Bio Platforms
Editas Medicine Receives FDA Rare Pediatric Disease Designation for the Treatment of Beta Thalassemia
Editas Medicine, Inc. recently announced US FDA granted Rare Pediatric Disease designation to EDIT-301, an investigational, gene-edited medicine for the treatment of beta thalassemia. The…
Catalent Acquires Commercial-Scale Cell Therapy Development & Manufacturing Facility From Erytech
Catalent recently announced it has acquired from Erytech Pharma its state-of-the-art, commercial-scale cell therapy manufacturing facility in Princeton, NJ, for $44.5 million. The deal includes…
Priothera Receives FDA Clearance of IND to start Phase 2b/3 Study With Mocravimod in AML Patients Undergoing Allogeneic HSCT
Priothera Ltd recently announced the US FDA has provided clearance to proceed with the company's Investigational New Drug (IND) application to begin its pivotal Phase…
EpicentRx BETA-PRIME Phase 1 Study Progresses After Demonstrating Good Safety & Tolerability
EpicentRx Inc. recently announced the initiation of the next stage of its Phase 1 trial, BETA-PRIME, with AdAPT-001, a TGF-beta (TGFß) trap-enhanced cancer targeting adenovirus.…
Brooklyn ImmunoTherapeutics Announces Establishment of New R&D Facility for Advancement of Engineered Cellular Medicines
Brooklyn ImmunoTherapeutics, Inc. recently announced the establishment of a new corporate and research and development facility at The Boardwalk at Science Center Drive in San Diego, CA, to support translational….
Cocrystal Pharma Collaborates With the NIAD to Evaluate COVID-19 Protease Inhibitors
Cocrystal Pharma, Inc. recently announced a Non-Clinical Evaluation Agreement (NCEA) with the National Institute of Allergy and Infectious Diseases (NIAID) for exploratory preclinical studies to….
Enzyvant Announces Plans to Expand Regenerative Medicine Manufacturing Capabilities
Enzyvant recently announced plans to develop a Good Manufacturing Practice (GMP)-compliant regenerative medicine manufacturing facility in Morrisville, NC, part of the Research Triangle Park area.…
Blue Water Vaccines Presents New Data Supporting Universal Influenza Vaccine Candidate
Blue Water Vaccines Inc. recently issued the below statement regarding its Form 8-K filed with the Securities and Exchange Commission (SEC) on April 20, 2022,…
Ultragenyx Announces First Patient Dosed in Pivotal Phase 2/3 Clinical Study for the Treatment of Osteogenesis Imperfecta
Ultragenyx Pharmaceutical Inc. recently announced the first patient has been dosed in a clinical study for the treatment of Osteogenesis Imperfecta (OI) in patients….
Processa Pharmaceuticals Doses First Patient in Amended Phase 1B Protocol for PCS6422 Combined With Capecitabine (Next-Generation Capecitabine)
Processa Pharmaceuticals, Inc. recently announced the first patient with advanced, refractory gastrointestinal cancer has been dosed in its amended maximum tolerated dose (MTD) Phase 1B…
BioXcel Therapeutics Announces Formation of OnkosXcel Therapeutics to Develop Medicines Focused in Oncology
BioXcel Therapeutics, Inc. recently announced the formation of wholly owned subsidiary OnkosXcel Therapeutics, Inc. to develop transformative medicines in….
KemPharm Doses First Subject in Phase 1 Clinical Trial Evaluating Cardiovascular Safety of Serdexmethylphenidate
KemPharm, Inc. recently announced the first subject has been dosed in a Phase 1 clinical trial designed to assess the relative cardiovascular safety of serdexmethylphenidate…
TFF Pharmaceuticals Announces Safety & Pharmacokinetic Data From Phase 1 Study of Niclosamide Inhalation Powder
TFF Pharmaceuticals, Inc. recently announced safety and pharmacokinetic (PK) data from its Phase 1 study of Niclosamide Inhalation Powder, which is being developed for the…
NEC & Shionogi Enter Strategic Research Collaboration for Novel Hepatitis B Therapeutic Vaccine
NEC Corporation and Shionogi & Co., Ltd. recently announced the execution of a strategic research collaboration agreement for the development of a novel hepatitis B therapeutic….
Black Diamond Therapeutics Announces First Patient Dosed in Phase 1 Study for MasterKey Inhibitor of EGFR for the Treatment of Glioblastoma & Non-Small Cell Lung Cancer
Black Diamond Therapeutics, Inc. recently announced the dosing of the first patient in the Phase 1 study evaluating BDTX-1535, a MasterKey inhibitor of epidermal growth…
Ocugen to Commercialize COVAXIN in Mexico, Rights Now Encompassing All of North America
Ocugen, Inc. and Bharat Biotech recently announced they have entered into an amendment to their Co-development, Supply and Commercialization Agreement to expand Ocugen’s exclusive….
Kinnate Biopharma Announces First Patient Dosed in Phase 1 Clinical Trial
Kinnate Biopharma Inc. recently announced the first patient has commenced treatment in its Phase 1 KN-4802 (NCT05242822) clinical trial evaluating its lead Fibroblast Growth Factor…
Halozyme to Acquire Antares Pharma to Create a Specialty Product & Drug Delivery Leader
Halozyme Therapeutics, Inc. and Antares Pharma, Inc. recently announced the companies have entered into a definitive agreement pursuant to which Halozyme will acquire Antares for….
Imcyse Announces First Patient Dosed in Adaptive Phase 1/2 Clinical Trial for Multiple Sclerosis
Imcyse recently announced the first patient has been dosed in the adaptive Phase 1/2 clinical trial evaluating Imotope IMCY-0141 in patients with relapsing-remitting multiple sclerosis….
BriaCell Receives FDA Fast Track Approval for Targeted Breast Cancer Immunotherapy
BriaCell Therapeutics Corp. recently announced the US FDA has granted Fast Track status to BriaCell’s lead candidate, Bria-IMT, for the treatment of metastatic breast cancer…
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).