Now that Ionis Pharmaceuticals Inc. and affiliate Akcea Therapeutics Inc. have gained FDA clearance – albeit delayed and with a black box warning – of antisense oligonucleotide Tegsedi (inotersen) for polyneuropathy of hereditary transthyretin (ATTR)-mediated amyloidosis in adults, speculation has begun about the drug's chances in the marketplace given the double-stranded small interfering RNA drug Onpattro (patisiran) from Alnylam Pharmaceuticals Inc., which won the FDA's go-ahead in August for the same indication. Read More
A U.S. subsidiary of Italy's privately held Leadiant Biosciences SpA has won FDA approval for Revcovi (elapegademase-lvlr), a new recombinant enzyme replacement therapy (ERT) for the ultra-rare genetic disorder adenosine deaminase severe combined immune deficiency. The condition, which affects as few as one in 1 million live births in the U.S., leaves infants and young children vulnerable to infections and can be fatal if not diagnosed and treated by age 2. Read More
LONDON – Acacia Pharma Group plc vowed to carry on with 2019 U.S. launch plans for its postoperative nausea and vomiting (PONV) product Barhemsys, despite receiving a complete response letter (CRL) from the FDA. Read More
DUBLIN – For many chasing the newest new thing in biotech, the tricarboxylic acid (TCA) cycle was something learned during an undergraduate biochemistry module and then promptly forgotten about. But startup firm Sitryx Therapeutics Ltd. is among an emerging minicluster of ventures that is going back over that well-trodden ground and exploring its links with immunology, in an effort to find a new generation of immune-acting drugs for cancer and inflammation. Read More
LONDON – NHS England has had a change of heart and agreed to fund treatment with Gilead Sciences Inc.'s Yescarta CAR T immunotherapy, after the company presented fresh evidence and agreed to offer a discount. Read More
SAN FRANCISCO — Summer's definitive failure of Summit Therapeutics plc's former lead candidate, ezutromid, led to disappointments across the Duchenne muscular dystrophy (DMD) community. Now with its pivot to antibiotics complete, its team expects to have one of just two novel mechanisms in the field's phase III lineup next year with ridinilazole. (See BioWorld, June 28, 2018.) Read More
PERTH, Australia – Dimerix Ltd. has begun recruiting patients for two separate phase II trials of its kidney disease candidate, DMX-200, in diabetic kidney disease and in orphan disease focal segmental glomerulosclerosis (FSGS). The Perth, Australia-based company hopes to replicate compelling data it saw in a subgroup of patients with type 2 diabetes, which could significantly open up the potential patient population for the drug. Read More
SAN FRANCISCO - With Motif Bio plc's new drug application for iclaprim in acute bacterial skin and skin structure infections awaiting a February 2019 review at the FDA, the company leveraged IDWeek to unveil new wound care data from its pivotal trials highlighting a potential advantage in the realm of kidney safety. Read More
Sarepta Therapeutics Inc., of Cambridge, Mass., established a long-term manufacturing agreement with Paragon Bioservices Inc., of Baltimore, to manufacture its microdystrophin Duchenne muscular dystrophy gene therapy and future gene therapy programs targeting diseases such as limb-girdle muscular dystrophy. Terms of the agreement weren't disclosed. Read More
Epizyme Inc., of Cambridge, Mass., closed its public offering of 9.6 million shares at $9 per share, which included 1.25 million shares issued through the underwriter's option, grossing the company $86.25 million. The company plans to use the proceeds to fund development and commercialization costs of tazemetostat outside of Japan, as well as for its other pipeline candidates, including EZM-8266, and for working capital and other general corporate purposes. Jefferies acted as book-running manager for the offering. Read More
Following a review of disabling and potentially long-lasting side effects associated with fluoroquinolone and quinolone antibiotics, the EMA's Pharmacovigilance Risk Assessment Committee (PRAC) Friday recommended that all drugs containing a quinolone antibiotic be removed from the market. Such drugs are authorized only for infections that should no longer be treated with this class of antibiotics, PRAC said. Read More
