Bio Platforms
Dupixent Approved in the US as the First-Ever Biologic Medicine for Patients With COPD
The US FDA has approved Dupixent (dupilumab) as an add-on maintenance treatment of adults with inadequately controlled chronic obstructive pulmonary disease (COPD) and an eosinophilic…
Evaxion Significantly Expands Vaccine Development Collaboration With MSD
Evaxion Biotech A/S recently announced it has entered into an option and license agreement with MSD (tradename of Merck & Co., Inc.) for two preclinical…
Acumen Pharmaceuticals Extends Collaboration With Lonza to Add Drug Product Manufacturing of Sabirnetug for Early Alzheimer’s Disease
Acumen Pharmaceuticals, Inc. recently announced it has extended its collaboration with Lonza to enable the potential future commercial launch of sabirnetug (ACU193). Sabirnetug is the…
GRI Bio Receives Authorization to Conduct Phase 2a Biomarker Study Evaluating GRI-0621
GRI Bio, Inc. recently announced the authorization of its Clinical Trial Application (CTA) by the Australian Medicines and Healthcare products Regulatory Agency (MHRA) and the…
ImmunoPrecise Antibodies Advances Therapeutic Innovation With Groundbreaking Rabbit Antibody Developments
ImmunoPrecise Antibodies Ltd. (IPA) recently announced the clinical progress achieved with rabbit monoclonal antibodies designed and developed using IPA’s proprietary B Cell Select platform for…
Palisade Bio Provides Update on Progress Toward Launch of Phase 1 Human Clinical Study for Lead Program for the Treatment of Ulcerative Colitis
Palisade Bio, Inc. recently provided an update on its progress toward the launch of its Phase 1 human clinical study for PALI-2108 for the treatment of…
GoLiver Therapeutics Awarded Innovation in Biotherapies & Bioproduction Call for its GOCLINICALS Project Dedicated to Novel Stem Cell-Based Liver Therapy
GoLiver Therapeutics recently announced it has won the Innovations in Biotherapies and Bioproduction call for proposal launched by the French government to make France a…
Lonza & Vertex Sign a Long-Term Commercial Supply Agreement for CASGEVY
Lonza, a global manufacturing partner to the pharmaceutical, biotech, and nutraceutical markets, recently announced the signature of a long-term commercial supply agreement for CASGEVY (exagamglogene…
Quell Therapeutics Expands Manufacturing Capabilities for CAR-Treg Cell Therapy Pipeline Through Partnership With eXmoor pharma
Quell Therapeutics Ltd and eXmoor pharma recently announced a strategic partnership for the manufacture of multiple autologous engineered CAR-Treg cell therapies in Quell’s pipeline targeting autoimmune…
KSQ Therapeutics Announces FDA Clearance of IND Application for a CRISPR/Cas9 Engineered Tumor-Infiltrating Lymphocyte Therapy
KSQ Therapeutics, Inc., in collaboration with The University of Texas MD Anderson Cancer Center (MD Anderson) and the Cell Therapy Manufacturing Center (CTMC), recently announced…
Araris Biotech AG Expands IP Portfolio With Acquisition of Innate Pharma’s Portfolio
Araris Biotech AG recently announced it has entered into an agreement with Innate Pharma. Under the agreement, Innate will assign its portfolio of patents related…
Barinthus Bio Initiates Phase 1 Clinical Trial of for the Treatment of Celiac Disease
Barinthus Biotherapeutics plc recently announced the initiation of its first-in-human Phase 1 trial of VTP-1000 in adults with celiac disease. The randomized, placebo-controlled clinical trial,…
Assembly Biosciences Reports Positive Interim Phase 1a Results From Clinical Trial Evaluating Long-Acting Helicase-Primase Inhibitor Candidate
Assembly Biosciences, Inc. recently announced positive interim pharmacokinetic (PK) and safety results from healthy participants in the Phase 1a portion of its ongoing Phase 1a/b…
Ventyx Biosciences Announces $27-Million Strategic Investment From Sanofi
Ventyx Biosciences, Inc. recently announced Sanofi has agreed to make a $27-million strategic investment in the company at an as-converted price of $3.8243 per share…
Hovione & iBET Announce the Establishment of a Venture in Cell & Gene Therapy Solutions
Hovione and iBET recently announced the official establishment of ViSync Technologies as a jointly owned venture. The creation of this new company, headquartered in the…
Tiziana Life Sciences Announces $4-Million Grant to Study Anti-CD3 in Alzheimer’s Disease
Tiziana Life Sciences, Ltd. recently announced the National Institutes of Health (NIH), National Institute on Aging have awarded a $4-million grant to Dr. Howard Weiner…
Oncolytics Biotech Reports Favorable Results for BRACELET-1 Breast Cancer Study
Oncolytics Biotech Inc. recently announced positive clinical results from BRACELET-1, its randomized Phase 2 study evaluating pelareorep in patients with HR+/HER2- advanced or metastatic breast cancer.…
Sonnet BioTherapeutics Completes Enrollment in Phase 1 Study of SON-1010 (IL12-FHAB) as a Monotherapy (SB101) for the Treatment of Solid Tumors
Sonnet BioTherapeutics Holdings, Inc. recently announced the completion of enrollment and initiation of dosing in its Phase 1 SB101 clinical trial of SON-1010 (IL12-FHAB) in…
TransCode Therapeutics Announces First Patients Treated in Phase 1 Clinical Trial With First-in-Class Lead Therapeutic Candidate
TransCode Therapeutics, Inc. recently announced dosing of the first two patients in its Phase 1 clinical trial with its lead candidate, TTX-MC138. Several additional patients…
Cullinan Therapeutics Receives Approval to Initiate its Global Phase 1 Clinical Trial for the Treatment of Systemic Lupus Erythematosus
Cullinan Therapeutics, Inc. received Human Research Ethics Committee (HREC) approval in Australia to initiate its global Phase 1 clinical trial to evaluate CLN-978, its CD19xCD3 bispecific…
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).