SAN FRANCISCO - As the number of clinical trials testing checkpoint inhibitors in combination immunotherapies climbs ever-skyward, efforts to find ideal synergies have met both disappointments and challenges. Executives in the thick of efforts to identify the next best moves in the field highlighted some of the setbacks and the need to set aside haste in favor of careful science, to be open to new possibilities and to re-examine which endpoints might provide the best measures in new trials.

Krystal Biotech Inc. is moving ahead with plans to start a pivotal phase III test of its off-the-shelf topical gene therapy for dystrophic epidermolysis bullosa (DEB) in the second half of 2019 after interim data from two patients in its ongoing phase I/II test met all primary efficacy endpoints. Pivotal testing of the candidate, KB-103, is expected to last about six months, with a potential six-month FDA review to follow. Company shares (NASDAQ:KRYS) climbed 27.6 percent on the news to close at $20.

Shares of Immune Design Corp. hit an all-time low Friday after the company scrapped phase III testing for one of its two lead candidates, the prime-boost cancer vaccine CMB-305 in synovial sarcoma. Signs that combining it with Tecentriq (atezolizumab, Roche Holding AG) in a phase II trial is unlikely to offer a survival benefit for relapsed synovial sarcoma patients also shaped the decision. All efforts at the West Coast company will now shift to its second lead cancer vaccine, the intratumoral TLR4 agonist G-100.

SAN FRANCISCO – Despite tremendous progress in the field of immunotherapy, leading immunologist Jeff Bluestone told an audience at the University of California, San Francisco Tuesday that significant questions remain around how to best navigate the field's future. Bluestone, a professor at UCSF and head of the Parker Institute for Cancer Immunotherapy, said that addressing combinations, understanding immune-related adverse events and autoimmunity will be key in advancing understanding of how to employ what he called an "explosion of new drugs."

SAN FRANCISCO — Summer's definitive failure of Summit Therapeutics plc's former lead candidate, ezutromid, led to disappointments across the Duchenne muscular dystrophy (DMD) community. Now with its pivot to antibiotics complete, its team expects to have one of just two novel mechanisms in the field's phase III lineup next year with ridinilazole. (See BioWorld, June 28, 2018.)

SAN FRANCISCO - With Motif Bio plc's new drug application for iclaprim in acute bacterial skin and skin structure infections awaiting a February 2019 review at the FDA, the company leveraged IDWeek to unveil new wound care data from its pivotal trials highlighting a potential advantage in the realm of kidney safety.

A U.S. subsidiary of Italy's privately held Leadiant Biosciences SpA has won FDA approval for Revcovi (elapegademase-lvlr), a new recombinant enzyme replacement therapy (ERT) for the ultra-rare genetic disorder adenosine deaminase severe combined immune deficiency. The condition, which affects as few as one in 1 million live births in the U.S., leaves infants and young children vulnerable to infections and can be fatal if not diagnosed and treated by age 2.

SAN FRANCISCO – Feeling unloved by Wall Street, undercompensated by payers and abandoned by big pharma, members of the Antimicrobials Working Group (AWG) and other key stakeholders gathered Friday morning at IDWeek to chart a path out of dire market circumstances.

SAN FRANCISCO – Nearly half of outpatient antibiotics are prescribed without an infection-related diagnosis, according to a new study, funded by the U.S. Agency for Healthcare Research and Quality. Though overprescribing of antibiotics is already a high-profile issue, the study is thought to be the first to look at overall outpatient antibiotic prescribing. It analyzed more than a half million prescriptions from 514 outpatient clinics.

SAN FRANCISCO — With progress toward a universal flu vaccine on the march, the global fight against influenza is building momentum. But as epidemics still lead to millions of severe cases of the infection each year, hospitalization and death are still stark realities for many patients. Now Roche Group and its partner, Shionogi & Co. Ltd., could soon add a new weapon to America's annual battle with the illness: a single-dose, oral treatment called baloxavir marboxil. Data the company is presenting at IDWeek on Saturday suggest the new medicine – up for priority review at the FDA with a Dec. 24 PDUFA date – could potentially offer significant benefits to both people at high risk for flu complications and those infected with type B strains, a subgroup where some antivirals have shown only limited efficacy or inconclusive data.