Study of the Safety, Tolerability and Efficacy of KPT-8602 in Patients With Relapsed/Refractory Cancer Indications

INCLUSION CRITERIA

1. Written informed consent obtained prior to any screening procedures and in accordance
with federal, local, and institutional guidelines.

2. Age ≥ 18 years.

Higher Risk Myelodysplastic Syndrome (Part F):

3. Documented diagnosis of MDS with 5-19% myeloblasts.

4. Patients should be intermediate-2 or high-risk MDS by International Prognostic Scoring
System (IPSS).

5. Patients believed to be IPSS high risk, without clearly meeting IPSS categories above
should be discussed with the medical monitor prior to enrolling.

6. HMA refractory patients including:

1. ≥ 2 cycles of azacitidine and/or decitabine or experimental agents (such as
SGI-110 or ASTX727 or similar) with clear progressive disease (PD) (no count
recovery with ≥50% increase in bone marrow blasts)

OR

2. ≥ 4 cycles of azacitidine and/or decitabine (or other hypomethylating therapy)
with lack of improvement (no CR/CRi/PR/HI).

7. Patients receiving a stable dose of erythropoiesis-stimulating agent (ESA) for at
least 1 month at the time of study entry may continue to receive ESA.

EXCLUSION CRITERIA

Patients in All Parts of the Study:

1. Major surgery within 4 weeks before C1D1.

2. Impaired cardiac function or clinically significant cardiac diseases.

3. Uncontrolled active severe systemic infection requiring parenteral antibiotics,
antivirals, or antifungals within one week prior to C1D1.

4. Patients with known symptomatic brain metastasis.

5. Prior malignancies:

1. Patients in All Parts of the Study: Patients with adequately resected basal or
squamous cell carcinoma of the skin, or adequately resected carcinoma in situ
(i.e. cervix) may enroll irrespective of the time of diagnosis.

2. Patients with Higher Risk MDS only: Concomitant malignancies or previous
malignancies with less than a 1-year disease free interval at the time of
enrollment.

INDICATION-SPECIFIC EXCLUSION CRITERIA

Higher risk Myelodysplastic Syndrome (Part F):

6. IPSS low or intermediate-1 risk MDS.

7. Evidence of transformation to AML by World Health Organization (WHO) (≥20% blasts in
bone marrow or peripheral blood).

8. Patients receiving granulocyte-colony stimulating factor (G-CSF) or granulocyte
macrophage-colony stimulating factor (GM-CSF) within the 3 weeks prior to C1D1.