Solid Biosciences(SLDB)

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Solid Biosciences(SLDB) - 2024 Q1 - Quarterly Report
2024-05-15 11:30
UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _________ to _________ Commission File Number: 001-38360 Solid Biosciences Inc. (Exact Name of Registrant as Specified in its Charter) Delaware 90-0943402 (Stat ...
Solid Biosciences(SLDB) - 2023 Q4 - Annual Report
2024-03-13 11:30
[PART I](index=9&type=section&id=PART%20I) This part provides an overview of the company's business, pipeline, intellectual property, competition, and regulatory environment, along with key risk factors, corporate information, and legal disclosures [Item 1. Business](index=9&type=section&id=Item%201.%20Business) Solid Biosciences develops gene therapies for rare neuromuscular and cardiac diseases, expanding its pipeline through innovation and acquisitions - Solid Biosciences is a life sciences company focused on advancing gene therapy candidates for Duchenne muscular dystrophy (SGT-003), Catecholaminergic polymorphic ventricular tachycardia (SGT-501), and other rare neuromuscular and cardiac diseases[22](index=22&type=chunk)[550](index=550&type=chunk) - The company's corporate vision is to build an innovation platform for high-value genetic medicines, integrating internal capabilities (vector core, animal models, optimized expression cassettes, novel capsids, regulatory expertise) and external collaborations[23](index=23&type=chunk)[551](index=551&type=chunk) - In December 2022, Solid Biosciences acquired AavantiBio, Inc., adding gene therapy programs for Friedreich's ataxia (AVB-202-TT) and BAG3-mediated dilated cardiomyopathy (AVB-401), along with related platform technologies[23](index=23&type=chunk)[551](index=551&type=chunk) [Overview](index=9&type=section&id=Overview) Solid Biosciences develops gene therapies for rare neuromuscular and cardiac diseases, driven by patient needs and strategic acquisitions - Solid Biosciences is focused on advancing gene therapy candidates for Duchenne muscular dystrophy (SGT-003), Catecholaminergic polymorphic ventricular tachycardia (SGT-501), and other cardiac/neuromuscular diseases[22](index=22&type=chunk)[550](index=550&type=chunk) - The company's mission is to improve the daily lives of patients with devastating neuromuscular and cardiac diseases, guided by fundamental principles including identifying meaningful therapies and collaborating with leading experts[22](index=22&type=chunk)[25](index=25&type=chunk)[550](index=550&type=chunk)[553](index=553&type=chunk) - The acquisition of AavantiBio, Inc. in December 2022 expanded Solid's pipeline to include AVB-202-TT for Friedreich's ataxia and AVB-401 for BAG3-mediated dilated cardiomyopathy, along with related platform technologies[23](index=23&type=chunk)[551](index=551&type=chunk) [Our Pipeline](index=9&type=section&id=Our%20Pipeline) The pipeline features gene transfer products for rare neuromuscular and cardiac diseases, utilizing modified AAV capsids for functional gene delivery - The company's current programs are gene transfer products designed to treat rare neuromuscular and cardiac diseases by delivering functional transgenes using viral capsids (vectors)[24](index=24&type=chunk)[552](index=552&type=chunk) - Adeno-associated virus (AAV) capsids are used as vehicles to deliver transgenes, modified to no longer self-replicate but retain the ability to introduce new genetic material into patient cells[24](index=24&type=chunk)[552](index=552&type=chunk) - AAV capsids have been approved for systemic delivery of transgenes and extensively studied in human clinical trials for multiple disease indications by third parties[24](index=24&type=chunk)[552](index=552&type=chunk) [Lead Neuromuscular Program](index=9&type=section&id=Lead%20Neuromuscular%20Program) The lead neuromuscular program, SGT-003 for Duchenne, cleared IND in November 2023, with Phase 1/2 trial dosing expected in Q2 2024 - Duchenne muscular dystrophy is a progressive, ultimately fatal genetic muscle-wasting disease affecting approximately **1 in 3,500 to 5,000 live male births**, with no cure and limited satisfactory treatments[25](index=25&type=chunk)[26](index=26&type=chunk)[27](index=27&type=chunk) - SGT-003 is designed to address the underlying genetic cause of Duchenne by delivering a synthetic microdystrophin transgene, expressed only in skeletal, cardiac, and respiratory muscles, using a novel AAV-SLB101 capsid for enhanced muscle tropism and reduced liver uptake[30](index=30&type=chunk)[33](index=33&type=chunk)[34](index=34&type=chunk) - The IND for SGT-003 was cleared in November 2023, and the Phase 1/2 INSPIRE Duchenne trial is expected to dose its first patient in **Q2 2024**, with initial safety updates in mid-2024 and initial data in **H2 2024**. SGT-003 has Orphan Drug and Fast Track designations[37](index=37&type=chunk)[38](index=38&type=chunk) [Lead Cardiac Program](index=12&type=section&id=Lead%20Cardiac%20Program) The lead cardiac program, SGT-501 for CPVT, aims to prevent arrhythmias with an AAV8 capsid, with IND submission anticipated in early 2025 - CPVT is a rare, life-threatening inherited arrhythmia syndrome, primarily affecting children, characterized by stress-induced arrhythmias and high mortality (up to **50%**)[40](index=40&type=chunk)[41](index=41&type=chunk) - SGT-501 is a gene therapy candidate for CPVT-1 (RYR2 gene mutation) and CPVT-2 (CASQ2 gene mutation), using an AAV8 capsid to deliver a functional CASQ2 transgene to regulate cardiac calcium and prevent arrhythmias[44](index=44&type=chunk)[45](index=45&type=chunk)[46](index=46&type=chunk) - Preclinical data supports SGT-501's development, with an IND submission for CPVT-1 anticipated in **early 2025**. SGT-501 has Orphan Drug designation from the FDA and EMA[48](index=48&type=chunk) [Other Candidates](index=13&type=section&id=Other%20Candidates) The company is developing preclinical candidates for rare cardiac diseases (AVB-401, SGT-601, SGT-701) and Friedreich's ataxia - Preclinical cardiac candidates include AVB-401 for BAG3-mediated DCM (using AAVrh74 capsid), SGT-601 for TNNT2 DCM, and SGT-701 for RBM20 DCM[49](index=49&type=chunk)[50](index=50&type=chunk) - A neuromuscular gene transfer program for Friedreich's ataxia (FA) is in preclinical development, aiming to target neurological and cardiac impairments through dual intravenous and intrathecal administration[51](index=51&type=chunk) [Platform Technologies](index=15&type=section&id=Platform%20Technologies) Platform technologies, including novel capsid libraries and dual gene expression, aim to enhance muscle tropism and reduce liver biodistribution - The company is developing novel capsid libraries and dual gene expression technology to package multiple transgenes into one capsid, aiming to enhance skeletal and/or cardiac muscle tropism[52](index=52&type=chunk)[53](index=53&type=chunk) - Preclinical data from novel capsid programs, including AAV-SLB101 (used in SGT-003), demonstrated increased muscle tropism, decreased liver biodistribution, and improved efficiency compared to AAV9[53](index=53&type=chunk)[54](index=54&type=chunk)[55](index=55&type=chunk) [Manufacturing and Supply](index=15&type=section&id=Manufacturing%20and%20Supply) Solid Biosciences is optimizing its transient transfection manufacturing process for gene therapy candidates and relies on third-party CMOs for drug product production, aiming for commercial-scale supply - The company is developing and optimizing a transient transfection manufacturing process for SGT-003, SGT-501, and other candidates to increase yield, robustness, and scalability[57](index=57&type=chunk)[58](index=58&type=chunk) - Solid Biosciences relies on third-party CMOs for clinical stage manufacture and plans to continue this reliance for future candidates, with a goal to establish commercial-scale supply capability[59](index=59&type=chunk)[60](index=60&type=chunk) [Intellectual Property](index=16&type=section&id=Intellectual%20Property) Solid Biosciences' commercial success depends on obtaining and maintaining intellectual property protection for its gene therapy candidates and platform technologies through patents and trade secrets - The company's commercial success depends on obtaining and maintaining intellectual property protection for its pipeline programs and platform technologies, operating without infringement, and preventing others from infringing its rights[61](index=61&type=chunk) - As of February 29, 2024, the patent portfolio includes owned and in-licensed patent families for Duchenne (microdystrophin, promoter sequences), CPVT, and modified AAV capsids[62](index=62&type=chunk)[68](index=68&type=chunk)[69](index=69&type=chunk)[71](index=71&type=chunk) - Patent prosecution is a lengthy process, and there is no guarantee that pending applications will result in issued patents or effectively prevent competitors. Patent terms are generally **20 years** from filing, with potential for extensions upon FDA approval[63](index=63&type=chunk)[64](index=64&type=chunk)[65](index=65&type=chunk) [Strategic Partnerships and Collaborations/Licenses](index=17&type=section&id=Strategic%20Partnerships%20and%20Collaborations%2FLicenses) Solid Biosciences has licensing agreements with various institutions and a key collaboration with Ultragenyx for Duchenne gene therapies, involving milestones and royalties - Solid Biosciences has licensing agreements with the University of Washington, University of Missouri, University of Florida, and Maugeri, involving annual fees, milestone payments, and royalties for its gene therapy candidates[72](index=72&type=chunk)[75](index=75&type=chunk)[81](index=81&type=chunk)[87](index=87&type=chunk)[94](index=94&type=chunk)[796](index=796&type=chunk)[801](index=801&type=chunk)[811](index=811&type=chunk)[817](index=817&type=chunk) - The collaboration with Ultragenyx Pharmaceutical Inc. (October 2020) granted an exclusive worldwide license for AAV8-based microdystrophin constructs for Duchenne, with Solid retaining rights for SGT-003[96](index=96&type=chunk)[103](index=103&type=chunk)[741](index=741&type=chunk) - The Ultragenyx collaboration includes up to **$255.0 million** in cumulative milestone payments per product and tiered royalties. Solid has options to co-fund development for profit sharing or increased royalties[98](index=98&type=chunk)[99](index=99&type=chunk)[100](index=100&type=chunk)[745](index=745&type=chunk)[746](index=746&type=chunk)[747](index=747&type=chunk) [Competition](index=23&type=section&id=Competition) The biotechnology industry is highly competitive, with Solid Biosciences facing larger, better-funded competitors in Duchenne (Sarepta, Pfizer) and CPVT (Armgo, Cardurion) - The biotechnology and pharmaceutical industries are highly competitive, with Solid Biosciences facing competition from larger, better-funded companies, academic institutions, and research institutions[104](index=104&type=chunk)[105](index=105&type=chunk)[351](index=351&type=chunk)[352](index=352&type=chunk) - In Duchenne, Sarepta Therapeutics received accelerated FDA approval for ELEVIDYS in **June 2023** and is seeking to broaden its indication. Pfizer and Genethon also have product candidates in Phase 3 and Phase 1/2/3 clinical development, respectively[106](index=106&type=chunk)[353](index=353&type=chunk) - For CPVT, competitors include Armgo Pharmaceuticals, Inc. and Cardurion Pharmaceuticals, Inc., both with small molecule product candidates in Phase 2 clinical trials[107](index=107&type=chunk)[353](index=353&type=chunk) [Government regulation and product licensure](index=23&type=section&id=Government%20regulation%20and%20product%20licensure) Biologic product development is subject to extensive U.S., EU, and UK regulations, including preclinical testing, multi-phase clinical trials, manufacturing compliance, and post-approval oversight - Biologic products, including gene therapies, are licensed by the FDA under the PHS Act and regulated under the FD&C Act, governing testing, manufacturing, safety, efficacy, labeling, and promotion[108](index=108&type=chunk) - The U.S. biologic product development process involves preclinical studies (GLP), IND submission, IRB/IBC approval, human clinical trials (GCPs) in phases (1, 2, 3), BLA submission, FDA review, manufacturing facility inspection (cGMP), and post-approval requirements[109](index=109&type=chunk)[110](index=110&type=chunk)[111](index=111&type=chunk)[114](index=114&type=chunk)[117](index=117&type=chunk)[145](index=145&type=chunk) - Special regulations for gene therapy products include CBER oversight, NIH Guidelines, specific FDA guidance documents (e.g., for rare diseases, neurodegenerative diseases), and recommendations for long-term patient monitoring (up to **5-15 years** for AAV capsids)[133](index=133&type=chunk)[134](index=134&type=chunk)[137](index=137&type=chunk) - Expedited programs like Fast Track, Breakthrough Therapy, Accelerated Approval, and Regenerative Advanced Therapy designations exist but do not change approval standards or guarantee faster development/approval[169](index=169&type=chunk)[171](index=171&type=chunk)[172](index=172&type=chunk) - Orphan Drug Designation provides **7 years** of market exclusivity in the U.S. and **10 years** in the EU for rare diseases, but does not guarantee approval or prevent competition from clinically superior or different products[164](index=164&type=chunk)[165](index=165&type=chunk)[167](index=167&type=chunk)[207](index=207&type=chunk)[208](index=208&type=chunk)[209](index=209&type=chunk) - Post-approval, products are subject to ongoing regulatory requirements for manufacturing (cGMP), labeling, advertising, safety reporting, and potential REMS. Non-compliance can lead to severe penalties[176](index=176&type=chunk)[177](index=177&type=chunk)[301](index=301&type=chunk)[302](index=302&type=chunk)[303](index=303&type=chunk) - The company is subject to federal and state data privacy laws (HIPAA, CCPA, CPRA) in the U.S. and GDPR in the EU/UK, which impose strict requirements on personal data processing and transfers, with potential for significant fines for non-compliance[181](index=181&type=chunk)[182](index=182&type=chunk)[183](index=183&type=chunk)[184](index=184&type=chunk)[216](index=216&type=chunk)[217](index=217&type=chunk)[219](index=219&type=chunk) - Healthcare reform laws (e.g., Health Care Reform Law, IRA) and government initiatives in the U.S. and abroad aim to control pharmaceutical pricing and reimbursement, potentially impacting product demand and revenue[223](index=223&type=chunk)[224](index=224&type=chunk)[229](index=229&type=chunk)[232](index=232&type=chunk)[234](index=234&type=chunk)[235](index=235&type=chunk)[239](index=239&type=chunk) [Human Capital](index=49&type=section&id=Human%20Capital) Solid Biosciences prioritizes attracting and retaining diverse talent, offering competitive compensation and fostering an inclusive environment, with **88 full-time employees** as of December 31, 2023 - Solid Biosciences prioritizes attracting, motivating, and retaining talented and diverse employees, offering competitive compensation and benefits, and fostering an equitable, inclusive, and empowering environment[241](index=241&type=chunk)[242](index=242&type=chunk) - As of December 31, 2023, the company employed **88 full-time employees**, with **65** in research and development and **23** in general and administrative positions; **19** employees hold Ph.D. or M.D. degrees[243](index=243&type=chunk) [Corporate Information](index=49&type=section&id=Corporate%20Information) Solid Biosciences Inc. maintains its principal executive offices in Charlestown, Massachusetts, and makes its SEC filings available free of charge on its website - The company's principal executive offices are located at 500 Rutherford Avenue, Third Floor, Charlestown, MA 02129, with telephone number (617) 337-4680[244](index=244&type=chunk) - Solid Biosciences makes its Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K, and amendments available free of charge on its website (www.solidbio.com) as soon as practicable after SEC filing[244](index=244&type=chunk)[245](index=245&type=chunk) [Item 1A. Risk Factors](index=50&type=section&id=Item%201A.%20Risk%20Factors) The company faces numerous risks, including acquisition integration challenges, substantial net losses, funding needs, development uncertainties for novel gene therapies, intense competition, and complex regulatory landscapes - The company's business is subject to risks including failure to realize anticipated benefits from the AavantiBio acquisition, significant net losses, and the need for additional funding[19](index=19&type=chunk)[247](index=247&type=chunk)[253](index=253&type=chunk)[256](index=256&type=chunk) - Key risks in product development include the novel technology of gene transfer candidates, potential undesirable side effects, clinical holds, difficulties in patient enrollment, and the uncertainty of regulatory approval[19](index=19&type=chunk)[273](index=273&type=chunk)[276](index=276&type=chunk)[282](index=282&type=chunk)[288](index=288&type=chunk)[295](index=295&type=chunk) - The company faces significant competition, manufacturing challenges, difficulties in establishing sales and marketing capabilities, and uncertainties regarding market acceptance and reimbursement for its products[20](index=20&type=chunk)[351](index=351&type=chunk)[365](index=365&type=chunk)[379](index=379&type=chunk)[384](index=384&type=chunk)[391](index=391&type=chunk) - Intellectual property risks include reliance on in-licensed patents, challenges to patent validity, and the difficulty of protecting trade secrets globally. Regulatory risks involve evolving gene therapy requirements, potential changes in orphan drug exclusivity, and the impact of healthcare reform on pricing[20](index=20&type=chunk)[317](index=317&type=chunk)[323](index=323&type=chunk)[454](index=454&type=chunk)[460](index=460&type=chunk)[494](index=494&type=chunk) [Risks related to the Acquisition](index=50&type=section&id=Risks%20related%20to%20the%20Acquisition) Risks from the AavantiBio acquisition include failure to realize anticipated benefits, integration difficulties, and potential stock price impact without commensurate stockholder benefits - The company may fail to realize anticipated benefits from the AavantiBio acquisition, or these benefits may take longer to achieve than expected, due to integration difficulties[247](index=247&type=chunk) - Potential integration difficulties include combining businesses, managing expanded operations, assimilating employees, maintaining morale, and attracting/retaining key personnel[247](index=247&type=chunk) - Stockholders may not realize benefits from the acquisition and related private placement commensurate with the ownership dilution experienced[250](index=250&type=chunk) [Risks related to our financial position and need for capital requirements](index=52&type=section&id=Risks%20related%20to%20our%20financial%20position%20and%20need%20for%20capital%20requirements) Solid Biosciences has incurred significant net losses and requires substantial additional funding beyond current capital, with future profitability being uncertain - The company has incurred significant net losses since inception, with **$96.0 million** in 2023, **$86.0 million** in 2022, and **$72.2 million** in 2021, and an accumulated deficit of **$658.8 million** as of December 31, 2023[253](index=253&type=chunk)[553](index=553&type=chunk)[554](index=554&type=chunk) - The company anticipates continued net losses and expects expenses to increase substantially due to advancing clinical trials (SGT-003), preclinical development, regulatory filings, manufacturing, and potential commercialization[253](index=253&type=chunk)[254](index=254&type=chunk)[554](index=554&type=chunk)[608](index=608&type=chunk)[610](index=610&type=chunk) - Additional funding will be required beyond current capital, which is estimated to last into **2026**. Failure to obtain necessary capital may force delays, limits, or termination of product development efforts[256](index=256&type=chunk)[555](index=555&type=chunk)[557](index=557&type=chunk)[608](index=608&type=chunk) - The company has never generated revenue from product sales and does not expect to for the foreseeable future, with profitability dependent on successful development and commercialization of product candidates[260](index=260&type=chunk)[559](index=559&type=chunk) [Risks related to the development of our product candidates](index=58&type=section&id=Risks%20related%20to%20the%20development%20of%20our%20product%20candidates) Developing novel gene therapies is high-risk, expensive, and time-consuming, with uncertainties in safety, efficacy, patient enrollment, and regulatory approval, potentially leading to delays or termination - Gene transfer candidates are based on novel technology, making development time and cost difficult to predict, with a high risk of failure and potential for unforeseen toxicity or lack of efficacy[273](index=273&type=chunk)[274](index=274&type=chunk)[275](index=275&type=chunk) - SGT-001 previously experienced a clinical hold due to a serious adverse event, and similar events could occur in future trials for SGT-003 or other candidates, leading to delays or termination[276](index=276&type=chunk)[282](index=282&type=chunk)[283](index=283&type=chunk) - The company has never completed a clinical trial and faces challenges in initiating and completing trials, including difficulties in patient enrollment, which could delay or prevent commercialization[284](index=284&type=chunk)[295](index=295&type=chunk)[296](index=296&type=chunk) - Preliminary or interim clinical data may change as more data becomes available, and success in early trials is not indicative of later-stage results, posing risks for regulatory approval[286](index=286&type=chunk)[287](index=287&type=chunk) - Regulatory approval is uncertain and may be for narrower indications or subject to significant limitations (e.g., REMS, post-marketing studies), impacting commercial value[298](index=298&type=chunk)[300](index=300&type=chunk) - The regulatory landscape for gene therapy is constantly evolving, with new guidelines and potential changes (e.g., FDORA, EU CTR) that could lengthen review processes and increase development costs[317](index=317&type=chunk)[318](index=318&type=chunk)[319](index=319&type=chunk)[321](index=321&type=chunk)[322](index=322&type=chunk) [Risks related to the manufacturing and commercialization of our product candidates](index=78&type=section&id=Risks%20related%20to%20the%20manufacturing%20and%20commercialization%20of%20our%20product%20candidates) Commercialization depends on successful collaborations, overcoming manufacturing challenges, establishing sales capabilities, and securing market acceptance and reimbursement, all subject to significant risks - Collaborations, including with Ultragenyx, may not be successful, as collaborators have discretion over resource allocation and may not perform as expected, potentially delaying or terminating development[360](index=360&type=chunk)[361](index=361&type=chunk)[362](index=362&type=chunk) - The company has limited gene therapy manufacturing experience and relies on third-party CMOs, facing risks of production problems, delays, and non-compliance with cGMP requirements[365](index=365&type=chunk)[367](index=367&type=chunk)[368](index=368&type=chunk)[375](index=375&type=chunk)[376](index=376&type=chunk) - Establishing internal sales, distribution, marketing, and medical affairs capabilities is expensive and time-consuming, and failure to do so or secure favorable third-party agreements could prevent product revenue generation[379](index=379&type=chunk)[380](index=380&type=chunk) - Commercial success depends on market acceptance by physicians, patients, and third-party payors, which is uncertain due to ethical, social, medical, and legal concerns about gene therapy, and potential unforeseen adverse events[384](index=384&type=chunk)[387](index=387&type=chunk)[388](index=388&type=chunk) - Insurance coverage and reimbursement for gene therapy products are uncertain and critical for patient access. Governments outside the U.S. often impose strict price controls, potentially limiting revenue[391](index=391&type=chunk)[392](index=392&type=chunk)[393](index=393&type=chunk)[394](index=394&type=chunk)[395](index=395&type=chunk) - International commercialization involves additional risks, including differing regulatory requirements, reduced intellectual property protection, economic instability, and compliance with foreign laws[396](index=396&type=chunk)[397](index=397&type=chunk)[398](index=398&type=chunk) [Risks related to our business operations](index=98&type=section&id=Risks%20related%20to%20our%20business%20operations) Business operations face risks from retaining key personnel, managing growth, employee misconduct, and cybersecurity breaches, all potentially leading to liabilities and reputational harm - The company's future success is highly dependent on retaining key executive team members, consultants, and advisors, and attracting qualified scientific and technical personnel in a competitive market[401](index=401&type=chunk)[402](index=402&type=chunk)[404](index=404&type=chunk) - Strategic plans involving workforce reductions (e.g., **35%** in April 2022, **18%** in December 2022) may not yield anticipated savings, could incur greater costs, and disrupt business operations and talent retention[405](index=405&type=chunk) - Employee misconduct, including non-compliance with regulatory standards (FDA, healthcare fraud laws) or improper use of clinical trial information, could lead to significant liability and reputational harm[407](index=407&type=chunk) - Internal computer systems and those of collaborators are vulnerable to security breaches and cyber-attacks, which could disrupt product development, lead to loss of proprietary information, and incur liability[452](index=452&type=chunk)[453](index=453&type=chunk) [Risks related to our intellectual property](index=100&type=section&id=Risks%20related%20to%20our%20intellectual%20property) Intellectual property risks include reliance on in-licensed patents, challenges to validity, expensive litigation, changes in patent law, and difficulties in protecting trade secrets globally - The company heavily relies on in-licensed patents and intellectual property, which may be subject to disagreements over contract interpretation, potentially narrowing rights or increasing financial obligations[454](index=454&type=chunk)[474](index=474&type=chunk)[475](index=475&type=chunk) - Obtaining and maintaining patent protection for candidates is uncertain; pending applications may not issue, and issued patents may be challenged, narrowed, or invalidated by competitors[460](index=460&type=chunk)[461](index=461&type=chunk)[463](index=463&type=chunk)[466](index=466&type=chunk)[468](index=468&type=chunk)[470](index=470&type=chunk) - Third parties may initiate legal proceedings alleging infringement, misappropriation, or violation of their intellectual property rights, leading to substantial liabilities, injunctions, or cessation of commercialization[482](index=482&type=chunk)[485](index=485&type=chunk)[487](index=487&type=chunk) - Changes in U.S. patent law (e.g., Leahy-Smith Act, Supreme Court decisions like Prometheus and Myriad) and evolving USPTO guidance could diminish patent value and impact the ability to protect product candidates[500](index=500&type=chunk)[501](index=501&type=chunk)[502](index=502&type=chunk)[503](index=503&type=chunk)[504](index=504&type=chunk) - Failure to protect trade secrets through confidentiality agreements or independent discovery by competitors could harm the business and competitive position[494](index=494&type=chunk)[495](index=495&type=chunk)[496](index=496&type=chunk) - Some in-licensed intellectual property from government-funded programs may be subject to 'march-in' rights, reporting requirements, and a preference for U.S. manufacturing, potentially limiting exclusive rights or ability to use non-U.S. manufacturers[491](index=491&type=chunk) [Risks related to ownership of our common stock](index=113&type=section&id=Risks%20related%20to%20ownership%20of%20our%20common%20stock) Common stock ownership concentration, potential future share sales, stock price volatility, and anti-takeover provisions pose risks to investors and corporate control - Executive officers, directors, and principal stockholders collectively own a significant percentage of common stock, enabling them to control or significantly influence all matters submitted for stockholder approval[512](index=512&type=chunk) - Sales of a substantial number of shares in the public market, including those from private placements and equity compensation plans, could cause the market price of common stock to drop significantly[513](index=513&type=chunk)[514](index=514&type=chunk)[515](index=515&type=chunk)[516](index=516&type=chunk) - The company's stock price has been and is likely to remain volatile, influenced by factors such as clinical trial results, competition, regulatory developments, and general market conditions[518](index=518&type=chunk)[519](index=519&type=chunk) - The company does not anticipate paying cash dividends in the foreseeable future, making capital appreciation the sole source of gain for investors[528](index=528&type=chunk) - Provisions in the company's certificate of incorporation, bylaws, and Delaware law (Section 203 DGCL) could discourage or prevent mergers, acquisitions, or changes in control, and limit stockholders' ability to replace management[526](index=526&type=chunk)[527](index=527&type=chunk) [Item 1B. Unresolved Staff Comments.](index=117&type=section&id=Item%201B.%20Unresolved%20Staff%20Comments.) The company has no unresolved staff comments from the SEC [Item 1C. Cybersecurity](index=117&type=section&id=Item%201C.%20Cybersecurity) Solid Biosciences manages cybersecurity risks through safeguards, training, and board oversight, with no known material risks to business strategy or financial condition - The company has processes for assessing, identifying, and managing cybersecurity risks, including physical, procedural, and technical safeguards, employee training, and incident simulations[531](index=531&type=chunk)[532](index=532&type=chunk) - The Audit Committee of the Board of Directors provides oversight of cybersecurity risk, receiving periodic updates from management[533](index=533&type=chunk) - The company does not believe there are currently any known cybersecurity risks reasonably likely to materially affect its business strategy, results of operations, or financial condition[533](index=533&type=chunk) [Item 2. Properties](index=118&type=section&id=Item%202.%20Properties) Solid Biosciences leases its corporate headquarters in Charlestown, Massachusetts, and additional lab/office spaces in North Carolina and Florida - The company leases its corporate headquarters in Charlestown, Massachusetts, consisting of approximately **49,869 square feet** of office, laboratory, R&D, and manufacturing space[534](index=534&type=chunk) - The headquarters lease has an initial term of approximately **ten years**, expiring in **2032**, with an option to extend for an additional **five years**[534](index=534&type=chunk) - Additional smaller laboratory and office spaces are leased in North Carolina and Florida[534](index=534&type=chunk) [Item 3. Legal Proceedings](index=118&type=section&id=Item%203.%20Legal%20Proceedings) As of December 31, 2023, Solid Biosciences is not aware of any material legal proceedings or claims - The company is not aware of any material legal proceedings or claims as of December 31, 2023[535](index=535&type=chunk)[794](index=794&type=chunk) [Item 4. Mine Safety Disclosures](index=118&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) Mine Safety Disclosures are not applicable to Solid Biosciences Inc [PART II](index=119&type=section&id=PART%20II) This part details the company's common stock market, financial condition, results of operations, market risk, and internal controls [Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=119&type=section&id=Item%205.%20Market%20for%20Registrant%27s%20Common%20Equity%2C%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) Solid Biosciences' common stock trades on Nasdaq under 'SLDB', with approximately **57 holders** of record as of February 13, 2024, and no equity security purchases or unregistered sales in 2023 - Solid Biosciences' common stock has traded on the Nasdaq Global Select Market under 'SLDB' since **January 26, 2018**[539](index=539&type=chunk) - As of **February 13, 2024**, the company had approximately **57 holders** of record for its common stock[543](index=543&type=chunk) - The company did not purchase any of its registered equity securities during the reporting period and had no unregistered securities sales in **2023** not previously disclosed[544](index=544&type=chunk)[545](index=545&type=chunk) [Market Information](index=119&type=section&id=Market%20Information) Solid Biosciences' common stock has been listed on the Nasdaq Global Select Market since **January 2018**, with performance compared against key indices - Common stock has been publicly traded on the Nasdaq Global Select Market under 'SLDB' since **January 26, 2018**[539](index=539&type=chunk) - A performance graph compares the company's common stock performance to The Nasdaq Composite Index and The Nasdaq Biotechnology Index from **December 31, 2018**, through **December 31, 2023**[540](index=540&type=chunk)[541](index=541&type=chunk) [Holders](index=119&type=section&id=Holders) As of **February 13, 2024**, Solid Biosciences had approximately **57 holders** of record for its common stock, with a higher actual number of beneficial owners - As of **February 13, 2024**, the company had approximately **57 holders** of record of its common stock[543](index=543&type=chunk) - The actual number of common stock holders is greater than the record holders, including beneficial owners whose shares are held in street name by brokers or other nominees[543](index=543&type=chunk) [Purchase of Equity Securities](index=119&type=section&id=Purchase%20of%20Equity%20Securities) Solid Biosciences did not purchase any of its registered equity securities during the period covered by this Annual Report on Form 10-K - The company did not purchase any of its registered equity securities during the period covered by this Annual Report on Form 10-K[544](index=544&type=chunk) [Recent Sales of Unregistered Securities](index=120&type=section&id=Recent%20Sales%20of%20Unregistered%20Securities) Solid Biosciences did not sell any unregistered securities during **2023** that had not been previously disclosed in SEC reports - The company did not sell any securities during the year ended **December 31, 2023**, that were not registered under the Securities Act or previously described in a Form 10-Q or Form 8-K[545](index=545&type=chunk) [Item 6. Reserved](index=120&type=section&id=Item%206.%20Reserved) This item is reserved and contains no information [Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=121&type=section&id=Item%207.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section analyzes Solid Biosciences' financial condition and results, highlighting substantial operating losses, funding needs, R&D expenses, and critical accounting policies - Solid Biosciences is a life sciences company focused on advancing gene therapy candidates for rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne and SGT-501 for CPVT[550](index=550&type=chunk) - The company has generated substantial operating losses since inception, with net losses of **$96.0 million** in 2023, **$86.0 million** in 2022, and **$72.2 million** in 2021, and an accumulated deficit of **$658.8 million** as of December 31, 2023[553](index=553&type=chunk)[554](index=554&type=chunk) - The company expects to incur significant expenses and operating losses for the foreseeable future, requiring substantial additional funding to support continuing operations beyond **2026**[555](index=555&type=chunk)[557](index=557&type=chunk)[608](index=608&type=chunk) [Overview](index=121&type=section&id=Overview) Solid Biosciences, a gene therapy company for rare neuromuscular and cardiac diseases, incurred significant losses but expects current capital to fund operations into **2026** - Solid Biosciences is a life sciences company focused on advancing gene therapy candidates for rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne and SGT-501 for CPVT[550](index=550&type=chunk) - The company completed the acquisition of AavantiBio, Inc. in **December 2022**, adding gene therapy programs for Friedreich's ataxia and BAG3-mediated dilated cardiomyopathy[551](index=551&type=chunk) Net Losses and Accumulated Deficit | Metric | 2023 (in millions) | 2022 (in millions) | 2021 (in millions) | | :----- | :----------------- | :----------------- | :----------------- | | Net Loss | $(96.0) | $(86.0) | $(72.2) | | Accumulated Deficit (as of Dec 31) | $(658.8) | N/A | N/A | - As of **December 31, 2023**, cash, cash equivalents, and available-for-sale securities totaled **$123.6 million**. With net proceeds of approximately **$104.0 million** from a **January 2024** private placement, the company expects to fund operations into **2026**[557](index=557&type=chunk) [Financial operations overview](index=123&type=section&id=Financial%20operations%20overview) The company's financial operations are characterized by a lack of product revenue and significant operating expenses, primarily in R&D and G&A, with personnel costs as a major component - The company has not generated any product revenue to date and does not expect to for the foreseeable future[559](index=559&type=chunk) - Operating expenses are classified into research and development, and general and administrative, with personnel costs (salaries, benefits, bonuses, equity-based compensation) being a significant component of both[560](index=560&type=chunk) [Research and development expenses](index=123&type=section&id=Research%20and%20development%20expenses) R&D expenses, central to Solid Biosciences, decreased slightly in **2023** to **$76.6 million** due to reduced SGT-001 costs, offset by increases for other programs - Research and development expenses primarily include costs for discovery efforts, and the development of SGT-003 and other candidates[561](index=561&type=chunk) - Key components of R&D expenses are third-party agreements (CROs, CMOs), personnel costs, laboratory supplies, regulatory approval costs, and intellectual property license expenses[561](index=561&type=chunk)[565](index=565&type=chunk) Research and Development Expenses by Program | Program | 2023 (in thousands) | 2022 (in thousands) | Change (2023 vs 2022) | | :-------- | :------------------ | :------------------ | :-------------------- | | SGT-001 | $3,432 | $24,844 | $(21,412) (↓86%) | | SGT-003 | $20,856 | $9,995 | $10,861 (↑109%) | | SGT-501 | $3,200 | $- | $3,200 (↑100%) | | Other development programs | $7,439 | $3,450 | $3,989 (↑116%) | | Unallocated R&D expenses | $41,636 | $40,131 | $1,505 (↑4%) | | **Total R&D expenses** | **$76,563** | **$78,420** | **$(1,857) (↓2%)** | [General and administrative expenses](index=127&type=section&id=General%20and%20administrative%20expenses) G&A expenses decreased to **$27.8 million** in **2023** due to lower legal fees and insurance, partially offset by increased personnel, IT, recruiting, and facilities costs - General and administrative expenses primarily consist of salaries, benefits, and equity-based compensation for executive, finance, business development, and administrative personnel[567](index=567&type=chunk) - Other G&A costs include legal fees, professional fees (accounting, auditing, tax, consulting), insurance, travel, acquisition costs, and facility-related expenses[567](index=567&type=chunk) - General and administrative expenses decreased by **$1.1 million** from **$28.9 million** in 2022 to **$27.8 million** in 2023, primarily due to lower legal fees and insurance premiums, partially offset by increases in personnel, IT, recruiting, and facilities costs[590](index=590&type=chunk) [Restructuring charges](index=127&type=section&id=Restructuring%20charges) Restructuring charges significantly decreased to **$(0.1) million** in **2023** from **$7.2 million** in **2022**, primarily due to severance from workforce reductions Restructuring Charges | Metric | 2023 (in millions) | 2022 (in millions) | | :----- | :----------------- | :----------------- | | Restructuring Charges | $(0.1) | $7.2 | - Restructuring charges in **2022** and **2023** were primarily due to severance and employee-related costs from workforce reductions (approximately **35%** in April 2022 and **18%** in December 2022)[569](index=569&type=chunk)[591](index=591&type=chunk) [Other income (expense), net](index=127&type=section&id=Other%20income%20%28expense%29%2C%20net) Total other income, net, decreased to **$8.2 million** in **2023** from **$20.5 million** in **2022**, mainly due to a non-recurring gain on acquisition in **2022** - Other income (expense), net, consists of interest income from cash, cash equivalents, and available-for-sale securities, and amortization/accretion of investment premiums/discounts[570](index=570&type=chunk) Other Income (Expense), Net | Metric | 2023 (in millions) | 2022 (in millions) | | :----- | :----------------- | :----------------- | | Total Other Income, Net | $8.2 | $20.5 | | Interest Income, Net | $7.1 | $2.6 | | Gain on Acquisition | $- | $18.2 | - The decrease in total other income in **2023** was primarily due to the absence of an **$18.2 million** gain on acquisition recorded in **2022**, partially offset by a **$4.5 million** increase in interest income[592](index=592&type=chunk) [Income taxes](index=127&type=section&id=Income%20taxes) The company uses an asset and liability approach for income taxes, recognizing deferred tax assets and liabilities with a valuation allowance for estimated realizable value - The company accounts for income taxes using an asset and liability approach, recognizing deferred tax assets and liabilities for future tax consequences[571](index=571&type=chunk) - A valuation allowance is established to reduce deferred tax assets to their estimated realizable value[571](index=571&type=chunk) - Uncertainty in income taxes is assessed using a two-step process: evaluating the likelihood of a tax position being sustained and determining the largest benefit with over **50%** likelihood of realization[572](index=572&type=chunk) [Critical accounting policies and use of estimates](index=127&type=section&id=Critical%20accounting%20policies%20and%20use%20of%20estimates) Financial statement preparation requires significant management estimates and assumptions, particularly for revenue recognition, R&D expenses, and equity-based compensation - Preparation of consolidated financial statements requires significant estimates and assumptions affecting reported asset/liability amounts and expense disclosures[573](index=573&type=chunk) - Critical accounting policies involve estimates related to revenue recognition, research and development expenses, and equity-based compensation[574](index=574&type=chunk) - Estimates are based on historical experience, known trends, and events, and are evaluated ongoingly, but actual results may differ[573](index=573&type=chunk) [Revenue recognition](index=128&type=section&id=Revenue%20recognition) Revenue is recognized when control of promised goods or services transfers to the customer, following a five-step model requiring significant judgment for performance obligations and variable consideration - Revenue is recognized when the customer obtains control of promised goods or services, in an amount reflecting expected consideration, following a five-step model under ASC 606[575](index=575&type=chunk)[576](index=576&type=chunk)[709](index=709&type=chunk) - Significant judgment is required to determine distinct performance obligations, measure transaction price (including variable consideration like milestones), and allocate price to obligations[577](index=577&type=chunk)[578](index=578&type=chunk)[710](index=710&type=chunk)[711](index=711&type=chunk) - For licenses, revenue is recognized when the license is transferred if distinct. For research and development services, revenue is generally recognized over time using a cost-to-cost input method[579](index=579&type=chunk)[581](index=581&type=chunk)[715](index=715&type=chunk)[716](index=716&type=chunk) [Accrued research and development expenses](index=129&type=section&id=Accrued%20research%20and%20development%20expenses) Accrued R&D expenses are estimated based on services received from third-party providers, with estimates subject to adjustment but historically not materially different from actual costs - Accrued research and development expenses are estimated by reviewing open contracts, communicating with personnel, and estimating service levels and costs for services not yet invoiced[582](index=582&type=chunk) - Estimates are based on services received and efforts expended under contracts with CROs and CMOs, considering the time period and level of effort for performance[582](index=582&type=chunk) - Adjustments are made if actual timing or effort varies from estimates, though historical accrual estimates have not been materially different from actual costs[582](index=582&type=chunk) [Equity-based compensation](index=129&type=section&id=Equity-based%20compensation) Equity-based compensation is measured at fair value on the grant date and expensed over the vesting period, totaling **$7.6 million** in **2023** - Equity-based compensation for employees, directors, and non-employees is measured at fair value on the grant date and expensed over the vesting period, generally using the straight-line method[583](index=583&type=chunk)[726](index=726&type=chunk) - Stock option fair values are estimated using the Black-Scholes option-pricing model, with expected volatility based on publicly traded peer companies and a 'simplified' method for expected term[585](index=585&type=chunk)[727](index=727&type=chunk) Equity-Based Compensation Expense | Expense Category | 2023 (in thousands) | 2022 (in thousands) | 2021 (in thousands) | | :--------------- | :------------------ | :------------------ | :------------------ | | Research and development | $3,094 | $2,756 | $6,289 | | General and administrative | $4,531 | $4,781 | $7,084 | | **Total** | **$7,625** | **$7,537** | **$13,373** | [Results of operations](index=130&type=section&id=Results%20of%20operations) Solid Biosciences reported a net loss of **$96.0 million** in **2023**, an increase from **$86.0 million** in **2022**, driven by reduced collaboration revenue and a non-recurring gain Consolidated Statements of Operations Summary | Metric | 2023 (in thousands) | 2022 (in thousands) | Change (2023 vs 2022) | | :-------------------------- | :------------------ | :------------------ | :-------------------- | | Collaboration revenue - related party | $0 | $8,094 | $(8,094) (↓100%) | | Total operating expenses | $104,252 | $114,546 | $(10,294) (↓9%) | | Loss from operations | $(104,252) | $(106,452) | $2,200 (↑2%) | | Total other income, net | $8,237 | $20,471 | $(12,234) (↓60%) | | **Net loss** | **$(96,015)** | **$(85,981)** | **$(10,034) (↓12%)** | - Collaboration revenue decreased by **$8.1 million (100%)** in 2023 due to the completion of research and development services under the Ultragenyx Collaboration Agreement in **Q2 2022**[587](index=587&type=chunk) - Other income, net, decreased by **$12.2 million (60%)** in 2023, primarily due to the absence of an **$18.2 million** gain on acquisition recorded in 2022, partially offset by a **$4.5 million** increase in interest income[592](index=592&type=chunk) [Liquidity and capital resources](index=132&type=section&id=Liquidity%20and%20capital%20resources) Solid Biosciences financed operations through equity sales, raising **$546.8 million** net proceeds through **2023**, with **$123.6 million** in cash and securities, supplemented by a **January 2024** private placement - The company has financed operations primarily through sales of redeemable preferred units, member units, common stock, and prefunded warrants, raising an aggregate of **$546.8 million** net proceeds through **December 31, 2023**[594](index=594&type=chunk) - As of **December 31, 2023**, cash, cash equivalents, and available-for-sale securities totaled **$123.6 million** (excluding **$1.8 million** restricted cash), with no outstanding debt[598](index=598&type=chunk) - In **January 2024**, the company completed a private placement, raising approximately **$104.0 million** in net proceeds from the sale of common stock and pre-funded warrants[599](index=599&type=chunk)[834](index=834&type=chunk) Cash Flow Summary | Activity | 2023 (in thousands) | 2022 (in thousands) | | :-------------------------- | :------------------ | :------------------ | | Net cash used in operating activities | $(94,180) | $(97,977) | | Net cash provided by investing activities | $9,689 | $59,157 | | Net cash provided by financing activities | $3,122 | $74,831 | [Funding requirements](index=134&type=section&id=Funding%20requirements) Solid Biosciences anticipates substantial expense increases for development and commercialization, requiring significant additional funds beyond **2026**, with no committed external sources - Expenses are expected to increase substantially due to advancing clinical development of SGT-003, initiating trials for other candidates, preclinical research, regulatory interactions, and establishing commercial infrastructure[608](index=608&type=chunk)[610](index=610&type=chunk) - Current cash, cash equivalents, and available-for-sale securities, combined with **January 2024** private placement proceeds, are projected to fund operating expenses and capital requirements into **2026**[608](index=608&type=chunk) - The company will need substantial additional funds beyond **2026**, with no committed external sources. Failure to raise capital could force delays, reductions, or termination of development programs or commercialization efforts[608](index=608&type=chunk)[612](index=612&type=chunk)[613](index=613&type=chunk)[614](index=614&type=chunk) [Contractual obligations and commitments](index=136&type=section&id=Contractual%20obligations%20and%20commitments) Solid Biosciences has lease obligations for office and lab space and potential future milestone payments and royalties under licensing agreements, totaling **$12.0 million** in potential milestones as of **December 31, 2023** - The company has lease obligations for office and lab space in Massachusetts, North Carolina, and Florida[615](index=615&type=chunk) - Under third-party licensing agreements, the company has agreed to make milestone payments and pay royalties based on specified milestones[616](index=616&type=chunk) - As of **December 31, 2023**, potential future milestone payments under these agreements totaled an aggregate of **$12.0 million**, none of which were assessed to be probable[819](index=819&type=chunk) [Recently issued accounting pronouncements](index=136&type=section&id=Recently%20issued%20accounting%20pronouncements) The company reviewed recently issued accounting standards and determined they will not materially impact its consolidated financial statements or operations [Item 7A. Quantitative and Qualitative Disclosures About Market Risk](index=136&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) Solid Biosciences is exposed to interest rate risk from short-term investments, but a **10%** change would not materially impact its financial position; inflation has not been material - The company is exposed to market risk from changes in interest rates, primarily affecting interest income from cash equivalents (money market accounts) and investments (treasury bills)[619](index=619&type=chunk) - Due to the short-term nature of its investment portfolio (maturities less than **one year**), an immediate **10%** change in market interest rates would not materially impact the fair value of the portfolio or financial results[619](index=619&type=chunk) - Inflation generally affects the company by increasing labor, research, manufacturing, and development costs. While not material in the last **three years**, future inflation could adversely affect operations[620](index=620&type=chunk) [Item 8. Financial Statements and Supplementary Data](index=137&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data) This item incorporates by reference the company's audited consolidated financial statements and supplementary data, included at the end of this Annual Report on Form 10-K - The required financial statements and supplementary data are included at the end of this Annual Report on Form 10-K, beginning on page F-1[622](index=622&type=chunk) [Item 9. Changes in and Disagreements With Accountants on Accounting and Financial Disclosure](index=137&type=section&id=Item%209.%20Changes%20in%20and%20Disagreements%20With%20Accountants%20on%20Accounting%20and%20Financial%20Disclosure) This item states that there have been no changes in or disagreements with accountants on accounting and financial disclosure matters - There are no changes in and disagreements with accountants on accounting and financial disclosure[623](index=623&type=chunk) [Item 9A. Controls and Procedures](index=137&type=section&id=Item%209A.%20Controls%20and%20Procedures) Management concluded that disclosure controls and internal control over financial reporting were effective as of **December 31, 2023**, with no independent auditor attestation required - As of **December 31, 2023**, management, with CEO and CFO participation, concluded that disclosure controls and procedures were effective at the reasonable assurance level[624](index=624&type=chunk)[626](index=626&type=chunk) - Management concluded that the company's internal control over financial reporting was effective as of **December 31, 2023**, based on the COSO framework[627](index=627&type=chunk)[628](index=628&type=chunk) - As a non-accelerated and smaller reporting company, an attestation report on internal control over financial reporting from the independent registered public accounting firm is not required[629](index=629&type=chunk) [Evaluation of Disclosure Controls and Procedures](index=137&type=section&id=Evaluation%20of%20Disclosure%20Controls%20and%20Procedures) Management assessed the effectiveness of disclosure controls and procedures as of **December 31, 2023**, concluding they were effective at a reasonable assurance level - Management, with CEO and CFO participation, evaluated the effectiveness of disclosure controls and procedures as of **December 31, 2023**[624](index=624&type=chunk) - Disclosure controls and procedures are designed to ensure timely recording, processing, summarizing, and reporting of information required by the Exchange Act[624](index=624&type=chunk) - Management concluded that disclosure controls and procedures were effective at the reasonable assurance level, recognizing inherent limitations of control systems[625](index=625&type=chunk)[626](index=626&type=chunk) [Management's Annual Report on Internal Control over Financial Reporting](index=137&type=section&id=Management%27s%20Annual%20Report%20on%20Internal%20Control%20over%20Financial%20Reporting) Management concluded that internal control over financial reporting was effective as of **December 31, 2023**, based on the COSO framework, with no independent auditor attestation required - Management is responsible for establishing and maintaining adequate internal control over financial reporting, designed to provide reasonable assurance regarding financial reporting reliability[627](index=627&type=chunk) - Management concluded that the company's internal control over financial reporting was effective as of **December 31, 2023**, based on the COSO framework[628](index=628&type=chunk) - As a non-accelerated filer and smaller reporting company, an attestation report from the independent registered public accounting firm on internal control over financial reporting is not required[629](index=629&type=chunk) [Changes in Internal Control over Financial Reporting](index=138&type=section&id=Changes%20in%20Internal%20Control%20over%20Financial%20Reporting) There have been no material changes in Solid Biosciences' internal control over financial reporting during the three months ended **December 31, 2023** - No changes in internal control over financial reporting occurred during the three months ended **December 31, 2023**, that materially affected or are reasonably likely to materially affect it[630](index=630&type=chunk) [Item 9B. Other Information](index=138&type=section&id=Item%209B.%20Other%20Information) No directors or officers adopted or terminated Rule 10b5-1 or non-Rule 10b5-1 trading arrangements during **Q4 2023** - None of the company's directors or officers adopted or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement during the fourth quarter of **2023**[631](index=631&type=chunk) [Item 9C. Disclosure Regarding Foreign Jurisdictions that Prevent Inspections](index=138&type=section&id=Item%209C.%20Disclosure%20Regarding%20Foreign%20Jurisdictions%20that%20Prevent%20Inspections) This item is not applicable to Solid Biosciences Inc [PART III](index=139&type=section&id=PART%20III) This part incorporates by reference information on directors, executive officers, compensation, security ownership, related transactions, and principal accountant fees [Item 10. Directors, Executive Officers and Corporate Governance](index=139&type=section&id=Item%2010.%20Directors%2C%20Executive%20Officers%20and%20Corporate%20Governance) Information on directors, executive officers, and corporate governance is incorporated by reference from the **2024** Annual Meeting of Stockholders proxy statement - Information on directors, executive officers, and corporate governance is incorporated by reference from the definitive proxy statement for the **2024** Annual Meeting of Stockholders[635](index=635&type=chunk) - The company has adopted a Code of Business Conduct and Ethics, available on its website, which applies to directors, executive officers, and employees[636](index=636&type=chunk) [Item 11. Executive Compensation](index=139&type=section&id=Item%2011.%20Executive%20Compensation) Information regarding executive compensation is incorporated by reference from the company's definitive proxy statement for its **2024** Annual Meeting of Stockholders - Information on executive compensation is incorporated by reference from the definitive proxy statement for the **2024** Annual Meeting of Stockholders[637](index=637&type=chunk) [Item 12. Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=139&type=section&id=Item%2012.%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) Information on security ownership of beneficial owners and management is incorporated by reference from the **2024** Annual Meeting of Stockholders proxy statement - Information on security ownership of certain beneficial owners and management, and related stockholder matters, is incorporated by reference from the definitive proxy statement for the **2024** Annual Meeting of Stockholders[638](index=638&type=chunk) [Item 13. Certain Relationships and Related Transactions, and Director Independence](index=139&type=section&id=Item%2013.%20Certain%20Relationships%20and%20Related%20Transactions%2C%20and%20Director%20Independence) Information on certain relationships, related transactions, and director independence is incorporated by reference from the **2024** Annual Meeting of Stockholders proxy statement - Information on certain relationships and related transactions, and director independence, is incorporated by reference from the definitive proxy statement for the **2024** Annual Meeting of Stockholders[639](index=639&type=chunk) [Item 14. Principal Accountant Fees and Services](index=139&type=section&id=Item%2014.%20Principal%20Accountant%20Fees%20and%20Services) Information regarding principal accountant fees and services is incorporated by reference from the company's definitive proxy statement for its **2024** Annual Meeting of Stockholders - Information on principal accountant fees and services is incorporated by reference from the definitive proxy statement for the **2024** Annual Meeting of Stockholders[640](index=640&type=chunk) [PART IV](index=140&type=section&id=PART%20IV) This part details the exhibits and financial statement schedules included in the Annual Report on Form 10-K [Item 15. Exhibits and Financial Statement Schedules](index=140&type=section&id=Item%2015.%20Exhibits%20and%20Financial%20Statement%20Schedules) This section lists the financial statements and supplementary data, along with a comprehensive list of exhibits filed as part of the Annual Report on Form 10-K - The item includes a list of financial statements (Consolidated Balance Sheets, Statements of Operations, Comprehensive Loss, Stockholders' Equity, Cash Flows, and Notes) and supplementary data[643](index=643&type=chunk) - All financial statement schedules have been omitted because they are not applicable, not required, or the information is shown in the financial statements or notes[643](index=643&type=chunk) - A comprehensive list of exhibits filed as part of this Annual Report on Form 10-K is provided, including agreements, certificates, and plans[644](index=644&type=chunk)[646](index=646&type=chunk)[647](index=647&type=chunk)[648](index=648&type=chunk) [Item 16. Form 10-K Summary](index=146&type=section&id=Item%2016.%20Form%2010-K%20Summary) This item states that a Form 10-K Summary is not applicable [SIGNATURES](index=147&type=section&id=SIGNATURES) This section contains the required signatures for the Annual Report on Form 10-K from key executives and directors, all dated **March 13, 2024** - The report is signed by the President, Chief Executive Officer and Director (Alexander Cumbo), Chief Financial Officer (Kevin Tan), Chairman of the Board (Ian F. Smith), and other Directors[656](index=656&type=chunk) - All signatures are dated **March 13, 2024**[656](index=656&type=chunk) [CONSOLIDATED FINANCIAL STATEMENTS](index=149&type=section&id=CONSOLIDATED%20FINANCIAL%20STATEMENTS) This section presents the company's audited consolidated financial statements, including the balance sheets, statements of operations, comprehensive loss, stockholders' equity, cash flows, and detailed notes [Report of Independent Registered Public Accounting Firm](index=149&type=section&id=Report%20of%20Independent%20Registered%20Public%20Accounting%20Firm) PricewaterhouseCoopers LLP issued an unqualified opinion on Solid Biosciences' consolidated financial statements, noting high auditor effort for external R&D costs - PricewaterhouseCoopers LLP issued an unqualified opinion on the consolidated financial statements for the period ended **December 31, 2023** and **2022**[660](index=660&type=chunk) - The audit was conducted in accordance with PCAOB standards, but an audit of internal control over financial reporting was not performed[661](index=661&type=chunk)[662](index=662&type=chunk) - A critical audit matter identified was the high degree of auditor effort in performing procedures related to the company's external research and development costs[664](index=664&type=chunk)[666](index=666&type=chunk) [Consolidated Balance Sheets](index=151&type=section&id=Consolidated%20Balance%20Sheets) Total assets decreased from **$260.3 million** in **2022** to **$164.9 million** in **2023**, primarily due to reductions in cash and available-for-sale securities Consolidated Balance Sheet Summary (in thousands) | Metric | December 31, 2023 | December 31, 2022 | | :-------------------------- | :------------------ | :------------------ | | **Assets:** | | | | Cash and cash equivalents | $74,015 | $155,384 | | Available-for-sale securities | $49,625 | $58,338 | | Total current assets | $129,734 | $219,638 | | Total assets | $164,939 | $260,252 | | **Liabilities:** | | | | Total current lia
Solid Biosciences(SLDB) - 2023 Q3 - Quarterly Report
2023-11-08 21:08
PART I. FINANCIAL INFORMATION [Financial Statements (Unaudited)](index=3&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents Solid Biosciences Inc.'s unaudited condensed consolidated financial statements, highlighting a decrease in cash, increased net loss, and significant cash usage for the periods ended September 30, 2023 - The company is a life sciences firm focused on gene therapies for neuromuscular and cardiac diseases, including **SGT-003** for **Duchenne muscular dystrophy**, with its pipeline expanded through the acquisition of **AavantiBio** in December 2022[22](index=22&type=chunk)[23](index=23&type=chunk) - The company believes its existing cash, cash equivalents, and available-for-sale securities of **$142.9 million** (as of Sept 30, 2023) are sufficient to fund operations **into 2025**, though it acknowledges the need for future financing[30](index=30&type=chunk)[89](index=89&type=chunk) Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | Sep 30, 2023 | Dec 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $49,037 | $155,384 | | Available-for-sale securities | $93,847 | $58,338 | | Total current assets | $148,346 | $219,638 | | Total assets | $184,728 | $260,252 | | **Liabilities & Equity** | | | | Total liabilities | $40,367 | $48,586 | | Total stockholders' equity | $144,361 | $211,666 | Condensed Consolidated Statements of Operations (in thousands, except per share data) | Metric | Q3 2023 | Q3 2022 | 9 Months 2023 | 9 Months 2022 | | :--- | :--- | :--- | :--- | :--- | | Collaboration revenue | $0 | $0 | $0 | $8,094 | | Research and development | $16,702 | $14,005 | $61,110 | $57,130 | | General and administrative | $6,412 | $7,127 | $20,940 | $21,330 | | Loss from operations | $(23,114) | $(21,132) | $(81,987) | $(71,886) | | Net loss | $(20,980) | $(20,410) | $(75,679) | $(70,830) | | Net loss per share | $(1.05) | $(2.71) | $(3.83) | $(9.42) | Condensed Consolidated Statements of Cash Flows (in thousands) | Activity | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | $(73,357) | $(63,497) | | Net cash used in investing activities | $(35,607) | $(6,943) | | Net cash provided by financing activities | $2,617 | $94 | | **Net decrease in cash** | **$(106,347)** | **$(70,346)** | [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=19&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's gene therapy pipeline, Q3 and YTD 2023 financial results, noting increased R&D expenses, decreased G&A, and liquidity sufficient into 2025 - The company is focused on advancing a diverse pipeline of gene therapy candidates for rare neuromuscular and cardiac diseases, including **SGT-003** for **Duchenne**, **SGT-501** for **CPVT**, **AVB-401** for **BAG3-mediated dilated cardiomyopathy**, and **AVB-202-TT** for **Friedreich's ataxia**[80](index=80&type=chunk) - As of September 30, 2023, the company had cash, cash equivalents, and available-for-sale securities of **$142.9 million**, which is expected to fund operating expenses and capital expenditure requirements **into 2025**[89](index=89&type=chunk)[121](index=121&type=chunk) Comparison of Operating Results (in thousands) | Metric | Q3 2023 | Q3 2022 | 9 Months 2023 | 9 Months 2022 | | :--- | :--- | :--- | :--- | :--- | | Collaboration Revenue | $0 | $0 | $0 | $8,094 | | R&D Expenses | $16,702 | $14,005 | $61,110 | $57,130 | | G&A Expenses | $6,412 | $7,127 | $20,940 | $21,330 | | Net Loss | $(20,980) | $(20,410) | $(75,679) | $(70,830) | - The increase in R&D expenses for Q3 2023 was primarily due to a **$1.5 million** increase in costs for SGT-003 and a **$0.8 million** increase for other development programs, offset by a **$1.3 million** decrease in costs for SGT-001 as development of SGT-003 was prioritized[107](index=107&type=chunk) - For the nine months ended September 30, 2023, R&D expenses increased by **$4.0 million**, driven by a **$13.0 million** increase in SGT-003 costs and a **$4.5 million** increase in other programs, offset by a **$16.5 million** decrease in SGT-001 costs[114](index=114&type=chunk) - Net cash used in operating activities increased to **$73.4 million** for the first nine months of 2023 from **$63.5 million** in the same period of 2022, primarily due to a higher net loss and changes in operating assets and liabilities[122](index=122&type=chunk)[123](index=123&type=chunk)[124](index=124&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=29&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's market risk primarily stems from interest rate changes affecting its short-term investment portfolio, with a 10% rate shift deemed immaterial - The company is exposed to market risk from changes in **interest rates**, primarily affecting interest income from its cash equivalents and available-for-sale securities (treasury bills and corporate bonds)[138](index=138&type=chunk) - Due to the short-term nature of its investment portfolio (contractual maturities of less than one year), the company states that an immediate **10%** change in market interest rates would not materially impact the fair market value of its portfolio or its financial results[138](index=138&type=chunk) [Controls and Procedures](index=29&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls and procedures were effective as of September 30, 2023, with no material changes to internal control over financial reporting during Q3 2023 - As of September 30, 2023, the company's President and Chief Executive Officer and Chief Financial Officer concluded that the disclosure controls and procedures were **effective** at the reasonable assurance level[139](index=139&type=chunk) - There were **no changes** in internal control over financial reporting during the three months ended September 30, 2023, that have materially affected, or are reasonably likely to materially affect, the company's internal control over financial reporting[140](index=140&type=chunk) PART II. OTHER INFORMATION [Legal Proceedings](index=30&type=section&id=Item%201.%20Legal%20Proceedings) The company is not aware of any material legal proceedings or claims as of September 30, 2023 - The company states there are **no material legal proceedings** as of the reporting date[142](index=142&type=chunk) [Risk Factors](index=30&type=section&id=Item%201A.%20Risk%20Factors) This section details risks including AavantiBio integration, significant net losses, future funding needs, gene therapy development challenges, and reliance on third-party manufacturing and IP - The company may fail to realize the anticipated benefits of its acquisition of **AavantiBio** and may encounter significant **integration difficulties**[145](index=145&type=chunk)[146](index=146&type=chunk) - The company has a history of **significant net losses** (**$75.7 million** for the nine months ended Sep 30, 2023) and expects to continue incurring losses, requiring **additional funding** which may not be available[145](index=145&type=chunk)[152](index=152&type=chunk) - The company's gene transfer candidates are based on **novel technology**, making it difficult to predict development time, cost, and regulatory approval; past clinical trials have been placed on hold by the **FDA**, and future trials may face similar issues or undesirable side effects[145](index=145&type=chunk)[174](index=174&type=chunk)[177](index=177&type=chunk) - The company has **limited gene therapy manufacturing experience** and relies on **third-party manufacturers**, which poses risks of production problems, delays, and unsatisfactory performance[146](index=146&type=chunk)[256](index=256&type=chunk)[265](index=265&type=chunk) - The business heavily relies on **in-licensed patents** and **intellectual property**; failure to maintain these licenses or protect its **IP** could allow competitors to develop similar products and adversely affect commercialization[146](index=146&type=chunk)[343](index=343&type=chunk)[349](index=349&type=chunk) - The company faces **significant competition** from larger, better-funded companies, including **Sarepta Therapeutics**, whose gene therapy **ELEVIDYS** received **FDA accelerated approval** in June 2023 for a similar patient population[242](index=242&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=82&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) No unregistered equity securities were sold during the three months ended September 30, 2023 - **No unregistered securities** were sold during the three months ended September 30, 2023[424](index=424&type=chunk) [Exhibits](index=83&type=section&id=Item%206.%20Exhibits) Exhibits include Gabriel Brooks' employment agreement and required CEO/CFO certifications under Sarbanes-Oxley Sections 302 and 906 - The exhibits filed with this report include an **employment agreement** for Gabriel Brooks and required **CEO/CFO certifications** under **Sarbanes-Oxley Sections 302 and 906**[426](index=426&type=chunk)
Solid Biosciences(SLDB) - 2023 Q2 - Quarterly Report
2023-08-14 20:03
PART I. FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=2&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents unaudited condensed consolidated financial statements, including balance sheets, operations, and cash flows, with detailed notes [Condensed Consolidated Balance Sheets](index=2&type=section&id=Condensed%20Consolidated%20Balance%20Sheets%20at%20June%2030,%202023%20and%20December%2031,%202022) Total assets and stockholders' equity decreased from December 2022 to June 2023, driven by reduced cash and increased accumulated deficit Condensed Consolidated Balance Sheets (in thousands) | Metric | June 30, 2023 (in thousands) | December 31, 2022 (in thousands) | Change (in thousands) | % Change | | :-------------------------------- | :----------------------------- | :------------------------------- | :-------------------- | :------- | | Cash and cash equivalents | $91,569 | $155,384 | $(63,815) | -41.07% | | Available-for-sale securities | $68,623 | $58,338 | $10,285 | 17.63% | | Total current assets | $167,475 | $219,638 | $(52,163) | -23.75% | | Total assets | $204,721 | $260,252 | $(55,531) | -21.34% | | Total current liabilities | $15,866 | $22,508 | $(6,642) | -29.51% | | Total liabilities | $40,993 | $48,586 | $(7,593) | -15.63% | | Total stockholders' equity | $163,728 | $211,666 | $(47,938) | -22.65% | | Accumulated deficit | $(617,437) | $(562,738) | $(54,699) | 9.72% | [Condensed Consolidated Statements of Operations](index=3&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20for%20the%20three%20and%20six%20months%20ended%20June%2030,%202023%20and%202022) No collaboration revenue for Q2 and H1 2023; net losses remained substantial, driven by reduced collaboration revenue and increased interest income Condensed Consolidated Statements of Operations (in thousands) | Metric (in thousands) | Three Months Ended June 30, 2023 | Three Months Ended June 30, 2022 | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :-------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Collaboration revenue | $0 | $6,169 | $0 | $8,094 | | R&D expenses | $19,777 | $23,180 | $44,408 | $43,125 | | G&A expenses | $7,129 | $6,851 | $14,528 | $14,203 | | Restructuring Charges | $(63) | $1,520 | $(63) | $1,520 | | Total operating expenses | $26,843 | $31,551 | $58,873 | $58,848 | | Loss from operations | $(26,843) | $(25,382) | $(58,873) | $(50,754) | | Interest income, net | $1,949 | $293 | $3,627 | $347 | | Net loss | $(24,629) | $(25,092) | $(54,699) | $(50,420) | | Net loss per share | $(1.25) | $(3.33) | $(2.79) | $(6.71) | - Collaboration revenue decreased to **$0** for both the three and six months ended June 30, 2023, from **$6,169 thousand** and **$8,094 thousand** respectively in the prior year, due to the completion of research and development services under the Ultragenyx Collaboration Agreement[10](index=10&type=chunk)[52](index=52&type=chunk) - Net loss per share significantly improved from **$(3.33)** to **$(1.25)** for the three months ended June 30, 2023, and from **$(6.71)** to **$(2.79)** for the six months ended June 30, 2023, despite increased net loss, primarily due to a substantial increase in weighted average shares outstanding[10](index=10&type=chunk) [Condensed Consolidated Statements of Comprehensive Loss](index=4&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss%20for%20the%20three%20and%20six%20months%20ended%20June%2030,%202023%20and%202022) Comprehensive loss for Q2 and H1 2023 was primarily driven by net loss, with minor impact from unrealized gains on securities Condensed Consolidated Statements of Comprehensive Loss (in thousands) | Metric (in thousands) | Three Months Ended June 30, 2023 | Three Months Ended June 30, 2022 | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :-------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Net loss | $(24,629) | $(25,092) | $(54,699) | $(50,420) | | Unrealized gain (loss) on available-for-sale securities | $9 | $(71) | $82 | $(77) | | Comprehensive loss | $(24,620) | $(25,163) | $(54,617) | $(50,497) | [Condensed Consolidated Statements of Changes in Stockholders' Equity](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Changes%20in%20Stockholders'%20Equity%20for%20the%20three%20and%20six%20months%20ended%20June%2030,%202023%20and%202022) Stockholders' equity decreased from December 2022 to June 2023, driven by net loss, partially offset by equity compensation and common stock sales Condensed Consolidated Statements of Changes in Stockholders' Equity | Metric | Balance at December 31, 2022 | Six Months Ended June 30, 2023 | Balance at June 30, 2023 | | :-------------------- | :--------------------------- | :----------------------------- | :----------------------- | | Common Stock (shares) | 19,556,732 | 487,657 | 20,044,389 | | Common Stock (amount in thousands) | $20 | $0 | $20 | | Additional paid-in capital (in thousands) | $774,452 | $6,679 | $781,131 | | Accumulated other comprehensive income (loss) (in thousands) | $(68) | $82 | $14 | | Accumulated deficit (in thousands) | $(562,738) | $(54,699) | $(617,437) | | Total stockholders' equity (in thousands) | $211,666 | $(47,938) | $163,728 | - During the six months ended June 30, 2023, the company issued **420,000 shares** of common stock through an 'at-the-market offering' sales agreement, generating net proceeds of **$2,539 thousand**[14](index=14&type=chunk)[73](index=73&type=chunk) - Equity-based compensation contributed **$4,062 thousand** to additional paid-in capital for the six months ended June 30, 2023[14](index=14&type=chunk)[78](index=78&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows%20for%20the%20six%20months%20ended%20June%2030,%202023%20and%202022) Operating cash usage increased, investing cash usage decreased, and financing activities provided more cash, mainly from common stock issuance Cash Flow Activity (in thousands) | Cash Flow Activity (in thousands) | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :-------------------------------- | :----------------------------- | :----------------------------- | | Net cash used in operating activities | $(55,460) | $(43,068) | | Net cash used in investing activities | $(10,972) | $(21,852) | | Net cash provided by financing activities | $2,617 | $95 | | Net decrease in cash, cash equivalents and restricted cash | $(63,815) | $(64,825)
Solid Biosciences (SLDB) Investor Presentation - Slideshow
2023-05-18 15:16
This presentation release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the company; the ability to successfully achieve and execute on the company's priorities and achieve key clinical milestones; the company's plans to present data from its Friedreich's ataxia program, next-generation Duchenne muscular dystrophy program, novel capsid program, and process developme ...
Solid Biosciences(SLDB) - 2023 Q1 - Quarterly Report
2023-05-11 11:41
UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _________ to _________ Commission File Number: 001-38360 Solid Biosciences Inc. (Exact Name of Registrant as Specified in its Charter) (State or other jurisdict ...
Solid Biosciences(SLDB) - 2022 Q4 - Annual Report
2023-03-23 11:30
Financial Performance - The company incurred a net loss of $86.0 million for the year ended December 31, 2022, with an accumulated deficit of $562.7 million as of the same date [237]. - The company expects to continue incurring significant operating losses for the foreseeable future as it focuses on research and development [237]. - The company has never generated revenue from product sales and does not expect to do so for the foreseeable future [244]. - The company will need to raise additional funds to continue operations beyond 2025, with no assurance that such funds will be available on acceptable terms [240]. Acquisition and Integration - The acquisition of AavantiBio was completed on December 2, 2022, and included pipeline programs for treating FA and BAG3 mediated dilated cardiomyopathy [230]. - There is a risk that the integration of AavantiBio may divert management's attention and disrupt ongoing business operations [231]. - The anticipated benefits of the acquisition may not be realized if integration challenges arise or if undisclosed liabilities surface [232]. - Future capital requirements will depend on the progress and costs associated with integrating AavantiBio and the results of clinical trials for SGT-003 and other candidates [241]. - The company may face increased litigation risks following the acquisition, which could adversely impact business operations [236]. Clinical Trials and Regulatory Challenges - The COVID-19 pandemic has caused disruptions in clinical trials, regulatory activities, and supply chains, impacting the ability to complete preclinical studies and clinical trials [247][248]. - Company has faced challenges in recruiting patients for clinical trials due to COVID-19, resulting in missed or postponed patient visits [249]. - Regulatory approvals may be delayed or impacted due to the redirection of resources by the FDA towards COVID-19 therapies, affecting the progress of the company's product candidates [250]. - The company has limited experience in regulatory submissions and has never completed a clinical trial, which poses risks for future product candidates [272]. - The company may face significant delays in clinical trials due to various factors, including patient recruitment and regulatory approvals [278]. - Regulatory approval for SGT-003, AVB-202-TT, and AVB-401 is uncertain, and even if trials are successful, approvals may be for narrower indications than sought [285]. Product Development and Safety Concerns - The regulatory approval process for gene transfer candidates is uncertain and can be more expensive and time-consuming compared to other products, with only a limited number of such products approved in the U.S. and EU [262]. - There is a risk of undesirable side effects from gene transfer candidates that could delay or prevent regulatory approval, impacting their commercial potential [263]. - Previous gene therapy treatments have reported significant adverse side effects, raising concerns about the safety and efficacy of the company's candidates [266]. - A serious adverse event related to SGT-001 occurred in a clinical trial, leading to a clinical hold by the FDA in November 2019, which was lifted in October 2020 [270]. - The anticipated dosing requirements for SGT-003 may increase the risk of adverse side effects, as seen in previous high-dose AAV vector administrations [268]. Manufacturing and Supply Chain Risks - The company currently relies on third-party manufacturers for SGT-003 and plans to do so for AVB-202-TT and AVB-401 programs, which exposes it to risks of delays and insufficient supply [351]. - The company does not have long-term supply arrangements for SGT-003, which may hinder its ability to scale production and meet future demand [353]. - There is a risk of significant disruption in supply due to factors such as equipment malfunctions, contamination, or public health issues like the COVID-19 pandemic [346]. - The company is at risk of contamination in its manufacturing processes, which could disrupt the supply of its product candidates and affect clinical development timelines [366]. Market and Competitive Landscape - The company faces significant competition from larger pharmaceutical and biotechnology firms, which may achieve regulatory approval before the company or develop more effective therapies [328]. - Competitors such as Pfizer and Sarepta Therapeutics are advancing in clinical trials for gene therapies, with Sarepta having submitted a BLA for its candidate with a PDUFA date of May 29, 2023 [329]. - The company's commercial opportunity may be diminished if competitors launch safer, more effective, or less expensive products first [330]. - The commercial success of gene therapy products may be limited by patients' immune responses, affecting the population amenable to treatment [360]. Regulatory Environment and Compliance - The FDA may require a Risk Evaluation and Mitigation Strategy (REMS) if SGT-003 or other candidates receive marketing approval, which could include a medication guide and communication plan [269]. - Compliance with ongoing regulatory requirements post-approval is necessary, and failure to do so could limit the ability to market future products [291]. - The company is subject to various stringent privacy laws and regulations, including HIPAA, which impose significant compliance costs and potential penalties for violations [405]. - The company faces risks related to anti-corruption laws, including the U.K. Bribery Act and the U.S. Foreign Corrupt Practices Act, which could lead to civil or criminal penalties if violated [416]. Financial and Operational Risks - The company faces potential pricing pressures from both federal and state healthcare reforms, which could limit revenue generation capabilities [396]. - The company has reduced its workforce by approximately 35% in April 2022 and 18% in December 2022 as part of a strategic plan to streamline operations, but may not realize the anticipated benefits from these reductions [381]. - The company is highly dependent on key employees, and the loss of these individuals could impede the achievement of its research and commercialization objectives [378]. - The company may engage in future acquisitions or strategic collaborations, which could increase capital requirements and introduce additional risks [376].
Solid Biosciences(SLDB) - 2022 Q3 - Quarterly Report
2022-11-10 12:32
UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _________ to _________ Commission File Number: 001-38360 Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Sec ...
Solid Biosciences (SLDB) Investor Presentation - Slideshow
2022-10-06 17:12
© 2022 Solid Biosciences 1 Strategic Update: Acquisition of AavantiBio and $75M PIPE September 30, 2022 Forward-Looking Statements and Industry and Market Data This presentation and various remarks we make during this presentation contain "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding: future expectations, plans and prospects for Solid Biosciences Inc. (the "Company" or "Solid"), AavantiBio, Inc. ("AavantiBio") and the ...
Solid Biosciences(SLDB) - 2022 Q2 - Quarterly Report
2022-08-11 11:30
UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _________ to _________ Commission File Number: 001-38360 Solid Biosciences Inc. (Exact Name of Registrant as Specified in its Charter) Delaware 90-0943402 (State ...