Pharmaceutical Technology Editors

Roche, a Swedish biotechnology company, announced on Feb. 7, 2023 positive data from its Phase III study (COMMODORE 2) evaluating the efficacy and safety of crovalimab, an investigational, novel anti-C5 recycling monoclonal antibody, in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood condition that causes the complement system to destroy red blood cells. The treatment is for PNH patients who have not been previously treated with complement inhibitors.

According to a company press release, crovalimab is designed to control PNH via a subcutaneous injection every four weeks. In this study, crovalimab met its co-primary efficacy endpoints of transfusion avoidance and control of hemolysis, or the ongoing destruction of red blood cells. Study results indicated that the drug achieved disease control and was non-inferior to eculizumab, a current standard of care given intravenously every two weeks.

“People with PNH may benefit from more options to achieve robust disease control with less frequent treatment intervals,” said Levi Garraway, chief medical officer and head of Global Product Development, Roche, in the release. “As the first global [P]hase III data for crovalimab, these results emphasize its potential to address these needs. We look forward to submitting these data to regulatory authorities, bringing us one step closer to making crovalimab available for people with PNH around the world.”

Articles

Roche’s Phase III global program, centered around the biologic crovalimab as a treatment for paroxysmal nocturnal haemoglobinuria, showed the drug was non-inferior to current standards of care.

Roche Reports Positive Data for Rare Blood Condition Treatment

In the latest closing report of the European Medicines Agency (EMA) and European Commission action plan on pediatrics in February 2023, there were key improvements highlighted to help with children’s medicines and combatting challenges that have been previously mentioned.

For example, over the past four years, EMA and relevant stakeholders wanted to better identify and raise awareness on the areas where medicines for children are particularly needed. Clinicians, pediatric patients, regulators, and more are being considered to further strengthen focus on unmet medical needs.

Another goal of the plan is to adapt regulatory processes to better support innovation to expedite the creation of pediatric investigation plans (PIPs) and to adapt and simplify processes overall. Under framework, like the stepwise PIP agreement, it will become possible in certain cases to agree on a partial development program. This will help to agree on PIPs for innovative medicines where crucial information needed to define certain parts of the plan is not yet available. 

Lastly, an increased alignment of data requirements between decision-makers will help to facilitate the compatibility of pediatric requirements between regulators, international partners, FDA, and more. This work has also been seen through the European network of pediatric research at EMA (Enpr-EMA) to help align international requirements for pediatric clinical trials authorization and standards.

The pediatric action plan was established to provide some immediate solutions to these challenges under the current regulatory framework. All these initiatives will contribute to the application of the Pediatric Regulation, with the adoption of the proposal expected by March 2023.

Clinicians, pediatric patients, regulators, and more are being considered to further strengthen focus on unmet medical needs.

Recent Initiatives to Support Development of Medicines for Children 

Thermo Fisher Scientific and Celltrio announced on Feb. 6, 2022 their collaboration to bring a fully automated cell culture system to biotherapeutics customers. The collaboration will integrate the Thermo Scientific Momentum workflow scheduling software with the Celltrio RoboCell cell line automation platform to address the critical unmet market need for high-throughput automated cell line culturing and maintenance.

Thermo Fisher will also immediately take on exclusive responsibility for sales, installation, and support of the RoboCell system in Europe. Celltrio’s RoboCell systems distributed in Europe will now use Momentum software for work scheduling and date management.

“Cell line scale-up challenges have long stifled the full potential of biotherapy research. While researchers have been able to automate some aspects of cell line production, full automation has been well out of reach,” said Hansjoerg Haas, senior director and general manager, laboratory automation, Thermo Fisher Scientific, in a company press release. “By collaborating with Celltrio, we’re bringing together the capabilities that make fully automated cell line culturing a reality. With the ability to effectively scale processes, biotherapeutics customers can focus on getting their innovative therapies to patients, paving the way to a better, healthier world.”
Source: 

Thermo Fisher and Celltrio are introducing a new automation platform to address critical scale-up challenges in biotherapeutics. 

Thermo Fisher Scientific Collaborates to Introduce Fully Automated Cell Line Automation Platform

Astellas announced an overhaul of its corporate management structure on Feb. 6, 2023. Among changes throughout the entire management chain, Naoki Okamura, Astellas’ current chief strategy officer and corporate executive vice-president, will take over as company CEO and president from Kenji Yasukawa. Yasukawa will stay on with the company as representative director and chairman of the board.

According to a company press release, after bottoming out in 2020, revenue has been on an upward trend; this change is an aggressive move to further accelerate company growth. It follows a transition in a business model that created products with a focus on specific disease areas to one that determines R&D areas from a “multifaceted perspective.”

In addition to changes at the top, the company has also appointed Claus Zieler as chief commercial officer, Adam Pearson as chief strategy officer, and Minoru Kikuoka as corporate executive vice-president.

Naoki Okamura, Astellas’ current chief strategy officer, will take over the role from Kenji Yasukawa.

Astellas to Appoint New CEO and President

The Center for Breakthrough Medicines (CBM), a contract development and manufacturing organization, announced the launch of its plasmid manufacturing offering, Precision Plasmids, on Feb. 2, 2023. The service is designed to provide phase-appropriate plasmids on demand for companies working on cell and gene therapies.

According to a company press release, the service uses next-generation sequencing to guarantee reproducibility of vector-based therapies that are manufactured using plasmids. Expected turnaround times are 12 weeks for plasmids from a good manufacturing practice (GMP) master bank, or 17+ weeks for those working from a starting R&D plasmid.

“As a starting material for mRNA [messenger RNA] vaccines and key components for vector-based cell and gene therapies, plasmids are in high demand for the foreseeable future,” said Dana Cipriano, senior-vice-president, Testing and Analytical Services & Plasmids, CBM, in the release. “CBM’s Precision Plasmids solve the capacity challenge by bringing a platform process, multiple scales, and segregated suites to meet any clinical and commercial production needs on demand.”

The plasmids are offered in three varieties: R&D, Pro, and GMP. The R&D plasmids grade for pre- and early clinical phases, while the Pro grade for toxicology studies, Phase I to II vector production, or as a starting material for mRNA. GMP grade services are expected to launch in April 2023.

CBM’s plasmid manufacturing offering is designed to provide phase-appropriate plasmids on demand for companies working on cell and gene therapies.

The Center for Breakthrough Medicines Announces Plasmid Manufacturing Service

iotaSciences, a spin-out from Oxford University specializing in single-cell handling solutions, announced completion of its new single-cell picker instrument on Feb. 6, 2023. The isoPick is designed to provide temperature-controlled handling of single cells during isolation, as well as picking through a workflow guided by touchscreen interfaces.

According to a company press release, the isoPick is intended for cell and gene therapy researchers, as well as the wider single-cell research community, as a means of providing verifiable isolation of single cells with high cell viability and stable single-cell outgrowth. Like the company's other instruments, it is designed to have a small footprint instrument that requires minimal maintenance.

iotaSciences isoPick is designed to expand handling solutions for cell biology and gene therapy applications.