Privately held Ribometrix Inc. sealed a deal with Basel, Switzerland-based Roche Holding AG’s Genentech arm that brings $25 million up front and potential milestone payments beyond $1 billion as the pair discovers and develops RNA-targeted small-molecule therapeutics against several targets. Ribometrix, of Durham, N.C., deploys a platform with methods that include structural analytic capabilities to identify 3D motifs in RNA molecules and then design binding candidates for therapeutic effects. Under the terms, Ribometrix and Genentech will collaborate on the discovery and preclinical development, with Genentech taking programs the rest of the way. The agreement includes tiered royalties, too.

Mycovia antifungal poised for NDA filing after phase III wins

Mycovia Pharmaceuticals Inc. said new phase III data on its oral antifungal, oteseconazole, showed it prevented recurring vulvovaginal candidiasis (RVVC) episodes for about a year in 95% of women with the condition who received it. The results, which replicated earlier phase III findings, put a planned NDA submission on track for the first half of this year, CEO Patrick Jordan said. Competitor Scynexis Inc. submitted an NDA for its VVC candidate, ibrexafungerp, in October 2020.

Imara wraps up a phase IIa in sickle cell with mixed results

Imara Inc. reported a mixed bag of phase IIa study data for its lead candidate, IMR-687, for treating sickle cell disease in adults. The upside includes a favorable safety profile, lower vaso-occlusive crises (VOC) and sickle cell-related pain crises along with fewer VOC-related hospitalizations. On the downside, the study’s biomarker results showed no meaningful changes in F-cells, fetal hemoglobin levels or hemoglobin levels from baseline through week 24. IMR-687 is a small-molecule inhibitor of PDE9, which degrades cyclic guanosine monophosphate, an active signaling molecule. Investors didn’t seem to like the data much as Boston-based Imara’s stock (NASDAQ:IMRA) was taking a beating at midday, with shares sagging 36%.

Moderna’s COVID-19 vaccine second to clear EMA approval

LONDON – The EMA has recommended approval of Moderna Inc.’s COVID-19 vaccine, becoming the fourth western regulator to do so, after Israel approved the product on Jan. 5 and the FDA and Health Canada in December. Moderna’s is the second COVID-19 vaccine to pass EMA scrutiny, after the agency issued a positive opinion on Pfizer Inc./Biontech SE’s Comirnaty on Dec. 21. “This vaccine provides us another tool to overcome the current emergency,” said Emer Cooke, executive director of the EMA, announcing the approval. “It is a testament to the efforts and commitment of all involved that we have this second positive vaccine recommendation just short of a year since the pandemic was declared by the World Health Organization,” she said.

Penciling in the right base corrects progeria mutation

Using an adenine base editor, researchers at the U.S. NIH, Vanderbilt University Medical Center and Harvard University have succeeded in treating the premature aging syndrome Hutchinson-Guilford progeria syndrome (progeria) in mice, extending the animals’ lifespan and preventing much of the vascular damage that is frequently the cause of death in children with progeria. The team also demonstrated successful editing in fibroblasts from children with progeria. They published their work in the Jan. 5, 2021, online issue of Nature. Wilbert Vermeij, a postdoctoral fellow at the Princess Maxima Center for Pediatric Oncology and the co-author of an editorial that accompanied the paper, told BioWorld that the paper described “a highly promising new development, that could hopefully significantly impact lives of progeria patients.”

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