Lantern Pharma Announces Three U.S. FDA Rare Pediatric Disease Designations Granted to LP-184 in Multiple Ultra Rare Children’s Cancers
Lantern Pharma (NASDAQ: LTRN) has received three rare pediatric disease designations (RPDD) from the FDA for its drug candidate LP-184 in malignant rhabdoid tumors, rhabdomyosarcoma, and hepatoblastoma. This brings the total RPDDs for LP-184 to four, including a previous designation for atypical teratoid rhabdoid tumors. The efficacy of LP-184 was demonstrated through tumor regression and extended event-free survival in models developed by the NCI-supported Pediatric Preclinical Testing Program.
RPDDs can lead to Priority Review Vouchers upon FDA marketing approval, which can expedite future drug reviews or be sold for significant sums. LP-184 is currently in a Phase 1A clinical trial enrolling 50-60 patients across various solid tumors. Lantern Pharma aims to use AI and data-driven approaches to revolutionize cancer drug development, potentially reducing costs and accelerating timelines for new therapies.
Lantern Pharma (NASDAQ: LTRN) ha ricevuto tre designazioni per malattie pediatriche rare (RPDD) dalla FDA per il suo candidato farmaco LP-184 in tumori rabdoidi maligni, rabdomiosarcoma e epatoblastoma. Questo porta il totale delle RPDD per LP-184 a quattro, inclusa una precedente designazione per tumori rabdoidi teratoidi atipici. L'efficacia di LP-184 è stata dimostrata attraverso la regressione del tumore e un prolungamento della sopravvivenza libera da eventi in modelli sviluppati dal Programma di Testing Preclinico Pediatrico supportato dal NCI.
Le RPDD possono portare a Voucher per Revisione Prioritaria dopo l'approvazione da parte della FDA, il che può accelerare le future revisioni dei farmaci o essere venduti per somme significative. LP-184 è attualmente in un trial clinico di Fase 1A che arruola 50-60 pazienti con vari tumori solidi. Lantern Pharma mira a utilizzare approcci basati su AI e sui dati per rivoluzionare lo sviluppo dei farmaci contro il cancro, potenzialmente riducendo i costi e accelerando i tempi di sviluppo delle nuove terapie.
Lantern Pharma (NASDAQ: LTRN) ha recibido tres designaciones para enfermedades pediátricas raras (RPDD) de la FDA para su candidato a fármaco LP-184 en tumores rabdoides malignos, rabdomiosarcoma y hepatoblastoma. Esto lleva el total de RPDDs para LP-184 a cuatro, incluida una designación previa para tumores rabdoides teratoides atípicos. La eficacia de LP-184 se demostró a través de la regresión del tumor y la prolongación de la supervivencia libre de eventos en modelos desarrollados por el Programa de Pruebas Preclínicas Pediátricas respaldado por el NCI.
Las RPDD pueden conducir a Vales de Revisión Prioritaria tras la aprobación de marketing de la FDA, lo que puede acelerar futuras revisiones de medicamentos o ser vendidos por sumas significativas. LP-184 está actualmente en un ensayo clínico de Fase 1A que enrola entre 50 y 60 pacientes con varios tumores sólidos. Lantern Pharma tiene la intención de utilizar enfoques impulsados por IA y datos para revolucionar el desarrollo de medicamentos contra el cáncer, lo que podría reducir costos y acelerar los plazos para nuevas terapias.
Lantern Pharma (NASDAQ: LTRN)은 악성 람브로이드 종양, 육종, 그리고 간모세포종에 대한 후보 약물 LP-184에 대해 FDA로부터 세 가지 희귀 소아 질병 지정 (RPDD)을 받았습니다. 이는 LP-184의 총 RPDD 수를 네 개로 늘리며, 이전에 atypical teratoid rhabdoid tumors에 대한 지정이 포함됩니다. LP-184의 효능은 NCI 지원 소아 임상 시험 프로그램에서 개발된 모델을 통해 종양 축소와 이벤트 없는 생존 연장을 통해 입증되었습니다.
RPDD는 FDA의 마케팅 승인 후 우선 검토 바우처를 유도할 수 있으며, 이는 향후 의약품 검토를 가속화하거나 상당한 금액으로 판매될 수 있습니다. LP-184는 현재 다양한 고형 종양에 대해 50-60명의 환자를 모집하는 1A상 임상 시험 진행 중입니다. Lantern Pharma는 AI와 데이터 기반 접근 방식을 사용하여 암 치료제 개발을 혁신하려 하며, 비용을 줄이고 새로운 치료에 대한 시간을 단축할 수 있을 것으로 기대하고 있습니다.
Lantern Pharma (NASDAQ: LTRN) a reçu trois demandes de désignation pour des maladies pédiatriques rares (RPDD) de la FDA pour son candidat médicament LP-184 dans les cas de tumeurs rhabdoïdes malignes, rhabdomyosarcome et hépatoblastome. Cela porte le total des RPDD pour LP-184 à quatre, y compris une désignation précédente pour des tumeurs rhabdoïdes atypiques teratoïdes. L'efficacité de LP-184 a été démontrée par la régression tumorale et l'allongement de la survie sans événements dans des modèles développés par le Programme de Test Préclinique Pédiatrique soutenu par le NCI.
Les RPDD peuvent mener à des Vouchers de Révision Prioritaire après approbation marketing de la FDA, ce qui peut permettre d'accélérer les révisions futures de médicaments ou d'être vendus à des sommes significatives. LP-184 est actuellement en essai clinique de phase 1A, recrutant 50 à 60 patients atteints de divers tumeurs solides. Lantern Pharma vise à utiliser des approches basées sur l'IA et des données pour révolutionner le développement des médicaments contre le cancer, ce qui pourrait réduire les coûts et accélérer les délais pour de nouvelles thérapies.
Lantern Pharma (NASDAQ: LTRN) hat von der FDA drei Bezeichnungen für seltene pädiatrische Krankheiten (RPDD) für den Arzneimittelkandidaten LP-184 bei bösartigen rhabdoiden Tumoren, Rhabdomyosarkom und Hepatoblastom erhalten. Dies erhöht die Gesamtzahl der RPDDs für LP-184 auf vier, einschließlich einer vorherigen Bezeichnung für atypische teratoide rhabdoide Tumoren. Die Wirksamkeit von LP-184 wurde durch Tumorrückbildung und verlängerte ereignisfreie Überlebenszeiten in Modellen, die vom NCI-unterstützten Programm für pädiatrische Präklinische Tests entwickelt wurden, nachgewiesen.
RPDDs können zu Prioritätsprüfungs-Vouchern nach der Zulassung durch die FDA führen, die zukünftige Arzneimittelprüfungen beschleunigen oder für bedeutende Beträge verkauft werden können. LP-184 befindet sich derzeit in einer klinischen Phase-1A-Studie, die 50-60 Patienten mit verschiedenen soliden Tumoren einschließt. Lantern Pharma hat das Ziel, KI- und datengestützte Ansätze zu nutzen, um die Entwicklung von Krebsmedikamenten zu revolutionieren, was potenziell die Kosten senken und die Zeitpläne für neue Therapien beschleunigen könnte.
- Received three new rare pediatric disease designations (RPDD) for LP-184, bringing the total to four
- Potential eligibility for Priority Review Vouchers, which can be sold for over $100 million
- LP-184 showed efficacy in tumor regression and extended event-free survival in preclinical models
- Currently conducting a Phase 1A clinical trial for LP-184 across various solid tumors
- None.
- Lantern Pharma was granted the rare pediatric disease designation (RPDD) for drug-candidate, LP-184, in three cancer indications: Malignant Rhabdoid Tumors, Rhabdomyosarcoma, and Hepatoblastoma.
- This brings the total number of RPDDs for LP-184 to 4, including one previously granted for ATRT (Atypical Teratoid Rhabdoid Tumors).
- Efficacy of LP-184 in these three rare pediatric cancers was demonstrated through evidence of tumor regression and extended event-free survival in specialized models developed as part of the Pediatric Preclinical Testing Program (PPTP), an initiative supported by the National Cancer Institute (NCI) to identify novel therapeutic agents that may have significant activity against childhood cancers.
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Companies with RPDDs might be eligible to receive a Priority Review Voucher (PRV) upon FDA marketing approval that could be redeemed to receive a priority review for any subsequent marketing application. If received, PRVs may be used by the sponsor or sold to another sponsor and have recently sold for over
(1).$100 million
"At Lantern, we're harnessing AI and data-driven approaches to revolutionize cancer drug development, aiming to dramatically reduce costs, accelerate timelines, and enhance precision in bringing new therapies to patients," stated Panna Sharma, CEO and President of Lantern Pharma. "Our recent breakthrough in identifying three additional, high-potential indications for LP-184 in pediatric cancers exemplifies this progress. We believe that 'AI for good' should address both blockbuster opportunities as well as rare, often overlooked pediatric cases. The FDA's Rare Pediatric Disease designation for these three potential programs is a testament to this commitment. We're acutely aware that patients and their families are relying on innovators like us to speed up therapy development. These designations mark a crucial step forward in advancing our expanding portfolio of pediatric programs targeting these devastating and rare cancers. It reinforces our dedication to transforming hope into tangible solutions for those who need them most."
Rare pediatric diseases are defined by the FDA as serious or life-threatening conditions primarily affecting children under 18, with fewer than 200,000 cases in the
Lantern’s investigational drug candidate, LP-184, has shown preclinical activity in a wide range of solid tumors, garnering it multiple orphan and rare pediatric designations. LP-184 is currently in a multi-center Phase 1A clinical trial that is expected to enroll approximately 50 to 60 patients across a wide range of solid tumors. Based on the results and findings from this clinical trial and other collaborative studies, Lantern will plan and potentially develop future clinical trials for specific pediatric patients in ATRT, MRT, RMS and Hepatoblastoma.
About MRT - Malignant Rhabdoid Tumors
Malignant rhabdoid tumors are rare childhood cancers that typically affect the kidneys and soft tissues, sometimes occurring in the brain as atypical teratoid rhabdoid tumors (ATRT). The kidney and soft tissue variant, known as malignant rhabdoid tumor (MRT), is most common in infants and toddlers, with an average diagnosis age of 15 months. In
About RMS - Rhabdomyosarcoma
Rhabdomyosarcoma is a rare cancerous tumor that develops in the body's soft tissues, which connect, support, and surround organs and other structures. It originates from rhabdomyoblast cells, which form early in embryonic development, making this cancer more prevalent in children than adults. The tumor commonly appears in the head, neck, bladder, vagina, arms, legs, and trunk, but can also occur in areas with minimal skeletal muscle, such as the prostate, middle ear, or bile duct system. Despite being the most common childhood soft-tissue sarcoma, rhabdomyosarcoma affects only about 250-300 children annually in
About Hepatoblastoma
Hepatoblastomas are the most common primary malignant liver tumors in pediatric patients, typically occurring within the first two years of life. These tumors are classified into two histologic types: epithelial and mixed. While most hepatoblastomas are sporadic, about one-third of cases are associated with genetic conditions such as Beckwith-Weidemann syndrome, familial adenomatous polyposis (FAP), Edward syndrome (trisomy 18), nephroblastoma, and Down syndrome. Infants with low birth weight are at a higher risk of developing hepatoblastoma, and there is evidence linking the tumor to preeclampsia and parental tobacco smoking before and during pregnancy. The most common genetic mutation in hepatoblastoma involves the Wnt signaling pathway, leading to the accumulation of beta-catenin, particularly in sporadic cases. In more aggressive cases, activation of TERT (human telomerase reverse transcriptase) and MYC signaling has been observed. Hepatoblastoma is a rare tumor, accounting for approximately
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Recent PRV (Priority Review Vouchers) sold and publicly disclosed in recent 12 months: Ipsen Pharma on 08/27/24 for |
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Source: Lantern Pharma Inc.
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