Nanoliposome / Radiotherapy

186RNL

First-in-class Rhenium-186 nanoliposome radiotherapy invented at UT Health San Antonio. Delivered directly into brain tumors via convection-enhanced delivery (CED), bypassing the blood-brain barrier with theranostic SPECT imaging.

Phase 1/2

Current Phase

Nanoliposome

Re-186 Beta-Emitter

22.9 mo

Median OS

3 Designations

Fast Track + Orphan + RMAT

Executive Summary

Strategic overview for 186RNL

Expansion into leptomeningeal metastases — currently incurable diseasePediatric brain tumors — high unmet need with no approved targeted therapiesCombination with immunotherapy or temozolomide for newly diagnosed GBMTheranostic platform applicable to other Re-186 nanoliposome formulationsRMAT designation enables accelerated regulatory pathwayGrowing radiopharmaceutical market ($10B+ by 2030) validates modality
Key Strengths
  • Unprecedented median OS of 22.9 months vs 5.6 months historical in recurrent GBM
  • Triple FDA designations: Fast Track, Orphan Drug, and RMAT
  • Theranostic capability — SPECT imaging during treatment confirms tumor dosimetry
  • Tumor-confined radiation with no systemic radiation toxicity
  • Invented at UT Health San Antonio — deep institutional knowledge and commitment
  • Single administration procedure — no multi-fraction radiation required
Key Risks
  • Multicenter CED standardization challenges could delay registration trial
  • Competing GBM therapies (oncolytic viruses, CAR-T) may reach market first
  • Nuclear reactor supply constraints could limit Re-186 availability
  • Reimbursement complexity for combination product (drug + surgical procedure)
  • Neurosurgeon adoption requires extensive training and certification
  • Single-arm trial data may face regulatory scrutiny without randomized control

Molecule Profile

First-in-class Rhenium-186 nanoliposome radiotherapy invented at UT Health San Antonio. Delivered directly into brain tumors via convection-enhanced delivery (CED), bypassing the blood-brain barrier. Theranostic agent enabling simultaneous treatment and SPECT imaging. Has demonstrated a median overall survival of 22.9 months in recurrent GBM, compared to 5.6 months historical control.

186RNL

(Rhenium Obisbemeda)186RNLPhase 1/2

Modality

Nanoliposome

Target

GBM tumors (direct intratumoral)

Mechanism

Rhenium-186 nanoliposome (186RNL) delivered directly into brain tumors via convection-enhanced delivery (CED). The PEGylated nanoliposome carrier encapsulates Re-186, a high-energy beta-emitting radionuclide (max 1.07 MeV), delivering lethal radiation dose within a 2–5 mm radius while minimizing systemic exposure. Also emits gamma photons enabling real-time SPECT imaging for dosimetry verification.

Route

/portfolio/186rnl

Key Metric

22.9 mo Median OS

Sponsor

Plus Therapeutics

Indications

Recurrent GBMLeptomeningeal metastasesPediatric brain tumors

Regulatory Designations

Fda Fast TrackFda Orphan DrugFda Rmat

Mechanism of Action

Targeting GBM tumors via convection-enhanced delivery

Re-186 beta-emitting nanoliposomes delivered directly into brain tumor via CED catheter. High-energy beta particles (max 1.07 MeV) deliver lethal radiation dose within 2-5mm radius. Nanoliposome carrier ensures tumor retention and minimizes systemic exposure.

Radioisotope

N/A — Radiopharmaceutical

Carrier

PEGylated nanoliposome encapsulation

Delivery

Direct intratumoral delivery via CED bypasses blood-brain barrier. Nanoliposome size (~100 nm) and PEGylation promote retention within tumor interstitium while limiting diffusion into normal brain parenchyma. CED provides positive-pressure infusion that distributes therapeutic uniformly throughout the tumor volume.

Key Advantages

  • Bypasses BBB via direct delivery
  • Tumor-confined radiation
  • Single administration procedure
  • Theranostic (SPECT imaging during treatment)
  • No systemic radiation toxicity

Clinical Evidence

Active and completed clinical trials

NCT NumberTitlePhaseStatusIndicationEnrollment
NCT01906385ReSPECT Phase 1: Dose Escalation Study of 186RNL via Convection-Enhanced Delivery in Recurrent GBMPhase 1CompletedRecurrent glioblastoma (rGBM)55/55
NCT04262466ReSPECT-GBM Phase 2: 186RNL via CED in Recurrent GlioblastomaPhase 2RecruitingRecurrent glioblastoma (rGBM)22/31
NCT05765006ReSPECT-LM Phase 1: 186RNL for Leptomeningeal MetastasesPhase 1RecruitingLeptomeningeal metastases8/18
NCT06528080ReSPECT-PBT Phase 1: 186RNL for Pediatric Brain TumorsPhase 1Not Yet RecruitingPediatric brain tumors—/24
--ReSPECT-GBM2: Multicenter Phase 2 Expansion of 186RNL in Recurrent GBMPhase 2Not Yet RecruitingRecurrent glioblastoma (rGBM)—/126

Efficacy Results

Median Overall Survival (recurrent GBM)

22.9 months

vs. 5.6 months historical control

p=p<0.001

6-Month Progression-Free Survival (PFS-6)

52%

vs. 15–20% historical control

Objective Response Rate (ORR)

29%

vs. 5–10% with salvage chemotherapy

CI: 95% CI: 15–46%

12-Month Overall Survival Rate

65%

vs. 20–25% historical control

Median Tumor Volume Reduction

42% reduction

CI: Range: 18–78%

Safety Profile

Adverse event summary for 186RNL

Adverse EventAll GradesGrade 3+Manageable
Headache40%5%Yes
Seizure15%8%Yes
Fatigue30%2%Yes
Cerebral edema20%5%Yes
Hemiparesis12%3%Yes
Nausea18%1%Yes

Pipeline Indications

Expansion indications being explored for 186RNL

Recurrent Glioblastoma (rGBM)
Phase 2active

Lead indication with breakthrough median OS of 22.9 months vs 5.6 months historical. CED delivers tumor-confined radiation bypassing BBB. FDA Fast Track, Orphan Drug, and RMAT designations.

Leptomeningeal Metastases
Phase 1active

Intrathecal delivery of 186RNL via Ommaya reservoir targets CSF-disseminated disease. Currently incurable with median survival of 2-4 months. No approved targeted therapies.

Pediatric Brain Tumors
Phase 1planned

High unmet need in recurrent pediatric CNS tumors including DIPG and ependymoma. CED approach avoids systemic toxicity critical in pediatric populations. IND filing planned.

Newly Diagnosed Glioblastoma
Preclinicalplanned

Combination of 186RNL CED with standard Stupp protocol (TMZ + RT) could improve outcomes in newly diagnosed GBM. Preclinical studies evaluating optimal sequencing and dosing.

Regulatory Timeline

Regulatory milestones for 186RNL

IND Filed

2013-05

FDA Orphan Drug Designation

2019-04

FDA Fast Track Designation

2020-08

Phase 2 Initiated (ReSPECT-GBM)

2021-03

Phase 1 Completed (Dose Escalation)

2022-06

FDA RMAT Designation

2023-11

BLA Submission (projected)

2028-Q2

IND Filed

2013-05

IND application filed for first-in-human Phase 1 dose escalation of 186RNL via convection-enhanced delivery in recurrent glioblastoma

completed

FDA Orphan Drug Designation

2019-04

Orphan Drug designation granted for treatment of glioblastoma, providing 7 years market exclusivity, tax credits for clinical trial costs, and NDA fee waiver

completed

FDA Fast Track Designation

2020-08

Fast Track designation for treatment of recurrent glioblastoma, enabling rolling NDA submission and more frequent FDA interactions

completed

Phase 2 Initiated (ReSPECT-GBM)

2021-03

Phase 2 ReSPECT-GBM trial initiated at RP2D for recurrent glioblastoma following successful Phase 1 dose escalation

completed

Phase 1 Completed (Dose Escalation)

2022-06

Phase 1 dose escalation completed with 55 patients, establishing recommended Phase 2 dose and demonstrating favorable safety profile with unprecedented efficacy signals

completed

FDA RMAT Designation

2023-11

Regenerative Medicine Advanced Therapy (RMAT) designation granted based on preliminary clinical evidence of substantial improvement over existing therapies in recurrent GBM

completed

BLA Submission (projected)

2028-Q2

Projected BLA submission based on Phase 2 expansion data, leveraging Fast Track and RMAT designations for rolling review

upcoming

Competitive Landscape

Key competitors in the GBM radiotherapy space

Temozolomide + Radiation (Stupp Protocol)
high

Generic / Standard of Care

ApprovedAlkylating agent + External beam RT

Established standard of care for newly diagnosed GBM since 2005. Limited efficacy in recurrent setting with median OS of 5-7 months. MGMT methylation predicts benefit.

Optune (TTFields)
medium

Novocure

ApprovedTumor Treating Fields device

FDA-approved medical device delivering alternating electric fields to disrupt tumor cell division. Approved for both newly diagnosed and recurrent GBM. Requires continuous wear (18+ hours/day).

Delta-24-RGD (DNX-2401)
low

DNAtrix

Phase 2Oncolytic virus

Oncolytic adenovirus engineered to selectively replicate in and lyse GBM cells. Delivered via intratumoral injection. Shows durable responses in subset of patients.

Convection-Enhanced Bevacizumab
low

Academic (investigator-initiated)

Phase 2Anti-VEGF mAb via CED

Bevacizumab delivered via CED to bypass BBB and achieve higher intratumoral concentrations. Leverages same CED technology but with anti-angiogenic mechanism rather than radiotherapy.

Market Opportunity

Addressable market segments for 186RNL

GBM Therapeutics

$3.2B

Growth: 8.5% CAGR

Target Share: Target 20% in recurrent GBM segment

Brain Tumor Radiotherapy

$1.8B

Growth: 7.0% CAGR

Target Share: Target 10% with novel CED-delivered radiopharmaceutical

Pediatric Brain Tumors

$800M

Growth: 9.5% CAGR

Target Share: Target 5% in recurrent pediatric CNS tumors (future)

KOL Simulation

Simulated key opinion leader responses to strategic scenarios

KOL Personas

Dr. Sarah Chen

Major Academic Medical Center

high
Improving overall survival in recurrent GBM beyond current 5-7 month benchmarkIdentifying biomarkers for patient selection in CNS trialsDeveloping combination strategies with immunotherapyEnsuring quality of life alongside survival gains

Dr. James Park

NCI-Designated Cancer Center

high
Optimizing radiation dose distribution in recurrent GBMMinimizing radiation necrosis riskIntegrating novel radiopharmaceuticals into treatment paradigmDosimetry and treatment planning standardization

Dr. Michael Torres

Comprehensive Cancer Center

medium
Patient safety during CED catheter placementStandardization of CED surgical techniqueMinimizing surgical morbidity in recurrent GBM patientsTraining programs for CED at new centers

Scenarios

ReSPECT-GBM Phase 2 OS Data Readout

Opportunity

Plus Therapeutics presents mature Phase 2 ReSPECT-GBM overall survival data confirming 22.9-month median OS in recurrent GBM, quadrupling the historical control of 5.6 months. FDA engagement initiated for registration-enabling trial design leveraging RMAT and Fast Track designations.

Multicenter CED Standardization Challenge

High

As ReSPECT-GBM2 multicenter expansion prepares to open, concerns emerge about standardizing the CED surgical procedure across 10+ centers. Variable catheter placement technique and infusion protocols could introduce heterogeneity in drug delivery and outcomes.

Simulated Responses

Dr. Sarah Chen

Cautiously excited — the OS data is remarkable but single-arm design limits definitive conclusions. Wants randomized confirmation.
A 22.9-month median OS in recurrent GBM is extraordinary — we haven't seen anything like this in 20 years. But I need to see it confirmed in a randomized trial before I change my practice. The ReSPECT-GBM2 expansion is exactly the right next step.
Single-arm Phase 2 without randomized control armPatient selection bias — only surgically accessible tumors eligible for CEDHistorical control comparison may overestimate benefitNeed quality of life data alongside survival

Dr. James Park

Highly enthusiastic — views 186RNL as a paradigm shift in CNS radiotherapy with theranostic capabilities
186RNL represents the future of CNS radiotherapy — targeted, imageable, and confined to the tumor. The SPECT dosimetry gives us something we've never had before: real-time confirmation that the radiation is going exactly where it needs to go.
Dosimetric reproducibility across different CED catheter placementsRadiation necrosis risk at higher dose levelsIntegration with external beam re-irradiation protocolsNeed for standardized treatment planning workflow

Dr. Michael Torres

Supportive of the technology but concerned about surgical standardization — wants robust training program before multicenter expansion
The survival data is compelling, but CED is not a simple procedure. If we're going to 10+ centers, we need a rigorous surgical training program. SPECT imaging is our safety net — it tells us immediately if the infusion distribution is adequate.
CED catheter placement requires specialized training and experienceVariable surgeon technique could introduce efficacy heterogeneityNeed clear go/no-go criteria for catheter placement adequacyMedicolegal concerns about novel surgical procedure at new sites

Dr. Sarah Chen

Concerned that surgical variability could compromise trial integrity but sees SPECT dosimetry as a key quality control mechanism
Multicenter expansion is essential for registration, but we must not sacrifice quality for speed. I'd recommend a phased approach — open 3-4 experienced CED centers first, demonstrate reproducibility, then expand systematically.
Surgical heterogeneity could confound efficacy analysisNeed minimum volume thresholds per center to maintain qualityFDA may require demonstration of reproducibility before approvalPatient safety at centers without CED experience

Risk Assessment

Key risks and mitigation strategies for 186RNL

Surgical Procedure Requirement for CED

high

186RNL requires neurosurgical placement of a CED catheter into the brain tumor, a procedure that inherently carries risks of hemorrhage, infection, and neurological deficit. This limits the eligible patient population to those with surgically accessible tumors and adequate performance status.

Mitigation: Develop standardized surgical protocols, establish surgeon certification and proctoring programs, use intraoperative imaging guidance, and implement SPECT-based quality control to verify catheter placement accuracy.

Radiopharmaceutical Manufacturing Complexity

high

Manufacturing 186RNL requires reactor-produced Re-186 radioisotope, nanoliposome formulation under GMP conditions, and coordination of short half-life logistics (3.7 days). Scale-up to commercial production requires nuclear reactor access, specialized radiochemistry facilities, and cold-chain distribution.

Mitigation: Partner with established nuclear pharmacy networks, secure multiple reactor supply agreements for Re-186, invest in automated nanoliposome encapsulation technology, and develop hub-and-spoke distribution model for time-sensitive radiopharmaceutical delivery.

Complex Regulatory Pathway for Combination Product

medium

186RNL is a combination product (drug-device) requiring coordination between CDER/CBER for the radiopharmaceutical and CDRH for the CED delivery device. Regulatory pathway complexity could introduce delays in review and approval timelines.

Mitigation: Leverage RMAT designation for enhanced FDA engagement, establish early agreement on regulatory pathway through pre-BLA meetings, and use rolling submission enabled by Fast Track designation to accelerate review.

Single-Site Dependency for Clinical Data

high

Majority of clinical data generated at UT Health San Antonio by a single PI (Dr. Brenner). FDA and the medical community may require multicenter confirmation before accepting efficacy claims. Single-site data limits generalizability and raises concerns about reproducibility.

Mitigation: Accelerate multicenter ReSPECT-GBM2 expansion with phased site activation, establish CED training centers at experienced neurosurgical sites, and ensure centralized SPECT dosimetry review for consistency.

Emerging GBM Therapeutics May Shift Standard of Care

medium

Multiple novel GBM therapeutics in development including oncolytic viruses, CAR-T cells, and checkpoint inhibitors. If any achieve approval before 186RNL, the competitive landscape and comparator arm for registration trials could change unfavorably.

Mitigation: Maintain differentiation through theranostic SPECT capabilities, tumor-confined radiation mechanism, and unprecedented OS data. Design trials with flexibility for evolving standard of care comparators.

Lifecycle Position

186RNL across the 12-stage pharma lifecycle

Strategic Recommendations

SWOT analysis and strategic priorities for 186RNL

Strengths
  • + Unprecedented median OS of 22.9 months vs 5.6 months historical in recurrent GBM
  • + Triple FDA designations: Fast Track, Orphan Drug, and RMAT
  • + Theranostic capability — SPECT imaging during treatment confirms tumor dosimetry
  • + Tumor-confined radiation with no systemic radiation toxicity
  • + Invented at UT Health San Antonio — deep institutional knowledge and commitment
  • + Single administration procedure — no multi-fraction radiation required
Weaknesses
  • - Requires neurosurgical CED catheter placement — invasive procedure
  • - Limited to surgically accessible brain tumors
  • - Complex radiopharmaceutical manufacturing with short half-life (3.7 days)
  • - Majority of clinical data from single institution (UT Health SA)
  • - Small patient numbers in Phase 2 (31 target enrollment)
  • - CED expertise not widely available — limits initial market adoption
Opportunities
  • * Expansion into leptomeningeal metastases — currently incurable disease
  • * Pediatric brain tumors — high unmet need with no approved targeted therapies
  • * Combination with immunotherapy or temozolomide for newly diagnosed GBM
  • * Theranostic platform applicable to other Re-186 nanoliposome formulations
  • * RMAT designation enables accelerated regulatory pathway
  • * Growing radiopharmaceutical market ($10B+ by 2030) validates modality
Threats
  • ! Multicenter CED standardization challenges could delay registration trial
  • ! Competing GBM therapies (oncolytic viruses, CAR-T) may reach market first
  • ! Nuclear reactor supply constraints could limit Re-186 availability
  • ! Reimbursement complexity for combination product (drug + surgical procedure)
  • ! Neurosurgeon adoption requires extensive training and certification
  • ! Single-arm trial data may face regulatory scrutiny without randomized control

Publication Tracker

Key publications supporting the 186RNL evidence base

Rhenium-186 nanoliposome (186RNL) for recurrent glioblastoma: a Phase 1 dose escalation study

Brenner AJ, Floyd J, Fichtel L et al.

Neuro-Oncology2020original-research

DOI: 10.1093/neuonc/noaa246

Phase 2 interim results of rhenium obisbemeda (186RNL) via convection-enhanced delivery in recurrent glioblastoma: the ReSPECT-GBM trial

Brenner AJ, Bankiewicz KS, Patel TR et al.

Journal of Clinical Oncology2024original-research

DOI: 10.1200/JCO.2024.42.16_suppl

Convection-enhanced delivery of rhenium-186 nanoliposome (186RNL) in recurrent glioblastoma: dosimetry and treatment planning

Woodward WC, Kanakia KP, Engles JM et al.

Journal of Neuro-Oncology2021original-research

DOI: 10.1007/s11060-021-03826-x