Bio Platforms
Immutep Announces First Participant Dosed in Phase 1 Study of First in Class Agonist LAG-3 Antibody
Immutep Limited recently announced the first participant has been successfully dosed in the first-in-human Phase 1 trial of IMP761. This first-in-class agonist LAG-3 antibody is…
Apollomics Announces Positive Preliminary Data of Vebreltinib in Patients With Non-CNS MET Fusion Solid Tumors From its Phase 2 Trial
Apollomics Inc. recently announced positive preliminary clinical data for the cohort of patients with non-CNS MET fusion solid tumors from the Phase 2 SPARTA trial…
Purdue & Akston Biosciences Sign Partnership Agreement for Dog Cancer Drug Development
Akston Biosciences Corporation and Purdue University recently announced a strategic partnership to co-develop an anti-cPD-L1 monoclonal antibody (mAb) immunotherapy to treat cancer in dogs. The underlying…
Eclipse Life Sciences Initiates Phase 2 Study for Novel Ophthalmology Steroid Implant for Diabetic Macular Edema
Eclipse Life Sciences, Inc. recently announced it has enrolled and dosed its first patient in the BETTIS-1 Phase 2 clinical trial (NCT06536491) for EC-104 fluocinolone…
MBX Biosciences Doses First Patient in Phase 2 Avail Trial for the Treatment of Hypoparathyroidism
MBX Biosciences, Inc. recently it has dosed the first patient in its Phase 2 Avail trial of MBX 2109, the company’s parathyroid hormone (PTH) peptide…
IN8bio Solidifies Position as a Clinical Leader of Gamma-Delta T Cell Therapy in Oncology With 100% of Treated AML Patients in Complete Remission& Receives FDA Guidance for Registrational Trial of INB-100
IN8bio, Inc. recently announced updated positive clinical data from both of the company’s Phase 1 investigator-sponsored trials of INB-100 for hematological malignancies and INB-200 for…
Wave Life Sciences Receives FDA Rare Pediatric Disease Designation for the Treatment of Duchenne Muscular Dystrophy
Wave Life Sciences Ltd. recently announced the US FDA has granted Rare Pediatric Disease Designation to WVE-N531 for the treatment of boys with Duchenne muscular…
Altamira Therapeutics Announces Significant Enhancement of Immune Checkpoint Inhibition Therapy in Combination With Zbtb46 mRNA Delivered With SemaPhore Nanoparticles in Animal Tumor Models
Altamira Therapeutics Ltd. recently announced the publication of a peer-reviewed article in Nature Immunology highlighting the important role of the Zbtb46 gene in the control…
Abzena Supplies Clinical Trial Material for Angiex’s Phase 1 Study of First-in-Class TM4SF1-Directed ADC
Angiex, a developer of Nuclear-Delivered Antibody-Drug Conjugate (ND-ADC) therapies for solid cancers, recently announced that patient dosing has begun for their Phase 1 clinical trial…
ANEW MEDICAL Announces Patents Issued in Major Asian Markets for Use of Genetic Therapy
ANEW MEDICAL, INC. recently announced the grant and issuance of patent number HK1259628 protecting the development of novel genetic approaches for the treatment of neurodegenerative…
Conduit Pharmaceuticals Enters Exclusive License Agreement With AstraZeneca for Multiple Assets
Conduit Pharmaceuticals Inc. recently announced it has entered into an agreement with AstraZeneca to exclusively license rights to develop AZD1656 and AZD5658, both HK-4 glucokinase…
Apellis & Sobi Announce Positive Topline Results From Phase 3 Study of Pegcetacoplan in C3G & Primary IC-MPGN
Apellis Pharmaceuticals, Inc. recently announced positive topline results from the Phase 3 VALIANT study investigating systemic pegcetacoplan in patients with C3 glomerulopathy (C3G) or primary immune…
Palisade Bio Selects Dose for Upcoming Phase 1 Clinical Study of PALI-2108 for Ulcerative Colitis
Palisade Bio, Inc. provided a progress update on the advancement of development for its lead drug candidate, PALI-2108 towards initiation of Phase 1 clinical trials in…
Oragenics Announces Concussion Drug Successfully Clears FDA-Required Cardiotoxicity Testing
Oragenics, Inc. recently announced its lead candidate for treating concussion successfully completed a study that indicates ONP-002 does not cause cardiotoxicity. ONP-002 is a new…
Nutcracker Therapeutics Announces Publication in ACS Nano Demonstrating the Therapeutic Viability of Nutshell Delivery Vehicles
Nutcracker Therapeutics, Inc. recently announced the publication of its latest peer-reviewed paper, which explores the fundamental aspects of its Nutshell platform of peptoid-based mRNA delivery vehicles.…
Agios to Receive $1.1 Billion in Milestone Payments
Agios Pharmaceuticals, Inc. recently announced it will receive a total of $1.1 billion in milestone payments following US FDA approval of vorasidenib for adult and…
Silo Pharma Announces Partnership With Global CRO for CNS Homing Peptide
Silo Pharma, Inc. recently announced it has entered into an agreement with WuXi AppTec (Hong Kong) Limited for a preclinical small animal study of SPU-16,…
Levicept Announces Positive Results of Phase 2 Trial of Novel Neurotrophin-3 Inhibitor for the Treatment of Patients With Moderate to Severe Osteoarthritis
Levicept Ltd recently announced positive results from its Phase 2 trial of LEVI-04 a novel and first in class neurotrophin-3 (NT-3) inhibitor, showing that LEVI-04…
George Medicines Files NDA for Novel Low Dose Triple Combination for Treatment of Hypertension Following Successful Phase 3 Development Program
George Medicines recently announced its submission to the US FDA of a New Drug Application (NDA) for GMRx2, George Medicines’ lead pipeline candidate for the…
Annovis’ Lead Compound Enhances Cognition Synergistically with GLP-1 Agonist
Annovis Bio Inc. recently announced new preclinical data demonstrating the synergistic effect of its lead compound, buntanetap, when combined with the glucagon-like peptide 1 (GLP-1)…
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).