Bio Platforms
CureVac Doses First Participant in Phase 1 Study With Multivalent Influenza Vaccine Candidate Based on Second-Generation mRNA Backbone Developed in Collaboration With GSK
CureVac N.V. recently announced it has dosed the first participant in a Phase 1 study of its seasonal influenza second-generation mRNA vaccine candidate, CVSQIV, developed…
Denali Therapeutics Announces Continued Progress in DNL310 Program for MPS II (Hunter Syndrome) Supporting Planned Initiation of Phase 2/3 Clinical Trial
DNL310 is an investigational brain-penetrant enzyme replacement therapy intended to treat both central nervous system (CNS) and peripheral manifestations of MPS II (Hunter syndrome). ….
Dyadic & Phibro Animal Health Announce Exclusive License Agreement to Develop & Commercialize Animal Health Vaccine
Dyadic International, Inc. and Phibro Animal Health recently announced they have entered into an exclusive license agreement for a Phibro targeted disease. This agreement follows…
SEngine Precision Medicine & Oncodesign Announce Collaboration Agreement for the Discovery of New Personalized Cancer Treatment for Aggressive & Untreatable Tumors
SEngine Precision Medicine Inc. and Oncodesign recently announced the signature of a research collaboration agreement for R&D of a new personalized cancer treatment for aggressive and…..
Xanadu Bio Obtains Licenses & Options to Novel Platform Technologies From Yale University to Develop Intranasal SARS CoV-2 mRNA Vaccine Booster
Xanadu Bio recently announced a broad exclusive license agreement from Yale University for a next-generation polymeric nanoparticle delivery platform known as PACE. Additionally, the company has executed….
Ovid Therapeutics & Healx to Enter Strategic Partnership
Ovid Therapeutics and Healx recently announced Healx has secured from Ovid an exclusive option to license rights to develop and commercialize gaboxadol. Under the agreement, Healx plans to….
Kintara Therapeutics Announces Issuance of New US Patent Related to VAL-083 & MGMT Resistance: Implications for Targeting Brain Tumor Stem Cells
Kintara Therapeutics, Inc. recently announced the US PTO has issued to Kintara United States Patent No. 11,234,955 covering a method of treating brain tumors including…
Adamis Pharmaceuticals Provides Enrollment Update of Subjects in Phase 2/3 Study of Tempol for the Treatment of COVID-19
Adamis Pharmaceuticals Corporation recently announced the enrollment and dosing of more than 100 subjects in the Company’s ongoing Phase 2/3 study of Tempol for the treatment of….
POP Biotechnologies Vaccine Technology to Enter Phase 3 Clinical Studies as Part of Eubiologics' COVID-19 Vaccine Candidate, EuCorVac-19
POP Biotechnologies recently announced the approval of the plan for a Phase 3 clinical study of EuCorVac-19, a COVID-19 vaccine candidate developed by South Korean…
Evelo Biosciences Announces Highly Significant Reductions in Clinically Validated Inflammatory Cytokines in EDP1815 Phase 2 Psoriasis Trial
Evelo Biosciences, Inc. recently announced the results of immunological biomarker analyses from its previously reported Phase 2 trial of orally-dosed EDP1815 in mild and moderate…
Theralase Demonstrates Proof-of-Concept for Canadian-Made COVID-19 Vaccine
Theralase Technologies Inc. has demonstrated proof-of-concept for the development of a Canadian-made SARS-CoV-2 (COVID-19) vaccine…..
uniQure Announces Dosing of First Patients in European Open-Label Clinical Trial of AMT-130 Gene Therapy in Huntington’s Disease
uniQure N.V. recently announced the dosing of the first two patients in its European open-label Phase 1b/2 clinical trial of AMT-130, a potential one-time gene-therapy…
NGM Bio’s NGM621 Receives Fast-Track Designation for the Treatment of Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration
NGM Biopharmaceuticals, Inc. recently announced the US FDA has granted Fast-Track designation to NGM621, a monoclonal antibody product candidate engineered to potently inhibit complement C3, for the treatment of….
Entasis Therapeutics Receives Acquisition Proposal From Existing Majority Stockholder Innoviva Inc.
Entasis Therapeutics Holdings Inc. recently announced its Board of Directors received a preliminary, non-binding proposal from its majority stockholder Innoviva, Inc. to acquire all….
Immunic Receives Notice of Allowance for Composition-of-Matter Patents in the US & Europe for IMU-935, a Potentially Best-in-Class Oral IL-17 Inhibitor
Immunic, Inc. recently announced the company received a Notice of Allowance from the USPTO for patent application 16/644581 titled IL-17 and IFN-gamma inhibition for the…
ImCheck Announces First Patients Dosed in Phase 2 of EVICTION Trial for ICT01
ImCheck Therapeutics recently announced the first patients have been dosed in the Phase 2a monotherapy expansion arm in the ongoing Phase 1/2a EVICTION clinical trial…
Pardes Biosciences Announces FDA Clearance of IND Application for Oral Antiviral Drug Candidate for the Treatment & Prevention of SARS-CoV-2 Infections
Pardes Biosciences, Inc. recently announced its Investigational New Drug (IND) application for PBI-0451 has been cleared by the US FDA. “As we enter our third…
Arch Scientists Publish Data on the Mechanism of Action & Efficacy of Lead Drug Candidate LSALT Peptide in the Prevention of Acute Kidney Injury
Arch Biopartners Inc. recently announced a scientific team led by Dr. Daniel Muruve at the University of Calgary, and their collaborators, have published a paper…
Pharmazz Inc. Submits INDA to India Central Drugs Standard Control Organization for Phase 2 Clinical Trial of Sovateltide in Hypoxic-Ischemic Encephalopathy in Neonates
Pharmazz, Inc. recently announced it has submitted an Investigational New Drug Application (INDA) to the India Central Drugs Standard Control Organization (CDSCO) for a Phase…
Silo Pharma Extends Exclusive Option Agreement for Homing Peptides
Silo Pharma, Inc. recently announced it has extended its exclusive option agreement with the University of Maryland, Baltimore, to explore a novel invention generally known as….
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).