Bio Platforms
Atsena Therapeutics Receives Orphan Drug Designation for Gene Therapy to Treat X-linked Retinoschisis
Atsena Therapeutics recently announced the US FDA has granted Orphan Drug Designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene…
Orbis Medicines & Vivtex Form Research & Exclusive Licensing Collaboration
Orbis Medicines recently announced a research collaboration and option to an exclusive license with Vivtex, a biotech company aiming to transform the development of oral…
Athira Pharma to Focus on Advancement of ATH-1105 for the Treatment of Neurodegenerative Diseases
Athira Pharma, Inc. recently announced that following the topline data readout from the Phase 2/3 LIFT-AD clinical trial of fosgonimeton to treat Alzheimer’s disease (AD)…
Voyager Advances Collaboration With Neurocrine; Third Gene Therapy Development Candidate Selected
Voyager Therapeutics, Inc. recently announced that the joint steering committee with its collaborator Neurocrine Biosciences, Inc. has selected a development candidate in a gene therapy…
Shuttle Pharma Expands Clinical Trial Site Enrollment for Phase 2 of Ropidoxuridine for Treatment of Patients With Glioblastoma
Shuttle Pharmaceuticals Holdings, Inc. recently announced it has entered into agreements with two additional site locations to administer the Phase 2 clinical trial of Ropidoxuridine…
Vivtex Enters New Research Agreement With Equillium to Develop GI-targeted Formulations for its First-in-Class Oral, Bi-Specific Peptide Therapy
Vivtex Corporation recently announced it has entered a research and licensing agreement with Equillium, Inc., a clinical-stage biotechnology company developing novel therapeutics to treat severe…
Nanoscope Therapeutics Announces End-of-Phase 2 Meeting With FDA & Plan to Initiate a Phase 3 Clinical Trial
Nanoscope Therapeutics Inc. recently announced a productive End-of-Phase 2 (EOP2) meeting with the US FDA for its clinical program evaluating MCO-010 for the treatment of…
Vico Therapeutics Announces Positive Interim Phase 1/2a Clinical Data of VO659 in Treatment of Huntington’s Disease
Vico Therapeutics B.V. recently announced new positive interim data in Huntington’s disease (HD) from the ongoing Phase 1/2a clinical trial of VO659, an investigational allele-preferential…
Biosplice Therapeutics Announces Initiation of AML & MDS Trial Sponsored by the National Cancer Institute
Biosplice Therapeutics, Inc. recently announced the US FDA has cleared the NCI-sponsored Investigational New Drug (IND) application for cirtuvivint. This small molecule inhibitor of CLK…
Vironexis Biotherapeutics Launches With FDA Clearance of IND Application for First-Ever Clinical Trial of an AAV-delivered Cancer Immunotherapy
Vironexis Biotherapeutics launched from stealth, unveiling its TransJoin AAV Gene Therapy Platform and a pipeline of more than ten product candidates for blood-based cancers, solid…
Monopar Announces Positive Early Human Clinical Data Validating the Tumor Targeting Ability of MNPR-101-Zr
Monopar Therapeutics Inc. recently announced positive early data from its ongoing open-label MNPR-101-Zr Phase 1 imaging and dosimetry clinical trial confirming MNPR-101-Zr’s tumor targeting ability…
Cellares & Sony Announce a Joint Development Collaboration to Integrate Flow Cytometry-Based Sorting & Online Analysis Into an Automated, High-Throughput Cell Therapy Manufacturing Platform
Cellares and Sony Corporation recently announced a collaboration to jointly develop and integrate advanced flow cytometry-based cell analysis and sorting solutions into the Cellares Cell…
TFF Pharmaceuticals Announces Positive Preclinical Data From Bivalent Universal Influenza Vaccine Candidates
TFF Pharmaceuticals, Inc recently announced positive preclinical data advancing a multivalent universal influenza vaccine manufactured by TFF to protect against seasonal and pandemic viruses. The…
Agios Announces FDA Orphan Drug Designation Granted for Treatment of Myelodysplastic Syndromes
Agios Pharmaceuticals, Inc. recently announced the US FDA has granted orphan drug designation to the company’s novel pyruvate kinase (PK) activator tebapivat (AG-946) for the…
Ilya Pharma Receives Rare Pediatric Disease Designation From US FDA
Ilya Pharma recently announced that it has received a Rare Pediatric Disease Designation by the US FDA. About SAVI: STING-associated vasculopathy with onset in infancy…
BriaCell Reports Positive Overall Survival in Metastatic Breast Cancer
BriaCell Therapeutics Corp. recently announced positive overall survival data of its Phase 2 clinical study of Bria-IMT in combination with an immune check point inhibitor…
Roivant Unveils New Pipeline Program of a Potential First-in-Class & Best-in-Category Inhaled Once-Daily Soluble Guanylate Cyclase Activator
Roivant recently announced its previously undisclosed pipeline program mosliciguat, a potential first-in-class, inhaled, once-daily sGC activator with targeted delivery to the lungs via dry powder…
Quotient Sciences & Vasa Therapeutics Announce Dosing of First Subjects With Novel Heart Failure Treatment
VS-041, a novel and potentially life-saving treatment for heart failure, developed by Vasa Therapeutics, a San Diego-based biotechnology company, has reached a major clinical milestone…
Incendia Therapeutics Enrolls First Patient in Phase 1c Clinical Trial of a Novel DDR1 Inhibitor
Incendia Therapeutics, a precision oncology company discovering and developing a novel class of therapies that reprogram the tumor microenvironment (TME), recently announced the first patient…
Incannex Healthcare Announces Strategic Financing for up to $60 Million
Incannex Healthcare Inc. recently announced it has entered into an agreement to issue up to $10 million in secured convertible notes to Arena Investors, LP…
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).