The team reviews hundreds of compounds from thousands of papers, press releases, and other sources each month to select candidates for Molecules of the Month. Here we have compiled a table of >70 additional molecules that were of interest in November 2023 along with some highlights from some of our favorites below.

Leniolisib is a selective PI3Kδ inhibitor that received approval in March 2023 from the FDA as a first-in-class treatment for activated PI3K-δ syndrome (APDS), a genetic immunodeficiency disorder caused by PI3Kδ activation. The leniolisib discovery program is notable for several reasons: it is a first approval for a PI3K inhibitor outside of oncology; the first approved drug for APDS; has a novel chemotype that appears to avoid safety issues that led to black box warnings seen with other PI3K molecules; and it overcame a polymorph issue with an early lead that led to inconsistent exposures.

Several multi-million dollar acquisitions or license agreements took place in November 2023 for hot drug targets, with assets from selective PARP1 inhibitors to GLP1R agonists, and others. This recent news roundup highlights the notable acquisitions by pharma companies for rights to molecules in a range of different indications, with illustrative patent examples for undisclosed structures.

Whereas covalent drugs were once considered risky for indications other than cancer due to the potential for idiosyncratic drug-induced liver injury and other toxicities, concerns appear to have softened as an increasing number of non-oncology covalent inhibitor programs have achieved successful outcomes. Here we highlight several recent anecdotes in covalent inhibitor drug discovery that seem to reflect these changing attitudes, including billion-dollar acquisitions and approvals outside oncology, and a few modern examples that showcase the increasingly rich science in this area.

The team reviews hundreds of compounds from thousands of papers, press releases, and other sources each month to select candidates for Molecules of the Month. Here we have compiled a table of >70 additional molecules that were of interest in October 2023 but we did not have space for in MOTM. A few highlights are also discussed, including Novartis’s Werner helicase (WRN) inhibitor, APJ agonist AMG985 molecular glue degrader golcadomide, and more in the full article.

The Hippo pathway is a well-known regulator of tissue growth and organ size and gets its name from the Hippo kinase (Hpo in Drosophila, MST1/2 in humans), which when mutated leads to “hippopotamus”-like tissue overgrowth. Recently, industry interest in the pathway has increased thanks to several independent studies in 2023, and the field appears to be at an important inflection point for drug discovery. Here we review current efforts and therapeutic hypotheses for drugging the TEAD transcription factors in cancer, including clinical molecules, paralog-selective molecules, and TEAD degraders.

KIN-3248 (Kinnate Biopharma) is a next-generation, irreversible, pan-fibroblast growth factor receptor inhibitor (FGFRi) in a first-in-human (FIH) dose-escalation study (NCT05242822) in adults with advanced tumors harboring FGFR2 and FGFR3 gene alterations. The program is an excellent case study for rational covalent drug design to address kinase mutations while maintaining sufficient PK properties for oral daily dosing in humans.

Peptidic GLP-1R agonists have received significant media coverage over the past year for their astounding efficacy in several indications. GLP-1R agonists now appear to be efficacious in reducing heart disease risk and in treating certain chronic kidney diseases, expanding their likely future use. This review by Oliver Philps provides an overview of GLP-1 agonists and where they fit in the treatment of diabetes, how the peptide drugs evolved over time to today’s weight loss medications, and what to expect next...

There continue to be opportunities for another KRAS(G12C) inhibitor to prove itself best-in-class.

This roundup highlights twelve KRAS(G12C) molecules to know, with links to recent case studies on Drug Hunter going in more detail.

The team reviews hundreds of compounds from thousands of papers, press releases, and other sources each month to select candidates for Molecules of the Month. Based on requests from readers to see more molecules, this article contains a table of >40 additional molecules that were of interest last month but not included in MOTM, with a few highlights including off-target toxicity seen in a KRAS(G12D) program and hits on challenging target classes.

Recently, brain-penetration data via PET was reported for several KRAS(G12C) inhibitors including AZD4747, adagrasib, and sotorasib. AZD4747 has been updated with additional PET data for KRAS(G12C) inhibitors from a recent conference in the UK. Additionally, Tobias Biberger has contributed an update to the recent AZD4747 case study highlighting its interesting medicinal chemistry route with some surprising steps including manipulations of an unprotected phenol, and the synthesis of its PET tracer...

Amgen’s first-in-class KRAS(G12C) inhibitor, sotorasib, recently encountered an unexpected but significant regulatory setback with the FDA at its October 5th Oncologic Drugs Advisory Committee Meeting (ODAC). Full approval for sotorasib had widely been expected in the industry, but the FDA ODAC voted 10 to 2 that progression-free survival (PFS), the key endpoint for comparing sotorasib to docetaxel chemotherapy, could not be reliably interpreted in the pivotal CodeBreaK200 (NCT04303780) study for KRASG12C+ late-stage NSCLC. What happened, and what does it mean for KRAS inhibitors?

Understanding how a small molecule ligand binds to its target is valuable in drug discovery, because it enables more efficient optimization through structure-based design, better mechanistic understanding of molecular pharmacology, and greater confidence in the therapeutic hypothesis from both safety and efficacy perspectives. Recently, Drug Hunter highlighted methods for target identification when the target is unknown.

Obtaining adequate drug exposure in the brain is key to treating CNS diseases effectively. Recently, Dennis Koester gave us a crash course in CNS drug discovery in a Drug Hunter Flash Talk. Here, he sums up some key points on how to find compounds that cross the blood-brain barrier.

LRRK2 has been a hotly pursued drug target for Parkinson’s Disease based on human genetics. Clinical advancement of LRRK2 inhibitors was initially stalled by concerns about on-target lung findings in primates, but these were ameliorated by a Merck/Genentech/Pfizer/MJFF study showing that these lung changes were reversible, and Biogen/Denali has currently a small molecule (BIIB122/DNL151) in Ph. II (NCT05348785) after demonstrating safety and CNS exposure in 145 healthy patients in Ph. I (NCT04557800)

In Wednesday’s Flash Talk webinar, we covered recent highlights in targeted protein degradation, with lots of great questions from the audience. The webinar and recording are available below to everyone thanks to our sponsor, Revvity Signals, and the slides are available for download to Premium members at the bottom.

This article compiles recent high-profile clinical readouts and related news with small molecules of general interest and structures where they are available. Targets and indications discussed in this article include BTK for MS, Axl, TYRO3, and Mer for oncology, HIF prolyl-hydroxylase in anemia, ET/AT in nephrology, a JAK2 inhibitor for myelofibrosis, and THR-beta in NASH. [...]

This article highlights million-dollar molecules that were recently in the news with related patent applications that give clues to their structures and properties. This edition includes Maze’s glycogen synthase 1 (GYS1) inhibitors that were recently licensed to Sanofi, allosteric androgen receptor (AR) modulators that may be of interest to targeted protein degradation researchers, brain-penetrant HER2 and ROCK2 inhibitors, and MDM2 degraders.

This article compiles 20+ recent small molecules of general interest in the news in April 2023, with structures where they are available. Capacity Bio’s MAS Receptor Agonists with $35M Raised for FIH Clinical Trials MAS modulator from WO2022165189 example 107 New start-up Capacity Bio backed by RA Capital, Insight Partners, and Remiges [...]

We couldn’t include everything in Molecules of the Month, but here’s a round-up of up of 16 additional molecules of potential interest that we didn’t have space for in April, including a highly selective caspase inhibitor from a non-catalytic cysteine, a GPR40 AgoPAM from BMS with activity on GLP-1, a few examples of both on- and off-target [...]

This “journal club” article highlights five recent articles of general interest to drug discovery scientists, including reviews on therapeutic areas of interest, principles and areas of medicinal chemistry and pharmacology, and a synthetic transformation worth keeping in mind. 1. A “Magic Chloro” Med. Chem. Review D. Chiodi and Y. Ishihara [...]

This article highlights six recent articles of interest in the field of targeted protein degradation including but not limited to potentially new ligases, recruiting motifs, and a discussion on the feasibility of CNS-penetrant degraders. 1. “Reinstating targeted protein degradation with DCAF1 PROTACs in CRBN PROTAC resistant settings” [...]

There were plenty of scientific highlights from the literature that we didn’t have space for in Molecules of the Month - here’s a reader’s digest rounding up ~20 molecules of potential interest that we didn’t have space to highlight, including Pfizer’s covalent heterobifunctional BTK degrader, BMS' brain-penetrant GSK-3β inhibitor, the [...]

Keep up with the latest updates on past Molecules of the Month! From a Ph. III oral HIF-PH inhibitor receiving a favorable response from the EMA to an oral mutant IDH1/2 inhibitor with FIC potential having favorable Ph. III results for hard-to-treat brain tumors, read more about the recent updates in the full article! Want updates like this [...]

This article highlights some recent million-dollar molecules, including: Gilead's exclusively licensed preclinical, oral IRAK4 degrader from Nurix Biohaven's exclusively licensed oral, brain-penetrant, dual TYK2/JAK1 inhibitor from Highlightll Pharmaceutical Eli Lilly's acquisition of a Ph. I oral AT 2 R inhibitor from Confo Therapeutics Read [...]

This article discusses scientifically interesting molecules that have unfortunately had negative recent readouts. Aptinyx’s therapeutic approach of targeting positive allosteric modulators (PAMs) of NMDA receptors has hit another setback in Ph. II for NYX-458. The oral, twice-daily MK2 inhibitor from Aclaris Therapeutics has results from its [...]

The AACR 2023 first disclosures presented in the Chemistry in Cancer Research’s “New Drugs on the Horizon” sessions are always fascinating. This article contains the structures of all of the novel small molecule first disclosures presented at AACR in the session, along with notes on their significance, and another molecule disclosed in the [...]

Merck’s oral PCSK9 inhibitor is a surprisingly bioavailable and orally effective macrocyclic peptide with picomolar activity. It recently demonstrated significant LDL cholesterol reduction in Ph. IIb, paving the way for it to enter Ph. III later this year. Roche’s oral, SMN2-directed RNA splicing modifier and first molecule of its kind to be [...]

Pfizer spinout, SpringWorks Therapeutics, was granted priority review by the FDA for its oral gamma secretase inhibitor for the treatment of soft-tissue desmoid tumors, which has no FDA-approved therapies. Meanwhile, Pharming Groups’ oral PI3Kδ inhibitor (licensed from Novartis) received the first and only FDA-approval for the treatment of [...]

The First Disclosures section of the 2023 ACS Spring Meeting in Indianapolis, organized by GSK’s Nikki Goodwin , covers three novel small molecules and an ADC this year. The molecules include: MK-0616, Merck’s macrocyclic oral PCSK9 inhibitor (presented by Abbas Walji ) NX-2127, Nurix’s oral BTK degrader (presented by Jeff Wu ) BI 1015550 [...]

On Monday, Mar. 27th (12:15 PM ET), our Drug Hunter team will be sponsoring a luncheon at ACS Spring 2023 in Indianapolis with a presentation on scientific gems from 2022’s drug approvals ( request an invitation here ). Each newly approved drug has an interesting scientific story, and you can see our overview of all 36 of the novel small [...]

In this update of small molecules in the news for February 2023, we will cover recent: - Molecules in Motion : clinical trial updates and FDA filings - Negative Readouts : halted, discontinued or altered clinical trials - Million-Dollar Molecules : recent M&A’s, IPOs, and other fundraising - Potential Approvals : recent NDA filings and [...]

Our Drug Hunter subscribers at Curie.Bio recently made headlines with the launch of their new >$500M venture fund with a new model for early-stage biotech financing to help founders launch therapeutics companies. Curie.Bio intends to act not only as an investor, but also as a day-to-day operating partner to help founders choose targets [...]

Mirati’s “Breakthrough” KRAS G12C inhibitor, adagrasib, was granted accelerated approval by the FDA in 2022. As it follows Amgen’s first-in-class KRAS G12C inhibitor, sotorasib, adagrasib is constantly scrutinized for any evidence it may be different, or better, than sotorasib. This deep dive reviews the current understanding on how adagrasib [...]

Our latest roundup of small molecules in the news includes two potential upcoming approvals: rezafungin Rezafungin (CD-101) is a once-weekly IV antifungal agent and next-generation echinocandin (glucan synthase inhibitor) that demonstrated non-inferiority vs. standard of care caspofungin (IV QD) (NCT03667690). An FDA AdCom voted 14 to 1 in [...]

In this update of small molecules in the news we will cover recent: (B/M)illion-Dollar Molecules: recent M&A’s, IPOs, and other fundraising Molecules in Motion: clinical trial updates and FDA filings Where Are They Now? updates on past Drug Hunter Molecules of the Month Potential Upcoming Approvals : recent NDA filings and upcoming PDUFA [...]

You can download our compilation of the 9 first-in-class small molecule drug approvals from 2022 below, which includes structures, drug targets and mechanisms of action, doses and routes of administration, and indication.

In 2022 there were 36 novel small molecule and large molecule drugs approved by the FDA, including 18 first-in-class drug approvals. This page contains posters and reviews of the science behind each molecule including the: 19 small molecule drug approvals (excluding atoms) 17 large molecule drug approvals (click to jump to poster) 9 first in [...]

Futibatinib (Lytgobi) is a rare alkyne-containing, irreversible FGFR1-4 inhibitor that received accelerated approval from the FDA in September 2022. Structurally distinct from other approved FGFR2/3 inhibitors, futibatinib treats a range of FGFR-aberrant tumors, including those with fusions or TKI-resistant mutants like the V565I/L gatekeeper [...]

Billion-Dollar Molecules: Etavopivat, an Allosteric PKR Kinase Activator for Sickle Cell Disease Abstract. Novo Nordisk has acquired Forma Therapeutics for $1.1B for its primary investigational drug, etavopivat. Etavopivat, a past Molecule of the Month , is an oral, once-daily allosteric activator of pyruvate kinase-R (PKR), which is [...]

Omidenepag isopropyl (Omlonti) is a prodrug of a prostaglandin E2 (EP2) receptor agonist that reduces pressure in the eye. Upon topical administration to the eye, the molecule penetrates the lipophilic and negatively charged corneal epithelium and is converted to the pharmacologically active form by the enzyme carboxylesterase-1. The [...]

In 2022, Turning Point Therapeutics ($TPTX) was acquired by BMS for a whopping $4.1 billion in cash. In November 2023, the most advanced molecule from Turning Point, repotrectinib, was approved for the treatment of ROS1+ NSCLC. This article explores the science behind repotrectinib, what made Turning Point so valuable, how we know, and what's next. [...]

PI3 kinases are part of the PI3K/Akt/mTOR signaling pathway that is downstream of Receptor Tyrosine Kinases (RTKs), G-protein-coupled receptors (GPCR), and GTPases such as KRAS. Since their identification in the 1980s, and due to their central role in cancer, the therapeutic targeting of the phosphatidylinositol-3-kinase (PI3K), and more [...]

There have been numerous reviews on the impact of AI/ML in drug discovery looking at broad numbers, but with the volume of companies in the space, few have spelled out exactly what these companies are working on to paint a picture of what the landscape of AI/ML in small molecule drug discovery actually looks like today. We concluded part II [...]

Despite significant investment over the past decade, only a handful of companies are now bringing clinical candidates discovered with the assistance of AI/ML forward. Though there’s a looseness to what should count as an “AI-discovered drug,” here we highlight internally-generated drug programs from companies focused on leveraging AI/ML in [...]

You’ve read about the top 10 molecules with publications from August, but what about the ones we found interesting that didn’t make it into Molecules of the Month? There were five runners-up from August 2022, including: a highly selective, dihydropyrimidinone-based CSF1R switch-control kinase inhibitor a non-competitive, allosteric CXCR1/2 [...]

In September 2022, deucravacitinib (Sotyktu) , a first-in-class oral, allosteric tyrosine kinase 2 (TYK2) inhibitor for moderate-to-severe plaque psoriasis, was approved by the FDA. Aside from its first-in-class mechanism of action, it is the first approved de novo deuterated drug. This premium deep dive into deucravacitinib includes: the [...]

Abstract. Voxelotor, a gram-daily reversible covalent aldehyde drug, is an approved first-in-class oral drug that prevents the hemoglobin polymerization that drives sickle cell disease. It was one of several agents in clinical trials acquired from Global Blood Therapeutics by Pfizer as part of an over $5 billion buyout. In this article, we [...]

In August 2022, olipudase alfa (Xenpozyme), a hydrolytic lysosomal sphingomyelin-specific enzyme indicated for non–CNS manifestations of acid sphingomyelinase deficiency (ASMD), was approved in the US and Europe. This Premium deep dive into Xenpozyme includes: the industry context, the clinical data, the target rationale, the mechanism of [...]

In August 2022, the European Medicines Agency granted Marketing Authorization to lenacapavir (Sunlenca), the first-in-class bi-annually dosed HIV capsid inhibitor indicated for multidrug-resistant HIV-1 infection. This Premium deep dive into Sunlenca includes: the industry context the clinical data the target rationale the mechanism of action [...]

We did a deep dive into the pharmaceutical industry’s 2022Q2/Q3 news, and there’s a ton of exciting updates to share. In this review, we highlight some emerging companies, technologies, and drug discovery modalities we thought drug hunters would find interesting, including: New strategies for covalent protein modification Different methods [...]

Our team conducted a review of all  the biotech acquisitions of 2021 and distilled it down to the acquisitions of drug discovery-focused biotechs. Navigate the 2021 M&A Series the top overall drug discovery M&A transactions by value (Part 1) the transactions with disclosed upfront payments (Part 2) the most valuable small molecule [...]

Amgen has acquired ChemoCentryx in a deal that includes lead molecule avacopan (Tavneos), a first-in-class orally bioavailable small molecule complement 5a receptor (C5aR) antagonist approved as an adjunctive treatment to standard of care therapy for adults with severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated [...]

You’ve read about the top 10 molecules with publications from July, but what about the ones we found interesting that didn’t make it into Molecules of the Month? There were six runners-up this month, including a selective covalent K-Ras(G12S) inhibitor with a butyrolactone warhead, a κ opioid receptor agonist, and a dinucleotide STING agonist [...]

This article is Part VI of a review series covering all the initial public offerings for the biotech and pharmaceutical industries in 2021. Pt. I and Pt. II provided an overview of 2021 IPOs by therapy area, technology, and therapeutic class. Pt. III and Pt. IV provided a deep dive into the 2021 IPOs for oncology-focused companies, while P.t [...]

Spinal Muscular Atrophy (SMA)—the leading genetic cause of childhood mortality—is a disease in which the gene SMN1 is deleted or mutated. The highly similar gene SMN2 can undergo alternative splicing to generate functional protein transcripts of the SMN1 gene product, although native levels of the protein are too low to overcome the pathology [...]

This article is Part 5 of a review series covering all the initial public offerings for the biotech and pharmaceutical industries in 2021. Part 1 and Part 2 provided an overview of 2021 IPOs by therapy area, technology, and therapeutic class. Part 3 and Part 4 provided a deep dive into the 2021 IPOs for oncology-focused companies developing [...]

Readers have been asking for more molecules, so we started a new feature that highlights a few of the molecules we liked that didn’t make it into Molecules of the Month. There were six runners-up this month, including an orally available ALK2 inhibitor and a direct AMPK activator for X-linked adrenoleukodystrophy. You can read more about all [...]

This short article and poster summary takes a deeper look into the 2021 IPOs for biotechnology-based therapeutics being developed by oncology-focused companies, including: Antibody-drug conjugates Monoclonal antibodies Protein-based therapeutics Cell and gene therapies Other biotechnology-based therapeutics This article is Part 4 of a review [...]

This article is Part 3 of a review series covering all of the initial public offerings for the biotech and pharmaceutical industries in 2021. Part 1 provided an overview of 2021 IPOs by therapy area, technology, and therapeutic class. Part 2 provided an overview of 2021 IPOs by therapeutic area. This short article takes a deeper look into the [...]

There’s so many interesting new publications that don’t necessarily feature key molecules, but are helpful for drug hunters to know about. Here are some other interesting reviews, technology, and targets to watch from April and May: April 2022 Best Practices : Understanding PK-PD for Protein Degraders: A Review . Derek Bartlett and Adam [...]

Target-driven drug discovery evolves over time and is influenced by understanding of disease biology, technical innovation, and most of all, clinical data. Our new series reviews drug targets with significant recent clinical data or industry events that have influenced or changed recent perceptions of them. Here we look at 10 targeted agents [...]

In June 2022, the FDA approved vutrisiran (Amvuttra), a small interfering RNA (siRNA) for polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults. This deep dive explores the drug in more detail, including the industry context, the clinical data, the target rationale, the mechanism of action, and the drug’s origins [...]

A first-in-class intravenous kappa opioid receptor (KOR) agonist, an oral soluble guanylate cyclase (sGC) stimulator, a first-in-class subcutaneous complement C3 inhibitor, and an oral contraceptive comprising a synthetic progesterone (drospirenone) and a plant-based estrogen (estetrol) make up the final group of novel drugs from 2021 that we [...]

This deep dive into 2021’s first-in-class large molecule drug approvals is the third in a series of focus articles on 2021’s novel large molecule drugs. See part one here , and part two here . For each of the 11 first-in-class large molecule drug approvals of 2021 (excluding vaccines), we’ll explore in more detail: how they work (MoA) key [...]

Readers have been asking for more molecules, so this month, we’re starting a new feature that will highlight a few of the molecules we liked that didn’t make it into Molecules of the Month. There were six runners-up this month, including an orally bioavailable, brain penetrant inhibitor of PAAN/MIF, and a STING agonist discovered using [...]

Our team conducted a review of all of the biotech mergers and acquisitions of 2021 and distilled it down to the acquisitions of drug discovery-focused biotechs here. There were 10 acquisitions of companies with large molecule drug assets in 2021. Here is a more in-depth look into all of the lead large molecules acquired in 2021, including a [...]

There were 16 acquisitions of companies with small molecule drug assets in 2021 and one merger. Here is a more in-depth look into all of the lead small molecules acquired in 2021, including a review of: The lead molecules in each acquisition and their structural representations where disclosed The mechanism of action, stage, and indication of [...]

There were two novel small molecules approved in May 2022: vonoprazan (Voquezna) : a potassium-competitive acid blocker (PCAB) tapinarof (Vtama) : an aryl hydrocarbon receptor (AhR) agonist And one novel large molecule approval: tirzepatide (Mounjaro) : a glucose-dependent insulinotropic polypeptide (GIP) receptor and glucagon-like peptide-1 [...]

This article is Part 2 of a review series covering all of the initial public offerings for the biotech and pharmaceutical industries in 2021. Part 1(link to article) provided an overview of 2021 IPOs by therapy area, technology, and therapeutic class. This short article takes a deeper look into the 2021 IPOs by technology and therapeutic [...]

In 2021, the FDA approved two new drugs for endocrinology-related indications, including severe hypoglycemia in diabetes, and chronic kidney disease (CKD) associated with type 2 diabetes. This article and slide deck explores each in more detail, including but not limited to their: target rationale discovery disease context original patents [...]

We did a comprehensive review of all the initial public offerings for the biotech and pharmaceutical industry in 2021. This article is Part 1 in a review series about companies focused on drug discovery that IPO’d during this time. You can find recent years’ IPOs summarized here: 2020 Small Molecule IPOs 2020 Large Molecule IPOs 2019 Small [...]

It’s best to be first, but drugs that are approved later with the same mechanism of action can also create significant value for patients and society by being best-in-class (BIC), or what clinicians refer to as “gold standard.” Recently we highlighted key factors that can make a follow-on drug be considered a best-in-class drug. This article [...]

In 2021 the FDA approved four new drugs for various immunological diseases, including a molecular glue, the first drug approved for AAV in a decade, and a ROCK kinase inhibitor originally investigated for cancer. This article and slide deck explores each in more detail, including but not limited to their: mechanisms of action target rationale [...]

The FDA in 2021 approved four novel small molecule drugs for various infectious diseases, including human immunodeficiency virus type-1 (HIV-1), post-transplant cytomegalovirus (CMV), vulvovaginal candidiasis, and human African trypanosomiasis (HAT; sleeping sickness). This article and slide deck explores each in more detail, including but [...]

We did a comprehensive review of industry news in 2022Q1 (there was.. a lot of stuff to cover). In this article, we highlight some emerging companies, technologies, and drug discovery modalities we thought drug hunters would find interesting, including: New platform companies focused on drug discovery Different approaches to glues and [...]

We did a comprehensive review of industry news in 2022Q1 (there was.. a lot of stuff to cover). This article highlights companies focused on drug discovery that IPO’d in the first quarter. The Drug Discovery IPO Market Slowdown As everyone knows, biotech’s been in a bear market since Feb. 2021 (with the entire $XBI index down more than 50% [...]

The FDA approved five new drugs in 2021 for use for genetic conditions, including molybdenum cofactor deficiency, Von Hippel-Lindau (VHL) disease, achondroplasia, and progressive familial intrahepatic cholestasis (PFIC). This article and slide deck reviews each in detail, including their mechanisms of action, originators, key clinical data [...]

There were two novel small molecules approved in April 2022: mavacamten (Camzyos), an oral cardiac myosin inhibitor oteseconazole (Vivjoy), an oral antifungal ( jump to section ) This deep dive explores each drug in more detail, including the industry context, the clinical data, the target rationale, mechanisms of action, and drug origins [...]

Momelotinib, an oral JAK1/2 inhibitor for myelofibrosis, is going to GSK as part of a $1.9 billion acquisition of Sierra Oncology. Anyone that’s ever worked on a kinase program probably looked at this structure and raised their eyebrows. $1.9 billion for another JAK2 inhibitor? It’s probably not super selective either, you might think… and [...]

What were the billion-dollar small molecules in 2021? This article and infographic, which is part 3 of a Premium series covering 2021 drug discovery M&A, covers: The ten largest drug discovery biotech acquisitions by total consideration in 2021 where the acquiree’s lead was a small molecule The lead molecules in each acquisition and their [...]

What were the billion-dollar large molecules in 2021? This article and infographic, which is part 4 of a Premium series covering 2021 drug discovery M&A, covers: The ten largest drug discovery biotech acquisitions by total consideration in 2021 where the acquiree’s lead was a large molecule The lead molecules in each acquisition and their [...]

This article and infographic highlights the drug discovery biotech acquisitions of 2021 where the upfront payment was disclosed, along with the structures of lead molecules in each acquisition where disclosed, mechanism of action, development stage, and indication. You can find Part I here [...]

We reviewed all of the biotech mergers and acquisitions announced in 2021 and did a deep dive on the list of biotech M&A transactions focused on drug discovery. The infographic below, which is part 1 of a Premium series covering 2021 drug discovery M&A, covers: The top ten overall small or large molecule drug acquisitions of 2021 The lead [...]

This article reviews the three novel drugs approved by the FDA in March 2022 in detail. together with Dr. Naveed Yasin, Jesse Keicher, Dr. Jennifer Huen, and Dr. Adrian Parodi Small molecules: Ztalmy ® ( ganaxolone ): An oral GABAA receptor positive allosteric modulator to treat seizures in cyclin-dependent kinase-like 5 deficiency disorder [...]

The FDA approved five novel small molecule drugs in neurology in 2021. Addressing a range of conditions from ADHD to relapsing MS, the 2021 neurology drug approvals are: atogepant (Qulipta): calcitonin gene-related peptide (CGRP) receptor antagonist ponsimod (Ponvory): selective sphingosine-1-phosphate (S1P) receptor 1 (S1PR1) modulator [...]

This article reviews the novel drugs approved by the FDA in February 2022. together with Dr. Naveed Yasin, Dr. Vinicius Texeira, and Dr. Jennifer Huen Three novel molecules were approved in February 2022: Small molecules: Pyrukynd (mitapivat) : An oral pyruvate kinase activator approved for the treatment of hemolytic anemia in adults with [...]

Merck’s oral, macrocyclic PCSK9 inhibitors are among the 12 candidates for 2021's "Molecule of the Year" and have already gotten quite a few votes for 2021’s “Molecule of the Year.” They’ve been called “gamechangers” by readers, with impressive human proof-of-concept. MK-0616 is a macrocyclic PCSK9 inhibitor with demonstrated once-daily [...]

together with Dr. Jennifer Huen, Saumil Patel, and Dr. Naveed Yasin In 2021, the FDA approved 9 non-radiological novel small molecule drugs for oncology. Excluding melphalan flufenamide, which has already been withdrawn, the remaining FDA-approved novel small molecule oncology drugs of 2021 are: umbralisib (PI3Kδ and CK1ε inhibitor) sotorasib [...]

In 2021 there were: 50 novel drugs approved by the FDA 27 that CDER considers first-in-class (FIC) 16 first-in-class small molecules (according to CDER) These drugs are the first in their therapeutic class to have a particular mechanism of action [...]

There were four novel drugs approved by the FDA in January 2022 (2 small molecules, 2 large molecules). The novel January 2022 new drug approvals appear below in this Drug Hunter minireview: See our overview of all 36 of the novel small molecule and large molecule FDA [...]

This deep dive into 2021’s novel large molecule drug approvals is the 2 nd in a series of focus articles on 2021’s novel large molecule drugs. See part one here . For each of the 16 large molecule drug approvals of 2021 (excluding vaccines), we’ll [...]

Antibody-drug conjugates (ADCs) have gone through several waves of enthusiasm since being first tested in animal models in the 1960’s. The first clinical trials with ADCs were conducted in the 1980’s, and an ADC was first approved in 2000 (Mylotarg, see below). Mylotarg, however, was famously withdrawn from the market first, before being [...]