Bio Platforms
Aptevo Therapeutics Introduces New Adaptir Bispecific Antibody Candidate
Aptevo Therapeutics Inc. recently introduced a new immuno-oncology candidate, APVO603, built on Aptevo’s proprietary ADAPTIR bispecific antibody platform. Jane Gross, PhD, Chief Scientific Officer for…
Novel Small Molecule Drug Discovery Platform Harnesses Immune System to Defeat Cancers, Viral Infections & Inflammatory Diseases
Now, the power of nucleic acid technology is being harnessed to design small molecule nucleic acid hybrid (SMNH) drugs that harness the patient’s own immune system to defeat…..
Rafael Pharmaceuticals Announces Expansion Into France of Pivotal Phase 3 Trial
Rafael Pharmaceuticals, Inc. recently announced the expansion of its Phase 3 clinical trial for patients with metastatic pancreatic cancer (AVENGER 500) into France. AVENGER 500…
Bellerophon Receives Orphan Drug Designation
Bellerophon Therapeutics, Inc. recently announced the US FDA has granted Orphan Drug Designation to nitric oxide for the treatment of Idiopathic Pulmonary Fibrosis (IPF). IPF…
Wave Life Sciences Announces Fast Track Designation from US FDA
Wave Life Sciences Ltd. recently announced the US FDA has granted Fast Track designation to suvodirsen for the treatment of Duchenne muscular dystrophy (DMD) in…
Ovid Therapeutics Announces First Patient Randomized in Pivotal Phase 3 Trial
Ovid Therapeutics Inc. recently announced the first patient has been randomized in the company’s single, pivotal Phase 3 NEPTUNE clinical trial evaluating OV101 (gaboxadol), a…
Cellectar Presents Data From Phase 1 Study
Cellectar Biosciences, Inc. recently announced Mr. Jarrod Longcor, chief business officer of Cellectar presented data from Cohort 6 of its Phase 1 dose escalation study…
Allena Pharmaceuticals Completes Enrollment in Pivotal Phase 3 Trial
Allena Pharmaceuticals, Inc. recently announced it has completed enrollment in its pivotal Phase 3 URIROX-1 clinical trial. URIROX-1 is a multi-center, global, randomized, double-blind, placebo-controlled…
TYME Presents Positive Circulating Tumor Cell Data
Tyme Technologies, Inc. recently reported encouraging data on circulating tumor cells (CTCs) and a correlation with decrease in risk of death using TYME's lead candidate,…
Kitov Pharma Presents Newly Released Data for Reversing Pancreatic Cancer Drug Resistance
Kitov Pharma Ltd. recently presented newly released proof-of-concept data showing evidence of NT-219 mechanism of action in reversing cancer drug resistance in PDX models. The…
ORYZON’s Ladademstat Shows Efficacy Signs in Relapsed SCLC
Oryzon Genomics, S.A. recently presented the first preliminary data from CLEPSIDRA, a Phase 2 trial investigating iadademstat in combination with standard-of-care in relapsing small cell…
ProBioGen Licenses Technology to Bayer
ProBioGen AG, a premier service and technology provider for complex therapeutic antibodies and glycoproteins, recently announced the closing of a license agreement with Bayer AG…
Flexion Therapeutics & Xenon Pharmaceuticals Announce Acquisition
Flexion Therapeutics, Inc. and Xenon Pharmaceuticals Inc. recently announced the companies have entered into a definitive agreement that provides Flexion with the global rights to…
Nabriva Therapeutics Earns $5-Million Milestone Payment
Nabriva Therapeutics plc recently announced that Nabriva has earned a $5-million milestone payment under its licensing agreement with Sinovant Sciences related to the U.S. regulatory…
AC Immune Receives Milestone Payment in Connection with Initiation of a Phase 2 Trial
AC Immune SA recently announced it has received a milestone payment from its partner Life Molecular Imaging in connection with the initiation of….
DURECT Earns $10-Million Milestone Payment From Gilead
DURECT Corporation recently announced that further development of a long-acting injectable HIV investigational product utilizing DURECT’s SABER technology has triggered a $10-million milestone payment from Gilead Sciences, Inc. to…….
Kaleido Biosciences to Collaborate with Gustave Roussy Cancer Center
Kaleido Biosciences, Inc. recently announced plans to develop Microbiome Metabolic Therapies (MMT) to enhance the effects of cancer immunotherapies under a new…….
Fate Therapeutics Announces FDA Clearance of IND Application
Fate Therapeutics, Inc. recently announced the US FDA has cleared the company’s Investigational New Drug (IND) application for FT596, the company’s first off-the-shelf chimeric antigen…
Krystal Biotech Announces Initiation of Phase 1/2 Study
Krystal Biotech Inc. recently announced the initiation of the Phase 1/2 study of KB105 in transglutaminase-1 (TGM1) deficient autosomal recessive congenital ichthyosis (ARCI). The study, termed…
Atossa Genetics Announces Completion of Enrollment & Dosing in Phase 1 Clinical Trial
Atossa Genetics Inc. recently announced that all participant activities have been completed in its Phase 1 clinical trial of a new proprietary modified-release oral tablet form…
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).