Bio Platforms
Ventyx Biosciences Announces Completion of Enrollment of Phase 2 Trial of VTX002 in Ulcerative Colitis & Phase 2 Trial of VTX958 in Plaque Psoriasis
Ventyx Biosciences, Inc. recently announced it has completed patient enrollment in the Phase 2 trial of VTX002 in ulcerative colitis and the Phase 2 SERENITY…
Axol Bioscience Collaborates With StrataStem to Deliver Stem Cell-Based ‘Clinical Trial in a Dish’ for Alzheimer’s Disease
Axol Bioscience Ltd. has signed an exclusive agreement with StrataStem to access and commercialize its extensive collection of Alzheimer’s Disease (AD) patient samples…..
European Patent Office to Grant ZyVersa Therapeutics’ Patent Application for Cholesterol Transport Mediator for Use in Patients With Diabetic Nephropathy/Diabetic Kidney Disease
ZyVersa Therapeutics, Inc. recently announces the European Patent Office has issued a “Notice of Intention to Grant” ZyVersa’s patent application covering the company’s Phase 2a-ready…
Needle-Free Vaccine Delivery Platform Aims to End Frozen Storage Needs & Improve Access
aVaxziPen’s innovative vaccine delivery platform aims to improve access to vaccines by removing the need for frozen storage and enabling easier distribution and deployment….
Anavex Life Sciences Announces Completion of Phase 2/3 Rett Syndrome Clinical Trial
Anavex Life Sciences Corp. recently announced the completion of dosing of all participants of the placebo-controlled EXCELLENCE Phase 2/3 study ANAVEX2-73-RS-003 in pediatric patients with…
SPECIAL FEATURE - Outsourcing Formulation Development & Manufacturing: Putting Customers First
Contributor Cindy H. Dubin speaks with several leading CDMOs from around the globe on their unique development and manufacturing capabilities and technologies, and presents real-world examples of how they have put these to use to produce innovative compounds, lower development costs, and shorten time to market.
Catalent Expands OneBio® Suite for Integrated Development, Manufacturing & Supply Across Biologic Modalities
Catalent recently announced it has expanded its integrated development, manufacturing and supply solution, OneBio® Suite, across a range of biologic modalities, including antibody and recombinant…
NovelMed Phase 1 Clinical Trial Shows Inhibition of the Alternative Pathway & Preservation of the Classical Pathway – A Long-Acting Anti-Properdin Monoclonal Antibody for PNH Patients
NovelMed Therapeutics, Inc. recently announced topline results from First-in-Human Phase 1 clinical trial of its complement blocker monoclonal anti-Properdin antibody, known as NM3086…..
Synlogic Announces Initiation of Synpheny-3 Global, Pivotal Phase 3 Study Evaluating SYNB1934 for Treatment of Phenylketonuria
Synlogic, Inc. recently announced the initiation of Synpheny-3, a global, pivotal Phase 3 study evaluating the efficacy and safety of SYNB1934 as a potential treatment…
Portage Biotech Reports Updated Interim Data for Lead iNKT Engager in Phase 1/2 Trial for the Treatment of Advanced Melanoma & Metastatic Non-Small Cell Lung Cancer
Portage Biotech Inc. recently announced updated interim data from the Phase 1 portion of the trial evaluating its lead invariant natural killer T cell (iNKT)…
Artax Biopharma Announces Positive Phase 1 Results for Company’s First-in-Class Oral Immunomodulator Developed to Treat T Cell-Mediated Diseases
Artax Biopharma, Inc. recently announced positive Phase 1 clinical trial results for AX-158, the company’s first-in-class oral small molecule immunomodulator to enter clinical development to…
Medigene Announces Pipeline Expansion Into Neoantigens With Programs Targeting Multiple KRAS Mutations & HLAs
Medigene AG recently announced its pipeline expansion into neoantigens (also known as oncogenic driver mutations) with KRAS (Kirsten rat sarcoma viral oncogene homologue) as the…
Coave Therapeutics Announces Positive 12-Month Data From Ongoing Phase 1/2 Clinical Trial of CTx-PDE6b in Patients With Retinitis Pigmentosa Caused by Bi-allelic Mutations in PDE6b
Coave Therapeutics recently announces the positive 12-month results from its Phase 1/2 trial evaluating the safety and efficacy of its innovative gene therapy, CTx-PDE6b, for…
Minoryx Gains FDA Approval to Initiate Phase 3 Trial in Patients With Cerebral Adrenoleukodystrophy
Minoryx Therapeutics recently announced US FDA approval of its Phase 3 clinical trial (CALYX) of lead candidate leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD)…
Evonik Launches Next-Generation Peptide for Biopharma Applications
Evonik now offers cQrex® KC, a performance-boosting peptide for cell culture media used in the manufacture of biological drugs. The new peptide enables cell culture process developers….
Positive Phase 2 Data of Novel Investigational Anti-CD40L Antibody Frexalimab Show Significantly Reduced Disease Activity in Relapsing Multiple Sclerosis
New data, being presented in a late-breaking session at the 2023 Consortium of Multiple Sclerosis Centers (CMSC) annual meeting, demonstrate that frexalimab, Sanofi’s novel second-generation…
MetrioPharm Receives Orphan Drug Designation for the Treatment of Duchenne Muscular Dystrophy
MetrioPharm AG recently announced the US FDA has granted Orphan Drug Designation for MP1032 for the treatment of Duchenne muscular dystrophy. DMD is the most…
Silo Pharma Commences Study of Novel Joint Homing Peptide SPU-21
Silo Pharma, Inc. recently announced its entry into a research agreement expanding the study of its novel joint homing peptides targeting rheumatoid arthritis (RA), designated…
OLON Enters ADC Market
OLON Group recently announced the start of construction of a new facility at its Rodano site (Milan, Italy) which will be entirely dedicated to managing and producing Ultra-Potent compounds, used for example as payloads and payload-linkers for antibody-drug conjugates (ADCs), one of the….
LIXTE Biotechnology’s Lead Clinical Compound Increases Effectiveness of Cancer Immunotherapy
LIXTE Biotechnology Holdings, Inc. recently announced a recently published article in the journal Cancer Research showed that PP2A, the pharmacologic target of LIXTE’s lead clinical…
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).