Alnylam Pharmaceuticals Inc. (ALNY), a biotechnology company developing a treatment for rare diseases, rose to its highest price ever after reporting positive results in an early trial of its drug.
Alnylam, based in Cambridge, Massachusetts, rose 16 percent to $43.53 at the close in New York, its biggest one-day increase since July 2012. It was the highest price since the company’s May 2004 initial public offering.
A subcutaneous injection of Alnylam’s experimental therapy ALN-TTRsc cut 80 percent of a protein called TTR in healthy volunteers, the company said today in a statement. In some people, TTR proteins can damage organs and nerves, causing degenerative disease.
“These data validate Alnylam’s platform technology,” said Alethia Young, an analyst with Deutsche Bank AG, said today in a note to clients. “We think 80 percent knockdown is an efficacy home-run, though our checks have suggested that 50 percent knockdown may be enough also.”
Alnylam said it plans later this year to take the drug into the second of three phases of testing usually required for regulatory approval. Alnylam said a Phase III trial could begin in 2014. It will be tested in patients with familial amyloidotic cardiomyopathy, a degenerative heart disease affecting at least 40,000 people worldwide in which the walls of the organ thicken, weakening its ability to pump blood.
Paris-based Sanofi (SAN)’s Genzyme unit is a partner with Alnylam to develop and market the drug in some parts of the world.
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