Bio Platforms
TriSalus Life Sciences Presents Late-Breaking Phase 1 Liver Metastasis Data; Supports Development of Innovative Immuno-oncology Approach for Liver & Pancreas Indications
TriSalus Life Sciences Inc. recently presented additional Phase 1 clinical data during the late-breaker oral presentation session at the Society of Immunotherapy for Cancer (SITC) 2023…
ProQR Strengthens Leading Intellectual Property Estate for ADAR-Mediated RNA Editing
ProQR Therapeutics N.V. recently announced it has further strengthened its leading intellectual property (IP) estate with the issuance of a new patent in the US…
Acumen Pharmaceuticals & Halozyme Enter Global Collaboration & License Agreement to Develop Subcutaneous Formulation of ACU193 With ENHANZE Technology
Acumen Pharmaceuticals, Inc. recently announced a global non-exclusive collaboration and license agreement with Halozyme Therapeutics Inc. to provide Acumen access to Halozyme’s ENHANZE drug delivery technology, based on….
Sitryx Announces Partner Eli Lilly & Company Exercises Option to SIT-011 for Chronic Autoimmune & Inflammatory Diseases
Sitryx Therapeutics recently announced Eli Lilly & Company has exercised its option to progress SIT-011. Lilly will now lead further development and commercialization of the Phase 1 ready….
Tract Bio Announces Publication Highlighting Stem Cell Variants Driving Lung Inflammation in Modulator-Treated Cystic Fibrosis Patients
Tract Bio recently announced the publication of a research article in the American Journal of Respiratory and Critical Care Medicine (AJRCCM) highlighting the stem cell variants…
XOMA Earns $5-Million Milestone Upon FDA Acceptance of Day One’s Tovorafenib NDA for Relapsed or Progressive Pediatric Low-Grade Glioma
XOMA Corporation recently announced it has earned a $5-million milestone related to the US FDA's acceptance of Day One Biopharmaceuticals’ New Drug Application (NDA) for…
Apollomics Announces Two New Cohorts in Global Phase 2 SPARTA Study of Vebreltinib in Non-Small Cell Lung Cancer & Other Solid Tumors With MET Dysregulation
Apollomics Inc. recently announced the addition of two new cohorts in its ongoing global multi-cohort Phase 2 SPARTA study (NCT03175224), which is evaluating vebreltinib (APL-101) in…
4DMT Gains Alignment With FDA on Plan to Lift Clinical Hold on Phase 1/2 INGLAXA Clinical Trial for 4D-310 for Fabry Disease Cardiomyopathy
4D Molecular Therapeutics recently announced alignment with the US FDA on a plan to lift the clinical hold on the Phase 1/2 INGLAXA clinical trial…
Karyopharm Announces Clinical Trial Collaboration With Bristol Myers Squibb
Karyopharm Therapeutics Inc. recently announced it has entered into a clinical trial collaboration and supply agreement with Bristol-Myers Squibb to evaluate the company’s proprietary investigational….
Flashpoint Therapeutics Announces $10M Seed Financing to Pioneer Nanotechnology-Enabled Medicines
Flashpoint Therapeutics recently announced $10M seed financing round led by Beta Lab, a leading deep tech venture capital firm headquartered in Riyadh, Saudi Arabia, with…
Tenaya Therapeutics Announces FDA Clearance to Begin Clinical Testing of Gene Therapy for the Treatment of PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy
Tenaya Therapeutics, Inc. recently announced the US FDA has provided clearance of the company’s Investigational New Drug (IND) application to initiate clinical testing of TN-401.…
Pfizer & BioNTech Announce Positive Topline Data for mRNA-Based Combination Vaccine Program Against Influenza & COVID-19
Pfizer Inc. and BioNTech SE recently announced positive topline results from a Phase 1/2 study (NCT05596734) evaluating the safety, tolerability, and immunogenicity of mRNA-based combination vaccine candidates…
Orchard Therapeutics Announces Additional Positive Data from Proof-of-Concept Study of OTL-203 in MPS-IH
Orchard Therapeutics recently announced a range of interim clinical outcomes, in addition to the previously reported neurological and skeletal results, from the company’s ongoing proof-of-concept…
Andelyn Biosciences & Purespring Therapeutics Partner to Manufacture Novel Gene Therapies for Kidney Diseases
Andelyn Biosciences, Inc. has recently partnered with Purespring Therapeutics to accelerate the manufacture of their gene therapies for the treatment of chronic renal diseases that…
Aldena Therapeutics Selects PCI Pharma Services to Manufacture & Distribute Innovative Investigational Treatment for Dermatological Diseases
Aldena Therapeutics has chosen PCI Pharma Services (PCI) to formulate, package, and distribute ALD-102, an siRNA-based investigational injectable treatment for….
Ovid Therapeutics & Ligand Pharmaceuticals Enter $30-Million Agreement
Ovid Therapeutics Inc. and Ligand Pharmaceuticals Incorporated recently announced Ligand has acquired a 13% portion of the royalties and milestones owed to Ovid related to the potential approval and commercialization of….
IMUNON Announces First Patient Enrolled in Phase 1/2 Clinical Trial of IMNN-001 in Combination With Bevacizumab in Advanced Ovarian Cancer
IMUNON, Inc. recently announced the first patient in a Phase 1/2 clinical trial evaluating IMNN-001 in combination with bevacizumab in advanced ovarian cancer has begun…
Lisata Therapeutics Announces EMA Has Granted LSTA1 Orphan Drug Designation for the Treatment of Pancreatic Cancer
Lisata Therapeutics, Inc. recently announced the European Medicines Agency (EMA) Committee for Orphan Medicinal Products has granted orphan drug designation to LSTA1, the company’s lead…
Monte Rosa Therapeutics Announces Strategic Collaboration With Roche
Collaboration combines Monte Rosa Therapeutics’ highly differentiated QuEEN discovery engine with Roche’s strong expertise in delivering transformative therapies to patients….
Lumen Bioscience Awarded $16.2 Million in DoD Funding to Advance LMN-201 Through Late-Stage Trials
Lumen Bioscience recently announced it was awarded $16.2 million by the US Department of Defense to advance LMN-201 through late-stage trials. LMN-201 is an investigational,…
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).