A while back, the U.S. Food and Drug Administration (FDA) ordered Solid Biosciences’ [NASDAQ: SLDB] to cease its clinical trials of “IGNITE DMD” Phase I/II after one of the young patients in the trials developed “serious adverse event.” FDA denied the company to resume the trials in July, even after the patient in question recovered, and no new patients developed complications, in addition to the company providing data to show the measures they had taken to ensure patients’ safety, and also to show safety related to their manufacturing process. FDA has instead asked Biosciences to provide more information on the trials as well as the safety measures they seek to undertake as extra caution going forward to resume the trials.
Three months down the line, FDA seems to have been satisfied with Biosciences. FDA has since lifted the ban on the clinical trials acknowledging that the company has responded appropriately to all the clinical hold questions. In the coming trials, the company wants to get rid of most empty viral capsids (the protein shell that contains viral material) as well as reduce the weight of child-patients to participate in these trials to 18 kilos, so that they can also cut down on the viral load of the SGT-001 therapy that the patients are exposed.
The IGNITE DMD trials are meant to find a medication that can slow down or reverse the effects of Duchenne, Muscular Dystrophy (DMD), a condition that causes leads to continued muscle wastage and weakness in boys from the age of two. It is for this reason that they are reducing the weight of the test-patients. SGT-OO1 is a gene therapy that the researchers use to inject a modified virus into the test-patients. It has a DNA coded to treat the condition that causes the disease.
The company is yet to actualize these trials, but will confirm their efficacy by 2021 since the FDA has now approved the continuation of the trials. Chardan analyst Gbola Amusa confirmed that indeed FDA had approved the continuation of the SGT-001 trials, a move that has set back on course addressing a $50 billion global opportunity. If this treatment receives approval, it will be among the few if not the only gene therapies that have been approved for human use in the world setting Biosciences on the top spot in the market.
Considering the difficulty involved with manufacturing such therapies, there are very few competitors in this field. A near-monopoly control of the market will see the stock of Biosciences rise from $4 to about $12.5, a price that is twice the one Amusa had valued the company before FDA approval.