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June 16, 2015 By Mark Terry , BioSpace.com Breaking News Staff Despite overall positive results, Avalanche Biotechnologies stock dropped after questions were raised regarding the company’s Phase II trial design for wet age-related macular degeneration. Avalanche Biotechnologies , located in Menlo Park, Calif., announced results yesterday of its Phase IIa study and a Phase I 36-month follow-up safety study for AVA-101. Avalanche indicated that the Phase IIa study met its 12-month primary endpoint, showing the drug was well tolerated and had a favorable safety profile. Patients also showed improved best corrected visual acuity (BCVA) compared to the control group, and demonstrated a positive trend in response rate. That all seems quite promising. “The results of this study confirm the Phase I safety results and suggest that AVA-101 could potentially benefit a significant portion of patients with wet AMD who require regular treatment with anti-VEGF therapy,” said Samuel Barone , Avalanche ’s chief medical officer in a statement. “The current standard of care in wet AMD requires frequent anti-VEGF infections, which present a significant burden for patients and their caregivers, and can result in reduced treatment compliance and under-treatment. Therefore, a product that can maintain or improve vision while reducing the number of treatment injections would represent a powerful new option for patients and physicians.” But something went wrong during the conference call yesterday afternoon when analysts began to examine more closely the study’s secondary efficacy endpoints. These were related to visual acuity, the number of rescue injections and measurements of retinal thickness. These, apparently, had significantly less clear results. In addition, analyst expressed concern that the way the study was designed did not show a statistically significant difference between the control arm and the AVA-101 patients. Also, there is some questions regarding a possible development deal between Avalanche and Regeneron Pharmaceuticals, Inc. for the rights to AVA-101 now. On May 5, 2014, Regeneron and Avalanche announced a collaboration to develop drugs for ophthalmologic diseases, particularly utilizing novel gene therapy vectors and proprietary molecules. As part of that deal, Regeneron has a time-limited right to first negotiation for AVA-101. Avalanche may provide more results at the American Academy of Ophthalmology (AAO) meeting in November about the drug and its possible deal with Regeneron . Tim Lugo , an analyst with William Blair , said on Benzinga , “We believe the performance of Lucentis in the control arm raises more questions than answers.” William Blair ’s report went on to say, “While the next major catalyst for shares is a potential deal between both Regeneron and Avalanche , it is likely beneficial for both parties to wait until Phase IIb results to enter discussions as both parties (and the market) will likely have difficulty in valuing AVA-101 as an asset until additional results are available.” Avalanche Biotechnologies stock was selling for $40.79 per share on June 11. It is currently selling for $18.75. It sold for a high of $60.08 on Jan. 7, 2015. William Blair downgraded the stock from Outperform to Market Perform . Suntrust also downgraded the study from Buy to Neutral . Jefferies maintained a Buy rating. On the other hand, analysts Joshua Schimmer , Steven Breazzano and Jerry Yang of Piper Jaffray are more positive overall about the company, noting in a research report, “the data is largely in line with our expectations, showing what we believe is real biological activity in a subset of responders with meaningful reduction in injection frequency, along with improvements in BCVA, but also with plenty of room for improvement.” They note that it’s still early and that the company’s overall “expanding gene therapy pipeline and vector evolution strategy… is underappreciated in our view.” After AstraZeneca CMO Abruptly Quits, Where Could He Be Headed? This week the chief medical officer of British drugmaker AstraZeneca PLC abruptly quit his post to become the chief executive officer of an unnamed, smaller biotech company. That’s lead BioSpace to wonder, with his background in R&D and in large companies like Pfizer Inc. , where will Briggs Morrison wind up? We want to know your thoughts. var _polldaddy = [] || _polldaddy; _polldaddy.push( { type: "iframe", auto: "1", domain: "biospace.polldaddy.com/s/", id: "after-astrazeneca-cmo-abruptly-quits-where-could-he-be-headed", placeholder: "pd_1434065855" } ); (function(d,c,j){if(!document.getElementById(j)){var pd=d.createElement(c),s;pd.id=j;pd.src=(' '==document.location.protocol)?' ':' ';s=document.getElementsByTagName(c)[0];s.parentNode.insertBefore(pd,s);}}(document,'script','pd-embed'));

Menlo Park’s Avalanche Biotechnologies Rolls Onto Nasdaq, Raises $102 Million For Its IPO July 31, 2014 By Krystle Vermes , BioSpace.com Breaking News Staff Avalanche Biotechnologies, Inc. , a California-based biotechnology company that focuses on developing gene therapy for ophthalmic diseases, announced today that it has raised $102 million in its IPO by pricing six million shares at $17 a piece. Originally, share projections were targeted between $13 and $15. Avalanche Biotechnologies’ shares are under the symbol “” on NASDAQ. The company is providing 5,400,000 shares in its initial offering. The offering is being led by Jefferies, Cowen, Piper Jaffray, and William Blair, who is the co-manager. Avalanche has given its underwriters a 30-day option to purchase up to 900,000 additional shares of common stock. Development partner and shareholder Regeneron made an agreement with Avalanche Biotechnologies to purchase $10 million worth of stock. Avalanche Biotechnologies was founded in 2006. The company focuses on creating unique gene therapies for severe eye diseases, and it will likely use its IPO income to propel its AVA-101 treatment for macular degeneration. Avalanche calls its form of gene therapy Ocular BioFactory. Patients who undergo treatment for macular degeneration receive an injection of AVA-101 into their eye during a brief surgical procedure. AVA-101 secretes therapeutic protein, which blocks the vascular endothelial growth factor in the eye and treats the disease. There is still skepticism surrounding Avalanche’s form of treatment and its level of safety. However, the company recently completed a Phase 1 trial in Australia that subjected eight macular degeneration patients to its treatment method with successful results. Currently, Avalanche is conducting Phase 2a which will expose 32 patients to its treatment. The results of this experiment will be released in 2015. In addition to putting its IPO money toward AVA-101, Avalanche is expected to use it on AVA-201, another preventative gene therapy for macular degeneration. Through its deal with Regeneron, the company is predicted to focus on the development of AVA-311 as well. This gene therapy would be used to treat X-linked retinoschisis, a rare eye disease that currently has no treatment. Gene therapy has not been embraced over the past 20 years. Today, there is only one gene therapy in the world that has been approved for use, sold by UniQure, and it is solely available in Europe. Avalanche is paving the way with its new forms of treatment that utilize adeno-associated viruses to address severe eye diseases, and it’s one of many companies using this method. Read more recent IPO news here . Help employers find you! Check out all the jobs and post your resume .