Bio Platforms
ImmunityBio Expands Vaccine Program to Accelerate Use of “Mix-and-Match” Technologies in Developing & Manufacturing Next-Generation COVID-19 Vaccines
ImmunityBio, Inc. recently announced the expansion of the company’s cancer and infectious disease vaccine programs to include self-amplifying self-adjuvating RNA (SASA-RNA), recombinant protein vaccine candidates, and potent adjuvant formulations that….
Artizan Biosciences & Biohaven Therapeutics Announce Therapeutic Development Program in Parkinson’s Disease
Artizan Biosciences, Inc. and Biohaven Therapeutics Ltd. recently announced the companies are initiating a therapeutic discovery and development program in Parkinson’s disease (PD), to exploit recent….
Verrica Pharmaceuticals Announces FDA Acceptance of its IND Application for Potential First-in-Class Oncolytic Peptide-Based Immunotherapy for the Treatment of Basal Cell Carcinoma
Verrica Pharmaceuticals Inc. recently announced the US FDA has accepted its Investigational New Drug Application (IND) for LTX-315, a potential first-in-class oncolytic peptide, for the…
Molecular Templates Announces Fast Track Designation Granted by FDA for MT-6402
Molecular Templates, Inc. recently announced the US FSA has granted Fast Track Designation for MT-6402 for the treatment of patients with advanced non-small cell lung…
First Subject Dosed in Biosplice Therapeutics Phase 1b Clinical Trial in Oncology
Biosplice Therapeutics, Inc. recently announced the dosing of the first subject in a Phase 1b, open-label, multi-center, dose-escalation, dose expansion clinical trial of cirtuvivint in…
Denali Therapeutics Announces Strategic Partner Takeda Exercises Option to Co-Develop & Co-Commercialize Brain-Penetrant Progranulin Replacement Therapy
Denali Therapeutics Inc. recently announced its strategic partner Takeda Pharmaceutical Company Limited has exercised an option, pursuant to an existing collaboration agreement between the two…
Alzheimer’s Disease Preventative Nasal Vaccine to be Tested at Boston's Brigham & Women’s Hospital
Boston's Brigham and Women’s Hospital recently announced it would test a nasal vaccine for Alzheimer’s disease. The move, the hospital said in a press release, represents the culmination of…
SOTIO Expands Its Antibody-Drug Conjugate Pipeline With Exclusive Collaboration & License Agreement With LegoChem Biosciences
SOTIO Biotech recently announced an exclusive, target-specific license and option agreement with LegoChem Biosciences Inc in which SOTIO will obtain rights to deploy LCB’s ADC technology for up to….
Vaxart Announces Publication of Complete Data From Preclinical COVID-19 Oral Vaccine Hamster Challenge Study
Vaxart, Inc. recently announced the Journal of Infectious Diseases has published complete data from Vaxart’s preclinical Hamster Challenge Study. The study shows Vaxart’s COVID-19 oral…
Moderna Announces Positive Data From Phase 2 Study of mRNA VEGF-A Therapeutic in Patients Undergoing Coronary Artery Bypass Grafting Surgery
Moderna, Inc. recently announced positive data from the AstraZeneca-led Phase 2 (EPICCURE) study evaluating the use of an mRNA therapeutic that encodes for vascular endothelial…
Inozyme Pharma Announces Dosing of First Patient in First-in-Human Clinical Trial of INZ-701 in Patients With ENPP1 Deficiency
Inozyme Pharma, Inc. recently announced the first patient has been dosed in its first-in-human Phase 1/2 clinical trial of INZ-701 in adult patients with ENPP1…
Emergex Announces Approval to initiate Phase 1 Clinical Trial of Its Next-Generation COVID-19 Vaccine Candidate
Emergex Vaccines Holding Limited recently announced it has received the necessary regulatory approvals to initiate a Phase 1 clinical trial to evaluate the safety and…
Harpoon Therapeutics Presents Data on its TriTAC-XR Platform
Harpoon Therapeutics, Inc. recently presented a poster with preclinical data on its TriTAC-XR T cell engager platform at the 36th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) in Washington, DC…..
Vaccitech’s VTP-300 Was Well-Tolerated & Induced T cells Against All Targeted HBV Antigens in Both Healthy Volunteers & Patients With Chronic HBV Infection in Interim Analyses
Vaccitech plc recently announced results from ongoing Phase 1 and Phase 1b/2a clinical trials of VTP-300, an immunotherapy candidate in development for the treatment of…
Oculis Announces Patient Dosing in its First Phase 3 Study for Investigational Topical Eye Drop Treatment for Diabetic Macular Edema
Oculis S.A. recently announced the first patients have been dosed in its Phase 3 DIAMOND trial evaluating the efficacy and safety of OCS-01 in Diabetic…
Intensity Therapeutics Reports Use of INT230-6 Alone or in Combination With Ipilimumab Shows Evidence of Direct Tumor Necrosis & Promising Overall Survival Results in Adult Subjects With Metastatic Sarcomas
Intensity Therapeutics, Inc. recently announced that data from its open-label Phase 1/2 study of novel lead asset, INT230-6, as a monotherapy or in combination with ipilimumab in…
Oncorus Announces Exclusive Licensing Agreement With Gaeta Therapeutics for Use of Locally Delivered Interleukin-12 Via Oncolytic Viral Expression in Combination With Immune Checkpoint Inhibitors
Oncorus, Inc. recently announced it has signed an exclusive licensing agreement with Gaeta Therapeutics Ltd., related to the use of locally delivered Interleukin-12 (IL-12) via…
Exavir Therapeutics Announces Preclinical Data Demonstrating Complete Elimination of HIV From Human Cells With LNP-Delivered Tat-Targeted CRISPR-Cas9
Exavir Therapeutics recently announced the publication of preclinical data for XVIR-TAT, Exavir’s proprietary gene editing program in early preclinical development as a potential cure for…
Finch Therapeutics Announces Positive Topline Data From Phase 2 PRISM-EXT Open-Label Study of Microbiome Therapy for Recurrent C. diff.
Finch Therapeutics Group, Inc. recently announced positive topline results from PRISM-EXT, an open-label extension of the company’s PRISM3 Phase 2 placebo-controlled trial evaluating CP101 for…
Mustang Bio Announces Exclusive Worldwide License Agreement With Leiden University Medical Centre for Clinical-Stage Lentiviral Gene Therapy With Curative Potential for RAG1 Severe Combined Immunodeficiency
Mustang Bio, Inc. recently announced that the company has executed an exclusive license agreement with Leiden University Medical Centre (LUMC) for a first-in-class ex vivo lentiviral gene therapy for 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).