- Duchenne (SGT-003): First participant dosed in the Phase 3 IMPACT DUCHENNE clinical trial, receipt of positive opinion on the Company's Pediatric Investigation Plan from the European Medicines Agency and Orphan drug designation from the European Commission mark important advancements in the program's global development -
- Continued progress achieved across the SGT-003 development program, including dosing of 47 participants in Phase 1/2 INSPIRE DUCHENNE clinical trial, and ongoing engagement with the FDA for guidance on a potential accelerated approval pathway for SGT-003 -
- Friedreich's Ataxia (SGT-212): Second participant dosed in the Phase 1b FALCON clinical trial; SGT-212 has been well tolerated with no serious adverse events observed -
- CPVT (SGT-501): Company anticipates dosing the first participant in the Phase 1b ARTEMIS clinical trial evaluating SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT) in H2 2026 -
- Capital Position: Cash, cash equivalents and available-for-sale securities of $380.7 million at March 31, 2026; the Company's cash runway is anticipated to extend into H1 2028 -
CHARLESTOWN, Mass., May 12, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the "Company" or "Solid"), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the first quarter ended March 31, 2026, and provided a business update.
Bo Cumbo, President and CEO of Solid Biosciences, stated: "The beginning of 2026 has seen Solid make meaningful strides as we continue to execute across our pipeline and platform development efforts. In Duchenne, we dosed the first participant in our Phase 3 IMPACT DUCHENNE clinical trial for SGT-003 and received Orphan drug designation from the European Commission, representing important milestones in the program's global advancement. We also continue to enroll and dose participants in the Phase 1/2 INSPIRE DUCHENNE clinical trial, which has maintained a consistent and encouraging safety and tolerability profile.
"As we progress through the year, we look forward to ongoing engagement with the FDA, focusing on our thoughtful approach to generating rigorous, well-controlled clinical data through our integrated, multi-country, multi-trial development program. We recognize the significant unmet need in Duchenne and see the potential for our novel therapy to offer a differentiated treatment option for families living with this unrelenting disease.
"In parallel, we are working closely with the UK Medicines and Healthcare products Regulatory Agency (MHRA) under our ILAP Innovation Passport and recently received a positive opinion from the European Medicines Agency $(EMA)$ on our Pediatric Investigation Plan, reflecting our efforts to inform potential development and regulatory pathways outside of the United States. We continue to build the organizational and geographic foundations to support future global development and have made the decision to establish dedicated ex-U.S. leadership with the planned addition of a Head of Europe.
"Beyond Duchenne, we have now dosed two participants in the Phase 1b FALCON clinical trial of our Friedreich's ataxia gene therapy program, SGT-212, which has been well tolerated by both participants. While still early, we are encouraged by initial clinical observations and remain focused on advancing the program thoughtfully as data emerge. Across our company, we continue to execute our plan and advance our programs with the fiscal discipline and operational rapidity required to deliver meaningful outcomes to patients and our shareholders," Mr. Cumbo concluded.
Company Updates
Neuromuscular Pipeline
SGT-003 Next-Generation Duchenne Muscular Dystrophy (Duchenne) Program
-- As announced on May 7, 2026, the Company reported that the first
participant was dosed in IMPACT DUCHENNE, the Phase 3 multi-country,
placebo-controlled, randomized, double-blind, clinical trial.
-- Solid has received a positive opinion from the European Medicines
Agency's Paediatric Committee on its Pediatric Investigation Plan
$(PIP)$, establishing alignment on its pediatric development
framework for SGT-003 in Europe.
-- The Company announced on April 28, 2026, that SGT-003 had been granted
Orphan drug designation by the European Commission $(EC)$.
-- SGT-003 has received numerous regulatory designations from global
health authorities, including the Innovation Passport designation
under the new U.K. Innovation License and Access Pathway program
and Fast Track, Orphan Drug and Rare Pediatric Disease
designations from the U.S. Food and Drug Administration.
-- SGT-003 continued to be generally well tolerated in the 47 participants
dosed in the INSPIRE DUCHENNE trial as of May 11, 2026.
-- The 47th participant was dosed on May 8, 2026.
-- The Company reported positive interim data from the ongoing Phase 1/2
INSPIRE DUCHENNE clinical trial on March 11, 2026, which continued to
suggest differentiated clinical, safety and tolerability profiles.
-- The Company announced on February 9, 2026, that it had reached alignment
with the U.S. Food and Drug Administration (FDA) on the overall study
design for IMPACT DUCHENNE.
-- Solid expects to continue discussions with the FDA to receive guidance on
a potential accelerated approval pathway for SGT-003 and expects to
provide an update as discussions progress.
SGT-212 for Friedreich's ataxia $(FA)$
-- Two participants have been dosed in the Phase 1b FALCON clinical trial,
and SGT-212 has been well tolerated as of May 11, 2026, with no serious
adverse events observed.
-- Participant dosing remains ongoing with initial data expected by year-end
2026, subject to participant enrollment.
Cardiac Pipeline
SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT)
-- Clinical trial sites have been activated and participant screening is
underway for the Phase 1b ARTEMIS trial.
-- The first participant is anticipated to be dosed in the second half of
2026, with initial safety data anticipated in the first half of 2027,
subject to participant enrollment.
Platform Technologies -- Capsids
-- In March 2026, AAV-SLB101, the Company's next-generation, muscle-tropic
capsid used in SGT-003, was renamed POLARIS-101$(TM)$.
-- Solid has executed more than 50 agreements, including licenses, with
corporations, institutions and academic labs for the use of
POLARIS-101(TM).
First Quarter 2026 Financial Highlights
-- Cash Position: Solid had $380.7 million in cash, cash equivalents, and
available-for-sale securities as of March 31, 2026, compared to $187.9
million as of December 31, 2025. The Company expects that its existing
cash, cash equivalents, and available-for-sale securities will be
sufficient to fund its operational runway into the first half of 2028.
-- Research and Development (R&D) Expenses: R&D expenses for the first
quarter of 2026 were $46.1 million, compared to $30.9 million for the
first quarter of 2025. The increase of $15.2 million in research and
development expenses was primarily due to a $13.6 million increase in
costs for SGT-003 primarily related to manufacturing and clinical costs,
a $3.3 million increase in personnel related expenses, a $2.5 million
increase in costs for SGT-212 primarily related to clinical costs and
license and milestone payments, and a $0.4 million increase in external
expenses primarily related to consulting and professional services,
partially offset by a $3.1 million decrease in costs for SGT-501
primarily related to lower manufacturing and research costs, and a $1.7
million decrease in costs for SGT-601 related to lower manufacturing and
research costs.
-- General and Administrative (G&A) Expenses: G&A expenses for the first
quarter of 2026 were $11.2 million, compared to $9.1 million for the
first quarter of 2025. The increase of $2.0 million was primarily related
to a $1.5 million increase in personnel related costs, a $0.5 million
increase in consulting services, and a $0.4 million increase in equipment
costs, partially offset by a $0.5 million decrease in legal fees.
-- Net Loss: Net loss for the first quarter of 2026 was $56.7 million,
compared to a net loss of $39.3 million for the first quarter of 2025.
About SGT-003
SGT-003 is an investigational gene therapy containing a novel microdystrophin construct and a proprietary, next-generation capsid, POLARIS-101(TM) (formerly known as AAV-SLB101), which was rationally designed to target integrin receptors, and has shown enhanced cardiac and skeletal muscle transduction with decreased liver targeting in data from the Phase 1/2 INSPIRE DUCHENNE clinical trial and in nonclinical studies. SGT-003's microdystrophin construct uniquely includes the R16/17 domains, which localize nNOS to the muscle. Nonclinical studies have shown that nNOS can improve blood flow to the muscle thereby reducing muscle breakdown from ischemia and muscle fatigue. Together, these design features suggest that SGT-003 could be a potential best-in-class investigational gene therapy for the treatment of Duchenne.
About the SGT-003 Development Program
The SGT-003 clinical development program consists of two multinational clinical trials -- the Phase 1/2 INSPIRE DUCHENNE trial and the Phase 3 IMPACT DUCHENNE trial -- which together were designed to generate a comprehensive data package to support potential global regulatory authorizations.
(MORE TO FOLLOW) Dow Jones Newswires
May 12, 2026 16:08 ET (20:08 GMT)
Comments