Preclinical research of Recombinant Human Laminin-111

Recombinant Human Laminin-111 (rhLAM-111) is being developed for two therapeutic areas

1] Protein therapy for LAM-211 deficiency – congenital muscular dystrophy type 1A (MDC1A)

2] Upregulation of utrophin and alpha7beta1 integrin for treatment of DMD

Questions and Answers

What stage is this research?

This research is pre-clinical, we are developing our manufacturing capability but have not yet advanced to clinical trials.

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 among other laboratories in the US, Canada and EU

Prothelia is funded by our company founders, and previous grants from NIH and advocacy groups such as the PPMD End Duchenne GAP program, Struggle Against Muscular Dystrophy (SAM) and Hope for Gus.

What is the goal or purpose of this research?

Manufacture of rhLAM-111 and clinical testing in patients with MDC1A and DMD.

What is the current state of this research?

This project is in preclinical development and we have demonstrated effectiveness in the mdx and dyW mouse models of Duchenne and MDC1A, respectively.

Laminin-111 protein therapy also enhances muscle regeneration and repair in the GRMD dog model of Duchenne.

Current efforts are focused on MDC1A and positive clinical data for MDC1A will accelerate clinical development for DMD.

rhLAM-111 will help all DMD patients regardless of their mutation.

rhLAM-111 has 2 mechanisms of action important for DMD

  1. Stabilizes skeletal and cardiac muscle through upregulation (increased expression) of modifier proteins alpha7beta1 integrin and utrophin.
  2. Direct stimulation of satellite cells and more efficient muscle regeneration.

What steps need to be completed before moving into a clinical trial?

  • Increase the scale of manufacturing
  • Pre-IND testing

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 DMD 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.

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