eRapa (encapsulated rapamycin) / Emtora Biosci, Biodexa Pharma - LARVOL DELTA

Pooled Results From the Phase 3 Trials of Nanoencapsulated Sirolimus Plus Pegadricase in Uncontrolled Gout: Characterization of Adverse Events of Special Interest (ACR Convergence 2026) - No abstract available

Adverse events • P3 data • Gout • Inflammatory Arthritis • Rheumatology

Evaluation of DISSOLVE I/II Stopping Rules for Nanoencapsulated Sirolimus Plus Pegadricase (NASP) in Patients (pts) With Uncontrolled Gout (ACR Convergence 2026) - No abstract available

Clinical • Gout • Inflammatory Arthritis • Rheumatology

Biodexa Pharmaceuticals PLC…is pleased to announce that it has exceeded the half-way point in the recruitment of subjects in its registrational Phase 3 trial of eRapa in Familial Adenomatous Polyposis (FAP), NCT06950385 (GlobeNewswire) - "As of today, 87 of a planned 168 subjects have been recruited into the Serenta trial. The trial is recruiting at 29 clinical sites across the US and five countries in Europe with a further three sites in Canada expected to be initiated shortly. The Company is planning a futility analysis after 25 Progression Free Survival (PFS) events and database lock after 75 PFS events in the Serenta trial. The Serenta protocol includes a composite endpoint which defines the nature of the PFS events."

Trial status • Genetic Disorders • Rare Diseases

Targeted Treatment for Hyperuricemia: The Drug Pipeline. (PubMed, Drugs Aging) - "Agents such as dotinurad have demonstrated effective urate lowering with favorable renal and hepatic safety profiles in older individuals, including patients with chronic kidney disease, suggesting potential advantages in this higher-risk population. Likewise, novel uricase-based strategies, including immune-tolerizing platforms such as nanoencapsulated sirolimus plus pegadricase (NASP), aim to overcome immunogenicity and treatment burden associated with pegloticase. Novel agents targeting the NLRP3 inflammasome and IL-1β appear to have promise for anti-inflammatory prophylaxis and flare treatment that are integral to effective ULT. Currently, with several next-generation agents in late-phase trials, efforts will be needed to enhance the generalizability of these studies to older patients with comorbidity that are common to real-world gout care."

Journal • Review • Cardiovascular • Chronic Kidney Disease • Gout • Immunology • Inflammatory Arthritis • Nephrology • Renal Disease • Rheumatology • IL1B • NLRP3

Efficacy and Safety of Nanoencapsulated Sirolimus plus Pegadricase: Results from the Randomized, Placebo-Controlled Phase 3 Trials. (PubMed, Arthritis Rheumatol) - "NASP treatment resulted in a significantly higher proportion of patients with complete SU response during Weeks 21-24 compared to placebo and was generally well-tolerated. NASP, an every-4-week treatment, can markedly alleviate disease burden in patients with UG."

Journal • P3 data • Dental Disorders • Gout • Infectious Disease • Inflammatory Arthritis • Rheumatology • Stomatitis

eRapa-FAP: Expanded Access to 0.5 mg eRapa for Familial Adenomatous Polyposis (clinicaltrials.gov) - P=N/A | N=0 | Available | Sponsor: Biodexa Pharmaceuticals

New trial • Genetic Disorders • APC

MTX230-301: Phase 3 trial of eRapa in patients with familial adenomatous polyposis (FAP) (clinicaltrialsregister.eu) - P2/3 | N=42 | Recruiting | Sponsor: Biodexa Limited, Rapamycin Holdings Inc. | N=32 ➔ 42

Enrollment change • Genetic Disorders • APC

Trial of Encapsulated Rapamycin (eRapa) for Bladder Cancer Prevention (clinicaltrials.gov) - P2 | N=166 | Active, not recruiting | Sponsor: Robert Svatek | Recruiting ➔ Active, not recruiting | Trial completion date: Jan 2026 ➔ Jul 2027 | Trial primary completion date: Jan 2026 ➔ Jan 2027

Enrollment closed • Trial completion date • Trial primary completion date • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Nanoencapsulated Sirolimus Plus Pegadricase (NASP) Provides a Sustained 48-Week Response, Reducing Serum Uric Acid and Preserving Renal Function in Patients With Uncontrolled Gout (UG) and Stage 3 Chronic Kidney Disease (NKF-SCM 2026) - P3 | "NASP (formerly SEL-212) is an investigational, novel, every 4-week, sequential, 2-component infusion therapy consisting of targeted nanoencapsulated sirolimus (NAS) and pegadricase that reduces serum uric acid (sUA). Across 48 weeks, NASP-treated patients showed a 96.3% reduction in cumulative sUA area under the curve versus PBO. Safety findings were consistent with those previously presented for the subset of patients with CKD3.3 CONCLUSION In patients with UG and CKD3, NASP stabilizes renal function and improves gout clinical outcomes over 48 weeks versus PBO."

Clinical • Chronic Kidney Disease • Gout • Inflammatory Arthritis • Nephrology • Renal Disease • Rheumatology

Phase II trial of encapsulated rapamycin to reduce polyp burden associated with familial adenomatous polyposis. (PubMed, Clin Cancer Res) - "eRapa was safe, tolerable and showed preliminary efficacy for FAP chemoprevention. The 0.5 mg daily every-other-week schedule will be evaluated in an upcoming phase III trial."

Journal • P2 data • Colonic Polyps • Colorectal Cancer • Genetic Disorders • Oncology • Small Intestinal Carcinoma

Chronic rapamycin treatment attenuates age-related motor deficits in sex-dependent manner in UM-HET3 mice. (PubMed, J Gerontol A Biol Sci Med Sci) - "Mice were supplemented with microencapsulated rapamycin at 14 ppm starting at 12 months of age. Our data show a novel beneficial effect of rapamycin on age-related motor deficits that is not sex-specific and that these changes are associated with reduction in protein carbonyls in brain regions linked to motor function. Furthermore, our results are consistent with the idea that rapamycin's beneficial effects are mediated, at least in part, by reducing oxidative stress and ER stress-mediated apoptosis."

Journal • Preclinical • GFAP

A Clinical Study Comparing eRapa and Rapamune in Healthy Adult Volunteers. (ANZCTR) - P1 | N=30 | Not yet recruiting | Sponsor: Biodexa Australia PTY Ltd

New P1 trial • Genetic Disorders

MTX230-301: Phase 3 trial of eRapa in patients with familial adenomatous polyposis (FAP) (clinicaltrialsregister.eu) - P2/3 | N=32 | Recruiting | Sponsor: Biodexa Limited, Rapamycin Holdings Inc.

New P2/3 trial • Genetic Disorders • APC

Nanoencapsulated Sirolimus Plus Pegadricase (NASP) in Uncontrolled Gout: Current Status and Future Directions. (PubMed, Drugs) - "The Food and Drug Administration recently approved the co-administration of methotrexate with pegloticase to inhibit ADA formation and enhance the efficacy and tolerability of pegloticase. Video abstract available online. Video Abstract."

Journal • Allergy • Gout • Inflammatory Arthritis • Rheumatology

Biodexa Activates First European Site for Registrational Phase 3 Serenta Trial in FAP (GlobeNewswire) - "...University of Bonn, Germany is now actively screening patients...The randomized, double-blind, placebo-controlled Serenta trial (NCT06950385) is designed to evaluate whether eRapa can prevent disease progression in patients with FAP....Nine additional European sites will activate during the next 2-3 months across the Netherlands, Spain, Denmark, and Italy."

Trial status • Genetic Disorders • Rare Diseases

Nanoencapsulated Sirolimus Plus Pegadricase (NASP) Reduces Gout Clinical Manifestations in Patients with Uncontrolled Gout (UG) and Stage 3 CKD (KIDNEY WEEK 2025) - P3 | "NASP (formerly SEL-212) is an investigational, novel, every 4-wk (Q4W), sequential 2-component infusion therapy consisting of targeted nanoencapsulated sirolimus (NAS) and pegadricase (P) that reduces serum uric acid (sUA). Safety was similar to the ITT population ( Table ), with only one case of proteinuria in each NASP arm (not considered drug related). Conclusion NASP was well tolerated and reduced disease burden in pts with UG and CKD."

Clinical • Chronic Kidney Disease • Gout • Inflammatory Arthritis • Nephrology • Renal Disease • Rheumatology

Effect of Nanoencapsulated Sirolimus Plus Pegadricase (NASP) on Kidney Outcomes: Results from the Phase 3 DISSOLVE Studies (KIDNEY WEEK 2025) - P3 | "Conclusion These data show stable renal function over the study period in NASP-treated patients with more patients having an improvement in CKD stage compared to PBO. Findings here suggest NASP may be a uricase treatment option for patients with UG and CKD-3."

P3 data • Chronic Kidney Disease • Gout • Inflammatory Arthritis • Nephrology • Renal Disease • Rheumatology

Biodexa Announces Approval of CTA in Europe for Phase 3 Serenta Trial in FAP First European patient expected to be enrolled in 4Q 2025 Addressable US - European Market Put at $7 Billion (GlobeNewswire) - "The CTA permits the Serenta trial to proceed in Europe, initially covering clinical sites in Denmark, Germany, Netherlands and Spain with Italy expected to be added in due course...The Serenta trial (NCT06950385) is a randomized, double-blind, placebo-controlled Phase 3 registrational study designed to evaluate the safety and efficacy of eRapa in patients diagnosed with FAP. Multiple sites in the US are actively screening eligible participants. It is expected that 168 patients will be recruited into the trial randomized 2:1 drug: placebo."

Trial status • Rare Diseases

Nanoencapsulated Sirolimus Plus Pegadricase Reduced Disease Burden in Patients With Uncontrolled Gout: Results From the Phase 3 DISSOLVE Trials (ACR Convergence 2025) - P3 | "NASP (formerly SEL-212) is an investigational, novel, every 4-wk (Q4W), sequential 2-component infusion therapy consisting of targeted nanoencapsulated sirolimus (NAS, formerly SEL-110), which mitigates the formation of antipegadricase antibodies, and a uricase (pegadricase [P]; formerly SEL-037), which reduces sUA. NASP-treated pts had meaningful reductions in disease burden and key clinical manifestations, as reflected by improvements in sUA levels and PROs. This therapy has a noteworthy impact on resolving acute gout symptoms over 6 treatments and shows promise for improving HRQOL."

Clinical • P3 data • Gout • Immunology • Inflammatory Arthritis • Rheumatology

Reduction in Tophi Observed in Patients with Uncontrolled Gout Treated with NASP: Results from Phase 3 DISSOLVE Studies (ACR Convergence 2025) - P3 | "NASP (formerly SEL-212) is a novel, every 4-week, sequential infusion therapy consisting of targeted immunomodulating, nanoencapsulated sirolimus (NAS; formerly SEL-110) co-administered with a pegylated uricase (formerly SEL-037) designed to reduce serum uric acid (sUA) levels in pts with UG. Treatment with NASP resulted in a greater tophus response compared to PBO. In addition, a robust sUA reduction, a key factor in tophus resolution, was observed in NASP-treated pts. These results highlight the effectiveness of NASP in lowering sUA and promoting tophus resolution."

Clinical • P3 data • Gout • Immunology • Inflammatory Arthritis • Musculoskeletal Diseases • Musculoskeletal Pain • Orthopedics • Rheumatology

Nanoencapsulated Sirolimus plus Pegadricase (NASP) Demonstrates Long Term Efficacy and Safety in Patients with Uncontrolled Gout: Results from the 24-week Double-blind Extension of the Phase 3 DISSOLVE I Study (ACR Convergence 2025) - P3 | "Nanoencapsulated sirolimus plus pegadricase (NASP, formerly SEL-212) is a novel investigational therapy delivered every 4 weeks as a sequential, two-component infusion consisting of targeted nanoencapsulated sirolimus and pegadricase, a pegylated uricase (Figure 1A). These data support the long-term durability of NASP as shown by sustained sUA reductions and decreased gout flares over time, with 100% on HD NASP and 92.3% on LD NASP being flare-free at the end of the extension phase. NASP was generally well tolerated with low discontinuation rates during the extension phase. Overall, NASP led to improvements in key clinical manifestations of UG."

Clinical • P3 data • Gout • Inflammatory Arthritis • Rheumatology

Nanoencapsulated Sirolimus plus Pegadricase (NASP) Demonstrates a Reduction in Gout Flares: Results from the Phase 3 DISSOLVE Studies (ACR Convergence 2025) - P3 | "NASP (formerly SEL-212) is an investigational, every 4-week, sequential infusion therapy designed to reduce sUA levels in patients (pts) with uncontrolled gout (UG), consisting of targeted immunomodulating, nanoencapsulated sirolimus (NAS; formerly SEL-110), sequentially administered with pegadricase (a pegylated uricase; formerly SEL-037). The proportion of pts with flares and number of flares decreased with NASP compared to PBO over the course of the study, with 95.2% and 94.3% of HD and LD-treated pts being flare-free during weeks 21–24. These results highlight the potential of NASP as an effective therapy for reducing disease burden and improving a key clinical outcome in pts with UG."

P3 data • Gout • Immunology • Inflammatory Arthritis • Rheumatology

Characterization of Infusion Reactions Within 1 Hour of Treatment With Nanoencapsulated Sirolimus Plus Pegadricase: Pooled Results From the Phase 3 DISSOLVE I and DISSOLVE II Trials (ACR Convergence 2025) - P3 | "NASP (formerly SEL-212) is an investigational, novel, every 4-wk (Q4W), sequential 2-component infusion therapy consisting of tolerogenic nanoencapsulated sirolimus (NAS, formerly SEL-110), which mitigates formation of antipegadricase antibodies, and a uricase (pegadricase [P]; formerly SEL-037), which reduces sUA...All pts fully recovered with appropriate medical intervention (mainly steroids and antihistamines) provided at the infusion center; 1 pt received epinephrine for anaphylaxis... The low incidence (4.0%) of pts experiencing IRs, including hypersensitivity reactions, within 1 h of NASP suggests that NAS mitigates ADA formation. No pts required in-patient hospitalization or intubation, and all IRs resolved with standard measures (mainly at the infusion center)."

P3 data • Gout • Immunology • Inflammatory Arthritis • Rheumatology

Updates in uricase therapy for gout. (PubMed, Curr Opin Rheumatol) - "Exogenous uricases available and those under development are discussed, focusing on immunomodulation and anti-inflammatory prophylaxis to reduce flares, prevent antidrug antibody formation and infusion reactions, and mitigate loss of efficacy in patients with uncontrolled gout needing uricase replacement therapy."

Journal • Developmental Disorders • Gout • Immunology • Inflammatory Arthritis • Oncology • Rheumatology

Biodexa Announces Filing of CTA in Europe for Phase 3 Serenta Trial in Familial Adenomatous Polyposis (FAP) (GlobeNewswire) - "Biodexa Pharmaceuticals...announced the filing of a Clinical Trial Application (CTA) with the European Medicines Agency (EMA) for its Serenta trial in patients with familial adenomatous polyposis (FAP), a mostly inherited disease that, if left untreated, almost always leads to colorectal cancer. The only current treatment option is sequential resection of much of the gastrointestinal tract...if approved, it would permit the Serenta trial to proceed in Europe, initially covering clinical sites in Denmark, Germany, Netherlands and Spain with Italy expected to be added in due course....The Serenta trial (NCT06950385) is a randomized, double-blind, placebo-controlled Phase 3 registrational study designed to evaluate the safety and efficacy of eRapa in patients diagnosed with FAP....Following the 106 day approval timeline for the CTA, it is expected the European sites will begin enrolling in the fourth quarter of this year."

Trial status • Rare Diseases