The US Food and Drug Administration (FDA) has given Rocket Pharmaceuticals approval to continue its pivotal Phase II trial of gene therapy RP-A501 in Danon disease.
This follows the regulator’s decision to put a clinical hold on the trial after a patient death, which occurred due to an acute systemic infection caused by treatment-induced capillary leak syndrome. Another patient experienced similar symptoms but recovered.
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This death transpired after a novel prophylactic complement C3 inhibitor was added to the treatment regimen, which Rocket incorporated into the trial protocol without informing investors.
Since then, Rocket has been dragged into a legal battle associated with the study, with investors alleging that the biotech hid key safety issues while failing to disclose protocol changes. This resulted in the company’s stock value dropping by 63% from $6.35 on 23 May before the news to $2.30 at opening on 27 May.
However, following the reignition of its Phase II trial in Danon disease, Rocket’s stocks have taken off once again, rising 31% from $2.91 at market close on 19 August to $3.83 upon market open on 20 August after the clinical hold was lifted.
The company is altering the doses that patients receive to be able to continue the trial, aligning them with reduced doses that exhibited efficacy in the Phase I trial (NCT03882437), the company said in a 19 August statement.
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By GlobalDataUse of the complement C3 inhibitor has also been discontinued, with the updated immunomodulatory regimen used in the trial designed to reflect what was administered in the Phase I paediatric cohort. This includes the use of Rapamune (sirolimus), rituximab and steroids.
According to Rocket, RP-A501 is the first gene therapy to demonstrate efficacy in a cardiovascular condition, with the drug exhibiting “potential to restore or stabilise cardiac function” in patients with Danon Disease.
Danon disease is a rare X-linked dominant genetic disorder that manifests with cardiomyopathy, skeletal myopathy, and intellectual disability.
If it were to be approved, it would be the first treatment available for the rare disease, which is characterised by heart muscle disease, intellectual disability and muscular weakness.
As RP-A501 is the only drug that is currently being developed in this indication, GlobalData analysts estimate that it will make $364m in sales by 2031 if approved.
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