Following the company’s COVID-19 manufacturing misstep, Emergent Biosolutions Inc. continued its struggle to rebalance as top-line data from a phase III study it participated in failed to hit its primary endpoints with statistically significant results. The data showed that adding anti-SARS-CoV-2 hyperimmunoglobulin to standard of care, inclusive of remdesivir, for hospitalized adult COVID-19 patients with symptoms for less than 12 days failed to provide clinical benefit when compared to standard of care plus placebo. The global, multicenter double-blind, placebo-controlled randomized study assessed the safety and efficacy of four immunoglobulin candidates, the SARS-CoV-2 immune globulin intravenous (human) (COVID-HIG) plus standard of care.
If these top-line phase III results were a grade card for 12- to 15-year-olds, they would be on the honor roll: The Pfizer Inc.-Biontech SE COVID-19 vaccine is 100% effective for the age group and produced robust antibody responses.
Charlene Liao, Immune-Onc Therapeutics Inc.’s CEO told BioWorld she has a solid plan for using the company’s new $73 million series B1 and B2 financing. “It is time to go beyond T cells and to consider myeloid checkpoints as the next wave of cancer immunotherapies,” she said. Immune-Onc will use the funding to target myeloid checkpoints, especially leukocyte immunoglobulin-like receptor subfamily B, as it continues to develop its blood cancer and solid tumor therapies.
It’s a first for Larry Miller. In his 30 years of working in pharma, he has never run a company that didn’t have a pack of near competitors scrambling to develop a therapy. “Not even close,” he told BioWorld. Miller, the CEO of Apnimed Inc., just saw the company close on a $25 million series B to help drive its lead program, a once-daily, oral obstructive sleep apnea (OSA) therapy, into a phase III registrational trial.
Top-line from Orphazyme A/S’ phase II/III trial of arimoclomol for treating inclusion body myositis, a muscle-wasting disease, failed to hit its primary and secondary endpoints. The data caused investors to pull back sharply as shares of Copenhagen-based Orphazyme (NASDAQ:ORPH) had dropped 28.97% on March 29 to close at $8.80 per share.
RNA therapy developer Proqr Therapeutics NV’s phase I/II study of adults with Usher syndrome and non-syndromic retinitis pigmentosa met all its key objectives, prompting the company to plan two parallel pivotal phase II/III studies that could start by year-end. The company wasted no time as it has already discussed next steps with the FDA to support the therapy’s registration “as soon as possible,” Aniz Girach, Proqr’s chief medical officer, told investors during a March 24 investor call.
A phase IIa study of Frequency Therapeutics Inc.’s lead candidate in treating hearing loss showed it fared no better than placebo, but the company said its faith in the candidate is bolstered by the belief the study was hampered by trial design issues and by data from another study.
Three companies set terms for their IPOs that, if launched, will go far in maintaining the year’s already powerful momentum. Largest of the three comes from Design Therapeutics Inc., which looks to raise net proceeds of $209.1 million to fund its Friedreich’s ataxia program through IND studies and a phase I trial.
Following a missed primary endpoint in its phase III study in treating anti-PD-1-refractory advanced melanoma, Idera Pharmaceuticals Inc. is considering stopping the registrational trial of its synthetic Toll-like receptor 9 (TLR9) agonist or pressing on toward the clinical trial’s second primary endpoint.
Merck & Co. Inc. is bringing in Amathus Therapeutics Inc. to collaborate on developing small molecules to treat neurodegenerative disease. The deal is one of several in the past 18 months that has expanded Merck’s presence in the space.
