Over the past 7 months, Dimension Therapeutics has been moving fast to take its place among a new wave of gene therapy developers looking to revolutionize the way some diseases are treated. Seeded by Fidelity Biosciences and working with a delivery vehicle developed by a noted researcher in the field, the Cambridge, MA-based biotech will later today unveil a $252 million licensing deal with Bayer--complete with a $20 million upfront--and a $30 million Series A round put up by Fidelity and new investor OrbiMed.
In the deal with Bayer, which is using its new pact to jump back into gene therapy, the pharma giant will fund the remaining preclinical work being done at Dimension on a treatment for hemophilia A as well as a followup Phase I/IIa study. Bayer can then take over for Phase III, with worldwide commercialization rights and a royalty stream reserved for Dimension, if all goes well.
That leaves a hemophilia B treatment--the more advanced program, which targets a smaller patient population-- in Dimension's hands, which the company founders say they can take all the way through to the market.
Dimension launched with an ambitious game plan. CEO Tom Beck and Chairman Ben Auspitz are both partners at Fidelity Biosciences, a division of Fidelity Investments which has made some major investments in drug development. And they hunted down information on "every company in existence in gene therapy" before starting Dimension, says Beck.
The key early move was striking a technology licensing deal with ReGenX, a spinoff from the University of Pennsylvania which is working with AAV technology developed by scientific founder James Wilson. Wilson runs the gene therapy program at the university. Dimension plans to use the AAV technology in-licensed from RegenX to insert a corrective gene to counter the defective one that causes hemophilia. And while Beck is cautious when asked if the biotech is shooting for a functional cure, he's excited by the animal data available that shows a longterm response that's been tracked for years.
The plan is to build a full pipeline of new gene therapy products.
"We do think of this as a company that is built to be sustaining," says Auspitz. "The model for us is Ultragenyx."
Ultragenyx under CEO Emil Kakkis, who's been acting as an adviser to Dimension, has put together an ambitious pipeline of rare disease therapies in lightning fashion, recently pulling off a successful IPO. Kakkis set out early on to build a company that would do deals and develop products all the way through to an approval, then marketing some of its own products in various regions of the world. And Dimension's founders see a similar path ahead for themselves.
Hemophilia A and B have attracted considerable R&D investments in recent years. Just days ago Biogen Idec racked up an FDA approval for Eloctate, a much-improved hemophilia A drug which produced a median annual bleed rate of 2.0 in a late-stage trial. That followed fast on the heels of an OK for a new hemophilia B drug, with Biogen engaged in a race to regulators with competing therapies from Bayer as well as Novo Nordisk ($NVO). Peak sales estimates for Eloctate for the larger hemophilia A market is hovering around $1.5 billion.
But Dimension and now Bayer are working on a preclinical gene therapy program that hopes to be a radical game changer for this disease, one in which victims of the disease can look forward to much, much more than weeks or months between bleeding episodes.
Dimension made the leap into gene therapy as a new generation of biotechs led by the likes of bluebird bio has been gaining headlines with some remarkable results on just a handful of patients. Gene therapies that target small patient populations can deliver proof of concept data with just a handful of patients. And late-stage testing can come quickly and be completed with relatively tiny groups, when you compare them to big diseases like diabetes or heart failure.