Kallyope Inc., a company leveraging connections between the gut and brain to develop new medicines for diabetes, obesity and other diseases, has raised $236 million in series D financing to support its work. Readouts of early clinical data for its most advanced programs, phase I small molecules for metabolic disease and gut barrier conditions, will start to arrive later this year, company CEO and President Jay Galeota told BioWorld.
The FDA has placed Logicbio Therapeutics Inc.’s phase I/II clinical trial of LB-001, an investigational AAV genome-editing therapy for treating pediatric patients with methylmalonic acidemia (MMA), on a clinical hold. So far, four patients have been dosed in the study and two have had serious adverse events related to the candidate, the company’s lead asset.
Despite success in other parts of the world, Opko Health Inc. and Pfizer Inc. are still struggling to gain U.S. FDA approval for the recombinant human growth hormone somatrogon in treating pediatric patients, drawing a complete response letter (CRL) with their BLA. The delay caused by the setback gives Skytrofa (lonapegsomatropin) from Ascendis Pharma A/S a chance to charge even further ahead in the pediatric market.
LONDON – Evotec SE put the biggest ever headline figure on one of its pharma drug discovery collaborations, announcing a $1 billion deal with Eli Lilly and Co. in metabolic diseases. The Hamburg, Germany-based company will be responsible for the discovery of drug candidates for the treatment of diabetes and chronic kidney disease against targets identified by Lilly or Evotec, or sourced externally. Lily has rights to up to five programs, to be developed in the partnership and to take on any subsequent development, clinical validation and commercialization activities. The collaboration initially runs for three years.
After discussions with the FDA, Applied Therapeutics Inc. said it will again delay an NDA filing for its galactosemia treatment, AT-007, until it has more time to discuss the filing with the agency.
Avrobio Inc., stung by variable outcomes in a phase II test of its investigational Fabry disease therapy, is quitting further enrollment in the program, one of the first from its gene therapy platform, Plato. The team's attention will shift instead to other clinical-stage lysosomal disorder programs amid "an increasingly challenging market and regulatory environment for Fabry disease," the company said.
Despite hitting the primary endpoint in its phase III study of pegzilarginase in treating arginase 1 deficiency, Aeglea Biotherapeutics Inc. had a tough go of it Dec. 6 as the price of its stock (NASDAQ:AGLE) plunged 36.5% to close at $3.81 per share. The study hit its primary endpoint with a statistically significant reduction in plasma arginine from baseline after 24 weeks of treatment with pegzilarginase (p<0.0001). The study results were positive enough to prompt Aeglea to say it planned to submit a BLA to the FDA in the first half of 2022.
I-Mab Biopharma Co. Ltd. formed a partnership with Hubei Jumpcan Pharmaceutical Co. Ltd. in a deal worth up to ¥2.016 billion (US$315.2 million) to develop, manufacture and commercialize recombinant human growth hormone (rhGH) eftansomatropin alfa (TJ-101) in mainland China.
Sangamo Therapeutics Inc. rolled out pleasing preliminary data from the first four patients treated in the phase I/II study known as Staar, evaluating isaralgagene civaparvovec, or ST-920, a gene therapy for Fabry disease. Results as of the Sept. 17, 2021, cutoff date from the four patients in the first two dose cohorts showed that the drug was generally well-tolerated, and all four patients exhibited above normal alpha-galactosidase A activity.
Travere Therapeutics Inc.’s mid-September deal with Vifor Pharma Group – a collaboration and licensing agreement for the commercialization of sparsentan in Europe, Australia and New Zealand – brought further, well-deserved attention to the dual endothelin angiotensin receptor antagonist, in the works for rare kidney disorders, specifically focal segmental glomerulosclerosis and IgA nephropathy.
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