Novo Holdings led Asceneuron’s $100 million Series C financing. The Merck Serono spinout’s lead program is an oral small molecule designed to prevent aggregation of tau protein in neurodegenerative disorders.
By Frank Vinluan Alzheimer’s disease patients can now choose between new two intravenously infused therapies that work by breaking up plaques of amyloid protein in the brain. Asceneuron takes a different approach with oral therapies that address the buildup of tau, a protein that’s also associated with the neurodegenerative disorder. The biotech now has $100 million to advance its lead program to Phase 2 testing.
The Series C financing announced Tuesday was led by Novo Holdings, the company that manages the assets of the Novo Nordisk Foundation, the controlling shareholder of metabolic drug giant Novo Nordisk.
Using a drug to target and break up pathological proteins associated with Alzheimer’s has been validated by Eisai’s Leqembi, approved by the FDA last year, and Kisunla, an Eli Lilly drug that won its regulatory nod this month. But infusions of these amyloid-busting antibody drugs come with the risk of bleeding and swelling complications in the brain.
Asceneuron’s lead drug candidate ASN51 is a small molecule designed to block OGA, an enzyme that plays a role in protein aggregation. By preventing tau aggregation, the drug is intended to slow Alzheimer’s progression. In Phase 1 tests in healthy volunteers, Asceneuron said results showed its brain-penetrating drug was taken up by the central nervous system and hit a high percentage of its OGA target enzymes. The next step is a Phase 2 clinical trial to assess the drug’s effect in Alzheimer’s patients.
Other companies are in various stages of development with drugs that target tau. AC Immune’s pipeline lists an anti-tau immunotherapy in mid-stage development under a partnership with Johnson & Johnson. Switzerland-based AC Immune also has anti-tau small molecules in preclinical development under a partnership with Eli Lilly. Meanwhile, Takeda Pharmaceutical is pursuing anti-tau small molecules under a research collaboration with Cure Network Dolby Acceleration Partners.
Asceneuron was founded in 2012, spun out of Merck Serono. The startup’s research has produced two clinical-stage neuro drugs. In addition to ASN51, Asceneuron developed ASN90, an experimental treatment for progressive supranuclear palsy (PSP), a rare neurological disorder associated with tau aggregation. Like the Asceneuron Alzheimer’s drug candidate, ASN90 is an oral small molecule designed to inhibit the OGA enzyme. Last year, Barcelona-based Ferrer paid an undisclosed sum to license global rights to ASN90 for PSP.
Asceneuron’s latest financing added new investors EQT life Sciences – LSP Dementia Fund, OrbiMed, and SR One. Earlier investors M Ventures, Sofinnova Partners, GSK Equities Investments Limited, and Johnson & Johson Innovation—JJDC also participated in the financing. With the new capital, Asceneuron said it plans to start a Phase 2 study this year for its Alzheimer’s disease drug. The startup says its approach to protein aggregation has potential applications in other neurodegenerative diseases. Asceneuron will also use its new capital to advance pre-clinical programs in Parkinson’s, amyotrophic lateral sclerosis, and other neurodegenerative indications.
“This high caliber life sciences investor syndicate further validates the potential of our OGA inhibitor pipeline and leadership in the field of tauopathies,” Asceneuron CEO Barbara Angehrn Pavik said in a prepared statement. “We are excited to advance our lead asset ASN51 into Phase 2 clinical development, recognizing its potential to significantly expand treatment options for patients with Alzheimer’s disease.”
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