Bio Platforms
Tagworks Pharmaceuticals Announces Initiation of Third Dose Level in Ongoing Phase 1 Clinical Trial for Solid Tumor Treatment
Tagworks Pharmaceuticals BV recently announced the third dose level has been initiated in the ongoing Phase 1 clinical trial evaluating TGW101, a first-in-class antibody-drug conjugate…
EXECUTIVE INTERVIEW - ALZpath: A Diagnostic Assay Changing the Future of Alzheimer’s Disease
Jacob Hunter, Chief Business Officer, ALZpath, Inc., discusses the development and deployment of the company’s proprietary pTau217 antibody, bridging the gap between detection and treatment to improve patient outcomes globally.
Lexicon Pharmaceuticals Announces Submission of Additional Data to FDA Supporting the Benefit-Risk Profile of Zynquista in Type 1 Diabetes
Lexicon Pharmaceuticals, Inc. recently announced it has submitted additional clinical data to the US FDA from ongoing third-party funded, investigator-initiated studies supporting the potential resubmission…
Novonesis & Novo Nordisk to Explore New Solutions for Metabolic Health
Novonesis and Novo Nordisk have entered a research partnership to explore the role of the gut microbiome in the maintenance of metabolic health. The gut…
TARGET DISCOVERY PLATFORM - Multi-Omics, SITESEEKER® Technology & the Future of Targeted Protein Degradation
Alberto Moreno, PhD, and Laura Butler, PhD, discuss the challenges associated with gene editing technologies, such as gene-trap, RNAi, and CRISPR technologies, as they do not directly provide functional characterization or inform on the presence of well-defined drug pockets or enzymatic activity of the target genes.
Onco3R Therapeutics Announces Approval of First Clinical Trial Application for Novel SIK3 Inhibitor O3R-5671 in Autoimmune Diseases
Onco3R Therapeutics recently announced the approval of a Clinical Trial Application (CTA) by the Belgian regulatory authorities for its SIK3 inhibitor O3R-5671. The trial will…
MBX Biosciences Doses First Participant in Phase 1 Trial of MBX 4291 for the Treatment of Obesity
MBX Biosciences, Inc. recently announced dosing of the first participant in its Phase 1 trial of MBX 4291, the company’s Precision Endocrine Peptide (PEP) glucagon-like…
Opus Genetics Doses First Patient in Pivotal LYNX-3 Phase 3 Trial Evaluating Phentolamine Ophthalmic Solution 0.75% in Keratorefractive Patients With Visual Disturbances Under Mesopic, Low-Contrast Conditions
Opus Genetics, Inc. recently announced the first patient has been dosed in LYNX-3, the company’s pivotal Phase 3 clinical trial evaluating Phentolamine Ophthalmic Solution 0.75%…
Regen BioPharma Explores Expanded Use of HemaXellerate for Chemotherapy-Induced Bone Marrow Suppression
Regen BioPharma, Inc. recently announced its ongoing efforts to expand the clinical development of HemaXellerate, its novel cellular therapy, to address chemotherapy-induced bone marrow suppression,…
Artelo Biosciences Announces Positive Interim Phase 2 CAReS Results for the Treatment of Cancer Anorexia-Cachexia Syndrome
Artelo Biosciences, Inc. recently announced highly encouraging interim results from its Phase 2 Cancer Appetite Recovery Study (CAReS) trial with ART27.13, the Company’s peripherally acting cannabinoid…
CG Oncology Completes Enrollment in PIVOT-006
CG Oncology, Inc. recently announced it has completed enrollment in its Phase 3, randomized, open-label PIVOT-006 study comparing adjuvant intravesical cretostimogene grenadenorepvec versus surveillance following…
Medicus Pharma Ltd. Completes Acquisition of Antev Limited
Medicus Pharma Ltd., a biotech/life sciences company focused on advancing the clinical development programs of novel and potentially disruptive therapeutic assets, and Antev Limited, a…
Propanc Biopharma Announces Plan to Acquire $100 Million of Ethereum
Propanc Biopharma, Inc. recently announced a plan to acquire $100 million of Ethereum over the next 12 months. The asset purchase of Ethereum will enhance…
Zenas BioPharma & Royalty Pharma Enter Obexelimab Funding Agreement for up to $300 Million
Zenas BioPharma, Inc. and Royalty Pharma plc recently announced Royalty Pharma will provide up to $300 million in funding in exchange for a royalty on…
Encoded Therapeutics Announces Regenerative Medicine Advanced Therapy Designation Granted by FDA for ETX101 in SCN1A+ Dravet Syndrome
Encoded Therapeutics Inc. recently announced the US FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ETX101 following review of preliminary seizure data from…
Step Pharma Announces First Participant Dosed in a Phase 1b Trial of Dencatistat for Essential Thrombocythaemia
Step Pharma recently announced the first participant has been dosed with its lead asset dencatistat in a phase 1b clinical trial for essential thrombocythaemia (ET).…
OMass Therapeutics Enters Exclusive Collaboration & License Agreement With Genentech
OMass Therapeutics recently announced it has entered into an exclusive collaboration and license agreement with Genentech for the rights to develop and commercialize OMass’ preclinical…
Sotagliflozin Provides Consistent Relative Risk Reduction in Heart Failure & Major Cardiovascular Events Across All Age Ranges, Including Greater Than 75
Lexicon Pharmaceuticals, Inc. recently announced a post-hoc analysis (Efficacy of Sotagliflozin Among Older Adults: A Pooled Analysis of SCORED and SOLOIST-WHF) of clinical data was…
TOLREMO Therapeutics Receives Two FDA Fast Track Designations
TOLREMO therapeutics AG recently announced its lead candidate, TT125-802, received two Fast Track designations from the US FDA for the treatment of non-small cell lung…
Wugen Secures $115 Million to Advance Pivotal Study of First-in-Class Allogeneic CAR-T Therapy
Wugen, Inc. recently announced the closing of $115 million equity financing led by Fidelity Management & Research Company, with participation from RiverVest Venture Partners, Lightchain Capital,…
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).