- Trial did not meet primary endpoint of statistically significant rate of change in GA lesion area using slope analysis over 52 weeks for NGM621 versus sham
- NGM621 showed favorable safety and tolerability, with no evidence of increased CNV conversion and no treatment-related SAEs
- Additional analyses to be presented in early November at The Retina Society Annual Scientific Meeting
SOUTH SAN FRANCISCO, CA, USA I October 17, 2022 I NGM Biopharmaceuticals, Inc. (NGM Bio) (Nasdaq: NGM), a biotechnology company focused on discovering and developing transformative therapeutics for patients, today announced topline efficacy and safety results from its randomized, double-masked, sham-controlled CATALINA Phase 2 trial of NGM621 in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). NGM621 is a humanized immunoglobulin G1 (IgG1) monoclonal antibody designed to inhibit activity of complement component 3 (C3). Over 52 weeks of treatment, NGM621 administered every four weeks (Q4W) (n=108) and every eight weeks (Q8W) (n=104) via intravitreal injection demonstrated a reduction in the rate of change in GA lesion area (slope) of 6.3% and 6.5%, respectively, compared to sham (n=106), which did not reach statistical significance in either arm. NGM621 demonstrated a favorable safety profile, with no evidence of increased choroidal neovascularization (CNV) conversions and numerically fewer cases of CNV in NGM621-treated patients compared to sham. In addition, there were no drug-related serious adverse events (SAEs).
“We’re disappointed that the CATALINA study did not meet its primary endpoint, particularly given the significant unmet medical need of patients impacted by GA,” said David J. Woodhouse, Ph.D., Chief Executive Officer at NGM Bio. “We continue to evaluate various pre-specified secondary endpoints and post-hoc analyses. We expect these additional findings, as well as the absence of treatment-related CNV conversion and the overall clean safety profile NGM621 showed in CATALINA, to provide important information regarding the treatment of patients with GA.”
“Through data from CATALINA, I believe we can gain insights not only related to NGM621, but also importantly into strategies to optimize clinical development programs for the GA field more broadly. As we have seen repeatedly, there can be high variability in outcomes among trials of complement inhibitors for GA, and we are still learning how such variability impacts the evaluation of therapeutics for this devastating disease process,” said Charles C. Wykoff, M.D., Ph.D., Director of Research at Retina Consultants Texas and an investigator for the CATALINA study. “While the CATALINA trial did not meet its primary endpoint, the notable CNV finding combined with signals from secondary and exploratory analyses suggest the possibility that NGM621 may have a role in treating GA. I look forward to evaluating further details from this trial and presenting the findings at the upcoming Retina Society Annual Meeting in November.”
CATALINA Phase 2 Study Safety Findings
NGM621 demonstrated a favorable safety and tolerability profile, with no evidence of increased treatment-related CNV conversions compared to sham and numerically fewer cases of CNV in NGM621-treated patients compared to sham. There were also no cases of endophthalmitis (infection of the eye) or optic ischemic neuropathy, a low overall rate of inflammation and, overall, no treatment-related SAEs. The most frequently reported ocular adverse events were those most likely to be attributed to the intravitreal injection procedure or to GA worsening.
“On behalf of the entire NGM Bio team, I’d like to thank the investigators and clinical trial staff involved in this study, our employees who contributed to this tremendous effort and, most importantly, the patients who participated in the study to help advance our understanding of NGM621 as a potential treatment for GA. We recognize the significant unmet need of GA patients for effective treatment solutions, and we remain hopeful that NGM621 may have the potential to demonstrate future clinical benefit,” said Hsiao D. Lieu, M.D., Chief Medical Officer at NGM Bio.
Conference Call / Webcast Details
NGM Bio will host a conference call and webcast with slide presentation at 8:00 a.m. ET (5:00 a.m. PT) today. To access the live webcast and slides, please visit the “Investors & Media” section of NGM Bio’s website at https://ir.ngmbio.com/. The webcast will be archived for 30 days.
NGM621 CATALINA Phase 2 Study Design
The Phase 2 CATALINA study enrolled 320 patients diagnosed with GA secondary to AMD in one or both eyes. The primary objectives of this multicenter, randomized, double-masked, sham-controlled study were to evaluate the efficacy and safety of NGM621 when given every four weeks or every eight weeks via intravitreal injections compared to sham injection control. Patients were randomized to one of four treatment groups in a ratio of 2:1:2:1 to receive intravitreal injections of NGM621 or sham injections every four weeks or every eight weeks for a total of 52 weeks with a final follow-up visit at 56 weeks. The primary efficacy endpoint was the rate of change in GA lesion area (slope), as measured by fundus autofluorescence imaging, over 52 weeks of treatment. The primary safety endpoints evaluated the incidence and severity of ocular and systemic adverse events from treatment with NGM621 compared to sham control.
Merck NGM621 Licensing Option
NGM621 falls within the scope of NGM Bio’s collaboration with Merck. Under the terms of the collaboration, following the CATALINA Phase 2 proof-of-concept trial, Merck has a one-time option to a worldwide, exclusive license for NGM621 and its related compounds, either alone or bundled with two additional undisclosed pre-clinical ophthalmology compounds and their related compounds. Merck is required to make its option decision in approximately three months. If Merck does not exercise its one-time option for NGM621, then NGM Bio will retain worldwide rights to NGM621, with a low, single digit royalty obligation to Merck if NGM Bio or a partner commercializes the program.
About Geographic Atrophy (GA)
GA is an advanced form of age-related macular degeneration characterized by progressive retinal cell loss that results in irreversible loss of vision. The disease affects approximately one million patients in the U.S. and five million patients globally. There are currently no treatments for GA approved by the FDA or the European Medicines Agency.
About NGM Bio
NGM Bio is focused on discovering and developing novel, life-changing medicines for people whose health and lives have been disrupted by disease. The company’s biology-centric drug discovery approach aims to seamlessly integrate interrogation of complex disease-associated biology and protein engineering expertise to unlock proprietary insights that are leveraged to generate promising product candidates and enable their rapid advancement into proof-of-concept studies. As explorers on the frontier of life-changing science, NGM Bio aspires to operate one of the most productive research and development engines in the biopharmaceutical industry. All therapeutic candidates in the NGM Bio pipeline have been generated by its in-house discovery engine, always led by biology and motivated by unmet patient need. Today, the company has seven programs in clinical development, including four in Phase 2 or 2b studies, including the recently completed NGM621 CATALINA trial, across three therapeutic areas: cancer, retinal diseases and liver and metabolic diseases. Visit us at www.ngmbio.com for more information.
SOURCE: NGM Bio
Post Views: 327
- Trial did not meet primary endpoint of statistically significant rate of change in GA lesion area using slope analysis over 52 weeks for NGM621 versus sham
- NGM621 showed favorable safety and tolerability, with no evidence of increased CNV conversion and no treatment-related SAEs
- Additional analyses to be presented in early November at The Retina Society Annual Scientific Meeting
SOUTH SAN FRANCISCO, CA, USA I October 17, 2022 I NGM Biopharmaceuticals, Inc. (NGM Bio) (Nasdaq: NGM), a biotechnology company focused on discovering and developing transformative therapeutics for patients, today announced topline efficacy and safety results from its randomized, double-masked, sham-controlled CATALINA Phase 2 trial of NGM621 in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). NGM621 is a humanized immunoglobulin G1 (IgG1) monoclonal antibody designed to inhibit activity of complement component 3 (C3). Over 52 weeks of treatment, NGM621 administered every four weeks (Q4W) (n=108) and every eight weeks (Q8W) (n=104) via intravitreal injection demonstrated a reduction in the rate of change in GA lesion area (slope) of 6.3% and 6.5%, respectively, compared to sham (n=106), which did not reach statistical significance in either arm. NGM621 demonstrated a favorable safety profile, with no evidence of increased choroidal neovascularization (CNV) conversions and numerically fewer cases of CNV in NGM621-treated patients compared to sham. In addition, there were no drug-related serious adverse events (SAEs).
“We’re disappointed that the CATALINA study did not meet its primary endpoint, particularly given the significant unmet medical need of patients impacted by GA,” said David J. Woodhouse, Ph.D., Chief Executive Officer at NGM Bio. “We continue to evaluate various pre-specified secondary endpoints and post-hoc analyses. We expect these additional findings, as well as the absence of treatment-related CNV conversion and the overall clean safety profile NGM621 showed in CATALINA, to provide important information regarding the treatment of patients with GA.”
“Through data from CATALINA, I believe we can gain insights not only related to NGM621, but also importantly into strategies to optimize clinical development programs for the GA field more broadly. As we have seen repeatedly, there can be high variability in outcomes among trials of complement inhibitors for GA, and we are still learning how such variability impacts the evaluation of therapeutics for this devastating disease process,” said Charles C. Wykoff, M.D., Ph.D., Director of Research at Retina Consultants Texas and an investigator for the CATALINA study. “While the CATALINA trial did not meet its primary endpoint, the notable CNV finding combined with signals from secondary and exploratory analyses suggest the possibility that NGM621 may have a role in treating GA. I look forward to evaluating further details from this trial and presenting the findings at the upcoming Retina Society Annual Meeting in November.”
CATALINA Phase 2 Study Safety Findings
NGM621 demonstrated a favorable safety and tolerability profile, with no evidence of increased treatment-related CNV conversions compared to sham and numerically fewer cases of CNV in NGM621-treated patients compared to sham. There were also no cases of endophthalmitis (infection of the eye) or optic ischemic neuropathy, a low overall rate of inflammation and, overall, no treatment-related SAEs. The most frequently reported ocular adverse events were those most likely to be attributed to the intravitreal injection procedure or to GA worsening.
“On behalf of the entire NGM Bio team, I’d like to thank the investigators and clinical trial staff involved in this study, our employees who contributed to this tremendous effort and, most importantly, the patients who participated in the study to help advance our understanding of NGM621 as a potential treatment for GA. We recognize the significant unmet need of GA patients for effective treatment solutions, and we remain hopeful that NGM621 may have the potential to demonstrate future clinical benefit,” said Hsiao D. Lieu, M.D., Chief Medical Officer at NGM Bio.
Conference Call / Webcast Details
NGM Bio will host a conference call and webcast with slide presentation at 8:00 a.m. ET (5:00 a.m. PT) today. To access the live webcast and slides, please visit the “Investors & Media” section of NGM Bio’s website at https://ir.ngmbio.com/. The webcast will be archived for 30 days.
NGM621 CATALINA Phase 2 Study Design
The Phase 2 CATALINA study enrolled 320 patients diagnosed with GA secondary to AMD in one or both eyes. The primary objectives of this multicenter, randomized, double-masked, sham-controlled study were to evaluate the efficacy and safety of NGM621 when given every four weeks or every eight weeks via intravitreal injections compared to sham injection control. Patients were randomized to one of four treatment groups in a ratio of 2:1:2:1 to receive intravitreal injections of NGM621 or sham injections every four weeks or every eight weeks for a total of 52 weeks with a final follow-up visit at 56 weeks. The primary efficacy endpoint was the rate of change in GA lesion area (slope), as measured by fundus autofluorescence imaging, over 52 weeks of treatment. The primary safety endpoints evaluated the incidence and severity of ocular and systemic adverse events from treatment with NGM621 compared to sham control.
Merck NGM621 Licensing Option
NGM621 falls within the scope of NGM Bio’s collaboration with Merck. Under the terms of the collaboration, following the CATALINA Phase 2 proof-of-concept trial, Merck has a one-time option to a worldwide, exclusive license for NGM621 and its related compounds, either alone or bundled with two additional undisclosed pre-clinical ophthalmology compounds and their related compounds. Merck is required to make its option decision in approximately three months. If Merck does not exercise its one-time option for NGM621, then NGM Bio will retain worldwide rights to NGM621, with a low, single digit royalty obligation to Merck if NGM Bio or a partner commercializes the program.
About Geographic Atrophy (GA)
GA is an advanced form of age-related macular degeneration characterized by progressive retinal cell loss that results in irreversible loss of vision. The disease affects approximately one million patients in the U.S. and five million patients globally. There are currently no treatments for GA approved by the FDA or the European Medicines Agency.
About NGM Bio
NGM Bio is focused on discovering and developing novel, life-changing medicines for people whose health and lives have been disrupted by disease. The company’s biology-centric drug discovery approach aims to seamlessly integrate interrogation of complex disease-associated biology and protein engineering expertise to unlock proprietary insights that are leveraged to generate promising product candidates and enable their rapid advancement into proof-of-concept studies. As explorers on the frontier of life-changing science, NGM Bio aspires to operate one of the most productive research and development engines in the biopharmaceutical industry. All therapeutic candidates in the NGM Bio pipeline have been generated by its in-house discovery engine, always led by biology and motivated by unmet patient need. Today, the company has seven programs in clinical development, including four in Phase 2 or 2b studies, including the recently completed NGM621 CATALINA trial, across three therapeutic areas: cancer, retinal diseases and liver and metabolic diseases. Visit us at www.ngmbio.com for more information.
SOURCE: NGM Bio
Post Views: 327
