Gene therapy developer Kate Therapeutics Inc. (KateTx), which is developing next-generation adeno-associated virus (AAV) vectors that target skeletal and cardiac muscle, has unveiled a $51 million series A round and a licensing deal with Astellas Pharma Inc. The company is building on the work of three scientific co-founders: Sharif Tabebordbar, who is also its chief scientific officer; Eric Wang, of the University of Florida; and Alan Beggs, of Boston Children’s Hospital. It is building on their respective capabilities in capsid engineering, researching the molecular pathology of myotonic dystrophy and other microsatellite repeat diseases, and probing the genetics of myopathies, as well as developing appropriate animal models of these conditions. It has disclosed two in-house programs, focused on myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy. The pact with Astellas concerns KT-430, an AAV-based gene therapy for X-linked myotubular myopathy.

Moving Upstream with $200M series B and TSLP-targeting asthma drug

Less than two years ago, in October 2021, a group of investors bid at auction on a promising monoclonal antibody with the potential to treat asthma, chronic rhinosinusitis and other inflammatory conditions, winning the asset known as UPB-101, the sole focus of the then-newly launched Upstream Bio Inc. The purchase, paid for by $200 million from a series A led by Orbimed and Maruho Co. Ltd., was made from Astellas Pharma Inc., which had decided to exit inflammation and was in the process of divesting assets. The Japanese company already had clinical data on the drug from a phase Ia single ascending-dose study in healthy volunteers. Since then, Upstream has brought the product into an ongoing, completely enrolled, phase Ib trial in 32 asthma patients, and announced the raising of a $200 million series B on June 8, a round that should bring it well beyond phase II readouts and into 2026. “UPB-101 targets TSLP [thymic stromal lymphoprotein] biology, which is upstream of many other targets,” Upstream CEO Samantha Truex told BioWorld.

Ultimovacs misses primary endpoint in phase II mesothelioma study

Following strong phase I study data released a year ago, Ultimovacs ASA’s lead candidate stumbled in the phase II, which did not meet the primary endpoint of progression-free survival (PFS) in second-line treatment of malignant pleural mesothelioma. The randomized, open-label, multicenter study saw 118 patients treated with UV-1 plus checkpoint inhibitors Keytruda (pembrolizumab, Merck & Co. Inc.) and Opdivo (nivolumab, Bristol Myers Squibb Co.) to see if there was a benefit compared to just treating with the checkpoint inhibitors. While missing the endpoint, the study did show a statistically significant positive PFS benefit for the those in the UV-1 arm. The company’s shares (OSE:ULTI) had a rough day on June 8, closing 29.5% lower at NOK82.40 (US$7.55).

Long-acting RSV prophylactic on adcom plate

After hearing from both Astrazeneca plc and the FDA this morning about the safety and efficacy data for nirsevimab as a respiratory syncytial virus (RSV) prophylactic for infants, the U.S. FDA’s Antimicrobial Drugs Advisory Committee is set to discuss the ramifications of using the long-acting RSV F protein inhibitor in infants whose mothers received a maternal RSV vaccine. If approved, nirsevimab will provide a one-dose alternative to Astrazeneca’s older RSV prevention drug, Synagis (palivizumab), which is marketed in the U.S. by Swedish Orphan Biovitrum AB under a 2018 deal. Synagis is dosed once a month throughout the RSV season.

China Medical System obtains second approval in 2023 for psoriasis drug

China’s NMPA granted a green light to China Medical System Holdings Ltd.’s tildrakizumab injection to treat adults with moderate to severe plaque psoriasis who are candidates for systemic therapy or phototherapy. Sold under the brand name Ilumetri, tildrakizumab is a humanized lgG1/κ monoclonal antibody designed to selectively bind to the p19 subunit of interleukin-23 (IL-23) and inhibit its interaction with the IL-23 receptor to block the release of pro-inflammatory cytokines and chemokines.

The BioWorld Insider Podcast: Preventing opioid overdoses with a smart patch

Free, no-prescription-required access to naloxone has reduced deaths from opioid overdoses by 11% in some communities, but they only work if someone with the reversal agent is available when a person overdoses. Resilient Lifescience Inc. hopes to save those lives, too. The company is developing a wearable patch that monitors vital signs and automatically administers naloxone when needed. In this edition of the BioWorld Insider podcast, Brad Holden, CEO of Resilient Lifescience, explained how the device works for overdoses as well as other potential applications for the new technology.

Also in the news

Akoya, Ascentage, Baudax, Beyondspring, Bioinvent, Capricor, Coya, Dtx, Durect, Emergex, Fibrogen, Fluoguide, Immvira, Itabmed, Janssen, Kite, Lianbio, Lixte, Med-Life Discoveries, Merz, Nascent, Ocular Therapeutix, Oncternal, Onquality, Opthea, Paxmedica, Replimune, Rigel, Sumitomo, Teva, Ventyx, Vera, Vistagen