Last month, Elan(ELN) spun off Prothena(PRTA). As we wrote at the time, Elan gave Prothena $125 million in cash and a research team with no pipeline. Elan itself now consists mostly of Tysabri, a blockbuster drug on which it is partnered with Biogen Idec(BIIB). We have to admit, we knew very little about Prothena, so we were grateful when a profile appeared this week on Xconomy.
Prothena is focusing on monocolonal antibodies designed to clear the body of misfolded or abnormal proteins that have been implicated in blood disorders and in neurodegenerative illnesses including Parkinson’s disease and Alzheimer’s disease. Other antibodies in the company’s portfolio attack molecules linked to inflammatory disease, cancer, diabetes, and Alzheimer’s.
Prothena CEO Dale Schenk, the former chief scientific officer at Elan, says the promise of Prothena’s drug pipeline will be more visible to investors now that the company is “out from under the umbrella of the Tysabri effort.”
Prothena employees, including Schenk were part of the Athena Neurosciences team which successfully developed Tysabri. Though, Elan left Prothena with no drugs in trials, Prothena hopes to begin its first Phase I trial early this year.
Prothena’s drug candidates were all “home-grown” within Elan’s former research units, Schenk says. The lead candidate, NEOD001, is an antibody designed to flag damaging deposits of proteins so they can be cleared away by the body’s scavenger cells.
NEOD001 attacks the problematic amyloid proteins found in two related diseases called systemic amyloidoses. The first disease is primary amyloidosis, a blood disorder in which plasma cells produce misfolded amyloid proteins that form deposits in various organs. About 1,200 to 3,200 new cases arise in the United States each year, Prothena estimates. The company received an orphan drug designation for NEOD001 from the FDA in 2012, and expects to begin a Phase I trial early this year for treatment of primary amylodosis and a related disease, secondary amyloidosis. In this disorder, the damaging protein deposits may have been triggered by another illness, such as an infection. About 8,000 patients in the US and Europe suffer from secondary amyloidosis.
Although the Phase I trial will focus primarily on the safety of NEOD001, Schenk says Prothena may glean some early hints about efficacy by looking at subsets among the trial participants who exhibit certain biomarkers.
Next in Prothena’s pipeline is NEOD002, an antibody designed to block the effects of misfolded forms of the protein synuclein, which have been linked to neurological disorders including Parkinson’s disease.
There is some interesting science here, no doubt, but with only 2.5 years of cash at the company’s current burn rate, we would be wary given the high failure rate of early drug candidates. Though we wouldn’t count this out, with no near-term catalysts, we think you’ll be able to buy this cheaper as the cash hoard dwindles.
Disclosure: The author holds no position in any stock mentioned
- Prothena of South San Francisco Gets Head Start as Elan Spinoff (xconomy.com)
- Elan Hives Off Prothena (health.einnews.com)
- Misfolded Protein Transmits Parkinson’s from Cell to Cell (scientificamerican.com)