Bio Platforms
CureVac Swiss AG Initiates Rolling Submission Process for mRNA-Based COVID-19 Vaccine Candidate
The CureVac Swiss AG, the Swiss subsidiary of CureVac N.V. recently announced initiation of a rolling submission for CVnCoV, the company’s mRNA-based COVID-19 vaccine….
XOMA’s Royalty Portfolio Grows With Addition of Three Royalty Assets
XOMA Corporation recently announced its portfolio of potential future milestone and royalty assets has increased with the addition of three Affimed N.V. innate cell engager…
New Biomaterial Regrows Blood Vessels & Bone According to RCSI Research
Scientists have developed a new biomaterial that regrows blood vessels and bone, potentially providing a single-stage approach when repairing large bone defects. The study, led…
Longeveron Announces Successful Completion of Phase 1/2 Clinical Study
Longeveron Inc. recently announced the completion of the company’s Phase 1/2 clinical study of the use of Lomecel-B to improve immune response to influenza (flu)…
LEXEO Therapeutics Announces FDA Fast-Track Designation
LEXEO Therapeutics recently announced the US FDA has granted Fast Track designation to LX1001, the company’s adeno-associated virus (AAV) mediated gene therapy program, for the…
Catalent Adds Cryogenic Capabilities at Philadelphia Clinical Supply Services Facility to Meet Growing Demand for Cell & Gene Therapy Development
Catalent recently announced it has made an investment to expand capabilities at its clinical supply services facility in Philadelphia to support sponsors developing cell and…
Context Therapeutics & Integral Molecular Enter Collaboration & License Agreement
Context Therapeutics and Integral Molecular recently announced a research collaboration and licensing agreement for the development of an anti-claudin 6 (CLDN6) bispecific monoclonal antibody….
AzurRx BioPharma Engages PPD to Manage Clinical Trial for Niclosamide as Treatment for Grade 1 Immune Checkpoint Inhibitor-Associated Colitis
AzurRx BioPharma, Inc. recently announced it has entered into an agreement with PPD, Inc. for its planned Phase 1b/2a clinical trial evaluating proprietary formulations of micronized….
SOTIO Demonstrates Strong Potential of SOT102 (ADC Targeting Claudin 18.2) for Treatment of Solid Tumors in Preclinical Studies
SOTIO recently announced new preclinical data of its antibody-drug conjugate (ADC), SOT102 (formerly SO-N102), for the treatment of solid tumors in a virtual….
Artelo Biosciences Doses First Patient in CAReS Study for the Treatment of Cancer-Related Anorexia & Weight Loss
Artelo Biosciences, Inc. recently announced the first patient has been dosed in the company’s Phase 1/2 Cancer Appetite Recovery Study (CAReS) of ART27.13….
Calixar Invests in Pipeline of Highly Druggable Membrane Protein Targets & Native Antigens
Calixar recently announced its €1M ($1.2M) investment in a new pipeline of complex therapeutic targets and native antigens of high relevance for pharmaceutical companies. This…
Longeveron Announces Positive Results of Phase 1 Clinical Study of Lomecel-B Cell Therapy
Longeveron Inc. recently announced the final results of its Phase 1 clinical study evaluating the safety and efficacy of intravenous (IV) administration of Lomecel-B, an…
Akston Biosciences Launches Phase 1/2 Clinical Trial of Second-Generation COVID-19 Vaccine
Akston Biosciences Corporation recently announced the first participants have been dosed in an open-label trial of AKS-452, its COVID-19 vaccine candidate. The trial is managed…
Catalent Biologics Completes Madison Facility Expansion to Double Clinical & Commercial Drug Substance Capacity
Catalent recently announced it has completed the expansion of two new suites at its biologics drug substance development and manufacturing facility in Madison, WI, and…
Akouos Receives Orphan Drug & Rare Pediatric Disease Designations
Akouos, Inc. recently announced the US FDA has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for AK-OTOF, a gene therapy…
Yumanity Therapeutics Announces Study Demonstrating In Vivo Efficacy of YTX-7739 in a Glioblastoma Multiforme (GBM) Mouse Model
Yumanity Therapeutics recently announced results of a study that demonstrate in vivo efficacy, including increased median overall survival, of YTX-7739 in a mouse model for…
AzurRx BioPharma Initiates Phase 2 Clinical Trial of Niclosamide for the Treatment of COVID-19 Gastrointestinal Infections
AzurRx BioPharma, Inc. recently announced it has initiated its Phase 2 RESERVOIR clinical trial of a proprietary oral formulation of micronized niclosamide (FW-1022) for the…
uniQure Announces Completion of Enrollment in First Cohort of Phase 1/2 Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
uniQure recently announced the completion of patient enrollment in the first dose cohort of a randomized, double-blinded, Phase 1/2 clinical trial of AMT-130 for the…
New England Journal of Medicine Publishes Results of the PULSAR Phase 2 Trial of Sotatercept in Patients With Pulmonary Arterial Hypertension
Acceleron Pharma Inc. recently announced the New England Journal of Medicine has published results of the PULSAR Phase 2 trial of sotatercept in patients with pulmonary…
I-Mab & ABL Bio Announce First Patient Dosed in Phase 1 Trial of Bispecific Antibody TJ-L14B/ABL503 in Patients With Advanced or Metastatic Solid Tumors
I-Mab and ABL Bio, Inc. recently announced the first patient has been dosed in a Phase 1 trial for bispecific antibody TJ-L14B/ABL503. The Phase 1 clinical trial is an open-label….
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).