The FDA has maintained a scientific maintain on a part 1/2 scientific trial of Strong Biosciences’ gene remedy therapy for Duchenne muscular dystrophy (DMD). Strong Bio might want to submit extra data on topics together with the overall viral load per affected person to steer the FDA to elevate the scientific maintain.
SGT-001, the adeno-associated viral vector-mediated gene switch remedy being examined within the trial, has run into repeated issues throughout its time within the clinic. The most recent stage of the gene remedy’s troubled improvement started in November, when the FDA positioned a part 1/2 trial on scientific maintain in response to a case of acute kidney harm and different signs in a baby who obtained the excessive dose of SGT-001.
Strong Bio has spent the months for the reason that begin of the scientific maintain gathering and sharing proof supposed to allay the FDA’s issues concerning the trial. Late final week, Strong Bio revealed that is still an ongoing effort.
The FDA obtained particulars of Strong Bio’s affected person security measures in April, main the company to ask for extra knowledge and analyses associated to a brand new manufacturing course of. Strong Bio submitted extra proof final month, just for the FDA to inform the biotech it nonetheless must do extra to get the scientific maintain lifted.
In disclosing the information, Strong Bio stated the FDA desires to see extra manufacturing data, up to date security and efficacy knowledge for all sufferers and particulars of complete viral load to be administered per affected person. Strong Bio co-founder and CEO Ilan Ganot expressed disappointment on the FDA’s choice however is but to surrender on the asset.
“We are going to proceed to work with the FDA as we consider the scientific improvement of SGT-001 may provide significant advantages to sufferers with this devastating illness,” Ganot stated in an announcement.
Not everybody shares Ganot’s perception in SGT-001. Shares in Strong Bio have traded at under $5 for the reason that FDA imposed the scientific maintain. In 2018, Strong Bio briefly traded above $50. Since then, Strong Bio has confronted a number of scientific holds and seen Sarepta Therapeutics submit knowledge on its rival DMD gene remedy that persuaded Roche to pay $750 million upfront for the ex-U.S. rights.