Bio Platforms
Saghmos Therapeutics Announces Notice of Allowance for Second US Patent for Phase 3-Ready Cardiorenal Metabolic Modulator
Saghmos Therapeutics, Inc. recently announced a Notice of Allowance for a second US patent for its Phase 3-ready product, ST-62516 (trimetazidine), a cardiorenal metabolic modulator.…
Anima Biotech Announces Preclinical Data of Candidate in Idiopathic Pulmonary Fibrosis
Anima Biotech recently announced positive preclinical data of its lung fibrosis candidate. This drug operates through a novel mRNA biology mechanism of action, opening new…
Immix Biopharma Awarded European Union Orphan Drug Designation for NXC-201 in AL Amyloidosis
Immix Biopharma, Inc. recently announced the European Commission (EC) has granted orphan drug designation to NXC-201 for the treatment of….
FundaMental Pharma GmbH Announces Groundbreaking Proof-of-Concept Study Using a TwinF Interface Inhibitor for the Treatment of Amyotrophic Lateral Sclerosis
FundaMental Pharma GmbH recently announced the publication of a groundbreaking study by Neurobiologists in Heidelberg using FundaMental Pharma’s small molecule TwinF interface inhibitor, FP802…..
Triumvira Immunologics Announces First Patient Dosed in Phase 1/2 Cell Therapy Trial
Triumvira Immunologics recently announced the first patient has been dosed in its TACTIC-3 trial, a Phase 1/2 study (NCT05862324) investigating the safety and efficacy of…
MiNK Therapeutics' AgenT-797 Shows Promising Results in the Treatment of Severe Acute Respiratory Distress
MiNK Therapeutics, Inc. recently announced the publication of results in Nature Communications from a Phase 1/2 study of agenT-797 in patients with moderate-to-severe acute respiratory…
Dyadic Announces Research & Development Collaboration Agreement With Global Biopharmaceutical Company
Dyadic International, Inc. recently announced it has signed a fully funded evaluation agreement including commercial option with an undisclosed leading global biopharmaceutical company to design…
Novo Holdings to Acquire Catalent
Catalent, Inc., a leader in enabling the development and supply of better treatments for patients worldwide, and Novo Holdings, a holding and investment company that is responsible for managing the assets and wealth of the Novo Nordisk Foundation, recently announced they….
Vaxart Announces Publication in Vaccines of Non-Human Primate Preclinical Data Demonstrating its Next-Generation Vaccine Candidates Elicit Mucosal & Systemic Immunogenicity & Reduce Viral Shedding After SARS-CoV-2 Challenge
Vaxart, Inc. recently announced the publication of preclinical non-human primate data demonstrating the potential of its COVID-19 vaccine to protect against multiple SARS-CoV-2 variants of…
Purple Biotech Reaches Recommended Phase 2 Dose for NT219
Purple Biotech Ltd. recently announced it has determined 100mg/kg is the recommended Phase 2 dose (RP2D) for NT219 in combination with cetuximab in the treatment…
Denali Therapeutics Announces Presentations on its Investigational Blood-Brain Barrier-Crossing Enzyme Replacement Therapies
Denali Therapeutics Inc. recently announced upcoming presentations from its Enzyme Transport Vehicle (ETV) development programs, tividenofusp alfa (DNL310) and DNL126 (ETV:SGSH), to be given at…
AustinPx Partners With Microsize on KinetiSol Technology, Partnership Strengthens Commercialization Options for AustinPx’s Clients
AustinPx and Microsize recently announced the strategic partnership to accelerate the commercial application of AustinPx’s KinetiSol Technology platform. The collaboration will enable….
BioNTech & DualityBio Receive FDA Fast Track Designation for Next-Generation Antibody-Drug Conjugate Candidate
BioNTech SE and Duality Biologics recently announced the US FDA granted Fast Track designation for BNT325/DB-1305 for the treatment of patients with platinum-resistant ovarian epithelial cancer….
Vaxxinity Announces Research Collaboration on Active Immunotherapies for Neurodegenerative Diseases With University of Florida
Vaxxinity, Inc. recently announced a research collaboration with the University of Florida’s (UF) Center for Translational Research in Neurodegenerative Disease (CTRND) to support its work on…
Jnana Therapeutics Announces Positive Clinical Proof of Concept Achieved With Potential First-in-Class Oral Treatment for PKU
Jnana Therapeutics recently announced positive, statistically significant interim results from its ongoing clinical study of JNT-517 in individuals with phenylketonuria (PKU). JNT-517, a small molecule inhibitor…
Oxford Biomedica Completes Acquisition of ABL Europe
The acquisition of ABL Europe, a pure-play European CDMO, strengthens Oxford Biomedica’s position as a world-leading cell and gene therapy CDMO. This strategic move broadens….
Longeveron Receives Notice of US Patent Allowance for the Technology Behind its Lead Investigation Product Lomecel-B
Longeveron Inc. recently announced it received a notice of patent allowance from the United States Patent and Trademark Office (PTO) for Medicinal Signaling Cells (MSCs),…
SPECIAL ROUNDTABLE - Leadership Panel: 4 Trends That Will Have the Most Impact on Drug Development in 2024
Contributor Cindy H. Dubin asked some of today’s life science leaders what they expect will have the greatest impact on drug development in 2024 and beyond.
DRUG DISCOVERY - Overcoming Traditional Challenges: Innovative Chemoproteomics Strategies to Revolutionize Drug Discovery
Ping Cao, PhD, and Irene Yuan believe their chemoproteomic platform offers a distinct advantage by enabling the simultaneous exploration of the entire proteome, setting it apart from other drug discovery technologies that typically focus on individual targets.
DRUG DEVELOPMENT - Using a Novel Deep Cyclic Inhibition Mechanism to Treat Broad Range of RAS-Mutant Cancers
Ben Zeskind, PhD, addresses the question: rather than targeting individual RAS mutations and treating chronically, is it possible to achieve broad therapeutic activity in a way that focuses on malignant cells while minimizing damage to healthy cells?
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).