Bio Platforms
Clene Announces Publication of Phase 2 CNM-Au8 Clinical Data for the Treatment of ALS
Clene Inc. and its wholly owned subsidiary Clene Nanomedicine, Inc. recently announced The Lancet’s journal eClinicalMedicine has published combined detailed analyses of the Phase 2…
Ocean Biomedical Announces US Patent Issued for Bispecific Cancer Immunotherapy Treatment With Demonstrated Killing of Tumor Cells in Lung Cancer, Brain Cancer (Glioblastoma) & Metastatic Melanoma
Patent broadens protection around Ocean Biomedical’s first-in-class anti-CHi3L1 antibody to include new bispecific approaches targeting immune checkpoints, with potential application in….
XOMA Acquires Royalty & Milestone Economics to Phase 3 First-In-Class Orphan Disease Asset for Niemann-Pick Disease Type C (NPC) & Phase 2 Oncology Asset
XOMA Corporation recently announced it has acquired the royalty and milestone rights associated with two assets from LadRx Corporation: arimoclomol, an oral therapeutic for Niemann-Pick…
ASLAN Pharmaceuticals Enters Strategic Licensing Deal With Zenyaku Kogyo for the Development & Commercialization of Eblasakimab
ASLAN Pharmaceuticals and Zenyaku Kogyo Co., Ltd., a subsidiary of privately held Zenyaku Holdings Co., Ltd. recently announced a strategic licensing agreement granting Zenyaku exclusive…
Lilly to Acquire DICE Therapeutics to Advance Innovation in Immunology
Eli Lilly and Company and DICE Therapeutics, Inc. recently announced a definitive agreement for Lilly to acquire DICE, a biopharmaceutical company that leverages its proprietary….
SCYNEXIS Announces Achievement of First Development Milestone of $25 Million Under Exclusive License Agreement With GSK
SCYNEXIS, Inc. recently announced the achievement of a $25-million performance-based development milestone under its exclusive license agreement with GSK for….
Silo Pharma’s SPU-21 Treatment for Autoimmune Disease Awarded US Patent
Silo Pharma, Inc. recently announced the US Patent and Trademark Office (USPTO) has issued US Patent No. 16/825,371 titled Peptide-Targeted Liposomal Delivery for Treatment, Diagnosis,…
NodThera Announces Positive First-in-Human Data for Two Brain Penetrant NLRP3 Inflammasome Inhibitors
NodThera recently announced positive data from first-in-human studies of its lead therapeutic candidates, NT-0249 and NT-0796, and provides an update on the company’s priority clinical…
Icosavax Initiates Phase 2 Trial of IVX-A12 Against RSV & hMPV in Older Adults
Icosavax, Inc. recently announced the initiation of a Phase 2 clinical trial of IVX-A12, a combination bivalent respiratory syncytial virus (RSV) and human metapneumovirus (hMPV)…
Curia Expands Cell Line Development Offering With CHOZN Platform
Curia signs license agreement with MilliporeSigma to support production of proteins and antibodies….
ANAVEX2-73 (blarcamesine) Receives Compassionate Use Authorization for Pediatric Patients With Rett Syndrome
Anavex Life Sciences Corp. recently announced the first patients with Rett syndrome from the ANAVEX2-73-RS-003 study (EXCELLENCE) are able to continue treatment with ANAVEX2-73 (blarcamesine)…
Helocyte Announces $3.22-Million Grant from the National Cancer Institute for Triplex Phase 2 Clinical Trial Program
Multicenter randomized placebo-controlled trials will evaluate the ability of the cytomegalovirus (CMV) vaccine to enhance protective virus-specific immunity in stem cell donors to reduce the risk of CMV events in high-risk transplant recipients….
Ventyx Biosciences Announces Initiation of Dosing in Phase 1 Trial of Novel CNS-Penetrant NLRP3 Inhibitor
Ventyx Biosciences, Inc. recently announced the first subject has been dosed in a Phase 1 trial of VTX3232, a novel central nervous system (CNS)-penetrant NLRP3…
HCW Biologics Granted Third Fundamental Patent for Methods of Treating Age-Related Disorders
HCW Biologics Inc. was granted US Patent No. 11,672,826 on June 13, 2023, which contains methods of use claims directed to administering the company’s clinical-stage…
PDS Biotechnology Announces Achievement of Efficacy Threshold in Stage 2 of the VERSATILE-002 Trial Evaluating PDS0101 & KEYTRUDA in Head & Neck Cancer
PDS Biotechnology Corporation recently announced it achieved the threshold for efficacy as per investigator assessment in Stage 2 of the VERSATILE-002 (NCT04260126) Phase 2 clinical…
PaxMedica Submits Clinical Trial Request in Autism Spectrum Disorder
PaxMedica, Inc. recently announced it has submitted a request to the South African Health Products Regulatory Authority (SAHPRA), seeking approval to conduct a double-blind, placebo-controlled…
Valo Therapeutics & Texcell Announce Research Collaboration to Evaluate Immune Responses to PeptiCRAd-1 in Phase 1 Clinical Study
Valo Therapeutics Oy recently announced a collaboration with global contract research organization, Texcell, to investigate immune responses to PeptiCRAd-1 in its Phase 1 clinical study.…
Salipro Biotech Expands Global Intellectual Property Portfolio With Granted Patent in Japan
Salipro Biotech AB recently announce the issuance of a new patent in Japan for its Salipro platform technology. The patent, titled Saposin lipoprotein particles and…
Mustang Bio Announces Updated Results From Waldenstrom Macroglobulinemia Cohort of Ongoing Phase 1/2 Clinical Trial of MB-106, CD20-Targeted Autologous CAR T Therapy
Mustang Bio, Inc. recently announced that updated data from the ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T cell therapy, show…
Quell Therapeutics Signs Collaboration, Exclusive Option & License Agreement With AstraZeneca to Develop, Manufacture & Commercialize Engineered Treg Cell Therapies for Autoimmune Diseases
Collaboration brings together Quell’s proprietary multi-modular T-regulatory (Treg) cell engineering and manufacturing expertise with AstraZeneca’s proven development and commercial capabilities as well as its deep therapeutic area knowledge….
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).