Rocket Pharmaceuticals (RCKT) announced positive Phase 1 test results for a gene therapy in heart disease — prompting RCKT stock to surge early Wednesday.
The gene therapy aims to treat Danon Disease, a genetic disorder that leads to severe and frequently fatal cardiomyopathy. Cardiomyopathy is when the heart muscle must work incredibly hard to pump blood to the rest of the body.
Early testing shows the gene therapy was well tolerated and reduced several key biomarkers of the disease, Rocket said in a news release.
"Based on these early results, we believe that a low dose of (the drug called) RP-A501 has the potential to confer meaningful therapeutic benefit with an overall manageable safety profile," Chief Executive Gaurav Shah said in a written statement.
In early trading on the stock market today, RCKT stock flew 81.7% higher, near 58.20. That put RCKT stock at its highest point since mid-2015. Shares are also well above their 50-day and 200-day moving averages, according to MarketSmith.com.
Danon Disease is caused by deficient levels of a protein called LAMP2B. Rocket tested a low dose of its drug in three patients, and then a high dose in two patients. Two patients who received a low dose of the gene therapy a year ago showed LAMP2B levels more than 50% above normal levels.
All three low-dose patients showed improvements in a marker of heart failure and improvement in a hallmark of Danon Disease. Two showed improvements on a marker used to diagnose heart attacks. Further, those two had improved cardiac output.
"These early results suggest a path to a potentially transformative option for Danon Disease, and possibly the first viable gene therapy approach for cardiac diseases," CEO Shah said.
Gene Therapy Safe In Low-Dose Group
The gene therapy also proved to be safe in three patients who received low dose. There were no unexpected side effects related to the gene therapy. All three low-dose patients experienced elevated enzymes. The elevated enzymes responded to corticosteroids and other immunosuppressive drugs. Patients' enzyme levels normalized one to two months after treatment.
One patient who received the highest dose experienced abnormally low levels of platelets and acute kidney injury. The patient returned to normal within three weeks. But Rocket said the event was related to the gene therapy.
Also, on Wednesday, Rocket announced an offering of RCKT stock. The biotech company is selling $175 million shares of its common stock. Underwriters will have a 30-day option to buy up to an additional 15% of the shares of RCKT stock sold in the offering.
Rocket said it will use the proceeds to fund development of its gene therapies.
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