Angiochem – 2012 Fierce 15

Dr. Jean-Paul Castaigne, CEO of Angiochem

Based: Montreal
Founded: 2003
CEO: Dr. Jean-Paul Castaigne
Clinical Focus: CNS diseases

The Scoop: The blood-brain barrier poses a major roadblock for ushering drugs from the bloodstream to the central nervous system, which is host to a wide variety of diseases with extremely high unmet medical needs. Include Parkinson's disease, Alzheimer's and many other ailments and conditions in this CNS equation. Enter Angiochem, a startup with a unique approach for penetrating the body's natural blockade to foreign molecules trying to reach brain cells--including the lion's share of drugs. London-based drugmaker GlaxoSmithKline ($GSK), a drug giant with high hopes for the rare-disease market, struck an accord with Angiochem early this year to work on delivering enzyme treatments for orphan indications into the brain. And Geron's ($GERN) most promising anti-tumor drug for cancers that spread to the brain comes from Angiochem, which stands to gain royalties and milestones in its partnership with the storied biotech.

What Makes it Fierce?

Next-generation drugs for cancer and rare diseases are all the rage in biopharma, and Angiochem has smartly applied its proprietary technology to both of these fields and others. And the Montreal-based biotech has bankrolled much of its work on researching new drugs for brain diseases with funds from partnerships, which have become ever-more vital to the survival of small developers amid the squeeze on venture dollars and other traditional sources of investment in the sector.

Angiochem considers itself a clinical-stage company as Geron has been studying GRN1005, the featured asset in the two biotechs' alliance, in a pair of mid-stage studies of the treatment to combat cancers that have spread to the brain from lung and breast cells. (The drug, which marries Angiochem's delivery tech with the known chemo med paclitaxel, became Geron's most promising drug after the developer earlier this month scrapped a Phase II study of its other top contender, imetelstat, for patients with HER2-negative breast cancer on disappointing trial results.)

Yet the most exciting developments for Angiochem this year have been in the rare-diseases arena. The company raised its profile and brought publicity to its preclinical efforts in transporting enzyme drugs for orphan disorders into the brain in February, when it announced a deal potentially worth $300 million with GlaxoSmithKline. Glaxo has initially tapped Angiochem for an early-stage program focused on an unspecified lysosomal storage disorder (LSD). Glaxo, Pfizer and other Big Pharma groups have invested in new LSD treatments, which are the bread and butter of the rare-disease businesses of Genzyme and Shire ($SPHG).

"The Glaxo deal was a big boost for the company not only in terms of having more money but also in terms of having enzymes to work with and the collaboration with really legitimate people," says Angiochem CEO Dr. Jean-Paul Castaigne. "We all noticed that the company has improved dramatically."

Genzyme and Shire have advanced several pioneering--and lucrative--enzyme drugs to market for a range of rare genetic diseases. However, the blood-brain barrier doesn't allow enzyme drugs such as Shire's Elaprase, a treatment for the LSD known as Hunter syndrome, to access and address deficiencies in the brain. Angiochem wants to help GSK and other players in the rare-disease arena overcome this hurdle.

To sneak enzymes and small-molecule drugs into the brain, Angiochem combines its engineered peptide compounds with the meds. The company's method of penetrating the blood-brain barrier, designing drugs to bind to the LRP-1 receptor, exploits knowledge of how insulin and other essential hormones are allowed to pass through the natural barrier. The company has human evidence, based on biopsies, that significant amounts of drug reached brain cells using this method, Castaigne says.

Geron offers the next big referendum on the value of Angiochem's technology, with results from its mid-stage studies of GRN1005 against brain metastases due out next year. Brain cancer and LSDs are entirely different diseases, but Angiochem uses the same tech platform for delivering drugs against all of those ailments and others. In the meantime, Castaigne says, his company is seeking more partnership deals in rare diseases and moving ahead with research of delivering monoclonal antibodies through the blood-brain barrier.

Partnership dollars have extended Angiochem's cash runway since the company's first-round financing in 2006. (Castaigne, a biopharma veteran with past stints at J&J, Novartis and Sanofi, says the small company has raised about $25 million from backers.) And the startup has hired Pamela Esposito, formerly of BioVex (which Amgen bought last year in a deal worth up to $1 billion), as a vice president to help generate tie-ups with new partners.

Investors: Business Development Bank of Canada, VIMAC Milestone Medica Fund, Valeo Management and angel backers in Quebec City.

-- Ryan McBride (Email | Twitter)

Angiochem – 2012 Fierce 15