With an eye to sustaining its multiple sclerosis (MS) franchise, Sanofi SA plucked a Bruton's tyrosine kinase (BTK) inhibitor from Principia Biopharma Inc. The Paris-based pharma agreed to pay $40 million up front, with milestone payments that could reach $765 million, for the oral candidate, PRN-2246, which recently became the subject of a phase I study in healthy volunteers.

Fortuna Fix Inc. is seeking to become the first company to advance a program into the clinic that uses autologous neural stem cells produced by direct reprogramming, dubbed drNPCs, to replace neuronal tissue lost to neurodegeneration or brain trauma.

In presentations at the American Society of Nephrology Kidney Week in New Orleans, some biopharmas looked to push the envelope while others sought redemption. The take-home message from abstracts and experts was that innovative leaps remain elusive in the field, foiled to an extent by challenging endpoints sought by regulators. But thanks to collaborative efforts, trial designs are starting to change and could yield breakthroughs in the years ahead.

Presenting during the late-breaking clinical trial posters at Kidney Week in New Orleans, Reata Pharmaceuticals Inc. did not disappoint with primary 12-week data from the phase II portion of its CARDINAL trial of bardoxolone methyl (bard) to treat chronic kidney disease (CKD) caused by Alport syndrome.

Bolstered hours earlier by FDA approval of the oral small-molecule Bruton's tyrosine kinase (BTK) inhibitor Calquence (acalabrutinib), Astrazeneca plc and its biologics research and development arm, Medimmune, quietly posted a miss for the phase III STRATOS 2 and TROPOS trials of tralokinumab, an anti-interleukin-13 (IL-13) immunoglobulin (IGE)-G4 monoclonal antibody, in severe, uncontrolled asthma.

All therapies targeting CD123 are not created equally, Stemline Therapeutics Inc. CEO Ivan Bergstein told BioWorld after the company reported top-line data confirming that the phase II pivotal trial of SL-401 hit its primary endpoint for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). Although other modalities, from CAR Ts to bispecifics, are directed to the interleukin-3 receptor, "you can't necessarily predict the safety and efficacy of a drug just because it hits the same target as another drug," Bergstein said.

Six quarters after it was co-founded, seeded and incubated by Atlas Venture, Kymera Therapeutics LLC debuted with a $30M series A round led by Atlas, with participation from Lilly Ventures and Amgen Ventures. In the words of Atlas partner Bruce Booth, co-founder and chairman, the Cambridge, Mass.-based firm is seeking to "drug the undruggable" by harnessing the power of targeted protein degradation to deliver therapeutic benefits.

Abbvie Inc. put competitors on notice by reporting that three phase III trials of its IL-23 antagonist, risankizumab (formerly BI-655066 and ABBV-066), that collectively enrolled more than 1,600 patients met the co-primary and ranked secondary endpoints, achieving greater efficacy than Stelara (ustekinumab, Johnson & Johnson) and its legacy drug, Humira (adalimumab), in patients with moderate to severe plaque psoriasis.

Macrogenics Inc. landed Incyte Corp. as an immuno-oncology (I-O) partner, securing $150 million up front for a collaboration and license agreement covering MGA-012, its phase I monoclonal antibody (MAb) that inhibits programmed cell death protein 1 (PD-1). Incyte gained exclusive global rights to develop and commercialize MGA-012 in all indications, although Macrogenics held on to the right to develop the asset in combination with other candidates in its pipeline. Should any of these combinations gain approval, Incyte will commercialize MGA-012 and Macrogenics will commercialize its internal assets.

With its lead asset in the clinic to assess its effectiveness to prevent Clostridium difficile (C. diff) infection (CDI), Finch Therapeutics Inc. looked two miles down the road and took a calculated step to expand its presence in the microbiome field, orchestrating a merger with neighboring Crestovo LLC, whose phase II CDI asset, CP-101, was deemed superior.