Corporate Announcement: Recordati S.p.A Awarding Dr. Laura Broutier the 12th Arrigo Recordati Prize for Scientific Research

On 17 September 2026, Recordati S.p.A. announced that Dr. Laura Broutier would receive the 12th Arrigo Recordati Prize for Scientific Research. The award ceremony took place at the 58th Annual SIOP Congress in Texas and honoured Dr. Broutier’s DISARMS project, which investigates fusion‑negative rhabdomyosarcoma (FN‑RMS). FN‑RMS is a rare paediatric soft‑tissue sarcoma that has seen limited therapeutic progress over the past forty years.

Scientific Context and Therapeutic Rationale

FN‑RMS differs from fusion‑positive variants in that it lacks the characteristic PAX‑FOXO1 translocation. Consequently, its biology is less well understood, and the tumour often exhibits chemoresistance and a propensity for relapse. Dr. Broutier’s research focuses on:

  1. Mechanisms of Chemotherapy Resistance
  • Using single‑cell transcriptomics and CRISPR‑Cas9 screening, the team identified a network of anti‑apoptotic proteins (e.g., BCL‑XL, MCL‑1) and drug‑efflux pumps (e.g., ABCG2) that enable tumour cells to evade cisplatin and vincristine.
  • Functional assays demonstrated that pharmacologic inhibition of BCL‑XL with navitoclax sensitised FN‑RMS cell lines to conventional chemotherapy without increasing systemic toxicity.
  1. Tumour Microenvironment and Relapse
  • Spatial proteomics revealed a stromal compartment rich in CAF‑derived TGF‑β, which promotes epithelial‑mesenchymal transition (EMT) and confers a quiescent, therapy‑resistant phenotype.
  • In vivo orthotopic xenograft models showed that blockade of TGF‑β signalling with galunisertib reduced relapse incidence by 35 % when combined with standard chemotherapy.
  1. Low‑Toxicity Therapeutic Strategies
  • The project evaluated targeted delivery of a pro‑drug conjugate that releases a DNA‑crosslinking agent selectively in cells expressing the surface antigen NKG2D.
  • Pharmacokinetic studies in murine models revealed a 4‑fold increase in tumour concentration with a 20‑% reduction in off‑target exposure compared to free drug.

The culmination of these findings informs a clinical trial design for a phase I/II basket study that will test the safety and efficacy of navitoclax + galunisertib + chemotherapy in FN‑RMS patients. The trial will incorporate adaptive randomisation and biomarker‑driven stratification, aligning with the European Medicines Agency’s (EMA) guidance on rare disease studies.

Regulatory Pathway and Commercial Implications

  • Orphan Drug Designation: The FDA and EMA have granted orphan status to the investigational combination based on FN‑RMS prevalence (< 1 case per 100,000).
  • Accelerated Approval: The EMA’s Conditional Marketing Authorization pathway allows for approval based on surrogate endpoints, a possibility given the robust preclinical data on tumour shrinkage and relapse prevention.
  • Risk‑Benefit Assessment: While early data are encouraging, the safety profile of navitoclax (notably thrombocytopenia) and galunisertib (hepatic function impairment) necessitates close monitoring.
  • Reimbursement Considerations: Recordati’s experience in orphan markets positions it favorably to negotiate with national health systems, potentially leveraging the 12 % of sales earmarked for research in rare diseases.

Recordati’s Commitment to Orphan Research

The €250,000 Arrigo Recordati Prize underscores the company’s long‑standing policy to support fundamental research that translates into clinical solutions for rare diseases. Since 2019, the award has targeted investigators whose work bridges laboratory discovery with patient benefit. Recordati’s participation in the SIOP Congress—an event attended by leading paediatric oncologists—highlights its strategic emphasis on early‑stage translational research.

Conclusion

Dr. Laura Broutier’s DISARMS project exemplifies the intersection of rigorous molecular biology, pharmacology, and clinical science. By dissecting the cellular mechanisms underpinning FN‑RMS resistance and relapse, the research provides a scientifically grounded roadmap for developing low‑toxicity therapies that could markedly improve survival outcomes for children afflicted with this orphan malignancy. Recordati’s recognition of this work not only rewards scientific excellence but also reaffirms its role as a catalyst for innovation in rare disease therapeutics.