This high-level overview of 2021’s novel large molecule drug approvals is the 2 nd in a series of focus articles on 2021’s new drugs (see the small molecules here ). You can find our deep-dive on 2021's large molecule drug approvals here . There were: [...]

Drug Hunter’s February 2022 Small Molecules of the Month include a glucokinase partial activator, an orexin receptor agonist intended to promote wakefulness rather than sleep, and an orally available diacid, among others. Thanks to Kim Huard, Julien Lefranc, and Bryan McKibben for their nominations and insights this month. Is BMS-820132 a prodrug? How is T-690 different from previous generations of GCS inhibitors? Which off-target is correlated to loss of appetite in rats? What were the starting points for these molecules? Premium Members can find these answers and more in the dedicated summar...

Announcement: Appointment of Drug Hunter Advisory Board Member: Gary Yeung, CFA Drug Hunter is pleased to introduce a new member of our Advisory Board, Gary Yeung . Gary Yeung is a life science executive (President, COO, CFO, and CBO), board member, advisor, and investor with extensive strategy, finance, business development, and operations [...]

We’ll be reviewing 2021’s novel FDA drug approvals in a series of articles, starting with this high-level overview of the small molecule drugs approved by the FDA in 2021. You can find Part 2 on First-in-Class Small Molecules here and the 2021 Large Molecules series here . Our deep dive on Small Molecule Oncology Approvals is also live [...]

Announcement: Appointment of Drug Hunter Advisory Board Member: Wendy Young, Ph. D . Drug Hunter is pleased to introduce a new member of our Advisory Board , Dr. Wendy Young. Wendy Young, Ph.D. is a seasoned drug hunter and biotechnology, pharma and life science executive with more than 25 years of experience in the discovery and development [...]

If you’re here reading about drug discovery for fun, you’re probably a drug hunter. You might be someone on your way to a leadership role in pharma or biotech. Maybe you’re decades into your career and already leading an R&D division. You don’t have a PhD in drug hunting, and it’s unlikely to be in your job description. But something makes [...]

We've just published the final set of Molecules of the Month for 2021, and now it's time to nominate your favorite molecules for 2021's Molecules of the Year! Nominees should have either been first disclosed in 2021 or had a major new update in 2021 (e.g. clinical trial readout, publication, business transaction, approval). The molecule must [...]

Retinol-binding protein 4 (RBP4) is the sole carrier for retinol (vitamin A). It may also play a role in diabetes and reduction of RBP4 may be beneficial for the ocular disease AMD. Belite Bio had a Ph. III trial ongoing for an oral RBP4 antagonist (LBS-008, tinlarebant ) in Stargardt Disease and intends to start a Ph. III trial for AMD in [...]

MRTX1133 was nominated as December 2021’s cover molecule by reviewers Joachim Rudolph and Julien Lefranc . MRTX1133 is a non-covalent inhibitor of KRAS G12D that demonstrated tumor regression in a mouse xenograft model when dosed IP (maximum efficacy at 10-30 mg/kg, activity as low as 3 mg/kg). Joachim says, “The discovery of MRTX1133 [...]

CC-90001 was nominated by reviewer Christian Kuttruff . CC-90001 is a JNK (c-Jun N-Terminal Kinase) inhibitor and a Phase II clinical candidate for idiopathic pulmonary fibrosis ( NCT03142191 ). Christian says, “Preclinical evidence suggests that c-Jun N-terminal kinase (JNK) enzyme function is required for key steps in the pulmonary fibrotic [...]

Mutated Huntingtin (HTT) protein is the cause of Huntington’s disease (HD). HTT-D3 is an oral, CNS-penetrant molecule with human HTT lowering activity in vivo used to illustrate the science around PTC Therapeutics’ Huntingtin’s disease program. PTC had filed patent applications for the HD program as early as November 2016 ( WO201700726 ). PTC [...]

Ulotaront (SEP-363856) is a Phase III candidate with FDA Breakthrough Therapy Designation for the treatment of schizophrenia with demonstrated efficacy based on a physician-rated scale (PANSS) in a 4-week placebo-controlled trial and continued improvement in an open-label extension study. Reviewer Jake Schwarz says, “ulotaront was developed [...]

Velsecorat (AZD7594) is an inhaled, non-steroidal glucocorticoid receptor modulator and antedrug (soft drug) for treatment of asthma and other respiratory conditions that has completed several Ph. II studies . While the paper states that the results support progression into Ph. III studies, a Ph. III study does not appear to have started and [...]

Vertex recently disclosed a CNS-penetrant ELOVL1 inhibitor, in which they described preclinical safety findings in rat and cyno. In this article, they disclose a structurally distinct series which displayed similar ocular toxicity in rat, and skin and CNS toxicities in cyno. A third structurally distinct series (undisclosed) also showed [...]

RP-3500 is an oral ATR kinase inhibitor and Ph. II clinical candidate for advanced solid tumors with ATRi-sensitizing mutations, alone or in combination with talazoparib (PARPi) or gemcitabine. Reviewer Anthony Vaganos says, “An ATR molecule ( ceralasertib ) is in clinical trials but studies in two different tumors in combination with PARP [...]

Gout is caused by the buildup of uric acid. The main treatments are drugs that increase uric acid secretion (uricosurics, e.g. probenecid ) and xanthine oxidase inhibitors which inhibit uric acid production (e.g. allopurinol ). URAT1 inhibitors like lesinurad (Ironwood Pharmaceuticals) and dotinurad are emerging treatments, inhibiting uric [...]

JAK kinases are now well-established targets in immunology, but the class has always had safety concerns hanging over it. The safety issues were exacerbated by a recent FDA decision to expand the black box warnings for the class for major cardiovascular events. Inhaled, lung-restricted JAK inhibitors have been of interest because they could [...]

Kinase inhibitors are increasingly being explored for diseases beyond cancer. While SGK1 has been a cancer target, SGK1 has also been implicated in osteoarthritis pathology, and the Sanofi team developed “compound 17a” to interrogate this. The starting point was identified from ligand-based virtual screening, focusing on the DFG-out [...]