Bio Platforms
Synairgen Announces Dosing Has Commenced With Its Inhaled Interferon Beta Product in US Government-Funded NIH ACTIV-2 Trial in COVID-19 Outpatients
Synairgen plc recently announced that dosing has begun in the inhaled interferon beta formulation (SNG001) sub-study of the ACTIV-2 Phase 2/3 trial evaluating patients with…
Enlivex Announces Issuance of New US Patent Covering Methods of Treating Gout, Inflammatory Bowel Disease, Crohn’s Disease, Ulcerative Colitis & GvHD Using Allocetra Immunotherapy
Enlivex Therapeutics Ltd. recently announced the US Patent and Trademark Office has issued US Patent No. 10,927,343 covering Allocetra, the company’s immunotherapy product candidate. The…
Samsung Biologics, National OncoVenture & Eutilex Obtains IND Approval From FDA
Samsung Biologics recently announced another successful Investigational New Drug (IND) clearance from the FDA for its client to begin clinical trials on a cancer immunotherapy, furthering the….
Maravai LifeSciences Expands Its Intellectual Property Portfolio
Maravai LifeSciences, Inc. recently announced the United States Patent and Trademark Office has issued a new patent, United States Patent No. 10,913,768, to the company’s TriLink Biotechnologies subsidiary…..
Celon Pharma Announces Successful Completion of Phase 1A Trial of CPL’116, JAK-ROCK Inhibitor
Celon Pharma S.A. recently announced the successful completion of its Phase 1A trial of its JAK/ROCK kinases dual inhibitor, CPL’116. CPL’116 was administered orally in….
Cue Biopharma Initiates Patient Dosing in Phase 1 Study of CUE-101 in Combination with KEYTRUDAr as First-line Treatment for HPV+ Recurrent/Metastatic Head & Neck Cancer
Cue Biopharma, Inc. recently announced the first patient was dosed in a Phase 1 dose escalation clinical trial of CUE-101 in combination with Merck's anti-PD-1…
OliX Pharmaceuticals Launches New Subsidiary to Develop mRNA Vaccines & Therapeutics
OliX Pharmaceuticals, Inc. recently announced the establishment of mCureX Therapeutics, Inc., a subsidiary focused on the research and development of messenger RNA (mRNA) vaccines and…
uniQure Announces Positive Recommendation From Data Safety Monitoring Board of Phase 1/2 Clinical Trial
uniQure N.V. recently announced that the independent Data Safety Monitoring Board (DSMB) overseeing the Phase 1/2 clinical trial of AMT-130 for the treatment of Huntington’s…
Trizell Signs Development & Manufacturing Agreement With Catalent for Macrophage-Based Advanced Cell Therapy
Catalent recently announced it has signed an agreement with Trizell GmbH, to support the manufacturing of Trizell’s Phase 1 cell therapy for the treatment of…
ProPerma™ Oral Formulation Technology
ProPerma™ uniquely improves both permeability and bioavailability of BCS III and BCS IV compounds by deploying an innovative and bespoke combination of GRAS and novel enhancing excipients…
Theratechnologies’ Lead Peptide Drug Conjugate TH1902 Receives FDA Fast-Track Designation
Theratechnologies Inc. recently announced the US FDA has granted fast-track designation to TH1902 as a single agent for the treatment of patients with sortilin positive…
Decibel & Catalent Sign Development & Manufacturing Agreement for Dual-Vector Gene Therapy for the Treatment of Congenital Hearing
Catalent and Decibel Therapeutics recently announced they have signed an agreement for Catalent to provide Decibel with process and analytical development, and CGMP clinical manufacturing…
Biogen Announces FDA Approval of PLEGRIDY (Peginterferon Beta-1a) Intramuscular Administration for Multiple Sclerosis
Biogen Inc. recently announced the US FDA has approved a new intramuscular (IM) injection route of administration for PLEGRIDY (peginterferon beta-1a) for the treatment of….
Beroni Acquires Majority Interest in Anti-Cancer Drug Development Company
Beroni Group recently announced it has completed its majority acquisition of the Sydney-based anti-cancer drug development company, PENAO Pty Ltd. As a result of this acquisition….
Albumedix Collaborates With CGT Catapult to Advance Cell & Gene Therapies
Albumedix Ltd. recently announced it has entered into a collaboration agreement with the Cell and Gene Therapy Catapult (CGT Catapult) to investigate the use of Albumedix´ proprietary albumin-based solutions for….
X-Chem Enters Multitarget Oncology Discovery Research Collaboration & License Agreement With Genentech
X-Chem, Inc. recently announced it has entered into a research collaboration and license agreement with Genentech, a member of the Roche Group. The goal of…
GSK & CureVac to Develop Next-Generation mRNA COVID-19 Vaccines
GlaxoSmithKline plc and CureVac N.V. recently announced a new €150-million collaboration, building on their existing relationship, to jointly develop next-generation mRNA vaccines for COVID-19 with…
Venthera Doses First Patient With Topical Therapy Targeting Genetic Drivers of Rare Vascular Anomalies
BridgeBio Pharma, Inc. and affiliate Venthera recently announced the first subject has been dosed in its Phase 1/2 clinical trial of BBP-681 (also known as…
Pacira BioSciences Announces Equity Investment in GeneQuine Biotherapeutics
Pacira BioSciences, Inc. recently announced that the company will lead an equity investment in GeneQuine Biotherapeutics GmbH…..
Relief Therapeutics & Acer Therapeutics Sign Option Agreement for Exclusivity to Negotiate a Collaboration & License Agreement
Relief Therapeutics Holding AG and Acer Therapeutics Inc. recently announced the companies have signed an Option Agreement providing exclusivity for the right to negotiate a potential collaboration and license….
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).