BIOCHEMISTRY, BIOMEDICINE & PHARMACEUTICS

Ionis Pharmaceuticals Highlights  - Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics - Three approved commercial RNA targeted therapies: SPINRAZA, TEGSEDI, WAYLIVRA - Numerous in house and out liscened collaborative program - Net income of $5 million on a non-GAAP basis  - Cash equivalents, and short-term investments of more than $2.3 billion,   Learn More

TEGSEDI (inotersen) injection, for subcutaneous use Inotersen is an antisense oligonucleotide (ASO) inhibitor of human transthyretin (TTR) protein synthesis. TEGSEDI contains inotersen sodium as the active ingredient. CLINICAL PHARMACOLOGY Mechanism of Action Inotersen is an antisense oligonucleotide that causes degradation of mutant and wild-type TTR mRNA through binding to the TTR mRNA, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.  Pharmacodynamics The pharmacodynamic effects of TEGSEDI were evaluated in hATTR amyloidosis patients treated with 284 mg TEGSEDI via subcutaneous injection once weekly. With repeat dosing, the mean percent decreases from baseline in serum TTR from Week 13 to Week 65 of treatment ranged from 68% to 74% (median range: 75% to 79%). Similar TTR reductions were observed regardless of TTR mutation, sex, age, or race. Serum TTR is a carrier of retinol-binding protein, which is involved in the transport of vitamin A in...