Bio Platforms
Cardiff Oncology Announces First Patient Dosed in Phase 2 Trial of Onvansertib in Patients With Metastatic Colorectal Cancer
Cardiff Oncology, Inc. recently announced the first patient was dosed this month with its investigational drug onvansertib in its Phase 2 ONSEMBLE trial (NCT05593328). The…
Caribou Biosciences Initiates Dose Expansion Portion of Phase 1 Trial in Second-Line LBCL Patients
Caribou Biosciences, Inc. recently announced initiation of the dose expansion portion of the CB-010 ANTLER Phase 1 trial in second-line patients with large B cell…
Tiziana Life Sciences to Proceed With Phase 2 Clinical Trial in Patients With Non-Active Secondary Progressive Multiple Sclerosis
Tiziana Life Sciences Ltd. recently announced it has received feedback based on the US FDA Type C meeting minutes related to the Phase 2 clinical trial of intranasal foralumab in patients with non-active SPMS…..
Covant & Boehringer Ingelheim Collaborate to Develop Novel ADAR1 Inhibitor for Use in Cancer Patients
Covant Therapeutics has recently entered into an exclusive research collaboration and worldwide licensing agreement with Boehringer Ingelheim covering Covant’s ADAR1 program. The companies jointly aim….
Acumen Pharmaceuticals Presents In Vitro Human Neuron Model for Evaluating Binding of Amyloid Beta Oligomers in Alzheimer’s Disease
Acumen Pharmaceuticals, Inc. has recently demonstrated the utility of a human in vitro model of iPSC-derived excitatory neurons for a better understanding of which forms…
Vaccitech Announces Positive Topline Final Data for HBV002 Study in People With Chronic Hepatitis B
Vaccitech plc recently announced positive topline final data from the HBV002 study (NCT04778904), a Phase 1b/2a clinical trial of VTP-300 in people with chronic Hepatitis…
Cyclerion Therapeutics Receives Orphan Drug Designation for the Treatment of Mitochondrial Diseases
Cyclerion Therapeutics, Inc. recently announced the US FDA has granted orphan drug designation to zagociguat (previously CY6463) for the treatment of mitochondrial diseases. Zagociguat is the first….
Cullinan Oncology Announces FDA Clearance of INDA for Novel Fusion Protein Harnessing IL-2 & IL-12 Cytokines
Cullinan Oncology, Inc. recently announced the US FDA has cleared its Investigational New Drug (IND) application for CLN-617, a fusion protein composed of two potent…
Jounce Therapeutics to be Acquired by Concentra Biosciences
Jounce Therapeutics, Inc. recently announced it has entered into a definitive merger agreement whereby Concentra Biosciences, LLC (Concentra) will acquire Jounce for $1.85 in cash per share plus….
Kala Pharmaceuticals Announces Positive Safety Update From Cohort 1 of Phase 2b Clinical Trial
Kala Pharmaceuticals, Inc. recently announced positive safety data from the first cohort of the CHASE (Corneal Healing After SEcretome therapy) Phase 2b clinical trial evaluating…
Macomics & Ono Pharmaceutical Partner to Discover & Develop Macrophage-Targeting Antibody Therapy for Cancer Treatment
Macomics Ltd recently announced it has entered into a worldwide drug discovery collaboration agreement with Ono Pharmaceutical Co., Ltd. to develop new immuno-oncology antibody drugs…
Arch Biopartners Receives Funding for Development of LSALT Peptide Drug Program
Arch Biopartners Inc. recently announced it is receiving advisory services and up to $4 million in funding from the National Research Council of Canada Industrial Research Assistance Program (NRC IRAP) to support the….
Vaxxinity Announces First Subjects Dosed in Phase 1 Clinical Trial of Investigational Anti-PCSK9 Vaccine to Treat Hypercholesterolemia
Vaxxinity, Inc. recently announced the first subjects have been dosed in a randomized, double-blind, placebo-controlled Phase 1 clinical trial of VXX-401, an investigational vaccine designed to lower low-density lipoprotein….
Allarity Therapeutics Doses First Patient in Phase 1b Clinical Trial Evaluating Dovitinib & Stenoparib Combination in Advanced Solid Tumors
Allarity Therapeutics, Inc. recently announced it has dosed the first patient in a Phase 1b clinical study evaluating the combination of stenoparib and dovitinib for…
Synlogic Announces Data Presentations at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting
Synlogic, Inc. recently announced positive data from the company’s Phase 2 Synpheny-1 study for phenylketonuria (PKU) were presented in a podium presentation yesterday during the…
Enlivex Receives Positive DSMB Recommendation & IMOH Clearance to Continue Phase 1/2 Trial
Enlivex Therapeutics Ltd. recently announced an independent Data and Safety Monitoring Board (DSMB) has completed an interim data review for the first cohort of patients…
Owlstone Medical Enters Partnership With Bicycle Therapeutics for the Development of Antigen-Targeted EVOC Probes for Early Cancer Detection
Owlstone Medical, the global leader in Breath Biopsy for applications in early disease detection and precision medicine, recently announced it has entered into a Research Agreement with Bicycle Therapeutics plc, a biotechnology company pioneering a new….
Pfizer Invests $43 Billion in Seagen Acquisition to Battle Cancer
Pfizer Inc. and Seagen Inc. recently announced they have entered into a definitive merger agreement under which Pfizer will acquire Seagen, a global biotechnology company that discovers, develops, and commercializes transformative cancer medicines, for $229 in cash….
Immutep Announces Initiation of Phase 2/3 Trial for Eftilagimod Alpha Plus Paclitaxel in Metastatic Breast Cancer
Immutep Limited recently announced the initiation of AIPAC-003 (Active Immunotherapy, Eftilagimod Alpha, and PAClitaxel), an integrated Phase 2/3 trial to evaluate eftilagimod alpha (efti) in combination…
Proteovant Therapeutics to Present Preclinical Data From Estrogen Receptor & IKZF2 Protein Degrader Programs
Proteovant Therapeutics, a Roivant Sciences (Nasdaq: ROIV) company leveraging the process of protein degradation to discover and develop transformative medicines for the treatment of patients…
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).