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Ionis (IONS) Completes Enrolment for Rare Disease Drug Study
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Ionis Pharmaceuticals (IONS - Free Report) announced full enrolment in a pivotal phase III study evaluating its investigational RNA-targeted therapy zilganersen for treating Alexander disease (AxD), a rare neurological condition.
Top-line data from the pivotal study is expected in the second half of 2025.
Though IONS did not specify the total number of patients enrolled in the study, a government website states that the study has a target enrolment of nearly 73 study participants aged between two to 65 with AxD across eight countries.
The study will randomize participants in a 2:1 ratio to receive zilganersen or placebo over a 60-week double-blind treatment period. After the 60-week double-blind treatment period, all study participants will receive zilganersen over a 180-week open-label treatment period followed by a 28-week post-treatment follow-up period.
The primary endpoint of this pivotal study is the percent change from baseline in gait speed as assessed by the 10-Meter Walk Test (10MWT) at the end of the 60-week treatment period.
Per Ionis, zilganersen is the first investigational medicine in clinical development for AxD patients. Management also claims that the pivotal study is the first one to address the underlying cause of AxD.
AxD is a rare neurological disease that affects a type of cell in the brain called astrocytes, which have multiple roles in the brain to support neurons and oligodendrocytes. People living with this condition are marked by cognitive dysfunction and progressive neurologic deterioration, including swallowing and the ability to control muscles for large movements. There are currently no disease-modifying medicines approved for AxD patients.
Zilganersen 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 be the first FDA-approved treatment for FCS.
Year to date, the stock has lost 6.4% compared with the industry’s 4.0% fall.
Image Source: Zacks Investment Research
Currently, Ionis’s pipeline consists of partnered and wholly-owned candidates. The company has partnerships with big pharma companies like AstraZeneca (AZN - Free Report) , Biogen (BIIB - Free Report) and Novartis (NVS - Free Report) . Its 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.
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.
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).
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 has partnered with Otsuka Pharmaceutical to market this drug across the Asia-Pacific region and Europe.
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.
Image: Bigstock
Ionis (IONS) Completes Enrolment for Rare Disease Drug Study
Ionis Pharmaceuticals (IONS - Free Report) announced full enrolment in a pivotal phase III study evaluating its investigational RNA-targeted therapy zilganersen for treating Alexander disease (AxD), a rare neurological condition.
Top-line data from the pivotal study is expected in the second half of 2025.
Though IONS did not specify the total number of patients enrolled in the study, a government website states that the study has a target enrolment of nearly 73 study participants aged between two to 65 with AxD across eight countries.
The study will randomize participants in a 2:1 ratio to receive zilganersen or placebo over a 60-week double-blind treatment period. After the 60-week double-blind treatment period, all study participants will receive zilganersen over a 180-week open-label treatment period followed by a 28-week post-treatment follow-up period.
The primary endpoint of this pivotal study is the percent change from baseline in gait speed as assessed by the 10-Meter Walk Test (10MWT) at the end of the 60-week treatment period.
Per Ionis, zilganersen is the first investigational medicine in clinical development for AxD patients. Management also claims that the pivotal study is the first one to address the underlying cause of AxD.
AxD is a rare neurological disease that affects a type of cell in the brain called astrocytes, which have multiple roles in the brain to support neurons and oligodendrocytes. People living with this condition are marked by cognitive dysfunction and progressive neurologic deterioration, including swallowing and the ability to control muscles for large movements. There are currently no disease-modifying medicines approved for AxD patients.
Zilganersen 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 be the first FDA-approved treatment for FCS.
Year to date, the stock has lost 6.4% compared with the industry’s 4.0% fall.
Image Source: Zacks Investment Research
Currently, Ionis’s pipeline consists of partnered and wholly-owned candidates. The company has partnerships with big pharma companies like AstraZeneca (AZN - Free Report) , Biogen (BIIB - Free Report) and Novartis (NVS - Free Report) . Its 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.
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.
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).
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 has partnered with Otsuka Pharmaceutical to market this drug across the Asia-Pacific region and Europe.
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.
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.