Bio Platforms
First Wave BioPharma Announces Exclusive Global License Agreement for Capeserod From Sanofi
First Wave to develop and repurpose Capeserod for gastrointestinal (GI) indications based on AI-empowered analyses, expanding the company’s GI-focused pipeline….
Anavex’s Phase 2b/3 Trial of Blarcamesine in Patients With Alzheimer’s Disease Shows Robust Clinical Efficacy & Slows Neurodegeneration
Anavex Life Sciences Corp. recently announced that a follow-on analysis of the landmark Phase 2b/3 study to treat early Alzheimer’s disease with the investigational drug…
Rencofilstat Produces Positive Outcomes in Translational Study on Idiopathic Pulmonary Fibrosis
Hepion Pharmaceuticals, Inc. recently announced positive results from a translational research study in which Hepion’s lead drug candidate, rencofilstat, was administered to diseased human lung tissue from….
Curia Supports Replicate Bioscience’s Phase 1 Clinical Trial of Novel Next-Generation RNA Vaccine
Supply of drug substance manufactured under cGMP supports first-in-human clinical study for RBI-4000 novel self-replicating RNA (srRNA)….
KBI Biopharma Expands Global Portfolio With Launch of SUREmAb for Faster Monoclonal Antibody Development & Manufacturing
KBI Biopharma, Inc., a JSR Life Sciences company, recently announced the launch of SUREmAb, an offering built on the robustness of KBI’s SUREtechnology Platform, that…
Salipro Biotech Enters Research Collaboration With Sumitomo Pharma to Advance Drug Discovery Program
Salipro Biotech AB recently announced that it has entered into a research collaboration with Sumitomo Pharma Co., Ltd. to reveal the mechanism of action and…
Lisata Therapeutics Announces First Patient Treated in BOLSTER Trial Novel Tumor-Targeting & Penetrating Peptide in Patients With Advanced Solid Tumors
Lisata Therapeutics, Inc. recently announced treatment of the first patient in the head and neck squamous cell carcinoma cohort of the BOLSTER trial, by Dr.…
Black Diamond Therapeutics Announces First Patients Dosed in Phase 1 Clinical Trial Expansion Cohorts Evaluating BDTX-1535 in Patients With Intrinsic Driver & Acquired Resistance EGFR Mutation Positive Non-Small Cell Lung Cancer
Black Diamond Therapeutics, Inc. recently announced the first patients dosed in mutation matched expansion cohorts of non-small cell lung cancer (NSCLC) in the ongoing Phase…
Immatics & Moderna Announce Strategic Multi-Platform Collaboration to Develop Innovative Oncology Therapeutics
Immatics, NV, and Moderna, Inc. recently announced a strategic research and development collaboration to pioneer novel and transformative therapies for cancer patients with high unmet….
MBX Biosciences Announces Positive Phase 1 Multiple Ascending Dose Data for Long-Acting Parathyroid Hormone Peptide Prodrug in Healthy Adults
MBX Biosciences, Inc. recently announced positive results from the multiple ascending dose (MAD) portion of the Phase 1 trial of MBX 2109, the company’s long-acting parathyroid…
Shape(TX) Collaborates With Otsuka to Develop Novel AAV Gene Therapies for Ocular Diseases
Shape(TX) and Otsuka Pharmaceutical Co., Ltd. recently announced a multi-target collaboration to develop intravitreally delivered adeno-associated viruses (AAVs) for ocular….
Valneva & Pfizer Report Positive Pediatric & Adolescent Phase 2 Booster Results for Lyme Disease Vaccine Candidate
Valneva SE and Pfizer Inc. recently announced positive pediatric and adolescent immunogenicity and safety data for their Lyme disease vaccine candidate, VLA15, when given….
Myricx Enters Antibody License Agreement With Biocytogen
Myricx Bio recently announced an antibody license agreement with Biocytogen Pharmaceuticals (Beijing) Co., Ltd, a global biotech company focusing on the….
Artelo Biosciences Completes Positive Pre-IND Meeting With FDA
Artelo Biosciences, Inc. recently announced it has completed a pre-IND meeting with the US FDA regarding the manufacturing, preclinical, and clinical development plan for ART26.12.…
Antion Announces Granting of Key Technology Patent in the United States
Novel patent solidifies Antion’s position as an industry leader in multiplex engineering of cell and gene therapies….
Eledon Pharmaceuticals Announces Publication of Data Showing Treatment With Tegoprubart Promotes Kidney & Islet Allograft Survival & Function in Nonhuman Primates
Eledon Pharmaceuticals, Inc. recently announced the publication of a study evaluating tegoprubart as an immunomodulatory monotherapy in nonhuman primate kidney and islet allotransplants. The study,…
Lisata Therapeutics Announces Outcome of Interim Futility Analysis for its Phase 2b ASCEND Trial in Metastatic Pancreatic Ductal Adenocarcinoma
Lisata Therapeutics, Inc. recently announced a positive outcome of the prespecified interim futility analysis for the ASCEND trial, a Phase 2b study evaluating LSTA1, Lisata’s…
Recipharm Partners With Ahead Therapeutics to Develop Rare Autoimmune Disease Therapy
Recipharm is partnering with Ahead Therapeutics to develop a ground-breaking treatment for a rare autoimmune disease, myasthenia gravis. Ahead Therapeutics is a Spanish start-up biotech…
CELL & GENE THERAPY - Cell & Gene Therapy’s Everest – The Challenges & Opportunities That Will Shape Success
Samir Acharya, PhD, Rajiv Vaidya, PhD, Laura Kerepesi, PhD, and Cyrill Kellerhals, MBA, provide their unique insights as they explore the challenges cell and gene therapy developers and manufacturers are currently facing, those they can expect to see in the future, and more critically, how to overcome them.
THERAPEUTIC FOCUS - Effect of NE3107 on the Pharmacokinetics Profile of Carbidopa/Levodopa in Patients With Parkinson’s Disease
Joseph M. Palumbo, MD, says addressing Parkinson’s via the inflammatory pathway offers a unique perspective that was virtually unheard of only 10 years ago.
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).