Bio Platforms
Arcturus Therapeutics Receives Orphan Drug Designation
Arcturus Therapeutics Holdings Inc. recently announced the US FDA has granted Orphan Drug Designation for the company’s lead product candidate………
Seelos Therapeutics Announces Acquisition of Exclusive Worldwide License of Gene Therapy Program
Seelos Therapeutics, Inc. recently announced the exclusive worldwide licensing of a gene therapy program targeting the regulation of the SNCA gene, which encodes alpha-synuclein expression, …
Wave Life Sciences Announces Initiation of Phase 2/3 Clinical Trial
Wave Life Sciences Ltd. recently announced the initiation of DYSTANCE 51, the Phase 2/3 efficacy and safety clinical trial of suvodirsen, an investigational stereopure oligonucleotide…
Novavax & Catalent Biologics Enter Strategic Partnership
Novavax, Inc. and Catalent Biologics’ Paragon Gene Therapy unit recently announced an arrangement under which Paragon Gene Therapy will assume the leases to two Novavax product development and manufacturing facilities, giving it immediate access to……..
Innate Pharma Announces Publication of Phase I Clinical Trial Results
Innate Pharma SA recently announced the online publication in The Lancet Oncology of the results from the completed Phase 1 dose-escalation and expansion clinical trial…
Ziopharm Oncology Completes Enrollment of Phase 1 Combination Trial
Ziopharm Oncology, Inc. recently announced completion of enrollment of the third cohort of a Phase 1 clinical trial evaluating Controlled IL-12 (Ad-RTS-hIL-12 plus veledimex, Ad+V),…
Janssen Announces Collaboration to Expand Data Science Research in Healthcare
Janssen Research & Development, LLC, part of the Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen), recently announced a collaboration with the University of California,…
From ASCO 2019: TILs a Cause for Hope in Melanoma, Cervical & Breast Cancers
As the Spring heat made the light dance around the skin of the largest convention center in America, winners emerged from ASCO last month. One of the most significant announcements (from Iovance Biotherapeutics Inc.) were two first-in-man studies of adoptive T cell therapy (ACT) in patients with melanoma and cervical cancer which had failed to respond to other treatments.
Oxford BioTherapeutics Announces Receipt of Milestone Payment From Boehringer Ingelheim
Oxford BioTherapeutics Ltd. recently announced that it has received a milestone payment from Boehringer Ingelheim (BI). In addition to OBT’s two clinical stage assets, MEN1309/OBT076…
Intensity Therapeutics Announces Clinical Collaboration With Merck
Intensity Therapeutics, Inc. recently announced it has entered into an agreement with Merck through a subsidiary to evaluate the combination of Intensity’s lead product candidate…
Kymab & LifeArc Enter Strategic Partnership to Discover New Medicines Using Kymab’s IntelliSelect Technologies
The agreement will enable LifeArc to enhance its antibody drug discovery capabilities into new medicinal products with Kymab’s proprietary IntelliSelect technology platforms.
Athenex Announces US FDA Allowance of IND Application
Athenex, Inc. recently announced that the US FDA has allowed the company’s Investigational New Drug (IND) application for the clinical investigation of PT01 (Pegtomarginase) for…
Krystal Biotech Announces Positive Results from Phase 2 Clinical Trial, Receives RMAT Designation
Krystal Biotech, Inc. recently announced positive results from its Phase 2 placebo-controlled clinical trial of KB103 (GEM-2) study and an update on results from Phase1…
PREMIA & AmoyDx Announce Strategic Partnership to Establish Asia Cancer Clinical Genomic Network
Loxo Oncology, Inc. recently entered into a strategic partnership agreement with Amoy Diagnostics Co., Ltd. and PREMIA Holdings (HK) Ltd.
Protalix BioTherapeutics Completes Enrollment in Phase 3 Trial
Protalix BioTherapeutics, Inc. recently announced the completion of enrollment in the Phase 3 BRIGHT clinical trial of pegunigalsidase alfa, or PRX‑102, for the treatment of…
Bavarian Nordic Initiates Phase 3 Trial of Vaccine
Bavarian Nordic A/S recently announced the initiation of a pivotal Phase 3 trial of the freeze-dried formulation of MVA-BN smallpox vaccine in 1,110 healthy, vaccinia-naïve…
Aclaris Therapeutics Announces Positive 6-Month Results from Phase 2 Clinical Trial
Aclaris Therapeutics, Inc. recently announced positive results from a Phase 2 open-label clinical trial of ATI-502 (AGA-201), an investigational topical Janus Kinase (JAK) 1/3 inhibitor, in patients with androgenetic alopecia (AGA), a condition commonly known as male/female-pattern baldness.
Gilead & Nurix Establish Lucrative Strategic Collaboration
Gilead Sciences, Inc. and Nurix Therapeutics, Inc. recently announced a global strategic collaboration to discover, develop, and commercialize a pipeline of innovative targeted protein degradation drugs for patients with cancer and other challenging diseases.
Developing Microfluidic Routes to Effective Nanoparticle Drug Delivery Systems
A Dolomite Microfluidics’ set-up is helping researchers in the University of Manchester’s Division of Pharmacy and Optometry to enhance drug delivery. Dr Annalisa Tirella explained:…
Array BioPharma Acquisition Will Accelerate Pfizer’s Position as a Major Player in Colorectal Cancer
Following the news that Pfizer has agreed to acquire Array BioPharma in a deal worth approximately $11.4 billion, Tajekesa Chapman, PhD, Senior Pharma Analyst at GlobalData, a leading data and analytics company, offers her view on the potential of this buy-out on the treatment of colorectal cancers.
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).