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Ionis (IONS) Up 6% on Detailed Data From Angelman Drug Study
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Ionis Pharmaceuticals (IONS - Free Report) reported detailed results from the phase I/IIa HALOS study evaluating its investigational antisense drug ION582 in patients with a rare genetic disorder called Angelman Syndrome (AS) that causes developmental disabilities and nerve-related symptoms.
Data from the HALOS study showed that treatment with ION582 demonstrated consistent and encouraging clinical improvement on measures assessing all functional domains, including communication, cognition and motor function. These improvements were observed by the study investigators using multiple assessment tools that use inputs from clinicians and parents.
Though Ionis had already reported these results in May, this time it provided numerical data backing them up. Per management, 97% of study participants who received medium and high doses of the drug achieved improvement in overall AS symptoms. This was measured using the Symptoms of Angelman Syndrome–Clinician Global Impression-Change (SAS-CGI-C) scale, which evaluates clinicians' impressions of AS symptoms in study participants.
Another scale called Bayley-4 showed that the improvements in communication, cognition and motor function exceeded those observed in the AS natural history studies. Overall, the drug was also safe and well tolerated in study participants.
Based on these results, Ionis intends to start a pivotal late-stage study on the AS drug in the first half of 2025. In this regard, it plans to meet with the regulatory authorities to discuss the study design.
Currently, there are no approved therapies for AS. The only other company close to initiating a late-stage study in AS is Ultragenyx Pharmaceutical (RARE - Free Report) . It recently completed an end-of-phase II meeting with the FDA for its own AS drug GTX-102. Some Wall Street analysts acknowledged that the results put Ionis close to Ultragenyx, which plans to start a pivotal late-stage study on GTX-12 by this year’s end.
Shares of Ionis were up nearly 6% on Monday, likely due to Ionis closing in toward its rival in AS drug development. The Ultragenyx drug is based on a mechanism that is similar to ION582.
Year to date, Ionis’ stock has registered breakeven growth compared with the industry’s 2.7% fall.
Image Source: Zacks Investment Research
Notably, Ionis’ long-time collaboration partner Biogen (BIIB - Free Report) had decided to opt out of licensing the rights to develop ION582 in May. However, Biogen did not specify any rationale for this decision.
Ionis is now developing the AS drug as part of its wholly-owned pipeline drugs, along with several other candidates that have shown encouraging progress in clinical studies.
Last month, the FDA accepted the company’s regulatory filing seeking approval for olezarsen to treat familial chylomicronemia syndrome (FCS). This filing is based on results from the phase III BALANCE study, which showed that treatment with olezarsen led to significant triglyceride-lowering and substantial reductions in acute pancreatitis attacks in FCS patients. If approved, olezarsen will be Ionis’ first medicine that will be launched independently. A final decision from the FDA is expected before Dec 19, 2024.
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).
In May, 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 partnered with Otsuka Pharmaceutical to market this drug across the Asia-Pacific region and Europe.
Image: Bigstock
Ionis (IONS) Up 6% on Detailed Data From Angelman Drug Study
Ionis Pharmaceuticals (IONS - Free Report) reported detailed results from the phase I/IIa HALOS study evaluating its investigational antisense drug ION582 in patients with a rare genetic disorder called Angelman Syndrome (AS) that causes developmental disabilities and nerve-related symptoms.
Data from the HALOS study showed that treatment with ION582 demonstrated consistent and encouraging clinical improvement on measures assessing all functional domains, including communication, cognition and motor function. These improvements were observed by the study investigators using multiple assessment tools that use inputs from clinicians and parents.
Though Ionis had already reported these results in May, this time it provided numerical data backing them up. Per management, 97% of study participants who received medium and high doses of the drug achieved improvement in overall AS symptoms. This was measured using the Symptoms of Angelman Syndrome–Clinician Global Impression-Change (SAS-CGI-C) scale, which evaluates clinicians' impressions of AS symptoms in study participants.
Another scale called Bayley-4 showed that the improvements in communication, cognition and motor function exceeded those observed in the AS natural history studies. Overall, the drug was also safe and well tolerated in study participants.
Based on these results, Ionis intends to start a pivotal late-stage study on the AS drug in the first half of 2025. In this regard, it plans to meet with the regulatory authorities to discuss the study design.
Currently, there are no approved therapies for AS. The only other company close to initiating a late-stage study in AS is Ultragenyx Pharmaceutical (RARE - Free Report) . It recently completed an end-of-phase II meeting with the FDA for its own AS drug GTX-102. Some Wall Street analysts acknowledged that the results put Ionis close to Ultragenyx, which plans to start a pivotal late-stage study on GTX-12 by this year’s end.
Shares of Ionis were up nearly 6% on Monday, likely due to Ionis closing in toward its rival in AS drug development. The Ultragenyx drug is based on a mechanism that is similar to ION582.
Year to date, Ionis’ stock has registered breakeven growth compared with the industry’s 2.7% fall.
Image Source: Zacks Investment Research
Notably, Ionis’ long-time collaboration partner Biogen (BIIB - Free Report) had decided to opt out of licensing the rights to develop ION582 in May. However, Biogen did not specify any rationale for this decision.
Ionis is now developing the AS drug as part of its wholly-owned pipeline drugs, along with several other candidates that have shown encouraging progress in clinical studies.
Last month, the FDA accepted the company’s regulatory filing seeking approval for olezarsen to treat familial chylomicronemia syndrome (FCS). This filing is based on results from the phase III BALANCE study, which showed that treatment with olezarsen led to significant triglyceride-lowering and substantial reductions in acute pancreatitis attacks in FCS patients. If approved, olezarsen will be Ionis’ first medicine that will be launched independently. A final decision from the FDA is expected before Dec 19, 2024.
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).
In May, 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 partnered with Otsuka Pharmaceutical to market this drug across the Asia-Pacific region and Europe.
Ionis Pharmaceuticals, Inc. Price
Ionis Pharmaceuticals, Inc. price | Ionis Pharmaceuticals, Inc. Quote
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.