Bio Platforms
Traws Pharma’s Bird Flu Drug Candidate Presented at ICAR
Traws Pharma, Inc. recently announced that positive data supporting the potential for tivoxavir marboxil (TXM) as a bird flu treatment was presented yesterday in a…
Silexion Therapeutics Announces Completion of Innovative Expanded Development Plan for SIL204
Silexion Therapeutics Corp. recently announced it has completed an expanded development plan for its next-generation siRNA candidate, SIL204, which the company’s management will be presenting…
Wugen Announces Dosing of First Patients in Pivotal Trial of Off-the-Shelf, Allogeneic, CD7-Targeted CAR-T Cell Therapy WU-CART-007
Wugen, Inc. recently announced the dosing of the first patients in its pivotal Phase 2 study evaluating WU-CART-007, a potential first-in-class, investigational, anti-CD7 CAR-T cell…
Esperion Aligns With FDA to Initiate Phase 3 Clinical Trials of Bempedoic Acid in Pediatric Heterozygous & Homozygous Familial Hypercholesterolemia
Esperion recently announced that following meetings with the US FDA, it has gained alignment on a regulatory path forward for initiating Phase 3 studies of…
Indaptus Therapeutics Reports New Data Demonstrating Successful Broad Immune System Activation in Weekly Dosing Trial of Decoy20
Indaptus Therapeutics, Inc. recently provided an update on key pharmacodynamic findings from the weekly dosing cohort of its ongoing Phase 1 trial of Decoy20. As…
Candel Therapeutics & IDEA Pharma Announce Strategic Partnership to Advance Path-to-Market Commercialization Efforts for CAN-2409
Candel Therapeutics, Inc. together with IDEA Pharma, a Division of SAI MedPartners (IDEA) recently announced a strategic commercial partnership. Under this agreement, IDEA will provide…
Second Data Safety Monitoring Board Issues Positive Recommendation to Continue BriaCell’s Phase 3 Study in Metastatic Breast Cancer
BriaCell Therapeutics Corp. recently announced the external Data Safety Monitoring Board (DSMB), an independent group of experts who review and monitor the safety data of…
Dyadic Announces Funding Award to Use C1 to Accelerate Development of Protein-Based Vaccines
Dyadic International, Inc. recently announced it has received a funding award from CEPI to use C1 to accelerate the development of protein-based vaccines. CEPI is…
Longeveron Announces Positive Type B Meeting With FDA Regarding Pathway to BLA for Laromestrocel in Alzheimer’s Disease
Longeveron Inc. recently announced the positive outcome of a Type B meeting with the US FDA, supporting the advancement of laromestrocel (Lomecel-B), a proprietary, scalable,…
Acumen Pharmaceuticals Announces Topline Results From Phase 1 Study of Subcutaneous Formulation of Sabirnetug
Acumen Pharmaceuticals, Inc. recently announced topline results from its Phase 1 study comparing pharmacokinetics (PK) between subcutaneous (SC) and intravenous (IV) formulations of sabirnetug in healthy…
Genenta Announces $21.9M Financing to Expand Pipeline as Brain Tumor Trial is Showing Promising Survival Rates
Genenta Science recently announced a $21.9 million financing through the issuance of a Mandatory Convertible Bond to ENEA Tech and Biomedical (ETB) to support the expansion…
Tract Bio Announces Publication in Gastroenterology Potential Targets for Esophageal Adenocarcinoma Identified Using stemECHOTM Cloning Platform
Tract Bio recently announced the publication of preclinical research in Gastroenterology. The article, “Evolution of Esophageal Adenocarcinoma from Precursor Lesion Stem Cells” describes results from…
llarity Therapeutics Announces Presentation of Phase 2 Clinical Data from Ongoing Trial in Advanced Ovarian Cancer Patients
Allarity Therapeutics, Inc. announced the presentation of new clinical data from its ongoing Phase 2 trial with stenoparib monotherapy in advanced Ovarian Cancer at the…
GRIN Therapeutics Receives FDA Orphan Drug Designation for Treatment of GRIN-Related Neurodevelopmental Disorder Treatment
GRIN Therapeutics, Inc. recently announced the US FDA granted Orphan Drug designation for its investigational drug, radiprodil, for the treatment of GRIN-related neurodevelopmental disorder (NDD).…
Cellphire Therapeutics CLPH-511 Fast Track Designation Granted
Cellphire Therapeutics, Inc. recntly announced the US FDA has granted Fast Track designation for CLPH-511 (Frozen Activated Platelets) Injectable Suspension for the treatment of acute…
Gradalis Announces Development of Clinically Relevant Exome Sequencing Bioinformatics Pipeline to Determine Clonal Tumor Mutation Burden
Gradalis, Inc. recently announced a peer-reviewed publication in Scientific Reports. The paper details the methods and validation of Gradalis’ exome sequencing procedure and associated bioinformatics…
Exogenus Therapeutics Chooses Lonza to Develop a GMP-Compliant Process to Manufacture Exo-101, its Lead Exosome-Based Candidate
Exogenus Therapeutics, a Portuguese biotech company, and Lonza, a global development and manufacturing partner to the pharmaceutical and biotech markets, announced today a collaboration to…
Kaerus Bioscience Successfully Completes Phase 1 Trial & Demonstrates Proof of Mechanism With its Novel BK Channel Modulator Being Developed for Fragile X Syndrome
Kaerus Bioscience recently announced the successful completion of its Phase 1 clinical trial of KER-0193, a novel BK channel modulator the company is developing for…
Purple Biotech Granted New Patent for NT219 in Combination With EGFR Antibody Therapy, Enhancing Global IP Protection in Major Markets
Purple Biotech Ltd. recently announced the U.S. Patent and Trademark Office has issued a patent for NT219 used in combination with epidermal growth factor receptor…
Jupiter Neurosciences & Aquanova AG Announce Strategic Collaboration to Develop Longevity & Healthspan Products
Jupiter Neurosciences, Inc. (NASDAQ: JUNS) (“Jupiter” or the “Company”), a clinical-stage pharmaceutical company developing JOTROL, a patented resveratrol-based platform, today announced a strategic partnership with…
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).