Bio Platforms
Chiasma Announces US Commercial Launch & Availability of the First & Only Oral Somatostatin Analog
Chiasma, Inc. recently announced the commercial launch and availability of MYCAPSSA (octreotide) capsules in the US for patients with acromegaly. MYCAPSSA, the first and only….
Novavax & Canada Reach Agreement in Principle to Acquire COVID-19 Vaccine
Novavax, Inc. recently announced it has reached an agreement in principle with the Government of Canada to supply up to 76 million doses of NVX-CoV2373,…
First Patient Dosed in AstraZeneca’s Phase 3 COVID-19 Vaccine Trial
Headlands Research is pleased to announce that on Friday, Aug. 28, its JEM Research Institute clinical trial site in Lake Worth, FL, successfully dosed the…
GENE-EDITING TECHNOLOGY - The Importance of Assessing Off-Target Effects of CRISPR Gene Editing
Mark Behlke, MD, PhD, says gene editing raises legitimate questions and fears about possible risks and misuse, and given the relative technical ease and low cost of CRISPR gene editing, this is likely to find widespread use in both medicine and agriculture.
PEPTIDE THERAPEUTICS - Oral Peptide Therapeutics – Opportunities Abound as Barriers Fall
John S. Vrettos, PhD, says numerous technologies are currently in development that are designed to enable the oral delivery of peptides. Though each has its unique set of properties and capabilities, all must overcome key obstacles to successfully deliver peptides via the oral route.
EXECUTIVE INTERVIEW - Nuritas Pharma: Life Changing Peptide Drug Discoveries for a Changing World
Nora Khaldi, PhD, Founder and Chief Executive Officer of Nuritas, discusses the company’s innovative approach to accelerating the discovery of novel therapeutic peptides that address unmet patient needs in multiple disease areas.
Rain Therapeutics Announces License Agreement for the Development of Proprietary RAD52 Inhibitors
Rain Therapeutics Inc. recently announced the signing of an exclusive, worldwide license agreement of a Drexel University research program to Rain for small molecule inhibitors of….
Catalent Signs Agreement With AstraZeneca to Expand Manufacturing Support for COVID-19 Vaccine
Catalent, Inc. recently announced that Catalent Cell & Gene Therapy will provide drug substance manufacturing to AstraZeneca for the University of Oxford’s adenovirus vector-based COVID-19 vaccine….
BioAge Discovers Key Pathway & Identifies Promising Phase 2-Ready Drug to Treat & Reverse Immune Aging, a Root Cause of COVID-19 Morbidity & Mortality in Older Patients
BioAge Labs, Inc. recently announced it has in-licensed a clinical-stage therapy with significant promise and potential in treating immune aging in older patients hospitalized with…
Organicell Provides Phase 1/2 Clinical Trial Update
Organicell Regenerative Medicine, Inc. recently announced it has partnered with Alternative Research Associates, LLC and Larkin Hospital in Miami, FL, to initiate a randomized, double-blinded,…
TFF Pharmaceuticals Enters into Worldwide Licensing Agreement With UNION Therapeutics
TFF Pharmaceuticals, Inc. recently announced it has entered into a worldwide exclusive licensing agreement with UNION therapeutics A/S (UNION) for its TFF technology used in…
Mustang Bio Announces Rare Pediatric Disease Designation
Mustang Bio, Inc. recently announced the US FDA has granted Rare Pediatric Disease Designation to MB-107, Mustang’s lentiviral gene therapy for the treatment of X-linked…
Avacta & LG Chem Life Sciences Expand Partnership
Avacta Group plc recently announced the expansion of its existing multi-target collaboration and development agreement with LG Chem Life Sciences to include new programs incorporating…
Scioto Biosciences Receives $26.5 Million Series B Investment From Genome & Company
Scioto Biosciences recently announced the closing of a strategic investment of up to $26.5 million from Genome & Company and other investors to join in an upcoming tranche….
Protalix BioTherapeutics & Chiesi Global Rare Diseases Announce US FDA Acceptance of Biologics License Application
Protalix BioTherapeutics, Inc. together with its development and commercialization partner Chiesi Global Rare Diseases recently announced the US FDA has accepted the Biologics License Application (BLA) and granted….
GigaGen Announces Publication of Research Describing a New Class of Drugs, Recombinant Hyperimmunes, Including its Novel COVID-19 Therapy, GIGA-2050
GigaGen presents a novel technology for producing a new class of drug, which it calls recombinant hyperimmunes…..
Multiple Headlands Research Sites Selected for AstraZeneca’s Phase 3 COVID-19 Vaccine Trial
Headlands Research recently announced its research centers have been selected to participate in AstraZeneca’s upcoming Phase 3 COVID-19 vaccine trial. Headlands has built an industry-leading…
Eyenovia & Arctic Vision Announce Exclusive Collaboration & License Agreement
Eyenovia, Inc. and Arctic Vision recently announced they have entered into an exclusive license agreement for Arctic Vision to develop and commercialize MicroPine for the…
Immutep Granted US Patent for Eftilagimod Alpha in Chemo-Immunotherapy Combination
Immutep Limited, a biotechnology company developing novel immunotherapy treatments for cancer and autoimmune disease, recently announced the grant of a new patent (number 10,736,940) titled…
Clearside Biomedical Announces US FDA Acceptance of IND Application
Clearside Biomedical, Inc. recently announced the US FDA has accepted its Investigational New Drug (IND) application for CLS-AX (axitinib injectable suspension), enabling initiation of a…
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).