Axovant Licenses Gene Therapy for Parkinson’s Disease from Oxford BioMedica

Lab and drug testing

Axovant Sciences, one of Vivek Ramaswamy’s biotech startups best known for its spectacular flameout over its Alzheimer’s drug in September 2017, is taking another shot, this time at Parkinson’s disease.

The company announced it has licensed exclusive worldwide rights to OXB-102, now dubbed AXO-Lenti-PD, from Oxford BioMedica The compound is an investigational gene therapy for Parkinson’s disease. It is designed to deliver three genes that encode a set of enzymes required for dopamine synthesis in the brain.

Oxford BioMedica focuses on lentiviral vector product development and manufacturing. It will continue as the clinical and commercial manufacturer of AXO-Lenti-PD.

Axovant says it plans to launch a Phase I/II dose-escalation trial of AXO-Lenti-PD in patients with advanced Parkinson’s disease by the end of 2018.

Part of Ramaswamy’s business strategy, aside from convincing investors to heavily invest in his companies, is to acquire cast-off drugs from other companies for bargain-bin prices. The key example is intepirdine, which is the Alzheimer’s drug that failed in a Phase III trial. Ramaswamy and Axovant bought the drug from GlaxoSmithKline for $5 million. It had shown a favorable safety and tolerability profile, and in a Phase IIb trial, showed immediate and sustained efficacy over placebo. But it had been abandoned by GSK after four clinical trials.

This appears to be something of a strategic pivot—although a very risky one—for the company. Axovant is paying an upfront payment of $30 million in cash, $5 million which will be applied as a credit towards the process development work and clinical supply. Oxford BioMedia will be eligible for various milestone payments that could exceed $812 million, as well as tiered royalties on net sales of the product if approved.

In addition, Roivant, Ramswamy’s parent company, will buy $25 million of Axovant common shares, which will support the clinical development of AXO-Lenti-PD.

The company is also shaking up its executive team, which has been undergoing a shakeup since September anyway. Last summer David Hung, former founder and chief executive officer of Medivation had taken on the chief executive role. But he left the company shortly after the Alzheimer’s failure. Pavan Cheruvu took over in February 2018.

Part of today’s announcement included that Fraser Wright will join the company as chief technology officer. He is the co-founder and former chief technology officer of Spark Therapeutics.

Cheruvu said in a statement, “Axovant remains committed to developing innovative treatments for serious neurodegenerative conditions such as Parkinson’s disease, and we are excited to partner with Oxford BioMedica, a recognized global leader in cell and gene therapy. We are also pleased to welcome Fraser to our leadership team.”

It’s anyone’s guess as to whether this shift will be successful. Parkinson’s is a slightly less volatile arena than Alzheimer’s, but it’s no picnic, either. As STAT points out, “If you bet $100 on Axovant Sciences nine months ago, you’re down 93 bucks.”

STAT further points out, “In biotech circles, the Axovant debacle became a referendum on Ramaswamy, who left a career as a hedge fund manager to build a constellation of biotech companies at work on drugs licensed from other firms.”

On the one hand, you have to admire someone who swings for the fences. But as any baseball fan knows, you’ve got to hit a homerun periodically if you do, and so far Ramswamy’s companies are striking out. That doesn’t mean all of them will—it seems likely that some of them, which are taking less risky approaches than Axovant, will get a break and become profitable. But gene therapy and Parkinson’s disease is still a tricky and difficult area to be taking big chances in. Maybe it’s good someone is willing to.

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