Bio Platforms
BriaCell Announces New Clinical Trial Site to Bring Novel Cancer Treatments to Advanced Breast Cancer Patients
BriaCell Therapeutics Corp. recently announced today it has added Mayo Clinic, Jacksonville, FL, as a clinical site in the Phase 1/2 study of BriaCell’s lead…
HUTCHMED Initiates a Phase 2/3 Trial of Sovleplenib for Warm Antibody Autoimmune Hemolytic Anemia
HUTCHMED (China) Limited recently announced it has initiated a Phase 2/3 trial of sovleplenib in adult patients with warm antibody autoimmune hemolytic anemia….
Silo Pharma Initiates Toxicity Study of its Proprietary Ketamine Formulation for Treatment of Fibromyalgia
Silo Pharma, Inc. recently announced it has initiated a preclinical toxicity study of its novel time-released, dosage controlled formulation of ketamine, designated as….
Two Cell & Gene Therapies Manufactured at Lonza Houston Reach US FDA Approval
Lonza recently announced two additional cell and gene therapies manufactured at its Houston (US) site have reached commercial approval in Q3 2022. ZYNTEGLO, for the…
Mustang Bio Announces First Patient Treated in Its Multicenter Phase 1/2 Clinical Trial of a First-in-Class CD20-targeted, Autologous CAR T Cell Therapy
Mustang Bio, Inc. recently announced the first patient has been treated in its multicenter, open-label, non-randomized Phase 1/2 clinical trial evaluating the safety and efficacy…
Monopar Announces Completion of Phase 2b Enrollment & Commencement of Phase 3 Enrollment in its VOICE Trial for Severe Oral Mucositis
Monopar Therapeutics Inc. recently announced an update on its actively enrolling Validive Phase 2b/3 VOICE clinical trial for the prevention of severe oral mucositis (SOM)…
MBX Biosciences Advances Phase 1 Clinical Trial of Long-Acting Parathyroid Hormone Peptide Prodrug
MBX Biosciences, Inc. recently announced it has initiated the multiple ascending dose portion of its ongoing Phase 1 clinical trial of MBX 2109. MBX 2109,…
Icosavax Initiates Phase 1 Trial of IVX-A12 Against RSV & hMPV in Older Adults
Icosavax, Inc. recently announced the initiation of a Phase 1 clinical trial of IVX-A12, a combination bivalent RSV and hMPV VLP vaccine candidate, in older…
EXTRACELLULAR VESICLES - Engineering Extracellular Vesicles to Create Next-Generation Therapeutics
David Lowe, PhD, Justin Hean, PhD, Dave Carter, PhD, and Antonin de Fougerolles, PhD, say EVs exhibit key properties that make them extremely attractive as therapeutics, particularly their safety profile and potential for low immunogenicity. In order to effectively unlock this potential, some key challenges remain, such as the development of EV product manufacture and characterization methodologies and rapid pharmacokinetics.
DRUG DISCOVERY - Getting the Most From a DNA-Encoded Library Screen
Matthew A. Clark, PhD, says despite this high level of interest, there remains variable understanding of how to maximize the potential of a DEL screen, and shares his company’s approach to DEL screening and how its partners get the most from this powerful technology.
PLATFORM TECHNOLOGY - The Evolution of Cancer Vaccines: Moving Beyond Failure & a New Era for Cancer Treatment
Jeremy R. Graff, PhD, says new studies show promising data with the use of mRNA-based vaccines and the injection of nanoparticles into regional lymph nodes to achieve disease stabilization. This has led to a potentially groundbreaking era of therapeutic cancer vaccines thanks to discoveries in identifying truncal targets, targeting tumor neoantigens and, notably, developing improved delivery technologies that stimulate a robust, targeted, and persistent immune response.
NANOPARTICLE ENGINEERING - Lighting the Way to a Patient-Centric Future
Christopher Worrall, PhD, discusses how nanoparticle engineering technologies could help improve compliance and patient outcomes, for both small-molecule and biological drugs, and how nanotechnology can help facilitate a shift toward more patient-centric medicine.
Alexion, AstraZeneca Rare Disease to Acquire LogicBio Therapeutics to Accelerate Growth in Genomic Medicine
LogicBio Therapeutics, Inc. recently announced it has entered into a definitive agreement under which AstraZeneca Rare Disease will acquire LogicBio. The proposed acquisition…
Biogen & Denali Therapeutics Announce Initiation of Phase 3 LIGHTHOUSE Study in Parkinson’s Disease Associated With LRRK2 Pathogenic Mutations
Biogen Inc. and Denali Therapeutics Inc. recently announced dosing has commenced in the global Phase 3 LIGHTHOUSE study to evaluate the efficacy and safety profile…
Zealand Pharma Announces Positive Results from Phase 3 Trial of Glepaglutide in Patients With Short Bowel Syndrome (EASE 1)
Zealand Pharma A/S recently announced positive topline results from the pivotal Phase 3 trial of glepaglutide, a long-acting GLP-2 analogue designed for once or twice…
Cornerstone Pharmaceuticals Initiates Phase 1 Trial of CPI-613 in Combination With Chemoradiation for the Treatment of Advanced Pancreatic Cancer
Cornerstone Pharmaceuticals, Inc. recently announced the initiation of a Phase 1 clinical trial evaluating CPI-613 (devimistat) in combination with chemoradiation for the treatment of advanced…
Genelux Corporation Initiates Pivotal Phase 3 Trial Evaluating Olvi-Vec for the Treatment of Platinum-Resistant/Refractory Ovarian Cancer
Genelux Corporation recently announced it has initiated OnPrime, a multi-center, randomized, open-label Phase 3 registrational trial evaluating the efficacy and safety of Olvi-Vec in combination…
Immunic Announces Positive Results from Single & Multiple Ascending Dose Parts of its Phase 1 Clinical Trial
Immunic, Inc. recently announced positive unblinded safety, tolerability and pharmacokinetic (PK) results from Part A (single ascending doses, SAD) and Part B (multiple ascending doses, MAD)…
Decibel Therapeutics Announces Submission of IND Application for Lead Gene Therapy Candidate
Decibel Therapeutics recently announced the submission of an Investigational New Drug (IND) application to the US FDA for a Phase 1/2 clinical trial in pediatric…
RIGImmune Announces Acquisition of Antiviral Company Subintro
RIGImmune Inc. recently announced the acquisition of Subintro, a biotechnology company specializing in the development and delivery of antiviral therapeutics for respiratory diseases caused by…
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).