Bio Platforms
Y-mAbs Therapeutics Announces FDA Clearance of IND
Y-mAbs Therapeutics, Inc. recently announced the US FDA has cleared the Investigational New Drug (IND) application for a humanized bispecific GD2 antibody.
Translate Bio Expands Patent Portfolio With Newly Issued US Patent Relating to its mRNA Therapeutics Platform
Translate Bio recently announced that the USPTO has issued US Patent No. 10,143,758, Liver Specific Delivery of Messenger RNA, which builds on the company’s 2009 patent filing…
Sienna Biopharmaceuticals Announces Topical Drug Study Results
Sienna Biopharmaceuticals, Inc. recently announced top-line results from a Phase 2b study of SNA-120 (pegcantratinib), the company’s lead drug candidate developed using its proprietary Topical…
GSK to Acquire Oncology Focused Biopharmaceutical Company
GlaxoSmithKline plc recently announced that the companies have entered into a definitive agreement pursuant to which GSK will acquire TESARO, an oncology focused company based…
Domain Therapeutics Signs R&D Collaboration & License Agreement With Boehringer Ingelheim
Domain Therapeutics recently announced announces a multi-target drug discovery collaboration and licensing agreement with Boehringer Ingelheim on orphan G Protein-Coupled Receptors (GPCRs) in the Central Nervous System (CNS) disease area.
SELLAS Life Sciences Announces Completion of Enrollment in Phase 2 Randomized Controlled Clinical Study
SELLAS Life Sciences Group, Inc. recently announced the completion of enrollment for a Phase 2 independent investigator-sponsored clinical trial of the combination of trastuzumab (Herceptin)…
Opthea Confirms Final Patient Enrolment in Phase 2b Wet AMD Clinical Trial
Opthea Limited recently announced the last patient has been enrolled in the company’s ongoing Phase 2b trial of OPT-302 for wet age-related macular degeneration (AMD),…
CBT Pharmaceuticals & Zhejiang Bossan Pharmaceutical Enter Collaboration & License Agreement
CBT Pharmaceuticals (CBT) and Zhejiang Bossan Pharmaceutical Co., Ltd (Bossan) recently announced they have entered into a clinical collaboration and license agreement to develop their…
Edge Therapeutics Enters Into Merger Agreement With PDS Biotechnology
Edge Therapeutics, Inc. and PDS Biotechnology Corporation recently announced their respective boards of directors have approved a definitive merger agreement. The merger is expected to…
Ajinomoto Bio-Pharma Services Introduces Bioconjugation Technology for ADC Manufacturing
Ajinomoto Bio-Pharma Services recently presented data at the World ADC Summit in San Diego this month describing a proprietary site-selective bioconjugation technology, AJICAP.
Evelo & Merck Enter Clinical Trial Collaboration
Evelo Biosciences, Inc. recently announced it has entered into a clinical trial collaboration agreement with Merck. The collaboration will evaluate EDP1503 in combination with KEYTRUDA (pembrolizumab).
OSE Immunotherapeutics & Oncology Physician Network GERCOR Announce Initiation of Phase 2 Clinical Trial
OSE Immunotherapeutics SA recently announced that the French National Agency for Medicines and Health Products Safety (ANSM) and the French Central Ethic Committee (CPP) approved…
BrainStorm Cell Therapeutics Announces Submission of IND
BrainStorm Cell Therapeutics Inc. recently announced that it has submitted an Investigational New Drug (IND) application with the US FDA to initiate a Phase 2…
Cadent Therapeutics Secures $40 Million Series B Financing to Advance Pipeline
Cadent Therapeutics recently announced it has raised $40 million in a Series B financing. In addition, the company has appointed Bob (Ibrahim) Dagher, MD, as…
Provectus Biopharmaceuticals Provides Drug Development Update
Provectus recently provided an update on the company’s small molecule clinical development program for its investigational immuno-dermatology drug PH-10 for the treatments of psoriasis and…
Aptinyx Exploratory Clinical Studies Provide First Evidence that NYX-2925 Elicits Rapid, Persistent, NMDAr-Mediated Pharmacodynamic Activity in Humans
Aptinyx Inc. recently announced that exploratory clinical studies of its novel NMDA receptor (NMDAr) modulator, NYX-2925, provide the first evidence that oral dosing of the…
Commercial Prospects for Immuno-Oncology Development Across Oncology Indications in Untapped Cancer Types or in Combinations
Immuno-Oncology (IO) development is booming across the 8MM (US, France, Germany, Italy, Spain, UK, Japan, and China) with IO agents being investigated in most cancer…
HUMAN MICROBIOME - Advancing New Frontiers in a Rapidly Emerging Market
Flo Orim, MD, PhD, focuses on biomedical aspects of research, development, and commercial endeavors in the human microbiome space, including essential background information, evolution of the field, and advances in basic research.
REGENERATIVE MEDICINE - Cell Therapy - The Quest for Finding the Cure
Aarti Chitale, MBA, believes as the therapeutic landscape evolves and transitions toward personalized and value-based care, regenerative medicine will play an increasingly important role, and moreover, the success of these therapies offers promising opportunities for previously untreated diseases areas.
GENETIC MODIFICATION THERAPIES - Clinical Applications & Technology Platforms
Laurie L. Sullivan and John Bergin, MS, MBA, say the growing interest on the part of large pharma or biotech companies is driving clinical development of genetic modification therapy candidates, and the rich late-stage pipeline for genetic modification therapy candidates is a driving force of growth.
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).