Recently, biotech company Omeros (NASDAQ:OMER) closed a debt financing for $32 million and submitted an individual new drug application (IND) for a new experimental orphan drug Wednesday, indicating that it's making progress with its drug development programs. The IND adds one more clinical program to an unusually strong and diverse pipeline for a pre-commercial biotech company. Although the odds for approval for any individual drug heading into clinical trials are slim, Omeros's pipeline offers multiple shots on goal, increasing the company's chance of long-term survival and market success. Heading into 2014 and beyond, investors should be watching three important programs to better gauge this company's potential.

OMS721 could be a sleeping blockbuster
The newest IND filing relates to OMS721, a monoclonal antibody targeting MASP-2, which is thought to regulate a critical pathway of the immune system. Omeros plans to test OMS721 for thrombotic micorangiopathies, which are orphan disorders of microcirculation in the organs, typically affecting the brain. Atypical hemolytic uremic syndrome (aHUS) and thrombocytopenic purpura (TTP) are two of the better-known members of that family of disorders.

Omeros is basing its Phase II trial design for the drug on results of a Phase I trial showing successful inhibition of its target pathway, plus feedback from the FDA gleaned in a recent pre-IND meeting.

Monoclonal antibody plus rare disease is a combination that has worked well for Alexion Pharmaceuticals' (NASDAQ:ALXN) Soliris, which is approved for paroxysmal nocturnal hemoglobinuria and— ding ding, you guessed it — aHUS.

Soliris is priced at an astonishing $440,000 per patient per year, providing Alexion with a revenue stream that has helped drive its stock price up more than tenfold since the drug was approved in 2007 (from around $11 per share to $180 per share).

OMS721 is technically a follow-on to Omeros's lead PharmacoSurgery candidate, OMS302, but its potential is interesting. Results of the first human tests of the compound will show whether the established activity of the compound translates to clinical benefit. If so, and if Omeros can prove the drug is safe and effective, it will have a shot at some of the market share currently enjoyed by Soliris. 

Surgery formulations offer solid pipeline value
A large portion of Omeros's pipeline is occupied by its PharmacoSurgery program. The term PharmacoSurgery refers to delivery of FDA-approved drugs throughout the duration of surgery directly to the surgical site to pre-empt complications that can otherwise develop.

Omeros's PharmacoSurgery candidate OMS302 met its endpoints of maintaining pupil dilation and decreasing post-operative pain in a Phase III clinical trial in intraocular lens replacement surgery. The drug, a combination of ketoralac and phenylephrine, is given during surgery by adding it to the standard irrigation solution which is infused into the chamber of the eye.

Omeros is working on similar PharmacoSurgery candidates for arthroscopic and urological surgery. PharmacoSurgery formed the basis of Omeros's initial public offering in October of 2009, when it raised $61.8 million selling 6,820,000 shares at $10 per share.

The PharmacoSurgery program in general, and OMS302 in particular, is another example of a pipeline asset that could generate a solid revenue stream on its own. The use of already-approved compounds decreases the perceived risk of the program by quite a bit. Omeros is adding the innovation of delivering those compounds directly to the site of surgery. Since surgical complications and resulting hospital re-admissions are a major source of concern for doctors and payors, these products are likely to be very appealing if approved.

Potential of GPCR screening
Tucked away at the bottom of the pipeline list is a screening program for GPCRs, the largest and arguably most important family of druggable targets in the body. About forty percent of all drugs are targeted at a GPCR, or G-protein-coupled receptor. These large proteins straddle cell membranes, and transmit signals from one side to another by changing conformation when certain molecules bind to them.

Because GPCRs are designed by nature to cross the fatty, slippery cell membrane, they can be difficult to study in vitro. An especially enigmatic group of GPCRs are known as "orphan GPCRs." This group of a hundred or more proteins are known as orphans because their natural ligands are not known. The identity of the "open sesame" molecules assigned by nature for each of those orphans is a major unsolved mystery of biology, and one with direct application to biotechnology, because many, perhaps most, of them could be druggable targets with significance in human disease.

When the natural ligand of an orphan GPCR is discovered, it is known as "de-orphanizing." However, Omeros has its own proprietary high-throughput assay which unlocks GPCRs; this means that it finds small molecules that can modulate these proteins, and it has reported unlocking 21 GPCRs thus far. Most of the potential indications are in oncology and central nervous system disorders.

Omeros's GPCR portfolio represents a tremendous and rapidly growing source of candidates for future development and licensing. In spite of being very early stage and thus extremely difficult to quantify, those GPCR assets garnered the company $25 million in a 2010 financing deal with Vulcan Capital and Washington State's Life Sciences Discovery Fund. Those investors will be entitled to a percentage of profits on future drugs developed from Omeros's GPCR program.  

But Wait, There's More

In addition to those three power programs, Omeros has an early pipeline of plasmin inhibitors, plus candidates for addiction, schizophrenia, and Huntington's disease. The company's plasmin program includes a set of antifibrinolytic agents for controlling blood loss during surgery that are designed to inhibit plasmin without inhibiting kallikrein and Factor XIa, which are problematic effects of Trasylol, a drug developed by Bayer and currently owned by Nordic Group Pharmaceuticals. The schizophrenia drug, OMS824, just handed in positive Phase IIa results that show good dosing tolerability of the drug and potential to be given in combination with standard antipsychotic drugs. The exciting clinical programs at this fledgling biotech make it a stock to watch going forward.

Editor's Note: The distinction between "de-orphanizing" and "unlocking" GPCRs was not clear in a previous version of this article.

Catherine Shaffer has no position in any stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. Try any of our Foolish newsletter services free for 30 days. We Fools may not all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.