Tag Archive for: investigational drug

The Irish biopharma company did not provide specific data in Thursday’s announcement but said that compared with placebo “there was not a statistically significant decrease” in post-traumatic stress disorder (PTSD) symptoms in patients treated with JZP150. The investigational drug also failed key secondary endpoints, including clinician- and patient-assessed disease severity.

In a briefing document for Tuesday’s advisory committee meeting, the FDA raised concerns about the potential off-target effects of Vertex Pharmaceuticals and CRISPR Therapeutics’ investigational gene edited therapy.

The California pharma is building up to its first-ever approval with promising late-stage data for its once-daily investigational acromegaly pill paltusotine, an alternative to the injectable standard of care.

The FDA was on a roll in the first half of 2023, approving more than two dozen novel treatments. And if the first six months is any indication, the biopharma industry should expect several more novel therapies to be greenlit, including a few more first-in-class treatments.

Nearly two years after the FDA poured cold water on a potential regulatory bid for BrainStorm Cell Therapeutics’ NurOwn, the investigational ALS therapy will get a public hearing. 

Altimmune released mid-stage data Tuesday showing pemvidutide helped reduce patients’ weight by 10%, but the company’s shares tumbled 55% over safety concerns with the obesity drug.

Company president and CEO Bernard Coulie, M.D., Ph.D. called the data “unprecedented in IPF clinical drug development”.

AstraZeneca dosed the first patient in a Phase II portion of an ongoing trial assessing a PD-1/TIGIT bispecific antibody, triggering a $7.5 million milestone payment to partner, Compugen.

The investigational drug in question, fasinumab, was being evaluated in late-stage studies for the treatment of osteoarthritis of the knee or hip and chronic low back pain in patients with concomitant osteoarthritis of the knee or hip.