shares shot higher Monday after the company announced positive late-stage trial results for its Hereditary Angiodema (HAE) drug DX-88, and will now complete its filing for FDA approval.
Dyax shares were up more than 10% to $4.78 in recent trading.
The company said that a phase III trial, dubbed Edema 4, successfully met its primary and secondary endpoints, replicating results from an earlier trial. Dyax also said the drug was well tolerated with no drug-related serious adverse events reported.
HAE causes periodic, acute episodes of painful swelling in a patient's extremities, gastrointestinal tract and, most dangerously, the airway passages. It's estimated that about 30% of HAE patients die due to suffocation caused when their airway swells shut.
The disease affects roughly 10,000 patients in North America, according to Dyax.
Cowen analyst Phil Nadeau, who has an outperform rating for the stock, said in a note to investors that his model projects $130 million in 2012 sales from DX-88 (Dyax posted revenue in its most recent quarter of $3.8 million, along with a loss of nearly $25 million)
With its first marketed drug, Dyax will enter a competitive market -- one that has seen a spate of regulatory delays -- for a rare disease that affects only 10,000 people in North America.
But Dyax's offering has its advantages.
DX-88 can be given to patients with a simple injection under the skin, instead of intravenously, which may make it more convenient and stronger competitor when launched.
Dyax is expected to file the last module of its application for DX-88 for FDA approval early in the fourth quarter based on the phase III study results announced on Monday. This means the company will likely have the product in market around mid-year 2009, writes Needham analyst Mark Monane, who has a buy rating and an $8 price target for the stock.
The company is in an exclusive negotiation period with Dompe regarding European rights to the DX-88 and has teamed up with
for North American rights to the drug in another indication.
Other HAE drug developers have won premium takeout prices in recent months.
( SHPGY) announced that it was buying Jerini and its lead product HAE drug Firazyr in a deal worth $521 million. However, Wall Street widely expects U.S. approval will require another clinical trial, giving Dyax a buffer (pending approval) before this competitor enters the market.
also announced in July that it would spend at least $443 million to buy Lev Pharmaceuticals, which is expecting to hear from the FDA on Cinryze, its intravenous HAE treatment seeking approval to treat and prevent attacks, on or before Oct. 14.
If approved, Cinryze could still compete with Behring's Berinert P, a prospective treatment seeking approval for acute HAE attacks -- not a prophylactic, or preventive treatment for the disease.
The FDA was expected to make its decision on Berinert P on or before Sept. 6, but the company confirmed with Wall Street analysts on Friday that it has been delayed (60-90 days), based on further "data slicing requirements" according to Susquehanna analyst Jason Kolbert.
Berinert P, like Cinryze, is given intravenously, not subcutaneously like DX-88 and Firazyr.
"From our conversations with HAE patients, we know that they prefer subcutaneous therapy," says Kolbert who has concerns regarding the market size estimates represented by consensus. Kolbert believes the bigger market opportunity for DX-88 is not in HAE, but in areas such as drug-induced edema and prevention of blood loss in surgery.
Dyax recently signed an agreement with Cubist for North American rights to DX-88 in blood loss prevention in heart surgery.