On May 13, 2020 NGM Biopharmaceuticals, Inc. (NGM) (Nasdaq: NGM), a biotechnology company focused on developing transformative therapeutics for patients, reported financial results for the period ending March 31, 2020 (Press release, NGM Biopharmaceuticals, MAY 13, 2020, View Source [SID1234557905]).
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"In the first quarter of 2020, we demonstrated strong execution across our broad pipeline in multiple therapeutic areas. As we continue to navigate the ever-evolving COVID-19 situation, we have been fortunate to continue to move forward with our programs, most notably with the initiation of our Phase 2b ALPINE 4 study of aldafermin in patients with compensated cirrhosis due to NASH, a very sick patient population for which there is no currently available treatment other than liver transplant," said David J. Woodhouse, Ph.D., Chief Executive Officer at NGM. "As previously announced, in the first quarter of 2020, we also initiated a Phase 1a/1b clinical study for NGM120 as a potential treatment for cancer and cancer anorexia/cachexia syndrome, or CACS, and a Phase 1 clinical study for NGM395 as a potential treatment for metabolic disease, bringing us to a total of six product candidates now in clinical development. As we advance our clinical programs during these uncertain and unsettling times, the safety and well-being of patients, healthcare workers and our employees remain our top priority. We are closely monitoring the impact of COVID-19 on our organization and business operations and, like others in our industry, are managing multiple challenges to mitigate disruptions in our ongoing and planned trials in order to remain on track with our development timelines. The high unmet medical needs targeted by each of our development programs provide strong motivation for us to remain focused on execution across our pipeline."
Throughout the unfolding COVID-19 situation, NGM has worked proactively to establish policies that are designed to enable the company to operate safely, efficiently and productively, preserving mission-critical functions necessary to advance key research and development activities while safeguarding the well-being of patients, study investigators, clinical research staff and NGM employees. For patients already enrolled in NGM clinical trials, the company is working closely with investigators and site staff to continue treatment in compliance with study protocols and to uphold trial integrity, while observing government and institutional guidelines. NGM is continuing to evaluate site initiations and patient enrollment on a case-by-case and patient-by-patient basis in close coordination with investigators and site staff. Some sites, both within and outside of the United States, continue to screen patients for studies, and new patients are being enrolled when appropriate. These internal and external efforts have allowed NGM to continue progress across its clinical development programs. While NGM has experienced a slower pace of site initiation and trial enrollment than originally anticipated in certain of its clinical studies, the impact of the COVID-19 pandemic, to date, has not resulted in a significant impact to the company’s development timelines.
"At this time, we remain on track with all previously provided clinical trial timing guidance and will continue to monitor screening, enrollment and site initiations across our pipeline to understand any potential future impact on timing," said Dr. Woodhouse. "I admire and am extremely grateful for the dedication and agility of our team, the ongoing commitment of study investigators and clinical study site staff, and the broader ecosystem that is enabling important research and development work to continue at NGM and across our industry."
Key First Quarter and Recent Highlights
Cardio-metabolic and liver disease
Initiated Phase 2b ALPINE 4 study of aldafermin in compensated NASH cirrhosis (F4). In March 2020, NGM dosed the first patient in the dose-ranging ALPINE 4 study to evaluate the safety and efficacy of aldafermin versus placebo in patients with biopsy-confirmed NASH cirrhosis. The primary efficacy objective is to evaluate the treatment effect on histology, defined as fibrosis regression of at least one stage without worsening of NASH. This global, multi-center study is expected to enroll approximately 150 patients who will be dosed with 0.3 mg, 1 mg, 3 mg of aldafermin or placebo for 48 weeks. Aldafermin is wholly-owned by NGM.
"We are pleased to have initiated our Phase 2b ALPINE 4 clinical study and thrilled we have achieved this significant milestone, marking our first study in F4 NASH patients with well-compensated cirrhosis," said Hsiao D. Lieu, M.D., Chief Medical Officer at NGM. "We anticipate that the COVID-19 pandemic will impact activation of additional trial sites, and we plan to work closely with our target sites to navigate their processes and needs in an effort to mitigate delays. Reversing fibrosis and bringing advanced stage NASH patients back from the brink of liver transplant could have a profound, potentially life-saving impact. Based on the rapid, robust anti-fibrotic treatment effect we have seen with aldafermin to date in F2 and F3 NASH patients, we are encouraged by the potential to see activity in a patient population facing a particularly critical need for effective therapeutic solutions."
Continued enrollment in Phase 2b study of aldafermin in NASH patients with Stage 2 or 3 (F2-F3) fibrosis. NGM has continued to enroll patients in the Phase 2b ALPINE 2/3 clinical study in patients with biopsy-confirmed NASH and F2-F3 liver fibrosis. The 24-week study is designed to enroll approximately 150 patients and will assess the efficacy, safety and tolerability of 0.3 mg, 1 mg and 3 mg doses of aldafermin compared to placebo. Despite a lower-than-anticipated pace of enrollment as a result of COVID-19, NGM expects to announce topline data from the study in the first half of 2021, as previously guided. However, the extended impact of COVID-19 on our timeline is difficult to predict.
Announced positive preliminary topline liver histology and biomarker data from 24-week Phase 2 study of aldafermin 1 mg in patients with NASH (Cohort 4). In February 2020, NGM announced positive preliminary topline results from a 24-week double-blind, randomized, placebo-controlled Phase 2 clinical study (Cohort 4) of aldafermin in NASH patients with F2-F3 fibrosis. Cohort 4 was the final reported cohort from NGM’s adaptive Phase 2 clinical study of aldafermin in NASH. Cohort 4 was powered to demonstrate the effect of 1 mg aldafermin treatment versus placebo on the primary endpoint of change in absolute liver fat content, which achieved statistical significance. In addition, the study assessed secondary and exploratory endpoints of liver histology and biomarkers of disease activity. The histology results revealed that treatment with aldafermin led to clinically meaningful improvements at 24 weeks versus placebo in fibrosis improvement of ≥1 stage with no worsening of NASH (38% of aldafermin-treated patients vs. 18% placebo) and in resolution of NASH with no worsening of liver fibrosis (24% of aldafermin-treated patients vs. 9% placebo). The study also demonstrated a statistically significant impact on the composite endpoint of both fibrosis improvement and resolution of NASH (22% in aldafermin-treated patients vs. 0% placebo). In the study, aldafermin continued to demonstrate a favorable tolerability profile.
Initiated Phase 1 study of NGM395 in overweight and obese healthy adults. As announced in March 2020, NGM initiated a Phase 1 single ascending dose clinical study evaluating the safety, tolerability and pharmacokinetics of NGM395, a long-acting growth differentiation factor 15 (GDF15) analog, in overweight and obese but otherwise healthy adults. NGM395 is wholly-owned by NGM.
Ophthalmic disease
Completed enrollment in Phase 1 study of NGM621 for the potential treatment of geographic atrophy (GA), an advanced dry form of age-related macular degeneration (AMD). The Phase 1 clinical study is designed to evaluate the safety, tolerability and pharmacokinetics of up to two intravitreal doses of NGM621 in patients with GA. NGM621 is an inhibitory antibody binding complement C3, a key node of all three complement pathways. NGM plans to present the Phase 1 results at a future scientific congress and to initiate a Phase 2 study in the second half of this year.
Cancer
Initiated Phase 1a/1b study of NGM120 for the potential treatment of CACS and cancer. As announced in February 2020, NGM initiated a Phase 1a/1b clinical study to evaluate NGM120, a first-in-class antagonistic antibody that binds glial cell-derived neurotrophic factor receptor alpha-like, or GFRAL, and inhibits GDF15 signaling, for the potential treatment of CACS and cancer. CACS is the uncontrolled wasting of both skeletal muscle and fat that is a common co-morbidity of cancer and is associated with shortened survival in cancer patients.
Merck Collaboration
Merck has a one-time option to license NGM pipeline programs, other than aldafermin and NGM395, following human proof-of-concept trials, under the terms of the companies’ ongoing strategic collaboration. Upon exercising any such options, Merck would lead global product development and commercialization for the resulting products, if approved. Prior to Merck initiating a Phase 3 study for a licensed program, NGM may elect to either receive milestone and royalty payments or, in certain cases, to co-fund development and participate in a global cost and revenue share arrangement of up to 50%. The agreement also provides NGM with the option to participate in the co-promotion of any co‑funded program in the United States.
First Quarter Financial Results
For the quarter ended March 31, 2020, NGM reported a net loss of $19.1 million compared to a net loss of $8.3 million for the corresponding period in 2019.
Related party revenue from our collaboration with Merck for the quarter ended March 31, 2020 was $24.4 million compared to $25.6 million for the corresponding period in 2019.
Research and development expenses for the quarter ended March 31, 2020 were $38.4 million compared to $29.5 million for the corresponding period in 2019. The increase in research and development expenses was primarily attributable to increases in external research and development expenses associated with the advancement of NGM’s growing pipeline, including aldafermin program expenses for Phase 2b clinical trials, and personnel-related expenses driven by increased headcount.
General and administrative expenses for the quarter ended March 31, 2020 were $6.6 million compared to $5.4 million for the corresponding period in 2019. The increase in general and administrative expenses was primarily attributable to increases in personnel-related expenses driven by increased headcount, insurance expenses, consulting expenses and other professional service expenses required to support NGM’s operations as a public company.
Cash, cash equivalents and short-term marketable securities were $328.5 million as of March 31, 2020, compared to $344.5 million as of December 31, 2019.