Bio Platforms
Longeveron Selects Clinical Research Organization for Phase 2 Alzheimer’s Disease Trial
Longeveron Inc. recently announced the selection of Biorasi LLC as its clinical research organization (CRO) to help conduct its Phase 2 clinical trial evaluating Lomecel-B…
XOMA Earns $35-Million Milestone Payment as Anti-TGFβ Antibody Enters Phase 3 Clinical Study in Metastatic Pancreatic Cancer
XOMA Corporation recently announced NIS793, an anti-TGFβ monoclonal antibody licensed from the company, has advanced to the Phase 3 development stage, triggering a $35-million milestone…
Zealand Pharma Announces First Subject Dosed in Phase 1 Trial of Amylin Analogue ZP8396 for the Treatment of Obesity
Zealand Pharma A/S recently announced the first patient has been dosed in a Phase 1 clinical trial evaluating the amylin analogue, ZP8396 as a potential…
Allena Pharmaceuticals Receives Fast Track Designation From FDA for the Development of ALLN-346 for Chronic Treatment of Hyperuricemia in Patients With Gout & Advanced Chronic Kidney Disease
Allena Pharmaceuticals, Inc. recently announced its orally administered, urate-degrading enzyme, ALLN-346, has received Fast Track designation from the US FDA. ALLN-346 is in Phase 2…
Stingthera Announces Clinical Collaboration With Merck to Evaluate SNX281 in Combination With KEYTRUDA in Certain Patients With Advanced Solid Tumors & Lymphoma
Stingthera, Inc. recently announced it has entered into a clinical trial collaboration agreement with Merck (known as MSD outside the US and Canada) to evaluate the combination of SNX281, Stingthera’s investigational….
uniQure Announces Latest Positive Recommendation From Data Safety Monitoring Board in Phase 1/2 Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
uniQure N.V. recently announced the positive recommendation by the independent Data Safety Monitoring Board (DSMB) following a review of safety data from the four patient…
Evox Therapeutics Expands Its Exosome Patent Portfolio
Evox Therapeutics Ltd recently announced it has been granted a new European patent, EP3706796, to add to its growing patent portfolio. This patent covers exosomes…
Dunad Therapeutics Enters Strategic Collaboration With Novartis to Develop Next-Generation Oral Targeted Protein Degrader Therapies
Dunad Therapeutics recently announced it has entered a strategic collaboration and license agreement with Novartis to generate orally bioavailable covalent and protein degrading small molecule drugs…..
Two New Studies Demonstrate In Vitro Bioactivity Potential of Micronized Dehydrated Human Amnion Chorion Membrane to Prevent Pathological Processes Underlying Osteoarthritis & Tendinopathy
MiMedx Group, Inc. recently announced two peer-reviewed studies investigating the use of micronized dehydrated human amnion chorion membrane (mdHACM) for the treatment of debilitating conditions…
Novavax & Serum Institute of India Receive Emergency Use Authorization for COVID-19 Vaccine in Indonesia
Novavax, Inc. and Serum Institute of India Pvt. Ltd. (SII) recently announced that the National Agency of Drug and Food Control of the Republic of…
Moderna Announces First Participant Dosed in Phase 3 Pivotal Registration Study of its mRNA Cytomegalovirus (CMV) Vaccine
Moderna, Inc. recently announced the first participant has been dosed in the Phase 3 pivotal registration study of mRNA-1647, the Company’s cytomegalovirus (CMV) vaccine candidate. This…
Ocugen, Inc. Announces Submission of IND to Initiate a Phase 3 Clinical Trial Evaluating COVID-19 Vaccine Candidate
Ocugen, Inc. recently announced that it has submitted an Investigational New Drug application (IND) with the US FDA to evaluate the COVID-19 vaccine….
Evaxion Biotech Announces Clinical Collaboration to Evaluate Lead Product Candidate with KEYTRUDA (pembrolizumab) in Patients With Melanoma
Evaxion Biotech A/S recently announced it has entered into a clinical trial collaboration and supply agreement with subsidiaries of Merck & Co., Inc., Kenilworth, NJ,…
TCR² Therapeutics Announces Clinical Trial Collaboration Agreement With Bristol Myers Squibb
TCR2 Therapeutics Inc. recently announced a clinical trial collaboration agreement with Bristol Myers Squibb to evaluate gavo-cel in combination with Opdivo (nivolumab) and Yervoy (ipilimumab)…
Cybrexa Therapeutics Announces Data Demonstrating Early Signals of Efficacy & Powerful Potential of Antigen-Independent Tumor Targeting
Cybrexa Therapeutics recently announced favorable early Phase 1 data for its lead therapeutic candidate, CBX-12 (alphalex-exatecan). The initial data show robust….
Krystal Biotech Announces Completion of the GEM-3 Pivotal Phase 3 Study Evaluating B-VEC for the Treatment of Dystrophic Epidermolysis Bullosa
Krystal Biotech, Inc. recently announced the last participant has completed the 26-week dosing period and 30-day safety follow up visit in the GEM-3 study, Krystal’s…
Catalent's Investment in Expanding its State-of-the-Art Gene Therapy Commercial Manufacturing Campus Increases to $360 Million
Catalent recently announced a $230-million expansion project to add three further commercial-scale viral vector manufacturing suites and associated support facilities and services at its gene…
Pfizer & BioNTech Announce Phase 3 Trial Data Showing High Efficacy of a Booster Dose of Their COVID-19 Vaccine
Pfizer Inc. and BioNTech SE recently announced topline results from a Phase 3 randomized, controlled trial evaluating the efficacy and safety of a 30-µg booster dose of…
Silence Therapeutics Provides mRNAi GOLD Platform & Pipeline Updates
Silence Therapeutics plc, a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (siRNA) therapeutics for the treatment of diseases with…
A New Study Using the SomaScan Platform Makes the Connection Between Genes & Proteins & Their Impact on Human Disease
In the largest proteogenomic study of its kind to date, an international research team led by scientists at the Berlin Institute of Health at Charité…
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).