Orum Therapeutics, Inc.

Plus, news about Caliway Pharmaceuticals, argenx, Halozyme Therapeutics, Verrica, Liquidia and Pharmosa: FDA lifts partial clinical hold on Avidity Biosciences’ drug: The treatment , called delpacibart etedesiran, is in the Phase 3 HARBOR trial in patients with myotonic dystrophy type 1, a progressive and often fatal neuromuscular disease with no approved therapies. The FDA first handed down a partial clinical hold in 2022, following a serious adverse event in an earlier-stage study. Avidity’s stock $RNA rose about 3% on Thursday. — Katherine Lewin Orum Therapeutics outlines IPO in South Korea: The biotech plans to list on the KOSDAQ market to help fund its protein degrader at a time when antibody-drug conjugates are still hot. Orum is seeking between $67 million to $80 million in the IPO. Earlier this year, it partnered with Vertex , and last year it sold an asset to Bristol Myers Squibb. — Kyle LaHucik Caliway Biopharmaceuticals also has IPO plans, in Taiwan: The biotech uplisted to the Taipei Exchange and raised about $206 million, or NT $6.4 billion, which it described as the “largest IPO in Taiwan’s biotech industry history.” The company expects to have Phase 2b results by the end of the year for a subcutaneous fat reduction injectable. It also has preclinical injections for hyperpigmentation and osteoarthritis. — Kyle LaHucik Argenx picks four new targets per deal with Halozyme Therapeutics: The company is using Halozyme’s ENHANZE drug delivery technology to pick a total of six targets. It will now pay Halozyme $30 million upfront for the exclusive rights to the four new targets. Argenx could also pay up to $85 million per target for milestone payments, regulatory approvals, and sales. Halozyme’s stock $HALO gained about 2% in premarket trading on Thursday. — Katherine Lewin Verrica slashes commercial team headcount: The West Chester, PA-based biotech said it cut its workforce by 47 employees in a bid to “streamline operations, reduce costs and preserve capital.” The layoffs are part of the company’s broader push to trim its commercial organization, which includes focusing on 35 sales territories, down from an original 80. It had 100 full-time employees as of Dec. 31. — Ayisha Sharma Liquidia expands drug licensing deal with Pharmosa: Under the terms of the updated pact , Liquidia will get the rights to Pharmosa’s pulmonary arterial hypertension candidate, L606, in markets outside of North America, including Japan and parts of Europe. Liquidia is set to pay Pharmosa $3.5 million upfront and up to $157.75 million in milestones in the new territories. — Ayisha Sharma

DUBLIN--( BUSINESS WIRE )--The "Degrader-Antibody Conjugates 2024: A Landscape Analysis of Stakeholders, Technologies, Pipeline and Partnering from an Industry Perspective" report has been added to ResearchAndMarkets.com's offering. This report provides you with a landscape description and analysis of degrader-antibody conjugate (DAC) stakeholders, platform technologies, development and discovery pipelines and partnering deals from an industry perspective as of September 2024. The emerging novel drug modality of degrader-antibody conjugates represents the convergence of the existing technologies of antibody-drug conjugation and targeted protein degradation with the goal of combining the strengths and avoiding the limitations of both technologies. DACs combine the specificity of antibodies with the efficiency of degraders of difficult to drug protein targets. Degrader-antibody conjugates provide plenty of opportunities for ADC companies to use a novel payload to improve efficacy and the therapeutic window and for targeted protein degradation (TPD) companies to improve cell-specificity, half-life and drug-like properties. The two main uses for Degrader-Antibody Conjugate therapy are: Intracellular Targeted Protein Degradation Extracellular Targeted Protein Degradation The report brings you up-to-date with information about and analysis of: Stakeholders in the field, by company profiles of DAC activities at pharmaceutical and TPD technology companies; DAC technologies using PROTAC and molecular glue for proteasomal degradation for intracellular TPD; Proteins-of-interest for intracellular degradation; E3 ligases used for intracellular degradation; DAC technologies using broadly acting degraders for extracellular TPD; DAC technologies with lysosomal pathway degraders; Clinical and non-clinical development pipeline of DACs; Profiles of clinical stage DACs; DAC discovery pipeline; Preclinical proof-of-concept of DAC conjugate technologies and constructs; Partnering activities (acquisition, licensing, collaborations) The original concept of degrader-antibody conjugates defines DACs as tumor-targeted antibodies chemically conjugated to proteolysis-targeting chimera (PROTACs). PROTACs are large heterobifunctional small molecules with one part binding to the intracellular protein of interest (PoI), e.g. Bruton's Tyrosine Kinase (BTK), via a linker to a second part which binds to an E3 ligase that ubiquitylates the PoI, resulting in proteasomal degradation in the cytosol. The majority of DAC discovery programs rely on this concept, but DAC using a molecular glue instead of a PROTAC are also being pursued and even are the first in clinical evaluation. Molecular glue-type small molecules act as an glue that enhances the interaction between an E3 ligase and a PoI, leading to its ubiquitylation and proteasomal degradation. PROTACs are easier to design than glues given their independent binding sites to the E3 ligase and the PoI. However, they are generally large molecules that can be challenging to develop, whereas glues are typically smaller and more "drug-like". The scope of degrader-antibody conjugates is currently being expanded into the extracellular space for degradation of membrane bound and soluble proteins. Extracellular targeted protein degradation involves bispecific biologics or small molecules that recruit the PoI, either membrane-bound or secreted (soluble), to a degradation machinery. Degradation may occur through a transmembrane E3 ligase, or a cytokine receptor or via a membrane bound recycling receptor for transport of the PoI to the lysosome, which is the typical pathway for the degradation of extracellular proteins. Key Topics Covered: 1 Executive Summary 2 Introduction 3 Analysis of Stakeholders in Degrader-Antibody Conjugate R&D 3.1 Pharmaceutical Companies 3.2 Technology Companies 4 Analysis of Degrader-Antibody Conjugate Technologies 4.1 Overview of Degrader-Antibody Conjugate Technologies 4.2 Degrader-Antibody Conjugates with Molecular Glue-type Degraders 4.3 Degrader-Antibody Conjugates with PROTAC-type Degraders 4.4 Degrader-Antibody Conjugates with Broadly Acting Degraders 4.5 Degrader-Antibody Conjugates with Lysosomal Pathway Degraders 5 Analysis of Degrader-Antibody Conjugate Research & Development 5.1 Clinical & Nonclinical Development Pipeline of Degrader-Antibody Conjugates 5.1.1 Profile of ORM-6151 5.1.2 Profile of ORM-5029 5.2 Pipeline of Discovery Stage Degrader-Antibody Conjugate Programs 5.3 Preclinical Proof-of-Concept of Degrader-Antibody Conjugate Technologies and Constructs 6 Analysis of Partnering in the Field of Degrader-Antibody Conjugate Partnering 6.1 Acquisition 6.2 Licensing 6.3 Discovery Collaborations 7 Profiles of Companies with Degrader-Antibody Conjugate R&D 7.1 Pharmaceutical Companies Bristol Myers Squibb CSPC Pharmaceutical Group Debiopharm Eisai GlaxoSmithKline Merck KGaA Merck & Co. (MSD) Novartis Pfizer Pierre Fabre Roche Vertex Pharmaceuticals 7.2 Technology Companies C4 Therapeutics Crossfire Oncology Cullgen EpiBiologics Firefly Bio InduPro Lycia Therapeutics NanoMedSyn NIBEC Nurix Therapeutics Orum Therapeutics Prelude Therapeutics Ubix Therapeutics VectorY Therapeutics 8 Profiles of Degrader-Antibody Conjugate Technologies AMFA CELMoD ADC Degraducer DELigase EpiTAC EPriL LYTAC & GELYTAC PROTAB PROxAb REULR TORPEDO TPD2 uSMITE VecTron For more information about this report visit https://www.researchandmarkets.com/r/aj64k4 About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Orum to receive a $15 million upfront payment and up to $310 million in potential option fees and milestone payments per target plus tiered royalties BOSTON, MA, USA & DAEJEON, South Korea I July 16, 2024 I Orum Therapeutics (“Orum” or the “Company”), a clinical-stage private biotechnology company pioneering the field of degrader-antibody conjugates (DACs), today announced a global, multi-target license and option agreement whereby it granted Vertex Pharmaceuticals (“Vertex”) (Nasdaq: VRTX) rights to conduct research using Orum’s Dual-Precision Targeted Protein Degradation (TPD² ® ) technology for the discovery of novel targeted conditioning agents for use with gene editing. Following the research period for each target, Vertex will have the option to obtain a worldwide, exclusive license to research, develop, manufacture, and commercialize DACs developed with Orum’s TPD² technology for that target. Under the terms of this agreement, Orum will receive an upfront payment of $15 million and is eligible to receive additional option payments and milestones potentially totaling up to $310 million per target for up to three targets, as well as tiered royalties on potential future global annual net sales. Vertex is responsible for all research, development, and commercialization. “Vertex is a leader in discovering and developing innovative medicines, including being the first to receive FDA approval of a CRISPR/Cas9 gene-edited therapy, and we are pleased they’ve selected Orum’s TPD² technology to discover novel targeted conditioning agents,” said Sung Joo Lee, Ph.D., CEO and founder of Orum Therapeutics. “This agreement with Vertex creates the potential to treat patients in a novel indication space with our leading targeted protein degradation approach for an exciting new therapeutic class of degrader-antibody conjugates.” About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry’s top places to work, including 14 consecutive years on Science magazine’s Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com or follow us on LinkedIn , Facebook , Instagram , YouTube and Twitter/X . About Orum’s TPD² Approach Orum’s unique Dual-Precision Targeted Protein Degradation (TPD²) approach builds novel targeted protein degraders combined with the precise tumor cell delivery mechanisms of antibodies to generate innovative, first-in-class, tumor-selective TPDs for the treatment of cancer. Orum has developed new molecular glue degrader payloads to specifically degrade an intracellular target protein within cancer cells via the E3 ubiquitin ligase pathway. Conjugated to antibodies, the payloads are designed to be delivered specifically to cancer cells and degrade the intracellular target protein and cause tumor cell death. About Orum Therapeutics Orum Therapeutics is a private, clinical-stage biotech pioneering the development of cell-specific, targeted protein degraders (TPD² ® ) and stabilizers (TPS²™) with the precision of antibody targeting to improve cancer treatment for more patients. The lead therapeutic candidate from the TPD² GSPT1 platform, ORM-5029, is in clinical development for the treatment of HER2-expressing solid tumors. The TPS² approach uses proprietary Cbl-b inhibitor payloads conjugated to immune cell- or tumor cell-targeting antibodies. Orum is located in Lexington, MA, US, and Daejeon, South Korea. For more info, visit www.orumrx.com . SOURCE: Orum Therapeutics