In this article, we will list the 5 stock picks of Billionaire Joe Edelman with huge upside potential. Please visit Billionaire Joe Edelman’s 10 Stock Picks with Huge Upside Potential if you would like to see the extended list and the methodology behind it.

5. Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM)

Perceptive Advisors’ Stake: $273 Million

Analyst Upside Potential: 74%

While Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM) faces high cash burn, hedge funds are piling into the stock due to its successful precision obesity pivot, which separates it from the broad-market GLP-1 (Ozempic/Wegovy) hype. In recent months, the firm has registered a series of landmark regulatory wins for their lead drug, IMCIVREE. In mid-March, the FDA granted full approval for IMCIVREE to treat acquired hypothalamic obesity, a high-unmet-need market. In late March, an EU body issued a positive opinion for the same indication. Funds view this as a transformative label expansion. Unlike generic obesity, hypothalamic obesity is rare and often follows brain tumors or surgery. This gives Rhythm a 100% market monopoly in this niche, providing pricing power that broad-market drugs cannot match.

There is also renewed focus on the rapidly scaling top-line performance of Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM). For the full year 2025, the firm reported a 50% year-over-year revenue increase, reaching roughly $194.8 million. Investors are positioning themselves in the stock ahead of the clinical shift from daily injections to oral pills. In early 2026, Rhythm initiated its Phase 3 trials for bivamelagon, a once-daily oral MC4R agonist. Smart money managers realize that an oral version would drastically increase patient adherence and market penetration.

4. Spyre Therapeutics, Inc. (NASDAQ:SYRE)

Perceptive Advisors’ Stake: $100 Million

Analyst Upside Potential: 88%

Following a string of clinical successes in early April, Spyre Therapeutics, Inc. (NASDAQ:SYRE) stock has become a favorite for managers looking to capitalize on the multi-billion dollar Inflammatory Bowel Disease market. A major catalyst for the shares was the recent announcement of Part A induction results from the Phase 2 SKYLINE trial for SPY001. The drug met its primary endpoint in patients with moderate-to-severe ulcerative colitis. The trial showed a 40% clinical remission rate at Week 12, which analysts from Goldman Sachs noted is potentially superior to currently available treatments like Entyvio. The data confirmed a pharmacokinetic profile that could allow for quarterly or even biannual subcutaneous dosing, a massive convenience advantage over the monthly or bi-monthly schedules of competitors.

Spyre Therapeutics, Inc. (NASDAQ:SYRE) has a unique strategy of combining best-in-class antibodies. Beyond SPY001, Spyre is developing long-acting antibodies targeting TL1A and IL-23. Hedge funds believe the real blockbuster potential lies in Spyre’s plan to test these antibodies in combination. Analysts estimate that successful combination therapies could redefine the standard of care in a global IBD market projected to reach $50 billion annually. Given the high efficacy and dosing convenience of its lead assets, many elite money managers view Spyre as a prime acquisition target for Big Pharma players like AbbVie or Takeda looking to defend their IBD franchises.

3. Celcuity Inc. (NASDAQ:CELC)

Perceptive Advisors’ Stake: $315 Million

Analyst Upside Potential: 92%

Celcuity Inc. (NASDAQ:CELC) stock went on a wild surge last year and hedge funds are continuing to buy the shares as they view the firm as a likely leader in the next generation of breast cancer treatments. A prominent catalyst for the company has been the stellar clinical results for gedatolisib, Celcuity’s lead candidate for HR+/HER2- advanced breast cancer. In March, results published in the Journal of Clinical Oncology confirmed that the gedatolisib triplet reduced the risk of disease progression or death by 76% compared to standard fulvestrant. The median progression-free survival (PFS) was 9.3 months for the triplet versus only 2.0 months for the control group. Funds view this 7.3-month incremental improvement as a best-in-class result that will likely disrupt the current standard of care.

There has been institutional positioning in Celcuity Inc. (NASDAQ:CELC) ahead of a major regulatory decision scheduled for this summer. In March, the firm announced that the FDA granted Priority Review for its New Drug Application for gedatolisib. The FDA has assigned a PDUFA goal date of July 2026. Institutional investors typically pile into de-risked biotech stocks three to four months before a PDUFA date, betting on a successful commercial launch. Celcuity is turning from a research lab into a commercial entity. It recently expanded its commercial headcount and added $30 million in debt financing.

READ MORE: 10 Best Long-Term Stocks to Buy According to Bill Ackman.

2. Nurix Therapeutics, Inc. (NASDAQ:NRIX)

Perceptive Advisors’ Stake: $67 Million

Analyst Upside Potential: 100%

Elite managers are piling into Nurix Therapeutics, Inc. (NASDAQ:NRIX) stock to capitalize on its leadership in Targeted Protein Degradation (TPD), a field many believe is the next frontier of medicine. A near-term catalyst for the shares has been the clinical momentum of its lead candidate, NX-5948. Recent data presentations have highlighted the drug’s ability to degrade Bruton’s tyrosine kinase (BTK) even in patients who have failed standard BTK inhibitors like ibrutinib. Analysts are specifically bullish on NX-5948’s unique ability to cross the blood-brain barrier. This allows it to target cancers that have spread to the central nervous system, a massive unmet need that existing drugs cannot effectively treat.

Nurix Therapeutics, Inc. (NASDAQ:NRIX) has an extensive collaboration network. The firm has multi-billion dollar partnerships with Gilead Sciences, Sanofi, and Pfizer. While retail sentiment is mixed due to current losses, $295 million trailing 12-month loss, the smart money is focused on the 44.4% annual revenue growth rate projected for the TPD market. Institutional investors are buying ahead of the initiation of Phase 3 trials for its lead assets. They view the current $1.7 billion valuation as a deep discount for a company with multiple best-in-class degraders in late-stage development.

1. Praxis Precision Medicines, Inc. (NASDAQ:PRAX)

Perceptive Advisors’ Stake: $588 Million

Analyst Upside Potential: 262%

Praxis Precision Medicines, Inc. (NASDAQ:PRAX) is still pre-revenue but Wall Street is piling into the stock because of a rare triple-catalyst setup in neurology. There are two major regulatory decisions that could transform the company into a commercial powerhouse in late 2026 and early 2027. In March, the FDA granted Priority Review for relutrigine in pediatric epilepsy. The PDUFA target action date is set for September 2026. Similarly, in April, the FDA accepted the NDA for ulixacaltamide with a PDUFA date of January 2027. This drug is the first positive Phase 3 program in essential tremor, a market that has seen no new innovation in decades. Recent data from the EMBRAVE trial was also positive.

In early April, Praxis Precision Medicines, Inc. (NASDAQ:PRAX) reported that 71% of patients treated with elsunersen achieved a >50% reduction in motor seizures. Analysts believe the success of elsunersen and relutrigine validates Praxis’s entire precision neuroscience platform. Funds view the company’s lead programs as having a combined $20 billion potential revenue opportunity. The firm has aggressive capital management, which has eliminated the bankruptcy risk often associated with clinical-stage biotech. After a successful follow-on offering in early 2026, Praxis ended Q1 with roughly $1.5 billion in cash. This extends its runway into 2028, ensuring it can fund the commercial launches of its own drugs without further dilution.

READ NEXT: Billionaire David Tepper’s 10 Small and Midcap Stock Picks with Huge Upside Potential and 10 Best Stocks to Invest In According to Billionaire Steve Cohen.

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