Bio Platforms
4DMT Announces FDA Clearance of IND Application for Genetic Medicine for the Treatment of Geographic Atrophy
4D Molecular Therapeutics recently announced the US FDA clearance of the Investigational New Drug Application (IND) for 4D-175, an R100 vector-based intravitreal genetic medicine, for…
IMUNON Announces Database Lock for Phase 2 OVATION 2 Study With IMNN-001 in Advanced Ovarian Cancer
IMUNON, Inc. recently announcef database lock for its Phase 2 OVATION 2 Study evaluating the safety and efficacy of IMNN-001 in patients with advanced ovarian…
Bio-Rad Extends Range of Anti-Biotherapeutic Antibodies to Accelerate Antibody Drug Development
Bio-Rad Laboratories, Inc. recently launched four new anti-idiotypic antibodies to the following drugs: atezolizumab (Tecentriq), avelumab (Bavencio), obinutuzumab (Gazyvaro), and ocrelizumab (Ocrevus), extending its range…
Medigene AG Announces Lead Selection for MDG2021, Expanding TCR-T KRAS Library Targeting Solid Tumors
Medigene AG recently announced it has selected its lead candidate for MDG2021, a T cell receptor engineered T cell (TCR-T) therapy targeting Kirsten rat sarcoma…
Neurona Therapeutics Receives FDA’s Regenerative Medicine Advanced Therapy Designation for NRTX-1001 in Focal Epilepsy
Neurona Therapeutics recently announced the US FDA has granted the company’s lead product candidate, NRTX-1001, the RMAT expedited program designation for drug-resistant mesial temporal lobe…
WACKER & CordenPharma Enter Pandemic Readiness After Successful Expansion & Qualification
WACKER and CordenPharma recently announced their joint consortium has officially entered the pandemic readiness state as of June 2024. Following a successful expansion and qualification…
Alvotech & Advanz Pharma Sign Commercialization Agreement
Alvotech and Advanz Pharma recently announced they have entered into an exclusive partnership agreement regarding the supply and commercialization of Alvotech’s proposed biosimilar to Eylea…
Arch Biopartners Announces University of Calgary Ethics Approval to Proceed With Phase 2 Trial for LSALT Peptide
Arch Biopartners Inc. recently announced the University of Calgary’s Conjoint Health Research Ethics Board (CHREB) has approved the Phase 2 trial for LSALT peptide targeting…
Zymeworks Announces FDA Clearance of IND Application for Novel 2+1 T-Cell Targeting Bispecific Antibody For Mesothelin-Expressing Cancers
Zymeworks Inc. recently announced the US FDA has cleared the investigational new drug (IND) application for ZW171, a novel 2+1 T-cell targeting bispecific antibody for…
Barinthus Bio Announces Strategic Pipeline Prioritization Following Positive Interim Data
Barinthus Biotherapeutics plc recently announced plans to prioritize its pipeline to focus on the development of VTP-300 in CHB and VTP-1000 in celiac disease. The…
Greenwich LifeSciences Announces $2.5-Million Private Placement
Greenwich LifeSciences, Inc. recently announced it has entered into a securities purchase agreement with its Chief Executive Officer, Snehal Patel, for the purchase and sale…
LIXTE Biotechnology Announces Collaboration on New Colon Cancer Clinical Trial
LIXTE Biotechnology Holdings, Inc. recently announced a collaboration with Roche and the Netherlands Cancer Institute (NKI) to conduct a new clinical trial in immune therapy unresponsive…
Evaxion Publishes Data, Showing 67% Objective Response Rate in Metastatic Melanoma for AI-Designed Personalized Cancer Vaccine
Evaxion Biotech A/S recently announced publication of data from its Phase 1 dose escalation study of its lead personalized cancer vaccine candidate, EVX-01, for metastatic…
Grifols’ Biotest Receives FDA Approval for Innovative Immunoglobulin
Grifols recently announced Biotest, a Grifols Group company, has received approval from the US FDA for Yimmugo, an innovative intravenous immunoglobulin (Ig) therapeutic, to treat primary…
Biosplice Announces Collaboration With Novo Nordisk in Diabetes
Biosplice Therapeutics, Inc. recently entered a collaboration with Novo Nordisk to develop drug candidates for the treatment of diabetes. This agreement builds upon Biosplice’s innovative…
CERo Therapeutics Completes IND-Enabling Activities Following Successful Manufacturing Runs for Lead Compound
CERo Therapeutics Holdings, Inc. recently announced it successfully executed pre-IND manufacturing activities for its lead therapeutic, CER-1236. The completion of multiple manufacturing runs is required…
Lisata Therapeutics Announces Full Enrollment of Pancreatic Cancer Cohort of CENDIFOX Trial
Lisata Therapeutics, Inc. recently announced the successful completion of patient enrollment for the pancreatic cancer cohort in the Phase 1b/2a CENDIFOX trial. This investigator-initiated trial,…
FDA Accepts Tiziana Life Sciences Fast Track Designation Submission for Treatment of Multiple Sclerosis
Tiziana Life Sciences, Ltd. recently announced it has received acceptance of its submission for intranasal foralumab to receive Fast Track Designation approval for the treatment…
Belite Bio Receives Sakigake (Pioneer Drug) Designation of Tinlarebant for Stargardt Disease
Belite Bio, Inc recently announced its lead pipeline, Tinlarebant, has been granted Sakigake Designation by the Ministry of Health, Labour and Welfare in Japan (MHLW)…
Enterprise Therapeutics Publishes Preclinical Profile of a Novel Inhaled ENaC Blocker
Enterprise Therapeutics Ltd recently announced the publication of a peer reviewed study in the Journal of Cystic Fibrosis. The paper describes low doses of 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).