PhaseBio raises $34M to test orphan disease strategy — focusing first on AstraZeneca drug

After spending the past three years repositioning itself as an orphan disease-focused biotech, PhaseBio is ready to roll with a fresh $34 million raise.

The Malvern, PA-based company owes its name to its elastin-like polypeptides platform technology, which creates therapeutic fusion proteins that undergo a fully reversible phase transition — thereby significantly extending their half lives.

Jonathan Mow

PhaseBio once saw an application for that tech in diabetes as well as cardio, attracting pharma giants AstraZeneca and Johnson & Johnson’s venture arm, as well as VC players New Enterprise Associates, Hatteras Venture Partners and Fletcher Spaght Ventures for its 2015 Series C, in which it got $40 million to, among other things, advance a once-weekly, long-acting basal insulin.

But that drug, like its long-acting GLP-1 that delivered seemingly approvable but not great Phase IIb results, has now been set aside.

“For type 2 diabetes, if you’re not gonna be first in class or best in class, it’s challenging, especially as a small biotech company developing assets for that space,” CEO Jonathan Mow tells me.

Instead, Mow’s team of 20 has now found a new lead drug in PB2452, a reversal agent for the blood thinner Brilinta (ticagrelor) for use in acute situations where patients are experiencing active bleeding or require urgent surgery.

“It’s very analogous to what Portola did with Andexxa, although Andexxa is in a different class of agents — it’s reversing factor Xa, and we are reversing ticagrelor which is P2Y12 antagonist,” Mow says.

John Sharp

While PhaseBio only licensed the drug from AstraZeneca — which markets Brilinta — late last year and is still in the middle of a proof-of-concept study, Mow sees his team wrapping up a Phase II and initiate a Phase III around the end of 2019.

“PB2452, can be developed in a much shorter time period, and it will pay for us to develop PB1046,” their in-house asset for pulmonary arterial hypertension that’s been in the pipeline for a while, he says. “So even though it’s only in Phase I, we will get to the later stages of development much more quickly than we will with PB1046.”

That combination of a short development timeline and a platform tech is what drew their new investors — Cormorant Asset Management, Rock Springs Capital and Mountain Group Partners — to participate in the round, CFO John Sharp says. Old supporters also came back for more.

The funding will also pay for a Phase II with PB1046 in PAH. It’s not an easy market to break into, but Mow says their analog vasoactive intestinal peptide — with its 60 hour half life and novel mechanism of action — will make room for itself alongside the likes of United Therapeutics, Actelion and Gilead.

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