Biotech Values

[DewDiligence]

AGTC -8%/AH on report on ocular inflammation and slow enrollment in XLRS trial (buried in 2Q16 financial PR):

http://finance.yahoo.com/news/agtc-announces-financial-results-business-202248162.html

AGTC is currently enrolling patients in a Phase 1/2 clinical trial for XLRS, a program on which the company is collaborating with Biogen. The clinical protocol anticipates enrollment of up to 27 patients. This trial is currently being conducted at six clinical sites that specialize in inherited retinal diseases. As of August 2016, the company had enrolled a total of eight patients, six of whom are in the lowest dose level group and two are in the middle dose level group.

The primary endpoint of this trial is safety and safety data to date has shown that the XLRS product candidate has been generally well tolerated. AGTC has observed mild to moderate ocular inflammation in the majority of patients which resolved either without treatment or after treatment with topical or oral corticosteroids.

Enrollment in the study has been slower than planned. Delays resulted from patients not meeting one or more eligibility criteria and as a result, the company has taken steps to increase the number of clinical sites and to enhance its outreach programs. The company made a protocol amendment to include the use of prophylactic corticosteroids that required further institutional review board approvals. Vendor errors required the company to re-test the study agent for a process component. Upon re-testing, the study agent met all specifications and the company now has several backup vendors as well as internal testing capabilities. The company believes it has resolved these factors in order to meet its future enrollment goals and is executing on a plan to complete the trial expeditiously.

The company is measuring a wide range of secondary endpoints that might provide early indications of potential efficacy and help to design a subsequent trial. The company has not observed any significant improvements in secondary endpoints in the six patients treated at the lowest dose level group. The company does not expect to report further data on the XLRS study this year, but intends to provide quarterly updates on its patient enrollment.

Now we know why AGTC delayed reporting early data from this trial—a matter that was discussed on this board in late 2015 (#msg-119486843).