Drug Hunter, Palo Alto, CA (2025)

11/18/2025

You can now take Drug Hunter with you wherever you go!

Our new mobile app puts the world of drug discovery right in your pocket — so you can read, learn, and explore whenever inspiration strikes.

Download the app today to:
- Access exclusive content faster
- Get notifications on the latest breakthroughs
- Stay connected with the community

Download the app today in the Apple App Store or Google Play Store.

11/17/2025

Patent Target Brief: Macrocycles Targeting the LAG3:MHC-II Protein–Protein Interactions | https://drughunters.com/3LLctrV

Immune checkpoint blockade reshaped cancer care: PD-1/CTLA-4 success showed that blocking inhibitory checkpoint nodes can restore effector T-cell function. In 2022, the FDA approved the first-in-class anti-LAG3 antibody, relatlimab (in combination with nivolumab), clinically validating LAG3. However, most LAG3 agents are antibodies with inherent limitations.

BMS is charting a different path: patent applications describing 12–13-residue macrocyclic peptides targeting the LAG3:MHC-II PPI. Markush structures, representative examples, and biological data are shown in this Patent Target Brief.

Read it on Drug Hunter: https://drughunters.com/3LLctrV

11/15/2025

Taletrectinib: A CNS-Active ROS1 Inhibitor Poised for First-Line Use in nSCLC | https://drughunters.com/4nV4dTq

Taletrectinib (Ibtrozi®), an oral, CNS-active ROS1 TKI from Nuvation Bio received full FDAapproval in June 2025 for the treatment of locally advanced or metastatic ROS1-positive nSCLC. ROS1, an integral receptor protein tyrosine kinase, becomes oncogenic when gene rearrangements create ROS1 fusion oncoproteins that drive signaling.

Several FDA-approved ROS1 inhibitors have demonstrated efficacy in nSCLC, beginning with the 2016 approval of crizotinib, but resistance mutations—notably G2032R—along with lack of CNS exposure and tolerability issues, have limited the utility of these drugs in many patients.

This case study reviews the evolution of ROS1 drugging in nSCLC, how polypharmacology has shaped both efficacy and treatment-limiting CNS toxicities across this class of TKIs, and technology advances that may sharpen patient identification for those possessing ROS1 rearrangements.

Read it on Drug Hunter: https://drughunters.com/4nV4dTq

11/14/2025

Will a New Binding Approach Reinvigorate Caspase-1 as a Therapeutic Target for Autoimmune Disorders? | https://drughunters.com/49dHj6j

Caspase-1, a key cysteine protease within the NLRP3 inflammasome, plays a critical role in activating pro-inflammatory cytokines and triggering pyroptosis. Inhibitors including VX-740 and VX-765 from Vertex showed promise in preclinical models, however, clinical efficacy remained elusive. A new approach could reinvigorate interest in targeting caspase-1. CIB-1476 binds to the inactive pro-caspase-1 zymogen, and early dpreclinical data suggests this strategy may offer more comprehensive suppression of inflammasome-driven inflammation. Be sure to check out our target brief to learn more about this complex target.

Read it on Drug Hunter: https://drughunters.com/49dHj6j

11/13/2025

📣Reminder
Process Development of Lenacapavir
https://drughunters.com/3JwIMu9
Thursday, November 20th, 2025
8 AM PT | 11 AM ET | 5 PM CET

Join us for this Flash Talk with Nathaniel Kadunce! Nat will share the development story behind lenacapavir, the first-in-class, long-acting injectable HIV capsid inhibitor approved by the FDA for both the treatment and prevention of HIV infection. He will present the commercial manufacturing route for this highly complex drug.

Nat will walk through multiple generations of route optimization for synthesizing lenacapavir’s intricate tricyclic pyrazole ring, highlighting the stereospecific introduction of the cyclopropane ring, followed by a series of oxidation and functionalization steps that build molecular complexity and culminate in a desulfurative fluorination to complete the core scaffold.

Secure your spot to understand how modern process chemistry teams tackle increasingly complex synthetic challenges!

Register here: https://drughunters.com/3JwIMu9

11/13/2025

Nerandomilast (BI 1015550): A Selective PDE4B Inhibitor Approved for IPF | https://drughunters.com/4488jR7

After almost a decade without new options, nerandomilast was recently FDA-approved for IPF, on the basis of reduced declines in forced vital capacity following robust preclinical lung-inflammation studies.

Mechanistically, it preferentially inhibits PDE4B over PDE4D which, together with limited brain exposure, underlies the cleaner emesis side effect profile versus earlier inhibitors.

Structural work on related selective inhibitors bound to PDE4B vs. PDE4D suggests a distinct, water-network–mediated binding mode that helps drive isoform selectivity.

Read it on Drug Hunter: https://drughunters.com/4488jR7

11/12/2025

Targeting NLRP3: DFV890 and Beyond
https://drughunters.com/3JTPcDv
Thursday, December 4th, 2025
8 AM PT | 11 AM ET | 5 PM CET

The NLRP3 inflammasome plays a crucial role in innate immunity, acting as a key molecular sensor that triggers inflammasome activation and drives IL-1β-mediated inflammation implicated in a broad range of human diseases.

In this Flash Talk, Angela MacKay, Director of Medicinal Chemistry, will present drug discovery projects targeting NLRP3. She will focus on the discovery story of Novartis' DFV890, which was initially developed by IFM-Tre. This potent and selective NLRP3 inhibitor is currently advancing through multiple Phase 2 clinical trials across several indications.

Angela will highlight how initial NRLP3 inhibitors were discovered through cell-based phenotypic screenings. The following identification of NLRP3 as a therapeutic target led to a broad portfolio of drug discovery programs spanning peripheral as well as CNS indications.

Sign up now to refresh your knowledge on the latest developments in targeting NLRP3 across a range of inflammatory indications: https://drughunters.com/3JTPcDv

11/12/2025

Repurposed Sheep Antiparasitic Monepantel (NUZ-001) Enters HEALEY-ALS Platform Trial | https://drughunters.com/47AY9L7

Monepantel (NUZ-001) is a veterinary anthelmintic being repurposed for ALS that is now advancing in an ALS Platform Trial. An mTOR inhibitor that induces autophagy, Neurizon Therapeutics' NUZ-001, aims to enhance the clearance of aggregation-prone proteins, a hallmark of ALS.

Originally, the approved veterinary liquid formulation, Zolvix®, was assessed in humans but was unpalatable with a high frequency of gastrointestinal adverse events, leading to its reformulation into an oral tablet. The Ph. 1 trial met primary safety endpoints, with observations of mTOR PD biomarker changes and CSF exposure.

The compound is the eighth therapy to enroll in the HEALEY-ALS platform trial, an operation that allows for multiple and continuous evaluation of therapies for the same disease, cutting time and cost of development to assess clinical candidates. NUZ-001 will begin dosing in the HEALEY Ph. 2/3 trial Q4 of 2025.

Read it on Drug Hunter: https://drughunters.com/47AY9L7

11/11/2025

In this Flash Talk, Alex Berne and Lucas Watkins presented an overview of the fundamentals of patent law.

They discussed how patent scope is defined by claims and highlighted key considerations that guide filing decisions. Strategic decisions about when and how to file, whether to secure an early priority date or strengthen claims with additional data, can have long-lasting business and scientific implications.

Through four landmark pharmaceutical legal cases, they showed how utility, novelty, nonobviousness, and enablement/written description often serve as key lines of argument in IP litigation.

If you’re interested in learning more about best practices in patent strategy and gaining expert insights into IP challenges, watch the full Flash Talk: https://drughunters.com/43hJqSU

11/10/2025

PAH Activator Sepiapterin’s Approval a Win For PKU Patients | https://drughunters.com/3Lva4l7

Sepiapterin (Sephience®), an orally bioavailable PAH (phenylalanine hydroxylase) activator from PTC Therapeutics, was approved in July 2025 for the treatment of PKU (phenylketonuria), a condition characterized by neurotoxic Phe (phenylalanine) accumulation.

A precursor to the PAH cofactor BH4 (tetrahydrobiopterin), sepiapterin has improved cellular uptake and CNS penetrance compared to the approved synthetic BH4 formulation, sapropterin (Kuvan®). In a head-to-head Ph. 2 study with sapropterin, sepiapterin led to greater decreases in blood Phe than sapropterin at both doses tested.

In a Ph. 3 trial , blood Phe levels were lowered in 73% of participants administered sepiapterin, and in an open-extension trial 97% of patients in a Phe tolerance protocol demonstrated the ability to liberalize their diet, a critical quality-of-life improvement.

Originally developed by Censa Pharmaceuticals, sepiapterin was developed by PTC Therapeutics after Censa’s acquisition in 2020.

Read it on Drug Hunter | https://drughunters.com/3Lva4l7

11/10/2025

📣 Your First Look at the Drug Hunter Drug Discovery Course!
https://drughunters.com/43lGuVj

Starting January 2026, the Drug Hunter team will release bite-size,

11/08/2025

Discovery of CHF6333: An Inhaled Neutrophil Elastase Inhibitor | https://drughunters.com/4hOdITb

NE (neutrophil elastase) is a key driver of airway damage, mucus obstruction, infection, and recurrent exacerbations in bronchiectasis.

CHF6333 is an inhaled, lung-restricted NE inhibitor from Farmaceutici designed to restore the protease/antiprotease balance locally in the lungs while minimizing systemic exposure and off-target toxicity. CHF6333 is now in Phase 1/2 clinical trials.

This case study summarizes the discovery and optimization of CHF6333, as presented by Fabio Rancati at the RSC/SCI 23rd Medicinal Chemistry Symposium.

Read it on Drug Hunter: https://drughunters.com/4hOdITb

Drug Hunter

11/18/2025

11/17/2025

11/15/2025

11/14/2025

11/13/2025

11/13/2025

11/12/2025

11/12/2025

11/11/2025

11/10/2025

11/10/2025

11/08/2025

Address

Telephone

Website

Alerts

Contact The Practice

Shortcuts

Share