Biotech Values

[DewDiligence]

Phase-2 Squalamine trial at U of Wisconsin:

This is GENR’s main phase-2 AMD trial testing Squalamine as a single agent. As this trial plans to enroll 100 patients, there are presumably several other sites too. Thanks to our regular iHub contributor, randychub, here is what we know about the Squalamine activity at Wisconsin:

http://wieyemd.ophth.wisc.edu/restrial.html#Phase
[Scan ¾ into the web page]

>>
A Phase 2, Multi-Center, Randomized, Masked, Controlled, Study of MSI-1256F (Squalamine Lactate) for the Treatment of Subfoveal Choroidal Neovascularization Associated with Age-Related Macular Degeneration

Principal Investigator: Barbara Blodi, MD
Study Coordinator: Alyson Skoldberg

Synopsis:
This study is designed to investigate the safety and efficacy of infusions of the anti-angiogenic drug Squalamine in the treatment of wet Age-Related Macular Degeneration. This is a 4:1 randomization trial that assigns participants to one of two dosages of Squalamine, or to one of two placebo groups. Patients will receive an infusion of Squalamine or placebo weekly for one month and then monthly for the following eleven months. Patients will also participate in three-month follow-up visits in the year after treatment has been completed.

Recruitment/Eligibility:
- Participants must be at least 50 years of age.
- Must have angiographically confirmed subfoveal choroidal neovascularization lesions in study eye [minimally classic to predominantly classic, OR active occult (deterioration of visual acuity by >=3 lines or 15 letters (ETDRS) within the past 6 months and/or presence of subretinal fluid and/or blood confirmed by OCT) CNV on fluorescein angiography], with the following angiographic criteria:

• Total lesion area (CNV and all components such as blood and fibrosis, excluding subretinal fluid or hard exudates) should be <=16 ETDRS disc areas.
• CNV under the geometric center of the foveal avascular zone.
• Area of fibrosis <25% of the area of the lesion.
• Area of subretinal blood <50% of the area of the lesion.
- May not have had previous treatment in study eye including photodynamic therapy, photocoagulation, injectable steroids or use of an antiogenic [sic] or anti-VEGF.
- Must have a visual acuity 20/32 to 20/200 in the study eye. The ETDRS score must be from 34-78 letters for the study eye.

For more information contact Alyson Skoldberg at [phone].
<<

--
Other than barring patients who have received prior treatment, the eligibility restrictions for this trial are minimal. For instance, all disease subgroups (predominantly classic, minimally classic, and occult) are admissible with the limitation being that occult patients must have disease that is progressing. This limitation is logical, because occult disease often becomes dormant, in which case no therapy is apt to provide a significant benefit.

Likewise, the eligibility restrictions on baseline lesion size (<=16 disk areas) and baseline visual acuity (20/32 to 20/200) are minimal, as the overwhelming majority of AMD patients meet these requirements.

The minimal eligibility restrictions (other than the requirement of no prior treatment), combined with the 4:1 randomization of the Squalamine arm to the placebo arm (making the likelihood of assignment to Squalamine 80%) should facilitate patient enrollment.

I like the fact that GENR is conducting this trial at such a high-quality institution as U of Wisconsin. Moreover, the PI, Dr. Barbara Blodi, is a well-known ophthalmology author (as you can see by googling her).

Now if we can just find out where else the study is being conducted!