New Canadian biotech aims for a novel approach to blood cancer, rare disease

By Ben Adams

AvroBio CEO Geoff MacKay

A new Toronto startup has burst into life this week as it announces two "transformative" ex vivo gene therapies to tackle both blood cancer and a hereditary disorder.

The company--known as AvroBio--comes from the labs of veteran scientists Christopher Paige and Jeffrey Medin. With the backing of Atlas Venture and a partnership with the Center for Commercialization of Regenerative Medicine (CCRM), both men and a new leadership team have taken their pre-clinical work to the University Health Network (UHN) in Toronto to create the new start-up.

And they don't plan to wait around, as they immediately announced that the first series of Phase 1 programs will be in the clinic by mid-2016 which will target acute myeloid leukemia (AVR-01) and Fabry disease (AVR-02).

AVR-01 is designed as an anti-cancer immunotherapy which triggers the immune system to first detect, and then eradicate, tumor cells--and follows the theory of newer oncology meds which aim to recruit a patient's immune system to recognize the disease.

Under AvroBio's approach, a patient's cancer cells will be genetically modified to express one of the most powerful immune signaling agents, the cytokine IL-12. The modified cells are then infused back into the patient via a one-time procedure, where they quickly activate cytotoxic CD4-positive T cells that specifically target tumor cells and eliminate the cancer. A long-lasting anti-cancer immune response is maintained via both CD4-positive and CD8-positive cytotoxic T cells.

Boston-based Ziopharm Oncology is also in early stage testing for a similar mechanism of action for Ad-RTS-IL-12, although it is targeting patients with melanoma. Bristol-Myers Squibb ($BMY) also uses a comparable approach with the approved skin cancer drug Yervoy, which works as a series of four infusions per patient over the course of a year.

AML accounts for about 25% of all adult leukemias, but has the lowest survival rate. There are few approved treatments on the market, although just this week Novartis ($NVS) bagged an FDA breakthrough tag for its new AML drug PKC412.

Meanwhile, AVR-02 is designed to deliver lasting benefits for Fabry disease patients. The company's approach is to genetically modify a patient's own cells by adding a functional copy of the faulty gene.

Fabry disease is a rare, inherited disorder--only around 11,000 people in the U.S. are estimated to carry the mutation that can cause it--yet it has been a big money spinner for Sanofi's ($SNY) Genzyme unit's, which leads the market with Fabrazyme, with a $200,000 price tag per patient.

The drug works as an enzyme replacement therapy yet involves lifetime biweekly intra-venous infusions.

But Genzyme is in midstage trials for an oral version of its treatment which would also remove the need for IV administration, with New Jersey's Amicus Therapeutics ($FOLD) one step ahead as it is expecting approval for its oral contender migalastat later this year.

However, both would still need to be taken regularly whereas AvroBio said that its drug, if successful, will infuse a patient's modified cells these back into the patient via a one-time procedure. The objective is to deliver long-lasting or permanent, continuous elevation of endogenous enzyme, and remove the need for constant treatment--potentially giving it an edge over its competitors.

Financial details were not disclosed, but the firm will need a strong and continuous backing if it is to bring these two treatments to market.

Geoff MacKay--formerly CEO of Massachusetts-based Organogenesis--becomes AvroBio's president and chief executive, with Kim Warren as its head of operations, Chris Mason as its chief science officer, and Deanna Petersen as its chief business officer.

This is also a boon for the Canadian life sciences sector. John Reid, director of the team at UHN's office of Technology Development and Commercialization, which negotiated the deal on behalf of UHN, said "This deal validates our vision that Toronto will be a world-class center for the commercialization of cell-based technology developed at UHN."

- read the release