Bio Platforms
OKYO Pharma Achieves 90% Enrollment in 240-Patient Phase 2 Clinical Trial of OK-101 to treat Dry Eye Disease
OKYO Pharma Limited recently announced it has enrolled and randomized 90% of the patients in its 240-patient Phase 2 multi-center, double-masked, placebo-controlled clinical trial of…
Denali Therapeutics Announces New Interim Data From Phase 1/2 Study of DNL310 in MPS II (Hunter Syndrome)
Denali Therapeutics Inc. recently announced new interim data from the ongoing open-label, single-arm Phase 1/2 study of DNL310 (ETV:IDS) in children with MPS II (Hunter…
Biostax Signs Collaboration Agreement With Immgenuity to Pursue Remission in HIV
Immune Therapeutics, Inc. d/b/a Biostax Corp. recently announced the signing of a research collaboration agreement with Immgenuity, Inc. The joint research will focus on using…
Eagle Pharmaceuticals Announces Positive Type C Meeting With FDA for an Estrogen Receptor Antagonist Used in the Treatment of Metastatic Breast Cancer in Post-Menopausal Women
Eagle Pharmaceuticals, Inc. recently announced a positive Type C meeting with the US FDA. Eagle and the FDA agreed on a path forward to advance…
Ocean Biomedical Announces New Patent for Anti-Fibrosis Discovery With Allowance in Alcoholic Liver Disease & Multiple Fibrotic Conditions
Ocean Biomedical, Inc. recently announced the United States Patent and Trademark Office has issued a patent covering Ocean’s anti-Chitinase 1 small molecule candidate. Dr. Jack…
Cellares Announces Bristol Myers Squibb has Joined Technology Adoption Partnership Program to Evaluate Automated Manufacturing of CAR-T Cell Therapy on the Cell Shuttle Platform
Cellares, the first Integrated Development and Manufacturing Organization (IDMO) dedicated to clinical and industrial-scale cell therapy manufacturing, recently announced global biopharmaceutical company and cell therapy….
GeoVax Receives Notice of Allowance for Malaria Vaccine Patent
GeoVax Labs, Inc. recently announced the US Patent and Trademark Office has issued a Notice of Allowance for Patent Application No. 17/726,254 titled Compositions and…
Vaxxas Opens World-Class Manufacturing Facility to Produce Proprietary Needle-Free Vaccine Patch For Late-Stage Clinical Trials & First Commercial Products
Vaxxas recently announced the opening of its first, state-of-the-art manufacturing facility in Brisbane, Queensland. The custom-built 60,000-sq-ft Vaxxas Biomedical Facility will serve as….
NKGen Biotech Announces First Patient Dosed in Phase 1 Clinical Trial of Allogeneic NK Cell Therapy Product Candidate for the Treatment of Solid Tumors
NKGen Biotech, Inc. recently announced the first patient has been dosed in a Phase 1, multi-center, open-label, dose-escalation study evaluating its cryopreserved investigational allogeneic blood-derived…
US FDA Approves Orphan Drug Designation for NXC-201 as a Treatment for Multiple Myeloma
Nexcella, Inc. recently announced the US FDA has granted Orphan Drug Designation (ODD) designation for NXC-201 for the treatment of a life-threatening form of blood…
Foundery Launches Inaugural Biotech Venture Creation Fund to Accelerate the Development of Novel Immunotherapies
Foundery’s co-development venture studio model aligns first-in-class drug development opportunities with financing and experimental execution to catalyze university discoveries….
BriaCell Awarded National Cancer Institute Grant to Advance its Bria-OTS Immunotherapy for Cancer
BriaCell Therapeutics Corp. recently announced the National Cancer Institute (NCI), the US federal government's principal agency for cancer research and training, has awarded the company…
Avidity Biosciences Receives FDA Orphan Drug Designation for for Treatment of Duchenne Muscular Dystrophy in People With Mutations Amenable to Exon 44 Skipping
Avidity Biosciences, Inc. recently announced the US FDA has granted Orphan Drug designation to AOC 1044, the company's investigational therapy in development for the treatment…
Tiziana Life Sciences Announces FDA IND Clearance of Intranasal Foralumab for the Treatment of Alzheimer’s Disease
Tiziana Life Sciences Ltd. recently announced the US FDA has cleared the IND application for intranasal foralumab to be studied in Alzheimer’s disease. Foralumab could be a potentially….
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…
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).