insitro Receives $25 Million in Milestone Payments from Bristol Myers Squibb for the Achievement of Discovery Milestones and the Selection of First Novel Genetic Target for ALS
insitro has received $25 million from Bristol Myers Squibb (BMY) for achieving discovery milestones and selecting the first novel target for amyotrophic lateral sclerosis (ALS) treatment. The milestone stems from their 2020 collaboration agreement. Using their proprietary machine learning platform, insitro developed over 200 engineered and patient ALS cell lines, along with high-content imaging and pooled optical screening technology to identify disease mechanisms and genetic modifiers. The platform successfully identified several novel gene targets that showed promise in reversing ALS-related deficits. Under the agreement terms, insitro received a $50 million upfront payment and could earn up to $2 billion in additional milestones plus royalties.
insitro ha ricevuto 25 milioni di dollari da Bristol Myers Squibb (BMY) per aver raggiunto importanti traguardi nella scoperta e per aver selezionato il primo nuovo bersaglio per il trattamento della sclerosi laterale amiotrofica (SLA). Questo traguardo deriva dall'accordo di collaborazione del 2020. Utilizzando la loro piattaforma proprietaria di machine learning, insitro ha sviluppato oltre 200 linee cellulari ingegnerizzate e derivate da pazienti con SLA, insieme a tecnologia di imaging ad alta definizione e screening ottico combinato per identificare i meccanismi della malattia e i modificatori genetici. La piattaforma ha identificato con successo diversi nuovi bersagli genici che hanno mostrato potenzialità nel ripristino dei deficit correlati alla SLA. Secondo i termini dell'accordo, insitro ha ricevuto un pagamento iniziale di 50 milioni di dollari e potrebbe guadagnare fino a 2 miliardi di dollari in ulteriori traguardi più royalty.
insitro ha recibido 25 millones de dólares de Bristol Myers Squibb (BMY) por alcanzar hitos en el descubrimiento y seleccionar el primer objetivo novedoso para el tratamiento de esclerosis lateral amiotrófica (ELA). Este hito proviene de su acuerdo de colaboración de 2020. Utilizando su plataforma de aprendizaje automático, insitro desarrolló más de 200 líneas celulares de ELA, tanto ingenierizadas como de pacientes, junto con tecnología de imagen de alto contenido y de detección óptica en grupo para identificar los mecanismos de la enfermedad y modificadores genéticos. La plataforma identificó con éxito varios nuevos objetivos génicos que mostraron potencial para revertir los déficits relacionados con la ELA. Según los términos del acuerdo, insitro recibió un pago inicial de 50 millones de dólares y podría ganar hasta 2 mil millones de dólares en hitos adicionales más regalías.
insitro는 Bristol Myers Squibb (BMY)로부터 근위축성 측삭 경화증 (ALS) 치료를 위한 첫 번째 새로운 타겟을 선정하고 발견 이정표를 달성한 대가로 2,500만 달러를 받았습니다. 이 이정표는 2020년 협력 계약에서 비롯된 것입니다. insitro는 고유의 기계 학습 플랫폼을 사용하여 ALS 환자와 관련한 200개 이상의 공학적 세포주를 개발하였고, 질병 메커니즘과 유전적 변수를 식별하기 위해 고해상도 이미징 및 집합 광학 스크리닝 기술을 함께 사용했습니다. 이 플랫폼은 ALS 관련 결손을 되돌리는 데 유망한 여러 새로운 유전자 표적을 성공적으로 식별했습니다. 계약 조건에 따라 insitro는 5천만 달러의 선불금을 받았으며, 추가 이정표 및 로열티에서 최대 20억 달러를 벌 수 있습니다.
insitro a reçu 25 millions de dollars de la part de Bristol Myers Squibb (BMY) pour avoir atteint des étapes de découverte et sélectionné la première cible novatrice pour le traitement de la sclérose latérale amyotrophique (SLA). Ce jalon découle de leur accord de collaboration de 2020. En utilisant leur plateforme de machine learning, insitro a développé plus de 200 lignées cellulaires ADA modifiées et patientes, ainsi que des technologies d'imagerie haute teneur et de dépistage optique en groupe pour identifier les mécanismes de la maladie et les modificateurs génétiques. La plateforme a réussi à identifier plusieurs nouvelles cibles génétiques prometteuses pour inverser les déficits liés à la SLA. Selon les termes de l'accord, insitro a reçu un paiement initial de 50 millions de dollars et pourrait gagner jusqu'à 2 milliards de dollars de jalons supplémentaires ainsi que des redevances.
insitro hat 25 Millionen Dollar von Bristol Myers Squibb (BMY) erhalten, um Entdeckungsmeilensteine zu erreichen und das erste neuartige Ziel für die Behandlung von amyotropher Lateralsklerose (ALS) auszuwählen. Dieser Meilenstein geht auf ihre Kooperationsvereinbarung aus dem Jahr 2020 zurück. Mit ihrer eigenen Plattform für maschinelles Lernen entwickelte insitro über 200 modifizierte und patientenbasierte ALS-Zelllinien sowie hochauflösende Bildgebung und poolingoptische Screening-Technologie, um Krankheitsmechanismen und genetische Modifikatoren zu identifizieren. Die Plattform identifizierte erfolgreich mehrere neuartige Genziele, die vielversprechend bei der Umkehrung von ALS-bedingten Defiziten waren. Laut den Bedingungen des Vertrages erhielt insitro eine Vorauszahlung von 50 Millionen Dollar und könnte zusätzlich bis zu 2 Milliarden Dollar an weiteren Meilensteinen sowie Lizenzgebühren verdienen.
- Received $25 million milestone payment from BMY
- Successfully identified and validated first novel ALS treatment target
- Potential to earn up to $2 billion in additional milestone payments plus royalties
- Platform demonstrated ability to reverse ALS-related deficits in testing
- None.
Insights
The milestone payments and target selection mark significant progress in developing novel ALS treatments. The
The platform's ability to revert protein and RNA profiles found in ALS patient spinal cords suggests potential therapeutic efficacy. With BMY's robust development capabilities and up to
The
The milestone achievement suggests promising early results in a high-unmet-need indication. Success in ALS could strengthen BMY's position in neuroscience and provide new revenue streams to offset patent cliffs in other areas. The deal structure aligns with industry trends of using milestone-based collaborations to access cutting-edge drug discovery platforms.
Distinctive machine learning platform combines proprietary disease models and screening techniques to reveal genetic insights that generate targets to transform ALS treatment
ALS is a devastating disease that occurs sporadically. It causes the progressive degeneration of nerve cells in the spinal cord and brain, leading to muscle spasms, impaired speech and gait, and eventual respiratory failure. Life expectancy from diagnosis is three to five years, and all cases are fatal.
To disentangle the complexity of ALS biology, insitro built and scaled a platform, using machine learning (ML), for drug discovery with three distinct proprietary elements, including:
- A collection of more than 200 engineered and patient ALS cell lines representing a comprehensive collection of ALS genetics and sporadic patients, complemented by a unique protocol for rapid, ML-enabled differentiation of motor neurons that model the disease;
- High-content imaging capabilities, fit-for-purpose for machine learning (ML), to identify disease mechanisms; and
- A proprietary, ML-enabled technology for pooled optical screening in human cells (POSH) to uncover genetic modifiers of disease-relevant cellular phenotypes.
Powered by this platform, researchers identified several novel gene targets that rescue functional deficits found in ALS patients. Knockdown of these targets reverted protein and RNA profiles that are found in ALS patient spinal cords, including proven markers of ALS pathology. These effects were also observed in cell lines capturing the genetics of ALS patients. The first credentialed target from the collaboration is now moving into drug discovery, while other targets continue to be studied.
“ALS is a grievous disease that causes immense suffering. Identifying potentially transformative targets with high conviction for such a complex disease is a testament to insitro’s state-of-the-art disease models and our cutting-edge cell-ML platform,” said Daphne Koller, Ph.D., founder and CEO of insitro. “Our team’s hard work and determination has been matched by the quality of our partnership with Bristol Myers Squibb scientists and leaders, who understood and invested in our vision early on. Thanks to both of those factors, our scientists were able to uncover meaningful insights and surface potentially viable targets that we hope will lead to meaningful treatments for patients in need.”
“Bristol Myers Squibb is committed to unlocking new frontiers in neuroscience, based on validated targets linked to causal human biology,” said Richard Hargreaves, Senior Vice President and Head of Bristol Myers Squibb’s Neuroscience Thematic Research Center. “We look forward to working with insitro to advance drug discovery for this novel target, as we strive to tackle some of the most pressing areas of unmet patient needs.”
With the selection of the first candidate target, the parties will continue to work closely to advance therapeutics targeted to this high value target and pathway. Bristol Myers Squibb will progress the program through clinical development and maintains the option for the selection of additional targets.
Under the terms of the collaboration agreement, insitro received a
About insitro
insitro is a machine learning-enabled drug discovery and development company creating a new approach for target and drug discovery. We integrate multimodal data from human cohorts and cellular models with the power of AI and machine learning. insitro is uncovering genetic targets and new therapeutic hypotheses, leveraging human and cell data to increase the probability of success. These insights provide the starting point for discovering new molecules, which we either build with our in-house, AI-enabled drug discovery platforms or with partners that extend our impact. With more than
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Gwynne Oosterbaan
gwynne@insitro.com
Dan Budwick
dan@1abmedia.com
Source: insitro
FAQ
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