NeOnc Technologies Holdings (NTHI) Study result summary

Study background and rationale

• NEO100-01 targets recurrent Grade 3 or 4 astrocytoma brain tumors with IDH1 mutations using intranasal delivery, a novel approach bypassing the blood-brain barrier.

• IDH1 mutant gliomas recur in nearly all cases, with post-recurrence survival typically only six to nine months and no effective treatments available.

• NEO100 is an ultra-pure perillyl alcohol delivered intranasally four times daily, aiming for non-invasive, chronic dosing at home.

• Mechanisms include induction of ER stress, inhibition of sodium-potassium ATPase, RAS inhibition, and cell cycle arrest, demonstrated in vitro and in vivo.

• The addressable US market is estimated at 7,000–11,000 new IDH1 mutant brain cancer cases annually.

Study design and patient population

• Phase II-A open-label, single-arm trial enrolled 24 patients (21 Grade 4, 3 Grade 3) with recurrent or progressive IDH1 mutant gliomas after prior radiation and temozolomide.

• Patients self-administered intranasal NEO100 four times daily in 28-day cycles at home.

• Study targeted a population with no approved targeted therapies and high recurrence rates.

• Study design aligns with conventional signal-seeking approaches for rare brain tumors.

• Future plans include a randomized phase III trial against lomustine for registrational purposes.

Efficacy and survival outcomes

• Primary endpoint met: six-month progression-free survival (PFS6) was 48.9%, more than double the 20% benchmark (p=0.0047).

• Median overall survival reached 26.09 months; 86.7% alive at six months, 60.9% at 12 months, and 54.1% at 24 months.

• Disease control rate (partial response + stable disease) was 66.7%.

• Objective response rate was 8.3% by RANO 2.0 criteria.

• Among 29 cumulative phase I/II patients, 55% were long-term survivors (>1 year post-recurrence), with some surviving over eight years.