Bio Platforms
Secarna Pharmaceuticals & Orbit Discovery Enter Collaboration to Discover & Develop Peptide-Conjugated Targeted Antisense Oligonucleotide Therapeutics
New platform capabilities extend the reach of Secarna’s antisense approaches, potentially offering new treatment options for a wider range of diseases….
Asahi Kasei to Acquire Calliditas Therapeutics AB to Accelerate Growth as a Global Healthcare Company
Asahi Kasei Corp. recently announced it will offer the shareholders of Calliditas Therapeutics AB to acquire the shares of the pharmaceutical company Calliditas for the purpose of….
selectION Announces Initiation of Phase 1b Clinical Trial Evaluating si-544 in Patients With Psoriasis Vulgaris or Psoriatic Arthritis
selectION, Inc. recently announced the initiation of a Phase 1b trial with its lead compound si-544 in adult patients with psoriasis vulgaris (Ps) or psoriatic…
Agios Announces $905-Million Purchase Agreement for Vorasidenib Royalty
Agios Pharmaceuticals, Inc. recently announced it has agreed to sell its rights to its 15% royalty on potential US net sales of Servier’s vorasidenib to….
GlycoNex & PrecisemAb Sign Technology Licensing Agreement to Advance Development of Novel Anti-Glycan Antibodies for Cancer Therapy
GlycoNex to advance development of novel pro-antibody cancer drugs that enhance tumor suppression while minimizing off-site binding to healthy tissues to improve safety and tolerability….
CARGO Therapeutics Announces $110-Million Private Placement Equity Financing
CARGO Therapeutics, Inc. recently announced it has entered into a securities purchase agreement for a private investment in public equity financing that is expected to…
Corcept Announces Primary Endpoint Met in Pivotal Phase 3 GRACE Trial of Relacorilant in Patients With Hypercortisolism
Corcept Therapeutics Incorporated recently announced that GRACE, the Phase 3 trial of its proprietary selective cortisol modulator relacorilant in patients with hypercortisolism (Cushing’s syndrome), met…
BriaCell Announces Clinical Supply Agreement With BeiGene for Bria-OTS First in Human Study
BriaCell Therapeutics Corp. recently announced a clinical supply agreement with BeiGene, Ltd. to evaluate the safety and efficacy of Bria-OTS, BriaCell’s next generation immunotherapy, in…
Coya Therapeutics Announces the Completion of a Controlled Phase 2 Study of Low Dose Interleukin-2 in Patients With Alzheimer’s Disease
Coya Therapeutics, Inc. recently announced the completion of a randomized, double-blind, placebo-controlled Phase 2 study of LD IL-2 in patients with mild-to-moderate AD. The study…
Amphista Therapeutics Unveils New Differentiated Mechanism of Action for BRD9 Degradation
Amphista’s bifunctional BRD9 degraders selectively induce BRD9 to DCAF16 proximity and serve as molecular glues, leading to strong and rapid degradation of BRD9….
Pneumagen Presents Positive Phase 2 Influenza Human Challenge Study Data
Pneumagen recently presented positive results from its Phase 2, proof-of-concept, Controlled Human Infection Model (CHIM) study in which healthy volunteers were challenged with influenza virus…
Cartesian Therapeutics Receives FDA Regenerative Medicine Advanced Therapy Designation for Descartes-08 for the Treatment of Myasthenia Gravis
Cartesian Therapeutics, Inc. recently announced the US FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation for Descartes-08 for the treatment of myasthenia gravis (MG).…
Indaptus Therapeutics Announces Completion of First Patient to Receive Multiple Doses of Broad-Based, “Pulse-Prime” Immuno-Oncology Therapy for Advanced Solid Tumors
Indaptus Therapeutics, Inc. recently announced the successful advancement of its Phase 1 trial for Decoy20, an investigational novel package of broad immune agonists for cancer…
Alvotech & Dr. Reddy’s Enter Commercialization Collaboration
Alvotech and Dr. Reddy’s Laboratories SA recently announced the companies have entered into a license and supply agreement for the commercialization of AVT03, Alvotech’s biosimilar candidate to….
Lipella Pharmaceuticals to Advance Lead Product Candidate to Phase 2b
Lipella Pharmaceuticals Inc. recently announced the receipt of US FDA Type-C meeting guidance regarding the LP-10 (liposomal tacrolimus) clinical program. Lipella expects to initiate a…
Limula Raises $6.8M to Democratise Access to Life-Saving Cell & Gene Therapies With Swiss-Made Manufacturing Platform
Over the past decade, several breakthrough Cell and Gene Therapy products have been approved in the US and Europe to turn stem cells and immune…
Molecure Receives Approval to Initiate Phase 2 Clinical Trial for the Treatment of Pulmonary Sarcoidosis
Molecure S.A. recently announced it has received national regulatory approvals from Denmark, France, Greece, Germany, and Norway to conduct a Phase 2 clinical trial for…
Sana Biotechnology Highlights Novel In Vivo Preclinical Data Showing Potential for Pluripotent Stem Cell-Derived Human Glial Progenitor Cell Transplantation as a Treatment for Huntington’s Disease and Other Glial-Based Neurodegenerative Conditions
Healthy transplanted human glial cells replaced diseased glial cells in the brains of human glial chimeric Huntingtin mice….
TFF Pharmaceuticals & Cleveland Clinic to Advance Multivalent Universal Influenza Vaccine Candidates into Preclinical Testing
TFF Pharmaceuticals, Inc recently announced that, in collaboration with Cleveland Clinic, the company is advancing multiple multivalent universal influenza vaccines to protect against seasonal and…
Lisata Therapeutics Receives Paediatric Investigation Plan Waiver From EMA for Certepetide in Pancreatic Cancer
Lisata Therapeutics, Inc. recently announced it has reached agreement with the European Medicines Agency (EMA) on a Paediatric Investigation Plan (PIP) for Lisata’s lead investigational…
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).