Briquilimab

Today, a brief rundown of news involving Tenvie Therapeutics and Jasper Therapeutics, as well as updates from Sana Biotechnology, Stoke Therapeutics and City Therapeutics that you may have missed.A new San Francisco-area startup, Tenvie Therapeutics, launched on Wednesday with $200 million in funding. Tenvie is supported by Arch Venture Partners, F-Prime Capital and Mubadala Capital, and is built around drug assets from another Arch-backed company, Denali Therapeutics. Tony Estrada, who oversaw drug discovery work at Denali for the better part of a decade, is now leading Tenvie. The companys most advanced programs target two kinds of proteins tied to neurodegeneration. Jacob BellJasper Therapeutics on Wednesday shared data from a Phase 1b/2a study testing its antibody drug briquilimab in people with a type of hives known as chronic spontaneous urticaria. The company described the results as positive, showing treatment helped reduce hives severity and itchiness among study participants given one of six briquilimab doses. Jasper said treatment was well tolerated, but disclosed three people experienced neutropenia, including one whose adverse event was rated as medically significant. Investors appeared less impressed with the data, however, sending shares in the company down by more than half on Wednesday morning. Ned PagliaruloShares in Sana Biotechnology tripled in value Wednesday morning after the company said researchers using its technology successfully transplanted pancreatic islet cells into a person with Type 1 diabetes without using immunosuppression. According to Sana's CEO, this outcome is the first proof of an allogeneic transplant surviving without immune suppression or protection in an individual whose immune system is fully functioning. Researchers from Sweden's Uppsala University Hospital are conducting the study in partnership with Sana, which has developed a “hypoimmune“ technology that can generate cells able to evade detection by the body's defenses. Sana is preparing to run its own study of an allogeneic islet cell therapy it's developing in-house for Type 1 diabetes. Ned PagliaruloStoke Therapeutics has finalized plans for a Phase 3 study testing its experimental drug for Dravet syndrome, a severe developmental disease characterized by regular seizures. According to the company, it secured “alignment“ from regulators in the U.S., Europe and Japan to proceed with a one-year trial testing the effect of its drug on major motor seizure frequency. Stoke expects to enroll about 150 children and teenagers with Dravet in the trial, which it's dubbing Emperor. Data are expected by the end of 2027, the company said. Ned PagliaruloAndy Orth, a veteran of Krystal Biotech and Alnylam Pharmaceuticals, will become CEO of City Therapeutics, the RNA drug developer disclosed Tuesday. He joins three months after City launched publicly with $135 million in funding and plans to develop next-generation medicines that work by RNA interference, the Nobel Prize-winning technology pioneered by Alnylam. Orth was previously Krystal's chief commercial officer and, before that, led Alnylam's U.S. business, helping launch the company's first approved drug, Onpattro. Former Alnylam CEO John Maraganore will remain City's executive chair. Ned Pagliarulo '

REDWOOD CITY, CA, USA I Decemer 02, 2024 I Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a clinical stage biotechnology company focused on development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) to address mast cell driven diseases such as chronic spontaneous urticaria (CSU), chronic inducible urticaria (CIndU) and asthma, today announced that the first patient has been dosed in Jasper’s Phase 1b/2a clinical challenge study evaluating briquilimab in allergic asthma, called ETESIAN ( E valuating T he E fficacy and S afety of briquilimab I n particip AN ts with allergic asthma). The ETESIAN study is evaluating a single administration of subcutaneous briquilimab in patients with asthma. “Dosing of the first patient in our ETESIAN study in asthma is a significant milestone, marking our third clinical program evaluating briquilimab in an inflammatory disease driven by unwanted mast cell activity,” said Edwin Tucker, M.D., Chief Medical Officer of Jasper. “Following dose escalation through Part 2 of the BEACON study in CSU, we obtained regulatory clearance to move directly to a subcutaneous 180mg dose in the ETESIAN study, which we believe will drive deep mast cell depletion in the airways and enable durable clinical benefit for patients with asthma. We look forward to providing enrollment updates as we progress through the study and anticipate reporting the initial data in the second half of 2025.” The Phase 1b/2a ETESIAN study is a single dose double-blind, placebo-controlled challenge study that is expected to enroll approximately 30 patients across as many as 7 sites in Canada with a key objective of demonstrating proof-of-concept in asthma utilizing a potential therapeutic dose to inform future trials in the broader asthma population. The study will be conducted utilizing a single 180mg dose of subcutaneous briquilimab and key assessments will include both early and late asthmatic response, changes in airway hyperresponsiveness, mast cell depletion and recovery, and safety. “Depletion of mast cells via inhibition of c-Kit is a novel mechanism with the potential to alleviate asthmatic response in patients underserved by existing therapies,” said Paul O’Byrne, M.D., Professor, Dean and Vice President of the Faculty of Health Sciences at McMaster University. “As a potent and targeted c-Kit inhibitor, I believe briquilimab has the potential to overcome the safety issues that have limited development of other c-Kit inhibiting agents and, in turn, serve as an important treatment option for patients suffering from asthma. I look forward to enrolling patients into the ETESIAN study.” About Briquilimab Briquilimab is a targeted aglycosylated monoclonal antibody that blocks stem cell factor from binding to the cell-surface receptor c-Kit, also known as CD117, thereby inhibiting signaling through the receptor. This inhibition disrupts the critical survival signal, leading to the depletion of the mast cells via apoptosis which removes the underlying source of the inflammatory response in mast cell driven diseases such as chronic urticaria and allergic asthma. Jasper is currently conducting clinical studies of briquilimab as a treatment in patients with CSU, CIndU or asthma. To date, briquilimab has a demonstrated efficacy and safety profile in more than 160 dosed participants and healthy volunteers, with clinical outcomes in CIndU, and as a conditioning agent in severe combined immunodeficiency (SCID), acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), Fanconi anemia (FA), and sickle cell disease (SCD). About Jasper Jasper is a clinical-stage biotechnology company focused on developing briquilimab, a monoclonal antibody targeting c-Kit (CD117) as a therapeutic for chronic mast and stem cell diseases such as chronic urticaria and asthma. To date, briquilimab has a demonstrated efficacy and safety profile in more than 160 dosed participants and healthy volunteers, with clinical outcomes in CIndU and as a conditioning agent in SCID, AML, MDS, FA, and SCD. For more information, please visit us at www.jaspertx.com . SOURCE: Jasper