Bio Platforms
EXECUTIVE INTERVIEW - Eloxx Pharmaceuticals: Developing Rare Disease Drugs for Nonsense Mutations
Bob Ward, Chairman and CEO at Eloxx Pharmaceuticals, discusses nonsense mutations and how his company’s goal is to bring safe and effective therapies to children and adults suffering from genetic diseases as quickly as possible.
Aptinyx Presents Data Demonstrating Positive Activity on Cognitive Performance & Biomarkers in a Preclinical Model
Aptinyx Inc. recently announced the presentation of preclinical data on its novel NMDA receptor modulator, NYX-458. The product candidate demonstrated positive recovery of performance on…
Altimmune Announces $3.7 Million in Additional BARDA Funding
Altimmune, Inc. recently announced the Biomedical Advanced Research and Development Authority (BARDA) is modifying its existing anthrax vaccine development contract with Altimmune by awarding an…
Cara Therapeutics Enters Commercial License Agreement With Enteris BioPharma
Cara Therapeutics, Inc. recently announced it has entered into a non-exclusive commercial license agreement with Enteris BioPharma, Inc. for oral formulation rights to…..
Viral Vectors Expected to Play Pivotal Role in Gene Therapy Pipeline for Neurology Indications
Viral vectors, such as adeno-associated virus (AAV), are expected to play a pivotal role in gene therapies for neurology indications, as this vector type has been found to be the most…..
Q BioMed & ChemVeda Announce Potential Chemotherapeutic Breakthrough
Q BioMed, Inc. and Chemveda Life Sciences recently announce the successful chemical synthesis of a unique natural compound that has shown remarkable efficacy as a potential………
Mustang Bio Announces $9.28-Million Grant to Fund Phase 1 Trial
Mustang Bio, Inc. recently announced that City of Hope has received a $9.28-million grant from the California Institute for Regenerative Medicine (CIRM) to fund an…
Preliminary Data Confirm Saphyr’s Potential to Replace Traditional Cytogenetics Methods
Bionano Genomics, Inc. recently announced a summary of results from two key studies presented this week at the 2019 Cancer Genomics Consortium Annual Meeting in…
AC Immune Announces Research Collaboration With University of Pennsylvania
AC Immune SA recently announced a research partnership with leading scientists in the Perelman School of Medicine at the University of Pennsylvania (Penn) focused on studying the pathological mechanisms of…..
CureVac Enters Exclusive Collaborative Research Agreement
CureVac AG recently announced it has entered into a Collaborative Research Agreement with Yale University for discovery research into mRNA-based……
INmune Bio Reports Positive Preliminary Data from INB03 Phase 1 Clinical Trial
INmune Bio, Inc. recently reported positive preliminary data during a presentation at Cambridge Healthtech Institute’s 7th Annual Immuno-Oncology Summit in Boston. INB03 is being developed…
Aquinox Pharmaceuticals & Neoleukin Therapeutics Announce Merger Agreement
Aquinox Pharmaceuticals, Inc. and Neoleukin Therapeutics, Inc. recently announced the two companies entered into a definitive merger agreement under which Aquinox agreed to the acquisition…
Celsion Announces Data Lock for First Interim Analysis in OPTIMA Phase 3 Study
Celsion Corporation recently announces the prescribed number of events has been reached for the first prespecified interim analysis of the OPTIMA Phase 3 Study with…
Eureka Therapeutics Announces Initiation of Phase 1/2 Clinical Trial
Eureka Therapeutics, Inc. recently announced initiation of Phase 1/2 clinical trial of ET140202 ARTEMIS T-cell therapy in liver cancer at City of Hope, a world-renowned…
Oncternal Therapeutics Announces Opening of Randomized Phase 2 Study
Oncternal Therapeutics, Inc. recently announced it has opened for enrollment its randomized Phase 2 study of cirmtuzumab, a ROR1-targeted monoclonal antibody, combined with ibrutinib in…
Spring Bank Announces Collaborative Research Agreement with University of Texas Southwestern Medical School
Spring Bank Pharmaceuticals, Inc. recently announced that it has entered into a research agreement with the University of Texas Southwestern Medical School to evaluate….
Principia Announces Update to Ongoing Phase 2 Clinical Trial
Principia Biopharma Inc. recently announced updates to its immune thrombocytopenia (ITP) program for PRN1008. The ongoing open-label, dose-finding clinical trial investigating PRN1008 in patients with…
Pfenex & Arcellx Announce Development, Evaluation & License Agreement
Pfenex Inc. and Arcellx, Inc. recently announced a Development, Evaluation, and License agreement under which Arcellx gains access to the proprietary Pfenex Expression Technology platform to advance……
Amicus Therapeutics & Catalent Biologics Enter Strategic Partnership
Amicus Therapeutics, Inc. has entered into a strategic manufacturing agreement with Paragon Gene Therapy, a unit of Catalent Biologics, for clinical manufacturing capabilities and capacity for….
Amicus Therapeutics & Brammer Bio Enter Strategic Development & Manufacturing Collaboration
Amicus Therapeutics, Inc. has entered into a strategic manufacturing collaboration with Thermo Fisher Scientific. Thermo Fisher, a best-in-class contract development and manufacturing organization (CDMO) with…
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).