Bio Platforms
ALX Oncology Doses First Patient in Phase 1 Dose Escalation Trial Evaluating ADC ALX2004 for the Treatment of EGFR-Expressing Solid Tumors
ALX Oncology Holdings Inc. recently announced the first patient has been dosed in the company’s Phase 1 clinical trial for ALX2004, a potential best- and…
BiomX Provides Update on BX004 Phase 2b Trial for the Treatment of Patients with Cystic Fibrosis
BiomX Inc. recently announced the US FDA has placed a clinical hold on the Phase 2b trial of BX004 for the treatment of patients with…
Invivyd Announces Continued Neutralizing Activity of PEMGARDA & VYD2311 Against Currently Dominant SARS-CoV-2 Variant XFG
Invivyd, Inc. recently announced positive, continued, clinically meaningful in vitro neutralization data for PEMGARDA (pemivibart) against the currently dominant and growing XFG variant of SARS-CoV-2.…
Valneva’s Chikungunya Vaccine Now Authorized in Canada for Individuals Aged 12 & Older
Valneva SE recently announced Health Canada has granted marketing authorization for its single-dose vaccine, IXCHIQ, for the prevention of disease caused by the chikungunya virus…
Opus Genetics Announces FDA Clearance of IND Application for Gene Therapy Candidate
Opus Genetics recently announced the US FDA has accepted its Investigational New Drug (IND) application for OPGx-BEST1, a gene therapy for the treatment of bestrophin-1…
Halda Therapeutics Receives FDA Fast Track Designation for Metastatic Castration-Resistant Prostate Cancer Treatment
Halda Therapeutics recently announced the US FDA granted Fast Track designation to HLD-0915, the company’s lead development candidate, for the treatment of patients with metastatic…
Superluminal Medicines Announces Collaboration With Eli Lilly to Advance Small Molecule Therapeutics for Cardiometabolic Diseases & Obesity
Superluminal Medicines recently announced a collaboration with Eli Lilly and Company to advance small molecule therapeutics targeting undisclosed GPCR targets relevant to cardiometabolic diseases and…
BriaCell Selected for MSK’s 2025 Therapeutics Accelerator Program for Bria-OTS+ for Cancer
BriaCell Therapeutics Corp. recently announced its acceptance into Memorial Sloan Kettering Cancer Center’s (MSK’s) Therapeutics Accelerator 2025 Cohort program. This project aims to accelerate the…
MAIA Biotechnology Granted Patent for Next-Generation Telomere-Targeting Agents for Cancer Therapy
MAIA Biotechnology, Inc. recently announced the European Patent Office has decided to grant a patent broadly covering a portfolio of ateganosine-based analogues for telomere-targeting anticancer…
NodThera Demonstrates Reversal of Neuroinflammation & Inflammation in Parkinson’s Disease With Oral NLRP3 Inhibitor
NodThera recently announced the full publication of positive data from its Parkinson’s disease Phase 1b/2a study evaluating the effects of NT-0796, its oral, brain-penetrant NLRP3…
AAVantgarde Bio Announces FDA Fast Track Designation for AAVB-039 for the Treatment of Stargardt Disease
AAVantgarde Bio recently announced the US FDA has granted Fast Track Designation for AAVB-039, the company’s gene therapy program for Stargardt disease secondary to biallelic…
Ocular Therapeutix Receives FDA Agreement Under Special Protocol Assessment for Registrational Trial of AXPAXLI in NPDR
Ocular Therapeutix, Inc. recently announced it has received written agreement regarding a registrational trial design from the US FDA under a Special Protocol Assessment (SPA)…
BBOT Debuts as a Publicly Traded Company Focused on RAS-Pathway Malignances With a Potential to Realize the Full Promise of KRAS & PI3K Inhibition
BridgeBio Oncology Therapeutics, Inc. (BBOT) recently announced the closing of its previously announced business combination with Helix Acquisition Corp. II (formerly Nasdaq: HLXB) (Helix), a…
FoRx Therapeutics Initiates First-in-Human Trial With Novel Anti-Cancer Drug Targeting DNA Damage Response
FoRx Therapeutics recently announced the dosing of the first patient in a first-in-human clinical study of FORX-428, a novel PARG inhibitor designed to target and…
GT Biopharma Advances Into Cohort 3 of GTB-3650 Phase 1 Trial Following Safety Review of Cohort 2
GT Biopharma, Inc. recently announced initiation of dosing in Cohort 3 of its Phase 1 dose escalation trial evaluating GTB-3650 for the treatment of relapsed or…
Legend Biotech Reports Recent Highlights & Second Quarter Results
Legend Biotech Corporation recently reported its second quarter 2025 unaudited financial results and key corporate highlights. “The groundbreaking 5-year survival data from CARTITUDE-1, with one-third…
Nanexa Signs Continuation of Feasibility Agreement With Major Pharmaceutical Company to Investigate PharmaShell Long-Acting Formulations in a Multi-Billion US Market
Nanexa has recently announced the signing of an extension of a feasibility agreement with a major pharmaceutical company to investigate the use of Nanexa´s proprietary…
Assembly Biosciences Reports Positive Interim Results From Phase 1b Clinical Study of Long-Acting Helicase-Primase Inhibitor Candidate Showing Reductions in Viral Shedding Rate & Genital Lesion Rate in Recurrent Genital Herpes
Assembly Biosciences, Inc. recently announced positive interim antiviral activity, clinical outcomes, safety, and pharmacokinetic (PK) results from a Phase 1b study evaluating ABI-5366, an investigational…
Genmab Announces Phase 3 Trial Met Dual Primary Endpoints in Patients With Relapsed/Refractory Follicular Lymphoma
Genmab A/S recently announced positive results of the Phase 3 EPCORE FL-1 trial evaluating subcutaneous epcoritamab, a bispecific antibody, in combination with rituximab andlenalidomide (R2) versus…
Cardurion Pharmaceuticals Completes Enrollment in Phase 2 Trials Evaluating a Novel PDE9 Inhibitor in Heart Failure
Cardurion Pharmaceuticals, Inc. recently announced it has completed enrollment in two global, multi-center, Phase 2 clinical trials assessing the safety and efficacy of CRD-750 in…
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).