A partial restriction on tests of an experimental and well-financed gene therapy for Duchenne muscular dystrophy (DMD) gave way to a full FDA clinical hold following a serious adverse reaction in the first patient dosed. The boy has since recovered. But shares of Solid Biosciences Inc. (NASDAQ:SLDB), which raised $133.7 million in a January IPO despite a then-partial hold on the candidate, SGT-001, fell 64.6 percent to $9.32 by Thursday's close. Read More

In the two years since Ideaya Biosciences Inc. closed its $40 million series A, the South San Francisco-based company has grown from two to 42 employees, identified lead programs in synthetic lethality and immunotherapy, prepared to file three INDs in the next 12 to 18 months and positioned itself to move multiple candidates into the clinic by the end of 2019. That trajectory was more than sufficient to attract $94 million in a series B financing that will see the company into the second half of 2021, according to CEO Yujiro S. Hata. Read More

Alexion Pharmaceuticals Inc. is looking to the second half of 2018 as the target for regulatory filings in the U.S., EU and Japan after ALXN-1210, its long-acting C5 complement inhibitor, hit noninferiority to Soliris (eculizumab) across co-primary and the four key secondary endpoints in the pivotal phase III Study 301 in complement inhibitor treatment-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH). Read More

As 22,000 participants prepare to descend on Chicago next month for the American Association for Cancer Research (AACR) 2018 meeting, the organization released many of the abstracts for presentations. Unfortunately, as Evercore ISI analyst Umer Raffat wrote in a note to clients, "Most abstracts of significance are all embargoed." Read More

The primary takeaway from Thursday's Senate Health, Education, Labor and Pensions (HELP) Committee hearing on the 340B program is that the committee leadership is serious about crafting legislation to bring more accountability to the prescription drug discount program that helps safety-net hospitals and community health centers provide charity care. Read More

HONG KONG – Looking to improve regulatory coordination and supervision of drug and food markets, the Chinese government may merge together several agencies – including the China FDA (CFDA) – into an umbrella State Drug Administration (SDA) with national scope. Read More

The FDA is suspending indefinitely the January 2017 intended use rule for drugs, devices and biologics to allow further consideration of comments, according to a notice to be published in Friday's Federal Register.  Read More

Arcus Biosciences Inc., of Hayward, Calif., said it priced its IPO of 8 million shares at $15 per share, the high point of its pricing range, for gross proceeds of $120 million.  Read More

Destiny Pharma plc, of Brighton, U.K., said its lead clinical candidate, exeporfinium chloride (XF-73), has been granted fast track designation by the FDA for the prevention of postsurgical staphylococcal infections such as methicillin resistant Staphylococcus aureus. Read More

Tapimmune Inc., of Jacksonville, Fla., said phase I data published in Clinical Cancer Research showed that TPIV-200, its multi-epitope T-cell vaccine targeting folate receptor alpha (FRa), in patients with ovarian and breast cancer showed safety and long-lasting immune responses.  Read More