Investigational gene therapy licensed for Parkinson’s disease: industry news round-up

Written by Sharon Salt, Editor

This week’s industry news round-up reveals the latest updates around spinal muscular atrophy, Parkinson’s disease and acute stroke. Find out more about our selection of the highlights below.
Our pick of the headlines include:

Roche discontinues work on olesoxime for spinal muscular atrophy

Roche (Basel, Switzerland) has disclosed that it has ended work on olesoxime – an oral compound intended to protect motor neurons – for the treatment of spinal muscular atrophy (SMA), having faced “many difficulties” during the development of the experimental neuroprotective drug.

In a statement shared online through TreatSMA (Royal Tunbridge Wells, UK), Roche discloses the most recent results of the OLEOS Phase II trial (NCT02628743), an open-label study in Europe on the long-term safety, tolerability and efficacy of olesoxime in type 2 and non-ambulatory type 3 SMA patients.

Despite positive results at 12 months of treatment, analysis at 18 months revealed deterioration of motor function. In addition to this, olesoxime presented other challenges, including its formulation, optimal dosage and requests from the US FDA and EMA to run a new Phase III trial, which Roche hoped to commence later this year.

In their statement, Roche noted that with the emergence of Spinraza® (nusinersen), requirements for how effective a new medication needs to be are now more demanding, which impacts the design and conduction of clinical studies.

“We understand the urgency of finding solutions for the SMA community and we have always tried to overcome the difficulties with olesoxime,” commented Sangeeta Jethwa, Head of Patient Partnership, Rare Diseases at Roche. “Based on all of the available evidence and the continued difficulties described above, we have decided to stop further development of olesoxime.”

Roche is continuing to advance its SMA program, which includes RG7916, a treatment candidate that modifies the splicing of premature RNA molecules to increase the production of full-length SMN. Current studies of RG7916 include the FIREFISH Phase II/III trial (NCT02913482) in babies with type 1 SMA, as well as the SUNFISH Phase II/III study (NCT02908685) and the JEWELFISH Phase II study (NCT03032172) in types 2 and 3.

Find out more about the FIREFISH trial here.


Axovant licenses investigational gene therapy for Parkinson’s disease

Axovant Sciences (Basel, Switzerland) has announced that is has licensed the exclusive worldwide rights to develop and commercialize OXB-102, now AXO-Lenti-PX, from Oxford BioMedica (Oxford, UK). A Phase I/II dose-escalation study of AXO-Lenti-PD in patients with advanced Parkinson’s disease (PD) is expected to be initiated by the end of 2018.

AXO-Lenti-PD is an investigational gene therapy for PD that delivers three genes encoding a critical set of enzymes required for dopamine synthesis in the brain, and is designed to provide patient benefit for multiple years following a single administration.

Axovant obtained rights to AXO-Lenti-PD, as well as its predecessor product ProSavin® under the terms of the license agreement with Oxford BioMedica. A Phase I/II study for ProSavin was successfully completed by Oxford BioMedica in 2014 and was reported to have met its primary endpoint. The results also revealed favorable safety and tolerability and a statistically significant improvement of motor function at 6 and 12 months.

“Axovant remains committed to developing innovative treatments for serious neurodegenerative conditions such as PD, and we are excited to partner with Oxford BioMedica, a recognized global leader in cell and gene therapy,” commented Pavan Cheruvu, Chief Executive Officer of Axovant.

“We will continue to pursue promising new therapeutic approaches based on transformative science, and will further expand our pipeline with high-quality assets like AXO-Lenti-PD. That is part of our long-term goal of building Axovant into a leader in the development and commercialization of innovative medicines for neurological indications.”

John Dawson, Chief Executive Officer of Oxford BioMedica stated: “We believe Axovant’s expertise and focus on neurological disorders, which includes PD, makes them an ideal development and commercialization partner for this program. Coupled with strong support and financial resources from parent company Roivant, we believe Axovant is well positioned to advance the development of AXO-Lenti-PD for the treatment of patients with Parkinson’s…”

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Biogen enters agreement to acquire TMS-007 for acute stroke

Biogen (MA, USA) announced earlier this week that it has entered an exclusive option agreement with TMS Co., Ltd. (Tokyo, Japan) to acquire TMS-007 and backup compounds for acute stroke.

TMS-007 is a plasminogen activator with a novel mechanisms of action associated with breaking down blood clots, and is believed to inhibit local inflammation at the site of thrombosis. This unique combination could position TMS-007 as a bent in class thrombolytic for individuals with acute ischemic stroke with potential for an extended treatment window as compared to current thrombolytic agents.

“TMS-007 complements our broader efforts in stroke, including our Phase III ready asset BIIB093 (intravenous glibenclamide), which targets prevention and treatment of edema in large hemispheric infarction, one of the most severe types of stroke. By growing our acute neurology portfolio, we aim to make new advances in a disease that in the past decade has seen limited therapeutic innovation,” explained Michael Ehlers, Executive Vice President in Research and Development at Biogen.

TMS-007 is a small molecule that has previously demonstrated an acceptable safety profile in a Phase I study and has also reduced infarct volume in experimental rodent and primate embolic and thrombotic stroke models.

The drug is currently being evaluated in a double-blind, placebo-controlled Phase II study in Japan, investigating the safety and efficacy of a single intravenous administration of TMS-007 in approximately 60–90 patients with acute ischemic stroke up to 12 hours after stroke onset. The Phase II study initiated with the first patient dosed in February 2018.

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