Bio Platforms
Qualigen Therapeutics Extends Research Agreement on RAS Program
Qualigen Therapeutics, Inc. recently announced the mutual extension of a research agreement with University of Louisville Research Foundation (ULRF). The revised agreement expands the company’s…
BioMed X Institute & Merck KGaA Start Second Research Project in Immunology
BioMed X recently announced the start of its new research project Regulatory T Cell Dysfunction in Autoimmunity and Inflammaging (TDA) in collaboration with Merck KGaA, Darmstadt,…
OncoArendi Therapeutics Announces License Option Agreement With University of Michigan
OncoArendi Therapeutics S.A. recently announced it has entered into an option-to-license agreement with Innovation Partnerships at the University of Michigan (U-M) to develop novel small…
NeuBase Therapeutics Presents New Preclinical Data for its Myotonic Dystrophy Type 1 Program Demonstrating Splice Rescue, Nuclear Aggregate Resolution & Myotonia Reversal
NeuBase Therapeutics, Inc. recently announced new preclinical data for its lead development candidate, NT-0231.F, to treat myotonic dystrophy type 1 (DM1). ….
Oxford Biomedica Broadens its Viral Vector Capabilities With the Launch of Oxford Biomedica Solutions
Oxford Biomedica plc recently announced it has completed its deal with Homology Medicines Inc. to establish Oxford Biomedica Solutions LLC, a new US-based full scope,…
Synthetic Biologics Completes Acquisition of VCN Biosciences
Synthetic Biologics, Inc. recently announced it has completed the acquisition of VCN Biosciences, S.L. (VCN) following the satisfaction of all closing conditions…..
Denali Therapeutics Announces Initiation of Phase 1/2 Clinical Trial
Denali Therapeutics Inc. recently announced dosing has begun in a Phase 1/2 clinical trial of DNL593 (PTV:PGRN) for the potential treatment of frontotemporal dementia (FTD)…
Vibalogics Completes Major Milestone in $50-Million Facility Expansion
Vibalogics has recently announced the completion of its expansion project at its GMP facility in Cuxhaven, Germany. The new building features biosafety level (BSL) 2…
Novartis, Voyager Therapeutics Reach License Option Agreement
Novartis recently announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three….
BioNTech & Regeneron Expand Strategic Collaboration
BioNTech SE recently announced the expansion of its strategic collaboration with Regeneron to advance the company’s FixVac candidate BNT116 in combination with Libtayo (cemiplimab), a PD-1 inhibitor….
Atossa Therapeutics Announces Issuance of Key US Patent Covering Endoxifen
Atossa Therapeutics, Inc. recently announced the US Patent and Trademark office has issued a new patent further strengthening Atossa’s intellectual property in its proprietary therapy…
Valneva Successfully Completes Pivotal Phase 3 Trial of Single-Shot Chikungunya Vaccine Candidate
Valneva SE recently announced the successful completion of the Phase 3 pivotal trial of its single-shot chikungunya vaccine candidate, VLA1553. The positive final analysis included…
VYNE Reports Positive Preclinical Data for Lead BET Inhibitor in Human Skin Model of Vitiligo
VYNE Therapeutics Inc. recently announced positive preclinical data in an ex vivo skin model of vitiligo. In the preclinical model, pan-bromodomain and extra-terminal (BET) inhibitor…
Targovax ASA & Agenus Announce Collaboration on Mutant KRAS Cancer Vaccine Adjuvanted With QS-21 STIMULON
Agenus and Targovax ASA recently announced they have entered into a clinical collaboration and supply agreement to combine Targovax’s TG mutant KRAS cancer vaccines with Agenus´s clinically validated….
Harpoon Therapeutics Granted Orphan Drug Designation for Small Cell Lung Cancer Treatment
Harpoon Therapeutics, Inc. recently announced the US FDA has granted Orphan Drug Designation for HPN328, a delta like ligand 3- (DLL3) targeting TriTAC, for the…
Evaxion Biotech Completes Recruitment for Phase 1/2a Clinical Trial
Evaxion Biotech A/S has recently now finalized recruitment for Phase 1/2a clinical trial of EVX-02 in adjuvant melanoma patients…..
Fulcrum Therapeutics Announces Phase 3 Clinical Trial
Fulcrum Therapeutics, Inc. recently announced its plans to initiate REACH, a Phase 3 clinical trial of losmapimod in people with facioscapulohumeral muscular dystrophy (FSHD), in…
Aravive Announces Positive Updated Data & New Biomarker Data From Phase 1b Study
Aravive, Inc. recently announced positive new data from the Phase 1b portion of the ongoing Phase 1b/2 trial of batiraxcept in clear cell renal cell…
Harpoon Therapeutics Receives FDA Fast Track Designation
Harpoon Therapeutics, Inc. recently announced the US FDA has granted Fast Track designation to HPN217, a BCMA-targeting TriTAC, for the treatment of patients with relapsed,…
SPECIAL FEATURE - Solubility & Bioavailability: Utilizing Enabling Technologies
Contributor Cindy H. Dubin interviews several leading companies on how they are using innovative technologies, such as lipid nanoparticles to achieve a high drug loading, combining anti-solvent continuous crystallization with micro-mixing technology to control crystallization and reduce crystal size, and how a robotic capsule can improve bioavailability in the range of 47% to 78%.
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).