Bio Platforms
Zai Lab Announces First Patient Treated in PANOVA-3 Phase 3 Pivotal Trial of Tumor Treating Fields in Pancreatic Cancer
Zai Lab Limited recently announced treatment of the first patient in Greater China in the PANOVA-3 trial, a Phase 3 pivotal trial of Tumor Treating…
SK Inc., the Second Largest Conglomerate in South Korea, Has Invested $350 Million in the Center for Breakthrough Medicines
The Center for Breakthrough Medicines recently announced it has received $350 million in equity financing from SK Inc.. CBM is partnering with SK to create…
Sana Biotechnology, IASO Biotherapeutics, and Innovent Biologics Announce Non-Exclusive License Agreement for Clinically Validated BCMA CAR Construct
Sana Biotechnology, Inc., IASO Biotherapeutics, and Innovent Biologics recently announced the companies entered into an agreement pursuant to which Sana obtained from IASO Bio and Innovent non-exclusive commercial rights to a clinically validated fully-human BCMA CAR construct for use in certain….
NeoCura & PhoreMost Announce Research Collaboration to Explore Novel Cancer Therapeutics
NeoCura Bio-Medical Technology Co., Ltd. and PhoreMost Limited recently announced an oncology drug discovery research collaboration. As part of the collaboration, PhoreMost’s SITESEEKER phenotypic screening platform and NeoCura’s full-process RNA drug design platform will….
Black Diamond Therapeutics Announces FDA Allowance of IND Application for a MasterKey Inhibitor of EGFR for the Treatment of Gliobastoma & Non-Small Cell Lung Cancer
Black Diamond Therapeutics, Inc. recently announced the US FDA has cleared an investigational new drug (IND) application for its MasterKey inhibitor BDTX-1535, an irreversible, mutant…
Harpoon Therapeutics Provides Pipeline Development Milestones for 2022
Harpoon Therapeutics, Inc. recently provided a pipeline milestone update on its TriTAC development programs. “We are excited to begin 2022 with a catalyst rich year…
PDS Biotech Granted Patent for its Novel HPV16 Immunotherapy
PDS Biotechnology Corporation recently announced it has been granted US Patent Application No. 15,724,818 titled Novel HPV16 Non HLA-Restricted T-cell Vaccines, Composition and Methods of…
BioNTech & Crescendo Biologics Announce Global Collaboration to Develop Multi-Specific Precision Immunotherapies
BioNTech SE and Crescendo Biologics Ltd. recently announced they have entered a multi-target discovery collaboration to develop novel immunotherapies for the treatment of patients with cancer…
Fate Therapeutics Announces FDA Clearance for FT536, a First-in-class MICA/B-targeted CAR NK Cell Product Candidate for the Treatment of Solid Tumors
Fate Therapeutics, Inc. recently announced the US FDA has cleared the company’s Investigational New Drug (IND) application for FT536, an off-the-shelf, multiplexed-engineered, iPSC-derived, chimeric antigen…
4D Molecular Therapeutics Announces FDA Fast Track Designation Granted to 4D-125 for the Treatment of X-linked Retinitis Pigmentosa
4D Molecular Therapeutics recently announced the US FDA has granted Fast Track Designation for 4D-125 for treatment of patients with inherited retinal dystrophies due to defects in….
Starton Therapeutics Receives Clinical Trial Authorization in Europe to Initiate Phase 1 Clinical Trial of STAR-LLD Continuous Delivery Lenalidomide
Starton Therapeutics Inc. recently announced it has received a Clinical Trial Authorization (CTA) in the Netherlands to initiate a Phase 1 study evaluating STAR-LLD bioavailability in….
Wheeler Bio Announces $14-Million Seed Round to Expand Access to Portable CMC
Wheeler Bio, a biomanufacturing company built to accelerate the translation of therapeutic innovation into clinical impact, recently announced the closing of a $14-million Seed financing…
Veru Announces FDA Grant of Fast Track Designation for Enobosarm for the Treatment of AR+ ER+ HER2- Metastatic Breast Cancer
Veru Inc. recently announced the US FDA has granted Fast Track designation to the Phase 3 registration program for the investigation of enobosarm, a selective androgen receptor targeting agonist, for the treatment of….
Castle Creek Biosciences Acquires Novavita Thera to Expand Innovative Cell & Gene Therapy Platform
Castle Creek Biosciences, Inc. recently announced it has acquired Novavita Thera, Inc., a preclinical gene therapy company focused on rare liver and metabolic diseases. The…
Eisai Enters Into Exclusive Licensing Agreement With Roivant Concerning Investigational Anticancer Agent H3B-8800, a Splicing Modulator
Eisai Co., Ltd. recently announced it has entered into a License Agreement granting the exclusive rights for global research, development, manufacture and sale of the…
Univercells Acquires SynHelix, Entering Into Synthetic Biology & the Race to Next-Generation DNA Synthesis
Univercells S.A. (Univercells) recently announced its acquisition of SynHelix, a biotechnology company that aims to debottleneck biotherapeutics development through an unprecedented robust, scalable, and….
Sosei Heptares & Verily to Collaborate to Generate Novel Drug Candidates Against GPCR Targets for Immune-Mediated Diseases
Sosei Group Corporation and Verily recently announced they have entered into a strategic research collaboration. The research agreement brings together the….
AVITA Medical Establishes Proof-of-Concept for Novel Treatments Using Genetically Modified Skin Cells
AVITA Medical, Inc. recently announced that preclinical data successfully established proof-of-concept in two key areas of cell-based gene therapy – skin rejuvenation and….
Biomea Fusion Releases Preclinical Data With BMF-219 in Diabetes
Biomea Fusion, Inc. recently announced BMF-219 displayed remarkable activity in both the Zucker Diabetic Fatty (ZDF) Rat and the Streptozotocin-Induced Diabetes (STZ) animal models of…
Aceragen Announces $3.5-Million Development Award from Cystic Fibrosis Foundation to Progress Novel Therapy for CF-Related Exacerbations
Aceragen, Inc. recently announced that its wholly owned subsidiary, Arrevus, Inc., has received an award from the Cystic Fibrosis Foundation for up to $3.5 million…
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).