AUF1 Gene Therapy Limited Muscle Loss After Injury in Mice
Mice kept near normal strength after a severe muscle injury when given extra AUF1, by virus or by mRNA. Two authors cofounded the company developing it, and no human has received it.
Pillar
Drug pipelines, platforms and company data, read against the primary source.
18 articles
Mice kept near normal strength after a severe muscle injury when given extra AUF1, by virus or by mRNA. Two authors cofounded the company developing it, and no human has received it.
A crossover trial in a rare disease, funded by the drugmaker: each patient treated one attack with the pill and one with placebo. The result is large and the analysis smaller than the enrollment.
A new mechanism, a positive primary endpoint and a modest effect size. Everything here comes from a company release and a congress talk, not a peer reviewed paper.
One drug in early clinical testing, a $245 million accumulated deficit, and a platform the company itself calls unproven. The prospectus is more candid than the headlines.
Every date below comes from the company itself. CagriSema has no published goal date, only a quarter. One file arrives after an advisory committee voted 9 to 3 against.
Four launches in three months. One produced a preprint about an enzyme system whose function is still unknown. The rest are access rules, pilots and promises.
Known risk factors explain at most about half of the 20% drop in heart events, and the frailest patients still benefit. Widening eligibility to a BMI of 25 is a different question.
Women lost 12.8% to 14.4% of their weight on the top dose without diabetes. The diabetes trial tells a shakier story, built partly on a subgroup of 10 women.
Efsitora matched daily insulin on HbA1c in four trials and trades 365 injections for 52. In type 1 diabetes, severe lows were three times as common, which is why the label stops at type 2.
Pelacarsen lowered Lp(a) in 8,323 HORIZON patients, but heart attacks and strokes did not fall. Why that gap between biomarker and outcome matters.
A daily tablet now cuts LDL by more than half, while the injectable version of the same drug class was linked to 20% fewer deaths in people with no prior heart attack.
Retatrutide's 20.8% assumes nobody quit, and Viking's 90% comes from a group of 11. The numbers behind the two biggest obesity drug headlines of September.
Against placebo, the endothelin blocker lowered systolic pressure by about 4 mmHg on top of three drugs. New post hoc data on obesity and kidney risk.
Novo won at the low end of the dose range, 12.4 versus 9.1 percent. Lilly won at the top, 23.3 versus 14.8, and lost up to one patient in four to side effects.
Trevogrumab and apitegromab saved kilograms of lean tissue in phase 2 trials, but no study has yet shown that it makes anyone stronger.
Orforglipron cleared a heart safety bar against insulin glargine, 4.2 versus 5.0 percent, and posted a mortality gap the trial was never built to prove.
ACHIEVE-3, 1,698 adults, 52 weeks: in the primary analysis HbA1c fell 1.91 points against 1.47. A real head to head, run by one of the duelists.
Sironax's FORGE-LM trial is half enrolled. The endpoint is body composition, which is the right question. Enrollment, though, is not a result.
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