SGT-001

近日，Solid Biosciences发布了其新推的DMD基因治疗管线SGT-003的1/2期临床研究INSPIRE DUCHENN的早期数据。 研究中前三名患者的积极数据，使得Solid公司当日股价上涨31%。该公司同日还宣布完成了2亿美元的股票发行。 延伸阅读：明星管线失败，Solid再出发 SGT-003是Solid公司继曾经DMD明星管线SGT-001失败后推出的下一代DMD候选药物，其采用AAV-SLB101作为载体，递送包含R16和R17 nNOS 结合域的微抗肌萎缩蛋白基因。 Solid公司报告了SGT-0031/2期临床研究INSPIRE DUCHENN的早期数据，前3名参与者报告的中期90天活检数据显示：通过Western Blot测量，平均微肌萎缩蛋白表达为110%，且多个反映肌肉健康的生物标志物均有所改善。 此外，还观察到患者可能获得心脏益处的早期积极信号：在第180天，依据左心室射血分数测量，两名参与者的平均心脏功能较基线提高了8%；而在第90天，一名入组时血清心脏高敏肌钙蛋白I（hs-cTnI）水平升高的患者，其hs-cTnI水平降低了36%。 安全性方面，截至2025年2月11日，SGT-003在接受给药的6名参与者中耐受性良好，未观察到严重不良事件。 目前，该研究正在进行患者招募，第7名患者已经于2025年2月17日接受治疗。Solid公司预计到2025年第四季度，将为约20名参与者提供治疗，并计划于2025年中期与FDA会面，讨论SGT-003的潜在加速批准途径。 参考资料： 1.https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-reports-positive-initial-clinical-data-next · · · · · · · ·

– Patients dosed in Phase 1/2 INSPIRE DUCHENNE trial of SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne); Dosing was well tolerated in all patients with initial data expected Q4 2024 – – Solid plans to expand patient dosing with additional clinical trial sites in the U.S., Canada and Europe and plans accelerated production of multiple GMP batches of SGT-003 to support trial expansion – – Targeting the submission of 3-4 INDs by the end of 2026, strategically selecting cardiac and neuromuscular diseases with large patient populations and high unmet need – including SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT); CPVT IND submission expected in 1H 2025 – – Company ends Q2 2024 with approximately $190.3 million in cash, cash equivalents, and available-for-sale securities; Solid has anticipated cash runway into 2026 – CHARLESTOWN, Mass., Aug. 13, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the second quarter ended June 30, 2024, and provided a business update. Bo Cumbo, President and CEO of Solid Biosciences, commented: “In the second quarter of 2024, we commenced patient dosing in the Phase 1/2 INSPIRE DUCHENNE trial of SGT-003 – a key milestone for our lead gene therapy candidate – which has been well tolerated to date. Given these results and ongoing patient interest in new Duchenne therapies, we plan to expand the trial with additional sites and continuous patient dosing, while carefully managing patient screening to ensure consistency of both baseline criteria and future clinical data. We remain on track to share initial safety, expression, and (90-day) functional data at the end of this year.” Gabriel Brooks, M.D., Chief Medical Officer of Solid Biosciences, commented: “We are very encouraged that dosing has been well tolerated with no observed serious adverse events and that the safety results were consistent with the IND-enabling NHP toxicology study. To date, in this study of SGT-003, immunosuppression was achieved with steroids alone. SGT-003 leverages a rationally designed, novel capsid, AAV-SLB101, with the intent to target and more potently transduce muscle. We believe that the safety data seen to date are encouraging for not only SGT-003, but also our proprietary capsid, AAV-SLB101. We firmly believe Duchenne patients remain in need of better treatment options, and SGT-003 with its proprietary capsid, enhanced manufacturing process and differentiated transgene (uniquely containing the nNOS binding domain and flexible construct design), represents a potential next-generation therapy.” Mr. Cumbo continued, “As an organization, we are steadfast in our commitment to develop a truly transformative next-generation treatment option for patients in need around the world. To that end, I am pleased to share that we have made significant progress in clinical trial site initiations, with a total of six sites anticipated to be recruiting patients in Q4 2024 – including a site in Canada, now that Health Canada has authorized our Clinical Trial Application (CTA). With additional sites in Europe expected to be activated in 1H 2025, Solid is positioned to deliver on our commitment to patients globally. “Beyond Duchenne, we continue to make strides across our pipeline beginning with SGT-501, our lead cardiac gene therapy program to treat CPVT, with an IND submission expected in the first half of 2025. “We have also made progress in furthering the ongoing development of our earlier stage drug candidates. Based upon the preliminary human safety data from our Duchenne program, along with the preclinical expression and distribution data generated in multiple internal programs to date, we have transitioned our BAG3 and TNNT2 cardiac programs to our proprietary capsid, AAV-SLB101. This change will position us to synergistically leverage our clinical experience and manufacturing expertise with AAV-SLB101 across the majority of our programs going forward. “With $190.3M as of June 30, 2024, we remain well positioned to execute across our strategically built, robust pipeline of neuromuscular and cardiac gene therapy candidates. Based on our current programs, and our goal of having three-to-four INDs approved by the end of 2026, I believe Solid is poised to become an industry-leading precision genetic medicines company,” Mr. Cumbo concluded. Additional Company Highlights AAV-SLB101, Solid’s proprietary capsid used in SGT-003, has been well tolerated in initial human, NHP, and mouse studies. 10 different academic labs and one corporation have begun utilizing AAV-SLB101.On July 1, 2024, Solid was added to the broad-market Russell 3000® Index as part of the annual reconstitution. Russell indexes are widely used by investment managers and institutional investors for index funds and as investment strategy benchmarks, and Solid’s inclusion brings enhanced exposure and visibility within the financial community.Solid presented an oral presentation and six posters at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting, which took place May 7-11. The presentations highlighted Solid’s AAV manufacturing and purification improvements, vector biology updates, and a comprehensive non-clinical data overview of SGT-003. Second Quarter 2024 Financial Highlights Cash Position: Solid had approximately $190.3 million in cash, cash equivalents, and available-for-sale securities as of June 30, 2024, compared to approximately $123.6 million as of December 31, 2023. The Company expects that its cash, cash equivalents, and available-for-sale securities as of June 30, 2024, will enable it to fund key strategic priorities into 2026.Research and Development (R&D) Expenses: R&D expenses for the three months ended June 30, 2024, were $19.5 million, compared to $19.8 million for the three months ended June 30, 2023. The decrease of $0.3 million was due to a $3.3 million decrease in manufacturing and research costs for SGT-003, a $0.3 million decrease in costs for SGT-001 due to our decision to deprioritize development of SGT-001, and a $0.4 million decrease in external expenses, offset by a $1.7 million increase in costs for SGT-501 primarily related to manufacturing and research, a $1.7 million increase in other product candidate costs primarily related to BAG3, and a $0.2 million increase in personnel related costs.General and Administrative (G&A) Expenses: G&A expenses for the three months ended June 30, 2024, were $8.3 million, compared to $7.1 million for the three months ended June 30, 2023. The increase of $1.2 million was primarily related to a $0.9 million increase in legal fees and a $0.3 million increase in recruiting and license fees.Net Loss: Net loss for the three months ended June 30, 2024, was $25.1 million compared to a net loss of $24.6 million for the same period in 2023. Basic and diluted net loss per share was $0.61 and $1.25 for the three-month periods ended June 30, 2024, and June 30, 2023, respectively. About Solid BiosciencesSolid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit www.solidbio.com. Forward-Looking StatementsThis press 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 goals, priorities and achieve key clinical milestones; the company’s SGT-003 program, including expectations for additional CTA filings, site activations, expanded clinical development, accelerated production of multiple GMP batches of SGT-003, initiation and enrollment in clinical trials, dosing, availability of clinical trial data and potential accelerated approval; the company’s expectations for submission of an IND for SGT-501 and to submit additional INDs by the end of 2026; the cash runway of the company and the sufficiency of the Company’s cash, cash equivalents, and available-for-sale securities to fund its operations; and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “working” and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the company’s ability to advance SGT-003, SGT-501, AVB-401 and other preclinical programs and capsid libraries on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies and early-stage clinical trials of the company’s product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne and other neuromuscular and cardiac treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-003, SGT-501, AVB-401 and other candidates, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the company’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in the company’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company’s views as of the date hereof and should not be relied upon as representing the company’s views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so. Solid Biosciences Investor Contact:Nicole AndersonDirector, Investor Relations and Corporate CommunicationsSolid Biosciences Inc.investors@solidbio.com Media Contact:Glenn SilverFINN Partnersglenn.silver@finnpartners.com (tables follow) SELECTED FINANCIAL INFORMATION (UNAUDITED) CONDENSED CONSOLIDATED BALANCE SHEETS June 30, December 31, (in thousands, except share data) 2024 2023 Cash and cash equivalents $95,854 $74,015 Available-for-sale securities 94,412 49,625 Prepaid expenses and other current assets 7,344 6,094 Operating lease, right-of-use assets 25,508 26,539 Property and equipment, net 5,438 6,624 Other non-current assets 515 209 Restricted cash 1,910 1,833 Total Assets $230,981 $164,939 Accounts payable $2,521 $2,032 Accrued expenses and other current liabilities 9,918 10,161 Operating lease liabilities 1,724 1,855 Finance lease liabilities 525 469 Operating lease liabilities, excluding current portion 22,095 22,707 Finance lease liabilities, excluding current portion 953 1,234 Total stockholders’ equity 193,245 126,481 Total Liabilities and Stockholders’ Equity $230,981 $164,939 Common stock outstanding 38,551,059 20,387,606 CONDENSED CONSOLIDATED STATEMENT OF OPERATIONS Three Months EndedJune 30, Six Months EndedJune 30, (in thousands, except per share data) 2024 2023 2024 2023 Operating expenses: Research and development $19,461 $19,777 $38,334 $44,408 General and administrative 8,327 7,129 16,316 14,528 Restructuring charges — (63) — (63)Total operating expenses 27,788 26,843 54,650 58,873 Loss from operations (27,788) (26,843) (54,650) (58,873)Other income, net: Interest income 2,565 2,077 5,216 3,884 Interest expense (88) (111) (183) (233)Other income, net 239 248 242 523 Total other income, net 2,716 2,214 5,275 4,174 Net loss $(25,072) $(24,629) $(49,375) $(54,699)Net loss per share, basic and diluted $(0.61) $(1.25) $(1.25) $(2.79)Weighted average shares of common stock outstanding basic and diluted 40,934,361 19,663,672 39,544,867 19,618,517