Bio Platforms
Pfenex Receives US FDA Approval for PF708 to Treat Osteoporosis
Pfenex Inc. recently announced the US FDA has approved the new drug application (NDA) for PF708 submitted under the 505(b)(2) regulatory pathway, with…
MilliporeSigma Becomes the First to Use Acoustic Technology for Cell Therapy Manufacturing
MilliporeSigma recently announced it has acquired FloDesign Sonics, of Wilbraham, MA, developer of a unique acoustic cell processing platform for the industrialization of cell and…
Akcea & Pfizer Announce $250-Million Licensing Agreement for Investigative Therapy
Akcea Therapeutics, Inc., a majority-owned affiliate of Ionis Pharmaceuticals, Inc., and Pfizer Inc. recently announced the companies have entered into a worldwide exclusive licensing agreement…
Cabaletta Bio Receives IND Clearance From FDA to Initiate First Clinical Trial
Cabaletta Bio, Inc. recently announced it has received clearance of its IND application from the US FDA to initiate a first-in-human clinical trial of…..
Ocugen & CanSinoBIO Enter Strategic Co-Development & Manufacturing Partnership
Ocugen, Inc. has recently entered into a strategic partnership with CanSino Biologics on Ocugen’s gene therapy pipeline product candidates for inherited retinal diseases, which are…
Novel Silica Delivery System Enters Final Preclinical Development Stage
N4 Pharma Plc recently announced results from the company’s latest research study into its novel silica nanoparticle for cancer treatments and vaccines, Nuvec. The work,…
AGTC & Otonomy Announce Strategic Collaboration to Develop & Commercialize Gene Therapy
Applied Genetic Technologies Corporation and Otonomy, Inc. recently announced they have entered into a strategic collaboration to co-develop and co-commercialize an AAV-based gene therapy to…
Entera Bio Reports Positive Results from a Phase 2 PK/PD Study of Oral PTH
Entera Bio Ltd. recently announced the presentation of positive results from its pharmacokinetic (PK) and pharmacodynamic (PD) study in patients with hypoparathyroidism treated with its…
Immutep to Receive Milestone Payment From GSK
Immutep Limited recently announced it will receive a milestone payment from GSK of $5.02 million related to the first patient being dosed in…..
Atlas Nanotech Announces Successful Development of First-Ever OTC Nanotechnology Vitamin A
Costas Inc. DBA Atlas Nanotech adds another unique nanotechnology medical product to its portfolio. Recently, CEO Dr. Julio Riestra announced the completion of development of…
Mustang Bio Announces Phase 1 Clinical Trial
Mustang Bio, Inc. recently announced that City of Hope, a world-renowned independent cancer research and treatment center, has opened and is beginning to treat its…
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,…
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).