Bio Platforms
Avalo Announces First Patient Dosed in Phase 2 Trial of AVTX-009 for the Treatment of Hidradenitis Suppurativa
Avalo Therapeutics, Inc. recently announced the first patient has been dosed in its Phase 2 LOTUS trial of AVTX-009 in hidradenitis suppurativa (HS). AVTX-009 is…
MiNK Therapeutics & Autonomous Therapeutics Announce Collaboration to Develop Novel Therapies Targeting Metastatic Tumors
MiNK Therapeutics and Autonomous Therapeutics, Inc. recently announced a research collaboration aimed at effectively targeting and treating metastatic tumors. This collaboration will leverage Autonomous’ precision…
Traws Pharma Announces Positive Topline Phase 1 Data for Flu Candidate
Traws Pharma, Inc. recently announced positive topline Phase 1 safety and pharmacokinetic results for its investigational one-dose influenza (flu) therapy, tivoxavir marboxil (tivoxavir). Tivoxavir was…
ImmuneSensor Therapeutics Initiates Dosing in Phase 1 Trial of Lead cGAS Inhibitor Drug Candidate
ImmuneSensor Therapeutics recently announced the first dose level cohort of healthy volunteers has been dosed in a Phase 1 randomized, double-blinded, placebo-controlled clinical trial of…
GRIN Therapeutics Announces Initiation of Astroscape Clinical Trial of Radiprodil for Treatment of Tuberous Sclerosis Complex & Focal Cortical Dysplasia Type II
GRIN Therapeutics Inc. recently announced initiation of the Astroscape study, its global open-label clinical trial of radiprodil, an investigational, selective and potent negative allosteric modulator…
NeOnc Technologies Receives Pharma Tech Outlook’s Top Drug Delivery Platform Award for 2024
NeOnc Technologies Holdings, Inc. has been recognized by Pharma Tech Outlook as the Top Drug Delivery Platform of 2024. According to the publisher of the…
Avillion Announces Positive High-Level Results From Phase 3 BATURA Trial With AstraZeneca’s Inhaled Anti-Inflammatory Medication Airsupra in Asthma Patients
Avillion Life Sciences Ltd recently announced the ongoing BATURA Phase 3b trial evaluating AstraZeneca’s Airsupra (albuterol/budesonide) in patients with intermittent or mild persistent asthma has…
Microbiotica Announces First Patient Dosed in its International Phase 1b Trial, MELODY-1 of MB097 Precision Microbiome Co-Therapy in Advanced Melanoma
Microbiotica recently announced the first patient has been dosed in its advanced melanoma (MELODY-1) trial. This international trial is due to recruit up to 40…
Cybrexa Therapeutics Announces First Patient Dosed With First-in-Class Peptide-Drug Conjugate in Phase 2 Trial in Ovarian Cancer
Cybrexa Therapeutics recently announced the first patient has been dosed in a Phase 2 clinical trial evaluating CBX-12 in patients with platinum-resistant or refractory ovarian…
Abivax Announces First Patient Enrolled in Phase 2b Trial of Obefazimod in Crohn’s Disease
Abivax SA recently announced the first patient was enrolled in its Phase 2b ENHANCE-CD (NCT06456593) trial evaluating obefazimod in patients with Crohn’s disease (CD). The…
Biodexa Provides Update on Progression Free & Overall Survival in Phase 1 Study of MTX110 in Recurrent Glioblastoma
Biodexa Pharmaceuticals PLC recently announced an update in respect of its open label Phase 1 study of MTX110 in recurrent glioblastoma. In October 2023, the…
Sterling Pharma Solutions Announces Partnership With Soligenix
Sterling Pharma Solutions and Soligenix recently announced a partnership that will see Sterling manufacture a GMP-quality synthetic hypericin developed by Soligenix, for its oncology drug,…
Recipharm Strengthens Pharmaceutical Development Capabilities With Strategic Investments
Recipharm is expanding its pharmaceutical development capabilities, through targeted investments and the integration of cutting-edge technologies. This investment bolsters its services for early- and late-…
Legend Biotech to Establish New, State-of-the-Art Cell Therapy R&D Facility in Philadelphia
Legend Biotech Corporation recently announced it is establishing a new, state-of-the-art research and development (R&D) facility in Philadelphia, PA, to advance its portfolio of next-generation…
First Patient Dosed in Phase 2 Trial of TLX250-CDx for Detection of Recurrent Kidney Cancer After Surgery
Telix Pharmaceuticals Limited recently announced the first patient has been dosed in a Phase 2 trial exploring the clinical utility of Telix’s first-in-class investigational PET1…
EVerZom Achieves First Bioproduction of Clinical-Grade Stem Cell-Derived Exosomes in Large-Scale Bioreactors
EVerZom recently announced it has reached a key milestone in its development with the production of a first exosome GMP batch in a 10L bioreactor…
Lonza Completes Acquisition of Large-Scale Biologics Site in Vacaville (US) From Roche
Lonza recently announced it has completed its acquisition of the Genentech large-scale biologics manufacturing site in Vacaville, CA, from Roche for $1.2 billion. The Vacaville…
PLATFORM TECHNOLOGY - Developing Novel Antisense Oligonucleotides for Neurodegenerative Diseases
Scott Schobel, MD, and Nicole Datson, PhD, are hopeful their novel platform for targeting disease-causing RNA with multiple modulating approaches will lead to major advances in treating genetic disease and improving the lives of patients and their families in the years ahead.
Abzena Expands Partnership With ProteoNic to Include 2G UNic Technology as Standard Offering for All Customers
ProteoNic recently announce an expanded partnership with Abzena, the leading end-to-end integrated CDMO for complex biologics and bioconjugates. Following the successful launch of AbZelectPRO, a high-producing…
Sagimet Receives FDA Breakthrough Therapy Designation for Denifanstat in MASH
Sagimet Biosciences Inc. recently announced the US FDA granted Breakthrough Therapy designation to denifanstat for treatment of noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate to…
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).