Bio Platforms
Therini Bio Announces First Patient Dosed in Phase 1b Trial of THN391 in Alzheimer’s Disease
Therini Bio, Inc. recently announced the first patient has been dosed in a Phase 1b trial evaluating THN391 for the treatment of Alzheimer's disease (AD).…
Breakthrough Study From UCLA Demonstrates Can-Fite’s Piclidenoson as a Treatment for Vascular Dementia
Can-Fite BioPharma Ltd. recently announced a leading group from University of Los Angeles (UCLA) demonstrates that Piclidenoson showed efficacy in an experimental model of vascular…
Inmagene Biopharmaceuticals Announces Completion of Merger With Ikena Oncology & Concurrent Private Placement of $75 Million
Inmagene Biopharmaceuticals recently announced the completion of its previously announced merger with Ikena Oncology, Inc. The combined company will operate under the name ImageneBio, Inc.…
Candel Therapeutics Receives EMA Orphan Designation for CAN-2409 for the Treatment of Pancreatic Cancer
Candel Therapeutics, Inc. recently announced the European Medicines Agency (EMA) has granted Orphan Designation for CAN-2409 (aglatimagene besadenovec) for the treatment of pancreatic cancer. This…
Rheumatologists Eye AbbVie’s Rinvoq, Roche/Genentech’s Gazyva & Bristol Myers Squibb’s Sotyktu as Front-Runners to Address Persistent Gaps in Systemic Lupus Erythematosus Care
Biologic use in systemic lupus erythematosus (SLE) is steadily rising, with US rheumatologists now treating close to 40% of their moderate-to-severe patients with biologics. Most…
Jupiter Neurosciences Highlights Critical Advancement of JOTROL Over Traditional Resveratrol With Breakthrough Bioavailability & CNS-Targeted Science
Jupiter Neurosciences, Inc. spotlights the scientific and clinical advantages of its proprietary resveratrol platform, JOTROL, over conventional resveratrol formulations. Resveratrol is one of the world’s most…
Avidity Biosciences Receives FDA Breakthrough Therapy Designation for Delpacibart Zotadirsen (del-zota) for the Treatment of DMD in People with Mutations Amenable to Exon 44 Skipping
Avidity Biosciences, Inc. recently announced the US FDA has granted Breakthrough Therapy designation to delpacibart zotadirsen (del-zota) for the treatment of Duchenne muscular dystrophy (DMD)…
Leriglitazone Marketing Authorization Application Submitted for Treatment of Cerebral Adrenoleukodystrophy Has Been Validated by EMA
Minoryx Therapeutics and Neuraxpharm Group recently announced the Marketing Authorization Application (MAA) for Minoryx’s lead candidate leriglitazone (NEZGLYAL) has been submitted to the European Medicines…
Inotrem Unveils Game-Changing Precision Medicine Strategy for Nangibotide Clinical Development
Inotrem recently announced the publication of its precision medicine strategy for its leading drug candidate nangibotide, a first-in-class TREM-1 inhibitor in septic shock. This work…
Polyrizon Reports Successful Intranasal Delivery of PL-14 Allergy Blocker in Latest Study
Polyrizon Ltd. recently announced encouraging preclinical results supporting the performance of its PL-14 Allergy Blocker, part of its proprietary Capture & Contain (C&C) platform. The…
etherna Achieves Major Milestones in its Bio-Reducible LNP Platform
etherna immunotherapies NV recently announced breakthrough innovation in efficient in vivo extrahepatic delivery of mRNA to several key tissues, including to hematopoietic and progenitor stem…
Monte Rosa Therapeutics Announces First Subjects Dosed in Phase 1 Study of a NEK7-Directed Molecular Glue Degrader for the Treatment of Multiple Inflammatory Diseases
Monte Rosa Therapeutics, Inc. recently announced the first subjects have been dosed in a Phase 1 study evaluating MRT-8102, a NEK7-directed MGD being developed for…
Tiziana Life Sciences Announces Immunologic Analysis of Nasal Foralumab in Moderate Alzheimer's Patient
Tiziana Life Sciences, Ltd. recently announced compelling immunologic findings from the treatment of a moderate Alzheimer's Disease (AD) patient with intranasal foralumab. Transcriptional analysis of white…
ImCheck’s Announces EMA Orphan Drug Designation for ICT01 as Treatment for Acute Myeloid Leukemia
ImCheck Therapeutics recently announced the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its lead program, ICT01, a humanized anti-butyrophilin 3A (BTN3A)…
Longeveron Announces Licensing of New Cardiac Selective Induced Pluripotent Stem Cell Technology for Cardiovascular Disease
Longeveron Inc. recently announced it has licensed an issued US patent (12,168,028 B2) for a stem cell technology from the University of Miami. The composition…
UroGen Announces 5-Year Long-Term Extension Study of the OPTIMA II Trial Demonstrates Long-Term Durability of Response to ZUSDURI in Patients With Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer
UroGen Pharma Ltd. recently announced the publication of results of a5-year long-term extension study of the Phase 2b OPTIMA II trial evaluating ZUSDURI (mitomycin) for…
ArriVent’s Topline Pivotal Phase 3 FURVENT Data for Firmonertinib in First-Line NSCLC EGFR Exon20 Insertion Mutations Projected to be Early 2026
ArriVent BioPharma, Inc. recently announced that topline firmonertinib monotherapy data from the global pivotal FURVENT Phase 3 (NCT05607550) study in first-line EGFR exon20 insertion mutant…
Lexicon Announces Patient-Reported Data on Diabetic Peripheral Neuropathic Pain
Lexicon Pharmaceuticals, Inc. recently announced data from a study of patients describing the impact of diabetic peripheral neuropathic pain (DPNP) despite treatment with standard of…
Lisata Therapeutics & WARPNINE Announce iLSTA Trial Enrollment Completion & Provide Preliminary Data Update
Lisata Therapeutics, Inc. and WARPNINE Incorporated recently announced the successful completion of patient enrollment in the Phase 1b/2a iLSTA trial (ACTRN12623000223639) in Australia. The study…
Genascence Announces FDA Grants Regenerative Medicine Advanced Therapy Designation to GNSC-001 for Knee Osteoarthritis
Genascence Corporation recently announced the US FDA has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to GNSC-001, a potential first-in-class gene therapy blocking interleukin…
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).