Bio Platforms
DRUG DELIVERY – ENHANZE (®): An Efficient Way to Optimize Biologic Therapies for Subcutaneous Administration
Michael J. LaBarre, PhD, discusses how the ENHANZE drug delivery technology has the potential to improve the pharmacokinetic profiles of co-administered drugs through increased dispersion, absorption, and bioavailability.
CHECKPOINT INHIBITORS - Novel Targets & Global Markets
Laurie L. Sullivan and Cheryl L. Barton, PhD, say as manufacturers expand the clinical utility and healthcare professionals become more familiar with their efficacy and safety profiles, the checkpoint inhibitor market will continue to expand, becoming the cornerstone of many cancer treatment regimens.
Adynxx Announces Merger Agreement With Alliqua BioMedical
Adynxx, Inc. recently provided a business and clinical development update. Adynxx entered into a merger agreement with Alliqua Biomedical, Inc. that will result in Adynxx…
ExCellThera’s Lead Technology Receives FDA Regenerative Medicine Advanced Therapy Designation
ExCellThera Inc. recently announced the US FDA has granted regenerative medicine advanced therapy (RMAT) designation to its lead technology, ECT-001, in the treatment of hematologic…
Autolus Therapeutics Receives FDA Orphan Drug Designation
Autolus Therapeutics plc recently announced the US FDA has granted orphan drug designation to autologous enriched T-cells genetically modified with a retroviral vector to express two chimeric antigen receptors targeting CD19 and CD22 (AUTO3) for the treatment of acute lymphoblastic leukemia (ALL).
CureVac Granted Patent Claiming the Combination of mRNA Vaccines With Anti-PD-L1 Antibodies
CureVac AG recently announced it has been granted a patent from the European Patent Office entitled, Combination of Vaccination and Inhibition of the PD-1 Pathway…
Rexahn & BioSense Global Announce Collaboration & License Agreement
Rexahn Pharmaceuticals, Inc. and BioSense Global LLC recently announced a collaboration and license agreement to advance the development and commercialization of RX-3117 for pancreatic cancer…
Kamada Announces FDA Acceptance of Inhaled AAT Program
Kamada Ltd. recently announced receipt of a letter from the US FDA stating the company has satisfactorily addressed the concerns and questions regarding its Inhaled…
Fibrocell Announces Development & Commercialization Collaboration With Castle Creek Pharmaceuticals
Fibrocell Science, Inc. recently announced the completion of a collaboration agreement with Castle Creek Pharmaceuticals to develop and commercialize Fibrocell’s lead gene therapy candidate, FCX-007,…
Soligenix Achieves Significant Enrollment Milestone for its Pivotal Phase 3 Clinical Trial
Soligenix, Inc. recently announced today it has reached a significant milestone in the Phase 3 clinical study (the “DOM-INNATE” study) for SGX942 (dusquetide) in the treatment of oral mucositis in patients with head and neck cancer (HNC).
Paragon Bioservices to be Acquired for $1.2 Billion
Catalent, Inc. and Paragon Bioservices, Inc. and Catalent Inc. recently announced they have entered into a definitive agreement under which Catalent will acquire Paragon for $1.2 billion.
Neon Therapeutics Announces Completion of Enrollment in Phase 1b Clinical Trial
Neon Therapeutics, Inc. recently announced the completion of enrollment in NT-002, its Phase 1b clinical trial evaluating NEO-PV-01 with KEYTRUDA (pembrolizumab) and chemotherapy in patients…
Thermo Fisher Scientific Announces Collaboration to Advance Biopharmaceutical Characterization & Monitoring Methods
Thermo Fisher Scientific and Genovis recently announced a collaborative research project to develop advanced end-to-end workflows for the preparation, characterization, and monitoring of novel and…
XOMA Acquires Royalty Rights to Five Hematology Candidates
XOMA Corporation recently announced it has agreed to acquire the rights to potential royalty payments and a portion of the potential milestone payments associated with…
Bellerophon Announces Agreement With FDA on Regulatory Approval Pathway for INOpulse
Bellerophon Therapeutics, Inc. recently announced it has reached agreement with the US FDA on the regulatory approval pathway for INOpulse in patients with Pulmonary Hypertension…
SomaLogic Announces New Collaboration With Janssen Research & Development
SomaLogic, Inc. recently announced it has entered into a collaborative agreement with Janssen Research & Development, LLC, one of the Janssen Pharmaceutical Companies of Johnson…
BioAtla & BeiGene Form Worldwide Development & Commercialization Collaboration
BioAtla , LLC and BeiGene, Ltd. recently announced the two companies have entered into a global co-development and collaboration agreement for the development, manufacturing, and commercialization of BioAtla’s investigational CAB CTLA-4 antibody (BA3071).
Angion Awarded $4.76-Million Department of Defense Grant for Drug Candidate
Angion Biomedica Corp. recently announced it had received a $4.76-million Department of Defense (DoD) follow-on grant for ANG-3070, its precision drug candidate for the treatment…
CytoDyn & Samsung BioLogics Enter $1-Billion Agreement
CytoDyn Inc. recently announced the execution of a comprehensive strategic agreement with Samsung BioLogics Co., Ltd. for the clinical and commercial manufacturing of leronlimab (PRO…
Amfora Licenses Gene-Editing Technology
Amfora recently announced it has reached a non-exclusive research and commercial license agreement with Corteva Agriscience, the Agriculture Division of DowDuPont, and the Broad Institute…
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).