Bio Platforms
First Patient Dosed with LIXTE’s LB-100 in New Clinical Trial to Treat Colorectal Cancer
LIXTE Biotechnology Holdings, Inc. recently announced the dosing of the first patient in a new clinical trial in collaboration with the Netherlands Cancer Institute (NKI)…
Galapagos Announces FDA Clearance of IND Application for Phase 1/2 ATALANTA-1 Study of CD19 CAR-T, GLPG5101, in Relapsed/Refractory Non-Hodgkin Lymphoma
Galapagos NV recently announced the US FDA has cleared its Investigational New Drug (IND) application for ATALANTA-1, a Phase 1/2 multicenter study evaluating the feasibility,…
Abata Therapeutics Receives FDA Fast Track Designation for Progressive Multiple Sclerosis Treatment
Abata Therapeutics recently announced the US FDA has granted Fast Track designation for ABA-101 for the treatment of patients with progressive multiple sclerosis (MS). The…
Emergex Receives Patent Protection for First-in-Class Influenza A Vaccines With Potential to Provide Long-Term T Cell Immunity
Emergex Vaccines Holding Limited recently announced it has received patent protection from the United States Patent and Trademark Office (USPTO) for its novel class of…
ST Pharm & Quantoom Biosciences Announce First Supply Agreement of SmartCap Under Extended Collaboration to Advance RNA Manufacturing
ST Pharm and Quantoom Biosciences recently announced an extended collaboration to accelerate the development and manufacturing of RNA-based vaccines and therapeutics. This collaboration brings together…
Opus Genetics Receives Rare Pediatric Disease Designation for Ocular Gene Therapy
Opus Genetics recently announced the US FDA has granted Rare Pediatric Disease designation (RPD) for its ocular gene therapy OPGx-LCA5 to treat patients with the…
ImmuneSensor Therapeutics Receives Clearance to Initiate First-in-Human Phase 1 Clinical Trial of cGAS inhibitor Drug Candidate
ImmuneSensor Therapeutics recently announced it has received Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) clearance by the Australian Therapeutic Goods Administration…
Processa Pharmaceuticals Announces Positive Preclinical Data for NGC-Iri
Processa Pharmaceuticals, Inc. recently announced positive data from preclinical studies that support the potential for NGC-Iri to have improved efficacy and a better side effect…
Abata Therapeutics Announces Strategic Investment From Bristol Myers Squibb
Abata Therapeutics recently announced it has received an equity investment from Bristol Myers Squibb to support the development of its Treg cell therapy products. Their…
TransCode Therapeutics Announces Phase 1 Clinical Trial Initiation
TransCode Therapeutics, Inc. recently announced initiation of its Phase 1 multicenter, open-label, clinical study of its lead therapeutic candidate, TTX-MC138. Two clinical trial sites have…
Denali Therapeutics Announces Publication Demonstrating the Potential of the Oligonucleotide Transport Vehicle Platform to Achieve Broad Biodistribution of Antisense Oligonucleotides in the CNS & Muscle Following Intravenous Administration
Denali Therapeutics Inc. recently announced publication of nonclinical data in the August 14, 2024, issue of Science Translational Medicine (link) demonstrating the ability of the…
Nuntius Therapeutics Announces Collaboration With Taiho Pharmaceutical to Develop Next-Generation mRNA Therapies Using Nuntius’ Proprietary Cell-Specific Delivery Technology
Nuntius Therapeutics recently announced it has entered into a collaboration agreement with Taiho Pharmaceutical Co., Ltd., a leading company in Japan for developing innovative medicines…
Prota Therapeutics Announces Publication of Long-Term Peanut Allergy Study Confirming Clinical Remission as the Optimal Treatment Outcome
Prota Therapeutics Pty Ltd recently announced publication in the journal Allergy of the first long-term study directly comparing remission and desensitization endpoints following food allergen…
Nykode Therapeutics Announces Issuance of Key Patent Covering Individualized Neoantigen Based Vaccines
Nykode Therapeutics ASA recently announced the United States Patent and Trademark Office (USPTO) has issued US Patent No. 12,059,459 titled Therapeutic Anticancer Neoepitope Vaccine. The…
Immutep Announces First Participant Dosed in Phase 1 Study of First in Class Agonist LAG-3 Antibody
Immutep Limited recently announced the first participant has been successfully dosed in the first-in-human Phase 1 trial of IMP761. This first-in-class agonist LAG-3 antibody is…
Apollomics Announces Positive Preliminary Data of Vebreltinib in Patients With Non-CNS MET Fusion Solid Tumors From its Phase 2 Trial
Apollomics Inc. recently announced positive preliminary clinical data for the cohort of patients with non-CNS MET fusion solid tumors from the Phase 2 SPARTA trial…
Purdue & Akston Biosciences Sign Partnership Agreement for Dog Cancer Drug Development
Akston Biosciences Corporation and Purdue University recently announced a strategic partnership to co-develop an anti-cPD-L1 monoclonal antibody (mAb) immunotherapy to treat cancer in dogs. The underlying…
Eclipse Life Sciences Initiates Phase 2 Study for Novel Ophthalmology Steroid Implant for Diabetic Macular Edema
Eclipse Life Sciences, Inc. recently announced it has enrolled and dosed its first patient in the BETTIS-1 Phase 2 clinical trial (NCT06536491) for EC-104 fluocinolone…
MBX Biosciences Doses First Patient in Phase 2 Avail Trial for the Treatment of Hypoparathyroidism
MBX Biosciences, Inc. recently it has dosed the first patient in its Phase 2 Avail trial of MBX 2109, the company’s parathyroid hormone (PTH) peptide…
IN8bio Solidifies Position as a Clinical Leader of Gamma-Delta T Cell Therapy in Oncology With 100% of Treated AML Patients in Complete Remission& Receives FDA Guidance for Registrational Trial of INB-100
IN8bio, Inc. recently announced updated positive clinical data from both of the company’s Phase 1 investigator-sponsored trials of INB-100 for hematological malignancies and INB-200 for…
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).