Phase 2 Study of Oral Epetraborole in Adults With Polycythemia Vera

NCT07891533 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: EBO-PV-201
• Study Type: interventional
• Target: 75
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Phase 2 trial to evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of oral epetraborole tablets for the treatment of adults with phlebotomy-dependent polycythemia vera (PV). The trial is designed to confirm the safety profile of oral epetraborole in the PV patient population and to identify 1 or more oral epetraborole doses for the treatment of phlebotomy-dependent PV. The study consists of an open-label Sentinel Cohort, an open-label Dose Selection Cohort (Part 1), followed by a double-blind, randomized, Placebo-Controlled Cohort (Part 2), and an optional Open-Label Extension (OLE) Cohort (Part 3)

Conditions

Polycythemia Vera (SNOMED CT: 109992005)

Keywords

Hematologic disease; Bone marrow disease; Myeloproliferative disorder; Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Hemic and Lymphatic Diseases; Polycythemia Vera; Epetraborole; Benzoxaborole; Boron; Phase 2[

Outcome Measures

Primary Outcomes (1)

• Part 2: Proportion of Participants per Treatment Group Maintaining Clinical Success defined as hematocrit control, not undergoing therapeutic phlebotomy and completing 12 weeks of treatment in Part 2

  Week 29 - Week 40

Study Arms (5)

Sentinel Cohort

EXPERIMENTAL

Open Label Fixed Dose Epetraborole

Drug: Epetraborole

Part 1: Dose Selection Cohort

EXPERIMENTAL

Open Label Epetraborole Dose Selection

Drug: Epetraborole

Part 2: Epetraborole (Double Blind)

EXPERIMENTAL

Fixed Dose Epetraborole

Drug: Epetraborole

Part 2: Placebo (Double Blind Randomized Withdrawal)

PLACEBO COMPARATOR

Placebo Control

Drug: Placebo

Part 3: Open Label Extension Cohort (Optional)

EXPERIMENTAL

Open Label Epetraborole

Drug: Epetraborole

Interventions

Epetraborole DRUG

Oral tablets

Part 1: Dose Selection Cohort; Part 2: Epetraborole (Double Blind); Part 3: Open Label Extension Cohort (Optional); Sentinel Cohort

Placebo DRUG

Oral tablets

Part 2: Placebo (Double Blind Randomized Withdrawal)

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Willing and able to read and provide written informed consent prior to the performance of any trial-specific procedures and willing to follow the protocol requirements
• Aged 18 to 75 years (inclusive) at Screening
• A confirmed diagnosis of PV according to ICC (Thiele, 2023), including Hgb/Hct level, EPO level, and JAK2 mutation status assessed by analysis of peripheral blood sampling
• Documented positive JAK2 V617F (exon 14) or JAK2 exon 12 mutation
• Hct \<45% at Screening
• Has records of all phlebotomies performed for at least 28 weeks (preferably up to 52 weeks) prior to Day 1
• Phlebotomy-dependent, defined as at least 3 phlebotomies during the 28 weeks prior to Day 1, including a phlebotomy within 8 weeks prior to Screening
• Either receiving OR not receiving cytoreductive therapy for PV, as follows:
• Participants who are not currently receiving cytoreductive therapy must have either never received it or been discontinued from it for at least 24 weeks prior to Day 1 and any AEs due to cytoreductive therapy have either recovered or are ≤ Grade 1 prior to Day 1
• Participants currently receiving cytoreductive therapy with HU, interferon, or busulfan must have received such therapy for at least 24 weeks prior to Day 1 and have been on a stable or decreasing dose for at least 8 weeks prior to Day 1, and with no planned change in dose during the trial
• ECOG score of 0, 1, or 2
• If FOCP
• Must not be currently breastfeeding
• If sexually active with a male partner: Must be either sexually abstinent or agree to use at least 2 medically accepted, effective methods of birth control from Screening through at least 1 week after the last dose of trial intervention
• Must have a negative urine pregnancy test at Screening and on Day 1; the Day 1 urine test will be confirmed by a serum pregnancy test.

You may not qualify if:

• History of clinically significant arterial or venous thrombosis (eg, deep vein thrombosis or splenic vein thrombosis) within 24 weeks prior to Day 1
• History of major bleeding events and/or a requirement for blood transfusion therapy due to bleeding in the 6 months prior to Day 1
• Meets the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (Tefferi, 2023)
• Platelets \>1000 × 109/L, WBC count \>25 × 109/L; or presence of any blast cells in peripheral blood at Screening
• Diagnosis of secondary polycythemia
• Prior, concomitant, or planned therapy with ruxolitinib
• Received any investigational drug within 6 weeks prior to Day 1 or 5 half-lives (whichever is longer) prior to Day 1 or has not recovered from adverse effects of prior administration of any investigational agent
• Prior exposure to epetraborole; drug intolerance or hypersensitivity to any compound from the same class as epetraborole
• History of prior drug-induced pneumonitis
• Severe hepatic impairment at Screening as evidenced by clinical signs of cirrhosis or end-stage hepatic disease
• Liver disease or liver injury at Screening as evidenced by:
• ALT or AST \>3 × ULN
• Total bilirubin \>2 × ULN
• eGFR \<30 mL/min/1.73 m2 at Screening
• Current skin malignancy or history of clinically significant skin cancer within 5 years prior to Day 1

Sponsors & Collaborators

• AN2 Therapeutics, Inc: lead

Study Sites (1)

501001

Perth, Australia

Location

MeSH Terms

Conditions

Polycythemia Vera; Hematologic Diseases; Bone Marrow Diseases; Myeloproliferative Disorders; Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Hemic and Lymphatic Diseases

Central Study Contacts

Cameron Trenor

CONTACT

650-331-9090; trials@an2therapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 17, 2026
• First Posted: October 23, 2026
• Study Start (Estimated): November 1, 2026
• Primary Completion (Estimated): April 1, 2029
• Study Completion (Estimated): June 1, 2030
• Last Updated: October 23, 2026

Record last verified: 2026-10

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)