Officials from Strongbridge Biopharma plc plan to make their case to the FDA about the potential for an accelerated approval pathway for Recorlev (levoketoconazole) to treat endogenous Cushing's syndrome after the open-label, single-arm SONICS study achieved statistical significance on the pre-specified primary endpoint.
Akouos Inc. raised $50 million in a tranched series A financing co-led by 5AM Ventures and New Enterprise Associates (NEA), which first committed to the newly formed gene therapy firm in a $7.6 million seed round in November 2017. Existing seed investor Partners Innovation Fund and new investors Sofinnova Ventures, RA Capital Management and Novartis Venture Fund joined the series A.
"What you have in this collaboration is two very like-minded, scientifically driven organizations that are coming together to bring very synergistic and complementary technologies to the field of CAR T cells and T-cell-based immunotherapy," said Philip Gregory, chief scientific officer of Bluebird Bio Inc., describing a five-year pact with Regeneron Pharmaceuticals Inc. to discover, develop and commercialize cancer cell therapies.
It's been a long trip down the rabbit hole for Insmed Inc. to move ALIS (amikacin liposome inhalation suspension) before an FDA advisory committee. Briefing documents for Tuesday's meeting of the Antimicrobial Drugs Advisory Committee (AMDAC) suggest the drug, formerly known as Arikayce and Arikace, may receive a balanced review for the treatment of nontuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC) in adults as part of a combination antibacterial approach.
Celtaxsys Inc. is already prepping the design of a phase III program for acebilustat after the oral leukotriene A4 hydrolase (LTA4H) inhibitor met with success in a phase II effort targeting treatment of cystic fibrosis (CF), irrespective of the causative genotype.
Biopharma veteran Geert Cauwenbergh, president and CEO of Rxi Pharmaceuticals Corp., has maintained steadfast faith in the potential of RNAi therapeutics even when the market has not. The Marlborough, Mass.-based RNAi company that he helms has struggled of late, forced in January to execute a 1-for-10 reverse stock split to regain compliance with Nasdaq's minimum bid price rule and to prioritize its pipeline by seeking to out-license dermatology and ophthalmology assets while focusing on immuno-oncology (I-O).
To the layperson, dry eye disease (DED), technically xerophthalmia, often is dismissed as an innocuous nuisance that can be treated with over-the-counter eye drops. But Anat Galor, an ophthalmologist in Miami and clinical expert with the American Academy of Ophthalmology (AAO), said the disease name "is a little bit of a misnomer" for a chronic indication that encompasses a variety of symptoms and can have serious consequences for patients. The multifactorial disease of the tears and ocular surface can result not only in burning and aching but also in visual disturbances and tear film instability that have the potential to damage the ocular surface.
Shares of Redhill Biopharma Ltd. (NASDAQ:RDHL) took a 14 percent bounce early Monday after the Tel Aviv, Israel-based company reported that the first phase III study of RHB-104 to test its hypothesis on Mycobacterium avium paratuberculosis (MAP) infection in Crohn's disease met its primary and key secondary endpoints. But those gains eroded, with shares closing at $8.75 for a loss of 68 cents, or 7.2 percent, after unanswered questions about the data, which remain under analysis, and the design of a follow-on phase III that company officials deemed likely once they have a chance to meet with the FDA.
"Like clockwork, Amgen just finds a way," Piper Jaffray's Christopher Raymond headlined his hot comment on second-quarter earnings from Amgen Inc. Numbers from the Thousand Oaks, Calif-based company were solid, if not spectacular. Quarterly revenues increased 4 percent, to $6.1 billion, over the second quarter of 2017, and global product sales, which included the company's first biosimilar, Kanjinti, grew 2 percent.