Industrial Impact
MOMA Therapeutics | January 04, 2024
MOMA Therapeutics, a biotechnology company focused on identifying and targeting highly dynamic, difficult to drug targets in cancer and other diseases, is pleased to announce a strategic collaboration and licensing agreement with Roche. This partnership provides Roche with access to MOMA’s proprietary KnowledgeBase platform for the identification and prosecution of a certain number of novel drug targets involved in promoting cancer cell growth and survival.
MOMA’s KnowledgeBase comprises integrated structure-function capabilities, advanced lead-finding technologies and computation-enabled lead optimization. It was built upon the concept that functionally related targets lacking sequence homology still possess three dimensional structural motifs that can be exploited to produce highly impactful therapies. To date, MOMA has utilized this bespoke platform to accelerate drug discovery in the ATPase target class, a class with a high number of genetically validated targets for which industry efforts to identify therapeutically viable drugs have been hampered by the extent of dynamic protein motion.
“Given its deep expertise and global footprint in oncology, Roche represents an ideal collaborator with whom to further advance the application of MOMA’s platform in a way that impacts patients’ lives. The vision for this collaboration was crafted jointly with Roche to enable each party to bring its strengths in pursuit of this shared goal. It also contributes to the long-term sustainability of MOMA’s core focus as we advance our rich pipeline of precision oncology programs to the clinic,” said Asit Parikh, M.D., Ph.D., chief executive officer of MOMA.
Through the collaboration, MOMA will receive $66 million as an upfront cash payment and is also eligible to receive discovery, development, and commercialization milestone payments potentially exceeding US$2 billion, as well as tiered royalties. MOMA will be primarily responsible for all activities for selected targets through to development candidate confirmation, whereas Roche will be responsible for IND-enabling activities and clinical development and commercialization. Additionally, if multiple collaboration assets reach pivotal clinical studies, MOMA will receive a right to co-fund late-stage development of one product in exchange for increased royalties in the US on this product.
“We are excited to join forces with MOMA, combining our leadership in oncology with MOMA’s deep expertise in drug discovery for difficult-to-drug and novel targets in oncology. The broader field of cancer dependencies is of high importance for Roche and we are looking forward to further deepening our knowledge and discovering novel targets involved in cancer cell growth and survival leveraging MOMA’s innovative platform,” said James Sabry, M.D., Ph.D., global head of pharma partnering, Roche.
“It is exciting to utilize our industry-leading knowledge in how to identify and drug highly dynamic proteins to deliver on a breadth of discovery programs in partnership with Roche,” added Peter Hammerman, M.D., Ph.D., chief scientific officer and head of development at MOMA. “Along with bringing two MOMA-owned high-impact programs to the clinic next year, we are making exciting progress towards our goal of addressing key unmet needs for patients living with advanced cancer.”
About MOMA Therapeutics
MOMA Therapeutics is committed to discovering the next generation of precision medicines by targeting highly dynamic proteins that underlie human disease. Bringing together seminal scientific advancements in biochemistry, biophysics, structural biology, chemistry, computation, and functional genomics, the company has established the KnowledgeBase platform to exploit a key vulnerability inherent to all dynamic proteins: their dependence on well-coordinated, stepwise changes in protein conformation. By focusing this platform on disease-causing targets, MOMA aims to develop high impact, precision medicines for patients with unmet medical needs. MOMA Therapeutics is a private company launched in 2020 with seasoned leadership, a highly specialized workforce with deep expertise in oncology discovery, world-class scientific founders, and financed by leading biotech investors.
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Medical
Andelyn Biosciences, Inc. | January 16, 2024
Andelyn Biosciences, Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has partnered with Armatus Bio to accelerate manufacturing of their gene therapy treatment for Charcot-Marie-Tooth Type 1A (CMT1A), a rare genetic neurological disease associated with independence-limiting disability and risk of fatal complications that has no approved therapies today. The partnership will seek to maximize program efficiency and speed to clinical studies of this novel, precision approach.
As part of the agreement, Armatus will leverage Andelyn's extensive experience in adeno-associated virus (AAV) production and its proprietary suspension platform for development activities, plasmid manufacturing, and viral vector toxicology and GMP clinical manufacturing. The partnership strengthens the working relationship and offers great hope for patients suffering from neuromuscular disease.
Both organizations, located in the well-established and growing biotech hub of Columbus, Ohio, are aligned in their belief that the rise of gene therapies will continue to advance human health.
Matt Niloff, Chief Commercial Officer at Andelyn Biosciences said, "We are extremely moved by Armatus Bio's commitment to address the urgent unmet needs in CMT1A and are impressed by its innovative therapeutic technology for the disease. We look forward to scaling up the process in our program-specific, configurable suspension platform, and accelerating the therapy into the clinic with the highest degree of quality."
"Andelyn has demonstrated its strong capabilities in manufacturing complex genetic medicines with high quality and consistency, which will be critical to accelerating our development efforts," said Brian Price, PhD, Chief Technical Officer of Armatus. "We look forward to collaborating with Andelyn on this program as we work toward supporting this population that urgently awaits innovative solutions."
From its time-tested experience, Andelyn is proud to offer its clients exceptional development, plasmid production, and quality manufacturing at its three Columbus, Ohio facilities. Andelyn is advancing client discoveries and manufacturing life-altering cell and gene therapies for rare and prevalent diseases.
About Andelyn Biosciences, Inc.
Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization and production of viral vectors for gene therapy. With more than 20 years of experience, Andelyn's deep scientific expertise has resulted in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of three Columbus, Ohio facilities, Andelyn supports its clients in developing curative cell and gene therapies from concept through plasmid development and manufacturing, process development, and cGMP clinical and commercial manufacturing. Andelyn's versatile capabilities include cGMP manufacturing capacity for both adherent and suspension processes up to a 2,000-liter capacity. An advanced digital model, quality system, full regulatory support and supply chain vertical integration help Andelyn accelerate the development and manufacturing of its clients' innovative cell and gene therapies.
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. The company's lead candidate for Charcot Marie Tooth Type 1A (CMT1A) has received Orphan Drug Designation and Rare Pediatric Drug Designation, and is advancing toward IND-enabling studies. The company is also developing a preclinical vectorized RNAi asset for the rare neuromuscular disease Facioscapulohumeral Muscular Dystrophy (FSHD).
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Industrial Impact
Nimbus Therapeutics | January 08, 2024
Nimbus Therapeutics, LLC a biotechnology company that designs and develops breakthrough medicines through its powerful computational drug discovery engine, announced the advancement and expansion of its pipeline with the addition of discovery programs targeting innate immunity pathways. These programs, targeting the salt-inducible kinase (SIK) family and cyclic GMP-AMP synthase (cGAS), represent promising opportunities to leverage Nimbus’ industry-leading computational and structure-based drug design expertise to develop highly selective, potent medicines addressing areas of significant unmet need.
"Building on the success of our TYK2 program, we are broadening our drug discovery engine to unlock new difficult-to-drug targets with compelling biology,” said Peter Tummino, Ph.D., Chief Scientific Officer of Nimbus. “SIK and cGAS are critical targets in highly prevalent diseases that are well suited to Nimbus’ structure-based drug design approach. We look forward to advancing our discovery and development programs across oncology, immunology, and metabolism to deliver transformative medicines to patients.”
Nimbus is advancing its Phase 1/2 trial (NCT05128487) of NDI-101150, a small molecule inhibitor of hematopoietic progenitor kinase 1 (HPK1), and has reported positive dose escalation data showing potential monotherapy clinical benefit for patients with solid tumors. Furthermore, the company expects to initiate IND-enabling activities this year for its oncology program targeting Werner syndrome helicase (WRN). In collaboration with Eli Lilly and Company, Nimbus continues to progress the development of novel targeted therapies that activate AMPK to potentially treat a broad range of metabolic disorders.
Nimbus continues to expand its platform capabilities with ongoing investments in cutting-edge technology for drug discovery. Alongside proprietary computational tools which the company has developed in-house, Nimbus’ platform leverages state-of-the-art technology through collaborations such as its recently announced partnership with Anagenex, a leader in generative AI for drug design.
Jeb Keiper, M.S., MBA, Nimbus’ Chief Executive Officer, will provide an overview of the company’s progress and pipeline and anticipated milestones for 2024 and beyond at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 at 7:30 am PT.
About Nimbus Therapeutics
Nimbus Therapeutics is a clinical-stage, structure-based drug discovery company developing novel small molecule medicines designed to act against well-validated but difficult-to-drug targets implicated in multiple human diseases. Nimbus combines leading-edge computational technologies with a tailored array of machine learning-based predictive modeling approaches. Nimbus’ pipeline includes a clinical-stage HPK1 inhibitor for the treatment of cancer (NCT05128487), as well as a diverse portfolio of preclinical programs focused on cancer, autoimmune conditions, and metabolic diseases. Nimbus is headquartered in Boston, Mass.
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Cell and Gene Therapy
Solvias | January 23, 2024
Solvias, a global provider of chemistry, manufacturing, and control (CMC) analytics, announced that it will perform analytical release testing services on the world's first CRISPR/Cas9 genome-edited cell therapy. The company has signed a long-term agreement with Vertex Pharmaceuticals for CASGEVY™ (exagamglogene autotemcel or exa-cel) which received U.S. Food & Drug Administration (FDA) approval for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises.
Solvias and Vertex Pharmaceuticals have worked together for several years to develop and validate test methods that are critical for the final release of patients' own edited cells, so that they can be delivered back to patients. The companies' collaboration included establishing the testing methods that will be scaled for commercializing CASGEVY. Solvias also has invested significantly in preparing one of its global facilities to support the commercial release work for this transformative therapy.
Archie Cullen, Chief Executive Officer, Solvias, stated
"Solvias is honored to be playing a critical role in delivering this breakthrough therapy to patients. Our decades of experience offering comprehensive GMP analytical services uniquely positions us to partner with companies in bringing their therapies to market. This collaboration highlights our deep scientific knowledge and creative solutions serving to advance even the most cutting-edge therapies."
In addition to gaining FDA approval, CASGEVY recently received a positive opinion from the European Medicines Agency's (EMA's) Committee for Medicinal Products for Human Use for conditional approval of the treatment of severe SCD and transfusion-dependent beta thalassemia (TDT).
About Solvias
Solvias is a global provider of chemistry, manufacturing, and control (CMC) analytics to the pharmaceutical, biotech, material science, and cosmetic industries. Its team of scientists and regulatory experts have years of experience in small molecules, biologics, and cell and gene therapies. The company offers comprehensive solutions from raw materials to drug products to final release testing, as well as API development and manufacturing for small molecules. Headquartered near Basel, Switzerland, Solvias operates five facilities to the highest standards and in accordance with ISO, GMP, GLP and FDA regulations.
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