RES & RES II

PV patients resistant to or intolerant of HU

Phlebotomy requirement

Splenomegaly

No prior JAK inhibitor

• RESPONSE

• RESPONSE II

Patients with PV(N = 149)

Resistant to or intolerant of HU

Phlebotomy requirement

No splenomegaly

ECOG PS score ≤ 2

• RESPONSE

• RESPONSE II

Crossover to Ruxolitinib Was Allowed on or After Week 28

• RESPONSE

• RESPONSE II

Primary endpoint

HCT control and spleen response at Week 32 defined as

HCT control: absence of phlebotomy eligibility from Week 8 to Week 32, with ≥ 1 phlebotomy before Week 8

Spleen response: ≥ 35% reduction in spleen volume by MRI scan at Week 32

• RESPONSE

• RESPONSE II

Primary endpoint

HCT control up to Week 28 defined as

HCT < 45% at Week 16 and maintained until Week 28

No phlebotomy from Week 4 to Week 28 with no more than one phlebotomy occurring post-randomization and prior to the Week 4 visit

• RESPONSE

• RESPONSE II

Durable HCT control at both Weeks 28 and 80 was achieved in 47% of ruxolitinib-treated patients (95% CI 35.6–59.3) and 3% of BAT-treated patients (95% CI 0.3–9.3)Patients who achieved response at Week 28 with ruxolitinib had a 78% probability of maintaining the response to Week 80

• RESPONSE

• RESPONSE II

Efficacy result:

In patients with PV who were resistant to or intolerant of HU, ruxolitinib was superior to BAT in

Controlling HCT without phlebotomy

Inducing complete hematologic remission

Reducing spleen volume

Improving symptoms

• RESPONSE

• RESPONSE II

Key inclusion criteria

Confirmed PV diagnosis

Resistant to or intolerant of HU

No palpable spleen

HCT level of 40–45% within 14 days of BL, and ≥ 2 phlebotomies, ≥ 4 weeks apart, within 24 weeks prior to screening or HCT > 45% with ≥ 1 phlebotomy within the 16 weeks prior to screening after undergoing a HCT control period

• RESPONSE

• RESPONSE II

Key inclusion criteria:

Confirmed PV diagnosis

Resistant to or intolerant of HU

Phlebotomy requirement

≥ 2 phlebotomies, ≥ 4 weeks apart, within 24 weeks prior to screening; and

≥ 1 phlebotomy within 16 weeksof screening, if patient has a HCT > 45% at screening

Splenomegaly ≥ 450 cm3 as measuredby MRI or CT scan

• RESPONSE

• RESPONSE II

86% of the Patients Receiving BAT Switched to Ruxolitinib

• RESPONSE

• RESPONSE II