The hematology world is a-twitter about the imminent approval of Zynteglo (betibeglogene darolentivec), the lentiglobin cell and gene therapy from Bluebird Bio Inc. that’s expected to become the first gene therapy to address the blood disease beta-thalassemia and the company’s first product to market. 

Next-gen bispecific antibodies (BsAbs) are on the move. The blockbuster potential of the class has galvanized biopharma, with deals and assets racing to capture the pole position as the entire field gains speed.

Next-gen bispecific antibodies (BsAbs) are on the move. The blockbuster potential of the class has galvanized biopharma, with deals and assets racing to capture the pole position as the entire field gains speed.

As the 37th Annual J.P. Morgan (JPM) Healthcare Conference was drawing to a close, a research team led by investigators from the NIH and Global Good generated considerable buzz with the disclosure of a computer algorithm that can analyze digital images of a woman's cervix and accurately identify precancerous changes that require medical attention. Researchers said the artificial intelligence (AI) approach, dubbed automated visual evaluation, offered the potential to transform cervical cancer screening, particularly in low-resource settings.

Ironwood Pharmaceuticals Inc. initiated a phase I study in healthy volunteers with IW-6463, its oral central nervous system (CNS)-penetrant soluble guanylate cyclase (sGC) stimulator, aimed at the treatment of serious and orphan CNS disorders, potentially including vascular dementia and Alzheimer's disease (AD). Data from the study are expected in the second half of the year, positioning IW-6463 to follow several ongoing programs into midstage development, but not by Ironwood. Instead, the sGC stimulator portfolio is set to transition to spinout Cyclerion Therapeutics Inc., with separation of the companies on track to complete in the first half of the year.

Elsalys Biotech SAS, a 2013 spinout from Transgene SA, achieved a significant milestone with the finding that lead candidate inolimomab (Leukotac) showed clinical benefit during long-term follow-up of up to 8.5 years in individuals treated in the phase III study in acute steroid-resistant graft-vs.-host disease (SR-aGVHD). The positive overall survival (OS) outcome in the long-term analysis positions the Lyon, France-based company for a regulatory filing in the EU next year and puts discussions with commercial partners on the front burner – admirable achievements for a company of just 16 employees that's raised less than €20 million (US$22.8 million).

Wren Therapeutics Ltd. chirped about its £18 million (US$23.3 million) series A round, raised from an international syndicate led by the Baupost Group with participation from Lifeforce Capital and high net worth individuals. The Cambridge, U.K.-based company, a 2016 spinout from the University of Cambridge and Sweden's Lund University, declined to warble about the details, but Wren is known to be focused on protein misfolding diseases – a broad development area where it has its pick of some four dozen indications.

Two weeks after Bristol-Myers Squibb (BMS) Co.'s historic $74 billion takeover bid, Celgene Corp. went on its own dealmaking spree, adding pacts with Kyn Therapeutics Inc. and Obsidian Therapeutics Inc. to its immuno-oncology (I-O) fold. Celgene, of Summit, N.J., gained an exclusive option to aryl hydrocarbon receptor (AHR) antagonist and kynurenine-degrading enzyme, or Kynase, programs from Kyn in return for $80 million up front, an equity stake and undisclosed milestone payments and sales royalties. The deal with Obsidian – financials entirely undisclosed – entails a multiyear collaboration that entitles Celgene to exclusive options to license cell therapies that use Obsidian's Destabilizing Domain (DD) technology to modulate IL-12 and CD40L activity.

In a major setback for the soft tissue sarcoma (STS) space – a complex disease with a dearth of treatment options – Eli Lilly and Co. said the confirmatory phase III ANNOUNCE study of Lartruvo (olaratumab) in combination with doxorubicin failed to meet the primary endpoints of overall survival (OS) in the full study population in advanced or metastatic STS or in the leiomyosarcoma (LMS) subpopulation.