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|Item 2.02.|| |
Results of Operations and Financial Condition.
Spokespersons of Solid Biosciences Inc. (the “Company”) plan to present the information in the presentation attached hereto as Exhibit 99.1 (the “Presentation”) at various meetings beginning on January 9, 2023, including investor and analyst meetings in connection with the J.P. Morgan Healthcare Conference. A copy of the presentation is attached as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference.
Although the Company has not finalized its full financial results for the fourth quarter and fiscal year ended December 31, 2022, the Company disclosed in the Presentation that it expects to report cash and investments of approximately $214 million as of December 31, 2022.
The estimated cash and investments figure is preliminary and unaudited, represents management’s estimate as of the date of this report is subject to completion of the Company’s financial closing procedures for the fourth quarter and fiscal year ended December 31, 2022, and does not present all necessary information for a complete understanding of the Company’s financial condition as of December 31, 2022, or the Company’s results of operations for the year ended December 31, 2022. The actual financial results may differ materially from the preliminary estimated financial information.
The information provided under Item 2.02 of this Current Report on Form 8-K (including Exhibit 99.1) shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended (the “Securities Act”), or the Exchange Act, except as expressly set forth by specific reference in such a filing.
|Item 5.02.|| |
Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers; Compensatory Arrangements of Certain Officers.
On January 6, 2023, the Company’s Board of Directors (the “Board”) appointed Kevin Tan as the Company’s Chief Financial Officer, effective as of January 9, 2023 (the “Appointment Date”). In connection with his appointment, Mr. Tan will serve as the Company’s principal financial officer and principal accounting officer.
In connection with Mr. Tan’s appointment, Stephen DiPalma, the Company’s Interim Chief Financial Officer and principal financial officer and principal accounting officer, will cease to serve in such roles, effective as of the Appointment Date.
Mr. Tan, age 45, served as the Chief Financial Officer at Selecta Biosciences, Inc., a biopharmaceutical company (“Selecta”), from September 2021 to November 2022. Prior to joining Selecta, Mr. Tan served as Treasurer at Sarepta Therapeutics, Inc. (“Sarepta”), a biotechnology company, from July 2020 to September 2021. Prior to becoming Treasurer at Sarepta, he served as Assistant Treasurer from May 2018 to June 2020. Before joining Sarepta, Mr. Tan worked as a freelance consultant from February 2017 to April 2018, providing independent financial advice and advisory services to individuals and private companies. From June 2012 to November 2016, Mr. Tan served as Senior Portfolio Manager – Public Market Investments at CPP Investments (f/k/a the Canada Pension Plan Investment Board). He has also served in various positions at Macquarie Capital (USA) Inc., Arrowhawk Capital Partners LLC, and Lehman Brothers Inc. (subsequently acquired by Barclays Capital Inc.). Mr. Tan holds a Bachelor of Commerce degree from Queen’s University at Kingston, as well as a Master of Engineering degree from The Graduate School at Princeton University, and a Master of Business Administration degree from the University of Chicago Booth School of Business.
On January 9, 2023, Mr. Tan entered into an employment agreement with the Company (the “Employment Agreement”). Pursuant to the Employment Agreement, Mr. Tan will be entitled to receive an annual base salary of $425,000. His base salary will be reviewed by the Board from time to time and is subject to change in the discretion of the Board.
Under the Employment Agreement, Mr. Tan is also eligible to earn an annual performance bonus, with a target bonus amount equal to a specified percentage of his annual base salary, based upon the Board’s assessment of his performance and the Company’s attainment of targeted goals as set by the Board in its sole discretion. The bonus may be in the form of cash, equity award(s), or a combination of cash and equity. Mr. Tan will be eligible for an annual discretionary bonus of up to 40% of his base salary. Mr. Tan must be employed on the date that bonuses are paid in order to receive the bonus, provided that if he is terminated by the Company without cause (as “cause” is defined in the Employment Agreement) between January 1 following the performance year and the date of payment, he will be entitled to the same bonus that he would have received had he remained employed through the payment date.
Effective as of the Appointment Date and subject to Board approval, the Company will grant Mr. Tan a nonstatutory stock option (the “Option”) to purchase 90,000 shares of the Company’s common stock, at an exercise price per share equal to the closing price of the common stock on the Nasdaq Global Select Market on the Appointment Date, which will vest as to 25% of the shares underlying the Option on the first anniversary of the Appointment Date and, following that, as to an additional 1/48th of the total shares underlying the Option upon his completion of each additional month of service over the 36-month period measured from the first anniversary of the Appointment Date. Effective as of the Appointment Date and subject to Board approval, the Company will also grant Mr. Tan restricted stock units with respect to 45,000 shares of the Company’s common stock (the “RSU”), which will vest as to 25% of the shares underlying the RSU on each anniversary of the Appointment Date, subject to continued service. The Option and the RSU will be granted as an inducement material to Mr. Tan’s acceptance of employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).
The Employment Agreement and the employment of Mr. Tan may be terminated as follows: (1) upon his death or at the election of the Company due to his “disability” (as disability is defined in the Employment Agreement); (2) at the Company’s election, with or without “cause”; and (3) at his election, with or without “good reason” (as good reason is defined in the Employment Agreement).
In the event of the termination of Mr. Tan’s employment by the Company without cause, or by such executive for good reason, prior to or more than twelve months following a “change in control” (as change in control is defined in the Employment Agreement), the executive is entitled to receive his base salary that has accrued and to which he is entitled as of the termination date, to the extent consistent with Company policy, accrued but unused paid time off through and including the termination date, unreimbursed business expenses for which expenses the executive has timely submitted appropriate documentation, and other amounts or benefits to which the executive is entitled in accordance with the terms of the benefit plans then-sponsored by the Company (collectively, the “Accrued Obligations”). In addition, subject to the executive’s execution and nonrevocation of a release of claims in the Company’s favor, the executive is entitled to (1) continued payment of his base salary, in accordance with the Company’s regular payroll procedures, for a period of 12 months and (2) provided he is eligible for and timely elects to continue receiving group medical insurance under COBRA and the payments would not result in the violation of nondiscrimination requirements of applicable law, payment by the Company of the portion of health coverage premiums the Company pays for similarly-situated, active employees who receive the same type of coverage, for a period of up to 12 months following his date of termination.
In the event of the termination of Mr. Tan’s employment by the Company without cause, or by such executive for good reason within twelve months following a change in control, the executive is entitled to receive the Accrued Obligations. In addition, subject to the executive’s execution and nonrevocation of a release of claims in the Company’s favor, the executive is entitled to (1) continued payment of his base salary, in accordance with the Company’s regular payroll procedures, for a period of 12 months, (2) provided the executive is eligible for and timely elects to continue receiving group medical insurance under COBRA and the payments would not result in the violation of nondiscrimination requirements of applicable law, payment by the Company of the portion of health coverage premiums the Company pays for similarly-situated, active employees who receive the same type of coverage, for a period of up to 12 months following his date of termination, (3) a lump sum payment equal to 100% of the executive’s target bonus for the year in which his employment is terminated or, if higher, the executive’s target bonus immediately prior to the change in control and (4) full vesting acceleration of any then-unvested equity awards that vest based solely based on the passage of time held by the executive, such that any such equity awards held by the executive become fully exercisable or non-forfeitable as of the termination date.
If Mr. Tan’s employment is terminated for any other reason, including as a result of his death or disability, for cause, or voluntarily by him without good reason, the Company’s obligations under the employment agreement cease immediately, and the executive is only entitled to receive the Accrued Obligations.
In addition, in connection with his appointment, Mr. Tan will enter into the Company’s standard form of indemnification agreement with the Company, a copy of which was filed as Exhibit 10.16 to the Company’s Registration Statement on Form S-1 (File No. 333-222357) filed with the SEC on December 29, 2017. Pursuant to the terms of the indemnification agreement, the Company may be required, among other things, to indemnify Mr. Tan for certain expenses (including attorneys’ fees), judgments, fines and settlement amounts reasonably incurred by him in any action or proceeding arising out of his service as an officer of the Company.
The foregoing description of the Employment Agreement does not purport to be complete and is qualified in its entirety by reference to the complete text of such agreement, a copy of which will be included as an exhibit to the Company’s Annual Report on Form 10-K for the year ended December 31, 2022.
|Item 7.01.|| |
Regulation FD Disclosure.
The information contained above in Item 2.02 related to the Presentation is hereby incorporated by reference into this Item 7.01.
A copy of the Company’s press release announcing Mr. Tan’s appointment as Chief Financial Officer is attached as Exhibit 99.2 to this Current Report on Form 8-K and is incorporated herein by reference.
The information provided under Item 7.01 of this Current Report on Form 8-K (including Exhibits 99.1 and 99.2) shall not be deemed “filed” for purposes of Section 18 of the Exchange Act or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act or the Exchange Act, except as expressly set forth by specific reference in such a filing.
By providing the information in Item 7.01 of this Current Report on Form 8-K (including Exhibits 99.1 and 99.2), the Company is not making an admission as to the materiality of any information herein. The information contained in this Current Report on Form 8-K is intended to be considered in the context of more complete information included in the Company’s filings with the SEC and other public announcements that the Company has made and may make from time to time by press release or otherwise. The Company undertakes no duty or obligation to update or revise the information contained in this Current Report on Form 8-K, although it may do so from time to time as its management believes is appropriate. Any such updating may be made through the filing of other reports or documents with the SEC, through press releases or through other public disclosures.
|Item 8.01.|| |
On January 9, 2023, the Company announced that it anticipates submitting an investigational new drug application (“IND”) for SGT-003 in the second half of 2023 and, subject to IND clearance, initiating patient dosing in late-2023.
|Item 9.01.|| |
Financial Statements and Exhibits.
|99.1||Solid Biosciences Inc. Presentation January 2023|
|99.2||Press Release, dated January 9, 2023|
|104||Cover Page Interactive Data File (formatted as Inline XBRL)|
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
|SOLID BIOSCIENCES INC.|
|Date: January 9, 2023||By:|
Name: Alexander Cumbo
Title: Chief Executive Officer
Solid Biosciences 41st Annual J.P. Morgan Healthcare Conference JANUARY 12, 2023 Exhibit 99.1
Forward Looking Statement This presentation 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 anticipated milestones, business focus and pipeline of the company; the cash runway of the company and the sufficiency of the company’s cash and investments to fund its operations; the company’s SGT-003 program, including expectations for filing an IND and initiating dosing, AVB-202 program, including expectations for filing an IND, and AVB-401 program; the company’s plans to present data from IGNITE DMD; the implication of preclinical data; 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 ability to recognize the anticipated benefits of Solid’s acquisition of AavantiBio; the outcome of any legal proceedings that may be instituted against Solid or AavantiBio following the announcement of the acquisition and related transactions; the ability to obtain or maintain the listing of the common stock of the combined company on the Nasdaq Stock Market following the acquisition; the company’s ability to advance its SGT-003, AVB-202, AVB-401 and other programs on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; obtain and maintain the necessary approvals from investigational review boards at clinical trial sites and independent data safety monitoring board; replicate in clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; whether the methodologies, assumptions and applications the company utilizes to assess particular safety or efficacy parameters will yield meaningful statistical results; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; successfully transition, optimize and scale its manufacturing process; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne and Friedreich’s ataxia treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-003, AVB-202, AVB-401 and other product 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.
2023 Expected To Be a Year of Transformation and Meaningful Advancements for Solid PIPELINE Opportunity to become a leading precision genetic medicines company within neuromuscular and cardiac genetic medicine PROCESS Differentiated CMC expertise, building a robust, scalable manufacturing process utilizing transient transfection PEOPLE Led by experienced team with deep expertise in precision genetic medicine Strategic pipeline of programs continuing to evolve with anticipated key milestones in 2023-2024 Solid has the people, process and pipeline to be a leader in precision genetic medicines for rare neuromuscular and cardiac diseases.
2023: Solid Biosciences At a Glance Developing Genetic Medicines for Patients with Rare Neuromuscular and Cardiac Diseases Completed merger between AavantiBio and Solid Biosciences in December 2022 Headquartered in Charlestown, MA Combined pipeline, novel capsid development, and expertise from both companies to drive growth SGT-003 SGT-003 utilizes AAV-SLB101 capsid which has demonstrated superior tropism to AAV9 IND expected for DMD µDys SGT-003 program with novel capsid and transient transfection process Friedrich’s Ataxia and AVB-202 AVB-202 rescued cardiac function and extended survival in preclinical models Drug candidate selection, and transition from HSV to TT has begun, and initiation of IND-enabling studies for AVB-202 expected in 2023 BAG3 Dilated Cardiomyopathy Rh74 capsid and cardiac-specific promoter combination increased cardiac expression while reducing expression in the liver Additional studies to be conducted evaluating the potential of using SLB101 to develop a genetic medicine for BAG3-mediated DCM Cardiac and Skeletal Capsid Library Developing next-generation AAV capsid libraries with two strategies designed to enhance cardiac and skeletal muscle tropism AAV-SLB101 has demonstrated more than double the transduction and expression in skeletal and cardiac muscle and half reduction in expression in the liver compared with AAV9 Solid is Positioned for Success Additional programs to be announced in 2023 Approximately $214 million in cash and investments as of December 31, 2022 expected to enable Solid to advance key strategic priorities into 2025. *Year-end financial results pending full audit
Led By Experienced Team With Deep Expertise in Precision Genetic Medicine Bo Cumbo President and CEO Paul Herzich Chief Technology Officer Roxana Donisa Dreghici, M.D. Head of Clinical Development Ty Howton, J.D. Chief Administrative Officer Carl Morris, Ph.D. Chief Scientific Officer Neuromuscular Jenny Marlowe, Ph.D. Chief Scientific Officer Friedreich’s Ataxia & Cardiac Pipeline Jessie Hanrahan, Ph.D. Chief Regulatory Officer Kevin Tan Chief Financial Officer
Platform / Genetic Expertise Cardiac Rare Genetic Replacement Therapy (undisclosed #1) Cardiac Rare Genetic Replacement Therapy (undisclosed #2) Neuromuscular DMD uDys Replacement Therapy Anticipated 2H-2023 Neuromuscular Frataxin Replacement Therapy Cardiac BAG3 Replacement Therapy Cardiac Genetic DCM Partnered multiple modalities Vector core Animal models Natural history Expression cassette optimization Clinical & research partners Novel regulatory elements Novel capsids 2025+ 2023 Solid is Well-Positioned to Execute on Multiple Programs in the Coming Years Merger Solidifies Solid as a Gene Therapy Platform Technology Company
AAV CARDIAC capsids enhance select cardiac tissue tropism and reduce liver targeting Rational Design approach used to engineer capsid candidates with the goal of improving SKELETAL MUSCLE tropism AAV-SLB101 AAV9 Wild Type AAV-SLB101 AAV9 DMDmdx Human Duchenne Cell Microdystrophin Expression NovCap 1 NovCap 2 NovCap 3 NovCap 4 Next Generation Therapies Start With Delivery Through Innovative Capsids Utilization of human (primary cells) and primate ( Cyno macaques) as selection models Multiple libraries with unique barcodes (ability to test replicate libraries in tandem) Employs RNA for positive cardiac selection and DNA for negative liver selection Enhancement of RNA pool using Magnetic Instant Capture ( MagIC Beads) technology Library generated with two different muscle- and cardiac-specific promoters Relies on combination of rational design and directed evolution
Combined Company Strengthens Process/Analytical Development & CMC Regulatory Team Capabilities Robust network of CDMO partners along with our internal MS&T expertise and dedicated resources support advancement of Solid’s pre-clinical and early-stage pipeline programs Process optimization and cGMP manufacturing services for FA Preclinical and analytical materials for proof-of-concept studies and future pipeline products Manufacturing services and supply for Duchenne Fully Integrated and Operational R&D/ PD/AD and Vector Core capabilities PD and GMP manufacturing services for gene therapy pipeline Internal Capabilities
SGT-001-HSV SGT-003-TT Process change (HSV to TT) and AAV-SLB101 capsid combined to increase µDys by 2.3x vs SGT-001 HSV Product with desired quality attributes supported by analytical data with TT process Product demonstrated high levels of in vitro and in vivo transgene expression vs HSV material In vivo expression increased by 1.4-2.0x in multiple mdx studies 28-day in vivo mdx mouse study. Microdystrophin expression measured in the quadriceps muscle using Western Blot (WB). Mean data are shown +/- SD relative a Reference of SGT-001 produced by the HSV process. n=5 per group. Transition to Transient Transfection Manufacturing and Use of the AAV-SLB101 Capsid Yielded Additive Improvements in Expression 2.5 2.0 1.5 1.0 0.5 0.0 Microdystrophin Expression Fold vs. SGT-001-TT
Solid is Well-Positioned for Strategic Pipeline Growth Leveraging and Integrating Learnings Across Indications Pipeline Strategy Strategic Manufacturing Partnerships and Vector Core Indication Selection Criteria Network of KOL Partnerships Internal Development Capabilities
Indications With High Unmet Need and Significant Market Opportunities Notes: In 2020, Solid entered into a collaboration agreement with Ultragenyx for the development of UX810, a next generation Duchenne construct comprised of Solid’s proprietary nNOS microdystrophin and Ultragenyx’s HeLa PCL manufacturing platform for use with AAV8 and Clade E variants thereof. Solid has the option to co-fund collaboration programs in return for a profit share or increased royalty payments at proof-of-concept Program Indication Research / Discovery Preclinical IND submission (Anticipated) NEUROMUSCULAR SGT-003 (AAV-SLB101) Duchenne 2H 2023 AVB-202 (cardiac and neuromuscular manifestations) Friedreich’s Ataxia CARDIAC AVB-401 (Dilated Cardiomyopathy (DCM)) BAG3-Mediated DCM AVB-501 (Dilated Cardiomyopathy (DCM)) Undisclosed AVB-601 (Hypertrophic Cardiomyopathy) Undisclosed Diversified Pipeline with Multiple Programs at Different Stages
Duchenne Muscular Dystrophy and Next Generation SGT-003
Next Generation and Potential For Best-In-Class With SGT-003 Disease Overview Caused by mutations in the dystrophin gene, which leads to the absence of the dystrophin protein Due to progressive and irreversible muscle loss, patients typically lose the ability to walk by their early-teens and succumb to respiratory or heart failure in their 30’s Epidemiology Most common life-limiting genetic disorder diagnosed in childhood Estimated 5,000 to 15,000 cases in the U.S. 1:3500-5000 newborn males affected Diagnosed between three and five years of age due to pronounced muscle weakness Planned Approach Drive functional microdystrophin expression in patients’ muscles and improve the course of the disease Deliver best-in-class microdystrophin transgene containing the nNOS binding domain via a novel, muscle-tropic capsid Utilize a transient transfection manufacturing process Duchenne Represents A Large Global Market Opportunity With Significant Unmet Need
Next-Generation Therapies Utilize Optimized Transgene, Capsid and Manufacturing Process Capsid Manufacturing Process Transgene nNOS Binding Domain AAV-SLB101 AAV9 Wild Type AAV-SLB101 AAV9 DMDmdx nNOS Microdystrophin uniquely includes the nNOS binding domain, important for prevention of activity-induced ischemia and associated muscle injury Rational design approach used to engineer capsid candidates with the goal of improving skeletal muscle tropism Process change from HSV to TT-based manufacturing has yielded a greater than two-fold increase in microdystrophin expression in mice for SGT-003 (TT) compared with SGT-001 (HSV) Next-Generation Construct Has Shown Promising Results in Preclinical Testing SGT-001-HSV SGT-003-TT 2.5 2.0 1.5 1.0 0.5 0.0 Microdystrophin Expression Fold vs. SGT-001-TT
Observations: Dose Dependent Expression Expression Localized to Functional Membrane All tissues reached 100% expression at all doses by day 29 Quantitative MS analysis will be performed to evaluate full dynamic range of expression Expression Achieved Early (Day 4) and Optimized by Day 29 Quadriceps – Day 4 Heart – Day 4 Diaphragm – Day 4 Quadriceps – Day 29 Heart – Day 29 Diaphragm – Day 29 Vehicle 3.0E13 1.0E14 3.0E14 Vehicle 3.0E13 1.0E14 3.0E14 Vehicle 3.0E13 1.0E14 3.0E14 Vehicle 3.0E13 1.0E14 3.0E14 Vehicle 3.0E13 1.0E14 3.0E14 Vehicle 3.0E13 1.0E14 3.0E14
SGT-003 with SLB001 Capsid Demonstrated Superior Tropism to AAV9 Positive biodistribution and expression data has the potential to translate into better efficacy Quad Heart Liver 5 4 3 2 1 0 Fold Change µDys vg/ug DNA vs. AAV9 (1E14vg/kg, D29) Fold Change µDys Expression vs. AAV9 (1E14vg/kg) AAV9 AAV-SLB101 3 2 1 0 Quadriceps (D29) CK Level (U/L) mdx Vehicle mdx AAV9 4000 3000 2000 1000 0 Serum CK (D29) mdx AAV9-SLB101 WT Vehicle 1E14 vg/kg Robust μDys Expression in mdx Mouse Tissue Specific Biodistribution and Liver De-targeting in mdx Mouse Reduced CK levels in Vivo in mdx Mouse
Increased biodistribution to skeletal & cardiac muscle resulted in increased transgene expression at lower doses* Reduced biodistribution in liver suggests tissue de-targeting and improved safety profile* * Average fold differences calculated from the five skeletal muscle tissues sampled, three cardiac muscles sampled, and the single liver sample NHP IV Administration of SLB101 with Constitutive Promoter and Reporter Gene BIODISTRIBUTION NHP Data Utilizing AAV-SLB101 Showed Improved Biodistribution in Cardiac and Skeletal Muscle with Decreased Hepatic Transduction vs AAV9 Fold vs. AAV9 AAV9 AAV9-SLB101 4.0 3.0 2.0 1.0 0.0 Liver Fold vs. AAV9 AAV9 AAV9-SLB101 4.0 3.0 2.0 1.0 0.0 Cardiac Muscle Fold vs. AAV9 AAV9 AAV9-SLB101 4.0 3.0 2.0 1.0 0.0 Skeletal Muscle
Friedreich’s Ataxia and AVB-202
AVB-202’s dual route of administration is differentiated to treat the primary manifestations of morbidity and mortality Disease Overview Monogenic disease caused by loss of frataxin with both neurological and cardiac manifestations affecting muscle control and coordination with possible loss of vision and hearing, and slurred speech Cardiac complications are the primary cause of death Substantial unmet need with no disease-modifying standard of care for the broad population1 Epidemiology 1 in every 40,000 to 50,000 people2,3 Carrier rate between 1:60 and 1:100 Average age of diagnosis is in the early-teens which leads to many undiagnosed patients1 Planned Approach Aim to address neurological and cardiac manifestations via dual IV and IT routes of administration Drug candidate selection and transition manufacturing process to transient transfection 1. FARA. 2. Durr et al, 1996. 3. NORD Friedreich’s Ataxia Represents A Large Market Opportunity With Significant Unmet Need And No Approved Therapies
AVB-202 Rescued Cardiac Function and Extended Survival in Cardiac FA Mouse Survival Ejection Fraction SDH Activity Robust frataxin expression levels suggest efficacy may be achieved at low doses SDH activity via histochemical stain on heart tissue sections. The percentage of tissue with high stain is quantified by image analysis software All groups analyzed 15-16 wks after dosing except for early euthanasia groups (FA vehicle = 11 wks; FA 1.0E14 vg/kg= 9.5 wks)
NHP Study: Favorable Safety Profile and Utility of Dual Route of Administration Dose Group IV Dose (vg/kg) IT Dose (vg/brain wt) IV/IT Low 6.0E+12 6.00E+13 IV/IT Mid 1.5E+13 1.50E+14 IV/IT High 3.0E+13 3.00E+14 IV Mid 1.5E+13 - IV High 3.0E+13 - IT Mid - 1.50E+14 IT High - 3.00E+14 Using a dual route of administration allows for optimized expression at lower dose vs IT or IV alone FXN protein (ng/mg) Vehicle IV/IT Low 10000 1000 100 10 1 Cardiac FXN levels IV/IT Mid IV/IT High IV Mid IV High IT Mid IT High FXN protein (ng/mg) Vehicle IV/IT Low 250 200 150 100 50 0 DRG FXN levels IV/IT Mid IV/IT High IV Mid IV High IT Mid IT High
BAG3 Mediated Dilated Cardiomyopathy
Attractive Indication with Clear Mechanistic Rationale, High Unmet Need, and Significant Market Opportunity Key Disease Highlights The BAG3 gene codes for the BCL-2-associated athanogene 3 protein Sufficient levels of functional BAG3 are required for healthy cardiac function BAG3 mutations lead to reduced BAG3 levels and dilated cardiomyopathy (DCM) Postulated mechanism: Decreased BAG3 leads to heat shock protein dysfunction, and a build-up of dysfunctional proteins in the sarcomere, causing myofilament damage, poor contraction and heart failure Epidemiology ~29,000 active patients in the US1, 2, 3 Most common presentation is dyspnea but can range from dyspnea to sudden death Activities of daily life are severely impacted Eventually heart failure sets in and death ensues Once patients are symptomatic, mortality is approximately 25% at one year and approximately 50% at five years5 No approved therapies address the underlying cause of disease. Symptomatic treatment is standard of care4 Planned Approach AAVrh74-delivered optimized BAG3 transgene with cardiac-specific promoter for safe and specific expression Additional studies to evaluate the potential of using AAV-SLB101 to develop a genetic medicine for BAG3-mediated DCM Optimized transient transfection manufacturing process 1. Dominguez et al, 2018. 2. Virani et al, 2021. 3. Clearview Analysis. 4. Shaw et al, 2018. 5. Bozkurt et al, 2007 Dilated Cardiomyopathy (BAG3) Is The First Program From Our Cardiac Pipeline
Data Illustrate Superior Cardiac Biodistribution and Transgene Expression Compared to AAV9 rh74+cardiac specific promotor showed better BD in the heart over AAV9 with a constitutive promotor. rh74-cardiac specific promotor combination showed increased cardiac expression, and decreased liver expression. BAG3 DCM Reducing liver expression while optimizing cardiac expression allows for a more targeted, lower dose AAV therapeutic. Cardiac Biodistribution Data support Solid’s targeted approach to genetic cardiomyopathies: BAG3 hBAG3 Expression
Driving the Future
Complete SGT-003 GLP tox for next-generation Duchenne therapy 1H 2023 Initiation of Patient Dosing for SGT-003 Late-2023 ` Drug candidate selection and initiation of IND-enabling studies for AVB-202 Report functional data from IGNITE DMD Early-2023 IND Submission for SGT-003 2H 2023 Approximately $214 million* in cash and investments as of December 31, 2022 expected to enable Solid to advance key strategic priorities into 2025 Continue to diversify pipeline through BD transactions *Year-end financial results pending full audit 2023 Anticipated Milestones
Thank You 41st Annual J.P. Morgan Healthcare Conference JANUARY 12, 2023
Solid Biosciences Appoints Kevin Tan, CFA, as Chief Financial Officer
CHARLESTOWN, MA January 9, 2023 Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing genetic medicines for neuromuscular and cardiac diseases, today announced the appointment of Kevin Tan, CFA, a seasoned industry professional, as Chief Financial Officer.
We are pleased to welcome Kevin at this important juncture in the companys evolution, said Bo Cumbo, President and Chief Executive Officer of Solid Biosciences. Having previously worked with Kevin, I am confident his leadership, execution focus, and financial acumen will support the companys advancement towards key business objectives. Kevin has a deep understanding of capital markets and the biopharmaceutical industry, which will be instrumental as we advance our pipeline. Kevins prior experience in gene therapy will be of significant value as he works alongside myself and our Chief Administrative Officer, Ty Howton, to establish partnerships with the potential to accelerate our current product candidates and expand our next generation of genetic therapies for neuromuscular and cardiac disease.
I am excited to join Bo, Ty and the rest of the experienced and dedicated management team at Solid Biosciences, said Mr. Tan. The companys balance sheet positions us well to execute on value driving inflection points in the coming years. With a growing pipeline and significant expertise in developing and manufacturing novel genetic medicines, Solid has substantial long-term potential. I look forward to helping the company realize the full value of its platform to bring meaningful treatments to patients in need.
Mr. Tan brings more than 20 years of financial experience to Solid, most recently as CFO at Selecta Biosciences. In this role, Mr. Tan was responsible for all financial functions, business development activities and investor relations. While there, he worked closely with the leadership team to determine and execute the overall corporate strategy. Prior to joining Selecta, Mr. Tan served as Treasurer at Sarepta Therapeutics where he was responsible for the liquidity and capital management of the company. At Sarepta, he led numerous financings across equity and debt markets and was a key member of the Business Development team. Prior to his time at Sarepta, Mr. Tan had an extensive career in financial services, most recently as Senior Portfolio Manager at CPP Investments where he managed billions in capital across the public markets.
Mr. Tan holds a Bachelor of Commerce from Queens University at Kingston, a Master of Engineering in Operations Research & Financial Engineering from Princeton University and a Masters of Business Administration from The University of Chicago Booth School of Business. Mr. Tan is a Chartered Financial Analyst.
About Solid Biosciences
Solid Biosciences is a life science company focused on advancing a portfolio of neuromuscular and cardiac programs, including SGT-003, a differentiated gene transfer candidate, for the treatment of Duchenne, AVB-202, a gene transfer candidate for the treatment of Friedreichs Ataxia, AVB-401 for BAG3 mediated dilated cardiomyopathy, and additional assets for the treatment of undisclosed cardiac diseases. Solid aims to be the center of excellence, bringing together those with expertise in science, technology, disease management and care. Patient-focused and founded by those directly impacted by Duchenne, Solids mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit www.solidbio.com.
This 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 anticipated milestones, business focus and pipeline of the company; the balance sheet and cash runway of the company; 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 managements 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 ability to recognize the anticipated benefits of Solids acquisition of AavantiBio; the outcome of any legal proceedings that may be instituted against Solid or AavantiBio following the announcement of the acquisition and related transactions; the ability to obtain or maintain the listing of the common stock of the combined company on the Nasdaq Stock Market following the acquisition; the companys ability to advance its SGT-003, AVB-202, AVB-401 and other programs on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; obtain and maintain the necessary approvals from investigational review boards at clinical trial sites and independent data safety monitoring board; replicate in clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; whether the methodologies, assumptions and applications the company utilizes to assess particular safety or efficacy parameters will yield meaningful statistical results; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; successfully transition, optimize and scale its manufacturing process; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne and Friedreichs ataxia treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-003, AVB-202, AVB-401 and other product 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 companys 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 companys most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the companys views as of the date hereof and should not be relied upon as representing the companys views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the companys 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.