Rilzabrutinib study highlights cognitive impairment in patients with chronic immune thrombocytopenia

[Rilzabrutinib image courtesy of PubChem]

A Phase 1/2 study (NCT03395210) investigating Sanofi (Nasdaq:SNY) Bruton tyrosine kinase inhibitor rilzabrutinib in immune thrombocytopenia (ITP) has also shed light on the cognitive impairment involved in the disease.

Patients with ITP, an autoimmune disease that involves increased platelet destruction, have often complained of fatigue, problems with cognition and other symptoms that physicians have dismissed, according to Dr. David Kuter, the director of clinical hematology at Massachusetts General Hospital, professor of medicine at Harvard Medical School, lead author of the NCT03395210 study. “What’s relevant about this study is that it is probably the first time that we’ve done real testing on patients to demonstrate there is a defect in ITP patients in terms of their cognition,” Kuter said.

The topic of cognitive impairment in ITP has …

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Sanofi announces publication of early study of rilzabrutinib for immune thrombocytopenia

Sanofi (Nasdaq:SNY) has announced that the New England Journal of Medicine (NEJM) has published positive data from the Phase 1/2 dose-finding study testing the safety, pharmacokinetics and clinical activity of rilzabrutinib for immune thrombocytopenia.

Immune thrombocytopenia is associated with bruising and bleeding. Fewer than 200,000 Americans are diagnosed with the condition each year.

Rilzabrutinib is an experimental oral Bruton’s tyrosine kinase (BTK) inhibitor. The drug candidate has been the focus of about 20 clinical trials to date, with indications ranging from asthma to urticaria.

The study, published in NEJM, focused on adults with heavily pre-treated immune thrombocytopenia (ITP), concluded that rilzabrutinib therapy led to a rapid and durable increase in platelet count. Rilzabrutinib also appeared to have a good safety profile.

The drug candidate may increase platelet counts in patients with immune thrombocytopenia. As the NEJM articl…

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