Bio Platforms
Cybrexa Therapeutics’ alphalex Platform Enables Tumor Targeting of Multiple PARP Inhibitors
Cybrexa Therapeutics recently announced preclinical data supporting the potential of its proprietary alphalex technology platform.
Caris Life Sciences Receives FDA Breakthrough Device Designation
Caris Life Sciences recently announced the US FDA has granted Breakthrough Device designation for the company’s MI Transcriptome companion diagnostic (CDx) test. Designed to detect…
Merus Announces First Patient Treated in Phase 1 Clinical Trial for Advanced Solid Tumors
Merus N.V. recently announced the first patient has been treated in its Phase 1 trial evaluating safety, tolerability, and preliminary efficacy of MCLA-145 for the…
Sarepta Announces Agreement With Nationwide Children’s Hospital for Rights to its Gene Therapy Program
Sarepta Therapeutics, Inc. recently announced it has recently signed an agreement with the Research Institute at Nationwide Children’s Hospital (Nationwide Children’s) giving Sarepta the exclusive…
Octapharma Study Demonstrates Cutaquig Efficacy & Safety
Octapharma presented clinical research results at the Clinical Immunology Society Annual Meeting in Atlanta demonstrating the efficacy and safety of cutaquig [Immune Globulin Subcutaneous (Human)…
HOOKIPA Achieves Research Milestone in Collaboration & License Agreement With Gilead
HOOKIPA Pharma Inc. recently announced it has achieved a further research milestone in its collaboration and license agreement with Gilead Sciences, Inc. for development of a therapeutic hepatitis B virus (HBV) vaccine.
Calixar Signs Exclusive Licensing Agreement With Regeneron
Calixar SAS (Calixar), a biotechnology company specialized in native membrane protein stabilization, recently announced it has entered into an exclusive licensing agreement with Regeneron Pharmaceuticals,…
Emendo Biotherapeutics Enters Licensing Option Agreement With Takeda
Emendo Biotherapeutics Inc. recently announced the company signed a licensing option agreement with Takeda Pharmaceutical Company Limited. As part of the transaction, Emendo will receive…
International Stem Cell Completes Enrollment & Dosing in its Parkinson's Disease Clinical Trial
International Stem Cell Corporation recently announced the completion of subject enrollment in its Phase 1 clinical trial of ISC-hpNSC for the treatment of Parkinson’s disease.…
Oculos & Optceutics Partner to Bring Expanded Services to Ophthalmic Innovators
Oculos and Optceutics recently announced they are entering into a co-promotion arrangement to provide broader services for clients spanning from early research through product approval.
Caris Life Sciences Acquires Pharmatech
Caris Life Sciences recently announced it has acquired Pharmatech, Inc., a pioneer of the original Just-In-Time research system with the largest research-ready oncology network.
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,…
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).