Biotech Ideas

Joe Edelman’s biotech fund annualized 30.2% net-of-fees return since its inception in 1999 is the best among all human-managed hedge funds.

Edelman’s top five holdings below make up about a third of his portfolio.

Neurocrine Biosciences (NBIX), San Diego, CA Edelman says Ingrezza, the first drug approved for the treatment of involuntary jerky movements associated with the use of antipsychotic medications, could also treat Tourette syndrome.

Amicus Therapeutics (FOLD), Cranbury, NJ Edelman has high hopes for the company’s treatment for Pompe disease, a rare muscle-wasting affliction that kills infants, which combines an engineered enzyme replacement with a molecular chaperone.

Global Blood Therapeutics (GBT), South San Francisco, CA Edelman is bullish on Voxelotor for treating sickle-cell anemia, a $5 billion global market. GBT is Perceptive’s biggest potential home run.

Alnylam Pharmaceuticals (ALNY), Cambridge, MA The company’s RNA-interference program includes a remedy called patisiran, which aims to inhibit the activity of a gene connected to a rare disorder that attacks organs and the nervous system.

Zogenix (ZGNX), Emeryville, CA The company is advancing the science of antiepileptic remedies by showing that targeting serotonin can be a game changer in maladies like Dravet syndrome epilepsy in children. Edelman expects its new drug, ZX008, will work in other rare seizure disorders.…