Five months after winning its first approval in Japan, Sanofi SA’s enzyme replacement therapy, Xenpozyme (olipudase alfa), earned a U.S. FDA nod for use in pediatric and adult patients with acid sphingomyelinase deficiency (ASMD), becoming the first medication designed to treat symptoms not related to the central nervous system.
In pursuit of "an opportunity to accelerate the establishment of clinical development and commercial capabilities in the U.K.," Neurocrine Biosciences Inc. said it will buy Diurnal Group plc for about £48.3 million (US$56.5 million). Cardiff, U.K.-based Diurnal is a specialty pharma developing hormone therapies for rare and chronic endocrine conditions. The all-cash transaction represented a 144% premium of the Aug. 26 closing price of Diurnal shares (LSE:DNL). Shares closed 134.7% higher Aug. 30 at £26.40.
Structure Therapeutics Inc. raised $33 million in a financing round to speed up clinical trials of its lead assets targeting chronic diseases and to improve its technology platform. Previously known as Shouti Inc., the company also has rebranded itself as Structure to “reflect its foundation in structural biology and computational design.”
The U.S. FDA’s summer 2021 approval of Skytrofa (lonapegsomatropin) from Ascendis Pharma A/S served to sharpen appetites for an even better therapy to treat for pediatric growth hormone deficiency (GHD). Among players in the forefront is Lumos Pharma Inc., with an oral candidate that could disrupt the competitive space.
Carmot Therapeutics Inc. has raised $160 million in series D financing to support a trio of early to midstage clinical programs focused on treating diabetes and obesity with peptide-based small-molecule incretin receptor modulators.
Verve Therapeutics Inc. has packed a lot into the past few weeks. The latest is a four-year research deal with Vertex Pharmaceuticals Inc. to find and develop an in vivo gene editing program for an undisclosed liver disease. Vertex will pick up the tab for program costs as Verve does the preclinical R&D. Verve is getting an up-front $60 million from Vertex, along with a $35 million equity investment.
The U.S. FDA has placed a clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating AT-845 following a serious adverse event of peripheral sensory neuropathy in one of the trial participants. AT-845 is an adeno-associated virus (AAV) gene replacement therapy being studied in adults with late-onset Pompe disease.
Investigators at Stanford University and Baylor College of Medicine have identified an exercise-induced appetite suppressant that led to weight loss when administered to obese mice. The molecule, Lac-Phe, has led to predictable excitement around the possibility of appetite-suppressing exercise in a pill.
New and updated preclinical and clinical data presented by biopharma firms at the Endocrine Society annual conference, including: Astellas, Bridgebio, Eiger, Horizon, Innovent, Neurocrine, Novo, Orphagen, Rhythm, Versanis.
The Cellular, Tissue and Gene Therapies Advisory Committee scrutinized Bluebird Bio Inc.’s gene therapy elivaldogene autotemcel (eli-cel) for early active cerebral adrenoleukodystrophy (CALD) in patients without a matched sibling donor.
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