Further data from Biogen’s anti-Lingo-1 antibody opicinumab are expected at next month's Ectrims meeting. The project failed its phase II in relapsing multiple sclerosis, but with Biogen noting an unexpected response with the middle doses the group does not look to be ditching it just yet.
Meanwhile Nanobiotix is awaiting interim data from the pivotal trial of its radiation amplification technology NBTXR3 in soft tissue sarcoma, which if positive could enable a European approval filing.
Biogen’s shares fell 13% when it reported the failure of opicinumab's Synergy trial in June, with the MAb missing both the primary and secondary endpoint (No bingo for Lingo, June 8, 2016).
On its second-quarter results call Biogen said the results produced a bell-shaped curve, with efficacy lost at the highest dose, but middle doses showing signals of efficacy. The anti-Lingo MAb had been tested at 3mg/kg, 10mg/kg, 30mg/kg and 100mg/kg versus placebo, concurrently with Avonex.
Further analysis is expected on September 16 at the Ectrims conference in London, and the results could determine the next steps for the drug. A phase I study recently started in healthy volunteers using opicinumab produced by two different manufacturing processes, a signal that the asset is not dead and buried yet.
Ectrims will also separately see the keenly awaited presentation of data from Novartis's phase III Expand trial of siponimod, toplined yesterday, one of the first studies specifically recruiting secondary progressive MS patients to show a positive result.
Biogen’s most valuable R&D project, aducanumab, is high risk given that it targets Alzheimer’s disease, and its MS franchise looks to be hitting a plateau, so clinging on to opicinumab might be a wise move (Biogen needs to fill pipeline or rethink strategy, July 22, 2016).
Interim phase II/III data on Nanobiotix’s NBTXR3 in soft tissue sarcoma (STS) will appear this quarter and if it is positive the French group ought to be able to file for CE mark approval, the European regulatory path for medical devices, according to Edison analysts. Approval itself could follow by the end of the year, giving Nanobiotix its first marketed product.
NBTXR3 is a nanoparticle radio-enhancer injected directly into tumours. Upon exposure to ionising radiation, such as X-rays, the hafnium oxide contained in the nanoparticles amplifies the dose of energy delivered to the tumour. The theory is that this increases the efficacy of radiotherapy, keeping the effect localised so as to limit damage to healthy tissue.
The STS study has enrolled 156 patients and is testing NBTXR3 plus radiation against radiation alone; the primary endpoint is complete pathologic response, a measure of tumour necrosis, and secondary endpoints include safety, tumour volume changes and surgical resection rates.
The interim look will include data on the first 104 patients in the trial, with the final analysis of all 156 enrolled patients due by the end of this year.
Stifel analysts point to data in head and neck cancer as being promising for NBTXR3’s chances of approval in STS. The phase I/II trial in head and neck is still ongoing, but initial data from seven evaluable patients – there are 10 in the study – showed a reduction of more than 50% in tumour volumes in patients aged over 65 with advanced cancers. But this is a tiny sample size, and the trial was uncontrolled and open-label.
Nevertheless, Stifel gives the asset an 80% chance of success in STS and forecasts peak global sales of €250m ($283m) in this indication.