How was MIMRYLO™ (rusfertide) studied and what were the results?

MIMRYLO was found to help people require fewer phlebotomies, keep hematocrit (HCT) below 45%, and provide relief from fatigue

About the clinical study

How was MIMRYLO studied?

MIMRYLO was evaluated in a clinical study called VERIFY in 293 adults with polycythemia vera (PV) who needed frequent rescue phlebotomies before entering the study. During the 7-month main study period, people received either MIMRYLO or placebo, along with the PV treatment they were already receiving. Rescue phlebotomies could be given if needed. Researchers measured how many people avoided rescue phlebotomies while keeping their HCT controlled. Participants who completed the first 7 months then stayed on or switched to MIMRYLO for the next 5 months.

What were the goals of the VERIFY study?

The primary study goal

(Measured during Months 5 to 7)

What percentage of people taking MIMRYLO did not need rescue phlebotomies to maintain HCT control?

Additional study goals

(Measured during Months 0 to 7)

On average, did people taking MIMRYLO require fewer rescue phlebotomies overall?
Did MIMRYLO help people keep their HCT under control?
Did MIMRYLO provide relief from fatigue?

Results with MIMRYLO

Most people with polycythemia vera (PV) taking MIMRYLO did not require rescue phlebotomies to keep their HCT controlled*

77% of people taking MIMRYLO did not require rescue phlebotomies

Infographic showing 77% of people receiving MIMRYLO did not require therapeutic phlebotomies during the main study period, vs 33% receiving placebo — *Study participants were eligible for phlebotomy if their HCT level reached or exceeded 45% and increased by at least 3 percentage points from baseline, or if it reached or exceeded 48% at any time.

MIMRYLO reduced the need for rescue phlebotomies

People receiving MIMRYLO required over 3x fewer rescue phlebotomies

Infographic showing average therapeutic phlebotomies per patient: 0.5 with MIMRYLO vs 1.8 with placebo, Months 0 to 7 — During the study, participants continued their current PV treatment.

MIMRYLO helped the majority of people with PV keep their HCT under control

63% of people taking MIMRYLO kept HCT below 45%^†

Infographic showing 63% of people receiving MIMRYLO kept HCT below 45% compared to 14% receiving placebo — 63% of people receiving MIMRYLO kept their HCT controlled below 45%^†, compared with 14% receiving placebo between months 0–7

^†A single HCT fluctuation ≥45% was permitted.

MIMRYLO provided relief from fatigue

In the VERIFY study, people taking MIMRYLO reported reduced fatigue compared to placebo at Month 7

During the study, a validated survey was used to capture self-reported fatigue over time.^‡

^‡The survey used to measure fatigue was the PROMIS Fatigue Short Form 8a, with lower scores indicating improvement.

MIMRYLO helped reduce HCT levels in as little as 4 weeks

HCT rapidly decreased within 4 weeks

A reduction in HCT was seen within 4 weeks after switching to MIMRYLO.

All individuals in images are actor portrayals.

IMPORTANT SAFETY INFORMATION

Before using MIMRYLO, tell your healthcare provider about all of your medical conditions, including if you:

are pregnant or plan to become pregnant. MIMRYLO may harm your unborn baby.
Females who are able to become pregnant:

Your healthcare provider may do a pregnancy test before you start treatment with MIMRYLO.
Use effective birth control (contraception) during treatment and for at least 30 days after the final dose of MIMRYLO.
Tell your healthcare provider right away if you become pregnant or think that you may be pregnant during treatment with MIMRYLO.
Stop taking MIMRYLO if you become pregnant.

are breastfeeding or plan to breastfeed. It is not known if MIMRYLO passes into your breast milk. Do not breastfeed during treatment with MIMRYLO and for 30 days after the final dose of MIMRYLO. Talk to your healthcare provider about the best way to feed your baby during this time.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What are the possible side effects of MIMRYLO?

MIMRYLO may cause serious side effects, including:

New or worsening increased blood platelets (thrombocytosis). MIMRYLO may increase blood platelet counts. Your healthcare provider will do blood tests when you start and during treatment with MIMRYLO to check your platelet counts.

Injection site reactions. MIMRYLO may cause injection-site reactions such as redness, itching, pain, swelling, bruising, hardening at the injection site, and irritation. If you get an injection site reaction, apply ice packs, steroid cream, or take medicines as needed to treat injection site pain and swelling.

Your healthcare provider may change your dose or stop treatment with MIMRYLO if you have side effects.

The most common side effects of MIMRYLO are:

injection site reactions

low red blood cell count (anemia)

These are not all of the possible side effects of MIMRYLO. Call your doctor for medical advice about side effects. You may report side effects to Takeda at 1-844-662-8532 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.