Ra Pharma gets $58.5M to challenge a rare disease's intravenous incumbent

Ra CEO Doug Treco

Cambridge, MA's Ra Pharma received $58.5 million from a mix of strategic and VC investors to take on Alexion's ($ALXN) intravenously infused orphan drug Soliris with a cheaper, subcutaneously delivered alternative.

Its preclinical peptide-inhibiting candidate aims to shake up the paroxysmal nocturnal hemoglobinuria market using an alternative form of drug delivery.

"Soliris is a really good product for Alexion," Ra CEO Doug Treco told sister publication FierceBiotech. "Our goal was to come up with an alternative therapy that would be more convenient for patients that would be as effective."

Alexion's med costs more than $500,000, another reason competition is needed.

Symptoms of the disease include severe anemia, hemoglobinuria, abdominal pain, smooth muscle dystonias, and fatigue.

Moving from half-hour biweekly intravenous injections to simpler subcutaneous shots would be a step in the right direction, but Ra told Xconomy it could go a step farther, saying its candidates could potentially be taken orally because they resemble small molecules.

On its website, Ra says that peptide modifications to increase a candidate's cell permeability--or its chances of penetrating cell walls so that it reaches its target--and other key features like bioavailability are often added after a candidate molecule has been identified, an approach that often fails to produce strong leads.

Ra uses its drug discovery platform to incorporate rare synthetic amino acids into peptides, avoiding the need to make modifications down the line.

The company focuses on diseases that are caused by dysregulation of the body's complement system, a part of the immune system that uses C5 proteins to recruit inflammatory cells to the site of injury, as well as so-called membrane attack complexes to kill pathogens. The problem is that an unregulated complement system can lead to uncontrolled attacks on red blood cells, which is where Ra's C5-inhibiting peptides come into play.

The company's also developing a complement system inhibitor to treat the eye condition age-related macular degeneration and extravascular hemolysis, which results in anemia.

The funding round was co-led by RA Capital Management, Novo Ventures, and Lightstone Ventures. VCs associated with Novartis ($NVS) and Amgen ($AMGN) also participated. And Ra announced that it has extended its multitarget collaboration with Merck ($MRK), first established in 2013.

- here's FierceBiotech's take
- read the release

Special Report: The top 10 most expensive drugs of 2013 - Soliris- Alexion