Firm Reports Encouraging Trial Results in Cystinosis

New data from a Wedbush report shows that study patients experienced improvements in visual-motor integration, motor coordination, and visual perception. Streetwise Reports takes a deeper look at Avrobio Inc.

Avrobio Inc. (AVRO:NASDAQ) released encouraging data from the Phase 1/2 clinical trial of its AVRRD-04 gene therapy for cystinosis, reported Wedbush analyst David Nierengarten in a May 17 research note.

Against this backdrop, the life sciences firm’s shares continue to be undervalued, presenting an opportunity for investors. Along with an Outperform rating on AVROBIO, Wedbush has a $4 per share target price on it. This 12-month target implies a significant return as the company’s current share price, in comparison, is $0.78.

“We would be buyers ahead of regulatory and anticipated data updates this year,” the analyst added.

Cystinosis, caused by a defect of the CTNS gene, is a disease in which the amino acid cystine builds up in the body and crystalizes in the lysosomes, thereby inhibiting their ability to function. Disease symptoms can include kidney diseases and failure, muscular and endocrine disorders, neurodevelopmental issues, bone softening and weakening, and spatial and visual-motor dysfunction.

The only available treatment for cystinosis is cysteamine, which decreases the amount of cystine in the cells but does not stop disease progression.

The new data from the Phase 1/2 study of AVRRD-04 in cystinosis, reported at the recent annual American Society of Gene and Cell Therapy meeting, show that study participants experienced improvements in several functions, Nierengarten noted. These include motor coordination, visual perception, and handgrip. Some patients also showed improved or stable photophobia while some had a decrease in their cystine crystal buildup.

All patients remained off of oral cysteamine, and four stayed off of cysteamine eye drops. AVRRD-04 was well tolerated; no adverse events were reported.

“These data continue to confirm lentiviral-mediated gene therapy’s benefits for neurological manifestations of lysosomal storage diseases,” wrote Nierengarten.

Looking forward, after a meeting with the U.S. Food and Drug Administration later this year, AVROBIO plans to launch another trial of AVRRD-04 in pre-renal transplant patients and, subsequently, post-renal transplant patients.

“This trial should be a registration-enabling study in cystinosis and a key catalyst for AVROBIO,” Nierengarten commented.

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Disclosures for Wedbush, Avrobio Inc., May 17, 2022

WS makes a market in the securities of AVROBIO, Inc. The analysts responsible for preparing research reports do not receive compensation based on specific investment banking activity. The analysts receive compensation that is based upon various factors including WS’ total revenues, a portion of which are generated by WS’ investment banking activities. The information herein is based on sources that we consider reliable, but its accuracy is not guaranteed. The information contained herein is not a representation by this corporation, nor is any recommendation made herein based on any privileged information. This information is not intended to be nor should it be relied upon as a complete record or analysis: neither is it an offer nor a solicitation of an offer to sell or buy any security mentioned herein. This firm, Wedbush Securities, its officers, employees, and members of their families, or anyone or more of them, and its discretionary and advisory accounts, may have a position in any security discussed herein or in related securities and may make, from time to time, purchases or sales thereof in the open market or otherwise. The information and expressions of opinion contained herein are subject to change without further notice. The herein mentioned securities may be sold to or bought from customers on a principal basis by this firm. Additional information with respect to the information contained herein may be obtained upon request. Wedbush Securities does and seeks to do business with companies covered in its research reports. Thus, investors should be aware that the firm may have a conflict of interest that could affect the objectivity of this report. Investors should consider this report as only a single factor in making their investment decision. Please see pages 3–7 of this report for analyst certification and important disclosure information.

Analyst Certification
We, David Nierengarten and Dennis Pak, certify that the views expressed in this report accurately reflect our personal opinions and that we have not and will not, directly or indirectly, receive compensation or other payments in connection with our specific recommendations or views contained in this report.