Bio Platforms
Provectus Biopharmaceuticals Announces Discovery of Unique PV-10-Induced STING Pathway Mechanism
Provectus recently announce that data from ongoing preclinical study of investigational autolytic cancer immunotherapy PV-10 (rose bengal disodium) was presented at the American Association for…
uniQure Announces First Two Patients Treated in Phase 1/2 Clinical Trial
uniQure NV recently announced that the first two patients in the Phase 1/2 clinical trial of AMT-130 for the treatment of Huntington’s disease have been…
Onconova Therapeutics Announces Initiation of a Phase 1/2a Study
Onconova Therapeutics, Inc. recently announced an investigator-initiated Phase 1/2a trial of oral rigosertib plus nivolumab in advanced metastatic KRAS mutated (KRAS+) lung adenocarcinoma has begun…
Rubius Therapeutics Highlights Preclinical Data for Oncology Product Candidate
Rubius Therapeutics, Inc. recently announced the presentation of preclinical data supporting its lead artificial antigen-presenting cell (aAPC) program, RTX-321, for the potential treatment of human…
Bionic Sight Doses First Patient in a Phase 1/2 Clinical Trial of a New Investigational Treatment for Blindness
Bionic Sight, LLC recently announced it has dosed the first patient in the company’s Phase 1/2 clinical trial of BS01, an optogenetic gene therapy for patients with….
Pfenex Announces US Commercial Launch of Teriparatide Injection
Pfenex Inc. recently announced that its commercialization partner, Alvogen, has launched Teriparatide Injection in the US. Teriparatide Injection (also referred to as PF708 and Bonsity)…
Ovid Therapeutics Announces Strategic Research Collaboration Focused on Accelerating the Development of New Treatments for Rare Neurological Diseases
Ovid Therapeutics Inc. recently announced a strategic research collaboration with Columbia University Irving Medical Center researchers to advance genetic based therapies for……
Immunic Announces FDA Allowance of its Phase 2 Trial of COVID-19 Drug
Immunic, Inc. recently announced receipt of regulatory allowance from the US FDA to initiate its Phase 2, CALVID-1 clinical trial of IMU-838, the company’s selective…
Precision BioSciences Announces Dosing of First Patient in Phase 1/2a Clinical Trial
Precision BioSciences, Inc. recently announced that the first patient has been dosed in a Phase 1/2a clinical trial of PBCAR269A, its third allogeneic chimeric antigen…
Arch Biopartners Submits IND Application to the FDA for Metablok (LSALT Peptide)
Arch Biopartners Inc. recently announced it has submitted an Investigational New Drug (IND) Application to the US FDA for its lead drug Metablok (LSALT peptide)…
GENE THERAPY - The Role Viral Vectors Play in Current Gene Therapy Development
Dieter Lingelbach, MBA, discusses the history and science of viral vectors, their current applications, and why they are a vital component in contemporary gene therapy development.
NEXT-GENERATION TUMOR TARGETING - Leveraging the Tumor Microenvironment to Change the Standard of Care
Vishwas Paralkar, PhD, highlights a unique platform technology that represents the first technology that has successfully been used to target cancer drugs to tumor cells in animal models, while sparing healthy tissue.
EXECUTIVE INTERVIEW - Adare Pharmaceuticals: A Virtual Acquisition is Possible
Ajay Damani, VP of Pharmaceutical Technologies Business Unit, Adare, and Maria Flynn, President and CEO, Orbis, discuss how both companies will benefit from the acquisition, future product development, and the challenges of a virtual acquisition.
Seelos Therapeutics Announces Initiation of Preclinical Study of SLS-004 in Parkinson’s Disease
Seelos Therapeutics, Inc. recently announced the initiation of a preclinical study of SLS-004 in Parkinson’s disease (PD) through an all-in-one lentiviral vector targeting the synuclein…
Atriva Therapeutics to Develop ATR-002 for Treatment of Patients with COVID-19 in Phase 2 Study
Atriva Therapeutics GmbH recently announced a clinical strategy to treat patients with moderate to severe COVID-19 who require hospitalization. ATR-002, an oral small molecule, has…
WindMIL Therapeutics Announces Dosing of First Patient in Phase 2 Clinical Study
WindMIL Therapeutics recently announced that the first patient has been dosed in the combination therapy portion of its Phase 2 clinical trial to assess the…
ARCA Biopharma Announces AB201 Development Program for Treatment of COVID-19 Associated Coagulopathy
ARCA biopharma, Inc. recently announced a new development program to evaluate AB201 (rNAPc2), a potent, selective inhibitor of tissue factor (TF), as a potential treatment…
Microfluidics Enables Reliable siRNA Drug Delivery for Inflammatory Diseases & Tumor Targeting
Researchers in the Department of Pharmacy at the Ludwig Maximilian University of Munich are using chips from Dolomite Microfluidics to reliably and consistently produce monodisperse…
KBP Biosciences Completes Enrollment of BLOCK CKD Phase 2b Study
KBP Biosciences recently announced it has completed patient enrollment of BLOCK CKD (Blood Pressure in Chronic Kidney Disease), its Phase 2b study of its lead…
Alethia Biotherapeutics Announces Receipt of FDA Authorization to Begin Phase 2 Development
Alethia Biotherapeutics recently announced the US FDA has cleared its Phase 2 Investigational new drug (IND) application for AB-16B5, a potent inhibitor of the epithelial…
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).