Talphera Announces Agreement with the FDA for Prior Approval Supplement Review to Reduce the Number of Patients in the NEPHRO CRRT Study
Talphera (NASDAQ: TLPH) has announced significant developments in its NEPHRO CRRT clinical study following a meeting with the FDA. The agency has agreed to review a Prior Approval Supplement (PAS) for reducing the number of patients in the study, with the submission expected within the week. The FDA's review process will take up to 30 days.
Additionally, the FDA approved two protocol changes that will expedite enrollment: allowing the inclusion of patients on continuous renal replacement therapy (CRRT) beyond 48 hours and permitting heparin-tolerant patients at certain institutions. These changes will be implemented through a five-day protocol amendment notice, requiring no additional FDA review.
The company aims to complete the NEPHRO CRRT study by year-end, with nafamostat being developed as an alternative anticoagulant for CRRT patients under breakthrough designation status.
Talphera (NASDAQ: TLPH) ha annunciato sviluppi significativi nel suo studio clinico NEPHRO CRRT dopo un incontro con la FDA. L'agenzia ha accettato di esaminare un Supplemento per Approvazione Precedente (PAS) per ridurre il numero di pazienti nello studio, con la presentazione prevista entro la settimana. Il processo di revisione della FDA richiederà fino a 30 giorni.
Inoltre, la FDA ha approvato due modifiche al protocollo che faciliteranno l'arruolamento: consentendo l'inclusione di pazienti in terapia sostitutiva renale continua (CRRT) oltre le 48 ore e permettendo pazienti tolleranti all'eparina in alcune istituzioni. Queste modifiche saranno attuate tramite un avviso di modifica del protocollo di cinque giorni, senza richiedere ulteriori revisioni da parte della FDA.
L'azienda punta a completare lo studio NEPHRO CRRT entro la fine dell'anno, con nafamostat in fase di sviluppo come anticoagulante alternativo per i pazienti CRRT sotto lo stato di designazione per rottura.
Talphera (NASDAQ: TLPH) ha anunciado desarrollos significativos en su estudio clínico NEPHRO CRRT tras una reunión con la FDA. La agencia ha aceptado revisar un Suplemento de Aprobación Previa (PAS) para reducir el número de pacientes en el estudio, con la presentación esperada dentro de una semana. El proceso de revisión de la FDA tomará hasta 30 días.
Además, la FDA aprobó dos cambios en el protocolo que agilizarán la inscripción: permitiendo la inclusión de pacientes en terapia de reemplazo renal continuo (CRRT) más allá de las 48 horas y permitiendo pacientes tolerantes a heparina en ciertas instituciones. Estos cambios se implementarán a través de un aviso de enmienda de protocolo de cinco días, sin requerir revisión adicional por parte de la FDA.
La empresa tiene como objetivo completar el estudio NEPHRO CRRT para fin de año, con nafamostat en desarrollo como anticoagulante alternativo para pacientes CRRT bajo estatus de designación innovadora.
탈페라(Talphera, NASDAQ: TLPH)가 FDA와의 회의 이후 NEPHRO CRRT 임상 연구에서 중요한 발전을 발표했습니다. FDA는 연구에 참여하는 환자 수를 줄이기 위한 사전 승인 보충 서류(PAS)를 검토하기로 동의했으며, 제출은 이번 주 내에 이루어질 것으로 예상됩니다. FDA의 검토 과정은 최대 30일이 걸릴 것입니다.
이와 함께 FDA는 enrollment를 가속화하기 위해 두 가지 프로토콜 변경을 승인했습니다: 48시간을 초과한 지속적인 신장 대체 요법(CRRT) 환자의 포함을 허용하고, 특정 기관에서 헤파린 내성이 있는 환자를 허용합니다. 이러한 변경 사항은 추가적인 FDA 검토 없이 5일 전의 프로토콜 수정 통지를 통해 시행될 것입니다.
회사는 연말까지 NEPHRO CRRT 연구를 완료할 계획이며, 나파모스타트는 혁신적인 지위에서 CRRT 환자를 위한 대체 항응고제로 개발되고 있습니다.
Talphera (NASDAQ: TLPH) a annoncé des développements significatifs dans son étude clinique NEPHRO CRRT suite à une réunion avec la FDA. L'agence a accepté d'examiner un Supplément d'Approbation Préalable (PAS) pour réduire le nombre de patients dans l'étude, avec une soumission attendue dans la semaine. Le processus de révision de la FDA prendra jusqu'à 30 jours.
De plus, la FDA a approuvé deux modifications du protocole qui faciliteront l'inscription : permettant l'inclusion de patients en thérapie de remplacement rénal continu (CRRT) au-delà de 48 heures et autorisant les patients tolérants à l'héparine dans certaines institutions. Ces changements seront mis en œuvre par un avis de modification de protocole de cinq jours, sans nécessiter d'examen supplémentaire de la FDA.
La société vise à terminer l'étude NEPHRO CRRT d'ici la fin de l'année, avec le nafamostat en développement en tant qu'anticoagulant alternatif pour les patients CRRT sous un statut de désignation de percée.
Talphera (NASDAQ: TLPH) hat bedeutende Entwicklungen in seiner NEPHRO CRRT klinischen Studie nach einem Treffen mit der FDA angekündigt. Die Behörde hat zugestimmt, ein Vorabgenehmigungszusatz (PAS) zur Reduzierung der Patientenzahl in der Studie zu prüfen, wobei die Einreichung innerhalb einer Woche erwartet wird. Der Prüfungsprozess der FDA wird bis zu 30 Tage in Anspruch nehmen.
Zusätzlich genehmigte die FDA zwei Protokolländerungen, die die Einschreibung beschleunigen werden: die Aufnahme von Patienten in kontinuierlicher Nierenersatztherapie (CRRT) über 48 Stunden hinaus zu erlauben und heparintolerante Patienten in bestimmten Institutionen zuzulassen. Diese Änderungen werden durch eine fünf Tage dauernde Protokolländerungsbenachrichtigung umgesetzt, die keine zusätzliche Überprüfung durch die FDA erfordert.
Das Unternehmen plant, die NEPHRO CRRT-Studie bis zum Jahresende abzuschließen, wobei Nafamostat als alternatives Antikoagulans für CRRT-Patienten unter dem Status der bahnbrechenden Bezeichnung entwickelt wird.
- FDA agrees to review reduction in patient numbers for NEPHRO CRRT study
- Two protocol changes approved to accelerate patient enrollment
- Breakthrough designation status maintained for nafamostat
- 30-day expedited review timeline for PAS
- Study completion timeline pushed to year-end
- PAS approval still pending and required before protocol amendment
Insights
This FDA agreement represents a pivotal development for Talphera's NEPHRO CRRT study. The potential reduction in patient numbers, combined with broadened inclusion criteria, could substantially accelerate the study's completion timeline and reduce costs. The FDA's willingness to review these modifications through a Prior Approval Supplement suggests confidence in the preliminary data and study design.
The two additional protocol changes are particularly significant:
- Allowing enrollment of patients on CRRT beyond 48 hours expands the eligible patient pool
- Including heparin-tolerant patients at certain institutions further broadens recruitment opportunities
The breakthrough designation status for nafamostat is important here - it indicates the FDA recognizes the urgent need for alternative anticoagulation options in CRRT patients. For context, current CRRT anticoagulation relies heavily on heparin, which carries significant bleeding risks. A successful nafamostat approval could address an important unmet medical need in critical care nephrology.
For a micro-cap company with a market cap of just
- Reduced patient recruitment costs
- Accelerated timeline to potential commercialization
- Lower overall clinical trial expenses
The 30-day review period for the PAS is remarkably quick compared to traditional supplemental application reviews, which can take months. This expedited timeline, combined with the breakthrough designation, suggests a potentially faster path to market for nafamostat. For investors, this efficiency in regulatory process could mean earlier revenue potential and reduced cash burn - critical factors for a company of this size.
Submission of a Prior Approval Supplement for a reduction in the number of patients in the NEPHRO CRRT study is expected within the coming week
The FDA agreed to two additional protocol changes expected to accelerate enrollment in the NEPHRO CRRT study
During this same meeting, the agency agreed to two other changes to broaden the clinical study inclusion criteria which would allow the Company to enroll patients already on continuous renal replacement therapy (CRRT) beyond 48 hours as well as heparin-tolerant patients at certain institutions. These changes are being made through a five-day protocol amendment notice to the FDA with no additional FDA review required.
"We continue to evaluate and focus on opportunities to support completion of the NEPHRO CRRT study by the end of the year. In addition to new clinical sites, our continuous improvement efforts in study execution were strengthened following our positive face-to-face meeting with the FDA where the agency agreed we should submit a PAS to reduce the number of patients in the NEPHRO CRRT study. We plan to submit the PAS in the coming week which requires a review period of up to 30 days," stated Shakil Aslam, MD, Talphera's Chief Medical Officer. "Our breakthrough designation status and close collaboration with the FDA continue to support our confidence in the importance of nafamostat as an alternative anticoagulant for patients on CRRT," continued Dr. Aslam.
About Talphera, Inc.
Talphera, Inc. is a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for use in medically supervised settings. Talphera's lead product candidate, Niyad™ is a lyophilized formulation of nafamostat and is currently being studied under an investigational device exemption (IDE) as an anticoagulant for the extracorporeal circuit, and has received Breakthrough Device Designation status from the
This release is intended for investors only. For additional information about Talphera, please visit www.talphera.com.
About Niyad and nafamostat
Nafamostat is a broad spectrum, synthetic serine protease inhibitor with anticoagulant, anti-inflammatory and potential anti-viral activities. Niyad™ is a lyophilized formulation of nafamostat and is currently being studied under an IDE, as an anticoagulant for the extracorporeal circuit, and has received Breakthrough Device Designation Status from the FDA. Talphera's registrational study of Niyad™ is named the NEPHRO CRRT (Nafamostat Efficacy in Phase 3 Registrational Continuous Renal Replacement Therapy) study. An ICD-10 procedural code, XY0YX37, has been issued for the extracorporeal introduction of nafamostat. The ICD-10 code is a specific/billable code that can be used to indicate a procedure. LTX-608 is a proprietary nafamostat formulation for direct IV infusion that may be investigated and developed for the treatment of acute respiratory distress syndrome (ARDS), disseminated intravascular coagulation (DIC), acute pancreatitis or as an anti-viral treatment, amongst other potential targets.
About the NEPHRO CRRT Study
The NEPHRO Study, which has received central IRB approval, is designed as a prospective, double-blinded trial to be conducted at up to 14 U.S. hospital intensive care units. The study will enroll and evaluate 166 adult patients undergoing renal replacement therapy, who cannot tolerate heparin or are at risk for bleeding. The primary endpoint of the study is mean post-filter activated clotting time using Niyad versus placebo over the first 24 hours. Key secondary endpoints include filter lifespan, number of filter changes over 72 hours, number of transfusions over 72 hours and dialysis efficacy (based on urea concentration) over the first 24 hours.
Forward-looking statements
This press release contains forward-looking statements based upon Talphera's current expectations and assumptions. These and any other forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking terminology such as "believe," "expect," "anticipate," "may," "if," "intends," "plans," "potential," "projected," "will," or the negative of these words or other comparable terminology, and include: Talphera's expectation that the NEPHRO study enrollment rates will accelerate due to the protocol changes, and will support the study completion by the end of 2025, and that the PAS will be submitted in the coming week. Talphera's discussion of its strategy, plans and intentions also include forward-looking statements, which are predictions, projections and other statements about future events that are based on current expectations and assumptions. These forward-looking statements involve risks and uncertainties that could cause actual results to differ materially from those projected, anticipated or implied by such statements, including: (i) risks relating to Talphera's product development activities, including that clinical studies may not be fully enrolled or completed and/or confirm any safety, efficacy or other potential developmental product characteristics described or assumed in this press release; (ii) Talphera's developmental product candidates may not be beneficial to patients or healthcare providers or be successfully commercialized; (iii) risks relating to Talphera's ability to obtain regulatory approvals for its developmental product candidates; (iv) risks related to the ability of Talphera and its business partners to implement development plans, commercial launch plans, forecasts and other business expectations; and (v) risks related to Talphera's liquidity and its ability to maintain capital resources sufficient to conduct its clinical studies. Although it is not possible to predict or identify all such risks and uncertainties, they may include, but are not limited to, those described under the caption "Risk Factors" and elsewhere in Talphera's annual, quarterly and current reports (i.e., Form 10-K, Form 10-Q and Form 8-K) as filed or furnished with the SEC and any subsequent public filings. You are cautioned not to place undue reliance on any such forward-looking statements, which speak only as of the date such statements were first made. To the degree financial information is included in this press release, it is in summary form only and must be considered in the context of the full details provided in Talphera's most recent annual, quarterly or current report as filed or furnished with the SEC. Talphera's SEC reports are available at www.talphera.com under the "Investors" tab. Except to the extent required by law, Talphera undertakes no obligation to publicly release the result of any revisions to these forward-looking statements to reflect new information, events or circumstances after the date hereof, or to reflect the occurrence of unanticipated events.
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SOURCE Talphera, Inc.
FAQ
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