Divesiran in Polycythemia Vera: What the Phase 2 SANRECO Results Tell Us
Our webinar with Alberto Martínez from Silence Therapeutics is now available. A conversation about the SANRECO results and the long journey a medicine takes before it can potentially reach patients.
How does a molecule move from a laboratory to potentially becoming a treatment for patients? This was one of the questions explored during our latest Global MPN webinar, where Alberto Martínez, Senior Director of Clinical Science at Silence Therapeutics, presented the Phase 2 results of SANRECO, a clinical trial investigating divesiran in people living with polycythemia vera (PV).
The session provided an opportunity not only to understand the study results, but also to explore something patients do not often get to see: how a medicine is developed, what each stage of a clinical trial means and why the process can take many years.
What is divesiran?
Divesiran is an investigational small interfering RNA, or siRNA, therapy.
In simple terms, siRNA technology can reduce the production of specific proteins by acting on the genetic instructions used to make them.
Divesiran is designed to target TMPRSS6, a pathway involved in iron regulation and red blood cell production. It is being investigated as a potential way to control hematocrit and reduce the need for phlebotomies in people living with PV. It is not currently an approved treatment and remains in clinical development.
SANRECO: 48 patients and one primary question
The Phase 2 part of SANRECO was an international, randomized, double-blind, placebo-controlled study involving 48 phlebotomy-dependent patients with PV.
Its primary goal was to determine how many patients could maintain hematocrit below 45% without requiring phlebotomy between weeks 18 and 36.
According to the results announced by Silence Therapeutics, a clinical response was achieved by: 88% of patients receiving divesiran, compared with 19% receiving placebo.
The mean number of phlebotomies during the 36-week period was also 0.2 per patient with divesiran compared with 2.1 with placebo. Improvements were additionally reported in iron markers and patient-reported symptom outcomes.
These findings are encouraging, but they come from a Phase 2 trial. Larger studies will be required to confirm efficacy and safety before any potential regulatory approval can be considered.
One injection every three months?
One of the topics that generated particular interest during the webinar was dosing frequency.
SANRECO evaluated divesiran as a subcutaneous injection every six weeks or every twelve weeks. Both schedules showed activity on the primary endpoint, and Silence Therapeutics has stated that its planned Phase 3 program will evaluate the once-every-12-weeks regimen.
If future studies continue to show favorable results, infrequent dosing could therefore become one of the characteristics evaluated as part of this investigational approach.
The questions patients wanted to ask
The final part of the webinar focused on questions that naturally arise when patients hear about new research.
Peter Löffelhardt and Alberto Martínez discussed:
how divesiran is administered;
why the same dosing approach can be studied across different patients;
how long treatment might need to continue;
long-term follow-up;
what may happen to iron levels when phlebotomy requirements decrease;
and what is currently known about other aspects of PV, including the spleen.
The discussion also highlighted the essential role that patients play in developing new medicines. Clinical research cannot progress without people willing to participate in clinical trials.
The next step: Phase 3
According to Silence Therapeutics, the next stage of development will be a Phase 3 trial, anticipated to begin during the first half of 2027. Phase 3 will need to evaluate divesiran in a larger patient population and provide more robust evidence regarding its efficacy and safety.
For now, divesiran remains an investigational therapy. The webinar provides a useful reminder that every positive clinical result matters, but also that developing a medicine requires time, evidence and several stages before it may become available to patients.
This content is provided for informational and educational purposes only. Divesiran is an investigational medicine and is not currently approved for the treatment of polycythemia vera. This information does not replace medical advice from your healthcare team and should not be interpreted as a treatment recommendation.
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