Pharmaceutical Technology Editors

FDA’s Center for Drug Evaluation and Research announced a new virtual workshop slated to happen on March 23–24, 2022. The public meeting, “Identification of Concepts and Terminology for Multi-Component Biomarkers,” will be held to discuss high-level terminology surrounding biomarkers.

According to an FDA press release, the goal of the symposium is to develop multi-component biomarker concepts and terminology, identify areas of conceptual language development, and discuss gaps in terminology for concepts and approaches as it relates to multi-component biomarkers.

The symposium will be held via live webcast for the public from 1:00–3:00 pm ET on both days. A full breakdown of the events schedule can be found 

. The public will also gain access to additional pre-recorded talks three weeks prior to the live event, which FDA recommends viewing ahead of the event.

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FDA will hold a symposium discussing the identification of concepts and terminology for multi-component biomarkers from March 23–24, 2022.

FDA to Hold Public Symposium on Multi-Identification Biomarkers

On Feb. 15, 2022, Thermo Fisher Scientific announced that it is investing $40 million to expand its single-use technology manufacturing facility in Millersburg, Penn. The expansion is part of a $650 million multi-year investment to enhance the company’s ability to provide flexible, scalable, and reliable bioprocessing production capacity. Thermo Fisher announced the 

. The expanded capacity will be for manufacturing critical materials used in developing new and existing biologics and vaccines, including for COVID-19.

Thermo Fisher has been expanding the Millersburg facility over the past two years after the facility was initially acquired in 2015. That first phase of expansion included the modernization of warehouse space and clean rooms. The second phase of expansion will include a new 47,000-ft

 warehouse and the creation of 100 new jobs at various levels. The facility is expected to be fully renovated by mid-2023 and will employ more than 1000 people upon completion.

“Since the start of the pandemic, the Millersburg site has been instrumental in supporting more than 20 pharmaceutical partners developing lifesaving therapies and vaccines,” said Mitch Kennedy, president, single use technologies, Thermo Fisher Scientific, in a company press release. “During the pandemic, our colleagues in Millersburg and around the world answered the call for increased production. Our expanded bioprocessing capabilities will ensure that we can continue to deliver essential supplies to our customers as demand surges.”

Thermo Fisher Scientific is expanding its Millersburg, Penn., site with a $40 million investment to support increased production of single-use technology for critical vaccines and biologics.

Thermo Fisher Scientific Invests $40 Million to Expand Bioprocessing Manufacturing Site

Biogen, a biotechnology company, and Xbrane Biopharma AB, a biotechnology research and development company, announced on Feb. 7, 2022, that they had entered into an agreement to develop, manufacture, and commercialize Xcimzane.

Xcimzane is a preclinical monoclonal antibody that is a proposed biosimilar referencing CIMZIA (certolizumab pegol). Certolizumab pegol is typically used in the treatment of rheumatoid arthritis, although it has also seen use in axial spondyloarthritis, psoriasis, and Crohn’s disease.

“We aim to bring more biosimilars products to more patients and more geographies and we are excited to bring this additional asset to our Biosimilars pipeline,” said Ian Henshaw, Head of Global Biosimilars at Biogen. “This preclinical biosimilar candidate has the potential to add another option for patients living with rheumatoid arthritis and other indications.”

“Given their vast development and commercialization experience, we are convinced that Biogen is the best possible partner we could have for Xcimzane,” said Martin Åmark, CEO of Xbrane Biopharma AB. “Today’s announcement confirms Xbrane’s ambition to become a global biosimilar developer.”

Per the terms of the agreement, Xbrane will be responsible for the completion of preclinical development of Xcimzane. Biogen will make an upfront payment of $8 million to Xbrane. Xbrane will also be eligible to receive up to $80 million in potential milestone payments, as well as tiered royalties. Biogen will gain exclusive global regulatory, manufacturing, and commercial rights to the drug.

The commercialization and licensing deal will see Biogen commercialize Xbrane’s Xcimzane, a proposed biosimilar for CIMZIA (certolizumab pegol).

Biogen and Xbrane Ink Agreement for Proposed Biosimilar

Intellia Therapeutics, a gene-editing company, and ONK Therapeutics, a company developing natural killer (NK) cell therapies in oncology, announced a licensing and collaboration agreement on Feb. 15, 2022. NK cells are specialized, naturally-occuring cells that are critical in immune activation against abnormal cells, including cancer cells.

According to a company press release, ONK will receive a non-exclusive license to Intelia’s proprietary 

 clustered regularly interspaced short palindromic repeats (CRISPR)/ associated protein-9 (Cas9)-based genome editing platform and its lipid nanoparticle (LNP)-based delivery technologies for the development of five allogeneic NK cell therapies. Additionally, ONK will receive exclusive rights to certain Intellia guide-RNAs (gRNAs) resulting from the collaboration for use in engineering the NK cell products.

“We look forward to working with ONK in the development of allogeneic NK cell therapies for patients with cancer,” said John Leanorad, president and CEO, Intellia, in the press release. “This collaboration, which combines Intellia’s industry-leading CRISPR technology platform and ONK’s expertise in NK cell technology, offers yet another powerful example of how we’re leveraging our strategic collaborations to address life-threatening diseases for patients in need.”

 genome editing and LNP delivery platforms with our suite of proprietary NK cell gene edits has the potential to create optimally engineered NK cells with enhanced cytotoxicity, persistence and an improved metabolic profile that hold tremendous promise to advance the treatment of both hematologic malignancies and solid tumors,” said Chris Nowers, CEO, ONK Therapeutics, in the press release. “We are excited to partner with Intellia and are looking forward to a collaboration that allows us to continue to deliver against our strategy, as we evolve into a clinical-stage company.”

ONK will be responsible for both preclinical and clinical development of the engineered NK cell therapies. Intellia will receive up to $184 million per product in development, various milestone payments, and up to mid-single digit royalties on potential future sales. Intellia will also be given the option to co-develop and co-commercialize up to two products worldwide, and will have the rights to lead commercialization in the U.S.

ONK will receive non-exclusive rights to Intellia’s proprietary ex-vivo CRISPR/Cas9 technology in the development of five allogeneic NK cell therapies.

Intellia and ONK Therapeutics Enter Gene-Editing Partnership

ImmunoGen, a US-based biopharmaceutical company specializing in antibody-drug conjugates (ADCs) for treating cancer, announced on Feb. 15, 2022 that it has entered into a global, multi-year definitive licensing agreement with Eli Lilly and Company (Lilly) that grants Lilly exclusive rights to research, develop, and commercialize ADCs directed to targets selected by Lilly based on ImmunoGen's camptothecin technology. The deal is potentially worth more than $1.7 billion.

Under the agreement, Lilly will pay ImmunoGen $13 million upfront for initial targets selected by Lilly. Lilly may also select a pre-specified number of additional targets under which ImmunoGen will be eligible to receive an additional $32.5 million in exercise fees if Lilly licenses the full number of targets. ImmunoGen is also eligible to receive up to $1.7 billion in potential target-program exercise fees and milestone payments dependent on certain pre-specified development, regulatory, and commercial milestones. ImmunoGen will be further eligible to receive tiered royalties as a percentage of worldwide commercial sales by Lilly. Lilly, meanwhile, is responsible for all costs associated with research and development. ImmunoGen will retain full rights to the camptothecin platform for all targets not covered by the Lilly license.

Camptothecins are a class of anticancer drugs targeting Type I topoisomerase. ImmunoGen's proprietary class of camptothecin linker-payloads are designed to optimize existing camptothecin technology, which allows for the potential delivery of a wider therapeutic window with enhanced safety and efficacy.

"Lilly has a proven track record of bringing transformative oncology medicines to market, and we are pleased that they selected our novel camptothecin technology to integrate with their efforts to develop next-generation ADCs," said Stacy Coen, ImmunoGen's senior vice-president and chief business officer, in a company press release. "This licensing agreement demonstrates ImmunoGen's continued innovation in ADCs, creates value from our intellectual property around a proprietary platform, and further enhances our ability to re-invest in our business as we build out our pipeline and accelerate our transformation into a fully-integrated oncology company."

ImmunoGen and Eli Lilly and Company have entered into an agreement that gives Lilly exclusive rights to research, develop, and commercialize ADCs designed for targets selected by Lilly from ImmunoGen’s camptothecin technology.

ImmunoGen Grants Lilly Exclusive Research, Development, and Commercialization License in $1.7+ Billion ADC Deal

FDA announced on Feb. 11, 2022, that it has granted an Emergency Use Authorization (EUA) to Eli Lilly and Company for bebtelovimab, a monoclonal antibody (mAb) designed for treatment of COVID-19. The EUA covers treatment of mild to moderate COVID-19 symptoms in adult and pediatric patients, with a positive COVID-19 test, who are at high risk for progression to severe COVID-19 and for whom alternative FDA approved/authorized COVID-19 treatment options are not accessible or clinically appropriate. According to FDA’s press release, laboratory testing showed that bebtelovimab retains activity against both the omicron variant and the BA.2 omicron subvariant.

The decision was based on a Phase II, randomized, single-dose clinical trial evaluating the efficacy of bebtelovimab alone and bebtelovimab combined with other mAbs in treating mild to moderate COVID-19. Rates of COVID-19 related hospitalization and death through Day 29 seen in those who received bebtelovimab alone or with other mAbs were generally lower than the placebo rate reported in prior trials of other mAbs in high-risk patients. However, FDA notes that conclusions are limited because this data compares different clinical trials done at different times, with different dominant variants.

“Today’s action makes available another monoclonal antibody that shows activity against omicron, at a time when we are seeking to further increase supply,” said Patrizia Cavazzoni, director of FDA’s Center for Drug Evaluation and Research, in the press release. “This authorization is an important step in meeting the need for more tools to treat patients as new variants of the virus continue to emerge.”

FDA has granted Eli Lilly and Company’s bebtelovimab, a monoclonal antibody designed for treatment of COVID-19, an Emergency Use Authorization (EUA).