Oral AMXT 1501 Dicaprate in Combination With DFMO

The objective of this study is to determine the safety and tolerability of oral AMXT 1501
dicaprate (AMXT1501) in combination with DFMO in patients with advanced solid tumors.
Secondary objectives include characterization of plasma pharmacokinetics (PK) of AMXT 1501 as
well as pharmacodynamic (PD) assessment of the impact of AMXT 1501 in combination with DFMO
on polyamine uptake by circulating lymphocytes (blood cells).

To these aims, the study will evaluate the safety, PK and PD profiles of orally-administered
AMXT 1501 and DFMO. Approximately, 52 patients will be enrolled to determine the maximum
tolerated dose (MTD) and recommended phase 2 dose (RP2D) of AMXT 1501 and DFMO in
combination. The MTD is defined as the highest dose level below at which dose escalation is
stopped.

Inclusion Criteria:

1. Understand and sign, written Institutional Review Board (IRB)-approved informed
consent form, and be willing to comply with all study procedures

2. Participants must be > 18 years of age

3. Patient is able to take oral medications

4. Histologically or cytologically documented disease

5. Unresectable, locally advanced or metastatic solid tumor for which no standard therapy
is recognized or for which standard therapy has failed

6. Has evaluable or measurable disease by RECIST v1.1 criteria

7. Provide tumor tissue and/or archival tissue from original diagnostic block, if
available for biomarker analysis

8. Performance status of 0 or 1 on the Eastern Cooperative Oncology Group (ECOG) scale

9. Normal auditory acuity: defined as a normal age-related audiogram without significant
hearing loss.

10. Must have adequate bone marrow and renal/hepatic function at the screening and
baseline visits, defined as:

1. Absolute neutrophil count (ANC) ≥ 1.5 x 10^9/L without granulocyte
colony-stimulating factor (G-CSF) support within 7 days preceding the lab
assessment.

2. Platelet ≥100 x 10^9/L, without transfusion within 7 days preceding the lab
assessment

3. Hemoglobin ≥9 g/dL, without transfusion support within 7 days preceding the lab
assessment.

4. Activated partial thromboplastin time/ partial thromboplastin time (aPTT/PTT)
≤1.5 x ULN

5. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT)≤ 2.5 × upper
limit of normal (ULN) (if liver metastases are present, then≤5 x ULN is allowed)

6. Total serum bilirubin≤1.5 x ULN, (except for patients with known Gilbert's
Syndrome≤3 x ULN is permitted)

7. Thyroid function (T4, and T3) are within normal limits

8. Renal: Serum creatinine < 1.5 x ULN or creatinine clearance ≥60 mL/min/1.73m2 for
patients with serum creatinine levels above 1.5 x ULN.

9. Any Grade 3 or higher lab abnormalities should be discussed and approved by the
Medical Monitor prior to enrollment (even if not considered clinically
significant);

11. Patient compliance and geographic proximity (as determined by the Principal
Investigator) that allow adequate follow-up.

12. Both male and female patients must be willing to consent to using highly effective
contraception prior to study entry, while on treatment and at least 3 months
thereafter.

Exclusion Criteria:

1. Have a seizure disorder where > 1 seizure has occurred within the last year.

2. Patient has clinically significant cardiovascular disease (e.g., significant cardiac
conduction abnormalities, uncontrolled hypertension, myocardial infarction, cardiac
arrhythmia or unstable angina, New York Heart Association Grade 3 or greater
congestive heart failure, serious cardiac arrhythmia requiring medication, Grade 2 or
greater peripheral vascular disease, and history of cerebrovascular accident (CVA))
within 6 months of enrollment.

3. History or presence of an abnormal electrocardiogram (ECG) that, in the Investigator's
opinion, is clinically meaningful. Screening QTcF interval >480 ms is excluded. In the
event that a single QTcF is >480 ms, the subject may enroll if the average QTcF for
the 3 ECGs is < 480ms. For patients with an intraventricular conduction delay (QRS
interval >120ms), the JTc interval may be used in place of the QTcF with Sponsor
approval. The JTc must be <340 ms if JTc is used in place of the QTcF. Patients with
an intraventricular delay due to a left bundle branch block are excluded; Note: QTcF
prolongation due to pacemaker may enroll if the JTc is normal.

4. Patient with treated (surgically excised or irradiated) and stable brain metastases
are eligible as long as the treatment was at least 4 weeks prior to initiation of
study drug and baseline brain computed tomography (CT) with contrast or magnetic
resonance imaging (MRI) within 2 weeks of initiation of study drug is negative for new
brain metastases. Subjects with stable brain metastases must not require therapy with
corticosteroids

5. Have major surgery, other than diagnostic surgery, within 4-weeks prior to Day 1

6. Have active, bacterial, viral, or fungal infections, requiring systemic therapy

7. Women who are pregnant or lactating. NOTE: Women of childbearing potential (WOCBP)
must have a "negative" serum pregnancy test within one week prior to treatment.

a) Women not OCBP defined as any of the following: i) Postmenopausal with > 1 year
since last menses and:

(1) If younger than 65 years old, with a follicle-stimulating hormone (FSH) > 40 mIU/mL (2)
If older than 65 years old and not on hormone replacement therapy (HRT), with a FSH > 30
mIU/mL (3) If older than 65 years old and on HRT, the FSH requirement in not applicable.
Postmenopausal females on HRT will be allowed if the treatment is stable for at least 6
months prior to dosing of study drug(s) (4) Written medical documentation of being
sterilized (e.g. hysterectomy, double oophorectomy, bilateral salpingectomy) with the
procedure performed at least 6 months prior to dosing study drug(s). Note: Tubal ligation
is not considered a form of permanent sterilization (5) Should a woman become pregnant or
suspect she is pregnant while participating in this study, she should inform her treating
physician immediately.

8) Have undergone treatment with radiation therapy, surgery, chemotherapy, immunotherapy or
investigational therapy within one month prior to study entry (6 weeks for nitrosoureas or
Mitomycin C). Patients may also not have any unresolved toxicity > grade 1 from previous
anticancer therapy, except for stable chronic toxicities that are not expected to resolve
(i.e. peripheral neuropathy, alopecia etc.)

9) Have an unwillingness or inability to comply with procedures required in this protocol

10) Has jaundice or known current active liver disease from any cause, including hepatitis
A (hepatitis A virus immunoglobulin M (IgM) positive), hepatitis B (hepatitis B virus
surface antigen (HBsAg) positive), or hepatitis C (hepatitis C virus (HCV) antibody
positive, confirmed by HCV ribonucleic acid)

11) Have a serious nonmalignant disease that in the opinion of the Investigator and/ or the
Medical Monitor, could compromise protocol objectives in the opinion of the investigator
and/or the sponsor

12) Patients who are currently receiving any other investigational agent or who have
received an investigational agent within the last 28 days

13) Known gastrointestinal disease or procedure that could interfere with the absorption of
study drug, including inability to swallow whole capsules or conditions that may interfere
with absorption The medical monitor should be contacted for any questions regarding this
exclusion criterion

14) Patients who have exhibited allergic reactions to a similar structural compound,
biological agent, or formulation