An Expanded Access Program of Ruxolitinib for the Treatment of Graft-Versus-Host Disease Following Allogeneic Hematopoietic Stem Cell Transplant

Key Inclusion Criteria:

- Male or female, 12 years of age or older.

- Have undergone an allo-HSCT from any donor source using bone marrow, peripheral blood
stem cells, or cord blood for hematologic malignancies. Recipients of nonmyeloablative
and myeloablative conditioning regimens are eligible.

- Clinically suspected all grades acute or chronic GVHD as per Minnesota-Center for
International Blood and Marrow Transplant Research (MN-CIBMTR) criteria, that is
refractory or intolerant to corticosteroids, occurring after allo-HSCT with any
conditioning regimen and any anti-GVHD prophylactic program. Clinical suspicion of
GVHD by the treating physician is also sufficient.

- Evidence of myeloid engraftment (eg, absolute neutrophil count ≥ 1.0 × 10^9/L for 3
consecutive days if ablative therapy was previously used). Use of growth factor
supplementation is allowed.

- Evidence of platelet engraftment (ie, platelets ≥ 20 × 10^9/L).

- Eastern Cooperative Oncology Group (ECOG) performance status 0 to 3.

- Be willing to avoid pregnancy or fathering children based on 1 of the following
criteria:

- Women of non-childbearing potential (ie, surgically sterile with a hysterectomy
and/or bilateral oophorectomy OR ≥ 12 months of amenorrhea).

- Woman of childbearing potential who has a negative serum pregnancy test at
screening and who agrees to take appropriate precautions to avoid pregnancy (with
at least 99% certainty) from screening through safety follow-up. Permitted
methods that are at least 99% effective in preventing pregnancy should be
communicated to the patient and their understanding confirmed.

- Man who agrees to take appropriate precautions to avoid fathering children (with
at least 99% certainty) from screening through safety follow-up. Permitted
methods that are at least 99% effective in preventing pregnancy should be
communicated to the patient and their understanding confirmed.

- Able to provide written informed consent and/or assent from the patient, parent, or
guardian.

Key Exclusion Criteria:

- Eligible for an existing and actively enrolling Incyte sponsored clinical trial for
ruxolitinib for the treatment of GVHD.

- Patients or legal guardians unable to review and sign informed consent form.

- Females who are pregnant or breastfeeding, and males and females who cannot comply
with requirements to avoid fathering a child or becoming pregnant.

- Patients with inadequate liver function (alanine aminotransferase above 4 × upper
limit of normal (ULN) or direct bilirubin 4 × ULN and the laboratory abnormalities are
considered to be due to underlying liver dysfunction) unless attributed to GVHD.

- Patients with end stage renal function (creatinine clearance (CrCl) < 15 mL/min or
glomerular filtration rate < 15 mL/min), regardless of whether hemodialysis is
required.

- Any underlying or current medical or psychiatric condition that, in the opinion of the
treating physician, would place the patient at an unacceptable risk if he or she were
to participate in the program.

- Previous allergic reactions to Janus kinase (JAK) inhibitors or excipients.

- Patients who are currently taking any anticancer therapy (eg, chemotherapy, radiation
therapy, surgery, immunotherapy, biologic therapy, hormonal therapy, or tumor
embolization).

- Patients taking any secondary GVHD therapy due to insufficient response/progression on
program treatment, including but not limited to ibrutinib, filgotinib, and other
off-label medications.

- Concomitant use of any JAK inhibitor.

- Initiating therapy with any investigational medication.

- Presence of an active uncontrolled infection. An active uncontrolled infection is
defined as hemodynamic instability attributable to sepsis or new symptoms, worsening
physical signs, or radiographic findings attributable to infection. Persisting fever
without signs or symptoms will not be interpreted as an active uncontrolled infection.

- Known HIV infection.

- Active hepatitis B virus (HBV) or hepatitis C virus infection that requires treatment
or at risk for HBV reactivation. At risk for HBV reactivation is defined as hepatitis
B surface antigen positive or anti-hepatitis B core antibody positive. Previous test
results obtained as part of standard of care before allo-HSCT that confirm a patient
is immune and not at risk for reactivation (ie, hepatitis B surface antigen negative,
surface antibody positive) may be used for purposes of eligibility.