Pharmaceutical Technology Editors

The European Medicines Agency’s (EMA’s) Pharmacovigilance Risk Assessment Committee (PRAC) has concluded that there is no evidence linking the viral vector in Zynteglo to a blood cancer known as acute myeloid leukemia (AML). The findings were released in a July 9, 2021 press release.

Two cases of AML in patients treated with an investigational medicine, bb1111, in a clinical trial for sickle cell disease were reviewed by PRAC. The investigational medicine and Zynteglo both use the same viral vector to deliver a working gene to blood cells and there were initial concerns that the viral vector was implicated in the development of AML.

Through its review, PRAC found that the viral vector was unlikely to be the cause of cancer. After examining all the evidence, PRAC concluded that there were more plausible explanations for AML cases, such as the conditioning treatment patients receive to clear out bone marrow cells and the higher risk of blood cancer in people with sickle cell disease.

As patients receiving Zynteglo also undergo conditioning treatment to clear out bone marrow cells, PRAC has recommended that healthcare professionals explicitly inform patients of an increased risk of blood cancers from medicines used in conditioning treatments. Additionally, PRAC has updated its recommendations for monitoring patients so that any potential signs of blood cancers are checked for on a yearly basis for 15 years. However, it was concluded that the benefits of Zynteglo continue to outweigh the risks.

Zynteglo is a one-time treatment for beta thalassemia, a blood disorder, in patients 12 years and older who require regular blood transfusions. The treatment received conditional marketing authorization in May 2019 and all new evidence will continue to be reviewed by EMA as it becomes available.

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EMA's safety committee, PRAC, has concluded that there is no evidence linking the viral vector in Zynteglo to a blood cancer known as acute myeloid leukemia (AML). 

EMA Concludes There is No Link Between Zynteglo and AML

On July 14, 2021, Eli Lilly and Company announced the acquisition of Protomer Technologies, a private biotech company specializing in a proprietary peptide- and protein-engineering platform, in a deal potentially worth over $1 billion based on the successful achievement of future development and commercial milestones. The acquisition gives Lilly access to Protomer’s engineering platform, which is used to identify and synthesize molecules that can sense glucose or other endogenous modulators of protein activity for the potential development of next-generation protein therapeutics.

Lilly previously led an equity investment in Protomer with the JDRF T1D Fund, a venture philanthropy fund focused on preventing and treating treat type 1 diabetes (T1D) that provided Lilly with 14% ownership of Protomer. With this new transaction, Lilly is acquiring the remainder of Protomer stock beyond its initial investment.

Protomer’s proprietary chemical biology-based platform enables the development of therapeutic peptides and proteins with tunable activity that can be controlled using small molecules. The company has used this approach to advance a portfolio of therapeutic candidates, including glucose-responsive insulins that can sense sugar levels in the blood and automatically activate as needed throughout the day.

"Lilly has long strived to make life better for people living with diabetes and we have a continued determination to provide real solutions, including innovation in insulin therapy. Glucose-sensing insulin is the next frontier and has the potential to revolutionize the treatment and quality of life of people with diabetes by dramatically improving both therapeutic efficacy and safety of insulin therapy," said Ruth Gimeno, vice-president, diabetes research and clinical investigation at Lilly, in a company press release. "Protomer's glucose-sensing insulin program, based on its proprietary molecular engineering of protein sensors (MEPS) platform, is showing significant promise, and Lilly is excited to enhance our diabetes pipeline with the company's innovative technology."

"We are excited to join Lilly, a leader in diabetes therapies, and advance our science with their support to better serve the needs of patients. This transaction validates our team's accomplishments, and we look forward to continuing our important work together with Lilly," said Alborz Mahdavi, CEO and founder of Protomer, in the press release. "We have been supported by JDRF since our inception and working closely with one of the leading organizations in type 1 diabetes research has been invaluable for us. The Protomer team is excited to embark on the next chapter of our work at Lilly as we focus our efforts on advancing glucose-responsive insulins and accelerating the development of these next-generation protein therapeutics."

Eli Lilly and Company has announced its acquisition of biotech firm Protomer Technologies, giving it a protein-engineering platform for next-generation protein therapeutics.

Lilly Acquires Protomer Technologies in Potential $1-Billion Deal

Moderna. announced a supply agreement in a July 12, 2021 press release with the government of Argentina for 20 million doses of Moderna’s COVID-19 vaccine or its updated variant booster vaccine candidate, if authorized. Delivery will begin the first quarter of 2022.

“We appreciate the support from the government of Argentina with this supply agreement for doses of the Moderna COVID-19 vaccine or our variant booster candidates,” said Stéphane Bancel, Chief Executive Officer of Moderna, in a press release. “We are committed to making our vaccine available around the world as we seek to address the pandemic with our COVID-19 vaccine.”

The Moderna COVID-19 vaccine is an mRNA vaccine against COVID-19 encoding for a prefusion stabilized form of the spike protein. Moderna has received emergency authorization for the vaccine for adults from health agencies in more than 50 countries and an Emergency Use Listing from WHO.

Moderna announces supply agreement with the Argentina government for 20 million doses of Moderna’s COVID-19 vaccine.

Moderna to Deliver 20 Million Doses of COVID-19 Vaccine to Argentina

FDA cleared Tevogen Bio’s investigational new drug application for its investigational COVID-19 treatment, TVGN-489, which is an allogeneic COVID-19-specific cytotoxic T lymphocyte (CTL), the company announced on July 12, 2021. 

This clearance allows Tevogen Bio to begin clinical trials, which will evaluate the safety of TVGN-489 in high-risk COVID-19 patients and will also measure viral load and long-term immune response as secondary endpoints. In addition, the results of the patients receiving TVGN-489 will be compared to patients treated with the current standard of care. The previous 

 for TVGN-489 demonstrated strong antiviral activity against SARS-CoV-2.

Programmed to detect targets from the entire viral genome, TVGN-489 is highly purified SARS-CoV-2 specific cytotoxic CD8+ T lymphocytes. The CTLs are anticipated to detect and kill virus-infected cells, which then allows the body to replace them with healthy, uninfected cells.

The IND for Tevogen Bio's TVGN-489, an allogeneic COVID-19-specific cytotoxic T lymphocyte, received rapid clearance from FDA.

FDA Clears Tevogen Bio’s IND Application for CTL COVID-19 Treatment 

Effective July 12, 2021, CDMO Albany Molecular Research, Inc. (AMRI) changed its name to Curia. According to the press release, the new name is to reinforce the company’s positioning as a CDMO—from R&D through to commercial manufacturing in pharmaceutical and biotechnology industries. “Curia” is derived from a Latin word that means purposeful assembly.

The company was founded 30 years ago in Albany, New York. Today, Curia employs more than 3000 people in 21 locations around the world, has 564 active patents, and has more than 20 treatments.

“Our new name reflects the assembled deep expertise of our people, the breadth of our products, services and solutions, and our relentless determination to help customers advance from curiosity to cure,” said Curia Chairman and CEO John Ratliff in the press release. “Our new brand honors our foundation in research and innovation while creating a platform for our ambitions of life-changing science so we can make ever-growing contributions to improving patients’ lives. Over the past three decades we have broadened and deepened our capabilities to become a leading provider of CDMO solutions. Today marks the beginning of the next chapter in our history.”

In addition, Curia has entered into an agreement to acquire Integrity Bio, Inc., a formulation and fill/finish organization in Camarillo, Ca., according to a July 13, 2021 press release. Integrity Bio recently expanded and added a second fill/finish line, which more than doubled its manufacturing capacity. According to the press release, Integrity Bio formulates more than 60 large molecules in a typical year, including proteins, antibodies, lipid nanoparticles, peptides, and vaccines in liquid and lyophilized form.

“The addition of Integrity Bio is a great fit with our growth strategy and our existing expertise, enhancing our biologics drug product formulation development as well as our fill-finish network,” said Curia Chairman and CEO John Ratliff, in the press release. “Integrity Bio also adds West Coast coverage to Curia’s East Coast and European capabilities. Together, we can make an even bigger impact on patients’ lives.”

The transaction is expected to close in the third quarter of 2021.

In a July 14, 2021 press release, Curia also announced that it has entered into an agreement to acquire LakePharma, a biologics drug discovery, clinical research, development, and manufacturing company that has facilities in California, Massachusetts, and Texas.

LakePharma’s range of technology platforms are used in projects from discovery to development to manufacturing, namely cell-line development, bioexpression systems, and viral vector production systems. The company has assisted in the development of more than 200 therapeutic or diagnostic products and served more than 1500 customers, including some of the top global biopharmaceutical companies.

Following the completed transaction and merge of Curia and LakePharma, the company will provide expertise in large and small molecules from drug discovery through drug substance manufacturing, sterile injectable formulation, and fill/finish production.

AMRI reinforces positioning as a CDMO with name change and acquires the formulation and fill/finish company, Integrity Bio, and biologics company, LakePharma.

AMRI Changes Name to Curia and Acquires Integrity Bio and LakePharma

SPI Pharma introduced an excipient platform for flash orally disintegrating tablets (ODTs), which enables disintegration in less than ten seconds. ODTs are good for anyone with difficulty swallowing.

This platform, UltraBurst, is designed for direct compression. According to the July 12, 2021 press release, it can deliver the fastest disintegration of preformulated products on the market and maintains hardness, friability, API loading, and assorted tablet weights for anything from prescription drugs to nutraceuticals.

"Across a wide range of different API loadings and tablet sizes, we can demonstrate significant reduction in disintegration time against the current market alternatives,” said John McInerney, General Manager of the Excipients and Drug Delivery Systems Business Unit. “We see reductions in disintegration times between 20–40% without loss of tablet hardness or increased friability.”

UltraBurst has a high disintegration speed, which is for all market segments, from prescriptions to nutraceuticals.