Plus Therapeutics Announces FDA Clearance of its Investigational New Drug Application using REYOBIQTM for the Treatment of Childhood Brain Cancer
Plus Therapeutics (NASDAQ:PSTV) has received FDA clearance for its Investigational New Drug (IND) application for REYOBIQ™, targeting pediatric brain cancers. The Phase 1/2a clinical trial, supported by a $3.0 million Department of Defense grant, will focus on treating children with high-grade glioma (HGG) and ependymoma.
The trial consists of two parts: a Phase 1a/b dose escalation study enrolling approximately 24 patients to determine maximum tolerated dose, and a Phase 2a efficacy study with 32 patients. REYOBIQ delivers targeted beta radiation directly to brain tumors while minimizing damage to healthy tissue. The therapy builds on promising results from the adult recurrent glioblastoma trial, which showed doubled overall survival in patients receiving therapeutic radiation doses.
Plus Therapeutics (NASDAQ:PSTV) ha ottenuto l'autorizzazione della FDA per la sua domanda di Nuovo Farmaco Sperimentale (IND) per REYOBIQ™, rivolto ai tumori cerebrali pediatrici. Lo studio clinico di Fase 1/2a, supportato da una borsa di studio da 3,0 milioni di dollari del Dipartimento della Difesa, si concentrerà sul trattamento di bambini con glioma di alto grado (HGG) ed ependimoma.
La sperimentazione è composta da due fasi: uno studio di escalation della dose di Fase 1a/b che arruolerà circa 24 pazienti per determinare la dose massima tollerata, e uno studio di efficacia di Fase 2a con 32 pazienti. REYOBIQ somministra radiazioni beta mirate direttamente ai tumori cerebrali, minimizzando i danni ai tessuti sani. La terapia si basa su risultati promettenti ottenuti nel trial su glioblastoma ricorrente negli adulti, che ha mostrato un raddoppio della sopravvivenza complessiva nei pazienti trattati con dosi terapeutiche di radiazioni.
Plus Therapeutics (NASDAQ:PSTV) ha recibido la aprobación de la FDA para su solicitud de Nuevo Fármaco en Investigación (IND) para REYOBIQ™, dirigido a cánceres cerebrales pediátricos. El ensayo clínico de Fase 1/2a, respaldado por una subvención de 3.0 millones de dólares del Departamento de Defensa, se enfocará en tratar a niños con glioma de alto grado (HGG) y ependimoma.
El ensayo consta de dos partes: un estudio de escalada de dosis de Fase 1a/b que inscribirá aproximadamente 24 pacientes para determinar la dosis máxima tolerada, y un estudio de eficacia de Fase 2a con 32 pacientes. REYOBIQ administra radiación beta dirigida directamente a los tumores cerebrales, minimizando el daño al tejido sano. La terapia se basa en resultados prometedores del ensayo en adultos con glioblastoma recurrente, que mostró una duplicación de la supervivencia global en pacientes que recibieron dosis terapéuticas de radiación.
Plus Therapeutics (NASDAQ:PSTV)가 소아 뇌종양을 대상으로 한 REYOBIQ™의 임상시험신청(IND)에 대해 FDA 승인을 받았습니다. 미 국방부의 300만 달러 지원금으로 지원되는 1/2a상 임상시험은 고등급 교모세포종(HGG)과 상피종을 가진 어린이 치료에 중점을 둘 예정입니다.
임상시험은 두 부분으로 구성됩니다: 최대 허용 용량을 결정하기 위해 약 24명의 환자를 등록하는 1a/b상 용량 증량 연구, 그리고 32명의 환자를 대상으로 하는 2a상 효능 연구입니다. REYOBIQ는 건강한 조직 손상을 최소화하면서 뇌종양에 직접 표적 베타 방사선을 전달합니다. 이 치료법은 성인 재발성 교모세포종 임상시험에서 치료 방사선 투여 환자의 전체 생존 기간이 두 배로 늘어난 유망한 결과를 바탕으로 합니다.
Plus Therapeutics (NASDAQ:PSTV) a reçu l'autorisation de la FDA pour sa demande de nouveau médicament expérimental (IND) concernant REYOBIQ™, destiné aux cancers cérébraux pédiatriques. L'essai clinique de phase 1/2a, soutenu par une subvention de 3,0 millions de dollars du Département de la Défense, se concentrera sur le traitement des enfants atteints de gliome de haut grade (HGG) et d'épendymome.
L'essai comprend deux parties : une étude d'escalade de dose de phase 1a/b recrutant environ 24 patients pour déterminer la dose maximale tolérée, et une étude d'efficacité de phase 2a avec 32 patients. REYOBIQ délivre une radiation bêta ciblée directement aux tumeurs cérébrales tout en minimisant les dommages aux tissus sains. Ce traitement s'appuie sur les résultats prometteurs de l'essai chez des adultes atteints de glioblastome récidivant, qui a montré un doublement de la survie globale chez les patients recevant des doses thérapeutiques de radiation.
Plus Therapeutics (NASDAQ:PSTV) hat die FDA-Zulassung für seinen Antrag auf ein Prüfpräparat (IND) für REYOBIQ™ erhalten, das sich gegen pädiatrische Hirntumore richtet. Die klinische Phase 1/2a Studie, unterstützt durch einen 3,0 Millionen Dollar Zuschuss des Verteidigungsministeriums, konzentriert sich auf die Behandlung von Kindern mit hochgradigem Gliom (HGG) und Ependymom.
Die Studie besteht aus zwei Teilen: Eine Phase 1a/b Dosis-Eskalationsstudie mit etwa 24 Patienten, um die maximal verträgliche Dosis zu bestimmen, und eine Phase 2a Wirksamkeitsstudie mit 32 Patienten. REYOBIQ liefert gezielte Betastrahlung direkt an Hirntumore und minimiert dabei Schäden am gesunden Gewebe. Die Therapie baut auf vielversprechenden Ergebnissen der Studie bei erwachsenen Patienten mit rezidivierendem Glioblastom auf, die eine Verdopplung des Gesamtüberlebens bei Patienten mit therapeutischer Strahlendosis zeigte.
- FDA clearance received for pediatric brain cancer IND application
- Secured $3.0M Department of Defense grant funding for the trial
- Previous adult trials showed doubled overall survival with therapeutic doses
- Novel delivery method bypasses blood-brain barrier for potentially improved outcomes
- Target diseases (HGG/ependymoma) have poor prognosis with 5-year survival rates as low as 22%
- Early-stage trial with no guaranteed success
- Limited patient population (3.3 cases per 100,000 persons)
Insights
FDA clearance for REYOBIQ in pediatric brain cancer marks significant progress; novel radiation delivery mechanism offers new hope for poor-prognosis patients.
The FDA's clearance of Plus Therapeutics' IND application for REYOBIQ represents a critical regulatory milestone in addressing one of pediatric oncology's most challenging diseases. High-grade gliomas and ependymomas in children have seen virtually no improvement in outcomes for decades, with 5-year survival rates as low as 22% despite aggressive surgery and radiation.
What makes REYOBIQ particularly promising is its innovative delivery mechanism. Using convection enhanced delivery (CED), this radiotherapeutic bypasses the blood-brain barrier to deliver radiation (Rhenium Re186 Obisbemeda) directly to tumor tissue. This approach potentially solves two critical challenges in pediatric neuro-oncology: delivering sufficient radiation to aggressive tumors while minimizing exposure to the developing brain.
The $3.0 million DoD grant funding this study demonstrates external validation of this approach. The two-part trial design is methodologically sound, with a Phase 1a/b determining maximum tolerated dose in approximately 24 patients, followed by a Phase 2a efficacy assessment in 32 patients.
Particularly encouraging is that this pediatric application builds upon promising adult data. Their ReSPECT-GBM trial in adult glioblastoma reportedly demonstrated doubled overall survival in patients receiving therapeutic radiation doses (>100 Gy) - though pediatric responses may differ.
While this remains an early clinical development step with years of testing ahead, the IND clearance represents meaningful progress in a field desperate for new approaches. The ability to precisely target radiation therapy could fundamentally change treatment paradigms for these devastating childhood cancers.
Phase 1/2a clinical trial currently supported by a
Trial will enroll children with high grade glioma and ependymoma
HOUSTON, June 25, 2025 (GLOBE NEWSWIRE) -- Plus Therapeutics, Inc. (Nasdaq: PSTV) (the “Company”), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system (CNS) cancers, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application (No. 168178) for REYOBIQTM (Rhenium Re186 Obisbemeda) for the treatment of pediatric patients with supratentorial recurrent, refractory, or progressive high-grade glioma (HGG) and ependymoma. The trial will be referred to as the ReSPECT-PBC trial and is funded by a
"We are very excited for the opportunity to study the novel radiotherapeutic, REYOBIQ, in difficult-to-treat pediatric brain tumors. Surgery and external beam radiation have been the mainstays of treatment for pediatric high-grade glioma and, sadly, outcomes have not improved for many decades. We believe this novel therapy that may allow higher doses of radiation while limiting the exposure to normal developing brain and delivering this agent via convection enhanced delivery (CED) directly into the tumor has the potential to make a big difference in our patients' lives and improve outcomes,” said Dr. Ashley S. Plant, M.D., Principal Investigator for the trial, attending physician in Hematology, Oncology, Neuro-oncology, and Stem Cell Transplantation at Ann & Robert H. Lurie Children's Hospital of Chicago, A.M. Khokhar Research Scholar and Associate Professor of Pediatrics at Feinberg School of Medicine Northwestern University. “I am extremely passionate about bringing this therapy into the pediatric space and hopeful about the benefits to my patients."
Phase 1/2a Trial Design Highlights:
The Phase 1/2a trial is a two-part, single-arm, prospective study aimed at determining the maximum tolerated dose (MTD), safety, and tolerability of REYOBIQ in pediatric patients aged 6 to 21 years (with consideration for patients up to 25 years on a case-by-case basis).
Key elements of the trial design include:
- Phase 1a/b (Dose Escalation): This phase will enroll an estimated 24 patients using a modified 3+3 dose escalation scheme to establish the MTD and recommended Phase 2 dose (RP2D). Safety assessment and alignment with the FDA will occur at defined intervals.
- Phase 2a: This phase will enroll approximately 32 patients (12 with ependymoma and 20 with HGG) at the RP2D to assess efficacy.
Pediatric HGG including ependymoma are rare (approximately 3.3 cases per 100,000 persons) but aggressive brain tumors with limited treatment options and poor prognosis, particularly in recurrent settings. Standard treatments, including surgical resection and external beam radiation therapy, often fail to prevent recurrence, with 5-year survival rates as low as
“This pediatric FDA clearance builds on our successful track record in the safe administration and promising efficacy signals observed in adult patients with more common central nervous cancers,” said Marc Hedrick, M.D., Plus Therapeutics President and Chief Executive Officer. “We expect REYOBIQ will offer much needed hope to children with aggressive and difficult-to-treat brain cancers and their families.”
REYOBIQ is a novel radiotherapeutic designed to deliver high doses of beta radiation directly to brain tumors while minimizing damage to surrounding healthy tissue. The ReSPECT-PBC (pediatric brain cancer) trial builds on promising preclinical data and clinical results from the Company’s adult recurrent glioblastoma trial (ReSPECT-GBM). As recently published in Nature Communications, ReSPECT-GBM demonstrated favorable safety and clinical response with a doubling of overall survival for those patients receiving a therapeutic dose of radiation defined as > 100 Gy.
For more information, please contact @plustherapeutics.com or visit www.clinicaltrials.gov (identifier to be assigned upon registration).
About REYOBIQ™ (rhenium Re186 obisbemeda)
REYOBIQ™ (rhenium Re186 obisbemeda) is a novel injectable radiotherapy specifically formulated to deliver direct targeted high dose radiation in CNS tumors in a safe, effective, and convenient manner to optimize patient outcomes. REYOBIQ™ has the potential to reduce off target risks and improve outcomes for CNS cancer patients, versus currently approved therapies, with a more targeted and potent radiation dose. Rhenium-186 is an ideal radioisotope for CNS therapeutic applications due to its short half-life, beta energy for destroying cancerous tissue, and gamma energy for real-time imaging. REYOBIQ™ is being evaluated for the treatment of recurrent glioblastoma and leptomeningeal metastases in the ReSPECT-GBM and ReSPECT-LM clinical trials. ReSPECT-GBM is supported by an award from the National Cancer Institute (NCI), part of the U.S. National Institutes of Health (NIH), and ReSPECT-LM is funded by a three-year
About Plus Therapeutics
Headquartered in Houston, Texas, Plus Therapeutics, Inc. is a clinical-stage pharmaceutical company developing targeted radiotherapeutics for difficult-to-treat cancers of the central nervous system with the potential to enhance clinical outcomes. Combining image-guided local beta radiation and targeted drug delivery approaches, the Company is advancing a pipeline of product candidates with lead programs in leptomeningeal metastases (LM) and recurrent glioblastoma (GBM) and pediatric brain cancer (PBC). The Company has built a supply chain through strategic partnerships that enable the development, manufacturing, and future potential commercialization of its products. For more information, visit https://plustherapeutics.com/.
Forward-Looking Statements
This press release contains statements that may be deemed “forward-looking statements” within the meaning of U.S. securities laws, including statements regarding trial design, clinical outcomes, product candidates, expected operations and upcoming developments. All statements in this press release other than statements of historical fact are forward-looking statements. These forward-looking statements may be identified by future verbs, as well as terms such as “expect” “potential,” “anticipating,” “planning” and similar expressions or the negatives thereof. Such statements are based upon certain assumptions and assessments made by management in light of their experience and their perception of historical trends, current conditions, expected future developments and other factors they believe to be appropriate.
The forward-looking statements included in this press release could differ materially from those expressed or implied by these forward-looking statements because of risks, uncertainties, and other factors that include, but are not limited to, the following: the Company’s ability to maintain the listing of its common stock on Nasdaq; risks related to a halt in trading or delisting of the Company’s common stock on Nasdaq; the early stage of the Company’s product candidates and therapies; the results of the Company’s research and development activities, including uncertainties relating to the clinical trials of its product candidates and therapies; the Company’s liquidity and capital resources and its ability to raise additional cash; the outcome of the Company’s partnering/licensing efforts, risks associated with laws or regulatory requirements applicable to it; market conditions; product performance; litigation or potential litigation; competition within the cancer diagnostics and therapeutics field; ability to develop and protect proprietary intellectual property or obtain licenses to intellectual property developed by others on commercially reasonable and competitive terms; challenges associated with radiotherapeutic manufacturing; production and distribution capabilities necessary to support the Company’s clinical trials and any commercial level product demand; and material security breach or cybersecurity attack affecting the Company’s operations or property. This list of risks, uncertainties, and other factors is not complete. Plus Therapeutics discusses some of these matters more fully, as well as certain risk factors that could affect Plus Therapeutics’ business, financial condition, results of operations, and prospects, in its reports filed with the SEC, including Plus Therapeutics’ annual report on Form 10-K for the fiscal year ended December 31, 2024, quarterly reports on Form 10-Q, and current reports on Form 8-K. These filings are available for review through the SEC’s website at www.sec.gov. Any or all forward-looking statements Plus Therapeutics makes may turn out to be incorrect and can be affected by inaccurate assumptions Plus Therapeutics might make or by known or unknown risks, uncertainties, and other factors, including those identified in this press release. Accordingly, you should not place undue reliance on the forward-looking statements made in this press release, which speak only as of its date. The Company assumes no responsibility to update or revise any forward-looking statements to reflect events, trends or circumstances after the date they are made unless the Company has an obligation under U.S. federal securities laws to do so.
Investor Contact
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