With four CRLs behind it and seven months ahead of its April 2023 PDUFA date, Mallinckrodt plc’s Terlivaz (terlipressin) has been approved by the U.S. FDA for treating hepatorenal syndrome (HRS-1). Terlivaz had a decade-long series of obstacles before getting the long-awaited approval. The vasopressin analogue selective for V1 receptors was approved, in part, on results from the phase III CONFIRM clinical trial of 300 patients, the largest-ever prospective study in assessing terlipressin in patients with HRS-1 in the U.S. and Canada. The study its primary endpoint of renal function improvement, avoidance of dialysis and short-term survival (p=0.012). The Dublin-based company’s stock (OTCMKTS:MNKPF) leapt at the news with shares trading 25% upwards at midday to $17.50 each.

Imugene announces AU$80M capital raise to build out immuno-oncology platform

Immuno-oncology company Imugene Ltd. announced an AU$80 million (US$53.9 million) capital raise that will allow the company to add additional studies within its immuno-oncology pipeline as well as pursue potential licensing opportunities. The funds raised will allow the company to interrogate its pipeline further in other indications or combinations, Imugene CEO Leslie Chong told BioWorld. “There’s a lot we could do with our current pipeline, and now our cashflow is greatly extended.” The placement consists of investments from two new institutional investors with significant biotech investment experience that are not being named, the CEO said.

E-nitiate Biopharma raises $14.4M to develop drugs for dermatosis

E-nitiate Biopharmaceuticals Co. Ltd. has raised ¥100 million (US$14.4 million) in a series A round to speed up the clinical trials of its lead assets, QY-201 and QY-101, and expand the pipeline. “Our strategy is to focus on the ‘blue ocean’ of the dermatosis market,” said Shi Jun, chief medical officer at E-nitiate Biopharma. “The first step is to enter China’s autoimmune skin diseases market.”

Investors scrutinize weight loss data from Altimmune’s fatty liver drug pemvidutide

Altimmune Inc. has come under scrutiny from investors after weight loss data from a phase Ib trial of its nonalcoholic fatty liver disease drug, pemvidutide, sent its shares on a roller coaster ride. It was always going to be hard to follow stellar data from Akero Inc. announced on Sept. 13 in the related disease nonalcoholic steatohepatitis (NASH) – where the fatty liver has become scarred and damaged. But investors questioned the latest data, despite the company’s upbeat assessment of the data from pemvidutide, a peptide-based GLP-1/glucagon dual receptor agonist in development for obesity and NASH. The company switched its focus to pemvidutide, licensed from South San Francisco-based Spitfire Pharma Inc. in 2019, after axing development of a vaccine and an immunotherapy for COVID-19 in June 2021.

Eloxx CF trial misses efficacy endpoint, shifting focus to Alport trial

After a midstage trial showed no benefit from adding ELX-02 (exaluren) to Kalydeco (ivacaftor, Vertex Pharmaceuticals Inc.) for certain cystic fibrosis (CF) patients, Eloxx Pharmaceuticals Inc. said it will discuss next steps for the program, its lead candidate, with the trial's sponsor, the CF Foundation. A proof-of-concept trial for EXL-02 in the rare kidney disease Alport syndrome will start later this year, the company said. But investor reaction to the phase II CF news was swift, sending company shares (NASDAQ:ELOX) 39.1% lower to 23 cents by midday.

Combination strategy enables brain-specific kinase inhibition

Using a two-drug combination, researchers at the University of California at San Francisco have been able to achieve brain-specific inhibition of several kinases. The team had previously demonstrated that a two-drug combination of mTOR-targeting drugs was effective in reducing the symptoms of alcohol use disorder. In their current paper, published in the Sept. 15, 2022, issue of Nature, they demonstrated that the same drug combination could extend survival in mouse models of glioblastoma while having minimal effects on mTOR activity in skeletal muscle.

Leadership needed in quest for fountain of youth

Geroscientists today called on U.S. lawmakers to modernize the regulatory approval path for new longevity treatments. After highlighting recent advances in biomarkers that could shorten clinical trials of such drugs, geroscientist Steve Horvath told members of a House Science, Space and Technology subcommittee, “We have an opportunity and, arguably, obligation to leverage recent biomedical breakthroughs to identify longevity interventions that may delay the onset of chronic diseases, and which may revolutionize the field of preventative medicine.” But to get there, Congress and others in government must recognize recent breakthroughs, including work on epigenetic clocks as potential biomarkers. Regulatory leadership is another must, he said.

The BioWorld Insider Podcast: $1B+ biopharma deals keep values afloat, even amid muted volume

BioWorld writers bring you the details of new R&D alliances, financings, IPOs and M&A every day. These stories are the heartbeat of the relationships between industry giants and their smaller, more innovative peers. But what's the top-line read for dealmaking these days? With nearly 1,000 biopharma deals completed through early August amid a volatile economic climate, there's a lot to talk about. Join the BioWorld Insider podcast as we talk with Senior Analyst Karen Carey, whose data-driven research delivers a detailed picture of the ups and downs.

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Adverum, Akero, Aldeyra, Alligator, Almirall, Alphamab, Alphyn, Altimmune, Anbogen, Aqualung, Ascendis, Aslan, Atea, Atma, Avrobio, Beyondspring, Biocytogen, Biolinerx, Biontech, Bryn, Caladrius, Carver, Centessa, Cidara, Edgewise, Effector, Fresh Tracks, Gameto, Genfit, Horizon, Ideaya, IMV, Larimar, Locus, MC2, Mindset, Minoryx, Nascent, NGM, Palisade, Pfizer, Qurient, Revive, Rhythm, Rigimmune, Sagimet, Sensorion, Spectrum, Syros, Tenax, Third Harmonic, Timber, Tiziana, Tracon, Transcenta, Triact, Triumvira, VBI