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Novartis' $12B Avidity bet suffers Phase 3 blow. Novartis has suffered another pipeline setback after del-desiran, an antibody-oligonucleotide conjugate acquired with Avidity Biosciences, failed to significantly improve hand function over placebo in the Phase 3 HARBOR study in myotonic dystrophy type 1. What now: The Swiss drugmaker said it saw signs of clinical activity across secondary and exploratory endpoints and will analyse the full dataset before discussing the programme's future with regulators. Why it matters: The result is an early test of the roughly $12 billion (€10.3 billion) Avidity acquisition, completed in February. Del-desiran was not just another programme in the deal: it was its most advanced asset. Novartis bought Avidity not only for three late-stage programmes in rare muscle diseases, but for its antibody-oligonucleotide conjugate, or AOC, platform. How it works: The technology combines an antibody targeting transferrin receptor 1, or TfR1, with an RNA payload. The antibody is designed to shuttle the payload into muscle cells, where siRNA or other oligonucleotides can alter disease-related RNA. Avidity has described its clinical work as the first demonstration of targeted systemic RNA delivery into muscle. The big picture: That delivery capability is a major part of what made Avidity strategically attractive. Most established RNA medicines remain limited in the tissues they can efficiently reach, making extrahepatic delivery one of the central challenges in the field. * AOCs aim to bridge that gap by combining the targeting ability of antibodies with the gene-silencing or RNA-modifying activity of oligonucleotides. Yes, but: Avidity is not the only company exploring this option. US-based Dyne Therapeutics is developing its own TfR1-targeted FORCE conjugates, including DYNE-101 in DM1 and DYNE-251 in Duchenne muscular dystrophy. Other companies are pursuing AOC approaches in muscle diseases, cancer and other indications. What's next: For Novartis, the question is therefore larger than whether del-desiran can still find a regulatory path. The company needs to show that AOC delivery works as a platform and that targeting different RNAs can repeatedly translate into clinically meaningful effects. * Two other major programmes from the acquisition now move further into the spotlight. Del-zota, targeting Duchenne muscular dystrophy in patients amenable to exon 44 skipping, is advancing toward regulatory submission. Novartis also plans discussions with the FDA on del-brax for facioscapulohumeral muscular dystrophy following positive biomarker data. Bad timing for Novartis: The setback comes only days after pelacarsen, Novartis' closely watched Lp(a)-lowering drug, failed to reduce cardiovascular events in the Phase 3 HORIZON study. It also follows the suspension this summer of several cell therapy trials after patient deaths. Meanwhile, Novartis is looking for new growth drivers as patent expirations put pressure on established products including Entresto. * There has been some good news: remibrutinib recently delivered positive Phase 3 results in relapsing multiple sclerosis. Still, two of three closely watched recent clinical readouts - pelacarsen and del-desiran - have now disappointed. Analysts had described the del-desiran readout as particularly important for validating the Avidity acquisition. Investors vote down: The market reaction was sharp: Novartis shares fell about 9% on Sept. 8, making it one of the company's worst trading days in recent years. * Despite the news, Novartis continues to forecast annual sales growth of 5% to 6% from 2025 through 2030 and has not abandoned del-desiran. The company plans to review the complete data and consult regulators on possible next steps. | (C) european biotechnology | Georg Kääb 8 September 2026 |

Dyne Therapeutics shares dropped 31% to $16.76 in premarket trading on Tuesday, with no official company announcement explaining the decline. The biotechnology firm is developing treatments for neuromuscular diseases, including z-rostudirsen for Duchenne muscular dystrophy, which has a regulatory decision date of 21 January 2027. The company recently reported a quarterly net loss of $178.6 million, or $1.08 per share, missing consensus estimates of $0.75 per share. The sharp decline stood in contrast to broader market moves, with the S&P 500 down 0.3% and the Nasdaq slipping 0.1% during the same period. No new clinical or regulatory developments were confirmed to explain the trading movement.

FDA nod of Astrazeneca's Etcamah brings new breast cancer option. Sep. 8, 2026 A little more than four months after an FDA advisory committee voted against Astrazeneca plc's Etcamah (camizestrant) to treat first-line breast cancer, the U.S. agency granted accelerated approval of the next-generation oral selective estrogen receptor degrader (SERD) and complete ER antagonist. The drug, cleared by the EU in July, was approved alongside Guardant Health's Guardant360 companion diagnostic assay that helps identify patients with estrogen receptor-1 mutations, bringing them a potentially effective treatment sooner than previous standard-of-care protocols, which have required radiographic progression before a treatment switch. In April, the FDA's Oncologic Drugs Advisory Committee voted against the drug, 6-3, saying it did not demonstrate a clinically meaningful benefit in treating those with HR+/HER2- breast cancer. Although the Serena-6 phase III trial met its progression-free survival primary endpoint (16 months camizestrant vs. 9.2 months control), the no votes reflected concerns over the randomized treatment switch to Etcamah based solely on detection of the ESR1 mutation, an acquired resistance to an aromatase inhibitor, which is typically used together with a cyclin-dependent kinase 4/6 inhibitor as first-line treatment. Damages from med-tech cyberattacks adding up. The breadth and depth of the recent cyberattacks on medical device companies continues to expand as more companies report that they too were hit and others more fully assess the impact an attack had on them. Novocure Ltd., of Jersey, U.K., is the most recent to disclose a cyber event. In an SEC 8-K filing last week, the company reported a cybersecurity incident that occurred in mid-August, about the same time other med-tech companies were being attacked. While it's still assessing the damage caused, Novocure said it doesn't believe the incident is "reasonably likely" to have a material impact on its financial condition or operations. Meanwhile, Boston Scientific Corp. is reporting just the opposite. In an 8-K filed today, Boston Scientific disclosed that the Aug. 25 cyber intrusion it experienced is likely to materially impact both its third quarter and fiscal 2026 results. Xenotransplantation: Pig kidneys function as bridge to human donor. Xenotransplantation has finally delivered true medical benefit, with pig kidneys functioning for long enough to provide a bridge to a matching human donor organ. Two of five recipients of pig kidneys genetically engineered by Egenesis Bio Inc. have successfully transitioned to receiving human donor transplants, in the first reported cases of a xenotransplant leading on to an allotransplantation. Phase IIIs in DM1, CV outcomes blow up for Novartis. Novartis AG suffered two phase III trial failures, as delpacibart etedesiran (del-desiran) fell short in the Harbor trial testing the antibody oligonucleotide conjugate in myotonic dystrophy type 1 (DM1) and pelacarsen fizzled in the cardiovascular (CV) outcomes Horizon study trying the antisense oligonucleotide (ASO) in patients with elevated lipoprotein (a), or Lp(a). In Harbor, del-desiran did not turn up statistically significant improvement vs. placebo on the primary endpoint of video hand opening time. In Horizon, pelacarsen as compared to placebo missed the primary endpoint of reducing the risk of CV events, a composite of CV death, non-fatal myocardial infarction, non-fatal stroke, and urgent coronary revascularization requiring hospitalization. Shares of Novartis (NYSE:NVS) were trading at $138.91, down $21.08, or 13%. Another DM1 player, Dyne Therapeutics Inc., saw its shares (NASDAQ:DYN) drop $5.19, or 21%, to sell for $19.09. Dyne is developing zeleciment basivarsen (DYNE-101), which consists of an ASO conjugated to an antigen-binding fragment that binds to the transferrin receptor 1. Brainchild secures $116M for its CAR T for childhood brain cancer. Brainchild Bio Inc. decided to take on two challenges simultaneously: it's developing CAR T therapeutics for brain cancer and it's doing it initially in a pediatric indication. The Seattle Children's spinout secured $116 million in series A financing for its lead product, BCB-276, for treating diffuse intrinsic pontine glioma, a relatively rare brain cancer that affects approximately 300 children per year in the U.S. The B7-H3-targeted autologous CAR T-cell therapy is dosed directly into the lateral ventricles of the central nervous system via an indwelling reservoir-catheter device, which allows for repeat dosing of the treatment. Based on promising phase I results in 21 patients, Brainchild recently launched a phase II study testing the cell therapy following treatment with ionizing radiation. Moonwalk snags $70M to advance its obesity drug into phase I development. Moonwalk Biosciences Inc. used its human genetics, epigenomics and multi-omics discovery platform to discover multiple gene targets involved in adipose tissue biology. With preclinical data in hand, the company secured a $70 million series B financing to develop its lead candidate MW-101, an siRNA targeting the expression of an undisclosed gene with the potential to decrease lipids stored in adipose tissue. The company expects to start first-in-human clinical studies in late 2027 following completion of ongoing IND-enabling studies. Ciliatech nabs FDA IDE for implant to treat glaucoma. Ciliatech SAS received U.S. FDA investigational device exemption (IDE) for a feasibility study of Intercil Uveal Spacer, its implant to treat glaucoma. The trial which will evaluate the safety and performance of the device, will focus on patients who fall into the "middle segment" treatment gap, between the conventional minimally invasive glaucoma surgery and more invasive filtering procedures. "We are addressing a population that has no solution," Olivier Benoit, CEO of Ciliatech, told BioWorld. Sky Labs shares extend rally after $15M Kosdaq IPO. Shares of Sky Labs Inc. (KOSDAQ:386380) have nearly tripled since its Sept. 4 debut, as the company seeks to turn data generated by its wearable rings into a clinical data platform for the industry and medical institutions. Despite weak IPO demand, Sky Labs' shares on the tech-heavy Kosdaq closed at ₩29,750 (US$22.10) Sept. 8, down 2.62% from the previous day but 197.5% higher than the offering price. The Seongnam-based company had pegged the offering of about 2 million shares to ₩10,000 per share, well below the target range of ₩13,000 to ₩16,000 per share. Korea Investment & Securities was the lead underwriter. Asia Bio Partnering: APAC biotechs rethink partnering playbook. Asia-Pacific biotechs that want to compete globally need to make decisions about corporate structure, intellectual property, financing and clinical development from the outset, panelists said during the recent Asia Bio Partnering Forum in Singapore. The region's partnering strategies are also moving beyond the familiar model of licensing Asian-developed assets to Western drugmakers. Companies and investors are experimenting with co-development agreements, territorial licenses and "reverse newcos" that bring assets originating outside China into locally established companies to capitalize on China's drug development infrastructure. A&As: new ceos for arletta, samsara, sense neuro, typewriter. New hires and promotions in the biopharma and med-tech industries. Financings: Typewriter's $56M series A; Superluminal adds $60M in series B. Biopharma and med-tech companies raising money in public or private financings. Holiday notice. BioWorld's offices were closed in observance of Labor Day in the U.S. No issue was published Monday, Sept. 7. To read more on related topics, click on one of the words below.