Last year, Paragon Biosciences and its portfolio companies advanced transformative science with a singular mission: to accelerate breakthroughs that improve lives. Across neurology, genetic disorders, regenerative medicine, and AI-driven drug discovery, we achieved milestones that not only moved programs forward but also strengthened the foundation for long-term impact.
Emalex Biosciences: Pioneering a New Era in Tourette Syndrome Care
Emalex is redefining possibilities for patients with Tourette syndrome. Emalex reported positive topline data for its Phase 3 study last year. With this data in hand, the company plans to submit a New Drug Application in the coming weeks, a critical step toward potentially delivering a new first-in-class therapy for Tourette syndrome.
The company also received authorization from the FDA for an Expanded Access Program, enabling physicians to request treatment for eligible patients who have exhausted existing therapeutic options. This authorization underscores the urgent need for innovation in this space.
Looking ahead, Emalex is rapidly scaling up its team and preparing initiatives for product introduction and patient programs, once approved. Fundraising and partnering efforts are underway to support these initiatives.
Beyond Tourette syndrome, Emalex is advancing a Phase 2 proof-of-concept study for refractory restless legs syndrome, an area of high unmet need. An FDA Investigational New Drug submission is planned for early 2026 to enable the launch of clinical studies.
Harmony Biosciences: Accelerating Growth and Expanding Impact
Harmony reported exceptional Q3 results with $239.5M in net product revenue, up 29% year-over-year, and raised full-year guidance to $845–$865M. Six years post-launch, Harmony is nearing blockbuster status, backed by $778M in cash and investments to fuel innovation and strategic growth.
The company is expanding its leadership in sleep/wake health with two new pitolisant formulations: high-dose (HD) and gastro-resistant (GR). Phase 3 trials for pitolisant HD are planned, with results expected in 2027, while positive data for pitolisant GR supports a New Drug Application in early 2026. Patent filings could extend exclusivity through 2044.
Harmony is also advancing a potential best-in-class orexin 2 receptor agonist for narcolepsy, idiopathic hypersomnia, and related disorders. The program recently dosed its first participant in Phase 1 trial. Topline results are expected in 2026. In epilepsy, Phase 3 studies for Dravet syndrome and Lennox-Gastaut syndrome are underway, with topline data expected in 2026.
Harmony’s pipeline strategy is catalyst rich. Multiple programs expected to deliver clinical and regulatory milestones for sustained growth and value creation, advancing innovative therapies for patients with rare neurological disorders.
CiRC Biosciences: Expanding Horizons in Regenerative Medicine
Over the past year, CiRC has confirmed the transformative potential of its regenerative medicine platform by successfully generating two distinct cell types. Building on the strong progress of its pioneering retinal program, CiRC has expanded into central nervous system disorders and delivered promising results with a neuron cell replacement initiative aimed at Parkinson’s disease.
This progress was further fueled by a strategic research collaboration with a large pharma partner to develop additional programs targeting refractory epilepsy and treatment-resistant narcolepsy. This collaboration combines CiRC’s expertise in cellular reprogramming with the pharma partner’s clinical and commercial capabilities to accelerate the potential development of off-the-shelf cell therapies.
Early research is showing exciting potential to change the course of Parkinson’s disease, rather than just managing symptoms. The goal is to restore healthy neuron function for a true disease modifying therapy. Initial results from animal studies are expected in 2026, and more programs are moving forward toward early proof-of-concept milestones in the year ahead.
Evozyne: Harnessing AI to Revolutionize Therapeutic Protein Design
Evozyne’s Generative AI platform is accelerating the design of next-generation protein therapies in immunology. Its first clinical candidate — a differentiated immunoglobulin protease for the treatment of autoimmune diseases — is on track for human trials in 2026 with the goal of reducing harmful autoantibodies that drive disease while minimizing side effects.
Additional novel programs in therapeutic development are targeting a variety of autoimmune disorders that have been historically difficult to treat. Programs in discovery include engineering a special protein (cytokine) to upregulate anti-inflammatory effects without elevating pro-inflammatory effects in the molecule. The goal is to make these treatments more effective without causing unwanted side effects, reducing the chances that the body will reject these treatments.
Evozyne’s leadership in protein engineering was recently highlighted in two prestigious, peer-reviewed publications:
- PNAS: Showcasing a collaboration with NVIDIA to accelerate protein design with tailored functions using advanced AI.
- microPublication Biology: Demonstrating synthetic rubisco activase enzymes engineered by Evozyne for therapeutic and sustainability applications.
With a world-class scientific team, strong investor backing, and a robust pipeline, Evozyne is poised for a transformative 2026.
Castle Creek Biosciences: Advancing Gene Therapy for DEB
Castle Creek has launched its pivotal Phase 3 study for its gene-modified cell therapy targeting dystrophic epidermolysis bullosa (DEB), a devastating, rare skin condition. In 2025, the company delivered significant progress on study enrollment and initial patient dosing is scheduled for 2026.
To address the global need for this regenerative medicine, Castle Creek secured ex-U.S. rights to its lead asset and began developing new formulations for DEB patients with unmet medical needs.
To guide execution in this critical stage, Castle Creek has strengthened its leadership team, adding three seasoned biopharma experts:
- Chris Larson, MS, MLS (ASCP), Vice President of Quality Control
- Rhea Williams, MPH, Vice President of Regulatory Affairs
- Michael Townsley, RN, Head of Quality Assurance
Market insights from this year confirm strong demand for effective DEB treatments. Physicians continue to encounter a large burden of DEB wounds and wound types poorly addressed by current treatment options.
With its ability to treat diverse wound types and sizes, Castle Creek’s therapy has the potential to set a new standard of care for DEB and offers a best-in-class solution that complements existing treatments to improve patient outcomes.
