Intercept Pharmaceuticals, Inc.

Novo Nordisk will seek regulatory approval of semaglutide, the key ingredient in its popular weight loss medicine, in a common liver disease following positive results in a Phase 3 trial, the company said Friday.Summary results disclosed in a press release show that semaglutide met both of its main goals in the first part of a late-stage, placebo controlled study of people with metabolic dysfunction-associated steatohepatitis, or MASH.Specifically, Novo said that, after 72 weeks of follow-up, 37% of people who received semaglutide and standard therapy had an improvement in their level of liver scarring with no worsening of their condition, versus 22.5% of those who received a placebo and typical care. Additionally, 63% of people on semaglutide saw their MASH resolve without scarring getting worse, compared to 34% of placebo recipients.According to Novo, semaglutide displayed a safe and well-tolerated profile, consistent with whats been seen in previous testing.Novo didnt provide further study details, leaving it unclear how well semaglutide performed on a variety of secondary measures, or how many people mightve stopped treatment due to side effects. Detailed results will be presented at a medical meeting later in 2024. The trials second phase will also continue for several years to assess semaglutides ability to prevent cirrhosis, a common MASH complication.Nonetheless, the results position semaglutide to potentially become the first drug of its kind available for MASH, a liver condition estimated to affect millions of people and one of the leading causes of liver transplants. Novo intends to seek approvals of semaglutide in MASH in the U.S. and Europe in the first half of 2025.We are very pleased about the ESSENCE clinical trial results and the potential of semaglutide to help people living with MASH, said Martin Holst Lange, Novos head of development in a statement, referring to the name of the Phase 3 trial. Among people with overweight or obesity, one in three live with MASH. This has a serious impact on their health and represents a significant unmet need.MASH is triggered by a toxic accumulation of fat in the liver and typically develops alongside other metabolic diseases like diabetes. Its long been a top target of drugmakers, but a difficult one to hit. Several promising medicines have failed in testing over the years, and the first to reach U.S. regulators a drug from Intercept Pharmaceuticals was rejected in 2023.A newer group of medicines has followed. Madrigal Pharmaceuticals Rezdiffra in March became the first approved drug for the condition and has since gotten off to a faster-than-expected launch. Other medicines from Akero Therapeutics and 89bio have shown some promise, too, though study results have been less clear.Now so-called GLP-1 drugs, like semaglutide, have emerged as a potential treatment option. These medications act on gut hormones and are proven to have powerful effects on weight loss, heart health and other conditions, and MASH has been seen as one of their next opportunities. Eli Lilly and partners Boehringer Ingelheim and Zealand Pharma have already reported encouraging findings in Phase 2 trials, sparking speculation from investors that GLP-1 drugs might upend the market for other MASH therapies.Novo hasnt yet convinced some Wall Street analysts that that will happen, however. Multiple issued notes on Friday arguing the findings dont clearly separate semaglutide from other MASH drugs, leaving room for other competitors. The results confirm that GLP-1 drugs will have utility in addressing some aspects of MASH, but arent a silver bullet or functional cure, wrote Leerink Partners Thomas Smith.The magnitude of the semaglutides placebo-adjusted effects on fibrosis are similar to Rezdiffra, added William Blair analyst Andy Hsieh. RBC Capital Markets Brian Abrahams noted that semaglutides effect size also wasnt as large as whats been seen in testing of 89bio and Akeros drugs, though there are caveats in comparing medicines across trials.We believe this outcome removes a potential overhang/worst-case scenario where semaglutide showed game-changingfibrosis improvement, wrote Leerinks Smith, who believes MASH will become a polypharmacy opportunity with room for multiple liver-directed mechanisms and treatment approaches.Shares of Madrigal climbed nearly 15% Friday morning. 89bio and Akero shares also ticked up in early trading. '

Seaport will use the $225m funding to advance its pipeline of experimental drugs for neuropsychiatric disorders. Credit: eamesBot via Shutterstock. PureTech-founded Seaport Therapeutics has raised $225m in a Series B financing round to support the development of new prodrug treatments for psychiatric disorders. Seaport plans to use the new funds to push forward its pipeline of experimental drugs, including its most advanced candidate, SPT-300, which is being advanced into a Phase IIb trial for major depressive disorder. SPT-300 is an oral prodrug of brexanolone, a neurosteroid compound with known anti-depressant effects. Topline results from Seaport’s SPT-300 Phase IIa proof-of-concept trial were reported in November 2023, where the study met its primary endpoint of salivary cortisol response. The financing was led by General Atlantic, with backing from several other major investors such as T Rowe Price, Foresite Capital, and Goldman Sachs . The latest funding comes after the company’s launch in April 2024, when it secured $100m in initial funding from founding investors ARCH Venture Partners, Sofinnova Investments, Third Rock Ventures, and co-founder Puretech Health . The funding will also support Seaport’s Glyph technology platform, which helps drugs be absorbed more efficiently by using the body’s lymphatic system. This platform could improve the delivery of drugs that have a low bioavailability, or have side effects such as liver enzyme elevations or hepatoxicity, says the company. See Also: FDA approves Astellas’ VYLOY for gastric cancer treatment Intercept’s liver disease drug Ocaliva faces FDA approval delay The company has several other candidates in its pipeline, including SPT-320, an experimental prodrug of agomelatine being advanced into Phase I studies to treat generalised anxiety disorder (GAD). By leveraging Glyph, SPT-320 bypasses liver first-pass metabolism, allowing for lower dosing and reduced liver exposure. Seaport is also developing SPT-348 for mood and neuropsychiatric disorders and has several preclinical programmes under its belt. Prodrugs are medications that start as inactive or less active, and once inside the body, are converted into the active form, usually through natural processes involving enzymes. Prodrugs can be used to enhance drug delivery, minimise side effects, and improve drug solubility and oral bioavailability. In the announcement accompanying the financing, Seaport’s founder Steve Paul said: “The development of important new neuropsychiatric medicines is often halted due to poor drug-like properties or unacceptable tolerability, challenges that our Glyph platform can now uniquely address.” The potential of prodrugs was recently in the spotlight in the case of xanomeline, which initially faced tolerability challenges, but once resolved, was approved Cobenfy (formerly KarXT) for schizophrenia. Xanomeline helps reduce symptoms of schizophrenia, but it can cause unpleasant side effects that include nausea and sweating. Trospium was added to the drug to block these side effects while still allowing xanomeline to work in the brain. Now, Cobenfy is expected to earn $2.99bn in revenues by 2030, as per GlobalData analysis. GlobalData is the parent company of Pharmaceutical Technology. New York-based Terran Biosciences is taking it a step further by developing two prodrug versions of Cobenfy. Terran aims to curb issues surrounding medication adherence by optimising its dosing schedule to a more convenient once-daily oral regimen (TerXT), and a multi-month injectable (TerXT LAI).

The Ion Torrent Oncomine Dx Target Test is the first FDA-approved next generation sequencing-based diagnostic. Credits: JHVEPhoto / Shutterstock. Thermo Fisher Scientific has announced that its in vitro diagnostic test, Oncomine, has won US Food and Drug Administration (FDA) approval for use as a companion diagnostic in grade 2 IDH-mutant glioma. As per the 21 October press announcement, Oncomine is now approved to be used to identify patients who are eligible for treatment with Servier Pharmaceuticals’ Voranigo (vorasidenib) tablets. Voranigo, which gained FDA approval in August 2024, is the first and only targeted therapy available for patients with grade 2 IDH-mutant glioma. The French drugmaker gained control over the asset in 2020 after purchasing Agios’ oncology pipeline for $1.8bn in 2020. Agios has since sold its royalty stake in Voranigo to Royalty Pharma for $905m. Under the terms of the agreement, Royalty Pharma will gain a 12% royalty on annual US sales up to $1bn and a 12% royalty on net US sales over $1bn. As a once-daily oral inhibitor of IDH1 and IDH2 enzymes, Voranigo is indicated for use in grade 2 IDH-mutant glioma patients following surgery, including biopsy or gross total resection. The Ion Torrent Oncomine Dx Target Test is the first FDA-approved sequencing-based diagnostic. Oncomine received its first approval as a companion diagnostic in 2017 and subsequently gained approval in August 2022 as a companion diagnostic for identifying non-small cell lung cancer (NSCLC) patients eligible for treatment with Daiichi Sankyo ’s Enhertu (fam-trastuzumab deruxtecan-nxki). See Also: FDA approves Astellas’ VYLOY for gastric cancer treatment Intercept’s liver disease drug Ocaliva faces FDA approval delay Oncomine is also approved for use in cholangiocarcinoma (CCA), medullary thyroid cancer (MTC) and thyroid cancer (TC). Daiichi and AstraZeneca ’s Enhertu has seen several wins this year. The drug gained conditional approval in China earlier this month and a breakthrough therapy designation for a specific type of breast cancer in August 2024. Additionally, in April, the treatment was granted accelerated approval for the treatment of unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-positive [immunohistochemistry (IHC) 3+] solid tumours. According to GlobalData’s consensus forecasts, Voranigo is expected to generate sales of $37m in 2030. Enhertu is projected to generate total sales of $13bn in 2030. GlobalData is the parent company of Pharmaceutical Technology .