Nonalcoholic Steatohepatitis

KNOXVILLE, Tenn.--( BUSINESS WIRE )--On January 16, 2025, Neuraly Inc. (Neuraly), a wholly owned subsidiary of D&D Pharmatech Inc., and Enigma Biomedical USA, Inc. (Enigma) announced the signing of a research license and commercialization option agreement for PMI04, a PET (Positron Emission Tomography) imaging biomarker of neuroinflammation developed by Neuraly. Under the agreement, Enigma acquires an exclusive research license for PMI04, as well as the option to negotiate commercialization rights based on the research results. PMI04 is a PET imaging biomarker that selectively binds to CSF-1R (Colony Stimulating Factor 1 Receptor) expressed in activated microglia. It enables visualization of the pathological activity of microglia, the fundamental contributors to neuroinflammatory responses, aiding in the diagnosis of various neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, and multiple sclerosis. Enigma plans to focus its development efforts on utilizing PMI04 for the assessment of neurodegenerative diseases. Currently, PET imaging targeting TSPO (Translocator Protein) is used in neuro research, but exhibits limited specificity, leading to challenging interpretation and quantitative assessment. In contrast, CSF-1R specifically binds to activated microglia, directly correlating with microglial proliferation and survival. This makes PMI04 a next-generation PET imaging biomarker capable of quantifying assessment, early detection, and pathological analysis of inflammation-related neurodegenerative diseases, which may ultimately benefit the development of therapeutics. With successful conclusion of the research, a commercialization license agreement will be established between the two companies, granting Enigma exclusive development and commercialization rights for PMI04. Neuraly will receive an upfront payment and milestone payments based on development and commercialization progress, along with sales royalties upon the commercial sales in the future. Seulki Lee, CEO of Neuraly stated "We hope this partnership will represent a significant step forward in providing alternatives for the diagnosis and treatment of neurodegenerative diseases. We will do our utmost to ensure that PMI04 is commercialized swiftly through close collaboration with Enigma." “We are delighted to have been selected as a development partner by Neuraly,” said Rick Hiatt, President and CEO of Enigma. “We will build on demonstrated successes with the best-in-class neuroimaging biomarkers MK-6240 (Cerveau Technologies) and NAV-4694 (Meilleur Technologies). We believe the Neuraly CSF-1R PET imaging biomarker has unique properties and will prove useful in developing current and future therapeutics for neurodegenerative disease. Our commitment is to expand the availability of this novel investigational imaging agent to the broader scientific community.” About Enigma Biomedical USA, Inc. Enigma Biomedical USA ’s vision is to be the premier provider of imaging biomarkers for neurological pathologies, associated information technology, and related tools to accelerate the development, approval, and adoption of effective therapies to treat neurodegenerative diseases. Enigma’s neuroimaging biomarkers provide Pharma and Academic researchers with state-of-the-art tools for enabling Disease-Modifying Therapy development with the highest precision and accuracy. In pursuit of this vision, subsidiaries of Enigma have provided the best-in-class Tau and Amyloid PET imaging biomarkers, MK-6240 and NAV-4694, to our partners to enable their research efforts. Both tracers have recently been acquired by a third party for further development. Enigma also recently announced a partnership with AbbVie to explore their novel 4R Tau PET Imaging Biomarkers. About D&D Pharmatech, Inc. and Neuraly Inc. D&D Pharmatech Inc. (D&D) , listed on the KOSDAQ in Korea, specializes in the development of GLP-1 receptor agonists. The company has established a long-term partnership with the Johns Hopkins University School of Medicine to develop imaging biomarkers related to neurodegenerative diseases. Additionally, D&D is conducting clinical trials for GLP-1 candidates in various indications, including a completion of a Phase 2 trial in the U.S. for the weekly injectable GLP-1 receptor agonist pegsebrenatide (NLY01) in Parkinson’s disease patients in 2023. Neuraly Inc. (Neuraly) , D&D's wholly-owned subsidiary located in Gaithersburg, Maryland, USA, specializes in IND-enabling studies and clinical development for D&D’s programs targeting neurodegenerative, metabolic, and fibrotic diseases. Neuraly has successfully cleared multiple INDs and conducted five clinical trials to date, including four completed studies and an ongoing Phase 2 trial of DD01 for metabolic dysfunction-associated steatohepatitis (MASH).

GSK is looking for hints of causality in Mendelian genetics—or patterns in biological inheritance—that cause significant disease early in life.\n Following GSK’s deal-heavy end to 2024, Chief Scientific Officer Tony Wood, Ph.D., doesn’t expect the trend to let up in the new year, zeroing in on partnerships that can expand knowledge about genetic factors driving disease. And, while Wood said the pharma still isn’t interested in joining the overcrowded obesity landscape, GSK is considering ways to build past the GLP-1 space.  GSK started this year’s J.P. Morgan Healthcare Conference by acquiring precision biotech IDRx, including its phase 3-ready gastrointestinal cancer candidate, for $1 billion.“Obviously, we have a growing interest in in rectal, colorectal and GI cancers,” Wood told Fierce Biotech on Monday at the conference, adding that IDRx has all the characteristics he and Chief Commercial Officer Luke Miels look for in business deals.“It\'s got an interesting clinical profile that could be accelerated,” Wood said. “That profile is anchored in something you can explain from the early characteristics of the molecule and strategically aligned with our growing interests in solid tumors associated with the GI tract.”The deal follows a string of partnerships penned over the last few months, including a $35 million upfront Alzheimer’s pact with Danish biotech Muna Therapeutics, a deal involving Rgenta’s RNA-targeting discovery platform, an antibody-drug conjugate deal with Duality, a $45 million upfront Relation-ship for fibrotic diseases and osteoarthritis, and a multi-target neurodegenerative pact with Vesalius Therapeutics.And Wood doesn’t expect the deals to slow down anytime soon. But what does the London-based Big Pharma look for in potential partners? The most optimal deals are “all about target finding” and “matching targets to patients,” Wood said.GSK has invested heavily in large-scale data sets for genetics, Wood explained, adding that the focus was a priority for former GSK CSO Hal Barron, M.D., who headed over to Altos Labs in 2022.Leveraging those deals and data, GSK hunts for signs of causality in Mendelian genetics—or patterns in biological inheritance—that cause significant disease early in life.The partnerships help the Big Pharma piece together what kind of impact particular pathways or forms of pharmacology can have, Wood said, providing GSK-4532990 in alcoholic liver disease (ALD) as a prime example.Back in 2021, GSK picked up the investigational siRNA oligonucleotide via a partnership with Arrowhead. The candidate targets HSD17B13, a gene that is involved in lipid metabolism and is mainly expressed in the liver.In a phase 1 study, the asset demonstrated a “durable reduction in HSD17B13 expression and reductions in key marker of liver injury,” according to a Dec. 17 event (PDF) about GSK’s early pipeline.Human studies show that naturally occurring gene variants protect against both alcohol-related and nonalcohol-related liver disease, GSK said, cutting risk by about 30% to 50% for carriers compared to those without.“This is one of the strongest levels of genetic validation we’ve seen for any target,” the Big Pharma said. “And, reassuringly, this protective effect is maintained even in the case of continued alcohol consumption.” “So, we\'ve got this lovely example of the genetics line up, [and] the targets line up in the cells that are showing the disease,” Wood told Fierce.That gives GSK confidence in the candidate, with a pair of phase 2 studies assessing the siRNA’s efficacy in both metabolic dysfunction-associated steatohepatitis patients with advanced fibrosis and patients with ALD.The program is emblematic of GSK’s plans for obesity—that is to say, Wood still doesn’t intend on moving directly into the heavily populated landscape, but that doesn’t mean the company isn’t touching obesity-related conditions.“It\'s very crowded, and I\'ve got an enormously exciting portfolio in front of me already,” Wood said, adding that he’s instead eyeing the later stages of diseases driven by inflammation.The Big Pharma is considering what comes after GLP-1s and what could be used with GLP-1s across lung, liver and kidney indications, he explained.“We\'re deliberately targeting mechanisms that are going to be at that more fibrotic end of the spectrum,” Wood said. “A lot of our thinking in that area is really about what remaining risk persists after you\'ve dealt with the problems of weight reduction.” 

– Completed enrollment in Phase 3 ENTRUST trial in patients with severe hypertriglyceridemia (SHTG); topline 26-week data expected in the second half of 2025 – – The Phase 3 ENLIGHTEN program in patients with non-cirrhotic (F2-F3) and compensated cirrhotic (F4) metabolic dysfunction-associated steatohepatitis (MASH) continues to enroll patients across both trials – – Cash, cash equivalents, and marketable securities totaled approximately $440 million as of December 31, 2024 – SAN FRANCISCO, Jan. 13, 2025 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today provided a corporate update and business outlook for 2025. “Looking ahead to 2025, we are well-positioned to build on the momentum from the initiation and ongoing enrollment of our ENLIGHTEN Phase 3 program in MASH and the successful completion of enrollment in our ENTRUST Phase 3 trial in SHTG and expect to announce topline data from the Company’s first Phase 3 trial in the second half of 2025,” said Rohan Palekar, CEO of 89bio. “We remain confident in pegozafermin’s potential as a potent anti-fibrotic agent with broad cardio-metabolic benefits. With a strengthened leadership team, commercial-scale manufacturing available, and a bolstered financial position, we continue to advance our Phase 3 programs toward potential Biologics License Application (BLA) and Marketing Authorization Application (MAA) filings, aiming to transform care for patients living with MASH and SHTG.” Key 2024 Highlights Completed enrollment in ENTRUST, the Phase 3 trial evaluating the efficacy, safety and tolerability of pegozafermin in patients with SHTG. 89bio expects to report topline 26-week data from this trial in the second half of 2025.Initiated ENLIGHTEN-Fibrosis and ENLIGHTEN-Cirrhosis, the Phase 3 trials in non-cirrhotic (F2-F3) MASH patients with fibrosis and in compensated cirrhotic (F4) MASH patients, in March and May of 2024, respectively. Both MASH studies continue to enroll patients globally.Obtained regulatory feedback from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) on clinical as well as Chemistry, Manufacturing, and Controls (CMC) requirements for marketing authorization filings for pegozafermin. The Company remains on track for potential BLA and MAA filings, pending positive clinical data.Bolstered its balance sheet through the completed equity follow-on offering for $143.8 million in gross proceeds and an amended credit facility with K2 HealthVentures, providing an aggregate principal of up to $150.0 million. Cash, cash equivalents, and marketable securities totaled approximately $440 million as of December 31, 2024 (preliminary unaudited).Strengthened its executive leadership team and Board of Directors with four seasoned industry veterans. Appointments include Teresa Perney, Ph.D., Chief Regulatory and Quality Officer; Francis Sarena, Chief Operating Officer; Charles McWherter, Ph.D., Board of Directors; and Martin Babler, Board of Directors. These additions bring extensive operational, clinical, regulatory, and commercial expertise to 89bio. About 89bio 89bio is a clinical-stage biopharmaceutical company dedicated to the development of best-in-class therapies for patients with liver and cardiometabolic diseases who lack optimal treatment options. The company is focused on rapidly advancing its lead candidate, pegozafermin, through Phase 3 clinical development for the treatment of metabolic dysfunction-associated steatohepatitis (MASH) and severe hypertriglyceridemia (SHTG). Pegozafermin is a specifically engineered, potentially best-in-class fibroblast growth factor 21 (FGF21) analog with unique glycoPEGylated technology that optimizes biological activity through an extended half-life. The company is headquartered in San Francisco. For more information, visit www.89bio.com or follow the company on LinkedIn. Forward-looking StatementsCertain statements in this press release may constitute "forward-looking statements" within the meaning of the federal securities laws, including, but not limited to, statements regarding the therapeutic potential and utility, efficacy and clinical benefits of pegozafermin, the safety and tolerability profile of pegozafermin, trial designs, clinical development plans and timing for pegozafermin, including the topline results from the ENTRUST Phase 3 trial in SHTG and enrollment in clinical trials, including enrollment of the Phase 3 ENLIGHTEN-Fibrosis trial and Phase 3 ENLIGHTEN-Cirrhosis trial in MASH and 89bio’s potential BLA and MAA filings. Words such as "may," "might," "will," "objective," "intend," "should," "could," "can," "would," "expect," "believe," "design," "estimate," "predict," "potential," "anticipate," "goal," "opportunity," "develop," "plan" or the negative of these terms, and similar expressions, or statements regarding intent, belief, or current expectations, are forward looking statements. While 89bio believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on information available to us on the date of this release. These forward-looking statements are based upon current estimates and assumptions and are subject to various risks and uncertainties (including, without limitation, those set forth in 89bio's filings with the Securities and Exchange Commission (SEC)), many of which are beyond 89bio's control and subject to change. Actual results could be materially different. Risks and uncertainties include: expectations regarding the timing and outcome of the ENLIGHTEN-Fibrosis Phase 3 trial and Phase 3 ENLIGHTEN-Cirrhosis trial in MASH and ENTRUST Phase 3 trial in SHTG; 89bio's ability to execute on its strategy; positive results from a clinical study may not necessarily be predictive of the results of future or ongoing clinical studies; 89bio's substantial dependence on the success of it lead product candidate; competition from competing products; the impact of general economic, health, industrial or political conditions in the United States or internationally; the sufficiency of 89bio's capital resources and its ability to raise additional capital; and other risks and uncertainties identified in 89bio’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 and other subsequent disclosure documents filed with the SEC. 89bio claims the protection of the Safe Harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. 89bio expressly disclaims any obligation to update or alter any statements whether as a result of new information, future events or otherwise, except as required by law. Investor Contact: Annie Chang89bio, Inc.annie.chang@89bio.com PJ KelleherLifeSci Advisors, LLC+1-617-430-7579pkelleher@lifesciadvisors.com Media Contact: Sheryl SeapyReal Chemistrysseapy@realchemistry.com