Bio Platforms
Ocugen Announces FDA Clearance of IND Amendment to Initiate Phase 3 Clinical Trial - First Gene Therapy to Enter Phase 3 With a Broad Retinitis Pigmentosa Indication
Ocugen, Inc. recently announced the US FDA has cleared the company’s Investigational New Drug (IND) amendment to initiate a Phase 3 clinical trial of OCU400,…
PolTREG Identifies Promising Efficacy Biomarker for Type-1 Diabetes in Patients Treated With its Treg Therapy in Combination With Rituximab
PolTREG S.A. recemtly announced it has published data in International Immunopharmacology, which suggest that PD-1+ T-cells are a dependable biomarker for efficacy in pediatric early-onset…
Medigene AG Secures European Patent for its iM-TCR Technology
Medigene AG recently announced it has been issued a patent by the European Patent Office protecting its inducible Medigene T cell receptor (iM-TCR) technology, which…
Biora Therapeutics Achieves Positive Interim Results for Clinical Trial of BT-600, Advancing NaviCap Platform Development
Biora Therapeutics, Inc. recently shared additional positive interim results from the single-ascending dose (SAD) clinical trial of BT-600, which is a drug-device combination consisting of…
Oruka Therapeutics & ARCA biopharma Announce Merger Agreement
Merger to create a company focused on advancing Oruka’s portfolio of novel biologics that aim to redefine the standard of care for patients with chronic skin diseases….
Lipella Pharmaceuticals Announces FDA Type C Meeting for LP-10 for Hemorrhagic Cystitis
Lipella Pharmaceuticals Inc. recently announced the US FDA has granted a Type C meeting request to discuss the company’s proposed Phase-2b clinical trial design for…
BioCorRx Pharmaceuticals Awarded New Patent for Novel Compound to Treat Pain, Depression & Schizophrenia
BioCorRx Inc. recently announced its subsidiary BioCorRx Pharmaceuticals Inc., a clinical-stage biopharmaceutical company, has been granted a new patent by the United States Patent and…
OrganaBio Launches On-Demand GMP-Compliant Hematopoietic Stem Cell Source for Advanced Therapy Manufacturing
OrganaBio recently announced the launch of HematoPAC-HSC-CB-GMP, on-demand current good manufacturing practice (cGMP)-compliant CD34+ hematopoietic stem cells (HSCs) ethically derived from fresh human cord blood.…
Ipsen & Sutro Biopharma Announce Exclusive Global Licensing Agreement for an ADC Targeting Solid Tumors
Ipsen and Sutro Biopharma recently announced an exclusive global licensing agreement for STRO-003, an antibody-drug conjugate in the final stages of pre-clinical development, which targets the….
Evaxion & Undisclosed Collaborator Announce Encouraging Results for Vaccine Antigens Against Staphylococcus Aureus Infection
Evaxion Biotech A/S recently announced the successful conclusion of a series of large non-rodent animal infection studies, testing antigens from Evaxion’s preclinical EVX-B1 vaccine candidate…
Corcept Completes Enrollment in Phase 3 Gradient Trial of Relacorilant in Patients With Adrenal Cushing’s Syndrome
Corcept Therapeutics Incorporated recently announced completion of enrollment in GRADIENT, a Phase 3 trial of its proprietary selective cortisol modulator relacorilant in patients with Cushing’s…
THERAPEUTIC FOCUS - Perspectives on Sigma-2 Modulation as a Therapeutic Modality for Slowing Age-Related Degenerative Disease
Anthony Caggiano, MD, PhD, believes there is evidence the sigma-2 receptor acts as a regulator of cellular processes, such as autophagy and protein trafficking, which are impaired in age-related degenerative diseases.
VACCINE DEVELOPMENT - Therapeutic Vaccines Development: At the Edge of a New Revolution
Mario Davinelli, PhD, Narcisa Mesaros, MD, David Morland, and Judith Neville, PhD, say therapeutic vaccines are an exciting area of research that has the potential to revolutionize the way we treat diseases.
PERSONALIZED MEDICINE - Exploring the Potential of the Aryl Hydrocarbon Receptor for Personalized Medicine
Pedro Moura Alves, PhD, reviews the intricacies of AhR immune sensing and signaling, and its potential for personalized treatment strategies to optimize therapeutic outcomes and minimize adverse effects. He also touches on some of the limitations hindering the advancement of knowledge in this crucial area of research, and the direction this emerging field may take in the coming years.
THERAPEUTIC FOCUS - Sarilumab Approval for Polymyalgia Rheumatica Highlights Enduring Unmet Medical Needs
David A. Katz, PhD, and Robert Jacks, MBA, MSE, say a new, effective treatment option is now available, promotion of the drug will increase disease awareness, and the success encourages others in the industry to continue and expand their PMR research. However, the proportion of patients who can benefit from the drug is limited, and significant unmet needs remain for all persons suffering from PMR.
Intravacc & Primrose Bio Announce Partnership to Enhance Conjugate Vaccine Development
The companies will leverage their combined experience in the development of conjugate vaccines for their clients….
Mustang Bio Announces Vision for CAR T-Cell Therapy Platform Expansion Into Autoimmune Diseases
Mustang Bio, Inc. recently announced its expansion into autoimmune diseases with MB-106, a personalized CD20-targeted, 3rd-generation autologous CAR T-cell therapy. MB-106 is being developed in…
Vidac Pharma Receives Japanese Patent Office Notice of Allowance for Cancer Drug Candidate
Vidac Pharma Holdings Plc. recently announced it has received a Notice of Allowance from the Japanese Patent Office for the composition and methods of use…
Immuneering Announces First Patient Dosed in its Phase 1/2a Trial of IMM-6-415 to Treat Advanced Solid Tumors With RAF or RAS Mutations
Immuneering Corporation recently announced the first patient has been dosed in its Phase 1/2a trial of IMM-6-415 to treat advanced solid tumors with RAF or…
Vector Pharma & Oncopeptides FZCO Announce Collaboration to Provide Pepaxti to Patients in the Middle East & North Africa
Oncopeptides AB and Vector Pharma FZCO recently announced a collaboration to commercialize Oncopeptides’ flagship drug Pepaxti (melphalan flufenamide) in the Middle….
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).