A Phase 3 Study of Rusfertide (PTG-300) in Patients with Polycythemia Vera

Prior treatment

• 3 - Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 2 months prior to randomization.
• 4 - Active or chronic bleeding within 2 months prior to randomization.
• 7 - Received busulfan, pipobroman or 32Phosphorus within 7 months prior to screening.

Other illness

• 5 - History of invasive malignancies within the last 5 years, except a) localized cured cancer (e.g. prostate cancer and cervical cancer). b) localized cured in situ or stage 1 squamous cell carcinoma of the skin, basal cell carcinoma of the skin, or in situ melanoma of the skin.
• 6 - Subjects with in situ or stage 1 squamous cell carcinoma of the skin, in situ or stage 1 basal cell carcinoma of the skin, or in situ melanoma of the skin identified during screening unless the cancer is adequately treated before randomization.

Lab values

• 1 - Clinically meaningful laboratory abnormalities at Screening

Other

• 2 - Subjects who require phlebotomy at hematocrit levels lower than 45%

• 1 - Male and female subjects aged 18 (or the minimum country specific age of consent if >18) years or older.
• 2 - Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of polycythemia vera
• 3 - Phlebotomy requiring defined as ALL of the following: a. At least 3 phlebotomies due to inadequate hematocrit control in 28 weeks before randomization or at least 5 phlebotomies due to inadequate hematocrit control in 1 year before randomization, and b. Last phlebotomy due to inadequate hematocrit control within 3 months before randomization, and c. No phlebotomy within 6 days prior to randomization (do not include day of phlebotomy and day of randomization in the 6-day count). Note: Phlebotomies performed within an 8-day period will be counted as a single phlebotomy
• 4 - CBC values immediately prior to randomization: a. Hematocrit <45%, b. WBC 4000/µL to 20,000/µL (inclusive) and c. Platelets 100,000/µL to 1,000,000/µL (inclusive).
• 5 - Subjects receiving cytoreductive therapy at randomization must be on a stable PV therapy regimen
• 6 - Subjects treated with phlebotomy alone at randomization must have stopped cytoreductive therapy 2 to 6 months before screening.