Bio Platforms
XOMA Enters Agreement to Acquire Kinnate Biopharma
Upon closing, XOMA anticipates adding approximately $9.5 million in cash to its balance sheet and several early-stage programs to potentially add to its royalty portfolio….
Voyager Therapeutics Reports Robust Preclinical Activity in Tau Silencing Gene Therapy Program for Alzheimer’s Disease; Advances Program Into Late Research
Voyager Therapeutics, Inc. recently announced new data from its two preclinical programs targeting pathological tau for the treatment of Alzheimer’s disease. Data on VY-TAU01, Voyager’s…
Monopar Receives Clearance to Proceed With First-in-Human Phase 1 Trial of Novel Radiopharmaceutical MNPR-101-Zr in Advanced Cancers
Monopar Therapeutics Inc. recently announced it has received Human Research Ethics Committee (HREC) clearance in Australia to commence a Phase 1 dosimetry trial of its…
Immuneering Receives FDA Fast Track Designation for IMM-1-104 in Pancreatic Cancer
Immuneering Corporation recently announced the US FDA granted Fast Track designation for its lead clinical-stage program, IMM-1-104, for the treatment of patients with pancreatic ductal…
Mestag Therapeutics Enlists Leading Cancer Biology & Immunology Advisors to Support Clinical Development of its Lead Oncology Program
Mestag Therapeutics recently announced the appointments of additional distinguished, world-leading advisors to guide the development of its lead program M300, a first-in-class antibody program designed…
OrphAI Therapeutics Receives Orphan Drug Designation for AIT-101 as a Treatment for Amyotrophic Lateral Sclerosis in the European Union
OrphAI Therapeutics Inc. recently announced it has received Orphan Drug Designation (ODD) in the European Union (EU) for the treatment of amyotrophic lateral sclerosis (ALS).…
Alvotech Announces Expected Global Market Entry Dates for Biosimilar to Stelara
Alvotech recently announced it has reached settlement agreements with Johnson & Johnson in Japan, Canada, and in the European Economic Area (EEA) for AVT04, a…
Vaxxinity’s Cholesterol Vaccine Candidate Successfully Lowers LDL-C: Preclinical Data Published
Preclinical data published in the Journal of Lipid Research indicate VXX-401 is well-tolerated, with no safety signals observed, and robustly reduces LDL-C in cynomolgus monkeys…..
TILT Biotherapeutics Awarded Grant from US Department of Defense for Ovarian Cancer Immunotherapy Research
TILT Biotherapeutics recently announced it has been selected by the US Department of Defense (DOD), America’s largest government agency, to receive a $2-million grant for…
NovelMed's Ruxoprubart Receives FDA Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria Treatment
NovelMed recently announced the US FDA has granted Orphan Drug Designation (ODD) to Ruxoprubart, an alternative pathway (AP) blocker anti-Bb antibody, for the treatment of…
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…
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).