Bio Platforms
MitoRx Therapeutics Announces Publication Showing Mitochondrial Sulfide Donor AP39 Significantly Alleviates Obesity
MitoRx Therapeutics recently announced a publication in Elsevier’s prestigious biomedical sciences journal Pharmacological Research. The work led by collaborators at Jagiellonian University Medical College showed…
TransCode Therapeutics Completes Initial Dosing of First Cohort in Phase 1 Trial With a microRNA-Targeted Technology
TransCode Therapeutics, Inc. recently announced it has dosed all patients in the first cohort of its Phase 1a dose-escalation clinical trial. The therapeutic candidate being…
Nanoscope Announces Plans to Submit BLA for Retinitis Pigmentosa Treatment
Nanoscope Therapeutics Inc. recently announced a productive FDA meeting for its clinical program evaluating MCO-010 for the treatment of retinitis pigmentosa (RP). Based on the regulatory…
Palisade Bio Cleared to Commence Phase 1 Clinical Study for Ulcerative Colitis Treatment
Palisade Bio, Inc. recently announced it has received a No Objection Letter from Health Canada for its Phase 1 human clinical study for PALI-2108 for…
FDA Grants Orphan Drug Designation to Can-Fite’s Namodenoson for Pancreatic Cancer
Can-Fite BioPharma Ltd. recently announced the its oncology drug candidate, Namodenoson, has been granted Orphan Drug Designation by the US FDA for the indication of…
Panavance Therapeutics Provides Update on Phase 1 Clinical Trial of Misetionamide in Pancreatic Cancer
Panavance Therapeutics Inc. recently announced the presentation of interim data from its Phase 1 clinical study evaluating misetionamide (GP-2250) in combination with gemcitabine at the 2024…
Sonnet BioTherapeutics Enters Licensing Agreement With Alkem Laboratories
Sonnet BioTherapeutics Holdings, Inc. recently announced it has entered into a licensing agreement with Alkem Laboratories Limited for the research, development, manufacturing, marketing, and commercialization…
HBC Immunology Completes In Vivo Drug Lead Studies for Prostrate Cancer Treatment
HBC Immunology (HBCI) recently announce the successful completion of its prostate cancer treatment xenograft studies with its lead peptide FT-002a in a novel oral formulation…
Qinotto & Lilly Enter Research Collaboration & License Agreement
Qinotto, Inc. recently announced it has entered a research collaboration and license agreement with Eli Lilly and Company (Lilly) to discover next-generation antibody-based vehicles for…
Mestag Therapeutics Announces License & Research Collaboration With MSD
Mestag Therapeutics recently announced it has entered into a license and collaboration agreement with MSD (tradename of Merck & Co., Inc., Rahway, NJ) to identify…
Avalo Announces First Patient Dosed in Phase 2 Trial of AVTX-009 for the Treatment of Hidradenitis Suppurativa
Avalo Therapeutics, Inc. recently announced the first patient has been dosed in its Phase 2 LOTUS trial of AVTX-009 in hidradenitis suppurativa (HS). AVTX-009 is…
MiNK Therapeutics & Autonomous Therapeutics Announce Collaboration to Develop Novel Therapies Targeting Metastatic Tumors
MiNK Therapeutics and Autonomous Therapeutics, Inc. recently announced a research collaboration aimed at effectively targeting and treating metastatic tumors. This collaboration will leverage Autonomous’ precision…
Traws Pharma Announces Positive Topline Phase 1 Data for Flu Candidate
Traws Pharma, Inc. recently announced positive topline Phase 1 safety and pharmacokinetic results for its investigational one-dose influenza (flu) therapy, tivoxavir marboxil (tivoxavir). Tivoxavir was…
ImmuneSensor Therapeutics Initiates Dosing in Phase 1 Trial of Lead cGAS Inhibitor Drug Candidate
ImmuneSensor Therapeutics recently announced the first dose level cohort of healthy volunteers has been dosed in a Phase 1 randomized, double-blinded, placebo-controlled clinical trial of…
GRIN Therapeutics Announces Initiation of Astroscape Clinical Trial of Radiprodil for Treatment of Tuberous Sclerosis Complex & Focal Cortical Dysplasia Type II
GRIN Therapeutics Inc. recently announced initiation of the Astroscape study, its global open-label clinical trial of radiprodil, an investigational, selective and potent negative allosteric modulator…
NeOnc Technologies Receives Pharma Tech Outlook’s Top Drug Delivery Platform Award for 2024
NeOnc Technologies Holdings, Inc. has been recognized by Pharma Tech Outlook as the Top Drug Delivery Platform of 2024. According to the publisher of the…
Avillion Announces Positive High-Level Results From Phase 3 BATURA Trial With AstraZeneca’s Inhaled Anti-Inflammatory Medication Airsupra in Asthma Patients
Avillion Life Sciences Ltd recently announced the ongoing BATURA Phase 3b trial evaluating AstraZeneca’s Airsupra (albuterol/budesonide) in patients with intermittent or mild persistent asthma has…
Microbiotica Announces First Patient Dosed in its International Phase 1b Trial, MELODY-1 of MB097 Precision Microbiome Co-Therapy in Advanced Melanoma
Microbiotica recently announced the first patient has been dosed in its advanced melanoma (MELODY-1) trial. This international trial is due to recruit up to 40…
Cybrexa Therapeutics Announces First Patient Dosed With First-in-Class Peptide-Drug Conjugate in Phase 2 Trial in Ovarian Cancer
Cybrexa Therapeutics recently announced the first patient has been dosed in a Phase 2 clinical trial evaluating CBX-12 in patients with platinum-resistant or refractory ovarian…
Abivax Announces First Patient Enrolled in Phase 2b Trial of Obefazimod in Crohn’s Disease
Abivax SA recently announced the first patient was enrolled in its Phase 2b ENHANCE-CD (NCT06456593) trial evaluating obefazimod in patients with Crohn’s disease (CD). The…
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).