Bio Platforms
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….
Vaccitech Reports Promising Interim Efficacy Analysis in Phase 1b/2a Clinical Study in Chronic HBV
Vaccitech plc recently announced a promising interim analysis of safety and efficacy data from the HBV002 study, including a review of surface antigen (HBsAg) levels…
Operations Commence at Genezen’s Lenti- & Retroviral Vector Process Development Lab
Genezen, Inc., a cell and gene therapy Contract Development and Manufacturing Organization (CDMO) focused on early-phase process development, vector production and analytical testing services, has officially….
ASLAN Pharmaceuticals & IQVIA Biotech Enter Strategic Collaboration
ASLAN Pharmaceuticals recently announced the appointment of IQVIA Biotech, a leading global provider of advanced analytics, technology solutions, and clinical research services to the life sciences industry, as its preferred….
Longeveron Granted Orphan Drug Designation by FDA for Lomecel-B to Treat Infants With Hypoplastic Left Heart Syndrome
Longeveron Inc. recently announced the US FDA has granted Orphan Drug Designation (ODD) for Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a…
Fulcrum Therapeutics Announces Additional HBG mRNA Induction From Higher Dose Cohorts in Phase 1 Healthy Adult Volunteer Trial of FTX-6058 for Sickle Cell Disease & New Preclinical Mechanism Data
Fulcrum Therapeutics, Inc. recently announced positive results from the 20-mg and 30-mg dose cohorts in healthy adult volunteers in its Phase 1 clinical trial of…
NGM Bio Announces Clinical Trial Collaboration With Merck Related to Ongoing Phase 1/2 Trial of an ILT2/ILT4 Dual Antagonist Antibody in Combination With Merck’s KEYTRUDA
NGM Biopharmaceuticals, Inc. recently announced a clinical trial collaboration and supply agreement with Merck to evaluate NGM707, NGM’s wholly owned novel ILT2/ILT4 dual antagonist antibody, in combination with….
Enlivex Receives Notice of Allowance for US Patent Application Covering the Treatment of Sepsis Patients With Allocetra
Enlivex Therapeutics Ltd. recently announced the US Patent and Trademark Office issued a notice of allowance for a new patent application (number 16/194,417) covering Allocetra,…
ARCA biopharma Announces Completion of Enrollment in Phase 2b ASPEN-COVID-19 Clinical Trial Evaluating rNAPc2 as a Potential Treatment for Patients Hospitalized With COVID-19
ARCA biopharma, Inc. recently announced that enrollment has been completed in ASPEN-COVID-19, the Phase 2b clinical trial evaluating rNAPc2 as a potential treatment for patients hospitalized…
Progenity Announces New Patent for Single-Molecule Detection Technology
Progenity, Inc. recently announced a new patent related to its single-molecule detection platform under development. The USPTO has issued U.S. Patent No. 11,186,863 titled Methods,…
SAB Biotherapeutics Announces SAB-176 Met its Primary Endpoint in Phase 2a Challenge Study in Adults Infected With Influenza Virus
SAB Biotherapeutics recently announced that SAB-176, its investigational therapeutic for the treatment of seasonal influenza, achieved statistically significant (p = 0.026) reductions in viral load and….
BioXcel Therapeutics Announces Extension of FDA Review Period of its NDA for BXCL501 for the Acute Treatment of Agitation Associated With Schizophrenia & Bipolar Disorders
BioXcel Therapeutics, Inc. recently announced the US FDA has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of BXCL501 for the acute treatment of….
2022 COMPANY PROFILES & CAPABILITIES
For each participating company, this section presents a detailed summary highlighting their core technologies, capabilities, technologies, and services.
APTAMER TECHNOLOGY - From Postal Codes to GPS: Building Better Drug Conjugates With Aptamers
David Bunka, PhD, and Emily Robinson, PhD, focus on a new breed of affinity binders that can be used as an alternative to antibody therapeutics and has the potential to reshape the industry, delivering new medicines with improved safety and efficacy profiles and reduced healthcare costs.
Heat Biologics Unveils a Novel Cellular Vaccine Platform Targeting Known & Unknown Emerging Biological Threats
Heat Biologics, Inc. recently unveiled its new RapidVax cellular vaccine platform at the 2021 World Antiviral Congress. RapidVax is designed as a rapid programmable vaccine…
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).