Pharmaceutical Technology Editors

Centogene, a commercial-stage data-generating company, has expanded its partnership with Agios Pharmaceuticals, under which Centogene will provide genetic testing and clinical trial support for Agios’ three global, pivotal trials in thalassemia and sickle cell disease. Under a previous agreement, Centogene provided support for Agios’ pyruvate kinase deficiency clinical program. 

The companies signed a commercial three-year fee-for-service agreement under which Agios will coordinate and bear the costs for the programs. Further financial terms were not disclosed. The collaboration will offer patients access to genetic testing to help identify causative mutations, including HBA1, HBA2, and HBB genes, Centogene stated in a Nov. 4, 2021, press release. 

Agios’ global clinical trials will enroll patients in North America, Europe, the Middle East and North Africa, the Asia-Pacific region, and Latin America. Samples will be collected using Centogene’s proprietary CentoCard for dried blood spot analysis. 

Centogene’s rare disease-centric Bio/Databank is positioned to accelerate precise diagnosis and allows for the continued buildup of insights that can help with analyzing the genetic factors causing rare diseases.

“Our data-driven insights enable both acceleration and de-risking of clinical trials,” said Andrin Oswald, CEO of Centogene, in the press release. “Combining both [Centogene]’s expertise in genetics and unique global insights will lead to a better understanding of the disease biology of those rare diseases. This partnership will support the advancement of Agios’ first-in-class PK activator as a potential therapy for thalassemia and sickle cell disease, two under-served patient communities in need of new treatment options.”

“As we look ahead to initiating three global, pivotal trials in thalassemia and sickle cell disease by the end of the year, we are pleased to expand our partnership with [Centogene] to better understand the underlying genetics of patients in our studies and the potential impact of our investigational medicine,” said Sarah Gheuens, chief medical officer at Agios, in the press release.

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Under the expanded agreement with Agios, Centogene will provide global clinical trial support for thalassemia and sickle cell diseases.

Centogene Expands Rare Blood Disease Partnership with Agios

Fujifilm is expanding its Armstrong R&D Center of Excellence at its headquarters in California, the company announced in a Nov. 10, 2021, press release. The expansion supports Fujifilm Irvine Scientific’s discovery research and its specialty media, technologies, and custom media services.

“With society’s increasing demand for global access to more high-quality, cost-effective vaccines, biopharmaceuticals, cell-based therapies, gene therapies, and 

 fertilization procedures, the need for a broadened focus on innovation is pivotal to expedite critical product and technology development,” said Bob Newman, chief scientific officer, Fujifilm Irvine Scientific, in the press release. “The expansion project will more than double the R&D facility’s footprint and will allow for more enhanced technical collaboration between R&D experts and customers to develop personalized solutions and enhance partnership opportunities.”

The expansion supports the company’s initiatives in manufacturing sciences and technologies, single-use technology development, and automation platforms for media development. The expanded footprint will also include dedicated space for analytical chemistry, virology, assisted reproductive technology, and cell-culture and molecular-based assay development. Facility construction began in October and is planned for completion by January 2022. The company expects to double its R&D headcount by the close of 2022.

Fujifilm is expanding its R&D footprint at its California facility. 

Fujifilm Irvine Scientific Expands Cell Culture R&D Center 

Baxter International announced its plans to invest approximately $100 million in expansions to its sterile fill/finish manufacturing facility in Halle/Westfalen, Germany on Nov. 11, 2021. The Halle/Westfalen facility is operated by Baxter BioPharma Solutions (BPS), a division of Baxter specializing in contract development and manufacturing of drug products for parenteral pharmaceuticals. Construction on the new facility is expected to begin in 2022 and complete in 2024.

The planned additions include new lyophilization equipment and a new aseptic syringe filling line. According to a company press release, the new lyophilization equipment is intended to further increase product stability and shelf life, while the additional aseptic line will help the company meet growing demand for pre-filled syringes in Europe and the United States.

“Baxter’s manufacturing site in Halle/Westfalen is one of the most advanced facilities in our global network and has seen significant growth over the last several years,” said Marie Keeley, general manager, Baxter BioPharma Solutions, in the press release. “By making this investment, we are building on recent expansions to help ensure this facility can serve our partners at the highest level now and well into the future.”

Baxter will invest $100 million in new lyophilization equipment and an additional aseptic syringe filling line at its Halle/Westfalen (Germany) location.

Baxter Invests in its German Sterile Fill/Finish Manufacturing Facility

Sotera Health, a global provider of sterilization, lab-testing, and advisory services, announced on Nov. 9., 2021, that it had acquired Regulatory Compliance Associates (RCA), a Wisconsin-based life sciences consulting company. Terms of the deal were not disclosed.

Founded in 2000, RCA specializes in quality, regulatory, and technical consulting for the pharmaceutical, medical device, and combination device industries. According to a company press release, this acquisition will allow RCA’s consulting capabilities to complement and enhance Sotera Health’s existing strengths in technical consulting and expert advisory services.

“Regulatory Compliance Associates is a premier provider of quality and regulatory services; known for its best-in-class solutions. It is also recognized for its strong relationships with its customers and regulators,” said Michael B. Petras Jr., chairman and CEO of Sotera Health, in the press release. “This acquisition will expand and further strengthen our technical consulting capabilities. We welcome RCA’s strong management and expert staff as they join us in our mission, Safeguarding Global Health.”

“Our mission is to provide our customers with unsurpassed technical expertise combined with quality service. We have earned a reputation for our customer service and are seen as an essential partner for our customers—and we know we have found the same in Sotera Health,” said Erika Porcelli, CEO of RCA, in the press release. “My goal for RCA is to continue our commitment to serve customers supporting their regulatory and compliance needs as they bring critical pharma and medical device products to market. We accomplish this by becoming part of Sotera Health, a global company whose breadth of services and expansive reach provide extraordinary value to the industries we are proud to serve.”

Sotera’s acquisition of Regulatory Compliance Associates (RCA) will expand its technical consulting services.

Sotera Health Acquires Life Sciences Consulting Company

REGENXBIO announced the closing of its collaboration and license agreement with AbbVie to develop and commercialize its gene therapy candidate, RGX-314, on Nov. 9, 2021. Under the terms of the agreement, REGENXBIO will receive an upfront payment of $370 million from AbbVie, with an additional $1.38 billion to be paid should additional development, regulatory, and commercial milestones be met.

RGX-314 is a potential one-time gene therapy for treatment of wet-age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases. According to a company press release, RGX-314 encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). VEGF pathways are believed to result in leaky blood vessels that contribute to the accumulation of fluid in the retina.

Currently, REGENXBIO is researching two separate routes of administration for RGX-314 to the eye. One is through a standardized subretinal delivery procedure, while the other delivers it through suprachoroidal space. The company has licensed exclusive rights to the SCS Microinjector from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.

Per the terms of the deal, REGENXBIO will be responsible for completion of the ongoing trials of RGX-314; costs of future trials will be shared across both companies. The companies will equally share any RGX-314 net sales in the United States, whereas AbbVie pay REGENXBIO tiered royalties on net sales of RGX-314 outside the U.S.

AbbVie will pay REGENXBIO up to $1.75 billion for the rights to develop and commercialize its treatment of various eye conditions.

REGENXBIO and AbbVie Officially Ink Eye Care Collaboration Deal Worth Up to $1.75 Billion

Rafael Pharmaceuticals, a pharmaceuticals company focused on cancer metabolism-based therapeutics, announced that the European Medicines Agency (EMA) had granted orphan drug designation (ODD) to their lead compound, CPI-613 (devimistat), for the treatment of refractory or relapsed Burkin’s lymphoma, a rare variant of non-Hodgkin’s lymphoma (NHL).

According to a company press release, approximately two out of 100 cases of NHL in the United Kingdom are identified as Burkitt’s lymphoma. However, it disproportionately affects children with NHL, affecting up to 30% of pediatric patients. Besides first-line therapy, no substantial treatment options currently exist for this form of NHL.

“Relapsed or refractory Burkitt’s lymphoma can be a devastating form of cancer that is in need of effective treatment options,” said Sanjeev Luther, president and CEO of Rafael Pharmaceuticals, in the press release. “Milestones such as this can offer hope for the patients and families who suffer from this rare form of lymphoma with few options to turn to.”

According to the release, devimistat is designed to target the mitochondrial tricarboxylic acid cycle, which is essential to tumor cell multiplication and survival. This is the third ODD granted to devimistat by EMA, following designations for pancreatic cancer and acute myeloid leukemia. Additionally, FDA has granted devimistat seven ODD indications, including one for Burkitt’s lymphoma.

“We have been investigating the use of devimistat for treatment in relapsed or refractory Burkitt’s lymphoma. It is a testament to our work and the potential of the compound to have received another orphan drug designation,” said Ariela Noy, medical oncologist for lymphoma and AIDS-associated cancers at Memorial Sloan Kettering Cancer Center (MSK), emeritus chair of the Lymphoma Working Group for the AIDS Malignancy Consortium, and principal investigator on Rafael’s Phase 2 trial in Burkitt’s lymphoma, in the press release. “HIV dramatically increases the risk of Burkitt’s lymphoma. Devimistat brings hope to the many patients with relapsed or refractory Burkitt’s lymphoma, as well as their loved ones.”

European Medicines Agency (EMA) granted Rafael’s CPI-613 (devismat) an orphan drug designation for the treatment of refractory or relapsed Burkin’s lymphoma.