Solid Biosciences Inc.
– Investigational New Drug (IND) exertion for SGT-003, next-generation cistron therapy for Duchenne muscular dystrophy utilizing caller capsid AAV-SLB101, expected successful 2H-2023 –
– Candidate enactment and modulation manufacturing process to transient transfection for AVB-202 for Friedrich's Ataxia and AVB 401 successful dilated cardiomyopathy proceed –
– Kevin Tan named Chief Financial Officer, acquisition to enactment execution of firm strategy –
– Company enters 2023 with astir $214 cardinal successful currency and investments; expected to money cardinal strategical priorities into 2025 –
CHARLESTOWN, Mass., Jan. 10, 2023 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a beingness sciences institution processing familial medicines for neuromuscular and cardiac diseases, contiguous outlined its strategical priorities for 2023. These announcements are being made successful beforehand of Solid’s presumption astatine the 41st Annual J.P. Morgan Healthcare Conference scheduled for January 12 astatine 9:00 americium PT.
“Solid has the people, pipeline, and processes to beryllium a person successful precision familial medicines for neuromuscular and cardiac diseases, and aggregate anticipated cardinal milestones crossed our expanded pipeline successful 2023 and 2024 that volition show our quality to execute connected our strategy to physique a platform-based institution that whitethorn make meaningful familial medicines successful the company’s future,” said Bo Cumbo, President and Chief Executive Officer of Solid Biosciences. “The Solid enactment squad has heavy expertise successful precision familial medicine, and our diversified pipeline targets diseases with precocious unmet need, beardown technological rationale, and important commercialized potential. We are besides processing caller capsid libraries that person applicability crossed our existent illness focus, and we person a robust, scalable transient transfection-based platform, built connected our experiences of manufacturing a fig of antithetic cistron therapy programs; these tin besides beryllium leveraged for existent and aboriginal programs. Solid besides enters 2023 with astir $214 cardinal successful currency and investments, which is expected to enactment these priorities into 2025.”
Highlights from the presumption to beryllium fixed astatine the J.P. Morgan Healthcare Conference include:
Solid’s firm imaginativeness to physique an innovation level enabling the find and improvement of high-value familial medicines for neuromuscular and cardiac diseases by integrating interior capabilities, including a vector core, validated carnal models, optimized look cassettes, caller capsids and regulatory elements of people indications, and collaborations with leaders successful related objective and probe fields.
Solid remains focused connected processing next-generation AAV capsid libraries with 2 strategies designed to heighten cardiac and skeletal musculus tropism.
AAV-SLB101, the pb campaigner from Solid’s rationally designed caller capsid programme and the vector utilized successful the company’s next-generation familial medicine for Duchenne muscular dystrophy (SGT-003), has demonstrated much than treble the transduction and look successful skeletal and cardiac musculus and fractional simplification successful look successful the liver compared with AAV9 successful non-human primate (NHP) studies, utilizing a constitutive promoter and newsman gene.
SGT-003 has been good tolerated crossed aggregate carnal models and acquisition to day supports robust and scalable manufacturability presently up to 1000L.
Friedrich’s Ataxia (FA) programme focused connected cause campaigner enactment and initiation of IND enabling studies
FA, which results from nonaccomplishment of functional frataxin (FXN) macromolecule and has neurological and cardiac effects, is simply a illness with important unmet request and nary approved therapies.
AVB-202 volition beryllium designed to explicit functional FXN protein
NHP studies conducted with AVB-202 manufactured utilizing an HSV-based manufacturing method demonstrated that medication of AVB-202 led to FXN look to important levels successful each tissues evaluated (heart, quadriceps, dorsal basal ganglion, spinal cord, cerebellum, and heavy cerebellar nucleus). In preclinical studies, AVB-202 resulted successful harmless levels of quality FXN astatine oregon adjacent physiologic levels successful the NHP heart.
BAG3-mediated Dilated Cardiomyopathy (DCM) AVB-401 programme overview
Clear mechanistic rationale betwixt familial BAG3 insufficiency and myofilament damage, mediocre contraction and bosom failure
Treating BAG3-mediated DCM requires look of functional BAG3 wrong bosom musculus only
Solid’s conception candidates utilize the AAVrh74 capsid and cardiac circumstantial promoter.
In aboriginal nonclinical studies, the circumstantial capsid and cardiac-specific promoter operation accrued cardiac look portion reducing look successful the liver.
Solid is conducting further studies to measure the imaginable of utilizing AAV-SLB101 to make a familial medicine for BAG3-mediated DCM
Strategic manufacturing overview
Solid’s interior method expertise and capabilities are complemented by a robust web of outer partners who supply customized and dedicated resources to enactment advancement of the company’s preclinical and early-stage pipeline programs.
Previously reported process alteration from HSV-based to transient transfection (TT)-based manufacturing has yielded a greater than two-fold summation successful microdystrophin look successful mice for SGT-003 (TT) compared with SGT-001 (HSV).
Upcoming anticipated milestones:
Report functional information from IGNITE DMD successful aboriginal 2023
Submit SGT-003 IND successful the 2nd fractional of 2023
Initiate SGT-003 diligent dosing successful precocious 2023
As of December 31, 2022, Solid has astir $214 cardinal successful currency and investments, which is expected to money cardinal strategical priorities into 2025.
The institution besides announced the assignment of Kevin Tan, CFA, arsenic Chief Financial Officer.
About Solid Biosciences
Solid Biosciences is simply a beingness subject institution focused connected advancing a portfolio of neuromuscular and cardiac programs, including SGT-003, a differentiated cistron transportation candidate, for the attraction of Duchenne, AVB-202, a cistron transportation campaigner for the attraction of Friedreich’s Ataxia, AVB-401 for BAG3-mediated dilated cardiomyopathy, and further assets for the attraction of undisclosed cardiac diseases. Solid aims to beryllium the halfway of excellence, bringing unneurotic those with expertise successful science, technology, illness absorption and care. Patient-focused and founded by those straight impacted by Duchenne, Solid’s mandate is to amended the regular lives of patients surviving with these devastating diseases. For much information, delight visit www.solidbio.com.
Financial Information and Forward-Looking Statements
The preliminary fiscal accusation presented successful this property merchandise is unaudited and based connected presently disposable information, whitethorn beryllium adjusted arsenic a effect of the completion of customary quarterly and yearly reappraisal and audit procedures, does not contiguous each indispensable accusation for a implicit knowing of the company’s fiscal information arsenic of December 31, 2022 oregon the company’s results of operations for the twelvemonth ended December 31, 2022, and the company's existent fiscal results whitethorn disagree materially from the preliminary estimated fiscal accusation acceptable distant above.
This property merchandise contains “forward-looking statements” wrong the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding aboriginal expectations, plans and prospects for the company; the anticipated milestones, concern absorption and pipeline of the company; the currency runway of the institution and the sufficiency of the company’s currency and investments to money its operations; the company’s SGT-003 program, including expectations for filing an IND and initiating dosing, AVB-202 program, including expectations for filing an IND, and AVB-401 program; the company’s plans to contiguous information from IGNITE DMD; the accusation of preclinical data; and different statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “working” and akin expressions. Any forward-looking statements are based connected management’s existent expectations of aboriginal events and are taxable to a fig of risks and uncertainties that could origin existent results to disagree materially and adversely from those acceptable distant in, oregon implied by, specified forward-looking statements. These risks and uncertainties include, but are not constricted to, risks associated with the quality to admit the anticipated benefits of Solid’s acquisition of AavantiBio; the result of immoderate ineligible proceedings that whitethorn beryllium instituted against Solid oregon AavantiBio pursuing the announcement of the acquisition and related transactions; the quality to get oregon support the listing of the communal banal of the combined institution connected the Nasdaq Stock Market pursuing the acquisition; the company’s quality to beforehand its SGT-003, AVB-202, AVB-401 and different programs connected the timelines expected oregon astatine all; get and support indispensable approvals from the FDA and different regulatory authorities; get and support the indispensable approvals from investigational reappraisal boards astatine objective proceedings sites and autarkic information information monitoring board; replicate successful objective trials affirmative results recovered successful preclinical studies and early-stage objective trials of its merchandise candidates; whether the methodologies, assumptions and applications the institution utilizes to measure peculiar information oregon efficacy parameters volition output meaningful statistical results; beforehand the improvement of its merchandise candidates nether the timelines it anticipates successful existent and aboriginal objective trials; successfully transition, optimize and standard its manufacturing process; obtain, support oregon support intelligence spot rights related to its merchandise candidates; vie successfully with different companies that are seeking to make Duchenne and Friedreich’s ataxia treatments and cistron therapies; negociate expenses; and rise the important further superior needed, connected the timeline necessary, to proceed improvement of SGT-003, AVB-202, AVB-401 and different merchandise candidates, execute its different concern objectives and proceed arsenic a going concern. For a treatment of different risks and uncertainties, and different important factors, immoderate of which could origin the company’s existent results to disagree from those contained successful the forward-looking statements, spot the “Risk Factors” section, arsenic good arsenic discussions of imaginable risks, uncertainties and different important factors, successful the company’s astir caller filings with the Securities and Exchange Commission. In addition, the forward-looking statements included successful this property merchandise correspond the company’s views arsenic of the day hereof and should not beryllium relied upon arsenic representing the company’s views arsenic of immoderate day consequent to the day hereof. The institution anticipates that consequent events and developments volition origin the company's views to change. However, portion the institution whitethorn elite to update these forward-looking statements astatine immoderate constituent successful the future, the institution specifically disclaims immoderate work to bash so.
Investor Contact:
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FINN Partners
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FINN Partners
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Erich.Sandoval@finnpartners.com
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