A Phase 1 Study Evaluating CPI-0610 in Patients With Previously Treated Multiple Myeloma

Inclusion Criteria:

- Adults (aged ≥ 18 years)

- Histologically or cytologically confirmed diagnosis of multiple myeloma that has
progressed despite at least one line of standard therapy

- Must have measurable disease, defined by one or more of following: (i) a serum M
protein > 0.5 g/dl measured by serum protein electrophoresis; (ii) urinary M protein
excretion > 200 mg/24 hours; (iii) serum free light chain (FLC) measurement > 10
mg/dl, provided that the serum FLC ratio is abnormal

- Eastern Cooperative Oncology Group (ECOG) performance status ≤2

- Adequate hematological, renal, hepatic, and coagulation laboratory assessments

- Written informed consent to participate in this study before the performance of any
study-related procedure

Exclusion Criteria:

- Current infection with HIV, Hepatitis B or Hepatitis C

- Impairment of gastrointestinal (GI) function or GI disease that may significantly
alter the absorption of CPI-0610, including any unresolved nausea, vomiting, or
diarrhea that is CTCAE grade >1

- Impaired cardiac function or clinically significant cardiac diseases, including any
of the following:

- Acute myocardial infarction or angina pectoris ≤ 6 months prior to starting
study drug

- Serum cardiac troponin (cTn) level ≥ 99% percentile of the upper reference limit

- QTcF > 470 msec on the screening ECG

- Left ventricular ejection fraction (LVEF) < 50%

- Uncontrolled cardiac arrhythmia (patients with rate-controlled atrial fibrillation
are not excluded.)

- Any other concurrent severe and/or uncontrolled concomitant medical condition that
could compromise participation in the study (e.g., clinically significant pulmonary
disease, clinically significant neurological disorder, active or uncontrolled
infection)

- Systemic anti-cancer treatment or radiotherapy less than 2 weeks before the first
dose of CPI-0610

- Treatment with an investigational small molecule less than 2 weeks before the first
dose of CPI-0610. In addition, the first dose of CPI-0610 should not occur before a
period equal to or greater than 5 half-lives of the small molecule investigational
agent has elapsed

- Treatment with a therapeutic antibody less than 4 weeks before the first dose of
CPI-0610. A minimum 2-week period between the last treatment with a therapeutic
antibody and the first dose of CPI-0610 may be permitted in patients with rapidly
progressive myeloma, following discussion with the medical monitor

- Treatment with medications that are known to be strong inhibitors or inducers of
CYP450 enzymes

- Treatment with medications that are known to carry a risk of Torsades de Pointes

- Immunosuppressive treatment that cannot be discontinued both prior to study entry and
for the duration of the study. Oral prednisone at a dose of 10 mg or less per day is
allowed, as are other oral corticosteroids given at glucocorticoid-equivalent doses.
Topical, nasal and inhaled corticosteroids are also allowed

- Pregnant or lactating women

- Women of child-bearing potential and men with reproductive potential, if they are
unwilling to use adequate contraception while on study therapy and for 3 months
thereafter

- Patients unwilling or unable to comply with the study protocol