If there is one adverse event from an investigational drug that you don't want in DMD - its myocarditis or inflammation of the heart muscle. The article Pfizer adds to gene therapy woes published 29 September 2021, reports that the Phase 3 trial being run by Pfizer has hit another roadblock: three cases of severe muscle weakness including two cases of myocarditis.
Myocarditis can lead to cardiomyopathy a disease of the heart muscle. Heart failure is a classic complication of DMD. By 6 years of age 25% of patients exhibit symptoms, increasing to 59% by age 10 and 100% in adulthood. I don't know if you noticed but I mentioned that myocarditis is an inflammation of the heart muscle. Just for emphasis, its worth restating the word inflammation.
Does gene therapy producing micro-dystrophin elicit inflammation? We have previously noted that the penny has now dropped that the body hasn't seen this micro-dystrophin before and the cell mediated immune response is to attack foreign bodies. Gene therapy may produce newly formed better performing muscle but its a no from the body, it will become a no from the DMD community and turn into a big no from life science investors. Regulators may and are continuing to work with Pharma and Biotech with gene therapy drug candidates in DMD because it holds the promise of a future cure. Unfortunately the future could be a long way off.
Meanwhile we are continuing to explore the effect of reducing Thrombospondin-1 (TSP-1) by 49%. Drawing from the article Thrombospondin 1 in Metabolic Diseases published in March 2021, an increase in cleaved TSP-1 is detected in adults with dilated cardiomyopathy. TSP-1 is implicated in cardiac fibrosis. Now there is an exciting opportunity for therapeutic intervention.
ANP Price at posting:
19.5¢ Sentiment: Buy Disclosure: Held
