Bio Platforms
Apellis & Sobi Announce Positive Topline Results From Phase 3 Study of Pegcetacoplan in C3G & Primary IC-MPGN
Apellis Pharmaceuticals, Inc. recently announced positive topline results from the Phase 3 VALIANT study investigating systemic pegcetacoplan in patients with C3 glomerulopathy (C3G) or primary immune…
Palisade Bio Selects Dose for Upcoming Phase 1 Clinical Study of PALI-2108 for Ulcerative Colitis
Palisade Bio, Inc. provided a progress update on the advancement of development for its lead drug candidate, PALI-2108 towards initiation of Phase 1 clinical trials in…
Oragenics Announces Concussion Drug Successfully Clears FDA-Required Cardiotoxicity Testing
Oragenics, Inc. recently announced its lead candidate for treating concussion successfully completed a study that indicates ONP-002 does not cause cardiotoxicity. ONP-002 is a new…
Nutcracker Therapeutics Announces Publication in ACS Nano Demonstrating the Therapeutic Viability of Nutshell Delivery Vehicles
Nutcracker Therapeutics, Inc. recently announced the publication of its latest peer-reviewed paper, which explores the fundamental aspects of its Nutshell platform of peptoid-based mRNA delivery vehicles.…
Agios to Receive $1.1 Billion in Milestone Payments
Agios Pharmaceuticals, Inc. recently announced it will receive a total of $1.1 billion in milestone payments following US FDA approval of vorasidenib for adult and…
Silo Pharma Announces Partnership With Global CRO for CNS Homing Peptide
Silo Pharma, Inc. recently announced it has entered into an agreement with WuXi AppTec (Hong Kong) Limited for a preclinical small animal study of SPU-16,…
Levicept Announces Positive Results of Phase 2 Trial of Novel Neurotrophin-3 Inhibitor for the Treatment of Patients With Moderate to Severe Osteoarthritis
Levicept Ltd recently announced positive results from its Phase 2 trial of LEVI-04 a novel and first in class neurotrophin-3 (NT-3) inhibitor, showing that LEVI-04…
George Medicines Files NDA for Novel Low Dose Triple Combination for Treatment of Hypertension Following Successful Phase 3 Development Program
George Medicines recently announced its submission to the US FDA of a New Drug Application (NDA) for GMRx2, George Medicines’ lead pipeline candidate for the…
Annovis’ Lead Compound Enhances Cognition Synergistically with GLP-1 Agonist
Annovis Bio Inc. recently announced new preclinical data demonstrating the synergistic effect of its lead compound, buntanetap, when combined with the glucagon-like peptide 1 (GLP-1)…
Compass Therapeutics Completes Patient Enrollment in Randomized Study of CTX-009 in Combination With Paclitaxel, and Provides an Update on the Development Plan in Biliary Tract Cancer
Compass Therapeutics, Inc. recently announced it completed enrollment of the planned 150 patients in COMPANION-002, its randomized Phase 2/3 clinical trial of CTX-009 in patients…
NeOnc Technologies Begins Enrollment for Phase 2 Clinical Trial of NEO100-02 for Patients Afflicted With Meningioma, Marking Second Concurrent Phase 2 Study of Leading CNS Cancer Drug Candidate
NeOnc Technologies Holdings, Inc. has begun patient enrollment for the Phase 2 clinical trial of NEO100-02, the company’s first of two drug candidates proceeding through…
Salubris Biotherapeutics Announces Approval to Initiate Phase 1 Clinical Trial of a 5T4-Targeted ADC in Solid Tumors
Salubris Biotherapeutics, Inc. recently announced it has received approval from the European Medicines Agency (EMA) to initiate a Phase 1 clinical trial of JK06 in…
MBX Biosciences Raises $63.5 Million to Advance Precision Endocrine Peptide Platform
MBX Biosciences, Inc. recently announced a $63.5-million Series C financing led by Deep Track Capital, with participation from new investors Driehaus Capital Management and funds…
FDA Accepted BLA for the First & Only Ticagrelor Reversal Agent for Filing & Priority Review
SFJ Pharmaceuticals, (SFJ), Sponsor of the bentracimab Biologics License Application (BLA), and SERB Pharmaceuticals (SERB), who acquired exclusive US rights to bentracimab from SFJ and…
FDA Approves Oral JAK Inhibitor for the Treatment of Severe Alopecia Areata
Sun Pharmaceutical Industries Limited recently announced the US FDA approved LEQSELVI (deuruxolitinib) 8 mg tablets for the treatment of adults with severe alopecia areata. Alopecia…
AC Immune Unveils Novel Therapeutic ADC Technology for Improved Efficacy in Neurodegenerative Diseases
AC Immune SA recently unveiled a novel class of neurodegenerative disease-fighting drug-candidates called morADC (Morphomer Antibody Drug Conjugate) in an oral presentation at the annual…
GeoVax Announces Phase 2 Plans for Cancer Therapy Following Clinical Advisory Committee Review
GeoVax Labs, Inc. recently announced the next steps for the clinical development of its Gedeptin cancer therapy, following a clinical advisory committee review. GeoVax and…
Vaccinex Reports Positive Data for SIGNAL-AD Phase 1b/2 Trial of Pepinemab in Alzheimer’s Disease
Vaccinex, Inc. recently announced positive results of its early stage SIGNAL-AD clinical trial of pepinemab antibody in AD. Topline results were presented by Eric Siemers,…
Ocular Therapeutix Announces First Patients Enrolled in Phase 3 SOL-R Wet AMD Study
Ocular Therapeutix, Inc. recently announced the first patients have been enrolled in the Phase 3 SOL-R clinical trial evaluating repeat dosing of AXPAXLI (axitinib intravitreal…
Anavex Life Sciences Announces Translational Biomarker Data for ANAVEX2-73 in Fragile X Syndrome (Major Cause of Autism)
Anavex Life Sciences Corp. recently reported positive preclinical results in directly to humans’ translatable biomarkers for individuals with FXS for ANAVEX2-73 (blarcamesine), in a disease…
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).