Biotech and pharmaceutical companies generally are involved in the lengthy process of getting their drug candidates to market through clinical trials. The amount of risk involved ranges from fair to great, should a study come back negative or a candidate not be approved. Some companies can see massive upside just from winning a single midstage trial or even achieving as much as a Fast Track designation from the U.S. Food and Drug Administration (FDA).
In simpler terms, these updates within the industry have the potential to make or break biopharma companies.
Here, 24/7 Wall St. includes a calendar of a few of the biggest companies expecting clinical trial and FDA updates in the first quarter of 2019.
It’s worth mentioning that these dates may be subject to change due to various outside and internal factors, or some of these dates may have changed already. Some date changes are positive developments, while some can be disasters if a company is deeply financed.
As a side note about the Prescription Drug User Fee Act (PDUFA): a Priority Review designation is granted to medicines that the FDA determines have the potential to provide significant improvements in the treatment, prevention or diagnosis of a disease.
Proteostatis Therapeutics Inc. (NASDAQ: PTI) previously released preliminary results from its ongoing Phase 1 cystic fibrosis (CF) study in the fourth quarter of 2018. Now final results from its fourth and highest dose cohort, patients receiving PTI-801 and PTI-808, are expected in the first quarter of 2019. Phase 1 full data from the triplet PTI-428 + PTI-801 + PTI-808 are expected as well. For some perspective, when the company last announced these results, shares jumped about 450% in a single day.
Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) is another firm with a CF pipeline. In just the third quarter of 2018 the company saw revenues of roughly $783 million from its CF products. Looking ahead to the first quarter of 2019, the company expects to see Phase 3 data from its triplet VX-445 in combination with tezacaftor and ivacaftor in patients with CF.
Supernus Pharmaceuticals Inc. (NASDAQ: SUPN) previously released Phase 3 data from two of its trials in children with attention deficit hyperactivity disorder (ADHD) in early December. While this met primary endpoints, there were concerns regarding effect size versus competition. Data from the first adolescent trial at end of December met its primary endpoint as well, but this still was a case of “sell the news.” The firm is expecting data from its second Phase 3 trial due in the first quarter of 2019.
AC Immune S.A. (NASDAQ: ACIU) initiated its Phase 2 study of ACI-24 in patients with mild Alzheimer’s disease in the third quarter of 2018. The Phase 2 trial will be conducted in several European countries, and the first patients have been screened. Results from this study are expected to be presented in the first quarter of 2019.
Zafgen Inc. (NASDAQ: ZFGN) is on track to release its midstage topline results for patients with diabetes in the first quarter of 2019. This is a Phase 2 proof-of-concept clinical trial exploring ZGN-1061’s full MetAP2 target engagement efficacy.
Sage Therapeutics Inc. (NASDAQ: SAGE) has a PDUFA goal date scheduled for March 19, 2019, for its New Drug Application (NDA) of Zulresso (brexanolone). Zulresso is an injection for the treatment of postpartum depression. This goal date was originally extended from December 19, 2018, but the FDA has not asked for any additional clinical data or information. Sage is also expecting Phase 3 clinical data from SAGE-217 in postpartum depression in January of 2019.
Bausch Health Companies Inc. (NYSE: BHC) received a Complete Response Letter from the FDA in June 2018 for Duobrii (halobetasol propionate and tazarotene) for the treatment of psoriasis. The firm resubmitted the NDA for Duobrii with a PDUFA action date of February 15, 2019.
Axovant Sciences Ltd. (NASDAQ: AXON) expects to see data from its Phase 1/2 trial for patients with Parkinson’s disease. The firm dosed its second patient in the midstage clinical trial of AXO-Lenti-PD in November 2018, with data expected in March 2019.
Solid Biosciences Inc. (NASDAQ: SLDB) is looking to release initial data from its Phase 1/2 study of SGT-001 in patients with Duchenne muscular dystrophy in the first quarter of 2019. The firm also said that it remains on track to provide data from its previously communicated interim analysis in the second half of 2019.