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Sangamo Therapeutics and Astellas Announce Capsid License Agreement to Deliver Genomic Medicines for Neurological Diseases

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Sangamo Therapeutics (SGMO) and Astellas Pharma have entered into a license agreement for Sangamo's proprietary neurotropic AAV capsid, STAC-BBB, which has shown strong blood-brain barrier penetration capabilities in nonhuman primates. The agreement grants Astellas worldwide exclusive rights to use STAC-BBB for one target, with options for four additional neurological disease targets.

Sangamo will receive a $20 million upfront license fee and could earn up to $1.3 billion in additional target fees and milestone payments across all five potential targets, plus tiered mid-to-high single-digit royalties on potential net sales. Astellas will handle all research, development, manufacturing, and commercialization activities, while Sangamo will complete the technology transfer.

Sangamo Therapeutics (SGMO) e Astellas Pharma hanno stipulato un accordo di licenza per il capside neurotrofico proprietario di Sangamo, STAC-BBB, che ha dimostrato forti capacità di penetrazione della barriera emato-encefalica nei primati non umani. L'accordo concede ad Astellas diritti esclusivi a livello mondiale per utilizzare STAC-BBB per un bersaglio, con opzioni per quattro ulteriori bersagli di malattie neurologiche.

Sangamo riceverà una commissione di licenza di 20 milioni di dollari e potrebbe guadagnare fino a 1,3 miliardi di dollari in ulteriori commissioni sui bersagli e pagamenti per traguardi su tutti e cinque i bersagli potenziali, oltre a diritti royalty a scala variabile a livello medio-alto su potenziali vendite nette. Astellas gestirà tutte le attività di ricerca, sviluppo, produzione e commercializzazione, mentre Sangamo completerà il trasferimento tecnologico.

Sangamo Therapeutics (SGMO) y Astellas Pharma han firmado un acuerdo de licencia para el capsidio neurotrópico propietario de Sangamo, STAC-BBB, que ha demostrado fuertes capacidades de penetración de la barrera hematoencefálica en primates no humanos. El acuerdo otorga a Astellas derechos exclusivos a nivel mundial para usar STAC-BBB para un objetivo, con opciones para cuatro objetivos adicionales de enfermedades neurológicas.

Sangamo recibirá una tarifa de licencia por adelantado de 20 millones de dólares y podría ganar hasta 1.3 mil millones de dólares en tarifas adicionales por objetivos y pagos por hitos en los cinco objetivos potenciales, además de regalías escalonadas de dígitos simples en medio a alto sobre las ventas netas potenciales. Astellas se encargará de todas las actividades de investigación, desarrollo, fabricación y comercialización, mientras que Sangamo completará la transferencia tecnológica.

상가모 테라퓨틱스 (SGMO)아스텔라스 제약은 상가모의 독점 신경 트로픽 AAV 캡시드 STAC-BBB에 대한 라이센스 계약을 체결했습니다. 이 캡시드는 비인간 영장류에서 강력한 혈뇌 장벽 침투 능력을 보여주었습니다. 이번 계약은 아스텔라스에게 STAC-BBB를 하나의 표적에 대해 독점적으로 사용할 수 있는 전세계 권리를 부여하며, 추가로 네 가지 신경 질환 표적에 대한 옵션을 제공합니다.

상가모는 2천만 달러의 선불 라이센스 요금을 받을 것이며, 다섯 가지 잠재적 표적 전반에 걸쳐 추가 표적 요금 및 이정표 지불을 통해 최대 13억 달러를 벌 수 있습니다. 또한 잠재적 순매출에 대한 중간에서 높은 단일 자릿수 비율의 로열티를 받을 수 있습니다. 아스텔라스는 모든 연구, 개발, 제조 및 상업화 활동을 담당하며, 상가모는 기술 이전을 완료합니다.

Sangamo Therapeutics (SGMO) et Astellas Pharma ont signé un accord de licence pour le capsid neurotropique propriétaire de Sangamo, STAC-BBB, qui a montré de fortes capacités de pénétration de la barrière hémato-encéphalique chez les primates non humains. Cet accord accorde à Astellas des droits exclusifs mondiaux pour utiliser STAC-BBB pour une cible, avec des options pour quatre cibles supplémentaires de maladies neurologiques.

Sangamo recevra une redevance de licence de 20 millions de dollars et pourrait gagner jusqu'à 1,3 milliard de dollars en frais supplémentaires par cible et paiements d'étapes pour les cinq cibles potentielles, ainsi que des redevances à plusieurs niveaux à un chiffre unique de moyen à élevé sur les ventes nettes potentielles. Astellas s'occupera de toutes les activités de recherche, développement, fabrication et commercialisation, tandis que Sangamo achèvera le transfert de technologie.

Sangamo Therapeutics (SGMO) und Astellas Pharma haben einen Lizenzvertrag für den proprietären neurotropen AAV-Capsid STAC-BBB von Sangamo abgeschlossen, der starke Fähigkeiten zur Penetration der Blut-Hirn-Schranke bei nicht-menschlichen Primaten gezeigt hat. Der Vertrag gewährt Astellas weltweit exklusive Rechte zur Nutzung von STAC-BBB für ein Ziel, mit Optionen für vier zusätzliche neurologische Krankheitsziele.

Sangamo wird eine Lizenzgebühr von 20 Millionen US-Dollar erhalten und könnte bis zu 1,3 Milliarden US-Dollar an zusätzlichen Zielgebühren und Meilensteinzahlungen für alle fünf potenziellen Ziele verdienen, sowie gestaffelte Einheitenlizenzen im mittleren bis hohen einstelligen Bereich auf potenzielle Nettoumsätze. Astellas übernimmt alle Forschungs-, Entwicklungs-, Produktions- und Kommerzialisierungsaktivitäten, während Sangamo den Technologietransfer abschließen wird.

Positive
  • Secured $20 million upfront license fee from Astellas
  • Potential to earn up to $1.3 billion in additional fees and milestone payments
  • Will receive tiered mid-to-high single-digit royalties on potential net sales
  • No development or commercialization costs for Sangamo
Negative
  • Revenue generation dependent on Astellas' success in development and commercialization
  • Milestone payments are not guaranteed and subject to development achievements

Insights

The $20 million upfront payment and potential $1.3 billion in milestone payments represent a significant cash infusion for Sangamo, whose current market cap is around $486 million. The deal structure, with tiered mid-to-high single-digit royalties, follows industry standards for early-stage biotech licensing agreements. The immediate cash boost strengthens Sangamo's balance sheet and extends their operational runway.

The agreement's value proposition lies in the potential expansion to five targets, creating multiple shots on goal. Each additional target requires separate fees, providing recurring revenue opportunities. The deal's structure minimizes Sangamo's development risks while maintaining upside exposure through milestone payments and royalties. Astellas' commitment to handle development, manufacturing and commercialization significantly reduces Sangamo's capital requirements.

For context, neurological drug delivery technologies command premium valuations due to the $50+ billion market opportunity in CNS therapeutics. The STAC-BBB platform's demonstrated efficacy in non-human primates positions it as a valuable asset in this space.

The STAC-BBB capsid technology addresses one of gene therapy's most challenging obstacles - crossing the blood-brain barrier efficiently. Traditional AAV vectors struggle with CNS penetration, often requiring invasive direct administration. An effective IV-delivered solution could revolutionize neurological gene therapy by enabling broader distribution and simplified administration.

The non-human primate data showing potent BBB penetration and neuronal transduction is particularly noteworthy, as these results typically translate well to humans. The platform's potential applications extend beyond the initial targets, making it a versatile tool for treating various neurological conditions.

Astellas' involvement validates the technology's potential - they're a major player in gene therapy with established manufacturing capabilities and a strong CNS focus. Their expertise in development and commercialization significantly increases the likelihood of successful product launches.

- Agreement grants Astellas rights to employ Sangamo’s novel proprietary capsid, STAC-BBB, for up to five potential neurological disease targets

- Sangamo to receive a $20 million upfront license fee and is eligible to earn up to $1.3 billion in additional licensed target fees and milestone payments across all five potential disease targets, as well as tiered royalties on potential net sales

RICHMOND, Calif. & TOKYO--(BUSINESS WIRE)-- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company and Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, “Astellas”), today announced they have entered into a license agreement allowing Astellas to leverage Sangamo’s novel proprietary neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, which has demonstrated potent blood-brain barrier penetration and neuronal transduction in nonhuman primates. The agreement grants Astellas a worldwide exclusive license to utilize the STAC-BBB capsid for one target, with the right to add up to four additional targets after paying additional licensed target fees to deliver their intravenously administered genomic medicines to treat certain neurological diseases.

Sandy Macrae, Chief Executive Officer, Sangamo
“We strongly believe in the potential of STAC-BBB, our industry-leading, intravenously delivered AAV capsid, to overcome the challenges associated with delivering therapies to the central nervous system. This agreement underscores the continued industry interest in our STAC-BBB capsid and reinforces our ongoing commitment to partnering with collaborators who understand its unique potential. We are delighted to license STAC-BBB to Astellas to advance potential treatments for neurological diseases with significant unmet medical needs.”

Adam Pearson, Chief Strategy Officer, Astellas
“Delivering treatments to the brain and central nervous system remains a highly complex challenge in the field of gene therapy. We believe that technologies such as Sangamo’s STAC-BBB capsid could prove critical in helping us deliver effective transformational treatments to patients suffering from serious genetic neurological conditions. We continue to build a world-class gene therapy pipeline and end-to-end discovery, development, manufacturing, and commercial capabilities. This agreement is another example of our commitment to delivering meaningful therapies for patients with genetic diseases.”

Under the terms of the agreement, Sangamo is responsible for completing a technology transfer related to the STAC-BBB capsid. Astellas is responsible for all research, preclinical and clinical development, regulatory interactions, manufacturing, and global commercialization of the resulting gene therapy products. Sangamo will receive a $20 million upfront license fee from Astellas and is eligible to earn up to $1.3 billion in additional licensed target fees and milestone payments across the five potential neurology disease targets, as well as tiered mid-to-high single digit royalties on potential net sales of such products, subject to certain specified reductions.

About Sangamo Therapeutics

Sangamo Therapeutics is a genomic medicine company dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options. Sangamo believes that its zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders and that its capsid discovery platform can expand delivery beyond currently available intrathecal delivery capsids, including in the central nervous system. Sangamo’s pipeline also includes multiple partnered programs and programs with opportunities for partnership and investment. To learn more, visit www.sangamo.com and connect with us on LinkedIn and Twitter/X.

About Astellas

Astellas is a global life sciences company committed to turning innovative science into VALUE for patients. We provide transformative therapies in disease areas that include oncology, ophthalmology, urology, immunology and women's health. Through our research and development programs, we are pioneering new healthcare solutions for diseases with high unmet medical need. Learn more at www.astellas.com.

About Astellas Gene Therapies

Astellas Gene Therapies is an Astellas Center of Excellence developing genetic medicines with the potential to deliver transformative value for patients. Our gene therapy drug discovery engine is built around innovative science, a validated AAV platform, and industry leading internal manufacturing capability with a particular focus on rare diseases of the eye, CNS and neuromuscular system. Astellas Gene Therapies will also be advancing additional Astellas gene therapy programs toward clinical investigation. Astellas Gene Therapies is based in South San Francisco, with manufacturing and laboratory facilities in South San Francisco, Calif., Sanford, N.C. and Tsukuba, Japan.

Sangamo Forward Looking Statements

This press release contains forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, statements relating to the potential for Astellas to develop genomic medicines to treat neurodegenerative diseases by leveraging Sangamo’s capsid delivery capabilities, the potential for intravenously administered STAC-BBB to address challenges in delivering therapeutics to the central nervous system, the potential of Sangamo’s capsids to deliver therapies treating neurological diseases, the potential for Astellas to expand its license to include additional targets and to perform research, preclinical and clinical development, manufacturing and global commercialization of licensed gene therapy products for each licensed target, expectations concerning Sangamo’s receipt of an upfront license fee, the potential for Sangamo to receive additional licensed target fees, manufacturing, development and commercial milestone payments, and royalties, and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the research and development process, including the results of preclinical studies and clinical trials; the regulatory approval process for product candidates; the potential for technological developments that obviate technologies used by Sangamo and its partners; the potential for Astellas to breach or terminate its agreement with Sangamo; and the potential for Sangamo to fail to realize its expected benefits from the Astellas agreement; Sangamo’s inability to secure additional collaboration partners; and Sangamo’s need for substantial additional funding to operate as a going concern. There can be no assurance that Sangamo will earn any milestone or royalty payments under the Astellas agreement or obtain regulatory approvals for product candidates arising from this agreement. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's and Astellas’s operations and businesses. These risks and uncertainties are described more fully in Sangamo’s Securities and Exchange Commission, or SEC, filings and reports, including in its Annual Report on Form 10-K for the year ended December 31, 2023, as supplemented by its Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, each filed with the SEC, and future filings and reports that Sangamo makes from time to time with the SEC. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

Astellas Cautionary Notes

In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management’s current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas’ intellectual property rights by third parties. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice.

Sangamo Contact

Investor Relations & Media Inquiries

Louise Wilkie

ir@sangamo.com

media@sangamo.com

Astellas Contacts

Sara Zelkovic

R&D Communications

+1-347-226-1459

sara.zelkovic@astellas.com

Corporate Communications

+81-3-3244-3201

Source: Sangamo Therapeutics, Inc.

FAQ

What is the value of Sangamo's (SGMO) licensing deal with Astellas?

The deal includes a $20 million upfront payment and potential earnings of up to $1.3 billion in additional fees and milestone payments, plus tiered mid-to-high single-digit royalties on potential net sales.

How many neurological disease targets does the SGMO-Astellas agreement cover?

The agreement covers one initial target with the option to add up to four additional targets, for a total of five potential neurological disease targets.

What is STAC-BBB and why is it significant for SGMO?

STAC-BBB is Sangamo's proprietary neurotropic AAV capsid that has demonstrated potent blood-brain barrier penetration and neuronal transduction in nonhuman primates, making it valuable for delivering therapies to the central nervous system.

What are Sangamo's (SGMO) responsibilities in the Astellas partnership?

Sangamo is only responsible for completing the technology transfer related to the STAC-BBB capsid, while Astellas handles all research, development, manufacturing, and commercialization activities.

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