Bio Platforms
PDS Biotech Announces Submission of Phase 3 Protocol to FDA to Initiate VERSATILE-003 Trial
PDS Biotechnology Corporation recently announced the submission to the US FDA of an updated Chemistry, Manufacturing and Controls (CMC) package and a Phase 3 multicenter…
Absci & Caltech Join Forces to Accelerate Affordable HIV Therapeutic Vaccine Development
Absci Corporation recently announced leading researchers at the California Institute of Technology (Caltech) in conjunction with Absci, a leader in AI drug creation, received a…
GeoVax Announces First Patients Vaccinated in Phase 2 Clinical Trial of COVID-19 Vaccine Booster in Patients With Chronic Lymphocytic Leukemia
GeoVax Labs, Inc. recently announced vaccinations have begun in an investigator-initiated clinical trial (ClinicalTrials.gov Identifier: NCT05672355) of GEO-CM04S1 in patients with chronic lymphocytic leukemia (CLL),…
Jnana Therapeutics Announces Dosing of First Participant in Phase 1b Clinical Trial in Individuals With PKU
Jnana Therapeutics recently announced the first participant has been dosed in its Phase 1b clinical trial of JNT-517 in individuals with phenylketonuria (PKU). JNT-517 is…
CalciMedica Announces First Patient Enrolled in International Expansion of CARPO Trial of Auxora in Acute Pancreatitis
CalciMedica Inc. recently announced its ongoing CARPO trial, a Phase 2b trial evaluating Auxora for the treatment of acute pancreatitis (AP) with accompanying systemic inflammatory…
Cardiff Oncology Announces New Lead Program in First-Line Metastatic Colorectal Cancer & Expanded Pfizer Relationship
Cardiff Oncology, Inc. recently announced plans to advance the company’s lead program to the first-line setting of metastatic colorectal cancer (mCRC) and conduct its new CRDF-004 trial with study execution support from Pfizer….
Lisata Therapeutics Announces US FDA Orphan Drug Designation Granted for the Treatment of Malignant Glioma
Lisata Therapeutics, Inc. recently announced the US FDA has granted Orphan Drug Designation to LSTA1, the company’s lead product candidate, for the treatment of malignant…
Comera Life Sciences Significantly Expands Intellectual Property Portfolio Strengthening the Proprietary SQore Platform
Comera Life Sciences Holdings, Inc. recently announced a significant expansion of its SQore patent portfolio with the issuance of four new patents and two new…
MBX Biosciences Announces US FDA Clearance of IND Application for the Treatment of Post-Bariatric Hypoglycemia
MBX Biosciences, Inc. recently announced the US FDA has cleared the Investigational New Drug (IND) application for MBX 1416, the company’s long-acting glucagon-like peptide 1…
Preclinical Data Shows Strong Immunogenicity & Protection With IMUNON’s PlaCCine DNA-Based Vaccines Modality
IMUNON, Inc. recently announced a manuscript titled Strong immunogenicity & protection in mice with PlaCCine: A COVID-19 DNA vaccine formulated with a functional polymer is available…
ProQR Therapeutics & Laboratoires Théa Announce Agreement for Théa to Acquire ProQR’s Sepofarsen & Ultevursen Ophthalmic Assets
ProQR Therapeutics N.V. and Laboratoires Théa recently announced an agreement in which ProQR will divest its late-stage ophthalmic assets, sepofarsen and….
Ocean Biomedical, Inc. Celebrates 95% Lung Cancer Tumor Reduction Results on World Lung Cancer Day
On World Lung Cancer Day, Ocean Biomedical, Inc. is sharing details of its multi-pronged Lung Cancer Program built on novel immunotherapy discoveries by Scientific Co-founder Dr. Jack A. Elias. He and his team have revealed that Chitinase 3-like-1 (CHi3L1) is a master regulator that inhibits….
Avenue Therapeutics Announces High Potency & Full Efficacy in a Model of Generalized Seizures for its Novel GABA-A Receptor Potentiator Supporting Phase 2 Clinical Study
Avenue Therapeutics, Inc. recently announced BAER-101, the company’s potentially best-in-class selective GABA-A α2,3 positive allosteric modulator (PAM), significantly suppressed seizures in a translational animal model…
AltruBio Announces Positive Topline Data from Phase 1 Trial of Immune Checkpoint Enhancer for the Treatment of Ulcerative Colitis
AltruBio Inc. recently announced positive topline data from its Phase 1 trial evaluating the company’s subcutaneously administered immune checkpoint enhancer (ICE), ALTB-268 in healthy volunteers. “We…
WHITEPAPER - Use of a Platform Formulation Technology to De-Risk Solid-State Variation in Drug Development
This white paper describes use of mesoporous silica as a porous carrier formulation technology to stabilize unstable polymorphs and to optimize solid state properties.
BioNTech Completes Acquisition of InstaDeep
BioNTech SE recently announced the completion of the acquisition of InstaDeep Ltd., a leading global technology company in the field of artificial intelligence (AI) and…
PharmAla Files Patent for Novel MDXX Molecule PharmAla-1
PharmAla Biotech Holdings Inc. recently announced it has filed a patent on the composition of matter of PharmAla-1. The patent filing follows a year of preclinical…
WHITEPAPER - Long-Acting Injectable Nanoparticle Formulations
Long acting injectable (LAI) formulations have been the subject of continued interest in the recent past due, in part, to their longer systemic circulation requiring less frequent dosing of drugs.
NodThera is First to Demonstrate Reduction in Neuroinflammation in the Clinic With Brain Penetrant NLRP3 Inflammasome Inhibitor
NodThera recently announced positive, initial data from four subjects in the elderly volunteer stage of its Phase 1b/2a study evaluating the effects of its lead…
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).