Veligrotug

With $95 million in hand, Khartis Therapeutics made its public debut Thursday with the goal of developing an oral medication for thyroid eye disease (TED) that could replace today's infusion-based treatments.The San Diego biotech is developing a selective small molecule inhibitor of insulin-like growth factor 1 receptor (IGF-1R), the same target behind Amgen's Tepezza (teprotumumab-trbw) and Viridian Therapeutics' newly approved Lumvoa (veligrotug-vvze).The newco has a pedigree in small molecule drug discovery. CEO and head of research Robert Hoffman, Chief Scientific Officer Craig Murphy and Executive Chairman Chris LeMasters previously worked at XinThera, a small molecule biotech acquired by Gilead Sciences in 2023. Hoffman led chemistry at XinThera, Murphy headed immunology and LeMasters was CEO."At XinThera we built camaraderie and execution know-how, and that experience provided the ideal platform to launch Khartis," Hoffman told FirstWord in an email.Chasing an oral optionThe funding total includes $50 million raised in a series B led by Forge Life Science Partners. Longwood Fund and Alexandria Venture Investments, alongside existing investors Foresite Capital, Lilly Asia Ventures and Nextech Invest also participated in the round.The start-up plans to use the cash to move its lead programme toward the clinic in the first quarter of 2027 as well as build out its broader pipeline of three additional programmes aimed at established immunology pathways.Tepezza and Lumvoa are antibodies administered by intravenous infusion, though the latter is distinguished by a shorter treatment course — five infusions over 12 weeks versus eight with Tepezza — along with late-stage data encompassing patients with both active and chronic disease (see – Physician Views Results: Lumvoa looks like worthy Tepezza challenger, but SC squeeze threatens both)."TED remains a serious burden for a highly underserved patient population, where existing treatments come with real safety tradeoffs," said Murphy in a company release Thursday. "We believe an oral, selective IGF-1R inhibitor can change that equation, helping to substantially open the market with a safer, more durable patient-friendly option."Other contendersKhartis isn't the only company developing an oral option for TED. Sling Therapeutics is further along with linsitinib, an oral IGF-1R inhibitor that has been studied in more than 800 patients and recently entered the Phase III ORBIT trial in patients with moderate-to-severe active disease. The company previously reported data from the Phase IIb/III LIDS study, in which the 150-mg twice-daily dose had a 52% proptosis responder rate. However, while statistically significant, that still came in below rates seen in Tepezza's pivotal trials.  There are other candidates in development as well, though unlike Khartis and Sling, they are antibodies rather than oral small molecules.Yarrow Bioscience is working on YB-101, an antibody designed to block the thyroid-stimulating hormone receptor (TSHR). The programme is being developed with partner GenSci in China and is designed for subcutaneous rather than intravenous administration, which could allow for less burdensome dosing.Ethyreal Bio, which itself recently emerged from stealth, is pursuing another TSHR-targeting antibody, called ETHY-001, that is also designed for subcutaneous administration. A first-in-human trial planned for the second half of 2026. The start-up presented the first preclinical data on ETHY-001 in June showing complete blockade of autoantibody-mediated TSHR activation across all patient serum samples tested.

Three years after Robert Hoffman sold XinThera to Gilead, the veteran Pfizer chemist has unveiled his latest venture. Hoffman co-founded Khartis Therapeutics in 2024 with other former leaders of XinThera after Gilead bought the latter biotech mainly for its promising oncology small molecules. On Thursday, San Diego-based Khartis officially emerged from stealth with a $50 million series B round that brings the company’s total fundraising haul to $95 million. The series B was led by Forge Life Science Partners, with Longwood Fund and Alexandria Venture Investments also pitching in alongside existing investors Foresite Capital, Lilly Asia Ventures and Nextech Invest. Khartis’ primary goal is to push a potential pill for thyroid eye disease (TED) into clinical trials. In TED, inflammation behind the eye causes bulging, irritation and a suite of other discomfiting symptoms. Khartis’ candidate is designed to target insulin-like growth factor 1 receptor (IGF-1R), the same protein already drugged by Amgen’s first-in-class blockbuster Tepezza and Viridian Therapeutics’ recently approved Lumvoa. Both these approved medicines are administered as IV infusions, a much more involved procedure than popping a pill. “TED remains a serious burden for a highly underserved patient population, where existing treatments come with real safety tradeoffs,” Craig Murphy, Ph.D., Khartis’ co-founder and chief scientific officer, said in an August 13 release. “We believe an oral, selective IGF-1R inhibitor can change that equation, helping to substantially open the market with a safer, more durable patient-friendly option.” The company plans to launch its first clinical trial for the pill in the first quarter of 2027. In addition to its TED candidate, Khartis has three other earlier-stage drugs developed internally. “This financing reflects strong conviction in our team, our science and the opportunity ahead,” CEO Hoffman said in the release. “With this backing, we will accelerate advancement of our lead program while continuing to do what this team does best: discovering and developing differentiated small molecule medicines against important, clinically validated targets.” TED has long proven a vexing disease to treat, with Immunovant and Alumis recently pulling back from their struggling infused drug candidates. Other therapies are still in the works at Yarrow Bioscience and Ethyreal Bio —but as antibodies, these assets also require an IV. There is at least one other oral IGF-1R inhibitor for TED in development, from Sling Therapeutics. That candidate, linsitinib, posted positive data from its ongoing phase 2b/3 trial in January 2025, and Sling is now recruiting patients in a confirmatory phase 3 study . Hoffman served as vice president of chemistry at XinThera after previously working as a research chemist at Pfizer for more than three decades. At Khartis, he’s keeping one foot in the science world as head of research while also assuming the CEO mantle for the first time. Murphy, too, was a vice president at XinThera before its sale to Gilead, heading up the immunology department. XinThera’s former CEO Chris LeMasters has also joined Khartis as executive chairman; before XinThera, he was chief operating officer at antifungal biotech Amplyx Pharmaceuticals, leading up to its 2021 acquisition by Hoffman’s old employer Pfizer. Editor's note: This story was updated at 4:30pm E.T. on Aug. 13 to include information on another oral TED candidate.