Late Stage Trial Miss Leaves Adults with Myotonic Dystrophy Still Waiting for a First Approved Treatment

HSBC adjusted ratings for biopharma stocks, citing high multiples and the need for stock-specific catalysts. Novartis was upgraded to Hold with a target price of 110 CHF, while Amgen was downgraded to Hold with a target of $425. AbbVie and Bayer were kept as Buy with raised targets. The changes reflect expectations for upcoming clinical trials and legal outcomes.

Novartis (NVS) shares fell 13% after two failed clinical trials, one for a muscle loss disease drug and another for a cholesterol-targeting drug. Amgen (AMGN) shares dropped 9%, impacting the Dow, as it develops a similar cholesterol drug. Novartis expects 5-6% annual sales growth through 2030. Both companies' stocks were affected by the trial results.

Novartis (NVS) paused eight CAR-T trials after patient deaths, prompting Bristol-Myers Squibb (BMY) to halt its own trials. BMY reported Q2 revenue of $13.0B (+6% YoY) and raised 2026 guidance to $49.0-50.0B. NVS reported Q2 sales of $14.4B (+3%) but reaffirmed guidance. BMY's growth portfolio outperformed, while NVS faces generic competition.

Novartis (NVS) announced that its late-stage study for del-desiran in myotonic dystrophy type 1 (DM1) failed to meet the primary endpoint. The stock fell on the news, marking the second pipeline setback for NVS in five days. Despite the failure, the study showed some clinical activity in secondary endpoints. NVS is evaluating the data and plans to discuss next steps with health authorities. Ionis Pharmaceuticals (IONS) shares also declined. NVS faces patent cliffs and generic competition for key

Novartis' experimental drug del-serine failed to meet primary endpoints in a Phase 3 trial for myotonic dystrophy type 1, showing no significant improvement in muscle function. The antisense oligonucleotide approach did not yield clinical benefits, highlighting challenges in rare disease drug development. This setback follows another failure in Novartis's neuromuscular portfolio, raising questions about its strategy in this area.

Novartis NVS shares fell for a third day after Deutsche Bank downgraded the stock to Hold, citing failures in two late-stage drug trials. The company's RNA therapy for myotonic dystrophy type 1 and a heart disease treatment with Ionis Pharmaceuticals IONS both failed. Analyst Emmanuel Papadakis expressed concerns about the broader Avidity platform.