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New real-world results for the treatment of hospitalized patients with esophageal variceal hemorrhage (EVH) and liver cirrhosis treated with terlipressin from Mallinckrodt plc look promising following treatment of nearly 200 patients in a U.K. hospital. Terlipressin is a potent vasopressin analog selective for V1 receptors. Although not approved in the United States, terlipressin is approved in many countries where it has been a standard of care for decades in the treatment for hepatorenal syndrome type 1 (HRS-1). In a real world test in the United Kingdom where terlipressin is approved, 195 patients EVH and liver cirrhosis patients were treated with the medication to determine bleeding control. The primary clinical outcome was control of bleeding within 36 hours to five days. Data from the study showed that 59.5% of patients (116 of 195) were able to see their bleeding controlled within that time. Of the patients who had an initial bleed control in 36 hours, rebleeding was observed in 40.5% within three days, 43.1% within five days and 45.7% within seven days. For those patients who saw their bleeding controlled by day 5, rebleeding was observed in 41.4% within three days, 43.2% within five days and 43.2% within seven days of the initial bleed control. Mallinckrodt noted that the study had limitations due to the retrospective nature of the study design. Data collected was limited to the information available and extracted from patient medical charts, the company added. Kevin Moore, lead investigator and principal clinical research fellow at the UCL Institute of Liver and Digestive Health at Royal Free Hospital at the University College London, said it is “critically important to expand our knowledge of evidence-based medicine for EVH, and investigational therapies like terlipressin.” Understanding the nature of the drug and its effect on these patients can help improve their outcome, Moore said. Elsewhere around the globe: Elicera Therapeutics – Sweden’s Elicera announced preclinical proof-of-concept data for its iTANK (immunoTherapies Activated with NAP for efficient Killing) platform that was developed to optimize the effect of CAR T-cell therapies. It does so by generating a dual mechanism-of-action. According to the company, in addition to the effect of CAR T-cells, CD8 + killer T-cells are activated, which gives a double attack on the tumor cells and creates a systemic and long-term immune response against cancer. Through the iTANK platform, Elicera hopes to address two current challenges CAR-T therapies face with solid tumors: the hostile tumor microenvironment inhibits the effects of CAR-T cells and there are difficulties in identifying a target antigen on solid tumors that is expressed on all tumor cells. RedHill Biopharma – Based in Israel and in North Carolina, RedHill presented four analyses from its Phase III Talicia and Movantik data at the ACG annual meeting. The first Talicia analysis evaluated physician-directed therapy data and confirmed high utilization of clarithromycin and low rates of H. pylori eradication. Utilization data based on U.S. prescriptions confirmed that clarithromycin-based regimens remain predominant, even though the majority of Americans have previously been prescribed a macrolide antibiotic. The authors conclude that since Talicia does not contain clarithromycin, and resistance to amoxicillin and rifabutin is very rare, Talicia can be used first-line with minimal concern for antibiotic resistance or CYP2C19 metabolism, the company said. The Movantik posters provided details on data from patients experiencing extreme opioid-induced constipation (OIC) with severe symptoms. Dose levels of 12.5 mg and 25 mg provided rapid onset with early symptom relief and consistent, predictable and sustained efficacy, the company said. Kadimastem – Also based in Israel, Kadimastem secured $5 million in private placement funds. The placement was led by Clover Wolf Capital and Alpha Capital Anstalt. Controlling shareholders Michel Revel and Julien Ruggieri also provided funding. The investment will be used to support Kadimastem’s future clinical trial activity and fund multiple strategic collaborations involving both the company’s ALS and Diabetes programs. Horizon Therapeutics –Ireland’s Horizon announced positive topline results from the MIRROR study showing a significant increase in efficacy using Krystexxa with the immunomodulator methotrexate as compared to the response rate of Krystexxa and placebo in patients with uncontrolled gout. Results show that 71% of patients who received Krystexxa with methotrexate achieved a complete and durable response compared to 40% of patients who received Krystexxa with placebo. No new safety concerns were identified, the company said. Cardiol Therapeutics – Based in Ontario, Cardiol received approval from Health Canada to move CardiolRx into Phase II. The trial will assess the safety and tolerability of CardiolRx as well as its impact on myocardial recovery in patients presenting with acute myocarditis. The approval follows the U.S. Food and Drug Administration (FDA) providing clearance to proceed with the company’s Investigational New Drug (IND) application to initiate this trial. Myocarditis is an acute inflammatory condition of the myocardium, characterized by inflammation of the heart muscle, which may result in chest pain, impaired cardiac function, atrial and ventricular arrhythmias, and conduction disturbances. Cyxone – Austria-based Cyxone and Medical University of Vienna continue to investigate drug candidate T20K in combination with a kappa opioid receptor agonist. In pre-clinical experiments, the company said T20K demonstrates “promising synergistic therapeutic effects and potential disease modifying features in multiple sclerosis.” The data strengthens the evidence that T20K acts as a potent ligand to the kappa opioid receptor. The receptor has been identified to play an important role in remyelination of neurons and to be involved in immunosuppressive and anti-inflammatory activities, making it a suitable drug target in the treatment of early and late stages of MS, the company said. Gedea Biotech – Sweden-based women’s health company Gedea Biotech secured a patent from the European Patent Office that covers the treatment of fungal infections with compound glucono-δ-lactone (GDL). Queen’s University – Queen’s University Belfast scientists have developed a computer-aided data tool that could improve treatment for a range of illnesses. The computer modeling tool will predict novel sites of binding for potential drugs that are more selective, leading to more effective drug targeting, increasing therapeutic efficacy and reducing side effects. According to the university, the data tool or protocol will uncover a novel class of compounds – allosteric drugs in G protein-coupled receptors (GPCRs). Agomab Therapeutics – Belgium-based Agomab will acquire Spain’s Origo Biopharma SL, a company developing organ-restricted small molecule drug candidates targeting the transforming growth factor beta (TGF-β) pathway for the treatment of fibrosis-related disorders. The combined organization will focus on translating growth factor signaling pathways into innovative therapies. Agomab’s hepatocyte growth factor (HGF)-targeting monoclonal antibodies together with Origo’s small molecule programs create a rich clinical pipeline of therapeutics that address fibrosis and organ failure in multiple therapeutic areas.

Sesen Bio said that it was not expecting a CRL from the FDA for its bladder cancer candidate Vicineum. But the rejected filing included thousands of violations and investigator misconduct, STAT News reported Wednesday. Thomas Cannell The 130-patient study had more than 2,000 violations, 215 of which were classified as major, STAT said. Independent monitors also reported three investigators to the FDA for a “serious noncompliance” that “placed subjects at risk of harm.” A patient died in 2016 of liver failure related to the drug, and two years later, the company said that there were no drug-related deaths at a urology conference. The company saw its stock crash, as it entered a halt at $6 per share last Friday and emerged battered with a $0.86 price. In June CEO Thomas Cannell said that the drug was on its way to generating between $1 billion and $3 billion. Then Monday, he blamed “toxic” and critical media coverage of the FDA’s approval of Biogen’s Alzheimer’s treatment Aduhelm for the CRL. — Josh Sullivan Bristol Myers picks up development of AI outfit’s immunology drug Rupert Vessey Bristol Myers Squibb has opted to pick up the development of an immune-modulating drug as part of a research collaboration with UK-based AI discovery outfit Exscientia, the partners said Wednesday. Exscientia will receive a $20 million milestone payment as part of the pick up with additional milestones and tiered royalties possible. Details on the drug were scarce, but the partners said it targets “a critical immunological kinase.” “We are pleased to in-license our first drug candidate resulting from our strategic collaboration with Exscientia,” Rupert Vessey, Bristol Myers’ president of early R&D said in a release. “Artificial intelligence and machine learning continue to play important roles in drug discovery and Exscientia has delivered a promising development candidate in the field of immunology. We look forward to our continued collaboration and further advancing this candidate for the potential benefit of patients with unmet medical need.” — Kyle Blankenship Werewolf hops aboard the Keytruda train with Merck trial collaboration Werewolf Therapeutics and Merck are teaming up for a new clinical trial. Randi Isaacs Looking at Werewolf’s WTX-124 candidate targeting IL-2, the pair will collaborate on a Phase I study evaluating the program’s safety and efficacy in solid tumors, the companies announced Wednesday. Researchers will examine WTX-124 both as a monotherapy and in combination with Merck’s Keytruda. “The clinical benefit of targeting IL-2 as a treatment for cancer has long been established; however, its utility has been limited by challenging toxicities,” Werewolf CMO Randi Isaacs said in a statement. “We believe WTX-124 has the potential to enhance therapeutic options for cancer patients as a monotherapy and when combined with checkpoint inhibitors like Keytruda.” Werewolf says it plans to submit an IND for the WTX-124 program in the first half of 2022. Should the IND be cleared, the Phase I study will start “promptly,” though no timeline was specified. — Max Gelman Penny stock sees shares jump at Phase II topline data Penny stock player AzurRx got a significant bump Wednesday after it reported topline data for a Phase II study. James Sapirstein Evaluating the company’s MS1819 program for the treatment of severe exocrine pancreatic insufficiency in patients with cystic fibrosis, AzurRx said the candidate in combination with standard of care resulted in an average 6.57-percentage point gain from baseline in the Coefficient of Fat Absorption, which the company says is a clinically meaningful result. Previous literature had said anything above a five-percentage point improvement is clinically meaningful. Data from Wednesday’s results come from 20 patients. “Topline data clearly show that combining MS1819 to the daily dose of PERT [standard of care] had clinical benefits for all patients and improved quality of life,” CEO James Sapirstein said in a statement. “Our next step with the MS1819 program is to finalize development of a new enteric-coated microbead formulation, which we believe will enable more of the medication to reach the small intestine thereby enhancing its therapeutic potential.” AzurRx shares $AZRX skyrocketed 46% in pre-market trading Wednesday, settling in the 35% to 40% range after the opening bell. The formulation work for the new enteric-coated microbead tech is expected to be completed by the end of the year. AzurRx plans to launch a bridging study in 2022 evaluating the formulation as a monotherapy. — Max Gelman SomaLogic does the Twist Emily Leproust SomaLogic and Twist Bioscience have agreed to a new collaboration, announced Wednesday morning. The deal will allow Twist access to SomaLogic’s platform to discover new antibodies and drug targets. No financial terms were disclosed for the deal. “We intend to use the antibodies discovered for our internal pipeline, moving the most promising candidates through preclinical development and then potentially outlicensing to a partner,” Twist CEO Emily Leproust said in a statement. — Max Gelman Biotech developing anti-inflammatory COPD treatment lands €20M Series A An Icelandic biotech that focuses on treating chronic obstructive pulmonary disease has secured a European Series A financing round, the company announced Wednesday. Maria Bech EpiEndo raised has €20 million, or $23.4 million. The round was led by Flerie Invest and Iðunn Venture Fund. Existing investor ABC Ventures participated, as well as the European Innovation Council Fund. Funds will go toward moving EP395 through clinical trials through Phase IIa. The drug entered Phase I trials in April. “This financing is a major step for EpiEndo, not only funding our lead compound through clinical development in patients with COPD, but also enabling exploration of other important potential therapeutic application areas such as dermatology and gastrointestinal disorders, where compromised epithelial integrity is known to contribute to disease pathophysiology,” CEO Maria Bech said in a press release. Scientists seek to make EP395 the first disease modifying non-antibiotic macrolide that can be used as a long-term treatment for COPD. It works by reducing inflammation in the epithelial barrier, and in the future, the company will try the candidate on patients with asthma, bronchiectasis and diffuse pan-bronchiolitis. — Josh Sullivan