Kees Been has banked the $20 million round needed to move a bold new development program for delaying or preventing Parkinson's toward the clinic. And the veteran biotech CEO at the helm of Lysosomal Therapeutics Inc. is moving forward with a group of high-profile backers in his corner.
The big idea at Cambridge, MA-based LTI is that Parkinson's can be linked to the same kind of lysosomal storage disorder behind Gaucher's disease. Atlas Venture helped seed the company (Bruce Booth is taking a leading role) and came back into the A round with just the kind of biopharma company the group likes to keep: Hatteras Venture Partners, Lilly Ventures, Sanofi-Genzyme BioVentures, Roche Venture Fund, Partners Innovation Fund and several original angel investors, including Orion Equity Partners, LLC, and LTI co- founders Henri Termeer and Bob Carpenter.
Termeer, of course, is best known for his long-running former job as CEO of Genzyme, which pioneered drugs for Gaucher's. And he was ideally positioned to understand the insights of Dimitri Krainc and a postdoctoral fellow named Joseph Mazzulli who worked at Krainc's lab at Massachusetts General Hospital.
The initial seed money that Been initially reviewed with FierceBiotech last spring was enough to do some preliminary work on molecules that can penetrate into the brain. Now this A round will back lead optimization for the molecules brought in from the NIH. LTI will also be working on a biomarker program that can clearly identify low levels of the lysosomal enzyme glucocerebrosidase (GCase) that they believe is a likely trigger for the deadly disease.
The biotech has a scientific odyssey to complete before it can hope to offer patients a new therapy, which has remained an elusive dream to patients. Not only does Been's team have to prove that their new drug works, they also have to prove that their theory about the cause of Parkinson's is accurate. But Been is happy with the progress the 10 staffers at LTI have made so far.
"The molecules do go into the brain quite nicely," says Been, the former CEO of EnVivo, about the work done so far. "We had a great starting point with licensed molecules from the NIH."
If LTI is right, their drug could one day be used much like the way cholesterol drugs are applied to patients at risk of heart disease. Anyone identified through biomarker tests as at-risk for the disease would be able to start treatment before any symptoms appear, possibly delaying or preventing the ailment. And LTI is looking to build a pipeline of related drugs, quite possibly with partners.
"I have to admit that we have significant interest from many pharma companies," says the CEO. "This whole biology is starting to unfold."
To capitalize on that interest, LTI just hired Darren Braccia as VP of business development. Braccia's last post was at Vertex.
- here's the release