Avalanche Biotech Goes Back to Drawing Board, Performs More Preclinical Studies on Eye Drug
August 14, 2015
By Mark Terry, BioSpace.com Breaking News Staff
Menlo Park, Calif.-based Avalanche Biotechnologies announced yesterday that it’s going back to the drawing board with its AVA-101. Instead of continuing on with clinical trials of the drug for wet age-related macular degeneration (wet AMD), the company indicates it will go back and perform more preclinical testing.
On June 15, Avalanche announced results of its Phase IIa study and a Phase I 36-month follow-up safety study for AVA-101. They said that the Phase IIa study met its 12-month primary endpoint, the drug was well-tolerated and had a favorable safety profile. Patients showed improved best corrected visual acuity (BCVA) compared to the control group and demonstrated a positive trend in response rate.
However, later in the day during a conference call, analysts questioned the study’s secondary efficacy endpoints. Those endpoints studied visual acuity, number of rescue injections and retinal thickness. In addition to the unclear results for secondary endpoints, analysts expressed concern over the study design and a lack of statistically significant difference between the control arm and the AVA-101 patients.
This, in turn, put a possible development deal between Avalanche and Regeneron Pharmaceuticals, Inc. for the rights to AVA-101 in jeopardy. The two companies inked a collaboration deal in May 2014 to collaborate on developing drugs for ophthalmologic diseases, and as part of the deal, Regeneron had a time-limited right to first negotiation for AVA-101.
Shortly afterward, on July 24, Thomas Chalberg, Jr., chief executive officer and president of Avalanche, resigned. Hull replaced him as interim chief executive and president. Hull was formerly senior vice president of business operations. Chalberg is staying on as a consultant and member of the company’s Scientific Advisory Board.
After more analysis of the data, Avalanche decided to do more laboratory work. “These analyses gave us more information about some of the factors, including dosing and administration variability, that may have contributed to the Phase IIa study results,” said Hans Hull, interim chief executive officer of Avalanche in a statement. “We are carefully focusing our resources and leveraging our scientific expertise to develop what we believe could be a transformative treatment for wet AMD that will help patients better manage this devastating disease.”
Not surprisingly, has not responded well to all the turmoil. On June 12, 2015, shares traded for $41.40. On June 16, shares had dropped to $17.05. They continued to drift downward and on Aug. 13 traded for $13.83. Shares are currently trading for $10.96. Shares traded for a high of $60.89 on Jan. 7, 2015.
In addition to the announcement regarding the clinical trial change, Avalanche announced its second quarter financial results. It indicated cash, cash equivalents and marketable securities of $279.6 million, compared to $159.4 million as of Dec. 31, 2014. Avalanche said it expected its burn rate to stay about the same over the next 18 months. Revenue from collaborative research was $203,000 for the second quarter, compared to $135,000 for the same period in 2014.