Expanded Access for ABI-009 in Patients With Advanced PEComa and Patients With a Malignancy With Relevant Genetic Mutations or mTOR Pathway Activation

Inclusion Criteria:

A patient will be eligible for inclusion in this expanded access program only if all of the
following criteria are met:

   1. a) Diagnosis of advanced (locally advanced and inoperable or metastatic) malignant
   PEComa confirmed by pathology and immunohistochemistry, whether or not previously
   exposed to an mTOR inhibitor, or b) diagnosis of any other malignancy with activation
   in any mTOR pathway component as identified by immunohistochemistry or an identified
   relevant rare genetic mutation in mTOR pathway genes, including but not limited to
   TSC1, TSC2, PIK3CA, PTEN, for which there are no FDA-approved treatments or no other
   comparable or satisfactory alternative therapies available whether or not they been
   previously exposed to a mTOR inhibitor.

   2. 18 years or older, with Eastern Cooperative Oncology Group (ECOG) performance status
   0, 1, or 2.

   3. Acceptable blood chemistry levels at screening (obtained ≤14 days prior to enrollment
   (local laboratory) including:

      1. total bilirubin ≤1.5 x upper limit of normal (ULN)

      2. AST ≤2.5 x ULN (≤5 x ULN if attributable to liver metastases)

      3. serum creatinine ≤1.5 x ULN

   4. Adequate biological parameters as demonstrated by the following blood counts at
   screening (obtained ≤14 days prior to enrollment, local laboratory):

      1. Absolute neutrophil count (ANC) ≥1.5 × 109/L;

      2. Platelet count ≥100,000/mm3 (100 × 109/L);

      3. Hemoglobin ≥8 g/dL.

   5. Fasting serum triglyceride <300 mg/dL; fasting serum total cholesterol <350 mg/dL.

   6. Male or non-pregnant and non-breast feeding female:

      - Females of child-bearing potential must agree to use effective contraception
      without interruption from 28 days prior to starting IP and while on
      investigational medication and have a negative serum pregnancy test (β -hCG)
      result at screening and agree to ongoing pregnancy testing during the course of
      the expanded access protocol, and after the end of treatment. A second form of
      birth control is required even if she has had a tubal ligation.

      - Male patients must practice abstinence or agree to use a condom during sexual
      contact with a pregnant female or a female of childbearing potential while
      participating in the expanded access protocol. A second form of birth control is
      required even if he has undergone a successful vasectomy.

   7. Ability to understand and sign informed consent.

Exclusion Criteria:

A patient will not be eligible for inclusion in this protocol if any of the following
criteria apply:

   1. Uncontrolled serious medical or psychiatric illness. Patients with a "currently
   active" second malignancy other than non-melanoma skin cancers, carcinoma in situ of
   the cervix, resected incidental prostate cancer (staged pT2 with Gleason Score ≤ 6 and
   postoperative PSA <0.5 ng/mL), or other adequately treated carcinoma-in-situ are
   ineligible. Patients are not considered to have a "currently active" malignancy if
   they have completed therapy and are free of disease for ≥1 year.

   2. In the treating physician's opinion, the potential risks outweigh the potential
   benefits of therapy with ABI-009.

   3. Prior exposure to ABI-009.