Small But Tenacious: Two Of San Diego's Emerging Biotechs Sophiris Bio Inc. And Lpath, Inc.

June 26, 2014

Under-the-radar biotech companies worth watching. Insight from two CEOs.

By Josh Baxt for BioSpace.com

There’s a reason why San Diego was chosen to host BIO 2014. The city has been a biomedical hotbed for decades. Companies like Hybritech paved the way, followed by Agouron, Idec, Illumina and hundreds of others.

But now, a new generation of San Diego biotechs has entered the mix. They’re small in size but driven to solve some of the world’s most pressing medical issues.

Sophiris Bio Inc.

Sophiris CEO Randy Woods In the pipeline: A new take on BPH

Randy Woods talks like a biotech evangelist. As CEO of Sophiris , he’s on a mission to shepherd the company’s treatment for benign prostatic hyperplasia (BPH) to market.

Google the word “benign” and BPH is the first item that comes up. BPH, or enlarged prostate, affects around 5 million men in the U.S., and they are eager for better treatments.

“About 50 percent of men over 50 have signs of BPH and that just increases exponentially with age,” says Woods. “As men grow older there’s excessive tissue growth in the area of the prostate called the transition zone. As the prostate grows the urethra gets constricted and men have a lot of difficulty urinating.”

Unfortunately, current treatments do not always get the job done. Drugs, such as alpha blockers and 5-alpha reductase inhibitors, can lose effectiveness, as well as causing sexual dysfunction and other side effects. Transurethral resection of the prostate (TURP), and other minimally invasive surgeries, provide longer-lasting relief but can also cause sexual or other side effects.

To reduce BPH, Sophiris has created a protein drug (PRX302) that doctors inject directly into the prostate. The drug is based on a bioengineered pore-forming protein. These proteins feature an active domain connected by six amino acids to an inhibitory tail. As long as the tail stays attached, the protein remains inactive.

In the original protein, the amino acids get cleaved by furin enzymes, but the engineered protein requires enzymatically-active PSA to become active. Conveniently, this PSA type is only found around the prostate.

Once the active domain is released, it burrows through cell membranes, destroying excess prostate tissue. In theory, a single injection can relieve symptoms for up to a year, and so far side effects have been minimal.

“The safety profile is amazing,” says Woods. “There’s no systemic detection of this drug, it only activates where you want it to work, in the prostrate.”

Cautious expansion

Sophiris has ten employees in their La Jolla office but may expand (carefully) as their drug moves forward. The company has successfully completed a Phase IIb study, improving several urination measures in men with moderate to severe BPH. Sophiris is currently enrolling for a phase III trial and pursuing a proof-of-concept study for the drug against localized prostate cancer. Woods believes PRX302 could be approved as early as 2017.

Lpath, Inc.

Lpath CEO Scott Pancoast Leveraging Lipids

It’s easy to get hung up on proteins. They’re relatively simple to study and perform the vast majority of biological functions. But what about other molecules – say bioactive lipids? They can have important signaling functions, mediating inflammation and other mechanisms linked to disease.

That’s the concept behind Lpath , which is developing monoclonal antibodies that bind to problematic lipids. The potential applications are huge – cancer, Multiple Sclerosis, wet macular degeneration – but the challenges are also enormous.

“How do you create antibodies for molecules so small they don’t even generate an immune response?” asks Lpath CEO Scott Pancoast, who has been with the company since its inception as an investor, board chair and executive. “The dogma in the 90s was you just couldn’t do it.”

But there were always people willing to try. Lpath’s founder, Roger Sabbadini, PhD, spent years working on the problem at San Diego State University. Like other researchers in the field, he knew that the key was isolating the lipid while it was attached to a protein.

“People would lay down antibodies and run the protein and lipid over it, but the signal was too weak,” says Pancoast. “Dr. Sabbadini reversed that, putting the protein with the lipid target face up and running the antibodies over it. After a few failures, he finally made it work.”

Collaborating with Pfizer

With only 25 employees and revenue from partnerships and NIH grants, Lpath has created monoclonal antibodies that bind to lipids S1P, LPA and LT. Their S1P inhibitor iSONEP, which is being developed in partnership with Pfizer, is currently in phase II trials for wet macular degeneration. The drug has shown efficacy in patients who weren’t benefiting from current treatments, reducing lesions by an average 25 percent.

Their other S1P inhibitor, ASONEP, has shown encouraging results against renal cell carcinoma and is currently in a phase IIa trial. Other, earlier stage, biologics are being tested for traumatic brain injuries, neuropathic pain, asthma and inflammation.

Like the Sophiris drug, these antibodies are showing excellent safety, since their only role is sponging up tiny lipids. The company is leveraging their partnership with Pfizer, with others under consideration, to move into phase III trials. Pancoast hopes the more advanced therapies will enter the clinic sometime next year.

Fueling the future

Lean by necessity, Lpath and Sophiris face the daunting task of moving products to the clinic without burning through their reserves. But early data is fueling optimism for both companies. If they can triangulate the approval process, and continue to convince investors, they should have promising futures.

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