| | | |   | Happy Halloween! Keep your kids healthy, and eat all their candy. Today: We have two down-votes from the FDA, we preview Moderna's earnings this week, and see a new, HSV-driven gene therapy startup launched. | | | Replay launches retina-focused gene therapy startup Genome-writing biotech Replay is launching a new gene therapy company today targeting inherited retinal diseases. It’s using a different sort of delivery vector, engineering herpes simplex virus to ferry a therapeutic payload. Eudora will be targeting diseases like retinitis pigmentosa, Stargardt disease, and Usher syndrome 1B — all diseases of the back of the eye. Replay has a somewhat unique corporate structure, which shares its core technology with the startups it spins out. Eudora is Replay’s fourth gene therapy company, all of which apply its proprietary synHSV technology. Replay says that its current HSV-based vectors can deliver up to eight times the payload of the adeno-associated viruses typically used in gene therapy — and a new version under development may be able to develop a payload that’s 30 times the payload of AAVs. This means that larger genes can, in theory, be inserted into a recipient’s DNA — or even several different genes. | Guidance for an underperforming Moderna Moderna will report its third-quarter earnings on Thursday this week. Despite underperforming so far this year — the company’s stock has plunged more than 50%, compared to the industry’s 31% decline in 2022 — analysts are mixed on their view of the mRNA giant. The demand for its Covid vaccines is waning, but it plans to increase the price of the inoculations as they shift from a contracted market to a commercial market, which according to SVB Securities analysts “substantially improves the company’s ability to meet 2023 revenue guidance.” Moderna is also expanding into non-Covid indications, with a particular focus on other highly infectious diseases. Although it is planning on entering the flu and RSV markets, Moderna will still face a lot of competition — which means analysts, such as those at Cowen, are also downgrading their expectations of Moderna’s performance in 2023. | From months to minutes: the need for speed in real-world evidence generation The process of identifying, acquiring, and evaluating the right real-world data, not to mention converting that data into evidence, is rife with failure and delay. It's time for eClinical innovators to step up with a single, self-service trusted research environment that unites continuously refreshed RWD with a flexible coding environment. Read what that solution needs to deliver, from cohort definition to computational power. | Ad comm vetoes pediatric neuroblastoma drug An advisory committee to the FDA late last week unanimously nixed a brain cancer treatment from Y-mAbs meant for children. The expert panel said the drug, called omburtamab, wasn’t shown to convincingly improve overall survival in a rare form of neuroblastoma in children. It voted 16-0 against the antibody therapy; the company’s shares fell 35% on the news in after-hours trading on Friday. The drug was granted priority review back in May, based on two Phase 2 studies whose data have not yet been published. Y-mAbs plans on releasing the data later this year.
This isn’t the first time Y-mAbs has had trouble with omburtamab. Two years ago, the FDA gave the company a refusal to file letter, citing concerns about manufacturing and control. That said, Y-mAbs released interim results from 32 patients who participated in a separate study of omburtamab — reporting an overall survival rate of 73.5%. An FDA decision on the drug is due by Nov. 30. | FDA rejects Gilead's Hep D drug Gilead last week received an FDA rejection for its hepatitis D drug, bulevirtide, BioSpace writes. The company acquired the investigative antiviral two years ago when it bought Germany’s Myr GmbH. The FDA said it was concerned by bulevirtide’s manufacture and delivery. Bulevirtide has already been granted conditional approval in Europe to treat HDV, the most severe form of viral hepatitis. It’s marketed under the brand name Helcludex. During its investor call, Gilead’s CMO said the company still needs to “fully digest” the rejection — and then resubmit a biologics license application as soon as possible. | More reads - As Sanofi’s Dupixent rolls on, influenza sales are icing on the cake, FiercePharma
- Biotechnology is creating ethical worries — and we’ve been here before, Ars Technica
- Drug that acts on brain’s dorsal raphe nucleus could rapidly treat depression, FierceBiotech
| Thanks for reading! Until tomorrow,  | | | |
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