Lone Star biotech banks $11.4M for PhII orphan drug odyssey

Austin-based Savara Pharmaceuticals has rounded up $11.4 million in cash to fuel the upstart biotech through a key transition phase: taking its lead development program through a crucial proof-of-concept study that could leave the biotech on the threshold of a late-stage trial. And it's done it with an unconventional group of angel backers who rarely find themselves in a multimillion-dollar round like this.

The NIH's National Heart, Lung and Blood Institute contributed $4 million of that in a grant while a collection of angel investors provided the lion's share of a $7.4 million Series B extension. Savara's new investors include members of the Tech Coast Angels and the North Texas Angel Network, along with returning investors from the Central Texas Angel Network and The Keiretsu Forum, which led the $8.6 million first tranche of the Series B. Individual investors also added to the round, which brings Savara's total raise to date to an impressive $19 million.

The money is being banked for the Phase II study of AeroVanc, a reformulation of vancomycin which Savara describes as the first inhaled antibiotic now being developed for MRSA infections in cystic fibrosis patients.

In two small Phase I studies, Savara "showed we can deliver concentrations of the drug into the lungs of cystic fibrosis patients" in doses big enough to combat MRSA, Taneli Jouhikainen, head of product development, tells FierceBiotech. In the Phase II ahead, the company will recruit 80 patients with persistent lung MRSA at 22 sites. "We should be done in about a year," he adds, with 6 months reserved for enrollment.

The new money will get the company through the Phase II and into late 2014, giving the company enough time to decide whether it will need a Phase IIb or would have the opportunity to go straight into a late-stage pivotal study. It would also give Savara--run by a virtual crew of 7--some time to check out the possibilities of organizing a more traditional venture group to finance the company as well as scout a partnership. 

Savara has kept a low profile until now. But CEO Rob Neville, who started out on the board after jumping in as an early investor, believes Savara has the kind of biotech strategy that could help it gradually grow into a specialty medicines company with a pipeline of products. Savara snagged orphan drug status from the FDA for AeroVanc, setting it up for a lengthy exclusive run on the market. It's aimed at about a third of the CF patients in the U.S., a hefty 10,000 patients, and reformulating vancomycin into an inhaled form provides the chance of meeting an unmet medical need with a well-known therapeutic. And the virtual staff is tapping a network of global CROs and CMOs to help keep costs to a minimum--a popular strategy in the startup community.

The angel investors also noted that the sizeable contribution marks a new stage for them.

"Completing a Series B of this size with 100% angel money is a major accomplishment," said Sergio Gurrieri, TCA lead for the Savara financing deal. "It demonstrates TCA's increasing effectiveness at syndicating deals with other angel networks to raise larger rounds and drive innovation.

- here's the release from Savara
- get the release from TCA