Clene (CLNN -3.74%), a clinical-stage biopharmaceutical company that focuses on neurodegenerative diseases, saw its shares rise 11.8% on Monday. The stock is up more than 17% so far this year.
Investors have been pushing up the stock since Clene's announcement on Thursday regarding positive early data from a phase 2 trial for its therapy CNM-Au8 in patients with amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease.
ALS is a neurodegenerative disease that affects spinal-cord and brain nerve cells, causing patients to gradually lose the ability to control muscle movement, leading to paralysis and death.
There is no cure yet for the disease, only treatments that might reduce its symptoms. About 5,000 new cases of ALS are diagnosed each year in the United States. The typical cost of treatment per patient is $1,433,922, according to one study.
CMS-Au8 is the biotech's lead therapy. It is also one of the lead drugs being studied in Massachusetts General Hospital's Healey ALS Trial with results expected in the second half of the year. CMS-Au8 is also in Clene trials to treat Parkinson's disease and multiple sclerosis.
The potential for a cure for ALS would be a huge opportunity. The compound annual growth rate for ALS treatments between 2022 and 2030 is expected to be 8.1%, according to one study, reaching $520.8 million by 2030. That's without a cure, which if CMS-Au8 can provide that, would make it a blockbuster drug.
Because no drug has been shown to cure ALS, investors may want to be cautious regarding Clene until its therapies can be shown to be effective in later-stage trials, either against ALS or other neurodegenerative diseases.
Bear in mind, too, that the company has little revenue, so investors would have to be patient to see much of a payoff. In its first-quarter report, it listed $30,000 in revenue, including $23,000 in royalty revenue.