Bio Platforms
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.
Organovo Partners With IIAM on Multi-Year Clinical Biomaterial Sourcing Agreement
Organovo Holdings, Inc. recently announced it has entered into a cell and tissue clinical sourcing agreement with the International Institute for the Advancement of Medicine (IIAM).
Nkarta Therapeutics Reports Potent Natural Killer Cell Anti-Cancer Activity in Preclinical Tumor Model
Nkarta Therapeutics recently announced preclinical data highlighting the anti-cancer activity of engineered NK cells in a presentation at the Society of Immunotherapy for Cancer meeting…
Alligator Bioscience & Aptevo Therapeutics Present New Preclinical Data
Alligator Bioscience and Aptevo Therapeutics Inc. recently announced new preclinical data for ALG.APV-527 at the Society for Immunotherapy of Cancer's (SITC) 33rd Annual Meeting being…
Intensity Therapeutics Highlights Data in Advanced Solid Tumors
Intensity Therapeutics, Inc. recently announced additional data from a Phase 1/2 clinical study of INT230-6, the company’s novel lead product candidate designed for direct intratumoral injection,…
Codexis Announces Results of Phase 1a Clinical Trial
Codexis, Inc. recently announced top-line results from its Phase 1a single ascending-dose study in healthy volunteers with CDX-6114, its orally administered enzyme candidate for the…
ProQR In-licenses Worldwide Rights to Ophthalmology Drug Candidate From Ionis Pharmaceuticals
ProQR Therapeutics N.V. recently announced the signing of an agreement with Ionis Pharmaceuticals to license QR-1123 (formerly IONIS-RHO-2.5Rx), an RNA medicine for autosomal dominant retinitis…
CytomX Therapeutics Presents Clinical Data From Probody Platform
CytomX Therapeutics, Inc. recently presented clinical results from two arms of the PROCLAIM (PRObody CLinical Assessment In Man) module, PROCLAIM-072. PROCLAIM-072 is an ongoing Phase…
VBI Vaccines Completes Vaccination in PROTECT Phase 3 Clinical Study
VBI Vaccines Inc. recently announced the last subject has received the last vaccination in the PROTECT Phase 3 study of Sci-B-Vac, the company’s third-generation prophylactic hepatitis B vaccine.
Principia Achieves $10 Million in Additional Milestones
Principia Biopharma Inc. recently announced the achievement of $10 million in additional milestones related to successful development activities conducted by Principia as part of the…
Athenex & Xiangxue Life Sciences Announce Preliminary Study Results
Athenex, Inc. recently announced that preliminary results of pilot studies in China in which patients received T-cell receptor affinity enhancing specific T-cell therapy (TAEST) showed encouraging…
Sarepta & Lysogene Announce Exclusive License Agreement
Sarepta Therapeutics, Inc. recently announced it has signed a license agreement with Lysogene (FR0013233475 - LYS), a pioneering biopharmaceutical company specializing in gene therapy targeting…
MeiraGTx Announces Acquisition of Vector Neurosciences
MeiraGTx Holdings Plc recently announced it has acquired Vector Neurosciences Inc. in an all-stock transaction. As a result of the acquisition, which was signed and…
Sitryx Launches With $30 Million to Develop Disease-Modifying Therapeutics
Sitryx recently announced it has closed its Series A financing round. The company, founded with seed funding from SV Health investors, raised a total of…
Amicus Therapeutics Enters Research & Development Collaboration
Amicus Therapeutics recently announced a major collaboration with the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania (Penn) to…
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).