FGF19

SOUTH SAN FRANCISCO, CA, USA I February 24, 2025 I NGM Biopharmaceuticals, Inc. (NGM Bio), a privately held biotechnology company focused on discovering and developing transformative therapeutics for patients, today announced that it has dosed the first participant in its EMERALD Phase 2 clinical study of NGM120, a GFRAL antagonist designed to block GDF15 activity, for the treatment of hyperemesis gravidarum (HG), a rare, debilitating condition of pregnancy. The leading cause of hospitalization in early pregnancy, HG is characterized by intractable nausea and vomiting, which can be as frequent as 10 to 15 times per day and results in dehydration, debility, weight loss and malnutrition. HG has a significant physical and psychosocial impact on patients and leads to overall higher rates of fetal loss and termination, preeclampsia, preterm birth, low birth weight, fetal malnutrition, maternal depression, and in some cases, suicidal thoughts. HG typically recurs in subsequent pregnancies. There are no approved therapies that target the underlying cause of HG. “We are proud to advance NGM120 into the clinic to evaluate its potential to address hyperemesis gravidarum, or HG, by targeting a major underlying cause of this debilitating condition,” said Wendy Yeh, M.D., Chief Medical Officer at NGM Bio. “Unfortunately, the severity and impact of HG are often underappreciated. At NGM, we understand how devastating this condition is, and we hear the pleas of women who experience the physical and mental toll of HG for an effective therapeutic solution. By inhibiting the activity of GDF15, NGM120 may potentially help reduce symptoms associated with HG, and, thereby, mitigate the impact and significant risks this condition can present during pregnancy.” Although GDF15 is a hormone that everyone produces, levels of GDF15 are higher in pregnant women and can be even higher in women with HG. The extremely high levels of GDF15 are believed to cause the nausea and vomiting that are hallmarks of HG. NGM120 is an antibody designed to bind to the receptor (known as GFRAL) for GDF15. This binding blocks the interaction of GDF15 with GFRAL, which, in turn, inhibits the activity of GDF15, potentially alleviating HG symptoms. NGM120 has been generally well-tolerated in over 140 non-pregnant participants treated in clinical trials to date. To learn more about NGM120 as a potential treatment for HG, link here . About the Randomized, Placebo-Controlled EMERALD Phase 2 Study The randomized, placebo-controlled EMERALD study is designed to assess the safety and tolerability of NGM120 in addition to standard of care and supportive care. Eligible pregnant participants diagnosed with HG who are enrolled in the study will receive a single subcutaneous dose of NGM120 or placebo on the first day of the assigned study period, which lasts seven days. Participants assigned to the NGM120 arm and participants assigned to the placebo arm will also receive the following: intravenous (IV) fluids with multivitamins on several days prior to and throughout the seven-day study period as supportive care; IV multivitamins according to the standard of care at each study center; and 4 mg ondansetron (a prescription medicine that counteracts nausea and vomiting) three times daily, per standard of care. The EMERALD study is enrolling participants in the United Kingdom ( ISRCTN76681798 ) and Australia (ACTRN12624001421527). To learn more, please visit the following clinical trial listings: United Kingdom (Link here ) Australia (Link here ) About NGM Bio NGM Biopharmaceuticals, Inc. (NGM Bio), a wholly owned subsidiary of NGM Bio Holdings, Inc., a privately held biotechnology company, is focused on discovering and developing novel, life-changing medicines for people whose health and lives have been disrupted by disease. NGM Bio’s biology-centric drug discovery approach aims to seamlessly integrate interrogation of complex disease-associated biology and protein engineering expertise to unlock proprietary insights that are leveraged to generate promising product candidates, enable their rapid advancement into proof-of-concept studies and address high unmet patient need. NGM Bio has three clinical-stage programs: NGM120, a GFRAL antagonist designed to block the activity of GDF15, in development for hyperemesis gravidarum and for cancer cachexia, and aldafermin, an engineered analog of the human hormone FGF19, in development for primary sclerosing cholangitis. Visit us at http://www.ngmbio.com for more information. Abbreviations FGF19 = fibroblast growth factor 19; GDF15 = growth differentiation factor 15; GFRAL = glial cell-derived neurotrophic factor receptor alpha-like SOURCE: NGM Bio

SHANGHAI, Sept. 18, 2024 /PRNewswire/ -- Abbisko Therapeutics (HKEX: 02256) is excited to announce the receipt of t he ESMO 2024 Best Poster Award on September 16, 2024. The award was received for the presentation titled "Updated Safety and Efficacy of Irpagratinib (ABSK011) in advanced hepatocellular carcinoma (aHCC) with FGF19 overexpression from a Phase 1 study". The update data from ABSK-011-101 study showed a tolerable safety profile and promising anti-tumor activity of Irpagratinib monotherapy in aHCC. Of note, in aHCC patients who were pretreated with both immune checkpoint inhibitor (ICI) and Tyrosine Kinase Inhibitor (TKI)—a population with high unmet need in the current treatment paradigm— the observed ORR and DCR was 44.8% and 79.3%, respectively, with a median duration of response (mDoR) of 7.4 months and median progression free survival (mPFS) of 5.5 months. Continue Reading 2024 ESMO Congress The poster, #983P, was presented at the Hepatocellular Carcinoma poster session on Sunday, September 16, 2024. The Best Poster Award was given at the conclusion of the Sunday Poster Session, with only one recipient being honored in HCC poster session. As of September 5, 2024, 122 patients have been enrolled, including 74 in the BID cohort with doses consisting of 160mg BID, 220mg BID, and 300mg BID. 5.4% of patients were BCLC Stage B, and 89.2% BCLC Stage C, 64.9% had a Child-Pugh (CP) Score of 5, 27% CP Score of 6, and 6.8% CP Score of 7. 64.9% of patients received multiple lines of prior therapy, 85.1% of patients had previously been treated with ICIs, and 75.7% of patients had previously been treated with both ICIs and mTKIs. The efficacy data show that forty pre-treated HCC patients with FGF19 overexpression were treated with irpagratinib 220 mg BID. Among the 38 evaluable patients, the response rate was 36.8% (14/38), and the disease control rate (DCR) was 78.9% (30/38). The response rate from the subset of patients who had previously received ICI and mTKI therapy was 44.8% (13/29). The longest observed DoR was 16.4 months and the mDoR was 7.4 months. DCR was 79.3% (23/29). mPFS was 5.5 months. Safety data show one dose-limiting toxicity (DLT) was observed in the 300 mg BID cohort. The most common treatment-related adverse effects (TRAEs, >20%) were ALT elevation, diarrhea, AST elevation, hyperphosphatemia, bilirubin elevation, alkaline phosphatase elevation, platelet decrease, and total bile acid elevation. Grade 3-4 treatment-related adverse events (>5%) included AST elevation, ALT elevation, and diarrhea. No grade 5 adverse events occurred. HCC is the main type of liver cancer, accounting for 85% to 90% of primary liver cancers. HCC is highly malignant, about 30% of which have abnormally high FGFR4 expression and a poor prognosis, and the existing treatment methods still cannot meet the long-term survival benefits. Currently, there is no approved standard of care for HCC patients who have progressed from first-line ICI-based therapies. The FGF19/FGFR4 signaling axis could be a novel therapeutic target for HCC. ABSK-011, a potent FGFR4 inhibitor, demonstrated a tolerable safety profile and promising anti-tumor activity as a single agent. Notably, the irpagratinib 220mg BID regimen exhibited a 44.8% ORR, 7.4 months mDoR and 5.5 months mPFS in heavily pre-treated HCC patients who had received both ICI and mTKI therapy, supporting further late-stage development of irpagratinib in such populations with substantial unmet medical need. In addition, the design of the phase II study of pimicotinib in combination with chemotherapy and with/without toripalimab as first-line treatment for advanced pancreatic ductal adenocarcinoma (PDAC) has been presented. About Irpagratinib (ABSK011) Irpagratinib is a highly selective FGFR4 small molecule inhibitor intended for the treatment of advanced solid tumors that present with abnormalities in the FGF19/FGFR4 signaling pathway (e.g., ligand FGF19 amplification/overexpression, FGFR4 mutation/amplification/fusion), including advanced HCC, cholangiocarcinoma, breast cancer, among others. The FGFR4 signaling pathway is a recognized and promising target for treating HCC. Clinical data with irpagratinib have demonstrated improved potency and anti-tumor efficacy, among other favorable therapeutic properties, compared to competitors. About Abbisko Therapeutics Founded in April 2016, Abbisko Therapeutics Co., Ltd., a subsidiary of Abbisko Cayman Limited (Stock Code on the Hong Kong Stock Exchange: 2256.HK), is an oncology-focused biopharmaceutical company founded in Shanghai, dedicated to the discovery and development of innovative medicines that treat unmet medical needs in China and globally. The Company was established by a group of seasoned drug hunters with rich R&D and managerial expertise from top multinational pharmaceutical companies. Since its founding, Abbisko Therapeutics has built an extensive pipeline of 16 innovative small molecule programs focused on precision oncology and immuno-oncology. Please visit for more information. SOURCE Abbisko WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

SOUTH SAN FRANCISCO, CA, USA I July 17, 2024 I NGM Biopharmaceuticals, Inc. (NGM Bio), a privately held biotechnology company focused on discovering and developing transformative therapeutics for patients, today announced a $122 million Series A financing led by TCG with participation from a select group of investors. NGM Bio will use the proceeds from the Series A financing to initiate a planned registrational trial of aldafermin, an engineered FGF19 analog, for the treatment of PSC, and to complete a planned Phase 2 trial of NGM120, a GDF15/GFRAL antagonist, for the treatment of HG. Both trials are expected to begin in the fourth quarter of 2024. “At the beginning of this year, we unveiled our strategy to advance clinical development efforts for two serious, rare conditions characterized by significant unmet patient need. This Series A capital strengthens our financial position, enabling us to progress our planned registrational trial of aldafermin in primary sclerosing cholangitis and to evaluate NGM120 in a proof-of-concept study for hyperemesis gravidarum,” said David J. Woodhouse, PhD, Chief Executive Officer at NGM Bio. “Having just completed a take-private transaction in April to bolster our efficiency and flexibility as a company, we’re grateful to TCG and our new investors for their support of these important programs, and we look forward to sharing more details on both trials in the coming months.” “The Column Group has long championed NGM’s mission to turn extraordinary scientific discoveries into meaningful therapeutics. NGM continues to show remarkable resilience and persistence in its pursuit of life-saving medicines while maintaining the highest level of scientific rigor. With this Series A investment, TCG is delighted to extend our investment in NGM and support the compelling opportunities presented by aldafermin and NGM120 as potential treatments for PSC and HG, respectively,” said Peter Svennilson, Founder and Managing Partner of TCG. Concurrent with the Series A financing, Mr. Svennilson joined NGM Bio’s Board of Directors. Aldafermin as a Potential Treatment for PSC PSC is a rare disease that irreparably damages the bile ducts, leading to bile acid dysregulation, which ultimately can result in end-stage liver disease, liver failure and bile duct cancer. NGM Bio’s advancement of aldafermin as a potential treatment for PSC is based on a large body of clinical data that supports its differentiated potential to address the bile acid dysregulation underpinning the disease, including NGM Bio’s prior Phase 2 study in PSC. NGM Bio anticipates that the registrational trial for aldafermin in PSC will utilize proposed surrogate endpoints with the goal of obtaining accelerated approval. Learn more about aldafermin as a potential treatment for PSC here . NGM120 as a Potential Treatment for HG HG is characterized by intractable nausea and vomiting (as frequent as 10 to 15 times per day), which results in dehydration, debility, weight loss and malnutrition. HG has a significant physical and psychosocial impact on patients and leads to overall higher rates of fetal loss and termination, preeclampsia, preterm birth, low birth weight, fetal malnutrition, maternal depression, and in some cases, suicidal thoughts. This condition is the second leading cause of hospitalization in pregnancy (second to preterm labor) and typically recurs in subsequent pregnancies. Development of NGM120 as a potential treatment for HG is rooted in NGM Bio’s decade-long efforts to advance the understanding of GDF15 biology and explore its therapeutic application in a number of disease areas. Recent genetic research confirms the link between HG and higher serum levels of GDF15. NGM120 is an antibody designed to block GDF15 signaling through its cognate receptor, GFRAL, in cells at the base of the brain involved in triggering nausea and vomiting and, thereby, may have therapeutic benefit for treating patients suffering from HG. Learn more about NGM120 as a potential treatment for HG here . Aldafermin and NGM120 were both discovered through NGM Bio’s in-house discovery engine. Abbreviations (in alphabetical order) FGF19 = fibroblast growth factor 19; GDF15 = growth differentiation factor 15; GFRAL = glial cell-derived neurotrophic factor receptor alpha-like About NGM Bio NGM Biopharmaceuticals, Inc. (NGM Bio), a wholly owned subsidiary of NGM Bio Holdings, Inc., a privately held biotechnology company, is focused on discovering and developing novel, life-changing medicines for people whose health and lives have been disrupted by disease. NGM Bio’s biology-centric drug discovery approach aims to seamlessly integrate interrogation of complex disease-associated biology and protein engineering expertise to unlock proprietary insights that are leveraged to generate promising product candidates, enable their rapid advancement into proof-of-concept studies and address high unmet patient need. Visit us at http://www.ngmbio.com for more information. SOURCE: NGM Biopharmaceuticals