AXSM
Axsome Therapeutics, INC
NASDAQ
8-K: Axsome Therapeutics' AXS-12 Achieves Primary Endpoint in Phase 3 Narcolepsy Trial
Sentiment:
Clinical Trial Results
26 November 2024 7:05 AM
Axsome Therapeutics announced that AXS-12 met its primary endpoint in a Phase 3 trial, demonstrating a statistically significant reduction in cataplexy attacks compared to placebo in patients with narcolepsy.
Better than expected
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.
Summary
- Axsome Therapeutics' AXS-12 (reboxetine) has successfully met the primary endpoint in the ENCORE Phase 3 trial for narcolepsy, showing a statistically significant reduction in the frequency of cataplexy attacks compared to placebo.
- The trial included a 6-month open-label treatment period followed by a 3-week double-blind, placebo-controlled withdrawal period.
- 68 patients were enrolled in the open-label phase, with 42 patients randomized in the double-blind phase to either continue AXS-12 or switch to placebo.
- Patients who switched to placebo experienced a significant increase of 10.29 cataplexy attacks per week compared to an increase of 1.32 attacks per week for those who continued on AXS-12.
- AXS-12 also demonstrated statistically significant improvements in cognition and overall narcolepsy symptoms compared to placebo.
- During the open-label phase, patients experienced a 71% reduction in cataplexy attacks at 1 month, which was sustained at 77% at 6 months.
- 72% of patients achieved a 50% reduction in cataplexy attacks at 1 month, increasing to 82% at 6 months.
- The percentage of cataplexy-free days increased from 14% at baseline to 61% at 1 month and 70% at 6 months.
- Excessive daytime sleepiness (EDS) improved, with mean ESS scores reduced by 5.6 points at 1 month and 7.3 points at 6 months.
- Cognition improved, with 55% of patients reporting improvement at 1 month and 59% at 6 months on the NSAQ, and 67% at 1 month and 70% at 6 months on the PGI-C.
- Overall narcolepsy status improved, with 90% of patients showing improvement at both 1 and 6 months on the CGI-C.
- Work impairment due to narcolepsy decreased from 53% at baseline to 24% at 6 months.
- The long-term safety profile of AXS-12 was consistent with previous trials, with no new safety signals detected.
Sentiment
Score: 9
Explanation: The document presents overwhelmingly positive results from a Phase 3 trial, with strong statistical significance and clear benefits for patients. The company's plan to move towards an NDA filing further boosts the positive sentiment.
Positives
- AXS-12 demonstrated a statistically significant reduction in cataplexy attacks compared to placebo.
- The drug showed statistically significant improvements in cognition and overall narcolepsy symptoms.
- A high percentage of patients achieved a 50% reduction in cataplexy attacks and improvements in EDS.
- The long-term safety profile of AXS-12 was consistent with previous trials.
- AXS-12 significantly increased the percentage of cataplexy-free days.
- The drug reduced work impairment due to narcolepsy.
- The results confirm the efficacy of AXS-12 in patients with narcolepsy with cataplexy, demonstrated in three positive controlled trials.
- The company plans to move expeditiously towards an NDA filing for AXS-12.
Negatives
- 17.6% of patients discontinued the trial due to adverse events during the 6-month open-label treatment period.
- The most common adverse events were nausea and tachycardia, each reported in 5.9% of patients.
Risks
- The company needs to successfully navigate the NDA filing process with the FDA.
- There is a risk that the FDA may not approve AXS-12 despite the positive trial results.
- The company needs to successfully commercialize AXS-12 if approved.
- There is a risk of unforeseen circumstances or disruptions to normal business operations.
Future Outlook
Axsome plans to move expeditiously towards an NDA filing for AXS-12 and intends to request a pre-NDA meeting with the FDA.
Management Comments
- Dr. Michael Thorpy commented that the results support AXS-12 as a potentially important new option for physicians and patients.
- Dr. Herriot Tabuteau, CEO of Axsome Therapeutics, stated that the results confirm the efficacy of AXS-12 and that the potential benefits are substantial and sustained with long-term treatment.
- Dr. Tabuteau also expressed pleasure with the improvements in cataplexy, excessive daytime sleepiness, and cognition.
Industry Context
This announcement is significant for the narcolepsy treatment landscape, as it provides further evidence for the efficacy of AXS-12, a novel treatment option. The positive results could position Axsome as a key player in the CNS therapeutics market.
Comparison to Industry Standards
- The results of the ENCORE trial are compared to previous trials of AXS-12, showing consistent efficacy and safety.
- The document references the need for new treatment options for narcolepsy, indicating that current treatments may not fully address the variety of disease presentations.
- The document does not directly compare AXS-12 to specific competitor products, but highlights the need for options that can address the variety in disease presentation, suggesting that AXS-12 may offer a unique approach.
Stakeholder Impact
- Shareholders are likely to react positively to the successful trial results.
- Patients with narcolepsy may benefit from a new treatment option.
- Physicians will have a new tool to manage narcolepsy symptoms.
- Employees of Axsome may experience increased job security and opportunities.
Next Steps
- Axsome plans to move expeditiously towards an NDA filing for AXS-12.
- The company intends to request a pre-NDA meeting with the FDA.
Key Dates
- November 26, 2024: Date of the press release announcing the results of the ENCORE Phase 3 trial.
Keywords
AXS-12, narcolepsy, cataplexy, reboxetine, clinical trial, Phase 3, FDA, cognition, excessive daytime sleepiness, CNS disorders
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
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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.
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.
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
30 January 2025 5:55 PM
Press Release
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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.
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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
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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.
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.
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
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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.
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.
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.
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