Bio Platforms
BioAegis Therapeutics Completes Enrollment in Phase 1b/2a Dose-Ranging Safety Study
BioAegis Therapeutics Inc. recently announced it has completed patient enrollment in its Phase 1b/2a study of recombinant plasma gelsolin (rhu-pGSN) as adjunctive therapy for patients…
Altimmune Announces Positive Results From NasoVAX Extension Study
Altimmune, Inc. recently announced additional positive data from a Phase 2 extension study of its NasoVAX intranasal influenza vaccine candidate. Data from this study demonstrated…
Enterome Announces Research Collaboration With Dana-Farber Cancer Institute
Enterome recently announced it has entered into a research and development collaboration with Dana-Farber Cancer Institute to evaluate and develop gut microbiome-derived antigens as potential cancer immunotherapies.
Imugene Announces First Patient Dosed in Open-Label Phase 2 Study
Imugene Limited recently announced dosing of the first patient in an open-label, randomized multi-centre Phase 2 study with its HER-Vaxx (IMU-131) cancer vaccine for the…
BioXcel Therapeutics Announces Addition of Merck KGaA & Pfizer to Clinical Collaboration With Nektar
BioXcel Therapeutics, Inc. recently announced the addition of Merck KGaA, Darmstadt, Germany, which operates its biopharmaceutical business as EMD Serono in the US and Canada,…
Aptose Biosciences & CrystalGenomics Announce Issuance of Patent for Oral Small Molecule
Aptose Biosciences Inc. recently announced the Australian Patent Office (APO) has issued Australian Patent No. 2013371146 for CG-806, a first-in-class, highly potent oral small molecule…
ContraFect Announces $6.94 Million in Funding from CARB-X
ContraFect Corporation recently announced that the Combating Antibiotic Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) has awarded the company up to $6.94 million in non-dilutive funding to…
Lipomedix Announces Initiation of Phase 1b Trial
Lipomedix recently announced the initiation of a Phase 1b multicenter trial (LIPORAD-2018) to identify the optimal dose, safety, and efficacy of its lead compound Promitil…
Apellis Resumes Dosing in Its Phase 3 Program for Patients With Geographic Atrophy
Apellis Pharmaceuticals Inc. recently announced that with the agreement of the independent safety monitoring committee for the company’s Phase 3 clinical program for APL-2 in…
Procarta Announces Significant Investment for New Class of Antibiotics
Procarta Biosystems recently announced $1.7 million of new funding from the Novo Holdings REPAIR Impact Fund to support development of novel therapies to combat antimicrobial resistance (AMR).
Biogen Announces Agreement to Acquire Nightstar Therapeutics
Biogen recently announced it has entered into an agreement to acquire Nightstar Therapeutics, a clinical-stage gene therapy company based in London, United Kingdom, which is focused on adeno-associated virus (AAV) treatments for inherited retinal disorders.
RNA EDITING - New Editing Technology Enables Body to Repair its Own RNA
Daniel de Boer explains how the Axiomer technology, a powerful RNA-editing technique that enables the body to repair itself, is being developed as a next-generation therapeutic option for genetic disorders.
NANOPARTICLE DELIVERY - Ultra-Small Particles Offer Big Hope in Cancer Theranostics
Kai Ma, PhD, Ulrich Wiesner, and Michelle Bradbury, MD, PhD, believe ultra-small sub-10 nm particles hold unique properties and represent an emerging area of investigation for biomedical applications.
Sarepta Therapeutics Announces Positive & Robust Expression & Biomarker Data
Sarepta Therapeutics, Inc. recently announced positive results from three Limb-girdle muscular dystrophy (LGMD) Type 2E clinical trial participants who received MYO-101. MYO-101 is a novel…
AKCEA-APO(a)-LRx Advances as Novartis Exercises Option to License
Akcea Therapeutics, Inc. recently announced that Novartis has exercised its option to license AKCEA-APO(a)-LRx, a drug to treat patients with elevated levels of lipoprotein(a), or Lp(a), and established cardiovascular disease (CVD).
BrainStorm Announces First Contracted US Clinical Site for Phase 2 Progressive MS Study
BrainStorm Cell Therapeutics Inc. recently announced Cleveland Clinic as the first US clinical site contracted for a planned Phase 2 open-label, multicenter study of repeated…
Mogrify Raises $3.7 Million to Accelerate Mission to Transform Cell Therapy Via Direct Cellular Conversion
Cell Mogrify Ltd (Mogrify) recently announced a second close on its seed funding, bringing the total raised to $3.7 million, and the appointment of Darrin…
ADC Therapeutics Announces First Patient Dosed in Phase I Clinical Trial
ADC Therapeutics recently announced the first patient has been dosed in a Phase I clinical trial evaluating the safety, tolerability, pharmacokinetics, and anti-tumor activity of…
CytoDyn Files for Orphan Drug Designation for Triple-Negative Breast Cancer
CytoDyn Inc. recently announced it was able to reduce by more than 98% the incidence of human breast cancer metastasis in a mouse xenograft model…
Sapreme Technologies to Develop an Oligonucleotide Delivery Platform
Sapreme Technologies has been awarded a $7.71 million grant together with a multidisciplinary consortium including 11 other academic and industrial parties.
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).