Bio Platforms
LEXEO Therapeutics Receives Rare Pediatric Disease Designation & Orphan Drug Designation
LEXEO Therapeutics recently announced the US FDA has granted Rare Pediatric Disease designation and Orphan Drug designation to LX2006 for the treatment of….
eTheRNA immunotherapies & Quantoom Biosciences Announce Strategic Collaboration for the Development of a Novel RNA Production
eTheRNA immunotherapies NV and Quantoom Biosciences S.A. recently announced they are to collaborate on the development of a revolutionary RNA production system (RPS) for both research and GMP-grade material…..
Orchard Therapeutics Announces Regulatory & Clinical Updates for Lead Gene Therapy Programs
Orchard Therapeutics recently announced several program updates for the company’s portfolio of approved and investigational hematopoietic stem cell (HSC) gene therapies. These updates follow recent…
CicloMed Announces Initiation of Phase 1B Trial of Fosciclopirox in Newly Diagnosed & Recurrent Urothelial Cancer Patients
CicloMed LLC recently announced that the Phase 1B clinical trial of fosciclopirox for the treatment of bladder cancer is now open. “We are excited to…
Agios Submits MAA to EMA for Mitapivat for Treatment of Adults With Pyruvate Kinase Deficiency
Agios Pharmaceuticals, Inc. recently announced it has submitted a Marketing Authorization Application (MAA) for mitapivat to the European Medicines Agency (EMA) for the treatment of adults with pyruvate kinase….
Veru Announces the Presentation of a Phase 1b/2 Clinical Study Update for Sabizabulin
Veru Inc. recently announced updated clinical results from the ongoing Phase 1b/2 clinical study of sabizabulin (VERU-111), an oral cytoskeleton disruptor being evaluated for the…
Onconova Therapeutics Provides Update on the Phase 1/2a Trial of Rigosertib-Nivolumab Combination in KRAS+ Non-Small Cell Lung Cancer
Onconova Therapeutics, Inc. recently announced an update on the investigator-initiated Phase 1/2a trial of oral rigosertib plus nivolumab in advanced metastatic KRAS mutated (KRAS+) non-small…
H-CYTE Announces Publication of Positive Real-World Data Relating to the Use of Innovative Treatment to Improve Overall Lung Health
H-CYTE, Inc. recently announced the publication of its clinical observational study titled, Longitudinal Assessment of FEV1 Change Following Autologous Cellular Therapy. The study, published in…
Denali Therapeutics Announces Initiation of Phase 2 Study by Partner Sanofi
Denali Therapeutics Inc. recently announced its partner Sanofi has commenced dosing in a Phase 2 study of DNL758 (SAR443122), a peripherally-restricted small molecule inhibitor of…
Catalyst Biosciences Receives FDA Fast Track Designation for Subcutaneous MarzAA for the Treatment of Episodic Bleeding in Factor VII Deficiency
Catalyst Biosciences, Inc. recently announced the US FDA has granted Fast Track Designation for Marzeptacog alfa (activated), MarzAA, the company’s subcutaneously (SQ) administered next-generation engineered…
Idorsia Ltd Announces the Initiation of Phase 3 Registration Study of Antithrombotic Treatment for Use at the Onset of AMI Symptoms
Idorsia Ltd recently announced the initiation of the Phase 3 registration study “SOS-AMI” to evaluate the efficacy and safety of self-administered subcutaneous selatogrel, Idorsia’s P2Y12…
Catalent to Acquire RheinCell Therapeutics, Strengthening a Path Toward Industrialization of Induced Pluripotent Stem Cell-Based Therapies
Catalent recently announced it has reached an agreement to acquire RheinCell Therapeutics GmbH, a developer and manufacturer of GMP-grade human induced pluripotent stem cells (iPSCs)…..
Viking Therapeutics Announces Initiation of Phase 1b Clinical Trial of VK0214 in Patients With X-ALD
Viking Therapeutics, Inc. recently announced the initiation of a Phase 1b clinical trial of VK0214, a novel small molecule agonist of the thyroid hormone receptor beta (TRb), in patients with X-linked….
Fulcrum Therapeutics Announces Results from ReDUX4 Trial With Losmapimod in Facioscapulohumeral Muscular Dystrophy Demonstrating Slowed Disease Progression & Improved Function
Fulcrum Therapeutics, Inc. recently announced results from the company’s Phase 2b trial, ReDUX4, in people with facioscapulohumeral muscular dystrophy (FSHD). Results being presented with….
Prothena Announces Bristol Myers Squibb Opt-In of Anti-Tau PRX005 as the First Program From Global Neuroscience Research & Development Collaboration
Prothena Corporation plc recently announced Bristol Myers Squibb exercised its option under the global neuroscience research and development collaboration to enter into an exclusive US license for….
VectivBio Receives FDA Orphan Drug Designation for Apraglutide for Acute Graft-Versus-Host Disease
VectivBio Holding AG recently announced the FDA has granted orphan drug designation to apraglutide, a next-generation, long-acting GLP-2 analog being developed for rare gastrointestinal (GI)…
Kura Oncology Doses First Patient in Phase 1b Expansion Cohorts With Menin Inhibitor KO-539
Kura Oncology, Inc. recently announced the first patient has been dosed in the Phase 1b portion of KOMET-001, a Phase 1/2 clinical trial of the…
CHMP Recommends Approval of RINVOQ (upadacitinib) for the Treatment of Atopic Dermatitis
AbbVie recently announced the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the approval of RINVOQ…
Aravive Announces Positive Initial Results from Phase 1b Portion of the Phase1b/2 Study of AVB-500 in Combination With Cabozantinib in Clear Cell Renal Carcinoma
Aravive Inc. recently announced positive initial results from the Phase 1b portion of its Phase 1b/2 study in patients dosed with 15 mg/kg of AVB-500…
Genezen Breaks Ground on cGMP Lentiviral Vector Production Facility
Genezen Laboratories, Inc., a cell and gene therapy Contract Development and Manufacturing Organization (CDMO) focused on early-phase process development, vector production, and analytical testing services,…
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).