When Beam was slapped with a clinical hold for its base-edited Car-T project BEAM-201, some harboured hopes that Verve’s VERVE-101, which uses the same technology, would escape this fate. For one, BEAM-201 involves multiple edits, while VERVE-101 makes a single base change. However, investors who kept the faith were punished today by an FDA hold on VERVE-101’s IND application; the move raises the question of whether the regulator is cautious about base editing in particular, or in vivo gene editing in general. Without knowing details of the agency’s concerns, it is hard to say. Some groups have managed to get in vivo projects into US trials, including Excision Biotherapeutics, whose EBT-101 uses Crispr to make multiple cuts in the HIV genome. Intellia, however, which reported more data over the weekend on its amyloidosis contender NTLA-2001, has so far only trialled its in vivo assets outside the US. One mark against Verve is that various, more conventional anti-PCSK9 therapies are available, and the FDA has recommended that gene editing therapies be reserved for patients with no other options. The Heart-1 study of VERVE-101 continues outside the US, but Verve’s stock sank 19% this morning.
To read Evaluate Vantage’s report into next-generation genetic medicines, including more information on Verve and Excision, click here.
|Status of selected gene edited projects|
|VERVE-101||Verve Therapeutics||In vivo base editing project targeting PCSK9 for high cholesterol||Ph1 Heart-1 enrolling in NZ & UK; US IND on clinical hold|
|BEAM-201||Beam Therapeutics||Ex vivo base edited Car-T for leukaemia/ lymphoma||US IND on clinical hold; Beam has submitted response to FDA|
|NTLA-2001||Intellia Therapeutics/Regeneron||In vivo Crispr editing project targeting TTR for amyloidosis||Ph1 enrolling in NZ, Sweden, UK; data reported|
|NTLA-2002||Intellia Therapeutics||In vivo Crispr editing project targeting KLKB1 for HAE||Ph1/2 enrolling in Netherlands, NZ, UK; data reported|
|EDIT-101||Editas Medicine||In vivo Crispr editing project targeting CEP290 for LCA10||IND cleared 2018; in ph1/2 Brilliance; disappointing data reported|
|EBT-101||Excision Biotherapeutics||In vivo Crispr therapy for HIV||IND cleared 2021; ph1 (NCT05144386) recruiting|
|Source: Evaluate Pharma & clinicaltrials.gov.|