Bio Platforms
Cue Biopharma Enters Strategic Collaboration & Option Agreement With Ono Pharmaceutical
Cue Biopharma, Inc. recently announced a collaboration and option agreement with Ono Pharmaceutical Co., Ltd. for CUE-401, a bispecific protein designed to induce and expand regulatory….
Eterna Therapeutics Enters Option & License Agreement With Lineage Cell Therapeutics to Develop Hypoimmune Pluripotent Cell Lines for Multiple Neurology Indications
Eterna Therapeutics Inc. recently announced it has entered into an exclusive option and license agreement with Lineage Cell Therapeutics, Inc. for the development of novel induced…
uniQure Announces the European Commission Approval of the First Gene Therapy for Adults With Hemophilia B
uniQure N.V. recently announced its partner, global biotechnology leader CSL, has received conditional marketing authorization (CMA) from the European Commission for HEMGENIX (etranacogene dezaparvovec), the first…
Processa Pharmaceuticals Announces Prioritization on Development of Next-Generation Chemotherapies
Processa Pharmaceuticals, Inc. reiterates the strategic prioritization of its pipeline of proprietary oncology drugs, defined as Next-Generation Chemotherapies (NGCs). Previous studies with these….
Icosavax Granted FDA Fast Track Designation for RSV & hMPV Vaccine Candidate
Icosavax, Inc. recently announced the US FDA has granted Fast Track designation for IVX-A12, a bivalent respiratory syncytial virus (RSV) and human metapneumovirus (hMPV) VLP…
Kadimastem Submits IND Application for its Phase 2a Clinical Trial With AstroRx for the Treatment of ALS
Kadimastem Ltd recently has submitted an Investigational New Drug (IND) application to the US FDA for an approval for a multi-site Phase 2a clinical trial to test its lead neurological….
Frontera Therapeutics Doses First Patient in a Trial of Gene Therapy for the Treatment of X-Linked Retinitis Pigmentosa
Frontera Therapeutics recently announced it has dosed the first patient in a clinical trial of its gene therapy product, FT-002. FT-002 is being studied for…
Cytovation Announces First Patient Dosed in Phase 2a Study Investigating CyPep-1 Monotherapy in Advanced Melanoma Refractory to Checkpoint Inhibitors
Cytovation ASA recently announced the first patient has been dosed in its Phase 2a study investigating CyPep-1 monotherapy in patients with advanced melanoma refractory to…
VYNE Therapeutics Announces Positive Results from Phase 1a Single & Multiple Ascending Dose Trial for Investigational Novel BET Inhibitor
VYNE Therapeutics Inc. recently announced positive results from the Phase 1a portion of its Phase 1a/b clinical trial evaluating its novel BET inhibitor, VYN201, for vitiligo…..
Akari Therapeutics Announces Preclinical Development Progress Toward Potential IND on Long-Acting PAS-Nomacopan for Geographic Atrophy
Akari Therapeutics, Plc recently announced updates on progress in the pre-clinical development program for long-acting PAS-nomacopan as a potential treatment for geographic atrophy (GA) secondary…
Nexcella Enters US GMP Manufacturing Agreement to Expand Ongoing Phase 1b/2 Clinical Trial
Nexcella, Inc. recently announced it has entered into a manufacturing agreement with a well-known United States Good Manufacturing Practice (GMP) cell therapy manufacturer that will…
BioXcel Therapeutics Announces Positive Full Data from Phase 2 Trial of BXCL701 in Rare, Aggressive Form of Prostate Cancer
BioXcel Therapeutics, Inc. recently announced full data from its Phase 2a trial of BXCL701, the company’s investigational, oral innate immune activator, in combination with….
Purple Biotech Announces First Patient Dosed in Open Label, Randomized Part of CM24 Phase 2 Clinical Trial
Purple Biotech Ltd. recently announced the first patient has been dosed in the randomized part of the open label Phase 2 clinical trial evaluating CM24…
Vaxart Doses First Subject in the Phase 2 Clinical Trial of its Bivalent Norovirus Candidate
Vaxart, Inc. recently announced it has dosed the first subject in the Phase 2 clinical trial of its oral tablet bivalent norovirus candidate. The dose-ranging…
Neurona Therapeutics Announces FDA Clearance of Expanded Inclusion Criteria for Phase 1/2 Clinical Trial of Regenerative Cell Therapy in Patients With Dominant Hemisphere Drug-Resistant Focal Epilepsy
Neurona Therapeutics recently announced the US FDA has cleared an amendment to Neurona’s ongoing Phase 1/2 clinical trial of NRTX-1001 in people with drug-resistant mesial…
Acumen Pharmaceuticals Completes Enrollment in Phase 1 Trial of First Monoclonal Antibody Developed to Selectively Target Toxic Aβ Oligomers in Patients With Early Alzheimer’s Disease
Acumen Pharmaceuticals, Inc. recently announced the completion of enrollment in its Phase 1 INTERCEPT-AD trial of ACU193 in patients with early Alzheimer’s disease…..
Sermonix Pharmaceuticals & Quantum Leap Healthcare Announce New Study Arm to Evaluate Lasofoxifene in Ongoing I-SPY 2 Clinical Trial
Sermonix Pharmaceuticals Inc. and Quantum Leap Healthcare Collaborative recently announced Sermonix’s investigational next-generation targeted endocrine therapy, lasofoxifene, will be evaluated in a new study arm…
Vaxxas Initiates Phase 1 Clinical Study of First Needle-Free, Inactivated Seasonal Influenza Vaccine Quadrivalent Delivered Using High-Density Microarray Patch
Vaxxas recently announced the initiation of a Phase 1 clinical trial with the first needle-free inactivated seasonal influenza vaccine quadrivalent (IIV4) candidate delivered using Vaxxas’…
Pantherna & Evaxion Announce Promising Preclinical mRNA Vaccine Data
Evaxion Biotech A/S and Pantherna Therapeutics GmbH recently announced preclinical proof of concept for the combination of the two companies’ key….
EuMentis Therapeutics Inc. Receives $3-Million Award from US DoD to Advance its Novel NMDA Receptor Antagonist for Traumatic Brain Injury
EuMentis Therapeutics Inc. recently announced it has received an award from the United States Department of Defense (DoD) to develop a novel small molecule therapeutic…
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).