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Ionis has granted Sobi global exclusive rights of olezarsen excl. the US, Canada & China to treat familial chylomicronemia syndrome (FCS) & severely elevated triglycerides; Ionis to independently market Olezarsen in the US, whereas Theratechnologies holds the rights in Canada
As per the deal, Ionis will get an upfront, with potential milestone payments &…
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Ionis has granted Ono Pharma an exclusive global license to develop & commercialize sapablursen for the treatment of polycythemia vera (PV)
As per the deal, Ionis will get $280M upfront & ~$660M in development, regulatory & sales milestones, plus mid-teens net sales-based royalties as well as Ionis will be responsible to complete ongoing…
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The EC has approved Wainzua to treat ATTRv-PN (stage 1/2 polyneuropathy) in adults pts based on P-III (NEURO-TTRansform) trial assessing eplontersen vs external PBO over 66wks., with follow-up to Wk. 85 & an end-of-trial evaluation. Eligible pts could then enter an ongoing OLE study
Trial showed sustained benefits in co-1EPs of serum transthyretin…
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The US FDA has approved Tryngolza (80mg, QM, self-administered via an auto-injector) as an adj. for reducing triglycerides (TG) in FCS patients, based on a P-III (BALANCE) study. Further submission is under review in the EU, with more filings planned Â
The P-III study involved FCS patients with fasting TG levels of ≥880mg/dL. Trial showed…
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The EMA’s CHMP has granted positive opinions to 5 Biologics and 1 New Chemical Entity in October 2024, leading to treatments for patients and advances in the healthcare industry
The major highlighted drugs were Novo Nordisk’s Alhemo to treat Haemophilia A or B with inhibitors and AstraZeneca & Ionis’ Wainzua for Hereditary Transthyretin-Mediated Amyloidosis
PharmaShots has…
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The opinion of Wainzua for ATTRv-PN (stage 1/2 polyneuropathy) was supported by its P-III (NEURO-TTRansform) study vs external PBO for over wk.66, with a follow-up until wk.85 & an end-of-trial evaluation. Eligible patients could then enter an ongoing OLE study
Study depicted sustained benefits in co-1EPs of serum transthyretin (TTR) levels & neuropathy…
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The second quarter of 2023 highlights multiple clinical trial results, approvals, and numerous deals. There are major alliances in this quarter which include Merck acquired Prometheus for ~$10.8B and Astellas entered into a definitive agreement to acquire Iveric Bio for ~$5.9B
The second quarter of the year also showcases multiple approvals of the therapies…
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The US FDA approved 6 NDAs and 4 BLA in April 2023, leading to treatments for patients and advances in the healthcare industry. The CDER and CBER approved 36 novel products in 2023
In April 2023, the major highlights drugs were, Trikafta approval for children with cystic fibrosis, Qalsody for the treatment of amyotrophic…
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The life science industry saw high deal activity in 2021 staying in the range of an even busier 2020.
Fourth-quarter 2021 set the tone with Recursion’s development and commercialization deal with Roche and Genentech for a total value of $12.7B, followed by Poseida Therapeutics’ development and commercialization deal with Takeda for $3.73B.
This article is based on the 2021…

