Bio Platforms
ReciBioPharm & GeneVentiv Therapeutics Partner to Advance First AAV-Based Gene Therapy for Haemophilia Patients With Inhibitors
ReciBioPharm has recently announced a collaboration agreement with GeneVentiv Therapeutics to advance development of an Adeno-Associated Virus (AAV)-based universal gene therapy for….
Anavex Life Sciences Initiates Placebo-Controlled US Phase 2 Clinical Trial of ANAVEX®3-71 in Schizophrenia
Anavex Life Sciences Corp. recently announced the first patient in its US FDA cleared placebo-controlled Phase 2 clinical study of ANAVEX3-71 for the treatment of…
Purple Biotech Reports Preclinical Proof of Concept for its Tribody Platform Technology
Cleavable capping technology confines therapeutic activity to the local tumor micro environment which increases the anticipated therapeutic window in patients….
Lirum Therapeutics Announces Positive Data on LX-101, a Novel Clinical-Stage Targeted Therapy Demonstrating Potent Preclinical Activity Against IGF-Related Cancers
Lirum Therapeutics, Inc. recently announced the acceptance for presentation of positive data on LX-101, a novel clinical-stage targeted therapy directed to the insulin-like growth factor…
GRI Bio Announces Notice of Allowance for Canadian Patent Covering Proprietary Natural Killer T Cell Modulators
GRI Bio, Inc. recently announced the Canadian Intellectual Property Office (CIPO) has issued a notice of allowance for Patent Application No. 3,007,970 titled, Prevention and…
Almirall & Eloxx Pharmaceuticals Enter Exclusive Agreement to License ZKN-013 for Rare Dermatological Diseases
Almirall, S.A. and Eloxx Pharmaceuticals, Inc. recently announced the companies have entered into an exclusive license agreement for the asset ZKN-013. Under the agreement, Almirall…
Terns Pharmaceuticals Announces Orphan Drug Designation Granted to TERN-701 for the Treatment of Chronic Myeloid Leukemia
Terns Pharmaceuticals, Inc. recently announced the US FDA granted Orphan Drug Designation for TERN-701 for the treatment of chronic myeloid leukemia (CML). TERN-701, the Company’s…
Cellares Unveils First cGMP-Compliant Cell Shuttle in its South San Francisco Center of Excellence
Cellares recently announced the completion of its first current Good Manufacturing Practice (cGMP)-compliant Cell Shuttle. The Cell Shuttle is an automated, ultra-high throughput, cell therapy…
Ajinomoto Bio-Pharma Services Garners Six CDMO Leadership Awards, Further Establishing its Industry Excellence
Ajinomoto Bio-Pharma Services recently received CDMO Leadership Awards in all six categories - Capabilities, Compatibility, Expertise, Quality, Reliability, and Service for both the Overall and…
Ubiquigent Enters Agreement With Astellas Subsidiary, Nanna Therapeutics
Agreement leverages Ubiquigent’s DUB-focused drug discovery platform to support the development of novel therapeutic candidates for multiple disease targets selected by Nanna Therapeutics….
Monte Rosa Therapeutics Announces Initiation of IND Enabling Studies for First-in-Class NEK7 Directed Molecular Glue Degrader & NLRP3/IL-1β Pathway Inhibitor
Monte Rosa Therapeutics, Inc. recently announced a novel development candidate, MRT-8102, a potent, highly selective and orally bioavailable NIMA related kinase 7 (NEK7)-directed MGD. MRT-8102…
Immuneering Doses First Patient in Phase 2a Clinical Trial of IMM-1-104 in RAS-mutant Solid Tumors
Immuneering Corporation recently announced the first patient has been dosed in the Phase 2a portion of its Phase 1/2a clinical trial of IMM-1-104, its lead…
Acelyrin Announces Positive Top-line Results From Phase 2b/3 Clinical Trial of Izokibep in Psoriatic Arthritis
ACELYRIN, INC. recently announced its global Phase 2b/3 clinical trial of izokibep in psoriatic arthritis (PsA) met the primary endpoint of ACR50 at week 16…
Athira Pharma Presents Clinical & Preclinical Data Supporting Therapeutic Potential of Fosgonimeton in Alzheimer’s & Parkinson’s Diseases
Athira Pharma, Inc. presented new clinical and preclinical data further highlighting the therapeutic potential of fosgonimeton at the AD/PD 2024 International Conference on Alzheimer’s and…
NodThera’s NLRP3 Inhibitor Reverses Neuroinflammation in Parkinson’s Disease Phase 1b/2a Trial
NodThera recently announced positive data from its Phase 1b/2a study in Parkinson’s disease patients, evaluating the effects of its oral, brain-penetrant NLRP3 inflammasome inhibitor NT-0796,…
Genezen Announces Strategic Process Development & Manufacturing Partnership Agreement With Seattle Children’s Research Institute
Seattle Children’s Research Institute and Genezen have unveiled a strategic manufacturing partnership for Seattle Children’s Research Institute’s X-linked agammaglobulinemia….
Olema Oncology Announces Data Highlighting Palazestrant’s Ability to Inhibit Wild-Type & Mutant ER+ Breast Cancer Both as Monotherapy & in Combination With CDK4/6 Inhibitors
Olema Pharmaceuticals, Inc. recently announced that Molecular Cancer Therapeutics, an American Association for Cancer Research journal, has selected as a featured article a data publication that…
BiomX Enters Merger Agreement With Adaptive Phage Therapeutics & Concurrent $50-Million Financing
Acquisition will create phage therapy company with an advanced pipeline with two Phase 2 assets, BX004 for the treatment of chronic pulmonary infections in cystic fibrosis patients and BX211 for the treatment of diabetic foot osteomyelitis….
Exclusive License Agreement Secured With Erganeo for EVerGel, EVerZom's Breakthrough Treatment for Fistulas & Fibrosis of the Digestive Tract
EVerZom, a CNRS/Université Paris Cité spin-off specializing in exosomes, has announced the signature of a second exclusive license agreement with Erganeo for the development of…
Allyx Therapeutics Announces Expansion Into Parkinson’s Disease Clinical Research With Lead Compound Following IND Acceptance
Allyx Therapeutics recently announced the US FDA has accepted its Investigational New Drug Application (IND), enabling the company to proceed with clinical research in Parkinson’s…
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).