Bio Platforms
First Participants Vaccinated in IMUNON’s Phase 1 Clinical Trial
IMUNON, Inc. recently announced the first participants have been treated in the IMNN-101 Phase 1 clinical trial. This proof-of-concept study of IMUNON’s proprietary PlaCCine platform…
Wistar Scientists Develop Novel Antibody Treatment for Kidney Cancer
Advanced clear cell renal cell carcinoma (ccRCC) is a deadly form of kidney cancer with few treatment options; even with new immunotherapies, only around one…
ExeVir Bio Announces Exceptional Virus Neutralization Potency of its Variant-Proof Antibody Against the SARS-CoV-2 Variant JN.1
ExeVir Bio recently announced new data demonstrating its antibodies are exceptionally potent in neutralizing the SARS-CoV-2 variant JN.1, the parental strain of the currently most…
PLATFORM TECHNOLOGY - Antibody Oligonucleotide Conjugates (AOCs™) - Revolutionizing a New Class of Targeted RNA Therapeutics
Arthur A. Levin, PhD, highlights the first-ever successful targeted delivery of RNA to muscle in humans, a revolutionary advancement for the field of RNA therapeutics that may help transform the opportunities to advance research targeting many previously untreatable diseases in the years ahead.
SPECIAL FEATURE - Outsourcing Formulation Development & Manufacturing: Going Beyond the Science to Become True Partners
Contributor Cindy H. Dubin speaks with leading CDMOs about how they are adapting to bio/pharma client needs, their capabilities in handling complex molecules, and how they are transforming from specialist contractors to true partners.
FORMULATION FORUM - Advances in Drug Delivery by Antibody Drug Conjugates (ADCs)
Shaukat Ali, PhD, and Jim Huang, PhD, focus on two aspects of drug delivery through ADCs. One, where an antibody is conjugated via a ligand with functionalized LNPs carrying cytotoxic drugs; and two, where an antibody is conjugated directly with drug through a linker at the specific site.
AMORPHOUS SOLID DISPERSION SPECIATION - Impact of Polymer Chemistry & Drug Properties
Wesley K. Tatum, PhD, focuses on the use of in vitro techniques for characterizing ASD performance and on how these techniques can be used to help better understand the role of polymer chemistry in ASD performance.
Biolojic Design Enters Multi-Target Drug Discovery Collaboration With Merck KGaA
Merck KGaA, Darmstadt, Germany, will leverage Biolojic Design’s AI capabilities to create potential best-in-class multi-specific antibodies that uniquely modulate biology….
Deciphera Pharmaceuticals Announces Oral Presentation of Results from MOTION Pivotal Phase 3 Study of Vimseltinib in Patients With Tenosynovial Giant Cell Tumor
Deciphera Pharmaceuticals, Inc. recently announced that results from the company’s MOTION pivotal Phase 3 study of vimseltinib in patients with TGCT are being highlighted in…
Denali Therapeutics Announces FDA Has Selected DNL126 (ETV:SGSH) for MPS IIIA (Sanfilippo Syndrome Type A) for START Pilot Program Intended to Accelerate Development of Rare Disease Therapies
Denali Therapeutics Inc. recently announced the US FDA has selected DNL126 for participation in the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot…
Longeveron Announces Contract Development & Manufacturing Business & First Contract
Longeveron Inc. recently announced the launch of its contract development and manufacturing business at the company’s 15,000-sq-ft state-of-the-art Good Manufacturing Practice (GMP) facility. This facility contains….
Aeterna Zentaris & Ceapro Complete Merger Transaction
Aeterna Zentaris Inc. and Ceapro Inc. recently announce the successful completion and closing of their all-stock merger of equals transaction (the Transaction), which was previously announced by….
uniQure Receives FDA Regenerative Medicine Advanced Therapy Designation for Investigational Gene Therapy in Huntington’s Disease
uniQure N.V. recently announced the US FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational gene therapy AMT-130 for the treatment of…
PhoreMost Achieves Second Milestone in Target Discovery Alliance With Boehringer Ingelheim
PhoreMost Ltd., has triggered a second milestone payment in its multi-project target discovery collaboration with Boehringer Ingelheim. This was achieved by identifying and validating novel…
Anocca AB & Shinobi Therapeutics Announce Strategic Partnership to Develop Allogeneic TCR-T Cell Therapies in Oncology
Anocca AB and Shinobi Therapeutics recently announced a strategic partnership to use Shinobi’s proprietary immune evasive iPS-T cell platform with novel candidate TCRs, discovered and…
Nxera Pharma & PrecisionLife Expand Strategic R&D Partnership Into New Therapeutic Area
Nxera Pharma Co. Ltd and PrecisionLife recently announced the expansion of their strategic R&D partnership into auto-immune disorders with the potential to identify new drug…
Elevar Therapeutics Reports Landmark Median Overall Survival of 23.8 Months in First-Line Treatment for Unresectable Hepatocellular Carcinoma
Elevar Therapeutics, Inc. recently announced the landmark overall survival (OS) analysis of camrelizumab and rivoceranib as a first-line treatment for unresectable hepatocellular carcinoma (uHCC) will…
Patient Dosing Commenced in Phase 2/3 Clinical Trial Protocol to Assess IHL-42X Drug in Patients With Obstructive Sleep Apnea
Incannex Healthcare Inc. recently announce patient dosing has commenced in the company’s Phase 2/3 clinical trial to assess safety and efficacy of IHL-42X in patients…
Sanofi Completes Acquisition of Inhibrx, Inc.
Sanofi recently announced the completion of its acquisition of Inhibrx, Inc. The acquisition adds SAR447537 (formerly INBRX-101) to Sanofi’s rare disease pipeline, underscoring the company’s…
Nxera Pharma to Receive $4.6 Million in Milestone Payments Rrom Centessa Pharmaceuticals
Nxera Pharma recently announced it expects to receive $4.6 million in milestone payments from Centessa Pharmaceuticals pursuant to a license agreement regarding Nxera technology used…
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).