Bio Platforms
SiSaf Announces Positive Preclinical Data for its SiS-ADO2 siRNA Program to Treat Rare Genetic Bone Disorder Osteopetrosis
SiSaf Ltd, an RNA delivery and therapeutics company, recently announce positive data confirming safety and efficacy of its Bio-Courier next-generation silicon stabilized hybrid lipid nanoparticles (sshLNP) as an….
Aptose’s New G3 Formulation of Luxeptinib Boosts Bioavailability
Aptose Biosciences Inc. recently announced the G3 formulation of luxeptinib, designed for rapid and efficient absorption, demonstrates approximately an 18-fold improvement in oral bioavailability….
Revolo Biotherapeutics Completes Enrollment in Phase 2 Trial
Revolo Biotherapeutics recently announced it has completed enrollment and has fully randomized all patients in a Phase 2a proof of concept clinical study investigating its…
Chiesi Global Rare Diseases Announces FDA Acceptance of BLA Filing for the Proposed Treatment of Alpha-Mannosidosis
Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., recently announced the US FDA has accepted the Biologics License Application (BLA) and granted…
Good Therapeutics Announces Acquisition of Conditionally Active PD-1-Regulated IL-2 Program by Roche
Good Therapeutics recently announced it has entered into a definitive merger agreement to be acquired by Roche. With this acquisition, Roche will gain….
Avalo Advances BTLA Agonist Fusion Protein to IND-Enabling Stage
Avalo Therapeutics, Inc. recently announced its human B and T Lymphocyte Attenuator (BTLA) agonist fusion protein, AVTX-008, has now entered IND-enabling studies, with a target…
Tenaya Therapeutics Announces FDA Clearance of IND & Initiation of Phase 1 Safety Study for an HDAC6 Inhibitor for Heart Failure With Preserved Ejection Fraction
Tenaya Therapeutics, Inc. recently announced clearance of its Investigational New Drug (IND) application to begin clinical testing of TN-301 by the US FDA. TN-301 is…
Synlogic Achieves Research Milestone in Collaboration With Roche for Development of Novel Synthetic Biotic to Treat Inflammatory Bowel Disease
Synlogic, Inc. recently announced it has received a milestone payment for the achievement of prespecified success criteria under the research collaboration agreement with Roche for….
Novo Nordisk to Acquire Forma Therapeutics & Expand Presence in Sickle Cell Disease & Rare Blood Disorders
Novo Nordisk and Forma Therapeutics, Holdings Inc. recently announced they have entered into a definitive agreement under which Novo Nordisk will acquire Forma Therapeutics for….
Vaxart Announces Positive Top-line Phase 2 Clinical Study Data Demonstrating Safety & Immunogenicity of Its Wuhan S-Only COVID-19 Pill Vaccine Candidate
Vaxart, Inc. recently reported positive top-line data from the first part of a planned two-part Phase 2 study of its Wuhan S-only oral pill COVID-19…
GENE THERAPY - Developing Affordable Point of Care CAR-T Therapies: Expanding Efficacy & Impact
Rimas Orentas, PhD, and Boro Dropulić, PhD, MBA, believe the future belongs to those who will be able to innovate rapidly, maintain regulatory confidence, and drive down costs to make CAR-T cell and other engineered cell therapies available to all who would benefit.
LIVE BIOTHERAPEUTIC PRODUCTS - Not All Microbiome Approaches Are Created Equal
Duncan Peyton says in comparison with other therapeutic classes, such as antibodies or gene therapy, the progress that has been made with LBPs to date has been rapid, and for the field to maintain this rate of progress and to establish LBPs as a mainstay in the treatment of patients across a variety of diseases, a number of key questions need to be addressed.
Laverock Therapeutics Founded to Develop the Next Generation of Programmable Cell Therapies
Laverock Therapeutics Ltd was founded to develop a unique gene silencing platform for the creation of programmable, allogeneic cell therapies. Having recently completed a….
Denali Therapeutics Announces New Interim Data from Phase 1/2 Study of DNL310 in MPS II (Hunter Syndrome)
Denali Therapeutics Inc. recently announced new interim results from a Phase 1/2 trial of DNL310 (ETV:IDS) in MPS II (Hunter syndrome). DNL310 is an investigational,…
FDA Grants Fast Track Designation for Longeveron’s Treatment for Hypoplastic Left Heart Syndrome in Infants
Longeveron Inc. recently announced the US FDA has granted Fast Track Designation to Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare…
WHITEPAPER - Key Considerations in Oral Delivery of Peptides – Factors to Consider While Evaluating Oral Administration
Peptide therapeutics are a hot topic in pharmaceutical R&D. While most peptides are administered parenterally, oral delivery of peptide therapeutics offers several advantages. Enteris BioPharma’s…
Immutep Granted Japanese Patent for Eftilagimod Alpha, a Soluble Lag-3 Protein, in Combination With a PD-1 Pathway Inhibitor
Immutep Limited recently announced the grant of a new patent (No. 7116547) titled Combined Preparations for the Treatment of Cancer or Infection by the Japanese…
Timber Pharmaceuticals Announces Publication of Results from Phase 2b CONTROL Study of FDA-Designated Breakthrough Therapy
Timber Pharmaceuticals, Inc. recently announced that results from the previously completed Phase 2b CONTROL study that evaluated TMB-001, a topical isotretinoin formulated using the company’s patented IPEG delivery system, in moderate-to-severe congenital ichthyosis (CI) are published….
Anthos Therapeutics Launches Second Phase 3 Clinical Trial of its Dual-Acting Factor XI Inhibitor
Anthos Therapeutics recently announced the initiation of recruitment for its second Phase 3 clinical trial investigating abelacimab, its novel dual-acting fully human monoclonal antibody targeting…
Ranok Therapeutics Announces Initiation of Patient Dosing in a Phase 1/2 Clinical Trial of First-in-Class BRD4-Targeting CHAMP Protein Degrader
Ranok Therapeutics recently announced the initiation of patient dosing in the US for a Phase 1/2 study of RNK05047. The trial, entitled CHAMP-1, will evaluate the safety, tolerability, pharmacokinetics, and….
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).