Randy Osborne

Takeda Pharmaceutical Co. Ltd.'s U.S. FDA go-ahead for Mimrylo (rusfertide) to treat erythrocytosis in adults with polycythemia vera (PV) came as little surprise, given the stellar phase III data that the hepcidin mimetic has yielded.

Takeda Pharmaceutical Co. Ltd.'s U.S. FDA go-ahead for Mimrylo (rusfertide) to treat erythrocytosis in adults with polycythemia vera (PV) gave partner Protagonist Therapeutics Inc. its second approval of the year. Takeda stands as “exceptionally well positioned” to make the most of Mimrylo in marketing the drug for PV, said Protagonist CEO Dinesh Patel on Aug. 31. “We expect Mimrylo to be used all across the PV patient population,” since the drug takes aim at erythrocytosis.

With an eye to diversifying beyond obesity, Eli Lilly and Co. made known its plan for the buyout of privately held Merida Biosciences Inc., investing as $2.875 billion in cash. The amount includes an up-front payment plus contingent milestone rewards, though a breakout of terms was not provided. Lilly, of Indianapolis, brings aboard Cambridge, Mass.-based Merida's lead program, MER-511, which has reached phase I development in Graves' disease and thyroid eye disease. Both are driven by thyroid-stimulating immunoglobulins, autoantibodies that activate the thyroid-stimulating hormone receptor.

Biohaven Ltd.'s quest for non-dilutive financing ended, at least for now, in an agreement with SK Biopharmaceuticals Co. Ltd., as the pair made known Aug. 26 their global licensing and collaboration in the busy epilepsy arena.

The previously debated matter of how best to order primary and secondary endpoints was not solved but became moot as Ultragenyx Pharmaceutical Inc. disclosed that the phase III Angelman syndrome trial called Aspire failed in both goals. Shares of Novato, Calif.-based Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) ended Sept. 3 at $14.84, down $11.68, or 44%, on word that the effort called Aspire with apazunersen (GTX-102) fell short of the primary endpoint – change from baseline in the Bayley-4 Cognitive raw score – along with the key secondary endpoint of net upside in the Multidomain Responder Index.

The U.S. FDA cleared Ionis Pharmaceuticals Inc.'s antisense therapy, Zanvastro (zilganersen), for Alexander disease (AxD) in pediatric and adult

How to get additional patients diagnosed and hit the estimated peak sales of $100 million for Zanvastro (zilganersen) became the next project for Ionis Pharmaceuticals Inc. after the U.S. FDA cleared the antisense drug for Alexander disease (AxD) in pediatric and adult patients, the first and only disease-modifying treatment for the ultra-rare, progressive and often fatal neurological disorder.

Novartis AG was hobbled by a pair of phase III trial failures, as delpacibart etedesiran fell short in the Harbor trial testing the antibody oligonucleotide conjugate in myotonic dystrophy type 1 (DM1) and pelacarsen, partnered with Ionis Pharmaceuticals Inc., fizzled in the cardiovascular (CV) outcomes Horizon study trying the antisense oligonucleotide in patients with elevated lipoprotein (a).

Aiming to “bend the curve toward longer disease-free survival” with improved safety in non-muscle-invasive bladder cancer (NMIBC), Tyra Biosciences Inc. CEO Todd Harris said the company has settled on a go-forward dose with oral dabogratinib in fibroblast growth factor receptor 3 (FGFR3)-altered, low-grade, intermediate-risk disease.

Upbeat data from an ongoing phase I/II trial put Opus Genetics Inc. on the road to pivotal work with gene therapy OPGx-BEST1, designed to treat a pair of rare eye diseases caused by mutations in the BEST1 gene: Best vitelliform macular dystrophy and autosomal recessive bestrophinopathy. Nothing is approved for either condition.