Bio Platforms
Aptamer Group Partners With BaseCure Therapeutics to Develop New Targeted Gene Therapies
Aptamer Group plc recently announced a new partnership with BaseCure Therapeutics for the development of Optimer-targeted gene therapies. The ability to precisely target gene therapies,…
First Wave BioPharma Announces Initiation of Phase 2 Trial Investigating Enhanced Adrulipase Formulation in Exocrine Pancreatic Insufficiency in Patients With Cystic Fibrosis
First Wave BioPharma, Inc. recently announced it will initiate its planned Phase 2 clinical trial of an enhanced enteric microgranule delivery formulation for adrulipase for…
Stemline Therapeutics Receives Approval for First & Only Treatment Specifically Indicated for Patients with ESR1 Mutations in ER+, HER2- Advanced or Metastatic Breast Cancer
The Menarini Group recently announced the US FDA has approved ORSERDU for the treatment of postmenopausal women or adult men, with ER+, HER2-, ESR1-mutated advanced…
Anti-Bb Antibody Receives US FDA Clearance to Start Clinical Trial in Naïve aHUS Patients
NovelMed recently announced the US FDA cleared the company’s investigational drug NM8074 to initiate an efficacy trial in patients with aHUS in coming months. NovelMed…
BioAegis Therapeutics Announces FDA Clearance of IND for its Inflammation Regulator Protein for the Treatment of ARDS
BioAegis Therapeutics, Inc. recently announced the US FDA has approved its Investigational New Drug (IND) application for recombinant plasma gelsolin to proceed for the….
INmune Bio, Inc. Announces Pre-Clinical Data That Support a Pioneering Approach to Treating Duchenne Muscular Dystrophy
INmune Bio, Inc. in collaboration with Professor Armando Villalta, PhD, of University California, Irvine School of Medicine, has shown targeting soluble TNF (sTNF) using a…
Ocuphire Announces Topline Results From Phase 2 Trial of Oral APX3330 in Diabetic Retinopathy & Plans for End-of-Phase 2 Meeting With FDA
Ocuphire Pharma, Inc. recently announced topline efficacy and safety results from its ZETA-1 Phase 2 trial evaluating oral APX3330 for the treatment of diabetic….
AC Immune’s Anti-Amyloid Beta Vaccine for Alzheimer’s Shows Positive Initial Interim Safety & Immunogenicity in Phase 1b/2 Trial
AC Immune SA recently announced the first interim safety, tolerability, and immunogenicity findings from the Phase 1b/2 ABATE trial of its anti-amyloid-beta (Abeta) vaccine ACI-24.060…
Inhibikase Therapeutics Announces FDA has Lifted the Full Clinical Hold on IkT-148009 in Parkinson’s Disease
Inhibikase Therapeutics, Inc. recently announced the US FDA has lifted the full Clinical Hold on IkT-148009 in Parkinson’s disease (PD). “We are grateful for the…
Eloxx Pharmaceuticals Announces First Patients Enrolled in Phase 2 Clinical Study Evaluating Treatment for Alport Syndrome
Eloxx Pharmaceuticals, Inc. recently announced the first patients have now been enrolled in its Phase 2 study of ELX-02 for the treatment of Alport syndrome…
Alvotech & Bioventure Announce Approval of AVT02 (Adalimumab) as Simlandi in Saudi Arabia
AVT02 as Simlandi is the first biosimilar approved under the strategic partnership between Alvotech and Bioventure….
SOTIO Doses First Patient in AURELIO-05 Phase 2 Trial of Nanrilkefusp Alfa in Combination With Cetuximab
SOTIO Biotech recently announced the first patient was dosed in its Phase 2 AURELIO-05 combination trial of nanrilkefusp alfa (previously known as SOT101), an IL-15 superagonist, and….
Evaxion Receives FDA Fast-Track Designation for Personalized Cancer Immunotherapy
Evaxion Biotech A/S recently announced the US FDA has granted fast track designation for the company’s personalized cancer therapy, EVX-01, in combination….
Strata Oncology Announces Addition of Gilead as Strata PATH Trial Collaborator
Strata Oncology, Inc. recently announced Gilead Sciences, Inc. has agreed to collaborate on the Strata Precision Indications for Approved THerapies (Strata PATH) trial by providing…
BriaCell Announces Positive End of Phase 2 Meeting With the FDA for Bria-IMT Combination in Advanced Metastatic Breast Cancer
BriaCell Therapeutics Corp. is pleased to announce that it has received agreement and positive feedback from its End of Phase 2 meeting with the FDA…
Avidity Biosciences Granted FDA Fast Track Designation for the Treatment of Facioscapulohumeral Muscular Dystrophy
Avidity Biosciences, Inc. recently announced the US FDA has granted Fast Track designation to AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). FSHD…
Teon Therapeutics Announces Clinical Trial Collaboration With Merck to Evaluate Novel Oral Immune Response Modifier in Combination with KEYTRUDA
Teon Therapeutics (Teon) recently announced it has entered into the clinical trial collaboration agreement with Merck for the combination arm of Teon’s ongoing, two-armed, open-label, dose….
Ingenza Collaborates With Amplifica to Advance Novel Alopecia Treatments
Industrial biotech specialist Ingenza recently announce it is collaborating with clinical-stage biopharmaceutical company Amplifica Holdings Group, Inc. to develop an innovative treatment to address androgenic…
Needle-Free Patch Technology for mRNA Vaccines Aims to End Need for Frozen Storage & Improve Access
CEPI, the Coalition for Epidemic Preparedness Innovations, and Vaxxas have recently signed a partnership agreement to advance the development of Vaxxas’ needle-free vaccine-patch delivery technology in a project….
Kinarus Therapeutics Reports Preclinical Data in Lung Fibrosis & Discloses Clinical Development Plan
Kinarus Therapeutics AG recently announced preclinical data supporting the potential effectiveness of its lead clinical candidate, KIN001, as an oral treatment for idiopathic pulmonary fibrosis…
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).