Not developing your gene therapy? Call Axovant

Axovant's need to rebuild its pipeline is benefiting gene therapy developers.


If you run a small-cap biotech company and have a gene therapy asset languishing in your patent portfolio then this could be your lucky day: Axovant is buying.

The latest beneficiary is Australia’s Benitec, up 110% today on news that Axovant was paying it $10m for a preclinical rare disease project. As Oxford Biomedica found out a month ago when a similar fate befell OXB-102, this kind of rescue is great for immediate sentiment. Whether buying in such disparate assets can turn Axovant into a gene therapy contender is a separate question.

After all, the only thing OXB-102 and AXO-AAV-OPMD, the subject of today’s Benitec deal, have in common is that they are both gene therapies. They are based on quite separate technologies, and are manufactured differently: the former is delivered using a lentivirus while the latter uses an AAV vector.

Undeterred, Axovant management told an analyst call today that it was building a “world-leading innovative neurology company”. Manufacturing was already in place for AXO-AAV-OPMD, it said, with plans to scale this up fivefold to meet clinical trial requirements.

Progressive muscle disease

AXO-AAV-OPMD is intended to treat oculopharyngeal muscular dystrophy (OPMD), a rare genetic disease characterised by slowly progressing muscle disease affecting the upper eyelids and throat in adults.

It is unusual in using a single AAV vector to combine RNAi with gene therapy; the former silences the mutant PABPN1 gene responsible for OPMD, while the latter introduces a functional copy of this gene. Benitec is best known for its RNA work, of which BB-401, an oncology asset using antisense technology, is the lead.

OXB-102, a second-generation version of Oxford Biomedica’s troubled Parkinson’s disease project Prosavin, brought the UK group a $30m windfall from Axovant last month (Axovant looks to Oxford Biomedica for gene therapy resurrection, June 6, 2018). Oxford Biomedica is focused mainly on lentivirus manufacturing, and is Novartis’s partner for the CAR-T therapy Kymriah.

Axovant is, of course, on a mission to reinvent itself after the abject failure of intepirdine, its much-hyped Alzheimer’s disease project. Under new management the group is “methodically rebuilding [its] pipeline” through licensing deals.

It says it will continue this pipeline expansion effort – hinting that now is the time for any biotech with a gene therapy asset for licensing to pitch it to the group, which had $154m in the bank at its fiscal year end in March.

Gene therapy first

The first phase I trial of AXO-AAV-OPMD is to begin next year, comparing the gene therapy versus placebo; Axovant told analysts it was not aware of Benitec’s “silence and replace” approach having ever been used in a clinical setting.

The deal also gives Axovant rights to five undisclosed CNS gene therapy assets, the first of which targets the C9orf72 gene for amyotrophic lateral sclerosis and frontotemporal dementia. This also uses an AAV vector.

How can Axovant square the manufacturing circle? Surely it would be economically unrealistic to maintain two completely different commercial manufacturing technologies, so perhaps further down the line OXB-102 could be delivered using AAV – though this might not be feasible, and would amount to a major product change.

Judging by Axovant’s share price reaction today – the stock opened up 17% – some investors might not be thinking ahead as far as commercialisation.

To contact the writer of this story email Jacob Plieth in London at or follow @JacobPlieth on Twitter

Share This Article