Clinical Benefits of High-Dose Nusinersen Confirmed in DEVOTE Study
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 4d ago
0mins
Should l Buy IONS?
Source: Globenewswire
- Significant Clinical Outcomes: The DEVOTE study demonstrated that the high-dose nusinersen regimen of 50 mg and 28 mg significantly improved motor function in treatment-naïve and previously treated patients, with a mean difference of 26.19 points in CHOP-INTEND scores compared to the matched sham group, indicating its potential in clinical applications.
- Neurodegeneration Slowed: The high-dose regimen showed a more rapid reduction in neurofilament levels compared to the 12 mg regimen, highlighting its advantages in slowing the progression of spinal muscular atrophy (SMA) and potentially improving patients' quality of life.
- Good Safety and Tolerability: The safety profile of the high-dose regimen was broadly consistent with the 12 mg regimen, with common adverse events such as pneumonia and respiratory failure observed, yet the overall incidence of adverse events remained within acceptable limits, supporting its clinical feasibility.
- FDA Review Progress: The high-dose nusinersen is currently under review by the U.S. FDA, with a decision expected by April 3, 2026, which will provide SMA patients with a more effective treatment option and further solidify Biogen's leadership position in the biopharmaceutical sector.
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Analyst Views on IONS
Wall Street analysts forecast IONS stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for IONS is 92.67 USD with a low forecast of 65.00 USD and a high forecast of 110.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
14 Analyst Rating
13 Buy
1 Hold
0 Sell
Strong Buy
Current: 83.480
Low
65.00
Averages
92.67
High
110.00
Current: 83.480
Low
65.00
Averages
92.67
High
110.00
About IONS
Ionis Pharmaceuticals, Inc. develops six marketed medicines for serious diseases, including medicines for neurologic and cardiovascular diseases. Its marketed medicines consist of TRYNGOLZA, WAINUA (eplontersen), SPINRAZA (nusinersen), QALSODY (tofersen), TEGSEDI (inotersen) and WAYLIVRA (volanesorsen). TRYNGOLZA reduces triglyceride levels in adults with familial chylomicronemia syndrome (FCS). WAINUA is approved for the treatment of the polyneuropathy of hereditary transthyretin-medicated amyloidosis (ATTRv-PN) in adults. SPINRAZA is used for the treatment of pediatric and adult patients with spinal muscular atrophy. QALSODY is approved for the treatment of Amyotrophic Lateral Sclerosis in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene, or SOD1-ALS. TEGSEDI is approved for the treatment of ATTRv-PN in adults. WAYLIVRA is approved as an adjunct to diet in adult patients with genetically confirmed FCS and at high risk of acute, potentially fatal pancreatitis.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Clinical Benefits of High-Dose Nusinersen Confirmed in DEVOTE Study
- Significant Clinical Outcomes: The DEVOTE study demonstrated that the high-dose nusinersen regimen of 50 mg and 28 mg significantly improved motor function in treatment-naïve and previously treated patients, with a mean difference of 26.19 points in CHOP-INTEND scores compared to the matched sham group, indicating its potential in clinical applications.
- Neurodegeneration Slowed: The high-dose regimen showed a more rapid reduction in neurofilament levels compared to the 12 mg regimen, highlighting its advantages in slowing the progression of spinal muscular atrophy (SMA) and potentially improving patients' quality of life.
- Good Safety and Tolerability: The safety profile of the high-dose regimen was broadly consistent with the 12 mg regimen, with common adverse events such as pneumonia and respiratory failure observed, yet the overall incidence of adverse events remained within acceptable limits, supporting its clinical feasibility.
- FDA Review Progress: The high-dose nusinersen is currently under review by the U.S. FDA, with a decision expected by April 3, 2026, which will provide SMA patients with a more effective treatment option and further solidify Biogen's leadership position in the biopharmaceutical sector.
See More
Clinical Findings on High-Dose Nusinersen Published
- Clinical Study Results: Biogen's DEVOTE study results indicate that the high-dose nusinersen (50 mg and 28 mg) demonstrates significant safety and efficacy in spinal muscular atrophy (SMA) patients, particularly in treatment-naïve infants, with a mean difference of 26.19 points in motor function improvement, p<0.0001, highlighting its clinical potential.
- Neurodegeneration Biomarker Improvement: The high-dose regimen significantly accelerated the reduction of neurofilament, a marker of neurodegeneration, suggesting its advantage in slowing disease progression, which could reshape treatment strategies for SMA and enhance patient quality of life.
- FDA Review Progress: The high-dose nusinersen is currently under review by the U.S. FDA, with a decision expected by April 3, 2026, potentially providing patients with a more effective treatment option and further solidifying Biogen's leadership in the SMA treatment landscape.
- Positive Feedback from Diverse Patient Groups: In the open-label part of the DEVOTE study, 40 participants showed mean increases of 1.8 points on the Hammersmith Functional Motor Scale and 1.2 points on the Revised Upper Limb Module after transitioning to the high-dose regimen, demonstrating broad applicability across different ages and disease stages, boosting Biogen's confidence in future market opportunities.
See More
Ionis Pharmaceuticals Insider Sold Shares Worth $323,490, According to a Recent SEC Filing
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Ark Invest Unveils 2026 Big Ideas, Forecasts 1.9% GDP Growth Boost
- Technological Acceleration: Ark Invest's report highlights that capital investment in disruptive innovation platforms could contribute 1.9 percentage points to annualized real GDP growth during this decade, emphasizing the potential of technologies like robotaxis and next-gen data centers.
- Strong ETF Performance: In 2025, Ark's Autonomous Technology & Robotics ETF and Space & Defense Innovation ETF achieved returns of 49.8% and 49.2%, respectively, showcasing the firm's ability to generate returns in emerging technology sectors.
- Bitcoin Holdings Surge: The report indicates that U.S. ETFs and public companies now hold 12% of total Bitcoin supply, with Bitcoin ETF balances up 19.7% and public company Bitcoin holdings up 73% in 2025, reflecting strong market demand for digital assets.
- Diverse Investment Opportunities: Ark Invest identifies several publicly traded companies, including Coinbase, Illumina, and Waymo, as key investment opportunities for the 2026 themes, indicating the firm's forward-looking approach to diversified investment strategies.
See More
Ionis Pharmaceuticals and Otsuka Gain EU Approval for Dawnzera to Treat Hereditary Angioedema
- Drug Approval: Ionis Pharmaceuticals and Otsuka Pharmaceutical's RNA-targeted medicine, Dawnzera, has received European Commission approval for patients aged 12 and older to prevent recurrent hereditary angioedema attacks, marking a significant advancement in rare disease treatment.
- Clinical Data Support: The approval follows a positive opinion from the EU drug regulator's expert panel in November, demonstrating that Dawnzera significantly reduced the mean monthly HAE attack rate in the OASIS-HAE and OASISplus studies, thereby improving patient quality of life.
- Economic Benefits: Ionis is entitled to a $15 million milestone payment and up to 30% in tiered royalties on net product sales from Dawnzera, which is expected to contribute significantly to the company's revenue growth.
- Market Outlook: The launch of Dawnzera not only opens new opportunities for Ionis in the European market but also strengthens its position in the Asia-Pacific region, further advancing the company's strategic focus in rare disease therapies.
See More
Ionis Pharmaceuticals and Otsuka Get EU Approval for Dawnzera, Securing $15M Milestone Payment
- Drug Approval: Ionis Pharmaceuticals and Otsuka Pharmaceutical announced that the European Commission has approved Dawnzera for preventing recurrent hereditary angioedema (HAE) attacks in patients aged 12 and older, marking a significant advancement in the rare disease sector.
- Milestone Payment: This approval entitles Ionis to a $15 million milestone payment and tiered royalties of up to 30% on sales, significantly enhancing the company's financial position.
- Clinical Research Support: The approval is based on Phase 3 OASIS-HAE and OASISplus studies, which demonstrated that Dawnzera effectively reduces HAE attacks, further solidifying its market potential.
- Market Outlook: With Dawnzera already approved in the U.S. in August 2025, the exclusive rights in Europe and the Asia-Pacific region are expected to drive Ionis's growth in the global rare disease market, enhancing its competitive edge.
See More
Clinical Benefits of High-Dose Nusinersen Confirmed in DEVOTE Study
- Significant Clinical Outcomes: The DEVOTE study demonstrated that the high-dose nusinersen regimen of 50 mg and 28 mg significantly improved motor function in treatment-naïve and previously treated patients, with a mean difference of 26.19 points in CHOP-INTEND scores compared to the matched sham group, indicating its potential in clinical applications.
- Neurodegeneration Slowed: The high-dose regimen showed a more rapid reduction in neurofilament levels compared to the 12 mg regimen, highlighting its advantages in slowing the progression of spinal muscular atrophy (SMA) and potentially improving patients' quality of life.
- Good Safety and Tolerability: The safety profile of the high-dose regimen was broadly consistent with the 12 mg regimen, with common adverse events such as pneumonia and respiratory failure observed, yet the overall incidence of adverse events remained within acceptable limits, supporting its clinical feasibility.
- FDA Review Progress: The high-dose nusinersen is currently under review by the U.S. FDA, with a decision expected by April 3, 2026, which will provide SMA patients with a more effective treatment option and further solidify Biogen's leadership position in the biopharmaceutical sector.
See More
Clinical Findings on High-Dose Nusinersen Published
- Clinical Study Results: Biogen's DEVOTE study results indicate that the high-dose nusinersen (50 mg and 28 mg) demonstrates significant safety and efficacy in spinal muscular atrophy (SMA) patients, particularly in treatment-naïve infants, with a mean difference of 26.19 points in motor function improvement, p<0.0001, highlighting its clinical potential.
- Neurodegeneration Biomarker Improvement: The high-dose regimen significantly accelerated the reduction of neurofilament, a marker of neurodegeneration, suggesting its advantage in slowing disease progression, which could reshape treatment strategies for SMA and enhance patient quality of life.
- FDA Review Progress: The high-dose nusinersen is currently under review by the U.S. FDA, with a decision expected by April 3, 2026, potentially providing patients with a more effective treatment option and further solidifying Biogen's leadership in the SMA treatment landscape.
- Positive Feedback from Diverse Patient Groups: In the open-label part of the DEVOTE study, 40 participants showed mean increases of 1.8 points on the Hammersmith Functional Motor Scale and 1.2 points on the Revised Upper Limb Module after transitioning to the high-dose regimen, demonstrating broad applicability across different ages and disease stages, boosting Biogen's confidence in future market opportunities.
See More
Ionis Pharmaceuticals Insider Sold Shares Worth $323,490, According to a Recent SEC Filing
See More
Ark Invest Unveils 2026 Big Ideas, Forecasts 1.9% GDP Growth Boost
- Technological Acceleration: Ark Invest's report highlights that capital investment in disruptive innovation platforms could contribute 1.9 percentage points to annualized real GDP growth during this decade, emphasizing the potential of technologies like robotaxis and next-gen data centers.
- Strong ETF Performance: In 2025, Ark's Autonomous Technology & Robotics ETF and Space & Defense Innovation ETF achieved returns of 49.8% and 49.2%, respectively, showcasing the firm's ability to generate returns in emerging technology sectors.
- Bitcoin Holdings Surge: The report indicates that U.S. ETFs and public companies now hold 12% of total Bitcoin supply, with Bitcoin ETF balances up 19.7% and public company Bitcoin holdings up 73% in 2025, reflecting strong market demand for digital assets.
- Diverse Investment Opportunities: Ark Invest identifies several publicly traded companies, including Coinbase, Illumina, and Waymo, as key investment opportunities for the 2026 themes, indicating the firm's forward-looking approach to diversified investment strategies.
See More
Ionis Pharmaceuticals and Otsuka Gain EU Approval for Dawnzera to Treat Hereditary Angioedema
- Drug Approval: Ionis Pharmaceuticals and Otsuka Pharmaceutical's RNA-targeted medicine, Dawnzera, has received European Commission approval for patients aged 12 and older to prevent recurrent hereditary angioedema attacks, marking a significant advancement in rare disease treatment.
- Clinical Data Support: The approval follows a positive opinion from the EU drug regulator's expert panel in November, demonstrating that Dawnzera significantly reduced the mean monthly HAE attack rate in the OASIS-HAE and OASISplus studies, thereby improving patient quality of life.
- Economic Benefits: Ionis is entitled to a $15 million milestone payment and up to 30% in tiered royalties on net product sales from Dawnzera, which is expected to contribute significantly to the company's revenue growth.
- Market Outlook: The launch of Dawnzera not only opens new opportunities for Ionis in the European market but also strengthens its position in the Asia-Pacific region, further advancing the company's strategic focus in rare disease therapies.
See More
Ionis Pharmaceuticals and Otsuka Get EU Approval for Dawnzera, Securing $15M Milestone Payment
- Drug Approval: Ionis Pharmaceuticals and Otsuka Pharmaceutical announced that the European Commission has approved Dawnzera for preventing recurrent hereditary angioedema (HAE) attacks in patients aged 12 and older, marking a significant advancement in the rare disease sector.
- Milestone Payment: This approval entitles Ionis to a $15 million milestone payment and tiered royalties of up to 30% on sales, significantly enhancing the company's financial position.
- Clinical Research Support: The approval is based on Phase 3 OASIS-HAE and OASISplus studies, which demonstrated that Dawnzera effectively reduces HAE attacks, further solidifying its market potential.
- Market Outlook: With Dawnzera already approved in the U.S. in August 2025, the exclusive rights in Europe and the Asia-Pacific region are expected to drive Ionis's growth in the global rare disease market, enhancing its competitive edge.
See More
