Updated Phase 2 Data Supporting Long-Term Efficacy of Rusfertide in Polycythemia Vera

[Manouche]

Summary of Results:

Therapeutic phlebotomies were essentially eliminated and a target hematocrit of less than 45 percent was maintained for the vast majority of patients treated with rusfertide

Rusfertide demonstrated long-term control of hematocrit, as well as durability of effect based on patients treated up to 18 months

Rusfertide treatment also led to reversal of iron deficiency as evidenced by increasing serum ferritin, mean corpuscular volume (MCV) and mean corpuscular hemoglobin (MCH) values

Rusfertide demonstrated similar efficacy in all patients, independent of risk group or prior and concurrent therapy

Benefits were observed in patient reported outcomes, as shown by improvement in PGI-C and reduction in MPN-SAF Symptom Scores, attributed largely to the sub-scores of fatigue and concentration, consistent with improvement in iron deficiency

The current data indicate that rusfertide is well tolerated. The most common adverse events observed were transient injection site reactions

"These results provide additional evidence that rusfertide may have a clinical benefit for patients with polycythemia vera," said Ronald Hoffman, MD, the Albert A. and Vera G. List Professor of Medicine and Director of the Myeloproliferative Disorders Research Program at Mount Sinai in New York. "The need for a new non-cytoreductive therapeutic option in PV is urgent. I look forward to the next steps in rusfertide's development for this disease, as it may alleviate the burden of phlebotomy for patients who cannot control their hematocrit levels through currently existing treatment options and need an alternative therapeutic approach."

The ongoing Phase 2 rusfertide study in PV is designed to monitor the safety profile and obtain evidence of efficacy in patients requiring frequent phlebotomies (at least three phlebotomies in the prior six months). The study design consists of three stages: a 16-week open-label stage with weekly subcutaneous doses from 10 to 80 mg and dose escalation or reduction, as necessary every four weeks; a 12-week maintenance period at doses that effect desired hematocrit levels; and then a randomized and blinded withdrawal stage (1:1 treatment vs. placebo) for up to 12 weeks. The study also has an open-label extension for up to three years to monitor long-term safety and other effects. The primary endpoint is the control of hematocrit below 45 percent during the blinded randomized withdrawal period. Other endpoints of this clinical proof-of-concept study include measurement of blood parameters (hematocrit and hemoglobin levels), reductions or delay in phlebotomy requirements, and symptoms related to quality of life.

Additional information is available at clinicaltrials.gov/ct2/show... and ptg300pvstudy.com/

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[JohnSC]

Great news. The more options we have the better. Thanks for posting this info.

[Meatloaf9]

Thank you for posting. We need all the options we can get.

[hunter5582]

Rusterfide (PTG-300) appears to be a very promising treatment option for PV. I was disappointed to not get into the clinical trial. The promise of being able to control erythrocytosis without having to tolerate iron deficiency is huge. Thanks for posting this.

[Manouche in reply to hunter5582]

Hi Hunter, your initial response to Pegasys is absolutely perfect. I don’t think you should have any regret about not joining the phase II rusterfide trial.

[hunter5582 in reply to Manouche]

I am going to be very interested to see the results of the PEG-IFN - Rusterfide combination treatment. It seems intuitively that it could be very effective. Time will tell.

[Manouche in reply to hunter5582]

Rusterfide alone will most certainly reduce dramatically many thrombo-embolic events and some other PV complications. Therefore, we could reasonably expect a very significant increase in life expectancy with both drugs.

[Bluetop]

Thanks for posting. Great to hear another promising option looming.