Bio Platforms
Olema Oncology Announces New Clinical Trial Collaboration & Supply Agreement
Olema Pharmaceuticals, Inc. recently announced a new clinical trial collaboration and supply agreement with Novartis in frontline metastatic breast cancer. Olema has also entered into…
PTC Therapeutics Enters Global License & Collaboration Agreement With Novartis
PTC Therapeutics, Inc. recently announced the signing of an exclusive global license and collaboration agreement with Novartis Pharmaceuticals Corporation, a subsidiary of Novartis AG (NYSE:…
NovelMed Receives FDA IND Approval for the First Anti-Bb Alternative Pathway Blocker for Treating Primary Immunoglobulin A Nephropathy (IgAN): A Renal Disorder
NovelMed recently announced the US FDA has granted clearance for Ruxoprubart, an investigational drug, to commence an efficacy trial for the treatment of Immunoglobulin A…
Rigel Announces R289 Granted Fast Track Designation by the FDA for Lower-Risk MDS
Rigel Pharmaceuticals, Inc. recently announced the US FDA has granted Fast Track designation to R289 for the treatment of patients with previously-treated transfusion dependent lower-risk…
Sana Biotechnology Announces Fast Track Designation for SC291 in Relapsed/Refractory Systemic Lupus Erythematosus
Sana Biotechnology, Inc. recently announced the US FDA granted Fast Track designation for SC291 in relapsed/refractory systemic lupus erythematosus (SLE), which includes extrarenal lupus and…
Lin BioScience Receives FDA Fast Track Designation For LBS-007
Lin BioScience recently announced its lead pipeline, LBS-007, has been granted Fast Track Designation by the US FDA for the treatment of acute myeloid leukemia.…
Marinomed Biotech AG Signs Agreement for Sale of Carragelose Business to Unither Pharmaceuticals
Marinomed Biotech AG has signed an agreement on the sale of its Carragelose business to the French CDMO Unither Pharmaceuticals. The contract provides for upfront…
Anocca Announces Submission of Clinical Trial Application for Multi-Asset Trial VIDAR-1 in Advanced Pancreatic Cancer
Anocca AB recently announced the submission of its first Clinical Trial Application (CTA) to the European Medicines Agency (EMA) for a Phase 1/2 multi-asset umbrella…
Anavex Life Sciences Announces Submission of Blarcamesine MAA for Treatment of Alzheimer’s Disease to EMA
Anavex Life Sciences Corp. recently announced the submission of the blarcamesine (ANAVEX2-73) MAA (Marketing Authorization Application) to the European Medicines Agency (EMA). The MAA submission…
Kanglin Biotechnology Announces $20-Million Financing to Advance the Development of Gene Therapies
Kanglin Biotechnology (Hangzhou) Co., Ltd, an innovative biotech company developing the next generation of gene therapies, recently announced the successful completion of a Series A…
IMUNON Announces Results From its End-of-Phase 2 Meeting With the FDA for its Lead IMNN-001 Clinical Program in Advanced Ovarian Cancer
IMUNON, Inc. recently announced the outcome of its recent End-of-Phase 2 in-person meeting with the US FDA, supporting the advancement of its investigational interleukin-12 (IL-12)…
Tenaya Therapeutics Doses First Patient in Phase 1b Clinical Trial for the Treatment of PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy
Tenaya Therapeutics, Inc. recently announced the first patient has been dosed with TN-401 gene therapy in the RIDGE-1 Phase 1b clinical trial at the University…
OncoVerity Secures Extended Series A to Advance Cusatuzumab in Newly Diagnosed AML
OncoVerity recently announced the closing of a Series A extension financing led by existing investors, argenx and RefinedScience. With this funding, OncoVerity aims to advance…
UMDF & Khondrion Announce The Mito Fund’s Investment in Khondrion to Support Development of One of the Most Advanced Drug Candidates for Primary Mitochondrial Disease
The United Mitochondrial Disease Foundation’s (UMDF) venture philanthropy arm, The Mito Fund, has invested in Khondrion, a Netherlands-based clinical stage biopharmaceutical company discovering and developing therapies…
BriaCell Announces First Patient Dosed With Bria-OTS in Metastatic Breast Cancer Study
BriaCell Therapeutics Corp. recently announced the first patient was dosed in its Phase 1/2 study (ClinicalTrials.gov identifier: NCT06471673) to evaluate the safety and efficacy of…
Dyadic Receives $3 Million Grant to Develop Cost-Effective Monoclonal Antibodies for RSV & Malaria Using C1 Platform Technology
Dyadic International, Inc. recently announced it has been awarded a $3 million grant from the Gates Foundation for the cell line development of monoclonal antibodies…
GRI Bio Showcases GRI-0621’s Potential to Reduce Inflammation, Type 1 Cytokines & Reduce Hepatic Fibrosis 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…
Eradivir’s Therapeutic Proven to Reduce Advanced-Stage Influenza Viral Loads Faster, More Thoroughly in Preclinical Studies Than Current Therapies
Eradivir has developed a patent-pending antiviral therapeutic that reduces lung viral loads of advanced-stage influenza in preclinical studies quicker and more effectively than currently available…
Verge Genomics Announces Milestones in Collaboration With Lilly to Discover & Develop Novel Treatments for ALS
Verge Genomics recently announced Eli Lilly & Company has opted to pursue the development of therapeutics against two validated drug targets for amyotrophic lateral sclerosis…
Voyager Selects Tau Silencing Gene Therapy Development Candidate for Alzheimer’s Disease
Voyager Therapeutics, Inc. recently announced it has selected a lead development candidate, VY1706, for its tau silencing gene therapy program in Alzheimer’s disease. The company…
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).