Bio Platforms
PharmaCyte Biotech Will Have All the Protection It Needs Upon Marketing Approval
PharmaCyte Biotech recently announced it is about to embark upon a planned US FDA Phase 2b clinical trial to treat locally advanced, inoperable pancreatic cancer (LAPC) at trial sites all over the US, and with that journey comes the….
Catalent to Acquire Leading Cell Therapy Company MaSTherCell Global
Catalent, Inc. recently announced it has agreed to acquire MaSTherCell Global, a company backed by Great Point Partners, SFPI-FPIM, and Orgenesis Inc. for an aggregate of…..
Conatus Pharmaceuticals & Histogen Enter Definitive Merger Agreement
Conatus Pharmaceuticals Inc. and Histogen Inc. recently announced the companies have entered into a definitive agreement under which Histogen will merge with a wholly owned subsidiary of….
Albireo Achieves Significant Milestones for Odevixibat Across Multiple Diseases
Albireo Pharma, Inc. recently announced a number of advances and new initiatives in the clinical program for odevixibat, an oral once daily capsule in development for….
BioPharmX & Timber Pharmaceuticals Announce Merger Agreement
BioPharmX Corporation and Timber Pharmaceuticals LLC recently announced they have entered into a definitive merger agreement. Under the terms of the merger agreement, subject to the approval of…..
Rubius Therapeutics Announces First Patient Dosed in Phase 1b Trial of RTX-134
Rubius Therapeutics, Inc. recently announced that the first patient has been dosed in its Phase 1b trial of RTX-134, an allogeneic, off-the-shelf cellular therapy for…
Avacta’s Proprietary TMAC Cancer Therapy Platform Demonstrates Successful Proof-of-Concept
Avacta Group plc recently announced it has demonstrated initial proof-of-concept for its proprietary new class of drug conjugate, TMAC, in a preclinical animal model of…
Cabaletta Bio Receives Orphan Drug Designation for DSG3-CAART
Cabaletta Bio, Inc. recently announced that the US FDA has granted Orphan Drug Designation for the company’s lead product candidate, DSG3-CAART, for the treatment of…
Akcea & Ionis Report Positive Topline Phase 2 Study Results
Akcea Therapeutics, Inc. and Ionis Pharmaceuticals, Inc. recently announced positive topline results from the Phase 2 study of AKCEA-ANGPTL3-LRx in patients with hypertriglyceridemia, type 2…
GeoVax & BravoVax to Collaborate on Development of Coronavirus Vaccine
GeoVax Labs, Inc. and BravoVax recently announced the signing of a Letter of Intent to jointly develop a vaccine against the new coronavirus (known as…
Krystal Biotech Breaks Ground On Second Commercial Gene Therapy Manufacturing Facility
Krystal Biotech, Inc. recently announced the ground breaking of the second commercial gene therapy facility in Findlay Township, PA. The Findlay-based Current Good Manufacturing Practice…
Catalent Biologics Supports DiaMedica's New Treatment
Catalent recently welcomed the news that DiaMedica Therapeutics has received US FDA approval to commence Phase 2 clinical trials of its lead candidate, DM199, for…
Ritter Pharmaceuticals & Qualigen Announce Merger Agreement
Ritter Pharmaceuticals, Inc. recently reported it has entered into a definitive reverse merger agreement with Qualigen, Inc. as well as expansion of its flagship FastPack diagnostic platform, whereby a wholly owned…..
Codexis & Nestlé Health Science Sign Development Agreement
Codexis, Inc. and Nestlé Health Science have signed an agreement to advance a lead candidate discovered through a Strategic Collaboration Agreement (SCA) into preclinical….
Mereo BioPharma & Oncologie Enter Global Licensing Agreement
Mereo BioPharma Group plc and Oncologie, Inc. recently announced a global license agreement for the development and commercialization of navicixizumab, an anti-DLL4/VEGF bispecific antibody currently…
CytoDyn Files for Breakthrough Therapy Designation With the FDA
CytoDyn Inc. recently announced it has filed for Breakthrough Therapy designation (BTD) with the US FDA for the use of leronlimab as an adjuvant therapy…
Adaptimmune Announces SPEAR T-cell Platform Delivers Initial Responses in Four Solid Tumor Indications
Adaptimmune Therapeutics plc recently announced two confirmed Partial Responses (PRs) – one in a patient with liver cancer and one in a patient with melanoma. There…
Novavax Granted Fast-Track Designation
Novavax, Inc. recently announced the US FDA has granted Fast-Track Designation for NanoFlu, its recombinant quadrivalent seasonal influenza vaccine candidate, adjuvanted with Matrix-M, in adults…
PERSONALIZED MEDICINE - Personalizing Cancer Immunotherapy: Trends in Biomarker Discovery
Emile Youssef, MD, PhD, says due to tumor heterogeneity, the plasticity and diversity of cancer cells, and a multitude of other factors, biomarker development is a challenge, and thus explores four trends in cancer biomarker discovery.
DEVELOPMENT TIMELINES - Drug Development Times, What it Takes - Part 1
Josef Bossart, PhD, explains how accurately estimating a product’s clinical development timelines is more than simply adding up the individual timelines, almost always optimistic. You need to “know” the numbers, not just calculate them.
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).