| | | |   | Hello, everyone. Damian here with some highly anticipated data from Alnylam Pharmaceuticals, news of a dramatic day at the FDA, and positive signs for Gilead Sciences' future in oncology. | | | Alnylam’s Phase 3 success comes with questions for Wall Street Detailed data from Alnylam Pharmaceuticals’ successful heart disease study showed a significant improvement over placebo, but the magnitude of its benefit will likely stir debate over the commercial future of the company’s drug.
In the study, presented today, Alnylam’s patisiran helped patients with ATTR-CM, a progressive heart disease, walk about 14.7 meters more over the course of six minutes compared to those who got placebo. The result was statistically significant, and it’s expected to be enough to win FDA approval for patisiran in ATTR-CM.
But it may come as a disappointment to Wall Street, where analysts expected a roughly 30-meter difference between patisiran and placebo on the six-minute walk test, a number that would match the observed benefit of the only approved treatment for ATTR-CM, a pill from Pfizer.
Read more. | Amylyx’s ALS drug succeeds in FDA do-over A panel of FDA advisers voted in favor of approving Amylyx Pharmaceuticals’ investigational treatment for ALS yesterday, reversing an earlier decision after the company promised to pull the drug if a confirmatory trial turns out negative.
As STAT’s Adam Feuerstein reports, the agency’s independent neurology advisers voted 7-2 that the drug, AMX0035, had enough evidence to merit an accelerated approval. Justin Klee, Amylyx’s co-CEO, said at the meeting that if AMX0035 fails in a Phase 3 trial slated to read out in 2024, the company would voluntarily stop marketing it.
The hearing was a sharp contrast to the last discussion of AMX0035, in which the FDA seemed skeptical of the company’s supportive evidence, and the same panel voted 6-4 against recommending approval.
Read more. | Science is making it possible to remove the word ‘serial’ from crime Fred Harran, Public Safety Director for the Bensalem Township police department, is bringing a new sense of safety to the residents of his community thanks to advancements in DNA analysis technology. Read more. | Gilead’s $21 billion gamble shows promise A Gilead Sciences’ drug prolonged the survival of patients with the most common form of breast cancer by just under 30%, according to data disclosed yesterday, a result that could present a new treatment option and help justify the company’s sizable investment in the medicine.
As STAT’s Adam Feuerstein reports, the drug, Trodelvy, showed a median overall survival of 14.4 months compared to 11.2 months for comparator chemotherapy. The survival benefit of 3.2 months was statistically significant and translated into a 21% reduction in the risk of death.
Gilead acquired Trodelvy in its $21 billion acquisition of Immunomedics in 2020, a price the drug’s commercial performance is yet to justify. The latest data, which Gilead has submitted to the FDA, could expand its use and improve Trodelvy’s sales.
Read more. | Relay’s drug for a rare cancer comes through in early study Relay Therapeutics’ experimental drug for cholangiocarcinoma, a rare cancer of the bile ducts, led to tumor shrinkage of a third or more in 15 of 17 patients with advanced disease, the company said yesterday.
As STAT’s Matthew Herper reports, the highest tested dose of Relay’s drug led to a roughly 88% response rate in a small trial. Other treatments in the same class showed only 30% to 40% responses.
The drug, RLY-4008, targets mutations to the protein FGFR2. Previous FGFR2 inhibitors had toxicities that limited dosing, including leading to high levels of phosphorus. RLY-4008 might prove to be more precise in targeting FGFR2, translating into a safer, more effective medicine.
Read more. | More reads - Judge invalidates parts of the ACA that mandate health coverage of many preventive services and drugs. STAT
- Donations by pharmaceutical manufacturers to independent patient assistance charities. Health Affairs
- Launch prices: tackling the next drug pricing challenge. STAT
- EU secures more doses of Bavarian monkeypox vaccine. Reuters
| Thanks for reading! Until tomorrow,  | | | |
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