Bio Platforms
PolTREG Launches Phase 2 Cell Therapy Trial in Children With Presymptomatic Diabetes
PolTREG S.A. has launched a placebo-controlled Phase 2 clinical trial with PTG-007 Treg cell therapy in presymptomatic type-1 diabetes (T1D) patients, after receiving approval from…
Rhythm Pharmaceuticals & Axovia Therapeutics Announce Joint Research Collaboration in Bardet-Biedl Syndrome
Rhythm Pharmaceuticals, Inc. and Axovia Therapeutics Ltd. recently announced a joint research collaboration designed to advance the understanding of Bardet-Biedl syndrome (BBS). “We believe there…
GRI Bio Raises $13.9 Million in Gross Proceeds Since the Beginning of 2024
GRI Bio, Inc. recently announced it closed its previously announced transaction involving the exercise of certain existing warrants to purchase an aggregate of 762,236 shares…
Avacta Expands its Pipeline with Two Novel Assets Developed Using its pre|CISION Platform
Avacta Group plc recently announced the addition of two novel preclinical oncology assets, AVA6103 and AVA7100, to its pipeline of pre|CISION-enabled drug conjugates. Avacta is…
Immutep Announces First-in-Human Phase 1 Study of IMP761 Progresses to Dose Escalation Portion of Trial
Immutep Limited recently announced the first part (Part A, single dose) of the placebo-controlled, double-blind first-in-human Phase I study evaluating IMP761 has been fully recruited…
BioAegis Therapeutics Enrolls First Patient in Phase 2 Clinical Trial of Immune Regulator for Treatment of Acute Respiratory Distress Syndrome
BioAegis Therapeutics recently announced the first patient has been enrolled in the company’s Phase 2 study of rhu-pGSN for the treatment of Acute Respiratory Distress…
Biognosys Presents Major Innovations Across the Spectrum of Proteomics Research Applications
Biognosys recently announced its participation in the 23rd Human Proteome Organization (HUPO) World Congress from October 20 to October 24 in Dresden, Germany. The company…
mRNA Licensing Agreements Surge 800% in Value as Confidence Grows Beyond Vaccines
Messenger ribonucleic acid (mRNA)-based innovator pharmaceuticals saw a staggering 800% increase in licensing agreement deal values from 2019 to 2024YTD, driven by the remarkable success…
Cullinan Therapeutics Receives IND Clearance for CLN-978 Administered Subcutaneously in Patients With Moderate-to-Severe Systemic Lupus Erythematosus
Cullinan Therapeutics, Inc. recently announced the US FDA cleared the Company’s Investigational New Drug (IND) Application for CLN-978 and its global Phase 1 clinical trial…
OKYO Pharma Announces Start of Phase 2 Clinical Trial to Treat Patients With Neuropathic Corneal Pain
OKYO Pharma Limited recently announced that screening and recruitment of patients has started for a Phase 2 trial of OK-101 to treat NCP. The Phase…
IgGenix Announces First Patient Dosed in Phase 1 Clinical Trial Evaluating IGNX001 in Peanut Allergy
IgGenix, Inc. recently announced the first patient dosed in its ACCELERATE Peanut Phase 1 clinical trial. The trial aims to evaluate IGNX001, a novel therapeutic…
Wave Life Sciences Announces First-Ever Therapeutic RNA Editing in Humans Achieved in RestorAATion-2 Trial of WVE-006 in Alpha-1 Antitrypsin Deficiency
Wave Life Sciences Ltd. recently announced positive proof-of-mechanism data from the ongoing Phase 1b/2a RestorAATion-2 study of WVE-006 in alpha-1 antitrypsin deficiency (AATD). WVE-006 is…
GRI Bio Presents Positive Preclinical Data Demonstrating GRI-0621’s Ability to Inhibit Invariant Natural Killer T Cell Activity & Reduce Important Inflammatory & Fibrotic Drivers in Idiopathic Pulmonary Fibrosis
GRI Bio, Inc. recently announced the presentation of positive preclinical data demonstrating its lead program GRI-0621 reduces important inflammatory and fibrotic drivers in Idiopathic Pulmonary…
Lipella Pharmaceuticals Announces Patent Allowance for Innovative Liposomal Drug Delivery Platform
Lipella Pharmaceuticals Inc. recently announced the receipt of a notice of allowance from the US Patent and Trademark Office (USPTO) for its proprietary liposomal drug…
Impact BioMedical Inc. Announces Issuance of Patent for Linebacker Technology Targeting Inflammatory Disease
Impact BioMedical Inc. recently announced a significant milestone in its innovative Linebacker technology platform. The Canadian Intellectual Property Office has issued Canadian Patent #3,024,728 titled…
Molecure & Ocean Biomedical Sign Exclusive Licensing Agreement for Development & Commercialization of Selective YKL-40 Inhibitors
Molecure S.A. recently signed an exclusive licensing agreement with Ocean Biomedical Inc. concerning the development and commercialization of the selective YKL-40 inhibitor program, including the…
Immuneering Granted Orphan Drug Designation for Pancreatic Cancer Treatment
Immuneering Corporation recently announced the US FDA has granted orphan drug designation to IMM-1-104 in the treatment of pancreatic cancer. IMM-1-104 is currently being evaluated…
Indaptus Therapeutics Initiates Unrestricted Enrollment of Patients on Decoy20 Weekly Dosing Based on Encouraging Safety Data
Indaptus Therapeutics, Inc. recently provided an update regarding key clinical advancements in its Phase 1 trial of lead drug candidate Decoy20. The Safety Review Committee…
Akari Therapeutics Announces Effectiveness of Form S-4 & General Meeting Date of November 7, 2024 Related to Peak Bio Merger
Akari Therapeutics, Plc recently announced the effectiveness of the Form S-4, originally filed with the SEC on September 13, 2024, related to the merger of…
FDA Clears Annovis to Launch Pivotal Phase 3 Alzheimer’s Studies, Paving the Way to NDAs
Annovis Bio Inc. recently announced the successful outcome of the End-of-Phase 2 meeting with the US FDA on October 10, 2024. During the meeting, the…
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).