Bio Platforms
Caribou Biosciences Announces $25-Million Equity Investment From Pfizer
Caribou Biosciences, Inc. recently announced Pfizer Inc. has made a $25-million equity investment in the company. Pfizer purchased 4,690,431 of Caribou common shares at a price of….
Akari Therapeutics Receives Positive Opinion on Orphan Drug Designation From EMA for Nomacopan for Treatment in Hematopoietic Stem Cell Transplantation
Akari Therapeutics, Plc recently announced the European Medicines Agency (EMA) has issued a positive opinion on the company’s application for orphan drug designation for nomacopan…
Ardena & RiboPro Forge Strategic Alliance to Support the RNA Revolution With End-to-End Manufacturing of Advanced mRNA & LNP Solutions
Leading contract development manufacturer of nanomedicines, Ardena, has joined forces with mRNA and LNP technology specialists RiboPro to form a new strategic commercial alliance aimed at…
Zealand Pharma Submits NDA to US FDA for Dasiglucagon in Congenital Hyperinsulinism
Zealand Pharma A/S recently announced the submission of a New Drug Application (NDA) to the US FDA for dasiglucagon for the prevention and treatment of…
Oxford BioTherapeutics Initiates Phase 1b Clinical Trial for OBT076 in Patients With Advanced Solid Tumors
Oxford BioTherapeutics recently announced it has initiated a Phase 1b combination trial evaluating its lead candidate, OBT076 - a CD205 targeting ADC - and Agenus’…
F-star Announces Strategic Collaboration & License Agreement With Takeda
F-star Therapeutics, an invoX company, recently announced it has entered into a strategic discovery collaboration and licensce agreement with….
Palisade Bio Announces Completion of Enrollment in LB1148 Dose Optimization Study
Palisade Bio, Inc. recently announced the completion of patient enrollment in its dose optimization study of LB1148. “Achieving our enrollment target in this study is an…
Enterome Announces First Patient Dosed in Phase 1/2 Trial With Third OncoMimics Immunotherapy in Metastatic Colorectal Cancer
Enterome recently announced the first patient was dosed in the Phase 1/2 AUDREY trial evaluating EO4010, the company’s third OncoMimics candidate, for the treatment of patients with….
WHITEPAPER - PLGA Nanoparticles - Bridging the Gap From R&D to GMP
Poly(lactic-co-glycolic acid) (PLGA) has emerged as a promising material for drug delivery and biomedical applications. Its exceptional biocompatibility, customizable degradation and release properties, and versatility have led to….
Harpoon Therapeutics Announces Completion of Planned Patient Enrollment in Phase 1 Study of HPN217 in Relapsed/Refractory Multiple Myeloma
Harpoon Therapeutics, Inc. recently announced the completion of planned patient enrollment in the Phase 1 dose escalation study evaluating the safety, tolerability, and pharmacokinetics of…
Inozyme Pharma Announces Dosing of First Infant With ENPP1 Deficiency in Phase 1b Trial
Inozyme Pharma, Inc. recently announced dosing of the first patient in its ENERGY-1 trial, a Phase 1b clinical trial of INZ-701 in infants with ENPP1 Deficiency.…
Ascendia Pharmaceuticals Develops Novel Nanoemulsion IV Formulation for Clopidogrel That Received IND Approval
Ascendia Pharmaceuticals CEO Jim Huang, PhD, recently announced Ascendia’s patented Emulsol® nanotechnology has been used to develop a novel nanoemulsion IV formulation for….
MoonLake Immunotherapeutics Achieves Landmark Milestone With Positive Phase 2 Results for Nanobody Sonelokimab in Hidradenitis Suppurativa
MoonLake Immunotherapeutics recently announced positive top-line results from its global Phase 2 MIRA trial evaluating the efficacy and safety of the Nanobody sonelokimab in patients with moderate-to-severe….
Larkspur Biosciences Announces First-in-Class Program Targeting Novel B Cell Checkpoint
Larkspur Biosciences recently reported one of their lead programs is a first-in-class B cell checkpoint, TIM-1. This announcement follows the publication by Larkspur co-founder Vijay…
Clene Announces Publication of Phase 2 CNM-Au8 Clinical Data for the Treatment of ALS
Clene Inc. and its wholly owned subsidiary Clene Nanomedicine, Inc. recently announced The Lancet’s journal eClinicalMedicine has published combined detailed analyses of the Phase 2…
Ocean Biomedical Announces US Patent Issued for Bispecific Cancer Immunotherapy Treatment With Demonstrated Killing of Tumor Cells in Lung Cancer, Brain Cancer (Glioblastoma) & Metastatic Melanoma
Patent broadens protection around Ocean Biomedical’s first-in-class anti-CHi3L1 antibody to include new bispecific approaches targeting immune checkpoints, with potential application in….
XOMA Acquires Royalty & Milestone Economics to Phase 3 First-In-Class Orphan Disease Asset for Niemann-Pick Disease Type C (NPC) & Phase 2 Oncology Asset
XOMA Corporation recently announced it has acquired the royalty and milestone rights associated with two assets from LadRx Corporation: arimoclomol, an oral therapeutic for Niemann-Pick…
ASLAN Pharmaceuticals Enters Strategic Licensing Deal With Zenyaku Kogyo for the Development & Commercialization of Eblasakimab
ASLAN Pharmaceuticals and Zenyaku Kogyo Co., Ltd., a subsidiary of privately held Zenyaku Holdings Co., Ltd. recently announced a strategic licensing agreement granting Zenyaku exclusive…
Lilly to Acquire DICE Therapeutics to Advance Innovation in Immunology
Eli Lilly and Company and DICE Therapeutics, Inc. recently announced a definitive agreement for Lilly to acquire DICE, a biopharmaceutical company that leverages its proprietary….
SCYNEXIS Announces Achievement of First Development Milestone of $25 Million Under Exclusive License Agreement With GSK
SCYNEXIS, Inc. recently announced the achievement of a $25-million performance-based development milestone under its exclusive license agreement with GSK for….
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).