Solid Bioscience Stock Price, News & Analysis

Solid Biosciences Inc. (NASDAQ: SLDB) is a precision genetic medicine company focused on developing gene therapy candidates for rare neuromuscular and cardiac diseases. According to the company’s public disclosures, its portfolio includes investigational programs for Duchenne muscular dystrophy (Duchenne), Friedreich’s ataxia (FA), catecholaminergic polymorphic ventricular tachycardia (CPVT), TNNT2-mediated dilated cardiomyopathy and additional fatal, genetic neuromuscular and cardiac diseases. Solid Biosciences is listed on the Nasdaq Global Select Market under the ticker symbol SLDB.

Business focus and therapeutic areas

Solid Biosciences describes itself as a life sciences and precision genetic medicine company developing gene therapy candidates for devastating rare diseases. Its work centers on neuromuscular and cardiac indications where there is significant unmet medical need. The company’s programs target conditions such as Duchenne, a genetic muscle-wasting disease predominantly affecting boys, Friedreich’s ataxia, a degenerative multisystem disease, CPVT, a rare inherited arrhythmia, and TNNT2-mediated dilated cardiomyopathy.

The company’s stated mission is to improve the daily lives of patients living with devastating rare diseases. It reports that it was founded by individuals directly impacted by Duchenne, and that it aims to unite experts in science, technology, disease management and care while advancing its pipeline and delivery platform.

Gene therapy pipeline

Solid Biosciences highlights several named investigational gene therapy candidates in its communications:

• SGT-003 for Duchenne muscular dystrophy (Duchenne). SGT-003 is described as an investigational gene therapy containing a differentiated microdystrophin construct and a proprietary, next-generation capsid, AAV-SLB101. The capsid was rationally designed to target integrin receptors and has shown enhanced cardiac and skeletal muscle transduction with decreased liver targeting in nonclinical studies. The microdystrophin construct includes the R16/17 binding domain, which localizes neuronal nitric oxide synthase (nNOS) to the muscle membrane.
• SGT-212 for Friedreich’s ataxia (FA). SGT-212 is described as a first-in-class, recombinant AAV-based gene replacement therapy designed to deliver full-length human frataxin (FXN) via a dual route of administration. It employs intradentate nucleus (IDN) infusion using an FDA-approved, stereotactic, precision MRI-guided neurosurgical device, followed by an intravenous (IV) infusion. The intent is to increase therapeutic FXN levels in the cerebellar dentate nuclei, cardiomyocytes and other systemic tissues to address neurologic, cardiac and systemic manifestations of FA.
• SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT). SGT-501 is described as a novel investigational gene therapy intended to augment CASQ2 protein levels to address ryanodine receptor (RYR2) instability and calcium dysregulation seen in CPVT.
• SGT-601 for TNNT2-mediated dilated cardiomyopathy. Public descriptions list SGT-601 among the company’s pipeline of gene therapy candidates targeting rare genetic cardiac disease.

In addition to these named programs, Solid Biosciences states that it is advancing additional gene therapy candidates for fatal, genetic neuromuscular and cardiac diseases.

Platform technologies and AAV-SLB101 capsid

Beyond individual product candidates, Solid Biosciences emphasizes its work on platform technologies designed to enhance gene therapy delivery. The company reports that it is developing libraries of genetic regulators and other enabling technologies with potential to significantly impact gene therapy delivery across the industry.

A central component of this platform is AAV-SLB101, the company’s proprietary, rationally designed next-generation capsid. AAV-SLB101 is described as being developed for enhanced skeletal muscle and cardiac tropism with reduced biodistribution to the liver. Company disclosures note that AAV-SLB101 has demonstrated increased transduction speed, enhanced skeletal and cardiac muscle tropism, decreased liver biodistribution and improved efficiency in preclinical models when compared to first-generation capsids.

AAV-SLB101 is used within SGT-003 and has been evaluated in the INSPIRE DUCHENNE Phase 1/2 clinical trial. Solid Biosciences reports that, as of specified safety cutoff dates, AAV-SLB101 has been generally well tolerated in participants dosed with SGT-003 and has shown compelling levels of vector transduction and protein expression, with reduced liver impact in available data sets.

The company also reports that it is licensing AAV-SLB101 to external partners. Public announcements describe non-exclusive agreements with corporations, institutions and academic laboratories, including a license and collaboration with Andelyn Biosciences, a cell and gene therapy contract development and manufacturing organization, allowing Andelyn to provide AAV-SLB101 access to its gene therapy clients.

Clinical development activities

Solid Biosciences’ pipeline is supported by multiple clinical-stage programs:

• INSPIRE DUCHENNE: a Phase 1/2 first-in-human, open-label, single-dose, multicenter clinical trial evaluating the safety, tolerability and efficacy of SGT-003 in pediatric participants with Duchenne muscular dystrophy at a specified dose level. SGT-003 is administered as a one-time intravenous infusion. Company filings describe interim clinical data, including microdystrophin expression, restoration of components of the dystrophin-associated protein complex and changes in biomarkers of muscle integrity, as well as an interim safety profile.
• IMPACT DUCHENNE: a Phase 3 randomized, double-blind, placebo-controlled ex-U.S. clinical trial assessing SGT-003 in pediatric participants outside the United States. Public disclosures note regulatory approvals to conduct this trial in Canada and Australia, with plans to expand to additional countries subject to regulatory approvals.
• FALCON: a first-in-human, open-label, multi-center Phase 1b clinical trial designed to evaluate the safety and tolerability of SGT-212 in participants diagnosed with Friedreich’s ataxia and cardiac hypertrophy. The trial is being conducted in the United States.
• ARTEMIS: a first-in-human, open-label, Phase 1b clinical trial evaluating the safety, tolerability and efficacy of SGT-501 for the treatment of CPVT. Company communications describe trial site activation and participant screening.

In addition, Solid Biosciences has reported regulatory designations for SGT-212, including U.S. Food and Drug Administration Fast Track, Rare Pediatric Disease and Orphan Drug designations, reflecting its development in a rare, serious disease setting. The company has also reported that SGT-003 has received the U.K. Innovation Passport Designation under the Innovative Licensing and Access Pathway.

Disease areas addressed

Solid Biosciences’ programs address specific rare diseases described in its public materials:

• Duchenne muscular dystrophy is described as a genetic muscle-wasting disease predominantly affecting boys, with symptoms usually appearing between three and five years of age. It is characterized as progressive, irreversible and ultimately fatal, with an estimated prevalence of thousands of cases in the United States.
• Friedreich’s ataxia is characterized as an inherited, life-threatening, degenerative multisystem disease caused by variants in the frataxin gene that disrupt production of the frataxin protein, a mitochondrial iron-binding protein involved in essential cellular processes including energy production. It is associated with progressive nervous system damage, movement problems and cardiac dysfunction, with cardiac complications identified as a primary cause of death.
• Catecholaminergic polymorphic ventricular tachycardia (CPVT) is described in company materials as a condition involving RYR2 instability and calcium dysregulation, for which SGT-501 is intended to augment CASQ2 protein levels.
• TNNT2-mediated dilated cardiomyopathy is listed as a target of SGT-601 in the company’s cardiac gene therapy pipeline.

Corporate status and listing

According to a Form 8-K filed with the U.S. Securities and Exchange Commission, Solid Biosciences’ common stock is registered under Section 12(b) of the Securities Exchange Act of 1934 and trades on the Nasdaq Global Select Market under the symbol SLDB. The filing lists the company’s class of securities as common stock with a stated par value per share.

The company has also reported inducement grants of restricted stock units to newly hired employees under a stock incentive plan, made as inducements material to employment in accordance with Nasdaq Listing Rule 5635(c)(4), indicating ongoing use of equity-based compensation.

Engagement with scientific and regulatory communities

Solid Biosciences participates in scientific and industry forums to present data from its programs and platform. Public announcements describe presentations and posters at the Global CardioVascular Clinical Trialists Forum, focusing on AAV-SLB101 and cardiac gene therapy, as well as participation in the J.P. Morgan Healthcare Conference, where the company provides corporate and pipeline updates.

The company has also highlighted its role in advocacy around Duchenne newborn screening, noting long-term involvement with a committee dedicated to implementing newborn screening for Duchenne and support for efforts that contributed to Duchenne being added to the U.S. Recommended Uniform Screening Panel.

Patient-centered orientation

In its descriptions, Solid Biosciences emphasizes a patient-focused orientation and notes that it was founded by individuals directly impacted by Duchenne. The company states that it seeks to unite stakeholders across science, technology, disease management and care, and that its work on gene therapy candidates and enabling technologies is directed toward improving outcomes and daily life for people living with rare neuromuscular and cardiac diseases.