Clene Nanomedicine (CLNN): A new als treatment with potential for approval this month

DD: Clene Nanomedicine (CLNN): A New Potential ALS Treatment

Clene Nanomedicine (CLNN) is a company dedicated to research in the neurodegenerative field.  It is currently developing disease-modifying treatments for people living with Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), and Parkinson’s Disease (PD).  Its product, CNM-Au8, contains a liquid suspension of gold nanoparticles designed to boost nerve cells’ energy production.  

Recently, CLNN finished Phase 2 trials for all three diseases; all had positive data and will advance to Phase 3 trials.  The MS and PD trials and potential approval are likely years away.  However, ALS is such a fucked-up disease with such few treatment options the FDA allows for accelerated approvals of drugs that have shown promise in treating it.  CLNN previously applied for this approval last year on the premise that CNM-Au8 lowered the Neurofilament Light Chains (NfL) in ALS patients.  NfL levels are one of the few semi-biomarkers they have for ALS but are generally nonspecific for neurodegenerative diseases.  CNM-Au8 was reported to reduce NfL levels by 18% in one study.  Not great compared to Tofersen, another early approval that lowers NfL levels by 35-50% for a specific mutation of the disease.  

So CLNN continued interpreting its data and has applied for accelerated approval again. They were granted a meeting with the FDA last month, and in a recent presentation, the CEO said he expects to receive notice from the FDA this month.

Here is why I think this drug should be approved by the FDA now.  CNM-Au8 was trialed in a couple of studies specific to ALS to try and speed along approvals.  It did not meet its primary or secondary endpoints in any of those trials.  So why should the drug be approved you ask??  CLNN released more data since the trials, and it all looks extremely promising.  CNM-Au8 showed a 48-70% decrease in risk of all-cause mortality depending on the specific studies/registries being used for comparisons.  NfL levels were also reduced by an average of 28% after about 1.5 years of treatment.  There was also significantly less decline in ALSFRS-R total score than CNM-Au8 NfL non-responders.  In a recent presentation, CLNN officials noted that if bulbar onset patients were excluded from the studies, CNM-Au8 likely would have hit all its primary and secondary endpoints.

How does this compare with other ALS drugs approved through the accelerated approval process?  The last ALS drug approved through this program was Relyvrio.  Phase 2 clinical trial data showed a 43% lower risk of death.  NfL levels were unaffected by treatment.  These trials were also heavily questioned by regulators, particularly in Europe.  Ultimately, after phase 3 trials, Relyvrio was shown to be ineffective and pulled from the market.  CNM-Au8 has far more data than Relyvrio and has shown to be one of the most effective drugs currently in trials for treating ALS.  21.6% of the stock is owned by insiders, and it is at a nearly all-time low. It has an average price target of $51. At the recent presentation, the CEO noted that larger pharmaceutical companies are also looking to acquire them.  Further, even if FDA approval does not happen through the accelerated process, the company has a strong pipeline with good data and, in my opinion, has the second most promising ALS pipeline drug.

I like the company, what they're trying to do, and the stock.  Check it out if you want.