Bio Platforms
FDA Grants Fast Track Designation for Longeveron’s Treatment for Hypoplastic Left Heart Syndrome in Infants
Longeveron Inc. recently announced the US FDA has granted Fast Track Designation to Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare…
WHITEPAPER - Key Considerations in Oral Delivery of Peptides – Factors to Consider While Evaluating Oral Administration
Peptide therapeutics are a hot topic in pharmaceutical R&D. While most peptides are administered parenterally, oral delivery of peptide therapeutics offers several advantages. Enteris BioPharma’s…
Immutep Granted Japanese Patent for Eftilagimod Alpha, a Soluble Lag-3 Protein, in Combination With a PD-1 Pathway Inhibitor
Immutep Limited recently announced the grant of a new patent (No. 7116547) titled Combined Preparations for the Treatment of Cancer or Infection by the Japanese…
Timber Pharmaceuticals Announces Publication of Results from Phase 2b CONTROL Study of FDA-Designated Breakthrough Therapy
Timber Pharmaceuticals, Inc. recently announced that results from the previously completed Phase 2b CONTROL study that evaluated TMB-001, a topical isotretinoin formulated using the company’s patented IPEG delivery system, in moderate-to-severe congenital ichthyosis (CI) are published….
Anthos Therapeutics Launches Second Phase 3 Clinical Trial of its Dual-Acting Factor XI Inhibitor
Anthos Therapeutics recently announced the initiation of recruitment for its second Phase 3 clinical trial investigating abelacimab, its novel dual-acting fully human monoclonal antibody targeting…
Ranok Therapeutics Announces Initiation of Patient Dosing in a Phase 1/2 Clinical Trial of First-in-Class BRD4-Targeting CHAMP Protein Degrader
Ranok Therapeutics recently announced the initiation of patient dosing in the US for a Phase 1/2 study of RNK05047. The trial, entitled CHAMP-1, will evaluate the safety, tolerability, pharmacokinetics, and….
Immatics Announces First Cancer Patient Treated With Second-Generation ACTengine TCR-T Candidate
Immatics N.V. recently announced the treatment of the first patient in its Phase 1b expansion cohort C (NCT03686124) evaluating IMA203CD8, the company’s second-generation TCR-T monotherapy…
Cue Biopharma Doses First Patient in Phase 1 Study of CUE-102 for Wilms’ Tumor 1 (WT1) - Expressing Cancers
Cue Biopharma, Inc. recently announced it has dosed the first patient in a Phase 1 dose escalation study evaluating CUE-102, its second clinical drug candidate…
AUM Biosciences Receives FDA Orphan Drug Designation for the Treatment of Solid Tumors With the NTRK Fusion Gene
AUM Biosciences recently announced the US FDA has granted Orphan Drug Designation (ODD) to AUM601, a highly selective, oral small molecule that inhibits not only…
Blue Water Vaccines Announces Exploration of its VLP Platform for Use in Monkeypox Vaccine Candidate
Blue Water Vaccines Inc. recently announced the company plans to explore the potential to develop a novel monkeypox vaccine using its norovirus shell and protrusion…
Ventyx Biosciences Announces Positive Topline Phase 1 Data for its Selective Allosteric TYK2 Inhibitor VTX958
Ventyx Biosciences, Inc. recently announced positive data from the company’s Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) trial of VTX958, a…
Immunic Receives Notice of Allowance for Composition-of-Matter Patent in the US for Small Molecule Modulator Targeting Restoration of Intestinal Barrier Function & Regeneration of Bowel Epithelium
Immunic, Inc. recently announced it has received a Notice of Allowance from the US Patent and Trademark Office (USPTO) for patent application 16/646130 titled Compound…
Artelo Biosciences Reports Positive Preclinical Results for Novel Inhibitor to Fatty Acid Binding Protein 5
Artelo Biosciences, Inc. recently announced publication of preclinical results indicating a novel fatty acid binding protein 5 (FABP5) inhibitor from the company’s FABP inhibitor platform…
Synlogic Announces Synthetic Biotic for Gout Developed in Partnership With Ginkgo Bioworks
Synlogic, Inc. recently announced a new drug candidate for the treatment of gout developed in partnership with Ginkgo Bioworks The new candidate, SYNB2081, is a Synthetic…
Valneva & Pfizer Initiate Phase 3 Study of Lyme Disease Vaccine Candidate
Valneva SE and Pfizer recently announced the initiation of a Phase 3 clinical study, Vaccine Against Lyme for Outdoor Recreationists (VALOR) (NCT05477524), to investigate the efficacy, safety and….
Fate Therapeutics Announces Preclinical Publication Highlighting Derivation of CD8αβ T Cells From TCR-CAR+ Induced Pluripotent Stem Cells
Fate Therapeutics, Inc. recently announced the publication of preclinical study results demonstrating the successful generation, durable anti-tumor response, and functional persistence of TCR-CAR+ iPSC-derived CD8αβ…
Rain Therapeutics Announces Completion of Enrollment in Phase 3 MANTRA Trial for Milademetan in Liposarcoma
Rain Therapeutics Inc. recently announced completion of enrollment in its Phase 3 MANTRA randomized, global, registrational trial of its lead product candidate, milademetan, an oral,…
BriaCell Secures License for Promising Novel Anti-Cancer Agent
BriaCell Therapeutics Corp. recently announced it has secured an exclusive license from University of Maryland, Baltimore County (UMBC) to develop and commercialize Soluble CD80 (sCD80)…
US Patent Issued to HCW Biologics for Foundational Platform Technology
HCW Biologics Inc. was granted US Patent No. 11,401,324, which contains claims for immunotherapeutic compounds comprised of a single-chain chimeric polypeptide with two target-binding domains…
PolyPid Announces Exclusive Licensing Agreement With Advanz Pharma
PolyPid Ltd. recently announced it has entered into an exclusive licensing agreement with ADVANZ PHARMA Corp. for the commercialization of its lead drug candidate, D-PLEX100 for the prevention of….
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).