As the only currently approved treatment option for Alpha-1 lung disease, plasma donor-derived Alpha-1 Antitrypsin protein (human alpha-1 proteinase inhibitor [A1PI] augmentation therapy) remains the current standard of care. pdAAT has improved patient outcomes but an opportunity exists to advance the standard of care in Alpha-1.
At Sanofi, a group of passionate scientists came together with a mission to develop a novel therapy for patients living with Alpha-1. Using science and innovation, the journey to develop the investigational drug SAR447537 (INBRX101) began.
SAR447537 (INBRX101) is a recombinant (artificial) form of augmentation therapy that has been engineered to last longer in the body. This means that SAR447537 (INBRX101) could potentially be given less frequently than currently approved pdAAT augmentation therapies, while keeping the levels of AAT in the normal range.
The first leg of the journey: The Phase 1 study
SAR447537 (INBRX101) was studied in an investigational study in 31 adults with Alpha-1. This Phase 1 study looked at the safety, pharmacokinetics (levels of drug in the body), and pharmacodynamics (how the study drug affects AAT levels) of SAR447537 (INBRX101). In the completed Phase 1 study, SAR447537 (INBRX101), when administered at single doses and multiple doses up to 120 mg/kg administered every 3 weeks for up to 3 doses, was found to be safe and tolerable.
Overall, the side effects reported were generally mild or moderate in severity and resolved on their own without needing additional treatment. The most frequently reported side effects were fatigue and infusion-related side effects (e.g., itching, blood pressure increase, etc.). This study also showed that the levels of functional AAT (the protein that helps protect the lungs from damage) that were measured in the blood were similar to levels of AAT that are seen in people without Alpha-1.1
The trail gets steeper
SAR447537 (INBRX-101) was investigated in the ElevAATe Study, a phase 2 randomized, double-blind, multicenter, active-control study designed to assess SAR447537 (INBRX-101) compared to Zemaira ® in patients with Alpha-1 -related emphysema.
Thank you to the Alpha-1 community for engaging with the Alpha-1 Research Registry at the Alpah-1 Foundation by expressing interest in this stage of the ElevAATe Study. It is because of volunteers like you in the Alpha-1 community why Sanofi, AATD Inc was able to complete enrollment and move to the next stage of the journey, the ElevAATe-OLE (open label extension) Study.
The ElevAATe-OLE Study is now enrolling.
The journey doesn’t end with ElevAATe
SAR447537 (INBRX101) is now being investigated in ElevAATe, a phase 2 randomized, double-blind, multicenter, active-control study designed to assess SAR447537 (INBRX101) compared to Zemaira ® in patients with Alpha-1 -related emphysema.
In this study, participants will receive SAR447537 (INBRX101) once every 3 weeks or every 4 weeks for approximately 3 years. The study drug and study visit assessments will be provided at no cost to participants and this study is planning to include home/remote health care options to potentially allow some participants to make less frequent visits to the study site.
How do I enroll?
To join the journey and learn more about ElevAATe-OLE study, complete the following pre-screening form to see if you may qualify. Please note this is not a full list of criteria and additional screening may be needed. Please click the link below to begin.
To access the Alpha-1 Foundation Pre-Screening, click here.
For any questions or concerns, please contact the Alpha-1 Foundation Research Registry Coordinators at 1-877-228-7321 ext. 245 or 252, or alpha-1registry@alpha1.org.
Kuhn BT, Veale A, Farah H, et al. Recombinant human AAT protein INBRX-101 demonstrates potential to achieve lung penetration and normal functional serum AAT levels in patients with AAT deficiency. Presented at: the ATS International Conference 2023; May 19-24, 2023; Washington, DC.
To access the Alpha-1 Foundation Pre-Screening, click here.
For any questions or concerns, please contact the Alpha-1 Foundation Research Registry Coordinators at 1-877-228-7321 ext. 245 or 252, or alpha-1registry@alpha1.org.
