Bio Platforms
PAN-VARIANT INHIBITORS - How Pan-Variant Inhibition Can Outsmart Cancer Treatment Resistance
Tim Clackson, PhD, says the goal in targeted oncology is to achieve pan-variant inhibition and, ideally, pan-variant inhibition should be accomplished with a single agent, avoiding the potential complications and toxicities of drug combinations.
ImmunoGen Announces a Global, Multi-Target License & Option Agreement With Vertex for the Use of ImmunoGen's ADC Technology in the Development of Novel Targeted Conditioning Agents
ImmunoGen, Inc. recently announced a global, multi-target license and option agreement whereby it granted Vertex Pharmaceuticals rights to conduct research using ImmunoGen's ADC technology to…
Incyclix Bio Announces FDA Clearance of IND Application for INX-315 in Patients With Advanced or Metastatic Cancer
Incyclix Bio, LLC recently announced the US FDA has cleared an investigational new drug (IND) application for the company’s lead compound, INX-315, a novel, potent…
EyePoint & Rallybio Announce Research Collaboration
Rallybio Corporation and EyePoint Pharmaceuticals, Inc recently announced a research collaboration. The partnership will evaluate sustained delivery of Rallybio’s inhibitor of complement component….
PDS Biotech Completes Successful Meeting With FDA for Triple Combination of PDS0101, PDS0301 & a Commercial Immune Checkpoint Inhibitor
Received guidance on registrational path for combination in recurrent/metastatic, immune checkpoint inhibitor refractory head and neck cancer….
IMUNON & Break Through Cancer Commence Enrollment in a Phase 1/2 Clinical Study of IMNN-001 in Combination With Avastin in Advanced Ovarian Cancer
IMUNON, Inc. and Break Through Cancer recently announced the commencement of patient enrollment in a collaboration to evaluate IMUNON’s IMNN-001 (formerly GEN-1) in combination with…
Nexcella Announces Patients Already Treated With CAR-T NXC-201; Estimates 100-Patient Total Enrollment for FDA Approval BLA Submission
Nexcella, Inc. recently announced 50 relapsed/refractory multiple myeloma patients have already been dosed with next-generation CAR-T NXC-201 in its ongoing Phase 1b/2a clinical trial. Nexcella…
Rhythm Pharmaceuticals Announces Acquisition of Xinvento B.V. & Portfolio of Investigational Therapeutics
Rhythm Pharmaceuticals, Inc. recently announced Rhythm’s Netherlands subsidiary, Rhythm Pharmaceuticals Netherlands B.V., acquired Xinvento B.V., a Netherlands-based biotech company focused on developing therapies for congenital…
Redx & Jounce Announce Recommended Business Combination
Redx Pharma and Jounce Therapeutics, Inc. recently announce an unanimously recommended Business Combination of the two companies via a proposed all share merger transaction. Redx…
VBL Therapeutics & Notable Labs Announce Definitive Merger Agreement
VBL Therapeutics and Notable Labs, Inc. recently announced they have entered into a definitive merger agreement. The combined company will focus on the advancement of…
BriaCell Reports Positive Survival Data in Advanced Metastatic Breast Cancer
BriaCell Therapeutics Corp. recently reported updated survival data from its previously disclosed group of 12 patients (11 patients enrolled in 2021/2022) in the ongoing Phase…
Ocean Biomedical Celebrates Discovery of Bispecific Antibodies & Immune Checkpoint Inhibitors That Kill Glioblastoma Cells & Melanoma Cells & Block the Metastasis of Malignant Melanoma Cells to the Lung by Over 90%
Ocean Biomedical, Inc. celebrates the discovery of bispecific antibodies that target Chitinase 3-like-1 and immune checkpoint inhibitors, killing glioblastoma cells and melanoma cells, and blocking the metastasis of malignant….
Cue Biopharma Enters Strategic Collaboration & Option Agreement With Ono Pharmaceutical
Cue Biopharma, Inc. recently announced a collaboration and option agreement with Ono Pharmaceutical Co., Ltd. for CUE-401, a bispecific protein designed to induce and expand regulatory….
Eterna Therapeutics Enters Option & License Agreement With Lineage Cell Therapeutics to Develop Hypoimmune Pluripotent Cell Lines for Multiple Neurology Indications
Eterna Therapeutics Inc. recently announced it has entered into an exclusive option and license agreement with Lineage Cell Therapeutics, Inc. for the development of novel induced…
uniQure Announces the European Commission Approval of the First Gene Therapy for Adults With Hemophilia B
uniQure N.V. recently announced its partner, global biotechnology leader CSL, has received conditional marketing authorization (CMA) from the European Commission for HEMGENIX (etranacogene dezaparvovec), the first…
Processa Pharmaceuticals Announces Prioritization on Development of Next-Generation Chemotherapies
Processa Pharmaceuticals, Inc. reiterates the strategic prioritization of its pipeline of proprietary oncology drugs, defined as Next-Generation Chemotherapies (NGCs). Previous studies with these….
Icosavax Granted FDA Fast Track Designation for RSV & hMPV Vaccine Candidate
Icosavax, Inc. recently announced the US FDA has granted Fast Track designation for IVX-A12, a bivalent respiratory syncytial virus (RSV) and human metapneumovirus (hMPV) VLP…
Kadimastem Submits IND Application for its Phase 2a Clinical Trial With AstroRx for the Treatment of ALS
Kadimastem Ltd recently has submitted an Investigational New Drug (IND) application to the US FDA for an approval for a multi-site Phase 2a clinical trial to test its lead neurological….
Frontera Therapeutics Doses First Patient in a Trial of Gene Therapy for the Treatment of X-Linked Retinitis Pigmentosa
Frontera Therapeutics recently announced it has dosed the first patient in a clinical trial of its gene therapy product, FT-002. FT-002 is being studied for…
Cytovation Announces First Patient Dosed in Phase 2a Study Investigating CyPep-1 Monotherapy in Advanced Melanoma Refractory to Checkpoint Inhibitors
Cytovation ASA recently announced the first patient has been dosed in its Phase 2a study investigating CyPep-1 monotherapy in patients with advanced melanoma refractory to…
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).