Key Outcomes:
- R-5780 (Rise Therapeutics) – P1, immuno-oncology: Dose-escalation complete; IDMC approved advancing to dose-expansion at the highest selected fixed dose in up to 33 cancer patients with PD-1-refractory tumors
- IMVT-1402 (Immunovant/Roivant) – P2 PoC, CLE: Missed primary endpoint (CLASI-A at wk 12 vs pbo, n=56); development in CLE discontinued. No impact on ongoing programs in Graves’ disease, MG, CIDP, Sjögren’s, and difficult-to-treat RA
- Cadonilimab (Akeso) – P3 COMPASSION-37, 1L gastric cancer: First patient enrolled in global head-to-head study of PD-1/CTLA-4 bispecific + CTx vs nivolumab + CTx in HER2-negative G/GEJ adenocarcinoma; follows positive China P3 COMPASSION-15 OS data
- Annamycin (Moleculin) – P2/3 MIRACLE, r/r AML: Part A enrollment complete (n=89); Part A data readout on track for Q1 2027. Blinded data continue to show no cardiotoxicity, a key differentiator vs conventional anthracycline
- FREHEMGO/denecimig (Novo Nordisk) – Haemophilia A: CHMP adopted positive opinion for EU approval in adults and children with or without FVIII inhibitors; mean ABR below 1 across P3 FRONTIER populations; EU launches targeted Q4 2026; US BLA already submitted
- Ocrevus (Roche) – paediatric relapsing MS: CHMP recommended EU approval for ages ≥10 based on P3 OPERETTA 2, showing non-inferior relapse risk and superior MRI outcomes vs fingolimod; consistent adult safety profile; US FDA already approved in May 2026
- ARIKAYCE (Insmed) – MAC lung disease: FDA granted Priority Review to sNDA seeking earlier-line use and conversion of 2018 accelerated approval to traditional approval (PDUFA Jan 28, 2027), based on P3b ENCORE in 425 treatment-naïve patients
- RZ-001 (Rznomics) – glioma: EMA COMP adopted a positive opinion for Orphan Medicinal Product Designation based on interim P1/2a data in recurrent GBM; formal EC grant would confer 10-year market exclusivity and regulatory benefits
- Novo Nordisk/Anthropic collaboration: Partnership to apply Anthropic’s AI models and Claude Science to drug discovery, biological reasoning, and R&D workflows, with Novo also leveraging frontier models for AI-driven software engineering
- Opzelura/Incyte – Specialised Therapeutics (Australia): ST expanded its Incyte partnership to commercialize ruxolitinib cream in Australia for vitiligo and AD; ST will handle regulatory submissions, medical affairs, and distribution
- Nanexa/Novo Nordisk – PharmaShell license: Global exclusive deal covering up to five peptide programs (obesity, T2D, cardiometabolic) targeting QM/Q4M injectable formulations; Nanexa eligible for up to €1.165B including €615M in upfront and development/regulatory milestones plus royalties
- Precision Neuroscience – Series D: Raised $250M (co-led by Pershing Square), bringing total to $430M; funds will support clinical expansion, FDA review of Layer 7 Cortical Interface, and commercialization build-out; 100+ procedures completed across 18 institutions
- LNK001 (Link Cell Therapies) – P1, clear cell RCC: First clinical dose administered at MD Anderson for this AND-gated CAIX/ENPP3 CAR-T; Series A upsized to $90M to support pipeline including a planned
September 22, 2026
Rise Therapeutics Completes R-5780 P1 Dose Escalation and Advances to Dose Expansion
Rise Therapeutics has completed dose-escalation in its P1 study of R-5780, its lead oral immuno-oncology candidate, and identified a recommended dose for further development. After reviewing safety data, an IDMC approved advancing to dose-expansion, where additional patients will receive the highest selected fixed dose to further assess safety, tolerability, and R-5780’s ability to enhance responses to PD-1 checkpoint inhibitors. This multi-dose P1 study plans to enrol up to 33 cancer patients, particularly those whose tumors are refractory to PD-1 inhibitors, evaluating R-5780’s safety, drug exposure, and clinical activity. R-5780 is an orally delivered, precision-directed synthetic-biology therapy designed to boost anti-tumor T-cell responses and re-sensitize tumors to checkpoint inhibition aiming to expand the population who can benefit from checkpoint therapies, including those who’ve stopped responding or never responded at all.
Source: PR Newswire PR Sep 22, 2026
September 23, 2026
IMVT-1402 Misses Primary Endpoint in Cutaneous Lupus Erythematosus Proof-of-Concept Study
Roivant released topline results from Immunovant’sP2 PoC study of IMVT-1402 (imeroprubart) in adults with CLE. The study did not achieve its primary endpoint, with IMVT-1402 failing to demonstrate a statistically significant improvement in percentage change from baseline in CLASI-A (Cutaneous Lupus Area and Severity Index-Activity) score at wk 12 vs pbo. The randomized study enrolled 56 patients with subacute or chronic CLE, with or without SLE, and evaluated IMVT-1402 600 mg QW SC vs pbo, followed by continued IMVT-1402 treatment at different dose levels. Based on the topline findings, Immunovant will discontinue further development of IMVT-1402 in CLE. Roivant stated that the result does not affect the ongoing development of IMVT-1402 in other indications, including potentially registrational programs in Graves’ disease, myasthenia gravis, chronic inflammatory demyelinating polyneuropathy and Sjögren’s disease. The company also continues to evaluate IMVT-1402 in difficult-to-treat rheumatoid arthritis.
Source: GlobeNewswire PR Sep 23, 2026
September 27, 2026
Akeso Enrolls First Patient in Global P3 Study of Cadonilimab vs. Nivolumab in First-Line Gastric Cancer
Akeso reported enrollment of the first patient inCOMPASSION-37 (AK104-311), a global, multicenter P3 head-to-head study evaluating cadonilimab (PD-1/CTLA-4 bispecific antibody) + CTx vs CTx with or without nivolumab as 1L treatment for patients with HER2-negative, previously untreated, unresectable or metastatic gastric or gastroesophageal junction (G/GEJ) adenocarcinoma. The study will assess whether dual PD-1/CTLA-4 blockade can improve outcomes across geographically and ethnically diverse populations compared with the current nivolumab-based treatment approach. The study follows positive P3 COMPASSION-15 results in China, where cadonilimab + CTx demonstrated an OS benefit across PD-L1 expression subgroups, including patients with low or negative PD-L1 expression. The global study is co-led by investigators including Yelena Janjigian (MSK), Markus Möhler, Jiafu Ji and Lin Shen; several were investigators in CheckMate 649, which established nivolumab plus chemotherapy as a first-line treatment option in advanced gastric cancer.
Source: PR Newswire PR Sep 27, 2026
September 29, 2026
Moleculin Completes Part A Enrollment in P2/3 MIRACLE Study of Annamycin in R/R AML
Moleculin Biotech completed and closed enrollment in Part A of the pivotal P2/3 MIRACLE study, evaluating Annamycin + cytarabine (AnnAraC) in adults with r/r Acute Myeloid Leukemia. A total of 89 patients were enrolled, with one additional patient in screening, and the company remains on track for a comprehensive Part A data readout in Q1 2027. The dataset is expected to provide efficacy and safety insights across patients previously treated with intensive 7+3 CTx or venetoclax-based regimens, with the final population expected to be approximately 50/50 between these groups. Part A is a randomized, double-blind comparison of two Annamycin doses + cytarabine versus cytarabine + pbo, with outcomes assessed after one treatment cycle. Moleculin also reported that blinded data continue to show no cardiotoxicity, a key differentiating feature of Annamycin vs conventional anthracyclines.
September 17, 2026
FREHEMGO (denecimig) Receives Positive CHMP Opinion for Haemophilia A
Novo Nordisk reported that the EMA’s CHMP has adopted a positive opinion recommending EU marketing authorisation for FREHEMGO (denecimig), a next-generation FVIIIa-mimetic bispecific antibody that bridges FIXa and FX for routine prophylaxis in haemophilia A patients (adults and children, with or without FVIII inhibitors). Backed by the FRONTIER study programme including FRONTIER 2 (significant reduction in annualised bleeding rates vs prior prophylaxis/on-demand treatment), FRONTIER 3 (consistent efficacy in children under 12), and FRONTIER 5 (no new safety signals switching from emicizumab, with device preference reported) the therapy achieved mean annualised bleeding rates below 1 across P3 populations, with many patients experiencing zero treated bleeds. FREHEMGO offers flexible SC dosing (QW, Q2W, or Q4W) via a pre-filled pen, with Novo targeting initial EU launches in Q4 2026, broader EU rollout from early 2027, and a U.S. BLA already submitted in Sep 2025.
Source: GlobeNewswire PR Sep 17, 2026
September 18, 2026
Ocrevus Receives Positive CHMP Opinion for Paediatric Relapsing MS
Roche reported that the EMA’s CHMP recommended EU approval of Ocrevus (ocrelizumab) IV infusion for children and adolescents aged 10 years and older with relapsing forms of multiple sclerosis. The recommendation is based on the P3 OPERETTA 2 study.
OPERETTA 2 study results:
Ocrevus is a CD20-directed B-cell-depleting monoclonal antibody, administered by IV infusion every six months; the U.S. FDA approved paediatric RRMS use in May 2026, and the final EC decision is expected following the CHMP opinion.
Source: GlobeNewswire PR Sep 18, 2026
September 21, 2026
FDA Grants Priority Review to Insmed’s ARIKAYCE sNDA for Earlier-Line MAC Lung Disease Treatment
The U.S. FDA accepted Insmed’s sNDA for ARIKAYCE (amikacin liposome inhalation suspension) for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial regimen and granted Priority Review, setting a PDUFA target action date of Jan 28, 2027. The application seeks to expand ARIKAYCE use to patients earlier in the disease course, including newly diagnosed and recurrent MAC lung disease, while also supporting conversion of its existing 2018 accelerated approval for refractory MAC lung disease to traditional approval. The sNDA is supported by the P3b ENCORE study, which enrolled 425 treatment-naïve patients with newly diagnosed MAC lung disease and met its primary endpoint. The study was also designed to fulfill the FDA post-marketing requirement associated with ARIKAYCE’s accelerated approval under the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD). Insmed also plans to discuss the ENCORE data with Japan’s PMDA in Q4 2026 to support a potential label expansion in Japan.
Source: PR Newswire PR Sep 21, 2026
September 30, 2026
Rznomics Receives Positive EMA Opinion for Orphan Medicinal Product Designation of RZ-001 in Glioma
Rznomics reported that the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) adopted a positive opinion on Orphan Medicinal Product Designation for RZ-001 (taspitimagene advec) in glioma. RZ-001 is an adenovirus-based gene therapy built on the company’s RNA trans-splicing ribozyme platform. It targets hTERT mRNA in cancer cells and replaces it with an RNA encoding a herpes simplex virus suicide gene (HSV-TK). Once activated by the oral prodrug valganciclovir, the suicide gene selectively kills the cancer cells. The opinion rests on interim P1/2a data in recurrent glioblastoma, and the COMP concluded that RZ-001 has enough potential to offer a “significant benefit” over existing European therapies. RZ-001 has been given to 10 patients in clinical trials and 3 under the U.S. FDA’s Expanded Access Program. Once the EC formally grants the designation, Rznomics will be eligible for up to 10 years of market exclusivity, EMA protocol assistance, and reduced regulatory fees. The company also points to its May 2026 FDA RMAT designation for hepatocellular carcinoma as further evidence of the platform’s versatility.
Source: PR Newswire PR Sep 30, 2026
September 16, 2026
Novo Nordisk and Anthropic Collaborate to Accelerate AI-Driven Drug Discovery
Novo Nordisk and Anthropic entered into a collaboration to accelerate drug discovery and development using advanced AI models, with an initial focus on applying Anthropic’s models and Claude Science to scientific reasoning and specific R&D workflows. The companies will jointly identify key drug-discovery challenges, develop targeted AI solutions for biological reasoning, and explore opportunities to shorten the path from research to new medicines. Novo will also deploy Anthropic’s frontier models to strengthen AI-driven software engineering, supporting the broader scaling of AI across its organisation. The collaboration builds on Novo’s ambition to become the world’s most AI-driven healthcare company and will initially test Claude Science on selected R&D problems where the combined capabilities of both companies could have the greatest impact.
Source: Novo Nordisk PR Sep 16, 2026
September 20, 2026
Specialised Therapeutics Expands Incyte Partnership to Commercialise Opzelura in Australia
Specialised Therapeutics (ST) expanded its existing partnership with Incyte Biosciences International to commercialise, register and distribute ruxolitinib cream (Opzelura) in Australia, extending their collaboration into inflammatory dermatology. Opzelura is a 1.5% topical JAK1/JAK2 inhibitor that is approved in the U.S. for non-segmental vitiligo in adults and adolescents ≥12 years and for mild-to-moderate AD in non-immunocompromised adults and children ≥2 years whose disease is inadequately controlled with, or for whom, topical prescription therapies are not advisable. Incyte will retain responsibility for development and manufacturing, while ST will oversee Australian regulatory submissions, medical affairs, marketing and distribution. ST’s immediate priority is to progress the Australian regulatory and reimbursement pathways, with submissions to healthcare authorities expected in due course, aiming to support timely access for eligible patients.
Source: PR Newswire PR Sep 20, 2026
September 25, 2026
Nanexa and Novo Nordisk Sign €1.165B Global License Deal for Long-Acting Injectables
Nanexa and Novo Nordisk entered into a global exclusive license and collaboration agreement covering Nanexa’s PharmaShell drug-delivery technology for up to five peptide-based programs targeting obesity, T2D and other cardiometabolic diseases. The program aim to develop QM or Q4M injectable formulations, potentially reducing dosing frequency. Novo Nordisk will lead global development and commercialization, while Nanexa is eligible for up to €1.165B ($1.32B), including €615M ($700M) in upfront and development/regulatory milestones, additional sales milestones and low-single-digit royalties on global net sales. PharmaShell uses an ultra-thin coating around individual drug particles to enable controlled, sustained drug release.
Source: PR Newswire PR Sep 25, 2026
September 24, 2026
Precision Neuroscience Closes Oversubscribed $250M Series D to Advance Brain-Computer Interface Development
Precision Neuroscience closed a oversubscribed $250M series D financing, co-led by Pershing Square Inc., the Ackman Oxman Institute and an undisclosed leading life sciences investment fund, bringing the company’s total capital raised since its 2021 founding to $430M. Additional investors included Duquesne Family Office, B Capital, ARK Invest, Invus, Mubadala Capital, Mirae Asset Capital, Korea Investment Partners, Hitachi Ventures and JSL Health Capital. Precision will use the proceeds to expand its clinical program, advance its Layer 7 Cortical Interface through further FDA review and build infrastructure for commercialization. The company has completed 100+ patient procedures across 18 major medical institutions, received FDA clearance for Layer 7, and established a commercial partnership with Medtronic to integrate its technology with an established neurosurgical platform. The company further highlighted peer-reviewed validation, including research published in Nature Biomedical Engineering and clinical work with Johns Hopkins, as well as the appointment of Dr. John Donoghue, founder of BrainGate, as a Scientific Advisor.
Source: GlobeNewswire PR Sep 24, 2026
September 28, 2026
Link Cell Therapies Initiates Clinical Dosing of Logic-Gated CAR-T and Upsizes Series A to $90M
Link Cell Therapies initiated clinical dosing of LNK001, an AND-gated CAR-T therapy targeting CAIX and ENPP3, in a P1 study for patients with advanced/metastatic clear cell renal cell carcinoma at MD Anderson Cancer Center. The dual-antigen approach is designed to improve tumor specificity and reduce the on-target, off-tumor toxicity associated with single-antigen CAIX targeting. The company also expanded its Series A financing from $60M to $90M, with the additional capital supporting clinical/preclinical development of its logic-gated CAR-T pipeline, including a planned first-in-human colorectal cancer program in 2027. Link appointed Jie D’Elia, Ph.D., as CEO and Friedrich Graf Finckenstein, M.D., as CMO, while co-founder Brian Slingerland became Executive Chair. LNK001 is manufactured at CTMC, a joint venture between Resilience and MD Anderson.
Source:PR Newswire PR Sep 28, 2026
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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.
