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Altasciences At CPHI Americas 2025 pmjackson Thu, 07/03/2025 - 13:41 In the ever-changing drug development world, staying agile is essential to enabling more informed decisions, faster. This blog was originally published in July 2025. Why does that matter? Beyond the science, what’s exciting is how this technology opens doors.
Targeting GPCRs to fight inflammatory diseases GPCR-targeting drugs are well known therapies for a range of disease types, including cardio-metabolic, central nervous system and endocrinological disorders. GPCR-targeting therapies such as DT-9046 represent a compelling opportunity in that respect, with major clinical and commercial potential.
This stands in contrast to traditional small-molecule inhibitors, which typically aim to block a protein’s activity or binding. Bristol Myers Squibb’s platform includes a range of small-molecule degraders, which exploit the cell’s own disposal mechanisms — typically by sending the target protein to the proteasome for destruction.
Novartis Finalizes Acquisition of Regulus Therapeutics, Strengthening Its Renal Disease Portfolio with Promising ADPKD Therapy Novartis AG, a leading global pharmaceutical company, has officially completed its acquisition of Regulus Therapeutics Inc. , a biotechnology firm known for its expertise in microRNA-targeting therapies.
New approach methodologies data In April, the US Food and Drug Administration (FDA) announced a plan to replace animal testing in the development of monoclonal antibodies and other therapies with validated “human-relevant” methods, including AI-based computational models evaluating toxicity, cellular lines and organoid toxicity.
Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments. Navigating the AI revolution: a roadmap for pharma’s future.
The data were revealed during an oral presentation (S137) at the 2025 European Hematology Association (EHA) Congress in Vienna, reflecting ongoing progress in developing new treatment options for patients battling this aggressive hematologic malignancy.
Targeted drug delivery and therapeutic development MSI offers significant advantages for those developing smallmolecule therapeutics. 7 In immune therapies, MSI helps monitor immune-cell distribution and off-target effects, optimising therapeutic strategies and improving patient outcomes. For instance, Cheng et al.
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. Drugmakers are working to coopt this process with targeted therapies. You can unsubscribe at anytime.
1] [2] It is a non- opioid , small-molecule analgesic that works as a selective inhibitor of Na v 1.8 1] [2] It is a non- opioid , small-molecule analgesic that works as a selective inhibitor of Na v 1.8 The application received Breakthrough Therapy , Fast Track and Priority Review designations by the FDA.
The acceptance, which follows the CDE’s grant of Priority Review status in May, represents an accelerated regulatory pathway for what may become the first approved systemic therapy for TGCT in China. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational smallmolecule developed by Abbisko Therapeutics Co.,
Ervogastat CAS 2186700-33-2 Non-alcoholic Steatohepatitis (NASH) with Liver Fibrosis (FAST TRACK – U.S.) 2-[5-[(3-Ethoxy-2-pyridinyl)oxy]-3-pyridinyl]- N -[(3 S )-tetrahydro-3-furanyl]-5-pyrimidinecarboxamide ( S )-2-(5-((3-Ethoxypyridin-2-yl]oxy]pyridin-3-yl)- N -(tetrahydrofuran-3-yl)pyrimidine-5-carboxamide PF 06865571 BSOIY5AKQW 407.4
billion by 2025, growing at a CAGR of 34.2% from 2020 to 2025[1]. Oncology: Expanding Access to Life-Saving Treatments In the field of oncology, biosimilars have made significant inroads, particularly in supportive care and targeted therapies. The global biosimilars market is projected to reach $69.4
2] The US Food and Drug Administration (FDA) granted the application for crinecerfont fast track , breakthrough therapy , orphan drug , and priority review designations. [2] Retrieved 25 January 2025. New Drug Therapy Approvals 2024 (PDF). January 2025. Archived from the original on 21 January 2025. 1 October 2024.
Case Study: How Altasciences Overcame Pharmacological Challenges in a GLP-1 IND-Enabling Study pmjackson Thu, 04/24/2025 - 21:43 For emerging therapies with pronounced pharmacological effects, thoughtful study design is critical to ensuring reliable and interpretable data. This blog was originally published in April 2025.
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. It’ll also boost investments in U.S. drug production.
These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. Smallmolecule GLP1s?
Acoltremon AR-15512 CAS 68489-09-8 WeightAverage: 289.419 Monoisotopic: 289.204179113 Chemical FormulaC 18 H 27 NO 2 FDA 2025, 5/28/2025, To treat the signs and symptoms of dry eye disease Tryptyr WS-12 WS 12 (1R,2S,5R)-N-(4-methoxyphenyl)-5-methyl-2-(propan-2-yl)cyclohexane-1-carboxamide Fema No. 29 May 2025. 28 May 2025.
TCR 2 is developing novel T-cell therapies for solid tumors and hematological cancers. The company is using its pioneering FasTCAR and TruUCAR technology platforms to discover and develop breakthrough cell therapies. Its lead candidate is CLN-081, an oral smallmolecule designed to be a next-generation, irreversible EGFR inhibitor.
New Drug Therapy Approvals 2024 (PDF). January 2025. Archived from the original on 21 January 2025. Retrieved 21 January 2025. ^ “Novel Drug Approvals for 2024” U.S. 1] [2] Levacetylleucine is a modified version of the amino acid leucine. [1] 1] It is the L -form of acetylleucine. 25 September 2024.
SENS-401 has demonstrated highly encouraging efficacy in preclinical models and we are excited to progress this smallmolecule towards clinical trials to address this significant unmet medical need in cancer patients undergoing cisplatin treatment. About Sensorion.
www.sensorion-pharma.com.
Double-digit revenue growth through 2025; acquisition strengthens AstraZeneca’s broad-based revenue and the company will further globalise Alexion’s portfolio . Rare diseases is a high-growth therapy area with rapid innovation and significant unmet medical need. Dedicated rare disease unit to be headquartered in Boston.
In 2019, we introduced Vision 3×3, the company’s strategic roadmap through 2025, to build a leading biopharma company by achieving sustainable growth through multiple approaches. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two.
Incredible advances in machine learning-driven tools allow us to predict the 3D structure of previously unsolved proteins, unlocking the power of structure-based drug design for drugging these targets, and most recently, the ability to predict protein assemblies, which contain both the protein and putative smallmolecule binders.
2] [3] [4] A regulatory application for approval of the medication is expected to be submitted by 2025. [2] 19] As of 2016, aticaprant has reached phase II clinical trials as an augmentation to antidepressant therapy for treatment-resistant depression. [20] 2] Aticaprant is taken by mouth. [1] nM vs. 24.0 mg and 82% for 25 mg. [15]
AbbVie announced positive top-line results from the Phase 3 ADVANCE and MOTIVATE studies, which evaluated the efficacy and safety of Skyrizi (risankizumab) for induction therapy in adult patients with moderate to severe Crohn’s disease (CD). AbbVie and Frontier Medicines, Corp., AbbVie and Frontier Medicines, Corp.,
Their goal is to pinpoint novel targets that could be modulated by smallmolecules or biologics to restore healthy cardiac function or prevent further deterioration in at-risk patients. The research will build upon an established cardiomyocyte platform developed during the initial phase of the collaboration.
The new site will be built within the vicinity of an existing Fujifilm site, and is scheduled to begin operations in the spring of 2025. “We are uniquely placed to support our customer supply chain needs providing end to end services from small to large scale bulk drug substance production, through to fill-finish and final packing.”
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. Influenza A virus" [Micrograph]. Retrieved from Flickr. His research, writing offer clues.
27 key programs highlighted, including assets that could potentially contribute revenue by 2025 and others in the 2026-2028 time frame. Major revenue contributions through 2025 anticipated from Oncology, Vaccines, Rare Disease and Inflammation and Immunology.
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. We urge the Senate to find a way to address the pill penalty in reconciliation.
While many early-stage private VCs (and especially those participating in recent M&A) are feeling good going into 2025, public investors lamented the poor performance of public portfolios and indices. overall sentiment was bifurcated. Herculean Clinical Impact: What Cant Incretins Do?! While some of these potential competitors (e.g.,
Farrag, Yoshimasa Kawaguchi, Kazuo Nagasawa, Tasuku Honjo, Kenji Chamoto, and Motonari Uesugi Journal of the American Chemical Society 2025 DOI: 10.1021/jacs.4c17837 Combination therapies with smallmolecules have also been explored, but their clinical implementation has been met with significant challenges.
Altasciences Receives 2025 CDMO Leadership Award in SmallMolecule Dosage Form Category pmjackson Thu, 03/27/2025 - 19:42 Laval, Qubec, March 27, 2025 Altasciences , a nine-time CRO Leadership Award winner, is proud to announce they have been awarded a 2025 CDMO Leadership Award in the SmallMolecule Dosage FormNorth America category.
Drug Target Review had the pleasure of attending SLAS Europe 2025 in Hamburg and what we saw was no mere showcase of cutting-edge tools, but a clear snapshot of where drug discovery is heading. So what really stood out at SLAS 2025? You can put everything into one platform, and multiple people can engage with the data, she explained.
Despite recent progress with AAV Gene Therapy and Antibody-Oligonucleotide Conjugates, a broad range of muscular dystrophies are still untreatable, and collectively represent an enormous unmet medical need. First, they provide guidance for the design and refinement of micro-dystrophin gene therapy constructs for DMD. 2024), Wan et al.
Drug development is hampered by high costs, long timelines and a low probability of success and complex therapies exacerbate these challenges. Traditional drugs for eye diseases like glaucoma are smallmolecules delivered by simple eye drops. 2024 [cited 2025 Feb 7]. Velez (2025). Lifescienceleader.com.
3 These models are pivotal for offering key predictive insights into the clearance of metabolically stable smallmolecules, maintaining phase I and II metabolic activities for up to four weeks. 9,10 According to IQ-MPS recommendations, liver-on-chip systems can differentiate between toxic and non-toxic smallmolecule analogues.
2025 Apr 1:10915818251327963. ETRIPAMIL CAS 1593673-23-4 AS ACETATE 512.64 ETRIPAMIL CAS 1593673-23-4 AS ACETATE 512.64 Milestone Pharmaceuticals Announces USAN Approval of Generic Name Etripamil for its Phase 2 Clinical Development Product for the Treatment of Paroxysmal Supraventricular Tachycardia. [3]. Ascah A, et al. Int J Toxicol.
Retrieved 17 April 2025. Jump up to: a b New Drug Therapy Approvals 2023 (PDF). 2] [5] It is taken by mouth. [2] 5] Omaveloxolone was approved for medical use in the United States in February 2023, [2] [5] [6] [7] [8] and in the European Union in February 2024. [3] 9] SYNTHESIS PATENT Sheikh, AY et al. 2] [5] It is taken by mouth. [2]
“Therapy for Alzheimer’s disease: Missing targets and functional markers?” “Versatile nanomaterials for Alzheimer’s disease: Pathogenesis inspired disease-modifying therapy” Journal of Controlled Release. Kg of androst-5-en-17-one-3,7-diol, 23.8 Kg 1,1,1,3,3,3-hexamethyldisilazane (HMDS) and 0.7
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