By Saabira Chaudhuri
Isis Pharmaceuticals Inc. (ISIS) said it has received fast-track
designation from the U.S. Food and Drug Administration for the
treatment for familial amyloid polyneuropathy, a type of genetic
disease.
ISIS-TTR is an antisense drug in development with
GlaxoSmithKline PLC (GSK) for the treatment of transthyretin
amyloidosis, a severe and rare genetic disease characterized by
progressive dysfunction of peripheral nerve and/or heart
tissues.
"ISIS-TTR is our most advanced drug from our severe and rare
disease franchise and represents a significant near-term commercial
opportunity for us," Chief Financial Officer B. Lynne Parshall
said. "We look forward to continuing to move ISIS-TTR toward the
market for patients who have very limited therapeutic options."
The ISIS-TTR drug is part of an agreement between Isis and Glaxo
to develop RNA therapeutics for rare and infectious diseases.
The companies recently amended the clinical development plan and
financial terms relating to ISIS-TTR to support a
registration-directed Phase 2/3 clinical study, expected to start
this month.
When the study begins, Isis will receive a $7.5 million
milestone payment and is eligible to earn an additional $50 million
in pre-licensing milestone payments to support the study. In
addition, Isis said it is eligible to receive regulatory and sales
milestones and double-digit royalties on sales of ISIS-TTR.
Isis earlier this week struck a deal with AstraZeneca PLC (AZN,
AZN.LN) to develop novel generation antisense therapeutics against
five cancer targets that includes a license to develop ISIS-STAT3,
a drug Isis is currently evaluating in an early clinical trial in
patients with advanced lymphomas.
Also this week, Isis inked a deal with Biogen Idec (BIIB) to
discover and develop antisense drugs against three undisclosed
targets to treat neurological or neuromuscular disorders that could
see Isis receive over $630 million.
Shares closed Wednesday at $9.57 and were inactive in recent
premarket trading. The stock has risen 40% in the past year.
Write to Saabira Chaudhuri at saabira.chaudhuri@dowjones.com
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