Arrowhead Pharmaceuticals Inc. said it voluntarily paused a phase I/II study of its RNAi candidate for treating cystic fibrosis (CF) out of an “abundance of caution” while considering its next steps. “This may delay our pulmonary program a bit, but it’s just part of drug development,” said Christopher Anzalone, Arrowhead’s CEO. The halt was prompted by signals of local lung inflammation found in an ongoing chronic toxicology study in rats.
DUBLIN – Verona Pharma plc is adding $25 million cash to its balance sheet, as well as $15 million worth of stock in Nuance Biotech Co. Ltd., and could earn up to $179 million more in milestones linked to the development in the greater China region of ensifentrine, its candidate maintenance therapy for chronic obstructive pulmonary disorder (COPD).
Pieris Pharmaceuticals Inc. is banking $20 million up front and could receive up to $1.4 billion more in preclinical, clinical and commercial milestones from a multi-program collaboration and license agreement in respiratory disease and ophthalmology with Genentech.
HONG KONG – Splisense Ltd. has closed a $28.5 million series B financing that it said will support pipeline development including its lead antisense oligonucleotide candidate, SPL84-23, for the potential treatment of cystic fibrosis. The Jerusalem-based company, which develops mRNA-altering therapies for CF and other genetic pulmonary diseases, attracted support for the round from Orbimed, Israel Biotech Fund, Biotel Ltd. and the Cystic Fibrosis Foundation.
A new FDA approval for United Therapeutics Corp.'s prostacyclin analogue, Tyvaso (treprostinil), has expanded its label to include the treatment of pulmonary hypertension associated with interstitial lung disease to improve exercise ability. The regulatory win could double the number of patients taking the medicine by the end of 2022, barring any COVID-19-related delay, said company President and CEO Michael Benkowitz.
Unveiling short-of-the-mark top-line results from the phase IIb trial with MS-1819 in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (EPI), Azurrx Biopharma Inc. CEO James Sapirstein told investors during a conference call that “even if we had shown great success” in the study, more work had to be done for the enzyme to be commercialized. “We need to fix our formulation,” he said. “I’ve been saying this for a long time. It’s not a surprise to us.”
As the firm doubles down on a therapy for the rare and terrible lung disease called autoimmune pulmonary alveolar proteinosis (aPAP), Savara Inc. met with little trouble raising money, disclosing March 15 a public offering that grossed $130 million.
Shares of mRNA therapeutics specialist Translate Bio Inc. (NASDAQ:TBIO) fell 30.9% to $17.76 on March 18 over concerns about a new interim analysis of phase I/II data for its cystic fibrosis (CF) candidate, MRT-5005. Several dosing regimens of the inhaled candidate failed to improve a key measure of lung function vs. placebo, in contrast to an earlier readout on other doses tested in the ongoing study, called Restore-CF.
More than a decade after first approving Actemra (tocilizumab) for the treatment of rheumatoid arthritis, the FDA has added a sixth use to its label: slowing the rate of decline in pulmonary function in adult patients with systemic sclerosis-associated interstitial lung disease.
Asalyxa Bio Inc. has closed on an oversubscribed seed financing of more than $2 million designed to advance its lead candidate, ASX-100, into the clinic in acute respiratory distress syndrome.
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