
- Acurx is well positioned to begin international Phase 3 clinical trials and has previously been granted FDA QIDP and Fast-Track Designation and has received SME (Small and Medium-sized Enterprise) designation by the EMA
C. difficile is the most important infectious cause of antibiotic-associated diarrhea worldwide and a leading cause of healthcare-associated infection in
Acurx's Executive Chairman, Bob DeLuccia, stated: We're very pleased to have received this positive EMA opinion as part of our EMA regulatory pathway, since there is a significant unmet need for an innovative antibiotic to treat children suffering from CDI. Our pediatric clinical trial program is designed to demonstrate that ibezapolstat will be a safe and effective treatment that could represent a transformative advance to achieve CDI cure, reduce recurrences, and preserve the gut microbiome in this vulnerable patient population. Importantly, this could lead to reduction of the overall antimicrobial exposure in children with CDI." He added: "Additionally, ibezapolstat would be eligible to apply for an additional one year of marketing exclusivity in
* Adapted from: Shirley DA, Tornel W, Warren CA, Moonah S. Clostridioides difficile Infection in Children: Recent Updates on Epidemiology, Diagnosis, Therapy. Pediatrics. 2023 Sep 1;152(3):e2023062307. doi: 10.1542/peds.2023-062307. PMID: 37560802; PMCID: PMC10471512.
About the Pediatric Committee (PDCO)
The Pediatric Committee (PDCO) is the European Medicines Agency's (EMA) scientific committee responsible for activities on medicines for children and to support the development of such medicines in the European Union by providing scientific expertise and defining pediatric needs. The PDCO issues an opinion on PIP as part of the regulatory process and the EMA adopts a final decision based on the PDCO's opinion.
About the Pediatric Investigation Plan (PIP)
A pediatric investigation plan (PIP) is a development plan aimed at ensuring that the necessary data are obtained through studies in children, to support the authorization of a medicine for children. As part of the regulatory process for the registration of new medicines in
Acurx previously announced that it had received positive regulatory guidance from the EMA during its Scientific Advice Procedure which confirmed that the clinical, non-clinical and CMC (Chemistry Manufacturing and Controls) information package submitted to EMA supports advancement of the ibezapolstat Phase 3 program and if the Phase 3 program is successful, supports the submission of a Marketing Authorization Application (MAA) for regulatory approval in
The primary efficacy analysis will be performed using a Modified Intent-To-Treat (mITT) population. This will result in an estimated 450 subjects in the mITT population, randomized in a 1:1 ratio to either ibezapolstat or standard- of-care vancomycin, enrolled into the initial Phase 3 trial. The trial design not only allows determination of ibezapolstat's ability to achieve Clinical Cure of CDI as measured 2 days after 10 days of oral treatment but also includes assessment of ibezapolstat's potential effect on reduction of CDI recurrence in the target population. In the event non-inferiority of ibezapolstat to vancomycin is demonstrated, further analysis will be conducted to test for superiority.
About the Ibezapolstat Phase 2 Clinical Trial
The completed multicenter, open-label single-arm segment (Phase 2a) study was followed by a double-blind, randomized, active-controlled, non-inferiority, segment (Phase 2b) at 28 US clinical trial sites which together comprise the Phase 2 clinical trial. This Phase 2 clinical trial was designed to evaluate the clinical efficacy of ibezapolstat in the treatment of CDI including pharmacokinetics and microbiome changes from baseline. from study centers in
In the Phase 2b trial segment, 32 patients with CDI were enrolled and randomized in a 1:1 ratio to either ibezapolstat 450 mg every 12 hours or vancomycin 125 mg orally every 6 hours, in each case, for 10 days and followed for 28 ± 2 days following the end of treatment for recurrence of CDI. The two treatments were identical in appearance, dosing times, and number of capsules administered to maintain the blind. In this Phase 2b trial segment, 15 out of 16 (
When Phase 2b results are combined with Phase 2a results, the Clinical Cure rate in patients with CDI was
In the Phase 2 clinical trial (both trial segments), the Company also evaluated pharmacokinetics (PK) and microbiome changes and test for anti-recurrence microbiome properties, including the change from baseline in alpha diversity and bacterial abundance, especially overgrowth of healthy gut microbiota Actinobacteria and Firmicute phylum species during and after therapy. Phase 2a data demonstrated complete eradication of colonic C. difficile by day three of treatment with ibezapolstat as well as the observed overgrowth of healthy gut microbiota, Actinobacteria and Firmicute phyla species, during and after therapy. Very importantly, emerging data show an increased concentration of secondary bile acids during and following ibezapolstat therapy which is known to correlate with colonization resistance against C. difficile. A decrease in primary bile acids and the favorable increase in the ratio of secondary-to-primary bile acids suggest that ibezapolstat may reduce the likelihood of CDI recurrence when compared to vancomycin. The company also reported positive extended clinical cure (ECC) data for ibezapolstat (IBZ), its lead antibiotic candidate, from the Company's recently completed Phase 2b clinical trial in patients with CDI. This exploratory endpoint showed that 5 of 5 IBZ patients followed for up to three months following Clinical Cure experienced no recurrence of infection. Furthermore, ibezapolstat-treated patients showed lower concentrations of fecal primary bile acids, and higher beneficial ratio of secondary to primary bile acids than vancomycin-treated patients.
About Ibezapolstat
Ibezapolstat is the Company's lead antibiotic candidate planning to advance to international Phase 3 clinical trials to treat patients with C. difficile infection. Ibezapolstat is a novel, orally administered antibiotic, being developed as a Gram-Positive Selective Spectrum (GPSS®) antibacterial. It is the first of a new class of DNA polymerase IIIC inhibitors under development by Acurx to treat bacterial infections. Ibezapolstat's unique spectrum of activity, which includes C. difficile but spares other Firmicutes and the important Actinobacteria phyla, appears to contribute to the maintenance of a healthy gut microbiome.
In June 2018, ibezapolstat was designated by the
About Clostridioides difficile Infection
According to the 2017 Update (published February 2018) of the Clinical Practice Guidelines for C.difficile Infection by the Infectious Diseases Society of America (IDSA) and Society or Healthcare Epidemiology of America (SHEA), CDI remains a significant medical problem in hospitals, in long-term care facilities and in the community. C. difficile is one of the most common causes of health care- associated infections in
About the Microbiome in C. difficile Infection and Bile Acid Metabolism
C. difficile can be a normal component of the healthy gut microbiome, but when the microbiome is thrown out of balance, the C. difficile can thrive and cause an infection. After colonization with C. difficile, the organism produces and releases the main virulence factors, the two large clostridial toxins A (TcdA) and B (TcdB). (Kachrimanidou, Microorganisms 2020.) TcdA and TcdB are exotoxins that bind to human intestinal epithelial cells and are responsible for inflammation, fluid and mucous secretion, as well as damage to the intestinal mucosa. Bile acids perform many functional roles in the GI tract, with one of the most important being maintenance of a healthy microbiome by inhibiting C. difficile growth. Primary bile acids, which are secreted by the liver into the intestines, promote germination of C. difficile spores and thereby increase the risk of recurrent CDI after successful treatment of an initial episode. On the other hand, secondary bile acids, which are produced by normal gut microbiota through metabolism of primary bile acids, do not induce C. difficile sporulation and therefore protect against recurrent disease. Since ibezapolstat treatment leads to minimal disruption of the gut microbiome, bacterial production of secondary bile acids continues which may contribute to an anti-recurrence effect. Beneficial effects of bile acids include a decrease in primary bile acids and an increase in secondary bile acids in patients with CDI, which was observed in the Company's Ph2a trial results and previously reported (Garey, CID, 2022). In the Ph2b trial, ibezapolstat-treated patients showed lower concentrations of fecal primary bile acids, and higher beneficial ratio of secondary to primary bile acids than vancomycin-treated patients.
About Acurx Pharmaceuticals, Inc.
Acurx Pharmaceuticals is a late-stage biopharmaceutical company focused on developing a new class of small molecule antibiotics for difficult-to-treat bacterial infections. The Company's approach is to develop antibiotic candidates with a Gram-positive selective spectrum (GPSS®) that blocks the active site of the Gram-positive specific bacterial enzyme DNA polymerase IIIC (pol IIIC), inhibiting DNA replication and leading to Gram-positive bacterial cell death. Its R&D pipeline includes antibiotic product candidates that target Gram-positive bacteria, including Clostridioides difficile, methicillin- resistant Staphylococcus aureus (MRSA), vancomycin resistant Enterococcus (VRE), drug- resistant Streptococcus pneumoniae (DRSP) and B. anthracis (anthrax; a Bioterrorism Category A Threat-Level pathogen). Acurx's lead product candidate, ibezapolstat, for the treatment of C. difficile Infection is Phase 3 ready with plans in progress to begin international clinical trials. The Company's preclinical pipeline includes development of an oral product candidate for treatment of ABSSSI (Acute Bacterial Skin and Skin Structure Infections), upon which a development program for treatment of inhaled anthrax is being planned in parallel.
To learn more about Acurx Pharmaceuticals and its product pipeline, please visit www.acurxpharma.com.
Forward-Looking Statements
Any statements in this press release about our future expectations, plans and prospects, including statements regarding our strategy, future operations, prospects, plans and objectives, and other statements containing the words "believes," "anticipates," "plans," "expects," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: whether ibezapolstat will benefit from the QIDP designation; whether ibezapolstat will advance through the clinical trial process on a timely basis; whether the results of the clinical trials of ibezapolstat will warrant the submission of applications for marketing approval, and if so, whether ibezapolstat will receive approval from the FDA or equivalent foreign regulatory agencies where approval is sought; whether, if ibezapolstat obtains approval, it will be successfully distributed and marketed; and other risks and uncertainties described in the Company's annual report filed with the Securities and Exchange Commission on Form 10-K for the year ended December 31, 2024, and in the Company's subsequent filings with the Securities and Exchange Commission. Such forward- looking statements speak only as of the date of this press release, and Acurx disclaims any intent or obligation to update these forward-looking statements to reflect events or circumstances after the date of such statements, except as may be required by law.
Investor Contact: Acurx Pharmaceuticals, Inc.; David P. Luci, President & CEO, Tel: 917-533-1469; Email: davidluci@acurxpharma.com
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SOURCE Acurx Pharmaceuticals, Inc.
FAQ
What regulatory milestone did Acurx Pharmaceuticals (ACXP) achieve for ibezapolstat in September 2025?
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