With a successful phase III study of plozasiran in hand, Arrowhead Pharmaceuticals Inc. plans to file an NDA with the U.S. FDA for treating the rare genetic disease familial chylomicronemia syndrome. The Palisade study hit its primary and multiplicity-controlled, key secondary endpoints, including statistically significant reductions in triglycerides, apolipoprotein C-III and the amount of acute pancreatitis in participants. There are no currently approved treatments in the U.S. for the syndrome. Arrowhead said it intends to file the NDA by the end of this year. Arrowhead has plenty of competition in the indication from Ionis Pharmaceuticals Inc., which produced positive phase III data in April using olezarsen. On June 26, the FDA accepted the NDA for olezarsen for priority review with a PDUFA action date of Dec. 19, 2024.

Dyne data deliver on dystrophin, but stock stalls on side effects

While Dyne Therapeutics Inc.’s DYNE-251 demonstrated high levels of dystrophin expression and functional improvement in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping, investors focused on three serious adverse events related to the drug, driving shares (NASDAQ:DYNE) down by 49% to a low of $23.55 in early trading on Sept. 3. The three treatment-emergent adverse events (TEAEs) occurred in two patients who were part of a 40-mg/kg dosing cohort and included acute kidney injury, thrombocytopenia and pancytopenia. They have since recovered. As a result of the TEAEs, however, all participants receiving the high dose were dropped down to the 20-mg/kg dose. Based on phase I/II Deliver results in which patients treated with 20 mg/kg of DYNE-251 every four weeks reached a mean absolute dystrophin expression of 3.71% of normal, the company intends to move into registrational cohorts.

Jacobio, Allist shake on ¥900M China deal for lung cancer combo

Jacobio Pharmaceuticals Group Co. Ltd. out-licensed rights for two lung cancer assets in China to Shanghai Allist Pharmaceuticals Co. Ltd. through a potential ¥900 million (US$126.4 million) deal. Beijing-headquartered Jacobio said Aug. 30 that it signed off development, regulatory and commercial milestone rights to both glecirasib, a KRAS G12C inhibitor first targeting non-small-cell lung cancer, and a SHP2 inhibitor called JAB-3312.

Vaxcyte climbs on phase I/II data with pneumococcal vaccine

Shares of Vaxcyte Inc. (NASDAQ:PCVX) were trading at $115.92, up $35.16, or 43%, on positive top-line results from the phase I/II study testing the safety, tolerability and immunogenicity of VAX-31, the San Carlos, Calif.-based firm’s 31-valent pneumococcal conjugate vaccine candidate designed to prevent invasive pneumococcal disease in 1,015 healthy adults aged 50 and older. The product was well-tolerated and turned up a safety profile at all doses studied through the full six-month evaluation period similar to Prevnar 20, the 20-valent conjugate vaccine from Pfizer Inc. that was first approved by the U.S. FDA in June 2021. An adult phase III program with VAX-31 is planned.

George’s triple-drug hypertension combo succeeds in phase III

George Medicines Ltd. has published positive data from two phase III studies showing GMRx2, a low-dose triple-drug combination treatment for hypertension, is superior to dual combinations of its components. The data, presented at the European Society of Cardiology meeting in London, Aug. 31, show the combination of the generic drugs telmisartan, amlodipine and indapamide was more effective than any of three dual therapies, significantly reducing both home- and clinic-measured blood pressure and improving blood pressure control rates.

Sanofi BTK inhibitor tolebrutinib hits phase III endpoints in MS

Sanofi SA’s brain-penetrant Bruton's tyrosine kinase (BTK) inhibitor, tolebrutinib, met the primary endpoint in the phase III Hercules trial in non-relapsing secondary progressive multiple sclerosis (nrSPMS). The first compound to show reduction in disability accumulation in MS, tolebrutinib delayed the time to onset of confirmed disability progression in people with nrSPMS, a population for which there are currently no approved therapies.

ARDD 2024: Age without disease, exercise muscles and win $111M

Aging is part of the life cycle and, although the effects are not manifest until after adulthood, it actually occurs from birth. The concept of senescence has traditionally been associated with aging. However, an embryo has senescent cells. In that case, what is aging, how can it be measured, and from what point in the life cycle? The 11th Aging Research & Drug Discovery Meeting (ARDD), Aug. 26-30, 2024, in Copenhagen, has brought together experts to discuss the latest advances in the field, establish consensus and debate objectives.

Holiday notice

BioWorld's offices were closed in observance of Labor Day in the U.S. No issue was published Monday, Sept. 2.

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