Bio Platforms
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….
Agenus Enters Clinical Collaboration With Nelum for Zalifrelimab Combination
Agenus Inc. recently announced that it has entered into a clinical collaboration with Nelum Corp. to evaluate the safety and efficacy of zalifrelimab, Agenus’ anti-CTLA-4 antibody….
Vaxart Announces Additional Preclinical COVID-19 Oral Vaccine Data & Publication
Vaxart, Inc. recently announced additional results from its SARS-CoV-2 Hamster Challenge Study, as well as a peer-reviewed publication in Nature Medicine resulting from a collaboration…
Antios Therapeutics Completes Phase 1b Clinical Trial of Active Site Polymerase Inhibitor Nucleotide
Antios Therapeutics, Inc. recently announced it has completed the Phase 1b clinical trial of ATI-2173, an Active Site Polymerase Inhibitor Nucleotide (ASPIN), in patients with…
ViGeneron Announces Research Collaboration With Daiichi Sankyo to Evaluate Novel Ophthalmic Gene Therapy
ViGeneron GmbH recently announced a research collaboration to utilize its novel engineered adeno-associated virus (vgAAV) vectors with Daiichi Sankyo Company, Limited for delivering….
Clene Nanomedicine Receives Patent Notice of Allowance for using Gold Nanocrystals for Treating MS
Clene Inc. recently announced its wholly owned subsidiary Clene Nanomedicine, Inc., a clinical-stage biopharmaceutical company, was issued a Notice of Allowance from the US Patent and Trademark Office (USPTO) for its invention for….
Cognate BioServices & Its Gene Therapy Division Cobra Biologics Announce Major Expansion
Cobra Biologics, the gene therapy division of the Cognate BioServices’ group, recently announced a multi-phase augmentation of its plasmid DNA services as a continuation of…
Cocrystal Pharma Announces Further Development of Influenza A/B Antiviral Compounds by Merck Under Exclusive Worldwide License & Collaboration Agreement
Cocrystal Pharma, Inc. recently announced it has completed all research obligations under the Merck exclusive worldwide license and collaboration agreement, and that Merck is now…
Quantum Genomics Launches Phase 3 Pivotal REFRESH Study
Quantum Genomics recently announced the launch of its Phase 3 REFRESH study in difficult-to-treat or resistant hypertension. This new study is part of firibastat’s Phase…
Gritstone Advances Second-Generation COVID-19 Vaccine
Gritstone Oncology, Inc. recently announced it is advancing development of a second-generation vaccine against SARS-CoV-2, the virus that causes COVID-19, with potential for both prolonged…
Caladrius Biosciences Treats First Patient in the Phase 2b FREEDOM Trial
Caladrius Biosciences, Inc. recently announced it has treated the first patient in its Phase 2b FREEDOM trial of CLBS16 as a therapy for coronary microvascular…
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).