Bio Platforms
Axovant Announces Global Licensing Agreement With Benitec Biopharma
Axovant Sciences recently announced it has licensed exclusive global rights to an investigational Silence-and-Replace gene therapy program from Benitec Biopharma for the treatment of oculopharyngeal…
iBio Establishes Strategic Commercial Relationship with CC-Pharming Ltd.
iBio, Inc. recently announced the commencement of a strategic commercial relationship with CC-Pharming Ltd. of Beijing, China for joint development of products and manufacturing facilities…
Codexis Doses First Subjects in Phase 1a Trial; Triggers Milestone
Codexis, Inc. recently announced it has dosed the first subjects in a first-in-human Phase 1a dose-escalation trial with CDX-6114, its orally administered enzyme therapeutic being…
Pfenex Receives Milestone From Merck
Pfenex Inc. recently announced the receipt of a milestone payment from Merck associated with the initiation of the first Phase 3 clinical study of (V114), an investigational polyvalent conjugate vaccine for the prevention of pneumococcal disease.
Immatics Enters Strategic Partnership With Genmab
Immatics Biotechnologies GmbH recently announced it has entered into a research collaboration and license agreement with Genmab A/S to develop next-generation, T-cell engaging bispecific immunotherapies targeting multiple cancer indications.
BioCanCell Announces Completion of $23-Million Financing
BioCanCell Ltd. recently announced it has completed a $22.9-million PIPE financing round. The financing was led by Shavit Capital, with investors from the US, Israel, and…
CrownBio Enters Partnership With Pierre Fabre to Accelerate Discovery & Development of Immuno-Oncology Agents
Crown Bioscience recently announced it has been selected by Pierre Fabre Research Institute as one of their partners to advance their immuno-oncology drug discovery pipeline.…
Celyad Doses First AML Patient at Final Dose Level in CYAD-01 THINK Trial & Begins Dosing First Patient With Second Cycle of Therapy
Celyad recently announced the first Acute Myeloid Leukemia (AML) patient was dosed with the first injections at the third and final dose level of CYAD-01…
Allena Pharmaceuticals Completes Animal Proof-of-Concept Study
Allena Pharmaceuticals, Inc. recently announced the completion of an animal proof-of-concept study supporting the selection of ALLN-346 as its lead product candidate for the treatment…
7 Hills Pharma Launches With $2-Million SBIR Grant & $4 Million in Seed Capital
7 Hills Pharma recently announced it has been awarded a $2-million Small Business Innovation Research (SBIR) grant by the National Institutes of Health (NIH) titled…
Zealand Pharma Achieves Milestone in First Phase 3 Trial
Zealand Pharma A/S recently announced that the primary and key secondary objectives of the first multinational Phase 3 clinical trial of dasiglucagon for the treatment…
FUJIFILM Cellular Dynamics Licenses University of California – Irvine’s Technologies
FUJIFILM Cellular Dynamics, Inc. (FCDI) recently announced it has entered into an exclusive patent license agreement with the University of California – Irvine (UCI) through its offices at UCI Applied Innovation to license and commercialize UCI’s technologies for derivation of microglia in the commercial research field and also a non-exclusive patent license agreement to commercialize microglia media formulation.
eFFECTOR Initiates Dosing of eFT508 in Phase 2 Expansion of Clinical Trial in Aggressive Form of Non-Hodgkin’s Lymphoma
eFFECTOR Therapeutics, Inc. recently announced that it has dosed the first patient in the Phase 2 expansion portion of its monotherapy trial of eFT508, the company’s oral, small molecule inhibitor of MNK1/2, for the treatment of relapsed, refractory non-germinal center B cell (non-GCB) diffuse large B cell lymphoma (DLBCL).
Axovant Licenses Investigational Gene Therapy for Parkinson’s Disease From Oxford BioMedica; Announces Key Leadership Team Addition
Axovant Sciences recently announced that it has licensed the exclusive worldwide rights to develop and commercialize OXB-102, now AXO-Lenti-PD, from Oxford BioMedica. AXO-Lenti-PD is an investigational gene therapy for Parkinson’s disease that delivers three genes encoding a critical set of enzymes required for dopamine synthesis in the brain.
Immunomodulation Therapies Designed to Suppress Tumors & Fight Infection
Immunomodulation Inc. recently outlined its upcoming plans to advance its investigational product candidate, IMM-010, following consistent positive data from preclinical studies.
Novel First-In-Class Immunotherapeutic APVO210 Features Unique Mechanism of Action Delivering IL-10
Aptevo Therapeutics Inc. recently announced the publication of preclinical data in Frontiers in Immunology highlighting the activity of APVO210 as a potent and selective immunosuppressive…
FDA Accepts Larotrectinib NDA & Grants Priority Review
Loxo Oncology, Inc. recently announced the US FDA has accepted the company’s New Drug Application (NDA) and granted Priority Review for larotrectinib for the treatment of adult and pediatric patients with locally advanced or metastatic solid tumors harboring an NTRK gene fusion
Amunix Announces Report by Bioverativ on Unprecedented Half-Life Obtained in Patients Treated With Novel, Long-Acting FVIII Utilizing XTEN Technology
Amunix Operating Inc. recently reported that Bioverativ (a Sanofi company) has announced preliminary Phase 1/2a safety and pharmacokinetic clinical data for BIVV001 (rFVIIIFc-VWF-XTEN), a novel and investigational factor VIII therapy for people with hemophilia A that incorporates Amunix’s XTEN technology to improve circulatory half-life.
Bioasis & WuXi Biologics Announce Initial Strategic Development & Manufacturing Collaboration
Bioasis Technologies, Inc.andWuxi Biologics recently announced an initial strategic collaboration for the development and manufacturing of xB3-001, Bioasis’ lead investigational biological candidate to treat brain cancer.
MimiVax Announces Positive Interim Results from Phase II Trial of SurVaxM
MimiVax LLC recently announced positive interim results from a multicenter Phase II study of SurVaxM in patients with newly diagnosed glioblastoma (nGBM).
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).