FDA Accepts Ionis' (IONS) NDA for Rare Disease Drug Olezarsen
Ionis Pharmaceuticals IONS announced that the FDA accepted the new drug application (NDA), seeking approval for its investigational RNA-targeted therapy olezarsen as a potential treatment for adults with familial chylomicronemia syndrome (FCS).
The FDA granted priority review to this NDA. A filing designated as a priority review reduces the review period by four months. A final decision is expected by Dec 19, 2024. Management has also stated that the FDA does not plan to hold an advisory committee meeting for Ionis’ NDA filing.
The NDA is supported by results from the phase III BALANCE study, where FCS patients treated with olezarsen achieved significant triglyceride-lowering and substantial reductions in acute pancreatitis attacks in FCS patients. Management is also planning to submit a similar regulatory filing for olezarsen with the EMA later this year.
FCS is a rare genetic disease marked by extremely elevated triglyceride levels. People living with this condition are at high risk of acute pancreatitis in addition to other chronic health issues such as fatigue and severe, recurrent abdominal pain.
Olezarsen is one of Ionis’ wholly-owned pipeline candidates, which it intends to launch independently in the United States. The drug’s commercial launch is part of management's broad strategy to deliver a steady flow of wholly-owned medicines to patients. If approved, the drug will not only be Ionis’ first independent product launch but also the first FDA-approved treatment for FCS.
In the past two years, Ionis has secured fast-track, orphan drug, and breakthrough therapy designations from the FDA for olezarsen in treating FCS. These designations have helped accelerate the drug’s clinical development and also provide Ionis with incentives, which include a potential seven years of market exclusivity after receiving the agency’s approval. Shares of Ionis rose over 5% on Tuesday likely due to these factors.
Year to date, the stock has lost 7.1% compared with the industry’s 7.8% fall.
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Alongside the NDA acceptance announcement, management also reported that it had completed enrolment in all three late-stage studies — CORE, CORE2, and ESSENCE — which are evaluating olezarsen for the treatment of severe hypertriglyceridemia (sHTG). Data from these studies are expected in the second half of 2025. Like FCS, Ionis also has a first-mover advantage in sHTG indication.
Ionis’ progress with the development and potential commercialization of its wholly-owned pipeline has been encouraging. Another wholly-owned candidate in the company’s pipeline is donidalorsen, which is evaluated as a potential treatment for a rare genetic disease called hereditary angioedema (HAE).
Last month, Ionis reported positive data from two phase III studies — OASIS-HAE and OASISplus — evaluating donidalorsen in patients aged 12 years and older with HAE. Data from these studies showed that treatment with the drug achieved significant and sustained reductions in mean monthly HAE attack rates and continued attack rate improvement of more than 90% with one year of treatment for monthly or every two-month dosing.
Based on the above results, Ionis is preparing to file an NDA for donidalorsen with the FDA later this year. The company has partnered with Otsuka Pharmaceutical to market this drug across the Asia-Pacific region and Europe.
Ionis’s pipeline also consists of partnered candidates. The company has partnerships with big pharma companies like AstraZeneca AZN, Biogen BIIB and Novartis NVS. The company’s collaboration with these drugmakers provides it with funds in the form of license fees, upfront payments, and milestone payments to invest in its internal pipeline development.
Biogen, AstraZeneca, and Novartis are Ionis’ partners for Qalsody (tofersen), Wainua (eplontersen), and pelacarsen, respectively. The FDA approved Wainua last year in December to treat adults with polyneuropathy caused by hereditary TTR amyloidosis (ATTRv-PN). Ionis and AstraZeneca are also working on expanding the drug’s label in other forms of amyloidosis. The companies are currently evaluating the drug in the phase III CARDIO-TTRANSform study to treat cardiomyopathy caused by hATTR amyloidosis (ATTR-CM). This indication has a larger market than ATTRv-PN. Data from this study is expected in the first half of 2025.
Biogen and Ionis are developing advanced treatments for neurological disorders. Ionis licensed Spinraza to Biogen, which is approved for treating spinal muscular atrophy in pediatric and adult patients. While Biogen is responsible for marketing Spinraza worldwide, Ionis receives royalties on Spinraza’s sales. Both companies have also developed Qalsody, which received FDA approval last year to treat amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) mutations.
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.
Ionis Pharmaceuticals, Inc. Price
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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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