Shares in Rocket Pharmaceuticals have been living up to their name, shooting up following encouraging early-stage clinical trial results from a gene therapy for a serious inherited rare heart disease.<\/p>\n
Results came from a phase 1 trial of RP-A501 for treatment of Danon Disease and sent shares up 75% on the Nasdaq to more than $56, a five-year high.<\/p>\n
The surging stock price indicates the markets confidence in gene therapy products after the successful launch of products such as Roche\/Spark Therapeutics Luxturna, a gene therapy for a rare inherited eye disease.<\/p>\n
Danon Disease is a rare X-linked disorder caused by genetic mutations in the LAMP2 gene and the therapy works by instructing the body to express a healthy copy of the LAMP2B protein in order to correct the condition.<\/p>\n
The disease that affects boys and men more severely causes accumulation of autophagosomes tiny structures that cause cells internal structures to break down in the heart muscle and other tissues.<\/p>\n
Together with a build-up of glycogen this can lead to severe and frequently fatal degradation of the heart muscle.<\/p>\n
RP-A501 could be the first gene therapy for the disease and the early data showed a positive increase in cardiac protein expression.<\/p>\n
As of November, three patients have been treated with a low dose of the therapy and two have been treated with a high dose.<\/p>\n
An early trial readout showed two patients with LAMP2B expression that was 50% more than normal, measured nine and 12 months after treatment.<\/p>\n
A 15%-20% increase could lead to clinically meaningful improvements in cardiac function and the trial reported a 50% decrease in a key biomarker of heart failure.<\/p>\n
There was also a reduction in myocardial cell disarray and a visible reduction in autophagic vacuoles, a hallmark of the disease.<\/p>\n
The company also noted stabilisation of three other measures a heart failure biomarker known as BNP, plus levels of transaminases and creatine kinase that also indicate skeletal and heart muscle damage.<\/p>\n
However one patient who received the highest dose and had a degree of immunity to the adeno-associated virus used in the therapy had an immune reaction classified as a serious adverse event.<\/p>\n
Rocket said the event was likely due to complement activation, resulting in reversible thrombocytopenia and acute kidney injury requiring a short round of haemodialysis.<\/p>\n
The patient returned to baseline within three weeks and regained normal kidney function.<\/p>\n
DrBarry Greenberg, director of the Advanced Heart Failure Treatment Program atUC San Diego Health, Professor of Medicine atUC San Diego School of Medicine, and the principal investigator said: Children with Danon Disease live with a heavy disease burden. Young boys are often severely afflicted.<\/p>\n
They show evidence of early onset skeletal muscle weakness and heart disease that can progress rapidly to end-stage with death occurring on the average before age 20. A heart transplant can be performed but is not curative and is associated with its own significant problems.<\/p>\n
The results-to-date for this first investigational gene therapy for monogenic heart failure show the potential for direct clinical benefit without emergence of unanticipated side effects of therapy.<\/p>\n
The company has also begun a stock offering of $175 million in shares to fund further development following the results.<\/p>\n<\/p>\n<\/p>\n<\/p>\n
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Rocket Pharmaceuticals in orbit after gene therapy read-out - - pharmaphorum<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" Shares in Rocket Pharmaceuticals have been living up to their name, shooting up following encouraging early-stage clinical trial results from a gene therapy for a serious inherited rare heart disease. Results came from a phase 1 trial of RP-A501 for treatment of Danon Disease and sent shares up 75% on the Nasdaq to more than $56, a five-year high. Continue reading