Bio Platforms
Ventyx Biosciences Announces $27-Million Strategic Investment From Sanofi
Ventyx Biosciences, Inc. recently announced Sanofi has agreed to make a $27-million strategic investment in the company at an as-converted price of $3.8243 per share…
Hovione & iBET Announce the Establishment of a Venture in Cell & Gene Therapy Solutions
Hovione and iBET recently announced the official establishment of ViSync Technologies as a jointly owned venture. The creation of this new company, headquartered in the…
Tiziana Life Sciences Announces $4-Million Grant to Study Anti-CD3 in Alzheimer’s Disease
Tiziana Life Sciences, Ltd. recently announced the National Institutes of Health (NIH), National Institute on Aging have awarded a $4-million grant to Dr. Howard Weiner…
Oncolytics Biotech Reports Favorable Results for BRACELET-1 Breast Cancer Study
Oncolytics Biotech Inc. recently announced positive clinical results from BRACELET-1, its randomized Phase 2 study evaluating pelareorep in patients with HR+/HER2- advanced or metastatic breast cancer.…
Sonnet BioTherapeutics Completes Enrollment in Phase 1 Study of SON-1010 (IL12-FHAB) as a Monotherapy (SB101) for the Treatment of Solid Tumors
Sonnet BioTherapeutics Holdings, Inc. recently announced the completion of enrollment and initiation of dosing in its Phase 1 SB101 clinical trial of SON-1010 (IL12-FHAB) in…
TransCode Therapeutics Announces First Patients Treated in Phase 1 Clinical Trial With First-in-Class Lead Therapeutic Candidate
TransCode Therapeutics, Inc. recently announced dosing of the first two patients in its Phase 1 clinical trial with its lead candidate, TTX-MC138. Several additional patients…
Cullinan Therapeutics Receives Approval to Initiate its Global Phase 1 Clinical Trial for the Treatment of Systemic Lupus Erythematosus
Cullinan Therapeutics, Inc. received Human Research Ethics Committee (HREC) approval in Australia to initiate its global Phase 1 clinical trial to evaluate CLN-978, its CD19xCD3 bispecific…
AC Immune Receives Second Milestone Payment Following Progress in Phase 2b Trial
AC Immune SA recently announced it will receive the second ReTain-related milestone payment (CHF 24.6 million) under its agreement with Janssen Pharmaceuticals, Inc. (Janssen), a…
ImmuneSensor Therapeutics Granted New US Patent for its Oral cGAS Inhibitors
ImmuneSensor Therapeutics recently announced the United States Patent and Trademark Office (USPTO) has issued US patent No. 12,091,387 containing claims covering compositions of matter and…
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…
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).