Lirafugratinib

Fort Lee, N.J., Jan. 28 – Elevar Therapeutics, Inc., a majority-owned subsidiary of HLB Co., Ltd. and a fully integrated biopharmaceutical company dedicated to elevating treatment experiences and outcomes for patients who have limited or inadequate therapeutic options, today announced it submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational drug lirafugratinib as a second-line treatment option for cholangiocarcinoma (CCA) patients with FGFR2 fusion or rearrangement. CCA, also known as bile duct cancer, is rare, with about 8,000 people in the U.S. diagnosed each year, according to the American Cancer Society. “This NDA reaffirms Elevar’s mission of bringing life-changing medicines to cancer patients worldwide, including for rare indications and for advanced stages where treatment options are limited,” said Dong-Gun Kim, the company’s chief executive officer. “We are excited to work with the FDA as it reviews the submission while simultaneously preparing for a potential commercial launch. We could not be more appreciative of the patients who participated in lirafugratinib-focused clinical trials and everyone who brought us to this crucial point in its development.” The NDA is supported by positive clinical data from the phase 1/2 ReFocus trial (NCT04526106), in which lirafugratinib demonstrated clinically meaningful anti-tumor activity, measured by objective response rate (ORR), duration of response (DoR), and progression-free survival (PFS), as well as manageable and tolerable safety in patients with advanced/metastatic CCA and other solid tumors with fibroblast growth factor receptor 2 (FGFR2) alterations. The abstract “Efficacy and safety of lirafugratinib in FGFRi-naïve cholangiocarcinoma (CCA) patients harboring FGFR2 fusions/rearrangements (FGFR2 f/r)” was presented earlier this month at the American Society of Clinical Oncology’s Gastrointestinal Cancers Symposium. Independent review committee-assessed ORR was 46.5%, and median DOR was 11.8 months (mos) (95% CI, 7.5-13.0), where 76.2% of responses lasted >6 mos. Median PFS was 11.3 mos (95% CI, 9.2, 14.8), with 12-month rate of 49.2%. Median OS was 22.8 mos (95% CI, 18.1-27.2), with 12-month rate of 74.6%. The disease control rate was 96.5%. Most common Grade ≥3 treatment-related adverse events (TRAEs) were palmar-plantar erythrodysesthesia (32.8%) and stomatitis (12.1%). TRAEs led to dose reduction, dose interruption, and treatment discontinuation were observed in 75.9%, 82.8%, and 4.3%, respectively. Elevar is also developing lirafugratinib for patients with other FGFR2-altered other solid tumors. The company acquired worldwide rights to develop and commercialize lirafugratinib in December 2024. Earlier that year, Relay Therapeutics met with the FDA to discuss data from the ReFocus trial and potential regulatory pathways. The FDA recommended the company first file an NDA for FGFR2-driven CCA, followed by a supplemental NDA for FGFR2-altered other solid tumors. For more information about lirafugratinib, visit ElevarTX.com. About Lirafugratinib Lirafugratinib (RLY-4008) is a potent, selective and oral small molecule inhibitor of FGFR2, a receptor tyrosine kinase that is frequently altered in certain cancers. FGFR2 is one of four members of the FGFR family, a set of closely related proteins with highly similar protein sequences and properties. Preclinically, lirafugratinib demonstrated FGFR2-dependent killing in cancer cell lines and induced regression in in vivo models with minimal inhibition of other targets, including other members of the FGFR family. In addition, lirafugratinib demonstrated strong activity against known clinical on-target resistance mutations in vitro and in vivo preclinical models. Lirafugratinib is currently being evaluated in a clinical trial to enroll additional patients with previously treated, advanced or metastatic solid tumors other than CCA harboring FGFR2 f/r, who have not treated with prior FGFR inhibitors. About Elevar Therapeutics Elevar Therapeutics, Inc. is a fully integrated biopharmaceutical company built on the promise of elevating treatment outcomes for patients who have limited or inadequate therapeutic options. A subsidiary of HLB Group based in Fort Lee, New Jersey, Elevar’s expertise is rooted in oncology and grounded in compassion. Our team of experienced scientists and industry leaders is highly focused on identifying and developing medicines for complex yet under-treated health conditions, with a shared goal of making a meaningful difference in the lives of patients. For more information, follow us on LinkedIn and Twitter or visit ElevarTX.com. Media Contact: Rosemary Ostmann RoseComm Cell: (201) 615-7751

Padcev and Keytruda's perioperative win, Chinese regulators' decision on Green Valley's Alzheimer's drug, and Fosun's DPP1 licensing deal made our news this week. The combination of Padcev and Keytruda delivered a phase 3 win in muscle-invasive bladder cancer. China did not renew the conditional approval for a controversial Alzheimer's disease drug. Fosun Pharma is out-licensing its DPP1 inhibitor for up to $645 million. And more.1. Pfizer and Astellas' Padcev, plus Keytruda, delivers 'breakthrough' win in new bladder cancer useAfter making waves in first-line metastatic bladder cancer, the combination of Pfizer and Astellas’ Padcev and Merck & Co.’s Keytruda has shown it can extend lives when used as a perioperative treatment around surgery for patients with cisplatin-ineligible muscle-invasive bladder cancer, according to a readout from the phase 3 EV-303 study, which is also known as Keynote-905.2. China did not renew approval of controversial Alzheimer's drug, regulator's records showChina’s drug regulator did not renew the conditional nod for Green Valley Pharmaceuticals’ controversial Alzheimer’s disease med sodium oligomannate (GV-971), according to public records. The move followed a protracted review that stretched past the expiration date of the drug’s existing licensure, triggering a production and marketing halt in May.3. Expedition inks $645M pact to pocket Fosun's lung disease asset in knapsackInflammatory disease startup Expedition Therapeutics is paying $17 million upfront, plus up to $628 million in potential milestones, for ex-China rights to Fosun Pharma’s DPP1 inhibitor XH-S004. The deal was inked right before the FDA approved Insmed’s brensocatib as the first DPP1 inhibitor. The approved indication for Insmed's therapy is non-cystic fibrosis bronchiectasis.4. Aurobindo acquires US drugmaker Lannett for $250M to bolster 'reshoring' effortAurobindo is paying $250 million to buy Lannett, which is known for manufacturing attention-deficit/hyperactivity disorder medicines and generic liquids. “The acquisition strengthens Aurobindo’s ability to serve the U.S. generics space and provides strategic diversification into a specialized, high-value therapeutic category,” the Indian company said in an announcement.5. HLB taps new Elevar CEO amid liver cancer setbacks, new FDA push (Korea Biomedical Review)Korea’s HLB has tapped Byan Kim as CEO of Elevar Therapeutics. Kim will continue as co-CEO of HLB Innovation and CEO of another HLB subsidiary, Verismo Therapeutics. The move comes after Elevar and Jiangsu Hengrui Pharmaceuticals’ PD-1/VEGFR combination suffered repeated FDA setbacks in liver cancer and as Elevar prepares to file an approval for lirafugratinib in FGFR2-altered cholangiocarcinoma.Other News of Note:6. RemeGen's telitacicept meets the mark in ph. 3 Sjögren's study in China7. Indian API maker Garonit Pharma plans $46.1M antiseptic product plant in New York8. Shilpa wins India approval for world's first NAFLD med, company says (Regulatory tracker)