In its first big pharma deal since it was founded around a cell programming technology in 2009, Immusoft Corp. signed Takeda Pharmaceutical Co. Ltd. to a research collaboration and license option targeting rare inherited metabolic disorders. The agreement brings an undisclosed up-front fee and research funding to Immusoft, which is also eligible to earn more than $900 million if all options are exercised and all milestones hit.
DUBLIN – Shares in Idorsia Ltd. were off almost 4% Oct. 11 on news that lucerastat missed the primary endpoint of the Modify phase III trial in Fabry disease. The double-blinded, placebo-controlled study, which recruited 118 adult patients, was designed to evaluate the effect of lucerastat on neuropathic pain. Patients were randomized to drug or placebo in a 2-to-1 ratio.
Attralus Inc.’s $116 million series B will advance pan-amyloid removal (PAR) therapeutics, putting lead prospect AT-03 into a phase I biodistribution study soon in systemic amyloidosis patients while advancing other programs, including AT-01, an amyloid-specific imaging radiotracer for diagnosis in the same indication.
The FDA has put a hold on a clinical study of a rare disease gene therapy BMN-307 from Biomarin Pharmaceutical Inc. after several mice developed liver tumors following a high dose in a preclinical trial.
Ascendis Pharma A/S kept mum on the cost of just-approved Skytrofa (lonapegsomatropin) for pediatric growth hormone deficiency (GHD) – saying only that “premium responsible pricing” would be put in place – but Wall Street speculated freely about revenues likely due from the first-ever weekly injection treatment.
Atavistik Bio Inc. has raised $60 million in a series A financing round to advance its preclinical molecules targeting genetically validated targets in metabolic diseases and cancer.
Jnana Therapeutics Inc. closed a $50 million series B round and took the covers off its lead program, a small-molecule inhibitor of the solute carrier transporter SLC6A19, which it is prepping for clinical trials in phenylketonuria (PKU).
The new case of myelodysplastic syndrome (MDS) that overshadowed Bluebird Bio Inc.’s earnings and resulted in a clinical hold by the FDA will be addressed similarly to an earlier hitch in the sickle cell disease program, said Andrew Obenshain, the company’s head of severe genetic diseases. “Essentially, we will try and do the same thing,” and exonerate the lentiviral vector, he said during a conference call with investors. “At this point, we don't have tumor cells or leukemia to analyze,” he noted.
Rivus Pharmaceuticals Inc. has emerged from stealth and disclosed a $35 million series A round to fund development of its pipeline of “controlled metabolic accelerators,” or CMAs, for treating cardiometabolic diseases.
Researchers have identified an evolutionarily conserved metabolic role for tissue-resident macrophages, they reported in the July 2, 2021, issue of Science. In a commentary published alongside the paper, Conan O’Brien and Ana Domingos from the University of Oxford asserted that the work “introduces a new, macrophage-centered paradigm in… energy storage.”
RSS
