Change access.
Keep the asset.
Non-invasive stimulation creates a temporary permeability window before ordinary systemic administration.
NEURO
Platform research · August 2026
What the data show.
What comes next.
A complete view of the platform: cross-species evidence, the clinical workflow, the Phase 0/1b strategy, commercialization logic and the path to human delivery.
Read the evidence ledger ↓01 / Executive reading
Change access.
AAV–ARSA
regional activity.
A functional enzyme readout shows delivery across deep brain regions versus a no-opening control.
Human exposure
and repeat-use safety.
The brain-oncology program uses scheduled resection to quantify drug levels, followed by repeated priming alongside standard care.
02 / Mechanism and workflow
A four-step
delivery sequence.
Prime
A 45-minute, non-invasive stimulation session transiently increases BBB permeability.
Infuse
The device is removed and the unchanged therapeutic is administered by standard IV infusion.
Deliver
The temporary opening increases exposure in the brain and infiltrative tumour margin.
Discharge
The workflow is ambulatory: no sedation, shaving, implant or overnight stay.
Platform advantage
Apertum creates a temporary BBB permeability window without surgery, implants, microbubbles or nanoparticles, then returns the patient to standard systemic care.
03 / Evidence ledger
Across species.
Into function.
| Model / asset | Outcome | Status | Platform value |
|---|---|---|---|
| Rodent | Efficacy and reversibility | Completed | Establishes reversible BBB opening and therapeutic delivery. |
| Porcine | BBB permeability | Completed | Extends permeability control into a large-animal model. |
| Non-human primate | Functional activity after systemic AAV–ARSA | Completed proof of concept | Demonstrates functional delivery across deep brain structures. |
| Antibody | 150 kDa biologic delivery | In progress | Expands the platform into large biologics. |
04 / NHP AAV–ARSA readout
A functional signal
in deep structures.
The 2026 Paris Brain Institute proof-of-concept study compared systemic AAV–ARSA plus Apertum treatment with a no-opening control.
ARSA activity increased across deep and cortical brain regions. Substantia nigra reached 646.1% of control, followed by caudate at 217.8%, putamen at 207.9% and thalamus at 195.0%.
Functional distribution: the pattern combines strong basal-ganglia and thalamic penetration with cortical gains, demonstrating region-spanning delivery after systemic administration.
Substantia nigra646.1%
Caudate217.8%
Putamen207.9%
Thalamus195.0%
Temporal cortex144.0%
Frontal cortex129.8%
Occipital cortex128.4%
Cerebellar white matter121.3%
Corpus callosum118.5%
White matter112.5%
Deep cerebellar nuclei110.1%
Hippocampus101.7%
Pons / brainstem98.7%
Cerebellar cortex96.6%
05 / Modality range
One access condition.
Four asset classes.
Baseline permeability threshold.
Delivery validated in rodent and porcine models.
NHP antibody validation in progress.
NHP delivery into deep structures validated with AAV–ARSA.
06 / Clinical program
Measure first.
Repeat second.
Phase 0 · N=30Proof of mechanism
Quantify delivery in tissue already scheduled for resection.
- Recurrent GBM and brain metastases
- Single 45-minute priming session
- IV doxorubicin or temozolomide
- Tumour core versus infiltrative margin
- LC–MS/MS ± fluorescence
- Exploratory ctDNA dynamics
Phase 1b · N=20Safety & early activity
Test repeated use alongside standard systemic therapy.
- Open-label, single-arm design
- Repeated 45-minute priming sessions
- TMZ-based standard therapy
- Interim reviews at 1 / 3 / 6 / 12 months
- Device-related AEs and DLTs
- RANO, PFS-6, OS-6 / OS-12 and steroid burden
Principal investigatorJames D. Battiste, MD, PhD · OU Health neuro-oncologyOU Health profile ↗
07 / Commercial strategy
Improve. Repurpose.
Rescue.
Improve
Increase brain exposure for approved or clinical-stage drugs without changing their structure. The commercial model combines upfront, milestone, per-treatment and royalty economics.
Repurpose
Enable peripherally approved drugs to pursue CNS indications—for example, systemic oncology assets in brain tumours.
Rescue
Revisit CNS programs shelved because delivery limited exposure despite a potentially useful biological mechanism.
Partner economics combine licensing, milestones, per-treatment revenue and 3–8% royalties, with strategic acquisition optionality through 2032.
08 / Financing plan
A $6M seed
toward human proof.
The $6M seed round is priced at a $25M pre-money valuation and funds first-in-human execution, modality expansion and initial pharma partnerships.
Salaries · 6 FTE$642K
FIH Phase 0/1b study$450K
MTA studies$200K
Device R&D$150K
IP & legal$150K
Business development & travel$50K
Initial 18-month operating allocation $1.642M across the core team, clinical work, MTAs, device R&D, IP and business development.
09 / Bottom line
Apertum has established a non-invasive, asset-agnostic CNS access platform across rodent, porcine and non-human-primate models. The next step is human proof of delivery.
Validated foundation: reversible BBB opening, large-animal permeability and functional NHP AAV–ARSA distribution across deep brain structures.
Clinical milestone: quantify human drug-exposure gains and establish repeat-use safety within an ambulatory workflow.
10 / Full platform brochure
Go deeper than
the landing page.
The 33-page brochure covers the preclinical evidence ladder, NHP ARSA readout, first-in-human program, regulatory path, commercial models and financing plan.