Bio Platforms
PLATFORM TECHNOLOGY - Overcoming the Challenges of Drug Brain Delivery With a Novel Brain Delivery Vector
Mei Mei Tian, PhD, explains how the xB3 platform has the potential to support development of treatments for CNS diseases that can treat thousands of patients in the years ahead.
VACCINE DEVELOPMENT - COVID-19 Vaccine Focusing on T Cells to Protect the Most Vulnerable
Jeffrey Wolf, MBA, JD, says while most vaccines in development are targeted to protect healthy people, few are focusing on those who are most at risk – seniors and those with co-morbidities like heart failure, obesity, or type 2 diabetes.
Assembly Biosciences & Door Pharmaceuticals Sign Collaboration & Option Agreement
Assembly Biosciences, Inc. and Door Pharmaceuticals, LLC recently announced the companies have signed an exclusive, 2-year collaboration and option agreement focused on the development of….
XOMA Earns $2-Million Milestone From Takeda
XOMA Corporation recently announced it has earned a $2-million milestone payment from Takeda Pharmaceutical Company Limited as the first patient has been dosed….
Beam Therapeutics Presents First Data Highlighting Base Editing Program for Glycogen Storage Disease Type Ia
Beam Therapeutics Inc. recently announced the company will present preclinical data from its liver-focused programs, including the first data highlighting its novel base-editing strategy for…
CalciMedica Announces Publication of Preclinical Data Demonstrating the Pathological Role of Microglial CRAC Channels in Ischemic Stroke
CalciMedica Inc. recently announced the publication of preclinical data demonstrating a pathological role for CRAC channel-mediated microglial calcium activity in ischemic brain injury in the…
BioAegis Therapeutics Expands Phase 2 COVID-19 Treatment Clinical Trial
BioAegis Therapeutics Inc. recently announced it has expanded its clinical trial in severe COVID-19 to additional sites in Europe. The study has recruited more than…
Apellis Announces FDA Acceptance & Priority Review of NDA
Apellis Pharmaceuticals, Inc. recently announced the US FDA has accepted and granted Priority Review designation for the New Drug Application (NDA) for pegcetacoplan for the treatment…
XOMA Earns First Milestone Payment From 2018 Royalty Purchase Agreement
XOMA Corporation recently announced it has earned a $1-million milestone payment from Agenus Inc. related to Merck’s advancement of MK-4830 into a Phase 2 study…
Croda Enters Contract With Pfizer for Innovative Delivery System for COVID-19 Vaccine
Croda International Plc recently announced it has entered into an agreement with Pfizer Inc. to supply novel excipients used in the manufacture of a COVID-19….
Altimmune Adds Lonza as a Manufacturing Partner for Supply of AdCOVID its Single-Dose Intranasal Vaccine Candidate for COVID-19
Altimmune, Inc. recently announced that it entered into an agreement with Lonza for the manufacturing of AdCOVID, Altimmune’s next-generation, single-dose intranasal vaccine candidate for COVID-19.…
4D Pharma Presents New Data From Two MRx0518 Clinical Trials
4D pharma plc recently announced new positive clinical data from two clinical trials of MRx0518, its lead immuno-oncology single strain Live Biotherapeutic candidate. The data was…
Axovant Gene Therapies Announces FDA Clearance of IND for Gene Therapy
Axovant Gene Therapies Ltd. recently announced the US FDA has lifted its clinical hold and cleared the Investigational New Drug (IND) Application to initiate a registrational study of….
Mateon Therapeutics & Windlas Biotech Enter Commercialization Agreement for Covid-19 Drug
Mateon Therapeutics recently announced an agreement with Windlas Biotech Pvt. Ltd. of India (Windlas) to commercialize ARTIVeda, Mateon’s lead ethnobiology….
Dolomite Microfluidics & MilliporeSigma Collaborate to Release Off-the-Shelf Microfluidic Device Kits for the Fabrication of PLGA Particles
Dolomite Microfluidics and MilliporeSigma have partnered to create a range of off-the-shelf NanoFabTx™ microfluidic device kits for the production of….
Pfizer & BioNTech Reach Agreement to Supply the EU With 200 Million Doses of COVID-19 Vaccine
Pfizer and BioNTech SE recently announced they have reached an agreement with the European Commission to supply 200 million doses of their investigational….
Novo Nordisk to Acquire Emisphere Technologies for $1.35 Billion
Emisphere Technologies, Inc. recently announced it has entered into a definitive agreement with Novo Nordisk, whereby Novo Nordisk will acquire Emisphere, on a cash-free, debt-free basis, for….
CymaBay Announces Study to Evaluate the Potential for GPR119 Agonists to Prevent Hypoglycemia in Type 1 Diabetes
CymaBay Therapeutics, Inc. recently announced it will provide its proprietary investigational GPR119 agonist, MBX-2982, and will assist in regulatory filings for….
eGenesis Announces Research Collaboration With Leading Academic Medical Center
eGenesis recently announced the initiation of a research collaboration with Duke University School of Medicine. The collaboration will encompass evaluation of gene-edited pancreatic islet….
Calithera Biosciences Announces Expansion of Ongoing Clinical Trial
Calithera Biosciences, Inc. recently announced the expansion of the company’s ongoing clinical trial evaluating telaglenastat in combination with Pfizer’s CDK 4/6 inhibitor palbociclib….
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).