Bio Platforms
Pheno Therapeutics Announces Worldwide License From UCB to a Novel Program for Neurodegenerative Disease
Pheno Therapeutics Limited. recently announced it has entered into an exclusive worldwide license agreement with UCB. The agreement grants Pheno Therapeutics access and rights to….
Emerald Health Pharmaceuticals Receives Safety Review Committee Approval to Proceed to Final Cohorts of EHP-101 Phase 2a Systemic Sclerosis Trial
Emerald Health Pharmaceuticals Inc. recently received a positive safety review following a pre-specified interim analysis by the Safety Review Committee (SRC) of its Phase 2a…
BioXcel Therapeutics Announces Promising Top-Line Results from Phase 2 Trial of BXCL701 in Aggressive Form of Rare Prostate Cancer
BioXcel Therapeutics, Inc. recently announced promising top-line data from its Phase 2 trial of BXCL701, the company's investigational, oral innate immune activator, in combination with…
Ajinomoto & Exelixis Enter License Agreement to Discover & Develop Novel Antibody-Drug Conjugates for the Treatment of Cancer
Ajinomoto Co., Inc. recently announced a license agreement with Exelixis, Inc. to incorporate AJICAP, Ajinomoto Co.’s proprietary site-specific bioconjugation and linker technologies, in the development of….
SAB Biotherapeutics Successfully Concludes IND-Enabling GLP Toxicology Study for Novel Immunotherapeutic for Type 1 Diabetes
SAB Biotherapeutics recently announced the successful completion of an IND-enabling GLP-tox study for SAB-142, further progressing the therapeutic as a way to prevent and/or delay onset…
Absci First to Create & Validate De Novo Antibodies With Zero-Shot Generative AI
This breakthrough unlocks the potential to accelerate time to clinic by over 50% and increase probability of success in the clinic….
Atavistik Bio Announces Collaboration With Plex Research to Enrich the Informatics Capabilities of its AMPS Platform & Accelerate the Discovery of Novel Small Molecule Therapeutics
Atavistik Bio recently announced it has entered into a collaboration agreement with Plex Research, a company providing a novel artificial intelligence (AI)-powered drug discovery platform.…
Vaccinex Announces First Patient Dosed with Anti-CCR8 Antibody Licensed to Surface Oncology
Vaccinex, Inc. recently announced its licensee, Surface Oncology dosed the first patient in its Phase 1/2 clinical study investigating SRF114, an antibody discovered using Vaccinex’s…
Imbria Enrolls First Patient in the Phase 2 IMPROVE-DiCE Clinical Trial Evaluating Ninerafaxstat in Patients With Heart Failure With Preserved Ejection Fraction
Imbria Pharmaceuticals, Inc. recently announced the enrollment of the first patient in Part 2 of the IMPROVE-DiCE Phase 2 clinical trial of ninerafaxstat in patients…
Centogene & Denali Therapeutics Extend World’s Largest Observational Study on Parkinson’s Disease Genetics
Centogene N.V. recently announced it has extended the Rostock International Parkinson's Disease (ROPAD) Study to recruit and genetically test additional patients over the next few…
Celltrion & Rani Therapeutics Partner on Development of Oral Monoclonal Antibody Treatment
Rani Therapeutics Holdings, Inc. recently announced it has partnered with Celltrion for the development of RT-111, an orally administered ustekinumab biosimilar….
Transgene Receives Approval to Start a Phase 1 Trial of Novel IL-12-Armed Oncolytic Virus Given by Intravenous Administration
Transgene recently announced it has received clinical trial application (CTA) approval from the French National Agency for the Safety of Medicines and Health Products (ANSM) to proceed with a Phase 1 clinical trial of TG6050, a novel oncolytic….
Frontera Therapeutics Doses First Patient in Phase 1 Clinical Trial for Gene Therapy for the Treatment of Leber Congenital Amaurosis-2
Frontera Therapeutics recently announced it has dosed the first patient in a Phase 1 clinical trial of its lead gene therapy program, FT-001, for the…
Sarepta & Catalent Expand Strategic Manufacturing Partnership With Commercial Supply Agreement for Duchenne Muscular Dystrophy Gene Therapy Candidate
Catalent, Inc. and Sarepta Therapeutics, Inc. recently announced the signing of a commercial supply agreement for Catalent to manufacture delandistrogene moxeparvovec (SRP-9001), Sarepta’s most advanced…
SAB Biotherapeutics Novel DiversitAb Platform Proven to Develop Anti-Idiotype Antibodies to Help Treat Autoimmune Diseases
SAB Biotherapeutics recently announced results from a project in collaboration with global biotechnology leader CSL confirming that SAB’s DiversitAb platform can generate functional fully human anti-idiotype polyclonal….
Ovid Therapeutics Dosed Healthy Volunteers With OV329 in Phase 1 Trial
Ovid Therapeutics Inc. recently dosed healthy volunteers with OV329 in late December 2022, as part of a Phase 1 study. The company is conducting the…
IMUNON Enters Collaborative Research Agreement With The Wistar Institute’s Vaccine & Immunotherapy Center to Research IMUNON’s PLACCINE Vaccine Platform
IMUNON, Inc. and The Wistar Institute recently announced the signing of a first collaborative research agreement to research and develop new vaccine formulations utilizing IMUNON’s PLACCINE modality for the….
Purple Biotech & Mor Research Applications Announce Research Collaboration to Identify Promising Investigational Oncology Treatments
Purple Biotech Ltd. recently announced a research collaboration with Mor Research Applications that provides Purple Biotech first access to early stage oncology product candidates owned by….
HRA Pharma Rare Diseases Registers the first Cushing’s Syndrome Treatment in Colombia
HRA Pharma Rare Diseases, an affiliate of HRA Pharma, a Perrigo Company, recently announced the registration of the first ever treatment for Cushing’s syndrome, Metopirone,…
Oncorus Announces Research Collaboration With Daewoong Pharmaceutical for the Development of Lipid Nanoparticle Formulations for mRNA Drug Candidates
Research collaboration leverages Oncorus’ proprietary ionizable amines, PEG lipids, LNP formulations, process development and manufacturing together with Daewoong’s mRNA expertise….
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).