Recombinant Human Laminin-111 (rhLAM-111) – A protein therapy for muscular dystrophies
Questions and Answers
What stage is this research?
This research is pre-clinical – the manufacturing capability is being developed but rhLAM-111 has not yet advanced to clinical trials. rhLAM-111 is being developed for two therapeutic areas:
- Protein therapy for LAM-211 deficiency: congenital muscular dystrophy type 1A (MDC1A)
- Protein therapy for dystrophin deficiency: Duchenne muscular dystrophy
Where is this research being done and who is funding this research?
LAM-111 research is performed at the University of Nevada, Reno and Prothelia Inc. in Massachusetts, and other laboratories in the US, Canada, and the EU.
RhLAM-111 is manufactured by BioLamina.
Prothelia is funded by our company founders, and previous grants from NIH, the PPMD End Duchenne GAP program, Struggle Against Muscular Dystrophy (SAM) and Hope for Gus.
What is the goal or purpose of this research?
Manufacturing of rhLAM-111 and clinical testing in patients with MDC1A and Duchenne.
What is the current state of this research?
This project is in preclinical development and we have demonstrated effectiveness in the mdx and canine GRMD models of Duchenne. RhLAM-111 will help all Duchenne patients regardless of their mutation. Current efforts are focused on MDC1A and positive clinical data for MDC1A will accelerate clinical development for Duchenne.
RhLAM-111 has 2 mechanisms of action critical to Duchenne:
- Stabilizes skeletal and cardiac muscle membranes through upregulation (increased expression) of modifier proteins alpha7beta1 integrin and utrophin.
- Direct stimulation of satellite cells and more efficient muscle regeneration.
- What steps need to be completed before moving into a clinical trial?
- We continue to develop and optimize the manufacturing process for rhLAM-111.
- Clinical trial designs for treatment of MDC1A are underway.
What is your best estimate for the length of time it will take to move this research into clinical trials?
Initiation of Phase 1 clinical testing in MDC1A patients is anticipated mid/2021 with proof of concept anticipated in 2022. Phase 2 clinical testing in Duchenne could begin following completion of Phase 1 testing in MDC1A patients.
Where would a clinical trial take place?
We have not made a decision but we anticipate clinical testing to occur at multiple sites.
Where can I learn more about this research?