12 Hidden Biotech Winners: How Early Conviction Translated Into Triple-Digit Returns in 2025

The biotech sector delivered a masterclass in opportunity recognition in 2025, with lesser-known clinical-stage companies capturing outsized investor interest. Reflecting on the year’s performance, a portfolio of underappreciated therapeutics companies—many focused on rare diseases, oncology breakthroughs, and unmet medical needs—generated transformational gains for early believers.

Rare Disease Specialists Leading the Charge

Palvella Therapeutics Inc. (PVLA) exemplified the rare disease opportunity when highlighted at $25 in March 2025. The company’s lead asset, QTORIN rapamycin, targets microcystic lymphatic malformations, cutaneous venous malformations, and clinically significant angiokeratomas—conditions with minimal treatment options. By late December, PVLA had surged to $114.69, subsequently settling near $100. The market recognized the multi-billion-dollar addressable space for QTORIN across multiple indications.

NANOBIOTIX (NBTX) pursued a physics-based approach to cancer therapeutics with NBTXR3 (JNJ-1900), licensed to Johnson & Johnson’s Janssen division. Flagged at $12.23 in September, the stock climbed to $30.35 by mid-October before stabilizing around $21. The company anticipates interim data from its NANORAY-312 trial in head-and-neck squamous cell carcinoma during H1 2027, with additional Phase 1 readouts expected throughout 2026.

Oncology Pipeline Acceleration

Celcuity Inc. (CELC) demonstrated dramatic momentum in breast and prostate cancer therapeutics. Gedatolisib, its lead candidate, reached an FDA New Drug Application submission milestone in November 2025 for HR+ HER2- advanced breast cancer. Initial positioning occurred at $12.60 in December 2024, with the stock subsequently reaching $112.64 in mid-December 2025—a remarkable nine-fold appreciation. Current levels near $99 still reflect substantial year-over-year gains.

Cogent Biosciences Inc. (COGT) marked progress in precision therapeutics with Bezuclastinib across non-advanced systemic mastocytosis, advanced systemic mastocytosis, and gastrointestinal stromal tumors (GIST). The company’s first NDA submission for Non-AdvSM represented a critical regulatory milestone. From a July entry point of $7.25, COGT ascended to $43.73 by early December, before moderating to $35—still up 380% annually.

Terns Pharmaceuticals Inc. (TERN) captured investor enthusiasm following CARDINAL trial data on TERN-701, its chronic myeloid leukemia candidate. November results showed 64% major molecular response rates within 24 weeks, with updated data revealing 75% response rates among patients receiving doses exceeding 320 mg daily. The company progressed from $5.98 in August to a peak of $48.26 in December.

Expansion Beyond Oncology

DBV Technologies S.A. (DBVT) ventured into food allergy treatment with VIASKIN Peanut patch. A Phase 3 trial achieved its primary endpoint in peanut-allergic children aged 4-7, positioning the company for BLA submission in H1 2026. Peak sales projections reached $1.5 billion by 2030. From $10.64 in October, the stock rose to $26.18 by mid-December, currently trading around $19.

Sol-Gel Technologies Ltd. (SLGL) operated in dermatology with FDA-approved TWYNEO and EPSOLAY already commercialized. The company advanced SGT-610 in Gorlin syndrome (Phase III, results expected Q4 2026) and SGT-210 in Darier disease (Phase 1b ongoing). Starting from $7.26 in May, SLGL peaked at $52.26 in October before settling near $43.

Strategic Acquisitions and Regulatory Approvals

Cidara Therapeutics Inc. (CDTX) achieved a notable exit when agreed acquisition by Merck at $221.50 per share (approximately $9.2 billion) was announced, closing expected in Q1 2026. The company’s influenza prevention therapy became an attractive bolt-on for the pharmaceutical giant.

Inhibrx Biosciences Inc. (INBX) demonstrated clinical validation when Ozekibart (INBRX-109) showed statistically significant progression-free survival improvements in chondrosarcoma registrational trials. FDA biologics license application submission is targeted for Q2 2026. From $18.35 in July, the stock climbed to an all-time high near $94.57 by late December.

Commercial Infrastructure and Platform Play

Nutex Health Inc. (NUTX) represented a divergent thesis—operational transformation rather than pipeline potential. The healthcare services provider reversed a $424 million net loss (2022) into $52 million net income (2024) and $59 million (first nine months of 2025). Revenue growth mirrored this turnaround: $219 million (2022) to $723.6 million (nine months 2025). From $33.56 in November 2024 and $70 in April 2025, NUTX reached $193.07 recently, closing near $182.23.

Early Detection and Precision Medicine

GRAIL Inc. (GRAL) pursued early cancer detection through its Galleri multi-cancer early detection test. The company announced plans for PMA submission to the FDA in Q1 2026, supported by NHS-Galleri and PATHFINDER 2 study data. Positioned at $48.50 in September, the stock surged to $115.76 by November before moderating to $88.

Zenas BioPharma Inc. (ZBIO) focused on autoimmune diseases with Obexelimab. Phase 2 MoonStone trial data in relapsing multiple sclerosis demonstrated 95% relative reduction in new gadolinium-enhancing T1 lesions. Phase 3 INDIGO data in IgG4-Related Disease remain pending. From $8.79 in December 2024, ZBIO reached $44.60 by year-end, consolidating around $35.

The Takeaway

These twelve companies—spanning rare diseases, oncology, immunotherapy, dermatology, and early detection—exemplified 2025’s biotech opportunity set. Substantial returns accrued to investors who recognized inflection points in clinical development timelines and regulatory catalysts. For those seeking similar opportunities in 2026, understanding therapeutic areas, trial design, and addressable market sizes remains foundational to identifying the next generation of breakout performers.