Bio Platforms
Rhythm Pharmaceuticals Optimizes Design of EMANATE & DAYBREAK Clinical Trials to Advance Setmelanotide for Rare Genetic Diseases of Obesity
Rhythm Pharmaceuticals, Inc. recently announced modifications intended to optimize its Phase 3 EMANATE and ongoing Phase 2 DAYBREAK trials to focus on rare patient populations,…
Medidata & Celsion Present Findings on Use of Synthetic Control Arm to Estimate Treatment Effect in Ovarian Cancer Trial
Celsion Corporation and Medidata recently announced they presented their findings on the use of a Synthetic Control Arm (SCA) in a completed Phase Ib dose-escalating study of GEN-1 in the neoadjuvant treatment of….
CELL & GENE THERAPY - State of the Industry – Where is C> Headed?
Bill Vincent believes if the industry wants to keep moving up the steep growth curve, then capacity and raw materials must be readily available. The innovations in therapies, platforms, and processes will all come with time, money, and increasing availability of resources.
Nurix Therapeutics Doses First Patient in Phase 1 Clinical Trial of Drug-Enhanced Cell Therapy for the Treatment of Patients With Solid Tumors
Nurix Therapeutics, Inc. recently announced the first patient has been dosed in its Phase 1 clinical trial of DeTIL-0255, a drug-enhanced tumor infiltrating lymphocyte therapy and….
Verrica Pharmaceuticals Announces First Patient Dosed in Phase 2 Study for Potential First-in-Class Oncolytic Peptide-Based Immunotherapy
Verrica Pharmaceuticals Inc. recently announced the first patient has been dosed in the company’s Phase 2 trial of LTX-315, a potential first-in-class oncolytic peptide, for the….
Processa Pharmaceuticals Announces First Patient Enrolled in Phase 2A Trial for the Treatment of Gastroparesis
Processa Pharmaceuticals, Inc. recently announced it has enrolled its first patient in the Phase 2A clinical trial of PCS12852 in patients with moderate-to-severe gastroparesis. Gastroparesis…
Agenus’ CD137 Agonist Advances in the Clinic, Triggering Milestone Payment From Gilead
Agenus Inc. recently announced receipt of a $5-million clinical milestone payment for AGEN2373 (conditionally active CD137 agonist). AGEN2373 is being evaluated in a Phase 1b…
PLATFORM TECHNOLOGY - Predictive Medicine, Biomarkers & the Multiple Unmet Needs in Acute Respiratory Distress Syndrome
Joe G.N. Garcia, MD, and Stan Miele believe the unmet need in ARDS is to identify reliable, validated ARDS biomarkers that minimize ARDS heterogeneity and allow for stratification of subject selection for enrollment in clinical trials of tailored therapies. Combined with a more streamlined drug-approval process, biomarker- and genotype-based treatment of specific ARDS endotypes has never been as within reach as it is today.
New BD Assay Advances Immunology Research Capabilities in the Developing Field of Multiomics
BD (Becton, Dickinson and Company) recently released the BD Rhapsody TCR/BCR Multiomic Assay, an innovative set of reagents that enables researchers to more easily and…
On-Demand Webinar: How to Safely Handle Your Antibody Drug Conjugate
Antibody Drug Conjugates (ADCs) have a highly specific mechanism of action which is an advantage for the treatment of several oncology indications…..
Novel Nuclear MicroRNA is Being Developed for the Treatment of Cardiovascular Disease
A novel angiogenic microRNA drug can be a new option for the treatment of ischemic cardiovascular disease, according to a new study published in PLOS…
Ocugen Announces First Patient Dosed in Phase 1/2 Clinical Trial for Gene Therapy Candidate
Ocugen, Inc. recently announced the first patient has been dosed in the Phase 1/2 clinical trial of OCU400, a modifier gene therapy candidate for the treatment of retinitis pigmentosa….
Outlook Therapeutics Submits BLA to FDA for ONS-5010 as a Treatment for Wet AMD
Outlook Therapeutics, Inc. recently announced it has submitted its Biologics License Application (BLA) to the US FDA for ONS-5010, an investigational therapy which, if approved,…
Cidara Therapeutics Announces Completion of Dosing the First Cohort of Healthy Volunteers in Phase 1 Trial
Cidara Therapeutics, Inc. recently announced the first cohort of healthy volunteers has been dosed in its Phase 1 trial of CD388, a highly potent long-acting…
Vyant Bio & OrganoTherapeutics Announce Strategic Collaboration
Vyant Bio, Inc. and OrganoTherapeutics recently announced they have entered into a collaboration agreement to work toward accelerating the discovery of drugs for the treatment of….
Qualigen Therapeutics Engages TD2 as Contract Research Organization for Preclinical Development
Qualigen Therapeutics, Inc. recently announced it has appointed the renowned contract research organization (CRO) Translational Drug Development (TD2) to lead the preclinical development of lead…
Mapi Pharma Receives US Patent Covering Methods of Suppressing or Alleviating Primary or Secondary Progressive MS With a Sustained-Release Glatiramer Acetate Depot
Mapi Pharma Ltd. recently announced it has been granted US Patent No. 11,167,003 titled Methods for suppressing or alleviating primary or secondary progressive multiple sclerosis….
eTheRNA Manufacturing Announces New LNP Formulation Development & Production Service
eTheRNA Manufacturing recently introduced a new Lipid Nanoparticle (LNP) formulation development and production service to support the discovery and early preclinical development of RNA-based….
Artizan Biosciences Announces Issuance of New US Patent to Cover Proprietary Discovery Platform
Artizan Biosciences, Inc. recently announced the US Patent and Trademark Office has issued US Patent No. 11,299,790 that broadens the use of the company’s proprietary…
Gb Sciences' Nanoparticle Encapsulation Technology Improves the Efficacy of Terpenes for Use in Chronic Pain Formulations
Gb Sciences, Inc. recently announced its sponsored study investigating the effect of nanoparticle encapsulation of three cannabis-based terpenes on their potential efficacy in pain management was….
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).