Bio Platforms
Sonnet BioTherapeutics Announces Topline Safety Data Following Successful Completion of SON-1010 Monotherapy Dose Escalation in Phase 1 SB101 Trial
Sonnet BioTherapeutics Holdings, Inc. recently announced that the results of SON-1010 at the highest dose have been formally evaluated by the Safety Review Committee in…
Mestag Therapeutics Announces Licensing of Novel Inflammatory Disease Target From its RAFT Platform
Mestag Therapeutics recently announced that Johnson & Johnson has exclusively licensed a novel, undisclosed target identified using Mestag’s Reversing Activated Fibroblast Technology (RAFT) platform under…
CERo Therapeutics Appoints Chris Ehrlich CEO
CERo Therapeutics Holdings, Inc. recently announced its Board of Directors has appointed Chris Ehrlich as CEO. Previously he held the position of Interim CEO. Mr.…
World’s First Zero-Off-Target Base-Edited NK Cell Therapy Receives IND Approval in Both China & US
Base Therapeutics recently announced its NK510 cell injection, the world’s first zero-off-target base-edited NK cell product, has received IND approvals from both the US FDA…
Groundbreaking Cretostimogene Grenadenorepvec Monotherapy Data Demonstrates Sustained, Durable Complete Responses in High-Risk BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
CG Oncology, Inc. recently announced topline data from the Phase 3 BOND-003 trial in patients with high-risk Non-Muscle Invasive Bladder Cancer (NMIBC) unresponsive to Bacillus…
Denali Therapeutics Announces First Participant Dosed in Phase 2a Study of LRRK2 Inhibitor, BIIB122, in LRRK2-Associated Parkinson’s Disease
Denali Therapeutics Inc. recently announced initiation of dosing in a global Phase 2a clinical study, BEACON, of the investigational drug leucine-rich repeat kinase 2 (LRRK2)…
8 Years Survival With Complete Cure for a Patient With Advanced Liver Cancer Being Treated With Namodenoson Drug
Can-Fite BioPharma Ltd. recently announced a patient currently treated with Namodenoson in a compassionate use program in Can-Fite’s Phase 2 Liver Cancer Study has an…
Amarna Therapeutics Partners With NorthX Biologics to Advance Gene Therapy Development
Amarna Therapeutics recently announced its strategic collaboration with NorthX Biologics, a leading biologics manufacturing partner. This partnership represents a major step in advancing the clinical…
Medigene Receives Milestone Payment from Regeneron Pharmaceuticals
Medigene AG recently announced a $1 million milestone payment from Regeneron Pharmaceuticals, Inc. was triggered. Regeneron purchased the MAGE-A4-TCR program as part of its acquisition…
Roivant Announces Topline Results From Phase 2 RESOLVE-Lung Study of Namilumab in Chronic Active Pulmonary Sarcoidosis
Kinevant Sciences recently announced its Phase 2 study failed to show treatment benefit in patients with chronic active pulmonary sarcoidosis. The Phase 2 RESOLVE-Lung study…
Palisade Bio Announces Preliminary Data From Phase 1 Clinical Study for the Treatment of Moderate-to-Severe Ulcerative Colitis Appears to be Safe & Well Tolerated
Palisade Bio, Inc recently announced preliminary results from its first three single ascending dose (SAD) cohorts in its ongoing Phase 1 human clinical study for…
Kairos Pharma Adds City of Hope Cancer Center for Phase 2 Clinical Trial
Kairos Pharma Ltd. recently announced the addition of City of Hope Cancer Center in Duarte, California to the Phase 2 clinical trial for ENV105 for…
Recursion Announces First Patient Dosed in Phase 1/2 Clinical Study of a Potential First-in-Class RBM39 Degrader for Biomarker-Enriched Solid Tumors & Lymphoma
Recursion recently announced the first patient has been dosed in its Phase 1/2 clinical trial of REC-1245, a new chemical entity for the treatment of…
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…
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).