NTSR1

AstraZeneca has terminated all development of vemircopan, the oral factor D inhibitor it acquired through the deal for Alexion in 2021. The UK major recorded an impairment charge of $753 million as a result, according to 2024 earnings that were reported on Thursday. Development of vemircopan for paroxysmal nocturnal hemoglobinuria was halted in November 2023, but now the drug is being abandoned completely. A Phase 2 trial in lupus nephritis or IgA nephropathy, and an early-stage trial in impaired hepatic function, have also been discontinued for lack of efficacy. Marc Dunoyer, the CEO of AstraZeneca’s rare disease unit, said it “is not immune to the odds of the industry, where not all of the pioneering work that you do is going to work.” He added that the decision to drop vemircopan was based on standard criteria, namely its benefits and risks, as well as the competitive situation. Despite the loss, AstraZeneca CEO Pascal Soriot told reporters at a London event: “I can say with no hesitation, the Alexion acquisition was a fantastic acquisition.” AstraZeneca also took a $165 million impairment on FPI-2059, a Phase 1-stage radioligand that was scrapped. The drug was part of last year’s purchase of Fusion Pharmaceuticals. FPI-2059 is a lutetium-labeled small molecule targeting neurotensin receptor 1 which Alexion itself licensed from Ipsen in 2021. Other radioligands remain in development. AstraZeneca’s lead radioconjugate, FPI-2265, is a beta-emitting, PSMA-targeting product. Data on its Phase 2 prostate cancer trial could come in the second half of this year. “The field of radioconjugates is a little behind the field of ADCs at the moment, but it holds great potential,” AstraZeneca’s oncology R&D leader Susan Galbraith said at the earnings event. Elsewhere, AstraZeneca shut down: The company also reiterated the termination of a mid-stage study of an orexin 1 receptor antagonist, AZD4041, in opioid use disorder. The shuttering emerged in November , with a spokesperson telling Endpoints News the move was due to “a potential drug-drug interaction.” It appears that AstraZeneca will put its long-acting amylin asset AZD6234 into a new Phase 2b trial in obesity, in combination with the GLP-1/glucagon agonist AZD9550. According to a record on the EU’s trial database , the trial will end in the middle of next year. AZD6234 is already in a separate mid-stage monotherapy trial, which could yield data late this year. The company claimed it is heading into an “unprecedented catalyst-rich 2025,” citing a handful of late-stage trials for indication expansions, such as the AVANZAR study of Datroway in first-line non-small cell lung cancer. David Fredrickson, the leader of the company’s oncology business, affirmed his confidence in the ADC despite its stumbles in late-stage trials in both lung and breast cancers. “We’ve been able to apply the lessons that we’ve learned from the previous Phase 3 and Phase 2 studies that have read out so far to the frontline AVANZAR study,” Fredrickson said. The trial is enriched for the non-squamous population “because we think that’s the opportunity to see the most benefit for Datroway in the front line.” Combining the drug with the checkpoint inhibitor Imfinzi could also increase the chances of success, he said. Other major Phase 3 readouts will come for products still in earlier stages, such as the SERD camizestrant in breast cancer, baxdrostat in hypertension, and eneboparatide in hypoparathyroidism.

SK Biopharmaceuticals, Korea Institute of Radiological and Medical Sciences collaborate on discovering and developing radiopharmaceutical drug candidates using an alpha-particle emitting radioisotope, actinium-225, for potential cancer treatments The two sides aim to submit an Investigational New Drug application by 2027, in line with SK Biopharmaceuticals' roadmap to strengthen its radiopharmaceutical therapy business SK Biopharmaceuticals further accelerates the expansion of its radiopharmaceutical therapy pipeline and capability, via strengthened internal resources and partnerships SEOUL, South Korea, Nov. 18, 2024 /PRNewswire/ -- SK Biopharmaceuticals, a biotech company, announced that it has entered into an agreement with the Korea Institute of Radiological and Medical Sciences (KIRAMS), Korea's premier research institution dedicated to the study and advancement of radiological and medical sciences, to collaborate on discovering and developing preclinical radiopharmaceutical drug candidates. This marks the first collaborative research agreement in which both sides will aim to discover radiopharmaceutical compounds and investigate novel oncological treatments, using actinium-225 (225Ac), an alpha-particle emitting radioisotope that selectively kills cancer cells. The research for radiopharmaceutical therapy (RPT) focuses on this application that has been gaining attention for its high potential in nuclear medicine. SK Biopharmaceuticals has already initiated its 225Ac-based research as it secured a supply of the radioisotope from TerraPower Isotopes, a subsidiary of TerraPower, a nuclear innovation company whose investors include SK Biopharmaceuticals' parent SK Inc. and Bill Gates. SK Biopharmaceuticals and KIRAMS will seek to submit an Investigational New Drug application by 2027, as they leverage each other's resources to optimize their drug discovery efforts. SK Biopharmaceuticals said it could significantly reduce the time and cost of new drug development, with KIRAMS' researchers, facilities and equipment using the radioisotope, enabling the company to further accelerate in securing and expanding its RPT pipeline and capability. The agreement is in line with SK Biopharmaceuticals' so-called "RPT Roadmap" recently introduced to become a global leading RPT player by 2027, as the company will fortify its RPT business by discovering new drug compounds, extending supply and production capacities, and developing a tech platform, via strengthened internal assets and strategic partnerships. In addition to the 225Ac supply agreement aligned with its roadmap, the company has in-licensed SKL35501 (FL-091) radiopharmaceutical compound targeting neurotensin receptor 1 (NTSR1) solid tumors. Jinkyung Lee, President of KIRAMS, stated, "Through this joint research agreement, we aim to take a leading role in developing innovative radiopharmaceutical therapeutics, in alignment with the Ministry of Science and ICT's strategic initiative to drive advancements in Radiation Biology. We look forward to contributing to the growth of the domestic radiopharmaceutical industry in close collaboration with SK Biopharmaceuticals." Donghoon Lee, CEO and President of SK Biopharmaceuticals, said "This agreement is one of key steps in enhancing SK Biopharmaceuticals' research capabilities. We will seek to spur SK Biopharmaceuticals to become a leading global RPT player through strengthened ties." About SK Biopharmaceuticals SK Biopharmaceuticals focuses on the research, development and commercialization of treatments for disorders of the central nervous system (CNS) and oncology. In 2017, SK Biopharmaceuticals established a research center to begin its expansion into oncology through research and development efforts. SK Biopharmaceuticals is the first and only Korean company to independently develop and commercialize an antiseizure medication, cenobamate (brand name: XCOPRI®) in the U.S. More than 100,000 patients have been treated globally with cenobamate, developed by SK Biopharmaceuticals and its U.S. subsidiary SK Life Science, Inc., since its launch in the U.S. in 2020. Cenobamate has successfully entered five major regions: North America, Europe, Asia, Central and South America, and the Middle East and North Africa. The company also has a pipeline of eight compounds in development in both CNS disorders and oncology. Additionally, SK Biopharmaceuticals is focused on the discovery of new treatments in oncology. For more information, visit SK Biopharmaceuticals' website at , and SK Life Science's website at . About Korea Institute of Radiological and Medical Sciences As a premier institution dedicated to the intersection of science, technology, and healthcare, the Korea Institute of Radiological and Medical Sciences (KIRAMS) is at the forefront of radiation medicine innovation. Ever since KIRAMS opened its doors as a radiation medicine research center in 1963, we have been contributing to the national development of science and technology, and to the promotion of the health of the people, based on our research in medical uses of radiation. By bringing the very first Cobalt-60 radiotherapy machine for cancer treatment to Korea in 1963, we were able to greatly increase the number of completely cured cancer patients. Following the restructuring of a cancer hospital in 1973, KIRAMS was leading domestic cancer treatment until the 1990s, by introducing diagnostic and treatment technologies that incorporated advanced science. In 2007, we became an independent institution, directly under the Ministry of Science and ICT, and since then we have established "Radiation Medicine" as a separate field of research and distinguished ourselves from other medical institutes by expanding the National Radiation Emergency Medical Center. And now, going beyond our original mission of defeating cancer, KIRAMS is evolving into a public institution committed to solving new problems for the country and for society, as well as for the benefit of the people. In other words, we are opening new horizons in radiation medicine by supporting specialized, research-centric hospitals engaged in advanced biomedical R&D, by consolidating research and detection of radiation in everyday life, which has recently become a social issue, and by establishing and operating the new Radioisotope Center for Pharmaceuticals to promote the research and use of radioisotopes for medical purposes. Leveraging our extensive experience and our accomplishments, we here at the Korea Institute of Radiological and Medical Sciences will not rest but continually develop into the number one specialist institute for radiotherapy trusted by the people. For more information, visit . SOURCE SK Biopharmaceuticals Co., Ltd WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

FDA's citations for Hengrui, Sanofi's $437 million expansion in India, and closure of the Singapore biotech Aslan made our news this week. A 10-page Form 483 has detailed the shortfalls at Hengrui Pharma's PD-1 plant behind a recent FDA rejection. Sanofi is investing heavily to expand its global capacity center in India. Singapore biotech Aslan Pharma is winding down. And more. 1. Behind Hengrui's surprise FDA rejection: Scathing Form 483 lists litany of shortfalls at PD-1 drug plant The Form 483 that led to the FDA’s rejection of Jiangsu Hengrui Pharma and Elevar Therapeutics’ PD-1 inhibitor camrelizumab included 10 observations issued to Hengrui’s subsidiary, Suzhou Suncadia Biopharmaceuticals. In addition, the FDA has upgraded a prior eight-observation Form 483 into a warning letter for Hengrui’s small-molecule facility in Lianyungang, China. 2. Sanofi to more than double its workforce at Hyderabad site with $437M expansion: reports Sanofi will invest 400 million euros through the end of the decade to expand its global capacity center in Hyderabad, India. Sanofi plans to grow the site’s workforce to 2,600 people—from 1,000 today—in two years, making it the largest of the company’s four global capacity hubs. The site will grow its digital capabilities, including utilizing artificial intelligence tools. 3. Aslan’s dream of challenging Dupixent dies as cash-strapped biotech winds down Aslan Pharmaceuticals, one of the first Singapore-based biotechs to rise to the global stage, is shutting down. With $18.4 million in cash as of the end of March, the company has decided to file for liquidation and has let go all its staffers. The biotech’s stock price was once worth nearly $390 a piece at its peak in 2018 until its lead program, the pan-HER drug varlitinib, failed. Before the decision to close doors, Aslan received a delisting warning from Nasdaq as its stock price had been trading below the $1 bid price. 4. J&J’s CAR-T partner Legend Biotech reportedly receives buyout offer Legend Biotech reportedly received a takeover bid and has tapped Centerview Partners as a financial adviser, according to StreetInsider. The stock price surged for Legend and its former parent and largest shareholder, GenScript. The report came a few weeks after two House lawmakers had requested an intelligence briefing on GenScript and its related operations, including Legend. 5. SK inks $572M deal for Full-Life prospect, advancing push to become Asia’s radiopharma leader SK Biopharmaceuticals inked a $571.5 million biobucks deal to license an NTSR1-directed radionuclide-drug conjugate candidate, FL-091, from Full-Life Technologies. NTSR1 is overexpressed in colorectal, breast, pancreatic, and head and neck cancers. The Korean company outlined plans a year ago to expand in oncology and identified radiopharmaceuticals as one of the modalities driving the push. Other News of Note: 6. Sotio taps Biocytogen to create bispecific ADCs in $326M deal 7. Aveo spins a silver lining in failed trial of kidney cancer drug Fotivda 8. BeiGene brings on Merck veteran as new CFO (release)