Bio Platforms
Evaxion to Unveil Potentially Groundbreaking AI-Immunology Precision Cancer Vaccine Concept
Evaxion Biotech A/S recently announced the presentation of its novel precision vaccine concept at the 65th American Society of Hematology (ASH) Annual Meeting, taking place…
Roivant & Priovant Announce Results From Phase 2 Study of Oral Brepocitinib in Systemic Lupus Erythematosus
Roivant and Priovant recently announced the Phase 2 study evaluating oral brepocitinib in adult patients with moderate to severe active lupus did not meet its…
GRI Bio Announces FDA Clearance of IND for Lead Program GRI-0621, a Type 1 Invariant NKT Antagonist for the Treatment of Idiopathic Pulmonary Fibrosis
GRI Bio, Inc. recently announced the US FDA has cleared the company’s Investigational New Drug (IND) application for GRI-0621 for the treatment of IPF. The…
ADvantage Therapeutics Commences Enrollment for Phase 2b Clinical Trial for Alzheimer’s Disease Treatment
ADvantage Therapeutics, Inc. recently announced the first patient enrolled in the company’s European Phase 2b clinical trial on its lead candidate AD04 for the treatment…
Alladapt Immunotherapeutics Receives FDA Fast Track Designation for the Treatment of Mono- and Multi-Food Allergies
Alladapt Immunotherapeutics, Inc. recently announced that ADP101, its investigational multi-food oral immunotherapy (mOIT) designed to simultaneously treat allergy to one or more of the world’s…
Zevra Therapeutics Completes Acquisition of Acer Therapeutics in its Journey to Become a Leading Rare Disease Company
Zevra Therapeutics, Inc. recently announced the completion of its acquisition of Acer Therapeutics Inc., which marks a significant step forward in executing Zevra’s strategy to become….
Medicenna Announces Compelling Survival Benefit From Phase 2b Study of Bizaxofusp in Recurrent Gliobastoma
Medicenna Therapeutics Corp. recently announced a poster presentation and an oral summary highlighting longer term follow up results from the Phase 2b clinical trial of…
Uppsala University Hospital & Sana Biotechnology Announce Authorization of the First-in-Human Clinical Trial Application for a Primary Islet Cell Treatment for Patients With Type 1 Diabetes
Sana Biotechnology, Inc. and Uppsala University Hospital in Sweden recently announced the Swedish Medical Products Agency has authorized Uppsala University’s Clinical Trial Application (CTA) to…
Mural Oncology Launches to Advance Pipeline of Novel Engineered Cytokine Immunotherapies
Mural Oncology plc recently launched as an independent, publicly traded, clinical-stage immuno-oncology company leveraging its core competencies in immune cell modulation and protein engineering to develop….
Evaxion Presents Proof-of-Principle for Its Unique AI Model Predicting Responses to Cancer Immunotherapy
Evaxion Biotech A/S recently presented for the first time its AI model designed to predict patient responses to cancer immunotherapy at the Biomarkers & Precision…
SciRhom Files Clinical Trial Application for First-in-Human Trials of Novel Antibody Against Autoimmune Diseases
SciRhom GmbH recently announced the achievement of a significant milestone in its development pipeline. The company has submitted a Clinical Trial Application for its lead…
Grifols Completes Cohort 1 in Clinical Study of Alpha-1 15%, Evaluating First-in-Human SQ Dosing Option for Patients With Alpha1-Antitrypsin Deficiency
Grifols recently announced it has completed Cohort 1 of its Phase 1/2 study (NCT04722887) evaluating Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% (Alpha-1 15%), a subcutaneous (SQ)…
QSAM Biosciences Signs Term Sheet to be Acquired by Telix Pharmaceuticals
QSAM Biosciences Inc. has signed a non-binding term sheet with Telix Pharmaceuticals (US) Inc., a subsidiary of Telix Pharmaceuticals Limited providing material terms for the acquisition of the company by….
Ocean Biomedical Announces Positive Preclinical Oncology Data for VRON-0300
Ocean Biomedical, Inc. recently announced its joint venture partner, Virion Therapeutics, LLC, recently presented highly compelling preclinical oncology data at the annual meeting of the…
Centogene, University College London & Global Team of Researchers Discover Gene Associated With New Neurodevelopmental Disease Linked to Early Onset Dystonia & Parkinsonism
Centogene N.V. recently announced the discovery of a new form of early onset dystonia and parkinsonism in the context of neurodevelopmental abnormalities associated to the…
Artax Granted Clinical Trial Authorization for AX-158 Phase 2a Psoriasis Study
Artax Biopharma, Inc. recently announced the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA) has granted clinical trial authorization (CTA) to evaluate AX-158 in…
BioXcel Therapeutics Reports Positive Overall Survival Results From Single-Arm, Open-Label Phase 2 Trial
BioXcel Therapeutics, Inc. recently announced positive overall survival (OS) data from its Phase 2 trial of BXCL701, the company's investigational oral innate immune activator, in…
Qualigen Therapeutics Announces First Patient Dosed in Phase 1a Clinical Trial for Treatment of Advanced or Metastatic Solid Tumors
Qualigen Therapeutics, Inc. recently announced the first patient in the Phase 1a clinical trial has been dosed with QN-302, a potential first-in-class, investigational G-Quadruplex (G4)-selective…
Yposkesi Launches AAVelocity, a Plug-and-Play AAV Platform
Yposkesi, SK pharmteco’s European clinical and commercial viral vector manufacturing subsidiary for Cell and Gene Therapies (C>), recently launched AAVelocity, a plug-and-play Adeno-Associated Virus (AAV)…
Kynexis Launched to Advance Precision Therapeutics for Brain Diseases
Kynexis recently launched with €57 million in Series A financing led by Forbion, with participation from Ysios Capital and Sunstone Life Science Ventures. Kynexis will advance its lead….
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).