Following more than a decade of development and a priority review, Pfizer Inc. has won FDA approval for Daurismo (glasdegib), a new drug that, when used with low-dose cytarabine (LDAC), has been shown to increase overall survival vs. cytarabine alone by a median of about four months for certain patients intolerant of intensive chemotherapy. The regulator also approved a supplemental new drug application in a similar indication for Venclexta (venetoclax), a drug marketed by Abbvie Inc. and Genentech/Roche Holding AG.

For anyone not hip-deep in the hot field of artificial intelligence (AI), its role in drug discovery — or in any endeavor, for that matter — can feel vague, especially relative to the concrete problems drugmakers face daily. The very definition of AI can seem fuzzy, touted at times as capable of doing everything from organizing your photos to driving your car. Yet, in the biopharma space, as dollars flow in and candidates rise up, clarity is surfacing, too, program by program, bringing with it a more down-to-earth explanation of how the technology is reshaping the discovery enterprise.

Just months after Swedish Orphan Biovitrum AB (Sobi) acquired global rights to emapalumab from Swiss developer Novimmune SA in a CHF450 million (US$453 million) deal, the companies have won an on-time FDA approval for the ultra-rare disease therapy. Branded as Gamifant, Sobi will market it for the treatment of pediatric and adult patients with the life-threatening inflammatory condition primary hemophagocytic lymphohistiocytosis (HLH) who have refractory, recurrent or progressive disease or intolerance to conventional therapies. It's the first and only FDA-approved therapy for the indication.

Roivant Sciences Ltd. has agreed to pay Gyeonggi-do, South Korea-based Intron Biotechnology Inc. up to $667.5 million for global rights to an experimental therapy for treating infectious diseases caused by antibiotic-resistant staphylococci. The deal, focused on Intron's tonabacase (SAL-200), positions the Basel, Switzerland-based company to begin a phase II trial of SAL-200 in 2019, it said.

Dual disappointments from the challenging field of advanced lung cancer announced late last week highlighted continuing problems in addressing the disease, still the leading cause of cancer death in the U.S. despite substantial progress. Astrazeneca plc and biologics arm Medimmune reported that, for people with metastatic non-small-cell lung cancer (NSCLC), neither the anti-PD-L1 antibody Imfinzi (durvalumab) nor a combination of that drug with the anti-CTLA-4 candidate tremelimumab improved overall survival vs. standard-of-care chemotherapy for previously untreated patients in the phase III Mystic trial.

Dual disappointments from the challenging field of advanced lung cancer announced Friday highlighted continuing challenges in addressing the disease, still the leading cause of cancer death in the U.S. despite substantial progress. Astrazeneca plc and its biologics arm Medimmune reported that, for people with metastatic non-small-cell lung cancer (NSCLC), neither the anti-PD-L1 antibody Imfinzi (durvalumab) nor a combination of that drug with the anti-CTLA-4 candidate tremelimumab improved overall survival vs. standard-of-care chemotherapy for previously-untreated patients in the phase III Mystic trial. Astrazeneca's partner Hutchison China Meditech Ltd. (Chi-Med) also reported phase III results showing that, despite improving progression-free survival, its small-molecule inhibitor of VEGF receptors, fruquintinib, failed to improve OS for advanced NSCLC patients who had failed two lines of systemic chemotherapy.

Ramping up its long-running interest in epigenetics, Boehringer Ingelheim GmbH (BI) has turned to Epizyme Inc. to help it inhibit undisclosed targets in two families of enzymes linked to the development of lung and other solid tumor cancers, helicase and histone acetyltransferase (HAT). The collaboration includes $20 million in near-term payouts for Epizyme plus up to $280.5 million in potential milestone payments and royalties. The targets are compelling not only because of "the clear role they play in cancer" but because both have patient stratification biomarkers, Boehringer said. Epizyme shares (NASDAQ:EPZM) rose 25 cents, or 3.4 percent, to $7.51 on Thursday.

SAN FRANCISCO – Acquist Therapeutics Inc., a Westfield, N.J.-based company targeting enzymes that regulate both production and excretion of uric acid (UA), reported that in human liver cells treated with a nonalcoholic steatohepatitis (NASH) promoter, its lead candidate, ACQT-1127, blocked a 70-fold increase in UA by more than 98 percent and reduced by more than half a marked increase in triglycerides. The candidate also brought dysregulated levels of seven NASH-related activators back within normal range, it said.

Ribometrix Inc., a Durham, N.C.-based startup developing small-molecule drugs to modulate RNA activity, has raised $30 million in a series A financing. The round, led by Merck KGaA's M Ventures, will help the company advance its discovery platform and its internal pipeline, it said.

SAN FRANCISCO – New data from two midstage studies of Gilead Sciences Inc.'s farnesoid X receptor (FXR) agonist reported during the American Association for the Study of Liver Diseases meeting showcased the company's ongoing efforts to establish new strengths beyond viral hepatitis C, where time and competition have eroded its dominance. Though still active on the next frontier of the hepatitis battle, hepatitis B virus, most of the company's liver disease pipeline today is focused on nonalcoholic steatohepatitis (NASH), primary sclerosing cholangitis (PSC) and primary biliary cirrhosis (PBC).