Ionis (IONS) Meets Rare Disease Drug Study Goal, Stock Up 8.5%
Ionis Pharmaceuticals IONS announced positive top-line results from the phase III BALANCE study on its investigational LICA drug olezarsen people with familial chylomicronemia syndrome (“FCS”).
The BALANCE study achieved its primary endpoint of statistically significant reduction in triglyceride (“TG”) levels at six months with an 80mg dose of olezarsen. Though Ionis did not share any numerical data/figures from the study, it did mention that study participants treated with the drug showed a 100% reduction in acute pancreatitis (inflammation of the pancreas), a key secondary endpoint.
Data from the study also demonstrated a dose-dependent effect following treatment with olezarsen. The study also evaluated a 50mg dose of the drug. Though treatment with this dosage level also substantially reduced pancreatitis, it did not achieve the BALANCE study’s primary endpoint.
Based on these results, Ionis plans to file a new drug application (“NDA”) for the drug with the FDA early next year.
A rare genetic disorder, FCS is marked by the body’s inability to break down fats and is marked by extremely high TG levels. People living with FCS are at high risk of acute pancreatitis and other chronic health issues such as fatigue and severe, recurrent abdominal pain. Per Ionis, FCS is estimated to impact 1-2 people per million worldwide.
Currently, there are no FDA-approved therapies for the treatment of FCS. Hence, people living with FCS maintain an extremely restrictive diet of less than 20 grams of fat per day. Investors were likely impressed with the results despite the lack of numerical data. Moreover, if the NDA were to be filed, the FDA would expedite the filing’s review as olezarsen was granted fast-track designation in FCS indication earlier this year in January.
Also, the above encouraging results from the BALANCE study highlight an important milestone for Ionis towards its goal of launching its first commercial product. Currently, IONS earns all its commercial revenues primarily through royalty payments on net sales of Biogen BIIB-marketed drug Spinraza and R&D revenues from partnered medicines. The company has yet to generate revenues from product sales.
Shares of Ionis were up 8.5% on Tuesday as investors anticipate a potential approval for olezarsen in FCS. In the year so far, the stock price has increased 22.5% against the industry’s 8.2% fall.
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Apart from FCS, Ionis is also evaluating olezarsen in three ongoing phase III studies – CORE, CORE2 and ESSENCE – as a potential treatment for severe hypertriglyceridemia (“SHTG”).
Ionis’ pipeline consists of both wholly owned as well as partnered pipelines. The company partners with many pharma giants like AstraZeneca AZN, Biogen, and Novartis NVS.
Biogen, AstraZeneca and Novartis are Ionis’ partners for tofersen, eplontersen and pelacarsen, respectively. Last December, AstraZeneca filed an NDA with the FDA seeking approval of eplontersen for polyneuropathy caused by hereditary TTR amyloidosis (ATTRv-PN). A final decision on the NDA is expected before this year’s end. Apart from ATTRv-PN, Ionis and AstraZeneca also evaluate eplontersen as a potential treatment for amyloid transthyretin cardiomyopathy (ATTR-CM) in the phase III CARDIO-TTRansform study.
Biogen and Ionis are collaborating on developing advanced treatments for neurological disorders. Ionis has licensed Spinraza to Biogen, which is approved for treating spinal muscular atrophy in pediatric and adult patients. While Biogen is responsible for commercializing Spinraza worldwide, Ionis receives royalties on Spinraza’s sales.
The company is developing another pipeline candidate, pelacarsen, in collaboration with Novartis. Ionis and Novartis are evaluating pelacarsen in the ongoing phase III cardiovascular outcome study, HORIZON, in patients with established cardiovascular disease and elevated lipoprotein(a) or Lp(a). Novartis is responsible for leading the candidate's global development and commercialization activities.
Zacks Rank
Ionis currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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