GEN Roundtable Panel Discusses Technologies and Strategies for Product Development

In January, San Francisco was the annual venue for the J.P. Morgan Healthcare Conference and the Biotech Showcase Conference. These two meetings together provided an opportunity to get a good sense of the current status and potential future state of the global biotech industry.

While these meetings in the City by the Bay progressed, GEN’s Editor-in-Chief, John Sterling, sat down with officials from seven companies engaged in bioproduct R&D or biomanufacturing for a roundtable discussion. (Originally, 10 companies were invited, but representatives from three of those firms were subsequently unable to attend).

The plan was to learn more about each company’s product or technology focus along with the strategy each was pursuing to compete in the biotech marketplace. The companies were chosen based on good science, product need, sound management, and the specific area of product/technology concentration. As part of the discussion, a video recording of each of the interviewees was also made. The participants included:

Jin Seok Hur, Ph.D., Technology Director, Novasep
Roslyn Brandon, D.V.M., Ph.D., President and CEO, Immunexpress
Ramesh Hariharan, Ph.D., Co-founder and Chief Technology Officer, Strand Life Sciences
Rick Gonzalez, President and CEO, Navidea Biopharmaceuticals
Andrew Hirsch, President and CEO, Bind Therapeutics
Chris Parker, Executive Vice President and Chief Business Officer, Cellular Dynamics International
Robert Clarke, Ph.D., President and CEO, Pulmatrix

What follows is a snapshot of each company and news about recent developments at the firms. Also included is a video clip from each interview. The goal is to offer a succinct and to-the-point description about each company and its planned path for success.


Novasep

Novasep works in seven different markets, pharmaceuticals and biopharmaceuticals among them. The firm is a CMO that also provides process engineering, equipment supply, and related services.

The company specializes in continuous bioprocessing and reports that its BiosSCR Lab next-generation continuous biochromatography equipment can purify a few grams of proteins per day. Novasep officials say they want to enable users to develop cost effective, high yield production processes while maintaining the same quality attributes of the protein. This past November the company announced that it had equipped the UK National Biologics Manufacturing Centre with BioSC Lab.

More recently Novasep signed a CDMO partnership with Advanced Biotherapeutics Consulting, a service provider that leverages the expertise of the Nantes Gene Therapy Institute led by Philippe Moullier, M.D., Ph.D. According to officials at both firms, the partnership will allow the companies to provide their respective customers with the full range of services relating to Adeno-Associated Virus (AAV) viral vector production. Novasep’s ambition is to build and offer an AAV platform to the gene therapy market.

And in late January Novasep said it is building a new synthesis laboratory and a kilo lab for its U.S. facility in Boothwyn, PA. This extension, which will permit Novasep to offer both chemistry and purification services, is designed to give Novasep the capability to produce the initial kilogram scale batches of synthetic molecules that are needed for biological testing and preclinical trials.


 


 

 


Immunexpress

Immunexpress, a molecular diagnostics company, developed a product pipeline to address the unmet clinical needs of sepsis patients, as well as the professional needs of clinicians, payers, and healthcare systems. The CDC notes that sepsis kills more than 258,000 Americans each year and leaves thousands of survivors with life-changing aftereffects. There are over 1 million cases of sepsis each year, and it is the ninth leading cause of disease-related deaths in the United States.

According to Immunexpress, its SeptiCyte® technology, unlike the traditional approach to sepsis diagnosis, quantifies specific molecular biomarkers from a patient’s immune system. These biomarkers, which capture signals in response to infection, may hold the key to the early detection of sepsis. They may also serve to guide the use and timing of drugs and other therapies.

Immunexpress says it has validated hundreds of SeptiCyte® nucleic acid and protein expression biomarkers that, in different combinations, answer specific clinical questions about sepsis. Patented biomarkers, algorithms, and analysis methods are used to interpret information about the sepsis host response. Immunexpress’ products are based on immune response technologies originally generated in novel large-animal models of sepsis.

Scientists recently presented data on the utility of SeptiCyte™ LAB in both adult and pediatric populations to aid in the clinical assessment of sepsis in critical care patients. One study found that, when tested in the Genotypes and Phenotypes of Pediatric SIRS and Sepsis (GAPPSS) trial cohorts, SeptiCyte LAB produced strong performance, comparable to that observed in adult populations, according to the researchers.

Another presentation revealed data from the VENUS A study, the prospective pivotal study which serves as the basis for the FDA filing for the SeptiCyte LAB. The product reportedly was able to effectively distinguish between sepsis and infection-negative systemic inflammation, a result that was not linked to measures of patient severity. Company officials point out that this prospective study further validates the results from five patient cohorts published in PLOS Medicine this past December.


 


 


Strand Life Sciences

Strand Life Sciences uses genomic profiling based on next-generation sequencing (NGS) technology to improve the care of cancer. The company’s StrandAdvantage pancancer panels were developed to provide medical oncologists with a deep understanding of genomic changes in solid tumors, and to do so in days.

Strand Life Sciences’ knowledge base of genomic variants, which is linked to FDA-approved targeted cancer therapies and drugs in open clinical trials, is designed to offer clinically actionable treatment options. The information is intended to be used by physicians in developing a targeted treatment plan. The ultimate goal is to enable clinicians to develop personalized treatment regimens for cancer patients.

The Strand NGS bioinformatics platform was created as a solution for NGS analysis teams. Designed by biologists, the product features an intuitive user interface, structured workflows, analysis- and experiment-specific workflows, benchmarked alignment algorithms, and a quality control manager. The Cleveland Clinic, Pfizer, Cincinnati Children’s, and Massachusetts General Hospital are among its clients.

In January, Venaxis, an in vitro diagnostic company focused on developing and commercializing its multibiomarker diagnostic blood test, the APPY1™, merged with Strand. APPY1 was designed to aid in the identification of patients at low risk for acute appendicitis.


 


 


Navidea Biopharmaceuticals

Navidea Biopharmaceuticals has established a niche in precision medicine. The company focuses on the development and commercialization of diagnostic agents and therapeutics for cancer, autoimmune diseases, dementia, and movement disorders.

One of Navidea’s products is Lymphoseek® (technetium Tc 99m tilmanocept) injection, a radiopharmaceutical agent that was approved by the FDA for lymphatic mapping or sentinel lymph node biopsy for patients with several different types of solid tumor cancers. Lymphoseek is also indicated for lymphatic mapping to locate lymph nodes in patients with solid tumors (when this procedure is a component of surgical management) and for sentinel lymph node biopsy (SLNB) in patients with clinically node negative squamous cell carcinoma (SCC) of the oral cavity, breast cancer, or melanoma. Lymphoseek received European approval for sentinel lymph node detection in melanoma, breast and oral cavity/head and neck cancers.

Last September, Navidea’s subsidiary, Macrophage Therapeutics, announced that it has developed a process for producing the first two therapeutic Manocept™ immunoconstructs, MT-1001, designed to specifically target and kill activated CD206+ macrophages, and MT-2001, designed to inhibit the inflammatory activity of activated CD206+ macrophages. Activated CD206+ macrophages are implicated in many diseases ranging from cancer to autoimmune diseases to central nervous system diseases such as Alzheimer’s disease and multiple sclerosis.

Navidea’s NAV4694, a second-generation PET imaging agent, is in Phase III development. The goal is to aid the differential diagnosis of mild cognitive impairment and dementia, including Alzheimer's disease, allowing patients to seek earlier treatment options.

The firm’s Manocept™ CD206 Targeting Platform for diagnostic and therapeutic applications is focused on CD206-targeting of macrophages across a range of potential disorders, including rheumatoid arthritis, Kaposi’s sarcoma, Crohn’s disease, cancer, tuberculosis, and vulnerable plaque.

Navidea recently reported that the first patient had been enrolled in a clinical study evaluating Lymphoseek injection in women with known cervical cancer.


 


 



Bind Therapeutics

Bind Therapeutics labels itself as a clinical-stage nanomedicine platform company. The company is developing Accurins®, a new class of targeted and programmable therapeutics based on the firm’s Medicinal Nanoengineering® platform. The company explains that Accurins are nanoparticles designed with specified physical and chemical characteristics. They are intended to target tumors at three levels: tissue, cellular, and molecular. They combine this triple targeting with a prolonged circulation time to concentrate the therapeutic payload at the targeted disease site, where it is then released in a controlled and timely manner, note company officials.

Bind has created Accurins on its own, initially in oncology, as well as Accurins in collaboration with biopharmaceutical companies. Its lead drug candidate, BIND-014, is currently enrolling patients with squamous histology non-small cell lung cancer (NSCLC) in a Phase II clinical trial called iNSITE1. The company is also evaluating BIND-014 in patients with advanced cervical cancer and head and neck cancers in a second Phase II trial called iNSITE2.

The company has collaborated or is collaborating with Pfizer, AstraZeneca, Roche, Merck, Macrophage Therapeutics (a subsidiary of Navidea Biopharmaceuticals) and Amgen to develop Accurins that are based on therapeutic payloads and are capable of targeting ligands from the collaborators’ product pipelines.

Bind recently entered into a research collaboration with Synergy Pharmaceuticals to engineer Accurins incorporating Synergy's proprietary uroguanylin analogs to explore the potential targeting of guanylate cyclase-C receptors expressed on tumors, specifically gastrointestinal malignancies.






Cellular Dynamics International

Cellular Dynamics International (CDI), a Fujifilm company, develops and manufactures human cells used in drug discovery, toxicity testing, stem cell banking, and cell therapy. A key part of CDI’s strategy is to partner with collaborators from around the world to combine biologically relevant human cells with the newest technologies to drive advancements in medicine.

CDI scientists maintain that their technology offers the potential to create induced pluripotent stem cells (iPSCs) from anyone. The technology, which starts with a standard blood draw, culminates with processes that allow the iPSCs that are created to develop into virtually any cell type in the human body. The company’s proprietary manufacturing system reportedly produces billions of cells daily, resulting in inventoried iCell® products and donor-specific MyCell® products that are used in drug and cell therapy development.

Last September, CDI announced the launch of the world’s largest publicly available stem cell bank, the California Institute for Regenerative Medicine hPSC Repository, and the availability of the first 300 iPSC lines. Two months later, CDI signed an agreement with Roche to supply CDI’s iPSC-derived iCell products to contribute to the identification of novel drug candidates at the early stages of drug discovery. Roche has exclusive access to specific iCell products to be employed in high-throughput screening and lead optimization for drug discovery.






Pulmatrix

Pulmatrix has focused on developing inhaled therapies to address serious pulmonary diseases using its iSperse™ (inhaled small particles easily respirable and emitted) dry powder technology. iSperse is designed to maximize local drug concentrations and reduce systemic side effects.

Pulmatrix’s Pur0200 incorporates a marketed long-acting muscarinic antagonist bronchodilator and is targeted against chronic obstructive pulmonary disease. It is currently being developed in Europe based on pharacokinetics bioequivalence, and a U.S. development program is being planned.

Pur1900 contains a large, complex antifungal compound intended for the treatment of cystic fibrosis (CF) patients. It is estimated that nearly 50% of patients with CF experience pulmonary fungal infections that can cause chronic bronchitis or allergic reactions, resulting in inflammation and poor long-term outcomes. Pur1900 is the first inhaled antifungal product candidate for CF and is in preclinical development.

Pur1500 incorporates a kinase inhibitor with anti-angiogenesis activity for the treatment of idiopathic pulmonary fibrosis (IPF), a progressive and generally fatal disease characterized by scarring of the lungs. IPF causes an irreversible loss of the tissue’s ability to transport oxygen. Pur1500 is intended to suppress the fibrotic pathway at the site of disease in the lung interstitium. The product is undergoing formulation feasibility studies.

In February, Pulmatrix agreed to collaborate with Capsugel to develop novel inhaled therapeutics to treat pulmonary disorders. Capsugel received exclusive rights to manufacture clinical trial and commercial batches of iSperse-based inhaled therapeutic candidates being developed by Pulmatrix and its potential development partners. Capsugel will contribute a combination of its spray-drying process development, scale-up expertise, and commercial manufacturing capabilities.
































 

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