Plus, news about Pliant Therapeutics, Replicate Bioscience, Denali, Laigo Bio and Auxilius Pharma:
📉 UniQure’s shares fall as it reiterates FDA’s negative feedback on Huntington’s study:
The FDA
said
in its meeting minutes to the company that the Phase 1/2 results on its Huntington’s disease gene therapy “are currently unlikely to provide the primary evidence” to support a filing, echoing previous comments that uniQure shared from the agency in November. UniQure said it’s requesting a follow-up meeting with the FDA for early next year. UniQure made waves when it reported in September that its Huntington’s treatment succeeded in a Phase 1/2 trial, meaning it had the potential to become the first gene therapy for the disease. But those prospects were subsequently quashed by the FDA. The company’s shares
$QURE
fell 10% at market open Thursday. —
Lei Lei Wu
✍ Neurimmune, AstraZeneca solidify their bond:
The Zurich startup will get up to $780 million from the UK pharma giant for the exclusive worldwide license to NI009. The preclinical monoclonal antibody aims to treat light chain (AL) amyloidosis.
Protego Biopharma
raised $130 million earlier this week for its experimental medicine in this area, and Johnson & Johnson’s Darzalex Faspro got
full approval
in the indication last month. AstraZeneca and Neurimmune already
work together
on a potential
ATTR-CM treatment candidate
.
— Kyle LaHucik
💊 Pliant Therapeutics’ dual-acting cancer pill shows early promise
:
The biotech’s dual selective inhibitor of αvβ8 and αvβ1, known as PLN-101095, produced one complete response and three partial responses in a Phase 1 trial. The data come from 10 heavily pretreated patients with checkpoint inhibitor-refractory solid tumors, who were in the three highest dose cohorts and received PLN-101095 in combination with Merck’s Keytruda. At a median time on treatment of 15 months, as of Nov. 30, 60% of patients had stable disease or tumor reduction. Two patients discontinued due to adverse events. Pliant
said
it would start a Phase 1b expansion trial of the drug in 2026.
— Elizabeth Cairns
🤝 Replicate Bioscience makes a deal:
The San Diego-based mRNA startup
is handing over
development of its experimental rabies vaccine to a Brazilian nonprofit called Instituto Butantan. The company first reported promising
results
from a Phase 1 study of the vaccine in early 2024, but said that it would need a partner to develop it further. The shot, dubbed RBI-4000, uses a second-generation version of mRNA technology called self-replicating RNA that mimics the ability of a virus to create copies of itself. Instituto Butantan will fund and run registrational studies of the vaccine and get exclusive rights to commercialize it in Brazil and other Latin American countries. Replicate retains the rights for the vaccine in other countries. —
Ryan Cross
💰 Denali’s royalty financing deal:
The South San Francisco biotech could get up to
$275 million
from Royalty Pharma, one of the lead royalty aggregators. If Denali’s tividenofusp alfa gets approved for Hunter syndrome in the US (with an expected accelerated approval decision by April 5), it would get $200 million from Royalty. Another $75 million is dependent on European Medicines Agency approval before the end of 2029. Denali hands over a 9.25% royalty on global net sales as part of the deal.
— Kyle LaHucik
💶 Laigo Bio gets €11.5M seed:
The Dutch biotech’s protein degradation platform attracted the interest of Kurma Partners, Curie Capital and Angelini Ventures, among others. Acting CEO Matthew Baker was also
named
the permanent chief executive. He was previously CEO at NeoPhore. Laigo is based on research on surface removal targeting chimeras from the UMC Utrecht lab of professor Madelon Maurice. Laigo is working on bispecific antibodies going after hot targets such as PD-L1xVEGF.
— Kyle LaHucik
💵 Auxilius Pharma raises Series A:
The Boston biotech collected $4 million for its once-daily investigational medicine for chronic stable angina. Named AUX-001, the drug is an extended-release formulation of nicorandil. The company hopes to get a 505(b)2 market authorization as soon as 2028.
— Kyle LaHucik
