Biotech Values

[DewDiligence]

DYAX pediatric results in CF:

[This phase-2a trial in children supplements the phase-2a trial in adults reported in 2002 (#msg-1946072). Not much data here on efficacy, but the parties intend to proceed to a phase-2b trial, according to the PR.]

http://biz.yahoo.com/bw/040224/245458_1.html

>>
Dyax Corp. and Debiopharm S.A. Report Positive Results from Phase IIa Clinical Trial with DX-890 'EPI-hNE4' in Children with Cystic Fibrosis

CAMBRIDGE, Mass. and LAUSANNE, Switzerland--(BUSINESS WIRE)--Feb. 24, 2004--Dyax Corp. (Nasdaq: DYAX) and Debiopharm S.A. today announced the preliminary results of a phase IIa dose escalating multicenter study conducted by Debiopharm in 34 pediatric patients with cystic fibrosis (CF) using DX-890 (also known as EPI-hNE4), a recombinant inhibitor of human neutrophil elastase discovered by Dyax. Human neutrophil elastase (hNE), an enzyme produced as part of the inflammatory response, is implicated in the loss of lung function in patients with CF and other clinical conditions.

The study, which was conducted by Debiopharm in Europe under its collaboration agreement with Dyax, evaluated the daily administration of DX-890 when given as a nebulized formulation at four concentrations (1 mg/ml in week 1, 2.5 mg/ml in week 2, 5 mg/ml in week 3, and 10 mg/ml in week 4), administered during a 10 minute nebulization session. This open label study measured the inhibitory effects of DX-890 on sputum hNE.

The preliminary results confirm the good tolerability and the expected pharmacological effect, inhibition of neutrophil elastase, in the sputum of pediatric CF patients when given as a nebulized formulation. A total of 34 children were recruited, of which 27 completed the protocol and were assessable. Of these, 20 responded to treatment as demonstrated by decreases in hNE activity in sputum. There was a pronounced dose effect. DX-890 was well tolerated, with only four of the 34 recruited patients experiencing minor treatment related side effects, mainly coughs.

In light of these results, Debiopharm S.A., in cooperation with Dyax Corp., is planning a larger multicenter phase IIb study later this year to detect potential clinical benefit and to confirm tolerability of DX-890 in CF patients.

"We are pleased and encouraged by the results of this phase IIa trial with DX-890, a Dyax discovered small protein with high affinity and specificity for human neutrophil elastase. I look forward to learning more about DX-890 from subsequent clinical efficacy trials," said Henry E. Blair, Chairman and CEO of Dyax Corp.

"In this trial we have demonstrated the safety and pharmacological effect of DX-890 in pediatric patients when administered by inhalation after nebulization," said Rolland-Yves Mauvernay, President and CEO of Debiopharm. "Cystic fibrosis is a debilitating illness which dramatically reduces life expectancy. With positive results from this trial it is essential that we now progress to conducting the phase IIb trial to demonstrate clinical benefit and define DX-890 dosing for a phase III subsequent trial."
<<