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Denali details accelerated approval plans; Jazz's $850M private offering

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Plus, more news about Circle Pharma, Emergent, Cytokinetics, Nxera Pharma, Neurocrine Biosciences, Simcere Zaiming, TargetRx and Calliditas:

Denali Therapeutics to seek accelerated approval in 2025: The decision to seek an expedited OK for its Hunter syndrome program, called DNL310, follows a years-long debate over whether heparan sulfate can serve as an effective surrogate biomarker for certain neurodegenerative diseases. Denali said that the FDA agreed that heparan sulfate levels in the cerebrospinal fluid are “reasonably likely to predict” a clinical benefit. Denali also plans to submit data on the neurofilament biomarker that Biogen used to get an ALS drug approved last year for certain genetic forms of the disease. Regenxbio, with whom Denali is competing in Hunter syndrome, is expected to launch a rolling submission for accelerated approval by the end of this month. — Max Gelman

Jazz to launch $850M private offering: The offering, coming in the form of “exchangeable senior notes due in 2030,” will help pay down about $350 million of Jazz’s outstanding loans. — Max Gelman

Circle Pharma closes $90M Series C: Circle said in July it was seeking to bring in $117 million when the company raised $54 million as part of its Series C round. The money will be used to fund Circle’s cyclin A/B RxL inhibitor, CID-078, in various tumor types. — Max Gelman

Emergent BioSolutions gets $250M loan: The money from Oak Hill Advisors is being used to repay the remaining outstanding loan that Emergent took out in 2018. Emergent’s debt is expected to be reduced by about $200 million by the end of this year, CEO Joe Papa said in a statement. Its stock $EBS was down about 9% on Tuesday morning. — Max Gelman

Cytokinetics touts more Phase 3 data for aficamten: In the open-label FOREST-HCM trial, a subgroup of 59 hypertrophic cardiomyopathy patients taking aficamten successfully scaled down their standard of care medication, which is defined as a minimum 50% dose reduction in at least one drug from baseline, according to results presented Sunday at the European Society of Cardiology’s annual meeting. Cytokinetics is currently preparing regulatory filings for the cardiac myosin inhibitor. — Ayisha Sharma

Nxera Pharma gets $35M payment from Neurocrine Biosciences: Completing the Phase 2 trial for its candidate in adults with schizophrenia triggered the payment for Nxera, previously known as Sosei Heptares. The trial met the primary endpoint, and Nxera plans to move the muscarinic M4 selective agonist into Phase 3 trials next year. — Katherine Lewin

Simcere Zaiming pays $20M to TargetRx for cancer asset: The oncology-focused biopharma is giving $20 million to Shenzhen TargetRx for commercial rights to a Phase 1 antitumor small molecule candidate in China. Simcere will gain marketing rights to TGRX-326 as well as other benefits and will compensate TargetRx for helping to promote the drug. — Anna Brown

Calliditas Therapeutics will delist following purchase by Asahi Kasei: Asahi now owns more than 90% of the company’s outstanding shares per its offer to purchase all shares of the Stockholm-based company. The delisting of the American shares is expected to occur around Sept. 23. — Katherine Lewin


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