Bio Platforms
Wave Life Sciences Announces Submission of First Clinical Trial Application for WVE-007 (siRNA Targeting INHBE) in Obesity
Wave Life Sciences Ltd. recently announced the submission of its first clinical trial application (CTA) for WVE-007 in obesity. WVE-007 is an investigational GalNAc-conjugated small…
Ikena Oncology & Inmagene Biopharmaceuticals Announce Agreement for Merger & Private Placement
Ikena Oncology, Inc. and Inmagene Biopharmaceuticals recently announced they have entered into a definitive merger agreement. In connection with the merger, Ikena has entered into…
GRI Bio Announces European Patent Office Issued a Decision to Grant Notice for Patent Covering GRI-0803 & its Library of 500+ Proprietary Compounds
GRI Bio, Inc. recently announced the European Patent Office (EPO) has issued a decision to grant notice for patent application number 19,166,502 titled, Oxygenated Amino-…
Apollomics Announces Top-line Results for Phase 3 Trial of Uproleselan in Patients With Relapsed or Refractory Acute Myeloid Leukemia
Apollomics Inc. recently announced the results from its Phase 3 bridging trial of uproleselan in China in patients with relapsed or refractory acute myeloid leukemia.…
Shuttle Pharma Enters Sponsored Research Agreement With the University of California, San Francisco to Advance PSMA Development Program
Shuttle Pharmaceuticals Holdings, Inc. recently announced a sponsored research agreement with the University of California, San Francisco (UCSF) to advance pre-clinical development of Shuttle Diagnostics,…
Assembly Biosciences Announces $30.1 Million Investment & Accelerated Funding From Gilead
Assembly Biosciences, Inc. recently announced an equity investment of $20.1 million by Gilead Sciences, Inc. to purchase additional Assembly Bio common stock and an amendment…
IMUNON Announces Positive CMC Meeting With FDA for Advanced Ovarian Cancer Treatment
IMUNON, Inc. recently announced the positive outcome of a Type C Chemistry, Manufacturing, and Controls (CMC) meeting with the US FDA regarding production of IMNN-001…
Vandria Announces First Subjects Dosed in First-in-Human Phase 1 Trial of VNA-318 Brain-Penetrant Mitophagy Inducer
Vandria SA recently announced the first subjects have been dosed in its first-in-human clinical trial of its lead Central Nervous System (CNS) compound VNA-318. Readout…
Coherus to Present Final Phase 2 Casdozokitug Combination Data in Patients with Metastatic Hepatocellular Carcinoma
Coherus BioSciences, Inc. recently announced an abstract highlighting final clinical and biomarker data from its Phase 2 clinical trial evaluating casdozokitug (casdozo), a selective and…
Channel Therapeutics Announces Positive Efficacy Data For a Depot Formulation of a NaV1.7 Inhibitor in a Preclinical In Vivo Nerve Block Model
Channel Therapeutics Corporation recently announced it achieved its endpoints in two pre-clinical in vivo models of its nerve block formulations for acute pain, showing material…
EyeDNA Therapeutics Receives Rare Pediatric Disease Designation From FDA for its Investigational Gene Therapy for Patients With Retinal Dystrophy Due to PDE6b Gene Mutations
eyeDNA Therapeutics recently announced that it has been granted a Rare Pediatric Disease Designation (RPDD) by the US FDA for HORA-PDE6b, its novel gene therapy…
Silexion Therapeutics Collaborates With Evonik on Advanced siRNA Formulation Development
Silexion Therapeutics Corp. recently announced its ongoing collaboration with Evonik, a global leader in specialty chemicals, for the development of an advanced siRNA formulation aimed…
GlycoNex Announces First Patient Dosed in Phase 3 Clinical Trial for Denosumab Biosimilar
GlycoNex, Inc. recently announced the first patient has been dosed in the Phase 3 clinical trial of its denosumab biosimilar, SPD8. This marks the initiation…
Immutep Announces Initial Safety Data From First-in-Human Phase 1 Trial Evaluating IMP761
Immutep Limited recently announced favorable initial safety data from the placebo-controlled, double-blind first-in-human Phase 1 study evaluating IMP761. Through the first three of five single…
Palvella Therapeutics Announces Closing of Merger With Pieris Pharmaceuticals & Concurrent Private Placement of $78.9 Million
Palvella Therapeutics, Inc. recently announced the completion of its previously announced merger with Pieris Pharmaceuticals, Inc. (Pieris). The combined company will operate under the name…
Atsena Therapeutics Announces Dosing Completed in Part A of Phase 1/2 Clinical Trial Evaluating Gene Therapy to Treat X-linked Retinoschisis
Atsena Therapeutics recently announced dosing has been completed in Part A of the LIGHTHOUSE study, a Phase 1/2 clinical trial evaluating subretinal injection of ATSN-201…
HUTCHMED to Receive Milestone Payment From Takeda
HUTCHMED recently announced it will receive a $10-million milestone payment by its partner Takeda. Takeda received a national reimbursement recommendation for FRUZAQLA (fruquintinib) for patients…
VelaLabs Announces Strategic Partnership to Support Biological Drugs Development
VelaLabs GmbH has signed a Memorandum of Understanding (MoU) with EarlyHealth Group to establish comprehensive solutions for the development of advanced biological drugs. Under the…
Immuneering Granted FDA Fast Track Designation for IMM-1-104 in Advanced Melanoma
Immuneering Corporation recently announced the US FDA has granted Fast Track designation for its lead clinical-stage program, IMM-1-104, as a treatment for patients with unresectable…
PDC*line Pharma Presents Primary Clinical Results From Phase I/II Trial on PDC*lung01 Cancer Vaccine
PDC*line Pharma recently announced the primary clinical results from the last cohort of patients in its Phase I/II clinical trial with PDC*lung01 (PDC-LUNG-101, NCT03970746), in…
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).