Bio Platforms
Vaccinex Announces $1.5-Million Registered Direct Offering & Concurrent Private Placement Priced At-the-Market Under Nasdaq Rules
Vaccinex, Inc. recently announced it has entered into definitive agreements with certain institutional investors for the purchase and sale of 193,000 shares of its common stock in a….
LIXTE's Lead Clinical Compound Can Force Cancer Cells to Give Up Their Cancer-Causing Properties
LIXTE Biotechnology Holdings, Inc. recently announced publication of preclinical data in the online journal, Cancer Discovery, showing its lead clinical compound, LB-100, can force cancer…
Intravacc Announces Positive Data of First in Human Intranasal OMV-Based Vaccine for SARS-CoV-2
Intravacc recently announced positive clinical data of a first in human (FIH) study of Avacc 10, an intranasal outer membrane vesicles (OMV) based booster vaccine…
Teva UK & Closed Loop Medicine Announce Strategic Partnership to Advance Development of Personalized Medicines
Teva UK Limited and Closed Loop Medicine Ltd have announced a strategic partnership to advance the development of personalized medicine. Under the agreement, Closed Loop Medicine and Teva UK will investigate….
Nanoscope Therapeutics Announces Positive Top-line Results From Randomized Controlled Trial of MCO-010 for Retinitis Pigmentosa
Nanoscope Therapeutics Inc. recently announced positive top-line results after the completion of the 2-year Phase 2b RESTORE randomized, controlled clinical trial of its lead program, MCO-010,…
Exothera Pledges to Fast-Track RNA Therapies With Expansion of World-First Continuous-Batch RNA Manufacturing Solution Into North America
Game-changing continuous-batch production of RNA service removes process development and scale-up steps through automation and significantly cuts clinical development time, while enhancing quality and yield….
Opus Genetics Announces Completion of Dosing in First Cohort of Phase 1/2 Trial of Gene Therapy in Patients with Rare Inherited Retinal Disease LCA5
Opus Genetics recently announced the first cohort has completed dosing in its open-label, dose-escalation Phase 1/2 clinical trial evaluating the subretinal delivery of OPGx-LCA5, an…
Verge Genomics & Ferrer Announce Agreement to Co-Develop Clinical-Stage ALS Therapy
Verge Genomics and Ferrer recently announced a strategic collaboration to co-develop VRG50635, Verge’s lead drug candidate, for the treatment of sporadic and familial forms of amyotrophic lateral sclerosis….
Imbria Pharmaceuticals Presents Results From IMPROVE-HCM Trial of Ninerafaxstat
Imbria Pharmaceuticals, Inc. recently announced the results from the Phase 2 IMPROVE-HCM clinical trial, evaluating ninerafaxstat, a novel cardiac mitotrope and partial fatty acid oxidation…
Bluejay Announces Receipt of PRIME Designation From EMA for Chronic Hepatitis Delta Virus Infection Treatment
Bluejay Therapeutics, Inc. recently announced the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to BJT-778 for the treatment of Chronic Hepatitis Delta…
IGC Pharma Announces Positive Interim Results for IGC-AD1 in Reducing Alzheimer’s Agitation
IGC Pharma, Inc. recently announced the results of an interim analysis of its ongoing Phase 2 trial investigating IGC-AD1 as a treatment for Agitation in…
Nykode Therapeutics Announces Topline Conclusions Affirming Prolonged Clinical Benefits From Matured Survival Data in Phase 2 C-02 Trial in Advanced Cervical Cancer
Nykode Therapeutics ASA recently announced key conclusions from the updated analysis from the Phase 2 VB-C-02 trial. The trial investigates the use of Nykode’s wholly…
CAMP4 Therapeutics Announces Dosing of First Participant in Phase 1 Clinical Study for Potential First-in-Class Therapeutic for Urea Cycle Disorders
Lead program in urea cycle disorders highlights power of CAMP4’s RAP Platform to increase gene expression using programmable, targeted medicines, with clinical candidate moving from screen-to-clinic within 3 years….
Lisata Therapeutics Announces US FDA Rare Pediatric Disease Designation Granted to LSTA1 for the Treatment of Osteosarcoma
Lisata Therapeutics, Inc. recently announced the US FDA has granted Rare Pediatric Disease Designation (RPDD) to LSTA1, the company’s lead product candidate, for the treatment…
Orchard Therapeutics Outlines US Launch Plans for the Only Approved Therapy for Children With Early Onset Metachromatic Leukodystrophy
Orchard Therapeutics recently announced the details of its US commercial launch of Lenmeldy (atidarsagene autotemcel), formerly known as OTL-200, the first FDA-approved therapy for the….
Immix Biopharma Announces Major Comprehensive Cancer Center as Lead Site for NXC-201 AL Amyloidosis Clinical Trial
Immix Biopharma, Inc. recently announced Memorial Sloan Kettering Cancer Center as lead clinical site for its NXC-201 relapsed/refractory AL Amyloidosis multi-site clinical trial. “We are…
OKYO Pharma to Release New & Comprehensive Data GFrom Phase 2 Dry Eye Disease Trial
OKYO Pharma Limited recently announced it will be releasing new and comprehensive efficacy data readout from the Phase 2 trial of OK-101 in dry eye disease on….
ACELYRIN Announces Positive Phase 1/2 PoC Data for First Subcutaneous Anti-IGF-1R to Demonstrate Clinical Responses in Thyroid Eye Disease
ACELYRIN, INC. recently announced positive proof-of-concept data from an ongoing Phase 1/2 trial of lonigutamab in thyroid eye disease (TED). Lonigutamab is a subcutaneously….
RespireRx Pharmaceuticals Reports Preclinical Research Demonstrating the Ability of its Lead Clinical AMPAkine to Improve Bladder Function After Spinal Cord Injury
RespireRx Pharmaceuticals Inc. recently announce that, as part of an ongoing collaboration, a scientist team led by Dr. David Fuller of the University of Florida…
Silo Pharma Exercises Option for Exclusive License Agreement for First-in-Class PTSD & Stress-Induced Anxiety Therapeutic
Silo Pharma, Inc. recently announced it has exercised its option to license SPC-15, a prophylactic treatment for stress-induced affective disorders including anxiety and PTSD, from…
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).