Bio Platforms
Yumanity Therapeutics Announces Definitive Agreements for Two Strategic Transactions
Yumanity Therapeutics, Inc. recently announced it has entered into definitive agreements for two strategic transactions. The first definitive agreement is an asset purchase….
Assembly Biosciences Initiates Phase 1b Clinical Trial Evaluating Next-Generation Core Inhibitor
Assembly Biosciences, Inc. recently announced the initiation of a Phase 1b trial of its investigational next-generation, highly potent core inhibitor ABI-H3733 (3733). 3733 has demonstrated…
Immunocore Announces Clinical Trial Collaboration With Sanofi
Immunocore Holdings Plc recently announced it has entered into a clinical trial collaboration and supply agreement with Sanofi…..
Evonik Invests $220 Million to Build New Lipid Production Facility for mRNA-based Therapies in the US
Evonik is building a new, highly flexible, global-scale production facility for pharmaceutical lipids in the US. The new plant at Evonik’s Tippecanoe site in Lafayette,…
Teon Therapeutics Announces FDA Acceptance of IND Application for Novel, Oral Immune Checkpoint Inhibitor
Teon Therapeutics recently announced the acceptance by the US FDA of Teon’s Investigational New Drug (IND) application for the study of TT-816. TT-816 is a novel, oral cannabinoid CB2 receptor….
PDS Biotechnology Granted FDA Fast Track Designation for Lead Candidate
PDS Biotechnology Corporation recently announced the US FDA has granted Fast Track designation to PDS0101 in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab). PDS0101 in combination…
Biogen & Samsung Bioepis’ & Biogen's BYOOVIZ (ranibizumab-nuna) Launches in the US
Samsung Bioepis Co., Ltd. and Biogen Inc. recently announced BYOOVIZ (ranibizumab-nuna), a biosimilar referencing LUCENTIS (ranibizumab), has been launched….
Vaxart Reports Positive Preliminary Data from Phase 1b Trial of its Oral Norovirus Vaccine Candidates in Elderly Adults
Results of the trial show a robust immune response across all doses, with a dose-dependent production of IgA antibody secreting cells (ASCs), consistent with previous…
PLATFORM TECHNOLOGY - Confident Silence: Delivering on the Promise of siRNA Therapies
Giles Campion, MD, says while the liver offers plenty of fertile ground for developing siRNAs therapies to treat many diseases, many disease-related genes are not highly expressed in the liver. For this reason, the next great challenge in the field is delivering siRNA to tissues outside the liver.
EXECUTIVE INTERVIEW - BIOVECTRA: mRNA & the Future of Pharma
Marc Sauer, PhD, discusses his new position, growth at BIOVECTRA, and how he believes mRNA technologies can best be leveraged to improve the lives of patients.
CordenPharma Increases xRNA-based Capabilities with a Strategic Investment in LNP Formulation Services at its Caponago Injectable Facility
CordenPharma has recently increased its xRNA capabilities at its sterile injectable facility in Caponago, Italy. The company reports that it is investing over €10M in new Lipid Nanoparticle (LNP) formulation,….
BIORESORBABLE POLYMERIC MATRICES - Convergence of Materials Science & Drug Development to Treat Challenging ENT Diseases
Maria Palasis, PhD, and Robert Kern, MD, review how The XTreo platform represents a unique and powerful convergence of materials science, drug development, and formulation chemistry, enabling the local delivery of medication to anatomical spaces not accessible by conventional therapeutic approaches.
EXECUTIVE INTERVIEW - Catalent: Developing & Delivering Billions of Doses of Drugs Every Year
Alessandro Maselli, President and Chief Operating Officer of Catalent, talks about his company’s unique approach to drug development partnerships as he prepares to become the company’s new President and Chief Executive Officer on July 1, 2022.
Denali Therapeutics & Biogen Announce Initiation of Phase 2b Study of LRRK2 Inhibitor in Parkinson’s Disease
Denali Therapeutics Inc. and Biogen Inc. recently announced that dosing has commenced in the global Phase 2b LUMA study to evaluate the efficacy and safety…
Agenus Advances Portfolio With 6 Clinical Collaborations
Agenus Inc. recently announced it has entered into three new clinical collaborations with Targovax, Oxford BioTherapeutics, and Immunogenesis, doubling its current number of….
Purple Biotech Presents Phase 1 Interim Monotherapy Data for NT219 Demonstrating Encouraging Safety & Efficacy Profile
Purple Biotech Ltd. recently announced positive interim safety and efficacy data from the Phase 1 study of NT219 in adults with advanced solid tumors…..
Yingli Pharma Doses First US Patient in Phase 1 Study of YL-13027 for Patients With Advanced Solid Tumors
Yingli Pharma US, Inc. recently announced the first patient has been dosed in its Phase 1 clinical study evaluating YL-13027, a potent and selective TGFβ…
Oncotelic Therapeutics Receives FDA Clearance for Phase 2 Clinical Trial of OT-101/Pembroluzimab Combination for Mesothelioma (M201)
Oncotelic Therapeutics, Inc. recently announced the clearance of the Phase 2 clinical trial protocol for mesothelioma after filing the protocol to the US FDA. Oncotelic…
Crinetics Pharmaceuticals Reports Positive Top-line Results Including Strong Adrenal Suppression from CRN04894 Phase 1 Study Multiple-Ascending Dose Cohorts
Crinetics Pharmaceuticals, Inc. recently announced positive results from the multiple-ascending dose (MAD) portion of a first-in-human Phase 1 clinical study of CRN04894, the company's first-in-class, investigational,…
Pharmazz Inc. Announces Positive Results of Phase 3 Clinical Trial Evaluating Sovateltide as a Treatment for Acute Cerebral Ischemic Stroke
Pharmazz, Inc. recently announced positive topline results of its Phase 3 clinical trial evaluating sovateltide as a treatment for acute ischemic stroke. The data at 90 days showed….
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).