'This data set cumulatively builds on previously presented scientific evidence demonstrating rusfertide's potential as the first-in-class, non-cytoreductive treatment option for polycythemia vera, a disease that currently has limited therapeutic options and a demonstrated significant unmet medical need,' said
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
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 https://clinicaltrials.gov/ct2/show/NCT04057040 and http://ptg300pvstudy.com/.
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About
Protagonist's pipeline includes rusfertide (PTG-300), an investigational, injectable hepcidin mimetic currently in a Phase 2 proof-of-concept clinical trial for polycythemia vera (PV), a Phase 2 study in PV subjects with high hematocrit levels, and a Phase 2 study for hereditary hemochromatosis. Based on the feedback provided by the FDA and EU regulatory authorities, the Company plans to initiate a single, global Phase 3 randomized, placebo-controlled trial evaluating the efficacy and safety of a once weekly, subcutaneously self-administered dose of rusfertide.
The Company is also evaluating an orally delivered, gut-restricted alpha-4-beta-7 integrin specific antagonist peptide (PN-943) currently in a Phase 2 study in adults with moderate to severe active ulcerative colitis (UC). Company is targeting ulcerative colitis as the initial indication.
The Company has a worldwide license and collaboration agreement with
Protagonist is headquartered in
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements regarding our intentions or current expectations concerning, among other things, potential benefits of rusfertide for the treatment of PV and our plans conduct a Phase 3 trial evaluating rusfertide for PV. In some cases, you can identify these statements by forward-looking words such as 'anticipate,' 'believe,' 'may,' 'will,' 'expect,' or the negative or plural of these words or similar expressions. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, our ability to develop and commercialize our product candidates, our ability to earn milestone payments under our collaboration agreements, the impact of the current COVID-19 pandemic on our discovery and development efforts, our ability to use and expand our programs to build a pipeline of product candidates, our ability to obtain and maintain regulatory approval of our product candidates, our ability to operate in a competitive industry and compete successfully against competitors that have greater resources than we do, and our ability to obtain and adequately protect intellectual property rights for our product candidates. Additional information concerning these and other risk factors affecting our business can be found in our periodic filings with the
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