Bio Platforms
BioXcel Therapeutics Announces First Patients Dosed in SERENITY III Phase 3 Trial for Acute Treatment of Agitation in Adults With Bipolar I or II Disorder or Schizophrenia
BioXcel Therapeutics, Inc., a biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, recently announced the first 13 patients have…
Avectas, CCRM & OmniaBio Expand Their Collaboration to Accelerate the Manufacture of Edited iPSCs Using SOLUPORE Technology
Avectas and CCRM, with its subsidiary OmniaBio Inc., have recently announced an expansion of their collaboration, to enable the development of gene-edited induced pluripotent stem….
Immutep Enters Second Clinical Trial Collaboration Agreement With Merck KGaA & Pfizer for New Combination Study of its First-in-Class LAG-3 Candidate, Eftilagimod Alpha & Avelumab to Treat Urothelial Cancer
New collaboration builds on encouraging clinical data previously reported from INSIGHT-004 in multiple solid tumor indications from the combination of eftilagimod alpha (efti) and avelumab (BAVENCIO)….
Context Therapeutics Nominates CTIM-76 Bispecific Antibody Candidate to Develop Treatment for Claudin 6-Positive Solid Tumors
Context Therapeutics Inc. recently announced the selection of CTIM-76, a T cell-engaging bispecific antibody, as its lead clinical development candidate to target Claudin 6 (CLDN6)…
INmune Bio Focused on Novel Clinical Trial Designs to Advance Next Generation of Alzheimer’s Disease Treatments
INmune Bio, Inc. recently participated in the 15th CTAD Conference in San Francisco November 29 to December 2. “This year’s CTAD is showcasing a potential…
Enlivex Receives Allocetra IND Clearance From the US FDA for Treatment of Patients With Advanced Solid Malignancies
Enlivex Therapeutics Ltd. recently announced the US FDA has cleared an Investigational New Drug (IND) application to study Allocetra in patients with advanced solid malignancies.…
uniQure Announces FDA Approval of First Gene Therapy for Adults With Hemophilia B
Historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy, and generates elevated and sustained factor IX levels….
RxCelerate Acquires Methuselah Health & Launches ProQuant World-Beating Proteomics Service
Next-generation proteomics technology made available to the biotech and pharmaceutical industry through the acquisition of Methuselah Health by leading R&D services provider, RxCelerate….
Evaxion Announces Promising Results From Phase 1/2a Clinical Trial of Personalized DNA Cancer Immunotherapy
Evaxion Biotech A/S recently announced promising clinical data from the Phase 1/2a first-in-human study of its DNA-based cancer immunotherapy, EVX-02….
Attralus & Ossianix Announce Option & License Agreement Using the TXP1 Brain Shuttle for Targeted Delivery of a Pan-Amyloid Removal Therapeutic for Neurodegenerative Disorders
Attralus, Inc. and Ossianix recently announced they have entered into a definitive agreement using Ossianix’ brain shuttle technology to enhance the targeted delivery of novel….
VYNE Therapeutics Announces First Subjects Dosed in Phase 1a/b Clinical Trial Evaluating Pan-BET Inhibitor for the Treatment of Vitiligo
VYNE Therapeutics Inc. recently announced the first subjects have been dosed in a Phase 1a/b clinical trial evaluating VYN201 for the treatment of vitiligo. VYN201…
Editas Medicine Announces Clinical Data Demonstrating Proof of Concept of EDIT-101 From Phase 1/2 BRILLIANCE Trial
Editas Medicine, Inc. recently announced clinical data from the Phase 1/2 BRILLIANCE trial of EDIT-101, an in vivo CRISPR/Cas9 genome editing medicine in a company-sponsored…
Monopar Announces Encouraging Clinical Data From Ongoing Camsirubicin Phase 1b Trial
Monopar Therapeutics Inc. recently released encouraging data from its ongoing Phase 1b open-label clinical trial of camsirubicin in advanced soft tissue sarcoma patients. The data…
Key Patent Issued on Eligo’s Gene-Editing Breakthroughs Applied to Skin Disorders
Eligo Bioscience recently announced the issuance by the USPTO of the first patent in a wide family covering genetic engineering of a key skin microbiome species, Cutibacterium acnes, to support….
ExCellThera Announces Completion of Phase 2 clinical Trials for UM171-Expanded Cell Therapy in High-Risk Blood Cancers
ExCellThera Inc. recently announced the completion of patient enrollment in its initial Phase 2 studies for high-risk leukemias and myelodysplastic syndromes (MDS). ECT-001 Cell Therapy,…
IMUNON Enters Technology Evaluation Agreement With Acuitas to Evaluate Placcine Plasmid DNA With Lipid Nanoparticle Delivery System
Successful combination of technologies will expand the opportunities for IMUNON’s DNA-based modality and broaden applications for the PLACCINE nucleic acid vaccine platform as an alternative to mRNA and protein vaccines….
Akari Therapeutics Granted FDA Rare Pediatric Disease Designation of Nomacopan for the Treatment of Pediatric HSCT-TMA
Akari Therapeutics, Plc recently announced the US FDA has granted the Rare Pediatric Disease Designation to nomacopan for the treatment of pediatric hematopoietic stem cell…
Minoryx & Neuraxpharm Announce Strategic Alliance to Provide New Therapy for Rare CNS Disease
The companies enter into a license agreement for the European rights to leriglitazone, currently under EMA review for the orphan indication X-linked Adrenoleukodystrophy (X-ALD)….
Evonik Wins Pharma Award for Polymer-Based Technology for mRNA Delivery
Evonik Health Care has won the prestigious CPHI Excellence in Pharma Award in the category Packaging and Drug Delivery. The winning innovation is a new…
Immutep Granted New Patents for First-in-Class LAG-3 Candidate, Eftilagimod Alpha in Chemo-Immunotherapy Combination
Immutep Limited recently announced the grant of two new patents (Nos. 7160345 and 10-2441425) titled Combined Preparations for the Treatment of Cancer by the Japanese Patent…
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).