Bio Platforms
Cue Biopharma Receives FDA Acceptance of IND Application for CUE-102 in Wilms’ Tumor 1 (WT1) - Expressing Cancers
Cue Biopharma, Inc. recently announced the US FDA has accepted the company's Investigational New Drug (IND) application for the evaluation of CUE-102, its second interleukin…
Immatics Initiates Phase 1 Clinical Trial to Evaluate Lead TCR Bispecific IMA401 in Patients With Advanced Solid Tumors
Immatics N.V. recently announced the initiation of a Phase 1 clinical trial with its T cell engaging receptor (TCER) IMA401 for patients with recurrent and/or refractory….
Evaxion Biotech Announces Successful Production of Personalized Cancer Immunotherapies in Phase 1/2a Clinical Trial
Evaxion Biotech A/S recently announced it has successfully produced all batches of personalized cancer immunotherapies for all patients enrolled in the Phase 1/2a clinical trial…
AUM Biosciences Enters Collaboration With Roche for Clinical Development of AUM001 in Combination With Anti-PD-L1 Therapy in Solid Tumor Indications
AUM Biosciences recently announced it has entered into a clinical trial collaboration and supply agreement with Roche to evaluate AUM001, the company’s novel, highly selective….
MoonLake Immunotherapeutics Announces Publication of New Long-Term Disease Control Data From Phase 2b Psoriasis Trial
MoonLake Immunotherapeutics AG recently announced the British Journal of Dermatology (BJD) has published a new data analysis, which can be accessed in the BJD online library, from the Phase 2b clinical trial assessing the effect of sonelokimab….
Imago BioSciences Announces First Participant Dosed in Investigator-Sponsored Study of Bomedemstat in Combination With Atezolizumab in Small Cell Lung Cancer
Imago BioSciences, Inc. recently announced the first participant has been treated in an investigator-sponsored Phase 1/2 study of bomedemstat in combination with atezolizumab (Tencentriq) during…
Lonza & ALSA Ventures Collaborate to Provide Development & Manufacturing Services for Biotech Firms
ALSA Ventures, a London based European biotech investment firm, and Lonza, a global development and manufacturing partner to the pharma, biotech, and nutrition industries, recently…
Entera Bio Files Patent Applications for Inventions that Optimize its Platform Technology for Oral Delivery of Large Molecule Therapeutics
Entera Bio Ltd. recently announced it recently filed multiple US patent applications to further strengthen the company’s patent protection and support future developments. These patent…
Amphista & Bristol Myers Squibb to Collaborate & Leverage Proprietary Eclipsys Targeted Protein Degradation Platform to Develop Novel Protein Degrading Therapeutics
Collaboration includes an upfront payment of $30 million, the potential for up to $1.25 billion in performance-based milestone payments and payments for a limited expansion of the collaboration, as well as royalties on global net sales of products….
Adial Pharmaceuticals Announces Research Collaboration With UC San Diego
Adial Pharmaceuticals, Inc. recently announced Purnovate, Inc., an Adial company focused on developing adenosine analogs for the treatment of major….
Novartis' Kymriah Receives EC Approval as First CAR-T Cell Therapy for Adults With Relapsed or Refractory Follicular Lymphoma
Novartis recently announced the European Commission (EC) has approved Kymriah (tisagenlecleucel), a CAR-T cell therapy, for the treatment of adult patients with relapsed or refractory (r/r)…
Compass Therapeutics Reports Positive Interim Phase 2 Data
Compass Therapeutics, Inc. recently reported additional interim results from a Phase 2 study of CTX-009 in combination with paclitaxel in patients with biliary tract cancers…
BioXcel Therapeutics Announces First Patient Dosed in TRANQUILITY II Phase 3 Trial for Acute Treatment of Agitation in Patients With Alzheimer’s Disease
BioXcel Therapeutics, Inc. recently announced the first patient has been dosed in the Phase 3 TRANQUILITY II study of BXCL501, the company’s proprietary, orally dissolving…
DepYmed Receives FDA Rare Pediatric Disease & Orphan Drug Designations for its Lead Clinical Candidate for the Treatment of Rett Syndrome
DepYmed Inc. recently announced that the US FDA has granted Rare Pediatric Disease and Orphan Drug designations for its lead clinical candidate, a small molecule…
GLOBAL REPORT – 2021 Global Drug Delivery & Formulation Report: Part 2, Notable Drug Delivery and Formulation Product Approvals and Technologies of 2021
In part 2 of this 3-part series, PharmaCircle, in collaboration with Drug Development & Delivery, focuses on notable drug delivery and formulation product approvals.
REGENERATIVE MEDICINE - Delivering on the Promise of Regenerative Medicine in Type 1 Diabetes
Thomas Donner, MD, explains how advances in stem-cell engineering, immune-evasion technologies, and vascularization of implanted cells have the potential to generate novel therapeutics that could lead to reduced treatment burden for patients with T1D and infuse new energy into efforts at finding a cure.
Vaxxinity Receives FDA Fast Track Designation for UB-311 for Treatment of Alzheimer’s Disease
Vaxxinity, Inc. recently announced that UB-311, an anti-amyloid beta immunotherapeutic vaccine, has been granted Fast Track designation by the US FDA for the treatment of Alzheimer’s disease…..
FDA Has Granted Veru a Pre-Emergency Use Authorization Meeting Date for Positive Sabizabulin Phase 3 COVID-19 Study
Veru Inc. recently announced the FDA has granted the company a pre-Emergency Use Authorization (EUA) meeting on May 10, 2022, for the positive Phase 3…
Denali Therapeutics Announces Achievement of RIPK1 Milestone for Phase 2 Clinical Trial Initiation in ALS by Sanofi
Denali Therapeutics Inc. recently announced its partner Sanofi has commenced dosing in a Phase 2 study, named HIMALAYA, of SAR443820 (DNL788) in individuals with amyotrophic…
Harpoon Therapeutics & Roche to Collaborate on Clinical Trials to Study Novel Immuno-Oncology Combination for Small Cell Lung Cancer
Harpoon Therapeutics, Inc. recently announced it has entered into a clinical supply agreement with F. Hoffmann-La Roche Ltd (Roche) to supply atezolizumab (Tecentriq) for use in 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).