Complex biotherapeutics present unique analytical challenges for developers.

Complex Biomolecules Require Analytical Evolution

FDA Approves Additional J&J Vaccine Batch; AstraZeneca Launches Variant Trial

Full Vaccine Approval is Key to Overcoming Hesitancy

Public-private consortium is tasked with advancing domestic pharmaceutical production.

HHS Strategizes for Domestic Drug Supply

New biotherapeutics both drive and benefit from innovative development technologies.

Innovation Demands and Spurs Invention

Meeting Development and Manufacturing Demands for Emerging Therapies

Technology Forum

On July 29, 2021, Emergent BioSolutions announced that FDA is allowing the company’s Bayview manufacturing facility in Baltimore, Md., to resume production of Johnson & Johnson’s (J&J) COVID-19 vaccine bulk drug substance. FDA’s decision comes after doing extensive reviews and is based on the diligent work and close coordination Emergent has conducted with J&J and the agency to execute a quality enhancement plan.

Since production was paused, Emergent has been working closely with FDA and J&J to address quality concerns and has developed and executed an action plan as well as committed extensive resources to bring operations up to FDA standards. The company expects to continue working with the agency throughout the manufacturing process to help ensure the stability of the J&J COVID-19 vaccine supply chain.

“We are proud to be resuming production of bulk COVID-19 vaccine batches following additional reviews and collaboration with FDA and our manufacturing partners,” said Robert Kramer, Emergent’s CEO, in a company press release. “We are in the unique business of producing life-saving medications for catastrophes that we hope never occur [such as] anthrax attacks, opioid overdoses, and COVID-19.”

“The American people should have high expectations of the partners its government chooses to help prepare them for disaster, and we have even higher expectations of ourselves,” Kramer added. “We have fallen short of those lofty ambitions over the past few months, but resumption of manufacturing is a key milestone, and we are grateful for the opportunity to help bring this global pandemic to an end. We’d like to thank our government partners as well as Johnson & Johnson for their support.”

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Emergent BioSolutions is being allowed to restart manufacturing operations for the J&J COVID-19 vaccine at its Bayview, Baltimore, Md., facility.

FDA Gives Green Light to Emergent BioSolutions to Resume COVID-19 Vaccine Manufacturing

Entries are open for the 18th Annual CPhI Pharma Awards, which are slated to take place on November 9, 2021, in Milan, Italy, at the Stella Polare Convention Centre. Awards in fifteen categories will be given out to innovators and thought leaders who are exploring uncharted territory in the pharma/biopharma industries.

Anyone involved in design, development, manufacture, supply, and distribution of large- and small-molecule drug products may apply. Winners will be selected by a panel of expert judges. The deadline for applications is August 6th, 2021.

This event is part of CPhI Worldwide, a pharma exhibition taking place November 9–11, 2021. Last year, the convention was cancelled as a result of the COVID-19 pandemic, with the entirely virtual CPhI: Festival of Pharma held in its place. This year’s event will be a hybrid of in-person and virtual activities.

“We host our awards because it’s important to recognize the innovative and inspirational work that is produced across the pharmaceutical industry” said Tara Dougal, head of content and insights, Informa Markets, in a July 19, 2021 company press release. “After a challenging year, the industry has been more committed than ever to advancing health and improving patient outcomes. It’s been a year in which we have seen the very best of pharma, with a record pace of innovation, discovery, and commercialization.”

The full awards list and submission requests can be found at 

Entries are now open for the 18th Annual CPhI Pharma Awards in Milan.

18th Annual CPhI Pharma Awards to Take Place in Milan, Italy

On July 29, 2021, VICO Therapeutics, a Netherlands-based biotech company focused on the development of RNA-modulating therapies for rare neurological disorders, announced that FDA had granted orphan drug designation (ODD) for VO659, an investigational antisense oligonucleotide therapy (AOT), in the treatment of Huntington’s disease (HD).

HD is a rare degenerative brain disease that results in motor disorders, impaired cognition, dementia, and various psychiatric manifestations. It is caused by a mutation to the CAG-trinucleotide repeat in the coding region of exon 1 of the HTT gene, resulting in a mutant huntingtin protein with an elongated polyglutamine (polyQ) stretch at its N-terminus. This grants a toxic gain-of-function to mutant protein forms, which results in widespread neuronal death.

VO659 is designed to suppress mutant proteins with the intent of slowing or halting the progression of disease. A month prior to this announcement, the drug had also received orphan drug designation for the treatment of spinocerebellar ataxia, a group of hereditary polyQ disorders that result in similar neuron degeneration.

ODD is granted to drugs intended for the safe and effective treatment, diagnosis, or treatment of rare (fewer than 200,000 cases) diseases or disorders. This designation grants various benefits, including seven years of market exclusivity following regulatory approval, exemption from FDA application fees for Huntington’s disease, and tax credits for qualified clinical trials.

“We are delighted that [FDA] has granted this orphan drug designation following the ODD for SCA one month ago,” said Rupert Sandbrink, chief medical officer at VICO, in a company press release. “This is affirmation of the potential of our [antisense oligonucleotide platform] approach.”

VICO Therapeutics’ drug candidate, VO659, for treating Huntington’s Disease was granted orphan drug designation by FDA. 

FDA Grants Orphan Drug Designation to VICO Therapeutics for Huntington’s Disease Drug 

Jill Wechsler is Pharmaceutical Technology's Washington Editor, jillwechsler7@gmail.com.

Despite the pressures and added regulatory challenges imposed by the COVID-19 pandemic, and the uproar over its surprise approval of Biogen’s new Alzheimer’s therapy Aduhelm, FDA is on track this year to authorize a notable number of new molecular entities (NMEs) and important biotech therapies. In the first half of 2021, the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) gave the nod to 29 novel therapies, slightly ahead of last year’s pace that saw the approval of a near-record 53 new drugs.

added up to 27 NMEs and biologics as of June 30, 2021, with several more additions in July. And 

 a new pneumococcal vaccine and new treatments for multiple myeloma and for lymphoma.

Almost half of the new drugs are treatments for cancer and related conditions, many benefitting from priority review and accelerated approval programs. That includes Jazz Pharmaceuticals’ new blood cancer therapy, a treatment for non-small cell lung cancer from Amgen, and Bristol Myers Squibb’s new CART-T therapy for multiple myeloma plus a cell therapy for large B-cell lymphoma.

Important non-cancer approvals also include the first new treatment for vaginal yeast infections in two decades and the first therapy for chronic weight management authorized since 2014, arising from approval of an added indication for Novo Nordisk’s type 2 diabetes injectable Wegovy (semaglutide) for obese or overweight adults. FDA also okayed new therapies for Duchenne muscular dystrophy, lupus nephritis, chronic heart failure, and notably, an extended release injectable to manage HIV, replacing daily pills with monthly injections.

There were advances in the over-the-counter drug world, too. In June, FDA approved the first nonprescription nasal antihistamine to expand treatment options for patients age six and older; younger children still must obtain Bayer Healthcare’s Astepro (0.15% azelastine hydrochloride nasal spray) on a prescription basis.

Important news on the approvals front continued in July, as 

FDA authorized its first interchangeable biosimilar

—Mylan’s Semglee once-daily insulin therapy, biosimilar to Sanofi’s Lantus.

FDA also approved an important treatment for high-risk, early-stage, triple-negative breast cancer, giving the nod to Merck’s Keytruda (pembrolizumab) to be used in combination with chemotherapy for this serious condition.

On the downside, FDA halted use of a treatment approved in February for relapsed or refractory multiple melanoma, Pepaxto, from the Swedish firm Oncopeptides, based on evidence from ongoing clinical studies of higher death rates among patients treated with the new drug versus an earlier therapy. Pepaxto gained accelerated approval, but now 

FDA has alerted patients and health professionals 

to the potential risk and has suspended enrollment in postapproval studies pending further examination of the safety signal.

CDER officials attribute their ability to maintain a fairly normal advise-and-consent process for innovative drugs during the past 18 months on the hard work of its dedicated staff, as well as efficiencies gained from the recent reorganization of its Office of New Drugs (OND). CDER Director Patrizia Cavazzoni noted increases in staff hiring and retention at the May 2021 annual meeting of the Food and Drug Law Institute (FDLI), attributing this surprising development to new remote work flexibilities due to COVID-19 restrictions. She also cited the success of large platform trials and clinical networks, as well as increased sponsor use of decentralized trials. The structural overhaul of OND has resulted in smaller, more flexible review offices with clearer areas of expertise and greater alignment of interrelated disease areas, 

Cavazzoni explained in recent testimony to the House Energy & Commerce Health subcommittee

, which held a hearing to explore the lack of new therapies for neurogenerative diseases.

In the first half of 2021, the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research approved 29 novel therapies.

New Drug Approvals on Track at FDA

Thermo Fisher Scientific has introduced Ion Torrent Oncomine immune repertoire assays, a new suite of next-generation sequencing (NGS) assays that are designed to detect potentially malignant clones of T cells and B cells, key players in immune response. The pan-clonality assays use proprietary Ion AmpliSeq technology to target multiple parts of B- and T-cell immune receptors. This is done using a single reaction with ultra-high sensitivity, which increases the probability of detecting malignant clones and decreasing the time to results, Thermo Fisher announced in a July 19, 2021 press release.

Detection of distinct receptor DNA sequences can inform diagnostic, prognostic, and therapy development studies, which can illuminate the progression of lymphoid cancers, including leukemias, lymphomas, and diseases such as multiple myeloma. Using NGS technology, detection sensitivity can be increased—compared to traditional testing methods, such as flow cytometry or quantitative polymerase chain reaction.

One of the challenges of current NGS-based immune repertoire analysis has been the need to run multiple secondary tests because of the high test failure rates of a typical single NGS assay, the company explained in the press release. The new Oncomine pan-clonality assays overcome this challenge by uniquely targeting multiple parts of the receptor to reduce the need for secondary tests and increase the probability of a single assay detecting a potentially malignant clone.

"Genomic testing can make a significant impact on researching and understanding these [blood] cancers—but we need to bring more testing to in-house laboratories where results can be made available quickly to healthcare professionals," said Garret Hampton, president of clinical next-generation sequencing at Thermo Fisher Scientific, in the press release. "The new Oncomine immune repertoire assays unlock the power of NGS to help researchers rapidly obtain a tremendous amount of insight about a lymphoid cancer sample, including its prevalence and an understanding of how it is evolving."

"With the ability to sequence multiple targets in a single reaction, our team can now get much more information from one assay when compared to single-target NGS tests," said John DeCoteau, medical director, University of Saskatchewan, Advanced Diagnostic Research Laboratory, in the press release. "Using the Oncomine BCR Pan-Clonality Assay, we were able to achieve a positive clonality detection rate of 93 percent in a cohort of multiple myeloma samples, without the need for secondary testing. That provides a remarkable improvement for laboratory efficiency and allows us to get results faster."

Thermo Fisher Scientific has added a new suite of immune repertoire assays that offers high detection rates of malignant clones, allowing researchers to better assess blood cancers.

Thermo Fisher Expands Hematology Oncology NGS Portfolio

The European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) is assessing data to evaluate whether Kineret (anakinra) can be used as a treatment for adult COVID-19 patients who are at an increased risk of severe respiratory failure.

Kineret is an immunosuppressant that has been authorized for use in the European Union for a number of inflammatory conditions since March 2002. It is believed that the API, anakinra, in Sobi’s Kineret could help reduce the inflammation and tissue damage associated with COVID-19. Anakinra works by blocking the activity of interleukin 1, which is a chemical messenger involved in immune processes that lead to inflammation.

Data from two ongoing clinical studies that are investigating the safety and efficacy of Kineret in adult patients hospitalized with COVID-19 are being evaluated by the CHMP. The committee’s opinion on whether or not Kineret’s indication can be extended will be forwarded to the European Commission, which will issue the final decision on marketing authorization for the European Union.

The outcome of the evaluation, which is expected to be released by October 2021, will be published by EMA.

Kineret (anakinra) is under evaluation by EMA's CHMP as a treatment for adult COVID-19 patients who are at an increased risk of severe respiratory failure.

CHMP to Assess Extended Indication of Kineret for COVID-19

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has taken the decision to start a rolling review of Vidprevtyn—Sanofi Pasteur’s COVID-19 vaccine—based on preliminary non-clinical and early clinical results. According to a July 20, 2021 press release, the early trial data suggest that Sanofi’s vaccine triggers the production of antibodies that target SARS-CoV-2 and may help protect against the disease.

Rolling reviews are employed by EMA to accelerate assessment of promising drug candidates during public health emergencies. All data on Vidprevtyn will be evaluated by EMA as soon as they become available, so that a decision on whether the vaccine benefits outweigh the risks can be made.

The rolling review process will be performed until sufficient evidence is acquired for a formal marketing authorization application. EMA has specified that further communication will be published once a formal application for marketing authorization has been submitted.

EMA's CHMP has taken the decision to start a rolling review of Vidprevtyn—Sanofi Pasteur’s COVID-19 vaccine.

EMA Starts Rolling Review of Sanofi’s COVID-19 Vaccine

ERS Genomics and Japan SLC, a biotechnology company that provides animal models to the research community, have signed a non-exclusive license agreement, allowing Japan SLC access to ERS Genomics’ clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 patent portfolio. According to a July 27, 2021 press release, through this access, Japan SLC will be able to expand its offering.

“We are proud to add Japan SLC to our growing list of partners in Japan applying CRISPR/Cas9 technology to its service offerings,” said Eric Rhodes, CEO of ERS Genomics, in the press release. “Japan SLC’s animal production and supply are of high quality, and we are very pleased that it has chosen to license CRISPR/Cas9 technology through ERS Genomics.”

“Our production and supply of laboratory animals will benefit greatly through the use of the revolutionary CRISPR/Cas9 technology,” added Hirotaka Takagi, president, Japan SLC, in the press release. “We are very excited to sign this licensing agreement with ERS Genomics which will enable us to produce CRISPR-edited mouse and rat models.”

The financial details of the non-exclusive agreement were not disclosed.

ERS Genomics and Japan SLC have signed a non-exclusive license agreement, allowing Japan SLC access to ERS Genomics’ CRISPR/Cas9 patent portfolio.

ERS Genomics Adds Japan SLC to List of Partners

On July 27, 2021, Byondis, a Netherlands-based clinical-stage biopharmaceutical company, and Glycotope, a Germany-based biotechnology company specializing in antibodies that target tumor-specific-carrying carbohydrate structures, have entered into a platform access agreement to discover and develop antibodies that target specific tumor-associated protein/carbohydrate combined glyco-epitopes (GlycoTargets).

Under the agreement, Byondis has gained exclusive rights to evaluate and develop antibodies against selected novel GlycoTargets and has the option to in-license these antibodies for development as antibody-drug conjugates (ADCs). Financial terms were not disclosed.

Glycosylation is strongly altered in cancer cells, which reflects the drastic changes in tumor metabolism. This alteration, in turn, results in new, highly tumor-specific GlycoTargets. Specific antibodies against these GlycoTargets might be highly cancer specific and have the potential to target a broad range of oncology indications, the companies stated in a press release.

Formulating the antibodies into ADCs can improve their cell-killing capability. But while earlier generation ADCs improved targeting and cell-killing, they were unstable in the bloodstream, which led to premature release of the cytotoxic payload that impacted healthy tissue and narrowed the therapeutic window. Byondis’s next generation ADCs are designed to be highly stable in circulation and carry an intricate, inactivated, and potent cytotoxic drug that rapidly self-destructs if prematurely released, thus limiting damage to healthy tissue and improving the therapeutic window, according to a Byondis press release.

“Selective tumor targeting is key to maximiz[ing] the potential of our linker-drug platforms to generate effective ADCs with low systemic toxicity. The antibodies generated by Glycotope are designed to deliver very high tumor selectivity. We are excited to join forces and align our highly complementary technologies to provide effective and safe ADCs for the benefit of cancer patients,” said Byondis Chief Scientific Officer Wim Dokter in the press release.

“This agreement with Byondis highlights the potential of our GlycoTarget discovery and technology platform,” said Henner Kollenberg, managing director of Glycotope, in the press release. “GlycoTargets are a unique class of highly tumor-specific antigens that offer reduced on-target/off-tumor toxicities. We believe that combining antibodies against these GlycoTargets with Byondis’ linker-drug platform has great potential to produce novel treatments for people suffering from diseases for which no adequate medical treatment option has been found so far.”

Byondis and Glycotope have formed an agreement to discover and develop antibodies that target specific glyco-epitopes.

Byondis and Glycotope Partner on Discovery and Development of Glyco-Epitope-Targeting Antibodies

Summa Equity has acquired a majority stake in Axion BioSystems, a US life science tools business specializing in microelectrode arrays (MEA) and impedance technology for single-cell analysis. Axion provides an integrated technology platform for cell activity monitoring, typically used by academia or biopharma customers for non-invasive, real-time study of cells. Axion’s technology was of particular interest to Summa with regards to cell and gene therapy (CGT), which could be instrumental in curing various acquired and genetic diseases.

“Summa has been focused on solutions in the CGT market for a long period of time, due to its alignment with our strategy and its significant potential for growth,” said Tommi Unkuri, a partner at Summa Equity, in a press release from July 16, 2021. “Axion BioSystems impressed us with its … scalable technology, which gives both academics and biopharma organizations the ability to go much deeper in their understanding of cell behavior, enabling faster, more accurate and more cost-efficient drug discovery, development and quality control.”

“We have been highly impressed with Summa’s understanding of the life sciences market, and belief in the potential of our technology to accelerate innovation in many areas of pharmaceuticals and healthcare,” said Tom O’Brien, CEO and president, Axion BioSystems in the press release. “We believe that they will bring tremendous value in helping us to increase our commercial capacity, reach, and market awareness to scale up significantly and have an even greater impact in the years to come.”

Summa Equity has acquired a majority stake in science tools business Axion BioSystems with the intent of further entering the CGT market. 

Featured Content

Complex Biomolecules Require Analytical Evolution

Full Vaccine Approval is Key to Overcoming Hesitancy

HHS Strategizes for Domestic Drug Supply

Innovation Demands and Spurs Invention

Technology Forum