Associate Director

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.

Dyne Therapeutics has commenced an underwritten public offering of $300 million of its common stock. The clinical-stage company, which focuses on treatments for genetically driven neuromuscular diseases, also plans to grant underwriters a 30-day option to purchase up to an additional $45 million of shares. Morgan Stanley, Jefferies and Evercore ISI are acting as joint book-running managers for the offering. LifeSci Capital and Raymond James are also serving as joint book-running managers, whilst Jones is acting as lead manager. The offering is being made pursuant to a shelf registration statement filed with the Securities and Exchange Commission on 5 March 2024. The company is currently developing clinical programmes for myotonic dystrophy type 1 and Duchenne muscular dystrophy.

Dyne gets early 2027 decision date for Duchenne therapy, analysts expect 'smooth' review. July 21, 2026 | If approved, Dyne Therapeutics' zeleciment rostudirsen could "capture the majority" of the exon-51 Duchenne muscular dystrophy market, given its better efficacy and dosing profile versus Sarepta Therapeutics' exon-skipping therapy Exondys 51, the current standard of care, according to Oppenheimer. The FDA has accepted Dyne Therapeutics' application for its investigational Duchenne muscular dystrophy therapy, setting a target action date of Jan. 21, 2027. "We anticipate a smooth approval," Oppenheimer told investors in a note on Monday, adding that the therapy, dubbed zeleciment rostudirsen (z-rostudirsen) is poised to "capture the majority of market share," given stronger dystrophin benefits and a more convenient dosing profile than the current standard treatment. Outside of symptomatic management through corticosteroids, Sarepta Therapeutics' Exondys 51 is a standard of care regimen for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. Dyne, however, could challenge Exondys with z-rostudirsen, which in the Phase 1/2 DELIVER trial outperformed Sarepta's asset, Jefferies said in a Monday note, adding that the early data cut looks "best-in-class." Exondys is dosed weekly, while z-rostudirsen is designed to be given once every four weeks - a "convenient" profile, the firm noted. Given its convenience and efficacy advantages, "z-rostudirsen could command a pricing premium" if approved, Jefferies continued. The analysts added that they expect uptake to be "robust" across three key patient subgroups: those who have yet to undergo Exondys treatment, those who have discontinued Sarepta's therapy and those who are currently receiving it. Dyne is seeking accelerated approval for z-rostudirsen in patients with DMD who are amenable to exon 51 skipping, according to a company release on Monday. Topline data from the registrational cohort of DELIVER, released December 2025, showed a 5.46% increase at six months in concentrations of the dystrophin protein, a key disease marker. Patients also saw improvements in key functional measures, including time-to-rise velocity and the 10-meter walk/run test. Lung function, a major driver of death in DMD, was preserved at six months. Dyne's exon-skipping therapy zeleciment rostudirsen resulted in an approximately sevenfold increase in dystrophin levels at six months and elicited functional improvements that are the "best ever" for this treatment class, Stifel analysts said. December 8, 2025 The FDA does not plan on holding an advisory committee meeting for z-rostudirsen, according to Dyne. Jefferies models more than $500 million in peak sales for z-rostudirsen, calling this estimate "conservative." Oppenheimer, on the other hand, did not provide a peak forecast for the z-rostudirsen, only noting that Dyne's therapy could secure a "majority" of the $1.5 billion exon-51 DMD market. In DMD, a rare and progressive neuromuscular disorder, mutations to the dystrophin protein render it dysfunctional or completely absent. Under healthy conditions, dystrophin plays a crucial role in maintaining the structure and function of muscles. Z-rostudirsen works by restoring the expression of near-full-length dystrophin, in turn restoring the function of the protein. A key player in the DMD space is Sarepta, which aside from Exondys owns the gene therapy Elevidys. Last year, two patients died after receiving Elevidys, which ultimately forced the FDA to slap a boxed warning on the product's label and limited its use to ambulatory patients four years and up. Sarepta also owns Amondys 45 and Vyondys 53 - both exon-skipping drugs like Exondys - which in November 2025 failed to elicit significant motor function improvements in the confirmatory Phase 3 ESSENCE study. Sarepta has nevertheless pushed for full approval of these products, with the FDA setting a decision date of Feb. 28, 2027. Sarepta Therapeutics is seeking to convert the accelerated approval of its therapeutic exon-skippers for Duchenne muscular dystrophy to full despite the drugs' failure to improve motor function in a confirmatory trial. July 1, 2026