MAIA Biotechnology Granted FDA Rare Pediatric Disease Designation for THIO as a Treatment for Pediatric High-Grade Gliomas
MAIA Biotechnology (NYSE: MAIA) announced that the FDA has granted Rare Pediatric Disease Designation for THIO in treating pediatric-type diffuse high-grade gliomas (PDHGG). This designation makes MAIA eligible for a priority review voucher upon future FDA approval, which can be sold as an asset with an average value of $100 million since 2015.
Previous research demonstrated THIO's effectiveness when combined with ionizing radiation in treating diffuse intrinsic pontine glioma (DIPG), a PDHGG subtype, showing significant decrease in cell proliferation. THIO, believed to be the only direct telomere-targeting agent in clinical development, also holds orphan drug designations for hepatocellular carcinoma, small cell lung cancer, and glioblastoma.
MAIA Biotechnology (NYSE: MAIA) ha annunciato che la FDA ha concesso la designazione di malattia pediatrica rara per THIO nel trattamento dei gliomi diffusi ad alto grado pediatrico (PDHGG). Questa designazione rende MAIA idonea per un voucher di revisione prioritaria in caso di futura approvazione da parte della FDA, che può essere venduto come un attivo con un valore medio di 100 milioni di dollari dal 2015.
Le ricerche precedenti hanno dimostrato l'efficacia di THIO quando combinato con radiazioni ionizzanti nel trattamento del glioma pontino intrinseco diffuso (DIPG), un sottotipo di PDHGG, mostrando una significativa riduzione nella proliferazione cellulare. THIO, ritenuto essere l'unico agente direttamente mirato ai telomeri attualmente in sviluppo clinico, possiede anche designazioni di farmaco orfano per il carcinoma epatocellulare, il cancro polmonare a piccole cellule e il glioblastoma.
MAIA Biotechnology (NYSE: MAIA) anunció que la FDA ha otorgado la designación de Enfermedad Pediátrica Rara para THIO en el tratamiento de gliomas difusos de alto grado tipo pediátrico (PDHGG). Esta designación hace que MAIA sea elegible para un vale de revisión prioritaria una vez que la FDA otorgue su aprobación, el cual puede ser vendido como un activo con un valor promedio de 100 millones de dólares desde 2015.
Investigaciones anteriores demostraron la efectividad de THIO cuando se combina con radiación ionizante en el tratamiento del glioma pontino difuso intrínseco (DIPG), un subtipo de PDHGG, mostrando una disminución significativa en la proliferación celular. THIO, que se cree es el único agente de objetivo directo a los telómeros en desarrollo clínico, también posee designaciones de medicamento huérfano para el carcinoma hepatocelular, el cáncer de pulmón de células pequeñas y el glioblastoma.
MAIA Biotechnology (NYSE: MAIA)는 FDA가 소아 고급 확산 교모세포종(PDHGG) 치료를 위한 THIO에 대해 희귀 소아 질환 지정을 부여했다고 발표했습니다. 이 지정을 통해 MAIA는 향후 FDA 승인 시 우선 심사 바우처를 받을 자격이 생기며, 이는 2015년부터 평균 1억 달러의 자산으로 판매될 수 있습니다.
이전 연구에 따르면 THIO는 PDHGG의 하위 유형인 확산 본체 교모세포종(DIPG) 치료 시 전리 방사선과 결합했을 때 유효성을 보여 세포 증식이 상당히 감소했습니다. THIO는 임상 개발 중 유일한 직접 텔로미어 표적제인 것으로 여겨지며, 간세포 암종, 소세포 폐암 및 교모세포종에 대한 고아약 지정을 보유하고 있습니다.
MAIA Biotechnology (NYSE: MAIA) a annoncé que la FDA a accordé la désignation de maladie pédiatrique rare pour THIO dans le traitement des gliomes difus à haut grade de type pédiatrique (PDHGG). Cette désignation rend MAIA éligible à un bon de révision prioritaire en cas d'approbation future par la FDA, qui peut être vendu en tant qu'actif d'une valeur moyenne de 100 millions de dollars depuis 2015.
Des recherches précédentes ont démontré l'efficacité de THIO lorsqu'il est associé à des radiations ionisantes dans le traitement du gliome intrinsèque diffus du pont (DIPG), un sous-type de PDHGG, montrant une diminution significative de la prolifération cellulaire. THIO, considéré comme le seul agent ciblant directement les télomères en cours de développement clinique, possède également des désignations de médicament orphelin pour le carcinome hépatocellulaire, le cancer du poumon à petites cellules et le glioblastome.
MAIA Biotechnology (NYSE: MAIA) gab bekannt, dass die FDA die Rare Pediatric Disease Designation für THIO zur Behandlung von diffusen hochgradigen Gliomen vom pädiatrischen Typ (PDHGG) erteilt hat. Diese Auszeichnung macht MAIA berechtigt, einen Prioritätsprüfungs-Voucher bei zukünftiger FDA-Zulassung zu erhalten, der seit 2015 als Vermögenswert im Durchschnittswert von 100 Millionen Dollar verkauft werden kann.
Frühere Forschungen haben die Wirksamkeit von THIO in Kombination mit ionisierender Strahlung zur Behandlung des diffusen intrinsischen pontine Glioms (DIPG), einer PDHGG-Unterart, demonstriert und eine signifikante Verringerung der Zellproliferation gezeigt. THIO, das als das einzige direkt auf Telomere zielende Mittel in der klinischen Entwicklung gilt, hat auch den Status eines Orphan Drug für das hepatocelluläre Karzinom, das kleinzellige Lungenkarzinom und das Glioblastom.
- FDA granted Rare Pediatric Disease Designation for THIO
- Eligibility for priority review voucher worth approximately $100 million upon FDA approval
- Demonstrated positive results in treating DIPG when combined with radiation
- Multiple orphan drug designations in three different cancer types
- THIO still requires FDA approval for PDHGG indication
- No current market revenue from THIO as it's still in clinical development
Insights
The FDA's rare pediatric disease designation for THIO represents a significant milestone for MAIA Biotechnology's development pipeline. The designation specifically targets pediatric-type diffuse high-grade gliomas (PDHGG), one of the most challenging childhood cancers to treat. THIO's unique mechanism as a telomere-targeting agent, combined with its ability to activate the immune system while bypassing tumor immunosuppression, shows promising potential in addressing an urgent unmet medical need.
The preliminary data from the collaboration with Nationwide Children's Hospital, demonstrating THIO's efficacy in combination with radiation therapy for DIPG (a PDHGG subtype), provides encouraging scientific validation. This designation, along with existing orphan drug designations in three other cancer types, strengthens THIO's development portfolio and potential market positioning.
This regulatory milestone significantly enhances MAIA's commercial prospects and potential market value. The rare pediatric disease designation's most valuable aspect is the eligibility for a priority review voucher (PRV) upon future FDA approval. With PRVs historically trading at around
The multiple orphan drug designations across different cancer types also create multiple paths to market, potentially reducing investment risk. This development could attract partnership opportunities or additional investor interest, particularly given the company's focus on hard-to-treat cancers.
“THIO is a versatile anti-cancer agent that has demonstrated positive results in multiple difficult to treat cancer types, including pediatric high-grade glioma, which is among the most treatment-resistant cancers in children. THIO is shown to activate the immune system while evading tumor immunosuppression, a novel therapeutic approach for this devastating childhood disease,” said MAIA Chairman and Chief Executive Officer Vlad Vitoc, M.D. “We are proud to receive the FDA’s Rare Pediatric Disease designation for THIO, which significantly bolsters our plans for continuing research in the PDHGG indication.”
MAIA’s Vice President and Head of Regulatory and Quality K. Robinson Lewis added, “Rare pediatric disease designation also offers a highly valuable incentive for MAIA. Upon FDA approval of a future new drug application in PDHGG, MAIA would be eligible to receive a priority review voucher that can be redeemed or sold as an asset at a very high valuation.”
Rare pediatric disease priority review vouchers (PRVs) can be redeemed by drug developers for FDA priority review of a different product or transferred or sold to another sponsor. Since 2015, FDA priority review vouchers have sold as assets at an average amount of
Previous research showcased THIO’s potency as a treatment for a PDHGG subtype known as diffuse intrinsic pontine glioma (DIPG). A research collaboration between MAIA and Nationwide Children's Hospital found that THIO combined with ionizing radiation (IR) resulted in significantly decreased cell proliferation and produced potent anticancer effects in highly aggressive DIPG. The data was presented in April 2024 at the American Association for Cancer Research (AACR) Annual Meeting.
MAIA collaborated with Only Orphans Cote for THIO’s designation request. Only Orphans Cote is a foremost provider of regulatory services and strategies for FDA orphan drug designations and marketing authorization.
In addition to its rare pediatric disease designation in PDHGG, THIO holds orphan drug designations (ODD) in three cancer types: hepatocellular carcinoma (HCC), small cell lung cancer (SCLC) and glioblastoma. MAIA believes that THIO is the only direct telomere-targeting agent currently in clinical development.
About THIO
THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine (THIO) induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. THIO-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with THIO followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. THIO is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.
About MAIA Biotechnology, Inc.
MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is THIO, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. For more information, please visit www.maiabiotech.com.
Forward Looking Statements
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1 Pharmaceutical Technology, GlobalData Pharma Intelligence Centre, January 2024
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Source: MAIA Biotechnology, Inc.
FAQ
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