Growing up in Jordan, where her mother ran the large University of Jordan Hospital, M3 Biotechnology Inc. President, CEO and co-founder Leen Kawas was ready to lead. She just didn't know it yet.
Blend Therapeutics Inc. said it will use a $38 million series C equity financing to carry a new lead candidate for neuroendocrine cancers into phase I studies by mid-year and what president and CEO Drew Fromkin told BioWorld Today will be "well through major data inflection points."
Dosing and new patient enrollment in all cohorts of Akashi Therapeutics Inc.'s phase Ib/IIa study of the FDA fast-tracked experimental Duchenne muscular dystrophy (DMD) medicine HT-100 (halofuginone) are being suspended after one of the patients receiving the highest study dose began experiencing serious, life-threatening health issues.
Missed efficacy endpoints in two phase III trials of Alkermes plc's lead candidate, the once-daily depression medicine ALKS 5461, dragged company shares down by 44.2 percent, to close at $33.71 on Thursday.
Cymabay Therapeutics Inc. said the FDA has agreed to designs for a trio of pivotal phase III studies to test the company's oral, once-daily gout candidate, arhalofenate, with Uloric (febuxostat, Takeda Pharmaceuticals America Inc.), a milestone that gives the company clarity that could help it launch the trials this year and secure a needed partner for the program as well.
A lack of reliable, timely and easy-to-access data is restricting the FDA's ability to stay on top of safety issues that it commits to tracking following expedited approval of new drugs, according to a report published by the Government Accountability Office (GAO) that examined both fast track and breakthrough designations.
FDA staff reviewing Sarepta Therapeutics Inc.'s eteplirsen (Exondys 51) expressed "considerable doubt" regarding how much the drug improves dystrophin production for patients with DMD and about whether improvements seen in small trials could be reliably attributed to the drug.
SAN FRANCISCO – The inaugural Digital Health Showcase, a start-up within the upstart Biotech Showcase meeting this week, highlighted what CBT Advisors founder and CEO Steve Dickman called "a kind of Cambrian explosion" of health care IT companies, still emerging with great speed and diversity and with little order imposed – something he sees as both "very exciting and very risky."
SAN FRANCISCO – FDA support for Mesoblast Ltd.'s plan to nearly halve the size of an ongoing phase III study in chronic heart failure and alter its endpoint will make the study significantly cheaper and faster to read out, while also making it more clinically and economically meaningful, CEO Silviu Itescu told BioWorld Today.
SAN FRANCISCO – Investors gathered to glean new details of the pending $160 billion combination of Pfizer Inc. and Allergan plc at the J.P. Morgan Healthcare Conference came away with little more than a better picture of each company's enthusiasm for the other and disappointment in outside appraisals of their pipelines.
