8-K: Sage Therapeutics and Biogen's SAGE-324 Fails to Meet Primary Endpoint in Phase 2 Essential Tremor Study

A Phase 2 study of SAGE-324 for essential tremor did not demonstrate a statistically significant dose-response relationship, leading to the termination of further development for this indication.

Summary

• Sage Therapeutics and Biogen announced topline results from their Phase 2 KINETIC 2 study of SAGE-324 (BIIB124) for essential tremor.
• The study did not show a statistically significant dose-response relationship on the primary endpoint, which was the change from baseline to Day 91 on the TETRAS Performance Subscale Item 4 total score.
• There were no statistically significant differences between any dose of SAGE-324 and placebo in the change from baseline on the TETRAS PS Item 4 Total Score or the TETRAS Activities of Daily Living Composite Score.
• The study involved 147 participants who were randomized to placebo, 15 mg, 30 mg, or 60 mg of SAGE-324 for a three-month period.
• Due to these results, Sage and Biogen will close the ongoing open-label safety study of SAGE-324 in essential tremor and will not pursue further clinical development for this indication.
• The companies are evaluating next steps for other potential indications.

Sentiment

Score: 3

Explanation: The document reports negative clinical trial results, leading to the termination of a development program, which is a significant setback for the company. The sentiment is therefore negative.

Positives

• The study provided valuable data that may inform future research and therapeutic approaches for essential tremor.
• The companies are evaluating next steps for other potential indications of SAGE-324.

Negatives

• SAGE-324 did not demonstrate efficacy in treating essential tremor in the Phase 2 study.
• The primary endpoint of the study was not met.
• There were no statistically significant differences between any dose of SAGE-324 and placebo.
• Further clinical development of SAGE-324 for essential tremor has been terminated.

Risks

• The failure of SAGE-324 in this study may impact the overall pipeline and valuation of Sage Therapeutics.
• There is a risk that other potential indications for SAGE-324 may also not be successful.
• The companies may face challenges in finding alternative uses for the drug.
• The termination of the study may lead to a loss of investment in the essential tremor program.

Future Outlook

Sage and Biogen are evaluating next steps for SAGE-324 in other potential indications, but no specific plans are currently in place. The companies have stated they will not pursue further clinical development of SAGE-324 in essential tremor.

Management Comments

• Laura Gault, MD, PhD, Chief Medical Officer, Sage Therapeutics, stated that they are disappointed that the results do not support further development of SAGE-324 in essential tremor.
• Katherine Dawson, MD, Head of Therapeutics Development Unit, Biogen, expressed disappointment but believes the findings add to the collective understanding of the condition.

Industry Context

The failure of SAGE-324 highlights the challenges in developing new treatments for essential tremor, a condition with limited pharmacological innovation over the past 50 years. This result may impact investor sentiment towards companies focused on neurological drug development.

Comparison to Industry Standards

• The failure of SAGE-324 in the KINETIC 2 study is a setback compared to other companies that have had success in developing treatments for neurological conditions.
• Other companies such as Acadia Pharmaceuticals and Teva Pharmaceuticals have had more success in developing treatments for neurological conditions, although not specifically for essential tremor.
• The lack of statistically significant results for SAGE-324 contrasts with the positive results seen in some other Phase 2 trials for different neurological conditions, such as those targeting Alzheimer's disease or Parkinson's disease.

Stakeholder Impact

• Shareholders may react negatively to the news of the failed clinical trial.
• Employees working on the SAGE-324 program may be affected by the termination of the study.
• Patients with essential tremor will not have this potential treatment option available.
• The results may impact the future collaboration between Sage and Biogen.

Next Steps

• Sage and Biogen will close the ongoing open-label safety study of SAGE-324 in essential tremor.
• The companies will not conduct further clinical development of SAGE-324 in essential tremor.
• Sage and Biogen are evaluating next steps for other potential indications of SAGE-324.

Key Dates

Keywords

SAGE-324, Biogen, Essential Tremor, Clinical Trial, Phase 2, KINETIC 2, Neuroactive Steroid, GABAA Receptor, Drug Development, CNS Depressant