Solid Biosciences Announces Licensing Agreement with Armatus Bio for the Use of AAV-SLB101, a Proprietary, Muscle-Targeted Capsid, in the Development of an RNAi Therapy to treat FSHD

Rhea-AI Summary

Solid Biosciences (SLDB) collaborates with Armatus Bio for FSHD treatment using AAV-SLB101 capsid. The agreement includes upfront payment, milestone payments, and royalties. AAV-SLB101 shows enhanced biodistribution and muscle cell expression in preclinical studies. Armatus will develop and commercialize ARM-201, a potential FSHD therapeutic, utilizing AAV-SLB101.

Medical Research Analyst

The collaboration between Solid Biosciences and Armatus Bio represents a significant stride in the development of genetic therapies for muscular disorders. The licensing of AAV-SLB101 for the FSHD program could potentially accelerate the development timeline and improve the efficacy of treatment options. AAV-SLB101's enhanced biodistribution and muscle cell expression are critical advancements, as targeted delivery is a pivotal challenge in gene therapy. The preclinical data indicating reduced liver biodistribution is also noteworthy, as off-target effects are a common concern with AAV vectors and minimizing these is crucial for safety and efficacy.

Financially, the deal structure with upfront payments, milestone payments and tiered royalties creates a diversified revenue stream for Solid Biosciences. This is a strategic move to monetize their proprietary capsid library, which could lead to a more stable financial position and potentially fuel further R&D investments. The impact on the stock market could be positive, reflecting the potential future earnings and the validation of Solid's technology by a peer in the industry.

Biotech Financial Analyst

From a financial perspective, the licensing agreement between Solid Biosciences and Armatus Bio is a strategic move that could enhance investor confidence. The structure of the deal, with an upfront payment and additional milestone-based payments, provides immediate and future income for Solid Biosciences and mitigates some of the financial risks associated with the costly development process of gene therapies. The tiered royalty agreement further aligns Solid's long-term interests with the success of the FSHD treatment, potentially providing a sustained revenue source contingent on commercial success.

For investors, this deal signifies Solid Biosciences' ability to leverage its proprietary technology to generate revenue and establish collaborations within the biotech industry. The market's reaction to such news typically depends on the perceived value of the technology being licensed and the financial terms of the deal. If the terms are favorable and the technology has a high potential market, it could lead to an uptick in Solid Biosciences' stock price as the market anticipates future revenue streams.

Genetic Therapies Market Analyst

The licensing agreement is an indicator of the growing interest and potential in the genetic therapies market, particularly for neuromuscular disorders like FSHD. AAV-SLB101's improved targeting capabilities could be a game-changer for gene therapy efficacy and safety, which are key concerns in this market. By out-licensing their capsid technology, Solid Biosciences not only capitalizes on their R&D investments but also potentially expands the reach and impact of their technology across different applications and diseases.

Market dynamics for genetic therapies are influenced by technological advancements, regulatory approvals and the successful development of treatments. This agreement could signal to the market that Solid Biosciences' capsid technology is both credible and valuable, potentially leading to increased interest from other companies and institutions looking to improve the delivery of gene therapies. This could have a ripple effect on the broader market, spurring innovation and investment in similar technologies.

CHARLESTOWN, Mass., March 07, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced a non-exclusive worldwide license and collaboration agreement with Armatus Bio for the use of Solid’s proprietary capsid AAV-SLB101 for the development and commercialization of Armatus’ vectorized RNAi candidate to treat Facioscapulohumeral muscular dystrophy (FSHD). The AAV-SLB101 capsid has been shown in preclinical studies to have enhanced biodistribution and improved expression in muscle cells.

Under the terms of the agreement, Solid granted Armatus a non-exclusive worldwide license to utilize AAV-SLB101 for treatment of FSHD and will provide Armatus AAV-SLB101 plasmid material, preclinical data characterizing AAV-SLB101, and manufacturing and regulatory know-how to enable development. In return, Solid will receive an upfront payment, payments upon the achievement of certain development and sales milestones, and tiered royalties on net sales for any products incorporating AAV-SLB101. Armatus will be responsible for the development and commercialization of licensed products incorporating AAV-SLB101.

“Our transaction with Armatus Bio is the first of what we intend to be a broad out-licensing of our capsid library to both companies and academic institutions. We designed AAV-SLB101 for enhanced biodistribution to skeletal muscle, which is intended to help improve the efficacy and reduce the toxicity of AAV-delivered gene therapies for muscular disorders,” said Bo Cumbo, President and Chief Executive Officer at Solid Biosciences. “AAV-SLB101 is the capsid for our next-generation Duchenne candidate, SGT-003, and we are excited to expand AAV-SLB101’s use to FSHD through this agreement with Armatus Bio.”

Armatus is developing ARM-201, a potential best-in-class, single dose therapeutic used for the treatment of FSHD, a progressive genetic neuromuscular disorder caused by DUX4 overexpression. ARM-201’s proprietary miRNA payload seeks to reduce DUX4, and thus arrest muscle weakening and atrophy, prevent further degeneration, and ameliorate the amount of inflammation and oxidative stress caused by FSHD.

“The use of Solid’s unique AAV-SLB101 capsid will help enable optimal patient dosing for our FSHD program. The preclinical data presented to date for AAV-SLB101 demonstrate increased muscle transduction while simultaneously reducing biodistribution in the liver. These data, combined with the data we have generated for ARM-201 give us high confidence in the potential to reduce total AAV dose levels compared to first generation capsids,” commented Brian Price, Ph.D., Chief Technology Officer at Armatus Bio. “Further, we are encouraged by the strong safety profile demonstrated by AAV-SLB101, which has a cleared IND as the capsid in Solid’s SGT-003 construct for the treatment of DMD. With this license in place, we are eager to advance our optimized ARM-201 construct as the lead candidate toward clinical evaluation.”

About AAV-SLB101
AAV-SLB101 is a proprietary, rationally designed capsid developed for enhanced muscle tropism and reduced liver uptake. With a robust preclinical package in mice and nonhuman primates, AAV-SLB101 has demonstrated increased transduction speed, enhanced skeletal and cardiac muscle tropism, decreased liver biodistribution and improved efficiency when compared to first generation capsids. The incorporation of AAV-SLB101 into AAV delivered therapies has the potential to be a step forward in the treatment of neuromuscular and cardiac diseases. Solid Biosciences aims to license AAV-SLB101 broadly to both companies and academic institutions pursuing treatments for rare diseases.

About Solid Biosciences
Solid Biosciences is a life sciences company focused on advancing a portfolio of gene therapy candidates for neuromuscular and cardiac programs, 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, AVB-202-TT for the treatment of Friedreich’s ataxia, 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.

About Armatus Bio
Armatus Bio is a privately held late preclinical stage biotechnology innovator leveraging vectorized RNAi to address urgent unmet medical needs in genetically-driven neurological diseases. Based in Columbus, Ohio, the company is led by a seasoned team with expertise in drug development and delivery, and partnered with world renowned experts in vector biology, genomics, and neurology.

Solid Biosciences Forward-Looking Statements
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 ability to successfully achieve and execute on the company’s priorities and achieve key clinical milestones; the company’s SGT-003 program, including expectations for initiating dosing; potential milestone payments or royalty payments in connection with the collaboration; the potential benefits of the collaboration; the safety or potential efficacy of AAV-SLB101; Solid’s plans to broadly license AAV-SLB101 and its capsid library to both companies and academic institutions; 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 each party’s ability to perform its obligations under the collaboration; the ability to recognize the anticipated benefits of Solid’s acquisition of AavantiBio; the company’s ability to advance SGT-003, SGT-501, AVB-401, AVB-202-TT 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 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, AVB-202-TT 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 Contact:
Leah Monteiro
VP, Investor Relations and Communications
617-766-3430
lmonteiro@solidbio.com

What collaboration did Solid Biosciences (SLDB) announce?

Solid Biosciences announced a non-exclusive worldwide license and collaboration agreement with Armatus Bio for the use of Solid’s proprietary capsid AAV-SLB101 for the development and commercialization of Armatus’ vectorized RNAi candidate to treat Facioscapulohumeral muscular dystrophy (FSHD).

What benefits does AAV-SLB101 offer according to preclinical studies?

AAV-SLB101 has shown enhanced biodistribution and improved expression in muscle cells in preclinical studies.

What will Solid receive under the collaboration agreement?

Solid will receive an upfront payment, payments upon certain development and sales milestones achievement, and tiered royalties on net sales for any products incorporating AAV-SLB101.

What is ARM-201 developed by Armatus for?

ARM-201 is developed by Armatus as a potential best-in-class, single dose therapeutic for the treatment of Facioscapulohumeral muscular dystrophy (FSHD).

What is the purpose of AAV-SLB101 in Armatus' FSHD program?

AAV-SLB101 will help enable optimal patient dosing for Armatus' FSHD program by demonstrating increased muscle transduction while reducing biodistribution in the liver.