Pharmaceutical Technology Editors

Catalent announced on Dec. 14, 2022 that it is set to establish a new biologics analytical center of excellence in Durham, NC, within North Carolina’s Research Triangle, to offer analytical development and testing for biologic drug modalities, including cell and gene therapies.

Catalent plans to invest up to $40 million into the 80,000-ft2 facility. When complete, the facility will provide comprehensive solutions that include bioassays, physico-chemical testing, and full product and process characterization, as well as process validation support, stability testing, in-process manufacturing and formulation analysis, and post-packaging identification. The facility is set to be completed by mid-2023 and is anticipated to support the hiring of over 200 scientists and technicians over the next five years.

“Catalent is committed to the continuous growth and expansion of its analytical capabilities to provide extensive CMC [chemistry, manufacturing, and controls] support and solutions that are compliant with current good laboratory and manufacturing practices. By establishing significant additional capacity on the East Coast, we can offer our customers greater flexibility and expanded support services across the biologics and advanced modalities pipeline,” said Jeremie Trochu, Catalent’s division head for BioAnalytics, in a company press release. “Our Kansas City and Durham sites will together provide comprehensive services and expert solutions to companies seeking analytical support across all phases of development, ultimately reducing bottlenecks and creating an accelerated path to regulatory approval for both drug substance and drug product programs across a wide range of advanced therapies.”

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Catalent is set to expand its biologics analytical services with its new facility in Durham, NC. 

Catalent to Expand its Biologics Analytical Services with New Facility

Emmes, a global clinical research organization, announced the creation of a dedicated cell and gene therapy center on Dec. 8, 2022. The facility, which will have a team of 100 researchers, is designed to support clients conducting global research in this area and will support clinical trials for clients developing cell and gene therapies across various therapeutic areas, including hematology, oncology, cardiology, ophthalmology, and rare diseases.

“Our reputation and the growth opportunities in cell and gene therapy research drove the decision to establish the center. We have supported more than 125 clinical trials that integrate cell or gene therapies and have a long record of successful collaboration with leading investigators in this field,” said Christine Dingivan, CEO, Emmes, in a company press release. “This new center centralizes our resources and facilitates expansion in our current strengths to other high-potential areas where we’re building expertise.”

“Emmes’ Cell and Gene Therapy Center will harmonize our diverse credentials across multiple therapeutic areas and functions under one roof, allowing our best practices and expertise to be leveraged even more efficiently in this complex environment of evolving therapies,” said Adam Mendizabal, vice-president, Emmes, in the release.

Emmes’ new facility is designed to support clients conducting cell and gene therapy research worldwide. 

Emmes Launches Cell and Gene Therapy Center

Vertex Pharmaceuticals, a biotechnology company based in Boston, Mass., and Entrada Therapeutics, a biopharmaceutical company also based in Boston and specializing in therapeutics derived from its endosomal escape vehicle (EEV) platform, announced a collaboration on Dec. 8, 2022. Under this $735 million collaboration, the companies will jointly work on the discovery and development of EEV therapeutics for myotonic dystrophy type 1 (DM1). The research will include ENTR-701, Entrada’s program for DM1 that is in late-stage preclinical development.

Under the agreement, Entrada will receive an upfront payment of $224 million from Vertex, as well as an equity investment of $26 million. Entrada is also eligible to receive an additional $485 million per the completion of certain research, development, regulatory, and commercial milestones, as well as tiered royalties on any net sales from products that result from this collaboration.

Also under the agreement, the companies will conduct a four-year global research collaboration in which Entrada will receive payments for research associated with ENTR-701 and other DM1-related research activities. Vertex will be responsible for global development, manufacturing, and commercialization of ENTR-701 and any additional programs stemming from the agreement.

ENTR-701 is a proprietary EEV-conjugated phosphorodiamidate morpholino oligomer designed to address the underlying cause of DM1. It achieves this through allele-specific targeting of the disease-associated trinucleotide repeats in dystrophia myotonica protein kinase transcripts. The goal of this mechanism of action is to restore the function of muscle blind-like proteins, correct the mis-splicing and aberrant expression of downstream transcripts, and restore normal muscle function.

“Vertex’s strategy is to discover and develop transformative medicines for people with serious diseases, and DM1 has therefore been a disease area of interest to Vertex for some time,” said David Altshuler, executive vice-president, Global Research, and chief scientific officer, Vertex, in a company press release. “Entrada’s innovative EEV approach, the significant progress in their DM1 program, and the potential for it to reach the clinic in the near-term hold exciting potential for patients. Working together, we believe we have the opportunity to develop a transformative treatment for this devastating disease.”

“Our collaboration with Vertex represents an important step for Entrada as we work to make intracellular therapeutics a reality through our novel EEV approach,” said Dipal Doshi, president and CEO, Entrada Therapeutics, in the press release. “DM1 is a progressive disease with no treatment options available. Working with Vertex will enable us to expeditiously move this program forward, while focusing the majority of our internal resources on advancing new therapeutic options for patients living with Duchenne and expanding our commitment to non-neuromuscular disease programs.”

Under the collaboration between Vertex and Entrada, the companies will work on discovering and developing intracellular endosomal escape vehicle therapeutics for myotonic dystrophy type 1.

Vertex and Entrada Therapeutics Enter Endosomal Escape Vehicle Collaboration 

Thermo Fisher Scientific announced the opening of a new facility in Hangzhou, China, on Dec. 8, 2022. The 80,000 m² current good manufacturing practices (CGMP) facility is designed to boost biologics and sterile development and manufacturing capabilities in China and the greater Asia-Pacific region.

According to a company press release, the facility will offer integrated clinical and commercial drug substance and drug product capabilities. This includes process development, cell line development, biologics drug substance manufacturing, and sterile fill-finish. The company also plans to feature commercial packaging and labeling capabilities in the future as well as expand its on-site bioreactors capacity from 2000 L to 5000 L.

"Hangzhou is a strategic addition to our global pharma services network and the latest example of our commitment to meeting customer demands globally,” said Mike Shafer, senior vice-president and president, Pharma Services, Thermo Fisher Scientific, in the release. “We continue to invest in capabilities to support the manufacturing needs of our customers, enabling them to serve more patients throughout the world.”

“Thermo Fisher has been in China for 40 years, serving this market through its bioproduction, clinical research, and pharma services businesses,” said Hann Pang, president, Thermo Fisher China, in the release. “Committed to our ‘in China, for China’ localization strategy, we are enhancing our workflow capabilities to fully support pharmaceutical and biotechnology companies in China and worldwide in helping more local innovations go global.”

Thermo Fisher Scientific’s new facility in Hangzhou, China, is designed to boost biologics and sterile development and manufacturing capabilities in the Asia-Pacific region.

Thermo Fisher Expands Biologics and Steriles Manufacturing Capabilities in China

 opportunity from FDA has been established for projects to further promote the potential use of real-world data (RWD) to generate real-world evidence (RWE) related to drug development. FDA hopes to encourage more projects that address these types of gaps in knowledge that can limit the use of RWD and RWE. 

The objectives of the new opportunity are to improve the quality and/or use of RWD, promote better understanding of RWE study designs, and develop specific tools to evaluate aspects of RWE generation. FDA encourages a variety of projects that are applicable to drugs and biologics, excluding devices.

To further meet the requirements of the 21st Century Cures Act, FDA developed an RWE Program and subsequently published an RWE Framework and as a series of RWD/RWE draft guidance documents about the use of RWE to assist a new indication for an approved drug or to satisfy post-approval study requirements.

In addition, FDA funded various demonstration projects involving external collaboration and engagements, including four U01 awards in 2020 that analyzed the use of RWD and RWE. The projects are almost complete, and the announcement of this recent funding opportunity emphasizes FDA’s efforts to continue to encourage innovative approaches and improved understanding of RWD and RWE.

FDA Announces New Funding Opportunity to Support the Potential Use of Real-World Data to Generate Real-World Evidence for Regulatory Decision-Making, Press Release, Dec. 15, 2022

The objectives of the new opportunity are to improve the quality and/or use of RWD, promote better understanding of RWE study designs, and develop specific tools to evaluate aspects of RWE generation.

FDA to Support Potential Use of Real-World Data to Generate Real-World Evidence for Regulatory Decision-Making

Amgen announced on Dec. 12, 2022 that it has agreed to acquire Horizon Therapeutics, an Irish biotechnology company focused on rare, autoimmune, and severe inflammatory diseases, for approximately $27.8 billion. According to a company press release, the move is intended to complement and strengthen Amgen’s therapeutics portfolio through receipt of Horizon’s rare disease therapeutics portfolio, while also allowing the company to “enhance the growth potential” of the newly acquired portfolio.

"The acquisition of Horizon is a compelling opportunity for Amgen and one that is consistent with our strategy of delivering long-term growth by providing innovative medicines that address the needs of patients who suffer from serious diseases,” said Robert A. Bradway, chairman and CEO, Amgen, in a company press release. “The potential new medicines in Horizon's pipeline strongly complement our own R&D portfolio. The acquisition of Horizon will drive growth in Amgen's revenue and [non-generally accepted accounting principles earnings per share] and is expected to be accretive from 2024."

"In nearly 15 years, we have built one of the fastest growing and most respected companies in the biotechnology industry from the ground up. We have accomplished a tremendous amount for patients, their families, and our customers, and created significant value for shareholders. These accomplishments are all rooted in our employees' deep commitment, dedication, and personal passion for those impacted by rare, autoimmune, and severe inflammatory diseases,” said Tim Walbert, chairman, president, and CEO, Horizon Therapeutics, in the release.

Amgen will purchase all shares of Horizon at $116.50, a premium of approximately 48% on their closing price of $78.76. A closing date was not disclosed in the initial press release.

Amgen’s $27.8 billion acquisition of Horizon Therapeutics, an Irish biotechnology company, is one of the largest of 2022.