Christmas came early for a number of biopharma companies this year as eight companies collectively raised $3.24 billion in public offerings. Both Structure Therapeutics Inc. and Terns Pharmaceuticals Inc. announced upsized offerings of $650 million each, and Kymera Therapeutics Inc. raised $602 million, placing all three in the top 10 follow-on offerings of the year.
With new results from Wave Life Sciences Ltd., Structure Therapeutics Inc. and Ascletis Pharma Inc., obesity management drugs continue to move forward in producing weight loss and move the market.
The activin E mechanism of action in obesity is having something of an early stage heyday with such players as Wave Life Sciences Ltd. and Ibio Inc. talking up data that have perked the ears of Wall Street.
As Wave Life Sciences Ltd. released more results from its ongoing phase Ib/IIa study of small interfering RNA editing oligonucleotide WVE-006 for treating alpha-1 antitrypsin deficiency, the company’s stock (NASDAQ:WVE) dropped by 16.8% to close at $8 on Sept. 3.
At the recent American Society of Gene and Cell Therapy (ASGCT) meeting, Wave Life Sciences Ltd. presented siRNAs designed to suppress expression of the liver gene inhibin subunit β E (INHBE). Human genetic data show that heterozygous INHBE loss-of-function carriers exhibit a healthy metabolic profile.
Wave Life Sciences Ltd. plans to file with the U.S. FDA for accelerated approval of WVE-N531, an exon skipping oligonucleotide for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping, a decision based on favorable data from the phase II Forward-53 study. The analysis was conducted after 48 weeks of treatment with 10 mg/kg of the drug dosed every two weeks. Forward-53 achieved all trial goals, turning up sustained exon skipping, muscle concentrations, and dystrophin restoration through 48 weeks and a 61-day tissue half-life that supports giving the DMD therapy once a month.
Wave Life Sciences Ltd. has submitted a clinical trial application (CTA) for WVE-007, an investigational GalNAc-conjugated small interfering RNA (siRNA) designed to silence inhibin βE (INHBE) gene expression, for the treatment of obesity.
While RNA-medicine developer Wave Life Sciences Ltd. brought in a clinical data win, it also got knocked back a step as a major collaborator will go its separate way. That didn’t stop Wave’s stock from standing strong on the day. The company’s ongoing phase Ib/IIa study of its A-to-I RNA editing oligonucleotide produced positive proof-of-mechanism data in treating alpha-1 antitrypsin deficiency, a rare, genetic condition that can lead to lung and liver disease.
Interim phase II data of Wave Life Sciences Ltd.’s oligonucleotide, WVE-N531, revealed “impressive” dystrophin expression, solid safety and the potential for once-monthly dosing for boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping. The findings drove Wave Life’s stock (NASDAQ:WVE) up by 53.4%, or $2.85, to close Sept. 24 at $8.19, after peaking earlier in the day at $8.35, its 52-week high.
