QIMR Berghofer attracts up to $63M for potential new multiple sclerosis treatment

A potentially ground-breaking new treatment in development for multiple sclerosis created at QIMR Berghofer Medical Research Institute has secured a deal worth up to $63 million in a substantial vote of confidence.

The investigational treatment, known as ATA188, is an allogeneic off-the-shelf Epstein-Barr virus T-cell therapy developed by QIMR Berghofer immunologist Professor Rajiv Khanna and licenced to Atara Biotherapeutics in 2015. It is currently manufactured at the Institute’s cell therapy manufacturing facility, Q Gen Cell Therapeutics.

US investment firm Maverick Capital has acquired the rights to a portion of the Institute’s royalties, including a cash payment of $28-million upfront and potential milestone payments of up to $35-million. The Institute has retained royalties preserving future benefits from the treatment.  

Professor Khanna said the deal was a substantial endorsement of the new treatment’s potential as a game-changing therapy.

“We share Maverick Capital’s optimism about this potentially transformative immunotherapy. During the Phase 1 trial in Queensland we saw a dramatic and sustained improvement in many patients with progressive multiple sclerosis,” Professor Khanna said.

“Some people with multiple sclerosis who had been dependent on a walking aid were able to move around unassisted for longer periods of time.

“These early results have given us a lot of hope that the treatment may improve, and potentially even reverse, debilitating multiple sclerosis symptoms ahead of the next stage of clinical trials being sponsored by Atara in Australia and the United States.”

Ben Silver from Maverick Capital says they are excited to provide a financial solution to further the Institute’s commitment to patients.

“This immunotherapy is a testament to the Institute’s unique approach to medical research, and we believe it will prove to be the first multiple sclerosis drug to actually reverse disease – not just slow progression.”

QIMR Berghofer Director and CEO, Professor Fabienne Mackay said the deal will help the Institute develop and commercialise future research-derived intellectual property.

“The work our researchers are doing to develop breakthrough cellular immunotherapies for patients with cancers and autoimmune disorders is world leading,” Professor Mackay said.

“This multimillion-dollar agreement allows us to invest in the development and commercialisation of other life-changing treatments.

“It is no substitute for the vital community support and highly-competitive research grants we rely on. But we’re committed to exploring new and sustainable ways to take our researchers’ discoveries from inception to bedside.”

The successful development of cell therapies stems from the Queensland Government’s $1.4 million funding injection to upgrade the facilities at Q-Gen Cell Therapeutics in 2018.

Treasurer and Minister for Trade and Investment Cameron Dick said QIMR Berghofer’s deal, highlighted the value of the Queensland Government’s approach to supporting industries like biotechnology.

“We know Queensland researchers are among the best in the world when it comes to fields like immunotherapy and biopharmaceuticals,” the Treasurer said.

“The technologies and treatments they develop are in demand around the world, but we want to ensure we can keep those highly skilled jobs here in Queensland.”

The partnership between QIMR Berghofer and Atara Biotherapeutics has secured $58 million in funding to the Institute to develop and manufacture ATA188, and other cellular therapies.

Atara is currently enrolling EMBOLD, a Phase 2 randomised, double-blind, placebo-controlled dose-expansion study evaluating the safety and efficacy of ATA188 in patients with progressive multiple sclerosis, across clinical sites in North America and Australia.

About multiple sclerosis

Multiple sclerosis is a chronic neurological disease estimated to affect more than 25,000 Australians and up to 2.8 million people worldwide. It can cause a range of symptoms including problems with coordination, balance, weakness, arm and leg functioning, cognitive problems and memory loss.