LAMININ-111 - Laminin-111, Integrin and Utrophin as a Potential Therapy for Duchenne

Frequently Asked Questions (FAQs) about this research:
 

What stage is this research?

  • This research is pre-clinical, we are developing our manufacturing capability but we 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’s funding has been primarily through NIH grants and advocacy groups including PPMD’s End Duchenne GAP program, Struggle Against Muscular Dystrophy (SAM) and Hope for Gus.

 

What is the goal or purpose of this research?

  • Our primary goal is production of rhLAM-111 for IND enabling studies and Phase 1 clinical trials.
  • We continue to generate new animal efficacy data in MDC1A and DMD animal models.

 

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. Current efforts are focused on MDC1A and positive clinical data for MDC1A may accelerate clinical development for DMD.  
  • The mechanism of action of rhLAM-111 in DMD is through 1] direct stimulation of satellite cells and muscle regeneration and 2] upregulation (increased expression) of the DMD modifier proteins alpha7beta1 integrin and utrophin. Both integrin and utrophin work together to restore lost muscle cell adhesion when dystrophin is missing at the muscle membrane. This approach should help all patients regardless of their dystrophin Glossary Link mutation.

 

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

  • We are currently building the manufacturing process to produce sufficient rhLAM-111 for all non-clinical purposes and phase 1 clinical needs.

 

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/2020 with proof of concept anticipated in 2022.  Phase 2 clinical testing in DMD could begin following completion of Phase 1 clinical testing and with available funding.  

 

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?

 

(Updated 5/4/2017)