![]() This patient had more advanced disease than the four other adult/older adolescent patients who received treatment in the low and high dose cohorts, as evidenced by diminished baseline left ventricle ejection fraction (35%) on echocardiogram and markedly elevated left ventricle filling pressure prior to treatment. ![]() No in vivo gene therapy has ever been approved, but the opportunity to deliver a gene therapy directly into the patient via an injection is thought to be significantly more convenient, and safe, than the ex-vivo approach, which involves a patient undergoing a preconditioning regime similar to chemotherapy to allow cells to be harvested from their body, taken to a lab and engineered, and then reintroduced into the patient.ĭespite the risks of the preconditioning, and dangers of a patient's immune system rejecting the engineered cells, leading to potentially fatal complications such as graft vs host disease, several ex-vivo therapies have been approved targeting hematological cancers, but the value the market attaches to an in vivo breakthrough can be evidenced not only by Rocket's share price gains in 2020, but also by the gains made by Intellia Therapeutics ( NTLA) stock after the company became the first to demonstrate a positive therapeutic effect for a in vivo therapy, in 2021, when its candidate NTLA-2001 had reduced TTR Serum levels in six patients with the disease Transthyretin (ATTR) Amyloidosis, Intellia's market cap briefly rose >$10bn.Īs covered in my previous note on Rocket, the company's stock price tanked after a patient in the Phase 1 study receiving high-dose RP-A501 suffered progressive heart failure and was forced to undergo a heart transplant. Gaurav Shah, M.D., Chief Executive Officer and President of Rocket commented that "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." RP-A501 demonstrated consistent stabilization or improvements across all of these clinical measures as of the data cutoff. Cardiac dysfunction is often rapidly progressive and severe, with concomitant reductions in cardiac output. Males with Danon Disease typically have elevated BNP, transaminases and creatine kinase as a result of skeletal and heart muscle damage. The results revealed that "Cardiac LAMP2B protein expression by immunohistochemical staining was greater than 50% of normal LAMP2B in two patients with follow-up data of up to one year," and that: The study actually began in September 2020, and when Rocket released data in December 2020, the results wowed the market - Rocket's stock price leapt from ~$32, to $60 overnight. Rocket has been conducting a Phase 1 study of RP-A501 in 7 patients. ![]() Preclinical models of DD have demonstrated that AAV-mediated transduction of the heart results in reconstitution of LAMP2B expression and improvement in cardiac function. When I last covered Rocket Pharmaceuticals in a deep dive note for Seeking Alpha back in February, I gave the gene therapy specialist a "Buy" rating based primarily on the promise of the company's exciting in vivo adeno-associated virus (“AAV”) program for Danon disease.ĭanon Disease ("DD") is "a multi-organ lysosomal-associated disorder leading to early death due to heart failure" with an estimated patient prevalence of 15k - 30k in the US and EU, according to Rocket's Q223 quarterly report, which goes on to state:ĭD is an X-linked dominant, monogenic rare inherited disorder characterized by progressive cardiomyopathy which is almost universally fatal in males even in settings where cardiac transplantation is available.ĭD predominantly affects males early in life and is characterized by absence of LAMP2B expression in the heart and other tissues. In this note I cover yesterday's highly positive developments in detail, and provide some thoughts around the prospects for further share price growth Investment Overview - The Value of In Vivo Gene Therapy, Rocket's Initial Success, Followed By Clinical Hold The market opportunity may be restricted but the historic opportunity in front of the company is arguably being undervalued by the market, as I suggested back in February. Rocket Pharmaceuticals ( NASDAQ: RCKT) stock was soaring yesterday as the company announced alignment with the FDA on its potentially pivotal study of in vivo gene therapy RP-A501, indicated for Danon Disease, which will begin this quarter.
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