Key Outcomes:
- Riliprubart (Sanofi) – P3 MOBILIZE, CIDP: Trial stopped after interim analysis showed insufficient efficacy likelihood; no safety concerns. No material impact on 2026 guidance
- TransCon PTH (Ascendis Pharma) – P3 PaTHway, Hypoparathyroidism: Wk 182 data show durable efficacy and favorable long-term safety, with normalized calcium, improved kidney function, and sustained QoL benefits
- Selinexor + Ruxolitinib (Menarini/Stemline) – P3 SENTRY, Myelofibrosis: Combo beat ruxolitinib alone on both co-primary endpoints (n=353); spleen response linked to better OS; manageable safety, no new signals
- Camzyos (BMS) – Adolescent oHCM: FDA accepted sNDA with Priority Review for ages 12-<18 (PDUFA Sep 30, 2026), based on P3 SCOUT-HCM meeting its primary endpoint
- SOT106 (SOTIO) – Osteosarcoma: FDA granted ODD to this LRRC15 ADC; strong preclinical activity; FIH trial planned Q2 2026
- AQNEURSA (IntraBio) – Ataxia Telangiectasia: EU label-expansion filing submitted (pending EMA validation); US FDA already granted Priority Review, PDUFA Sep 19, 2026
- ADRX-0405 (Adcentrx) – Advanced Solid Tumors: China’s NMPA cleared the IND, allowing Chinese sites to join the global P1a/1b trial of this STEAP1-targeting ADC in mCRPC, gastric cancer, and NSCLC; dose-escalation completion is expected by Q4 2026
- Lasme-cel (Cellectis) – r/r B-ALL: FDA granted RMAT designation based on P1 BALLI-01 data; first allogeneic CAR-T in a pivotal trial for this indication; P2 now enrolling, final P1 data at EHA 2026
- Tecentriq (Roche) – Stage III dMMR/MSI-H Colon Cancer: FDA granted Priority Review to sBLA based on P3 ATOMIC data (PDUFA Oct 9, 2026); could become first immunotherapy-based adjuvant SOC
- Tzield (Sanofi) – Newly Diagnosed Stage 3 T1D: FDA granted accelerated approval for patients aged 8-17, first disease-modifying therapy in this setting, based on P3 PROTECT beta-cell preservation data; confirmatory P3 BETA-PRESERVE ongoing
- Ouro Medicines Acquisition (Gilead/Lakefront): Deal completed, bringing gamgertamig – a BCMA×CD3 bispecific for severe autoimmune diseases like AIHA and ITP – into Gilead’s pipeline. Upfront payment was $1.675B (split between the two companies) + up to $500M in milestones; Lakefront will run early-stage work while Gilead leads late-stage development and commercialization, with registrational trials targeted for as early as 2027
- Intravacc/SynphaBase Partnership: The two companies will combine synthetic oligosaccharide and glycochemistry expertise with conjugation and GMP manufacturing capabilities to support development of next-generation conjugate vaccines
- Adaptive Innovations – Series A: Raised $50M (co-led by Felicis and Bain Capital Ventures) to expand its AI-powered home healthcare platform, grow engineering/operations teams, and enter new U.S. state markets; total funding to date now $60M
- City Therapeutics – Series B: Raised $99.5M (led by Viking Global Investors) to advance its RNAi pipeline, including programs for thromboembolic disorders and Stargardt disease, plus two more programs expected to enter the clinic by the end of 2026; total funding raised to date exceeds $230M
June 10, 2026
Riliprubart Fails to Meet Efficacy Expectations in P3 MOBILIZE Study
Sanofi reported that the P3 MOBILIZE study of riliprubart in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) who are refractory to SOC treatments has been stopped after an independent data monitoring committee’s interim analysis concluded the study is unlikely to show sufficient efficacy; no safety concerns were identified. Sanofi said it will evaluate continuation of other riliprubart studies, including the P3 VITALIZE study in IVIg-treated CIDP patients, and will work with investigators to wind down MOBILIZE and ensure patient care transitions. The company will thoroughly analyze MOBILIZE data to guide future research, and noted the termination will not have a material financial impact nor change its 2026 guidance.
Source: Sanofi PR Jun 10, 2026
June 13, 2026
Ascendis Pharma Releases Long-Term Phase 3 Success for TransCon PTH in Hypoparathyroidism
Ascendis Pharma released wk 182 results from the completed P3 PaTHway study, which showed that long-term treatment with TransCon PTH (palopegteriparatide) continued to provide durable efficacy and a favorable safety profile in adults with hypoparathyroidism. The therapy continued to replicate the physiological actions of endogenous parathyroid hormone, maintaining normal serum and urine calcium levels, improving kidney function, supporting bone health, and delivering durable quality-of-life benefits.
Long-Term Results through Wk 182 from the P3 PaTHway Study:
Source: Ascendis Pharma PR Jun 13, 2026
June 14, 2026
Menarini Releases P3 SENTRY Data on Selinexor + Ruxolitinib in Myelofibrosis at EHA 2026
Menarini and its subsidiary Stemline released new data from the pivotal P3 SENTRY study, to be presented as a late-breaking oral at EHA 2026 Congress. The study, run by Karyopharm Therapeutics in collaboration with Menarini, tested 60 mg selinexor + ruxolitinib vs ruxolitinib monotherapy in 353 patients with previously untreated myelofibrosis, in a randomized, double-blind, placebo-controlled design with two co-primary endpoints: spleen volume reduction ≥35% (SVR35) and absolute total symptom score (Abs-TSS).
Key Efficacy Results:
*Analysis among patients who completed a spleen assessment or discontinued before wk 36.
*VAF – Variant Allele Frequency
Highlights
A post-hoc analysis of the P3 SENTRY study indicated that SVR35 may be associated with improved OS, and new findings from the P1 SENTRY study support this observation. The spleen volume benefit was consistent across prespecified subgroups, including patients on lower ruxolitinib doses (<15 mg/day). A pre-specified exploratory VAF reduction was more common in the combination arm, hinting at a potential disease-modifying effect.
Safety Profile:
The combination showed a manageable safety and tolerability profile, consistent with the known effects of selinexor + ruxolitinib, and no new safety issues were observed.
Source: Menarini PR Jun 14, 2026
June 01, 2026
FDA Grants Priority Review to Camzyos for Adolescents with Obstructive HCM
BMS announced that the U.S. FDA has accepted for Priority Review a sNDA for Camzyos as a treatment for adolescents aged 12 to <18 years with symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM). If approved, Camzyos would become the first cardiac myosin inhibitor (CMI) available for this patient population. The FDA has assigned a PDUFA target action date of Sep 30, 2026. The submission is supported by data from the P3 SCOUT-HCM study, which met its primary endpoint by demonstrating a clinically meaningful and statistically significant reduction in Valsalva left ventricular outflow tract (LVOT) gradient at Wk 28 vs pbo. The safety profile in adolescents was consistent with that observed in adults, with no patients experiencing a left ventricular ejection fraction (LVEF) below 50%. Study results from the 28-wk study period were presented at the American College of Cardiology Annual Scientific Session & Expo 2026 and simultaneously published in the NEJM.
Source: BMS PR Jun 01, 2026
June 03, 2026
FDA Grants ODD to SOT106 for Osteosarcoma
SOTIO Biotech received ODD for SOT106 to treat osteosarcoma. SOT106 is a next-gen ADC targeting LRRC15, a clinically validated protein broadly expressed across sarcoma subtypes and tumor-associated stroma. Preclinical studies demonstrated strong anti-tumor activity in both soft tissue sarcoma and osteosarcoma models, along with favourable tolerability and a potentially high therapeutic index. The company plans to initiate a FIH trial in Q2 2026 to evaluate the therapy in patients. The designation highlights the need for new treatment options in osteosarcoma, where therapeutic advances have been limited for decades and outcomes remain poor for recurrent or metastatic disease.
Source: SOTIO PR Jun 03, 2026
June 05, 2026
MAA Submission for AQNEURSA’s Label Expansion
IntraBio has submitted an EMA variation application to extend AQNEURSA’s existing EU label (currently approved for NPC) to include treatment of Ataxia‑Telangiectasia (A‑T), positioning it as a first‑in‑class therapy in this ultra‑rare indication within the EEA. The variation application will first undergo EMA validation, and only after successful validation will the formal scientific review process begin. In the U.S., the FDA has accepted IntraBio’s sNDA for AQNEURSA in A‑T and granted it Priority Review, with a PDUFA target action date set for Sep 19, 2026.
Sources: BusinessWire PR Jun 05, 2026
June 08, 2026
China NMPA Clears IND for Adcentrx’s STEAP1-Targeting ADC ADRX-0405 in Advanced Solid Tumors
Adcentrx Therapeutics received IND clearance from NMPA (China) for ADRX-0405, a potential first-in-class ADC targeting STEAP1, enabling the inclusion of Chinese clinical sites in its ongoing global P1a/1b study. The study is evaluating ADRX-0405 in patients with advanced solid tumors, including metastatic castration-resistant prostate cancer (mCRPC), gastric cancer, and NSCLC. ADRX-0405 combines a humanized IgG1 antibody with a novel topoisomerase inhibitor payload using Adcentrx’s proprietary i-Conjugation platform, designed to enhance payload delivery and stability through a drug-antibody ratio of 8 (DAR 8). Preclinical studies demonstrated favourable PK, safety, and anti-tumor activity across multiple tumor models. The company expects the P1a dose-escalation portion of the study to be completed by Q4 2026, while the NMPA clearance is expected to accelerate patient enrolment and expand geographic representation in the study.
Source: Adcentrx Therapeutics PR Jun 08, 2026
June 09, 2026
Cellectis Secures FDA RMAT Designation for Lasme-cel in Relapsed/Refractory B-ALL
Cellectis received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA for lasmecabtagene timgedleucel (lasme-cel), its CD22-targeted allogeneic CAR-T therapy for patients with r/r B-cell acute lymphoblastic leukemia (r/r B-ALL). The designation is supported by P1 BALLI-01 data demonstrating promising efficacy and a manageable safety profile, highlighting lasme-cel’s potential to address a significant unmet need in this heavily pretreated patient population. Notably, lasme-cel is the first allogeneic CAR-T therapy to enter a pivotal trial in r/r B-ALL, and the RMAT status is expected to facilitate closer interactions with the FDA and potentially accelerate its development and review. Cellectis also noted that the pivotal P2 portion of the BALLI-01 study is currently enrolling patients, with final P1 results scheduled for presentation at the 2026 EHA Congress.
Source: GlobeNewswire PR Jun 09, 2026
June 11, 2026
Roche’s Tecentriq Receives FDA Priority Review for Stage III dMMR/MSI-H Colon Cancer
Roche reported that the U.S. FDA has accepted and granted Priority Review to its sBLA for Tecentriq and Tecentriq Hybreza + CTx as adjuvant treatment for patients with stage III deficient mismatch repair (dMMR) or microsatellite instability-high (MSI-H) colon cancer following surgery. The application is supported by positive results from the P3 ATOMIC study.
Key results from the ATOMIC study:
The study findings were recently published in the NEJM. The FDA has assigned a PDUFA date of Oct 9, 2026, and if approved, Tecentriq could become the first immunotherapy-based adjuvant SOC for patients with stage III dMMR/MSI-H colon cancer.
Source: Roche PR Jun 11, 2026
June 12, 2026
FDA Approves Sanofi’s Tzield as First Disease-Modifying Therapy for Newly Diagnosed Stage 3 T1D
Sanofi reported that the U.S. FDA has granted accelerated approval to Tzield (teplizumab-mzwv) for children aged 8-17 years who have been recently diagnosed with Stage 3 T1D. The approval makes Tzield the first disease-modifying therapy for patients with newly diagnosed Stage 3 T1D, designed to slow the decline of endogenous insulin production by targeting the underlying autoimmune process. The decision was supported by results from the P3 PROTECT study, which showed a significant preservation of beta-cell function compared with placebo, along with safety data from more than 900 treated patients. Tzield demonstrated a safety profile consistent with previous studies, and its continued approval will be supported by the ongoing confirmatory P3 BETA-PRESERVE study, which is currently enrolling patients.
Source: GlobeNewswire PR Jun 12, 2026
June 04, 2026
Gilead and Lakefront Complete Ouro Medicines Acquisition to Strengthen Autoimmune Disease Pipeline
Gilead Sciences and Lakefront Biotherapeutics completed the acquisition of Ouro Medicines to advance T-cell engager therapies for autoimmune diseases. The deal adds gamgertamig (OM336), a clinical-stage BCMA×CD3 bispecific T-cell engager, to Gilead’s inflammation portfolio. Gamgertamig is being developed for severe antibody-mediated autoimmune diseases, including autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP), and has received both FTD and ODD from the U.S. FDA. The therapy is designed to achieve rapid and deep plasma cell and B-cell depletion through a limited subcutaneous treatment course, with the potential to provide durable disease control, and is expected to enter registrational studies as early as 2027. Gilead acquired all the outstanding equity of Ouro Medicines for $1,675M and up to $500M in contingent milestone payments. Lakefront and Gilead will equally split the upfront payment, subject to customary adjustments, and contingent milestone payments of up to $500M. While Lakefront acquired substantially all of Ouro’s team and operational assets. Lakefront will lead ongoing and future P1/2 studies of gamgertamig, whereas Gilead will oversee registrational and late-stage development and retain worldwide commercialization rights outside Keymed’s territories. Lakefront is also eligible to receive tiered royalties of 20%-23% on net sales of gamgertamig from Gilead. Lakefront acquired rights to three preclinical autoimmune programs from Ouro, with Gilead retaining an option to join each program after proof-of-concept in exchange for $75M per program and a 50/50 profit split. The deal also allows Lakefront to independently deploy at least $500M of its cash, including up to $150M for share buybacks. Following the transaction, Lakefront expects a year-end 2026 cash balance of approx. €2B ($2.32B), supporting future strategic investments and growth initiatives.
Source: Gilead Sciences PR Jun 04, 2026
June 15, 2026
Intravacc and SynphaBase Partner to Accelerate Conjugate Vaccine Development
Intravacc and SynphaBase have entered into a strategic collaboration to advance the development of next-generation conjugate vaccines. The partnership combines SynphaBase’s expertise in synthetic oligosaccharides and glycochemistry with Intravacc’s capabilities in conjugation process development and GMP-compliant vaccine manufacturing. Under this strategic partnership, vaccine developers will have access to an integrated development pathway that spans synthetic oligosaccharide supply, conjugation development, and clinical-stage vaccine production.
Source: GlobeNewswire PR Jun 15, 2026
June 02, 2026
Adaptive Innovations Raises $50M Series A to Expand AI-Powered Home Healthcare Platform
Adaptive Innovations secured $50M in Series A financing. The funding round was co-led by Felicis and Bain Capital Ventures, with additional backing from Optum Ventures and several other investors. The company plans to use the proceeds to expand its engineering and operations teams, enhance its software platform, and broaden its presence into additional U.S. state markets. With this round, Adaptive Innovations has raised a total of $60M to date.
Source: FinSMEs PR Jun 02, 2026
June 08, 2026
City Therapeutics Raises $99.5M Series B to Advance RNAi Therapeutics Platform
City Therapeutics closed a $99.5M Series B financing round, with Viking Global Investors leading the round. New investors include Sofinnova Investments, together with Casdin Capital and NYBC Ventures, joining the previous investors such as ARCH Venture Partners, Fidelity Management & Research Company, Invus, Slate Path Capital, Rock Springs Capital, Regeneron Ventures, AN Ventures, and some undisclosed venture capital firms. City Therapeutics will use the funding to drive forward its RNAi therapeutic pipeline, to build on its next-generation RNAi technology platform, and to further engage in business development activities. This financing round will allow City Therapeutics to speed up its ongoing P1 clinical trial for its CITY-FXI treatment in treating thromboembolic disorders. Furthermore, this round of funding will enable it to continue developing CITY-RBP4, which could potentially become the first-in-class RNAi therapeutic agent for Stargardt disease, as well as two more programs set for clinic entry before the end of 2026. City Therapeutics also highlighted collaborations with the biopharmaceutical companies Biogen and Bausch + Lomb, illustrating the increasing trust in its RNAi platform. With this financing round, City Therapeutics raised a total of over $230M so far.
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Babli Singh is a Senior Analyst with strong analytical skills and a deep understanding of the biopharmaceutical and healthcare industries. She specializes in monitoring emerging trends and distilling complex information into concise, insightful summaries that deliver clear value and actionable intelligence for a global professional audience.
