News briefing: Ionis reads out positive data for prekallikrein-targeting drug; PPD collaborates on lupus research
In a compassionate-use pilot study for hereditary angioedema (HAE), Ionis’ PKKRx and PKK-LRx were shown to reduce the rate of breakthrough attacks in two patients.
PKK-LRx is currently in Phase II development to treat the rare genetic disease, which causes rapid and painful inflammation attacks in the hands, feet, limbs, face, abdomen, larynx and trachea. The ligand-conjugated antisense (LICA) oligonucleotide is designed to reduce the production of prekallikrein (PKK).
Two patients in the compassionate-use study — 24- and 27-year-old women with severe bradykinin-mediated forms of angioedema — were given the unconjugated parent drug PKKRx, followed by ligand-conjugated PKK-LRx.
The mean monthly attack rate decreased from 1.2 to 0.25 for Patient 1, and from 7.9 to 1.0 in Patient 2, according to a report in the New England Journal of Medicine. And no adverse effects were reported.
“These findings support continued development of the ligand-conjugated ASO inhibitor of hepatic prekallikrein expression for the treatment of patients with severe hereditary angioedema who have limited response to or unacceptable side effects from current therapies,” the report states. — Nicole DeFeudis
PPD partners with Lupus Research Alliance affiliate to advance lupus research
PPD has entered into a collaboration deal with Lupus Therapeutics, an affiliate of the Lupus Research Alliance, to expand lupus research for the CRO’s global customer base.
With the deal, PPD will be able to optimize its trials and protocols thanks to LT’s own clinical investigator network. LT in turn gets access to better medicines and therapies for its own patients, the companies said. Financial terms of the deal were not disclosed.
Through that network, PPD will get access to more than 50 research sites with over 200 affiliated investigators, who collectively see more than 20,000 active systemic lupus erythematosus (SLE) patients.
PPD’s CMO Karen Kaucic said in a statement that the CRO is excited to partner with a company that has “site staff who have experience across multiple studies, a known, reliable network of investigators and sites ready to initiate clinical trials awarded work.”
Lupus is a chronic condition in which a person’s immune system attacks its own tissues and organs, causing inflammation that can damage any part of the body. Lupus is difficult to diagnose because its signs and symptoms can imitate a wide variety of other ailments. — Max Gelman
Magenta CFO hits the exit as the CEO brings in 2 new top execs for the team
There’s a shakeup underway in the top team running Magenta Therapeutics.
CEO Jason Gardner is now looking for a replacement for chief operating and financial officer Jason Ryan, who’s switching to a consultancy role for personal reasons.
Then there are two new execs: Lisa Olson, a longtime AbbVie vet, is taking the CSO position while Kevin Johnson moves into a post running regulatory and quality control. He had been in pretty much the same job at Imara and got his PhD at the UNC School of Medicine. — John Carroll
Xencor collabs with MD Anderson on tumor treatments
California-based biotech Xencor and the University of Texas MD Anderson Cancer Center are teaming up on the treatment for a variety of cancers.
The deal will run for the next five years as the two parties study Xencor’s portfolio of XmAb drug candidates. This includes bispecific antibodies as well as engineered cytokines, and the biotech currently has nine bispecifics and one cytokine in Phase I trials.
Among Xencor’s bispecific candidates are potential treatments for acute myeloid leukemia, B cell malignancies and neuroendocrine tumors and gastrointestinal stromal tumors. The biotech is also developing bispecifics that activate within the tumor microenvironment. — Max Gelman