Jill Murphy

ten23 health, a contract development and manufacturing organization (CDMO), announced a capacity expansion for the manufacture of sterile drug product at its Visp facility, according to a press release.

Due to customer demand for sterile fill and finish services, the expansion will head to VIVA 1, Visp Valais, Switzerland, at a sterile manufacturing facility. Following successful completion of the GMP inspection by Swissmedic and an obtained GMP license, the new storage areas are set to become operational in September 2023. Additionally, the output of ten23’s existing sterile manufacturing facility in Visp will be gradually increased by a further 50% capacity by hiring more staff to operate an additional shift.

According to the press release, further expansions of the pharmaceutical development and manufacturing services of ten23 are happening at the BASE and VIVA facilities to continue to support complex sterile product development, testing, and manufacturing. Two additional filling lines for sterile commercial supplies of ready-to-use syringes, cartridges, and vials and liquid or freeze-dried clinical and commercial vial supplies will all be a part of VIVA 2. This project is signed for manufacturing in 2024 and 2025.

“We are thrilled to offer our customers additional sterile manufacturing capacity for sophisticated and highly precise Drug Product-presentations such as syringes and cartridges, increased capacity until the two new production lines becoming fully operational.” said Professor Hanns-Christian Mahler, PhD, CEO of ten23 health, in a press release. “With recent demand increases for clinical as well as commercial drug launches, our customers are keen to take advantage of increased capacity from the existing VIVA 1® facility, before the new plant VIVA 2® will be utilized for larger scales.”

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Further expansions of the pharmaceutical development and manufacturing services of ten23 are happening at the BASE and VIVA facilities to continue to support complex sterile product development, testing, and manufacturing. 

ten23 Expands Sterile Manufacturing Capacity at VIVA 1 Facility 

On Aug. 28, 2023, Embark Laboratories announced it has been acquired by Novo Nordisk, including its lead metabolic program, in addition to entering a three-year research and development collaboration to discover and develop novel pharmaceuticals to treat obesity and related comorbidities, according to the press release.

Under the acquisition agreement for Embark Biotech, Novo Nordisk receives the full rights to develop and commercialize the lead program. Additionally, the research and development collaboration with Embark Laboratories provides Novo Nordisk with the option to acquire selected assets based on the Embark Biotech findings across several indications, including obesity and type 2 diabetes, according to the release.

“Novo Nordisk has been engaged in obesity research for 25 years, and we continuously search for new ways to address this serious chronic disease. We are excited about the opportunity to advance Embark Biotech’s lead program and look forward to co-creating novel treatments for cardiometabolic diseases with Embark Laboratories to complement our in-house R&D,” said Brian Finan, vice president of Obesity Research at Novo Nordisk, in a press release.

“There is a clear strategic fit between the novel biology we have discovered and Novo Nordisk’s expertise and focus on developing new drugs in the cardiometabolic space. We are thrilled to pass on the baton for our lead metabolic program to Novo Nordisk. This deal and Embark’s success are direct outcomes of the unique and innovative ecosystem in Denmark that has been cultivated by the BioInnovation Institute and the University of Copenhagen and through initiatives from the Novo Nordisk Foundation and the Innovation Fund Denmark,” said Zach Gerhart-Hines, chief technology officer at Embark Laboratories in the release.

Novo Nordisk receives the full rights to develop and commercialize the lead program. 

Novo Nordisk Acquires Embark Biotech

Zevra Therapeutics and Acer Therapeutics announced on Aug. 31, 2023 that they have entered a definitive agreement where Zevra would acquire Acer in a merger transaction having a total potential value for Acer stockholders of up to $91 million, according to a press release.

Additionally, additional cash payments are possible in relation to the Contingent Value Rights with respect to milestones involving Acer’s early-stage program ACER-2820 (emetine). Zevra also purchased Acer’s secured debt at a discount from Nantahala Capital through a series of transactions in capital efficient structure, according to the release. Both companies have a focus to developing and commercializing treatments for rare diseases with a strong focus on patients and supporting communities with little to no existing therapeutic options.

“We believe that Acer’s portfolio of rare disease programs, including the recent US commercial approval of OLPRUVA for urea cycle disorders UCDs, is a perfect strategic fit for Zevra and creates significant opportunity for us to positively impact patient lives while creating shareholder value,” said Joshua Schafer, chief commercialization officer and executive vice president of Business Development of Zevra Therapeutics, in a press release. “We are excited about Acer’s clinical programs and are confident in the potential of OLPRUVA to bring UCD patients a more convenient and cost-effective treatment option over current therapies. Acer would bring unique rare disease operations and capabilities that would serve as a foundation to support the commercialization of Zevra’s pipeline as it advances.”

“This merger would support Zevra's vision of becoming a leading rare disease company bringing life-changing therapies to patients with a significant unmet need,” said Christal Mickle, Zevra’s interim chief executive officer and chief development officer in the press release. “The commercial launch of OLPRUVA in the US requires a small, highly-focused commercial team, which is complementary to what we intend to build for arimoclomol, our product candidate for the treatment of Niemann-Pick disease Type C (NPC). We believe there is potential to realize significant synergies across our commercial organizations as both UCDs and NPC are metabolic related conditions and there is overlap among those physicians that treat both disorders.”

Both companies have a focus to developing and commercializing treatments for rare diseases with a strong focus on patients and supporting communities with little to no existing therapeutic options.

Zevra Therapeutics to Acquire Acer Therapeutics 

In this episode of the Drug Solutions podcast, Jill Murphy, Editor, speaks with Dr. Graziella Piras, senior director of Strategic Marketing at 908 Devices, about drug development, specifically related to monitoring bioprocess parameters and other important technologies important to moving the process forward.

Graziella has over 15 years of experience in developing products, applications, and solutions for upstream bioprocessing. Before joining 908 Devices, she held various positions in marketing and R&D at Thermo Fisher Scientific, leading projects to support the Cell Culture and Cell Therapy business.

Graziella obtained her Ph.D. in Biochemistry & Molecular Biology in Belgium. She did her postdoc studying the role of epigenetic regulation on cancer and development at the National Cancer Institutes in Maryland.

 presents the Drug Solutions podcast, where the editors will chat with industry experts from across the pharmaceutical and biopharmaceutical supply chain. Join us as experts share insights into your biggest questions—from the technologies, to strategies, to regulations related to the development and manufacture of drug products.

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Drug Solutions Podcast: Breaking Through the Barriers of Drug Development

keyboard closeup | Image credit: © Karramba Production - stock.adobe.com

Clinical trials are essential for establishing the efficacy of new drugs, observing patients’ reactions to treatments, and representing populations of people that may not always be discussed in everyday conversation. Although there are many diseases and disorders that are waiting for treatments, there are many promising trials in the pipeline that may bring major news for these conditions sooner than we think, even if there are some minor setbacks.

In the gene therapy space, Sarepta Therapeutics is looking to get an approval for Elevidys (delandistrogene moxeparvovec-rokl) through the EMBARK trial. This drug may be a breakthrough for patients with Duchenne muscular dystrophy who have been waiting for a treatment for many years. After receiving an accelerated approval for the drug for patients between 4 and 5 years of age, it has been the company’s mission to expand from the narrow population and address the doubts surrounding Elevidys. Although the trial is ongoing with 126 patients in a controlled environment, the results will either lead to regulators growing the age restrictions or having to remove the drug from the market (1).

We have seen very quick removals from the drug market before. An example of this is FDA’s decision to pause new patients on Merck KGaA’s multiple sclerosis drug, evobrutinib. They were able to advance in the Bruton’s tyrosine kinase (BTK) inhibitors class to continue to slow the progression of nerve disease multiple sclerosis, but as of April 2023, it was removed at FDA’s decision due to “laboratory results which suggested drug-induced liver injury during Phase III studies.” Although a Phase III trial for evobrutinib is set to continue as planned, Merck has had to alter their study that involves patients who have been taking the drug for fewer than 70 days (2).

The growing number of clinical trials is very exciting, and I am looking forward to continuing to hear about the latest treatments being tested in the mental health space, cancer space, and any other therapeutic area that takes up the pharmaceutical industry. Even with the setbacks we hear from some of the trials, I am positive that we will get the best results, and help for so many patient populations will finally be achieved.

BioPharma Dive. 10 Clinical Trials to Watch in the Second Half of 2023. BioPharmaDive.com, June 28, 2023.

Reuters. US Pauses New Patients on Merck MS Drug in Blow to Shares. Reuters.com, April 12, 2023

When referring to this article, please cite it as Murphy, J. Clinical Trials and their Impact on the Pharma Industry. 

There are many promising trials in the pipeline that may bring big news for major therapeutic areas.

Clinical Trials and their Impact on the Pharma Industry

The healthcare space is at its peak in technology and other digital transformations: machine learning, artificial intelligence (AI), and other aspects are showing promise for helping patients and improving efficiency in the labs. However, there have been significant data integrity lapses that have caused consequences in the pipeline, such as manufacturing plants closing, drug shortages, and US Food and Drug Administration (FDA) approvals not going through (1).

Shailesh Vengurlekar, senior vice president of Quality & Regulatory Affairs, LGM Pharma, echoes this challenge in today’s world of drug substance testing. “Some of the biggest challenges facing drug substance testing include supply chain deficiencies, and data integrity,” he says. “Post-pandemic supply chains are still recovering, and the pharmaceutical industry is no exception. Data integrity continues to be a major topic of concern as well and is one of the factors driving the industry’s embrace of digital transformation.”

Another major challenge is the lack of significance when large data sets are being interpreted over a sustained period. Quality-by-design (QBD) approaches are a way to ensure proper data interpretation in this type of scenario to help monitor and analyze the trends of biologics and other drugs (2).

Regulatory action was taken to try and improve this hurdle. FDA released a data integrity guidance in December 2018 due to the increased data integrity violations during current good manufacturing practice (CGMP) inspections in recent years. This included more regulatory actions, like warning letters, import alerts, and consent decrees to attempt to ensure the safety, efficacy, and quality of drugs (3).

Interpreting data and understanding the various components of biologic drug substance testing is an important skillset to know as a lab personnel. According to Khanh Ngo Courtney, senior director of Biologics at Element, the characterization and routine testing of biological therapeutics, with cell and gene therapies being a specific focus, require advanced analytical techniques and an understanding of the biology itself. Mahesh Bhalgat, chief operating officer of Syngene International, said on the fact that although many labs and lab personnel are technically sound, there is still a lack of understanding and decision-making on the use of analysis models for biological product characterization and for the mechanism of action (MoA) studies (2).

A way to solve this is by making sure the lab can provide as many assurances of product safety, strength, purity, quality, as possible, all of which depend on the data being discussed at the lab itself.Any data-driven decisions must be reproducible and based upon data that can support those outcomes (1).

Further, following the ALCOA method is another way that personnel can consider making sure they are on track with the right data:

Attributable: This is specific to staff members via audit trails and e-signatures and includes no password sharing and compliance to Part 11.

Legible: Make sure your data is usable and readable during internal audits.

Contemporaneous: Keep records of the tasks at hand during the performance of a task, not before or later.

Original: Store data in its original format, not manipulated, stored elsewhere, or shared with others.

Accurate: Use a system that minimizes errors, raw data and analytical results presented in a proper format (1).

This method also highlights the elements of making sure data are complete, consistent, enduring, and available, which can help ensure proper interpretation and lessening the hurdles that have come with data integrity.

Even with the challenge of data integrity at hand, there is hope for drug substance testing to continue adapting to the technological transformations that are growing at a fast pace. Additionally, as more process knowledge and analytical method data become available, optimization of process and analytical methods will continue to be required as part of continuous improvement concepts.

Vengurlekar feels that in the next 5–10 years, drug substance testing will see increased efforts to standardize testing protocols and ensure that the quality of results across laboratories improve, leading to more consistent and reliable testing outcomes. As for Courtney, many current methods all have their own difficulties, but finding the right analytical solution for the intended purpose is an unmet need for many quality-defining methods for advanced therapeutic molecules in this current day and age.

Chapman, J. Data Integrity 101: Why is it important? Redica Systems. 22 Oct 2020.

Mirasol, F. Biologics Testing Highlights Need for Analytical Skills. Pharm. Technol. 2022, 46 (1) 38–40.

UW-Madison School of Pharmacy. The Trouble with Biologics: Analyzing Large Molecules and Data Integrity. University of Wisconsin-Madison (accessed 11 August 2023).

Volume 35, No.9
September 2023
Pages 23-28, 29

When referring to this article, please cite it as Murphy, J. 

Data Integrity’s Impact in Drug Substance Testing

Interpreting data and understanding the various components of biologic 
drug substance testing is an important skillset to know as a lab personnel.
