Axovant Sciences has renamed itself Axovant Gene Therapies to put a seal on its switch from repurposing small-molecule drugs toward becoming a player at the cutting edge of genetic medicine.
The speed with which Axovant has made the transition after its unsuccessful attempts to develop dementia drugs has been impressive, and according to analysts at Jefferies, the company’s stock is “poised to bounce in 2019” thanks in part to three gene therapy trial readouts due before the end of the year.
The Swiss biotech says it has two early-stage gene therapy trials due to generate results next month, including the SUNRISE-PD phase 1/2 study of AXO-Lenti-PD in patents with Parkinson’s disease—a program licensed from Oxford Biomedica last June—and an investigator-led phase 1/2 study of AXO-AAV-GM2 for the pediatric disease GM2 gangliosidosis.
Later in the year it will should have a preliminary readout with GM1 gangliosidosis therapy AXO-AAV-GM1, as the first patient is scheduled to be treated with that candidate in the first half of this year. In gene therapy trials, even tiny updates on a handful of patients are watched with great interest and can lead to big stock swings, as has been seen this week with Sangamo and Solid Bio trials.
“We consider success in new AXO-AAV-GM1 and AXO-AAV-GM2 programs as all upside and currently only value AXO-Lenti-PD,” said the Jefferies analysts.
The analysts also think a positive readout for the Parkinson’s trial is very possible given that an earlier, less-potent form of the gene therapy—called ProSavin—has been shown to improve motor function as measured by the UPDRS Part III scale at six and 12 months in a 15-patient study.
In preclinical models, AXO-Lenti-PD has been shown to increase the production of dopamine 10-fold compared to ProSavin. Deficiency in dopamine-producing neurons is what leads to the motor symptoms in Parkinson’s such as tremor and rigid muscles.
Axovant has also completely revamped its management in the last few months, with new CEO Pavan Cheruvu, M.D., bringing in a series of new hires to beef up its gene therapy expertise, and it also has a slew of new investors on board following a $30 million financing in December.
Jefferies thinks the stock is relatively cheap with potential upside of 200-300% against a downside of 50%.
“Over the last year, Axovant has become leaner and more cutting-edge in its scientific approach, but we are no less ambitious in our drive to make a difference for patients,” said Cheruvu.
“We look forward to continuing this momentum with a rich set of clinical development milestones this quarter, including the first data readouts in our AXO-Lenti-PD and AXO-AAV-GM2 programs next month, and additional milestones across our pipeline throughout 2019.”
The name change will result in a change in Axovant’s Nasdaq ticker symbol from AXON to AXGT.