Bio Platforms
BioXcel Therapeutics Initiates Pivotal Phase 3 Program of BXCL501 for Acute Treatment of Agitation in Patients With Alzheimer’s Disease
BioXcel Therapeutics, Inc. recently announced the initiation of its pivotal Phase 3 program for BXCL501, the company’s proprietary, orally dissolving thin film formulation of dexmedetomidine,…
SWK Holdings’ Subsidiary, Enteris BioPharma, Receives $5-Million Milestone Payment From Cara Therapeutics
SWK Holdings Corporation recently announced its wholly owned subsidiary, Enteris BioPharma, received a $5- million milestone payment from Cara Therapeutics related to the license agreement for Peptelligence….
WACKER & CordenPharma Develop Solutions for Formulating Lipid Nanoparticles, Expanding Their Portfolio for Advanced Medicines
WACKER and CordenPharma have signed a development partnership in the field of Lipid Nanoparticle formulation. The two companies announced they plan to jointly develop know-how…
Immunic, Inc. Publishes Positive Results From the Single & Multiple Ascending Dose Parts of its Phase 1 Clinical Trial of IMU-935, a Potentially Best-in-Class Oral IL-17 Inhibitor
Immunic, Inc. recently announced positive unblinded safety, tolerability and pharmacokinetic (PK) results from Part A (single ascending doses, SAD) and Part B (multiple ascending doses, MAD)…
Immatics & Bristol Myers Squibb Enter Global Exclusive License for TCR Bispecific Program IMA401
Immatics N.V. and Bristol Myers Squibb recently announced they have entered into a license, development, and commercialization agreement for Immatics’ TCR Bispecific candidate, IMA401…..
Adagio Therapeutics Reports Reduction in In Vitro Neutralizing Activity of ADG20 Against Omicron SARS-CoV-2 Variant
Adagio Therapeutics, Inc. recently provided an update following external in vitro analyses to evaluate neutralizing activity of ADG20 against the Omicron SARS-CoV-2 variant. The in…
atai Life Sciences Announces Successful Outcome of Phase 2a Biomarker Trial of RL-007 in Cognitive Impairment Associated With Schizophrenia
atai Life Sciences recently announced positive topline data from its Phase 2a study of RL-007 for Cognitive Impairment Associated with Schizophrenia (CIAS). The 32-patient, single-arm,…
HiberCell to Collaborate With Merck on Phase 2 Clinical Trial of Odetiglucan in Combination With KEYTRUDA in Patients With Metastatic Breast Cancer
HiberCell recently announced a clinical trial collaboration with Merck. This metastatic breast cancer clinical trial evaluates KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, in combination with….
Vaccitech Acquires Avidea Technologies to Expand Product Pipeline & Strengthen Scientific Leadership in Immunotherapies & Vaccines
Vaccitech plc recently announced that it has acquired US-based Avidea Technologies, Inc. The consideration to Avidea’s existing shareholders is $40 million….
Pfizer to Acquire Arena Pharmaceuticals for $6.7 Billion
Pfizer Inc. and Arena Pharmaceuticals, Inc. recently announced the companies have entered into a definitive agreement under which Pfizer will acquire Arena, a clinical-stage company developing….
Precision BioSciences Reports Clinical Program Updates for Its Allogeneic CAR T Pipeline
Precision BioSciences, Inc. recently announced program updates across its allogeneic CAR T cell therapy pipeline, including updated data for its Phase 1/2a clinical study of PBCAR0191 with enhanced lymphodepletion (eLD) presented at….
Akoya Biosciences Announces Groundbreaking Collaboration With PathAI to Combine Spatial Biology With AI-Powered Tools to Facilitate Discovery of Novel Predictive Biomarkers
Akoya Biosciences, Inc. and PathAI recently announced a collaboration to advance the discovery and validation of novel predictive biomarkers for immunotherapies. The partners will leverage…
NextCure & Collaborators Provide Data on Targets in Development
NextCure, Inc. recently announced new data in oral and poster presentations at the American Society of Hematology (ASH) annual meeting in Atlanta, GA, and on…
Arrivo BioVentures Emerges From Stealth Mode With a Robust Pipeline & Promising Data
After launching the company over 5 years ago with a team of seasoned serial entrepreneurs, $49 million in committed capital, and the promise to assemble…
Halberd Successfully Eradicates Interluekin-1 in Its Continued Progress Toward Eliminating Neurodegenerative Diseases
Halberd Corporation has demonstrated 100% eradication of Interleukin-1 (IL-1) from synthetic cerebral spinal fluid (CSF) in preliminary testing. Interleukin-1 is associated with numerous diseases ranging from autoimmune…
Intensity Therapeutics Reports Promising Early Efficacy Results Using INT230-6 as a Monotherapy or in Combination With Pembrolizumab in Metastatic Breast Cancer
Intensity Therapeutics, Inc. recently reported safety, pharmacokinetic, biomarker, and efficacy data using INT230-6, with and without pembrolizumab, in heavily pretreated refractory breast cancer patients as part of the….
UniQure Announces Pivotal Phase 3 HOPE-B Study Meets Primary Endpoint for Hemophilia B Gene Therapy
CSL Behring and uniQure N.V. recently announced that etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe to moderately…
Checkpoint Therapeutics Announces Initiation of CONTERNO Phase 3 Trial of Cosibelimab Combined With Chemotherapy in Patients With First-Line Non-Squamous Non-Small Cell Lung Cancer
Checkpoint Therapeutics, Inc. recently announced the initiation of the CONTERNO study, a global, randomized Phase 3 trial of cosibelimab in combination with pemetrexed and platinum…
BrainStorm Cell Therapeutics & Catalent Announce Completion of Technology Transfer for NurOwn Manufacturing
BrainStorm Cell Therapeutics Inc. and Catalent recently announced that the technology transfer for NurOwn manufacturing at Catalent’s facility has been finalized….
Fortress Biotech, Cyprium Therapeutics & Sentynl Therapeutics Announce the Initiation of Rolling Submission of an NDA for Treatment of Menkes Disease
Cyprium Therapeutics, Inc., a Fortress Biotech, Inc. partner company, with support from its licensing partner Sentynl Therapeutics, Inc., a wholly owned subsidiary of Cadila Healthcare Limited, recently announced the initiation of a rolling submission of a New Drug Application….
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).