Bio Platforms
Akari Therapeutics & Peak Bio Announce Definitive Agreement to Merge as Equals
Akari Therapeutics, Plc and Peak Bio Inc. recently announce a definitive agreement to merge as equals in an all-stock transaction. The combined entity will operate…
Elizabeth Hickman to Succeed Tim Scott as AustinPx CEO
AustinPx, developers of the next generation amorphous dispersion platform, KinetiSol Technology, and a contract development and manufacturing organization (CDMO), recently announced Elizabeth Hickman, Chief Business Officer, will succeed….
Indaptus Therapeutics Announces Positive Results From Second Cohort of Phase 1 Trial; Company Initiates Multi-Dose Cohort
Indaptus Therapeutics, Inc. recently announced positive results from the second cohort of its Phase 1 INDP-D101 trial. Patients continue to exhibit a broad immune response…
Unicycive Therapeutics Announces Orphan Drug Designation Granted for UNI-494 for the Prevention of Delayed Graft Function in Kidney Transplant Patients
Unicycive Therapeutics, Inc. recently announced the US FDA has granted orphan drug designation (ODD) to UNI-494 for the prevention of Delayed Graft Function (DGF) in kidney….
Neurogene Announces Expansion & Plans for More Rapid Patient Enrollment of Rett Syndrome Gene Therapy Clinical Trial
Neurogene Inc. recently announced the expansion of its ongoing Phase 1/2 gene therapy clinical trial for NGN-401 for female pediatric patients with Rett syndrome and…
GRI Bio Receives MHRA Authorization to Conduct Phase 2a Biomarker Study Evaluating GRI-0621 in the UK
GRI Bio, Inc. recently announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to initiate…
Fortress Biotech & Cyprium Therapeutics Announce $4.1-Million Grant From NINDS to Further Develop Gene Therapy for Menkes Disease
Cyprium Therapeutics, Inc. recently announced the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH) has awarded a 3-year…
Kinnate Biopharma Sells Investigational Pan-RAF Inhibitor to Pierre Fabre Laboratories
Kinnate Biopharma Inc. and Pierre Fabre Médicament, SAS recently announced their agreement to the sale of the company’s investigational pan-RAF inhibitor, exarafenib, and other pan-RAF…
DRUG DEVELOPMENT EXECUTIVE - Thermo Fisher Scientific: What to Expect From the Next Wave of RNA-Based Therapeutics
Dr. Dale Patterson, Vice President and General Manager, Molecular Biology at Thermo Fisher Scientific, delves into the world of RNA therapies and understand their next act beyond COVID-19.
Apalutamide Study Again Demonstrates the Advantages of Nanoforming Over Traditional Cancer Treatment Formulations
Nanoform Finland Plc recently announced it had received positive results from its own preclinical, in-vivo study of a nanocrystalline-enabled apalutamide oral formulation, which shows potential…
VACCINE DEVELOPMENT - A Healthier Global Population Through a Universal Influenza & Multi-Virus Vaccine
Jeff Fischer, MBA, says we need to have an eye toward universal influenza or multi-virus vaccines that have the ability to provide broad coverage as viral strains continue to evolve.
ENGINEERING BIOLOGY - Scaling Engineering Biology to Accelerate Advances in Healthcare
Raquel Sanches-Kuiper and Matthew Hayes, PhD, say the development of a benchtop synthesis platform has the potential to expedite the discovery and development time for drugs and biotherapeutics, greatly accelerating the rate at which engineering biology can shape the future of healthcare.
BioCina & NovaCina Forge Strategic Alliance to Offer Biologics Developers Integrated Drug Substance & Drug Product Solutions
BioCina Pty Ltd. and NovaCina Pty Ltd. recently announced a strategic partnership to provide integrated drug substance and drug product solutions for….
Dyadic Announces Strategic Partnership Agreement to Develop Affordable Rabies Prophylactics & Vaccines Using its Proprietary C1 Cell Microbial Protein Production Platform
Dyadic International, Inc. recently announced its Dutch subsidiary, Dyadic Nederland BV has entered into a strategic partnership agreement and collaboration with Rabian BV, a Dutch…
Memorial Sloan Kettering Cancer Center Now Enrolling Patients in Phase 1/2 Clinical Trial of IMUNON’s IMNN-001 in Combination With Bevacizumab in Advanced Ovarian Cancer
IMUNON, Inc. recently announced Memorial Sloan Kettering Cancer Center has joined MD Anderson Cancer Center in enrolling patients in a Phase 1/2 clinical trial evaluating…
ABVC's New Horizon for Oncology & Hematology Aims at Cancer Market of Several Hundred Billion Dollars
ABVC BioPharma, Inc. recently provided an update on its development of combination therapy for the treatment of cancer and immunity booster dietary supplements. The company is currently marketing….
Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich’s Ataxia in Collaboration With Neurocrine Biosciences, Triggering Milestone Payment
Voyager Therapeutics, Inc. recently announced the joint steering committee with its collaborator Neurocrine Biosciences has selected a lead development candidate in the Friedreich’s ataxia (FA) program. The candidate combines….
MoonLake Immunotherapeutics Announces Positive Feedback From FDA & EMA on Regulatory Path for Phase 3 Program
MoonLake Immunotherapeutics recently announced the successful outcome of its end-of-Phase 2 interactions with the US FDA as well as positive feedback from its interactions with…
Lirum Therapeutics Presents Positive Data on Novel Clinical-Stage Targeted Therapy
Lirum Therapeutics, Inc. recently announced today it presented positive data on LX-101, a novel clinical-stage targeted therapy directed to the insulin growth factor-1 receptor (IGF-1R),…
LIXTE Biotechnology Enters Exclusive Immune Oncology Patent License Agreement With NINDS & NCI
LIXTE Biotechnology Holdings, Inc. recently announced the signing of an exclusive patent license agreement with the National Institute of Neurological Disorders and Stroke (NINDS) and National…
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).