Every once in a while, a stock comes along that makes even the most seasoned investors do a double-take.
I’m talking about the kind where a major Wall Street firm slaps a price target on it that implies the shares could literally triple – or more – over the next 12-18 months. Most of the time those calls feel like wishful thinking. But every now and then the math actually checks out, the story is compelling, and the risk/reward looks absurdly lopsided in your favor.
That’s exactly what seems to be happening right now with one under-the-radar biopharmaceutical company focused on rare diseases.
Why JPMorgan Believes This Stock Could More Than Triple
Late November, JPMorgan updated their conviction list for 2026. Out of hundreds of rated stocks, only one carried an implied upside of over 225% from current levels. That stock trades on Nasdaq, has a market cap around $3.5 billion, and has been quietly building what analysts believe could be a blockbuster franchise in ultra-rare genetic disorders.
Shares have been beaten down more than 20% over the past year – which, frankly, is the only reason this opportunity even exists today.
The Core Thesis: One Drug Almost Nobody Is Being Priced at Zero
At the heart of the bull case is a late-stage asset called setrusumab, currently in pivotal trials for osteogenesis imperfecta – commonly known as brittle bone disease.
This isn’t some obscure indication with a tiny market. While exact patient numbers vary, conservative estimates suggest over 40,000 addressable patients across the US and Europe alone, with peak sales potential routinely modeled north of $1 billion annually by the most optimistic analysts.
Yet here’s the kicker: the current share price appears to ascribe almost zero value to setrusumab. The market is essentially pricing the company purely on its existing commercial portfolio and cash balance – as if the biggest pipeline candidate doesn’t even exist.
In my experience covering biotech for years, that kind of disconnect rarely lasts once meaningful clinical data starts rolling in.
“At current levels, we see minimal value being ascribed to setrusumab (or the broader pipeline). On positive data, we continue to see upside into the mid-$60s on the low end.”
Lead JPMorgan biotech analyst, November 2025
Recent Clinical Updates That Changed Everything
Last summer, the company released interim looks from two pivotal studies – Cosmic and Orbit – that appeared to validate the mechanism of action in a way that surprised even the bulls.
Patients treated with setrusumab showed meaningful improvements in bone mineral density and, crucially, a trend toward fewer fractures – the exact clinical endpoint that matters most to regulators and physicians.
Perhaps more importantly, the safety profile has remained clean. For a disease where patients (many of them children) suffer spontaneous fractures multiple times per year, a therapy that can strengthen bones without serious side effects would be practice-changing.
Full data readouts are expected sometime in the first half of 2026. That’s the binary event everyone is circling on their calendars.
Two Additional Catalysts the Market Is Ignoring
- Priority Review Vouchers (PRVs): The company is on track to receive not one, but two of these golden tickets from the FDA. Recent transactions have seen PRVs change hands for $130–160 million each. That alone could inject $250–300 million of non-dilutive cash – essentially 8–10% of the current market cap – with zero execution risk.
- Commercial momentum in approved products: Existing rare-disease therapies continue to post 20–30% year-over-year growth. Management has guided to GAAP profitability by 2027 even excluding any contribution from setrusumab. Add in a potential blockbuster, and the earnings power becomes explosive.
When you back out the cash runway and the value of the current business, you’re essentially getting the setrusumab opportunity for free. Or, looked at another way, you’re paying nothing for the rest of an increasingly valuable rare-disease platform.
How the Valuation Math Works
Let’s run some quick numbers (always dangerous with biotech, but bear with me).
Current enterprise value sits roughly $2.8 billion after netting out cash and the expected PRV proceeds.
- Existing commercial assets + cash → conservatively worth $3–4 billion on their own in a takeout scenario.
- Setrusumab, even at a highly discounted 3–4× peak sales multiple on $1 billion potential → another $3–4 billion.
- Rest of pipeline (multiple Phase 2/3 programs in ultra-rare indications) → option value easily $1–2 billion.
Add it up and you’re looking at $7–10 billion of fundamental value against a $3.5 billion market cap today. Even if you haircut every assumption by 30–40%, the current price still looks unreasonably cheap.
Risks? Of Course – This Is Biotech
Nobody is claiming this is a risk-free home run. Clinical trials can always disappoint. Regulatory hurdles remain. Competition could emerge longer term.
But the beauty of the current setup is the asymmetry. The stock has already priced in a significant amount of bad news. Downside feels relatively contained to the mid-$20s, while upside on positive data could easily push shares north of $100 within 12–18 months.
When Wall Street’s smartest analysts are willing to stick their necks out with a 225%+ upside call, it’s usually worth paying attention.
Final Takeaway
Sometimes the best opportunities hide in plain sight – beaten-down stocks in unpopular sectors, overlooked by the broader market, quietly executing while everyone else chases the hot trend.
Right now, rare-disease biotech fits that description perfectly. And within that niche, one company stands out as having the clearest path to massive re-rating over the next 12–24 months.
Whether shares actually triple or “only” double will depend on clinical data next year. But the setup – strong existing business, pristine balance sheet, multiple non-dilutive cash catalysts, and a potential blockbuster being priced at zero – feels almost too good to ignore.
For investors comfortable with biotech volatility, this might be one of those rare chances to buy a high-conviction idea when the crowd is looking the other way.
As always, do your own homework. But if JPMorgan is right, we could be talking about one of the biggest winners of 2026.
