Novartis eyes up another gene therapy – Vantage

Apelliss intravitreal formulation of pegcetacoplan targeted C3, and produced a hit and a miss in its twin phase 3 studies, leaving that projects approval prospects in doubt.

Gyroscope recently reported interim data from the phase 1/2 Focus study of GT005, which is delivered via subretinal injection and uses an AAV2 vector. The trial found no serious adverse events among 28 patients, but there was one case of choroidal neovascularisation, which was possibly related to GT005. Apelliss pegcetacoplan was also linked with neovascularisations in phase 2, although this was not so much of an issue in phase 3.

Focus also showed biomarkers going in the desired direction: 11 of 13 evaluable patients had increased complement factor I; all of these 11 had decreases in certain proteins associated with complement activation.

It will now be up to Novartis to prove that this translates into a benefit on geographic atrophy progression. Two phase 2 trials, Explore and Horizon, are ongoing.

Doubling down

Novartis was not the only big pharma to see potential in Gyroscope: Sanofi, another serial acquirer, made a $40m investment in the UK group last month. Other major players are also getting involved in geographic atrophy, with Roche this week striking a small licensing deal with Lineage Cell Therapies.

In many ways, the Gyroscope deal makes sense for Novartis: the group already has a presence in gene therapy and has made no secret of its intention to expand here. In the past year or so it has also picked up the early-stage ocular gene therapy players Vedere Bio and Arctos Medical.

And, with gene therapy price tags depressed, now is probably a good time to buy. But big questions still hang over the field in general, notably around safety, durability and commercial viability.

Even Zolgensma, a relative success story, has not been without its problems, and with US sales flattening the jury is still out on whether the product will justify the $8.7bn that Novartis paid for its developer, Avexis.

In a few years it should become apparent whether Novartiss decision to stick to its gene therapy guns was laudable or just another example of the sunk-cost fallacy.

The undisputed winner from todays takeout is Syncona, Gyroscopes founding investor, which also had a successful exit with Biogens 2019 takeout of another eye gene therapy player, Nightstar. That deal turned out to be a dud, however, and Novartis will have to hope that Gyroscope does better.

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Novartis eyes up another gene therapy - Vantage