Modus Therapeutics AB

▎药明康德内容团队编辑本期看点1. 用于治疗粘多糖病II型患者的基因疗法RGX-121使患者持续获益已达三年，有望明年递交上市申请。2. 疫苗组合疗法在治疗HIV的临床试验中展现积极疗效，使正在接受抗逆转录病毒治疗的HIV感染者无需接受抗逆转录病毒治疗（ART）治疗的时间延长。3. 安斯泰来用于治疗迟发型庞贝病的基因替代疗法积极数据公布，患者停用酶替代疗法51周时的功能检测结果依然保持稳定。4. 治疗法布里病的基因疗法ST-920早期临床数据公布，观察到关键生物标志物α-半乳糖苷酶A活性的持续升高，最长的已持续超过两年。药明康德内容团队整理RGX-121：公布1/2/3期临床试验数据REGENXBIO公司宣布了其候选基因疗法RGX-121用于治疗19岁以下的II型黏多糖贮积症（MPS II）患者的1/2/3期试验的积极数据。MPS II是一种罕见的神经退行性溶酶体贮积症，由溶酶体酶艾杜糖酸-2-硫酸酯（I2S）酶缺乏引起。这种关键酶活性的降低或丧失会导致糖胺聚糖（GAG）在溶酶体的积累，从而引起溶酶体功能障碍和神经退行性病变。目前该病的标准疗法为酶替代疗法。RGX-121是一种一次性的基因疗法，旨在使用AAV9载体将编码I2S酶的人艾杜糖酸-2-硫酸酯酶（IDS）基因递送至中枢神经系统（CNS）。此次公布的结果显示，RGX-121在3种剂量水平下均具有良好的耐受性，没有药物相关的严重不良事件（SAEs）。一次性给予RGX-121后，患者脑脊液（CSF）中GAGs呈剂量依赖性的减少令人鼓舞。其中，队列3（使用关键计划剂量水平）患者的CSF GAGs的降幅最大，在给药后第48周时已接近正常水平。此外，在RGX-121给药三年后，患者的神经发育和日常活动技能也获得了持续的改善。该公司表示计划在2024年递交生物制品许可申请（BLA）。HTI：公布1/2a期临床试验数据AELIX Therapeutics公司公布了其与吉利德科学（Gilead Sciences）合作开展的一项名为AELIX-003的临床研究的数据。该研究旨在评估接受ART的HIV感染者使用含有AELIX的HTI T细胞疫苗和吉利德科学的Toll样受体7（TLR7）激动剂vesatolimod治疗方案的安全性、耐受性、免疫原性和疗效。Vesatolimod是一种在研强效选择性口服TLR7激动剂，可直接激活浆细胞样树突状细胞。在这项研究中，vesatolimod会激活体内HIV的表达，增强疫苗诱导的免疫反应，从而消除病毒感染的细胞。T细胞疫苗的HTI免疫原含有特异性HIV抗原区域，那些具有“控制器表型”（controller phenotypes）的患者对于该区域有丰富的T细胞免疫反应，这意味着它们可以在不使用抗逆转录病毒药物的情况下极大程度地控制HIV。HTI疫苗旨在将免疫反应重新聚集于HIV中特别易受攻击的部位，包括从潜伏病毒库中激活的病毒。在这项研究中，所有受试者接受疫苗/vesatolimod治疗后将在仔细监测下暂停使用抗逆转录病毒药物，以确定干预措施是否能有效控制HIV水平。结果显示，该研究达到了安全性、耐受性和免疫原性的主要和次要终点。HTI疫苗与vesatolimod联用可诱导高水平的HTI特异性T细胞反应，使HIV感染者无需ART的时间延长。病毒库高的疫苗接种者能够在没有ART的情况下维持病毒缓解24周，而所有病毒库高的安慰剂组受试者在前12周内就需要开始ART治疗。AT845：公布1/2期临床试验数据安斯泰来（Astellas Pharma）更新了其正在进行的1/2期临床试验FORTIS的初步安全性和疗效数据，该试验评估了其在研腺相关病毒（AAV）基因替代疗法AT845在迟发型庞贝病（LOPD）成人患者中的安全性、耐受性和疗效。庞贝病是由于缺乏酸性α-葡萄糖苷酶（GAA）引起的遗传性溶酶体储存障碍，GAA的水平降低或缺失导致肌肉和其他组织溶酶体中底物糖原的积累，因此又称为2型糖原累积病。这是一种会造成骨骼肌与心肌失能、通常为致命性的罕见疾病。目前，酶替代疗法（ERT）是该病唯一的获批疗法，这是一种慢性治疗，每两周需输注一次，完全依赖于组织从血浆中摄取GAA。此次公布的数据显示，4名受试者中有3名在AT845给药后停止使用ERT，在停用ERT 19、44和51周时，他们的功能检测结果保持稳定。对接受过治疗的受试者（包括中止ERT的受试者）的持续评估显示，疾病功能终点（包括用力肺活量和6 min步行测试）持续保持稳定。所有受试者对AT845输注的耐受性良好，大多数治疗中出现的不良反应为轻度（1级），并被认为与治疗无关。ST-920：公布1/2期临床试验的新数据Sangamo Therapeutics公司于近日公布了其用于治疗法布里病（Fabry disease）患者的基因疗法ST-920的1/2期临床试验STAAR的最新初步结果。法布里病是一种溶酶体贮积症，由α-半乳糖苷酶A基因（GLA）突变引起，从而导致α-Gal A活性缺乏，无法正常代谢鞘氨醇三聚己糖苷（Gb3）。Gb3在体内的积聚可以对许多重要器官造成破坏，例如肾脏、心脏、神经、眼睛、肠道以及皮肤。ST-920是一款一次性输注、肝脏靶向的基因疗法。此疗法通过AAV向患者肝脏递送功能性GLA基因拷贝，从而使细胞恢复生产α-Gal A。此次更新的数据截至2022年10月20日，在13名患者中观察到α-Gal A活性的持续升高，最长的已持续超过两年。在6个月时接受首批肾脏活检的患者中，其中1名患者的Gb3的清除率达78%，肾小球足细胞损失减少77%。所有剂量递增试验中的患者均不用再接受ERT，至今不需要恢复ERT治疗。身体健康状况SF-36评分显示，患者的平均一般健康评分在临床意义和统计学意义上均有显著增加。该公司计划将于2023年针对该疗法开展3期试验。FBX-101：公布1/2期临床试验的新数据Forge Biologics公布了其新型基因疗法FBX-101在Krabbe病患者中开展的1/2期临床试验RESKUE的初步数据。Krabbe病是由一种负责分解某类脂质（如psychosine）的溶酶体酶半乳糖甘油糖苷酶（GALC）的基因功能缺失突变引起的。如果GALC功能缺失，psychosine在细胞中的毒性积累会导致大脑和周围神经系统的髓鞘细胞死亡，进而引起患者运动功能的丧失。目前，骨髓移植是治疗中枢神经系统性Krabbe病患者的标准治疗，但患者依然会发展为周围神经系统疾病，并最终导致死亡。FBX-101是一种腺相关病毒血清型rh10（AAVrh10）基因疗法，通常在造血干细胞移植后进行。FBX-101能够向中枢和外周神经系统中的细胞递送GALC基因的功能拷贝，已被证明能在功能上纠正中枢和周围神经病变和与Krabbe病相关的行为障碍，并显著提高该病动物模型的寿命。之前公布的1/2期试验结果显示，接受FBX-101治疗6个月时，患者白细胞中GALC的活性显著提高，运动功能得到改善，且大脑正常发育。Forge公司此次公布了患者接受治疗12个月时的随访数据，研究人员在患者的血浆和CSF中观察到GALC转基因的持续高表达，且患者的运动功能和大脑发育保持正常。此外，尚未观察到与治疗相关的SAE、肝酶升高或抗AAVrh10抗体的产生。IMC-M113V：公布1期临床试验数据Immunocore公司公布了其在研TCR疗法IMC-M113V的安全性和活性数据。这是一种双特异性可溶性TCR疗法，用于治疗HIV病毒感染者。IMC-M113V建立在Immunocore公司旗下ImmTAX技术基础上，旨在消除持续被艾滋病毒感染的CD4+细胞（“储库”）。IMC-M113V的靶点是Gag蛋白的肽段，该蛋白由被HIV感染的细胞表面HLA*A02递呈。减少这些细胞的数量是在没有抗逆转录病毒药物的情况下可能达到病毒抑制状态的一种方法，有望实现功能性治愈。此次公布的是一项开放标签的1/2期临床试验，旨在确定IMC-M113V治疗HIV感染者的安全性和生物活性的剂量，这些患者的HLA-A*02:01呈阳性、接受抑制性抗逆转录病毒疗法不超过7年。结果显示，所有的3种剂量下的IMC-M113V均表现出良好的耐受性，没有严重的不良事件，没有血液学或化学方面的明显变化，也没有出现细胞因子释放综合征（CRS）或神经毒性。在整个用药和随访过程中，血浆病毒载量保持抑制。在接受15 mg剂量的患者中有一半达到了预先设定的药效学活性指标。Allocetra：获西班牙药品和医疗器械管理局批准开展1/2期临床试验Allocetra是Enlivex Therapeutics公司正在开发的一种通用的现货型细胞疗法，旨在将巨噬细胞重新编程为稳态状态。将非稳态的巨噬细胞重编程为稳态状态对于重新平衡免疫系统和解决危及生命的疾病至关重要。在临床前研究中，使用Allocetra联用免疫检查点抑制剂（PD-1抑制剂或CTLA-4抑制剂）使患有卵巢癌的小鼠及患有腹膜间皮瘤的小鼠获得了具有统计学意义的生存获益。此前，Allocetra已在美国和以色列获批开展临床试验。DNL310：公布1/2期临床试验的新数据Denali Therapeutics公司公布了其针对MPS II开展的在研酶替代疗法DNL310的1/2期临床试验的新中期数据。DNL310是一款由IDS蛋白与Denali的酶转运载体（ETV）结合的融合蛋白药物。Denali的转运载体平台含有可与天然转运受体结合的工程化Fc片段，通过与在血脑屏障上表达的天然转运受体结合，借着转胞作用（transcytosis）能够将大分子递送到大脑。DNL310被设计为可以递送IDS蛋白至包含脑部的全身细胞与组织的溶酶体中，以对GAG进行分解。此次更新的数据显示，在接受超过49周的DNL310治疗后，研究人员观察到患者在多个量表评分上的积极变化，包括Vineland适应行为量表第二版（VABS-II）、用于评估认知能力的贝利婴幼儿发展量表（BSID-III）以及总体印象量表。听觉脑干反应测试的结果显示，DNL310能够改善患者的听力。其他生物标志物数据表明，DNL310能够使脑脊液中的硫酸乙酰肝素快速并持续正常化至正常健康水平，并改善溶酶体功能相关的生物标志物。目前，使用DNL310治疗的数据已长达两年，其安全性依旧与标准护理保持一致。BXQ-350：公布1期临床试验数据Bexion Pharmaceuticals公司公布了其S1P激活剂BXQ-350在患有复发性实体瘤（包括复发性恶性脑肿瘤）的儿童和年轻人中的1期临床试验数据。BXQ-350是一种由多功能、溶酶体激活蛋白Saposin C和磷脂酰丝氨酸组成的制剂，在体外和体内显示出临床前抗肿瘤作用，特别是在结直肠、脑和其他实体瘤中。Bexion公司已完成两个针对成人和儿科人群的单药1期试验。BXQ-350在这些项目中表现出强大的安全性，并证明了BXQ-350单药在一系列肿瘤中具有活性，包括多形性胶质母细胞瘤（GBM），结直肠癌和阑尾癌。此外，其他临床和非临床数据表明，BXQ-350在化疗诱导的周围神经病变中同样具有活性。BXQ-350在此次公布数据的研究中达到了安全性和最大计划剂量的目标，支持Bexion公司在新诊断的弥漫性内源性脑桥胶质瘤（DIPG）和弥漫性中线胶质瘤（DMG）中开展儿科1期研究。BA3182：IND申请获得FDA许可BA3182是BioAtla公司开发的条件激活的生物（CAB）EpCAM/CD3双特异性T细胞接合抗体，包含两个EpCAM结合位点和两个CD3ε结合位点，用于治疗晚期腺癌。EpCAM在实体瘤中的高水平表达使其成为抗体分子治疗上皮癌，特别是腺癌的有吸引力的靶标。然而，EpCAM在正常细胞中也非常普遍，传统的EpCAM/CD3双特异性抗体的使用会面临剂量限制毒性问题。体外研究表明，CAB双特异性抗体BA3182与靶蛋白人EpCAM和人CD3结合，在肿瘤微环境（TME）的特征性酸性条件下具有高特异性和亲和力，而在正常细胞的碱性生理条件下（pH 7.4）的结合力大大降低。体内研究表明，BA3182相比于非CAB变体的治疗指数提高了100倍以上。在获得了FDA的IND批准后，BioAtla公司将进行一项首次人体1期研究，以评估BA3182在晚期腺癌患者中的安全性、药代动力学（PK）和有效性。AL01211：公布1期临床试验的初步数据AceLink Therapeutics公司公布了其用于治疗法布里病的新型GCS抑制剂在健康受试者中的1期试验的初步数据。AL01211具有优秀的效力（个位数纳摩尔半抑制浓度IC50）、高选择性和其他良好的、可支持每日一次口服的药物特性，为目前需频繁静脉注射ERT的法布里病患者提供了口服小分子疗法的潜在选择。此次公布的结果显示，AL01211在健康受试者中的安全性和耐受性良好，并能使血浆药效学生物标志物呈剂量依赖性的降低。BX004：公布1b/2a期临床试验数据BiomX公司公布了其噬菌体鸡尾酒疗法BX004用于治疗铜绿假单胞菌引起慢性肺部感染的囊性纤维化（CF）患者的安全性和有效性数据。铜绿假单胞菌是引起CF患者肺部感染和死亡的主要因素。BX004旨在靶向和破坏铜绿假单胞菌。研究结果显示，BX004治疗在所有患者和剂量水平上均安全且耐受性良好。研究人员还观察到了初步的疗效信号，相比于安慰剂组，接受BX004治疗的CF患者的细菌负荷显著减少。CDX-7108：公布1期临床试验数据Codexis公司与Nestlé Health Science公司共同公布了一项1期临床试验的数据，该试验旨在评估其候选产品CDX-7108的安全性，耐受性，PK和药效学。CDX-7108是一种脂肪酶变体，专门设计用于克服现有胰酶替代疗法（PERT）的局限性。PERT是胰腺外分泌功能不全（EPI）的主要治疗方法，这是一种使人衰弱的胃肠道疾病，由损害胰腺功能的疾病引起，例如胰腺炎、胰腺癌、克罗恩病、乳糜泻和囊性纤维化。CDX-7108对胃中的酸性条件高度稳定，并对上肠中的蛋白酶具有抗性。此次公布的初步数据显示，与安慰剂相比，接受CDX-7108的患者的脂质吸收有所改善。此外，在参与单次剂量递增和多次剂量递增研究的48名受试者中没有发现安全问题。Sevuparin：公布1b期临床试验数据Modus Therapeutics公司公布了其创新专有多糖药物sevuparin用于治疗败血症的早期临床试验的积极数据。Sevuparin具有多模式的作用机制，包括抗炎，抗粘附和抗聚集作用，是一种具有明显减弱的抗凝血特征的类肝素，与常规类肝素相比，可以给予高几倍的剂量，而没有相关的出血副作用风险。此次公布的试验结果显示，在脂多糖（LPS）诱导产生短暂炎症反应的健康受试者中，所有3种剂量水平下的sevuparin都是安全的，且耐受性良好。此外，sevuparin治疗使受试者的某些白细胞群水平呈剂量依赖性增加，在统计学上具有显著性，并能使LPS诱导的呼吸频率增加呈剂量依赖性的抑制。这些发现表明sevuparin有潜力作为全身炎症（包括败血症和感染性休克）的治疗方法。CD40.HIVRI.Env：公布1期临床试验的中期数据LinKinVax公司公布了其靶向树突状细胞的候选疫苗CD40.HIVRI.Env在健康受试者中的1期安全性和免疫原性结果。树突状细胞是免疫系统的关键激活剂，CD40.HIVRI.Env是一种将HIV包膜蛋白与特异性靶向树突状细胞表面CD40受体的单克隆抗体结合而成的候选疫苗，旨在预防艾滋病毒感染。此次公布的数据显示，该候选疫苗的安全性和耐受性良好，且能够诱导早期、显著和持续的免疫反应。大家都在看药明康德为全球生物医药行业提供一体化、端到端的新药研发和生产服务，服务范围涵盖化学药研发和生产、生物学研究、临床前测试和临床试验研发、细胞及基因疗法研发、测试和生产等领域。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看）[1] LinKinVax Welcomes Interim Results of the ANRS VRI06 Phase I Trial Evaluating a Novel HIV Vaccine Candidate. Retrieved February 24, 2023, from https://www.businesswire.com/news/home/20230222005313/en/[2] BiomX Announces Positive Results from Part 1 of the Phase 1b/2a Study Evaluating BX004 for Treatment of Chronic Pulmonary Infections in Patients with Cystic Fibrosis. Retrieved February 24, 2023, from https://www.globenewswire.com/news-release/2023/02/22/2612975/0/en/BiomX-Announces-Positive-Results-from-Part-1-of-the-Phase-1b-2a-Study-Evaluating-BX004-for-Treatment-of-Chronic-Pulmonary-Infections-in-Patients-with-Cystic-Fibrosis.html[3] AELIX has presented promising top-line results from the clinical study AELIX-003 at the 2023 Conference on Retroviruses and Opportunistic Infections (CROI). Retrieved February 24, 2023, from https://www.aelixtherapeutics.com/aelix-therapeutics-presents-positive-results-from-randomized-placebo-controlled-phase-iia-therapeutic-hiv-vaccine-and-immune-modulator-combination-clinical-trial/[4] Immunocore announces initial Phase 1 safety and pharmacodynamic activity data with first soluble TCR therapy for people living with HIV，Retrieved February 2rd, 2023 from https://ir.immunocore.com/news-releases/news-release-details/immunocore-announces-initial-phase-1-safety-and-pharmacodynamic[5] Modus Therapeutics announces positive topline data from its Phase 1b LPS provocation study evaluating the potential of sevuparin for treatment of sepsis. Retrieved February 2rd, 2023 from https://www.modustx.com/press-release?releaseIdentifier=bef7616d-cdc3-4eae-830e-01a83e23f4af[6] Astellas Announces Update on Preliminary Safety and Efficacy Data from FORTIS Study of Investigational AT845 in Adults with Late-Onset Pompe Disease. Retrieved February 24, 2023, from https://www.prnewswire.com/news-releases/astellas-announces-update-on-preliminary-safety-and-efficacy-data-from-fortis-study-of-investigational-at845-in-adults-with-late-onset-pompe-disease-301750884.html[7] Additional Positive Interim Data from Phase I/II/III CAMPSIITE™ Trial of REGENXBIO's RGX-121 for the Treatment of MPS II (Hunter Syndrome) Presented at 19th Annual WORLDSymposiumTM. Retrieved February 24, 2023, from https://www.prnewswire.com/news-releases/additional-positive-interim-data-from-phase-iiiiii-campsiite-trial-of-regenxbios-rgx-121-for-the-treatment-of-mps-ii-hunter-syndrome-presented-at-19th-annual-worldsymposiumtm-301753564.html[8] Sangamo Therapeutics Announces Evidence of Clinical Benefit in Phase 1/2 STAAR Study in Fabry Disease. Retrieved February 24, 2023, from https://www.businesswire.com/news/home/20230222005671/en[9] Denali Therapeutics Announces New Interim Data from DNL310 Phase 1/2 Study for MPS II and DNL126 Preclinical Data for MPS IIIA at WORLDSymposium™. Retrieved February 24, 2023, from https://www.denalitherapeutics.com/investors/press-release?id=9226&type=api[10] Codexis and Nestlé Health Science Announce Interim Results from Phase 1 Clinical Trial of CDX-7108 for Exocrine Pancreatic Insufficiency. Retrieved February 25, 2023, from https://www.globenewswire.com/news-release/2023/02/23/2614809/24825/en/Codexis-and-Nestl%C3%A9-Health-Science-Announce-Interim-Results-from-Phase-1-Clinical-Trial-of-CDX-7108-for-Exocrine-Pancreatic-Insufficiency.html[11] Bexion Pharmaceuticals, Inc. Announces Publication of Pediatric Phase 1 (KOURAGE) Results. Retrieved February 25, 2023, from https://www.prnewswire.com/news-releases/bexion-pharmaceuticals-inc-announces-publication-of-pediatric-phase-1-kourage-results-301754621.html[12] BioAtla Announces FDA Clearance of Investigational New Drug Application for BA3182, a CAB-EpCAMxCAB-CD3 Bispecific T-Cell Engager for the Treatment of Advanced Adenocarcinoma. Retrieved February 25, 2023, from https://ir.bioatla.com/news-releases/news-release-details/bioatla-announces-fda-clearance-investigational-new-drug-0[13] AceLink Presents Phase 1 Study of AL01211 to Treat Fabry Disease at WORLD Symposium. Retrieved February 25, 2023, from https://www.businesswire.com/news/home/20230223005278/en/[14] Enlivex Receives Clearance From Spanish Agency of Medicines and Medical Devices For Treatment of Patients with Advanced Solid Malignancies in the Ongoing Allocetra Phase I/II Clinical Trial. Retrieved February 25, 2023, from https://enlivex.com/investor-relations/#latest-news[15] Forge Biologics Reports Positive FBX-101 Clinical Updates for Patients with Krabbe Disease at WORLDSymposium. Retrieved February 25, 2023, from https://www.businesswire.com/news/home/20230223005450/en/免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

STOCKHOLM, Sweden I February 21, 2023 I Modus Therapeutics AB (“Modus”), a company developing innovative treatments for patients with major unmet medical needs, announces positive top-line data from its Phase 1b lipopolysaccharide (LPS) provocation study, a key step in evaluating the potential of its lead asset, sevuparin, as a treatment for sepsis and other conditions with systemic inflammation. In this study, healthy volunteers received LPS to induce a transient systemic inflammation reaction together with one of three dose levels of sevuparin, or placebo for 6 hours. They were then followed up at 24 hours post treatment. Provocation with LPS is a well-established model used to characterize the early stages of septic inflammation by provoking a range of measurable symptoms. All three dose levels of sevuparin were found to be safe and well tolerated throughout the study period, confirming a favorable safety profile of the candidate drug under induced inflammatory conditions. Furthermore, sevuparin treatment induced statistically significant and dose-dependent increases in the levels of certain white blood cell populations as well as a dose-dependent inhibition of the increase in respiratory rate induced by LPS. These findings are indicative of clinically relevant and immunomodulatory effects exerted by sevuparin in a state of systemic inflammation. The study outcome strengthens the potential for sevuparin as a treatment for systemic inflammation including sepsis and septic shock. This is an area of high unmet medical need as current treatment options fail to address the high disease burden of these critically ill patients. Sevuparin also demonstrated a favorable safety and tolerability profile when combined with the blood thinning heparin (enoxaparin), which is an important standard of care in severely ill patient populations that need thrombosis prophylaxis. The positive top-line data from this trial will be used to design the Modus Phase 2a study of sevuparin in patients with sepsis. For example, this data will inform the dose of sevuparin to be assessed, the dosing schedule and the patient population for the planned patient study. John Öhd, Chief Executive Officer of Modus Therapeutics commented: “We are delighted by the encouraging results from our LPS-challenge study, a very important milestone in our mission to develop sevuparin as a fundamental change in the treatment for sepsis and other conditions with systemic inflammation. The results enhance our understanding of the immunomodulatory action of sevuparin and reinforce its potential in this area of extremely high unmet need. These data will also allow us to develop an optimized trial protocol for our planned Phase 2a trial in sepsis patients. We would like to thank all our collaborators, who were critical to the success of this initial clinical study, for their continued support, and we look forward to providing more updates in the future.” The Phase 1b study was conducted in collaboration with The Centre for Human Drug Research in Leiden, The Netherlands, an independent Contract Research Organisation (CRO), which specializes in advanced early clinical drug research based on its leading expertise in inflammation models. About Modus Therapeutics and sevuparin Modus Therapeutics is a Swedish biotechnology company headquartered in Stockholm that develops sevuparin with a focus on diseases with a high unmet medical need. The company's focus in the near future is to develop sevuparin for patients with sepsis / septic shock, which is a serious and often fatal condition. Modus Therapeutics is listed on the Nasdaq First North Growth market (“MODTX”). More information is available at www.modustx.com . Sevuparin is a clinical stage, innovative proprietary polysaccharide drug with a multimodal mechanism of action, including anti-inflammatory, anti-adhesive and anti-aggregate effects. Sevuparin is a heparinoid with markedly attenuated anti-coagulation features that allows severalfold higher doses to be given, compared to regular heparinoids, without the associated risk for bleeding side-effects. Two routes of administration of sevuparin are currently being tested – an IV formulation for in-patient administration and a subcutaneous formulation that allows ambulatory and home care administration. SOURCE : Modus Therapeutics

→ Sam Samad is out as CFO of Illumina as chief strategy and corporate development officer Joydeep Goswami slides in for him on an interim basis starting July 8. Goswami joined Francis deSouza ’s crew in 2019 as SVP of corporate development and strategic planning after his time as president of Thermo Fisher ’s clinical next-generation sequencing (NGS) and oncology business unit. Guardant Health , a primary liquid biopsy screening test competitor, filed a motion to dismiss Illumina’s case against the company in late May. Its co-founders and co-CEOs, Helmy Eltoukhy and AmirAli Talasaz , are former staffers at Illumina, which is alleging that the pair infringed on intellectual property to develop Guardant’s technology. “Illumina’s lawsuit frivolously challenges our ownership and authorship of our intellectual property, which is the backbone of our vital work,” Guardant Health general counsel John Saia said in a March statement. → Swiss gene therapy player Anjarium Biosciences jumped into the gene therapy 2.0 fray with a Series A in September 2021 that featured contributions from Abingworth , Gimv and Pfizer ’s venture arm, and this week Stephen Yoo has hitched a ride as CEO. Yoo held clinical development posts at MedImmune / AstraZeneca before taking the CMO job at Regenxbio , and since 2019 he was CMO and at Third Harmonic Bio , a role presently occupied by Edward Conner . → Massachusetts solid tumor biotech Xilio Therapeutics has promoted Martin Huber to president of the company. Even with his new role, Huber will still maintain responsibilities as president of R&D. Huber joined Xilio after a stint as SVP and CMO of Tesaro (acquired by GSK ). Earlier on in his career, Huber was SVP, oncology clinical research at Merck and held roles at Schering-Plough , Roche and Rhone-Poulenc Rorer . Huber currently sits on the boards of Mersana Therapeutics and Syndax Pharmaceuticals . In addition to Huber’s promotion, Xilio is pulling out some seats on its board for former Alexion and Biogen CFO Paul Clancy as chair (succeeding Dan Lynch , who is transitioning to strategic advisor of the company) and Surface Oncology CEO Robert Ross . → With former Rafael Holdings boss Ameet Mallik running the show, ADC Therapeutics has installed David Gilman as chief business & strategy officer. Gilman spent his short snippet of time at Novartis Oncology as global head of portfolio strategy and business development, overlapping with Mallik’s run as head of US operations. Gilman eventually left Novartis in 2019 to become a partner at ClearView Healthcare Partners . → Replimune phased out Andrea Pirzkall ’s position in 2021 after a year on the job “without cause,” but she has since resurfaced at San Francisco-based Asher Bio as CMO. Before her stint at Replimune, Pirzkall was BeiGene ’s executive director of clinical development and also spent a decade at Genentech . Asher Bio garnered the support of Third Rock and RA Capital with its $108 million Series B last September as it develops its lead candidate, a CD8-targeting IL-2. → Alex Zhavoronkov’s global AI player Insilico Medicine — which days ago added a robotics lab to the company with a fresh injection of $60 million — is bumping up their CMO Feng Ren into the role of co-CEO. Ren will be in charge of the development side of things as the company begins testing more of their drugs in humans while Zhavoronkov will handle the AI portion of the company. Ren, a Harvard grad, joined the company last February after a stint in Shanghai. Earlier in his career, Ren spent 11 years with GSK R&D China, culminating in his role as head of chemistry. → Deciphera sang the restructuring blues after a late-stage trial fail with Qinlock last fall, sending 35% of its workforce packing in a move that CEO Steve Hoerter hopes will keep the lights on through 2024. As they press forward, Big Pharma vet Kelley Dealhoy has stepped in as CBO. In a total of 10 years with Novartis, Dealhoy held such roles as VP of US business development for the pharmaceutical division, global head of business development and M&A for the consumer division, and more recently, VP of business development for the oncology division. → Colin McCracken is moseying down to synthetic bio company Evonetix to take over the reins as CEO. McCracken joins the Cambridge, UK-based company from Fluidigm , where he served as SVP and CCO. Prior to that stint, McCracken had gigs at Thermo Fisher (VP and general manager and VP of sales); Life Technologies (VP and head of European sales); and Qiagen (national sales manager). → The heat is on … and then it wasn’t, when Heat Bio rebranded to NightHawk Biosciences in April. NightHawk has tapped Stephan Kutzer as interim CEO of its CDMO subsidiary Scorpion Biological Services , which will be building a new biomanufacturing site in the Little Apple of Manhattan, KS. After 18 years at Lonza , 10 of those as president, divisional CEO and COO of the pharma biotech & customer manufacturing division, Kutzer held the top spot at Alcami Corporation , a company he also chaired. → Merck KGaA is promoting Larisa Amir to the role of managing director of its healthcare R&D center in Israel. Amir joined Merck KGaA in 2009 and served in a number of roles before culminating in her last position as global head of products quality LCM. Amir is also a member of the Merck Global Healthcare Discovery Technology Leadership team. Prior to joining Merck, Amir was COO at Procognia . → On July 1, Melissa Bradford-Klug will debut as COO of Akari Therapeutics , now helmed by former Enzyvant chief Rachelle Jacques . Bradford-Klug has worked in the rare disease space for Greater China as president and CBO of RareStone Group since January 2020; the former Mallinckrodt and AMAG Pharmaceuticals vet has CEO experience with Keryx Biopharmaceuticals and women’s health-focused Mayfield Pharmaceuticals . → Before striking a $25 million immuno-oncology deal with Exelixis , Sweden’s BioInvent named Sylvie Ryckebusch as CBO, diving into an executive position after assisting BioInvent part-time since 2019. In her 12 years at Merck KGaA / EMD Serono , Ryckebusch was head of neurology and autoimmune disease licensing, business development. BioInvent also added Forbion ’s Nanna Lüneborg and Redmile Group ’s Natalie Berner to the board of directors on Thursday, the same day the Exelixis deal was announced. → TCR upstart Marengo Therapeutics already added some gravitas to the team with FDA and Sana vet Ke Liu ’s appointment as chief development officer, and it’s CFO Svetlana Makhni who takes center stage a month later. Makhni, the ex-finance chief of Bierman and Escient Pharmaceuticals , cut her teeth at Blackstone , Westbrook Partners , Goldman Sachs and BMO Capital Markets . In November 2021, Apple Tree Partners poured $80 million into creating Marengo, a biotech taking aim at Vβ TCR variants. → Domain Therapeutics , the French-Canadian GPCR player which bagged a $42 million Series A back in May, has promoted Xavier Leroy to CSO, who first joined the startup back in 2019 as chief technology officer. Leroy has also served as head of drug discovery at iTeos Therapeutics and owns GPCR experience from his 13 years at Actelion . → Helsinki-based Valo Therapeutics (not to be confused with Flagship ’s Valo Health ) has picked up Hemanshu Shah as CBO. Shah has Big Pharma experience galore from 13 years at Bristol Myers Squibb and a year as J&J ’s global commercial leader, oncology. From 2012-14, he was VP of global marketing and global therapeutic head for transplantation, immunology & infectious diseases with Astellas , and before accepting this role, Shah served as VP, corporate development at Transgene . → Hangzhou, China-based Ascletis — the first pre-revenue biotech to list on the HKEX in 2018 — has rolled out the welcome mat for John Gargiulo as CBO. A key cog in the US business operations of Daiichi Sankyo , Gargiulo concluded his 18 years with AstraZeneca’s Enhertu partner as North American president and CEO, and from there, he was a principal for GPG Enterprises . → Briggs Morrison did a switcheroo with Michael Metzger at Syndax Pharmaceuticals in February, as Morrison ceded his CEO post to Metzger to take on the role of president and lead R&D. Against that backdrop, Syndax’s CFO vacancy has been filled by Keith Goldan , who leaves the same role at Optinose just as the biotech gained more traction with its nasal spray Xhance for patients with chronic sinusitis in a second Phase III trial. → Attempting to separate itself in a burgeoning protein degradation field, RA Capital -backed Avilar Therapeutics has introduced Adam Muzikant to the squad as CBO. Muzikant was at the negotiating table as business chief of Flexion Therapeutics when the Zilretta maker was sold to Pacira Biosciences in October 2021. Before joining Flexion, Muzikant was a business development exec with AMAG Pharmaceuticals and Synta Pharmaceuticals . → Riverside Partners’ portfolio company Syner-G BioPharma Group is ushering in Ron Kraus as CEO, succeeding Prabu Nambiar . Kraus joins from Cytel , where he served as COO. Prior to that, Kraus spent two decades at Parexel International in a variety of roles including global head of Parexel Consulting . → After jumping on board as CEO of GlycoEra last month, Ganesh Kaundinya has shored up his leadership with a longtime colleague. Tanmoy Ganguly will take on CSO duties at the glycobiology and protein degradation biotech, pivoting from Evelo Biosciences , where he was a VP in charge of pharmacology, research analytics and bioinformatics. Ganguly spent a significant portion of his career at Momenta , which Kaundinya founded in 2001 and was sold to J&J in 2020 for $6.5 billion. → Heavy nova : There’s a pair of promotions at Newtown, PA cancer biotech Onconova , as Mark Guerin (COO and CFO) and Adar Makovski Silverstein (senior director and head of corporate development) climb the ladder . Guerin tacks on another role after serving as CFO of Onconova since September 2013, with the company’s $78 million IPO predating his arrival by two months. We covered Silverstein’s appointment as director, corporate development last December after her nearly three years in business development, external R&D at Amgen . → Eye disease-focused Clearside Biomedical has recruited Susan Coultas as chief clinical officer. Coultas most recently served as SVP of clinical development at Kala Pharmaceuticals . Before that, Coultas had gigs at Alcon Laboratories , CibaVision Ophthalmics , Sucampo Pharmaceuticals , Bausch + Lomb and InfoQuest Clinical Network . → Durham, NC-based Aerami Therapeutics has appointed Lisa Yañez as COO. Before joining the C-suite at Aerami, Yañez held executive positions at Acceleron (VP of global strategic marketing) and United Therapeutics (VP of marketing, analytics and training). Earlier, she was a product manager with GSK, Allergan and Genentech. → Cary, NC-based Allucent has tapped Katy Moore as president of clinical pharmacology, modeling and simulation. Moore joins with a wealth of experience, having served as global head of clinical pharmacology at ViiV Healthcare ; headed the viral diseases and oncology clinical pharmacology, modeling and simulation teams at GSK; and also led worldwide business development teams at GSK. → Philip Okala has been named system president at City of Hope, filling a crucial role at one of the nation’s leading cancer research centers in September. Okala will run clinical care and research sites under the City of Hope umbrella, such as City of Hope Los Angeles, City of Hope Orange County, Translational Genomics Research Institute (TGen), Beckman Research Institute of City of Hope and Cancer Treatment Centers of America. Okala comes to City to Hope after five years as COO of the University of Pennsylvania Health System. → Dag Nesse has been named director of clinical operations at EpiEndo Pharmaceuticals out of Reykjavik, which raised a modest €20 million in its Series A last summer. Neese most recently served as senior director, head of clinical operations at Calliditas Therapeutics , and he has also served as head of operations at Modus Therapeutics . → Vanessa Salazar has moved to Boston gene therapy biotech STRM.BIO as VP of business development. Prior to joining STRM.BIO, she was executive director of corporate development and strategic partnerships at Passage Bio . Salazar, who started her career with a 16-year run at GSK, was also corporate development lead at Spark Therapeutics . → One day after being slapped with a Form 483 for multiple production issues, German CDMO Rentschler Biopharma is welcoming aboard Kassim Kolia as VP, business development. Kolia comes aboard boasting experience from FinVector & Oy , Eden Biodesign /Allergan, Lonza and Aptuit (part of Evotec ). → Bristol Myers has expanded its board of directors, appointing Deepak Bhatt to the board’s Science & Technology Committee. Formerly with the Cleveland Clinic and VA Boston Healthcare, Bhatt is the executive director of interventional cardiovascular programs at Brigham and Women’s Hospital. → During this week’s convention in San Diego, BIO dipped into the communications realm to appoint Mike Huckman to the board of directors. A three-time Emmy winner, Huckman spent 10 years as the pharmaceuticals reporter at CNBC and is now global practice leader for executive communications at Real Chemistry . → Verve Therapeutics , which just last month became the third biotech — behind Intellia and Editas Medicine — to test its gene editing technology in humans, has made way for Bo Cumbo on its board of directors. Cumbo, the ex- Sarepta chief commercial officer, currently serves as president and CEO of AavantiBio . → You may recall that Wendy Dixon retired from Alkermes ’ board of directors in what the biotech called a“continued refreshment process,” but another opportunity has opened up for her on the board of directors at Iovance . Dixon, the former marketing exec at Bristol Myers and Merck, has seats on the boards of Arvinas and Black Diamond . → Visiox Pharma is bringing aboard Vicente Anido as a strategic advisor. Anido was ousted as CEO at Aerie Pharmaceuticals in September 2021 and was also chief executive at ISTA Pharmaceuticals . → June Bray is taking a seat on the board of directors of Cortexyme, joining at a turbulent time for the San Francisco-based biotech. Most recently, Bray was SVP, global regulatory affairs and medical writing at Allergan, and prior to that, Bray was with Organon and Berlex Laboratories . To say the company has had it rough would be an understatement: A failed attempt to bring forward an unorthodox approach to treating Alzheimer’s has led to a complete remake at the troubled biotech (transitioning its name to Quince Therapeutics ) last month, with Novosteo CEO Dirk Thye and CMO Karen Smith coming aboard to help right the ship. → US-Dutch gamma delta T cell biotech Lava Therapeutics is bubbling up to the Peer Review surface by electing James Noble and Jay Backstrom to the board of directors. Noble co-founded Adaptimmune in 2008 and was CEO until 2019, and Backstrom — the ex-CMO and head of regulatory affairs at Celgene —became a board member at Disc Medicine not too long ago after wrapping up his time leading R&D at Acceleron. → Kathy Dong has been elected to the board of directors at Neuron23 , Nancy Stagliano ’s neuro startup from the folks at Westlake Village BioPartners . A nine-year Gilead vet who held various marketing roles at the California drugmaker, Dong has been the COO of Star Therapeutics since 2019. → Swedish pharma company NorthX Biologics is adding some new faces to its board of directors with the appointments of Lotta Ljungqvist , Richard Bergström , Mathias Uhlén and Lars Backsell . Here’s a quick summary of each new member: Ljungqvist formerly served as president and CEO of GE for the Nordic region and is also the ex-CEO of Cytiva’s Testa Center for Bioprocess Innovation; Bergström was appointed Sweden’s vaccine coordinator during the Covid-19 epidemic and was previously director general of the European Federation of Pharmaceutical Industries and Associations; meanwhile, Uhlén is a professor in microbiology at the Royal Institute of Technology and Backsell is a co-founder and former CEO of Recipharm .