Tharimmune Announces Positive FDA Feedback on NDA Path for TH104 for Prophylaxis of Respiratory and/or CNS Depression for Ultrapotent Opioid Exposure
Tharimmune (NASDAQ:THAR) received positive FDA feedback regarding the development of TH104, a buccal film formulation containing nalmefene, for prophylaxis against ultrapotent opioid exposure. The FDA confirmed that no additional clinical trials will be required for the New Drug Application (NDA) submission via the 505(b)(2) pathway.
The company is developing TH104 for two indications:
- Moderate-to-severe chronic pruritus in patients with primary biliary cholangitis (PBC)
- Temporary prophylaxis of respiratory and/or nervous system depression in military personnel and chemical incident responders exposed to high-potency opioids
The FDA's decision is based on existing safety and efficacy data of nalmefene, combined with Tharimmune's pharmacokinetic data. The company will use 'in silico' computer simulations to predict TH104's effectiveness as prophylaxis. Tharimmune is also advancing its Chemistry, Manufacturing, and Controls (CMC) plan to meet NDA requirements.
Tharimmune (NASDAQ:THAR) ha ricevuto un feedback positivo dalla FDA riguardo allo sviluppo di TH104, una formulazione in film buccale contenente nalmefene, per la profilassi contro l'esposizione a oppioidi ultrapotenti. La FDA ha confermato che non saranno necessari ulteriori studi clinici per la presentazione della Nuova Domanda di Farmaco (NDA) tramite il percorso 505(b)(2).
L'azienda sta sviluppando TH104 per due indicazioni:
- Prurito cronico da moderato a severo nei pazienti con colangite biliare primaria (PBC)
- Profilassi temporanea della depressione respiratoria e/o del sistema nervoso nei militari e nei soccorritori in caso di incidenti chimici esposti a oppioidi ad alta potenza
La decisione della FDA si basa sui dati di sicurezza ed efficacia esistenti di nalmefene, combinati con i dati farmacocinetici di Tharimmune. L'azienda utilizzerà simulazioni 'in silico' per prevedere l'efficacia di TH104 come profilassi. Tharimmune sta anche avanzando il suo piano di Chimica, Produzione e Controlli (CMC) per soddisfare i requisiti dell'NDA.
Tharimmune (NASDAQ:THAR) recibió comentarios positivos de la FDA sobre el desarrollo de TH104, una formulación en película bucal que contiene nalmefeno, para la profilaxis contra la exposición a opioides ultrapotentes. La FDA confirmó que no se requerirán ensayos clínicos adicionales para la presentación de la Nueva Solicitud de Medicamento (NDA) a través de la vía 505(b)(2).
La empresa está desarrollando TH104 para dos indicaciones:
- Prurito crónico de moderado a severo en pacientes con colangitis biliar primaria (PBC)
- Profilaxis temporal de depresión respiratoria y/o del sistema nervioso en personal militar y respondedores a incidentes químicos expuestos a opioides de alta potencia
La decisión de la FDA se basa en los datos existentes de seguridad y eficacia de nalmefeno, combinados con los datos farmacocinéticos de Tharimmune. La empresa utilizará simulaciones por computadora 'in silico' para predecir la efectividad de TH104 como profilaxis. Tharimmune también está avanzando en su plan de Química, Fabricación y Controles (CMC) para cumplir con los requisitos de la NDA.
Tharimmune (NASDAQ:THAR)는 초강력 오피오이드 노출에 대한 예방을 위해 nalmefene이 포함된 구강 필름 제형인 TH104 개발에 대해 FDA로부터 긍정적인 피드백을 받았습니다. FDA는 505(b)(2) 경로를 통한 신약 신청(NDA) 제출에 추가 임상 시험이 필요하지 않다고 확인했습니다.
회사는 TH104를 두 가지 적응증에 대해 개발하고 있습니다:
- 원발성 담도염(PBC) 환자의 중등도에서 중증 만성 가려움증
- 고강도 오피오이드에 노출된 군인 및 화학 사고 대응자의 호흡기 및/또는 신경계 우울증의 일시적 예방
FDA의 결정은 nalmefene의 기존 안전성 및 효능 데이터와 Tharimmune의 약리학적 데이터에 기반하고 있습니다. 회사는 TH104의 예방 효과를 예측하기 위해 'in silico' 컴퓨터 시뮬레이션을 사용할 것입니다. Tharimmune은 또한 NDA 요구 사항을 충족하기 위해 화학, 제조 및 품질 관리(CMC) 계획을 진행하고 있습니다.
Tharimmune (NASDAQ:THAR) a reçu des retours positifs de la FDA concernant le développement de TH104, une formulation en film buccal contenant du nalmefène, pour la prophylaxie contre l'exposition à des opioïdes ultrapuissants. La FDA a confirmé qu'aucun essai clinique supplémentaire ne sera nécessaire pour la soumission de la Demande de Nouveau Médicament (NDA) via le chemin 505(b)(2).
L'entreprise développe TH104 pour deux indications :
- Prurit chronique modéré à sévère chez les patients atteints de cholangite biliaire primitive (PBC)
- Prophylaxie temporaire de la dépression respiratoire et/ou du système nerveux chez le personnel militaire et les intervenants en cas d'incidents chimiques exposés à des opioïdes de haute puissance
La décision de la FDA repose sur les données existantes de sécurité et d'efficacité du nalmefène, combinées aux données pharmacocinétiques de Tharimmune. L'entreprise utilisera des simulations informatiques 'in silico' pour prédire l'efficacité de TH104 en tant que prophylaxie. Tharimmune fait également avancer son plan de Chimie, Fabrication et Contrôles (CMC) pour répondre aux exigences de la NDA.
Tharimmune (NASDAQ:THAR) erhielt positives Feedback von der FDA bezüglich der Entwicklung von TH104, einer buccalen Filmformulierung, die Nalmefen enthält, zur Prophylaxe gegen ultrapotente Opioid-Exposition. Die FDA bestätigte, dass keine zusätzlichen klinischen Studien für die Einreichung des New Drug Application (NDA) über den 505(b)(2)-Weg erforderlich sein werden.
Das Unternehmen entwickelt TH104 für zwei Indikationen:
- Moderates bis schweres chronisches Jucken bei Patienten mit primärer biliärer Cholangitis (PBC)
- Vorübergehende Prophylaxe von Atem- und/oder Nervensystemdepression bei Militärpersonal und Chemieunfallhelfern, die hochpotenten Opioiden ausgesetzt sind
Die Entscheidung der FDA basiert auf bestehenden Sicherheits- und Wirksamkeitsdaten von Nalmefen, kombiniert mit den pharmakokinetischen Daten von Tharimmune. Das Unternehmen wird 'in silico' Computersimulationen verwenden, um die Wirksamkeit von TH104 als Prophylaxe vorherzusagen. Tharimmune arbeitet auch an seinem Chemie-, Herstellungs- und Kontrollplan (CMC), um die Anforderungen der NDA zu erfüllen.
- FDA approval to pursue 505(b)(2) pathway without additional clinical trials
- Potential expedited development and market entry for TH104
- Dual indication potential expanding market opportunities
- Leveraging existing safety and efficacy data reduces development costs
- Success depends on computer simulation ('in silico') acceptance by FDA
- CMC requirements still need to be met before NDA submission
Insights
Tharimmune's receipt of positive FDA feedback for their TH104 drug represents a significant regulatory win. The FDA's confirmation that no additional clinical trials will be required for the 505(b)(2) New Drug Application pathway is particularly valuable for several reasons.
The 505(b)(2) pathway allows Tharimmune to leverage existing data for nalmefene (the active ingredient), substantially reducing both the time and cost to potential approval. This approach is especially powerful as it enables the company to utilize an established drug while providing a novel delivery mechanism (buccal film) for a new indication.
What makes this regulatory strategy particularly clever is Tharimmune's use of pharmacokinetic data and "in silico" modeling (computer simulation) to support their application. Rather than conducting extensive new human trials, they can demonstrate efficacy through mathematical models that predict how TH104 will perform as prophylaxis against opioid exposure.
This development pathway significantly de-risks the regulatory process for their second indication targeting military personnel and chemical incident responders. Moving forward with their Chemistry, Manufacturing, and Controls plan represents the next critical step, focusing on consistency in manufacturing their buccal film formulation to FDA standards.
This FDA feedback represents a major positive catalyst for Tharimmune's business strategy and market potential. The company is effectively executing a dual-indication approach for TH104, significantly expanding its addressable market beyond the original chronic pruritus indication.
The newly pursued indication targeting prophylaxis against ultrapotent opioid exposure opens potential markets in government/military contracts and emergency response sectors - typically characterized by more stable, long-term purchasing agreements compared to traditional pharmaceutical markets.
The FDA's acceptance of the 505(b)(2) pathway without requiring additional clinical trials delivers substantial value by:
- Dramatically reducing development costs
- Accelerating time-to-market potential
- Preserving capital resources for other development priorities
For a company with Tharimmune's
The repurposing strategy for nalmefene demonstrates smart resource allocation, as the company creates new value through innovative delivery (buccal film) of an established compound. This approach balances innovation with efficiency - particularly important for smaller biotechs with resources.
-Received positive feedback on New Drug Application submission via the 505(b)(2) pathway
-Company leveraging existing human pharmacokinetic data with TH104
-Advancing CMC plan to meet requirements of a New Drug Application
BRIDGEWATER, NJ / ACCESS Newswire / March 31, 2025 / Tharimmune, Inc. (Nasdaq:THAR) ("Tharimmune" or the "Company"), a clinical-stage biotechnology company focused on immunology and inflammation, today announced in addition to developing TH104 for the proposed indication of moderate-to-severe chronic pruritus in patients with primary biliary cholangitis ("PBC"), which utilizes an approved active ingredient, now formulated as a buccal film, Tharimmune is seeking to develop TH104 for the additional proposed indication of "Temporary Prophylaxis of Respiratory and/or Nervous System Depression in Military Personnel and Chemical Incident Responders Entering and Area Contaminated with High-Potency Opioids", for which we have submitted a Pre-Investigational New Drug Application ("PIND"). With respect to our PIND for this additional proposed indication for TH104, Tharimmune recently received positive feedback from the U.S. Food and Drug Administration (FDA) regarding a regulatory pathway that will allow the Company to submit a 505(b)(2) New Drug Application (NDA) for TH104. Importantly, the FDA has confirmed that no additional clinical trials will be required prior to NDA submission for this indication.
"This feedback from the FDA marks a significant milestone for Tharimmune and for individuals who may face the threat of ultrapotent opioid exposure," said Randy Milby, CEO. "We believe that a rapidly biodegradable buccal film offers prophylactic protection against respiratory distress in such situations represents a crucial advancement. The 505(b)(2) pathway along with leveraging the established safety and efficacy of the active ingredient, allows us to expedite the development and potential availability of this important product without the need for further clinical trials."
The foundation for the FDA's alignment on the submission of a 505(b)(2) NDA without additional clinical trials rests on the extensive existing data related to the approved active ingredient nalmefene embedded in TH104. This includes a comprehensive understanding of its safety profile, established through years of use in approved products, and evidence of its efficacy for opioid overdose. Coupling this wealth of information with the Company's pharmacokinetic (PK) data enables Tharimmune to build a model that will serve for an "in silico" submission. This type of submission refers to analyses conducted via computer simulation, allowing researchers to predict how TH104 will work as a prophylaxis against opioid exposure.
Specifically, by inputting detailed data from previous human studies conducted by Tharimmune that characterized the PK of the active ingredient via buccal administration as well as existing published data through other routes of administration, sophisticated mathematical models can simulate the expected drug levels and duration of action achievable with TH104. This approach allows for a scientifically sound prediction of TH104 behavior in the body without the need to repeat extensive human clinical trials that have already established the fundamental PK properties of nalmefene. The FDA recognizes the value of this approach when a new formulation or route of administration is being developed for a well-understood active ingredient.
Furthermore, Tharimmune is actively progressing its Chemistry, Manufacturing, and Controls (CMC) plan to meet the stringent requirements for filing an NDA with the FDA. This comprehensive plan encompasses all aspects of the manufacturing process, quality control measures, and product stability to ensure the consistent production of a high-quality buccal film formulation known as TH104. More information regarding the FDA's feedback will be made available on the investor relations section of Tharimmune's website in future filings.
About Tharimmune, Inc.
Tharimmune is a clinical-stage biotechnology company developing a diverse portfolio of therapeutic candidates in immunology, inflammation and oncology. Its lead clinical asset, TH104, aims to suppress chronic pruritus associated with primary biliary cholangitis (PBC), a rare autoimmune liver disease with no known cure. The expanded pipeline includes TH023, an oral TNF-alpha inhibitor offering a new approach to treating autoimmune diseases. Tharimmune is also advancing early-stage multispecific biologics targeting unique epitopes against multiple solid tumors through its proprietary EpiClick™ Technology. The Company has a license agreement with OmniAb, Inc. to access their antibody discovery technology for targeting specified disease markers. For more information, please visit: www.tharimmune.com.
Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, contained in this press release, including statements regarding the timing and design of Tharimmune's future Phase 2 trial, Tharimmune's strategy, future operations, future financial position, projected costs, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "continue," "could," "depends," "estimate," "expect," "intend," "may," "ongoing," "plan," "potential," "predict," "project," "target," "should," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements. Factors that may cause such differences, include, but are not limited to, those discussed under Risk Factors set forth in our Annual Report on Form 10-K for the year ended December 31, 2024 and other periodic reports filed by the Company from time to time with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this release. Subsequent events and developments may cause the Company's views to change; however, the Company does not undertake and specifically disclaims any obligation to update or revise any forward-looking statements to reflect new information, future events or circumstances or to reflect the occurrences of unanticipated events, except as may be required by applicable law. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.
Contacts
Tharimmune, Inc.
ir@tharimmune.com
Alliance Advisors IR
Tirth T. Patel
tpatel@allianceadvisors.com
212-201-6614
SOURCE: Tharimmune Inc.
View the original press release on ACCESS Newswire
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