Study of Safety and Efficacy in Patients With Malignant Rhabdoid Tumors (MRT) and Neuroblastoma

Due to lack of efficacy, enrollment in the study was stopped at the end of dose escalation
(sites were notified of the early enrollment halt on 7-Aug-2014) and the dose-expansion part
was not conducted. Due to halted enrollment and/or lack of complete responses (CR) and
partial responses (PR), efficacy analysis was only performed in terms of TTP for the patients
treated during the dose-escalation part at the maximum tolerated dose (MTD) and recommended
dose expansion (RDE).

Inclusion Criteria:

- Confirmed diagnosis of MRT or, neuroblastoma or in dose escalation part, other tumors
with documented evidence of D-cyclin-CDK4/6-INK4a-Rb pathway abnormalities (dose
escalation part only),

- Patients with CNS disease should be on stable doses of steroids for at least 7 days
prior to first dose of LEE011 with no plans for escalation.

- In expansion part, patients must have at least one measurable disease as defined by
RECIST v1.1.

- Patients must have a Lansky (≤ 16 years) or Karnofsky (> 16 years) score of at least
50.

Exclusion Criteria:

- Prior history of QTc prolongation or QTcF > 450 ms on screening ECG.

- Patients with the following laboratory values during screening:

- Serum creatinine > 1.5 x upper limit of normal (ULN) for age

- Total bilirubin >1.5 x ULN for age

- Alanine aminotransferase (ALT)/serum glutamic pyruvic transaminase (SGPT) > 3 x
ULN for age; aspartate aminotransferase (AST)/serum glutamic oxaloacetic
transaminase(SGOT) > 3 x ULN for age except in patients with tumor involvement of
the liver who must have AST/SGOT and ALT/SGPT ≤ 5 x ULN for age. For the purpose
of this study, the ULN for SGPT/ALT is 45 U/L.

- Patients who are currently receiving treatment with agents that are metabolized
predominantly through CYP3A4/5 and have a narrow therapeutic window and/or agents that
are known strong inducers or inhibitors CYP3A4/5 are prohibited. In particular,
enzyme-inducing antiepileptic drugs (EIAEDs).

- Other severe acute or chronic medical or psychiatric condition or laboratory
abnormality that may interfere with the interpretation of study results, and in the
judgment of the investigator would make the patient inappropriate for the study.