A Phase 1/2 Study to Evaluate OTX-2002 in Patients with Hepatocellular Carcinoma and Other Solid Tumor Types Known for Association with the MYC Oncogene

This is a Phase 1/2 open-label study to evaluate the safety, tolerability,

pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of OTX-2002 as a

single agent and in combination with standard of care in patients with hepatocellular

carcinoma (HCC) and other solid tumor types known for association with the MYC oncogene.

The study consists of Part 1 (OTX-2002 monotherapy) and Part 2 (OTX-2002 combined with

standard of care in hepatocellular carcinoma).

In Part 1, during dose escalation, participants with HCC and other solid tumors that

progressed on, relapsed after, are refractory to, or are intolerant of standard of care

for which no treatment options are available will be administered an intravenous infusion

of OTX-2002 as a single agent. The escalation will be conducted using a 3+3 design, with

the primary endpoint of dose limiting toxicity (DLT), maximum tolerated dose (MTD), and

incidence of treatment emergent adverse events (TEAEs). In Part 1 expansion, 15-25

participants with BCLC stage B or C, Child-Pugh Class A HCC who have received at least 1

prior line of systemic anticancer treatment, and without available subsequent standard of

care, will receive OTX-2002 at the recommended dose for expansion (RDE) for monotherapy.

The primary endpoint of Part 1 expansion will be overall response rate (ORR) and duration

of response (DoR).

In Part 2, during safety run-in, participants with BCLC stage B or C, Child-Pugh Class A

HCC who have received at least 1 prior line of systemic anticancer treatment, and without

available subsequent standard of care, will receive OTX-2002 at the selected dose in

combination with standard of care therapies at the local approved dose. The primary

endpoint of Part 2 safety run-in will be DLT, MTD, and incidence of TEAE. Once the

combination therapies have been determined to be tolerable in the safety run-in, 15-25

HCC participants will be enrolled in Part 2 expansion for each of the combination

therapies. The primary endpoint of Part 2 expansion will be ORR and DoR.