Bio Platforms
Arcturus Therapeutics & Catalent Announce Partnership to Manufacture mRNA-Based COVID-19 Vaccine
Arcturus Therapeutics Holdings Inc. and Catalent, Inc.recently announced a partnership to support the expected manufacture of Arcturus’ COVID-19 mRNA vaccine candidate (LUNAR-COV19), intended to….
Valneva & Pfizer Announce Collaboration to Co-Develop & Commercialize Vaccine
Valneva SE (Valneva) recently announced a collaboration to develop and commercialize Valneva’s Lyme disease vaccine candidate….
GLOBAL REPORT - 2019 Global Drug Delivery & Formulation Report: Part 3, Notable Drug Delivery & Formulation Transactions & Technologies of 2019
In part 3 of this 4-part series, PharmaCircle, in collaboration with Drug Development & Delivery, focuses on several notable drug delivery and formulation transactions and technologies.
Quotient Sciences & CytoAgents Accelerate Potential Treatment for COVID-19 Cytokine Storm
A collaboration to accelerate the development of a lead COVID-19 drug candidate into human clinical trials was recently announced today by Quotient Sciences, a leading…
Trio Pharmaceuticals & Ajinomoto Bio-Pharma Services Enter Development Collaboration for a Novel Antibody Therapeutic
Trio Pharmaceuticals, Inc. and Ajinomoto Bio-Pharma Services recently announced a development collaboration agreement to evaluate AJICAP, a proprietary site-specific conjugation technology offered by…
Avadel Pharmaceuticals Announces Positive Topline Results from its Pivotal Phase 3 REST-ON Trial
Avadel Pharmaceuticals plc recently announced positive topline data from its pivotal Phase 3 REST-ON trial assessing the safety and efficacy of FT218, an investigational, once-nightly…
Orchard Therapeutics Announces First Patient Dosed with OTL-201 Gene Therapy
Orchard Therapeutics recently announced that the first patient has been dosed in an open-label, proof-of-concept investigational study of OTL-201, an ex vivo autologous hematopoietic stem cell…
Harpoon Therapeutics Doses First Patient with HPN217, a BCMA Targeting TriTAC, for Multiple Myeloma
Harpoon Therapeutics, Inc. recently announced the first patient has been dosed with HPN217 in a Phase 1/2 clinical trial focused on relapsed, refractory multiple myeloma…
BioAegis Demonstrates Gelsolin Therapy Can Quell the COVID-19 Cytokine Storm & Promote Tissue Repair
BioAegis Therapeutics Inc. has highlighted recently published gene expression data in animal studies in which recombinant human plasma gelsolin (rhu-pGSN) significantly increased survival in pneumonia…
Santhera & Cold Spring Harbor Laboratory to Investigate Lonodelestat in COVID-19-Related ARDS
Santhera Pharmaceuticals recently announced it has entered into a collaboration agreement with Cold Spring Harbor Laboratory (CSHL) to investigate the potential of lonodelestat (POL6014), a…
Histogen Announces Investigational Device Exemption Application
Histogen Inc. recently announced it has submitted an Investigational Device Exemption (IDE) application with the US FDA for the initiation of a….
Mogrify & Sangamo Announce Collaboration & Exclusive License Agreement
Mogrify Ltd and Sangamo Therapeutics recently announced they have executed a collaboration and exclusive license agreement for Sangamo to develop….
Mustang Bio Receives Advanced Therapy Medicinal Product Classification
Mustang Bio, Inc. recently announced that the European Medicines Agency (EMA) has granted Advanced Therapy Medicinal Product (ATMP) classification to….
Q BioMed Initiates Rapid Development of Novel COVID-19 Therapeutics
Q BioMed Inc. recently announced that together with its technology partner, Mannin Research, they are accelerating the rapid development of novel drugs for the treatment of life-threatening complications caused by COVID-19 and….
Catalyst Biosciences Completes Phase 2b Trial of Subcutaneous Factor IX Dalcinonacog Alfa (DalcA)
Catalyst Biosciences, Inc. recently announced completion of dosing and the 30-day follow-up period for its Phase 2b trial of SQ dalcinonacog alfa (DalcA). “We are…
INmune Bio Announces Initiation of Clinical Program to Determine if Company’s Platform May Prevent Complications of Cytokine Storm Caused by COVID-19
INmune Bio, Inc. recently announced the initiation of a therapeutic program to treat patients with pulmonary complications from COVID-19 infection with its dominant-negative TNF inhibitor…
Vaxart Announces Positive Preclinical Data for its Oral COVID-19 Vaccine Program
Vaxart, Inc., a clinical-stage biotechnology company developing oral recombinant vaccines that are administered by tablet rather than by injection, recently announced it has obtained positive…
New COVID-19 Clinical Trial Supported by Octapharma USA
Octapharma USA is supporting a new investigator initiated clinical trial led by George Sakoulas, MD, of Sharp Memorial Hospital in San Diego, CA, focused on treating the most critical patients at the….
TFF Pharmaceuticals Announces R&D Collaboration for a Universal Influenza Vaccine
TFF Pharmaceuticals, Inc. recently announced that it has entered into a research collaboration with the University of Georgia’s Center for Vaccines and Immunology (CVI), along with….
HALIX Enters Collaboration for GMP Manufacturing of a COVID-19 Vaccine
HALIX B.V. has recently joined a consortium of partners under the guidance of the University of Oxford, to provide GMP manufacturing services supporting the large scale production of….
What are Bio Platforms?
Platforms (or asset-independent technologies to capture all kinds of capabilities that can be leveraged across many different drug candidate assets rather than just discovery tools that the term ‘platform’ immediately brings to mind) are ubiquitous in modern pharma. They are the product of an arms race, to secure access to the best capabilities in key areas.
Platform technologies are considered a valuable tool to improve efficiency and quality in drug product development. The basic idea is that a platform, in combination with a risk-based approach, is the most systematic method to leverage prior knowledge for a given new molecule. Furthermore, such a platform enables a continuous improvement by adding data for every new molecule developed by this approach, increasing the robustness of the platform.
But it has often been said that access to the latest technological platforms to aid efficient drug discovery and development is limited to Big Pharma, which can more easily justify the costs of creating and operating these platforms.
Benefits of Bio Platforms
Platform technologies have the ability to radically improve upon current products and generate completely novel products. In this sense, they open up new arenas for drug discovery and development, potentially increasing the number of therapeutic options for patients. Once a single compound or therapeutic has been generated and demonstrates a clinical benefit in patients, it is more likely this platform technology can successfully be applied to other therapeutic areas, derisking future compounds/products.
Complex drugs by their very nature are challenging and costly to manufacture. This, in turn, translates into higher costs for patients and other payers. In order to provide safe and effective therapies at a reasonable price, it is necessary for the industry to develop manufacturing technologies that reduce costs and provide a consistent product. While the initial investment may be larger, manufacturing costs will be lower over time as the manufacturing process is solidified.
Scale and Investment of Bio Platforms
Despite the initial upfront costs, platform technologies inevitably provide pragmatic solutions to production challenges, while yielding safer and more effective therapeutic products. It has often been said that one of the key features that distinguishes “Big Pharma” from biotech is access to the latest technological platforms to aid efficient drug discovery and development.
These platforms range from vast chemical libraries, ultra-high throughput screening and huge genetic databases in discovery, to predictive toxicology platforms, cutting-edge ‘omics’ and even deep-seated knowledge of particular therapeutic areas in development. All these platforms have two things in common: They can be used on any (or many) development candidate assets, and they cost huge sums to establish in the first place, and in a few cases each time they are used as well. Hence their restriction to the largest pharmaceutical companies (and a few of the so-called “big biotechs” that are, in many ways, indistinguishable from the old-guard pharma).
Only when you have hundreds of active projects can you justify the cost of creating and operating these platforms. Or so the mantra goes. It is access to these platforms that keeps the big companies ahead in the race to discover and develop the best medicines (or at least counterbalance the disadvantages of being large and slow-moving, depending on your point of view). But is that just an assertion? How much evidence is there to support the proposition that the efficiency gains due to these platforms outstrips the cost of creating and maintaining them?
Keeping these technologies “cutting edge” has become so expensive that increasingly we hear pharma companies talking of “pre-competitive” approaches to develop the next generation. A group of companies might develop a platform capability they then share. The principle goal of such initiatives is to access even grander and more expensive tools than individual companies could afford, rather than to dramatically cut costs (although sharing platforms rather than developing the same thing in parallel in each silo should at least keep a lid on rising costs).