Nine Biotech Companies Hope FDA Will Bend Rules in Their Favor

Persistent firms downplay weak trial data

Writing for The Street, columnist Adam Feurstein has identified nine biotech firms that hope to convince the FDA to approve their respective drugs by bending the rules or lowering current standards. Two companies, PTC Therapeutics and CytRx, even believe they can win FDA nods with submissions consisting entirely of negative data from failed studies, Feuerstein says.

Alkermes plans to supply the FDA with a clinical data package consisting of information from three phase 3 studies of ALKS-5461 in patients with treatment-resistant depression. Two of these studies failed to reach their primary endpoints, but Alkermes argues that the “totality” of the data shows that ALKS-5461 is effective. The company intends to present its findings to the FDA in the second quarter of this year.

CytRx hit bottom in July 2016 when its sarcoma drug aldoxorubicin failed a pivotal phase 3 trial. The company proceeded to delete half of the patients enrolled in the study and re-analyzed the data to produce an outcome showing that aldoxorubicin worked. A new drug application is expected to be filed in the fourth quarter.

Dynavax Technologies’ preventive hepatitis B vaccine Heplisav-B has been rejected twice by the FDA. The company recently resubmitted more data to the agency, hoping to overcome its objections. An approval decision is expected in August.

In September 2015, Intra-Cellular Therapies scored with its phase 3 trial of the schizophrenia drug ITI-007. One year later, however, the treatment flopped in another phase 3 investigation. Later this month, the company is meeting with the FDA to discuss its mixed findings and to obtain feedback on filing a new drug application.

Novan is also meeting with the FDA to discuss mediocre results from two phase 3 studies of its acne drug SB204. The company would like to seek approval of the compound in the first quarter of 2018 without having to conduct another study.

Portola Pharmaceuticals’ anticoagulant drug betrixaban failed a phase 3 study in March 2016, although an “exploratory” analysis of the same data showed a reduced risk for blood clots. The FDA accepted a new drug application for the drug, and told Portola that an advisory committee meeting would not be convened. Whether that was an encouraging or discouraging signal will become clear in June when the agency announces its approval decision.

PTC Therapeutics’ drug ataluren has never demonstrated a statistically significant benefit in studies of patients with cystic fibrosis or Duchenne muscular dystrophy (DMD) caused by nonsense mutations. Nevertheless, the company essentially forced the FDA to review ataluren in DMD by filing for approval “over protest.” The agency’s decision is expected in October.

Rigel Pharmaceuticals expects to submit a new drug application for fostamatinib in immune thrombocytopenia to the FDA by the end of March. The application, however, will consist of mixed data from phase 3 studies that achieved and missed primary endpoints.

Source: The Street; March 8, 2017.