$Tonix Pharmaceuticals (TNXP.US)$ This is a high-risk, high-...
This is a high-risk, high-reward bet that could turn 4 digits into 5 digits (or 5 digits into 6 digits) if approved between June and August 2025 without further dilution. It offers the opportunity for at least 4x returns or possibly up to 10x, but it could also result in a complete loss of your investment. This opportunity is only suitable for high-risk takers who have conducted thorough due diligence and are prepared to lose the money without regret. Invest only a small percentage of your capital, an amount you can afford to lose, if you choose to take this risk.
Historically, the FDA's Center for Drug Evaluation and Research (CDER) has approved approximately 75% of novel drug applications.However, approval rates can vary based on factors such as the drug's therapeutic area, clinical trial data robustness, and the presence of unmet medical needs.
Why This Approval Could Be Groundbreaking
If approved, TNX-102 SL would be the first drug for fibromyalgia approved in over 15 years. Developing effective treatments for fibromyalgia has proven challenging, with multiple drug candidates failing at late stages in the approval process:
- Mirogabalin: Developed by Daiichi Sankyo as a successor to pregabalin (Lyrica), this drug aimed to improve efficacy and reduce side effects. However, in 2017, it failed to show significant pain reduction in Phase 3 trials, leading to its discontinuation.
- Esreboxetine: This selective norepinephrine reuptake inhibitor by Pfizer initially showed promise but failed to deliver sufficient benefits over existing medications in Phase 3 trials.
- Sodium Oxybate: Marketed for narcolepsy, this drug was evaluated for fibromyalgia but rejected by the FDA in 2010 due to safety concerns, including respiratory depression and misuse potential.
- NYX-2925: Aptinyx's NMDA receptor modulator failed to show significant pain reduction in Phase 2 trials in 2022, leading to the discontinuation of its development.
These cases highlight the complexities of developing fibromyalgia treatments. Despite these challenges, TNX-102 SL has demonstrated strong results in Phase 3 trials, raising hopes for a new therapeutic option.
Why TNX-102 SL May Have a Higher Approval Chance
Examining why previous drug candidates failed reveals a common theme: severe side effects outweighed potential benefits. In contrast, TNX-102 SL has shown a mild side effect profile, which could improve its chances of approval.
Side Effects of Previously Failed Drugs:
- Sodium Oxybate: Associated with nausea, dizziness, somnolence, respiratory depression, and dependence risk.
- Esreboxetine: Caused dry mouth, nausea, fatigue, and cardiovascular issues like hypertension and tachycardia.
- Mirogabalin: Linked to dizziness, somnolence, weight gain, and dependence risk similar to pregabalin.
TNX-102 SL Side Effects:
- Common:
- Mild somnolence
- Transient tongue numbness (due to the sublingual delivery method)
- Implications: TNX-102 SL's side effects appear significantly less severe, particularly in terms of cardiovascular and dependence risks.
Historical Context and Efficacy of Current FDA-Approved Fibromyalgia Drugs
The three FDA-approved fibromyalgia drugs—Pregabalin (Lyrica), Duloxetine (Cymbalta), and Milnacipran (Savella)—also exhibit side effects but were deemed acceptable given their efficacy:
- Pregabalin (Lyrica): Approved in 2007, it reduced pain and improved sleep quality but caused dizziness and somnolence.
- Duloxetine (Cymbalta): Approved in 2008, it improved pain and function but caused nausea and dry mouth.
- Milnacipran (Savella): Approved in 2009, it alleviated pain and fatigue but caused headache and hypertension.
TNX-102 SL's Efficacy:
In the RESILIENT study, TNX-102 SL achieved:
- Statistically significant reduction in daily pain (p=0.00005)
- Improvements in sleep quality, fatigue, and overall function
-The p-value of 0.00005 indicates a highly statistically significant result, meaning there is an extremely low probability (0.005%) that the observed reduction in daily pain occurred due to chance.
Example:
It means there’s only a 0.005% chance (1 in 20,000) that the results were due to random luck.
In other words, the pain reduction observed in the trial is very likely real and not a coincidence.
This level of statistical significance strongly supports the efficacy of TNX-102 SL in reducing daily pain in the study population. While the magnitude of pain reduction would depend on the actual data (e.g., percentage or numerical pain score reduction), the p-value highlights that the result is robust and clinically meaningful in the context of the trial.
Conclusion
Based on the drug's demonstrated efficacy, mild side effect profile, and the history of FDA-approved fibromyalgia treatments, TNX-102 SL appears to have a better chance of approval compared to previously failed candidates. However, the final decision will hinge on the FDA's comprehensive review of its safety and efficacy data. For investors, this is a speculative play, suitable only for those prepared to accept both the potential for high rewards and the risk of total loss.
Disclaimer: Community is offered by Moomoo Technologies Inc. and is for educational purposes only.
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Arrayfunction : Amazing post! It's awesome to see someone take the time to educate people about the nuisances of biotech. I also appreciate how you outlined the risk involved multiple times. This is great content. I've always wondered why retail investors don't come together on a platform like Stack Overflow so we can all learn from each other's unique insights. I think that was Peter Lynch's advice? Not sure, I don't know much about the history of fund managers
72638079 Arrayfunction : I agree, just started actively trading recently and find it hard to find a consolidated place of educated investors having open discussion. Can we start groups on here?
Arrayfunction 72638079 : I honestly have no clue!