Eli Lilly plots NDA as second JAK study backs baricitinib for alopecia areata; Alnylam heads to FDA after full vutrisiran data pan out
Eli Lilly has posted positive data from its second Phase III study testing its JAK inhibitor baricitinib in spurring hair regrowth among patients suffering from severe alopecia areata.
Investigators reported a dose-dependent response to the drug, with 35% of the subjects in the 4 mg arm achieving 80% or more scalp hair coverage against 22% in the 2 mg arm. That’s in line with the first Phase III readout.
That leaves Lilly following up with a new drug application later in the year, looking to capitalize on their breakthrough drug designation.
Yet to be seen, though, is what regulators will think regarding the safety issues. The FDA barred the use of the 4 mg dose for rheumatoid arthritis, worried about the longterm safety profile of the drug, which is shared by the entire class. Lilly had once insisted on using the 4 mg dose for RA, to no avail.
Lotus Mallbris, vice president of immunology development at Lilly, noted: “We look forward to discussing with global regulators data from the BRAVE-AA clinical trial program for this important potential treatment, which could be the first approved for people living with alopecia areata.” — John Carroll
Alnylam heads to FDA after full vutrisiran data pans out
Alnylam is plowing ahead with its vutrisiran program as it reported full data from its Phase III study at AAN on Monday.
The data confirm the previously reported topline results from January, showing vutrisiran topped a historical placebo control in reducing the symptoms of polyneuropathy tied to a protein folding disorder — transthyretin-mediated amyloidosis — from baseline after nine months. As such, the company has submitted its NDA to treat the hereditary version of the disease, where the primary and secondary endpoints were also met.
Alnylam had measured the experimental drug against Onpattro, the company’s approved RNAi therapy for ATTR. The treatments resulted in an average of a 17-point difference in a clinical testing scale, with the change favoring vutrisiran.
Should all go well with regulators, vutrisiran stands ready to launch sometime in 2022. It would mark the fourth RNAi therapy Alnylam has pushed across the finish line. — Max Gelman
VC Forbion closes at hard cap as it raises $428 million for growth fund
In its final stage of fundraising, Forbion has raised $428 million for its growth fund to focus on investing in late-stage European life science companies, the company announced Tuesday, and have announced its first investments.
Forbion has invested in Ireland-based SynOx Therapeutics, NewAmsterdam Pharma, of the Netherlands and the UK’s Gyroscope Therapeutics, which filed for an IPO this week. Gyroscope announced in March that it was hustling through Phase II clinical trials for candidates GT005, a gene therapy for patients with geographic atrophy.
These are the first three of between 10-12 investments in what the company calls “the most promising European late-stage sciences companies.” The venture capital firm is focused on investing in companies that develop therapies for “areas of high medical need.”
The fund — dubbed Forbion Growth Opportunities I — launched in July 2020 and closed at the hard cap. Investors include Pantheon, Eli Lilly, Horizon Therapeutics, the Belgian Growth Fund and New Waves Investments. — Josh Sullivan