Bio Platforms
Artelo Biosciences Reports Positive Preclinical Results for Lead Program in Cancer-Related Cachexia
Artelo Biosciences, Inc. recently announced ART27.13, a peripherally selective G-Protein Coupled Receptor (GPCR) full agonist currently in clinical development for cancer-related anorexia, shows promising preclinical…
ChemoCentryx Reports PK & PD Results From Ongoing Phase 1 Trial of Orally Administered PD-L1 Inhibitor
ChemoCentryx, Inc. recently announced the presentation of preclinical data and initial pharmacokinetic (PK) and pharmacodynamic (PD) data from the ongoing Phase 1 clinical study of…
Inozyme Pharma Provides Update for Phase 1/2 Clinical Trials in ABCC6 Deficiency & ENPP1 Deficiency
Inozyme Pharma, Inc. recently announced the first patient has been dosed in its Phase 1/2 clinical trial of INZ-701 in adult patients with ABCC6 Deficiency…
Caladrius Biosciences Treats First Patient in the Phase 1b Trial for the Treatment of Diabetic Kidney Disease
Caladrius Biosciences, Inc. recently announced the first patient has been treated in its Phase 1b open-label, proof-of-concept study of CLBS201 for the treatment of diabetic…
LIXTE Biotechnology Announces Preclinical Results of its Collaboration With the Netherlands Cancer Institute, Revealing Striking Anti-Cancer Activity
LIXTE Biotechnology Holdings, Inc. recently announced Professor René Bernards, Netherlands Cancer Institute (NKI), Amsterdam, presented new data from promising drug combinations of LIXTE’s lead clinical…
Venomtech Announces New Drug Development Collaboration With Charles River
Venomtech is collaborating with Charles River Laboratories, International Inc. to help drug developers explore venom-derived compounds for a wide range of….
WHITE PAPER: Controlled Synthesis of Lipid Nanoparticles Using the Automated Nanoparticle System
Particle Works’ leading microfluidic technology offers a unique automated nanoparticle generation platform for the production of homogeneous, controllable, and reproducible LNPs….
Matinas BioPharma & BioNTech Announce Exclusive Research Collaboration to Evaluate Novel Delivery Technology for mRNA-based Vaccines
Matinas BioPharma and BioNTech SE recently announced they have entered into an exclusive research collaboration to evaluate the combination of mRNA formats and….
Krystal Biotech Announces Home Dosing in B-VEC Open Label Extension Study
Krystal Biotech, Inc. recently announced it plans to offer patients with dystrophic epidermolysis bullosa (DEB), who are enrolled in the GEM-3 open label extension study,…
VectorBuilder to Expand With $500-Million Gene Delivery Research & Manufacturing Campus
VectorBuilder Inc. recently announced the construction of a new R&D and manufacturing center in Guangzhou, China. The Gene Delivery Research and Manufacturing Campus will significantly expand…
Context Therapeutics Announces Encouraging Preclinical Data From Two Programs
Context Therapeutics Inc. recently announced encouraging preclinical data from two pipeline programs, including in vivo combination and immunomodulation data evaluating onapristone extended release (ONA-XR), the company’s…
Enteris BioPharma Highlighted as Game Changer in Special Feature on Innovative Technologies for Improving Drug Solubility & Bioavailability
Enteris BioPharma, Inc., a biotechnology company developing innovative drug products based on its proprietary delivery technologies, and a wholly-owned subsidiary of SWK Holdings Corporation (Nasdaq:…
FREEZE-DRYING MICROSCOPY - Unravelling the Complexities of Freeze-Drying Pharmaceuticals With Advanced Microscopy Techniques
Paul Matejtschuk, PhD, Prof Yvonne Perrie, and Robert Gurney, PhD, say FDM is being used to analyze the most challenging formulations that require sophisticated stabilization methods. By altering and adapting the three main freeze-drying stages to specific formulations, researchers are providing the tools needed for the pharma industry to improve its drug development processes and maintain drug stability when scaling up to production.
Catalent Acquires Facility in Oxfordshire to Expand Biologics Capabilities in the UK & Across Europe
Catalent recently announced the acquisition from Vaccine Manufacturing and Innovation Centre UK Limited (VMIC Ltd.) of a biologics development and manufacturing facility currently under….
Rhythm Pharmaceuticals Optimizes Design of EMANATE & DAYBREAK Clinical Trials to Advance Setmelanotide for Rare Genetic Diseases of Obesity
Rhythm Pharmaceuticals, Inc. recently announced modifications intended to optimize its Phase 3 EMANATE and ongoing Phase 2 DAYBREAK trials to focus on rare patient populations,…
Medidata & Celsion Present Findings on Use of Synthetic Control Arm to Estimate Treatment Effect in Ovarian Cancer Trial
Celsion Corporation and Medidata recently announced they presented their findings on the use of a Synthetic Control Arm (SCA) in a completed Phase Ib dose-escalating study of GEN-1 in the neoadjuvant treatment of….
CELL & GENE THERAPY - State of the Industry – Where is C> Headed?
Bill Vincent believes if the industry wants to keep moving up the steep growth curve, then capacity and raw materials must be readily available. The innovations in therapies, platforms, and processes will all come with time, money, and increasing availability of resources.
Nurix Therapeutics Doses First Patient in Phase 1 Clinical Trial of Drug-Enhanced Cell Therapy for the Treatment of Patients With Solid Tumors
Nurix Therapeutics, Inc. recently announced the first patient has been dosed in its Phase 1 clinical trial of DeTIL-0255, a drug-enhanced tumor infiltrating lymphocyte therapy and….
Verrica Pharmaceuticals Announces First Patient Dosed in Phase 2 Study for Potential First-in-Class Oncolytic Peptide-Based Immunotherapy
Verrica Pharmaceuticals Inc. recently announced the first patient has been dosed in the company’s Phase 2 trial of LTX-315, a potential first-in-class oncolytic peptide, for the….
Processa Pharmaceuticals Announces First Patient Enrolled in Phase 2A Trial for the Treatment of Gastroparesis
Processa Pharmaceuticals, Inc. recently announced it has enrolled its first patient in the Phase 2A clinical trial of PCS12852 in patients with moderate-to-severe gastroparesis. Gastroparesis…
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).