CAPR: My Current Favorite Biotech

CAPR: A Biotech with Enormous Potential in 2026

This isn’t financial advice, just my own DD. Disclaimer: I have a long position in CAPR ($10.69 average)

TLDR: CAPR has FDA approval decision for their drug, Deramiocel, on August 22nd, 2026. I think their phase 3 data looks fantastic and has a strong chance of being approved. If it is approved, I predict that this stock will be trading around $60 per share (over $70 per share if given a broad label by the FDA) by the end of summer. There is potential for it to be trading over $100 per share by the end of 2026 if FDA gives a broad label including treatment of cardiomyopathy.

This is my favorite biotech stock right now. It is currently trading right around $34.42 per share ($2 billion market cap). I think this stock will almost double in value (\\\~$60 per share) by mid-to-late summer this year. CAPR has developed a drug called Deramiocel which treats Duchennes Muscular Dystrophy, a serious pediatric disease that primarily affects boys.

\*\*1. FDA Approval\*\*: CAPR received a rejection letter for the drug last year from the FDA, but it was based on their phase 2 study data. CAPR found significant positive effects of their drug in the phase 2 study, but it was a small sample size, so the statistical significance was found using non-parametric analysis (meaning it wasn’t a normal distribution, which makes sense considering the small sample size). The FDA rejected it based on that analysis method.

CAPR finished their phase 3 study in late 2025 and announced top line data from it in December 2025, which revealed they had hit their primary and secondary end points of the study. That news skyrocketed the stock from $6 per share to settling around $25 per share.

The phase 3 “Hope” study has a rigorous study design and much larger sample size than their phase 2 study. CAPR resubmitted their application for Deramiocel to the FDA in early 2026 and the FDA accepted the application, and set a decision date for August 22nd, 2026. That is the absolute latest we will find out the decision, because there’s a chance it could be announced sooner than that (due to the drug being a treatment for a serious, rare pediatric disease). CAPR held a presentation at a medical conference in early March which provided much more in depth analysis of their phase 3 results, and the data looks fantastic. Deramiocel hit not only their primary end point, but all secondary endpoints.

When CAPR received the CRL last year, there were no CMC issues mentioned in the CRL by the FDA. Therefore, I think the FDA approval odds for Deramiocel are very high (90-95% in my opinion). If the drug gets approved by the FDA, CAPR stock is going to sky rocket again, and I predict it will settle around $60 per share, and that is my conservative estimate.

\*\*2. Patient Population\*\*: There are a couple more catalysts that could send it higher before the end of the year. Deramiocel was given Orphan Drug Designation by the FDA for Becker Muscular Dystrophy (a less serious case compared to DMD). If the FDA gives a broad label to Deramiocel, meaning it can be used to also treat BMD, then that will be a big increase in patient population for the drug. There are about 15,000 people with DMD in the US, with 350-400 new patients diagnosed each year. There are about 5,000 people in the USA with Becker MD (60-120 new patients each year). If Deramiocel is given a broad label to treat even cardiomyopathy, then that be a massive increase in potential patient population. My more aggressive prediction is that CAPR could potentially hit \\\~$100 per share before the end of 2026, if these other catalysts happen.

Those patient numbers are just for the US. CAPR has said they will look to get EU approval after they get approval in the US. There are 25,000-30,000 people with DMD in the EU. There are 8,000-12,000 people with Becker MD in the EU.

\*\*3. Deramiocel Production Capability\*\*: CAPR has a production facility in San Diego, CA. They are currently able to produce enough Deramiocel to treat 500 patients each year. However, their facility has additional clean rooms to expand production capabilities. The expansion is supposed to be completed in late 2027. After expansion, CAPR will be able to produce enough Deramiocel to treat 3,000 patients each year. They are also currently stockpiling Deramiocel as they await the upcoming FDA decision. The rumor (from a StockTwits user named LineBacker04, who has done extensive DD on CAPR) is that CAPR will have enough Deramiocel to treat an extra 300-350 patients this year. CAPR has not said how much Deramiocel will cost, but their only DMD drug competitor, which is developed by SRPT, costs $3 million per patient. That SRPT drug, Elevidys, has toxicity issues and the drug has a warning label. If CAPR charged $3 million (I don’t think they’ll charge that high) for Deramiocel, then they already have the potential to make $1.5 billion worth of Deramiocel, and $9 billion by the end of 2027. If they charge $1 million for it, then they can currently produce $500 million already, and $3 billion by end of 2027.

\*\*4. CAPR Financials & FDA Approval Impact:\*\* CAPR is that they are good on cash through 2027, so they won’t be making offerings/diluting any time soon if at all. They have over $300 million cash on hand. They have about $14.5 million in debt.

If CAPR receives FDA approval, I don’t think it will be a “sell the news” situation either, for a couple reasons. First, there is a fairly high amount of shorted shares of CAPR right now (over 19%), which could cause a short squeeze if approved. Second, CAPR will most likely receive a PRV from the FDA of Deramiocel is approved, since it treats a rare, serious pediatric disease. CAPR can sell that PRV for $100-$200 million, or they could use it for their Stealth X vaccine in their pipeline (still early development for that, but research is going to ramp up for that in 2027). Third, CAPR will receive an $80 million milestone payment from Nippon for FDA approval.

All in all, I think CAPR has a high change to 2X by this summer, and potentially triple or even quadruple (best case scenario) by the end of 2026.