Bio Platforms
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…
Ocuphire Pharma Enters Global License Agreement for Development & Commercialization of Nyxol Eye Drops for Reversal of Mydriasis, Presbyopia & Night Vision Disturbances
Ocuphire Pharma, Inc. recently announced it has concluded an exclusive license agreement with FamyGen Life Sciences, Inc. for the development and commercialization of Nyxol across…
Insilico Medicine Signs Strategic Research Collaboration With Sanofi Worth up to $1.2 Billion
Agreement includes aggregate upfront and target nomination fees of up to $21.5 million for up to 6 targets, and additional R&D and sales milestones plus tier-based mid-single to low double-digit royalties….
Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Clinical Trial of IMCIVREE (setmelanotide) in Bardet-Biedl Syndrome
Rhythm Pharmaceuticals, Inc. recently announced that previously disclosed results from a Phase 3 clinical trial that evaluated IMCIVREE (setmelanotide), an MC4R agonist, in patients with…
SomaLogic Teams With Molecular Genomics to Bring the 7,000-Plex SomaScan Platform to Asia
SomaLogic, Inc. recently announced Singapore-based Molecular Genomics will be the first site in Asia to offer the 7,000-plex SomaScan Assay and provide SomaScan data to…
Fate Therapeutics Announces Exercise by ONO Pharmaceutical of Option to HER2-targeted CAR T-Cell Product Candidate for Solid Tumors
Fate Therapeutics, Inc. recently announced that ONO Pharmaceutical Co., Ltd. (ONO) has exercised its option to FT825/ONO-8250, a multiplexed-engineered, iPSC-derived, chimeric antigen receptor (CAR) T-cell…
Actym Therapeutics Reports Preclinical Data Demonstrating Ability to Generate Anti-Tumor Immunity
Actym Therapeutics recently announced an abstract highlighting detailed in vivo and in vitro preclinical data of its lead candidate, ACTM-838, was accepted for presentation at the Society…
Exelixis & Catalent Enter New License Agreement for Three Antibody-Drug Conjugate Programs With the Potential to Accelerate Exelixis’ Biologics Pipeline
Exelixis, Inc. and Catalent, Inc. recently announced a new license agreement under which Catalent’s Redwood Bioscience subsidiary will grant Exelixis an exclusive license to three target programs with….
Servier & OSE Immunotherapeutics Announce Completion of Patient Enrollment in the Phase 2a Clinical Trial of OSE-127/S95011 in Primary Sjögren’s Syndrome
Servier and OSE Immunotherapeutics SA recently announced the completion of patient enrollment in the Phase 2a clinical trial evaluating the efficacy and safety of monoclonal…
APEIRON Respiratory Therapies Announces Positive Results From Phase I Trial of Inhaled APN01
APEIRON Respiratory Therapies GmbH recently announced positive final results from a Phase 1 clinical trial of inhaled APN01 (alunacedase alfa), a soluble recombinant version of…
Inozyme Pharma Announces First Self-Administration of INZ-701 in Ongoing ENPP1 Deficiency Phase 1/2 Clinical Trial
Inozyme Pharma, Inc. recently announced the first self-administration of INZ-701 in the open-label Phase 2 extension portion of the ongoing Phase 1/2 clinical trial of INZ-701…
Jounce Therapeutics Earns Clinical Milestone Payment Under Exclusive License Agreement With Gilead Sciences
Jounce Therapeutics, Inc. recently announced earning a $15-million clinical milestone payment from Gilead Sciences, Inc. under the exclusive license agreement for….
Acumen Publishes Phase 1 Trial Design & Clinical Development Plan for an Anti-Amyloid Beta Oligomer Antibody for Alzheimer’s Disease
Acumen Pharmaceuticals, Inc. recently announced it published its development rationale and clinical development plan for ACU193, the first clinical-stage monoclonal antibody that selectively targets toxic soluble….
Rhythm Pharmaceuticals Announces Setmelanotide Granted Breakthrough Therapy Designation for Hypothalamic Obesity
Rhythm Pharmaceuticals, Inc. recently announced that setmelanotide received Breakthrough Therapy Designation from the US FDA for the treatment of hypothalamic obesity. The FDA's Breakthrough Therapy…
Processa Successfully Identifies Next-Generation Capecitabine Dosage Regimens for Phase 2B Trial
Processa Pharmaceuticals, Inc. recently announced positive results from its ongoing Next-Generation Capecitabine (NGC) Phase 1B trial. The data collected has allowed Processa to estimate the…
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).