Coave Therapeutics aims to move its lead gene therapy program, CTx-PDE6b, for a form of retinitis pigmentosa (RP) into a pivotal trial in 2025 on the back of a phase I/II study, which uncovered a preliminary efficacy signal in one patient subgroup.
Pfizer Inc. has positive phase III data for its hemophilia treatment as it wades deeper into an indication that already has plenty of competition and at least one company with earnings of more than $1 billion. Pfizer’s marstacimab in treating hemophilia could lead to the first once-weekly subcutaneous treatment for hemophilia B and could end up being the first treatment administered as a flat dose for treating hemophilia A or B.
Regenerative medicine product Stemchymal, an allogeneic adipose-derived mesenchymal stem cell treatment, missed the primary efficacy endpoints in two phase II spinocerebellar ataxia trials conducted in Japan and Korea, but a subpopulation analysis showed efficacy signals in patients with more severe conditions, Reprocell Inc. reported.
Exact Sciences Corp.’s long-term analysis of results from the Detecting cancers Earlier Through Elective mutation-based blood Collection and Testing (DETECT-A) study found that all patients diagnosed and treated for stage I or II cancers identified through its blood-based multi-cancer early detection (MCED) test remained cancer-free more than four years after diagnosis. Half of all the participants with cancer detected remained alive at four years, notable because most of the detected cancers had no recommended screening tests or standards. The results will be presented at the American Society of Clinical Oncology meeting June 2-6.
Renal denervation (RDN) was described as the “comeback kid” at the recent EuroPCR conference in Paris where participants argued that following the success seen in several sham-controlled trials, there is now no doubt about the safety and the efficacy of the technology as a treatment for hypertension. The device-based procedure should now be used as a therapy option to reduce high blood pressure in patients, delegates heard.
Lack of efficacy brought the development of two investigational agents for amyotrophic lateral sclerosis (ASL) to a halt over the past week. On May 23, Wave Life Sciences Inc. disclosed that its stereopure antisense oligonucleotide WVE-004 failed to demonstrate clinical benefit after 24 weeks of treatment on a phase Ib/IIa trial in familial ALS patients or frontotemporal dementia patients. And on May 25, Apellis Pharmaceuticals Inc. and its partner, Swedish Orphan Biovitrum International AB, said that pegcetacoplan failed to meet its primary endpoint of a one-year phase II trial in patients with sporadic disease.
The U.K. government has announced £121 million (US$148 million) in funding to reboot the commercial clinical trials system after a sharp decline saw the number of industry-sponsored studies falling by 44% from 2017 to 2021.
Reddress Ltd. secured $26 million in a series D financing aimed at further advancing its autologous, point-of-care wound management solution. The funds will be used to grow uptake of its Actigraft product suite, pursue global partnerships and expand its blood-based technology to treat more conditions.
Providing no details, Mirati Therapeutics Inc. failed to meet its overall survival primary endpoint in the phase III Sapphire trial with sitravatinib. The receptor tyrosine kinase inhibitor was tested in combination with anti-PD-1 checkpoint inhibitor Opdivo (nivolumab, Bristol Myers Squibb Co.) vs. docetaxel in patients with second- or third-line advanced nonsquamous non-small-cell lung cancer. According to Mirati, this patient population consists of about 70,000 people in the U.S. and Europe who have derived prior benefit from a PD-(L)1 inhibitor.