Bio Platforms
IVERIC bio Enters Strategic Manufacturing Relationship With Catalent's Paragon Gene Therapy
IVERIC bio, Inc. and Catalent Biologics recently announced they have entered into an agreement for production and manufacturing of GMP-grade adeno-associated virus (AAV) vector for IVERIC bio’s gene therapy product candidates.
Innovate Biopharmaceuticals Announces Start of First Phase 3 Clinical Trial
Innovate Biopharmaceuticals, Inc. recently announced the start of the first Phase 3 clinical trial in celiac disease. Innovate expects to provide further updates as screening…
Deciphera Pharmaceuticals and Zai Lab Limited Announce Exclusive License Agreement
Deciphera Pharmaceuticals, Inc. and Zai Lab Ltd. recently announced an exclusive license agreement to advance the development and commercialization of ripretinib in Greater China (mainland…
InflaRx Treats First Patient in Phase 2a Clinical Trial
InflaRx N.V. recently announced the treatment of the first patient in a Phase 2a clinical trial evaluating the company’s lead product candidate, IFX-1, in Pyoderma…
Oncologie Closes $80-Million Financing for Clinical-Stage Pipeline
Oncologie recently announced it has closed an $80M Series B financing. Existing investors, led by Nan Fung Life Sciences and Pivotal BioVentures China, are joined…
Ligand Subsidiary Vernalis & PhoreMost Limited Announce Collaboration for Novel Oncology Target
Ligand Pharmaceuticals Incorporated and PhoreMost Limited recently announced the signing of a research collaboration agreement between Ligand’s subsidiary Vernalis and PhoreMost on an undisclosed novel oncology target.
Biogen Completes Acquisition of Nightstar Therapeutics for Approximately $800 Million
Biogen recently announced it has completed its acquisition of Nightstar Therapeutics, a clinical-stage gene therapy company, which is focused on adeno-associated virus (AAV) treatments for inherited retinal disorders.
ASLAN Pharmaceuticals Completes Phase 1 Study
ASLAN Pharmaceuticals recently announced the successful completion of its single ascending dose (SAD) study testing the first-in-class therapeutic antibody ASLAN004 in healthy volunteers, and the…
BioXcel Therapeutics Announces FDA Clearance of IND Application
BioXcel Therapeutics recently announced an update of its immuno-oncology program for BXCL701, an orally available systemic innate-immune activator with dual mechanisms of action. BTI is…
Allena Pharmaceuticals Announces Interim Study Results
Allena Pharmaceuticals, Inc. recently announced interim data from Study 206, its Phase 2 basket clinical trial of reloxaliase, an orally administered, recombinant oxalate-degrading enzyme. Study…
VBI Vaccines Reports Data From Part A of the Ongoing Phase 1/2a Study
VBI Vaccines Inc. recently presented a poster at the American Society of Clinical Oncology (ASCO) Annual Meeting that exhibited expanded clinical data from Part A…
Treos Bio Announces Positive Interim Results from a Phase 1/2 Clinical Trial
Treos Bio Limited reported positive interim results from its first-in-man Phase 1/2 study of its precision cancer vaccine with maintenance therapy in patients with metastatic…
Nanobiotix Announces Launch of Nanotechnology Platform
NANOBIOTIX recently announced the launch of new subsidiary Curadigm, dedicated to redefining the therapeutic balance between bioavailability, toxicity, and efficacy across the pharmaceutical industry. The…
Pfenex & Alvogen Announce EMA Accepts Marketing Authorization Application
Pfenex Inc. and Alvogen recently announced that the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) submitted by our partner Alvogen for…
Fluidigm Introduces Advanta RNA Fusions NGS Library Prep Assay
Fluidigm Corporation recently introduced the Advanta™ RNA Fusions NGS Library Prep Assay for detection of over 380 gene fusion pairs from solid tumors and hematological…
Biomunex Pharmaceuticals Establishes US Subsidiary
Biomunex Pharmaceuticals recently announced that it has established its US subsidiary, Biomunex Pharmaceuticals Inc., in Cambridge, MA.
CureVac Enters Into Exclusive Sponsored Research Agreement
CureVac AG, a fully integrated biopharmaceutical company pioneering the field of mRNA-based drugs, recently announced it has entered into a sponsored research agreement (SRA) with…
ProBioGen & Vaccitech Sign License Agreement
ProBioGen AG and Vaccitech, Ltd. jointly announced signing a license agreement in which Vaccitech will gain access to ProBioGen’s proprietary technology platform based on the…
Codexis Announces CodeEvolver Technology Transfer & License Agreement With Global Pharmaceutical Leader
Codexis, Inc. recently announced the signing of a CodeEvolver platform agreement with Novartis. Under the terms of the agreement, Codexis has granted a non-exclusive license…
Adaptimmune & Alpine Immune Sciences Announce Collaboration & License Agreement
Adaptimmune Therapeutics plc and Alpine Immune Sciences, Inc. recently announced a collaboration and license agreement to develop next-generation SPEAR T-cell products that incorporate Alpine’s secreted…
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).