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FDA Awards Breakthrough Therapy Status for AMD Drug
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Iveric Bio Inc. shares traded 25% higher Friday after the company reported that the U.S. FDA has granted breakthrough therapy designation for its avacincaptad pegol for use in the treatment of geographic atrophy secondary to age-related macular degeneration.

Biopharmaceutical company Iveric bio, Inc. (ISEE:NASDAQ), which concentrates its efforts on discovering and developing new treatments options for orphan inherited retinal diseases, Thursday announced that "the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for avacincaptad pegol (ACP, also known as Zimura®), a novel investigational complement C5 inhibitor for the treatment of geographic atrophy (GA) secondary to Age-Related Macular Degeneration (AMD)."

The company advised that the FDA's authorization was based upon its analysis of the 12-month primary endpoint data from its two pivotal Phase 3 GATHER1 and GATHER2 clinical trials. Iveric highlighted that during the combined GATHER clinical programs, ACP produced improvement in efficacy rates by up to 35%, which were shown to increase over time.

The firm pointed out that ACP is the first and only treatment to be assigned Breakthrough Therapy designation (BTD) for use in addressing GA secondary to AMD. Iveric Bio stated that BTD is issued for the purpose of accelerating development and the regulatory review process for new medicines that demonstrate potential for use in the treatment of serious conditions and diseases with high unmet medical need.

The company's CEO, Glenn P. Sblendorio, commented, "We believe this Breakthrough designation reflects the fact that both GATHER1 and GATHER2 met their primary endpoint with a safety profile that meets the stringent criteria required . . . We are now focused on the execution of our full NDA submission and launch preparation, with the possibility of being first to market."

"We look forward to working collaboratively with the FDA to expedite the review timeline for avacincaptad pegol and to potentially bringing a new therapy to AMD patients impacted by GA," Sblendorio added.

The company noted that as part of the rolling review process, it recently submitted the first part of a New Drug Application (NDA) for ACP to the FDA. The data filed with the NDA included the full clinical data gathered during the GATHER1 and GATHER2 trials. The firm stated that it expects to file the second and final part of the NDA by CYE/22.

Iveric Bio's President Pravin U. Dugel, M.D. remarked, "In both GATHER1 and GATHER2, avacincaptad pegol consistently showed a treatment effect with the first measurement at month six that was persistent and continued to increase over time, with observed efficacy rates of up to 35% . . . We believe ACP has the potential to safely and effectively preserve central vision by saving photoreceptor cells for patients living with this life-changing disease that leads to irreversible blindness."

"We believe ACP has the potential to safely and effectively preserve central vision by saving photoreceptor cells for patients living with this life-changing disease that leads to irreversible blindness," said President Dugel.

The company advised that the FDA's decision to authorize BTD was based upon 12-month primary efficacy endpoint data collected from the Phase 3 GATHER1 and GATHER2 clinical studies, which were established to measure the safety and efficacy of ACP in patients with GA located inside and/or outside of the clinical fovea.

The firm stated that the GATHER2 trial was designed to meet the demands of the FDA's special protocol assessment (SPA) request, which entailed keeping track and calculating "the mean rate of growth (slope) in GA area from baseline to month 12."

The company reported that after 12 months, these results demonstrated a significant treatment difference of 35% in the GATHER1 trial and 18% in the GATHER2 study compared to the respective control groups. Importantly, participants in both studies exhibited no serious incidents of endophthalmitis, intraocular inflammation, or vasculitis.

The company stated that "AMD is the major cause of moderate and severe loss of central vision in aging adults, affecting both eyes in the majority of patients." The disease affects the macula, which lies in the center of the retina and is responsible for central vision. Those diagnosed with AMD experience a thinning and loss of retinal pigment epithelial cells and underlying blood vessels and atrophy of retinal tissue in the macula. As geographic atrophy expands to advanced or late stages, even further irreversible vision loss occurs. The firm noted that currently, there are no approved treatments for patients with geographic atrophy secondary to AMD in either the U.S. or Europe.

Iveric Bio is a biopharmaceutical company based in Parsippany, N.J., that is involved in identifying and developing medicines for use in treating inherited orphan retinal diseases. The firm is primarily focused on creating safe and effective treatments for debilitating retinal diseases such as early-stage AMD.

The company's leading pipeline asset called Zimura® (avacincaptad pegol), is now being evaluated in two separate Phase 3 trials with combined enrollments of 486 individuals. The studies are both designed to determine the safety, efficacy, and viability of Zimura as a potential treatment for reducing or limiting geographic atrophy in patients with dry AMD.

The firm is also studying Zimura in its Phase 2b STAR clinical trial as a potential candidate for treating autosomal recessive Stargardt disease. The randomized study is expected to enroll a total of 120 patients who will be administered either a Zimura or sham injection for 18 months. The primary goal of the trial is to measure the change in ellipsoid zone defect as assessed by en face SD-OCT.

Iveric Bio started off today with a market cap of around US$2.61 billion, with approximately 120.72 million shares outstanding and a short interest of about 7.45%. ISEE shares opened 19% higher Thursday at US$20.54 (+US$3.30, +19.14%) over Wednesday's US$17.24 closing price. The stock closed for trading Friday at US$21.82 (+US$0.17, +79%). and trades today between US$20.23 and US$22.44 per share.


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