Shares of clinical-stage gene-therapy company, Adverum Biotechnologies, Inc. ADVM, have gained after the FDA lifted the clinical hold on the second cohort of phase I trial on lead candidate, ADVM-022.
Adverum’s shares have skyrocketed 187.3% year to date, significantly outperforming 1.6% growth registered by the industry.
The hold was lifted as the independent data monitoring committee (DMC) unanimously voted to proceed with dose escalation per protocol, following their review of the safety data from the first cohort. No patient in the first cohort has experienced a serious adverse event (SAE), with the first patient completing the 24-week (6 month) assessment.
The phase I study, OPTIC, is evaluating the candidate for age-related macular degeneration (wet AMD).
As a result, the study can now evaluate a higher dose of 2 x 10^12 vg/eye, which is three times the dose of 6 x 10^11 vg/eye evaluated in the first cohort.
However, Adverum will begin dosing in the second cohort with a lower dose of 2 x 10^11 vg/eye, which is three times lesser than the dosage in the first cohort based on the robust preliminary anatomical response observed to date.
The dosing of patients in the second cohort will start next month. Adverum is of the opinion that at a dose of 6 x 10^11 vg/eye, ADVM-022 has demonstrated the potential to provide sustained efficacy following a single intravitreal injection.
Meanwhile, ADVM-022 is on partial clinical hold for dosing patients with the highest dose of 6 x 10^12 vg/eye in the third cohort. The company does not plan to dose at this level, due to the robust preliminary anatomical response observed in the first cohort of patients. Nevertheless, Adverum will continue to work closely with the FDA to resolve the remaining chemistry, manufacturing and controls (“CMC”) comments.
Adverum intends to present 24-week primary and secondary outcomes from the first cohort of patients in the OPTIC trial in the second half of 2019.
We remind investors that the candidate was granted Fast Track designation by the FDA for wet AMD in September 2018.
The development of this candidate is crucial for the company as it discontinued the development of ADVM 043, a gene-therapy candidate for the treatment of alpha-1 antitrypsin (“A1AT”) deficiency, in November 2018.
The company also has collaboration agreements with Editas Medicine, Inc. EDIT and Regeneron Pharmaceuticals, Inc. REGN for other candidates.
The market potential of wet AMD is significant as it is a leading cause of vision loss in patients above 60 years of age. There are approximately 1.2 million individuals in the United States and 3 million globally affected by wet AMD. Approximately 150,000 to 200,000 people are estimated to be affected by wet AMD in the United States every year.
However, competition is stiff from the likes of Novartis NVS, among others.
Adverum currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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