HitGen, Inc.

By targeting a dysfunctional DNA repair system, inhibitors could slow or halt the progression of Huntington’s disease. Image: Shutterstock/ Andrii Yalanskyi. LoQus23 closed a round of Series A financing to advance development of its lead candidate for Huntington’s Disease. The Cambridge, UK-based biotechnology company announced in a 2 October release it had raised £35m ($43m) in this latest round, led by venture capital firm Forbion. The proceeds will advance preclinical and clinical research of LoQus23’s foremost asset, its small molecule MutSβ inhibitor. Along with Forbion were prior investors Novartis Venture Fund and SV Health Investors’ Dementia Discovery Fund. In addition, Forbion General Partner Rogier Rooswinkel will join the LoQus23 board of directors. LoQus23’s MutSβ inhibitor is set to enter clinical development in 2026. The allosteric small molecule targets MutSβ, a protein component of the mismatch repair system (MMR) strongly implicated as the central cause of Huntington’s Disease. The MMR system, which when functioning properly resolves errors in DNA structure, is known to drive disease pathophysiology by increasing CAG nucleotide repeats within the Huntingtin gene. Once these repeats reach a critical threshold, patients’ symptoms of neuronal dysfunction progress and ultimately lead to death. LoQus23 aims to slow or halt this progression by inhibiting the MMR system. See Also: Fresenius Kabi wins FDA approval for Stelara biosimilar Otulfi BMS and Prime ink potential $3.5bn deal to develop T cell therapies The company has established a series of small molecule inhibitors targeting MutSβ and MutSα, another MMR component. The proteins are thought to be relevant in up to 30 triplet-repeat diseases. Aside from Huntington’s Disease, these inhibitors are being investigated to treat myotonic dystrophy and broader genetic disorders. In 2019, LoQus23 raised $5.9m in seed capital to advance its candidates, then a further $9.4m in 2021. In 2022, the company entered into a research collaboration with the Chinese biotech HitGen to screen for small molecule candidates using its DNA-encoded library platform. HitGen has also partnered with Pfizer , Boehringer Ingelheim , and Cancer Research UK among others to leverage its high throughput screening capabilities. Huntington’s Disease affects roughly 30,000 patients in the US alone, for whom there is not yet any disease-modifying treatment available. Rogier Rooswinkel, the newly appointed LoQus23 board member, said that for these patients, “MutSβ seems the most promising and best validated target, with the potential to bring disease progression to a halt.” Dr David Reynolds, LoQus23 CEO, said, “This financing will enable us to develop key clinical data to support the development of our exciting lead programme.” He also emphasised the company’s focus on developing an oral formulation for the therapy to increase patient adherence and convenience.

Lipigon Pharmaceuticals AB (Lipigon) meddelar idag att bolagets utvecklingspartner Leaderna Pharmaceuticals Ltd (Leaderna) har behandlat den sista patienten i en klinisk fas 1-studie vars syfte är att utvärdera säkerheten och tolerabiliteten hos läkemedelskandidaten Lipisense® i en kinesisk population. Samtidigt pågår uppstarten av en klinisk fas 2-studie av Lipisense® i Sverige. Studien genomförs inom ramen för bolagets kommersiella utvecklingsavtal med det kinesisk-amerikanska läkemedelsbolaget Leaderna. Leaderna är en avknoppning från den globala bioteknikkoncernen HitGen Inc. och är specialiserat på utvecklingen av avancerade RNA-läkemedel inom metabolism, hjärt-kärlsjukdomar och inflammation. Lipisense® utvecklas som en potentiell behandling av patienter med kraftigt förhöjda nivåer av blodfettet triglycerider och har tidigare visat goda resultat i kliniska studier genomförda i Europa. I den nuvarande studien genomförs ytterligare utvärderingar av läkemedelskandidatens farmakologiska säkerhet och tolerabilitet i friska personer. Totalt omfattar studien 24 personer som erhållit single ascending dose (SAD) med Lipisense® eller placebo. De övergripande resultaten väntas under slutet av första kvartalet 2025. "Vi är mycket glada att vår kommersiella utvecklingspartner gör snabba framsteg i den kliniska utvecklingen av Lipisense®. Fas 1-studien är det första steget i det utvecklingsprogram som syftar till att på sikt tillgängliggöra vår innovativa läkemedelskandidat på den kinesiska marknaden. Vi ser fram emot att ta del av studiens resultat och fortsätta samarbetet i den kliniska utvecklingen av Lipisense® ", säger dr Stefan K. Nilsson, vd för Lipigon. Om Lipisense® Lipisense® är en läkemedelskandidat baserad på antisens-teknologi, avsedd att minska produktionen av ANGPTL4-protein i levern. Genetiska data visar att ANGPTL4 är en oberoende riskfaktor för både hjärt-kärlsjukdom och typ 2-diabetes. Genom att blockera det RNA som kodar för ANGPTL4, förhindrar Lipisense® att detta sjukdomsfrämjande protein bildas. Om Leaderna Leaderna Therapeutics Ltd. (Leaderna), en spin-off från HitGen Inc. (682222.SH), fokuserar på banbrytande forskning och utveckling av syntetiska oligonukleotidläkemedel. Genom att utnyttja sin avancerade siRNA-design, modifiering, utvärdering och leveransplattform har Leaderna framgångsrikt etablerat en robust pipeline inriktad på metabolism, hjärt-kärlsjukdomar och inflammation. För ytterligare information, vänligen kontakta: Stefan K. Nilsson, VD, Lipigon E-post: stefan@lipigon.se Telefon: 0705-78 17 68 Om Lipigon Lipigon Pharmaceuticals AB är ett läkemedelsbolag i klinisk fas som utvecklar läkemedel med nya, unika verkningsmekanismer (first-in-class) för sjukdomar orsakade av rubbningar i kroppens hantering av fetter. Bolagets verksamhet bygger på över 50 års lipidforskning vid Umeå universitet. Lipigon fokuserar initialt på ovanliga sjukdomar som kan ge särläkemedelsstatus och på nischindikationer, men på sikt har bolaget möjlighet att rikta sig mot bredare indikationer inom området, som diabetes och hjärt-kärlsjukdom. Lipigons pipeline består av tre aktiva projekt: RNA-läkemedelskandidaten Lipisense® mot förhöjda triglycerider där fas II-studier godkändes i februari 2024, ett RNA-läkemedelsprojekt mot lungskada samt ett småmolekylsprojekt för behandling av dyslipidemi (allmänna blodfettsrubbningar) i samarbete med HitGen Inc. Läs mer på www.lipigon.se. Bolagets aktie (LPGO) är föremål för handel på Nasdaq First North Growth Market. Certified Adviser är G&W Fondkommission. Bifogade filer

The FDA's approval for Yuhan's J&J-partnered Lazcluze, Merck and Kelun's revised ADC deal, and U.S. lawmakers' scrutiny of clinical trials run at Chinese military hospitals made our news this week. Yuhan's Lazcluze, licensed by Johnson & Johnson, has made history as the first Korea-developed novel cancer drug to be approved by the FDA. Merck has optioned in on one Kelun Biotech antibody-drug conjugate but returned another. A few U.S. lawmakers have asked the FDA to examine clinical trials involving sites at Chinese military hospitals. And more. 1. Targeting AZ's Tagrisso, Johnson & Johnson's Rybrevant combo wins key FDA nod in first-line lung cancerThe FDA has approved Johnson & Johnson’s Rybrevant and Lazcluze for newly diagnosed EGFR-mutant non-small cell lung cancer. The combo will compete with AstraZeneca’s Tagrisso. J&J in-licensed Lazcluze, a third-generation EGFR tyrosine kinase inhibitor, from Korea’s Yuhan in 2018. The drug is now the first Korea-developed novel anti-cancer therapy to be approved by the FDA.2. Merck & Co. bags one Kelun ADC for $37.5M, boots out another asset as pipeline tinkering continuesMerck and Kelun Biotech have reworked their multi-asset ADC deal. The New Jersey pharma has decided to return the CLDN18.2-directed SKB315. Although no reason was offered, the CLDN18.2 space has become very crowded with multiple firms exploring various modalities. Merck also agreed to pay $37.5 million to take up an option on a new candidate, a bispecific ADC coded SKB571, although the exact drug target remains unclear.3. Lawmakers urge FDA to investigate clinical trials run in tandem with China's militaryLawmakers on the House Select Committee on the CCP are asking the FDA to scrutinize clinical trials conducted at Chinese military hospitals. The lawmakers questioned the trustworthiness of data generated at those sites and potential risks for U.S. intellectual property. Trials of Eli Lilly’s Alzheimer’s disease drug Kisunla and Pfizer’s cancer med Inlyta were raised as examples. 4. Despite looming BIOSECURE threat, pharma contracting giant WuXi Bio scoops up new projectsMeanwhile, as the House is reportedly gearing up for a vote on the BIOSECURE Act in September, WuXi Biologics said it had added 61 new projects in the first six months of 2024, half of which came from the U.S. Fifty-two of the new projects are for preclinical candidates and five for early clinical work. The number of new projects compares favorably to the company’s haul during the same period last year.5. FDA allows BioNTech-MediLink ADC trial to resume with lower, safer dosesAfter a safety scare in mid-June, the FDA has allowed MediLink Therapeutics to restart a phase 1 trial of its BioNTech-partnered HER3-directed ADC. Before the halt, the trial had recorded three deaths across two dose cohorts. Now, BioNTech said it will focus solely on doses no higher than 3mg/kg as MediLink “had observed a dose level-dependent trend of treatment-related adverse events.”6. Denmark's Adcendo signs $1B biobuck deal for Multitude’s anti-TF ADCDenmark’s Adcendo has signed a $1 billion biobucks deal to get ex-China rights to a preclinical ADC from Sino-American biotech Multitude Therapeutics. The candidate, coded ADCE-T02, targets tissue factor. A phase 1 trial of the drug is scheduled to start in Australia in the fourth quarter, and Adcendo is planning to get FDA’s green light to launch a U.S. study “in the near future.”7. Eisai, Biogen's Leqembi nabs UK nod, but reimbursement remains elusiveThe U.K. has approved Eisai and Biogen’s Leqembi for early-stage Alzheimer’s disease but excluded patients with two copies of the ApoE4 gene out of safety concerns. Still, the National Institute for Health and Care Excellence has tentatively denied coverage of the drug under Britain’s taxpayer-funded insurance program. The drug cost watchdog cited Leqembi’s “relatively small benefits” and high costs.Other News of Note: 8. Astellas employee held in China indicted by China’s prosecutors9. Insulet unveils $200M insulin delivery device production site in Malaysia10. Pathalys raises $105M series B to prepare Japan-approved kidney disease drug for FDA11. FDA rebukes another troubled Eugia production site with a warning letter12. Korea’s Bridge Biotherapeutics, China’s HitGen sign drug discovery pact for cancer (release)13. Pfizer pulls smoking cessation drug Champix from Korean market (Korea Biomedical Review)