ATL-201

For five years, Atalanta Therapeutics operated with one investor. Now, F-Prime Capital is getting a bevy of financial support to propel two of the Boston biotech’s RNAi drugs for rare neurological disorders into human trials. Atalanta said Tuesday that investors in its $97 million Series B include EQT Life Sciences, the corporate venture arms of Sanofi and Novartis, RiverVest Venture Partners, funds managed by abrdn, Pictet Alternative Advisors, Mirae Asset Financial Group and GHR Foundation. The oversubscribed funding came at a step-up, CEO Alicia Secor said in an interview, and that was welcome news after the biotech had layoffs and lost a partnership with yearslong collaborator Biogen last year. Secor said Atalanta regained the rights to two undisclosed preclinical programs it was working on with Biogen, which is currently going through its own reshuffling, Endpoints News reported last week. Atalanta’s partnership with Roche’s Genentech remains ongoing and is “absolutely thriving,” Secor said. The startup emerged in January 2021 with $110 million by way of F-Prime’s funding and payments from Genentech and Biogen. The biotech was co-founded by Nobel laureate Craig Mello — one of RNAi’s pioneers — in addition to UMass Chan Medical School’s Anastasia Khvorova and Neil Aronin, an expert in Huntington’s disease and practicing endocrinologist. In the field of RNAi, Atalanta hopes to distinguish itself with a divalent construct. “We have two siRNA duplexes joined by a linker,” Secor said. “The chemistry is what imparts different results from anything that has been out there. So what we see is unprecedented knockdown [and] broad distribution across all brain regions.” Alnylam broke open the field for RNAi medicines, having secured multiple drug approvals since Onpattro’s launch in 2018. Its longtime former CEO, John Maraganore, revealed a new biotech last year to work on the next generation of RNAi candidates. “We feel like we have potentially the best-in-class oligo platform for CNS delivery,” Secor said. “It’s been one of the holy grails. I think oligonucleotides have proven their value. RNAi, as a technology, is really expanding into all different tissue types.” The neuroscience field, one of the riskiest in clinical development, has seen a resurgence in recent years, but the “universe of CNS targets remains untapped in so many ways,” Secor said. “We know that our technology can basically knock down any transcript in the CNS.” Atalanta plans to ask health regulators to clear its first two clinical trials later this year for ATL-201 and ATL-101 in patients with KCNT1-related epilepsy and Huntington’s disease, respectively. A few thousand infants and children in the US, and a similar number in Europe, have the genetic KCNT1 form of epilepsy, which leads to about 20 to 50 seizures per day, Secor said, and the mortality rate is about 50% by age 10. In preclinical studies in mice with seizures, Atalanta’s therapy showed a 70% reduction in seizures by knocking down the levels of KCNT1 by 50%, according to Secor. She said the “amazing preclinical data” have not yet been made public. Atalanta is also researching additional targets for Huntington’s, including MSH3, Secor said. It’s workshopping programs in Alzheimer’s disease (CD33 and APOE), pain and other CNS disorders. In pain, it’s looking at trigeminal neuralgia. And it’s pursuing NaV1.7, a sodium channel that’s been targeted for years by some pharmas. Vertex , SiteOne Therapeutics and others are also going after a similar pain-signaling channel called NaV1.8. “It’s about time we had a revolution in the industry for non-opioid pain solutions,” she said.