AXSM
Axsome Therapeutics, INC
NASDAQ
8-K: Axsome Therapeutics' AXS-12 Achieves Primary Endpoint in Phase 3 Narcolepsy Trial
Sentiment:
Clinical Trial Results
25 March 2024 9:02 AM
Axsome Therapeutics announced that AXS-12 (reboxetine) significantly reduced cataplexy attacks compared to placebo in a Phase 3 trial for narcolepsy.
Better than expected
The results of the SYMPHONY trial were better than expected, as AXS-12 demonstrated statistically significant improvements across multiple endpoints compared to placebo.
Summary
- Axsome Therapeutics' AXS-12 met its primary endpoint in the SYMPHONY Phase 3 trial, demonstrating a statistically significant reduction in weekly cataplexy attacks compared to placebo.
- The trial involved 90 patients with narcolepsy with cataplexy, who were randomized to receive either AXS-12 or a placebo for 5 weeks.
- AXS-12 showed an 83% reduction in cataplexy attacks at week 5, compared to a 66% reduction in the placebo group (p=0.018).
- A significant reduction in cataplexy attacks was observed as early as week 1, with a 56% reduction for AXS-12 compared to 31% for placebo (p=0.007).
- 33% of patients on AXS-12 achieved remission of cataplexy (100% reduction from baseline) at week 5, compared to 9.5% on placebo (p=0.008).
- AXS-12 also significantly reduced excessive daytime sleepiness (EDS) severity, with a mean reduction of 1.8 points on the CGI-S scale compared to 0.9 points for placebo (p=0.027).
- The drug improved concentration and memory, as measured by the Cognitive Function Items of the FOSQ-10 (p=0.004).
- AXS-12 also improved overall narcolepsy severity and patient function and quality of life, as measured by the CGI-S for narcolepsy (p=0.007) and the FOSQ-10 total score (p=0.005), respectively.
- The most common adverse events in the AXS-12 arm were dry mouth, nausea, and constipation, all of which were mild to moderate.
- The trial showed a low rate of discontinuation due to adverse events, with one patient in each arm discontinuing the trial.
Sentiment
Score: 9
Explanation: The document presents very positive results from a Phase 3 trial, with statistically significant improvements across multiple endpoints and a favorable safety profile. This suggests a high likelihood of regulatory approval and market success, leading to a very positive sentiment.
Positives
- AXS-12 demonstrated a substantial and statistically significant reduction in cataplexy attacks compared to placebo.
- The drug showed rapid improvement in cataplexy symptoms, with significant reductions observed as early as week 1.
- A significant percentage of patients achieved remission of cataplexy while on AXS-12.
- AXS-12 significantly reduced excessive daytime sleepiness (EDS) severity.
- The treatment improved cognitive function, specifically concentration and memory.
- AXS-12 improved overall narcolepsy severity and patient quality of life.
- The drug was well-tolerated with a low rate of discontinuation due to adverse events.
- AXS-12 has been granted Orphan Drug Designation, potentially providing market exclusivity and fee waivers.
Negatives
- The most commonly reported adverse events in the AXS-12 arm were dry mouth, nausea, and constipation, although these were generally mild to moderate.
- There was a small number of patients who discontinued the trial due to adverse events, one in each of the AXS-12 and placebo arms.
Risks
- The company's future success depends on the continued commercial success of their existing products and the success of their clinical trials.
- There are risks associated with obtaining and maintaining regulatory approvals for their product candidates.
- The company faces risks related to intellectual property protection and the successful implementation of their research and development programs.
- Market acceptance of the company's products and product candidates is not guaranteed.
- The company's anticipated capital requirements may be impacted by unforeseen circumstances or disruptions to normal business operations.
Future Outlook
The company plans to complete the ongoing open-label safety extension trial of AXS-12 and work towards bringing the treatment to individuals living with narcolepsy. They also plan to present the detailed results of the SYMPHONY trial at upcoming scientific meetings.
Management Comments
- Dr. Michael Thorpy stated that AXS-12 represents a meaningful enhancement to the treatment options for narcolepsy patients and clinicians.
- Dr. Herriot Tabuteau, CEO of Axsome Therapeutics, said that the SYMPHONY trial results confirm the promise and potential of AXS-12 for the treatment of narcolepsy.
Industry Context
The announcement is significant as it addresses the unmet need in narcolepsy treatment, where many patients continue to experience symptoms despite existing therapies. The positive results of the AXS-12 trial suggest a potential new treatment option with a novel mechanism of action.
Comparison to Industry Standards
- Current treatments for narcolepsy include wake-promoting agents, oxybates, stimulants, and antidepressants, but many patients still experience persistent symptoms.
- The SYMPHONY trial results show that AXS-12 has the potential to offer a more effective treatment option, particularly in reducing cataplexy, which is a significant burden for patients.
- The rapid reduction in cataplexy attacks and the achievement of remission in a notable percentage of patients are promising results compared to existing treatments.
- The concurrent improvements observed in cataplexy, excessive daytime sleepiness, cognition, and overall function suggest that AXS-12 may provide a more comprehensive approach to managing narcolepsy symptoms compared to some current therapies.
Stakeholder Impact
- Shareholders are likely to react positively to the strong clinical trial results.
- Patients with narcolepsy may benefit from a new treatment option that addresses multiple symptoms.
- Physicians will have a new therapeutic option for managing narcolepsy.
- Employees of Axsome Therapeutics may experience increased job security and opportunities for growth.
Next Steps
- The company will complete the ongoing open-label safety extension trial of AXS-12.
- Axsome plans to present the detailed results of the SYMPHONY trial at upcoming scientific meetings.
- The company will work to bring AXS-12 to individuals living with narcolepsy.
Key Dates
- October 2018: AXS-12 was granted Orphan Drug Designation for the treatment of narcolepsy.
- March 25, 2024: Axsome Therapeutics issued a press release announcing the results of its SYMPHONY Phase 3 trial of AXS-12 in narcolepsy.
Keywords
AXS-12, narcolepsy, cataplexy, reboxetine, clinical trial, Phase 3, excessive daytime sleepiness, cognitive function, norepinephrine, dopamine
AXSM
Axsome Therapeutics, INC
NASDAQ
Sector: TBD
Filings with Classifications
Delay expected
9 June 2025 7:05 AM
Regulatory Update
- The Refusal to File (RTF) letter from the FDA necessitates an additional controlled trial for AXS-14.
- This new trial is anticipated to be initiated in the fourth quarter of 2025, indicating a delay in the regulatory pathway and potential market entry for AXS-14.
Worse than expected
9 June 2025 7:05 AM
Regulatory Update
- The FDA issued a Refusal to File (RTF) letter for the AXS-14 NDA, indicating the application was not sufficiently complete for substantive review.
- This requires Axsome to conduct an additional controlled trial, delaying the potential approval and commercialization of AXS-14.
Capital raise
13 May 2025 7:06 AM
8-K Filing
- Axsome conducted a private placement of 140,000 shares of common stock to Blackstone at a price of $107.14 per share.
- The gross proceeds from the private placement were approximately $15 million.
- Purchasers agreed to a lock-up period of 120 days from the closing date, restricting the sale or transfer of the securities.
Capital raise
5 May 2025 4:47 PM
Quarterly Report
- The Company may use a combination of public and private equity offerings, debt financings, other third-party funding, strategic alliances, licensing arrangements or marketing and distribution arrangements if market conditions are favorable or as a result of other strategic considerations to finance its future cash needs.
Delay expected
5 May 2025 4:47 PM
Quarterly Report
- If Subtenant is delayed in completing the New Premises Subtenant Work and vacate the Original Premises within four (4) months of the New Premises Commencement Date, subject to Force Majeure, then, notwithstanding anything to the contrary contained herein, Subtenant shall not be required to pay holdover rent as set forth in Section 3 above for the amount of time that Prime Landlords actions delayed Subtenant in completing the New Premises Subtenant Work (such amount of time, the Prime Landlord Delay) and the Original Premises Termination Date shall be extended by such period of Prime Landlord Delay; provided however that maximum amount of time for the delay in Subtenant paying holdover rent pursuant to Section 3 hereof shall be an additional thirty (30) days so that if Subtenant has not vacated the Original Premises and delivered possession of the Original Premises to Sublandlord due to Prime Landlord Delay within five (5) months of the New Premises Commencement Date, for whatever reason whatsoever, Subtenant shall pay such holdover rent pursuant to Section 3 hereof.
Better than expected
5 May 2025 4:47 PM
Quarterly Report
- Revenue increased significantly year over year.
- Net loss decreased year over year.
Better than expected
5 May 2025 7:33 AM
Quarterly Report
- The company's revenue growth exceeded expectations, driven by strong sales of AUVELITY and SUNOSI.
- The approval of SYMBRAVO and positive clinical trial results for solriamfetol in ADHD and SYMBRAVO in migraine patients were better than expected.
Worse than expected
1 April 2025 7:06 AM
Press Release
- The PARADIGM Phase 3 trial did not meet its primary endpoint in the overall patient population, indicating worse than expected results.
Better than expected
25 March 2025 7:06 AM
Clinical Trial Results
- The FOCUS Phase 3 trial achieved both its primary and key secondary endpoints, demonstrating statistically significant improvements in ADHD symptoms and disease severity compared to placebo.
Capital raise
18 February 2025 5:10 PM
Annual Results
- The company may need to raise additional financing to support its continuing operations.
- The company may seek to fund its operations through public or private equity, debt financings, or other sources.
Worse than expected
18 February 2025 5:10 PM
Annual Results
- The company's net loss increased from 2023 to 2024.
- Research and development expenses increased from 2023 to 2024.
- Selling, general, and administrative expenses increased from 2023 to 2024.
Delay expected
18 February 2025 7:06 AM
Annual Results
- Topline results from the ENGAGE Phase 3 trial of solriamfetol in BED are now anticipated in 2026.
Better than expected
18 February 2025 7:06 AM
Annual Results
- The company's revenue growth exceeded expectations, driven by strong sales of Auvelity and Sunosi.
- The FDA approval of Symbravo was a positive development, expanding the company's commercial portfolio.
- The successful completion of Phase 3 trials for AXS-05 and AXS-12 positions the company for potential NDA submissions and future product launches.
Better than expected
30 January 2025 5:55 PM
Press Release
- SYMBRAVO demonstrated statistically significant superiority compared to rizatriptan on sustained pain freedom from 2 to 24 hours.
Better than expected
30 December 2024 7:56 AM
Clinical Trial Results
- The ACCORD-2 trial met its primary endpoint with a statistically significant result, demonstrating a 3.6-fold lower risk of relapse compared to placebo, which is better than expected.
- The ACCORD-2 trial also met its key secondary endpoint, showing statistically significant prevention of relapse of agitation, which is better than expected.
- AXS-05 reduced the worsening of overall Alzheimer's disease severity compared to placebo in the ACCORD-2 trial, which is better than expected.
Better than expected
26 November 2024 7:05 AM
Clinical Trial Results
- The trial met its primary endpoint with statistically significant results, showing a reduction in cataplexy attacks compared to placebo.
- The drug also demonstrated statistically significant improvements in cognition and overall narcolepsy symptoms, exceeding expectations.
Worse than expected
12 November 2024 9:43 AM
Quarterly Report
- The company's net loss increased compared to the same period last year, indicating worse than expected results.
Capital raise
12 November 2024 9:43 AM
Quarterly Report
- The company may use a combination of public and private equity offerings, debt financings, other third-party funding, strategic alliances, licensing arrangements or marketing and distribution arrangements if market conditions are favorable or as a result of other strategic considerations to finance its future cash needs.
Better than expected
12 November 2024 7:07 AM
Quarterly Report
- The company's revenue growth significantly exceeded expectations, with Auvelity sales growing by 113% year-over-year.
- The company is progressing its pipeline faster than expected with multiple topline results expected in the near term.
- The company has sufficient cash to fund operations into cash flow positivity, which is better than expected.
Better than expected
1 October 2024 5:24 PM
Loan Agreement Amendment
- The loan amendment provides better terms for Axsome, including an increased tranche commitment, extended availability periods, and more flexible performance covenants.
Capital raise
5 August 2024 4:30 PM
Quarterly Report
- The company may use a combination of public and private equity offerings, debt financings, other third-party funding, strategic alliances, licensing arrangements or marketing and distribution arrangements if market conditions are favorable or as a result of other strategic considerations to finance its future cash needs.
Worse than expected
5 August 2024 4:30 PM
Quarterly Report
- The company reported a net loss of $79.3 million for the quarter, which is worse than the net loss of $67.2 million reported for the same period in the previous year.
Better than expected
5 August 2024 7:05 AM
Quarterly Report
- The company's revenue growth of 87% year-over-year and Auvelity sales growth of 135% year-over-year significantly exceeded expectations.
Delay expected
5 June 2024 7:38 AM
Settlement Announcement
- The settlement delays the entry of a generic version of Sunosi until at least June 30, 2042.
Capital raise
6 May 2024 5:20 PM
Quarterly Report
- The company may use a combination of public and private equity offerings, debt financings, other third-party funding, strategic alliances, licensing arrangements or marketing and distribution arrangements if market conditions are favorable or as a result of other strategic considerations to finance its future cash needs.
Worse than expected
6 May 2024 5:20 PM
Quarterly Report
- The company's net loss increased significantly compared to the same period last year, indicating worse than expected financial performance.
Worse than expected
6 May 2024 7:05 AM
Quarterly Report
- The company's net loss of $68.4 million for the quarter was significantly worse than the $11.2 million loss in the same period of 2023.
Worse than expected
25 March 2024 9:06 AM
Patient Survey Results Announcement
- The survey results show that a high percentage of patients continue to experience symptoms despite being on treatment, indicating that current treatments are not as effective as expected.
Better than expected
25 March 2024 9:02 AM
Clinical Trial Results
- The results of the SYMPHONY trial were better than expected, as AXS-12 demonstrated statistically significant improvements across multiple endpoints compared to placebo.
Better than expected
20 February 2024 7:05 AM
Annual Results
- The company's revenue growth of 309% year-over-year significantly exceeded expectations.
- Auvelity's sales of $130.1 million in its first full year of launch surpassed initial projections.
- Sunosi's 67% year-over-year revenue growth was also better than anticipated.
Delay expected
20 February 2024 7:05 AM
Annual Results
- The completion of the ADVANCE-2 trial for Alzheimer's disease agitation is now anticipated in the second half of 2024, due to enrollment trends and market dynamics, which is a delay from previous expectations.
Disclaimer: This summary was generated by artificial intelligence and its accuracy is not guaranteed. The information provided here is for general informational purposes only and does not constitute financial advice, recommendation, or endorsement of any kind. It may contain errors or omissions. You should not rely on this information to make financial decisions. Always seek the advice of a qualified financial professional before making any investment or financial decisions. Use of this information is at your own risk.