Date: 2018-08-02
Type of information: Commercialisation agreement
Compound: Tegsedi™ (inotersen) and Waylivra™ (volanesorsen)
Company: Akcea Therapeutics (USA - CA), a subsidiary of Ionis Pharmaceuticals (USA - CA) PTC Therapeutics (USA - NJ)
Therapeutic area: Rare diseases - Genetic diseases
Type agreement: commercialisation
Action mechanism:
- antisense drug/antisense oligonucleotide. Inotersen is an antisense oligonucleotide designed to reduce the production of transthyretin to treat patients with TTR amyloidosis (ATTR), a severe, rare and fatal disease. In patients with ATTR, both the mutant and wild-type (wt) TTR builds up as fibrils in tissues, such as the peripheral nerves, heart, gastrointestinal system, eyes, kidneys, central nervous system, thyroid and bone marrow. The presence of TTR fibrils interferes with the normal functions of these tissues. As the TTR protein fibrils enlarge, more tissue damage occurs and the disease worsens, resulting in poor quality of life and eventually death.
- Tegsedi™ has received marketing authorization approval from the European Commission (EC) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). The drug is also the subject of a pending new drug application in the U.S. and Canada. Tegsedi™ has a PDUFA date of October 6, 2018.
- antisense oligonucleotide. Volanesorsen (phosphorothioate oligonucleotide targeted to apolipoprotein C-III (ISIS-APOCIIIRxSIS-APOCIIIRx) is an antisense drug that targets apoC-III, a protein produced in the liver that plays a central role in the regulation of serum triglycerides.
- The drug is under regulatory review in the U.S., Europe and Canada for the treatment of people with familial chylomicronemia syndrome (FCS). Waylivra™ recently received a positive vote from the FDA'sDivision of Metabolism and Endocrinology Products Advisory Committee and has a PDUFA date of August 30, 2018. Waylivra™ is also in clinical development for Familial Partial Lipodystrophy, or FPL.
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Disease: hereditary transthyretin amyloidosis (hATTR), familial chylomicronemia syndrome (FCS)
Details:
- • On August 2, 2018, PTC Therapeutics and Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, announced a collaboration under which PTC will commercialize two of Akcea's rare disease drugs in Latin America (LATAM): Tegsedi™ (inotersen) and Waylivra™ (volanesorsen).
Financial terms:
- Under the agreement, PTC will gain exclusive rights to Tegsedi™ (inotersen) and Waylivra™ (volanesorsen) in Latin America and certain Caribbean countries. PTC will pay Akcea an upfront licensing fee of $18 million, $12 million which is due on signing and $6 million which will be paid on the earlier of FDA or EMA approval of Waylivra™. In addition, PTC will pay Akcea regulatory milestones in the PTC territory up to a total of $8 million. Akcea is also eligible to receive royalties from PTC in the mid-twenty percent range on net sales of each drug in the PTC territory. PTC's obligation to pay Akcea royalties begins on the earlier of 12 months after the first commercial sale of a product in Brazil or the date that PTC recognizes revenue of at least $10 million in LATAM.
- The collaboration will be governed by a joint steering committee with representation from both parties. Akcea gained the global rights to Tegsedi™ and Waylivra™ through licenses from Ionis. Milestone payments and royalties that Akcea receives from PTC for Tegsedi™ will be split 60% to Ionis and 40% to Akcea. All Waylivra™ milestone payments and royalties that Akcea receives from PTC will be split 50/50 with Ionis.
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