Solid Biosciences’ (SLDB) Co-Founder, President, and CEO Ilan Ganot gave an inspirational
presentation highlighting the commitment of the company to “solving” Duchenne’s Muscular
Dystrophy (DMD). With a personal stake in finding a therapy that brings benefit to patients,
having a son who was diagnosed with DMD, Ganot stressed the importance of the company’s 360
degree approach for DMD therapies including disease-modifying therapies – to address symptoms
in patients, corrective therapies – to address the genetic cause of DMD, assistive devices to
support mobility, all while improving the understanding of the disease.
The presentation was mainly focused on SGT-001, a micro-dystrophin gene therapy, Solid
Biosciences’ most advanced therapy for DMD, which is currently in a Phase I/II trial. Although no
new data was presented, the initial results on safety and micro-dystrophin expression are
expected later in Q1 2019 and an interim analysis of data from the study should be ready for a H2
2019 readout. Based on animal models, the company is expecting micro-dystrophin expression
levels of about 10% for the lower dose of SGT-001, but it also plans to initiate a dose ascending
study following an analysis of data from the IGNITE DMD phase I/II trial. Data on how microdystrophin translates into a functional benefit is yet to be generated however, Solid Biosciences is
hoping to achieve positive results from secondary endpoints in its IGNITE DMD study. Company
representatives in the Q&A session noted that a key differentiator of the transgene that they
developed is a unique domain that restores and localizes nNOS, which based on preclinical data,
they believe leads to increased functional strength.
The issue of the Suspected Unexpected Serious Adverse Reaction in the first dosed patient in the
IGNITE trial was also addressed. Company representatives in the Q&A session said that although
unfortunate, the event helped the company gain important insights into platelet decreases in
patients on the gene therapy and aided in the management of further patients including enabling
early detection. They noted that it may be due to AAV9, rather than their particular product,
though they did not elaborate. The company did not comment on the frequency of platelet
decreases in patients or on whether patients beyond the first one needed eculizumab to correct
the defect. They said they did not disclose the latter because they want to have a good
understanding of whether it is required or not, and will disclose more information in an update in
Q1.
During the Q&A session when asked about the differentiating factors between SGT-001 and other
therapies the company’s representatives spoke of the inclusion of adolescents in the clinical
trials, a group typically omitted due to the advanced stage of disease, as well as of the superiority
of the preclinical results of the individual components of the therapy, as indicated above.
The presentation ended with a brief overview of the company’s pipeline and plans for further
development. This included an anti-LTBP4 disease-modifying therapy to address fibrosis in DMD
patients and a planned pipeline expansion with next generation screening for new vector
candidates.
presentation highlighting the commitment of the company to “solving” Duchenne’s Muscular
Dystrophy (DMD). With a personal stake in finding a therapy that brings benefit to patients,
having a son who was diagnosed with DMD, Ganot stressed the importance of the company’s 360
degree approach for DMD therapies including disease-modifying therapies – to address symptoms
in patients, corrective therapies – to address the genetic cause of DMD, assistive devices to
support mobility, all while improving the understanding of the disease.
The presentation was mainly focused on SGT-001, a micro-dystrophin gene therapy, Solid
Biosciences’ most advanced therapy for DMD, which is currently in a Phase I/II trial. Although no
new data was presented, the initial results on safety and micro-dystrophin expression are
expected later in Q1 2019 and an interim analysis of data from the study should be ready for a H2
2019 readout. Based on animal models, the company is expecting micro-dystrophin expression
levels of about 10% for the lower dose of SGT-001, but it also plans to initiate a dose ascending
study following an analysis of data from the IGNITE DMD phase I/II trial. Data on how microdystrophin translates into a functional benefit is yet to be generated however, Solid Biosciences is
hoping to achieve positive results from secondary endpoints in its IGNITE DMD study. Company
representatives in the Q&A session noted that a key differentiator of the transgene that they
developed is a unique domain that restores and localizes nNOS, which based on preclinical data,
they believe leads to increased functional strength.
The issue of the Suspected Unexpected Serious Adverse Reaction in the first dosed patient in the
IGNITE trial was also addressed. Company representatives in the Q&A session said that although
unfortunate, the event helped the company gain important insights into platelet decreases in
patients on the gene therapy and aided in the management of further patients including enabling
early detection. They noted that it may be due to AAV9, rather than their particular product,
though they did not elaborate. The company did not comment on the frequency of platelet
decreases in patients or on whether patients beyond the first one needed eculizumab to correct
the defect. They said they did not disclose the latter because they want to have a good
understanding of whether it is required or not, and will disclose more information in an update in
Q1.
During the Q&A session when asked about the differentiating factors between SGT-001 and other
therapies the company’s representatives spoke of the inclusion of adolescents in the clinical
trials, a group typically omitted due to the advanced stage of disease, as well as of the superiority
of the preclinical results of the individual components of the therapy, as indicated above.
The presentation ended with a brief overview of the company’s pipeline and plans for further
development. This included an anti-LTBP4 disease-modifying therapy to address fibrosis in DMD
patients and a planned pipeline expansion with next generation screening for new vector
candidates.
