Ionis and GSK's New Drug Application Receives FDA Priority Review
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Apr 28 2026
0mins
Source: Newsfilter
- Breakthrough Therapy Designation: The FDA has accepted the New Drug Application for bepirovirsen and granted it Breakthrough Therapy and Priority Review designations, indicating its potential for significant improvement in chronic hepatitis B treatment, with a PDUFA date set for October 26, 2026.
- Clinical Trial Results: In the Phase 3 B-Well 1 and B-Well 2 trials, bepirovirsen demonstrated statistically significant functional cure rates, particularly in patients with lower baseline HBsAg levels, highlighting its efficacy and safety in chronic hepatitis B patients.
- Significant Market Need: With over 250 million people affected by chronic hepatitis B globally and 1.7 million in the U.S. requiring lifelong antiviral therapy, the success of bepirovirsen could greatly enhance patient quality of life and reduce liver cancer risk.
- Strategic Partnership: Ionis's collaboration with GSK began in 2019, providing Ionis with upfront and milestone payments, along with a 10-12% royalty on sales, showcasing the commercial potential of this new drug development.
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Analyst Views on IONS
Wall Street analysts forecast IONS stock price to rise
14 Analyst Rating
13 Buy
1 Hold
0 Sell
Strong Buy
Current: 77.330
Low
65.00
Averages
92.67
High
110.00
Current: 77.330
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.
Bepirovirsen Shows Significant Efficacy in Chronic Hepatitis B Trials
- Functional Cure Breakthrough: In the pivotal B-Well 1 and B-Well 2 trials, bepirovirsen achieved a 19% functional cure rate overall and 26% in patients with lower viral activity, significantly outperforming the 0% rate of standard care, providing new hope for chronic hepatitis B patients.
- Significant Treatment Outcomes: Notably, 49% of bepirovirsen recipients achieved a hepatitis B surface antigen level of ≤100 IU/mL one year post-treatment, indicating enhanced immune control associated with a significant reduction in liver cancer risk, potentially improving patient outcomes.
- Global Impact: With over 240 million people affected by chronic hepatitis B, the successful development of bepirovirsen could transform treatment paradigms for this large patient population, reducing reliance on lifelong antiviral therapy and improving quality of life.
- Rapid Regulatory Progress: Bepirovirsen is currently under priority review by the FDA with Breakthrough and Fast Track Designations, with the first regulatory decisions anticipated in Q3 2026, highlighting its potential market value and prospects.
See More
GSK's Antisense Drug Shows Promise in Hepatitis B Trials
- Clinical Trial Success: GSK's antisense drug bepirovirsen, developed with Ionis Pharma, achieved a 19% functional cure rate in two late-stage trials, significantly surpassing the current standard treatment's less than 1% cure rate, marking a new hope for chronic hepatitis B treatment.
- Significant Treatment Effects: In the B-Well 1 and B-Well 2 Phase 3 trials, six months of bepirovirsen therapy resulted in undetectable hepatitis B virus DNA and surface antigens for at least six months post-therapy, demonstrating clinically meaningful efficacy.
- Good Safety Profile: Conducted across 29 countries with chronic hepatitis patients, the studies indicated that bepirovirsen has an acceptable safety and tolerability profile consistent with previous trials, laying a solid foundation for its future market application.
- Long-term Impact Potential: GSK's science chief Tony Wood emphasized that bepirovirsen not only offers the potential for significantly improved functional cure rates but also may reduce the risk of long-term liver complications, including cancer, indicating a major advancement in chronic hepatitis B treatment.
See More
Citi Initiates Bullish Coverage on SMid Cap Biotechs
- Analyst Rating Upgrade: Citi analyst Eric Joseph initiated Buy ratings on five biotech companies including Ionis (IONS), Alnylam (ALNY), and BioMarin (BMRN), reflecting a positive outlook for significant growth in the cardiovascular, metabolism, and endocrine sectors, which indicates strong confidence in the biotech industry.
- Ionis Price Target: The analyst set a $115 price target for Ionis, emphasizing that its pipeline based on antisense oligonucleotides provides a solid foundation for durable growth, showcasing confidence in its future performance.
- BioMarin Valuation Discount: BioMarin received a $75 price target, with analysts noting its EV/Rev ratio of 4x is significantly lower than peers' 8-9x, indicating a substantial valuation discount that may attract investor interest.
- Alnylam Investment Opportunity: Alnylam's price target is set at $380, with analysts arguing that despite its year-to-date underperformance, the impressive market launch of its heart disease therapy Amvuttra presents an attractive buying opportunity, signaling potential for future growth.
See More
Biogen's Alzheimer Therapy Trial Shows Promising Results
- Clinical Trial Results: Biogen's Phase 2 CELIA study indicates that diranersen, an experimental therapy for Alzheimer's disease, slowed clinical decline across all doses in early-stage patients, highlighting the drug's potential in treating Alzheimer's.
- Biomarker Impact: The study demonstrated significant reductions in tau pathology associated with Alzheimer's, providing the first evidence from a randomized Phase 2 trial of a tau-directed therapy showing robust biomarker impact and cognitive benefits.
- Safety and Tolerability: In the 416-patient trial, diranersen's safety and tolerability profile was consistent with data from a Phase 1b trial, reinforcing confidence in its safety characteristics for further development.
- Registration Development Outlook: Biogen expressed that the Phase 2 data gives them the confidence to advance diranersen into registrational development, indicating the drug's promising future in the Alzheimer's treatment landscape.
See More
Ionis Pharmaceuticals (IONS.US) Director Plans to Sell $4.35 Million in Common Stock via Form 144
Stock Sale Announcement: Ionis Pharmaceuticals' director, Joseph Loscalzo, plans to sell 54,321 shares of the company's common stock on May 4, with an estimated market value of around $4.35 million.
Reduction in Shareholding: Loscalzo has decreased his shareholding in Ionis Pharmaceuticals by 1,032 shares since March 3, 2026, with a total value of approximately $82,833.
See More
Ionis Pharmaceuticals to Participate in Multiple Investor Conferences
- Investor Conference Schedule: Ionis Pharmaceuticals announced participation in the Bank of America Securities 2026 Health Care Conference on May 12, 2026, the RBC Capital Markets Global Healthcare Conference on May 19, 2026, and the Goldman Sachs 47th Annual Global Healthcare Conference on June 10, 2026, showcasing its innovations in RNA-targeted medicines.
- Live Webcast and Replays: Presentations will be accessible via a live webcast on the Investors & Media section of the Ionis website, with replays available within 48 hours post-event, ensuring investors stay updated on the company's latest developments.
- Company Background: For three decades, Ionis has focused on inventing medicines that improve the futures of patients with serious diseases, currently boasting marketed medicines and a leading pipeline in neurology, cardiometabolic diseases, and areas of high patient need, reflecting its deep expertise in the biopharmaceutical sector.
- Innovation-Driven Technology: As a pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies while advancing new approaches in gene editing, demonstrating a profound understanding of disease biology and industry-leading technology aimed at delivering life-changing treatments for patients.
See More
Bepirovirsen Shows Significant Efficacy in Chronic Hepatitis B Trials
- Functional Cure Breakthrough: In the pivotal B-Well 1 and B-Well 2 trials, bepirovirsen achieved a 19% functional cure rate overall and 26% in patients with lower viral activity, significantly outperforming the 0% rate of standard care, providing new hope for chronic hepatitis B patients.
- Significant Treatment Outcomes: Notably, 49% of bepirovirsen recipients achieved a hepatitis B surface antigen level of ≤100 IU/mL one year post-treatment, indicating enhanced immune control associated with a significant reduction in liver cancer risk, potentially improving patient outcomes.
- Global Impact: With over 240 million people affected by chronic hepatitis B, the successful development of bepirovirsen could transform treatment paradigms for this large patient population, reducing reliance on lifelong antiviral therapy and improving quality of life.
- Rapid Regulatory Progress: Bepirovirsen is currently under priority review by the FDA with Breakthrough and Fast Track Designations, with the first regulatory decisions anticipated in Q3 2026, highlighting its potential market value and prospects.
See More
GSK's Antisense Drug Shows Promise in Hepatitis B Trials
- Clinical Trial Success: GSK's antisense drug bepirovirsen, developed with Ionis Pharma, achieved a 19% functional cure rate in two late-stage trials, significantly surpassing the current standard treatment's less than 1% cure rate, marking a new hope for chronic hepatitis B treatment.
- Significant Treatment Effects: In the B-Well 1 and B-Well 2 Phase 3 trials, six months of bepirovirsen therapy resulted in undetectable hepatitis B virus DNA and surface antigens for at least six months post-therapy, demonstrating clinically meaningful efficacy.
- Good Safety Profile: Conducted across 29 countries with chronic hepatitis patients, the studies indicated that bepirovirsen has an acceptable safety and tolerability profile consistent with previous trials, laying a solid foundation for its future market application.
- Long-term Impact Potential: GSK's science chief Tony Wood emphasized that bepirovirsen not only offers the potential for significantly improved functional cure rates but also may reduce the risk of long-term liver complications, including cancer, indicating a major advancement in chronic hepatitis B treatment.
See More
Citi Initiates Bullish Coverage on SMid Cap Biotechs
- Analyst Rating Upgrade: Citi analyst Eric Joseph initiated Buy ratings on five biotech companies including Ionis (IONS), Alnylam (ALNY), and BioMarin (BMRN), reflecting a positive outlook for significant growth in the cardiovascular, metabolism, and endocrine sectors, which indicates strong confidence in the biotech industry.
- Ionis Price Target: The analyst set a $115 price target for Ionis, emphasizing that its pipeline based on antisense oligonucleotides provides a solid foundation for durable growth, showcasing confidence in its future performance.
- BioMarin Valuation Discount: BioMarin received a $75 price target, with analysts noting its EV/Rev ratio of 4x is significantly lower than peers' 8-9x, indicating a substantial valuation discount that may attract investor interest.
- Alnylam Investment Opportunity: Alnylam's price target is set at $380, with analysts arguing that despite its year-to-date underperformance, the impressive market launch of its heart disease therapy Amvuttra presents an attractive buying opportunity, signaling potential for future growth.
See More
Biogen's Alzheimer Therapy Trial Shows Promising Results
- Clinical Trial Results: Biogen's Phase 2 CELIA study indicates that diranersen, an experimental therapy for Alzheimer's disease, slowed clinical decline across all doses in early-stage patients, highlighting the drug's potential in treating Alzheimer's.
- Biomarker Impact: The study demonstrated significant reductions in tau pathology associated with Alzheimer's, providing the first evidence from a randomized Phase 2 trial of a tau-directed therapy showing robust biomarker impact and cognitive benefits.
- Safety and Tolerability: In the 416-patient trial, diranersen's safety and tolerability profile was consistent with data from a Phase 1b trial, reinforcing confidence in its safety characteristics for further development.
- Registration Development Outlook: Biogen expressed that the Phase 2 data gives them the confidence to advance diranersen into registrational development, indicating the drug's promising future in the Alzheimer's treatment landscape.
See More
Ionis Pharmaceuticals (IONS.US) Director Plans to Sell $4.35 Million in Common Stock via Form 144
Stock Sale Announcement: Ionis Pharmaceuticals' director, Joseph Loscalzo, plans to sell 54,321 shares of the company's common stock on May 4, with an estimated market value of around $4.35 million.
Reduction in Shareholding: Loscalzo has decreased his shareholding in Ionis Pharmaceuticals by 1,032 shares since March 3, 2026, with a total value of approximately $82,833.
See More
Ionis Pharmaceuticals to Participate in Multiple Investor Conferences
- Investor Conference Schedule: Ionis Pharmaceuticals announced participation in the Bank of America Securities 2026 Health Care Conference on May 12, 2026, the RBC Capital Markets Global Healthcare Conference on May 19, 2026, and the Goldman Sachs 47th Annual Global Healthcare Conference on June 10, 2026, showcasing its innovations in RNA-targeted medicines.
- Live Webcast and Replays: Presentations will be accessible via a live webcast on the Investors & Media section of the Ionis website, with replays available within 48 hours post-event, ensuring investors stay updated on the company's latest developments.
- Company Background: For three decades, Ionis has focused on inventing medicines that improve the futures of patients with serious diseases, currently boasting marketed medicines and a leading pipeline in neurology, cardiometabolic diseases, and areas of high patient need, reflecting its deep expertise in the biopharmaceutical sector.
- Innovation-Driven Technology: As a pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies while advancing new approaches in gene editing, demonstrating a profound understanding of disease biology and industry-leading technology aimed at delivering life-changing treatments for patients.
See More
