Denali's Accelerated Approval Plans; Jazz's $850M Offering

Denali Therapeutics plans to seek accelerated approval for its Hunter syndrome treatment, DNL310, in 2025. This decision follows prolonged discussions about using heparan sulfate as a surrogate biomarker for neurodegenerative diseases. The FDA has agreed that heparan sulfate levels in cerebrospinal fluid could reasonably predict clinical benefits. Additionally, Denali intends to submit data on the neurofilament biomarker, similar to what Biogen used to get an ALS drug approved for certain genetic forms. Denali is competing with Regenxbio, which is expected to start a rolling submission for accelerated approval by the end of this month.

Jazz Pharmaceuticals announces $850M private offering:
Jazz Pharmaceuticals is launching an $850 million private offering of "exchangeable senior notes due in 2030." The funds will be used to repay approximately $350 million of Jazz's outstanding loans.

Circle Pharma secures $90M in Series C funding:
Circle Pharma has successfully closed a $90 million Series C funding round. The money will be directed toward advancing its cyclin A/B RxL inhibitor, CID-078, for various tumor types. Circle had initially aimed to raise $117 million earlier in July and had already secured $54 million as part of this round.

Emergent BioSolutions receives $250M loan:
Emergent BioSolutions has obtained a $250 million loan from Oak Hill Advisors. This loan will help repay the remaining debt from a loan taken out in 2018. Emergent expects to reduce its debt by about $200 million by the end of the year. However, its stock saw a 9% decline on Tuesday morning.

Cytokinetics presents Phase 3 data for aficamten:
Cytokinetics has released more Phase 3 data for its cardiac myosin inhibitor, aficamten. In the open-label FOREST-HCM trial, 59 patients with hypertrophic cardiomyopathy managed to reduce their standard of care medication doses by at least 50%. The results were presented at the European Society of Cardiology’s annual meeting. Cytokinetics is now preparing regulatory filings for aficamten.

Nxera Pharma gets $35M from Neurocrine Biosciences:
Nxera Pharma, formerly known as Sosei Heptares, has received a $35 million payment from Neurocrine Biosciences. The payment was triggered by the completion of a Phase 2 trial for its candidate in treating adults with schizophrenia. The trial met its primary endpoint, and Nxera plans to move the drug into Phase 3 trials next year.

Simcere Zaiming acquires cancer asset from TargetRx for $20M:
Simcere Zaiming has paid $20 million to Shenzhen TargetRx for the commercial rights to a Phase 1 antitumor small molecule candidate in China. The deal includes marketing rights to TGRX-326 and other benefits. Simcere will also compensate TargetRx for assisting in promoting the drug.

Calliditas Therapeutics to delist following acquisition by Asahi Kasei:
Calliditas Therapeutics will delist its American shares following its acquisition by Asahi Kasei, which now owns more than 90% of the company’s outstanding shares. The delisting is expected around September 23rd.

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